Document and Entity Information
Document and Entity Information - USD ($) $ in Millions | 12 Months Ended | ||
Dec. 31, 2017 | Mar. 09, 2018 | Jun. 30, 2017 | |
Document and Entity Information | |||
Entity Registrant Name | Voyager Therapeutics, Inc. | ||
Entity Central Index Key | 1,640,266 | ||
Document Type | 10-K | ||
Document Period End Date | Dec. 31, 2017 | ||
Amendment Flag | false | ||
Current Fiscal Year End Date | --12-31 | ||
Entity Current Reporting Status | Yes | ||
Entity Voluntary Filers | No | ||
Entity Well-known Seasoned Issuer | No | ||
Entity Filer Category | Accelerated Filer | ||
Entity Public Float | $ 94.4 | ||
Entity Common Stock, Shares Outstanding | 32,206,786 | ||
Document Fiscal Year Focus | 2,017 | ||
Document Fiscal Period Focus | FY |
Balance Sheets
Balance Sheets - USD ($) $ in Thousands | Dec. 31, 2017 | Dec. 31, 2016 |
Current assets: | ||
Cash and cash equivalents | $ 31,530 | $ 36,641 |
Marketable securities | 137,522 | 137,777 |
Prepaid expenses and other current assets | 2,738 | 4,368 |
Total Current Assets | 171,790 | 178,786 |
Property, Plant and Equipment, Net | 10,283 | 7,893 |
Deposits and other non-current assets | 1,304 | 1,527 |
Marketable securities | 1,100 | 1,360 |
Total Assets | 184,477 | 189,566 |
Current liabilities: | ||
Accounts Payable | 1,020 | 550 |
Accrued expenses | 11,497 | 6,488 |
Deferred revenue, current portion | 3,380 | 6,764 |
Total current liabilities | 15,897 | 13,802 |
Deferred rent, net of current portion | 5,337 | 4,999 |
Deferred revenue, net of current portion | 28,180 | 34,818 |
Other non-current liabilities | 1,012 | 25 |
Total Liabilities | 50,426 | 53,644 |
Stockholders’ equity: | ||
Preferred stock, $0.001 par value: 5,000,000 shares authorized; no shares issued and outstanding at December 31, 2017 and 2016 | 0 | 0 |
Common stock, $0.001 par value: 120,000,000 shares authorized; 31,572,044 and 25,597,912 shares issued and outstanding at December 31, 2017 and 2016, respectively | 32 | 26 |
Additional paid-in capital | 295,019 | 225,963 |
Accumulated other comprehensive gain | (287) | (52) |
Accumulated deficit | (160,713) | (90,015) |
Total stockholders’ deficit | 134,051 | 135,922 |
Total liabilities and stockholders’ equity | $ 184,477 | $ 189,566 |
Balance Sheets (Parenthetical)
Balance Sheets (Parenthetical) - $ / shares | Dec. 31, 2017 | Dec. 31, 2016 |
Preferred Stock, par value (in dollars per share) | $ 0.001 | $ 0.001 |
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 (in dollars per share) | $ 0.001 | $ 0.001 |
Common Stock, Shares Authorized | 120,000,000 | 120,000,000 |
Common Stock, Shares, Issued | 31,572,044 | 25,597,912 |
Common Stock, Shares, Outstanding | 31,572,044 | 25,597,912 |
Statement of Operations and Com
Statement of Operations and Comprehensive Loss - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2017 | Dec. 31, 2016 | Dec. 31, 2015 | |
Income Statement [Abstract] | |||
Collaboration revenue | $ 10,135 | $ 14,220 | $ 17,334 |
Operating expenses: | |||
Research and development | 62,260 | 42,249 | 27,679 |
General and administrative | 19,738 | 13,270 | 9,909 |
Total operating expenses | 81,998 | 55,519 | 37,588 |
Operating loss | (71,863) | (41,299) | (20,254) |
Other income (expense), net: | |||
Interest income | 1,227 | 976 | 332 |
Other (expense) income, net | (62) | 182 | (9,750) |
Total other income (expense), net | 1,165 | 1,158 | (9,418) |
Loss before income taxes | (70,698) | (40,141) | (29,672) |
Income tax provision | (52) | ||
Net loss | (70,698) | (40,193) | (29,672) |
Other comprehensive loss | |||
Net unrealized (loss) gain on available-for-sale-securities, net of tax expense of $128 for the year ended December 31, 2016 | (235) | 199 | (251) |
Total other comprehensive (loss) income | (235) | 199 | (251) |
Comprehensive loss | (70,933) | (39,994) | (29,923) |
Reconciliation of net loss to net loss attributable to common stockholders: | |||
Accretion of redeemable convertible preferred stock to redemption value | (7,373) | ||
Accrued dividends on series A preferred stock | (1,245) | ||
Net loss attributable to common stockholders | $ (70,698) | $ (40,193) | $ (38,290) |
Net loss per share attributable to common stock, basic and diluted | $ 2.64 | $ 1.59 | $ 9.14 |
Weighted-average common shares outstanding, basic and diluted | 26,803,711 | 25,302,414 | 4,191,210 |
Statement of Operations and Co5
Statement of Operations and Comprehensive Loss (Parenthetical) $ in Thousands | 12 Months Ended |
Dec. 31, 2016USD ($) | |
Income Statement [Abstract] | |
Tax expense | $ 128 |
Consolidate Statements of Equit
Consolidate Statements of Equity - USD ($) $ in Thousands | Redeemable Convertible Preferred Stock Series A | Redeemable Convertible Preferred Stock Series B | Common Stock | Additional Paid-in Capital | Accumulated Other Comprehensive Loss | Accumulated Deficit | Total |
Stockholders' Equity, Beginning Balance at Dec. 31, 2014 | $ 21,979 | $ 1 | $ (20,831) | $ (20,830) | |||
Shares, Outstanding, Beginning Balance at Dec. 31, 2014 | 25,000,000 | 814,834 | |||||
Issuance of common stock | $ 6 | $ 72,948 | 72,954 | ||||
Issuance of common stock (in shares) | 5,750,000 | ||||||
Issuance of preferred stock, net of discount and issuance costs | $ 19,999 | $ 84,780 | |||||
Issuance of preferred stock, net of discount and issuance costs (in shares) | 20,000,000 | 30,000,001 | |||||
Reclassification of tranche rights upon issuance of Series A redeemable convertible preferred stock | $ 16,055 | ||||||
Conversion of redeemable convertible preferred stock to common stock | $ (60,182) | $ (90,005) | $ 18 | 144,675 | 5,494 | 150,187 | |
Conversion of redeemable convertible preferred stock to common stock (common stock shares) | 17,647,054 | ||||||
Conversion of redeemable convertible preferred stock to common stock (preferred stock shares) | (45,000,000) | (30,000,001) | |||||
Vesting of restricted common stock | 22 | 22 | |||||
Vesting of restricted common stock (in shares) | 717,747 | ||||||
Exercises of vested stock options | 10 | 10 | |||||
Exercises of vested stock options (in shares) | 1,344 | ||||||
Stock based compensation expense | 4,027 | 4,027 | |||||
Unrealized loss on available for-sale securities | $ (251) | (251) | |||||
Accretion of redeemable convertible preferred stock to redemption value | $ (2,149) | $ (5,225) | 2,560 | 4,813 | 7,373 | ||
Net loss | (29,672) | (29,672) | |||||
Stockholders' Equity, Ending Balance at Dec. 31, 2015 | $ 25 | 219,122 | (251) | (49,822) | 169,074 | ||
Shares, Outstanding, Ending Balance at Dec. 31, 2015 | 24,930,979 | ||||||
Vesting of restricted common stock | $ 1 | 17 | 18 | ||||
Vesting of restricted common stock (in shares) | 601,501 | ||||||
Exercises of vested stock options | 514 | 514 | |||||
Exercises of vested stock options (in shares) | 65,432 | ||||||
Stock based compensation expense | 6,310 | 6,310 | |||||
Unrealized loss on available for-sale securities | 199 | 199 | |||||
Net loss | (40,193) | (40,193) | |||||
Stockholders' Equity, Ending Balance at Dec. 31, 2016 | $ 26 | 225,963 | (52) | (90,015) | 135,922 | ||
Shares, Outstanding, Ending Balance at Dec. 31, 2016 | 25,597,912 | ||||||
Issuance of common stock | $ 5 | 57,989 | 57,994 | ||||
Issuance of common stock (in shares) | 5,175,000 | ||||||
Vesting of restricted common stock | $ 1 | 12 | 13 | ||||
Vesting of restricted common stock (in shares) | 573,803 | ||||||
Exercises of vested stock options | 1,363 | 1,363 | |||||
Exercises of vested stock options (in shares) | 158,677 | ||||||
Stock based compensation expense | 9,129 | 9,129 | |||||
Unrealized loss on available for-sale securities | (235) | (235) | |||||
Issuance of common stock under ESPP | 563 | 563 | |||||
Issuance of common stock under ESPP (in shares) | 66,652 | ||||||
Net loss | (70,698) | (70,698) | |||||
Stockholders' Equity, Ending Balance at Dec. 31, 2017 | $ 32 | $ 295,019 | $ (287) | $ (160,713) | $ 134,051 | ||
Shares, Outstanding, Ending Balance at Dec. 31, 2017 | 31,572,044 |
Consolidated Statements of Equi
Consolidated Statements of Equity (Parenthetical) - USD ($) $ in Thousands | 12 Months Ended | 24 Months Ended | |
Dec. 31, 2015 | Dec. 31, 2015 | Dec. 31, 2017 | |
Issuance costs | $ 200 | ||
Common Stock | |||
Discounts and issuance costs | 7,500 | $ 7,500 | $ 4,100 |
Redeemable Convertible Preferred Stock Series A | |||
Issuance costs | 1 | 32 | |
Redeemable Convertible Preferred Stock Series B | |||
Issuance costs | 220 | ||
Discount | $ 5,000 | $ 5,000 |
Statement of Cash Flows
Statement of Cash Flows - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2017 | Dec. 31, 2016 | Dec. 31, 2015 | |
Cash flow from operating activities | |||
Net loss | $ (70,698) | $ (40,193) | $ (29,672) |
Adjustments to reconcile net loss to net cash used in operating activities: | |||
Stock-based compensation expense | 9,238 | 6,310 | 4,027 |
Depreciation | 1,595 | 612 | 600 |
Amortization of premiums and discounts on marketable securities | (24) | 696 | 452 |
Change in fair value of preferred stock tranche liability | 9,750 | ||
In-kind research and development expenses | 113 | 1,182 | 2,316 |
Other non-cash items | 46 | 709 | (277) |
Changes in operating assets and liabilities: | |||
Prepaid expenses and other current assets | 1,630 | (847) | (234) |
Other non-current assets | 7 | 14 | |
Deferred revenue | (10,135) | (14,582) | 52,666 |
Accounts payable | 470 | (62) | (942) |
Accrued expenses | 4,900 | 2,636 | 2,788 |
Other non-current liabilities | 1,000 | (189) | |
Lease incentive benefit | 515 | 1,050 | |
Net cash (used in) provided by operating activities | (61,350) | (42,482) | 41,299 |
Cash flow from investing activities | |||
Purchases of property and equipment | (3,985) | (5,029) | (1,030) |
Change in restricted cash | (421) | ||
Purchases of marketable securities | (147,296) | (112,350) | (220,399) |
Proceeds from maturities or sales of marketable securities | 147,600 | 165,100 | 26,660 |
Net cash (used in) provided by investing activities | (3,681) | 47,300 | (194,769) |
Cash flow from financing activities | |||
Proceeds from the issuance of stock net of discount and issuance costs | 57,994 | 104,779 | |
Proceeds from the exercise of stock options | 1,363 | 514 | 72,965 |
Proceeds from the purchase of common stock under ESPP | 563 | ||
Net cash provided by financing activities | 59,920 | 514 | 177,744 |
Net (decrease) increase in cash and cash equivalents | (5,111) | 5,332 | 24,274 |
Cash and cash equivalents, beginning of period | 36,641 | 31,309 | 7,035 |
Cash and cash equivalents, end of period | $ 31,530 | 36,641 | 31,309 |
Supplemental Disclosure of Cash and non-cash Activities | |||
Capital expenditures incurred but not yet paid | $ 242 | ||
Accretion of redeemable convertible preferred stock to redemption value | 7,373 | ||
Conversion of redeemable convertible preferred stock to common stock | $ 150,187 |
Nature of business
Nature of business | 12 Months Ended |
Dec. 31, 2017 | |
Nature of business | |
Nature of business | VOYAGER THERAPEUTICS INC. NOTES TO CONSOLIDATED FINANCIAL STATEMENTS 1. Nature of business The Company is a clinical-stage gene therapy company focused on developing life-changing treatments for patients suffering from severe neurological diseases. The Company is focused on neurological diseases where it believes an adeno-associated virus (“AAV”) gene therapy approach that either increases or decreases the production of a specific protein can slow or reduce the symptoms experienced by patients, and therefore have a clinically meaningful impact. The Company has built a product engine that it believes positions itself to be the leading company at the intersection of AAV gene therapy and severe neurological disease. The Company’s product engine enables it to engineer, optimize, manufacture and deliver its AAV-based gene therapies that have the potential to provide durable efficacy following a single administration. Additionally, the Company is working to identify novel AAV capsids, which are the outer viral protein shells that enclose the genetic material of the virus payload. The Company’s team of experts in the fields of AAV gene therapy and neuroscience first identifies and selects severe neurological diseases that are well-suited for treatment using AAV gene therapy. The Company then engineers and optimizes AAV vectors for delivery of the virus payload to the targeted tissue or cells. The Company’s manufacturing process employs an established system that it believes will enable production of high quality AAV vectors at commercial-scale. Finally, the Company leverages established routes of administration and advances in dosing techniques to optimize delivery of its AAV gene therapies to target cells that are critical to the disease of interest either directly to discrete regions of the brain or, more broadly, to the spinal cord region. The Company is devoting substantially all of its efforts to product research and development, market development, and raising capital. The Company is subject to risks common to companies in the biotechnology and gene therapy industry, including but not limited to, risks of failure of preclinical studies and clinical trials, the need to obtain marketing approval for its drug product candidates, the need to successfully commercialize and gain market acceptance of its drug product candidates, dependence on key personnel, protection of proprietary technology, compliance with government regulations, development by competitors of technological innovations, and ability to transition from pilot-scale manufacturing to large-scale production of products The Company has incurred annual net operating losses in every year since inception. As of December 31, 2017, the Company had an accumulated deficit of $160.7 million. The Company has not generated any product revenue and has financed its operations primarily through public offerings and private placements of its equity securities and funding from its collaboration with Sanofi Genzyme . There can be no assurance that the Company will be able to obtain additional debt or equity financing or generate product revenue or revenue from collaborative partners on terms acceptable to the Company, on a timely basis or at all. The failure of the Company to obtain sufficient funds on acceptable terms when needed could have a material adverse effect on the Company’s business, results of operations, and financial condition. |
Summary of significant accounti
Summary of significant accounting policies | 12 Months Ended |
Dec. 31, 2017 | |
Summary of significant accounting policies | |
Summary of significant accounting policies | 2. Summary of significant accounting policies The following is a summary of significant accounting policies followed in the preparation of these financial statements. Basis of presentation The accompanying consolidated financial statements include those of the Company and its subsidiary, Voyager Securities Corporation, after elimination of all intercompany accounts and transactions. The accompanying consolidated financial statements have been prepared in conformity with accounting principles generally accepted in the United States of America (“GAAP”). Public offerings On October 29, 2015, in preparation for the Company’s IPO, the Company’s Board of Directors and stockholders approved a 1-for-4.25 reverse split of the Company’s common stock, which became effective on October 29, 2015. All share and per share amounts in the consolidated financial statements and notes thereto have been retroactively adjusted for all periods presented to give effect to this reverse split, including reclassifying an amount equal to the reduction in par value of common stock to additional paid-in capital. On November 16, 2015, the Company completed the sale of 5,750,000 shares of its common stock in its initial public offering (the “IPO”), at a price to the public of $14.00 per share, resulting in net proceeds to the Company of $72.9 million after deducting underwriting discounts, commissions and offering expenses payable by the Company. Upon the closing of the IPO, all of the Company’s outstanding redeemable convertible preferred stock automatically converted into shares of common stock, resulting in the issuance of an additional 17,647,054 shares of common stock. The significant increase in shares outstanding in November 2015 effected the year-over-year comparability of the Company’s net loss per share calculations. On November 7, 2017, the Company completed the sale of 5,175,000 shares of its common stock in a public offering at a price to the public of $12.00 per share, resulting in net proceeds to the Company of $58.0 million after deducting underwriting discounts, commissions, and offering expenses payable by the Company. Use of Estimates The preparation of consolidated financial statements in conformity with GAAP requires management to make estimates and assumptions that affect the amounts reported in the consolidated financial statements and accompanying notes. On an ongoing basis, the Company’s management evaluates its estimates, which include, but are not limited to, estimates related to revenue recognition, accrued expenses, valuation of the tranche rights, stock‑based compensation expense, income taxes and the fair value of common stock. The Company bases its estimates on historical experience and other market specific or other relevant assumptions that it believes to be reasonable under the circumstances. Actual results may differ from those estimates or assumptions. Fair Value of Financial Instruments ASC Topic 820, Fair Value Measurement (“ASC 820”), establishes a fair value hierarchy for instruments measured at fair value that distinguishes between assumptions based on market data (observable inputs) and the Company’s own assumptions (unobservable inputs). Observable inputs are inputs that market participants would use in pricing the asset or liability based on market data obtained from sources independent of the Company. Unobservable inputs are inputs that reflect the Company’s assumptions about the inputs that market participants would use in pricing the asset or liability, and are developed based on the best information available in the circumstances. ASC 820 identifies fair value as the exchange price, or exit price, representing the amount that would be received to sell an asset or paid to transfer a liability in an orderly transaction between market participants. As a basis for considering market participant assumptions in fair value measurements, ASC 820 establishes a three‑tier fair value hierarchy that distinguishes between the following: · Level 1 —Quoted market prices in active markets for identical assets or liabilities. · Level 2 —Inputs other than Level 1 inputs that are either directly or indirectly observable, such as quoted market prices, interest rates, and yield curves. · Level 3 —Unobservable inputs developed using estimates of assumptions developed by the Company, which reflect those that a market participant would use. To the extent that the valuation is based on models or inputs that are less observable or unobservable in the market, the determination of fair value requires more judgment. Accordingly, the degree of judgment exercised by the Company in determining fair value is greatest for instruments categorized in Level 3. A financial instrument’s level within the fair value hierarchy is based on the lowest level of any input that is significant to the fair value measurement. The carrying amounts reflected in the balance sheets for cash and cash equivalents, prepaid expenses and other current assets, accounts payable and accrued expenses approximate their fair values, due to their short‑term nature. Cash and Cash Equivalents The Company considers all highly liquid investments purchased with original maturities of 90 days or less at acquisition to be cash equivalents. Cash and cash equivalents include cash held in banks and amounts held in money market funds. Marketable Securities The Company classifies marketable debt securities with a remaining maturity of greater than three months when purchased as available‑for‑sale. Marketable debt securities with a remaining maturity date greater than one year and marketable equity securities are classified as non‑current where the Company has the intent and ability to hold these securities for at least the next 12 months. During 2016, the Company invested in a supplier and received common stock and warrants to purchase common stock in that entity. The common stock is considered an available-for-sale marketable equity security and is included in non-current marketable securities, and the warrants are included in non-current assets. All available for sale securities are carried at fair value with the unrealized gains and losses included in other comprehensive income (loss) as a component of stockholders’ equity until realized. Any premium or discount arising at purchase is amortized and/or accreted to interest income and/or expense. Realized gains and losses are determined using the specific identification method and are included in other income (expense). If any adjustment to fair value reflects a decline in value of the investment, the Company considers all available evidence to evaluate the extent to which the decline is “other than temporary” and, if so, recognizes the loss through a charge to the Company’s statement of operations and comprehensive loss. No other than temporary losses have been recognized. Cash, cash equivalents, and marketable securities as of December 31, 2017 and 2016 consist of the following: Amortized Unrealized Unrealized Fair Cost Gains Losses Value (in thousands) As of December 31, 2017 Money market funds included in cash and cash equivalents $ 30,469 $ — $ — $ 30,469 Marketable securities: U.S. Treasury notes 137,560 — 38 137,522 Equity securities 1,220 — 120 1,100 Total marketable securities $ 138,780 $ — $ 158 $ 138,622 Total money market funds and marketable securities $ 169,249 $ — $ 158 $ 169,091 As of December 31, 2016 Money market funds included in cash and cash equivalents $ 36,003 $ — $ — $ 36,003 Marketable securities: U.S. Treasury notes 130,237 2 66 130,173 U.S. Government agency bonds 7,604 — — 7,604 Total debt securities $ 137,841 $ 2 $ 66 $ 137,777 Equity securities 1,220 140 — 1,360 Total marketable securities $ 139,061 $ 142 $ 66 $ 139,137 Total money market funds and marketable securities $ 175,064 $ 142 $ 66 $ 175,140 All of the Company’s marketable debt securities at December 31, 2017 and 2016 have a contractual maturity of one year or less. Restricted Cash At December 31, 2017 and 2016, the Company maintained restricted cash totaling approximately $0.7 million held in the form of money market accounts as collateral for the Company’s facility lease obligation. The balance is included within deposits in other non‑current assets in the accompanying consolidated balance sheets. Property and Equipment Property and equipment consists of laboratory equipment, furniture and office equipment, and leasehold improvements and is stated at cost, less accumulated depreciation. Maintenance and repairs that do not improve or extend the lives of the respective assets are expensed to operations as incurred; while costs of major additions and betterments are capitalized. Depreciation is calculated over the estimated useful lives of the assets using the straight‑line method. Impairment of Long‑Lived Assets The Company evaluates long‑lived assets for potential impairment when events or changes in circumstances indicate the carrying value of the assets may not be recoverable. Recoverability is measured by comparing the book values of the assets to the expected future net undiscounted cash flows that the assets are expected to generate. If such assets are considered to be impaired, the impairment to be recognized is measured by the amount by which the book values of the assets exceed their fair value. The Company has not recognized any impairment losses from inception through December 31, 2017. Revenue Recognition As of December 31, 2017, all of the Company’s revenue is generated exclusively from its collaboration agreement with Sanofi Genzyme Corporation, a Sanofi company (“S anofi Genzyme”) . The Company recognizes revenue in accordance with ASC Topic 605, Revenue Recognition (“ASC 605”). Accordingly, revenue is recognized for each unit of accounting when all of the following criteria are met: · Persuasive evidence of an arrangement exists; · Delivery