Cover
Cover - USD ($) $ in Millions | 12 Months Ended | ||
Dec. 31, 2020 | Feb. 28, 2021 | Jun. 30, 2020 | |
Document And Entity Information | |||
Document Type | 10-K | ||
Document Annual Report | true | ||
Document Period End Date | Dec. 31, 2020 | ||
Current Fiscal Year End Date | --12-31 | ||
Document Transition Report | false | ||
Entity File Number | 001-36080 | ||
Entity Registrant Name | IVERIC bio, Inc. | ||
Entity Incorporation, State or Country Code | DE | ||
Entity Tax Identification Number | 20-8185347 | ||
Entity Address, Address Line One | Five Penn Plaza, | ||
Entity Address, Address Line Two | Suite 2372 | ||
Entity Address, City or Town | New York, | ||
Entity Address, State or Province | NY | ||
Entity Address, Postal Zip Code | 10001 | ||
City Area Code | 212 | ||
Local Phone Number | 845-8200 | ||
Title of 12(b) Security | Common Stock, $0.001 par value | ||
Trading Symbol | ISEE | ||
Security Exchange Name | NASDAQ | ||
Entity Well-known Seasoned Issuer | No | ||
Entity Voluntary Filers | No | ||
Entity Current Reporting Status | Yes | ||
Entity Interactive Data Current | Yes | ||
Entity Filer Category | Non-accelerated Filer | ||
Entity Small Business | true | ||
Entity Emerging Growth Company | false | ||
ICFR Auditor Attestation Flag | true | ||
Entity Shell Company | false | ||
Entity Public Float | $ 442.6 | ||
Entity Common Stock, Shares Outstanding (in shares) | 90,140,797 | ||
Documents Incorporated by Reference | Part III of this Annual Report incorporates by reference information from the definitive Proxy Statement for the registrant's 2021 Annual Meeting of Shareholders, which is expected to be filed with the Securities and Exchange Commission not later than 120 days after the registrant's fiscal year ended December 31, 2020. | ||
Entity Central Index Key | 0001410939 | ||
Amendment Flag | false | ||
Document Fiscal Year Focus | 2020 | ||
Document Fiscal Period Focus | FY |
Consolidated Balance Sheets
Consolidated Balance Sheets - USD ($) $ in Thousands | Dec. 31, 2020 | Dec. 31, 2019 |
Current assets | ||
Cash and cash equivalents | $ 66,373 | $ 125,699 |
Available for sale securities | 143,674 | 0 |
Prepaid expenses and other current assets | 4,791 | 2,043 |
Income tax receivable | 1,765 | 882 |
Total current assets | 216,603 | 128,624 |
Property and equipment, net | 26 | 173 |
Right-of-use assets, net | 120 | 496 |
Income tax receivable, non-current | 0 | 882 |
Other assets | 5 | 12 |
Total assets | 216,754 | 130,187 |
Current liabilities | ||
Accrued research and development expenses | 12,284 | 6,860 |
Accounts payable and accrued expenses | 12,792 | 5,629 |
Lease liability, current | 54 | 495 |
Total current liabilities | 25,130 | 12,984 |
Lease liability, non-current | 61 | 0 |
Total liabilities | 25,191 | 12,984 |
Stockholders' equity | ||
Preferred stock—$0.001 par value, 5,000,000 shares authorized, no shares issued or outstanding | 0 | 0 |
Common stock—0.001 par value, 200,000,000 shares authorized, 90,120,797 and 49,627,064 shares issued and outstanding at December 31, 2020 and December 31, 2019, respectively | 90 | 50 |
Additional paid-in capital | 756,543 | 597,679 |
Accumulated deficit | (565,073) | (480,526) |
Accumulated other comprehensive income | 3 | 0 |
Total stockholders' equity | 191,563 | 117,203 |
Total liabilities and stockholders' equity | $ 216,754 | $ 130,187 |
Consolidated Balance Sheets (Pa
Consolidated Balance Sheets (Parenthetical) - $ / shares | Dec. 31, 2020 | Dec. 31, 2019 |
Statement of Financial Position [Abstract] | ||
Preferred stock, par value (in dollars per share) | $ 0.001 | $ 0.001 |
Preferred stock, shares issued (in shares) | 0 | 0 |
Preferred stock, shares outstanding (in shares) | 0 | 0 |
Preferred stock, shares authorized (in shares) | 5,000,000 | 5,000,000 |
Common stock, par value (in dollars per share) | $ 0.001 | $ 0.001 |
Common stock, shares authorized (in shares) | 200,000,000 | 200,000,000 |
Common stock, shares issued (in shares) | 90,120,797 | 49,627,064 |
Common stock, shares outstanding (in shares) | 90,120,797 | 49,627,064 |
Consolidated Statements of Oper
Consolidated Statements of Operations - USD ($) shares in Thousands, $ in Thousands | 12 Months Ended | ||
Dec. 31, 2020 | Dec. 31, 2019 | Dec. 31, 2018 | |
Operating expenses: | |||
Research and development | $ 62,784 | $ 39,644 | $ 41,737 |
General and administrative | 25,952 | 21,628 | 23,612 |
Total operating expenses | 88,736 | 61,272 | 65,349 |
Loss from operations | (88,736) | (61,272) | (65,349) |
Interest income | 500 | 2,151 | 2,389 |
Gain on extinguishment of Royalty Purchase Liability | 0 | 0 | 125,000 |
Other income (expense), net | (6) | 151 | (16) |
Income (loss) before income tax benefit | (88,242) | (58,970) | 62,024 |
Income tax benefit | 3,695 | 111 | 1,063 |
Net income (loss) | $ (84,547) | $ (58,859) | $ 63,087 |
Net income (loss) per common share: | |||
Basic (in dollars per share) | $ (1.14) | $ (1.39) | $ 1.70 |
Dilutive (in dollars per share) | $ (1.14) | $ (1.39) | $ 1.70 |
Weighted average common shares outstanding: | |||
Basic (in shares) | 74,185 | 42,224 | 37,061 |
Dilutive (in shares) | 74,185 | 42,224 | 37,088 |
Consolidated Statements of Comp
Consolidated Statements of Comprehensive Income (Loss) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2020 | Dec. 31, 2019 | Dec. 31, 2018 | |
Statement of Comprehensive Income [Abstract] | |||
Net income | $ (84,547) | $ (58,859) | $ 63,087 |
Other comprehensive income | |||
Unrealized gain on available for sale securities, net of tax | 3 | 0 | 0 |
Other comprehensive income | 3 | 0 | 0 |
Comprehensive income (loss) | $ (84,544) | $ (58,859) | $ 63,087 |
Consolidated Statements of Stoc
Consolidated Statements of Stockholders' Equity - USD ($) shares in Thousands, $ in Thousands | Total | Underwritten Offering | Private Placement | Junior Series A Preferred Stock | Common Stock | Common StockUnderwritten Offering | Common StockPrivate Placement | Additional paid-in capital | Additional paid-in capitalUnderwritten Offering | Additional paid-in capitalPrivate Placement | Accumulated Deficit | Accumulated Other Comprehensive Income (Loss) |
Balance at beginning of period (in shares) at Dec. 31, 2017 | 0 | 36,110 | ||||||||||
Balance at beginning of period at Dec. 31, 2017 | $ 38,041 | $ 0 | $ 36 | $ 522,759 | $ (484,754) | $ 0 | ||||||
Increase (Decrease) in Stockholders' Equity | ||||||||||||
Issuance of common stock related to acquisition (in shares) | 5,175 | |||||||||||
Issuance of common stock related to acquisition | 11,694 | $ 5 | 11,689 | |||||||||
Issuance of common stock under employee stock compensation plans and warrants (in shares) | 112 | |||||||||||
Issuance of common stock under employee stock compensation plans and warrants | 65 | $ 0 | 65 | |||||||||
Share-based compensation | 11,072 | 11,072 | ||||||||||
Net income | 63,087 | 63,087 | ||||||||||
Unrealized gain on available for sale securities, net of tax | 0 | |||||||||||
Balance at end of period (in shares) at Dec. 31, 2018 | 0 | 41,397 | ||||||||||
Ending balance at Dec. 31, 2018 | 123,959 | $ 0 | $ 41 | 545,585 | (421,667) | 0 | ||||||
Increase (Decrease) in Stockholders' Equity | ||||||||||||
Issuance of common stock and pre-funded warrants, net (in shares) | 7,750 | |||||||||||
Issuance of common stock and pre-funded warrants, net | 42,565 | $ 8 | 42,557 | |||||||||
Issuance of common stock related to acquisition (in shares) | 75 | |||||||||||
Issuance of common stock related to acquisition | 85 | 85 | ||||||||||
Issuance of common stock under employee stock compensation plans (in shares) | 405 | |||||||||||
Issuance of common stock under employee stock compensation plans | 273 | $ 1 | 272 | |||||||||
Share-based compensation | 9,180 | 9,180 | ||||||||||
Net income | (58,859) | (58,859) | ||||||||||
Unrealized gain on available for sale securities, net of tax | 0 | |||||||||||
Balance at end of period (in shares) at Dec. 31, 2019 | 0 | 49,627 | ||||||||||
Ending balance at Dec. 31, 2019 | 117,203 | $ 0 | $ 50 | 597,679 | (480,526) | 0 | ||||||
Increase (Decrease) in Stockholders' Equity | ||||||||||||
Issuance of common stock and pre-funded warrants, net (in shares) | 2,500 | 28,504 | 8,649 | |||||||||
Issuance of common stock and pre-funded warrants, net | 0 | $ 116,883 | $ 33,237 | $ 2 | $ 28 | $ 9 | (2) | $ 116,855 | $ 33,228 | |||
Issuance of common stock under employee stock compensation plans and warrants (in shares) | 841 | |||||||||||
Issuance of common stock under employee stock compensation plans and warrants | 461 | $ 1 | 460 | |||||||||
Share-based compensation | 8,323 | 8,323 | ||||||||||
Net income | (84,547) | (84,547) | ||||||||||
Unrealized gain on available for sale securities, net of tax | 3 | 3 | ||||||||||
Balance at end of period (in shares) at Dec. 31, 2020 | 0 | 90,121 | ||||||||||
Ending balance at Dec. 31, 2020 | $ 191,563 | $ 0 | $ 90 | $ 756,543 | $ (565,073) | $ 3 |
Consolidated Statements of Cash
Consolidated Statements of Cash Flows - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2020 | Dec. 31, 2019 | Dec. 31, 2018 | |
Operating Activities | |||
Net income (loss) | $ (84,547) | $ (58,859) | $ 63,087 |
Adjustments to reconcile net loss to net cash (used in) provided by operating activities | |||
Depreciation | 143 | 162 | 183 |
Amortization of premium and discounts on investment securities | 462 | 0 | 0 |
Non-cash charge on acquired in-process research and development | 0 | 0 | 5,619 |
Gain on sale of property and equipment | 0 | (150) | 0 |
Non-cash gain on extinguishment of royalty purchase agreement | 0 | 0 | (125,000) |
Share-based compensation | 8,323 | 9,180 | 11,072 |
Stock-based consideration in connection with license acquisition | 0 | 85 | 0 |
Changes in operating assets and liabilities: | |||
Income tax receivable | 0 | 1,765 | 1,387 |
Prepaid expense and other assets | (2,742) | 45 | 1,070 |
Accrued interest receivable | (323) | 0 | 0 |
Accrued research and development expenses | 5,424 | (477) | 2,353 |
Accounts payable and accrued expenses | 7,163 | (241) | (1,682) |
Net cash used in operating activities | (66,097) | (48,490) | (41,911) |
Investing Activities | |||
Purchase of marketable securities | (143,810) | 0 | 0 |
Proceeds from sale of assets | 0 | 150 | 0 |
Net cash provided by (used in) investing activities | (143,810) | 150 | 0 |
Financing Activities | |||
Proceeds from employee stock plan purchases and stock option exercises | 461 | 273 | 65 |
Net cash provided by financing activities | 150,581 | 42,838 | 6,140 |
Net change in cash and cash equivalents | (59,326) | (5,502) | (35,771) |
Cash and cash equivalents | |||
Beginning of period | 125,699 | 131,201 | 166,972 |
End of period | 66,373 | 125,699 | 131,201 |
End of period | |||
Interest | (3,327) | (1,893) | (2,467) |
Income taxes received, net | |||
Supplemental disclosures of non-cash information related to investing activities | 3 | 0 | 0 |
Common stock issued to acquire net assets of Inception 4, Inc. | 0 | 0 | 11,694 |
Operating right-of-use assets obtained in exchange for lease obligations | 166 | 1,469 | |
Public Stock Offering | |||
Financing Activities | |||
Proceeds from issuance of common stock and pre-funded warrants, net, and related to acquisition | 116,883 | 42,565 | 0 |
Private Placement | |||
Financing Activities | |||
Proceeds from issuance of common stock and pre-funded warrants, net, and related to acquisition | 33,237 | 0 | 0 |
Inception 4 | |||
Financing Activities | |||
Proceeds from issuance of common stock and pre-funded warrants, net, and related to acquisition | $ 0 | $ 0 | $ 6,075 |
Business
Business | 12 Months Ended |
Dec. 31, 2020 | |
Organization, Consolidation and Presentation of Financial Statements [Abstract] | |
Business | Business Description of Business and Organization IVERIC bio, Inc. (the “Company” or “IVERIC”) is a science-driven biopharmaceutical company focused on the discovery and development of novel treatment options for retinal diseases with significant unmet medical needs. The Company is currently developing both therapeutic product candidates for age-related retinal diseases and gene therapy product candidates for orphan inherited retinal diseases ("IRDs"). The Company believes that both therapeutics and gene therapy serve important roles in drug development and providing potential treatment options for patients suffering from retinal diseases. The Company's therapeutics portfolio consists of Zimura® (avacincaptad pegol), a complement C5 inhibitor, and its preclinical product candidate IC-500, a High temperature requirement A serine peptidase 1 protein ("HtrA1") inhibitor. The Company is targeting the following diseases with Zimura: • Geographic Atrophy ("GA"), which is the advanced stage of age-related macular degeneration ("AMD"), and is characterized by marked thinning or atrophy of retinal tissue, leading to irreversible loss of vision; and • autosomal recessive Stargardt disease ("STGD1"), which is an orphan inherited condition characterized by progressive damage to the central portion of the retina (the "macula") and other retinal tissue, leading to loss of vision. The Company previously also evaluated Zimura in combination with Lucentis® (ranibizumab), an anti-vascular endothelial growth factor ("anti-VEGF") agent, for the treatment of wet AMD, for which the Company completed a Phase 2a clinical trial during the fourth quarter of 2018. The Company is developing IC-500 for GA secondary to AMD and potentially other age-related retinal diseases. The Company's gene therapy portfolio consists of two product candidates in preclinical development (IC-100 and IC-200) and several ongoing research and development programs, each of which uses adeno-associated virus ("AAV") for gene delivery. These AAV mediated gene therapy programs are targeting the following orphan IRDs: • rhodopsin-mediated autosomal dominant retinitis pigmentosa ("RHO-adRP"), which is characterized by progressive and severe bilateral loss of vision leading to blindness; • IRDs associated with mutations in the BEST1 gene, including Best vitelliform macular dystrophy ("Best disease"), which is generally characterized by bilateral egg yolk-like lesions in the macula, which, over time, progress to atrophy and loss of vision; • Leber Congenital Amaurosis type 10 ("LCA10"), which is characterized by severe bilateral loss of vision at or soon after birth; • autosomal recessive Stargardt disease; and • IRDs associated with mutations in the USH2A gene, which include Usher syndrome type 2A and USH2A -associated nonsyndromatic autosomal recessive retinitis pigmentosa. The Company is developing IC-100 for RHO-adRP and IC-200 for BEST1 -related IRDs. |
Summary of Significant Accounti
Summary of Significant Accounting Policies | 12 Months Ended |
Dec. 31, 2020 | |
Accounting Policies [Abstract] | |
Summary of Significant Accounting Policies | Summary of Significant Accounting Policies Basis of Presentation and Consolidation The accompanying consolidated financial statements have been prepared in accordance with accounting principles generally accepted in the United States of America ("GAAP") and include all adjustments necessary for the fair presentation of the Company's financial position for the periods presented. The consolidated financial statements include the accounts of the Company and its subsidiaries. All intercompany balances and transactions have been eliminated in consolidation. Segment and geographic information Operating segments are defined as components of an enterprise about which separate discrete information is available for evaluation by the chief operating decision maker, or decision making group, in deciding how to allocate resources and in assessing performance. The Company views its operations and manages its business in one operating and reporting segment. Use of Estimates The preparation of financial statements and related disclosures in conformity with GAAP requires management to make estimates and judgments that affect the amounts reported in the financial statements and accompanying notes. The Company bases its estimates and judgments on historical experience and on various other assumptions that it believes are reasonable under the circumstances. The amounts of assets and liabilities reported in the Company's Consolidated Balance Sheets and the amount of expenses reported for each of the periods presented are affected by estimates and assumptions, which are used for, but not limited to, accounting for research and development costs, accounting for share-based compensation and accounting for income taxes. Actual results could differ from those estimates. Cash, Cash Equivalents and Available for Sale Securities The Company considers all highly liquid investments with an original maturity of 90 days or less when purchased to be cash equivalents. The carrying amounts reported in the Balance Sheets for cash and cash equivalents are valued at cost, which approximates their fair value. The Company considers securities with original maturities of greater than 90 days to be available for sale securities. Available for sale securities with original maturities of greater than one year are recorded as non-current assets. Available for sale securities are recorded at fair value and unrealized gains and losses are recorded within other comprehensive income. On a quarterly basis, the Company reviews the status of each security in an unrealized loss position, to evaluate the existence of potential credit losses. The Company first considers whether it intends to sell, or if it is more likely than not that the Company will be required to sell the security before recovery of its amortized cost basis. If either of the criteria regarding intent or requirement to sell is met, the security’s amortized cost basis is written down to fair value through income. For securities that do not meet this criteria, the Company considers a number of factors to determine if the decline in fair value has resulted from credit losses or other factors, including but not limited to: (1) the extent of the decline; (2) changes to the rating of the security by a rating agency; (3) any adverse conditions specific to the security; and (4) other market conditions that may affect the fair value of the security. If this assessment indicates that a credit loss exists and the present value of cash flows expected to be collected is less than the amortized cost basis, an allowance for credit losses is required for the credit loss. Any impairment that has not been recorded through an allowance for credit losses is recognized in other comprehensive income. As of December 31, 2020, the Company had cash, cash equivalents and available for sale securities of approximately $210.0 million. The Company believes that its cash, cash equivalents and available for sale securities as of December 31, 2020 will be sufficient to fund its operations and capital expenditure requirements as currently planned for at least the next 12 months from the filing of the Company's Annual Report on Form 10-K. Concentration of Credit Risk The Company's financial instruments that are exposed to concentration of credit risk consist primarily of cash, cash equivalents and available for sale securities. The Company maintains its cash in bank accounts, the balance of which generally exceed federally insured limits. The Company maintains its cash equivalents and available for sale securities in investments in money market funds, in U.S. Treasury securities and asset-backed securities and investment-grade corporate debt securities with original maturities of 90 days or less. The Company believes it is not exposed to significant credit risk on its cash and cash equivalents. Concentration of Suppliers The Company historically relied upon a single third-party manufacturer to provide the drug substance for Zimura on a purchase order basis. The Company also historically relied upon a single third-party manufacturer to provide fill/finish services for clinical supplies of Zimura. The Company has engaged one additional third-party manufacturer to provide drug substance for Zimura and one additional third-party manufacturer to provide fill/finish services for clinical supplies of Zimura. In addition, the Company currently relies upon a single third-party supplier to supply on a purchase order basis the polyethylene glycol starting material used to manufacture Zimura. Furthermore, the Company and its contract manufacturers currently rely upon sole-source suppliers of certain raw materials and other specialized components of production used in the manufacture and fill/finish of Zimura. The Company currently relies exclusively upon a single third-party contract manufacturer for IC-100 and IC-200, and also relies on sole-source suppliers for certain starting materials used in the manufacture of such product candidates. The Company currently relies upon a single third-party contract manufacturer to conduct process development, scale-up and GMP manufacture of the drug substance for IC-500 for preclinical toxicology studies and early-stage clinical trials and a single third-party contract manufacturer to conduct formulation development activities for IC-500. If the Company’s third-party manufacturers or fill/finish service providers should become unavailable to the Company for any reason, including as a result of capacity constraints, different business objectives, financial difficulties, insolvency or the COVID-19 pandemic, the Company believes that there are a limited number of potential replacement manufacturers, and the Company likely would incur added costs and delays in identifying or qualifying such replacements. Foreign Currency Translation The Company considers the U.S. dollar to be its functional currency. Expenses denominated in foreign currencies are translated at the exchange rate on the date the expense is incurred. The effect of exchange rate fluctuations on translating foreign currency assets and liabilities into U.S. dollars is included in the Consolidated Statements of Operations and Comprehensive Loss. Foreign exchange transaction gains and losses are included in the results of operations and are not material in the Company's financial statements. Financial Instruments Cash equivalents are reflected in the accompanying financial statements at fair value. The carrying amount of accounts payable and accrued expenses, including accrued research and development expenses, approximates fair value due to the short-term nature of those instruments. ASC 820, Fair Value Measurements and Disclosures , defines fair value as the price that would be received to sell an asset, or paid to transfer a liability, in the principal or most advantageous market in an orderly transaction between market participants on the measurement date. The fair value standard also establishes a three-level hierarchy, which requires an entity to maximize the use of observable inputs and minimize the use of unobservable inputs when measuring fair value. The Company reviews investments on a periodic basis for other than temporary impairments. This review is subjective as it requires management to evaluate whether an event or change in circumstances has occurred in the period that may have a significant adverse effect on the fair value of the investment. The Company uses the market approach to measure fair value for its financial assets. The market approach uses prices and other relevant information generated by market transactions involving identical or comparable assets. The Company classifies its corporate debt securities within the fair value hierarchy as Level 2 assets, as it primarily utilizes quoted market prices or rates for similar instruments to value these securities. Leases The Company determines if an arrangement contains a lease at inception. For arrangements where the Company is the lessee, it recognizes a right-to-use ("ROU") lease asset and operating lease liability on the Company's Consolidated Balance Sheet. ROU lease assets represent the Company's right to use the underlying asset for the lease term and the lease obligation represents the Company's commitment to make the lease payments arising from the lease. Right-of-use lease assets and obligations are recognized at the commencement date based on the present value of remaining lease payments over the lease term. As the Company’s leases do not provide an implicit discount rate, the Company has used an estimated incremental borrowing rate based on the information available at the commencement date in determining the present value of lease payments. The ROU lease asset includes any lease payments made prior to commencement and excludes any lease