Document and Entity Information
Document and Entity Information - shares | 6 Months Ended | |
Jun. 30, 2019 | Jul. 26, 2019 | |
Document And Entity Information [Abstract] | ||
Document Type | 10-Q | |
Amendment Flag | false | |
Document Period End Date | Jun. 30, 2019 | |
Document Fiscal Year Focus | 2019 | |
Document Fiscal Period Focus | Q2 | |
Trading Symbol | NTLA | |
Entity Registrant Name | INTELLIA THERAPEUTICS, INC. | |
Entity Central Index Key | 0001652130 | |
Current Fiscal Year End Date | --12-31 | |
Entity Filer Category | Large Accelerated Filer | |
Entity Small Business | false | |
Entity Emerging Growth Company | false | |
Entity Common Stock, Shares Outstanding | 48,543,069 | |
Entity Current Reporting Status | Yes | |
Entity Shell Company | false | |
Entity File Number | 001-37766 | |
Entity Tax Identification Number | 364785571 | |
Entity Address, Address Line One | 40 Erie Street | |
Entity Address, Address Line Two | Suite 130 | |
Entity Address, City or Town | Cambridge | |
Entity Address, State or Province | Massachusetts | |
Entity Address, Postal Zip Code | 02139 | |
City Area Code | 857 | |
Local Phone Number | 285-6200 |
Condensed Consolidated Balance
Condensed Consolidated Balance Sheets (Unaudited) - USD ($) $ in Thousands | Jun. 30, 2019 | Dec. 31, 2018 |
Current Assets: | ||
Cash and cash equivalents | $ 49,140 | $ 58,856 |
Marketable securities - current | 221,959 | 255,203 |
Accounts receivable | 4,188 | 7,547 |
Proceeds due from at-the-market offerings | 27,140 | |
Prepaid expenses and other current assets | 4,710 | 3,371 |
Total current assets | 307,137 | 324,977 |
Marketable securities - noncurrent | 4,739 | |
Property and equipment, net | 16,694 | 17,061 |
Operating lease right-of-use assets | 20,923 | |
Other assets | 2,948 | 5,277 |
Total Assets | 352,441 | 347,315 |
Current Liabilities: | ||
Accounts payable | 1,848 | 2,708 |
Accrued expenses | 11,898 | 10,742 |
Current portion of lease liability | 5,065 | |
Current portion of deferred revenue | 19,652 | 27,122 |
Total current liabilities | 38,463 | 40,572 |
Deferred revenue, net of current portion | 22,508 | 28,810 |
Long-term lease liability | 14,604 | |
Other long-term liabilities | 13 | |
Commitments and contingencies | ||
Stockholders’ Equity: | ||
Common stock, $0.0001 par value; 120,000,000 shares authorized; 47,714,575 and 45,224,480 shares issued and outstanding at June 30, 2019 and December 31, 2018, respectively | 5 | 5 |
Additional paid-in capital | 525,574 | 478,968 |
Accumulated other comprehensive income (loss) | 255 | (28) |
Accumulated deficit | (248,968) | (201,025) |
Total stockholders’ equity | 276,866 | 277,920 |
Total Liabilities and Stockholders’ Equity | $ 352,441 | $ 347,315 |
Condensed Consolidated Balanc_2
Condensed Consolidated Balance Sheets (Unaudited) (Parenthetical) - $ / shares | Jun. 30, 2019 | Dec. 31, 2018 |
Statement Of Financial Position [Abstract] | ||
Common stock, par value | $ 0.0001 | $ 0.0001 |
Common stock, shares authorized | 120,000,000 | 120,000,000 |
Common stock, shares issued | 47,714,575 | 45,224,480 |
Common stock, shares outstanding | 47,714,575 | 45,224,480 |
Condensed Consolidated Statemen
Condensed Consolidated Statements of Operations and Comprehensive Loss (Unaudited) - USD ($) shares in Thousands, $ in Thousands | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2019 | Jun. 30, 2018 | Jun. 30, 2019 | Jun. 30, 2018 | |
Income Statement [Abstract] | ||||
Collaboration revenue | $ 11,118 | $ 7,677 | $ 21,551 | $ 15,146 |
Type of Revenue [Extensible List] | us-gaap:LicenseAndServiceMember | us-gaap:LicenseAndServiceMember | us-gaap:LicenseAndServiceMember | us-gaap:LicenseAndServiceMember |
Operating expenses: | ||||
Research and development | $ 25,460 | $ 23,467 | $ 49,169 | $ 45,960 |
General and administrative | 13,118 | 7,805 | 23,651 | 15,211 |
Total operating expenses | 38,578 | 31,272 | 72,820 | 61,171 |
Operating loss | (27,460) | (23,595) | (51,269) | (46,025) |
Interest income | 1,777 | 1,376 | 3,646 | 2,450 |
Net loss | $ (25,683) | $ (22,219) | $ (47,623) | $ (43,575) |
Net loss per share, basic and diluted | $ (0.56) | $ (0.52) | $ (1.05) | $ (1.03) |
Weighted average shares outstanding, basic and diluted | 45,814 | 42,836 | 45,526 | 42,441 |
Other comprehensive income: | ||||
Unrealized gain on marketable securities | $ 196 | $ 283 | ||
Comprehensive loss | $ (25,487) | $ (22,219) | $ (47,340) | $ (43,575) |
Condensed Consolidated Statem_2
Condensed Consolidated Statements of Cash Flows (Unaudited) - USD ($) $ in Thousands | 6 Months Ended | 62 Months Ended | |
Jun. 30, 2019 | Jun. 30, 2018 | Jun. 30, 2019 | |
CASH FLOWS FROM OPERATING ACTIVITIES: | |||
Net loss | $ (47,623) | $ (43,575) | |
Adjustments to reconcile net loss to net cash used in operating activities: | |||
Depreciation and amortization | 2,621 | 2,085 | |
Loss (gain) on disposal of property and equipment | 17 | (29) | |
Equity-based compensation | 8,996 | 9,008 | |
Accretion of investment discounts | (2,630) | ||
Non-cash lease expense | 462 | ||
Changes in operating assets and liabilities: | |||
Accounts receivable | 3,359 | 1,862 | |
Prepaid expenses and other current assets | (1,339) | 199 | |
Accounts payable | (293) | (1,599) | |
Accrued expenses | 947 | 305 | |
Deferred revenue | (13,772) | (9,009) | |
Other assets | 125 | 564 | |
Other long-term liabilities | (76) | ||
Net cash used in operating activities | (49,130) | (40,265) | |
CASH FLOWS FROM INVESTING ACTIVITIES: | |||
Purchases of property and equipment | (2,474) | (2,750) | |
Purchases of marketable securities | (182,582) | ||
Maturities of marketable securities | 214,000 | ||
Net cash provided by (used in) investing activities | 28,944 | (2,750) | |
CASH FLOWS FROM FINANCING ACTIVITIES: | |||
Proceeds from common stock offerings, net of offering costs | 7,912 | $ 580,200 | |
Proceeds from options exercised | 2,024 | 7,277 | |
Issuance of shares through employee stock purchase plan | 534 | 598 | |
Net cash provided by financing activities | 10,470 | 7,875 | |
Net decrease in cash and cash equivalents | (9,716) | (35,140) | |
Cash and cash equivalents, beginning of period | 58,856 | 340,678 | |
Cash and cash equivalents, end of period | 49,140 | 305,538 | $ 49,140 |
SUPPLEMENTAL DISCLOSURES OF CASH FLOW INFORMATION: | |||
Purchases of property and equipment unpaid at period end | 867 | $ 1,417 | |
Proceeds from at-the-market offerings unpaid at period end | 27,140 | ||
Right-of-use assets acquired under operating leases | $ 1,343 |
Overview and Basis of Presentat
Overview and Basis of Presentation | 6 Months Ended |
Jun. 30, 2019 | |
Organization Consolidation And Presentation Of Financial Statements [Abstract] | |
Overview and Basis of Presentation | 1. Overview and Basis of Presentation Intellia Therapeutics, Inc. (“Intellia” or the “Company”) is a genome editing company focused on developing curative therapeutics utilizing a biological tool known as CRISPR/Cas9, which stands for C lustered, R egularly I nterspaced S hort P alindromic R epeats (“CRISPR”)/CRISPR associated 9 (“Cas9”) is a technology for genome editing, the process of altering selected sequences of genomic deoxyribonucleic acid (“DNA”). transform medicine by editing disease-associated genes with a single treatment course, and that it can also be used to create novel engineered cell therapies that can replace a patient’s diseased cells or effectively target various cancers and autoimmune diseases. The Company is leveraging its leading scientific expertise, clinical development experience and intellectual property (“IP”) position to unlock a broad set of therapeutic applications for CRISPR/Cas9 genome editing and to develop a potential new class of therapeutic products. The condensed consolidated financial statements of the Company included herein have been prepared, without audit, pursuant to the rules and regulations of the Securities and Exchange Commission (“SEC”). Certain information and footnote disclosures normally included in financial statements prepared in accordance with accounting principles generally accepted in the United States of America (“U.S. GAAP”) have been condensed or omitted from this report, as is permitted by such rules and regulations. Accordingly, these condensed consolidated financial statements should be read in conjunction with the financial statements and notes thereto included in the Company’s Annual Report on Form 10-K (“Annual Report”) for the year ended December 31, 2018. The unaudited condensed consolidated financial statements include the accounts of Intellia Therapeutics, Inc. and its wholly owned, controlled subsidiary, Intellia Securities Corp. All intercompany balances and transactions have been eliminated in consolidation. Comprehensive loss is comprised of net loss and gain/loss on marketable securities. In the opinion of management, the information furnished reflects all adjustments, all of which are of a normal and recurring nature, necessary for a fair presentation of the results for the reported interim periods. The Company considers events or transactions that occur after the balance sheet date but before the financial statements are issued to provide additional evidence relative to certain estimates or to identify matters that require additional disclosure. The results of operations for interim periods are not necessarily indicative of results to be expected for the full year or any other interim period. Liquidity Since its inception through June 30, 2019, the Company has raised an aggregate of $580.2 million to fund its operations, of which $146.7 million was through its collaboration agreements, $170.5 million was from its initial public offering and concurrent private placements, $141.0 million was from a follow-on public offering, $85.0 million was from the sale of convertible preferred stock and $37.0 On October 12, 2018, the Company filed a Registration Statement on Form S-3 (the “Shelf”) with the SEC in relation to the registration of common stock, preferred stock, warrants and/or units of any combination thereof (collectively, the “Securities”). The Company also simultaneously entered into an Open Market Sale Agreement (the “Sales Agreement”) with Jefferies LLC, (the “Sales Agent”), to provide for the offering, issuance and sale by the Company of up to an aggregate amount of $100.0 million of its common stock from time to time in “at-the-market” offerings under the Shelf and subject to the limitations thereof. The Company will pay to the Sales Agent cash commissions of 3.0 percent of the gross proceeds of sales of common stock under the Sales Agreement. I 2,210,397 16.41 35.2 0.1 recorded as a current asset offerings with trade dates in June 2019 that were settled by July 2, 2019. |
Summary of Significant Accounti
Summary of Significant Accounting Policies | 6 Months Ended |
Jun. 30, 2019 | |
Accounting Policies [Abstract] | |
Summary of Significant Accounting Policies | 2. Summary of Significant Accounting Policies The Company’s significant accounting policies are described in Note 2, “Summary of Significant Accounting Policies”, in our Annual Report. There have been no material changes during the six months ended June 30, 2019, other than the Company’s adoption of Accounting Standards Codification (“ASC”) 842 (as defined below) which is discussed in detail in this note Marketable Securities The following table summarizes the Company’s available-for-sale marketable securities as of June 30, 2019 and December 31, 2018 at net book value: June 30, 2019 Amortized Cost Gross Unrealized Gains Gross Unrealized Losses Estimated Fair Value (In thousands) Marketable securities: U.S. Treasury securities $ 146,552 $ 161 $ - $ 146,713 Financial institution debt securities 53,041 69 - 53,110 Corporate debt securities 18,892 - - 18,892 Other asset-backed securities 7,958 25 - 7,983 Total $ 226,443 $ 255 $ - $ 226,698 December 31, 2018 Amortized Cost Gross Unrealized Gains Gross Unrealized Losses Estimated Fair Value (In thousands) Marketable securities: U.S. Treasury securities $ 165,959 $ 2 $ (13 ) $ 165,948 Financial institution debt securities 65,436 1 (17 ) 65,420 Corporate debt securities 23,836 - (1 ) 23,835 Total $ 255,231 $ 3 $ (31 ) $ 255,203 The amortized cost of available-for-sale securities is adjusted for amortization of premiums and accretion of discounts to maturity. At June 30, 2019 and December 31, 2018, the balance in the Company’s accumulated other comprehensive income (loss) was composed of activity related to the Company’s available-for-sale marketable securities. There were no realized gains or losses in the period ended June 30, 2019, and as a result, the Company did not reclassify any amounts out of accumulated other comprehensive income (loss) during the period. The Company did not have any securities in an unrealized loss position at June 30, 2019. Leases Effective January 1, 2019, the Company adopted ASC Topic 842, Leases Leases At the inception of an arrangement, the Company determines whether an arrangement is or contains a lease based on the unique facts and circumstances present in the arrangement. Most leases with a term greater than one year are recognized on the balance sheet as right-of-use assets and short-term and long-term lease liabilities, as applicable. The Company has elected not to recognize leases with terms of 12 months or less on the balance sheet. The Company typically only includes an initial lease term in its assessment of a lease arrangement. Options to renew a lease are not included in the Company’s assessment unless there is reasonable certainty that the Company will renew. The Company monitors its plans to renew its material leases on a quarterly basis. Operating lease liabilities and their corresponding right-of-use assets are recorded based on the present value of lease payments over the expected remaining lease term. Certain adjustments to the right-of-use asset may be required for items such as incentives received. The interest rate implicit in the Company’s leases is typically not readily determinable. As a result, the Company utilizes its incremental borrowing rate to discount lease payments , which reflects the fixed rate at which the Company could borrow on a collateralized basis the amount of the lease payments in the same currency, for a similar term, in a similar economic environment. In its transition to ASC 842, the Company utilized the remaining lease term of its leases in determining the appropriate incremental borrowing rates. In accordance with ASC 842, components of a lease should be allocated between lease components (e.g., land, building, etc.) and non-lease components (e.g., common area maintenance, consumables, etc.). The fixed and in-substance fixed contract consideration must be allocated based on the respective relative fair values to the lease components and non-lease components. Although separation of lease and non-lease components is otherwise required, an expedient is available whereby the entity may account for each lease component and related non-lease component together as a single lease component. For new and amended leases beginning in 2019 and after, the Company has elected to account for the lease and non-lease components together as a single lease component for all underlying assets and allocate all of the contract consideration to the lease component only. Revenue Recognition The Company adopted Accounting Standards Update (“ Revenue from Contracts with Customers (Topic 606) At inception, the Company determines whether contracts are within the scope of ASC 606 or other topics. For contracts that are determined to be within the scope of ASC 606, revenue is recognized when a customer obtains control of promised goods or services. The amount of revenue recognized reflects the consideration to which the Company expects to be entitled to receive in exchange for these goods and services. To achieve this core principle, the Company applies the following five steps (i) identify the contract with the customer; (ii) identify the performance obligations in the contract; (iii) determine the transaction price; (iv) allocate the transaction price to the performance obligations in the contract; and (v) recognize revenue when or as the Company satisfies a performance obligation. The Company only applies the five-step model to contracts when the Company determines that collection of substantially all consideration for goods and services that are transferred is probable based on the customer’s intent and ability to pay the promised consideration. Performance obligations promised in a contract are identified based on the goods and services that will be transferred to the customer that are both capable of being distinct and are distinct in the context of the contract. To the extent a contract includes multiple promised goods and services, the Company applies judgment to determine whether promised goods and services