0001558370-24-002196 | 10-K | XBRL Viewer

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Stock award plan - Stock Option Activity (Details) - Stock option - USD ($) $ / shares in Units, $ in Thousands	12 Months Ended
	Dec. 31, 2023	Dec. 31, 2022	Dec. 31, 2021
Number of options
Outstanding at the beginning of the period (in shares)	11,502,417	10,772,582	9,663,677
Granted (in shares)	1,117,284	1,685,435	2,487,234
Exercised (in shares)	(822,482)	(496,863)	(635,871)
Forfeited/Cancelled (in shares)	(2,196,820)	(458,737)	(742,458)
Outstanding at the end of the period (in shares)	9,600,399	11,502,417	10,772,582
Vested or Expected to vest at the end of the period (in shares)	1,796,687
Exercisable at the end of the period (in shares)	7,650,948
Weighted- average exercise price
Outstanding at the beginning of the period (in dollars per share)	$ 43.33	$ 43.66	$ 38.72
Granted (in dollars per share)	40.19	38.55	61.36
Exercised (in dollars per share)	29.25	29.45	28.01
Forfeited/Cancelled (in dollars per share)	45.85	48.75	52.04
Outstanding at the end of the period (in dollars per share)	43.59	$ 43.33	$ 43.66
Vested or Expected to vest at the end of the period (in dollars per share)	45.85
Exercisable at the end of the period (in dollars per share)	$ 43.08
Weighted- average remaining contractual term
Outstanding at the end of the period	5 years 4 months 20 days
Vested or Expected to vest at the end of the period	8 years 1 month 28 days
Exercisable at the end of the period	4 years 8 months 4 days
Aggregate intrinsic value (in thousands)
Outstanding at the end of the period (in dollars)	$ 8,814
Vested or Expected to vest at the end of the period (in dollars)	366
Exercisable at the end of the period (in dollars)	$ 8,387

Insider Trading Arrangements

3 Months Ended

Dec. 31, 2023

Trading Arrangements, by Individual

Material Terms of Trading Arrangement

Director and Officer Trading Arrangements

A portion of the compensation of our directors and officers (as defined in Rule 16a-1(f) under the Securities Exchange Act of 1934, as amended, or the Exchange Act) is in the form of equity awards and, from time to time, directors and officers engage in open-market transactions with respect to the securities acquired pursuant to such equity awards or other Company securities, including to satisfy tax withholding obligations when equity awards vest or are exercised, and for diversification or other personal reasons.

Transactions in Company securities by directors and officers are required to be made in accordance with our insider trading policy, which requires that the transactions be in accordance with applicable U.S. federal securities laws that prohibit trading while in possession of material nonpublic information. Rule 10b5-1 under the Exchange Act provides an affirmative defense that enables directors and officers to prearrange transactions in Company securities in a manner that avoids concerns about initiating transactions while in possession of material nonpublic information.

The following table describes, for the quarterly period covered by this report, each trading arrangement for the sale or purchase of Company securities adopted or terminated by our directors and officers that is either (1) a contract, instruction or written plan intended to satisfy the affirmative defense conditions of Rule 10b5-1(c), or a “Rule 10b5-1 trading arrangement”, or (2) a “non-Rule 10b5-1 trading arrangement” (as defined in Item 408(c) of Regulation S-K):



Name (Title)	Action Taken (Date of Action)	Type of Trading Arrangement	Nature of Trading Arrangement	Duration of Trading Arrangement	Aggregate Number of Securities
Mark Boulding (Chief Legal Officer)	Adoption (October 31, 2023)	Rule 10b5-1 trading arrangement	Sale	Until February 14, 2025 or such earlier date upon which all transactions are completed.	Up to 215,899 shares.
Matthew Klein (Chief Executive Officer)	Adoption (December 28, 2023)	Rule 10b5-1 trading arrangement for sell-to-cover transactions for RSUs granted on December28, 2023	Sale	Until final settlement of RSUs on or around December 29, 2026	Indeterminable (1)

(1) The number of shares subject to this RSU grant that will be sold to satisfy applicable tax withholding obligations upon vesting is unknown as the number will vary based on the extent to which vesting conditions are satisfied and the market price of the Company’s common stock at the time of settlement. This trading arrangement provides for the automatic sale of shares that would otherwise be issuable on each settlement date of the RSU in an amount sufficient to satisfy the applicable withholding obligation, with the proceeds of the sale delivered to the Company in satisfaction of the applicable withholding obligation.

Non-Rule 10b5-1 Arrangement Adopted

false

Common Stock Trading Arrangement | Mark Boulding

Trading Arrangements, by Individual

Name

Mark Boulding

Title

Chief Legal Officer

Rule 10b5-1 Arrangement Adopted

true

Adoption Date

October 31, 2023

Termination Date

February 14, 2025

Aggregate Available

215,899

Restricted Stock Unit Trading Arrangement | Matthew Klein

Trading Arrangements, by Individual

Name

Matthew Klein

Title

Chief Executive Officer

Rule 10b5-1 Arrangement Adopted

true

Adoption Date

December 28, 2023

Termination Date

December 29, 2026

Fair value of financial instruments and investments - Convertible senior notes (Details) - Convertible debt - 1.50% Convertible senior notes due 2026 - USD ($) $ in Thousands	Dec. 31, 2023	Dec. 31, 2022	Sep. 30, 2019
Long-term debt
Principal	$ 287,500	$ 287,500	$ 287,500
Interest rate ( as a percent )			1.50%
Significant other observable inputs (level 2)
Long-term debt
Fair value of convertible notes	$ 265,300	$ 281,700

- Definition

Line items represent financial concepts included in a table. These concepts are used to disclose reportable information associated with domain members defined in one or many axes to the table.

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+ Details

Fixed assets

12 Months Ended

Dec. 31, 2023

Property, Plant and Equipment [Abstract]

Fixed assets

4. Fixed assets

Fixed assets, net were as follows at December 31, 2023 and 2022:


	December 31,
	2023		2022
Leasehold improvements	$	30,166	$	28,969
Computer equipment and software		17,503		15,332
Machinery and lab equipment		62,837		47,496
Furniture and fixtures		3,849		3,812
Assets in process		24,008		14,349
		138,363		109,958
Less accumulated depreciation		(51,274)		(37,368)
Total	$	87,089	$	72,590

Depreciation expense was approximately $13.9 million, $12.3 million, and $9.4 million for the years ended December 31, 2023, 2022, and 2021, respectively.

Fair value of financial instruments and investments - Hierarchy (Details) - USD ($) $ in Thousands	Dec. 31, 2023	Dec. 31, 2022
Financial assets and liabilities measured at fair value on recurring basis
Marketable securities - available for sale	$ 260,104	$ 22,610
Contingent consideration payable	36,300	164,000
Transfers from Level 1 to Level 2	0	0
Transfers from Level 2 to Level 1	0	0
Recurring basis
Financial assets and liabilities measured at fair value on recurring basis
Marketable securities - available for sale	260,104	22,610
Marketable securities - equity investments	22,634	108,261
ClearPoint Equity Investments	6,074	10,965
ClearPoint convertible debt security	12,553	15,231
Recurring basis \| Quoted prices in active markets for identical assets (Level 1)
Financial assets and liabilities measured at fair value on recurring basis
Marketable securities - equity investments	22,634	108,261
ClearPoint Equity Investments	6,074	10,965
Recurring basis \| Significant other observable inputs (Level 2)
Financial assets and liabilities measured at fair value on recurring basis
Marketable securities - available for sale	260,104	22,610
ClearPoint convertible debt security	12,553	15,231
Recurring basis \| Development and Regulatory Milestones
Financial assets and liabilities measured at fair value on recurring basis
Contingent consideration payable	26,600	82,500
Recurring basis \| Development and Regulatory Milestones \| Significant unobservable inputs (Level 3)
Financial assets and liabilities measured at fair value on recurring basis
Contingent consideration payable	26,600	82,500
Recurring basis \| Net Sales Milestones and Royalties
Financial assets and liabilities measured at fair value on recurring basis
Contingent consideration payable	9,700	81,500
Recurring basis \| Net Sales Milestones and Royalties \| Significant unobservable inputs (Level 3)
Financial assets and liabilities measured at fair value on recurring basis
Contingent consideration payable	$ 9,700	$ 81,500

Geographic information (Details) $ in Thousands	12 Months Ended
Geographic information (Details) $ in Thousands	Dec. 31, 2023 USD ($) segment	Dec. 31, 2022 USD ($) segment	Dec. 31, 2021 USD ($)
Segment Reporting Information [Line Items]
Number of operating segments \| segment	1	1
Total assets	$ 1,895,698	$ 1,705,619
Fixed assets, net	87,089	72,590
Revenue	937,822	698,801	$ 538,593
United States
Segment Reporting Information [Line Items]
Total assets	1,582,962	1,473,770
Fixed assets, net	86,421	71,754
Revenue	531,661	381,862
Non-US
Segment Reporting Information [Line Items]
Total assets	312,736	231,849
Fixed assets, net	668	836
Revenue	$ 406,161	$ 316,939

- Definition

Sum of the carrying amounts as of the balance sheet date of all assets that are recognized. Assets are probable future economic benefits obtained or controlled by an entity as a result of past transactions or events.

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+ Details

Fair value of financial instruments and investments - Available-for-sale securities (Details) - USD ($) $ in Thousands	Dec. 31, 2023	Dec. 31, 2022
Debt Securities, Available-for-sale [Line Items]
Securities in an unrealized loss position less than 12 months - Unrealized losses	$ (12)
Securities in an unrealized loss position less than 12 months - Fair Value	44,446
Securities in an unrealized loss position greater than 12 months - Unrealized losses	(2)	$ (499)
Securities in an unrealized loss position greater than 12 months - Fair Value	1,648	10,186
Total - Unrealized losses	(14)	(499)
Total - Fair Value	46,094	10,186
Commercial paper
Debt Securities, Available-for-sale [Line Items]
Securities in an unrealized loss position less than 12 months - Unrealized losses	(12)
Securities in an unrealized loss position less than 12 months - Fair Value	44,446
Total - Unrealized losses	(12)
Total - Fair Value	44,446
Corporate debt securities
Debt Securities, Available-for-sale [Line Items]
Securities in an unrealized loss position greater than 12 months - Unrealized losses	(2)	(499)
Securities in an unrealized loss position greater than 12 months - Fair Value	1,648	10,186
Total - Unrealized losses	(2)	(499)
Total - Fair Value	$ 1,648	$ 10,186

- Definition

Line items represent financial concepts included in a table. These concepts are used to disclose reportable information associated with domain members defined in one or many axes to the table.

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+ Details

Income taxes - Provision for Taxes (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2023	Dec. 31, 2022	Dec. 31, 2021
Current:
U.S. Federal	$ 0	$ 0	$ 0
U.S. State and Local	27,226	(4,224)	(3,844)
Foreign	(4,003)	(1,582)	(1,340)
Deferred:
U.S. Federal	36,408	23,689	0
U.S. State and Local	10,521	10,587	(377)
Foreign	(646)	0	0
Total tax benefit (expense)	$ 69,506	$ 28,470	$ (5,561)

- Definition

Amount of current income tax expense (benefit) and deferred income tax expense (benefit) pertaining to continuing operations.

+ References + Details

Intangible assets and goodwill - Indefinite-lived Intangibles (Details) - USD ($) $ in Thousands	3 Months Ended	12 Months Ended
	Jun. 30, 2023	Dec. 31, 2023	Dec. 31, 2022
Indefinite-lived intangibles
Impairment		$ (217,800)	$ (33,384)
Intangible assets, net		379,497	705,891
Upstaza
Indefinite-lived intangibles
Impairment		0
Indefinite-lived intangible assets
Indefinite-lived intangibles
Beginning Balance		453,566	453,600
Impairment		(217,800)
Ending Balance		235,766	453,566
Indefinite-lived intangible assets \| Upstaza
Indefinite-lived intangibles
Beginning Balance		235,766
Impairment		0
Ending Balance		235,766	235,766
Indefinite-lived intangible assets \| PTC-FA and PTC-AS
Indefinite-lived intangibles
Impairment	$ (217,800)	(217,800)
Indefinite-lived intangible assets \| PTC-FA
Indefinite-lived intangibles
Beginning Balance		112,500
Impairment		(112,500)
Ending Balance			112,500
Indefinite-lived intangible assets \| PTC-AS
Indefinite-lived intangibles
Beginning Balance		105,300
Impairment		$ (105,300)
Ending Balance			$ 105,300

Net loss per share - Numerator and Denominator (Details) - USD ($) $ / shares in Units, $ in Thousands	12 Months Ended
	Dec. 31, 2023	Dec. 31, 2022	Dec. 31, 2021
Numerator
Net loss - Basic	$ (626,604)	$ (559,017)	$ (523,901)
Net loss - Diluted	$ (626,604)	$ (559,017)	$ (523,901)
Denominator
Basic	74,838,392	71,728,634	70,466,393
Diluted	74,838,392	71,728,634	70,466,393
Net loss per share - Basic	$ (8.37)	$ (7.79)	$ (7.43)
Net loss per share - Diluted	$ (8.37)	$ (7.79)	$ (7.43)

- Definition

The amount of net income (loss) for the period per each share of common stock or unit outstanding during the reporting period.

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- Definition

The amount of net income (loss) for the period available to each share of common stock or common unit outstanding during the reporting period and to each share or unit that would have been outstanding assuming the issuance of common shares or units for all dilutive potential common shares or units outstanding during the reporting period.

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-Subparagraph (c)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147482689/260-10-45-60B

+ Details

- Definition

Amount, after deduction of tax, noncontrolling interests, dividends on preferred stock and participating securities, and addition from assumption of issuance of common shares for dilutive potential common shares; of income (loss) available to common shareholders.

+ References + Details

Intangible assets and goodwill

12 Months Ended

Dec. 31, 2023

Goodwill and Intangible Assets Disclosure [Abstract]

Intangible assets and goodwill

17. Intangible assets and goodwill

Definite-lived intangibles

Definite lived intangible assets consisted of the following at December 31, 2023 and 2022:


		Ending Balance at				Foreign		Ending Balance at
Definite-lived		December 31,				currency		December 31,
intangibles assets, gross		2022		Additions		translation		2023
Emflaza	$	420,253	$	107,164	$	—	$	527,417
Waylivra		9,316		533		369		10,218
Tegsedi		7,109		5,839		374		13,322
Upstaza		89,550		—		—		89,550
Total definite-lived intangibles, gross	$	526,228	$	113,536	$	743	$	640,507


		Ending Balance at				Foreign		Ending Balance at
Definite-lived		December 31,				currency		December 31,
intangibles assets, accumulated amortization		2022		Amortization		translation		2023
Emflaza	$	(266,023)	$	(212,595)	$	—	$	(478,618)
Waylivra		(2,751)		(1,080)		(134)		(3,965)
Tegsedi		(1,709)		(1,498)		(104)		(3,311)
Upstaza		(3,420)		(7,462)		—		(10,882)
Total definite-lived intangibles, accumulated amortization	$	(273,903)	$	(222,635)	$	(238)	$	(496,776)

Total definite-lived intangibles, net							$	143,731

Marathon is entitled to receive contingent payments from the Company based on annual net sales of Emflaza beginning in 2018, up to a specified aggregate maximum amount over the expected commercial life of the asset. In accordance with the guidance for an asset acquisition, the Company records the milestone payment when it becomes payable to Marathon and increase the cost basis for the Emflaza rights intangible asset. For the year ended December 31, 2023, milestone payments of $107.2 million were recorded. These payments are being amortized over the remaining useful life of the Emflaza rights asset on a straight line basis. As of December 31, 2023, a milestone payable to Marathon of $52.7 million was recorded on the balance sheet within accounts payable and accrued expenses.

Akcea is also entitled to receive royalty payments subject to certain terms set forth in the Tegsedi-Waylivra Agreement related to sales of Waylivra and Tegsedi. In accordance with the guidance for an asset acquisition, the Company records royalty payments when they become payable to Akcea and increase the cost basis for the Waylivra and Tegsedi intangible assets, respectively. For the year ended December 31, 2023, royalty payments of $5.8 million and $0.5 million were recorded for Tegsedi and Waylivra, respectively. As of December 31, 2023, a royalty payable of $1.6 million and $0.4 million for Tegsedi and Waylivra, respectively, was recorded on the balance sheet within accounts payable and accrued expenses.

For the years ended December 31, 2023, 2022, and 2021, the Company recognized amortization expense of $222.6 million, $116.6 million, and $54.8 million respectively, related to the Emflaza rights, Waylivra, Tegsedi, and Upstaza intangible assets.

The estimated future amortization of the Emflaza rights, Waylivra, Tegsedi, and Upstaza intangible assets is expected to be as follows:


	As of December 31, 2023
2024	$	59,312
2025		10,512
2026		10,512
2027		10,512
2028 and thereafter		52,883
Total	$	143,731

The weighted average remaining amortization period of the definite-lived intangibles as of December 31, 2023 is 6.4 years.

Indefinite-lived intangibles

Indefinite lived intangible assets consisted of the following at December 31, 2023 and 2022:


		Ending Balance at				Reclass from				Foreign		Ending Balance at
Indefinite-lived		December 31,				Indefinite Lived to				currency		December 31,
intangibles assets		2022		Additions		Definite Lived		Impairment		translation		2023
Upstaza	$	235,766	$	—	$	—	$	—	$	—	$	235,766
PTC-FA		112,500		—		—		(112,500)		—		—
PTC-AS		105,300		—		—		(105,300)		—		—
Total indefinite-lived intangibles	$	453,566	$	—	$	—	$	(217,800)	$	—	$	235,766

Total intangible assets, net											$	379,497

In connection with the acquisition of the Company’s gene therapy platform from Agilis, the Company acquired rights to Upstaza, for the treatment of AADC deficiency. AADC deficiency is a rare CNS disorder arising from reductions in the enzyme AADC that result from mutations in the dopa decarboxylase gene. The gene therapy platform also includes PTC-FA, an asset targeting Friedreich ataxia, a rare and life-shortening neurodegenerative disease caused by a single defect in the FXN gene which causes reduced production of the frataxin protein. Additionally, the gene therapy platform includes two other programs targeting CNS disorders, including PTC-AS for Angelman syndrome, a rare, genetic, neurological disorder characterized by severe developmental delays.

In accordance with the acquisition method of accounting, the Company allocated the acquisition cost for the Agilis Merger to the underlying assets acquired and liabilities assumed, based upon the estimated fair values of those assets and liabilities at the date of acquisition. The Company classified the fair value of the acquired IPR&D as indefinite lived intangible assets until the successful completion or abandonment of the associated research and development efforts. As of December 31, 2022, the value allocated to the indefinite lived intangible assets was $453.6 million.

In May 2023, as part of the Company’s strategic portfolio prioritization, the Company decided to discontinue its preclinical and early research programs in its gene therapy platform, which included PTC-FA and PTC-AS. As a result, the Company determined that the PTC-FA and PTC-AS indefinite-lived intangible assets were fully impaired and recorded impairment expense of $217.8 million during the second quarter of 2023, which is recorded as intangible asset impairment in the statement of operations. As of December 31, 2023, the remaining indefinite lived intangible asset balance is $235.8 million, consisting solely of Upstaza, which the Company plans to continue to develop and commercialize.

The Company performed an annual test for its indefinite-lived intangible asset as of October 1, 2023 and concluded that no impairment exists as of December 31, 2023.

Goodwill

As a result of the Agilis Merger on August 23, 2018, the Company recorded $82.3 million of goodwill. There have been no changes to the balance of goodwill since the date of the Agilis Merger. Accordingly, the goodwill balance as of December 31, 2023 and 2022 was $82.3 million. The Company performed an annual impairment test for goodwill as of October 1, 2023. The Company’s single reporting unit had a negative carrying value and thus the Company determined there was no impairment of goodwill.

Leases - Lease payments (Details) $ in Thousands	Dec. 31, 2023 USD ($)
Operating leases
2024	$ 18,352
2025	20,450
2026	19,992
2027	17,945
2027 and thereafter	175,847
Total lease payments	252,586
Less: Imputed Interest expense	141,957
Total	110,629
Finance leases
2024	3,000
2025	3,000
2026	3,000
2025	3,000
2027 and thereafter	15,000
Total lease payments	27,000
Less: Imputed Interest expense	6,816
Total	$ 20,184

Capital structure - Narrative (Details) - USD ($) $ / shares in Units, $ in Millions		1 Months Ended	12 Months Ended
	Oct. 27, 2022	Aug. 31, 2019	Dec. 31, 2023	Dec. 31, 2022	Dec. 31, 2021	Jun. 30, 2021	May 31, 2021
Common stock
Number of shares issued in transaction (in shares)			0	0	0
Common stock, authorized shares (in shares)			250,000,000	250,000,000		250,000,000	125,000,000
Maximum
Common stock
Aggregate value of remaining shares to be issued and sold			$ 93.0
Stock Purchase Agreement
Common stock
Net consideration received on transaction	$ 50.0
Number of shares issued in transaction (in shares)	1,095,290
Price per share (in USD per share)	$ 45.65
Number of days within which to register stock sale with the SEC per terms of the agreement	60 days
Period of time from the closing date that the entity agrees to keep the stock sale registration effective	6 months
Period of time from the closing date that the purchaser agrees not to resell or transfer entity's stock without written approval	90 days
Censa
Common stock
Development milestone obligation payments made			$ 30.0
Shares issued for milestone payment			657,462
Development milestone obligation cash payments made			$ 0.4
Sales Agreement \| Common stock \| Maximum
Common stock
Net consideration received on transaction		$ 125.0

Intangible assets and goodwill - Future Amortization (Details) $ in Thousands	Dec. 31, 2023 USD ($)
Goodwill and Intangible Assets Disclosure [Abstract]
2024	$ 59,312
2025	10,512
2026	10,512
2027	10,512
2028 and thereafter	52,883
Total	$ 143,731

Fair value of financial instruments and investments - Fair Value Liabilities Measured (Details) - Agilis	Dec. 31, 2023 USD ($)	Dec. 31, 2022 USD ($)	Dec. 31, 2021 USD ($)
Development and Regulatory Milestones \| Valuation Technique, Discounted Cash Flow \| Probability of Success \| Weighted Average
Fair Value Valuation Inputs
Rights Exchange Settlement measurement	0.90
Development and Regulatory Milestones \| Valuation Technique, Discounted Cash Flow \| Measurement Input, Discount Rate \| Weighted Average
Fair Value Valuation Inputs
Rights Exchange Settlement measurement	0.059
Development and Regulatory Milestones \| Commitments \| Valuation Technique, Discounted Cash Flow \| Development and Regulatory Milestone \| Minimum
Fair Value Valuation Inputs
Potential milestones	$ 0	$ 0
Development and Regulatory Milestones \| Commitments \| Valuation Technique, Discounted Cash Flow \| Development and Regulatory Milestone \| Maximum
Fair Value Valuation Inputs
Potential milestones	$ 31,000,000	$ 331,000,000
Development and Regulatory Milestones \| Commitments \| Valuation Technique, Discounted Cash Flow \| Probability of Success \| Minimum
Fair Value Valuation Inputs
Rights Exchange Settlement measurement	0.85	0.25
Development and Regulatory Milestones \| Commitments \| Valuation Technique, Discounted Cash Flow \| Probability of Success \| Maximum
Fair Value Valuation Inputs
Rights Exchange Settlement measurement	0.92	0.92
Development and Regulatory Milestones \| Commitments \| Valuation Technique, Discounted Cash Flow \| Measurement Input, Discount Rate \| Minimum
Fair Value Valuation Inputs
Rights Exchange Settlement measurement	0.058	0.062
Development and Regulatory Milestones \| Commitments \| Valuation Technique, Discounted Cash Flow \| Measurement Input, Discount Rate \| Maximum
Fair Value Valuation Inputs
Rights Exchange Settlement measurement	0.061	0.083
Development and Regulatory Milestones \| Significant unobservable inputs (Level 3)
Fair Value Valuation Inputs
Fair value	$ 26,600,000
Development and Regulatory Milestones \| Significant unobservable inputs (Level 3) \| Commitments
Fair Value Valuation Inputs
Fair value	$ 26,600,000	$ 82,500,000	$ 139,300,000
Net Sales Milestones and Royalties \| Valuation Technique, Option Pricing Model \| Probability of Success \| Weighted Average
Fair Value Valuation Inputs
Rights Exchange Settlement measurement	0.93
Net Sales Milestones and Royalties \| Valuation Technique, Option Pricing Model \| Measurement Input, Discount Rate \| Weighted Average
Fair Value Valuation Inputs
Rights Exchange Settlement measurement	0.110
Net Sales Milestones and Royalties \| Commitments \| Valuation Technique, Option Pricing Model \| Sales Milestones \| Minimum
Fair Value Valuation Inputs
Potential milestones	$ 0	0
Net Sales Milestones and Royalties \| Commitments \| Valuation Technique, Option Pricing Model \| Sales Milestones \| Maximum
Fair Value Valuation Inputs
Potential milestones	$ 50,000,000	$ 150,000,000
Net Sales Milestones and Royalties \| Commitments \| Valuation Technique, Option Pricing Model \| Probability of Success \| Minimum
Fair Value Valuation Inputs
Rights Exchange Settlement measurement	0.85	0.25
Net Sales Milestones and Royalties \| Commitments \| Valuation Technique, Option Pricing Model \| Probability of Success \| Maximum
Fair Value Valuation Inputs
Rights Exchange Settlement measurement	1	1
Net Sales Milestones and Royalties \| Commitments \| Valuation Technique, Option Pricing Model \| Percentage of Sales for Royalties
Fair Value Valuation Inputs
Rights Exchange Settlement measurement	0
Net Sales Milestones and Royalties \| Commitments \| Valuation Technique, Option Pricing Model \| Percentage of Sales for Royalties \| Minimum
Fair Value Valuation Inputs
Rights Exchange Settlement measurement		0.02
Net Sales Milestones and Royalties \| Commitments \| Valuation Technique, Option Pricing Model \| Percentage of Sales for Royalties \| Maximum
Fair Value Valuation Inputs
Rights Exchange Settlement measurement		0.06
Net Sales Milestones and Royalties \| Commitments \| Valuation Technique, Option Pricing Model \| Measurement Input, Discount Rate
Fair Value Valuation Inputs
Rights Exchange Settlement measurement	0.11	0.115
Net Sales Milestones and Royalties \| Significant unobservable inputs (Level 3)
Fair Value Valuation Inputs
Fair value	$ 9,700,000
Net Sales Milestones and Royalties \| Significant unobservable inputs (Level 3) \| Commitments
Fair Value Valuation Inputs
Fair value	$ 9,700,000	$ 81,500,000	$ 100,600,000

Consolidated Balance Sheets - USD ($) $ in Thousands	Dec. 31, 2023	Dec. 31, 2022
Current assets:
Cash and cash equivalents	$ 594,001	$ 279,834
Marketable securities	282,738	130,871
Trade and royalty receivables, net	160,822	155,614
Inventory, net	30,577	21,808
Prepaid expenses and other current assets	150,491	105,658
Total current assets	1,218,629	693,785
Fixed assets, net	87,089	72,590
Intangible assets, net	379,497	705,891
Goodwill	82,341	82,341
Operating lease ROU assets	91,896	102,430
Deposits and other assets	36,246	48,582
Total assets	1,895,698	1,705,619
Current liabilities:
Accounts payable and accrued expenses	391,983	320,366
Deferred revenue	801	1,351
Operating lease liabilities- current	13,002	9,370
Finance lease liabilities- current	3,000	3,000
Liability for sale of future royalties - current	194,314	72,149
Total current liabilities	603,100	406,236
Long-term debt	284,213	571,722
Contingent consideration payable	36,300	164,000
Deferred tax liability	55,905	102,834
Operating lease liabilities- noncurrent	97,627	100,860
Finance lease liabilities- noncurrent	17,184	18,675
Liability for sale of future royalties- noncurrent	1,619,783	685,737
Other long-term liabilities	141	2,641
Total liabilities	2,714,253	2,052,705
Stockholders' deficit:
Common stock, $0.001 par value. Authorized 250,000,000 shares; issued and outstanding 75,708,889 shares at December 31, 2023. Authorized 250,000,000 shares; issued and outstanding 73,104,692 shares at December 31, 2022	75	72
Additional paid-in capital	2,466,233	2,305,020
Accumulated other comprehensive (loss) income	(1,285)	4,796
Accumulated deficit	(3,283,578)	(2,656,974)
Total stockholders' deficit	(818,555)	(347,086)
Total liabilities and stockholders' deficit	$ 1,895,698	$ 1,705,619

Accounts payable and accrued expenses - Restructuring (Details) $ in Millions	12 Months Ended
	Dec. 31, 2023 USD ($)
Restructuring costs
Accrued restructuring costs	$ 9.0
Selling, general and administrative
Restructuring costs
Restructuring costs	$ 32.8

Summary of significant accounting policies - Narrative (Details) $ in Thousands	1 Months Ended	3 Months Ended	12 Months Ended
	Jun. 30, 2022 USD ($)	Jun. 30, 2023 USD ($)	Dec. 31, 2023 USD ($) item	Dec. 31, 2023 USD ($) segment	Dec. 31, 2023 USD ($)	Dec. 31, 2023 USD ($)	Dec. 31, 2023 USD ($) country	Dec. 31, 2022 USD ($) segment country	Dec. 31, 2021 USD ($) country	Dec. 31, 2019 USD ($)	Dec. 31, 2018 USD ($)
New Accounting Pronouncements or Change in Accounting Principle [Line Items]
Number of operating segments \| segment				1				1
Number of reporting units			1	1
Allowance for credit loss			$ 0	$ 0	$ 0	$ 0	$ 0	$ 0
Increase in allowance for credit losses					0			0
Allowance for doubtful accounts receivable			1,200	1,200	1,200	$ 1,200	1,200	300
Bad debt expense					900			200	$ 100
Revenue					937,822			698,801	538,593
Cash consideration received from Royalty Purchase Agreement					1,000,000
State income tax provision					(27,226)			$ 4,224	$ 3,844
Income taxes
Federal income tax statutory rate						21.00%		21.00%	21.00%
Deferred tax liability			63,388	63,388	63,388	$ 63,388	63,388	$ 107,320
Deferred tax benefit					46,930			34,276	$ (377)
Impairment of long-lived assets					0
Non cash realized foreign exchange loss								16,887
Increase in current taxable income resulting from the elimination of current deduction of qualifying R&D costs under Section 174.					300,300
Impairment of indefinite-lived intangibles excluding goodwill					217,800			33,384
Impairment of goodwill					0
Upstaza
Income taxes
Impairment of indefinite-lived intangibles excluding goodwill					0
Indefinite-lived intangible assets
Income taxes
Impairment of indefinite-lived intangibles excluding goodwill					217,800
Indefinite-lived intangible assets \| PTC-FA and PTC-AS
Income taxes
Impairment of indefinite-lived intangibles excluding goodwill		$ 217,800			217,800
Indefinite-lived intangible assets \| Upstaza
Income taxes
Impairment of indefinite-lived intangibles excluding goodwill					0
Agilis
Income taxes
Deferred tax liability											$ 122,000
Impairment of goodwill					0
Prepaids and other current assets
New Accounting Pronouncements or Change in Accounting Principle [Line Items]
Deferred research and development advance payments, short-term			2,600	2,600	2,600	2,600	2,600	2,400
Deposits and other assets
New Accounting Pronouncements or Change in Accounting Principle [Line Items]
Bank guarantee denominated in foreign currency			600	600	600	600	600
Deferred research and development advance payments, long-term			$ 4,700	$ 4,700	4,700	$ 4,700	$ 4,700	3,900
Hopewell Campus \| Minimum
New Accounting Pronouncements or Change in Accounting Principle [Line Items]
Term for letter of credit (in years)										5 years
Hopewell Campus \| Deposits and other assets
New Accounting Pronouncements or Change in Accounting Principle [Line Items]
Letters of credit										$ 7,500
Hopewell Campus \| Fifth Anniversary
New Accounting Pronouncements or Change in Accounting Principle [Line Items]
Letters of credit										$ 3,800
Term for letter of credit (in years)										5 years
Warren Premises
New Accounting Pronouncements or Change in Accounting Principle [Line Items]
Period after which letter of credit may be reduced if lease is not in default	5 years
Warren Premises \| Deposits and other assets
New Accounting Pronouncements or Change in Accounting Principle [Line Items]
Letters of credit	$ 8,100
Warren Premises \| Fifth Anniversary
New Accounting Pronouncements or Change in Accounting Principle [Line Items]
Letters of credit	$ 4,100
Non-US
New Accounting Pronouncements or Change in Accounting Principle [Line Items]
Revenue					406,161			316,939
United States
New Accounting Pronouncements or Change in Accounting Principle [Line Items]
Revenue					531,661			$ 381,862
Net product revenue \| Geographic concentration risk
New Accounting Pronouncements or Change in Accounting Principle [Line Items]
Number of countries exceeding 10% of net product sales \| country							2	2	1
Percentage of net product sales threshold						10.00%		10.00%	10.00%
Net product revenue
New Accounting Pronouncements or Change in Accounting Principle [Line Items]
Revenue					661,249			$ 535,228	$ 428,904
Net product revenue \| Non-US
New Accounting Pronouncements or Change in Accounting Principle [Line Items]
Revenue					406,100			316,900	241,600
Net product revenue \| Net product revenue \| Geographic concentration risk \| Russia
New Accounting Pronouncements or Change in Accounting Principle [Line Items]
Revenue					86,000			59,700
Net product revenue \| Net product revenue \| Geographic concentration risk \| United States
New Accounting Pronouncements or Change in Accounting Principle [Line Items]
Revenue					255,100			218,300
Emflaza \| United States
New Accounting Pronouncements or Change in Accounting Principle [Line Items]
Revenue					255,100			218,300	187,300
Translarna \| Non-US
New Accounting Pronouncements or Change in Accounting Principle [Line Items]
Revenue					$ 355,800			$ 288,600	$ 236,000