incentives. The lease term may include options to extend or terminate the lease when it is reasonably certain that the Company will exercise that option. Operating lease expense is recognized on a straight-line basis over the lease term, subject to any changes in the lease or expectations regarding the terms. Variable lease costs such as common area costs and property taxes are expensed as incurred. For all office lease agreements the Company combines lease and nonlease components. Leases with an initial term of 12 months or less are not recorded on the Company's Consolidated Balance Sheet. Property and Equipment Property and equipment, which consists mainly of clinical equipment, computers, software, other office equipment, and leasehold improvements, are carried at cost less accumulated depreciation. Depreciation is computed over the estimated useful lives of the respective assets, generally three Research and Development The Company's research and development expenses primarily consist of costs associated with the manufacturing, development, and preclinical and clinical testing of the Company's product candidates and costs associated with its collaborative gene therapy sponsored research programs. The Company's research and development expenses consist of: • external research and development expenses incurred under arrangements with third parties, such as academic research collaborators, contract research organizations ("CROs") and contract development and manufacturing organizations ("CDMOs") and other vendors for the production and analysis of drug substance and drug product; and • employee-related expenses for employees dedicated to research and development activities, including salaries, benefits and share-based compensation expense. Research and development expenses also include costs of acquired product licenses, in-process research and development, and related technology rights where there is no alternative future use, costs of prototypes used in research and development, consultant fees and amounts paid to collaborators. All research and development expenses are charged to operations as incurred in accordance with ASC 730, Research and Development . The Company accounts for non-refundable advance payments for goods and services that will be used in future research and development activities as expenses when the service has been performed or when the goods have been received, rather than when the payment is made. Income Taxes The Company utilizes the liability method of accounting for deferred income taxes, as set forth in ASC 740, Income Taxes. Under this method, deferred tax assets and liabilities are recognized for the expected future tax consequences of temporary differences between the carrying amounts and the tax basis of assets and liabilities. A valuation allowance is established against deferred tax assets when, based on the weight of available evidence, it is more likely than not that some or all of the deferred tax assets will not be realized. The Company's policy is to record interest and penalties on uncertain tax positions as income tax expense. Share-Based Compensation The Company follows the provisions of ASC 718, Compensation—Stock Compensation, which requires the measurement and recognition of compensation expense for all share-based payment awards made to employees and non-employee directors and consultants, including employee stock options, restricted stock units (“RSUs”) and options granted to employees to purchase shares under the 2016 Employee Stock Purchase Plan (the “ESPP”). Share-based compensation expense is based on the grant date fair value estimated in accordance with the provisions of ASC 718 and is generally recognized as an expense over the requisite service period, net of estimated forfeitures. For grants containing performance-based vesting provisions, expense is recognized over the estimated achievement period only when the performance-based milestone is deemed probable of achievement. If performance-based milestones are later determined not to be probable of achievement, then all previously recorded stock-based compensation expense associated with such options will be reversed during the period in which the Company makes this determination. The Company estimates forfeitures at the time of grant and revises those estimates in subsequent periods if actual forfeitures differ from its estimates. The Company uses historical data to estimate pre-vesting forfeitures and record share-based compensation expense only for those awards that are expected to vest. To the extent that actual forfeitures differ from the Company's estimates, the difference is recorded as a cumulative adjustment in the period the estimates were revised. Stock Options The Company estimates the fair value of stock options granted to employees, non-employees and non-employee directors on the date of grant using the Black-Scholes option-pricing model. The Company's computation of stock-price volatility is based on daily historical volatility during the time period that corresponds to the expected option term. The Company's computation of expected term is determined using the expected term of stock option grants to employees based on an analysis of actual option exercises. The Company utilizes a dividend yield of zero based on the fact that the Company has never paid cash dividends to stockholders and has no current intentions to pay cash dividends. The risk-free interest rate is based on the zero-coupon U.S. Treasury yield at the date of grant for a term equivalent to the expected term of the option. The weighted-average assumptions used to estimate grant date fair value of stock options using the Black-Scholes option pricing model were as follows for the years ended December 31, 2020, 2019 and 2018: Years ended December 31, 2020 2019 2018 Expected common stock price volatility 118% 110% 85% Risk-free interest rate 0.22%-1.34% 1.38%-2.54% 2.39%-2.95% Expected term of options (years) 4.6 4.8 6.0 Expected dividend yield — — — RSUs The Company estimates the fair value of RSUs granted to employees using the closing market price of the Company's common stock on the date of grant. ESPP In April 2016, the Company's board of directors adopted the ESPP pursuant to which the Company may sell up to an aggregate of 1,000,000 shares of its common stock. The ESPP was approved by the Company’s stockholders in June 2016. The ESPP is considered compensatory and the fair value of the discount and look back provision are estimated using the Black-Scholes option-pricing model and recognized over the six month withholding period prior to purchase. Share-based compensation expense includes expenses related to stock options and RSUs granted to employees, non-employee directors and consultants, as well as the option granted to employees to purchase shares under the ESPP, all of which have been reported in the Company’s Statements of Operations as follows: Years ended December 31, 2020 2019 2018 Research and development $ 4,166 $ 4,260 $ 4,967 General and administrative 4,157 4,920 6,105 Total $ 8,323 $ 9,180 $ 11,072 Recently Adopted Accounting Pronouncements In June 2016, the Financial Accounting Standards Board (the "FASB") issued Accounting Standard Update ("ASU") 2016-13 Financial Instruments (Topic 326) Measurement of Credit Losses on Financial Instrument (“CECL”). ASU 2016-13 requires an allowance for expected credit losses on financial assets be recognized as early as day one of the instrument. This ASU departs from the incurred loss model which means the probability threshold is removed. It considers more forward-looking information and requires the entity to estimate its credit losses as far as it can reasonably estimate. This ASU is effective for fiscal years beginning after December 15, 2019, including interim periods within those fiscal years. The Company adopted this accounting standard effective January 1, 2020 with no material impact to its financial statements. In August 2018, the FASB issued ASU No. 2018-13, Fair Value Measurement (Topic 820) , which modifies the disclosure requirements on fair value measurements in Topic 820, Fair Value Measurement, including, among other changes, the consideration of costs and benefits when evaluating disclosure requirements. For public companies, the amendments became effective for annual reporting periods beginning after December 15, 2019, including interim periods within those annual periods. The Company adopted this accounting standard effective January 1, 2020 with no material impact to its financial statements. In August 2018, the FASB issued ASU No. 2018-15, Intangibles - Goodwill and Other - Internal-Use Software (Subtopic 350-40): Customer’s Accounting for Implementation Costs Incurred in a Cloud Computing Arrangement That Is a Service Contract (a consensus of the FASB Emerging Issues Task Force). ASU 2018-15 aligns the requirements for capitalizing implementation costs incurred in a hosting arrangement that is a service contract with the requirements for capitalizing implementation costs incurred to develop or obtain internal-use software (and hosting arrangements that include an internal-use software license). This guidance became effective for fiscal years beginning after December 15, 2019, and for interim periods within those fiscal years. The Company adopted this accounting standard effective January 1, 2020 with no material impact to its financial statements. In November 2018, the FASB issued ASU No. 2018-18, Collaborative Arrangements (Topic 808) , which clarifies the interaction between the guidance for collaborative arrangements (Topic 808) and the new revenue recognition standard (Topic 606). For public companies, the amendments became effective for annual reporting periods beginning after December 15, 2019, including interim periods within those annual periods. The Company adopted this accounting standard effective January 1, 2020 with no material impact to its financial statements. In December 2019, the FASB issued ASU No. 2019-12, Simplifying the Accounting for Income Taxes (“ASU 2019-12”), which simplifies the accounting for income taxes, eliminates certain exceptions within ASC 740, Income Taxes , and clarifies certain aspects of the current guidance to promote consistency among reporting entities. ASU 2019-12 is effective for fiscal years beginning after December 15, 2021. Most amendments within the standard are required to be applied on a prospective basis, while certain amendments must be applied on a retrospective or modified retrospective basis. The Company adopted this accounting standard effective January 1, 2020 with no material impact to its financial statements. |
Common Stock
Common Stock | 12 Months Ended |
Dec. 31, 2020 | |
Equity [Abstract] | |
Common Stock | Common Stock June 2020 Public Offering and Private Placement In June 2020, the Company closed an underwritten public offering in which it sold 28,503,220 shares of its common stock, which includes the exercise in full of the underwriters’ option to purchase additional shares of its common stock, at a price to the public of $4.10 per share, and at a price to the underwriters of $3.854 per share. The Company also sold to certain investors in lieu of common stock, pre-funded warrants to purchase 1,914,280 shares of its common stock at a price to the public of $4.099 per share underlying each pre-funded warrant, and at a price to the underwriters of $3.853 per share underlying each pre-funded warrant. The pre-funded warrants are immediately exercisable with certain restrictions and do not expire. Concurrently with the June 2020 public offering, the Company completed a private placement exempt from the registration requirements of the Securities Act of 1933, as amended (the "Securities Act"), in which it sold 8,649,453 shares of its common stock to affiliates of Vivo Capital, LLC and Samsara BioCapital, LP (the "Private Placement Purchasers"), at a purchase price equal to $4.10 per share, which is the price to the public in the public offering. The shares in the private placement were issued pursuant to a stock purchase agreement entered into among the Company and the Private Placement Purchasers. The net proceeds from the public offering and private placement, after deducting underwriting discounts, placement agent fees and other offering expenses of approximately $10.1 million, was approximately $150.1 million. December 2019 Public Offering On December 6, 2019, the Company completed an underwritten public offering in which it sold 6,250,000 shares of its common stock at a public offering price of $4.00 per share, and it sold to certain investors pre-funded warrants to purchase 3,750,000 shares of its common stock at a public offering price of $3.999 per share underlying each warrant. The offering included an option for the underwriters to purchase up to an additional 1,500,000 shares of its common stock, which was subsequently exercised in full by the underwriters. The shares of its common stock were sold to the underwriters at a purchase price of $3.736 per share and the pre-funded warrants were sold to the underwriters at a purchase price of $3.735 per share underlying each warrant. The pre-funded warrants are immediately exercisable with certain restrictions and do not expire. The net proceeds from the offering, after deducting underwriting discounts and commissions of $3.0 million and other offering expenses of $0.4 million, was approximately $42.6 million. The Company evaluated the pre-funded warrants for liability or equity classification in accordance with the provisions of ASC 480, Distinguishing Liabilities from Equity , and ASC 815-40, Derivatives and Hedging . Based on the provisions governing the pre-funded warrants in the applicable agreement, the Company determined that the pre-funded warrants meet the criteria required to be classified as an equity award subject to the guidance in ASC 815-10 and 815-40 and should effectively be treated as outstanding common shares in both basic and diluted EPS calculations. miniCEP290 Program - University of Massachusetts |
Net Income (Loss) Per Common Sh
Net Income (Loss) Per Common Share | 12 Months Ended |
Dec. 31, 2020 | |
Earnings Per Share [Abstract] | |
Net Income (Loss) Per Common Share | Net Income (Loss) Per Common ShareBasic and diluted net loss per common share is determined by dividing net loss by the weighted average common shares and pre-funded warrants outstanding during the period. Basic and diluted shares outstanding includes the weighted average effect of the Company's outstanding pre-funded warrants as the exercise of such pre-funded warrants requires nominal consideration to be given for the delivery of the corresponding shares of common stock. As of December 31, 2020 and 2019, the Company had 3,164,280 and 3,750,000 pre-funded warrants outstanding, respectively, which if exercised, would increase the number of shares of common stock issued and outstanding. For the periods when there is a net loss, shares underlying stock options and RSUs have been excluded from the calculation of diluted net loss per common share because the effect of including such shares would be anti-dilutive. Therefore, the weighted average common shares used to calculate both basic and diluted net loss per common share would be the same. The following table sets forth the computation of basic and diluted net income (loss) per common share for the periods indicated: Years ended December 31, 2020 2019 2018 Basic and diluted net income (loss) per common share calculation: Net income (loss) $ (84,547) $ (58,859) $ 63,087 Weighted average common shares outstanding - basic 74,185 42,224 37,061 Plus: net effect of dilutive stock options and unvested restricted stock units — — 27 Weighted average common shares outstanding - dilutive 74,185 42,224 37,088 Net income (loss) per common share - basic $ (1.14) $ (1.39) $ 1.70 Net income (loss) per common share - diluted $ (1.14) $ (1.39) $ 1.70 The following potentially dilutive securities have been excluded from the computations of diluted weighted average common shares outstanding for the periods presented, as the effect of including such shares would be anti-dilutive: Years ended December 31, 2020 2019 2018 Stock options outstanding 8,928 6,780 5,873 Restricted stock units 1,958 1,481 601 Total 10,886 8,261 6,474 |
Cash, Cash Equivalents and Avai
Cash, Cash Equivalents and Available for Sale Securities | 12 Months Ended |
Dec. 31, 2020 | |
Cash and Cash Equivalents [Abstract] | |
Cash, Cash Equivalents and Available for Sale Securities | Cash, Cash Equivalents and Available for Sale Securities The Company considers all highly liquid investments purchased with original maturities of 90 days or less at the date of purchase to be cash equivalents. As of December 31, 2020 and December 31, 2019 the Company had cash and cash equivalents of approximately $66.4 million and $125.7 million, respectively. Cash and cash equivalents at December 31, 2020 and December 31, 2019 included cash of $8.4 million and $3.2 million, respectively. As of December 31, 2020 and December 31, 2019, cash and cash equivalents also included $58.0 million and $122.5 million, respectively, of investments in money market funds and certain short-term investment-grade corporate debt securities with original maturities of 90 days or less. The Company considers securities with original maturities of greater than 90 days at the date of purchase to be available for sale securities. The Company had available for sale securities of approximately $143.7 million at December 31, 2020. The available for sale securities listed at December 31, 2020 mature in one year or less. The Company held no available for sale securities at December 31, 2019. The Company evaluates securities with unrealized losses, if any, to determine whether the decline in fair value has resulted from credit loss or other factors. The Company has determined that there were no credit losses in fair value of its investments as of December 31, 2020. Factors considered in determining whether a loss resulted from a credit loss or other factors included the length of time and extent to which the investment’s fair value has been less than the cost basis, the financial condition and near-term prospects of the investee, the extent of the loss related to credit of the issuer, the expected cash flows from the security, the Company’s intent to sell the security, and whether or not the Company will be required to sell the security before the recovery of its amortized cost. The Company classifies these securities as available for sale. However, the Company has not sold and does not currently intend to sell its investments and the Company believes it is more likely than not that the Company will recover the carrying value of these investments. Available for sale securities, including carrying value and estimated fair values, are summarized as follows: As of December 31, 2020 Amortized Cost Gross Unrealized Gains Gross Unrealized Losses Fair Value U.S. Treasury securities $ 54,225 $ 8 $ — $ 54,233 Corporate debt securities 79,282 4 (10) 79,276 Asset-backed securities 10,164 1 — 10,165 Total $ 143,671 $ 13 $ (10) $ 143,674 The Company's available for sale securities are reported at fair value on the Company's balance sheet. Unrealized gains (losses) are reported within accumulated other comprehensive loss in the statements of comprehensive income (loss). The cost of securities sold and any realized gains/losses from the sale of available for sale securities are based on the specific identification method. The changes in accumulated other comprehensive income (loss) associated with the unrealized loss on available for sale securities for the years ended December 31, 2020 and December 31, 2019 were as follows: Years ended December 31, 2020 2019 Beginning balance $ — $ — Current period changes in fair value before reclassifications, net of tax 3 — Amounts reclassified from accumulated other comprehensive income, net of tax — — Total other comprehensive income $ 3 $ — Ending balance $ 3 $ — |
Fair Value Measurements
Fair Value Measurements | 12 Months Ended |
Dec. 31, 2020 | |
Fair Value Disclosures [Abstract] | |
Fair Value Measurements | Fair Value Measurements ASC 820, Fair Value Measurements and Disclosures , defines fair value as the price that would be received to sell an asset, or paid to transfer a liability, in the principal or most advantageous market in an orderly transaction between market participants on the measurement date. The fair value standard also establishes a three-level hierarchy, which requires an entity to maximize the use of observable inputs and minimize the use of unobservable inputs when measuring fair value. The Company reviews investments on a periodic basis for other than temporary impairments. This review is subjective as it requires management to evaluate whether an event or change in circumstances has occurred in the period that may have a significant adverse effect on the fair value of the investment. The Company uses the market approach to measure fair value for its financial assets. The market approach uses prices and other relevant information generated by market transactions involving identical or comparable assets. The Company classifies its investment-grade corporate debt securities within the fair value hierarchy as Level 2 assets, as it primarily utilizes quoted market prices or rates for similar instruments to value these securities. The valuation hierarchy is based upon the transparency of inputs to the valuation of an asset or liability on the measurement date. The three levels are defined as follows: • Level 1—inputs to the valuation methodology are quoted prices (unadjusted) for an identical asset or liability in an active market. The Company's Level 1 assets consist of investments in money market funds and U.S. Treasury securities. • Level 2—inputs to the valuation methodology include quoted prices for a similar asset or liability in an active market or model-derived valuations in which all significant inputs are observable for substantially the full term of the asset or liability. The Company's Level 2 assets may consist of investments in investment-grade corporate debt securities. • Level 3—inputs to the valuation methodology are unobservable and significant to the fair value measurement of the asset or liability. The Company does not hold any assets that are measured using Level 3 inputs. The Company classifies its corporate debt securities within the fair value hierarchy as Level 2 assets, as it primarily utilizes quoted market prices or rates for similar instruments to value these securities. The following table presents, for each of the fair value hierarchy levels required under ASC 820, the Company's assets and liabilities that are measured at fair value on a recurring basis as of December 31, 2020: Fair Value Measurement Using Quoted prices in Significant other Significant Assets Investments in money market funds* $ 58,042 $ — $ — Investments in U.S. Treasury securities $ 54,233 $ — $ — Investments in corporate debt securities* $ — $ 79,275 $ — Investments in asset-backed securities $ — $ 10,166 $ — The following table presents, for each of the fair value hierarchy levels required under ASC 820, the Company's assets and liabilities that are measured at fair value on a recurring basis as of December 31, 2019: Fair Value Measurement Using Quoted prices in Significant other Significant Assets Investments in money market funds* $ 108,585 $ — $ — Investments in corporate debt securities* $ — $ 13,875 $ — * Investments in money market funds and corporate debt securities with maturities less than 90 days are reflected in cash and cash equivalents in the accompanying Balance Sheets. No transfer of assets between Level 1 and Level 2 of the fair value measurement hierarchy occurred during the years ended December 31, 2020 or December 31, 2019. |
Inception 4 Acquisition
Inception 4 Acquisition | 12 Months Ended |
Dec. 31, 2020 | |
Asset Acquisition [Abstract] | |