are both capable of being distinct and distinct in the context of the contract. If these criteria are not met, the promised goods and services are accounted for as a combined performance obligation. The transaction price is determined based on the consideration to which the Company will be entitled in exchange for transferring goods and services to the customer. To the extent the transaction price includes variable consideration, the Company estimates the amount of variable consideration that should be included in the transaction price utilizing either the expected value method or the most likely amount method, depending on the nature of the variable consideration. Variable consideration is included in the transaction price if, in the Company’s judgment, it is probable that a significant future reversal of cumulative revenue under the contract will not occur. Any estimates, including the effect of the constraint on variable consideration, are evaluated at each reporting period for any changes. Determining the transaction price requires significant judgment, which is discussed in further detail for each of the Company’s collaboration agreements in Note 5. In addition, neither of the Company’s contracts as of June 30, 2019 contained a significant financing component. If the contract contains a single performance obligation, the entire transaction price is allocated to the single performance obligation. Contracts that contain multiple performance obligations require an allocation of the transaction price to each performance obligation on a relative standalone selling price basis unless the transaction price is variable and meets the criteria to be allocated entirely to a performance obligation or to a distinct service that forms part of a single performance obligation. The consideration to be received is allocated among the separate performance obligations based on relative standalone selling prices. The Company typically determines standalone selling prices using an adjusted market assessment approach model. The Company satisfies performance obligations either over time or at a point in time. Revenue is recognized over time if either (i) the customer simultaneously receives and consumes the benefits provided by the entity’s performance, (ii) the entity’s performance creates or enhances an asset that the customer controls as the asset is created or enhanced, or (iii) the entity’s performance does not create an asset with an alternative use to the entity and the entity has an enforceable right to payment for performance completed to date. If the entity does not satisfy a performance obligation over time, the related performance obligation is satisfied at a point in time by transferring the control of a promised good or service to a customer. As of June 30, 2019, the Company’s only revenue recognized is related to collaboration agreements with third parties which are either within the scope of ASC 606, under which the Company licenses certain rights to its product candidates to third parties, or within the scope of ASC 808, Collaborative Arrangements Licenses of intellectual property: If the license to the Company’s IP is determined to be distinct from the other performance obligations identified in the arrangement, the Company recognizes revenues from consideration allocated to the license when the license is transferred to the customer and the customer is able to use and benefit from the licenses. For licenses that are combined with other promises, the Company utilizes judgment to assess the nature of the combined performance obligation to determine whether the combined performance obligation is satisfied over time or at a point in time and, if over time, the appropriate method of measuring progress for purposes of recognizing revenue. The Company evaluates the measure of progress each reporting period and, if necessary, adjusts the measure of performance and related revenue recognition. Milestone payments: At the inception of each arrangement that includes development milestone payments, the Company evaluates the probability of reaching the milestones and estimates the amount to be included in the transaction price using the most likely amount method. If it is probable that a significant revenue reversal would not occur in the future, the associated milestone value is included in the transaction price. Milestone payments that are not within the control of the Company or the licensee, such as regulatory approvals, are not considered probable of being achieved until those approvals are received and therefore revenue recognized is constrained as management is unable to assert that a reversal of revenue would not be possible. The transaction price is then allocated to each performance obligation on a relative standalone selling price basis, for which the Company recognizes revenue as or when the performance obligations under the contract are satisfied. At the end of each subsequent reporting period, the Company re-evaluates the probability of achievement of such development milestones and any related constraint, and if necessary, adjusts its estimate of the overall transaction price. Any such adjustments are recorded on a cumulative catch-up basis, which would affect collaboration revenues and earnings in the period of adjustment. Royalties: For arrangements that include sales-based royalties, including milestone payments based on levels of sales, if the license is deemed to be the predominant item to which the royalties relate, the Company recognizes revenue at the later of (i) when the related sales occur, or (ii) when the performance obligation to which some or all of the royalty has been allocated has been satisfied (or partially satisfied). To date, the Company has not recognized any royalty revenue resulting from any of its collaboration agreements. The Company receives payments from its customers based on billing schedules established in each contract. The Company’s contract liabilities consist of deferred revenue. Upfront payments and fees are recorded as deferred revenue upon receipt or when due and may require deferral of revenue recognition to a future period until the Company satisfies its obligations under these arrangements. The Company also considers the nature and contractual terms of an arrangement and assesses whether the arrangement involves a joint operating activity pursuant to which the Company is an active participant and is exposed to significant risks and rewards with respect to the arrangement. If the Company is an active participant and is exposed to the significant risks and rewards with respect to the arrangement, the Company accounts for the arrangement under ASC 808 . Based on this consideration, the Company accounts for its Co-Development and Co-Promotion Agreement (“Co/Co”) with Regeneron Pharmaceuticals, Inc. (“Regeneron”) under ASC 808. Because ASC 808 does not provide recognition and measurement guidance for collaborative arrangements, the Company has analogized to ASC 606. Refer to Note 5 for additional information regarding the Company’s collaboration agreements . The following table presents changes in the Company’s contract liabilities during the six months ended June 30, 2019 and 2018 (in thousands): Balance at Beginning of Period Additions Deductions Balance at End of Period Six Months Ended June 30, 2019 Contract liabilities: Deferred revenue $ 55,932 $ 2,000 $ (15,772 ) $ 42,160 Balance at Beginning of Period Additions Deductions Balance at End of Period Six Months Ended June 30, 2018 Contract liabilities: Deferred revenue $ 59,868 $ 2,000 $ (11,009 ) $ 50,859 During the six months ended June 30, 2019 and 2018, the Company recognized the following revenues as a result of changes in the contract liability balance (in thousands): Revenue recognized in the period from: Six Months Ended June 30, 2019 Six Months Ended June 30, 2018 Amounts included in the contract liability at the beginning of the period $ 15,772 $ 11,009 Costs to obtain and fulfill a contract The Company did not incur any expenses to obtain collaboration agreements and costs to fulfill those contracts do not generate or enhance resources of the Company. As such, no costs to obtain or fulfill a contract have been capitalized in any period. Recent Accounting Pronouncements In February 2016, the Financial Accounting Standards Board (“FASB”) issued ASU No. 2016-02, Leases Leases The Company adopted the new standard using the required modified retrospective approach on January 1, 2019 and used the effective date as its date of initial application. As a result, prior periods are presented in accordance with the previous guidance in ASC Topic 840, Leases . ASC 842 provides several optional practical expedients in transition. The Company elected the package of practical expedients which allows the Company to not reassess its existing conclusions on lease identification, classification, and initial direct costs. Further, the Company elected the hindsight practical expedient and utilized the short-term lease exemption for all leases with an original term of 12 months or less, for purposes of applying the recognition and measurement requirements of the new standard. The Company also elected the practical expedient which allows it to not separate lease and non-lease components for all its leases. The adoption of the new standard on January 1, 2019 resulted in the recognition of operating lease liabilities of $20.6 million, and right-of-use assets of $22.3 million on the Company’s condensed consolidated balance sheet relating to its leases. Further, an adjustment to retained earnings of $0.3 million was recognized due to the use of hindsight being applied in updating the lease term for one of the Company’s property leases. The adoption of the standard did not have a material effect on the Company’s condensed consolidated statements of operations and comprehensive loss or condensed consolidated statements of cash flows Refer to Note 6, “Leases”, for the Company’s current lease commitments. In June 2018, the FASB issued ASU 2018-07, Compensation—Stock Compensation (Topic 718): Improvements to Nonemployee Share-Based Payment Accounting , which simplifies the accounting for share-based payments to nonemployees by aligning it with the accounting for share-based payments to employees, with certain exceptions. have a material effect on the Company’s condensed consolidated financial statements. In August 2018, the FASB issued ASU 2018-13, Fair Value Measurement (Topic 820): Disclosure Framework – Changes to the Disclosure Requirements for Fair Value Measurement, (“ASU 2018-13”). |
Fair Value Measurements
Fair Value Measurements | 6 Months Ended |
Jun. 30, 2019 | |
Fair Value Disclosures [Abstract] | |
Fair Value Measurements | 3. Fair Value Measurements The Company classifies fair value-based measurements using a three-level hierarchy that prioritizes the inputs used to measure fair value. This hierarchy requires entities to maximize the use of observable inputs and minimize the use of unobservable inputs. The three levels of inputs used to measure fair value are as follows: Level 1, quoted market prices in active markets for identical assets or liabilities; Level 2, observable inputs other than quoted market prices included in Level 1, such as quoted market prices for markets that are not active or other inputs that are observable or can be corroborated by observable market data; and Level 3, unobservable inputs that are supported by little or no market activity and that are significant to the fair value of the assets or liabilities, including certain pricing models, discounted cash flow methodologies and similar techniques that use significant unobservable inputs. The Company’s financial instruments as of June 30, 2019 and December 31, 2018 included cash and cash equivalents, marketable securities, accounts receivable and accounts payable. As of June 30, 2019 and December 31, 2018, the Company’s financial assets recognized at fair value on a recurring basis consisted of the following: Fair Value as of June 30, 2019 Total Level 1 Level 2 Level 3 (In thousands) Cash equivalents $ 45,799 $ 45,799 $ - $ - Marketable securities: U.S. Treasury securities 146,713 146,713 - - Financial institution debt securities 53,110 - 53,110 - Corporate debt securities 18,892 - 18,892 - Other asset-backed securities 7,983 - 7,983 - Total marketable securities 226,698 146,713 79,985 - Total $ 272,497 $ 192,512 $ 79,985 $ - Fair Value as of December 31, 2018 Total Level 1 Level 2 Level 3 (In thousands) Cash equivalents $ 45,986 $ 45,986 $ - $ - Marketable securities: U.S. Treasury securities 165,948 165,948 - - Financial institution debt securities 65,420 - 65,420 - Corporate debt securities 23,835 - 23,835 - Total marketable securities 255,203 165,948 89,255 - Total $ 301,189 $ 211,934 $ 89,255 $ - The Company’s financial assets, which include cash equivalents and marketable securities, have been initially valued at the transaction price, and subsequently revalued at the end of each reporting period, utilizing third-party pricing services or other observable market data. The pricing services utilize industry standard valuation models and observable market inputs to determine value . After completing our validation procedures, the Company did not adjust or override any fair value measurements provided by the pricing services as of June 30, 2019 or December Other financial instruments, including accounts receivable and accounts payable, are carried at cost, which approximate fair value due to the short duration and term to maturity. |
Accrued Expenses
Accrued Expenses | 6 Months Ended |
Jun. 30, 2019 | |
Payables And Accruals [Abstract] | |
Accrued Expenses | 4. Accrued Expenses Accrued expenses consisted of the following: June 30, 2019 December 31, 2018 (In thousands) Accrued legal and professional expenses $ 4,837 $ 1,633 Accrued employee compensation and benefits 3,803 6,175 Accrued research and development 2,485 2,328 Accrued other 773 606 Total accrued expenses $ 11,898 $ 10,742 |
Collaborations
Collaborations | 6 Months Ended |
Jun. 30, 2019 | |
Organization Consolidation And Presentation Of Financial Statements [Abstract] | |
Collaborations | 5. Collaborations To accelerate the development and commercialization of CRISPR/Cas9-based products in multiple therapeutic areas, the Company has formed, and intends to seek other opportunities to form, strategic alliances with collaborators who can augment its leadership in CRISPR/Cas9 therapeutic development. Novartis Institutes for BioMedical Research (“Novartis”) In December 2014, the Company entered into a strategic collaboration agreement with Novartis, as amended, (the “2014 Novartis Agreement”) primarily focused on the development of new ex vivo Agreement Structure. The parties agreed to engage in collaborative research activities using its CRISPR/Cas9 platform to identify and research therapeutic, prophylactic and palliative products and services relating to the following applications: a) HSCs and b) CAR-Ts. In addition, in the last two years of the collaboration term, Novartis may engage in research and development of a limited number of targets using the Company’s platform. Scope of Collaboration. During the five-year collaboration term parties may research potential therapeutic, prophylactic and palliative applications of the CRISPR/Cas9 technology in HSCs and CAR-T cells. Research expenses incurred by the Company in support of the collaboration are reimbursed by Novartis. HSC Program. The Company and Novartis agreed to conduct research of HSC targets under a research plan agreed upon by both parties. Within the HSC therapeutic space, Novartis may obtain exclusive rights to a limited number of these HSC targets, to be selected by Novartis in a series of selection windows. The Company has the right to choose a limited number of HSC targets for its exclusive development and commercialization per the specified selection schedule. Following these selections by Novartis and the Company, Novartis may obtain rights to research an additional limited number of HSC targets on a non-exclusive basis. Novartis is required to use commercially reasonable efforts to research, develop and commercialize at least one HSC product directed to each of their selected HSC targets . CAR-T Program . The Company has also agreed to collaborate with Novartis on research activities for CAR-T cell targets. After completion of the activities contemplated by the parties’ CAR-T cell program research plan, Novartis will assume sole responsibility for developing any products that it selects, arising from that research plan and the costs and expenses of developing, manufacturing and commercializing its selected research targets. Novartis is required to use commercially reasonable efforts to research, develop or commercialize at least one CAR-T cell product directed to each of its selected CAR-T cell