0001410939-22-000059 | 10-Q | XBRL Viewer

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Summary of Significant Accounting Policies - Property and Equipment (Details)	3 Months Ended
	Mar. 31, 2022
Minimum
Property and equipment
Estimated useful live (in years)	3 years
Maximum
Property and equipment
Estimated useful live (in years)	10 years

Share-Based Compensation - Summary of Outstanding RSU Awards Granted (Details) - Restricted stock units shares in Thousands	3 Months Ended
	Mar. 31, 2022 $ / shares shares
Restricted Stock Units
Outstanding, beginning balance (in shares) \| shares	2,246
Awarded (in shares) \| shares	22
Vested (in shares) \| shares	(31)
Forfeited (in shares) \| shares	(37)
Outstanding, ending balance (in shares) \| shares	2,200
Outstanding, expected to vest (in shares) \| shares	2,024
Weighted Average Grant-Date Fair Value
Outstanding, beginning balance (in usd per share) \| $ / shares	$ 9.82
Awarded (in usd per share) \| $ / shares	15.02
Vested (in usd per share) \| $ / shares	6.33
Forfeited (in usd per share) \| $ / shares	10.52
Outstanding, ending balance (in usd per share) \| $ / shares	9.91
Outstanding, expected to vest (in usd per share) \| $ / shares	$ 9.91

Summary of Significant Accounting Policies (Tables)

3 Months Ended

Mar. 31, 2022

Accounting Policies [Abstract]

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

The weighted-average assumptions used to estimate grant date fair value of stock options using the Black-Scholes option pricing model were as follows for the three month periods ended March 31, 2022 and 2021:


	Three Months Ended March 31,
	2022	2021
Expected common stock price volatility	101%	113%
Risk-free interest rate	1.38%-1.63%	0.39%-0.96%
Expected term of options (years)	5.2	5.3
Expected dividend yield	—	—

Fair Value Measurements - (Tables)

3 Months Ended

Mar. 31, 2022

Fair Value Disclosures [Abstract]

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

The following table presents, for each of the fair value hierarchy levels required under ASC 820, the Company's assets and liabilities that are measured at fair value on a recurring basis as of March 31, 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*	$	181,948	$	—	$	—
Investments in U.S. Treasury securities	$	36,030	$	—	$	—
Investments in corporate debt securities	$	—	$	100,210	$	—
Investments in asset-backed securities	$	—	13,515		$	—
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.


	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.

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

3 Months Ended

Mar. 31, 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 March 31, 2022
	Amortized Cost		Gross Unrealized Gains		Gross Unrealized Losses		Fair Value
U.S. Treasury securities	$	36,194	$	—	$	(164)	$	36,030
Corporate debt securities	100,383		—		(173)		100,210
Asset-backed securities	13,567		—		(52)		13,515
Supranational securities	4,046		—		(5)		4,041
Total	$	154,190	$	—	$	(394)	$	153,796


	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 changes in accumulated other comprehensive income associated with the unrealized gain on available for sale securities during the three months ended March 31, 2022 and 2021, respectively, were as follows:


	Three months ended March 31,
	2022		2021
Beginning balance	$	(90)	$	3
Current period changes in fair value before reclassifications, net of tax	(304)		(1)
Amounts reclassified from accumulated other comprehensive income, net of tax	—		—
Total other comprehensive loss	$	(304)	$	(1)
Ending balance	$	(394)	$	2

Share-Based Compensation - Summary of Stock Option Activity (Details) - Stock options shares in Thousands	3 Months Ended
	Mar. 31, 2022 $ / shares shares
Number of Shares Underlying Options
Outstanding, beginning balance (in shares) \| shares	10,861
Granted (in shares) \| shares	352
Exercised (in shares) \| shares	(651)
Forfeited (in shares) \| shares	(92)
Outstanding, ending balance (in shares) \| shares	10,470
Vested and exercisable, end of period (in shares) \| shares	5,248
Vested and expected to vest, end of period (in shares) \| shares	10,052
Weighted Average Exercise Price
Outstanding, beginning balance (in usd per share) \| $ / shares	$ 10.94
Granted (in usd per share) \| $ / shares	16.13
Exercised (in usd per share) \| $ / shares	2.85
Forfeited (in usd per share) \| $ / shares	9.58
Outstanding, ending balance (in usd per share) \| $ / shares	11.63
Vested and exercisable, end of period (in usd per share) \| $ / shares	12.43
Vested and expected to vest, end of period (in usd per share) \| $ / shares	$ 11.67

