Cover
Cover - shares | 9 Months Ended | |
Sep. 30, 2021 | Nov. 05, 2021 | |
Cover [Abstract] | ||
Document Type | 10-Q | |
Document Quarterly Report | true | |
Document Period End Date | Sep. 30, 2021 | |
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, $0.001 par value per share | |
Trading Symbol | ISEE | |
Security Exchange Name | NASDAQ | |
Entity Current Reporting Status | Yes | |
Entity Interactive Data Current | Yes | |
Entity Filer Category | Non-accelerated Filer | |
Smaller Reporting Company | true | |
Emerging Growth Company | false | |
Entity Shell Company | false | |
Entity Common Stock, Shares Outstanding (in shares) | 114,641,279 | |
Entity Central Index Key | 0001410939 | |
Amendment Flag | false | |
Current Fiscal Year End Date | --12-31 | |
Document Fiscal Year Focus | 2021 | |
Document Fiscal Period Focus | Q3 |
Condensed Unaudited Consolidate
Condensed Unaudited Consolidated Balance Sheets - USD ($) $ in Thousands | Sep. 30, 2021 | Dec. 31, 2020 |
Current assets | ||
Cash and cash equivalents | $ 179,879 | $ 66,373 |
Available for sale securities | 62,091 | 143,674 |
Prepaid expenses and other current assets | 4,070 | 4,791 |
Income tax receivable | 0 | 1,765 |
Total current assets | 246,040 | 216,603 |
Property and equipment, net | 2 | 26 |
Right-of-use asset, net | 1,748 | 120 |
Other assets | 0 | 5 |
Total assets | 247,790 | 216,754 |
Current liabilities | ||
Accrued research and development expenses | 11,368 | 12,284 |
Accounts payable and accrued expenses | 8,130 | 12,792 |
Lease liability | 945 | 54 |
Total current liabilities | 20,443 | 25,130 |
Lease liability, non-current | 861 | 61 |
Total liabilities | 21,304 | 25,191 |
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, 104,237,878 and 90,120,797 shares issued and outstanding at September 30, 2021 and December 31, 2020, respectively | 104 | 90 |
Additional paid-in capital | 872,955 | 756,543 |
Accumulated deficit | (646,562) | (565,073) |
Accumulated other comprehensive income | (11) | 3 |
Total stockholders' equity | 226,486 | 191,563 |
Total liabilities and stockholders' equity | $ 247,790 | $ 216,754 |
Condensed Unaudited Consolida_2
Condensed Unaudited Consolidated Balance Sheets (Parenthetical) - $ / shares | Sep. 30, 2021 | Dec. 31, 2020 |
Statement of Financial Position [Abstract] | ||
Preferred stock, par value (in dollars per share) | $ 0.001 | $ 0.001 |
Preferred stock, shares authorized (in shares) | 5,000,000 | 5,000,000 |
Preferred stock, shares issued (in shares) | 0 | 0 |
Preferred stock, shares outstanding (in shares) | 0 | 0 |
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) | 104,237,878 | 90,120,797 |
Common stock, shares outstanding (in shares) | 104,237,878 | 90,120,797 |
Condensed Unaudited Consolida_3
Condensed Unaudited Consolidated Statements of Operations and Comprehensive Loss - USD ($) shares in Thousands | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2021 | Sep. 30, 2020 | Sep. 30, 2021 | Sep. 30, 2020 | |
Operating expenses: | ||||
Research and development | $ 17,935,000 | $ 18,841,000 | $ 59,972,000 | $ 45,311,000 |
General and administrative | 6,648,000 | 6,643,000 | 21,688,000 | 17,930,000 |
Total operating expenses | 24,583,000 | 25,484,000 | 81,660,000 | 63,241,000 |
Loss from operations | (24,583,000) | (25,484,000) | (81,660,000) | (63,241,000) |
Interest income | 42,000 | 33,000 | 184,000 | 437,000 |
Other expense, net | (10,000) | 1,000 | (13,000) | (6,000) |
Loss before income tax benefit | (24,551,000) | (25,450,000) | (81,489,000) | (62,810,000) |
Income tax benefit | 0 | 0 | 0 | 3,695,000 |
Net loss | (24,551,000) | (25,450,000) | (81,489,000) | (59,115,000) |
Comprehensive loss | $ (24,565,000) | $ (25,449,000) | $ (81,503,000) | $ (59,114,000) |
Net loss per common share: | ||||
Basic (in dollars per share) | $ (0.23) | $ (0.27) | $ (0.84) | $ (0.87) |
Diluted (in dollars per share) | $ (0.23) | $ (0.27) | $ (0.84) | $ (0.87) |
Weighted average common shares outstanding: | ||||
Basic (in shares) | 105,217 | 92,675 | 97,370 | 67,931 |
Diluted (in shares) | 105,217 | 92,675 | 97,370 | 67,931 |
Condensed Unaudited Consolida_4
Condensed Unaudited Consolidated Statements of Stockholders' Equity - USD ($) shares in Thousands, $ in Thousands | Total | Underwritten Offering | Private Placement | 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, 2019 | 0 | 49,627 | ||||||||||
Balance at beginning of period at Dec. 31, 2019 | $ 117,203 | $ 0 | $ 50 | $ 597,679 | $ (480,526) | $ 0 | ||||||
Increase (Decrease) in Stockholders' Equity | ||||||||||||
Issuance of common stock under employee stock compensation plan (in shares) | 105 | |||||||||||
Issuance of common stock under employee stock compensation plans | 179 | 179 | ||||||||||
Share-based compensation | 2,324 | 2,324 | ||||||||||
Net loss | (15,076) | (15,076) | ||||||||||
Balance at end of period (in shares) at Mar. 31, 2020 | 0 | 49,732 | ||||||||||
Balance at end of period at Mar. 31, 2020 | 104,630 | $ 0 | $ 50 | 600,182 | (495,602) | 0 | ||||||
Balance at beginning of period (in shares) at Dec. 31, 2019 | 0 | 49,627 | ||||||||||
Balance at beginning of period at Dec. 31, 2019 | 117,203 | $ 0 | $ 50 | 597,679 | (480,526) | 0 | ||||||
Increase (Decrease) in Stockholders' Equity | ||||||||||||
Net loss | (59,115) | |||||||||||
Balance at end of period (in shares) at Sep. 30, 2020 | 0 | 89,554 | ||||||||||
Balance at end of period at Sep. 30, 2020 | 214,586 | $ 0 | $ 90 | 754,136 | (539,641) | 1 | ||||||
Balance at beginning of period (in shares) at Mar. 31, 2020 | 0 | 49,732 | ||||||||||
Balance at beginning of period at Mar. 31, 2020 | 104,630 | $ 0 | $ 50 | 600,182 | (495,602) | 0 | ||||||
Increase (Decrease) in Stockholders' Equity | ||||||||||||
Issuance of common stock, net of issuance costs (in shares) | 28,503 | 8,649 | ||||||||||
Issuance of common stock, net of issuance costs | $ 116,883 | $ 33,237 | $ 28 | $ 9 | $ 116,855 | $ 33,228 | ||||||
Issuance of common stock under the exercise of pre-funded warrants (in shares) | 2,500 | |||||||||||
Issuance of common stock under the exercise of pre-funded warrants | 0 | $ 2 | (2) | |||||||||
Issuance of common stock under employee stock compensation plan (in shares) | 105 | |||||||||||
Issuance of common stock under employee stock compensation plans | 7 | 7 | ||||||||||
Share-based compensation | 1,873 | 1,873 | ||||||||||
Net loss | (18,589) | (18,589) | ||||||||||
Balance at end of period (in shares) at Jun. 30, 2020 | 0 | 89,489 | ||||||||||
Balance at end of period at Jun. 30, 2020 | 238,041 | $ 0 | $ 89 | 752,143 | (514,191) | 0 | ||||||
Increase (Decrease) in Stockholders' Equity | ||||||||||||
Issuance of common stock under employee stock compensation plan (in shares) | 65 | |||||||||||
Issuance of common stock under employee stock compensation plans | 147 | $ 1 | 146 | |||||||||
Share-based compensation | 1,847 | 1,847 | ||||||||||
Net loss | (25,450) | (25,450) | ||||||||||
Unrealized loss on available for sale securities, net of tax | 1 | 1 | ||||||||||
Balance at end of period (in shares) at Sep. 30, 2020 | 0 | 89,554 | ||||||||||
Balance at end of period at Sep. 30, 2020 | 214,586 | $ 0 | $ 90 | 754,136 | (539,641) | 1 | ||||||
Balance at beginning of period (in shares) at Dec. 31, 2020 | 0 | 90,121 | ||||||||||
Balance at beginning of period at Dec. 31, 2020 | 191,563 | $ 0 | $ 90 | 756,543 | (565,073) | 3 | ||||||
Increase (Decrease) in Stockholders' Equity | ||||||||||||
Issuance of common stock under employee stock compensation plan (in shares) | 49 | |||||||||||
Issuance of common stock under employee stock compensation plans | 129 | 129 | ||||||||||
Share-based compensation | 2,292 | 2,292 | ||||||||||
Net loss | (26,795) | (26,795) | ||||||||||
Unrealized loss on available for sale securities, net of tax | (1) | (1) | ||||||||||
Balance at end of period (in shares) at Mar. 31, 2021 | 0 | 90,170 | ||||||||||
Balance at end of period at Mar. 31, 2021 | 167,188 | $ 0 | $ 90 | 758,964 | (591,868) | 2 | ||||||
Balance at beginning of period (in shares) at Dec. 31, 2020 | 0 | 90,121 | ||||||||||
Balance at beginning of period at Dec. 31, 2020 | 191,563 | $ 0 | $ 90 | 756,543 | (565,073) | 3 | ||||||
Increase (Decrease) in Stockholders' Equity | ||||||||||||
Net loss | (81,489) | |||||||||||
