PTC Therapeutics (PTCT) 10-Q 9 Nov 15 2015 Q3 Quarterly report Financial data

Document and Entity Information

Document and Entity Information - shares	9 Months Ended
	Sep. 30, 2015	Nov. 04, 2015
Document and Entity Information
Entity Registrant Name	PTC THERAPEUTICS, INC.
Entity Central Index Key	1,070,081
Document Type	10-Q
Document Period End Date	Sep. 30, 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		34,271,694
Document Fiscal Year Focus	2,015
Document Fiscal Period Focus	Q3

Consolidated Balance Sheets

Consolidated Balance Sheets - USD ($) $ in Thousands	Sep. 30, 2015	Dec. 31, 2014
Current assets:
Cash and cash equivalents	$ 140,712	$ 49,748
Marketable securities	230,809	265,493
Prepaid expenses and other current assets	4,627	3,885
Receivables, net	8,016	4,445
Total current assets	384,164	323,571
Fixed assets, net	8,799	9,159
Deposits and other assets	3,137	489
Total assets	396,100	333,219
Current liabilities:
Accounts payable and accrued expenses	31,695	29,121
Deferred revenue		3,354
Total current liabilities	31,695	32,475
Long-term debt	93,198
Other long-term liabilities	2,056	2,277
Total liabilities	126,949	34,752
Stockholders' equity:
Common stock, $0.001 par value. Authorized 125,000,000 shares; issued and outstanding 33,915,059 shares at September 30, 2015. Authorized 125,000,000 shares; issued and outstanding 32,898,392 shares at December 31, 2014	34	33
Additional paid-in capital	812,294	721,722
Accumulated other comprehensive loss	(1,127)	(737)
Accumulated deficit	(542,050)	(422,551)
Total stockholders' equity	269,151	298,467
Total liabilities and stockholders' equity	$ 396,100	$ 333,219

Consolidated Balance Sheets (Pa

Consolidated Balance Sheets (Parenthetical) - $ / shares	Sep. 30, 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	33,915,059	32,898,392
Common stock, outstanding shares	33,915,059	32,898,392

Consolidated Statements of Oper

Consolidated Statements of Operations - USD ($) $ in Thousands	3 Months Ended		9 Months Ended
	Sep. 30, 2015	Sep. 30, 2014	Sep. 30, 2015	Sep. 30, 2014
Revenues:
Net product revenue	$ 9,772	$ 81	$ 21,002	$ 81
Collaboration revenue	2	716	547	11,280
Grant revenue	2	897	2,483	1,226
Total revenues	9,776	1,694	24,032	12,587
Operating expenses:
Research and development	30,640	18,765	86,768	52,967
Selling, general and administrative	21,368	10,530	56,193	26,803
Total operating expenses	52,008	29,295	142,961	79,770
Loss from operations	(42,232)	(27,601)	(118,929)	(67,183)
Interest (expense)/income, net	(852)	354	170	774
Other expense, net	(51)	(35)	(507)	(75)
Loss before income tax expense	(43,135)	(27,282)	(119,266)	(66,484)
Income tax expense	(88)		(233)
Net loss	$ (43,223)	$ (27,282)	$ (119,499)	$ (66,484)
Weighted-average shares outstanding:
Basic and diluted (in shares)	33,908,853	29,351,693	33,528,833	28,441,827
Net loss per share - basic and diluted (in dollars per share)	$ (1.27)	$ (0.93)	$ (3.56)	$ (2.34)

Consolidated Statements of Comp

Consolidated Statements of Comprehensive Loss - USD ($) $ in Thousands	3 Months Ended		9 Months Ended
	Sep. 30, 2015	Sep. 30, 2014	Sep. 30, 2015	Sep. 30, 2014
Consolidated Statements of Comprehensive Loss
Net loss	$ (43,223)	$ (27,282)	$ (119,499)	$ (66,484)
Other comprehensive loss:
Unrealized loss on marketable securities	(483)	(159)	(582)	(139)
Foreign currency translation (loss)/ gain	(149)		192
Comprehensive loss	$ (43,855)	$ (27,441)	$ (119,889)	$ (66,623)

Consolidated Statements of Cash

Consolidated Statements of Cash Flows - USD ($) $ in Thousands	9 Months Ended
	Sep. 30, 2015	Sep. 30, 2014
Cash flows from operating activities
Net loss	$ (119,499)	$ (66,484)
Adjustments to reconcile net loss to net cash used in operating activities:
Depreciation	2,079	1,693
Change in valuation of warrant liability	(152)	78
Non-cash interest expense	736
Amortization of debt issuance costs	37
Amortization of premiums on investments	1,319	1,225
Share-based compensation expense	26,130	12,605
Changes in operating assets and liabilities:
Prepaid expenses and other current assets	(748)	(1,232)
Receivables	(3,582)	(338)
Deposits and other assets	183	(937)
Accounts payable and accrued expenses	2,871	4,286
Other long-term liabilities	(69)	(68)
Deferred revenue	(3,310)	(109)
Net cash used in operating activities	(94,005)	(49,281)
Cash flows from investing activities
Purchases of fixed assets	(1,764)	(1,156)
Purchases of marketable securities	(134,299)	(132,602)
Sale & redemptions of marketable securities	167,082	101,775
Net cash provided by/(used in) investing activities	31,019	(31,983)
Cash flows from financing activities
Payments on long-term debt		(49)
Proceeds from exercise of options	8,689	389
Net proceeds from public offerings		118,383
Debt issuance costs related to convertible notes	(4,650)
Proceeds from issuance of convertible notes	150,000
Net cash provided by financing activities	154,039	118,723
Effect of exchange rate changes on cash	(89)
Net increase in cash and cash equivalents	90,964	37,459
Cash and cash equivalents, beginning of period	49,748	15,414
Cash and cash equivalents, end of period	140,712	52,873
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 loss on marketable securities	$ (582)	$ (139)