targets. In Vivo Program. During the last two years of the five-year collaboration term, Novartis has the option to select a limited number of targets for research, development and commercialization of in vivo therapies using the Company’s CRISPR/Cas9 platform, on a non-exclusive basis. Following Novartis’ selection of each in vivo target, Novartis may offer the Company the right to participate in the research and development of such targets, in which case an in vivo program research plan for such target will be entered into between the Company and Novartis. Novartis is required to use commercially reasonable efforts to research, develop or commercialize at least one in vivo product directed to each of its selected targets. Novartis’ in vivo target selections are subject to certain restrictions, including that the targets, or all targets within a limited number of organs: (i) have not already been reserved by the Company pursuant to its limited right to do so under the agreement; (ii) are not the subject of a collaboration or pending collaboration with a third party; and (iii) are not the subject of ongoing or planned research and development by the Company. Governance. The parties formed HSC and CAR-T steering committees with responsibility for oversight of these respective research programs and approval of the associated research plans. Beginning in December 2018, the HSC steering committee became responsible for the ocular stem cell (“OSC”) program. These steering committees in turn are overseen by a joint steering committee. The above steering committees are comprised by an equal number of representatives from each party. Financial Terms . The Company received an upfront technology access payment from Novartis of $10.0 million in January 2015 and was entitled to additional technology access fees of $20.0 million and quarterly research payments of $1.0 million, or up to $20.0 million in the aggregate, during the five-year research term. To date, the Company has received $20.0 million in technology access fees and $17.0 million in research payments related to these programs. In addition, for each Novartis product under the collaboration (whether HSC or CAR-T, and beginning as of December 2018, OSC), subject to certain conditions, the Company may be eligible to receive (i) up to $30.3 million in development milestones, including for the filing of an investigational new drug (“IND”) application and for the dosing of the first patient in each of Phase IIa, Phase IIb and Phase III clinical trials, (ii) up to $50.0 million in regulatory milestones for the product’s first indication, including regulatory approvals in the U.S. and European Union (“EU”), (iii) up to $50.0 million in regulatory milestones for the product’s second indication, if any, including U.S. and EU regulatory approvals, (iv) royalties on net sales in the mid-single digits, and (v) net sales milestone payments of up to $100.0 million. The Company is also eligible to receive payments for: (i) each additional HSC target selected by Novartis beyond its initial defined allocation, for which it will receive $1.0 million for each target, (ii) each in vivo target that Novartis selects as described above, and (iii) any exercise by Novartis of certain license options under the 2014 Novartis Agreement. Upon completion of the research collaboration term in December 2019, Novartis has the option to internalize the Intellia platform for a $50.0 million fee, which will allow them to select a limited number of additional CRISPR/Cas9 genome editing targets over 5 years. Up to $20.0 million of the internalization fee will be credited towards any milestone payments for any additional post-internalization targets (up to $4.0 million per target). Equity Investments. Additionally, at the inception of the arrangement, at which time the Company was a privately held company, Novartis invested $9.0 million to purchase the Company’s Class A-1 and Class A-2 Preferred Units. The difference between the cash proceeds received from Novartis for the units and the $11.6 million estimated fair value of those units at the date of issuance was determined to be $2.6 million. Accordingly, $2.6 million of the upfront technology access payment was allocated to record the preferred units purchased by Novartis at fair value. License Grant to Novartis. In the 2014 Novartis Agreement, the Company granted to Novartis a license to its CRISPR/Cas9 platform technology, including a sublicense to certain platform rights licensed from Caribou Biosciences, Inc. (“Caribou”), that is exclusive in the HSC, CAR-T cell and in vivo fields with respect to each target selected by Novartis pursuant to the agreement and the research plan as long as Novartis continues to use commercially reasonable efforts to research, develop, and commercialize CRISPR-edited products directed to such targets. Upon the expiration of the collaboration term, Novartis shall have the option to access and obtain a non-exclusive license to the Company’s CRISPR/Cas9 platform technology to research, develop and commercialize potential therapeutic, prophylactic and palliative products and services for a limited number of certain approved targets selected by Novartis, exercisable upon written notice to the Company within 30 days after the expiration of the collaboration term. Such approved targets are subject to certain restrictions, including that the targets may not have been already reserved by the Company pursuant to its limited right to do so under the agreement, may not be the subject of an existing out license of the Company’s CRISPR/Cas9 platform to a third party and may not be the subject of ongoing or planned research and development by the Company. This non-exclusive license will have a term of five years commencing upon the completion of the technology transfer by the Company enabling Novartis to practice such licensed rights, and Novartis may not select more than a specified number of approved targets in each year of this license term. License Grant to Intellia. Novartis granted the Company a non-exclusive license to its IP covering small molecule for HSC expansion and to its LNP platform technology to research, develop and commercialize HSC and genome editing products, respectively, in the 2014 Novartis Agreement . Intellectual Property. IP that the Company develops within the collaboration related to the Company’s CRISPR/Cas9 platform will be owned solely by the Company, while all other IP developed within the collaboration, including IP covering products arising from the collaboration, will be jointly owned by the Company and Novartis. 2018 Amendment to the Agreement. In December 2018, the Company entered into an amendment to this agreement with Novartis (the “Novartis Amendment”) which expanded the scope of the 2014 Novartis Agreement to include the ex vivo development of CRISPR/Cas9-based cell therapies using limbal stem cells (“LSCs”) , a type of OSC, primarily against gene targets selected by Novartis in exchange for a one-time payment of $10.0 million which the Company received in December 2018. The governance, milestones and royalties associated with any LSC program will follow those for the HSC programs. As part of the Novartis Amendment, Intellia rights to Novartis’ lipid nanoparticle (“LNP”) technology were expanded to include use on all genome editing applications in both in vivo and ex vivo settings . Term and Termination . The collaboration term ends in December 2019. The term of the agreement expires on the later of (i) the expiration of Novartis’ payment obligations under the agreement and (ii) the date of expiration of the last-to-expire of the patent rights licensed to the Company or Novartis under the agreement. Novartis’ royalty payment obligations expire on a country-by-country and product-by-product basis upon the later of (i) the expiration of the last valid claim of the royalty-bearing patents covering such product in such country or (ii) 10 years after the first commercial sale of such product in such country. The Company may terminate the agreement if Novartis or its affiliates institute a patent challenge against its IP rights, and all improvements thereto, licensed to Novartis under the agreement. Novartis may terminate the agreement, without cause, upon 90 days’ written notice to the Company subject to certain conditions, including its payment of any accrued and future obligations as if the collaboration had continued through December 2019. Either party may terminate the agreement in the event of the other party’s uncured material breach or bankruptcy—or insolvency-related events. Accounting Analysis. The Company has concluded that the 2014 Novartis Agreement and the Novartis Amendment are subject to ASC 606 and has assessed its accounting for them accordingly . The Company evaluated the promised goods and services under the 2014 Novartis Agreement and determined that it included two performance obligations: (1) a combined performance obligation representing a series of distinct goods and services including the licenses to research, develop and commercialize HSC products and their associated research activities and the licenses to research, develop and commercialize CAR-T cell products and their associated research activities; and (2) the preferred units. The Company determined that the transaction price of the 2014 Novartis Agreement was $59.0 million consisting of the following consideration: (1) the upfront technology access payment of $10.0 million; (2) the additional technology access fees of $20.0 million; (3) the Company’s estimate of variable consideration of $20.0 million related to the quarterly research payments; and (4) the payment for the preferred units of $9.0 million. None of the clinical or regulatory milestones were included in the transaction price, as all milestone amounts were fully constrained. As part of its evaluation of the constraint, the Company considered numerous factors, including that receipt of the milestones is outside the control of the Company and contingent upon future regulatory progress and the licensee’s efforts. Any consideration related to sales-based milestones and royalties will be recognized when the related sales occur as they were determined to relate predominantly to the licenses granted to Novartis and therefore have also been excluded from the transaction price. The Company will re-evaluate the transaction price in each reporting period and when events whose outcomes are resolved or other changes in circumstances occur The Company first allocated $11.6 million of the transaction price to the preferred units to record the preferred units purchased by Novartis at fair value. The Company then allocated the remaining $47.4 million of the transaction price to the remaining combined performance obligation of the licenses and associated research activities for HSC and CAR-T cell products. Revenue allocated to the combined performance obligation of the licenses and associated research activities for HSC and CAR-T cell products is being recognized on a straight-line basis over a period of five years, which, in management’s judgment, is the best measure of progress towards satisfying the performance obligation and represents the Company’s best estimate of the period of the obligation. The Company determined that there is only one combined performance obligation identified under the Novartis Amendment, representing a series of distinct goods and services including the licenses to research, develop and commercialize products using LSCs and their associated research and development services related to the research, development and commercialization of products using LSCs, and allocated the $10.0 million transaction price accordingly. Revenue allocated to this performance obligation is being recognized on a straight-line basis over a period of approximately one year, which, in management’s judgment, is the best measure of progress towards satisfying the performance obligation and represents the Company’s best estimate of the period of the obligation. Revenue Recognition - Collaboration Revenue. Through June 30, 2019, excluding amounts allocated to Novartis’ purchase of the Company’s Class A-1 and Class A-2 Preferred Units, the Company had recorded a total of $55.4 million in cash and accounts receivable under the 2014 Novartis Agreement and the Novartis Amendment. Through June 30, 2019, the Company has recognized $48.4 million of collaboration revenue, including $4.8 million and $9.5 million during the three and six months ended June 30, 2019, respectively, and $2.4 million and $4.7 million during the three and six months ended June 30, 2018, respectively, in the condensed consolidated statements of operations and comprehensive loss related to the 2014 Novartis Agreement and the Novartis Amendment. As of June 30, 2019, there was approximately $9.0 million of the aggregate transaction price remaining to be recognized, which will be recognized through December 2019. As of June 30, 2019 and December 31, 2018, the Company had accounts receivable of $1.0 million and $6.0 million, respectively, and deferred revenue of $7.0 million and $14.5 million, respectively, related to the 2014 Novartis Agreement and the Novartis Amendment. Regeneron Pharmaceuticals, Inc. In April 2016, the Company entered into a license and collaboration agreement with Regeneron (the “Regeneron Agreement”). Agreement Structure. The Regeneron Agreement has two principal components: i) a product development component under which the parties will research, develop and commercialize CRISPR/Cas-based therapeutic products primarily focused on genome editing in the liver, and ii) a technology collaboration component, pursuant to which the Company and Regeneron will engage in research and development activities aimed at discovering and developing novel technologies and improvements to CRISPR/Cas technology to enhance the Company’s genome editing platform. Under this agreement, the Company also may access the Regeneron Genetics Center and proprietary mouse models to be provided by Regeneron for a limited number of the Company’s liver programs. Scope of Collaboration. Under the terms of the six-year collaboration, Regeneron may obtain exclusive rights for up to 10 targets to be chosen by Regeneron during the collaboration term, subject to a target selection process and various adjustments and limitations set forth in the agreement. Of these 10 total targets, Regeneron may select up to five non-liver targets, while the remaining targets must be focused in the liver. Certain non-liver targets from the Company’s ongoing and planned research at the time, as well as any targets included in another of the Company’s collaborations, are excluded from this collaboration. At the inception of the agreement, Regeneron selected the first of its 10 targets, transthyretin amyloidosis (“ATTR”), which is subject to a Co/Co between the Company and Regeneron, the general terms and conditions for which were outlined within the Regeneron Agreement. Research Collaboration. Research activities under the collaboration will be governed by evaluation and research and development plans that will outline the parties’ responsibilities under, anticipated timelines of and budgets for, the various programs. The Company will assist Regeneron with the preliminary evaluation of its selected liver targets, and Regeneron will be responsible for preclinical research and conducting clinical development, manufacturing and commercialization of products directed to each of its exclusive targets. The Company may assist, as requested by Regeneron, with the later discovery and research of product candidates directed to any selected target. For each selected target, Regeneron is required to use commercially reasonable efforts to submit regulatory filings necessary to achieve IND acceptance for at least one product directed to each applicable target, and following IND acceptance for at least one product, to develop and commercialize such product. Reserved Liver Targets. The Company retains the exclusive right to solely develop products via CRISPR genome editing directed against certain specified genetic targets. During the collaboration term and subject to a target selection process, the Company has the right to choose additional liver targets for its own development using commercially reasonable efforts. Certain targets that either the Company or Regeneron select during the term may be subject to further co-development and co-commercialization arrangements at the Company or Regeneron’s option, as applicable, which either can exercise pursuant to defined conditions. Governance. The parties formed a joint steering committee, which is responsible for setting research objectives and overseeing the general strategies and activities undertaken by the parties under the Regeneron Agreement. Additionally, the parties formed