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Document and Entity Information - shares	9 Months Ended
Document and Entity Information - shares	Sep. 30, 2014	Nov. 05, 2014
Document and Entity Information
Entity Registrant Name	PTC THERAPEUTICS, INC.
Entity Central Index Key	0001070081
Document Type	10-Q
Document Period End Date	Sep. 30, 2014
Amendment Flag	false
Current Fiscal Year End Date	--12-31
Entity Current Reporting Status	Yes
Entity Filer Category	Non-accelerated Filer
Entity Common Stock, Shares Outstanding		33,602,541
Document Fiscal Year Focus	2014
Document Fiscal Period Focus	Q3

Balance sheets - USD ($) $ in Thousands	Sep. 30, 2014	Dec. 31, 2013
Current assets:
Cash and cash equivalents	$ 52,873	$ 15,414
Marketable securities	156,516	127,053
Prepaid expenses and other current assets	2,831	1,599
Receivables, net	1,296	958
Total current assets	213,516	145,024
Fixed assets, net	6,193	6,730
Deposits and other assets	1,086	149
Total assets	220,795	151,903
Current liabilities:
Accounts payable and accrued expenses	16,493	12,207
Current portion of long-term debt		49
Deferred revenue	769	878
Total current liabilities	17,262	13,134
Other long-term liabilities	2,237	2,227
Total liabilities	19,499	15,361
Stockholders' equity:
Preferred stock, $0.001 par value. Undesignated 5,000,000 shares; issued and outstanding 0 shares at September 30, 2014 and December 31, 2013
Common stock, $0.001 par value. Authorized 125,000,000 shares; issued and outstanding 29,373,327 shares at September 30, 2014. Authorized 125,000,000 shares; issued and outstanding 23,803,282 shares at December 31, 2013	30	24
Additional paid-in capital	596,617	465,246
Accumulated other comprehensive (loss) income	(69)	70
Accumulated deficit	(395,282)	(328,798)
Total stockholders' equity	201,296	136,542
Total liabilities and stockholders' equity	$ 220,795	$ 151,903

Balance sheets (Parenthetical) - $ / shares	Sep. 30, 2014	Dec. 31, 2013
Balance sheets
Preferred stock, par value (in dollars per share)	$ 0.001	$ 0.001
Preferred stock, undesignated shares	5,000,000	5,000,000
Preferred stock, issued shares	0	0
Preferred stock, outstanding shares	0	0
Common stock, par value (in dollars per share)	$ 0.001	$ 0.001
Common stock, authorized shares	125,000,000	125,000,000
Common stock, issued shares	29,373,327	23,803,282
Common stock, outstanding shares	29,373,327	23,803,282

Statements of operations - USD ($) $ in Thousands	3 Months Ended		9 Months Ended
Statements of operations - USD ($) $ in Thousands	Sep. 30, 2014	Sep. 30, 2013	Sep. 30, 2014	Sep. 30, 2013
Revenues:
Net product sales	$ 81		$ 81
Collaboration revenue	716	$ 15,455	11,280	$ 27,395
Grant revenue	897	834	1,226	2,890
Total revenues	1,694	16,289	12,587	30,285
Operating expenses:
Research and development	18,765	13,886	52,967	39,855
General and administrative	10,530	6,679	26,803	17,735
Total operating expenses	29,295	20,565	79,770	57,590
Loss from operations	(27,601)	(4,276)	(67,183)	(27,305)
Interest income (expense), net	354	27	774	(6,250)
Loss on extinguishment of debt		(130)		(130)
Other income (expense), net	(35)	(37)	(75)	(3)
Net loss	(27,282)	(4,416)	(66,484)	(33,688)
Deemed dividend				(18,249)
Gain on exchange of convertible preferred stock in connection with recapitalization				3,391
Net loss attributable to common stockholders	$ (27,282)	$ (4,416)	$ (66,484)	$ (48,546)
Weighted-average shares outstanding:
Basic and diluted (in shares)	29,351,693	23,803,282	28,441,827	8,995,167
Net loss per share applicable to common stockholders-basic and diluted (in dollars per share)	$ (0.93)	$ (0.19)	$ (2.34)	$ (5.40)

Statements of cash flows - USD ($) $ in Thousands	9 Months Ended
Statements of cash flows - USD ($) $ in Thousands	Sep. 30, 2014	Sep. 30, 2013
Cash flows from operating activities
Net loss	$ (66,484)	$ (33,688)
Adjustments to reconcile net loss to net cash used in operating activities:
Depreciation	1,693	1,811
Change in valuation of warrant liability	78	3
Non-cash interest expense		6,049
Loss on extinguishment of debt		130
Amortization of premiums on investments	1,225
Share-based compensation expense	12,605	5,142
Changes in operating assets and liabilities:
Prepaid expenses and other current assets	(1,232)	(1,288)
Grant and collaboration receivables	(338)	230
Deposits and other assets	(937)	34
Accounts payable and accrued expenses	4,286	3,181
Other long-term liabilities	(68)	(262)
Deferred revenue	(109)	(13,801)
Net cash used in operating activities	(49,281)	(32,459)
Cash flows from investing activities
Purchases of fixed assets	(1,156)	(474)
Purchases of marketable securities	(132,602)	(139,731)
Sale & redemption of marketable securities	101,775	6,508
Net cash used in investing activities	(31,983)	(133,697)
Cash flows from financing activities
Payments on long-term debt	(49)	(4,960)
Net proceeds from sale of Series Four convertible preferred stock		60,785
Proceeds from exercise of options	389
Net proceeds from public offerings	118,383	131,650
Net cash provided by financing activities	118,723	187,475
Net increase in cash and cash equivalents	37,459	21,319
Cash and cash equivalents, beginning of period	15,414	2,726
Cash and cash equivalents, end of period	52,873	24,045
Supplemental disclosure of cash information
Cash paid for interest	1	366
Supplemental disclosures of non-cash information related to investing and financing activities
Change in unrealized gain (loss) on marketable securities	$ (139)	(40)
Change in carry value of preferred securities resulting from recapitalization		$ 3,391