Income taxes - Narrative (Details) - USD ($) $ in Thousands	12 Months Ended
Income taxes - Narrative (Details) - USD ($) $ in Thousands	Dec. 31, 2023	Dec. 31, 2022	Dec. 31, 2021	Dec. 31, 2018
Operating loss carryforwards
Taxable income before consideration of NOL	$ 102,500
State income tax benefit	27,226	$ (4,224)	$ (3,844)
Deferred tax valuation allowance	833,810	672,172
Valuation allowance, deferred tax asset, increase (decrease)	161,600	146,600
Deferred tax liability on indefinite-lived intangibles acquired	$ 55,905	$ 102,834
Federal income tax provision at statutory rate	21.00%	21.00%	21.00%
Uncertain tax position	$ 0
Increase in current taxable income resulting from the elimination of current deduction of qualifying R&D costs under Section 174.	300,300
Undistributed earnings from non-US subsidiaries	345,900
Undistributed earnings from non-US subsidiaries, tax	0
Unrecognized tax benefits	1,360	$ 27,217
Agilis
Operating loss carryforwards
Deferred tax liability on indefinite-lived intangibles acquired				$ 122,000
Federal
Operating loss carryforwards
Net operating loss carryforwards	287,000
Research and development credit carryforwards	35,900
Research and development credit carryforwards expiring during the period	600
Orphan Drug Credit carryover	169,600
State
Operating loss carryforwards
Net operating loss carryforwards	208,800
Foreign Tax Authority
Operating loss carryforwards
Net operating loss carryforwards	$ 12,100

Leases - Narrative (Details) $ in Thousands		1 Months Ended	12 Months Ended
Leases - Narrative (Details) $ in Thousands	Jul. 01, 2020 USD ($) ft² item	May 31, 2022 USD ($) ft² period building	Dec. 31, 2023 USD ($) lease	Dec. 31, 2022 USD ($)	Dec. 31, 2021 USD ($)
Leases
Operating lease, expense			$ 29,000	$ 25,200	$ 21,400
Finance Lease, Liability			20,184
Finance lease liabilities- current			3,000	3,000
Finance lease liabilities- noncurrent			17,184	18,675
Aggregate rent, Initial term			252,586
Operating lease ROU assets			91,896	102,430
Operating Lease, Liability			110,629
MassBio
Leases
Finance lease cost			1,500	1,600
Finance lease liabilities- current			3,000	3,000
Finance lease liabilities- noncurrent			$ 17,200	$ 18,700
Minimum
Leases
Operating lease, term of contract			2 months 12 days
Maximum
Leases
Operating lease, term of contract			15 years 4 months 24 days
Renewal term			15 years
Principal Office - NJ
Leases
Number of operating leases \| lease			2
Lessee, Operating Lease, Existence of Option to Terminate [true false]			false
Hopewell Campus
Leases
Operating lease, term of contract	15 years
Area of real estate property \| ft²	220,500
Renewal term	10 years
Number of renewable terms \| item	2
Lessee, Operating Lease, Existence of Option to Extend [true false]	true
Aggregate rent, Initial term	$ 111,500
Percent of market rate	95.00%
Warren Premises
Leases
Operating lease, term of contract		17 years
Number of buildings leased \| building		2
Area of real estate property \| ft²		360,000
Renewal term		5 years
Number of renewable terms \| period		3
Lessee, Operating Lease, Existence of Option to Extend [true false]		true
Aggregate rent, Initial term		$ 163,000
Operating lease ROU assets		28,900
Operating Lease, Liability		$ 28,900
Base rent abatement period		3 years
Base rent abatement		$ 18,600
Aggregate rent net of base rate abatement, initial term		144,400
Allowance for lease improvements		36,200
Commitment to fund construction account		3,600
Payment due from lessor at issuance of temporary certificate of occupancy		$ 5,000

Income taxes (Tables)

12 Months Ended

Dec. 31, 2023

Income Tax Disclosure [Abstract]

Schedule of Income before Income Tax, Domestic and Foreign

The loss from operations before tax benefit (expense) consisted of the following for the years ended December 31, 2023, 2022, and 2021:


	2023		2022		2021
Domestic	$	(784,744)	$	(591,126)	$	(487,726)
Foreign		88,634		3,639		(30,614)
Total	$	(696,110)	$	(587,487)	$	(518,340)

Components of Income Tax Provision

The Income Tax Provision consisted of the following for the years ended December 31, 2023, 2022 and 2021:


	2023		2022		2021
Current:
U.S. Federal	$	—	$	—	$	—
U.S. State and Local		27,226		(4,224)		(3,844)
Foreign		(4,003)		(1,582)		(1,340)
Deferred:
U.S. Federal		36,408		23,689		—
U.S. State and Local		10,521		10,587		(377)
Foreign		(646)		—		—
Total tax benefit (expense)	$	69,506	$	28,470	$	(5,561)

Schedule of reconciliation of the U.S. statutory income tax rate to the Company's effective tax rate

A reconciliation of the U.S. statutory income tax rate to the Company’s effective tax rate is as follows:


	December 31,
	2023		2022		2021
Federal income tax provision at statutory rate	21.00	%	21.00	%	21.00	%
State income tax provision, net of federal benefit	0.32		3.07		(0.74)
Permanent differences	(1.43)		(1.83)		(4.06)
Research and development	4.59		5.89		4.50
Change in valuation allowances	(16.86)		(23.36)		(29.03)
Change in deferred tax assets	—		(0.10)		12.05
Foreign tax rate differential	0.05		(0.17)		0.01
Tax rate change	(1.26)		0.34		0.01
Release (Accrual) of uncertain tax positions	3.71		—		(4.78)
Other	(0.12)		—		(0.03)
Effective income tax rate	10.00	%	4.84	%	(1.07)	%

Schedule of significant components of the Company's deferred tax assets and liabilities


	December 31,
	2023		2022
Assets:
Noncurrent deferred income taxes	$	—	$	—
Liabilities:
Noncurrent deferred income taxes		(55,905)		(102,834)
Deferred income taxes - net	$	(55,905)	$	(102,834)

The significant components of the Company’s deferred tax assets and liabilities at December 31, 2023 and 2022 are as follows:


	2023		2022
Deferred tax assets:
Accrued expense	$	25,400	$	2,124
Amortization		137,808		52,532
Federal tax credits		205,485		174,802
State tax credits		9,817		9,787
Federal net operating losses		60,270		69,957
State net operating losses		18,680		10,316
Foreign net operating losses		4,052		4,837
Capitalized research and development costs		149,683		110,219
Share based compensation and other		30,757		27,054
Liability for sale of future royalties		190,659		185,589
Noncash interest expense		9,410		30,160
Other comprehensive loss		(728)		(719)
Total gross deferred tax assets		841,293		676,658
Less valuation allowance		(833,810)		(672,172)
Total deferred tax assets, net of valuation allowance	$	7,483	$	4,486
Deferred tax liabilities:
Depreciation	$	(7,483)	$	(4,486)
Indefinite lived intangible		(55,905)		(102,834)
Total gross deferred tax liabilities		(63,388)		(107,320)
Net deferred tax assets (liabilities)	$	(55,905)	$	(102,834)

Schedule of unrecognized tax benefits

A reconciliation of the gross amount of unrecognized tax benefits, excluding accrued interest and penalties, is as follows:


	Unrecognized Tax Benefits
Balance at December 31, 2022		27,217
Reductions based on settlements or expiration of statute of limitations		(24,671)
Interest received		(1,186)
Balance at December 31, 2023	$	1,360

Fair value of financial instruments and investments - Marketable Securities, Unrealized Gains (Losses) (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2023	Dec. 31, 2022
Debt Securities, Available-for-sale [Line Items]
Amortized Cost	$ 259,778	$ 23,104
Gross Unrealized, Gain	340	5
Gross Unrealized, Loss	(14)	(499)
Fair Value	260,104	22,610
Write downs of available for sale debt securities	0	0
Increase in allowance for credit losses	0	0
Realized gain (loss) from sale of marketable securities	(300)	4,000
Commercial paper
Debt Securities, Available-for-sale [Line Items]
Amortized Cost	117,044	12,419
Gross Unrealized, Gain	128	5
Gross Unrealized, Loss	(12)	0
Fair Value	117,160	12,424
Corporate debt securities
Debt Securities, Available-for-sale [Line Items]
Amortized Cost	1,650	10,685
Gross Unrealized, Gain	0	0
Gross Unrealized, Loss	(2)	(499)
Fair Value	1,648	$ 10,186
Government obligations
Debt Securities, Available-for-sale [Line Items]
Amortized Cost	141,084
Gross Unrealized, Gain	212
Gross Unrealized, Loss	0
Fair Value	$ 141,296

Consolidated Statements of Cash Flows - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2023	Dec. 31, 2022	Dec. 31, 2021
Cash flows from operating activities
Net Income (Loss)	$ (626,604)	$ (559,017)	$ (523,901)
Adjustments to reconcile net loss to net cash used in operating activities:
Depreciation and amortization	236,590	128,836	64,134
Non-cash operating lease expense	9,827	9,884	7,386
Non-cash royalty revenue related to sale of future royalties	(93,460)	(48,738)	(23,460)
Non-cash interest expense on liability related to sale of future royalties	104,790	72,639	77,683
Intangible asset impairment	217,800	33,384
Change in valuation of contingent consideration	(127,700)	(25,900)	(500)
Unrealized loss on ClearPoint Equity Investments	1,515	3,560	6,078
Unrealized loss on ClearPoint convertible debt security	2,678	5,740	8,281
Unrealized (gain) loss on marketable securities - equity investments	(2,517)	7,992	(1,673)
Realized loss for the sale of Clearpoint Equity Investment	782
Realized gain on redemption of marketable securities- equity investments	(4,383)
Non-cash stock consideration, milestone payment	29,570
Disposal of asset	806	80
Amortization of (discounts) premiums on investments, net	(2,200)	1,713	5,299
Deferred income taxes	(46,930)	(34,276)	377
Amortization of debt issuance costs	1,873	1,901	1,848
Loss on extinguishment of debt	55,625
Share-based compensation expense	101,636	110,333	103,513
Unrealized foreign currency transaction (gains) losses, net	(14,113)	13,263	47,391
Non cash foreign currency remeasurement loss on intercompany loan		16,887
Changes in operating assets and liabilities:
Inventory, net	(8,183)	(6,668)	1,800
Prepaid expenses and other current assets	(44,992)	(51,621)	(15,310)
Trade and royalty receivables, net	(1,539)	(48,468)	(44,991)
Deposits and other assets	5,222	(2,913)	(232)
Accounts payable and accrued expenses	48,346	27,542	45,659
Other liabilities	(2,307)	(4,558)	(6,704)
Deferred revenue	(550)	1,351	(4,010)
Payments on contingent consideration		(9,600)
Net cash used in operating activities	(158,418)	(356,654)	(251,332)
Cash flows from investing activities
Purchases of fixed assets	(28,438)	(32,016)	(28,213)
Purchases of marketable securities - available for sale	(174,086)	(52,764)	(333,148)
Purchases of marketable securities - equity investments	(38,398)	(22,787)	(210,018)
Sale and redemption of marketable securities- available for sale	21,544	405,234	843,498
Sale and redemption of marketable securities - equity investments	132,228	112,958	4,281
Sale and redemption of ClearPoint Equity Investments	2,594
Acquisition of product rights and licenses	(92,181)	(120,444)	(57,118)
Purchase of equity investment in ClearPoint			(100)
Net cash (used in) provided by investing activities	(176,737)	290,181	219,182
Cash flows from financing activities
Proceeds from exercise of options	24,040	14,632	17,309
Repayment of senior secured term loan	(300,000)
Debt extinguishment costs related to senior secured term loan	(81,933)
Cash consideration received from Royalty Purchase Agreement	1,000,000
Debt issuance costs related to secured term loan	(282)	(11,454)
Proceeds from issuance of senior secured term loan		300,000
Repayment of Convertible Notes		(150,000)
Payments on deferred and contingent consideration obligation		(40,400)
Proceeds from employee stock purchase plan	5,954	6,450	5,792
Payment of finance lease principal	(1,379)	(1,276)	(2,224)
Proceeds from stock purchase agreement		50,000
Net cash provided by financing activities	646,400	167,952	20,877
Effect of exchange rate changes on cash	3,114	(2,772)	(7,821)
Net increase (decrease) in cash and cash equivalents	314,359	98,707	(19,094)
Cash and cash equivalents, and restricted cash beginning of period	295,925	197,218	216,312
Cash and cash equivalents, and restricted cash end of period	610,284	295,925	197,218
Supplemental disclosure of cash information
Cash paid for interest	36,131	18,463	9,588
Cash paid for income taxes	14,155	4,922	7,708
Supplemental disclosure of non-cash investing and financing activity
Unrealized gain (loss) on marketable securities, net of tax	820	108	(2,502)
Right-of-use assets obtained in exchange for operating lease obligations		35,817	645
Acquisition of product rights and licenses	54,618	33,239	$ 22,294
Milestone payable	$ 2,500
Debt issuance costs related to senior secured term loan		159
Capital expenditures unpaid at the end of period		$ 308

Summary of significant accounting policies - Liability for Sale of Future Royalties (Details) $ in Thousands			12 Months Ended
	Oct. 18, 2023 USD ($) Option	Jul. 17, 2020 USD ($)	Dec. 31, 2023 USD ($)
Assigned royalty payments
Cash consideration received from Royalty Purchase Agreement			$ 1,000,000
Assigned royalty payment, retained percentage		57.067%
Remaining potential milestones that can be achieved			$ 150,000
Effective interest rate of the liability component			10.80%
Loss on extinguishment of debt			$ 137,558
Aggregate Royalty Purchase Agreements \| Royalty Pharma
Assigned royalty payments
Total percentage of royalty rights assigned	80.9777%
Original Royalty Purchase Agreement
Assigned royalty payments
Assigned royalty payment, percentage		42.933%
Cash consideration received from Royalty Purchase Agreement		$ 650,000
Royalty purchase agreement termination period once there are no further royalty payment obligations		60 days
Royalty purchase agreement, payment maximum	$ 1,300,000	$ 1,300,000
Original Royalty Purchase Agreement \| Royalty Pharma
Assigned royalty payments
Royalty purchase agreement, payment maximum	1,300,000
A&R Royalty Purchase Agreement
Assigned royalty payments
Cash consideration received from Royalty Purchase Agreement	$ 1,000,000
Royalty purchase agreement termination period once there are no further royalty payment obligations	60 days
Effective interest rate of the liability component	10.80%
Retained Royalty Rights percentage sold	38.0447%
Percentage of total royalty payable after threshold royalties received	66.6667%
Additional cash consideration	$ 500,000
Number of put options held by the company \| Option	5
Maximum number of put options which can be exercised before Royalty Pharma can exercise a call option \| Option	2
Maximum percentage of remaining retained royalty rights that are available upon exercise of call option	50.00%
Fair value of liability for sale of future royalties.	$ 1,809,900
Loss on extinguishment of debt	$ 44,900
A&R Royalty Purchase Agreement \| Royalty Pharma
Assigned royalty payments
Percentage of maximum royalty payable until assigned royalty rights	90.4888%
Percentage of maximum royalty payable thereafter assigned royalty rights	83.3333%

Summary of significant accounting policies (Policies)

12 Months Ended

Dec. 31, 2023

Accounting Policies [Abstract]

Basis of presentation

The accompanying consolidated financial statements have been prepared in accordance with U.S. generally accepted accounting principles (“GAAP”) and include all adjustments necessary for the fair presentation of the Company’s financial position for the periods presented.

Use of estimates

The preparation of financial statements in conformity with GAAP requires management to make estimates and assumptions that affect the amounts reported in the financial statements and accompanying notes. Significant estimates in these consolidated financial statements have been made in connection with the calculation of net product sales, royalty revenue, certain accruals related to the Company’s research and development expenses, valuation procedures for liability for sale of future royalties, indefinite lived intangible assets annual impairment assessment, fair value of the contingent consideration, and the provision for or benefit from income taxes. Actual results could differ from those estimates. Changes in estimates are reflected in reported results in the period in which they become known.

Restricted Cash

Restricted cash included in deposits and other assets on the consolidated balance sheet relates to an unconditional, irrevocable and transferable letter of credit that was entered into during the twelve-month period ended December 31, 2019 in connection with obligations under a facility lease for the Company’s leased biologics manufacturing facility in Hopewell Township, New Jersey. The amount of the letter of credit is $7.5 million, is to be maintained for a term of not less than five years and has the potential to be reduced to $3.8 million if after five years from the lease commencement the Company is not in default of its lease. Restricted cash also contains an unconditional, irrevocable and transferable letter of credit that was entered into during June 2022 in connection with obligations for the Company’s new facility lease in Warren, New Jersey. The amount of the letter of credit is $8.1 million and has the potential to be reduced to $4.1 million if after five years the Company is not in default of its lease. Both amounts are classified within deposits and other assets on the consolidated balance sheet due to the long-term nature of the letter of credit. Restricted cash also includes a bank guarantee of $0.6 million denominated in a foreign currency.

The following table provides a reconciliation of cash, cash equivalents, and restricted cash reported within the consolidated balance sheet that sum to the total of the same amounts shown in the statement of cash flows:


	End of			Beginning of
	period-		period-
	December 31,		December 31,
	2023		2022
Cash and cash equivalents	$	594,001	$	279,834
Restricted cash included in deposits and other assets		16,283		16,091
Total Cash, cash equivalents and restricted cash per statement of cash flows	$	610,284	$	295,925

Consolidation

The consolidated financial statements include the accounts of PTC Therapeutics, Inc. and its wholly owned subsidiaries. All inter-company accounts, transactions, and profits have been eliminated in consolidation.

Segment and geographic information

Operating segments are defined as components of an enterprise about which separate discrete information is available for evaluation by the chief operating decision maker, or decision making group, in deciding how to allocate resources and in assessing performance. The Company views its operations and manages its business in one operating and reporting segment.

Cash equivalents

The Company considers all highly liquid investments with a maturity of 90 days or less at the time of purchase to be cash equivalents. Cash equivalents are carried at cost which approximates fair value due to their short-term nature.

Marketable securities

The Company’s marketable securities consists of both debt securities and equity investments. The Company considers its investments in debt securities with original maturities of greater than 90 days to be available for sale securities. Securities under this classification are recorded at fair value and unrealized gains and losses within accumulated other comprehensive income. The estimated fair value of the available for sale securities is determined based on quoted market prices or rates for similar instruments. In addition, the cost of debt securities in this category is adjusted for amortization of premium and accretion of discount to maturity. For available for sale debt securities in an unrealized loss position, the Company assesses whether it intends to sell or if it is more likely than not that the Company will be required to sell the security before recovery of its amortized cost basis. If either of the criteria regarding intent or requirement to sell is met, the security’s amortized cost basis is written down to fair value. If the criteria are not met, the Company evaluates whether the decline in fair value has resulted from a credit loss or other factors. In making this assessment, management considers, among other factors, the extent to which fair value is less than amortized cost, any changes to the rating of the security by a rating agency, and adverse conditions specifically related to the security. If this assessment indicates that a credit loss exists, the present value of cash flows expected to be collected from the security are compared to the amortized cost basis of the security. If the present value of the cash flows expected to be collected is less than the amortized cost basis, a credit loss exists and an allowance for credit losses is recorded for the credit loss, limited by the amount that the fair value is less than the amortized costs basis. Any impairment that has not been recorded through an allowance for credit losses is recognized in other comprehensive income. For the years ended December 31, 2023 and 2022, no allowance was recorded for credit losses.

Marketable securities that are equity investments are measured at fair value, as it is readily available, and as such are classified as Level 1 assets. Unrealized holding gains and losses for these equity investments are components of other income (expense), net within the consolidated statement of operations.

Concentration of credit risk

The Company’s financial instruments that are exposed to credit risks consist primarily of cash and cash equivalents, available-for-sale marketable securities and accounts receivable. The Company maintains its cash and cash equivalents in bank accounts, which, at times, exceed federally insured limits. The Company has not experienced any credit losses in these accounts and does not believe it is exposed to any significant credit risk on these funds. The Company’s investment policy includes guidelines on the quality of the financial institutions and financial instruments the Company is allowed to invest in, which the Company believes minimizes the exposure to concentration of credit risk.

The Company is subject to credit risk from its accounts receivable related to its product sales. The payment terms are predetermined and the Company evaluates the creditworthiness of each customer or distributor on a regular basis. The Company reserves all uninsured amounts billed directly to a patient until the time of cash receipt as collectability is not reasonably assured at the time the product is received. To date, the Company has not incurred any material credit losses.

Fixed assets

Fixed assets are stated at cost. Depreciation is computed starting when the asset is placed into service on a straight-line basis over the estimated useful life of the related asset as follows:

Leasehold improvements		Lesser of useful life or lease term
Computer equipment and software		3 years
Machinery and lab equipment		7 years
Furniture and fixtures		7 years

Inventory and cost of product sales

Inventory

Inventories are stated at the lower of cost and net realizable value with cost determined on a first-in, first-out basis by product. The Company capitalizes inventory costs associated with products following regulatory approval when future commercialization is considered probable and the future economic benefit is expected to be realized. Products which may be used in clinical development programs are included in inventory and charged to research and development expense when the product enters the research and development process and no longer can be used for commercial purposes. Inventory used for marketing efforts are charged to selling, general and administrative expense. Amounts related to clinical development programs and marketing efforts are immaterial.

The following table summarizes the components of the Company’s inventory for the periods indicated:


	December 31, 2023		December 31, 2022
Raw materials	$	952	$	1,078
Work in progress		17,991		14,074
Finished goods		11,634		6,656
Total inventory	$	30,577	$	21,808

The Company periodically reviews its inventories for excess amounts or obsolescence and writes down obsolete or otherwise unmarketable inventory to its estimated net realizable value. The Company recorded write downs of $12.5 million and $1.7 million for the years ended December 31, 2023 and 2022, respectively, primarily related to adjustments to inventory reserves and product approaching expiration. Additionally, though the Company’s product is subject to strict quality control and monitoring which it performs throughout the manufacturing processes, certain batches or units of product may not meet quality specifications resulting in a charge to cost of product sales. For the years ended December 31, 2023 and December 31, 2022, these amounts were immaterial.

Cost of product sales

Cost of product sales consists of the cost of inventory sold, manufacturing and supply chain costs, storage costs, amortization of the acquired intangible asset, royalty payments associated with net product sales, and royalty payments to collaborative partners associated with royalty revenues and collaboration revenue related to milestones. Production costs are expensed as cost of product sales when the related products are sold or royalty revenues and collaboration revenue milestones are earned.

Accumulated other comprehensive income (loss)

Accumulated other comprehensive income (loss) consists of unrealized gains or losses on marketable securities and foreign currency translation adjustments.

Revenue recognition

Net product revenue

The Company’s net product revenue primarily consists of sales of Translarna in territories outside of the U.S. for the treatment of nmDMD and sales of Emflaza in the U.S. for the treatment of DMD. The Company recognizes revenue when its performance obligations with its customers have been satisfied. The Company’s performance obligations are to provide products based on customer orders from distributors, hospitals, specialty pharmacies or retail pharmacies. The performance obligations are satisfied at a point in time when the Company’s customer obtains control of the product, which is typically upon delivery. The Company invoices its customers after the products have been delivered and invoice payments are generally due within 30 to 90 days of the invoice date. The Company determines the transaction price based on fixed consideration in its contractual agreements. Contract liabilities arise in certain circumstances when consideration is due for goods the Company has yet to provide. As the Company has identified only one distinct performance obligation, the transaction price is allocated entirely to product sales. In determining the transaction price, a significant financing component does not exist since the timing from when the Company delivers product to when the customers pay for the product is typically less than one year. Customers in certain countries pay in advance of product delivery. In those instances, payment and delivery typically occur in the same month.

The Company records product sales net of any variable consideration, which includes discounts, allowances, rebates related to Medicaid and other government pricing programs, and distribution fees. The Company uses the expected value or most likely amount method when estimating its variable consideration, unless discount or rebate terms are specified within contracts. The identified variable consideration is recorded as a reduction of revenue at the time revenues from product sales are recognized. These estimates for variable consideration are adjusted to reflect known changes in factors and may impact such estimates in the quarter those changes are known. Revenue recognized does not include amounts of variable consideration that are constrained.

During the years ended December 31, 2023, 2022, and 2021, net product sales in the United States were $255.1 million, $218.3 million, and $187.3 million, respectively, consisting solely of sales of Emflaza, and net product sales outside of the United States were $406.1 million, $316.9 million, and $241.6 million respectively, consisting of sales of Translarna, Tegsedi, Waylivra, and Upstaza. Translarna net product revenues made up $355.8 million, $288.6 million, and $236.0 million of the net product sales outside of the United States for the years ended December 31, 2023, 2022, and 2021, respectively. During the years ended December 31, 2023 and 2022, two countries, the United States and Russia, accounted for at least 10% of the Company’s net product sales, representing $255.1 million and $86.0 million, and $218.3 million and $59.7 million of net product sales, respectively. During the year ended December 31, 2021, only the United States accounted for at least 10% of the Company’s net product sales.

In relation to customer contracts, the Company incurs costs to fulfill a contract but does not incur costs to obtain a contract. These costs to fulfill a contract do not meet the criteria for capitalization and are expensed as incurred. The Company considers any shipping and handling costs that are incurred after the customer has obtained control of the product as a cost to fulfill a promise. Shipping and handling costs associated with finished goods delivered to customers are recorded as a selling expense.

Collaboration and royalty revenue

The terms of these agreements typically include payments to the Company of one or more of the following: nonrefundable, upfront license fees; milestone payments; research funding and royalties on future product sales. In addition, the Company generates service revenue through agreements that generally provide for fees for research and development services and may include additional payments upon achievement of specified events.

At the inception of a collaboration arrangement, the Company needs to first evaluate if the arrangement meets the criteria in Financial Accounting Standards Board (“FASB”) Accounting Standards Codification (“ASC”) Topic 808 “Collaborative Arrangements” to then determine if ASC Topic 606 is applicable by considering whether the collaborator meets the definition of a customer. If the criteria are met, the Company assesses the promises in the arrangement to identify distinct performance obligations.

For licenses of intellectual property, the Company assesses, at contract inception, whether the intellectual property is distinct from other performance obligations identified in the arrangement. If the licensing of intellectual property is determined to be distinct, revenue is recognized for nonrefundable, upfront license fees when the license is transferred to the customer and the customer can use and benefit from the license. If the licensing of intellectual property is determined not to be distinct, then the license will be bundled with other promises in the arrangement into one distinct performance obligation. The Company needs to determine if the bundled performance obligation is satisfied over time or at a point in time. If the Company concludes that the nonrefundable, upfront license fees will be recognized over time, the Company will need to assess the appropriate method of measuring proportional performance.

For milestone payments, the Company assesses, at contract inception, whether the development or sales-based milestones are considered probable of being achieved. If it is probable that a significant revenue reversal will occur, the Company will not record revenue until the uncertainty has been resolved. Milestone payments that are contingent upon regulatory approval are not considered probable of being achieved until the applicable regulatory approvals or other external conditions are obtained as such conditions are not within the Company’s control. If it is probable that a significant revenue reversal will not occur, the Company will estimate the milestone payments using the most likely amount method. The Company will re-assess the development and sales-based milestones each reporting period to determine the probability of achievement. The Company recognizes royalties from product sales at the later of when the related sales occur or when the performance obligation to which the royalty has been allocated has been satisfied. If it is probable that a significant revenue reversal will not occur, the Company will estimate the royalty payments using the most likely amount method.

The Company recognizes revenue for reimbursements of research and development costs under collaboration agreements as the services are performed. The Company records these reimbursements as revenue and not as a reduction of research and development expenses as the Company has the risks and rewards as the principal in the research and development activities.

For the years ended December 31, 2023, 2022, and 2021, the Company has recognized $100.0 million, $50.1 million, and $55.0 million of collaboration revenue, respectively, related to the SMA License Agreement with Roche.

For the years ended December 31, 2023, 2022 and 2021, the Company has recognized $168.9 million, $113.5 million, and $54.6 million of royalty revenue, respectively, related to Evrysdi.

Manufacturing Revenue

The Company has manufacturing services related to the production of plasmid deoxyribonucleic acid (“DNA”) and adeno-associated virus (“AAV”) vectors for gene therapy applications for external customers. Performance obligations vary but may include manufacturing plasmid DNA and/or AAV vectors, material testing, stability studies, and other services related to material development. The transaction prices for these arrangements are fixed and include amounts stated in the contracts for each promised service. Typically, the performance obligations within a manufacturing contract are highly interdependent, in which case, the Company will combine them into a single performance obligation. The Company has determined that the assets created have no alternative use to the Company, and the Company has an enforceable right to payment for the performance completed to date, therefore revenue related to these services are recognized over time and is measured using an output method based on performance of manufacturing milestones completed to date.

Manufacturing service contracts may also include performance obligations related to project management services or obtaining materials from third parties. The Company has determined that these are separate performance obligations for which revenue is recognized at the point in time the services are performed. For performance obligations related to obtaining third party materials, the Company has determined that it is the principal as the Company has control of the materials and has discretion in setting the price. Therefore, the Company recognizes revenue on a gross basis related to obtaining third party materials.

Certain arrangements require a portion of the contract consideration to be received in advance at the commencement of the contract, and such advance payment is initially recorded as a contract liability. A contract asset may be recognized in the event the Company’s satisfaction of performance obligations outpaces customer billings.

For the year ended December 31, 2023, the Company recognized $7.7 million of manufacturing revenue related to plasmid DNA and AAV vector production for external customers. No manufacturing revenue was recognized for the years ended December 31, 2022 and 2021. As of December 31, 2023, the Company has contract assets of $0.2 million and remaining performance obligations of $0.8 million related to the production of plasmid DNA and AAV vectors for gene therapy applications for external customers. For the period ended December 31, 2022, the Company had remaining performance obligations of $1.4 million and no contracts assets related to plasmid DNA and AAV production for external customers.

Allowance for doubtful accounts

The Company maintains an allowance for estimated losses resulting from the inability of its customers to make required payments. The Company estimates uncollectible amounts based upon current customer receivable balances, the age of customer receivable balances, the customer’s financial condition and current economic trends. The Company also assesses whether an allowance for expected credit losses may be required which includes a review of the Company’s receivables portfolio, which are pooled on a customer basis or country basis. In making its assessment of whether an allowance for credit losses is required, the Company considers its historical experience with customers, current balances, levels of delinquency, regulatory and legal environments, and other relevant current and future forecasted economic conditions. For the years ended December 31, 2023 and 2022, no allowance was recorded for credit losses. The allowance for doubtful accounts was $1.2 million as of December 31, 2023 and $0.3 million as of December 31, 2022. For the years ended December 31, 2023, 2022 and 2021, bad debt expense was $0.9 million, $0.2 million, and $0.1 million, respectively.

Liability for sale of future royalties

Liability for sale of future royalties

On July 17, 2020, the Company, RPI Intermediate Finance Trust (“RPI”), and, for the limited purposes set forth in the agreement, Royalty Pharma PLC, entered into a royalty purchase agreement (the “Original Royalty Purchase

Agreement”). Pursuant to the Original Royalty Purchase Agreement, the Company sold to RPI 42.933% (the “Original Assigned Royalty Rights”) of the Company’s right to receive sales-based royalty payments (the “Royalty”) on worldwide net sales of Evrysdi and any other product developed pursuant to the License and Collaboration Agreement (the “License Agreement”), dated as of November 23, 2011, by and among the Company, Roche and, for the limited purposes set forth therein, the SMA Foundation under the SMA program. In consideration for the sale of the Original Assigned Royalty Rights, RPI paid the Company $650.0 million in cash consideration. The Company has retained a 57.067% interest in the Royalty and all economic rights to receive the remaining potential regulatory and sales milestone payments under the License Agreement, which remaining milestone payments equal $150.0 million in the aggregate as of December 31, 2023. The Original Royalty Purchase Agreement was set to terminate 60 days following the earlier of the date on which Roche is no longer obligated to make any payments of the Royalty pursuant to the License Agreement and the date on which RPI has received $1.3 billion in respect of the Original Assigned Royalty Rights.

