0001410939-22-000090 | 10-Q | XBRL Viewer

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Cover - shares	6 Months Ended
Cover - shares	Jun. 30, 2022	Jul. 22, 2022
Cover [Abstract]
Document Type	10-Q
Document Quarterly Report	true
Document Period End Date	Jun. 30, 2022
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	8 Sylvan Way
Entity Address, Postal Zip Code	07054
Entity Address, City or Town	Parsippany,
Entity Address, State or Province	NJ
City Area Code	609
Local Phone Number	474-6755
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		118,077,814
Entity Central Index Key	0001410939
Amendment Flag	false
Current Fiscal Year End Date	--12-31
Document Fiscal Year Focus	2022
Document Fiscal Period Focus	Q2

Condensed Unaudited Consolidated Balance Sheets - USD ($) $ in Thousands	Jun. 30, 2022	Dec. 31, 2021
Current assets
Cash and cash equivalents	$ 141,113	$ 261,447
Available for sale securities	170,850	120,302
Prepaid expenses and other current assets	5,259	5,739
Total current assets	317,222	387,488
Property and equipment, net	514	348
Right-of-use asset, net	2,020	1,522
Total assets	319,756	389,358
Current liabilities
Accrued research and development expenses	16,577	14,403
Accounts payable and accrued expenses	8,178	12,856
Lease liability	1,724	952
Total current liabilities	26,479	28,211
Lease liability, non-current	331	619
Total liabilities	26,810	28,830
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, 116,874,198 and 115,277,012 shares issued and outstanding at June 30, 2022 and December 31, 2021, respectively	117	115
Additional paid-in capital	1,056,751	1,040,098
Accumulated deficit	(763,394)	(679,595)
Accumulated other comprehensive income	(528)	(90)
Total stockholders' equity	292,946	360,528
Total liabilities and stockholders' equity	$ 319,756	$ 389,358

Condensed Unaudited Consolidated Balance Sheets (Parenthetical) - $ / shares	Jun. 30, 2022	Dec. 31, 2021
Statement of Financial Position [Abstract]
Preferred stock, par value (in usd 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 usd per share)	$ 0.001	$ 0.001
Common stock, shares authorized (in shares)	200,000,000	200,000,000
Common stock, shares issued (in shares)	116,874,198	115,277,012
Common stock, shares outstanding (in shares)	116,874,198	115,277,012

Condensed Unaudited Consolidated Statements of Operations and Comprehensive Loss - USD ($) shares in Thousands, $ in Thousands	3 Months Ended		6 Months Ended
	Jun. 30, 2022	Jun. 30, 2021	Jun. 30, 2022	Jun. 30, 2021
Operating expenses:
Research and development	$ 33,647	$ 23,488	$ 56,204	$ 42,037
General and administrative	16,106	6,718	28,219	15,040
Total operating expenses	49,753	30,206	84,423	57,077
Loss from operations	(49,753)	(30,206)	(84,423)	(57,077)
Interest income	482	65	615	142
Other expense, net	8	(2)	9	(3)
Loss before income tax benefit	(49,263)	(30,143)	(83,799)	(56,938)
Income tax benefit	0	0	0	0
Net loss	(49,263)	(30,143)	(83,799)	(56,938)
Comprehensive loss	$ (49,397)	$ (30,142)	$ (84,237)	$ (56,938)
Net loss per common share:
Basic (in usd per share)	$ (0.41)	$ (0.32)	$ (0.70)	$ (0.61)
Diluted (in usd per share)	$ (0.41)	$ (0.32)	$ (0.70)	$ (0.61)
Weighted average common shares outstanding:
Basic (in shares)	119,687	93,409	119,223	93,382
Diluted (in shares)	119,687	93,409	119,223	93,382

Condensed Unaudited Consolidated Statements of Stockholders' Equity - USD ($) shares in Thousands, $ in Thousands	Total	Preferred Stock	Common Stock	Additional paid-in capital	Accumulated Deficit	Accumulated Other Comprehensive Income (Loss)
Balance at beginning of period (in shares) at Dec. 31, 2020		0	90,121
Beginning balance 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
Ending balance 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
Beginning balance at Dec. 31, 2020	191,563	$ 0	$ 90	756,543	(565,073)	3
Increase (Decrease) in Stockholders' Equity
Net loss	(56,938)
Balance at end of period (in shares) at Jun. 30, 2021		0	90,387
Ending balance at Jun. 30, 2021	139,573	$ 0	$ 90	761,491	(622,011)	3
Balance at beginning of period (in shares) at Mar. 31, 2021		0	90,170
Beginning balance 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
Ending balance at Jun. 30, 2021	139,573	$ 0	$ 90	761,491	(622,011)	3
Balance at beginning of period (in shares) at Dec. 31, 2021		0	115,277
Beginning balance at Dec. 31, 2021	360,528	$ 0	$ 115	1,040,098	(679,595)	(90)
Increase (Decrease) in Stockholders' Equity
Issuance of common stock under employee stock compensation plan (in shares)			697
Issuance of common stock under employee stock compensation plans	2,080		$ 1	2,079
Share-based compensation	5,386			5,386
Net loss	(34,536)				(34,536)
Unrealized loss on available for sale securities, net of tax	(304)					(304)
Balance at end of period (in shares) at Mar. 31, 2022		0	115,974
Ending balance at Mar. 31, 2022	333,154	$ 0	$ 116	1,047,563	(714,131)	(394)
Balance at beginning of period (in shares) at Dec. 31, 2021		0	115,277
Beginning balance at Dec. 31, 2021	360,528	$ 0	$ 115	1,040,098	(679,595)	(90)
Increase (Decrease) in Stockholders' Equity
Net loss	(83,799)
Balance at end of period (in shares) at Jun. 30, 2022		0	116,874
Ending balance at Jun. 30, 2022	292,946	$ 0	$ 117	1,056,751	(763,394)	(528)
Balance at beginning of period (in shares) at Mar. 31, 2022		0	115,974
Beginning balance at Mar. 31, 2022	333,154	$ 0	$ 116	1,047,563	(714,131)	(394)
Increase (Decrease) in Stockholders' Equity
Issuance of common stock under employee stock compensation plan (in shares)			900
Issuance of common stock under employee stock compensation plans	2,801		$ 1	2,800
Share-based compensation	6,388			6,388
Net loss	(49,263)				(49,263)
Unrealized loss on available for sale securities, net of tax	(134)					(134)
Balance at end of period (in shares) at Jun. 30, 2022		0	116,874
Ending balance at Jun. 30, 2022	$ 292,946	$ 0	$ 117	$ 1,056,751	$ (763,394)	$ (528)

- Definition

The portion of profit or loss for the period, net of income taxes, which is attributable to the parent.

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- Definition

Total of all stockholders' equity (deficit) items, net of receivables from officers, directors, owners, and affiliates of the entity which are attributable to the parent. The amount of the economic entity's stockholders' equity attributable to the parent excludes the amount of stockholders' equity which is allocable to that ownership interest in subsidiary equity which is not attributable to the parent (noncontrolling interest, minority interest). This excludes temporary equity and is sometimes called permanent equity.

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+ Details

Condensed Unaudited Consolidated Statements of Cash Flows - USD ($) $ in Thousands	6 Months Ended
	Jun. 30, 2022	Jun. 30, 2021
Operating Activities
Net loss	$ (83,799)	$ (56,938)
Adjustments to reconcile net loss to net cash used in operating activities
Depreciation and other expense	45	18
Amortization of premium and discounts on investment securities	540	742
Share-based compensation	11,774	4,371
Changes in operating assets and liabilities:
Income tax receivable	0	1,765
Prepaid expense and other assets	480	990
Accrued interest receivable	57	92
Accrued research and development expenses	2,174	3,128
Accounts payable and accrued expenses	(4,678)	(4,149)
Change in working capital	(14)	73
Net cash used in operating activities	(73,421)	(49,908)
Investing Activities
Purchase of marketable securities	(96,498)	(39,314)
Purchase of property and equipment	(211)	0
Maturities of marketable securities	44,915	72,635
Net cash used in investing activities	(51,794)	33,321
Financing Activities
Proceeds from employee stock plan purchases	4,881	577
Net cash provided by financing activities	4,881	577
Net increase (decrease) in cash and cash equivalents	(120,334)	(16,010)
Cash and cash equivalents
Beginning of period	261,447	66,373
End of period	141,113	50,363
Supplemental disclosure of cash paid
Income tax refunds received	0	1,765
Supplemental disclosures of non-cash information related to investing activities
Operating right-of-use assets obtained in exchange for lease obligations	$ 953	$ 2,086

Business	6 Months Ended
Business	Jun. 30, 2022
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 earlier 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 in September 2022. The Company continues to pursue various lifecycle management programs for Zimura. In June 2022, the Company entered into a license agreement (the “DelSiTech License Agreement”) with DelSiTech Ltd. (“DelSiTech”) for a worldwide, exclusive license under specified patent rights and know-how to develop and commercialize new formulations of Zimura using DelSiTech’s silica-based sustained release technology for treating diseases of the human eye. 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 for IC-500 to the FDA in mid-2023. 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. As the Company focuses its efforts on and prioritizes the development and potential commercialization of Zimura, it has been considering its development options for IC-100 and IC-200, which the Company has been developing for RHO-adRP and BEST1-related IRDs, respectively. It is currently planning to seek a collaborator for the future development and potential commercialization of these product candidates.