Summary of significant accounting policies	9 Months Ended
Summary of significant accounting policies	Sep. 30, 2014
Summary of significant accounting policies
Summary of significant accounting policies	2.Summary of significant accounting policies The Company’s complete listing of significant accounting policies are described in note 2 of the notes to the Company’s audited financial statements as of December 31, 2013 included in the Company’s Annual Report on Form 10-K filed with the Securities and Exchange Commission (SEC) on March 6, 2014 (2013 Form 10-K). There have been no changes to our accounting policies during the quarter. Basis of Presentation The accompanying financial information as of September 30, 2014 and for the three and nine months ended September 30, 2014 and 2013 has been prepared by the Company, without audit, pursuant to the rules and regulations of the SEC. Certain information and footnote disclosures normally included in financial statements prepared in accordance with generally accepted accounting principles in the United States (GAAP) have been condensed or omitted pursuant to such rules and regulations. These interim financial statements should be read in conjunction with the Company’s audited financial statements as of December 31, 2013 and notes thereto included in the 2013 Form 10-K. In the opinion of management, the unaudited financial information as of September 30, 2014 and for the three and nine months ended September 30, 2014 and 2013 reflects all adjustments, which are normal recurring adjustments, necessary to present a fair statement of financial position, results of operations and cash flows. The results of operations for the three and nine month periods ended September 30, 2014 are not necessarily indicative of the results to be expected for the year ended December 31, 2014 or for any other interim period or for any other future year. Use of estimates The preparation of financial statements in conformity with GAAP requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities and disclosure of contingent assets and liabilities at the date of the financial statements, and the reported amounts of revenues and expenses during the reporting period. Actual results could differ from those estimates. Recently issued accounting standard In May 2014, the Financial Accounting Standards Board (FASB) issued Accounting Standards Update (ASU) No. 2014-09, Revenue from Contracts with Customers. ASU 2014-09 will eliminate transaction- and industry-specific revenue recognition guidance under current GAAP and replace it with a principle-based approach for determining revenue recognition. ASU 2014-09 will require that companies recognize revenue based on the value of transferred goods or services as they occur in the contract. The ASU also will require additional disclosure about the nature, amount, timing and uncertainty of revenue and cash flows arising from customer contracts, including significant judgments and changes in judgments and assets recognized from costs incurred to obtain or fulfill a contract. ASU 2014-09 is effective for annual reporting periods beginning after December 15, 2016. Early application is not permitted. Entities can transition to the standard either retrospectively or as a cumulative-effect adjustment as of the date of adoption. Presently, the Company is assessing what effect the adoption of ASU 2014-09 will have on its financial statements and accompanying notes. In August 2014, the FASB issued ASU 2014-15, “Presentation of Financial Statements—Going Concern—Disclosures of Uncertainties about an entity’s Ability to Continue as a Going Concern.” ASU 2014-15 provides new guidance related to management’s responsibility to evaluate whether there is substantial doubt about an entity’s ability to continue as a going concern by incorporating and expanding upon certain principles that are currently in U.S. auditing standards and to provide related footnote disclosures. This new guidance is effective for the annual period ending after December 15, 2016, and for annual periods and interim periods thereafter. The requirements of ASU 2014-15 are not expected to have a significant impact on the Condensed Consolidated Financial Statements. Revenue Recognition To date, PTC’s net product sales have consisted solely of sales of Translarna for the treatment of DMD caused by nonsense mutations. The Company applies the revenue recognition guidance in accordance with Financial Accounting Standards Board (“FASB”) Accounting Standards Codification (“ASC”) Subtopic 605-15, Revenue Recognition—Products. The Company recognizes revenue from product sales when there is persuasive evidence that an arrangement exists, title to product and associated risk of loss has passed to the customer, the price is fixed or determinable, collectability is reasonably assured and the Company has no further performance obligations. The Company has recorded revenue on sales where Translarna is available on a reimbursed early access program and typically paid for by a government authority or institution. The Company generally recognizes revenue for these named patient or cohort early access programs once the product is shipped through to the government authority or institution on a cash basis if all other revenue recognition criteria have been met. The Company records revenue net of estimated discounts and rebates. Allowances are recorded as a reduction of revenue at the time revenues from product sales are recognized. Allowances for government rebates and discounts are established based on the actual payer information, which is known at the time of delivery, and the government-mandated discounts applicable to government-funded programs. These allowances are adjusted to reflect known changes in the factors that may impact such allowances in the quarter those changes are known.

Fair value of financial instruments and marketable securities

9 Months Ended

Sep. 30, 2014

Fair value of financial instruments and marketable securities

Fair value of financial instruments and marketable securities

3.Fair value of financial instruments and marketable securities

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).

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.

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).

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 are reflected in the accompanying financial statements at fair value. The carrying amount of grant and collaboration receivables, accounts payable and accrued expenses, and debt approximates fair value due to the short-term nature of those instruments.

Fair value of certain marketable 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 investments, the Company used the fair value as determined by its investment advisors using observable inputs other than quoted prices.

The Company reviews its investments on a periodic basis for other-than-temporary impairments. This review is subjective, as it requires management to evaluate whether an event or change in circumstances has occurred in that period that may have a significant adverse effect on the fair value of the investment.

The following represents the fair value using the hierarchy described in Note 3 for the Company’s financial assets and liabilities that are required to be measured at fair value on a recurring basis as of September 30, 2014 and December 31, 2013:

	September 30, 2014
	Total		Quoted prices in active markets for identical assets (level 1)		Significant other observable inputs (level 2)		Significant unobservable inputs (level 3)
Marketable securities	$	156,516	$	—	$	156,516	$	—
Warrant liability	136		—		—		136

	December 31, 2013
	Total		Quoted prices in active markets for identical assets (level 1)		Significant other observable inputs (level 2)		Significant unobservable inputs (level 3)
Marketable securities	$	127,053	$	—	$	127,053	$	—
Warrant Liability	58		—		—		58

The following is a summary of marketable securities accounted for as available-for-sale securities at September 30, 2014 and December 31, 2013:

	September 30, 2014
	Amortized		Gross Unrealized					Fair
	Cost		Gains		Losses			Value
Corporate debt securities	$	122,561	$	112	$	(156	)	$	122,517
Government obligations	34,024		8		(33		)	33,999
	$	156,585	$	120	$	(189	)	$	156,516

	December 31, 2013
	Amortized		Gross Unrealized					Fair
	Cost		Gains		Losses			Value
Commercial paper	$	14,993	$	5	$	—		$	14,998
Corporate debt securities	111,989		97		(31		)	112,055
	$	126,982	$	102	$	(31	)	$	127,053

At September 30, 2014 and December 31, 2013, the Company held securities with an unrealized loss position that were not considered to be other-than-temporarily impaired as the Company has the ability to hold such investments until recovery of their fair value.

Marketable securities on the balance sheet at September 30, 2014 and December 31, 2013 mature as follows:

	September 30, 2014
	Less Than 12 Months		More Than 12 Months
Corporate debt securities	$	65,860	$	56,657
Government obligations	—		33,999
Total Marketable securities	$	65,860	$	90,656

	December 31, 2013
	Less Than 12 Months		More Than 12 Months
Commercial paper	$	14,998	$	—
Corporate debt securities	54,159		57,896
Total Marketable securities	$	69,157	$	57,896

Level 3 valuation

The warrant liability is classified in Other long-term liabilities on the Company’s balance sheet. The warrant liability is marked-to-market each reporting period with the change in fair value recorded as a gain or loss within Other income (expense), net on the Company’s statement of operations until the warrants are exercised, expire or other facts and circumstances lead the warrant liability to be reclassified as an equity instrument. The fair value of the warrant liability is determined at each reporting period by utilizing the Black-Scholes option pricing model.

The table presented below is a summary of changes in the fair value of the Company’s Level 3 valuation for warrant liability for the period ended September 30, 2014:

	Level 3 assets
Beginning balance as of December 31, 2013	$	58
Change in fair value of warrant liability	78
Ending balance as of September 30, 2014	$	136

Fair value of the warrant liability is estimated using an option-pricing model, which includes variables such as the expected volatility based on guideline public companies, the stock fair value, and the estimated time to a liquidity event. The significant assumptions used in preparing the option pricing model for valuing the Company’s warrants as of September 30, 2014 include (i) volatility (64%—72%), (ii) risk free interest rate (0.83%—1.78%), (iii) strike price ($128.00-$2,520), (iv) fair value of common stock ($44.01), and (v) expected life (2.71—4.98 years). The significant assumptions used in preparing the option pricing model for valuing the Company’s warrants as of December 31, 2013 include (i) volatility (61-89%), (ii) risk free interest rate (0.07%—2.10%), (iii) strike price ($128.00—$2,520.00), (iv) fair value of common stock ($16.97), and (v) expected life (0.30—5.70 years). See Note 6 for a description of the warrants issued in connection with the convertible notes.