Balance at end of period (in shares) at Sep. 30, 2021 | 0 | 104,238 | ||||||||||
Balance at end of period at Sep. 30, 2021 | 226,486 | $ 0 | $ 104 | 872,955 | (646,562) | (11) | ||||||
Balance at beginning of period (in shares) at Mar. 31, 2021 | 0 | 90,170 | ||||||||||
Balance at beginning of period at Mar. 31, 2021 | 167,188 | $ 0 | $ 90 | 758,964 | (591,868) | 2 | ||||||
Increase (Decrease) in Stockholders' Equity | ||||||||||||
Issuance of common stock under employee stock compensation plan (in shares) | 217 | |||||||||||
Issuance of common stock under employee stock compensation plans | 448 | 448 | ||||||||||
Share-based compensation | 2,079 | 2,079 | ||||||||||
Net loss | (30,143) | (30,143) | ||||||||||
Unrealized loss on available for sale securities, net of tax | 1 | 1 | ||||||||||
Balance at end of period (in shares) at Jun. 30, 2021 | 0 | 90,387 | ||||||||||
Balance at end of period at Jun. 30, 2021 | 139,573 | $ 0 | $ 90 | 761,491 | (622,011) | 3 | ||||||
Increase (Decrease) in Stockholders' Equity | ||||||||||||
Issuance of common stock, net of issuance costs (in shares) | 13,398 | |||||||||||
Issuance of common stock, net of issuance costs | $ 107,757 | $ 14 | $ 107,743 | |||||||||
Issuance of common stock under employee stock compensation plan (in shares) | 453 | |||||||||||
Issuance of common stock under employee stock compensation plans | 1,095 | 1,095 | ||||||||||
Share-based compensation | 2,626 | 2,626 | ||||||||||
Net loss | (24,551) | (24,551) | ||||||||||
Unrealized loss on available for sale securities, net of tax | (14) | (14) | ||||||||||
Balance at end of period (in shares) at Sep. 30, 2021 | 0 | 104,238 | ||||||||||
Balance at end of period at Sep. 30, 2021 | $ 226,486 | $ 0 | $ 104 | $ 872,955 | $ (646,562) | $ (11) |
Condensed Unaudited Consolida_5
Condensed Unaudited Consolidated Statements of Cash Flows - USD ($) $ in Thousands | 9 Months Ended | |
Sep. 30, 2021 | Sep. 30, 2020 | |
Operating Activities | ||
Net loss | $ (81,489) | $ (59,115) |
Adjustments to reconcile net loss to net cash used in operating activities | ||
Depreciation and other expense | 24 | 108 |
Amortization of premium and discounts on investment securities | 918 | 115 |
Share-based compensation | 6,997 | 6,044 |
Changes in operating assets and liabilities: | ||
Income tax receivable | 1,765 | 0 |
Prepaid expense and other assets | 726 | 542 |
Accrued interest receivable | 449 | (112) |
Accrued research and development expenses | (916) | 6,007 |
Accounts payable and accrued expenses | (4,662) | 1,383 |
Change in working capital | 63 | 0 |
Net cash used in operating activities | (76,125) | (45,028) |
Investing Activities | ||
Purchase of marketable securities | (56,245) | (108,305) |
Maturities of marketable securities | 136,447 | 0 |
Net cash provided by (used in) investing activities | 80,202 | (108,305) |
Financing Activities | ||
Proceeds from employee stock plan purchases | 1,672 | 333 |
Net cash provided by financing activities | 109,429 | 150,453 |
Net increase (decrease) in cash and cash equivalents | 113,506 | (2,880) |
Cash and cash equivalents | ||
Beginning of period | 66,373 | 125,699 |
End of period | 179,879 | 122,819 |
Supplemental disclosure of cash paid | ||
Income tax refunds received | 1,765 | 3,327 |
Underwritten Offering | ||
Financing Activities | ||
Proceeds from issuance of common stock | 107,757 | 116,883 |
Private Placement | ||
Financing Activities | ||
Proceeds from issuance of common stock | $ 0 | $ 33,237 |
Business
Business | 9 Months Ended |
Sep. 30, 2021 | |
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 treatments for retinal diseases with significant unmet medical needs. The Company is committed to having a positive impact on patients’ lives by delivering high-quality, safe and effective treatments designed to address debilitating retina diseases including all stages of age-related macular degeneration (“AMD”). The Company’s lead asset is its clinical stage product candidate Zimura® (avacincaptad pegol), a complement C5 inhibitor. It is currently targeting the following diseases with Zimura: • Geographic Atrophy (“GA”), which is the advanced stage of AMD, and is characterized by marked thinning or atrophy of retinal tissue, leading to irreversible loss of vision; • intermediate AMD, which is an earlier stage of AMD that precedes GA; 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. In July 2021, the Company completed patient enrollment for GATHER2, its Phase 3 clinical trial evaluating the safety and efficacy of Zimura for the treatment of GA secondary to AMD. The Company has also received a written agreement from the U.S. Food and Drug Administration (“FDA”) under a Special Protocol Assessment for the overall design of GATHER2. The Company expects topline data from the GATHER2 trial to become available during the second half of 2022, approximately one year after the enrollment of the last patient plus the time needed for database lock and analysis. The Company also plans to initiate a Phase 3 clinical trial evaluating Zimura for patients with intermediate AMD during 2022. The Company expects data from this trial, if positive, together with other supportive data, may be sufficient to file a supplemental new drug application with the FDA and a supplemental marketing authorization application with the European Medicines Agency. In addition to Zimura, the Company is developing its preclinical product candidate IC-500, a High temperature requirement A serine peptidase 1 protein (“HtrA1”) inhibitor, for GA secondary to AMD and potentially other age-related retinal diseases. Based on current timelines, the Company expects to submit an investigational new drug application to the FDA for IC-500 during the second half of 2022. The Company’s portfolio also includes two preclinical stage gene therapy product candidates (IC-100 and IC-200) and several ongoing gene therapy research programs, each of which uses adeno-associated virus (“AAV”) for gene delivery. These AAV mediated gene therapy programs are targeting the following orphan inherited retinal diseases (“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”); • Leber Congenital Amaurosis type 10 (“LCA10”), which is characterized by severe bilateral loss of vision at or soon after birth; • STGD1; and • IRDs associated with mutations in the USH2A gene, which include Usher syndrome type 2A, and USH2A-associated non-syndromic autosomal recessive retinitis pigmentosa. |
Summary of Significant Accounti
Summary of Significant Accounting Policies | 9 Months Ended |
Sep. 30, 2021 | |
Accounting Policies [Abstract] | |
Summary of Significant Accounting Policies | Summary of Significant Accounting Policies The Company’s significant accounting policies are described in Note 2, “Summary of Significant Accounting Policies,” in the notes to the audited consolidated financial statements included in the Company’s Annual Report on Form 10-K (“Annual Report”) for the year ended December 31, 2020 filed with th e Securities and Exchange Commission (“SEC”) on March 4, 2021. Basis of Presentation and Consolidation In the opinion of management, the Company’s condensed consolidated financial statements include all adjustments, consisting of normal recurring accruals, necessary for a fair statement of the Company’s financial statements for interim periods in accordance with accounting principles generally accepted in the United States (“U.S. GAAP”). The consolidated financial statements include the accounts of the Company and its subsidiaries. All intercompany balances and transactions have been eliminated in consolidation. The information included in this quarterly report on Form 10-Q should be read in conjunction with the Company’s audited consolidated financial statements and the accompanying notes included in the Annual Report. The year-end condensed consolidated balance sheet data presented for comparative purposes was derived from the Company’s audited financial statements but does not include all disclosures required by U.S. GAAP. The results of operations for the nine months ended September 30, 2021 are not necessarily indicative of the operating results for the full year or for any other subsequent interim period. 