Document and Entity Information
Document and Entity Information - shares | 9 Months Ended | |
Sep. 30, 2016 | Nov. 04, 2016 | |
Document And Entity Information [Abstract] | ||
Document Type | 10-Q | |
Amendment Flag | false | |
Document Period End Date | Sep. 30, 2016 | |
Document Fiscal Year Focus | 2,016 | |
Document Fiscal Period Focus | Q3 | |
Trading Symbol | RGNX | |
Entity Registrant Name | REGENXBIO Inc. | |
Entity Central Index Key | 1,590,877 | |
Current Fiscal Year End Date | --12-31 | |
Entity Filer Category | Non-accelerated Filer | |
Entity Common Stock, Shares Outstanding | 26,475,379 |
Balance Sheets
Balance Sheets - USD ($) $ in Thousands | Sep. 30, 2016 | Dec. 31, 2015 |
Current assets | ||
Cash and cash equivalents | $ 28,108 | $ 54,116 |
Marketable securities | 63,662 | 60,025 |
Accounts receivable | 679 | 2,136 |
Prepaid expenses | 2,171 | 1,020 |
Other current assets | 2,000 | 851 |
Total current assets | 96,620 | 118,148 |
Marketable securities | 93,087 | 102,226 |
Property and equipment, net | 5,804 | 538 |
Cost method investments | 300 | |
Restricted cash | 225 | |
Other assets | 239 | 168 |
Total assets | 195,975 | 221,380 |
Current liabilities | ||
Accounts payable | 5,376 | 1,014 |
Accrued expenses and other current liabilities | 9,006 | 3,198 |
Advance payments | 127 | |
Total current liabilities | 14,382 | 4,339 |
Deferred rent, net of current portion | 1,367 | 233 |
Total liabilities | 15,749 | 4,572 |
Commitments and contingencies (Note 6) | ||
Stockholders’ equity | ||
Preferred stock; $0.0001 par value; 10,000 shares authorized, and no shares issued and outstanding at September 30, 2016 and December 31, 2015 | ||
Common stock; $0.0001 par value; 100,000 shares authorized at September 30, 2016 and December 31, 2015; 26,475 and 26,313 shares issued and outstanding at September 30, 2016 and December 31, 2015, respectively | 3 | 3 |
Additional paid-in capital | 274,349 | 269,144 |
Accumulated other comprehensive income (loss) | 853 | (719) |
Accumulated deficit | (94,979) | (51,620) |
Total stockholders’ equity | 180,226 | 216,808 |
Total liabilities and stockholders’ equity | $ 195,975 | $ 221,380 |
Balance Sheets (Parenthetical)
Balance Sheets (Parenthetical) - $ / shares | Sep. 30, 2016 | Dec. 31, 2015 |
Statement Of Financial Position [Abstract] | ||
Preferred stock, par value | $ 0.0001 | $ 0.0001 |
Preferred stock, shares authorized | 10,000,000 | 10,000,000 |
Preferred stock, shares issued | 0 | 0 |
Preferred stock, shares outstanding | 0 | 0 |
Common stock, par value | $ 0.0001 | $ 0.0001 |
Common stock, shares authorized | 100,000,000 | 100,000,000 |
Common stock, shares issued | 26,475,000 | 26,313,000 |
Common stock, shares outstanding | 26,475,000 | 26,313,000 |
Statements of Operations and Co
Statements of Operations and Comprehensive Loss - USD ($) shares in Thousands, $ in Thousands | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2016 | Sep. 30, 2015 | Sep. 30, 2016 | Sep. 30, 2015 | |
Revenues | ||||
License revenue | $ 65 | $ 65 | $ 2,638 | $ 635 |
License revenue from related party | 1,000 | 2,000 | ||
Reagent sales | 47 | 61 | 213 | 200 |
Grant revenue | 13 | 14 | 42 | 305 |
Total revenues | 125 | 1,140 | 2,893 | 3,140 |
Costs of revenues | ||||
Licensing costs (including amounts to related parties) | 13 | 213 | 528 | 527 |
Costs of reagent sales (including amounts to related parties) | 22 | 44 | 101 | 94 |
Research and development (including amounts to related parties) | 12,560 | 5,664 | 29,423 | 12,471 |
General and administrative (including amounts to related parties) | 6,200 | 2,567 | 17,848 | 7,671 |
Other operating expenses (income) | (2) | (1) | (136) | 15 |
Total operating expenses | 18,793 | 8,487 | 47,764 | 20,778 |
Loss from operations | (18,668) | (7,347) | (44,871) | (17,638) |
Other Income (Expense) | ||||
Investment income | 514 | 15 | 1,512 | 23 |
Interest expense | (20) | |||
Total other income (expense) | 514 | 15 | 1,512 | 3 |
Net loss | (18,154) | (7,332) | (43,359) | (17,635) |
Other Comprehensive Income (Loss) | ||||
Unrealized gain (loss) on available-for-sale securities | 332 | (26) | 1,572 | (26) |
Total other comprehensive income (loss) | 332 | (26) | 1,572 | (26) |
Comprehensive loss | (17,822) | (7,358) | (41,787) | (17,661) |
Reconciliation of net loss to net loss applicable to common stockholders | ||||
Net loss | (18,154) | (7,332) | (43,359) | (17,635) |
Net accretion and dividends on convertible preferred stock | (1,747) | |||
Net gain on extinguishment of convertible preferred stock | 759 | |||
Net loss applicable to common stockholders | $ (18,154) | $ (7,332) | $ (43,359) | $ (18,623) |
Basic and diluted net loss per common share | $ (0.69) | $ (1.52) | $ (1.64) | $ (5.48) |
Weighted-average basic and diluted common shares | 26,469 | 4,809 | 26,386 | 3,397 |
Statements of Cash Flows
Statements of Cash Flows - USD ($) $ in Thousands | 9 Months Ended | |
Sep. 30, 2016 | Sep. 30, 2015 | |
Cash flows from operating activities | ||
Net loss | $ (43,359) | $ (17,635) |
Adjustments to reconcile net loss to net cash used in operating activities | ||
Stock-based compensation expense | 5,031 | 2,059 |
Net amortization of premiums and accretion of discounts on marketable debt securities | 1,502 | 1 |
Depreciation and amortization | 264 | 43 |
Realized gains on sales of marketable securities | (20) | |
Unrealized foreign currency transaction gains | (2) | (4) |
Imputed interest on related party promissory notes | 13 | |
Changes in operating assets and liabilities | ||
Accounts receivable | 1,459 | 1,277 |
Prepaid expenses | (1,151) | (1,372) |
Other current assets | (1,149) | (127) |
Other assets | (71) | (128) |
Accounts payable | 3,595 | 317 |
Accrued expenses and other current liabilities | 4,591 | 758 |
Due to related party under services agreement | (34) | |
Other related party payables | (3,412) | |
Advance payments | (127) | (26) |
Deferred rent | 1,302 | 198 |
Net cash used in operating activities | (28,135) | (18,072) |
Cash flows from investing activities | ||
Restricted cash | (225) | |
Purchases of marketable securities | (32,262) | (19,065) |
Maturities of marketable securities | 38,131 | |
Sales of marketable securities | 23 | |
Purchases of property and equipment | (3,714) | (394) |
Net cash provided by (used in) investing activities | 1,953 | (19,459) |
Cash flows from financing activities | ||
Proceeds from exercise of stock options | 174 | 100 |
Proceeds from initial public offering of common stock, net of underwriting discounts and commissions | 148,233 | |
Issuance costs for initial public offering | (618) | |
Net cash provided by financing activities | 174 | 241,734 |
Net increase (decrease) in cash and cash equivalents | (26,008) | 204,203 |
Cash and cash equivalents | ||
Beginning of period | 54,116 | 1,121 |
End of period | 28,108 | 205,324 |
Supplemental cash flow information | ||
Cash paid for interest | 7 | |
Supplemental disclosures of non-cash investing and financing activities | ||
Purchases of property and equipment in accounts payable and accrued expenses | $ 1,816 | 34 |
Issuance costs for initial public offering in accounts payable and accrued expenses | 2,431 | |
Conversion of related party promissory notes into Series C convertible preferred stock | 1,389 | |
Conversion of convertible preferred stock into common stock upon initial public offering | 111,392 | |
Series C Convertible Preferred Stock [Member] | ||
Cash flows from financing activities | ||
Proceeds from issuance of convertible preferred stock, net of transaction costs | 26,021 | |
Supplemental disclosures of non-cash investing and financing activities | ||
Conversion of accrued service fees to related party | 2,403 | |
Series D Convertible Preferred Stock [Member] | ||
Cash flows from financing activities | ||
Proceeds from issuance of convertible preferred stock, net of transaction costs | $ 67,998 |
Nature of Business
Nature of Business | 9 Months Ended |
Sep. 30, 2016 | |
Organization Consolidation And Presentation Of Financial Statements [Abstract] | |
Nature of Business | 1. Nature of Business REGENXBIO Inc. (the Company) was formed on July 16, 2008 in the state of Delaware as ReGenX, LLC, and on December 22, 2009, changed its name to ReGenX Biosciences, LLC. On September 16, 2014, the Company converted from a limited liability company (LLC) to a C-corporation, and changed its name to REGENXBIO Inc. The Company is a leading biotechnology company focused on the development, commercialization and licensing of recombinant adeno-associated virus (AAV) gene therapy. The Company’s proprietary AAV gene delivery platform (NAV® Technology Platform) consists of exclusive rights to over 100 novel AAV vectors, including AAV7, AAV8, AAV9 and AAVrh10. The Company’s NAV® Technology Platform is being applied by the Company, as well as by third-party licensees, in the development of product candidates for a variety of diseases with unmet needs. Initial Public Offering On September 22, 2015, the Company completed its initial public offering (IPO) whereby the Company sold 7,245 shares of common stock (inclusive of 945 shares of common stock sold by the Company pursuant to the full exercise of an option to purchase additional shares granted to the underwriters in connection with the offering) at a price of $22.00 per share. The shares began trading on The Nasdaq Global Select Market on September 17, 2015. The aggregate net proceeds received by the Company from the offering were $145,184, net of underwriting discounts and commissions and offering expenses payable by the Company. Upon the closing of the IPO, all outstanding shares of convertible preferred stock converted into 16,298 shares of common stock. Liquidity and Risks As of September 30, 2016, the Company had generated an accumulated deficit of $94,979 since inception. As the Company continues to incur losses, transition to profitability is dependent upon the successful development, approval and commercialization of its product candidates and achieving a level of revenues adequate to support the Company’s cost structure. The Company may never achieve profitability, and unless and until it does, the Company will continue to need to raise additional capital. As of September 30, 2016, the Company had cash, cash equivalents and marketable securities of $184,857, which management believes is sufficient to fund operations for at least the next 12 months. The Company is subject to risks common to companies in the biotechnology industry, including, but not limited to, development by the Company or its competitors of technological innovations, risks of failure of clinical trials, dependence on key personnel, protection of proprietary technology, compliance with government regulations and ability to transition from preclinical manufacturing to commercial production of products. |
Summary of Significant Accounti
Summary of Significant Accounting Policies | 9 Months Ended |
Sep. 30, 2016 | |
Accounting Policies [Abstract] | |