Pursuant to the guidance in ASC 470-10-25-2, the Company determined that the $650.0 million cash consideration obtained pursuant to the Original Royalty Purchase Agreement should be classified as debt and recorded it as “liability for sale of future royalties-current” and “liability for sale of future royalties-noncurrent” on the Company’s consolidated balance sheet based on the timing of the expected payments to be made to RPI at the time of the transaction. The liability was subsequently amortized using the effective interest method over the life of the arrangement, in accordance with the respective guidance, utilizing the prospective method to account for subsequent changes in the estimated future payments to be made to RPI.

On October 18, 2023, the Company, Royalty Pharma Investments 2019 ICAV (“Royalty Pharma”), and, for the limited purposes set forth in the agreement, Royalty Pharma plc, entered into an Amended and Restated Royalty Purchase Agreement (the “A&R Royalty Purchase Agreement”), which amends and restates in its entirety the Original Royalty Purchase Agreement. Pursuant to the A&R Royalty Purchase Agreement, the Company has sold or agreed to sell to Royalty Pharma certain portions of the Company’s remaining Royalty on worldwide net sales of Evrysdi and any other product (the “Products”) developed pursuant to the SMA License Agreement (all such retained Royalty rights of the Company, the “Retained Royalty Rights,” and all such Royalty rights that are sold to Royalty Pharma pursuant to the A&R Royalty Purchase Agreement, the “A&R Assigned Royalty Rights”). At closing, Royalty Pharma paid the Company $1.0 billion in cash consideration for 38.0447% of the Company’s Retained Royalty Rights (which is in addition to the 42.9330% assigned to Royalty Pharma in connection with the Original Royalty Purchase Agreement, for a total of 80.9777% of the total Royalty) until such time as Royalty Pharma has received payments in respect of the Original Assigned Royalty Rights equal to $1.3 billion in the aggregate, and thereafter 66.6667% of the total Royalty. In addition, the Company may sell to Royalty Pharma the remainder of the Company’s Retained Royalty Rights in exchange for an aggregate of $500.0 million in additional cash consideration after the closing of the A&R Royalty Purchase Agreement, less royalties received in respect of the Retained Royalty Rights put to Royalty Pharma, which will be payable by Royalty Pharma pursuant to five put options held by the Company that are exercisable at the Company’s option between January 1, 2024 and December 31, 2025. If the Company exercises two or fewer of the put options, Royalty Pharma may exercise a call option during the period from and after January 1, 2026 until and including March 31, 2026 for up to 50% of the remainder of the Company’s Retained Royalty Rights less amounts exercised by the Company via its put options at a purchase price that is proportional to the purchase price of the Company’s unexercised put options. Royalty Pharma’s exercise of the call option would result in Royalty Pharma owning 90.4888% of the total Royalty until such time as Royalty Pharma has received payments in respect of the Original Assigned Royalty Rights equal to $1.3 billion in the aggregate, and thereafter 83.3333% of the total Royalty. The A&R Royalty Purchase Agreement will terminate 60 days following the date on which Roche is no longer obligated to make any payments of the Royalty pursuant to the License Agreement.

The change in rights and obligations from the A&R Royalty Purchase Agreement resulted in a change in the terms of the liability for sale of future royalties, which was evaluated by the Company in accordance with ASC 470-50, Debt —

Modifications and Extinguishments. The Company determined that the present value of the cash flows under the A&R Royalty Purchase Agreement were substantially different from the present value of the cash flows under the Original Royalty Purchase Agreement. This resulted in the derecognition of the old liability for sale of future royalties and the new liability for sale of future royalties being recorded at fair value, which was determined to be $1,809.9 million as of the date of the A&R Royalty Purchase Agreement. This resulted in an extinguishment loss of $44.9 million, which was recorded within loss on extinguishment of debt, within the Company’s statement of operations.

The fair value for the new liability for sale of future royalties on the date of the A&R Royalty Purchase Agreement was based on the Company’s estimates of future royalties expected to be paid to Royalty Pharma over the life of the arrangement, which was determined using forecasts from market data sources, which are considered Level 3 inputs. The liability is being amortized using the effective interest method over the life of the arrangement, in accordance with ASC 470 and ASC 835. The initial annual effective interest rate was determined to be 10.8%. The Company utilizes the prospective method to account for subsequent changes in the estimated future payments to be made to Royalty Pharma and updates the effective interest rate on a quarterly basis. Issuance costs related to the transaction were determined to be immaterial. Refer to Note 7 for further details.

Leases

The Company determines if an arrangement is a lease at inception. This determination generally depends on whether the arrangement conveys to the Company the right to control the use of an explicitly or implicitly identified fixed asset for a period of time in exchange for consideration. Control of an underlying asset is conveyed to the Company if the Company obtains the rights to direct the use of and to obtain substantially all of the economic benefits from using the underlying asset. The Company has lease agreements which include lease and non-lease components, which the Company accounts for as a single lease component for all leases. Operating and finance leases are classified as right of use ("ROU") assets, short term lease liabilities, and long term lease liabilities. Operating and finance lease ROU assets and lease liabilities are recognized at the commencement date based on the present value of lease payments over the lease term. ROU assets are amortized and lease liabilities accrete to yield straight-line expense over the term of the lease. Lease payments included in the measurement of the lease liability are comprised of fixed payments.

Variable lease payments associated with the Company’s leases are recognized when the event, activity, or circumstance in the lease agreement on which those payments are assessed occurs. Variable lease payments are presented in the Company’s consolidated statements of operations in the same line item as expense arising from fixed lease payments for operating leases.

Leases with an initial term of 12 months or less are not recorded on the consolidated balance sheet and the Company recognizes lease expense for these leases on a straight-line basis over the lease term. The Company applies this policy to all underlying asset categories.

A lessee is required to discount its unpaid lease payments using the interest rate implicit in the lease or, if that rate cannot be readily determined, its incremental borrowing rate. As most of the Company’s leases do not provide an implicit rate, the Company uses its incremental borrowing rate based on the information available at the commencement date in determining the present value of lease payments. The Company gives consideration to its recent debt issuances as well as publicly available data for instruments with similar characteristics when calculating its incremental borrowing rates.

The lease term for all of the Company’s leases includes the non-cancellable period of the lease plus any additional periods covered by either a Company option to extend (or not to terminate) the lease that the Company is reasonably

certain to exercise, or an option to extend (or not to terminate) the lease controlled by the lessor. Leasehold improvements are capitalized and depreciated over the lesser of useful life or lease term. See Note 5 Leases for additional information.

Research and development costs

Research and development expenses include the clinical development costs associated with the Company’s product development programs and research and development costs associated with the Company’s discovery programs. These expenses include internal research and development costs and the costs of research and development conducted on behalf of the Company by third parties, including sponsored university-based research agreements and clinical study vendors. All research and development costs are expensed as incurred. Costs incurred in obtaining technology licenses are charged immediately to research and development expense if the technology licensed has not reached technological feasibility and has no alternative future uses.

Advance payments made for goods and services that will be used in future research and development activities are deferred if the contracted party has not yet performed the related activities. The amount deferred is then expensed when the research and development activities are performed. As of December 31, 2023 and 2022, the short term deferred research and development advance payments were $2.6 million and $2.4 million, respectively, and are classified as prepaid expenses and other current assets on the consolidated balance sheet. As of December 31, 2023 and 2022, the long term deferred research and development advance payments were $4.7 million and $3.9 million, respectively, and are classified as deposits and other assets on the consolidated balance sheet.

Fair value of financial instruments

The Company follows the fair value measurement rules, which provides guidance on the use of fair value in accounting and disclosure for assets and liabilities when such accounting and disclosure is called for by other accounting literature. These rules establish a fair value hierarchy for inputs to be used to measure fair value of financial assets and liabilities. This hierarchy prioritizes the inputs to valuation techniques used to measure fair value into three levels: Level 1 (highest priority), Level 2, and Level 3 (lowest priority).

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Level 1—Unadjusted quoted prices in active markets for identical assets or liabilities that the Company has the ability to access at the balance sheet date.

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Level 2—Inputs other than quoted prices included within Level 1 that are observable for the asset or liability, either directly or indirectly. Level 2 inputs include quoted prices for similar assets and liabilities in active markets, quoted prices for identical or similar assets or liabilities in markets that are not active, inputs other than quoted prices that are observable for the asset or liability (i.e., interest rates, yield curves, etc.), and inputs that are derived principally from or corroborated by observable market data by correlation or other means (market corroborated inputs).

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Level 3—Inputs are unobservable and reflect the Company’s assumptions as to what market participants would use in pricing the asset or liability. The Company develops these inputs based on the best information available.

Cash equivalents, marketable securities, and equity investments are reflected in the accompanying financial statements at fair value. The carrying amount of receivables and accounts payable and accrued expenses approximates fair value due to the short-term nature of those instruments.

Share-based compensation

The Company measures the cost of employee services received in exchange for an award of equity instruments based on the grant date fair value of the award. Restricted stock awards are measured based on the fair market values of the underlying stock on the dates of grant. For service type awards, share-based compensation expense is recognized on a straight-line basis over the period during which the employee is required to provide service in exchange for the entire award.

The fair value of options is calculated using the Black-Scholes option pricing model to determine the fair value of stock options on the date of grant based on key assumptions such as expected volatility and expected term. The Company estimates the expected volatility of options utilizing the Company’s historical stock volatility. The Company estimates the expected term of options utilizing the Company’s historical exercise data. The risk-free rate of the option is based on U.S. Government Securities Treasury Constant Maturities yields at the date of grant for a term similar to the expected term of the option. In connection with the adoption of FASB Accounting Standards Update (“ASU”) 2016-9, the Company made a policy election to continue its methodology for estimating its forfeiture rate.

Stock-based compensation expense for performance stock units (“PSUs”) is determined using the grant date fair value, which is the quoted closing market price per share of the Company’s common stock on the Nasdaq Global Select Market on the grant date. Stock-based compensation expense for the PSUs will not be recognized until the achievement of the performance goal is deemed probable (the “Probable Date”), a determination that requires significant judgment by management, as the achievement of these goals have inherent risk and uncertainties. At the Probable Date, the Company records a cumulative catch-up expense for the portion of the grant date fair value attributable to the period from the grant date to the Probable Date. The remaining expense is recognized over the remaining service period on a straight-line basis.

Income Taxes

Income taxes

On December 15, 2022, the European Union (EU) member states formally adopted the EU’s Pillar Two Directive, which generally provides for a minimum effective tax rate of 15%, as established by the Organization for Economic Co-operation and Development (OECD) Pillar Two Framework that was supported by over 130 countries worldwide. The EU effective dates are January 1, 2024, and January 1, 2025, for different aspects of the directive. A significant number of other countries are also implementing similar legislation. As a result, the tax laws in the U.S. and other countries in which PTC and its affiliates do business could change on a prospective or retroactive basis and any such changes could materially adversely affect the Company’s business. The Company is continuing to evaluate the potential impact on future periods of the Pillar Two Framework, pending legislative adoption by additional individual countries, including those within the EU.

On December 22, 2017, the U.S. government enacted the 2017 Tax Cuts and Jobs Act (“TCJA”), which significantly revised U.S. tax law by, among other provisions, lowering the U.S. federal statutory income tax rate to 21%, imposing a mandatory one-time transition tax on previously deferred foreign earnings, and eliminating or reducing certain income tax deductions. The Global Intangible Low-tax Income ("GILTI") provisions of the TCJA require the Company to include in its U.S. income tax return foreign subsidiary earnings in excess of an allowable return on the foreign subsidiary’s tangible assets. The Company has elected to account for GILTI tax in the period in which it is incurred, and therefore has not provided any deferred tax impacts of GILTI in its consolidated financial statements for the period ended December 31, 2023.

Starting in 2022, TCJA amendments to IRC Section 174 no longer permits an immediate deduction for research and development (R&D) expenditures in the tax year that such costs are incurred. Instead, these IRC Section 174 development

costs must now be capitalized and amortized over either a five- or 15-year period, depending on the location of the activities performed. The new amortization period begins with the midpoint of any taxable year that IRC Section 174 costs are first incurred, regardless of whether the expenditures were made prior to or after July 1 and runs until the midpoint of year five for activities conducted in the United States or year 15 in the case of development conducted on foreign soil. This tax law change resulted in an increased current taxable income of the Company by $300.3 million for the year ended December 31, 2023.

Deferred tax assets and liabilities are recognized for the future tax consequences attributable to differences between the financial statement carrying amounts of existing assets and liabilities and their respective tax bases and net operating loss and credit carryforwards. Deferred tax assets and liabilities are measured at rates expected to apply to taxable income in the years in which those temporary differences and carryforwards are expected to be recovered or settled. The effect on deferred tax assets and liabilities of a change in tax rates is recognized in the statement of operations in the period that includes the enactment date. A valuation allowance is recorded when it is not more likely than not that all or a portion of the net deferred tax assets will be realized.

On August 23, 2018, the Company completed its acquisition of Agilis Biotherapeutics, Inc. (“Agilis”), pursuant to an Agreement and Plan of Merger, dated as of July 19, 2018 (the “Agilis Merger Agreement”), by and among the Company, Agility Merger Sub, Inc., a Delaware corporation and the Company’s wholly owned, indirect subsidiary, Agilis and, solely in its capacity as the representative, agent and attorney-in-fact of the equityholders of Agilis, Shareholder Representative Services LLC, (the “Agilis Merger”). The Company recorded a deferred tax liability in conjunction with the Agilis Merger of $122.0 million in 2018, related to the tax basis difference in the In-Process Research and Development, or IPR&D, indefinite-lived intangibles acquired. The Company’s policy is to record a deferred tax liability related to acquired IPR&D which may eventually be realized either upon amortization of the asset when the research is completed and a product is successfully launched or the write-off of the asset if it is abandoned or unsuccessful. In July 2022, the Company received EMEA approval for a portion of the IPR&D assets, and thus, began the amortization of the intangible.

In May 2023, as part of a strategic portfolio prioritization, the Company announced the discontinuation of its preclinical and early research programs for its gene therapy platform, which included programs for Friedreich ataxia and Angelman syndrome. In conjunction with the announcement, the Company recorded an impairment to its indefinite-lived intangible for IP research and development relating to the Friedreich ataxia and Angelman syndrome gene therapy assets. As a result of the impairment, the Company recorded a deferred tax benefit of $46.9 million during the 2023 tax year.

Foreign currency

The functional currencies of the Company’s foreign subsidiaries primarily are the local currencies of the country in which the subsidiary operates. The Company had an intercompany loan which was recorded on a non-U.S. subsidiary and was formally denominated in U.S. dollars and was remeasured into local currency using the exchange rate as of the balance sheet date. During the year ended December 31, 2022, the loan agreement was amended to change the denomination from U.S. dollars to local currency, and the Company recorded a non-cash realized foreign exchange loss of $16.9 million, which was recorded within other income (expense), net on the consolidated statement of operations. The intercompany loan was settled during the year ended December 31, 2023. The Company’s asset and liability accounts, including the intercompany loan, are translated using the current exchange rate as of the balance sheet date. Stockholders’ equity accounts are translated using historical rates at the balance sheet date. Revenue and expense accounts are translated using a weighted average exchange rate over the period ended on the balance sheet date. Adjustments resulting from the translation of the financial statements of the Company’s foreign subsidiaries into U.S. dollars are accumulated as a separate component of stockholders’ equity within other comprehensive income. Gains or losses resulting from transactions denominated in foreign currencies are included in other income or expense, within the consolidated statements of income.

Net (loss) income per share

Basic net (loss) income per share is calculated by dividing the net (loss) income attributable to common stockholders by the weighted average number of common shares outstanding for the period, without consideration for common stock equivalents. Diluted net income per share is calculated by dividing the net income attributable to common stockholders by the weighted-average number of common share equivalents outstanding for the period determined using the treasury-stock method and the if-converted method. During periods in which the Company incurs net losses, both basic and diluted loss per share is calculated by dividing the net loss by the weighted average shares outstanding—potentially dilutive securities are excluded from the calculation because their effect would be anti-dilutive. Dilutive common stock equivalents are comprised of options and unvested restricted stock outstanding under the Company’s stock option plans.

Business combinations and asset acquisitions

The Company evaluates acquisitions of assets and other similar transactions to assess whether or not the transaction should be accounted for as a business combination or asset acquisition by first applying a screen to determine if substantially all of the fair value of the gross assets acquired is concentrated in a single identifiable asset or group of similar identifiable assets. If the screen is met, the transaction is accounted for as an asset acquisition. If the screen is not met, further determination is required as to whether or not the Company has acquired inputs and processes that have the ability to create outputs, which would meet the requirements of a business. If determined to be a business combination, the Company accounts for the transaction under the acquisition method of accounting as indicated in ASU 2017-01, “Business Combinations”, which requires the acquiring entity in a business combination to recognize the fair value of all assets acquired, liabilities assumed, and any non-controlling interest in the acquiree and establishes the acquisition date as the fair value measurement point. Accordingly, the Company recognizes assets acquired and liabilities assumed in business combinations, including contingent assets and liabilities, and non-controlling interest in the acquiree based on the fair value estimates as of the date of acquisition. In accordance with ASC 805, the Company recognizes and measures goodwill as of the acquisition date, as the excess of the fair value of the consideration paid over the fair value of the identified net assets acquired.

The consideration for the Company’s business acquisitions may include future payments that are contingent upon the occurrence of a particular event or events. The obligations for such contingent consideration payments are recorded at fair value on the acquisition date. The contingent consideration obligations are then evaluated each reporting period. Changes in the fair value of contingent consideration, other than changes due to payments, are recognized as a gain or loss and recorded within the change in the fair value of deferred and contingent consideration in the consolidated statements of operations.

If determined to be an asset acquisition, the Company accounts for the transaction under ASC 805-50, which requires the acquiring entity in an asset acquisition to recognize assets acquired and liabilities assumed based on the cost to the acquiring entity on a relative fair value basis, which includes transaction costs in addition to consideration given. No gain or loss is recognized as of the date of acquisition unless the fair value of noncash assets given as consideration differs from the assets’ carrying amounts on the acquiring entity’s books. Consideration transferred that is noncash will be measured based on either the cost (which will be measured based on the fair value of the consideration given) or the fair value of the assets acquired and liabilities assumed, whichever is more reliably measurable. Goodwill is not recognized in an asset acquisition and any excess consideration transferred over the fair value of the net assets acquired is allocated to the identifiable assets based on relative fair values.

Contingent consideration payments in asset acquisitions are recognized when the contingency is resolved and the consideration is paid or becomes payable (unless the contingent consideration meets the definition of a derivative, in which

case the amount becomes part of the basis in the asset acquired). Upon recognition of the contingent consideration payment, the amount is included in the cost of the acquired asset or group of assets.

Finite-lived intangible assets

The Company records the fair value of purchased intangible assets with finite useful lives as of the transaction date of a business combination or asset acquisition. Purchased intangible assets with finite useful lives are amortized to their estimated residual values over their estimated useful lives.

Impairment of long-lived assets

The Company monitors its long-lived assets and finite-lived intangibles for indicators of impairment. If such indicators are present, the Company assesses the recoverability of affected assets by determining whether the carrying value of such assets is less than the sum of the undiscounted future cash flows of the assets. If such assets are found not to be recoverable, the Company measures the amount of such impairment by comparing the carrying value of the assets to the fair value of the assets, with the fair value generally determined based on the present value of the expected future cash flows associated with the assets. The Company believes that no impairment of long-lived assets exists as of December 31, 2023.

Indefinite-lived intangible assets

Indefinite-lived intangible assets consist of IPR&D. IPR&D acquired directly in a transaction other than a business combination is capitalized if the projects will be further developed or have an alternative future use; otherwise they are expensed. The fair values of IPR&D projects and license agreement assets acquired in business combinations are capitalized. Several methods may be used to determine the estimated fair value of the IPR&D and license agreement asset acquired in a business combination. The Company utilizes the “income method” and uses estimated future net cash flows that are derived from projected sales revenues and estimated costs. These projections are based on factors such as relevant market size, patent protection, and expected pricing and industry trends. The estimated future net cash flows are then discounted to the present value using an appropriate discount rate. These assets are treated as indefinite-lived intangible assets until completion or abandonment of the projects, at which time the assets are amortized over the remaining useful life or written off, as appropriate. Intangible assets with indefinite lives, including IPR&D, are tested for impairment if impairment indicators arise and, at a minimum, annually. However, an entity is permitted to first assess qualitative factors to determine if a quantitative impairment test is necessary. Further testing is only required if the entity determines, based on the qualitative assessment, that it is more likely than not that an indefinite-lived intangible asset’s fair value is less than its carrying amount. Otherwise, no further impairment testing is required. The indefinite-lived intangible asset impairment test consists of a one-step analysis that compares the fair value of the intangible asset with its carrying amount. If the carrying amount of an intangible asset exceeds its fair value, an impairment loss is recognized in an amount equal to that excess. The Company considers many factors in evaluating whether the value of its intangible assets with indefinite lives may not be recoverable, including, but not limited to, expected growth rates, the cost of equity and debt capital, general economic conditions, the Company’s outlook and market performance of the Company’s industry and recent and forecasted financial performance.

The Company performed an annual test for its remaining indefinite-lived intangible asset as of October 1, 2023 and concluded that no impairment exists as of December 31, 2023.

Goodwill

Goodwill represents the amount of consideration paid in excess of the fair value of net assets acquired as a result of the Company’s business acquisitions accounted for using the acquisition method of accounting. Goodwill is not amortized and is subject to impairment testing at a reporting unit level on an annual basis or when a triggering event occurs that may indicate the carrying value of the goodwill is impaired. The Company reassesses its reporting units as part of its annual segment review. As of December 31, 2023, the Company concluded that it continues to operate as one reporting unit. An entity is permitted to first assess qualitative factors to determine if a quantitative impairment test is necessary. Further testing is only required if the entity determines, based on the qualitative assessment, that it is more likely than not that the fair value of the reporting unit is less than its carrying amount. The Company performed an annual test for goodwill as of October 1, 2023. The Company’s single reporting unit had a negative carrying value and thus the Company determined there was no impairment of goodwill.

Recent accounting pronouncements

In November 2023, the FASB issued ASU 2023-07, Segment Reporting (Topic 280) – Improvements to Reportable Segment Disclosures. This ASU requires that a public entity provide additional segment disclosures on an interim and annual basis. The amendments in this ASU should be applied retrospectively to all prior periods presented in the financial statements, unless impracticable. Upon transition, the segment expense categories and amounts disclosed in the prior periods should be based on the significant segment expense categories identified and disclosed in the period of adoption. The ASU is effective for fiscal years beginning after December 15, 2023 and interim periods within fiscal years beginning after December 15, 2024. Early adoption is permitted. The Company is currently planning to adopt this guidance when effective. The Company is assessing the impact of the adoption on the Company’s consolidated financial statements and accompanying footnotes.

In December 2023, the FASB issued ASU 2023-09, Improvements to Income Tax Disclosures. ASU 2023-09 enhances the transparency about income tax information through improvements to income tax disclosures primarily related to the rate reconciliation and income taxes paid information. The guidance is effective for public business entities for annual periods beginning after December 15, 2024. For entities other than public business entities, the amendments are effective for annual periods beginning after December 15, 2025. Early adoption is permitted. The Company is currently planning to adopt this guidance when effective. The Company is assessing the impact of the adoption on the Company’s consolidated financial statements and accompanying footnotes.

The Company

12 Months Ended

Dec. 31, 2023

Organization, Consolidation and Presentation of Financial Statements [Abstract]

The Company

1. The Company

PTC Therapeutics, Inc. (the “Company” or “PTC”) is a global biopharmaceutical company focused on the discovery, development and commercialization of clinically differentiated medicines that provide benefits to patients with rare disorders. PTC’s ability to innovate to identify new therapies and to globally commercialize products is the foundation that drives investment in a robust and diversified pipeline of transformative medicines. PTC’s mission is to provide access to best-in-class treatments for patients who have little to no treatment options. PTC’s strategy is to leverage its strong scientific and clinical expertise and global commercial infrastructure to bring therapies to patients. PTC believes that this allows it to maximize value for all of its stakeholders.

The Company has two products, Translarna™ (ataluren) and Emflaza^® (deflazacort), for the treatment of Duchenne muscular dystrophy (“DMD”), a rare, life threatening disorder. Translarna has marketing authorization in the European Economic Area (the “EEA”) for the treatment of nonsense mutation Duchenne muscular dystrophy (“nmDMD”) in ambulatory patients aged 2 years and older. In July 2020, the European Commission (“EC”) approved the removal of the statement “efficacy has not been demonstrated in non-ambulatory patients” from the indication statement for Translarna. Translarna also has marketing authorization in Russia for the treatment of nmDMD in patients aged two years and older, and in Brazil for the treatment of nmDMD in ambulatory patients two years and older and for continued treatment of patients that become non-ambulatory, as well as in various other countries. Emflaza is approved in the United States for the treatment of DMD in patients two years and older.

The Company’s marketing authorization for Translarna in the EEA is subject to annual review and renewal by the EC following reassessment by the European Medicines Agency (“EMA”) of the benefit-risk balance of the authorization, which the Company refers to as the annual EMA reassessment. In September 2022, the Company submitted a Type II variation to the EMA to support conversion of the conditional marketing authorization for Translarna to a standard marketing authorization, which included a report on the placebo-controlled trial of Study 041 and data from the open-label extension. In February 2023, the Company also submitted an annual marketing authorization renewal request to the EMA. In September 2023, the Committee for Medicinal Products for Human Use (“CHMP’), gave a negative opinion on the conversion of the conditional marketing authorization to full marketing authorization of Translarna for the treatment of nmDMD and a negative opinion on the renewal of the existing conditional marketing authorization of Translarna for the treatment of nmDMD. On January 25, 2024, the CHMP issued a negative opinion for the renewal of the conditional marketing authorization following a re-examination procedure. In accordance with EMA regulations, the EC has 67 days to adopt the opinion. If the EC adopts the negative opinion, Translarna would no longer have marketing authorization in the member states of the EEA.

Translarna is an investigational new drug in the United States. Following the Company’s announcement of top-line results from the placebo-controlled trial of Study 041 in June 2022, the Company submitted a meeting request to the U.S. Food and Drug Administration (“FDA”) to gain clarity on the regulatory pathway for a potential re-submission of a New Drug Application (“NDA”) for Translarna. The FDA provided initial written feedback that Study 041 does not provide substantial evidence of effectiveness to support NDA re-submission. The Company held a Type C meeting with the FDA in the fourth quarter of 2023 to discuss the totality of Translarna data. Based on this discussion, the FDA suggested that the Company request a pre-submission Type C meeting to discuss the specific contents of an NDA resubmission based on results from Study 041 and from the Company’s international drug registry study for nmDMD patients receiving Translarna. This meeting is scheduled for March 2024.

The Company has developed Upstaza (eladocagene exuparvovec), a gene therapy used for the treatment of Aromatic L-Amino Acid Decarboxylase (“AADC”) deficiency (“AADC deficiency”), a rare central nervous system (“CNS”)

disorder arising from reductions in the enzyme AADC that results from mutations in the dopa decarboxylase gene. In July 2022, the EC approved Upstaza for the treatment of AADC deficiency for patients 18 months and older within the EEA. In November 2022, the Medicines and Healthcare Products Regulatory Agency approved Upstaza for the treatment of AADC deficiency for patients 18 months and older within the United Kingdom. The Company is also preparing a biologics license application (“BLA”) for Upstaza for the treatment of AADC deficiency in the United States and anticipates submitting a BLA to the FDA in March 2024.

The Company holds the rights for the commercialization of Tegsedi^® (inotersen) and Waylivra^® (volanesorsen) for the treatment of rare diseases in countries in Latin America and the Caribbean pursuant to the Collaboration and License Agreement (the “Tegsedi-Waylivra Agreement”), dated August 1, 2018, by and between the Company and Akcea Therapeutics, Inc. (“Akcea”), a subsidiary of Ionis Pharmaceuticals, Inc. Tegsedi has received marketing authorization in the United States, the European Union (the “EU”) and Brazil for the treatment of stage 1 or stage 2 polyneuropathy in adult patients with hereditary transthyretin amyloidosis (“hATTR amyloidosis”). In August 2021, ANVISA, the Brazilian health regulatory authority, approved Waylivra as the first treatment for familial chylomicronemia syndrome (“FCS”) in Brazil. In December 2022, ANVISA approved Waylivra for the treatment of familial partial lipodystrophy (“FPL”). Waylivra has also received marketing authorization in the EU for the treatment of FCS.

The Company also has a spinal muscular atrophy (“SMA”) collaboration with F. Hoffman-La Roche Ltd and Hoffman-La Roche Inc. (referred to collectively as “Roche”) and the Spinal Muscular Atrophy Foundation (“SMA Foundation”). The SMA program has one approved product, Evrysdi^® (risdiplam), which was approved by the FDA in August 2020 for the treatment of SMA in adults and children two months and older and by the EC in March 2021 for the treatment of 5q SMA in patients two months and older with a clinical diagnosis of SMA Type 1, Type 2 or Type 3 or with one to four SMN2 copies. Evrysdi has also received marketing authorization for the treatment of SMA in over 100 countries. In May 2022, the FDA approved a label expansion for Evrysdi to include infants under two months old with SMA. In August 2023, the EC approved an extension of the Evrysdi marketing authorization to include infants under two months old in the EU.

One of the Company’s most advanced clinical stage molecules is sepiapterin. Sepiapterin is a precursor to intracellular tetrahydrobiopterin, which is a critical enzymatic cofactor involved in metabolism and synthesis of numerous metabolic products, for orphan diseases. In May 2023, the Company announced that the primary endpoint was achieved in its registration-directed Phase 3 trial for sepiapterin for phenylketonuria (“PKU”). The primary endpoint of the study was the achievement of statistically-significant reduction in blood Phe level. PTC expects to submit a marketing authorization application (“MAA”) to the EMA for sepiapterin for the treatment of PKU in the EEA in March 2024. Additionally, the Company participated in a pre-NDA meeting with the FDA in the third quarter of 2023. At that meeting, the FDA stated that the sepiapterin clinical safety and efficacy data supported NDA submission for the treatment of pediatric and adult PKU patients. However, the FDA has requested that PTC completes a 26-week nonclinical mouse study to assess sepiapterin carcinogenicity potential prior to NDA submission. Based on the timing to complete this study, PTC expects to submit an NDA to the FDA for sepiapterin for the treatment of PKU no later than the third quarter of 2024 and PTC intends to discuss with the FDA the potential for an earlier submission if PTC is permitted to submit the 26-week mouse study report during the NDA review.

In addition to the Company’s SMA program, the Company’s splicing platform also includes PTC518, which is being developed for the treatment of Huntington’s disease (“HD”). The Company initiated a Phase 2 study of PTC518 for the treatment of HD in the first quarter of 2022, which consists of an initial 12-week placebo-controlled phase focused on safety, pharmacology and pharmacodynamic effects followed by a nine-month placebo-controlled phase focused on PTC518 biomarker effect. In June 2023, the Company announced interim data from the 12-week placebo-controlled phase. The Phase 2 study is actively ongoing outside the United States, while it has been paused within the United States as the

FDA requested additional data to allow the Phase 2 study to proceed. The Company expects results from the 12-month interim data from the Phase 2 study of PTC518 for the treatment of HD in the second quarter of 2024. The Company expects to submit a safety data update to the FDA in the second quarter of 2024 to support lifting of the partial clinical hold on the program.

The Company’s ferroptosis and inflammation platform consists of small molecule compounds that target oxidoreductase enzymes that regulate oxidative stress and inflammatory pathways central to the pathology of a number of CNS diseases. The two most advanced molecules in the Company’s ferroptosis and inflammation platform are vatiquinone and utreloxastat. The Company announced topline results from a registration-directed Phase 3 trial of vatiquinone in children and young adults with Friedreich ataxia, called MOVE-FA, in May 2023. While the study did not meet its primary endpoint, vatiquinone treatment did demonstrate significant benefit on key disease subscales, including the upright stability subscale, as well as on other disease relevant endpoints. In the first quarter of 2024, the Company met with the FDA, who expressed willingness to review an NDA for vatiquinone for the treatment of Friedreich ataxia based on the MOVE-FA trial as well as data from the ongoing open label extension study following the MOVE-FA trial, potentially allowing for the submission of an NDA in late 2024. In the first quarter of 2024, the Company also received scientific advice from the EMA on the MOVE-FA trial results, in which the EMA stated that the MOVE-FA data would likely not be sufficient for conditional authorization. The Company initiated a Phase 2 registration directed trial of utreloxastat for amyotrophic lateral sclerosis, or ALS, in the first quarter of 2022. The Company expect topline results from this trial in the fourth quarter of 2024.

In addition, the Company has a pipeline of product candidates and discovery programs that are in early clinical, pre-clinical and research and development stages focused on the development of new treatments for multiple therapeutic areas for rare diseases.

