10-Q 1 alny-20220930.htm 10-Q alny-20220930
false2022--12-31Q300011786700.003494100011786702022-01-012022-09-3000011786702022-10-21xbrli:shares00011786702022-09-30iso4217:USD00011786702021-12-31iso4217:USDxbrli:shares0001178670us-gaap:ProductMember2022-07-012022-09-300001178670us-gaap:ProductMember2021-07-012021-09-300001178670us-gaap:ProductMember2022-01-012022-09-300001178670us-gaap:ProductMember2021-01-012021-09-300001178670us-gaap:CollaborativeArrangementMember2022-07-012022-09-300001178670us-gaap:CollaborativeArrangementMember2021-07-012021-09-300001178670us-gaap:CollaborativeArrangementMember2022-01-012022-09-300001178670us-gaap:CollaborativeArrangementMember2021-01-012021-09-300001178670us-gaap:RoyaltyMember2022-07-012022-09-300001178670us-gaap:RoyaltyMember2021-07-012021-09-300001178670us-gaap:RoyaltyMember2022-01-012022-09-300001178670us-gaap:RoyaltyMember2021-01-012021-09-3000011786702022-07-012022-09-3000011786702021-07-012021-09-3000011786702021-01-012021-09-300001178670us-gaap:CommonStockMember2021-12-310001178670us-gaap:AdditionalPaidInCapitalMember2021-12-310001178670us-gaap:AccumulatedOtherComprehensiveIncomeMember2021-12-310001178670us-gaap:RetainedEarningsMember2021-12-310001178670us-gaap:CommonStockMember2022-01-012022-03-310001178670us-gaap:AdditionalPaidInCapitalMember2022-01-012022-03-3100011786702022-01-012022-03-310001178670us-gaap:AccumulatedOtherComprehensiveIncomeMember2022-01-012022-03-310001178670us-gaap:RetainedEarningsMember2022-01-012022-03-310001178670us-gaap:CommonStockMember2022-03-310001178670us-gaap:AdditionalPaidInCapitalMember2022-03-310001178670us-gaap:AccumulatedOtherComprehensiveIncomeMember2022-03-310001178670us-gaap:RetainedEarningsMember2022-03-3100011786702022-03-310001178670us-gaap:CommonStockMember2022-04-012022-06-300001178670us-gaap:AdditionalPaidInCapitalMember2022-04-012022-06-3000011786702022-04-012022-06-300001178670us-gaap:AccumulatedOtherComprehensiveIncomeMember2022-04-012022-06-300001178670us-gaap:RetainedEarningsMember2022-04-012022-06-300001178670us-gaap:CommonStockMember2022-06-300001178670us-gaap:AdditionalPaidInCapitalMember2022-06-300001178670us-gaap:AccumulatedOtherComprehensiveIncomeMember2022-06-300001178670us-gaap:RetainedEarningsMember2022-06-3000011786702022-06-300001178670us-gaap:CommonStockMember2022-07-012022-09-300001178670us-gaap:AdditionalPaidInCapitalMember2022-07-012022-09-300001178670alny:ConvertibleSeniorNotesDue2027Member2022-07-012022-09-300001178670us-gaap:AccumulatedOtherComprehensiveIncomeMember2022-07-012022-09-300001178670us-gaap:RetainedEarningsMember2022-07-012022-09-300001178670us-gaap:CommonStockMember2022-09-300001178670us-gaap:AdditionalPaidInCapitalMember2022-09-300001178670us-gaap:AccumulatedOtherComprehensiveIncomeMember2022-09-300001178670us-gaap:RetainedEarningsMember2022-09-300001178670us-gaap:CommonStockMember2020-12-310001178670us-gaap:AdditionalPaidInCapitalMember2020-12-310001178670us-gaap:AccumulatedOtherComprehensiveIncomeMember2020-12-310001178670us-gaap:RetainedEarningsMember2020-12-3100011786702020-12-310001178670us-gaap:CommonStockMember2021-01-012021-03-310001178670us-gaap:AdditionalPaidInCapitalMember2021-01-012021-03-3100011786702021-01-012021-03-310001178670us-gaap:AccumulatedOtherComprehensiveIncomeMember2021-01-012021-03-310001178670us-gaap:RetainedEarningsMember2021-01-012021-03-310001178670us-gaap:CommonStockMember2021-03-310001178670us-gaap:AdditionalPaidInCapitalMember2021-03-310001178670us-gaap:AccumulatedOtherComprehensiveIncomeMember2021-03-310001178670us-gaap:RetainedEarningsMember2021-03-3100011786702021-03-310001178670us-gaap:CommonStockMember2021-04-012021-06-300001178670us-gaap:AdditionalPaidInCapitalMember2021-04-012021-06-3000011786702021-04-012021-06-300001178670us-gaap:AccumulatedOtherComprehensiveIncomeMember2021-04-012021-06-300001178670us-gaap:RetainedEarningsMember2021-04-012021-06-300001178670us-gaap:CommonStockMember2021-06-300001178670us-gaap:AdditionalPaidInCapitalMember2021-06-300001178670us-gaap:AccumulatedOtherComprehensiveIncomeMember2021-06-300001178670us-gaap:RetainedEarningsMember2021-06-3000011786702021-06-300001178670us-gaap:CommonStockMember2021-07-012021-09-300001178670us-gaap:AdditionalPaidInCapitalMember2021-07-012021-09-300001178670us-gaap:AccumulatedOtherComprehensiveIncomeMember2021-07-012021-09-300001178670us-gaap:RetainedEarningsMember2021-07-012021-09-300001178670us-gaap:CommonStockMember2021-09-300001178670us-gaap:AdditionalPaidInCapitalMember2021-09-300001178670us-gaap:AccumulatedOtherComprehensiveIncomeMember2021-09-300001178670us-gaap:RetainedEarningsMember2021-09-3000011786702021-09-30alny:product0001178670country:USalny:ONPATTROMember2022-07-012022-09-300001178670country:USalny:ONPATTROMember2021-07-012021-09-300001178670country:USalny:ONPATTROMember2022-01-012022-09-300001178670country:USalny:ONPATTROMember2021-01-012021-09-300001178670srt:EuropeMemberalny:ONPATTROMember2022-07-012022-09-300001178670srt:EuropeMemberalny:ONPATTROMember2021-07-012021-09-300001178670srt:EuropeMemberalny:ONPATTROMember2022-01-012022-09-300001178670srt:EuropeMemberalny:ONPATTROMember2021-01-012021-09-300001178670alny:NonUSOrEuropeMemberalny:ONPATTROMember2022-07-012022-09-300001178670alny:NonUSOrEuropeMemberalny:ONPATTROMember2021-07-012021-09-300001178670alny:NonUSOrEuropeMemberalny:ONPATTROMember2022-01-012022-09-300001178670alny:NonUSOrEuropeMemberalny:ONPATTROMember2021-01-012021-09-300001178670alny:ONPATTROMember2022-07-012022-09-300001178670alny:ONPATTROMember2021-07-012021-09-300001178670alny:ONPATTROMember2022-01-012022-09-300001178670alny:ONPATTROMember2021-01-012021-09-300001178670alny:AmvuttraMembercountry:US2022-07-012022-09-300001178670alny:AmvuttraMembercountry:US2021-07-012021-09-300001178670alny:AmvuttraMembercountry:US2022-01-012022-09-300001178670alny:AmvuttraMembercountry:US2021-01-012021-09-300001178670alny:AmvuttraMembersrt:EuropeMember2022-07-012022-09-300001178670alny:AmvuttraMembersrt:EuropeMember2021-07-012021-09-300001178670alny:AmvuttraMembersrt:EuropeMember2022-01-012022-09-300001178670alny:AmvuttraMembersrt:EuropeMember2021-01-012021-09-300001178670alny:AmvuttraMember2022-07-012022-09-300001178670alny:AmvuttraMember2021-07-012021-09-300001178670alny:AmvuttraMember2022-01-012022-09-300001178670alny:AmvuttraMember2021-01-012021-09-300001178670country:USalny:GIVLAARIMember2022-07-012022-09-300001178670country:USalny:GIVLAARIMember2021-07-012021-09-300001178670country:USalny:GIVLAARIMember2022-01-012022-09-300001178670country:USalny:GIVLAARIMember2021-01-012021-09-300001178670alny:GIVLAARIMembersrt:EuropeMember2022-07-012022-09-300001178670alny:GIVLAARIMembersrt:EuropeMember2021-07-012021-09-300001178670alny:GIVLAARIMembersrt:EuropeMember2022-01-012022-09-300001178670alny:GIVLAARIMembersrt:EuropeMember2021-01-012021-09-300001178670alny:NonUSOrEuropeMemberalny:GIVLAARIMember2022-07-012022-09-300001178670alny:NonUSOrEuropeMemberalny:GIVLAARIMember2021-07-012021-09-300001178670alny:NonUSOrEuropeMemberalny:GIVLAARIMember2022-01-012022-09-300001178670alny:NonUSOrEuropeMemberalny:GIVLAARIMember2021-01-012021-09-300001178670alny:GIVLAARIMember2022-07-012022-09-300001178670alny:GIVLAARIMember2021-07-012021-09-300001178670alny:GIVLAARIMember2022-01-012022-09-300001178670alny:GIVLAARIMember2021-01-012021-09-300001178670country:USalny:OXLUMOMember2022-07-012022-09-300001178670country:USalny:OXLUMOMember2021-07-012021-09-300001178670country:USalny:OXLUMOMember2022-01-012022-09-300001178670country:USalny:OXLUMOMember2021-01-012021-09-300001178670alny:OXLUMOMembersrt:EuropeMember2022-07-012022-09-300001178670alny:OXLUMOMembersrt:EuropeMember2021-07-012021-09-300001178670alny:OXLUMOMembersrt:EuropeMember2022-01-012022-09-300001178670alny:OXLUMOMembersrt:EuropeMember2021-01-012021-09-300001178670alny:NonUSOrEuropeMemberalny:OXLUMOMember2022-07-012022-09-300001178670alny:NonUSOrEuropeMemberalny:OXLUMOMember2021-07-012021-09-300001178670alny:NonUSOrEuropeMemberalny:OXLUMOMember2022-01-012022-09-300001178670alny:NonUSOrEuropeMemberalny:OXLUMOMember2021-01-012021-09-300001178670alny:OXLUMOMember2022-07-012022-09-300001178670alny:OXLUMOMember2021-07-012021-09-300001178670alny:OXLUMOMember2022-01-012022-09-