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Document and Entity Information - shares	3 Months Ended
Document and Entity Information - shares	Mar. 31, 2015	Apr. 30, 2015
Document and Entity Information
Entity Registrant Name	PTC THERAPEUTICS, INC.
Entity Central Index Key	0001070081
Document Type	10-Q
Document Period End Date	Mar. 31, 2015
Amendment Flag	false
Current Fiscal Year End Date	--12-31
Entity Current Reporting Status	Yes
Entity Filer Category	Accelerated Filer
Entity Common Stock, Shares Outstanding		33,848,276
Document Fiscal Year Focus	2015
Document Fiscal Period Focus	Q1

Consolidated Balance Sheets - USD ($) $ in Thousands	Mar. 31, 2015	Dec. 31, 2014
Current assets:
Cash and cash equivalents	$ 36,825	$ 49,748
Marketable securities	243,642	265,493
Prepaid expenses and other current assets	6,116	3,885
Receivables, net	3,178	4,445
Total current assets	289,761	323,571
Fixed assets, net	9,408	9,159
Deposits and other assets	351	489
Total assets	299,520	333,219
Current liabilities:
Accounts payable and accrued expenses	23,532	29,121
Deferred revenue	373	3,354
Total current liabilities	23,905	32,475
Other long-term liabilities	2,295	2,277
Total liabilities	26,200	34,752
Common stock, $0.001 par value. Authorized 125,000,000 shares; issued and outstanding 33,483,306 shares at March 31, 2015. Authorized 125,000,000 shares; issued and outstanding 32,898,392 shares at December 31, 2014	33	33
Additional paid-in capital	734,489	721,722
Accumulated other comprehensive loss	(736)	(737)
Accumulated deficit	(460,466)	(422,551)
Total stockholders' equity	273,320	298,467
Total liabilities and stockholders' equity	$ 299,520	$ 333,219

Consolidated Statements of Operations - USD ($) $ in Thousands	3 Months Ended
	Mar. 31, 2015	Mar. 31, 2014
Revenues:
Net product revenue	$ 5,069
Collaboration revenue	338	$ 9,147
Grant revenue	2,075	70
Total revenues	7,482	9,217
Operating expenses:
Research and development	27,938	15,889
Selling, general and administrative	17,615	7,540
Total operating expenses	45,553	23,429
Loss from operations	(38,071)	(14,212)
Interest income	524	171
Other (expense) income, net	(368)	(57)
Net loss	$ (37,915)	$ (14,098)
Weighted-average shares outstanding:
Basic and diluted (in shares)	33,067,752	24,492,487
Net loss per share - basic and diluted (in dollars per share)	$ (1.15)	$ (0.58)

Consolidated Statements of Comprehensive Loss - USD ($) $ in Thousands	3 Months Ended
	Mar. 31, 2015	Mar. 31, 2014
Consolidated Statements of Comprehensive Loss
Net loss	$ (37,915)	$ (14,098)
Other comprehensive loss:
Unrealized gain on marketable securities	125	10
Foreign currency translation loss	(124)
Comprehensive loss	$ (37,914)	$ (14,088)

- Definition

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

+ References + Details

Consolidated Statements of Cash Flows - USD ($) $ in Thousands	3 Months Ended
	Mar. 31, 2015	Mar. 31, 2014
Cash flows from operating activities
Net loss	$ (37,915)	$ (14,098)
Adjustments to reconcile net loss to net cash used in operating activities:
Depreciation	634	588
Change in valuation of warrant liability	41	55
Amortization of premiums on investments	486	414
Share-based compensation expense	9,748	3,705
Changes in operating assets and liabilities:
Prepaid expenses and other current assets	(2,231)	(358)
Receivables	1,267	120
Deposits and other assets	138	17
Accounts payable and accrued expenses	(5,589)	(3,472)
Other long-term liabilities	(23)	(23)
Deferred revenue	(2,981)	(386)
Net cash used in operating activities	(36,425)	(13,438)
Cash flows from investing activities
Purchases of fixed assets	(883)	(186)
Purchases of marketable securities	(19,108)	(25,354)
Sale & redemption of marketable securities	40,598	21,273
Net cash provided/ used in investing activities	20,607	(4,267)
Cash flows from financing activities
Payments on long-term debt		(37)
Proceeds from exercise of options	3,019
Net proceeds from public offerings		118,183
Net cash provided by financing activities	3,019	118,146
Effect of exchange rate changes on cash	(124)
Net increase/(decrease) in cash and cash equivalents	(12,923)	100,441
Cash and cash equivalents, beginning of period	49,748	15,414
Cash and cash equivalents, end of period	36,825	115,855
Supplemental disclosure of cash information
Cash paid for interest		1
Supplemental disclosures of non-cash information related to investing and financing activities
Change in unrealized gain (loss) on marketable securities	$ 125	$ 10