As of December 31, 2023, the Company had an accumulated deficit of approximately $3,283.6 million. The Company has financed its operations to date primarily through the private offerings of convertible senior notes (see Note 7), public and “at the market offerings” of common stock, proceeds from royalty purchase agreements (see Note 2), net proceeds from its borrowings under its credit agreement with Blackstone (see Note 7), private placements of its convertible preferred stock and common stock, collaborations, bank and institutional lender debt, other convertible debt, grant funding and clinical trial support from governmental and philanthropic organizations and patient advocacy groups in the disease area addressed by the Company’s product candidates. The Company has also relied on revenue generated from net sales of Translarna for the treatment of nmDMD in territories outside of the United States since 2014, Emflaza for the treatment of DMD in the United States since 2017 and Upstaza for the treatment of AADC deficiency in the EEA since May 2022. The Company has also relied on revenue associated with milestone and royalty payments from Roche pursuant to the License and Collaboration Agreement (the “SMA License Agreement”) dated as of November 23, 2011, by and among the Company, Roche and, for the limited purposes set forth therein, the SMA Foundation, under its SMA program. The Company expects that cash flows from the sales of its products, together with the Company’s cash, cash equivalents and marketable securities, will be sufficient to fund its operations for at least the next twelve months.

Consolidated Statements of Operations - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2023	Dec. 31, 2022	Dec. 31, 2021
Revenues:
Revenue	$ 937,822	$ 698,801	$ 538,593
Operating expenses:
Cost of product sales, excluding amortization of acquired intangible assets	65,486	44,678	32,328
Amortization of intangible assets	222,635	116,554	54,751
Research and development	666,563	651,496	540,684
Selling, general and administrative	332,540	325,998	285,773
Change in the fair value of contingent consideration	(127,700)	(25,900)	(500)
Intangible asset impairment	217,800	33,384
Total operating expenses	1,377,324	1,146,210	913,036
Loss from operations	(439,502)	(447,409)	(374,443)
Interest expense, net	(129,180)	(90,871)	(86,022)
Other income (expense), net	10,130	(49,207)	(57,875)
Loss on extinguishment of debt	(137,558)
Loss before income tax benefit (expense)	(696,110)	(587,487)	(518,340)
Income tax benefit (expense)	69,506	28,470	(5,561)
Net loss attributable to common stockholders	$ (626,604)	$ (559,017)	$ (523,901)
Weighted-average shares outstanding:
Basic	74,838,392	71,728,634	70,466,393
Diluted	74,838,392	71,728,634	70,466,393
Net loss per share-basic and diluted (in dollars per share)
Basic	$ (8.37)	$ (7.79)	$ (7.43)
Diluted	$ (8.37)	$ (7.79)	$ (7.43)
Net product revenue
Revenues:
Revenue	$ 661,249	$ 535,228	$ 428,904
Collaboration revenue
Revenues:
Revenue	100,030	50,052	55,046
Royalty revenue
Revenues:
Revenue	168,856	113,521	54,643
Manufacturing revenue
Revenues:
Revenue	$ 7,687	$ 0	$ 0

Revenue recognition - Performance Obligations (Details) - USD ($) $ in Millions	Dec. 31, 2023	Dec. 31, 2022
Remaining Performance Obligation
Remaining performance obligations		$ 1.4
Revenue, Remaining Performance Obligation, Expected Timing of Satisfaction, Start Date [Axis]: 2024-01-01
Remaining Performance Obligation
Remaining performance obligations	$ 0.8
Revenue, performance obligation, period	1 year

Fair value of financial instruments and investments - (Tables)

12 Months Ended

Dec. 31, 2023

Fair Value Disclosures [Abstract]

Schedule of marketable securities

The table presented below is a summary of changes in the fair value for the Company’s marketable securities – equity investments, ClearPoint Equity Investments, and ClearPoint convertible debt security for the years ended December 31, 2023 and 2022:


	Ending						Foreign						Ending
	Balance at						Currency						Balance at
	December 31,		Unrealized		Realized		Unrealized		Investments		Redemptions/		December 31,
	2022		Gain/(Loss)		Gain/(Loss)		Gain		Purchased		Sale		2023
Marketable securities - equity investments	$	108,261		2,517		4,383		1,384		38,432		(132,343)	$	22,634
ClearPoint Equity Investments		10,965		(1,515)		(782)		—		—		(2,594)		6,074
ClearPoint Convertible Debt Security		15,231		(2,678)		—		—		—		—		12,553
Total Fair Value	$	134,457	$	(1,676)	$	3,601	$	1,384	$	38,432	$	(134,937)	$	41,261


	Ending				Foreign						Ending
	Balance at				Currency						Balance at
	December 31,		Unrealized		Unrealized		Investments				December 31,
	2021		Gain/(Loss)		Gain/(Loss)		Purchased		Redemptions		2022
Marketable securities - equity investments	$	206,973		(7,992)		(549)		22,787		(112,958)	$	108,261
ClearPoint Equity Investments		14,525		(3,560)		—		—		—		10,965
ClearPoint Convertible Debt Security		20,971		(5,740)		—		—		—		15,231
Total Fair Value	$	242,469	$	(17,292)	$	(549)	$	22,787	$	(112,958)	$	134,457

Schedule of financial assets and liabilities that are required to be measured at fair value on a recurring basis

The following represents the fair value using the hierarchy described in Note 2 for the Company’s financial assets and liabilities that are required to be measured at fair value on a recurring basis as of December 31, 2023 and 2022:


	December 31, 2023
			Quoted prices		Significant
			in active		other		Significant
			markets for		observable		unobservable
			identical assets		inputs		inputs
	Total		(level 1)		(level 2)		(level 3)
Marketable securities - available for sale	$	260,104	$	—	$	260,104	$	—
Marketable securities - equity investments	$	22,634	$	22,634	$	—	$	—
ClearPoint Equity Investments	$	6,074	$	6,074	$	—	$	—
ClearPoint convertible debt security	$	12,553	$	—	$	12,553	$	—
Contingent consideration payable- development and regulatory milestones	$	26,600	$	—	$	—	$	26,600
Contingent consideration payable- net sales milestones	$	9,700	$	—	$	—	$	9,700


	December 31, 2022
			Quoted prices		Significant
			in active		other		Significant
			markets for		observable		unobservable
			identical assets		inputs		inputs
	Total		(level 1)		(level 2)		(level 3)
Marketable securities - available for sale	$	22,610	$	—	$	22,610	$	—
Marketable securities - equity investments	$	108,261	$	108,261	$	—	$	—
ClearPoint Equity Investments	$	10,965	$	10,965	$	—	$	—
ClearPoint convertible debt security	$	15,231	$	—	$	15,231	$	—
Contingent consideration payable- development and regulatory milestones	$	82,500	$	—	$	—	$	82,500
Contingent consideration payable- net sales milestones and royalties	$	81,500	$	—	$	—	$	81,500

Summary of marketable securities accounted for as available-for-sale debt securities

The following is a summary of marketable securities accounted for as available for sale debt securities at December 31, 2023 and 2022:


	December 31, 2023
	Amortized		Gross Unrealized
	Cost		Gains		Losses		Fair Value
Commercial paper	$	117,044	$	128	$	(12)	$	117,160
Corporate debt securities		1,650		—		(2)		1,648
Government Obligations		141,084		212		—		141,296
Total	$	259,778	$	340	$	(14)	$	260,104


	December 31, 2022
	Amortized		Gross Unrealized
	Cost		Gains		Losses		Fair Value
Commercial paper	$	12,419	$	5	$	—	$	12,424
Corporate debt securities		10,685		—		(499)		10,186
Total	$	23,104	$	5	$	(499)	$	22,610

Summary of unrealized losses and fair values of available-for-sale debt securities in a continuous unrealized loss position

The unrealized losses and fair values of available for sale debt securities that have been in an unrealized loss position for a period of less than and greater than or equal to 12 months as of December 31, 2023 are as follows:


	December 31, 2023
	Securities in an unrealized loss				Securities in an unrealized loss
	position less than 12 months				position greater than or equal to 12 months				Total
	Unrealized losses		Fair Value		Unrealized losses		Fair Value		Unrealized losses		Fair Value
Commercial paper	$	(12)		44,446		—		—		(12)		44,446
Corporate debt securities	$	—		—		(2)		1,648		(2)	$	1,648
Total	$	(12)	$	44,446	$	(2)	$	1,648	$	(14)	$	46,094


	December 31, 2022
	Securities in an unrealized loss				Securities in an unrealized loss
	position less than 12 months				position greater than or equal to 12 months				Total
	Unrealized losses		Fair Value		Unrealized losses		Fair Value		Unrealized losses		Fair Value
Corporate debt securities	$	—		—		(499)		10,186		(499)	$	10,186
Total	$	—	$	—	$	(499)	$	10,186	$	(499)	$	10,186

Schedule of available-for-sale debt securities

Available for sale debt securities on the balance sheet at December 31, 2023 and 2022 mature as follows:


	December 31, 2023
	Less Than		More Than
	12 Months		12 Months
Commercial paper	$	117,160	$	—
Corporate debt securities		1,648		—
Government obligations		141,296		—
Total	$	260,104	$	—


	December 31, 2022
	Less Than		More Than
	12 Months		12 Months
Commercial paper	$	12,424	$	—
Corporate debt securities		—		10,186
Total	$	12,424	$	10,186

Summary of changes in the fair value of the Company's Level 3 valuation for contingent consideration payable

The table presented below is a summary of changes in the fair value of the Company’s Level 3 valuation for the contingent consideration payables for the years ended December 31, 2023, and 2022:



	Contingent consideration payable-		Contingent consideration payable-
	development and regulatory		net sales milestones and royalties
	milestones - Agilis		- Agilis
Beginning balance as of December 31, 2021	$	139,300	$	100,600
Additions		—		—
Change in fair value		(6,800)		(19,100)
Payments		(50,000)		—
Ending balance as of December 31, 2022	$	82,500	$	81,500
Additions		—		—
Change in fair value		(55,900)		(71,800)
Payments		—		—
Ending balance as of December 31, 2023	$	26,600	$	9,700

Fair Value Measurement Inputs and Valuation Techniques

The following significant unobservable inputs were used in the valuation of the contingent consideration payables for the years ended December 31, 2023 and 2022:


	December 31, 2023
	Fair Value	Valuation Technique	Unobservable Input	Range
Contingent consideration payable- development and regulatory milestones	$26,600	Probability-adjusted discounted cash flow	Potential development and regulatory milestones Probabilities of success Discount rates Projected years of payments	$0 - $31 million 85% - 92% 5.8% - 6.1% 2024 - 2026
Contingent considerable payable- net sales milestones and royalties	$9,700	Option-pricing model with Monte Carlo simulation	Potential net sales milestones Probabilities of success Potential percentage of net sales for royalties Discount rate Projected years of payments	$0 - $50 million 85% - 100% 0% 11% 2026 - 2034


	December 31, 2022
	Fair Value	Valuation Technique	Unobservable Input	Range
Contingent consideration payable- development and regulatory milestones	$82,500	Probability-adjusted discounted cash flow	Potential development and regulatory milestones Probabilities of success Discount rates Projected years of payments	$0 - $331 million 25% - 92% 6.2% - 8.3% 2023 - 2029
Contingent considerable payable- net sales milestones and royalties	$81,500	Option-pricing model with Monte Carlo simulation	Potential net sales milestones Probabilities of success Potential percentage of net sales for royalties Discount rate Projected years of payments	$0 - $150 million 25% - 100% 2% - 6% 11.5% 2025 - 2041

- Definition

Tabular disclosure of investment in debt security measured at fair value with change in fair value recognized in other comprehensive income (available-for-sale).

+ References

Reference 1: http://www.xbrl.org/2009/role/commonPracticeRef
-Topic 320
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 9
-Subparagraph (a)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147481800/320-10-50-9

Reference 2: http://www.xbrl.org/2009/role/commonPracticeRef
-Topic 320
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 3
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147481800/320-10-50-3

Reference 3: http://www.xbrl.org/2009/role/commonPracticeRef
-Topic 320
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 2
-Subparagraph (b)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147481800/320-10-50-2

Reference 4: http://www.xbrl.org/2009/role/commonPracticeRef
-Topic 320
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 2
-Subparagraph (c)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147481800/320-10-50-2

Reference 5: http://www.xbrl.org/2009/role/commonPracticeRef
-Topic 320
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 2
-Subparagraph (a)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147481800/320-10-50-2

Reference 6: http://www.xbrl.org/2009/role/commonPracticeRef
-Topic 320
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 2
-Subparagraph (aa)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147481800/320-10-50-2

Reference 7: http://www.xbrl.org/2009/role/commonPracticeRef
-Topic 320
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 2
-Subparagraph (aaa)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147481800/320-10-50-2

Reference 8: http://www.xbrl.org/2009/role/commonPracticeRef
-Topic 320
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 2
-Subparagraph (d)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147481800/320-10-50-2

Reference 9: http://www.xbrl.org/2009/role/commonPracticeRef
-Topic 320
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 3
-Subparagraph (a)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147481800/320-10-50-3

Reference 10: http://www.xbrl.org/2009/role/commonPracticeRef
-Topic 320
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 3
-Subparagraph (b)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147481800/320-10-50-3

Reference 11: http://www.xbrl.org/2009/role/commonPracticeRef
-Topic 320
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 3
-Subparagraph (c)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147481800/320-10-50-3

Reference 12: http://www.xbrl.org/2009/role/commonPracticeRef
-Topic 320
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 3
-Subparagraph (d)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147481800/320-10-50-3

+ Details

Summary of significant accounting policies

12 Months Ended

Dec. 31, 2023

Accounting Policies [Abstract]

Summary of significant accounting policies

2. Summary of significant accounting policies

Basis of presentation

Use of estimates

Restricted Cash


	End of			Beginning of
	period-		period-
	December 31,		December 31,
	2023		2022
Cash and cash equivalents	$	594,001	$	279,834
Restricted cash included in deposits and other assets		16,283		16,091
Total Cash, cash equivalents and restricted cash per statement of cash flows	$	610,284	$	295,925

Consolidation

Segment and geographic information

Cash equivalents

Marketable securities

Concentration of credit risk

Fixed assets

Fixed assets are stated at cost. Depreciation is computed starting when the asset is placed into service on a straight-line basis over the estimated useful life of the related asset as follows:

Leasehold improvements		Lesser of useful life or lease term
Computer equipment and software		3 years
Machinery and lab equipment		7 years
Furniture and fixtures		7 years

Inventory and cost of product sales

Inventory

The following table summarizes the components of the Company’s inventory for the periods indicated:


	December 31, 2023		December 31, 2022
Raw materials	$	952	$	1,078
Work in progress		17,991		14,074
Finished goods		11,634		6,656
Total inventory	$	30,577	$	21,808

Cost of product sales

Accumulated other comprehensive income (loss)

Accumulated other comprehensive income (loss) consists of unrealized gains or losses on marketable securities and foreign currency translation adjustments.

Revenue recognition

Net product revenue

Collaboration and royalty revenue

For the years ended December 31, 2023, 2022 and 2021, the Company has recognized $168.9 million, $113.5 million, and $54.6 million of royalty revenue, respectively, related to Evrysdi.

Manufacturing Revenue

Allowance for doubtful accounts

Liability for sale of future royalties

Leases

Research and development costs

Fair value of financial instruments

●

Level 1—Unadjusted quoted prices in active markets for identical assets or liabilities that the Company has the ability to access at the balance sheet date.

●

Share-based compensation

Income taxes

Foreign currency

Net (loss) income per share

Business combinations and asset acquisitions

Finite-lived intangible assets

Impairment of long-lived assets

Indefinite-lived intangible assets

The Company performed an annual test for its remaining indefinite-lived intangible asset as of October 1, 2023 and concluded that no impairment exists as of December 31, 2023.

Goodwill

Recent accounting pronouncements

Debt - Liability for Sale of Future Royalties (Details) $ in Thousands	12 Months Ended
	Dec. 31, 2023 USD ($)
Debt Disclosure [Abstract]
Beginning balance	$ 757,886
Less: Non-cash royalty revenue payable to RPI	(93,460)
Plus: non-cash interest expense recognized	104,790
Cash consideration	1,000,000
Loss on Debt Extinguishment	44,881
Ending balance	$ 1,814,097
Effective interest rate of the liability component	10.80%

Capital structure

12 Months Ended

Dec. 31, 2023

Stockholders' Equity Note [Abstract]

Capital structure

8. Capital structure

Common stock

In August 2019, the Company entered into an At the Market Offering Sales Agreement (the “Sales Agreement”) with Cantor Fitzgerald and RBC Capital Markets, LLC (together, the “Sales Agents”), pursuant to which, the Company may offer and sell shares of its common stock, having an aggregate offering price of up to $125.0 million from time to time through the Sales Agents by any method that is deemed to be an “at the market offering” as defined in Rule 415(a)(4) promulgated under the Securities Act of 1933, as amended. No shares were sold pursuant to the Sales Agreement during the years ended December 31, 2023, 2022, and 2021. The remaining shares of the Company’s common stock available to be issued and sold, under the Sales Agreement, have an aggregate offering price of up to $93.0 million as of December 31, 2023.

In June 2021, the Company filed a Certificate of Amendment to its Restated Certificate of Incorporation, which increased the number of authorized shares of the Company’s common stock from 125,000,000 to 250,000,000 shares.

In connection with the execution of the Blackstone Credit Agreement, the Company and certain entities affiliated with the Lenders (the “Purchasers”) also entered into a stock purchase agreement (the “Stock Purchase Agreement”) on the Closing Date for the sale and issuance of 1,095,290 shares of common stock (the “Shares”) to the Purchasers at a price of $45.65 per share, for an aggregate purchase price of approximately $50.0 million. The per share price represents the closing price of the Company’s common stock on the Nasdaq Global Select Market on October 26, 2022.

Under the Stock Purchase Agreement, the Company agreed to register the resale of the Shares on a registration statement to be filed with the Securities and Exchange Commission within 60 days of the Closing Date. The Company agreed to keep such registration statement effective for a period of six months following the Closing Date. In addition, subject to certain conditions, the Purchasers were entitled to participate in registered underwritten public offerings by the Company during such period.

Pursuant to the terms of the Stock Purchase Agreement, the Purchasers and certain of their controlled affiliates agreed not to, without the prior written approval of the Company and subject to specified conditions, directly or indirectly acquire shares of the Company’s outstanding common stock in excess of specified thresholds, seek or propose any acquisition of all or substantially all of the assets of the Company, seek or propose a merger or other business combination involving the Company, solicit proxies or consents with respect to any securities of the Company, seek to influence the management, board of directors or policies of the Company, or undertake other specified actions related to the potential acquisition of additional equity interests in the Company, or to encourage others to do any of the above (the “Standstill Restrictions”). The Standstill Restrictions terminated upon the termination of the Blackstone Credit Agreement.

The Purchasers also agreed not to sell or transfer the Shares without the prior written approval of the Company for a period of 90 days following the Closing Date, subject to certain exceptions.

In February 2023, the Company completed enrollment of its Phase 3 placebo-controlled clinical trial for sepiapterin for PKU. In connection with this event and pursuant to the Censa Merger Agreement, the Company paid a $30.0 million development milestone to the former Censa securityholders during the year ended December 31, 2023. The Company elected to pay this milestone in the form of shares of its common stock, less certain cash payments in accordance with the Censa Merger Agreement. Pursuant to such election, the Company issued 657,462 shares of its common stock and paid $0.4 million to the former Censa securityholders.

As of December 31, 2023, the Company’s number of authorized shares of common stock was 250,000,000.

- Definition

The entire disclosure for equity.

+ References

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+ Details

Pay vs Performance Disclosure - USD ($) $ in Thousands	12 Months Ended
Pay vs Performance Disclosure - USD ($) $ in Thousands	Dec. 31, 2023	Dec. 31, 2022	Dec. 31, 2021
Pay vs Performance Disclosure
Net Income (Loss)	$ (626,604)	$ (559,017)	$ (523,901)

- Definition

The portion of profit or loss for the period, net of income taxes, which is attributable to the parent.

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+ Details

Fixed assets (Tables)

12 Months Ended

Dec. 31, 2023

Property, Plant and Equipment [Abstract]

Schedule of fixed assets, net

Fixed assets, net were as follows at December 31, 2023 and 2022:


	December 31,
	2023		2022
Leasehold improvements	$	30,166	$	28,969
Computer equipment and software		17,503		15,332
Machinery and lab equipment		62,837		47,496
Furniture and fixtures		3,849		3,812
Assets in process		24,008		14,349
		138,363		109,958
Less accumulated depreciation		(51,274)		(37,368)
Total	$	87,089	$	72,590

Stock award plan - Share-based compensation expense (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2023	Dec. 31, 2022	Dec. 31, 2021
Stock option plan
Share-based compensation expense	$ 101,636	$ 110,333	$ 103,513
Unrecognized compensation cost	$ 119,600
Weighted average remaining service period for recognition of unrecognized compensation cost	2 years 1 month 6 days
Research and development
Stock option plan
Share-based compensation expense	$ 52,941	55,869	53,632
Selling, general and administrative
Stock option plan
Share-based compensation expense	$ 48,695	$ 54,464	$ 49,881

- Definition

Line items represent financial concepts included in a table. These concepts are used to disclose reportable information associated with domain members defined in one or many axes to the table.

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-URI https://asc.fasb.org//1943274/2147480429/718-10-50-2

Reference 18: http://www.xbrl.org/2003/role/disclosureRef
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-Name Accounting Standards Codification
-Section 50
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-Subparagraph (c)(2)(iii)(02)
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Reference 19: http://www.xbrl.org/2003/role/disclosureRef
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Reference 20: http://www.xbrl.org/2003/role/disclosureRef
-Topic 718
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 2
-Subparagraph (d)(1)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147480429/718-10-50-2

Reference 21: http://www.xbrl.org/2003/role/disclosureRef
-Topic 718
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-Name Accounting Standards Codification
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-Paragraph 2
-Subparagraph (d)(2)
-Publisher FASB
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Reference 22: http://www.xbrl.org/2003/role/disclosureRef
-Topic 718
-SubTopic 10
-Name Accounting Standards Codification
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-Subparagraph (e)(1)
-Publisher FASB
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Reference 23: http://www.xbrl.org/2003/role/disclosureRef
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Reference 24: http://www.xbrl.org/2003/role/disclosureRef
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Reference 25: http://www.xbrl.org/2003/role/disclosureRef
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Reference 26: http://www.xbrl.org/2003/role/disclosureRef
-Topic 718
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-Subparagraph (f)(2)(iii)
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Reference 27: http://www.xbrl.org/2003/role/disclosureRef
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-Name Accounting Standards Codification
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-Publisher FASB
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Reference 28: http://www.xbrl.org/2003/role/disclosureRef
-Topic 718
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-Section 50
-Paragraph 2
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-Publisher FASB
-URI https://asc.fasb.org//1943274/2147480429/718-10-50-2

+ Details

Other comprehensive income (loss) and accumulated other comprehensive items - (Tables)

12 Months Ended

Dec. 31, 2023

Comprehensive Income (Loss), Net of Tax, Attributable to Parent [Abstract]

Summary of other comprehensive income (loss) and the changes in accumulated other comprehensive items

The following table summarizes other comprehensive (loss) income and the changes in accumulated other comprehensive items, by component, for the years ended December 31, 2023, 2022, and 2021, respectively.


	Unrealized				Total
	Gains (Losses)				Accumulated
	On		Foreign		Other
	Marketable		Currency		Comprehensive
	Securities, net of tax		Translation		Items
Balance at December 31, 2020	$	1,900	$	(62,857)	$	(60,957)
Other comprehensive (loss) income before reclassifications		(3,279)		39,177		35,898
Amounts reclassified from other comprehensive items		777		—		777
Other comprehensive (loss) income		(2,502)		39,177		36,675
Balance at December 31, 2021	$	(602)	$	(23,680)	$	(24,282)
Other comprehensive income before reclassifications		4,072		28,970		33,042
Amounts reclassified from other comprehensive items		(3,964)		—		(3,964)
Other comprehensive income		108		28,970		29,078
Balance at December 31, 2022	$	(494)	$	5,290	$	4,796
Other comprehensive income (loss) before reclassifications		1,135		(6,901)		(5,766)
Amounts reclassified from other comprehensive items		(315)		—		(315)
Other comprehensive income (loss)		820		(6,901)		(6,081)
Balance at December 31, 2023	$	326	$	(1,611)	$	(1,285)

Revenue recognition - Collaboration and Royalty Revenue (Details) $ in Thousands	1 Months Ended		12 Months Ended
	Sep. 30, 2022 USD ($)	Dec. 31, 2021 USD ($)	Dec. 31, 2023 USD ($)	Dec. 31, 2022 USD ($)	Dec. 31, 2021 USD ($) payment	Aug. 31, 2021 USD ($)	Mar. 31, 2021 USD ($)	Nov. 30, 2011 USD ($)
Revenue recognition
Remaining potential milestones that can be achieved			$ 150,000
Revenue			937,822	$ 698,801	$ 538,593
Collaboration revenue
Revenue recognition
Revenue			100,030	50,052	55,046
Royalty revenue
Revenue recognition
Revenue			168,856	113,521	54,643
SMA License Agreement \| Collaboration revenue
Revenue recognition
Revenue			100,000	50,100	55,000
SMA License Agreement \| Royalty revenue
Revenue recognition
Revenue			168,900	$ 113,500	$ 54,600
Research and Development and Sales Milestones \| Collaboration revenue
Revenue recognition
Number of milestone payments. \| payment					3
Research And Development Event Milestones \| SMA License Agreement
Revenue recognition
Remaining potential milestones that can be achieved			0
Milestone payments received						$ 10,000	$ 20,000
Sales Milestones \| SMA License Agreement
Revenue recognition
Remaining potential milestones that can be achieved			150,000
Net sales milestone threshold achieved	$ 750,000	$ 500,000	1,500,000
Sales Milestones \| SMA License Agreement \| Prepaids and other current assets
Revenue recognition
Milestone payments due	$ 50,000	$ 25,000	$ 100,000
Maximum \| Research And Development Event Milestones \| SMA License Agreement \| Roche
Revenue recognition
Remaining potential milestones that can be achieved								$ 135,000
Maximum \| Sales Milestones \| SMA License Agreement \| Roche
Revenue recognition
Remaining potential milestones that can be achieved								$ 325,000

Other comprehensive income (loss) and accumulated other comprehensive items

12 Months Ended

Dec. 31, 2023

Comprehensive Income (Loss), Net of Tax, Attributable to Parent [Abstract]

Other comprehensive income (loss) and accumulated other comprehensive items

11. Other comprehensive (loss) income and accumulated other comprehensive items

Other comprehensive (loss) income includes changes in equity that are excluded from net loss, such as unrealized gains and losses on marketable securities.


	Unrealized				Total
	Gains (Losses)				Accumulated
	On		Foreign		Other
	Marketable		Currency		Comprehensive
	Securities, net of tax		Translation		Items
Balance at December 31, 2020	$	1,900	$	(62,857)	$	(60,957)
Other comprehensive (loss) income before reclassifications		(3,279)		39,177		35,898
Amounts reclassified from other comprehensive items		777		—		777
Other comprehensive (loss) income		(2,502)		39,177		36,675
Balance at December 31, 2021	$	(602)	$	(23,680)	$	(24,282)
Other comprehensive income before reclassifications		4,072		28,970		33,042
Amounts reclassified from other comprehensive items		(3,964)		—		(3,964)
Other comprehensive income		108		28,970		29,078
Balance at December 31, 2022	$	(494)	$	5,290	$	4,796
Other comprehensive income (loss) before reclassifications		1,135		(6,901)		(5,766)
Amounts reclassified from other comprehensive items		(315)		—		(315)
Other comprehensive income (loss)		820		(6,901)		(6,081)
Balance at December 31, 2023	$	326	$	(1,611)	$	(1,285)

Reclassified amounts from other comprehensive items were determined using the actual realized gains and losses from the sales of marketable securities.

Summary of significant accounting policies (Tables)

12 Months Ended

Dec. 31, 2023

Accounting Policies [Abstract]

Reconciliation of cash


	End of			Beginning of
	period-		period-
	December 31,		December 31,
	2023		2022
Cash and cash equivalents	$	594,001	$	279,834
Restricted cash included in deposits and other assets		16,283		16,091
Total Cash, cash equivalents and restricted cash per statement of cash flows	$	610,284	$	295,925

Schedule of estimated useful life of related assets

Leasehold improvements		Lesser of useful life or lease term
Computer equipment and software		3 years
Machinery and lab equipment		7 years
Furniture and fixtures		7 years

Schedule of Inventory

The following table summarizes the components of the Company’s inventory for the periods indicated:


	December 31, 2023		December 31, 2022
Raw materials	$	952	$	1,078
Work in progress		17,991		14,074
Finished goods		11,634		6,656
Total inventory	$	30,577	$	21,808

Income taxes - Tax Rate Reconciliation (Details)	12 Months Ended
Income taxes - Tax Rate Reconciliation (Details)	Dec. 31, 2023	Dec. 31, 2022	Dec. 31, 2021
Reconciliation of the U.S. statutory income tax rate to the entity's effective tax rate
Federal income tax provision at statutory rate	21.00%	21.00%	21.00%
State income tax provision, net of federal benefit	0.32%	3.07%	(0.74%)
Permanent differences	(1.43%)	(1.83%)	(4.06%)
Research and development	4.59%	5.89%	4.50%
Change in valuation allowances	(16.86%)	(23.36%)	(29.03%)
Change in deferred tax assets		(0.10%)	12.05%
Foreign tax rate differential	0.05%	(0.17%)	0.01%
Tax rate change	(1.26%)	0.34%	0.01%
Release (accrual) of uncertain tax positions	3.71%		(4.78%)
Other	(0.12%)		(0.03%)
Effective income tax rate	10.00%	4.84%	(1.07%)

Debt - Convertible Notes (Details) - 1.50% Convertible senior notes due 2026 - Convertible debt - USD ($) $ in Thousands	Dec. 31, 2023	Dec. 31, 2022	Jan. 01, 2021	Sep. 30, 2019
Long-term debt
Principal	$ 287,500	$ 287,500		$ 287,500
Less: Debt issuance costs	(3,287)	(4,456)
Less: Debt discount, net			$ 0
Net carrying amount	$ 284,213	$ 283,044

- Definition

Line items represent financial concepts included in a table. These concepts are used to disclose reportable information associated with domain members defined in one or many axes to the table.

+ References

+ Details

- Definition

Amount, after deduction of unamortized premium (discount) and debt issuance cost, of long-term debt. Excludes lease obligation.

+ References + Details

401(k) plan

12 Months Ended

Dec. 31, 2023

Retirement Benefits [Abstract]

401(k) plan

16. 401(k) plan

The Company maintains a 401(k) plan for its employees. Employee contributions are voluntary. The Company may match employee contributions in amounts to be determined at the Company’s sole discretion. The Company provided an 100% matching contribution for up to the first 6% of each contributing employee’s base salary contributions for the years ended December 31, 2023, 2022 and 2021, respectively. The Company made matching contributions to the 401(k) plan and recorded expense of approximately $10.9 million, $8.4 million, and $6.6 million for the years ended December 31, 2023, 2022 and 2021, respectively.

Stock award plan - Restricted Stock (Details) - USD ($) $ / shares in Units, $ in Thousands	1 Months Ended	12 Months Ended
	Dec. 31, 2023	Dec. 31, 2023	Dec. 31, 2022	Dec. 31, 2021
Number of Shares
Share-based compensation expense		$ 101,636	$ 110,333	$ 103,513
Restricted stock awards and units
Number of Shares
Balance at the beginning of the period (in shares)		2,516,336
Granted (in shares)		2,104,128
Vested (in shares)		(920,488)
Forfeited (in shares)		(833,706)
Balance at the end of the period (in shares)	2,866,270	2,866,270	2,516,336
Weighted Average Grant Date Fair Value
Balance at the beginning of the period (in dollars per share)		$ 45.67
Granted (in dollars per share)		38.75
Vested (in dollars per share)		43.74
Forfeited (in dollars per share)		42.31
Balance at the end of the period (in dollars per share)	$ 41.82	$ 41.82	$ 45.67
Performance-based restricted stock units (PSUs)
Number of Shares
Granted (in shares)	150,000
Share-based compensation expense	$ 0
Performance period	2 years

Net loss per share

12 Months Ended

Dec. 31, 2023

Earnings Per Share [Abstract]

Net loss per share

9. Net loss per share

Basic and diluted net loss per share is computed by dividing net loss available to common stockholders by the weighted-average number of common shares outstanding. Potentially dilutive securities were excluded from the diluted calculation because their effect would be anti-dilutive.

The following table sets forth the computation of basic and diluted net loss per share for common stockholders:


	Year ended December 31,
	2023			2022			2021
Numerator
Net loss	$	(626,604)		$	(559,017)		$	(523,901)
Denominator
Denominator for basic and diluted net loss per share		74,838,392			71,728,634			70,466,393
Net loss per share:
Basic and diluted	$	(8.37)	*	$	(7.79)	*	$	(7.43)	*

*	For the years ended December 31, 2023, 2022, and 2021, the Company experienced a net loss and therefore did not report any dilutive share impact.

The following table shows historical dilutive common share equivalents outstanding, which are not included in the above historical calculation, as the effect of their inclusion is anti-dilutive during each period.


