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 | DRNA | |
Entity Registrant Name | DICERNA PHARMACEUTICALS INC | |
Entity Central Index Key | 1,399,529 | |
Current Fiscal Year End Date | --12-31 | |
Entity Filer Category | Non-accelerated Filer | |
Entity Common Stock, Shares Outstanding | 20,753,001 |
Condensed Consolidated Balance
Condensed Consolidated Balance Sheets - USD ($) $ in Thousands | Sep. 30, 2016 | Dec. 31, 2015 |
CURRENT ASSETS: | ||
Cash and cash equivalents | $ 32,544 | $ 56,058 |
Held-to-maturity investments | 25,002 | 38,551 |
Prepaid expenses and other current assets | 1,836 | 1,532 |
Total current assets | 59,382 | 96,141 |
NONCURRENT ASSETS: | ||
Property and equipment-net | 2,373 | 2,684 |
Assets held in restriction | 1,116 | 1,116 |
Other noncurrent assets | 78 | 82 |
Total noncurrent assets | 3,567 | 3,882 |
TOTAL ASSETS | 62,949 | 100,023 |
CURRENT LIABILITIES: | ||
Accounts payable | 3,657 | 2,621 |
Accrued expenses and other current liabilities | 6,160 | 6,380 |
Total current liabilities | 9,817 | 9,001 |
TOTAL LIABILITIES | 9,817 | 9,001 |
STOCKHOLDERS' EQUITY: | ||
Preferred stock, $0.0001 par value-5,000,000 shares authorized, no shares issued and outstanding at September 30, 2016 and December 31, 2015 | ||
Common stock, $0.0001 par value-150,000,000 shares authorized at September 30, 2016 and December 31, 2015; 20,753,001 and 20,594,575 shares issued and outstanding at September 30, 2016 and December 31, 2015, respectively | 2 | 2 |
Additional paid-in capital | 294,864 | 287,263 |
Accumulated deficit | (241,734) | (196,243) |
Total stockholders' equity | 53,132 | 91,022 |
TOTAL LIABILITIES AND STOCKHOLDERS' EQUITY | $ 62,949 | $ 100,023 |
Condensed Consolidated Balance3
Condensed Consolidated 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 | 5,000,000 | 5,000,000 |
Preferred stock, shares issued | 0 | 0 |
Preferred stock, shares outstanding | 0 | 0 |
Common stock, par value | $ 0.0001 | $ 0.0001 |
Common stock, shares authorized | 150,000,000 | 150,000,000 |
Common stock, shares issued | 20,753,001 | 20,594,575 |
Common stock, shares outstanding | 20,753,001 | 20,594,575 |
Condensed Consolidated Statemen
Condensed Consolidated Statements of Operations - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2016 | Sep. 30, 2015 | Sep. 30, 2016 | Sep. 30, 2015 | |
Income Statement [Abstract] | ||||
Revenues | $ 162 | $ 162 | $ 184 | |
Operating expenses: | ||||
Research and development | 10,061 | $ 12,142 | 32,357 | 32,708 |
General and administrative | 4,338 | 4,857 | 13,478 | 14,822 |
Total operating expenses | 14,399 | 16,999 | 45,835 | 47,530 |
Loss from operations | (14,237) | (16,999) | (45,673) | (47,346) |
Interest income | 61 | 55 | 182 | 142 |
Net loss | $ (14,176) | $ (16,944) | $ (45,491) | $ (47,204) |
Net loss per share-basic and diluted | $ (0.68) | $ (0.82) | $ (2.20) | $ (2.47) |
Weighted average shares outstanding-basic and diluted | 20,752,416 | 20,592,840 | 20,708,600 | 19,097,230 |
Condensed Consolidated Stateme5
Condensed Consolidated Statements of Cash Flows - USD ($) $ in Thousands | 9 Months Ended | |
Sep. 30, 2016 | Sep. 30, 2015 | |
CASH FLOWS FROM OPERATING ACTIVITIES: | ||
Net loss | $ (45,491) | $ (47,204) |
Adjustments to reconcile net loss to net cash used in operating activities: | ||
Depreciation and amortization | 627 | 529 |
Net amortization of premium/discount on investments | 65 | 100 |
Stock-based compensation | 7,067 | 7,374 |
Changes in operating assets and liabilities: | ||
Prepaid expenses and other assets | (300) | (459) |
Accounts payable | 983 | 133 |
Accrued expenses and other liabilities | (108) | 3,998 |
Net cash used in operating activities | (37,157) | (35,529) |
CASH FLOWS FROM INVESTING ACTIVITIES: | ||
Changes in assets held in restriction | 264 | |
Purchases of property and equipment | (375) | (995) |
Maturities of held-to-maturity investments | 33,500 | 53,500 |
Purchases of held-to-maturity investments | (20,016) | (36,130) |
Net cash provided by investing activities | 13,109 | 16,639 |
CASH FLOWS FROM FINANCING ACTIVITIES: | ||
Proceeds from stock option exercises and issuances under Employee Stock Purchase Plan | 561 | 403 |
Settlement of restricted stock for tax withholding | (27) | (75) |
Proceeds from public offering of common stock, net of costs | 45,438 | |
Net cash provided by financing activities | 534 | 45,766 |
(DECREASE) INCREASE IN CASH AND CASH EQUIVALENTS | (23,514) | 26,876 |
CASH AND CASH EQUIVALENTS - Beginning of period | 56,058 | 26,067 |
CASH AND CASH EQUIVALENTS - End of period | 32,544 | $ 52,943 |
SUPPLEMENTAL CASH FLOW INFORMATION: | ||
Property and equipment purchases included in accounts payable and accrued expenses | $ 52 |
Description of Business and Bas
Description of Business and Basis of Presentation | 9 Months Ended |
Sep. 30, 2016 | |
Accounting Policies [Abstract] | |
Description of Business and Basis of Presentation | 1. Description of Business and Basis of Presentation Nature of business Dicerna Pharmaceuticals, Inc. and its subsidiaries (the Company) is a biopharmaceutical company focused on the discovery and development of innovative subcutaneously delivered RNA interference (RNAi)-based pharmaceuticals using our GalXC ™ RNAi platform for the treatment of rare diseases involving the liver and for other therapeutic areas involving the liver such as chronic liver diseases, cardiovascular diseases, and viral infectious diseases. All of the Company’s GalXC drug discovery and development efforts are based on the therapeutic modality of RNAi, a highly potent and specific mechanism for silencing the activity of a targeted gene. In this naturally occurring biological process, double-stranded RNA molecules induce the enzymatic destruction of the mRNA of a target gene that contains sequences that are complementary to one strand of the therapeutic double-stranded RNA molecule. The Company’s approach is to design proprietary double-stranded RNA molecules that have the potential to engage the enzyme Dicer and initiate an RNAi process to silence a specific target gene. These proprietary molecules are generally referred to as Dicer Substrate short-interfering RNAs (DsiRNAs). Our GalXC RNAi platform utilizes a particular Dicer Substrate structure configured for subcutaneous delivery to the liver. Due to the enzymatic nature of RNAi, a single GalXC molecule incorporated into the RNAi machinery can destroy hundreds or thousands of mRNAs from the targeted gene. The Company is using its proprietary GalXC RNAi platform to build a broad pipeline in these therapeutic areas. The Company intends to discover, develop and commercialize novel therapeutics either on its own or in collaboration with pharmaceutical partners. The Company continues to be subject to a number of risks common to companies in similar stages of development. Principal among these risks are the uncertainties of technological innovations, which are particularly high in the field of drug discovery and development, dependence on key individuals, development of the same or similar technological