Summary of Significant Accounting Policies

6 Months Ended

Jun. 30, 2022

Accounting Policies [Abstract]

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, 2021 filed with the Securities and Exchange Commission (“SEC”) on February 24, 2022.

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 six months ended June 30, 2022 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 Condensed Unaudited 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 Condensed Unaudited Consolidated Balance Sheets for cash and cash equivalents are valued at cost, which approximates their fair value.

Available for Sale Securities

The Company considers debt 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.

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 Condensed Unaudited 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. ROU 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 Condensed Unaudited 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 to ten years, using the straight-line method. Amortization of leasehold improvements is recorded over the shorter of the lease term or estimated useful life of the related asset.

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 three and six month periods ended June 30, 2022 and 2021:


	Three Months Ended June 30,		Six Months Ended June 30,
	2022	2021	2022	2021
Expected common stock price volatility	76%	113%	83%	113%
Risk-free interest rate	2.53%-2.99%	0.89%-0.94%	1.38%-2.99%	0.31%-0.96%
Expected term of options (years)	4.8	5.2	4.9	5.1
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.

Net Loss Per Common Share

6 Months Ended

Jun. 30, 2022

Earnings Per Share [Abstract]

Net Loss Per Common Share

Net Loss Per Common ShareBasic and diluted net loss per common share is determined by dividing net loss by the weighted average common shares and pre-funded warrants outstanding during the period. Basic and diluted shares outstanding includes the weighted average effect of the Company's outstanding pre-funded warrants as the exercise of such pre-funded warrants requires nominal consideration to be given for the delivery of the corresponding shares of common stock. As of June 30, 2022 and 2021, 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 June 30,				Six Months Ended June 30,
	2022		2021		2022		2021
Basic and diluted net loss per common share calculation:
Net loss	$	(49,263)	$	(30,143)	$	(83,799)	$	(56,938)
Weighted average common shares outstanding - basic and dilutive	119,687		93,409		119,223		93,382
Net loss per share of common stock - basic and diluted	$	(0.41)	$	(0.32)	$	(0.70)	$	(0.61)

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 June 30,		Six Months Ended June 30,
	2022	2021	2022	2021
Stock options outstanding	10,546	9,233	10,546	9,233
Restricted stock units	2,215	1,953	2,215	1,953
Total	12,761	11,186	12,761	11,186

Licensing and Commercialization Agreements	6 Months Ended
Licensing and Commercialization Agreements	Jun. 30, 2022
Organization, Consolidation and Presentation of Financial Statements [Abstract]
Licensing and Commercialization Agreements	Licensing and Commercialization Agreements On June 30, 2022, the Company entered into the DelSiTech License Agreement with DelSiTech. Under the DelSiTech License Agreement, DelSiTech granted the Company a worldwide, exclusive license under specified patent rights and know-how to develop, have developed, make, have made, use, offer to sell, sell, have sold, otherwise commercialize, export and import Zimura using DelSiTech’s silica-based sustained release technology for the treatment of diseases of the eye in humans (the “Licensed Product”). The Company may grant sublicenses of the licensed patent rights and know-how without DelSiTech’s consent. The Company has agreed to pay DelSiTech, within 60 days after execution of the DelSiTech License Agreement, a €1.25 million upfront license fee, which was recognized as a research and development expense during the three months ended June 30, 2022. Under the DelSiTech License Agreement, the Company is further obligated to pay DelSiTech, up to an aggregate of €35.0 million, if the Company achieves specified clinical and development milestones with respect to the Licensed Product. In addition, the Company is also obligated to pay DelSiTech up to an aggregate of €60.0 million if the Company achieves specified commercial sales milestones with respect to worldwide net sales of the Licensed Product. Due to the uncertainty of the achievement of these milestones, the Company will account for any additional payments if and when such milestones are met. The Company is also obligated to pay DelSiTech royalties at a low single-digit percentage of net sales of the Licensed Product. The royalties payable by the Company are subject to reduction under specified circumstances. The Company’s obligation to pay royalties under the DelSiTech License Agreement will continue on a country-by-country basis until the later of: (a) the expiration of the last-to-expire licensed patent rights covering the Licensed Product in the country of sale, or (b) expiration of all regulatory exclusivity for the Licensed Product in the country of sale. Future milestones and royalties will be recognized in their entirety when achieved. Unless earlier terminated by the Company or DelSiTech, the DelSiTech License Agreement will expire on a country-by-country basis upon the expiration of the Company’s obligation to pay royalties to DelSiTech on net sales of the Licensed Product. Upon expiration of the DelSiTech License Agreement, the licenses granted by DelSiTech to the Company will become fully paid up and irrevocable. The Company may terminate the agreement at any time for any reason upon 60 days’ prior written notice to DelSiTech. Either party may also terminate the DelSiTech License Agreement if the other party materially breaches the DelSiTech License Agreement and does not cure such breach within a specified cure period. Following any termination of the DelSiTech License Agreement prior to expiration of the term of the DelSiTech License Agreement, all rights to the licensed patent rights and know-how that DelSiTech granted to the Company will revert to DelSiTech, subject to the Company’s right to sell off any Licensed Product in the Company’s inventory as of the effectiveness of such termination.

Cash, Cash Equivalents and Available for Sale Securities

6 Months Ended

Jun. 30, 2022

Cash, Cash Equivalents, and Short-Term Investments [Abstract]

Cash, Cash Equivalents and Available for Sale Securities

Cash, Cash Equivalents and Available for Sale SecuritiesThe 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 June 30, 2022 and December 31, 2021, the Company had cash and cash equivalents of

approximately $141.1 million and $261.4 million, respectively. Cash and cash equivalents included cash of $9.7 million at June 30, 2022 and $9.9 million at December 31, 2021. Cash and cash equivalents at June 30, 2022 and December 31, 2021 included $131.4 million and $251.5 million, respectively, of investments in money market funds.

The Company considers debt securities with original maturities of greater than 90 days at the date of purchase to be available for sale securities. As of June 30, 2022 and December 31, 2021, the Company held available for sale securities of $170.9 million and $120.3 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 June 30, 2022. 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 June 30, 2022 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.

Available for sale securities, including carrying value and estimated fair values, are summarized as follows:


	As of June 30, 2022
	Amortized Cost			Gross Unrealized Gains		Gross Unrealized Losses		Fair Value
U.S. Treasury securities	$	48,152		$	—	$	(290)	$	47,862
Corporate debt securities	103,151		103,151	1		(178)		102,974
Asset-backed securities	20,074			—		(60)		20,014
Supranational securities	—			—		—		—
Total	$	171,377		$	1	$	(528)	$	170,850


	As of December 31, 2021
	Amortized Cost		Gross Unrealized Gains		Gross Unrealized Losses		Fair Value
U.S. Treasury securities	$	18,201	$	—	$	(16)	$	18,185
Corporate debt securities	82,138		—		(57)		82,081
Asset-backed securities	16,009		—		(14)		15,995
Supranational securities	4,044		—		(3)		4,041
Total	$	120,392	$	—	$	(90)	$	120,302

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 months ended June 30, 2022 and 2021, respectively, were as follows:


	Three months ended June 30,				Six Months Ended June 30, 2022
	2022		2021		2022		2021
Beginning balance	$	(394)	$	2	$	(90)	$	3
Current period changes in fair value before reclassifications, net of tax	(134)		1		(438)		—
Amounts reclassified from accumulated other comprehensive income, net of tax	—		—		—		—
Total other comprehensive loss	$	(134)	$	1	(438)		—
Ending balance	$	(528)	$	3	$	(528)	$	3

Fair Value Measurements

6 Months Ended

Jun. 30, 2022

Fair Value Disclosures [Abstract]

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 June 30, 2022:


	Fair Value Measurement Using
	Quoted prices in active markets for identical assets (Level 1)		Significant other observable inputs (Level 2)		Significant unobservable inputs (Level 3)
Assets
Investments in money market funds*	$	131,399	$	—	$	—
Investments in U.S. Treasury securities	$	47,862	$	—	$	—
Investments in corporate debt securities	$	—	$	102,974	$	—
Investments in asset-backed securities	$	—	20,014		$	—
Investments in supranational securities	$	—	—		$	—

*Investments in money market funds are reflected in cash and cash equivalents in the accompanying Condensed Unaudited Consolidated Balance Sheets.