	As of December 31,
	2023	2022	2021
Stock Options	9,600,399	11,502,417	10,772,582
Unvested restricted stock awards and units	2,866,270	2,516,336	1,519,831
Total	12,466,669	14,018,753	12,292,413

401(k) plan (Details) - USD ($) $ in Millions	12 Months Ended
401(k) plan (Details) - USD ($) $ in Millions	Dec. 31, 2023	Dec. 31, 2022	Dec. 31, 2021
Retirement Benefits [Abstract]
Matching contribution (as a percent of total salary)	100.00%	100.00%	100.00%
Percentage of employee's base salary, matched by employer	6.00%	6.00%	6.00%
Expense recorded	$ 10.9	$ 8.4	$ 6.6

Summary of significant accounting policies - Reconciliation of cash (Details) - USD ($) $ in Thousands	Dec. 31, 2023	Dec. 31, 2022	Dec. 31, 2021	Dec. 31, 2020
Accounting Policies [Abstract]
Cash and cash equivalents	$ 594,001	$ 279,834
Restricted cash included in deposits and other assets	16,283	16,091
Total Cash, cash equivalents and restricted cash per statement of cash flows	$ 610,284	$ 295,925	$ 197,218	$ 216,312

Consolidated Statements of Stockholders' (Deficit) Equity - USD ($) $ in Thousands	Common stock	Additional paid-in capital Cumulative Effect Adjustment	Additional paid-in capital	Accumulated other comprehensive (loss) income	Accumulated deficit Cumulative Effect Adjustment	Accumulated deficit	Cumulative Effect Adjustment	Total
Balance (in shares) at Dec. 31, 2020	69,718,096
Balance at the beginning of the period at Dec. 31, 2020	$ 70	$ (175,236)	$ 2,171,746	$ (60,957)	$ 54,796	$ (1,628,877)	$ (120,440)	$ 481,982
Increase (Decrease) in Stockholders' Equity
Exercise of options (in shares)	635,871
Exercise of options	$ 1		17,308					17,309
Restricted stock vesting and issuance (in shares)	307,658
Issuance of common stock in connection with an employee stock purchase plan (in shares)	166,601
Issuance of common stock in connection with an employee stock purchase plan			5,792					5,792
Share-based compensation expense			103,513					103,513
Receivable from investor			483					483
Other						25		25
Net Income (Loss)						(523,901)		(523,901)
Comprehensive income				36,675				36,675
Balance (in shares) at Dec. 31, 2021	70,828,226
Balance at the end of the period at Dec. 31, 2021	$ 71		2,123,606	(24,282)		(2,097,957)		1,438
Increase (Decrease) in Stockholders' Equity
Issuance of common stock related to stock purchase agreement (in shares)	1,095,290
Issuance of common stock related to stock purchase agreement	$ 1		49,999					50,000
Exercise of options (in shares)	496,863
Exercise of options			14,632					14,632
Restricted stock vesting and issuance (in shares)	490,008
Issuance of common stock in connection with an employee stock purchase plan (in shares)	194,305
Issuance of common stock in connection with an employee stock purchase plan			6,450					6,450
Share-based compensation expense			110,333					110,333
Net Income (Loss)						(559,017)		(559,017)
Comprehensive income				29,078				29,078
Balance (in shares) at Dec. 31, 2022	73,104,692
Balance at the end of the period at Dec. 31, 2022	$ 72		2,305,020	4,796		(2,656,974)		(347,086)
Increase (Decrease) in Stockholders' Equity
Exercise of options (in shares)	822,482
Exercise of options	$ 1		24,039					24,040
Restricted stock vesting and issuance (in shares)	915,203
Restricted stock vesting and issuance, net	$ 1							1
Issuance of common stock in connection with an employee stock purchase plan (in shares)	209,050
Issuance of common stock in connection with an employee stock purchase plan			5,954					5,954
Issuance of common stock in connection with a milestone payable (in shares)	657,462
Issuance of common stock in connection with a milestone payable	$ 1		29,569					29,570
Share-based compensation expense			101,636					101,636
Receivable from investor			15					15
Net Income (Loss)						(626,604)		(626,604)
Comprehensive income				(6,081)				(6,081)
Balance (in shares) at Dec. 31, 2023	75,708,889
Balance at the end of the period at Dec. 31, 2023	$ 75		$ 2,466,233	$ (1,285)		$ (3,283,578)		$ (818,555)

Consolidated Statements of Comprehensive Loss - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2023	Dec. 31, 2022	Dec. 31, 2021
Statement of Comprehensive Income [Abstract]
Net Income (Loss)	$ (626,604)	$ (559,017)	$ (523,901)
Other comprehensive income (loss):
Unrealized gain (loss) on marketable securities, net of tax	820	108	(2,502)
Foreign currency translation (loss) gain, net of tax	(6,901)	28,970	39,177
Comprehensive loss	$ (632,685)	$ (529,939)	$ (487,226)

- Definition

The portion of profit or loss for the period, net of income taxes, which is attributable to the parent.

+ References

+ Details

Fair value of financial instruments and investments - Summary of changes in the fair value of the Company's Level 3 valuation (Details) - USD ($) $ in Thousands	1 Months Ended	12 Months Ended
	Jul. 31, 2022	Dec. 31, 2023	Dec. 31, 2022
Changes in the fair value of warrant liability and SARs liability
Change in fair value - Income Statement location		Business Combination, Contingent Consideration Arrangements, Change in Amount of Contingent Consideration, Asset
PTC-FA and PTC-AS
Changes in the fair value of warrant liability and SARs liability
Change in fair value		$ 128,400
Ending Balance		0
Upstaza
Changes in the fair value of warrant liability and SARs liability
Ending Balance		36,300
Development and Regulatory Milestones \| Significant unobservable inputs (Level 3) \| Agilis
Changes in the fair value of warrant liability and SARs liability
Ending Balance		26,600
Development and Regulatory Milestones \| Significant unobservable inputs (Level 3) \| Agilis \| Commitments
Changes in the fair value of warrant liability and SARs liability
Beginning Balance		82,500	$ 139,300
Change in fair value		(55,900)	(6,800)
Payments	$ (50,000)		(50,000)
Ending Balance		26,600	82,500
Net Sales Milestones and Royalties \| Significant unobservable inputs (Level 3) \| Agilis
Changes in the fair value of warrant liability and SARs liability
Ending Balance		9,700
Net Sales Milestones and Royalties \| Significant unobservable inputs (Level 3) \| Agilis \| Commitments
Changes in the fair value of warrant liability and SARs liability
Beginning Balance		81,500	100,600
Change in fair value		(71,800)	(19,100)
Ending Balance		$ 9,700	$ 81,500

Consolidated Balance Sheets (Parenthetical) - $ / shares	Dec. 31, 2023	Dec. 31, 2022
Statement of Financial Position [Abstract]
Common stock, par value (in dollars per share)	$ 0.001	$ 0.001
Common stock, authorized shares (in shares)	250,000,000	250,000,000
Common stock, issued shares (in shares)	75,708,889	73,104,692
Common stock, outstanding shares (in shares)	75,708,889	73,104,692

Accounts payable and accrued expenses

12 Months Ended

Dec. 31, 2023

Payables and Accruals [Abstract]

Accounts payable and accrued expenses

6. Accounts payable and accrued expenses

Accounts payable and accrued expenses at December 31, 2023 and 2022 consist of the following:


	December 31,
	2023		2022
Employee compensation, benefits, and related accruals	$	62,643	$	62,669
Income tax payable		—		4,712
Consulting and contracted research		27,500		38,882
Professional fees		2,246		3,093
Sales allowance		77,176		63,787
Sales rebates		131,334		67,355
Royalties		74,111		40,546
Accounts payable		6,045		27,268
Other		10,928		12,054
Total	$	391,983	$	320,366

During the year ended December 31, 2023, the Company incurred $32.8 million of restructuring costs from a reduction in workforce in connection with the Company’s strategic pipeline prioritization and discontinuation of its preclinical and early research programs in its gene therapy platform. The costs are included in research and development and selling, general and administrative expenses on the Company’s consolidated statement of operations. As of December 31, 2023, the remaining $9.0 million of accrued restructuring costs are included above within employee compensation, benefits, and related accruals.

Commitments and contingencies

12 Months Ended

Dec. 31, 2023

Commitments and Contingencies Disclosure [Abstract]

Commitments and contingencies

14. Commitments and contingencies

Under various agreements, the Company will be required to pay royalties and milestone payments upon the successful development and commercialization of products. The Company has entered into funding agreements with The Wellcome Trust Limited (“Wellcome Trust”) for the research and development of small molecule compounds in connection with its oncology and antibacterial programs. As the Company has discontinued development under its antibacterial program, it no longer expects that milestone and royalty payments from the Company to Wellcome Trust will apply under that agreement, resulting in a change to the total amount of development and regulatory milestone payments the Company may become obligated to pay for this program. Under the oncology platform funding agreement, to the extent that the Company develops and commercializes certain program intellectual property on a for-profit basis itself or in collaboration with a partner (provided the Company retains overall control of worldwide commercialization), the Company may become obligated to pay to Wellcome Trust development and regulatory milestone payments and single-digit royalties on sales of any research program product. The Company’s obligation to pay such royalties would continue on a country-by-country basis until the longer of the expiration of the last patent in the program intellectual property in such country covering the research program product and the expiration of market exclusivity of such product in such country. The Company made the first development milestone payment of $0.8 million to Wellcome Trust under the oncology platform funding agreement during the second quarter of 2016. During the year ended December 31, 2022, the Company incurred $2.5 million of development milestones in connection with the enrollment of patients in the registration-directed Phase 2/3 trial of unesbulin for the treatment of LMS, which is recorded in accounts payable and accrued expenses on the balance sheet

and will be payable upon the earlier to occur of the first dose administered to the last patient enrolled in the study or the termination of dosing of all patients in the study. However, as part the Company's continuous platform review, the Company has decided to deprioritize its programs for unesbulin for the treatment of diffuse intrinsic pontine glioma and leiomyosarcoma. Accordingly, the Company no longer expects to pay additional milestones to Wellcome Trust under this agreement.

The Company has also entered into a collaboration agreement with the SMA Foundation. The Company may become obligated to pay the SMA Foundation single-digit royalties on worldwide net product sales of any collaboration product that is successfully developed and subsequently commercialized or, with respect to collaboration products the Company outlicenses, including Evrysdi, a specified percentage of certain payments the Company receives from its licensee. The Company is not obligated to make such payments unless and until annual sales of a collaboration product exceed a designated threshold. Since inception, the SMA Foundation has earned $52.5 million, $35.3 million which was paid and $17.2 million which was accrued as of December 31, 2023. The Company has reached its obligations to make such payments to the SMA Foundation of an aggregate of $52.5 million as of December 31, 2023.

Pursuant to the asset purchase agreement ("Asset Purchase Agreement") between the Company and Marathon Pharmaceuticals, LLC (now known as Complete Pharma Holdings, LLC) (“Marathon”), Marathon is entitled to receive contingent payments from the Company based on annual net sales of Emflaza up to a specified aggregate maximum amount over the expected commercial life of the asset. In addition, Marathon received a $50.0 million sales-based milestone during the year ended December 31, 2022.

Pursuant to the Agilis Merger Agreement with Agilis, Agilis equityholders were previously entitled to receive contingent consideration payments from the Company based on (i) the achievement of certain development milestones up to an aggregate maximum amount of $60.0 million, (ii) the achievement of certain regulatory approval milestones together with a milestone payment following the receipt of a priority review voucher up to an aggregate maximum amount of $535.0 million, (iii) the achievement of certain net sales milestones up to an aggregate maximum amount of $150.0 million, and (iv) a percentage of annual net sales for Friedreich ataxia and Angelman syndrome during specified terms, ranging from 2%-6%. The Company was required to pay $40.0 million of the development milestone payments upon the passing of the second anniversary of the closing of the Agilis Merger, regardless of whether the applicable milestones have been achieved.

Pursuant to the terms of the Rights Exchange Agreement, the Participating Rightholders canceled and forfeited their rights under the Agilis Merger Agreement to receive (i) $174.0 million, in the aggregate, of potential milestone payments based on the achievement of certain regulatory milestones and (ii) $37.6 million, in the aggregate, of $40.0 million in development milestone payments that would have been due upon the passing of the second anniversary of the closing of the Agilis Merger, regardless of whether the milestones are achieved.

The Rights Exchange Agreement has no effect on the Agilis Merger Agreement other than to provide for the cancellation and forfeiture of the Participating Rightholders’ rights to receive $211.6 million, in the aggregate, of the milestone payments described above. As a result, all other rights and obligations under the Agilis Merger Agreement remain in effect pursuant to their terms, including the Company’s obligation to pay up to an aggregate maximum amount of $20.0 million upon the achievement of certain development milestones (representing the remaining portion of potential development milestone payments for which rights were not canceled and forfeited pursuant to the Rights Exchange Agreement while excluding the remaining $2.4 million milestone payment that was due and paid upon the passing of the second anniversary of the closing of the Agilis Merger), up to an aggregate maximum amount of $361.0 million upon the achievement of certain regulatory milestones (representing the remaining portion of potential regulatory milestone payments for which rights were not canceled and forfeited pursuant to the Rights Exchange Agreement), up to a maximum

aggregate amount of $150.0 million upon the achievement of certain net sales milestones and a percentage of annual net sales for Friedreich ataxia and Angelman syndrome during specified terms, ranging from 2% to 6%, pursuant to the terms of the Agilis Merger Agreement.

In July 2022, the EC approved Upstaza for the treatment of AADC deficiency for patients 18 months and older within the EEA. As a result of such approval, the Company paid the former equityholders of Agilis $50.0 million in accordance with the terms of the Agilis Merger Agreement in the year ended December 31, 2022. In May 2023, as part of the Company’s strategic portfolio prioritization, the Company decided to discontinue its preclinical and early research programs in its gene therapy platform, which included programs for Friedreich ataxia and Angelman syndrome. As a result, the Company does not expect the milestones related to Friedreich ataxia and Angelman syndrome to be achieved. In addition, the Company does not expect to pay the 2% to 6% royalties on annual net sales related to Friedreich ataxia and Angelman syndrome. As of December 31, 2023, the remaining potential development and regulatory milestones the Company expects to achieve is $31.1 million, and the remaining potential sales milestones the Company expects to achieve is $50.0 million, both of which relate solely to Upstaza.

On October 25, 2019, the Company completed the acquisition of substantially all of the assets of BioElectron Technology Corporation (“BioElectron”), a Delaware corporation, including certain compounds that the Company has begun to develop as part of its Bio-e platform, pursuant to an asset purchase agreement by and between the Company and BioElectron, dated October 1, 2019 (the “BioElectron Asset Purchase Agreement”). BioElectron was a private company with a pipeline focused on inflammatory and central nervous system (CNS) disorders. The lead program, vatiquinone, is in late stage development for CNS disorders with substantial unmet need and significant commercial opportunity that are complementary to PTC’s existing pipeline.

Subject to the terms and conditions of the BioElectron Asset Purchase Agreement, BioElectron may become entitled to receive contingent milestone payments of up to $200.0 million (in cash or in shares of the Company’s common stock, as determined by the Company) from the Company based on the achievement of certain regulatory and net sales milestones. Subject to the terms and conditions of the BioElectron Asset Purchase Agreement, BioElectron may also become entitled to receive contingent payments based on a percentage of net sales of certain products.

Subject to the terms and conditions of the Agreement and Plan of Merger, dated as of May 5, 2020 (the “Censa Merger Agreement”) by and among the Company, Hydro Merger Sub, Inc., the Company’s wholly owned, indirect subsidiary, and, solely in its capacity as the representative, agent and attorney-in-fact of the securityholders of Censa, Shareholder Representative Services LLC (such merger pursuant thereto, the “Censa Merger”), former Censa securityholders may become entitled to receive contingent payments from the Company based on (i) the achievement of certain development and regulatory milestones up to an aggregate maximum amount of $217.5 million for sepiapterin’s two most advanced programs and receipt of a priority review voucher from the FDA as set forth in the Censa Merger Agreement, (ii) $109.0 million in development and regulatory milestones for each additional indication of sepiapterin, (iii) the achievement of certain net sales milestones up to an aggregate maximum amount of $160.0 million, (iv) a percentage of annual net sales during specified terms, ranging from single to low double digits of the applicable net sales threshold amount, and (v) any sublicense fees paid to the Company in consideration of any sublicense of Censa’s intellectual property to commercialize sepiapterin, on a country-by-country basis, which contingent payment shall equal to a mid-double digit percentage of any such sublicense fees.

Censa Merger Agreement. Pursuant to such election, the Company issued 657,462 shares of its common stock and paid $0.4 million to the former Censa securityholders. The Company expects to make payments to the former Censa securityholders of $65.0 million in the aggregate in cash upon the potential achievement in 2024 of regulatory milestones relating to sepiapterin pursuant to the Censa Merger Agreement.

The Company also has the Tegsedi-Waylivra Agreement for the commercialization of Tegsedi and Waylivra, and products containing those compounds in countries in Latin America and the Caribbean. Akcea is entitled to receive royalty payments subject to certain terms set forth in the Tegsedi-Waylivra Agreement.

The Company has employment agreements with certain employees which require the funding of a specific level of payments, if certain events, such as a change in control or termination without cause, occur. Additionally, the Company has royalty payments associated with Translarna, Emflaza, and Upstaza net product revenue, payable quarterly or annually in accordance with the terms of the related agreements.

From time to time in the ordinary course of its business, the Company is subject to claims, legal proceedings and disputes. The Company is not currently aware of any material legal proceedings against it.

Debt - Senior Secured Term Loan (Details) - USD ($) $ in Thousands			12 Months Ended
	Oct. 19, 2023	Oct. 27, 2022	Dec. 31, 2023	Dec. 31, 2022	Dec. 31, 2021
Debt Facility
Repayment of senior secured term loan			$ (300,000)
Prepayment premiums, expenses and other exit fees			81,933
Loss on extinguishment of debt			137,558
Interest expense
Amortization of debt issuance costs			$ 1,873	$ 1,901	$ 1,848
Effective interest rate			10.80%
Blackstone Credit Agreement
Debt Facility
Maximum borrowing capacity		$ 950,000
Minimum consolidated liquidity covenant		100,000
Increased minimum consolidated liquidity covenant after consummation of acquisitions meeting specified thresholds		$ 200,000
Interest expense
Contractual interest expense			$ 30,198	6,069
Amortization of debt issuance costs			702	290
Total			$ 30,900	$ 6,359
Effective interest rate			13.10%	12.20%
Blackstone Credit Agreement \| Adjusted Term SOFR
Debt Facility
Basis spread on variable rate		7.25%
Floor interest rate		1.00%
Blackstone Credit Agreement \| Base Rate
Debt Facility
Basis spread on variable rate		6.25%
Floor interest rate		2.00%
Committed Loan Facility
Debt Facility
Maximum borrowing capacity		$ 450,000
Fund draw term		18 months
Loan term		7 years
Senior Secured Term Loan
Debt Facility
Debt issuance costs capitalized			$ 11,600
Principal		$ 300,000	300,000	$ 300,000
Less: Debt issuance costs				(11,322)
Repayment of senior secured term loan	$ (300,000)		(300,000)
Net carrying amount				$ 288,678
Payment of accrued interest	2,100
Prepayment premiums, expenses and other exit fees	82,000		82,000
Legal fees	$ 200
Loss on extinguishment of debt			92,700
Debt issuance costs			$ 10,700
Committed Delayed Draw Term Loan Facility
Debt Facility
Maximum borrowing capacity		150,000
Uncommitted Incremental Facility
Debt Facility
Maximum borrowing capacity		$ 500,000

Cover - USD ($)	12 Months Ended
Cover - USD ($)	Dec. 31, 2023	Feb. 27, 2024	Jun. 30, 2023
Cover [Abstract]
Document Type	10-K
Document Annual Report	true
Document Period End Date	Dec. 31, 2023
Document Transition Report	false
Entity File Number	001-35969
Entity Registrant Name	PTC THERAPEUTICS, INC.
Entity Incorporation, State or Country Code	DE
Entity Tax Identification Number	04-3416587
Entity Address, Address Line One	100 Corporate Court
Entity Address, City or Town	South Plainfield,
Entity Address, State or Province	NJ
Entity Address, Postal Zip Code	07080
City Area Code	908
Local Phone Number	222-7000
Title of 12(b) Security	Common Stock, $0.001 par value per share
Trading Symbol	PTCT
Security Exchange Name	NASDAQ
Entity Well-known Seasoned Issuer	Yes
Entity Voluntary Filers	No
Entity Current Reporting Status	Yes
Entity Interactive Data Current	Yes
Entity Filer Category	Large Accelerated Filer
Entity Small Business	false
Entity Emerging Growth Company	false
ICFR Auditor Attestation Flag	true
Document Financial Statement Error Correction	false
Entity Shell Company	false
Entity Public Float			$ 2,330,616,515
Entity Common Stock, Shares Outstanding		76,608,030
Documents Incorporated By Reference	DOCUMENTS INCORPORATED BY REFERENCE Part III of this Annual Report incorporates by reference information from the definitive Proxy Statement for the registrant’s 2024 Annual Meeting of Shareholders which is expected to be filed with the Securities and Exchange Commission not later than 120 days after the registrant’s fiscal year ended December 31, 2023.
Entity Central Index Key	0001070081
Current Fiscal Year End Date	--12-31
Document Fiscal Year Focus	2023
Document Fiscal Period Focus	FY
Amendment Flag	false
Auditor Name	Ernst & Young LLP
Auditor Firm ID	42
Auditor Location	Iselin, New Jersey

Accounts payable and accrued expenses - Components (Details) - USD ($) $ in Thousands	Dec. 31, 2023	Dec. 31, 2022
Payables and Accruals [Abstract]
Employee compensation, benefits, and related accruals	$ 62,643	$ 62,669
Income tax payable		4,712
Consulting and contracted research	27,500	38,882
Professional fees	2,246	3,093
Sales allowance	77,176	63,787
Sales rebates	131,334	67,355
Royalties	74,111	40,546
Accounts payable	6,045	27,268
Other	10,928	12,054
Accounts payable and accrued expenses	$ 391,983	$ 320,366

Debt - Convertible Notes Interest Expense (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2023	Dec. 31, 2022	Dec. 31, 2021
Long-term debt
Amortization of debt issuance costs	$ 1,873	$ 1,901	$ 1,848
Effective interest rate of the liability component	10.80%
Convertible debt \| 3.00% Convertible senior notes due 2022
Long-term debt
Contractual interest expense		2,800
Amortization of debt issuance costs		460
Total		$ 3,260
Effective interest rate of the liability component		3.50%
Convertible debt \| 1.50% Convertible senior notes due 2026
Long-term debt
Contractual interest expense	$ 4,305	$ 4,313
Amortization of debt issuance costs	1,171	1,150
Total	$ 5,476	$ 5,463
Effective interest rate of the liability component	1.90%	1.90%

- Definition

Line items represent financial concepts included in a table. These concepts are used to disclose reportable information associated with domain members defined in one or many axes to the table.

+ References

+ Details

Summary of significant accounting policies - Inventory (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2023	Dec. 31, 2022
Accounting Policies [Abstract]
Raw materials	$ 952	$ 1,078
Work in progress	17,991	14,074
Finished goods	11,634	6,656
Total inventory	30,577	21,808
Inventory write-down	$ 12,500	$ 1,700

Revenue recognition - Net Product Sales (Details) $ in Thousands	12 Months Ended
	Dec. 31, 2023 USD ($) country Distributor	Dec. 31, 2022 USD ($) country Distributor	Dec. 31, 2021 USD ($) country Distributor
Revenue recognition
Revenue	$ 937,822	$ 698,801	$ 538,593
United States
Revenue recognition
Revenue	531,661	381,862
Non-US
Revenue recognition
Revenue	406,161	316,939
Net product revenue
Revenue recognition
Contract with Customer, Liability	$ 0	$ 0
Net product revenue \| Geographic concentration risk
Revenue recognition
Number of countries exceeding 10% of net product sales \| country	2	2	1
Percentage of net product sales threshold	10.00%	10.00%	10.00%
Net product revenue \| Customer concentration risk
Revenue recognition
Number of distributors \| Distributor	2	2	2
Percentage of net product sales threshold	10.00%	10.00%	10.00%
Net product revenue
Revenue recognition
Revenue	$ 661,249	$ 535,228	$ 428,904
Net product revenue \| Non-US
Revenue recognition
Revenue	406,100	316,900	241,600
Net product revenue \| Net product revenue \| Geographic concentration risk \| United States
Revenue recognition
Revenue	255,100	218,300
Net product revenue \| Net product revenue \| Geographic concentration risk \| Russia
Revenue recognition
Revenue	86,000	59,700
Emflaza \| United States
Revenue recognition
Revenue	255,100	218,300	187,300
Translarna \| Non-US
Revenue recognition
Revenue	$ 355,800	$ 288,600	$ 236,000

Income taxes - Unrecognized Tax Benefits (Details) $ in Thousands	12 Months Ended
	Dec. 31, 2023 USD ($)
Uncertain income tax benefits
Beginning Balance	$ 27,217
Reductions based on settlements or expiration of statute of limitations	(24,671)
Interest received	(1,186)
Ending Balance	$ 1,360

Leases - (Tables)

12 Months Ended

Dec. 31, 2023

Leases

Schedule of lease costs

The components of lease expense were as follows:


	Year Ended December 31, 2023		Year Ended December 31, 2022		Year Ended December 31, 2021

Operating Lease Cost
Fixed lease cost	$	21,952	$	19,804	$	16,411
Variable lease cost		5,846		4,557		4,361
Short-term lease cost		1,186		808		656
Total operating lease cost	$	28,984	$	25,169	$	21,428

Supplemental lease term and discount rate information related to leases was as follows:


	December 31, 2023		December 31, 2022
Weighted-average remaining lease terms - operating leases (years)	11.55		11.61
Weighted-average discount rate - operating leases	8.69	%	8.61	%
Weighted-average remaining lease terms - finance lease (years)	9.01		10.01
Weighted-average discount rate - finance lease	7.80	%	7.80	%

Schedule of supplemental cash flow information related to leases

Supplemental cash flow information related to leases was as follows:


	Year Ended December 31,
	2023		2022		2021
Cash paid for amounts included in the measurement of lease liabilities:
Operating cash flows from operating leases	$	15,338	$	14,736	$	13,683
Financing cash flows from finance lease		1,379		1,276		2,224
Operating cash flows from finance lease		1,621		1,724		776

Right-of-use assets obtained in exchange for lease obligations:
Operating leases	$	—	$	35,817	$	645

Schedule of future lease payments - Operating leases

Future minimum lease payments under non-cancelable leases as of December 31, 2023 were as follows:


	Operating Leases		Finance Lease
2024	$	18,352	$	3,000
2025		20,450		3,000
2026		19,992		3,000
2027		17,945		3,000
2028 and thereafter		175,847		15,000
Total lease payments		252,586		27,000
Less: Imputed Interest expense		141,957		6,816
Total	$	110,629	$	20,184

The Company (Details) - USD ($) $ in Thousands	Dec. 31, 2023	Dec. 31, 2022
Long-term debt
Accumulated deficit	$ (3,283,578)	$ (2,656,974)

- Definition

Line items represent financial concepts included in a table. These concepts are used to disclose reportable information associated with domain members defined in one or many axes to the table.

+ References

+ Details

- Definition

Amount of accumulated undistributed earnings (deficit).

+ References + Details

Summary of significant accounting policies - Manufacturing Revenue (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2023	Dec. 31, 2022	Dec. 31, 2021
Revenue recognition
Revenue	$ 937,822	$ 698,801	$ 538,593
Remaining performance obligations		1,400
Manufacturing revenue
Revenue recognition
Revenue	7,687	0	$ 0
Contract with customer, asset	200	0
Remaining performance obligations	$ 800	$ 1,400

- Definition

Line items represent financial concepts included in a table. These concepts are used to disclose reportable information associated with domain members defined in one or many axes to the table.

+ References + Details

- Definition

+ References

+ Details

Stock award plan - (Tables)

12 Months Ended

Dec. 31, 2023

Share-based Payment Arrangement [Abstract]

Summary of stock option activity

Stock option activity—A summary of stock option activity is as follows:


				Weighted-
		Weighted-		average		Aggregate
		average		remaining		intrinsic
	Number of	exercise		contractual		value(in
	options	price		term		thousands)

Outstanding at December 31, 2020	9,663,677	$	38.72
Granted	2,487,234	$	61.36
Exercised	(635,871)	$	28.01
Forfeited	(742,458)	$	52.04
Outstanding at December 31, 2021	10,772,582	$	43.66
Granted	1,685,435	$	38.55
Exercised	(496,863)	$	29.45
Forfeited	(458,737)	$	48.75
Outstanding at December 31, 2022	11,502,417	$	43.33
Granted	1,117,284	$	40.19
Exercised	(822,482)	$	29.25
Forfeited/Cancelled	(2,196,820)	$	45.85
Outstanding at December 31, 2023	9,600,399	$	43.59	5.39	years	$	8,814
Vested or Expected to vest at December 31, 2023	1,796,687	$	45.85	8.16	years	$	366
Exercisable at December 31, 2023	7,650,948	$	43.08	4.68	years	$	8,387

Schedule of assumptions used to estimate fair values of grants made on the date of grant

The fair values of grants made in the years ended December 31, 2023, 2022 and 2021 were contemporaneously estimated on the date of grant using the following assumptions:



	2023	2022	2021
Risk-free interest rate	3.54% - 4.69%	1.55% - 4.57%	0.51% - 1.24%
Expected volatility	53% - 56%	54% - 74%	74% - 89%
Expected term	5.5 years	5.5 years	5.5 years

Summary of information on the Company's restricted stock

The following table summarizes information on the Company’s restricted stock awards and units:


	Restricted Stock Awards and Units
		Weighted
		Average
		Grant
	Number of	Date
	Shares	Fair Value
Unvested at December 31, 2022	2,516,336	$	45.67
Granted	2,104,128		38.75
Vested	(920,488)		43.74
Forfeited	(833,706)		42.31
Unvested at December 31, 2023	2,866,270	$	41.82

Schedule of share-based compensation expense recorded in the statement of operations

The Company recorded share-based compensation expense in the statement of operations related to incentive stock options, nonstatutory stock options, restricted stock awards, restricted stock units and the ESPP as follows:


	Year ended December 31,
	2023		2022		2021
Research and development	$	52,941	$	55,869	$	53,632
Selling, general and administrative		48,695		54,464		49,881
Total	$	101,636	$	110,333	$	103,513

Fair value of financial instruments and investments - Marketable Securities (Details) $ / shares in Units, $ in Thousands	1 Months Ended	12 Months Ended
	Jan. 31, 2020 USD ($) $ / shares	Dec. 31, 2023 USD ($) fund	Dec. 31, 2022 USD ($)	Dec. 31, 2021 USD ($)
Changes in fair value
Unrealized Gain/(Loss)		$ 2,517	$ (7,992)	$ 1,673
Realized Gain/(Loss)		4,383
Investments Purchased		38,398	22,787	210,018
Redemptions/Sale		(132,228)	(112,958)	(4,281)
Unrealized Gain/(Loss)		(1,515)	(3,560)	(6,078)
Realized Gain/(Loss)		(782)
Redemptions/Sale		(2,594)
Unrealized Gain/(Loss)		(2,678)	(5,740)	(8,281)
Beginning Balance - Total		134,457	242,469
Unrealized Gain/(Loss) - Total		(1,676)	(17,292)
Realized Gain/(Loss) - Total		3,601
Foreign Currency Unrealized Gain/(Loss) - Total		1,384	(549)
Investments Purchased - Total		38,432	22,787
Redemptions/Sale - Total		(134,937)	(112,958)
Ending Balance - Total		$ 41,261	134,457	242,469
Equity Investments
Number of mutual funds denominated in a foreign currency \| fund		1
ClearPoint Convertible note
Purchase of convertible note		$ 174,086	52,764	333,148
Marketable securities - equity investments
Changes in fair value
Beginning Balance		108,261	206,973
Unrealized Gain/(Loss)		2,517	(7,992)
Realized Gain/(Loss)		4,383
Foreign Currency Unrealized Gain/(Loss)		1,384	(549)
Investments Purchased		38,432	22,787
Redemptions/Sale		(132,343)	(112,958)
Ending Balance		22,634	108,261	206,973
ClearPoint Equity Investment
Changes in fair value
Beginning Balance		10,965	14,525
Unrealized Gain/(Loss)		(1,515)	(3,560)
Realized Gain/(Loss)		(782)
Redemptions/Sale		(2,594)
Ending Balance		6,074	10,965	14,525
ClearPoint convertible debt
Changes in fair value
Beginning Balance		15,231	20,971
Unrealized Gain/(Loss)		(2,678)	(5,740)
Ending Balance		$ 12,553	$ 15,231	$ 20,971
ClearPoint Convertible note
Purchase of convertible note	$ 10,000
Conversion price - convertible note \| $ / shares	$ 6.00
Term of convertible note	5 years

Leases - Supplemental lease terms (Details)	Dec. 31, 2023	Dec. 31, 2022
Leases
Weighted-average remaining lease terms - operating leases (years)	11 years 6 months 18 days	11 years 7 months 9 days
Weighted-average discount rate - operating leases	8.69%	8.61%
Weighted-average remaining lease terms - finance lease (years)	9 years 3 days	10 years 3 days
Weighted-average discount rate - finance lease	7.80%	7.80%

Stock award plan

12 Months Ended

Dec. 31, 2023

Share-based Payment Arrangement [Abstract]

Stock award plan

10. Stock award plan

In May 2013, the Company’s Board of Directors and stockholders approved the 2013 Long Term Incentive Plan, which became effective upon the closing of the Company’s IPO. The 2013 Long Term Incentive Plan provides for the grant of incentive stock options, nonstatutory stock options, restricted stock awards and other stock-based awards. On June 8, 2022 (the “Restatement Effective Date”), the Company’s stockholders approved the Amended and Restated 2013 Long-Term Incentive Plan (the “Amended 2013 LTIP”). The Amended 2013 LTIP provides for the grant of incentive stock options, nonstatutory stock options, restricted stock units and other stock-based awards. The number of shares of common stock reserved for issuance under the Amended 2013 LTIP is the sum of (A) the number of shares of the Company’s common stock (up to 16,724,212 shares) that is equal to the sum of (1) the number of shares issued under the 2013 Long-Term Incentive Plan prior to the Restatement Effective Date, (2) the number of shares that remain available for issuance under the 2013 Long-Term Incentive Plan immediately prior to the Restatement Effective Date and (3) the number of shares subject to awards granted under the 2013 Long-Term Incentive Plan prior to the Restatement Effective Date that are outstanding as of the Restatement Effective Date, plus (B) from and after the Restatement Effective Date, an additional 8,475,000 shares of Common Stock. As of December 31, 2023, awards for 8,680,209 shares of common stock were available for issuance under the Amended 2013 LTIP.

