Document_and_Entity_Informatio
Document and Entity Information | 9 Months Ended | |
Sep. 30, 2013 | Nov. 13, 2013 | |
Document and Entity Information | ' | ' |
Entity Registrant Name | 'PTC THERAPEUTICS, INC. | ' |
Entity Central Index Key | '0001070081 | ' |
Document Type | '10-Q | ' |
Document Period End Date | 30-Sep-13 | ' |
Amendment Flag | 'false | ' |
Current Fiscal Year End Date | '--12-31 | ' |
Entity Current Reporting Status | 'Yes | ' |
Entity Filer Category | 'Non-accelerated Filer | ' |
Entity Common Stock, Shares Outstanding | ' | 24,917,089 |
Document Fiscal Year Focus | '2013 | ' |
Document Fiscal Period Focus | 'Q3 | ' |
Balance_sheets
Balance sheets (USD $) | Sep. 30, 2013 | Dec. 31, 2012 |
Current assets: | ' | ' |
Cash and cash equivalents | $24,044,819 | $2,725,702 |
Marketable securities | 133,182,565 | ' |
Prepaid expenses and other current assets | 2,126,575 | 855,750 |
Grant and collaboration receivables, net | 783,851 | 1,013,813 |
Total current assets | 160,137,810 | 4,595,265 |
Fixed assets, net | 6,942,956 | 8,280,037 |
Deposits and other assets | 163,349 | 197,050 |
Total assets | 167,244,115 | 13,072,352 |
Current liabilities: | ' | ' |
Accounts payable and accrued expenses | 10,205,493 | 7,023,971 |
Current portion of long-term debt | 84,234 | 4,444,171 |
Deferred revenue | 3,631,430 | 16,690,747 |
Total current liabilities | 13,921,157 | 28,158,889 |
Deferred revenue, less current portion | ' | 741,667 |
Long-term debt, less current portion | ' | 438,810 |
Other long-term liabilities | 2,290,445 | 2,549,719 |
Total liabilities | 16,211,602 | 31,889,085 |
Stockholders' (deficit) equity: | ' | ' |
Common stock, $0.001 par value. Authorized 17,000,000 shares; issued and outstanding 4,526 shares at December 31, 2012. Authorized 125,000,000 shares; issued and outstanding 23,803,282 shares at September 30, 2013 | 24,344 | 545 |
Additional paid-in capital | 461,960,966 | 177,583,672 |
Accumulated other comprehensive loss | -40,467 | ' |
Accumulated deficit | -310,912,330 | -277,224,718 |
Total stockholders' (deficit) equity | 151,032,513 | -99,640,501 |
Total liabilities, convertible preferred stocks and stockholders' (deficit) equity | 167,244,115 | 13,072,352 |
Series One convertible preferred stock | ' | ' |
Stockholders' (deficit) equity: | ' | ' |
Preferred stock | ' | 62,263,852 |
Series Two convertible preferred stock | ' | ' |
Stockholders' (deficit) equity: | ' | ' |
Preferred stock | ' | 18,182,129 |
Series Three convertible preferred stock | ' | ' |
Stockholders' (deficit) equity: | ' | ' |
Preferred stock | ' | 377,787 |
Preferred stock | ' | ' |
Stockholders' (deficit) equity: | ' | ' |
Preferred stock | ' | ' |
Balance_sheets_Parenthetical
Balance sheets (Parenthetical) (USD $) | Sep. 30, 2013 | Dec. 31, 2012 |
Common stock, par value (in dollars per share) | $0.00 | $0.00 |
Common stock, Authorized shares | 125,000,000 | 17,000,000 |
Common stock, issued shares | 23,803,282 | 4,526 |
Common stock, outstanding shares | 23,803,282 | 4,526 |
Series One convertible preferred stock | ' | ' |
Preferred stock, designated shares | ' | 2,000,000 |
Preferred stock, issued shares | ' | 1,483,337 |
Preferred stock, outstanding shares | ' | 1,483,337 |
Series Two convertible preferred stock | ' | ' |
Preferred stock, designated shares | ' | 13,750,000 |
Preferred stock, issued shares | ' | 10,701,405 |
Preferred stock, outstanding shares | ' | 10,701,405 |
Series Three convertible preferred stock | ' | ' |
Preferred stock, designated shares | ' | 13,750,000 |
Preferred stock, issued shares | ' | 2,853,517 |
Preferred stock, outstanding shares | ' | 2,853,517 |
Preferred stock | ' | ' |
Preferred stock, par value (in dollars per share) | $0.00 | ' |
Preferred stock, undesignated shares | 5,000,000 | ' |
Preferred stock, issued shares | 0 | ' |
Preferred stock, outstanding shares | 0 | ' |
Statements_of_operations
Statements of operations (USD $) | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2013 | Sep. 30, 2012 | Sep. 30, 2013 | Sep. 30, 2012 | |
Revenues: | ' | ' | ' | ' |
Collaboration revenue | $15,455,361 | $6,042,746 | $27,395,315 | $22,860,543 |
Grant revenue | 834,212 | 1,152,330 | 2,890,134 | 4,445,430 |
Total revenues and non-cash cancellation revenue | 16,289,573 | 7,195,076 | 30,285,449 | 27,305,973 |
Operating expenses: | ' | ' | ' | ' |
Research and development | 13,886,196 | 10,466,454 | 39,855,115 | 36,688,786 |
General and administrative | 6,678,992 | 3,783,929 | 17,735,294 | 11,390,938 |
Total operating expenses | 20,565,188 | 14,250,383 | 57,590,409 | 48,079,724 |
Loss from operations | -4,275,615 | -7,055,307 | -27,304,960 | -20,773,751 |
Interest (expense) income, net | 26,695 | -247,325 | -6,249,534 | -1,007,586 |
Loss on extinguishment of debt | -129,963 | ' | -129,963 | ' |
Other income (expense), net | -37,616 | 5,507 | -3,155 | 1,818,279 |
Net loss | -4,416,499 | -7,297,125 | -33,687,612 | -19,963,058 |
Deemed dividend | ' | ' | -18,248,768 | ' |
Gain on exchange of convertible preferred stock in connection with recapitalization | ' | ' | 3,390,750 | 159,954,069 |
Less beneficial conversion charge | ' | ' | ' | -377,787 |
Net (loss) income attributable to common stockholders | ($4,416,499) | ($7,297,125) | ($48,545,630) | $139,613,224 |
Weighted-average shares outstanding: | ' | ' | ' | ' |
Basic (in shares) | 23,803,282 | 4,545 | 8,995,167 | 2,937 |
Weighted-average shares outstanding: | ' | ' | ' | ' |
Diluted (in shares) | 23,803,282 | 4,545 | 8,995,167 | 13,593 |
Net (loss) income per share applicable to common stockholders-basic (in dollars per share) | ($0.19) | ($1,605.53) | ($5.40) | $182.41 |
Net (loss) income per share applicable to common stockholders-diluted (in dollars per share) | ($0.19) | ($1,605.53) | ($5.40) | $39.41 |
Statements_of_comprehensive_lo
Statements of comprehensive loss (USD $) | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2013 | Sep. 30, 2012 | Sep. 30, 2013 | Sep. 30, 2012 | |
Statements of comprehensive loss | ' | ' | ' | ' |
Net loss | ($4,416,499) | ($7,297,125) | ($33,687,612) | ($19,963,058) |
Other comprehensive loss: | ' | ' | ' | ' |
Unrealized loss on marketable securities | -39,695 | ' | -40,467 | ' |
Comprehensive loss | ($4,456,194) | ($7,297,125) | ($33,728,079) | ($19,963,058) |
Statements_of_cash_flows
Statements of cash flows (USD $) | 9 Months Ended | |
Sep. 30, 2013 | Sep. 30, 2012 | |
Cash flows from operating activities | ' | ' |
Net loss | ($33,687,612) | ($19,963,058) |
Adjustments to reconcile net loss to net cash used in operating activities: | ' | ' |
Depreciation | 1,810,850 | 2,048,682 |
Change in valuation of warrant liability | 3,158 | -1,818,279 |
Non-cash interest expense | 6,048,580 | 204,422 |
Loss on extinguishment of debt | 129,963 | ' |
Share-based compensation expense | 5,142,187 | 1,815,722 |
Changes in operating assets and liabilities: | ' | ' |
Prepaid expenses and other current assets | -1,287,875 | 2,250,089 |
Grant and collaboration receivables | 229,962 | 75,698 |
Deposits and other assets | 33,699 | 24,399 |
Accounts payable and accrued expenses | 3,181,522 | -4,748,005 |
Other long-term liabilities | -262,432 | -68,432 |
Deferred revenue | -13,800,984 | -16,711,980 |
Net cash used in operating activities | -32,458,982 | -36,890,742 |
Cash flows from investing activities | ' | ' |
Purchases of fixed assets | -473,769 | -183,511 |
Purchases of marketable securities | -139,730,579 | ' |
Maturities of marketable securities | 6,507,547 | ' |
Net cash used in investing activities | -133,696,801 | -183,511 |
Cash flows from financing activities | ' | ' |
Payments on long-term debt | -4,960,151 | -6,240,116 |
Net proceeds from sale of Series One convertible preferred stock | ' | 29,354,752 |
Net proceeds from sale of Series Four convertible preferred stock | 60,785,363 | ' |
Net proceeds from initial public offering | 131,649,688 | ' |
Net cash provided by financing activities | 187,474,900 | 23,114,636 |
Net (decrease) increase in cash and cash equivalents | 21,319,117 | -13,959,617 |
Cash and cash equivalents, beginning of period | 2,725,702 | 28,431,410 |
Cash and cash equivalents, end of period | 24,044,819 | 14,471,793 |
Supplemental disclosure of cash information | ' | ' |
Cash paid for interest | 365,589 | 1,009,478 |
Supplemental disclosures of non-cash information related to investing and financing activities | ' | ' |
Change in unrealized loss on marketable securities | -40,467 | ' |
Change in carry value of preferred securities resulting from recapitalization | $3,390,750 | $159,576,282 |
The_Company
The Company | 9 Months Ended |
Sep. 30, 2013 | |
The Company | ' |
The Company | ' |
1. The Company | |
PTC Therapeutics, Inc. (the Company or PTC) was incorporated as a Delaware corporation on March 31, 1998. The Company is a biopharmaceutical company focused on the discovery and development of orally administered, proprietary small-molecule drugs that target post-transcriptional control processes. | |
The Company has devoted substantially all of its efforts to research and development, including clinical trials. The Company has not completed development of any drugs. The Company has not generated product revenue to date and is subject to a number of risks similar to those of other early stage companies, including dependence on key individuals, the difficulties inherent in the development of commercially usable products, the potential need to obtain additional capital necessary to fund the development of its products, and competition from other companies. As of September 30, 2013, the Company had an accumulated deficit of approximately $310.9 million. The Company has financed its operations to date primarily through the issuance and sale of its common stock in its initial public offering (see note 7 below), private placements of its convertible preferred stock, collaborations, bank debt, convertible debt financings, grant funding and clinical trial support from governmental and philanthropic organizations and patient advocacy groups in the disease areas addressed by the Company’s product candidates. The Company believes that its existing cash, cash equivalents, including the Company’s net proceeds from its initial public offering of common stock, and marketable securities provide for sufficient resources to fund its currently planned operations through the fourth quarter of 2015. |