Summary of significant accounting policies	3 Months Ended
Summary of significant accounting policies	Mar. 31, 2015
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, 2014 included in the Company’s Annual Report on Form 10-K filed with the Securities and Exchange Commission (SEC) on March 2, 2015 (2014 Form 10-K). Basis of Presentation The accompanying financial information as of March 31, 2015 and for the three months ended March 31, 2015 and 2014 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, 2014 and notes thereto included in the 2014 Form 10-K. In the opinion of management, the unaudited financial information as of March 31, 2015 and for the three months ended March 31, 2015 and 2014 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 month period ended March 31, 2015 are not necessarily indicative of the results to be expected for the year ended December 31, 2015 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. Inventories and cost of product revenue On August 4, 2014, the Company was notified that the European Commission, or EC, granted 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 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. This marketing authorization is subject to annual review and renewal by the European Medicines Agency, or EMA following its reassessment of the risk-benefit balance of the authorization and is further conditioned on the Company’s ability to complete its global, confirmatory Phase 3 clinical trial in nm DMD, which it refers to as ACT DMD, and submit the final report, including additional efficacy and safety data from the trial during 2015. The launch in these countries is on a country by country basis. The Company does do not have sufficient history or experience from which to accurately forecast product sales or demand generation. As such, the Company has not capitalized inventory and will not capitalize inventory until the completion of ACT DMD and satisfaction of the EMA conditions or until the Company can reasonably predict future product sales. The costs incurred related to the manufacturing of Translarna have been recorded as research and development expense in the statements of operations. The Company’s cost of product sales includes royalties and other miscellaneous selling costs, which were not material and therefore included as a component of research and development costs in the current year presentation. The time period over which this inventory is consumed will depend on a number of factors, including the amount of future Translarna sales, and the ability to utilize inventory prior to its expiration date. 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 The Company recognizes revenue when amounts are realized or realizable and earned. Revenue is considered realizable and earned when the following criteria are met: (1) persuasive evidence of an arrangement exists; (2) delivery has occurred or services have been rendered; (3) the price is fixed or determinable; and (4) collection of the amounts due are reasonably assured. Net Product Sales PTC’S net product sales have consisted solely of sales of Translarna for the treatment of nmDMD in territories outside of the U.S. 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 records revenue on sales where Translarna is available either on a commercial basis or through a reimbursed early access program and typically paid for by a government authority or institution. Prior to January 1, 2015, the Company recognized revenue for commercial and reimbursed early access program sales on a cash basis once the product was shipped on behalf of the government authority or institution and payment had been received, if all other revenue recognition criteria were met. Beginning in the first quarter of 2015, the Company is recognizing revenue for Translarna as product is shipped, as the Company has established a pattern of collectability. 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 at the time of delivery. These allowances are adjusted to reflect known changes in factors that may impact such allowances in the quarter those changes are known. Collaboration and Grant Revenue The terms of these agreements typically include payments to the Company of one or more of the following: nonrefundable, upfront license fees; milestone payments; research funding and royalties on future product sales. In addition, the Company generates service revenue through agreements that generally provide for fees for research and development services and may include additional payments upon achievement of specified events. The Company evaluates all contingent consideration earned, such as a milestone payment, using the criteria as provided by the Financial Accounting Standards Board (FASB), guidance on the milestone method of revenue recognition. At the inception of a collaboration arrangement, the Company evaluates if a milestone payment is substantive. The criteria requires that (1) the Company determines if the milestone is commensurate with either its performance to achieve the milestone or the enhancement of value resulting from our activities to achieve the milestone; (2) the milestone be related to past performance; and (3) the milestone be reasonable relative to all deliverable and payment terms of the collaboration arrangement. If these criteria are met then the contingent milestones can be considered a substantive milestone and will be recognized as revenue in the period that the milestone is achieved. The Company recognizes royalties as earned in accordance with the terms of various research and collaboration agreements. If not substantive, the contingent consideration is allocated to the existing units of accounting based on relative selling price and recognized following the same basis previously established for the associated unit of accounting. The Company recognizes revenue for reimbursements of research and development costs under collaboration agreements as the services are performed. The Company records these reimbursements as revenue and not as a reduction of research and development expenses as the Company has the risks and rewards as the principal in the research and development activities.