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 reportable segment. Use of Estimates The preparation of financial statements and related disclosures in conformity with U.S. 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 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. Financial Instruments Cash equivalents and available for sale securities 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. Accounting Standards Codification, or 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. 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 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. 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 balances 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, 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, cash equivalents and available for sale securities. 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 fill/finish services 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. Leases The Company determines if an arrangement contains a lease at inception. For arrangements where the Company is the lessee, it recognizes a right-of-use (“ROU”) 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. 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 and laboratory 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 gene therapy 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, non-employees and non-employee directors, 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-employee directors and consultants 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 nine month periods ended September 30, 2021 and 2020: Three Months Ended September 30, Nine Months Ended September 30, 2021 2020 2021 2020 Expected common stock price volatility 113% 117% 113% 117% Risk-free interest rate 0.68%-0.84% 0.22%-0.26% 0.31%-0.96% 0.22%-1.34% Expected term of options (years) 5.2 4.8 5.2 4.6 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. Recent Accounting Pronouncements The Company has evaluated recent accounting pronouncements through the date the financial statements were issued and filed with the SEC and believes that there are none that will have a material impact on the Company’s financial statements. |
Common Stock
Common Stock | 9 Months Ended |
Sep. 30, 2021 | |
Equity [Abstract] | |
Common Stock | Common Stock In July 2021, the Company completed an underwritten public offering in which the Company sold 13,397,500 shares of its common stock, which included the exercise in full of the underwriters’ option to purchase an additional 1,747,500 shares of the Company’s common stock, at a price to the public of $8.60 per share and at a price to the underwriters of $8.084 per share. The net proceeds from the July 2021 public offering, after deducting underwriting discounts and commissions and other expenses payable by the Company totaling approximately $7.4 million, was approximately $107.8 million. In June 2020, the Company completed an underwritten public offering in which the Company sold 28,503,220 shares of its common stock, which included the exercise in full of the underwriters’ option to purchase additional shares of the Company’s common stock, at a price to the public of $4.100 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. Concurrently with the June 2020 public offering, the Company completed a private placement in which the Company sold 8,649,453 shares of its common stock to affiliates of Vivo Capital, LLC and Samsara BioCapital, LP, at a sale price equal to the price to the public in the underwritten public offering. The net proceeds from the June 2020 public offering and private placement, after deducting underwriting discounts, underwriting and placement agent commissions and other expenses totaling approximately $10.1 million, was approximately $150.1 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 earnings per share calculations. |
Net Loss Per Common Share
Net Loss Per Common Share | 9 Months Ended |
Sep. 30, 2021 | |
Earnings Per Share [Abstract] | |
Net Loss Per Common Share | Net Loss Per Common Share Basic 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 September 30, 2021 and September 30, 2020, the Company had 3,164,280 pre-funded warrants outstanding, 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 loss per common share for the periods indicated: Three Months Ended September 30, Nine Months Ended September 30, 2021 2020 2021 2020 Basic and diluted net loss per common share calculation: Net loss $ (24,551) $ (25,450) $ (81,489) $ (59,115) Weighted average common shares outstanding - basic and dilutive 105,217 92,675 97,370 67,931 Net loss per share of common stock - basic and diluted $ (0.23) $ (0.27) $ (0.84) $ (0.87) 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: Three Months Ended September 30, Nine Months Ended September 30, 2021 2020 2021 2020 Stock options outstanding 9,089 7,426 9,089 7,426 Restricted stock units 1,903 1,727 1,903 1,727 Total 10,992 9,153 10,992 9,153 |
Cash, Cash Equivalents and Avai
Cash, Cash Equivalents and Available for Sale Securities | 9 Months Ended |
Sep. 30, 2021 | |
Cash, Cash Equivalents, and Short-term Investments [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 September 30, 2021 and December 31, 2020, the Company had cash and cash equivalents of approximately $179.9 million and $66.4 million, respectively. Cash and cash equivalents included cash of $97.0 million at September 30, 2021 and $8.4 million at December 31, 2020. Cash and cash equivalents at September 30, 2021 and December 31, 2020 included $82.9 million and $58.0 million, respectively, of investments in money market funds. The Company considers securities with original maturities of greater than 90 days at the date of purchase to be available for sale securities. As of September 30, 2021 and December 31, 2020, the Company held available for sale securities of $62.1 million and $143.7 million, respectively, all of which have maturities of less than one year. 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 September 30, 2021. 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. The Company believes that its existing cash, cash equivalents and available for sale securities as of September 30, 2021 will be sufficient to fund its currently planned capital expenditure requirements and operating expenses for at least the next 12 months from the filing of this Quarterly Report on Form 10-Q. See Note 10 to the Financial Statements for information about the Company’s follow-on public offering completed in October 2021. Available for sale securities, including carrying value and estimated fair values, are summarized as follows: As of September 30, 2021 Amortized Cost Gross Unrealized Gains Gross Unrealized Losses Fair Value U.S. Treasury securities $ 8,999 $ — $ — $ 8,999 Corporate debt securities 43,614 — (8) 43,606 Asset-backed securities 9,489 — (3) 9,486 Total $ 62,102 $ — $ (11) $ 62,091 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 other comprehensive income in the statements of comprehensive 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 associated with the unrealized gain on available for sale securities during the three and nine months ended September 30, 2021 and 2020, respectively, were as follows: Three months ended September 30, Nine Months Ended September 30, 2021 2021 2020 2021 2020 Beginning balance $ 3 $ — $ 3 $ — Current period changes in fair value before reclassifications, net of tax (14) 1 (14) 1 Amounts reclassified from accumulated other comprehensive income, net of tax — — — — Total other comprehensive income (loss) $ (14) $ 1 (14) 1 Ending balance $ (11) $ 1 $ (11) $ 1 |
Share-Based Compensation
Share-Based Compensation | 9 Months Ended |
Sep. 30, 2021 | |
Share-based Payment Arrangement [Abstract] | |
