Document and Entity Information
Document and Entity Information - shares | 6 Months Ended | |
Jun. 30, 2016 | Jul. 29, 2016 | |
Document And Entity Information [Abstract] | ||
Document Type | 10-Q | |
Amendment Flag | false | |
Document Period End Date | Jun. 30, 2016 | |
Document Fiscal Year Focus | 2,016 | |
Document Fiscal Period Focus | Q2 | |
Trading Symbol | ADVM | |
Entity Registrant Name | Adverum Biotechnologies, Inc. | |
Entity Central Index Key | 1,501,756 | |
Current Fiscal Year End Date | --12-31 | |
Entity Filer Category | Accelerated Filer | |
Entity Common Stock, Shares Outstanding | 41,348,194 |
Condensed Consolidated Balance
Condensed Consolidated Balance Sheets (Unaudited) - USD ($) $ in Thousands | Jun. 30, 2016 | Dec. 31, 2015 |
Current assets: | ||
Cash and cash equivalents | $ 241,302 | $ 221,348 |
Marketable securities | 37,732 | |
Receivable from collaborative partner | 1,029 | 449 |
Prepaid expenses and other current assets | 1,239 | 1,463 |
Total current assets | 243,570 | 260,992 |
Property and equipment, net | 3,985 | 3,187 |
Intangible assets | 16,650 | |
Deposit and other long-term assets | 140 | 140 |
Total assets | 264,345 | 264,319 |
Current liabilities: | ||
Accounts payable | 2,419 | 605 |
Restructuring liabilities | 25 | 1,013 |
Accrued expenses and other current liabilities | 4,144 | 4,007 |
Deferred rent, current portion | 81 | 66 |
Deferred revenue, current portion | 1,228 | 883 |
Total current liabilities | 7,897 | 6,574 |
Long-term liabilities: | ||
Deferred rent, net of current portion | 404 | 447 |
Deferred revenue, net of current portion | 5,936 | 4,706 |
Deferred tax liability | 2,081 | |
Other noncurrent liabilities | 373 | |
Total liabilities | 16,691 | 11,727 |
Commitments and contingencies (Note 8) | ||
Stockholders’ equity: | ||
Preferred stock, $0.0001 par value, 5,000,000 shares authorized; no shares issued and outstanding | ||
Common stock, $0.0001 par value, 300,000,000 shares authorized at June 30, 2016 and December 31, 2015; 41,322,357 and 25,858,722 shares issued and outstanding at June 30, 2016 and December 31, 2015, respectively | 4 | 3 |
Additional paid-in capital | 408,866 | 336,768 |
Accumulated other comprehensive income (loss) | 4 | (11) |
Accumulated deficit | (161,220) | (84,168) |
Total stockholders’ equity | 247,654 | 252,592 |
Total liabilities and stockholders’ equity | $ 264,345 | $ 264,319 |
Condensed Consolidated Balance3
Condensed Consolidated Balance Sheets (Parenthetical) (Unaudited) - $ / shares | Jun. 30, 2016 | Dec. 31, 2015 |
Statement Of Financial Position [Abstract] | ||
Preferred stock, par value | $ 0.0001 | $ 0.0001 |
Preferred stock, shares authorized | 5,000,000 | 5,000,000 |
Preferred stock, shares issued | 0 | 0 |
Preferred stock, shares outstanding | 0 | 0 |
Common stock, par value | $ 0.0001 | $ 0.0001 |
Common stock, shares authorized | 300,000,000 | 300,000,000 |
Common stock, shares issued | 41,322,357 | 25,858,722 |
Common stock, shares outstanding | 41,322,357 | 25,858,722 |
Condensed Consolidated Statemen
Condensed Consolidated Statements of Operations and Comprehensive Loss (Unaudited) - USD ($) shares in Thousands, $ in Thousands | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2016 | Jun. 30, 2015 | Jun. 30, 2016 | Jun. 30, 2015 | |
Income Statement [Abstract] | ||||
Collaboration revenue | $ 307 | $ 203 | $ 572 | $ 406 |
Operating expenses: | ||||
Research and development | 7,955 | 5,126 | 15,410 | 10,747 |
General and administrative | 5,114 | 4,959 | 13,432 | 9,102 |
Goodwill impairment charge | 49,120 | 49,120 | ||
Total operating expenses | 62,189 | 10,085 | 77,962 | 19,849 |
Operating loss | (61,882) | (9,882) | (77,390) | (19,443) |
Other income | ||||
Other income, net | 222 | 116 | 338 | 168 |
Total other income, net | 222 | 116 | 338 | 168 |
Net loss | (61,660) | (9,766) | (77,052) | (19,275) |
Other comprehensive loss: | ||||
Net unrealized gain on marketable securities | 45 | 6 | 10 | |
Foreign currency translation adjustment | (4) | 10 | (8) | |
Comprehensive loss | $ (61,664) | $ (9,721) | $ (77,036) | $ (19,273) |
Net loss per share attributable to common stockholders-basic and diluted | $ (1.76) | $ (0.38) | $ (2.50) | $ (0.76) |
Weighted-average common shares outstanding-basic and diluted | 35,044 | 25,555 | 30,825 | 25,223 |
Condensed Consolidated Stateme5
Condensed Consolidated Statements of Cash Flows (Unaudited) - USD ($) | 6 Months Ended | |
Jun. 30, 2016 | Jun. 30, 2015 | |
Cash flows from operating activities: | ||
Net loss | $ (77,052,000) | $ (19,275,000) |
Adjustments to reconcile net loss to net cash used in operating activities: | ||
Depreciation and amortization | 663,000 | 265,000 |
Stock-based compensation expense | 6,903,000 | 1,332,000 |
Goodwill impairment charge | 49,120,000 | |
Amortization of premium on marketable securities | 317,000 | |
Changes in operating assets and liabilities: | ||
Receivable from collaborative partner | (580,000) | |
Prepaid expenses and other current assets | 1,069,000 | (47,000) |
Deposit and other long-term assets | (1,000) | |
Accounts payable | 646,000 | 534,000 |
Accrued expenses and other current liabilities | (1,706,000) | 59,000 |
Restructuring liabilities | (988,000) | |
Deferred revenue | 1,575,000 | (406,000) |
Deferred rent | (27,000) | 230,000 |
Net cash used in operating activities | (20,377,000) | (16,992,000) |
Cash flows from investing activities: | ||
Purchases of marketable securities | (88,429,000) | |
Maturities of marketable securities | 37,738,000 | 8,960,000 |
Purchases of property and equipment | (1,126,000) | (1,485,000) |
Cash acquired in business acquisition | 3,449,000 | |
Net cash provided by (used in) investing activities | 40,061,000 | (80,954,000) |
Cash flows from financing activities: | ||
Proceeds from sales of common stock, net of offering cost | 138,954,000 | |
Proceeds from issuance of common stock pursuant to option exercises | 349,000 | 131,000 |
Net cash provided by financing activities | 349,000 | 139,085,000 |
Effect of foreign currency exchange rate on cash and cash equivalents | (79,000) | (6,000) |
Net increase in cash and cash equivalents | 19,954,000 | 41,133,000 |
Cash and cash equivalents at beginning of period | 221,348,000 | 159,404,000 |
Cash and cash equivalents at end of period | 241,302,000 | 200,537,000 |
Supplemental schedule of noncash investing and financing information | ||
Issuance of common stock and exchange of stock options for business acquisition | 64,845,000 | |
Fixed assets in accounts payable and accrued expenses and other current liabilities | $ 153,000 | $ 875,000 |
Organization and Basis of Prese
Organization and Basis of Presentation | 6 Months Ended |
Jun. 30, 2016 | |
Accounting Policies [Abstract] | |
Organization and Basis of Presentation | 1. Organization and Basis of Presentation Adverum Biotechnologies, Inc. (the “Company”, “we” or “us”) was incorporated in Delaware on July 17, 2006 as Avalanche Biotechnologies, Inc. and changed its name to Adverum Biotechnologies, Inc. on May 11, 2016. The Company is headquartered in Menlo Park, California. The Company is a gene therapy company committed to discovering and developing novel medicines that can offer potentially life-changing therapeutic benefit to patients suffering from chronic or debilitating and rare diseases. Since the Company’s inception, it has devoted its efforts principally to performing research and development activities, including conducting preclinical studies, early clinical trials, filing patent applications, obtaining regulatory approvals, hiring personnel, and raising capital to support these activities. The Company has not generated any revenue from the sale of products since its inception. The Company has experienced net losses since its inception and has an accumulated deficit of $161.2 million as of June 30, 2016. The Company expects to incur losses and have negative net cash flows from operating activities as it expands its portfolio and engages in further research and development activities. The Company believes that it has sufficient funds to continue operations for at least the next 36 months. On May 11, 2016, the acquisition closing date, the Company completed its previously announced acquisition of all the outstanding shares of Annapurna Therapeutics SAS, a French simplified joint stock company ( Annapurna Agreement Pursuant to the terms of the Agreement, the Company issued 14,087,246 shares of the Company’s common stock, par value $0.0001 per share, for all of the issued and outstanding capital stock of Annapurna. All outstanding options and other rights to purchase capital stock of Annapurna were converted into the Company’s options for common stock. Refer to Note 3 for more details. Upon completion of the acquisition, the Company changed its name to “Adverum Biotechnologies, Inc.”. The Company’s shares of common stock listed on The NASDAQ Global Market, previously trading through the close of business on Wednesday, May 11, 2016 under the ticker symbol “AAVL,” commenced trading on The NASDAQ Global Market under the ticker symbol “ADVM” on Thursday, May 12, 2016. Follow-on Offerings —In January 2015, the Company completed a public offering of 2,369,375 shares of its common stock, which included 359,918 shares the Company issued pursuant to the underwriters’ exercise of their option to purchase additional shares. The Company received net proceeds of approximately $130.6 million, after underwriting discounts, commissions and offering expenses. In March 2015, (i) the Company received net proceeds of approximately $8.3 million, after discounts and other issuance costs, which resulted from the sale of 230,000 common shares, and (ii) the Company issued 230,000 common shares to a shareholder that exercised warrants prior to the initial public offering. The accompanying unaudited condensed consolidated financial statements have been prepared in accordance with U.S. generally accepted accounting principles (U.S. GAAP) and following the requirements of the Securities and Exchange Commission (SEC) for interim reporting. As permitted under those rules, certain footnotes or other financial information that are normally required by U.S. GAAP can be condensed or omitted. These condensed consolidated financial statements have been prepared on the same basis as the Company’s annual consolidated financial statements and, in the opinion of management, reflect all adjustments, consisting only of normal recurring adjustments, which are necessary for a fair statement of the Company’s consolidated financial information. The results of operations for the six month period ended June 30, 2016 are not necessarily indicative of the results to be expected for the full year or any other future period. The balance sheet as of December 31, 2015 has been derived from audited consolidated financial statements at that date but does not include all of the information required by U.S. GAAP for complete consolidated financial statements. The accompanying condensed consolidated financial statements and related financial information should be read in conjunction with the audited consolidated financial statements and the related notes thereto included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2015 filed with the SEC. |
Summary of Significant Accounti
Summary of Significant Accounting Policies | 6 Months Ended |
Jun. 30, 2016 | |
Accounting Policies [Abstract] | |