Inception 4 Acquisition | Inception 4 Acquisition In October 2018, the Company entered into an Agreement and Plan of Merger (the “Inception 4 Merger Agreement”) by and among the Company, Orion Ophthalmology Merger Sub, Inc., a Delaware corporation and a direct, wholly owned subsidiary of the Company (“Merger Sub I”), Orion Ophthalmology LLC, a Delaware limited liability company and a direct, wholly owned subsidiary of the Company (“Orion”), Inception 4, Inc., a Delaware corporation ("Inception 4"), and, solely in its capacity as the representative, agent and attorney-in-fact of the equityholders of Inception 4, Fortis Advisors LLC, a Delaware limited liability company. Pursuant to the Inception 4 Merger Agreement, in October 2018, the Company acquired Inception 4 through the merger of Merger Sub I with and into Inception 4, with Inception 4 surviving as a direct, wholly owned subsidiary of the Company, and as part of the same overall transaction, the merger of Inception 4 with and into Orion, with Orion surviving as a direct, wholly owned subsidiary of the Company (the transactions are collectively referred to as the "Inception 4 Merger"). Prior to the Inception 4 Merger, Inception 4 was a privately held biotechnology company focused on the research and development of small molecule HtrA1 inhibitors for age-related retinal diseases in humans. Pursuant to the terms of the Inception 4 Merger Agreement, as upfront consideration, the Company issued approximately 5.2 million shares of the Company’s common stock to the former equityholders of Inception 4, which were valued at approximately $11.7 million based on the closing price of the Company’s common stock on the acquisition date equal to $2.26 per share. Additionally, subject to the terms and conditions of the Inception 4 Merger Agreement, the former equityholders of Inception 4 will be entitled to receive contingent payments up to an aggregate of $105.0 million from the Company for the achievement of specified clinical and regulatory milestones, with $45 million of such potential payments relating to GA and $60 million of such potential payments relating to wet AMD. The future milestone payments will be payable in the form of shares of the Company's common stock, calculated based on the price of its common stock over a five-trading day period preceding the achievement of the relevant milestone, unless and until the issuance of such shares would, together with all other shares issued in connection with the Inception 4 Merger, exceed an overall maximum limit of approximately 7.2 million shares, which is equal to 19.9% of the number of issued and outstanding shares of the Company's common stock as of the close of business on the business day prior to the closing date of the Inception 4 Merger, and will be payable in cash thereafter. At closing, the Company acquired all of Inception 4's assets, which included intellectual property and in-process research and development (“IPR&D”) associated with Inception 4’s HtrA1 inhibitor program and approximately $6.1 million in cash. There were no other tangible assets, leases or liabilities. The Company evaluated the acquisition of Inception 4 under ASU No. 2017-01, Business Combinations: Clarifying the Definition of a Business . Based on the results of the analysis performed, the Company concluded that substantially all of the fair value of the gross assets acquired under the Inception 4 Merger Agreement is concentrated in a single identifiable asset. As a result, the Company concluded that the acquisition of Inception 4 does not represent an acquisition of a business and does not qualify as a business combination to be accounted for under ASC 805. Further, the Company concluded that the legal entity is not a variable interest entity under ASC 810 - Consolidation . Consequently, the Company concluded that the acquisition of Inception 4 should be accounted for as an asset acquisition under ASC 805-50. As the contingent consideration is not a derivative under ASC 815 - Derivatives and Hedging , any contingent payments will be recognized when earned or achieved and such amounts will be expensed upon payment for pre-commercial activities and capitalized for regulatory approvals and post-commercial activities. Further, as the acquired IPR&D has no alternative future use, in accordance with ASC 730, at closing, the Company charged to expense its allocated fair value of $6.9 million, which is the total acquisition consideration including transaction costs of $1.3 million less the cash acquired. |
Licensing and Commercialization
Licensing and Commercialization Agreement | 12 Months Ended |
Dec. 31, 2020 | |
Organization, Consolidation and Presentation of Financial Statements [Abstract] | |
Licensing and Commercialization Agreement | Licensing and Commercialization Agreements Zimura License Agreement with Archemix Corp. In September 2011, the Company entered into an amended and restated exclusive license agreement with Archemix Corp. ("Archemix") relating to anti-C5 aptamers (as amended, the "C5 License Agreement"). The C5 License Agreement superseded a July 2007 agreement between the Company and Archemix. Under the C5 License Agreement, the Company holds exclusive worldwide licenses, subject to certain pre–existing rights, under specified patents and technology owned or controlled by Archemix to develop, make, use, sell, offer for sale, distribute for sale, import and export pharmaceutical products comprised of or derived from an anti-C5 aptamer, including Zimura, for the prevention, treatment, cure or control of human indications, diseases, disorders or conditions of the eye, adnexa of the eye, orbit and optic nerve, other than certain expressly excluded applications. In connection with the C5 License Agreement, the Company paid Archemix an upfront licensing fee of $1.0 million and issued to Archemix an aggregate of 2,000,000 shares of its series A-1 preferred stock and 500,000 shares of its series B-1 preferred stock. The Company has paid Archemix an aggregate of $9.0 million in fees based on its achievement of specified clinical milestone events under the C5 License Agreement, including two milestone payments of $1.0 million and $6.0 million, respectively, triggered by the positive 12-month data from, and by completion of, the GATHER1 trial, which the Company paid in March 2020 and October 2020, respectively. Under the C5 License Agreement, for each anti-C5 aptamer product that the Company may develop under the agreement, including Zimura, it is obligated to make additional payments to Archemix of up to an aggregate of $50.5 million if it achieves specified development, clinical and regulatory milestones, with $24.5 million of such payments relating to a first indication, $23.5 million of such payments relating to second and third indications and $2.5 million of such payments relating to sustained delivery applications. Under the C5 License Agreement, it is also obligated to make additional payments to Archemix of up to an aggregate of $22.5 million if it achieves specified commercial milestones based on net product sales of all anti-C5 products licensed under the agreement. It is also obligated to pay Archemix a double-digit percentage of specified non-royalty payments it may receive from any sublicensee of its rights under the C5 License Agreement. The Company is not obligated to pay Archemix a running royalty based on net product sales in connection with the C5 License Agreement. Unless earlier terminated, the C5 License Agreement will expire upon the latest of 12 years after the first commercial sale in any country of the last licensed product, the expiration of the last-to-expire valid claim of the licensed patents that covers a licensed product, and the date on which no further payments of sublicensing income are to be received by the Company. Either the Company or Archemix may terminate the C5 License Agreement if the other party materially breaches the agreement and the breach remains uncured for a specified period. Archemix may also terminate the C5 License Agreement, or may convert the Company's exclusive license under the agreement to a non-exclusive license, if the Company challenges or assists a third party in challenging the validity or enforceability of any of the patents licensed under the agreement. The Company may terminate the agreement at any time and for any or no reason effective at the end of a specified period following its written notice of termination to Archemix. IC-100 Agreements with the University of Florida Research Foundation and the University of Pennsylvania In June 2018, the Company entered into an exclusive global license agreement (the "RHO-adRP License Agreement") with the University of Florida Research Foundation, Incorporated ("UFRF") and the University of Pennsylvania ("Penn" and collectively with UFRF, the "Licensors"). Under the agreement, the Licensors granted the Company a worldwide, exclusive license under specified patent rights and a worldwide, non-exclusive license under specified know-how, including specified preclinical data, to manufacture, develop and commercialize certain AAV gene therapy products for the treatment of rhodopsin-mediated diseases. The rights granted under the RHO-adRP License Agreement included certain patent rights covering IC-100, the Company's novel AAV gene therapy product candidate intended to treat RHO-adRP. In June 2018, the Company paid UFRF, on behalf of both Licensors, a $0.5 million upfront license issuance fee in connection with entry into the agreement, which was recorded as a research and development expense, as well as accrued patent prosecution expenses of approximately $30 thousand, which was recorded as a general and administrative expense. Under the agreement, the Company agreed to pay an annual license maintenance fee in the low double-digit thousands of dollars, which will be payable on an annual basis until the first commercial sale of a licensed product. In addition, the Company agreed to reimburse UFRF for the costs and expenses of patent prosecution and maintenance related to the licensed patent rights. The Company further agreed to pay UFRF, on behalf of both Licensors, up to an aggregate of $23.5 million if the Company achieves specified clinical, marketing approval and reimbursement approval milestones with respect to a licensed product and additionally, up to an aggregate of $70.0 million if the Company achieves specified commercial sales milestones with respect to a licensed product. The Company is also obligated to pay UFRF, on behalf of both Licensors, royalties at a low single-digit percentage of net sales of licensed products. Such royalties are subject to customary reductions for lack of patent coverage and loss of regulatory exclusivity. In addition, such royalties with respect to any licensed product in any country may be offset by a specified portion of any royalty payments actually paid by the Company in such country under third-party licenses for patent rights or other intellectual property rights that are necessary to manufacture, develop and commercialize the licensed product in such country. The Company's obligation to pay royalties under the RHO-adRP License Agreement will continue on a licensed product-by-licensed product and country-by-country basis until the latest of: • the expiration of the last-to-expire licensed patent rights covering a licensed product in the country of sale; • the expiration of regulatory exclusivity covering a licensed product in the country of sale; and • ten years from the first commercial sale of the applicable licensed product in the country of sale. Beginning on the earlier of (i) the calendar year following the first commercial sale of a licensed product and (ii) the first business day of 2031, the Company is also obligated to pay certain minimum royalties, not to exceed an amount in the low hundreds of thousands of dollars on an annual basis, which minimum royalties are creditable against the Company's royalty obligation with respect to net sales of licensed products due for the year in which the minimum royalty is paid. In addition, if the Company or an affiliate sublicenses any of the licensed patent rights to a third party, the Company will be obligated to pay UFRF, on behalf of both Licensors, a low double-digit percentage of the consideration received in exchange for such sublicense. If the Company receives a rare pediatric disease priority review voucher from the FDA in connection with obtaining marketing approval for a licensed product and the Company subsequently uses such priority review voucher in connection with a different product candidate, the Company will be obligated to pay UFRF, on behalf of both Licensors, aggregate payments in the low double-digit millions of dollars based on certain marketing approval and commercial sales milestones with respect to such other product candidate. If the Company sells such priority review voucher to a third party, it will be obligated to pay UFRF, on behalf of both licensors, a low double-digit percentage of any consideration received from such third party in connection with such sale. Unless earlier terminated by the Company, the RHO-adRP License Agreement will expire upon the expiration of the Company’s obligation to pay royalties to UFRF on net sales of licensed products. The Company may terminate the agreement at any time for any reason upon prior written notice to UFRF. Penn or UFRF may terminate the RHO-adRP License Agreement in the event of certain breaches by the Company or in the event of certain insolvency events regarding the Company. In addition to the exclusive license agreement, the Company and Penn also entered into to a Master-Sponsored Research Agreement (the "RHO-adRP MSRA"), facilitated by the Penn Center for Innovation. Under the RHO-adRP MSRA, the Company and Penn conducted several preclinical studies of IC-100, as well as a natural history study of RHO-adRP patients. The total amount of funding for the sponsored research covered by statements of work under the RHO-adRP MSRA that the Company has committed to date and is expecting to commit to is in the low single-digit millions of dollars. IC-200 Agreements with University of Pennsylvania and University of Florida Research Foundation In April 2019, the Company entered into an exclusive global license agreement (the "BEST1 License Agreement") with Penn and UFRF. The Company entered into the BEST1 License Agreement by exercising its exclusive option rights under an option agreement that it previously entered into with Penn and UFRF in October 2018. Under the BEST1 License Agreement, Penn and UFRF granted the Company a worldwide, exclusive license under specified patent rights and specified know-how and a worldwide, non-exclusive license under other specified know-how to research, develop, manufacture and commercialize certain AAV gene therapy products, including IC-200, for the treatment of Best disease and other BEST1- related IRDs. In May 2019, the Company paid Penn, for the benefit of the Licensors, a $0.2 million upfront license issuance fee, which was recorded as a research and development expense, and the Company paid UFRF accrued patent prosecution expenses of approximately $18 thousand, which was recorded as a general and administrative expense. The Company has also agreed to pay Penn, for the benefit of the Licensors, an annual license maintenance fee in the low double-digit thousands of dollars, which will be payable on an annual basis until the first commercial sale of a licensed product. In addition, the Company have agreed to pay Penn, for the benefit of the Licensors, a one-time patent grant fee in the low triple-digit thousands of dollars, upon the issuance of a U.S. patent that claims inventions disclosed in the licensed patent rights or know-how or inventions generated under certain related sponsored research agreements with Penn or UFRF, and that is exclusively licensed to the Company. Furthermore, it has agreed to reimburse Penn and UFRF for the costs and expenses of patent prosecution and maintenance related to the licensed patent rights. The Company has further agreed to pay Penn, for the benefit of the Licensors, up to an aggregate of $15.7 million if it achieves specified clinical, marketing approval and reimbursement approval milestones with respect to one licensed product, and up to an aggregate of an additional $3.1 million if it achieves these same milestones with respect to a different licensed product. In addition, it has agreed to pay Penn, for the benefit of the Licensors, up to an aggregate of $48.0 million if it achieves specified commercial sales milestones with respect to one licensed product, and up to an aggregate of an additional $9.6 million if it achieves these same milestones with respect to a different licensed product. The Company is also obligated to pay Penn, for the benefit of the Licensors, royalties at a low single-digit percentage of net sales of licensed products. Such royalties are subject to customary deductions, credits, and reductions for lack of patent coverage and loss of regulatory exclusivity. In addition, such royalties with respect to any licensed product in any country may be offset by a specified portion of any royalty payments actually paid by the Company in such country under third-party licenses to patent rights or other intellectual property rights that are necessary to research, develop, manufacture and commercialize the licensed product in such country. Its obligation to pay royalties under the BEST1 License Agreement will continue on a licensed product-by-licensed product and country-by-country basis until the latest of: • the expiration of the last-to-expire licensed patent rights covering the sale of the applicable licensed product in the country of sale; • the expiration of regulatory exclusivity covering the applicable licensed product in the country of sale; and • 10 years from the first commercial sale of the applicable licensed product in the country of sale. Beginning on the earlier of the calendar year following the first commercial sale of a licensed product and calendar year 2032, the Company is also obligated to pay certain minimum royalties, not to exceed an amount in the mid tens of thousands of dollars on an annual basis, which minimum royalties are creditable against its royalty obligation with respect to net sales of licensed products due in the year the minimum royalty is paid. If the Company or any of its affiliates sublicense any of the licensed patent rights to a third party, it will be obligated to pay Penn, for the benefit of the Licensors, a high single-digit to a mid ten's percentage of the consideration received in exchange for such sublicense, with the applicable percentage based upon the stage of development of the sublicensed product at the time it or the applicable affiliate enters into the sublicense. If the Company receives a rare pediatric disease priority review voucher from the FDA in connection with obtaining marketing approval for a licensed product and the Company subsequently uses such priority review voucher in connection with a different product candidate outside the scope of the BEST1 License Agreement, it will be obligated to pay Penn, for the benefit of the Licensors, aggregate payments in the low double-digit millions of dollars based on certain approval and commercial sales milestones with respect to such other product candidate. In addition, if it sells such a priority review voucher to a third party, it will be obligated to pay Penn, for the benefit of the Licensors, a high single-digit percentage of any consideration received from such third party in connection with such sale. The BEST1 License Agreement, unless earlier terminated by the Company or Penn or UFRF, will expire upon the expiration of the Company's obligation to pay royalties on net sales of licensed products. Before the effectiveness of an IND for a licensed product, the Company may terminate the BEST1 License Agreement with respect to such licensed product or in its entirety, at any time for any reason upon prior written notice to Penn and UFRF. Following the effectiveness of an IND for a licensed product, it may terminate the BEST1 License Agreement with respect to such licensed product by providing Penn prior written notice and a certification that it is ceasing all use, research and development and commercialization of such licensed product, subject to certain limited exceptions. It may also terminate the BEST1 License Agreement if Penn or UFRF materially breaches the BEST1 License Agreement and does not cure such breach within a specified cure period. Penn or UFRF may terminate the BEST1 License Agreement if the Company materially breaches the BEST1 License Agreement and does not cure such breach within a specified cure period, if it experiences a specified insolvency event, if it ceases to carry on the entirety of its business related to the licensed patent rights, if it ceases for more than four consecutive quarters to make any payment of earned royalties on net sales of licensed products following the commencement of commercialization thereof, unless such cessation is based on safety concerns that the Company is actively attempting to address, or if it, any of its affiliates or any of its sublicensees challenges or assists a third party in challenging the validity, scope, patentability, and/or enforceability of the licensed patent rights. If it materially breaches certain diligence obligations under the BEST1 License Agreement with respect to only one licensed product, then Penn and UFRF may only terminate its rights and licenses under the BEST1 License Agreement for such licensed product, but not for other licensed products. In addition to the exclusive license agreement, the Company and Penn also entered into a Master-Sponsored Research Agreement (the "BEST1 MSRA"), facilitated by the Penn Center for Innovation. Under the BEST1 MSRA, the Company and Penn are conducting preclinical studies of IC-200, as well as natural history studies of patients with BEST1 -related IRDs. The total amount of funding for the sponsored research covered by statements of work under the BEST1 MSRA that the Company has committed to date and expects to commit to is in the low single-digit millions of dollars. License Agreement with University of Massachusetts for the miniCEP290 Program In July 2019, the Company entered into the miniCEP290 License Agreement with UMass by exercising its exclusive option rights under an option agreement and a sponsored research agreement that it previously entered into with UMass in February 2018. Under the miniCEP290 License Agreement, UMass granted it a worldwide, exclusive license under specified patent rights and specified biological materials and a non-exclusive license under specified know-how to make, have made, use, offer to sell, sell, have sold and import products for the treatment of diseases associated with mutations in the CEP290 gene, including LCA10. In July 2019, the Company issued to UMass 75,000 shares of its common stock following execution of the miniCEP290 License Agreement pursuant to an exemption from registration afforded by Section 4(a)(2) of the Securities Act. In September 2019, it paid UMass a $0.4 million upfront license fee, which was recorded as a research and development expense, and it paid UMass accrued patent prosecution expenses of approximately $18 thousand, which was recorded as a general and administrative expense. The Company has also agreed to pay UMass an annual license maintenance fee in the low double-digit thousands of dollars, which will be payable on an annual basis until the expiration of the royalty term for the licensed products. Furthermore, it has agreed to reimburse UMass for the costs and expenses of patent prosecution and maintenance related to the licensed patent rights. The Company has further agreed to pay UMass up to an aggregate of $14.75 million in cash and issue up to 75,000 shares of its common stock if it achieves specified clinical and regulatory milestones with respect to a licensed product. In addition, the Company has agreed to pay UMass up to an aggregate of $48.0 million if it achieves specified commercial sales milestones with respect to a licensed product. The Company is also obligated to pay UMass royalties at a low single-digit percentage of net sales of licensed products. Its obligation to pay royalties under the miniCEP290 License Agreement will continue on a licensed product-by-licensed product and country-by-country basis until the later of: (a) the expiration of the last-to-expire licensed patent rights covering the sale of the applicable licensed product in the country of sale, or (b) 10 years from the first commercial sale of the applicable licensed product in the country of sale. Beginning with the calendar year following receipt of marketing approval for a licensed product, it is also obligated to pay certain minimum royalties, not to exceed an amount in the mid-double-digit thousands of dollars on an annual basis, which minimum royalties are creditable against its royalty obligation with respect to net sales of licensed products due in the year the minimum royalty is paid. If the Company or any of its affiliates sublicenses any of the licensed patent rights or know-how to a third party, it will be obligated to pay UMass a high single-digit to a mid-tens percentage of the consideration received in exchange for such sublicense, with the applicable percentage based upon the stage of development of the licensed products at the time it or the applicable affiliate enters into the sublicense. If the Company receives a rare pediatric disease priority review voucher from the FDA in connection with obtaining marketing approval for a licensed product, and it subsequently uses such priority review voucher in connection with a different product candidate outside the scope of the miniCEP290 License Agreement, it will be obligated to pay UMass a low-tens percentage of the fair market value of the priority review voucher at the time of approval of such product candidate and a low-twenties percentage of the fair market value of the priority review voucher at the time of achievement of a specified commercial sales milestone for such other product candidate. In addition, if it sells such a priority review voucher to a third party, it will be obligated to pay UMass a low-thirties percentage of any consideration received from such third party in connection with such sale. The miniCEP290 License Agreement, unless earlier terminated by the Company or UMass, will expire upon the expiration of its obligation to pay royalties to UMass on net sales of licensed products. The Company may terminate the miniCEP290 License Agreement at any time for any reason upon prior written notice to UMass. It may also terminate the miniCEP290 License Agreement if UMass materially breaches the miniCEP290 License Agreement and does not cure such breach within a specified cure period. UMass may terminate the miniCEP290 License Agreement if the Company materially breaches the miniCEP290 License Agreement and does not cure such breach within a specified cure period. |