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
	Mar. 31, 2022	Mar. 31, 2021
Basic and diluted net loss per common share calculation:
Net loss	$ (34,536)	$ (26,795)
Weighted average common shares outstanding - basic (in shares)	118,755	93,311
Weighted average common shares outstanding - dilutive (in shares)	118,755	93,311
Net loss per share of common stock - basic (in usd per share)	$ (0.29)	$ (0.29)
Net loss per share of common stock - diluted (in usd per share)	$ (0.29)	$ (0.29)
Anti-dilutive securities
Total	12,670	11,216
Stock options outstanding
Anti-dilutive securities
Total	10,470	9,178
Restricted stock units
Anti-dilutive securities
Total	2,200	2,038

Share-Based Compensation - Summary of Share-Based Compensation Expense by Award Type (Details) - USD ($) $ in Thousands	3 Months Ended
	Mar. 31, 2022	Mar. 31, 2021
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Stock-based compensation expense	$ 5,386	$ 2,292
Stock options
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Stock-based compensation expense	3,323	1,469
Restricted stock units
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Stock-based compensation expense	2,005	792
Employee stock purchase plan
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Stock-based compensation expense	$ 58	$ 31

Condensed Unaudited Consolidated Balance Sheets (Parenthetical) - $ / shares	Mar. 31, 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)	115,973,952	115,277,012
Common stock, shares outstanding (in shares)	115,973,952	115,277,012

Condensed Unaudited Consolidated Statements of Cash Flows - USD ($) $ in Thousands	3 Months Ended
	Mar. 31, 2022	Mar. 31, 2021
Operating Activities
Net loss	$ (34,536)	$ (26,795)
Adjustments to reconcile net loss to net cash used in operating activities
Depreciation and other expense	21	13
Amortization of premium and discounts on investment securities	300	395
Share-based compensation	5,386	2,292
Changes in operating assets and liabilities:
Prepaid expense and other assets	435	(516)
Accrued interest receivable	(66)	122
Accrued research and development expenses	(1,486)	848
Accounts payable and accrued expenses	(7,562)	(5,816)
Change in working capital	(4)	0
Net cash used in operating activities	(37,512)	(29,457)
Investing Activities
Purchase of marketable securities	(48,897)	(26,710)
Purchase of property and equipment	(116)	0
Maturities of marketable securities	14,865	19,000
Net cash used in investing activities	(34,148)	(7,710)
Financing Activities
Proceeds from employee stock plan purchases	2,080	129
Net cash provided by financing activities	2,080	129
Net increase (decrease) in cash and cash equivalents	(69,580)	(37,038)
Cash and cash equivalents
Beginning of period	261,447	66,373
End of period	$ 191,867	$ 29,335

Net Loss Per Common Share - Narrative (Details) - shares	Mar. 31, 2022	Mar. 31, 2021
Earnings Per Share [Abstract]
Pre-funded warrants outstanding (in shares)	3,164,280	3,164,280

Cash, Cash Equivalents and Available for Sale Securities - Narrative (Details) - USD ($) $ in Thousands	Mar. 31, 2022	Dec. 31, 2021
Cash, Cash Equivalents, and Short-term Investments [Abstract]
Cash and cash equivalents	$ 191,867	$ 261,447
Cash	9,900	9,900
Investments in money market funds	181,900	251,500
Available for sale securities	$ 153,796	$ 120,302

