Document and Entity Information
Document and Entity Information - shares | 6 Months Ended | |
Jun. 30, 2017 | Jul. 28, 2017 | |
Document And Entity Information [Abstract] | ||
Document Type | 10-Q | |
Amendment Flag | false | |
Document Period End Date | Jun. 30, 2017 | |
Document Fiscal Year Focus | 2,017 | |
Document Fiscal Period Focus | Q2 | |
Trading Symbol | RGLS | |
Entity Registrant Name | REGULUS THERAPEUTICS INC. | |
Entity Central Index Key | 1,505,512 | |
Current Fiscal Year End Date | --12-31 | |
Entity Filer Category | Accelerated Filer | |
Entity Common Stock, Shares Outstanding | 103,785,730 |
Condensed Balance Sheets
Condensed Balance Sheets - USD ($) $ in Thousands | Jun. 30, 2017 | Dec. 31, 2016 |
Current assets: | ||
Cash and cash equivalents | $ 12,538 | $ 14,941 |
Short-term investments | 27,549 | 61,170 |
Contract and other receivables | 690 | 1,657 |
Prepaid materials, net | 6,149 | 5,552 |
Prepaid expenses and other current assets | 3,018 | 4,154 |
Total current assets | 49,944 | 87,474 |
Property and equipment, net | 10,753 | 11,830 |
Intangibles, net | 823 | 1,015 |
Other assets | 340 | 342 |
Total assets | 61,860 | 100,661 |
Current liabilities: | ||
Accounts payable | 4,901 | 5,840 |
Accrued liabilities | 4,717 | 5,577 |
Accrued compensation | 1,433 | 2,318 |
Current portion of deferred revenue | 72 | 72 |
Total current liabilities | 11,123 | 13,807 |
Term loan, less debt issuance costs | 19,830 | 19,802 |
Deferred revenue, less current portion | 1,957 | 1,993 |
Deferred rent, less current portion | 8,672 | 8,840 |
Other long-term liabilities | 283 | 144 |
Total liabilities | 41,865 | 44,586 |
Commitments and Contingencies | ||
Stockholders’ equity: | ||
Common stock, $0.001 par value; 200,000,000 shares authorized, 53,182,330 and 52,924,805 shares issued and outstanding at June 30, 2017 (unaudited) and December 31, 2016, respectively | 53 | 53 |
Additional paid-in capital | 335,612 | 329,496 |
Accumulated other comprehensive loss | (88) | (123) |
Accumulated deficit | (315,582) | (273,351) |
Total stockholders’ equity | 19,995 | 56,075 |
Total liabilities and stockholders’ equity | $ 61,860 | $ 100,661 |
Condensed Balance Sheets (Paren
Condensed Balance Sheets (Parenthetical) - $ / shares | Jun. 30, 2017 | Dec. 31, 2016 |
Statement of Financial Position [Abstract] | ||
Common stock, par value (in dollars per share) | $ 0.001 | $ 0.001 |
Common stock, shares authorized | 200,000,000 | 200,000,000 |
Common stock, shares issued | 53,182,330 | 52,924,805 |
Common stock, shares outstanding | 53,182,330 | 52,924,805 |
Condensed Statements of Operati
Condensed Statements of Operations and Comprehensive Loss - USD ($) $ in Thousands | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2017 | Jun. 30, 2016 | Jun. 30, 2017 | Jun. 30, 2016 | |
Revenues: | ||||
Total revenues | $ 18 | $ 483 | $ 36 | $ 972 |
Operating expenses: | ||||
Research and development | 14,278 | 18,007 | 30,030 | 34,772 |
General and administrative | 7,057 | 3,664 | 11,016 | 8,767 |
Total operating expenses | 21,335 | 21,671 | 41,046 | 43,539 |
Loss from operations | (21,317) | (21,188) | (41,010) | (42,567) |
Other income (expense): | ||||
Interest and other income | 184 | 180 | 398 | 372 |
Interest and other expense | (603) | (90) | (1,149) | (114) |
Loss before income taxes | (21,736) | (21,098) | (41,761) | (42,309) |
Income tax benefit | 128 | 8 | 132 | 13 |
Net loss | (21,608) | (21,090) | (41,629) | (42,296) |
Other comprehensive loss: | ||||
Unrealized gain on short-term investments, net | 10 | 9 | 36 | 50 |
Comprehensive loss | $ (21,598) | $ (21,081) | $ (41,593) | $ (42,246) |
Net loss per share, basic and diluted (in dollars per share) | $ (0.41) | $ (0.40) | $ (0.78) | $ (0.80) |
Weighted average shares used to compute basic and diluted net loss per share (in shares) | 53,182,330 | 52,782,643 | 53,086,887 | 52,746,657 |
Strategic Alliances and Collaborations | ||||
Revenues: | ||||
Total revenues | $ 18 | $ 483 | $ 36 | $ 972 |
Condensed Statements of Cash Fl
Condensed Statements of Cash Flows - USD ($) $ in Thousands | 6 Months Ended | |
Jun. 30, 2017 | Jun. 30, 2016 | |
Operating activities | ||
Net loss | $ (41,629) | $ (42,296) |
Adjustments to reconcile net loss to net cash used in operating activities | ||
Depreciation and amortization expense | 1,357 | 928 |
Stock-based compensation | 5,247 | 5,986 |
Amortization of premium on investments, net | 181 | 359 |
Other | 190 | 45 |
Change in operating assets and liabilities: | ||
Contracts and other receivables | 967 | 9,878 |
Prepaid materials | (597) | 2,834 |
Prepaid expenses and other assets | 1,106 | (1,970) |
Accounts payable | (907) | 787 |
Accrued liabilities | (814) | (1,063) |
Accrued compensation | (885) | (529) |
Deferred revenue | (36) | (972) |
Deferred rent and other liabilities | (187) | (262) |
Net cash used in operating activities | (36,007) | (26,275) |
Investing activities | ||
Purchases of short-term investments | (4,453) | (30,231) |
Sales and maturities of short-term investments | 37,929 | 47,522 |
Purchases of property and equipment | (124) | (266) |
Acquisition of intangibles | (16) | (34) |
Net cash provided by investing activities | 33,336 | 16,991 |
Financing activities | ||
Proceeds from borrowing under term loan, net | 0 | 19,819 |
Proceeds from issuance of common stock | 265 | 363 |
Proceeds from exercise of common stock options | 3 | 265 |
Principal payments on other long-term obligations | 0 | (83) |
Net cash provided by financing activities | 268 | 20,364 |
Net (decrease) increase in cash and cash equivalents | (2,403) | 11,080 |
Cash and cash equivalents at beginning of period | 14,941 | 15,960 |
Cash and cash equivalents at end of period | 12,538 | 27,040 |
Supplemental disclosure of cash flow information | ||
Net changes in restricted cash | 0 | (794) |
Interest paid | (946) | (54) |
Income taxes paid | (1) | (1) |
Supplemental disclosure of non-cash investing and financing activities | ||
Allowance for tenant improvements | 0 | 6,545 |
Amounts accrued for property and equipment | 110 | 221 |
Amounts accrued for patent expenditures | 0 | 7 |
Unpaid debt issuance costs | $ 0 | $ 38 |
Basis of Presentation and Summa
Basis of Presentation and Summary of Significant Accounting Policies | 6 Months Ended |
Jun. 30, 2017 | |
Accounting Policies [Abstract] | |
Basis of Presentation and Summary of Significant Accounting Policies | Basis of Presentation and Summary of Significant Accounting Policies Basis of Presentation The accompanying unaudited condensed financial statements have been prepared in accordance with U.S. generally accepted accounting principles (“GAAP”) for interim financial information and the instructions to Form 10-Q and Article 10 of Regulation S-X. Accordingly, they do not include all of the information and footnotes required by GAAP for complete financial statements. In management’s opinion, the accompanying financial statements reflect all adjustments, consisting of normal recurring adjustments, considered necessary for a fair presentation of the results for the interim periods presented. Interim financial results are not necessarily indicative of results anticipated for the full year. These unaudited condensed financial statements should be read in conjunction with the audited financial statements and footnotes included in our Annual Report on Form 10-K for the year ended December 31, 2016 , from which the balance sheet information herein was derived. We have incurred losses in each year since our inception. We expect to continue to incur significant expenses and operating losses for the foreseeable future in connection with our research and preclinical and clinical development of our product candidates. In order to continue to fund our research and development activities, we will need to seek additional capital. This may occur through strategic alliance and licensing arrangements and/or future public or private debt or equity financings. Sufficient funding may not be available, or if available, may be on terms that significantly dilute or otherwise adversely affect the rights of existing stockholders. If adequate funds are not available in the future, we may need to delay, reduce the scope of or put on hold one or more or our clinical and/or preclinical programs while we seek strategic alternatives. In May 2017, we implemented a corporate restructuring to streamline our operations, reduce our operating expenses, extend our cash runway and focus our resources on our most promising programs. In connection with the restructuring, we committed to a reduction in our total workforce by approximately 30% percent, to approximately 65 employees. We completed the workforce reduction in June 2017. We recorded charges of approximately $3.2 million for employee severance and other related termination benefits, including $1.3 million in net adjustments to non-cash stock-based compensation. All payments associated with the corporate restructuring were paid in full as of June 30, 2017. As of June 30, 2017, we had cash, cash equivalents and short-term investments of $40.1 million . We adopted Financial Accounting Standards Board, or FASB, Accounting Standards Update, or ASU, 2014-15, Presentation of Financial Statements - Going Concern , effective December 31, 2016. We have evaluated and concluded that there are no conditions or events, considered individually or in the aggregate, that raise substantial doubt about our ability to continue as a going concern for a period of one year following the date that these financial statements are issued. Use of Estimates Our condensed financial statements are prepared in accordance with GAAP, which requires us to make estimates and assumptions that affect the reported amounts of assets, liabilities, revenues and expenses and the disclosure of contingent assets and liabilities in our financial statements and accompanying notes. An estimated loss contingency is accrued in our financial statements if it is probable that a liability has been incurred and the amount of the loss can be reasonably estimated. Although these estimates are based on our knowledge of current events and actions we may undertake in the future, actual results may ultimately differ from these estimates and assumptions. Revenue Recognition Our revenues generally consist of upfront payments for licenses or options to obtain licenses in the future, milestone payments and payments for other research services under strategic alliance and collaboration agreements. We recognize revenues when all four of the following criteria are met: (1) persuasive evidence of an arrangement exists; (2) delivery of the products and/or services has occurred; (3) the selling price is fixed or determinable; and (4) collectability is reasonably assured. Multiple element arrangements, such as our strategic alliance agreement with Sanofi, are analyzed to determine whether the deliverables within the agreement can be separated or whether they must be accounted for as a single unit of accounting. Deliverables under the agreement will be accounted for as separate units of accounting provided that (i) a delivered item has value to the customer on a stand-alone basis; and (ii) if the agreement includes a general right of return relative to the delivered item, delivery or performance of the undelivered item is considered probable and substantially in the control of the vendor. The allocation of consideration amongst the deliverables under the agreement is derived using a “best estimate of selling price” if vendor specific objective evidence and third-party evidence of fair value is not available. If the delivered element does not have stand-alone value, the arrangement is then accounted for as a single unit of accounting, and we recognize the consideration received under the