isee-20210630
0001410939FALSE12/312021Q2P3YP5D00014109392021-01-012021-06-30xbrli:shares00014109392021-07-30iso4217:USD00014109392021-06-3000014109392020-12-31iso4217:USDxbrli:shares00014109392021-04-012021-06-3000014109392020-04-012020-06-3000014109392020-01-012020-06-300001410939us-gaap:PreferredStockMember2020-12-310001410939us-gaap:CommonStockMember2020-12-310001410939us-gaap:AdditionalPaidInCapitalMember2020-12-310001410939us-gaap:RetainedEarningsMember2020-12-310001410939us-gaap:ComprehensiveIncomeMember2020-12-310001410939us-gaap:CommonStockMember2021-01-012021-03-310001410939us-gaap:AdditionalPaidInCapitalMember2021-01-012021-03-3100014109392021-01-012021-03-310001410939us-gaap:RetainedEarningsMember2021-01-012021-03-310001410939us-gaap:ComprehensiveIncomeMember2021-01-012021-03-310001410939us-gaap:PreferredStockMember2021-03-310001410939us-gaap:CommonStockMember2021-03-310001410939us-gaap:AdditionalPaidInCapitalMember2021-03-310001410939us-gaap:RetainedEarningsMember2021-03-310001410939us-gaap:ComprehensiveIncomeMember2021-03-3100014109392021-03-310001410939us-gaap:CommonStockMember2021-04-012021-06-300001410939us-gaap:AdditionalPaidInCapitalMember2021-04-012021-06-300001410939us-gaap:RetainedEarningsMember2021-04-012021-06-300001410939us-gaap:ComprehensiveIncomeMember2021-04-012021-06-300001410939us-gaap:PreferredStockMember2021-06-300001410939us-gaap:CommonStockMember2021-06-300001410939us-gaap:AdditionalPaidInCapitalMember2021-06-300001410939us-gaap:RetainedEarningsMember2021-06-300001410939us-gaap:ComprehensiveIncomeMember2021-06-300001410939us-gaap:PreferredStockMember2019-12-310001410939us-gaap:CommonStockMember2019-12-310001410939us-gaap:AdditionalPaidInCapitalMember2019-12-310001410939us-gaap:RetainedEarningsMember2019-12-3100014109392019-12-310001410939us-gaap:CommonStockMember2020-01-012020-03-310001410939us-gaap:AdditionalPaidInCapitalMember2020-01-012020-03-3100014109392020-01-012020-03-310001410939us-gaap:RetainedEarningsMember2020-01-012020-03-310001410939us-gaap:PreferredStockMember2020-03-310001410939us-gaap:CommonStockMember2020-03-310001410939us-gaap:AdditionalPaidInCapitalMember2020-03-310001410939us-gaap:RetainedEarningsMember2020-03-310001410939us-gaap:ComprehensiveIncomeMember2020-03-3100014109392020-03-310001410939isee:UnderwrittenOfferingMemberus-gaap:CommonStockMember2020-04-012020-06-300001410939isee:UnderwrittenOfferingMemberus-gaap:AdditionalPaidInCapitalMember2020-04-012020-06-300001410939isee:UnderwrittenOfferingMember2020-04-012020-06-300001410939us-gaap:PrivatePlacementMemberus-gaap:CommonStockMember2020-04-012020-06-300001410939us-gaap:PrivatePlacementMemberus-gaap:AdditionalPaidInCapitalMember2020-04-012020-06-300001410939us-gaap:PrivatePlacementMember2020-04-012020-06-300001410939us-gaap:CommonStockMember2020-04-012020-06-300001410939us-gaap:AdditionalPaidInCapitalMember2020-04-012020-06-300001410939us-gaap:RetainedEarningsMember2020-04-012020-06-300001410939us-gaap:PreferredStockMember2020-06-300001410939us-gaap:CommonStockMember2020-06-300001410939us-gaap:AdditionalPaidInCapitalMember2020-06-300001410939us-gaap:RetainedEarningsMember2020-06-300001410939us-gaap:ComprehensiveIncomeMember2020-06-3000014109392020-06-300001410939isee:UnderwrittenOfferingMember2021-01-012021-06-300001410939isee:UnderwrittenOfferingMember2020-01-012020-06-300001410939us-gaap:PrivatePlacementMember2021-01-012021-06-300001410939us-gaap:PrivatePlacementMember2020-01-012020-06-30isee:productCandidate0001410939isee:AdenoAssociatedVirusMediatedGeneTherapyProgramMember2021-06-30isee:segment0001410939srt:MinimumMember2021-01-012021-06-300001410939srt:MaximumMember2021-01-012021-06-30xbrli:pure0001410939us-gaap:EmployeeStockOptionMember2021-04-012021-06-300001410939us-gaap:EmployeeStockOptionMember2020-04-012020-06-300001410939us-gaap:EmployeeStockOptionMember2021-01-012021-06-300001410939us-gaap:EmployeeStockOptionMember2020-01-012020-06-300001410939us-gaap:EmployeeStockMembersrt:MaximumMember2016-04-300001410939us-gaap:EmployeeStockMember2016-04-012016-04-300001410939isee:UnderwrittenOfferingMember2020-06-012020-06-300001410939isee:UnderwrittenOfferingMember2020-06-300001410939isee:UnderwrittenOfferingUnderwritersMember2020-06-3000014109392020-06-012020-06-300001410939us-gaap:PrivatePlacementMember2020-06-300001410939isee:PrivatePlacementUnderwritersMember2020-06-300001410939us-gaap:PrivatePlacementMember2020-06-012020-06-300001410939isee:EmployeeAndNonemployeeStockOptionMember2021-04-012021-06-300001410939isee:EmployeeAndNonemployeeStockOptionMember2020-04-012020-06-300001410939isee:EmployeeAndNonemployeeStockOptionMember2021-01-012021-06-300001410939isee:EmployeeAndNonemployeeStockOptionMember2020-01-012020-06-300001410939us-gaap:RestrictedStockMember2021-04-012021-06-300001410939us-gaap:RestrictedStockMember2020-04-012020-06-300001410939us-gaap:RestrictedStockMember2021-01-012021-06-300001410939us-gaap:RestrictedStockMember2020-01-012020-06-300001410939us-gaap:USTreasurySecuritiesMember2021-06-300001410939us-gaap:CorporateDebtSecuritiesMember2021-06-300001410939us-gaap:AssetBackedSecuritiesMember2021-06-300001410939us-gaap:AccumulatedNetUnrealizedInvestmentGainLossMember2021-03-310001410939us-gaap:AccumulatedNetUnrealizedInvestmentGainLossMember2020-03-310001410939us-gaap:AccumulatedNetUnrealizedInvestmentGainLossMember2020-12-310001410939us-gaap:AccumulatedNetUnrealizedInvestmentGainLossMember2019-12-310001410939us-gaap:AccumulatedNetUnrealizedInvestmentGainLossMember2021-04-012021-06-300001410939us-gaap:AccumulatedNetUnrealizedInvestmentGainLossMember2020-04-012020-06-300001410939us-gaap:AccumulatedNetUnrealizedInvestmentGainLossMember2021-01-012021-06-300001410939us-gaap:AccumulatedNetUnrealizedInvestmentGainLossMember2020-01-012020-06-300001410939us-gaap:AccumulatedNetUnrealizedInvestmentGainLossMember2021-06-300001410939us-gaap:AccumulatedNetUnrealizedInvestmentGainLossMember2020-06-300001410939isee:StockIncentive2013PlanMember2013-08-312021-06-300001410939isee:StockIncentive2013PlanMember2021-01-012021-06-300001410939isee:StockIncentive2013PlanMember2021-06-300001410939us-gaap:EmployeeStockOptionMemberisee:TwoThousandNineteenInducementPlanMember2019-10-310001410939us-gaap:EmployeeStockOptionMemberisee:TwoThousandNineteenInducementPlanMember2020-03-012020-03-310001410939us-gaap:EmployeeStockOptionMemberisee:TwoThousandNineteenInducementPlanMember2021-02-012021-02-280001410939us-gaap:EmployeeStockOptionMemberisee:TwoThousandNineteenInducementPlanMember2021-06-300001410939us-gaap:RestrictedStockUnitsRSUMember2021-04-012021-06-300001410939us-gaap:RestrictedStockUnitsRSUMember2020-04-012020-06-300001410939us-gaap:RestrictedStockUnitsRSUMember2021-01-012021-06-300001410939us-gaap:RestrictedStockUnitsRSUMember2020-01-012020-06-300001410939isee:StockIncentive2013PlanMember2021-04-012021-06-300001410939isee:StockIncentive2013PlanMember2020-04-012020-06-300001410939isee:StockIncentive2013PlanMember2021-01-012021-06-300001410939isee:StockIncentive2013PlanMember2020-01-012020-06-300001410939us-gaap:ResearchAndDevelopmentExpenseMember2021-04-012021-06-300001410939us-gaap:ResearchAndDevelopmentExpenseMember2020-04-012020-06-300001410939us-gaap:ResearchAndDevelopmentExpenseMember2021-01-012021-06-300001410939us-gaap:ResearchAndDevelopmentExpenseMember2020-01-012020-06-300001410939us-gaap:GeneralAndAdministrativeExpenseMember2021-04-012021-06-300001410939us-gaap:GeneralAndAdministrativeExpenseMember2020-04-012020-06-300001410939us-gaap:GeneralAndAdministrativeExpenseMember2021-01-012021-06-300001410939us-gaap:GeneralAndAdministrativeExpenseMember2020-01-012020-06-300001410939us-gaap:EmployeeStockOptionMember2020-12-310001410939us-gaap:EmployeeStockOptionMember2021-06-300001410939us-gaap:RestrictedStockMember2020-12-310001410939us-gaap:RestrictedStockMember2021-01-012021-06-300001410939us-gaap:RestrictedStockMember2021-06-300001410939us-gaap:EmployeeStockMemberisee:StockIncentive2013PlanMember2021-06-300001410939us-gaap:EmployeeStockMember2021-04-012021-06-300001410939us-gaap:EmployeeStockMember2020-04-012020-06-300001410939us-gaap:EmployeeStockMember2021-01-012021-06-300001410939us-gaap:EmployeeStockMember2020-01-012020-06-300001410939isee:CARESActMember2021-04-300001410939us-gaap:MoneyMarketFundsMemberus-gaap:FairValueInputsLevel1Memberus-gaap:FairValueMeasurementsRecurringMember2021-06-300001410939us-gaap:FairValueInputsLevel2Memberus-gaap:MoneyMarketFundsMemberus-gaap:FairValueMeasurementsRecurringMember2021-06-300001410939us-gaap:MoneyMarketFundsMemberus-gaap:FairValueInputsLevel3Memberus-gaap:FairValueMeasurementsRecurringMember2021-06-300001410939us-gaap:USTreasurySecuritiesMemberus-gaap:FairValueInputsLevel1Memberus-gaap:FairValueMeasurementsRecurringMember2021-06-300001410939us-gaap:FairValueInputsLevel2Memberus-gaap:USTreasurySecuritiesMemberus-gaap:FairValueMeasurementsRecurringMember2021-06-300001410939us-gaap:USTreasurySecuritiesMemberus-gaap:FairValueInputsLevel3Memberus-gaap:FairValueMeasurementsRecurringMember2021-06-300001410939us-gaap:CorporateDebtSecuritiesMemberus-gaap:FairValueInputsLevel1Memberus-gaap:FairValueMeasurementsRecurringMember2021-06-300001410939us-gaap:FairValueInputsLevel2Memberus-gaap:CorporateDebtSecuritiesMemberus-gaap:FairValueMeasurementsRecurringMember2021-06-300001410939us-gaap:CorporateDebtSecuritiesMemberus-gaap:FairValueInputsLevel3Memberus-gaap:FairValueMeasurementsRecurringMember2021-06-300001410939us-gaap:AssetBackedSecuritiesMemberus-gaap:FairValueInputsLevel1Memberus-gaap:FairValueMeasurementsRecurringMember2021-06-300001410939us-gaap:FairValueInputsLevel2Memberus-gaap:AssetBackedSecuritiesMemberus-gaap:FairValueMeasurementsRecurringMember2021-06-300001410939us-gaap:AssetBackedSecuritiesMemberus-gaap:FairValueInputsLevel3Memberus-gaap:FairValueMeasurementsRecurringMember2021-06-300001410939us-gaap:MoneyMarketFundsMemberus-gaap:FairValueInputsLevel1Memberus-gaap:FairValueMeasurementsRecurringMember2020-12-310001410939us-gaap:FairValueInputsLevel2Memberus-gaap:MoneyMarketFundsMemberus-gaap:FairValueMeasurementsRecurringMember2020-12-310001410939us-gaap:MoneyMarketFundsMemberus-gaap:FairValueInputsLevel3Memberus-gaap:FairValueMeasurementsRecurringMember2020-12-310001410939us-gaap:USTreasurySecuritiesMemberus-gaap:FairValueInputsLevel1Memberus-gaap:FairValueMeasurementsRecurringMember2020-12-310001410939us-gaap:FairValueInputsLevel2Memberus-gaap:USTreasurySecuritiesMemberus-gaap:FairValueMeasurementsRecurringMember2020-12-310001410939us-gaap:USTreasurySecuritiesMemberus-gaap:FairValueInputsLevel3Memberus-gaap:FairValueMeasurementsRecurringMember2020-12-310001410939us-gaap:CorporateDebtSecuritiesMemberus-gaap:FairValueInputsLevel1Memberus-gaap:FairValueMeasurementsRecurringMember2020-12-310001410939us-gaap:FairValueInputsLevel2Memberus-gaap:CorporateDebtSecuritiesMemberus-gaap:FairValueMeasurementsRecurringMember2020-12-310001410939us-gaap:CorporateDebtSecuritiesMemberus-gaap:FairValueInputsLevel3Memberus-gaap:FairValueMeasurementsRecurringMember2020-12-310001410939us-gaap:AssetBackedSecuritiesMemberus-gaap:FairValueInputsLevel1Memberus-gaap:FairValueMeasurementsRecurringMember2020-12-310001410939us-gaap:FairValueInputsLevel2Memberus-gaap:AssetBackedSecuritiesMemberus-gaap:FairValueMeasurementsRecurringMember2020-12-310001410939us-gaap:AssetBackedSecuritiesMemberus-gaap:FairValueInputsLevel3Memberus-gaap:FairValueMeasurementsRecurringMember2020-12-310001410939isee:ArchemixCorpMemberisee:AchievementOfSpecifiedClinicalAndRegulatoryMilestonesMembersrt:MaximumMemberus-gaap:LicenseAgreementTermsMemberisee:C5LicensedProductsMember2011-09-300001410939isee:ArchemixCorpMemberisee:FirstIndicationMembersrt:MaximumMemberus-gaap:LicenseAgreementTermsMemberisee:C5LicensedProductsMember2011-09-300001410939isee:ArchemixCorpMembersrt:MaximumMemberus-gaap:LicenseAgreementTermsMemberisee:C5LicensedProductsMemberisee:SecondandThirdIndicationMember2011-09-300001410939isee:ArchemixCorpMembersrt:MaximumMemberisee:SustainedDeliveryApplicationsMemberus-gaap:LicenseAgreementTermsMemberisee:C5LicensedProductsMember2011-09-300001410939isee:ArchemixCorpMembersrt:MaximumMemberus-gaap:LicenseAgreementTermsMemberisee:AchievementOfSpecifiedCommercialMilestonesMemberisee:C5LicensedProductsMember2011-09-300001410939isee:UniversityofFloridaResearchFoundationUFRFMemberisee:RHOadRPLicenseAgreementMemberisee:SpecifiedClinicalMarketingApprovalandReimbursementApprovalMilestonesRelatedtoaLicensedProductMember2018-12-310001410939isee:UniversityofFloridaResearchFoundationUFRFMemberisee:RHOadRPLicenseAgreementMemberisee:SpecifiedCommercialSalesMilestonesRelatedtoaLicensedProductMember2018-12-310001410939isee:UniversityOfFloridaResearchFoundationUFRFAndUniversityOfPennsylvaniaPennMemberisee:PrimaryLicensedProductMemberus-gaap:ResearchAndDevelopmentExpenseMemberisee:SpecifiedClinicalMarketingApprovalandReimbursementApprovalMilestonesRelatedtoaLicensedProductMemberisee:BEST1LicenseAgreementMember2019-05-012019-05-31isee:licensedProduct00014109392019-05-012019-05-310001410939isee:UniversityOfFloridaResearchFoundationUFRFAndUniversityOfPennsylvaniaPennMemberus-gaap:ResearchAndDevelopmentExpenseMemberisee:OtherLicensedProductMemberisee:SpecifiedClinicalMarketingApprovalandReimbursementApprovalMilestonesRelatedtoaLicensedProductMemberisee:BEST1LicenseAgreementMember2019-05-012019-05-310001410939isee:UniversityOfFloridaResearchFoundationUFRFAndUniversityOfPennsylvaniaPennMemberisee:PrimaryLicensedProductMemberus-gaap:ResearchAndDevelopmentExpenseMemberisee:SpecifiedCommercialSalesMilestonesRelatedtoaLicensedProductMemberisee:BEST1LicenseAgreementMember2019-05-012019-05-310001410939isee:UniversityOfFloridaResearchFoundationUFRFAndUniversityOfPennsylvaniaPennMemberus-gaap:ResearchAndDevelopmentExpenseMemberisee:OtherLicensedProductMemberisee:SpecifiedCommercialSalesMilestonesRelatedtoaLicensedProductMemberisee:BEST1LicenseAgreementMember2019-05-012019-05-310001410939isee:UniversityOfMassachusettsUMassMemberisee:MiniCEP290Memberus-gaap:ResearchAndDevelopmentExpenseMemberisee:AchievementOfSpecifiedClinicalAndRegulatoryMilestonesMemberus-gaap:LicenseAgreementTermsMember2019-05-012019-05-310001410939isee:UniversityOfMassachusettsUMassMemberisee:MiniCEP290Memberus-gaap:ResearchAndDevelopmentExpenseMemberus-gaap:LicenseAgreementTermsMemberisee:SpecifiedCommercialSalesMilestonesRelatedtoaLicensedProductMember2019-05-012019-05-310001410939isee:Inception4Member2018-10-300001410939isee:GAProductMemberisee:Inception4Member2018-10-302018-10-300001410939isee:Inception4Memberisee:WetAMDProductMember2018-10-302018-10-300001410939isee:Inception4Member2018-10-302018-10-3000014109392021-04-232021-04-2300014109392021-06-222021-06-220001410939isee:PublicStockOfferingMemberus-gaap:SubsequentEventMember2021-07-012021-07-310001410939us-gaap:OverAllotmentOptionMemberus-gaap:SubsequentEventMember2021-07-012021-07-310001410939isee:PublicStockOfferingMemberus-gaap:SubsequentEventMember2021-07-300001410939us-gaap:OverAllotmentOptionMemberus-gaap:SubsequentEventMember2021-07-300001410939us-gaap:SubsequentEventMember2021-07-012021-07-31
Table of Contents                                 
UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
WASHINGTON, D.C. 20549
FORM 10-Q
(Mark One)  
 QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934
For the quarterly period ended June 30, 2021
Or
 TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934
For the transition period from            to         

