Document and Entity Information
Document and Entity Information - USD ($) $ in Millions | 12 Months Ended | ||
Dec. 31, 2016 | Mar. 10, 2017 | Jun. 30, 2016 | |
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, 2016 | ||
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 | $ 156.9 | ||
Entity Common Stock, Shares Outstanding | 26,848,943 | ||
Document Fiscal Year Focus | 2,016 | ||
Document Fiscal Period Focus | FY |
Balance Sheets
Balance Sheets - USD ($) $ in Thousands | Dec. 31, 2016 | Dec. 31, 2015 |
Assets, Current | ||
Cash and cash equivalents, fair value | $ 36,641 | $ 31,309 |
Marketable securities | 137,777 | 163,028 |
Prepaid Expense and Other Assets, Current | 4,368 | 1,557 |
Total Current Assets | 178,786 | 195,894 |
Property, Plant and Equipment, Net | 7,893 | 3,234 |
Deposits and other non-current assets | 1,527 | 321 |
Marketable securities | 1,360 | 30,008 |
Total Assets | 189,566 | 229,457 |
Liabilities, Current | ||
Accounts Payable | 550 | 612 |
Accrued expenses | 6,488 | 3,430 |
Deferred rent, current portion | 300 | |
Deferred revenue, current portion | 6,764 | 19,589 |
Total current liabilities | 13,802 | 23,931 |
Deferred rent, net of current portion | 4,999 | 1,015 |
Deferred revenue, net of current portion | 34,818 | 35,393 |
Other non-current liabilities | 25 | 44 |
Total Liabilities | 53,644 | 60,383 |
Stockholders' Equity Attributable to Parent | ||
Common stock, $0.001 par value: 120,000,000 shares authorized; 25,597,912 and 24,930,979 shares issued and outstanding at December 31, 2016 and 2015, respectively | 26 | 25 |
Additional Paid in Capital | 225,963 | 219,122 |
Accumulated other comprehensive gain | (52) | (251) |
Accumulated deficit | (90,015) | (49,822) |
Total stockholders’ deficit | 135,922 | 169,074 |
Total liabilities, redeemable convertible preferred stock and stockholders’ deficit | $ 189,566 | $ 229,457 |
Balance Sheets (Parenthetical)
Balance Sheets (Parenthetical) - $ / shares | Dec. 31, 2016 | Dec. 31, 2015 |
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 | 25,597,912 | 24,930,979 |
Common Stock, Shares, Outstanding | 25,597,912 | 24,930,979 |
Redeemable Preferred Stock | ||
Preferred Stock, par value (in dollars per share) | $ 0.001 | $ 0.001 |
Preferred Stock, Shares Authorized | 5,000,000 | 5,000,000 |
Redeemable Convertible Preferred Stock Series A | ||
Redeemable convertible preferred stock, issued (in shares) | 20,000,000 | |
Preferred Stock, Shares Authorized | 45,000,000 | |
Redeemable Convertible Preferred Stock Series B | ||
Redeemable convertible preferred stock, issued (in shares) | 30,000,001 |
Statement of Operations and Com
Statement of Operations and Comprehensive Loss - USD ($) | 12 Months Ended | ||
Dec. 31, 2016 | Dec. 31, 2015 | Dec. 31, 2014 | |
Income Statement [Abstract] | |||
Collaboration revenue | $ 14,220,000 | $ 17,334,000 | |
Operating Expenses [Abstract] | |||
Research and development | 42,249,000 | 27,679,000 | $ 8,898,000 |
General and administrative | 13,270,000 | 9,909,000 | 5,469,000 |
Total operating expenses | 55,519,000 | 37,588,000 | 14,367,000 |
Operating loss | (41,299,000) | (20,254,000) | (14,367,000) |
Other expense, net | |||
Interest income | 976,000 | 332,000 | (1,000) |
Other expense, net | 182,000 | (9,750,000) | (1,949,000) |
Total other expense, net | 1,158,000 | (9,418,000) | (1,950,000) |
Loss before income taxes | (40,141,000) | (29,672,000) | (16,317,000) |
Income tax benefit | 52,000 | 0 | |
Net loss | (40,193,000) | (29,672,000) | (16,317,000) |
Other comprehensive loss | |||
Net unrealized gain (loss) on available-for-sale-securities, net | 199,000 | (251,000) | |
Other comprehensive loss | 199,000 | (251,000) | |
Comprehensive loss | (39,994,000) | (29,923,000) | (16,317,000) |
Reconciliation of net loss to net loss attributable to common stockholder: | |||
Accretion of redeemable convertible preferred stock to redemption value | (7,373,000) | (1,366,000) | |
Accrued dividends on series A preferred stock | (1,245,000) | ||
Net loss attributable to common stockholders | $ 40,193,000 | $ 38,290,000 | $ 17,683,000 |
Net loss per share attributable to common stock, basic and diluted | $ 1.59 | $ 9.14 | $ 27.83 |
Weighted-average common shares outstanding, basic and diluted | 25,302,414 | 4,191,210 | 635,448 |
Statement of Operations and Co5
Statement of Operations and Comprehensive Loss (Parenthetical) $ in Thousands | 12 Months Ended |
Dec. 31, 2016USD ($) | |
Income Statement [Abstract] | |
Other Comprehensive Income (Loss), Unrealized Holding Gain (Loss) on Securities Arising During Period, Tax | $ 128 |
Redeemable Convertible Preferre
Redeemable Convertible Preferred Stock and Stockholders' Equity (Deficit) - 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, Begining Balance at Dec. 31, 2013 | $ (3,833) | $ (3,833) | |||||
Shares, Outstanding, Beginning Balance at Dec. 31, 2013 | 2,352 | ||||||
Issuance of common stock for services | $ 1 | $ 251 | 252 | ||||
Issuance of common stock for services | 494,118 | ||||||
Initial issuance of Series A redeemable convertible preferred stock, including exchange of convertible notes payable and net of tranche rights and issuance costs | $ 3,878 | ||||||
Initial issuance of Series A redeemable convertible preferred stock, including exchange of convertible notes payable and net of tranche rights and issuance costs ( in shares) | 6,500,000 | ||||||
Issuance of preferred stock, net of discount and issuance costs | $ 18,491 | ||||||
Reclassification of tranche rights upon issuance of Series A redeemable convertible preferred stock | (1,756) | ||||||
Net loss | (16,317) | (16,317) | |||||
Vesting of restricted common stock | 9 | 9 | |||||
Vesting of restricted common stock (in shares) | 318,364 | ||||||
Stock-based compensation expense | 425 | 425 | |||||
Accretion of redeemable convertible preferred stock to redemption value (Preferred Stock) | 1,366 | ||||||
Accretion of redeemable convertible preferred stock to redemption value | 685 | 681 | 1,366 | ||||
Stockholders' Equity, Ending Balance at Dec. 31, 2014 | $ 21,979 | $ 1 | (20,831) | (20,830) | |||
Shares, Outstanding, Ending Balance at Dec. 31, 2014 | 25,000,000 | 814,834 | |||||
Issuance of preferred stock, net of discount and issuance costs (in shares) | 18,500,000 | ||||||
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 | |||||
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) | |||||
Net loss | (29,672) | (29,672) | |||||
Vesting of restricted common stock | 22 | 22 | |||||
Vesting of restricted common stock (in shares) | 717,747 | ||||||
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 (Preferred Stock) | $ 2,149 | $ 5,225 | |||||
Accretion of redeemable convertible preferred stock to redemption value | 2,560 | 4,813 | 7,373 | ||||
Exercises of vested stock options | 10 | 10 | |||||
Exercises of vested stock options (in shares) | 1,344 | ||||||
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 | ||||||
Issuance of preferred stock, net of discount and issuance costs (in shares) | 20,000,000 | 30,000,001 | |||||
Net loss | (40,193) | (40,193) | |||||
Vesting of restricted common stock | $ 1 | 17 | 18 | ||||
Vesting of restricted common stock (in shares) | 601,501 | ||||||
Stock-based compensation expense | 6,310 | 6,310 | |||||
Unrealized loss on available for-sale securities | 199 | 199 | |||||
Exercises of vested stock options | 514 | 514 | |||||
Exercises of vested stock options (in shares) | 65,432 | ||||||
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 |
Redeemable Convertible Preferr7
Redeemable Convertible Preferred Stock and Stockholders' Equity (Deficit) (Parenthetical) - USD ($) $ in Thousands | 6 Months Ended | 12 Months Ended | |
Dec. 31, 2013 | Dec. 31, 2016 | Dec. 31, 2015 | |
Preferred Stock | |||
Issuance costs | $ 9 | ||
Redeemable Convertible Preferred Stock Series A | Preferred Stock | |||
Convertible Notes Payable | $ 2,929 | ||
Tranche Rights | 2,600 | ||
Issuance costs | $ 22 | $ 1 | |
Redeemable Convertible Preferred Stock Series B | |||
Issuance costs | 200 | ||
Redeemable Convertible Preferred Stock Series B | Preferred Stock | |||
Issuance costs | 220 | ||
Discount | $ 5,000 |
Statement of Cash Flows
Statement of Cash Flows - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2016 | Dec. 31, 2015 | Dec. 31, 2014 | |
Cash flow from operating activities | |||
Net loss | $ (40,193) | $ (29,672) | $ (16,317) |
Adjustments to reconcile net loss to net cash used in operating activities: | |||
Stock-based compensation expense | 6,310 | 4,027 | 425 |
Depreciation | 612 | 600 | 184 |
Amortization of premiums and discounts on marketable securities | 696 | 452 | |
Change in fair value of preferred stock tranche liability | 9,750 | 1,949 | |
Non-cash interest on convertible promissory notes payable | 2 | ||
Expense related to shares issued in connection with services performed | 250 | ||
In-kind research and development expenses | 1,182 | 2,316 | |
Other non-cash items | 709 | (277) | 342 |
Changes in operating assets and liabilities: | |||
Prepaid expenses and other current assets | (847) | (234) | (1,185) |
Other non-current assets | 7 | 14 | (7) |
Deferred revenue | (14,582) | 52,666 | |
Accounts payable | (62) | (942) | 604 |
Accrued expenses | 2,636 | 2,788 | 537 |
Other non-current liabilities | (189) | 186 | |
Lease incentive benefit | 1,050 | 1,112 | |
Net cash provided by (used in) operating activities | (42,482) | 41,299 | (11,918) |
Cash flow from investing activities | |||
Purchases of property and equipment | (5,029) | (1,030) | (2,988) |
Change in restricted cash | (421) | (314) | |
Purchases of marketable securities | (112,350) | (220,399) | |
Proceeds from maturities of marketable securities | 165,100 | 26,660 | |
Net cash used in investing activities | 47,300 | (194,769) | (3,302) |
Cash flow from financing activities | |||
Proceeds from issuance of Series A redeemable convertible preferred stock and tranche rights, net of issuance costs | 104,779 | 22,040 | |
Proceeds from issuance of common stock and restricted stock | 72,955 | ||
Proceeds from the exercise of stock options | 514 | 10 | 80 |
Net cash provided by financing activities | 514 | 177,744 | 22,120 |
Net increase in cash and cash equivalents | 5,332 | 24,274 | 6,900 |
Cash and cash equivalents, beginning of period | 31,309 | 7,035 | 135 |
Cash and cash equivalents, end of period | 36,641 | 31,309 | 7,035 |
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 | 1,366 | |
Exchange of promissory notes payable and accrued interest into Series A redeemable convertible preferred stock and tranche rights | $ 2,929 | ||
Conversion of redeemable convertible preferred stock to common stock | $ 150,187 |
Nature of business
Nature of business | 12 Months Ended |
Dec. 31, 2016 | |
Nature of business | |
Nature of business | VOYAGER THERAPEUTICS INC. NOTES TO CONSOLIDATED FINANCIAL STATEMENTS 1. Nature of business Voyager Therapeutics, Inc. (“the Company”) is a clinical‑stage gene therapy company focused on developing life‑changing treatments for patients suffering from severe diseases of the central nervous system (the “CNS”). The Company focuses on CNS diseases where it believes that an adeno‑associated virus (“AAV”) gene therapy approach can have a clinically meaningful impact by either increasing or decreasing the production of a specific protein. The Company has created a product engine that 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 directly to the CNS. The Company’s pipeline consists of six programs for CNS indications, including advanced Parkinson's disease; a monogenic form of amyotrophic lateral sclerosis; Huntington's disease; Friedreich's ataxia; frontotemporal dementia / Alzheimer’s disease; and severe, chronic pain. 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 pre‑clinical 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, 2016, the Company had incurred losses of $90.0 million. The Company has not generated any product revenues 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, 2016 | |