Fair value of financial instruments and marketable securities

3 Months Ended

Mar. 31, 2015

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 March 31, 2015 and December 31, 2014:

	March 31, 2015
	Total		Quoted prices in active markets for identical assets (level 1)		Significant other observable inputs (level 2)		Significant unobservable inputs (level 3)
Marketable securities	$	243,642	$	—	$	243,642	$	—
Warrant liability	229		—		—		229

	December 31, 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	$	265,493	$	—	$	265,493	$	—
Warrant Liability	188		—		—		188

The following is a summary of marketable securities accounted for as available-for-sale securities at March 31, 2015 and December 31, 2014:

	March 31, 2015
	Amortized		Gross Unrealized					Fair
	Cost		Gains		Losses			Value
Commercial paper	$	19,748	$	26	$	—		$	19,774
Corporate debt securities	186,810		44		(320		)	186,534
Government obligations	37,346		21		(33		)	37,334
	$	243,904	$	91	$	(353	)	$	243,642

	December 31, 2014
	Amortized		Gross Unrealized					Fair
	Cost		Gains		Losses			Value
Corporate debt securities	$	230,379	$	80	$	(428	)	$	230,031
Government obligations	35,501		7		(46		)	35,462
	$	265,880	$	87	$	(474	)	$	265,493

At March 31, 2015 and December 31, 2014, 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 March 31, 2015 and December 31, 2014 mature as follows:

	March 31, 2015
	Less Than 12 Months		More Than 12 Months
Commercial paper	$	19,774	$	—
Corporate debt securities	138,306		48,228
Government obligations	11,312		26,022
Total Marketable securities	$	169,392	$	74,250

	December 31, 2014
	Less Than 12 Months		More Than 12 Months
Corporate debt securities	$	157,758	$	72,273
Government obligations	6,003		29,459
Total Marketable securities	$	163,761	$	101,732

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 March 31, 2015:

	Level 3 assets
Beginning balance as of December 31, 2014	$	188
Change in fair value of warrant liability	41
Ending balance as of March 31, 2015	$	229

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 March 31, 2015 include (i) volatility (68%—70%), (ii) risk free interest rate (0.56%—1.13%), (iii) strike price ($128-$2,520), (iv) fair value of common stock ($60.85), and (v) expected life (2.21—4.48 years). The significant assumptions used in preparing the option pricing model for valuing the Company’s warrants as of December 31, 2014 include (i) volatility (68%-70%), (ii) risk free interest rate (0.89%—1.65%), (iii) strike price ($128—$2,520), (iv) fair value of common stock ($51.77), and (v) expected life (2.50—4.70 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

3 Months Ended

Mar. 31, 2015

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 months ended March 31, 2015:

	Unrealized Gains/(Losses) On Marketable Securities			Foreign Currency Translation			Total Accumulated Other Comprehensive Items
Balance at December 31, 2014	$	(387	)	$	(350	)	$	(737	)
Other comprehensive income/(loss) before reclassifications	125			(124		)	1
Amounts reclassified from other comprehensive items	—			—			—
Other comprehensive income/(loss)	125			(124		)	1
Balance at March 31, 2015	$	(262	)	$	(474	)	$	(736	)

- 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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+ Details

Warrants

3 Months Ended

Mar. 31, 2015

Warrants

Warrants

6.Warrants

All of the Company’s outstanding warrants were classified as liabilities as of March 31, 2015 and December 31, 2014 because they contained non-standard antidilution provisions.

The following is a summary of the Company’s outstanding warrants as of March 31, 2015:

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

The following is a summary of the Company’s outstanding warrants as of December 31, 2014:

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

- 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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-Publisher FASB
-Name Accounting Standards Codification
-Topic 505
-SubTopic 10
-Section 50
-Paragraph 11
-URI http://asc.fasb.org/extlink&oid=6928386&loc=d3e21564-112644

Reference 14: http://www.xbrl.org/2003/role/presentationRef
-Publisher SEC
-Name Regulation S-X (SX)
-Number 210
-Section 08
-Paragraph d
-Article 4

Reference 15: http://www.xbrl.org/2003/role/presentationRef
-Publisher SEC
-Name Regulation S-X (SX)
-Number 210
-Section 02
-Paragraph 29, 30, 31
-Article 5

Reference 16: http://www.xbrl.org/2003/role/presentationRef
-Publisher SEC
-Name Staff Accounting Bulletin (SAB)
-Number Topic 4
-Section E

Reference 17: http://www.xbrl.org/2003/role/presentationRef
-Publisher SEC
-Name Regulation S-X (SX)
-Number 210
-Section 08
-Article 4

Reference 18: http://www.xbrl.org/2003/role/presentationRef
-Publisher SEC
-Name Staff Accounting Bulletin (SAB)
-Number Topic 4
-Section C

Reference 19: http://www.xbrl.org/2003/role/presentationRef
-Publisher SEC
-Name Regulation S-X (SX)
-Number 210
-Section 04
-Article 3

+ Details

Net loss per share

3 Months Ended

Mar. 31, 2015

Net loss per share

Net loss per share

7.Net loss per share

Basic earnings per share is computed by dividing net income (loss) by the weighted-average number of common shares outstanding. Diluted earnings per share is computed by dividing net income (loss) 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 loss per share:

	Three months ended March 31,
	2015			2014
Numerator
Net loss	$	(37,915	)	$	(14,098	)
Denominator
Denominator for basic and diluted net loss per share	33,067,752			24,492,487
Net loss per share:
Basic and diluted	$	(1.15	)*	$	(0.58	)*