Commission file number: 001-36080
IVERIC bio, Inc.
(Exact name of registrant as specified in its charter)
Delaware 20-8185347
(State or other jurisdiction of incorporation or organization)(I.R.S. Employer Identification No.)
Five Penn Plaza Suite 2372 10001
New York, NY(Zip Code)
(Address of principal executive offices)
(212845-8200
(Registrant's telephone number, including area code)

Securities registered pursuant to Section 12(b) of the Act:
Title of each classTrading Symbol(s)Name of each exchange on which registered
Common Stock, $0.001 par value per shareISEEThe Nasdaq Global Select Market
Indicate by check mark whether the registrant (1) has filed all reports required to be filed by Section 13 or 15(d) of the Securities Exchange Act of 1934 during the preceding 12 months (or for such shorter period that the registrant was required to file such reports), and (2) has been subject to such filing requirements for the past 90 days.  Yes     No
Indicate by check mark whether the registrant has submitted electronically every Interactive Data File required to be submitted pursuant to Rule 405 of Regulation S-T (§232.405 of this chapter) during the preceding 12 months (or for such shorter period that the registrant was required to submit such files).  Yes     No
Indicate by check mark whether the registrant is a large accelerated filer, an accelerated filer, a non-accelerated filer, a smaller reporting company, or an emerging growth company. See the definitions of “large accelerated filer,” “accelerated filer,” “smaller reporting company,” and “emerging growth company” in Rule 12b-2 of the Exchange Act.
Large accelerated filerAccelerated filerNon-accelerated filerSmaller reporting companyEmerging growth company
If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act.
Indicate by check mark whether the registrant is a shell company (as defined in Rule 12b-2 of the Exchange Act).  Yes     No
As of July 30, 2021 there were 104,009,398 shares of Common Stock, $0.001 par value per share, outstanding.