arrangement as revenue on a straight-line basis, which approximates effort over our estimated period of performance, which for us is typically the expected term of the research and development plan. Milestones We apply the milestone method of accounting to recognize revenue from milestone payments when earned, as evidenced by written acknowledgment from the collaborator or other persuasive evidence that the milestone has been achieved and the payment is non-refundable, provided that the milestone event is substantive. A milestone event is defined as an event (i) that can only be achieved based in whole or in part on either our performance or on the occurrence of a specific outcome resulting from our performance; (ii) for which there is substantive uncertainty at the inception of the arrangement that the event will be achieved; and (iii) that would result in additional payments being due to us. Events for which the occurrence is either contingent solely upon the passage of time or the result of a counterparty’s performance are not considered to be milestone events. A milestone event is substantive if all of the following conditions are met: (i) the consideration is commensurate with either our performance to achieve the milestone, or the enhancement of the value to the delivered item(s) as a result of a specific outcome resulting from our performance to achieve the milestone; (ii) the consideration relates solely to past performance; and (iii) the consideration is reasonable relative to all the deliverables and payment terms (including other potential milestone consideration) within the arrangement. We assess whether a milestone is substantive at the inception of each arrangement. If a milestone is deemed non-substantive, we will account for that milestone payment using a method consistent with the related units of accounting for the arrangement over the estimated performance period. Deferred Revenue Amounts received prior to satisfying the above revenue recognition criteria are recorded as deferred revenue in the accompanying balance sheets. Amounts not expected to be recognized within the next 12 months are classified as non-current deferred revenue. Stock-Based Compensation We account for stock-based compensation expense related to stock options granted to employees and members of our board of directors by estimating the fair value of each stock option on the date of grant using the Black-Scholes option pricing model. We recognize stock-based compensation expense using the accelerated multiple-option approach. Under the accelerated multiple-option approach (also known as the graded-vesting method), we recognize compensation expense over the requisite service period for each separately vesting tranche of the award as though the award was in substance multiple awards, resulting in accelerated expense recognition over the vesting period. For performance-based awards granted to employees (i) the fair value of the award is determined on the grant date, (ii) we assess the probability of the individual milestones under the award being achieved and (iii) the fair value of the shares subject to the milestone is expensed over the implicit service period commencing once management believes the performance criteria is probable of being met. We account for stock options granted to non-employees using the fair value approach. Stock options granted to non-employees are subject to periodic revaluation over their vesting terms. Clinical Trial and Preclinical Study Accruals We make estimates of our accrued expenses for clinical trial and preclinical study activities as of each balance sheet date in our financial statements based on the facts and circumstances known to us at that time. These accruals are based upon estimates of costs incurred and fees that may be associated with services provided by clinical trial investigational sites, clinical research organizations (“CROs”) and for other clinical trial-related activities. Payments under certain contracts with such parties depend on factors such as successful enrollment of patients, site initiation and the completion of clinical trial milestones. In accruing for these services, we estimate the time period over which services will be performed and the level of effort to be expended in each period. If possible, we obtain information regarding unbilled services directly from these service providers. However, we may be required to estimate these services based on other information available to us. If we underestimate or overestimate the activities or fees associated with a study or service at a given point in time, adjustments to research and development expenses may be necessary in future periods. Historically, our estimated accrued liabilities have approximated actual expense incurred. Subsequent changes in estimates may result in a material change in our accruals. Prepaid Materials We capitalize the purchase of certain raw materials and related supplies for use in the manufacturing of drug product in our clinical development programs, as we have determined that these materials have alternative future use. We can use these raw materials and related supplies in multiple clinical drug products, and therefore have future use independent of the development status of any particular drug program until it is utilized in the manufacturing process. We periodically review these capitalized materials for indicators of impairment, including shelf life, continued alternative future use and obsolescence, and write down the asset to its net realizable value in the period it which it is identified. As of June 30, 2017 and December 31, 2016 , our prepaid materials balance was $6.1 million, net of a $0.6 million reserve, and $5.6 million , net of a $0.6 million reserve, respectively. Recent Accounting Pronouncements In May 2014, the FASB issued ASU No. 2014-09, Revenue from Contracts with Customers (Topic 606) , which outlines a single comprehensive model for entities to use in accounting for revenue arising from contracts with customers and supersedes most current revenue recognition guidance, including industry-specific guidance. ASU 2014-09 outlines a five-step process for revenue recognition that focuses on transfer of control, as opposed to transfer of risk and rewards, and also requires enhanced disclosures regarding the nature, amount, timing and uncertainty of revenues and cash flows from contracts with customers. Major provisions include determining which goods and services are distinct and require separate accounting (performance obligations), how variable consideration (which may include change orders and claims) is recognized, whether revenue should be recognized at a point in time or over time and ensuring the time value of money is considered in the transaction price. The FASB issued supplemental adoption guidance and clarification to ASU No. 2014-09 in March 2016, April 2016 and May 2016 within ASU No. 2016-08, Revenue from Contracts with Customers: Principal vs. Agent Considerations , ASU No. 2016-10, Revenue from Contracts with Customers: Identifying Performance Obligations and Licensing and ASU No. 2016-12, Revenue from Contracts with Customers: Narrow-Scope Improvements and Practical Expedients , respectively. ASU No. 2014-09 and the related supplemental ASUs are effective for fiscal years beginning after December 15, 2017 and interim periods therein. The ASU permits two methods of adoption: the full retrospective method or the modified retrospective method. We plan to apply the modified retrospective method upon adoption in the first quarter of 2018 and currently do not anticipate that the adoption of this ASU will have a material impact with regard to our current contracts. In January 2016, the FASB issued ASU No. 2016-01, Recognition and Measurement of Financial Assets and Financial Liabilities , which eliminates the requirement for public companies to disclose the method(s) and significant assumptions used to estimate the fair value for financial instruments measured at amortized cost on the balance sheet. Additionally, the standard requires public companies to use the exit price notion when measuring the fair value of financial instruments for disclosure purposes. Furthermore, the standard requires presentation of financial assets and liabilities by measurement category and form of financial asset on the balance sheet or accompanying notes to the financial statements. The standard is effective for annual reporting periods beginning after December 15, 2017, including interim periods within those annual reporting periods. Early application is permitted. The adoption of this guidance will have no impact on our financial statements. In February 2016, the FASB issued ASU No. 2016-02, Leases, which increases transparency and comparability among organizations by requiring recognition of lease assets and lease liabilities on the balance sheet and disclosure of key information about leasing arrangements. The standard is effective for annual reporting periods beginning after December 15, 2018, including interim periods within those annual reporting periods. Early application is permitted. We are currently evaluating the impact of adoption on our financial statements. In March 2016, the FASB issued ASU No. 2016-09, Compensation – Stock Compensation: Improvements to Employee Share-Based Payment Accounting, which was intended to simplify various aspects of accounting for share-based payment transactions. The new guidance requires immediate recognition of all excess tax benefits and deficiencies in the income statement, requires classification of excess tax benefits as an operating activity as opposed to a financing activity in the statements of cash flows and allows the Company to make an accounting policy election to either estimate the number of awards expected to vest or account for forfeitures when they occur. The standard is effective for annual reporting periods beginning after December 15, 2016, and interim periods within those annual reporting periods. We applied this standard in the first quarter of 2017 using the modified retrospective method of adoption. In conjunction with this adoption, we applied an accounting policy election to account for forfeitures as they occur. Upon adoption, we reversed a deferred tax asset related to the balance of unrecognized excess tax benefits of $7.4 million , with an offsetting adjustment to the valuation allowance. Under the modified retrospective method of adoption, we recorded an adjustment of $0.6 million to accumulated deficit with a corresponding offset to additional paid-in capital. In August 2016, the FASB issued ASU No. 2016-15, Statement of Cash Flows: Classification of Certain Cash Receipts and Cash Payments , which addresses the presentation and classification of certain cash receipts and cash payments in the statement of cash flows under Accounting Standards Codification 230. The standard is effective for annual reporting periods beginning after December 15, 2017, and interim periods within those annual reporting periods. The adoption of this guidance will have no impact on our financial statements. In November 2016, the FASB issued ASU No. 2016-18, Statement of Cash Flows: Restricted Cash , which requires restricted cash and restricted cash equivalents to be included with cash and cash equivalents when reconciling the beginning-of-period and end-of-period total amounts shown on the statement of cash flows. The standard is effective for annual reporting periods beginning after December 15, 2017, and interim periods within those annual reporting periods. Early application is permitted. Upon its adoption in 2018, we will include $1.3 million of restricted cash in our disclosed balance of cash and cash equivalents at the beginning of the period for 2016. We do not expect any additional impact on our financial statements. In May 2017, the FASB issued ASU No. 2017-09, Compensation - Stock Compensation: Scope of Modification Accounting , which provides clarity and guidance around which changes to the terms or conditions of a share-based payment award require an entity to apply modification accounting in Topic 718. The standard is effective for annual reporting periods beginning after December 15, 2017, and interim periods within those annual reporting periods. The adoption of this guidance will have no impact on our financial statements unless we have modification accounting in accordance with Topic 718. |