Summary of Significant Accounting Policies | 2. Summary of Significant Accounting Policies Basis of Presentation and Unaudited Interim Financial Information The accompanying financial statements are unaudited and have been prepared in conformity with accounting principles generally accepted in the United States of America (GAAP). Any reference in these notes to applicable guidance is meant to refer to the authoritative GAAP as found in the Accounting Standards Codification (ASC) and Accounting Standards Update (ASU) of the Financial Accounting Standards Board (FASB). The interim unaudited financial statements have been prepared on the same basis as the annual audited financial statements as of and for the year ended December 31, 2015 included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2015, filed with the Securities and Exchange Commission (SEC) on March 3, 2016. Certain information and footnote disclosures required by GAAP which are normally included in the Company’s annual financial statements have been condensed or omitted pursuant to SEC rules and regulations for interim reporting. In the opinion of management, the accompanying financial statements reflect all adjustments, which include all normal and recurring adjustments necessary for the fair statement of the Company’s financial position as of September 30, 2016, and the results of its operations and its cash flows for the interim periods ended September 30, 2016 and 2015. The results of operations for the interim periods are not necessarily indicative of the results of operations to be expected for the full year, any other interim periods, or any future year or period. These interim financial statements should be read in conjunction with the audited financial statements as of and for the year ended December 31, 2015, and the notes thereto, which are included in the Company’s Annual Report on Form 10-K. Use of Estimates The preparation of financial statements in conformity with GAAP requires management to make estimates and assumptions that affect the reported amounts in the financial statements and accompanying notes. Actual results could materially differ from those estimates. Management considers many factors in selecting appropriate financial accounting policies and controls, and in developing the estimates and assumptions that are used in the preparation of these financial statements. Management must apply significant judgment in this process. In addition, other factors may affect estimates, including: expected business and operational changes, sensitivity and volatility associated with the assumptions used in developing estimates and whether historical trends are expected to be representative of future trends. The estimation process often may yield a range of potentially reasonable estimates of the ultimate future outcomes and management must select an amount that falls within that range of reasonable estimates. This process may result in actual results differing materially from those estimated amounts used in the preparation of the financial statements. Estimates are used in the following areas, among others: stock-based compensation expense, accrued research and development expenses and the fair value of financial instruments. Restricted Cash Restricted cash includes money market mutual funds used to collateralize an irrevocable letter of credit as required by the Company’s lease agreement for its office space in New York, New York. Fair Value of Financial Instruments The Company is required to disclose information on all assets and liabilities reported at fair value that enables an assessment of the inputs used in determining the reported fair values. FASB ASC Topic 820, Fair Value Measurements and Disclosures • Level 1—Valuations based on unadjusted quoted prices in active markets for identical assets or liabilities that the Company has the ability to access at the measurement date. • Level 2—Valuations based on quoted prices for similar assets or liabilities in markets that are not active or for which all significant inputs are observable, either directly or indirectly. • Level 3—Valuations that require inputs that reflect the Company’s own assumptions that are both significant to the fair value measurement and unobservable. To the extent that 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 fair values of the Company’s Level 2 instruments are based on quoted market prices or broker or dealer quotations for similar assets. These investments are initially valued at the transaction price and subsequently valued utilizing third party pricing providers or other market observable data. Please refer to Note 4 for further information on the fair value measurement of the Company’s financial instruments. Net Loss Per Share The Company computes net loss per share in conformity with the two-class method required for participating securities. The Company considers all series of convertible preferred stock outstanding prior to the IPO to be participating securities. The holders of convertible preferred stock outstanding prior to the IPO were entitled to receive preferential dividends in the event that a dividend was to be paid to the holders of common stock, and did not have a contractual obligation to share in the losses of the Company. As such, the Company’s net losses for the three and nine months ended September 30, 2015 were not allocated to these participating securities. In connection with the IPO, all outstanding shares of convertible preferred stock were automatically converted into shares of common stock. Basic net loss per share is calculated by dividing net loss applicable to holders of common stock by the weighted-average shares outstanding during the period, without consideration for common stock equivalents. Diluted net loss per share is calculated by adjusting weighted-average shares outstanding for the dilutive effect of common stock equivalents outstanding for the period, determined using the treasury-stock method. For purposes of the diluted net loss per share calculation, convertible preferred stock, outstanding stock options and withholdings under the employee stock purchase plan are considered to be common stock equivalents but have been excluded from the calculation of diluted net loss per share, as their effect would be anti-dilutive. Contingently convertible shares in which conversion is based on non-market-priced contingencies are excluded from the calculations of both basic and diluted net loss per share until the contingency has been fully met. Accordingly, basic and diluted net loss per share were the same for all periods presented. Recently Announced Accounting Pronouncements In May 2014, the FASB issued ASU No. 2014-09, Revenue from Contracts with Customers (Topic 606), Revenue Recognition. Revenue from Contracts with Customers (Topic 606), Revenue from Contracts with Customers (Topic 606): Identifying Performance Obligations and Licensing Revenue from Contracts with Customers (Topic 606): Narrow-Scope Improvements and Practical Expedients In June 2016, the FASB issued ASU 2016-13, Financial Instruments—Credit Losses (Topic 326): Measurement of Credit Losses on Financial Instruments In March 2016, the FASB issued ASU 2016-09, Compensation—Stock Compensation (Topic 718): Improvements to Employee Share-Based Payment Accounting In February 2016, the FASB issued ASU 2016-02, Leases (Topic 842) Leases (Topic 840) In January 2016, the FASB issued ASU 2016-01, Financial Instruments—Overall (Topic 825-10): Recognition and Measurement of Financial Assets and Financial Liabilities, In August 2014, the FASB issued ASU 2014-15, Presentation of Financial Statements—Going Concern (Subtopic 205-40): Disclosure of Uncertainties about an Entity’s Ability to Continue as a Going Concern, |
Marketable Securities
Marketable Securities | 9 Months Ended |
Sep. 30, 2016 | |
Investments Debt And Equity Securities [Abstract] | |
Marketable Securities | 3. Marketable Securities The following table presents a summary of the Company’s marketable securities, which consist solely of available-for-sale securities: Amortized Cost Unrealized Gains Unrealized Losses Fair Value September 30, 2016 Corporate bonds $ 155,596 $ 292 $ (34 ) $ 155,854 Common equity securities 300 595 — 895 $ 155,896 $ 887 $ (34 ) $ 156,749 Amortized Cost Unrealized Gains Unrealized Losses Fair Value December 31, 2015 Corporate bonds $ 157,977 $ 4 $ (759 ) $ 157,222 Commercial paper 4,990 — — 4,990 Common equity securities 3 36 — 39 $ 162,970 $ 40 $ (759 ) $ 162,251 As of December 31, 2015, the Company’s common equity securities consisted of shares of common stock of Dimension Therapeutics, Inc. (Dimension), which became a publicly traded company in October 2015. The Company obtained these shares in connection with a license granted to Dimension in October 2013. As of September 30, 2016, the Company had sold all of its shares of Dimension common stock. As of September 30, 2016, the Company’s common equity securities consisted of shares of common stock of Audentes Therapeutics, Inc. (Audentes), which became a publicly traded company in July 2016. The Company obtained these shares in connection with a license granted to Audentes in July 2013. The Company is restricted from trading these securities until January 2017 pursuant to a lock-up agreement entered into in connection with Audentes’ IPO. The Company has classified these shares as available-for-sale securities and recognized an unrealized gain of $595 which is included in accumulated other comprehensive income as of September 30, 2016. Prior to Audentes’ IPO, the shares were not marketable and were accounted for as a cost method investment on the Company’s balance sheets. As of September 30, 2016 and December 31, 2015, no available-for-sale securities had remaining maturities greater than three years. The amortized cost of available-for-sale securities is adjusted for amortization of premiums and accretion of discounts to maturity. As of September 30, 2016 and December 31, 2015, the balance in the Company’s accumulated other comprehensive income (loss) consisted solely of net unrealized gains and losses on available-for-sale securities. For the nine months ended September 30, 2016, the Company recognized net unrealized gains on available-for-sale securities of $1,572, which is included in other comprehensive income for the period. The Company recognized realized gains of $20 on the sale or maturity of available-for-sale securities during the nine months ended September 30, 2016, which were reclassified out of accumulated other comprehensive income (loss) during the period. The realized gains on available-for-sale securities related solely to the sale of Dimension common stock during the period. The aggregate fair value of securities held by the Company in an unrealized loss position for less than 12 months as of September 30, 2016 and December 31, 2015 was $40,512 and $155,486, respectively. The Company did not hold any securities in an unrealized loss position for more than 12 months as of December 31, 2015. The aggregate fair value of securities held by the Company in an unrealized loss position for more than 12 months as of September 30, 2016 was $4,116. The aggregate unrealized loss for those securities in an unrealized loss position for more than 12 months as of September 30, 2016 was $6. As of September 30, 2016, securities held by the Company which were in an unrealized loss position consisted of eleven investment grade corporate bonds. The Company has the intent and ability to hold such securities until recovery and has determined that none of its investments were other-than-temporarily impaired as of September 30, 2016 or December 31, 2015. |
Fair Value of Financial Instrum
Fair Value of Financial Instruments | 9 Months Ended |
Sep. 30, 2016 | |
Fair Value Disclosures [Abstract] | |