Financing Agreement with Novo H
Financing Agreement with Novo Holdings A/S | 12 Months Ended |
Dec. 31, 2020 | |
Related Party Transactions [Abstract] | |
Financing Agreement with Novo Holdings A/S | Financing Agreement with Novo Holdings A/S In May 2013, the Company entered into a Purchase and Sale Agreement (the "Novo Agreement") with Novo Holdings A/S ("Novo" and formerly Novo A/S), pursuant to which the Company obtained financing in three tranches in an aggregate amount equal to $125.0 million in return for the sale to Novo of aggregate royalties of a mid-single digit percentage on worldwide sales of (a) Fovista, (b) certain Fovista-related Products ("Fovista-Related Products"), and (c) certain other antagonists of platelet-derived growth factor ("PDGF"), if any, that the Company initiated development activities for prior to the fifth anniversary of the Novo Agreement (collectively, the "Anti-PDGF Royalty Products"). The Company received the cash payments from Novo in each of May 2013, January 2014 and November 2014. the Company received cash payments of $41.7 million, or $125.0 million in the aggregate. The $125.0 million in aggregate proceeds from the three financing tranches under the Novo Agreement represented the full funding available under the Novo Agreement, and prior to December 31, 2018, was recorded as a royalty purchase liability on the Company's Balance Sheet, in accordance with ASC 730, Research and Development . Because there was a significant related party relationship between the Company and Novo, the Company treated its obligation to make royalty payments under the Novo Agreement as an implicit obligation to repay the funds advanced by Novo. On December 31, 2018, the Company and Novo entered into a letter agreement (the "Novo Termination Agreement"), that terminated the Novo Agreement. As a result of the Novo Termination Agreement, Novo relinquished all rights to receive royalties based on net sales of Anti-PDGF Royalty Products. In exchange, the Company agreed to forbear from any future filing, prosecution, maintenance or enforcement of any intellectual property rights related to Anti-PDGF Royalty Products, and from granting any third party any right or license in any such intellectual property rights or clinical study data generated by or on behalf of the Company, its affiliates, licensees or sublicensees, for the development, manufacture or commercialization of Anti-PDGF Royalty Products or any other antagonists of PDGF. The foregoing restriction does not apply to gene therapies (so long as the Company does not grant any rights or licenses to intellectual property or clinical study data related to Fovista or Fovista-Related Products). The Company further agreed, until December 31, 2028, not to develop, manufacture, seek or obtain regulatory approval for, or commercialize any Anti-PDGF Royalty Products without the prior written consent of Novo. The foregoing restriction does not apply to any gene therapies (so long as the Company does not utilize data related to Fovista or Fovista-Related Products). |
Property and Equipment
Property and Equipment | 12 Months Ended |
Dec. 31, 2020 | |
Property, Plant and Equipment [Abstract] | |
Property and Equipment | Property and Equipment Property and equipment as of December 31, 2020 and 2019 were as follows: Useful Life December 31, December 31, Manufacturing and clinical equipment 7 - 10 $ 47 $ 47 Computer, software and other office equipment 5 933 933 980 980 Accumulated depreciation (954) (807) Property and equipment, net $ 26 $ 173 For the years ended December 31, 2020, 2019 and 2018, depreciation expense was $0.1 million, $0.2 million and $0.2 million, respectively. |
Income Taxes
Income Taxes | 12 Months Ended |
Dec. 31, 2020 | |
Income Tax Disclosure [Abstract] | |
Income Taxes | Income Taxes The Company utilizes the liability method of accounting for deferred income taxes. Under this method, deferred tax assets and liabilities are recognized for the expected future tax consequences of temporary differences between the carrying amounts and the tax basis of assets and liabilities. A valuation allowance is established against deferred tax assets when, based on the weight of available evidence, it is more likely than not that some or all of the deferred tax assets will not be realized. The Company's policy is to record interest and penalties on uncertain tax positions as income tax expense. Deferred income taxes reflect the net effects of temporary differences between the carrying amounts of assets and liabilities for financial reporting purposes and the amounts used for income tax purposes. A reconciliation of the statutory U.S. federal rate to the Company's effective tax rate is as follows: Years ended December 31, 2020 2019 2018 Percent of pre-tax income: U.S. federal statutory income tax rate 21.0 % 21.0 % 21.0 % State taxes, net of federal benefit 14.8 % 10.9 % 18.5 % Permanent items 0.2 % (1.0) % 4.0 % Impact of state rate changes 0.1 % 0.1 % (0.2) % Research and development credit 3.1 % 2.8 % (2.4) % Change in valuation allowance (34.8) % (33.6) % (42.6) % Effective income tax rate 4.4 % 0.2 % (1.7) % The components of income tax benefit are as follows: Years ended December 31, 2020 2019 2018 Current: Federal $ — $ — $ — State (3,695) (111) (1,063) Deferred: Federal — — — State — — — Income tax benefit $ (3,695) $ (111) $ (1,063) Significant components of the Company's deferred tax assets (liabilities) for 2020 and 2019 consist of the following: As of December 31, 2020 2019 Deferred tax assets (liabilities) License and technology payments $ 7,479 $ 7,116 Share-based compensation 23,266 22,131 Accrued expenses 427 362 Right-of-use asset (40) (163) Lease obligation 38 162 Depreciation 6 (18) Federal and state net operating loss carryforwards 130,524 105,375 Research and development credits 9,959 7,353 Other 7 6 Deferred income tax assets 171,666 142,324 Valuation allowance (171,666) (142,324) Net deferred tax assets $ — $ — The Company considers whether it is more likely than not that some portion or all of the deferred tax assets will not be realized. The ultimate realization of deferred tax assets is dependent upon the generation of taxable income during the periods in which the temporary differences representing net future deductible amounts become deductible. The Company incurred tax losses in 2020 and 2019. The Company has carried forward its federal and state tax losses due to the inability of carryback claims. Federal NOLs incurred prior to 2018 will begin to expire in 2034 if not utilized. Post 2017 Federal NOLs have an unlimited life. The state NOLs are expected to begin to expire in 2028. Due to the Company's history of losses and lack of other positive evidence to support taxable income, the Company has recorded a valuation allowance against those remaining deferred tax assets that are not expected to be realized. As of December 31, 2020, the Company has federal NOL carryforwards of approximately $445.0 million. For the year ended December 31, 2020, the Company recorded a benefit from income taxes of $3.7 million to reflect the settlement of a local tax audit and the associated decrease in the Company's related uncertain tax position, including interest and penalties. For the years ended December 31, 2019 and 2018, the Company recorded an income tax benefit of $0.1 million and $1.1 million, respectively, due primarily to reflect the settlements of local tax audits. During 2019, the Company received $1.8 million related to the refund of the aforementioned AMT credits and recorded a tax receivable of $1.8 million to reflect the refund of AMT credits it expects to receive over the next three years. On March 27, 2020, in response to the COVID-19 pandemic, the U.S. Congress enacted the Coronavirus Aid, Relief, and Economic Security Act ("CARES Act"). The CARES Act provides numerous tax provisions and other stimulus measures, including the immediate refund of minimum tax credits. The Company has recorded a current tax receivable of approximately $1.8 million in anticipation of this refund. During 2020, the Company applied for a refund of the remaining AMT credits of $1.8 million and anticipates receiving this refund in 2021. The Company generated net income before income taxes for the year ended December 31, 2018. This income was due to the Novo Termination Agreement. With respect to the remaining deferred tax assets, except for the AMT credits previously discussed above, there was no change in the amount of assets realizable at December 31, 2020. Pursuant to ASC 740, Income Taxes , the Company routinely evaluates the likelihood of success if challenged on income tax positions claimed on its income tax returns. During the year ended December 31, 2020, the Company reduced its uncertain tax liability by approximately $5.2 million following the settlement of state tax audits. The Company increased certain deferred tax assets by $0.3 million and increased the corresponding valuation allowance by an equivalent amount. The Company's position with respect to uncertain tax positions is set forth below: Opening balance $ 12,227 Gross amount of increases in unrecognized tax benefits during the period - current year provisions — Gross amount of increases in unrecognized tax benefits during the period - prior year provisions — Gross amount of decreases in unrecognized tax benefits during the period - other (659) Decreases due to settlement with tax authorities during the period (4,845) Reduction of unrecognized tax benefits due to expiration of the state of limitations during the period — Closing Balance $ 6,723 The Company will continue to evaluate its ability to realize its deferred tax assets on a periodic basis and will adjust such amounts in light of changing facts and circumstances including, but not limited to, future projections of taxable income, tax legislation, rulings by relevant tax authorities, the progress of ongoing tax audits and the regulatory approval of product candidates currently under development. Any additional changes to the valuation allowance recorded on deferred tax assets in the future would impact the Company’s income taxes. |
Operating Leases
Operating Leases | 12 Months Ended |
Dec. 31, 2020 | |
Leases [Abstract] | |
Operating Leases | Operating Leases The Company's principal executive offices was previously at One Penn Plaza, New York, NY. The term of this lease expired in December 2020. Effective December 1, 2020, the Company changed the location of its principal executive offices to Five Penn Plaza, New York, NY. The Company entered into an office service agreement with Regus Management Group, LLC for use of office space at this location. This agreement currently expires in May 2021. The Company leases office space located in Cranbury, New Jersey under non-cancelable operating lease arrangements. The lease for the Company's Cranbury office commenced in February 2020 and expires in February 2023. In December 2020, the Company entered into a lease agreement for office space located in Parsippany, New Jersey. The Company will recognize a right-to-use asset and lease liability when the term of the lease commences, which is expected to be in early 2021. The term of the lease for the Company's Parsippany office will expire in August 2023. For the years ended December 31, 2020, 2019 and 2018, lease and rent expense was $0.6 million, $1.0 million, and $0.8 million, respectively. Cash paid from operating cash flows for amounts included in the measurement of lease liabilities was $0.6 million and $1.0 million for the years ended December 31, 2020 and 2019, respectively. At December 31, 2020, the Company's operating leases had a weighted average remaining lease term of 2.1 years and a weighted average estimated incremental borrowing rate of 3.4% . The following presents the maturity of the Company's operating lease liabilities as of December 31, 2020: December 31, 2020 2021 $ 57 2022 58 2023 5 Total minimum lease payments 120 Less imputed interest (5) Present value of minimum lease payments 115 Less current portion 54 Total long-term operating lease liabilities $ 61 |
Commitments and Contingencies
Commitments and Contingencies | 12 Months Ended |
Dec. 31, 2020 | |
Commitments and Contingencies Disclosure [Abstract] | |
Commitments and Contingencies | Commitments and Contingencies Zimura - Archemix Corp. The Company is party to an agreement with Archemix Corp., or Archemix, under which the Company in-licensed rights in certain patents, patent applications and other intellectual property related to Zimura and pursuant to which the Company may be required to pay sublicense fees and make milestone payments (the "C5 License Agreement"). For each anti-C5 aptamer product that the Company may develop under the agreement, including Zimura, the Company is obligated to make additional payments to Archemix of up to an aggregate of $50.5 million if the Company achieves specified development, clinical and regulatory milestones, with $24.5 million of such payments relating to a first indication, $23.5 million of such payments relating to second and third indications and $2.5 million of such payments relating to sustained delivery applications. Under the C5 License Agreement, the Company is also obligated to make additional payments to Archemix of up to an aggregate of $22.5 million if the Company achieves specified commercial milestones based on net product sales of all anti-C5 licensed products. The Company is also obligated to pay Archemix a double-digit percentage of specified non-royalty payments the Company may receive from any sublicensee of its rights under the C5 License Agreement. The Company is not obligated to pay Archemix a running royalty based on net product sales in connection with the C5 License Agreement. IC-100 - University of Florida Research Foundation and the University of Pennsylvania Under its exclusive license agreement with UFRF and Penn for rights to IC-100, the Company is obligated to make payments to UFRF, for the benefit of the Licensors, of up to an aggregate of $23.5 million if the Company achieves specified clinical, marketing approval and reimbursement approval milestones with respect to a licensed product and up to an aggregate of an additional $70.0 million if the Company achieves specified commercial sales milestones with respect to a licensed product. The Company is also obligated to pay UFRF, for the benefit of the Licensors, a low single-digit percentage of net sales of licensed products. The Company is also obligated to pay UFRF, for the benefit of the Licensors, a double-digit percentage of specified non-royalty payments the Company may receive from any third-party sublicensee of the licensed patent rights. Further, if the Company receives a rare pediatric disease priority review voucher from the FDA in connection with obtaining marketing approval for a licensed product and the Company subsequently uses such priority review voucher in connection with a different product candidate, the Company will be obligated to pay UFRF, for the benefit of the Licensors, aggregate payments in the low double-digit millions of dollars based on certain approval and commercial sales milestones with respect to such other product candidate. In addition, if the Company sells such a priority review voucher to a third party, the Company will be obligated to pay UFRF, for the benefit of the Licensors, a low double-digit percentage of any consideration received from such third party in connection with such sale. IC-200 - University of Pennsylvania and the University of Florida Research Foundation Under its exclusive license agreement with Penn and UFRF for rights to IC-200, the Company is obligated to make payments to Penn, for the benefit of the Licensors, of up to an aggregate of $15.7 million if the Company achieves specified clinical, marketing approval and reimbursement approval milestones with respect to one licensed product and up to an aggregate of an additional $3.1 million if the Company achieves these same milestones with respect to a different licensed product. In addition, the Company is obligated to make payments to Penn, for the benefit of the Licensors, of up to an aggregate of $48.0 million if the Company achieves specified commercial sales milestones with respect to one licensed product and up to an aggregate of an additional $9.6 million if the Company achieves these same milestones with respect to a different licensed product. The Company is also obligated to pay Penn, for the benefit of the Licensors, a low single-digit percentage of net sales of licensed products. The Company is also obligated to pay Penn, for the benefit of the Licensors, a high single-digit to a mid-ten's percentage of specified non-royalty payments the Company may receive from any third-party sublicensee of the licensed patent rights, with the applicable percentage based upon the stage of development of the sublicensed product at the time the Company enters into the sublicense. Further, if the Company receives a rare pediatric disease priority review voucher from the FDA in connection with obtaining marketing approval for a licensed product and the Company subsequently uses such priority review voucher in connection with a different product candidate outside the scope of the agreement, the Company will be obligated to pay Penn, for the benefit of the Licensors, aggregate payments in the low double-digit millions of dollars based on certain approval and commercial sales milestones with respect to such other product candidate. In addition, if the Company sells such a priority review voucher to a third party, the Company will be obligated to pay Penn, for the benefit of the Licensors, a high single-digit percentage of any consideration received from such third party in connection with such sale. miniCEP290 Program - University of Massachusetts Under the miniCEP290 License Agreement, the Company is obligated to pay UMass up to an aggregate of $14.75 million in cash and issue up to 75,000 shares of common stock of the Company if the Company achieves specified clinical and regulatory milestones with respect to a licensed product. In addition, the Company is obligated to pay UMass up to an aggregate of $48.0 million if the Company achieves specified commercial sales milestones with respect to a licensed product. The Company is also obligated to pay UMass royalties at a low single-digit percentage of net sales of licensed products. If the Company or any of its affiliates sublicenses any of the licensed patent rights or know-how to a third party, the Company will be obligated to pay UMass a high single-digit to a mid-tens percentage of the consideration received in exchange for such sublicense, with the applicable percentage based upon the stage of development of the licensed products at the time the Company or the applicable affiliate enters into the sublicense. If the Company receives a priority review voucher from the FDA in connection with obtaining marketing approval for a licensed product, and the Company subsequently uses such priority review voucher in connection with a different product candidate outside the scope of the agreement, the Company will be obligated to pay UMass a low-tens percentage of the fair market value of the priority review voucher at the time of approval of such product candidate and a low-twenties percentage of the fair market value of the priority review voucher at the time of achievement of a specified commercial sales milestone for such product candidate. In addition, if the Company sells such a priority review voucher to a third party, the Company will be obligated to pay UMass a low-thirties percentage of any consideration received from such third party in connection with such sale. IC-500 - Former Equityholders of Inception 4 Under the Inception 4 Merger Agreement, the Company is obligated to make payments to the former equityholders of Inception 4 of up to an aggregate of $105 million, subject to the terms and conditions of the Inception 4 Merger Agreement, if the Company achieves certain specified clinical and regulatory milestones with respect to a product candidate from its HtrA1 inhibitor program, including IC-500, with $45 million of such potential payments relating to GA and $60 million of such potential payments relating to wet AMD. Under the Inception 4 Merger Agreement, the Company does not owe any commercial milestones or royalties based on net sales. The future milestone payments will be payable in the form of shares of the Company's common stock, calculated based on the price of its common stock over a five-trading day period preceding the achievement of the relevant milestone, unless and until the issuance of such shares would, together with all other shares issued in connection with the Inception 4 Merger, exceed an overall maximum limit of approximately 7.2 million shares, which is equal to 19.9% of the number of issued and outstanding shares of the Company's common stock as of the close of business on the business day prior to the closing date of the Inception 4 Merger, and will be payable in cash thereafter. The Inception 4 Merger Agreement also includes customary indemnification obligations to the former equityholders of Inception 4, including for breaches of the representations and warranties, covenants and agreements of the Company and its subsidiaries (other than Inception 4) in the Inception 4 Merger Agreement. Employment Contracts The Company also has letter agreements with certain employees that require the funding of a specific level of payments if certain events, such as a termination of employment in connection with a change in control or termination of employment by the employee for good reason or by the Company without cause, occur. Contract Service Providers In addition, in the course of normal business operations, the Company has agreements with contract service providers to assist in the performance of the Company’s research and development and manufacturing activities. Expenditures to CROs and CDMOs represent significant costs in preclinical and clinical development. Subject to required notice periods and the Company’s obligations under binding purchase orders and any cancellation fees that the Company may be obligated to pay, the Company can elect to discontinue the work under these agreements at any time. Legal Proceedings On January 11, 2017, a putative class action lawsuit was filed against the Company and certain of its current and former executive officers in the United States District Court for the Southern District of New York, captioned Frank Micholle v. IVERIC bio, Inc., et al., No. 1:17-cv-00210. On March 9, 2017, a related putative class action lawsuit was filed against