300001178670alny:OXLUMOMember2021-01-012021-09-300001178670us-gaap:ProductMember2022-09-300001178670us-gaap:ProductMember2021-12-310001178670us-gaap:CollaborativeArrangementMemberalny:RegeneronPharmaceuticalsIncorporationMember2022-07-012022-09-300001178670us-gaap:CollaborativeArrangementMemberalny:RegeneronPharmaceuticalsIncorporationMember2021-07-012021-09-300001178670us-gaap:CollaborativeArrangementMemberalny:RegeneronPharmaceuticalsIncorporationMember2022-01-012022-09-300001178670us-gaap:CollaborativeArrangementMemberalny:RegeneronPharmaceuticalsIncorporationMember2021-01-012021-09-300001178670us-gaap:CollaborativeArrangementMemberalny:NovartisMember2022-07-012022-09-300001178670us-gaap:CollaborativeArrangementMemberalny:NovartisMember2021-07-012021-09-300001178670us-gaap:CollaborativeArrangementMemberalny:NovartisMember2022-01-012022-09-300001178670us-gaap:CollaborativeArrangementMemberalny:NovartisMember2021-01-012021-09-300001178670us-gaap:CollaborativeArrangementMemberalny:VirBiotechnologyIncMember2022-07-012022-09-300001178670us-gaap:CollaborativeArrangementMemberalny:VirBiotechnologyIncMember2021-07-012021-09-300001178670us-gaap:CollaborativeArrangementMemberalny:VirBiotechnologyIncMember2022-01-012022-09-300001178670us-gaap:CollaborativeArrangementMemberalny:VirBiotechnologyIncMember2021-01-012021-09-300001178670us-gaap:CollaborativeArrangementMemberalny:OtherCollaborationsMember2022-07-012022-09-300001178670us-gaap:CollaborativeArrangementMemberalny:OtherCollaborationsMember2021-07-012021-09-300001178670us-gaap:CollaborativeArrangementMemberalny:OtherCollaborationsMember2022-01-012022-09-300001178670us-gaap:CollaborativeArrangementMemberalny:OtherCollaborationsMember2021-01-012021-09-300001178670alny:RegeneronPharmaceuticalsIncorporationMemberalny:ClinicalTrialAndManufacturingMember2022-07-012022-09-300001178670alny:ExternalServicesMemberalny:RegeneronPharmaceuticalsIncorporationMember2022-07-012022-09-300001178670alny:OtherMemberalny:RegeneronPharmaceuticalsIncorporationMember2022-07-012022-09-300001178670alny:RegeneronPharmaceuticalsIncorporationMemberalny:ClinicalTrialAndManufacturingMember2021-07-012021-09-300001178670alny:ExternalServicesMemberalny:RegeneronPharmaceuticalsIncorporationMember2021-07-012021-09-300001178670alny:OtherMemberalny:RegeneronPharmaceuticalsIncorporationMember2021-07-012021-09-300001178670alny:ClinicalTrialAndManufacturingMemberalny:OtherCollaborationsMember2022-07-012022-09-300001178670alny:ExternalServicesMemberalny:OtherCollaborationsMember2022-07-012022-09-300001178670alny:OtherMemberalny:OtherCollaborationsMember2022-07-012022-09-300001178670alny:ClinicalTrialAndManufacturingMemberalny:OtherCollaborationsMember2021-07-012021-09-300001178670alny:ExternalServicesMemberalny:OtherCollaborationsMember2021-07-012021-09-300001178670alny:OtherMemberalny:OtherCollaborationsMember2021-07-012021-09-300001178670alny:ClinicalTrialAndManufacturingMember2022-07-012022-09-300001178670alny:ExternalServicesMember2022-07-012022-09-300001178670alny:OtherMember2022-07-012022-09-300001178670alny:ClinicalTrialAndManufacturingMember2021-07-012021-09-300001178670alny:ExternalServicesMember2021-07-012021-09-300001178670alny:OtherMember2021-07-012021-09-300001178670alny:RegeneronPharmaceuticalsIncorporationMemberalny:ClinicalTrialAndManufacturingMember2022-01-012022-09-300001178670alny:ExternalServicesMemberalny:RegeneronPharmaceuticalsIncorporationMember2022-01-012022-09-300001178670alny:OtherMemberalny:RegeneronPharmaceuticalsIncorporationMember2022-01-012022-09-300001178670alny:RegeneronPharmaceuticalsIncorporationMemberalny:ClinicalTrialAndManufacturingMember2021-01-012021-09-300001178670alny:ExternalServicesMemberalny:RegeneronPharmaceuticalsIncorporationMember2021-01-012021-09-300001178670alny:OtherMemberalny:RegeneronPharmaceuticalsIncorporationMember2021-01-012021-09-300001178670alny:ClinicalTrialAndManufacturingMemberalny:OtherCollaborationsMember2022-01-012022-09-300001178670alny:ExternalServicesMemberalny:OtherCollaborationsMember2022-01-012022-09-300001178670alny:OtherMemberalny:OtherCollaborationsMember2022-01-012022-09-300001178670alny:ClinicalTrialAndManufacturingMemberalny:OtherCollaborationsMember2021-01-012021-09-300001178670alny:ExternalServicesMemberalny:OtherCollaborationsMember2021-01-012021-09-300001178670alny:OtherMemberalny:OtherCollaborationsMember2021-01-012021-09-300001178670alny:ClinicalTrialAndManufacturingMember2022-01-012022-09-300001178670alny:ExternalServicesMember2022-01-012022-09-300001178670alny:OtherMember2022-01-012022-09-300001178670alny:ClinicalTrialAndManufacturingMember2021-01-012021-09-300001178670alny:ExternalServicesMember2021-01-012021-09-300001178670alny:OtherMember2021-01-012021-09-300001178670alny:GlobalStrategicCollaborationMemberalny:RegeneronPharmaceuticalsIncorporationMember2019-04-012019-04-300001178670alny:GlobalStrategicCollaborationMemberalny:RegeneronPharmaceuticalsIncorporationMembersrt:MaximumMember2019-04-012019-04-30alny:target0001178670alny:GlobalStrategicCollaborationMemberalny:RegeneronPharmaceuticalsIncorporationMemberalny:FundingAtProgramInitiationMember2019-04-300001178670alny:GlobalStrategicCollaborationMemberalny:FundingAtLeadCandidateIdentificationMemberalny:RegeneronPharmaceuticalsIncorporationMember2019-04-300001178670alny:GlobalStrategicCollaborationMemberalny:RegeneronPharmaceuticalsIncorporationMemberalny:FundingAnAnnualDiscoveryMember2019-04-300001178670alny:GlobalStrategicCollaborationMemberalny:RegeneronPharmaceuticalsIncorporationMembersrt:MaximumMember2019-04-30xbrli:pure0001178670alny:GlobalStrategicCollaborationMemberalny:RegeneronPharmaceuticalsIncorporationMemberalny:ResearchServicesObligationMember2022-09-300001178670alny:GlobalStrategicCollaborationMemberalny:RegeneronPharmaceuticalsIncorporationMemberalny:ResearchServicesObligationMember2022-01-012022-09-300001178670alny:GlobalStrategicCollaborationMemberalny:RegeneronPharmaceuticalsIncorporationMemberalny:C5LicenseObligationMember2022-09-300001178670alny:GlobalStrategicCollaborationMemberalny:RegeneronPharmaceuticalsIncorporationMemberalny:C5LicenseObligationMember2022-01-012022-09-300001178670alny:GlobalStrategicCollaborationMemberalny:C5CoCoObligationMemberalny:RegeneronPharmaceuticalsIncorporationMember2022-09-300001178670alny:GlobalStrategicCollaborationMemberalny:C5CoCoObligationMemberalny:RegeneronPharmaceuticalsIncorporationMember2022-01-012022-09-300001178670alny:GlobalStrategicCollaborationMemberalny:RegeneronPharmaceuticalsIncorporationMember2022-01-012022-09-300001178670alny:GlobalStrategicCollaborationMemberalny:RegeneronPharmaceuticalsIncorporationMember2022-09-302022-09-300001178670alny:GlobalStrategicCollaborationMemberalny:RegeneronPharmaceuticalsIncorporationMemberalny:ResearchServicesObligationMember2022-09-302022-09-300001178670alny:GlobalStrategicCollaborationMemberalny:RegeneronPharmaceuticalsIncorporationMemberalny:ResearchServicesObligationMember2021-12-310001178670alny:GlobalStrategicCollaborationMemberalny:RegeneronPharmaceuticalsIncorporationMemberalny:C5LicenseObligationMember2022-09-302022-09-300001178670alny:GlobalStrategicCollaborationMemberalny:RegeneronPharmaceuticalsIncorporationMemberalny:C5LicenseObligationMember2021-12-310001178670alny:GlobalStrategicCollaborationMemberalny:C5CoCoObligationMemberalny:RegeneronPharmaceuticalsIncorporationMember2022-09-302022-09-300001178670alny:GlobalStrategicCollaborationMemberalny:C5CoCoObligationMemberalny:RegeneronPharmaceuticalsIncorporationMember2021-12-310001178670alny:GlobalStrategicCollaborationMemberalny:RegeneronPharmaceuticalsIncorporationMember2022-09-300001178670alny:GlobalStrategicCollaborationMemberalny:RegeneronPharmaceuticalsIncorporationMember2021-12-310001178670alny:GlobalStrategicCollaborationMemberalny:RegeneronPharmaceuticalsIncorporationMemberalny:ResearchServicesObligationMember2022-07-012022-09-300001178670alny:GlobalStrategicCollaborationMemberalny:RegeneronPharmaceuticalsIncorporationMemberalny:ResearchServicesObligationMember2021-07-012021-09-300001178670alny:GlobalStrategicCollaborationMemberalny:RegeneronPharmaceuticalsIncorporationMemberalny:ResearchServicesObligationMember2021-01-012021-09-300