	Fair Value Measurement Using
	Quoted prices in active markets for identical assets (Level 1)		Significant other observable inputs (Level 2)		Significant unobservable inputs (Level 3)
Assets
Investments in money market funds*	$	251,488	$	—	$	—

Investments in U.S. Treasury securities	$	18,185	$	—	$	—
Investments in corporate debt securities	$	—	$	82,081	$	—
Investments in asset-backed securities	$	—	$	15,995	$	—
Investments in supranational securities	$	—	$	4,041	$	—

*Investments in money market funds are reflected in cash and cash equivalents in the accompanying Condensed Unaudited Consolidated Balance Sheets.

No transfer of assets between Level 1 and Level 2 of the fair value measurement hierarchy occurred during the three and six months ended June 30, 2022.

Share-Based Compensation

6 Months Ended

Jun. 30, 2022

Share-Based Payment Arrangement [Abstract]

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 15,624,000 additional shares to the 2013 Plan, including for 2022, an increase of approximately 2,542,000 shares. As of June 30, 2022, the Company had approximately 3,507,000 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, September 2021, December 2021 and May 2022, the Company's board of directors further amended the 2019 Inducement Plan to reserve an additional 600,000 shares, an additional 1,000,000 shares, an additional 1,000,000 shares and an additional 1,000,000 shares, respectively, of its common stock for issuance under the plan. As of June 30, 2022, the Company had approximately 1,341,000 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 June 30,				Six Months Ended June 30,
	2022		2021		2022		2021
Stock options	$	3,750	$	1,042	$	7,074	$	2,511
Restricted stock units	2,571		1,003		4,576		1,795
Employee stock purchase plan	67		34		124		65
Total	$	6,388	$	2,079	$	11,774	$	4,371

The Company allocated stock-based compensation expense in the Company’s Consolidated Statements of Operations and Comprehensive Loss as follows:


	Three Months Ended June 30,				Six Months Ended June 30,
	2022		2021		2022		2021
Research and development	$	2,766	$	1,319	$	5,427	$	2,662
General and administrative	3,622		760		6,347		1,709
Total	$	6,388	$	2,079	$	11,774	$	4,371

Stock Options

A summary of the stock option activity, weighted average exercise prices, options outstanding, exercisable and expected to vest as of June 30, 2022 is as follows (in thousands except weighted average exercise price):


	Number of Shares Underlying Options	Weighted Average Exercise Price
Outstanding, December 31, 2021	10,861	$	10.94
Granted	1,222	$	14.79
Exercised	(1,400)	$	3.31
Forfeited	(138)	$	14.78


Outstanding, June 30, 2022	10,545	$	12.35
Vested and exercisable, June 30, 2022	4,811	$	13.21
Vested and expected to vest, June 30, 2022	10,087	$	12.38

As of June 30, 2022, there were approximately $41.3 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 3.1 years.

RSUs

The following table presents a summary of the Company's outstanding RSU awards granted as of June 30, 2022 (in thousands except weighted average grant-date fair value):


	Restricted Stock Units	Weighted Average Grant-Date Fair Value
Outstanding, December 31, 2021	2,246	$	9.82
Awarded	192	$	12.22
Vested	(182)	$	6.99
Forfeited	(41)	$	10.80
Outstanding, June 30, 2022	2,215	$	10.24
Outstanding, expected to vest	2,038	$	10.24

As of June 30, 2022, there were approximately $16.3 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 2.8 years.

ESPP

As of June 30, 2022, there were 738,315 shares available for future purchases under the ESPP. There were no shares issued under the ESPP during the three months ended June 30, 2022 and 2021, respectively. There were 15,095 and 24,422 shares of common stock issued under the ESPP during the six months ended June 30, 2022 and 2021, respectively. Cash proceeds from ESPP purchases were $184 thousand and $121 thousand during the six months ended June 30, 2022 and 2021, respectively.

Commitments and Contingencies	6 Months Ended
Commitments and Contingencies	Jun. 30, 2022
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. Zimura Sustained Release Delivery Technology - DelSiTech Under the DelSiTech License Agreement with DelSiTech, the Company is obligated to make payments up to an aggregate of €35.0 million, if the Company achieves specified clinical and development milestones with respect to a Licensed Product. In addition, the Company is also obligated to pay DelSiTech up to an aggregate of €60.0 million if the Company achieves specified commercial sales milestones with respect to worldwide net sales of the Licensed Product. The Company is also obligated to pay DelSiTech royalties at a low single-digit percentage of net sales of the Licensed Product. The royalties payable by the Company are subject to reduction under specified circumstances. 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 Equity holders 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 equity holders 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-trading day period preceding the achievement of the relevant milestone, unless and until the issuance of such shares would, together with all other shares issued in connection with the acquisition, exceed an overall maximum limit of approximately 7.2 million shares, which is equal to 19.9% of the number of issued and outstanding shares of the Company's common stock as of the close of business on the business day prior to the closing date of the Inception 4 acquisition, and will be payable in cash thereafter. The Inception 4 Merger Agreement also includes customary indemnification obligations to the former equity holders of Inception 4, including for breaches of the representations and warranties, covenants and agreements of the Company and its subsidiaries (other than Inception 4) in the Inception 4 Merger Agreement. Employment Contracts The Company also has letter agreements with certain employees that require the funding of a specific level of payments if certain events, such as a termination of employment in connection with a change in control or termination of employment by the employee for good reason or by the Company without cause, occur. Contract Service Providers In addition, in the course of normal business operations, the Company has agreements with contract service providers to assist in the performance of the Company’s research and development and manufacturing activities. Expenditures to CROs and CDMOs represent significant costs in preclinical and clinical development. Subject to required notice periods and the Company’s obligations under binding purchase orders and any cancellation fees that the Company may be obligated to pay, the Company can elect to discontinue the work under these agreements at any time. Legal Proceedings On January 11, 2017, a putative class action lawsuit was filed against the Company and certain of its current and former executive officers in the United States District Court for the Southern District of New York, captioned Frank Micholle v. IVERIC bio, Inc., et al., No. 1:17-cv-00210. On March 9, 2017, a related putative class action lawsuit was filed against the Company and the same group of its current and former executive officers in the United States District Court for the Southern District of New York, captioned Wasson v. IVERIC bio, Inc., et al., No. 1:17-cv-01758. These cases were consolidated on March 13, 2018. On June 4, 2018, the lead plaintiff filed a consolidated amended complaint (the “CAC”). The CAC purports to be brought on behalf of shareholders who purchased the Company’s common stock between March 2, 2015 and December 12, 2016. The CAC generally alleges that the Company and certain of its officers violated Sections 10(b) and/or 20(a) of the Securities Exchange Act of 1934 and Rule 10b-5 promulgated thereunder by making allegedly false and/or misleading statements concerning the results of the Company’s Phase 2b trial and the prospects of the Company’s Phase 3 trials for Fovista in combination with anti-VEGF agents for the treatment of wet AMD. The CAC seeks unspecified damages, attorneys’ fees, and other costs. The Company and individual defendants filed a motion to dismiss the CAC on July 27, 2018. On September 18, 2019, the court issued an order dismissing some, but not all, of the allegations in the CAC. On November 18, 2019, the Company and the individual defendants filed an answer to the complaint. On June 12, 2020, the lead plaintiff filed a motion for class certification. On August 11, 2020, the defendants filed a notice of non-opposition to lead plaintiff's motion for class certification. 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 and submitted the agreement to the court for approval. Under the terms of the settlement agreement, the Company agreed to pay $29 million, which includes the attorneys' fees and costs and expenses for the plaintiffs' counsel. On March 17, 2022, the court provided a preliminary approval of the settlement. In April 2022, the Company’s insurance carriers paid the full amount of the settlement directly to the plaintiffs’ escrow account. A hearing to determine whether the settlement should be granted final approval is scheduled for September 8, 2022. The Company does not expect this settlement, if granted final approval in its current form, 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 2022. On October 18, 2021, the parties notified the court overseeing the Pacheco matter that they had reached an agreement in principle to settle the action. On January 27, 2022, the parties executed a settlement agreement, which has been submitted to the court for approval. 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 2022. On January 27, 2022, the parties executed a settlement agreement. 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.