The Company

The Company	9 Months Ended
	Sep. 30, 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 five 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 submission of the final report, including additional safety and efficacy data, from its global, confirmatory Phase 3 clinical trial in nmDMD, which it refers to as ACT DMD, during 2015. See “Risk Factors - Risks Related to Regulatory Approval of our Product and our Product Candidates” on page 61 for further detail regarding the annual EMA reassessment process, including a description of the risk-benefit balance. In October 2015, the Company announced results from ACT DMD, including that the trial did not meet the primary efficacy endpoint of change from baseline at week 48 in distance walked in the 6-minute walk test, or 6MWT, which we also refer to as six-minute walk distance, or 6MWD, in the overall intent-to-treat, or ITT, study population, as it showed a 15 meter benefit (p=0.213) in favor of Translarna compared to placebo, which was not statistically significant. Analyses of data from pre-specified subgroups, including patients with baseline 6MWD of 300 - 400 meters, was also conducted. A pre-specified meta-analysis was also performed of combined data from the ACT DMD and ambulatory decline phase patients from the Company’s randomized, double-blind, placebo controlled, Phase 2b clinical trial evaluating the long-term safety and efficacy of Translarna in patients with nmDMD, or the Phase 2b trial. For further discussion of ACT DMD, see “Recent Developments” in Item 2. Management’s Discussion and Analysis of Financial Condition and Results of Operations.” See “Risk Factors - Risks Related to the Development and Commercialization of our Product and Product Candidates” on page 40 for further detail regarding how ACT DMD results could impact our ability to commercialize Translarna. The Company launched Translarna on a commercial basis in Germany in December 2014 and in Austria, Denmark, Norway, and Sweden in 2015. The Company expects to expand its launch activities across the EEA in the fourth quarter of 2015 and in future years, subject to successful completion of pricing and reimbursement negotiations. The market access process, including pricing and reimbursement negotiations, varies from country to country and can take over 18 months in certain circumstances. Concurrently, the Company has been making Translarna available under 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. During 2015, the Company’s revenues have been and are expected to be primarily generated from sales of Translarna in counties in the EEA where pricing and reimbursement approval is obtained at acceptable levels and in other territories where permitted to distribute Translarna under early access programs, or EAPs. The Company 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, 2015, the Company had an accumulated deficit of approximately $542.1 million. The Company has financed its operations to date primarily through the private offering in August 2015 of 3.00% convertible senior notes due 2022 (see Note 9), 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.

Summary of significant accounti

Summary of significant accounting policies	9 Months Ended
	Sep. 30, 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 September 30, 2015 and for the three and nine months ended September 30, 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 September 30, 2015 and for the three and nine months ended September 30, 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 and nine month periods ended September 30, 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 nmDMD in ambulatory patients aged five years and older. The marketing authorization allows the Company to market Translarna in the EEA. The launch in these countries is on a country by country basis. 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 submission of the final report, including additional efficacy and safety data, from ACT DMD during 2015. In the third quarter of 2015, the EMA approved the annual renewal of the marketing authorization for Translarna. If we fail to satisfy renewal requirements, or if it is determined that the balance of risks and benefits of using Translarna changes materially, the European Commission could, at the EMA’s recommendation, vary, suspend, withdraw or refuse to renew the marketing authorization for Translarna or require additional clinical trials. The Company does 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 consolidated statements of operations. The Company’s cost of product sales includes royalties and other miscellaneous selling costs, which were not material and therefore were included as a component of research and development costs in the current period 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 standards 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. In July 2015, the Financial Accounting Standards Board voted to delay the effective date of this standard until the first quarter of 2018. Companies are permitted to early adopt the standard in the first quarter of 2017. 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 its financial statements and accompanying notes. In April 2015, the FASB issued an amendment to U.S. GAAP to simplify the balance sheet presentation of the costs for issuing debt. The changes were adopted in ASU No. 2015-03, “Interest - Imputation of Interest (Subtopic 835-30): Simplifying the Presentation of Debt Issue Costs”. Public companies will have to apply the amendments for reporting periods that begin after December 15, 2015. This amendment requires adoption by revising the balance sheets for periods prior to the effective date. The Company is currently evaluating the impact of this ASU and does not believe the adoption of this ASU will have a material impact on its financial statements and accompanying notes. 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 instrum