Net Loss Per Share
Net Loss Per Share | 6 Months Ended |
Jun. 30, 2017 | |
Earnings Per Share [Abstract] | |
Net Loss Per Share | Net Loss Per Share Basic net loss per share is calculated by dividing net loss by the weighted average number of common shares outstanding for the period, without consideration for common stock equivalents. Diluted net loss per share is calculated by dividing net loss by the weighted-average number of common share equivalents outstanding for the period determined using the treasury-stock method. Dilutive common stock equivalents are comprised of options outstanding under our stock option plans. For all periods presented, there is no difference in the number of shares used to calculate basic and diluted net loss per share. Potentially dilutive securities not included in the calculation of diluted net loss per share because to do so would be anti-dilutive consisted of 2,140,111 and 2,869,643 shares attributable to common stock options for the three and six months ended June 30, 2017 , respectively, compared to 3,055,143 and 2,639,630 shares attributable to common stock options for the same periods in 2016. |
Investments
Investments | 6 Months Ended |
Jun. 30, 2017 | |
Investments, Debt and Equity Securities [Abstract] | |
Investments | Investments We invest our excess cash primarily in commercial paper and debt instruments of financial institutions, corporations, U.S. government-sponsored agencies and the U.S. Treasury. As of June 30, 2017 , our short-term investments had a weighted average maturity of less than one year. The following tables summarize our short-term investments (in thousands): Maturity (in years) Amortized cost Unrealized Estimated fair value Gains Losses As of June 30, 2017 Corporate debt securities 1 or less $ 21,264 $ — $ (9 ) $ 21,255 Certificates of deposit 1 or less 3,785 — — 3,785 U.S. Treasury securities 1 or less 2,010 — (1 ) 2,009 Debt securities of U.S. government-sponsored agencies 1 or less 500 — — 500 Total $ 27,559 $ — $ (10 ) $ 27,549 Maturity (in years) Amortized cost Unrealized Estimated fair value Gains Losses As of December 31, 2016 Corporate debt securities 2 or less $ 49,185 $ 12 $ (77 ) $ 49,120 Certificates of deposit 1 or less 9,291 — — 9,291 Commercial paper 1 or less 1,247 — — 1,247 U.S. Treasury securities 1 or less 1,001 — (1 ) 1,000 Debt securities of U.S. government-sponsored agencies 1 or less 512 — — 512 Total $ 61,236 $ 12 $ (78 ) $ 61,170 |
Fair Value Measurements
Fair Value Measurements | 6 Months Ended |
Jun. 30, 2017 | |
Fair Value Disclosures [Abstract] | |
Fair Value Measurements | Fair Value Measurements We have certain financial assets recorded at fair value which have been classified as Level 1, 2, or 3 within the fair value hierarchy as described in the accounting standards for fair value measurements. Accounting standards define fair value as the exchange price that would be received for an asset or paid to transfer a liability (an exit price) in the principal or most advantageous market for the asset or liability in an orderly transaction between market participants as of the measurement date. Market participants are buyers and sellers in the principal market that are (i) independent, (ii) knowledgeable, (iii) able to transact, and (iv) willing to transact. The accounting standards provide an established hierarchy for inputs used in measuring fair value that maximizes the use of observable inputs and minimizes the use of unobservable inputs by requiring that the most observable inputs be used when available. Observable inputs are inputs that market participants would use in valuing the asset or liability and are developed based on market data obtained from independent sources. Unobservable inputs are inputs that reflect our assumptions about the factors that market participants would use in valuing the asset or liability. The accounting standards prioritize the inputs used in measuring the fair value into the following hierarchy: • Level 1 includes financial instruments for which quoted market prices for identical instruments are available in active markets. • Level 2 includes financial instruments for which there are inputs other than quoted prices included within Level 1 that are observable for the instrument such as quoted prices for similar instruments in active markets, quoted prices for identical or similar instruments in markets with insufficient volume or infrequent transactions (less active markets) or model-driven valuations in which significant inputs are observable or can be derived principally from, or corroborated by, observable market data. • Level 3 includes financial instruments for which fair value is derived from valuation techniques in which one or more significant inputs are unobservable, including management’s own assumptions. Financial Assets Measured at Fair Value The following table presents our fair value hierarchy for assets measured at fair value on a recurring basis as of June 30, 2017 and December 31, 2016 (in thousands): Fair value as of June 30, 2017 Total Level 1 Level 2 Level 3 Assets: Cash equivalents $ 10,630 $ 10,630 $ — $ — Corporate debt securities 21,255 — 21,255 — Certificates of deposit 3,785 — 3,785 — U.S. treasury securities 2,009 — 2,009 — Debt securities of U.S. government-sponsored agencies 500 — 500 — $ 38,179 $ 10,630 $ 27,549 $ — Fair value as of December 31, 2016 Total Level 1 Level 2 Level 3 Assets: Cash equivalents $ 13,578 $ 13,578 $ — $ — Corporate debt securities 49,120 — 49,210 — Certificates of deposit 9,291 — 9,291 — Commercial paper 1,247 — 1,247 — U.S. treasury securities 1,000 — 1,000 — Debt securities of U.S. government-sponsored agencies 512 — 512 — $ 74,748 $ 13,578 $ 61,260 $ — We obtain pricing information from quoted market prices or quotes from brokers/dealers. We generally determine the fair value of our investment securities using standard observable inputs, including reported trades, broker/dealer quotes, bids and/or offers. Refer to Note 3 for information regarding our investments. |
Term Loan
Term Loan | 6 Months Ended |
Jun. 30, 2017 | |
Debt Disclosure [Abstract] | |
Term Loan | Term Loan On June 17, 2016, we entered into a loan and security agreement ("Loan Agreement") with Oxford Finance, LLC, ("Oxford"), pursuant to which Oxford agreed to lend us up to $30.0 million , issuable in two separate term loans of $20.0 million (the "Term A Loan") and $10.0 million (the "Term B Loan"). On June 22, 2016, we received $20.0 million in proceeds from the Term A Loan, net of debt issuance costs. The ability to borrow on the Term B Loan expired on March 31, 2017. We refer to all amounts outstanding under the Loan Agreement as the Term Loan. The outstanding Term Loan will mature on June 1, 2020 (the “Maturity Date”) and we will have interest-only payments through June 1, 2018, followed by 24 equal monthly payments of principal and unpaid accrued interest. The Term Loan will bear interest at a floating per annum rate equal to (i) 8.51% plus (ii) the greater of (a) the 30 day U.S. Dollar LIBOR rate reported in The Wall Street Journal on the last business day of the month that immediately precedes the month in which the interest will accrue and (b) 0.44% . We have the option to prepay all, but not less than all, of the borrowed amount, provided that we will be obligated to pay a prepayment fee equal to (i) 2% of the outstanding principal balance of the Term Loan if prepayment is made prior to the second anniversary of the funding date of the Term Loan, provided no prepayment fee would have been due in connection with a prepayment made on or prior to the first anniversary of the funding date of the Term Loan in connection with an acquisition of our company, or (ii) 1% of the Term Loan prepaid thereafter and prior to the Maturity Date. We will be required to make a final payment of 5.5% of the principal balance outstanding, payable on the earlier of (i) the Maturity Date, (ii) acceleration of the Term Loan, or (iii) the prepayment of the Term Loan. We may use the proceeds from the Term Loan solely for working capital and to fund our general business requirements. Our obligations under the Loan Agreement are secured by a first priority security interest in substantially all of our current and future assets, other than our intellectual property. We have also agreed not to encumber our intellectual property assets, except as permitted by the Loan Agreement. The Loan Agreement defines certain events of default, including instances of a material adverse change in our operations, that may require prepayment of the outstanding Term Loan. No such events have occurred or are anticipated as of June 30, 2017 . As of June 30, 2017 , we had $20.0 million outstanding under the Term Loan. The Term Loan was recorded at its initial carrying value of $20.0 million , less debt issuance costs of approximately $0.2 million . In connection with the Term Loan, the debt issuance costs have been recorded as a debt discount in our consolidated balance sheets, which are being accreted to interest expense over the life of the Term Loan using an effective interest rate of 8.98% . The exit fee is being accrued over the life of the Term Loan through interest expense. As of June 30, 2017 , we were in compliance with all covenants under the Loan Agreement. Future principal payments for the Term Loan due under the Loan Agreement are as follows (in thousands): 2017 $ — 2018 5,000 2019 10,000 2020 5,000 $ 20,000 |
Stockholders' Equity
Stockholders' Equity | 6 Months Ended |
Jun. 30, 2017 | |
Disclosure of Compensation Related Costs, Share-based Payments [Abstract] | |
Stockholders' Equity | Stockholders’ Equity Shares Reserved for Future Issuance The following shares of common stock were reserved for future issuance as of June 30, 2017 : Common stock options outstanding 10,819,036 Common stock available for future grant under 2012 Equity Incentive Plan 917,027 Common stock available for future grant under 2015 Inducement Plan 23,126 Employee Stock Purchase Plan 1,844,527 Total common shares reserved for future issuance 13,603,716 The following table summarizes our stock option activity under all equity incentive plans for the six months ended June 30, 2017 (shares in thousands): Number of options Weighted average exercise price Options outstanding at December 31, 2016 8,931 $ 6.70 Granted 4,255 $ 1.44 Exercised (8 ) $ 0.38 Canceled/forfeited/expired (2,359 ) $ 5.86 Options outstanding at June 30, 2017 10,819 $ 4.82 Stock-Based Compensation The following table summarizes the weighted average assumptions used to estimate the fair value of stock options and performance stock awards granted to employees under our 2012 Equity Incentive Plan and 2015 Inducement Plan and the shares purchasable under our Employee Stock Purchase Plan during the periods presented: Three months ended Six months ended 2017 2016 2017 2016 Stock options Risk-free interest rate 1.9 % 1.5 % 2.0 % 1.4 % Volatility 89.5 % 79.1 % 89.4 % 79.7 % Dividend yield — — — — Expected term (years) 6.1 5.8 6.1 5.9 Performance stock options Risk-free interest rate — 0.4 % 2.1 % 1.4 % Volatility — 78.5 % 89.9 % 79.3 % Dividend yield — — — — Expected term (years) 0 5.5 5.6 6.0 Employee stock purchase plan shares Risk-free interest rate 0.9 % 0.5 % 0.8 % 0.4 % Volatility 111.5 % 82.6 % 113.8 % 81.0 % Dividend yield — — — — Expected term (years) 0.5 0.5 0.5 0.5 The following table summarizes the allocation of our stock-based compensation expense for all stock awards during the periods presented, including the adjustments to stock-based compensation expense associated with our May 2017 corporate restructuring (in thousands): Three months ended Six months ended 2017 2016 2017 2016 Research and development $ 650 $ 1,299 $ 1,758 $ 2,731 Research and development-restructuring related adjustments (1,399 ) — (1,399 ) — General and administrative 935 939 2,209 3,255 General and administrative-restructuring related adjustments 2,679 — 2,679 — Total $ 2,865 $ 2,238 $ 5,247 $ 5,986 In connection with our May 2017 corporate restructuring, we recorded a reversal of stock-based compensation in research and development expenses of $1.4 million for each of the three and six months ended June 30, 2017, as a result of the cancellation of unvested stock options. We recorded additional stock-based compensation in general and administrative expenses of $2.7 million for each of the three and six months ended June 30, 2017, as a result of termination provisions within certain employment agreements. |