Summary_of_significant_account
Summary of significant accounting policies | 9 Months Ended |
Sep. 30, 2013 | |
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, 2012 included in the final prospectus dated June 19, 2013 related to the Company’s initial public offering. | |
Basis of Presentation | |
The accompanying financial information as of September 30, 2013 and for the three and nine months ended September 30, 2012 and 2013 has been prepared by the Company, without audit, pursuant to the rules and regulations of the Securities and Exchange Commission (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, 2012 and notes thereto included in the final prospectus related to the Company’s initial public offering. | |
In the opinion of management, the unaudited financial information as of September 30, 2013 and for three and nine months ended September 30, 2012 and 2013 reflects all adjustments, which are normal recurring adjustments, necessary to present a fair statement of financial position, results of operations and cash flows. The results of operations for the three and nine month periods ended September 30, 2013 are not necessarily indicative of the results to be expected for the year ended December 31, 2013 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. | |
Reverse stock split | |
On March 7, 2013, the Company effected a one-for-120 reverse stock split of its common stock, pursuant to which each 120 shares of the Company’s outstanding common stock were reclassified and combined into one share of common stock. All common stock share and per share references in the financial statements and notes thereto have been restated to reflect the reverse stock split on a retroactive basis. | |
Marketable Securities | |
Management determines the classification of marketable securities at the time of purchase and reevaluates such designation as of each balance sheet date. Marketable securities are classified as available-for-sale and carried at fair value, with any unrealized gain or loss recorded as a separate component of stockholders’ (deficit) equity. | |
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. | |
The Company’s revenue is generated primarily through collaborative research and development and licensing agreements and grants. | |
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. | |
For existing collaborations entered into prior to the adoption in 2011 of the revised multiple element revenue recognition guidance described below, the Company recognized revenue consistent with the approach established at the inception of each arrangement. For these existing collaborations, where the Company has continuing involvement, the Company recorded nonrefundable, upfront fees as deferred revenue and recognizes revenue on a straight-line basis as collaboration revenue over the expected performance period. | |
For new collaborations or for material modifications made to existing collaborations, in 2011 and thereafter, the Company adopted the updated multiple element revenue recognition guidance. Under this guidance, all non-contingent arrangement consideration is allocated to the identified units of accounting based on their relative selling price at inception of the collaboration arrangement. The Company derives the selling price using a combination of internal subjective and available external objective information, such as comparable transactions. The Company recognizes revenue commensurate with delivery, such as in the case with delivery of a license, or ratably over the course of a service period, as appropriate, such as in the case of ongoing research and development activities. | |
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 milestone payments are 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 its 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 as substantive milestones and will be recognized as revenue in the period that the milestone is achieved. | |
The Company recognizes royalties as earned in accordance with the terms of various research and collaboration agreements. If not substantive, the contingent consideration is allocated to the existing units of accounting based on relative selling price and recognized following the same basis previously established for the associated unit of accounting. The Company recognizes revenue for reimbursements of research and development costs under collaboration agreements as the services are performed. The Company records these reimbursements as revenue and not as a reduction of research and development expenses as the Company has the risks and rewards as the principal in the research and development activities. | |
Fair value of financial instruments | |
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. | |
Warrant liability | |
Warrants to purchase the Company’s common stock with nonstandard antidilution provisions and preferred stock that include a put feature, regardless of the probability or likelihood that may conditionally obligate the issuer to ultimately transfer assets, are classified as liabilities and are recorded at their estimated fair value at each reporting period. Any change in fair value of these warrants is recorded as gain (loss) on warrant valuation each reporting period in Other income (expense), net on the Company’s statement of operations. | |
Share-based compensation | |
The Company measures the cost of employee services received in exchange for an award of equity instruments based on the grant date fair value of the award. For service type awards, share-based compensation expense is recognized on a straight-line basis over the period during which the employee is required to provide service in exchange for the entire award. For awards that vest or begin vesting upon achievement of a performance condition, the Company estimates the likelihood of satisfaction of the performance condition and recognizes compensation expense when achievement of the performance condition is deemed probable using an accelerated attribution model. | |
The fair value of options is calculated using the Black-Scholes option pricing model to determine the fair value of stock options on the date of grant based on key assumptions, such as expected volatility and expected term. As a new public company, the Company does not have sufficient history to estimate the volatility of its common stock price or the expected life of the options. The Company calculates expected volatility based on reported data for similar publicly traded companies for which historical information is available and will continue to do so until the historical volatility of its common stock is sufficient to measure expected volatility for future option grants. |
Fair_value_of_financial_instru
Fair value of financial instruments and marketable securities | 9 Months Ended | |||||||||||||
Sep. 30, 2013 | ||||||||||||||
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 | ||||||||||||||
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 2 for the Company’s financial assets that are required to be measured at fair value on a recurring basis as of December 31, 2012 and September 30, 2013: | ||||||||||||||
December 31, 2012 | ||||||||||||||
Total | Quoted prices | Significant | Significant | |||||||||||
in active | other | unobservable | ||||||||||||
markets for | observable | inputs | ||||||||||||
identical assets | inputs | (level 3) | ||||||||||||
(level 1) | (level 2) | |||||||||||||
Warrant liability | $ | 95,661 | $ | — | $ | — | $ | 95,661 | ||||||
September 30, 2013 | ||||||||||||||
Total | Quoted prices | Significant | Significant | |||||||||||
in active | other | unobservable | ||||||||||||
markets for | observable | inputs | ||||||||||||
identical assets | inputs | (level 3) | ||||||||||||
(level 1) | (level 2) | |||||||||||||
Marketable securities | $ | 133,182,565 | $ | — | $ | 133,182,565 | $ | — | ||||||
Warrant liability | 98,819 | — | — | 98,819 | ||||||||||
The following is a summary of marketable securities accounted for as available-for-sale securities at September 30, 2013: | ||||||||||||||
September 30, 2013 | ||||||||||||||
Amortized | Gross Unrealized | Fair | ||||||||||||
Cost | Gains | Losses | Value | |||||||||||
Commercial paper | $ | 24,978,703 | $ | 20,527 | $ | — | $ | 24,999,230 | ||||||
U.S. corporate debt securities | 108,244,329 | 19,642 | (80,636 | ) | 108,183,335 | |||||||||
$ | 133,223,032 | $ | 40,169 | $ | (80,636 | ) | $ | 133,182,565 | ||||||
At September 30, 2013, the Company held securities with an unrealized loss position that were not considered to be other-than-temporarily impaired as the Company has the ability to hold such investments until recovery of their fair value. | ||||||||||||||
Marketable securities on the balance sheet at September 30, 2013 mature as follows: | ||||||||||||||
September 30, 2013 | ||||||||||||||
Less Than | More Than | |||||||||||||
12 Months | 12 Months | |||||||||||||
Commercial paper | $ | 24,999,230 | $ | — | ||||||||||
U.S. corporate debt securities | 57,824,657 | 50,358,678 | ||||||||||||
