Document_and_Entity_Informatio
Document and Entity Information | 3 Months Ended | |
Mar. 31, 2015 | Apr. 30, 2015 | |
Document and Entity Information | ||
Entity Registrant Name | PTC THERAPEUTICS, INC. | |
Entity Central Index Key | 1070081 | |
Document Type | 10-Q | |
Document Period End Date | 31-Mar-15 | |
Amendment Flag | FALSE | |
Current Fiscal Year End Date | -19 | |
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
Consolidated Balance Sheets (USD $) | Mar. 31, 2015 | Dec. 31, 2014 |
In Thousands, unless otherwise specified | ||
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_Balance_Sheets_Pa
Consolidated Balance Sheets (Parenthetical) (USD $) | Mar. 31, 2015 | Dec. 31, 2014 |
Consolidated Balance Sheets | ||
Common stock, par value (in dollars per share) | $0.00 | $0.00 |
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 |
Consolidated_Statements_of_Ope
Consolidated Statements of Operations (USD $) | 3 Months Ended | |
In Thousands, except Share data, unless otherwise specified | 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_Com
Consolidated Statements of Comprehensive Loss (USD $) | 3 Months Ended | |
In Thousands, unless otherwise specified | 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) |
Consolidated_Statements_of_Cas
Consolidated Statements of Cash Flows (USD $) | 3 Months Ended | |
In Thousands, unless otherwise specified | 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 |
The_Company
The Company | 3 Months Ended |
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. | |
Summary_of_significant_account
Summary of significant accounting policies | 3 Months Ended |
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_instru
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 | Significant | Significant | |||||||||||
in active | other | unobservable | ||||||||||||
markets for | observable | inputs | ||||||||||||
identical assets | inputs | (level 3) | ||||||||||||
(level 1) | (level 2) | |||||||||||||
Marketable securities | $ | 243,642 | $ | — | $ | 243,642 | $ | — | ||||||
Warrant liability | 229 | — | — | 229 | ||||||||||
December 31, 2014 | ||||||||||||||
Total | Quoted | Significant | Significant | |||||||||||
prices | other | unobservable | ||||||||||||
in active | observable | inputs | ||||||||||||
markets for | inputs | (level 3) | ||||||||||||
identical | (level 2) | |||||||||||||
assets | ||||||||||||||
(level 1) | ||||||||||||||
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 | More Than | |||||||||||||
12 Months | 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 | More Than | |||||||||||||
12 Months | 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. | ||||||||||||||
Other_comprehensive_income_los
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 | Foreign | Total | |||||||||
Gains/(Losses) | Currency | Accumulated | |||||||||
On | Translation | Other | |||||||||
Marketable | Comprehensive | ||||||||||
Securities | 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 | ) | ||
Accounts_payable_and_accrued_e
Accounts payable and accrued expenses | 3 Months Ended | |||||||
Mar. 31, 2015 | ||||||||
Accounts payable and accrued expenses | ||||||||
Accounts payable and accrued expenses | ||||||||
5.Accounts payable and accrued expenses | ||||||||
Accounts payable and accrued expenses at March 31, 2015 and December 31, 2014 consist of the following: | ||||||||
March 31, | December 31, | |||||||
2015 | 2014 | |||||||
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 | ||||||
$ | 23,532 | $ | 29,121 | |||||
Warrants
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 | Exercise | Expiration | |||||||
shares | price | ||||||||
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 | Exercise | Expiration | |||||||
shares | price | ||||||||
Common stock | 6,250 | $ | 128.00 | 2017 | |||||
Common stock | 7,030 | $ | 128.00 | 2019 and 2020 | |||||
Common stock | 130 | $ | 2,520.00 | 2019 | |||||
Net_loss_per_share
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
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 | Weighted- | Weighted- | Aggregate | |||||||||
options | average | average | intrinsic | |||||||||
exercise | remaining | value | ||||||||||
price | contractual | |||||||||||
term | ||||||||||||
(in | ||||||||||||
thousands) | ||||||||||||
Outstanding at December 31, 2014 | 3,432,972 | $ | 25 | |||||||||
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 | $ | 33.6 | 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 | Weighted | |||||||||||
Shares | 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. | ||||||||||||
Commitments_and_contingencies
Commitments and contingencies | 3 Months Ended | ||||
Mar. 31, 2015 | |||||
Commitments and contingencies | |||||
Commitments and contingencies | |||||
9. Commitments and contingencies | |||||
Operating leases | |||||
The Company leases office space under a noncancelable operating lease through February 2019. Rent expense was approximately $0.3 million for the three months ended March 31, 2015. The Company also leases certain office equipment under operating leases. Future minimum lease payments as of March 31, 2015 are as follows: | |||||
2015 | $ | 1,016 | |||
2016 | 946 | ||||