Fair Value of Financial Instruments | 4. Fair Value of Financial Instruments Financial instruments reported at fair value on a recurring basis include cash equivalents and marketable securities. Cash equivalents consist solely of money market mutual funds. Marketable securities consist of corporate debt securities, including corporate bonds and commercial paper, as well as common equity securities as disclosed in Note 3. The following tables present the fair value of cash equivalents and marketable securities in accordance with the hierarchy discussed in Note 2: Quoted Significant prices other Significant in active observable unobservable markets inputs inputs (Level 1) (Level 2) (Level 3) Total September 30, 2016 Money market mutual funds (cash equivalents) $ — $ 28,108 $ — $ 28,108 Corporate bonds (marketable securities) — 155,854 — 155,854 Common equity securities (marketable securities) 895 — — 895 $ 895 $ 183,962 $ — $ 184,857 Quoted Significant prices other Significant in active observable unobservable markets inputs inputs (Level 1) (Level 2) (Level 3) Total December 31, 2015 Money market mutual funds (cash equivalents) $ — $ 54,104 $ — $ 54,104 Corporate bonds (marketable securities) — 157,222 — 157,222 Commercial paper (marketable securities) — 4,990 — 4,990 Common equity securities (marketable securities) 39 — — 39 $ 39 $ 216,316 $ — $ 216,355 Management estimates that the carrying amounts of its accounts receivable, accounts payable and accrued expenses and other current liabilities approximate fair value due to the short-term nature of those instruments. The Company has determined that it is not practicable to estimate the fair value of cost method investments. The Company has not identified any events or changes in circumstances that would have an adverse effect on the fair value of its cost method investments reported as of December 31, 2015. |
Property and Equipment, Net
Property and Equipment, Net | 9 Months Ended |
Sep. 30, 2016 | |
Property Plant And Equipment [Abstract] | |
Property and Equipment, Net | 5. Property and Equipment, Net Property and equipment, net consists of the following: September 30, 2016 December 31, 2015 Computer equipment and software $ 865 $ 458 Lab equipment 756 — Furniture and fixtures 870 105 Leasehold improvements 3,657 55 Total property and equipment 6,148 618 Accumulated depreciation and amortization (344 ) (80 ) Property and equipment, net $ 5,804 $ 538 |
Commitments and Contingencies
Commitments and Contingencies | 9 Months Ended |
Sep. 30, 2016 | |
Commitments And Contingencies Disclosure [Abstract] | |
Commitments and Contingencies | 6. Commitments and Contingencies Lease Agreements The Company recognizes rent expense on a straight-line basis over the term of its operating leases commencing on the date the Company takes possession of the leased property. Tenant improvement allowances which are considered to be lease incentives from the lessor are recorded as deferred rent and amortized as a reduction of rent expense over the term of the lease from the possession date. In March 2015, the Company entered into a 5.5-year, non-cancelable operating lease for office space in Rockville, Maryland. The lease commenced in April 2015, and expires in September 2020. The Company has options to extend the lease for up to 6 years. Initial monthly payments required under the lease were $24 beginning in October 2015 and escalate annually in accordance with the lease. In September 2015, and again in November 2015, the Company amended its operating lease in Rockville, Maryland to include additional office and laboratory space and extend the term of the lease for its existing space to October 2020. The lease for the additional space commenced in April 2016, and has a 5-year term expiring in March 2021. The Company has options to extend the lease for the additional space to be coterminous with the Company’s existing lease at that facility. Initial monthly payments required under the lease for the additional space were $41 and escalate annually in accordance with the lease. The Company received a $286 tenant improvement allowance from the landlord which will be deferred and amortized on a straight-line basis as a reduction of rent expense over the term of lease. In January 2016, the Company entered into a 7.5-year, non-cancelable operating lease for additional office space in Rockville, Maryland. The lease commenced in February 2016, and expires in September 2023. Initial monthly payments required under the lease are $38 beginning seven months from the commencement date and escalate annually in accordance with the lease agreement. The Company received a $725 tenant improvement allowance from the landlord which will be deferred and amortized on a straight-line basis as a reduction of rent expense over the term of lease. In May 2016, the Company entered into a 51-month, non-cancelable operating lease for additional office space in New York, New York. The lease commenced in July 2016, and expires in October 2020. Initial monthly payments required under the lease are $25 beginning three months from the commencement date and escalate annually in accordance with the lease agreement. Under the terms of the lease agreement, the Company has provided the landlord with an irrevocable letter of credit of $225 which the landlord may draw upon in the event of any uncured default by the Company under the terms of the lease. As of September 30, 2016, the Company has recorded restricted cash of $225 as collateral to the financial institution which issued the letter of credit. The Company entered into a short-term operating lease for office space in Gaithersburg, Maryland which expired in October 2016. As of September 30, 2016, future minimum lease payments under non-cancelable operating leases are as follows: Operating Leases 2016 (remainder of year) $ 387 2017 1,589 2018 1,637 2019 1,685 2020 1,611 Thereafter 1,614 Total minimum lease payments $ 8,523 Licenses Granted to the Company Licenses granted to the Company may require the Company to make future payments relating to sublicense fees, milestone fees for milestones achieved in the future and royalties on future sales of licensed products. Additionally, the Company may be responsible for the cost of the maintenance of the intellectual property as incurred by its licensors. Up-front fees to obtain licensed technology are included in research and development expenses and patent maintenance costs are included in general and administrative expenses in the statements of operations and comprehensive loss. Sublicense fees are based on a specified percentage of license fees earned by the Company and are included in licensing costs in the statements of operations and comprehensive loss. Royalties on sales of licensed reagents for use in research and development are included in costs of reagent sales in the statements of operations and comprehensive loss. The Company has not commercialized any product candidates or paid any royalties under these agreements other than for the sales of licensed reagents. The Trustees of the University of Pennsylvania . In February 2009, the Company entered into a license agreement, as amended, with The Trustees of the University of Pennsylvania (Penn) for exclusive, worldwide rights to certain patents owned by Penn underlying the Company’s NAV® Technology Platform. Under the terms of the agreement, in consideration for the license, the Company issued to Penn 24.5 percent of the then outstanding membership interest in the LLC on a fully diluted basis after issuance. The Company is obligated to pay Penn royalties on net sales and sublicense fees, if any. Additionally, the Company is obligated to reimburse Penn for certain costs incurred related to the maintenance of the licensed patents. In April 2016, the Company entered into an agreement with Penn whereby the Company will fund clinical trial activities performed by Penn for RGX-501, the Company’s product candidate for homozygous familial hypercholesterolemia (HoFH). In connection with the agreement, the Company amended its license from Penn to include exclusive license rights to data, results and other information generated in connection with the RGX-501 clinical trial. Expenses incurred by the Company related to its license from Penn were as follows: Three Months Ended September 30, Nine Months Ended September 30, 2016 2015 2016 2015 Sublicense fees $ 7 $ 107 $ 264 $ 264 Royalties on sales of reagents 2 2 11 8 Maintenance of licensed patents 42 29 85 118 $ 51 $ 138 $ 360 $ 390 As of September 30, 2016 and December 31, 2015, the Company had accrued $22 and $440, respectively, in expenses payable to Penn under the license agreement, which are included in accounts payable and accrued expenses on the Company’s balance sheets. Until September 30, 2015, the Company considered Penn to be a related party. See Note 9 for further information on related party transactions with Penn. GlaxoSmithKline LLC. In March 2009, the Company entered into a license agreement, as amended, with GlaxoSmithKline LLC (GSK) for exclusive, worldwide rights to certain patents underlying the Company’s NAV® Technology Platform which are owned by Penn and exclusively licensed to GSK. Under the terms of the agreement, in consideration for the license, the Company issued to GSK 19.9 percent of the then outstanding membership interest in the LLC on a fully diluted basis after issuance. The Company is obligated to pay GSK royalties on net sales and sublicense fees, if any. Additionally, the Company is obligated to reimburse GSK for certain costs incurred and invoiced to the Company related to the maintenance of the licensed patents. The Company is obligated to pay GSK up to $1,500 upon the achievement of various milestones. As of September 30, 2016, no milestones have been achieved, or deemed probable of achievement, and accordingly no milestone payments were payable to GSK. Expenses incurred by the Company related to its license from GSK were as follows: Three Months Ended September 30, Nine Months Ended September 30, 2016 2015 2016 2015 Sublicense fees $ 7 $ 107 $ 264 $ 264 Royalties on sales of reagents 1 1 7 5 Maintenance of licensed patents 89 57 319 474 $ 97 $ 165 $ 590 $ 743 As of September 30, 2016 and December 31, 2015, the Company had accrued $74 and $526, respectively, in expenses payable to GSK under the license agreement, which are included in accounts payable and accrued expenses on the Company’s balance sheets. Until September 30, 2015, the Company considered GSK to be a related party. See Note 9 for further information on related party transactions with GSK. ARIAD Pharmaceuticals, Inc. In November 2010, the Company entered into a license agreement with ARIAD Pharmaceuticals, Inc. (ARIAD), for exclusive, worldwide rights to certain patents owned and exclusively licensed by ARIAD. In consideration for the license, the Company issued Class A Units of the LLC to ARIAD with a fair value of $726. Under the terms of the agreement, the Company is obligated to pay ARIAD royalties on net sales, and sublicense fees, if any. Additionally, the Company is obligated to pay ARIAD up to $2,300 and annual maintenance fees of $50 upon the achievement of various milestones. As of September 30, 2016, no milestones have been achieved, or deemed probable of achievement, and accordingly no milestone payments or maintenance fees were payable to ARIAD. Additionally, the Company has not incurred any royalties or sublicense fees payable to ARIAD since the inception of the agreement. There were no amounts due to ARIAD under the agreement as of September 30, 2016 and December 31, 2015. Regents of the University of Minnesota. In November 2014, the Company entered into a license agreement with Regents of the University of Minnesota (Minnesota), for an exclusive license under certain patent rights to commercialize products covered by the licensed patent rights in any country or territory in which a licensed patent has been issued and is unexpired, or a licensed patent application is pending. In consideration for the license, the Company paid an up-front fee of $25 and reimbursed Minnesota for patent maintenance expenses of $9. Under the terms of the agreement, the Company is obligated to pay Minnesota annual maintenance fees between $5 and $15 per year on each anniversary date of the agreement. Additionally, the Company is obligated to pay royalties on net sales and sublicense fees, if any, and up to $125 per licensed product upon the achievement of various milestones. As of September 30, 2016, no milestones have been achieved, or deemed probable of achievement, and accordingly no milestone payments were payable to Minnesota. Additionally, the Company has not incurred any royalties or sublicense fees payable to Minnesota since the inception of the agreement. As of September 30, 2016 and December 31, 2015, the Company had accrued $25 and $0, respectively, in patent maintenance expenses payable to Minnesota under the license agreement. Guarantees and Indemnifications In the normal course of business, the Company enters into agreements that contain a variety of representations and provide for general indemnification. The Company’s exposure under these agreements is unknown because it involves claims that may be made against the Company in the future. To date, the Company has not paid any claims or been required to defend any action related to its indemnification obligations. As of September 30, 2016 and December 31, 2015, the Company did not have any material indemnification claims that were probable or reasonably possible and consequently has not recorded any related liabilities. |