has occurred or services have been rendered; · The seller’s price to the buyer is fixed or determinable; and · Collectability is reasonably assured. Amounts received prior to satisfying the revenue recognition criteria are recorded as deferred revenue in the Company’s balance sheet. Amounts expected to be recognized as revenue within 12 months following the balance sheet date are classified as deferred revenue, current portion. Amounts not expected to be recognized as revenue within the 12 months following the balance sheet date are included in noncurrent liabilities and classified as deferred revenue, net of current portion. The Company analyzes the multiple element arrangements based on the guidance in ASC Topic 605‑25, Revenue Recognition—Multiple Element Arrangements (“ASC 605‑25”). Pursuant to the guidance in ASC 605‑25, the Company evaluates multiple element arrangements to determine (1) the deliverables included in the arrangement and (2) whether the individual deliverables represent separate units of accounting or whether they must be accounted for as a combined unit of accounting. This evaluation involves subjective determinations and requires management to make judgments about the individual deliverables and whether such deliverables are separate from other aspects of the contractual relationship. Deliverables are considered separate units of accounting provided that: (i) the delivered item(s) has value to the customer on a standalone basis and (ii) if the arrangement includes a general right of return relative to the delivered item(s), delivery or performance of the undelivered item(s) is considered probable and substantially within control of the Company. In assessing whether an item has standalone value, the Company considers factors such as the research, manufacturing and commercialization capabilities of the collaboration partner and the availability of the associated expertise in the general marketplace. In addition, the Company considers whether the collaboration partner can use the other deliverable(s) for their intended purpose without the receipt of the remaining element(s), whether the value of the deliverable is dependent on the undelivered item(s) and whether there are other vendors that can provide the undelivered element(s). The Company’s collaboration agreement does not contain a general right of return relative to any delivered items. Arrangement consideration that is fixed or determinable is allocated among the separate units of accounting using the relative selling price method. Then the applicable revenue recognition criteria in ASC 605 are applied to each of the separate units of accounting in determining the appropriate period and pattern of recognition. The Company determines the selling price of a unit of accounting following the hierarchy of evidence prescribed by ASC 605‑25. Accordingly, the Company determines the estimated selling price for units of accounting within each arrangement using vendor specific objective evidence (“VSOE”) of selling price, if available, third-party evidence (“TPE”) of selling price if VSOE is not available, or best estimate of selling price (“BESP”) if neither VSOE or TPE is available. The Company has only used BESP to estimate the selling price, since it has not had VSOE or TPE of selling price of any units of accounting to date. Determining BESP for a unit of accounting requires significant judgment. In developing the BESP for a unit of accounting, the Company considers applicable market conditions and relevant entity specific factors, including factors that were contemplated in negotiating the agreement with the customer and estimated costs. The Company validates BESP for units of accounting by evaluating whether changes in the key assumptions used to determine the BESP will have a significant effect on the allocation of arrangement consideration between multiple units of accounting. Options are considered substantive if, at the inception of the arrangement, the Company is at risk as to whether the collaboration partner will choose to exercise the option. Factors that the Company considers in evaluating whether an option is substantive include the cost to exercise the option, the overall objective of the arrangement, the benefit the collaborator might obtain from the arrangement without exercising the option and the likelihood the option will be exercised. When an option is considered substantive, the Company does not consider the option or item underlying the option to be a deliverable at the inception of the arrangement and the associated option fees are not included in allocable consideration, assuming the option is not priced at a significant and incremental discount. Conversely, when an option is not considered substantive, the Company would consider the option, including other deliverables contingent upon the exercise of the option, to be a deliverable at the inception of the arrangement and a corresponding amount would be included in allocable arrangement consideration. In addition, if the price of the option includes a significant incremental discount, the option would be included as a deliverable at the inception of the arrangement. The Company recognizes arrangement consideration allocated to each unit of accounting when all of the revenue recognition criteria in ASC 605 are satisfied for that particular unit of accounting. The Company will recognize revenue associated with license options upon exercise of the option, if the underlying license has standalone value from the other deliverables to be provided subsequent to delivery of the license. If the license does not have standalone value, the amounts allocated to the license option will be combined with the related undelivered items as a single unit of accounting. The amounts allocated to the license option in the Sanofi Genzyme agreement will be deferred until the option is exercised. The revenue recognition upon option exercise will be determined based on whether the license has standalone value from the remaining deliverables under the arrangement at the time of exercise. The Company recognizes the amounts associated with research and development services, alliance joint steering committees and development advisory committees ratably over the associated period of performance. If there is no discernible pattern of performance and/or objectively measurable performance measures do not exist, then the Company recognizes revenue under the arrangement on a straight‑line basis over the period the Company is expected to complete its performance obligations. Conversely, if the pattern of performance in which the service is provided to the customer can be determined and objectively measurable performance exist, then the Company recognizes revenue under the arrangement using the proportional performance method. Revenue recognized is limited to the lesser of the cumulative amount of payments received or the cumulative revenue earned determined using the straight line method or proportional performance, as applicable, as of the period end date. At the inception of an arrangement that includes milestone payments, the Company evaluates whether each milestone is substantive and at risk to both parties on the basis of the contingent nature of the milestone. This evaluation includes an assessment of whether: (i) the consideration is commensurate with either the Company’s performance to achieve the milestone or the enhancement of the value of the delivered item(s) as a result of a specific outcome resulting from the Company’s performance to achieve the milestone, (ii) the consideration relates solely to past performance and (iii) the consideration is reasonable relative to all the deliverables and payment terms within the arrangement. The Company evaluates factors such as clinical, regulatory, commercial and other risks that must be overcome to achieve the respective milestone and the level of effort and investment required to achieve the respective milestone in making this assessment. There is considerable judgment involved in determining whether a milestone satisfies all the criteria required to conclude that a milestone is substantive. In accordance with ASC Topic 605‑28, Revenue Recognition—Milestone Method (“ASC 605‑28”) clinical and regulatory milestones that are considered substantive, will be recognized as revenue in their entirety upon successful accomplishment of the milestone, assuming all other revenue recognition criteria are met. Milestones that are not considered substantive would be recognized as revenue over the remaining period of performance, assuming all other revenue recognition criteria are met. Revenue from commercial milestone payments will be accounted for as royalties and recorded as revenue upon achievement of the milestone, assuming all other revenue recognition criteria are met. The Company will recognize royalty revenue in the period of sale of the related product(s), based on the underlying contract terms, provided that the reported sales are reliably measurable and the Company has no remaining performance obligations, assuming all other revenue recognition criteria are met. The Company also considers the impact of potential future payments it makes in its role as a vendor to its customers or collaboration partners and evaluates if these potential future payments could be reductions of revenue from that customer. If the potential future payments to the customer are (i) a separately identifiable benefit and (ii) the fair value of the identifiable benefit can be reasonably estimated, then the payments are accounted for separately from the revenue received from the customer. If however, both of these criteria are not satisfied, then the payments are treated as a reduction of revenue. Research and Development Research and development costs are charged to expense as incurred in performing research and development activities. The costs include employee compensation costs, external research, consultant costs, sponsored research, in‑kind services provided under the Sanofi Genzyme agreement, license fees, process development and facilities costs. Facilities costs primarily include the allocation of rent, utilities and depreciation. Research Contract Costs and Accruals The Company has entered into various research and development contracts with research institutions and other companies. These agreements are generally cancelable. The Company records accruals for estimated ongoing research costs. When evaluating the adequacy of the accrued liabilities, the Company analyzes progress of the studies, including the phase or completion of events, invoices received and contracted costs. Significant judgments and estimates may be made in determining the accrued balances at the end of any reporting period. Actual results could differ from the Company’s estimates. The Company’s historical accrual estimates have not been materially different from the actual costs. Patent Costs The Company expenses patent application and related legal costs as incurred and classifies such costs as general and administrative expenses in the accompanying statements of operations. Stock‑Based Compensation Expense The Company accounts for its stock‑based compensation awards in accordance with ASC Topic 718 Compensation—Stock Compensation (“ASC 718”). ASC 718 requires all stock‑based payments to employees and directors, including grants of restricted stock and stock options, to be recognized as expense in the statements of operations based on their grant date fair values. Grants of restricted stock and stock options to other service providers, referred to as non‑employees, are required to be recognized as expense in the statements of operations based on their vesting date fair values. The Company estimates the fair value of options granted using the Black‑Scholes option pricing model. The Company uses the fair value of its common stock to determine the fair value of restricted stock awards. The Black‑Scholes option pricing model requires inputs based on certain subjective assumptions, including (a) the expected stock price volatility, (b) the calculation of expected term of the award, (c) the risk‑free interest rate and (d) expected dividends. Due to a lack of company-specific historical and implied volatility data, the Company bases the estimate of expected volatility on the historical volatility of a group of similar companies that are publicly traded, blended with the most recent period of historic volatility of its common stock. The historical volatility is calculated based on a period of time commensurate with the expected term assumption. The computation of expected volatility is based on the historical volatility of a representative group of companies with similar characteristics to the Company, including stage of product development and life science industry focus. The Company uses the simplified method as prescribed by the SEC Staff Accounting Bulletin No. 107, Share‑Based Payment , to calculate the expected term for stock options granted to employees as it does not have sufficient historical exercise data to provide a reasonable basis upon which to estimate the expected term. For stock options granted to non‑employees, the Company utilizes the contractual term of the arrangement as the basis for the expected term assumption. The risk‑free interest rate is based on a treasury instrument whose term is consistent with the expected term of the stock options. The expected dividend yield is assumed to be zero as the Company has never paid dividends and has no current plans to pay any dividends on its common stock. The Company expenses the fair value of its stock‑based compensation awards to employees on a straight‑line basis over the associated service period, which is generally the period in which the related services are received. Stock‑based compensation awards to non‑employees are adjusted through stock‑based compensation expense at each reporting period end to reflect the current fair value of such awards and are expensed on a straight‑line basis. The Company records the expense for stock‑based compensation awards subject to performance conditions over the remaining service period when management determines that achievement of the performance condition is probable. Management evaluates when the achievement of a performance condition is probable based on the expected satisfaction of the performance conditions as of the reporting date. Income Taxes Income taxes are recorded in accordance with ASC Topic 740, Income Taxes (“ASC 740”), which provides for deferred taxes using an asset and liability approach. Under this method, deferred tax assets and liabilities are determined based on the difference between the financial reporting and the tax reporting basis of assets and liabilities and are measured using the enacted tax rates and laws that are expected to be in effect when the differences are expected to reverse. The Company provides a valuation allowance against net deferred tax assets unless, based upon the weight of available evidence, it is more likely than not that the deferred tax assets will be realized. The Company accounts for uncertain tax positions in accordance with the provisions of ASC 740. When uncertain tax positions exist, the Company recognizes the tax benefit of tax positions to the extent that the benefit will more likely than not be realized. The determination as to whether the tax benefit will more likely than not be realized is based upon the technical merits of the tax position as well as consideration of the available facts and circumstances. As of December 31, 2017, the Company does not have any significant uncertain tax positions. Comprehensive Loss Comprehensive loss is comprised of net loss and other comprehensive income or loss. Other comprehensive gain or loss consists of unrealized gains or losses on marketable securities. Net Loss Per Share Attributable to Common Stockholders Basic net loss per share attributable to common stockholders is calculated by dividing the net loss attributable to common stockholders by the weighted‑average number of shares of common stock outstanding during the period, without consideration for potentially dilutive securities. Diluted net loss per share attributable to common stockholders is computed by dividing the net loss attributable to common stockholders by the weighted‑average number of shares of common stock and potentially dilutive securities outstanding for the period determined using the treasury‑stock and if‑converted methods. For purposes of the diluted net loss per share attributable to common stockholders calculation, redeemable convertible preferred stock, unvested restricted common stock, and outstanding stock options are considered to be potentially dilutive securities, but are excluded from the calculation of diluted net loss per share attributable to common stockholders because their effect would be anti‑dilutive and therefore, basic and diluted net loss per share attributable to common stockholders were the same for all periods presented. The following table sets forth the outstanding potentially dilutive securities that have been excluded in the calculation of diluted net loss per share attributable to common stockholders because to do so would be anti‑dilutive: As of December 31, 2017 2016 2015 Unvested restricted common stock 557,979 1,167,984 1,818,261 Shares reserved for issuance under stock plans 5,375,431 4,226,265 2,905,458 Total 5,933,410 5,394,249 4,723,719 All of the Company’s outstanding convertible preferred stock automatically converted into shares of common stock as of November 16, 2015, resulting in the issuance by the Company of an additional 17,647,054 shares of common stock. Concentrations of Credit Risk and Off‑Balance Sheet Risk The Company has no financial instruments with off‑balance sheet risk such as foreign exchange contracts, option contracts or other foreign hedging arrangements. Financial instruments that potentially subject the Company to a concentration of credit risk are cash and cash equivalents. The Company’s cash is held in accounts at a financial institution that may exceed federally insured limits. The Company has not experienced any credit losses in such accounts and does not believe it is exposed to any significant credit risk on these funds. Concentration of Suppliers The Company is dependent on a third‑party manufacturer to supply certain products for research and development activities in its programs. In particular, the Company relies on a sole manufacturer to supply it with specific vectors related to the Company’s research and development programs. Segment Information Operating segments are defined as components of an enterprise about which separate discrete information is available for evaluation by the chief operating decision maker in deciding how to allocate resources and assess performance. The Company and the Company’s chief operating decision maker, the Company’s Chief Executive Officer, views the Company’s operations and manages its business as a single operating segment, which is the business of developing and commercializing gene therapies. Recent Accounting Pronouncements In May 2014, the FASB issued ASU No. 2014-09, Revenue from Contracts with Customers (Topic 606) (“ASU 2014-09”). Subsequently, the FASB also issued ASU 2015-14, Revenue from Contracts with Customers (Topic 606), which adjusted the effective date of ASU 2014-09; ASU No. 2016-08, Revenue from Contracts with Customers (Topic 606): Principal versus Agent Considerations (Reporting Revenue Gross versus Net), which amends the principal-versus-agent implementation guidance and illustrations in ASU 2014-09; ASU No. 2016-10, Revenue from Contracts with Customers (Topic 606): Identifying Performance Obligations and Licensing, which clarifies identifying performance obligation and licensing implementation guidance and illustrations in ASU 2014-09; and ASU No. 2016-12, Revenue from Contracts with Customers (Topic 606): Narrow-Scope Improvements and Practical Expedients, which addresses implementation issues and is intended to reduce the cost and complexity of applying the new revenue standard in ASU 2014-09 (collectively, the “Revenue ASUs”). The Revenue ASUs provide an accounting standard for a single comprehensive model for use in accounting for revenue arising from contracts with customers and supersedes most current revenue recognition guidance. The accounting standard is effective for interim and annual periods beginning after December 15, 2017, with an option to early adopt for interim and annual periods beginning after December 15, 2016. The guidance permits two methods of adoption: retrospectively to each prior reporting period presented (the full retrospective method), or retrospectively with the cumulative effect of initially applying the guidance recognized at the date of initial application (the modified retrospective method). As of December 31, 2017, the Company had one revenue generating arrangement, which was the Sanofi Genzyme Collaboration, as defined below. The Company has elected to adopt the Revenue ASUs effective as of January 1, 2018, under the modified retrospective method. The Company has substantially completed its assessment of the effect that this new standard will have on its consolidated financial statements. The most significant effect of the adoption is expected to be the method of revenue recognition for services performed over time. Under the previous accounting standards, revenue was recognized over the estimated period of performance while revenue will be recognized based on a proportional performance model under the Revenue ASUs. This change in accounting policy, along with certain other factors, is expected to result in an adjustment of approximately $18.0 million to $23.0 million, representing an increase of deferred revenue with an offset to accumulated deficit on January 1, 2018 to reflect the cumulative effect of the accounting changes made upon the adoption of the standard. In addition, the Company expects that the changes in accounting for contingent milestone payments will have a significant effect on the future accounting treatment for the arrangement. The previous accounting guidance contained specific guidance related to the accounting for milestone payments, including, if certain criteria were met, the ability to recognize all consideration related to the milestone once that milestone was achieved. The Revenue ASUs do not contain guidance specific to milestone payments, thereby requiring potential milestone payments to be considered in accordance with the overall revenue recognition model. As a result, revenue from contingent milestone payments may be recog |
Fair Value Measurements
Fair Value Measurements | 12 Months Ended |
Dec. 31, 2017 | |
Fair Value Measurements | |
Fair value of financial measurements | 3. Fair value measurements Assets and liabilities measured at fair value on a recurring basis as of December 31, 2017 and 2016 are as follows: Quoted Prices Significant in Active Other Significant Markets for Observable Unobservable Identical Assets Inputs Inputs Assets Total (Level 1) (Level 2) (Level 3) (in thousands) December 31, 2017 Money market funds included in cash and cash equivalents $ 30,469 $ 30,469 $ — $ — Marketable securities: U.S. Treasury notes 137,522 137,522 — — Equity securities 1,100 1,100 — — Total marketable securities $ 138,622 $ 138,622 $ — $ — Warrants to purchase equity securities 569 — 569 — Total $ 169,660 $ 169,091 $ 569 $ — December 31, 2016 Money market funds included in cash and cash equivalents $ 36,003 $ 36,003 $ — $ — Marketable securities: U.S. Treasury notes 130,173 130,173 — — U.S. Government agency securities 7,604 — 7,604 — Equity securities 1,360 1,360 — — Total marketable securities $ 139,137 $ 131,533 $ 7,604 $ — Warrants to purchase equity securities 792 — 792 — Total $ 175,932 $ 167,536 $ 8,396 $ — The Company measures the fair value of money market funds, U.S. Treasuries and equity securities based on quoted prices in active markets for identical securities. The Level 2 debt securities include U.S. Government agency securities that are valued either based on recent trades of securities in inactive markets or based on quoted market prices of similar instruments and other significant inputs derived from or corroborated by observable market data. The Level 2 equity securities include warrants used to purchase equity securities that are valued using the Black-Scholes model. The Black-Scholes option pricing model requires inputs based on certain subjective assumptions, including (a) the expected stock price volatility, (b) the calculation of expected term of the awards, (c) the risk-free interest rate, and (d) expected dividends. The assumptions utilized to value the warrants to purchase equity securities as of December 31, 2017 and 2016 are as follows: As of December 31, 2017 2016 Risk-free interest rate 2.0 % 1.8 % Expected dividend yield — % — % Expected term (in years) 3.7 4.7 Expected volatility 103.5 % 97.5 % The expected volatility is based on the historic volatility for the equity securities underlying the warrants and is calculated based on a period of time commensurate with the expected term assumption. The expected term is based on the remaining contractual life of the warrants on each measurement date. The risk-free interest rate is based on a treasury instrument whose term is consistent with the expected term of the warrants. The expected dividend yield is assumed to be zero as the entity that issued the warrants has never paid and has not indicated any intention to pay dividends. |