Fair value of financial instruments and marketable securities	9 Months Ended
	Sep. 30, 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 this 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, 2015 and December 31, 2014: September 30, 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 $ $ — $ $ — Warrant liability — — 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 $ $ — $ $ — Warrant Liability — — The following is a summary of marketable securities accounted for as available-for-sale securities at September 30, 2015 and December 31, 2014: September 30, 2015 Amortized Gross Unrealized Fair Cost Gains Losses Value Corporate debt securities $ $ $ ) $ Government obligations ) $ $ $ ) $ December 31, 2014 Amortized Gross Unrealized Fair Cost Gains Losses Value Corporate debt securities $ $ $ ) $ Government obligations ) $ $ $ ) $ At September 30, 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 September 30, 2015 and December 31, 2014 mature as follows: September 30, 2015 Less Than 12 Months More Than 12 Months Corporate debt securities $ $ Government obligations Total Marketable securities $ $ December 31, 2014 Less Than 12 Months More Than 12 Months Corporate debt securities $ $ Government obligations Total Marketable securities $ $ Level 3 valuation The warrant liability is classified in Other long-term liabilities on the Company’s consolidated 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 expense, net on the Company’s consolidated 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, 2015: Level 3 assets Beginning balance as of December 31, 2014 $ Change in fair value of warrant liability ) Ending balance as of September 30, 2015 $ 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, 2015 include (i) volatility (62%—70%), (ii) risk free interest rate (0.49%—1.15%), (iii) strike price ($128.00-$2,520.00), (iv) fair value of common stock ($26.70), and (v) expected life (1.71—3.98 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 ($128.00—$2,520.00), (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.

Other comprehensive loss and ac

Other comprehensive loss and accumulated other comprehensive items	9 Months Ended
	Sep. 30, 2015
Other comprehensive loss and accumulated other comprehensive items
Other comprehensive loss and accumulated other comprehensive items	4. Other comprehensive 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, 2015: Unrealized Gains/(Losses) On Marketable Securities Foreign Currency Translation Total Accumulated Other Comprehensive Items Balance at June 30, 2015 $ ) $ ) $ ) Other comprehensive loss before reclassifications ) ) ) Amounts reclassified from other comprehensive items — — — Other comprehensive loss ) ) ) Balance at September 30, 2015 $ ) $ ) $ ) Unrealized Gains/(Losses) On Marketable Securities Foreign Currency Translation Total Accumulated Other Comprehensive Items Balance at December 31, 2014 $ ) $ ) $ ) Other comprehensive income/(loss) before reclassifications ) ) Amounts reclassified from other comprehensive items — — — Other comprehensive income/(loss) ) ) Balance at September 30, 2015 $ ) $ ) $ )

Accounts payable and accrued ex

Accounts payable and accrued expenses	9 Months Ended
	Sep. 30, 2015
Accounts payable and accrued expenses
Accounts payable and accrued expenses	5. Accounts payable and accrued expenses Accounts payable and accrued expenses at September 30, 2015 and December 31, 2014 consist of the following: September 30, 2015 December 31, 2014 Employee compensation, benefits, and related accruals $ $ Consulting and contracted research Professional fees Accounts payable Other $ $

Warrants

Warrants	9 Months Ended
	Sep. 30, 2015
Warrants.
Warrants	6. Warrants All of the Company’s outstanding warrants were classified as liabilities as of September 30, 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 September 30, 2015 and December 31, 2014: Warrant shares Exercise price Expiration Common stock $ Common stock $ 2019 and 2020 Common stock $

Net loss per share

Net loss per share	9 Months Ended
	Sep. 30, 2015
Net loss per share
Net loss per share	7. Net loss per share Basic earnings per share is computed by dividing net loss by the weighted-average number of common shares outstanding. Diluted earnings per share is computed by dividing net 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 September 30, Nine Months Ended September 30, 2015 2014 2015 2014 Numerator Net loss $ ) $ ) $ ) $ ) Denominator Denominator for basic and diluted net loss per share Net loss per share: Basic and diluted $ )* $ )* $ )* $ )* *In the three and nine months ended September 30, 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 September 30, 2015 2014 Stock Options Unvested restricted stock Total In August 2015, the Company issued, at par value, $150.0 million aggregate principal amount of 3.0% convertible senior notes due 2022 (the Convertible Notes) (see Note 9, Convertible Senior Notes). The conversion rate for the Convertible Notes was initially, and remains 17.7487 shares of the Company’s common stock per $1,000 principal amount of the Convertible Notes, which is equivalent to an initial conversion price of approximately $56.34 per share of the Company’s common stock. For the three and nine months ended September 30, 2015, there was no dilutive effect of the Convertible Notes as the stock price did not exceed the conversion price and the Company reported a loss.

Stock award plan

Stock award plan	9 Months Ended
	Sep. 30, 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) 238,427 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 September 30, 2015, the Company issued a total of 2,086,300 stock options to various employees. Of those, 691,100 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 $ Granted $ Exercised ) $ Forfeited ) $ Expired ) $ Outstanding at September 30, 2015 $ 8.58 years $ Vested or Expected to vest at September 30, 2015 $ 8.56 years $ Exercisable at September 30, 2015 $ 7.89 years $ The fair value of grants made in the nine months ended September 30, 2015 was contemporaneously estimated on the date of grant using the following assumptions: Nine months ended September 30, 2015 Risk-free interest rate 1.49% — 2.01% 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 nine month period ended September 30, 2015 was $32.32 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 $ Granted — $ — Vested ) $ Forfeited ) $ Unvested at September 30, 2015 $ The Company recorded share-based compensation expense in the statement of operations as follows: Three Months Ended September 30, Nine Months Ended September 30, 2015 2014 2015 2014 Research and development $ $ $ $ General and administrative Total $ $ $ $ As of September 30, 2015, there was approximately $75.2 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 2.9 years.