Summary of Significant Accounting Policies | 2. Summary of Significant Accounting Policies The accounting policies followed in the preparation of the interim condensed consolidated financial statements are consistent in all material respects with those presented in Note 2 to the consolidated financial statements included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2015. The Company added significant accounting policy below as a result of Annapurna acquisition. Valuation of Long‑Lived Assets and Purchased Intangible Assets The Company evaluates the carrying value of amortizable long‑lived assets, whenever events, changes in business circumstances or planned use of long‑lived assets indicate that their carrying amounts may not be fully recoverable or that their useful lives are no longer appropriate. If these facts and circumstances exist, the Company assesses for recovery by comparing the carrying values of long‑lived assets with their future undiscounted net cash flows. If the comparison indicates that impairment exists, long‑lived assets are written down to their respective fair value based on discounted cash flows. Significant management judgment is required in the forecast of future operating results that is used in the preparation of expected undiscounted cash flows. No impairment indicators were noted for the Company’s long-lived assets, fixed assets, in the periods presented. The Company also evaluates the carrying value of intangible assets (not subject to amortization) related to in‑process research and development (“IPR&D”) assets, which are considered to be indefinite‑lived until the completion or abandonment of the associated research and development efforts. Accordingly, amortization of the IPR&D assets will not occur until the product reaches commercialization. During the period the assets are considered indefinite‑lived, they will be tested for impairment on an annual basis, as well as between annual tests if the Company become aware of any events occurring or changes in circumstances that would indicate that the fair values of the IPR&D assets are less than their carrying amounts. If and when development is complete, which generally occurs when regulatory approval to market the product is obtained, the associated IPR&D assets would be deemed definite‑lived and would then be amortized based on their estimated useful lives at that point in time. If the related project is terminated or abandoned, the Company may have an impairment related to the IPR&D asset, calculated as the excess of their carrying value over fair value. The Company estimated fair value of IPR&D assets acquired in Annapurna transaction at the acquisition closing date, May 11, 2016. No impairment was recorded at June 30, 2016, as there were no changes in the fair value of IPR&D assets between May 11 and June 30, 2016. Recently Issued Accounting Pronouncements — In May 2014, the Financial Accounting Standards Board ( FASB) issued Accounting Standard Update (ASU) No. 2014-09, Revenue from Contracts with Customers (Topic 606), which supersedes the revenue recognition requirements in Accounting Standard Codification (ASC) 605 , Revenue Recognition . This ASU is based on the principle that revenue is recognized to depict the transfer of goods or services to customers in an amount that reflects the consideration to which the entity expects to be entitled in exchange for those goods or services. The ASU also requires 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. Companies may adopt ASU 2014-09 using a full retrospective approach or report the cumulative effect as of the date of adoption. In July 2015, the FASB voted to approve a one-year deferral of the effective date to December 15, 2017 for interim and annual reporting periods beginning after that date and permitted early adoption of the standard, but not before the original effective date of December 15, 2016 . The Company is evaluating the application of this ASU and method of adoption, but has not yet determined the potential effects it may have on the Company’s consolidated financial statements. In August 2014, the FASB issued ASU 2014-15, Presentation of Financial Statements—Going Concern (Subtopic 205-40): Disclosure of Uncertainties about an Entity’s Ability to Continue as a Going Concern In February 2016, the FASB issued ASU No. 2016-2, Leases In March 2016, the FASB issued ASU No. 2016-9, Compensation-Stock Compensation: Improvements to Employee Share-Based Payment Accounting . ASU 2016-9 simplifies several aspects of the accounting for share-based payment award transactions, including: (1) the income tax consequences, (2) classification of awards as either equity or liabilities, and (3) classification in the consolidated statement of cash flows. The new standard is effective for fiscal years and interim periods within those fiscal years beginning after December 15, 2016, with early adoption permitted. The Company has not yet determined the method of adoption and the potential effect the new standard will have on the Company’s consolidated financial statements. The Company has reviewed other recent accounting pronouncements and concluded they are either not applicable to the business or no material effect is expected on the consolidated financial statements as a result of future adoption. |
Acquisition of Annapurna
Acquisition of Annapurna | 6 Months Ended |
Jun. 30, 2016 | |
Business Combinations [Abstract] | |
Acquisition of Annapurna | 3. Acquisition of Annapurna (a) Purchase Price Allocation On May 11, 2016, the Company completed the acquisition of all outstanding equity interests of Annapurna. Annapurna is a privately held French limited liability company and has two wholly-owned subsidiaries, Annapurna, Inc. in the U.S. and Annapurna Therapeutics Limited in Ireland. Annapurna is a biopharmaceutical company focused on discovering and developing new gene therapy products for people living with severe diseases. The primary reasons for the acquisition were to diversify the technology platforms within the Company’s research and development portfolio and to apply the Company’s resources and expertise in gene vectors development to advance Annapurna programs through development and clinical trials. Annapurna’s results of operations and fair value of assets acquired and liabilities assumed are included in the Company’s condensed consolidated financial statements from the date of acquisition. The purchase price consideration was estimated to be $64.8 million, which was based on the Company’s common stock closing price on NASDAQ on the acquisition closing date of $4.14 per share. A total of 14,087,246 shares of the Company’s common stock were issued to shareholders of Annapurna in exchange for all common and preferred stock outstanding at the closing date. Annapurna stockholders did not receive any fractional shares of the Company’s common stock in connection with the acquisition. Instead of receiving any fractional shares, each Annapurna stockholders were paid an amount in cash (without interest) equal to such fraction amount multiplied by the average 10 business days sale price of the Company’s common stock on NASDAQ from the acquisition date. Annapurna Series O preferred shares issued to founders were canceled prior to the acquisition date and were not included in the purchase price consideration. Vesting of certain of Annapurna’s options and unvested common stock shares was accelerated at the closing date. The fair value of awards related to accelerated vesting of options and shares of $0.9 million was excluded from the purchase price consideration and included in the Company’s operating expenses post acquisition. A portion of the purchase price has been attributed to the exchange of Annapurna’s options and other rights to purchase capital stock outstanding at the acquisition closing date for corresponding common stock options of the Company at an exchange ratio of 9.54655. The Company reserved 3,673,940 shares for the future exercise of the Company’s stock options. The total fair value of assumed Annapurna’s stock options and stock-based awards was estimated at $14.7 million on the acquisition date, using the Black-Scholes pricing model, assuming no dividends, expected volatilities of 80% and 89%, risk-free interest rates of 1.4% and 1.1%, and expected lives of six and ten years for employees and non-employees awards, respectively. Of the total fair value, $7.4 million has been attributed as pre-combination service and included as part of the total purchase price consideration. The post-combination attribution of $7.2 million will be recognized as compensation expense over the remaining requisite service period. The Company has included $1.1 million in stock-based compensation expense related to the vesting of exchanged stock options and day-one post combination compensation expenses related to accelerated vesting of options and shares in its condensed consolidated statement of operations during the second quarter 2016. Total purchase price consideration was as follows (in thousands): Fair value of common shares issued $ 58,321 Fair value of the Company's common share options exchanged for Annapurna stock options and other awards attributable to pre-combination services 7,422 Less: value of common stock and options accelerated vesting at the closing date (898 ) Total purchase price consideration $ 64,845 The transaction has been accounted for using the acquisition method based on ASC 805, Business Combinations, Valuing certain components of the acquisition, primarily intangible assets acquired, deferred taxes, uncertain tax positions and accrued liabilities required us to make significant estimates that may be adjusted in the future; consequently, the fair value of identifiable assets acquired and liabilities assumed are considered preliminary. Final determination of these estimates could result in an adjustment to the preliminary purchase price allocation, with an offsetting adjustment to goodwill. Preliminary allocation of total purchase price consideration is as follows (in thousands): Cash $ 3,449 Prepaid expenses and other assets 865 Property and equipment 185 Acquired intangible assets 16,650 Goodwill 49,120 Accounts payable (1,118 ) Accrued liabilities (1,848 ) Other noncurrent liabilities (377 ) Deferred tax liabilities (2,081 ) Total purchase price allocation $ 64,845 The identifiable intangible assets acquired consist of in-process research and development (IPR&D) assets related to products in development, as summarized in the table below (in thousands): IPR&D - Alpha-1 antitrypsin deficiency $ 12,150 IPR&D - Hereditary angioedema 4,500 Total acquired intangible assets $ 16,650 The fair value of each IPR&D asset is estimated using the income approach and calculated using cash flow projections adjusted for inherent risks regarding regulatory approval, promotion, and distribution, discounted at a rate of approximately 11.0%. The Company acquired two additional intangible assets relating to the Friedreich’s Ataxia (FA) and severe allergy programs but the fair value of each of these assets was determined to be nominal and is not included in the total acquired intangible assets. All IPR&D intangible assets acquired are currently classified as indefinite-lived and are not currently being amortized. IPR&D asset becomes definite-lived upon the completion or abandonment of the associated research and development efforts, and will be amortized from that time over an estimated useful life based on respective patent terms. The fair value of each IPR&D asset will continue to be evaluated for impairment on an annual basis or more often if the Company identifies impairment indicators that would require earlier testing. Based on the preliminary fair values above, an amount of $49.1 million has been allocated to goodwill, which represents the excess of the purchase price over the fair values assigned to the net assets acquired. The full amount of the preliminary value of goodwill has been assigned to the entire Company, since management has determined that the Company has only one reporting unit. The goodwill is not deductible for tax purposes. The amount of net loss of Annapurna included in the consolidated statements of operations from the acquisition date, through the period ended June 30, 2016 was $1.2 million. Annapurna did not generate any revenues prior or post acquisition. The following table presents the unaudited pro forma results for the three and six months ended June 30, 2016 and 2015. The pro forma financial information combines the results of operations of Adverum and Annapurna as though the businesses had been combined as of the beginning of fiscal 2015. The pro forma financial information is presented for informational purposes only, and is not indicative of the results of operations that would have been achieved in the current or any future periods. Three Months Ended June 30, Six Months Ended June 30, 2016 2015 2016 2015 Pro forma information Collaboration revenue $ 307 $ 203 $ 572 $ 406 Net loss $ (62,572 ) $ (10,533 ) $ (80,846 ) $ (21,956 ) Basic and diluted loss per share $ (1.52 ) $ (0.27 ) $ (1.97 ) $ (0.56 ) Pro-forma adjustments included the following: · Actual acquisition-related transaction costs of $0.6 million and $2.5 million for three and six months ended June 2016 were excluded from the 2016 pro forma results above. As these expenses were incurred prior to the closing of the acquisition, they were not included in the 2015 pro forma results. · Stock-based compensation expense related to accelerated vesting associated with the acquisition of $0.9 million was excluded from the 2016 pro forma results and was recorded in the six months ended June 30, 2015. · Stock-based compensation expense related to options granted to executives upon the acquisition closing of $0.1 million and $0.2 million was included in the 2016 and 2015 pro forma results above. · Interest expense related to convertible notes and changes in fair value of preferred stock warrants of $0.5 million for the three and six month periods ended June 30, 2015, $0.5 million for the three month period ended June 30, 2016, and $1.0 million for the six month period ended June 30, 2016, were excluded form 2015 and 2016 pro-forma results above, as the convertible notes and warrants were settled prior to the acquisition closing. · Bonuses paid in connection with closing of the acquisition in May 2016 of $0.4 million were excluded from the 2016 pro forma results and were recorded in the six months ended June 30, 2015. The