Prepaid expenses and other curr
Prepaid expenses and other current assets | 12 Months Ended |
Dec. 31, 2017 | |
Prepaid expense and other current assets | |
Prepaid expenses and other current assets | 4. Prepaid expenses and other current assets Prepaid expenses and other current assets consist of the following: As of December 31, 2017 2016 (in thousands) Prepaid research and development contracts $ 1,330 $ 1,094 Other current assets 766 541 Prepaid insurance 520 430 Accrued interest receivable 122 339 Tenant improvement receivable — 1,964 Total $ 2,738 $ 4,368 |
Property and equipment, net
Property and equipment, net | 12 Months Ended |
Dec. 31, 2017 | |
Property and equipment, net | |
Property and equipment, net | 5. Property and equipment, net Property and equipment, net consists of the following: As of December 31, 2017 2016 (in thousands) Leasehold improvements $ 6,421 $ 1,341 Laboratory equipment 5,262 3,306 Furniture and office equipment 1,565 526 Other 25 242 Construction in progress — 3,873 Total property and equipment 13,273 9,288 Less: accumulated depreciation (2,990) (1,395) Property and equipment, net $ 10,283 $ 7,893 The Company recorded $1.6 million, $0.6 million, and $0.6 million in depreciation expense during the years ended December 31, 2017, 2016, and 2015, respectively. Construction-in-progress as of December 31, 2016 includes $3.0 million related to costs which were reimbursable by the landlord. Refer to Note 7 “Commitments and contingencies” for further details. |
Accrued expenses
Accrued expenses | 12 Months Ended |
Dec. 31, 2017 | |
Accrued expenses | |
Accrued expenses | 6. Accrued expenses Accrued expenses consist of the following: As of December 31, 2017 2016 (in thousands) Research and development costs $ 5,780 $ 2,384 Employee compensation costs 3,383 2,399 Professional services 1,762 698 Accrued goods and services 388 842 Patent costs 120 89 Other 64 76 Total $ 11,497 $ 6,488 |
Commitments and contingencies
Commitments and contingencies | 12 Months Ended |
Dec. 31, 2017 | |
Commitments and Contingencies | |
Commitments and contingencies | 7. Commitments and contingencies Operating Leases During March 2014, the Company entered into an agreement to lease the 75 Sidney Street facility under a non‑cancelable operating lease that would expire December 15, 2019. The lease includes two renewal options, each for five-year terms and at fair market value upon exercise. The lease contains escalating rent clauses which require higher rent payments in future years. The Company expenses rent on a straight‑line basis over the term of the lease, including any rent‑free periods. In January 2016, the Company executed an amendment to extend the 75 Sidney Street lease and executed an agreement to lease the 64 Sidney Street facility until December 31, 2024. The additional facility includes laboratory and office space, and was ready for occupancy in early 2017. In February 2018, the Company executed an amendment to lease an additional approximately 11,000 square feet at 75 Sidney Street that is intended to support its continued growth. The additional facility includes laboratory and office space, and will be ready for occupancy in the first half of 2018. The table below includes estimated payments related to the amended 75 Sidney Street lease and the lease for 64 Sidney Street as of December 31, 2017 and does not include the effect of the amendment executed in February 2018. The Company has received leasehold improvement incentives from the landlord totaling $1.3 million and $3.5 million for 75 Sidney Street and 64 Sidney Street, respectively, as of December 31, 2017. The Company recorded these incentives as a component of deferred rent and is amortizing these incentives as a reduction of rent expense over the life of the lease. The leasehold improvements have been recorded as fixed assets. The Company is entitled to receive approximately $0.3 million of leasehold improvements for the space related to the amendment of the lease executed in February 2018. Rent expense of approximately $2.9 million, $2.0 million, and $0.9 million was incurred during the years ended December 31, 2017, 2016, and 2015, respectively. Future annual minimum lease payments at December 31, 2017 are as follows: Total Minimum Lease Payments (in thousands) 2018 3,290 2019 3,382 2020 3,762 2021 3,868 2022 3,977 2023+ 8,295 $ 26,574 . Significant Agreements Sanofi Genzyme Collaboration Agreement Summary of Agreement In February 2015, the Company entered into an agreement with Sanofi Genzyme (the “Sanofi Genzyme Collaboration Agreement”) which included a non‑refundable upfront payment of $65.0 million. In addition, contemporaneous with entering into the Sanofi Genzyme Collaboration Agreement, Sanofi Genzyme entered into a Series B Stock Purchase Agreement, under which Sanofi Genzyme purchased 10,000,000 shares of Series B Preferred Stock for $30.0 million. The fair value of the Series B Preferred Stock at the time of issuance was approximately $25.0 million. The $5.0 million premium over the fair value is accounted for as additional consideration under the Sanofi Genzyme Collaboration Agreement. Under the Sanofi Genzyme Collaboration Agreement, the Company granted Sanofi Genzyme an exclusive option to license, develop and commercialize (i) ex‑U.S. rights to the following programs, which are referred to as Split Territory Programs; VY‑AADC (“Parkinson’s Program”), VY‑FXN01 (“Friedreich’s Ataxia Program”), a future program to be designated by Sanofi Genzyme (“ Future Program), and VY‑HTT01 (“Huntington’s Program”), with an incremental option to co‑commercialize VY‑HTT01 in the United States and (ii) worldwide rights to VY‑SMN101 (“Spinal Muscular Atrophy Program”). Sanofi Genzyme ’s option for the Split Territory Programs and the Spinal Muscular Atrophy Program is triggered following the completion of the first proof‑of‑principle human clinical study (“POP Study”), on a program by program basis. Prior to any option exercise by Sanofi Genzyme , the Company will collaborate with Sanofi Genzyme in the development of products under each Split Territory Program and the Spinal Muscular Atrophy Program pursuant to a written development plan and under the guidance of an Alliance Joint Steering Committee (“AJSC”), comprised of an equal number of employees from the Company and Sanofi Genzyme . The Company is required to use commercially reasonable efforts to develop products under each Split Territory Program and the Spinal Muscular Atrophy Program through the completion of the applicable POP Study. During the development of these joint programs, the activities are guided by a Development Advisory Committee (“DAC”). The DAC may elect to utilize certain Sanofi Genzyme technology relating to the Parkinson’s Program, the Huntington’s Program or generally with the manufacture of Split Territory Program products. The Company is solely responsible for all costs incurred in connection with the development of the Split Territory Programs and the Spinal Muscular Atrophy Program products prior to the exercise of an option by Sanofi Genzyme with the exception of the following: (i) at the Company’s request and upon mutual agreement, Sanofi Genzyme will provide “in‑kind” services valued at up to $5.0 million and (ii) Sanofi Genzyme shall be responsible for the costs and expenses of activities under the Huntington’s Program development plan to the extent such activities are covered by financial support Sanofi Genzyme is entitled to receive from a patient advocacy group, collectively Sanofi Genzyme “in‑kind” and other funding. Other than the Parkinson’s Program (for which a POP Study has already been completed), if the Company does not initiate a POP Study for a given Split Territory Program by December 31, 2026 (or for the Future Program by the tenth anniversary of the date the Future Program is nominated by Sanofi Genzyme ), and Sanofi Genzyme has not terminated the Sanofi Genzyme Collaboration Agreement with respect to the collaboration program, then Sanofi Genzyme shall be entitled, as its sole and exclusive remedy, to a credit of $10.0 million for each such program against other milestone or royalty payments payable by Sanofi Genzyme under the Sanofi Genzyme Collaboration Agreement. However, if the POP Study is not initiated due to a regulatory delay or a force majeure event, such time period shall be extended for so long as such delay continues. With the exception of the Parkinson’s Program, Sanofi Genzyme is required to pay an option exercise payment of $20.0 million or $30.0 million for each Split Territory Program, as well as the Spinal Muscular Atrophy Program. Upon Sanofi Genzyme ’s exercise of its option to license a given product in a Split Territory Program (“Split Territory Licensed Product”), the Company will have sole responsibility for the development of such Split Territory Licensed Product in the United States and Sanofi Genzyme shall have sole responsibility for development of such Split Territory Licensed Product in the rest of the world. The Company and Sanofi Genzyme will have shared responsibility for execution of ongoing development of such Split Territory Licensed Product that is not specific to either territory, including costs associated therewith. The Company is responsible for all commercialization activities relating to Split Territory Licensed Products in the United States, including all of the associated costs. Sanofi Genzyme is responsible for all commercialization activities relating to the Split Territory Licensed Products in the rest of the world, including all of the associated costs. If Sanofi Genzyme exercised its co‑commercialization rights, Sanofi Genzyme will be the lead party responsible for all commercialization activities related to Huntington’s Licensed Product in the United States. Upon exercise of the option, Sanofi Genzyme shall have the sole right to develop the Spinal Muscular Atrophy Product worldwide. Sanofi Genzyme shall be responsible for all of the development costs that occur after the option exercise date for the Spinal Muscular Atrophy Program. Sanofi Genzyme is also responsible for commercialization activities relating to the Spinal Muscular Atrophy Product worldwide. Sanofi Genzyme is required to pay the Company for specified regulatory and commercial milestones, if achieved, up to $540.0 million across all programs. The Company is no longer entitled to receive a total of $105.0 million related to regulatory and commercial milestone payments for VY-AADC as a result of Sanofi Genzyme’s decision to not exercise its option for the Parkinson’s Program (the “PD Opt-Out”). The regulatory approval milestones are payable upon either regulatory approval in the United States or regulatory and reimbursement approval in the European Union and range from $40.0 million to $50.0 million per milestone, with an aggregate total of $220.0 million, after accounting for the PD Opt-Out. The commercial milestones are payable upon achievement of specified annual net sales in each program and range from $50.0 million to $100.0 million per milestone, with an aggregate total of $320.0 million, after accounting for the PD Opt-Out . In addition, to the extent any Split Territory Licensed Products or the Spinal Muscular Atrophy Licensed Product are commercialized, the Company is entitled to tiered royalty payments ranging from the mid‑single digits to mid‑teens based on a percentage of net sales by Sanofi Genzyme . Sanofi Genzyme is entitled to receive tiered royalty payments related to sales of Split Territory Licensed Product ranging from the low‑single digits to mid‑single digits based on a percentage of net sales by the Company depending on whether the Company uses Sanofi Genzyme technology in the Split Territory Licensed Product. If Sanofi Genzyme elects to co‑commercialize VY‑HTT01 in the United States, the Company and Sanofi Genzyme will share in any profits or losses from VY‑HTT01 product sales. The Sanofi Genzyme Collaboration Agreement will continue in effect until the later of (i) the expiration of the last to expire of the option rights and (ii) the expiration of all payment obligations unless sooner terminated by the Company or Sanofi Genzyme . The Company and Sanofi Genzyme have customary termination rights including the right to terminate for an uncured material breach of the agreement committed by the other party and Sanofi Genzyme has the right to terminate for convenience. Accounting Analysis The Sanofi Genzyme Collaboration Agreement includes the following deliverables: (i) research and development services for each of the Split Territory License Programs and the Spinal Muscular Atrophy Program, (ii) participation in the AJSC, (iii) participation in the DAC and (iv) the option to obtain a development and commercial license in the Parkinson’s Program and related deliverables. The Company has determined that the option to obtain a development and commercial license in the Parkinson’s program is not a substantive option for accounting purposes, primarily because there is no additional option exercise payment payable by Sanofi Genzyme at the time the option is exercised. Therefore, the option to obtain a license and other obligations of the Company that are contingent upon exercise of the option are considered deliverables at the inception of the arrangement. The options in the other Split Territory Programs and the Spinal Muscular Atrophy Program are considered substantive as there is substantial option exercise payments payable by Sanofi Genzyme upon exercise. In addition, as a result of the uncertainties related to the discovery, research, development and commercialization activities, the Company is at risk with regard to whether Sanofi Genzyme will exercise the options. Moreover, the substantive options are not priced at a significant incremental discount. Accordingly, the substantive options are not considered deliverables at the inception of the arrangement and the associated option exercise payments are not included in allocable arrangement consideration. The Company has also determined that any obligations which are contingent upon the exercise of a substantive option are not considered deliverables at the outset of the arrangement, as these deliverables are contingent upon the exercise of the options. In addition, any option exercise payments associated with the substantive options are not included in the allocable arrangement consideration. The Company has concluded that each of the deliverables identified at the inception of the arrangement has standalone value from the other undelivered elements. Additionally, the Sanofi Genzyme Collaboration Agreement does not include return rights related to the initial collaboration term. Accordingly, each deliverable qualifies as a separate unit of accounting. The Company has identified $79.3 million of allocable arrangement consideration consisting of the $65.0 million upfront fee, the $5.0 million premium paid in excess of fair value of the Series B Preferred Stock and $9.3 million of Sanofi Genzyme “in‑kind” and other funding. The Company has allocated the allocable arrangement consideration based on the relative selling price of each unit of accounting. For all units of accounting, the Company determined the selling price using the BESP. The Company determined the BESP for the service related deliverable for the research and development activities based on internal estimates of the costs to perform the services, including expected internal expenses and expenses with third parties for services and supplies, marked up to include a reasonable profit margin and adjusted for the scope of the potential license. Significant inputs used to determine the total expense of the research and development activities include, the length of time required and the number and costs of various studies that will be performed to complete the POP Study. The BESP for the AJSC and DAC have been estimated based on the costs incurred to participate in the committees, marked up to include a reasonable profit margin. The BESP for the license option was determined based on the estimated value of the license and related deliverables adjusted for the estimated probability that the option would be exercised by Sanofi Genzyme . Based on the relative selling price allocation, the allocable arrangement consideration was allocated as follows: Unit of Accounting Amount (in thousands) Research and Development Services for: Huntington’s Program $ 15,662 Parkinson’s Program 6,648 Friedreich’s Ataxia Program 16,315 Spinal Muscular Atrophy Program 32,050 Future Program 2,464 Committee Obligations: AJSC 147 DAC 227 License Option and related deliverables 5,743 Total $ 79,256 The Company recognizes the amounts associated with research and development services on a straight-line basis over the period of service as there is no discernable pattern or objective measure of performance for the services. Similarly, the Company recognizes the amount associated with the committee obligations on a straight-line basis over the period of service consistent with the expected pattern of performance. The amount allocated to the license option was deferred until October 2017, when Sanofi Genzyme decided that it would not exercise its option to the Parkinson’s Program (the “PD Option”). In October 2017, Sanofi Genzyme decided not to exercise the PD Option. Therefore, in the year ended December 31, 2017, the Company recognized revenue of $5.5 million of consideration which had been allocated to the PD Option. In addition, revenue recognized during the years ended December 31, 2017 and 2016 include amounts recognized related to consideration allocated to research and development services for various programs under the Sanofi Genzyme Collaboration Agreement. During 2017 the Company reassessed the estimated period of performance for each of the units of accounting and determined that the estimated period would be extended for two units of accounting. During 2016 the Company deprioritized the development of VY-SMN101 for the treatment of Spinal Muscular Atrophy. As a result, the Company ceased recognizing the revenue allocated to this program. These adjustments were made on a prospective basis and resulted in decreases in revenue recognized by $2.1 million and $9.5 million, respectively, for the year ended December 31, 2017. The Company has evaluated all of the milestones that may be received in connection with the Split Territory Licensed Product and the Spinal Muscular Atrophy Program Licensed Product. In evaluating if a milestone is substantive, the Company assesses whether: (i) the consideration is commensurate with either the Company’s performance to achieve the milestone or the enhancement of the value of the delivered item(s) as a result of a specific outcome resulting from the Company performance to achieve the milestone, (ii) the consideration relates solely to past performance and (iii) the consideration is reasonable relative to all of the deliverables and payment terms within the arrangement. All regulatory milestones are considered substantive on the basis of the contingent nature of the milestone, specifically reviewing factors such as the scientific, clinical, regulatory, and other risks that must be overcome to achieve the milestone as well as the level of effort and investment required. Accordingly, such amounts will be recognized as revenue in full in the period in which the associated milestone is achieved, assuming all other revenue recognition criteria are met. All commercial milestones will be accounted for in the same manner as royalties and recorded as revenue upon achievement of the milestone, assuming all other revenue recognition criteria are met. During the years ended December 31, 2017, 2016, and 2015, the Company recognized $10.1 million, $14.2 million, and $17.3 million, respectively, of revenue associated with its collaboration with Sanofi Genzyme related to research and development services performed during the period and for consideration allocated to the PD Option, which was recognized during 2017. As of December 31, 2017, there is $31.6 million of deferred revenue related to the Sanofi Genzyme Collaboration Agreement, which is classified as either current or noncurrent in the accompanying balance sheet based on the period the services are expected to be delivered. Costs incurred relating to the programs that Sanofi Genzyme has the option to license under the Sanofi Genzyme Collaboration Agreement consist of internal and external research and development costs, which primarily include: salaries and benefits, lab supplies and preclinical research studies. All costs are included in research and development expenses in the Company’s statement of operations during the year ended December 31, 2017. The Company estimates that the majority of research and development expense during the period relate to programs for which Sanofi Genzyme had or has an option right. MRI Interventions License and Securities Purchase Agreements Summary of Agreement In September 2016, the Company entered into securities purchase and license agreements with MRI Interventions, Inc. (“MRIC”). MRIC is the primary supplier of the ClearPoint System, which is being used by the Company in ongoing development and clinical trials. The Company paid $2.0 million for shares of MRIC common stock and a warrant to purchase additional shares of MRIC common stock under a securities purchase agreement. The license agreement provided for certain rights to MRIC technology and for MRIC to transfer the rights and know-how to manufacture the ClearPoint System to enable the Company to utilize an alternative supplier for the ClearPoint System for use in the Company’s development and clinical trials. Accounting Analysis During the three months ended March 31, 2017, the Company terminated the license agreement with MRIC, and all prior and future commitments and obligations under such agreement became null and void. As of December 31, 2017, the Company continued to hold the common stock and warrants to purchase additional shares of common stock as an available-for-sale security and non-current asset, respectively. Other Agreements During 2017, 2016, and 2015, the Company entered into various agreements with contract research organizations and institutions to license intellectual property. In consideration for the licensed rights the Company generally made upfront payments, which were recorded as research and development expense as the acquired technologies were considered in‑process research and development. During the years ended December 31, 2017, 2016, and 2015, the Company paid $0.3 million, $0.6 million, and $0.1 million, respectively, in upfront license fees. The license agreements also obligate the Company to make additional payments that are contingent upon specific clinical trial and regulatory approval milestones being achieved as well as royalties on future product sales. The agreements to license intellectual property include potential milestone payments that are dependent upon the development of products licensed under the agreements and contingent upon the achievement of clinical trial or regulatory approval milestones. The maximum aggregate potential milestone payments payable by the Company total approximately $18.0 million. Additionally, under the terms of one agreement, the Company has options to license intellectual property to be used in the development of therapies for four additional disease indications. If the Company exercises all of the options under the agreement, it would be obligated to pay aggregate upfront fees of up to approximately $1.6 million and milestone payments that are contingent upon clinical trial results and regulatory approval of $5.0 million per disease indication, or up to $20.0 million in total. As of December 31, 2017 and 2016, there have been no milestones achieved. The Company can generally terminate the license agreements upon 30‑90 days prior written notice. Additionally, certain license agreements require the Company to reimburse the licensor for certain past and ongoing patent related expenses. During the year ended December 31, 2017, 2016, and 2015, the Company incurred $0.8 million, $1.8 million, and $0.3 million of expense, respectively, related to these reimbursable patent costs which are recorded as general and administrative expense During September 2016, the Company entered into a research and development funding arrangement with a non-profit organization that provides up to $4.0 million in funding to the Company upon the achievement of clinical and development milestones. The agreement provides that the Company repay amounts received under certain circumstances including termination of the agreement, and to pay an amount up to 2.6 times the funding received upon successful development and commercialization of any products developed. During 2017, the Company earned a milestone payment of $1.0 million. The Company has evaluated the arrangement and has concluded that it represents a research and development financing arrangement as it is probable that the Company will repay amounts received under the arrangement. As a result, the $1.0 million earned through 2017 is recorded as a non-current liability in the consolidated balance sheet. Litigation The Company is not a party to any litigation and does not have contingency reserves established for any litigation liabilities as of December 31, 2017 or December 31, 2016. |