*In the three months ended March 31, 2015 and 2014, 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 March 31,
	2015	2014

Stock Options	4,627,833	3,133,830
Unvested restricted stock	364,970	748,456
Total	4,992,803	3,882,286

Stock award plan

3 Months Ended

Mar. 31, 2015

Stock award plan

Stock award 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 Equity and Long Term 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) 245,000 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, 2009 Equity and Long Term Incentive 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, 2015 through March 31, 2015, the Company issued a total of 1,456,550 stock options to various employees. Of those, 93,350 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, 2014	3,432,972		$	25.00
Granted	1,456,550		$	51.02
Exercised	(234,830	)	$	12.86
Forfeited	(26,265	)	$	47.52
Expired	(594	)	$	388.53
Outstanding at March 31, 2015	4,627,833		$	33.60	8.9 years	$	140,399
Vested or Expected to vest at March 31, 2015	4,332,726		$	32.74	8.9 years	$	133,232
Exercisable at March 31, 2015	1,037,262		$	30.98	8.2 years	$	45,137

The fair value of grants made in the three months ended March 31, 2015 was contemporaneously estimated on the date of grant using the following assumptions:

		Three months ended March 31, 2015
Risk-free interest rate		1.54% — 1.77%
Expected volatility		67%-69%
Expected term		5.50 - 6.11 years

The Company assumed no expected dividends for all grants. The weighted average grant date fair value of options granted during the three month period ended March 31, 2015 was $31.82 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, 2015	718,400		$	10.72
Granted	—		—
Vested	(350,084	)	$	10.59
Forfeited	(3,346	)	$	10.82
Unvested at March 31, 2015	364,970		$	10.85

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

	Three Months Ended March 31,
	2015		2014

Research and development	$	4,667	$	1,944
Selling, general and administrative	5,081		1,761
Total	$	9,748	$	3,705

As of March 31, 2015 there was approximately $70.6 million of total unrecognized compensation cost related to unvested share-based compensation arrangements granted under the 2009 Equity and Long Term Incentive Plan, 2013 Long Term Incentive Plan and equity awards made pursuant to the NASDAQ inducement grant exception for new hires. This cost is expected to be recognized as share-based compensation expense over the weighted average remaining service period of approximately 3.0 years.

Summary of significant accounting policies (Policies)	3 Months Ended
Summary of significant accounting policies (Policies)	Mar. 31, 2015
Summary of significant accounting policies
Basis of presentation	Basis of Presentation The accompanying financial information as of March 31, 2015 and for the three months ended March 31, 2015 and 2014 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, 2014 and notes thereto included in the 2014 Form 10-K. In the opinion of management, the unaudited financial information as of March 31, 2015 and for the three months ended March 31, 2015 and 2014 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 month period ended March 31, 2015 are not necessarily indicative of the results to be expected for the year ended December 31, 2015 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.
Inventories and cost of product revenue	Inventories and cost of product revenue On August 4, 2014, the Company was notified that the European Commission, or EC, granted 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 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. This marketing authorization is subject to annual review and renewal by the European Medicines Agency, or EMA following its reassessment of the risk-benefit balance of the authorization and is further conditioned on the Company’s ability to complete its global, confirmatory Phase 3 clinical trial in nm DMD, which it refers to as ACT DMD, and submit the final report, including additional efficacy and safety data from the trial during 2015. The launch in these countries is on a country by country basis. The Company does do not have sufficient history or experience from which to accurately forecast product sales or demand generation. As such, the Company has not capitalized inventory and will not capitalize inventory until the completion of ACT DMD and satisfaction of the EMA conditions or until the Company can reasonably predict future product sales. The costs incurred related to the manufacturing of Translarna have been recorded as research and development expense in the statements of operations. The Company’s cost of product sales includes royalties and other miscellaneous selling costs, which were not material and therefore included as a component of research and development costs in the current year presentation. The time period over which this inventory is consumed will depend on a number of factors, including the amount of future Translarna sales, and the ability to utilize inventory prior to its expiration date.
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 The Company recognizes revenue when amounts are realized or realizable and earned. Revenue is considered realizable and earned when the following criteria are met: (1) persuasive evidence of an arrangement exists; (2) delivery has occurred or services have been rendered; (3) the price is fixed or determinable; and (4) collection of the amounts due are reasonably assured. Net Product Sales PTC’S net product sales have consisted solely of sales of Translarna for the treatment of nmDMD in territories outside of the U.S. 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 records revenue on sales where Translarna is available either on a commercial basis or through a reimbursed early access program and typically paid for by a government authority or institution. Prior to January 1, 2015, the Company recognized revenue for commercial and reimbursed early access program sales on a cash basis once the product was shipped on behalf of the government authority or institution and payment had been received, if all other revenue recognition criteria were met. Beginning in the first quarter of 2015, the Company is recognizing revenue for Translarna as product is shipped, as the Company has established a pattern of collectability. 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 at the time of delivery. These allowances are adjusted to reflect known changes in factors that may impact such allowances in the quarter those changes are known. Collaboration and Grant Revenue The terms of these agreements typically include payments to the Company of one or more of the following: nonrefundable, upfront license fees; milestone payments; research funding and royalties on future product sales. In addition, the Company generates service revenue through agreements that generally provide for fees for research and development services and may include additional payments upon achievement of specified events. The Company evaluates all contingent consideration earned, such as a milestone payment, using the criteria as provided by the Financial Accounting Standards Board (FASB), guidance on the milestone method of revenue recognition. At the inception of a collaboration arrangement, the Company evaluates if a milestone payment is substantive. The criteria requires that (1) the Company determines if the milestone is commensurate with either its performance to achieve the milestone or the enhancement of value resulting from our activities to achieve the milestone; (2) the milestone be related to past performance; and (3) the milestone be reasonable relative to all deliverable and payment terms of the collaboration arrangement. If these criteria are met then the contingent milestones can be considered a substantive milestone and will be recognized as revenue in the period that the milestone is achieved. The Company recognizes royalties as earned in accordance with the terms of various research and collaboration agreements. If not substantive, the contingent consideration is allocated to the existing units of accounting based on relative selling price and recognized following the same basis previously established for the associated unit of accounting. The Company recognizes revenue for reimbursements of research and development costs under collaboration agreements as the services are performed. The Company records these reimbursements as revenue and not as a reduction of research and development expenses as the Company has the risks and rewards as the principal in the research and development activities.