the Company and the same group of its current and former executive officers in the United States District Court for the Southern District of New York, captioned Wasson v. IVERIC bio, Inc., et al., No. 1:17-cv-01758. These cases were consolidated on March 13, 2018. On June 4, 2018, the lead plaintiff filed a consolidated amended complaint (the “CAC”). The CAC purports to be brought on behalf of shareholders who purchased the Company’s common stock between March 2, 2015 and December 12, 2016. The CAC generally alleges that the Company and certain of its officers violated Sections 10(b) and/or 20(a) of the Securities Exchange Act of 1934 and Rule 10b-5 promulgated thereunder by making allegedly false and/or misleading statements concerning the results of the Company’s Phase 2b trial and the prospects of the Company’s Phase 3 trials for Fovista in combination with anti-VEGF agents for the treatment of wet AMD. The CAC seeks unspecified damages, attorneys’ fees, and other costs. The Company and individual defendants filed a motion to dismiss the CAC on July 27, 2018. On September 18, 2019, the court issued an order dismissing some, but not all, of the allegations in the CAC. On November 18, 2019, the Company and the individual defendants filed an answer to the complaint. On June 12, 2020, the lead plaintiff filed a motion for class certification. On August 11, 2020, the defendants filed a notice of non-opposition to lead plaintiff's motion for class certification. This case is currently in the discovery phase. On February 7, 2018, a shareholder derivative action was filed against the members of the Company’s Board of Directors in the New York Supreme Court Commercial Division, captioned Cano v. Guyer, et al., No. 650601/2018. The complaint alleges that the defendants breached their fiduciary duties to the Company by adopting a compensation plan that overcompensates the non-employee members of the Board relative to boards of companies of comparable market capitalization and size. The complaint also alleges that the defendants were unjustly enriched as a result of the alleged conduct. The complaint purports to seek unspecified damages, on behalf of the Company, attorneys’ fees, and other costs, as well as an order directing the Company to reform and improve its corporate governance and internal procedures to comply with applicable laws. The Company filed a motion to dismiss this case on May 14, 2018. On June 4, 2018, the plaintiff filed an amended complaint. On June 25, 2018, the Company filed a renewed motion to dismiss this case. On December 3, 2018, the parties filed a stipulation of settlement that contemplates that the Company will adopt certain compensation-related governance reforms and does not obligate the defendants or the Company to pay any monetary damages. The court approved the settlement at a hearing on March 12, 2019. As part of the settlement, in April 2019 the Company paid $0.3 million in fees and costs to plaintiff's counsel. As contemplated by the settlement, the Company's board of directors adopted certain compensation-related governance reforms, including a non-employee director compensation policy, which its stockholders approved on May 15, 2019 at its 2019 annual meeting. On August 31, 2018, a shareholder derivative action was filed against current and former members of the Company's Board of Directors and certain current and former officers of the Company in the United States District Court for the Southern District of New York, captioned Luis Pacheco v. David R. Guyer, et al., Case No. 1:18-cv-07999. The complaint, which is based substantially on the facts alleged in the CAC, alleges that the defendants breached their fiduciary duties to the Company and wasted the Company's corporate assets by failing to oversee the Company's business, and also alleges that the defendants were unjustly enriched as a result of the alleged conduct, including through receipt of bonuses, stock options and similar compensation from the Company, and through sales of the Company's stock between March 2, 2015 and December 12, 2016. The complaint purports to seek unspecified damages on the Company's behalf, attorneys’ fees, and other costs, as well as an order directing the Company to reform and improve its corporate governance and internal procedures to comply with applicable laws, including submitting certain proposed amendments to the Company's corporate charter, bylaws and corporate governance policies for vote by the Company's stockholders. On December 14, 2018, the Company filed a motion to dismiss the complaint. On September 19, 2019, the court denied its motion to dismiss this complaint. This matter was subsequently referred to a special litigation committee of the Company's board of directors. On February 18, 2020, the Company filed an answer to the complaint. The Company and the plaintiff agreed to stay this litigation while the special litigation committee conducts its investigation. On May 4, 2020, the court approved the stipulation and stayed the litigation through November 1, 2020. By agreement of the parties, the court has since extended the stay through March 13, 2021. The Company also entered into tolling agreements with the defendant directors to December 2021. On October 16, 2018, the Company’s board of directors received a shareholder demand to investigate and commence legal proceedings against certain members of the Company’s board of directors. The demand alleges facts that are substantially similar to the facts alleged in the CAC and the Pacheco complaint and asserts claims that are substantially similar to the claims asserted in the Pacheco complaint. On January 30, 2019, the Company’s board of directors received a second shareholder demand from a different shareholder to investigate and commence legal proceedings against certain current and former members of the Company’s board of directors based on allegations that are substantially similar to the allegations contained in the first demand letter. These shareholder demands have been referred to a demand review committee of the Company's board of directors. The Company has entered into tolling agreements with the directors named in the demands to December 2021. The Company denies any and all allegations of wrongdoing and intends to vigorously defend against these lawsuits. The Company is unable, however, to predict the outcome of these matters at this time. Moreover, any conclusion of these matters in a manner adverse to the Company and for which it incurs substantial costs or damages not covered by the Company's directors’ and officers’ liability insurance would have a material adverse effect on its financial condition and business. In addition, the litigation could adversely impact the Company's reputation and divert management’s attention and resources from other priorities, including the execution of its business plan and strategies that are important to the Company's ability to grow its business, any of which could have a material adverse effect on the Company's business. |
Stock-Based Compensation and Co
Stock-Based Compensation and Compensation Plans | 12 Months Ended |
Dec. 31, 2020 | |
Share-based Payment Arrangement [Abstract] | |
Stock-Based Compensation and Compensation Plans | Stock-Based Compensation and Compensation Plans The Company adopted its 2007 Stock Incentive Plan (the "2007 Plan") for employees, non-employee directors and consultants for the purpose of advancing the interests of the Company's stockholders by enhancing its ability to attract, retain and motivate persons who are expected to make important contributions to the Company. The 2007 Plan provided for the granting of stock option awards, RSUs, and other stock-based and cash-based awards. Following the effectiveness of the 2013 Stock Incentive Plan described below in connection with the closing of the Company's initial public offering, the Company is no longer granting additional awards under the 2007 Plan. In August 2013, the Company's board of directors adopted, and the Company's stockholders approved, the 2013 Stock Incentive Plan (the "2013 Plan"), which became effective immediately prior to the closing of the Company's initial public offering. In June 2015, the Company’s board of directors adopted a first amendment to the 2013 Plan. The 2013 Plan provides for the grant of incentive stock options, nonstatutory stock options, stock appreciation rights, RSUs, restricted stock awards and other stock-based awards. Upon the effectiveness of the 2013 Plan, the number of shares of the Company's common stock that were reserved for issuance under the 2013 Plan was the sum of (1) a number of shares (up to a maximum of approximately 3,359,641 shares) that is equal to the sum of 739,317 shares (the number of shares of the Company's common stock then available for issuance under the 2007 Plan), and such number of shares of the Company's common stock that are subject to outstanding awards under the 2007 Plan that expire, terminate or are otherwise surrendered, canceled, forfeited or repurchased by the Company at their original issuance price pursuant to a contractual repurchase right, plus (2) an annual increase, to be added the first business day of each fiscal year, beginning with the fiscal year ending December 31, 2014 and continuing until, and including, the fiscal year ending December 31, 2023, equal to the lowest of 2,542,372 shares of the Company's common stock, 4% of the number of shares of the Company's common stock outstanding on the first day of the fiscal year and an amount determined by its board of directors. The Company's employees, officers, directors, consultants and advisors are eligible to receive awards under the 2013 Plan. However, incentive stock options may only be granted to employees of the Company. Annual increases under the evergreen provisions of the 2013 Plan have resulted in the addition of an aggregate of approximately 13,081,000 additional shares to the 2013 Plan, including for 2021, an increase of approximately 2,542,000 shares. As of December 31, 2020, the Company had approximately 421,000 shares available for grant under the 2013 Plan. In October 2019, the Company's board of directors adopted the 2019 Inducement Stock Incentive Plan (the “2019 Inducement Plan”) to reserve 1,000,000 shares of its common stock to be used exclusively for grants of awards to individuals that were not previously employees or directors of the Company as a material inducement to such individuals’ entry into employment with the Company within the meaning of Rule 5635(c)(4) of the Nasdaq Listing Rules. In March 2020, the Company's board of directors amended the 2019 Inducement Plan to reserve an additional 1,000,000 shares of its common stock for issuance under the plan and in February 2021, the Company's board of directors further amended the 2019 Inducement Plan to reserve an additional 600,000 shares of its common stock for issuance under the plan. The terms and conditions of the 2019 Inducement Plan are substantially similar to those of the 2013 Plan. As of December 31, 2020, the Company had approximately 555,000 shares available for grant under the 2019 Inducement Plan. In April 2016, the board of directors adopted the ESPP pursuant to which the Company may sell up to an aggregate of 1,000,000 shares of common stock. The ESPP was approved by the Company’s stockholders in June 2016. The ESPP allows eligible employees to purchase common stock at a price per share equal to 85% of the lower of the fair market value of the common stock at the beginning or end of each six month offering period during the term of the ESPP. The first offering period began in September 2016. A summary of the stock option activity, weighted average exercise prices, options outstanding and exercisable as of December 31, 2020, 2019 and 2018 is as follows (in thousands except weighted average exercise price): Years ended December 31, 2020 2019 2018 Common Weighted Common Weighted Common Weighted Outstanding, December 31, 2019 6,780 $ 10.89 5,903 $ 13.72 5,284 $ 19.58 Granted 2,630 $ 6.10 1,610 $ 4.26 1,293 $ 1.67 Exercised (109) $ 2.89 (80) $ 2.66 — $ — Expired or forfeited (373) $ 19.49 (653) $ 21.16 (674) $ 36.54 Outstanding, December 31, 2020 8,928 $ 9.22 6,780 $ 10.89 5,903 $ 13.72 Years ended December 31, 2020 2019 2018 Options exercisable at December 31, 2020 4,462 3,317 2,709 Weighted average grant date fair value (per share) of options granted during the period $ 13.51 $ 3.37 $ 1.20 As of December 31, 2020, there were approximately 8,526,000 options outstanding, net of estimated forfeitures, that had vested or are expected to vest. The weighted-average exercise price of these options was $9.42 per option; the weighted-average remaining contractual life of these options was 7.6 years; and the aggregate intrinsic value of these options was approximately $20.6 million. A summary of the stock options outstanding and exercisable as of December 31, 2020 is as follows (in thousands except exercise prices and weighted average exercise price): December 31, 2020 Options Outstanding Options Exercisable Range of Exercise Prices Total Weighted Weighted Number Weighted $1.13-$2.90 1,352 7.7 $ 1.68 808 $ 1.79 $2.91-$3.18 2,053 7.5 $ 2.99 1,180 $ 2.94 $3.19-$4.88 1,790 7.7 $ 4.24 1,022 $ 4.39 $4.89-$8.77 2,491 9.6 $ 6.64 211 $ 5.24 $8.78-$73.22 1,242 3.8 $ 40.08 1,241 $ 40.08 8,928 7.6 $ 9.22 4,462 $ 13.51 Aggregate Intrinsic Value $ 20,553 $ 11,741 Cash proceeds from, and the aggregate intrinsic value of, stock options exercised during the years ended December 31, 2020, 2019 and 2018, respectively, were as follows: Years ended December 31, 2020 2019 2018 Cash proceeds from options exercised $ 314 $ 192 $ — Aggregate intrinsic value of options exercised $ 393 $ 135 $ — In connection with stock option awards granted to employees, the Company recognized approximately $4.7 million, $5.9 million and $7.6 million in share-based compensation expense during the years ended December 31, 2020, 2019 and 2018, respectively, net of expected forfeitures. As of December 31, 2020, there were approximately $8.6 million of unrecognized compensation costs, net of estimated forfeitures, related to stock option awards granted to employees, which are expected to be recognized over a remaining weighted average period of 5.8 years. In connection with stock option awards granted to consultants, the Company recognized approximately $0.2 million, $0.3 million and $0.2 million in share-based compensation expense during the years ended December 31, 2020, 2019 and 2018, respectively, net of expected forfeitures. As of December 31, 2020, there were approximately $0.1 million of unrecognized compensation costs, net of estimated forfeitures, related to stock option awards granted to consultants, which are expected to be recognized over a remaining weighted average period of 2.4 years. The following table presents a summary of the Company's outstanding RSU awards granted as of December 31, 2020 (in thousands except weighted average grant-date fair value): Restricted Weighted Average Outstanding, December 31, 2019 1,481 $ 7.79 Awarded 1,232 $ 6.06 Vested (657) $ 7.20 Forfeited (98) $ 6.74 Outstanding, December 31, 2020 1,958 $ 6.95 As of December 31, 2020, there were approximately 1,725,000 RSUs outstanding, net of estimated forfeitures, that are expected to vest. The weighted-average fair value of these RSUs was $5.25 per share; and the aggregate intrinsic value of these RSUs was approximately $11.9 million. In connection with RSUs granted to employees, the Company recognized approximately $3.3 million, $3.0 million and $3.3 million in share-based compensation expense during the years ended December 31, 2020, 2019 and 2018, respectively, net of expected forfeitures. As of December 31, 2020, there was approximately $14.4 million of unrecognized compensation costs, net of estimated forfeitures, related to RSUs granted to employees, which are expected to be recognized over a remaining weighted average period of 2.0 years. The total fair value of the RSUs that vested during the year ended December 31, 2020 was $4.6 million. In connection with RSUs granted to consultants, the Company recognized approximately $0.1 million in share-based compensation expense during the year ended December 31, 2020, net of expected forfeitures. In connection with RSUs granted to consultants, the Company recognized approximately a de minimis amount in share-based compensation expense during the year ended December 31, 2019. As of December 31, 2020, there were approximately $0.1 million of unrecognized compensation costs, net of estimated forfeitures, related to RSUs granted to consultants, which are expected to be recognized over a remaining weighted average period of 2.9 years. In connection with the ESPP made available to employees, the Company recognized approximately $0.1 million and $0.1 million of share-based compensation expense during the years ended December 31, 2020 and December 31, 2019, respectively, net of expected forfeitures. As of December 31, 2020, there was a de minimis amount of unrecognized compensation costs, net of estimated forfeitures, related to the ESPP, which are expected to be recognized over 0.2 years. There were 76,402 shares of common stock issued under the ESPP during the year ended December 31, 2020. Cash proceeds from ESPP purchases were approximately $147 thousand during the year ended December 31, 2020. There were 70,466 shares of common stock issued under the ESPP during the year ended December 31, 2019. As of December 31, 2020, 805,361 shares were available for future purchases under the ESPP. |
Employee Benefit Plan
Employee Benefit Plan | 12 Months Ended |
Dec. 31, 2020 | |
Retirement Benefits [Abstract] | |
Employee Benefit Plan | Employee Benefit PlanThe Company maintains a defined contribution 401(k) plan available to employees. Employee contributions are voluntary and are determined on an individual basis, limited by the maximum amounts allowable under federal tax regulations. The Company's matching contributions to employees totaled approximately $0.3 million, $0.2 million and $0.2 million during the years ended December 31, 2020, 2019 and 2018, respectively. |
Selected Quarterly Financial In
Selected Quarterly Financial Information (unaudited) | 12 Months Ended |
Dec. 31, 2020 | |
Quarterly Financial Information Disclosure [Abstract] | |
Selected Quarterly Financial Information (unaudited) | Selected Quarterly Financial Information (unaudited) The following is a summary of the quarterly results of operations for the years ended December 31, 2020 and 2019: 2020 March 31 June 30 September 30 December 31 Research and development expenses $ 13,750 $ 12,720 $ 18,841 $ 17,473 General and administrative expenses 4,998 6,289 6,643 8,022 Loss from operations (18,748) (19,009) (25,484) (25,495) Net income (loss) attributable to common stockholders $ (15,076) $ (18,589) $ (25,450) $ (25,432) Basic earnings (loss) per common share $ (0.28) $ (0.32) $ (0.27) $ (0.27) Diluted earnings (loss) per common share $ (0.28) $ (0.32) $ (0.27) $ (0.27) 2019 March 31 June 30 September 30 December 31 Research and development expenses $ 7,685 $ 10,009 $ 10,383 $ 11,567 General and administrative expenses 5,481 5,198 4,674 6,275 Income (loss) from operations (13,166) (15,207) (15,057) (17,842) Net income (loss) attributable to common stockholders $ (12,501) $ (14,443) $ (14,437) $ (17,478) Basic earnings (loss) per common share $ (0.30) $ (0.35) $ (0.35) $ (0.39) Diluted earnings (loss) per common share $ (0.30) $ (0.35) $ (0.35) $ (0.39) |
Summary of Significant Accoun_2
Summary of Significant Accounting Policies (Policies) | 12 Months Ended |
Dec. 31, 2020 | |
Accounting Policies [Abstract] | |
Basis of Presentation | Basis of Presentation and Consolidation The accompanying consolidated financial statements have been prepared in accordance with accounting principles generally accepted in the United States of America ("GAAP") and include all adjustments necessary for the fair presentation of |
Basis of Consolidation | The consolidated financial statements include the accounts of the Company and its subsidiaries. All intercompany balances and transactions have been eliminated in consolidation. |
Segment and geographic information | Segment and geographic information Operating segments are defined as components of an enterprise about which separate discrete information is available for evaluation by the chief operating decision maker, or decision making group, in deciding how to allocate resources and in assessing performance. The Company views its operations and manages its business in one operating and reporting segment. |
Use of Estimates | Use of Estimates The preparation of financial statements and related disclosures in conformity with GAAP requires management to make estimates and judgments that affect the amounts reported in the financial statements and accompanying notes. The Company bases its estimates and judgments on historical experience and on various other assumptions that it believes are reasonable under the circumstances. The amounts of assets and liabilities reported in the Company's Consolidated Balance Sheets and the amount of expenses reported for each of the periods presented are affected by estimates and assumptions, which are used for, but not limited to, accounting for research and development costs, accounting for share-based compensation and accounting for income taxes. Actual results could differ from those estimates. |
Cash and Cash Equivalents | Cash, Cash Equivalents and Available for Sale Securities The Company considers all highly liquid investments with an original maturity of 90 days or less when purchased to be cash equivalents. The carrying amounts reported in the Balance Sheets for cash and cash equivalents are valued at cost, which approximates their fair value. |
Available for Sale Securities | The Company considers securities with original maturities of greater than 90 days to be available for sale securities. Available for sale securities with original maturities of greater than one year are recorded as non-current assets. Available for sale securities are recorded at fair value and unrealized gains and losses are recorded within other comprehensive income. On a quarterly basis, the Company reviews the status of each security in an unrealized loss position, to evaluate the existence of potential credit losses. The Company first considers whether it intends to sell, or if it is more likely than not that the Company will be required to sell the security before recovery of its amortized cost basis. If either of the criteria regarding intent or requirement to sell is met, the security’s amortized cost basis is written down to fair value through income. For securities that do not meet this criteria, the Company considers a number of factors to determine if the decline in fair value has resulted from credit losses or other factors, including but not limited to: (1) the extent of the decline; (2) changes to the rating of the security by a rating agency; (3) any adverse conditions specific to the security; and (4) other market conditions that may affect the fair value of the security. If this assessment indicates that a credit loss exists and the present value of cash flows expected to be collected is less than the amortized cost basis, an allowance for credit losses is required for the credit loss. Any impairment that has not been recorded through an allowance for credit losses is recognized in other comprehensive income. |
Concentration of Credit Risk | Concentration of Credit Risk The Company's financial instruments that are exposed to concentration of credit risk consist primarily of cash, cash equivalents and available for sale securities. The Company maintains its cash in bank accounts, the balance of which generally exceed federally insured limits. The Company maintains its cash equivalents and available for sale securities in investments in money market funds, in U.S. Treasury securities and asset-backed securities and investment-grade corporate debt securities with original maturities of 90 days or less. The Company believes it is not exposed to significant credit risk on its cash and cash equivalents. |