001178670alny:GlobalStrategicCollaborationMemberalny:RegeneronPharmaceuticalsIncorporationMemberalny:C5LicenseObligationMember2022-07-012022-09-300001178670alny:GlobalStrategicCollaborationMemberalny:RegeneronPharmaceuticalsIncorporationMemberalny:C5LicenseObligationMember2021-07-012021-09-300001178670alny:GlobalStrategicCollaborationMemberalny:RegeneronPharmaceuticalsIncorporationMemberalny:C5LicenseObligationMember2021-01-012021-09-300001178670alny:GlobalStrategicCollaborationMemberalny:C5CoCoObligationMemberalny:RegeneronPharmaceuticalsIncorporationMember2022-07-012022-09-300001178670alny:GlobalStrategicCollaborationMemberalny:C5CoCoObligationMemberalny:RegeneronPharmaceuticalsIncorporationMember2021-07-012021-09-300001178670alny:GlobalStrategicCollaborationMemberalny:C5CoCoObligationMemberalny:RegeneronPharmaceuticalsIncorporationMember2021-01-012021-09-300001178670alny:GlobalStrategicCollaborationMemberalny:RegeneronPharmaceuticalsIncorporationMember2022-07-012022-09-300001178670alny:GlobalStrategicCollaborationMemberalny:RegeneronPharmaceuticalsIncorporationMember2021-07-012021-09-300001178670alny:GlobalStrategicCollaborationMemberalny:RegeneronPharmaceuticalsIncorporationMember2021-01-012021-09-300001178670alny:NovartisMemberalny:ProductAlliancesMember2013-02-012022-09-300001178670alny:NovartisMemberalny:ProductAlliancesMember2013-02-012013-02-280001178670alny:NovartisAGMemberalny:A2013TheMedicinesCompanyCollaborationMemberalny:ProductAlliancesMember2013-02-012013-02-280001178670alny:NovartisMemberalny:ProductAlliancesMembersrt:MinimumMember2022-09-300001178670alny:NovartisMembersrt:MaximumMemberalny:ProductAlliancesMember2022-09-300001178670alny:NovartisLicenseAgreementMember2021-12-012021-12-310001178670alny:NovartisAGMember2022-01-012022-09-300001178670alny:NovartisAGMembersrt:MinimumMember2022-01-012022-09-300001178670alny:NovartisLicenseAgreementMember2022-09-302022-09-300001178670alny:NovartisLicenseAgreementMember2022-09-300001178670alny:VirBiotechnologyIncMember2022-09-30alny:program0001178670alny:VirBiotechnologyIncMember2022-01-012022-09-30alny:candidate0001178670alny:VirBiotechnologyIncMember2022-09-302022-09-300001178670alny:PeptiDreamIncMember2021-07-012021-07-310001178670alny:PeptiDreamIncMember2021-07-012021-09-300001178670alny:BlackstoneGroupIncMemberus-gaap:CollaborativeArrangementMember2020-04-300001178670alny:BlackstoneGroupIncMemberus-gaap:CollaborativeArrangementMember2022-01-012022-09-300001178670alny:BlackstoneGroupIncMemberus-gaap:CollaborativeArrangementMembersrt:ScenarioForecastMember2030-01-010001178670alny:BlackstoneGroupIncMemberus-gaap:CollaborativeArrangementMember2020-04-012020-04-300001178670alny:BlackstoneGroupIncMemberus-gaap:CollaborativeArrangementMember2021-09-012021-09-300001178670alny:BlackstoneGroupIncMemberus-gaap:CollaborativeArrangementMember2022-09-300001178670alny:BlackstoneGroupIncMemberus-gaap:CollaborativeArrangementMember2021-12-310001178670us-gaap:FairValueMeasurementsRecurringMemberus-gaap:MoneyMarketFundsMember2022-09-300001178670us-gaap:FairValueMeasurementsRecurringMemberus-gaap:FairValueInputsLevel1Memberus-gaap:MoneyMarketFundsMember2022-09-300001178670us-gaap:FairValueMeasurementsRecurringMemberus-gaap:FairValueInputsLevel2Memberus-gaap:MoneyMarketFundsMember2022-09-300001178670us-gaap:FairValueMeasurementsRecurringMemberus-gaap:FairValueInputsLevel3Memberus-gaap:MoneyMarketFundsMember2022-09-300001178670us-gaap:FairValueMeasurementsRecurringMemberus-gaap:USTreasurySecuritiesMember2022-09-300001178670us-gaap:FairValueMeasurementsRecurringMemberus-gaap:FairValueInputsLevel1Memberus-gaap:USTreasurySecuritiesMember2022-09-300001178670us-gaap:FairValueMeasurementsRecurringMemberus-gaap:FairValueInputsLevel2Memberus-gaap:USTreasurySecuritiesMember2022-09-300001178670us-gaap:FairValueMeasurementsRecurringMemberus-gaap:FairValueInputsLevel3Memberus-gaap:USTreasurySecuritiesMember2022-09-300001178670us-gaap:FairValueMeasurementsRecurringMemberus-gaap:CommercialPaperMember2022-09-300001178670us-gaap:FairValueMeasurementsRecurringMemberus-gaap:FairValueInputsLevel1Memberus-gaap:CommercialPaperMember2022-09-300001178670us-gaap:FairValueMeasurementsRecurringMemberus-gaap:FairValueInputsLevel2Memberus-gaap:CommercialPaperMember2022-09-300001178670us-gaap:FairValueMeasurementsRecurringMemberus-gaap:FairValueInputsLevel3Memberus-gaap:CommercialPaperMember2022-09-300001178670us-gaap:CorporateDebtSecuritiesMemberus-gaap:FairValueMeasurementsRecurringMember2022-09-300001178670us-gaap:CorporateDebtSecuritiesMemberus-gaap:FairValueMeasurementsRecurringMemberus-gaap:FairValueInputsLevel1Member2022-09-300001178670us-gaap:CorporateDebtSecuritiesMemberus-gaap:FairValueMeasurementsRecurringMemberus-gaap:FairValueInputsLevel2Member2022-09-300001178670us-gaap:CorporateDebtSecuritiesMemberus-gaap:FairValueMeasurementsRecurringMemberus-gaap:FairValueInputsLevel3Member2022-09-300001178670us-gaap:FairValueMeasurementsRecurringMemberus-gaap:USGovernmentSponsoredEnterprisesDebtSecuritiesMember2022-09-300001178670us-gaap:FairValueMeasurementsRecurringMemberus-gaap:FairValueInputsLevel1Memberus-gaap:USGovernmentSponsoredEnterprisesDebtSecuritiesMember2022-09-300001178670us-gaap:FairValueMeasurementsRecurringMemberus-gaap:FairValueInputsLevel2Memberus-gaap:USGovernmentSponsoredEnterprisesDebtSecuritiesMember2022-09-300001178670us-gaap:FairValueMeasurementsRecurringMemberus-gaap:FairValueInputsLevel3Memberus-gaap:USGovernmentSponsoredEnterprisesDebtSecuritiesMember2022-09-300001178670us-gaap:FairValueMeasurementsRecurringMember2022-09-300001178670us-gaap:FairValueMeasurementsRecurringMemberus-gaap:FairValueInputsLevel1Member2022-09-300001178670us-gaap:FairValueMeasurementsRecurringMemberus-gaap:FairValueInputsLevel2Member2022-09-300001178670us-gaap:FairValueMeasurementsRecurringMemberus-gaap:FairValueInputsLevel3Member2022-09-300001178670us-gaap:FairValueMeasurementsRecurringMemberus-gaap:MoneyMarketFundsMember2021-12-310001178670us-gaap:FairValueMeasurementsRecurringMemberus-gaap:FairValueInputsLevel1Memberus-gaap:MoneyMarketFundsMember2021-12-310001178670us-gaap:FairValueMeasurementsRecurringMemberus-gaap:FairValueInputsLevel2Memberus-gaap:MoneyMarketFundsMember2021-12-310001178670us-gaap:FairValueMeasurementsRecurringMemberus-gaap:FairValueInputsLevel3Memberus-gaap:MoneyMarketFundsMember2021-12-310001178670us-gaap:FairValueMeasurementsRecurringMemberus-gaap:USTreasurySecuritiesMember2021-12-310001178670us-gaap:FairValueMeasurementsRecurringMemberus-gaap:FairValueInputsLevel1Memberus-gaap:USTreasurySecuritiesMember2021-12-310001178670us-gaap:FairValueMeasurementsRecurringMemberus-gaap:FairValueInputsLevel2Memberus-gaap:USTreasurySecuritiesMember2021-12-310001178670us-gaap:FairValueMeasurementsRecurringMemberus-gaap:FairValueInputsLevel3Memberus-gaap:USTreasurySecuritiesMember2021-12-310001178670us-gaap:CorporateDebtSecuritiesMemberus-gaap:FairValueMeasurementsRecurringMember2021-12-310001178670us-gaap:CorporateDebtSecuritiesMemberus-gaap:FairValueMeasurementsRecurringMemberus-gaap:FairValueInputsLevel1Member2021-12-310001178670us-gaap:CorporateDebtSecuritiesMemberus-gaap:FairValueMeasurementsRecurringMemberus-gaap:FairValueInputsLevel2Member2021-12-310001178670us-gaap:CorporateDebtSecuritiesMemberus-gaap:FairValueMeasurementsRecurringMemberus-gaap:FairValueInputsLevel3Member2021-12-310001178670us-gaap:FairValueMeasurementsRecurringMemberus-gaap:USGovernmentSponsoredEnterprisesDebtSecuritiesMember2021-12-310001178670us-gaap:FairValueMeasurementsRecurringMemberus-gaap:FairValueInputsLevel1Memberus-gaap:USGovernmentSponsoredEnterprisesDebtSecuritiesMember2021-12-310001178670us-gaap:FairValueMeasurementsRecurringMemberus-gaap:FairValueInputsLevel2Memberus-gaap:USGovernmentSponsoredEnterprisesDebtSecuritiesMember2021-12-310001178670us-gaap:FairValueMeasurementsRecurringMemberus-gaap:FairValueInputsLevel3Memberus-gaap:USGovernmentSponsoredEnterprisesDebtSecuritiesMember2021-12-310001178670us-gaap:FairValueMeasurementsRecurringMemberus-gaap:CommercialPaperMember2021-12-310001178670us-gaap:FairValueMeasurementsRecurringMemberus-gaap:FairValueInputsLevel1Memberus-gaap:CommercialPaperMember2021-12-310001178670us-gaap:FairValueMeasurementsRecurringMemberus-gaap:FairValueInputsLevel2Memberus-gaap:CommercialPaperMember2021-12-310001178670us-gaap:FairValueMeasurementsRecurringMemberus-gaap:FairValueInputsLevel3Memberus-gaap:CommercialPaperMember2021-12-310001178670us-gaap:CertificatesOfDepositMemberus-gaap:FairValueMeasurementsRecurringMember2021-12-310001178670us-gaap:CertificatesOfDepositMemberus-gaap:FairValueMeasurementsRecurringMemberus-gaap:FairValueInputsLevel1Member2021-12-310001178670us-gaap:CertificatesOfDepositMemberus-gaap:FairValueMeasurementsRecurringMemberus-gaap:FairValueInputsLevel2Member2021-12-310001178670us-gaap:CertificatesOfDepositMemberus-gaap:FairValueMeasurementsRecurringMemberus-gaap:FairValueInputsLevel3Member2021-12-310001178670us-gaap:FairValueMeasurementsRecurringMemberus-gaap:MunicipalNotesMember2021-12-310001178670us-gaap:FairValueMeasurementsRecurringMemberus-gaap:FairValueInputsLevel1Memberus-gaap:MunicipalNotesMember2021-12-310001178670us-gaap:FairValueMeasurementsRecurringMemberus-gaap:FairValueInputsLevel2Memberus-gaap:MunicipalNotesMember2021-12-310001178670us-gaap:FairValueMeasurementsRecurringMemberus-gaap:FairValueInputsLevel3Memberus-gaap:MunicipalNotesMember2021-12-310001178670us-gaap:FairValueMeasurementsRecurringMember2021-12-310001178670us-gaap:FairValueMeasurementsRecurringMemberus-gaap:FairValueInputsLevel1Member2021-12-310001178670us-gaap:FairValueMeasurementsRecurringMemberus-gaap:FairValueInputsLevel2Member2021-12-310001178670us-gaap:FairValueMeasurementsRecurringMemberus-gaap:FairValueInputsLevel3Member2021-12-310001178670us-gaap:USTreasurySecuritiesMember2022-09-300001178670us-gaap:CorporateDebtSecuritiesMember2022-09-300001178670us-gaap:USGovernmentSponsoredEnterprisesDebtSecuritiesMember2022-09-300001178670us-gaap:CommercialPaperMember2022-09-300001178670us-gaap:USTreasurySecuritiesMember2021-12-310001178670us-gaap:CorporateDebtSecuritiesMember2021-12-310001178670us-gaap:USGovernmentSponsoredEnterprisesDebtSecuritiesMember2021-12-310001178670us-gaap:CommercialPaperMember2021-12-310001178670us-gaap:CertificatesOfDepositMember2021-12-310001178670us-gaap:MunicipalNotesMember2021-12-310001178670us-gaap:OtherAssetsMember2022-09-300001178670us-gaap:OtherAssetsMember2021-12-310001178670alny:AccumulatedLossOnInvestmentInJointVentureMember2021-12-310001178670us-gaap:AccumulatedDefinedBenefitPlansAdjustmentMember2021-12-310001178670us-gaap:AccumulatedNetUnrealizedInvestmentGainLossMember2021-12-310001178670us-gaap:AccumulatedTranslationAdjustmentMember2021-12-310001178670alny:AccumulatedLossOnInvestmentInJointVentureMember2022-01-012022-09-300001178670us-gaap:AccumulatedDefinedBenefitPlansAdjustmentMember2022-01-012022-09-300001178670us-gaap:AccumulatedNetUnrealizedInvestmentGainLossMember2022-01-012022-09-300001178670us-gaap:AccumulatedTranslationAdjustmentMember2022-01-012022-09-300001178670alny:AccumulatedLossOnInvestmentInJointVentureMember2022-09-300001178670us-gaap:AccumulatedDefinedBenefitPlansAdjustmentMember2022-09-300001178670us-gaap:AccumulatedNetUnrealizedInvestmentGainLossMember2022-09-300001178670us-gaap:AccumulatedTranslationAdjustmentMember2022-09-300001178670alny:AccumulatedLossOnInvestmentInJointVentureMember2020-12-310001178670us-gaap:AccumulatedDefinedBenefitPlansAdjustmentMember2020-12-310001178670us-gaap:AccumulatedNetUnrealizedInvestmentGainLossMember2020-12-310001178670us-gaap:AccumulatedTranslationAdjustmentMember2020-12-310001178670alny:AccumulatedLossOnInvestmentInJointVentureMember2021-01-012021-09-300001178670us-gaap:AccumulatedDefinedBenefitPlansAdjustmentMember2021-01-012021-09-300001178670us-gaap:AccumulatedNetUnrealizedInvestmentGainLossMember2021-01-012021-09-300001178670us-gaap:AccumulatedTranslationAdjustmentMember2021-01-012021-09-300001178670alny:AccumulatedLossOnInvestmentInJointVentureMember2021-09-300001178670us-gaap:AccumulatedDefinedBenefitPlansAdjustmentMember2021-09-300001178670us-gaap:AccumulatedNetUnrealizedInvestmentGainLossMember2021-09-300001178670us-gaap:AccumulatedTranslationAdjustmentMember2021-09-300001178670us-gaap:SecuredDebtMemberus-gaap:LineOfCreditMember2020-04-30alny:tranche0001178670us-gaap:SecuredDebtMemberus-gaap:LineOfCreditMemberalny:TrancheOneLoanMember2020-01-012020-12-310001178670us-gaap:SecuredDebtMemberalny:TrancheTwoLoanMemberus-gaap:LineOfCreditMember2021-01-012021-06-300001178670us-gaap:SecuredDebtMemberalny:TrancheThreeLoanMemberus-gaap:LineOfCreditMember2021-01-012021-12-310001178670us-gaap:SecuredDebtMemberus-gaap:LineOfCreditMember2022-09-300001178670us-gaap:SecuredDebtMemberus-gaap:LineOfCreditMember2022-07-012022-09-300001178670us-gaap:SecuredDebtMemberus-gaap:LondonInterbankOfferedRateLiborSwapRateMemberus-gaap:LineOfCreditMember2022-01-012022-09-300001178670us-gaap:SecuredDebtMemberus-gaap:LineOfCreditMember2022-01-012022-09-300001178670us-gaap:SecuredDebtMemberus-gaap:LineOfCreditMember2021-12-310001178670us-gaap:SecuredDebtMemberus-gaap:LineOfCreditMember2020-04-012020-04-300001178670alny:ConvertibleSeniorNotesDue2027InitialAmountMemberus-gaap:ConvertibleDebtMember2022-09-120001178670alny:ConvertibleSeniorNotesDue2027Memberus-gaap:ConvertibleDebtMember2022-09-122022-09-120001178670alny:ConvertibleSeniorNotesDue2027AdditionalAmountMemberus-gaap:ConvertibleDebtMember2022-09-130001178670alny:ConvertibleSeniorNotesDue2027Memberus-gaap:ConvertibleDebtMember2022-09-132022-09-130001178670alny:ConvertibleSeniorNotesDue2027Memberus-gaap:ConvertibleDebtMember2022-09-130001178670alny:ConvertibleSeniorNotesDue2027Memberus-gaap:ConvertibleDebtMember2022-09-152022-09-150001178670alny:ConvertibleSeniorNotesDue2027Memberalny:DebtConversionTermsOneMemberus-gaap:ConvertibleDebtMember2022-09-152022-09-15alny:day0001178670alny:ConvertibleSeniorNotesDue2027Memberalny:DebtConversionTermsTwoMemberus-gaap:ConvertibleDebtMember2022-09-152022-09-150001178670alny:ConvertibleSeniorNotesDue2027Memberus-gaap:ConvertibleDebtMember2022-09-120001178670alny:ConvertibleSeniorNotesDue2027Memberus-gaap:ConvertibleDebtMember2022-09-300001178670alny:ConvertibleSeniorNotesDue2027Memberus-gaap:ConvertibleDebtMember2022-01-012022-09-300001178670us-gaap:ConvertibleDebtMemberus-gaap:CallOptionMember2022-09-30iso4217:USDalny:Unit0001178670us-gaap:ConvertibleDebtMemberus-gaap:CallOptionMember2022-09-120001178670us-gaap:ConvertibleDebtMemberus-gaap:CallOptionMember2022-01-012022-09-300001178670us-gaap:CollaborativeArrangementMemberalny:BlackstoneLifeSciencesMemberalny:VutrisiranAndZilebesiranMember2020-08-012020-08-310001178670us-gaap:CollaborativeArrangementMemberalny:HELIOSBPhase3ClinicalTrialMemberalny:BlackstoneLifeSciencesMember2020-08-012020-08-310001178670us-gaap:CollaborativeArrangementMemberalny:BlackstoneLifeSciencesMemberalny:ALNAGTPhase2ClinicalTrialMember2020-08-012020-08-310001178670us-gaap:CollaborativeArrangementMemberalny:BlackstoneLifeSciencesMemberalny:ALNAGTPhase3ClinicalTrialMember2020-08-012020-08-310001178670alny:VutrisiranMemberus-gaap:CollaborativeArrangementMemberalny:BlackstoneLifeSciencesMember2020-08-012020-08-310001178670alny:BlackstoneLifeSciencesMember2022-09-300001178670us-gaap:DerivativeMember2021-12-310001178670us-gaap:DerivativeMember2022-01-012022-09-300001178670us-gaap:DerivativeMember2022-09-300001178670us-gaap:ResearchAndDevelopmentExpenseMember2022-07-012022-09-300001178670us-gaap:ResearchAndDevelopmentExpenseMember2021-07-012021-09-300001178670us-gaap:ResearchAndDevelopmentExpenseMember2022-01-012022-09-300001178670us-gaap:ResearchAndDevelopmentExpenseMember2021-01-012021-09-300001178670us-gaap:SellingGeneralAndAdministrativeExpensesMember2022-07-012022-09-300001178670us-gaap:SellingGeneralAndAdministrativeExpensesMember2021-07-012021-09-300001178670us-gaap:SellingGeneralAndAdministrativeExpensesMember2022-01-012022-09-300001178670us-gaap:SellingGeneralAndAdministrativeExpensesMember2021-01-012021-09-300001178670us-gaap:EmployeeStockOptionMember2022-01-012022-09-300001178670us-gaap:EmployeeStockOptionMember2021-01-012021-09-300001178670us-gaap:RestrictedStockMember2022-01-012022-09-300001178670us-gaap:RestrictedStockMember2021-01-012021-09-300001178670us-gaap:ConvertibleDebtMember2022-01-012022-09-300001178670us-gaap:ConvertibleDebtMember2021-01-012021-09-30

UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
Washington, D.C. 20549
____________________________________________
FORM 10-Q
____________________________________________
QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934
For the quarterly period ended September 30, 2022
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 001-36407
__________________________________________
ALNYLAM PHARMACEUTICALS, INC.
(Exact Name of Registrant as Specified in Its Charter)
__________________________________________
Delaware
(State or Other Jurisdiction of
Incorporation or Organization)
77-0602661
(I.R.S. Employer
Identification No.)
675 West Kendall Street,
Henri A. Termeer Square
Cambridge, MA
(Address of Principal Executive Offices)
02142
(Zip Code)
(617) 551-8200
(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, $0.01 par value per shareALNYThe Nasdaq Stock Market LLC
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  x   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  x   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 filerxAccelerated filer
Non-accelerated filerSmaller reporting company
Emerging 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  x
At October 21, 2022, the registrant had 123,028,234 shares of Common Stock, $0.01 par value per share, outstanding.




ALNYLAM PHARMACEUTICALS, INC.
QUARTERLY REPORT ON FORM 10-Q

TABLE OF CONTENTS
PAGE
NUMBER

“Alnylam,” ONPATTRO®, GIVLAARI®, OXLUMO®, AMVUTTRA®, Alnylam Act®, Alnylam Assist®, GEMINI™ and IKARIA™ are trademarks and registered trademarks of Alnylam Pharmaceuticals, Inc. Our logo, trademarks and service marks are property of Alnylam. All other trademarks or service marks appearing in this Quarterly Report on Form 10-Q are the property of their respective holders.
2

CAUTIONARY NOTE REGARDING FORWARD-LOOKING STATEMENTS
This Quarterly Report on Form 10-Q contains forward-looking statements within the meaning of the federal securities laws, Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. We intend these forward-looking statements to be covered by the safe harbor provisions for forward-looking statements contained in the Private Securities Litigation Reform Act of 1995 and are including this statement for purposes of complying with those safe harbor provisions. All statements other than statements of historical facts contained in this Quarterly Report on Form 10-Q are forward-looking statements. In some cases, you can identify forward-looking statements by terminology such as “may,” “will,” “should,” “could,” “expects,” “plans,” “intends,” “anticipates,” “believes,” “estimates,” “predicts,” “potential,” “continue,” or the negative of these terms or other comparable terminology. These forward-looking statements include, but are not limited to, statements about:
our views with respect to the potential for approved and investigational RNAi therapeutics, including ONPATTRO, GIVLAARI, OXLUMO, AMVUTTRA, Leqvio® (inclisiran), fitusiran and zilebesiran;
our plans for additional global regulatory filings and the continuing product launches of ONPATTRO, GIVLAARI, OXLUMO, AMVUTTRA and our partner's plans with respect to Leqvio;
risks related to the direct or indirect impact of the novel coronavirus, or COVID-19, global pandemic, emerging or future variants of COVID-19 or any future pandemic, such as the scope and duration of the pandemic, government actions and restrictive measures implemented in response, the effectiveness of vaccination and booster vaccination campaigns, material delays in diagnoses of rare diseases, initiation or continuation of treatment for diseases addressed by our products, or in patient enrollment in clinical trials, potential clinical trial, regulatory review and inspection or supply chain disruptions, and other potential impacts to our business;
our expectations regarding potential market size for, and the successful commercialization of, ONPATTRO, GIVLAARI, OXLUMO, AMVUTTRA, Leqvio or any future products;
our ability to obtain and maintain regulatory approvals and pricing and reimbursement for ONPATTRO, GIVLAARI, OXLUMO, AMVUTTRA or any future products, and our partners' ability with respect to Leqvio and fitusiran;
the progress of our research and development programs, including programs in both rare and prevalent diseases;
our future ability to successfully expand the indications for ONPATTRO, AMVUTTRA and OXLUMO;
the potential for improved product profiles to emerge from our new technologies, including our IKARIA and GEMINI platforms and our ability to successfully advance our delivery efforts in extrahepatic tissues;
our current and anticipated clinical trials and expectations regarding the reporting of data from these trials;
any impact of the on-going conflict in Ukraine and the imposition of related government sanctions on our business and operations, including disruptions to our clinical trials;
the timing of regulatory filings and interactions with or actions or advice of regulatory authorities, which may affect the design, initiation, timing, continuation and/or progress of clinical trials or result in the need for additional pre-clinical and/or clinical testing or the timing or likelihood of regulatory approvals;
the status of our manufacturing operations and any delays, interruptions or failures in the manufacture and supply of ONPATTRO, GIVLAARI, OXLUMO, AMVUTTRA or any of our product candidates (or other products or product candidates being developed and commercialized by our partners), by our or their contract manufacturers or by us or our partners;
our progress continuing to build and leverage our global commercial infrastructure;
the possible impact of any competing products on the commercial success of ONPATTRO, GIVLAARI, OXLUMO, AMVUTTRA and Leqvio, as well as our product candidates, and, our, or with respect to Leqvio or fitusiran, our partners', ability to compete against such products;
our ability to manage our growth and operating expenses;
our views and plans with respect to our 5-year Alnylam P5x25 strategy and our intentions to achieve the metrics associated with this strategy, including to become a top five biotech company by the end of 2025;
our belief that our current cash balance should enable us to achieve a self-sustainable profile without the need for future equity financing;
our expectations regarding the length of time our current cash, cash equivalents and marketable equity and debt securities will support our operations based on our current operating plan;
our dependence on third parties for development, manufacture and distribution of products;
3