Operating Leases

6 Months Ended

Jun. 30, 2022

Leases [Abstract]

Operating Leases

The Company leases office space located in Cranbury, New Jersey and Parsippany, New Jersey under non-cancelable operating lease arrangements. In May 2022, the Company amended its Cranbury office space lease to extend the lease period by one year through the end of February 2024.

In addition, in June 2022 the Company amended its Parsippany office lease to include an additional portion of the premises consisting of approximately 34,836 square feet of the third floor of the building. The Parsippany lease expires at the end of August 2023.

As of June 30, 2022, the Company recognized additional right-of-use assets and lease liabilities of approximately $1.0 million, which represents the present value of its remaining lease payments using a weighted average estimated incremental borrowing rate of 8%.

For the three and six months ended June 30, 2022, lease expense was $0.2 million and $0.5 million, respectively. Cash paid from operating cash flows for amounts included in the measurement of lease liabilities was $0.2 million and $0.5 million, respectively, for the three and six months ended June 30, 2022. At June 30, 2022, the Company's operating leases had a weighted average remaining lease term of 1.2 years.

The following presents the maturity of the Company’s operating lease liabilities as of June 30, 2022:


	June 30, 2022
Remainder of 2022	$	902
2023	1,216
2024	11
Total remaining obligation	$	2,129
Less imputed interest	(73)
Present value of lease liabilities	$	2,056

Subsequent Events	6 Months Ended
Subsequent Events	Jun. 30, 2022
Subsequent Events [Abstract]
Subsequent Events	Subsequent Events On July 26, 2022 (the “Closing Date”), the Company and certain of its subsidiaries (the “Subsidiary Borrowers”) entered into a Loan and Security Agreement (the “Loan Agreement”) with Hercules Capital, Inc. (“Hercules”), in its capacity as administrative agent and collateral agent (in such capacity, the “Agent”) and as a lender, Silicon Valley Bank (“SVB”) and certain other financial institutions that from time to time become parties to the Loan Agreement as lenders (collectively, the “Lenders”). The Loan Agreement provides for term loans in an aggregate principal amount of up to $250.0 million under multiple tranches (the “2022 Term Loan Facility”), available as follows: (i) a term loan advance in the amount of $50.0 million on the Closing Date; (ii) subject to the Company’s announcement that the GATHER2 trial evaluating Zimura in GA has achieved its protocol-specified primary endpoint and the Company has a sufficient clinical data package to support the submission of an NDA to the FDA for Zimura in GA (“Milestone 1”), a second tranche consisting of term loan advances in the aggregate principal amount of $50.0 million available at the Company’s option beginning on the date that Milestone 1 is achieved through December 15, 2022; (iii) subject to the Company’s submission of an NDA to the FDA for Zimura in GA and the FDA accepting such NDA for review (“Milestone 2”), a third tranche consisting of term loan advances in the aggregate principal amount of $25.0 million, available at the Company’s option beginning on the date that Milestone 2 is achieved through September 30, 2023; (iv) subject to FDA approval of Zimura in GA with a label generally consistent with that sought in the Company’s NDA (“Milestone 3”), a fourth tranche consisting of term loan advances in the aggregate principal amount of $75.0 million, available at the Company’s option beginning on the date that Milestone 3 is achieved and continuing through the earlier of (x) September 30, 2024 and (y) the date that is ninety (90) days after the date that Milestone 3 is achieved; and (v) subject to approval by the Lenders’ investment committee in its discretion, a fifth tranche of additional term loans in an aggregate principal amount of up to $50.0 million, available on or before the Amortization Date (as defined below). With the exception of the first $50.0 million tranche available on the Closing Date, each of the tranches may be drawn down in $5.0 million increments at the Company's election. The Company has agreed to use the proceeds of the 2022 Term Loan Facility for working capital and general corporate purposes. Notwithstanding limitations and restrictions imposed by covenants in the Loan Agreement, the Company is permitted to engage in certain specified transactions. For example, the terms of the Loan Agreement provide that the Company may issue convertible notes in an aggregate principal amount of not more than $400.0 million, provided that such notes are unsecured, have a maturity date no earlier than six months following the Maturity Date (as defined below), and meet certain other conditions. The Loan Agreement also provides that the Company may enter into royalty interest financing transactions that are subordinated to the 2022 Term Loan Facility, have a maturity date no earlier than six months following the Maturity Date, and meet certain other conditions. Following the achievement of Milestone 3, the Loan Agreement also provides for a possible additional revolving credit facility of up to $50.0 million, which will be formula-based and backed by the Company’s accounts receivables. This potential revolving credit facility is not an existing facility under the Loan Agreement, is not committed, and is subject to agreement among the Company and the Lenders. The Company may enter into non-exclusive and certain specified exclusive licensing arrangements with respect to core intellectual property and non-exclusive and exclusive licensing arrangements or otherwise transfer non-core intellectual property without the consent of the Lenders. The Company may also enter into certain permitted acquisitions, subject to a limit on total cash consideration for acquisitions consummated during specified periods. Additionally, the Company must provide the Lenders the opportunity to invest up to $10.0 million in any equity financing, subject to certain exclusions, that is broadly marketed to multiple investors and in which the Company receives net cash proceeds of $75.0 million or more in any one or series of related financings (or in the case of any such equity financing that is a registered offering, use its commercially reasonable efforts to provide such opportunity to the Lenders). The 2022 Term Loan Facility will mature on August 1, 2027 (the “Maturity Date”). The outstanding principal balance of the 2022 Term Loan Facility bears interest at a floating interest rate per annum equal to the greater of either (i) (x) the lesser of the Wall Street Journal prime rate and 6.25% plus (y) 4.00% or (ii) 8.75%. The per annum Interest rate is capped at 10.25%. Accrued interest is payable monthly following the funding of each term loan. The Company may make payments of interest only, without any loan amortization payments, for a period of forty-two (42) months following the Closing Date, which period may be extended to the Maturity Date if (i) Milestone 3 has been achieved and (ii) no default or event of default exists under the Loan Agreement. At the end of the interest only period (the “Amortization Date”), the Company is required to begin repayment of the outstanding principal of the 2022 Term Loan Facility in equal monthly installments. As collateral for the obligations under the 2022 Term Loan Facility, the Company has granted to Agent for the benefit of the Lenders a senior security interest in substantially all of its and each Subsidiary Borrower’s property, inclusive of intellectual property, with certain limited exceptions set forth in the Loan Agreement. The Loan Agreement contains customary closing and commitment fees, prepayment fees and provisions, events of default and representations, warranties and affirmative and negative covenants, including a financial covenant requiring the Company to maintain certain levels of cash in accounts subject to a control agreement in favor of the Agent (the “Qualified Cash”) during the period commencing on May 15, 2023 through August 14, 2024. Commencing on August 15, 2024, the Company will also be required to maintain a certain minimum amount of trailing six-month net product revenue from the sale of Zimura, tested on a quarterly basis. The revenue covenant will be waived at any time at which the Company (x) (i) maintains a market capitalization in excess of $600.0 million and (ii) maintains Qualified Cash in an amount greater than or equal to fifty percent (50%) of the outstanding 2022 Term Loan Facility at such time or (y) maintains Qualified Cash in an amount greater than or equal to ninety percent (90%) of the outstanding 2022 Term Loan Facility at such time. Upon the occurrence of an event of default, including a material adverse effect, subject to certain exceptions, on the business, operations, properties, assets or financial condition of the Company and the Subsidiary Borrowers taken as a whole, and subject to any specified cure periods, all amounts owed by the Company may be declared immediately due and payable by the Lenders. As of the Closing Date, the Company was in compliance with all applicable covenants under the Loan Agreement. In addition, the Company is required to make a final payment fee (the “End of Term Charge”) upon the earlier of (i) the Maturity Date or (ii) the date the Company prepays, in full or in part, the outstanding principal balance of the 2022 Term Loan Facility. The End of Term Charge is 4.25% of the aggregate original principal amount of the term loans repaid or prepaid under the Loan Agreement. The Company may, at its option, prepay the term loans in full or in part, subject to a prepayment penalty equal to (i) 2.0% of the principal amount prepaid if the prepayment occurs prior to the first anniversary of the Closing Date, (ii) 1.5% of the principal amount prepaid if the prepayment occurs on or after the first anniversary and prior to the second anniversary of the Closing Date, and (iii) 0.75% of the principal amount prepaid if the prepayment occurs on or after the second anniversary and prior to the third anniversary of the Closing Date.