Total Marketable securities | $ | 82,823,887 | $ | 50,358,678 | ||||||||||
There were no marketable securities as of December 31, 2012. | ||||||||||||||
Level 3 valuation | ||||||||||||||
The warrant liability is classified in Other long-term liabilities on the Company’s balance sheet. The warrant liability is marked-to-market each reporting period with the change in fair value recorded as a gain or loss within Other income (expense), net on the Company’s statement of operations until the warrants are exercised, expire or other facts and circumstances lead the warrant liability to be reclassified as an equity instrument. The fair value of the warrant liability is determined at each reporting period by utilizing the Black-Scholes option pricing model. | ||||||||||||||
The table presented below is a summary of changes in the fair value of the Company’s Level 3 valuation for warrant liability for the period ended September 30, 2013: | ||||||||||||||
Level 3 assets | ||||||||||||||
Beginning balance as of December 31, 2012 | $ | 95,661 | ||||||||||||
Warrants issued in connection with convertible promissory notes | 6,000,000 | |||||||||||||
Deemed dividend | 18,248,768 | |||||||||||||
Exercise of warrants issued in connection with convertible promissory notes | (24,248,765 | ) | ||||||||||||
Change in fair value of warrant liability | 3,155 | |||||||||||||
Ending balance as of September 30, 2013 | $ | 98,819 | ||||||||||||
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 December 31, 2012 include (i) volatility (87%), (ii) risk free interest rate (0.16%—1.18%), (iii) strike price ($16), (iv) fair value of preferred shares ($2.35), and (v) expected life (1—7 years). The significant assumptions used in preparing the option pricing model for valuing the Company’s warrants as of September 30, 2013 include (i) volatility (69-87%), (ii) risk free interest rate (0.02%—1.705%), (iii) strike price ($128), (iv) fair value of common shares ($21.46), and (v) expected life (.22—5.98 years). See Note 7 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 | 9 Months Ended | |||||||
Sep. 30, 2013 | ||||||||
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 table summarizes other comprehensive loss and the changes in accumulated other comprehensive items for the three months ended September 30, 2013: | ||||||||
Unrealized Losses | Total | |||||||
On Marketable | Accumulated | |||||||
Securities | Other | |||||||
Comprehensive | ||||||||
Items | ||||||||
Balance at June 30, 2013 | $ | (772 | ) | $ | (772 | ) | ||
Other comprehensive loss before reclassifications | (39,695 | ) | (39,695 | ) | ||||
Amounts reclassified from other comprehensive items | — | — | ||||||
Other comprehensive loss | (39,695 | ) | (39,695 | ) | ||||
Balance at September 30, 2013 | $ | (40,467 | ) | $ | (40,467 | ) | ||
The following table summarizes other comprehensive loss and the changes in accumulated other comprehensive items for the nine months ended September 30, 2013: | ||||||||
Unrealized Losses | Total | |||||||
On Marketable | Accumulated | |||||||
Securities | Other | |||||||
Comprehensive | ||||||||
Items | ||||||||
Balance at December 31, 2012 | $ | — | $ | — | ||||
Other comprehensive loss before reclassifications | (40,467 | ) | (40,467 | ) | ||||
Amounts reclassified from other comprehensive items | — | — | ||||||
Other comprehensive loss | (40,467 | ) | (40,467 | ) | ||||
Balance at September 30, 2013 | $ | (40,467 | ) | $ | (40,467 | ) |
Accounts_payable_and_accrued_e
Accounts payable and accrued expenses | 9 Months Ended | |||||||
Sep. 30, 2013 | ||||||||
Accounts payable and accrued expenses | ' | |||||||
Accounts payable and accrued expenses | ' | |||||||
5. Accounts payable and accrued expenses | ||||||||
Accounts payable and accrued expenses at December 31, 2012 and September 30, 2013 consist of the following: | ||||||||
December 31, | September 30, | |||||||
2012 | 2013 | |||||||
Employee compensation, benefits, and related accruals | $ | 3,096,475 | $ | 3,659,084 | ||||
Consulting and contracted research | 2,515,678 | 3,556,350 | ||||||
Professional fees | 559,228 | 1,532,458 | ||||||
Accounts payable | 621,591 | 1,017,122 | ||||||
Other | 230,999 | 440,479 | ||||||
$ | 7,023,971 | $ | 10,205,493 |
Debt
Debt | 9 Months Ended |
Sep. 30, 2013 | |
Debt | ' |
Debt | ' |
6. Debt | |
In July 2013, the Company paid in full the outstanding principal and interest of $2.6 million due under promissory notes issued in connection with a $25 million secured debt facility with a syndicate of two lenders. The notes were secured by substantially all of the Company’s assets except for intellectual property and carried a fixed interest rate of 13.65%. As a result of this transaction, the Company recorded a loss on extinguishment of debt of $0.1 million on the Company’s statement of operations for the three months ended September 30, 2013, The loss on extinguishment of debt primarily represented the write off of related deferred financing costs, the acceleration of recognition of debt extinguishment fees and the prepayment premium payable. |
Capital_structure
Capital structure | 9 Months Ended | ||||||||
Sep. 30, 2013 | |||||||||
Capital structure | ' | ||||||||
Capital structure | ' | ||||||||
7. Capital structure | |||||||||
2013 Recapitalization | |||||||||
During January and February of 2013, the Company entered into a “bridge” financing arrangement with certain existing investors providing for the issuance by the Company of an aggregate of $6 million of convertible promissory notes and warrants to purchase 2,527,675 shares of Series One convertible preferred stock (Series One) and Series Two convertible preferred stock (Series Two). The warrants have a per share exercise price of $0.01, and as such, they are referred to as “penny warrants”. This bridge financing was closed in anticipation of the March 2013 Series Four financing event, which the Company refers to as the “2013 recapitalization”. | |||||||||
The Company allocated the proceeds of the convertible promissory notes between debt and warrant liability. Since the value of the warrants exceeded the proceeds from the convertible notes issued to existing investors, the value of the warrant in excess of the proceeds is considered a deemed dividend and reflected as an equity transaction in the financial statements. The Company recorded $6 million to interest expense related to the debt discount associated with the convertible debt during the quarter ended March 31, 2013. | |||||||||
On March 7, 2013, the Company closed a private placement of a new series of convertible preferred stock that resulted in the 2013 recapitalization. In this private placement, the Company issued and sold an aggregate of 4,497,035 shares of its Series Four senior preferred stock (Series Four) for an aggregate purchase price of approximately $54 million. Including the $6 million raised with the bridge financing, total gross proceeds raised during the quarter ended March 31, 2013 was approximately $60 million. In addition, the Company issued an aggregate of 502,919 shares of Series Four upon the share settlement of the convertible promissory notes described above that were issued in January and February 2013. | |||||||||
In connection with this private placement, the Company effected a one-for-120 reverse stock split of its common stock and an exchange of outstanding shares of Series One, Series Two and Series Three convertible preferred stock (Series Three) into an aggregate of 6,700,487 shares of a new series of Series Five junior preferred stock (Series Five). In addition, the Company issued an aggregate of 2,527,675 shares of Series One and Series Two upon the exercise of the warrants issued in connection with the bridge loan that were immediately exchanged for 2,095,515 shares of Series Five during the 2013 recapitalization. | |||||||||
The Company accounted for the 2013 recapitalization as an extinguishment of its Series One, Series Two and Series Three convertible preferred stock and recorded the Series Five shares at their fair value as of the recapitalization date. In accordance with authoritative accounting guidance, the Company recorded a gain attributable to the common stockholders on the extinguishment of the Series One, Series Two and Series Three. The gain of approximately $3.4 million represents the excess of the Series One, Series Two and Series Three over the fair value of the shares Series Five issued in connection with the recapitalization. | |||||||||
Valuation—The value of the Company was estimated using the PWERM. The PWERM considered the most significant near-term driver of value for the Company as the Company’s ability to complete a Phase 3 clinical trial of ataluren for the treatment of Duchenne muscular dystrophy caused by nonsense mutations (nmDMD). The remaining scenarios in the PWERM related to funding the completion of the Phase 3 clinical trial for nmDMD. The path to raising this money made up the remaining nodes in the PWERM. | |||||||||
After identifying the various potential liquidity scenarios and their likely timing, a pre-money enterprise value was assigned to each scenario based on a combination of management’s guidance and recent trends in the capital markets. The resulting enterprise value for each liquidity event was divided by the total shares that would be outstanding under each scenario to arrive at a price per share for the common and preferred classes of stock. Each scenario was then assigned an outcome probability based on management’s estimates. The resulting probability weighted share values were then discounted to present value at a rate that reflects general industry risks (but not Company specific risks). | |||||||||