Concentration of Suppliers | Concentration of Suppliers The Company historically relied upon a single third-party manufacturer to provide the drug substance for Zimura on a purchase order basis. The Company also historically relied upon a single third-party manufacturer to provide fill/finish services for clinical supplies of Zimura. The Company has engaged one additional third-party manufacturer to provide drug substance for Zimura and one additional third-party manufacturer to provide fill/finish services for clinical supplies of Zimura. In addition, the Company currently relies upon a single third-party supplier to supply on a purchase order basis the polyethylene glycol starting material used to manufacture Zimura. Furthermore, the Company and its contract manufacturers currently rely upon sole-source suppliers of certain raw materials and other specialized components of production used in the manufacture and fill/finish of Zimura. The Company currently relies exclusively upon a single third-party contract manufacturer for IC-100 and IC-200, and also relies on sole-source suppliers for certain starting materials used in the manufacture of such product candidates. The Company currently relies upon a single third-party contract manufacturer to conduct process development, scale-up and GMP manufacture of the drug substance for IC-500 for preclinical toxicology studies and early-stage clinical trials and a single third-party contract manufacturer to conduct formulation development activities for IC-500. If the Company’s third-party manufacturers or fill/finish service providers should become unavailable to the Company for any reason, including as a result of capacity constraints, different business objectives, financial difficulties, insolvency or the COVID-19 pandemic, the Company believes that there are a limited number of potential replacement manufacturers, and the Company likely would incur added costs and delays in identifying or qualifying such replacements. |
Foreign Currency Translation | Foreign Currency Translation The Company considers the U.S. dollar to be its functional currency. Expenses denominated in foreign currencies are translated at the exchange rate on the date the expense is incurred. The effect of exchange rate fluctuations on translating foreign currency assets and liabilities into U.S. dollars is included in the Consolidated Statements of Operations and Comprehensive Loss. Foreign exchange transaction gains and losses are included in the results of operations and are not material in the Company's financial statements. |
Financial Instruments | Financial Instruments Cash equivalents are reflected in the accompanying financial statements at fair value. The carrying amount of accounts payable and accrued expenses, including accrued research and development expenses, approximates fair value due to the short-term nature of those instruments. ASC 820, Fair Value Measurements and Disclosures , defines fair value as the price that would be received to sell an asset, or paid to transfer a liability, in the principal or most advantageous market in an orderly transaction between market participants on the measurement date. The fair value standard also establishes a three-level hierarchy, which requires an entity to maximize the use of observable inputs and minimize the use of unobservable inputs when measuring fair value. The Company reviews investments on a periodic basis for other than temporary impairments. This review is subjective as it requires management to evaluate whether an event or change in circumstances has occurred in the period that may have a significant adverse effect on the fair value of the investment. The Company uses the market approach to measure fair value for its financial assets. The market approach uses prices and other relevant information generated by market transactions involving identical or comparable assets. The Company classifies its corporate debt securities within the fair value hierarchy as Level 2 assets, as it primarily utilizes quoted market prices or rates for similar instruments to value these securities. |
Leases | Leases The Company determines if an arrangement contains a lease at inception. For arrangements where the Company is the lessee, it recognizes a right-to-use ("ROU") lease asset and operating lease liability on the Company's Consolidated Balance Sheet. ROU lease assets represent the Company's right to use the underlying asset for the lease term and the lease obligation represents the Company's commitment to make the lease payments arising from the lease. Right-of-use lease assets and obligations are recognized at the commencement date based on the present value of remaining lease payments over the lease term. As the Company’s leases do not provide an implicit discount rate, the Company has used an estimated incremental borrowing rate based on the information available at the commencement date in determining the present value of lease payments. The ROU lease asset includes any lease payments made prior to commencement and excludes any lease incentives. |
Property and Equipment | Property and Equipment Property and equipment, which consists mainly of clinical equipment, computers, software, other office equipment, and leasehold improvements, are carried at cost less accumulated depreciation. Depreciation is computed over the estimated useful lives of the respective assets, generally three |
Research and Development | Research and Development The Company's research and development expenses primarily consist of costs associated with the manufacturing, development, and preclinical and clinical testing of the Company's product candidates and costs associated with its collaborative gene therapy sponsored research programs. The Company's research and development expenses consist of: • external research and development expenses incurred under arrangements with third parties, such as academic research collaborators, contract research organizations ("CROs") and contract development and manufacturing organizations ("CDMOs") and other vendors for the production and analysis of drug substance and drug product; and • employee-related expenses for employees dedicated to research and development activities, including salaries, benefits and share-based compensation expense. Research and development expenses also include costs of acquired product licenses, in-process research and development, and related technology rights where there is no alternative future use, costs of prototypes used in research and development, consultant fees and amounts paid to collaborators. All research and development expenses are charged to operations as incurred in accordance with ASC 730, Research and Development . The Company accounts for non-refundable advance payments for goods and services that will be used in future research and development activities as expenses when the service has been performed or when the goods have been received, rather than when the payment is made. |
Income Taxes | Income Taxes The Company utilizes the liability method of accounting for deferred income taxes, as set forth in ASC 740, Income Taxes. Under this method, deferred tax assets and liabilities are recognized for the expected future tax consequences of temporary differences between the carrying amounts and the tax basis of assets and liabilities. A valuation allowance is established against deferred tax assets when, based on the weight of available evidence, it is more likely than not that some or all of the deferred tax assets will not be realized. The Company's policy is to record interest and penalties on uncertain tax positions as income tax expense. |
Share-Based Compensation | Share-Based Compensation The Company follows the provisions of ASC 718, Compensation—Stock Compensation, which requires the measurement and recognition of compensation expense for all share-based payment awards made to employees and non-employee directors and consultants, including employee stock options, restricted stock units (“RSUs”) and options granted to employees to purchase shares under the 2016 Employee Stock Purchase Plan (the “ESPP”). Share-based compensation expense is based on the grant date fair value estimated in accordance with the provisions of ASC 718 and is generally recognized as an expense over the requisite service period, net of estimated forfeitures. For grants containing performance-based vesting provisions, expense is recognized over the estimated achievement period only when the performance-based milestone is deemed probable of achievement. If performance-based milestones are later determined not to be probable of achievement, then all previously recorded stock-based compensation expense associated with such options will be reversed during the period in which the Company makes this determination. The Company estimates forfeitures at the time of grant and revises those estimates in subsequent periods if actual forfeitures differ from its estimates. The Company uses historical data to estimate pre-vesting forfeitures and record share-based compensation expense only for those awards that are expected to vest. To the extent that actual forfeitures differ from the Company's estimates, the difference is recorded as a cumulative adjustment in the period the estimates were revised. Stock Options The Company estimates the fair value of stock options granted to employees, non-employees and non-employee directors on the date of grant using the Black-Scholes option-pricing model. The Company's computation of stock-price volatility is based on daily historical volatility during the time period that corresponds to the expected option term. The Company's computation of expected term is determined using the expected term of stock option grants to employees based on an analysis of actual option exercises. The Company utilizes a dividend yield of zero based on the fact that the Company has never paid cash dividends to stockholders and has no current intentions to pay cash dividends. The risk-free interest rate is based on the zero-coupon U.S. Treasury yield at the date of grant for a term equivalent to the expected term of the option. The weighted-average assumptions used to estimate grant date fair value of stock options using the Black-Scholes option pricing model were as follows for the years ended December 31, 2020, 2019 and 2018: Years ended December 31, 2020 2019 2018 Expected common stock price volatility 118% 110% 85% Risk-free interest rate 0.22%-1.34% 1.38%-2.54% 2.39%-2.95% Expected term of options (years) 4.6 4.8 6.0 Expected dividend yield — — — RSUs The Company estimates the fair value of RSUs granted to employees using the closing market price of the Company's common stock on the date of grant. ESPP In April 2016, the Company's board of directors adopted the ESPP pursuant to which the Company may sell up to an aggregate of 1,000,000 shares of its common stock. The ESPP was approved by the Company’s stockholders in June 2016. The ESPP is considered compensatory and the fair value of the discount and look back provision are estimated using the Black-Scholes option-pricing model and recognized over the six month withholding period prior to purchase. Share-based compensation expense includes expenses related to stock options and RSUs granted to employees, non-employee directors and consultants, as well as the option granted to employees to purchase shares under the ESPP, all of which have been reported in the Company’s Statements of Operations as follows: Years ended December 31, 2020 2019 2018 Research and development $ 4,166 $ 4,260 $ 4,967 General and administrative 4,157 4,920 6,105 Total $ 8,323 $ 9,180 $ 11,072 |
Recently Adopted and Recent Accounting Pronouncements | Recently Adopted Accounting Pronouncements In June 2016, the Financial Accounting Standards Board (the "FASB") issued Accounting Standard Update ("ASU") 2016-13 Financial Instruments (Topic 326) Measurement of Credit Losses on Financial Instrument (“CECL”). ASU 2016-13 requires an allowance for expected credit losses on financial assets be recognized as early as day one of the instrument. This ASU departs from the incurred loss model which means the probability threshold is removed. It considers more forward-looking information and requires the entity to estimate its credit losses as far as it can reasonably estimate. This ASU is effective for fiscal years beginning after December 15, 2019, including interim periods within those fiscal years. The Company adopted this accounting standard effective January 1, 2020 with no material impact to its financial statements. In August 2018, the FASB issued ASU No. 2018-13, Fair Value Measurement (Topic 820) , which modifies the disclosure requirements on fair value measurements in Topic 820, Fair Value Measurement, including, among other changes, the consideration of costs and benefits when evaluating disclosure requirements. For public companies, the amendments became effective for annual reporting periods beginning after December 15, 2019, including interim periods within those annual periods. The Company adopted this accounting standard effective January 1, 2020 with no material impact to its financial statements. In August 2018, the FASB issued ASU No. 2018-15, Intangibles - Goodwill and Other - Internal-Use Software (Subtopic 350-40): Customer’s Accounting for Implementation Costs Incurred in a Cloud Computing Arrangement That Is a Service Contract (a consensus of the FASB Emerging Issues Task Force). ASU 2018-15 aligns the requirements for capitalizing implementation costs incurred in a hosting arrangement that is a service contract with the requirements for capitalizing implementation costs incurred to develop or obtain internal-use software (and hosting arrangements that include an internal-use software license). This guidance became effective for fiscal years beginning after December 15, 2019, and for interim periods within those fiscal years. The Company adopted this accounting standard effective January 1, 2020 with no material impact to its financial statements. In November 2018, the FASB issued ASU No. 2018-18, Collaborative Arrangements (Topic 808) , which clarifies the interaction between the guidance for collaborative arrangements (Topic 808) and the new revenue recognition standard (Topic 606). For public companies, the amendments became effective for annual reporting periods beginning after December 15, 2019, including interim periods within those annual periods. The Company adopted this accounting standard effective January 1, 2020 with no material impact to its financial statements. In December 2019, the FASB issued ASU No. 2019-12, Simplifying the Accounting for Income Taxes (“ASU 2019-12”), which simplifies the accounting for income taxes, eliminates certain exceptions within ASC 740, Income Taxes , and clarifies certain aspects of the current guidance to promote consistency among reporting entities. ASU 2019-12 is effective for fiscal years beginning after December 15, 2021. Most amendments within the standard are required to be applied on a prospective basis, while certain amendments must be applied on a retrospective or modified retrospective basis. The Company adopted this accounting standard effective January 1, 2020 with no material impact to its financial statements. |
Summary of Significant Accoun_3
Summary of Significant Accounting Policies (Tables) | 12 Months Ended |
Dec. 31, 2020 | |
Accounting Policies [Abstract] | |
Schedule of Weighted-average Aassumptions Used to Estimate Grant Date Fair Value of Stock Options | The weighted-average assumptions used to estimate grant date fair value of stock options using the Black-Scholes option pricing model were as follows for the years ended December 31, 2020, 2019 and 2018: Years ended December 31, 2020 2019 2018 Expected common stock price volatility 118% 110% 85% Risk-free interest rate 0.22%-1.34% 1.38%-2.54% 2.39%-2.95% Expected term of options (years) 4.6 4.8 6.0 Expected dividend yield — — — |
Schedule of Share-based Compensation Expense | Share-based compensation expense includes expenses related to stock options and RSUs granted to employees, non-employee directors and consultants, as well as the option granted to employees to purchase shares under the ESPP, all of which have been reported in the Company’s Statements of Operations as follows: Years ended December 31, 2020 2019 2018 Research and development $ 4,166 $ 4,260 $ 4,967 General and administrative 4,157 4,920 6,105 Total $ 8,323 $ 9,180 $ 11,072 |
Net Loss Per Common Share (Tabl
Net Loss Per Common Share (Tables) | 12 Months Ended |
Dec. 31, 2020 | |
Earnings Per Share [Abstract] | |
Schedule of Computation of Basic and Diluted Net Income (Loss) per Common Share | The following table sets forth the computation of basic and diluted net income (loss) per common share for the periods indicated: Years ended December 31, 2020 2019 2018 Basic and diluted net income (loss) per common share calculation: Net income (loss) $ (84,547) $ (58,859) $ 63,087 Weighted average common shares outstanding - basic 74,185 42,224 37,061 Plus: net effect of dilutive stock options and unvested restricted stock units — — 27 Weighted average common shares outstanding - dilutive 74,185 42,224 37,088 Net income (loss) per common share - basic $ (1.14) $ (1.39) $ 1.70 Net income (loss) per common share - diluted $ (1.14) $ (1.39) $ 1.70 |
Schedule of Potentially Dilutive Securities Excluded from the Computations of Diluted Weighted Average Common Shares Outstanding | The following potentially dilutive securities have been excluded from the computations of diluted weighted average common shares outstanding for the periods presented, as the effect of including such shares would be anti-dilutive: Years ended December 31, 2020 2019 2018 Stock options outstanding 8,928 6,780 5,873 Restricted stock units 1,958 1,481 601 Total 10,886 8,261 6,474 |
Cash and Cash Equivalents (Tabl
Cash and Cash Equivalents (Tables) | 12 Months Ended |
Dec. 31, 2020 | |
Cash and Cash Equivalents [Abstract] | |
Schedule of Available-for-Sale Securities | Available for sale securities, including carrying value and estimated fair values, are summarized as follows: As of December 31, 2020 Amortized Cost Gross Unrealized Gains Gross Unrealized Losses Fair Value U.S. Treasury securities $ 54,225 $ 8 $ — $ 54,233 Corporate debt securities 79,282 4 (10) 79,276 Asset-backed securities 10,164 1 — 10,165 Total $ 143,671 $ 13 $ (10) $ 143,674 |
Schedule of Changes in Accumulated Other Comprehensive Income (Loss) for Unrealized Loss on Available for Sale Securities | The changes in accumulated other comprehensive income (loss) associated with the unrealized loss on available for sale securities for the years ended December 31, 2020 and December 31, 2019 were as follows: Years ended December 31, 2020 2019 Beginning balance $ — $ — Current period changes in fair value before reclassifications, net of tax 3 — Amounts reclassified from accumulated other comprehensive income, net of tax — — Total other comprehensive income $ 3 $ — Ending balance $ 3 $ — |
Fair Value Measurements - (Tabl
Fair Value Measurements - (Tables) | 12 Months Ended |
Dec. 31, 2020 | |
Fair Value Disclosures [Abstract] | |
Schedule of Assets and Liabilities Measured at Fair Value on a Recurring Basis | The following table presents, for each of the fair value hierarchy levels required under ASC 820, the Company's assets and liabilities that are measured at fair value on a recurring basis as of December 31, 2020: Fair Value Measurement Using Quoted prices in Significant other Significant Assets Investments in money market funds* $ 58,042 $ — $ — Investments in U.S. Treasury securities $ 54,233 $ — $ — Investments in corporate debt securities* $ — $ 79,275 $ — Investments in asset-backed securities $ — $ 10,166 $ — The following table presents, for each of the fair value hierarchy levels required under ASC 820, the Company's assets and liabilities that are measured at fair value on a recurring basis as of December 31, 2019: Fair Value Measurement Using Quoted prices in Significant other Significant Assets Investments in money market funds* $ 108,585 $ — $ — Investments in corporate debt securities* $ — $ 13,875 $ — * Investments in money market funds and corporate debt securities with maturities less than 90 days are reflected in cash and cash equivalents in the accompanying Balance Sheets. |
Property and Equipment (Tables)
Property and Equipment (Tables) | 12 Months Ended |
Dec. 31, 2020 | |
Property, Plant and Equipment [Abstract] | |
Schedule of Property and Equipment | Property and equipment as of December 31, 2020 and 2019 were as follows: Useful Life December 31, December 31, Manufacturing and clinical equipment 7 - 10 $ 47 $ 47 Computer, software and other office equipment 5 933 933 980 980 Accumulated depreciation (954) (807) Property and equipment, net $ 26 $ 173 |
Income Taxes (Tables)
Income Taxes (Tables) | 12 Months Ended |
Dec. 31, 2020 | |
Income Tax Disclosure [Abstract] | |
Schedule of Reconciliation of the Statutory U.S. Federal Rate to the Company's Effective Tax Rate | A reconciliation of the statutory U.S. federal rate to the Company's effective tax rate is as follows: Years ended December 31, 2020 2019 2018 Percent of pre-tax income: U.S. federal statutory income tax rate 21.0 % 21.0 % 21.0 % State taxes, net of federal benefit 14.8 % 10.9 % 18.5 % Permanent items 0.2 % (1.0) % 4.0 % Impact of state rate changes 0.1 % 0.1 % (0.2) % Research and development credit 3.1 % 2.8 % (2.4) % Change in valuation allowance (34.8) % (33.6) % (42.6) % Effective income tax rate 4.4 % 0.2 % (1.7) % |
Schedule of Components of Income Tax (Benefit) Expense | The components of income tax benefit are as follows: Years ended December 31, 2020 2019 2018 Current: Federal $ — $ — $ — State (3,695) (111) (1,063) Deferred: Federal — — — State — — — Income tax benefit $ (3,695) $ (111) $ (1,063) |
Schedule of Significant Components of the Company's Deferred Tax Assets (Liabilities) | Significant components of the Company's deferred tax assets (liabilities) for 2020 and 2019 consist of the following: As of December 31, 2020 2019 Deferred tax assets (liabilities) License and technology payments $ 7,479 $ 7,116 Share-based compensation 23,266 22,131 Accrued expenses 427 362 Right-of-use asset (40) (163) Lease obligation 38 162 Depreciation 6 (18) Federal and state net operating loss carryforwards 130,524 105,375 Research and development credits 9,959 7,353 Other 7 6 Deferred income tax assets 171,666 142,324 Valuation allowance (171,666) (142,324) Net deferred tax assets $ — $ — |
Schedule of Position With Respect to Uncertain Tax Positions | The Company's position with respect to uncertain tax positions is set forth below: Opening balance $ 12,227 Gross amount of increases in unrecognized tax benefits during the period - current year provisions — Gross amount of increases in unrecognized tax benefits during the period - prior year provisions — Gross amount of decreases in unrecognized tax benefits during the period - other (659) Decreases due to settlement with tax authorities during the period (4,845) Reduction of unrecognized tax benefits due to expiration of the state of limitations during the period — Closing Balance $ 6,723 |
Operating Leases (Tables)
Operating Leases (Tables) | 12 Months Ended |
Dec. 31, 2020 | |
Leases [Abstract] | |
Schedule of Operating Lease Liability Maturities | The following presents the maturity of the Company's operating lease liabilities as of December 31, 2020: December 31, 2020 2021 $ 57 2022 58 2023 5 Total minimum lease payments 120 Less imputed interest (5) Present value of minimum lease payments 115 Less current portion 54 Total long-term operating lease liabilities $ 61 |
Stock-Based Compensation and _2
Stock-Based Compensation and Compensation Plans (Tables) | 12 Months Ended |
Dec. 31, 2020 | |
Share-based Payment Arrangement [Abstract] | |
Summary of the Stock Option Activity Including Weighted Average Exercise Prices, Options Outstanding and Exercisable | A summary of the stock option activity, weighted average exercise prices, options outstanding and exercisable as of December 31, 2020, 2019 and 2018 is as follows (in thousands except weighted average exercise price): Years ended December 31, 2020 2019 2018 Common Weighted Common Weighted Common Weighted Outstanding, December 31, 2019 6,780 $ 10.89 5,903 $ 13.72 5,284 $ 19.58 Granted 2,630 $ 6.10 1,610 $ 4.26 1,293 $ 1.67 Exercised (109) $ 2.89 (80) $ 2.66 — $ — Expired or forfeited (373) $ 19.49 (653) $ 21.16 (674) $ 36.54 Outstanding, December 31, 2020 8,928 $ 9.22 6,780 $ 10.89 5,903 $ 13.72 Years ended December 31, 2020 2019 2018 Options exercisable at December 31, 2020 4,462 3,317 2,709 Weighted average grant date fair value (per share) of options granted during the period $ 13.51 $ 3.37 $ 1.20 |