unaudited condensed pro forma information does not include any anticipated synergies that may be achievable subsequent to the date of acquisition. b) Impairment evaluation for intangible assets and goodwill As the Company recorded goodwill and IPR&D intangible assets upon the acquisition of Annapurna, the Company is required to test goodwill and indefinite lived intangible assets for impairment on an annual basis or more frequently if indicators of impairment exist. The Company operates as one reporting unit and goodwill was recorded to this reporting unit. During the second quarter the Company noted a continuing decrease in its stock price that resulted in the market capitalization being less than the carrying value of the Company’s net assets as of June 30, 2016. As the operating losses are expected to increase significantly in the following years due to continuing pre-clinical and expected clinical trials, the Company concluded that it is more likely than not that the fair value of the Company’s one reporting unit is less than its carrying value and as a result performed a step one goodwill impairment analysis. In performing the step one analysis, the Company determined the fair value of the reporting unit using a market-based approach. The Company multiplied the stock price of $3.16 on June 30, 2016 by the 41.3 million common shares outstanding and applied a control premium to estimate the common equity value on a controlling basis. As the fair value was less than the carrying value of the Company’s net assets, the Company proceeded to step two of the impairment analysis. The second step of the analysis includes allocating the calculated fair value (determined in the step one analysis) of the reporting unit to its assets and liabilities to determine an implied fair value of goodwill. The implied fair value of goodwill was determined in the same manner as the amount of goodwill recognized in an acquisition. That is, the estimated fair value of the reporting unit was allocated to all of the assets and liabilities as if the Company had been acquired and the estimated fair value was the purchase price paid. As part of this assessment the Company considered preliminary valuation of Annapurna net assets acquired, excluding goodwill, as their fair value from May 11, 2016, the acquisition closing date, to June 30, 2016 did not change. The Company also noted that the fair value of current assets and liabilities approximates their carrying value due to their short-term nature, the Company’s cash and cash equivalent balance is higher than the fair value estimated in the step one analysis, and the fair value of fixed assets approximates their recorded value as most of the Company’s fixed assets are acquired in the last couple of years. Based on this analysis, the implied fair value of the goodwill was zero. Accordingly, the Company recorded a goodwill impairment charge of $49.1 million in the condensed consolidated statements of operations and comprehensive loss for the period ended June 30, 2016. As the Annapurna purchase price allocation is preliminary and the amount of goodwill might change during the measurement period, the recorded impairment charge reflects the Company’s best estimate as of June 30, 2016. We did not impair recorded IPR&D intangible assets, as their carrying value at June 30, 2016 approximated their fair value. We noted no significant changes in IPR&D intangible assets fair values from May 11, 2016, the acquisition closing date, to June 30, 2016. |
Cash Equivalents and Marketable
Cash Equivalents and Marketable Securities | 6 Months Ended |
Jun. 30, 2016 | |
Cash And Cash Equivalents [Abstract] | |
Cash Equivalents and Marketable Securities | 4. Cash Equivalents and Marketable Securities The Company did not hold any marketable securities as of June 30, 2016, all investments were held in money market funds and are treated as cash equivalents. . The following is a summary of the cash equivalents and marketable securities as of December 31, 2015: December 31, 2015 Amortized Cost Basis Unrealized Gains Unrealized Loses Estimated Fair Value Money Market Funds $ 208,588 $ — $ — $ 208,588 Certificates of Deposit 1,680 — — 1,680 U.S. Treasury Securities 15,046 — (4 ) 15,042 U.S. Government Agency Securities 21,012 — (2 ) 21,010 246,326 — (6 ) 246,320 Less: Cash Equivalents (208,588 ) — — (208,588 ) Total Marketable Securities $ 37,738 $ — $ (6 ) $ 37,732 As of December 31, 2015, the contractual maturities of the Company’s marketable securities were less than one year. The Company has not sold any securities prior to their maturities and so does not consider any losses on these investment to be other-than-temporarily impaired. There were no sales of available-for-sale securities in any of the periods presented. |
Fair Value Measurements and Fai
Fair Value Measurements and Fair Value of Financial Instruments | 6 Months Ended |
Jun. 30, 2016 | |
Fair Value Disclosures [Abstract] | |
Fair Value Measurements and Fair Value of Financial Instruments | 5. Fair Value Measurements and Fair Value of Financial Instruments The authoritative guidance on fair value measurements establishes a three-tier fair value hierarchy for disclosure of fair value measurements as follows: Level 1 : Quoted prices in active markets for identical assets or liabilities. Level 2 : Observable inputs other than Level 1 prices, such as quoted prices for similar assets or liabilities, quoted prices in markets that are not active, or other inputs that are observable or can be corroborated by observable market data for substantially the full term of the assets or liabilities. Level 3 : Unobservable inputs that are supported by little or no market activity and that are significant to the fair value of the assets or liabilities. Assets and liabilities measured at fair value are classified in their entirety based on the lowest level of input that is significant to the fair value measurement. The Company’s assessment of the significance of a particular input to the fair value measurement in its entirety requires management to make judgments and consider factors specific to the asset or liability. The fair value of Level 1 securities are determined using quoted prices in active markets for identical assets. Level 1 securities consist of highly liquid money market funds. Financial assets and liabilities are considered Level 2 when their fair values are determined using inputs that are observable in the market or can be derived principally from or corroborated by observable market data such as pricing for similar securities, recently executed transactions, cash flow models with yield curves, and benchmark securities. In addition, Level 2 financial instruments are valued using comparisons to like-kind financial instruments and models that use readily observable market data as their basis. U.S. Treasury securities, U.S. government agency securities and certificate of deposit are valued primarily using market prices of comparable securities, bid/ask quotes, interest rate yields and prepayment spreads and are included in Level 2. There were no transfers within the hierarchy during the six months ended June 30, 2016 and the year ended December 31, 2015. As of June 30, 2016 and December 31, 2015, the Company had no Level 3 assets or liabilities. The following table summarizes, for assets recorded at fair value on a recurring basis, the respective fair value and the classification by level of input within the fair value hierarchy as described above (in thousands): Quoted Prices Significant Other Significant Total In Observable Inputs Unobservable Inputs Carrying (Level 1) (Level 2) (Level 3) June 30, 2016 Assets: Money Market Funds - cash equivalent $ 236,821 $ 236,821 $ — $ — Total Cash Equivalents $ 236,821 $ 236,821 $ — $ — December 31, 2015 Assets: Money Market Funds - cash equivalent $ 208,588 $ 208,588 $ — $ — Certificates of Deposit 1,680 — 1,680 — U.S. Treasury Securities 15,042 — 15,042 — U.S. Government Agency Securities 21,010 — 21,010 — Total Cash Equivalents and Marketable Securities $ 246,320 $ 208,588 $ 37,732 $ — Non-financial and financial assets such as intangible assets, property, plant, and equipment are evaluated for impairment and adjusted to their fair value using Level 3 inputs, only when impairment is recognized. Fair values are considered Level 3 when management makes significant assumptions in developing a discounted cash flow model based upon a number of considerations including projections of revenues, earnings and a discount rate. In addition, in evaluating the fair value of goodwill impairment, further corroboration is obtained using our market capitalization. |
Significant Agreements
Significant Agreements | 6 Months Ended |
Jun. 30, 2016 | |
Research And Development [Abstract] | |
Significant Agreements | 6. Significant Agreements Regeneron In May 2014, the Company entered into a research collaboration and license agreement with Regeneron to discover, develop and commercialize novel gene therapy products for the treatment of ophthalmologic diseases. The collaboration covers up to eight distinct therapeutic targets (collaboration targets). The Company and Regeneron will collaborate during the initial research period of three years that can be extended by Regeneron for up to an additional five years. During the research period, Regeneron has the option to obtain an exclusive worldwide license for a collaboration target’s further development by giving written notice to the Company and paying $2.0 million per target. If Regeneron exercises its option, it will be responsible for all further development and commercialization of the target. The Company is then eligible to receive contingent payments of up to $80.0 million upon achievement of certain development and regulatory milestones for product candidates directed toward each collaboration target, for a combined total of up to $640.0 million in potential milestone payments for product candidates directed toward all eight collaboration targets, plus a royalty in the low- to mid-single-digits on worldwide net sales of collaboration products. For any two collaboration targets, the Company has an option to share up to 35% of the worldwide product candidate development costs and profits. If the Company exercises this option, the Company will not be eligible for milestone and royalty payments discussed above but rather the Company will share development costs and profits with Regeneron. The agreement will expire with respect to each collaboration target upon the earlier of the (a) expiration of the research term if the option right has not been triggered by the end of the research term or (b) expiration of the option right if the option right has not been exercised by Regeneron. If the option right has been exercised, the agreement in connection with each collaboration target will expire upon expiration of all payment obligations by Regeneron. In addition, the agreement, or Regeneron’s rights to any target development under the agreement, may terminate early under the following situations: · Regeneron may terminate the agreement for convenience at any time on a target by target basis or in totality upon a 30-day notice. · Each party can terminate the agreement if another party commits a material breach or material default in performance of its obligations and such breach or default is not cured within 60 days. · The agreement is automatically terminated upon initiation of any bankruptcy proceedings, reorganization or dissolution of either party. · The Company can terminate the agreement upon 30-day notice if Regeneron challenges the validity, scope or enforceability of any Company patent. University of California In May 2010, the Company entered into a license agreement, as amended, with the Regents of the University of California (Regents) for exclusive rights in the U.S. to certain patents owned by the Regents. Under the terms of the agreement, the Company paid an upfront license fee of $100,000 and agreed to reimburse the Regents for patent-related expenses. The Company is obligated to pay the Regents royalties on net sales, if any, as well as an annual maintenance fee of $50,000 beginning in the calendar year after the first commercial sale of a licensed product and milestone payments related to the achievement of certain clinical and regulatory goals totaling up to $900,000 for the first indication and $500,000 for each additional indication for up to two additional indications. Through June 30, 2016, none of these goals had been achieved, and no milestones were payable. Cornell University In August 2014, as amended in December 2015, Annapurna entered into a master service agreement with Cornell University for assistance in regulatory affairs, overall project management and parameter development. Per the amended agreement, Annapurna will pay Cornell $13.3 million ratably over 4 years for these services, as services will be performed. In December 2015, Annapurna Therapeutics