The rights and preferences of the shares of Series Four and Series Five are as follows: | |||||||||
Dividends—The holders of Series Four and Series Five, in preference to the holders of common stock, are entitled to noncumulative dividends when and if declared by the Board of Directors. | |||||||||
Liquidation—Upon the liquidation, dissolution, reorganization or winding-up of the Company, the holders of Series Four will be entitled to receive, before any distribution or payment is made to any other class of security, an amount equal to the original issuance price, plus all declared, but unpaid, dividends. To the extent there are excess assets to distribute, the holders of Series Five will be entitled to receive, before any distribution or payment is made to the holders of the common stock, an amount equal to the stated liquidation preference, plus all declared, but unpaid, dividends. To the extent there are remaining assets to distribute, the holders of common stock will be entitled to receive such remaining assets. | |||||||||
Voting—Each holder of Series Four and Series Five are entitled to cast the number of votes into which such holder’s shares would convert. Except as required by law, holders of common stock have limited voting rights. Additionally, except as required by law, and except in certain enumerated circumstances, holders of Series Four and Series Five shall vote together with the holders of common stock as a single class. | |||||||||
Conversion—Each share of Series Four and Series Five is convertible at any time at the option of the holder into one share of common stock. These conversion ratios are subject to adjustment for certain dilutive events, including certain types of stock splits or stock dividends or future recapitalizations. | |||||||||
In May 2013, the Company issued and sold an additional 375,000 shares of Series Four, at a price per share of $12.00, for an aggregate purchase price of $4.5 million. | |||||||||
Common Stock | |||||||||
In May 2013, the Company’s Board of Directors and stockholders approved an amendment to the Company’s certificate of incorporation increasing the number of authorized shares of common stock to 125,000,000. | |||||||||
Initial Public Offering | |||||||||
In June 2013, the Company closed the initial public offering of its common stock pursuant to a registration statement on Form S-1, as amended. The Company issued and sold an aggregate of 9,627,800 shares of common stock under the registration statement at a public offering price of $15.00 per share, including 1,255,800 shares pursuant to the exercise by the underwriters of an over-allotment option. The Company received net proceeds from the initial public offering of approximately $131.6 million, after deducting underwriting discounts and commissions and other offering expenses payable by the Company. | |||||||||
Upon closing the initial public offering, all outstanding shares of the Series Four and Series Five were converted into 14,170,956 shares of common stock. | |||||||||
Warrants | |||||||||
All of the Company’s outstanding warrants were classified as liabilities as of December 31, 2012 because they contained either non-standard antidilution provisions or they were exercisable into preferred shares that included a put feature. | |||||||||
The following is a summary of the Company’s outstanding warrants as of December 31, 2012: | |||||||||
Warrant | Exercise | Expiration | |||||||
shares | price | ||||||||
Series Two | 24,712 | $ | 16 | 2014 | |||||
Series Two | 50,000 | $ | 16 | 2017 | |||||
Series Two | 56,250 | $ | 16 | 2019 and 2020 | |||||
Common stock | 645 | $ | 2,520 | 2013 and 2014 | |||||
In connection with the 2013 recapitalization, all of the Series Two outstanding warrants became warrants to purchase Series Five. In connection with the Company’s initial public offering all of the Series Five outstanding warrants became warrants to purchase common stock. | |||||||||
The following is a summary of the Company’s outstanding warrants as of September 30, 2013: | |||||||||
Warrant | Exercise | Expiration | |||||||
shares | price | ||||||||
Common stock | 3,088 | $ | 128 | 2014 | |||||
Common stock | 6,250 | $ | 128 | 2017 | |||||
Common stock | 7,030 | $ | 128 | 2019 and 2020 | |||||
Common stock | 645 | $ | 2,520 | 2013 and 2014 |
Net_income_loss_per_share
Net income (loss) per share | 9 Months Ended | |||||||
Sep. 30, 2013 | ||||||||
Net income (loss) per share | ' | |||||||
Net income (loss) per share | ' | |||||||
8. Net income (loss) per share | ||||||||
Basic earnings per share is computed by dividing net income (loss) available to common stockholders by the weighted-average number of common shares outstanding. Diluted earnings per share is computed by dividing net income (loss) available to common stockholders by the weighted-average number of common shares plus the effect of dilutive potential common shares outstanding during the period. | ||||||||
The Series One, Series Two and Series Three outstanding during 2012 (subsequent to the recapitalization) participated in the earnings of the Company through dividend rights. Accordingly, the Company measured earnings per share based upon the two-class method prior to the Company’s initial public offering in June 2013. | ||||||||
The following tables set forth the computation of basic and diluted net income (loss) per share for common stockholders: | ||||||||
Three months ended September 30, | ||||||||
2012 | 2013 | |||||||
Numerator | ||||||||
Net loss attributable to common stockholders | $ | (7,297,125 | ) | $ | (4,416,499 | ) | ||
Denominator | ||||||||
Denominator for basic net loss per share | 4,545 | 23,803,282 | ||||||
Net loss per share: | ||||||||
Basic and diluted | $ | (1,605.53 | )* | $ | (0.19 | )* | ||
* In the three months ended September 30, 2012 and 2013, the Company experienced a net loss and therefore did not report any dilutive share impact. | ||||||||
Nine months ended September 30, | ||||||||
2012 | 2013 | |||||||
Numerator | ||||||||
Net loss | $ | (19,963,058 | ) | $ | (33,687,612 | ) | ||
Deemed dividend | — | (18,248,768 | ) | |||||
Gain on exchange of convertible preferred stock in connection with recapitalization | 159,954,069 | 3,390,750 | ||||||
Less beneficial conversion charge | (377,787 | ) | — | |||||
Less net income attributable to participating securities | (139,077,495 | ) | — | |||||
Net income (loss) attributable to common stockholders | $ | 535,729 | $ | (48,545,630 | ) | |||
Denominator | ||||||||
Denominator for basic net income (loss) per share | 2,937 | 8,995,167 | ||||||
Effect of dilutive securities | ||||||||
Series Three convertible preferred stock | 10,656 | — | ||||||
Denominator for diluted net income (loss) per share | 13,593 | 8,995,167 | ||||||
Net income (loss) per share: | ||||||||
Basic | $ | 182.41 | $ | (5.40 | )* | |||
Diluted | $ | 39.41 | $ | (5.40 | )* | |||
* In the nine months ended September 30, 2013, the Company experienced a net loss and therefore did not report any dilutive share impact. | ||||||||
The following table shows historical dilutive common share equivalents outstanding, which are not included in the above historical calculation, as the effect of their inclusion is anti-dilutive during each period. | ||||||||
As of September 30, | ||||||||
2012 | 2013 | |||||||
Stock Options | 44,218 | 1,987,860 | ||||||
Unvested restricted stock | — | 1,113,807 | ||||||
Total | 44,218 | 3,101,667 |
Stock_option_plan
Stock option plan | 9 Months Ended | ||||||||||||||
Sep. 30, 2013 | |||||||||||||||
Stock option plan | ' | ||||||||||||||
Stock option plan | ' | ||||||||||||||
9. Stock option plan | |||||||||||||||
On March 5, 2013, the Company’s Board of Directors approved the 2013 Stock Incentive Plan, which provides for the granting of stock option awards, stock appreciation rights, restricted stock, restricted stock units and other stock-based awards in the aggregate of 739,937 shares of common stock. On March 5, 2013, the Board approved a grant of 735,324 shares of restricted stock and 4,613 stock options. There are no additional shares available for issuance under this plan. | |||||||||||||||
In May 2013, the Company’s Board of Directors and stockholders increased by 2,500,000 the number of shares authorized under the 2009 Stock Incentive Plan, which provides for the granting of stock option awards, restricted stock awards, and other stock-based and cash-based awards. | |||||||||||||||
In May 2013, the Company’s Board of Directors and stockholders approved the 2013 Long Term Incentive Plan, which became effective upon the closing of the Company’s IPO. The 2013 Long Term Incentive Plan provides for the grant of incentive stock options, nonstatutory stock options, restricted stock awards and other stock-based awards. The number of shares of common stock reserved for issuance under the 2013 Long Term Incentive Plan is the sum of (1) 122,296 shares of common stock available for issuance under the Company’s 2009 Equity and Long Term Incentive Plan and 2013 Stock Incentive Plan, (2) the number of shares (up to 3,040,444 shares) equal to the sum of the number of shares of common stock subject to outstanding awards under the Company’s 1998 Employee, Director and Consultant Stock Option Plan and 2013 Stock Incentive Plan that expire, terminate or are otherwise surrendered, cancelled, forfeited or repurchased by the Company at their original issuance price pursuant to a contractual repurchase right plus (3) an annual increase, to be added on the first day of each fiscal year until the expiration of the 2013 Long Term Incentive Plan, equal to the lowest of 2,500,000 shares of common stock, 4% of the number of shares of common stock outstanding on the first day of the fiscal year and an amount determined by the Company’s Board of Directors. | |||||||||||||||