Significant Agreements
Significant Agreements | 9 Months Ended |
Sep. 30, 2016 | |
Organization Consolidation And Presentation Of Financial Statements [Abstract] | |
Significant Agreements | 7. Significant Agreements See Note 6 for license agreements granted to the Company. Licenses Granted by the Company The Company has granted a number of intellectual property licenses to other biotechnology and pharmaceutical companies. The terms of the licenses vary, however licenses may be exclusive or non-exclusive and may be sublicensable by the licensee. Licenses may grant intellectual property rights for purposes of internal and preclinical research and development only, or may include the rights, or options to obtain future rights, to commercialize drug therapies for specific diseases using the Company’s NAV® Technology. License agreements generally have a term equal to the life of the underlying patents and are terminable only at the option of the licensee. License agreements may require licensees to pay non-refundable up-front fees, option fees and annual maintenance fees. Additional contingent consideration under the licenses may include sublicense fees, milestone fees and royalties on net sales of commercialized products. Sublicense fees vary by license and range from a mid-single digit percentage to a low-double digit percentage of license fees received by licensees as a result of sublicenses. Royalties on net sales of commercialized products vary by license and range from a mid-single digit percentage to a low-teen percentage of net sales by licensees. Milestone fees are payable to the Company upon the achievement of specific clinical and regulatory developments by licensees. As of September 30, 2016, the Company’s license agreements, excluding additional licenses that could be granted upon the exercise of options by licensees, could result in aggregate milestone fees payable to the Company of up to $500 upon the submission of preclinical regulatory filings, $23,650 upon the commencement of various stages of clinical trials, $37,000 upon the submission of regulatory approval filings, $91,500 upon the approval of commercial products by regulatory agencies and $92,000 upon the achievement of specified sales targets for licensed products. License revenue consists of the following: Three Months Ended September 30, Nine Months Ended September 30, 2016 2015 2016 2015 Up-front fees and option fees for commercial licenses $ — $ 1,000 $ 2,000 $ 2,000 Maintenance fees for commercial licenses 65 65 395 245 Milestone fees — — — 250 Research and other license revenue — — 243 140 $ 65 $ 1,065 $ 2,638 $ 2,635 |
Stock-based Compensation
Stock-based Compensation | 9 Months Ended |
Sep. 30, 2016 | |
Disclosure Of Compensation Related Costs Sharebased Payments [Abstract] | |
Stock-based Compensation | 8. Stock-based Compensation Stock-based Compensation Expense The Company’s stock-based compensation expense by award type is as follows: Three Months Ended September 30, Nine Months Ended September 30, 2016 2015 2016 2015 Stock options $ 1,800 $ 1,349 $ 5,002 $ 2,059 Employee stock purchase plan 29 — 29 — $ 1,829 $ 1,349 $ 5,031 $ 2,059 The Company has recorded aggregate stock-based compensation expense in the statement of operations and comprehensive loss as follows: Three Months Ended September 30, Nine Months Ended September 30, 2016 2015 2016 2015 Research and development $ 814 $ 1,101 $ 1,824 $ 1,413 General and administrative 1,015 248 3,207 646 $ 1,829 $ 1,349 $ 5,031 $ 2,059 Stock Options In September 2014, the Board of Directors adopted the 2014 Stock Plan (2014 Plan). In June 2015, the Board of Directors adopted the 2015 Equity Incentive Plan (2015 Plan), which became effective on September 16, 2015, the date on which the registration statement for the IPO was declared effective. The 2015 Plan replaced the 2014 Plan, and as of the effective date of the 2015 Plan, no further awards may be issued under the 2014 Plan. Any options or awards outstanding under the 2014 Plan as of the effective date of the 2015 Plan remained outstanding and effective. The initial amount of shares authorized for issuance under the 2015 Plan was 2,952. The number of authorized shares under the 2015 Plan automatically increases annually on January 1, beginning January 1, 2016, by the lesser of (i) 4% of the total number of shares of common stock outstanding on December 31 of the prior year, or (ii) a number of common shares determined by the Board of Directors. Effective January 1, 2016, the Board of Directors authorized an additional 1,053 shares to be issued under the 2015 Plan. As of September 30, 2016, the total number of shares of common stock authorized for issuance under the 2015 Plan and 2014 Plan is 7,178, of which 1,848 remain available for future grants under the 2015 Plan. The 2014 Plan and 2015 Plan provide for the issuance of stock options, stock appreciation rights, restricted and unrestricted stock awards and performance cash awards to employees, members of the Board of Directors and consultants of the Company. No stock appreciation rights, restricted or unrestricted stock awards or performance cash awards have been granted under the 2014 Plan and 2015 Plan since the inception of the plans. Stock options under the 2014 Plan and 2015 Plan generally expire ten years following the date of grant. Options typically vest over a four year period, but vesting provisions can vary by award based on the discretion of the Board of Directors. Certain awards issued by the Company include performance conditions that must be achieved in order for vesting to occur. Stock options under the 2014 Plan and 2015 Plan carry an exercise price at least equal to the estimated fair value of the Company’s common stock on the date of grant. Shares of common stock underlying awards previously issued under the 2014 Plan and 2015 Plan which are reacquired by the Company, withheld by the Company in payment of the purchase price, exercise price or withholding taxes, expired, cancelled due to forfeiture or otherwise terminated other than by exercise, are added to the number of shares of common stock available for issuance under the 2015 Plan. Shares available for issuance under the 2015 Plan may be either authorized but unissued shares of the Company’s common stock or common stock reacquired by the Company and held in treasury. The 2015 Plan expires in June 2025, ten years from the date it was adopted by the Board of Directors, unless earlier terminated. The following table summarizes stock option activity under the 2014 Plan and 2015 Plan: Weighted- average Weighted- Remaining average Contractual Aggregate Exercise Life Intrinsic Shares Price (Years) Value (a) Outstanding at December 31, 2015 3,684 $ 5.52 9.1 $ 44,472 Granted 1,696 $ 12.30 Exercised (162 ) $ 1.08 Cancelled or forfeited (178 ) $ 14.62 Outstanding at September 30, 2016 5,040 $ 7.62 8.7 $ 58,033 Exercisable at September 30, 2016 1,898 $ 3.26 8.3 $ 23,110 Vested and expected to vest at September 30, 2016 5,040 $ 7.62 8.7 $ 58,033 (a) The aggregate intrinsic value is calculated as the difference between the exercise price of the underlying options and the fair value of the common stock for the options that were in the money at the dates reported. The weighted-average grant date fair value of options granted during the nine months ended September 30, 2016 was $8.18. During the nine months ended September 30, 2016, the total number of stock options exercised was 162, resulting in total proceeds of $174. The total intrinsic value of options exercised during the nine months ended September 30, 2016 was $1,692. Stock Options Granted to Employees . The fair value of options granted to employees was estimated at the date of grant using the Black-Scholes valuation model with the following weighted-average assumptions: Nine Months Ended September 30, 2016 2015 Expected volatility 75 % 67 % Expected term (in years) 6.2 6.1 Risk-free interest rate 1.5 % 1.7 % Expected dividend yield 0.0 % 0.0 % As of September 30, 2016, there was $19,003 of unrecognized stock-based compensation expense related to stock option awards to employees, which is expected to be recognized over a weighted-average period of 3.23 years. Stock Options Granted to Non-employees . Stock-based compensation expense related to stock options granted to non-employees is recognized as the stock options are earned. The Company believes that the estimated fair value of the stock options is more readily measurable than the fair value of the services rendered. The Company used the following weighted-average assumptions in estimating non-employees’ stock-based compensation expense: Nine Months Ended September 30, 2016 2015 Expected volatility 83 % 71 % Expected term (in years) 8.1 9.7 Risk-free interest rate 1.6 % 2.1 % Expected dividend yield 0.0 % 0.0 % Employee Stock Purchase Plan In June 2015, the Company’s Board of Directors adopted the 2015 Employee Stock Purchase Plan (2015 ESPP), which became effective on September 16, 2015, the date on which the registration statement for the IPO was declared effective. The 2015 ESPP authorizes the initial issuance of up to a total of 254 shares of the Company’s common stock to participating employees. The number of shares reserved for issuance under the 2015 ESPP automatically increases on the first business day of each fiscal year, commencing in 2016, by a number equal to the lesser of (i) 1% of the shares of common stock outstanding on the last business day of the prior fiscal year; or (ii) the number of shares determined by the Company’s Board of Directors. Unless otherwise determined by the administrator of the 2015 ESPP, two offering periods of six months’ duration will begin each year on January 1 and July 1. The Board of Directors did not authorize any further shares to be reserved for issuance under the 2015 ESPP in January 2016. As of September 30, 2016, no shares have been issued under the 2015 ESPP. |
Related Party Transactions
Related Party Transactions | 9 Months Ended |
Sep. 30, 2016 | |
Related Party Transactions [Abstract] | |