- 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

Other comprehensive income (loss) and accumulated other comprehensive items

9 Months Ended

Sep. 30, 2014

Other comprehensive income (loss) and accumulated other comprehensive items

Other comprehensive income (loss) and accumulated other comprehensive items

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

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

The following tables summarize other comprehensive income (loss) and the changes in accumulated other comprehensive items for the three and nine months ended September 30, 2014:

	Unrealized Gains/ (Losses) On Marketable Securities			Total Accumulated Other Comprehensive Items

Balance at June 30, 2014	$	90		$	90
Other comprehensive income/(loss) before reclassifications	(159		)	(159		)
Amounts reclassified from other comprehensive items	—			—
Other comprehensive income/(loss)	(159		)	(159		)
Balance at September 30, 2014	$	(69	)	$	(69	)

	Unrealized Gains/ (Losses) On Marketable Securities			Total Accumulated Other Comprehensive Items

Balance at December 31, 2013	$	70		$	70
Other comprehensive income/(loss) before reclassifications	(139		)	(139		)
Amounts reclassified from other comprehensive items	—			—
Other comprehensive income/(loss)	(139		)	(139		)
Balance at September 30, 2014	$	(69	)	$	(69	)

- Definition

The entire disclosure for comprehensive income, which includes, but is not limited to, 1) the amount of income tax expense or benefit allocated to each component of other comprehensive income, including reclassification adjustments, 2) the reclassification adjustments for each classification of other comprehensive income and 3) the ending accumulated balances for each component of comprehensive income.

+ References

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-Name Accounting Standards Codification
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+ Details

Capital structure

9 Months Ended

Sep. 30, 2014

Capital structure

Capital structure

6.Capital structure

2013 Recapitalization

During January and February of 2013, the Company entered into a “bridge” financing arrangement with certain existing investors providing for the issuance by the Company of an aggregate of $6 million of convertible promissory notes and warrants to purchase 2,527,675 shares of Series One convertible preferred stock (Series One) and Series Two convertible preferred stock (Series Two). The warrants have a per share exercise price of $0.01, and as such, they are referred to as “penny warrants”. This bridge financing was closed in anticipation of the March 2013 Series Four financing event, which the Company refers to as the “2013 recapitalization”.

The Company allocated the proceeds of the convertible promissory notes between debt and warrant liability. Since the value of the warrants exceeded the proceeds from the convertible notes issued to existing investors, the value of the warrant in excess of the proceeds is considered a deemed dividend and reflected as an equity transaction in the financial statements. The Company recorded $6.0 million to interest expense related to the debt discount associated with the convertible debt during the quarter ended March 31, 2013.

On March 7, 2013, the Company closed a private placement of a new series of convertible preferred stock that resulted in the 2013 recapitalization. In this private placement, the Company issued and sold an aggregate of 4,497,035 shares of its Series Four senior preferred stock (Series Four) for an aggregate purchase price of approximately $54.0 million. Including the $6.0 million raised with the bridge financing, total gross proceeds raised during the quarter ended March 31, 2013 was approximately $60.0 million. In addition, the Company issued an aggregate of 502,919 shares of Series Four upon the share settlement of the convertible promissory notes described above that were issued in January and February 2013.

In connection with this private placement, the Company effected a one-for-120 reverse stock split of its common stock and an exchange of outstanding shares of Series One, Series Two and Series Three convertible preferred stock (Series Three) into an aggregate of 6,700,487 shares of a new series of Series Five junior preferred stock (Series Five). In addition, the Company issued an aggregate of 2,527,675 shares of Series One and Series Two upon the exercise of the warrants issued in connection with the bridge loan that were immediately exchanged for 2,095,515 shares of Series Five during the 2013 recapitalization.

The Company accounted for the 2013 recapitalization as an extinguishment of its Series One, Series Two and Series Three convertible preferred stock and recorded the Series Five shares at their fair value as of the recapitalization date. In accordance with authoritative accounting guidance, the Company recorded a gain attributable to the common stockholders on the extinguishment of the Series One, Series Two and Series Three. The gain of approximately $3.4 million represents the excess of the Series One, Series Two and Series Three over the fair value of the shares Series Five issued in connection with the recapitalization.

Initial Public Offering

In June 2013, the Company closed the initial public offering of its common stock pursuant to a registration statement on Form S-1, as amended. The Company issued and sold an aggregate of 9,627,800 shares of common stock under the registration statement at a public offering price of $15.00 per share, including 1,255,800 shares pursuant to the exercise by the underwriters of an over-allotment option. The Company received net proceeds from the initial public offering of approximately $131.6 million, after deducting underwriting discounts and commissions and other offering expenses payable by the Company.

Upon closing the initial public offering, all outstanding shares of the Series Four and Series Five were converted into 14,170,956 shares of common stock.

Follow-On Offering

In February 2014, the Company closed a follow-on public offering of its common stock pursuant to a registration statement on Form S-1, as amended. The Company issued and sold an aggregate of 5,163,265 shares of common stock under the registration statement at a public offering price of $24.50 per share, including 673,469 shares pursuant to the exercise by the underwriters of an over-allotment option. The Company received net proceeds from the follow –on public offering of approximately $118.4 million, after deducting underwriting discounts and commissions and other offering expenses payable by the Company.

Warrants

All of the Company’s outstanding warrants were classified as liabilities as of September 30, 2014 and December 31, 2013 because they contained non-standard antidilution provisions.

The following is a summary of the Company’s outstanding warrants as of September 30, 2014:

	Warrant shares	Exercise price		Expiration
Common stock	6,250	$	128.00	2017
Common stock	7,030	$	128.00	2019-2020
Common stock	130	$	2,520.00	2019

The following is a summary of the Company’s outstanding warrants as of December 31, 2013:

	Warrant shares	Exercise price		Expiration
Common stock	1,428	$	128.00	2014
Common stock	6,250	$	128.00	2017
Common stock	7,030	$	128.00	2019 and 2020
Common stock	452	$	2,520.00	2014

In connection with the 2013 recapitalization, all of the Series Two outstanding warrants became warrants to purchase Series Five. In connection with the Company’s initial public offering all of the Series Five outstanding warrants became warrants to purchase common stock.

- Definition

The entire disclosure for shareholders' equity comprised of portions attributable to the parent entity and noncontrolling interest, including other comprehensive income. Includes, but is not limited to, balances of common stock, preferred stock, additional paid-in capital, other capital and retained earnings, accumulated balance for each classification of other comprehensive income and amount of comprehensive income.

+ References

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Reference 11: http://www.xbrl.org/2003/role/presentationRef
-Publisher FASB
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-Publisher FASB
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Reference 14: http://www.xbrl.org/2003/role/presentationRef
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Reference 15: http://www.xbrl.org/2003/role/presentationRef
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Reference 16: http://www.xbrl.org/2003/role/presentationRef
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Reference 17: http://www.xbrl.org/2003/role/presentationRef
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Reference 18: http://www.xbrl.org/2003/role/presentationRef
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Reference 19: http://www.xbrl.org/2003/role/presentationRef
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+ Details

Net loss per share

9 Months Ended

Sep. 30, 2014

Net loss per share

Net loss per share

7.Net loss per share

Basic earnings per share is computed by dividing net loss available to common stockholders by the weighted-average number of common shares outstanding. Diluted earnings per share is computed by dividing net income (loss) available to common stockholders by the weighted-average number of common shares plus the effect of dilutive potential common shares outstanding during the period.