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”). Initial public offering 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 and commissions and offering expenses payable by the Company. 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 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. 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 is expected to impact the year-over-year comparability of the Company’s net loss per share calculations. Use of Estimates The preparation of financial statements in conformity with GAAP requires management to make estimates and assumptions that affect the amounts reported in the 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 when purchased of greater than three months 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. Although available to be sold to meet operating needs or otherwise, securities are generally held through maturity. The cost of securities sold is determined based on the specific identification method for purposes of recording realized gains and losses. During 2016 and 2015, there were no realized gains or losses on sales of investments, and no investments were adjusted for other than temporary declines in fair value Available‑for‑sale securities are maintained by an investment manager and may consist of U.S. Treasury securities and U.S. government agency securities. Available‑for‑sale securities are carried at fair value with the unrealized gains and losses included in other comprehensive income as a component of stockholders’ equity (deficit) 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 unrealized loss through a charge to the Company’s statement of operations and comprehensive loss. Cash, cash equivalents, and marketable securities as of December 31, 2016 and 2015 consist of the following: Amortized Unrealized Unrealized Fair Cost Gains Losses Value (in thousands) As of December 31, 2016 Money market funds included in cash and cash equivalents $ $ $ $ Marketable securities: U.S. Treasury notes U.S. Government agency bonds — — Total debt securities $ $ $ $ Equity securities — Total marketable securities $ $ $ $ Total money market funds and marketable securities $ $ $ $ As of December 31, 2015 Money market funds included in cash and cash equivalents $ $ — $ — $ Marketable securities: U.S. Treasury notes — U.S. Government agency bonds — Total marketable securities $ $ — $ $ Total money market funds and marketable securities $ $ — $ $ The estimated fair value of the Company’s marketable securities balance at December 31, 2016, by contractual maturity, is as follows: Due in one year or less $ Restricted Cash At December 31, 2016 and 2015, the Company maintained restricted cash totaling approximately $0.7 million and $0.3 million, respectively, held in the form of money market accounts as collateral for the Company’s facility lease obligation and credit cards. The balance is included within deposits and other non‑current assets in the accompanying 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, 2016. Revenue Recognition As of December 31, 2016, all of the Company’s revenue is generated exclusively from its collaboration agreement with Sanofi- Genzyme Corporation, a Sanofi company (“ Sanofi- 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 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 of 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 its 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 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 of the Company’s common stock, the Company has based its estimate of expected volatility on the historical volatility of a group of similar companies that are publicly traded. 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, 2016, 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 (in common stock equivalent shares): As of December 31, 2016 2015 2014 Redeemable convertible preferred stock — — Unvested restricted common stock Outstanding stock options — Total 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 and expects to continue to rely 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). The Company will adopt the Revenue ASUs effective January 1, 2018. The Company has not yet determined which adoption method will be utilized. As of December 31, 2016, revenue is generated exclusively from the Company’s collaboration arrangement with Sanofi-Genzyme. The Company is currently evaluating the potential impact that Topic 606 may have on its financial position and results of operations as it relates to this single arrangement. The adoption of the Revenue ASUs is expected to have a significant impact on the Company’s notes to consolidated financial statements and its internal controls over financial reporting. 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 of the guidance on its consolidated financial statements. In March 2016, the FASB issued ASU No. 2016-09, Compensation – Stock Compensation (Topic 718): Improvements to Employee Share-Based Payment Accounting (“ASU 2016-09”), which simplifies several aspects of the accounting for employee share-based payment transactions, including income taxes consequences, classification of awards as either equity or liabilities, and classification in the statement of cash flows. ASU 2016-09 is effective for |
Fair Value Measurements
Fair Value Measurements | 12 Months Ended |
Dec. 31, 2016 | |
Fair Value Measurements | |
Fair value of financial measurements | 3. Fair value measurements All Convertible Preferred Stock converted at the time of the IPO, therefore there were no liabilities outstanding as of December 31, 2016 and 2015. Assets and liabilities measured at fair value on a recurring basis as of December 31, 2016 and 2015 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, 2016 Money market funds included in cash and cash equivalents $ $ $ — $ — Marketable securities: U.S. Treasury notes — — U.S. Government agency securities — — Equity securities — — Total marketable securities $ $ $ $ — Warrants to purchase equity securities — — Total $ $ $ $ — December 31, 2015 Money market funds included in cash and cash equivalents $ $ $ — $ — Marketable securities: U.S. Treasury notes — — U.S. Government agency securities — — Total $ $ $ $ — 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 on the acquisition date, September 2, 2016 and as of December 31, 2016 are as follows: As of September 2, As of December 31, 2016 2016 Risk-free interest rate % % Expected dividend yield — % — % Expected term (in years) Expected volatility % % 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, 2016 | |
Prepaid expense and other current assets | |
Prepaid expenses and other current assets | 4. Prepaid expenses and other current assets Prepaid expense and other current assets consist of the following: As of December 31, 2016 2015 (in thousands) Tenant improvement receivable $ $ — Prepaid research and development contracts Other current assets Accrued interest receivable Prepaid insurance Total $ $ |
Property and equipment, net
Property and equipment, net | 12 Months Ended |
Dec. 31, 2016 | |
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, 2016 2015 (in thousands) Laboratory equipment $ $ Leasehold improvements Furniture and office equipment Construction in progress — Other — Total property and equipment Less: accumulated depreciation Property and equipment, net $ $ The Company recorded $0.6 million, $0.6 million, and $0.2 million in depreciation expense during the years ended December 31, 2016, 2015, and 2014 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, 2016 | |
Accrued expenses | |
Accrued expenses | 6. Accrued expenses Accrued expenses consist of the following: As of December 31, 2016 2015 (in thousands) Research and development costs $ $ Employee compensation costs Accrued goods and services — Professional services Patent costs Other Total $ $ |
Commitments and contingencies
Commitments and contingencies | 12 Months Ended |
Dec. 31, 2016 | |
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 expires 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. The table below includes estimated payments related to the amended 75 Sidney Street lease and the new lease for 64 Sidney Street to December 2024. The Company received leasehold improvement incentives from the landlord totaling $1.3 million and $3.5 million for 75 Sidney Street and 64 Sidney Street, respectively. The Company recorded these incentives as a component of deferred rent and will amortize these incentives as a reduction of rent expense over the life of the lease. The leasehold improvements have been recorded as fixed assets. Rent expense of approximately $2.0 million, $0.9 million, and $0.7 million was incurred during the years ended December 31, 2016, 2015, and 2014, respectively. Future annual minimum lease payments at December 31, 2016 are as follows: Total Minimum Lease Payments (in thousands) 2017 2018 2019 2020 2021 2022+ $ . Significant Agreements Sanofi- Genzyme Collaboration Agreement Summary of Agreement In February 2015, the Company entered into an agreement with Sanofi- Genzyme (“Collaboration Agreement”), which included a non‑refundable upfront payment of $65.0 million. In addition, contemporaneous with entering into the 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 Collaboration Agreement. Under the 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‑AADC01 (“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 VY‑SMN101 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 VY‑AADC01 Program, the VY‑HTT01 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 commenced), 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 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 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 $645.0 million across all programs. 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 $265.0 million. 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 $380.0 million. 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 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 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 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 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 best estimate of selling price (“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 $ Parkinson’s Program Friedreich’s Ataxia Program Spinal Muscular Atrophy Program Future Program Committee Obligations: AJSC DAC License Option and related deliverables Total $ 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 amounts allocated to the license option 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 at the time of exercise. During 2016 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, the Company and Sanofi-Genzyme deprioritized and agreed to pause the development of VY-SMN101, and reduced the estimates related to the amount of “in-kind” services that would be provided by Sanofi-Genzyme. As a result, the Company is no longer recognizing the amount allocated to the VY-SMN101 program. These adjustments were made on a prospective basis and will result in decreases in revenue recognized by $2.4 million per quarter. 