Strategic Alliances and Collabo
Strategic Alliances and Collaborations | 6 Months Ended |
Jun. 30, 2017 | |
Organization, Consolidation and Presentation of Financial Statements [Abstract] | |
Strategic Alliances and Collaborations | Strategic Alliances and Collaborations The following table summarizes our total revenues from our strategic alliances and collaborations during the periods presented (in thousands): Three months ended Six months ended 2017 2016 2017 2016 Sanofi $ 18 $ 18 $ 36 $ 36 AstraZeneca — 465 — 936 Total $ 18 $ 483 $ 36 $ 972 Sanofi In July 2012, we amended and restated our collaboration and license agreement with Sanofi to expand the potential therapeutic applications of the micro RNA alliance targets to be developed under such agreement. We determined that the elements within the strategic alliance agreement with Sanofi should be treated as a single unit of accounting because the delivered elements did not have stand-alone value to Sanofi. The following elements were delivered as part of the strategic alliance with Sanofi: (1) a license for up to four micro RNA targets; and (2) a research license under our technology alliance. In June 2013, the original research term expired, upon which we and Sanofi entered into an option agreement pursuant to which Sanofi was granted an exclusive right to negotiate the co-development and commercialization of certain of our unencumbered micro RNA programs and we were granted the exclusive right to negotiate with Sanofi for co-development and commercialization of certain miR-21 anti-miRs in oncology and Alport syndrome. In July 2013, we received an upfront payment of $2.5 million , of which $1.25 million is creditable against future amounts payable by Sanofi to us under any future co-development and commercialization agreement we enter pursuant to the option agreement. Revenue associated with the creditable portion of this option payment remained deferred as of June 30, 2017 , and will remain deferred until its application to a creditable transaction. The non-creditable portion of this payment, $1.25 million , was recognized as revenue over the option period from the effective date of the option agreement in June 2013 through the expiration of the option period in January 2014. In February 2014, we and Sanofi entered into a second amended and restated collaboration and license agreement (the “2014 Sanofi Amendment”) to renew our strategic alliance to discover, develop and commercialize micro RNA therapeutics to focus on specific orphan disease and oncology targets. Under the terms of our renewed alliance, Sanofi will have opt-in rights to our clinical fibrosis program targeting miR-21 for the treatment of Alport syndrome, our preclinical program targeting miR-21 for oncology indications, and our preclinical program targeting miR-221/222 for hepatocellular carcinoma (“HCC”). We are responsible for developing each of these programs to proof-of-concept, at which time Sanofi has an exclusive option on each program. If Sanofi chooses to exercise its option on any of these programs, Sanofi will reimburse us for a significant portion of our preclinical and clinical development costs and will also pay us an option exercise fee for any such program, provided that $1.25 million of the $2.5 million upfront option fee paid to us by Sanofi in connection with the June 2013 option agreement will be creditable against such option exercise fee. We are eligible to receive royalties on micro RNA therapeutic products commercialized by Sanofi and will have the right to co-promote these products. In connection with the 2014 Sanofi Amendment, we entered into a Common Stock Purchase Agreement (the “Purchase Agreement”), pursuant to which we sold 1,303,780 shares of our common stock to Aventisub LLC (formerly Aventis Holdings, Inc.) (“Aventis”), an entity affiliated with Sanofi, in a private placement at a price per share of $7.67 for an aggregate purchase price of $10.0 million . Under the terms of the Purchase Agreement, Aventis was not permitted to sell, transfer, make any short sale of, or grant any option for the sale of any common stock for the 12 -month period following its effective date. The Purchase Agreement and the 2014 Sanofi Amendment were negotiated concurrently and were therefore evaluated as a single agreement. Based upon restricted stock studies of similar duration and a Black-Scholes valuation to measure the discount for lack of marketability, approximately $0.4 million of the proceeds from the Purchase Agreement was attributed to the 2014 Sanofi Amendment, and represents consideration for the value of the program targeting miR-221/222 for HCC. As this element does not have stand-alone value, we are recognizing the $0.4 million allocated consideration into revenue ratably over the estimated period of performance of the miR-221/222 program. As of June 30, 2017 , deferred revenue associated with the Purchase Agreement and the 2014 Sanofi Amendment was $0.2 million , which we are expecting to recognize over the remaining estimated period of performance of approximately three years. We are eligible to receive milestone payments of up to $101.8 million for proof-of-concept option exercise fees (net of $1.25 million creditable, as noted above), $15.0 million for clinical milestones and up to $300.0 million for regulatory and commercial milestones. In addition, we are entitled to receive royalties based on a percentage of net sales of any products from the miR-21 and miR-221/222 programs which, in the case of sales in the United States, will be in the middle of the 10 to 20% range, and, in the case of sales outside of the United States, will range from the low end to the middle of the 10 to 20% range, depending upon the volume of sales. If we exercise our option to co-promote a product, we will continue to be eligible to receive royalties on net sales of each product in the United States at the same rate, unless we elect to share a portion of Sanofi’s profits from sales of such product in the United States in lieu of royalties. We have evaluated the contingent event-based payments under the 2014 Sanofi Amendment and determined that the milestone payments meet the definition of substantive milestones. Accordingly, revenue for these achievements will be recognized in their entirety in the period when the milestone is achieved and collectability is reasonably assured. Other contingent event-based payments under the 2014 Sanofi Amendment for which payment is contingent upon the results of Sanofi’s performance will not be accounted for using the milestone method. Such payments will be recognized as revenue over the remaining estimated period of performance, if any, and when collectability is reasonably assured. AstraZeneca In August 2012, we entered into a collaboration and license agreement with AstraZeneca. Under the terms of the agreement, we agreed to collaborate with AstraZeneca to identify, research and develop compounds targeting three micro RNA alliance targets primarily in the fields of cardiovascular diseases, metabolic diseases and oncology. Pursuant to the agreement, we granted AstraZeneca an exclusive, worldwide license to develop, manufacture and commercialize lead compounds designated by AstraZeneca in the course of the collaboration activities against the alliance targets for all human therapeutic uses. Under the terms of the agreement we were required to use commercially reasonable efforts to perform all research, development and manufacturing activities described in the research plan, at our cost, until the acceptance of an investigational new drug application ("IND") or the end of the research term, which expired in August 2016. Under the terms of the agreement, we received an upfront payment of $3.0 million in October 2012. We determined the elements within the agreement should be treated as a single unit of accounting because the delivered element, the license, did not have stand-alone value. As a result, we recognized revenue related to the upfront payment on a straight-line basis over the period of performance, which was four years based on the term of the research and development plan which expired in August 2016. In connection with the collaboration and license agreement and concurrently with our initial public offering, we sold AstraZeneca 6,250,000 shares of our common stock in a private placement at a price per share of $4.00 . Under the terms of the Common Stock Purchase Agreement ("CSPA"), AstraZeneca could not sell, transfer, make any short sale of, or grant any option for the sale of any common stock for a 365 -day period following the effective date of our initial public offering. The CSPA and collaboration and license agreement were negotiated concurrently and were therefore evaluated as a single agreement. Based upon restricted stock studies of similar duration and a Black-Scholes valuation to measure a discount for lack of marketability, $4.3 million was attributed to the collaboration and license agreement. We recognized the $4.3 million into revenue ratably over the period of performance of the research and development plan under the collaboration, which expired in August 2016. In March 2015, we earned a $2.5 million preclinical milestone and in December 2015, we earned a $10.0 million clinical milestone. We determined the milestones to be substantive and recognized revenue upon achievement of each milestone. In June 2017, AstraZeneca delivered written notice of their election to terminate the collaboration and license agreement. Effective upon the termination of the agreement, AstraZeneca’s rights with respect to RG-125(AZD4076) will revert back to us. In accordance with the Agreement, the termination will become effective in June 2018, 12 months following the date of delivery of the notice by AstraZeneca. |
Related Party Transactions
Related Party Transactions | 6 Months Ended |
Jun. 30, 2017 | |
Related Party Transactions [Abstract] | |