Commitments and Contingencies	3 Months Ended
Commitments and Contingencies	Mar. 31, 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. IC-100 - University of Florida and the University of Pennsylvania Under its exclusive license agreement with the University of Florida Research Foundation, Incorporated (“UFRF”) and the University of Pennsylvania (“Penn”) for rights to IC-100, the Company is obligated to make payments to UFRF, for the benefit of Penn and UFRF (together, the “Licensors”), of up to an aggregate of $23.5 million if the Company achieves specified clinical, marketing approval and reimbursement approval milestones with respect to a licensed product and up to an aggregate of an additional $70.0 million if the Company achieves specified commercial sales milestones with respect to a licensed product. The Company is also obligated to pay UFRF, for the benefit of the Licensors, a low single-digit percentage of net sales of licensed products. The Company is also obligated to pay UFRF, for the benefit of the Licensors, a double-digit percentage of specified non-royalty payments the Company may receive from any third-party sublicensee of the licensed patent rights. Further, if the Company receives a rare pediatric disease priority review voucher from the FDA in connection with obtaining marketing approval for a licensed product and the Company subsequently uses such priority review voucher in connection with a different product candidate, the Company will be obligated to pay UFRF, for the benefit of the Licensors, aggregate payments in the low double-digit millions of dollars based on certain approval and commercial sales milestones with respect to such other product candidate. In addition, if the Company sells such a priority review voucher to a third party, the Company will be obligated to pay UFRF, for the benefit of the Licensors, a low double-digit percentage of any consideration received from such third party in connection with such sale. IC-200 - University of Pennsylvania and the University of Florida Under its exclusive license agreement with Penn and UFRF for rights to IC-200, the Company is obligated to make payments to Penn, for the benefit of the Licensors, of up to an aggregate of $15.7 million if the Company achieves specified clinical, marketing approval and reimbursement approval milestones with respect to one licensed product and up to an aggregate of an additional $3.1 million if the Company achieves these same milestones with respect to a different licensed product. In addition, the Company is obligated to make payments to Penn, for the benefit of the Licensors, of up to an aggregate of $48.0 million if the Company achieves specified commercial sales milestones with respect to one licensed product and up to an aggregate of an additional $9.6 million if the Company achieves these same milestones with respect to a different licensed product. The Company is also obligated to pay Penn, for the benefit of the Licensors, a low single-digit percentage of net sales of licensed products. The Company is also obligated to pay Penn, for the benefit of the Licensors, a high single-digit to a mid-teen percentage of specified non-royalty payments the Company may receive from any third-party sublicensee of the licensed patent rights, with the applicable percentage based upon the stage of development of the sublicensed product at the time the Company enters into the sublicense. Further, if the Company receives a rare pediatric disease priority review voucher from the FDA in connection with obtaining marketing approval for a licensed product and the Company subsequently uses such priority review voucher in connection with a different product candidate outside the scope of the agreement, the Company will be obligated to pay Penn, for the benefit of the Licensors, aggregate payments in the low double-digit millions of dollars based on certain approval and commercial sales milestones with respect to such other product candidate. In addition, if the Company sells such a priority review voucher to a third party, the Company will be obligated to pay Penn, for the benefit of the Licensors, a high single-digit percentage of any consideration received from such third party in connection with such sale. miniCEP290 Program - University of Massachusetts Under its exclusive license agreement with the University of Massachusetts (“UMass”) for its miniCEP290 program, which targets LCA10, which is associated with mutations in the CEP290 gene, the Company is obligated to pay UMass up to an aggregate of $14.75 million in cash and issue up to 75,000 shares of common stock of the Company if the Company achieves specified clinical and regulatory milestones with respect to a licensed product. In addition, the Company is obligated to pay UMass up to an aggregate of $48.0 million if the Company achieves specified commercial sales milestones with respect to a licensed product. The Company is also obligated to pay UMass royalties at a low single-digit percentage of net sales of licensed products. If the Company or any of its affiliates sublicenses any of the licensed patent rights or know-how to a third party, the Company will be obligated to pay UMass a high single-digit to a mid-tens percentage of the consideration received in exchange for such sublicense, with the applicable percentage based upon the stage of development of the licensed products at the time the Company or the applicable affiliate enters into the sublicense. If the Company receives a priority review voucher from the FDA in connection with obtaining marketing approval for a licensed product, and the Company subsequently uses such priority review voucher in connection with a different product candidate outside the scope of the agreement, the Company will be obligated to pay UMass a low-tens percentage of the fair market value of the priority review voucher at the time of approval of such product candidate and a low-twenties percentage of the fair market value of the priority review voucher at the time of achievement of a specified commercial sales milestone for such product candidate. In addition, if the Company sells such a priority review voucher to a third party, the Company will be obligated to pay UMass a low-thirties percentage of any consideration received from such third party in connection with such sale. IC-500 - Former Equityholders of Inception 4 Under the agreement and plan of merger between the Company and Inception 4, Inc. (“Inception 4”), pursuant to which the Company acquired IC-500 and its other HtrA1 inhibitors (the “Inception 4 Merger Agreement”), the Company is obligated to make payments to the former equityholders of Inception 4 of up to an aggregate of $105 million, subject to the terms and conditions of the Inception 4 Merger Agreement, if the Company achieves certain specified clinical and regulatory milestones with respect to IC-500 or any other product candidate from its HtrA1 inhibitor program, with $45 million of such potential payments relating to GA and $60 million of such potential payments relating to wet AMD. Under the Inception 4 Merger Agreement, the Company does not owe any commercial milestones or royalties based on net sales. The future milestone payments will be payable in the form of shares of the Company's common stock, calculated based on the price of its common stock over a five-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 equityholders of Inception 4, including for breaches of the representations and warranties, covenants and agreements of the Company and its subsidiaries (other than Inception 4) in the Inception 4 Merger Agreement. Employment Contracts The Company also has letter agreements with certain employees that require the funding of a specific level of payments if certain events, such as a termination of employment in connection with a change in control or termination of employment by the employee for good reason or by the Company without cause, occur. Contract Service Providers In addition, in the course of normal business operations, the Company has agreements with contract service providers to assist in the performance of the Company’s research and development and manufacturing activities. Expenditures to CROs and CDMOs represent significant costs in preclinical and clinical development. Subject to required notice periods and the Company’s obligations under binding purchase orders and any cancellation fees that the Company may be obligated to pay, the Company can elect to discontinue the work under these agreements at any time. Legal Proceedings On January 11, 2017, a putative class action lawsuit was filed against the Company and certain of its current and former executive officers in the United States District Court for the Southern District of New York, captioned Frank Micholle v. IVERIC bio, Inc., et al., No. 1:17-cv-00210. On March 9, 2017, a related putative class action lawsuit was filed against the Company and the same group of its current and former executive officers in the United States District Court for the Southern District of New York, captioned Wasson v. IVERIC bio, Inc., et al., No. 1:17-cv-01758. These cases were consolidated on March 13, 2018. On June 4, 2018, the lead plaintiff filed a consolidated amended complaint (the “CAC”). The CAC purports to be brought on behalf of shareholders who purchased the Company’s common stock between March 2, 2015 and December 12, 2016. The CAC generally alleges that the Company and certain of its officers violated Sections 10(b) and/or 20(a) of the Securities Exchange Act of 1934 and Rule 10b-5 promulgated thereunder by making allegedly false and/or misleading statements concerning the results of the Company’s Phase 2b trial and the prospects of the Company’s Phase 3 trials for Fovista in combination with anti-VEGF agents for the treatment of wet AMD. The CAC seeks unspecified damages, attorneys’ fees, and other costs. The Company and individual defendants filed a motion to dismiss the CAC on July 27, 2018. On September 18, 2019, the court issued an order dismissing some, but not all, of the allegations in the CAC. On November 18, 2019, the Company and the individual defendants filed an answer to the complaint. On June 12, 2020, the lead plaintiff filed a motion for class certification. On August 11, 2020, the defendants filed a notice of non-opposition to lead plaintiff's motion for class certification. 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. 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.