A summary of stock option activity is as follows: | |||||||||||||||
Number of | Exercise | Weighted- | Weighted- | Aggregate | |||||||||||
options | price | average | average | intrinsic | |||||||||||
exercise | remaining | value | |||||||||||||
price | contractual | ||||||||||||||
term | |||||||||||||||
(in thousands) | |||||||||||||||
Outstanding at December 31, 2012 | 42,394 | $ | 218.40-$1,149.60 | $ | 469.48 | ||||||||||
Granted | 2,007,713 | $ | 10.59-$10.85 | $ | 10.85 | ||||||||||
Exercised | — | — | — | ||||||||||||
Forfeited | (62,247 | ) | $ | 10.85—$1,149.60 | $ | 17.1 | |||||||||
Outstanding at September 30, 2013 | 1,987,860 | $ | 10.59—$1,149.60 | $ | 20.44 | 9.51 years | $ | 20,652 | |||||||
Exercisable at September 30, 2013 | 61,834 | $ | 10.59—$1,149.60 | $ | 290.39 | 6.06 years | $ | 265 | |||||||
The fair value of grants made in the period ended September 30, 2013 was contemporaneously estimated on the date of grant using the following assumptions: | |||||||||||||||
Nine months ended | |||||||||||||||
September 30, 2013 | |||||||||||||||
Risk-free interest rate | 0.85%-1.69% | ||||||||||||||
Expected volatility | 87%-88% | ||||||||||||||
Expected term | 5.00 to 6.00 years | ||||||||||||||
Expected dividend yield | 0.00% | ||||||||||||||
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, 2013 was $7.86 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 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, 2013 | — | — | |||||||||||||
Granted | 1,131,524 | $ | 10.68 | ||||||||||||
Vested | — | $ | — | ||||||||||||
Forfeited | (17,717 | ) | $ | 10.63 | |||||||||||
Unvested at September 30, 2013 | 1,113,807 | $ | 10.68 | ||||||||||||
The Company recorded share-based compensation expense in the statement of operations as follows: | |||||||||||||||
Three Months Ended September 30, | Nine Months Ended September 30, | ||||||||||||||
2012 | 2013 | 2012 | 2013 | ||||||||||||
Research and development | $ | 193,199 | $ | 676,499 | $ | 653,462 | $ | 1,377,640 | |||||||
General and administrative | 327,201 | 1,963,197 | 1,162,260 | 3,764,547 | |||||||||||
Total | $ | 520,400 | $ | 2,639,696 | $ | 1,815,722 | $ | 5,142,187 | |||||||
As of December 31, 2012 and September 30, 2013, there was approximately $2.2 million and $21.0 million, respectively of total unrecognized compensation cost related to unvested share-based compensation arrangements granted under the 1998, 2009 and 2013 Plans. This cost is expected to be recognized as share-based compensation expense over the weighted average remaining service period of approximately 2.28 years. |
Collaboration_Revenue
Collaboration Revenue | 9 Months Ended |
Sep. 30, 2013 | |
Collaboration Revenue. | ' |
Collaboration Revenue | ' |
10. Collaboration Revenue | |
On August 8, 2013, the Company announced the selection of a development candidate in its spinal muscular atrophy collaboration with F. Hoffman-La Roche Ltd and Hoffman-La Roche Inc. (Roche) and the Spinal Muscular Atrophy Foundation. The achievement of this milestone triggered a $10.0 million payment to the Company from Roche. The Company considered this milestone event substantive because the applicable criteria of its revenue recognition policy (see Note 2) would be satisfied and recorded it as collaboration revenue for the three months ended September 30, 2013. |
Summary_of_significant_account1
Summary of significant accounting policies (Policies) | 9 Months Ended |
Sep. 30, 2013 | |
Summary of significant accounting policies | ' |
Basis of Presentation | ' |
Basis of Presentation | |
The accompanying financial information as of September 30, 2013 and for the three and nine months ended September 30, 2012 and 2013 has been prepared by the Company, without audit, pursuant to the rules and regulations of the Securities and Exchange Commission (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, 2012 and notes thereto included in the final prospectus related to the Company’s initial public offering. | |
In the opinion of management, the unaudited financial information as of September 30, 2013 and for three and nine months ended September 30, 2012 and 2013 reflects all adjustments, which are normal recurring adjustments, necessary to present a fair statement of financial position, results of operations and cash flows. The results of operations for the three and nine month periods ended September 30, 2013 are not necessarily indicative of the results to be expected for the year ended December 31, 2013 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. | |
Reverse stock split | ' |
Reverse stock split | |
On March 7, 2013, the Company effected a one-for-120 reverse stock split of its common stock, pursuant to which each 120 shares of the Company’s outstanding common stock were reclassified and combined into one share of common stock. All common stock share and per share references in the financial statements and notes thereto have been restated to reflect the reverse stock split on a retroactive basis. | |
Marketable Securities | ' |
Marketable Securities | |
Management determines the classification of marketable securities at the time of purchase and reevaluates such designation as of each balance sheet date. Marketable securities are classified as available-for-sale and carried at fair value, with any unrealized gain or loss recorded as a separate component of stockholders’ (deficit) equity. | |
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. | |
The Company’s revenue is generated primarily through collaborative research and development and licensing agreements and grants. | |
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. | |
For existing collaborations entered into prior to the adoption in 2011 of the revised multiple element revenue recognition guidance described below, the Company recognized revenue consistent with the approach established at the inception of each arrangement. For these existing collaborations, where the Company has continuing involvement, the Company recorded nonrefundable, upfront fees as deferred revenue and recognizes revenue on a straight-line basis as collaboration revenue over the expected performance period. | |
For new collaborations or for material modifications made to existing collaborations, in 2011 and thereafter, the Company adopted the updated multiple element revenue recognition guidance. Under this guidance, all non-contingent arrangement consideration is allocated to the identified units of accounting based on their relative selling price at inception of the collaboration arrangement. The Company derives the selling price using a combination of internal subjective and available external objective information, such as comparable transactions. The Company recognizes revenue commensurate with delivery, such as in the case with delivery of a license, or ratably over the course of a service period, as appropriate, such as in the case of ongoing research and development activities. | |
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 milestone payments are 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 its 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 as substantive milestones and will be recognized as revenue in the period that the milestone is achieved. | |
The Company recognizes royalties as earned in accordance with the terms of various research and collaboration agreements. If not substantive, the contingent consideration is allocated to the existing units of accounting based on relative selling price and recognized following the same basis previously established for the associated unit of accounting. The Company recognizes revenue for reimbursements of research and development costs under collaboration agreements as the services are performed. The Company records these reimbursements as revenue and not as a reduction of research and development expenses as the Company has the risks and rewards as the principal in the research and development activities. | |
Fair value of financial instruments | ' |
Fair value of financial instruments | |
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. | |
Warrant liability | ' |
Warrant liability | |
Warrants to purchase the Company’s common stock with nonstandard antidilution provisions and preferred stock that include a put feature, regardless of the probability or likelihood that may conditionally obligate the issuer to ultimately transfer assets, are classified as liabilities and are recorded at their estimated fair value at each reporting period. Any change in fair value of these warrants is recorded as gain (loss) on warrant valuation each reporting period in Other income (expense), net on the Company’s statement of operations. | |
Share-based compensation | ' |
Share-based compensation | |
The Company measures the cost of employee services received in exchange for an award of equity instruments based on the grant date fair value of the award. For service type awards, share-based compensation expense is recognized on a straight-line basis over the period during which the employee is required to provide service in exchange for the entire award. For awards that vest or begin vesting upon achievement of a performance condition, the Company estimates the likelihood of satisfaction of the performance condition and recognizes compensation expense when achievement of the performance condition is deemed probable using an accelerated attribution model. | |