Summary of Significant Accounting Policies (Policies)	6 Months Ended
Summary of Significant Accounting Policies (Policies)	Jun. 30, 2022
Accounting Policies [Abstract]
Basis of Presentation	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 six months ended June 30, 2022 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. The Company views its operations and manages its business in one operating and reportable segment.
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 Condensed Unaudited 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 Condensed Unaudited Consolidated 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 debt 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.
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 Condensed Unaudited 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. ROU 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 Condensed Unaudited 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 to ten years, using the straight-line method. Amortization of leasehold improvements is recorded over the shorter of the lease term or estimated useful life of the related asset.
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.
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 Accounting Policies (Tables)

6 Months Ended

Jun. 30, 2022

Accounting Policies [Abstract]

Schedule of weighted-average assumptions used to estimate grant date fair value of stock options


	Three Months Ended June 30,		Six Months Ended June 30,
	2022	2021	2022	2021
Expected common stock price volatility	76%	113%	83%	113%
Risk-free interest rate	2.53%-2.99%	0.89%-0.94%	1.38%-2.99%	0.31%-0.96%
Expected term of options (years)	4.8	5.2	4.9	5.1
Expected dividend yield	—	—	—	—

Net Loss Per Common Share - (Tables)

6 Months Ended

Jun. 30, 2022

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 June 30,				Six Months Ended June 30,
	2022		2021		2022		2021
Basic and diluted net loss per common share calculation:
Net loss	$	(49,263)	$	(30,143)	$	(83,799)	$	(56,938)
Weighted average common shares outstanding - basic and dilutive	119,687		93,409		119,223		93,382
Net loss per share of common stock - basic and diluted	$	(0.41)	$	(0.32)	$	(0.70)	$	(0.61)

Schedule of potentially dilutive securities that have been excluded from the computations of diluted weighted average common shares outstanding


	Three Months Ended June 30,		Six Months Ended June 30,
	2022	2021	2022	2021
Stock options outstanding	10,546	9,233	10,546	9,233
Restricted stock units	2,215	1,953	2,215	1,953
Total	12,761	11,186	12,761	11,186

Cash, Cash Equivalents and Available for Sale Securities (Tables)

6 Months Ended

Jun. 30, 2022

Cash, Cash Equivalents, and Short-Term Investments [Abstract]

Schedule of for sale securities

Available for sale securities, including carrying value and estimated fair values, are summarized as follows:


	As of June 30, 2022
	Amortized Cost			Gross Unrealized Gains		Gross Unrealized Losses		Fair Value
U.S. Treasury securities	$	48,152		$	—	$	(290)	$	47,862
Corporate debt securities	103,151		103,151	1		(178)		102,974
Asset-backed securities	20,074			—		(60)		20,014
Supranational securities	—			—		—		—
Total	$	171,377		$	1	$	(528)	$	170,850


	As of December 31, 2021
	Amortized Cost		Gross Unrealized Gains		Gross Unrealized Losses		Fair Value
U.S. Treasury securities	$	18,201	$	—	$	(16)	$	18,185
Corporate debt securities	82,138		—		(57)		82,081
Asset-backed securities	16,009		—		(14)		15,995
Supranational securities	4,044		—		(3)		4,041
Total	$	120,392	$	—	$	(90)	$	120,302

Schedule of changes in accumulated other comprehensive income

The


	Three months ended June 30,				Six Months Ended June 30, 2022
	2022		2021		2022		2021
Beginning balance	$	(394)	$	2	$	(90)	$	3
Current period changes in fair value before reclassifications, net of tax	(134)		1		(438)		—
Amounts reclassified from accumulated other comprehensive income, net of tax	—		—		—		—
Total other comprehensive loss	$	(134)	$	1	(438)		—
Ending balance	$	(528)	$	3	$	(528)	$	3

Fair Value Measurements - (Tables)

6 Months Ended

Jun. 30, 2022

Fair Value Disclosures [Abstract]

Schedule of assets and liabilities that are measured at fair value on a recurring basis


	Fair Value Measurement Using
	Quoted prices in active markets for identical assets (Level 1)		Significant other observable inputs (Level 2)		Significant unobservable inputs (Level 3)
Assets
Investments in money market funds*	$	131,399	$	—	$	—
Investments in U.S. Treasury securities	$	47,862	$	—	$	—
Investments in corporate debt securities	$	—	$	102,974	$	—
Investments in asset-backed securities	$	—	20,014		$	—
Investments in supranational securities	$	—	—		$	—

*Investments in money market funds are reflected in cash and cash equivalents in the accompanying Condensed Unaudited Consolidated Balance Sheets.


	Fair Value Measurement Using
	Quoted prices in active markets for identical assets (Level 1)		Significant other observable inputs (Level 2)		Significant unobservable inputs (Level 3)
Assets
Investments in money market funds*	$	251,488	$	—	$	—

Investments in U.S. Treasury securities	$	18,185	$	—	$	—
Investments in corporate debt securities	$	—	$	82,081	$	—
Investments in asset-backed securities	$	—	$	15,995	$	—
Investments in supranational securities	$	—	$	4,041	$	—

*Investments in money market funds are reflected in cash and cash equivalents in the accompanying Condensed Unaudited Consolidated Balance Sheets.

Share-Based Compensation (Tables)

6 Months Ended

Jun. 30, 2022

Share-Based Payment Arrangement [Abstract]

Schedule of stock-based compensation by award type

Stock-based compensation by award type was as follows:


	Three Months Ended June 30,				Six Months Ended June 30,
	2022		2021		2022		2021
Stock options	$	3,750	$	1,042	$	7,074	$	2,511
Restricted stock units	2,571		1,003		4,576		1,795
Employee stock purchase plan	67		34		124		65
Total	$	6,388	$	2,079	$	11,774	$	4,371

Schedule of allocated stock-based compensation expense

The Company allocated stock-based compensation expense in the Company’s Consolidated Statements of Operations and Comprehensive Loss as follows:


	Three Months Ended June 30,				Six Months Ended June 30,
	2022		2021		2022		2021
Research and development	$	2,766	$	1,319	$	5,427	$	2,662
General and administrative	3,622		760		6,347		1,709
Total	$	6,388	$	2,079	$	11,774	$	4,371

Summary of the stock option activity including weighted average exercise prices, options outstanding and exercisable


	Number of Shares Underlying Options	Weighted Average Exercise Price
Outstanding, December 31, 2021	10,861	$	10.94
Granted	1,222	$	14.79
Exercised	(1,400)	$	3.31
Forfeited	(138)	$	14.78


Outstanding, June 30, 2022	10,545	$	12.35
Vested and exercisable, June 30, 2022	4,811	$	13.21
Vested and expected to vest, June 30, 2022	10,087	$	12.38

Summary of outstanding shares of RSU awards

The following table presents a summary of the Company's outstanding RSU awards granted as of June 30, 2022 (in thousands except weighted average grant-date fair value):


	Restricted Stock Units	Weighted Average Grant-Date Fair Value
Outstanding, December 31, 2021	2,246	$	9.82
Awarded	192	$	12.22
Vested	(182)	$	6.99
Forfeited	(41)	$	10.80
Outstanding, June 30, 2022	2,215	$	10.24
Outstanding, expected to vest	2,038	$	10.24

Operating Leases (Tables)

6 Months Ended

Jun. 30, 2022

Leases [Abstract]

Schedule of Maturity of Operating Lease Liability

The following presents the maturity of the Company’s operating lease liabilities as of June 30, 2022:


	June 30, 2022
Remainder of 2022	$	902
2023	1,216
2024	11
Total remaining obligation	$	2,129
Less imputed interest	(73)
Present value of lease liabilities	$	2,056

Business (Details)	Jun. 30, 2022 product_candidate
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 Accounting Policies - Segment and Geographic Information (Details)	6 Months Ended
	Jun. 30, 2022 segment
Accounting Policies [Abstract]
Number of operating segments	1
Number of reporting segments	1