TABLE OF CONTENTS

i

Table of Contents                                 
FORWARD-LOOKING STATEMENTS
This Quarterly Report on Form 10-Q contains forward-looking statements that involve substantial risks and uncertainties. All statements, other than statements of historical facts, contained in this Quarterly Report on Form 10-Q, including statements regarding our strategy, future operations, future financial position, future revenues, projected costs, prospects, plans and objectives of management, are forward-looking statements. The words “anticipate,” “believe,” “goals,” “estimate,” “expect,” “intend”, “may,” “might,” “plan,” “predict,” “project,” “target,” “potential,” “will,” “would,” “could,” “should,” “continue” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words.
The forward-looking statements in this Quarterly Report on Form 10-Q include, among other things, statements about:
the potential benefits of our business plan and strategy to develop our therapeutic and gene therapy product candidates and pursue our gene therapy research programs;
our expectations regarding the impact of results from GATHER1, our completed Phase 3 clinical trial evaluating Zimura for the treatment of Geographic Atrophy (GA) secondary to age-related macular degeneration, on our business and regulatory strategy, including our plans to pursue further development of Zimura in GA and earlier forms of dry age-related macular degeneration;
the timing, costs, conduct and outcome of GATHER2, our ongoing Phase 3 clinical trial evaluating Zimura for the treatment of GA secondary to age-related macular degeneration, including expectations regarding receipt of top-line data from the trial and regarding patient retention, and expectations regarding the potential for Zimura to receive regulatory approval for the treatment of GA secondary to age-related macular degeneration based on the clinical trial results we have received to date and the future results from the GATHER2 clinical trial and any other trials we or a potential collaborator may conduct;
our plans and expectations for developing Zimura in drusen secondary to age-related macular degeneration (drusen) and potentially other earlier forms of dry age-related macular degeneration, and pursuing lifecycle improvement programs for Zimura;
the timing, costs, conduct and outcome of STAR, our ongoing Phase 2b screening trial evaluating Zimura for the treatment of autosomal recessive Stargardt disease, including expectations regarding the recruitment of additional patients for this trial;
our ability to establish and maintain arrangements and capabilities for the manufacture of our therapeutic and gene therapy product candidates, including scale up and validation of the manufacturing process for Zimura drug substance and securing the supply of Zimura drug product and the polyethylene glycol (PEG) starting material for our expected needs;
the actual and expected effects of the COVID-19 pandemic and related response measures on our business and operations, including the timing, costs, conduct and outcome of our research and development programs, the work and well-being of our employees, and our financial position;
our expectations related to our use of available cash;
our estimates regarding expenses, future revenues, capital requirements and needs for, and ability to obtain, additional financing;
our plans and ability to consummate business development transactions, including potential collaboration or out-licensing opportunities for further development and potential commercialization of our product candidates, and in-licenses or other opportunities to acquire rights to additional product candidates or technologies to treat retinal diseases, including as part of lifecycle management programs for Zimura;
the timing, costs, conduct and outcome of our ongoing and planned clinical trials, including our strategy to conduct a Phase 1/2 clinical trial for IC-200 focusing on autosomal recessive bestrophinopathy, statements regarding the timing of the initiation and completion of, and the receipt of results from, such clinical trials, the costs to conduct such clinical trials, and the impact of the results of such clinical trials on our business strategy;
1

Table of Contents                                 
the timing, costs, conduct and outcome of our ongoing and planned research and preclinical development activities, including statements regarding the timing of the initiation and completion of, and the receipt of results from, such activities, the costs to conduct such activities, and the impact of the results of such activities on our business strategy;
the timing of and our ability to submit investigational new drug applications for, and to obtain marketing approval of our product candidates, and the ability of our product candidates to meet existing or future regulatory standards;
the potential advantages of our product candidates and other technologies that we are pursuing, including the advantages and limitations of inhibition of complement factor C5 and HtrA1, including our hypotheses regarding complement inhibition and HtrA1 inhibition as potentially relevant mechanisms of action to treat GA and other forms or stages of age-related macular degeneration, and of gene therapy, including the use of minigenes;
our estimates regarding the number of patients affected by the diseases our product candidates and development programs are intended to treat;
our estimates regarding the potential market opportunity for our product candidates;
our sales, marketing and distribution capabilities and strategy;
the rate and degree of potential market acceptance and clinical utility of our product candidates, if approved;
the potential receipt of revenues from future sales of our product candidates, if approved;
our personnel and human capital resources;
our intellectual property position;
the impact of existing and new governmental laws and regulations; and
our competitive position.
We may not actually achieve the plans, intentions or expectations disclosed in our forward-looking statements, and our stockholders should not place undue reliance on our forward-looking statements. Actual results or events could differ materially from the plans, intentions and expectations disclosed in the forward-looking statements we make. We have included important factors in the cautionary statements included in this Quarterly Report on Form 10-Q, particularly in the “Risk Factors” section, that could cause actual results or events to differ materially from the forward-looking statements that we make. Our forward-looking statements do not reflect the potential impact of any future acquisitions, mergers, licenses, dispositions, joint ventures or investments we may make.
You should read this Quarterly Report on Form 10-Q and the documents that we have filed as exhibits to this Quarterly Report on Form 10-Q and our other periodic reports, completely and with the understanding that our actual future results may be materially different from what we expect. The forward-looking statements contained in this Quarterly Report on Form 10-Q are made as of the date of this Quarterly Report on Form 10-Q, and we do not assume any obligation to update any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by applicable law.
This Quarterly Report on Form 10-Q includes statistical and other industry and market data that we obtained from industry publications and research, surveys and studies conducted by third parties. Industry publications and third-party research, surveys and studies generally indicate that their information has been obtained from sources believed to be reliable, although they do not guarantee the accuracy or completeness of such information.
Summary of Principal Risk Factors
The following is a summary of the principal factors that make an investment in our company speculative or risky. This summary does not address all of the risks and uncertainties that we face. Additional risk and uncertainties not presently known to us or that we presently deem less significant may also impair our business operations. Additional discussion of the risks summarized in this summary, and other risks that we face, can be found in Part II, Item 1A. Risk Factors section of this Quarterly Report on
Form 10-Q, and should be carefully considered, together with other information in this Quarterly Report on Form 10-Q and
our other filings with the Securities Exchange Commission, before making an investment decision regarding our common
stock. The forward-looking statements discussed above are qualified by these risk factors. If any of the following risks occur,
our business, financial condition, results of operations and future growth prospects could be materially and adversely affected.
2

Table of Contents                                 

1.We are a development-stage company without any approved products. The value of your investment is highly dependent on the success of our research and development programs, which carry numerous risks.

2.We have had a history of operating at significant losses and expect to continue to do so until we can successfully commercialize one or more of our product candidates, if ever. We may never achieve profitability.

3.We may need additional financing in order to finish developing and start commercializing one or more of our product candidates, if approved. Securing financing may be challenging and/or dilutive to our shareholders, and if we are unable to secure financing when needed, we may need to curtail our development programs or planned commercialization activities.

4.The COVID-19 pandemic has adversely affected our business, for example, by impacting the initiation and conduct of our clinical trials, the work of our academic collaborators and contract manufacturing organizations, and aspects of our supply chain. Because of the ongoing and fluid nature of the pandemic, it will continue to affect our business.

5.Drug development is inherently risky with numerous scientific, technical, regulatory and other challenges. A promising drug candidate can fail at any time and for any number of reasons.

6.We are pursuing the development of our product candidates using novel mechanisms of action targeting indications for which there are no approved products. These include, for example, complement inhibition and inhibition of High temperature requirement A serine peptidase 1 protein for GA; complement inhibition for drusen and autosomal recessive Stargardt disease; and gene therapy for autosomal recessive bestrophinopathy and other BEST1-related inherited retinal diseases and rhodopsin-mediated autosomal dominant retinitis pigmentosa. These approaches carry numerous scientific, regulatory and other risks.

7.Regulatory authorities, including the U.S. Food and Drug Administration, or FDA, and the European Medicines Agency, or EMA, may disagree with the design of or our conclusions from the GATHER1 trial. Since receipt of the 12-month results from GATHER1, we have not had any formal interactions with the EMA regarding our planned regulatory pathway for Zimura in GA and the EMA and other regulatory authorities may disagree with the requirements of the FDA. The results of the GATHER2 trial may not replicate the results of the GATHER1 trial. We may need to conduct additional clinical trials or nonclinical studies for Zimura in order to obtain marketing approval.