Redeemable Convertible Preferre
Redeemable Convertible Preferred Stock | 12 Months Ended |
Dec. 31, 2017 | |
Redeemable Convertible Preferred Stock Disclosure [Abstract] | |
Redeemable Convertible Preferred Stock [Text Block] | 8. Redeemable convertible preferred stock In November 2015, upon the closing of the Company’s IPO, all issued and outstanding redeemable convertible preferred stock was automatically converted into 17,647,054 shares of common stock, see Note 2. The Company’s redeemable convertible preferred stock (“Preferred Stock”), has been classified as temporary equity on the accompanying balance sheets instead of in stockholders’ deficit in accordance with authoritative guidance for the classification and measurement of redeemable securities as the redeemable convertible preferred stock is redeemable at the option of the holder after the redemption date, February 2021. The Company has authorized preferred stock amounting to 5,000,000 shares as of December 31, 2017 and 2016. The authorized preferred stock was classified under stockholders’ equity at December 31, 2017. Series A Preferred Stock The Company issued 45,000,000 shares of Series A Preferred Stock during 2014 and 2015. These shares were issued at various closings in 2014 and 2015 for $1.00 per share. The shares were issued in exchange for cash proceeds of $42.0 million, net of issuance costs of $32.0 thousand, and the exchange of outstanding redeemable Convertible Notes, including accrued interest, of approximately $2.9 million. Tranche Rights Issued with Series A Preferred Stock Included in the terms of the January 2014 Series A Preferred Stock Purchase Agreement were certain rights (“Tranche Rights”), granted to the investors of Series A Preferred Stock purchased in January 2014, including the holders of the redeemable Convertible Notes who exchanged the redeemable Convertible Notes. The Tranche Rights obligated the investors in Series A Preferred Stock to purchase and the Company to sell an additional 18,500,000 shares of Series A Preferred Stock at $1.00 per share contingent upon successful near term in licensing and progress on initial experiments and research and development planning (“Tranche Right I”). In addition, the investors were obligated to purchase, and the Company was obligated to sell an additional 20,000,000 shares of Series A Preferred Stock upon the development of project engine and achievement of certain clinical milestones (“Tranche Right II”). In addition, the Tranche Rights allowed the investors the ability to purchase the additional shares at their option at any time. The Tranche Rights were transferrable by the investors, subject to approval by the Board. The Company has concluded the Tranche Rights meet the definition of a freestanding financial instrument, as the Tranche Rights were legally detachable and separately exercisable from the Series A Preferred Stock. Therefore, the Company allocated the proceeds between the Tranche Rights and the Series A Preferred Stock. As the Series A Preferred Stock was redeemable at the holder’s option, the Tranche Rights were classified as an asset or liability and were initially recorded at fair value. The Tranche Rights were measured at fair value at each reporting period. Since the Tranche Rights were subject to fair value accounting, the Company allocated the proceeds to the Tranche Rights based on the fair value at the date of issuance with the remaining proceeds being allocated to the Series A Preferred Stock. The estimated fair value of the Tranche Rights was determined using a probability weighted present value model that considers the probability of closing a tranche, the estimated future value of Series A Preferred Stock each closing, and the investment required at each closing. Future values were converted to present value using a discount rate appropriate for probability adjusted cash flows. Tranche Right I was initially recorded as an asset of $1.5 million as the purchase price of the additional shares was greater than the estimated value of the Series A Preferred Stock at the expected settlement date. The Company issued 18,500,000 additional shares under Tranche Right I, in three separate closings during the year ended December 31, 2014 with total proceeds of $18.5 million, net of issuance costs. Prior to each closing, any change in the value of Tranche Right I was recorded as other financing expense. The fair value of the portion of the Tranche Right I settled at each closing was reclassified to Series A Preferred Stock. Tranche Right II was initially recorded as a liability of $4.1 million as the purchase price of the additional shares was less than the estimated price of the Series A Preferred Stock at the expected settlement date. In February 2015, Tranche Right II was settled when the Company closed the final issuance of Series A Preferred Stock. The Company recognized expense of $9.8 million related to the mark to market of Tranche Right II during the year ended December 31, 2015, which was included in other financing expense. The fair value of the Tranche Right II settled at closing was reclassified to Series A Preferred Stock. The initial carrying amount of the Series A Preferred Stock issued upon the closing of Tranche Right II amounted to approximately $36.1 million which exceeded the redemption value, therefore the carrying value was not subsequently adjusted. However, the Company reflected accrued dividends of approximately $1.2 million related to this issuance in the net loss attributable to common stockholders for the year ended December 31, 2015. Series B Preferred Stock The Company issued 30,000,001 shares of Series B Preferred Stock during 2015. These shares were issued for $3.00 per share. This issuance resulted in cash proceeds of $89.8 million, net of issuance costs of $0.2 million. Additionally, a discount of $5.0 million was recorded against the proceeds as the amount paid by Sanofi Genzyme was in excess of fair value of the Series B Preferred Stock at issuance. |
Common Stock
Common Stock | 12 Months Ended |
Dec. 31, 2017 | |
Common stock Abstract | |
Stockholders' Equity Note Disclosure [Text Block] | 9. Common stock As of December 31, 2017 and 2016, the Company had authorized 120,000,000 shares of Common Stock, at $0.001 par value per share. General The voting, dividend and liquidation rights of the holders of the Common Stock are subject to and qualified by the rights, powers and preferences of the holders of Preferred Stock. The Common Stock has the following characteristics: Voting The holders of shares of Common Stock are entitled to one vote for each share of Common Stock held at all meetings of stockholders and written actions in lieu of meetings. Dividends The holders of shares of Common Stock are entitled to receive dividends, if and when declared by the Board of Directors. No dividends have been declared or paid by the Company since its inception. Liquidation The holders of shares of Common Stock are entitled to share ratably in the Company’s remaining assets available for distribution to its stockholders in the event of any voluntary or involuntary liquidation, dissolution or winding up of the Company or upon occurrence of a deemed liquidation event. Shares Reserved For Future Issuance As of December 31, 2017 2016 Shares reserved for vesting of restricted stock awards under the Founder Agreements 366,914 628,679 Shares reserved for vesting of restricted stock awards under the 2014 Option and Stock Plan 191,065 539,305 Shares reserved for exercise of stock options 3,143,566 1,871,237 Shares reserved for issuances under the 2015 Stock Option Plan 1,501,005 1,825,174 Shares reserved for issuances under the 2015 Employee Stock Purchase Plan 730,860 529,854 5,933,410 5,394,249 |
Stock-based compensation
Stock-based compensation | 12 Months Ended |
Dec. 31, 2017 | |
Stock based compensation | |
Disclosure of Compensation Related Costs, Share-based Payments [Text Block] | 10. Stock‑based compensation 2015 Stock Option Plan In October 2015, the Company ’ s board of directors and stockholders approved the 2015 Stock Option and Incentive Plan “(2015 Stock Option Plan”), which became effective upon the completion of the IPO. The 2015 Stock Option Plan provides the Company with the flexibility to use various equity-based incentive and other awards as compensation tools to motivate its workforce. These tools include stock options, stock appreciation rights, restricted stock, restricted stock units, unrestricted stock, performance share awards and cash-based awards. The 2015 Stock Option Plan replaced the 2014 Plan (as defined below). Any options or awards outstanding under the 2014 Plan remained outstanding and effective. The number of shares initially reserved for issuance under the 2015 Stock Option Plan is the sum of (i) 1,311,812 shares of common stock and (ii) the number of shares under the 2014 Plan that are not needed to fulfill the Company ’ s obligations for awards issued under the 2014 Plan as a result of forfeiture, expiration, cancellation, termination or net issuances of awards thereunder. The number of shares of common stock that may be issued under the 2015 Stock Option Plan is also subject to increase on the first day of each fiscal year by up to 4% of the Company ’ s issued and outstanding shares of common stock on the immediately preceding December 31. Effective January 1, 2016, 2017, and 2018 an additional 1,069,971, 1,070,635, and 1,285,200 shares, respectively, were added to the Company’s 2015 Stock Option Plan for future issuance. During the year ended December 31, 2017, the Company issued 1,700,000 stock options to employees and directors and 20,000 stock options to non‑employees. As of December 31, 2017, there were 1,501,005 shares available for future issuance under the 2015 Stock Option Plan. 2014 Stock Option and Grant Plan In January 2014, the Company adopted the 2014 Stock Option and Grant Plan (the “2014 Plan”), under which it could grant incentive stock options, non‑qualified stock options, restricted stock awards, unrestricted stock awards, or restricted stock units to purchase up to 823,529 shares of Common Stock to employees, officers, directors and consultants of the Company. In April 2014, the Company amended the Plan to allow for the issuance of up to 1,411,764 shares of Common Stock. In August 2014, April 2015, August 2015 and October 2015 the Company further amended the Plan to allow for the issuance of up to 2,000,000, 2,047,058, 2,669,411 and 2,998,823 shares of Common Stock, respectively. During 2014 the Company issued only restricted stock awards under the Plan and during 2015 the Company only granted stock options. The terms of stock awards agreements, including vesting requirements, were determined by the Board of Directors and were subject to the provisions of the 2014 Plan. Restricted stock awards granted by the Company generally vest based on each grantee’s continued service with the Company during a specified period following grant. Awards granted to employees generally vest over four years, with 25% vesting on the one year anniversary and 75% vesting ratably, on a monthly basis, over the remaining three years. Awards granted to non‑employee consultants generally vest monthly over a period of one to four years. Founder Awards In January 2014, the Company issued 1,188,233 shares of restricted stock to its Founders at an original issuance price of $0.0425 per share. Of the total restricted shares awarded to the Founders, 835,292 shares generally vest over one to four years, based on each Founder’s continued service to the Company in varying capacity as a Scientific Advisory Board member, consultant, director, officer or employee, as set forth in each grantee’s individual restricted stock purchase agreement. The remaining 352,941 of the shares issued will begin vesting upon the achievement of certain performance objectives as well as continued service to the Company, as set forth in the agreements. These performance conditions are tied to certain milestone events specific to the Company’s corporate goals, including but not limited to preclinical and clinical development milestones related to the Company’s product candidates. Stock‑based compensation expense associated with these performance‑based awards will be recognized when the achievement of the performance condition is considered probable, using management’s best estimates. Management concluded that the achievement of the performance milestone for one of the three performance-based awards had been met during 2016. Accordingly, stock‑based compensation expense in the amount of $1.4 million and $1.1 million was recorded in the years ended December 31, 2017 and 2016, respectively. No stock-based compensation expense was recorded related to the performance-based awards in the year ended December 31, 2015. 2015 Employee Stock Purchase Plan In October 2015, the Company’s board of directors and stockholders approved the 2015 Employee Stock Purchase Plan (the “2015 ESPP”). Under the 2015 ESPP, all full-time employees of the Company are eligible to purchase common stock of the Company twice per year, at the end of each six-month payment period. During each payment period, eligible employees who so elect, may authorize payroll deductions in an amount of 1% to 10% (whole percentages only) of the employee’s base pay for each payroll period. At the end of each payment period, the accumulated deductions are used to purchase shares of common stock from the Company at a discount. A total of 262,362 shares of common stock were initially authorized for issuance under this plan. The 2015 ESPP became effective upon the completion of the IPO. Effective January 1, 2016, 2017, and 2018, a total of 267,492, 267,658, and 321,300 shares of common stock, respectively, were added to the 2015 ESPP . The Company issued 66,652 shares of common stock under the 2015 ESPP in the year ended December 31, 2017. Stock-based Compensation Expense Total compensation cost recognized for all stock-based compensation awards in the statements of operations and comprehensive loss is as follows: Year ended December 31, 2017 2016 2015 (in thousands) Research and development $ 5,367 $ 4,296 $ 3,218 General and administrative 3,871 2,014 809 Total stock-compensation expense $ 9,238 $ 6,310 $ 4,027 Restricted Stock A summary of the status of and changes in unvested restricted stock was as follows: Weighted Average Grant Date Fair Value Shares Per Share Unvested restricted common stock as of December 31, 2016 1,167,984 $ 0.76 Issued — Vested (573,803) $ 0.77 Repurchased (36,202) $ 0.67 Unvested restricted common stock as of December 31, 2017 557,979 $ 0.70 The expense related to awards granted to employees and non‑employees was $0.5 million and $2.7 million, respectively, for the year ended December 31, 2017. The expense related to awards granted to employees and non-employees was $0.5 million and $2.6 million, respectively, for the year ended December 31, 2016. The expense related to awards granted to employees and non-employees was $0.5 million and $2.6 million, respectively, for the year ended December 31, 2015. As of December 31, 2017, the Company had unrecognized stock‑based compensation expense related to its unvested restricted stock awards of $4.5 million, which is expected to be recognized over the remaining weighted average vesting period of 0.38 years. Stock Options A summary of the status of, and changes in, stock options was as follows: Weighted Remaining Aggregate Average Contractual Intrinsic Exercise Life Value Shares Price (in years) (in thousands) Outstanding at December 31, 2016 1,871,237 $ 10.21 Granted 1,720,000 $ 13.07 Exercised (158,677) $ 8.59 Cancelled or forfeited (288,994) $ 10.50 Outstanding at December 31, 2017 3,143,566 $ 11.82 8.6 $ 15,991 Exercisable at December 31, 2017 955,587 $ 10.45 8.1 $ 5,933 Vested and expected to vest at December 31, 2017 3,143,566 $ 11.82 8.6 $ 15,991 Using the Black‑Scholes option pricing model, the weighted average fair value of options granted to employees and directors during the year ended December 31, 2017 was $8.48. The stock-based compensation expense related to stock option awards granted to employees and directors was $5.5 million, $3.0 million, and $0.7 million for the years ended December 31, 2017, 2016, and 2015, respectively. The fair value of each option issued to employees and directors was estimated at the date of grant using the Black‑Scholes option pricing model with the following weighted‑average assumptions: Year ended December 31, 2017 2016 2015 Risk-free interest rate 2.0 % 1.5 % 1.6 % Expected dividend yield — % — % — % Expected term (in years) 6.0 6.0 6.0 Expected volatility 73.7 % 73.1 % 78.6 % Using the Black‑Scholes option pricing model, the weighted average grant date fair value of options granted to non‑employees during the year ended December 31, 2017 was $6.88. Unvested options granted to non‑employees are revalued at each measurement period until they vest. The expense related to stock option awards granted to non‑employees was $0.4 million, $0.2 million, and $0.3 million for the years ended December 31, 2017, 2016, and 2015, respectively. As of December 31, 2017, the Company had unrecognized stock‑based compensation expense related to its unvested stock options of $16.1 million which is expected to be recognized over the remaining weighted average vesting period of 2.66 years. The fair value of each option issued to non‑employees was estimated at each vesting and reporting date using the Black‑Scholes option pricing model. The reporting date fair value was determined using the following weighted‑average assumptions: As of December 31, 2017 2016 2015 Risk-free interest rate 2.4 % 2.1 % 2.0 % Expected dividend yield — % — % — % Expected term (in years) 8.5 9.1 10.0 Expected volatility 76.2 % 83.3 % 84.0 % |
401(k) Savings Plan
401(k) Savings Plan | 12 Months Ended |
Dec. 31, 2017 | |
401(k) Savings Plan | |
401(k) Savings Plan | 11. 401(k) Savings plan The Company has a defined‑contribution savings plan under Section 401(k) of the Internal Revenue 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. The Company expensed approximately $0.5 million and $0.3 million related to employer contributions made during the years ended December 31, 2017 and 2016, respectively. The Company did not make any contributions to the 401(k) Plan during 2015. |
Income taxes
Income taxes | 12 Months Ended |
Dec. 31, 2017 | |
Income taxes | |
Income taxes | 12. Income taxes The Company recognizes deferred tax assets and liabilities for the expected future tax consequences of events that have been recognized in the Company’s financial statements or tax returns. Under this method, deferred tax assets and liabilities are determined based on differences between the financial statement carrying amounts and the tax bases of the assets and liabilities using the enacted tax rates in effect in the years in which the differences are expected to reverse. A valuation allowance against deferred tax assets is recorded if, based on the weight of the available evidence, it is more likely than not that some or all of the deferred tax assets will not be realized. The Company accounts for uncertain tax positions using a more-likely-than-not threshold for recognizing and resolving uncertain tax positions. The evaluation of uncertain tax positions is based on factors including, but not limited to, changes in the law, the measurement of tax positions taken or expected to be taken in tax returns, the effective settlement of matters subject to audit, new audit activity, and changes in facts or circumstances related to a tax position. The Company evaluates its tax positions on an annual basis. On December 22, 2017, legislation commonly known as the Tax Cuts and Jobs Act (the “Tax Act”) was signed into law. The Tax Act, among other changes, reduces the U.S. federal corporate tax rate from 34% to 21%, requires taxpayers to pay a one-time transition tax on earnings of certain foreign subsidiaries that were previously tax deferred and creates new taxes on certain foreign sourced earnings. As of December 31, 2017, the Company did not have any foreign subsidiaries and the international aspects of the Tax Act are not applicable. In connection with the initial analysis on the impact of the Tax Act, the Company remeasured certain deferred tax assets and liabilities based on the rates at which they are expected to reverse in the future, which is generally 21%. The remeasurement of the Company's deferred tax balance was primarily offset by application of its valuation allowance. However, the reduction of the U.S. federal corporate tax rate resulted in increases to the amounts reflected in “Deferred Rate Change” and “Change in valuation allowance” captions for the year ended December 31, 2017 in the Company’s tax reconciliation table compared to those amounts disclosed for the years ended December 31, 2016 and 2015. The change in the U.S. federal corporate tax rate, which is effective January 1, 2018, is also reflected in the Company’s deferred tax table. The Company is still in the process of analyzing the impact to the Company of the Tax Act. On December 22, 2017, the SEC staff issued SAB 118 to address the application of GAAP in situations when a registrant does not have the necessary information available, prepared, or analyzed (including computations) in reasonable detail to complete the accounting for certain income tax effects of the Tax Act. The Company has recognized the provisional tax impacts related to the revaluation of the deferred tax assets and liabilities and included these amounts in its consolidated financial statements for the year ended December 31, 2017. The ultimate impact may differ from these provisional amounts due to, among other things, additional analysis, changes in interpretations and assumptions the Company has made, additional regulatory guidance that may be issued, and actions the Company may take as a result of the Tax Act, which could result in changes to the provisional tax impacts during 2018. The provision for income taxes is as follows: Year ended December 31, 2017 2016 (in thousands) Current Federal $ — $ 180 State — — Total current — 180 Deferred Federal — (111) State — (17) Total