The fair value of options is calculated using the Black-Scholes option pricing model to determine the fair value of stock options on the date of grant based on key assumptions, such as expected volatility and expected term. As a new public company, the Company does not have sufficient history to estimate the volatility of its common stock price or the expected life of the options. The Company calculates expected volatility based on reported data for similar publicly traded companies for which historical information is available and will continue to do so until the historical volatility of its common stock is sufficient to measure expected volatility for future option grants. |
Fair_value_of_financial_instru1
Fair value of financial instruments and marketable securities (Tables) | 9 Months Ended | |||||||||||||
Sep. 30, 2013 | ||||||||||||||
Fair value of financial instruments and marketable securities | ' | |||||||||||||
Schedule of financial assets that are required to be measured at fair value on a recurring basis | ' | |||||||||||||
December 31, 2012 | ||||||||||||||
Total | Quoted prices | Significant | Significant | |||||||||||
in active | other | unobservable | ||||||||||||
markets for | observable | inputs | ||||||||||||
identical assets | inputs | (level 3) | ||||||||||||
(level 1) | (level 2) | |||||||||||||
Warrant liability | $ | 95,661 | $ | — | $ | — | $ | 95,661 | ||||||
September 30, 2013 | ||||||||||||||
Total | Quoted prices | Significant | Significant | |||||||||||
in active | other | unobservable | ||||||||||||
markets for | observable | inputs | ||||||||||||
identical assets | inputs | (level 3) | ||||||||||||
(level 1) | (level 2) | |||||||||||||
Marketable securities | $ | 133,182,565 | $ | — | $ | 133,182,565 | $ | — | ||||||
Warrant liability | 98,819 | — | — | 98,819 | ||||||||||
Summary of marketable securities accounted for as available-for-sale securities | ' | |||||||||||||
September 30, 2013 | ||||||||||||||
Amortized | Gross Unrealized | Fair | ||||||||||||
Cost | Gains | Losses | Value | |||||||||||
Commercial paper | $ | 24,978,703 | $ | 20,527 | $ | — | $ | 24,999,230 | ||||||
U.S. corporate debt securities | 108,244,329 | 19,642 | (80,636 | ) | 108,183,335 | |||||||||
$ | 133,223,032 | $ | 40,169 | $ | (80,636 | ) | $ | 133,182,565 | ||||||
Schedule of marketable securities on the balance sheet | ' | |||||||||||||
September 30, 2013 | ||||||||||||||
Less Than | More Than | |||||||||||||
12 Months | 12 Months | |||||||||||||
Commercial paper | $ | 24,999,230 | $ | — | ||||||||||
U.S. corporate debt securities | 57,824,657 | 50,358,678 | ||||||||||||
Total Marketable securities | $ | 82,823,887 | $ | 50,358,678 | ||||||||||
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, 2012 | $ | 95,661 | ||||||||||||
Warrants issued in connection with convertible promissory notes | 6,000,000 | |||||||||||||
Deemed dividend | 18,248,768 | |||||||||||||
Exercise of warrants issued in connection with convertible promissory notes | (24,248,765 | ) | ||||||||||||
Change in fair value of warrant liability | 3,155 | |||||||||||||
Ending balance as of September 30, 2013 | $ | 98,819 |
Other_comprehensive_income_los1
Other comprehensive income (loss) and accumulated other comprehensive items (Tables) | 9 Months Ended | |||||||
Sep. 30, 2013 | ||||||||
Other comprehensive income (loss) and accumulated other comprehensive items | ' | |||||||
Summary of other comprehensive loss and the changes in accumulated other comprehensive items | ' | |||||||
Unrealized Losses | Total | |||||||
On Marketable | Accumulated | |||||||
Securities | Other | |||||||
Comprehensive | ||||||||
Items | ||||||||
Balance at June 30, 2013 | $ | (772 | ) | $ | (772 | ) | ||
Other comprehensive loss before reclassifications | (39,695 | ) | (39,695 | ) | ||||
Amounts reclassified from other comprehensive items | — | — | ||||||
Other comprehensive loss | (39,695 | ) | (39,695 | ) | ||||
Balance at September 30, 2013 | $ | (40,467 | ) | $ | (40,467 | ) | ||
Unrealized Losses | Total | |||||||
On Marketable | Accumulated | |||||||
Securities | Other | |||||||
Comprehensive | ||||||||
Items | ||||||||
Balance at December 31, 2012 | $ | — | $ | — | ||||
Other comprehensive loss before reclassifications | (40,467 | ) | (40,467 | ) | ||||
Amounts reclassified from other comprehensive items | — | — | ||||||
Other comprehensive loss | (40,467 | ) | (40,467 | ) | ||||
Balance at September 30, 2013 | $ | (40,467 | ) | $ | (40,467 | ) |
Accounts_payable_and_accrued_e1
Accounts payable and accrued expenses (Tables) | 9 Months Ended | |||||||
Sep. 30, 2013 | ||||||||
Accounts payable and accrued expenses | ' | |||||||
Schedule of components of accounts payable and accrued expenses | ' | |||||||
December 31, | September 30, | |||||||
2012 | 2013 | |||||||
Employee compensation, benefits, and related accruals | $ | 3,096,475 | $ | 3,659,084 | ||||
Consulting and contracted research | 2,515,678 | 3,556,350 | ||||||
Professional fees | 559,228 | 1,532,458 | ||||||
Accounts payable | 621,591 | 1,017,122 | ||||||
Other | 230,999 | 440,479 | ||||||
$ | 7,023,971 | $ | 10,205,493 |
Capital_structure_Tables
Capital structure (Tables) | 9 Months Ended | ||||||||
Sep. 30, 2013 | |||||||||
Capital structure | ' | ||||||||
Summary of the Company's outstanding warrants | ' | ||||||||
The following is a summary of the Company’s outstanding warrants as of December 31, 2012: | |||||||||
Warrant | Exercise | Expiration | |||||||
shares | price | ||||||||
Series Two | 24,712 | $ | 16 | 2014 | |||||
Series Two | 50,000 | $ | 16 | 2017 | |||||
Series Two | 56,250 | $ | 16 | 2019 and 2020 | |||||
Common stock | 645 | $ | 2,520 | 2013 and 2014 | |||||
The following is a summary of the Company’s outstanding warrants as of September 30, 2013: | |||||||||
Warrant | Exercise | Expiration | |||||||
shares | price | ||||||||
Common stock | 3,088 | $ | 128 | 2014 | |||||
Common stock | 6,250 | $ | 128 | 2017 | |||||
Common stock | 7,030 | $ | 128 | 2019 and 2020 | |||||
Common stock | 645 | $ | 2,520 | 2013 and 2014 |
Net_income_loss_per_share_Tabl
Net income (loss) per share (Tables) | 9 Months Ended | |||||||
Sep. 30, 2013 | ||||||||
Net income (loss) per share | ' | |||||||
Schedule of computation of basic and diluted net income (loss) per share for common stockholders | ' | |||||||
Three months ended September 30, | ||||||||
2012 | 2013 | |||||||
Numerator | ||||||||
Net loss attributable to common stockholders | $ | (7,297,125 | ) | $ | (4,416,499 | ) | ||
Denominator | ||||||||
Denominator for basic net loss per share | 4,545 | 23,803,282 | ||||||
Net loss per share: | ||||||||
Basic and diluted | $ | (1,605.53 | )* | $ | (0.19 | )* | ||
* In the three months ended September 30, 2012 and 2013, the Company experienced a net loss and therefore did not report any dilutive share impact. | ||||||||
Nine months ended September 30, | ||||||||
2012 | 2013 | |||||||
Numerator | ||||||||
Net loss | $ | (19,963,058 | ) | $ | (33,687,612 | ) | ||
Deemed dividend | — | (18,248,768 | ) | |||||
Gain on exchange of convertible preferred stock in connection with recapitalization | 159,954,069 | 3,390,750 | ||||||
Less beneficial conversion charge | (377,787 | ) | — | |||||
Less net income attributable to participating securities | (139,077,495 | ) | — | |||||
Net income (loss) attributable to common stockholders | $ | 535,729 | $ | (48,545,630 | ) | |||
Denominator | ||||||||
Denominator for basic net income (loss) per share | 2,937 | 8,995,167 | ||||||
Effect of dilutive securities | ||||||||
Series Three convertible preferred stock | 10,656 | — | ||||||
Denominator for diluted net income (loss) per share | 13,593 | 8,995,167 | ||||||
Net income (loss) per share: | ||||||||
Basic | $ | 182.41 | $ | (5.40 | )* | |||
Diluted | $ | 39.41 | $ | (5.40 | )* | |||
* In the nine months ended September 30, 2013, the Company experienced a net loss and therefore did not report any dilutive share impact. | ||||||||
Schedule of historical dilutive common share equivalents outstanding | ' | |||||||
As of September 30, | ||||||||
2012 | 2013 | |||||||
Stock Options | 44,218 | 1,987,860 | ||||||
Unvested restricted stock | — | 1,113,807 | ||||||
Total | 44,218 | 3,101,667 |
Stock_option_plan_Tables
Stock option plan (Tables) | 9 Months Ended | ||||||||||||||
Sep. 30, 2013 | |||||||||||||||
Stock option plan | ' | ||||||||||||||
Summary of stock option activity | ' | ||||||||||||||
Number of | Exercise | Weighted- | Weighted- | Aggregate | |||||||||||
options | price | average | average | intrinsic | |||||||||||
exercise | remaining | value | |||||||||||||
price | contractual | ||||||||||||||
term | |||||||||||||||
(in thousands) | |||||||||||||||
Outstanding at December 31, 2012 | 42,394 | $ | 218.40-$1,149.60 | $ | 469.48 | ||||||||||
Granted | 2,007,713 | $ | 10.59-$10.85 | $ | 10.85 | ||||||||||
Exercised | — | — | — | ||||||||||||
Forfeited | (62,247 | ) | $ | 10.85—$1,149.60 | $ | 17.1 | |||||||||
Outstanding at September 30, 2013 | 1,987,860 | $ | 10.59—$1,149.60 | $ | 20.44 | 9.51 years | $ | 20,652 | |||||||
Exercisable at September 30, 2013 | 61,834 | $ | 10.59—$1,149.60 | $ | 290.39 | 6.06 years | $ | 265 | |||||||
Schedule of assumptions used to estimate fair value of grants made on the date of grant | ' | ||||||||||||||
Nine months ended | |||||||||||||||
September 30, 2013 | |||||||||||||||
Risk-free interest rate | 0.85%-1.69% | ||||||||||||||
Expected volatility | 87%-88% | ||||||||||||||
Expected term | 5.00 to 6.00 years | ||||||||||||||
Expected dividend yield | 0.00% | ||||||||||||||
Summary of information on the Company's restricted stock | ' | ||||||||||||||
Restricted Stock | |||||||||||||||
Number of | Weighted | ||||||||||||||
Shares | Average Grant | ||||||||||||||
Date Fair Value | |||||||||||||||
January 1, 2013 | — | — | |||||||||||||
Granted | 1,131,524 | $ | 10.68 | ||||||||||||
Vested | — | $ | — | ||||||||||||
Forfeited | (17,717 | ) | $ | 10.63 | |||||||||||