a Joint Development and Commercialization Committee (“JDCC”) to oversee all profit share products under the Co/Co discussed below. The JDCC will have responsibility for overseeing the development, manufacture, regulatory matters, and commercialization (including pricing and reimbursement) of ATTR, as the first profit share product under the collaboration agreement. Financial Terms. The Company received a nonrefundable upfront payment of $75.0 million. In addition, on Regeneron programs that are not subject to co-development and co-promotion agreements the Company may be eligible to earn, on a per-licensed target basis, (i) up to $25.0 million in development milestones, including for the dosing of the first patient in each of Phase I, Phase II and Phase III clinical trials, (ii) up to $110.0 million in regulatory milestones, including for the acceptance of a regulatory filing in the U.S., and for obtaining regulatory approval in the U.S. and in certain other identified countries, and (iii) up to $185.0 million in sales-based milestone payments. The Company is also eligible to earn royalties ranging from the high single digits to low teens, in each case, on a per-product basis, which royalties are potentially subject to various reductions and offsets and incorporate the Company’s existing low- to mid-single-digit royalty obligations under a license agreement with Caribou. In addition, Regeneron is obligated to fund 50.0 percent of the research and development costs for the ATTR program. Equity Investments. In connection with this collaboration, Regeneron purchased $50.0 million of the Company’s common stock in a private placement under a Stock Purchase Agreement concurrent with the Company’s initial public offering. Term and Termination . The collaboration term ends in April 2022, except that Regeneron may make a one-time payment of $25.0 million to extend the term for an additional two-year period. The agreement will continue until the date when no royalty or other payment obligations are due, unless earlier terminated in accordance with the terms of the agreement. Regeneron’s royalty payment obligations expire on a country-by-country and product-by-product basis upon the later of (i) the expiration of the last valid claim of the royalty-bearing patents covering such product in such country, (ii) 12 years from the first commercial sale of such product in such country, or (iii) the expiration of regulatory exclusivity for such product. The Company may terminate the agreement on a target-by-target basis if Regeneron does not proceed with the development of a product directed to a selected target within specified periods of time. Regeneron may terminate the agreement, without cause, upon 180 days written notice to the Company, either in its entirety or on a target-by-target basis, in which event, certain rights in the terminated targets and associated IP revert to the Company, as described in the agreement. Following such termination, the Company may owe Regeneron royalties, in certain circumstances, up to mid-single digits on any terminated targets that the Company subsequently commercializes on a product-by-product basis for a period of 12 years after the first commercial sale of any such products. Either party may terminate the agreement either in its entirety or with respect to the technology collaboration or one or more of the targets selected by Regeneron, in the event of the other party’s uncured material breach. Co-Development and Co-Promotion Agreement. In July 2018, the Company and Regeneron finalized the form of the Co/Co that will be used as the basis for each Co/Co agreement directed to a target. Simultaneously, the Company and Regeneron executed the Co/Co agreement directed to the first collaboration target, ATTR, for which the Company will be the clinical and commercial Lead Party (see below). As such, Regeneron will be the Participating Party (see below) and will share equally in worldwide development costs and profits as long as it funds half of the defined research and development costs attributable to the ATTR program. Co-Development and Co-Promotion: Agreement Structure . Under the Co/Co a greement, Regeneron has the right to exercise up to at least five Co/Co options on the Company’s liver targets (other than the Company’s reserved liver targets), while the Company may exercise at least one Co/Co option on Regeneron’s liver targets, the exact number of Co/Co options being subject to certain conditions of the target selection process. Co/Co options must be exercised (or forfeited) once a target reaches a defined preclinical stage. Within 15 days of exercising the Co/Co option, the party exercising the option must pay $ 1.5 million to the other party as compensation for prior work. The ATTR program was exempted from this payment. One Party will be the “Lead Party” and the other Party the “Participating Party”. The Lead Party shall have control and primary responsibility for the development, manufacturing, regulatory and commercial activities. The Participating Party shall have the right to consult on these activities through its participation on the JDCC and will have the right to co-fund development and commercialization activities in exchange for a share of profits. In general, the parties will share equally in worldwide development costs and profits of any future products. Prior to reaching a specific development milestone, the Participating Party may elect to reduce its share of worldwide development costs and profits by 50.0 percent . Co-Development and Co-Promotion: Termination. Either party may terminate by providing 180 days written notice. If the Company terminates, the product becomes a Regeneron product, and is subject to all future milestone and royalty payment obligations under the Regeneron agreement. If Regeneron terminates and has contributed at least $5.0 million in development costs, the Company will pay low- to mid-single digit royalties on the net sales of the product, depending on co-funding percentage, stage at termination, if any, and Regeneron IP incorporated into the product. Accounting Analysis. The Company determined that the Regeneron Agreement is within the scope of ASC 606. The Company evaluated the promised goods and services under the Regeneron Agreement and determined that the Regeneron Agreement included three performance obligations: (1) a combined performance obligation including the licenses to targets and the associated research activities and evaluation plans; (2) a combined performance obligation including the technology collaboration and associated research activities; and (3) the common stock. The Company also concluded that the ATTR Co/Co meets the definition of a collaborative arrangement per ASC 808, which is outside of the scope of ASC 606. Since ASC 808 does not provide recognition and measurement guidance for collaborative arrangements, the Company has analogized to ASC 606. As such, the Company classifies payments received or made under the cost sharing provisions of the ATTR Co/Co as a component of revenues in the condensed consolidated statements of operations and comprehensive loss. Under the Regeneron Agreement, the Company determined that the transaction price was $125.0 million, consisting of the following consideration: (1) the nonrefundable upfront payment of $75.0 million; and (2) the payment for the common stock of $50.0 million. None of the clinical or regulatory milestones were included in the transaction price, as all milestone amounts were fully constrained. As part its evaluation of the constraint, the Company considered numerous factors, including that receipt of the milestones is outside the control of the Company and contingent upon success in future regulatory progress and the licensee’s efforts. Any consideration related to sales-based milestones and royalties will be recognized when the related sales occur as they were determined to relate predominantly to the licenses granted to Regeneron and therefore have also been excluded from the transaction price. The Company will re-evaluate the transaction price in each reporting period and when events whose outcome are resolved or other changes in circumstances occur. The Company first allocated $50.0 million of the transaction price to the common stock. The common stock was sold at its standalone selling price and the Company concluded that the total discount inherent in the arrangement is entirely attributable to the combined performance obligation including the licenses to targets and associated research activities and evaluation plans and the combined performance obligation including the technology collaboration and associated research activities. As such, the remaining $75.0 million of the transaction price was allocated to the combined performance obligation including the licenses to targets and associated research activities and evaluation plans and the combined performance obligation including the technology collaboration and associated research activities on a relative standalone selling price basis. The Company estimated the standalone selling price of each combined performance obligation by taking into consideration internal estimates of research and development personnel needed to perform the research and development services, estimates of expected cash outflows to third parties for services and supplies, selling prices of comparable transactions and typical gross profit margins. As a result of this evaluation, the Company allocated $63.8 million to the combined performance obligation including the licenses to targets and associated research activities and evaluation plans and $11.2 million to the combined performance obligation including the technology collaboration and associated research activities. The $63.8 million allocated to the combined performance obligation including the licenses to targets and associated research activities and evaluation plans is being recognized on a straight-line basis over the six-year performance period of the arrangement, which, in management’s judgment, is the best measure of progress towards satisfying the performance obligation and represents the Company’s best estimate of the period of the obligation. The $ 11.2 million allocated to the combined performance obligation including the technology collaboration and associated research activities is being recognized on a straight-line basis over a period beginning with the inception of the technology collaboration in September 2016 through the end of the arrangement, which, in management’s judgment, is the best measure of progress towards satisfying the performance obligation and represents the Company’s best estimate of the period of the obligation . Revenue Recognition – Collaboration Revenue. Through June 30, 2019, excluding the amounts allocated to Regeneron’s purchase of the Company’s common stock, the Company recorded a $75.0 million upfront payment and $17.9 million for research and development services under the Regeneron Agreement. Through June 30, 2019, the Company has recognized $57.7 million of collaboration revenue, including $6.3 million and $12.0 million during the three and six months ended June 30, 2019, respectively, and $5.3 million and $10.4 million during the three and six months ended June 30, 2018, respectively, in the condensed consolidated statements of operations and comprehensive loss related to this arrangement. This includes $3.2 million and $5.8 million during the three and six months ended June 30, 2019, respectively, and $2.1 million and $4.1 million during the three and six months ended June 30, 2018, respectively, representing payments due from Regeneron pursuant to the ATTR Co/Co, which is accounted for under ASC 808. As of June 30, 2019, there was approximately $35.2 million of the aggregate transaction price remaining to be recognized, which will be recognized ratably through April 2022. As of June 30, 2019 and December 31, 2018, the Company had accounts receivable of $3.2 million and $1.5 million, respectively, and deferred revenue of $35.2 million and $41.4 million, respectively, related to this arrangement. |
Leases
Leases | 6 Months Ended |
Jun. 30, 2019 | |
Leases [Abstract] | |
Leases | 6. L In October 2014, the Company entered into an agreement to lease office and laboratory space at 130 Brookline Street in Cambridge, Massachusetts under an operating lease agreement with a term through January 2020, with an option to extend the term of the lease for an additional five-year period. In April 2019, the Company executed an amendment to the lease to extend the term of the lease for the additional five-year period, through January 2025. Upon the execution of the original lease, the Company provided a $0.3 million security deposit. The Company has recorded this security deposit in other assets on the condensed consolidated balance sheets. In applying the ASC 842 transition guidance, the Company retained the classification of this lease as operating and recorded a lease liability and a right-of-use asset on the ASC 842 effective date with the five-year extension included in the lease term, based on the Company’s election of the hindsight practical expedient as the Company was reasonably certain to exercise this option term. In March 2019, the Company entered into a separate agreement to sublease additional office and laboratory space at 130 Brookline Street in Cambridge, Massachusetts under an operating sublease agreement with a term through April 2021, with two options to extend the agreement by one year each, for a total option period of up to two years. Upon commencement of the lease in April 2019, the Company recognized a right-of-use asset and lease liability of approximately $1.3 million. In January 2016, the Company entered into a ten-year agreement to lease office and laboratory space at 40 Erie Street in Cambridge, Massachusetts under an operating lease agreement, with an option to terminate the lease at the end of the sixth year and an option to extend the term of the lease for an additional three years. Upon the execution of this lease, the Company provided a $2.2 million security deposit, which has been recorded in other assets on the condensed consolidated balance sheets. In addition, the Company had prepaid $2.2 million in lease payments as of December 31, 2018 under the terms of this lease. Throughout the term of its leases, the Company is responsible for paying certain costs and expenses, in addition to the rent, as specified in the lease, including a proportionate share of applicable taxes, operating expenses and utilities. The variable portion of these costs are expensed as incurred and are disclosed as variable lease cost. The following table contains a summary of the lease costs recognized under Topic 842 and other information pertaining to the Company’s operating leases for the three and six months ended June 30, 2019: Three Months Ended Six Months Ended June 30, 2019 (in thousands) Lease cost Operating lease cost $ 1,880 $ 3,583 Variable lease cost 460 997 Total lease cost $ 2,340 $ 4,580 Other information Operating cash flows used for operating leases $ 1,635 $ 3,122 Operating lease liabilities arising from obtaining right-of-use assets 1,343 1,343 Weighted average remaining lease term 3.5 years 3.5 years Weighted average discount rate 9.00 % 9.00 % Future minimum lease payments under the Company’s non-cancelable operating leases as of June 30, 2019, are as follows: Future Operating Lease Payments Year Ending December 31, (in thousands) 2019 (excluding the six months ended June 30, 2019) $ 3,355 2020 6,483 2021 6,618 2022 4,732 2023 871 Thereafter 943 Total lease payments $ 23,002 Less: imputed interest (3,333 ) Less: leases not yet commenced - Total operating lease liabilities at June 30, 2019 $ 19,669 Future minimum lease payments under the Company’s non-cancelable operating leases as of December 31, 2018, are as follows: Year Ending December 31, (In 2019 $ 5,616 2020 4,963 2021 5,507 2022 3,861 Thereafter - $ 19,947 |
Equity-Based Compensation
Equity-Based Compensation | 6 Months Ended |
Jun. 30, 2019 | |
Disclosure Of Compensation Related Costs Sharebased Payments [Abstract] | |