our expectations regarding our corporate collaborations, including potential future licensing fees and milestone and royalty payments under existing or future agreements;
our ability to obtain, maintain and protect our intellectual property;
our ability to attract and retain qualified key management and scientists, development, medical and commercial staff, consultants and advisors and to successfully execute on our Alnylam P5x25 strategy;
the outcome of litigation, including our patent infringement suits against Pfizer, Inc., BioNTech SE and Moderna, Inc., or other legal proceedings or of any current or future government investigation, including the investigation related to the subpoena received on or about April 9, 2021 pertaining to our marketing and promotion of ONPATTRO in the United States, or U.S.;
regulatory developments in the U.S. and foreign countries;
the impact of laws and regulations;
developments relating to our competitors and our industry;
our ability to satisfy our payment obligations, remain in compliance with covenants under our debt agreements and to service the interest on or to refinance our indebtedness, including our convertible notes, or to make cash payments in connection with any conversion of the Notes, to the extent required;
our expectations regarding the effect of the capped call transactions and regarding the anticipated market activities of the option counterparties and/or their respective affiliates; and
other risks and uncertainties, including those listed under the caption Part II, Item 1A, "Risk Factors" of this Quarterly Report on Form 10-Q.
The risks set forth above are not exhaustive. Other sections of this Quarterly Report on Form 10-Q may include additional factors that could adversely affect our business and financial performance. Moreover, we operate in a very competitive and rapidly changing environment. New risk factors emerge from time to time and it is not possible for management to predict all risk factors, nor can we assess the impact of all risk factors on our business or the extent to which any factor, or combination of factors, may cause actual results to differ materially from those contained in any forward-looking statements. Any forward-looking statements in this Quarterly Report on Form 10-Q reflect our current views with respect to future events and with respect to our business and future financial performance, and involve known and unknown risks, uncertainties and other factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by these forward-looking statements. Factors that may cause actual results to differ materially from current expectations include, among other things, those described under Part II, Item 1A, "Risk Factors" and elsewhere in this Quarterly Report on Form 10-Q. Given these uncertainties, you should not place undue reliance on these forward-looking statements. Except as required by law, we assume no obligation to update or revise these forward-looking statements for any reason, even if new information becomes available in the future. You are advised, however, to consult any further disclosure we make in our reports filed with the SEC.
This Quarterly Report on Form 10-Q may include data that we obtained from industry publications and third-party research, surveys and studies. 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. This Quarterly Report on Form 10-Q also may include data based on our own internal estimates and research, including estimates regarding the impact of the ongoing COVID-19 pandemic (or related pandemic caused by coronavirus variants) on our financial statements and business operations. Our internal estimates have not been verified by any independent source and, while we believe any data obtained from industry publications and third-party research, surveys and studies are reliable, we have not independently verified such data. Such third-party data, as well as our internal estimates and research, are subject to a high degree of uncertainty and risk due to a variety of factors, including those described in Part II, Item 1A, "Risk Factors" and elsewhere in this Quarterly Report on Form 10-Q. These and other factors could cause our results to differ materially from those expressed in this Quarterly Report on Form 10-Q.

4


PART I. FINANCIAL INFORMATION

ALNYLAM PHARMACEUTICALS, INC.
CONDENSED CONSOLIDATED BALANCE SHEETS
(In thousands, except per share amounts)
(Unaudited)


September 30, 2022December 31, 2021
ASSETS
Current assets:
Cash and cash equivalents$1,073,228 $819,975 
Marketable debt securities1,169,050 1,548,617 
Marketable equity securities23,051 66,972 
Accounts receivable, net184,513 198,571 
Inventory115,489 86,363 
Prepaid expenses and other current assets125,516 88,078 
Total current assets2,690,847 2,808,576 
Property, plant and equipment, net514,821 501,958 
Operating lease right-of-use assets218,802 231,675 
Restricted investments49,389 40,891 
Other assets61,396 60,204 
Total assets$3,535,255 $3,643,304 
LIABILITIES AND STOCKHOLDERS’ (DEFICIT) EQUITY
Current liabilities:
Accounts payable$40,572 $73,426 
Accrued expenses510,579 395,174 
Operating lease liability41,581 40,548 
Deferred revenue144,208 149,483 
Liability related to the sale of future royalties35,851 37,079 
Total current liabilities772,791 695,710 
Operating lease liability, net of current portion266,323 281,347 
Deferred revenue, net of current portion132,930 152,360 
Convertible debt1,015,975  
Long-term debt 675,697 
Liability related to the sale of future royalties, net of current portion1,231,873 1,151,024 
Other liabilities183,001 98,963 
Total liabilities3,602,893 3,055,101 
Commitments and contingencies (Note 14)
Stockholders’ (deficit) equity:
Preferred stock, $0.01 par value per share, 5,000 shares authorized and no shares issued and outstanding as of September 30, 2022 and December 31, 2021
  
Common stock, $0.01 par value per share, 250,000 shares authorized; 122,991 shares issued and outstanding as of September 30, 2022; 120,182 shares issued and outstanding as of December 31, 2021
1,230 1,202 
Additional paid-in capital6,336,771 6,058,453 
Accumulated other comprehensive loss(43,783)(33,259)
Accumulated deficit(6,361,856)(5,438,193)
Total stockholders’ (deficit) equity(67,638)588,203 
Total liabilities and stockholders’ (deficit) equity$3,535,255 $3,643,304 
The accompanying notes are an integral part of these condensed consolidated financial statements.
5

ALNYLAM PHARMACEUTICALS, INC.
CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS
(In thousands, except per share amounts)
(Unaudited)
Three Months Ended
September 30,
Nine Months Ended
September 30,
2022202120222021
Statements of Operations
Revenues:
Net product revenues$232,267 $167,044 $632,654 $463,624 
Net revenues from collaborations29,297 20,136 64,267 121,328 
Royalty revenue2,742 453 5,462 800 
Total revenues264,306 187,633 702,383 585,752 
Operating costs and expenses:
Cost of goods sold36,507 28,091 94,002 81,370 
Cost of collaborations and royalties4,609 4,572 23,549 21,110 
Research and development245,371 194,572 620,976 563,106 
Selling, general and administrative235,859 142,075 560,314 434,257 
Total operating costs and expenses522,346 369,310 1,298,841 1,099,843 
Loss from operations(258,040)(181,677)(596,458)(514,091)
Other (expense) income:
Interest expense(41,084)(40,274)(126,055)(106,205)
Other (expense) income, net(30,233)17,715 (120,873)28,454 
Loss on the extinguishment of debt(76,586) (76,586) 
Total other expense, net(147,903)(22,559)(323,514)(77,751)
Loss before income taxes(405,943)(204,236)(919,972)(591,842)
Benefit from (provision for) income taxes23 (278)(3,691)(2,522)
Net loss$(405,920)$(204,514)$(923,663)$(594,364)
Net loss per common share - basic and diluted$(3.32)$(1.72)$(7.62)$(5.04)
Weighted-average common shares used to compute basic and diluted net loss per common share122,166 119,141 121,158 118,005 
Statements of Comprehensive Loss
Net loss$(405,920)$(204,514)$(923,663)$(594,364)
Other comprehensive (loss) income:
Unrealized loss on marketable securities(2,053)(64)(11,004)(312)
Foreign currency translation gain460 1,857 377 9,530 
Defined benefit pension plans, net of tax34 58 103 174 
Total other comprehensive (loss) income(1,559)1,851 (10,524)9,392 
Comprehensive loss$(407,479)$(202,663)$(934,187)$(584,972)




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

ALNYLAM PHARMACEUTICALS, INC.
CONDENSED CONSOLIDATED STATEMENTS OF STOCKHOLDERS’ (DEFICIT) EQUITY
(In thousands)
(Unaudited)
Common StockAdditional
Paid-in
Capital
Accumulated
Other
Comprehensive
Loss
Accumulated
Deficit
Total
Stockholders’
(Deficit) Equity
SharesAmount
Balance as of December 31, 2021120,182 $1,202 $6,058,453 $(33,259)$(5,438,193)$588,203 
Exercise of common stock options, net of tax withholdings524 5 28,054 — — 28,059 
Issuance of common stock under equity plans23 — — — —  
Stock-based compensation expense— — 30,051 — — 30,051 
Other comprehensive loss— — — (4,806)— (4,806)
Net loss— — — — (240,341)(240,341)
Balance as of March 31, 2022120,729 1,207 6,116,558 (38,065)(5,678,534)401,166 
Exercise of common stock options, net of tax withholdings192 2 13,890 — — 13,892 
Issuance of common stock under equity plans71 1 8,089 — — 8,090 
Stock-based compensation expense— — 34,453 — — 34,453 
Other comprehensive loss— — — (4,159)— (4,159)
Net loss— — — — (277,402)(277,402)
Balance as of June 30, 2022120,992 1,210 6,172,990 (42,224)(5,955,936)176,040 
Exercise of common stock options, net of tax withholdings1,657 17 153,259 — — 153,276 
Issuance of common stock under equity plans342 3 — — — 3 
Stock-based compensation expense— — 129,133 — — 129,133 
Purchase of capped calls related to convertible debt— — (118,611)— — (118,611)
Other comprehensive loss— — — (1,559)— (1,559)
Net loss— — — — (405,920)(405,920)
Balance as of September 30, 2022122,991 $1,230 $6,336,771 $(43,783)$(6,361,856)$(67,638)