8.Gene therapy is a novel area of disease treatment that is subject to numerous risks and uncertainties. We have no prior experience conducting clinical development of a gene therapy product.

9.We may discover safety issues with our product candidates due to known and currently unknown factors, which could hamper their further development.

10.Manufacturing our product candidates, including our gene therapies, is technically complex, expensive and time consuming. We may face issues with scaling up and validating the manufacturing process for Zimura. We may not be able to secure adequate supply of Zimura drug product and the PEG starting material for our future needs, including potential commercial launch. Issues with manufacturing can derail the further development or commercialization of our product candidates.

11.We face substantial competition from large pharmaceutical companies, smaller biotech companies and others.

12.To commercialize any of our product candidates, if approved, we will need to set up a sales and marketing infrastructure. The success of our commercialization efforts will depend in part on the degree of acceptance of our product candidates by patients, the medical community and payors.

13.We do not have any internal manufacturing facilities and rely heavily on our third-party contract manufacturers. They may have different business priorities than we do and may fail to meet our expectations or follow regulatory requirements, including current good manufacturing practices requirements.

14.We rely heavily on our third-party contract research organizations as well as our clinical trial sites and academic collaborators. They may have different priorities than we do and may fail to follow regulatory requirements, including good laboratory practice, good clinical practice and other data integrity requirements.

3

Table of Contents                                 
15.If we choose to pursue a collaboration or out-license for the further development and potential commercialization of any of our product candidates, we may not be able to enter into a collaboration or out-license on favorable terms, or at all. Even if we are able to do so, the collaboration or out-license may not be successful.

16.We rely on patents to protect our proprietary position. We may not obtain the patent rights that we seek and/or we may not be able to exclude our competitors from relevant markets. We may be subject to litigation involving our patents or those of third parties.

17.We are highly dependent on our information security systems and those of third parties we work with. A cybersecurity incident may cause interruptions to the progress of our development programs and operations, financial or regulatory penalties and/or harm to our reputation.

18.We rely on a limited number of employees to conduct our operations, including supervising our outside vendors. The skills needed to advance our research and development programs and plan for eventual commercialization of our product candidates are highly specialized. We plan to hire additional qualified personnel, including commercialization personnel, to support the growth of our business. Hiring these personnel and retaining existing employees may be challenging.

19.We need to satisfy numerous regulatory requirements in order to secure marketing approval and reimbursement approval, if applicable, for any of our product candidates. These requirements differ across jurisdictions. Failure to satisfy and maintain those requirements can preclude us from commercializing our products.

20.We and any commercialization partners are subject to numerous healthcare laws and regulations governing our relationships with patients, healthcare professionals and third-party payors. Failure to comply with these requirements may adversely affect our business, including as we prepare for potential commercialization of Zimura.

21.The reimbursement and payment regime for pharmaceutical products in the United States remains in flux, including as a result of the implementation of and litigation involving the Affordable Care Act. There are ongoing, and often bipartisan, efforts to reduce the prices of pharmaceutical products.
USE OF TRADEMARKS
    The trademarks, trade names and service marks appearing in this Quarterly Report on Form 10-Q are the property of their respective owners. We have omitted the ® and ™ designations, as applicable, for the trademarks named in this Quarterly Report on Form 10-Q after their first reference in this Quarterly Report on Form 10-Q.
4

Table of Contents                                 
PART I—FINANCIAL INFORMATION
Item 1. Financial Statements
IVERIC bio, Inc.
Condensed Unaudited Consolidated Balance Sheets
(in thousands, except share and per share data)
 June 30, 2021December 31, 2020
Assets  
Current assets  
Cash and cash equivalents$50,363 $66,373 
Available for sale securities109,519 143,674 
Prepaid expenses and other current assets3,806 4,791 
Income tax receivable 1,765 
Total current assets163,688 216,603 
Property and equipment, net8 26 
Right-of-use asset, net1,971 120 
Other assets 5 
Total assets$165,667 $216,754 
Liabilities and Stockholders' Equity   
Current liabilities  
Accrued research and development expenses$15,412 $12,284 
Accounts payable and accrued expenses8,643 12,792 
Lease liability937 54 
Total current liabilities24,992 25,130 
Lease liability, non-current1,102 61 
Total liabilities26,094 25,191 
Stockholders' equity  
Preferred stock—$0.001 par value, 5,000,000 shares authorized, no shares issued or outstanding
  
Common stock—$0.001 par value, 200,000,000 shares authorized, 90,386,770 and 90,120,797 shares issued and outstanding at June 30, 2021 and December 31, 2020, respectively
90 90 
Additional paid-in capital761,491 756,543 
Accumulated deficit(622,011)(565,073)
Accumulated other comprehensive income3 3 
Total stockholders' equity139,573 191,563 
Total liabilities and stockholders' equity$165,667 $216,754 
   
The accompanying unaudited notes are an integral part of these financial statements.
3

Table of Contents                                 
IVERIC bio, Inc.
Condensed Unaudited Consolidated Statements of Operations and Comprehensive Loss
(in thousands, except per share data)
 Three Months Ended June 30,Six Months Ended June 30,
 2021202020212020
Operating expenses:   
Research and development$23,488 $12,720 $42,037 $26,470 
General and administrative6,718 6,289 15,040 11,287 
Total operating expenses30,206 19,009 57,077 37,757 
Loss from operations(30,206)(19,009)(57,077)(37,757)
Interest income65 46 142 404 
Other expense, net(2)(12)(3)(7)
Loss before income tax benefit(30,143)(18,975)(56,938)(37,360)
Income tax benefit 386  3,695 
Net loss$(30,143)$(18,589)$(56,938)$(33,665)
Comprehensive loss$(30,142)$(18,589)$(56,938)$(33,665)
Net loss per common share:  
Basic and diluted$(0.32)$(0.32)$(0.61)$(0.61)
Weighted average common shares outstanding:
Basic and diluted93,409 57,421 93,382 55,424 
   
The accompanying unaudited notes are an integral part of these financial statements.

4

Table of Contents                                 
IVERIC bio, Inc.
Condensed Unaudited Consolidated Statements of Stockholders' Equity
(in thousands)
 Preferred StockCommon StockAdditional
paid-in
capital
Accumulated
Deficit
Accumulated
Other
Comprehensive
Income (Loss)
Total
 SharesAmountSharesAmount
Balance at December 31, 2020 $ 90,121 $90 $756,543 $(565,073)$3 $191,563 
Issuance of common stock under employee stock compensation plans— — 49 — 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 March 31, 2021 $ 90,170 $90 $758,964 $(591,868)$2 $167,188 
Issuance of common stock under employee stock compensation plans — — 217 — 448 — — 448 
Share-based compensation— — — — 2,079 — — 2,079 
Net loss— — — — — (30,143)— (30,143)
Unrealized loss on available for sale securities, net of tax— $— — $— $— $— $1 $1 
Balance at June 30, 2021 $ 90,387 $90 $761,491 $(622,011)$3 $139,573 
 Preferred StockCommon StockAdditional
paid-in
capital
Accumulated
Deficit
Accumulated
Other
Comprehensive
Income (Loss)
Total
 SharesAmountSharesAmount
Balance at December 31, 2019 $ 49,627 $50 $597,679 $(480,526)$— $117,203 
Issuance of common stock under employee stock compensation plans— — 105 — 179 — — 179 
Share-based compensation— — — — 2,324 — — 2,324 
Net loss— — — — — (15,076)— (15,076)
Balance at March 31, 2020 $ 49,732 $50 $600,182 $(495,602)$ $104,630 
Issuance of common stock and pre-funded warrants through underwritten offering, net of issuance costs— — 28,503 28 116,855 — — 116,883 
Issuance of common stock in connection with private offering, net of issuance costs.— — 8,649 9 33,228 — — 33,237 
Issuance of common stock under the exercise of pre-funded warrants— — 2,500 2 (2)— —  
Issuance of common stock under employee stock compensation plans— — 105 — 7 — — 7 
Share-based compensation— — — — 1,873 — — 1,873 
Net loss— — — — — (18,589)— (18,589)
Balance at Balance at June 30, 2020 $ 89,489 $89 $752,143 $(514,191)$ $238,041 

The accompanying unaudited notes are an integral part of these financial statements.
5

Table of Contents                                 
IVERIC bio, Inc.
Condensed Unaudited Consolidated Statements of Cash Flows
(in thousands)
 Six Months Ended June 30,
 20212020
Operating Activities  
Net loss$(56,938)$(33,665)
Adjustments to reconcile net loss to net cash used in operating activities  
Depreciation and other expense18 77 
Amortization of premium and discounts on investment securities742  
Share-based compensation4,371 4,197 
Changes in operating assets and liabilities:  
Income tax receivable1,765  
Prepaid expense and other assets990 (155)
Accrued interest receivable92  
Accrued research and development expenses3,128 271 
Accounts payable and accrued expenses(4,149)(995)
Change in working capital73  
Net cash used in operating activities(49,908)(30,270)
Investing Activities  
Purchase of marketable securities(39,314) 
Maturities of marketable securities72,635  
Net cash provided by investing activities33,321  
Financing Activities  
Proceeds from employee stock plan purchases 577 186 
Proceeds from follow-on public offering, net 116,883 
Proceeds from private-placement, net 33,237 
Net cash provided by financing activities577 150,306 
Net decrease in cash and cash equivalents(16,010)120,036 
Cash and cash equivalents  
Beginning of period66,373 125,699 
End of period$50,363 $245,735 
Supplemental disclosure of cash paid
Income tax refunds received$1,765 $3,327 

   The accompanying unaudited notes are an integral part of these financial statements.
6