The following tables set forth the computation of basic and diluted net income (loss) per share for common stockholders:

	Three months ended September 30,
	2014			2013
Numerator
Net loss attributable to common stockholders	$	(27,282	)	$	(4,416	)
Denominator
Denominator for basic and diluted net loss per share	29,351,693			23,803,282
Net loss per share:
Basic and diluted	$	(0.93	)*	$	(0.19	)*

In the three months ended September 30, 2014 and 2013, the Company experienced a net loss and therefore did not report any dilutive share impact.

	Nine months ended September 30,
	2014			2013
Numerator
Net loss	$	(66,484	)	$	(33,688	)
Deemed dividend	—			(18,249		)
Gain on exchange of convertible preferred stock in connection with recapitalization	—			3,391
Net loss attributable to common stockholders	$	(66,484	)	$	(48,546	)
Denominator
Denominator for basic and diluted net loss per share	28,441,827			8,995,167
Net loss per share:
Basic and diluted	$	(2.34	)*	$	(5.40	)*

*In the nine months ended September 30, 2014 and 2013, 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 September 30,
	2014	2013

Stock Options	3,443,778	1,987,860
Unvested restricted stock	729,320	1,113,807
Total	4,173,098	3,101,667

Stock option plan

9 Months Ended

Sep. 30, 2014

Stock option plan

Stock option plan

8.Stock award plan

On March 5, 2013, the Company’s Board of Directors approved the 2013 Stock Incentive Plan, which provides for the granting of stock option awards, stock appreciation rights, restricted stock, restricted stock units and other stock-based awards in the aggregate of 739,937 shares of common stock. On March 5, 2013, the Board approved a grant of 735,324 shares of restricted stock and 4,613 stock options. There are no additional shares available for issuance under this plan.

In May 2013, the Company’s Board of Directors and stockholders increased by 2,500,000 the number of shares authorized under the 2009 Stock Incentive Plan, which provides for the granting of stock option awards, restricted stock awards, and other stock-based and cash-based awards.

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. The number of shares of common stock reserved for issuance under the 2013 Long Term Incentive Plan is the sum of (1) 122,296 shares of common stock available for issuance under the Company’s 2009 Equity and Long Term Incentive Plan and 2013 Stock Incentive Plan, (2) the number of shares (up to 3,040,444 shares) equal to the sum of the number of shares of common stock subject to outstanding awards under the Company’s 1998 Employee, Director and Consultant Stock Option Plan and 2013 Stock Incentive Plan that expire, terminate or are otherwise surrendered, cancelled, forfeited or repurchased by the Company at their original issuance price pursuant to a contractual repurchase right plus (3) an annual increase, to be added on the first day of each fiscal year until the expiration of the 2013 Long Term Incentive Plan, equal to the lowest of 2,500,000 shares of common stock, 4% of the number of shares of common stock outstanding on the first day of the fiscal year and an amount determined by the Company’s Board of Directors.

From January 1, 2014 through September 30, 2014, the Company issued a total of 1,469,496 stock options to various employees. Of those, 448,800 were inducement grants for non-statutory stock options. The inducement grant awards were made pursuant to the NASDAQ inducement grant exception as a material component of our new hires’ employment compensation.

A summary of stock option activity is as follows:

	Number of options		Weighted- average exercise price		Weighted- average remaining contractual term	Aggregate intrinsic value
						(in thousands)
Outstanding at December 31, 2013	2,095,592		$	20.24
Granted	1,469,496		$	29.57
Exercised	(35,500	)	$	10.96
Forfeited	(85,775	)	$	11.78
Expired	(35	)	$	646.66
Outstanding at September 30, 2014	3,443,778		$	24.52	8.9 yrs	$	84,456
Vested or Expected to vest at September 30, 2014	3,217,812		$	23.01	9.0 yrs	$	79,675
Exercisable at September 30, 2014	722,835		$	36.23	8.3 yrs	$	22,463

The fair value of grants made in the period ended September 30, 2014 was contemporaneously estimated on the date of grant using the following assumptions:

		Nine months ended September 30, 2014
Risk-free interest rate		0.11%—2.04%
Expected volatility		79%—91%
Expected term		5.5 years—6.25 years

The Company assumed no expected dividends for all grants. The weighted average grant date fair value of options granted during the nine month period ended September 30, 2014 was $21.70 per share.

The Company uses the “simplified method” to determine the expected term of options. Under this method, the expected term represents the average of the vesting period and the contractual term. The expected volatility of share options was estimated based on a historical volatility analysis of peers that were similar to the Company with respect to industry, stage of life cycle, size, and financial leverage. 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.

Restricted Stock Awards—Restricted stock awards are granted subject to certain restrictions, including in some cases service or time 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:

	Restricted Stock
	Number of Shares		Weighted Average Grant Date Fair Value
January 1, 2014	1,110,226		$	10.68
Granted	—		—
Vested	(371,280	)	$	10.60
Forfeited	(9,626	)	$	10.66
Unvested at September 30, 2014	729,320		$	10.72

The Company recorded share-based compensation expense in the statement of operations as follows:

	Three Months Ended September 30,				Nine Months Ended September 30,
	2014		2013		2014		2013

Research and development	$	2,363	$	677	$	6,517	$	1,378

General and administrative	2,258		1,963		6,088		3,764

Total	$	4,621	$	2,640	$	12,605	$	5,142

As of September 30, 2014 there was approximately $38.7 million of total unrecognized compensation cost related to unvested share-based compensation arrangements granted under the 1998, 2009 and 2013 Plans and the inducement grants pursuant to the NASDAQ inducement grant exception. This cost is expected to be recognized as share-based compensation expense over the weighted average remaining service period of approximately 2.70 years.