innovations by the Company’s competitors and protection of proprietary technology. The Company’s ability to fund its planned preclinical and clinical operations, including completion of its clinical trials, will depend on its ability to raise capital through a combination of public or private equity offerings, debt financings, and research collaborations and license agreements. If we are unable to generate funding from one or more of these sources within a reasonable timeframe, we may have to delay, reduce or terminate our research and development programs, pre-clinical or clinical trials or undergo reductions in our workforce or other corporate restructuring activities. Based on our current operating plan, we believe that our available cash, cash equivalents and held-to-maturity investments will be sufficient to fund our planned level of operations for at least the next 12 months. We cannot predict whether additional funding will be available to us on acceptable terms or at all. In May 2015, the Company completed the sale of 2,750,000 shares of common stock in a public offering of its common stock at a price to the public of $17.75 per share, resulting in proceeds to the Company of $45.4 million after deducting underwriting discounts and commissions of approximately $2.9 million and offering costs incurred by the Company of approximately $0.4 million. In September 2016, the Company announced that it transitioned its development program for primary hyperoxaluria (PH) to its GalXC-based DCR-PHXC product candidate from DCR-PH1, a lipid nanoparticle (LNP) formulated RNAi compound. DCR-PH1 was being studied in two clinical trials; one in patients with PH type 1 (PH1) and another in normal healthy volunteers. As part of this transition, the clinical studies for DCR-PH1 are being discontinued. The Company also announced the discontinuation of development efforts on its proprietary product candidate DCR-MYC for the treatment of MYC-related cancers, which was being studied in two clinical trials; a Phase 1 trial in patients with advanced solid tumors and hematological malignancies, including an expansion cohort in patients with pancreatic neuroendocrine tumors; and a Phase 1b/2 trial in patients with advanced hepatocellular carcinoma. Both clinical studies for DCR-MYC are being discontinued. Since our inception in October 2006, we have devoted substantial resources to the research and development of DsiRNA and GalXC molecules, drug delivery technologies and the protection and enhancement of our intellectual property estate. We have no products approved for sale and all of our revenue to date has been collaboration revenue or government grant revenue. To date, we have funded our operations primarily through public offerings of our common stock, private placements of preferred stock and convertible debt securities, from research funding, license fees, option exercise fees, preclinical payments under our research collaboration and license agreement with the global pharmaceutical company Kyowa Hakko Kirin Co., Ltd. (KHK) and from government grants. More particularly, since our inception and through September 30, 2016, we have raised an aggregate of $266.9 million to fund our operations, of which approximately $45.4 million was from the May 2015 follow-on offering of common stock, $0.3 million was from a federal government grant from the National Institutes of Health (NIH) covering our work on cancer treatment research, $92.7 million was from the initial public offering of our common stock, which closed on February 4, 2014, $110.5 million was from the sale of preferred stock and convertible debt securities (including $3.0 million from the 2013 bridge note financing), $17.5 million was through our collaboration and license agreement with KHK and $0.5 million was from a federal government grant for our Qualifying Therapeutic Discovery Project in November 2010. As of September 30, 2016, we had cash and cash equivalents and held-to-maturity investments of $57.5 million and we also had $1.1 million in assets held in restriction. Basis of presentation and consolidation The accompanying unaudited condensed consolidated financial statements have been prepared in conformity with accounting principles generally accepted in the U.S. (GAAP) and in accordance with the rules and regulations of the Securities and Exchange Commission (SEC) for interim financial information. Accordingly, they do not include all of the information and notes required by GAAP for a complete set of financial statements. The unaudited condensed consolidated interim financial statements have been prepared on the same basis as the annual financial statements and, in the opinion of management, reflect all adjustments, which include only normal recurring adjustments, necessary to present fairly the Company’s financial position at September 30, 2016 and results of operations and cash flows for the interim periods ended September 30, 2016 and 2015. These unaudited condensed consolidated interim financial statements should be read in conjunction with the audited consolidated financial statements and notes thereto included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2015. The results of the three and nine months ended September 30, 2016 are not necessarily indicative of the results to be expected for the year ending December 31, 2016 or for any other interim period or for any other future year. Summary of Significant Accounting Policies Revenue Recognition Grant revenue is recognized in the period in which the related grant research and activities are incurred, provided that the conditions under which the grant was provided have been met and the Company only has perfunctory obligations outstanding. Any amounts received in advance of revenue recognition are classified as deferred revenue in the consolidated balance sheets. Costs associated with grants are included in research and development expenses in the consolidated statements of operations. Recent Accounting Pronouncements Stock Compensation In March 2016, the FASB issued ASU No. 2016-09, Compensation—Stock Compensation (Topic 718): Improvements to Employee Share-Based Payment Accounting. The new standard involves several aspects of the accounting for share-based payment transactions, including the income tax consequences, classification of awards as either equity or liabilities and classification on the statement of cash flows. The new standard will be effective for us on January 1, 2017. We are currently evaluating the potential impact that this standard may have on our financial position, results of operations and statement of cash flows. Leases In February 2016, the Financial Accounting Standards Board (FASB) issued Accounting Standards Update (ASU) No. 2016-02, Leases (Topic 842). The ASU requires lessees to put most leases on their balance sheets as a liability for the obligation to make lease payments and as a right-of-use asset, but recognize expenses on the income statements in a manner similar to today’s accounting. The guidance also eliminates the current