Summary of the Stock Options Outstanding and Exercisable | A summary of the stock options outstanding and exercisable as of December 31, 2020 is as follows (in thousands except exercise prices and weighted average exercise price): December 31, 2020 Options Outstanding Options Exercisable Range of Exercise Prices Total Weighted Weighted Number Weighted $1.13-$2.90 1,352 7.7 $ 1.68 808 $ 1.79 $2.91-$3.18 2,053 7.5 $ 2.99 1,180 $ 2.94 $3.19-$4.88 1,790 7.7 $ 4.24 1,022 $ 4.39 $4.89-$8.77 2,491 9.6 $ 6.64 211 $ 5.24 $8.78-$73.22 1,242 3.8 $ 40.08 1,241 $ 40.08 8,928 7.6 $ 9.22 4,462 $ 13.51 Aggregate Intrinsic Value $ 20,553 $ 11,741 |
Schedule of Cash Proceeds From, and the Aggregate Intrinsic Value of, Stock Options Exercised | Cash proceeds from, and the aggregate intrinsic value of, stock options exercised during the years ended December 31, 2020, 2019 and 2018, respectively, were as follows: Years ended December 31, 2020 2019 2018 Cash proceeds from options exercised $ 314 $ 192 $ — Aggregate intrinsic value of options exercised $ 393 $ 135 $ — |
Summary of the Company's Outstanding Shares of RSU Awards | The following table presents a summary of the Company's outstanding RSU awards granted as of December 31, 2020 (in thousands except weighted average grant-date fair value): Restricted Weighted Average Outstanding, December 31, 2019 1,481 $ 7.79 Awarded 1,232 $ 6.06 Vested (657) $ 7.20 Forfeited (98) $ 6.74 Outstanding, December 31, 2020 1,958 $ 6.95 |
Selected Quarterly Financial _2
Selected Quarterly Financial Information (unaudited) (Tables) | 12 Months Ended |
Dec. 31, 2020 | |
Quarterly Financial Information Disclosure [Abstract] | |
Summary of the Quarterly Results of Operations | The following is a summary of the quarterly results of operations for the years ended December 31, 2020 and 2019: 2020 March 31 June 30 September 30 December 31 Research and development expenses $ 13,750 $ 12,720 $ 18,841 $ 17,473 General and administrative expenses 4,998 6,289 6,643 8,022 Loss from operations (18,748) (19,009) (25,484) (25,495) Net income (loss) attributable to common stockholders $ (15,076) $ (18,589) $ (25,450) $ (25,432) Basic earnings (loss) per common share $ (0.28) $ (0.32) $ (0.27) $ (0.27) Diluted earnings (loss) per common share $ (0.28) $ (0.32) $ (0.27) $ (0.27) 2019 March 31 June 30 September 30 December 31 Research and development expenses $ 7,685 $ 10,009 $ 10,383 $ 11,567 General and administrative expenses 5,481 5,198 4,674 6,275 Income (loss) from operations (13,166) (15,207) (15,057) (17,842) Net income (loss) attributable to common stockholders $ (12,501) $ (14,443) $ (14,437) $ (17,478) Basic earnings (loss) per common share $ (0.30) $ (0.35) $ (0.35) $ (0.39) Diluted earnings (loss) per common share $ (0.30) $ (0.35) $ (0.35) $ (0.39) |
Summary of Significant Accoun_4
Summary of Significant Accounting Policies - Segment and Geographic Information (Details) | 12 Months Ended |
Dec. 31, 2020segment | |
Accounting Policies [Abstract] | |
Number of operating segments | 1 |
Number of reporting segments | 1 |
Summary of Significant Accoun_5
Summary of Significant Accounting Policies - Cash and Cash Equivalents (Details) $ in Millions | Dec. 31, 2020USD ($) |
Accounting Policies [Abstract] | |
Cash, cash equivalents and available for sale securities | $ 210 |
Summary of Significant Accoun_6
Summary of Significant Accounting Policies - Property and Equipment (Details) | 12 Months Ended |
Dec. 31, 2020 | |
Minimum | |
Property and equipment | |
Estimated useful lives | 3 years |
Maximum | |
Property and equipment | |
Estimated useful lives | 10 years |
Summary of Significant Accoun_7
Summary of Significant Accounting Policies - Share-Based Compensation (Details) - USD ($) $ in Thousands | 12 Months Ended | |||
Dec. 31, 2020 | Dec. 31, 2019 | Dec. 31, 2018 | Apr. 30, 2016 | |
Share-Based Compensation | ||||
Expected dividend yield | 0.00% | |||
Share-based compensation expense | $ 8,323 | $ 9,180 | $ 11,072 | |
Research and development | ||||
Share-Based Compensation | ||||
Share-based compensation expense | 4,166 | 4,260 | 4,967 | |
General and administrative | ||||
Share-Based Compensation | ||||
Share-based compensation expense | 4,157 | 4,920 | $ 6,105 | |
ESPP | ||||
Share-Based Compensation | ||||
Share-based compensation expense | $ 100 | $ 100 | ||
ESPP | Maximum | ||||
Share-Based Compensation | ||||
Number of shares reserved for issuance under the Plan (in shares) | 1,000,000 |
Summary of Significant Accoun_8
Summary of Significant Accounting Policies - Schedule of Weighted-Average Assumptions (Details) | 12 Months Ended | ||
Dec. 31, 2020 | Dec. 31, 2019 | Dec. 31, 2018 | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||
Risk-free interest rate, minimum | 0.22% | 1.38% | 2.39% |
Risk-free interest rate, maximum | 1.34% | 2.54% | 2.95% |
Expected dividend yield | 0.00% | ||
Stock options | |||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||
Expected common stock price volatility | 118.00% | 110.00% | 85.00% |
Expected term of options (years) | 4 years 7 months 6 days | 4 years 9 months 18 days | 6 years |
Expected dividend yield | 0.00% | 0.00% | 0.00% |
Common Stock - Narrative (Detai
Common Stock - Narrative (Details) - USD ($) $ / shares in Units, $ in Thousands | Dec. 06, 2019 | Jul. 22, 2019 | Jun. 30, 2020 | Jul. 31, 2019 | Dec. 31, 2020 | Dec. 31, 2019 | Dec. 31, 2018 |
Class of Stock [Line Items] | |||||||
Number of warrants issued (in shares) | 1,914,280 | ||||||
Payments of stock issuance costs | $ 3,000 | $ 10,100 | |||||
Proceeds from issuance of common stock and pre-funded warrants, net, and related to acquisition | $ 150,100 | ||||||
Payment of other stock issuance costs | 400 | ||||||
Sale of stock, consideration received on transaction | $ 42,600 | ||||||
License Agreements | University Of Massachusetts (UMass) | miniCEP290 | |||||||
Class of Stock [Line Items] | |||||||
Shares issued for license fees (in shares) | 75,000 | 75,000 | |||||
Shares issued for license agreement (in shares) | 100,000 | ||||||
Underwritten Offering | |||||||
Class of Stock [Line Items] | |||||||
Sale of stock, number of shares issued in transaction (in shares) | 28,503,220 | ||||||
Sale of stock, price per share (in dollars per share) | $ 4.10 | ||||||
Underwritten Offering, Underwriters | |||||||
Class of Stock [Line Items] | |||||||
Sale of stock, price per share (in dollars per share) | $ 3.854 | ||||||
Private Placement | |||||||
Class of Stock [Line Items] | |||||||
Sale of stock, number of shares issued in transaction (in shares) | 8,649,453 | ||||||
Warrant price (USD per share) | $ 4.099 | ||||||
Proceeds from issuance of common stock and pre-funded warrants, net, and related to acquisition | $ 33,237 | $ 0 | $ 0 | ||||
Private Placement, Underwriters | |||||||
Class of Stock [Line Items] | |||||||
Warrant price (USD per share) | $ 3.853 | ||||||
Public Stock Offering | |||||||
Class of Stock [Line Items] | |||||||
Sale of stock, number of shares issued in transaction (in shares) | 6,250,000 | ||||||
Sale of stock, price per share (in dollars per share) | $ 4 | ||||||
Number of warrants issued (in shares) | 3,750,000 | ||||||
Warrant price (USD per share) | $ 3.999 | ||||||
Proceeds from issuance of common stock and pre-funded warrants, net, and related to acquisition | $ 116,883 | $ 42,565 | $ 0 | ||||
Over-Allotment Option | |||||||
Class of Stock [Line Items] | |||||||
Sale of stock, number of shares issued in transaction (in shares) | 1,500,000 | ||||||
Sale of stock, price per share (in dollars per share) | $ 3.736 | ||||||
Warrant price (USD per share) | $ 3.735 |
Net Income (Loss) Per Common _2
Net Income (Loss) Per Common Share - Narrative (Details) - shares | Dec. 31, 2020 | Dec. 31, 2019 |
Earnings Per Share [Abstract] | ||
Pre-funded warrants outstanding (in shares) | 3,164,280 | 3,750,000 |
Net Income (Loss) Per Common _3
Net Income (Loss) Per Common Share - Schedule of Basic and Diluted Net Income (Loss) Per Common Share (Details) - USD ($) $ / shares in Units, shares in Thousands, $ in Thousands | 3 Months Ended | 12 Months Ended | |||||||||
Dec. 31, 2020 | Sep. 30, 2020 | Jun. 30, 2020 | Mar. 31, 2020 | Dec. 31, 2019 | Sep. 30, 2019 | Jun. 30, 2019 | Mar. 31, 2019 | Dec. 31, 2020 | Dec. 31, 2019 | Dec. 31, 2018 | |
Basic and diluted net loss per common share calculation: | |||||||||||
Net income (loss) | $ (84,547) | $ (58,859) | $ 63,087 | ||||||||
Weighted average common shares outstanding - basic (in shares) | 74,185 | 42,224 | 37,061 | ||||||||
Plus: net effect of dilutive stock options and unvested restricted stock units (in shares) | 0 | 0 | 27 | ||||||||
Weighted average common shares outstanding - dilutive (in shares) | 74,185 | 42,224 | 37,088 | ||||||||
Net income (loss) per common share - basic (USD per share) | $ (0.27) | $ (0.27) | $ (0.32) | $ (0.28) | $ (0.39) | $ (0.35) | $ (0.35) | $ (0.30) | $ (1.14) | $ (1.39) | $ 1.70 |
Net income (loss) per common share - diluted (USD per share) | $ (0.27) | $ (0.27) | $ (0.32) | $ (0.28) | $ (0.39) | $ (0.35) | $ (0.35) | $ (0.30) | $ (1.14) | $ (1.39) | $ 1.70 |
Net Income (Loss) Per Common _4
Net Income (Loss) Per Common Share - Schedule of Potentially Dilutive Securities (Details) - shares shares in Thousands | 12 Months Ended | ||
Dec. 31, 2020 | Dec. 31, 2019 | Dec. 31, 2018 | |
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | |||
Total (in shares) | 10,886 | 8,261 | 6,474 |
Stock options outstanding | |||
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | |||
Total (in shares) | 8,928 | 6,780 | 5,873 |
Restricted stock units | |||
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | |||
Total (in shares) | 1,958 | 1,481 | 601 |
Cash, Cash Equivalents and Av_2
Cash, Cash Equivalents and Available for Sale Securities - Narrative (Details) - USD ($) | Dec. 31, 2020 | Dec. 31, 2019 |
Cash and Cash Equivalents [Abstract] | ||
Cash and cash equivalents | $ 66,373,000 | $ 125,699,000 |
Cash | 8,400,000 | 3,200,000 |
Cash equivalents | 58,000,000 | 122,500,000 |
Available for sale debt securities | $ 143,674,000 | $ 0 |
Cash, Cash Equivalents and Av_3
Cash, Cash Equivalents and Available for Sale Securities - Schedule of Available for Sale Securities (Details) - USD ($) | 12 Months Ended | |
Dec. 31, 2020 | Dec. 31, 2019 | |
Debt Securities, Available-for-sale [Line Items] | ||
Amortized Cost | $ 143,671,000 | |
Gross Unrealized Gains | 13,000 | |
Gross Unrealized Losses | (10,000) | |
Fair Value | 143,674,000 | $ 0 |
U.S. Treasury securities | ||
Debt Securities, Available-for-sale [Line Items] | ||
Amortized Cost | 54,225,000 | |
Gross Unrealized Gains | 8,000 | |
Gross Unrealized Losses | 0 | |
Fair Value | 54,233,000 | |
Corporate debt securities | ||
Debt Securities, Available-for-sale [Line Items] | ||
Amortized Cost | 79,282,000 | |
Gross Unrealized Gains | 4,000 | |
Gross Unrealized Losses | (10,000) | |
Fair Value | 79,276,000 | |
Asset-backed securities | ||
Debt Securities, Available-for-sale [Line Items] | ||
Amortized Cost | 10,164,000 | |
Gross Unrealized Gains | 1,000 | |
Gross Unrealized Losses | 0 | |
Fair Value | $ 10,165,000 |
Cash, Cash Equivalents and Av_4
Cash, Cash Equivalents and Available for Sale Securities - Schedule of Changes in Accumulated Other Comprehensive Income (Loss) for Unrealized Loss on Available for Sale Securities (Details) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2020 | Dec. 31, 2019 | Dec. 31, 2018 | |
Accumulated Other Comprehensive Income [Roll Forward] | |||
Balance at beginning of period | $ 117,203 | $ 123,959 | $ 38,041 |
Other comprehensive income | 3 | 0 | 0 |
Ending balance | 191,563 | 117,203 | 123,959 |
AOCI, Accumulated Gain (Loss), Debt Securities, Available-for-sale, Parent | |||
Accumulated Other Comprehensive Income [Roll Forward] | |||
Balance at beginning of period | 0 | 0 | |
Current period changes in fair value before reclassifications, net of tax | 3 | 0 | |
Amounts reclassified from accumulated other comprehensive income, net of tax | 0 | 0 | |
Other comprehensive income | 3 | 0 | |
Ending balance | $ 3 | $ 0 | $ 0 |
Fair Value Measurements - Asset
Fair Value Measurements - Assets and Liabilities Measured at Fair Value on a Recurring Basis (Details) - USD ($) | Dec. 31, 2020 | Dec. 31, 2019 |
Fair Value Measurements | ||
Transfer of assets from Level 1 to Level 2 of the fair value measurement hierarchy | $ 0 | $ 0 |
Recurring basis | Quoted prices in active markets for identical assets (Level 1) | Money market funds | ||
Fair Value Measurements | ||
Investments in money market funds | 58,042,000 | 108,585,000 |
Recurring basis | Quoted prices in active markets for identical assets (Level 1) | U.S. Treasury securities | ||
Fair Value Measurements | ||
Investments in money market funds | 54,233,000 | |
Recurring basis | Quoted prices in active markets for identical assets (Level 1) | Corporate debt securities | ||
Fair Value Measurements | ||
Investments in money market funds | 0 | 0 |
Recurring basis | Quoted prices in active markets for identical assets (Level 1) | Asset-backed securities | ||
Fair Value Measurements | ||
Investments in money market funds | 0 | |
Recurring basis | Significant other observable inputs (Level 2) | Money market funds | ||
Fair Value Measurements | ||
Investments in money market funds | 0 | 0 |
Recurring basis | Significant other observable inputs (Level 2) | U.S. Treasury securities | ||
Fair Value Measurements | ||
Investments in money market funds | 0 | |
Recurring basis | Significant other observable inputs (Level 2) | Corporate debt securities | ||
Fair Value Measurements | ||
Investments in money market funds | 79,275,000 | 13,875,000 |
Recurring basis | Significant other observable inputs (Level 2) | Asset-backed securities | ||
Fair Value Measurements | ||
Investments in money market funds | 10,166,000 | |
Recurring basis | Significant unobservable inputs (Level 3) | Money market funds | ||
Fair Value Measurements | ||
Investments in money market funds | 0 | 0 |
Recurring basis | Significant unobservable inputs (Level 3) | U.S. Treasury securities | ||
Fair Value Measurements | ||
Investments in money market funds | 0 | |
Recurring basis | Significant unobservable inputs (Level 3) | Corporate debt securities | ||
Fair Value Measurements | ||
Investments in money market funds | 0 | $ 0 |
Recurring basis | Significant unobservable inputs (Level 3) | Asset-backed securities | ||
Fair Value Measurements | ||
Investments in money market funds | $ 0 |
Inception 4 Acquisition (Detail
Inception 4 Acquisition (Details) - USD ($) $ / shares in Units, shares in Millions, $ in Millions | Oct. 30, 2018 | Oct. 31, 2018 | Dec. 31, 2018 |
Schedule Of Asset Acquisition [Line Items] | |||
Share price (USD per share) | $ 2.26 | ||
Non-cash charge on acquired in-process research and development | $ 6.9 | ||
Transaction costs | $ 1.3 | ||
Inception 4 | |||
Schedule Of Asset Acquisition [Line Items] | |||
Number of shares issued in asset acquisition (in shares) | 5.2 | ||
Value of shares issued in asset acquisition | $ 11.7 | ||
Contingent consideration | $ 105 | $ 105 | |
Contingently issuable shares, asset acquisition (in shares) | 7.2 | 7.2 | |
Contingently issuable shares, asset acquisition, as a percentage of total shares outstanding | 19.90% | 19.90% | |
Cash acquired in asset acquisition | $ 6.1 | ||
GA | Inception 4 | |||
Schedule Of Asset Acquisition [Line Items] | |||
Clinical and marketing approval milestones | 45 | ||
Wet AMD | Inception 4 | |||
Schedule Of Asset Acquisition [Line Items] | |||
Clinical and marketing approval milestones | $ 60 |
Licensing and Commercializati_2
Licensing and Commercialization Agreement (Details) $ in Thousands | Jul. 22, 2019shares | Oct. 31, 2020USD ($) | Mar. 31, 2020USD ($) | Sep. 30, 2019USD ($) | Jul. 31, 2019USD ($)shares | May 31, 2019USD ($)licensedProductshares | Jun. 30, 2018USD ($) | Sep. 30, 2011USD ($)shares | Oct. 31, 2020milestone_payment_number | Dec. 31, 2020 | Oct. 31, 2020USD ($) | Dec. 31, 2018USD ($) |
Agreement | ||||||||||||
Number of licensed products | licensedProduct | 1 | |||||||||||
RHO-adRP License Agreement | University of Florida Research Foundation (UFRF) | ||||||||||||
Agreement | ||||||||||||
Licensing agreement term | 10 years | |||||||||||
RHO-adRP License Agreement | University of Florida Research Foundation (UFRF) | Specified Clinical, Marketing Approval and Reimbursement Approval Milestones Related to a Licensed Product | ||||||||||||
Agreement | ||||||||||||
Amount to be paid on achievement of milestone | $ 23,500 | |||||||||||
RHO-adRP License Agreement | University of Florida Research Foundation (UFRF) | Specified Commercial Sales Milestones Related to a Licensed Product | ||||||||||||
Agreement | ||||||||||||
Amount to be paid on achievement of milestone | $ 70,000 | |||||||||||
BEST1 License Agreement | University Of Florida Research Foundation (UFRF) And University Of Pennsylvania (Penn) | ||||||||||||
Agreement | ||||||||||||
Licensing agreement term | 10 years | |||||||||||
Research and development | RHO-adRP License Agreement | University of Florida Research Foundation (UFRF) | ||||||||||||
Agreement | ||||||||||||
Payments for license fees | $ 500 | |||||||||||
Research and development | RHO-adRP License Agreement | University of Florida Research Foundation (UFRF) | Specified Clinical, Marketing Approval and Reimbursement Approval Milestones Related to a Licensed Product | ||||||||||||
Agreement | ||||||||||||
Payments for license fees | 23,500 | |||||||||||
Research and development | RHO-adRP License Agreement | University of Florida Research Foundation (UFRF) | Specified Commercial Sales Milestones Related to a Licensed Product | ||||||||||||
Agreement | ||||||||||||
Payments for license fees | 70,000 | |||||||||||
Research and development | BEST1 License Agreement | University Of Pennsylvania | Specified Clinical, Marketing Approval and Reimbursement Approval Milestones Related to a Licensed Product | Primary Licensed Product | ||||||||||||
Agreement | ||||||||||||
Payments for license fees | $ 200 | |||||||||||
Research and development | BEST1 License Agreement | University Of Florida Research Foundation (UFRF) And University Of Pennsylvania (Penn) | Specified Clinical, Marketing Approval and Reimbursement Approval Milestones Related to a Licensed Product | Primary Licensed Product | ||||||||||||
Agreement | ||||||||||||
Payments for license fees | 15,700 | |||||||||||
Research and development | BEST1 License Agreement | University Of Florida Research Foundation (UFRF) And University Of Pennsylvania (Penn) | Specified Clinical, Marketing Approval and Reimbursement Approval Milestones Related to a Licensed Product | Other Licensed Product | ||||||||||||
Agreement | ||||||||||||
Payments for license fees | 3,100 | |||||||||||
Research and development | BEST1 License Agreement | University Of Florida Research Foundation (UFRF) And University Of Pennsylvania (Penn) | Specified Commercial Sales Milestones Related to a Licensed Product | Primary Licensed Product | ||||||||||||
Agreement | ||||||||||||
Payments for license fees | 48,000 | |||||||||||
Research and development | BEST1 License Agreement | University Of Florida Research Foundation (UFRF) And University Of Pennsylvania (Penn) | Specified Commercial Sales Milestones Related to a Licensed Product | Other Licensed Product | ||||||||||||
Agreement | ||||||||||||
Payments for license fees | 9,600 | |||||||||||
General and administrative | RHO-adRP License Agreement | University of Florida Research Foundation (UFRF) | ||||||||||||
Agreement | ||||||||||||
Payments for license fees | $ 30 | |||||||||||
General and administrative | BEST1 License Agreement | University of Florida Research Foundation (UFRF) | Specified Clinical, Marketing Approval and Reimbursement Approval Milestones Related to a Licensed Product | Primary Licensed Product | ||||||||||||
Agreement | ||||||||||||
Payments for license fees | 18 | |||||||||||
License Agreements | Archemix | C5 Licensed Product | ||||||||||||
Agreement | ||||||||||||
Licensing agreement term | 12 years | |||||||||||
License Agreements | Archemix | Achievement of specified clinical and regulatory milestones | C5 Licensed Product | ||||||||||||
Agreement | ||||||||||||
Payments for license fees | $ 6,000 | $ 1,000 | $ 1,000 | $ 9,000 | ||||||||
Number of milestone payments | milestone_payment_number | 2 | |||||||||||
Trial data period | 12 months | |||||||||||
License Agreements | Archemix | First indication | C5 Licensed Product | ||||||||||||
Agreement | ||||||||||||
Amount to be paid on achievement of milestone | 24,500 | |||||||||||
License Agreements | Archemix | Second and third indication | C5 Licensed Product | ||||||||||||
Agreement | ||||||||||||
Amount to be paid on achievement of milestone | 23,500 | |||||||||||
License Agreements | Archemix | Sustained delivery applications | C5 Licensed Product | ||||||||||||
Agreement | ||||||||||||
Amount to be paid on achievement of milestone | $ 2,500 | |||||||||||
License Agreements | University Of Massachusetts (UMass) | miniCEP290 | ||||||||||||
Agreement | ||||||||||||
Shares issued for license fees (in shares) | shares | 75,000 | 75,000 | ||||||||||
Licensing agreement term | 10 years | |||||||||||
License Agreements | Research and development | University Of Massachusetts (UMass) | miniCEP290 | ||||||||||||
Agreement | ||||||||||||
Payments for license fees | $ 400 | |||||||||||
License Agreements | Research and development | University Of Massachusetts (UMass) | Achievement of specified clinical and regulatory milestones | miniCEP290 | ||||||||||||
Agreement | ||||||||||||
Payments for license fees | $ 14,750 | $ 14,750 | ||||||||||
Shares issued for license fees (in shares) | shares | 75,000 | 75,000 | ||||||||||
License Agreements | Research and development | University Of Massachusetts (UMass) | Specified Commercial Sales Milestones Related to a Licensed Product | miniCEP290 | ||||||||||||
Agreement | ||||||||||||
Payments for license fees | $ 48,000 | $ 48,000 | ||||||||||
License Agreements | General and administrative | University Of Massachusetts (UMass) | miniCEP290 | ||||||||||||
Agreement | ||||||||||||
Payments for license fees | $ 18 | |||||||||||
License Agreements | Series A Preferred Stock | Archemix | Achievement of specified clinical and regulatory milestones | C5 Licensed Product | ||||||||||||
Agreement | ||||||||||||
Shares issued for license fees (in shares) | shares | 2,000,000 | |||||||||||
License Agreements | Series B Preferred Stock | Archemix | Achievement of specified clinical and regulatory milestones | C5 Licensed Product | ||||||||||||
Agreement | ||||||||||||
Shares issued for license fees (in shares) | shares | 500,000 | |||||||||||
Maximum | License Agreements | Archemix | Achievement of specified clinical and regulatory milestones | C5 Licensed Product | ||||||||||||
Agreement | ||||||||||||
Amount to be paid on achievement of milestone | $ 50,500 | |||||||||||
Maximum | License Agreements | Archemix | First indication | C5 Licensed Product | ||||||||||||
Agreement | ||||||||||||
Amount to be paid on achievement of milestone | 24,500 | |||||||||||
Maximum | License Agreements | Archemix | Second and third indication | C5 Licensed Product | ||||||||||||
Agreement | ||||||||||||
Amount to be paid on achievement of milestone | 23,500 | |||||||||||
Maximum | License Agreements | Archemix | Sustained delivery applications | C5 Licensed Product | ||||||||||||
Agreement | ||||||||||||
Amount to be paid on achievement of milestone | 2,500 | |||||||||||
Maximum | License Agreements | Archemix | Achievement of specified commercial milestones | C5 Licensed Product | ||||||||||||
Agreement | ||||||||||||
Amount to be paid on achievement of milestone | $ 22,500 |
Financing Agreement with Novo_2
Financing Agreement with Novo Holdings A/S (Details) | 1 Months Ended | 12 Months Ended | 19 Months Ended | ||||
Nov. 30, 2014USD ($) | Jan. 31, 2014USD ($) | May 31, 2013USD ($)tranche | Dec. 31, 2020USD ($) | Dec. 31, 2019USD ($) | Dec. 31, 2018USD ($) | Nov. 30, 2014USD ($) | |
Agreement | |||||||
Gain on extinguishment of Royalty Purchase Liability | $ 0 | $ 0 | $ 125,000,000 | ||||
Novo A/S | Novo Agreement | Fovista, Fovista-Related Products, and Other Products | |||||||
Agreement | |||||||
Number of tranches in financing | tranche | 3 | ||||||
Aggregate royalty rights | $ 125,000,000 | ||||||
Proceeds from royalty purchase agreement | $ 41,700,000 | $ 41,700,000 | 41,700,000 | $ 125,000,000 | |||
Royalty purchase liability | $ 125,000,000 |
Property and Equipment (Details
Property and Equipment (Details) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2020 | Dec. 31, 2019 | Dec. 31, 2018 | |
Property and Equipment | |||
Property and equipment, gross | $ 980 | $ 980 | |
Accumulated depreciation | (954) | (807) | |
Property and equipment, net | 26 | 173 | |