Convertible Senior Notes

Convertible Senior Notes	9 Months Ended
	Sep. 30, 2015
Convertible Senior Notes.
Convertible Senior Notes	9. Convertible Senior Notes In August 2015, the Company issued, at par value, $150.0 million aggregate principal amount of 3.0% convertible senior notes due 2022. The Convertible Notes bear cash interest at a rate of 3.0% per year, payable semi-annually on February 15 and August 15 of each year, beginning on February 15, 2016. The Convertible Notes will mature on August 15, 2022, unless earlier repurchased or converted. The net proceeds to the Company from the offering were $145.4 million after deducting the initial purchasers’ discounts and commissions and the offering expenses payable by the Company. The Convertible Notes are governed by an indenture (the Convertible Notes Indenture) with U.S Bank National Association as trustee (the Convertible Notes Trustee). Holders may convert their Convertible Notes at their option at any time prior to the close of business on the business day immediately preceding February 15, 2022 only under the following circumstances: · during any calendar quarter commencing on or after September 30, 2015 (and only during such calendar quarter), if the last reported sale price of the Company’s common stock for at least 20 trading days (whether or not consecutive) during a period of 30 consecutive trading days ending on the last trading day of the immediately preceding calendar quarter is greater than or equal to 130% of the conversion price on each applicable trading day; · during the five business day period after any five consecutive trading day period (the “measurement period”) in which the trading price (as defined in the Convertible Notes Indenture) per $1,000 principal amount of Convertible Notes for each trading day of the measurement period was less than 98% of the product of the last reported sale price of the Company’s common stock and the conversion rate on each such trading day; · during any period after the Company has issued notice of redemption until the close of business on the scheduled trading day immediately preceding the relevant redemption date; or · upon the occurrence of specified corporate events. On or after February 15, 2022, until the close of business on the business day immediately preceding the maturity date, holders may convert their Convertible Notes at any time, regardless of the foregoing circumstances. Upon conversion, the Company will pay cash up to the aggregate principal amount of the Convertible Notes to be converted and deliver shares of its common stock in respect of the remainder, if any, of its conversion obligation in excess of the aggregate principal amount of Convertible Notes being converted. The conversion rate for the Convertible Notes was initially, and remains, 17.7487 shares of the Company’s common stock per $1,000 principal amount of the Convertible Notes, which is equivalent to an initial conversion price of approximately $56.34 per share of the Company’s common stock. The Company may not redeem the Convertible Notes prior to August 20, 2018. The Company may redeem for cash all or any portion of the Convertible Notes, at its option, on or after August 20, 2018 if the last reported sale price of its common stock has been at least 130% of the conversion price then in effect on the last trading day of, and for at least 19 other trading days (whether or not consecutive) during, any 30 consecutive trading day period ending on, and including, the trading day immediately preceding the date on which the Company provides notice of redemption, at a redemption price equal to 100% of the principal amount of the Convertible Notes to be redeemed, plus accrued and unpaid interest to, but excluding, the redemption date. No sinking fund is provided for the Convertible Notes, which means that the Company is not required to redeem or retire the Convertible Notes periodically. If the Company undergoes a “fundamental change” (as defined in the Indenture governing the Convertible Notes Indenture), subject to certain conditions, holders of the Convertible Notes may require the Company to repurchase for cash all or part of their Convertible Notes at a repurchase price equal to 100% of the principal amount of the Convertible Notes to be repurchased, plus accrued and unpaid interest to, but excluding, the fundamental change repurchase date. The Convertible Notes Indenture contains customary events of default with respect to the Convertible Notes, including that upon certain events of default (including the Company’s failure to make any payment of principal or interest on the Convertible Notes when due and payable) occurring and continuing, the Convertible Notes Trustee by notice to the Company, or the holders of at least 25% in principal amount of the outstanding Convertible Notes by notice to the Company and the Convertible Notes Trustee, may, and the Convertible Notes Trustee at the request of such holders (subject to the provisions of the Convertible Notes Indenture) shall, declare 100% of the principal of and accrued and unpaid interest, if any, on all the Convertible Notes to be due and payable. In case of certain events of bankruptcy, insolvency or reorganization, involving the Company or a significant subsidiary, 100% of the principal of and accrued and unpaid interest on the Convertible Notes will automatically become due and payable. Upon such a declaration of acceleration, such principal and accrued and unpaid interest, if any, will be due and payable immediately. The Company accounts for the Convertible Notes as a liability and equity component where the carrying value of the liability component will be valued based on a similar instrument. In accounting for the issuance of the Convertible Notes, the Company separated the Convertible Notes into liability and equity components. The carrying amount of the liability component was calculated by measuring the fair value of a similar liability that does not have an associated convertible feature. The carrying amount of the equity component representing the conversion option was determined by deducting the fair value of the liability component from the par value of the Convertible Notes as a whole. The excess of the principal amount of the liability component over its carrying amount, referred to as the debt discount, is amortized to interest expense over the seven-year term of the Convertible Notes. The equity component is not re-measured as long as it continues to meet the conditions for equity classification. In accounting for the transaction costs related to the issuance of the Convertible Notes, the Company allocated the total costs incurred to the liability and equity components of the Convertible Notes based on their relative values. Transaction costs attributable to the liability component are amortized to interest expense over the seven-year term of the Convertible Notes, and transaction costs attributable to the equity component are netted with the equity components in stockholders’ equity. Additionally, the Company initially recorded a net deferred tax liability of $22.3 million in connection with the Notes. The Convertible Notes consist of the following: Liability component September 30, 2015 December 31, 2014 (in thousands) Principal $ $ — Less: Debt discount, net(1) ) — Net carrying amount $ $ — (1) Included in the consolidated balance sheets within convertible senior notes (due 2022) and amortized to interest expense over the remaining life of the Convertible Notes using the effective interest rate method. The fair value of the Convertible Notes was approximately $93.2 million as of September 30, 2015. The Company estimates the fair value of its Convertible Notes utilizing market quotations for debt that have quoted prices in active markets. Since the Convertible Notes do not trade on a daily basis in an active market, the fair value estimates are based on market observable inputs based on borrowing rates currently available for debt with similar terms and average maturities (Level 2). As of September 30, 2015, the remaining contractual life of the Convertible Notes is approximately 6.9 years. The following table sets forth total interest expense recognized related to the Convertible Notes: Three Months Ended September 30, 2015 2014 (in thousands) Contractual interest expense $ $ — Amortization of debt issuance costs — Amortization of debt discount — Total $ $ — Effective interest rate of the liability component % —