real estate-specific provisions for all entities. For calendar-year public entities, the guidance becomes effective in 2019 and interim periods within that year. Early adoption is permitted for all entities. The Company has not chosen early adoption for this ASU and is currently evaluating its effect on the Company’s consolidated financial statements. Income Taxes In November 2015, the FASB issued ASU No. 2015-17, Income Taxes (Topic 740): Balance Sheet Classification of Deferred Taxes. ASU No. 2015-17 simplifies the presentation of deferred income taxes by eliminating the separate classification of deferred income tax assets and liabilities into current and noncurrent amounts in the consolidated balance sheet statement of financial position. The amendments in the update require that all deferred tax assets and liabilities be classified as noncurrent in the consolidated balance sheet. The amendments in this update are effective for annual periods beginning after December 15, 2017, and interim periods therein and may be applied either prospectively or retrospectively to all periods presented. Early adoption is permitted. The Company has early adopted this standard in the fourth quarter of 2015 on a prospective basis, and it did not have an effect on the Company’s consolidated financial statements. Going Concern In August 2014, the FASB issued ASU No. 2014-15, Disclosure of Uncertainties about an Entity’s Ability to Continue as a Going Concern Revenue Recognition In May 2014, the FASB issued ASU No. 2014-09, Revenue from Contracts with Customers (Topic 606). ASU No. 2014-09 amends the guidance for accounting for revenue from contracts with customers. This ASU supersedes the revenue recognition requirements in Accounting Standards Codification Topic 605, Revenue Recognition, and creates a new Topic 606, Revenue from Contracts with Customers. This guidance was originally pronounced to become effective for fiscal years beginning after December 15, 2016, with early adoption not permitted. On July 9, 2015, the FASB decided to defer the effective date of the ASU by one year. As a result, the Company will be required to apply the new revenue standard to annual reporting periods beginning after December 15, 2017, and would be permitted to adopt the ASU early, but not before the original public organization effective date (annual periods beginning after December 15, 2016). Two adoption methods are permitted: retrospectively to all prior reporting periods presented, with certain practical expedients permitted; or retrospectively with the cumulative effect of initially adopting the ASU recognized at the date of initial application. The Company has not yet determined which adoption method it will utilize or the effect, if any, the adoption of this guidance will have on its consolidated financial statements through December 31, 2015 as the Company has yet to record revenue from contracts with customers. |
Revenue Recognition
Revenue Recognition | 9 Months Ended |
Sep. 30, 2016 | |
Text Block [Abstract] | |
Revenue Recognition | 2. Revenue Recognition NIH Grants In April 2015, the National Cancer Institute (NCI), a division of the National Institutes of Health (NIH), awarded the Company a grant related to cancer treatment research. The project period for this grant covered a six month period which commenced in April 2015, with total funds available of approximately $0.2 million. The payment of the NIH grant award was based upon subcontractor and internal costs incurred that are specifically covered by the grant, and where applicable, a facilities and administrative rate that provides funding for overhead expenses. In August 2016, the NCI awarded an additional $2.0 million for a second phase of the grant covering the period September 1, 2016 to February 28, 2018. Of this amount, $1.0 million is committed funding and the additional funding commitment is expected in the third quarter of 2017, subject to NCI approval and availability of funds. The Company recognized zero and $0.2 million of revenue associated with the NIH grant awards for the three and nine month periods ended September 30, 2015, respectively, and $0.1 million of revenue for the three and nine month periods ended September 30, 2016. |
Held-to-maturity investments
Held-to-maturity investments | 9 Months Ended |
Sep. 30, 2016 | |
Text Block [Abstract] | |
Held-to-maturity investments | 3. Held-to-maturity investments The Company invests its excess cash balances in short-term fixed-income investments. The Company determines the appropriate classification of investments at the time of purchase and re-evaluates such designation as of each balance sheet date. Debt securities carried at amortized cost are classified as held-to-maturity when the Company has the positive intent and ability to hold the securities to maturity. The following tables provide information relating to held-to-maturity investments: At September 30, 2016: Amortized Cost Gross Unrealized Gains Gross Unrealized Losses Fair Value Held-to-maturity investments U.S. Government treasury and agency securities $ 25,002 $ 14 $ — $ 25,016 At December 31, 2015: Amortized Cost Gross Unrealized Gains Gross Unrealized Losses Fair Value Held-to-maturity investments U.S. Government treasury and agency securities $ 38,551 $ — $ (47 ) $ 38,504 The amortized cost and fair value of held-to-maturity investments by contractual maturities at September 30, 2016, are as follows (in thousands): Held-to-Maturity Amortized Cost Fair Value Maturing in one year or less $ 25,002 $ 25,016 Total $ 25,002 $ 25,016 |
Stock-Based Compensation
Stock-Based Compensation | 9 Months Ended |
Sep. 30, 2016 | |
Disclosure of Compensation Related Costs, Share-based Payments [Abstract] | |
Stock-Based Compensation | 4. Stock-Based Compensation Stock option grants to employees During the three and nine month periods ended September 30, 2016, the Company granted stock options to purchase 30,000 and 1,475,275 shares of common stock to employees with grant date fair values of $0.1 million and $6.9 million, respectively, compared to 195,700 and 1,045,626 shares of common stock with grant date fair values of $1.0 million and $10.3 million, for the comparable three and nine month periods in 2015. Employee stock-based compensation for the three and nine month periods ended September 30, 2016 were $2.3 million and $7.1 million as compared to $2.4 million and $7.2 million, respectively, for the comparable 2015 periods. The assumptions used to estimate the grant date fair value for 2016 and 2015 grants were as follows: Three Months Ended September 30, 2016 Nine Months Ended September 30, 2016 Stock price $3.21 $3.21 – $9.09 Expected option term (in years) 6.25 5.50 – 6.25 Expected volatility 76.1% 70.9% – 76.1% Risk-free interest rate 1.17% 1.17% – 1.71% Expected dividend yield 0.00% 0.00% Three Months Ended September 30, 2015 Nine Months Ended September 30, 2015 Stock price $8.21 $8.21 – $24.03 Expected option term (in years) 6.25 5.50 – 6.25 Expected volatility 71% 67% – 71% Risk-free interest rate 1.54% 1.51% – 1.81% Expected dividend yield 0.00% 0.00% |