Share-Based Compensation | Share-Based Compensation Pursuant to the evergreen provisions of the Company's 2013 stock incentive plan (the “2013 Plan”), annual increases 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 September 30, 2021, the Company had approximately 3,031,000 shares available for grant under the 2013 Plan. In October 2019, the Company's board of directors adopted its 2019 Inducement Stock Incentive Plan (the “2019 Inducement Plan”) to reserve initially 1,000,000 shares of its common stock to be used exclusively for grants of awards to individuals who were not previously employees or directors of the Company as a material inducement to such individuals’ entry into employment with the Company within Rule 5635(c)(4) of the Nasdaq Listing Rules. The terms and conditions of the 2019 Inducement Plan are substantially similar to those of the 2013 Plan. 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 and September 2021, the Company's board of directors further amended the 2019 Inducement Plan to reserve an additional 600,000 shares, and an additional 1,000,000 shares, respectively, of its common stock for issuance under the plan. As of September 30, 2021, the Company had approximately 1,314,000 shares available for grant under the 2019 Inducement Plan. Share-based compensation expense, net of estimated forfeitures, includes expenses related to stock options and RSUs granted to employees, non-employee directors and consultants, as well as options granted to employees to purchase shares under the ESPP. Stock-based compensation by award type was as follows: Three Months Ended September 30, Nine Months Ended September 30, 2021 2020 2021 2020 Stock options $ 1,485 $ 1,089 $ 3,997 $ 3,556 Restricted stock units 1,101 723 2,896 2,411 Employee stock purchase plan 40 35 104 77 Total $ 2,626 $ 1,847 $ 6,997 $ 6,044 The Company allocated stock-based compensation expense in the Company’s Consolidated Statements of Operations and Comprehensive Loss as follows: Three Months Ended September 30, Nine Months Ended September 30, 2021 2020 2021 2020 Research and development $ 1,476 $ 882 $ 4,138 $ 2,986 General and administrative 1,150 965 2,859 3,058 Total $ 2,626 $ 1,847 $ 6,997 $ 6,044 Stock Options A summary of the stock option activity, weighted average exercise prices, options outstanding, exercisable and expected to vest as of September 30, 2021 is as follows (in thousands except weighted average exercise price): Number of Shares Underlying Options Weighted Outstanding, December 31, 2020 8,928 $ 9.22 Granted 969 $ 7.84 Exercised (487) $ 2.89 Forfeited (321) $ 4.49 Outstanding, September 30, 2021 9,089 $ 9.85 Vested and exercisable, September 30, 2021 5,154 $ 12.71 Vested and expected to vest, September 30, 2021 8,768 $ 9.99 As of September 30, 2021, there were approximately $15.3 million of unrecognized compensation costs, net of estimated forfeitures, related to stock option awards grants, which are expected to be recognized over a remaining weighted average period of 3.0 years. RSUs The following table presents a summary of the Company's outstanding RSU awards granted as of September 30, 2021 (in thousands except weighted average grant-date fair value): Restricted Weighted Average Outstanding, December 31, 2020 1,958 $ 6.95 Awarded 329 $ 7.44 Vested (181) $ 4.32 Forfeited (203) $ 21.60 Outstanding, September 30, 2021 1,903 $ 5.76 Outstanding, Expected to vest 1,750 $ 5.76 As of September 30, 2021, there were approximately $7.5 million of unrecognized compensation costs, net of estimated forfeitures, related to RSUs grants, which are expected to be recognized over a remaining weighted average period of 2.7 years. ESPP |
Income Taxes
Income Taxes | 9 Months Ended |
Sep. 30, 2021 | |
Income Tax Disclosure [Abstract] | |
Income Taxes | Income Taxes For the three and nine months ended September 30, 2021 and the three months ended September 2020, the Company recorded no income tax benefit. For the nine months ended September 30, 2020, the Company recorded a $3.7 million income tax benefit primarily to reflect a settlement of a local tax audit. In response to the COVID-19 pandemic, the U.S. Congress enacted the Coronavirus Aid, Relief, and Economic Security Act (“CARES Act”) on March 27, 2020. The CARES Act provides numerous tax provisions and other stimulus measures, including the immediate refund of minimum tax credits. In April 2021, the Company received the remaining balance, approximately $1.8 million, of its minimum tax credits refund. The Company will continue to evaluate its ability to realize its deferred tax assets on a quarterly basis and will adjust such amounts in light of changing facts and circumstances including, but not limited to, future projections of taxable income, tax |
Fair Value Measurements
Fair Value Measurements | 9 Months Ended |
Sep. 30, 2021 | |
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 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 September 30, 2021: Fair Value Measurement Using Quoted prices in Significant other Significant Assets Investments in money market funds* $ 82,872 $ — $ — Investments in U.S. Treasury securities $ 8,999 $ — $ — Investments in corporate debt securities $ — $ 43,606 $ — Investments in asset-backed securities $ — $ 9,486 $ — * Investments in money market funds are reflected in cash and cash equivalents in the accompanying Consolidated Balance Sheets. 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 — * Investments in money market funds are reflected in cash and cash equivalents in the accompanying Consolidated Balance Sheets. No transfer of assets between Level 1 and Level 2 of the fair value measurement hierarchy occurred during the three and nine months ended September 30, 2021. |
Commitments and Contingencies
Commitments and Contingencies | 9 Months Ended |
Sep. 30, 2021 | |
Commitments and Contingencies Disclosure [Abstract] | |
Commitments and Contingencies | Commitments and Contingencies Zimura - Archemix Corp. The Company is party to an agreement with Archemix Corp. (“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”). Under 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 products licensed under the agreement. 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 and the University of Pennsylvania Under its exclusive license agreement with the University of Florida Research Foundation, Incorporated (“UFRF”) and the University of Pennsylvania (“Penn”) for rights to IC-100, the Company is obligated to make payments to UFRF, for the benefit of Penn and UFRF (together, 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 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-teen 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 its exclusive license agreement with the University of Massachusetts (“UMass”) for its miniCEP290 program, which targets LCA10, which is associated with mutations in the CEP290 gene, 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 agreement and plan of merger between the Company and Inception 4, Inc. (“Inception 4”), pursuant to which the Company acquired IC-500 and its other HtrA1 inhibitors (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 IC-500 or any other product candidate from its HtrA1 inhibitor program, 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 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. On April 23, 2021, the court issued an order staying the action until July 1, 2021, 10 days after a mediation scheduled for June 21, 2021. On July 1, 2021, following the June 21, 2021 mediation, the parties notified the court that they had reached an agreement in principle to settle the class action. On September 8, 2021, the parties executed a settlement agreement, which has been submitted to the court for approval. The Company does not expect this settlement, if finalized as currently contemplated, to have a material impact on its financial condition. 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 (“SLC”) 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 SLC 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 June 26, 2021. The Company also entered into tolling agreements with the defendant directors to December 2021. The SLC and the plaintiffs in this derivative matter and the one described below recently agreed in principle on a settlement and are conducting settlement agreement discussions. 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 were referred to a demand review committee of the Company's board of directors. On May 6, 2021, the shareholders who served the October 16, 2018 demand filed a shareholder derivative action against current and former members of the Company’s Board of Directors and certain current and former officers of the Company in the New York Supreme Court, captioned Brian Ferber et al., derivatively on behalf of Ophthotech Corporation v. Axel Bolte et al., Index No. 154462/2021. The complaint asserts the same claims as those asserted in the Pacheco complaint and is based on factual allegations that are materially similar to the allegations in the Pacheco complaint. On June 22, 2021, the parties filed a stipulation staying the Ferber action until 60 days after the SLC concludes its investigation. The Company has entered into tolling agreements with the directors named in the demands to December 2021. The SLC and the plaintiffs in this derivative matter and the one described above recently agreed in principle on a settlement and are conducting settlement agreement discussions. 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, including that of the settlement agreement discussions, 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. |
