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UNITED STATES

SECURITIES AND EXCHANGE COMMISSION

Washington, D.C. 20549

FORM 10-Q

(Mark One)

     QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934

For the quarterly period ended September 30, 2023

or

         TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934

For the transition period from            to

Commission file number: 001-35969

PTC Therapeutics, Inc.

(Exact name of registrant as specified in its charter)

Delaware

    

04-3416587

(State or other jurisdiction of incorporation or organization)

(I.R.S. Employer Identification No.)

100 Corporate Court

    

South Plainfield, NJ

07080

(Address of principal executive offices)

(Zip Code)

(908) 222-7000

(Registrant’s telephone number, including area code)

Securities registered pursuant to Section 12(b) of the Act:

Title of each class

    

Trading Symbol(s)

    

Name of each exchange on which registered

Common Stock, $0.001 par value per share

PTCT

Nasdaq Global Select Market

Indicate by check mark whether the registrant (1) has filed all reports required to be filed by Section 13 or 15(d) of the Securities Exchange Act of 1934 during the preceding 12 months (or for such shorter period that the registrant was required to file such reports), and (2) has been subject to such filing requirements for the past 90 days. Yes þ  No 

Indicate by check mark whether the registrant has submitted electronically every Interactive Data File required to be submitted pursuant to Rule 405 of Regulation S-T (§232.405 of this chapter) during the preceding 12 months (or for such shorter period that the registrant was required to submit such files). Yes þ  No 

Indicate by check mark whether the registrant is a large accelerated filer, an accelerated filer, a non-accelerated filer, a smaller reporting company, or an emerging growth company. See the definitions of “large accelerated filer”, “accelerated filer,” “smaller reporting company,” and "emerging growth company" in Rule 12b-2 of the Exchange Act.

Large accelerated filer

þ

Accelerated filer

Non-accelerated filer

Smaller reporting company

Emerging growth company

If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act.

Indicate by check mark whether the registrant is a shell company (as defined in Rule 12b-2 of the Exchange Act). Yes   No þ

As of October 24, 2023, there were 75,463,145 shares of Common Stock, $0.001 par value per share, outstanding.

i

FORWARD-LOOKING STATEMENTS

This Quarterly Report on Form 10-Q contains forward-looking statements that involve substantial risks and uncertainties. All statements, other than statements of historical facts, contained in this Quarterly Report on Form 10-Q, including statements regarding our strategy, future operations, future financial position, future revenues, projected costs, prospects, plans and objectives of management, are forward-looking statements. The words “anticipate,” “believe,” “estimate,” “expect,” “intend,” “may,” “might,” “plan,” “predict,” “project,” “target,” “potential,” “will,” “would,” “could,” “should,” “continue,” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words.

The forward-looking statements in this Quarterly Report on Form 10-Q include, among other things, statements about:

our ability to negotiate, secure and maintain adequate pricing, coverage and reimbursement terms and processes on a timely basis, or at all, with third-party payors for our products or product candidates that we commercialize or may commercialize in the future;
our ability to maintain our conditional marketing authorization of TranslarnaTM (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy, or nmDMD, in the European Economic Area, or EEA, and our marketing authorizations in other jurisdictions in which Translarna has been approved, including whether the European Medicines Agency, or EMA, determines in the re-examination process of the Committee for Medicinal Products for Human Use’s negative opinion that the benefit-risk balance of Translarna authorization supports renewal of such authorization;
our ability to utilize results from Study 041 to support a renewal of the conditional marketing authorization for Translarna for the treatment of nmDMD in the EEA and to support a marketing approval for Translarna for the treatment of nmDMD in the United States;
expectations with respect to our ability to commercialize UpstazaTM (eladocagene exuparvovec) for the treatment of Aromatic L-Amino Acid Decarboxylase, or AADC deficiency, in the EEA, any potential regulatory submissions and potential approvals for our product candidates, our manufacturing capabilities and the potential financial impact and benefits of our leased biologics manufacturing facility and the potential achievement of development, regulatory and sales milestones and contingent payments that we may be obligated to make;
the anticipated period of market exclusivity for Emflaza® (deflazacort) for the treatment of Duchenne muscular dystrophy in the United States under the Orphan Drug Act of 1983;
our expectations with respect to the commercial status of Evrysdi® (risdiplam) and our program directed against spinal muscular atrophy in collaboration with F. Hoffmann La Roche Ltd and Hoffmann La Roche Inc. and the Spinal Muscular Atrophy Foundation and our estimates regarding future revenues from sales-based royalty payments or the achievement of milestones in that program;
our expectations and the potential financial impact and benefits related to our Collaboration and License Agreement with a subsidiary of Ionis Pharmaceuticals, Inc. including with respect to the timing of regulatory approval of Tegsedi® (inotersen) and WaylivraTM (volanesorsen) in countries in which we are licensed to commercialize them, the commercialization of Tegsedi and Waylivra, and our expectations with respect to royalty payments by us based on our potential achievement of certain net sales thresholds;
the timing and scope of our commercialization of our products and product candidates;
our estimates regarding the potential market opportunity for our products or product candidates, including the size of eligible patient populations and our ability to identify such patients;
our ability to obtain additional and maintain existing reimbursed named patient and cohort early access programs for our products on adequate terms, or at all;