Unvested at September 30, 2013 | 1,113,807 | $ | 10.68 | ||||||||||||
Schedule of share-based compensation expense recorded in the statement of operations | ' | ||||||||||||||
Three Months Ended September 30, | Nine Months Ended September 30, | ||||||||||||||
2012 | 2013 | 2012 | 2013 | ||||||||||||
Research and development | $ | 193,199 | $ | 676,499 | $ | 653,462 | $ | 1,377,640 | |||||||
General and administrative | 327,201 | 1,963,197 | 1,162,260 | 3,764,547 | |||||||||||
Total | $ | 520,400 | $ | 2,639,696 | $ | 1,815,722 | $ | 5,142,187 |
The_Company_Details
The Company (Details) (USD $) | Sep. 30, 2013 | Dec. 31, 2012 |
The Company | ' | ' |
Accumulated deficit | $310,912,330 | $277,224,718 |
Summary_of_significant_account2
Summary of significant accounting policies (Details) (Common stock) | 0 Months Ended |
Mar. 07, 2013 | |
Common stock | ' |
Reverse stock split | ' |
Reverse stock split ratio | 0.0083 |
Fair_value_of_financial_instru2
Fair value of financial instruments and marketable securities (Details) (USD $) | Sep. 30, 2013 | Dec. 31, 2012 |
Financial assets measured at fair value on recurring basis | ' | ' |
Marketable securities | $133,182,565 | ' |
Recurring basis | Total | ' | ' |
Financial assets measured at fair value on recurring basis | ' | ' |
Marketable securities | 133,182,565 | ' |
Warrant liability | 98,819 | 95,661 |
Recurring basis | Significant other observable inputs (level 2) | ' | ' |
Financial assets measured at fair value on recurring basis | ' | ' |
Marketable securities | 133,182,565 | ' |
Recurring basis | Significant unobservable inputs (level 3) | ' | ' |
Financial assets measured at fair value on recurring basis | ' | ' |
Warrant liability | $98,819 | $95,661 |
Fair_value_of_financial_instru3
Fair value of financial instruments and marketable securities (Details 2) (USD $) | 9 Months Ended |
Sep. 30, 2013 | |
Marketable securities accounted for as available-for-sale securities | ' |
Amortized Cost | $133,223,032 |
Gross Unrealized Gains | 40,169 |
Gross Unrealized Losses | -80,636 |
Total Marketable securities | 133,182,565 |
Commercial paper | ' |
Marketable securities accounted for as available-for-sale securities | ' |
Amortized Cost | 24,978,703 |
Gross Unrealized Gains | 20,527 |
Total Marketable securities | 24,999,230 |
U.S. corporate debt securities | ' |
Marketable securities accounted for as available-for-sale securities | ' |
Amortized Cost | 108,244,329 |
Gross Unrealized Gains | 19,642 |
Gross Unrealized Losses | -80,636 |
Total Marketable securities | $108,183,335 |
Fair_value_of_financial_instru4
Fair value of financial instruments and marketable securities (Details 3) (USD $) | Sep. 30, 2013 | Dec. 31, 2012 |
Marketable securities on the balance sheet | ' | ' |
Total Marketable securities, Less Than 12 Months | $82,823,887 | ' |
Total Marketable securities, More Than 12 Months | 50,358,678 | ' |
Marketable securities | ' | 0 |
Commercial paper | ' | ' |
Marketable securities on the balance sheet | ' | ' |
Total Marketable securities, Less Than 12 Months | 24,999,230 | ' |
U.S. corporate debt securities | ' | ' |
Marketable securities on the balance sheet | ' | ' |
Total Marketable securities, Less Than 12 Months | 57,824,657 | ' |
Total Marketable securities, More Than 12 Months | $50,358,678 | ' |
Fair_value_of_financial_instru5
Fair value of financial instruments and marketable securities (Details 4) (USD $) | 9 Months Ended | 12 Months Ended |
Sep. 30, 2013 | Dec. 31, 2012 | |
Changes in the fair value of warrant liability | ' | ' |
Deemed dividend | $18,248,768 | ' |
Warrant | ' | ' |
Changes in the fair value of warrant liability | ' | ' |
Beginning balance | 95,661 | ' |
Warrants issued in connection with convertible promissory notes | 6,000,000 | ' |
Deemed dividend | 18,248,768 | ' |
Exercise of warrants issued in connection with convertible promissory notes | -24,248,765 | ' |
Change in fair value of warrant liability | 3,155 | ' |
Ending balance | $98,819 | $95,661 |
Assumption used to estimate the fair value of warrant liability by utilizing the Black-Scholes option-pricing model | ' | ' |
Volatility (as a percent) | ' | 87.00% |
Strike price (in dollars per share) | $128 | $16 |
Warrant | Minimum | ' | ' |
Assumption used to estimate the fair value of warrant liability by utilizing the Black-Scholes option-pricing model | ' | ' |
Volatility (as a percent) | 69.00% | ' |
Risk-free interest rate (as a percent) | 0.02% | 0.16% |
Expected life | '2 months 19 days | '1 year |
Warrant | Maximum | ' | ' |
Assumption used to estimate the fair value of warrant liability by utilizing the Black-Scholes option-pricing model | ' | ' |
Volatility (as a percent) | 87.00% | ' |
Risk-free interest rate (as a percent) | 1.71% | 1.18% |
Expected life | '5 years 11 months 23 days | '7 years |
Warrant | Preferred shares | ' | ' |
Assumption used to estimate the fair value of warrant liability by utilizing the Black-Scholes option-pricing model | ' | ' |
Fair value of common shares (in dollars per share) | $21.46 | $2.35 |
Other_comprehensive_income_los2
Other comprehensive income (loss) and accumulated other comprehensive items (Details) (USD $) | 3 Months Ended | 9 Months Ended |
Sep. 30, 2013 | Sep. 30, 2013 | |
Other comprehensive income (loss) and accumulated other comprehensive items | ' | ' |
Balance at the beginning of the period | ($772) | ' |
Other comprehensive loss before reclassifications | -39,695 | -40,467 |
Other comprehensive loss | -39,695 | -40,467 |
Balance at the end of the period | -40,467 | -40,467 |
Unrealized Losses On Marketable Securities | ' | ' |
Other comprehensive income (loss) and accumulated other comprehensive items | ' | ' |
Balance at the beginning of the period | -772 | ' |
Other comprehensive loss before reclassifications | -39,695 | -40,467 |
Other comprehensive loss | -39,695 | -40,467 |
Balance at the end of the period | ($40,467) | ($40,467) |
Accounts_payable_and_accrued_e2
Accounts payable and accrued expenses (Details) (USD $) | Sep. 30, 2013 | Dec. 31, 2012 |
Accounts payable and accrued expenses | ' | ' |
Employee compensation, benefits, and related accruals | $3,659,084 | $3,096,475 |
Consulting and contracted research | 3,556,350 | 2,515,678 |
Professional fees | 1,532,458 | 559,228 |
Accounts payable | 1,017,122 | 621,591 |
Other | 440,479 | 230,999 |
Accounts payable and accrued expenses | $10,205,493 | $7,023,971 |
Debt_Details
Debt (Details) (USD $) | 1 Months Ended | 3 Months Ended | 9 Months Ended |
Jul. 31, 2013 | Sep. 30, 2013 | Sep. 30, 2013 | |
Debt | ' | ' | ' |
Paid outstanding principal and interest, due under promissory notes issued | $2,600,000 | ' | ' |
Loss on extinguishment of debt | ' | -129,963 | -129,963 |
Secured debt facility | ' | ' | ' |
Debt | ' | ' | ' |
Maximum borrowing capacity | $25,000,000 | ' | ' |
Number of lenders | 2 | ' | ' |
Fixed interest rate of notes (as a percent) | 13.65% | ' | ' |
Capital_structure_Details
Capital structure (Details) (USD $) | 3 Months Ended | 9 Months Ended | 0 Months Ended | 0 Months Ended | 1 Months Ended | 0 Months Ended | 1 Months Ended | 3 Months Ended | 0 Months Ended | ||||||||
Mar. 31, 2013 | Sep. 30, 2013 | Sep. 30, 2012 | 31-May-13 | Dec. 31, 2012 | Mar. 07, 2013 | Feb. 28, 2013 | Mar. 07, 2013 | 31-May-13 | Mar. 07, 2013 | Mar. 07, 2013 | Mar. 07, 2013 | Jun. 30, 2013 | Jun. 30, 2013 | Mar. 31, 2013 | Feb. 28, 2013 | Mar. 07, 2013 | |
Series One and Series Two | Series One and Series Two | Series Four | Series Four | Series Five | Series Five | Common stock | Common stock | Series Four and Series Five Convertible Preferred Stock | Convertible promissory notes | Convertible promissory notes | Convertible promissory notes | ||||||
Warrants | Warrants | Warrants | Series Four | ||||||||||||||
Capital structure | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' |
Principal amount of convertible promissory notes issued | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | $6,000,000 | ' |
Warrants issued to purchase shares | ' | ' | ' | ' | ' | ' | 2,527,675 | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' |
Exercise price (in dollars per share) | ' | ' | ' | ' | ' | ' | $0.01 | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' |
Debt discount recorded to interest expense | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | 6,000,000 | ' | ' |
Shares issued and sold | ' | ' | ' | ' | ' | 2,527,675 | ' | 4,497,035 | 375,000 | ' | ' | ' | 9,627,800 | ' | ' | ' | ' |
Aggregate gross proceeds | ' | ' | 29,354,752 | ' | ' | ' | ' | 54,000,000 | ' | ' | ' | ' | ' | ' | ' | ' | ' |
Amount raised in bridge financing | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | 6,000,000 | ' | ' |
Gross proceeds raised | 60,000,000 | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' |
Shares issued upon conversion of convertible promissory notes | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | 502,919 |
Reverse stock split ratio | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | 0.0083 | ' | ' | ' | ' | ' |
Number of shares issued upon conversion | ' | ' | ' | ' | ' | ' | ' | ' | ' | 6,700,487 | 2,095,515 | ' | ' | 14,170,956 | ' | ' | ' |
Gain on extinguishment of shares of Series One, Two and Three stock | ' | 3,390,750 | 159,954,069 | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' |
Share price (in dollars per share) | ' | ' | ' | ' | ' | ' | ' | ' | $12 | ' | ' | ' | $15 | ' | ' | ' | ' |
Aggregate purchase price | ' | ' | ' | ' | ' | ' | ' | ' | 4,500,000 | ' | ' | ' | ' | ' | ' | ' | ' |
Common stock | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' |
Common stock, Authorized shares | ' | 125,000,000 | ' | 125,000,000 | 17,000,000 | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' |