Summary of significant accounting policies (Policies)	9 Months Ended
Summary of significant accounting policies (Policies)	Sep. 30, 2014
Summary of significant accounting policies
Basis of Presentation	Basis of Presentation The accompanying financial information as of September 30, 2014 and for the three and nine months ended September 30, 2014 and 2013 has been prepared by the Company, without audit, pursuant to the rules and regulations of the SEC. Certain information and footnote disclosures normally included in financial statements prepared in accordance with generally accepted accounting principles in the United States (GAAP) have been condensed or omitted pursuant to such rules and regulations. These interim financial statements should be read in conjunction with the Company’s audited financial statements as of December 31, 2013 and notes thereto included in the 2013 Form 10-K. In the opinion of management, the unaudited financial information as of September 30, 2014 and for the three and nine months ended September 30, 2014 and 2013 reflects all adjustments, which are normal recurring adjustments, necessary to present a fair statement of financial position, results of operations and cash flows. The results of operations for the three and nine month periods ended September 30, 2014 are not necessarily indicative of the results to be expected for the year ended December 31, 2014 or for any other interim period or for any other future year.
Use of estimates	Use of estimates The preparation of financial statements in conformity with GAAP requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities and disclosure of contingent assets and liabilities at the date of the financial statements, and the reported amounts of revenues and expenses during the reporting period. Actual results could differ from those estimates.
Recently issued accounting standard	Recently issued accounting standard In May 2014, the Financial Accounting Standards Board (FASB) issued Accounting Standards Update (ASU) No. 2014-09, Revenue from Contracts with Customers. ASU 2014-09 will eliminate transaction- and industry-specific revenue recognition guidance under current GAAP and replace it with a principle-based approach for determining revenue recognition. ASU 2014-09 will require that companies recognize revenue based on the value of transferred goods or services as they occur in the contract. The ASU also will require additional disclosure about the nature, amount, timing and uncertainty of revenue and cash flows arising from customer contracts, including significant judgments and changes in judgments and assets recognized from costs incurred to obtain or fulfill a contract. ASU 2014-09 is effective for annual reporting periods beginning after December 15, 2016. Early application is not permitted. Entities can transition to the standard either retrospectively or as a cumulative-effect adjustment as of the date of adoption. Presently, the Company is assessing what effect the adoption of ASU 2014-09 will have on its financial statements and accompanying notes. In August 2014, the FASB issued ASU 2014-15, “Presentation of Financial Statements—Going Concern—Disclosures of Uncertainties about an entity’s Ability to Continue as a Going Concern.” ASU 2014-15 provides new guidance related to management’s responsibility to evaluate whether there is substantial doubt about an entity’s ability to continue as a going concern by incorporating and expanding upon certain principles that are currently in U.S. auditing standards and to provide related footnote disclosures. This new guidance is effective for the annual period ending after December 15, 2016, and for annual periods and interim periods thereafter. The requirements of ASU 2014-15 are not expected to have a significant impact on the Condensed Consolidated Financial Statements.
Revenue recognition	Revenue Recognition To date, PTC’s net product sales have consisted solely of sales of Translarna for the treatment of DMD caused by nonsense mutations. The Company applies the revenue recognition guidance in accordance with Financial Accounting Standards Board (“FASB”) Accounting Standards Codification (“ASC”) Subtopic 605-15, Revenue Recognition—Products. The Company recognizes revenue from product sales when there is persuasive evidence that an arrangement exists, title to product and associated risk of loss has passed to the customer, the price is fixed or determinable, collectability is reasonably assured and the Company has no further performance obligations. The Company has recorded revenue on sales where Translarna is available on a reimbursed early access program and typically paid for by a government authority or institution. The Company generally recognizes revenue for these named patient or cohort early access programs once the product is shipped through to the government authority or institution on a cash basis if all other revenue recognition criteria have been met. The Company records revenue net of estimated discounts and rebates. Allowances are recorded as a reduction of revenue at the time revenues from product sales are recognized. Allowances for government rebates and discounts are established based on the actual payer information, which is known at the time of delivery, and the government-mandated discounts applicable to government-funded programs. These allowances are adjusted to reflect known changes in the factors that may impact such allowances in the quarter those changes are known.

- Definition

Disclosure of accounting policy for revenue recognition. If the entity has different policies for different types of revenue transactions, the policy for each material type of transaction is generally disclosed. If a sales transaction has multiple element arrangements (for example, delivery of multiple products, services or the rights to use assets) the disclosure may indicate the accounting policy for each unit of accounting as well as how units of accounting are determined and valued. The disclosure may encompass important judgment as to appropriateness of principles related to recognition of revenue. The disclosure also may indicate the entity's treatment of any unearned or deferred revenue that arises from the transaction.

+ References + Details

Fair value of financial instruments and marketable securities (Tables)

9 Months Ended

Sep. 30, 2014

Fair value of financial instruments and marketable securities

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

	September 30, 2014
	Total		Quoted prices in active markets for identical assets (level 1)		Significant other observable inputs (level 2)		Significant unobservable inputs (level 3)
Marketable securities	$	156,516	$	—	$	156,516	$	—
Warrant liability	136		—		—		136

	December 31, 2013
	Total		Quoted prices in active markets for identical assets (level 1)		Significant other observable inputs (level 2)		Significant unobservable inputs (level 3)
Marketable securities	$	127,053	$	—	$	127,053	$	—
Warrant Liability	58		—		—		58

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

	September 30, 2014
	Amortized		Gross Unrealized					Fair
	Cost		Gains		Losses			Value
Corporate debt securities	$	122,561	$	112	$	(156	)	$	122,517
Government obligations	34,024		8		(33		)	33,999
	$	156,585	$	120	$	(189	)	$	156,516

	December 31, 2013
	Amortized		Gross Unrealized					Fair
	Cost		Gains		Losses			Value
Commercial paper	$	14,993	$	5	$	—		$	14,998
Corporate debt securities	111,989		97		(31		)	112,055
	$	126,982	$	102	$	(31	)	$	127,053

Schedule of marketable securities on the balance sheet

	September 30, 2014
	Less Than 12 Months		More Than 12 Months
Corporate debt securities	$	65,860	$	56,657
Government obligations	—		33,999
Total Marketable securities	$	65,860	$	90,656

	December 31, 2013
	Less Than 12 Months		More Than 12 Months
Commercial paper	$	14,998	$	—
Corporate debt securities	54,159		57,896
Total Marketable securities	$	69,157	$	57,896

Summary of changes in the fair value of the Company's Level 3 valuation for warrant liability

	Level 3 assets
Beginning balance as of December 31, 2013	$	58
Change in fair value of warrant liability	78
Ending balance as of September 30, 2014	$	136

Capital structure (Tables)

9 Months Ended

Sep. 30, 2014

Capital structure

Summary of the Company's outstanding warrants

The following is a summary of the Company’s outstanding warrants as of September 30, 2014:

	Warrant shares	Exercise price		Expiration
Common stock	6,250	$	128.00	2017
Common stock	7,030	$	128.00	2019-2020
Common stock	130	$	2,520.00	2019

The following is a summary of the Company’s outstanding warrants as of December 31, 2013:

	Warrant shares	Exercise price		Expiration
Common stock	1,428	$	128.00	2014
Common stock	6,250	$	128.00	2017
Common stock	7,030	$	128.00	2019 and 2020
Common stock	452	$	2,520.00	2014

- Definition

Tabular disclosure of warrants or rights issued. Warrants and rights outstanding are derivative securities that give the holder the right to purchase securities (usually equity) from the issuer at a specific price within a certain time frame. Warrants are often included in a new debt issue to entice investors by a higher return potential. The main difference between warrants and call options is that warrants are issued and guaranteed by the company, whereas options are exchange instruments and are not issued by the company. Also, the lifetime of a warrant is often measured in years, while the lifetime of a typical option is measured in months. Disclose the title of issue of securities called for by warrants and rights outstanding, the aggregate amount of securities called for by warrants and rights outstanding, the date from which the warrants or rights are exercisable, and the price at which the warrant or right is exercisable.

+ References + Details

Net loss per share (Tables)

9 Months Ended

Sep. 30, 2014

Net loss per share

Schedule of computation of basic and diluted net loss per share for common stockholders

	Three months ended September 30,
	2014			2013
Numerator
Net loss attributable to common stockholders	$	(27,282	)	$	(4,416	)
Denominator
Denominator for basic and diluted net loss per share	29,351,693			23,803,282
Net loss per share:
Basic and diluted	$	(0.93	)*	$	(0.19	)*

In the three months ended September 30, 2014 and 2013, the Company experienced a net loss and therefore did not report any dilutive share impact.