2017 | 923 | ||||
2018 | 1,035 | ||||
2019 | 176 | ||||
$ | 4,096 | ||||
Other contingencies | |||||
Under various agreements, the Company will be required to pay royalties and milestone payments upon the successful development and commercialization of products. The Company has entered into funding agreements with The Wellcome Trust Limited (Wellcome Trust) for the research and development of small molecule compounds. To the extent that the Company develops and commercializes program intellectual property on a for-profit basis, it may become obligated to pay to Wellcome Trust development and regulatory milestone payments of up to an aggregate of $68.9 million and single-digit royalties on sales of any research program product. The Company’s obligation to pay such royalties would continue on a country-by-country basis until the longer of the expiration of the last patent in the program intellectual property in such country covering the research program product and the expiration of market exclusivity of such product in such country. | |||||
The Company has also entered into a collaboration agreement with the SMA Foundation. The Company may become obligated to pay the SMA Foundation single-digit royalties on worldwide net product sales of any collaboration product that we successfully develop and subsequently commercialize or, if the Company outlicenses rights to a collaboration product, a specified percentage of certain payments the Company receives from the licensee. The Company is not obligated to make such payments unless and until annual sales of a collaboration product exceed a designated threshold. The Company’s obligation to make such payments would end upon its payment to the SMA Foundation of a specified amount. | |||||
The Company has employment agreements with certain employees that require the funding of a specific level of payments, if certain events, such as a change in control or termination without cause, occur. | |||||
Collaboration_Revenue
Collaboration Revenue | 3 Months Ended |
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. | |
Summary_of_significant_account1
Summary of significant accounting policies (Policies) | 3 Months Ended |
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. | |
Fair_value_of_financial_instru1
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 | Significant | Significant | |||||||||||
in active | other | unobservable | ||||||||||||
markets for | observable | inputs | ||||||||||||
identical assets | inputs | (level 3) | ||||||||||||
(level 1) | (level 2) | |||||||||||||
Marketable securities | $ | 243,642 | $ | — | $ | 243,642 | $ | — | ||||||
Warrant liability | 229 | — | — | 229 | ||||||||||
December 31, 2014 | ||||||||||||||
Total | Quoted | Significant | Significant | |||||||||||
prices | other | unobservable | ||||||||||||
in active | observable | inputs | ||||||||||||
markets for | inputs | (level 3) | ||||||||||||
identical | (level 2) | |||||||||||||
assets | ||||||||||||||
(level 1) | ||||||||||||||
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 | More Than | |||||||||||||
12 Months | 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 | More Than | |||||||||||||
12 Months | 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 | ||||||||||||
Other_comprehensive_income_los1
Other comprehensive income (loss) and accumulated other comprehensive items (Tables) | 3 Months Ended | ||||||||||
Mar. 31, 2015 | |||||||||||
Other comprehensive income (loss) and accumulated other comprehensive items | |||||||||||
Summary of other comprehensive income (loss) and the changes in accumulated other comprehensive items | Unrealized | Foreign | Total | ||||||||
Gains/(Losses) | Currency | Accumulated | |||||||||
On | Translation | Other | |||||||||
Marketable | Comprehensive | ||||||||||
Securities | 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 | ) | ||
Accounts_payable_and_accrued_e1
Accounts payable and accrued expenses (Tables) | 3 Months Ended | |||||||
Mar. 31, 2015 | ||||||||
Accounts payable and accrued expenses | ||||||||
Schedule of components of accounts payable and accrued expenses | ||||||||
March 31, | December 31, | |||||||
2015 | 2014 | |||||||
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 | ||||||
$ | 23,532 | $ | 29,121 | |||||
Warrants_Tables
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 | Exercise | Expiration | |||||||
shares | price | ||||||||
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 | Exercise | Expiration | |||||||
shares | price | ||||||||
Common stock | 6,250 | $ | 128.00 | 2017 | |||||
Common stock | 7,030 | $ | 128.00 | 2019 and 2020 | |||||
Common stock | 130 | $ | 2,520.00 | 2019 | |||||
Net_loss_per_share_Tables
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
Stock award plan (Tables) | 3 Months Ended | |||||||||||
Mar. 31, 2015 | ||||||||||||
Stock award plan | ||||||||||||
Summary of stock option activity | Number of | Weighted- | Weighted- | Aggregate | ||||||||
options | average | average | intrinsic | |||||||||
exercise | remaining | value | ||||||||||
price | contractual | |||||||||||
term | ||||||||||||
(in | ||||||||||||
thousands) | ||||||||||||
Outstanding at December 31, 2014 | 3,432,972 | $ | 25 | |||||||||