Fair Value Measurements
Fair Value Measurements | 9 Months Ended |
Sep. 30, 2016 | |
Fair Value Disclosures [Abstract] | |
Fair Value Measurements | 5. Fair Value Measurements Fair value is an exit price, representing the amount that would be received from the sale of an asset or paid to transfer a liability in an orderly transaction between market participants. As such, fair value is a market-based measurement that should be determined based on assumptions that market participants would use in pricing an asset or liability. Valuation techniques used to measure fair value are performed in a manner to maximize the use of observable inputs and minimize the use of unobservable inputs. As a basis for considering such assumption the accounting literature establishes a three-tier value hierarchy which prioritizes the inputs used in measuring fair value as follows: (Level 1) observable inputs, such as quoted prices in active markets; (Level 2) inputs other than the quoted prices in active markets that are observable either directly or indirectly; and (Level 3) unobservable inputs for which there is little or no market data, which requires the Company to develop its own assumptions. A summary of the Company’s assets that are measured or disclosed at fair value as of September 30, 2016 and December 31, 2015 are presented below: Description At September 30, 2016 Level 1 Level 2 Level 3 Cash equivalents Money market fund $ 27,821 $ 27,821 $ — $ — Held-to-maturity investments U.S. treasury securities 25,015 — 25,015 — Assets held in restriction Money market fund 1,116 — 1,116 — Total $ 53,952 $ 27,821 $ 26,131 $ — Description At December 31, 2015 Level 1 Level 2 Level 3 Cash equivalents Money market fund $ 45,557 $ 45,557 $ — $ — Held-to-maturity investments U.S. treasury securities 38,504 — 38,504 — Assets held in restriction Money market fund 1,116 — 1,116 — Total $ 85,177 $ 45,557 $ 39,620 $ — The Company’s cash equivalents, which are in money market funds, are classified within Level 1 of the fair value hierarchy because they are valued using quoted prices as of September 30, 2016 and December 31, 2015, respectively. The Company’s assets held in restriction bore interest at the prevailing market rates for instruments with similar characteristics and, accordingly, the carrying value of these instruments also approximated their fair value and the financial instruments were classified within Level 2 of the fair value hierarchy because the inputs to the fair value measurement are valued using observable inputs as of September 30, 2016 and December 31, 2015, respectively. The Company’s held-to-maturity investments bore interest at the prevailing market rates for instruments with similar characteristics. The financial instruments were classified within Level 2 of the fair value hierarchy because the inputs to the fair value measurement are observable inputs as of September 30, 2016 and December 31, 2015, respectively. For the three and nine month periods ended September 30, 2016 and 2015 there were no transfers between Level 1 and Level 2. |
Commitments and Contingencies
Commitments and Contingencies | 9 Months Ended |
Sep. 30, 2016 | |
Commitments and Contingencies Disclosure [Abstract] | |
Commitments and Contingencies | 6. Commitments and Contingencies Facility lease On July 11, 2014, the Company executed a non-cancelable operating lease for office and laboratory space in Cambridge, Massachusetts. The lease agreement obligates the Company to minimum lease payments totaling $9.6 million over the six-year lease term. The lease commenced on December 1, 2014. As part of the lease agreement, the Company established a $1.1 million letter of credit, secured by a money market account which is included in assets held in restriction at September 30, 2016 and December 31, 2015. As of September 30, 2016, the remaining lease obligations were $6.9 million, of which $1.6 million is payable within the next year. City of Hope license agreement In September 2007, the Company entered into a license agreement with City of Hope, an independent academic research and medical center (the “Medical Center”). In consideration for the right to develop, manufacture, and commercialize products based on certain of the Medical Center’s intellectual property, the Company paid a one-time, non-refundable license fee and issued shares of common stock as consideration for the license. The Company is required to pay an annual license maintenance fee, reimburse the Medical Center for patent costs incurred, and pay an amount within the range of $5.0 million to $10.0 million upon the achievement of certain milestones, and royalties on future sales, if any. There were no sublicense and other fees accrued at September 30, 2016 and December 31, 2015. The license agreement will remain in effect until the expiration of the last patents or copyrights licensed under the agreement or until all obligations under the agreement with respect to payment of milestones have terminated or expired. The Company may terminate the license agreement at any time upon 90 days written notice to the Medical Center. The Company recorded research and development expense, related to the agreement with the Medical Center, of zero and $0.1 million for the three and nine month periods ended September 30, 2016 and 2015, respectively. Plant Bioscience Limited license agreement In September 2013, the Company entered into a commercial license agreement with Plant Bioscience Limited (PBL), pursuant to which PBL has granted to the Company a license to certain of its U.S. patents and patent applications to research, discover, develop, manufacture, sell, import and export, products incorporating one or more short RNA molecules (SRMs). The Company has paid PBL a one-time, non-refundable signature fee and will pay PBL a nomination fee for any additional SRMs nominated by the Company under the agreement. The Company is further obligated to pay PBL milestone payments upon achievement of certain clinical and regulatory milestones. During 2014, the Company paid $0.1 million to PBL based on meeting a clinical milestone. In addition, PBL is entitled to receive royalties of any net sale revenue of any licensed product candidates sold by the Company. The Company did not record any research and development expense, related to this agreement, during the three and nine month periods ended September 30, 2016 and 2015, respectively. Arbutus Biopharma Corporation license agreement In November 2014, the Company signed a licensing and collaboration agreement with Arbutus Biopharma Corporation (Arbutus) to license Arbutus’ LNP delivery technology for exclusive use in the Company’s primary hyperoxaluria type 1 (PH1) development program. The Company will use Arbutus’ LNP technology to deliver DCR-PH1, for the treatment of PH1. As of September 30, 2016, the Company paid $3.0 million in cumulative license fees. There were no license fees recorded in the three and nine month periods ended September 30, 2016 and 2015, respectively. Arbutus is