Condensed Unaudited Consolidated Balance Sheets - USD ($) $ in Thousands	Mar. 31, 2022	Dec. 31, 2021
Current assets
Cash and cash equivalents	$ 191,867	$ 261,447
Available for sale securities	153,796	120,302
Prepaid expenses and other current assets	5,304	5,739
Income tax receivable	0	0
Total current assets	350,967	387,488
Property and equipment, net	443	348
Right-of-use asset, net	1,295	1,522
Other assets	0	0
Total assets	352,705	389,358
Current liabilities
Accrued research and development expenses	12,917	14,403
Accounts payable and accrued expenses	5,294	12,856
Lease liability	955	952
Total current liabilities	19,166	28,211
Lease liability, non-current	385	619
Total liabilities	19,551	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, 115,973,952 and 115,277,012 shares issued and outstanding at March 31, 2022 and December 31, 2021, respectively	116	115
Additional paid-in capital	1,047,563	1,040,098
Accumulated deficit	(714,131)	(679,595)
Accumulated other comprehensive income	(394)	(90)
Total stockholders' equity	333,154	360,528
Total liabilities and stockholders' equity	$ 352,705	$ 389,358

Share-Based Compensation - Narrative (Details) - USD ($) $ in Thousands	1 Months Ended				3 Months Ended		12 Months Ended
	Dec. 31, 2021	Sep. 30, 2021	Feb. 28, 2021	Mar. 31, 2020	Mar. 31, 2022	Mar. 31, 2021	Dec. 31, 2021	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					$ 38,000
Expected weighted average period to recognize share-based compensation costs (in years)					3 years 2 months 12 days
Restricted stock units
Stock Option and Compensation Plans
Expected weighted average period to recognize share-based compensation costs (in years)					3 years
Unrecognized compensation costs, net of estimated forfeitures					$ 16,900
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
Common stock issued (in shares)					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)					15,624,000		2,542,000
Number of shares of common stock available for issuance under the Plan (in shares)					3,717,000
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	600,000	1,000,000
Number of shares of common stock available for issuance under the Plan (in shares)					1,121,000
Number of initial shares reserved for issuance under the Plan (in shares)								1,000,000

Business (Details)	Mar. 31, 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

Cash, Cash Equivalents and Available for Sale Securities

3 Months Ended

Mar. 31, 2022

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

Cash, Cash Equivalents and Available for Sale Securities

The Company considers all highly liquid investments purchased with original maturities of 90 days or less at the date of purchase to be cash equivalents. As of March 31, 2022 and December 31, 2021, the Company had cash and cash equivalents of approximately $191.9 million and $261.4 million, respectively. Cash and cash equivalents included cash of $9.9 million at March 31, 2022 and $9.9 million at December 31, 2021. Cash and cash equivalents at March 31, 2022 and December 31, 2021 included $181.9 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 March 31, 2022 and December 31, 2021, the Company held available for sale securities of $153.8 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 March 31, 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 March 31, 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 March 31, 2022
	Amortized Cost		Gross Unrealized Gains		Gross Unrealized Losses		Fair Value
U.S. Treasury securities	$	36,194	$	—	$	(164)	$	36,030
Corporate debt securities	100,383		—		(173)		100,210
Asset-backed securities	13,567		—		(52)		13,515
Supranational securities	4,046		—		(5)		4,041
Total	$	154,190	$	—	$	(394)	$	153,796


	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 March 31, 2022 and 2021, respectively, were as follows:


	Three months ended March 31,
	2022		2021
Beginning balance	$	(90)	$	3
Current period changes in fair value before reclassifications, net of tax	(304)		(1)
Amounts reclassified from accumulated other comprehensive income, net of tax	—		—
Total other comprehensive loss	$	(304)	$	(1)
Ending balance	$	(394)	$	2

Share-Based Compensation

3 Months Ended

Mar. 31, 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 March 31, 2022, the Company had approximately 3,717,000 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 and December 2021, 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 and an additional 1,000,000 shares, respectively, of its common stock for issuance under the plan. As of March 31, 2022, the Company had approximately 1,121,000 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 March 31,
	2022		2021
Stock options	$	3,323	$	1,469
Restricted stock units	2,005		792
Employee stock purchase plan	58		31
Total	$	5,386	$	2,292

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


	Three Months Ended March 31,
	2022		2021
Research and development	$	2,661	$	1,343
General and administrative	2,725		949
Total	$	5,386	$	2,292

Stock Options

A summary of the stock option activity, weighted average exercise prices, options outstanding, exercisable and expected to vest as of March 31, 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	352	$	16.13
Exercised	(651)	$	2.85
Forfeited	(92)	$	9.58


Outstanding, March 31, 2022	10,470	$	11.63
Vested and exercisable, March 31, 2022	5,248	$	12.43
Vested and expected to vest, March 31, 2022	10,052	$	11.67

As of March 31, 2022, there were approximately $38.0 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 3.2 years.

RSUs

The following table presents a summary of the Company's outstanding RSU awards granted as of March 31, 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	22	$	15.02
Vested	(31)	$	6.33
Forfeited	(37)	$	10.52
Outstanding, March 31, 2022	2,200	$	9.91
Outstanding, expected to vest	2,024	$	9.91

As of March 31, 2022, there were approximately $16.9 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 3.0 years.

ESPP

As of March 31, 2022, there were 738,315 shares available for future purchases under the ESPP. There were 15,095 and 24,422 shares issued under the ESPP during the three months ended March 31, 2022 and 2021, respectively. Cash proceeds from ESPP purchases were $184 thousand and $121 thousand during the three months ended March 31, 2022 and 2021, respectively.

Summary of Significant Accounting Policies - Share-Based Compensation (Details) - shares	1 Months Ended	3 Months Ended
	Jun. 30, 2016	Mar. 31, 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 (in months)	6 months

Business	3 Months Ended
Business	Mar. 31, 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 during the third quarter of 2022, approximately one year after the enrollment of the last patient plus the time needed for database lock and analysis. 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 currently plans to seek a collaborator for the future development and potential commercialization of these product candidates.

Summary of Significant Accounting Policies (Policies)	3 Months Ended
Summary of Significant Accounting Policies (Policies)	Mar. 31, 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”).
Basis of Consolidation	The consolidated financial statements include the accounts of the Company and its subsidiaries. All intercompany balances and transactions have been eliminated in consolidation. 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 three months ended March 31, 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. Right-of-use lease assets and obligations are recognized at the commencement date based on the present value of remaining lease payments over the lease term. As the Company’s leases do not provide an implicit discount rate, the Company has used an estimated incremental borrowing rate based on the information available at the commencement date in determining the present value of lease payments. ROU lease asset includes any lease payments made prior to commencement and excludes any lease incentives. The lease term may include options to extend or terminate the lease when it is reasonably certain that the Company will exercise that option. Operating lease expense is recognized on a straight-line basis over the lease term, subject to any changes in the lease or expectations regarding the terms. Variable lease costs such as common area costs and property taxes are expensed as incurred. For all office lease agreements the Company combines lease and nonlease components. Leases with an initial term of 12 months or less are not recorded on the Company's 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.