1

our estimates regarding expenses, future revenues, third-party discounts and rebates, capital requirements and needs for additional financing, including our ability to maintain the level of our expenses consistent with our internal budgets and forecasts and to secure additional funds on favorable terms or at all;
the timing and conduct of our ongoing, planned and potential future clinical trials and studies in our splicing, metabolic, Bio-e and oncology programs as well as studies in our products for maintaining authorizations, label extensions and additional indications, including the timing of initiation, enrollment and completion of the trials and the period during which the results of the trials will become available;
our ability to realize the anticipated benefits of our acquisitions or other strategic transactions, including the possibility that the expected impact of benefits from the acquisitions or strategic transactions will not be realized or will not be realized within the expected time period, significant transaction costs, the integration of operations and employees into our business, our ability to obtain marketing approval of our product candidates we acquired from the acquisitions or other strategic transactions and unknown liabilities;
the rate and degree of market acceptance and clinical utility of any of our products or product candidates;
the ability and willingness of patients and healthcare professionals to access our products and product candidates through alternative means if pricing and reimbursement negotiations in the applicable territory do not have a positive outcome;
the timing of, and our ability to obtain additional marketing authorizations for our products and product candidates;
the ability of our products and our product candidates to meet existing or future regulatory standards;
our ability to complete Study 041, a multicenter, randomized, double-blind, 18-month, placebo-controlled clinical trial of Translarna for the treatment of nmDMD followed by an 18-month open-label extension, according to the protocol agreed with the EMA;
the potential receipt of revenues from future sales of our products or product candidates;
our sales, marketing and distribution capabilities and strategy, including the ability of our third-party manufacturers to manufacture and deliver our products and product candidates in clinically and commercially sufficient quantities and the ability of distributors to process orders in a timely manner and satisfy their other obligations to us;
our ability to establish and maintain arrangements for the manufacture of our products and product candidates that are sufficient to meet clinical trial and commercial launch requirements;
the extent, timing and financial aspects of our strategic pipeline prioritization and reductions in workforce;
our ability to complete any post-marketing requirements imposed by regulatory agencies with respect to our products;
our expectations with respect to the potential financial impact and benefits of our leased biologics manufacturing facility and our ability to satisfy our obligations under the terms of the lease agreement for such facility;
our ability to satisfy our obligations under the indenture governing our 1.50% convertible senior notes due September 15, 2026;
our regulatory submissions, including with respect to timing and outcome of regulatory review;
our plans to advance our earlier stage programs and pursue research and development of other product candidates, including our splicing, metabolic, Bio-e and oncology programs;

2

our expectations with respect to the COVID-19 pandemic and related response measures and their effects on our business, operations, clinical trials, potential regulatory submissions and approvals, our collaborators, contract research organizations, suppliers and manufacturers;
whether we may pursue business development opportunities, including potential collaborations, alliances, and acquisition or licensing of assets and our ability to successfully develop or commercialize any assets to which we may gain rights pursuant to such business development opportunities;
the potential advantages of our products and any product candidate;
our intellectual property position;
the impact of government laws and regulations;
the impact of litigation that has been or may be brought against us or of litigation that we are pursuing against others; and
our competitive position.