Related Party Transactions | Related Party Transactions We have entered into certain agreements with related parties in the ordinary course of business to license intellectual property and to procure research and development support services. In September 2014, we entered into an agreement with Sanofi-Aventis Deutschland GmbH (“Sanofi Deutschland”), a contract manufacturing subsidiary of Sanofi, for the manufacture of certain drug substance requirements and other services to support our preclinical and clinical activities associated with the RG-012 program. Pursuant to this agreement, we engaged Sanofi Deutschland to manufacture RG-012 drug product and perform stability studies on our behalf. Expenses incurred under the agreement for services performed or out-of-pocket expenses were less than $0.1 million for the three and six months ended June 30, 2017 , respectively, compared to less than $0.1 million and $0.8 million for the same periods in 2016. In February 2015, we entered into a letter agreement with Alnylam Pharmaceuticals, Inc. ("Alnylam") pursuant to which we and Alnylam agreed to the financial terms for certain technology acquired by Alnylam within the licensed patent rights under our Amended and Restated License and Collaboration Agreement (the “Additional Patent Rights”) with Alnylam and Ionis Pharmaceuticals, Inc. In addition to any royalties payable by us to Alnylam pursuant to the terms of the Amended and Restated License and Collaboration Agreement, we agreed to pay Alnylam an additional low single-digit royalty on net sales of certain products utilizing the Additional Patent Rights, with the exact royalty percentage payable being dependent on the total amount of net sales during the calendar year. We also agreed to pay Alnylam milestone payments on certain products utilizing the additional patent rights of up to $33.0 million per product upon the achievement of certain regulatory milestone events. There was no activity under this agreement for the three or six months ended June 30, 2017 and 2016 . |
Subsequent Events
Subsequent Events | 6 Months Ended |
Jun. 30, 2017 | |
Subsequent Events [Abstract] | |
Subsequent Events | Subsequent Events In July 2017, we completed an underwritten public offering of 50,600,000 shares of common stock at an offering price of $0.91 per share. We received net proceeds from the offering of approximately $43.0 million after deducting underwriting discounts, commissions and other estimated offering expenses payable by us. |
Basis of Presentation and Sum15
Basis of Presentation and Summary of Significant Accounting Policies (Policies) | 6 Months Ended |
Jun. 30, 2017 | |
Accounting Policies [Abstract] | |
Basis of Presentation | Basis of Presentation The accompanying unaudited condensed financial statements have been prepared in accordance with U.S. generally accepted accounting principles (“GAAP”) for interim financial information and the instructions to Form 10-Q and Article 10 of Regulation S-X. Accordingly, they do not include all of the information and footnotes required by GAAP for complete financial statements. In management’s opinion, the accompanying financial statements reflect all adjustments, consisting of normal recurring adjustments, considered necessary for a fair presentation of the results for the interim periods presented. Interim financial results are not necessarily indicative of results anticipated for the full year. These unaudited condensed financial statements should be read in conjunction with the audited financial statements and footnotes included in our Annual Report on Form 10-K for the year ended December 31, 2016 , from which the balance sheet information herein was derived. |
Use of Estimates | Use of Estimates Our condensed financial statements are prepared in accordance with GAAP, which requires us to make estimates and assumptions that affect the reported amounts of assets, liabilities, revenues and expenses and the disclosure of contingent assets and liabilities in our financial statements and accompanying notes. An estimated loss contingency is accrued in our financial statements if it is probable that a liability has been incurred and the amount of the loss can be reasonably estimated. Although these estimates are based on our knowledge of current events and actions we may undertake in the future, actual results may ultimately differ from these estimates and assumptions. |
Revenue Recognition | Revenue Recognition Our revenues generally consist of upfront payments for licenses or options to obtain licenses in the future, milestone payments and payments for other research services under strategic alliance and collaboration agreements. We recognize revenues when all four of the following criteria are met: (1) persuasive evidence of an arrangement exists; (2) delivery of the products and/or services has occurred; (3) the selling price is fixed or determinable; and (4) collectability is reasonably assured. Multiple element arrangements, such as our strategic alliance agreement with Sanofi, are analyzed to determine whether the deliverables within the agreement can be separated or whether they must be accounted for as a single unit of accounting. Deliverables under the agreement will be accounted for as separate units of accounting provided that (i) a delivered item has value to the customer on a stand-alone basis; and (ii) if the agreement includes a general right of return relative to the delivered item, delivery or performance of the undelivered item is considered probable and substantially in the control of the vendor. The allocation of consideration amongst the deliverables under the agreement is derived using a “best estimate of selling price” if vendor specific objective evidence and third-party evidence of fair value is not available. If the delivered element does not have stand-alone value, the arrangement is then accounted for as a single unit of accounting, and we recognize the consideration received under the arrangement as revenue on a straight-line basis, which approximates effort over our estimated period of performance, which for us is typically the expected term of the research and development plan. Milestones We apply the milestone method of accounting to recognize revenue from milestone payments when earned, as evidenced by written acknowledgment from the collaborator or other persuasive evidence that the milestone has been achieved and the payment is non-refundable, provided that the milestone event is substantive. A milestone event is defined as an event (i) that can only be achieved based in whole or in part on either our performance or on the occurrence of a specific outcome resulting from our performance; (ii) for which there is substantive uncertainty at the inception of the arrangement that the event will be achieved; and (iii) that would result in additional payments being due to us. Events for which the occurrence is either contingent solely upon the passage of time or the result of a counterparty’s performance are not considered to be milestone events. A milestone event is substantive if all of the following conditions are met: (i) the consideration is commensurate with either our performance to achieve the milestone, or the enhancement of the value to the delivered item(s) as a result of a specific outcome resulting from our performance to achieve the milestone; (ii) the consideration relates solely to past performance; and (iii) the consideration is reasonable relative to all the deliverables and payment terms (including other potential milestone consideration) within the arrangement. We assess whether a milestone is substantive at the inception of each arrangement. If a milestone is deemed non-substantive, we will account for that milestone payment using a method consistent with the related units of accounting for the arrangement over the estimated performance period. Deferred Revenue Amounts received prior to satisfying the above revenue recognition criteria are recorded as deferred revenue in the accompanying balance sheets. Amounts not expected to be recognized within the next 12 months are classified as non-current deferred revenue. |
Stock-Based Compensation | Stock-Based Compensation We account for stock-based compensation expense related to stock options granted to employees and members of our board of directors by estimating the fair value of each stock option on the date of grant using the Black-Scholes option pricing model. We recognize stock-based compensation expense using the accelerated multiple-option approach. Under the accelerated multiple-option approach (also known as the graded-vesting method), we recognize compensation expense over the requisite service period for each separately vesting tranche of the award as though the award was in substance multiple awards, resulting in accelerated expense recognition over the vesting period. For performance-based awards granted to employees (i) the fair value of the award is determined on the grant date, (ii) we assess the probability of the individual milestones under the award being achieved and (iii) the fair value of the shares subject to the milestone is expensed over the implicit service period commencing once management believes the performance criteria is probable of being met. We account for stock options granted to non-employees using the fair value approach. Stock options granted to non-employees are subject to periodic revaluation over their vesting terms. |
Clinical Trial and Preclinical Study Accruals | Clinical Trial and Preclinical Study Accruals We make estimates of our accrued expenses for clinical trial and preclinical study activities as of each balance sheet date in our financial statements based on the facts and circumstances known to us at that time. These accruals are based upon estimates of costs incurred and fees that may be associated with services provided by clinical trial investigational sites, clinical research organizations (“CROs”) and for other clinical trial-related activities. Payments under certain contracts with such parties depend on factors such as successful enrollment of patients, site initiation and the completion of clinical trial milestones. In accruing for these services, we estimate the time period over which services will be performed and the level of effort to be expended in each period. If possible, we obtain information regarding unbilled services directly from these service providers. However, we may be required to estimate these services based on other information available to us. If we underestimate or overestimate the activities or fees associated with a study or service at a given point in time, adjustments to research and development expenses may be necessary in future periods. Historically, our estimated accrued liabilities have approximated actual expense incurred. Subsequent changes in estimates may result in a material change in our accruals. |
Prepaid Materials | Prepaid Materials We capitalize the purchase of certain raw materials and related supplies for use in the manufacturing of drug product in our clinical development programs, as we have determined that these materials have alternative future use. We can use these raw materials and related supplies in multiple clinical drug products, and therefore have future use independent of the development status of any particular drug program until it is utilized in the manufacturing process. We periodically review these capitalized materials for indicators of impairment, including shelf life, continued alternative future use and obsolescence, and write down the asset to its net realizable value in the period it which it is identified. |