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 year ended December 31, 2016 and 2015, the Company recognized $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. As of December 31, 2016, there is $41.6 million of deferred revenue related to the 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 Collaboration Agreement consist of internal and external research and development costs, which primarily include: salaries and benefits, lab supplies and preclinical research studies. The Company does not separately track or segregate the amount of costs incurred under the Collaboration Agreement. All costs are included in research and development expenses in the Company’s statement of operations during the year ended December 31, 2016. The Company estimates that the majority of research and development expense during the period relate to programs for which Sanofi- Genzyme has an option right. University of Massachusetts (“UMass”) and MassBiologics Collaboration In January 2014, UMass and the Company entered into a Collaboration Agreement wherein the Company granted UMass 23,529 shares of common stock, valued at $12.0 thousand, which was recorded as research and development expense. This was the only payment made under the Collaboration Agreement until it was amended by the Collaboration Agreement entered into with UMass and MassBiologics in October 2014. On October 20, 2014, the Company entered into a Collaboration Agreement with UMass and MassBiologics (of the UMass Medical School). Under the agreement, the Company shall (i) fund certain projects that will be conducted by UMass or MassBiologics, (ii) fund certain educational programs of UMass, including post‑doctoral research at the Company’s laboratories beginning in 2015, and (iii) collaborate with MassBiologics to establish scalable processes for manufacturing recombinant adeno‑associated viral vector products using current good manufacturing practices. In November 2014, the parties agreed to the first project under this agreement whereby the Company will fund approximately $2.9 million over a sixteen month period for certain research and development services performed by MassBiologics. The project commenced in January 2015 and completed during 2016. Research and development costs incurred by MassBiologics under the project agreement were expensed by the Company as incurred. MRI Interventions License and Securities Purchase Agreements Summary of Agreement In September 2016, the Company entered into a 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. Under the Securities Purchase Agreement, the Company paid $2.0 million for shares of MRIC common stock and a warrant to purchase additional shares of MRIC common stock. The License Agreement provides for certain rights to MRIC technology, and for MRIC to transfer the rights and know-how to manufacture the ClearPoint System, in order to enable the Company to utilize an alternative supplier for the ClearPoint System for use in the Company’s development and clinical trials. Upon completion of the transfer of the manufacturing and know how, the Company may be obligated to purchase an additional $1.0 million of MRIC common stock at the then fair market value. In addition, the Company has purchased approximately $0.3 million of supplies for use in the ongoing development and clinical trials and has agreed to negotiate a Development and Supply agreement in the future. Accounting Analysis The Company has accounted for its interest in MRIC common stock under ASC 320 “Investments - Debt and Equity Securities” since the Company does not have the ability to control or exercise significant influence over the operations of MRIC. As the MRIC common stock is actively traded, the Company has accounted for the investment in MRIC common stock as an available-for-sale marketable security. The $2.0 million payment was allocated to the common stock and warrant, based on relative fair value. The investment in MRIC common stock was initially recorded at $1.2 million and the warrant was recorded at $0.8 million. The shares of MRIC common stock are classified as non-current available-for-sale marketable securities and the warrants are classified as other assets in the accompanying balance sheet. Remeasurement gains related to the marketable securities are recorded as a component of other comprehensive income and gains related to the warrant are recorded as a component of other expenses in the consolidated statement of operations. The Company concluded that the fair value of the potential obligation to purchase an additional $1.0 million of common stock was insignificant as the share acquisition price will be based on the market price of the MRIC common stock upon completion of the transfer of manufacturing and know how. Other Agreements During 2016, 2015, and 2014, 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, 2016 and 2015, the Company paid $0.6 million and $0.1 million respectively, in up‑front 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 $12.0 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 up‑front fees of up to approximately $1.5 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, 2016 and 2015, 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, 2016 and 2015, the Company incurred $1.8 million and $0.3 million of expense, respectively, related to these reimbursable patent costs which are recorded as general and administrative expense 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, 2016 or December 31, 2015. |
Redeemable Convertible Prefer16
Redeemable Convertible Preferred Stock | 12 Months Ended |
Dec. 31, 2016 | |
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 has authorized preferred stock amounting to 5,000,000 shares as of December 31, 2016 and 2015. The authorized preferred stock was classified under stockholders’ equity at December 31, 2016. 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. Series A Preferred Stock 45,000,000 shares of Series A Preferred Stock were issued 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 are obligated to purchase and the Company is 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 are legally detachable and separately exercisable from the Series A Preferred Stock. Therefore, the Company has allocated the proceeds between the Tranche Rights and the Series A Preferred Stock. As the Series A Preferred Stock is redeemable at the holder’s option, the Tranche Rights are classified as an asset or liability and are initially recorded at fair value. The Tranche Rights are measured at fair value at each reporting period. Since the Tranche Rights are 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 are 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. The Company recognized income of $0.3 million related to the mark to market of Tranche Right I during the year ended December 31, 2014, which is included in other financing expense. 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. The Company recognized expense of $2.2 million related to the mark to market of Tranche Right II during the year ended December 31, 2014, which is included in other financing expense. 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 period ended December 31, 2015, which is 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 exceeds the redemption value, therefore the carrying value is not being subsequently adjusted. However, the Company has reflected accrued dividends of approximately $1.2 million related to this issuance in the net loss attributable to common shareholders for the year ended December 31, 2015. Series B Preferred Stock 30,000,001 shares of Series B Preferred Stock were issued 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. Preferred Stock The rights, preferences, and privileges of the Preferred Stock are listed below: Conversion Shares of Preferred Stock are convertible at any time at the option of the holder into such number of shares as is determined by dividing the original issuance price by the conversion price in effect at the time. Immediately prior to the IPO, the conversion price was $4.25 for Series A Preferred Stock and $12.75 for Series B Preferred Stock, subject to adjustments to reflect the issuance of Common Stock, options, warrants, or other rights to subscribe for or to purchase Common Stock for a consideration per share, less than the conversion price then in effect and subsequent stock dividends and stock splits. In addition any reorganization, recapitalization, reclassification, consolidation or merger in which common stock is exchanged for securities, cash or other property. All outstanding shares of Preferred Stock are automatically converted upon the completion of either an IPO resulting in gross proceeds to the Company of at least $50.0 million or the vote or written consent of 67% of the then outstanding shares of preferred stock. Dividends Holders of Preferred Stock are entitled to receive, before any cash is paid out or set aside for any Common Stock, cash dividends at a rate of 8% of the original purchase price per share annually (the “Accruing Dividends”). The dividends accrue cumulatively on a daily basis and are payable only when, and if, declared by the Board of Directors or upon liquidation or redemption. In addition, the holders of Preferred Stock are entitled to additional dividends based on dividends declared to common stockholders, thereby giving the preferred stockholders the right to participate in undistributed earnings of the Company above the stated per share dividend rate. The preferred stockholders do not have a contractual obligation to share in the losses of the Company. Redemption The Preferred Stock is redeemable at the option of the holder after the redemption date of February 2021. The redemption value of the Preferred Stock is equal to $3.00 per share for Series B Preferred Stock and $1.00 per share for Series A Preferred Stock plus any accrued but unpaid dividends. Accordingly, the Preferred Stock is being accreted to redemption value through its redemption date, including accruals for cumulative dividend rights. If the initial carrying value exceeds the redemption value the carrying value is not adjusted. Liquidation Preference Holders of Series B Preferred Stock and Series A Preferred Stock have preference to the assets of the Company in the event of any voluntary or involuntary liquidation, dissolution or winding‑up of the Company, equal to $3.00 per share for Series B Preferred Stock and $1.00 per share for Series A Preferred Stock, plus any accrued but unpaid dividends, whether or not declared, plus any dividends declared but unpaid thereon, on a pari passu basis. After the payment of the preference amounts to the holders of Series B Preferred Stock and Series A Preferred Stock, the remaining assets of the Company are to be distributed among the holders of Series A Preferred Stock and holders of Common Stock on a pro rata basis. However, if the aggregate amount which the holders of Series A Preferred Stock would be entitled to receive exceeds $2.50 per share (subject to appropriate adjustment in the event of any stock dividend, stock split, combination, reclassification or other similar event) (the “Maximum Participant Amount”), each holder of Series A Preferred Stock will receive the greater of the Maximum Participant Amount or the amount such holder would have received if all shares of Series A Preferred Stock had been converted into Common Stock immediately prior to such liquidation. Voting Rights Holders of Series A Preferred Stock and Series B Preferred Stock are entitled to vote as a single class with the holders of Common Stock on all matters submitted for vote to the Stockholders of the Company. The holders of Preferred Stock are entitled to one vote for each equivalent common share on an as‑converted basis. In addition, the holders of Series A Preferred Stock are entitled to elect two (2) directors. The remaining directors shall be elected by the holders of Common Stock voting together with the holders of the Series B Preferred Stock as one class on an as‑converted basis. The holders of Series A Preferred Stock and Series B Preferred Stock have certain protective rights as defined. These protective rights require the Required Vote before action can be taken to (i) increase or decrease the number of shares of Series A Preferred Stock or Series B Preferred Stock that the Company has authority to issue, (ii) change the par value of the Series A Preferred Stock or Series B Preferred Stock, (iii) amend the Certificate of Incorporation in any way that adversely affects the holders of the Series A Preferred Stock or Series B Preferred Stock. |
Common Stock
Common Stock | 12 Months Ended |
Dec. 31, 2016 | |
Common stock Abstract | |
Stockholders' Equity Note Disclosure [Text Block] | 9. Common stock As of December 31, 2016 and 2015, 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. Cash dividends may not be declared or paid to holders of shares of Common Stock until all accrued unpaid dividends on Series A Preferred Stock and Series B Preferred Stock have been paid in accordance with their terms. No dividends have been declared or paid by the Company since its inception. Liquidation After payment to of their respective liquidation preferences to the holders of shares of Series A Preferred Stock and Series B Preferred Stock, 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, 2016 2015 Shares reserved for vesting of restricted stock awards under the Founder Agreements Shares reserved for vesting of restricted stock awards under the 2014 Option and Stock Plan Shares reserved for exercise of stock options Shares reserved for issuances under the 2015 Stock Option Plan Shares reserved for employee stock purchase plan |
Stock-based compensation
Stock-based compensation | 12 Months Ended |
Dec. 31, 2016 | |