	Nine months ended September 30,
	2014			2013
Numerator
Net loss	$	(66,484	)	$	(33,688	)
Deemed dividend	—			(18,249		)
Gain on exchange of convertible preferred stock in connection with recapitalization	—			3,391
Net loss attributable to common stockholders	$	(66,484	)	$	(48,546	)
Denominator
Denominator for basic and diluted net loss per share	28,441,827			8,995,167
Net loss per share:
Basic and diluted	$	(2.34	)*	$	(5.40	)*

*In the nine months ended September 30, 2014 and 2013, 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 September 30,
	2014	2013

Stock Options	3,443,778	1,987,860
Unvested restricted stock	729,320	1,113,807
Total	4,173,098	3,101,667

Stock option plan (Tables)

9 Months Ended

Sep. 30, 2014

Stock option plan

Summary of stock option activity

	Number of options		Weighted- average exercise price		Weighted- average remaining contractual term	Aggregate intrinsic value
						(in thousands)
Outstanding at December 31, 2013	2,095,592		$	20.24
Granted	1,469,496		$	29.57
Exercised	(35,500	)	$	10.96
Forfeited	(85,775	)	$	11.78
Expired	(35	)	$	646.66
Outstanding at September 30, 2014	3,443,778		$	24.52	8.9 yrs	$	84,456
Vested or Expected to vest at September 30, 2014	3,217,812		$	23.01	9.0 yrs	$	79,675
Exercisable at September 30, 2014	722,835		$	36.23	8.3 yrs	$	22,463

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

		Nine months ended September 30, 2014
Risk-free interest rate		0.11%—2.04%
Expected volatility		79%—91%
Expected term		5.5 years—6.25 years

Summary of information on the Company's restricted stock

	Restricted Stock
	Number of Shares		Weighted Average Grant Date Fair Value
January 1, 2014	1,110,226		$	10.68
Granted	—		—
Vested	(371,280	)	$	10.60
Forfeited	(9,626	)	$	10.66
Unvested at September 30, 2014	729,320		$	10.72

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

	Three Months Ended September 30,				Nine Months Ended September 30,
	2014		2013		2014		2013

Research and development	$	2,363	$	677	$	6,517	$	1,378

General and administrative	2,258		1,963		6,088		3,764

Total	$	4,621	$	2,640	$	12,605	$	5,142

Fair value of financial instruments and marketable securities (Details 4) - USD ($) $ / shares in Units, $ in Thousands	9 Months Ended	12 Months Ended
	Sep. 30, 2014	Dec. 31, 2013	Jun. 30, 2013
Common stock
Assumption used to estimate the fair value of warrant liability by utilizing the Black-Scholes option-pricing model
Fair value of shares (in dollars per share)			$ 15.00
Warrant
Changes in the fair value of warrant liability
Beginning balance	$ 58
Change in fair value of warrant liability	78
Ending balance	$ 136	$ 58
Warrant \| Minimum
Assumption used to estimate the fair value of warrant liability by utilizing the Black-Scholes option-pricing model
Volatility (as a percent)	64.00%	61.00%
Risk-free interest rate (as a percent)	0.83%	0.07%
Strike price (in dollars per share)	$ 128.00	$ 128.00
Expected life	2 years 8 months 16 days	3 months 18 days
Warrant \| Maximum
Assumption used to estimate the fair value of warrant liability by utilizing the Black-Scholes option-pricing model
Volatility (as a percent)	72.00%	89.00%
Risk-free interest rate (as a percent)	1.78%	2.10%
Strike price (in dollars per share)	$ 2,520.00	$ 2,520.00
Expected life	4 years 11 months 23 days	5 years 8 months 12 days
Warrant \| Common stock
Assumption used to estimate the fair value of warrant liability by utilizing the Black-Scholes option-pricing model
Fair value of shares (in dollars per share)	$ 44.01	$ 16.97

Accounts payable and accrued expenses (Details) - USD ($) $ in Thousands	Sep. 30, 2014	Dec. 31, 2013
Accounts payable and accrued expenses
Employee compensation, benefits, and related accruals	$ 5,729	$ 5,103
Consulting and contracted research	5,709	4,006
Professional fees	3,592	1,294
Accounts payable	518	1,124
Other	945	680
Accounts payable and accrued expenses	$ 16,493	$ 12,207

Capital structure (Details) $ / shares in Units, $ in Thousands			1 Months Ended	3 Months Ended	9 Months Ended
	Feb. 12, 2014 USD ($) $ / shares shares	Mar. 07, 2013 USD ($) shares	Jun. 30, 2013 USD ($) $ / shares shares	Mar. 31, 2013 USD ($)	Sep. 30, 2014 USD ($) shares	Sep. 30, 2013 USD ($)	Dec. 31, 2013 shares	Feb. 28, 2013 USD ($) $ / shares shares
Capital structure
Gross proceeds raised \| $				$ 60,000
Gain on extinguishment of shares of Series One, Two and Three stock \| $						$ 3,391
Net proceeds from public offerings \| $					$ 118,383	$ 131,650
Common stock
Common stock, authorized shares					125,000,000		125,000,000
Series One and Series Two \| Warrant
Capital structure
Warrants issued to purchase shares								2,527,675
Exercise price (in dollars per share) \| $ / shares								$ 0.01
Shares issued and sold		2,527,675
Series Four convertible preferred stock
Capital structure
Shares issued and sold		4,497,035
Aggregate gross proceeds \| $		$ 54,000
Series Five convertible preferred stock
Capital structure
Number of shares issued upon conversion		6,700,487
Series Five convertible preferred stock \| Warrant
Capital structure
Number of shares issued upon conversion		2,095,515
Common stock
Capital structure
Shares issued and sold	5,163,265		9,627,800
Reverse stock split ratio		0.0083
Share price (in dollars per share) \| $ / shares			$ 15.00
Public offering price (in dollars per share) \| $ / shares	$ 24.50
Common stock shares issuable pursuant to the exercise by the underwriters of an over-allotment option			1,255,800
Net proceeds from initial public offering \| $			$ 131,600
Net proceeds from public offerings \| $	$ 118,400
Common stock \| Maximum
Capital structure
Common stock shares issuable pursuant to the exercise by the underwriters of an over-allotment option	673,469
Series Four and Series Five Convertible Preferred Stock
Capital structure
Number of shares issued upon conversion			14,170,956
Convertible promissory notes
Capital structure
Principal amount of convertible promissory notes issued \| $								$ 6,000
Debt discount recorded to interest expense \| $				6,000
Amount raised in bridge financing \| $				$ 6,000
Convertible promissory notes \| Series Four convertible preferred stock
Capital structure
Shares issued upon conversion of convertible promissory notes		502,919

Net loss per share (Details) - USD ($) $ / shares in Units, $ in Thousands	3 Months Ended		9 Months Ended
	Sep. 30, 2014	Sep. 30, 2013	Sep. 30, 2014	Sep. 30, 2013
Numerator
Net loss	$ (27,282)	$ (4,416)	$ (66,484)	$ (33,688)
Deemed dividend				(18,249)
Gain on exchange of convertible preferred stock in connection with recapitalization				3,391
Net loss attributable to common stockholders	$ (27,282)	$ (4,416)	$ (66,484)	$ (48,546)
Denominator
Denominator for basic and diluted net loss per share (in shares)	29,351,693	23,803,282	28,441,827	8,995,167
Net loss per share:
Basic and diluted (in dollars per share)	$ (0.93)	$ (0.19)	$ (2.34)	$ (5.40)