The accompanying notes are an integral part of these condensed consolidated financial statements.
7

ALNYLAM PHARMACEUTICALS, INC.
CONDENSED CONSOLIDATED STATEMENTS OF STOCKHOLDERS’ (DEFICIT) EQUITY
(In thousands)
(Unaudited)
Common StockAdditional
Paid-in
Capital
Accumulated
Other
Comprehensive
Loss
Accumulated
Deficit
Total
Stockholders’
Equity
SharesAmount
Balance as of December 31, 2020116,427 $1,164 $5,644,074 $(43,622)$(4,585,369)$1,016,247 
Exercise of common stock options, net of tax withholdings614 6 47,028 — — 47,034 
Issuance of common stock under equity plans280 3 (3)— —  
Stock-based compensation expense— — 56,295 — — 56,295 
Other comprehensive income— — — 6,905 — 6,905 
Net loss— — — — (200,291)(200,291)
Balance as of March 31, 2021117,321 1,173 5,747,394 (36,717)(4,785,660)926,190 
Exercise of common stock options, net of tax withholdings993 10 75,439 — — 75,449 
Issuance of common stock under equity plans282 3 7,715 — — 7,718 
Stock-based compensation expense— — 32,928 — — 32,928 
Other comprehensive income— — — 636 — 636 
Net loss— — — — (189,559)(189,559)
Balance as of June 30, 2021118,596 1,186 5,863,476 (36,081)(4,975,219)853,362 
Exercise of common stock options, net of tax withholdings883 9 71,490 — — 71,499 
Issuance of common stock under equity plans1 —  — —  
Stock-based compensation expense— — 33,695 — — 33,695 
Other comprehensive income— — — 1,851 — 1,851 
Net loss— — — — (204,514)(204,514)
Balance as of September 30, 2021119,480 $1,195 $5,968,661 $(34,230)$(5,179,733)$755,893 



The accompanying notes are an integral part of these condensed consolidated financial statements.
8

ALNYLAM PHARMACEUTICALS, INC.
CONDENSED CONSOLIDATED STATEMENTS OF CASH FLOWS
(In thousands)
(Unaudited)
Nine Months Ended September 30,
20222021
Cash flows from operating activities:
Net loss$(923,663)$(594,364)
Non-cash adjustments to reconcile net loss to net cash used in operating activities:
Depreciation and amortization30,157 36,192 
Amortization and interest accretion related to operating leases30,508 32,051 
Non-cash interest expense on liability related to the sale of future royalties79,621 87,197 
Stock-based compensation187,882 121,135 
Realized and unrealized loss (gain) on marketable equity securities40,108 (61,273)
Loss on extinguishment of debt76,586  
Change in fair value of development derivative liability70,776 19,654 
Other20,607 15,568 
Changes in operating assets and liabilities:
Accounts receivable, net7,672 (42,350)
Inventory(15,158)(15,655)
Prepaid expenses and other assets(47,194)(48,765)
Accounts payable, accrued expenses and other liabilities89,152 48,738 
Operating lease liability(31,718)(29,586)
Deferred revenue(24,632)(60,094)
Net cash used in operating activities(409,296)(491,552)
Cash flows from investing activities:
Purchases of property, plant and equipment(50,424)(54,486)
Purchases of marketable securities(1,253,584)(973,146)
Sales and maturities of marketable securities1,626,848 1,172,100 
Proceeds from maturity of restricted investments89,951 25,975 
Purchases of restricted investments(98,451)(26,140)
Other investing activities(5,075)(4,198)
Net cash provided by investing activities309,265 140,105 
Cash flows from financing activities:
Proceeds from exercise of stock options and other types of equity, net202,646 201,227 
Proceeds from convertible debt, net1,016,888  
Repayment of term loan(762,107) 
Purchases of capped calls related to convertible debt(118,611) 
Proceeds from the sale of future royalties 500,000 
Proceeds from development derivative23,500 16,100 
Proceeds from term loan facility 250,000 
Other financing activities (12,500)
Net cash provided by financing activities362,316 954,827 
Effect of exchange rate changes on cash, cash equivalents and restricted cash(9,049)(5,968)
Net increase in cash, cash equivalents and restricted cash253,236 597,412 
Cash, cash equivalents and restricted cash, beginning of period822,153 499,046 
Cash, cash equivalents and restricted cash, end of period$1,075,389 $1,096,458 
Supplemental disclosure of cash flows:
Cash paid for interest$43,932 $17,703 
The accompanying notes are an integral part of these condensed consolidated financial statements.
9

ALNYLAM PHARMACEUTICALS, INC.
NOTES TO CONDENSED CONSOLIDATED FINANCIAL STATEMENTS
(Unaudited)


1. NATURE OF BUSINESS
Alnylam Pharmaceuticals, Inc. (also referred to as Alnylam, we, our or us) commenced operations on June 14, 2002 as a biopharmaceutical company seeking to develop and commercialize novel therapeutics based on ribonucleic acid interference, or RNAi. We are committed to the advancement of our company strategy of building a multi-product, global, commercial biopharmaceutical company with a deep and sustainable clinical pipeline of RNAi therapeutics for future growth and a robust, organic research engine for sustainable innovation and great potential for patient impact. Since inception, we have focused on discovering, developing and commercializing RNAi therapeutics by establishing and maintaining a strong intellectual property position in the RNAi field, establishing strategic alliances with leading pharmaceutical and life sciences companies, generating revenues through licensing agreements, and ultimately developing and commercializing RNAi therapeutics globally, either independently or with our strategic partners. We have devoted substantially all of our efforts to business planning, research, development, manufacturing and early commercial efforts, acquiring, filing and expanding intellectual property rights, recruiting management and technical staff, and raising capital.
In early 2021, we launched our Alnylam P5x25 strategy, which focuses on our planned transition to a top five biotech company by the end of 2025. With Alnylam P5x25, we aim to deliver transformative rare and prevalent disease medicines for patients around the world through sustainable innovation, delivering exceptional financial performance and driving profitability.
As of September 30, 2022, we have five products that have received marketing approval, including one partnered product, and multiple late-stage investigational programs advancing towards potential commercialization. As of September 30, 2022, we generated worldwide product revenues from four commercialized products, ONPATTRO, GIVLAARI, OXLUMO and AMVUTTRA, primarily in the U.S., Europe and Japan.
2. BASIS OF PRESENTATION AND PRINCIPLES OF CONSOLIDATION
The accompanying condensed consolidated financial statements of Alnylam are unaudited and have been prepared in accordance with accounting principles generally accepted in the United States of America, or GAAP, applicable to interim periods and, in the opinion of management, include all normal and recurring adjustments that are necessary to state fairly the results of operations for the reported periods. Our condensed consolidated financial statements have also been prepared on a basis substantially consistent with, and should be read in conjunction with, our audited consolidated financial statements for the year ended December 31, 2021, which were included in our Annual Report on Form 10-K that was filed with the Securities and Exchange Commission on February 10, 2022. The year-end condensed consolidated balance sheet data was derived from our audited financial statements but does not include all disclosures required by GAAP. The results of our operations for any interim period are not necessarily indicative of the results of our operations for any other interim period or for a full fiscal year.
The accompanying condensed consolidated financial statements reflect the operations of Alnylam and our wholly-owned subsidiaries. All intercompany accounts and transactions have been eliminated.
Our significant accounting policies are described in Note 2 of the Notes to Consolidated Financial Statements included in our Annual Report on Form 10-K for the year ended December 31, 2021. Updates to our significant accounting policies, including the convertible debt policy, are discussed below.
Use of Estimates
The preparation of financial statements in conformity with GAAP requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities and the disclosure of contingent assets and liabilities at the date of the condensed consolidated financial statements and the reported amounts of revenues and expenses during the reporting period. In our condensed consolidated financial statements, we use estimates and assumptions related to our inventory valuation and related reserves, liability related to the sale of future royalties, development derivative liability, income taxes, revenue recognition, research and development expenses, and stock-based compensation. We base our estimates on historical experience and on various other assumptions that we believe to be reasonable. Actual results could differ from those estimates.
The full extent to which the ongoing COVID-19 pandemic will directly or indirectly impact our business, results of operations and financial condition, including sales, expenses, reserves and allowances, the supply of our products and product candidates, clinical trials and research and development costs, will depend on future developments that are highly uncertain, including as a result of new information that may emerge concerning COVID-19 and variants thereof, and the actions taken to contain or treat it or vaccinate against it, as well as the economic impact on local, regional, national and international customers and markets. We have made estimates of the impact of COVID-19 within our financial statements and there may be changes to those estimates in future periods. Actual results may differ from these estimates.
10