Depreciation expense | $ 143 | 162 | $ 183 |
Minimum | |||
Property and Equipment | |||
Useful life | 3 years | ||
Maximum | |||
Property and Equipment | |||
Useful life | 10 years | ||
Manufacturing and clinical equipment | |||
Property and Equipment | |||
Property and equipment, gross | $ 47 | 47 | |
Manufacturing and clinical equipment | Minimum | |||
Property and Equipment | |||
Useful life | 7 years | ||
Manufacturing and clinical equipment | Maximum | |||
Property and Equipment | |||
Useful life | 10 years | ||
Computer, software and other office equipment | |||
Property and Equipment | |||
Useful life | 5 years | ||
Property and equipment, gross | $ 933 | $ 933 |
Income Taxes - Schedule of Effe
Income Taxes - Schedule of Effective Income Tax Rate Reconciliation (Details) | 12 Months Ended | ||
Dec. 31, 2020 | Dec. 31, 2019 | Dec. 31, 2018 | |
Percent of pre-tax income: | |||
U.S. federal statutory income tax rate | 21.00% | 21.00% | 21.00% |
State taxes, net of federal benefit | 14.80% | 10.90% | 18.50% |
Permanent items | 0.20% | (1.00%) | 4.00% |
Impact of state rate changes | 0.10% | 0.10% | (0.20%) |
Research and development credit | 3.10% | 2.80% | (2.40%) |
Change in valuation allowance | (34.80%) | (33.60%) | (42.60%) |
Effective income tax rate | 4.40% | 0.20% | (1.70%) |
Income Taxes - Schedule of Comp
Income Taxes - Schedule of Components of Income Tax (Benefit) Expense (Details) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2020 | Dec. 31, 2019 | Dec. 31, 2018 | |
Current: | |||
Federal | $ 0 | $ 0 | $ 0 |
State | (3,695) | (111) | (1,063) |
Deferred: | |||
Federal | 0 | 0 | 0 |
State | 0 | 0 | 0 |
Income tax benefit | $ (3,695) | $ (111) | $ (1,063) |
Income Taxes - Schedule of Sign
Income Taxes - Schedule of Significant Components of Deferred Tax Assets (Liabilities) (Details) - USD ($) $ in Thousands | Dec. 31, 2020 | Dec. 31, 2019 |
Deferred tax assets (liabilities) | ||
License and technology payments | $ 7,479 | $ 7,116 |
Share-based compensation | 23,266 | 22,131 |
Accrued expenses | 427 | 362 |
Right-of-use asset | (40) | (163) |
Lease obligation | 38 | 162 |
Depreciation | 6 | (18) |
Federal and state net operating loss carryforwards | 130,524 | 105,375 |
Research and development credits | 9,959 | 7,353 |
Other | 7 | 6 |
Deferred income tax assets | 171,666 | 142,324 |
Valuation allowance | (171,666) | (142,324) |
Net deferred tax assets | $ 0 | |
Net deferred tax assets | $ 0 |
Income Taxes - Narrative (Detai
Income Taxes - Narrative (Details) - USD ($) $ in Thousands | 12 Months Ended | |||
Dec. 31, 2021 | Dec. 31, 2020 | Dec. 31, 2019 | Dec. 31, 2018 | |
Operating Loss Carryforwards [Line Items] | ||||
Income tax benefit | $ 3,695 | $ 111 | $ 1,063 | |
Income tax receivable | 1,765 | 882 | ||
Settlement of state franchise tax audit | 5,200 | |||
Increase in deferred tax assets and corresponding valuation allowance | 300 | |||
CARES Act | ||||
Operating Loss Carryforwards [Line Items] | ||||
Income tax receivable | 1,800 | |||
Proceeds from income tax refunds | $ 1,800 | |||
CARES Act | Forecast | ||||
Operating Loss Carryforwards [Line Items] | ||||
Proceeds from income tax refunds | $ 1,800 | |||
Domestic Tax Authority | ||||
Operating Loss Carryforwards [Line Items] | ||||
Federal net operating loss carryforwards | $ 445,000 |
Income Taxes - Schedule of Posi
Income Taxes - Schedule of Position with Respect to Uncertain Tax Positions (Details) $ in Thousands | 12 Months Ended |
Dec. 31, 2020USD ($) | |
Reconciliation of Unrecognized Tax Benefits, Excluding Amounts Pertaining to Examined Tax Returns [Roll Forward] | |
Opening balance | $ 12,227 |
Gross amount of increases in unrecognized tax benefits during the period - current year provisions | 0 |
Gross amount of increases in unrecognized tax benefits during the period - prior year provisions | 0 |
Gross amount of decreases in unrecognized tax benefits during the period - other | (659) |
Decreases due to settlement with tax authorities during the period | (4,845) |
Reduction of unrecognized tax benefits due to expiration of the state of limitations during the period | 0 |
Closing Balance | $ 6,723 |
Operating Leases - Narrative (D
Operating Leases - Narrative (Details) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2020 | Dec. 31, 2019 | Dec. 31, 2018 | |
Leases [Abstract] | |||
Operating lease right-of-use asset | $ 120 | $ 496 | |
Operating lease liability | 115 | ||
Operating lease rent expense | 600 | 1,000 | |
Operating lease rent expense | $ 800 | ||
Cash paid from operating cash flows | $ 600 | $ 1,000 | |
Operating lease, weighted average remaining lease term | 2 years 1 month 6 days | ||
Weighted average estimated incremental borrowing rate | 3.40% |
Operating Leases - Schedule of
Operating Leases - Schedule of Operating Lease Liability Maturities (Details) - USD ($) $ in Thousands | Dec. 31, 2020 | Dec. 31, 2019 |
Future minimum rental commitments under non-cancelable operating leases | ||
2021 | $ 57 | |
2022 | 58 | |
2023 | 5 | |
Total minimum lease payments | 120 | |
Less imputed interest | (5) | |
Present value of minimum lease payments | 115 | |
Less current portion | 54 | $ 495 |
Total long-term operating lease liabilities | $ 61 | $ 0 |
Commitments and Contingencies -
Commitments and Contingencies - Narrative (Details) - USD ($) $ in Thousands | Jul. 22, 2019 | Oct. 30, 2018 | Oct. 31, 2020 | Mar. 31, 2020 | Sep. 30, 2019 | Jul. 31, 2019 | May 31, 2019 | Apr. 30, 2019 | Oct. 31, 2018 | Jun. 30, 2018 | Sep. 30, 2011 | Oct. 31, 2020 | Dec. 31, 2018 |
Cano v. Guyer, et al., No. 650601/2018 | |||||||||||||
Commitments and contingencies | |||||||||||||
Litigation settlement awared to other party | $ 300 | ||||||||||||
License Agreements | Archemix | C5 Licensed Product | Achievement of specified clinical and regulatory milestones | |||||||||||||
Commitments and contingencies | |||||||||||||
Payments for license fees | $ 6,000 | $ 1,000 | $ 1,000 | $ 9,000 | |||||||||
License Agreements | Archemix | C5 Licensed Product | Achievement of specified clinical and regulatory milestones | Maximum | |||||||||||||
Commitments and contingencies | |||||||||||||
Amount to be paid on achievement of milestone | 50,500 | ||||||||||||
License Agreements | Archemix | C5 Licensed Product | First indication | |||||||||||||
Commitments and contingencies | |||||||||||||
Amount to be paid on achievement of milestone | 24,500 | ||||||||||||
License Agreements | Archemix | C5 Licensed Product | First indication | Maximum | |||||||||||||
Commitments and contingencies | |||||||||||||
Amount to be paid on achievement of milestone | 24,500 | ||||||||||||
License Agreements | Archemix | C5 Licensed Product | Second and third indication | |||||||||||||
Commitments and contingencies | |||||||||||||
Amount to be paid on achievement of milestone | 23,500 | ||||||||||||
License Agreements | Archemix | C5 Licensed Product | Second and third indication | Maximum | |||||||||||||
Commitments and contingencies | |||||||||||||
Amount to be paid on achievement of milestone | 23,500 | ||||||||||||
License Agreements | Archemix | C5 Licensed Product | Sustained delivery applications | |||||||||||||
Commitments and contingencies | |||||||||||||
Amount to be paid on achievement of milestone | 2,500 | ||||||||||||
License Agreements | Archemix | C5 Licensed Product | Sustained delivery applications | Maximum | |||||||||||||
Commitments and contingencies | |||||||||||||
Amount to be paid on achievement of milestone | 2,500 | ||||||||||||
License Agreements | Archemix | C5 Licensed Product | Achievement of specified commercial milestones | Maximum | |||||||||||||
Commitments and contingencies | |||||||||||||
Amount to be paid on achievement of milestone | $ 22,500 | ||||||||||||
License Agreements | University Of Massachusetts (UMass) | miniCEP290 | |||||||||||||
Commitments and contingencies | |||||||||||||
Shares issued for license fees (in shares) | 75,000 | 75,000 | |||||||||||
RHO-adRP License Agreement | University of Florida Research Foundation (UFRF) | Specified Clinical, Marketing Approval and Reimbursement Approval Milestones Related to a Licensed Product | |||||||||||||
Commitments and contingencies | |||||||||||||
Amount to be paid on achievement of milestone | $ 23,500 | ||||||||||||
RHO-adRP License Agreement | University of Florida Research Foundation (UFRF) | Specified Commercial Sales Milestones Related to a Licensed Product | |||||||||||||
Commitments and contingencies | |||||||||||||
Amount to be paid on achievement of milestone | $ 70,000 | ||||||||||||
Inception 4 | |||||||||||||
Commitments and contingencies | |||||||||||||
Contingent consideration | $ 105,000 | $ 105,000 | |||||||||||
Contingently issuable shares, asset acquisition (in shares) | 7,200,000 | 7,200,000 | |||||||||||
Contingently issuable shares, asset acquisition, as a percentage of total shares outstanding | 19.90% | 19.90% | |||||||||||
Inception 4 | GA | |||||||||||||
Commitments and contingencies | |||||||||||||
Clinical and marketing approval milestones | $ 45,000 | ||||||||||||
Inception 4 | Wet AMD | |||||||||||||
Commitments and contingencies | |||||||||||||
Clinical and marketing approval milestones | $ 60,000 | ||||||||||||
Research and development | License Agreements | University Of Massachusetts (UMass) | miniCEP290 | |||||||||||||
Commitments and contingencies | |||||||||||||
Payments for license fees | $ 400 | ||||||||||||
Research and development | License Agreements | University Of Massachusetts (UMass) | miniCEP290 | Achievement of specified clinical and regulatory milestones | |||||||||||||
Commitments and contingencies | |||||||||||||
Payments for license fees | $ 14,750 | $ 14,750 | |||||||||||
Shares issued for license fees (in shares) | 75,000 | 75,000 | |||||||||||
Research and development | License Agreements | University Of Massachusetts (UMass) | miniCEP290 | Specified Commercial Sales Milestones Related to a Licensed Product | |||||||||||||
Commitments and contingencies | |||||||||||||
Payments for license fees | $ 48,000 | $ 48,000 | |||||||||||
Research and development | RHO-adRP License Agreement | University of Florida Research Foundation (UFRF) | |||||||||||||
Commitments and contingencies | |||||||||||||
Payments for license fees | $ 500 | ||||||||||||
Research and development | RHO-adRP License Agreement | University of Florida Research Foundation (UFRF) | Specified Clinical, Marketing Approval and Reimbursement Approval Milestones Related to a Licensed Product | |||||||||||||
Commitments and contingencies | |||||||||||||
Payments for license fees | 23,500 | ||||||||||||
Research and development | RHO-adRP License Agreement | University of Florida Research Foundation (UFRF) | Specified Commercial Sales Milestones Related to a Licensed Product | |||||||||||||
Commitments and contingencies | |||||||||||||
Payments for license fees | $ 70,000 | ||||||||||||
Research and development | BEST1 License Agreement | University Of Florida Research Foundation (UFRF) And University Of Pennsylvania (Penn) | Primary Licensed Product | Specified Clinical, Marketing Approval and Reimbursement Approval Milestones Related to a Licensed Product | |||||||||||||
Commitments and contingencies | |||||||||||||
Payments for license fees | 15,700 | ||||||||||||
Research and development | BEST1 License Agreement | University Of Florida Research Foundation (UFRF) And University Of Pennsylvania (Penn) | Primary Licensed Product | Specified Commercial Sales Milestones Related to a Licensed Product | |||||||||||||
Commitments and contingencies | |||||||||||||
Payments for license fees | 48,000 | ||||||||||||
Research and development | BEST1 License Agreement | University Of Florida Research Foundation (UFRF) And University Of Pennsylvania (Penn) | Other Licensed Product | Specified Clinical, Marketing Approval and Reimbursement Approval Milestones Related to a Licensed Product | |||||||||||||
Commitments and contingencies | |||||||||||||
Payments for license fees | 3,100 | ||||||||||||
Research and development | BEST1 License Agreement | University Of Florida Research Foundation (UFRF) And University Of Pennsylvania (Penn) | Other Licensed Product | Specified Commercial Sales Milestones Related to a Licensed Product | |||||||||||||
Commitments and contingencies | |||||||||||||
Payments for license fees | $ 9,600 |
Stock-Based Compensation and _3
Stock-Based Compensation and Compensation Plans - Narrative (Details) - USD ($) $ / shares in Units, $ in Thousands | 1 Months Ended | 12 Months Ended | |||||||
Feb. 28, 2021 | Mar. 31, 2020 | Apr. 30, 2016 | Aug. 31, 2013 | Dec. 31, 2021 | Dec. 31, 2020 | Dec. 31, 2019 | Dec. 31, 2018 | Oct. 31, 2019 | |
Stock Option and Compensation Plans | |||||||||
Options outstanding (in shares) | 8,526,000 | ||||||||
Weighted-average exercise price of options (in dollars per share) | $ 9.42 | ||||||||
Weighted-average remaining contractual life of options | 7 years 7 months 6 days | ||||||||
Options, vested and expected to vest, outstanding, aggregate intrinsic value | $ 20,600 | ||||||||
Share-based compensation expense | $ 8,323 | $ 9,180 | $ 11,072 | ||||||
Shares issued under ESPP (in shares) | 70,466 | ||||||||
ESPP | |||||||||
Stock Option and Compensation Plans | |||||||||
Number of shares of common stock available for issuance under the Plan (in shares) | 805,361 | ||||||||
Purchase price of common stock as percentage of fair market value | 85.00% | ||||||||
Share-based compensation expense | $ 100 | $ 100 | |||||||
Expected weighted average period to recognize share-based compensation costs | 2 months 12 days | ||||||||
Common stock issued (in shares) | 76,402 | ||||||||
Proceeds from stock plans | $ 147 | ||||||||
Restricted stock units | |||||||||
Stock Option and Compensation Plans | |||||||||
Share-based compensation expense | 0 | ||||||||
RSUs expected to vest (in shares) | 1,725,000 | ||||||||
Weighted-average fair value of RSUs expected to vest (in dollars per share) | $ 5.25 | ||||||||
Aggregate intrinsic value of RSUs expected to vest | $ 11,900 | ||||||||
Total fair value vested | 4,600 | ||||||||
Employees | Stock options | |||||||||
Stock Option and Compensation Plans | |||||||||
Share-based compensation expense | 4,700 | 5,900 | 7,600 | ||||||
Unrecognized share-based compensation costs for option awards, net of estimated forfeitures | $ 8,600 | ||||||||
Expected weighted average period to recognize share-based compensation costs | 5 years 9 months 18 days | ||||||||
Employees | Restricted stock units | |||||||||
Stock Option and Compensation Plans | |||||||||
Share-based compensation expense | $ 3,300 | 3,000 | 3,300 | ||||||
Expected weighted average period to recognize share-based compensation costs | 2 years | ||||||||
Unrecognized compensation costs, net of estimated forfeitures | $ 14,400 | ||||||||
Consultants | Non-employee Options | |||||||||
Stock Option and Compensation Plans | |||||||||
Share-based compensation expense | 200 | $ 300 | $ 200 | ||||||
Unrecognized share-based compensation costs for option awards, net of estimated forfeitures | $ 100 | ||||||||
Expected weighted average period to recognize share-based compensation costs | 2 years 4 months 24 days | ||||||||
Consultants | Restricted stock units | |||||||||
Stock Option and Compensation Plans | |||||||||
Expected weighted average period to recognize share-based compensation costs | 2 years 10 months 24 days | ||||||||
Unrecognized compensation costs, net of estimated forfeitures | $ 100 | ||||||||
Maximum | ESPP | |||||||||
Stock Option and Compensation Plans | |||||||||
Number of shares reserved for issuance under the Plan (in shares) | 1,000,000 | ||||||||
2013 Plan | |||||||||
Stock Option and Compensation Plans | |||||||||
Number of shares of common stock available for issuance under the Plan (in shares) | 421,000 | ||||||||
Annual increase in shares reserved for issuance under the Plan (in shares) | 2,542,372 | ||||||||
Annual increase in shares reserved for issuance under the Plan (as a percent) | 4.00% | ||||||||
Increase in number of shares available under the Plan (in shares) | 13,081,000 | ||||||||
2013 Plan | Subsequent Event | |||||||||
Stock Option and Compensation Plans | |||||||||
Increase in number of shares available under the Plan (in shares) | 2,542,000 | ||||||||
2013 Plan | Maximum | |||||||||
Stock Option and Compensation Plans | |||||||||
Number of shares reserved for issuance under the Plan (in shares) | 3,359,641 | ||||||||
2007 Plan | |||||||||
Stock Option and Compensation Plans | |||||||||
Number of shares of common stock available for issuance under the Plan (in shares) | 739,317 | ||||||||
2019 Inducement Plan | Stock options | |||||||||
Stock Option and Compensation Plans | |||||||||
Number of shares reserved for issuance under the Plan (in shares) | 1,000,000 | ||||||||
Number of shares of common stock available for issuance under the Plan (in shares) | 555,000 | ||||||||
Increase in number of shares available under the Plan (in shares) | 1,000,000 | ||||||||
2019 Inducement Plan | Stock options | Subsequent Event | |||||||||
Stock Option and Compensation Plans | |||||||||
Increase in number of shares available under the Plan (in shares) | 600,000 |
Stock-Based Compensation and _4
Stock-Based Compensation and Compensation Plans - Summary of Stock Option Activity (Details) - $ / shares shares in Thousands | 12 Months Ended | ||
Dec. 31, 2020 | Dec. 31, 2019 | Dec. 31, 2018 | |
Common Stock Options | |||
Outstanding at beginning of year (in shares) | 6,780 | 5,903 | 5,284 |
Granted (in shares) | 2,630 | 1,610 | 1,293 |
Exercised (in shares) | (109) | (80) | 0 |
Expired or forfeited (in shares) | (373) | (653) | (674) |
Outstanding at end of year (in shares) | 8,928 | 6,780 | 5,903 |
Weighted Average Exercise Price | |||
Outstanding at beginning of year (in dollars per share) | $ 10.89 | $ 13.72 | $ 19.58 |
Granted (in dollars per share) | 6.10 | 4.26 | 1.67 |
Exercised (in dollars per share) | 2.89 | 2.66 | 0 |
Expired or forfeited (in dollars per share) | 19.49 | 21.16 | 36.54 |
Outstanding at end of year (in dollars per share) | $ 9.22 | $ 10.89 | $ 13.72 |
Options exercisable at end of year (in shares) | 4,462 | 3,317 | 2,709 |
Weighted average grant date fair value (per share) of options granted during the period (in dollars per share) | $ 13.51 | $ 3.37 | $ 1.20 |
Stock-Based Compensation and _5
Stock-Based Compensation and Compensation Plans - Options Outstanding and Exercisable by Exercise Price and Options Exercised (Details) $ / shares in Units, shares in Thousands, $ in Thousands | 12 Months Ended |
Dec. 31, 2020USD ($)$ / sharesshares | |
Options Outstanding | |
Total Options Outstanding (in shares) | shares | 8,928 |
Weighted Average Remaining Life (Years) | 7 years 7 months 6 days |
Weighted Average Exercise Price (in dollars per share) | $ 9.22 |
Aggregate Intrinsic Value of Options Outstanding | $ | $ 20,553 |
Options Exercisable | |
Number Exercisable (in shares) | shares | 4,462 |
Weighted Average Exercise Price (in dollars per share) | $ 13.51 |
Aggregate Intrinsic Value of Options Exercisable | $ | $ 11,741 |
$1.13-$2.90 | |
Range of Exercise Prices | |
Exercise prices, low end of range (in dollars per share) | $ 1.13 |
Exercise prices, high end of range (in dollars per share) | $ 2.90 |
Options Outstanding | |
Total Options Outstanding (in shares) | shares | 1,352 |
Weighted Average Remaining Life (Years) | 7 years 8 months 12 days |
Weighted Average Exercise Price (in dollars per share) | $ 1.68 |
Options Exercisable | |
Number Exercisable (in shares) | shares | 808 |
Weighted Average Exercise Price (in dollars per share) | $ 1.79 |
$2.91-$3.18 | |
Range of Exercise Prices | |
Exercise prices, low end of range (in dollars per share) | 2.91 |
Exercise prices, high end of range (in dollars per share) | $ 3.18 |
Options Outstanding | |
Total Options Outstanding (in shares) | shares | 2,053 |
Weighted Average Remaining Life (Years) | 7 years 6 months |
Weighted Average Exercise Price (in dollars per share) | $ 2.99 |
Options Exercisable | |
Number Exercisable (in shares) | shares | 1,180 |
Weighted Average Exercise Price (in dollars per share) | $ 2.94 |
$3.19-$4.88 | |
Range of Exercise Prices | |
Exercise prices, low end of range (in dollars per share) | 3.19 |
Exercise prices, high end of range (in dollars per share) | $ 4.88 |
Options Outstanding | |
Total Options Outstanding (in shares) | shares | 1,790 |
Weighted Average Remaining Life (Years) | 7 years 8 months 12 days |
Weighted Average Exercise Price (in dollars per share) | $ 4.24 |
Options Exercisable | |
Number Exercisable (in shares) | shares | 1,022 |
Weighted Average Exercise Price (in dollars per share) | $ 4.39 |
$4.89-$8.77 | |
Range of Exercise Prices | |
Exercise prices, low end of range (in dollars per share) | 4.89 |
Exercise prices, high end of range (in dollars per share) | $ 8.77 |
Options Outstanding | |
Total Options Outstanding (in shares) | shares | 2,491 |
Weighted Average Remaining Life (Years) | 9 years 7 months 6 days |
Weighted Average Exercise Price (in dollars per share) | $ 6.64 |
Options Exercisable | |
Number Exercisable (in shares) | shares | 211 |
Weighted Average Exercise Price (in dollars per share) | $ 5.24 |
$8.78-$73.22 | |
Range of Exercise Prices | |
Exercise prices, low end of range (in dollars per share) | 8.78 |
Exercise prices, high end of range (in dollars per share) | $ 73.22 |
Options Outstanding | |
Total Options Outstanding (in shares) | shares | 1,242 |
Weighted Average Remaining Life (Years) | 3 years 9 months 18 days |
Weighted Average Exercise Price (in dollars per share) | $ 40.08 |
Options Exercisable | |
Number Exercisable (in shares) | shares | 1,241 |
Weighted Average Exercise Price (in dollars per share) | $ 40.08 |
Stock-Based Compensation and _6
Stock-Based Compensation and Compensation Plans - Schedule of Cash Proceeds From and Aggregate Intrinsic Value of Stock Options Exercised (Details) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2020 | Dec. 31, 2019 | Dec. 31, 2018 | |
Share-based Payment Arrangement [Abstract] | |||
Cash proceeds from options exercised | $ 314 | $ 192 | $ 0 |
Aggregate intrinsic value of options exercised | $ 393 | $ 135 | $ 0 |
Stock-Based Compensation and _7
Stock-Based Compensation and Compensation Plans - Summary of Outstanding RSU Awards Granted (Details) - Restricted stock units shares in Thousands | 12 Months Ended |
Dec. 31, 2020$ / sharesshares | |
Restricted Stock Units | |
Outstanding, beginning of period (in shares) | shares | 1,481 |
Awarded (in shares) | shares | 1,232 |
Vested (in shares) | shares | (657) |
Forfeited (in shares) | shares | (98) |
Outstanding, end of period (in shares) | shares | 1,958 |
Weighted Average Grant-Date Fair Value | |
Outstanding, beginning of period (in dollars per share) | $ / shares | $ 7.79 |
Awarded (in dollars per share) | $ / shares | 6.06 |
Vested (in dollars per share) | $ / shares | 7.20 |
Forfeited (in dollars per share) | $ / shares | 6.74 |
Outstanding, end of period (in dollars per share) | $ / shares | $ 6.95 |
Employee Benefit Plan (Details)
Employee Benefit Plan (Details) - USD ($) $ in Millions | 12 Months Ended | ||
Dec. 31, 2020 | Dec. 31, 2019 | Dec. 31, 2018 | |
Retirement Benefits [Abstract] | |||
Defined contribution plan, cost recognized | $ 0.3 | $ 0.2 | $ 0.2 |
Selected Quarterly Financial _3
Selected Quarterly Financial Information (unaudited) (Details) - USD ($) $ / shares in Units, $ in Thousands | 3 Months Ended | 12 Months Ended | |||||||||
Dec. 31, 2020 | Sep. 30, 2020 | Jun. 30, 2020 | Mar. 31, 2020 | Dec. 31, 2019 | Sep. 30, 2019 | Jun. 30, 2019 | Mar. 31, 2019 | Dec. 31, 2020 | Dec. 31, 2019 | Dec. 31, 2018 | |
Quarterly Financial Information Disclosure [Abstract] | |||||||||||
Research and development expenses | $ 17,473 | $ 18,841 | $ 12,720 | $ 13,750 | $ 11,567 | $ 10,383 | $ 10,009 | $ 7,685 | $ 62,784 | $ 39,644 | $ 41,737 |
General and administrative expenses | 8,022 | 6,643 | 6,289 | 4,998 | 6,275 | 4,674 | 5,198 | 5,481 | 25,952 | 21,628 | 23,612 |
Income (loss) from operations | (25,495) | (25,484) | (19,009) | (18,748) | (17,842) | (15,057) | (15,207) | (13,166) | $ (88,736) | $ (61,272) | $ (65,349) |
Net income (loss) attributable to common stockholders | $ (25,432) | $ (25,450) | $ (18,589) | $ (15,076) | $ (17,478) | $ (14,437) | $ (14,443) | $ (12,501) | |||
Basic (in dollars per share) | $ (0.27) | $ (0.27) | $ (0.32) | $ (0.28) | $ (0.39) | $ (0.35) | $ (0.35) | $ (0.30) | $ (1.14) | $ (1.39) | $ 1.70 |
Dilutive (in dollars per share) | $ (0.27) | $ (0.27) | $ (0.32) | $ (0.28) | $ (0.39) | $ (0.35) | $ (0.35) | $ (0.30) | $ (1.14) | $ (1.39) | $ 1.70 |