We may not actually achieve the plans, intentions or expectations disclosed in our forward-looking statements, and you should not place undue reliance on our forward-looking statements. Actual results or events could differ materially from the plans, intentions and expectations disclosed in the forward-looking statements we make. We have included important factors in the cautionary statements included in this Quarterly Report on Form 10-Q, particularly in Part II, Item 1A. Risk Factors as well as in Part I, Item 1A. Risk Factors in our Annual Report on Form 10-K for the year ended December 31, 2022, that we believe could cause actual results or events to differ materially from the forward-looking statements that we make. Our forward-looking statements do not reflect the potential impact of any future acquisitions, mergers, dispositions, joint ventures or investments we may make.

You should read this Quarterly Report on Form 10-Q and the documents that we have filed as exhibits to this Quarterly Report on Form 10-Q and our Annual Report on Form 10-K for the year ended December 31, 2022 completely and with the understanding that our actual future results may be materially different from what we expect. We do not assume any obligation to update any forward-looking statements whether as a result of new information, future events or otherwise, except as required by applicable law.

In this Quarterly Report on Form 10-Q, unless otherwise stated or the context otherwise requires, references to “PTC,” “PTC Therapeutics,” “the Company,” “we,” “us,” “our,” and similar references refer to PTC Therapeutics, Inc. and, where appropriate, its subsidiaries. The trademarks, trade names and service marks appearing in this Quarterly Report on Form 10-Q are the property of their respective owners.

All website addresses given in this Quarterly Report on Form 10-Q are for information only and are not intended to be an active link or to incorporate any website information into this document.

3

PART I—FINANCIAL INFORMATION

Item 1. Financial Statements.

PTC Therapeutics, Inc.

Consolidated Balance Sheets (unaudited)

In thousands (except shares)

September 30, 

December 31, 

    

2023

    

2022

Assets

Current assets:

 

 

  

Cash and cash equivalents

$

165,157

$

279,834

Marketable securities

 

129,653

 

130,871

Trade and royalty receivables, net

 

165,718

 

155,614

Inventory, net

 

35,752

 

21,808

Prepaid expenses and other current assets

 

44,082

 

105,658

Total current assets

 

540,362

 

693,785

Fixed assets, net

 

85,031

 

72,590

Intangible assets, net

 

423,342

 

705,891

Goodwill

 

82,341

 

82,341

Operating lease ROU assets

94,072

102,430

Deposits and other assets

 

34,737

 

48,582

Total assets

$

1,259,885

$

1,705,619

Liabilities and stockholders’ deficit

 

  

 

  

Current liabilities:

 

  

 

  

Accounts payable and accrued expenses

$

370,731

$

320,366

Deferred revenue

 

1,224

 

1,351

Operating lease liabilities- current

11,703

9,370

Finance lease liabilities- current

2,611

3,000

Liability for sale of future royalties- current

105,849

72,149

Total current liabilities

 

492,118

 

406,236

Long-term debt

 

573,174

 

571,722

Contingent consideration payable

 

39,000

 

164,000

Deferred tax liability

 

51,927

 

102,834

Operating lease liabilities- noncurrent

99,682

100,860

Finance lease liabilities- noncurrent

17,184

18,675

Liability for sale of future royalties- noncurrent

657,469

685,737

Other long-term liabilities

141

2,641

Total liabilities

 

1,930,695

 

2,052,705

Stockholders’ deficit:

 

  

 

  

Common stock, $0.001 par value. Authorized 250,000,000 shares; issued and outstanding 75,459,022 shares at September 30, 2023. Authorized 250,000,000 shares; issued and outstanding 73,104,692 shares at December 31, 2022.

 

75

 

72

Additional paid-in capital

 

2,447,292

 

2,305,020

Accumulated other comprehensive income

 

9,609

 

4,796

Accumulated deficit

 

(3,127,786)

 

(2,656,974)

Total stockholders’ deficit

 

(670,810)

 

(347,086)

Total liabilities and stockholders’ deficit

$

1,259,885

$

1,705,619

See accompanying unaudited notes.

4

PTC Therapeutics, Inc.