deferred — (128) Total tax expense $ — $ 52 A reconciliation of the expected income tax (benefit) computed using the federal statutory income tax rate at the Company’s effective tax rate is as follows: Year ended December 31, 2017 2016 2015 Income tax computed at federal statutory tax rate 34.0 % 34.0 % 34.0 % State taxes, net of federal benefit 6.1 % 5.6 % 4.1 % General business credit carryovers 5.0 % 4.2 % 3.1 % Non-deductible expenses (4.1) % (4.0) % (15.5) % Deferred rate change (21.8) % — % — % Change in valuation allowance (19.2) % (40.2) % (25.7) % Total — % (0.4) % — % The Company has incurred net operating losses (“NOLs”) since June 2013. At December 31, 2017, the Company had federal and state net operating loss carryforwards of $89.8 million and $91.7 million, respectively, which expire beginning in 2033. As of December 31, 2017, the Company also had federal and state research and development tax credit carryforwards of $6.6 million and $2.3 million, respectively, which expire beginning in 2028. As of December 31, 2017, the Company had state investment credits of $0.3 million, which expire beginning in 2018. Under the provisions of the Internal Revenue Code, certain substantial changes in the Company’s ownership may result in a limitation on the amount of NOL carryforwards and research and development credit carryforwards that may be utilized annually to offset future taxable income and taxes payable. In general, an ownership change, as defined by Section 382, results from transactions that increase the ownership of 5% stockholders or public groups in the stock of a corporation by more than 50 percent in the aggregate over a three-year period. During 2016, the Company completed a study through June 30, 2016, to determine whether any ownership change has occurred since the Company’s formation and has determined that transactions have resulted in three ownership changes, as defined by Section 382. There could be additional ownership changes in the future that could further limit the amount of NOLs and tax credit carryforwards that the Company can utilize. The significant components of the Company’s deferred tax assets and (liabilities) as of December 31, 2017 and 2016 are as follows: Year ended December 31, 2017 2016 (in thousands) Deferred tax assets: Net operating loss carryforwards $ 24,642 $ 8,927 Tax credit carryforwards 8,832 4,284 Deferred rent 1,458 1,964 Deferred revenue 8,622 16,333 Non-deductible expenses 817 714 Intangibles 832 998 Stock compensation 1,361 672 Total deferred tax assets 46,564 33,892 Less valuation allowance (44,953) (31,361) Net deferred tax assets 1,611 2,531 Deferred tax liabilities Depreciation and amortization (1,611) (2,501) Unrealized gain on available-for-sale securities — (30) Net deferred taxes $ — $ — As required by ASC 740, management has evaluated the positive and negative evidence bearing upon the realizability of its deferred tax assets, which principally comprise NOL carryforwards, research and development credit carryforwards, and capitalized license and organization costs. Management has determined that it is more likely than not that the Company will not recognize the benefits of its federal and state deferred tax assets, and as a result, a valuation allowance of $45.0 million and $31.4 million has been established at December 31, 2017 and 2016, respectively. The change in valuation allowance was $13.6 million for the year ended December 31, 2017, primarily due to additional operating losses incurred by the Company for the year ended December 31, 2017, partially offset by the federal rate reduction from 34% to 21% as a result of the Tax Act. The primary reason for the difference between the income tax expense recorded by the Company and the amount of income tax expense at statutory income tax rates was the change in the valuation allowance. At December 31, 2017 and 2016, the Company had no unrecognized tax benefits. The Company has not as yet conducted a study of its research and development credit carryforwards. This study may result in an adjustment to the Company’s research and development credit carryforwards; however, until a study is completed, and any adjustment is known, no amounts are being presented as an uncertain tax position. A full valuation allowance has been provided against the Company’s research and development credits, and if an adjustment is required, this adjustment would be offset by an adjustment to the valuation allowance. Thus, there would be no impact to the balance sheets or statements of operations if an adjustment were required. Interest and penalty charges, if any, related to unrecognized tax benefits would be classified as income tax expense in the accompanying statements of operations. As of December 31, 2017 and 2016, the Company has no accrued interest related to uncertain tax positions. Since the Company is in a loss carryforward position, it is generally subject to examination by the U.S. federal, state, and local income tax authorities for all tax years in which a loss carryforward is available. |
Related party transactions
Related party transactions | 12 Months Ended |
Dec. 31, 2017 | |
Related party transactions | |
Related party transactions | 13. Related‑party transactions Since inception, the Company received consulting and management services from one of its investors. The total amount of consulting and management services provided by this investor was approximately $31.8 thousand, $0.1 million, and $1.3 million during the years ended December 31, 2017, 2016, and 2015, respectively. During the years ended December 31, 2017, 2016, and 2015, the Company recognized $10.1 million, $14.2 million, and $17.3 million, respectively, of revenue associated with its collaboration with Sanofi Genzyme related to research and development services provided during these periods. The Company also recognized $0.1 million, $1.2 million, and $2.3 million of expense during the years ended December 31, 2017, 2016, and 2015, respectively, related to in‑kind services provided by Sanofi Genzyme associated with the collaboration arrangement. |
Selected quarterly financial da
Selected quarterly financial data (unaudited) | 12 Months Ended |
Dec. 31, 2017 | |
Quarterly Financial Information Disclosure [Abstract] | |
Selected Quarterly Financial Data | 14. Selected quarterly financial data (unaudited) The following table contains quarterly financial information for 2017 and 2016. The Company believes that the following information reflects all normal recurring adjustments necessary for a fair statement of the information for the periods presented. The operating results for any quarter are not necessarily indicative of results for any future period. 2017 First Second Third Fourth Quarter Quarter Quarter Quarter Total (amounts in thousands, except per share data) Collaboration revenue $ 1,464 $ 1,177 $ 1,148 $ 6,346 $ 10,135 Total operating expenses 18,986 19,816 24,503 18,693 81,998 Loss from operations (17,522) (18,639) (23,355) (12,347) (71,863) Net loss attributable to common stockholders (16,648) (18,876) (23,346) (11,828) (70,698) Net loss per share applicable to common stockholders – basic and diluted $ (0.65) $ (0.73) $ (0.89) $ (0.40) $ (2.64) 2016 First Second Third Fourth Quarter Quarter Quarter Quarter Total (amounts in thousands, except per share data) Collaboration revenue $ 4,830 $ 3,720 $ 3,308 $ 2,362 $ 14,220 Total operating expenses 12,297 13,338 13,679 16,205 55,519 Loss from operations (7,467) (9,618) (10,371) (13,843) (41,299) Net loss attributable to common stockholders (7,188) (9,335) (8,996) (14,674) (40,193) Net loss per share applicable to common stockholders – basic and diluted $ (0.29) $ (0.37) $ (0.35) $ (0.57) $ (1.59) |
Subsequent events
Subsequent events | 12 Months Ended |
Dec. 31, 2017 | |
Subsequent events | |
Subsequent events | 15. Subsequent events Lease Amendment In February 2018, the Company executed an amendment to lease an additional approximately 11,000 square feet at 75 Sidney Street that is intended to support its continued growth. The additional facility includes laboratory and office space, and will be ready for occupancy in the first half of 2018. AbbVie Collaboration In February 2018, the Company entered into an exclusive collaboration and option agreement with AbbVie Biotechnology Ltd (“AbbVie”), for the research, development, and commercialization of AAV and other virus-based gene therapy products for the treatment of diseases of the central nervous system and other neurodegenerative diseases related to defective or excess aggregation of tau protein in the human brain, including Alzheimer’s disease. Under the terms of the agreement, the Company received an upfront payment of $69.0 million and may receive future development and regulatory milestone payments and royalties. Under the terms of the agreement, the Company will perform |
Summary of significant accoun24
Summary of significant accounting policies (Policies) | 12 Months Ended |
Dec. 31, 2017 | |
Summary of significant accounting policies | |
Basis of presentation | The accompanying consolidated financial statements include those of the Company and its subsidiary, Voyager Securities Corporation, after elimination of all intercompany accounts and transactions. The accompanying consolidated financial statements have been prepared in conformity with accounting principles generally accepted in the United States of America (“GAAP”). |
Public offerings | On October 29, 2015, in preparation for the Company’s IPO, the Company’s Board of Directors and stockholders approved a 1-for-4.25 reverse split of the Company’s common stock, which became effective on October 29, 2015. All share and per share amounts in the consolidated financial statements and notes thereto have been retroactively adjusted for all periods presented to give effect to this reverse split, including reclassifying an amount equal to the reduction in par value of common stock to additional paid-in capital. On November 16, 2015, the Company completed the sale of 5,750,000 shares of its common stock in its initial public offering (the “IPO”), at a price to the public of $14.00 per share, resulting in net proceeds to the Company of $72.9 million after deducting underwriting discounts, commissions and offering expenses payable by the Company. Upon the closing of the IPO, all of the Company’s outstanding redeemable convertible preferred stock automatically converted into shares of common stock, resulting in the issuance of an additional 17,647,054 shares of common stock. The significant increase in shares outstanding in November 2015 effected the year-over-year comparability of the Company’s net loss per share calculations. On November 7, 2017, the Company completed the sale of 5,175,000 shares of its common stock in a public offering at a price to the public of $12.00 per share, resulting in net proceeds to the Company of $58.0 million after deducting underwriting discounts, commissions, and offering expenses payable by the Company. |
Use of estimates | The preparation of consolidated financial statements in conformity with GAAP requires management to make estimates and assumptions that affect the amounts reported in the consolidated financial statements and accompanying notes. On an ongoing basis, the Company’s management evaluates its estimates, which include, but are not limited to, estimates related to revenue recognition, accrued expenses, valuation of the tranche rights, stock‑based compensation expense, income taxes and the fair value of common stock. The Company bases its estimates on historical experience and other market specific or other relevant assumptions that it believes to be reasonable under the circumstances. Actual results may differ from those estimates or assumptions. |
Fair Value of Financial Instruments | ASC Topic 820, Fair Value Measurement (“ASC 820”), establishes a fair value hierarchy for instruments measured at fair value that distinguishes between assumptions based on market data (observable inputs) and the Company’s own assumptions (unobservable inputs). Observable inputs are inputs that market participants would use in pricing the asset or liability based on market data obtained from sources independent of the Company. Unobservable inputs are inputs that reflect the Company’s assumptions about the inputs that market participants would use in pricing the asset or liability, and are developed based on the best information available in the circumstances. ASC 820 identifies fair value as the exchange price, or exit price, representing the amount that would be received to sell an asset or paid to transfer a liability in an orderly transaction between market participants. As a basis for considering market participant assumptions in fair value measurements, ASC 820 establishes a three‑tier fair value hierarchy that distinguishes between the following: · Level 1 —Quoted market prices in active markets for identical assets or liabilities. · Level 2 —Inputs other than Level 1 inputs that are either directly or indirectly observable, such as quoted market prices, interest rates, and yield curves. · Level 3 —Unobservable inputs developed using estimates of assumptions developed by the Company, which reflect those that a market participant would use. To the extent that the valuation is based on models or inputs that are less observable or unobservable in the market, the determination of fair value requires more judgment. Accordingly, the degree of judgment exercised by the Company in determining fair value is greatest for instruments categorized in Level 3. A financial instrument’s level within the fair value hierarchy is based on the lowest level of any input that is significant to the fair value measurement. The carrying amounts reflected in the balance sheets for cash and cash equivalents, prepaid expenses and other current assets, accounts payable and accrued expenses approximate their fair values, due to their short‑term nature. |
Cash and cash equivalents | The Company considers all highly liquid investments purchased with original maturities of 90 days or less at acquisition to be cash equivalents. Cash and cash equivalents include cash held in banks and amounts held in money market funds. |
Marketable securities | The Company classifies marketable debt securities with a remaining maturity of greater than three months when purchased as available‑for‑sale. Marketable debt securities with a remaining maturity date greater than one year and marketable equity securities are classified as non‑current where the Company has the intent and ability to hold these securities for at least the next 12 months. During 2016, the Company invested in a supplier and received common stock and warrants to purchase common stock in that entity. The common stock is considered an available-for-sale marketable equity security and is included in non-current marketable securities, and the warrants are included in non-current assets. All available for sale securities are carried at fair value with the unrealized gains and losses included in other comprehensive income (loss) as a component of stockholders’ equity until realized. Any premium or discount arising at purchase is amortized and/or accreted to interest income and/or expense. Realized gains and losses are determined using the specific identification method and are included in other income (expense). If any adjustment to fair value reflects a decline in value of the investment, the Company considers all available evidence to evaluate the extent to which the decline is “other than temporary” and, if so, recognizes the loss through a charge to the Company’s statement of operations and comprehensive loss. No other than temporary losses have been recognized. Cash, cash equivalents, and marketable securities as of December 31, 2017 and 2016 consist of the following: Amortized Unrealized Unrealized Fair Cost Gains Losses Value (in thousands) As of December 31, 2017 Money market funds included in cash and cash equivalents $ 30,469 $ — $ — $ 30,469 Marketable securities: U.S. Treasury notes 137,560 — 38 137,522 Equity securities 1,220 — 120 1,100 Total marketable securities $ 138,780 $ — $ 158 $ 138,622 Total money market funds and marketable securities $ 169,249 $ — $ 158 $ 169,091 As of December 31, 2016 Money market funds included in cash and cash equivalents $ 36,003 $ — $ — $ 36,003 Marketable securities: U.S. Treasury notes 130,237 2 66 130,173 U.S. Government agency bonds 7,604 — — 7,604 Total debt securities $ 137,841 $ 2 $ 66 $ 137,777 Equity securities 1,220 140 — 1,360 Total marketable securities $ 139,061 $ 142 $ 66 $ 139,137 Total money market funds and marketable securities $ 175,064 $ 142 $ 66 $ 175,140 All of the Company’s marketable debt securities at December 31, 2017 and 2016 have a contractual maturity of one year or less. |
Restricted cash | At December 31, 2017 and 2016, the Company maintained restricted cash totaling approximately $0.7 million held in the form of money market accounts as collateral for the Company’s facility lease obligation. The balance is included within deposits in other non‑current assets in the accompanying consolidated balance sheets. |
Property and equipment | Property and equipment consists of laboratory equipment, furniture and office equipment, and leasehold improvements and is stated at cost, less accumulated depreciation. Maintenance and repairs that do not improve or extend the lives of the respective assets are expensed to operations as incurred; while costs of major additions and betterments are capitalized. Depreciation is calculated over the estimated useful lives of the assets using the straight‑line method. |
Impairment of long-lived assets | The Company evaluates long‑lived assets for potential impairment when events or changes in circumstances indicate the carrying value of the assets may not be recoverable. Recoverability is measured by comparing the book values of the assets to the expected future net undiscounted cash flows that the assets are expected to generate. If such assets are considered to be impaired, the impairment to be recognized is measured by the amount by which the book values of the assets exceed their fair value. The Company has not recognized any impairment losses from inception through December 31, 2017. |
Revenue Recognition | As of December 31, 2017, all of the Company’s revenue is generated exclusively from its collaboration agreement with Sanofi Genzyme Corporation, a Sanofi company (“S anofi Genzyme”) . The Company recognizes revenue in accordance with ASC Topic 605, Revenue Recognition (“ASC 605”). Accordingly, revenue is recognized for each unit of accounting when all of the following criteria are met: · Persuasive evidence of an arrangement exists; · Delivery has occurred or services have been rendered; · The seller’s price to the buyer is fixed or determinable; and · Collectability is reasonably assured. Amounts received prior to satisfying the revenue recognition criteria are recorded as deferred revenue in the Company’s balance sheet. Amounts expected to be recognized as revenue within 12 months following the balance sheet date are classified as deferred revenue, current portion. Amounts not expected to be recognized as revenue within the 12 months following the balance sheet date are included in noncurrent liabilities and classified as deferred revenue, net of current portion. The Company analyzes the multiple element arrangements based on the guidance in ASC Topic 605‑25, Revenue Recognition—Multiple Element Arrangements (“ASC 605‑25”). Pursuant to the guidance in ASC 605‑25, the Company evaluates multiple element arrangements to determine (1) the deliverables included in the arrangement and (2) whether the individual deliverables represent separate units of accounting or whether they must be accounted for as a combined unit of accounting. This evaluation involves subjective determinations and requires management to make judgments about the individual deliverables and whether such deliverables are separate from other aspects of the contractual relationship. Deliverables are considered separate units of accounting provided that: (i) the delivered item(s) has value to the customer on a standalone basis and (ii) if the arrangement includes a general right of return relative to the delivered item(s), delivery or performance of the undelivered item(s) is considered probable and substantially within control of the Company. In assessing whether an item has standalone value, the Company considers factors such as the research, manufacturing and commercialization capabilities of the collaboration partner and the availability of the associated expertise in the general marketplace. In addition, the Company considers whether the collaboration partner can use the other deliverable(s) for their intended purpose without the receipt of the remaining element(s), whether the value of the deliverable is dependent on the undelivered item(s) and whether there are other vendors that can provide the undelivered element(s). The Company’s collaboration agreement does not contain a general right of return relative to any delivered items. Arrangement consideration that is fixed or determinable is allocated among the separate units of accounting using the relative selling price method. Then the applicable revenue recognition criteria in ASC 605 are applied to each of the separate units of accounting in determining the appropriate period and pattern of recognition. The Company determines the selling price of a unit of accounting following the hierarchy of evidence prescribed by ASC 605‑25. Accordingly, the Company determines the estimated selling price for units of accounting within each arrangement using vendor specific objective evidence (“VSOE”) of selling price, if available, third-party evidence (“TPE”) of selling price if VSOE is not available, or best estimate of selling price (“BESP”) if neither VSOE or TPE is available. The Company has only used BESP to estimate the selling price, since it has not had VSOE or TPE of selling price of any units of accounting to date. Determining BESP for a unit of accounting requires significant judgment. In developing the BESP for a unit of accounting, the Company considers applicable market conditions and relevant entity specific factors, including factors that were contemplated in negotiating the agreement with the customer and estimated costs. The Company validates BESP for units of accounting by evaluating whether changes in the key assumptions used to determine the BESP will have a significant effect on the allocation of arrangement consideration between multiple units of accounting. Options are considered substantive if, at the inception of the arrangement, the Company is at risk as to whether the collaboration partner will choose to exercise the option. Factors that the Company considers in evaluating whether an option is substantive include the cost to exercise the option, the overall objective of the arrangement, the benefit the collaborator might obtain from the arrangement without exercising the option and the likelihood the option will be exercised. When an option is considered substantive, the Company does not consider the option or item underlying the option to be a deliverable at the inception of the arrangement and the associated option fees are not included in allocable consideration, assuming the option is not priced at a significant and incremental discount. Conversely, when an option is not considered substantive, the Company would consider the option, including other deliverables contingent upon the exercise of the option, to be a deliverable at the inception of the arrangement and a corresponding amount would be included in allocable arrangement consideration. In addition, if the price of the option includes a significant incremental discount, the option would be included as a deliverable at the inception of the arrangement. The Company recognizes arrangement consideration allocated to each unit of accounting when all of the revenue recognition criteria in ASC 605 are satisfied for that particular unit of accounting. The Company will recognize revenue associated with license options upon exercise of the option, if the underlying license has standalone value from the other deliverables to be provided subsequent to delivery of the license. If the license does not have standalone value, the amounts allocated to the license option will be