Equity-Based Compensation | 7 . Equity-Based Compensation Equity-based compensation expense is classified in the condensed consolidated statements of operations and comprehensive loss as follows: Three Months Ended June 30, Six Months Ended June 30, 2019 2018 2019 2018 (In thousands) Research and development $ 2,096 $ 2,633 $ 3,879 $ 5,026 General and administrative 2,308 2,268 5,117 3,982 Total $ 4,404 $ 4,901 $ 8,996 $ 9,008 Restricted Stock Restricted stock is measured at fair value based on the quoted price of the Company’s common stock. The following table summarizes the Company’s restricted stock activity for the six months ended June 30, 2019: Number of Shares Weighted Average Grant Date Fair Value per Share Unvested restricted stock as of December 31, 2018 109,073 $ 15.53 Granted - - Vested (36,462 ) 1.34 Cancelled - - Unvested restricted stock as of June 30, 2019 72,611 $ 22.66 As of June 30, 2019, there was $0.8 Stock Options The weighted average grant date fair value of options, estimated as of the grant date using the Black-Scholes option pricing model, was $9.22 Three Months Ended June 30, Six Months Ended June 30, 2019 2018 2019 2018 Risk-free interest rate 2.1% 2.8% 2.4% 2.6% Expected life of options 5.5-6.0 years 5.5-6.25 years 5.5-6.0 years 5.5-6.25 years Expected volatility of underlying stock 67.9% 87.7% 68.6% 89.0% Expected dividend yield 0.0% 0.0% 0.0% 0.0% The following is a summary of stock option activity for the six months ended June 30, 2019: Number of Options Weighted Average Exercise Price per Share Weighted Average Remaining Contractual Term Aggregate Intrinsic Value (In years) (In thousands) Outstanding at December 31, 2018 5,037,663 $ 15.63 Granted 793,813 14.54 Exercised (233,872 ) 8.65 Forfeited (282,108 ) 18.79 Outstanding at June 30, 2019 5,315,496 $ 15.61 8.14 $ 14,978 Exercisable at June 30, 2019 2,165,526 $ 13.43 7.01 $ 10,193 As of June 30, 2019, there was $34.1 Of the unvested restricted stock outstanding and stock options outstanding as of June 30, 2019, 71,875 213,750 obtaining certain scientific, financial and regulatory milestones through 2020 and 188,750 are not included in computing the diluted (loss) earnings per share because the performance criteria had not been met as of the end of the reporting period |
Loss Per Share
Loss Per Share | 6 Months Ended |
Jun. 30, 2019 | |
Earnings Per Share [Abstract] | |
Loss Per Share | 8 . Loss Per Share The Company calculates basic (loss) earnings per share by dividing (loss) income by the weighted average number of common shares outstanding. The Company computes diluted (loss) earnings per share after giving consideration to the dilutive effect of stock options and unvested restricted stock that are outstanding during the period, except where such securities would be anti-dilutive. Basic and diluted loss per share was calculated as follows: Three Months Ended June 30, Six Months Ended June 30, 2019 2018 2019 2018 (In thousands) Net loss $ (25,683 ) $ (22,219 ) $ (47,623 ) $ (43,575 ) Weighted average shares outstanding, basic and diluted 45,814 42,836 45,526 42,441 Net loss per share, basic and diluted $ (0.56 ) $ (0.52 ) $ (1.05 ) $ (1.03 ) The following common stock equivalents were excluded from the calculation of diluted loss per share because their inclusion would have been anti-dilutive: Six Months Ended June 30, 2019 2018 (In thousands) Unvested restricted stock 73 261 Stock options 5,315 4,881 5,388 5,142 |
Stockholders_ Equity
Stockholders’ Equity | 6 Months Ended |
Jun. 30, 2019 | |
Equity [Abstract] | |
Stockholders’ Equity | 9 . Stockholders’ Equity The following tables present changes in stockholders’ equity for the six-month periods ended June 30, 2019 and 2018 (in thousands, except share data): Additional Accumulated Other Total Common Paid-In Comprehensive Accumulated Stockholders’ Shares Amount Capital Income (Loss) Deficit Equity (Deficit) Balance at December 31, 2018 45,224,480 $ 5 $ 478,968 $ (28 ) $ (201,025 ) $ 277,920 Retroactive adjustment to beginning accumulated deficit for adoption of ASC 842 - - - - (320 ) (320 ) Issuance of common stock through at-the-market offerings, net of issuance costs of $120 223,818 - 3,639 - - 3,639 Exercise of stock options 30,800 - 360 - - 360 Equity-based compensation - - 4,592 - - 4,592 Other comprehensive gain - - - 87 - 87 Net loss - - - - (21,940 ) (21,940 ) Balance at March 31, 2019 45,479,098 $ 5 $ 487,559 $ 59 $ (223,285 ) $ 264,338 Issuance of common stock through at-the-market offerings, net of issuance costs of $3 1,986,579 - 31,413 - - 31,413 Exercise of stock options 203,072 - 1,664 - - 1,664 Issuance of shares under employee stock purchase plan 45,826 - 534 - - 534 Equity-based compensation - - 4,404 - - 4,404 Other comprehensive gain - - - 196 - 196 Net loss - - - - (25,683 ) (25,683 ) Balance at June 30, 2019 47,714,575 $ 5 $ 525,574 $ 255 $ (248,968 ) $ 276,866 Additional Accumulated Other Total Common Paid-In Comprehensive Accumulated Stockholders’ Shares Amount Capital Income (Loss) Deficit Equity (Deficit) Balance at December 31, 2017 42,384,623 $ 4 $ 421,706 $ - $ (121,113 ) $ 300,597 Retroactive adjustment to beginning accumulated deficit for adoption of ASU 2014-09 - - - - 5,431 5,431 Exercise of stock options 709,321 - 6,720 - - 6,720 Cancellation of restricted stock (2,022 ) - - - - - Equity-based compensation - - 4,107 - - 4,107 Net loss - - - - (21,356 ) (21,356 ) Balance at March 31, 2018 43,091,922 $ 4 $ 432,533 $ - $ (137,038 ) $ 295,499 Exercise of stock options 68,163 - 557 - - 557 Issuance of shares under employee stock purchase plan 32,707 - 598 - - 598 Cancellation of restricted stock (4,780 ) - - - - - Equity-based compensation - - 4,901 - - 4,901 Net loss - - - - (22,219 ) (22,219 ) Balance at June 30, 2018 43,188,012 $ 4 $ 438,589 $ - $ (159,257 ) $ 279,336 |
Related Party Transactions
Related Party Transactions | 6 Months Ended |
Jun. 30, 2019 | |
Related Party Transactions [Abstract] | |
Related Party Transactions | 10 . Related Party Transactions Caribou Biosciences In July 2014, the Company licensed from Caribou certain IP (the “Caribou License”) and entered into an arrangement under which Caribou provided research and development services. In addition, under the license agreement the Company agreed to pay 30 percent of Caribou’s patent prosecution, filing and maintenance costs under its IP license agreement with Caribou. Caribou owned less than 5 percent of the Company’s voting interests as of June 30, 2019. On October 17, 2018, the Company initiated an arbitration proceeding with JAMS against Caribou asserting that Caribou is violating the terms and conditions of the Caribou License, as well as other contractual and legal rights, by using and seeking to license to third parties technology covered by two patent families (described in, for instance, PCT No. PCT/US2016/015145 and PCT No. PCT/US2016/064860, and related patents and applications) relating to specific structural or chemical modifications of guide RNAs, that were purportedly invented or controlled by Caribou, in the Company’s exclusive human therapeutic field. Caribou asserted that the two families of IP are outside the scope of the Company’s field of use under the license rights granted to the Company under the Caribou License. In accordance with the Caribou License, the Company submitted a demand for arbitration seeking among other relief a declaration that the disputed IP was included within the scope of its field of use under the Caribou License. A decision from the arbitration panel is expected during the third quarter of 2019. The Company recognized general and administrative expense of $0.7 million and $0.9 million during the three and six-month periods ended June 30, 2019 and $0.3 million and $0.5 million during the three and six-month periods ended June 30, 2018, respectively, related to the Company’s obligation to pay 30 percent of Caribou’s patent costs Novartis Institutes for BioMedical Research In connection with its entry into the collaboration and license agreement and related equity transactions with Novartis, in 2015 the Company issued Novartis capital stock, and in May 2016, Novartis acquired 277,777 shares of the Company’s common stock in a private placement transaction concurrent with the Company’s IPO. Novartis owned less than 10 The Company recognized collaboration revenue of $4.8 Collaborations |
Summary of Significant Accoun_2
Summary of Significant Accounting Policies (Policies) | 6 Months Ended |
Jun. 30, 2019 | |
Accounting Policies [Abstract] | |
Marketable Securities | Marketable Securities The following table summarizes the Company’s available-for-sale marketable securities as of June 30, 2019 and December 31, 2018 at net book value: June 30, 2019 Amortized Cost Gross Unrealized Gains Gross Unrealized Losses Estimated Fair Value (In thousands) Marketable securities: U.S. Treasury securities $ 146,552 $ 161 $ - $ 146,713 Financial institution debt securities 53,041 69 - 53,110 Corporate debt securities 18,892 - - 18,892 Other asset-backed securities 7,958 25 - 7,983 Total $ 226,443 $ 255 $ - $ 226,698 December 31, 2018 Amortized Cost Gross Unrealized Gains Gross Unrealized Losses Estimated Fair Value (In thousands) Marketable securities: U.S. Treasury securities $ 165,959 $ 2 $ (13 ) $ 165,948 Financial institution debt securities 65,436 1 (17 ) 65,420 Corporate debt securities 23,836 - (1 ) 23,835 Total $ 255,231 $ 3 $ (31 ) $ 255,203 The amortized cost of available-for-sale securities is adjusted for amortization of premiums and accretion of discounts to maturity. At June 30, 2019 and December 31, 2018, the balance in the Company’s accumulated other comprehensive income (loss) was composed of activity related to the Company’s available-for-sale marketable securities. There were no realized gains or losses in the period ended June 30, 2019, and as a result, the Company did not reclassify any amounts out of accumulated other comprehensive income (loss) during the period. The Company did not have any securities in an unrealized loss position at June 30, 2019. |
Leases | Leases Effective January 1, 2019, the Company adopted ASC Topic 842, Leases Leases At the inception of an arrangement, the Company determines whether an arrangement is or contains a lease based on the unique facts and circumstances present in the arrangement. Most leases with a term greater than one year are recognized on the balance sheet as right-of-use assets and short-term and long-term lease liabilities, as applicable. The Company has elected not to recognize leases with terms of 12 months or less on the balance sheet. The Company typically only includes an initial lease term in its assessment of a lease arrangement. Options to renew a lease are not included in the Company’s assessment unless there is reasonable certainty that the Company will renew. The Company monitors its plans to renew its material leases on a quarterly basis. Operating lease liabilities and their corresponding right-of-use assets are recorded based on the present value of lease payments over the expected remaining lease term. Certain adjustments to the right-of-use asset may be required for items such as incentives received. The interest rate implicit in the Company’s leases is typically not readily determinable. As a result, the Company utilizes its incremental borrowing rate to discount lease payments , which reflects the fixed rate at which the Company could borrow on a collateralized basis the amount of the lease payments in the same currency, for a similar term, in a similar economic environment. In its transition to ASC 842, the Company utilized the remaining lease term of its leases in determining the appropriate incremental borrowing rates. In accordance with ASC 842, components of a lease should be allocated between lease components (e.g., land, building, etc.) and non-lease components (e.g., common area maintenance, consumables, etc.). The fixed and in-substance fixed contract consideration must be allocated based on the respective relative fair values to the lease components and non-lease components. Although separation of lease and non-lease components is otherwise required, an expedient is available whereby the entity may account for each lease component and related non-lease component together as a single lease component. For new and amended leases beginning in 2019 and after, the Company has elected to account for the lease and non-lease components together as a single lease component for all underlying assets and allocate all of the contract consideration to the lease component only. |
Revenue Recognition | Revenue Recognition The Company adopted Accounting Standards Update (“ Revenue from Contracts with Customers (Topic 606) At inception, the Company determines whether contracts are within the scope of ASC 606 or other topics. For contracts that are determined to be within the scope of ASC 606, revenue is recognized when a customer obtains control of promised goods or services. The amount of revenue recognized reflects the consideration to which the Company expects to be entitled to receive in exchange for these goods and services. To achieve this core principle, the Company applies the following five steps (i) identify the contract with the customer; (ii) identify the performance obligations in the contract; (iii) determine the transaction price; (iv) allocate the transaction price to the performance obligations in the contract; and (v) recognize revenue when or as the Company satisfies a performance obligation. The Company only applies the five-step model to contracts when the Company determines that collection of substantially all consideration for goods and services that are transferred is probable based on the customer’s intent and ability to pay the promised consideration. Performance obligations promised in a contract are identified based on the goods and services that will be transferred to the customer that are both capable of being distinct and are distinct in the context of the contract. To the extent a contract includes multiple promised goods and services, the Company applies judgment to determine whether promised goods and services are both capable of being distinct and distinct in the context of the contract. If these criteria are not met, the promised goods and services are accounted for as a combined performance obligation. The transaction price is determined based on the consideration to which the Company will be entitled in exchange for transferring goods and services to the customer. To the extent the transaction price includes variable consideration, the Company estimates the amount of variable consideration that should be included in the transaction price utilizing either the expected value method or the most likely amount method, depending on the nature of the variable consideration. Variable consideration is included in the transaction price if, in the Company’s judgment, it is probable that a significant future reversal of cumulative revenue under the contract will not occur. Any estimates, including the effect of the constraint on variable consideration, are evaluated at each reporting period for any changes. Determining the transaction price requires significant judgment, which is discussed in further detail for each of the Company’s collaboration agreements in Note 5. In addition, neither of the Company’s contracts as of June 30, 2019 contained a significant financing component. If the contract contains a single performance obligation, the entire transaction price is allocated to the single performance obligation. Contracts that contain multiple performance obligations require an allocation of the transaction price to each performance obligation on a relative standalone selling price basis unless the transaction price is variable and meets the criteria to be allocated entirely to a performance obligation or to a distinct service that forms part of a single performance obligation. The consideration to be received is allocated among the separate performance obligations based on relative standalone selling prices. The Company typically determines standalone selling prices using an adjusted market assessment approach model. The Company satisfies performance obligations either over time or at a point in time. Revenue is recognized over time if either (i) the customer simultaneously receives and consumes the benefits provided by the entity’s performance, (ii) the entity’s performance creates or enhances an asset that the customer controls as the asset is created or enhanced, or (iii) the entity’s performance does not create an asset with an alternative use to the entity and the entity has an enforceable right to payment for performance completed to date. If the entity does not satisfy a performance obligation over time, the related performance obligation is satisfied at a point in time by transferring the control of a promised good or service to a customer. As of June 30, 2019, the Company’s only revenue recognized is related to collaboration agreements with third parties which are either within the scope of ASC 606, under which the Company licenses certain rights to its product candidates to third parties, or within the scope of ASC 808, Collaborative Arrangements Licenses of intellectual property: If the license to the Company’s IP is determined to be distinct from the other performance obligations identified in the arrangement, the Company recognizes revenues from consideration allocated to the license when the license is transferred to the customer and the customer is able to use and benefit from the licenses. For licenses that are combined