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, or 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 our 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. Any options or awards outstanding under the 2014 Stock Option 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 and 2017, an additional 1,069,971 and 1,070,635 shares, respectively, were added to the Company’s 2015 Stock Option Plan for future issuance. During the year ended December 31, 2016, the Company issued 966,060 stock options to employees and directors and 23,500 stock options to non‑employees. As of December 31, 2016, there were 1,825,174 shares available for future issuance under the 2015 Stock Option Plan. 2014 Stock Option and Grant Plan In January 2014, the Company adopted the Voyager Therapeutics, Inc. 2014 Stock Option and Grant Plan (the “2014 Plan”), under which it may 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, are determined by the Board of Directors and are 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. During the year ended December 31, 2014, the Company granted a total of 1,597,988 shares of restricted stock to employees and 110,960 shares of restricted stock to non‑employee consultants at an original issuance price of $0.04 per share. 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. As of December 31, 2016, management has concluded that the achievement of the performance milestone for one of the three performance-based awards had been met during the year. Accordingly, stock‑based compensation expense in the amount of $1.1 million was recorded as of December 31, 2016. 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 and 2017, 267,492 and 267,658 shares of common stock, respectively, were added to the 2015 ESPP . 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, 2016 2015 2014 (in thousands) Research and development $ $ $ General and administrative Total stock-compensation expense $ $ $ 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, 2015 $ Issued — Vested $ Repurchased $ Unvested restricted common stock as of 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, 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. The expense related to awards granted to employees and non-employees was $0.2 million and $0.2 million, respectively, for the year ended December 31, 2014. As of December 31, 2016, the Company had unrecognized stock‑based compensation expense related to its unvested restricted stock awards of $10.7 million, which is expected to be recognized over the remaining weighted average vesting period of 1.33 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, 2015 $ Granted $ Exercised $ Cancelled or forfeited $ Outstanding at December 31, 2016 $ $ Exercisable at December 31, 2016 $ $ Vested and expected to vest at December 31, 2016 $ $ 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, 2016 was $7.66. The expense related to awards granted to employees and directors was $3.0 million and $0.7 million for the years ended December 31, 2016 and 2015, respectively. There were no stock options granted during the year ended December 31, 2014. 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, 2016 2015 Risk-free interest rate % % Expected dividend yield — % — % Expected term (in years) Expected volatility % % 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, 2016 was $9.98. Unvested options granted to non‑employees are revalued at each measurement period until they vest. The expense related to awards granted to non‑employees was $0.2 million and $0.3 million for the years ended December 31, 2016, and 2015, respectively. There were no stock options granted during the year ended December 31, 2014. 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, 2016 2015 Risk-free interest rate % % Expected dividend yield — % — % Expected term (in years) Expected volatility % % As of December 31, 2016, the Company had unrecognized stock‑based compensation expense related to its unvested stock options of $8.7 million which is expected to be recognized over the remaining weighted average vesting period of 2.71 years. |
401(k) Savings Plan
401(k) Savings Plan | 12 Months Ended |
Dec. 31, 2016 | |
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 did not make any contributions to the 401(k) Plan through December 31, 2015. During 2016, the Company expensed approximately $0.3 million related to employer contributions made during the year. |
Income taxes
Income taxes | 12 Months Ended |
Dec. 31, 2016 | |
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 provision (benefit) for income taxes is as follows: Year ended December 31, 2016 2015 (in thousands) Current Federal $ $ — State — — Total current — Deferred Federal — State — Total deferred — Total tax expense $ $ — For the year ended December 31, 2016, the Company recorded a tax provision of $0.2 million attributed to alternative minimum tax, or AMT, based mainly on the recognition of deferred revenue for income tax purposes related to the Company’s Sanofi-Genzyme Collaboration Agreement. For the year ended December 31, 2016, the Company recorded a tax benefit of $0.1 million. The Company’s overall income tax provision was offset by an income tax benefit recorded to continuing operations of $0.1 million associated with the recognition of the corresponding income tax associated with unrealized gains included in other comprehensive income. The net tax effect resulted in an overall income tax provision recorded to continuing operations of $0.1 million. The corresponding income tax expense has been recorded in other comprehensive income. The Company recorded no income tax provision (benefit) for the year ended December 31, 2015. Intraperiod tax allocation rules require the Company to allocate its provision for income taxes between continuing operations and other categories of earnings, such as other comprehensive income. In periods in which the Company has a year-to-date pre-tax loss from continuing operations and pre-tax income in other categories of earnings, such as other comprehensive income, the Company must allocate the tax provision to the other categories of earnings. The Company then records a related tax benefit in continuing operations. The Company has incurred net operating losses (NOLs) since June 2013. At December 31, 2016, the Company had federal and state net operating loss carryforwards of $23.1 million and $20.4 million, respectively, which expire beginning in 2033. As of December 31, 2016, the Company also had federal and state research and development tax credit carryforwards of $3.1 million and $1.5 million, respectively, which expire beginning in 2028. 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% shareholders 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, that may impact the future annual utilization of existing tax attributes. There could also 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, 2016 and 2015 are as follows: Year ended December 31, 2016 2015 (in thousands) Deferred tax assets: Net operating loss carryforwards $ $ Tax credit carryforwards Deferred rent Deferred revenue — Non-deductible expenses Intangibles Stock compensation Total deferred tax assets Less valuation allowance Net deferred tax assets Deferred tax liabilities Depreciation and amortization Unrealized gain on available-for-sale securities — 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 $31.4 million and $15.2 million has been established at December 31, 2016 and 2015, respectively. The change in valuation allowance was $16.2 million for the year ended December 31, 2016, primarily due to additional operating losses incurred by the Company for the year ended December 31, 2016. The Company net operating loss carryforwards related to excess tax benefits is de minimis as of the December 31, 2016 and is not included in the deferred tax assets. The Company will adopt ASU 2016-09, Improvements to Employee Share-Based Payment Accounting , during the quarter ended March 31, 2017 upon which the net operating loss carryforward deferred tax assets will be increased by the excess tax benefits with a corresponding increase to the Company’s valuation allowance. The adoption of ASU 2016-09 will have no material impact to the Company’s income statement, balance sheet, or retained earnings. 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. At December 31, 2016 and 2015, 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. 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, 2016 2015 2014 Income tax computed at federal statutory tax rate % % % State taxes, net of federal benefit % % % General business credit carryovers % % % Non-deductible expenses % % % Change in valuation allowance % % % Total % — % — % |
Related party transactions
Related party transactions | 12 Months Ended |
Dec. 31, 2016 | |
Related party transactions | |
Related party transactions | 13. Related‑party transactions Since inception, the Company received consulting and management services from one of its investors. In January 2014, the Company issued 470,589 shares of common stock as partial compensation for these services. The fair value of the shares was approximately $0.2 million. The total amount of consulting and management services provided by this investor was approximately $0.1 million, $0.1 million, and $1.3 million during the years ended December 31, 2016, 2015, and 2014, respectively. During the years ended December 31, 2016 and 2015, the Company recognized $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 this period. The Company also recognized $1.2 million and $2.3 million of expense during the years ended December 31, 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, 2016 | |
Quarterly Financial Information Disclosure [Abstract] | |
Selected Quarterly Financial Data | 14. Selected quarterly financial data (unaudited) The following table contains quarterly financial information for 2016 and 2015. 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. 2016 First Second Third Fourth Quarter Quarter Quarter Quarter Total (in thousands, except per share data) Collaboration revenue $ $ $ $ $ Total operating expenses Loss from operations Net loss attributable to common shareholders Net loss per share applicable to common stockholders – basic and diluted $ $ $ $ $ 2015 First Second Third Fourth Quarter Quarter Quarter Quarter Total (in thousands, except per share data) Collaboration revenue $ $ $ $ $ Total operating expenses Loss from operations Net loss attributable to common shareholders Net loss per share applicable to common stockholders – basic and diluted $ $ $ $ $ |
Summary of significant accoun23
Summary of significant accounting policies (Policies) | 12 Months Ended |
Dec. 31, 2016 | |
Summary of significant accounting policies | |
Basis of presentation | 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”). |
Initial public offering | 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 and commissions and offering expenses payable by the Company. 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 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. 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 is expected to impact the year-over-year comparability of the Company’s net loss per share calculations. |
Use of estimates | The preparation of financial statements in conformity with GAAP requires management to make estimates and assumptions that affect the amounts reported in the 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 when purchased of greater than three months 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. Although available to be sold to meet operating needs or otherwise, securities are generally held through maturity. The cost of securities sold is determined based on the specific identification method for purposes of recording realized gains and losses. During 2016 and 2015, there were no realized gains or losses on sales of investments, and no investments were adjusted for other than temporary declines in fair value Available‑for‑sale securities are maintained by an investment manager and may consist of U.S. Treasury securities and U.S. government agency securities. Available‑for‑sale securities are carried at fair value with the unrealized gains and losses included in other comprehensive income as a component of stockholders’ equity (deficit) 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 unrealized loss through a charge to the Company’s statement of operations and comprehensive loss. Cash, cash equivalents, and marketable securities as of December 31, 2016 and 2015 consist of the following: Amortized Unrealized Unrealized Fair Cost Gains Losses Value (in thousands) As of December 31, 2016 Money market funds included in cash and cash equivalents $ $ $ $ Marketable securities: U.S. Treasury notes U.S. Government agency bonds — — Total debt securities $ $ $ $ Equity securities — Total marketable securities $ $ $ $ Total money market funds and marketable securities $ $ $ $ As of December 31, 2015 Money market funds included in cash and cash equivalents $ $ — $ — $ Marketable securities: U.S. Treasury notes — U.S. Government agency bonds — Total marketable securities $ $ — $ $ Total money market funds and marketable securities $ $ — $ $ The estimated fair value of the Company’s marketable securities balance at December 31, 2016, by contractual maturity, is as follows: Due in one year or less $ |