Summary of Significant Accounting Policies - Property and Equipment (Details)	6 Months Ended
	Jun. 30, 2022
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 Accounting Policies - Share-Based Compensation (Details) - shares	1 Months Ended	6 Months Ended
	Jun. 30, 2016	Jun. 30, 2022	Apr. 30, 2016
Share-Based Compensation
Expected dividend yield		0.00%
Employee stock purchase plan
Share-Based Compensation
Number of initial shares reserved for issuance under the Plan (in shares)			1,000,000
Purchase period	6 months

Summary of Significant Accounting Policies - Schedule of Weighted-Average Assumptions (Details)	3 Months Ended		6 Months Ended
	Jun. 30, 2022	Jun. 30, 2021	Jun. 30, 2022	Jun. 30, 2021
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	76.00%	113.00%	83.00%	113.00%
Risk-free interest rate, minimum	2.53%	0.89%	1.38%	0.31%
Risk-free interest rate, maximum	2.99%	0.94%	2.99%	0.96%
Expected term of options (years)	4 years 9 months 18 days	5 years 2 months 12 days	4 years 10 months 24 days	5 years 1 month 6 days
Expected dividend yield	0.00%	0.00%	0.00%	0.00%

Net Loss Per Common Share - Narrative (Details) - shares	Jun. 30, 2022	Jun. 30, 2021
Earnings Per Share [Abstract]
Pre-funded warrants outstanding (in shares)	3,164,280	3,164,280

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				6 Months Ended
	Jun. 30, 2022	Mar. 31, 2022	Jun. 30, 2021	Mar. 31, 2021	Jun. 30, 2022	Jun. 30, 2021
Basic and diluted net loss per common share calculation:
Net loss	$ (49,263)	$ (34,536)	$ (30,143)	$ (26,795)	$ (83,799)	$ (56,938)
Weighted average common shares outstanding - basic (in shares)	119,687		93,409		119,223	93,382
Weighted average common shares outstanding - dilutive (in shares)	119,687		93,409		119,223	93,382
Net loss per share of common stock - basic (in usd per share)	$ (0.41)		$ (0.32)		$ (0.70)	$ (0.61)
Net loss per share of common stock - diluted (in usd per share)	$ (0.41)		$ (0.32)		$ (0.70)	$ (0.61)

- Definition

The amount of net income (loss) for the period per each share of common stock or unit outstanding during the reporting period.

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- Definition

The amount of net income (loss) for the period available to each share of common stock or common unit outstanding during the reporting period and to each share or unit that would have been outstanding assuming the issuance of common shares or units for all dilutive potential common shares or units outstanding during the reporting period.

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Reference 9: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 815
-SubTopic 40
-Section 65
-Paragraph 1
-Subparagraph (f)
-URI https://asc.fasb.org/extlink&oid=126732423&loc=SL123482106-238011

Reference 10: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 942
-SubTopic 220
-Section S99
-Paragraph 1
-Subparagraph (SX 210.9-04(27))
-URI https://asc.fasb.org/extlink&oid=120399700&loc=SL114874048-224260

Reference 11: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 260
-SubTopic 10
-Section 50
-Paragraph 1
-Subparagraph (a)
-URI https://asc.fasb.org/extlink&oid=124432515&loc=d3e3550-109257

Reference 12: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 944
-SubTopic 220
-Section S99
-Paragraph 1
-Subparagraph (SX 210.7-04(23))
-URI https://asc.fasb.org/extlink&oid=120400993&loc=SL114874131-224263

Reference 13: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 250
-SubTopic 10
-Section 50
-Paragraph 11
-Subparagraph (a)
-URI https://asc.fasb.org/extlink&oid=124431687&loc=d3e22694-107794

Reference 14: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 260
-SubTopic 10
-Section 45
-Paragraph 7
-URI https://asc.fasb.org/extlink&oid=126958026&loc=d3e1337-109256

Reference 15: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 815
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-Section 65
-Paragraph 1
-Subparagraph (e)(4)
-URI https://asc.fasb.org/extlink&oid=126732423&loc=SL123482106-238011

Reference 16: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 220
-SubTopic 10
-Section S99
-Paragraph 2
-Subparagraph (SX 210.5-03(25))
-URI https://asc.fasb.org/extlink&oid=126953954&loc=SL114868664-224227

+ Details

- Definition

The portion of profit or loss for the period, net of income taxes, which is attributable to the parent.

+ References

+ Details

Net Loss Per Common Share - Potentially Dilutive Securities (Details) - shares shares in Thousands	3 Months Ended		6 Months Ended
	Jun. 30, 2022	Jun. 30, 2021	Jun. 30, 2022	Jun. 30, 2021
Anti-dilutive securities
Total	12,761	11,186	12,761	11,186
Stock options outstanding
Anti-dilutive securities
Total	10,546	9,233	10,546	9,233
Restricted stock units
Anti-dilutive securities
Total	2,215	1,953	2,215	1,953

Licensing and Commercialization Agreements (Details) - DelSiTech - License Agreement	3 Months Ended	6 Months Ended
	Jun. 30, 2022 EUR (€)	Jun. 30, 2022 EUR (€)
Research and Development Arrangement, Contract to Perform for Others [Line Items]
Payment period		60 days
Termination notice period		60 days
Zimura
Research and Development Arrangement, Contract to Perform for Others [Line Items]
Payments for license fees	€ 1,250,000
Zimura \| Achievement of specified clinical and development milestones
Research and Development Arrangement, Contract to Perform for Others [Line Items]
Amount to be paid on achievement of milestone	€ 35,000,000	€ 35,000,000
Zimura \| Achievement of specified commercial sales milestones
Research and Development Arrangement, Contract to Perform for Others [Line Items]
Payments for license fees		€ 60,000,000

Cash, Cash Equivalents and Available for Sale Securities - Narrative (Details) - USD ($) $ in Thousands	Jun. 30, 2022	Dec. 31, 2021
Cash, Cash Equivalents, and Short-Term Investments [Abstract]
Cash and cash equivalents	$ 141,113	$ 261,447
Cash	9,700	9,900
Investments in money market funds	131,400	251,500
Available for sale securities	$ 170,850	$ 120,302

Cash, Cash Equivalents and Available for Sale Securities - Schedule of Available for Sale Securities (Details) - USD ($) $ in Thousands	Jun. 30, 2022	Dec. 31, 2021
Debt Securities, Available-for-sale [Line Items]
Amortized Cost	$ 171,377	$ 120,392
Gross Unrealized Gains	1	0
Gross Unrealized Losses	(528)	(90)
Fair Value	170,850	120,302
U.S. Treasury securities
Debt Securities, Available-for-sale [Line Items]
Amortized Cost	48,152	18,201
Gross Unrealized Gains	0	0
Gross Unrealized Losses	(290)	(16)
Fair Value	47,862	18,185
Corporate debt securities
Debt Securities, Available-for-sale [Line Items]
Amortized Cost	103,151	82,138
Gross Unrealized Gains	1	0
Gross Unrealized Losses	(178)	(57)
Fair Value	102,974	82,081
Asset-backed securities
Debt Securities, Available-for-sale [Line Items]
Amortized Cost	20,074	16,009
Gross Unrealized Gains	0	0
Gross Unrealized Losses	(60)	(14)
Fair Value	20,014	15,995
Supranational securities
Debt Securities, Available-for-sale [Line Items]
Amortized Cost	0	4,044
Gross Unrealized Gains	0	0
Gross Unrealized Losses	0	(3)
Fair Value	$ 0	$ 4,041

Cash, Cash Equivalents and Available for Sale Securities - Schedule of Accumulated Other Comprehensive Income (Details) - USD ($) $ in Thousands	3 Months Ended		6 Months Ended
	Jun. 30, 2022	Jun. 30, 2021	Jun. 30, 2022	Jun. 30, 2021
AOCI Attributable to Parent, Net of Tax [Roll Forward]
Beginning balance	$ 333,154	$ 167,188	$ 360,528	$ 191,563
Ending balance	292,946	139,573	292,946	139,573
Accumulated other comprehensive income associated with unrealized gain on available for sale securities
AOCI Attributable to Parent, Net of Tax [Roll Forward]
Beginning balance	(394)	2	(90)	3
Current period changes in fair value before reclassifications, net of tax	(134)	1	(438)	0
Amounts reclassified from accumulated other comprehensive income, net of tax	0	0	0	0
Total other comprehensive loss	(134)	1	(438)	0
Ending balance	$ (528)	$ 3	$ (528)	$ 3