There are no additional shares of common stock available for issuance under the Company’s 1998 Employee, Director and Consultant Stock Option Plan, 2009 Equity and Long Term Incentive Plan or 2013 Stock Incentive Plan.

In January 2020, the Company’s Board of Directors approved the 2020 Inducement Stock Incentive Plan. The 2020 Inducement Stock Incentive Plan provides for the grant of incentive stock options, nonstatutory stock options, restricted stock awards and other stock-based awards, initially up to an aggregate of 1,000,000 shares of common stock. Any grants made under the 2020 Inducement Stock Incentive Plan must be made pursuant to the Nasdaq Listing Rule 5635(c)(4)

inducement grant exception as a material component of the Company’s new hires’ employment compensation. In December 2020, the Company’s Board of Directors approved an additional 1,000,000 shares of common stock that may be issued under the 2020 Inducement Stock Incentive Plan. In April 2022, the Company’s Board of Directors approved a reduction in the total number of shares of common stock that may be issued under the 2020 Inducement Stock Incentive Plan to 1,300,000 shares. In December 2022, the Company’s Board of Directors approved an additional 1,700,000 shares of common stock that may be issued under the 2020 Inducement Stock Inventive Plan. As of December 31, 2023, awards for 1,834,322 shares of common stock are available for issuance under the 2020 Inducement Stock Incentive Plan.

The Board of Directors has the authority to select the individuals to whom options are granted and determine the terms of each option, including (i) the number of shares of common stock subject to the option; (ii) the date on which the option becomes exercisable; (iii) the option exercise price, which, in the case of incentive stock options, must be at least 100% (110% in the case of incentive stock options granted to a stockholder owning in excess of 10% of the Company’s stock) of the fair market value of the common stock as of the date of grant; and (iv) the duration of the option (which, in the case of incentive stock options, may not exceed ten years). Options typically vest over a four-year period.

Inducement stock option awards

Pursuant to the Nasdaq inducement grant exception, during the year ended December 31, 2023, the Company issued options to purchase an aggregate of 139,040 shares of common stock to certain new hire employees at a weighted-average exercise price of $41.25 per share under the 2020 Inducement Stock Incentive Plan. Additionally, during the year ended December 31, 2023, the Company issued 105,370 restricted stock units under the 2020 Inducement Stock Incentive Plan. An aggregate of 270,583 of options and 79,195 of restricted stock units previously granted as inducement awards were forfeited during the year ended December 31, 2023 in connection with employee separations from the Company.

Stock option activity—A summary of stock option activity is as follows:


				Weighted-
		Weighted-		average		Aggregate
		average		remaining		intrinsic
	Number of	exercise		contractual		value(in
	options	price		term		thousands)

Outstanding at December 31, 2020	9,663,677	$	38.72
Granted	2,487,234	$	61.36
Exercised	(635,871)	$	28.01
Forfeited	(742,458)	$	52.04
Outstanding at December 31, 2021	10,772,582	$	43.66
Granted	1,685,435	$	38.55
Exercised	(496,863)	$	29.45
Forfeited	(458,737)	$	48.75
Outstanding at December 31, 2022	11,502,417	$	43.33
Granted	1,117,284	$	40.19
Exercised	(822,482)	$	29.25
Forfeited/Cancelled	(2,196,820)	$	45.85
Outstanding at December 31, 2023	9,600,399	$	43.59	5.39	years	$	8,814
Vested or Expected to vest at December 31, 2023	1,796,687	$	45.85	8.16	years	$	366
Exercisable at December 31, 2023	7,650,948	$	43.08	4.68	years	$	8,387

The fair values of grants made in the years ended December 31, 2023, 2022 and 2021 were contemporaneously estimated on the date of grant using the following assumptions:



	2023	2022	2021
Risk-free interest rate	3.54% - 4.69%	1.55% - 4.57%	0.51% - 1.24%
Expected volatility	53% - 56%	54% - 74%	74% - 89%
Expected term	5.5 years	5.5 years	5.5 years

The Company assumed no expected dividends for all grants. The weighted average grant date fair value of options granted during the years ended December 31, 2023, 2022 and 2021 was $21.27, $23.54, and $43.05 per share, respectively.

Restricted Stock Awards and Restricted Stock Units—Restricted stock awards and restricted stock units are granted subject to certain restrictions, including in some cases service conditions (restricted stock). The grant-date fair value of restricted stock awards, which has been determined based upon the market value of the Company’s shares on the grant date, is expensed over the vesting period.

The following table summarizes information on the Company’s restricted stock awards and units:


	Restricted Stock Awards and Units
		Weighted
		Average
		Grant
	Number of	Date
	Shares	Fair Value
Unvested at December 31, 2022	2,516,336	$	45.67
Granted	2,104,128		38.75
Vested	(920,488)		43.74
Forfeited	(833,706)		42.31
Unvested at December 31, 2023	2,866,270	$	41.82

Performance-based Restricted Stock Units—In December 2023, the Company granted 150,000 performance-based restricted stock units (“PSUs”) to its Chief Executive Officer, Dr. Matthew Klein, which will vest only if challenging performance goals relating to development and regulatory milestones are achieved over an approximately two year performance period. As of December 31, 2023, the achievements of the performance goals have not yet been deemed probable and therefore no expense has been recognized to date.

Employee Stock Purchase Plan—In June 2016, the Company established an Employee Stock Purchase Plan (as amended, “ESPP” or the “Plan”) for certain eligible employees. The Plan is administered by the Company’s Board of Directors or a committee appointed by the Board. In June 2021, the Plan was amended to increase the total number of shares available for purchase under the Plan from one million shares to two million shares of the Company’s common stock. Employees may participate over a six-month period through payroll withholdings and may purchase, at the end of the six-month period, the Company’s common stock at a purchase price of at least 85% of the closing price of a share of the Company’s common stock on the first business day of the offering period or the closing price of a share of the Company’s common stock on the last business day of the offering period, whichever is lower. No participant will be granted a right to purchase the Company’s common stock under the Plan if such participant would own more than 5% of the total combined voting power of the Company or any subsidiary of the Company after such purchase. For the period ending December 31, 2023, the Company recorded $2.4 million in compensation expense related to the ESPP.


	Year ended December 31,
	2023		2022		2021
Research and development	$	52,941	$	55,869	$	53,632
Selling, general and administrative		48,695		54,464		49,881
Total	$	101,636	$	110,333	$	103,513

As of December 31, 2023, there was approximately $119.6 million of total unrecognized compensation cost related to unvested share-based compensation arrangements granted under the Company’s Plans. This cost is expected to be recognized as compensation expense over the weighted average remaining service period of approximately 2.1 years.

Accounts payable and accrued expenses - (Tables)

12 Months Ended

Dec. 31, 2023

Payables and Accruals [Abstract]

Schedule of components of accounts payable and accrued expenses

Accounts payable and accrued expenses at December 31, 2023 and 2022 consist of the following:


	December 31,
	2023		2022
Employee compensation, benefits, and related accruals	$	62,643	$	62,669
Income tax payable		—		4,712
Consulting and contracted research		27,500		38,882
Professional fees		2,246		3,093
Sales allowance		77,176		63,787
Sales rebates		131,334		67,355
Royalties		74,111		40,546
Accounts payable		6,045		27,268
Other		10,928		12,054
Total	$	391,983	$	320,366

Fixed assets (Details) - USD ($) $ in Thousands	12 Months Ended
Fixed assets (Details) - USD ($) $ in Thousands	Dec. 31, 2023	Dec. 31, 2022	Dec. 31, 2021
Fixed assets
Gross fixed assets	$ 138,363	$ 109,958
Less accumulated depreciation and amortization	(51,274)	(37,368)
Property, Plant and Equipment, Net, Total	87,089	72,590
Depreciation and amortization	13,900	12,300	$ 9,400
Leasehold improvements
Fixed assets
Gross fixed assets	30,166	28,969
Computer equipment and software
Fixed assets
Gross fixed assets	17,503	15,332
Machinery and lab equipment
Fixed assets
Gross fixed assets	62,837	47,496
Furniture and fixtures
Fixed assets
Gross fixed assets	3,849	3,812
Assets in process
Fixed assets
Gross fixed assets	$ 24,008	$ 14,349

Revenue recognition - Manufacturing Revenue (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2023	Dec. 31, 2022	Dec. 31, 2021
Revenue recognition
Revenue	$ 937,822	$ 698,801	$ 538,593
Manufacturing revenue
Revenue recognition
Revenue	7,687	0	$ 0
Contract with customer, asset	200	0
Contract liability revenues recognized in period	1,400
Manufacturing revenue \| Deferred revenue
Revenue recognition
Contract with customer, liability	800	1,400
Manufacturing revenue \| Prepaids and other current assets
Revenue recognition
Contract with customer, asset	$ 200	$ 0

- Definition

Line items represent financial concepts included in a table. These concepts are used to disclose reportable information associated with domain members defined in one or many axes to the table.

+ References + Details

- Definition

+ References

+ Details

Debt

12 Months Ended

Dec. 31, 2023

Debt Disclosure [Abstract]

Debt

7. Debt

Liability for sale of future royalties

The following table shows the activity within the “liability for sale of future royalties- current” and “liability for sale of future royalties- noncurrent” accounts for the year ended December 31, 2023:


	Year Ended December 31,
Liability for sale of future royalties- (current and noncurrent)	2023
Beginning balance as of December 31, 2022	$	757,886
Less: Non-cash royalty revenue payable to RPI		(93,460)
Plus: Non-cash interest expense recognized		104,790
Plus: Cash Consideration		1,000,000
Plus: Loss on Debt Extinguishment		44,881
Ending balance	$	1,814,097
Effective interest rate as of December 31, 2023		10.8%

Non-cash interest expense is recorded in the statement of operations within “Interest expense, net”.

Senior Secured Term Loan

On October 27, 2022 (the “Closing Date”), the Company entered into a credit agreement (the “Blackstone Credit Agreement”) for fundings of up to $950.0 million consisting of a committed loan facility of $450.0 million and further contemplating the potential for up to $500.0 million of additional financing, to the extent that the Company requested such additional financing and subject to the Lenders’ agreement to provide such additional financing and to mutual agreement on terms, among the Company, certain subsidiaries of the Company (together with the Company, the “Loan Parties”) and funds and other affiliated entities advised or managed by Blackstone Life Sciences and Blackstone Credit (collectively, “Blackstone”, and such lenders, together with their permitted assignees, the “Lenders” and each a “Lender”) and Wilmington Trust, National Association, as the administrative agent for the Lenders.

The Blackstone Credit Agreement provided for a senior secured term loan facility funded on the Closing Date in the aggregate principal amount of $300.0 million (the “Initial Loans”) and a committed delayed draw term loan facility of up to $150.0 million (the “Delayed Draw Loans” and, together with the Initial Loans, the “Loans”) to be funded at the Company’s request within 18 months of the Closing Date subject to specified conditions. In addition, the Blackstone Credit Agreement contemplated the potential for further financings by Blackstone, by providing for incremental discretionary uncommitted further financings of up to $500.0 million. The Company capitalized approximately $11.6 million of debt issuance costs which are presented on the balance sheet as a direct deduction from the debt liability and are being amortized over the term of the senior secured term loan facility using the effective interest rate method.

The Loans were to mature on the date that is seven years from the Closing Date. Borrowings under the Blackstone Credit Agreement bore interest at a variable rate equal to, at the Company’s option, either an adjusted Term SOFR rate plus seven and a quarter percent (7.25%) or the Base Rate plus six and a quarter percent (6.25%), subject to a floor of one percent (1%) and two percent (2%) with respect to Term SOFR rate and Base Rate (each as defined in the Blackstone Credit Agreement), respectively. Payment of the Loans were subject to certain premiums specified in the Blackstone Credit Agreement, in each case, from the date of the applicable Loan funded.

All obligations under the Blackstone Credit Agreement were secured, subject to certain exceptions and specified inclusions, by security interests in certain assets of the Loan Parties, including (1) intellectual property and other assets related to Translarna, Emflaza, Upstaza, sepiapterin and, until certain release conditions were met, vatiquinone, in each case, together with any other forms, formulations, or methods of delivery of any such products, and regardless of trade or brand name, (2) future acquired intellectual property (but not internally developed intellectual property unrelated to other intellectual property collateral) and other related assets, and (3) the equity interests held by the Loan Parties in certain of their subsidiaries. The Blackstone Credit Agreement contained certain negative covenants with which the Company was to remain in compliance. The Blackstone Credit Agreement also required that the Company maintained consolidated liquidity of at least $100.0 million as of the last day of each fiscal quarter, which was to be increased to $200.0 million upon the Company consummating acquisitions meeting certain consolidated thresholds described therein. In addition, the Company was required under conditions specified in the Blackstone Credit Agreement to fund a reserve account up to certain amounts specified therein. The funds in the reserve account were available to prepay the Loans at any time at the Company’s option, and were, if funded, subject to release upon certain further conditions. Upon any such release, such funds were freely available for use by the Company subject to the generally applicable terms and conditions of the Blackstone Credit Agreement. The Blackstone Credit Agreement contained certain customary representations and warranties, affirmative covenants and provisions relating to events of default.

On October 19, 2023, the Company terminated the Blackstone Credit Agreement. In connection with the termination of the Credit Agreement, the Company repaid outstanding principal of $300.0 million, accrued interest of $2.1 million, an additional $82.0 million in prepayment premiums, exit fees, and creditor expenses, and $0.2 million in legal fees. The Company recorded a loss on the extinguishment of debt of $92.7 million which is included on the statement of operations for the period ended December 31, 2023. The loss on extinguishment of debt consisted of $82.0 million in prepayment

premiums, exit fees, and creditor expenses and debt issuance costs of $10.7 million. All liens and security interests securing the loans made pursuant to the Blackstone Credit Agreement were released upon termination.

The Blackstone Credit Agreement consisted of the following:


		Year ended December 31,
	2023			2022
Principal	$	300,000	$	300,000
Less: Debt issuance costs		—		(11,322)
Repayment of senior secured term loan		(300,000)		—
Net carrying amount	$	—	$	288,678

The following table sets forth total interest expense recognized related to the Blackstone Credit Agreement:


	Year Ended			Year Ended
	December 31,			December 31,
	2023			2022
Contractual interest expense	$	30,198		$	6,069
Amortization of debt issuance costs		702			290
Total	$	30,900		$	6,359
Effective interest rate		13.1	%		12.2	%

2026 Convertible Notes

In September 2019, the Company issued, at par value, $287.5 million aggregate principal amount of 1.50% convertible senior notes due 2026, which included an option to purchase up to an additional $37.5 million in aggregate principal amount of the 2026 Convertible Notes, which was exercised in full by the initial purchasers. The 2026 Convertible Notes bear cash interest at a rate of 1.50% per year, payable semi-annually on March 15 and September 15 of each year, beginning on March 15, 2020. The 2026 Convertible Notes will mature on September 15, 2026, unless earlier repurchased or converted. The net proceeds to the Company from the offering were $279.3 million after deducting the initial purchasers’ discounts and commissions and the offering expenses payable by the Company.

The 2026 Convertible Notes are governed by an indenture (the “2026 Convertible Notes Indenture”) with U.S Bank National Association as trustee (the “2026 Convertible Notes Trustee”).

Holders of the 2026 Convertible Notes may convert their 2026 Convertible Notes at their option at any time prior to the close of business on the business day immediately preceding March 15, 2026 only under the following circumstances:

●

during any calendar quarter commencing on or after December 31, 2019 (and only during such calendar quarter), if the last reported sale price of the Company’s common stock for at least 20 trading days (whether or not consecutive) during a period of 30 consecutive trading days ending on the last trading day of the immediately preceding calendar quarter is greater than or equal to 130% of the conversion price on each applicable trading day;

●

during the five business day period after any five consecutive trading day period (the “measurement period”) in which the trading price (as defined in the 2026 Convertible Notes Indenture) per $1,000 principal amount of 2026 Convertible Notes for each trading day of the measurement period was less than 98% of the product of the last reported sale price of the Company’s common stock and the conversion rate on each such trading day;

●

during any period after the Company has issued notice of redemption until the close of business on the scheduled trading day immediately preceding the relevant redemption date; or

●

upon the occurrence of specified corporate events.

On or after March 15, 2026, until the close of business on the business day immediately preceding the maturity date, holders may convert their 2026 Convertible Notes at any time, regardless of the foregoing circumstances. Upon conversion, the Company will pay or deliver, as the case may be, cash, shares of the Company’s common stock or any combination thereof at the Company’s election.

The conversion rate for the 2026 Convertible Notes was initially, and remains, 19.0404 shares of the Company’s common stock per $1,000 principal amount of the 2026 Convertible Notes, which is equivalent to an initial conversion price of approximately $52.52 per share of the Company’s common stock. The conversion rate may be subject to adjustment in some events but will not be adjusted for any accrued and unpaid interest.

The Company was not permitted to redeem the 2026 Convertible Notes prior to September 20, 2023. The Company may redeem for cash all or any portion of the 2026 Convertible Notes, at its option, if the last reported sale price of its common stock has been at least 130% of the conversion price then in effect on the last trading day of, and for at least 19 other trading days (whether or not consecutive) during, any 30 consecutive trading day period ending on, and including, the trading day immediately preceding the date on which the Company provides notice of redemption, at a redemption price equal to 100% of the principal amount of the 2026 Convertible Notes to be redeemed, plus accrued and unpaid interest to, but excluding, the redemption date. No sinking fund is provided for the 2026 Convertible Notes, which means that the Company is not required to redeem or retire the 2026 Convertible Notes periodically.

If the Company undergoes a “fundamental change” (as defined in the 2026 Convertible Notes Indenture), subject to certain conditions, holders of the 2026 Convertible Notes may require the Company to repurchase for cash all or part of their 2026 Convertible Notes at a repurchase price equal to 100% of the principal amount of the 2026 Convertible Notes to be repurchased, plus accrued and unpaid interest to, but excluding, the fundamental change repurchase date.

The 2026 Convertible Notes represent senior unsecured obligations and will rank senior in right of payment to the Company’s future indebtedness that is expressly subordinated in right of payment to the notes, equal in right of payment to the Company’s existing and future unsecured indebtedness that is not so subordinated, effectively junior in right of payment to any of the Company’s secured indebtedness to the extent of the value of the assets securing such indebtedness, and structurally subordinated to all existing and future indebtedness and other liabilities (including trade payables) incurred by the Company’s subsidiaries. The 2026 Convertible Notes Indenture contains customary events of default with respect to the 2026 Convertible Notes, including that upon certain events of default (including the Company’s failure to make any payment of principal or interest on the 2026 Convertible Notes when due and payable) occurring and continuing, the 2026 Convertible Notes Trustee by notice to the Company, or the holders of at least 25% in principal amount of the outstanding 2026 Convertible Notes by notice to the Company and the Convertible Notes Trustee, may, and the 2026 Convertible Notes Trustee at the request of such holders (subject to the provisions of the 2026 Convertible Notes Indenture) will, declare 100% of the principal of and accrued and unpaid interest, if any, on all the 2026 Convertible Notes to be due and

payable. In case of certain events of bankruptcy, insolvency or reorganization, involving the Company or a significant subsidiary, 100% of the principal of and accrued and unpaid interest on the 2026 Convertible Notes will automatically become due and payable. Upon such a declaration of acceleration, such principal and accrued and unpaid interest, if any, will be due and payable immediately.

Prior to the adoption of ASU 2020-06, the Company accounted for the 2026 Convertible Notes as a liability and equity component where the carrying value of the liability component was valued based on a similar instrument. In accounting for the issuance of the 2026 Convertible Notes, the Company separated the 2026 Convertible Notes into liability and equity components. The carrying amount of the liability component was calculated by measuring the fair value of a similar liability that did not have an associated convertible feature. The carrying amount of the equity component representing the conversion option was determined by deducting the fair value of the liability component from the par value of the 2026 Convertible Notes as a whole. The excess of the principal amount of the liability component over its carrying amount, referred to as the debt discount, was amortized to interest expense over the seven-year term of the 2026 Convertible Notes. The equity component was not re-measured as long as it continued to meet the conditions for equity classification. The equity component recorded at issuance related to the 2026 Convertible Notes was $123.0 million and was recorded in additional paid-in capital.

In accounting for the transaction costs related to the issuance of the 2026 Convertible Notes, the Company allocated the total costs incurred to the liability and equity components of the 2026 Convertible Notes based on their relative values. Transaction costs attributable to the liability component were amortized to interest expense over the seven-year term of the 2026 Convertible Notes, and transaction costs attributable to the equity component were netted with the equity components in stockholders’ equity. Additionally, the Company initially recorded a net deferred tax liability of $25.3 million in connection with the 2026 Convertible Notes.

Effective January 1, 2021 the Company adopted ASU 2020-06. After adoption, the Company now accounts for the 2026 Convertible Notes as a single liability measured at amortized cost. As the equity component is no longer required to be split into a separate component, the Company recorded an adjustment for the initial $123.0 million that was allocated to additional paid in capital and $16.1 million of life to date interest expense recorded as amortization of debt discount. Additionally, the net deferred tax liability recorded for the 2026 Convertible Notes was reversed. The principal amount of the liability over its carrying amount is amortized to interest expense over the seven-year term of the 2026 Convertible Notes. Since the 2026 Convertible Notes are classified as a single liability, there is no debt discount required to be amortized.

The 2026 Convertible Notes consist of the following:


		Year ended December 31,
	2023			2022
Principal	$	287,500	$	287,500
Less: Debt issuance costs		(3,287)		(4,456)
Net carrying amount	$	284,213	$	283,044

As of December 31, 2023, the remaining contractual life of the 2026 Convertible Notes is approximately 2.7 years.

The following table sets forth total interest expense recognized related to the 2026 Convertible Notes:


	Year ended
	December 31,
	2023			2022
Contractual interest expense	$	4,305		$	4,313
Amortization of debt issuance costs		1,171			1,150
Total	$	5,476		$	5,463
Effective interest rate		1.9	%		1.9	%

In April 2022, under the terms of the 2026 Convertible Notes Indenture, the Company paid additional interest on the 2026 Convertible Notes at a rate equal to 0.5% per annum, for a total interest payment of approximately $2.1 million, for the period beginning September 25, 2020 and ending March 14, 2022. In September 2022, under the terms of the 2026 Convertible Notes Indenture, the Company paid additional interest on the 2026 Convertible Notes at a rate equal to 0.5% per annum, for a total interest payment of approximately $0.1 million, for the period beginning March 15, 2022 and ending April 8, 2022. These amounts are not included in the table above, but were recorded as interest expense, net within the statement of operations for the year ended December 31, 2022.

2022 Convertible Notes

In August 2015, the Company issued, at par value, $150.0 million aggregate principal amount of 3.00% convertible senior notes due 2022 (the “2022 Convertible Notes”). On August 15, 2022, the Company repaid the outstanding principal amount and accrued interest, totaling $152.3 million, of the 2022 Convertible Notes that was due upon maturity in accordance with the terms of the notes.

The following table sets forth total interest expense recognized related to the 2022 Convertible Notes:


	Year ended
	December 31,
	2023			2022
Contractual interest expense	$	—		$	2,800
Amortization of debt issuance costs		—			460
Total	$	—		$	3,260
Effective interest rate		—	%		3.5	%

Stock award plan - Narrative (Details) - USD ($) $ / shares in Units, $ in Thousands		1 Months Ended		12 Months Ended
	Jun. 08, 2022	Dec. 31, 2022	Dec. 31, 2020	Dec. 31, 2023	Dec. 31, 2022	Dec. 31, 2021	Apr. 30, 2022	Jun. 30, 2021	May 31, 2021	Jan. 31, 2020
Stock option plan
Share-based compensation expense				$ 101,636	$ 110,333	$ 103,513
Unrecognized compensation cost				$ 119,600
Weighted average remaining service period for recognition of unrecognized compensation cost				2 years 1 month 6 days
Stock option
Stock option plan
Granted (in shares)				1,117,284	1,685,435	2,487,234
Options forfeited (in shares)				2,196,820	458,737	742,458
Expected dividend yield (as a percent)				0.00%	0.00%	0.00%
Weighted average grant date fair value (in dollars per share)				$ 21.27	$ 23.54	$ 43.05
Stock option \| Maximum
Stock option plan
Vesting period				4 years
Employee Stock Purchase Plan
Stock option plan
Number of shares authorized (in shares)								2,000,000	1,000,000
Award requisite service period				6 months
Share-based compensation expense				$ 2,400
Employee Stock Purchase Plan \| Minimum
Stock option plan
Purchase price of common stock, percent				85.00%
Employee stock purchase plan, voting percentage limit				5.00%
1998 Employee, Director, and Consultant Stock Option Plan, 2009 Equity and Long-term Incentive Plan, and 2013 Stock Incentive Plan
Stock option plan
Number of shares available for issuance				0
Amended 2013 LTIP
Stock option plan
Number of additional shares authorized (in shares)	8,475,000
Amended 2013 LTIP \| Maximum
Stock option plan
Number of shares authorized (in shares)	16,724,212
2013 Long Term Incentive Plan
Stock option plan
Number of shares available for issuance				8,680,209
2020 Inducement Stock Incentive Plan
Stock option plan
Number of shares available for issuance				1,834,322
Number of additional shares authorized (in shares)			1,000,000
2020 Inducement Stock Incentive Plan \| Maximum
Stock option plan
Number of shares authorized (in shares)										1,000,000
2020 Inducement Stock Incentive Plan \| Common stock \| Maximum
Stock option plan
Number of shares authorized (in shares)							1,300,000
Number of additional shares authorized (in shares)		1,700,000
2020 Inducement Stock Incentive Plan \| Stock option
Stock option plan
Granted (in shares)				139,040
Stockholder's specified ownership percentage				10.00%
Expiration period				10 years
Granted (in dollars per share)				$ 41.25
Options forfeited (in shares)				270,583
2020 Inducement Stock Incentive Plan \| Stock option \| Minimum
Stock option plan
Stock options granted, exercise price as percentage of the fair market value of common stock at grant date				100.00%
Stock options granted to stockholder with specified ownership percentage, exercise price as percentage of the fair market value of common stock at grant date				110.00%
2020 Inducement Stock Incentive Plan \| Restricted stock units
Stock option plan
Granted (in shares)				105,370
Units forfeited (in shares)				79,195

Subsequent events

12 Months Ended

Dec. 31, 2023

Subsequent Events [Abstract]

Subsequent events

18. Subsequent events

The Company has evaluated all subsequent events and transactions through the filing date. There were no material events that impacted the audited consolidated financial statements or disclosures.

Leases - Lease costs (Details) - USD ($) $ in Thousands	12 Months Ended
Leases - Lease costs (Details) - USD ($) $ in Thousands	Dec. 31, 2023	Dec. 31, 2022	Dec. 31, 2021
Leases
Fixed lease cost	$ 21,952	$ 19,804	$ 16,411
Variable lease cost	5,846	4,557	4,361
Short-term lease cost	1,186	808	656
Total operating lease cost	$ 28,984	$ 25,169	$ 21,428

Geographic information (Tables)

12 Months Ended

Dec. 31, 2023

Segments, Geographical Areas [Abstract]

Summary of financial information based on geographical location


	Year Ended December 31, 2023
	United States		Non-US		Total
Total assets	$	1,582,962	$	312,736	$	1,895,698
Fixed assets, net	$	86,421	$	668	$	87,089
Revenue	$	531,661	$	406,161	$	937,822

	Year Ended December 31, 2022
	United States		Non-US		Total
Total assets	$	1,473,770	$	231,849	$	1,705,619
Fixed assets, net	$	71,754	$	836	$	72,590
Revenue	$	381,862	$	316,939	$	698,801

Intangible assets and goodwill - Goodwill (Details) - USD ($) $ in Thousands	12 Months Ended	64 Months Ended
	Dec. 31, 2023	Dec. 31, 2023	Dec. 31, 2022	Aug. 23, 2018
Goodwill
Goodwill	$ 82,341	$ 82,341	$ 82,341
Impairment of goodwill	0
Agilis
Goodwill
Goodwill	82,300	82,300	$ 82,300	$ 82,300
Changes in goodwill		$ 0
Impairment of goodwill	$ 0

- Definition

Amount after accumulated impairment loss of an asset representing future economic benefits arising from other assets acquired in a business combination that are not individually identified and separately recognized.

+ References + Details

- Definition

Line items represent financial concepts included in a table. These concepts are used to disclose reportable information associated with domain members defined in one or many axes to the table.