Restricted cash | At December 31, 2016 and 2015, the Company maintained restricted cash totaling approximately $0.7 million and $0.3 million, respectively, held in the form of money market accounts as collateral for the Company’s facility lease obligation and credit cards. The balance is included within deposits and other non‑current assets in the accompanying balance sheets. |
Property and equipement | 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, 2016. |
Revenue Recognition | As of December 31, 2016, all of the Company’s revenue is generated exclusively from its collaboration agreement with Sanofi- Genzyme Corporation, a Sanofi company (“ Sanofi- 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 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 of 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 its 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 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 of the Company’s common stock, the Company has based its estimate of expected volatility on the historical volatility of a group of similar companies that are publicly traded. 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, 2016, 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 (in common stock equivalent shares): As of December 31, 2016 2015 2014 Redeemable convertible preferred stock — — Unvested restricted common stock Outstanding stock options — Total 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 and expects to continue to rely 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). The Company will adopt the Revenue ASUs effective January 1, 2018. The Company has not yet determined which adoption method will be utilized. As of December 31, 2016, revenue is generated exclusively from the Company’s collaboration arrangement with Sanofi-Genzyme. The Company is currently evaluating the potential impact that Topic 606 may have on its financial position and results of operations as it relates to this single arrangement. The adoption of the Revenue ASUs is expected to have a significant impact on the Company’s notes to consolidated financial statements and its internal controls over financial reporting. 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 of the guidance on its consolidated financial statements. In March 2016, the FASB issued ASU No. 2016-09, Compensation – Stock Compensation (Topic 718): Improvements to Employee Share-Based Payment Accounting (“ASU 2016-09”), which simplifies several aspects of the accounting for employee share-based payment transactions, including income taxes consequences, classification of awards as either equity or liabilities, and classification in the statement of cash flows. ASU 2016-09 is effective for annual reporting periods beginning after December 15, 2016, including interim periods within those annual reporting periods. The Company is currently in the process of evaluating the impact of the guidance on its consolidated financial statements. |
Summary of significant accoun24
Summary of significant accounting policies (Tables) | 12 Months Ended |
Dec. 31, 2016 | |
Summary of significant accounting policies | |
Schedule of Marketable securities | Cash, cash equivalents, and marketable securities as of December 31, 2016 and 2015 consist of the following: Amortized Unrealized Unrealized Fair Cost Gains Losses Value (in thousands) As of December 31, 2016 Money market funds included in cash and cash equivalents $ $ $ $ Marketable securities: U.S. Treasury notes U.S. Government agency bonds — — Total debt securities $ $ $ $ Equity securities — Total marketable securities $ $ $ $ Total money market funds and marketable securities $ $ $ $ As of December 31, 2015 Money market funds included in cash and cash equivalents $ $ — $ — $ Marketable securities: U.S. Treasury notes — U.S. Government agency bonds — Total marketable securities $ $ — $ $ Total money market funds and marketable securities $ $ — $ $ |
Estimated fair value of marketable securities by contractual maturity | Amortized Unrealized Unrealized Fair Cost Gains Losses Value (in thousands) As of December 31, 2016 Money market funds included in cash and cash equivalents $ $ $ $ Marketable securities: U.S. Treasury notes U.S. Government agency bonds — — Total debt securities $ $ $ $ Equity securities — Total marketable securities $ $ $ $ Total money market funds and marketable securities $ $ $ $ As of December 31, 2015 Money market funds included in cash and cash equivalents $ $ — $ — $ Marketable securities: U.S. Treasury notes — U.S. Government agency bonds — Total marketable securities $ $ — $ $ Total money market funds and marketable securities $ $ — $ $ The estimated fair value of the Company’s marketable securities balance at December 31, 2016, by contractual maturity, is as follows: Due in one year or less $ |
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 (in common stock equivalent shares): As of December 31, 2016 2015 2014 Redeemable convertible preferred stock — — Unvested restricted common stock Outstanding stock options — Total |
Fair Value Measurements (Tables
Fair Value Measurements (Tables) | 12 Months Ended |
Dec. 31, 2016 | |
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, 2016 and 2015 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, 2016 Money market funds included in cash and cash equivalents $ $ $ — $ — Marketable securities: U.S. Treasury notes — — U.S. Government agency securities — — Equity securities — — Total marketable securities $ $ $ $ — Warrants to purchase equity securities — — Total $ $ $ $ — December 31, 2015 Money market funds included in cash and cash equivalents $ $ $ — $ — Marketable securities: U.S. Treasury notes — — U.S. Government agency securities — — Total $ $ $ $ — |
Assumptions | The assumptions utilized to value the warrants to purchase equity securities on the acquisition date, September 2, 2016 and as of December 31, 2016 are as follows: As of September 2, As of December 31, 2016 2016 Risk-free interest rate % % Expected dividend yield — % — % Expected term (in years) Expected volatility % % |
Prepaid expenses and other cu26
Prepaid expenses and other current assets (Tables) | 12 Months Ended |
Dec. 31, 2016 | |
Prepaid expense and other current assets | |
Schedule of prepaid expense and other current assets | 4. Prepaid expenses and other current assets Prepaid expense and other current assets consist of the following: As of December 31, 2016 2015 (in thousands) Tenant improvement receivable $ $ — Prepaid research and development contracts Other current assets Accrued interest receivable Prepaid insurance Total $ $ |
Property and equipment, net (Ta
Property and equipment, net (Tables) | 12 Months Ended |
Dec. 31, 2016 | |
Property and equipment, net | |
Schedule of property and equipment, net | Property and equipment, net consists of the following: As of December 31, 2016 2015 (in thousands) Laboratory equipment $ $ Leasehold improvements Furniture and office equipment Construction in progress — Other — Total property and equipment Less: accumulated depreciation Property and equipment, net $ $ |
Accrued expenses (Tables)
Accrued expenses (Tables) | 12 Months Ended |
Dec. 31, 2016 | |
Accrued expenses | |
Schedule of accrued expenses | Accrued expenses consist of the following: As of December 31, 2016 2015 (in thousands) Research and development costs $ $ Employee compensation costs Accrued goods and services — Professional services Patent costs Other Total $ $ |
Commitments and contingencies (
Commitments and contingencies (Tables) | 12 Months Ended |
Dec. 31, 2016 | |
Commitments and Contingencies | |
Future annual minimum lease payments | Future annual minimum lease payments at December 31, 2016 are as follows: Total Minimum Lease Payments (in thousands) 2017 2018 2019 2020 2021 2022+ $ |
Allocable arrangement consideration allocation | Unit of Accounting Amount (in thousands) Research and Development Services for: Huntington’s Program $ Parkinson’s Program Friedreich’s Ataxia Program Spinal Muscular Atrophy Program Future Program Committee Obligations: AJSC DAC License Option and related deliverables Total $ |
Common Stock (Tables)
Common Stock (Tables) | 12 Months Ended |
Dec. 31, 2016 | |
Common stock Abstract | |
Schedule of shares reserved for future issuance | Shares Reserved For Future Issuance As of December 31, 2016 2015 Shares reserved for vesting of restricted stock awards under the Founder Agreements Shares reserved for vesting of restricted stock awards under the 2014 Option and Stock Plan Shares reserved for exercise of stock options Shares reserved for issuances under the 2015 Stock Option Plan Shares reserved for employee stock purchase plan |
Stock based compensation (Table
Stock based compensation (Tables) | 12 Months Ended |
Dec. 31, 2016 | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |
Compensation cost recognized for all stock-based compensation awards | Year ended December 31, 2016 2015 2014 (in thousands) Research and development $ $ $ General and administrative Total stock-compensation expense $ $ $ |
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, 2015 $ Issued — Vested $ Repurchased $ Unvested restricted common stock as of December 31, 2016 $ |
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, 2015 $ Granted $ Exercised $ Cancelled or forfeited $ Outstanding at December 31, 2016 $ $ Exercisable at December 31, 2016 $ $ Vested and expected to vest at December 31, 2016 $ $ |
Assumptions | The assumptions utilized to value the warrants to purchase equity securities on the acquisition date, September 2, 2016 and as of December 31, 2016 are as follows: As of September 2, As of December 31, 2016 2016 Risk-free interest rate % % Expected dividend yield — % — % Expected term (in years) Expected volatility % % |
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, 2016 2015 Risk-free interest rate % % Expected dividend yield — % — % Expected term (in years) Expected volatility % % |
Non-employees stock options | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |
Assumptions | As of December 31, 2016 2015 Risk-free interest rate % % Expected dividend yield — % — % Expected term (in years) Expected volatility % % |
Income taxes (Tables)
Income taxes (Tables) | 12 Months Ended |
Dec. 31, 2016 | |
Income taxes | |
Provision (benefit) for income taxes | The provision (benefit) for income taxes is as follows: Year ended December 31, 2016 2015 (in thousands) Current Federal $ $ — State — — Total current — Deferred Federal — State — Total deferred — Total tax expense $ $ — |
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, 2016 and 2015 are as follows: Year ended December 31, 2016 2015 (in thousands) Deferred tax assets: Net operating loss carryforwards $ $ Tax credit carryforwards Deferred rent Deferred revenue — Non-deductible expenses Intangibles Stock compensation Total deferred tax assets Less valuation allowance Net deferred tax assets Deferred tax liabilities Depreciation and amortization Unrealized gain on available-for-sale securities — Net deferred taxes $ — $ — |
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, 2016 2015 2014 Income tax computed at federal statutory tax rate % % % State taxes, net of federal benefit % % % General business credit carryovers % % % Non-deductible expenses % % % Change in valuation allowance % % % Total % — % — % |
Selected quarterly financial 33
Selected quarterly financial data (unaudited) (Tables) | 12 Months Ended |
Dec. 31, 2016 | |