- Definition

+ References

+ Details

Fair Value Measurements - Assets and Liabilities Measured at Fair Value on a Recurring Basis (Details) - Recurring basis - USD ($) $ in Thousands	Jun. 30, 2022	Dec. 31, 2021
Quoted prices in active markets for identical assets (Level 1) \| Investments in money market funds
Fair Value Measurements
Investments	$ 131,399	$ 251,488
Quoted prices in active markets for identical assets (Level 1) \| Investments in U.S. Treasury securities
Fair Value Measurements
Investments	47,862	18,185
Quoted prices in active markets for identical assets (Level 1) \| Investments in corporate debt securities
Fair Value Measurements
Investments	0	0
Quoted prices in active markets for identical assets (Level 1) \| Investments in asset-backed securities
Fair Value Measurements
Investments	0	0
Quoted prices in active markets for identical assets (Level 1) \| Investments in supranational securities
Fair Value Measurements
Investments	0	0
Significant other observable inputs (Level 2) \| Investments in money market funds
Fair Value Measurements
Investments	0	0
Significant other observable inputs (Level 2) \| Investments in U.S. Treasury securities
Fair Value Measurements
Investments	0	0
Significant other observable inputs (Level 2) \| Investments in corporate debt securities
Fair Value Measurements
Investments	102,974	82,081
Significant other observable inputs (Level 2) \| Investments in asset-backed securities
Fair Value Measurements
Investments	20,014	15,995
Significant other observable inputs (Level 2) \| Investments in supranational securities
Fair Value Measurements
Investments	0	4,041
Significant unobservable inputs (Level 3) \| Investments in money market funds
Fair Value Measurements
Investments	0	0
Significant unobservable inputs (Level 3) \| Investments in U.S. Treasury securities
Fair Value Measurements
Investments	0	0
Significant unobservable inputs (Level 3) \| Investments in corporate debt securities
Fair Value Measurements
Investments	0	0
Significant unobservable inputs (Level 3) \| Investments in asset-backed securities
Fair Value Measurements
Investments	0	0
Significant unobservable inputs (Level 3) \| Investments in supranational securities
Fair Value Measurements
Investments	$ 0	$ 0

Share-Based Compensation - Narrative (Details) - USD ($) $ in Thousands	1 Months Ended					3 Months Ended		6 Months Ended		114 Months Ended
	May 31, 2022	Dec. 31, 2021	Sep. 30, 2021	Feb. 28, 2021	Mar. 31, 2020	Jun. 30, 2022	Jun. 30, 2021	Jun. 30, 2022	Jun. 30, 2021	Jun. 30, 2022	Oct. 31, 2019	Apr. 30, 2016
Stock options
Stock Option and Compensation Plans
Unrecognized share-based compensation costs for option awards, net of estimated forfeitures						$ 41,300		$ 41,300		$ 41,300
Expected weighted average period to recognize share-based compensation costs (in years)								3 years 1 month 6 days
Restricted stock units
Stock Option and Compensation Plans
Expected weighted average period to recognize share-based compensation costs (in years)								2 years 9 months 18 days
Unrecognized compensation costs, net of estimated forfeitures						$ 16,300		$ 16,300		$ 16,300
Employee stock purchase plan
Stock Option and Compensation Plans
Number of initial shares reserved for issuance under the Plan (in shares)												1,000,000
Number of shares available for future purchase (in shares)						738,315		738,315		738,315
Common stock issued (in shares)						0	0	15,095	24,422
Proceeds from stock plans								$ 184	$ 121
2013 Plan
Stock Option and Compensation Plans
Increase in number of shares available under the Plan (in shares)								2,542,000		15,624,000
Number of shares of common stock available for issuance under the Plan (in shares)						3,507,000		3,507,000		3,507,000
2019 Inducement Plan \| Stock options
Stock Option and Compensation Plans
Increase in number of shares available under the Plan (in shares)	1,000,000	1,000,000	1,000,000	600,000	1,000,000
Number of shares of common stock available for issuance under the Plan (in shares)						1,341,000		1,341,000		1,341,000
Number of initial shares reserved for issuance under the Plan (in shares)											1,000,000

Share-Based Compensation - Summary of Share-Based Compensation Expense by Award Type (Details) - USD ($) $ in Thousands	3 Months Ended		6 Months Ended
	Jun. 30, 2022	Jun. 30, 2021	Jun. 30, 2022	Jun. 30, 2021
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Stock-based compensation expense	$ 6,388	$ 2,079	$ 11,774	$ 4,371
Stock options
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Stock-based compensation expense	3,750	1,042	7,074	2,511
Restricted stock units
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Stock-based compensation expense	2,571	1,003	4,576	1,795
Employee stock purchase plan
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Stock-based compensation expense	$ 67	$ 34	$ 124	$ 65

Share-Based Compensation - Schedule of Allocated Stock-based Compensation Expense (Details) - USD ($) $ in Thousands	3 Months Ended		6 Months Ended
	Jun. 30, 2022	Jun. 30, 2021	Jun. 30, 2022	Jun. 30, 2021
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Stock-based compensation expense	$ 6,388	$ 2,079	$ 11,774	$ 4,371
Research and development
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Stock-based compensation expense	2,766	1,319	5,427	2,662
General and administrative
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Stock-based compensation expense	$ 3,622	$ 760	$ 6,347	$ 1,709

Share-Based Compensation - Summary of Stock Option Activity (Details) - Stock options shares in Thousands	6 Months Ended
	Jun. 30, 2022 $ / shares shares
Number of Shares Underlying Options
Outstanding, beginning balance (in shares) \| shares	10,861
Granted (in shares) \| shares	1,222
Exercised (in shares) \| shares	(1,400)
Forfeited (in shares) \| shares	(138)
Outstanding, ending balance (in shares) \| shares	10,545
Vested and exercisable, end of period (in shares) \| shares	4,811
Vested and expected to vest, end of period (in shares) \| shares	10,087
Weighted Average Exercise Price
Outstanding, beginning balance (in usd per share) \| $ / shares	$ 10.94
Granted (in usd per share) \| $ / shares	14.79
Exercised (in usd per share) \| $ / shares	3.31
Forfeited (in usd per share) \| $ / shares	14.78
Outstanding, ending balance (in usd per share) \| $ / shares	12.35
Vested and exercisable, end of period (in usd per share) \| $ / shares	13.21
Vested and expected to vest, end of period (in usd per share) \| $ / shares	$ 12.38

Share-Based Compensation - Summary of Outstanding RSU Awards Granted (Details) - Restricted stock units shares in Thousands	6 Months Ended
	Jun. 30, 2022 $ / shares shares
Restricted Stock Units
Outstanding, beginning balance (in shares) \| shares	2,246
Awarded (in shares) \| shares	192
Vested (in shares) \| shares	(182)
Forfeited (in shares) \| shares	(41)
Outstanding, ending balance (in shares) \| shares	2,215
Restricted stock units, Outstanding, expected to vest (in shares) \| shares	2,038
Weighted Average Grant-Date Fair Value
Outstanding, beginning balance (in usd per share) \| $ / shares	$ 9.82
Awarded (in usd per share) \| $ / shares	12.22
Vested (in usd per share) \| $ / shares	6.99
Forfeited (in usd per share) \| $ / shares	10.80
Outstanding, ending balance (in usd per share) \| $ / shares	10.24
Weighted average grant-date fair value, Outstanding, expected to vest (in usd per share) \| $ / shares	$ 10.24