Related Party Transactions | 9. Related Party Transactions The Trustees of the University of Pennsylvania Penn received an equity interest in the Company as consideration for the intellectual property license granted to the Company in February 2009, and prior to September 30, 2015, the Company considered Penn to be a related party. See Note 6 for further information, including costs incurred by the Company, regarding its license from Penn. In addition to the license agreement, Penn also provides manufacturing and research and development services to the Company. Penn was considered a related party of the Company for the three and nine months ended September 30, 2015. During the three and nine months ended September 30, 2015, the Company incurred $40 and $80, respectively, in services from Penn for the manufacturing of reagents for sale by the Company as well as $2,493 and $5,136, respectively, in research and development services provided by Penn. As a result of various factors, including the dilution to the Company’s stockholders resulting from the sale and issuance of Series C convertible preferred stock (Series C Preferred Stock) and Series D convertible preferred stock (Series D Preferred Stock), and the closing of the Company’s IPO on September 22, 2015, the Company no longer considers Penn to be a related party subsequent to the period ended September 30, 2015. GlaxoSmithKline LLC GSK received an equity interest in the Company as consideration for the intellectual property license granted to the Company in March 2009, and prior to September 30, 2015, the Company considered GSK to be a related party. See Note 6 for further information, including costs incurred by the Company, regarding its license from GSK. As a result of various factors, including the dilution to the Company’s stockholders resulting from the sale and issuance of Series C Preferred Stock and Series D Preferred Stock, and the closing of the Company’s IPO on September 22, 2015, the Company no longer considers GSK to be a related party subsequent to the period ended September 30, 2015. Dimension Therapeutics, Inc. The Company and a number of its members, directors and management received common stock of Dimension as consideration for a license granted to Dimension in October 2013. Additionally, at the date of the license, three of the Company’s board members also served on the board of directors of Dimension. As a result, prior to September 30, 2015, the Company considered Dimension to be a related party. As of September 30, 2015, the Company no longer had any overlapping board members with Dimension. Additionally, the Company’s directors and management do not have significant influence over Dimension. Accordingly, the Company no longer considers Dimension to be a related party subsequent to the period ended September 30, 2015. During the three and nine months ended September 30, 2015, the Company recognized license revenue of $1,000 and $2,000, respectively, from commercial licenses granted to Dimension, which is included in license revenue from related party in the statements of operations and comprehensive loss. Revenue earned and recognized from Dimension subsequent to September 30, 2015 is no longer included in revenue from related party in the statements of operations and comprehensive loss. In 2014, the Company received $200 from Dimension for the purchase of materials owned by the Company and used in the Company’s manufacturing process for research and development and clinical trials. The $200 was recognized as a gain on disposal of the material as the material was delivered to Dimension. As of September 30, 2015, the Company had recognized $73 of the total gain on disposal and had recorded a liability of $127 related to undelivered material. During the three and nine months ended September 30, 2015, the Company recognized gains of $5 and $26, respectively, for the delivery of the material to Dimension. FoxKiser The Company was party to a services agreement, as amended from time to time, with FoxKiser LLP (FoxKiser), an affiliate of certain stockholders of the Company and an affiliate of one current and one former member of the Board of Directors, which was terminated in January 2015. Under the agreement, the Company paid a fixed monthly fee plus an additional support fee, as determined by FoxKiser on a monthly basis, as consideration for office facilities, equipment, supplies, general management, accounting, financial management, human resources, legal, secretarial, regulatory compliance and other services provided to the Company. The amounts outstanding to FoxKiser under the services agreement in excess of 30 days from their due date accrued interest at 1.5 percent per month, compounding monthly. The Company allocated the service fees from FoxKiser between research and development and general and administrative expense. For the three and nine months ended September 30, 2015, costs incurred under the services agreement with FoxKiser were $0 and $345, respectively. In January 2015, the Company entered into an operating lease with FoxKiser for office space in Washington, D.C. The lease agreement, which had a month-to-month term, required monthly payments of $20. The lease was terminated in April 2015. Rent expense to FoxKiser during the three and nine months ended September 30, 2015 was $0 and $60, respectively. The Company entered into a Professional Services Agreement with FoxKiser effective as of January 1, 2016, pursuant to which the Company incurs a fixed monthly fee of $80 in consideration for certain strategic planning, development and regulatory services to be provided by FoxKiser. The Professional Services Agreement has an initial term of one year and is terminable by either party, at any time, upon sixty days’ prior written notice to the other party. Costs incurred under the Professional Services Agreement with FoxKiser for the three and nine months ended September 30, 2016 were $240 and $720, which have been recorded as research and development expenses in the statements of operations and comprehensive loss. Chief Scientific Advisor In September 2014, the Company entered into an advisory agreement with its Chief Scientific Advisor, who is also the Chairman of the Company’s Scientific Advisory Board. The agreement was amended in May 2015 and expires in March 2017. During the three months ended September 30, 2016 and 2015, the Company incurred advisory fees of $62 and $45, respectively, to the Chief Scientific Advisor under the agreement. During the nine months ended September 30, 2016 and 2015, the Company incurred advisory fees of $187 and $135, respectively, to the Chief Scientific Advisor under the agreement. Additionally, since the inception of the agreement, the Company has granted options to purchase 212 shares of common stock to the Chief Scientific Advisor as compensation for services to be provided to the Company, which vest partially upon the completion of future service conditions and partially upon the achievement of specified performance conditions as set forth in the award agreements. |
Net Loss Per Share
Net Loss Per Share | 9 Months Ended |
Sep. 30, 2016 | |
Earnings Per Share [Abstract] | |
Net Loss Per Share | 10. Net Loss Per Share The following potentially dilutive securities outstanding at the end of the period were excluded from the computations of diluted weighted-average shares outstanding for the periods indicated as they would be anti-dilutive: Three and Nine Months Ended September 30, 2016 2015 Stock options issued and outstanding 5,040 3,464 ESPP shares 12 — 5,052 3,464 Amounts in the table above reflect the common stock equivalents of the noted instruments. |
Supplemental Disclosures
Supplemental Disclosures | 9 Months Ended |
Sep. 30, 2016 | |
Payables And Accruals [Abstract] | |
Supplemental Disclosures | 11. Supplemental Disclosures Accrued expenses and other current liabilities consists of the following: September 30, 2016 December 31, 2015 Accrued external research and development expenses $ 3,721 $ 835 Accrued personnel costs 3,339 1,371 Accrued purchases of property and equipment 1,055 — Accrued external general and administrative expenses 449 559 Accrued sublicense fees and royalties on reagent sales — 260 Other accrued expenses and current liabilities 442 173 $ 9,006 $ 3,198 |
Summary of Significant Accoun17
Summary of Significant Accounting Policies (Policies) | 9 Months Ended |
Sep. 30, 2016 | |
Accounting Policies [Abstract] | |
Basis of Presentation and Unaudited Interim Financial Information | Basis of Presentation and Unaudited Interim Financial Information The accompanying financial statements are unaudited and have been prepared in conformity with accounting principles generally accepted in the United States of America (GAAP). Any reference in these notes to applicable guidance is meant to refer to the authoritative GAAP as found in the Accounting Standards Codification (ASC) and Accounting Standards Update (ASU) of the Financial Accounting Standards Board (FASB). The interim unaudited financial statements have been prepared on the same basis as the annual audited financial statements as of and for the year ended December 31, 2015 included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2015, filed with the Securities and Exchange Commission (SEC) on March 3, 2016. Certain information and footnote disclosures required by GAAP which are normally included in the Company’s annual financial statements have been condensed or omitted pursuant to SEC rules and regulations for interim reporting. In the opinion of management, the accompanying financial statements reflect all adjustments, which include all normal and recurring adjustments necessary for the fair statement of the Company’s financial position as of September 30, 2016, and the results of its operations and its cash flows for the interim periods ended September 30, 2016 and 2015. The results of operations for the interim periods are not necessarily indicative of the results of operations to be expected for the full year, any other interim periods, or any future year or period. These interim financial statements should be read in conjunction with the audited financial statements as of and for the year ended December 31, 2015, and the notes thereto, which are included in the Company’s Annual Report on Form 10-K. |
Use of Estimates | Use of Estimates The preparation of financial statements in conformity with GAAP requires management to make estimates and assumptions that affect the reported amounts in the financial statements and accompanying notes. Actual results could materially differ from those estimates. Management considers many factors in selecting appropriate financial accounting policies and controls, and in developing the estimates and assumptions that are used in the preparation of these financial statements. Management must apply significant judgment in this process. In addition, other factors may affect estimates, including: expected business and operational changes, sensitivity and volatility associated with the assumptions used in developing estimates and whether historical trends are expected to be representative of future trends. The estimation process often may yield a range of potentially reasonable estimates of the ultimate future outcomes and management must select an amount that falls within that range of reasonable estimates. This process may result in actual results differing materially from those estimated amounts used in the preparation of the financial statements. Estimates are used in the following areas, among others: stock-based compensation expense, accrued research and development expenses and the fair value of financial instruments. |
Restricted Cash | Restricted Cash Restricted cash includes money market mutual funds used to collateralize an irrevocable letter of credit as required by the Company’s lease agreement for its office space in New York, New York. |
Fair Value of Financial Instruments | Fair Value of Financial Instruments The Company is required to disclose information on all assets and liabilities reported at fair value that enables an assessment of the inputs used in determining the reported fair values. FASB ASC Topic 820, Fair Value Measurements and Disclosures • Level 1—Valuations based on unadjusted quoted prices in active markets for identical assets or liabilities that the Company has the ability to access at the measurement date. • Level 2—Valuations based on quoted prices for similar assets or liabilities in markets that are not active or for which all significant inputs are observable, either directly or indirectly. • Level 3—Valuations that require inputs that reflect the Company’s own assumptions that are both significant to the fair value measurement and unobservable. To the extent that 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 fair values of the Company’s Level 2 instruments are based on quoted market prices or broker or dealer quotations for similar assets. These investments are initially valued at the transaction price and subsequently valued utilizing third party pricing providers or other market observable data. Please refer to Note 4 for further information on the fair value measurement of the Company’s financial instruments. |