Quarterly Financial Information Disclosure [Abstract] | |
Schedule of Quarterly Financial Information | 2016 First Second Third Fourth Quarter Quarter Quarter Quarter Total (in thousands, except per share data) Collaboration revenue $ $ $ $ $ Total operating expenses Loss from operations Net loss attributable to common shareholders Net loss per share applicable to common stockholders – basic and diluted $ $ $ $ $ 2015 First Second Third Fourth Quarter Quarter Quarter Quarter Total (in thousands, except per share data) Collaboration revenue $ $ $ $ $ Total operating expenses Loss from operations Net loss attributable to common shareholders Net loss per share applicable to common stockholders – basic and diluted $ $ $ $ $ |
Nature of business (Details)
Nature of business (Details) $ in Millions | 12 Months Ended |
Dec. 31, 2016USD ($) | |
Nature of business | |
Net loss since inception | $ 90 |
Summary of significant accoun35
Summary of significant accounting policies - Basis of Presentation - (Details) $ / shares in Units, $ in Millions | Nov. 16, 2015USD ($)$ / sharesshares | Oct. 29, 2015 | Nov. 30, 2015shares | Dec. 31, 2015shares | Dec. 31, 2016shares |
Common stock sales | 24,930,979 | 25,597,912 | |||
Reverse split ratio | 4.25 | ||||
Shares issued upon conversion of redeemable convertible preferred stock | 17,647,054 | 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 accoun36
Summary of significant accounting policies - Marketable Securities - (Details) - USD ($) $ in Thousands | 12 Months Ended | |||
Dec. 31, 2016 | Dec. 31, 2015 | Dec. 31, 2014 | Dec. 31, 2013 | |
Schedule of Investments [Line Items] | ||||
Cash and Cash Equivalents, at Carrying Value | $ 36,641 | $ 31,309 | $ 7,035 | $ 135 |
Total Money Market Funds And Marketable Securities Fair Value | 175,140 | 222,637 | ||
Total Money Market Funds And Marketable Securities Amortized Cost | 175,064 | 222,888 | ||
Gross Unrealized Gain | 142 | |||
Gross Unrealized Loss | 66 | 251 | ||
Available-for-sale Securities, Debt Maturities, Fair Value, Rolling Maturity [Abstract] | ||||
Due in one year or less | 137,777 | |||
Restricted Cash and Investments [Abstract] | ||||
Restricted cash | 300 | |||
Marketable Securities. | ||||
Schedule of Investments [Line Items] | ||||
Marketable debt securities, amortized cost | 139,061 | 193,287 | ||
Marketable securities | 139,137 | 193,036 | ||
Gross Unrealized Gain | 142 | |||
Gross Unrealized Loss | 66 | 251 | ||
Available-for-sale Securities, Debt Maturities, Fair Value, Rolling Maturity [Abstract] | ||||
Total marketable securities | 139,137 | 193,036 | ||
Money market funds | ||||
Schedule of Investments [Line Items] | ||||
Cash and Cash Equivalents, at Carrying Value | 36,003 | 29,601 | ||
Cash and Cash Equivalents, Fair Value Disclosure | 36,003 | 29,601 | ||
U.S. Treasury notes | ||||
Schedule of Investments [Line Items] | ||||
Marketable debt securities, amortized cost | 158,166 | |||
Marketable securities | 157,981 | |||
Gross Unrealized Loss | 185 | |||
Available-for-sale Securities, Debt Maturities, Fair Value, Rolling Maturity [Abstract] | ||||
Total marketable securities | 157,981 | |||
U.S. Treasury notes | Marketable Securities. | ||||
Schedule of Investments [Line Items] | ||||
Marketable debt securities, amortized cost | 130,237 | |||
Marketable securities | 130,173 | |||
Gross Unrealized Gain | 2 | |||
Gross Unrealized Loss | 66 | |||
Available-for-sale Securities, Debt Maturities, Fair Value, Rolling Maturity [Abstract] | ||||
Total marketable securities | 130,173 | |||
U. S. Government agency bonds | Marketable Securities. | ||||
Schedule of Investments [Line Items] | ||||
Marketable debt securities, amortized cost | 7,604 | 35,121 | ||
Marketable securities | 7,604 | 35,055 | ||
Gross Unrealized Loss | 66 | |||
Available-for-sale Securities, Debt Maturities, Fair Value, Rolling Maturity [Abstract] | ||||
Total marketable securities | 7,604 | $ 35,055 | ||
Debt securities | Marketable Securities. | ||||
Schedule of Investments [Line Items] | ||||
Marketable debt securities, amortized cost | 137,841 | |||
Marketable securities | 137,777 | |||
Gross Unrealized Gain | 2 | |||
Gross Unrealized Loss | 66 | |||
Available-for-sale Securities, Debt Maturities, Fair Value, Rolling Maturity [Abstract] | ||||
Total marketable securities | 137,777 | |||
Equity securities | Marketable Securities. | ||||
Schedule of Investments [Line Items] | ||||
Marketable debt securities, amortized cost | 1,220 | |||
Marketable securities | 1,360 | |||
Gross Unrealized Gain | 140 | |||
Available-for-sale Securities, Debt Maturities, Fair Value, Rolling Maturity [Abstract] | ||||
Total marketable securities | $ 1,360 |
Summary of significant accoun37
Summary of significant accounting policies - Deferred Issuance Costs - (Details) - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2016 | Dec. 31, 2015 | |
Summary of significant accounting policies | ||
Other comprehensive loss | $ 199 | $ (251) |
Summary of significant accoun38
Summary of significant accounting policies - Net loss per share - (Details) - USD ($) $ in Thousands | 3 Months Ended | 12 Months Ended | |||||||||
Dec. 31, 2016 | Sep. 30, 2016 | Jun. 30, 2016 | Mar. 31, 2016 | Dec. 31, 2015 | Sep. 30, 2015 | Jun. 30, 2015 | Mar. 31, 2015 | Dec. 31, 2016 | Dec. 31, 2015 | Dec. 31, 2014 | |
Earnings Per Share, Pro Forma [Abstract] | |||||||||||
Antidilutive Securities Excluded from Computation of Earnings Per Share (in common stock equivalent shares) | 5,394,249 | 4,723,719 | 8,461,169 | ||||||||
Add: Changes in fair value of preferred stock tranche liability | $ 9,750 | $ 1,949 | |||||||||
Net loss attributable to common stockholders | $ (14,674) | $ (8,996) | $ (9,335) | $ (7,188) | $ (8,817) | $ (6,914) | $ (6,746) | $ (15,813) | $ (40,193) | (38,290) | (17,683) |
Accretion of redeemable convertible preferred stock to redemption value | (7,373) | $ (1,366) | |||||||||
Add: Accrued dividends on temporary equity | $ 1,245 | ||||||||||
Weighted average number of common shares outstanding, basic and diluted (in shares) | 25,302,414 | 4,191,210 | 635,448 | ||||||||
Redeemable convertible preferred stock | |||||||||||
Earnings Per Share, Pro Forma [Abstract] | |||||||||||
Antidilutive Securities Excluded from Computation of Earnings Per Share (in common stock equivalent shares) | 5,882,352 | ||||||||||
Unvested restricted stock awards | |||||||||||
Earnings Per Share, Pro Forma [Abstract] | |||||||||||
Antidilutive Securities Excluded from Computation of Earnings Per Share (in common stock equivalent shares) | 1,167,984 | 1,818,261 | 2,578,817 | ||||||||
Outstanding stock options | |||||||||||
Earnings Per Share, Pro Forma [Abstract] | |||||||||||
Antidilutive Securities Excluded from Computation of Earnings Per Share (in common stock equivalent shares) | 4,226,265 | 2,905,458 | 0 |
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, 2015 |
Fair value assumptions | |||
Risk-free interest rate | 1.80% | 1.20% | |
Expected term (in years) | 4 years 8 months 12 days | 5 years | |
Expected volatility | 97.50% | 92.30% | |
Estimate of Fair Value | |||
Assets, Fair Value Disclosure [Abstract] | |||
Investments, Fair Value Disclosure | $ 139,137 | ||
Warrant | 792 | ||
Total assets measured at fair value | 175,932 | $ 222,637 | |
Level 1 | Estimate of Fair Value | |||
Assets, Fair Value Disclosure [Abstract] | |||
Investments, Fair Value Disclosure | 131,533 | ||
Total assets measured at fair value | 167,536 | 187,582 | |
Level 2 | Estimate of Fair Value | |||
Assets, Fair Value Disclosure [Abstract] | |||
Investments, Fair Value Disclosure | 7,604 | ||
Warrant | 792 | ||
Total assets measured at fair value | 8,396 | 35,055 | |
U.S. Treasury notes | Estimate of Fair Value | |||
Assets, Fair Value Disclosure [Abstract] | |||
Investments, Fair Value Disclosure | 130,173 | 157,981 | |
U.S. Treasury notes | Level 1 | Estimate of Fair Value | |||
Assets, Fair Value Disclosure [Abstract] | |||
Investments, Fair Value Disclosure | 130,173 | 157,981 | |
U. S. Government agency bonds | Estimate of Fair Value | |||
Assets, Fair Value Disclosure [Abstract] | |||
Investments, Fair Value Disclosure | 7,604 | 35,055 | |
U. S. Government agency bonds | Level 2 | Estimate of Fair Value | |||
Assets, Fair Value Disclosure [Abstract] | |||
Investments, Fair Value Disclosure | 7,604 | 35,055 | |
Equity securities | Estimate of Fair Value | |||
Assets, Fair Value Disclosure [Abstract] | |||
Investments, Fair Value Disclosure | 1,360 | ||
Equity securities | Level 1 | Estimate of Fair Value | |||
Assets, Fair Value Disclosure [Abstract] | |||
Investments, Fair Value Disclosure | 1,360 | ||
Money market funds | |||
Assets, Fair Value Disclosure [Abstract] | |||
Cash and cash equivalents | 36,003 | 29,601 | |
Money market funds | Estimate of Fair Value | |||
Assets, Fair Value Disclosure [Abstract] | |||
Cash and cash equivalents | 36,003 | 29,601 | |
Money market funds | Level 1 | Estimate of Fair Value | |||
Assets, Fair Value Disclosure [Abstract] | |||
Cash and cash equivalents | $ 36,003 | $ 29,601 |
Prepaid expenses and other cu40
Prepaid expenses and other current assets (Details) - USD ($) $ in Thousands | Dec. 31, 2016 | Dec. 31, 2015 |
Prepaid expense and other current assets | ||
Tenant improvement receivable | $ 1,964 | |
Prepaid expenses | 1,094 | $ 340 |
Prepaid research and development contracts | 541 | 382 |
Accrued interest receivable | 339 | 411 |
Other current assets | 430 | 424 |
Total | $ 4,368 | $ 1,557 |
Property and equipment, net (De
Property and equipment, net (Details) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2016 | Dec. 31, 2015 | Dec. 31, 2014 | |
Property, Plant and Equipment [Line Items] | |||
Property, Plant and Equipment, Gross | $ 9,288 | $ 4,018 | |
Less: accumulated depreciation | (1,395) | (784) | |
Property and equipment, net | 7,893 | 3,234 | |
Depreciation expense | 612 | 600 | $ 184 |
Construction in progress | 3,000 | ||
Leasehold improvements | |||
Property, Plant and Equipment [Line Items] | |||
Property, Plant and Equipment, Gross | 1,341 | 1,336 | |
Furniture and office equipment | |||
Property, Plant and Equipment [Line Items] | |||
Property, Plant and Equipment, Gross | 526 | $ 499 | |
Construction in progress | |||
Property, Plant and Equipment [Line Items] | |||
Property, Plant and Equipment, Gross | 3,873 | ||
Other. | |||
Property, Plant and Equipment [Line Items] | |||
Property, Plant and Equipment, Gross | $ 242 |
Accrued expenses (Details)
Accrued expenses (Details) - USD ($) $ in Thousands | Dec. 31, 2016 | Dec. 31, 2015 |
Accrued expenses | ||
Research and development costs | $ 2,384 | $ 1,329 |
Employee compensation costs | 2,399 | 1,338 |
Accrued goods and services | 842 | |
Professional services | 698 | 350 |
Patent costs | 89 | 235 |
Other | 76 | 178 |
Total | $ 6,488 | $ 3,430 |
Commitments and contingencies -
Commitments and contingencies - Operating Leases - (Details) $ in Thousands | 1 Months Ended | 12 Months Ended | ||
Mar. 31, 2014item | Dec. 31, 2016USD ($) | Dec. 31, 2015USD ($) | Dec. 31, 2014USD ($) | |
Number of renewal options | item | 2 | |||
Term of each renewal option | 5 years | |||
Rent expense | $ 900 | $ 2,000 | $ 700 | |
Operating Leases, Future Minimum Payments Due, Fiscal Year Maturity [Abstract] | ||||
2,017 | 3,211 | |||
2,018 | 3,290 | |||
2,019 | 3,382 | |||
2,020 | 3,762 | |||
2,021 | 3,868 | |||
2022 + | 12,273 | |||
Total minimum lease payments | 29,786 | |||
75 Sidney Street | ||||
Leasehold incentive received from landlord | 1,300 | |||
64 Sidney Street | ||||
Leasehold incentive received from landlord | $ 3,500 |
Commitments and contingencies44
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 | 645 |
Regulatory approval milestone | Genzyme | |
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | |
Aggregate maximum milestone payments to be received from collaborative partner | 265 |
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 |
Commerical milestone | Genzyme | |
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | |
Aggregate maximum milestone payments to be received from collaborative partner | 380 |
Commerical milestone | Minimum | Genzyme | |
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | |
Per Milestone, maximum milestone payments to be received from collaborative partner | 50 |
Commerical 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 contingencies45