Table of Contents                                 
IVERIC bio, Inc.
Notes to Condensed Unaudited Consolidated Financial Statements
(in thousands, except per share data)
1. Business
Description of Business and Organization
IVERIC bio, Inc. (the “Company” or “IVERIC”) is a science-driven biopharmaceutical company focused on the discovery and development of novel treatment options for retinal diseases with significant unmet medical needs. The Company is currently developing both therapeutic product candidates for age-related retinal diseases and gene therapy product candidates for orphan inherited retinal diseases (“IRDs”). The Company believes that both therapeutics and gene therapy serve important roles in drug development and providing potential treatment options for patients suffering from retinal diseases.
The Company's therapeutics portfolio consists of its clinical stage product candidate Zimura® (avacincaptad pegol), a complement C5 inhibitor, and its preclinical product candidate IC-500, a High temperature requirement A serine peptidase 1 protein (“HtrA1”) inhibitor. The Company is currently targeting the following diseases with Zimura:
Geographic Atrophy (“GA”), which is the advanced stage of age-related macular degeneration (“AMD”), and is characterized by marked thinning or atrophy of retinal tissue, leading to irreversible loss of vision;
drusen secondary to AMD (“drusen”), which is an earlier stage of AMD and is characterized by yellow deposits under the retina which may grow larger and more numerous over time and potentially lead to GA and loss of vision; and
autosomal recessive Stargardt disease (“STGD1”), which is an orphan inherited condition characterized by progressive damage to the central portion of the retina (the “macula”) and other retinal tissue, leading to loss of vision.
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 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 top-line data from the GATHER2 trial to become available during the second half of 2022, approximately one year after the enrollment of the last patient plus the time needed for database lock and analysis.
The Company plans to begin clinical development of Zimura for drusen in 2022. The Company is developing IC-500 for GA secondary to AMD and potentially other age-related retinal diseases.
The Company's gene therapy portfolio consists of two product candidates in preclinical development (IC-200 and IC-100) and several ongoing research programs, each of which uses adeno-associated virus (“AAV”) for gene delivery. These AAV mediated gene therapy programs are targeting the following orphan IRDs:
autosomal recessive bestrophinopathy (“ARB”), which is characterized by loss of central vision and abnormal changes in electrophysiology, and other IRDs associated with mutations in the BEST1 gene;
rhodopsin-mediated autosomal dominant retinitis pigmentosa (“RHO-adRP”), which is characterized by progressive and severe bilateral loss of vision leading to blindness;
Leber Congenital Amaurosis type 10 (“LCA10”), which is characterized by severe bilateral loss of vision at or soon after birth;
autosomal recessive Stargardt disease; and
IRDs associated with mutations in the USH2A gene, which include Usher syndrome type 2A and USH2A-associated nonsyndromatic autosomal recessive retinitis pigmentosa.
The Company is developing IC-200 for ARB and other BEST1-related IRDs, and IC-100 for RHO-adRP.
2. Summary of Significant Accounting Policies
The Company’s significant accounting policies are described in Note 2, “Summary of Significant Accounting Policies,” in the notes to the audited consolidated financial statements included in the Company’s Annual Report on Form 10-K (“Annual Report”) for the year ended December 31, 2020 filed with the Securities and Exchange Commission (“SEC”) on March 4, 2021.
7

Table of Contents                                 
Basis of Presentation and Consolidation
In the opinion of management, the Company’s condensed consolidated financial statements include all adjustments, consisting of normal recurring accruals, necessary for a fair statement of the Company’s financial statements for interim periods in accordance with accounting principles generally accepted in the United States (“U.S. GAAP”). The consolidated financial statements include the accounts of the Company and its subsidiaries. All intercompany balances and transactions have been eliminated in consolidation. The information included in this quarterly report on Form 10-Q should be read in conjunction with the Company’s audited consolidated financial statements and the accompanying notes included in the Annual Report.
The year-end condensed consolidated balance sheet data presented for comparative purposes was derived from the Company’s audited financial statements but does not include all disclosures required by U.S. GAAP. The results of operations for the six months ended June 30, 2021 are not necessarily indicative of the operating results for the full year or for any other subsequent interim period.
Segment and Geographic Information
Operating segments are defined as components of an enterprise about which separate discrete information is available for evaluation by the chief operating decision maker, or decision-making group, in deciding how to allocate resources and in assessing performance. The Company views its operations and manages its business in one operating and reportable segment.
Use of Estimates
The preparation of financial statements and related disclosures in conformity with U.S. GAAP requires management to make estimates and judgments that affect the amounts reported in the financial statements and accompanying notes. The Company bases its estimates and judgments on historical experience and on various other assumptions that it believes are reasonable under the circumstances. The amounts of assets and liabilities reported in the Company's Consolidated Balance Sheets and the amount of expenses reported for each of the periods presented are affected by estimates and assumptions, which are used for, but not limited to, accounting for research and development costs, accounting for share-based compensation and accounting for income taxes. Actual results could differ from those estimates.
Cash and Cash Equivalents
The Company considers all highly liquid investments with an original maturity of 90 days or less when purchased to be cash equivalents. The carrying amounts reported in the Balance Sheets for cash and cash equivalents are valued at cost, which approximates their fair value.
Available for Sale Securities
The Company considers securities with original maturities of greater than 90 days to be available for sale securities. Available for sale securities with original maturities of greater than one year are recorded as non-current assets. Available for sale securities are recorded at fair value and unrealized gains and losses are recorded within other comprehensive income.
On a quarterly basis, the Company reviews the status of each security in an unrealized loss position, to evaluate the existence of potential credit losses. The Company first considers whether it intends to sell, or if it is more likely than not that the Company will be required to sell the security before recovery of its amortized cost basis. If either of the criteria regarding intent or requirement to sell is met, the security’s amortized cost basis is written down to fair value through income. For securities that do not meet this criteria, the Company considers a number of factors to determine if the decline in fair value has resulted from credit losses or other factors, including but not limited to: (1) the extent of the decline; (2) changes to the rating of the security by a rating agency; (3) any adverse conditions specific to the security; and (4) other market conditions that may affect the fair value of the security. If this assessment indicates that a credit loss exists and the present value of cash flows expected to be collected is less than the amortized cost basis, an allowance for credit losses is required for the credit loss. Any impairment that has not been recorded through an allowance for credit losses is recognized in other comprehensive income.
Financial Instruments
Cash equivalents and available for sale securities are reflected in the accompanying financial statements at fair value. The carrying amount of accounts payable and accrued expenses, including accrued research and development expenses, approximates fair value due to the short-term nature of those instruments.
Accounting Standards Codification, or ASC 820, Fair Value Measurements and Disclosures, defines fair value as the price that would be received to sell an asset, or paid to transfer a liability, in the principal or most advantageous market in an orderly transaction between market participants on the measurement date. The fair value standard also establishes a three-level
8

Table of Contents                                 
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-200 and IC-100 and also relies on sole-source suppliers for certain starting materials used in the manufacture of such product candidates. The Company currently relies upon a single third-party contract manufacturer to conduct process development, scale-up and GMP manufacture of the drug substance for IC-500 for preclinical toxicology studies and early-stage clinical trials and a single third-party contract manufacturer to conduct fill/finish services for IC-500. If the Company’s third-party manufacturers or fill/finish service providers should become unavailable to the Company for any reason, including as a result of capacity constraints, different business objectives, financial difficulties, insolvency or the COVID-19 pandemic, the Company believes that there are a limited number of potential replacement manufacturers, and the Company likely would incur added costs and delays in identifying or qualifying such replacements.
Foreign Currency Translation
The Company considers the U.S. dollar to be its functional currency. Expenses denominated in foreign currencies are translated at the exchange rate on the date the expense is incurred. The effect of exchange rate fluctuations on translating foreign currency assets and liabilities into U.S. dollars is included in the Consolidated Statements of Operations and Comprehensive Loss. Foreign exchange transaction gains and losses are included in the results of operations and are not material in the Company's financial statements.
9

Table of Contents                                 
Leases
The Company determines if an arrangement contains a lease at inception. For arrangements where the Company is the lessee, it recognizes a right-of-use (“ROU”) asset and operating lease liability on the Company's Consolidated Balance Sheet. ROU lease assets represent the Company's right to use the underlying asset for the lease term and the lease obligation represents the Company's commitment to make the lease payments arising from the lease. Right-of-use lease assets and obligations are recognized at the commencement date based on the present value of remaining lease payments over the lease term. As the Company’s leases do not provide an implicit discount rate, the Company has used an estimated incremental borrowing rate based on the information available at the commencement date in determining the present value of lease payments. ROU lease asset includes any lease payments made prior to commencement and excludes any lease incentives. The lease term may include options to extend or terminate the lease when it is reasonably certain that the Company will exercise that option. Operating lease expense is recognized on a straight-line basis over the lease term, subject to any changes in the lease or expectations regarding the terms. Variable lease costs such as common area costs and property taxes are expensed as incurred. For all office lease agreements the Company combines lease and nonlease components. Leases with an initial term of 12 months or less are not recorded on the Company's Consolidated Balance Sheet.
Property and Equipment
Property and equipment, which consists mainly of clinical 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
10