Subsequent events

Subsequent events	9 Months Ended
	Sep. 30, 2015
Subsequent events
Subsequent events	10. Subsequent events The Company has evaluated all subsequent events and transactions through the filing date. There were no material events that impacted the unaudited consolidated financial statements or disclosures.

Summary of significant accoun17

Summary of significant accounting policies (Policies)	9 Months Ended
	Sep. 30, 2015
Summary of significant accounting policies
Basis of presentation	Basis of Presentation The accompanying financial information as of September 30, 2015 and for the three and nine months ended September 30, 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 September 30, 2015 and for the three and nine months ended September 30, 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 and nine month periods ended September 30, 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 nmDMD in ambulatory patients aged five years and older. The marketing authorization allows the Company to market Translarna in the EEA. The launch in these countries is on a country by country basis. 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 submission of the final report, including additional efficacy and safety data, from ACT DMD during 2015. In the third quarter of 2015, the EMA approved the annual renewal of the marketing authorization for Translarna. If we fail to satisfy renewal requirements, or if it is determined that the balance of risks and benefits of using Translarna changes materially, the European Commission could, at the EMA’s recommendation, vary, suspend, withdraw or refuse to renew the marketing authorization for Translarna or require additional clinical trials. The Company does 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 consolidated statements of operations. The Company’s cost of product sales includes royalties and other miscellaneous selling costs, which were not material and therefore were included as a component of research and development costs in the current period 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 standards	Recently issued accounting standards 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. In July 2015, the Financial Accounting Standards Board voted to delay the effective date of this standard until the first quarter of 2018. Companies are permitted to early adopt the standard in the first quarter of 2017. 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 its financial statements and accompanying notes. In April 2015, the FASB issued an amendment to U.S. GAAP to simplify the balance sheet presentation of the costs for issuing debt. The changes were adopted in ASU No. 2015-03, “Interest - Imputation of Interest (Subtopic 835-30): Simplifying the Presentation of Debt Issue Costs”. Public companies will have to apply the amendments for reporting periods that begin after December 15, 2015. This amendment requires adoption by revising the balance sheets for periods prior to the effective date. The Company is currently evaluating the impact of this ASU and does not believe the adoption of this ASU will have a material impact on its financial statements and accompanying notes.
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.

Fair value of financial instr18

Fair value of financial instruments and marketable securities (Tables)	9 Months Ended
	Sep. 30, 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	September 30, 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 $ $ — $ $ — Warrant liability — — 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 $ $ — $ $ — Warrant Liability — —
Summary of marketable securities accounted for as available-for-sale securities	September 30, 2015 Amortized Gross Unrealized Fair Cost Gains Losses Value Corporate debt securities $ $ $ ) $ Government obligations ) $ $ $ ) $ December 31, 2014 Amortized Gross Unrealized Fair Cost Gains Losses Value Corporate debt securities $ $ $ ) $ Government obligations ) $ $ $ ) $
Schedule of marketable securities on the balance sheet	September 30, 2015 Less Than 12 Months More Than 12 Months Corporate debt securities $ $ Government obligations Total Marketable securities $ $ December 31, 2014 Less Than 12 Months More Than 12 Months Corporate debt securities $ $ Government obligations Total Marketable securities $ $
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 $ Change in fair value of warrant liability ) Ending balance as of September 30, 2015 $

Other comprehensive loss and 19

Other comprehensive loss and accumulated other comprehensive items (Tables)	9 Months Ended
	Sep. 30, 2015
Other comprehensive loss and accumulated other comprehensive items
Summary of other comprehensive income (loss) and the changes in accumulated other comprehensive items	Unrealized Gains/(Losses) On Marketable Securities Foreign Currency Translation Total Accumulated Other Comprehensive Items Balance at June 30, 2015 $ ) $ ) $ ) Other comprehensive loss before reclassifications ) ) ) Amounts reclassified from other comprehensive items — — — Other comprehensive loss ) ) ) Balance at September 30, 2015 $ ) $ ) $ ) Unrealized Gains/(Losses) On Marketable Securities Foreign Currency Translation Total Accumulated Other Comprehensive Items Balance at December 31, 2014 $ ) $ ) $ ) Other comprehensive income/(loss) before reclassifications ) ) Amounts reclassified from other comprehensive items — — — Other comprehensive income/(loss) ) ) Balance at September 30, 2015 $ ) $ ) $ )

Accounts payable and accrued 20

Accounts payable and accrued expenses (Tables)	9 Months Ended
	Sep. 30, 2015
Accounts payable and accrued expenses
Schedule of components of accounts payable and accrued expenses	September 30, 2015 December 31, 2014 Employee compensation, benefits, and related accruals $ $ Consulting and contracted research Professional fees Accounts payable Other $ $