with other promises, the Company utilizes judgment to assess the nature of the combined performance obligation to determine whether the combined performance obligation is satisfied over time or at a point in time and, if over time, the appropriate method of measuring progress for purposes of recognizing revenue. The Company evaluates the measure of progress each reporting period and, if necessary, adjusts the measure of performance and related revenue recognition. Milestone payments: At the inception of each arrangement that includes development milestone payments, the Company evaluates the probability of reaching the milestones and estimates the amount to be included in the transaction price using the most likely amount method. If it is probable that a significant revenue reversal would not occur in the future, the associated milestone value is included in the transaction price. Milestone payments that are not within the control of the Company or the licensee, such as regulatory approvals, are not considered probable of being achieved until those approvals are received and therefore revenue recognized is constrained as management is unable to assert that a reversal of revenue would not be possible. The transaction price is then allocated to each performance obligation on a relative standalone selling price basis, for which the Company recognizes revenue as or when the performance obligations under the contract are satisfied. At the end of each subsequent reporting period, the Company re-evaluates the probability of achievement of such development milestones and any related constraint, and if necessary, adjusts its estimate of the overall transaction price. Any such adjustments are recorded on a cumulative catch-up basis, which would affect collaboration revenues and earnings in the period of adjustment. Royalties: For arrangements that include sales-based royalties, including milestone payments based on levels of sales, if the license is deemed to be the predominant item to which the royalties relate, the Company recognizes revenue at the later of (i) when the related sales occur, or (ii) when the performance obligation to which some or all of the royalty has been allocated has been satisfied (or partially satisfied). To date, the Company has not recognized any royalty revenue resulting from any of its collaboration agreements. The Company receives payments from its customers based on billing schedules established in each contract. The Company’s contract liabilities consist of deferred revenue. Upfront payments and fees are recorded as deferred revenue upon receipt or when due and may require deferral of revenue recognition to a future period until the Company satisfies its obligations under these arrangements. The Company also considers the nature and contractual terms of an arrangement and assesses whether the arrangement involves a joint operating activity pursuant to which the Company is an active participant and is exposed to significant risks and rewards with respect to the arrangement. If the Company is an active participant and is exposed to the significant risks and rewards with respect to the arrangement, the Company accounts for the arrangement under ASC 808 . Based on this consideration, the Company accounts for its Co-Development and Co-Promotion Agreement (“Co/Co”) with Regeneron Pharmaceuticals, Inc. (“Regeneron”) under ASC 808. Because ASC 808 does not provide recognition and measurement guidance for collaborative arrangements, the Company has analogized to ASC 606. Refer to Note 5 for additional information regarding the Company’s collaboration agreements . The following table presents changes in the Company’s contract liabilities during the six months ended June 30, 2019 and 2018 (in thousands): Balance at Beginning of Period Additions Deductions Balance at End of Period Six Months Ended June 30, 2019 Contract liabilities: Deferred revenue $ 55,932 $ 2,000 $ (15,772 ) $ 42,160 Balance at Beginning of Period Additions Deductions Balance at End of Period Six Months Ended June 30, 2018 Contract liabilities: Deferred revenue $ 59,868 $ 2,000 $ (11,009 ) $ 50,859 During the six months ended June 30, 2019 and 2018, the Company recognized the following revenues as a result of changes in the contract liability balance (in thousands): Revenue recognized in the period from: Six Months Ended June 30, 2019 Six Months Ended June 30, 2018 Amounts included in the contract liability at the beginning of the period $ 15,772 $ 11,009 Costs to obtain and fulfill a contract The Company did not incur any expenses to obtain collaboration agreements and costs to fulfill those contracts do not generate or enhance resources of the Company. As such, no costs to obtain or fulfill a contract have been capitalized in any period. |
Recent Accounting Pronouncements | Recent Accounting Pronouncements In February 2016, the Financial Accounting Standards Board (“FASB”) issued ASU No. 2016-02, Leases Leases The Company adopted the new standard using the required modified retrospective approach on January 1, 2019 and used the effective date as its date of initial application. As a result, prior periods are presented in accordance with the previous guidance in ASC Topic 840, Leases . ASC 842 provides several optional practical expedients in transition. The Company elected the package of practical expedients which allows the Company to not reassess its existing conclusions on lease identification, classification, and initial direct costs. Further, the Company elected the hindsight practical expedient and utilized the short-term lease exemption for all leases with an original term of 12 months or less, for purposes of applying the recognition and measurement requirements of the new standard. The Company also elected the practical expedient which allows it to not separate lease and non-lease components for all its leases. The adoption of the new standard on January 1, 2019 resulted in the recognition of operating lease liabilities of $20.6 million, and right-of-use assets of $22.3 million on the Company’s condensed consolidated balance sheet relating to its leases. Further, an adjustment to retained earnings of $0.3 million was recognized due to the use of hindsight being applied in updating the lease term for one of the Company’s property leases. The adoption of the standard did not have a material effect on the Company’s condensed consolidated statements of operations and comprehensive loss or condensed consolidated statements of cash flows Refer to Note 6, “Leases”, for the Company’s current lease commitments. In June 2018, the FASB issued ASU 2018-07, Compensation—Stock Compensation (Topic 718): Improvements to Nonemployee Share-Based Payment Accounting , which simplifies the accounting for share-based payments to nonemployees by aligning it with the accounting for share-based payments to employees, with certain exceptions. have a material effect on the Company’s condensed consolidated financial statements. In August 2018, the FASB issued ASU 2018-13, Fair Value Measurement (Topic 820): Disclosure Framework – Changes to the Disclosure Requirements for Fair Value Measurement, (“ASU 2018-13”). |
Summary of Significant Accoun_3
Summary of Significant Accounting Policies (Tables) | 6 Months Ended |
Jun. 30, 2019 | |
Accounting Policies [Abstract] | |
Summary of Available-for-sale Marketable Securities | The following table summarizes the Company’s available-for-sale marketable securities as of June 30, 2019 and December 31, 2018 at net book value: June 30, 2019 Amortized Cost Gross Unrealized Gains Gross Unrealized Losses Estimated Fair Value (In thousands) Marketable securities: U.S. Treasury securities $ 146,552 $ 161 $ - $ 146,713 Financial institution debt securities 53,041 69 - 53,110 Corporate debt securities 18,892 - - 18,892 Other asset-backed securities 7,958 25 - 7,983 Total $ 226,443 $ 255 $ - $ 226,698 December 31, 2018 Amortized Cost Gross Unrealized Gains Gross Unrealized Losses Estimated Fair Value (In thousands) Marketable securities: U.S. Treasury securities $ 165,959 $ 2 $ (13 ) $ 165,948 Financial institution debt securities 65,436 1 (17 ) 65,420 Corporate debt securities 23,836 - (1 ) 23,835 Total $ 255,231 $ 3 $ (31 ) $ 255,203 |
Summary of Changes in Contract Liabilities | The following table presents changes in the Company’s contract liabilities during the six months ended June 30, 2019 and 2018 (in thousands): Balance at Beginning of Period Additions Deductions Balance at End of Period Six Months Ended June 30, 2019 Contract liabilities: Deferred revenue $ 55,932 $ 2,000 $ (15,772 ) $ 42,160 Balance at Beginning of Period Additions Deductions Balance at End of Period Six Months Ended June 30, 2018 Contract liabilities: Deferred revenue $ 59,868 $ 2,000 $ (11,009 ) $ 50,859 |
Summary of Revenues Recognized Resulting From Changes in Contract Liability Balance | During the six months ended June 30, 2019 and 2018, the Company recognized the following revenues as a result of changes in the contract liability balance (in thousands): Revenue recognized in the period from: Six Months Ended June 30, 2019 Six Months Ended June 30, 2018 Amounts included in the contract liability at the beginning of the period $ 15,772 $ 11,009 |
Fair Value Measurements (Tables
Fair Value Measurements (Tables) | 6 Months Ended |
Jun. 30, 2019 | |
Fair Value Disclosures [Abstract] | |
Summary of Financial Assets Recognized at Fair Value on Recurring Basis | As of June 30, 2019 and December 31, 2018, the Company’s financial assets recognized at fair value on a recurring basis consisted of the following: Fair Value as of June 30, 2019 Total Level 1 Level 2 Level 3 (In thousands) Cash equivalents $ 45,799 $ 45,799 $ - $ - Marketable securities: U.S. Treasury securities 146,713 146,713 - - Financial institution debt securities 53,110 - 53,110 - Corporate debt securities 18,892 - 18,892 - Other asset-backed securities 7,983 - 7,983 - Total marketable securities 226,698 146,713 79,985 - Total $ 272,497 $ 192,512 $ 79,985 $ - Fair Value as of December 31, 2018 Total Level 1 Level 2 Level 3 (In thousands) Cash equivalents $ 45,986 $ 45,986 $ - $ - Marketable securities: U.S. Treasury securities 165,948 165,948 - - Financial institution debt securities 65,420 - 65,420 - Corporate debt securities 23,835 - 23,835 - Total marketable securities 255,203 165,948 89,255 - Total $ 301,189 $ 211,934 $ 89,255 $ - |
Accrued Expenses (Tables)
Accrued Expenses (Tables) | 6 Months Ended |
Jun. 30, 2019 | |
Payables And Accruals [Abstract] | |
Schedule of Accrued Expenses | Accrued expenses consisted of the following: June 30, 2019 December 31, 2018 (In thousands) Accrued legal and professional expenses $ 4,837 $ 1,633 Accrued employee compensation and benefits 3,803 6,175 Accrued research and development 2,485 2,328 Accrued other 773 606 Total accrued expenses $ 11,898 $ 10,742 |
Leases (Tables)
Leases (Tables) | 6 Months Ended |
Jun. 30, 2019 | |
Leases [Abstract] | |
Summary of Lease Costs and Other Information | The following table contains a summary of the lease costs recognized under Topic 842 and other information pertaining to the Company’s operating leases for the three and six months ended June 30, 2019: Three Months Ended Six Months Ended June 30, 2019 (in thousands) Lease cost Operating lease cost $ 1,880 $ 3,583 Variable lease cost 460 997 Total lease cost $ 2,340 $ 4,580 Other information Operating cash flows used for operating leases $ 1,635 $ 3,122 Operating lease liabilities arising from obtaining right-of-use assets 1,343 1,343 Weighted average remaining lease term 3.5 years 3.5 years Weighted average discount rate 9.00 % 9.00 % |
Schedule of Future Minimum Lease Payments | Future minimum lease payments under the Company’s non-cancelable operating leases as of June 30, 2019, are as follows: Future Operating Lease Payments Year Ending December 31, (in thousands) 2019 (excluding the six months ended June 30, 2019) $ 3,355 2020 6,483 2021 6,618 2022 4,732 2023 871 Thereafter 943 Total lease payments $ 23,002 Less: imputed interest (3,333 ) Less: leases not yet commenced - Total operating lease liabilities at June 30, 2019 $ 19,669 Future minimum lease payments under the Company’s non-cancelable operating leases as of December 31, 2018, are as follows: Year Ending December 31, (In 2019 $ 5,616 2020 4,963 2021 5,507 2022 3,861 Thereafter - $ 19,947 |
Equity-Based Compensation (Tabl
Equity-Based Compensation (Tables) | 6 Months Ended |
Jun. 30, 2019 | |
Disclosure Of Compensation Related Costs Sharebased Payments [Abstract] | |
Schedule of Equity-Based Compensation Expense | Equity-based compensation expense is classified in the condensed consolidated statements of operations and comprehensive loss as follows: Three Months Ended June 30, Six Months Ended June 30, 2019 2018 2019 2018 (In thousands) Research and development $ 2,096 $ 2,633 $ 3,879 $ 5,026 General and administrative 2,308 2,268 5,117 3,982 Total $ 4,404 $ 4,901 $ 8,996 $ 9,008 |
Summary of Restricted Stock Activity | The following table summarizes the Company’s restricted stock activity for the six months ended June 30, 2019: Number of Shares Weighted Average Grant Date Fair Value per Share Unvested restricted stock as of December 31, 2018 109,073 $ 15.53 Granted - - Vested (36,462 ) 1.34 Cancelled - - Unvested restricted stock as of June 30, 2019 72,611 $ 22.66 |
Summary of Weighted Average Assumptions Used to Compute Fair Value of Option Granted | Key assumptions used to apply this pricing model were as follows: Three Months Ended June 30, Six Months Ended June 30, 2019 2018 2019 2018 Risk-free interest rate 2.1% 2.8% 2.4% 2.6% Expected life of options 5.5-6.0 years 5.5-6.25 years 5.5-6.0 years 5.5-6.25 years Expected volatility of underlying stock 67.9% 87.7% 68.6% 89.0% Expected dividend yield 0.0% 0.0% 0.0% 0.0% |
Summary of Stock Option Activity | The following is a summary of stock option activity for the six months ended June 30, 2019: Number of Options Weighted Average Exercise Price per Share Weighted Average Remaining Contractual Term Aggregate Intrinsic Value (In years) (In thousands) Outstanding at December 31, 2018 5,037,663 $ 15.63 Granted 793,813 14.54 Exercised (233,872 ) 8.65 Forfeited (282,108 ) 18.79 Outstanding at June 30, 2019 5,315,496 $ 15.61 8.14 $ 14,978 Exercisable at June 30, 2019 2,165,526 $ 13.43 7.01 $ 10,193 |
Loss Per Share (Tables)
Loss Per Share (Tables) | 6 Months Ended |
Jun. 30, 2019 | |
Earnings Per Share [Abstract] | |
Schedule of Basic and Diluted Net Loss Per Share | Basic and diluted loss per share was calculated as follows: Three Months Ended June 30, Six Months Ended June 30, 2019 2018 2019 2018 (In thousands) Net loss $ (25,683 ) $ (22,219 ) $ (47,623 ) $ (43,575 ) Weighted average shares outstanding, basic and diluted 45,814 42,836 45,526 42,441 Net loss per share, basic and diluted $ (0.56 ) $ (0.52 ) $ (1.05 ) $ (1.03 ) |
Potential Dilutive Securities Excluded from Computation of Diluted Net Loss Per Common Share | The following common stock equivalents were excluded from the calculation of diluted loss per share because their inclusion would have been anti-dilutive: Six Months Ended June 30, 2019 2018 (In thousands) Unvested restricted stock 73 261 Stock options 5,315 4,881 5,388 5,142 |
Stockholders_ Equity (Tables)
Stockholders’ Equity (Tables) | 6 Months Ended |
Jun. 30, 2019 | |
Equity [Abstract] | |
Schedule of Changes in Stockholders’ Equity | The following tables present changes in stockholders’ equity for the six-month periods ended June 30, 2019 and 2018 (in thousands, except share data): Additional Accumulated Other Total Common Paid-In Comprehensive Accumulated Stockholders’ Shares Amount Capital Income (Loss) Deficit Equity (Deficit) Balance at December 31, 2018 45,224,480 $ 5 $ 478,968 $ (28 ) $ (201,025 ) $ 277,920 Retroactive adjustment to beginning accumulated deficit for adoption of ASC 842 - - - - (320 ) (320 ) Issuance of common stock through at-the-market offerings, net of issuance costs of $120 223,818 - 3,639 - - 3,639 Exercise of stock options 30,800 - 360 - - 360 Equity-based compensation - - 4,592 - - 4,592 Other comprehensive gain - - - 87 - 87 Net loss - - - - (21,940 ) (21,940 ) Balance at March 31, 2019 45,479,098 $ 5 $ 487,559 $ 59 $ (223,285 ) $ 264,338 Issuance of common stock through at-the-market offerings, net of issuance costs of $3 1,986,579 - 31,413 - - 31,413 Exercise of stock options 203,072 - 1,664 - - 1,664 Issuance of shares under employee stock purchase plan 45,826 - 534 - - 534 Equity-based compensation - - 4,404 - - 4,404 Other comprehensive gain - - - 196 - 196 Net loss - - - - (25,683 ) (25,683 ) Balance at June 30, 2019 47,714,575 $ 5 $ 525,574 $ 255 $ (248,968 ) $ 276,866 Additional Accumulated Other Total Common Paid-In Comprehensive Accumulated Stockholders’ Shares Amount Capital Income (Loss) Deficit Equity (Deficit) Balance at December 31, 2017 42,384,623 $ 4 $ 421,706 $ - $ (121,113 ) $ 300,597 Retroactive adjustment to beginning accumulated deficit for adoption of ASU 2014-09 - - - - 5,431 5,431 Exercise of stock options 709,321 - 6,720 - - 6,720 Cancellation of restricted stock (2,022 ) - - - - - Equity-based compensation - - 4,107 - - 4,107 Net loss - - - - (21,356 ) (21,356 ) Balance at March 31, 2018 43,091,922 $ 4 $ 432,533 $ - $ (137,038 ) $ 295,499 Exercise of stock options 68,163 - 557 - - 557 Issuance of shares under employee stock purchase plan 32,707 - 598 - - 598 Cancellation of restricted stock (4,780 ) - - - - - Equity-based compensation - - 4,901 - - 4,901 Net loss - - - - (22,219 ) (22,219 ) Balance at June 30, 2018 43,188,012 $ 4 $ 438,589 $ - $ (159,257 ) $ 279,336 |