Net Loss Per Share | Net Loss Per Share The Company computes net loss per share in conformity with the two-class method required for participating securities. The Company considers all series of convertible preferred stock outstanding prior to the IPO to be participating securities. The holders of convertible preferred stock outstanding prior to the IPO were entitled to receive preferential dividends in the event that a dividend was to be paid to the holders of common stock, and did not have a contractual obligation to share in the losses of the Company. As such, the Company’s net losses for the three and nine months ended September 30, 2015 were not allocated to these participating securities. In connection with the IPO, all outstanding shares of convertible preferred stock were automatically converted into shares of common stock. Basic net loss per share is calculated by dividing net loss applicable to holders of common stock by the weighted-average shares outstanding during the period, without consideration for common stock equivalents. Diluted net loss per share is calculated by adjusting weighted-average shares outstanding for the dilutive effect of common stock equivalents outstanding for the period, determined using the treasury-stock method. For purposes of the diluted net loss per share calculation, convertible preferred stock, outstanding stock options and withholdings under the employee stock purchase plan are considered to be common stock equivalents but have been excluded from the calculation of diluted net loss per share, as their effect would be anti-dilutive. Contingently convertible shares in which conversion is based on non-market-priced contingencies are excluded from the calculations of both basic and diluted net loss per share until the contingency has been fully met. Accordingly, basic and diluted net loss per share were the same for all periods presented. |
Recently Announced Accounting Pronouncements | Recently Announced Accounting Pronouncements In May 2014, the FASB issued ASU No. 2014-09, Revenue from Contracts with Customers (Topic 606), Revenue Recognition. Revenue from Contracts with Customers (Topic 606), Revenue from Contracts with Customers (Topic 606): Identifying Performance Obligations and Licensing Revenue from Contracts with Customers (Topic 606): Narrow-Scope Improvements and Practical Expedients In June 2016, the FASB issued ASU 2016-13, Financial Instruments—Credit Losses (Topic 326): Measurement of Credit Losses on Financial Instruments In March 2016, the FASB issued ASU 2016-09, Compensation—Stock Compensation (Topic 718): Improvements to Employee Share-Based Payment Accounting In February 2016, the FASB issued ASU 2016-02, Leases (Topic 842) Leases (Topic 840) In January 2016, the FASB issued ASU 2016-01, Financial Instruments—Overall (Topic 825-10): Recognition and Measurement of Financial Assets and Financial Liabilities, In August 2014, the FASB issued ASU 2014-15, Presentation of Financial Statements—Going Concern (Subtopic 205-40): Disclosure of Uncertainties about an Entity’s Ability to Continue as a Going Concern, |
Marketable Securities (Tables)
Marketable Securities (Tables) | 9 Months Ended |
Sep. 30, 2016 | |
Investments Debt And Equity Securities [Abstract] | |
Summary of Company Marketable Securities | The following table presents a summary of the Company’s marketable securities, which consist solely of available-for-sale securities: Amortized Cost Unrealized Gains Unrealized Losses Fair Value September 30, 2016 Corporate bonds $ 155,596 $ 292 $ (34 ) $ 155,854 Common equity securities 300 595 — 895 $ 155,896 $ 887 $ (34 ) $ 156,749 Amortized Cost Unrealized Gains Unrealized Losses Fair Value December 31, 2015 Corporate bonds $ 157,977 $ 4 $ (759 ) $ 157,222 Commercial paper 4,990 — — 4,990 Common equity securities 3 36 — 39 $ 162,970 $ 40 $ (759 ) $ 162,251 |
Fair Value of Financial Instr19
Fair Value of Financial Instruments (Tables) | 9 Months Ended |
Sep. 30, 2016 | |
Fair Value Disclosures [Abstract] | |
Schedule of Fair Value of Cash Equivalents and Marketable Securities | The following tables present the fair value of cash equivalents and marketable securities in accordance with the hierarchy discussed in Note 2: Quoted Significant prices other Significant in active observable unobservable markets inputs inputs (Level 1) (Level 2) (Level 3) Total September 30, 2016 Money market mutual funds (cash equivalents) $ — $ 28,108 $ — $ 28,108 Corporate bonds (marketable securities) — 155,854 — 155,854 Common equity securities (marketable securities) 895 — — 895 $ 895 $ 183,962 $ — $ 184,857 Quoted Significant prices other Significant in active observable unobservable markets inputs inputs (Level 1) (Level 2) (Level 3) Total December 31, 2015 Money market mutual funds (cash equivalents) $ — $ 54,104 $ — $ 54,104 Corporate bonds (marketable securities) — 157,222 — 157,222 Commercial paper (marketable securities) — 4,990 — 4,990 Common equity securities (marketable securities) 39 — — 39 $ 39 $ 216,316 $ — $ 216,355 |
Property and Equipment, Net (Ta
Property and Equipment, Net (Tables) | 9 Months Ended |
Sep. 30, 2016 | |
Property Plant And Equipment [Abstract] | |
Schedule of Property and Equipment | Property and equipment, net consists of the following: September 30, 2016 December 31, 2015 Computer equipment and software $ 865 $ 458 Lab equipment 756 — Furniture and fixtures 870 105 Leasehold improvements 3,657 55 Total property and equipment 6,148 618 Accumulated depreciation and amortization (344 ) (80 ) Property and equipment, net $ 5,804 $ 538 |
Commitments and Contingencies (
Commitments and Contingencies (Tables) | 9 Months Ended |
Sep. 30, 2016 | |
Schedule of Future Minimum Lease Payments Under Non-Cancelable Operating Leases | As of September 30, 2016, future minimum lease payments under non-cancelable operating leases are as follows: Operating Leases 2016 (remainder of year) $ 387 2017 1,589 2018 1,637 2019 1,685 2020 1,611 Thereafter 1,614 Total minimum lease payments $ 8,523 |
The Trustees of the University of Pennsylvania [Member] | |
Summary of Expenses Incurred by Company | Expenses incurred by the Company related to its license from Penn were as follows: Three Months Ended September 30, Nine Months Ended September 30, 2016 2015 2016 2015 Sublicense fees $ 7 $ 107 $ 264 $ 264 Royalties on sales of reagents 2 2 11 8 Maintenance of licensed patents 42 29 85 118 $ 51 $ 138 $ 360 $ 390 |
GlaxoSmithKline LLC [Member] | |
Summary of Expenses Incurred by Company | Expenses incurred by the Company related to its license from GSK were as follows: Three Months Ended September 30, Nine Months Ended September 30, 2016 2015 2016 2015 Sublicense fees $ 7 $ 107 $ 264 $ 264 Royalties on sales of reagents 1 1 7 5 Maintenance of licensed patents 89 57 319 474 $ 97 $ 165 $ 590 $ 743 |
Significant Agreements (Tables)
Significant Agreements (Tables) | 9 Months Ended |
Sep. 30, 2016 | |
Organization Consolidation And Presentation Of Financial Statements [Abstract] | |
Schedule of License Revenue | License revenue consists of the following: Three Months Ended September 30, Nine Months Ended September 30, 2016 2015 2016 2015 Up-front fees and option fees for commercial licenses $ — $ 1,000 $ 2,000 $ 2,000 Maintenance fees for commercial licenses 65 65 395 245 Milestone fees — — — 250 Research and other license revenue — — 243 140 $ 65 $ 1,065 $ 2,638 $ 2,635 |
Stock-based Compensation (Table
Stock-based Compensation (Tables) | 9 Months Ended |
Sep. 30, 2016 | |
Stock-Based Compensation Expense by Award Type | The Company’s stock-based compensation expense by award type is as follows: Three Months Ended September 30, Nine Months Ended September 30, 2016 2015 2016 2015 Stock options $ 1,800 $ 1,349 $ 5,002 $ 2,059 Employee stock purchase plan 29 — 29 — $ 1,829 $ 1,349 $ 5,031 $ 2,059 |
Stock-Based Compensation Expense | The Company has recorded aggregate stock-based compensation expense in the statement of operations and comprehensive loss as follows: Three Months Ended September 30, Nine Months Ended September 30, 2016 2015 2016 2015 Research and development $ 814 $ 1,101 $ 1,824 $ 1,413 General and administrative 1,015 248 3,207 646 $ 1,829 $ 1,349 $ 5,031 $ 2,059 |
Schedule of Fair Value of Options Granted to Employees Valuation Assumptions | The fair value of options granted to employees was estimated at the date of grant using the Black-Scholes valuation model with the following weighted-average assumptions: Nine Months Ended September 30, 2016 2015 Expected volatility 75 % 67 % Expected term (in years) 6.2 6.1 Risk-free interest rate 1.5 % 1.7 % Expected dividend yield 0.0 % 0.0 % |
Non Employee Stock Option [Member] | |
Schedule of Non-Employees Stock Purchase Plan Valuation Assumptions | The Company used the following weighted-average assumptions in estimating non-employees’ stock-based compensation expense: Nine Months Ended September 30, 2016 2015 Expected volatility 83 % 71 % Expected term (in years) 8.1 9.7 Risk-free interest rate 1.6 % 2.1 % Expected dividend yield 0.0 % 0.0 % |
2014 and 2015 Equity Incentive Plan [Member] | |
Summary of Stock Option Activity | The following table summarizes stock option activity under the 2014 Plan and 2015 Plan: Weighted- average Weighted- Remaining average Contractual Aggregate Exercise Life Intrinsic Shares Price (Years) Value (a) Outstanding at December 31, 2015 3,684 $ 5.52 9.1 $ 44,472 Granted 1,696 $ 12.30 Exercised (162 ) $ 1.08 Cancelled or forfeited (178 ) $ 14.62 Outstanding at September 30, 2016 5,040 $ 7.62 8.7 $ 58,033 Exercisable at September 30, 2016 1,898 $ 3.26 8.3 $ 23,110 Vested and expected to vest at September 30, 2016 5,040 $ 7.62 8.7 $ 58,033 (a) The aggregate intrinsic value is calculated as the difference between the exercise price of the underlying options and the fair value of the common stock for the options that were in the money at the dates reported. |
Net Loss Per Share (Tables)
Net Loss Per Share (Tables) | 9 Months Ended |
Sep. 30, 2016 | |
Earnings Per Share [Abstract] | |
Schedules for Computation of Diluted Weighted-Average Shares Outstanding | The following potentially dilutive securities outstanding at the end of the period were excluded from the computations of diluted weighted-average shares outstanding for the periods indicated as they would be anti-dilutive: Three and Nine Months Ended September 30, 2016 2015 Stock options issued and outstanding 5,040 3,464 ESPP shares 12 — 5,052 3,464 |
Supplemental Disclosures (Table
Supplemental Disclosures (Tables) | 9 Months Ended |
Sep. 30, 2016 | |
Payables And Accruals [Abstract] | |
Schedules of Accrued Expenses and Other Current Liabilities | Accrued expenses and other current liabilities consists of the following: September 30, 2016 December 31, 2015 Accrued external research and development expenses $ 3,721 $ 835 Accrued personnel costs 3,339 1,371 Accrued purchases of property and equipment 1,055 — Accrued external general and administrative expenses 449 559 Accrued sublicense fees and royalties on reagent sales — 260 Other accrued expenses and current liabilities 442 173 $ 9,006 $ 3,198 |
Nature of Business - Additional
Nature of Business - Additional Information (Detail) - USD ($) $ / shares in Units, shares in Thousands, $ in Thousands | Sep. 22, 2015 | Sep. 30, 2016 | Dec. 31, 2015 |
Class of Stock [Line Items] | |||
Accumulated deficit | $ (94,979) | $ (51,620) | |