Commitments and Contingencies (Details)			3 Months Ended	6 Months Ended
Commitments and Contingencies (Details)	Jun. 22, 2021	Apr. 23, 2021	Jun. 30, 2022 EUR (€)	Jun. 30, 2022 USD ($) licensed_product shares	Jun. 30, 2022 EUR (€) licensed_product shares	Jun. 30, 2022 EUR (€) shares	Sep. 08, 2021 USD ($)
Commitments and contingencies
Litigation stay period (in days)	60 days	10 days
Settled Litigation
Commitments and contingencies
Settlement to be paid							$ 29,000,000
Inception 4
Commitments and contingencies
Contingent consideration arrangements, maximum amount				$ 105,000,000
Share price calculation base, period (in days)				5 days	5 days
Contingent consideration arrangements, maximum amount (in shares) \| shares				7,200,000		7,200,000
Contingent consideration arrangements, contingently issuable shares as a percentage of shares outstanding				19.90%	19.90%
GA Product \| Inception 4
Commitments and contingencies
Clinical and marketing approval milestones				$ 45,000,000
Wet AMD Product \| Inception 4
Commitments and contingencies
Clinical and marketing approval milestones				60,000,000
DelSiTech \| Zimura \| License Agreement
Commitments and contingencies
Payments for license fees \| €			€ 1,250,000
Achievement of specified clinical and regulatory milestones \| Archemix \| C5 Licensed Product \| Maximum \| License Agreement
Commitments and contingencies
Amount to be paid on achievement of milestone				50,500,000
Achievement of specified clinical and regulatory milestones \| University Of Massachusetts (UMass) \| miniCEP290 \| License Agreement \| Research and Development Expense
Commitments and contingencies
Payments for license fees				$ 14,750,000
Shares issued for license fees (in shares) \| shares				75,000	75,000
First indication \| Archemix \| C5 Licensed Product \| Maximum \| License Agreement
Commitments and contingencies
Amount to be paid on achievement of milestone				$ 24,500,000
Second and third indication \| Archemix \| C5 Licensed Product \| Maximum \| License Agreement
Commitments and contingencies
Amount to be paid on achievement of milestone				23,500,000
Sustained delivery applications \| Archemix \| C5 Licensed Product \| Maximum \| License Agreement
Commitments and contingencies
Amount to be paid on achievement of milestone				2,500,000
Achievement of specified commercial milestones \| Archemix \| C5 Licensed Product \| Maximum \| License Agreement
Commitments and contingencies
Amount to be paid on achievement of milestone				22,500,000
Specified clinical, marketing approval and reimbursement approval milestones with respect to a licensed product \| University of Florida Research Foundation (UFRF) \| RHO-adRP License Agreement
Commitments and contingencies
Amount to be paid on achievement of milestone				$ 23,500,000
Specified clinical, marketing approval and reimbursement approval milestones with respect to a licensed product \| University Of Florida Research Foundation (UFRF) And University Of Pennsylvania (Penn) \| BEST1 License Agreement \| Research and Development Expense
Commitments and contingencies
Number of licensed products \| licensed_product				1	1
Specified clinical, marketing approval and reimbursement approval milestones with respect to a licensed product \| University Of Florida Research Foundation (UFRF) And University Of Pennsylvania (Penn) \| Primary Licensed Product \| BEST1 License Agreement \| Research and Development Expense
Commitments and contingencies
Payments for license fees				$ 15,700,000
Specified clinical, marketing approval and reimbursement approval milestones with respect to a licensed product \| University Of Florida Research Foundation (UFRF) And University Of Pennsylvania (Penn) \| Other Licensed Product \| BEST1 License Agreement \| Research and Development Expense
Commitments and contingencies
Payments for license fees				3,100,000
Specified commercial sales milestones with respect to a licensed product \| University of Florida Research Foundation (UFRF) \| RHO-adRP License Agreement
Commitments and contingencies
Amount to be paid on achievement of milestone				$ 70,000,000
Specified commercial sales milestones with respect to a licensed product \| University Of Florida Research Foundation (UFRF) And University Of Pennsylvania (Penn) \| BEST1 License Agreement \| Research and Development Expense
Commitments and contingencies
Number of licensed products \| licensed_product				1	1
Specified commercial sales milestones with respect to a licensed product \| University Of Florida Research Foundation (UFRF) And University Of Pennsylvania (Penn) \| Primary Licensed Product \| BEST1 License Agreement \| Research and Development Expense
Commitments and contingencies
Payments for license fees				$ 48,000,000
Specified commercial sales milestones with respect to a licensed product \| University Of Florida Research Foundation (UFRF) And University Of Pennsylvania (Penn) \| Other Licensed Product \| BEST1 License Agreement \| Research and Development Expense
Commitments and contingencies
Payments for license fees				9,600,000
Specified commercial sales milestones with respect to a licensed product \| University Of Massachusetts (UMass) \| miniCEP290 \| License Agreement \| Research and Development Expense
Commitments and contingencies
Payments for license fees				$ 48,000,000
Achievement of specified clinical and development milestones \| DelSiTech \| Zimura \| License Agreement
Commitments and contingencies
Amount to be paid on achievement of milestone \| €						€ 35,000,000
Achievement of specified commercial sales milestones \| DelSiTech \| Zimura \| License Agreement
Commitments and contingencies
Payments for license fees \| €					€ 60,000,000

Operating Leases - Narrative (Details) $ in Thousands	3 Months Ended	6 Months Ended
Operating Leases - Narrative (Details) $ in Thousands	Jun. 30, 2022 USD ($) ft²	Jun. 30, 2022 USD ($) ft²	May 31, 2022	Jan. 01, 2022 USD ($)	Dec. 31, 2021 USD ($)
Lessee, Lease, Description [Line Items]
Right-of-use asset, net	$ 2,020	$ 2,020		$ 1,000	$ 1,522
Present value of lease liabilities	2,056	2,056		$ 1,000
Weighted average estimated incremental borrowing rate				8.00%
Operating lease rent expense	200	500
Cash paid from operating cash flows	$ 200	$ 500
Operating lease, weighted average remaining lease term	1 year 2 months 12 days	1 year 2 months 12 days
Cranbury office lease
Lessee, Lease, Description [Line Items]
Lease, extended term			1 year
Parsippany Office Lease
Lessee, Lease, Description [Line Items]
Area of lease property \| ft²	34,836	34,836

Operating Leases - Operating Lease Liability Maturities (Details) - USD ($) $ in Thousands	Jun. 30, 2022	Jan. 01, 2022
Leases [Abstract]
Remainder of 2022	$ 902
2023	1,216
2024	11
Total remaining obligation	2,129
Less imputed interest	(73)
Present value of lease liabilities	$ 2,056	$ 1,000

Subsequent Events (Details) - Subsequent Event - 2022 Term Loan	Jul. 26, 2022 USD ($)
Subsequent Event [Line Items]
Lenders option to invest in equity financing	$ 10,000,000
Net cash proceeds	75,000,000
Line of Credit
Subsequent Event [Line Items]
Aggregate principal amount (up to)	$ 250,000,000
Capped interest rate per annum	10.25%
Interest-only payment, period	42 months
Covenant revenue from sale, maintained market capitalization	$ 600,000,000
End term charges percentage	4.25%
Percentage of prepayment penalty, prepayment occurs prior to first anniversary of closing date	2.00%
Percentage of prepayment penalty, prepayment occurs on or after the first anniversary and prior to the second anniversary of the closing date	1.50%
Percentage of prepayment penalty, prepayment occurs occurs on or after the second anniversary and prior to the third anniversary of the closing date	0.75%
Line of Credit \| Minimum
Subsequent Event [Line Items]
Qualified Cash percent	50.00%
Line of Credit \| Maximum
Subsequent Event [Line Items]
Qualified Cash percent	90.00%
Line of Credit \| The Wall Street Journal prime rate
Subsequent Event [Line Items]
Basis spread on variable rate	6.25%
Line of Credit \| The Wall Street Journal prime rate \| Minimum
Subsequent Event [Line Items]
Basis spread on variable rate	4.00%
Line of Credit \| The Wall Street Journal prime rate \| Maximum
Subsequent Event [Line Items]
Basis spread on variable rate	8.75%
Line of Credit \| On Closing Date
Subsequent Event [Line Items]
Aggregate principal amount (up to)	$ 50,000,000
Line of Credit \| Milestone 1 is achieved through December 15, 2022
Subsequent Event [Line Items]
Aggregate principal amount (up to)	50,000,000
Drawn down increments at company's election	5,000,000
Line of Credit \| Milestone 2 is achieved through September 30, 2023
Subsequent Event [Line Items]
Aggregate principal amount (up to)	25,000,000
Drawn down increments at company's election	5,000,000
Line of Credit \| Milestone 3 is achieved
Subsequent Event [Line Items]
Aggregate principal amount (up to)	75,000,000
Drawn down increments at company's election	5,000,000
Line of Credit \| Subject to FDA approval of Zimura in GA
Subsequent Event [Line Items]
Aggregate principal amount (up to)	50,000,000
Drawn down increments at company's election	5,000,000
Revolving Credit Facility \| Milestone 3 is achieved
Subsequent Event [Line Items]
Aggregate principal amount (up to)	50,000,000
Convertible Notes
Subsequent Event [Line Items]
Aggregate principal amount (up to)	$ 400,000,000