+ References

Reference 1: http://www.xbrl.org/2003/role/disclosureRef
-Topic 350
-SubTopic 20
-Name Accounting Standards Codification
-Section 50
-Paragraph 1
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147482573/350-20-50-1

Reference 2: http://www.xbrl.org/2003/role/exampleRef
-Topic 350
-SubTopic 20
-Name Accounting Standards Codification
-Section 55
-Paragraph 24
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147482548/350-20-55-24

Reference 3: http://www.xbrl.org/2003/role/disclosureRef
-Topic 350
-SubTopic 20
-Name Accounting Standards Codification
-Section 50
-Paragraph 1
-Subparagraph (a)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147482573/350-20-50-1

Reference 4: http://www.xbrl.org/2003/role/disclosureRef
-Topic 350
-SubTopic 20
-Name Accounting Standards Codification
-Section 50
-Paragraph 1
-Subparagraph (b)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147482573/350-20-50-1

Reference 5: http://www.xbrl.org/2003/role/disclosureRef
-Topic 350
-SubTopic 20
-Name Accounting Standards Codification
-Section 50
-Paragraph 1
-Subparagraph (c)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147482573/350-20-50-1

Reference 6: http://www.xbrl.org/2003/role/disclosureRef
-Topic 350
-SubTopic 20
-Name Accounting Standards Codification
-Section 50
-Paragraph 1
-Subparagraph (d)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147482573/350-20-50-1

Reference 7: http://www.xbrl.org/2003/role/disclosureRef
-Topic 350
-SubTopic 20
-Name Accounting Standards Codification
-Section 50
-Paragraph 1
-Subparagraph (e)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147482573/350-20-50-1

Reference 8: http://www.xbrl.org/2003/role/disclosureRef
-Topic 350
-SubTopic 20
-Name Accounting Standards Codification
-Section 50
-Paragraph 1
-Subparagraph (f)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147482573/350-20-50-1

Reference 9: http://www.xbrl.org/2003/role/disclosureRef
-Topic 350
-SubTopic 20
-Name Accounting Standards Codification
-Section 50
-Paragraph 1
-Subparagraph (g)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147482573/350-20-50-1

Reference 10: http://www.xbrl.org/2003/role/disclosureRef
-Topic 350
-SubTopic 20
-Name Accounting Standards Codification
-Section 50
-Paragraph 1
-Subparagraph (h)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147482573/350-20-50-1

Reference 11: http://www.xbrl.org/2003/role/disclosureRef
-Topic 350
-SubTopic 20
-Name Accounting Standards Codification
-Section 50
-Paragraph 1A
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147482573/350-20-50-1A

Reference 12: http://www.xbrl.org/2003/role/disclosureRef
-Topic 350
-SubTopic 20
-Name Accounting Standards Codification
-Section 50
-Paragraph 2
-Subparagraph (b)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147482573/350-20-50-2

+ Details

Summary of significant accounting policies - Collaboration and Royalty Revenue (Details) - USD ($) $ in Thousands	1 Months Ended		12 Months Ended
	Sep. 30, 2022	Dec. 31, 2021	Dec. 31, 2023	Dec. 31, 2022	Dec. 31, 2021
Collaboration and royalty revenue
Revenue			$ 937,822	$ 698,801	$ 538,593
Collaboration revenue
Collaboration and royalty revenue
Revenue			100,030	50,052	55,046
Royalty revenue
Collaboration and royalty revenue
Revenue			168,856	113,521	54,643
SMA License Agreement \| Collaboration revenue
Collaboration and royalty revenue
Revenue			100,000	50,100	55,000
SMA License Agreement \| Royalty revenue
Collaboration and royalty revenue
Revenue			168,900	$ 113,500	$ 54,600
SMA License Agreement \| Sales Milestones
Collaboration and royalty revenue
Net sales milestone threshold achieved	$ 750,000	$ 500,000	1,500,000
SMA License Agreement \| Sales Milestones \| Prepaids and other current assets
Collaboration and royalty revenue
Milestone payments due	$ 50,000	$ 25,000	$ 100,000

- Definition

+ References

+ Details

Intangible assets and goodwill - (Tables)

12 Months Ended

Dec. 31, 2023

Goodwill and Intangible Assets Disclosure [Abstract]

Schedule of definite lived intangible assets

Definite lived intangible assets consisted of the following at December 31, 2023 and 2022:


		Ending Balance at				Foreign		Ending Balance at
Definite-lived		December 31,				currency		December 31,
intangibles assets, gross		2022		Additions		translation		2023
Emflaza	$	420,253	$	107,164	$	—	$	527,417
Waylivra		9,316		533		369		10,218
Tegsedi		7,109		5,839		374		13,322
Upstaza		89,550		—		—		89,550
Total definite-lived intangibles, gross	$	526,228	$	113,536	$	743	$	640,507


		Ending Balance at				Foreign		Ending Balance at
Definite-lived		December 31,				currency		December 31,
intangibles assets, accumulated amortization		2022		Amortization		translation		2023
Emflaza	$	(266,023)	$	(212,595)	$	—	$	(478,618)
Waylivra		(2,751)		(1,080)		(134)		(3,965)
Tegsedi		(1,709)		(1,498)		(104)		(3,311)
Upstaza		(3,420)		(7,462)		—		(10,882)
Total definite-lived intangibles, accumulated amortization	$	(273,903)	$	(222,635)	$	(238)	$	(496,776)

Total definite-lived intangibles, net							$	143,731

Schedule of estimated future amortization of intangible assets

The estimated future amortization of the Emflaza rights, Waylivra, Tegsedi, and Upstaza intangible assets is expected to be as follows:


	As of December 31, 2023
2024	$	59,312
2025		10,512
2026		10,512
2027		10,512
2028 and thereafter		52,883
Total	$	143,731

Schedule of indefinite lived intangible assets

Indefinite lived intangible assets consisted of the following at December 31, 2023 and 2022:


		Ending Balance at				Reclass from				Foreign		Ending Balance at
Indefinite-lived		December 31,				Indefinite Lived to				currency		December 31,
intangibles assets		2022		Additions		Definite Lived		Impairment		translation		2023
Upstaza	$	235,766	$	—	$	—	$	—	$	—	$	235,766
PTC-FA		112,500		—		—		(112,500)		—		—
PTC-AS		105,300		—		—		(105,300)		—		—
Total indefinite-lived intangibles	$	453,566	$	—	$	—	$	(217,800)	$	—	$	235,766

Total intangible assets, net											$	379,497

Stock award plan - Assumptions Used (Details) - Stock option	12 Months Ended
	Dec. 31, 2023	Dec. 31, 2022	Dec. 31, 2021
Valuation assumptions
Expected term (in years)	5 years 6 months	5 years 6 months	5 years 6 months
Minimum
Valuation assumptions
Risk-free interest rate (as a percent)	3.54%	1.55%	0.51%
Expected volatility (as a percent)	53.00%	54.00%	74.00%
Maximum
Valuation assumptions
Risk-free interest rate (as a percent)	4.69%	4.57%	1.24%
Expected volatility (as a percent)	56.00%	74.00%	89.00%

Income taxes - Loss from operations before tax (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2023	Dec. 31, 2022	Dec. 31, 2021
Income Tax Disclosure [Abstract]
Domestic	$ (784,744)	$ (591,126)	$ (487,726)
Foreign	88,634	3,639	(30,614)
Loss before income tax benefit (expense)	$ (696,110)	$ (587,487)	$ (518,340)

- Definition

Amount of income (loss) from continuing operations, including income (loss) from equity method investments, before deduction of income tax expense (benefit), and income (loss) attributable to noncontrolling interest.

+ References

Reference 1: http://www.xbrl.org/2003/role/disclosureRef
-Topic 944
-SubTopic 220
-Name Accounting Standards Codification
-Section S99
-Paragraph 1
-Subparagraph (SX 210.7-04(11))
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147483586/944-220-S99-1

Reference 2: http://www.xbrl.org/2003/role/disclosureRef
-Topic 280
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 22
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147482810/280-10-50-22

Reference 3: http://www.xbrl.org/2003/role/disclosureRef
-Topic 280
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 30
-Subparagraph (b)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147482810/280-10-50-30

Reference 4: http://www.xbrl.org/2003/role/disclosureRef
-Topic 280
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 32
-Subparagraph (f)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147482810/280-10-50-32

Reference 5: http://www.xbrl.org/2003/role/exampleRef
-Topic 280
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 31
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147482810/280-10-50-31

Reference 6: http://www.xbrl.org/2003/role/disclosureRef
-Topic 280
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 32
-Subparagraph (c)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147482810/280-10-50-32

Reference 7: http://www.xbrl.org/2003/role/disclosureRef
-Topic 942
-SubTopic 235
-Name Accounting Standards Codification
-Section S99
-Paragraph 1
-Subparagraph (SX 210.9-05(b)(2))
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147479557/942-235-S99-1

Reference 8: http://fasb.org/us-gaap/role/ref/legacyRef
-Name Accounting Standards Codification
-Section 25
-Paragraph 1
-SubTopic 20
-Topic 940
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147481913/940-20-25-1

Reference 9: http://fasb.org/us-gaap/role/ref/legacyRef
-Topic 220
-SubTopic 10
-Name Accounting Standards Codification
-Section S99
-Paragraph 2
-Subparagraph (SX 210.5-03(10))
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147483621/220-10-S99-2

Reference 10: http://fasb.org/us-gaap/role/ref/legacyRef
-Topic 942
-SubTopic 220
-Name Accounting Standards Codification
-Section S99
-Paragraph 1
-Subparagraph (SX 210.9-04(15))
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147483589/942-220-S99-1

+ Details

Debt - Narrative (Details)			1 Months Ended		12 Months Ended	18 Months Ended
Debt - Narrative (Details)	Aug. 15, 2022 USD ($)	Jan. 01, 2021 USD ($)	Apr. 08, 2022 USD ($)	Sep. 30, 2019 USD ($) D $ / shares	Dec. 31, 2023 USD ($)	Mar. 14, 2022 USD ($)	Dec. 31, 2022 USD ($)	Dec. 31, 2021 USD ($)	Aug. 31, 2015 USD ($)
Long-term debt
Long-term debt					$ 284,213,000		$ 571,722,000
Additional paid-in capital					2,466,233,000		2,305,020,000
1.50% Convertible senior notes due 2026
Long-term debt
Net deferred tax liability in connection with convertible notes								$ 25,300,000
Convertible debt \| 1.50% Convertible senior notes due 2026
Long-term debt
Net carrying amount					284,213,000		283,044,000
Debt principal amount				$ 287,500,000	$ 287,500,000		$ 287,500,000
Debt instrument additional amount available for repurchase				$ 37,500,000
Interest rate ( as a percent )				1.50%
Net proceeds from issuance of convertible notes				$ 279,300,000
Trading days, number \| D				20
Consecutive trading days, period \| D				30
Stock price trigger				130.00%
Business days, period				5 days
Consecutive trading-day period				5 days
Maximum product of the closing sale price of shares of the Company's common stock and the applicable conversion rate for such trading day				98.00%
Conversion ratio				19.0404
Conversion price per share (in dollars per share) \| $ / shares				$ 52.52
Convertible instruments principal and unpaid interest payable upon events of default				100.00%
Minimum percentage of principal held by convertible debt instrument holders required to issue notice for declaration of principal and unpaid interest payable upon events of default				25.00%
Term of the convertible notes		7 years		7 years	2 years 8 months 12 days
Equity component of convertible notes, net				$ 123,000,000.0
Debt discount to be amortized		$ 0
Incremental interest rate per annum			0.50%			0.50%
Additional interest paid			$ 100,000			$ 2,100,000
Convertible debt \| 1.50% Convertible senior notes due 2026 \| Redemption on or after September 20, 2023
Long-term debt
Trading days, number \| D				19
Consecutive trading days, period \| D				30
Stock price trigger				130.00%
Redemption price as percent of principal amount				100.00%
Sinking fund				$ 0
Convertible debt \| 3.00% Convertible senior notes due 2022
Long-term debt
Debt principal amount									$ 150,000,000.0
Interest rate ( as a percent )									3.00%
Repayment of convertible notes including accrued interest	$ 152,300,000
Cumulative Effect Adjustment \| 1.50% Convertible senior notes due 2026
Long-term debt
Additional paid-in capital		(123,000,000.0)
Amortization of debt discount		$ 16,100,000

Leases

12 Months Ended

Dec. 31, 2023

Leases

Leases

5. Leases

The Company leases office space in South Plainfield, New Jersey for its principal office under two noncancelable operating leases through August 2024, in addition to office and laboratory space in Bridgewater, New Jersey and other locations throughout the United States, as well as office space in various countries for international employees primarily through workspace providers.

The Company also leases approximately 220,500 square feet of office, manufacturing and laboratory space at a facility located in Hopewell Township, New Jersey (the “Campus”) pursuant to a Lease Agreement (the “Hopewell Lease”) with Hopewell Campus Owner LLC (the “Landlord”). The rental term of the Hopewell Lease commenced on July 1, 2020 and has an initial term of fifteen years (the “Hopewell Initial Term”), with two consecutive ten year renewal periods, each at the Company’s option. The aggregate rent for the Hopewell Initial Term will be approximately $111.5 million. The rental rate for the renewal periods will be 95% of the Prevailing Market Rate (as defined in the Hopewell Lease) and determined

at the time of the exercise of the renewal. The Company is also responsible for maintaining certain insurance and the payment of proportional taxes, utilities and common area operating expenses. The Hopewell Lease contains customary events of default, representations, warranties and covenants.

In May 2022, the Company entered into a Lease Agreement (the “Warren Lease”) with Warren CC Acquisitions, LLC (the “Warren Landlord”) relating to the lease of two entire buildings comprised of approximately 360,000 square feet of shell condition, modifiable space (the “Warren Premises”) at a facility located in Warren, New Jersey. The rental term of the Warren Lease commenced on June 1, 2022 (the “Commencement Date”), with an initial term of seventeen years (the “Warren Initial Term”), followed by three consecutive five-year renewal periods at the Company’s option. The aggregate base rent for the Warren Initial Term will be approximately $163.0 million; provided, however, that if the Company is not subject to an Event of Default (as defined in the Warren Lease), the Company will be entitled to a base rent abatement over the first three years of the Warren Initial Term of approximately $18.6 million, reducing the Company’s total base rent obligation to $144.4 million. The rental rate for the renewal periods will be at the Fair Market Rental Value (as defined in the Warren Lease) and determined at the time of the exercise of the renewal. Beginning in the second lease year, the Company is also responsible for the payment of all taxes and operating expenses for the Warren Premises. As a result, the Company recorded an operating lease ROU asset of $28.9 million and an operating lease ROU liability of $28.9 million as of the Commencement Date.

The Company is developing the Warren Premises into office and laboratory space. The Company is entitled to an allowance of approximately $36.2 million to be provided by the Warren Landlord to be used towards such improvements. The Landlord is providing the allowance to cover those assets that are real property improvements, such as structural components, roofs, flooring, etc., whose useful lives are typically longer in nature. Upon the first issuance of a temporary certificate of occupancy for the Warren Premises, the Company will receive $5.0 million from the Landlord, which the Company has committed to fund into the construction account. The Company evaluated the leasehold improvements under ASC 842 and determined that the Company will be the owner of the improvements, and therefore the $36.2 million allowance and $5.0 million due from the Landlord were treated as lease incentives at the commencement of the lease and included in the calculation of the lease ROU asset and lease ROU liability, effectively reducing both at Commencement Date. In connection with the execution of the Warren Lease, the Company also committed to fund a construction account with $3.6 million to go towards the Company’s improvements of the Warren Premises. Subject to the terms of the Warren Lease, the Company has a right of first offer to purchase the Warren Premises if the Warren Landlord receives a bona fide third party offer to purchase the Warren Premises or the Warren Landlord decides to sell the Warren Premises.

The Company also has a finance lease related to its commercial manufacturing agreement with MassBiologics of the University of Massachusetts Medical School (“MassBio”). As of December 31, 2023, the balance of the finance lease liabilities-current and finance lease liabilities-non-current are $3.0 million and $17.2 million, respectively, and are directly related to the Company’s MassBio agreement. As of December 31, 2022, the balance of the finance lease liabilities-current and finance lease liabilities non-current were $3.0 million and $18.7 million, respectively. Additionally, during the years ended December 31, 2023 and December 31, 2022, the Company recorded finance lease costs of $1.5 million and $1.6 million, respectively, related to interest on the lease liability.

The Company also leases certain vehicles, lab equipment, and office equipment under operating leases. The Company’s operating leases have remaining lease terms ranging from 0.2 years to 15.4 years and certain leases include renewal options to extend the lease for up to 15 years. Rent expense was $29.0 million, $25.2 million, and $21.4 million for the years ended December 31, 2023, 2022 and 2021.

The components of lease expense were as follows:


	Year Ended December 31, 2023		Year Ended December 31, 2022		Year Ended December 31, 2021

Operating Lease Cost
Fixed lease cost	$	21,952	$	19,804	$	16,411
Variable lease cost		5,846		4,557		4,361
Short-term lease cost		1,186		808		656
Total operating lease cost	$	28,984	$	25,169	$	21,428

Total operating lease cost is a component of operating expenses on the consolidated statements of operations.

Supplemental lease term and discount rate information related to leases was as follows:


	December 31, 2023		December 31, 2022
Weighted-average remaining lease terms - operating leases (years)	11.55		11.61
Weighted-average discount rate - operating leases	8.69	%	8.61	%
Weighted-average remaining lease terms - finance lease (years)	9.01		10.01
Weighted-average discount rate - finance lease	7.80	%	7.80	%

Supplemental cash flow information related to leases was as follows:


	Year Ended December 31,
	2023		2022		2021
Cash paid for amounts included in the measurement of lease liabilities:
Operating cash flows from operating leases	$	15,338	$	14,736	$	13,683
Financing cash flows from finance lease		1,379		1,276		2,224
Operating cash flows from finance lease		1,621		1,724		776

Right-of-use assets obtained in exchange for lease obligations:
Operating leases	$	—	$	35,817	$	645

Future minimum lease payments under non-cancelable leases as of December 31, 2023 were as follows:


	Operating Leases		Finance Lease
2024	$	18,352	$	3,000
2025		20,450		3,000
2026		19,992		3,000
2027		17,945		3,000
2028 and thereafter		175,847		15,000
Total lease payments		252,586		27,000
Less: Imputed Interest expense		141,957		6,816
Total	$	110,629	$	20,184

Commitments and contingencies - Narrative (Details) - USD ($) $ in Thousands	1 Months Ended	12 Months Ended
	Jul. 31, 2022	Dec. 31, 2023	Dec. 31, 2022	May 29, 2020	Apr. 29, 2020	Jun. 30, 2016
Other Commitments
Milestone payable		$ 2,500
Accrued royalties		74,111	$ 40,546
Wellcome Trust
Other Commitments
Development milestone payment obligations		2,500				$ 800
Censa
Other Commitments
Asset acquisition, development and regulatory milestones				$ 109,000
Development milestone obligation payments made		30,000
Development milestone obligation cash payments made		$ 400
Shares issued for milestone payment		657,462
Censa \| Maximum
Other Commitments
Asset acquisition, milestone, amount				217,500
Asset acquisition, net sales milestone				$ 160,000
Former Censa Securityholders
Other Commitments
Potential developmental and regulatory milestones		$ 65,000
Development milestone obligation payments made		30,000
Development milestone obligation cash payments made		$ 400
Shares issued for milestone payment		657,462
Agilis Merger Agreement \| Marathon
Other Commitments
Development milestone payment obligations			$ 50,000
SMA License Agreement \| SMA Foundation
Other Commitments
Royalty expense		$ 52,500
Royalty payments		35,300
Accrued royalties		17,200
SMA License Agreement \| SMA Foundation \| Maximum
Other Commitments
Potential royalty payments due on net product sales		52,500
Agilis
Other Commitments
Development milestone payments which the entity is obligated to pay		40,000
Potential developmental and regulatory milestones		31,100
Potential sales milestones		50,000			$ 150,000
Milestone obligation payments made	$ 50,000
Development milestone obligation payments made		$ 2,400
Agilis \| Minimum
Other Commitments
Product sales (as a percent)		2.00%			2.00%
Agilis \| Maximum
Other Commitments
Development milestone payment obligations		$ 60,000
Priority review voucher amount		535,000
Potential sales milestones		$ 150,000
Product sales (as a percent)		6.00%			6.00%
Development milestone payment obligations, net of cancellation and forfeiture		$ 20,000
Contingent liability, milestone, potential achievements, priority review voucher amount, net of cancellation and forfeiture		361,000
Agilis \| Agilis Merger Agreement
Other Commitments
Development milestone payment obligations		40,000
Agilis \| Rights Exchange Agreement
Other Commitments
Contingent liability cancellation and forfeiture of potential milestone payments		174,000
Development milestone payment obligations, cancellation and forfeiture		37,600
Contingent liability, cancellation and forfeiture		211,600
BioElectron \| Maximum
Other Commitments
Development milestone payment obligations		$ 200,000

Income taxes

12 Months Ended

Dec. 31, 2023

Income Tax Disclosure [Abstract]

Income taxes

13. Income taxes

The loss from operations before tax benefit (expense) consisted of the following for the years ended December 31, 2023, 2022, and 2021:


	2023		2022		2021
Domestic	$	(784,744)	$	(591,126)	$	(487,726)
Foreign		88,634		3,639		(30,614)
Total	$	(696,110)	$	(587,487)	$	(518,340)

The Income Tax Provision consisted of the following for the years ended December 31, 2023, 2022 and 2021:


	2023		2022		2021
Current:
U.S. Federal	$	—	$	—	$	—
U.S. State and Local		27,226		(4,224)		(3,844)
Foreign		(4,003)		(1,582)		(1,340)
Deferred:
U.S. Federal		36,408		23,689		—
U.S. State and Local		10,521		10,587		(377)
Foreign		(646)		—		—
Total tax benefit (expense)	$	69,506	$	28,470	$	(5,561)

A reconciliation of the U.S. statutory income tax rate to the Company’s effective tax rate is as follows:


	December 31,
	2023		2022		2021
Federal income tax provision at statutory rate	21.00	%	21.00	%	21.00	%
State income tax provision, net of federal benefit	0.32		3.07		(0.74)
Permanent differences	(1.43)		(1.83)		(4.06)
Research and development	4.59		5.89		4.50
Change in valuation allowances	(16.86)		(23.36)		(29.03)
Change in deferred tax assets	—		(0.10)		12.05
Foreign tax rate differential	0.05		(0.17)		0.01
Tax rate change	(1.26)		0.34		0.01
Release (Accrual) of uncertain tax positions	3.71		—		(4.78)
Other	(0.12)		—		(0.03)
Effective income tax rate	10.00	%	4.84	%	(1.07)	%

Accounting for income taxes under U.S. GAAP requires that individual tax-paying entities of the company offset all deferred tax liabilities and assets within each particular tax jurisdiction and present them as a noncurrent deferred tax liability or asset. Amounts in different tax jurisdictions cannot be offset against each other. The noncurrent deferred income tax asset is recorded within deposits and other assets on the balance sheet. The amount of deferred income taxes are as follows:


	December 31,
	2023		2022
Assets:
Noncurrent deferred income taxes	$	—	$	—
Liabilities:
Noncurrent deferred income taxes		(55,905)		(102,834)
Deferred income taxes - net	$	(55,905)	$	(102,834)

The significant components of the Company’s deferred tax assets and liabilities at December 31, 2023 and 2022 are as follows:


	2023		2022
Deferred tax assets:
Accrued expense	$	25,400	$	2,124
Amortization		137,808		52,532
Federal tax credits		205,485		174,802
State tax credits		9,817		9,787
Federal net operating losses		60,270		69,957
State net operating losses		18,680		10,316
Foreign net operating losses		4,052		4,837
Capitalized research and development costs		149,683		110,219
Share based compensation and other		30,757		27,054
Liability for sale of future royalties		190,659		185,589
Noncash interest expense		9,410		30,160
Other comprehensive loss		(728)		(719)
Total gross deferred tax assets		841,293		676,658
Less valuation allowance		(833,810)		(672,172)
Total deferred tax assets, net of valuation allowance	$	7,483	$	4,486
Deferred tax liabilities:
Depreciation	$	(7,483)	$	(4,486)
Indefinite lived intangible		(55,905)		(102,834)
Total gross deferred tax liabilities		(63,388)		(107,320)
Net deferred tax assets (liabilities)	$	(55,905)	$	(102,834)

For the year ended December 31, 2023, the Company generated taxable income in the U.S. of $102.5 million. The Company has not recorded any federal income tax provision after considering the federal NOL, section 250 deduction, available general business credits, and foreign tax credits. The Company recorded a state income tax benefit of $27.2 million which is primarily attributable to the receipt of an outstanding state tax refund received during the year ended December 31, 2023, and the subsequent release of the associated ASC 740 income tax reserve.

At December 31, 2023 and 2022, the Company recorded a valuation allowance against its net deferred tax assets of $833.8 million and $672.2 million, respectively. The change in the valuation allowance during the years ended December 31, 2023 and 2022 was $161.6 million and $146.6 million, respectively. A valuation allowance has been recorded since, in the judgment of management, these assets are not more likely than not to be realized. The ultimate realization of deferred tax assets is dependent upon the generation of future taxable income during periods in which those temporary differences and carryforwards become deductible or are utilized. As of December 31, 2023, the Company had $287.0 million, $208.8 million, and $12.1 million of federal, state, and foreign net operating loss carryforwards, respectively.

The Company recorded a deferred tax liability in conjunction with the Agilis Merger of $122.0 million in 2018, related to the tax basis difference in the IPRD indefinite-lived intangibles acquired. The Company’s policy is to record a deferred tax liability related to acquired IPR&D which may eventually be realized either upon amortization of the asset when the research is completed, and a product is successfully launched or the write-off of the asset if it is abandoned or unsuccessful. In July 2022, the Company received EMEA approval for a portion of the IPR&D assets, and thus, began the amortization of the intangible. In May 2023, as part of a strategic portfolio prioritization, the Company announced the discontinuation of its preclinical and early research programs in gene therapy, which included programs for Friedreich ataxia and

Angelman syndrome. In conjunction with the announcement, the Company recorded an impairment to its indefinite-lived intangible for IP research and development relating to the Friedreich ataxia and Angelman syndrome gene therapy assets. As a result of the impairment, the Company recorded a deferred tax benefit during the 2023 tax year.

As of December 31, 2023, research and development credit carryforward for federal purposes is $35.9 million. In addition, the Orphan Drug Credit Carryover available as of December 31, 2023 is $169.6 million. The Company had research and development carryforwards expiring of $0.6 million in 2023, with additional expiration in 2024 and onwards if not otherwise utilized as projected.

The income tax benefit (expense) for the years ended December 31, 2023 and 2022 differed from the amounts computed by applying the U.S. federal income tax rate of 21% to loss before tax expense as a result of the IPR&D assets becoming partially amortizable in 2022, foreign taxes, the impact of temporary difference, including the updated section 174, the impact of permanent differences, including “global intangible low-taxed income” (“GILTI”), tax credits generated, true up of net operating loss carryforwards, and increase in the Company’s valuation allowance.

Under the 2017 Tax Cuts and Jobs Act, the ability to currently deduct qualifying research and experimental costs under section 174, as well as software development costs, are eliminated for tax years beginning after December 31, 2021. Under the new rule, these costs must be capitalized and amortized over a five-year or fifteen-year period, depending on whether the research is conducted in the U.S. or abroad, respectively. The rule resulted in an increased current taxable income of the Company by $300.3 million for the tax year ended December 31, 2023.

The Company applies the elements of FASB ASC 740-10 regarding accounting for uncertainty in income taxes. This clarifies the accounting for uncertainty in income taxes recognized in financial statements and required impact of a tax position to be recognized in the financial statements if that position is more likely than not of being sustained by the taxing authority. As of December 31, 2023, the Company recorded unrecognized tax benefits in the amount of $1.4 million including interest and penalties through 2023. The Company’s policy is to recognize interest and penalties related to tax matters within the income tax provision. Tax years beginning in 2014 are generally subject to examination by taxing authorities, although net operating losses from all years are subject to examinations and adjustments for at least three years following the year in which the attributes are used. The Company is currently under a wage tax audit in Germany for tax years 2018 through 2021. Although the outcome of tax audits is always uncertain, the company does not expect any adjustment to result for these years as of December 31, 2023.

For all years through December 31, 2016, the Company generated research credits but has not conducted a study to document the qualified activities. This study may result in an adjustment to the Company’s research and development credit carryforwards; however, until a study is completed and any adjustment is known, no amounts are being presented as an uncertain tax position. A full valuation allowance has been provided against the Company’s research and development credits and, if an adjustment is required, this adjustment would be offset by an adjustment to the deferred tax asset established for the research and development credit carryforwards and the valuation allowance.

As a result of U.S. tax reform legislation, distributions of profits from non-U.S. subsidiaries are not expected to cause a significant incremental U.S. tax impact in the future. However, distributions may be subject to non-U.S. withholding taxes if profits are distributed from certain jurisdictions. As of December 31, 2023, for purposes of ASC 740-10-25-3, the Company had $345.9 million of undistributed earnings from non-U.S. subsidiaries that it intends to reinvest permanently in its non-U.S. operations. As these ASC 740-10-25-3 earnings are considered permanently reinvested, no tax provision has been accrued. It is not feasible to estimate the amount of tax that might be payable on the eventual remittance of such earnings.

Unrecognized Tax Benefits

A reconciliation of the gross amount of unrecognized tax benefits, excluding accrued interest and penalties, is as follows:


	Unrecognized Tax Benefits
Balance at December 31, 2022		27,217
Reductions based on settlements or expiration of statute of limitations		(24,671)
Interest received		(1,186)
Balance at December 31, 2023	$	1,360

Uncertain tax positions, for which management’s assessment is that there is a more than 50% probability of sustaining the position upon challenge by a taxing authority based upon its technical merits, are subject to certain recognition and measurement criteria. The nature of the uncertain tax positions is often very complex and subject to change, and the amounts at issue can be substantial. The Company develops its cumulative probability assessment of the measurement of uncertain tax positions using internal experience, judgment, and assistance from professional advisors. The Company re-evaluates these uncertain tax positions on a quarterly basis based on a number of factors including, but not limited to, changes in facts or circumstances, changes in tax law, and effectively settled issues under audit and new audit activity. Any change in these factors could result in the recognition of a tax benefit or an additional charge to the tax provision.

The Company records penalties and tax-related interest expense on unrecognized tax benefits as a component of the provision for income taxes in the accompanying consolidated statement of operations. The Company has not recorded any interest and penalties related to uncertain tax positions for the year ended December 31, 2023, in the accompanying consolidated balance sheet. Future changes in the Company’s unrecognized tax benefits will affect the Company’s annual effective tax rate.

- Definition

The entire disclosure for income taxes. Disclosures may include net deferred tax liability or asset recognized in an enterprise's statement of financial position, net change during the year in the total valuation allowance, approximate tax effect of each type of temporary difference and carryforward that gives rise to a significant portion of deferred tax liabilities and deferred tax assets, utilization of a tax carryback, and tax uncertainties information.

+ References

Reference 1: http://www.xbrl.org/2003/role/disclosureRef
-Topic 946
-SubTopic 20
-Name Accounting Standards Codification
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+ Details

Geographic information

12 Months Ended

Dec. 31, 2023

Segments, Geographical Areas [Abstract]

Geographic information

15. Geographic information

The Company views its operations and manages its business in one operating segment. The following table presents financial information based on the geographic location of the facilities of the Company as of and for the years ended:


	Year Ended December 31, 2023
	United States		Non-US		Total
Total assets	$	1,582,962	$	312,736	$	1,895,698
Fixed assets, net	$	86,421	$	668	$	87,089
Revenue	$	531,661	$	406,161	$	937,822

	Year Ended December 31, 2022
	United States		Non-US		Total
Total assets	$	1,473,770	$	231,849	$	1,705,619
Fixed assets, net	$	71,754	$	836	$	72,590
Revenue	$	381,862	$	316,939	$	698,801

- Definition

The entire disclosure for reporting segments including data and tables. Reportable segments include those that meet any of the following quantitative thresholds a) it's reported revenue, including sales to external customers and intersegment sales or transfers is 10 percent or more of the combined revenue, internal and external, of all operating segments b) the absolute amount of its reported profit or loss is 10 percent or more of the greater, in absolute amount of 1) the combined reported profit of all operating segments that did not report a loss or 2) the combined reported loss of all operating segments that did report a loss c) its assets are 10 percent or more of the combined assets of all operating segments.

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+ Details

Intangible assets and goodwill - Definite-lived Intangibles (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2023	Dec. 31, 2022	Dec. 31, 2021
Definite-lived intangible assets
Milestone Payable	$ 2,500
Accrued royalties	74,111	$ 40,546
Definite-lived intangibles
Amortization	(222,635)	(116,554)	$ (54,751)
Finite-Lived Intangible Assets, Net	$ 143,731
Weighted Average
Definite-lived intangible assets
Remaining amortization period	6 years 4 months 24 days
Emflaza asset acquisition
Definite-lived intangible assets
Milestone obligation payments recorded	$ 107,200
Marathon \| Emflaza asset acquisition \| Accounts payable and accrued expenses
Definite-lived intangible assets
Milestone Payable	52,700
Akcea \| Waylivra
Definite-lived intangible assets
Royalty payments	500
Akcea \| Waylivra \| Accounts payable and accrued expenses
Definite-lived intangible assets
Accrued royalties	400
Akcea \| Tegsedi
Definite-lived intangible assets
Royalty payments	5,800
Akcea \| Tegsedi \| Accounts payable and accrued expenses
Definite-lived intangible assets
Accrued royalties	1,600
Definite-lived intangible assets
Definite-lived intangibles
Beginning balance - Definite-lived assets, Gross	526,228
Additions	113,536
Reclass from Indefinite Lived to Definite Lived	743
Impairment	640,507
Ending balance - Definite-lived assets, Gross		526,228
Beginning balance - Accumulated amortization	(273,903)
Amortization	(222,635)
Foreign Currency Translation - Accumulated amortization	(238)
Ending balance - Accumulated amortization	(496,776)	(273,903)
Finite-Lived Intangible Assets, Net	143,731
Definite-lived intangible assets \| Emflaza
Definite-lived intangibles
Beginning balance - Definite-lived assets, Gross	420,253
Additions	107,164
Impairment	527,417
Ending balance - Definite-lived assets, Gross		420,253
Beginning balance - Accumulated amortization	(266,023)
Amortization	(212,595)
Ending balance - Accumulated amortization	(478,618)	(266,023)
Definite-lived intangible assets \| Waylivra
Definite-lived intangibles
Beginning balance - Definite-lived assets, Gross	9,316
Additions	533
Reclass from Indefinite Lived to Definite Lived	369
Impairment	10,218
Ending balance - Definite-lived assets, Gross		9,316
Beginning balance - Accumulated amortization	(2,751)
Amortization	(1,080)
Foreign Currency Translation - Accumulated amortization	(134)
Ending balance - Accumulated amortization	(3,965)	(2,751)
Definite-lived intangible assets \| Tegsedi
Definite-lived intangibles
Beginning balance - Definite-lived assets, Gross	7,109
Additions	5,839
Reclass from Indefinite Lived to Definite Lived	374
Impairment	13,322
Ending balance - Definite-lived assets, Gross		7,109
Beginning balance - Accumulated amortization	(1,709)
Amortization	(1,498)
Foreign Currency Translation - Accumulated amortization	(104)
Ending balance - Accumulated amortization	(3,311)	(1,709)
Definite-lived intangible assets \| Upstaza
Definite-lived intangibles
Beginning balance - Definite-lived assets, Gross	89,550
Impairment	89,550
Ending balance - Definite-lived assets, Gross		89,550
Beginning balance - Accumulated amortization	(3,420)
Amortization	(7,462)
Ending balance - Accumulated amortization	$ (10,882)	$ (3,420)

Net loss per share - Antidilutive (Details) - shares	12 Months Ended
Net loss per share - Antidilutive (Details) - shares	Dec. 31, 2023	Dec. 31, 2022	Dec. 31, 2021
Net loss per share
Total shares excluded from calculation (in shares)	12,466,669	14,018,753	12,292,413
Employee Stock Option
Net loss per share
Total shares excluded from calculation (in shares)	9,600,399	11,502,417	10,772,582
Restricted stock awards and units
Net loss per share
Total shares excluded from calculation (in shares)	2,866,270	2,516,336	1,519,831

Other comprehensive income (loss) and accumulated other comprehensive items (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2023	Dec. 31, 2022	Dec. 31, 2021
Other comprehensive income (loss)
Balance at the beginning of the period	$ (347,086)	$ 1,438	$ 481,982
Balance at the end of the period	(818,555)	(347,086)	1,438
Unrealized Gains (Losses) On Marketable Securities, net of tax
Other comprehensive income (loss)
Balance at the beginning of the period	(494)	(602)	1,900
Other comprehensive income (loss) before reclassifications	1,135	4,072	(3,279)
Amounts reclassified from other comprehensive items	(315)	(3,964)	777
Other comprehensive income (loss)	820	108	(2,502)
Balance at the end of the period	326	(494)	(602)
Foreign Currency Translation
Other comprehensive income (loss)
Balance at the beginning of the period	5,290	(23,680)	(62,857)
Other comprehensive income (loss) before reclassifications	(6,901)	28,970	39,177
Other comprehensive income (loss)	(6,901)	28,970	39,177
Balance at the end of the period	(1,611)	5,290	(23,680)
AOCI Attributable to Parent
Other comprehensive income (loss)
Balance at the beginning of the period	4,796	(24,282)	(60,957)
Other comprehensive income (loss) before reclassifications	(5,766)	33,042	35,898
Amounts reclassified from other comprehensive items	(315)	(3,964)	777
Other comprehensive income (loss)	(6,081)	29,078	36,675
Balance at the end of the period	$ (1,285)	$ 4,796	$ (24,282)

- Definition

Amount after tax and reclassification adjustments of other comprehensive income (loss).