- 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)

3 Months Ended

Mar. 31, 2015

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

	March 31, 2015
	Total		Quoted prices in active markets for identical assets (level 1)		Significant other observable inputs (level 2)		Significant unobservable inputs (level 3)
Marketable securities	$	243,642	$	—	$	243,642	$	—
Warrant liability	229		—		—		229

	December 31, 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	$	265,493	$	—	$	265,493	$	—
Warrant Liability	188		—		—		188

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

	March 31, 2015
	Amortized		Gross Unrealized					Fair
	Cost		Gains		Losses			Value
Commercial paper	$	19,748	$	26	$	—		$	19,774
Corporate debt securities	186,810		44		(320		)	186,534
Government obligations	37,346		21		(33		)	37,334
	$	243,904	$	91	$	(353	)	$	243,642

	December 31, 2014
	Amortized		Gross Unrealized					Fair
	Cost		Gains		Losses			Value
Corporate debt securities	$	230,379	$	80	$	(428	)	$	230,031
Government obligations	35,501		7		(46		)	35,462
	$	265,880	$	87	$	(474	)	$	265,493

Schedule of marketable securities on the balance sheet

	March 31, 2015
	Less Than 12 Months		More Than 12 Months
Commercial paper	$	19,774	$	—
Corporate debt securities	138,306		48,228
Government obligations	11,312		26,022
Total Marketable securities	$	169,392	$	74,250

	December 31, 2014
	Less Than 12 Months		More Than 12 Months
Corporate debt securities	$	157,758	$	72,273
Government obligations	6,003		29,459
Total Marketable securities	$	163,761	$	101,732

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, 2014	$	188
Change in fair value of warrant liability	41
Ending balance as of March 31, 2015	$	229

Warrants (Tables)

3 Months Ended

Mar. 31, 2015

Warrants

Summary of the Company's outstanding warrants

The following is a summary of the Company’s outstanding warrants as of March 31, 2015:

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

The following is a summary of the Company’s outstanding warrants as of December 31, 2014:

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

- 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)

3 Months Ended

Mar. 31, 2015

Net loss per share

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

	Three months ended March 31,
	2015			2014
Numerator
Net loss	$	(37,915	)	$	(14,098	)
Denominator
Denominator for basic and diluted net loss per share	33,067,752			24,492,487
Net loss per share:
Basic and diluted	$	(1.15	)*	$	(0.58	)*

*In the three months ended March 31, 2015 and 2014, 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 March 31,
	2015	2014

Stock Options	4,627,833	3,133,830
Unvested restricted stock	364,970	748,456
Total	4,992,803	3,882,286

Stock award plan (Tables)

3 Months Ended

Mar. 31, 2015

Stock award 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, 2014	3,432,972		$	25.00
Granted	1,456,550		$	51.02
Exercised	(234,830	)	$	12.86
Forfeited	(26,265	)	$	47.52
Expired	(594	)	$	388.53
Outstanding at March 31, 2015	4,627,833		$	33.60	8.9 years	$	140,399
Vested or Expected to vest at March 31, 2015	4,332,726		$	32.74	8.9 years	$	133,232
Exercisable at March 31, 2015	1,037,262		$	30.98	8.2 years	$	45,137

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

		Three months ended March 31, 2015
Risk-free interest rate		1.54% — 1.77%
Expected volatility		67%-69%
Expected term		5.50 - 6.11 years