entitled to receive additional payments of $22.0 million in aggregate development milestones, plus a mid-single-digit royalty on future PH1 sales. This partnership also includes a supply agreement with Arbutus providing clinical drug supply and regulatory support. Carnegie Institution of Washington license agreement In January 2009, the Company entered into a license agreement with the Carnegie Institution of Washington (Carnegie), pursuant to which Carnegie has granted to us a worldwide, non-exclusive license under certain of its patents and patent applications relating to genetic inhibition by double-stranded RNA molecules for internal research, screening and development of product candidates for human and non-human diagnostic and therapeutic uses. We have paid Carnegie a one-time upfront fee and will in addition pay an annual license fee during the term of the agreement. We are further obligated to make two additional payments of $0.1 million each upon achievement of the filing with the FDA of an NDA for a licensed product candidate and the first commercial sale of a licensed product candidate or licensed method. Carnegie is entitled to receive royalties on any net sale revenue from licensed product candidates sold by us, with the royalty rate to be further negotiated between Carnegie and us in good faith reflecting customary rates in the industry. The agreement will terminate with respect to each licensed product candidate upon the last to expire of any valid claim within the licensed patent rights. Each party may terminate the agreement upon any uncured material breach by the other party. We may terminate the agreement at any time for any reason upon written notice to Carnegie. Any patents associated with this license will expire in 2018, removing any obligations. The Company recorded research and development expense, related to the agreement with Carnegie, of zero and $0.03 million for the three and nine month periods ended September 30, 2016 and 2015, respectively. |
Litigation
Litigation | 9 Months Ended |
Sep. 30, 2016 | |
Commitments and Contingencies Disclosure [Abstract] | |
Litigation | 7. Litigation On June 10, 2015, Alnylam Pharmaceuticals, Inc. (Alnylam) filed a complaint against the Company in the Superior Court of Middlesex County, Massachusetts. The complaint alleges misappropriation of confidential, proprietary, and trade secret information, as well as other related claims, in connection with the Company’s hiring of a number of former employees of Merck & Co., Inc. (Merck) and its discussions with Merck regarding the acquisition of its subsidiary, Sirna Therapeutics, Inc. (Sirna), which was subsequently acquired by Alnylam. The complaint seeks among other things, unspecified damages, attorneys’ fees, and an order permanently enjoining the Company from disclosing or using any of Alnylam’s confidential information or trade secrets. The Company believes that these allegations lack merit, has filed an answer denying all liability and intends to continue to vigorously defend all claims asserted. At this time, the Company has not recorded a liability in connection with these matters because it believes that any potential loss is neither probable nor reasonably estimable. From time to time, the Company may be subject to various claims and legal proceedings. If the potential loss from any claim, asserted or unasserted, or legal proceeding is considered probable and the amount is reasonably estimable, the Company will accrue a liability for the estimated loss. There were no litigation liabilities outstanding as of September 30, 2016 and December 31, 2015. |
Description of Business and B13
Description of Business and Basis of Presentation (Policies) | 9 Months Ended |
Sep. 30, 2016 | |
Accounting Policies [Abstract] | |
Nature of business | Nature of business Dicerna Pharmaceuticals, Inc. and its subsidiaries (the Company) is a biopharmaceutical company focused on the discovery and development of innovative subcutaneously delivered RNA interference (RNAi)-based pharmaceuticals using our GalXC ™ RNAi platform for the treatment of rare diseases involving the liver and for other therapeutic areas involving the liver such as chronic liver diseases, cardiovascular diseases, and viral infectious diseases. All of the Company’s GalXC drug discovery and development efforts are based on the therapeutic modality of RNAi, a highly potent and specific mechanism for silencing the activity of a targeted gene. In this naturally occurring biological process, double-stranded RNA molecules induce the enzymatic destruction of the mRNA of a target gene that contains sequences that are complementary to one strand of the therapeutic double-stranded RNA molecule. The Company’s approach is to design proprietary double-stranded RNA molecules that have the potential to engage the enzyme Dicer and initiate an RNAi process to silence a specific target gene. These proprietary molecules are generally referred to as Dicer Substrate short-interfering RNAs (DsiRNAs). Our GalXC RNAi platform utilizes a particular Dicer Substrate structure configured for subcutaneous delivery to the liver. Due to the enzymatic nature of RNAi, a single GalXC molecule incorporated into the RNAi machinery can destroy hundreds or thousands of mRNAs from the targeted gene. The Company is using its proprietary GalXC RNAi platform to build a broad pipeline in these therapeutic areas. The Company intends to discover, develop and commercialize novel therapeutics either on its own or in collaboration with pharmaceutical partners. The Company continues to be subject to a number of risks common to companies in similar stages of development. Principal among these risks are the uncertainties of technological innovations, which are particularly high in the field of drug discovery and development, dependence on key individuals, development of the same or similar technological innovations by the Company’s competitors and protection of proprietary technology. The Company’s ability to fund its planned preclinical and clinical operations, including completion of its clinical trials, will depend on its ability to raise capital through a combination of public or private equity offerings, debt financings, and research collaborations and license agreements. If we are unable to generate funding from one or more of these sources within a reasonable timeframe, we may have to delay, reduce or terminate our research and development programs, pre-clinical or clinical trials or undergo reductions in our workforce or other corporate restructuring activities. Based on our current operating plan, we believe that our available cash, cash equivalents and held-to-maturity investments will be sufficient to fund our planned level of operations for at least the next 12 months. We cannot predict whether additional funding will be available to us on acceptable terms or at all. In May 2015, the Company completed the sale of 2,750,000 shares of common stock in a public offering of its common stock at a price to the public of $17.75 per share, resulting