Limited entered into three licensing agreements with Cornell University, pursuant to which Annapurna will advance its ANN-001, ANN-002 and ANN-004 programs, which were each based on gene-therapy programs initiated at the Department of Genetic Medicine at Weill Cornell. A1AT Deficiency License Agreement : Under this agreement, Annapurna Therapeutics Limited holds an exclusive license to certain technology related to alpha-1 antitrypsin (“A1AT”) deficiency and rights to an IND application to initiate clinical studies of gene therapy for A1AT. HAE License Agreement : Under this agreement, Annapurna Therapeutics Limited holds an exclusive license to certain technology related to hereditary angioedema (“HAE”) and a non-exclusive license to certain other intellectual property related to the HAE program. Allergy License Agreement : Under this agreement, Annapurna Therapeutics Limited holds an exclusive license to certain patents related to allergens and a non-exclusive license to certain other technology related to allergens. The Company may terminate any of these license agreements for convenience upon ninety days written notice. Across these three license agreements, Cornell University is entitled to receive aggregate annual maintenance fees ranging from $30,000 to $300,000 per year, up to $16.0 million in aggregate milestone payments and royalties on sales in the low single-digits, subject to adjustments and minimum thresholds. In addition, under a master services agreement with Cornell University, Annapurna will utilize the university to scale production of gene therapies by manufacturing processes that the institution has already used to produce Good Manufacturing Practice (GMP) material for other gene-therapy trials. The Company accrued $0.5 million as of June 30, 2016 and recorded expenses of $0.5 million (post Annapurna’s acquisition) for the period from May 11, 2016 through June 30, 2016, related to Cornell agreements. No milestone payments were probable to achieve and none were recorded as of June 30, 2016. Dr. Crystal, Chairman of Genetic Medicine, the Bruce Webster Professor of Internal Medicine and a Professor of Genetic Medicine and of Medicine at Weill Cornell, served as a consultant to Annapurna since inception and will continue to provide services to the Company for the annual compensation of $0.3 million. Dr. Crystal also owns common shares of the Company and he does not have significant influence on the Company’s operations. REGENXBIO A1AT Deficiency/Allergy License Agreement : In October 2015, Annapurna Therapeutics Limited entered into an exclusive worldwide license to certain intellectual property in order to make, have made, use, import, sell and offer for sale certain licensed products for the treatment of A1AT deficiency. Additionally under this agreement, the Company has an option to be granted an exclusive worldwide license to certain intellectual property related to the treatment of severe allergies. Under this license agreement, REGENXBIO is eligible to receive annual maintenance fees, up to approximately $20.0 million in combined milestone payments and royalties in the mid-to-high single digits. Friedreich’s Ataxia License Agreement : In April 2014, Annapurna entered into an exclusive worldwide license to certain intellectual property related to the Friedreich’s Ataxia (“FA”) program to make, have made, use, import, sell and offer for sale licensed products using AAVrh10 for FA where the vector is administered by any route except directly to the central nervous system (FA Systemic). Under the terms of this license agreement, Annapurna also has an option to obtain a non-exclusive worldwide license to make, have made, use, import, sell and offer for sale licensed products using a single vector for each of FA where the vector is administered directly to the central nervous system and FA Systemic. Under this license agreement, REGENXBIO is eligible to receive annual maintenance fees, up to $13.85 million in combined milestone fees and royalties in the mid-to-high single digits. The Company accrued $46,000 as of June 30, 2016 and recorded expenses of $20,000 (post Annapurna’s acquisition) for the period May 11, 2016 through June 30, 2016, related to REGENXBIO agreements. No milestone payments were probable to achieve and none were recorded as of June 30, 2016. Inserm Transfert In July 2014, Annapurna entered into an agreement with Inserm Transfert whereby Annapurna holds an exclusive license to certain patents to develop, make, have made, use, import, offer for sale and sell or otherwise distribute products for the treatment of Friedreich’s ataxia and a non-exclusive license to certain other intellectual property related to the FA program. The Agreement was amended in October 2015 to increase the scope of the intellectual property under the licenses. Under this agreement, Inserm Transfert is entitled to receive certain de minimis license payments, certain development milestone payments of up to approximately €2 million in the aggregate and royalties on sales in the low single-digits, subject to adjustments. The Company did not have any accruals relating to Inserim as of June 30, 2016 and nor did the Company record any expenses (post Annapurna’s acquisition) for the period from May 11, 2016 through June 30, 2016, related to this agreement. No milestone payments were probable to achieve and none were recorded as of June 30, 2016. |
Property and Equipment, Net
Property and Equipment, Net | 6 Months Ended |
Jun. 30, 2016 | |
Property Plant And Equipment [Abstract] | |
Property and Equipment, Net | 7. Property and Equipment, Net Property and equipment, net consists of the following (in thousands): June 30, 2016 December Computer equipment and software $ 259 $ 234 Laboratory equipment 3,688 3,041 Furniture and fixtures 552 552 Leasehold improvements 939 351 Construction in progress 201 — Total property and equipment 5,639 4,178 Less accumulated depreciation and amortization (1,654 ) (991 ) Property and equipment, net $ 3,985 $ 3,187 Depreciation and amortization expense related to property and equipment for the three months ended June 30, 2016 and 2015 was $343,000 and $147,000, respectively. Depreciation and amortization expense related to property and equipment for the six months ended June 30, 2016 and 2015 was $663,000 and $265,000, respectively. |
Accrued Expenses and Other Curr
Accrued Expenses and Other Current Liabilities | 6 Months Ended |
Jun. 30, 2016 | |
Payables And Accruals [Abstract] | |
Accrued Expenses and Other Current Liabilities | 8. Accrued Expenses and Other Current Liabilities Accrued expenses and other current liabilities consist of the following (in thousands): June 30, 2016 December Employees’ compensation expenses $ 1,608 $ 2,047 Accrued professional services 608 1,177 Accrued preclinical costs $ 1,844 $ 642 Accrued clinical and process development costs — 101 Other 84 40 Total accrued expenses and other current liabilities $ 4,144 $ 4,007 |
Other Liabilities
Other Liabilities | 6 Months Ended |
Jun. 30, 2016 | |
Other Liabilities Disclosure [Abstract] | |
Other Liabilities | 9. Other liabilities Due to the innovative nature of Annapurna’s product candidate development programs, Annapurna has benefited from certain sources of financial assistance from Banque Publique d’Investissement (“BPI France”). BPI France provides financial assistance and support to emerging French enterprises to facilitate the development and commercialization of innovative technologies. The funds received by the Company are intended to finance its research and development efforts and the recruitment of specific personnel. The Company has received such funding in the form of conditional advances. In August 2015, BPI France granted Annapurna a €750,000 interest free conditional advance, of which €500,000 was drawn down as of December 31, 2015. The remaining €250,000 advance was not and is not expected to be drawn down on. Payments are scheduled in equal quarterly amounts of €25,000 from September 30, 2017 to June 30, 2022. This payment schedule will be modified if the Company will receive revenue from license or product sales before advances are paid in full. The Company calculated 7% imputed interest expense on these advances that recorded it as a discount at the issuance date. The discount is amortized as an interest expense over the life of advances. As of June 30, 2016 the carrying value of the conditional advance was $373,000 and the Company recorded $15,000 interest expense from the acquisition closing date to June 30, 2016. |
Commitments and Contingencies
Commitments and Contingencies | 6 Months Ended |
Jun. 30, 2016 | |
Commitments And Contingencies Disclosure [Abstract] | |
Commitments and Contingencies | 10. Commitments and Contingencies Collaborations and License Agreements In August 2014, as amended December 2015, Annapurna entered into a master service agreement with Cornell University for assistance in regulatory affairs, overall project management and parameter development. Per the amended agreement, Annapurna will pay Cornell $13.3 million ratably over 4 years for these services. The Company is a party to various agreements, principally relating to licensed technology that requires payment of annual maintenance fees and future payments relating to milestones or royalties on future sales of specified products. Refer to Note 6 for further details. The Company expenses the annual maintenance fees on a straight-line basis and accrues the aggregate balances until invoiced or paid. Through June 30, 2016, none of the goals had been achieved under the license agreements and no cash milestones were accrued or payable. Because the achievement of these milestones is not fixed and determinable, such commitments have not been included in the Company’s condensed consolidated balance sheets. Guarantees and Indemnifications In the normal course of business, the Company enters into contracts and agreements that contain a variety of representations and warranties and provide for indemnification for certain liabilities. The exposure under these agreements is unknown because it involves claims that may be made against the Company in the future but have not yet been made. To date, the Company has not paid any claims or been required to defend any action related to its indemnification obligations. However, the Company may record charges in the future as a result of these indemnification obligations. The Company also has indemnification obligations to its directors and executive officers for specified events or occurrences, subject to some limits, while they are serving at the Company’s request in such capacities. There have been no claims to date and the Company believes the fair value of these indemnification agreements is minimal. Accordingly, the Company has not recorded any liabilities for these agreements as of June 30, 2016. Legal Proceedings From time to time, the Company may become involved in litigation and other legal actions. The Company estimates the range of liability related to any pending litigation where the amount and range of loss can be estimated. The Company records its best estimate of a loss when the loss is considered probable. Where a liability is probable and there is a range of estimated loss with no best estimate in the range, the Company records a charge equal to at least the minimum estimated liability for a loss contingency when both of the following conditions are met: (i) information available prior to issuance of the financial statements indicates that it is probable that a liability had been incurred at the date of the financial statements and (ii) the range of loss can be reasonably estimated. In July 2015, three putative securities class action lawsuits were filed against the Company and certain of its officers in the United States District Court for the Northern District of California, each on behalf of a purported class of persons and entities who purchased or otherwise acquired its publicly traded securities between July 31, 2014 and June 15, 2015. The lawsuits assert claims under the Securities Exchange Act of 1934 (Exchange Act) and the Securities Act of 1933, as amended (Securities Act) and allege that the defendants made materially false and misleading statements and omitted allegedly material information related to, among other things, the Phase 2a clinical trial for AVA-101 and the prospects of AVA-101. The complaints seek unspecified damages, attorneys’ fees and other costs. An amended consolidated complaint was filed in February 2016, and the Company’s motion to dismiss that consolidated complaint is pending. In December 2015, a putative securities class action lawsuit was filed against us, our board of directors, underwriters of our January 13, 2015, follow-on public stock offering, and two of our institutional stockholders, in the Superior Court of the State of California for the County of San Mateo. The complaint alleges that, in connection with our follow-on stock offering, the defendants violated the Securities Act in essentially the same manner alleged by the consolidated federal action: by allegedly making materially false and misleading statements and by allegedly omitting material information related to the Phase 2a clinical trial for AVA-101 and the prospects of AVA-101. The complaint seeks unspecified compensatory and rescissory damages, attorneys’ fees and other costs. The plaintiff has dismissed the two institutional stockholder defendants, and the Company’s motion to stay or to dismiss this action is pending. The Company believes that the claims in the asserted actions are without merit and intend to defend the lawsuits vigorously. The Company expects to incur costs associated with defending the actions. While the Company has various insurance policies related to the risks associated with its business, including directors’ and officers’ liability insurance policies, there is no assurance that the Company will be successful in its defense of the actions, that its insurance coverage, which contains a self-insured retention, will be sufficient, or that its insurance carriers will cover all claims or litigation costs. Due to the inherent uncertainties of litigation, the Company cannot reasonably predict at this time the timing or outcomes of these matters or estimate the amount of losses, or range of losses, if any, or their effect, if any, on its condensed consolidated financial statements. |