Summary of information on the Company's restricted stock

	Restricted Stock
	Number of Shares		Weighted Average Grant Date Fair Value
January 1, 2015	718,400		$	10.72
Granted	—		—
Vested	(350,084	)	$	10.59
Forfeited	(3,346	)	$	10.82
Unvested at March 31, 2015	364,970		$	10.85

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

	Three Months Ended March 31,
	2015		2014

Research and development	$	4,667	$	1,944
Selling, general and administrative	5,081		1,761
Total	$	9,748	$	3,705

Fair value of financial instruments and marketable securities (Details 4) - Warrants - USD ($) $ / shares in Units, $ in Thousands	3 Months Ended	12 Months Ended
	Mar. 31, 2015	Dec. 31, 2014
Changes in the fair value of warrant liability
Beginning balance	$ 188
Change in fair value of warrant liability	41
Ending balance	$ 229	$ 188
Minimum
Assumption used to estimate the fair value of warrant liability by utilizing the Black-Scholes option-pricing model
Volatility (as a percent)	68.00%	68.00%
Risk-free interest rate (as a percent)	0.56%	0.89%
Strike price (in dollars per share)	$ 128	$ 128
Expected life	2 years 2 months 16 days	2 years 6 months
Maximum
Assumption used to estimate the fair value of warrant liability by utilizing the Black-Scholes option-pricing model
Volatility (as a percent)	70.00%	70.00%
Risk-free interest rate (as a percent)	1.13%	1.65%
Strike price (in dollars per share)	$ 2,520	$ 2,520
Expected life	4 years 5 months 23 days	4 years 8 months 12 days
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)	$ 60.85	$ 51.77

Accounts payable and accrued expenses (Details) - USD ($) $ in Thousands	Mar. 31, 2015	Dec. 31, 2014
Accounts payable and accrued expenses
Employee compensation, benefits, and related accruals	$ 3,365	$ 9,312
Consulting and contracted research	11,691	9,349
Professional fees	3,382	3,334
Accounts payable	3,139	4,128
Other	1,955	2,998
Accounts payable and accrued expenses	$ 23,532	$ 29,121

Stock award plan (Details) - USD ($) $ / shares in Units, $ in Thousands		1 Months Ended	3 Months Ended
	Mar. 05, 2013	May 31, 2013	Mar. 31, 2015
Stock option
Number of options
Outstanding at the beginning of the period (in shares)			3,432,972
Granted (in shares)			1,456,550
Exercised (in shares)			(234,830)
Forfeited (in shares)			(26,265)
Expired (in shares)			(594)
Outstanding at the end of the period (in shares)			4,627,833
Vested or expected to vest at the end of the period (in shares)			4,332,726
Exercisable at the end of the period (in shares)			1,037,262
Weighted-average exercise price
Outstanding at the beginning of the period (in dollars per share)			$ 25.00
Granted (in dollars per share)			51.02
Exercised (in dollars per share)			12.86
Forfeited (in dollars per share)			47.52
Expired (in dollars per share)			388.53
Outstanding at the end of the period (in dollars per share)			33.60
Vested or expected to vest at the end of the period (in dollars per share)			32.74
Exercisable at the end of the period (in dollars per share)			$ 30.98
Weighted-average remaining contractual term
Outstanding at the beginning of the period			8 years 10 months 24 days
Vested or expected to vest at the end of the period			8 years 10 months 24 days
Exercisable at the end of the period			8 years 2 months 12 days
Valuation assumptions
Inducement grants for non-statutory stock options			93,350
Weighted average grant date fair value (in dollars per share)			$ 31.82
Aggregate intrinsic value
Outstanding at the end of the period (in dollars)			$ 140,399
Vested or expected to vest at the end of the period (in dollars)			133,232
Exercisable at the end of the period (in dollars)			$ 45,137
Stock option \| Minimum
Valuation assumptions
Risk-free interest rate (as a percent)			1.54%
Expected volatility (as a percent)			67.00%
Expected term			5 years 6 months
Stock option \| Maximum
Valuation assumptions
Risk-free interest rate (as a percent)			1.77%
Expected volatility (as a percent)			69.00%
Expected term			6 years 1 month 10 days
2009 Equity and Long Term Incentive Plan
Stock option plan
Number of additional shares authorized		2,500,000
2013 Stock Incentive Plan
Stock option plan
Number of shares available for issuance	0
2013 Stock Incentive Plan \| Stock option
Stock option plan
Number of shares of restricted stock granted	4,613
2013 Stock Incentive Plan \| Restricted stock
Stock option plan
Number of shares of restricted stock granted	735,324
2013 Stock Incentive Plan \| Common stock
Stock option plan
Number of shares authorized	739,937
2009 Equity and Long Term Incentive Plan and 2013 Stock Incentive Plan \| Common stock
Stock option plan
Number of shares available for issuance		245,000
2009 Equity and Long Term Incentive Plan and 2013 Stock Incentive Plan \| Common stock \| Maximum
Stock option plan
Number of shares subject to outstanding awards		3,040,444
2013 Long Term Incentive Plan \| Minimum
Stock option 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%