combined with the related undelivered items as a single unit of accounting. The amounts allocated to the license option in the Sanofi Genzyme agreement will be deferred until the option is exercised. The revenue recognition upon option exercise will be determined based on whether the license has standalone value from the remaining deliverables under the arrangement at the time of exercise. The Company recognizes the amounts associated with research and development services, alliance joint steering committees and development advisory committees ratably over the associated period of performance. If there is no discernible pattern of performance and/or objectively measurable performance measures do not exist, then the Company recognizes revenue under the arrangement on a straight‑line basis over the period the Company is expected to complete its performance obligations. Conversely, if the pattern of performance in which the service is provided to the customer can be determined and objectively measurable performance exist, then the Company recognizes revenue under the arrangement using the proportional performance method. Revenue recognized is limited to the lesser of the cumulative amount of payments received or the cumulative revenue earned determined using the straight line method or proportional performance, as applicable, as of the period end date. At the inception of an arrangement that includes milestone payments, the Company evaluates whether each milestone is substantive and at risk to both parties on the basis of the contingent nature of the milestone. This evaluation includes an assessment of whether: (i) the consideration is commensurate with either the Company’s performance to achieve the milestone or the enhancement of the value of the delivered item(s) as a result of a specific outcome resulting from the Company’s performance to achieve the milestone, (ii) the consideration relates solely to past performance and (iii) the consideration is reasonable relative to all the deliverables and payment terms within the arrangement. The Company evaluates factors such as clinical, regulatory, commercial and other risks that must be overcome to achieve the respective milestone and the level of effort and investment required to achieve the respective milestone in making this assessment. There is considerable judgment involved in determining whether a milestone satisfies all the criteria required to conclude that a milestone is substantive. In accordance with ASC Topic 605‑28, Revenue Recognition—Milestone Method (“ASC 605‑28”) clinical and regulatory milestones that are considered substantive, will be recognized as revenue in their entirety upon successful accomplishment of the milestone, assuming all other revenue recognition criteria are met. Milestones that are not considered substantive would be recognized as revenue over the remaining period of performance, assuming all other revenue recognition criteria are met. Revenue from commercial milestone payments will be accounted for as royalties and recorded as revenue upon achievement of the milestone, assuming all other revenue recognition criteria are met. The Company will recognize royalty revenue in the period of sale of the related product(s), based on the underlying contract terms, provided that the reported sales are reliably measurable and the Company has no remaining performance obligations, assuming all other revenue recognition criteria are met. The Company also considers the impact of potential future payments it makes in its role as a vendor to its customers or collaboration partners and evaluates if these potential future payments could be reductions of revenue from that customer. If the potential future payments to the customer are (i) a separately identifiable benefit and (ii) the fair value of the identifiable benefit can be reasonably estimated, then the payments are accounted for separately from the revenue received from the customer. If however, both of these criteria are not satisfied, then the payments are treated as a reduction of revenue. |
Research and development | Research and development costs are charged to expense as incurred in performing research and development activities. The costs include employee compensation costs, external research, consultant costs, sponsored research, in‑kind services provided under the Sanofi Genzyme agreement, license fees, process development and facilities costs. Facilities costs primarily include the allocation of rent, utilities and depreciation. |
Research contract costs and accruals | The Company has entered into various research and development contracts with research institutions and other companies. These agreements are generally cancelable. The Company records accruals for estimated ongoing research costs. When evaluating the adequacy of the accrued liabilities, the Company analyzes progress of the studies, including the phase or completion of events, invoices received and contracted costs. Significant judgments and estimates may be made in determining the accrued balances at the end of any reporting period. Actual results could differ from the Company’s estimates. The Company’s historical accrual estimates have not been materially different from the actual costs. |
Patent costs | The Company expenses patent application and related legal costs as incurred and classifies such costs as general and administrative expenses in the accompanying statements of operations. |
Stock-based compensation expense | The Company accounts for its stock‑based compensation awards in accordance with ASC Topic 718 Compensation—Stock Compensation (“ASC 718”). ASC 718 requires all stock‑based payments to employees and directors, including grants of restricted stock and stock options, to be recognized as expense in the statements of operations based on their grant date fair values. Grants of restricted stock and stock options to other service providers, referred to as non‑employees, are required to be recognized as expense in the statements of operations based on their vesting date fair values. The Company estimates the fair value of options granted using the Black‑Scholes option pricing model. The Company uses the fair value of its common stock to determine the fair value of restricted stock awards. The Black‑Scholes option pricing model requires inputs based on certain subjective assumptions, including (a) the expected stock price volatility, (b) the calculation of expected term of the award, (c) the risk‑free interest rate and (d) expected dividends. Due to a lack of company-specific historical and implied volatility data, the Company bases the estimate of expected volatility on the historical volatility of a group of similar companies that are publicly traded, blended with the most recent period of historic volatility of its common stock. The historical volatility is calculated based on a period of time commensurate with the expected term assumption. The computation of expected volatility is based on the historical volatility of a representative group of companies with similar characteristics to the Company, including stage of product development and life science industry focus. The Company uses the simplified method as prescribed by the SEC Staff Accounting Bulletin No. 107, Share‑Based Payment , to calculate the expected term for stock options granted to employees as it does not have sufficient historical exercise data to provide a reasonable basis upon which to estimate the expected term. For stock options granted to non‑employees, the Company utilizes the contractual term of the arrangement as the basis for the expected term assumption. The risk‑free interest rate is based on a treasury instrument whose term is consistent with the expected term of the stock options. The expected dividend yield is assumed to be zero as the Company has never paid dividends and has no current plans to pay any dividends on its common stock. The Company expenses the fair value of its stock‑based compensation awards to employees on a straight‑line basis over the associated service period, which is generally the period in which the related services are received. Stock‑based compensation awards to non‑employees are adjusted through stock‑based compensation expense at each reporting period end to reflect the current fair value of such awards and are expensed on a straight‑line basis. The Company records the expense for stock‑based compensation awards subject to performance conditions over the remaining service period when management determines that achievement of the performance condition is probable. Management evaluates when the achievement of a performance condition is probable based on the expected satisfaction of the performance conditions as of the reporting date. |
Income taxes | Income taxes are recorded in accordance with ASC Topic 740, Income Taxes (“ASC 740”), which provides for deferred taxes using an asset and liability approach. Under this method, deferred tax assets and liabilities are determined based on the difference between the financial reporting and the tax reporting basis of assets and liabilities and are measured using the enacted tax rates and laws that are expected to be in effect when the differences are expected to reverse. The Company provides a valuation allowance against net deferred tax assets unless, based upon the weight of available evidence, it is more likely than not that the deferred tax assets will be realized. The Company accounts for uncertain tax positions in accordance with the provisions of ASC 740. When uncertain tax positions exist, the Company recognizes the tax benefit of tax positions to the extent that the benefit will more likely than not be realized. The determination as to whether the tax benefit will more likely than not be realized is based upon the technical merits of the tax position as well as consideration of the available facts and circumstances. As of December 31, 2017, the Company does not have any significant uncertain tax positions. |
Comprehensive loss | Comprehensive loss is comprised of net loss and other comprehensive income or loss. Other comprehensive gain or loss consists of unrealized gains or losses on marketable securities. |
Net loss per share | Basic net loss per share attributable to common stockholders is calculated by dividing the net loss attributable to common stockholders by the weighted‑average number of shares of common stock outstanding during the period, without consideration for potentially dilutive securities. Diluted net loss per share attributable to common stockholders is computed by dividing the net loss attributable to common stockholders by the weighted‑average number of shares of common stock and potentially dilutive securities outstanding for the period determined using the treasury‑stock and if‑converted methods. For purposes of the diluted net loss per share attributable to common stockholders calculation, redeemable convertible preferred stock, unvested restricted common stock, and outstanding stock options are considered to be potentially dilutive securities, but are excluded from the calculation of diluted net loss per share attributable to common stockholders because their effect would be anti‑dilutive and therefore, basic and diluted net loss per share attributable to common stockholders were the same for all periods presented. The following table sets forth the outstanding potentially dilutive securities that have been excluded in the calculation of diluted net loss per share attributable to common stockholders because to do so would be anti‑dilutive: As of December 31, 2017 2016 2015 Unvested restricted common stock 557,979 1,167,984 1,818,261 Shares reserved for issuance under stock plans 5,375,431 4,226,265 2,905,458 Total 5,933,410 5,394,249 4,723,719 All of the Company’s outstanding convertible preferred stock automatically converted into shares of common stock as of November 16, 2015, resulting in the issuance by the Company of an additional 17,647,054 shares of common stock. |
Concentration of credit and off-balance sheet risk | The Company has no financial instruments with off‑balance sheet risk such as foreign exchange contracts, option contracts or other foreign hedging arrangements. Financial instruments that potentially subject the Company to a concentration of credit risk are cash and cash equivalents. The Company’s cash is held in accounts at a financial institution that may exceed federally insured limits. The Company has not experienced any credit losses in such accounts and does not believe it is exposed to any significant credit risk on these funds. |
Concentration of suppliers | The Company is dependent on a third‑party manufacturer to supply certain products for research and development activities in its programs. In particular, the Company relies on a sole manufacturer to supply it with specific vectors related to the Company’s research and development programs. |
Segment information | Operating segments are defined as components of an enterprise about which separate discrete information is available for evaluation by the chief operating decision maker in deciding how to allocate resources and assess performance. The Company and the Company’s chief operating decision maker, the Company’s Chief Executive Officer, views the Company’s operations and manages its business as a single operating segment, which is the business of developing and commercializing gene therapies. |
Recent accounting pronouncements | In May 2014, the FASB issued ASU No. 2014-09, Revenue from Contracts with Customers (Topic 606) (“ASU 2014-09”). Subsequently, the FASB also issued ASU 2015-14, Revenue from Contracts with Customers (Topic 606), which adjusted the effective date of ASU 2014-09; ASU No. 2016-08, Revenue from Contracts with Customers (Topic 606): Principal versus Agent Considerations (Reporting Revenue Gross versus Net), which amends the principal-versus-agent implementation guidance and illustrations in ASU 2014-09; ASU No. 2016-10, Revenue from Contracts with Customers (Topic 606): Identifying Performance Obligations and Licensing, which clarifies identifying performance obligation and licensing implementation guidance and illustrations in ASU 2014-09; and ASU No. 2016-12, Revenue from Contracts with Customers (Topic 606): Narrow-Scope Improvements and Practical Expedients, which addresses implementation issues and is intended to reduce the cost and complexity of applying the new revenue standard in ASU 2014-09 (collectively, the “Revenue ASUs”). The Revenue ASUs provide an accounting standard for a single comprehensive model for use in accounting for revenue arising from contracts with customers and supersedes most current revenue recognition guidance. The accounting standard is effective for interim and annual periods beginning after December 15, 2017, with an option to early adopt for interim and annual periods beginning after December 15, 2016. The guidance permits two methods of adoption: retrospectively to each prior reporting period presented (the full retrospective method), or retrospectively with the cumulative effect of initially applying the guidance recognized at the date of initial application (the modified retrospective method). As of December 31, 2017, the Company had one revenue generating arrangement, which was the Sanofi Genzyme Collaboration, as defined below. The Company has elected to adopt the Revenue ASUs effective as of January 1, 2018, under the modified retrospective method. The Company has substantially completed its assessment of the effect that this new standard will have on its consolidated financial statements. The most significant effect of the adoption is expected to be the method of revenue recognition for services performed over time. Under the previous accounting standards, revenue was recognized over the estimated period of performance while revenue will be recognized based on a proportional performance model under the Revenue ASUs. This change in accounting policy, along with certain other factors, is expected to result in an adjustment of approximately $18.0 million to $23.0 million, representing an increase of deferred revenue with an offset to accumulated deficit on January 1, 2018 to reflect the cumulative effect of the accounting changes made upon the adoption of the standard. In addition, the Company expects that the changes in accounting for contingent milestone payments will have a significant effect on the future accounting treatment for the arrangement. The previous accounting guidance contained specific guidance related to the accounting for milestone payments, including, if certain criteria were met, the ability to recognize all consideration related to the milestone once that milestone was achieved. The Revenue ASUs do not contain guidance specific to milestone payments, thereby requiring potential milestone payments to be considered in accordance with the overall revenue recognition model. As a result, revenue from contingent milestone payments may be recognized earlier under the Revenue ASUs than under ASC 605, based on an assessment of the probability of achievement of the milestone and the likelihood of a significant reversal of such milestone revenue at each reporting date. This assessment may result in recognizing milestone revenue before the milestone event has been achieved. As of the adoption date, no consideration from contingent milestones was included in the arrangement consideration. The finalization of the Company’s assessment may result in significant changes to its estimates that may materially impact its preliminary estimate of the cumulative effect. The Company has implemented appropriate changes to its internal controls to support revenue recognition and additional revenue-related disclosures under the new standards. In October 2016, the FASB issued ASU No. 2016-16, Income Taxes (Topic 740): Intra-Entity Transfers of Assets Other Than Inventory (“ASU 2016-16”), which removes the prohibition in ASC 740 against the immediate recognition of the current and deferred income tax effects of intra-entity transfers of assets other than inventory. ASU 2016-16 is effective for the Company for annual periods beginning after December 15, 2017, and interim periods within those annual periods. Early adoption is permitted as of the beginning of a fiscal year for which neither the annual nor the interim (if applicable) financial statements have been issued. The Company is currently in the process of evaluating the impact that the adoption of this guidance will have on its consolidated financial statements and related disclosures. In February 2016, the FASB issued ASU No. 2016-02, a comprehensive new lease accounting standard, which provides revised guidance on accounting for lease arrangements by both lessors and lessees and requires lessees to recognize a lease liability and a right-of-use asset for most leases. The new guidance is effective for fiscal years beginning after December 15, 2018, and interim periods within those fiscal years, with early adoption permitted. The new standard must be applied using a modified retrospective transition approach which requires application of the new guidance for all periods presented. The Company is currently in the process of evaluating the impact that the adoption of this guidance will have on its consolidated financial statements and related disclosures. |
Summary of significant accoun25
Summary of significant accounting policies (Tables) | 12 Months Ended |
Dec. 31, 2017 | |
Summary of significant accounting policies | |
Schedule of Marketable securities | Cash, cash equivalents, and marketable securities as of December 31, 2017 and 2016 consist of the following: Amortized Unrealized Unrealized Fair Cost Gains Losses Value (in thousands) As of December 31, 2017 Money market funds included in cash and cash equivalents $ 30,469 $ — $ — $ 30,469 Marketable securities: U.S. Treasury notes 137,560 — 38 137,522 Equity securities 1,220 — 120 1,100 Total marketable securities $ 138,780 $ — $ 158 $ 138,622 Total money market funds and marketable securities $ 169,249 $ — $ 158 $ 169,091 As of December 31, 2016 Money market funds included in cash and cash equivalents $ 36,003 $ — $ — $ 36,003 Marketable securities: U.S. Treasury notes 130,237 2 66 130,173 U.S. Government agency bonds 7,604 — — 7,604 Total debt securities $ 137,841 $ 2 $ 66 $ 137,777 Equity securities 1,220 140 — 1,360 Total marketable securities $ 139,061 $ 142 $ 66 $ 139,137 Total money market funds and marketable securities $ 175,064 $ 142 $ 66 $ 175,140 |
Outstanding potentially dilutive securities excluded in the calculation of diluted net loss per share | The following table sets forth the outstanding potentially dilutive securities that have been excluded in the calculation of diluted net loss per share attributable to common stockholders because to do so would be anti‑dilutive: As of December 31, 2017 2016 2015 Unvested restricted common stock 557,979 1,167,984 1,818,261 Shares reserved for issuance under stock plans 5,375,431 4,226,265 2,905,458 Total 5,933,410 5,394,249 4,723,719 |
Fair Value Measurements (Tables
Fair Value Measurements (Tables) | 12 Months Ended |
Dec. 31, 2017 | |
Fair Value Measurements | |
Assets and liabilities measured at fair value on a recurring basis | Assets and liabilities measured at fair value on a recurring basis as of December 31, 2017 and 2016 are as follows: Quoted Prices Significant in Active Other Significant Markets for Observable Unobservable Identical Assets Inputs Inputs Assets Total (Level 1) (Level 2) (Level 3) (in thousands) December 31, 2017 Money market funds included in cash and cash equivalents $ 30,469 $ 30,469 $ — $ — Marketable securities: U.S. Treasury notes 137,522 137,522 — — Equity securities 1,100 1,100 — — Total marketable securities $ 138,622 $ 138,622 $ — $ — Warrants to purchase equity securities 569 — 569 — Total $ 169,660 $ 169,091 $ 569 $ — December 31, 2016 Money market funds included in cash and cash equivalents $ 36,003 $ 36,003 $ — $ — Marketable securities: U.S. Treasury notes 130,173 130,173 — — U.S. Government agency securities 7,604 — 7,604 — Equity securities 1,360 1,360 — — Total marketable securities $ 139,137 $ 131,533 $ 7,604 $ — Warrants to purchase equity securities 792 — 792 — Total $ 175,932 $ 167,536 $ 8,396 $ — |
Assumptions | The assumptions utilized to value the warrants to purchase equity securities as of December 31, 2017 and 2016 are as follows: As of December 31, 2017 2016 Risk-free interest rate 2.0 % 1.8 % Expected dividend yield — % — % Expected term (in years) 3.7 4.7 Expected volatility 103.5 % 97.5 % |
Prepaid expenses and other cu27
Prepaid expenses and other current assets (Tables) | 12 Months Ended |
Dec. 31, 2017 | |
Prepaid expense and other current assets | |
Schedule of prepaid expense and other current assets | 4. Prepaid expenses and other current assets Prepaid expenses and other current assets consist of the following: As of December 31, 2017 2016 (in thousands) Prepaid research and development contracts $ 1,330 $ 1,094 Other current assets 766 541 Prepaid insurance 520 430 Accrued interest receivable 122 339 Tenant improvement receivable — 1,964 Total $ 2,738 $ 4,368 |
Property and equipment, net (Ta
Property and equipment, net (Tables) | 12 Months Ended |
Dec. 31, 2017 | |
Property and equipment, net | |
Schedule of property and equipment, net | Property and equipment, net consists of the following: As of December 31, 2017 2016 (in thousands) Leasehold improvements $ 6,421 $ 1,341 Laboratory equipment 5,262 3,306 Furniture and office equipment 1,565 526 Other 25 242 Construction in progress — 3,873 Total property and equipment 13,273 9,288 Less: accumulated depreciation (2,990) (1,395) Property and equipment, net $ 10,283 $ 7,893 |
Accrued expenses (Tables)
Accrued expenses (Tables) | 12 Months Ended |
Dec. 31, 2017 | |
Accrued expenses | |
Schedule of accrued expenses | Accrued expenses consist of the following: As of December 31, 2017 2016 (in thousands) Research and development costs $ 5,780 $ 2,384 Employee compensation costs 3,383 2,399 Professional services 1,762 698 Accrued goods and services 388 842 Patent costs 120 89 Other 64 76 Total $ 11,497 $ 6,488 |
Commitments and contingencies (
Commitments and contingencies (Tables) | 12 Months Ended |
Dec. 31, 2017 | |
Commitments and Contingencies | |