Stock Option Plans
Stock Option Plans | 6 Months Ended |
Jun. 30, 2016 | |
Disclosure Of Compensation Related Costs Sharebased Payments [Abstract] | |
Stock Option Plans | 11. Stock Option Plans The Company’s 2014 Equity Incentive Award Plan (2014 Plan) permits the issuance of stock options (options), restricted stock units (RSUs) and other types of awards to employees, directors, and consultants. As of June 30, 2016, a total of 13,162,656 shares of common stock were authorized for issuance and 2,241,693 shares were available for future grants under the 2014 Plan. In July 2014, the Company’s board of directors and its stockholders approved the establishment of the 2014 Employee Stock Purchase Plan (2014 ESPP). During the six months ended June 30, 2016 32,425 shares were issued under the 2014 ESPP and no shares were issued in the same period for 2015. A total of 675,383 shares of common stock have been reserved for issuance under the 2014 ESPP and 623,381 were available for issuance under the 2014 ESPP as of June 30, 2016. The following table summarizes option activity under our stock plans and related information: WEIGHTED- NUMBER OF WEIGHTED- AVERAGE AGGREGATE SHARE AVERAGE EXERCISE CONTRACTUAL LIFE INTRINSIC VALUE (a) (IN PRICE (IN YEARS) (IN THOUSANDS) Balance at January 1, 2016 5,494 $ 8.75 Options granted 4,975 $ 1.25 Options exercised (1,233 ) $ 0.40 Options cancelled (511 ) $ 23.83 Balance at June 30, 2016 8,725 $ 4.77 8.9 15,473 Vested and expected to vest as of June 30, 2016 8,622 $ 4.72 8.9 15,471 Exercisable as of June 30, 2016 3,760 $ 3.62 8.2 8,997 (a) The aggregate intrinsic value is calculated as the difference between the option exercise price and the closing price of common stock of $3.16 per share as of June 30, 2016. Options granted number includes the Company’s stock options for 3,673,940 common stock shares issued in exchange for Annapurna stock options at $0.21 exercise price per share. The weighted-average fair values of options granted and exchanged during the six months ended June 30, 2016 and 2015 were $1.62 and $27.90, respectively. The total intrinsic value of options exercised during the six months ended June 30, 2016 and 2015 were $6.3 million and $10.6 million, respectively. The Company has recorded aggregate stock-based compensation expense related to the issuance of stock option awards to employees and nonemployees in the condensed consolidated statement of operations and comprehensive loss as follows (in thousands): Three Months Ended Six Months Ended June 30, June 30, 2016 2015 2016 2015 Research and development $ 1,912 $ (445 ) $ 4,140 $ (161 ) General and administrative 914 1,004 2,770 1,493 Total share-based compensation $ 2,826 $ 559 $ 6,910 $ 1,332 Stock-based compensation expense included additional charges of $0.3 million and $1.1 million, recorded in general and administrative expense, and zero and $1.4 million, recorded in research and development expense, related to stock modifications in connection with separation agreements three Company’s executives for the three-months and six-months ended June 30, 2016, respectively. Restricted Stock Units Restricted stock units, or RSUs, are share awards that entitle the holder to receive freely tradable shares of our common stock upon vesting. The fair value of RSUs is based upon the closing sales price of our common stock on the grant date. During the six months ended June 30, 2016 and 2015, the Company granted a total of 1,066,283 and 97,000 RSUs, respectively. RSUs granted to employees generally vest over a two-to-four year period. The following table summarizes the RSUs activity under our stock plans and related information: Number of Weighted-Average Shares Grant-Date (in thousands) Fair Value (in dollars) Outstanding at December 31, 2015 632 $ 13.07 Granted 1,066 $ 4.63 Vested and released (165 ) $ 11.60 Forfeited (342 ) $ 7.85 Outstanding at June 30, 2016 1,191 $ 6.50 The total fair value of RSUs that vested for the six months ended June 30, 2016 $2.2 million and no shares were vested for the six months ended June 30, 2015. As of June 30, 2016, there was $5.3 million of unrecognized compensation cost related to unvested RSUs that we expect to recognize over a weighted-average period of 2.8 years. Stock Options Granted to Employees The fair value of each option issued to employees was estimated at the date of grant using the Black-Scholes valuation model with the following weighted-average assumptions: Three Months Ended Six Months Ended June 30, June 30, 2016 2015 2016 2015 Option grants: Expected volatility 81 % 76 % 81 % 77 % Expected term (in years) 5.9 6.0 5.9 6.1 Expected dividend yield — — — — Risk-free interest rate 1.3 % 1.6 % 1.3 % 1.6 % As of June 30, 2016, there was $18.4 million of unrecognized stock-based compensation expense related to employees’ awards that is expected to be recognized over a weighted-average period of 3.0 years. Stock Options Granted to Non-Employees Stock-based compensation related to stock options granted to non-employees is measured and recognized as the stock options are earned. The Company believes that the estimated fair value of the stock options is more readily measurable than the fair value of the services rendered. The following weighted-average assumptions were used in estimating non-employees’ stock-based compensation expenses: Three Months Ended Six Months Ended June 30, June 30, 2016 2015 2016 2015 Option grants: Expected volatility 84 % 80 % 83 % 80 % Expected term (in years) 8.0 8.0 7.5 7.8 Expected dividend yield — — — — Risk-free interest rate 1.6 % 2.0 % 1.6 % 1.9 % As of June 30, 2016, there was $2.5 million of unrecognized stock-based compensation expense related to non-employees’ awards that is expected to be recognized over a weighted-average period of 1.3 years. |
401(k) Savings Plan
401(k) Savings Plan | 6 Months Ended |
Jun. 30, 2016 | |
Postemployment Benefits [Abstract] | |
401(k) Savings Plan | 12. 401(k) Savings Plan The Company established a defined-contribution savings plan under Section 401(k) of the Code (the 401(k) Plan). The 401(k) Plan covers all employees who meet defined minimum age and service requirements, and allows participants to defer a portion of their annual compensation on a pretax basis. For the three months ended June 30, 2016 and 2015, the Company contributed $0.1 million and $0 to the 401(k) Plan, respectively. For the six months ended June 30, 2016 and 2015, the Company contributed $0.2 million and $0.1 million to the 401(k) Plan, respectively |
Net Loss Per Share
Net Loss Per Share | 6 Months Ended |
Jun. 30, 2016 | |
Earnings Per Share [Abstract] | |
Net Loss Per Share | 13. Net Loss Per Share Basic net loss per share is calculated by dividing the net loss by the weighted-average number of shares of common stock outstanding for the period. Diluted net loss per share attributable is computed by giving effect to all potential dilutive common stock equivalents outstanding for the period. Diluted net loss per share is the same as basic net loss per share for all periods presented, since the effects of potentially dilutive securities are antidilutive. We have excluded stock options and RSUs to purchase approximately 9.9 million and 5.2 million shares of our common stock that were outstanding as of June 30, 2016 and 2015, respectively, in the computation of diluted net loss per share attributable to common stockholders because their effect was antidilutive. |
Subsequent Events
Subsequent Events | 6 Months Ended |
Jun. 30, 2016 | |
Subsequent Events [Abstract] | |
Subsequent Events | 14. Subsequent Events In August 2016, the Company entered into a collaboration, option and license agreement with Editas Medicine, Inc. (“ Editas Editas may exercise the option, with respect to a designated initial indication, until the first anniversary of the effective date of the agreement. With respect to the four other Indications, Editas may exercise the option until the third anniversary of the effective date, provided that the option will expire on the second anniversary of the effective date if Editas has not exercised the option with respect to the initial indication or any other indication by such date. Upon each exercise of the option, Editas will pay the Company a $1.0 million fee. If Editas elects to develop a product using certain of the Company’s proprietary vectors, the Company will be eligible to receive additional payments relating to development and commercialization milestone payments for such product, and tiered royalties on net sales of such product, subject to certain adjustments. |
Summary of Significant Accoun20
Summary of Significant Accounting Policies (Policies) | 6 Months Ended |
Jun. 30, 2016 | |
Accounting Policies [Abstract] | |
Valuation of Long-Lived Assets and Purchased Intangible Assets | Valuation of Long‑Lived Assets and Purchased Intangible Assets The Company evaluates the carrying value of amortizable long‑lived assets, whenever events, changes in business circumstances or planned use of long‑lived assets indicate that their carrying amounts may not be fully recoverable or that their useful lives are no longer appropriate. If these facts and circumstances exist, the Company assesses for recovery by comparing the carrying values of long‑lived assets with their future undiscounted net cash flows. If the comparison indicates that impairment exists, long‑lived assets are written down to their respective fair value based on discounted cash flows. Significant management judgment is required in the forecast of future operating results that is used in the preparation of expected undiscounted cash flows. No impairment indicators were noted for the Company’s long-lived assets, fixed assets, in the periods presented. The Company also evaluates the carrying value of intangible assets (not subject to amortization) related to in‑process research and development (“IPR&D”) assets, which are considered to be indefinite‑lived until the completion or abandonment of the associated research and development efforts. Accordingly, amortization of the IPR&D assets will not occur until the product reaches commercialization. During the period the assets are considered indefinite‑lived, they will be tested for impairment on an annual basis, as well as between annual tests if the Company become aware of any events occurring or changes in circumstances that would indicate that the fair values of the IPR&D assets are less than their carrying amounts. If and when development is complete, which generally occurs when regulatory approval to market the product is obtained, the associated IPR&D assets would be deemed definite‑lived and would then be amortized based on their estimated useful lives at that point in time. If the related project is terminated or abandoned, the Company may have an impairment related to the IPR&D asset, calculated as the excess of their carrying value over fair value. The Company estimated fair value of IPR&D assets acquired in Annapurna transaction at the acquisition closing date, May 11, 2016. No impairment was recorded at June 30, 2016, as there were no changes in the fair value of IPR&D assets between May 11 and June 30, 2016. |