Recent Accounting Pronouncements | Recent Accounting Pronouncements In May 2014, the FASB issued ASU No. 2014-09, Revenue from Contracts with Customers (Topic 606) , which outlines a single comprehensive model for entities to use in accounting for revenue arising from contracts with customers and supersedes most current revenue recognition guidance, including industry-specific guidance. ASU 2014-09 outlines a five-step process for revenue recognition that focuses on transfer of control, as opposed to transfer of risk and rewards, and also requires enhanced disclosures regarding the nature, amount, timing and uncertainty of revenues and cash flows from contracts with customers. Major provisions include determining which goods and services are distinct and require separate accounting (performance obligations), how variable consideration (which may include change orders and claims) is recognized, whether revenue should be recognized at a point in time or over time and ensuring the time value of money is considered in the transaction price. The FASB issued supplemental adoption guidance and clarification to ASU No. 2014-09 in March 2016, April 2016 and May 2016 within ASU No. 2016-08, Revenue from Contracts with Customers: Principal vs. Agent Considerations , ASU No. 2016-10, Revenue from Contracts with Customers: Identifying Performance Obligations and Licensing and ASU No. 2016-12, Revenue from Contracts with Customers: Narrow-Scope Improvements and Practical Expedients , respectively. ASU No. 2014-09 and the related supplemental ASUs are effective for fiscal years beginning after December 15, 2017 and interim periods therein. The ASU permits two methods of adoption: the full retrospective method or the modified retrospective method. We plan to apply the modified retrospective method upon adoption in the first quarter of 2018 and currently do not anticipate that the adoption of this ASU will have a material impact with regard to our current contracts. In January 2016, the FASB issued ASU No. 2016-01, Recognition and Measurement of Financial Assets and Financial Liabilities , which eliminates the requirement for public companies to disclose the method(s) and significant assumptions used to estimate the fair value for financial instruments measured at amortized cost on the balance sheet. Additionally, the standard requires public companies to use the exit price notion when measuring the fair value of financial instruments for disclosure purposes. Furthermore, the standard requires presentation of financial assets and liabilities by measurement category and form of financial asset on the balance sheet or accompanying notes to the financial statements. The standard is effective for annual reporting periods beginning after December 15, 2017, including interim periods within those annual reporting periods. Early application is permitted. The adoption of this guidance will have no impact on our financial statements. In February 2016, the FASB issued ASU No. 2016-02, Leases, which increases transparency and comparability among organizations by requiring recognition of lease assets and lease liabilities on the balance sheet and disclosure of key information about leasing arrangements. The standard is effective for annual reporting periods beginning after December 15, 2018, including interim periods within those annual reporting periods. Early application is permitted. We are currently evaluating the impact of adoption on our financial statements. In March 2016, the FASB issued ASU No. 2016-09, Compensation – Stock Compensation: Improvements to Employee Share-Based Payment Accounting, which was intended to simplify various aspects of accounting for share-based payment transactions. The new guidance requires immediate recognition of all excess tax benefits and deficiencies in the income statement, requires classification of excess tax benefits as an operating activity as opposed to a financing activity in the statements of cash flows and allows the Company to make an accounting policy election to either estimate the number of awards expected to vest or account for forfeitures when they occur. The standard is effective for annual reporting periods beginning after December 15, 2016, and interim periods within those annual reporting periods. We applied this standard in the first quarter of 2017 using the modified retrospective method of adoption. In conjunction with this adoption, we applied an accounting policy election to account for forfeitures as they occur. Upon adoption, we reversed a deferred tax asset related to the balance of unrecognized excess tax benefits of $7.4 million , with an offsetting adjustment to the valuation allowance. Under the modified retrospective method of adoption, we recorded an adjustment of $0.6 million to accumulated deficit with a corresponding offset to additional paid-in capital. In August 2016, the FASB issued ASU No. 2016-15, Statement of Cash Flows: Classification of Certain Cash Receipts and Cash Payments , which addresses the presentation and classification of certain cash receipts and cash payments in the statement of cash flows under Accounting Standards Codification 230. The standard is effective for annual reporting periods beginning after December 15, 2017, and interim periods within those annual reporting periods. The adoption of this guidance will have no impact on our financial statements. In November 2016, the FASB issued ASU No. 2016-18, Statement of Cash Flows: Restricted Cash , which requires restricted cash and restricted cash equivalents to be included with cash and cash equivalents when reconciling the beginning-of-period and end-of-period total amounts shown on the statement of cash flows. The standard is effective for annual reporting periods beginning after December 15, 2017, and interim periods within those annual reporting periods. Early application is permitted. Upon its adoption in 2018, we will include $1.3 million of restricted cash in our disclosed balance of cash and cash equivalents at the beginning of the period for 2016. We do not expect any additional impact on our financial statements. In May 2017, the FASB issued ASU No. 2017-09, Compensation - Stock Compensation: Scope of Modification Accounting , which provides clarity and guidance around which changes to the terms or conditions of a share-based payment award require an entity to apply modification accounting in Topic 718. The standard is effective for annual reporting periods beginning after December 15, 2017, and interim periods within those annual reporting periods. The adoption of this guidance will have no impact on our financial statements unless we have modification accounting in accordance with Topic 718. |
Fair Value Measurement | Fair Value Measurements We have certain financial assets recorded at fair value which have been classified as Level 1, 2, or 3 within the fair value hierarchy as described in the accounting standards for fair value measurements. Accounting standards define fair value as the exchange price that would be received for an asset or paid to transfer a liability (an exit price) in the principal or most advantageous market for the asset or liability in an orderly transaction between market participants as of the measurement date. Market participants are buyers and sellers in the principal market that are (i) independent, (ii) knowledgeable, (iii) able to transact, and (iv) willing to transact. The accounting standards provide an established hierarchy for inputs used in measuring fair value that maximizes the use of observable inputs and minimizes the use of unobservable inputs by requiring that the most observable inputs be used when available. Observable inputs are inputs that market participants would use in valuing the asset or liability and are developed based on market data obtained from independent sources. Unobservable inputs are inputs that reflect our assumptions about the factors that market participants would use in valuing the asset or liability. The accounting standards prioritize the inputs used in measuring the fair value into the following hierarchy: • Level 1 includes financial instruments for which quoted market prices for identical instruments are available in active markets. • Level 2 includes financial instruments for which there are inputs other than quoted prices included within Level 1 that are observable for the instrument such as quoted prices for similar instruments in active markets, quoted prices for identical or similar instruments in markets with insufficient volume or infrequent transactions (less active markets) or model-driven valuations in which significant inputs are observable or can be derived principally from, or corroborated by, observable market data. • Level 3 includes financial instruments for which fair value is derived from valuation techniques in which one or more significant inputs are unobservable, including management’s own assumptions. |
Investments (Tables)
Investments (Tables) | 6 Months Ended |
Jun. 30, 2017 | |
Investments, Debt and Equity Securities [Abstract] | |
Summary of Short-Term Investments | The following tables summarize our short-term investments (in thousands): Maturity (in years) Amortized cost Unrealized Estimated fair value Gains Losses As of June 30, 2017 Corporate debt securities 1 or less $ 21,264 $ — $ (9 ) $ 21,255 Certificates of deposit 1 or less 3,785 — — 3,785 U.S. Treasury securities 1 or less 2,010 — (1 ) 2,009 Debt securities of U.S. government-sponsored agencies 1 or less 500 — — 500 Total $ 27,559 $ — $ (10 ) $ 27,549 Maturity (in years) Amortized cost Unrealized Estimated fair value Gains Losses As of December 31, 2016 Corporate debt securities 2 or less $ 49,185 $ 12 $ (77 ) $ 49,120 Certificates of deposit 1 or less 9,291 — — 9,291 Commercial paper 1 or less 1,247 — — 1,247 U.S. Treasury securities 1 or less 1,001 — (1 ) 1,000 Debt securities of U.S. government-sponsored agencies 1 or less 512 — — 512 Total $ 61,236 $ 12 $ (78 ) $ 61,170 |
Fair Value Measurements (Tables
Fair Value Measurements (Tables) | 6 Months Ended |
Jun. 30, 2017 | |
Fair Value Disclosures [Abstract] | |
Fair Value Hierarchy for Assets and Liabilities Measured at Fair Value on Recurring Basis | The following table presents our fair value hierarchy for assets measured at fair value on a recurring basis as of June 30, 2017 and December 31, 2016 (in thousands): Fair value as of June 30, 2017 Total Level 1 Level 2 Level 3 Assets: Cash equivalents $ 10,630 $ 10,630 $ — $ — Corporate debt securities 21,255 — 21,255 — Certificates of deposit 3,785 — 3,785 — U.S. treasury securities 2,009 — 2,009 — Debt securities of U.S. government-sponsored agencies 500 — 500 — $ 38,179 $ 10,630 $ 27,549 $ — Fair value as of December 31, 2016 Total Level 1 Level 2 Level 3 Assets: Cash equivalents $ 13,578 $ 13,578 $ — $ — Corporate debt securities 49,120 — 49,210 — Certificates of deposit 9,291 — 9,291 — Commercial paper 1,247 — 1,247 — U.S. treasury securities 1,000 — 1,000 — Debt securities of U.S. government-sponsored agencies 512 — 512 — $ 74,748 $ 13,578 $ 61,260 $ — |
Term Loan (Tables)
Term Loan (Tables) | 6 Months Ended |
Jun. 30, 2017 | |
Debt Disclosure [Abstract] | |
Schedule of Future Principal Payments | Future principal payments for the Term Loan due under the Loan Agreement are as follows (in thousands): 2017 $ — 2018 5,000 2019 10,000 2020 5,000 $ 20,000 |
Stockholders' Equity (Tables)
Stockholders' Equity (Tables) | 6 Months Ended |
Jun. 30, 2017 | |
Disclosure of Compensation Related Costs, Share-based Payments [Abstract] | |
Common Stock Reserved for Future Issuance | The following shares of common stock were reserved for future issuance as of June 30, 2017 : Common stock options outstanding 10,819,036 Common stock available for future grant under 2012 Equity Incentive Plan 917,027 Common stock available for future grant under 2015 Inducement Plan 23,126 Employee Stock Purchase Plan 1,844,527 Total common shares reserved for future issuance 13,603,716 |
Stock Option Activity | The following table summarizes our stock option activity under all equity incentive plans for the six months ended June 30, 2017 (shares in thousands): Number of options Weighted average exercise price Options outstanding at December 31, 2016 8,931 $ 6.70 Granted 4,255 $ 1.44 Exercised (8 ) $ 0.38 Canceled/forfeited/expired (2,359 ) $ 5.86 Options outstanding at June 30, 2017 10,819 $ 4.82 |