Future annual minimum lease payments | Future annual minimum lease payments at December 31, 2017 are as follows: Total Minimum Lease Payments (in thousands) 2018 3,290 2019 3,382 2020 3,762 2021 3,868 2022 3,977 2023+ 8,295 $ 26,574 |
Allocable arrangement consideration allocation | Unit of Accounting Amount (in thousands) Research and Development Services for: Huntington’s Program $ 15,662 Parkinson’s Program 6,648 Friedreich’s Ataxia Program 16,315 Spinal Muscular Atrophy Program 32,050 Future Program 2,464 Committee Obligations: AJSC 147 DAC 227 License Option and related deliverables 5,743 Total $ 79,256 |
Common Stock (Tables)
Common Stock (Tables) | 12 Months Ended |
Dec. 31, 2017 | |
Common stock Abstract | |
Schedule of shares reserved for future issuance | Shares Reserved For Future Issuance As of December 31, 2017 2016 Shares reserved for vesting of restricted stock awards under the Founder Agreements 366,914 628,679 Shares reserved for vesting of restricted stock awards under the 2014 Option and Stock Plan 191,065 539,305 Shares reserved for exercise of stock options 3,143,566 1,871,237 Shares reserved for issuances under the 2015 Stock Option Plan 1,501,005 1,825,174 Shares reserved for issuances under the 2015 Employee Stock Purchase Plan 730,860 529,854 5,933,410 5,394,249 |
Stock based compensation (Table
Stock based compensation (Tables) | 12 Months Ended |
Dec. 31, 2017 | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |
Compensation cost recognized for all stock-based compensation awards | Year ended December 31, 2017 2016 2015 (in thousands) Research and development $ 5,367 $ 4,296 $ 3,218 General and administrative 3,871 2,014 809 Total stock-compensation expense $ 9,238 $ 6,310 $ 4,027 |
Summary of status and changes in unvested restricted stock | Weighted Average Grant Date Fair Value Shares Per Share Unvested restricted common stock as of December 31, 2016 1,167,984 $ 0.76 Issued — Vested (573,803) $ 0.77 Repurchased (36,202) $ 0.67 Unvested restricted common stock as of December 31, 2017 557,979 $ 0.70 |
Summary of status and changes in stock options | Weighted Remaining Aggregate Average Contractual Intrinsic Exercise Life Value Shares Price (in years) (in thousands) Outstanding at December 31, 2016 1,871,237 $ 10.21 Granted 1,720,000 $ 13.07 Exercised (158,677) $ 8.59 Cancelled or forfeited (288,994) $ 10.50 Outstanding at December 31, 2017 3,143,566 $ 11.82 8.6 $ 15,991 Exercisable at December 31, 2017 955,587 $ 10.45 8.1 $ 5,933 Vested and expected to vest at December 31, 2017 3,143,566 $ 11.82 8.6 $ 15,991 |
Assumptions | The assumptions utilized to value the warrants to purchase equity securities as of December 31, 2017 and 2016 are as follows: As of December 31, 2017 2016 Risk-free interest rate 2.0 % 1.8 % Expected dividend yield — % — % Expected term (in years) 3.7 4.7 Expected volatility 103.5 % 97.5 % |
Employees and directors stock options | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |
Assumptions | The fair value of each option issued to employees and directors was estimated at the date of grant using the Black‑Scholes option pricing model with the following weighted‑average assumptions: Year ended December 31, 2017 2016 2015 Risk-free interest rate 2.0 % 1.5 % 1.6 % Expected dividend yield — % — % — % Expected term (in years) 6.0 6.0 6.0 Expected volatility 73.7 % 73.1 % 78.6 % |
Non-employees stock options | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |
Assumptions | As of December 31, 2017 2016 2015 Risk-free interest rate 2.4 % 2.1 % 2.0 % Expected dividend yield — % — % — % Expected term (in years) 8.5 9.1 10.0 Expected volatility 76.2 % 83.3 % 84.0 % |
Income taxes (Tables)
Income taxes (Tables) | 12 Months Ended |
Dec. 31, 2017 | |
Income taxes | |
Provision for income taxes | The provision for income taxes is as follows: Year ended December 31, 2017 2016 (in thousands) Current Federal $ — $ 180 State — — Total current — 180 Deferred Federal — (111) State — (17) Total deferred — (128) Total tax expense $ — $ 52 |
Schedule of reconciliation of the expected income tax (benefit) computed using the federal statutory income tax rate to the effective income tax rate | A reconciliation of the expected income tax (benefit) computed using the federal statutory income tax rate at the Company’s effective tax rate is as follows: Year ended December 31, 2017 2016 2015 Income tax computed at federal statutory tax rate 34.0 % 34.0 % 34.0 % State taxes, net of federal benefit 6.1 % 5.6 % 4.1 % General business credit carryovers 5.0 % 4.2 % 3.1 % Non-deductible expenses (4.1) % (4.0) % (15.5) % Deferred rate change (21.8) % — % — % Change in valuation allowance (19.2) % (40.2) % (25.7) % Total — % (0.4) % — % |
Schedule of principal components of the Company’s deferred tax assets and liabilities | The significant components of the Company’s deferred tax assets and (liabilities) as of December 31, 2017 and 2016 are as follows: Year ended December 31, 2017 2016 (in thousands) Deferred tax assets: Net operating loss carryforwards $ 24,642 $ 8,927 Tax credit carryforwards 8,832 4,284 Deferred rent 1,458 1,964 Deferred revenue 8,622 16,333 Non-deductible expenses 817 714 Intangibles 832 998 Stock compensation 1,361 672 Total deferred tax assets 46,564 33,892 Less valuation allowance (44,953) (31,361) Net deferred tax assets 1,611 2,531 Deferred tax liabilities Depreciation and amortization (1,611) (2,501) Unrealized gain on available-for-sale securities — (30) Net deferred taxes $ — $ — |
Selected quarterly financial 34
Selected quarterly financial data (unaudited) (Tables) | 12 Months Ended |
Dec. 31, 2017 | |
Quarterly Financial Information Disclosure [Abstract] | |
Schedule of Quarterly Financial Information | 2017 First Second Third Fourth Quarter Quarter Quarter Quarter Total (amounts in thousands, except per share data) Collaboration revenue $ 1,464 $ 1,177 $ 1,148 $ 6,346 $ 10,135 Total operating expenses 18,986 19,816 24,503 18,693 81,998 Loss from operations (17,522) (18,639) (23,355) (12,347) (71,863) Net loss attributable to common stockholders (16,648) (18,876) (23,346) (11,828) (70,698) Net loss per share applicable to common stockholders – basic and diluted $ (0.65) $ (0.73) $ (0.89) $ (0.40) $ (2.64) 2016 First Second Third Fourth Quarter Quarter Quarter Quarter Total (amounts in thousands, except per share data) Collaboration revenue $ 4,830 $ 3,720 $ 3,308 $ 2,362 $ 14,220 Total operating expenses 12,297 13,338 13,679 16,205 55,519 Loss from operations (7,467) (9,618) (10,371) (13,843) (41,299) Net loss attributable to common stockholders (7,188) (9,335) (8,996) (14,674) (40,193) Net loss per share applicable to common stockholders – basic and diluted $ (0.29) $ (0.37) $ (0.35) $ (0.57) $ (1.59) |
Nature of business (Details)
Nature of business (Details) - USD ($) $ in Thousands | 1 Months Ended | 12 Months Ended | ||
Feb. 28, 2015 | Dec. 31, 2017 | Dec. 31, 2016 | Dec. 31, 2015 | |
Net loss | $ (70,698) | $ (40,193) | $ (29,672) | |
Accumulated deficit | $ (160,713) | $ (90,015) | ||
Collaborative Arrangement | ||||
Collaborative Arrangement Nonredeemable Up Front Fee Received | $ 65,000 | |||
Redeemable Convertible Preferred Stock Series B | ||||
Redeemable convertible preference stock issued (in shares) | 30,000,001 | |||
Redeemable Convertible Preferred Stock Series B | Collaborative Arrangement | ||||
Collaborative Arrangement Nonredeemable Up Front Fee Received | 30,000 | |||
Genzyme | Collaborative Arrangement | ||||
Collaborative Arrangement Nonredeemable Up Front Fee Received | $ 65,000 | |||
Genzyme | Redeemable Convertible Preferred Stock Series B | Collaborative Arrangement | ||||
Issuance of stock (in shares) | 10,000,000 |
Summary of significant accoun36
Summary of significant accounting policies - Basis of Presentation - (Details) $ / shares in Units, $ in Millions | Nov. 07, 2017USD ($)$ / sharesshares | Nov. 16, 2015USD ($)$ / sharesshares | Oct. 29, 2015 | Dec. 31, 2015shares | Dec. 31, 2017shares | Dec. 31, 2016shares |
Common stock sales | 5,175,000 | 31,572,044 | 25,597,912 | |||
Price per share | $ / shares | $ 12 | |||||
Net proceeds | $ | $ 58 | |||||
Reverse split ratio | 0.235 | |||||
Shares issued upon conversion of redeemable convertible preferred stock | 17,647,054 | |||||
IPO | ||||||
Common stock sales | 5,750,000 | |||||
Price per share | $ / shares | $ 14 | |||||
Net proceeds | $ | $ 72.9 | |||||
Common Stock | ||||||
Shares issued upon conversion of redeemable convertible preferred stock | 17,647,054 |
Summary of significant accoun37
Summary of significant accounting policies - Marketable Securities - (Details) - USD ($) $ in Thousands | 12 Months Ended | |||
Dec. 31, 2017 | Dec. 31, 2016 | Dec. 31, 2015 | Dec. 31, 2014 | |
Amortized cost basis | ||||
Cash and cash equivalents | $ 31,530 | $ 36,641 | $ 31,309 | $ 7,035 |
Marketable debt securities, amortized cost | 138,780 | 137,841 | ||
Total marketable securities, amortized cost | 139,061 | |||
Total money market funds and marketable securities amortized cost | 169,249 | 175,064 | ||
Unrealized gains | ||||
Debt securities, unrealized gains | 2 | |||
Total marketable securities, unrealized gains | 142 | |||
Total money market funds and marketable securities, unrealized gains | 142 | |||
Unrealized losses | ||||
Debt securities, unrealised losses | 66 | |||
Available-for-sale Securities, Gross Unrealized Loss, Total | 158 | 66 | ||
Total money market funds and marketable securities | 158 | 66 | ||
Fair value | ||||
Available-for-sale Securities, Debt Securities | 138,622 | 137,777 | ||
Marketable securities, fair value | 139,137 | |||
Total Money Market Funds And Marketable Securities Fair Value | 169,091 | 175,140 | ||
Money market funds | ||||
Amortized cost basis | ||||
Cash and cash equivalents | 30,469 | 36,003 | ||
Fair value | ||||
Cash and cash equivalents, fair value | 30,469 | 36,003 | ||
Deposits and other non-current assets | ||||
Restricted Cash and Investments [Abstract] | ||||
Restricted cash | 700 | |||
U.S. Treasury notes | ||||
Amortized cost basis | ||||
Marketable debt securities, amortized cost | 137,560 | 130,237 | ||
Unrealized gains | ||||
Debt securities, unrealized gains | 2 | |||
Unrealized losses | ||||
Debt securities, unrealised losses | 38 | 66 | ||
Fair value | ||||
Available-for-sale Securities, Debt Securities | 137,522 | 130,173 | ||
U. S. Government agency securities | ||||
Amortized cost basis | ||||
Marketable debt securities, amortized cost | 7,604 | |||
Fair value | ||||
Available-for-sale Securities, Debt Securities | 7,604 | |||
Equity Securities | ||||
Amortized cost basis | ||||
Marketable debt securities, amortized cost | 1,220 | |||
Marketable equity securities, amortized cost | 1,220 | |||
Unrealized gains | ||||
Equity securities unrealized gains | 140 | |||
Unrealized losses | ||||
Equity securities, unrealized losses | 120 | |||
Fair value | ||||
Available-for-sale Securities, Debt Securities | $ 1,100 | |||
Marketable equity securities, fair value | $ 1,360 |
Summary of significant accoun38
Summary of significant accounting policies - Deferred Issuance Costs - (Details) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2017 | Dec. 31, 2016 | Dec. 31, 2015 | |
Summary of significant accounting policies | |||
Other comprehensive loss | $ (235) | $ 199 | $ (251) |
Summary of significant accoun39
Summary of significant accounting policies - Net loss per share - (Details) - USD ($) $ in Thousands | Jan. 01, 2018 | Dec. 31, 2017 | Sep. 30, 2017 | Jun. 30, 2017 | Mar. 31, 2017 | Dec. 31, 2016 | Sep. 30, 2016 | Jun. 30, 2016 | Mar. 31, 2016 | Dec. 31, 2017 | Dec. 31, 2016 | Dec. 31, 2015 |
Earnings Per Share, Pro Forma [Abstract] | ||||||||||||
Antidilutive Securities Excluded from Computation of Earnings Per Share (in common stock equivalent shares) | 5,933,410 | 5,394,249 | 4,723,719 | |||||||||
Add: Changes in fair value of preferred stock tranche liability | $ 9,750 | |||||||||||
Net loss attributable to common stockholders | $ (11,828) | $ (23,346) | $ (18,876) | $ (16,648) | $ (14,674) | $ (8,996) | $ (9,335) | $ (7,188) | $ (70,698) | $ (40,193) | (38,290) | |
Accretion of redeemable convertible preferred stock to redemption value | (7,373) | |||||||||||
Add: Accrued dividends on temporary equity | $ 1,245 | |||||||||||
Weighted average number of common shares outstanding, basic and diluted (in shares) | 26,803,711 | 25,302,414 | 4,191,210 | |||||||||
Increase in deferred revenue | $ (10,135) | $ (14,582) | $ 52,666 | |||||||||
Minimum | Forecast | ||||||||||||
Earnings Per Share, Pro Forma [Abstract] | ||||||||||||
Increase in deferred revenue | $ 18,000 | |||||||||||
Maximum | Forecast | ||||||||||||
Earnings Per Share, Pro Forma [Abstract] | ||||||||||||
Increase in deferred revenue | $ 23,000 | |||||||||||
Unvested restricted stock awards | ||||||||||||
Earnings Per Share, Pro Forma [Abstract] | ||||||||||||
Antidilutive Securities Excluded from Computation of Earnings Per Share (in common stock equivalent shares) | 557,979 | 1,167,984 | 1,818,261 | |||||||||
Outstanding stock options | ||||||||||||
Earnings Per Share, Pro Forma [Abstract] | ||||||||||||
Antidilutive Securities Excluded from Computation of Earnings Per Share (in common stock equivalent shares) | 5,375,431 | 4,226,265 | 2,905,458 |
Fair Value Measurements - Asset
Fair Value Measurements - Assets measured on a recurring basis - (Details) - USD ($) $ in Thousands | Dec. 31, 2016 | Sep. 02, 2016 | Dec. 31, 2017 |
Fair value assumptions | |||
Risk-free interest rate | 1.80% | 2.00% | |
Expected term (in years) | 4 years 8 months 12 days | 3 years 8 months 12 days | |
Expected volatility | 97.50% | 103.50% | |
Estimate of Fair Value | |||
Assets, Fair Value Disclosure [Abstract] | |||
Investments, Fair Value Disclosure | $ 139,137 | $ 138,622 | |
Warrants to purchase equity securities | 792 | 569 | |
Total assets measured at fair value | 175,932 | 169,660 | |
Level 1 | Estimate of Fair Value | |||
Assets, Fair Value Disclosure [Abstract] | |||
Investments, Fair Value Disclosure | 131,533 | 138,622 | |
Total assets measured at fair value | 167,536 | 169,091 | |
Level 2 | Estimate of Fair Value | |||
Assets, Fair Value Disclosure [Abstract] | |||
Investments, Fair Value Disclosure | 7,604 | ||
Warrants to purchase equity securities | 792 | 569 | |
Total assets measured at fair value | 8,396 | 569 | |
U.S. Treasury notes | Estimate of Fair Value | |||
Assets, Fair Value Disclosure [Abstract] | |||
Investments, Fair Value Disclosure | 130,173 | 137,522 | |
U.S. Treasury notes | Level 1 | Estimate of Fair Value | |||
Assets, Fair Value Disclosure [Abstract] | |||
Investments, Fair Value Disclosure | 130,173 | 137,522 | |
U. S. Government agency securities | Estimate of Fair Value | |||
Assets, Fair Value Disclosure [Abstract] | |||
Investments, Fair Value Disclosure | 7,604 | ||
U. S. Government agency securities | Level 2 | Estimate of Fair Value | |||
Assets, Fair Value Disclosure [Abstract] | |||
Investments, Fair Value Disclosure | 7,604 | ||
Equity Securities | Estimate of Fair Value | |||
Assets, Fair Value Disclosure [Abstract] | |||
Investments, Fair Value Disclosure | 1,360 | 1,100 | |
Equity Securities | Level 1 | Estimate of Fair Value | |||
Assets, Fair Value Disclosure [Abstract] | |||
Investments, Fair Value Disclosure | 1,360 | 1,100 | |
Money market funds | |||
Assets, Fair Value Disclosure [Abstract] | |||
Cash and cash equivalents | 36,003 | 30,469 | |
Money market funds | Estimate of Fair Value | |||
Assets, Fair Value Disclosure [Abstract] | |||
Cash and cash equivalents | 36,003 | 30,469 | |
Money market funds | Level 1 | Estimate of Fair Value | |||
Assets, Fair Value Disclosure [Abstract] | |||
Cash and cash equivalents | $ 36,003 | $ 30,469 |
Fair Value Measurements - Serie
Fair Value Measurements - Series A Preferred Stock - (Details) | Nov. 07, 2017$ / shares |
Fair Value Measurements | |
Estimated future value at closing | $ 12 |
Prepaid expenses and other cu42
Prepaid expenses and other current assets (Details) - USD ($) $ in Thousands | Dec. 31, 2017 | Dec. 31, 2016 |
Prepaid expense and other current assets | ||
Tenant improvement receivable | $ 1,330 | $ 1,094 |
Prepaid expenses | 766 | 541 |
Prepaid research and development contracts | 520 | 430 |
Accrued interest receivable | 122 | 339 |
Other current assets | 1,964 | |
Total | $ 2,738 | $ 4,368 |
Property and equipment, net (De
Property and equipment, net (Details) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2017 | Dec. 31, 2016 | Dec. 31, 2015 | |
Property, Plant and Equipment [Line Items] | |||
Property, Plant and Equipment, Gross | $ 13,273 | $ 9,288 | |
Less: accumulated depreciation | (2,990) | (1,395) | |
Property and equipment, net | 10,283 | 7,893 | |
Depreciation expense | 1,595 | 612 | $ 600 |
Costs reimbursable | 3,000 | ||
Laboratory equipment | |||
Property, Plant and Equipment [Line Items] | |||
Property, Plant and Equipment, Gross | 6,421 | 1,341 | |
Leasehold improvements | |||
Property, Plant and Equipment [Line Items] | |||
Property, Plant and Equipment, Gross | 5,262 | 3,306 | |
Furniture and office equipment | |||
Property, Plant and Equipment [Line Items] | |||
Property, Plant and Equipment, Gross | 1,565 | 526 | |
Construction in Progress | |||
Property, Plant and Equipment [Line Items] | |||
Property, Plant and Equipment, Gross | $ 25 | 242 | |
Other | |||
Property, Plant and Equipment [Line Items] | |||
Property, Plant and Equipment, Gross | $ 3,873 |
Accrued expenses (Details)
Accrued expenses (Details) - USD ($) $ in Thousands | Dec. 31, 2017 | Dec. 31, 2016 |
Accrued expenses | ||
Research and development costs | $ 5,780 | $ 2,384 |
Employee compensation costs | 3,383 | 2,399 |
Professional services | 1,762 | 698 |
Accrued goods and services | 388 | 842 |
Other | 64 | 76 |
Patent costs | 120 | 89 |
Total | $ 11,497 | $ 6,488 |
Commitments and contingencies -
Commitments and contingencies - Operating Leases - (Details) $ in Thousands | 1 Months Ended | 12 Months Ended | |||
Feb. 28, 2018USD ($)ft² | Mar. 31, 2014item | Dec. 31, 2017USD ($) | Dec. 31, 2016USD ($) | Dec. 31, 2015USD ($) | |
Number of renewal options | item | 2 | ||||
Rent expense | $ 2,000 | $ 2,900 | $ 900 | ||
Operating Leases, Future Minimum Payments Due, Fiscal Year Maturity [Abstract] | |||||
2,018 | 3,290 | ||||
2,019 | 3,382 | ||||
2,020 | 3,762 | ||||
2,021 | 3,868 | ||||
2,022 | 3,977 | ||||
2023 + | 8,295 | ||||
Total minimum lease payments | 26,574 | ||||
75 Sidney Street | |||||
Additional space leased | ft² | 11,000 | ||||
Term of each renewal option | 5 years | ||||
Leasehold incentive received from landlord | 1,300 | ||||
64 Sidney Street | |||||
Leasehold incentive received from landlord | $ 3,500 | ||||
Forecast | |||||
Leasehold improvement receivable | $ 300 |
Commitments and contingencies46
Commitments and contingencies - Significant Agreements - (Details) - Collaborative Arrangement $ in Millions | 1 Months Ended |
Feb. 28, 2015USD ($)shares | |
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | |
Non-refundable upfront payment received | $ 65 |
Premium over fair value on temporary equity issued | 5 |
Genzyme | |
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | |
Non-refundable upfront payment received | 65 |
Premium over fair value on temporary equity issued | 5 |
Amount of reduction in milestone or royalty payments to be made by collaborative partner for each program specified | 10 |
Per Milestone, maximum milestone payments to be received from collaborative partner | 540 |
Regulatory approval milestone | Genzyme | |
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | |
Aggregate maximum milestone payments to be received from collaborative partner | 220 |
Regulatory approval milestone | Minimum | Genzyme | |
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | |
Per Milestone, maximum milestone payments to be received from collaborative partner | 40 |
Regulatory approval milestone | Maximum | Genzyme | |
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | |
Per Milestone, maximum milestone payments to be received from collaborative partner | 50 |
Commercial milestone | Genzyme | |
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | |
Aggregate maximum milestone payments to be received from collaborative partner | 320 |
Commercial milestone | Minimum | Genzyme | |
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | |
Per Milestone, maximum milestone payments to be received from collaborative partner | 50 |
Commercial milestone | Maximum | Genzyme | |
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | |
Per Milestone, maximum milestone payments to be received from collaborative partner | 100 |
Spinal Muscular Atrophy Program | Genzyme | |
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | |
Maximum potential in-kind services to be provided by collaborative partner | 5 |
Option exercise payment required from collaborative partner for each program specified | 30 |
Split Territory Program | Genzyme | |
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | |
Option exercise payment required from collaborative partner for each program specified | 20 |
Redeemable Convertible Preferred Stock Series B | |
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | |
Non-refundable upfront payment received | 30 |
Fair value of temporary equity issued | $ 25 |
Redeemable Convertible Preferred Stock Series B | Genzyme | |
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | |
Temporary equity issued (in shares) | shares | 10,000,000 |
Commitments and contingencies47
Commitments and contingencies - Accounting Analysis - (Details) - USD ($) $ in Thousands | 1 Months Ended | 3 Months Ended | 12 Months Ended | ||||||||||
Sep. 30, 2016 | Feb. 28, 2015 | Dec. 31, 2017 | Sep. 30, 2017 | Jun. 30, 2017 | Mar. 31, 2017 | Dec. 31, 2016 | Sep. 30, 2016 | Jun. 30, 2016 | Mar. 31, 2016 | Dec. 31, 2017 | Dec. 31, 2016 | Dec. 31, 2015 | |
Allocable arrangement consideration allocation | |||||||||||||
Committee Obligations: Alliance Joint Steering Committee ("AJSC") | $ 147 | ||||||||||||
Committee Obligations: Development Advisory Committee ("DAC") | 227 | ||||||||||||
License Option and related deliverables | 5,743 | ||||||||||||
Total allocable arrangement consideration allocations. | 79,256 | ||||||||||||
Income Statement Related Disclosures [Abstract] | |||||||||||||
Revenue adjustment due to development deprioritization | $ 9,500 | ||||||||||||
Collaboration revenue | $ 6,346 | $ 1,148 | $ 1,177 | $ 1,464 | $ 2,362 | $ 3,308 | $ 3,720 | $ 4,830 | 10,135 | $ 14,220 | $ 17,334 | ||
Huntington's Program | |||||||||||||
Allocable arrangement consideration allocation | |||||||||||||
Research and development services | 15,662 | ||||||||||||
Parkinson's Program | |||||||||||||
Allocable arrangement consideration allocation | |||||||||||||
Research and development services | 6,648 | ||||||||||||
Income Statement Related Disclosures [Abstract] | |||||||||||||
Revenue recognized | 5,500 | ||||||||||||
Friedreich's Ataxia Program | |||||||||||||
Allocable arrangement consideration allocation | |||||||||||||
Research and development services | 16,315 | ||||||||||||
Spinal Muscular Atrophy Program | |||||||||||||
Allocable arrangement consideration allocation | |||||||||||||
Research and development services | 32,050 | ||||||||||||
Future Program | |||||||||||||
Allocable arrangement consideration allocation | |||||||||||||
Research and development services | 2,464 | ||||||||||||
Collaborative Arrangement | |||||||||||||
Allocable arrangement consideration | |||||||||||||
Collaborative Arrangement Nonredeemable Up Front Fee Received | 65,000 | ||||||||||||
Additional consideration, premium over fair value on temporary equity issued | 5,000 | ||||||||||||
Genzyme | |||||||||||||
Income Statement Related Disclosures [Abstract] | |||||||||||||
Revenue adjustment due to development deprioritization | 2,100 | ||||||||||||
Genzyme | Collaborative Arrangement | |||||||||||||
Allocable arrangement consideration | |||||||||||||
Collaborative Arrangement Nonredeemable Up Front Fee Received | 65,000 | ||||||||||||
Additional consideration, premium over fair value on temporary equity issued | 5,000 | ||||||||||||
In-kind services and other funding to be provided by collaborative partner | 9,300 | ||||||||||||
Total allocable arrangement consideration | 79,300 | ||||||||||||
Income Statement Related Disclosures [Abstract] | |||||||||||||
Collaboration revenue | 10,100 | $ 14,200 | $ 17,300 | ||||||||||
Deferred Revenue | $ 31,600 | $ 31,600 | |||||||||||
MRIC | |||||||||||||