Cash, cash equivalents and marketable securities | $ 184,857 | ||
Common Stock [Member] | |||
Class of Stock [Line Items] | |||
Conversion of convertible preferred stock into common stock upon initial public offering, Shares | 16,298 | ||
Initial Public Offering [Member] | |||
Class of Stock [Line Items] | |||
Issuance of convertible preferred stock, shares | 7,245 | ||
Stock issuance price per share | $ 22 | ||
Initial Public Offering [Member] | Common Stock [Member] | |||
Class of Stock [Line Items] | |||
Net proceeds from issuance of common stock | $ 145,184 | ||
Overallotment Option [Member] | |||
Class of Stock [Line Items] | |||
Issuance of convertible preferred stock, shares | 945 |
Marketable Securities - Summary
Marketable Securities - Summary of Company Marketable Securities (Detail) - USD ($) $ in Thousands | Sep. 30, 2016 | Dec. 31, 2015 |
Schedule Of Available For Sale Securities [Line Items] | ||
Amortized Cost | $ 155,896 | $ 162,970 |
Unrealized Gains | 887 | 40 |
Unrealized Losses | (34) | (759) |
Fair Value | 156,749 | 162,251 |
Common Equity Securities [Member] | ||
Schedule Of Available For Sale Securities [Line Items] | ||
Amortized Cost | 300 | 3 |
Unrealized Gains | 595 | 36 |
Fair Value | 895 | 39 |
Corporate Bonds [Member] | ||
Schedule Of Available For Sale Securities [Line Items] | ||
Amortized Cost | 155,596 | 157,977 |
Unrealized Gains | 292 | 4 |
Unrealized Losses | (34) | (759) |
Fair Value | $ 155,854 | 157,222 |
Commercial Paper [Member] | ||
Schedule Of Available For Sale Securities [Line Items] | ||
Amortized Cost | 4,990 | |
Fair Value | $ 4,990 |
Marketable Securities - Additio
Marketable Securities - Additional Information (Detail) | 3 Months Ended | 9 Months Ended | 12 Months Ended | ||
Sep. 30, 2016USD ($)Bond | Sep. 30, 2015USD ($) | Sep. 30, 2016USD ($)Bond | Sep. 30, 2015USD ($) | Dec. 31, 2015USD ($) | |
Schedule Of Available For Sale Securities [Line Items] | |||||
Unrealized gain (loss) on available-for-sale securities | $ 332,000 | $ (26,000) | $ 1,572,000 | $ (26,000) | |
Available for sale securities remaining maturities greater than three years | 0 | 0 | $ 0 | ||
Realized gains or losses available for sale securities | 20,000 | ||||
Aggregate fair value of securities in an unrealized loss position for less than twelve months | 40,512,000 | 40,512,000 | 155,486,000 | ||
Aggregate fair value of securities in an unrealized loss position for more than twelve months | $ 4,116,000 | 4,116,000 | |||
Aggregate unrealized loss position for more than twelve months | 6,000 | ||||
Other-than-temporary impaired | $ 0 | $ 0 | |||
Number of investment grade corporate bonds | Bond | 11 | 11 | |||
Common Equity Securities [Member] | |||||
Schedule Of Available For Sale Securities [Line Items] | |||||
Unrealized gain (loss) on available-for-sale securities | $ 595,000 | ||||
Common Equity Securities [Member] | Dimension Therapeutics, Inc. [Member] | |||||
Schedule Of Available For Sale Securities [Line Items] | |||||
Realized gains or losses available for sale securities | $ 20,000 |
Fair Value of Financial Instr29
Fair Value of Financial Instruments - Schedule of Fair Value of Cash Equivalents and Marketable Securities (Detail) - USD ($) $ in Thousands | Sep. 30, 2016 | Dec. 31, 2015 |
Fair Value Liabilities Measured On Recurring Basis Unobservable Input Reconciliation [Line Items] | ||
Available for sale securities | $ 156,749 | $ 162,251 |
Assets fair value disclosure | 184,857 | 216,355 |
Common Equity Securities [Member] | ||
Fair Value Liabilities Measured On Recurring Basis Unobservable Input Reconciliation [Line Items] | ||
Available for sale securities | 895 | 39 |
Corporate Bonds [Member] | ||
Fair Value Liabilities Measured On Recurring Basis Unobservable Input Reconciliation [Line Items] | ||
Available for sale securities | 155,854 | 157,222 |
Commercial Paper [Member] | ||
Fair Value Liabilities Measured On Recurring Basis Unobservable Input Reconciliation [Line Items] | ||
Available for sale securities | 4,990 | |
Money Market Mutual Funds [Member] | ||
Fair Value Liabilities Measured On Recurring Basis Unobservable Input Reconciliation [Line Items] | ||
Money market mutual funds (cash equivalents) | 28,108 | 54,104 |
Quoted Prices in Active Markets (Level 1) [Member] | ||
Fair Value Liabilities Measured On Recurring Basis Unobservable Input Reconciliation [Line Items] | ||
Assets fair value disclosure | 895 | 39 |
Quoted Prices in Active Markets (Level 1) [Member] | Common Equity Securities [Member] | ||
Fair Value Liabilities Measured On Recurring Basis Unobservable Input Reconciliation [Line Items] | ||
Available for sale securities | 895 | 39 |
Significant Other Observable Inputs (Level 2) [Member] | ||
Fair Value Liabilities Measured On Recurring Basis Unobservable Input Reconciliation [Line Items] | ||
Assets fair value disclosure | 183,962 | 216,316 |
Significant Other Observable Inputs (Level 2) [Member] | Corporate Bonds [Member] | ||
Fair Value Liabilities Measured On Recurring Basis Unobservable Input Reconciliation [Line Items] | ||
Available for sale securities | 155,854 | 157,222 |
Significant Other Observable Inputs (Level 2) [Member] | Commercial Paper [Member] | ||
Fair Value Liabilities Measured On Recurring Basis Unobservable Input Reconciliation [Line Items] | ||
Available for sale securities | 4,990 | |
Significant Other Observable Inputs (Level 2) [Member] | Money Market Mutual Funds [Member] | ||
Fair Value Liabilities Measured On Recurring Basis Unobservable Input Reconciliation [Line Items] | ||
Money market mutual funds (cash equivalents) | $ 28,108 | $ 54,104 |
Property and Equipment Net - Sc
Property and Equipment Net - Schedule of Property and Equipment (Detail) - USD ($) $ in Thousands | Sep. 30, 2016 | Dec. 31, 2015 |
Property Plant And Equipment [Line Items] | ||
Total property and equipment | $ 6,148 | $ 618 |
Accumulated depreciation and amortization | (344) | (80) |
Property and equipment, net | 5,804 | 538 |
Computer Equipment and Software [Member] | ||
Property Plant And Equipment [Line Items] | ||
Total property and equipment | 865 | 458 |
Lab Equipment [Member] | ||
Property Plant And Equipment [Line Items] | ||
Total property and equipment | 756 | |
Furniture and Fixtures [Member] | ||
Property Plant And Equipment [Line Items] | ||
Total property and equipment | 870 | 105 |
Leasehold Improvements [Member] | ||
Property Plant And Equipment [Line Items] | ||
Total property and equipment | $ 3,657 | $ 55 |
Commitments and Contingencies -
Commitments and Contingencies - Additional Information (Detail) | 1 Months Ended | 9 Months Ended | 12 Months Ended | |||||||
May 31, 2016USD ($) | Jan. 31, 2016USD ($) | Oct. 31, 2015USD ($) | Sep. 30, 2015USD ($) | Mar. 31, 2015 | Nov. 30, 2014USD ($) | Nov. 30, 2010USD ($) | Mar. 31, 2009USD ($) | Sep. 30, 2016USD ($)Milestone | Dec. 31, 2015USD ($) | |
Other Commitments [Line Items] | ||||||||||
Tenant improvement allowance received | $ 286,000 | |||||||||
Restricted cash as collateral with financial institution | $ 225,000 | |||||||||
Claims paid to date related to indemnification issues | 0 | |||||||||
Accruals or expenses related to indemnification issues | $ 0 | $ 0 | ||||||||
The Trustees of the University of Pennsylvania [Member] | ||||||||||
Other Commitments [Line Items] | ||||||||||
Percentage of ownership interest issued as consideration for license agreement | 24.50% | |||||||||
Accrued expenses | $ 22,000 | 440,000 | ||||||||
GlaxoSmithKline LLC [Member] | ||||||||||
Other Commitments [Line Items] | ||||||||||
Percentage of ownership interest issued as consideration for license agreement | 19.90% | |||||||||
Accrued expenses | $ 74,000 | 526,000 | ||||||||
Number of milestone | Milestone | 0 | |||||||||
Milestone payment obligation | $ 1,500,000 | $ 0 | ||||||||
ARIAD Pharmaceuticals, Inc [Member] | ||||||||||
Other Commitments [Line Items] | ||||||||||
Number of milestone | Milestone | 0 | |||||||||
Annual maintenance fees | $ 50,000 | $ 0 | ||||||||
Milestone payments | 0 | |||||||||
Royalty expense incurred | 0 | |||||||||
Sublicense fees | 0 | |||||||||
Amounts due under agreement | $ 0 | 0 | ||||||||
ARIAD Pharmaceuticals, Inc [Member] | Maximum [Member] | ||||||||||
Other Commitments [Line Items] | ||||||||||
Contractual obligation | 2,300,000 | |||||||||
ARIAD Pharmaceuticals, Inc [Member] | Common Class A [Member] | ||||||||||
Other Commitments [Line Items] | ||||||||||
Fair value of stock issued for license | $ 726,000 | |||||||||
Regents of the University of Minnesota [Member] | ||||||||||
Other Commitments [Line Items] | ||||||||||
Number of milestone | Milestone | 0 | |||||||||
Annual maintenance fees | $ 9,000 | |||||||||
Milestone payments | $ 0 | |||||||||
Amounts due under agreement | $ 25,000 | $ 0 | ||||||||
Up-front fee paid | 25,000 | |||||||||
Regents of the University of Minnesota [Member] | Maximum [Member] | Annual License Maintenance Fee [Member] | ||||||||||
Other Commitments [Line Items] | ||||||||||
Contractual obligation | 15,000 | |||||||||
Regents of the University of Minnesota [Member] | Maximum [Member] | Royalties And Sublicense Fees [Member] | ||||||||||
Other Commitments [Line Items] | ||||||||||
Contractual obligation | 125,000 | |||||||||
Regents of the University of Minnesota [Member] | Minimum [Member] | Annual License Maintenance Fee [Member] | ||||||||||
Other Commitments [Line Items] | ||||||||||
Contractual obligation | $ 5,000 | |||||||||
Rockville, Maryland [Member] | ||||||||||
Other Commitments [Line Items] | ||||||||||
Lease period | 7 years 6 months | 5 years | 5 years 6 months | |||||||
Lease expiration date | Sep. 30, 2023 | Mar. 31, 2021 | Sep. 30, 2020 | |||||||
Options to extend the lease | 6 years | |||||||||
Lease commenced date | 2016-02 | 2015-04 | ||||||||
Monthly lease payments | $ 38,000 | $ 24,000 | $ 41,000 | |||||||
Lease term | Extend the term of the lease for its existing space to October 2020. | |||||||||
Tenant improvement allowance received | $ 725,000 | |||||||||
New York, New York [Member] | ||||||||||
Other Commitments [Line Items] | ||||||||||
Lease expiration date | Oct. 31, 2020 | |||||||||
Lease commenced date | 2016-07 | |||||||||
Monthly lease payments | $ 25,000 | |||||||||
Lease expiration term | 51 months | |||||||||
Letters of credit | $ 225,000 |
Commitments and Contingencies32
Commitments and Contingencies - Schedule of Future Minimum Lease Payments Under Non-Cancelable Operating Leases (Detail) $ in Thousands | Sep. 30, 2016USD ($) |
Operating Leases Future Minimum Payments Due [Abstract] | |
2016 (remainder of year) | $ 387 |
2,017 | 1,589 |
2,018 | 1,637 |
2,019 | 1,685 |
2,020 | 1,611 |
Thereafter | 1,614 |
Total minimum lease payments | $ 8,523 |
Commitments and Contingencies33
Commitments and Contingencies - Summary of Expenses Incurred by Company (Detail) - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2016 | Sep. 30, 2015 | Sep. 30, 2016 | Sep. 30, 2015 | |
GlaxoSmithKline LLC [Member] | ||||
Other Commitments [Line Items] | ||||
Total related party transaction expense | $ 97 | $ 165 | $ 590 | $ 743 |
Sublicense Fees [Member] | GlaxoSmithKline LLC [Member] | ||||
Other Commitments [Line Items] | ||||
Total related party transaction expense | 7 | 107 | 264 | 264 |
Royalties on Sales of Reagents [Member] | GlaxoSmithKline LLC [Member] | ||||
Other Commitments [Line Items] | ||||
Total related party transaction expense | 1 | 1 | 7 | 5 |
Maintenance of Licensed Patents [Member] | GlaxoSmithKline LLC [Member] | ||||
Other Commitments [Line Items] | ||||
Total related party transaction expense | 89 | 57 | 319 | 474 |
License Fees [Member] | The Trustees of the University of Pennsylvania [Member] | ||||
Other Commitments [Line Items] | ||||
Total related party transaction expense | 51 | 138 | 360 | 390 |
License Fees [Member] | Sublicense Fees [Member] | The Trustees of the University of Pennsylvania [Member] | ||||