- Definition

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

+ References + Details

Stock option plan (Details) - USD ($) $ / shares in Units, $ in Thousands		1 Months Ended	9 Months Ended
	Mar. 05, 2013	May 31, 2013	Sep. 30, 2014
Stock option
Number of options
Outstanding at the beginning of the period (in shares)			2,095,592
Granted (in shares)			1,469,496
Forfeited (in shares)			(85,775)
Expired (in shares)			(35)
Outstanding at the end of the period (in shares)			3,443,778
Vested or expected to vest at the end of the period (in shares)			3,217,812
Exercisable at the end of the period (in shares)			722,835
Exercised (in shares)			(35,500)
Weighted-average exercise price
Outstanding at the beginning of the period (in dollars per share)			$ 20.24
Granted (in dollars per share)			29.57
Forfeited (in dollars per share)			11.78
Expired (in dollars per share)			646.66
Outstanding at the end of the period (in dollars per share)			24.52
Vested or expected to vest at the end of the period (in dollars per share)			23.01
Exercisable at the end of the period (in dollars per share)			36.23
Exercised (in dollars per share)			$ 10.96
Weighted-average remaining contractual term
Outstanding at the end of the period			8 years 10 months 24 days
Vested or expected to vest at the end of the period			9 years
Exercisable at the end of the period			8 years 3 months 18 days
Aggregate intrinsic value
Outstanding at the end of the period (in dollars)			$ 84,456
Vested or expected to vest at the end of the period (in dollars)			79,675
Exercisable at the end of the period (in dollars)			$ 22,463
Valuation assumptions
Weighted average grant date fair value (in dollars per share)			$ 21.70
Inducement grants for non-statutory stock options.			448,800
Stock option \| Minimum
Valuation assumptions
Risk-free interest rate (as a percent)			0.11%
Expected volatility (as a percent)			79.00%
Expected term			5 years 6 months
Stock option \| Maximum
Valuation assumptions
Risk-free interest rate (as a percent)			2.04%
Expected volatility (as a percent)			91.00%
Expected term			6 years 3 months
2013 Stock Incentive Plan
Stock award plan
Number of shares available for issuance	0
2013 Stock Incentive Plan \| Stock option
Stock award plan
Number of shares of restricted stock granted	4,613
2013 Stock Incentive Plan \| Restricted stock
Stock award plan
Number of shares of restricted stock granted	735,324
2013 Stock Incentive Plan \| Common stock
Stock award plan
Number of shares authorized	739,937
2009 Equity and Long-Term Incentive Plan
Stock award plan
Number of additional shares authorized		2,500,000
2009 Equity and Long Term Incentive Plan and 2013 Stock Incentive Plan \| Common stock
Stock award plan
Number of shares available for issuance		122,296
2013 Long Term Incentive Plan \| Minimum
Stock award plan
Annual increase in the number of shares on the first day of the fiscal year		2,500,000
Annual increase in the number of shares outstanding on the first day of the fiscal year (as a percent)		4.00%
2013 Long Term Incentive Plan \| Common stock \| Maximum
Stock award plan
Number of shares subject to outstanding awards		3,040,444

Stock option plan (Details 2) - Restricted stock	9 Months Ended
Stock option plan (Details 2) - Restricted stock	Sep. 30, 2014 $ / shares shares
Number of Shares
Balance at the beginning of the period (in shares) \| shares	1,110,226
Vested (in shares) \| shares	(371,280)
Forfeited (in shares) \| shares	(9,626)
Balance at the end of the period (in shares) \| shares	729,320
Weighted Average Grant Date Fair Value
Balance at the beginning of the period (in dollars per share) \| $ / shares	$ 10.68
Vested (in dollars per share) \| $ / shares	10.60
Forfeited (in dollars per share) \| $ / shares	10.66
Balance at the end of the period (in dollars per share) \| $ / shares	$ 10.72

The Company	9 Months Ended
The Company	Sep. 30, 2014
The Company
The Company	1.The Company PTC Therapeutics, Inc. (the Company or PTC) was incorporated as a Delaware corporation on March 31, 1998. During the second quarter of 2014, a wholly-owned subsidiary was established in Bermuda to hold certain intellectual property rights of the Company. In addition, wholly-owned subsidiaries in Ireland, Denmark, Italy, Germany, Switzerland and France were established during 2014. PTC is a biopharmaceutical company focused on the discovery and development of orally administered, proprietary small molecule drugs that target post-transcriptional control processes. The Company’s internally discovered pipeline addresses multiple therapeutic areas, including rare disorders, oncology and infectious diseases. PTC has developed proprietary technologies that PTC applies in our drug discovery activities and in collaborations with leading biopharmaceutical companies. The Company’s lead candidate is ataluren, an investigational new drug in the US, for the treatment of patients with genetic disorders that arise from a type of genetic mutation known as a nonsense mutation. The brand name of ataluren is Translarna™. On August 4, 2014, the Company was notified that the European Commission, or EC, granted conditional marketing authorization for Translarna for the treatment of Duchenne muscular dystrophy caused by nonsense mutations, or nmDMD, in ambulatory patients aged five years and older. The conditional marketing authorization allows the Company to market Translarna in the European Economic Area, or EEA, which is comprised of the 28 member states of the European Union plus Norway, Iceland and Liechtenstein. The conditional marketing authorization is subject to an annual review by the EMA and the Company will seek to renew the approval on an annual basis until its obligations have been fulfilled and the approval is converted from a conditional approval into a full approval. The Company has not generated significant product revenue to date and is subject to a number of risks similar to those of other early stage companies, including dependence on key individuals, the difficulties inherent in the development of commercially usable products, the potential need to obtain additional capital necessary to fund the development of its products, and competition from other companies. As of September 30, 2014, the Company had an accumulated deficit of approximately $395.3 million. The Company has financed its operations to date primarily through public offerings of common stock in February 2014 and October 2014 (see Notes 6 and 10 below), its initial public offering of common stock in June 2013 (see Note 6 below), private placements of its convertible preferred stock, collaborations, bank debt, convertible debt financings, 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.

Collaboration Revenue	9 Months Ended
Collaboration Revenue	Sep. 30, 2014
Collaboration Revenue
Collaboration Revenue	9.Collaboration Revenue On January 22, 2014, the Company announced the initiation of a Phase 1 clinical program in its spinal muscular atrophy collaboration with F. Hoffman-La Roche Ltd and Hoffman-La Roche Inc. (Roche) and the Spinal Muscular Atrophy Foundation which triggered a $7.5 million milestone payment from Roche. The Company considered this milestone event substantive because the applicable criteria of its revenue recognition policy would be satisfied and recorded it as collaboration revenue for the three months ended March 31, 2014.

Subsequent events	9 Months Ended
Subsequent events	Sep. 30, 2014
Subsequent events
Subsequent events	10.Subsequent events On October 16, 2014, the Company closed an underwritten public offering of its common stock pursuant to a registration statement on Form S-3. The Company issued and sold an aggregate of 3,450,000 shares of common stock under the registration statement at a public offering price of $36.25 per share, including 450,000 shares issued upon exercise by the underwriters of their option to purchase additional shares. The Company received net proceeds of approximately $117.5 million after deducting underwriting discounts and commissions and other offering expenses payable by the Company.