in proceeds to the Company of $45.4 million after deducting underwriting discounts and commissions of approximately $2.9 million and offering costs incurred by the Company of approximately $0.4 million. In September 2016, the Company announced that it transitioned its development program for primary hyperoxaluria (PH) to its GalXC-based DCR-PHXC product candidate from DCR-PH1, a lipid nanoparticle (LNP) formulated RNAi compound. DCR-PH1 was being studied in two clinical trials; one in patients with PH type 1 (PH1) and another in normal healthy volunteers. As part of this transition, the clinical studies for DCR-PH1 are being discontinued. The Company also announced the discontinuation of development efforts on its proprietary product candidate DCR-MYC for the treatment of MYC-related cancers, which was being studied in two clinical trials; a Phase 1 trial in patients with advanced solid tumors and hematological malignancies, including an expansion cohort in patients with pancreatic neuroendocrine tumors; and a Phase 1b/2 trial in patients with advanced hepatocellular carcinoma. Both clinical studies for DCR-MYC are being discontinued. Since our inception in October 2006, we have devoted substantial resources to the research and development of DsiRNA and GalXC molecules, drug delivery technologies and the protection and enhancement of our intellectual property estate. We have no products approved for sale and all of our revenue to date has been collaboration revenue or government grant revenue. To date, we have funded our operations primarily through public offerings of our common stock, private placements of preferred stock and convertible debt securities, from research funding, license fees, option exercise fees, preclinical payments under our research collaboration and license agreement with the global pharmaceutical company Kyowa Hakko Kirin Co., Ltd. (KHK) and from government grants. More particularly, since our inception and through September 30, 2016, we have raised an aggregate of $266.9 million to fund our operations, of which approximately $45.4 million was from the May 2015 follow-on offering of common stock, $0.3 million was from a federal government grant from the National Institutes of Health (NIH) covering our work on cancer treatment research, $92.7 million was from the initial public offering of our common stock, which closed on February 4, 2014, $110.5 million was from the sale of preferred stock and convertible debt securities (including $3.0 million from the 2013 bridge note financing), $17.5 million was through our collaboration and license agreement with KHK and $0.5 million was from a federal government grant for our Qualifying Therapeutic Discovery Project in November 2010. As of September 30, 2016, we had cash and cash equivalents and held-to-maturity investments of $57.5 million and we also had $1.1 million in assets held in restriction. |
Basis of presentation and consolidation | Basis of presentation and consolidation The accompanying unaudited condensed consolidated financial statements have been prepared in conformity with accounting principles generally accepted in the U.S. (GAAP) and in accordance with the rules and regulations of the Securities and Exchange Commission (SEC) for interim financial information. Accordingly, they do not include all of the information and notes required by GAAP for a complete set of financial statements. The unaudited condensed consolidated interim financial statements have been prepared on the same basis as the annual financial statements and, in the opinion of management, reflect all adjustments, which include only normal recurring adjustments, necessary to present fairly the Company’s financial position at September 30, 2016 and results of operations and cash flows for the interim periods ended September 30, 2016 and 2015. These unaudited condensed consolidated interim financial statements should be read in conjunction with the audited consolidated financial statements and notes thereto included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2015. The results of the three and nine months ended September 30, 2016 are not necessarily indicative of the results to be expected for the year ending December 31, 2016 or for any other interim period or for any other future year. |
Summary of Significant Accounting Policies | Summary of Significant Accounting Policies |
Revenue Recognition | Revenue Recognition Grant revenue is recognized in the period in which the related grant research and activities are incurred, provided that the conditions under which the grant was provided have been met and the Company only has perfunctory obligations outstanding. Any amounts received in advance of revenue recognition are classified as deferred revenue in the consolidated balance sheets. Costs associated with grants are included in research and development expenses in the consolidated statements of operations. |
Recent Accounting Pronouncements | Recent Accounting Pronouncements Stock Compensation In March 2016, the FASB issued ASU No. 2016-09, Compensation—Stock Compensation (Topic 718): Improvements to Employee Share-Based Payment Accounting. The new standard involves several aspects of the accounting for share-based payment transactions, including the income tax consequences, classification of awards as either equity or liabilities and classification on the statement of cash flows. The new standard will be effective for us on January 1, 2017. We are currently evaluating the potential impact that this standard may have on our financial position, results of operations and statement of cash flows. Leases In February 2016, the Financial Accounting Standards Board (FASB) issued Accounting Standards Update (ASU) No. 2016-02, Leases (Topic 842). The ASU requires lessees to put most leases on their balance sheets as a liability for the obligation to make lease payments and as a right-of-use asset, but recognize expenses on the income statements in a manner similar to today’s accounting. The guidance also eliminates the current real estate-specific provisions for all entities. For calendar-year public entities, the guidance becomes effective in 2019 and interim periods within that year. Early adoption is permitted for all entities. The Company has not chosen early adoption for this ASU and is currently evaluating its effect on the Company’s consolidated financial statements. Income Taxes In November 2015, the FASB issued ASU No. 2015-17, Income Taxes (Topic 740): Balance Sheet Classification of Deferred Taxes. ASU No. 2015-17 simplifies the presentation of deferred income taxes by eliminating the separate classification of deferred income tax assets and liabilities into current and noncurrent amounts in the consolidated balance sheet statement of financial position. The amendments in the update require that all deferred tax assets and liabilities be classified as noncurrent in the consolidated balance sheet. The amendments in this update are effective for annual periods beginning after December 15, 2017, and interim periods therein