Assumptions Used to Estimate Fair Value of Stock Options and Performance Stock and Employee Stock Purchase Plan | The following table summarizes the weighted average assumptions used to estimate the fair value of stock options and performance stock awards granted to employees under our 2012 Equity Incentive Plan and 2015 Inducement Plan and the shares purchasable under our Employee Stock Purchase Plan during the periods presented: Three months ended Six months ended 2017 2016 2017 2016 Stock options Risk-free interest rate 1.9 % 1.5 % 2.0 % 1.4 % Volatility 89.5 % 79.1 % 89.4 % 79.7 % Dividend yield — — — — Expected term (years) 6.1 5.8 6.1 5.9 Performance stock options Risk-free interest rate — 0.4 % 2.1 % 1.4 % Volatility — 78.5 % 89.9 % 79.3 % Dividend yield — — — — Expected term (years) 0 5.5 5.6 6.0 Employee stock purchase plan shares Risk-free interest rate 0.9 % 0.5 % 0.8 % 0.4 % Volatility 111.5 % 82.6 % 113.8 % 81.0 % Dividend yield — — — — Expected term (years) 0.5 0.5 0.5 0.5 |
Stock-Based Compensation | The following table summarizes the allocation of our stock-based compensation expense for all stock awards during the periods presented, including the adjustments to stock-based compensation expense associated with our May 2017 corporate restructuring (in thousands): Three months ended Six months ended 2017 2016 2017 2016 Research and development $ 650 $ 1,299 $ 1,758 $ 2,731 Research and development-restructuring related adjustments (1,399 ) — (1,399 ) — General and administrative 935 939 2,209 3,255 General and administrative-restructuring related adjustments 2,679 — 2,679 — Total $ 2,865 $ 2,238 $ 5,247 $ 5,986 In connection with our May 2017 corporate restructuring, we recorded a reversal of stock-based compensation in research and development expenses of $1.4 million for each of the three and six months ended June 30, 2017, as a result of the cancellation of unvested stock options. We recorded additional stock-based compensation in general and administrative expenses of $2.7 million for each of the three and six months ended June 30, 2017, as a result of termination provisions within certain employment agreements. |
Strategic Alliances and Colla20
Strategic Alliances and Collaborations (Tables) | 6 Months Ended |
Jun. 30, 2017 | |
Organization, Consolidation and Presentation of Financial Statements [Abstract] | |
Revenue from Strategic Alliances | The following table summarizes our total revenues from our strategic alliances and collaborations during the periods presented (in thousands): Three months ended Six months ended 2017 2016 2017 2016 Sanofi $ 18 $ 18 $ 36 $ 36 AstraZeneca — 465 — 936 Total $ 18 $ 483 $ 36 $ 972 |
Basis of Presentation and Sum21
Basis of Presentation and Summary of Significant Accounting Policies (Detail) $ in Thousands | 1 Months Ended | 3 Months Ended | 6 Months Ended | ||||
Jun. 30, 2017USD ($)employee | Jun. 30, 2017USD ($)employee | Jun. 30, 2016USD ($) | Jun. 30, 2017USD ($)employee | Jun. 30, 2016USD ($) | May 31, 2017 | Dec. 31, 2016USD ($) | |
Property, Plant and Equipment [Line Items] | |||||||
Workforce reduction, commitment rate | 30.00% | ||||||
Number of employees | employee | 65 | 65 | 65 | ||||
Severance costs | $ 3,200 | ||||||
Non-cash stock-based compensation | 1,300 | ||||||
Cash and cash equivalents and marketable securities | 40,100 | $ 40,100 | $ 40,100 | ||||
Prepaid materials | 6,149 | 6,149 | 6,149 | $ 5,552 | |||
Prepaid expenses reserve | 600 | 600 | 600 | 600 | |||
Stock-based compensation expenses | 2,865 | $ 2,238 | 5,247 | $ 5,986 | |||
Restricted cash | 1,300 | 1,300 | 1,300 | ||||
Prepaid Materials | |||||||
Property, Plant and Equipment [Line Items] | |||||||
Prepaid materials | $ 6,100 | $ 6,100 | $ 6,100 | $ 5,600 |
Basis of Presentation and Sum22
Basis of Presentation and Summary of Significant Accounting Policies (Recent Accounting Pronouncements) (Details) - USD ($) $ in Thousands | 3 Months Ended | 6 Months Ended | ||||
Jun. 30, 2017 | Mar. 31, 2017 | Jun. 30, 2016 | Mar. 31, 2016 | Jun. 30, 2017 | Jun. 30, 2016 | |
New Accounting Pronouncements or Change in Accounting Principle [Line Items] | ||||||
Excess tax benefit amount | $ 7,400 | |||||
Stock-based compensation expenses | $ 2,865 | $ 2,238 | $ 5,247 | $ 5,986 | ||
Accounting Standards Update 2016-09 [Member] | ||||||
New Accounting Pronouncements or Change in Accounting Principle [Line Items] | ||||||
Stock-based compensation expenses | $ 600 | |||||
Accounting Standards Update 2016-09 [Member] | Additional Paid-in Capital [Member] | ||||||
New Accounting Pronouncements or Change in Accounting Principle [Line Items] | ||||||
Cumulative effect of new accounting principle in period of adoption | $ 600 |
Net Loss Per Share (Detail)
Net Loss Per Share (Detail) - shares | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2017 | Jun. 30, 2016 | Jun. 30, 2017 | Jun. 30, 2016 | |
Earnings Per Share [Abstract] | ||||
Potentially dilutive securities not included in calculation of diluted net loss per share (in shares) | 2,140,111 | 3,055,143 | 2,869,643 | 2,639,630 |
Investments (Summary of Short-T
Investments (Summary of Short-Term Investments) (Detail) - USD ($) $ in Thousands | 6 Months Ended | 12 Months Ended |
Jun. 30, 2017 | Dec. 31, 2016 | |
Investment [Line Items] | ||
Short term investment maturity weighted average (less than) | 1 year | |
Amortized cost | $ 27,559 | $ 61,236 |
Unrealized Gains | 0 | 12 |
Unrealized Losses | (10) | (78) |
Estimated fair value | $ 27,549 | $ 61,170 |
Corporate debt securities | ||
Investment [Line Items] | ||
Maturity (or less) (in years) | 1 year | 2 years |
Amortized cost | $ 21,264 | $ 49,185 |
Unrealized Gains | 0 | 12 |
Unrealized Losses | (9) | (77) |
Estimated fair value | $ 21,255 | $ 49,120 |
Certificates of deposit | ||
Investment [Line Items] | ||
Maturity (or less) (in years) | 1 year | 1 year |
Amortized cost | $ 3,785 | $ 9,291 |
Unrealized Gains | 0 | 0 |
Unrealized Losses | 0 | 0 |
Estimated fair value | $ 3,785 | $ 9,291 |
Commercial paper | ||
Investment [Line Items] | ||
Maturity (or less) (in years) | 1 year | |
Amortized cost | $ 1,247 | |
Unrealized Gains | 0 | |
Unrealized Losses | 0 | |
Estimated fair value | $ 1,247 | |
U.S. Treasury securities | ||
Investment [Line Items] | ||
Maturity (or less) (in years) | 1 year | 1 year |
Amortized cost | $ 2,010 | $ 1,001 |
Unrealized Gains | 0 | 0 |
Unrealized Losses | (1) | (1) |
Estimated fair value | $ 2,009 | $ 1,000 |
Debt securities of U.S. government-sponsored agencies | ||
Investment [Line Items] | ||
Maturity (or less) (in years) | 1 year | 1 year |
Amortized cost | $ 500 | $ 512 |
Unrealized Gains | 0 | 0 |
Unrealized Losses | 0 | 0 |
Estimated fair value | $ 500 | $ 512 |
Fair Value Measurements (Fair V
Fair Value Measurements (Fair Value Hierarchy for Assets and Liabilities Measured at Fair Value on Recurring Basis) (Detail) - Fair Value, Measurements, Recurring - USD ($) $ in Thousands | Jun. 30, 2017 | Dec. 31, 2016 |
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items] | ||
Assets measured at fair value on a recurring basis | $ 38,179 | $ 74,748 |
Cash equivalents | ||
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items] | ||
Assets measured at fair value on a recurring basis | 10,630 | 13,578 |
Corporate debt securities | ||
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items] | ||
Assets measured at fair value on a recurring basis | 21,255 | 49,120 |
Certificates of deposit | ||
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items] | ||
Assets measured at fair value on a recurring basis | 3,785 | 9,291 |
Commercial paper | ||
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items] | ||
Assets measured at fair value on a recurring basis | 1,247 | |
U.S. Treasury securities | ||
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items] | ||
Assets measured at fair value on a recurring basis | 2,009 | 1,000 |
Debt securities of U.S. government-sponsored agencies | ||
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items] | ||
Assets measured at fair value on a recurring basis | 500 | 512 |
Level 1 | ||
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items] | ||
Assets measured at fair value on a recurring basis | 10,630 | 13,578 |
Level 1 | Cash equivalents | ||
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items] | ||
Assets measured at fair value on a recurring basis | 10,630 | 13,578 |
Level 1 | Corporate debt securities | ||
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items] | ||
Assets measured at fair value on a recurring basis | 0 | 0 |
Level 1 | Certificates of deposit | ||
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items] | ||
Assets measured at fair value on a recurring basis | 0 | 0 |
Level 1 | Commercial paper | ||
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items] | ||
Assets measured at fair value on a recurring basis | 0 | |
Level 1 | U.S. Treasury securities | ||
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items] | ||
Assets measured at fair value on a recurring basis | 0 | 0 |
Level 1 | Debt securities of U.S. government-sponsored agencies | ||
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items] | ||
Assets measured at fair value on a recurring basis | 0 | 0 |
Level 2 | ||
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items] | ||
Assets measured at fair value on a recurring basis | 27,549 | 61,260 |
Level 2 | Cash equivalents | ||
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items] | ||
Assets measured at fair value on a recurring basis | 0 | 0 |
Level 2 | Corporate debt securities | ||
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items] | ||
Assets measured at fair value on a recurring basis | 21,255 | 49,210 |
Level 2 | Certificates of deposit | ||
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items] | ||
Assets measured at fair value on a recurring basis | 3,785 | 9,291 |
Level 2 | Commercial paper | ||
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items] | ||
Assets measured at fair value on a recurring basis | 1,247 | |
Level 2 | U.S. Treasury securities | ||
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items] | ||
Assets measured at fair value on a recurring basis | 2,009 | 1,000 |
Level 2 | Debt securities of U.S. government-sponsored agencies | ||
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items] | ||
Assets measured at fair value on a recurring basis | 500 | 512 |
Level 3 | ||
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items] | ||
Assets measured at fair value on a recurring basis | 0 | 0 |
Level 3 | Cash equivalents | ||
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items] | ||
Assets measured at fair value on a recurring basis | 0 | 0 |
Level 3 | Corporate debt securities | ||
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items] | ||
Assets measured at fair value on a recurring basis | 0 | 0 |
Level 3 | Certificates of deposit | ||
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items] | ||
Assets measured at fair value on a recurring basis | 0 | 0 |
Level 3 | Commercial paper | ||
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items] | ||
Assets measured at fair value on a recurring basis | 0 | |
Level 3 | U.S. Treasury securities | ||
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items] | ||
Assets measured at fair value on a recurring basis | 0 | 0 |
Level 3 | Debt securities of U.S. government-sponsored agencies | ||
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items] | ||
Assets measured at fair value on a recurring basis | $ 0 | $ 0 |
Term Loan (Details)
Term Loan (Details) | Jun. 22, 2016USD ($) | Jun. 17, 2016USD ($)loan | Jun. 30, 2017USD ($) | Dec. 31, 2016USD ($) |
Debt Instrument [Line Items] | ||||
Term loan, less debt issuance costs | $ 19,830,000 | $ 19,802,000 | ||