The Company (Details1) $ in Thousands	Aug. 04, 2014 state	Sep. 30, 2014 USD ($)	Dec. 31, 2013 USD ($)
The Company
Accumulated deficit \| $		$ 395,282	$ 328,798
Minimum age of ambulatory patient	5 years
Number of member states of the European Union \| state	28

Fair value of financial instruments and marketable securities (Details 2) - USD ($) $ in Thousands	Sep. 30, 2014	Dec. 31, 2013
Marketable securities accounted for as available-for-sale securities
Amortized Cost	$ 156,585	$ 126,982
Gross Unrealized Gains	120	102
Gross Unrealized Losses	(189)	(31)
Fair Value	156,516	127,053
Commercial paper
Marketable securities accounted for as available-for-sale securities
Amortized Cost		14,993
Gross Unrealized Gains		5
Fair Value		14,998
Corporate Debt Securities [Member]
Marketable securities accounted for as available-for-sale securities
Amortized Cost	122,561	111,989
Gross Unrealized Gains	112	97
Gross Unrealized Losses	(156)	(31)
Fair Value	122,517	$ 112,055
Government Agencies Debt Securities [Member]
Marketable securities accounted for as available-for-sale securities
Amortized Cost	34,024
Gross Unrealized Gains	8
Gross Unrealized Losses	(33)
Fair Value	$ 33,999

Statements of comprehensive loss - USD ($) $ in Thousands	3 Months Ended		9 Months Ended
Statements of comprehensive loss - USD ($) $ in Thousands	Sep. 30, 2014	Sep. 30, 2013	Sep. 30, 2014	Sep. 30, 2013
Statements of comprehensive loss
Net loss	$ (27,282)	$ (4,416)	$ (66,484)	$ (33,688)
Other comprehensive loss:
Unrealized loss on marketable securities	(159)	(40)	(139)	(40)
Comprehensive loss	$ (27,441)	$ (4,456)	$ (66,623)	$ (33,728)

Summary of significant accounting policies (Details)	Mar. 07, 2013
Common stock
Reverse stock split
Reverse stock split ratio	0.0083

Fair value of financial instruments and marketable securities (Details) - USD ($) $ in Thousands	Sep. 30, 2014	Dec. 31, 2013
Financial assets and liabilities measured at fair value on recurring basis
Marketable securities	$ 156,516	$ 127,053
Recurring basis \| Total
Financial assets and liabilities measured at fair value on recurring basis
Marketable securities	156,516	127,053
Warrant liability	136	58
Recurring basis \| Significant other observable inputs (level 2)
Financial assets and liabilities measured at fair value on recurring basis
Marketable securities	156,516	127,053
Recurring basis \| Significant unobservable inputs (level 3)
Financial assets and liabilities measured at fair value on recurring basis
Warrant liability	$ 136	$ 58

Fair value of financial instruments and marketable securities (Details 3) - USD ($) $ in Thousands	Sep. 30, 2014	Dec. 31, 2013
Marketable securities on the balance sheet
Total Marketable securities, Less Than 12 Months	$ 65,860	$ 69,157
Total Marketable securities, More Than 12 Months	90,656	57,896
Commercial paper
Marketable securities on the balance sheet
Total Marketable securities, Less Than 12 Months		14,998
Corporate Debt Securities [Member]
Marketable securities on the balance sheet
Total Marketable securities, Less Than 12 Months	65,860	54,159
Total Marketable securities, More Than 12 Months	56,657	$ 57,896
Government Agencies Debt Securities [Member]
Marketable securities on the balance sheet
Total Marketable securities, More Than 12 Months	$ 33,999

Other comprehensive income (loss) and accumulated other comprehensive items (Details) - USD ($) $ in Thousands	3 Months Ended	9 Months Ended
	Sep. 30, 2014	Sep. 30, 2014
Other comprehensive income and accumulated other comprehensive items
Balance at the beginning of the period	$ 90	$ 70
Other comprehensive income/(losses) before reclassifications	(159)	(139)
Other comprehensive income/(loss)	(159)	(139)
Balance at the end of the period	(69)	(69)
Unrealized Gains/(Losses) On Marketable Securities
Other comprehensive income and accumulated other comprehensive items
Balance at the beginning of the period	90	70
Other comprehensive income/(losses) before reclassifications	(159)	(139)
Other comprehensive income/(loss)	(159)	(139)
Balance at the end of the period	$ (69)	$ (69)

Capital structure (Details 2) - Warrant - Common stock - $ / shares	Sep. 30, 2014	Dec. 31, 2013
2014.
Warrants
Warrant shares		1,428
Exercise price (in dollars per share)		$ 128.00
2017
Warrants
Warrant shares	6,250	6,250
Exercise price (in dollars per share)	$ 128.00	$ 128.00
2019 and 2020
Warrants
Warrant shares	7,030	7,030
Exercise price (in dollars per share)	$ 128.00	$ 128.00
2019
Warrants
Warrant shares	130
Exercise price (in dollars per share)	$ 2,520.00
2014
Warrants
Warrant shares		452
Exercise price (in dollars per share)		$ 2,520.00

Net loss per share (Details 2) - shares	9 Months Ended
Net loss per share (Details 2) - shares	Sep. 30, 2014	Sep. 30, 2013
Net loss per share
Total shares excluded from calculation	4,173,098	3,101,667
Stock Options
Net loss per share
Total shares excluded from calculation	3,443,778	1,987,860
Restricted stock
Net loss per share
Total shares excluded from calculation	729,320	1,113,807

Stock option plan (Details 3) - USD ($) $ in Thousands	3 Months Ended		9 Months Ended
Stock option plan (Details 3) - USD ($) $ in Thousands	Sep. 30, 2014	Sep. 30, 2013	Sep. 30, 2014	Sep. 30, 2013
Share-based compensation expense recorded in the statement of operations
Share-based compensation expense	$ 4,621	$ 2,640	$ 12,605	$ 5,142
Unrecognized compensation cost	38,700		$ 38,700
Weighted average remaining service period for recognition of unrecognized compensation cost			2 years 8 months 12 days
Research and development
Share-based compensation expense recorded in the statement of operations
Share-based compensation expense	2,363	677	$ 6,517	1,378
General and administrative
Share-based compensation expense recorded in the statement of operations
Share-based compensation expense	$ 2,258	$ 1,963	$ 6,088	$ 3,764

Collaboration Revenue (Details) $ in Millions	Jan. 22, 2014 USD ($)
Collaboration agreement \| Roche and SMA Foundation \| Clinical development milestones
Collaboration Revenue
Milestone payment received from Roche	$ 7.5

Subsequent events (Details) - USD ($) $ / shares in Units, $ in Millions			1 Months Ended
	Feb. 12, 2014	Mar. 07, 2013	Oct. 31, 2014	Jun. 30, 2013
Series Four convertible preferred stock
Subsequent events
Shares issued		4,497,035
Common stock
Subsequent events
Shares issued	5,163,265			9,627,800
Share price (in dollars per share)				$ 15.00
Subsequent event
Subsequent events
Share price (in dollars per share)			$ 36.25
Subsequent event \| Scenario, Forecast [Member]
Subsequent events
Net proceeds			$ 117.5
Subsequent event \| Follow On Public Offering [Member]
Subsequent events
Shares issued			3,450,000
Subsequent event \| Over Allotment Option [Member]
Subsequent events
Shares issued			450,000