Stock award plan - (Details 2) - Restricted stock	3 Months Ended
Stock award plan - (Details 2) - Restricted stock	Mar. 31, 2015 $ / shares shares
Number of Shares
Balance at the beginning of the period (in shares) \| shares	718,400
Vested (in shares) \| shares	(350,084)
Forfeited (in shares) \| shares	(3,346)
Balance at the end of the period (in shares) \| shares	364,970
Weighted Average Grant Date Fair Value
Balance at the beginning of the period (in dollars per share) \| $ / shares	$ 10.72
Vested \| $ / shares	10.59
Forfeited (in dollars per share) \| $ / shares	10.82
Balance at the end of the period (in dollars per share) \| $ / shares	$ 10.85

Commitments and contingencies (Details) $ in Thousands	3 Months Ended
Commitments and contingencies (Details) $ in Thousands	Mar. 31, 2015 USD ($)
Commitments and contingencies
Operating Leases, Rent Expense, Net	$ 300
Operating Leases, Future Minimum Payments Due, Fiscal Year Maturity
2015	1,016
2016	946
2017	923
2018	1,035
2019	176
Total future minimum lease payments	4,096
Funding agreement \| Wellcome trust \| Maximum
Other contingencies
Development and regulatory milestone payments which the entity may be obligated to pay	$ 68,900

The Company	3 Months Ended
The Company	Mar. 31, 2015
The Company
The Company	1.The Company PTC Therapeutics, Inc. (the Company or PTC) was incorporated as a Delaware corporation on March 31, 1998. PTC is a global biopharmaceutical company focused on the discovery, development and commercialization of orally administered, small molecule therapeutics targeting an area of RNA biology referred to as post-transcriptional control. The letters “PTC” in the corporate name are an acronym for post-transcriptional control processes, which are the regulatory events that occur in cells during and after a messenger RNA is copied from DNA through the transcription process. The Company has discovered all of its compounds currently under development using its proprietary technologies. The Company plans to continue to develop these compounds both on its own and through selective collaboration arrangements with leading pharmaceutical and biotechnology companies. The Company believes that systematically targeting post-transcriptional control processes represents an unexploited approach to drug discovery and development. The Company’s internally discovered pipeline addresses multiple therapeutic areas, including rare disorders, oncology and infectious diseases. The Company’s lead product, Translarna™ (ataluren) received marketing authorization from the European Commission, or EC, in August 2014 for the treatment of nonsense mutation Duchenne muscular dystrophy, or nmDMD, in ambulatory patients age 5 years and over in the 31 member states of the European Economic Area, or EEA. This marketing authorization is subject to annual review and renewal by the European Medicines Agency, or EMA following its reassessment of the risk-benefit balance of the authorization and is further conditioned on the Company’s ability to complete its global, confirmatory Phase 3 clinical trial in nmDMD, which it refers to as ACT DMD, and submit the final report, including additional efficacy and safety data from the trial, during 2015. See “Risk Factors—Risks Related to Regulatory Approval of our Product Candidates” for further detail regarding the EMA’s approval process, including a description of the risk-benefit balance. The Company launched Translarna on a commercial basis in Germany in December 2014 and expects to expand its launch activities across the EEA throughout 2015 and future years, subject to successful completion of pricing and reimbursement negotiations. Concurrently, the Company has been pursuing reimbursed early access programs in selected countries where those mechanisms exist, both within Europe and in those countries outside of Europe that will reference the marketing authorization described above. 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 March 31, 2015, the Company had an accumulated deficit of approximately $460.4 million. The Company has financed its operations to date primarily through public offerings of common stock in February 2014 and October 2014, its initial public offering of common stock in June 2013, 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	3 Months Ended
Collaboration Revenue	Mar. 31, 2015
Collaboration Revenue
Collaboration Revenue	10.Collaboration Revenue In January 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.

The Company $ in Thousands	Aug. 04, 2014 state	Mar. 31, 2015 USD ($)	Dec. 31, 2014 USD ($)
The Company
Minimum age of ambulatory patient	5 years
Number of member states of the European Union \| state	31
Accumulated deficit \| $		$ (460,466)	$ (422,551)

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

Consolidated Balance Sheets (Parenthetical) - $ / shares	Mar. 31, 2015	Dec. 31, 2014
Consolidated Balance Sheets
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	33,483,306	32,898,392
Common stock, outstanding shares	33,483,306	32,898,392