Commitments and contingencies - Accounting Analysis - (Details) - USD ($) $ in Thousands | 1 Months Ended | 3 Months Ended | 12 Months Ended | ||||||||
Feb. 28, 2015 | Dec. 31, 2016 | Sep. 30, 2016 | Jun. 30, 2016 | Mar. 31, 2016 | Dec. 31, 2015 | Sep. 30, 2015 | Jun. 30, 2015 | Mar. 31, 2015 | 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 on account of change in estimated period of performance | $ 2,400 | ||||||||||
Collaboration revenue | $ 2,362 | $ 3,308 | $ 3,720 | $ 4,830 | $ 4,937 | $ 4,937 | $ 4,884 | $ 2,576 | 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 | ||||||||||
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 | 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 | 14,200 | $ 17,300 | |||||||||
Deferred Revenue | $ 41,600 | $ 41,600 | |||||||||
Redeemable Convertible Preferred Stock Series B | Collaborative Arrangement | |||||||||||
Allocable arrangement consideration | |||||||||||
Collaborative Arrangement Nonredeemable Up Front Fee Received | $ 30,000 |
Commitments and contingencies46
Commitments and contingencies - UMass and MassBiologics Collaboration - (Details) - USD ($) $ in Thousands | 1 Months Ended | 12 Months Ended | |
Nov. 30, 2014 | Jan. 31, 2014 | Dec. 31, 2015 | |
Common Stock | |||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | |||
Stock Issued During Period Shares New Issues | 5,750,000 | ||
University Of Massachusetts (UMass) Collaboration Agreement | Common Stock | |||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | |||
Stock Issued During Period Shares New Issues | 23,529 | ||
Issuance of common stock | $ 12 | ||
Umass and MassBiologics Collaboration Agreement | |||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | |||
Amount funded to collaborative partner for research and development services to be performed. | $ 2,900 | ||
Term of funding to collaborative partner for research and devlopment services to be performed | 16 months |
Commitments and contingencies47
Commitments and contingencies - Other Agreements - (Details) | Jan. 01, 2017USD ($) | Sep. 30, 2016USD ($) | Dec. 31, 2016USD ($)item | Dec. 31, 2015USD ($)item |
MRI Interventions License and Securities Purchase Agreements | ||||
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 | $ 12,000,000 | |||
Aggregate upfront fees to be paid to collaborative partner | $ 1,500,000 | |||
Number of milestones achieved | item | 0 | 0 | ||
Patent costs incurred | $ 1,800,000 | $ 300,000 | ||
Loss Contingency [Abstract] | ||||
Contingency reserves for litigation liabilities | 0 | |||
Minimum | ||||
MRI Interventions License and Securities Purchase Agreements | ||||
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 | ||||
MRI Interventions License and Securities Purchase Agreements | ||||
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 | ||||
MRI Interventions License and Securities Purchase Agreements | ||||
Payments of upfront license fees | $ 100,000 | $ 600,000 | ||
MRI Interventions Inc. | ||||
MRI Interventions License and Securities Purchase Agreements | ||||
Payment for common stock | $ 2,000,000 | |||
Supplies purchased under agreement | $ 300,000 | |||
Investment in common stock | 1,200,000 | |||
Warrant | $ 800,000 | |||
MRI Interventions Inc. | Forecast | ||||
MRI Interventions License and Securities Purchase Agreements | ||||
Payment for common stock | $ 1,000,000 |
Redeemable Convertible Prefer48
Redeemable Convertible Preferred Stock - Series A Preferred Stock- Series A Preferred Stock- (Details) - USD ($) $ / shares in Units, $ in Thousands | Nov. 16, 2015 | Nov. 30, 2015 | Dec. 31, 2016 | Dec. 31, 2015 | Dec. 31, 2014 |
Temporary Equity [Line Items] | |||||
Shares issued upon conversion of redeemable convertible preferred stock | 17,647,054 | 17,647,054 | |||
Exchange of outstanding convertible notes and accrued interest into Series A redeemable convertible preferred stock and tranche rights | $ 2,929 | ||||
Redeemable Convertible Preferred Stock Series A | |||||
Temporary Equity [Line Items] | |||||
Preferred Stock, Shares Authorized | 45,000,000 | 45,000,000 | |||
Redeemable convertible preferred stock, issued (in shares) | 20,000,000 | 18,500,000 | |||
Price per share | $ 1 | $ 1 | |||
Proceeds from issuance of stock, net of issuance costs and exchange of outstanding convertible notes | $ 42,000 | ||||
Proceeds from issuance of stock, net of issuance costs | 32 | ||||
Exchange of outstanding convertible notes and accrued interest into Series A redeemable convertible preferred stock and tranche rights | $ 2,900 | ||||
Redeemable Convertible Preferred Stock Series B | |||||
Temporary Equity [Line Items] | |||||
Redeemable convertible preferred stock, issued (in shares) | 30,000,001 | ||||
Price per share | $ 3 | ||||
Issuance costs | $ 200 | ||||
Discount recorded against proceeds received for temporary equity related to payment in excess over fair value | $ 5,000 | ||||
Redeemable Preferred Stock | |||||
Temporary Equity [Line Items] | |||||
Preferred Stock, Shares Authorized | 5,000,000 | 5,000,000 | |||
Common Stock | |||||
Temporary Equity [Line Items] | |||||
Shares issued upon conversion of redeemable convertible preferred stock | 17,647,054 |
Redeemable Convertible Prefer49
Redeemable Convertible Preferred Stock - Tranche Rights Issued with Series A Preferred Stock - (Details) - USD ($) $ / shares in Units, $ in Thousands | 12 Months Ended | ||
Dec. 31, 2016 | Dec. 31, 2015 | Dec. 31, 2014 | |
Fair value of Tranche Rights | |||
Change in fair value of preferred stock tranche liability | $ 9,750 | $ 1,949 | |
Accrued dividends on temporary equity | 1,245 | ||
Tranche Right I | |||
Fair value of Tranche Rights | |||
Fair value of Tranche Right Asset | 1,500 | ||
Proceeds from issuance of stock, net of issuance costs | 18,500 | ||
Tranche Right II | |||
Fair value of Tranche Rights | |||
Fair value of Tranche Right Liability | $ 4,100 | ||
Other financing expense | Tranche Right I | |||
Fair value of Tranche Rights | |||
Change in fair value of preferred stock tranche liability | 300 | ||
Other financing expense | Tranche Right II | |||
Fair value of Tranche Rights | |||
Change in fair value of preferred stock tranche liability | $ 9,800 | 2,200 | |
Series B preferred stock | |||
Fair value of Tranche Rights | |||
Proceeds from issuance of stock, net of issuance costs | $ 89,800 | ||
Redeemable Convertible Preferred Stock Series A | |||
Temporary Equity [Line Items] | |||
Price per share | $ 1 | $ 1 | |
Fair value of Tranche Rights | |||
Proceeds from issuance of stock, net of issuance costs | $ 32 | ||
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 | ||
Fair value of Tranche Rights | |||
Issuance of stock (in shares) | 18,500,000 | ||
Redeemable Convertible Preferred Stock Series A | 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 | |||
Redeemable convertible preferred stock, initial carrying amount | $ 36,100 | ||
Accrued dividends on temporary equity | $ 1,200 | ||
Redeemable Convertible Preferred Stock Series B | |||
Temporary Equity [Line Items] | |||
Price per share | $ 3 |
Redeemable Convertible Prefer50
Redeemable Convertible Preferred Stock - Series B Preferred Stock - (Details) - USD ($) $ / shares in Units, $ in Thousands | 12 Months Ended | ||
Dec. 31, 2016 | Dec. 31, 2015 | Dec. 31, 2014 | |
Temporary Equity [Line Items] | |||
Exchange of outstanding convertible notes and accrued interest into Series A redeemable convertible preferred stock and tranche rights | $ 2,929 | ||
Series B preferred stock | |||
Temporary Equity [Line Items] | |||
Proceeds from issuance of stock, net of issuance costs | $ 89,800 | ||
Redeemable Convertible Preferred Stock Series B | |||
Temporary Equity [Line Items] | |||
Redeemable convertible preferred stock, issued (in shares) | 30,000,001 | ||
Price per share | $ 3 | ||
Issuance costs | $ 200 | ||
Discount Recorded Against Proceeds Received For Temporary Equity Related To Excess Over Aggregate Fair Value | $ 5,000 |
Redeemable Convertible Prefer51
Redeemable Convertible Preferred Stock - Conversion - (Details) $ / shares in Units, $ in Millions | 12 Months Ended |
Dec. 31, 2016USD ($)directorVote$ / shares | |
Temporary Equity [Line Items] | |
Minimum gross proceeds from IPO that triggers conversion of temporary equity | $ | $ 50 |
Minimum vote or written consent from outstanding temporary equity shares that triggers conversion of temporary equity (as a percent) | 67.00% |
Dividend rate (as a percent) | 8.00% |
Number of votes for each equivalent common share of temporary equity | Vote | 1 |
Number of directors temporary equity holders may elect | director | 2 |
Redeemable Convertible Preferred Stock Series A | |
Temporary Equity [Line Items] | |
Temporary equity conversion price (in dollars per share) | $ 4.25 |
Temporary equity liquidation preference (in dollars per share) | 1 |
Minimum aggregate liquidation preference payments to temporary equity holders that trigger specified alternate preference payout (in dollars per share) | 2.50 |
Redeemable Convertible Preferred Stock Series B | |
Temporary Equity [Line Items] | |
Temporary equity conversion price (in dollars per share) | 12.75 |
Temporary equity redemption price (in dollars per share) | $ 3 |
Common Stock (Details)
Common Stock (Details) | 12 Months Ended | 35 Months Ended | |||
Dec. 31, 2016Vote$ / sharesshares | Dec. 31, 2016$ / sharesshares | Dec. 31, 2015$ / sharesshares | Oct. 31, 2015shares | Dec. 31, 2014$ / sharesshares | |
Class of Stock [Line Items] | |||||
Common Stock, Shares Authorized | 120,000,000 | 120,000,000 | 120,000,000 | ||
Common stock, par value (in dollars per share) | $ / shares | $ 0.001 | $ 0.001 | $ 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) | 5,394,249 | 5,394,249 | 4,723,719 | ||
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) | 628,679 | 628,679 | 853,680 | ||
Vesting of restricted stock awards | 2014 Stock Option and Stock Plan | |||||
Class of Stock [Line Items] | |||||
Shares available for future issuance (in shares) | 539,305 | 539,305 | 964,581 | ||
Exercise of stock options | |||||
Class of Stock [Line Items] | |||||
Shares available for future issuance (in shares) | 1,871,237 | 1,871,237 | 1,022,617 | ||
Issuance under share based compensation plans | |||||
Class of Stock [Line Items] | |||||
Shares available for future issuance (in shares) | 529,854 | 529,854 | 262,362 | ||
Issuance under share based compensation plans | 2015 Stock Option Plan | |||||
Class of Stock [Line Items] | |||||
Shares available for future issuance (in shares) | 1,825,174 | 1,825,174 | 1,620,479 |
Stock based compensation - 2014
Stock based compensation - 2014 Stock Option and Grant Plan - (Details) | Jan. 01, 2017shares | Jan. 01, 2016shares | Oct. 31, 2015shares | Jan. 31, 2014$ / sharesshares | Dec. 31, 2016USD ($)$ / sharesshares | Dec. 31, 2015USD ($) | Dec. 31, 2014USD ($)shares | Aug. 31, 2015shares | Apr. 30, 2015shares | Aug. 31, 2014shares | Apr. 30, 2014shares |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||||||
Shares available for future issuance (in shares) | 5,394,249 | 4,723,719 | |||||||||
Stock-based compensation | $ | $ 6,310,000 | $ 4,027,000 | $ 425,000 | ||||||||
Unrecognized stock-based compensation expense | $ | $ 10,700,000 | ||||||||||
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) | 1,825,174 | ||||||||||
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 | |||||
Restricted stock granted, original issued price (in dollars per share) | $ / shares | $ 0.04 | ||||||||||
2015 Employee Stock Purchase Plan | |||||||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||||||
Additional shares added to stock option plan | 267,658 | 267,492 | |||||||||
Number of shares authorized for stock-based compensation plan (in shares) | 262,362 | ||||||||||
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 | |||||||||
Employees restricted stock | |||||||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||||||
Stock-based compensation | $ | $ 500,000 | 500,000 | 200,000 | ||||||||
Employees restricted stock | 2014 Stock Option and Grant Plan - Employees | |||||||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||||||
Restricted stock granted (in shares) | 1,597,988 | ||||||||||
Non-employee consultants restricted stock | |||||||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||||||
Stock-based compensation | $ | $ 2,600,000 | 2,600,000 | $ 200,000 | ||||||||