Income Statement Related Disclosures [Abstract] | |||||||||||||
Payments for shares | $ 2,000 | ||||||||||||
Redeemable Convertible Preferred Stock Series B | Collaborative Arrangement | |||||||||||||
Allocable arrangement consideration | |||||||||||||
Collaborative Arrangement Nonredeemable Up Front Fee Received | $ 30,000 |
Commitments and contingencies48
Commitments and contingencies - Other Agreements - (Details) | 1 Months Ended | 12 Months Ended | ||
Sep. 30, 2016USD ($)multiple | Dec. 31, 2017USD ($)item | Dec. 31, 2016USD ($)item | Dec. 31, 2015USD ($) | |
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | ||||
Number of additional disease indications for which the Company has options to license intellectual property | item | 4 | |||
Aggregate maximum milestone payments to be paid to collaborative partner | $ 18,000,000 | |||
Aggregate upfront fees to be paid to collaborative partner | $ 1,600,000 | |||
Number of milestones achieved | item | 0 | 0 | ||
Patent costs incurred | $ 800,000 | $ 1,800,000 | $ 300,000 | |
Maximum funding | $ 4,000,000 | |||
Funding multiple | multiple | 2.60 | |||
Milestone payment liability | 1,000,000 | |||
Loss Contingency [Abstract] | ||||
Contingency reserves for litigation liabilities | 0 | |||
Minimum | ||||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | ||||
Per Milestone and per disease indication, maximum milestone payments to be paid to collaborative partner | $ 5,000,000 | |||
Number of days written notice required by reporting entity to terminate agreement | 30 days | |||
Maximum | ||||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | ||||
Per Milestone and per disease indication, maximum milestone payments to be paid to collaborative partner | $ 20,000,000 | |||
Number of days written notice required by reporting entity to terminate agreement | 90 days | |||
Various contract research organizations and institutions | Research and development | ||||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | ||||
Payments of upfront license fees | $ 300,000 | $ 600,000 | $ 100,000 |
Commitments and contingencies49
Commitments and contingencies - UMass and MassBiologics Collaboration - (Details) - shares | 12 Months Ended | |
Dec. 31, 2017 | Dec. 31, 2015 | |
Common Stock | ||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | ||
Issuance of common stock (in shares) | 5,175,000 | 5,750,000 |
Redeemable Convertible Prefer50
Redeemable Convertible Preferred Stock - Series A Preferred Stock- (Details) - USD ($) $ / shares in Units, $ in Thousands | Nov. 16, 2015 | Nov. 30, 2015 | Dec. 31, 2015 | Dec. 31, 2014 | Dec. 31, 2015 | Dec. 31, 2017 | Nov. 07, 2017 | Dec. 31, 2016 |
Temporary Equity [Line Items] | ||||||||
Shares issued upon conversion of redeemable convertible preferred stock | 17,647,054 | |||||||
Preferred stock, authorized | 5,000,000 | 5,000,000 | ||||||
Price per share | $ 12 | |||||||
Issuance costs | $ 200 | |||||||
Redeemable Convertible Preferred Stock | ||||||||
Temporary Equity [Line Items] | ||||||||
Shares issued upon conversion of redeemable convertible preferred stock | 17,647,054 | |||||||
Redeemable Convertible Preferred Stock Series A | ||||||||
Temporary Equity [Line Items] | ||||||||
Redeemable convertible preference stock issued (in shares) | 20,000,000 | 45,000,000 | ||||||
Price per share | $ 1 | $ 1 | $ 1 | |||||
Proceeds from issuance of stock, net of issuance costs and exchange of outstanding convertible notes | $ 42,000 | $ 42,000 | ||||||
Issuance costs | 1 | $ 32 | ||||||
Exchange of outstanding convertible notes and accrued interest | $ 2,900 | $ 2,900 | ||||||
Redeemable Convertible Preferred Stock Series B | ||||||||
Temporary Equity [Line Items] | ||||||||
Redeemable convertible preference stock issued (in shares) | 30,000,001 | |||||||
Price per share | $ 3 | $ 3 | ||||||
Proceeds from issuance of stock, net of issuance costs | $ 89,800 | |||||||
Issuance costs | 220 | |||||||
Discount recorded against proceeds received for temporary equity related to payment in excess over fair value | $ 5,000 | |||||||
Common Stock | ||||||||
Temporary Equity [Line Items] | ||||||||
Shares issued upon conversion of redeemable convertible preferred stock | 17,647,054 |
Redeemable Convertible Prefer51
Redeemable Convertible Preferred Stock - Tranche Rights Issued with Series A Preferred Stock - (Details) - USD ($) $ / shares in Units, $ in Thousands | 1 Months Ended | 12 Months Ended | ||
Jan. 31, 2014 | Dec. 31, 2015 | Dec. 31, 2014 | Nov. 07, 2017 | |
Temporary Equity [Line Items] | ||||
Price per share | $ 12 | |||
Fair value of Tranche Rights | ||||
Change in fair value of preferred stock tranche liability | $ 9,750 | |||
Accrued dividends on temporary equity | $ 1,245 | |||
Tranche Right I | ||||
Fair value of Tranche Rights | ||||
Derivative Asset | $ 1,500 | |||
Issuance of stock (in shares) | 18,500,000 | |||
Proceeds from issuance of stock, net of issuance costs | $ 18,500 | |||
Tranche Right II | ||||
Temporary Equity [Line Items] | ||||
Number of additional shares of temporary equity contingently obligated to be purchased by temporary equity holders | 20,000,000 | |||
Fair value of Tranche Rights | ||||
Fair value of Tranche Right Liability | $ 4,100 | |||
Redeemable Convertible Preferred Stock Series A | ||||
Temporary Equity [Line Items] | ||||
Price per share | $ 1 | $ 1 | ||
Fair value of Tranche Rights | ||||
Redeemable convertible preferred stock, initial carrying amount | $ 36,100 | |||
Redeemable Convertible Preferred Stock Series A | Tranche Right I | ||||
Temporary Equity [Line Items] | ||||
Number of additional shares of temporary equity contingently obligated to be purchased by temporary equity holders | 18,500,000 | |||
Price per share | $ 1 | |||
Redeemable Convertible Preferred Stock Series B | ||||
Temporary Equity [Line Items] | ||||
Price per share | $ 3 | |||
Fair value of Tranche Rights | ||||
Proceeds from issuance of stock, net of issuance costs | $ 89,800 |
Redeemable Convertible Prefer52
Redeemable Convertible Preferred Stock - Series B Preferred Stock - (Details) - USD ($) $ / shares in Units, $ in Thousands | 12 Months Ended | |
Dec. 31, 2015 | Nov. 07, 2017 | |
Temporary Equity [Line Items] | ||
Price per share | $ 12 | |
Issuance costs | $ 200 | |
Redeemable Convertible Preferred Stock Series B | ||
Temporary Equity [Line Items] | ||
Redeemable convertible preference stock issued (in shares) | 30,000,001 | |
Price per share | $ 3 | |
Proceeds from issuance of stock, net of issuance costs | $ 89,800 | |
Issuance costs | 220 | |
Discount Recorded Against Proceeds Received For Temporary Equity Related To Excess Over Aggregate Fair Value | $ 5,000 |
Common Stock (Details)
Common Stock (Details) | 12 Months Ended | 35 Months Ended | ||
Dec. 31, 2017Vote$ / sharesshares | Dec. 31, 2016$ / sharesshares | Oct. 31, 2015shares | Dec. 31, 2014shares | |
Class of Stock [Line Items] | ||||
Common Stock, Shares Authorized | 120,000,000 | 120,000,000 | ||
Common stock, par value (in dollars per share) | $ / shares | $ 0.001 | $ 0.001 | ||
Number of votes entitled for each share of common stock | Vote | 1 | |||
Dividends declared | $ / shares | 0 | |||
Dividends paid | $ / shares | $ 0 | |||
Shares available for future issuance (in shares) | 5,933,410 | 5,394,249 | ||
2015 Stock Option Plan | ||||
Class of Stock [Line Items] | ||||
Shares available for future issuance (in shares) | 1,311,812 | |||
Vesting of restricted stock awards | Founder Awards | ||||
Class of Stock [Line Items] | ||||
Shares available for future issuance (in shares) | 366,914 | 628,679 | ||
Vesting of restricted stock awards | 2014 Stock Option and Stock Plan | ||||
Class of Stock [Line Items] | ||||
Shares available for future issuance (in shares) | 191,065 | 539,305 | ||
Exercise of stock options | ||||
Class of Stock [Line Items] | ||||
Shares available for future issuance (in shares) | 3,143,566 | 1,871,237 | ||
Issuance under share based compensation plans | ||||
Class of Stock [Line Items] | ||||
Shares available for future issuance (in shares) | 730,860 | 529,854 | ||
Issuance under share based compensation plans | 2015 Stock Option Plan | ||||
Class of Stock [Line Items] | ||||
Shares available for future issuance (in shares) | 1,501,005 | 1,825,174 |
Stock based compensation - 2014
Stock based compensation - 2014 Stock Option and Grant Plan - (Details) $ / shares in Units, $ in Thousands | Jan. 01, 2018shares | Jan. 01, 2017shares | Jan. 01, 2016shares | Oct. 31, 2015shares | Jan. 31, 2014$ / sharesshares | Dec. 31, 2017USD ($)shares | Dec. 31, 2016USD ($) | Dec. 31, 2015USD ($) | Aug. 31, 2015shares | Apr. 30, 2015shares | Dec. 31, 2014shares | Aug. 31, 2014shares | Apr. 30, 2014shares |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||||||||
Shares available for future issuance (in shares) | 5,933,410 | 5,394,249 | |||||||||||
Stock-based compensation | $ | $ 9,238 | $ 6,310 | $ 4,027 | ||||||||||
Unrecognized stock-based compensation expense | $ | $ 4,500 | ||||||||||||
2014 Stock Option and Grant Plan (2014 Plan) | |||||||||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||||||||
Shares available for future issuance (in shares) | 1,501,005 | ||||||||||||
Number of shares authorized for stock-based compensation plan (in shares) | 2,998,823 | 823,529 | 2,669,411 | 2,047,058 | 2,000,000 | 1,411,764 | |||||||
2015 Employee Stock Purchase Plan | |||||||||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||||||||
New stock options issued | 66,652 | ||||||||||||
Share-based Compensation Arrangement by Share-based Payment Award, Shares Issued in Period | 66,652 | ||||||||||||
Number of shares authorized for stock-based compensation plan (in shares) | 267,658 | 267,492 | 262,362 | ||||||||||
2015 Employee Stock Purchase Plan | Forecast | |||||||||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||||||||
Number of shares authorized for stock-based compensation plan (in shares) | 321,300 | ||||||||||||
2015 Employee Stock Purchase Plan | Minimum | |||||||||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||||||||
Percentage of payroll deduction | 1 | ||||||||||||
2015 Employee Stock Purchase Plan | Maximum | |||||||||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||||||||
Percentage of payroll deduction | 10 | ||||||||||||
2015 Stock Option Plan | |||||||||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||||||||
Shares available for future issuance (in shares) | 1,311,812 | ||||||||||||
Percentage limit of issued and outstanding shares upto which additional shares may be issued | 4 | ||||||||||||
Additional shares added to stock option plan | 1,070,635 | 1,069,971 | |||||||||||
2015 Stock Option Plan | Forecast | |||||||||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||||||||
Additional shares added to stock option plan | 1,285,200 | ||||||||||||
Employees restricted stock | |||||||||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||||||||
Stock-based compensation | $ | $ 500 | 500 | 500 | ||||||||||
Non-employee consultants restricted stock | |||||||||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||||||||
Stock-based compensation | $ | 2,700 | 2,600 | 2,600 | ||||||||||
Founders restricted stock | |||||||||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||||||||
Stock-based compensation | $ | 1,400 | 1,100 | |||||||||||
Restricted stock granted (in shares) | 1,188,233 | ||||||||||||
Restricted stock granted, original issued price (in dollars per share) | $ / shares | $ 0.0425 | ||||||||||||
Founders restricted stock | Vesting period, tranche one | |||||||||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||||||||
Restricted stock granted (in shares) | 835,292 | ||||||||||||
Founders restricted stock | Vesting period, tranche two | |||||||||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||||||||
Restricted stock granted (in shares) | 352,941 | ||||||||||||
Founders restricted stock | Minimum | Vesting period, tranche one | |||||||||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||||||||
Vesting period | 1 year | ||||||||||||
Founders restricted stock | Maximum | Vesting period, tranche one | |||||||||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||||||||
Vesting period | 4 years | ||||||||||||
Stock options, all inclusive | |||||||||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||||||||
Unrecognized stock-based compensation expense | $ | $ 16,100 | ||||||||||||
Stock options granted (in shares) | 1,720,000 | ||||||||||||
Employees and directors stock options | |||||||||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||||||||
Stock-based compensation | $ | $ 5,500 | 3,000 | 700 | ||||||||||
Employees and directors stock options | 2014 Stock Option and Grant Plan (2014 Plan) | |||||||||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||||||||
Stock options granted (in shares) | 1,700,000 | ||||||||||||
Vesting period | 4 years | ||||||||||||
Employees and directors stock options | 2014 Stock Option and Grant Plan (2014 Plan) | Vesting period, tranche one | |||||||||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||||||||
Vesting period | 1 year | ||||||||||||
Vesting percentage | 25.00% | ||||||||||||
Employees and directors stock options | 2014 Stock Option and Grant Plan (2014 Plan) | Vesting period, tranche two | |||||||||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||||||||
Vesting period | 3 years | ||||||||||||
Vesting percentage | 75.00% | ||||||||||||
Non-employees stock options | |||||||||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||||||||
Stock-based compensation | $ | $ 400 | 200 | 300 | ||||||||||
Non-employees stock options | 2014 Stock Option and Grant Plan (2014 Plan) | |||||||||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||||||||
Stock options granted (in shares) | 20,000 | ||||||||||||
Non-employees stock options | 2014 Stock Option and Grant Plan (2014 Plan) | Minimum | |||||||||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||||||||
Vesting period | 1 year | ||||||||||||
Non-employees stock options | 2014 Stock Option and Grant Plan (2014 Plan) | Maximum | |||||||||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||||||||
Vesting period | 4 years | ||||||||||||
Research and development | |||||||||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||||||||
Stock-based compensation | $ | $ 5,367 | 4,296 | 3,218 | ||||||||||
General and administrative | |||||||||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||||||||
Stock-based compensation | $ | $ 3,871 | $ 2,014 | $ 809 |
Stock based compensation - Rest
Stock based compensation - Restricted Stock - (Details) - USD ($) $ / shares in Units, $ in Thousands | 12 Months Ended | ||
Dec. 31, 2017 | Dec. 31, 2016 | Dec. 31, 2015 | |
Restricted stock disclosures | |||
Stock-based compensation | $ 9,238 | $ 6,310 | $ 4,027 |
Unrecognized stock-based compensation expense | $ 4,500 | ||
Remaining weighted-average remaining vesting period | 4 months 17 days | ||
Unvested restricted stock awards | |||
Shares | |||
Balance, beginning (in shares) | 1,167,984 | ||
Vested (in shares) | (573,803) | ||
Repurchased (in shares) | (36,202) | ||
Balance, ending (in shares) | 557,979 | 1,167,984 | |
Weighted Average Grant Date Fair Value Per Share | |||
Balance, beginning (in dollars per share) | $ 0.76 | ||
Vested (in dollars per share) | 0.77 | ||
Repurchased (in dollars per share) | 0.67 | ||
Balance, ending (in dollars per share) | $ 0.70 | $ 0.76 | |
Employees restricted stock | |||
Restricted stock disclosures | |||
Stock-based compensation | $ 500 | $ 500 | 500 |
Non-employee consultants restricted stock | |||
Restricted stock disclosures | |||
Stock-based compensation | $ 2,700 | $ 2,600 | $ 2,600 |
Stock based compensation - Stoc
Stock based compensation - Stock Options - (Details) - USD ($) $ / shares in Units, $ in Thousands | 12 Months Ended | ||
Dec. 31, 2017 | Dec. 31, 2016 | Dec. 31, 2015 | |
Options disclosures | |||
Stock-based compensation | $ 9,238 | $ 6,310 | $ 4,027 |
Stock options, all inclusive | |||
Share-based Compensation Arrangement by Share-based Payment Award, Options, Outstanding [Roll Forward] | |||
Outstanding, beginning balance (in shares) | 1,871,237 | ||
Granted (in shares) | 1,720,000 | ||
Exercised (in shares) | (158,677) | ||
Cancelled or forfeited (in shares) | (288,994) | ||
Outstanding, ending balance (in shares) | 3,143,566 | 1,871,237 | |
Exercisable (in shares) | 955,587 | ||
Vested and expected to vest (in shares) | 3,143,566 | ||
Weighted Average Exercise Price | |||
Outstanding (in dollars per share) | $ 10.21 | ||
Granted (in dollars per share) | 13.07 | ||
Exercised (in dollars per share) | 8.59 | ||
Cancelled or forfeited (in dollars per share) | 10.50 | ||
Outstanding (in dollars per share) | 11.82 | $ 10.21 | |
Exercisable (in dollars per share) | 10.45 | ||
Vested and expected to vest (in dollars per share) | $ 11.82 | ||
Remaining Contractual Life | |||
Outstanding | 8 years 7 months 6 days | ||
Exercisable | 8 years 1 month 6 days | ||
Vested and expected to vest | 8 years 7 months 6 days | ||
Aggregate Intrinsic Value | |||
Outstanding | $ 15,991 | ||
Exercisable | 5,933 | ||
Vested and expected to vest | $ 15,991 | ||
Employees and directors stock options | |||
Options disclosures | |||
Weighted average fair value of grants (in dollars per share) | $ 8.48 | ||
Stock-based compensation | $ 5,500 | $ 3,000 | 700 |
Non-employees stock options | |||
Options disclosures | |||
Weighted average fair value of grants (in dollars per share) | $ 6.88 | ||
Stock-based compensation | $ 400 | $ 200 | $ 300 |
Stock based compensation - Blac
Stock based compensation - Black-Scholes options pricing model - (Details) - USD ($) $ / shares in Units, $ in Thousands | 12 Months Ended | ||
Dec. 31, 2017 | Dec. 31, 2016 | Dec. 31, 2015 | |
Fair value assumptions | |||
Risk-free interest rate | 1.60% | ||
Expected term | 6 years | ||
Expected volatility | 78.60% | ||
Options disclosures | |||
Stock-based compensation | $ 9,238 | $ 6,310 | $ 4,027 |
Unrecognized stock-based compensation expense | $ 4,500 | ||
Remaining weighted-average remaining vesting period | 4 months 17 days | ||
Stock options, all inclusive | |||
Options disclosures | |||
Stock options granted (in shares) | 1,720,000 | ||
Unrecognized stock-based compensation expense | $ 16,100 | ||
Remaining weighted-average remaining vesting period | 2 years 7 months 28 days | ||
Employees and directors stock options | |||
Fair value assumptions | |||
Risk-free interest rate | 2.00% | 1.50% | |
Expected term | 6 years | 6 years | |
Expected volatility | 73.70% | 73.10% | |
Options disclosures | |||
Weighted average fair value of grants (in dollars per share) | $ 8.48 | ||
Stock-based compensation | $ 5,500 | $ 3,000 | $ 700 |
Non-employees stock options | |||
Fair value assumptions | |||
Risk-free interest rate | 2.40% | 2.10% | 2.00% |
Expected term | 8 years 6 months | 9 years 1 month 6 days | 10 years |
Expected volatility | 76.20% | 83.30% | 84.00% |
Options disclosures | |||
Weighted average fair value of grants (in dollars per share) | $ 6.88 | ||
Stock-based compensation | $ 400 | $ 200 | $ 300 |
401(k) Savings Plan (Details)
401(k) Savings Plan (Details) - USD ($) $ in Millions | 12 Months Ended | |
Dec. 31, 2017 | Dec. 31, 2016 | |
401(k) Savings Plan | ||
Employer contribution expense | $ 0.5 | $ 0.3 |
Income taxes (Details)
Income taxes (Details) $ in Thousands | 12 Months Ended |
Dec. 31, 2016USD ($) | |
Current | |
Federal | $ 180 |
Total current | 180 |
Deferred | |
Federal | (111) |
State | (17) |
Total deferred | (128) |
Total tax expense | $ 52 |
Income taxes -Components of def
Income taxes -Components of deferred tax assets and liabilities -(Details) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2017 | Dec. 31, 2016 | Dec. 31, 2015 | |
Income taxes | |||
Tax expense recognized in OCI | $ 128 | ||
Income Tax Expense (Benefit) | 52 | ||
Other Comprehensive Income (Loss), Unrealized Holding Gain (Loss) on Securities Arising During Period, Net of Tax | $ (235) | 199 | $ (251) |
Deferred tax assets: | |||
Deferred Tax Assets, Operating Loss Carryforwards | 24,642 | 8,927 | |
Tax credit carryforwards | 8,832 | 4,284 | |
Deferred rent | 1,458 | 1,964 | |
Deferred revenue | 8,622 | 16,333 | |
Non‑deductible expenses | 817 | 714 | |
Intangibles | 832 | 998 | |
Stock Compensation | 1,361 | 672 | |
Total deferred tax assets | 46,564 | 33,892 | |
Less valuation allowance | (44,953) | (31,361) | |
Net deferred tax assets | 1,611 | 2,531 | |
Deferred tax liabilities | |||
Depreciation and amortization | (1,611) | (2,501) | |
Unrealized gain on available-for-sale securities | 30 | ||
Net deferred taxes | $ 0 | $ 0 |
Income taxes - Operating loss c
Income taxes - Operating loss carryforwards - (Details) - USD ($) | Jan. 01, 2018 | Dec. 31, 2017 | Dec. 31, 2016 | Dec. 31, 2015 |
Operating Loss Carryforwards [Line Items] | ||||
Valuation Allowance | $ 44,953,000 | $ 31,361,000 | ||
Change in the valuation allowance | 13,600,000 | |||
Unrecognized tax benefits | $ 0 | $ 0 | ||
Effective Income Tax Rate Reconciliation, Percent [Abstract] | ||||
Income tax computed at federal statutory tax rate | 34.00% | 34.00% | 34.00% | |
State taxes, net of federal benefit | 6.10% | 5.60% | 4.10% | |
General business credit carryovers | 5.00% | 4.20% | 3.10% | |
Non‑deductible expenses | (4.10%) | (4.00%) | (15.50%) | |
Deferred rate change | (21.80%) | |||
Change in valuation allowance | (19.20%) | (40.20%) | (25.70%) | |
Total | (0.40%) | |||
Forecast | ||||
Effective Income Tax Rate Reconciliation, Percent [Abstract] | ||||
Income tax computed at federal statutory tax rate | 21.00% | |||
Federal | ||||
Operating Loss Carryforwards [Line Items] | ||||
Net operating loss carryforwards | $ 89,800,000 | |||
State | ||||
Operating Loss Carryforwards [Line Items] | ||||
Net operating loss carryforwards | 91,700,000 | |||
Investment Tax Credit expiring in 2018 | 300,000 | |||
Research and development tax credits carryforwards | Federal | ||||
Operating Loss Carryforwards [Line Items] | ||||
Research and development tax credit carryforwards | 6,600,000 | |||
Research and development tax credits carryforwards | State | ||||
Operating Loss Carryforwards [Line Items] | ||||
Research and development tax credit carryforwards | $ 2,300,000 |
Related party transactions (Det
Related party transactions (Details) - USD ($) $ in Millions | 12 Months Ended | ||
Dec. 31, 2017 | Dec. 31, 2016 | Dec. 31, 2015 | |
Investor | Consulting and management services | |||
Related Party Transaction [Line Items] | |||
Total amount of services received | $ 31.8 | $ 0.1 | $ 1.3 |
Genzyme | Collaboration arrangement | |||
Related Party Transaction [Line Items] | |||
Revenue recognized | 10.1 | 14.2 | 17.3 |
Expense recognized | $ 0.1 | $ 1.2 | $ 2.3 |
Selected quarterly financial 63
Selected quarterly financial data (unaudited) (Details) - USD ($) $ / shares in Units, $ in Thousands | 3 Months Ended | 12 Months Ended | |||||||||
Dec. 31, 2017 | Sep. 30, 2017 | Jun. 30, 2017 | Mar. 31, 2017 | Dec. 31, 2016 | Sep. 30, 2016 | Jun. 30, 2016 | Mar. 31, 2016 | Dec. 31, 2017 | Dec. 31, 2016 | Dec. 31, 2015 | |
Quarterly Financial Information Disclosure [Abstract] | |||||||||||
Total revenue | $ 6,346 | $ 1,148 | $ 1,177 | $ 1,464 | $ 2,362 | $ 3,308 | $ 3,720 | $ 4,830 | $ 10,135 | $ 14,220 | $ 17,334 |
Total operating expenses | 18,693 | 24,503 | 19,816 | 18,986 | 16,205 | 13,679 | 13,338 | 12,297 | 81,998 | 55,519 | 37,588 |
Loss from operations | (12,347) | (23,355) | (18,639) | (17,522) | (13,843) | (10,371) | (9,618) | (7,467) | (71,863) | (41,299) | (20,254) |
Net loss attributable to common stockholders | $ (11,828) | $ (23,346) | $ (18,876) | $ (16,648) | $ (14,674) | $ (8,996) | $ (9,335) | $ (7,188) | $ (70,698) | $ (40,193) | $ (38,290) |
Net loss per share attributable to common stock, basic and diluted | $ (0.40) | $ (0.89) | $ (0.73) | $ (0.65) | $ (0.57) | $ (0.35) | $ (0.37) | $ (0.29) | $ (2.64) | $ (1.59) | $ (9.14) |
Subsequent events - (Details)
Subsequent events - (Details) $ in Millions | 1 Months Ended |
Feb. 28, 2018USD ($)ft² | |
75 Sidney Street | |
Subsequent Event [Line Items] | |
Additional space leased | ft² | 11,000 |
75 Sidney Street | Subsequent Event | |
Subsequent Event [Line Items] | |
Additional space leased | ft² | 11,000 |
AbbVie | Collaborative Arrangement | Subsequent Event | |
Subsequent Event [Line Items] | |
Payment received | $ | $ 69 |
Development and regulatory milestone payments | $ | $ 895 |