Fair value of financial instruments and marketable securities (Details) - USD ($) $ in Thousands	Mar. 31, 2015	Dec. 31, 2014
Financial assets and liabilities measured at fair value on recurring basis
Marketable securities	$ 243,642	$ 265,493
Recurring basis \| Total
Financial assets and liabilities measured at fair value on recurring basis
Marketable securities	243,642	265,493
Warrant liability	229	188
Recurring basis \| Significant other observable inputs (level 2)
Financial assets and liabilities measured at fair value on recurring basis
Marketable securities	243,642	265,493
Recurring basis \| Significant unobservable inputs (level 3)
Financial assets and liabilities measured at fair value on recurring basis
Warrant liability	$ 229	$ 188

Fair value of financial instruments and marketable securities (Details 2) - USD ($) $ in Thousands	Mar. 31, 2015	Dec. 31, 2014
Marketable securities accounted for as available-for-sale securities
Amortized Cost	$ 243,904	$ 265,880
Gross Unrealized Gains	91	87
Gross Unrealized Losses	(353)	(474)
Fair Value	243,642	265,493
Commercial paper
Marketable securities accounted for as available-for-sale securities
Amortized Cost	19,748
Gross Unrealized Gains	26
Fair Value	19,774
Corporate debt securities
Marketable securities accounted for as available-for-sale securities
Amortized Cost	186,810	230,379
Gross Unrealized Gains	44	80
Gross Unrealized Losses	(320)	(428)
Fair Value	186,534	230,031
Government obligations
Marketable securities accounted for as available-for-sale securities
Amortized Cost	37,346	35,501
Gross Unrealized Gains	21	7
Gross Unrealized Losses	(33)	(46)
Fair Value	$ 37,334	$ 35,462

Fair value of financial instruments and marketable securities (Details 3) - USD ($) $ in Thousands	Mar. 31, 2015	Dec. 31, 2014
Marketable securities on the balance sheet
Total Marketable securities, Less Than 12 Months	$ 169,392	$ 163,761
Total Marketable securities, More Than 12 Months	74,250	101,732
Commercial paper
Marketable securities on the balance sheet
Total Marketable securities, Less Than 12 Months	19,774
Corporate debt securities
Marketable securities on the balance sheet
Total Marketable securities, Less Than 12 Months	138,306	157,758
Total Marketable securities, More Than 12 Months	48,228	72,273
Government obligations
Marketable securities on the balance sheet
Total Marketable securities, Less Than 12 Months	11,312	6,003
Total Marketable securities, More Than 12 Months	$ 26,022	$ 29,459

Other comprehensive income (loss) and accumulated other comprehensive items (Details) $ in Thousands	3 Months Ended
	Mar. 31, 2015 USD ($)
Other comprehensive income and accumulated other comprehensive items
Balance at the beginning of the period	$ (737)
Other comprehensive income/(loss) before reclassifications	1
Other comprehensive income/(loss)	1
Balance at the end of the period	(736)
Unrealized Gains/(Losses) On Marketable Securities
Other comprehensive income and accumulated other comprehensive items
Balance at the beginning of the period	(387)
Other comprehensive income/(loss) before reclassifications	125
Other comprehensive income/(loss)	125
Balance at the end of the period	(262)
Foreign Currency Translation
Other comprehensive income and accumulated other comprehensive items
Balance at the beginning of the period	(350)
Other comprehensive income/(loss) before reclassifications	(124)
Other comprehensive income/(loss)	(124)
Balance at the end of the period	$ (474)

Warrants (Details) - Warrants - Common stock - $ / shares	Mar. 31, 2015	Dec. 31, 2014
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	130
Exercise price (in dollars per share)	$ 2,520.00	$ 2,520.00

Net loss per share (Details) - USD ($) $ / shares in Units, $ in Thousands	3 Months Ended
	Mar. 31, 2015	Mar. 31, 2014
Numerator
Net loss	$ (37,915)	$ (14,098)
Denominator
Denominator for basic and diluted net loss per share (in shares)	33,067,752	24,492,487
Net loss per share:
Basic and diluted (in dollars per share)	$ (1.15)	$ (0.58)

Net loss per share (Details 2) - shares	3 Months Ended
Net loss per share (Details 2) - shares	Mar. 31, 2015	Mar. 31, 2014
Net loss per share
Total shares excluded from calculation	4,992,803	3,882,286
Stock options
Net loss per share
Total shares excluded from calculation	4,627,833	3,133,830
Restricted stock
Net loss per share
Total shares excluded from calculation	364,970	748,456

Stock award plan (Details 3) - USD ($) $ in Thousands	3 Months Ended
Stock award plan (Details 3) - USD ($) $ in Thousands	Mar. 31, 2015	Mar. 31, 2014
Share-based compensation expense recorded in the statement of operations
Share-based compensation expense	$ 9,748	$ 3,705
Unrecognized compensation cost	$ 70,600
Weighted average remaining service period for recognition of unrecognized compensation cost	3 years
Research and development
Share-based compensation expense recorded in the statement of operations
Share-based compensation expense	$ 4,667	1,944
Selling, general and administrative
Share-based compensation expense recorded in the statement of operations
Share-based compensation expense	$ 5,081	$ 1,761