and may be applied either prospectively or retrospectively to all periods presented. Early adoption is permitted. The Company has early adopted this standard in the fourth quarter of 2015 on a prospective basis, and it did not have an effect on the Company’s consolidated financial statements. Going Concern In August 2014, the FASB issued ASU No. 2014-15, Disclosure of Uncertainties about an Entity’s Ability to Continue as a Going Concern Revenue Recognition In May 2014, the FASB issued ASU No. 2014-09, Revenue from Contracts with Customers (Topic 606). ASU No. 2014-09 amends the guidance for accounting for revenue from contracts with customers. This ASU supersedes the revenue recognition requirements in Accounting Standards Codification Topic 605, Revenue Recognition, and creates a new Topic 606, Revenue from Contracts with Customers. This guidance was originally pronounced to become effective for fiscal years beginning after December 15, 2016, with early adoption not permitted. On July 9, 2015, the FASB decided to defer the effective date of the ASU by one year. As a result, the Company will be required to apply the new revenue standard to annual reporting periods beginning after December 15, 2017, and would be permitted to adopt the ASU early, but not before the original public organization effective date (annual periods beginning after December 15, 2016). Two adoption methods are permitted: retrospectively to all prior reporting periods presented, with certain practical expedients permitted; or retrospectively with the cumulative effect of initially adopting the ASU recognized at the date of initial application. The Company has not yet determined which adoption method it will utilize or the effect, if any, the adoption of this guidance will have on its consolidated financial statements through December 31, 2015 as the Company has yet to record revenue from contracts with customers. |
Held-to-maturity investments (T
Held-to-maturity investments (Tables) | 9 Months Ended |
Sep. 30, 2016 | |
Text Block [Abstract] | |
Schedule of Held-To-Maturity Investments | The following tables provide information relating to held-to-maturity investments: At September 30, 2016: Amortized Cost Gross Unrealized Gains Gross Unrealized Losses Fair Value Held-to-maturity investments U.S. Government treasury and agency securities $ 25,002 $ 14 $ — $ 25,016 At December 31, 2015: Amortized Cost Gross Unrealized Gains Gross Unrealized Losses Fair Value Held-to-maturity investments U.S. Government treasury and agency securities $ 38,551 $ — $ (47 ) $ 38,504 |
Amortized Cost and Fair Value of Held-To-Maturity Investments | The amortized cost and fair value of held-to-maturity investments by contractual maturities at September 30, 2016, are as follows (in thousands): Held-to-Maturity Amortized Cost Fair Value Maturing in one year or less $ 25,002 $ 25,016 Total $ 25,002 $ 25,016 |
Stock-Based Compensation (Table
Stock-Based Compensation (Tables) | 9 Months Ended |
Sep. 30, 2016 | |
Employee Stock Option [Member] | |
Schedule of Valuation Assumptions | The assumptions used to estimate the grant date fair value for 2016 and 2015 grants were as follows: Three Months Ended September 30, 2016 Nine Months Ended September 30, 2016 Stock price $3.21 $3.21 – $9.09 Expected option term (in years) 6.25 5.50 – 6.25 Expected volatility 76.1% 70.9% – 76.1% Risk-free interest rate 1.17% 1.17% – 1.71% Expected dividend yield 0.00% 0.00% Three Months Ended September 30, 2015 Nine Months Ended September 30, 2015 Stock price $8.21 $8.21 – $24.03 Expected option term (in years) 6.25 5.50 – 6.25 Expected volatility 71% 67% – 71% Risk-free interest rate 1.54% 1.51% – 1.81% Expected dividend yield 0.00% 0.00% |
Fair Value Measurements (Tables
Fair Value Measurements (Tables) | 9 Months Ended |
Sep. 30, 2016 | |
Fair Value Disclosures [Abstract] | |
Schedule of Assets Measured or Disclosed at Fair Value | A summary of the Company’s assets that are measured or disclosed at fair value as of September 30, 2016 and December 31, 2015 are presented below: Description At September 30, 2016 Level 1 Level 2 Level 3 Cash equivalents Money market fund $ 27,821 $ 27,821 $ — $ — Held-to-maturity investments U.S. treasury securities 25,015 — 25,015 — Assets held in restriction Money market fund 1,116 — 1,116 — Total $ 53,952 $ 27,821 $ 26,131 $ — Description At December 31, 2015 Level 1 Level 2 Level 3 Cash equivalents Money market fund $ 45,557 $ 45,557 $ — $ — Held-to-maturity investments U.S. treasury securities 38,504 — 38,504 — Assets held in restriction Money market fund 1,116 — 1,116 — Total $ 85,177 $ 45,557 $ 39,620 $ — |
Description of Business and B17
Description of Business and Basis of Presentation - Additional Information (Detail) - USD ($) $ / shares in Units, $ in Thousands | Feb. 04, 2014 | May 31, 2015 | Nov. 30, 2010 | Sep. 30, 2015 | Sep. 30, 2016 | Sep. 30, 2015 | Dec. 31, 2013 | Dec. 31, 2015 |
Organization Consolidation And Presentation Of Financial Statements [Line Items] | ||||||||
Proceeds from issuance of common stock | $ 92,700 | $ 45,438 | ||||||
Aggregate amount raised to fund operations | $ 266,900 | |||||||
Proceeds from preferred stock and convertible debt securities | 110,500 | |||||||
Proceeds from bridge note financing | $ 3,000 | |||||||
Cash and cash equivalents and held-to-maturity investments | 57,500 | |||||||
Assets held in restriction | 1,116 | $ 1,116 | ||||||
National Institutes of Health [Member] | ||||||||
Organization Consolidation And Presentation Of Financial Statements [Line Items] | ||||||||
Funds available under revenue recognition grant | $ 200 | 300 | ||||||
Qualifying Therapeutic Discovery Project Program [Member] | ||||||||
Organization Consolidation And Presentation Of Financial Statements [Line Items] | ||||||||
Funds available under revenue recognition grant | $ 500 | |||||||
KHK Agreement [Member] | ||||||||
Organization Consolidation And Presentation Of Financial Statements [Line Items] | ||||||||
Proceeds from collaboration and license agreement | $ 17,500 | |||||||
IPO [Member] | ||||||||
Organization Consolidation And Presentation Of Financial Statements [Line Items] | ||||||||
Common stock shares issued | 2,750,000 | |||||||
Sale of stock price per share | $ 17.75 | |||||||
Proceeds from issuance of common stock | $ 45,400 | |||||||
Underwriting discounts and commissions | 2,900 | |||||||
Offering cost incurred | 400 | |||||||
Follow-on Offering [Member] | ||||||||
Organization Consolidation And Presentation Of Financial Statements [Line Items] | ||||||||
Proceeds from issuance of common stock | $ 45,400 |
Revenue Recognition - Additiona
Revenue Recognition - Additional Information (Detail) - National Institutes of Health [Member] - USD ($) $ in Millions | 1 Months Ended | 3 Months Ended | 6 Months Ended | 9 Months Ended | ||
Aug. 31, 2016 | Sep. 30, 2016 | Sep. 30, 2015 | Sep. 30, 2015 | Sep. 30, 2016 | Sep. 30, 2015 | |
Revenue Recognition, Multiple-deliverable Arrangements [Line Items] | ||||||
Funds available under revenue recognition grant | $ 0.2 | $ 0.3 | ||||