Warrants (Tables)

Warrants (Tables)	9 Months Ended
	Sep. 30, 2015
Warrants.
Summary of the Company's outstanding warrants	The following is a summary of the Company’s outstanding warrants as of September 30, 2015 and December 31, 2014: Warrant shares Exercise price Expiration Common stock $ Common stock $ 2019 and 2020 Common stock $

Net loss per share (Tables)

Net loss per share (Tables)	9 Months Ended
	Sep. 30, 2015
Net loss per share
Schedule of computation of basic and diluted net loss per share for common stockholders	Three Months Ended September 30, Nine Months Ended September 30, 2015 2014 2015 2014 Numerator Net loss $ ) $ ) $ ) $ ) Denominator Denominator for basic and diluted net loss per share Net loss per share: Basic and diluted $ )* $ )* $ )* $ )* * In the three and nine months ended September 30, 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 September 30, 2015 2014 Stock Options Unvested restricted stock Total

Stock award plan (Tables)

Stock award plan (Tables)	9 Months Ended
	Sep. 30, 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 $ Granted $ Exercised ) $ Forfeited ) $ Expired ) $ Outstanding at September 30, 2015 $ 8.58 years $ Vested or Expected to vest at September 30, 2015 $ 8.56 years $ Exercisable at September 30, 2015 $ 7.89 years $
Schedule of assumptions used to estimate fair value of grants made on the date of grant	Nine months ended September 30, 2015 Risk-free interest rate 1.49% — 2.01% 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 $ Granted — $ — Vested ) $ Forfeited ) $ Unvested at September 30, 2015 $
Schedule of share-based compensation expense recorded in the statement of operations	Three Months Ended September 30, Nine Months Ended September 30, 2015 2014 2015 2014 Research and development $ $ $ $ General and administrative Total $ $ $ $

Convertible Senior Notes (Table

Convertible Senior Notes (Tables)	9 Months Ended
	Sep. 30, 2015
Convertible Senior Notes.
Summary of convertible notes	Liability component September 30, 2015 December 31, 2014 (in thousands) Principal $ $ — Less: Debt discount, net(1) ) — Net carrying amount $ $ — (1) Included in the consolidated balance sheets within convertible senior notes (due 2022) and amortized to interest expense over the remaining life of the Convertible Notes using the effective interest rate method.
Summary of interest expense recognized related to the Convertible Notes	Three Months Ended September 30, 2015 2014 (in thousands) Contractual interest expense $ $ — Amortization of debt issuance costs — Amortization of debt discount — Total $ $ — Effective interest rate of the liability component % —

The Company (Details)

The Company (Details) $ in Thousands	Aug. 04, 2014	Aug. 31, 2014state	Sep. 30, 2015USD ($)	Aug. 31, 2015	Dec. 31, 2014USD ($)
Minimum age of ambulatory patient	5 years	5 years
Number of member states of the European Economic Area		31
Period of market access for launching new product			18 months
Accumulated deficit | $			$ (542,050)		$ (422,551)
Convertible debt | 3.0% Convertible senior notes due 2022
Interest rate				3.00%

Summary of significant accoun26

Summary of significant accounting policies (Details)	Aug. 04, 2014	Aug. 31, 2014
Summary of significant accounting policies
Minimum age of ambulatory patient	5 years	5 years

Fair value of financial instr27

Fair value of financial instruments and marketable securities (Details) - USD ($) $ in Thousands	Sep. 30, 2015	Dec. 31, 2014
Financial assets and liabilities measured at fair value on recurring basis
Marketable securities	$ 230,809	$ 265,493
Recurring basis | Total
Financial assets and liabilities measured at fair value on recurring basis
Marketable securities	230,809	265,493
Warrant liability	36	188
Recurring basis | Significant other observable inputs (level 2)
Financial assets and liabilities measured at fair value on recurring basis
Marketable securities	230,809	265,493
Recurring basis | Significant unobservable inputs (level 3)
Financial assets and liabilities measured at fair value on recurring basis
Warrant liability	$ 36	$ 188

Fair value of financial instr28

Fair value of financial instruments and marketable securities (Details 2) - USD ($) $ in Thousands	Sep. 30, 2015	Dec. 31, 2014
Marketable securities accounted for as available-for-sale securities
Amortized Cost	$ 231,777	$ 265,880
Gross Unrealized Gains	69	87
Gross Unrealized Losses	(1,037)	(474)
Fair Value	230,809	265,493
Corporate debt securities
Marketable securities accounted for as available-for-sale securities
Amortized Cost	198,102	230,379
Gross Unrealized Gains	52	80
Gross Unrealized Losses	(987)	(428)
Fair Value	197,167	230,031
Government obligations
Marketable securities accounted for as available-for-sale securities
Amortized Cost	33,675	35,501
Gross Unrealized Gains	17	7
Gross Unrealized Losses	(50)	(46)
Fair Value	$ 33,642	$ 35,462

Fair value of financial instr29

Fair value of financial instruments and marketable securities (Details 3) - USD ($) $ in Thousands	Sep. 30, 2015	Dec. 31, 2014
Marketable securities on the balance sheet
Total Marketable securities, Less Than 12 Months	$ 164,120	$ 163,761
Total Marketable securities, More Than 12 Months	66,689	101,732
Corporate debt securities
Marketable securities on the balance sheet
Total Marketable securities, Less Than 12 Months	144,846	157,758
Total Marketable securities, More Than 12 Months	52,321	72,273
Government obligations
Marketable securities on the balance sheet
Total Marketable securities, Less Than 12 Months	19,274	6,003
Total Marketable securities, More Than 12 Months	$ 14,368	$ 29,459