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- Definition

Amount of equity (deficit) attributable to parent. Excludes temporary equity and equity attributable to noncontrolling interest.

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-URI https://asc.fasb.org//1943274/2147483575/946-220-S99-3

Reference 7: http://www.xbrl.org/2003/role/disclosureRef
-Topic 946
-SubTopic 220
-Name Accounting Standards Codification
-Section S99
-Paragraph 3
-Subparagraph (SX 210.6-09(7))
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147483575/946-220-S99-3

Reference 8: http://www.xbrl.org/2009/role/commonPracticeRef
-Topic 235
-SubTopic 10
-Name Accounting Standards Codification
-Section S99
-Paragraph 1
-Subparagraph (SX 210.4-08(g)(1)(ii))
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147480678/235-10-S99-1

Reference 9: http://www.xbrl.org/2009/role/commonPracticeRef
-Topic 323
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 3
-Subparagraph (c)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147481687/323-10-50-3

Reference 10: http://www.xbrl.org/2009/role/commonPracticeRef
-Topic 825
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 28
-Subparagraph (f)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147482907/825-10-50-28

Reference 11: http://fasb.org/us-gaap/role/ref/legacyRef
-Topic 210
-SubTopic 10
-Name Accounting Standards Codification
-Section S99
-Paragraph 1
-Subparagraph (SX 210.5-02(29))
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Reference 12: http://fasb.org/us-gaap/role/ref/legacyRef
-Topic 210
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-Name Accounting Standards Codification
-Section S99
-Paragraph 1
-Subparagraph (SX 210.5-02(31))
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Reference 13: http://fasb.org/us-gaap/role/ref/legacyRef
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Reference 14: http://fasb.org/us-gaap/role/ref/legacyRef
-Topic 310
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-Name Accounting Standards Codification
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-Paragraph 2
-Subparagraph (SAB Topic 4.E)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147480418/310-10-S99-2

+ Details

Summary of significant accounting policies - Fixed Assets (Details)	Dec. 31, 2023
Computer equipment and software
Fixed assets
Estimated useful life	3 years
Machinery and lab equipment
Fixed assets
Estimated useful life	7 years
Furniture, fixtures, machinery and lab equipment
Fixed assets
Estimated useful life	7 years

Fair value of financial instruments and investments

12 Months Ended

Dec. 31, 2023

Fair Value Disclosures [Abstract]

Fair value of financial instruments and investments

3. Fair value of financial instruments and investments

The Company follows the fair value measurement rules, which provide guidance on the use of fair value in accounting and disclosure for assets and liabilities when such accounting and disclosure is called for by other accounting literature. Cash equivalents, marketable securities, and equity investments are reflected in the accompanying financial statements at fair value. The carrying amount of receivables and accounts payable and accrued expenses approximate fair value due to the short-term nature of those instruments.

The Company uses the market approach to measure fair value for its marketable securities. The market approach uses prices and other relevant information generated by market transactions involving identical or comparable assets. The

Company’s marketable securities are classified as Level 2 as they primarily utilize broker quotes in a nonactive market to value these securities.

The Company owns common stock in ClearPoint Neuro, Inc. (“ClearPoint”) (formerly MRI Interventions, Inc.), a publicly traded medical device company. The ClearPoint equity investments (collectively, the “ClearPoint Equity Investments”) represent financial instruments, and therefore, are recorded at fair value, which is readily determinable. The ClearPoint Equity Investments are components of prepaids and other current assets as of December 31, 2023 and deposits and other assets as of December 31, 2022 on the consolidated balance sheet. The Company classifies its equity investments in ClearPoint as a Level 1 asset within the fair value hierarchy, as the value is based on a quoted market price in an active market, which is not adjusted.

In January 2020, the Company purchased a $10.0 million convertible note from ClearPoint that the Company can convert into ClearPoint shares at a conversion rate of $6.00 per share at any point throughout the term of the loan, which matures five years from the purchase date. The Company determined that the convertible note represents an available for sale debt security and the Company has elected to record it at fair value under ASC 825. The Company classifies its ClearPoint convertible debt security as a Level 2 asset within the fair value hierarchy, as the value is based on inputs other than quoted prices that are observable. The fair value of the ClearPoint convertible debt security is determined at each reporting period by utilizing a Black-Scholes option pricing model, as well as a present value of expected cash flows from the debt security utilizing the risk free rate and the estimated credit spread as of the valuation date as the discount rate. The convertible debt security is considered to be long term and is included as a component of deposits and other assets on the consolidated balance sheet. Other than the ClearPoint Equity Investment and the ClearPoint convertible debt security, no other items included in deposits and other assets and prepaids and other current assets on the consolidated balance sheets are fair valued.

The Company has investments in mutual funds, including one that is denominated in a foreign currency. All of these are equity investments and are classified as marketable securities on the Company’s consolidated balance sheets. These equity investments are reported at fair value, as they are readily available, and as such are classified as Level 1 assets. Unrealized holding gains and losses for these equity investments are included as components of other income (expense), net within the consolidated statement of operations.


	Ending						Foreign						Ending
	Balance at						Currency						Balance at
	December 31,		Unrealized		Realized		Unrealized		Investments		Redemptions/		December 31,
	2022		Gain/(Loss)		Gain/(Loss)		Gain		Purchased		Sale		2023
Marketable securities - equity investments	$	108,261		2,517		4,383		1,384		38,432		(132,343)	$	22,634
ClearPoint Equity Investments		10,965		(1,515)		(782)		—		—		(2,594)		6,074
ClearPoint Convertible Debt Security		15,231		(2,678)		—		—		—		—		12,553
Total Fair Value	$	134,457	$	(1,676)	$	3,601	$	1,384	$	38,432	$	(134,937)	$	41,261


	Ending				Foreign						Ending
	Balance at				Currency						Balance at
	December 31,		Unrealized		Unrealized		Investments				December 31,
	2021		Gain/(Loss)		Gain/(Loss)		Purchased		Redemptions		2022
Marketable securities - equity investments	$	206,973		(7,992)		(549)		22,787		(112,958)	$	108,261
ClearPoint Equity Investments		14,525		(3,560)		—		—		—		10,965
ClearPoint Convertible Debt Security		20,971		(5,740)		—		—		—		15,231
Total Fair Value	$	242,469	$	(17,292)	$	(549)	$	22,787	$	(112,958)	$	134,457

Fair value of marketable securities that are classified as available for sale debt securities is based upon market prices using quoted prices in active markets for identical assets quoted on the last day of the period. In establishing the estimated fair value of the remaining available for sale debt securities, the Company used the fair value as determined by its investment advisors using observable inputs other than quoted prices.


	December 31, 2023
			Quoted prices		Significant
			in active		other		Significant
			markets for		observable		unobservable
			identical assets		inputs		inputs
	Total		(level 1)		(level 2)		(level 3)
Marketable securities - available for sale	$	260,104	$	—	$	260,104	$	—
Marketable securities - equity investments	$	22,634	$	22,634	$	—	$	—
ClearPoint Equity Investments	$	6,074	$	6,074	$	—	$	—
ClearPoint convertible debt security	$	12,553	$	—	$	12,553	$	—
Contingent consideration payable- development and regulatory milestones	$	26,600	$	—	$	—	$	26,600
Contingent consideration payable- net sales milestones	$	9,700	$	—	$	—	$	9,700


	December 31, 2022
			Quoted prices		Significant
			in active		other		Significant
			markets for		observable		unobservable
			identical assets		inputs		inputs
	Total		(level 1)		(level 2)		(level 3)
Marketable securities - available for sale	$	22,610	$	—	$	22,610	$	—
Marketable securities - equity investments	$	108,261	$	108,261	$	—	$	—
ClearPoint Equity Investments	$	10,965	$	10,965	$	—	$	—
ClearPoint convertible debt security	$	15,231	$	—	$	15,231	$	—
Contingent consideration payable- development and regulatory milestones	$	82,500	$	—	$	—	$	82,500
Contingent consideration payable- net sales milestones and royalties	$	81,500	$	—	$	—	$	81,500

No transfers of assets between Level 1 and Level 2 of the fair value measurement hierarchy occurred during the years ended December 31, 2023 and 2022.

The following is a summary of marketable securities accounted for as available for sale debt securities at December 31, 2023 and 2022:


	December 31, 2023
	Amortized		Gross Unrealized
	Cost		Gains		Losses		Fair Value
Commercial paper	$	117,044	$	128	$	(12)	$	117,160
Corporate debt securities		1,650		—		(2)		1,648
Government Obligations		141,084		212		—		141,296
Total	$	259,778	$	340	$	(14)	$	260,104


	December 31, 2022
	Amortized		Gross Unrealized
	Cost		Gains		Losses		Fair Value
Commercial paper	$	12,419	$	5	$	—	$	12,424
Corporate debt securities		10,685		—		(499)		10,186
Total	$	23,104	$	5	$	(499)	$	22,610

For available for sale debt securities in an unrealized loss position, the Company assesses whether it intends to sell or if it is more likely than not that the Company will be required to sell the security before recovery of its amortized cost basis. If either of the criteria regarding intent or requirement to sell is met, the security’s amortized cost basis is written down to fair value. For the years ended December 31, 2023 and 2022, no write downs occurred. The Company does not intend to sell the investments and it is not more likely than not that the Company will be required to sell the investments before recovery of their amortized cost basis, which may be maturity. The Company also reviews its available for sale debt securities in an unrealized loss position and evaluates whether the decline in fair value has resulted from credit losses or other factors. This review is subjective, as it requires management to evaluate whether an event or change in circumstances has occurred in that period that may be related to credit issues. For the years ended December 31, 2023 and 2022, no allowance was recorded for credit losses. Unrealized gains and losses are reported as a component of accumulated other comprehensive (loss) income in stockholders’ deficit.

For the year ended December 31, 2023, the Company had $0.3 million of realized losses from the sale of available for sale debt securities. For the year ended December 31, 2022, the Company had $4.0 million of realized gains from the sale of available for sale debt securities. Realized gains and losses are reported as a component of interest expense, net in the consolidated statement of operations.


	December 31, 2023
	Securities in an unrealized loss				Securities in an unrealized loss
	position less than 12 months				position greater than or equal to 12 months				Total
	Unrealized losses		Fair Value		Unrealized losses		Fair Value		Unrealized losses		Fair Value
Commercial paper	$	(12)		44,446		—		—		(12)		44,446
Corporate debt securities	$	—		—		(2)		1,648		(2)	$	1,648
Total	$	(12)	$	44,446	$	(2)	$	1,648	$	(14)	$	46,094


	December 31, 2022
	Securities in an unrealized loss				Securities in an unrealized loss
	position less than 12 months				position greater than or equal to 12 months				Total
	Unrealized losses		Fair Value		Unrealized losses		Fair Value		Unrealized losses		Fair Value
Corporate debt securities	$	—		—		(499)		10,186		(499)	$	10,186
Total	$	—	$	—	$	(499)	$	10,186	$	(499)	$	10,186

Available for sale debt securities on the balance sheet at December 31, 2023 and 2022 mature as follows:


	December 31, 2023
	Less Than		More Than
	12 Months		12 Months
Commercial paper	$	117,160	$	—
Corporate debt securities		1,648		—
Government obligations		141,296		—
Total	$	260,104	$	—


	December 31, 2022
	Less Than		More Than
	12 Months		12 Months
Commercial paper	$	12,424	$	—
Corporate debt securities		—		10,186
Total	$	12,424	$	10,186

The Company classifies all of its marketable securities as current as they are all either available for sale debt securities or equity investments and are available for current operations.

Convertible senior notes

In September 2019, the Company issued $287.5 million of 1.5% convertible senior notes due September 15, 2026 (the “2026 Convertible Notes). The fair value of the 2026 Convertible Notes, which differs from their carrying values, is influenced by interest rates, the Company’s stock price and stock price volatility and is determined by prices for the 2026 Convertible Notes observed in market trading which are Level 2 inputs. The estimated fair value of the 2026 Convertible Notes at December 31, 2023 and December 31, 2022 was $265.3 million and $281.7 million, respectively.

Level 3 valuation

The contingent consideration payable is fair valued each reporting period with the change in fair value recorded as a gain or loss within the change in the fair value of contingent consideration on the consolidated statements of operations. The fair value of the development and regulatory milestones are estimated utilizing a probability adjusted, discounted cash flow approach. The discount rates are estimated utilizing Corporate B rated bonds maturing in the years of expected payments based on the Company’s estimated development timelines for the acquired product candidate. The fair value of the net sales milestones is determined utilizing a valuation framework that estimates net sales volatility to simulate a range of possible payment scenarios. The average of the payments in these scenarios is then discounted to calculate present fair value.

In May 2023, as part of the Company’s strategic portfolio prioritization, the Company decided to discontinue its preclinical and early research programs in its gene therapy platform, which included programs for Friedreich ataxia and Angelman syndrome. As a result, the Company fully impaired the Friedreich ataxia and Angelman syndrome intangible assets and determined that the fair value for all of the contingent consideration payable related to Friedreich ataxia and Angelman syndrome was $0. The change in fair value for the contingent consideration payable related to Friedreich ataxia and Angelman syndrome for the year ended December 31, 2023 was $128.4 million and is included in the change in fair value of the contingent consideration in the statement of operations. The remaining contingent consideration as of December 31, 2023 is $36.3 million, which is solely related to the development and regulatory milestones, and net sales milestones, for Upstaza.

As of December 31, 2023, the weighted average discount rate for the Upstaza development and regulatory milestones was 5.9% and the weighted average probability of success was 90%. As of December 31, 2023, the weighted average discount rate for the Upstaza net sales milestones was 11.0% and the weighted average probability of success for the net sales milestones was 93%.

The table presented below is a summary of changes in the fair value of the Company’s Level 3 valuation for the contingent consideration payables for the years ended December 31, 2023, and 2022:



	Contingent consideration payable-		Contingent consideration payable-
	development and regulatory		net sales milestones and royalties
	milestones - Agilis		- Agilis
Beginning balance as of December 31, 2021	$	139,300	$	100,600
Additions		—		—
Change in fair value		(6,800)		(19,100)
Payments		(50,000)		—
Ending balance as of December 31, 2022	$	82,500	$	81,500
Additions		—		—
Change in fair value		(55,900)		(71,800)
Payments		—		—
Ending balance as of December 31, 2023	$	26,600	$	9,700

In July 2022, the EC approved Upstaza for the treatment of AADC deficiency for patients 18 months and older within the EEA, which triggered a $50.0 million milestone payment to the former equityholders of Agilis in accordance with the terms of the Agilis Merger Agreement. In accordance with ASC 230, the portion of the $50.0 million milestone payment up to the acquisition date fair value of the contingent consideration liability is classified as a financing outflow and the amount paid in excess of the acquisition date fair value of that liability is classified as an operating outflow on the consolidated statements of cash flows for the year ended December 31, 2022.

The following significant unobservable inputs were used in the valuation of the contingent consideration payables for the years ended December 31, 2023 and 2022:


	December 31, 2023
	Fair Value	Valuation Technique	Unobservable Input	Range
Contingent consideration payable- development and regulatory milestones	$26,600	Probability-adjusted discounted cash flow	Potential development and regulatory milestones Probabilities of success Discount rates Projected years of payments	$0 - $31 million 85% - 92% 5.8% - 6.1% 2024 - 2026
Contingent considerable payable- net sales milestones and royalties	$9,700	Option-pricing model with Monte Carlo simulation	Potential net sales milestones Probabilities of success Potential percentage of net sales for royalties Discount rate Projected years of payments	$0 - $50 million 85% - 100% 0% 11% 2026 - 2034


	December 31, 2022
	Fair Value	Valuation Technique	Unobservable Input	Range
Contingent consideration payable- development and regulatory milestones	$82,500	Probability-adjusted discounted cash flow	Potential development and regulatory milestones Probabilities of success Discount rates Projected years of payments	$0 - $331 million 25% - 92% 6.2% - 8.3% 2023 - 2029
Contingent considerable payable- net sales milestones and royalties	$81,500	Option-pricing model with Monte Carlo simulation	Potential net sales milestones Probabilities of success Potential percentage of net sales for royalties Discount rate Projected years of payments	$0 - $150 million 25% - 100% 2% - 6% 11.5% 2025 - 2041

The contingent consideration payables are classified Level 3 liabilities as their valuation requires substantial judgment and estimation of factors that are not currently observable in the market. If different assumptions were used for the various inputs to the valuation approaches, including but not limited to, assumptions involving probability adjusted sales estimates for the gene therapy platform and estimated discount rates, the estimated fair value could be significantly higher or lower than the fair value determined.

- Definition

The entire disclosure for the fair value of financial instruments (as defined), including financial assets and financial liabilities (collectively, as defined), and the measurements of those instruments as well as disclosures related to the fair value of non-financial assets and liabilities. Such disclosures about the financial instruments, assets, and liabilities would include: (1) the fair value of the required items together with their carrying amounts (as appropriate); (2) for items for which it is not practicable to estimate fair value, disclosure would include: (a) information pertinent to estimating fair value (including, carrying amount, effective interest rate, and maturity, and (b) the reasons why it is not practicable to estimate fair value; (3) significant concentrations of credit risk including: (a) information about the activity, region, or economic characteristics identifying a concentration, (b) the maximum amount of loss the entity is exposed to based on the gross fair value of the related item, (c) policy for requiring collateral or other security and information as to accessing such collateral or security, and (d) the nature and brief description of such collateral or security; (4) quantitative information about market risks and how such risks are managed; (5) for items measured on both a recurring and nonrecurring basis information regarding the inputs used to develop the fair value measurement; and (6) for items presented in the financial statement for which fair value measurement is elected: (a) information necessary to understand the reasons for the election, (b) discussion of the effect of fair value changes on earnings, (c) a description of [similar groups] items for which the election is made and the relation thereof to the balance sheet, the aggregate carrying value of items included in the balance sheet that are not eligible for the election; (7) all other required (as defined) and desired information.

+ References + Details

Fair value of financial instruments and investments - Marketable Securities, Balance Sheet Disclosures (Details) - USD ($) $ in Thousands	Dec. 31, 2023	Dec. 31, 2022
Debt Securities, Available-for-sale [Line Items]
Marketable securities, Less Than 12 Months	$ 260,104	$ 12,424
Marketable securities, More Than 12 Months		10,186
Commercial paper
Debt Securities, Available-for-sale [Line Items]
Marketable securities, Less Than 12 Months	117,160	12,424
Corporate debt securities
Debt Securities, Available-for-sale [Line Items]
Marketable securities, Less Than 12 Months	1,648
Marketable securities, More Than 12 Months		$ 10,186
Government obligations
Debt Securities, Available-for-sale [Line Items]
Marketable securities, Less Than 12 Months	$ 141,296

- Definition

Line items represent financial concepts included in a table. These concepts are used to disclose reportable information associated with domain members defined in one or many axes to the table.

+ References

+ Details

Net loss per share - (Tables)

12 Months Ended

Dec. 31, 2023

Earnings Per Share [Abstract]

Schedule of computation of basic and diluted net loss available to common stockholders

The following table sets forth the computation of basic and diluted net loss per share for common stockholders:


	Year ended December 31,
	2023			2022			2021
Numerator
Net loss	$	(626,604)		$	(559,017)		$	(523,901)
Denominator
Denominator for basic and diluted net loss per share		74,838,392			71,728,634			70,466,393
Net loss per share:
Basic and diluted	$	(8.37)	*	$	(7.79)	*	$	(7.43)	*

*	For the years ended December 31, 2023, 2022, and 2021, the Company experienced a net loss and therefore did not report any dilutive share impact.

Schedule of historical dilutive common share equivalents outstanding


	As of December 31,
	2023	2022	2021
Stock Options	9,600,399	11,502,417	10,772,582
Unvested restricted stock awards and units	2,866,270	2,516,336	1,519,831
Total	12,466,669	14,018,753	12,292,413

Leases - Cash flow (Details) - USD ($) $ in Thousands	12 Months Ended
Leases - Cash flow (Details) - USD ($) $ in Thousands	Dec. 31, 2023	Dec. 31, 2022	Dec. 31, 2021
Leases
Operating cash flows from operating leases	$ 15,338	$ 14,736	$ 13,683
Financing cash flows from finance lease	1,379	1,276	2,224
Operating cash flows from finance leases	$ 1,621	1,724	776
Right-of-use assets obtained in exchange for operating lease obligations		$ 35,817	$ 645

Income taxes - Deferred Tax Assets and Liabilities (Details) - USD ($) $ in Thousands	Dec. 31, 2023	Dec. 31, 2022
Deferred tax assets:
Accrued expense	$ 25,400	$ 2,124
Amortization	137,808	52,532
Federal tax credits	205,485	174,802
State tax credits	9,817	9,787
Federal net operating losses	60,270	69,957
State net operating losses	18,680	10,316
Foreign net operating losses	4,052	4,837
Capitalized research and development costs	149,683	110,219
Share based compensation and other	30,757	27,054
Liability for sale of future royalties	190,659	185,589
Noncash interest expense	9,410	30,160
Other comprehensive loss	(728)	(719)
Total gross deferred tax assets	841,293	676,658
Less valuation allowance	(833,810)	(672,172)
Total deferred tax assets, net of valuation allowance	7,483	4,486
Deferred tax liabilities:
Depreciation	(7,483)	(4,486)
Indefinite lived intangible	(55,905)	(102,834)
Total gross deferred tax liabilities	(63,388)	(107,320)
Net deferred tax assets (liabilities)	$ (55,905)	$ (102,834)

Revenue recognition

12 Months Ended

Dec. 31, 2023

Revenue from Contract with Customer [Abstract]

Revenue recognition

12. Revenue recognition

Net product sales

During the years ended December 31, 2023, 2022, and 2021, net product sales in the United States were $255.1 million, $218.3 million, and $187.3 million, respectively, consisting solely of sales of Emflaza, and net product sales outside of the United States were $406.1 million, $316.9 million, and $241.6 million respectively, consisting of sales of Translarna, Tegsedi, Waylivra, and Upstaza. Translarna net product revenues made up $355.8 million, $288.6 million, and $236.0 million of the net product sales outside the United States for the years ended December 31, 2023, 2022, and 2021, respectively. During the years ended December 31, 2023 and 2022, two countries, the United States and Russia, accounted for at least 10% of the Company’s net product sales, representing $255.1 million and $86.0 million, and $218.3 million and $59.7 million of the net product sales, respectively. During the year ended December 31, 2021, only the United States accounted for at least 10% of the Company’s net product sales. For the years ended December 31, 2023, 2022, and 2021, two of the Company’s distributors each accounted for over 10% of the Company’s net product sales.

As of December 31, 2023 and 2022, the Company does not have a contract liabilities balance related to net product sales, and has not made significant changes to the judgments made in applying ASC Topic 606.

Collaboration revenue and Royalty revenue

In November 2011, the Company and the Spinal Muscular Atrophy Foundation (“SMA Foundation”) entered into a licensing and collaboration agreement with F. Hoffman-La Roche Ltd and Hoffman- La Roche Inc. (collectively, “Roche”). Under the terms of the SMA License Agreement, Roche acquired an exclusive worldwide license to the Company’s SMA program.

Under the agreement, the Company is eligible to receive additional payments from Roche if specified events are achieved with respect to each licensed product, including up to $135.0 million in research and development event milestones, up to $325.0 million in sales milestones upon achievement of certain sales events, and up to double digit royalties on worldwide annual net sales of a commercial product.

For the years ended December 31, 2023, 2022, and 2021, the Company recognized revenue related to the licensing and collaboration agreement with Roche of $100.0 million, $50.1 million, and $55.0 million, respectively. The below summarizes the milestone achievements associated with the Company’s SMA program during the years ended December 31, 2023, 2022, and 2021.

The SMA program currently has one approved product, Evrysdi, which was approved in August 2020 by the FDA for the treatment of SMA in adults and children two months and older. The first commercial sale of Evrysdi in the EU was made in March 2021. This event triggered a $20.0 million milestone payment to the Company from Roche. The first commercial sale in Japan was made in August 2021, which was the final research and development milestone received by the Company. This event triggered a $10.0 million payment to the Company from Roche. In December 2021, the Company recorded its first sales milestone of $25.0 million for the achievement of $500.0 million in worldwide annual net sales from Evrysdi. The Company recorded these three milestone payments as collaboration revenue for the year ended December 31, 2021.

In September 2022, the Company recognized a sales milestone of $50.0 million for the achievement of $750.0 million in worldwide annual net sales from Evrysdi, which is recorded on the balance sheet within prepaid expenses and other current assets as of December 31, 2022. For the year ended December 31, 2023, the Company recognized a sales milestone

of $100.0 million for the achievement of $1.5 billion in worldwide annual net sales from Evrysdi, which is recorded on the balance sheet within prepaid expenses and other current assets as of December 31, 2023. The remaining potential sales milestones as of December 31, 2023 is $150.0 million upon achievement of certain sales events. As of December 31, 2023, the Company does not have any remaining research and development milestones that can be received.

In addition to research and development and sales milestones, the Company is eligible to receive up to double-digit royalties on worldwide annual net sales of a commercial product under the SMA License Agreement. For the years ended December 31, 2023, 2022, and 2021 the Company has recognized $168.9 million, $113.5 million, and $54.6 million of royalty revenue related to Evrysdi, respectively.

Manufacturing Revenue

For the year ended December 31, 2023, the Company recognized $7.7 million of manufacturing revenue related to the production of DNA and AAV vectors for gene therapy applications for external customers. No manufacturing revenue was recognized for the years ended December 31, 2022 and 2021. The Company has not made significant changes to the judgments made in applying ASC Topic 606 for the years ended 2023, 2022, and 2021.

As of December 31, 2023 and 2022, the Company has a contract liabilities balance of $0.8 million and $1.4 million, respectively, relating to the production of plasmid DNA and AAV vectors for gene therapy applications for external customers, which is recorded within deferred revenue on the consolidated balance sheet. For the year ended December 31, 2023, the Company recognized $1.4 million related to the amounts included in the contract liability balance at the beginning of the period.

As of December 31, 2023, the Company has contract assets of $0.2 million related to plasmid DNA and AAV production for external customers, which is recorded within prepaid expenses and other current assets on the consolidated balance sheet. The Company did not have any contract assets for the period ending December 31, 2022.

Remaining performance obligations

As of December 31, 2023, and 2022, the Company has remaining performance obligations of $0.8 million and $1.4 million, respectively, related to the production of plasmid DNA and AAV vectors for gene therapy applications for external customers. The Company expects to recognize revenue over the next one year, as the specific timing for satisfying the performance obligations is contingent upon a number of factors, including customers’ needs and schedules.

- Definition

The entire disclosure of revenue from contract with customer to transfer good or service and to transfer nonfinancial asset. Includes, but is not limited to, disaggregation of revenue, credit loss recognized from contract with customer, judgment and change in judgment related to contract with customer, and asset recognized from cost incurred to obtain or fulfill contract with customer. Excludes insurance and lease contracts.

+ References

Reference 1: http://www.xbrl.org/2003/role/disclosureRef
-Topic 606
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 9
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147479806/606-10-50-9

Reference 2: http://www.xbrl.org/2003/role/disclosureRef
-Topic 606
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 10
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147479806/606-10-50-10

Reference 3: http://www.xbrl.org/2003/role/disclosureRef
-Topic 606
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 15
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147479806/606-10-50-15

Reference 4: http://www.xbrl.org/2003/role/disclosureRef
-Topic 606
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 12
-Subparagraph (a)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147479806/606-10-50-12

Reference 5: http://www.xbrl.org/2003/role/disclosureRef
-Topic 606
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 12
-Subparagraph (b)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147479806/606-10-50-12

Reference 6: http://www.xbrl.org/2003/role/disclosureRef
-Topic 606
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 12
-Subparagraph (c)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147479806/606-10-50-12

Reference 7: http://www.xbrl.org/2003/role/disclosureRef
-Topic 606
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 12
-Subparagraph (d)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147479806/606-10-50-12

Reference 8: http://www.xbrl.org/2003/role/disclosureRef
-Topic 606
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 12
-Subparagraph (e)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147479806/606-10-50-12

Reference 9: http://www.xbrl.org/2003/role/disclosureRef
-Topic 606
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 13
-Subparagraph (b)(2)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147479806/606-10-50-13

Reference 10: http://www.xbrl.org/2003/role/disclosureRef
-Name Accounting Standards Codification
-Topic 606
-Publisher FASB
-URI https://asc.fasb.org//606/tableOfContent

+ Details

Debt - (Tables)

12 Months Ended

Dec. 31, 2023

Debt Instrument, Redemption [Line Items]

Summary of liability for sale of future royalties


	Year Ended December 31,
Liability for sale of future royalties- (current and noncurrent)	2023
Beginning balance as of December 31, 2022	$	757,886
Less: Non-cash royalty revenue payable to RPI		(93,460)
Plus: Non-cash interest expense recognized		104,790
Plus: Cash Consideration		1,000,000
Plus: Loss on Debt Extinguishment		44,881
Ending balance	$	1,814,097
Effective interest rate as of December 31, 2023		10.8%

1.50% Convertible senior notes due 2026

Debt Instrument, Redemption [Line Items]

Summary of convertible notes

The 2026 Convertible Notes consist of the following:


		Year ended December 31,
	2023			2022
Principal	$	287,500	$	287,500
Less: Debt issuance costs		(3,287)		(4,456)
Net carrying amount	$	284,213	$	283,044

Summary of interest expense recognized related to debt

The following table sets forth total interest expense recognized related to the 2026 Convertible Notes:


	Year ended
	December 31,
	2023			2022
Contractual interest expense	$	4,305		$	4,313
Amortization of debt issuance costs		1,171			1,150
Total	$	5,476		$	5,463
Effective interest rate		1.9	%		1.9	%

3.00% Convertible senior notes due 2022

Debt Instrument, Redemption [Line Items]

Summary of interest expense recognized related to debt

The following table sets forth total interest expense recognized related to the 2022 Convertible Notes:


	Year ended
	December 31,
	2023			2022
Contractual interest expense	$	—		$	2,800
Amortization of debt issuance costs		—			460
Total	$	—		$	3,260
Effective interest rate		—	%		3.5	%

Blackstone Credit Agreement

Debt Instrument, Redemption [Line Items]

Schedule of credit agreement

The Blackstone Credit Agreement consisted of the following:


		Year ended December 31,
	2023			2022
Principal	$	300,000	$	300,000
Less: Debt issuance costs		—		(11,322)
Repayment of senior secured term loan		(300,000)		—
Net carrying amount	$	—	$	288,678

Summary of interest expense recognized related to debt

The following table sets forth total interest expense recognized related to the Blackstone Credit Agreement:


	Year Ended			Year Ended
	December 31,			December 31,
	2023			2022
Contractual interest expense	$	30,198		$	6,069
Amortization of debt issuance costs		702			290
Total	$	30,900		$	6,359
Effective interest rate		13.1	%		12.2	%