Subsequent Events
Subsequent Events | 9 Months Ended |
Sep. 30, 2021 | |
Subsequent Events [Abstract] | |
Subsequent Events | Subsequent EventsIn October 2021, the Company completed an underwritten public offering in which the Company sold 10,350,000 shares of its common stock, which included the exercise in full of the underwriters’ option to purchase 1,350,000 shares of the Company’s common stock, at a price to the public of $16.75 per share and at a price to the underwriters of $15.745 per share. The net proceeds from the October 2021 public offering, after deducting underwriting discounts and commissions and other expenses payable by the Company totaling approximately $10.8 million, was approximately $162.6 million. |
Summary of Significant Accoun_2
Summary of Significant Accounting Policies - (Policies) | 9 Months Ended |
Sep. 30, 2021 | |
Accounting Policies [Abstract] | |
Basis of Presentation and Consolidation | Basis of Presentation and ConsolidationIn the opinion of management, the Company’s condensed consolidated financial statements include all adjustments, consisting of normal recurring accruals, necessary for a fair statement of the Company’s financial statements for interim periods in accordance with accounting principles generally accepted in the United States (“U.S. GAAP”). |
Consolidation | The consolidated financial statements include the accounts of the Company and its subsidiaries. All intercompany balances and transactions have been eliminated in consolidation. The information included in this quarterly report on Form 10-Q should be read in conjunction with the Company’s audited consolidated financial statements and the accompanying notes included in the Annual Report. The year-end condensed consolidated balance sheet data presented for comparative purposes was derived from the Company’s audited financial statements but does not include all disclosures required by U.S. GAAP. The results of operations for the nine months ended September 30, 2021 are not necessarily indicative of the operating results for the full year or for any other subsequent interim period. |
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. |
Use of Estimates | Use of Estimates The preparation of financial statements and related disclosures in conformity with U.S. 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 and Cash Equivalents 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 | 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. |
Financial Instruments | Financial Instruments Cash equivalents and available for sale securities 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. Accounting Standards Codification, or 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. 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 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. |
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 balances 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, 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, cash equivalents and available for sale securities. |
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 fill/finish services 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. |
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-of-use (“ROU”) 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. 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 Property and equipment, which consists mainly of clinical and laboratory 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 gene therapy 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, non-employees and non-employee directors, 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-employee directors and consultants 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. 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. |
Recent Accounting Pronouncements | Recent Accounting Pronouncements The Company has evaluated recent accounting pronouncements through the date the financial statements were issued and filed with the SEC and believes that there are none that will have a material impact on the Company’s financial statements. |
Summary of Significant Accoun_3
Summary of Significant Accounting Policies - (Tables) | 9 Months Ended |
Sep. 30, 2021 | |
Accounting Policies [Abstract] | |
Schedule of weighted-average assumptions 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 nine month periods ended September 30, 2021 and 2020: Three Months Ended September 30, Nine Months Ended September 30, 2021 2020 2021 2020 Expected common stock price volatility 113% 117% 113% 117% Risk-free interest rate 0.68%-0.84% 0.22%-0.26% 0.31%-0.96% 0.22%-1.34% Expected term of options (years) 5.2 4.8 5.2 4.6 Expected dividend yield — — — — |
Net Loss Per Common Share - (Ta
Net Loss Per Common Share - (Tables) | 9 Months Ended |
Sep. 30, 2021 | |
Earnings Per Share [Abstract] | |
Schedule of computation of basic and diluted net loss per common share | The following table sets forth the computation of basic and diluted net loss per common share for the periods indicated: Three Months Ended September 30, Nine Months Ended September 30, 2021 2020 2021 2020 Basic and diluted net loss per common share calculation: Net loss $ (24,551) $ (25,450) $ (81,489) $ (59,115) Weighted average common shares outstanding - basic and dilutive 105,217 92,675 97,370 67,931 Net loss per share of common stock - basic and diluted $ (0.23) $ (0.27) $ (0.84) $ (0.87) |
Schedule of potentially dilutive securities that have been 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: Three Months Ended September 30, Nine Months Ended September 30, 2021 2020 2021 2020 Stock options outstanding 9,089 7,426 9,089 7,426 Restricted stock units 1,903 1,727 1,903 1,727 Total 10,992 9,153 10,992 9,153 |
Cash, Cash Equivalents and Av_2
Cash, Cash Equivalents and Available for Sale Securities (Tables) | 9 Months Ended |
Sep. 30, 2021 | |
Cash, Cash Equivalents, and Short-term Investments [Abstract] | |
Schedule of Available for Sale Securities | Available for sale securities, including carrying value and estimated fair values, are summarized as follows: As of September 30, 2021 Amortized Cost Gross Unrealized Gains Gross Unrealized Losses Fair Value U.S. Treasury securities $ 8,999 $ — $ — $ 8,999 Corporate debt securities 43,614 — (8) 43,606 Asset-backed securities 9,489 — (3) 9,486 Total $ 62,102 $ — $ (11) $ 62,091 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 Accumulated Other Comprehensive Income | The changes in accumulated other comprehensive income associated with the unrealized gain on available for sale securities during the three and nine months ended September 30, 2021 and 2020, respectively, were as follows: Three months ended September 30, Nine Months Ended September 30, 2021 2021 2020 2021 2020 Beginning balance $ 3 $ — $ 3 $ — Current period changes in fair value before reclassifications, net of tax (14) 1 (14) 1 Amounts reclassified from accumulated other comprehensive income, net of tax — — — — Total other comprehensive income (loss) $ (14) $ 1 (14) 1 Ending balance $ (11) $ 1 $ (11) $ 1 |
Share-Based Compensation - (Tab
Share-Based Compensation - (Tables) | 9 Months Ended |
Sep. 30, 2021 | |
Share-based Payment Arrangement [Abstract] | |