Non-employee consultants restricted stock | 2014 Stock Option and Grant Plan - Non-employee Consultants | |||||||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||||||
Restricted stock granted (in shares) | 110,960 | ||||||||||
Founders restricted stock | |||||||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||||||
Stock-based compensation | $ | $ 1,100,000 | ||||||||||
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 | $ | $ 8,700,000 | ||||||||||
Stock options granted (in shares) | 989,560 | 0 | |||||||||
Employees and directors stock options | |||||||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||||||
Stock-based compensation | $ | $ 3 | 700,000 | |||||||||
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) | 966,060 | ||||||||||
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 | $ | $ 200,000 | $ 300,000 | |||||||||
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) | 23,500 | ||||||||||
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 |
Stock based compensation - Foun
Stock based compensation - Founder Awards - (Details) - USD ($) $ / shares in Units, $ in Thousands | Jan. 01, 2017 | Jan. 01, 2016 | Jan. 31, 2014 | Dec. 31, 2016 | Dec. 31, 2015 | Dec. 31, 2014 | Oct. 31, 2015 |
2015 Employee Stock Purchase Plan | |||||||
Stock-based compensation | $ 6,310 | $ 4,027 | $ 425 | ||||
Research and development | |||||||
2015 Employee Stock Purchase Plan | |||||||
Stock-based compensation | 4,296 | 3,218 | 297 | ||||
General and administrative | |||||||
2015 Employee Stock Purchase Plan | |||||||
Stock-based compensation | 2,014 | $ 809 | $ 128 | ||||
Founders restricted stock | |||||||
Share-based Compensation Arrangement by Share-based Payment Award, Compensation Cost [Line Items] | |||||||
Restricted stock granted (in shares) | 1,188,233 | ||||||
Restricted stock granted, original issued price (in dollars per share) | $ 0.0425 | ||||||
2015 Employee Stock Purchase Plan | |||||||
Stock-based compensation | $ 1,100 | ||||||
Vesting period, tranche one | Founders restricted stock | |||||||
Share-based Compensation Arrangement by Share-based Payment Award, Compensation Cost [Line Items] | |||||||
Restricted stock granted (in shares) | 835,292 | ||||||
Vesting period, tranche two | Founders restricted stock | |||||||
Share-based Compensation Arrangement by Share-based Payment Award, Compensation Cost [Line Items] | |||||||
Restricted stock granted (in shares) | 352,941 | ||||||
Minimum | Vesting period, tranche one | Founders restricted stock | |||||||
Share-based Compensation Arrangement by Share-based Payment Award, Compensation Cost [Line Items] | |||||||
Vesting period | 1 year | ||||||
Maximum | Vesting period, tranche one | Founders restricted stock | |||||||
Share-based Compensation Arrangement by Share-based Payment Award, Compensation Cost [Line Items] | |||||||
Vesting period | 4 years | ||||||
2015 Employee Stock Purchase Plan | |||||||
2015 Employee Stock Purchase Plan | |||||||
Number of shares authorized for stock-based compensation plan (in shares) | 262,362 | ||||||
Additional shares added to stock option plan | 267,658 | 267,492 | |||||
2015 Employee Stock Purchase Plan | Minimum | |||||||
2015 Employee Stock Purchase Plan | |||||||
Stock purchase plan payroll deduction percent | 1.00% | ||||||
2015 Employee Stock Purchase Plan | Maximum | |||||||
2015 Employee Stock Purchase Plan | |||||||
Stock purchase plan payroll deduction percent | 10.00% |
Stock based compensation - Rest
Stock based compensation - Restricted Stock - (Details) - USD ($) $ / shares in Units, $ in Thousands | 12 Months Ended | ||
Dec. 31, 2016 | Dec. 31, 2015 | Dec. 31, 2014 | |
Restricted stock disclosures | |||
Stock-based compensation | $ 6,310 | $ 4,027 | $ 425 |
Unrecognized stock-based compensation expense | $ 10,700 | ||
Remaining weighted-average remaining vesting period | 1 year 3 months 29 days | ||
Unvested restricted stock awards | |||
Shares | |||
Balance, beginning (in shares) | 1,818,261 | ||
Vested (in shares) | (601,501) | ||
Repurchased (in shares) | (48,776) | ||
Balance, ending (in shares) | 1,167,984 | 1,818,261 | |
Weighted Average Grant Date Fair Value Per Share | |||
Balance, beginning (in dollars per share) | $ 0.76 | ||
Vested (in dollars per share) | 0.79 | ||
Repurchased (in dollars per share) | 1.11 | ||
Balance, ending (in dollars per share) | $ 0.76 | $ 0.76 | |
Employees restricted stock | |||
Restricted stock disclosures | |||
Stock-based compensation | $ 500 | $ 500 | 200 |
Non-employee consultants restricted stock | |||
Restricted stock disclosures | |||
Stock-based compensation | $ 2,600 | $ 2,600 | $ 200 |
Stock based compensation - Stoc
Stock based compensation - Stock Options - (Details) - USD ($) | 12 Months Ended | ||
Dec. 31, 2016 | Dec. 31, 2015 | Dec. 31, 2014 | |
Options disclosures | |||
Stock-based compensation | $ 6,310,000 | $ 4,027,000 | $ 425,000 |
Stock options, all inclusive | |||
Share-based Compensation Arrangement by Share-based Payment Award, Options, Outstanding [Roll Forward] | |||
Outstanding, beginning balance (in shares) | 1,022,617 | ||
Granted (in shares) | 989,560 | 0 | |
Exercised (in shares) | (65,432) | ||
Cancelled or forfeited (in shares) | (75,508) | ||
Outstanding, ending balance (in shares) | 1,871,237 | 1,022,617 | |
Exerciseable (in shares) | 458,737 | ||
Vested and expected to vest (in shares) | 1,871,237 | ||
Weighted Average Exercise Price | |||
Oustanding (in dollars per share) | $ 8.35 | ||
Granted (in dollars per share) | 11.90 | ||
Exercised (in dollars per share) | 7.85 | ||
Cancelled or forfeited (in dollars per share) | 9.62 | ||
Oustanding (in dollars per share) | 10.21 | $ 8.35 | |
Exercisable (in dollars per share) | 9.02 | ||
Vested and expected to vest (in dollars per share) | $ 10.21 | ||
Remaining Contractual Life | |||
Outstanding | 8 years 10 months 24 days | ||
Exerciseable | 8 years 6 months | ||
Vested and expected to vest | 8 years 10 months 24 days | ||
Aggregate Intrinsic Value | |||
Oustanding | $ 5,232,000 | ||
Exerciseable | 1,759,000 | ||
Vested and expected to vest | $ 5,232,000 | ||
Employees and directors stock options | |||
Options disclosures | |||
Weighted average fair value of grants (in dollars per share) | $ 7.66 | ||
Stock-based compensation | $ 3 | $ 700,000 | |
Non-employees stock options | |||
Options disclosures | |||
Weighted average fair value of grants (in dollars per share) | $ 9.98 | ||
Stock-based compensation | $ 200,000 | $ 300,000 |
Stock based compensation - Blac
Stock based compensation - Black-Scholes options pricing model - (Details) - USD ($) | 12 Months Ended | ||
Dec. 31, 2016 | Dec. 31, 2015 | Dec. 31, 2014 | |
Options disclosures | |||
Stock-based compensation | $ 6,310,000 | $ 4,027,000 | $ 425,000 |
Unrecognized stock-based compensation expense | $ 10,700,000 | ||
Remaining weighted-average remaining vesting period | 1 year 3 months 29 days | ||
Stock options, all inclusive | |||
Options disclosures | |||
Stock options granted (in shares) | 989,560 | 0 | |
Unrecognized stock-based compensation expense | $ 8,700,000 | ||
Remaining weighted-average remaining vesting period | 2 years 8 months 16 days | ||
Employees and directors stock options | |||
Fair value assumptions | |||
Risk-free interest rate | 1.50% | 1.60% | |
Expected term | 6 years | 6 years | |
Expected volatility | 73.10% | 78.60% | |
Options disclosures | |||
Weighted average fair value of grants (in dollars per share) | $ 7.66 | ||
Stock-based compensation | $ 3 | $ 700,000 | |
Non-employees stock options | |||
Fair value assumptions | |||
Risk-free interest rate | 2.10% | 2.00% | |
Expected term | 9 years 1 month 6 days | 10 years | |
Expected volatility | 83.30% | 84.00% | |
Options disclosures | |||
Weighted average fair value of grants (in dollars per share) | $ 9.98 | ||
Stock-based compensation | $ 200,000 | $ 300,000 |
Income taxes (Details)
Income taxes (Details) - USD ($) | 6 Months Ended | 12 Months Ended | |
Dec. 31, 2013 | Dec. 31, 2016 | Dec. 31, 2015 | |
Income Tax Expense (Benefit), Continuing Operations, by Jurisdiction [Abstract] | |||
Current Federal Tax Expense (Benefit) | $ 180,000 | ||
Current Income Tax Expense (Benefit), Total | 180,000 | ||
Deferred Federal Income Tax Expense (Benefit) | (111,000) | ||
Deferred State and Local Income Tax Expense (Benefit) | (17,000) | ||
Deferred Income Tax Expense (Benefit), Total | (128,000) | ||
Income Tax Expense (Benefit), Total | 52,000 | $ 0 | |
Accrued income tax provision | 200,000 | ||
Deferred tax assets: | |||
Deferred Tax Assets, Operating Loss Carryforwards | 8,927,000 | 12,521,000 | |
Tax credit carryforwards | 4,284,000 | 1,969,000 | |
Deferred rent | 1,964,000 | 516,000 | |
Deferred revenue | 16,333,000 | ||
Non‑deductible expenses | 714,000 | 577,000 | |
Intangibles | 998,000 | 376,000 | |
Stock Compensation | 672,000 | 115,000 | |
Total deferred tax assets | 33,892,000 | 16,074,000 | |
Less valuation allowance | (31,361,000) | (15,207,000) | |
Net deferred tax assets | 2,531,000 | 867,000 | |
Deferred tax liabilities—depreciation and amortization | (2,501,000) | (867,000) | |
Unrealized gain on available-for-sale securities | (30,000) | ||
Net deferred taxes | 0 | 0 | |
Change in the valuation allowance | 16,200,000 | ||
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 | 5.50% | 5.60% | 4.10% |
General business credit carryovers | 2.20% | 4.20% | 3.10% |
Non‑deductible expenses | (5.00%) | (4.00%) | (15.50%) |
Change in valuation allowance | (36.70%) | (40.20%) | (25.70%) |
Effective income tax rate | (0.40%) | ||
Tax Year 2033 | Federal | |||
Income Tax Expense (Benefit), Continuing Operations, by Jurisdiction [Abstract] | |||
Net operating loss carryforwards | $ 23,100,000 | ||
Tax Year 2033 | State | |||
Income Tax Expense (Benefit), Continuing Operations, by Jurisdiction [Abstract] | |||
Net operating loss carryforwards | 20,400,000 | ||
Research and development tax credits carryforwards | Tax Year 2028 | Federal | |||
Income Tax Expense (Benefit), Continuing Operations, by Jurisdiction [Abstract] | |||
Tax credits carryforwards | 3,100,000 | ||
Research and development tax credits carryforwards | Tax Year 2028 | State | |||
Income Tax Expense (Benefit), Continuing Operations, by Jurisdiction [Abstract] | |||
Tax credits carryforwards | $ 1,500,000 |
Related party transactions (Det
Related party transactions (Details) - USD ($) $ in Thousands | 1 Months Ended | 12 Months Ended | ||
Jan. 31, 2014 | Dec. 31, 2016 | Dec. 31, 2015 | Dec. 31, 2014 | |
Related Party Transaction [Line Items] | ||||
Fair value of common stock issued as partial compensation for consulting and management services | $ 252 | |||
Investor | Consulting and management services | ||||
Related Party Transaction [Line Items] | ||||
Common stock issued as partial compensation for consulting and management services (in shares) | 470,589 | |||
Fair value of common stock issued as partial compensation for consulting and management services | $ 200 | |||
Total amount of services received | $ 100 | $ 100 | $ 1,300 | |
Genzyme | Collaboration arrangement | ||||
Related Party Transaction [Line Items] | ||||
Revenue recognized | 14,200 | 17,300 | ||
Expense recognized | $ 1,200 | $ 2,300 |
Selected quarterly financial 60
Selected quarterly financial data (unaudited) (Details) - USD ($) $ / shares in Units, $ in Thousands | 3 Months Ended | 12 Months Ended | |||||||||
Dec. 31, 2016 | Sep. 30, 2016 | Jun. 30, 2016 | Mar. 31, 2016 | Dec. 31, 2015 | Sep. 30, 2015 | Jun. 30, 2015 | Mar. 31, 2015 | Dec. 31, 2016 | Dec. 31, 2015 | Dec. 31, 2014 | |
Quarterly Financial Information Disclosure [Abstract] | |||||||||||
Total revenue | $ 2,362 | $ 3,308 | $ 3,720 | $ 4,830 | $ 4,937 | $ 4,937 | $ 4,884 | $ 2,576 | $ 14,220 | $ 17,334 | |
Total operating expenses | 16,205 | 13,679 | 13,338 | 12,297 | 12,377 | 8,956 | 8,851 | 7,404 | 55,519 | 37,588 | $ 14,367 |
Loss from operations | (13,843) | (10,371) | (9,618) | (7,467) | (7,440) | (4,019) | (3,967) | (4,828) | (41,299) | (20,254) | (14,367) |
Net loss attributable to common stockholders | $ (14,674) | $ (8,996) | $ (9,335) | $ (7,188) | $ (8,817) | $ (6,914) | $ (6,746) | $ (15,813) | $ (40,193) | $ (38,290) | $ (17,683) |
Net loss per share attributable to common stock, basic and diluted | $ (0.57) | $ (0.35) | $ (0.37) | $ (0.29) | $ (0.67) | $ (5.25) | $ (5.80) | $ (15.81) | $ (1.59) | $ (9.14) | $ (27.83) |