Other Commitments [Line Items] | ||||
Total related party transaction expense | 7 | 107 | 264 | 264 |
License Fees [Member] | Royalties on Sales of Reagents [Member] | The Trustees of the University of Pennsylvania [Member] | ||||
Other Commitments [Line Items] | ||||
Total related party transaction expense | 2 | 2 | 11 | 8 |
License Fees [Member] | Maintenance of Licensed Patents [Member] | The Trustees of the University of Pennsylvania [Member] | ||||
Other Commitments [Line Items] | ||||
Total related party transaction expense | $ 42 | $ 29 | $ 85 | $ 118 |
Significant Agreements - Additi
Significant Agreements - Additional Information (Detail) $ in Thousands | 9 Months Ended |
Sep. 30, 2016USD ($) | |
License Agreements [Abstract] | |
Milestone fee upon submission of preclinical regulatory filings | $ 500 |
Milestone fee upon commencement of clinical trials in humans | 23,650 |
Milestone fee upon submission of regulatory approval filings | 37,000 |
Milestone fee upon approval of commercial products by regulatory agencies | 91,500 |
Milestone fee upon achievement of specified sales targets for licensed products | $ 92,000 |
Significant Agreements - Schedu
Significant Agreements - Schedule of License Revenue (Detail) - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2016 | Sep. 30, 2015 | Sep. 30, 2016 | Sep. 30, 2015 | |
License Agreement Revenue Recognition [Abstract] | ||||
Up-front fees and option fees for commercial licenses | $ 1,000 | $ 2,000 | $ 2,000 | |
Maintenance fees for commercial licenses | $ 65 | 65 | 395 | 245 |
Milestone fees | 250 | |||
Research and other license revenue | 243 | 140 | ||
Licenses revenue including amounts from related parties | $ 65 | $ 1,065 | $ 2,638 | $ 2,635 |
Stock-based Compensation - Stoc
Stock-based Compensation - Stock-Based Compensation Expense by Award Type (Detail) - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2016 | Sep. 30, 2015 | Sep. 30, 2016 | Sep. 30, 2015 | |
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||
Stock-based compensation expense | $ 1,829 | $ 1,349 | $ 5,031 | $ 2,059 |
Stock Option [Member] | ||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||
Stock-based compensation expense | 1,800 | $ 1,349 | 5,002 | $ 2,059 |
Employee Stock Purchase Plan [Member] | ||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||
Stock-based compensation expense | $ 29 | $ 29 |
Stock-based Compensation - St37
Stock-based Compensation - Stock-Based Compensation Expense (Detail) - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2016 | Sep. 30, 2015 | Sep. 30, 2016 | Sep. 30, 2015 | |
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||
Stock-based compensation expense | $ 1,829 | $ 1,349 | $ 5,031 | $ 2,059 |
Research and Development Costs [Member] | ||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||
Stock-based compensation expense | 814 | 1,101 | 1,824 | 1,413 |
General and Administrative [Member] | ||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||
Stock-based compensation expense | $ 1,015 | $ 248 | $ 3,207 | $ 646 |
Stock-based Compensation - Addi
Stock-based Compensation - Additional Information (Detail) - USD ($) $ / shares in Units, $ in Thousands | Jan. 02, 2016 | Sep. 16, 2015 | Sep. 30, 2016 | Sep. 30, 2015 | Jan. 31, 2016 |
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |||||
Proceeds from exercise of stock options | $ 174 | $ 100 | |||
Unrecognized stock-based compensation expense related to employees' awards | $ 19,003 | ||||
Stock Option [Member] | |||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |||||
Unrecognized stock-based compensation, weighted-average period | 3 years 2 months 23 days | ||||
2015 Equity Incentive Plan [Member] | |||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |||||
Common stock shares authorized for issuance | 2,952,000 | 7,178,000 | |||
Percentage of annual increase in number of shares available for future issuance | 4.00% | ||||
Additional shares to be issued | 1,053,000 | ||||
Shares available for future grants | 1,848,000 | ||||
Stock options expiration date | 10 years | ||||
Stock options vesting period | 4 years | ||||
Stock plan expiration date | 2025-06 | ||||
Weighted-average fair values of options granted | $ 8.18 | ||||
Exercise of stock options, Shares | 162,000 | ||||
Total intrinsic value of options exercised | $ 1,692 | ||||
2015 Employee Stock Purchase Plan [Member] | |||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |||||
Share-based compensation arrangement by share-based payment award, percentage of outstanding stock minimum | 1.00% | ||||
Share-based compensation arrangement by share-based payment award, description | The number of shares reserved for issuance under the 2015 ESPP automatically increases on the first business day of each fiscal year, commencing in 2016 | ||||
Additional shares to be authorized | 0 | ||||
2015 Employee Stock Purchase Plan [Member] | Maximum [Member] | |||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |||||
Common stock shares authorized for issuance | 254,000 |
Stock-based Compensation - Summ
Stock-based Compensation - Summary of Stock Option Activity (Detail) - 2014 and 2015 Equity Incentive Plan [Member] - USD ($) $ / shares in Units, shares in Thousands, $ in Thousands | 9 Months Ended | 12 Months Ended |
Sep. 30, 2016 | Dec. 31, 2015 | |
Share-based Compensation Arrangement by Share-based Payment Award, Options, Nonvested, Number of Shares [Roll Forward] | ||
Shares Outstanding, Beginning Balance | 3,684 | |
Shares, Granted | 1,696 | |
Shares, Exercised | (162) | |
Shares, Cancelled or forfeited | (178) | |
Shares Outstanding, Ending Balance | 5,040 | 3,684 |
Shares, Exercisable | 1,898 | |
Shares, Vested and expected to vest | 5,040 | |
Share-based Compensation Arrangement by Share-based Payment Award, Options, Outstanding, Weighted Average Exercise Price [Abstract] | ||
Weighted-average Exercise Price Outstanding, Beginning Balance | $ 5.52 | |
Weighted-average Exercise Price, Granted | 12.30 | |
Weighted-average Exercise Price, Exercised | 1.08 | |
Weighted-average Exercise Price, Cancelled or forfeited | 14.62 | |
Weighted-average Exercise Price, Outstanding, Ending Balance | 7.62 | $ 5.52 |
Weighted-average Exercise Price, Exercisable | 3.26 | |
Weighted-average Exercise Price, Vested and expected to vest | $ 7.62 | |
Share-based Compensation Arrangement by Share-based Payment Award, Options, Additional Disclosures [Abstract] | ||
Weighted-average Remaining Contractual Life (Years) Outstanding | 8 years 8 months 12 days | 9 years 1 month 6 days |
Weighted-average Remaining Contractual Life (Years), Exercisable | 8 years 3 months 18 days | |
Weighted-average Remaining Contractual Life (Years), Vested and expected to vest | 8 years 8 months 12 days | |
Aggregate Intrinsic Value Outstanding | $ 58,033 | $ 44,472 |
Aggregate Intrinsic Value, Exercisable | 23,110 | |
Aggregate Intrinsic Value, Vested and expected to vest | $ 58,033 |
Stock-based Compensation - Sche
Stock-based Compensation - Schedule of Employees Stock Purchase Plan Valuation Assumptions (Detail) | 9 Months Ended | |
Sep. 30, 2016 | Sep. 30, 2015 | |
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||
Expected volatility | 75.00% | 67.00% |
Expected term (in years) | 6 years 2 months 12 days | 6 years 1 month 6 days |
Risk-free interest rate | 1.50% | 1.70% |
Expected dividend yield | 0.00% | 0.00% |
Non Employee Stock Option [Member] | ||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||
Expected volatility | 83.00% | 71.00% |
Expected term (in years) | 8 years 1 month 6 days | 9 years 8 months 12 days |
Risk-free interest rate | 1.60% | 2.10% |
Expected dividend yield | 0.00% | 0.00% |
Related Party Transactions - Ad
Related Party Transactions - Additional Information (Detail) - USD ($) shares in Thousands | Jan. 02, 2016 | Jan. 31, 2015 | Sep. 30, 2016 | Sep. 30, 2015 | Sep. 30, 2016 | Sep. 30, 2015 | Dec. 31, 2014 | Dec. 31, 2015 |
Related Party Transaction [Line Items] | ||||||||
License revenue from license agreements | $ 65,000 | $ 65,000 | $ 2,638,000 | $ 635,000 | ||||
Liability related to undelivered material | $ 127,000 | |||||||
Service Agreements [Member] | ||||||||
Related Party Transaction [Line Items] | ||||||||
Amount received for purchase of materials from related party | $ 80,000 | |||||||
The Trustees of the University of Pennsylvania [Member] | Manufacturing of Reagents for Sale [Member] | ||||||||
Related Party Transaction [Line Items] | ||||||||
Related party transaction expense | 40,000 | 80,000 | ||||||
The Trustees of the University of Pennsylvania [Member] | Research and Development Services [Member] | ||||||||
Related Party Transaction [Line Items] | ||||||||
Related party transaction expense | 2,493,000 | 5,136,000 | ||||||
Dimension Therapeutics, Inc. [Member] | ||||||||
Related Party Transaction [Line Items] | ||||||||
License revenue from license agreements | 1,000,000 | 2,000,000 | ||||||
Amount received for purchase of materials from related party | $ 200,000 | |||||||
Gain on disposal of the material to related party | 5,000 | 26,000 | $ 73,000 | |||||
Liability related to undelivered material | 127,000 | 127,000 | ||||||
FoxKiser,LLP [Member] | ||||||||
Related Party Transaction [Line Items] | ||||||||
Monthly lease payments | $ 20,000 | |||||||
Rent expense | 0 | 60,000 | ||||||
FoxKiser,LLP [Member] | Service Agreements [Member] | ||||||||
Related Party Transaction [Line Items] | ||||||||
Related party transaction expense | 0 | 345,000 | ||||||
Related party transaction, description | The amounts outstanding to FoxKiser under the services agreement in excess of 30 days from their due date accrued interest at 1.5 percent per month, compounding monthly. | |||||||
Accrued interest compounding monthly on amount outstanding | 1.50% | |||||||
FoxKiser,LLP [Member] | Service Agreements [Member] | Research and Development Costs [Member] | ||||||||
Related Party Transaction [Line Items] | ||||||||
Related party transaction expense | 240,000 | $ 720,000 | ||||||
Chief Scientific Advisor [Member] | ||||||||
Related Party Transaction [Line Items] | ||||||||
Related party transaction expenses from transactions with related party advisory fees | $ 62,000 | $ 45,000 | $ 187,000 | $ 135,000 | ||||
Shares, Granted | 212 |
Net Loss Per Share - Schedules
Net Loss Per Share - Schedules for Computation of Diluted Weighted-Average Shares Outstanding (Detail) - shares shares in Thousands | 9 Months Ended | |
Sep. 30, 2016 | Sep. 30, 2015 | |
Antidilutive Securities Excluded From Computation Of Earnings Per Share [Line Items] | ||
Anti-dilutive securities excluded from computation of diluted weighted average shares outstanding | 5,052 | 3,464 |
ESPP Shares [Member] | ||
Antidilutive Securities Excluded From Computation Of Earnings Per Share [Line Items] | ||
Anti-dilutive securities excluded from computation of diluted weighted average shares outstanding | 12 | |
Stock Options Issued and Outstanding [Member] | ||
Antidilutive Securities Excluded From Computation Of Earnings Per Share [Line Items] | ||
Anti-dilutive securities excluded from computation of diluted weighted average shares outstanding | 5,040 | 3,464 |
Supplemental Disclosures - Sche
Supplemental Disclosures - Schedules of Accrued Expenses and Other Current Liabilities (Detail) - USD ($) $ in Thousands | Sep. 30, 2016 | Dec. 31, 2015 |
Payables And Accruals [Abstract] | ||
Accrued external research and development expenses | $ 3,721 | $ 835 |
Accrued personnel costs | 3,339 | 1,371 |
Accrued purchases of property and equipment | 1,055 | |
Accrued external general and administrative expenses | 449 | 559 |
Accrued sublicense fees and royalties on reagent sales | 260 | |
Other accrued expenses and current liabilities | 442 | 173 |
Accrued expenses and other current liabilities | $ 9,006 | $ 3,198 |