Consolidated Statements of Operations (unaudited)

In thousands (except shares and per share amounts)

Three Months Ended September 30, 

Nine Months Ended September 30, 

    

2023

    

2022

    

2023

    

2022

Revenues:

 

  

 

  

  

 

  

Net product revenue

$

144,038

$

134,186

$

506,187

$

407,720

Collaboration revenue

 

50,017

 

6

 

50,024

Royalty revenue

50,173

32,924

117,857

73,645

Manufacturing revenue

2,365

6,716

Total revenues

 

196,576

 

217,127

 

630,766

 

531,389

Operating expenses:

Cost of product sales, excluding amortization of acquired intangible assets

 

9,493

14,011

 

36,368

 

33,785

Amortization of acquired intangible assets

 

58,649

31,023

 

145,461

 

80,790

Research and development

 

164,212

165,462

 

545,210

 

462,802

Selling, general and administrative

 

80,886

80,118

 

256,249

 

233,280

Change in the fair value of contingent consideration

 

1,500

(5,300)

 

(125,000)

 

(32,200)

Intangible asset impairment

217,800

Total operating expenses

 

314,740

 

285,314

 

1,076,088

 

778,457

Loss from operations

 

(118,164)

 

(68,187)

 

(445,322)

 

(247,068)

Interest expense, net

 

(28,160)

(20,880)

 

(84,905)

 

(66,371)

Other expense, net

 

(20,266)

(38,141)

 

(8,832)

 

(84,355)

Loss before income tax benefit

 

(166,590)

 

(127,208)

 

(539,059)

 

(397,794)

Income tax benefit

 

33,620

17,893

 

68,247

 

9,666

Net loss attributable to common stockholders

$

(132,970)

$

(109,315)

$

(470,812)

$

(388,128)

Weighted-average shares outstanding:

Basic and diluted (in shares)

 

75,377,997

71,654,671

 

74,618,611

 

71,415,849

Net loss per share—basic and diluted (in dollars per share)

$

(1.76)

$

(1.53)

$

(6.31)

$

(5.43)

See accompanying unaudited notes.

5

PTC Therapeutics, Inc.

Consolidated Statements of Comprehensive Loss (unaudited)

In thousands

Three Months Ended September 30, 

Nine Months Ended September 30, 

     

2023

     

2022

     

2023

     

2022

Net loss

$

(132,970)

$

(109,315)

$

(470,812)

$

(388,128)

Other comprehensive income (loss):

 

  

 

  

 

 

  

Unrealized gain (loss) on marketable securities, net of tax

 

19

736

 

470

 

(2,333)

Foreign currency translation gain, net of tax

 

11,021

25,218

 

4,343

 

62,820

Comprehensive loss

$

(121,930)

$

(83,361)

$

(465,999)

$

(327,641)

See accompanying unaudited notes.

6

PTC Therapeutics, Inc.

Consolidated Statements of Stockholders’ (Deficit) Equity (unaudited)

In thousands (except shares)

 

 

Accumulated

 

 

 

Additional

 

other

 

Total

Three months ended September 30, 2023

Common stock

paid-in

 

comprehensive

Accumulated

stockholders’

    

Shares

    

Amount

    

capital

    

(loss) income

    

deficit

    

deficit

Balance, June 30, 2023

75,318,233

$

75

$

2,416,904

$

(1,431)

$

(2,994,816)

$

(579,268)

Exercise of options

 

113,795

3,446

3,446

Restricted stock vesting and issuance, net

 

26,994

Share-based compensation expense

 

26,942

26,942

Net loss

 

(132,970)

(132,970)

Comprehensive income

 

11,040

11,040

Balance, September 30, 2023

 

75,459,022

$

75

$

2,447,292

$

9,609

$

(3,127,786)

$

(670,810)

 

 

Accumulated

 

 

 

Additional

 

other

 

Total

Three months ended September 30, 2022

Common stock

paid-in

 

comprehensive

Accumulated

stockholders’

    

Shares

    

Amount

    

capital

    

income

    

deficit

    

deficit

Balance, June 30, 2022

 

71,505,889

$

71

$

2,184,230

$

10,251

$

(2,376,770)

$

(182,218)

Exercise of options

 

331,379

10,005

10,005

Restricted stock vesting and issuance, net

 

17,624

Share-based compensation expense

 

28,670

28,670

Net loss

 

 

 

 

(109,315)

 

(109,315)

Comprehensive income

 

 

 

 

25,954

 

25,954

Balance, September 30, 2022

 

71,854,892

$

71

$

2,222,905

$

36,205

$

(2,486,085)

$

(226,904)

 

 

Accumulated

 

 

 

Additional

 

other

 

Total

Nine months ended September 30, 2023

Common stock

paid-in

 

comprehensive

Accumulated

stockholders’

    

Shares

    

Amount

    

capital

    

income

    

deficit

    

deficit

Balance, December 31, 2022

 

73,104,692

$

72

$

2,305,020

$

4,796

$

(2,656,974)