Net Loss Per Common Share - (Tables)

3 Months Ended

Mar. 31, 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 March 31,
	2022		2021
Basic and diluted net loss per common share calculation:
Net loss	$	(34,536)	$	(26,795)
Weighted average common shares outstanding - basic and dilutive	118,755		93,311
Net loss per share of common stock - basic and diluted	$	(0.29)	$	(0.29)

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

The following potentially dilutive securities have been excluded from the computations of diluted weighted average common shares outstanding for the periods presented, as the effect of including such shares would be anti-dilutive:


	Three Months Ended March 31,
	2022	2021
Stock options outstanding	10,470	9,178
Restricted stock units	2,200	2,038
Total	12,670	11,216

Fair Value Measurements

3 Months Ended

Mar. 31, 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.


	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*	$	181,948	$	—	$	—
Investments in U.S. Treasury securities	$	36,030	$	—	$	—
Investments in corporate debt securities	$	—	$	100,210	$	—
Investments in asset-backed securities	$	—	13,515		$	—
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.


	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 months ended March 31, 2022.

Summary of Significant Accounting Policies - Schedule of Weighted-Average Assumptions (Details)	3 Months Ended
	Mar. 31, 2022	Mar. 31, 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	101.00%	113.00%
Risk-free interest rate, minimum	1.38%	0.39%
Risk-free interest rate, maximum	1.63%	0.96%
Expected term of options (years)	5 years 2 months 12 days	5 years 3 months 18 days
Expected dividend yield	0.00%	0.00%

Share-Based Compensation (Tables)

3 Months Ended

Mar. 31, 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 March 31,
	2022		2021
Stock options	$	3,323	$	1,469
Restricted stock units	2,005		792
Employee stock purchase plan	58		31
Total	$	5,386	$	2,292

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 March 31,
	2022		2021
Research and development	$	2,661	$	1,343
General and administrative	2,725		949
Total	$	5,386	$	2,292

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	352	$	16.13
Exercised	(651)	$	2.85
Forfeited	(92)	$	9.58


Outstanding, March 31, 2022	10,470	$	11.63
Vested and exercisable, March 31, 2022	5,248	$	12.43
Vested and expected to vest, March 31, 2022	10,052	$	11.67

Summary of outstanding shares of RSU awards

The following table presents a summary of the Company's outstanding RSU awards granted as of March 31, 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	22	$	15.02
Vested	(31)	$	6.33
Forfeited	(37)	$	10.52
Outstanding, March 31, 2022	2,200	$	9.91
Outstanding, expected to vest	2,024	$	9.91

Fair Value Measurements - Assets and Liabilities Measured at Fair Value on a Recurring Basis (Details) - Recurring basis - USD ($) $ in Thousands	Mar. 31, 2022	Dec. 31, 2021
Quoted prices in active markets for identical assets (Level 1) \| Money market funds
Fair Value Measurements
Investments	$ 181,948	$ 251,488
Quoted prices in active markets for identical assets (Level 1) \| Investments in U.S. Treasury securities
Fair Value Measurements
Investments	36,030	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) \| 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	100,210	82,081
Significant other observable inputs (Level 2) \| Investments in asset-backed securities
Fair Value Measurements
Investments	13,515	15,995
Significant other observable inputs (Level 2) \| Investments in supranational securities
Fair Value Measurements
Investments	4,041	4,041
Significant unobservable inputs (Level 3) \| 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

Net Loss Per Common Share

3 Months Ended

Mar. 31, 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 March 31, 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 March 31,
	2022		2021
Basic and diluted net loss per common share calculation:
Net loss	$	(34,536)	$	(26,795)
Weighted average common shares outstanding - basic and dilutive	118,755		93,311
Net loss per share of common stock - basic and diluted	$	(0.29)	$	(0.29)


	Three Months Ended March 31,
	2022	2021
Stock options outstanding	10,470	9,178
Restricted stock units	2,200	2,038
Total	12,670	11,216