Table of Contents                                 
achievement. If performance-based milestones are later determined not to be probable of achievement, then all previously recorded stock-based compensation expense associated with such options will be reversed during the period in which the Company makes this determination.
The Company estimates forfeitures at the time of grant and revises those estimates in subsequent periods if actual forfeitures differ from its estimates. The Company uses historical data to estimate pre-vesting forfeitures and record share-based compensation expense only for those awards that are expected to vest. To the extent that actual forfeitures differ from the Company's estimates, the difference is recorded as a cumulative adjustment in the period the estimates were revised.
Stock Options
The Company estimates the fair value of stock options granted to employees, non-employee directors and consultants on the date of grant using the Black-Scholes option-pricing model. The Company's computation of stock-price volatility is based on daily historical volatility during the time period that corresponds to the expected option term. The Company's computation of expected term is determined using the expected term of stock option grants to employees based on an analysis of actual option exercises. The Company utilizes a dividend yield of zero based on the fact that the Company has never paid cash dividends to stockholders and has no current intentions to pay cash dividends. The risk-free interest rate is based on the zero-coupon U.S. Treasury yield at the date of grant for a term equivalent to the expected term of the option.
The weighted-average assumptions used to estimate grant date fair value of stock options using the Black-Scholes option pricing model were as follows for the six month periods ended June 30, 2021 and 2020:
 Three Months Ended June 30,Six Months Ended June 30,
 2021202020212020
Expected common stock price volatility113%118%113%117%
Risk-free interest rate
0.89%-0.94%
0.26%-0.36%
0.31%-0.96%
0.26%-1.34%
Expected term of options (years)5.24.55.14.5
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.
3. Common Stock
In June 2020, the Company completed an underwritten public offering in which the Company sold 28,503,220 shares of its common stock, which includes the exercise in full of the underwriters’ option to purchase additional shares of the Company’s common stock, at a price to the public of $4.100 per share and at a price to the underwriters of $3.854 per share. The Company also sold to certain investors in lieu of common stock, pre-funded warrants to purchase 1,914,280 shares of its common stock at a price to the public of $4.099 per share underlying each pre-funded warrant, and at a price to the underwriters of $3.853 per share underlying each pre-funded warrant.
Concurrently with the June 2020 public offering, the Company completed a private placement in which the Company sold 8,649,453 shares of its common stock to affiliates of Vivo Capital, LLC and Samsara BioCapital, LP, at a sale price equal to the price to the public in the underwritten public offering.
The net proceeds from the public offering and private placement, after deducting underwriting discounts, underwriting and placement agent commissions and other expenses totaling $10.1 million, was approximately $150.1 million.
11

Table of Contents                                 
The Company evaluated the pre-funded warrants for liability or equity classification in accordance with the provisions of ASC 480, Distinguishing Liabilities from Equity, and ASC 815-40, Derivatives and Hedging. Based on the provisions governing the pre-funded warrants in the applicable agreement, the Company determined that the pre-funded warrants meet the criteria required to be classified as an equity award subject to the guidance in ASC 815-10 and 815-40 and should effectively be treated as outstanding common shares in both basic and diluted earnings per share calculations.
4. Net Loss Per Common Share
Basic and diluted net loss per common share is determined by dividing net loss by the weighted average common shares and pre-funded warrants outstanding during the period. Basic and diluted shares outstanding includes the weighted average effect of the Company's outstanding pre-funded warrants as the exercise of such pre-funded warrants requires nominal consideration to be given for the delivery of the corresponding shares of common stock. As of June 30, 2021, the Company had 3,164,280 pre-funded warrants outstanding, which if exercised, would increase the number of shares of common stock issued and outstanding. For the periods when there is a net loss, shares underlying stock options and RSUs have been excluded from the calculation of diluted net loss per common share because the effect of including such shares would be anti-dilutive. Therefore, the weighted average common shares used to calculate both basic and diluted net loss per common share would be the same.
The following table sets forth the computation of basic and diluted net loss per common share for the periods indicated:
 Three Months Ended June 30,Six Months Ended June 30,
 2021202020212020
Basic and diluted net loss per common share calculation:  
Net loss$(30,143)$(18,589)$(56,938)$(33,665)
Weighted average common shares outstanding - basic and dilutive93,409 57,421 93,382 55,424 
Net loss per share of common stock - basic and diluted$(0.32)$(0.32)$(0.61)$(0.61)
The following potentially dilutive securities have been excluded from the computations of diluted weighted average common shares outstanding for the periods presented, as the effect of including such shares would be anti-dilutive:
 Three Months Ended June 30,Six Months Ended June 30,
 2021202020212020
Stock options outstanding9,233 7,155 9,233 7,155 
Restricted stock units1,953 1,636 1,953 1,636 
Total11,186 8,791 11,186 8,791 

5. 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 June 30, 2021 and December 31, 2020, the Company had cash and cash equivalents of approximately $50.4 million and $66.4 million, respectively. Cash and cash equivalents included cash of $2.0 million at June 30, 2021 and $8.4 million at December 31, 2020. Cash and cash equivalents at June 30, 2021 and December 31, 2020 included $48.4 million and $58.0 million, respectively, of investments in money market funds.
The Company considers securities with original maturities of greater than 90 days at the date of purchase to be available for sale securities. As of June 30, 2021 and December 31, 2020, the Company held available for sale securities of $109.5 million and $143.7 million, respectively, all of which have maturities of less than one year.
The Company evaluates securities with unrealized losses, if any, to determine whether the decline in fair value has resulted from credit loss or other factors. The Company has determined that there were no credit losses in fair value of its investments as of June 30, 2021. Factors considered in determining whether a loss resulted from a credit loss or other factors included the length of time and extent to which the investment’s fair value has been less than the cost basis, the financial condition and near-term prospects of the investee, the extent of the loss related to credit of the issuer, the expected cash flows from the security, the Company’s intent to sell the security, and whether or not the Company will be required to sell the security before the recovery of its amortized cost.
12

Table of Contents
The Company classifies these securities as available for sale. However, the Company has not sold and does not currently intend to sell its investments and the Company believes it is more likely than not that the Company will recover the carrying value of these investments.
The Company believes that its existing cash, cash equivalents and available for sale securities as of June 30, 2021 will be sufficient to fund its currently planned capital expenditure requirements and operating expenses for at least the next 12 months from the filing of this Quarterly Report on Form 10-Q. See Note 10 to the Financial Statements for information about the Company’s follow-on public offering completed in July 2021.
Available for sale securities, including carrying value and estimated fair values, are summarized as follows:
 As of June 30, 2021
 Amortized CostGross Unrealized GainsGross Unrealized LossesFair Value
U.S. Treasury securities$27,123 $4 $ $27,127 
Corporate debt securities71,923 1 (2)71,922 
Asset-backed securities10,470   10,470 
Total$109,516 $5 $(2)$109,519 
The Company’s available for sale securities are reported at fair value on the Company’s balance sheet. Unrealized gains (losses) are reported within other comprehensive income in the statements of comprehensive loss. The cost of securities sold and any realized gains/losses from the sale of available for sale securities are based on the specific identification method. The changes in accumulated other comprehensive income associated with the unrealized gain on available for sale securities during the three and six months ended June 30, 2021 and 2020, respectively, were as follows:
 Three months ended June 30,Six Months Ended June 30, 2021
 2021202020212020
Beginning balance$2 $ $3 $ 
Current period changes in fair value before reclassifications, net of tax1    
Amounts reclassified from accumulated other comprehensive income, net of tax    
Total other comprehensive income$1 $   
Ending balance$3 $ $3 $ 
6. Share-Based Compensation
Pursuant to the evergreen provisions of the Company's 2013 stock incentive plan (the “2013 Plan”), annual increases have resulted in the addition of an aggregate of approximately 13,081,000 additional shares to the 2013 Plan, including for 2021, an increase of approximately 2,542,000 shares. As of June 30, 2021, the Company had approximately 2,847,000 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, the Company's board of directors further amended the 2019 Inducement Plan to reserve an additional 600,000 shares of its common stock for issuance under the plan. As of June 30, 2021, the Company had approximately 731,000 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
13

Table of Contents
under the ESPP. Stock-based compensation by award type was as follows:
 Three Months Ended June 30,Six Months Ended June 30,
 2021202020212020
Stock options$1,042 $1,093 $2,511 $2,467 
Restricted stock units1,003 752 1,795 1,688 
Employee stock purchase plan34 28 65 42 
Total$2,079 $1,873 $4,371 $4,197 
The Company allocated stock-based compensation expense in the Company’s Consolidated Statements of Operations and Comprehensive Loss as follows:
 Three Months Ended June 30,Six Months Ended June 30,
 2021202020212020
Research and development$1,319 $951 $2,662 $2,104 
General and administrative760 922 1,709 2,093 
Total$2,079 $1,873 $4,371 $4,197 
Stock Options
A summary of the stock option activity, weighted average exercise prices, options outstanding, exercisable and expected to vest as of June 30, 2021 is as follows (in thousands except weighted average exercise price):
 Number of Shares Underlying OptionsWeighted
Average
Exercise
Price
Outstanding, December 31, 20208,928 $9.22 
Granted549 $6.66 
Exercised(144)$3.18 
Forfeited(100)$4.91 
Outstanding, June 30, 20219,233 $9.47 
Vested and exercisable, June 30, 20215,148 $12.67 
Vested and expected to vest, June 30, 20218,903 $9.62 
As of June 30, 2021, there were approximately $14.5 million of unrecognized compensation costs, net of estimated forfeitures, related to stock option awards grants, which are expected to be recognized over a remaining weighted average period of 3.0 years.
RSUs
The following table presents a summary of the Company's outstanding RSU awards granted as of June 30, 2021 (in thousands except weighted average grant-date fair value):
Restricted
Stock
Units
Weighted Average
Grant-Date
Fair Value
Outstanding, December 31, 20201,958 $6.95 
Awarded201 $6.57 
Vested(98)$3.31 
Forfeited(108)$36.14 
Outstanding, June 30, 20211,953 $5.48 
Outstanding, Expected to vest1,795 $5.48 
As of June 30, 2021, there were approximately $8.0 million of unrecognized compensation costs, net of estimated forfeitures, related to RSUs grants, which are expected to be recognized over a remaining weighted average period of 2.8 years.
14