Schedule of Allocation of Share-based Compensation Costs by Award Type | Stock-based compensation by award type was as follows: Three Months Ended September 30, Nine Months Ended September 30, 2021 2020 2021 2020 Stock options $ 1,485 $ 1,089 $ 3,997 $ 3,556 Restricted stock units 1,101 723 2,896 2,411 Employee stock purchase plan 40 35 104 77 Total $ 2,626 $ 1,847 $ 6,997 $ 6,044 |
Schedule of Allocation of Share-based Compensation Costs by Financial Statement Line | The Company allocated stock-based compensation expense in the Company’s Consolidated Statements of Operations and Comprehensive Loss as follows: Three Months Ended September 30, Nine Months Ended September 30, 2021 2020 2021 2020 Research and development $ 1,476 $ 882 $ 4,138 $ 2,986 General and administrative 1,150 965 2,859 3,058 Total $ 2,626 $ 1,847 $ 6,997 $ 6,044 |
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, exercisable and expected to vest as of September 30, 2021 is as follows (in thousands except weighted average exercise price): Number of Shares Underlying Options Weighted Outstanding, December 31, 2020 8,928 $ 9.22 Granted 969 $ 7.84 Exercised (487) $ 2.89 Forfeited (321) $ 4.49 Outstanding, September 30, 2021 9,089 $ 9.85 Vested and exercisable, September 30, 2021 5,154 $ 12.71 Vested and expected to vest, September 30, 2021 8,768 $ 9.99 |
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 September 30, 2021 (in thousands except weighted average grant-date fair value): Restricted Weighted Average Outstanding, December 31, 2020 1,958 $ 6.95 Awarded 329 $ 7.44 Vested (181) $ 4.32 Forfeited (203) $ 21.60 Outstanding, September 30, 2021 1,903 $ 5.76 Outstanding, Expected to vest 1,750 $ 5.76 |
Fair Value Measurements - (Tabl
Fair Value Measurements - (Tables) | 9 Months Ended |
Sep. 30, 2021 | |
Fair Value Disclosures [Abstract] | |
Schedule of assets and liabilities that are 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 September 30, 2021: Fair Value Measurement Using Quoted prices in Significant other Significant Assets Investments in money market funds* $ 82,872 $ — $ — Investments in U.S. Treasury securities $ 8,999 $ — $ — Investments in corporate debt securities $ — $ 43,606 $ — Investments in asset-backed securities $ — $ 9,486 $ — * Investments in money market funds are reflected in cash and cash equivalents in the accompanying Consolidated Balance Sheets. 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 — * Investments in money market funds are reflected in cash and cash equivalents in the accompanying Consolidated Balance Sheets. |
Business - Narrative (Details)
Business - Narrative (Details) | Sep. 30, 2021segment |
Adeno-Associated Virus Mediated Gene Therapy Program | |
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items] | |
Number of product candidates in preclinical development | 2 |
Summary of Significant Accoun_4
Summary of Significant Accounting Policies - Segment and Geographic Information (Details) | 9 Months Ended |
Sep. 30, 2021segment | |
Accounting Policies [Abstract] | |
Number of operating segments | 1 |
Number of reporting segments | 1 |
Summary of Significant Accoun_5
Summary of Significant Accounting Policies - Property and Equipment (Details) | 9 Months Ended |
Sep. 30, 2021 | |
Minimum | |
Property and equipment | |
Estimated useful live (in years) | 3 years |
Maximum | |
Property and equipment | |
Estimated useful live (in years) | 10 years |
Summary of Significant Accoun_6
Summary of Significant Accounting Policies - Share-Based Compensation (Details) - shares | 1 Months Ended | 9 Months Ended |
Apr. 30, 2016 | Sep. 30, 2021 | |
Share-Based Compensation | ||
Expected dividend yield | 0.00% | |
Employee stock purchase plan | ||
Share-Based Compensation | ||
Purchase period (in months) | 6 months | |
Employee stock purchase plan | Maximum | ||
Share-Based Compensation | ||
Number of initial shares reserved for issuance under the Plan (in shares) | 1,000,000 |
Summary of Significant Accoun_7
Summary of Significant Accounting Policies - Schedule of Weighted-Average Assumptions (Details) | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2021 | Sep. 30, 2020 | Sep. 30, 2021 | Sep. 30, 2020 | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Expected dividend yield | 0.00% | |||
Stock options | ||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Expected common stock price volatility | 113.00% | 117.00% | 113.00% | 117.00% |
Risk-free interest rate, minimum | 0.68% | 0.22% | 0.31% | 0.22% |
Risk-free interest rate, maximum | 0.84% | 0.26% | 0.96% | 1.34% |
Expected term of options (years) | 5 years 2 months 12 days | 4 years 9 months 18 days | 5 years 2 months 12 days | 4 years 7 months 6 days |
Expected dividend yield | 0.00% | 0.00% | 0.00% | 0.00% |
Common Stock (Details)
Common Stock (Details) - USD ($) $ / shares in Units, $ in Thousands | 1 Months Ended | 9 Months Ended | |||
Jul. 31, 2021 | Jun. 30, 2020 | Sep. 30, 2021 | Sep. 30, 2020 | Jul. 30, 2021 | |
Subsidiary, Sale of Stock [Line Items] | |||||
Payments of stock issuance costs | $ 7,400 | $ 10,100 | |||
Proceeds from issuance of common stock | $ 150,100 | ||||
Warrants issued in transaction (in shares) | 1,914,280 | ||||
Public Stock Offering | |||||
Subsidiary, Sale of Stock [Line Items] | |||||
Number of shares issued in transaction (in shares) | 13,397,500 | ||||
Sale of stock price (in dollars per share) | $ 8.60 | ||||
Proceeds from issuance of common stock | $ 107,800 | ||||
Over-Allotment Option | |||||
Subsidiary, Sale of Stock [Line Items] | |||||
Number of shares issued in transaction (in shares) | 1,747,500 | ||||
Sale of stock price (in dollars per share) | $ 8.084 | ||||
Underwritten Offering | |||||
Subsidiary, Sale of Stock [Line Items] | |||||
Number of shares issued in transaction (in shares) | 28,503,220 | ||||
Sale of stock price (in dollars per share) | $ 4.100 | ||||
Proceeds from issuance of common stock | $ 107,757 | $ 116,883 | |||
Underwritten Offering, Underwriters | |||||
Subsidiary, Sale of Stock [Line Items] | |||||
Sale of stock price (in dollars per share) | $ 3.854 | ||||
Private Placement | |||||
Subsidiary, Sale of Stock [Line Items] | |||||
Number of shares issued in transaction (in shares) | 8,649,453 | ||||
Proceeds from issuance of common stock | $ 0 | $ 33,237 | |||
Exercise price of pre-funded warrants (in dollars per share) | $ 4.099 | ||||
Private Placement, Underwriters | |||||
Subsidiary, Sale of Stock [Line Items] | |||||
Exercise price of pre-funded warrants (in dollars per share) | $ 3.853 |
Net Loss Per Common Share - Nar
Net Loss Per Common Share - Narrative (Details) - shares | Sep. 30, 2021 | Sep. 30, 2020 |
Earnings Per Share [Abstract] | ||
Pre-funded warrants outstanding (in shares) | 3,164,280 | 3,164,280 |
Net Loss Per Common Share -Sche
Net Loss Per Common Share -Schedules of Net Loss Per Share and Dilutive Securities (Details) - USD ($) $ / shares in Units, shares in Thousands, $ in Thousands | 3 Months Ended | 9 Months Ended | ||||||
Sep. 30, 2021 | Jun. 30, 2021 | Mar. 31, 2021 | Sep. 30, 2020 | Jun. 30, 2020 | Mar. 31, 2020 | Sep. 30, 2021 | Sep. 30, 2020 | |
Basic and diluted net loss per common share calculation: | ||||||||
Net loss | $ (24,551) | $ (30,143) | $ (26,795) | $ (25,450) | $ (18,589) | $ (15,076) | $ (81,489) | $ (59,115) |
Weighted average common shares outstanding - basic (in shares) | 105,217 | 92,675 | 97,370 | 67,931 | ||||
Weighted average common shares outstanding - dilutive (in shares) | 105,217 | 92,675 | 97,370 | 67,931 | ||||
Net loss per share of common stock - basic (in dollars per share) | $ (0.23) | $ (0.27) | $ (0.84) | $ (0.87) | ||||
Net loss per share of common stock - diluted (in dollars per share) | $ (0.23) | $ (0.27) | $ (0.84) | $ (0.87) | ||||
Anti-dilutive securities | ||||||||
Total (in shares) | 10,992 | 9,153 | 10,992 | 9,153 | ||||
Stock options outstanding | ||||||||
Anti-dilutive securities | ||||||||
Total (in shares) | 9,089 | 7,426 | 9,089 | 7,426 | ||||
Restricted stock units | ||||||||
Anti-dilutive securities | ||||||||
Total (in shares) | 1,903 | 1,727 | 1,903 | 1,727 |
Cash, Cash Equivalents and Av_3
Cash, Cash Equivalents and Available for Sale Securities - Narrative (Details) - USD ($) $ in Thousands | Sep. 30, 2021 | Dec. 31, 2020 |
Cash, Cash Equivalents, and Short-term Investments [Abstract] | ||
Cash and cash equivalents | $ 179,879 | $ 66,373 |
Cash | 97,000 | 8,400 |
Investments in money market funds | 82,900 | 58,000 |