Revenue associated with NIH grant award | $ 0.1 | $ 0 | $ 0.1 | $ 0.2 | ||
Additional funds available for second phase of grant | $ 2 | |||||
Committed funding under the revenue recognition grant | $ 1 |
Held-to-Maturity Investments -
Held-to-Maturity Investments - Schedule of Held-to-Maturity Investments (Detail) - USD ($) $ in Thousands | Sep. 30, 2016 | Dec. 31, 2015 |
Schedule of Held-to-maturity Securities [Line Items] | ||
Held-to-maturity investments, Amortized Cost | $ 25,002 | |
Held-to-maturity investments, Fair Value, Total | 25,016 | |
U.S. Government Treasury and Agency Securities [Member] | ||
Schedule of Held-to-maturity Securities [Line Items] | ||
Held-to-maturity investments, Amortized Cost | 25,002 | $ 38,551 |
Held-to-maturity investments, Gross Unrealized Gains | 14 | |
Held-to-maturity investments, Gross Unrealized Losses | (47) | |
Held-to-maturity investments, Fair Value, Total | $ 25,016 | $ 38,504 |
Held-to-Maturity-Investments -
Held-to-Maturity-Investments - Amortized Cost and Fair Value of Held-to-Maturity Investments (Detail) - USD ($) $ in Thousands | Sep. 30, 2016 | Dec. 31, 2015 |
Investments, Debt and Equity Securities [Abstract] | ||
Held-to-Maturity securities, Maturing in one year or less, Amortized Cost | $ 25,002 | $ 38,551 |
Held to Maturity, Amortized Cost, Total | 25,002 | |
Held-to-Maturity securities, Maturing in one year or less, Fair Value | 25,016 | |
Held-to-Maturity, Fair Value, Total | $ 25,016 |
Stock-Based Compensation - Addi
Stock-Based Compensation - Additional Information (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 | $ 7,067 | $ 7,374 | ||
Employee Stock Option [Member] | ||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Stock option, granted | 30,000 | 195,700 | 1,475,275 | 1,045,626 |
Fair value of stock options | $ 100 | $ 1,000 | $ 6,900 | $ 10,300 |
Stock-based compensation expense | $ 2,300 | $ 2,400 | $ 7,100 | $ 7,200 |
Stock-Based Compensation - Sche
Stock-Based Compensation - Schedule of Valuation Assumptions (Detail) - Employee Stock Option [Member] - $ / shares | 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 price | $ 3.21 | $ 8.21 | ||
Expected option term (in years) | 6 years 3 months | 6 years 3 months | ||
Expected volatility | 76.10% | 71.00% | ||
Risk-free interest rate | 1.17% | 1.54% | ||
Expected dividend yield | 0.00% | 0.00% | 0.00% | 0.00% |
Minimum [Member] | ||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Stock price | $ 3.21 | $ 8.21 | ||
Expected option term (in years) | 5 years 6 months | 5 years 6 months | ||
Expected volatility | 70.90% | 67.00% | ||
Risk-free interest rate | 1.17% | 1.51% | ||
Maximum [Member] | ||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Stock price | $ 9.09 | $ 24.03 | ||
Expected option term (in years) | 6 years 3 months | 6 years 3 months | ||
Expected volatility | 76.10% | 71.00% | ||
Risk-free interest rate | 1.71% | 1.81% |
Fair Value Measurements - Sched
Fair Value Measurements - Schedule of Assets Measured or Disclosed at Fair Value (Detail) - USD ($) $ in Thousands | Sep. 30, 2016 | Dec. 31, 2015 |
Held-to-maturity investments | ||
U.S. treasury securities | $ 25,016 | |
Assets held in restriction | ||
Total | 53,952 | $ 85,177 |
Money Market Fund [Member] | ||
Cash equivalents | ||
Money market fund | 27,821 | 45,557 |
Assets held in restriction | ||
Restricted investments, at fair value | 1,116 | 1,116 |
US Treasury Securities [Member] | ||
Held-to-maturity investments | ||
U.S. treasury securities | 25,015 | 38,504 |
Level 1 [Member] | ||
Assets held in restriction | ||
Total | 27,821 | 45,557 |
Level 1 [Member] | Money Market Fund [Member] | ||
Cash equivalents | ||
Money market fund | 27,821 | 45,557 |
Level 2 [Member] | ||
Assets held in restriction | ||
Total | 26,131 | 39,620 |
Level 2 [Member] | Money Market Fund [Member] | ||
Assets held in restriction | ||
Restricted investments, at fair value | 1,116 | 1,116 |
Level 2 [Member] | US Treasury Securities [Member] | ||
Held-to-maturity investments | ||
U.S. treasury securities | $ 25,015 | $ 38,504 |
Fair Value Measurements - Addit
Fair Value Measurements - Additional Information (Detail) - USD ($) | Sep. 30, 2016 | Sep. 30, 2015 |
Debt Instrument Fair Value Carrying Value [Abstract] | ||
Transfers between Level 1 and Level 2 | $ 0 | $ 0 |
Commitments and Contingencies -
Commitments and Contingencies - Additional Information (Detail) | Jul. 11, 2014USD ($) | Sep. 30, 2016USD ($) | Sep. 30, 2015USD ($) | Sep. 30, 2016USD ($)Payments | Sep. 30, 2015USD ($) | Dec. 31, 2015USD ($) | Dec. 31, 2014USD ($) | Dec. 01, 2014USD ($) |
Loss Contingencies [Line Items] | ||||||||
Research and development expense | $ 10,061,000 | $ 12,142,000 | $ 32,357,000 | $ 32,708,000 | ||||
Cambridge Massachusetts , 2014 Operating Lease Agreement [Member] | ||||||||
Loss Contingencies [Line Items] | ||||||||
Operating minimum lease payments | $ 9,600,000 | |||||||
Operating minimum lease payments term | 6 years | |||||||
Letter of credit | $ 1,100,000 | |||||||
Deferred rent obligation | Dec. 1, 2014 | |||||||
Remaining lease obligation | 6,900,000 | 6,900,000 | ||||||
Operating lease obligation, payable within the next year | 1,600,000 | $ 1,600,000 | ||||||
City of Hope License Agreement [Member] | ||||||||
Loss Contingencies [Line Items] | ||||||||
License agreement date | Sep. 30, 2007 | |||||||
Expenses related to license agreement | $ 0 | $ 0 | ||||||
Termination of license agreement description | The Company may terminate the license agreement at any time upon 90 days written notice | |||||||
Research and development expense | 0 | 0 | $ 100,000 | 100,000 | ||||
City of Hope License Agreement [Member] | Minimum [Member] | ||||||||
Loss Contingencies [Line Items] | ||||||||
Expenses related to license agreement | 5,000,000 | |||||||
City of Hope License Agreement [Member] | Maximum [Member] | ||||||||
Loss Contingencies [Line Items] | ||||||||
Expenses related to license agreement | $ 10,000,000 | |||||||
Plant Bioscience Limited License Agreement [Member] | ||||||||
Loss Contingencies [Line Items] | ||||||||
License agreement date | Sep. 30, 2013 | |||||||
Research and development expense | 0 | 0 | $ 0 | 0 | ||||
Payments on commencement of clinical trial | $ 100,000 | |||||||
Arbutus Biopharma Corporation License Agreement [Member] | ||||||||
Loss Contingencies [Line Items] | ||||||||
Expenses related to license agreement | 3,000,000 | |||||||
Aggregate development milestones and royalty | 22,000,000 | |||||||
Additional license cost incurred | 0 | 0 | 0 | 0 | ||||
Carnegie Institution of Washington License Agreement [Member] | ||||||||
Loss Contingencies [Line Items] | ||||||||
Research and development expense | $ 0 | $ 30,000 | 0 | $ 30,000 | ||||
Additional milestone payments | $ 100,000 | |||||||
Number of additional milestone payments | Payments | 2 |
Litigation - Additional Informa
Litigation - Additional Information (Detail) - USD ($) | Sep. 30, 2016 | Dec. 31, 2015 |
Commitments and Contingencies Disclosure [Abstract] | ||
Outstanding litigation liabilities | $ 0 | $ 0 |