Table of Contents
ESPP
As of June 30, 2021, there were 780,939 shares available for future purchases under the ESPP. There were no shares issued under the ESPP during the three months ended June 30, 2021 and 2020, respectively. There were 24,422 shares of common stock issued under the ESPP during the six months ended June 30, 2021. Cash proceeds from ESPP purchases were $121 thousand during the six months ended June 30, 2021. There were 43,581 shares of common stock issued under the ESPP during the six months ended June 30, 2020. Cash proceeds from ESPP purchases were $42 thousand during the six months ended June 30, 2020.
7. Income Taxes    
For the three and six months ended June 30, 2021, the Company recorded no income tax benefit. For the three and six months ended June 30, 2020, the Company recorded an income tax benefit of $0.4 million and $3.7 million, respectively, primarily to reflect a settlement of a local tax audit.
In response to the COVID-19 pandemic, the U.S. Congress enacted the Coronavirus Aid, Relief, and Economic Security Act (“CARES Act”) on March 27, 2020. The CARES Act provides numerous tax provisions and other stimulus measures, including the immediate refund of minimum tax credits.  In April 2021, the Company received the remaining balance, approximately $1.8 million, of its minimum tax credits refund.
The Company will continue to evaluate its ability to realize its deferred tax assets on a quarterly basis and will adjust such amounts in light of changing facts and circumstances including, but not limited to, future projections of taxable income, tax legislation, rulings by relevant tax authorities, the progress of ongoing tax audits and the regulatory approval of products currently under development. Any additional changes to the valuation allowance recorded on deferred tax assets in the future would impact the Company’s income taxes.
8. Fair Value Measurements
ASC 820, Fair Value Measurements and Disclosures, defines fair value as the price that would be received to sell an asset, or paid to transfer a liability, in the principal or most advantageous market in an orderly transaction between market participants on the measurement date. The fair value standard also establishes a three-level hierarchy, which requires an entity to maximize the use of observable inputs and minimize the use of unobservable inputs when measuring fair value.
The following table presents, for each of the fair value hierarchy levels required under ASC 820, the Company's assets
15

Table of Contents                                 
and liabilities that are measured at fair value on a recurring basis as of June 30, 2021:
 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*$48,435 $ $ 
Investments in U.S. Treasury securities$27,127 $ $ 
Investments in corporate debt securities$ $71,922 $ 
Investments in asset-backed securities$ $10,470 $ 
The following table presents, for each of the fair value hierarchy levels required under ASC 820, the Company's assets and liabilities that are measured at fair value on a recurring basis as of December 31, 2020:
 Fair Value Measurement Using
 Quoted prices in
active markets for
identical assets
(Level 1)
Significant other
observable inputs
(Level 2)
Significant
unobservable
inputs
(Level 3)
Assets   
Investments in money market funds*$58,042 $ $ 
Investments in U.S. Treasury securities 54,233   
Investments in corporate debt securities$ $79,275 $ 
Investments in asset-backed securities  10,166  
*Investments in money market funds are reflected in cash and cash equivalents in the accompanying Consolidated Balance Sheets.
No transfer of assets between Level 1 and Level 2 of the fair value measurement hierarchy occurred during the three and six months ended June 30, 2021.
9. 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
16

Table of Contents                                 
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
17

Table of Contents                                 
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. Discovery remains stayed while the parties negotiate the terms of the settlement agreement. The Company does not expect this settlement, if finalized as currently contemplated, to have a material impact on its financial condition.
On August 31, 2018, a shareholder derivative action was filed against current and former members of the Company's Board of Directors and certain current and former officers of the Company in the United States District Court for the Southern District of New York, captioned Luis Pacheco v. David R. Guyer, et al., Case No. 1:18-cv-07999. The complaint, which is based substantially on the facts alleged in the CAC, alleges that the defendants breached their fiduciary duties to the Company and wasted the Company's corporate assets by failing to oversee the Company's business, and also alleges that the defendants were unjustly enriched as a result of the alleged conduct, including through receipt of bonuses, stock options and similar compensation from the Company, and through sales of the Company's stock between March 2, 2015 and December 12, 2016. The complaint purports to seek unspecified damages on the Company's behalf, attorneys’ fees, and other costs, as well as an order directing the Company to reform and improve its corporate governance and internal procedures to comply with applicable laws, including submitting certain proposed amendments to the Company's corporate charter, bylaws and corporate governance policies for vote by the Company's stockholders. On December 14, 2018, the Company filed a motion to dismiss the complaint. On September 19, 2019, the court denied its motion to dismiss this complaint. This matter was subsequently referred to a special litigation committee (“SLC”) of the Company's board of directors. On February 18, 2020, the Company filed an answer to the complaint. The Company and the plaintiff agreed to stay this litigation while the special litigation committee conducts its
18

Table of Contents                                 
investigation.  On May 4, 2020, the court approved the stipulation and stayed the litigation through November 1, 2020. By agreement of the parties, the court has since extended the stay through June 26, 2021. The Company also entered into tolling agreements with the defendant directors to December 2021.
On October 16, 2018, the Company’s board of directors received a shareholder demand to investigate and commence legal proceedings against certain members of the Company’s board of directors. The demand alleges facts that are substantially similar to the facts alleged in the CAC and the Pacheco complaint and asserts claims that are substantially similar to the claims asserted in the Pacheco complaint. On January 30, 2019, the Company’s board of directors received a second shareholder demand from a different shareholder to investigate and commence legal proceedings against certain current and former members of the Company’s board of directors based on allegations that are substantially similar to the allegations contained in the first demand letter. These shareholder demands were referred to a demand review committee of the Company's board of directors. On May 6, 2021, the shareholders who served the October 16, 2018 demand filed a shareholder derivative action against current and former members of the Company’s Board of Directors and certain current and former officers of the Company in the New York Supreme Court, captioned Brian Ferber et al., derivatively on behalf of Ophthotech Corporation v. Axel Bolte et al., Index No. 154462/2021. The complaint asserts the same claims as those asserted in the Pacheco complaint and is based on factual allegations that are materially similar to the allegations in the Pacheco complaint. On June 22, 2021, the parties filed a stipulation staying the Ferber action until 60 days after the SLC concludes its investigation. The Company has entered into tolling agreements with the directors named in the demands to December 2021.
The Company denies any and all allegations of wrongdoing and intends to vigorously defend against these lawsuits. The Company is unable, however, to predict the outcome of these matters at this time. Moreover, any conclusion of these matters in a manner adverse to the Company and for which it incurs substantial costs or damages not covered by the Company's directors’ and officers’ liability insurance would have a material adverse effect on its financial condition and business. In addition, the litigation could adversely impact the Company's reputation and divert management’s attention and resources from other priorities, including the execution of its business plan and strategies that are important to the Company's ability to grow its business, any of which could have a material adverse effect on the Company's business.
10. Subsequent Events
In July 2021, the Company completed an underwritten public offering in which the Company sold 13,397,500 shares of its common stock, which included the exercise in full of the underwriters’ option to purchase an additional 1,747,500 shares of the Company’s common stock, at a price to the public of $8.60 per share and at a price to the underwriters of $8.084 per share.
The net proceeds from the public offering, after deducting underwriting discounts and commissions and other expenses payable by the Company totaling approximately $7.4 million, was $107.8 million.

19

Table of Contents                                 
Item 2.    Management's Discussion and Analysis of Financial Condition and Results of Operations
The following discussion and analysis of our financial condition and results of operations should be read together with our financial statements and related notes appearing elsewhere in this Quarterly Report on Form 10-Q and the audited financial statements and related notes and management’s discussion and analysis of financial condition and results of operations for the year ended December 31, 2020 included in our Annual Report on Form 10-K filed with the Securities and Exchange Commission on March 4, 2021. Some of the information contained in this discussion and analysis or set forth elsewhere in this Quarterly Report on Form 10-Q, including information with respect to our plans and strategy for our business and related financing, includes forward-looking statements that involve risks and uncertainties and should be read together with the “Risk Factors” section of this Quarterly Report on Form 10-Q for a discussion of important factors that could cause actual results to differ materially from the results described in or implied by the forward-looking statements contained in the following discussion and analysis.

Overview
We are a science-driven biopharmaceutical company focused on the discovery and development of novel treatment options for retinal diseases with significant unmet medical needs. We are currently developing both therapeutic product candidates for age-related retinal diseases and gene therapy product candidates for orphan inherited retinal diseases, or IRDs. We believe that both therapeutics and gene therapy serve important roles in drug development and providing potential treatment options for patients suffering from retinal diseases.
Our therapeutics portfolio consists of our clinical stage product candidate Zimura® (avacincaptad pegol), a complement C5 inhibitor, and our preclinical product candidate IC-500, a High temperature requirement A serine peptidase 1 protein, or HtrA1, inhibitor. We are currently targeting the following diseases with Zimura:
Geographic Atrophy, or GA, which is the advanced stage of age-related macular degeneration, or AMD, and is characterized by marked thinning or atrophy of retinal tissue, leading to irreversible loss of vision;
drusen secondary to AMD, which we refer to as drusen, which is an earlier stage of AMD and is characterized by yellow deposits under the retina which may grow larger and more numerous over time and potentially lead to GA and loss of vision; and
autosomal recessive Stargardt disease, or STGD1, which is an orphan inherited condition characterized by progressive damage to the central portion of the retina, or the macula, and other retinal tissue, leading to loss of vision.
In July 2021, we completed patient enrollment for GATHER2, our Phase 3 clinical trial evaluating the safety and efficacy of Zimura for the treatment of GA secondary to AMD, with a total of 448 patients enrolled. We also received a written agreement from the U.S. Food and Drug Administration, or FDA, under a Special Protocol Assessment, or SPA, for the overall design of GATHER2. We expect top-line data from the GATHER2 trial to become available during the second half of 2022, approximately one year after the enrollment of the last patient plus the time needed for database lock and analysis.
We plan to begin clinical development of Zimura for drusen in 2022. We are developing IC-500 for GA secondary to AMD and potentially other age-related retinal diseases.
Our gene therapy portfolio consists of two product candidates in preclinical development (IC-200 and IC-100) and several ongoing research programs, each of which uses adeno-associated virus, or AAV, for gene delivery. These AAV mediated gene therapy programs are targeting the following orphan IRDs:
autosomal recessive bestrophinopathy, or ARB, which is characterized by loss of central vision and abnormal changes in electrophysiology, and other IRDs associated with mutations in the BEST1 gene;
rhodopsin-mediated autosomal dominant retinitis pigmentosa, or RHO-adRP, which is characterized by progressive and severe bilateral loss of vision leading to blindness;
Leber Congenital Amaurosis type 10, or LCA10, which is characterized by severe bilateral loss of vision at or soon after birth;
autosomal recessive Stargardt disease; and
20