Available for sale securities | $ 62,091 | $ 143,674 |
Cash, Cash Equivalents and Av_4
Cash, Cash Equivalents and Available for Sale Securities - Schedule of Available for Sale Securities (Details) - USD ($) $ in Thousands | Sep. 30, 2021 | Dec. 31, 2020 |
Debt Securities, Available-for-sale [Line Items] | ||
Amortized Cost | $ 62,102 | $ 143,671 |
Gross Unrealized Gains | 0 | 13 |
Gross Unrealized Losses | (11) | (10) |
Fair Value | 62,091 | 143,674 |
U.S. Treasury securities | ||
Debt Securities, Available-for-sale [Line Items] | ||
Amortized Cost | 8,999 | 54,225 |
Gross Unrealized Gains | 0 | 8 |
Gross Unrealized Losses | 0 | 0 |
Fair Value | 8,999 | 54,233 |
Corporate debt securities | ||
Debt Securities, Available-for-sale [Line Items] | ||
Amortized Cost | 43,614 | 79,282 |
Gross Unrealized Gains | 0 | 4 |
Gross Unrealized Losses | (8) | (10) |
Fair Value | 43,606 | 79,276 |
Asset-backed securities | ||
Debt Securities, Available-for-sale [Line Items] | ||
Amortized Cost | 9,489 | 10,164 |
Gross Unrealized Gains | 0 | 1 |
Gross Unrealized Losses | (3) | 0 |
Fair Value | $ 9,486 | $ 10,165 |
Cash, Cash Equivalents and Av_5
Cash, Cash Equivalents and Available for Sale Securities - Schedule of Accumulated Other Comprehensive Income (Details) - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2021 | Sep. 30, 2020 | Sep. 30, 2021 | Sep. 30, 2020 | |
Accumulated Other Comprehensive Income (Loss) [Line Items] | ||||
Balance at beginning of period | $ 139,573 | $ 238,041 | $ 191,563 | $ 117,203 |
Balance at end of period | 226,486 | 214,586 | 226,486 | 214,586 |
AOCI, Accumulated Gain (Loss), Debt Securities, Available-for-sale, Parent | ||||
Accumulated Other Comprehensive Income (Loss) [Line Items] | ||||
Balance at beginning of period | 3 | 0 | 3 | 0 |
Current period changes in fair value before reclassifications, net of tax | (14) | 1 | (14) | 1 |
Amounts reclassified from accumulated other comprehensive income, net of tax | 0 | 0 | 0 | 0 |
Total other comprehensive income (loss) | (14) | 1 | (14) | 1 |
Balance at end of period | $ (11) | $ 1 | $ (11) | $ 1 |
Share-Based Compensation - Narr
Share-Based Compensation - Narrative (Details) - USD ($) $ in Thousands | 1 Months Ended | 3 Months Ended | 9 Months Ended | 97 Months Ended | |||||
Sep. 30, 2021 | Feb. 28, 2021 | Mar. 31, 2020 | Sep. 30, 2021 | Sep. 30, 2020 | Sep. 30, 2021 | Sep. 30, 2020 | Sep. 30, 2021 | Oct. 31, 2019 | |
Stock options | |||||||||
Stock Option and Compensation Plans | |||||||||
Unrecognized share-based compensation costs for option awards, net of estimated forfeitures | $ 15,300 | $ 15,300 | $ 15,300 | $ 15,300 | |||||
Expected weighted average period to recognize share-based compensation costs (in years) | 3 years | ||||||||
Restricted stock units | |||||||||
Stock Option and Compensation Plans | |||||||||
Expected weighted average period to recognize share-based compensation costs (in years) | 2 years 8 months 12 days | ||||||||
Unrecognized compensation costs, net of estimated forfeitures | $ 7,500 | $ 7,500 | $ 7,500 | $ 7,500 | |||||
Employee stock purchase plan | |||||||||
Stock Option and Compensation Plans | |||||||||
Common stock issued (in shares) | 27,529 | 32,821 | 51,951 | 76,402 | |||||
Proceeds from stock plans | $ 146 | $ 147 | |||||||
2013 Plan | |||||||||
Stock Option and Compensation Plans | |||||||||
Increase in number of shares available under the Plan (in shares) | 2,542,000 | 13,081,000 | |||||||
Number of shares of common stock available for issuance under the Plan (in shares) | 3,031,000 | 3,031,000 | 3,031,000 | 3,031,000 | |||||
2013 Plan | Employee stock purchase plan | |||||||||
Stock Option and Compensation Plans | |||||||||
Number of shares available for future purchase (in shares) | 753,410 | 753,410 | 753,410 | 753,410 | |||||
2019 Inducement Plan | Stock options | |||||||||
Stock Option and Compensation Plans | |||||||||
Increase in number of shares available under the Plan (in shares) | 1,000,000 | 600,000 | 1,000,000 | ||||||
Number of shares of common stock available for issuance under the Plan (in shares) | 1,314,000 | 1,314,000 | 1,314,000 | 1,314,000 | |||||
Number of initial shares reserved for issuance under the Plan (in shares) | 1,000,000 |
Share-Based Compensation - Summ
Share-Based Compensation - Summary of Share-Based Compensation Expense by Award Type (Details) - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2021 | Sep. 30, 2020 | Sep. 30, 2021 | Sep. 30, 2020 | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Stock-based compensation expense | $ 2,626 | $ 1,847 | $ 6,997 | $ 6,044 |
Stock options | ||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Stock-based compensation expense | 1,485 | 1,089 | 3,997 | 3,556 |
Restricted stock units | ||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Stock-based compensation expense | 1,101 | 723 | 2,896 | 2,411 |
Employee stock purchase plan | ||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Stock-based compensation expense | $ 40 | $ 35 | $ 104 | $ 77 |
Share-Based Compensation - Su_2
Share-Based Compensation - Summary of Share-Based Compensation Expense by Financial Statement Line (Details) - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2021 | Sep. 30, 2020 | Sep. 30, 2021 | Sep. 30, 2020 | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Stock-based compensation expense | $ 2,626 | $ 1,847 | $ 6,997 | $ 6,044 |
Research and development | ||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Stock-based compensation expense | 1,476 | 882 | 4,138 | 2,986 |
General and administrative | ||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Stock-based compensation expense | $ 1,150 | $ 965 | $ 2,859 | $ 3,058 |
Share-Based Compensation - Su_3
Share-Based Compensation - Summary of Stock Option Activity (Details) - Stock options shares in Thousands | 9 Months Ended |
Sep. 30, 2021$ / sharesshares | |
Number of Shares Underlying Options | |
Outstanding, beginning balance (in shares) | shares | 8,928 |
Granted (in shares) | shares | 969 |
Exercised (in shares) | shares | (487) |
Forfeited (in shares) | shares | (321) |
Outstanding, ending balance (in shares) | shares | 9,089 |
Vested and exercisable, end of period (in shares) | shares | 5,154 |
Vested and expected to vest, end of period (in shares) | shares | 8,768 |
Weighted Average Exercise Price | |
Outstanding, beginning balance (in dollars per share) | $ / shares | $ 9.22 |
Granted (in dollars per share) | $ / shares | 7.84 |
Exercised (in dollars per share) | $ / shares | 2.89 |
Forfeited (in dollars per share) | $ / shares | 4.49 |
Outstanding, ending balance (in dollars per share) | $ / shares | 9.85 |
Vested and exercisable, end of period (in dollars per share) | $ / shares | 12.71 |
Vested and expected to vest, end of period (in dollars per share) | $ / shares | $ 9.99 |
Share-Based Compensation - Su_4
Share-Based Compensation - Summary of Outstanding RSU Awards Granted (Details) - Restricted stock units shares in Thousands | 9 Months Ended |
Sep. 30, 2021$ / sharesshares | |
Restricted Stock Units | |
Outstanding, beginning balance (in shares) | shares | 1,958 |
Awarded (in shares) | shares | 329 |
Vested (in shares) | shares | (181) |
Forfeited (in shares) | shares | (203) |
Outstanding, ending balance (in shares) | shares | 1,903 |
Outstanding, expected to vest (in shares) | shares | 1,750 |
Weighted Average Grant-Date Fair Value | |
Outstanding, beginning balance (in dollars per share) | $ / shares | $ 6.95 |
Awarded (in dollars per share) | $ / shares | 7.44 |
Vested (in dollars per share) | $ / shares | 4.32 |
Forfeited (in dollars per share) | $ / shares | 21.60 |
Outstanding, ending balance (in dollars per share) | $ / shares | 5.76 |
Outstanding, expected to vest (in dollars per share) | $ / shares | $ 5.76 |
Income Taxes - Narrative (Detai
Income Taxes - Narrative (Details) - USD ($) | 3 Months Ended | 9 Months Ended | ||||
Sep. 30, 2021 | Sep. 30, 2020 | Sep. 30, 2021 | Sep. 30, 2020 | Apr. 30, 2021 | Dec. 31, 2020 | |
Tax Credit Carryforward [Line Items] | ||||||
Income tax benefit | $ 0 | $ 0 | $ 0 | $ 3,695,000 | ||
Income tax receivable | $ 0 | $ 0 | $ 1,765,000 | |||
CARES Act | ||||||
Tax Credit Carryforward [Line Items] | ||||||
Income tax receivable | $ 1,800,000 |