Debt Financing Agreement | ||||
Debt Instrument [Line Items] | ||||
Maximum borrowing capacity | $ 30,000,000 | |||
Number of term loans | loan | 2 | |||
Period for principal and interest payments | 24 months | |||
Stated interest rate, percentage | 8.51% | |||
Prepayment fee, option one | 2.00% | |||
Prepayment fee, option two | 1.00% | |||
Debt extinguishment fee | 5.50% | |||
Term loan, less debt issuance costs | $ 20,000,000 | |||
Amounts borrowed | 20,000,000 | |||
Deferred finance cost | $ 200,000 | |||
Debt instrument effective rate | 8.98% | |||
Debt Financing Agreement | London Interbank Offered Rate (LIBOR) | ||||
Debt Instrument [Line Items] | ||||
Basis spread on variable rate, percentage | 0.44% | |||
Debt Financing Agreement Tranche A | ||||
Debt Instrument [Line Items] | ||||
Maximum borrowing capacity | $ 20,000,000 | |||
Proceeds from borrowing under term loan | $ 20,000,000 | |||
Term loan, less debt issuance costs | $ 20,000,000 | |||
Debt Financing Agreement Tranche B | ||||
Debt Instrument [Line Items] | ||||
Maximum borrowing capacity | $ 10,000,000 |
Term Loan (Future Principal Pay
Term Loan (Future Principal Payments) (Details) - USD ($) $ in Thousands | Jun. 30, 2017 | Dec. 31, 2016 |
Debt Instrument [Line Items] | ||
Long-term Debt | $ 19,830 | $ 19,802 |
Debt Financing Agreement Tranche A | ||
Debt Instrument [Line Items] | ||
Future Repayments of Principal, 2017 | 0 | |
Future Repayments of Principal, 2018 | 5,000 | |
Future Repayments of Principal, 2019 | 10,000 | |
Future Repayments of Principal, 2020 | 5,000 | |
Long-term Debt | $ 20,000 |
Stockholders' Equity (Common St
Stockholders' Equity (Common Stock Reserved for Future Issuance) (Detail) - shares | Jun. 30, 2017 | Dec. 31, 2016 |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||
Common stock options outstanding | 10,819,036 | 8,931,000 |
Total common shares reserved for future issuance | 13,603,716 | |
Common stock available for future grant under 2012 Equity Incentive Plan | ||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||
Common stock available for future grant | 917,027 | |
Common stock available for future grant under 2015 Inducement Plan | ||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||
Common stock available for future grant | 23,126 | |
Employee Stock Purchase Plan | ||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||
Common stock available for future grant | 1,844,527 |
Stockholders' Equity (Stock Opt
Stockholders' Equity (Stock Option Activity) (Detail) | 6 Months Ended |
Jun. 30, 2017$ / sharesshares | |
Number of options | |
Number of options, outstanding at December 31, 2016 (in shares) | shares | 8,931,000 |
Number of options, Granted (in shares) | shares | 4,255,000 |
Number of options, Exercised (in shares) | shares | (8,000) |
Number of options, Canceled/forfeited/expired (in shares) | shares | (2,359,000) |
Number of options, outstanding at March 31, 2017 (in shares) | shares | 10,819,036 |
Weighted average exercise price | |
Weighted average exercise price, Options outstanding at December 31, 2016 (in dollars per share) | $ / shares | $ 6.70 |
Weighted average exercise price, Granted (in dollars per share) | $ / shares | 1.44 |
Weighted average exercise price, Exercised (in dollars per share) | $ / shares | 0.38 |
Weighted average exercise price, Canceled/forfeited/expired (in dollars per share) | $ / shares | 5.86 |
Weighted average exercise price, Options outstanding at March 31, 2017 (in dollars per share) | $ / shares | $ 4.82 |
Stockholders' Equity (Assumptio
Stockholders' Equity (Assumptions Used to Estimate Fair Value of Stock Options and Performance Stock and Employee Stock Purchase Plan) (Detail) | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2017 | Jun. 30, 2016 | Jun. 30, 2017 | Jun. 30, 2016 | |
Employee Stock Purchase Plan | ||||
Weighted average assumptions | ||||
Risk-free interest rate | 0.90% | 0.50% | 0.80% | 0.40% |
Volatility | 111.50% | 82.60% | 113.80% | 81.00% |
Dividend yield | 0.00% | 0.00% | 0.00% | 0.00% |
Expected term (years) | 6 months | 6 months | 6 months | 6 months |
Stock options | ||||
Weighted average assumptions | ||||
Risk-free interest rate | 1.90% | 1.50% | 2.00% | 1.40% |
Volatility | 89.50% | 79.10% | 89.40% | 79.70% |
Dividend yield | 0.00% | 0.00% | 0.00% | 0.00% |
Expected term (years) | 6 years 1 month 6 days | 5 years 9 months 18 days | 6 years 1 month 6 days | 5 years 10 months 24 days |
Performance stock options | ||||
Weighted average assumptions | ||||
Risk-free interest rate | 0.00% | 0.40% | 2.10% | 1.40% |
Volatility | 0.00% | 78.50% | 89.90% | 79.30% |
Dividend yield | 0.00% | 0.00% | 0.00% | 0.00% |
Expected term (years) | 0 years | 5 years 6 months | 5 years 7 months 6 days | 6 years |
Stockholders' Equity (Stock-Bas
Stockholders' Equity (Stock-Based Compensation Expense Allocation) (Detail) - USD ($) $ in Thousands | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2017 | Jun. 30, 2016 | Jun. 30, 2017 | Jun. 30, 2016 | |
Employee Service Share-based Compensation, Allocation of Recognized Period Costs [Line Items] | ||||
Stock-based compensation expenses | $ 2,865 | $ 2,238 | $ 5,247 | $ 5,986 |
Research and development | ||||
Employee Service Share-based Compensation, Allocation of Recognized Period Costs [Line Items] | ||||
Stock-based compensation expenses | 650 | 1,299 | 1,758 | 2,731 |
Research and development-restructuring related adjustments | ||||
Employee Service Share-based Compensation, Allocation of Recognized Period Costs [Line Items] | ||||
Stock-based compensation expenses | (1,399) | 0 | (1,399) | 0 |
General and administrative | ||||
Employee Service Share-based Compensation, Allocation of Recognized Period Costs [Line Items] | ||||
Stock-based compensation expenses | 935 | 939 | 2,209 | 3,255 |
General and administrative-restructuring related adjustments | ||||
Employee Service Share-based Compensation, Allocation of Recognized Period Costs [Line Items] | ||||
Stock-based compensation expenses | $ 2,679 | $ 0 | $ 2,679 | $ 0 |
Strategic Alliances and Colla32
Strategic Alliances and Collaborations (Revenue from Strategic Alliances) (Detail) - USD ($) $ in Thousands | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2017 | Jun. 30, 2016 | Jun. 30, 2017 | Jun. 30, 2016 | |
Revenue from External Customer [Line Items] | ||||
Total revenues | $ 18 | $ 483 | $ 36 | $ 972 |
Strategic Alliances and Collaborations | ||||
Revenue from External Customer [Line Items] | ||||
Total revenues | 18 | 483 | 36 | 972 |
Strategic Alliances and Collaborations | Sanofi | ||||
Revenue from External Customer [Line Items] | ||||
Total revenues | 18 | 18 | 36 | 36 |
Strategic Alliances and Collaborations | AstraZeneca | ||||
Revenue from External Customer [Line Items] | ||||
Total revenues | $ 0 | $ 465 | $ 0 | $ 936 |
Strategic Alliances and Colla33
Strategic Alliances and Collaborations (Detail) | 1 Months Ended | 6 Months Ended | 8 Months Ended | |||||||
Dec. 31, 2015USD ($) | Mar. 31, 2015USD ($) | Feb. 28, 2014USD ($)$ / sharesshares | Jul. 31, 2013USD ($) | Oct. 31, 2012USD ($)$ / sharesshares | Aug. 31, 2012target | Jul. 31, 2012target | Jun. 30, 2017USD ($)shares | Jan. 31, 2014USD ($) | Dec. 31, 2016USD ($)shares | |
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | ||||||||||
Common stock, shares issued | shares | 53,182,330 | 52,924,805 | ||||||||
Common stock value | $ 53,000 | $ 53,000 | ||||||||
Sanofi | ||||||||||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | ||||||||||
Number of collaborative areas granted | target | 4 | |||||||||
Deferred revenue | $ 400,000 | |||||||||
Sanofi | Private Placement | ||||||||||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | ||||||||||
Common stock, shares issued | shares | 1,303,780 | |||||||||
Price per share (in dollars per share) | $ / shares | $ 7.67 | |||||||||
Common stock value | $ 10,000,000 | |||||||||
Restriction period in which Alliances could not sell, transfer, make any short sale of, or grant any option for the sale of any common stock | 12 months | |||||||||
Sanofi | Minimum | United States | ||||||||||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | ||||||||||
Royalties based on percentage of net sales | 10.00% | |||||||||
Sanofi | Minimum | Outside of the United States | ||||||||||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | ||||||||||
Royalties based on percentage of net sales | 10.00% | |||||||||
Sanofi | Maximum | United States | ||||||||||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | ||||||||||
Royalties based on percentage of net sales | 20.00% | |||||||||
Sanofi | Maximum | Outside of the United States | ||||||||||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | ||||||||||
Royalties based on percentage of net sales | 20.00% | |||||||||
Sanofi | Development Commercialization And License Agreement | ||||||||||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | ||||||||||
Initial upfront option payment | $ 2,500,000 | |||||||||
Deferred revenue creditable against future milestones | $ 1,250,000 | $ 1,250,000 | $ 1,250,000 | |||||||
Upfront payment non-creditable portion recognized | $ 1,250,000 | |||||||||
Sanofi | Common Stock Purchase Agreement | ||||||||||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | ||||||||||
Deferred revenue | $ 200,000 | |||||||||
Deferred revenue recognition period | 3 years | |||||||||
Sanofi | Proof-of-Concept Trial | ||||||||||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | ||||||||||
Potential revenue through milestone payments | $ 101,800,000 | |||||||||
Sanofi | Regulatory and Commercialization Milestones | ||||||||||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | ||||||||||
Potential revenue through milestone payments | 300,000,000 | |||||||||
Sanofi | Clinical | ||||||||||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | ||||||||||
Potential revenue through milestone payments | $ 15,000,000 | |||||||||
AstraZeneca | ||||||||||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | ||||||||||
Number of collaborative areas granted | target | 3 | |||||||||
Initial upfront option payment | $ 3,000,000 | |||||||||
Common stock, shares issued | shares | 6,250,000 | |||||||||
Price per share (in dollars per share) | $ / shares | $ 4 | |||||||||
Restriction period in which Alliances could not sell, transfer, make any short sale of, or grant any option for the sale of any common stock | 365 days | |||||||||
Deferred revenue | $ 4,300,000 | |||||||||
Expected term of research and development plan (years) | 4 years | |||||||||
AstraZeneca | Preclinical | ||||||||||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | ||||||||||
Milestone payments, earned | $ 2,500,000 | |||||||||
AstraZeneca | Clinical | ||||||||||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | ||||||||||
Milestone payments, earned | $ 10,000,000 |
Related Party Transactions (Det
Related Party Transactions (Detail) - USD ($) | 1 Months Ended | 3 Months Ended | 6 Months Ended | ||
Feb. 28, 2015 | Jun. 30, 2017 | Jun. 30, 2016 | Jun. 30, 2017 | Jun. 30, 2016 | |
Sanofi | |||||
Related Party Transaction [Line Items] | |||||
Expenses incurred for services performed or out-of-pocket expenses under the Sanofi agreement (less than) | $ 100,000 | $ 100,000 | $ 100,000 | $ 800,000 | |
Alnylam | |||||
Related Party Transaction [Line Items] | |||||
Potential milestone payments to related party | $ 33,000,000 |
Subsequent Events (Details)
Subsequent Events (Details) - Public Offering - Subsequent Event $ / shares in Units, $ in Millions | 1 Months Ended |
Jul. 31, 2017USD ($)$ / sharesshares | |
Subsequent Event [Line Items] | |
Number of shares issued (in shares) | shares | 50,600,000 |
Sale price per share (USD per share) | $ / shares | $ 0.91 |
Consideration received | $ | $ 43 |