Overview and Basis of Present_2
Overview and Basis of Presentation - Additional Information (Detail) - USD ($) $ / shares in Units, $ in Thousands | Jul. 02, 2019 | Oct. 12, 2018 | Nov. 30, 2018 | Jun. 30, 2019 | Jun. 30, 2019 |
Organization Consolidation And Presentation Of Financial Statements Disclosure [Line Items] | |||||
Proceeds from common stock offering | $ 7,912 | $ 580,200 | |||
Proceeds from issuance of convertible preferred stock | 85,000 | ||||
Proceeds due from at-the-market offerings | 27,140 | 27,140 | |||
Collaborative Arrangement [Member] | |||||
Organization Consolidation And Presentation Of Financial Statements Disclosure [Line Items] | |||||
Proceeds from common stock offering | $ 146,700 | ||||
Sales Agreement [Member] | |||||
Organization Consolidation And Presentation Of Financial Statements Disclosure [Line Items] | |||||
Proceeds from common stock offering | $ 28,500 | $ 35,200 | |||
Percentage of gross proceeds from common stock as sales agent cash commission | 3.00% | 3.00% | 3.00% | ||
Number of common stock issued upon conversion of shares | 1,659,300 | 2,210,397 | |||
Common stock price per share | $ 18 | $ 16.41 | $ 16.41 | ||
Sales Agreement [Member] | General and Administrative Expenses [Member] | |||||
Organization Consolidation And Presentation Of Financial Statements Disclosure [Line Items] | |||||
Legal accounting and other fees | $ 400 | $ 100 | |||
Sales Agreement [Member] | Maximum [Member] | |||||
Organization Consolidation And Presentation Of Financial Statements Disclosure [Line Items] | |||||
Proceeds from common stock offering | $ 100,000 | ||||
Initial Public Offering and Concurrent Private Placements [Member] | |||||
Organization Consolidation And Presentation Of Financial Statements Disclosure [Line Items] | |||||
Proceeds from common stock offering | $ 170,500 | ||||
Follow-on public Offering [Member] | |||||
Organization Consolidation And Presentation Of Financial Statements Disclosure [Line Items] | |||||
Proceeds from common stock offering | 141,000 | ||||
At-the-market Offering [Member] | |||||
Organization Consolidation And Presentation Of Financial Statements Disclosure [Line Items] | |||||
Proceeds from common stock offering | $ 37,000 | ||||
At-the-market Offering [Member] | Subsequent Event [Member] | |||||
Organization Consolidation And Presentation Of Financial Statements Disclosure [Line Items] | |||||
Proceeds from common stock offering | $ 27,100 |
Summary of Significant Accoun_4
Summary of Significant Accounting Policies - Summary of Available -for-sale Marketable Securities (Detail) - USD ($) $ in Thousands | Jun. 30, 2019 | Dec. 31, 2018 |
Marketable Securities [Line Items] | ||
Amortized Cost | $ 226,443 | $ 255,231 |
Gross Unrealized Gains | 255 | 3 |
Gross Unrealized Losses | (31) | |
Estimated Fair Value | 226,698 | 255,203 |
U.S. Treasury Securities [Member] | ||
Marketable Securities [Line Items] | ||
Amortized Cost | 146,552 | 165,959 |
Gross Unrealized Gains | 161 | 2 |
Gross Unrealized Losses | (13) | |
Estimated Fair Value | 146,713 | 165,948 |
Financial Institution Debt Securities [Member] | ||
Marketable Securities [Line Items] | ||
Amortized Cost | 53,041 | 65,436 |
Gross Unrealized Gains | 69 | 1 |
Gross Unrealized Losses | (17) | |
Estimated Fair Value | 53,110 | 65,420 |
Corporate Debt Securities [Member] | ||
Marketable Securities [Line Items] | ||
Amortized Cost | 18,892 | 23,836 |
Gross Unrealized Losses | (1) | |
Estimated Fair Value | 18,892 | $ 23,835 |
Other Asset Backed Securities [Member] | ||
Marketable Securities [Line Items] | ||
Amortized Cost | 7,958 | |
Gross Unrealized Gains | 25 | |
Estimated Fair Value | $ 7,983 |
Summary of Significant Accoun_5
Summary of Significant Accounting Policies - Additional Information (Detail) - USD ($) | 6 Months Ended | ||
Jun. 30, 2019 | Jan. 01, 2019 | Dec. 31, 2018 | |
Summary Of Significant Accounting Policies [Line Items] | |||
Realized loss on marketable securities | $ 0 | ||
Expenses incurred to obtain collaboration agreements and costs to fulfill contracts | 0 | ||
Costs to obtain or fulfill contract capitalized | 0 | ||
Operating lease, liability | 19,669,000 | ||
Operating lease right-of-use assets | 20,923,000 | ||
Retained earnings | $ (248,968,000) | $ (201,025,000) | |
ASU 2016-02 [Member] | |||
Summary Of Significant Accounting Policies [Line Items] | |||
Operating lease, liability | $ 20,600,000 | ||
Operating lease right-of-use assets | 22,300,000 | ||
ASU 2016-02 [Member] | Restatement Adjustment [Member] | |||
Summary Of Significant Accounting Policies [Line Items] | |||
Retained earnings | $ 300,000 |
Summary of Significant Accoun_6
Summary of Significant Accounting Policies - Summary of Changes in Contract Liabilities (Detail) - USD ($) $ in Thousands | 6 Months Ended | |
Jun. 30, 2019 | Jun. 30, 2018 | |
Contract liabilities: | ||
Deferred revenue, Balance at Beginning of Period | $ 55,932 | $ 59,868 |
Deferred revenue, Additions | 2,000 | 2,000 |
Deferred revenue, Deductions | (15,772) | (11,009) |
Deferred revenue, Balance at End of Period | $ 42,160 | $ 50,859 |
Summary of Significant Accoun_7
Summary of Significant Accounting Policies - Summary of Revenues Recognized Resulting From Changes in Contract Liability Balance (Detail) - USD ($) $ in Thousands | 6 Months Ended | |
Jun. 30, 2019 | Jun. 30, 2018 | |
Revenue From Contract With Customer [Abstract] | ||
Amounts included in the contract liability at the beginning of the period | $ 15,772 | $ 11,009 |
Fair Value Measurements - Summa
Fair Value Measurements - Summary of Financial Assets Recognized at Fair Value on Recurring Basis (Detail) - Fair Value on Recurring Basis [Member] - USD ($) $ in Thousands | Jun. 30, 2019 | Dec. 31, 2018 |
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Cash equivalents | $ 45,799 | $ 45,986 |
Marketable securities: | ||
Marketable securities | 226,698 | 255,203 |
Total | 272,497 | 301,189 |
U.S. Treasury Securities [Member] | ||
Marketable securities: | ||
Marketable securities | 146,713 | 165,948 |
Financial Institution Debt Securities [Member] | ||
Marketable securities: | ||
Marketable securities | 53,110 | 65,420 |
Corporate Debt Securities [Member] | ||
Marketable securities: | ||
Marketable securities | 18,892 | 23,835 |
Other Asset Backed Securities [Member] | ||
Marketable securities: | ||
Marketable securities | 7,983 | |
Level 1 [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Cash equivalents | 45,799 | 45,986 |
Marketable securities: | ||
Marketable securities | 146,713 | 165,948 |
Total | 192,512 | 211,934 |
Level 1 [Member] | U.S. Treasury Securities [Member] | ||
Marketable securities: | ||
Marketable securities | 146,713 | 165,948 |
Level 2 [Member] | ||
Marketable securities: | ||
Marketable securities | 79,985 | 89,255 |
Total | 79,985 | 89,255 |
Level 2 [Member] | Financial Institution Debt Securities [Member] | ||
Marketable securities: | ||
Marketable securities | 53,110 | 65,420 |
Level 2 [Member] | Corporate Debt Securities [Member] | ||
Marketable securities: | ||
Marketable securities | 18,892 | $ 23,835 |
Level 2 [Member] | Other Asset Backed Securities [Member] | ||
Marketable securities: | ||
Marketable securities | $ 7,983 |
Accrued Expenses - Schedule of
Accrued Expenses - Schedule of Accrued Expenses (Detail) - USD ($) $ in Thousands | Jun. 30, 2019 | Dec. 31, 2018 |
Payables And Accruals [Abstract] | ||
Accrued legal and professional expenses | $ 4,837 | $ 1,633 |
Accrued employee compensation and benefits | 3,803 | 6,175 |
Accrued research and development | 2,485 | 2,328 |
Accrued other | 773 | 606 |
Total accrued expenses | $ 11,898 | $ 10,742 |
Collaborations - Additional Inf
Collaborations - Additional Information (Detail) | 1 Months Ended | 3 Months Ended | 6 Months Ended | 12 Months Ended | 39 Months Ended | 54 Months Ended | ||||||
Jul. 31, 2018USD ($)shares | Apr. 30, 2016USD ($) | Jan. 31, 2015USD ($) | Jun. 30, 2019USD ($) | Jun. 30, 2018USD ($) | Jun. 30, 2019USD ($)PerformanceObligation | Jun. 30, 2018USD ($) | Dec. 31, 2019USD ($) | Jun. 30, 2019USD ($) | Jun. 30, 2019USD ($) | Dec. 31, 2018USD ($) | Dec. 31, 2017USD ($) | |
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | ||||||||||||
Collaboration revenue | $ 11,118,000 | $ 7,677,000 | $ 21,551,000 | $ 15,146,000 | ||||||||
Deferred revenue | 42,160,000 | 50,859,000 | 42,160,000 | 50,859,000 | $ 42,160,000 | $ 42,160,000 | $ 55,932,000 | $ 59,868,000 | ||||
Novartis [Member] | ||||||||||||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | ||||||||||||
Additional payments received on each target | 1,000,000 | |||||||||||
Estimated fair value of units | $ 11,600,000 | |||||||||||
Difference between cash proceeds received and estimated fair value of preferred units | 2,600,000 | |||||||||||
Novartis [Member] | Maximum [Member] | ||||||||||||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | ||||||||||||
Development based milestone payments under agreement | 30,300,000 | |||||||||||
Regulatory based milestone payments for first indication | 50,000,000 | |||||||||||
Regulatory based milestone payments for second indication | 50,000,000 | |||||||||||
Sales based milestone payments under agreement | 100,000,000 | |||||||||||
Novartis [Member] | 2014 Novartis Agreement [Member] | ||||||||||||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | ||||||||||||
Deferred revenue additions | 10,000,000 | 10,000,000 | ||||||||||
Technology access fees | 20,000,000 | 20,000,000 | 20,000,000 | 20,000,000 | 20,000,000 | |||||||
Quarterly research payments | $ 1,000,000 | 20,000,000 | 20,000,000 | 20,000,000 | 20,000,000 | |||||||
Research Payments | 17,000,000 | 17,000,000 | 17,000,000 | 17,000,000 | ||||||||
Research term | 5 years | |||||||||||
Proceeds from issuance of preferred stock | $ 9,000,000 | |||||||||||
One time collaboration payment | 10,000,000 | |||||||||||
Termination period of agreement | 90 days | |||||||||||
Number of performance obligations | PerformanceObligation | 2 | |||||||||||
Transaction price | $ 59,000,000 | |||||||||||
Transaction price allocated to preferred units purchased at fair value | 11,600,000 | |||||||||||
Novartis [Member] | 2014 Novartis Agreement [Member] | HSC and CAR-T Cell Products [Member] | ||||||||||||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | ||||||||||||
Remaining transaction price allocated to combined performance obligation of licenses and associated research activities | $ 47,400,000 | |||||||||||
Combined performance obligation of licenses and associated research activities, revenue recognition period | 5 years | |||||||||||
Novartis [Member] | 2014 Novartis Agreement [Member] | LSCs [Member] | ||||||||||||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | ||||||||||||
Number of performance obligations | PerformanceObligation | 1 | |||||||||||
Transaction price | $ 10,000,000 | |||||||||||
Combined performance obligation of licenses and associated research activities, revenue recognition period | 1 year | |||||||||||
Novartis [Member] | 2014 Novartis Agreement [Member] | Scenario, Forecast [Member] | ||||||||||||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | ||||||||||||
Internalization fee | $ 50,000,000 | |||||||||||
Period allowed for editing limited number of platform targets | 5 years | |||||||||||
Novartis [Member] | 2014 Novartis Agreement [Member] | Maximum [Member] | ||||||||||||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | ||||||||||||
Total research funding | $ 20,000,000 | |||||||||||
Novartis [Member] | 2014 Novartis Agreement [Member] | Maximum [Member] | Scenario, Forecast [Member] | ||||||||||||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | ||||||||||||
Internalization fee milestone payment | $ 20,000,000 | |||||||||||
Milestone payment for additional post-internalization targets | $ 4,000,000 | |||||||||||
Novartis [Member] | Unit Purchase Agreement [Member] | ||||||||||||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | ||||||||||||
Proceeds from issuance of preferred stock | $ 9,000,000 | |||||||||||
Novartis [Member] | Novartis Arrangement [Member] | ||||||||||||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | ||||||||||||
Deferred revenue additions | 55,400,000 | |||||||||||
Collaboration revenue | 4,800,000 | 2,400,000 | $ 9,500,000 | 4,700,000 | 48,400,000 | |||||||
Aggregate transaction price remaining to be recognized | 9,000,000 | $ 9,000,000 | 9,000,000 | 9,000,000 | ||||||||
Aggregate transaction price remaining to be recognized, period | through December 2019 | |||||||||||
Accounts receivable | 1,000,000 | $ 1,000,000 | 1,000,000 | 1,000,000 | 6,000,000 | |||||||
Deferred revenue | 7,000,000 | $ 7,000,000 | 7,000,000 | 7,000,000 | 14,500,000 | |||||||
Regeneron Pharmaceuticals Inc. [Member] | Regeneron Agreement [Member] | ||||||||||||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | ||||||||||||
Deferred revenue additions | $ 75,000,000 | |||||||||||
One time collaboration payment | $ 25,000,000 | |||||||||||
Termination period of agreement | 180 days | |||||||||||
Number of performance obligations | PerformanceObligation | 3 | |||||||||||
Percentage of certain research and development costs to be funded | 50.00% | |||||||||||
Collaboration term extension period | 2 years | |||||||||||
Royalty payment obligation expiration period | 12 years | |||||||||||
Transaction price allocated to common stock | $ 50,000,000 | |||||||||||
Remaining transaction price allocated to combined performance obligation | 75,000,000 | |||||||||||
Amount allocated to licenses to targets and associated research activities and evaluation plans | 63,800,000 | |||||||||||
Amount allocated to technology collaboration and associated research activities | $ 11,200,000 | |||||||||||
Licenses to targets and associated research activities and evaluation plans performance period | 6 years | |||||||||||
Regeneron Pharmaceuticals Inc. [Member] | Regeneron Agreement [Member] | Maximum [Member] | ||||||||||||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | ||||||||||||
Development based milestone payments under agreement | $ 25,000,000 | |||||||||||
Sales based milestone payments under agreement | 185,000,000 | |||||||||||
Regulatory based milestone payments under agreement | 110,000,000 | |||||||||||
Regeneron Pharmaceuticals Inc. [Member] | Stock Purchase Agreement [Member] | ||||||||||||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | ||||||||||||
Purchase of common stock through private placement | $ 50,000,000 | |||||||||||
Regeneron Pharmaceuticals Inc. [Member] | Co-Development and Co-Promotion Agreement Member [Member] | ||||||||||||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | ||||||||||||
Termination period of agreement | 180 days | |||||||||||
Compensation for prior work | $ 1,500,000 | |||||||||||
Payment of royalty percentage on net product sales | 50.00% | |||||||||||
Regeneron Pharmaceuticals Inc. [Member] | Co-Development and Co-Promotion Agreement Member [Member] | Minimum [Member] | ||||||||||||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | ||||||||||||
Options exercisable, number of shares exercisable | shares | 5 | |||||||||||
Collaborative arrangement obligation to be fund in development costs | $ 5,000,000 | |||||||||||
Regeneron Pharmaceuticals Inc. [Member] | Regeneron Arrangement | ||||||||||||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | ||||||||||||
Deferred revenue additions | $ 75,000,000 | 75,000,000 | ||||||||||
Transaction price | 125,000,000 | |||||||||||
Collaboration revenue | 6,300,000 | 5,300,000 | 12,000,000 | 10,400,000 | 57,700,000 | |||||||
Aggregate transaction price remaining to be recognized | 35,200,000 | $ 35,200,000 | 35,200,000 | 35,200,000 | ||||||||
Aggregate transaction price remaining to be recognized, period | through April 2022 | |||||||||||
Deferred revenue | 35,200,000 | $ 35,200,000 | 35,200,000 | 35,200,000 | 41,400,000 | |||||||
Purchase of common stock through private placement | 50,000,000 | |||||||||||
Payments due | 3,200,000 | $ 2,100,000 | 5,800,000 | $ 4,100,000 | ||||||||
Accounts receivable | $ 3,200,000 | $ 3,200,000 | 3,200,000 | $ 3,200,000 | $ 1,500,000 | |||||||