Fair value of financial instr30

Fair value of financial instruments and marketable securities (Details 4) - Warrants - USD ($) $ / shares in Units, $ in Thousands	9 Months Ended	12 Months Ended
	Sep. 30, 2015	Dec. 31, 2014
Changes in the fair value of warrant liability
Beginning balance	$ 188
Change in fair value of warrant liability	(152)
Ending balance	$ 36	$ 188
Minimum
Assumption used to estimate the fair value of warrant liability by utilizing the Black-Scholes option-pricing model
Volatility (as a percent)	62.00%	68.00%
Risk-free interest rate (as a percent)	0.49%	0.89%
Strike price (in dollars per share)	$ 128	$ 128
Expected life	1 year 8 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)	1.15%	1.65%
Strike price (in dollars per share)	$ 2,520	$ 2,520
Expected life	3 years 11 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)	$ 26.70	$ 51.77

Other comprehensive loss and 31

Other comprehensive loss and accumulated other comprehensive items (Details) - USD ($) $ in Thousands	3 Months Ended	9 Months Ended
	Sep. 30, 2015	Sep. 30, 2015
Other comprehensive income (loss) and accumulated other comprehensive items
Balance at the beginning of the period	$ (495)	$ (737)
Other comprehensive income/(loss) before reclassifications	(632)	(390)
Other comprehensive income/(loss)	(632)	(390)
Balance at the end of the period	(1,127)	(1,127)
Unrealized Gains/(Losses) On Marketable Securities
Other comprehensive income (loss) and accumulated other comprehensive items
Balance at the beginning of the period	(486)	(387)
Other comprehensive income/(loss) before reclassifications	(483)	(582)
Other comprehensive income/(loss)	(483)	(582)
Balance at the end of the period	(969)	(969)
Foreign Currency Translation
Other comprehensive income (loss) and accumulated other comprehensive items
Balance at the beginning of the period	(9)	(350)
Other comprehensive income/(loss) before reclassifications	(149)	192
Other comprehensive income/(loss)	(149)	192
Balance at the end of the period	$ (158)	$ (158)

Accounts payable and accrued 32

Accounts payable and accrued expenses (Details) - USD ($) $ in Thousands	Sep. 30, 2015	Dec. 31, 2014
Accounts payable and accrued expenses
Employee compensation, benefits, and related accruals	$ 7,744	$ 9,312
Consulting and contracted research	9,089	9,349
Professional fees	2,975	3,334
Accounts payable	8,715	4,128
Other	3,172	2,998
Accounts payable and accrued expenses	$ 31,695	$ 29,121

Warrants (Details)

Warrants (Details) - Warrants - Common stock - $ / shares	Sep. 30, 2015	Dec. 31, 2014
2,017
Warrants
Warrant shares	6,250	6,250
Exercise price (in dollars per share)	$ 128	$ 128
2019 and 2020
Warrants
Warrant shares	7,030	7,030
Exercise price (in dollars per share)	$ 128	$ 128
2,019
Warrants
Warrant shares	130	130
Exercise price (in dollars per share)	$ 2,520	$ 2,520

Net loss per share (Details)

Net loss per share (Details) - USD ($) $ / shares in Units, $ in Thousands	3 Months Ended		9 Months Ended
	Sep. 30, 2015	Sep. 30, 2014	Sep. 30, 2015	Sep. 30, 2014
Numerator
Net loss	$ (43,223)	$ (27,282)	$ (119,499)	$ (66,484)
Denominator
Denominator for basic and diluted net loss per share (in shares)	33,908,853	29,351,693	33,528,833	28,441,827
Net loss per share:
Basic and diluted (in dollars per share)	$ (1.27)	$ (0.93)	$ (3.56)	$ (2.34)

Net loss per share (Details 2)

Net loss per share (Details 2)	1 Months Ended	9 Months Ended
	Aug. 31, 2015USD ($)$ / shares	Sep. 30, 2015USD ($)shares	Sep. 30, 2014shares
Net loss per share
Total shares excluded from calculation		5,141,399	4,173,098
Stock options
Net loss per share
Total shares excluded from calculation		4,788,264	3,443,778
Unvested restricted stock
Net loss per share
Total shares excluded from calculation		353,135	729,320
Convertible debt | 3.0% Convertible senior notes due 2022
Convertible Senior Notes.
Principal amount of Notes | $	$ 150,000,000	$ 150,000,000
Interest rate	3.00%
Conversion ratio	17.7487
Common stock per principal amount | $	$ 1,000
Conversion price per share | $ / shares	$ 56.34

Stock award plan (Details)