Recently Issued Accounting Pronouncements | Recently Issued Accounting Pronouncements — In May 2014, the Financial Accounting Standards Board ( FASB) issued Accounting Standard Update (ASU) No. 2014-09, Revenue from Contracts with Customers (Topic 606), which supersedes the revenue recognition requirements in Accounting Standard Codification (ASC) 605 , Revenue Recognition . This ASU is based on the principle that revenue is recognized to depict the transfer of goods or services to customers in an amount that reflects the consideration to which the entity expects to be entitled in exchange for those goods or services. The ASU also requires 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. Companies may adopt ASU 2014-09 using a full retrospective approach or report the cumulative effect as of the date of adoption. In July 2015, the FASB voted to approve a one-year deferral of the effective date to December 15, 2017 for interim and annual reporting periods beginning after that date and permitted early adoption of the standard, but not before the original effective date of December 15, 2016 . The Company is evaluating the application of this ASU and method of adoption, but has not yet determined the potential effects it may have on the Company’s consolidated financial statements. In August 2014, the FASB issued ASU 2014-15, Presentation of Financial Statements—Going Concern (Subtopic 205-40): Disclosure of Uncertainties about an Entity’s Ability to Continue as a Going Concern In February 2016, the FASB issued ASU No. 2016-2, Leases In March 2016, the FASB issued ASU No. 2016-9, Compensation-Stock Compensation: Improvements to Employee Share-Based Payment Accounting . ASU 2016-9 simplifies several aspects of the accounting for share-based payment award transactions, including: (1) the income tax consequences, (2) classification of awards as either equity or liabilities, and (3) classification in the consolidated statement of cash flows. The new standard is effective for fiscal years and interim periods within those fiscal years beginning after December 15, 2016, with early adoption permitted. The Company has not yet determined the method of adoption and the potential effect the new standard will have on the Company’s consolidated financial statements. The Company has reviewed other recent accounting pronouncements and concluded they are either not applicable to the business or no material effect is expected on the consolidated financial statements as a result of future adoption. |
Acquisition of Annapurna (Table
Acquisition of Annapurna (Tables) | 6 Months Ended |
Jun. 30, 2016 | |
Business Combinations [Abstract] | |
Summary of Total Purchase Price Consideration | Total purchase price consideration was as follows (in thousands): Fair value of common shares issued $ 58,321 Fair value of the Company's common share options exchanged for Annapurna stock options and other awards attributable to pre-combination services 7,422 Less: value of common stock and options accelerated vesting at the closing date (898 ) Total purchase price consideration $ 64,845 |
Components of Preliminary Allocation of Total Purchase Price Consideration | Preliminary allocation of total purchase price consideration is as follows (in thousands): Cash $ 3,449 Prepaid expenses and other assets 865 Property and equipment 185 Acquired intangible assets 16,650 Goodwill 49,120 Accounts payable (1,118 ) Accrued liabilities (1,848 ) Other noncurrent liabilities (377 ) Deferred tax liabilities (2,081 ) Total purchase price allocation $ 64,845 |
Summary of Identifiable Intangible Assets Acquired | The identifiable intangible assets acquired consist of in-process research and development (IPR&D) assets related to products in development, as summarized in the table below (in thousands): IPR&D - Alpha-1 antitrypsin deficiency $ 12,150 IPR&D - Hereditary angioedema 4,500 Total acquired intangible assets $ 16,650 |
Schedule of Pro Forma Financial Information | The following table presents the unaudited pro forma results for the three and six months ended June 30, 2016 and 2015. The pro forma financial information combines the results of operations of Adverum and Annapurna as though the businesses had been combined as of the beginning of fiscal 2015. The pro forma financial information is presented for informational purposes only, and is not indicative of the results of operations that would have been achieved in the current or any future periods. Three Months Ended June 30, Six Months Ended June 30, 2016 2015 2016 2015 Pro forma information Collaboration revenue $ 307 $ 203 $ 572 $ 406 Net loss $ (62,572 ) $ (10,533 ) $ (80,846 ) $ (21,956 ) Basic and diluted loss per share $ (1.52 ) $ (0.27 ) $ (1.97 ) $ (0.56 ) |
Cash Equivalents and Marketab22
Cash Equivalents and Marketable Securities (Tables) | 6 Months Ended |
Jun. 30, 2016 | |
Cash And Cash Equivalents [Abstract] | |
Summary of Cash Equivalents and Marketable Securities | The following is a summary of the cash equivalents and marketable securities as of December 31, 2015: December 31, 2015 Amortized Cost Basis Unrealized Gains Unrealized Loses Estimated Fair Value Money Market Funds $ 208,588 $ — $ — $ 208,588 Certificates of Deposit 1,680 — — 1,680 U.S. Treasury Securities 15,046 — (4 ) 15,042 U.S. Government Agency Securities 21,012 — (2 ) 21,010 246,326 — (6 ) 246,320 Less: Cash Equivalents (208,588 ) — — (208,588 ) Total Marketable Securities $ 37,738 $ — $ (6 ) $ 37,732 |
Fair Value Measurements and F23
Fair Value Measurements and Fair Value of Financial Instruments (Tables) | 6 Months Ended |
Jun. 30, 2016 | |
Fair Value Disclosures [Abstract] | |
Financial Assets Measured and Recognized at Fair Value on Recurring Basis | The following table summarizes, for assets recorded at fair value on a recurring basis, the respective fair value and the classification by level of input within the fair value hierarchy as described above (in thousands): Quoted Prices Significant Other Significant Total In Observable Inputs Unobservable Inputs Carrying (Level 1) (Level 2) (Level 3) June 30, 2016 Assets: Money Market Funds - cash equivalent $ 236,821 $ 236,821 $ — $ — Total Cash Equivalents $ 236,821 $ 236,821 $ — $ — December 31, 2015 Assets: Money Market Funds - cash equivalent $ 208,588 $ 208,588 $ — $ — Certificates of Deposit 1,680 — 1,680 — U.S. Treasury Securities 15,042 — 15,042 — U.S. Government Agency Securities 21,010 — 21,010 — Total Cash Equivalents and Marketable Securities $ 246,320 $ 208,588 $ 37,732 $ — |
Property and Equipment, Net (Ta
Property and Equipment, Net (Tables) | 6 Months Ended |
Jun. 30, 2016 | |
Property Plant And Equipment [Abstract] | |
Schedule of Property and Equipment, Net | Property and equipment, net consists of the following (in thousands): June 30, 2016 December Computer equipment and software $ 259 $ 234 Laboratory equipment 3,688 3,041 Furniture and fixtures 552 552 Leasehold improvements 939 351 Construction in progress 201 — Total property and equipment 5,639 4,178 Less accumulated depreciation and amortization (1,654 ) (991 ) Property and equipment, net $ 3,985 $ 3,187 |
Accrued Expenses and Other Cu25
Accrued Expenses and Other Current Liabilities (Tables) | 6 Months Ended |
Jun. 30, 2016 | |
Payables And Accruals [Abstract] | |
Schedule of Accrued Expenses and Other Current Liabilities | Accrued expenses and other current liabilities consist of the following (in thousands): June 30, 2016 December Employees’ compensation expenses $ 1,608 $ 2,047 Accrued professional services 608 1,177 Accrued preclinical costs $ 1,844 $ 642 Accrued clinical and process development costs — 101 Other 84 40 Total accrued expenses and other current liabilities $ 4,144 $ 4,007 |
Stock Option Plans (Tables)
Stock Option Plans (Tables) | 6 Months Ended |
Jun. 30, 2016 | |
Stock-Based Compensation Expense Related to Issuance of Stock Option Awards to Employees and Nonemployees | The Company has recorded aggregate stock-based compensation expense related to the issuance of stock option awards to employees and nonemployees in the condensed consolidated statement of operations and comprehensive loss as follows (in thousands): Three Months Ended Six Months Ended June 30, June 30, 2016 2015 2016 2015 Research and development $ 1,912 $ (445 ) $ 4,140 $ (161 ) General and administrative 914 1,004 2,770 1,493 Total share-based compensation $ 2,826 $ 559 $ 6,910 $ 1,332 |
Summary of Restricted Stock Units Activity | The following table summarizes the RSUs activity under our stock plans and related information: Number of Weighted-Average Shares Grant-Date (in thousands) Fair Value (in dollars) Outstanding at December 31, 2015 632 $ 13.07 Granted 1,066 $ 4.63 Vested and released (165 ) $ 11.60 Forfeited (342 ) $ 7.85 Outstanding at June 30, 2016 1,191 $ 6.50 |
Schedule of Fair Value of Option Issued to Employees Valuation Assumptions | The fair value of each option issued to employees was estimated at the date of grant using the Black-Scholes valuation model with the following weighted-average assumptions: Three Months Ended Six Months Ended June 30, June 30, 2016 2015 2016 2015 Option grants: Expected volatility 81 % 76 % 81 % 77 % Expected term (in years) 5.9 6.0 5.9 6.1 Expected dividend yield — — — — Risk-free interest rate 1.3 % 1.6 % 1.3 % 1.6 % |
Non Employee Stock Option [Member] | |
Schedule of Non-Employees Stock Purchase Plan Valuation Assumptions | The following weighted-average assumptions were used in estimating non-employees’ stock-based compensation expenses: Three Months Ended Six Months Ended June 30, June 30, 2016 2015 2016 2015 Option grants: Expected volatility 84 % 80 % 83 % 80 % Expected term (in years) 8.0 8.0 7.5 7.8 Expected dividend yield — — — — Risk-free interest rate 1.6 % 2.0 % 1.6 % 1.9 % |
2006 and 2014 Equity Incentive Plan [Member] | |
Summary of Stock Options Activity | The following table summarizes option activity under our stock plans and related information: WEIGHTED- NUMBER OF WEIGHTED- AVERAGE AGGREGATE SHARE AVERAGE EXERCISE CONTRACTUAL LIFE INTRINSIC VALUE (a) (IN PRICE (IN YEARS) (IN THOUSANDS) Balance at January 1, 2016 5,494 $ 8.75 Options granted 4,975 $ 1.25 Options exercised (1,233 ) $ 0.40 Options cancelled (511 ) $ 23.83 Balance at June 30, 2016 8,725 $ 4.77 8.9 15,473 Vested and expected to vest as of June 30, 2016 8,622 $ 4.72 8.9 15,471 Exercisable as of June 30, 2016 3,760 $ 3.62 8.2 8,997 (a) The aggregate intrinsic value is calculated as the difference between the option exercise price and the closing price of common stock of $3.16 per share as of June 30, 2016. |
Organization and Basis of Pre27
Organization and Basis of Presentation - Additional Information (Detail) - USD ($) $ / shares in Units, $ in Thousands | May 11, 2016 | Mar. 31, 2015 | Jan. 31, 2015 | Jun. 30, 2016 | Jun. 30, 2015 | Dec. 31, 2015 |
Class of Stock [Line Items] | ||||||
Date of incorporation | Jul. 17, 2006 | |||||
Change of entity name date | May 12, 2016 | |||||
Accumulated deficit | $ (161,220) | $ (84,168) | ||||
Common stock, par value | $ 0.0001 | $ 0.0001 | ||||
Issuance of common stock | 230,000 | 2,369,375 | ||||
Net proceeds from issuance of common stock | $ 8,300 | $ 130,600 | $ 138,954 | |||
Over-Allotment Option [Member] | ||||||
Class of Stock [Line Items] | ||||||
Issuance of common stock | 359,918 | |||||
Annapurna Therapeutics SAS [Member] | ||||||
Class of Stock [Line Items] | ||||||
Number of shares issued to acquired an entity | 14,087,246 | |||||
Common stock, par value | $ 0.0001 |
Summary of Significant Accoun28
Summary of Significant Accounting Policies - Additional Information (Detail) - USD ($) | 2 Months Ended | 6 Months Ended | |
Jun. 30, 2016 | Jun. 30, 2016 | Jun. 30, 2015 | |
Summary Of Significant Accounting Policies [Line Items] | |||
Impairment losses of long-lived assets | $ 0 | $ 0 | |
Annapurna Therapeutics SAS [Member] | |||
Summary Of Significant Accounting Policies [Line Items] | |||
Acquisition closing date | May 11, 2016 | ||
IPR&D [Member] | Annapurna Therapeutics SAS [Member] | |||
Summary Of Significant Accounting Policies [Line Items] | |||
Impairment of intangible assets | $ 0 | ||
Changes in fair value of assets | $ 0 |
Acquisition of Annapurna - Addi
Acquisition of Annapurna - Additional Information (Detail) | May 11, 2016USD ($)Segment$ / sharesshares | May 31, 2016USD ($) | Jun. 30, 2016USD ($) | Jun. 30, 2016USD ($) | Jun. 30, 2015USD ($) | Jun. 30, 2016USD ($) | Jun. 30, 2015USD ($) |
Business Acquisition [Line Items] | |||||||
Total purchase price consideration | $ 64,845,000 | ||||||
Expected volatility | 81.00% | 76.00% | 81.00% | 77.00% | |||
Risk-free interest rate | 1.30% | 1.60% | 1.30% | 1.60% | |||
Expected lives | 5 years 10 months 24 days | 6 years | 5 years 10 months 24 days | 6 years 1 month 6 days | |||
Non Employee Stock Option [Member] | |||||||
Business Acquisition [Line Items] | |||||||
Expected volatility | 84.00% | 80.00% | 83.00% | 80.00% | |||
Risk-free interest rate | 1.60% | 2.00% | 1.60% | 1.90% | |||
Expected lives | 8 years | 8 years | 7 years 6 months | 7 years 9 months 18 days | |||
Annapurna Therapeutics SAS [Member] | |||||||
Business Acquisition [Line Items] | |||||||
Date of acquisition | May 11, 2016 | ||||||
Total purchase price consideration | $ 64,845,000 | ||||||
Number of shares issued to acquired an entity | shares | 14,087,246 | ||||||