Regeneron Pharmaceuticals Inc. [Member] | Regeneron Arrangement | Research and Development Services [Member] | ||||||||||||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | ||||||||||||
Collaboration revenue | $ 17,900,000 |
Leases - Additional Information
Leases - Additional Information (Detail) - USD ($) $ in Thousands | 1 Months Ended | 6 Months Ended | ||||
Apr. 30, 2019 | Mar. 31, 2019 | Jan. 31, 2016 | Oct. 31, 2014 | Jun. 30, 2019 | Dec. 31, 2018 | |
Lessee Lease Description [Line Items] | ||||||
Operating lease right-of-use assets | $ 20,923 | |||||
Operating lease, liability | $ 19,669 | |||||
130 Brookline Street [Member] | ||||||
Lessee Lease Description [Line Items] | ||||||
Operating lease, description | In October 2014, the Company entered into an agreement to lease office and laboratory space at 130 Brookline Street in Cambridge, Massachusetts under an operating lease agreement with a term through January 2020, with an option to extend the term of the lease for an additional five-year period. In April 2019, the Company executed an amendment to the lease to extend the term of the lease for the additional five-year period, through January 2025. | |||||
Operating lease expiration | 2020-01 | |||||
Lessee operating lease extended expiration date | Jan. 31, 2025 | |||||
Operating lease, existence of option to extend | true | |||||
Operating lease, options to extend | option to extend the term of the lease for an additional five-year period. In April 2019, the Company executed an amendment to the lease to extend the term of the lease for the additional five-year period, through January 2025. | |||||
Operating lease, renewal term | 5 years | 5 years | ||||
Lessee operating sublease option to extend | two options to extend the agreement by one year each, for a total option period of up to two years. | |||||
Operating sublease expiration | 2021-04 | |||||
Operating sublease, existence of option to extend | true | |||||
Operating lease right-of-use assets | $ 1,300 | |||||
Operating lease, liability | $ 1,300 | |||||
130 Brookline Street [Member] | Maximum [Member] | ||||||
Lessee Lease Description [Line Items] | ||||||
Operating sublease, renewal term | 2 years | |||||
130 Brookline Street [Member] | Other Assets [Member] | ||||||
Lessee Lease Description [Line Items] | ||||||
Lease security deposit | $ 300 | |||||
40 Erie Street [Member] | ||||||
Lessee Lease Description [Line Items] | ||||||
Operating lease, description | In January 2016, the Company entered into a ten-year agreement to lease office and laboratory space at 40 Erie Street in Cambridge, Massachusetts under an operating lease agreement, with an option to terminate the lease at the end of the sixth year and an option to extend the term of the lease for an additional three years. | |||||
Operating lease, existence of option to extend | true | |||||
Operating lease, options to extend | option to extend the term of the lease for an additional three years. | |||||
Operating lease, renewal term | 3 years | |||||
Operating lease, term of contract | 10 years | |||||
Operating lease, existence of option to terminate | true | |||||
Operating lease, option to terminate | option to terminate the lease at the end of the sixth year | |||||
Operating lease, terminate term | 6 years | |||||
Prepaid lease | $ 2,200 | |||||
40 Erie Street [Member] | Other Assets [Member] | ||||||
Lessee Lease Description [Line Items] | ||||||
Lease security deposit | $ 2,200 |
Leases - Summary of Lease Costs
Leases - Summary of Lease Costs and Other Information (Detail) $ in Thousands | 3 Months Ended | 6 Months Ended |
Jun. 30, 2019USD ($) | Jun. 30, 2019USD ($) | |
Lease cost | ||
Operating lease cost | $ 1,880 | $ 3,583 |
Variable lease cost | 460 | 997 |
Total lease cost | 2,340 | 4,580 |
Other information | ||
Operating cash flows used for operating leases | 1,635 | 3,122 |
Operating lease liabilities arising from obtaining right-of-use assets | $ 1,343 | $ 1,343 |
Weighted average remaining lease term | 3 years 6 months | 3 years 6 months |
Weighted average discount rate | 9.00% | 9.00% |
Leases - Schedule of Future Min
Leases - Schedule of Future Minimum Lease Payments (Detail) - USD ($) $ in Thousands | Jun. 30, 2019 | Dec. 31, 2018 |
Leases [Abstract] | ||
2019 (excluding the six months ended June 30, 2019) | $ 3,355 | |
2019 | $ 5,616 | |
2020 | 6,483 | 4,963 |
2021 | 6,618 | 5,507 |
2022 | 4,732 | 3,861 |
2023 | 871 | |
Thereafter | 943 | |
Total lease payments | 23,002 | $ 19,947 |
Less: imputed interest | (3,333) | |
Operating lease, liability | $ 19,669 |
Equity-Based Compensation - Sch
Equity-Based Compensation - Schedule of Equity-Based Compensation Expense (Detail) - USD ($) $ in Thousands | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2019 | Jun. 30, 2018 | Jun. 30, 2019 | Jun. 30, 2018 | |
Share-based Compensation Arrangement by Share-based Payment Award, Compensation Cost [Line Items] | ||||
Equity-based compensation expense | $ 4,404 | $ 4,901 | $ 8,996 | $ 9,008 |
Research and Development [Member] | ||||
Share-based Compensation Arrangement by Share-based Payment Award, Compensation Cost [Line Items] | ||||
Equity-based compensation expense | 2,096 | 2,633 | 3,879 | 5,026 |
General and Administrative [Member] | ||||
Share-based Compensation Arrangement by Share-based Payment Award, Compensation Cost [Line Items] | ||||
Equity-based compensation expense | $ 2,308 | $ 2,268 | $ 5,117 | $ 3,982 |
Equity-Based Compensation - Sum
Equity-Based Compensation - Summary of Restricted Stock Activity (Detail) | 6 Months Ended |
Jun. 30, 2019$ / sharesshares | |
Disclosure Of Compensation Related Costs Sharebased Payments [Abstract] | |
Number of Shares, Unvested, Beginning balance | shares | 109,073 |
Number of Shares, Vested | shares | (36,462) |
Number of Shares, Unvested, Ending balance | shares | 72,611 |
Weighted Average Grant Date Fair Value per Share, Unvested, Beginning balance | $ / shares | $ 15.53 |
Weighted Average Grant Date Fair Value per Share, Vested | $ / shares | 1.34 |
Weighted Average Grant Date Fair Value per Share, Unvested, Ending balance | $ / shares | $ 22.66 |
Equity-Based Compensation - Add
Equity-Based Compensation - Additional Information (Detail) - USD ($) $ / shares in Units, $ in Millions | 3 Months Ended | 6 Months Ended | |||
Jun. 30, 2019 | Jun. 30, 2018 | Jun. 30, 2019 | Jun. 30, 2018 | Dec. 31, 2018 | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||
Unrecognized equity-based compensation expense related to restricted stock | $ 0.8 | $ 0.8 | |||
Weighted average grant date fair value per share | $ 9.22 | $ 16.64 | $ 9.07 | $ 17.94 | |
Unrecognized compensation cost related to stock options | $ 34.1 | $ 34.1 | |||
Unvested restricted stock outstanding | 72,611 | 72,611 | 109,073 | ||
Stock options outstanding | 5,315,496 | 5,315,496 | 5,037,663 | ||
Vesting description | The performance-based restricted stock units and performance-based stock options vest upon obtaining certain scientific, financial and regulatory milestones through 2020 | ||||
Potential dilutive securities excluded from computation of diluted net loss per common share | 5,388,000 | 5,142,000 | |||
Restricted Stock [Member] | |||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||
Weighted average period of unrecognized compensation costs | 1 year 4 months 24 days | ||||
Stock Options [Member] | |||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||
Weighted average period of unrecognized compensation costs | 2 years 7 months 6 days | ||||
Performance Based Restricted Stock Units [Member] | |||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||
Unvested restricted stock outstanding | 71,875 | 71,875 | |||
Potential dilutive securities excluded from computation of diluted net loss per common share | 71,875 | ||||
Performance Based Stock Options [Member] | |||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||
Stock options outstanding | 213,750 | 213,750 | |||
Potential dilutive securities excluded from computation of diluted net loss per common share | 188,750 |
Equity-Based Compensation - S_2
Equity-Based Compensation - Summary of Weighted Average Assumptions Used to Compute Fair Value of Option Granted (Detail) | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2019 | Jun. 30, 2018 | Jun. 30, 2019 | Jun. 30, 2018 | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Risk-free interest rate | 2.10% | 2.80% | 2.40% | 2.60% |
Expected volatility of underlying stock | 67.90% | 87.70% | 68.60% | 89.00% |
Expected dividend yield | 0.00% | 0.00% | 0.00% | 0.00% |
Minimum [Member] | ||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Expected life of options | 5 years 6 months | 5 years 6 months | 5 years 6 months | 5 years 6 months |
Maximum [Member] | ||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Expected life of options | 6 years | 6 years 3 months | 6 years | 6 years 3 months |
Equity-Based Compensation - S_3
Equity-Based Compensation - Summary of Stock Option Activity (Detail) - USD ($) $ / shares in Units, $ in Thousands | 6 Months Ended |
Jun. 30, 2019 | |
Disclosure Of Compensation Related Costs Sharebased Payments [Abstract] | |
Number of Options, Outstanding, Beginning Balance | 5,037,663 |
Number of options, Granted | 793,813 |
Number of options, Exercised | (233,872) |
Number of options, Forfeited | (282,108) |
Number of Options, Outstanding, Ending Balance | 5,315,496 |
Number of Options, Exercisable | 2,165,526 |
Weighted Average Exercise Price per Share, Outstanding, Beginning Balance | $ 15.63 |
Weighted Average Exercise Price per Share, Granted | 14.54 |
Weighted Average Exercise Price per Share, Exercised | 8.65 |
Weighted Average Exercise Price per Share, Forfeited | 18.79 |
Weighted Average Exercise Price per Share, Outstanding, Ending Balance | 15.61 |
Weighted Average Exercise Price per Share, Exercisable | $ 13.43 |
Weighted Average Remaining Contractual Term, Outstanding | 8 years 1 month 20 days |
Weighted Average Remaining Contractual Term, Exercisable | 7 years 3 days |
Aggregate Intrinsic Value, Outstanding | $ 14,978 |
Aggregate Intrinsic Value, Exercisable | $ 10,193 |
Loss Per Share - Schedule of Ba
Loss Per Share - Schedule of Basic and Diluted Net Loss Per Share (Detail) - USD ($) $ / shares in Units, shares in Thousands, $ in Thousands | 3 Months Ended | 6 Months Ended | ||||
Jun. 30, 2019 | Mar. 31, 2019 | Jun. 30, 2018 | Mar. 31, 2018 | Jun. 30, 2019 | Jun. 30, 2018 | |
Earnings Per Share [Abstract] | ||||||
Net loss | $ (25,683) | $ (21,940) | $ (22,219) | $ (21,356) | $ (47,623) | $ (43,575) |
Weighted average shares outstanding, basic and diluted | 45,814 | 42,836 | 45,526 | 42,441 | ||
Net loss per share, basic and diluted | $ (0.56) | $ (0.52) | $ (1.05) | $ (1.03) |
Loss Per Share - Potential Dilu
Loss Per Share - Potential Dilutive Securities Excluded from Computation of Diluted Net Loss Per Common Share (Detail) - shares shares in Thousands | 6 Months Ended | |
Jun. 30, 2019 | Jun. 30, 2018 | |
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | ||
Potential dilutive securities excluded from computation of diluted net loss per common share | 5,388 | 5,142 |
Unvested Restricted Stock [Member] | ||
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | ||
Potential dilutive securities excluded from computation of diluted net loss per common share | 73 | 261 |
Stock Options [Member] | ||
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | ||
Potential dilutive securities excluded from computation of diluted net loss per common share | 5,315 | 4,881 |
Stockholders Equity - Schedule
Stockholders Equity - Schedule of Changes in Stockholders Equity (Detail) - USD ($) $ in Thousands | 3 Months Ended | 6 Months Ended | ||||
Jun. 30, 2019 | Mar. 31, 2019 | Jun. 30, 2018 | Mar. 31, 2018 | Jun. 30, 2019 | Jun. 30, 2018 | |
Class Of Stock [Line Items] | ||||||
Beginning balance | $ 264,338 | $ 277,920 | $ 295,499 | $ 300,597 | $ 277,920 | $ 300,597 |
Beginning balance, shares | 45,224,480 | 45,224,480 | ||||
Issuance of common stock through at-the-market offerings, net of issuance costs | 31,413 | $ 3,639 | ||||
Exercise of stock options | 1,664 | 360 | 557 | 6,720 | ||
Exercise of stock options, shares | 233,872 | |||||
Issuance of shares under employee stock purchase plan | 534 | 598 | ||||
Equity-based compensation | 4,404 | 4,592 | 4,901 | 4,107 | ||
Other comprehensive gain | 196 | 87 | ||||
Net loss | (25,683) | (21,940) | (22,219) | (21,356) | $ (47,623) | (43,575) |
Ending balance | $ 276,866 | 264,338 | 279,336 | 295,499 | $ 276,866 | 279,336 |
Ending balance, shares | 47,714,575 | 47,714,575 | ||||
ASC 842 [Member] | ||||||
Class Of Stock [Line Items] | ||||||
Retroactive adjustment to beginning accumulated deficit | (320) | |||||
ASU 2014-09 [Member] | ||||||
Class Of Stock [Line Items] | ||||||
Retroactive adjustment to beginning accumulated deficit | 5,431 | |||||
Common Stock [Member] | ||||||
Class Of Stock [Line Items] | ||||||
Beginning balance | $ 5 | $ 5 | $ 4 | $ 4 | $ 5 | $ 4 |
Beginning balance, shares | 45,479,098 | 45,224,480 | 43,091,922 | 42,384,623 | 45,224,480 | 42,384,623 |
Issuance of common stock through at-the-market offering, net of issuance costs, shares | 1,986,579 | 223,818 | ||||
Exercise of stock options, shares | 203,072 | 30,800 | 68,163 | 709,321 | ||
Issuance of shares under employee stock purchase plan, shares | 45,826 | 32,707 | ||||
Cancellation of restricted stock, shares | (4,780) | (2,022) | ||||
Ending balance | $ 5 | $ 5 | $ 4 | $ 4 | $ 5 | $ 4 |
Ending balance, shares | 47,714,575 | 45,479,098 | 43,188,012 | 43,091,922 | 47,714,575 | 43,188,012 |
Additional Paid-In Capital [Member] | ||||||
Class Of Stock [Line Items] | ||||||
Beginning balance | $ 487,559 | $ 478,968 | $ 432,533 | $ 421,706 | $ 478,968 | $ 421,706 |
Issuance of common stock through at-the-market offerings, net of issuance costs | 31,413 | 3,639 | ||||
Exercise of stock options | 1,664 | 360 | 557 | 6,720 | ||
Issuance of shares under employee stock purchase plan | 534 | 598 | ||||
Equity-based compensation | 4,404 | 4,592 | 4,901 | 4,107 | ||
Ending balance | 525,574 | 487,559 | 438,589 | 432,533 | 525,574 | 438,589 |
Accumulated Other Comprehensive Income (Loss) [Member] | ||||||
Class Of Stock [Line Items] | ||||||
Beginning balance | 59 | (28) | (28) | |||
Other comprehensive gain | 196 | 87 | ||||
Ending balance | 255 | 59 | 255 | |||
Accumulated Deficit [Member] | ||||||
Class Of Stock [Line Items] | ||||||
Beginning balance | (223,285) | (201,025) | (137,038) | (121,113) | (201,025) | (121,113) |
Net loss | (25,683) | (21,940) | (22,219) | (21,356) | ||
Ending balance | $ (248,968) | (223,285) | $ (159,257) | (137,038) | $ (248,968) | $ (159,257) |
Accumulated Deficit [Member] | ASC 842 [Member] | ||||||
Class Of Stock [Line Items] | ||||||
Retroactive adjustment to beginning accumulated deficit | $ (320) | |||||
Accumulated Deficit [Member] | ASU 2014-09 [Member] | ||||||
Class Of Stock [Line Items] | ||||||
Retroactive adjustment to beginning accumulated deficit | $ 5,431 |
Stockholders Equity - Schedul_2
Stockholders Equity - Schedule of Changes in Stockholders Equity (Parenthetical) (Detail) - USD ($) $ in Thousands | 3 Months Ended | |
Jun. 30, 2019 | Mar. 31, 2019 | |
Equity [Abstract] | ||
Stock issuance cost,net | $ 3 | $ 120 |
Related Party Transactions - Ad
Related Party Transactions - Additional Information (Detail) - USD ($) | 1 Months Ended | 3 Months Ended | 6 Months Ended | |||||
May 31, 2016 | Jul. 31, 2014 | Jun. 30, 2019 | Jun. 30, 2018 | Jun. 30, 2019 | Jun. 30, 2018 | Dec. 31, 2018 | Dec. 31, 2017 | |
Related Party Transaction [Line Items] | ||||||||
General and administrative expenses | $ 13,118,000 | $ 7,805,000 | $ 23,651,000 | $ 15,211,000 | ||||
Collaboration revenue | 11,118,000 | 7,677,000 | 21,551,000 | 15,146,000 | ||||
Deferred revenue | $ 42,160,000 | $ 50,859,000 | $ 42,160,000 | $ 50,859,000 | $ 55,932,000 | $ 59,868,000 | ||
Caribou [Member] | ||||||||
Related Party Transaction [Line Items] | ||||||||
Percentage of related party obligation | 30.00% | 30.00% | 30.00% | 30.00% | 30.00% | |||
General and administrative expenses | $ 700,000 | $ 300,000 | $ 900,000 | $ 500,000 | ||||
Caribou [Member] | Minimum [Member] | ||||||||
Related Party Transaction [Line Items] | ||||||||
Percentage of shares owned | 5.00% | 5.00% | ||||||
Novartis [Member] | Collaborative Arrangement [Member] | ||||||||
Related Party Transaction [Line Items] | ||||||||
Issuance of common stock through at-the-market offering, net of issuance costs, shares | 277,777 | |||||||
Collaboration revenue | $ 4,800,000 | $ 2,400,000 | $ 9,500,000 | $ 4,700,000 | ||||
Accounts receivable | 1,000,000 | 1,000,000 | 6,000,000 | |||||
Deferred revenue | $ 7,000,000 | $ 7,000,000 | $ 14,500,000 | |||||
Novartis [Member] | Minimum [Member] | Collaborative Arrangement [Member] | ||||||||
Related Party Transaction [Line Items] | ||||||||
Percentage of shares owned | 10.00% | 10.00% |