Initial Public Offering | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' |
Common stock shares issuable pursuant to the exercise by the underwriters of an over-allotment option | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | 1,255,800 | ' | ' | ' | ' |
Net proceeds from initial public offering after deducting underwriting discounts and commissions and other offering expenses payable | ' | $131,649,688 | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | $131,600,000 | ' | ' | ' | ' |
Capital_structure_Details_2
Capital structure (Details 2) (Warrants, USD $) | Sep. 30, 2013 | Dec. 31, 2012 |
Series Two | 2014 | ' | ' |
Warrants | ' | ' |
Warrant shares | ' | 24,712 |
Exercise price (in dollars per share) | ' | $16 |
Series Two | 2017 | ' | ' |
Warrants | ' | ' |
Warrant shares | ' | 50,000 |
Exercise price (in dollars per share) | ' | $16 |
Series Two | 2019 and 2020 | ' | ' |
Warrants | ' | ' |
Warrant shares | ' | 56,250 |
Exercise price (in dollars per share) | ' | $16 |
Common stock | 2014 | ' | ' |
Warrants | ' | ' |
Warrant shares | 3,088 | ' |
Exercise price (in dollars per share) | $128 | ' |
Common stock | 2017 | ' | ' |
Warrants | ' | ' |
Warrant shares | 6,250 | ' |
Exercise price (in dollars per share) | $128 | ' |
Common stock | 2019 and 2020 | ' | ' |
Warrants | ' | ' |
Warrant shares | 7,030 | ' |
Exercise price (in dollars per share) | $128 | ' |
Common stock | 2013 and 2014 | ' | ' |
Warrants | ' | ' |
Warrant shares | 645 | 645 |
Exercise price (in dollars per share) | $2,520 | $2,520 |
Net_income_loss_per_share_Deta
Net income (loss) per share (Details) (USD $) | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2013 | Sep. 30, 2012 | Sep. 30, 2013 | Sep. 30, 2012 | |
Numerator | ' | ' | ' | ' |
Net loss | ($4,416,499) | ($7,297,125) | ($33,687,612) | ($19,963,058) |
Deemed dividend | ' | ' | -18,248,768 | ' |
Gain on exchange of convertible preferred stock in connection with recapitalization | ' | ' | 3,390,750 | 159,954,069 |
Less beneficial conversion charge | ' | ' | ' | -377,787 |
Less net income attributable to participating securities | ' | ' | ' | -139,077,495 |
Net loss attributable to common stockholders | ($4,416,499) | ($7,297,125) | ($48,545,630) | $535,729 |
Denominator | ' | ' | ' | ' |
Denominator for basic net income (loss) per share (in shares) | 23,803,282 | 4,545 | 8,995,167 | 2,937 |
Effect of dilutive securities Series Three convertible preferred stock (in shares) | ' | ' | ' | 10,656 |
Denominator for diluted net income (loss) per share (in shares) | 23,803,282 | 4,545 | 8,995,167 | 13,593 |
Net loss per share: | ' | ' | ' | ' |
Basic and diluted (in dollars per share) | ($0.19) | ($1,605.53) | ' | ' |
Basic (in dollars per share) | ($0.19) | ($1,605.53) | ($5.40) | $182.41 |
Diluted (in dollars per share) | ($0.19) | ($1,605.53) | ($5.40) | $39.41 |
Net_income_loss_per_share_Deta1
Net income (loss) per share (Details 2) | 9 Months Ended | |
Sep. 30, 2013 | Sep. 30, 2012 | |
Net income (loss) per share | ' | ' |
Total shares excluded from calculation | 3,101,667 | 44,218 |
Stock Options | ' | ' |
Net income (loss) per share | ' | ' |
Total shares excluded from calculation | 1,987,860 | 44,218 |
Unvested restricted stock | ' | ' |
Net income (loss) per share | ' | ' |
Total shares excluded from calculation | 1,113,807 | ' |
Stock_option_plan_Details
Stock option plan (Details) (USD $) | 9 Months Ended | 0 Months Ended | 1 Months Ended | ||||||||
In Thousands, except Share data, unless otherwise specified | Sep. 30, 2013 | Sep. 30, 2013 | Sep. 30, 2013 | Sep. 30, 2013 | Mar. 05, 2013 | Mar. 05, 2013 | Mar. 05, 2013 | 31-May-13 | 31-May-13 | 31-May-13 | 31-May-13 |
Restricted stock | Stock options | Stock options | Stock options | 2013 Stock Incentive Plan | 2013 Stock Incentive Plan | 2013 Stock Incentive Plan | 2009 Stock Incentive Plan | 2013 Long Term Incentive Plan | 2013 Long Term Incentive Plan | 2009 Equity and Long Term Incentive Plan and 2013 Stock Incentive Plan | |
Minimum | Maximum | Restricted stock | Stock options | Common stock | Common stock | Common stock | Common stock | ||||
Minimum | Maximum | ||||||||||
Stock option plan | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' |
Number of shares authorized | ' | ' | ' | ' | ' | ' | 739,937 | ' | ' | ' | ' |
Number of additional shares authorized | ' | ' | ' | ' | ' | ' | ' | 2,500,000 | ' | ' | ' |
Number of shares available for issuance | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | 122,296 |
Number of shares subject to outstanding awards | ' | ' | ' | ' | ' | ' | ' | ' | ' | 3,040,444 | ' |
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% | ' | ' |
Number of options | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' |
Outstanding at the beginning of the period (in shares) | ' | 42,394 | ' | ' | ' | ' | ' | ' | ' | ' | ' |
Granted (in shares) | ' | 2,007,713 | ' | ' | 735,324 | 4,613 | ' | ' | ' | ' | ' |
Forfeited (in shares) | ' | -62,247 | ' | ' | ' | ' | ' | ' | ' | ' | ' |
Outstanding at the end of the period (in shares) | ' | 1,987,860 | ' | ' | ' | ' | ' | ' | ' | ' | ' |
Exercisable at the end of the period (in shares) | ' | 61,834 | ' | ' | ' | ' | ' | ' | ' | ' | ' |
Exercise price | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' |
Outstanding at the beginning of the period (in dollars per share) | ' | ' | $218.40 | $1,149.60 | ' | ' | ' | ' | ' | ' | ' |
Granted (in dollars per share) | ' | ' | $10.59 | $10.85 | ' | ' | ' | ' | ' | ' | ' |
Forfeited (in dollars per share) | ' | ' | $10.85 | $1,149.60 | ' | ' | ' | ' | ' | ' | ' |
Outstanding at the end of the period (in dollars per share) | ' | ' | $10.59 | $1,149.60 | ' | ' | ' | ' | ' | ' | ' |
Exercisable at the end of the period (in dollars per share) | ' | ' | $10.59 | $1,149.60 | ' | ' | ' | ' | ' | ' | ' |
Weighted-average exercise price | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' |
Outstanding at the beginning of the period (in dollars per share) | ' | $469.48 | ' | ' | ' | ' | ' | ' | ' | ' | ' |
Granted (in dollars per share) | ' | $10.85 | ' | ' | ' | ' | ' | ' | ' | ' | ' |
Forfeited (in dollars per share) | ' | $17.10 | ' | ' | ' | ' | ' | ' | ' | ' | ' |
Outstanding at the end of the period (in dollars per share) | ' | $20.44 | ' | ' | ' | ' | ' | ' | ' | ' | ' |
Exercisable at the end of the period (in dollars per share) | ' | $290.39 | ' | ' | ' | ' | ' | ' | ' | ' | ' |
Weighted-average remaining contractual term | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' |
Outstanding at the end of the period | ' | '9 years 6 months 4 days | ' | ' | ' | ' | ' | ' | ' | ' | ' |
Exercisable at the end of the period | ' | '6 years 22 days | ' | ' | ' | ' | ' | ' | ' | ' | ' |
Aggregate intrinsic value | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' |
Outstanding at the end of the period (in dollars) | ' | $20,652 | ' | ' | ' | ' | ' | ' | ' | ' | ' |
Exercisable at the end of the period (in dollars) | ' | $265 | ' | ' | ' | ' | ' | ' | ' | ' | ' |
Valuation assumptions | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' |
Risk-free interest rate (as a percent) | ' | ' | 0.85% | 1.69% | ' | ' | ' | ' | ' | ' | ' |
Expected volatility (as a percent) | ' | ' | 87.00% | 88.00% | ' | ' | ' | ' | ' | ' | ' |
Expected term | ' | ' | '5 years | '6 years | ' | ' | ' | ' | ' | ' | ' |
Expected dividend yield (as a percent) | ' | 0.00% | ' | ' | ' | ' | ' | ' | ' | ' | ' |
Weighted average grant date fair value (in dollars per share) | ' | $7.86 | ' | ' | ' | ' | ' | ' | ' | ' | ' |
Number of Shares | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' |
Granted (in shares) | 1,131,524 | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' |
Forfeited (in shares) | -17,717 | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' |
Unvested at the end of the period (in shares) | 1,113,807 | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' |
Weighted Average Grant Date Fair Value | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' |
Granted (in dollars per share) | $10.68 | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' |
Forfeited (in dollars per share) | $10.63 | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' |
Unvested at the end of the period (in dollars per share) | $10.68 | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' |
Stock_option_plan_Details_2
Stock option plan (Details 2) (USD $) | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2013 | Sep. 30, 2012 | Sep. 30, 2013 | Sep. 30, 2012 | |
Share-based compensation expense recorded in the statement of operations | ' | ' | ' | ' |
Share-based compensation expense | $2,639,696 | $520,400 | $5,142,187 | $1,815,722 |
Research and development | ' | ' | ' | ' |
Share-based compensation expense recorded in the statement of operations | ' | ' | ' | ' |
Share-based compensation expense | 676,499 | 193,199 | 1,377,640 | 653,462 |
General and administrative | ' | ' | ' | ' |
Share-based compensation expense recorded in the statement of operations | ' | ' | ' | ' |
Share-based compensation expense | $1,963,197 | $327,201 | $3,764,547 | $1,162,260 |
Stock_option_plan_Details_3
Stock option plan (Details 3) (1998, 2009 and 2013 Plans, USD $) | 9 Months Ended | |
In Millions, unless otherwise specified | Sep. 30, 2013 | Dec. 31, 2012 |
1998, 2009 and 2013 Plans | ' | ' |
Additional disclosures | ' | ' |
Unrecognized compensation cost | $21 | $2.20 |
Weighted average remaining service period for recognition of unrecognized compensation cost | '2 years 3 months 11 days | ' |
Collaboration_Revenue_Details
Collaboration Revenue (Details) (USD $) | 0 Months Ended |
In Millions, unless otherwise specified | Aug. 08, 2013 |
Collaboration Revenue. | ' |
Milestone payment received from Roche | $10 |