Fair value of awards related to accelerated vesting of options and shares | $ 900,000 | ||||||
Common stock exchange ratio | 9.54655 | 9.54655 | 9.54655 | ||||
Acquisition costs | $ 600,000 | $ 2,500,000 | |||||
Fair value, discounted rate | 11.00% | ||||||
Goodwill | $ 49,120,000 | $ 0 | 0 | 0 | |||
Number of reporting unit | Segment | 1 | ||||||
Amount of net loss included in the consolidated statements of operations from acquisition date | 1,200,000 | ||||||
Revenues from the acquisition date | $ 0 | ||||||
Interest expense related to convertible notes and changes in fair value of preferred stock warrants | 500,000 | $ 500,000 | 1,000,000 | $ 500,000 | |||
Bonuses paid | $ 400,000 | ||||||
Annapurna Therapeutics SAS [Member] | Stock Options [Member] | |||||||
Business Acquisition [Line Items] | |||||||
Shares reserved for future exercise of stock options | shares | 3,673,940 | ||||||
Fair value of estimated stock options and stock-based awards at acquisition | $ 14,700,000 | ||||||
Dividends | 0.00% | ||||||
Expected volatility | 80.00% | ||||||
Risk-free interest rate | 1.40% | ||||||
Expected lives | 6 years | ||||||
Fair value of stock options attributed as pre-combination service and included as part of acquisition consideration | $ 7,400,000 | ||||||
Fair value of stock options post-combination attribution recognized as compensation expense | $ 7,200,000 | ||||||
Stock-based compensation expense related to vesting of stock options | $ 1,100,000 | ||||||
Annapurna Therapeutics SAS [Member] | Stock Options [Member] | Executives [Member] | |||||||
Business Acquisition [Line Items] | |||||||
Stock-based compensation expense related to vesting of stock options | $ 100,000 | $ 200,000 | |||||
Annapurna Therapeutics SAS [Member] | Non Employee Stock Option [Member] | |||||||
Business Acquisition [Line Items] | |||||||
Dividends | 0.00% | ||||||
Expected volatility | 89.00% | ||||||
Risk-free interest rate | 1.10% | ||||||
Expected lives | 10 years | ||||||
Annapurna Therapeutics SAS [Member] | Common Shares [Member] | |||||||
Business Acquisition [Line Items] | |||||||
Number of shares issued to acquired an entity | shares | 14,087,246 | ||||||
Share price of common stock on date of acquisition | $ / shares | $ 4.14 |
Acquisition of Annapurna - Summ
Acquisition of Annapurna - Summary of Total Purchase Price Consideration (Detail) - USD ($) $ in Thousands | May 11, 2016 | Jun. 30, 2016 |
Business Acquisition [Line Items] | ||
Total purchase price consideration | $ 64,845 | |
Annapurna Therapeutics SAS [Member] | ||
Business Acquisition [Line Items] | ||
Less: value of common stock and options accelerated vesting at the closing date | $ (898) | |
Total purchase price consideration | 64,845 | |
Annapurna Therapeutics SAS [Member] | Common Shares [Member] | ||
Business Acquisition [Line Items] | ||
Fair value of common shares issued/common share options exchanged | 58,321 | |
Annapurna Therapeutics SAS [Member] | Common Shares [Member] | Stock Options [Member] | ||
Business Acquisition [Line Items] | ||
Fair value of common shares issued/common share options exchanged | $ 7,422 |
Acquisition of Annapurna - Comp
Acquisition of Annapurna - Components of Preliminary Allocation of Total Purchase Price Consideration (Detail) - Annapurna Therapeutics SAS [Member] - USD ($) $ in Thousands | Jun. 30, 2016 | May 11, 2016 |
Business Acquisition [Line Items] | ||
Cash | $ 3,449 | |
Prepaid expenses and other assets | 865 | |
Property and equipment | 185 | |
Acquired intangible assets | 16,650 | |
Goodwill | $ 0 | 49,120 |
Accounts payable | (1,118) | |
Accrued liabilities | (1,848) | |
Other noncurrent liabilities | (377) | |
Deferred tax liabilities | (2,081) | |
Total purchase price allocation | $ 64,845 |
Acquisition of Annapurna - Su32
Acquisition of Annapurna - Summary of Identifiable Intangible Assets Acquired (Detail) - Annapurna Therapeutics SAS [Member] $ in Thousands | May 11, 2016USD ($) |
Acquired Indefinite Lived Intangible Assets [Line Items] | |
Acquired intangible assets | $ 16,650 |
IPR&D [Member] | |
Acquired Indefinite Lived Intangible Assets [Line Items] | |
Acquired intangible assets | 16,650 |
IPR&D [Member] | Alpha-1 Antitrypsin Deficiency [Member] | |
Acquired Indefinite Lived Intangible Assets [Line Items] | |
Acquired intangible assets | 12,150 |
IPR&D [Member] | Hereditary Angiodema [Member] | |
Acquired Indefinite Lived Intangible Assets [Line Items] | |
Acquired intangible assets | $ 4,500 |
Acquisition of Annapurna - Sche
Acquisition of Annapurna - Schedule of Pro Forma Financial Information (Detail) - Annapurna Therapeutics SAS [Member] - USD ($) $ / shares in Units, $ in Thousands | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2016 | Jun. 30, 2015 | Jun. 30, 2016 | Jun. 30, 2015 | |
Pro forma information | ||||
Collaboration revenue | $ 307 | $ 203 | $ 572 | $ 406 |
Net loss | $ (62,572) | $ (10,533) | $ (80,846) | $ (21,956) |
Basic and diluted loss per share | $ (1.52) | $ (0.27) | $ (1.97) | $ (0.56) |
Acquisition of Annapurna - Ad34
Acquisition of Annapurna - Additional Information1 (Detail) - USD ($) | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2016 | Jun. 30, 2016 | May 11, 2016 | Dec. 31, 2015 | |
Business Acquisition [Line Items] | ||||
Common shares outstanding | 41,322,357 | 41,322,357 | 25,858,722 | |
Goodwill impairment charge | $ 49,120,000 | $ 49,120,000 | ||
Annapurna Therapeutics SAS [Member] | ||||
Business Acquisition [Line Items] | ||||
Stock price | $ 3.16 | $ 3.16 | ||
Common shares outstanding | 41,300,000 | 41,300,000 | ||
Goodwill | $ 0 | $ 0 | $ 49,120,000 | |
Goodwill impairment charge | 49,100,000 | |||
Annapurna Therapeutics SAS [Member] | IPR&D [Member] | ||||
Business Acquisition [Line Items] | ||||
Impairment of intangible assets | $ 0 |
Cash Equivalents and Marketab35
Cash Equivalents and Marketable Securities - Additional Information (Detail) | 6 Months Ended |
Jun. 30, 2016USD ($)Security | |
Cash And Cash Equivalents [Abstract] | |
Number of available-for-securities held | Security | 0 |
Contractual maturities of marketable securities | Less than one year |
Proceeds from sales of available-for-sale securities | $ | $ 0 |
Cash Equivalents and Marketab36
Cash Equivalents and Marketable Securities - Summary of Cash Equivalents and Marketable Securities (Detail) $ in Thousands | Dec. 31, 2015USD ($) |
Cash and Cash Equivalents [Line Items] | |
Amortized Cost Basis | $ 37,738 |
Unrealized Loses | (6) |
Estimated Fair Value | 37,732 |
Less: Cash Equivalents, Amortized Cost Basis | (208,588) |
Less: Cash Equivalents, Unrealized Gains | 0 |
Less: Cash Equivalents, Unrealized Loses | 0 |
Less: Cash Equivalents, Estimated Fair Value | (208,588) |
Cash Equivalents And Marketable Securities [Member] | |
Cash and Cash Equivalents [Line Items] | |
Amortized Cost Basis | 246,326 |
Unrealized Loses | (6) |
Estimated Fair Value | 246,320 |
Money Market Funds [Member] | |
Cash and Cash Equivalents [Line Items] | |
Amortized Cost Basis | 208,588 |
Estimated Fair Value | 208,588 |
Certificates of Deposit [Member] | |
Cash and Cash Equivalents [Line Items] | |
Amortized Cost Basis | 1,680 |
Estimated Fair Value | 1,680 |
U.S. Treasury Securities [Member] | |
Cash and Cash Equivalents [Line Items] | |
Amortized Cost Basis | 15,046 |
Unrealized Loses | (4) |
Estimated Fair Value | 15,042 |
US Government Agency Securities [Member] | |
Cash and Cash Equivalents [Line Items] | |
Amortized Cost Basis | 21,012 |
Unrealized Loses | (2) |
Estimated Fair Value | $ 21,010 |
Fair Value Measurements and F37
Fair Value Measurements and Fair Value of Financial Instruments - Additional Information (Detail) - USD ($) | 6 Months Ended | 12 Months Ended |
Jun. 30, 2016 | Dec. 31, 2015 | |
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Liabilities transferred within Level 3 | $ 0 | $ 0 |
Total assets | 236,821,000 | 246,320,000 |
Significant Unobservable Inputs (Level 3) [Member] | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Total assets | 0 | 0 |
Total liabilities | $ 0 | $ 0 |
Fair Value Measurements and F38
Fair Value Measurements and Fair Value of Financial Instruments - Financial Assets Measured and Recognized at Fair Value on Recurring Basis (Detail) - USD ($) | Jun. 30, 2016 | Dec. 31, 2015 |
Assets: | ||
Total assets | $ 236,821,000 | $ 246,320,000 |
Money Market Funds - Cash Equivalent [Member] | ||
Assets: | ||
Total assets | 236,821,000 | 208,588,000 |
Certificates of Deposit [Member] | ||
Assets: | ||
Total assets | 1,680,000 | |
U.S. Treasury Securities [Member] | ||
Assets: | ||
Total assets | 15,042,000 | |
Quoted Prices in Active Markets (Level 1) [Member] | ||
Assets: | ||
Total assets | 236,821,000 | 208,588,000 |
Quoted Prices in Active Markets (Level 1) [Member] | Money Market Funds - Cash Equivalent [Member] | ||
Assets: | ||
Total assets | 236,821,000 | 208,588,000 |
Significant Other Observable Inputs (Level 2) [Member] | ||
Assets: | ||
Total assets | 37,732,000 | |
Significant Other Observable Inputs (Level 2) [Member] | Certificates of Deposit [Member] | ||
Assets: | ||
Total assets | 1,680,000 | |
Significant Other Observable Inputs (Level 2) [Member] | U.S. Treasury Securities [Member] | ||
Assets: | ||
Total assets | 15,042,000 | |
Significant Unobservable Inputs (Level 3) [Member] | ||
Assets: | ||
Total assets | $ 0 | 0 |
U.S. Government Agency Securities [Member] | ||
Assets: | ||
Total assets | 21,010,000 | |
U.S. Government Agency Securities [Member] | Significant Other Observable Inputs (Level 2) [Member] | ||
Assets: | ||
Total assets | $ 21,010,000 |
Significant Agreements - Additi
Significant Agreements - Additional Information (Detail) € in Millions | 1 Months Ended | 2 Months Ended | 6 Months Ended | |||||
Dec. 31, 2015USD ($)Agreement | Oct. 31, 2015USD ($) | Oct. 31, 2015EUR (€) | May 31, 2014USD ($)Target | Apr. 30, 2014USD ($) | May 31, 2010USD ($) | Jun. 30, 2016USD ($) | Jun. 30, 2016USD ($) | |
University of California [Member] | ||||||||
License Agreement [Line Items] | ||||||||
Upfront license fee paid | $ 100,000 | |||||||
Annual maintenance fee payable | 50,000 | |||||||
Aggregate amount of milestone payments for first indication | 900,000 | |||||||
Additional milestone payments for up to two additional indications | $ 500,000 | |||||||
Additional milestone payments recognized | $ 0 | |||||||
Cornell University [Member] | ||||||||
License Agreement [Line Items] | ||||||||
Additional milestone payments recognized | $ 0 | |||||||
Service fees payable | $ 13,300,000 | |||||||
Service fees, payable period | 4 years | |||||||
Number of license agreements | Agreement | 3 | |||||||
License agreement termination notice period | 90 days | |||||||
Aggregate milestone payments and royalties on sales | $ 16,000,000 | |||||||
License agreement, accrued expenses | $ 500,000 | 500,000 | ||||||
License agreement, recorded expenses | 500,000 | |||||||
Cornell University [Member] | Consultant [Member] | ||||||||
License Agreement [Line Items] | ||||||||
Annual compensation | 300,000 | |||||||
Cornell University [Member] | Minimum [Member] | ||||||||
License Agreement [Line Items] | ||||||||
Annual maintenance fee payable | 30,000 | |||||||
Cornell University [Member] | Maximum [Member] | ||||||||
License Agreement [Line Items] | ||||||||
Annual maintenance fee payable | 300,000 | |||||||
REGENXBIO [Member] | ||||||||
License Agreement [Line Items] | ||||||||
Additional milestone payments recognized | 0 | |||||||
License agreement, accrued expenses | 46,000 | 46,000 | ||||||
License agreement, recorded expenses | 20,000 | |||||||
REGENXBIO [Member] | A1AT Deficiency/Allergy License Agreement [Member] | ||||||||
License Agreement [Line Items] | ||||||||
Aggregate milestone payments and royalties on sales | $ 20,000,000 | |||||||
REGENXBIO [Member] | Friedreich's Ataxia License Agreement [Member] | ||||||||
License Agreement [Line Items] | ||||||||
Aggregate milestone payments and royalties on sales | $ 13,850,000 | |||||||
Inserm Transfert [Member] | ||||||||
License Agreement [Line Items] | ||||||||
Additional milestone payments recognized | 0 | |||||||
Aggregate milestone payments and royalties on sales | € | € 2 | |||||||
License agreement, accrued expenses | 0 | $ 0 | ||||||
License agreement, recorded expenses | $ 0 | |||||||
Regeneron Corporation [Member] | ||||||||
License Agreement [Line Items] | ||||||||
Potential target of collaborations | Target | 8 | |||||||
Initial collaboration term | 3 years | |||||||
Additional collaboration term | 5 years | |||||||
Collaboration fee for exclusive worldwide license per target | $ 2,000,000 | |||||||
Potential milestone earnings | 640,000,000 | |||||||
Reimbursement of research and development expense | 35.00% | |||||||