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 | $ | 33.6 | 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 | Weighted | |||||||||||
Shares | 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 | ||||||||
Commitments_and_contingencies_
Commitments and contingencies (Tables) | 3 Months Ended | ||||
Mar. 31, 2015 | |||||
Commitments and contingencies | |||||
Schedule of future minimum lease payments | 2015 | $ | 1,016 | ||
2016 | 946 | ||||
2017 | 923 | ||||
2018 | 1,035 | ||||
2019 | 176 | ||||
$ | 4,096 | ||||
The_Company1
The Company (USD $) | 0 Months Ended | ||
In Thousands, unless otherwise specified | Aug. 04, 2014 | Mar. 31, 2015 | Dec. 31, 2014 |
state | |||
The Company | |||
Minimum age of ambulatory patient | 5 years | ||
Number of member states of the European Union | 31 | ||
Accumulated deficit | ($460,466) | ($422,551) |
Fair_value_of_financial_instru2
Fair value of financial instruments and marketable securities (Details) (USD $) | Mar. 31, 2015 | Dec. 31, 2014 |
In Thousands, unless otherwise specified | ||
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_instru3
Fair value of financial instruments and marketable securities (Details 2) (USD $) | Mar. 31, 2015 | Dec. 31, 2014 |
In Thousands, unless otherwise specified | ||
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_instru4
Fair value of financial instruments and marketable securities (Details 3) (USD $) | Mar. 31, 2015 | Dec. 31, 2014 |
In Thousands, unless otherwise specified | ||
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 |
Fair_value_of_financial_instru5
Fair value of financial instruments and marketable securities (Details 4) (Warrants, USD $) | 3 Months Ended | 12 Months Ended |
In Thousands, except Per Share data, unless otherwise specified | 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 | |
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 |
Other_comprehensive_income_los2
Other comprehensive income (loss) and accumulated other comprehensive items (Details) (USD $) | 3 Months Ended |
In Thousands, unless otherwise specified | Mar. 31, 2015 |
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) |
Accounts_payable_and_accrued_e2
Accounts payable and accrued expenses (Details) (USD $) | Mar. 31, 2015 | Dec. 31, 2014 |
In Thousands, unless otherwise specified | ||
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 |
Warrants_Details
Warrants (Details) (Warrants, Common stock, USD $) | Mar. 31, 2015 | Dec. 31, 2014 |
2017 | ||
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 |
2019 | ||
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 $) | 3 Months Ended | |
In Thousands, except Share data, unless otherwise specified | 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
Net loss per share (Details 2) | 3 Months Ended | |
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
Stock award plan (Details) (USD $) | 3 Months Ended | 1 Months Ended | 0 Months Ended |
In Thousands, except Share data, unless otherwise specified | Mar. 31, 2015 | 31-May-13 | Mar. 05, 2013 |
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 | ||
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) | $389 | ||
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
Stock award plan - (Details 2) (Restricted stock, USD $) | 3 Months Ended |
Mar. 31, 2015 | |
Restricted stock | |
Number of Shares | |
Balance at the beginning of the period (in shares) | 718,400 |
Vested (in shares) | -350,084 |
Forfeited (in shares) | -3,346 |
Balance at the end of the period (in shares) | 364,970 |
Weighted Average Grant Date Fair Value | |
Balance at the beginning of the period (in dollars per share) | $10.72 |
Vested | $10.59 |
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 $) | 3 Months Ended | |
Mar. 31, 2015 | Mar. 31, 2014 | |
Share-based compensation expense recorded in the statement of operations | ||
Share-based compensation expense | $9,748,000 | $3,705,000 |
Unrecognized compensation cost | 70,600,000 | |
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 | 4,667,000 | 1,944,000 |
Selling, general and administrative | ||
Share-based compensation expense recorded in the statement of operations | ||
Share-based compensation expense | $5,081,000 | $1,761,000 |
Commitments_and_contingencies_1
Commitments and contingencies (Details) (USD $) | 3 Months Ended |
Mar. 31, 2015 | |
Commitments and contingencies | |
Operating Leases, Rent Expense, Net | $300,000 |
Operating Leases, Future Minimum Payments Due, Fiscal Year Maturity | |
2015 | 1,016,000 |
2016 | 946,000 |
2017 | 923,000 |
2018 | 1,035,000 |
2019 | 176,000 |
Total future minimum lease payments | 4,096,000 |
Funding agreement | Wellcome trust | Maximum | |
Other contingencies | |
Development and regulatory milestone payments which the entity may be obligated to pay | $68,900,000 |
Collaboration_Revenue_Details
Collaboration Revenue (Details) (Collaboration agreement, Roche and SMA Foundation, Clinical development milestones, USD $) | 0 Months Ended |
In Millions, unless otherwise specified | Jan. 22, 2014 |
Collaboration agreement | Roche and SMA Foundation | Clinical development milestones | |
Collaboration Revenue | |
Milestone payment received from Roche | $7.50 |