Table of Contents                                 
IRDs associated with mutations in the USH2A gene, which include Usher syndrome type 2A, or Usher 2A, and USH2A-associated non-syndromic autosomal recessive retinitis pigmentosa.
We are developing IC-200 for ARB and other BEST1-related IRDs, and IC-100 for RHO-adRP.
Research and Development Pipeline
We have summarized the current status of our ongoing research and development programs in the table below.
https://cdn.kscope.io/f233ae03caffb1b5c766df7db18b7f1b-isee-20210630_g1.jpg
*We have an option to exclusively in-license intellectual property resulting from these research programs.
Therapeutic Development Programs
Zimura
Zimura, our complement C5 inhibitor, is a chemically-synthesized, pegylated RNA aptamer. Aptamers are short molecules made up of a single stranded nucleic acid sequence or amino acid sequence that binds molecular targets with high selectivity and specificity. The following are brief descriptions of our ongoing clinical trials for Zimura and certain post-hoc analyses we conducted for the completed GATHER1 trial, and our manufacturing activities for Zimura.
GATHER2 (GA secondary to AMD - Ongoing)
Trial Design
GATHER2, also known as ISEE2008, is an international, randomized, double-masked, sham controlled, multi-center Phase 3 clinical trial evaluating the safety and efficacy of Zimura for the treatment of GA secondary to AMD. In this trial, patients are randomized to receive either monthly administration of Zimura 2 mg or sham during the first 12 months of the trial, at which time we plan to analyze the primary efficacy endpoint of the mean rate of growth (slope) estimated based on GA area, as measured by fundus autofluorescence, FAF, based on readings at three time points: baseline, month 6 and month 12. At month 12, patients in the Zimura 2 mg arm are re-randomized to receive either monthly or every other month administration of Zimura 2 mg. The final evaluation for all patients will take place at month 24.
Trial Progress
In July 2021, we completed patient enrollment in GATHER2 with a total of 448 patients enrolled. We expect 12-month top-line data from this trial to become available during the second half of 2022, approximately one year after the enrollment of the last patient plus the time needed for database lock and analysis. If the 12-month results are positive, we plan to file applications with the FDA and the European Medicines Agency, or EMA, for marketing approval of Zimura for GA secondary to AMD.
We are focusing on patient retention and continue to closely monitor the COVID-19 pandemic and its potential effect on the trial. We are targeting patient retention for the trial, as measured by the injection fidelity rate through month 12, of greater than 90%. The 12-month injection fidelity rate for GATHER1 was 87%. The injection fidelity rate is calculated by dividing the
21

Table of Contents                                 
total number of actual injections for all patients by the total number of expected injections based on the total number of patients enrolled in the trial.
Special Protocol Assessment
In July 2021, we received a written agreement from the FDA under a SPA for the overall design of GATHER2. The SPA is a procedure by which the FDA provides a clinical trial sponsor with an official evaluation and written guidance on the design of a proposed protocol intended to form the basis for a new drug application, or NDA. In connection with our SPA, the FDA recommended, and we accepted, modifying the primary efficacy endpoint for the GATHER2 trial from the mean rate of change in GA area over 12 months measured by FAF at three timepoints: baseline, month 6 and month 12, to the mean rate of growth (slope) estimated based on GA area measured by FAF in at least three timepoints: baseline, month 6 and month 12.
GATHER1 (GA secondary to AMD - Completed)
Trial Summary
GATHER1, also known as OPH2003, is an international, randomized, double-masked, sham controlled, multi-center Phase 2/3 clinical trial evaluating the safety and efficacy of Zimura for the treatment of GA secondary to AMD. We enrolled 286 patients in this trial across multiple treatment groups, including various Zimura doses and sham control groups, and patients were treated and followed for 18 months. In October 2019, we announced positive 12-month data from this trial and in June 2020, we completed this trial and announced 18-month data from this trial, which supported the 12-month data. We believe that the GATHER1 trial qualifies as the first of two Phase 3 trials typically required by the FDA for marketing approval of a pharmaceutical product.
Post-hoc Analysis of GATHER1
In parallel discussions with those for the GATHER2 SPA, the FDA indicated that, as part of a future NDA for Zimura, the results from GATHER1 will be considered using the original prespecified primary efficacy endpoint analysis (mean rate of change in GA area over 12 months measured by FAF at three timepoints: baseline, month 6 and month 12), together with a post-hoc analysis using the same FDA-preferred method that will be used prospectively for the GATHER2 trial (mean rate of growth (slope) estimated based on GA area measured by FAF in the relevant timepoints). The 12 month and 18 month results of this post-hoc analysis, as compared to the results of the original prespecified analysis for GATHER1, for the two treatment arms as compared to their corresponding sham arms, are described below. Safety results from GATHER1 were not impacted as part of this analysis.
Although we believe that the post-hoc analysis from the GATHER1 trial are consistent with the positive results
from the original prespecified analysis from the trial, any analyses, whether prespecified or post-hoc, that are intended to
support an application for marketing approval are a matter of review for the FDA and other regulatory authorities.

Zimura 2 mg Data

MRM AnalysisZimura 2 mg
(N = 67)
Sham
(N = 110)
Difference% DifferenceP-Value
12 Month Sq. Rt. Transformation:
Mean Rate of Change in GA Area (mm)0.2920.4020.11027.38%
0.0072(a)
Mean Rate of GA Growth (Slope) (mm)0.2830.3920.10927.73%
0.0063(b)
12 Month Observed Data:
Mean Rate of Change in GA Area (mm2)
1.5922.290.69730.45%
0.0059(b)
Mean Rate of GA Growth (Slope) (mm2)
1.2211.8890.66835.37%
0.0050(b)
18 Month Sq. Rt. Transformation:
Mean Rate of Change in GA Area (mm)0.4300.5990.16828.11%
0.0014(b)
Mean Rate of GA Growth (Slope) (mm)0.4510.6070.15625.75%
0.0027(b)
18 Month Observed Data:
Mean Rate of Change in GA Area (mm2)
2.4313.5871.15632.24%
0.0009(b)
Mean Rate of GA Growth (Slope) (mm2)
1.9142.9511.03735.13%
0.0023(b)

22

Table of Contents                                 
Explanatory notes:
the estimates for the Zimura 2 mg group vs. sham are from the mixed-effects repeated measures, or MRM, model, drawing on all available data, including data from groups with different randomization ratios in Part 1 and Part 2 of the trial, and should not be interpreted as directly observed data;
(a)     indicates prespecified primary endpoint; statistically significant;
(b)     indicates descriptive p-value.

Zimura 4 mg Data


MRM AnalysisZimura 4 mg
(N = 83)
Sham
(N = 84)
Difference% DifferenceP-Value
12 Month Sq. Rt. Transformation:
Mean Rate of Change in GA Area (mm)0.3210.4440.12427.81%
0.0051(a)
Mean Rate of GA Growth (Slope) (mm)0.3070.4160.10926.31%
0.0100(b)
12 Month Observed Data:
Mean Rate of Change in GA Area (mm2)
2.0612.7700.70925.59%
0.0082(b)
Mean Rate of GA Growth (Slope) (mm2)
1.6742.2730.59926.34%
0.0147(b)
18 Month Sq. Rt. Transformation:
Mean Rate of Change in GA Area (mm)0.3910.5590.16729.97%
0.0021(b)
Mean Rate of GA Growth (Slope) (mm)0.3730.5120.13927.11%
0.0086(b)
18 Month Observed Data:
Mean Rate of Change in GA Area (mm2)
2.4603.4861.02629.44%
0.0034(b)
Mean Rate of GA Growth (Slope) (mm2)
2.1423.0100.86828.82%
0.0106(b)

Explanatory notes:
(a)     indicates prespecified primary endpoint; statistically significant;
(b)     indicates descriptive p-value.

Zimura 2 mg Data by Part
We enrolled patients for the GATHER1 trial in two different parts, Part 1 and Part 2, with different dosages and randomization ratios in each Part. Twenty-five patients receiving Zimura 2 mg were enrolled in Part 1 of the trial and 42 patients receiving Zimura 2 mg were enrolled in Part 2 of the trial.
Below are the month 12 and month 18 results for the Zimura 2 mg group as compared to its corresponding sham group, for both Part 1 and Part 2, using both the original prespecified primary efficacy endpoint analysis for the GATHER1 trial and the post-hoc analysis using the FDA-preferred method that will be used prospectively for the GATHER2 trial:

Part 1 Only Data

MRM AnalysisZimura 2 mg
(N = 25)
Sham
(N = 26)
Difference% Difference
12 Month Sq. Rt. Transformation:
Mean Rate of Change in GA Area (mm)0.3290.4220.09322.07%
Mean Rate of GA Growth (Slope) (mm)0.3070.4230.11627.39%
12 Month Observed Data:
Mean Rate of Change in GA Area (mm2)
1.9102.5930.68326.35%
Mean Rate of GA Growth (Slope) (mm2)
1.6552.2380.58426.08%
18 Month Sq. Rt. Transformation:
Mean Rate of Change in GA Area (mm)0.4640.6350.17026.84%
Mean Rate of GA Growth (Slope) (mm)0.4460.6300.18429.23%
18 Month Observed Data:
23

Table of Contents                                 
Mean Rate of Change in GA Area (mm2)
2.7894.1031.31432.03%
Mean Rate of GA Growth (Slope) (mm2)
2.4823.3930.91126.85%


Part 2 Only Data

MRM AnalysisZimura 2 mg
(N = 42)
Sham
(N = 84)
Difference% Difference
12 Month Sq. Rt. Transformation:
Mean Rate of Change in GA Area (mm)0.308