Stock award plan (Details) - USD ($) $ / shares in Units, $ in Thousands	Mar. 05, 2013	May. 31, 2013	Sep. 30, 2015
Stock option
Number of options
Outstanding at the beginning of the period (in shares)			3,432,972
Granted (in shares)			2,086,300
Exercised (in shares)			(654,748)
Forfeited (in shares)			(74,366)
Expired (in shares)			(1,894)
Outstanding at the end of the period (in shares)			4,788,264
Vested or expected to vest at the end of the period (in shares)			4,497,155
Exercisable at the end of the period (in shares)			1,132,683
Weighted-average exercise price
Outstanding at the beginning of the period (in dollars per share)			$ 25
Granted (in dollars per share)			52.04
Exercised (in dollars per share)			13.27
Forfeited (in dollars per share)			42.46
Expired (in dollars per share)			285.48
Outstanding at the end of the period (in dollars per share)			37.64
Vested or expected to vest at the end of the period (in dollars per share)			37
Exercisable at the end of the period (in dollars per share)			$ 32.21
Weighted-average remaining contractual term
Outstanding at the end of the period			8 years 6 months 29 days
Vested or expected to vest at the end of the period			8 years 6 months 22 days
Exercisable at the end of the period			7 years 10 months 21 days
Aggregate intrinsic value
Outstanding at the end of the period (in dollars)			$ 20,137
Vested or expected to vest at the end of the period (in dollars)			19,018
Exercisable at the end of the period (in dollars)			$ 10,312
Valuation assumptions
Inducement grants for non-statutory stock options			691,100
Expected dividend yield (as a percent)			0.00%
Weighted average grant date fair value (in dollars per share)			$ 32.32
Stock option | Minimum
Valuation assumptions
Risk-free interest rate (as a percent)			1.49%
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)			2.01%
Expected volatility (as a percent)			69.00%
Expected term			6 years 1 month 10 days
2013 Stock Incentive Plan
Stock option plan
Number of shares available for issuance	0
2013 Stock Incentive Plan | Unvested restricted stock
Stock option plan
Number of shares of restricted stock granted	735,324
2013 Stock Incentive Plan | Stock option
Stock option plan
Number of shares of restricted stock granted	4,613
2013 Stock Incentive Plan | Common stock
Stock option plan
Number of shares authorized	739,937
2009 Equity and Long Term Incentive Plan
Stock option plan
Number of additional shares authorized		2,500,000
2009 Equity and Long Term Incentive Plan and 2013 Stock Incentive Plan | Common stock
Stock option plan
Number of shares available for issuance		238,427
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)

Stock award plan (Details 2) - Unvested restricted stock	9 Months Ended
	Sep. 30, 2015$ / sharesshares
Number of Shares
Balance at the beginning of the period (in shares) | shares	718,400
Vested (in shares) | shares	(361,919)
Forfeited (in shares) | shares	(3,346)
Balance at the end of the period (in shares) | shares	353,135
Weighted Average Grant Date Fair Value
Balance at the beginning of the period (in dollars per share)	$ 10.72
Vested (in dollars per share)	10.60
Forfeited (in dollars per share)	10.82
Balance at the end of the period (in dollars per share)	$ 10.85

Stock award plan (Details 3)

Stock award plan (Details 3) - USD ($) $ in Thousands	3 Months Ended		9 Months Ended
	Sep. 30, 2015	Sep. 30, 2014	Sep. 30, 2015	Sep. 30, 2014
Share-based compensation expense recorded in the statement of operations
Share-based compensation expense	$ 8,054	$ 4,621	$ 26,130	$ 12,605
Unrecognized compensation cost	75,200		$ 75,200
Weighted average remaining service period for recognition of unrecognized compensation cost			2 years 10 months 24 days
Research and development
Share-based compensation expense recorded in the statement of operations
Share-based compensation expense	3,828	2,363	$ 12,452	6,517
General and administrative
Share-based compensation expense recorded in the statement of operations
Share-based compensation expense	$ 4,226	$ 2,258	$ 13,678	$ 6,088

Convertible Senior Notes (Detai

Convertible Senior Notes (Details)	1 Months Ended	3 Months Ended	9 Months Ended
	Aug. 31, 2015USD ($)item$ / shares	Sep. 30, 2015USD ($)	Sep. 30, 2015USD ($)
Net carrying amount		$ 93,198,000	$ 93,198,000
Amortization of debt issuance costs			37,000
3.0% Convertible senior notes due 2022 | Convertible debt
Principal amount of Notes	$ 150,000,000	150,000,000	150,000,000
Less: Debt discount, net		(56,802,000)	(56,802,000)
Net carrying amount		93,198,000	93,198,000
Interest rate	3.00%
Net proceeds from issuance of convertible notes	$ 145,400,000
Trading days, number | item	20
Consecutive trading days, period	30 days
Stock price trigger (as a percent)	130.00%
Business days, period	5 days
Consecutive trading-day period	5 days
Common stock per principal amount	$ 1,000
Maximum product of the closing sale price of shares of the Company's common stock and the applicable conversion rate for such trading day (as a percent)	98.00%
Conversion ratio	17.7487
Conversion price per share | $ / shares	$ 56.34
Convertible debt instrument holders required to issue notice for declaration of principal and unpaid interest payable upon events of default (as a percent of principal amount by instrument holders)	25
Convertible Instruments Principal And Unpaid Interest Payable Upon Events Of Default	100.00%
Term of the convertible notes	7 years
Net deferred tax liability in connection with convertible notes	$ 22,300,000
Fair value of convertible notes		93,200,000	$ 93,200,000
Remaining contractual life of the convertible notes			6 years 10 months 24 days
Contractual interest expense		584,000
Amortization of debt issuance costs		37,000
Amortization of debt discount		736,000
Total		$ 1,357,000
Effective interest rate of the liability component (as a percent)		11.00%	11.00%
3.0% Convertible senior notes due 2022 | Convertible debt | Redemption on or after August 20, 2018
Trading days, number | item	19
Consecutive trading days, period	30 days
Stock price trigger (as a percent)	130.00%
Redemption price (as a percent)	100.00%