Condensed Unaudited Consolidated Statements of Operations and Comprehensive Loss - USD ($) shares in Thousands, $ in Thousands	3 Months Ended
	Mar. 31, 2022	Mar. 31, 2021
Operating expenses:
Research and development	$ 22,557	$ 18,549
General and administrative	12,113	8,322
Total operating expenses	34,670	26,871
Loss from operations	(34,670)	(26,871)
Interest income	133	77
Other expense, net	1	(1)
Loss before income tax benefit	(34,536)	(26,795)
Income tax benefit	0	0
Net loss	(34,536)	(26,795)
Comprehensive loss	$ (34,840)	$ (26,796)
Net loss per common share:
Basic (in usd per share)	$ (0.29)	$ (0.29)
Diluted (in usd per share)	$ (0.29)	$ (0.29)
Weighted average common shares outstanding:
Basic (in shares)	118,755	93,311
Diluted (in shares)	118,755	93,311

Summary of Significant Accounting Policies - Segment and Geographic Information (Details)	3 Months Ended
	Mar. 31, 2022 segment
Accounting Policies [Abstract]
Number of operating segments	1
Number of reporting segments	1

Share-Based Compensation - Schedule of Allocated Stock-based Compensation Expense (Details) - USD ($) $ in Thousands	3 Months Ended
	Mar. 31, 2022	Mar. 31, 2021
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Stock-based compensation expense	$ 5,386	$ 2,292
Research and development
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Stock-based compensation expense	2,661	1,343
General and administrative
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Stock-based compensation expense	$ 2,725	$ 949

Summary of Significant Accounting Policies

3 Months Ended

Mar. 31, 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 three months ended March 31, 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. Right-of-use lease assets and obligations are recognized at the commencement date based on the present value of remaining lease payments over the lease term. As the Company’s leases do not provide an implicit discount rate, the Company has used an estimated incremental borrowing rate based on the information available at the commencement date in determining the present value of lease payments. ROU lease asset includes any lease payments made prior to commencement and excludes any lease incentives. The lease term may include options to extend or terminate the lease when it is reasonably certain that the Company will exercise that option. Operating lease expense is recognized on a straight-line basis over the lease term, subject to any changes in the lease or expectations regarding the terms. Variable lease costs such as common area costs and property taxes are expensed as incurred. For all office lease agreements the Company combines lease and nonlease components. Leases with an initial term of 12 months or less are not recorded on the Company's 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.


	Three Months Ended March 31,
	2022	2021
Expected common stock price volatility	101%	113%
Risk-free interest rate	1.38%-1.63%	0.39%-0.96%
Expected term of options (years)	5.2	5.3
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.

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, 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)

- Definition

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

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+ Details

- 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.

+ References

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-Name Accounting Standards Codification
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+ Details