Agreement termination scenario description | Regeneron may terminate the agreement for convenience at any time on a target by target basis or in totality upon a 30-day notice.Each party can terminate the agreement if another party commits a material breach or material default in performance of its obligations and such breach or default is not cured within 60 days.The agreement is automatically terminated upon initiation of any bankruptcy proceedings, reorganization or dissolution of either party.The Company can terminate the agreement upon 30-day notice if Regeneron challenges the validity, scope or enforceability of any Company patent. | |||||||
Regeneron Corporation [Member] | Development and Regulatory Milestone [Member] | ||||||||
License Agreement [Line Items] | ||||||||
Potential milestone earnings | $ 80,000,000 |
Property and Equipment, Net - S
Property and Equipment, Net - Schedule of Property and Equipment, Net (Detail) - USD ($) $ in Thousands | Jun. 30, 2016 | Dec. 31, 2015 |
Property, Plant and Equipment [Line Items] | ||
Total property and equipment | $ 5,639 | $ 4,178 |
Less accumulated depreciation and amortization | (1,654) | (991) |
Property and equipment, net | 3,985 | 3,187 |
Computer Equipment and Software [Member] | ||
Property, Plant and Equipment [Line Items] | ||
Total property and equipment | 259 | 234 |
Laboratory Equipment [Member] | ||
Property, Plant and Equipment [Line Items] | ||
Total property and equipment | 3,688 | 3,041 |
Furniture and Fixtures [Member] | ||
Property, Plant and Equipment [Line Items] | ||
Total property and equipment | 552 | 552 |
Leasehold Improvements [Member] | ||
Property, Plant and Equipment [Line Items] | ||
Total property and equipment | 939 | $ 351 |
Construction in Progress [Member] | ||
Property, Plant and Equipment [Line Items] | ||
Total property and equipment | $ 201 |
Property and Equipment, Net - A
Property and Equipment, Net - Additional Information (Detail) - USD ($) | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2016 | Jun. 30, 2015 | Jun. 30, 2016 | Jun. 30, 2015 | |
Property Plant And Equipment [Abstract] | ||||
Depreciation and amortization expense | $ 343,000 | $ 147,000 | $ 663,000 | $ 265,000 |
Accrued Expenses and Other Cu42
Accrued Expenses and Other Current Liabilities - Schedule of Accrued Expenses and Other Current Liabilities (Detail) - USD ($) $ in Thousands | Jun. 30, 2016 | Dec. 31, 2015 |
Accrued Expenses And Other Current Liabilities [Abstract] | ||
Employees’ compensation expenses | $ 1,608 | $ 2,047 |
Accrued professional services | 608 | 1,177 |
Accrued preclinical costs | 1,844 | 642 |
Accrued clinical and process development costs | 101 | |
Other | 84 | 40 |
Total accrued expenses and other current liabilities | $ 4,144 | $ 4,007 |
Other Liabilities - Additional
Other Liabilities - Additional Information (Detail) - BPI France [Member] | 2 Months Ended | 6 Months Ended | |||
Jun. 30, 2016USD ($) | Jun. 30, 2016EUR (€) | Jun. 30, 2016EUR (€) | Dec. 31, 2015EUR (€) | Aug. 31, 2015EUR (€) | |
Line Of Credit Facility [Line Items] | |||||
Interest free conditional advance, conditions | The funds received by the Company are intended to finance its research and development efforts and the recruitment of specific personnel. | ||||
Interest free conditional advance, maximum borrowing capacity | € 750,000 | ||||
Interest free conditional advance | $ 373,000 | € 500,000 | |||
Interest free conditional advance, remaining borrowing capacity | € 250,000 | ||||
Interest free conditional advance, frequency of payments | Quarterly | ||||
Interest free conditional advance, periodic payment | € 25,000 | ||||
Interest free conditional advance, beginning date of periodic payment | Sep. 30, 2017 | ||||
Interest free conditional advance, ending date of periodic payment | Jun. 30, 2022 | ||||
Interest free conditional advance, imputed interest rate | 7.00% | 7.00% | |||
Interest free conditional advance, interest expense | $ | $ 15,000 |
Commitments and Contingencies -
Commitments and Contingencies - Additional Information (Detail) | 1 Months Ended | 6 Months Ended | |
Dec. 31, 2015USD ($)Claim | Jul. 31, 2015Claim | Jun. 30, 2016USD ($)Claim | |
Other Commitments [Line Items] | |||
Accrued royalties | $ 0 | ||
Claims paid to date related to indemnification issues | $ 0 | ||
Number of claims to date | Claim | 1 | 3 | 0 |
Accruals or expenses related to indemnification issues | $ 0 | ||
Cornell University [Member] | |||
Other Commitments [Line Items] | |||
Service fees payable | $ 13,300,000 | ||
Service fees, payable period | 4 years |
Stock Option Plans - Additional
Stock Option Plans - Additional Information (Detail) $ / shares in Units, $ in Millions | 3 Months Ended | 6 Months Ended | |
Jun. 30, 2016USD ($)Executiveshares | Jun. 30, 2016USD ($)Executive$ / sharesshares | Jun. 30, 2015USD ($)$ / sharesshares | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||
Weighted-average fair values of options granted and exchanged | $ / shares | $ 1.62 | $ 27.90 | |
Total intrinsic value of options exercised | $ | $ 6.3 | $ 10.6 | |
Number of executives affected by modifications of stock-based compensation awards | Executive | 3 | 3 | |
Unrecognized stock-based compensation, weighted-average period | 3 years | ||
Unrecognized stock-based compensation expense related to employees' awards | $ | $ 18.4 | $ 18.4 | |
Restricted Stock Units [Member] | |||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||
RSU granted | shares | 1,066,283 | 97,000 | |
Total fair values of RSUs vested | $ | $ 2.2 | $ 0 | |
Unrecognized compensation cost | $ | 5.3 | $ 5.3 | |
Unrecognized stock-based compensation, weighted-average period | 2 years 9 months 18 days | ||
Non Employee Stock Option [Member] | |||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||
Unrecognized stock-based compensation, weighted-average period | 1 year 3 months 18 days | ||
Unrecognized stock-based compensation expense related to employees' awards | $ | 2.5 | $ 2.5 | |
Minimum [Member] | Restricted Stock Units [Member] | |||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||
Restricted stock units granted to employees vesting period | 2 years | ||
Maximum [Member] | Restricted Stock Units [Member] | |||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||
Restricted stock units granted to employees vesting period | 4 years | ||
Research and Development [Member] | |||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||
Stock-based compensation expense for modifications | $ | 0 | $ 1.4 | |
General and Administrative [Member] | |||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||
Stock-based compensation expense for modifications | $ | $ 0.3 | $ 1.1 | |
Annapurna Therapeutics SAS [Member] | |||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||
Number of stock options granted | shares | 3,673,940 | ||
Stock options exercise price granted | $ / shares | $ 0.21 | ||
2014 Equity Incentive Award Plan [Member] | |||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||
Common stock shares authorized for issuance | shares | 13,162,656 | 13,162,656 | |
Shares available for future grants | shares | 2,241,693 | 2,241,693 | |
2014 Employee Stock Purchase Plan [Member] | |||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||
Shares available for future grants | shares | 623,381 | 623,381 | |
Common stock, shares issued | shares | 32,425 | 0 | |
Common stock shares available for future grant | shares | 675,383 | 675,383 |
Stock Option Plans - Summary of
Stock Option Plans - Summary of Stock Options Activity (Detail) - 2006 Equity Incentive Plan [Member] $ / shares in Units, $ in Thousands | 6 Months Ended |
Jun. 30, 2016USD ($)$ / sharesshares | |
Share-based Compensation Arrangement by Share-based Payment Award, Options, Nonvested, Number of Shares [Roll Forward] | |
NUMBER OF SHARES, Beginning Balance | shares | 5,494,000 |
NUMBER OF SHARES, Options granted | shares | 4,975,000 |
NUMBER OF SHARES, Options exercised | shares | (1,233,000) |
NUMBER OF SHARES, Options cancelled | shares | (511,000) |
NUMBER OF SHARES, Ending Balance | shares | 8,725,000 |
NUMBER OF SHARES, Vested and expected to vest | shares | 8,622,000 |
NUMBER OF SHARES, Exercisable | shares | 3,760,000 |
Share-based Compensation Arrangement by Share-based Payment Award, Options, Outstanding, Weighted Average Exercise Price [Abstract] | |
WEIGHTED-AVERAGE EXERCISE PRICE, Beginning Balance | $ / shares | $ 8.75 |
WEIGHTED-AVERAGE EXERCISE PRICE, Options granted | $ / shares | 1.25 |
WEIGHTED-AVERAGE EXERCISE PRICE, Options exercised | $ / shares | 0.40 |
WEIGHTED-AVERAGE EXERCISE PRICE, Options cancelled | $ / shares | 23.83 |
WEIGHTED-AVERAGE EXERCISE PRICE, Ending Balance | $ / shares | 4.77 |
WEIGHTED-AVERAGE EXERCISE PRICE, Vested and expected to vest | $ / shares | 4.72 |
WEIGHTED-AVERAGE EXERCISE PRICE, Exercisable | $ / shares | $ 3.62 |
Share-based Compensation Arrangement by Share-based Payment Award, Options, Additional Disclosures [Abstract] | |
WEIGHTED-AVERAGE REMAINING CONTRACTUAL LIFE (IN YEARS) | 8 years 10 months 24 days |
WEIGHTED-AVERAGE REMAINING CONTRACTUAL LIFE (IN YEARS), Vested and expected to vest | 8 years 10 months 24 days |
WEIGHTED-AVERAGE REMAINING CONTRACTUAL LIFE (IN YEARS), Exercisable | 8 years 2 months 12 days |
AGGREGATE INTRINSIC VALUE, Options outstanding | $ | $ 15,473 |
AGGREGATE INTRINSIC VALUE, Vested and expected to vest | $ | 15,471 |
AGGREGATE INTRINSIC VALUE, Exercisable | $ | $ 8,997 |
Stock Option Plans - Summary 47
Stock Option Plans - Summary of Stock Options Activity (Parenthetical) (Detail) | 6 Months Ended |
Jun. 30, 2016$ / shares | |
Equity [Abstract] | |
Aggregate intrinsic value of option per share | $ 3.16 |
Stock Option Plans - Stock-Base
Stock Option Plans - Stock-Based Compensation Expense Related to Issuance of Stock Option Awards to Employees and Nonemployees (Detail) - USD ($) $ in Thousands | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2016 | Jun. 30, 2015 | Jun. 30, 2016 | Jun. 30, 2015 | |
Share-based Compensation Arrangement by Share-based Payment Award, Compensation Cost [Line Items] | ||||
Total share-based compensation | $ 2,826 | $ 559 | $ 6,910 | $ 1,332 |
Research and Development [Member] | ||||
Share-based Compensation Arrangement by Share-based Payment Award, Compensation Cost [Line Items] | ||||
Total share-based compensation | 1,912 | (445) | 4,140 | (161) |
General and Administrative [Member] | ||||
Share-based Compensation Arrangement by Share-based Payment Award, Compensation Cost [Line Items] | ||||
Total share-based compensation | $ 914 | $ 1,004 | $ 2,770 | $ 1,493 |
Stock Option Plans - Summary 49
Stock Option Plans - Summary of Restricted Stock Units Activity (Detail) - Restricted Stock Units [Member] - $ / shares | 6 Months Ended | |
Jun. 30, 2016 | Jun. 30, 2015 | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||
Number of Shares, Beginning Balance | 632,000 | |
Number of Shares, Granted | 1,066,283 | 97,000 |
Number of Shares, Vested and released | (165,000) | |
Number of Shares, Forfeited | (342,000) | |
Number of Shares, Ending Balance | 1,191,000 | |
Weighted-Average Grant-Date Fair Value, Beginning Balance | $ 13.07 | |
Weighted-Average Grant-Date Fair Value, Granted | 4.63 | |
Weighted-Average Grant-Date Fair Value, Vested and released | 11.60 | |
Weighted-Average Grant-Date Fair Value, Forfeited | 7.85 | |
Weighted-Average Grant-Date Fair Value, Ending Balance | $ 6.50 |
Stock Option Plans - Schedule o
Stock Option Plans - Schedule of Employees Stock Purchase Plan Valuation Assumptions (Detail) | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2016 | Jun. 30, 2015 | Jun. 30, 2016 | Jun. 30, 2015 | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Expected volatility | 81.00% | 76.00% | 81.00% | 77.00% |
Expected lives | 5 years 10 months 24 days | 6 years | 5 years 10 months 24 days | 6 years 1 month 6 days |
Risk-free interest rate | 1.30% | 1.60% | 1.30% | 1.60% |
Non Employee Stock Option [Member] | ||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Expected volatility | 84.00% | 80.00% | 83.00% | 80.00% |
Expected lives | 8 years | 8 years | 7 years 6 months | 7 years 9 months 18 days |
Risk-free interest rate | 1.60% | 2.00% | 1.60% | 1.90% |
401(k) Savings Plan - Additiona
401(k) Savings Plan - Additional Information (Detail) - USD ($) $ in Millions | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2016 | Jun. 30, 2015 | Jun. 30, 2016 | Jun. 30, 2015 | |
Compensation And Retirement Disclosure [Abstract] | ||||
Contribution by company | $ 0.1 | $ 0 | $ 0.2 | $ 0.1 |
Net Loss Per Share - Additional
Net Loss Per Share - Additional Information (Detail) - shares shares in Millions | 6 Months Ended | |
Jun. 30, 2016 | Jun. 30, 2015 | |
Earnings Per Share [Abstract] | ||
Antidilutive common stock equivalents excluded from calculation of diluted net loss per share | 9.9 | 5.2 |
Subsequent Events - Additional
Subsequent Events - Additional Information (Detail) - Editas Medicine Inc [Member] - Subsequent Event [Member] $ in Millions | Aug. 09, 2016USD ($) |
Subsequent Event [Line Items] | |
Non-refundable upfront payment receivable | $ 1 |
Collaboration, option and license agreement fee receivable | $ 1 |