Fair Value Measurements - Asset
Fair Value Measurements - Assets and Liabilities Measured at Fair Value on a Recurring Basis (Details) - USD ($) $ in Thousands | Sep. 30, 2021 | Dec. 31, 2020 |
Fair Value Measurements | ||
Transfer of assets from Level 1 to Level 2 of the fair value measurement hierarchy | $ 0 | |
Recurring basis | Quoted prices in active markets for identical assets (Level 1) | Money market funds | ||
Fair Value Measurements | ||
Investments | 82,872 | $ 58,042 |
Recurring basis | Quoted prices in active markets for identical assets (Level 1) | U.S. Treasury securities | ||
Fair Value Measurements | ||
Investments | 8,999 | 54,233 |
Recurring basis | Quoted prices in active markets for identical assets (Level 1) | Corporate debt securities | ||
Fair Value Measurements | ||
Investments | 0 | 0 |
Recurring basis | Quoted prices in active markets for identical assets (Level 1) | Asset-backed securities | ||
Fair Value Measurements | ||
Investments | 0 | 0 |
Recurring basis | Significant other observable inputs (Level 2) | Money market funds | ||
Fair Value Measurements | ||
Investments | 0 | 0 |
Recurring basis | Significant other observable inputs (Level 2) | U.S. Treasury securities | ||
Fair Value Measurements | ||
Investments | 0 | 0 |
Recurring basis | Significant other observable inputs (Level 2) | Corporate debt securities | ||
Fair Value Measurements | ||
Investments | 43,606 | 79,275 |
Recurring basis | Significant other observable inputs (Level 2) | Asset-backed securities | ||
Fair Value Measurements | ||
Investments | 9,486 | 10,166 |
Recurring basis | Significant unobservable inputs (Level 3) | Money market funds | ||
Fair Value Measurements | ||
Investments | 0 | 0 |
Recurring basis | Significant unobservable inputs (Level 3) | U.S. Treasury securities | ||
Fair Value Measurements | ||
Investments | 0 | 0 |
Recurring basis | Significant unobservable inputs (Level 3) | Corporate debt securities | ||
Fair Value Measurements | ||
Investments | 0 | 0 |
Recurring basis | Significant unobservable inputs (Level 3) | Asset-backed securities | ||
Fair Value Measurements | ||
Investments | $ 0 | $ 0 |
Commitments and Contingencies -
Commitments and Contingencies - Narrative (Details) $ in Thousands | Jun. 22, 2021 | Apr. 23, 2021 | Oct. 30, 2018USD ($)shares | May 31, 2019USD ($)licensedProductshares | Dec. 31, 2018USD ($) | Sep. 30, 2011USD ($) |
Commitments and contingencies | ||||||
Number of licensed products | licensedProduct | 1 | |||||
Litigation stay period (in days) | 60 days | 10 days | ||||
Inception 4 | ||||||
Commitments and contingencies | ||||||
Contingent consideration arrangements, maximum amount | $ 105,000 | |||||
Share price calculation base, period (in days) | 5 days | |||||
Contingent consideration arrangements, maximum amount (in shares) | shares | 7,200,000 | |||||
Contingent consideration arrangements, contingently issuable shares as a percentage of shares outstanding | 19.90% | |||||
GA Product | Inception 4 | ||||||
Commitments and contingencies | ||||||
Clinical and marketing approval milestones | $ 45,000 | |||||
Wet AMD Product | Inception 4 | ||||||
Commitments and contingencies | ||||||
Clinical and marketing approval milestones | $ 60,000 | |||||
Archemix | C5 Licensed Product | Achievement of specified clinical and regulatory milestones | Maximum | License Agreements | ||||||
Commitments and contingencies | ||||||
Amount to be paid on achievement of milestone | $ 50,500 | |||||
Archemix | C5 Licensed Product | First indication | Maximum | License Agreements | ||||||
Commitments and contingencies | ||||||
Amount to be paid on achievement of milestone | 24,500 | |||||
Archemix | C5 Licensed Product | Second and third indication | Maximum | License Agreements | ||||||
Commitments and contingencies | ||||||
Amount to be paid on achievement of milestone | 23,500 | |||||
Archemix | C5 Licensed Product | Sustained delivery applications | Maximum | License Agreements | ||||||
Commitments and contingencies | ||||||
Amount to be paid on achievement of milestone | 2,500 | |||||
Archemix | C5 Licensed Product | Achievement of specified commercial milestones | Maximum | License Agreements | ||||||
Commitments and contingencies | ||||||
Amount to be paid on achievement of milestone | $ 22,500 | |||||
University of Florida Research Foundation (UFRF) | Specified clinical, marketing approval and reimbursement approval milestones with respect to a licensed product | RHO-adRP License Agreement | ||||||
Commitments and contingencies | ||||||
Amount to be paid on achievement of milestone | $ 23,500 | |||||
University of Florida Research Foundation (UFRF) | Specified commercial sales milestones with respect to a licensed product | RHO-adRP License Agreement | ||||||
Commitments and contingencies | ||||||
Amount to be paid on achievement of milestone | $ 70,000 | |||||
University Of Florida Research Foundation (UFRF) And University Of Pennsylvania (Penn) | Primary Licensed Product | Specified clinical, marketing approval and reimbursement approval milestones with respect to a licensed product | Research and Development Expense | BEST1 License Agreement | ||||||
Commitments and contingencies | ||||||
Payments for license fees | $ 15,700 | |||||
University Of Florida Research Foundation (UFRF) And University Of Pennsylvania (Penn) | Primary Licensed Product | Specified commercial sales milestones with respect to a licensed product | Research and Development Expense | BEST1 License Agreement | ||||||
Commitments and contingencies | ||||||
Payments for license fees | 48,000 | |||||
University Of Florida Research Foundation (UFRF) And University Of Pennsylvania (Penn) | Other Licensed Product | Specified clinical, marketing approval and reimbursement approval milestones with respect to a licensed product | Research and Development Expense | BEST1 License Agreement | ||||||
Commitments and contingencies | ||||||
Payments for license fees | 3,100 | |||||
University Of Florida Research Foundation (UFRF) And University Of Pennsylvania (Penn) | Other Licensed Product | Specified commercial sales milestones with respect to a licensed product | Research and Development Expense | BEST1 License Agreement | ||||||
Commitments and contingencies | ||||||
Payments for license fees | 9,600 | |||||
University Of Massachusetts (UMass) | miniCEP290 | Achievement of specified clinical and regulatory milestones | License Agreements | Research and Development Expense | ||||||
Commitments and contingencies | ||||||
Payments for license fees | $ 14,750 | |||||
Shares issued for license fees (in shares) | shares | 75,000 | |||||
University Of Massachusetts (UMass) | miniCEP290 | Specified commercial sales milestones with respect to a licensed product | License Agreements | Research and Development Expense | ||||||
Commitments and contingencies | ||||||
Payments for license fees | $ 48,000 |
Subsequent Event - Narrative (D
Subsequent Event - Narrative (Details) - USD ($) $ / shares in Units, $ in Thousands | 1 Months Ended | |||
Oct. 31, 2021 | Jul. 31, 2021 | Jun. 30, 2020 | Jul. 30, 2021 | |
Subsequent Event [Line Items] | ||||
Payments of stock issuance costs | $ 7,400 | $ 10,100 | ||
Proceeds from issuance of common stock | $ 150,100 | |||
Public Stock Offering | ||||
Subsequent Event [Line Items] | ||||
Number of shares issued in transaction (in shares) | 13,397,500 | |||
Sale of stock price (in dollars per share) | $ 8.60 | |||
Proceeds from issuance of common stock | $ 107,800 | |||
Over-Allotment Option | ||||
Subsequent Event [Line Items] | ||||
Number of shares issued in transaction (in shares) | 1,747,500 | |||
Sale of stock price (in dollars per share) | $ 8.084 | |||
Subsequent Event | ||||
Subsequent Event [Line Items] | ||||
Payments of stock issuance costs | $ 10,800 | |||
Proceeds from issuance of common stock | $ 162,600 | |||
Subsequent Event | Public Stock Offering | ||||
Subsequent Event [Line Items] | ||||
Number of shares issued in transaction (in shares) | 10,350,000 | |||
Sale of stock price (in dollars per share) | $ 16.75 | |||
Subsequent Event | Over-Allotment Option | ||||
Subsequent Event [Line Items] | ||||
Number of shares issued in transaction (in shares) | 1,350,000 | |||
Sale of stock price (in dollars per share) | $ 15.745 |