Commitments and Contingencies (Details)			3 Months Ended
Commitments and Contingencies (Details)	Jun. 22, 2021	Apr. 23, 2021	Mar. 31, 2022 USD ($) licensedProduct shares	Sep. 08, 2021 USD ($)
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
Contingent consideration arrangements, maximum amount (in shares) \| shares			7,200,000
Contingent consideration arrangements, contingently issuable shares as a percentage of shares outstanding			19.90%
GA Product \| Inception 4
Commitments and contingencies
Clinical and marketing approval milestones			$ 45,000,000
Wet AMD Product \| Inception 4
Commitments and contingencies
Clinical and marketing approval milestones			60,000,000
Archemix \| C5 Licensed Product \| Achievement of specified clinical and regulatory milestones \| Maximum \| License Agreements
Commitments and contingencies
Amount to be paid on achievement of milestone			50,500,000
Archemix \| C5 Licensed Product \| First indication \| Maximum \| License Agreements
Commitments and contingencies
Amount to be paid on achievement of milestone			24,500,000
Archemix \| C5 Licensed Product \| Second and third indication \| Maximum \| License Agreements
Commitments and contingencies
Amount to be paid on achievement of milestone			23,500,000
Archemix \| C5 Licensed Product \| Sustained delivery applications \| Maximum \| License Agreements
Commitments and contingencies
Amount to be paid on achievement of milestone			2,500,000
Archemix \| C5 Licensed Product \| Achievement of specified commercial milestones \| Maximum \| License Agreements
Commitments and contingencies
Amount to be paid on achievement of milestone			22,500,000
University of Florida Research Foundation (UFRF) \| Specified clinical, marketing approval and reimbursement approval milestones with respect to a licensed product \| RHO-adRP License Agreement
Commitments and contingencies
Amount to be paid on achievement of milestone			23,500,000
University of Florida Research Foundation (UFRF) \| Specified commercial sales milestones with respect to a licensed product \| RHO-adRP License Agreement
Commitments and contingencies
Amount to be paid on achievement of milestone			$ 70,000,000
University Of Florida Research Foundation (UFRF) And University Of Pennsylvania (Penn) \| Specified clinical, marketing approval and reimbursement approval milestones with respect to a licensed product \| Research and Development Expense \| BEST1 License Agreement
Commitments and contingencies
Number of licensed products \| licensedProduct			1
University Of Florida Research Foundation (UFRF) And University Of Pennsylvania (Penn) \| Specified commercial sales milestones with respect to a licensed product \| Research and Development Expense \| BEST1 License Agreement
Commitments and contingencies
Number of licensed products \| licensedProduct			1
University Of Florida Research Foundation (UFRF) And University Of Pennsylvania (Penn) \| Primary Licensed Product \| Specified clinical, marketing approval and reimbursement approval milestones with respect to a licensed product \| Research and Development Expense \| BEST1 License Agreement
Commitments and contingencies
Payments for license fees			$ 15,700,000
University Of Florida Research Foundation (UFRF) And University Of Pennsylvania (Penn) \| Primary Licensed Product \| Specified commercial sales milestones with respect to a licensed product \| Research and Development Expense \| BEST1 License Agreement
Commitments and contingencies
Payments for license fees			48,000,000
University Of Florida Research Foundation (UFRF) And University Of Pennsylvania (Penn) \| Other Licensed Product \| Specified clinical, marketing approval and reimbursement approval milestones with respect to a licensed product \| Research and Development Expense \| BEST1 License Agreement
Commitments and contingencies
Payments for license fees			3,100,000
University Of Florida Research Foundation (UFRF) And University Of Pennsylvania (Penn) \| Other Licensed Product \| Specified commercial sales milestones with respect to a licensed product \| Research and Development Expense \| BEST1 License Agreement
Commitments and contingencies
Payments for license fees			9,600,000
University Of Massachusetts (UMass) \| miniCEP290 \| Achievement of specified clinical and regulatory milestones \| License Agreements \| Research and Development Expense
Commitments and contingencies
Payments for license fees			$ 14,750,000
Shares issued for license fees (in shares) \| shares			75,000
University Of Massachusetts (UMass) \| miniCEP290 \| Specified commercial sales milestones with respect to a licensed product \| License Agreements \| Research and Development Expense
Commitments and contingencies
Payments for license fees			$ 48,000,000

Cash, Cash Equivalents and Available for Sale Securities - Schedule of Accumulated Other Comprehensive Income (Details) - USD ($) $ in Thousands	3 Months Ended
	Mar. 31, 2022	Mar. 31, 2021
AOCI Attributable to Parent, Net of Tax [Roll Forward]
Beginning balance	$ 360,528	$ 191,563
Ending balance	333,154	167,188
AOCI, Accumulated Gain (Loss), Debt Securities, Available-for-sale, Parent
AOCI Attributable to Parent, Net of Tax [Roll Forward]
Beginning balance	(90)	3
Current period changes in fair value before reclassifications, net of tax	(304)	(1)
Amounts reclassified from accumulated other comprehensive income, net of tax	0	0
Total other comprehensive loss	(304)	(1)
Ending balance	$ (394)	$ 2

- Definition

+ References

+ Details

Cash, Cash Equivalents and Available for Sale Securities - Schedule of Available for Sale Securities (Details) - USD ($) $ in Thousands	Mar. 31, 2022	Dec. 31, 2021
Debt Securities, Available-for-sale [Line Items]
Amortized Cost	$ 154,190	$ 120,392
Gross Unrealized Gains	0	0
Gross Unrealized Losses	(394)	(90)
Fair Value	153,796	120,302
U.S. Treasury securities
Debt Securities, Available-for-sale [Line Items]
Amortized Cost	36,194	18,201
Gross Unrealized Gains	0	0
Gross Unrealized Losses	(164)	(16)
Fair Value	36,030	18,185
Corporate debt securities
Debt Securities, Available-for-sale [Line Items]
Amortized Cost	100,383	82,138
Gross Unrealized Gains	0	0
Gross Unrealized Losses	(173)	(57)
Fair Value	100,210	82,081
Asset-backed securities
Debt Securities, Available-for-sale [Line Items]
Amortized Cost	13,567	16,009
Gross Unrealized Gains	0	0
Gross Unrealized Losses	(52)	(14)
Fair Value	13,515	15,995
Supranational securities
Debt Securities, Available-for-sale [Line Items]
Amortized Cost	4,046	4,044
Gross Unrealized Gains	0	0
Gross Unrealized Losses	(5)	(3)
Fair Value	$ 4,041	$ 4,041