$

(347,086)

Exercise of options

 

806,407

1

23,770

23,771

Restricted stock vesting and issuance, net

 

773,157

1

1

Issuance of common stock in connection with an employee stock purchase plan

 

117,304

3,805

3,805

Issuance of common stock in connection with a milestone payable

657,462

1

29,569

29,570

Share-based compensation expense

 

85,128

85,128

Net loss

 

(470,812)

(470,812)

Comprehensive income

 

4,813

4,813

Balance, September 30, 2023

 

75,459,022

$

75

$

2,447,292

$

9,609

$

(3,127,786)

$

(670,810)

 

 

Accumulated

 

 

 

Additional

 

other

 

Total

Nine months ended September 30, 2022

Common stock

paid-in

 

comprehensive

Accumulated

stockholders’

    

Shares

    

Amount

    

capital

    

(loss) income

    

deficit

    

equity (deficit)

Balance, December 31, 2021

 

70,828,226

$

71

$

2,123,606

$

(24,282)

$

(2,097,957)

$

1,438

Exercise of options

456,399

 

 

13,203

 

 

13,203

Restricted stock vesting and issuance, net

479,004

 

 

 

 

Issuance of common stock in connection with an employee stock purchase plan

 

91,263

 

 

3,107

 

 

 

3,107

Share-based compensation expense

 

 

 

82,989

 

 

 

82,989

Net loss

 

 

 

 

 

(388,128)

 

(388,128)

Comprehensive income

 

 

 

 

60,487

 

 

60,487

Balance, September 30, 2022

 

71,854,892

$

71

$

2,222,905

$

36,205

$

(2,486,085)

$

(226,904)

See accompanying unaudited notes.

7

PTC Therapeutics, Inc.

Consolidated Statements of Cash Flows (unaudited)

In thousands

Nine Months Ended September 30, 

    

2023

    

2022

Cash flows from operating activities

Net loss

$

(470,812)

$

(388,128)

Adjustments to reconcile net loss to net cash used in operating activities:

 

  

Depreciation and amortization

 

155,885

89,971

Non-cash operating lease expense

 

8,156

7,505

Non-cash royalty revenue related to sale of future royalties

(50,599)

(31,617)

Non-cash interest expense on liability related to sale of future royalties

56,031

55,778

Intangible asset impairment

217,800

Change in valuation of contingent consideration

 

(125,000)

(32,200)

Unrealized loss on ClearPoint Equity Investments

 

3,107

1,113

Unrealized loss on ClearPoint convertible debt security

4,594

2,392

Unrealized (gain) loss on marketable securities- equity investments

(5,484)

11,319

Realized loss on the sale of Clearpoint Equity Investment

782

Non-cash stock consideration, milestone payment

29,570

Disposal of asset

135

81

Deferred income taxes

(50,909)

Amortization of (discounts) premiums on investments, net

 

(89)

1,792

Amortization of debt issuance costs

 

1,574

1,316

Share-based compensation expense

 

85,128

82,989

Unrealized foreign currency transaction losses, net

 

3,394

71,246

Changes in operating assets and liabilities:

 

Inventory, net

 

(14,121)

(768)

Prepaid expenses and other current assets

 

59,467

(35,615)

Trade and royalty receivables, net

 

(9,887)

(24,773)

Deposits and other assets

 

4,597

(3,752)

Accounts payable and accrued expenses

 

40,434

1,568

Other liabilities

 

(1,756)

(908)

Deferred revenue

 

(127)

Net cash used in operating activities

$

(58,130)

$

(190,691)

Cash flows from investing activities

 

 

Purchases of fixed assets

$

(22,872)

$

(23,394)

Purchases of marketable securities- available for sale

(40,429)

Purchases of marketable securities- equity investments

(26,378)

(22,787)

Sale and redemption of marketable securities- available for sale

21,544

341,990

Sale and redemption of marketable securities- equity investments

12,078

104,431

Sale and redemption of ClearPoint Equity Investments

2,594

Acquisition of product rights and licenses

(69,285)

(102,069)

Net cash (used in) provided by investing activities

$

(82,319)

$

257,742

Cash flows from financing activities

 

 

Proceeds from exercise of options

$

23,771

$

13,203

Repayment of Convertible Notes

(150,000)

Proceeds from employee stock purchase plan

 

3,805

3,107

Debt issuance costs related to senior secured term loan

(282)

Payment of finance lease principal

(1,379)

(1,276)

Net cash provided by (used in) financing activities

$

25,915

$

(134,966)

Effect of exchange rate changes on cash

 

19

(8,792)

Net decrease in cash and cash equivalents

 

(114,515)

 

(76,707)

Cash and cash equivalents, and restricted cash beginning of period

 

295,925

197,218

Cash and cash equivalents, and restricted cash end of period

$

181,410

$

120,511

Supplemental disclosure of cash information

 

 

Cash paid for interest

$

34,020

$

12,679

Cash paid for income taxes

13,631

4,029

Supplemental disclosure of non-cash investing and financing activity

 

 

  

Unrealized gain (loss) on marketable securities, net of tax

$

470

$

(2,333)

Right-of-use assets obtained in exchange for operating lease obligations

35,294

Acquisition of product rights and licenses

44,963

27,828

Milestone payable

2,500

50,000

See accompanying unaudited notes.

8

PTC Therapeutics, Inc.

Notes to Consolidated Financial Statements (unaudited)

September 30, 2023

In thousands (except share and per share amounts unless otherwise noted)

1.        The Company

PTC Therapeutics, Inc. (the “Company” or “PTC”) is a science-driven 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.

PTC has a portfolio pipeline that includes several commercial products and product candidates in various stages of development, including clinical, pre-clinical and research and discovery stages, focused on the development of new treatments for multiple therapeutic areas for rare diseases relating to neurology, metabolism and oncology.

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 and in Russia for the treatment of nmDMD in patients aged two years and older. Translarna also has marketing authorization in Brazil for the treatment of nmDMD in ambulatory patients two years and older and for continued treatment of patients that become non-ambulatory. 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. 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. The Company has requested re-examination of the CHMP opinion regarding the renewal of the existing conditional marketing authorization. In re-examination, the Company intends to address concerns raised during the previous review process on the benefit demonstrated in Translarna clinical trials and regarding the robustness of the Strategic Targeting of Registries and International Database of Excellence, or STRIDE, real world registry. In accordance with EMA guidelines, the Company expects the CHMP opinion following the re-examination process to occur in late January 2024, with EC ratification of the opinion within the following 67 days.

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 an NDA re-submission. The Company then had an informal meeting with the FDA, during which the Company discussed the potential path to an NDA re-submission for Translarna. Based on the meeting discussion, the Company has scheduled an additional Type C meeting with the FDA to review the totality of data

9

collected to date, including dystrophin and other mechanistic data as well as additional analyses that could support the benefit of Translarna.

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 the BLA shortly after its pre-BLA meeting that is scheduled for December 2023, pending the outcome of such meeting.

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”). The Company began to make commercial sales of Tegsedi for the treatment of hATTR amyloidosis in Brazil in the second quarter of 2022 and it continues to make Tegsedi available in certain other countries within Latin America and the Caribbean through early access programs (“EAP Programs”). In August 2021, ANVISA, the Brazilian health regulatory authority, approved Waylivra as the first treatment for familial chylomicronemia syndrome (“FCS”) in Brazil and the Company began to make commercial sales of Waylivra in Brazil in the third quarter of 2022 while continuing to make Waylivra available in certain other countries within Latin America and the Caribbean through EAP Programs. 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 also received marketing authorization for the treatment of SMA in Brazil in October 2020 and Japan in June 2021. 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. 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 Company expects the next data update from the Phase 2 study of PTC518 for the treatment of HD in the first half of 2024. In the Phase 2 study, enrollment outside of the United States remains active and ongoing while enrollment within the United States is paused as the FDA requested additional data to allow the Phase 2 study to proceed. The Company had a Type A meeting with the FDA to review the clinical safety data needed to enable resumption of enrollment in the United States. At that meeting, the FDA stated that the existing three-month safety data could support 12-week dosing and that six months of clinical safety data could support dosing in the 12-month Phase 2 study.

The most advanced molecule in the Company’s metabolic platform is sepiapterin, 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. 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

10

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. PTC expects to submit an NDA to the FDA for sepiapterin for the treatment of PKU by the end of 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 review process of the NDA. Additionally, PTC expects to submit a marketing authorization application (“MAA”) to the EMA for sepiapterin for the treatment of PKU in the EEA in the first half of 2024.

The Company’s Bio-e 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 Bio-e platform are vatiquinone and utreloxastat. The Company announced topline results from a