ALNYLAM PHARMACEUTICALS, INC.
NOTES TO CONDENSED CONSOLIDATED FINANCIAL STATEMENTS
(Unaudited)
Liquidity
Based on our current operating plan, we believe that our cash, cash equivalents and marketable securities as of September 30, 2022, together with the cash we expect to generate from product sales and under our current alliances, including milestones and royalties on Leqvio sales, will be sufficient to enable us to advance our Alnylam P5x25 strategy for at least the next 12 months from the filing of this Quarterly Report on Form 10-Q.
Recently Adopted Accounting Pronouncements
In August 2020, the Financial Accounting Standards Board, or FASB, issued Accounting Standards Update, or ASU, 2020-06, Debt—Debt with Conversion and Other Options (Subtopic 470-20) and Derivatives and Hedging—Contracts in Entity’s Own Equity (Subtopic 815-40): Accounting for Convertible Instruments and Contracts in an Entity’s Own Equity, which simplifies the accounting for certain financial instruments with characteristics of liabilities and equity, including convertible instruments and contracts in an entity’s own equity. Specifically, ASU 2020-06 simplifies accounting for the issuance of convertible instruments by removing certain separation models required under existing guidance. In addition, the standard removes certain settlement conditions that are required for equity contracts to qualify for the derivative scope exception and requires use of the “if-converted” method when calculating the dilutive impact of convertible debt on earnings per share, or EPS. ASU 2020-06 is effective for financial statements issued for fiscal years beginning after December 15, 2021, and interim periods within those fiscal years, with early adoption permitted. We adopted the new standard on January 1, 2022 and there was no impact on the date of adoption. During the third quarter of 2022, we issued convertible notes as described in Note 10, Convertible Debt.
3. NET PRODUCT REVENUES
Net product revenues consist of the following:
Three Months Ended
September 30,
Nine Months Ended
September 30,
(In thousands)2022202120222021
ONPATTRO
United States$67,196 $51,247 $200,588 $153,109 
Europe57,217 51,019 167,185 136,576 
Rest of World20,537 18,051 67,614 46,422 
Total144,950 120,317 435,387 336,107 
AMVUTTRA
United States25,060  25,060  
Europe169  169  
Total25,229  25,229  
GIVLAARI
United States31,169 22,372 84,505 62,502 
Europe12,477 7,568 36,059 22,461 
Rest of World2,013 1,893 5,522 2,173 
Total45,659 31,833 126,086 87,136 
OXLUMO
United States6,383 5,236 18,916 13,156 
Europe9,348 9,658 25,099 27,225 
Rest of World698  1,937  
Total16,429 14,894 45,952 40,381 
Total net product revenues$232,267 $167,044 $632,654 $463,624 
11

ALNYLAM PHARMACEUTICALS, INC.
NOTES TO CONDENSED CONSOLIDATED FINANCIAL STATEMENTS
(Unaudited)
The following table presents the balance of our receivables related to our net product revenues:
(In thousands)As of September 30,
2022
As of December 31,
2021
Receivables included in “Accounts receivable, net”$160,162 $124,906 

4. NET REVENUES FROM COLLABORATIONS
Net revenues from collaborations consist of the following:
Three Months Ended September 30,Nine Months Ended September 30,
(In thousands)2022202120222021
Regeneron Pharmaceuticals$21,979 $14,161 $34,405 $90,908 
Novartis AG5,803 4,160 27,472 21,179 
Vir Biotechnology441 1,233 1,137 8,033 
Other1,074 582 1,253 1,208 
Total$29,297 $20,136 $64,267 $121,328 
    
The following table presents the balance of our receivables and contract liabilities related to our collaboration agreements:
(In thousands)As of September 30, 2022As of December 31, 2021
Receivables included in “Accounts receivable, net”$21,933 $73,266 
Contract liabilities included in “Deferred revenue”$73,625 $88,627 
We recognized revenue of $11.7 million and $17.0 million in the three and nine months ended September 30, 2022, respectively, and revenue of $12.7 million and $65.2 million in the three and nine months ended September 30, 2021, respectively, that was included in the contract liability balance at the beginning of the period.
In order to determine revenue recognized in the period from contract liabilities, we first allocate revenue to the individual contract liability balance outstanding at the beginning of the period until the revenue exceeds that balance. If additional consideration is received on those contracts in subsequent periods, we assume all revenue recognized in the reporting period first applies to the beginning contract liability as opposed to a portion applying to the new consideration for the period.
The following table provides research and development expenses incurred by type, for which we recognize net revenue, that are directly attributable to our collaboration agreements, by collaboration partner:
Three Months Ended September 30,
20222021
(In thousands)Clinical Trial and ManufacturingExternal ServicesOtherClinical Trial and ManufacturingExternal ServicesOther
Regeneron Pharmaceuticals$9,812 $1,154 $8,982 $3,152 $285 $10,964 
Other 357  572 37 728 
Total$9,812 $1,511 $8,982 $3,724 $322 $11,692 

Nine Months Ended September 30,
20222021
(In thousands)Clinical Trial and ManufacturingExternal ServicesOtherClinical Trial and ManufacturingExternal ServicesOther
Regeneron Pharmaceuticals$12,926 $2,141 $27,935 $17,343 $547 $34,082 
Other156 679 337 2,976 745 3,844 
Total$13,082 $2,820 $28,272 $20,319 $1,292 $37,926 
12

ALNYLAM PHARMACEUTICALS, INC.
NOTES TO CONDENSED CONSOLIDATED FINANCIAL STATEMENTS
(Unaudited)
The research and development expenses incurred for the agreements included in the table above consist of costs incurred for (i) clinical expenses, including manufacturing of clinical product, (ii) external services including consulting services and lab supplies and services, and (iii) other expenses, including professional services, facilities and overhead allocations, and a reasonable estimate of compensation and related costs as billed to our counterparties, for which we recognize net revenues from collaborations. For the three and nine months ended September 30, 2022 and 2021, we did not incur material selling, general and administrative expenses related to our collaboration agreements.
In addition, we recognized a reduction to our research and development expenses of $2.3 million and $9.7 million for the three and nine months ended September 30, 2022, respectively, and of $3.3 million and $12.7 million for the three and nine months ended September 30, 2021, respectively, from cost reimbursement due under certain of our collaboration agreements with Regeneron Pharmaceuticals, Inc., or Regeneron, accounted for under Accounting Standards Codification, or ASC, Topic 808, Collaborative Arrangements, or ASC 808.
Product Alliances
Regeneron Pharmaceuticals, Inc.
In April 2019, we entered into a global, strategic collaboration with Regeneron to discover, develop and commercialize RNAi therapeutics for a broad range of diseases by addressing therapeutic targets expressed in the eye and central nervous system, or CNS, in addition to a select number of targets expressed in the liver, which we refer to as the Regeneron Collaboration. The Regeneron Collaboration is governed by a Master Agreement, referred to as the Regeneron Master Agreement, which became effective on May 21, 2019. In connection with the Regeneron Master Agreement, we and Regeneron entered into (i) a binding co-co collaboration term sheet covering the continued development of cemdisiran, our C5 small interfering RNA, or siRNA, currently in Phase 2 development for C5 complement-mediated diseases, as a monotherapy and (ii) a binding license term sheet to evaluate anti-C5 antibody-siRNA combinations for C5 complement-mediated diseases including evaluating the combination of Regeneron’s pozelimab (REGN3918), currently in Phase 3 development, and cemdisiran. The C5 co-co collaboration and license agreements were executed in August 2019.
Under the terms of the Regeneron Collaboration, we are working exclusively with Regeneron to discover RNAi therapeutics for eye and CNS diseases for an initial research period of approximately five years, which we refer to as the Initial Research Term. Regeneron has an option to extend the Initial Research Term (referred to as the Research Term Extension Period, and together with the Initial Research Term, the Research Term) for up to an additional five years, for a research term extension fee of up to $400.0 million. The Regeneron Collaboration also covers a select number of RNAi therapeutic programs designed to target genes expressed in the liver, including our previously announced collaboration with Regeneron to identify RNAi therapeutics for the chronic liver disease nonalcoholic steatohepatitis. We retain broad global rights to all of our other unpartnered liver-directed clinical and pre-clinical pipeline programs. The Regeneron Collaboration is governed by a joint steering committee that is comprised of an equal number of representatives from each party.
Regeneron leads development and commercialization for all programs targeting eye diseases (subject to limited exceptions), entitling us to certain potential milestone and royalty payments pursuant to the terms of a license agreement, the form of which has been agreed upon by the parties. We and Regeneron are alternating leadership on CNS and liver programs covered by the Regeneron Collaboration, with the lead party retaining global development and commercial responsibility. For such CNS and liver programs, both we and Regeneron have the option at lead candidate selection to enter into a co-co collaboration agreement, the form of which has been agreed upon by the parties, whereby both companies will share equally all costs of, and profits from, all development and commercialization activities under the program. If the non-lead party elects to not enter into a co-co collaboration agreement with respect to a given CNS or liver program, we and Regeneron will enter into a license agreement with respect to such program and the lead party will be the “Licensee” for the purposes of the license agreement. If the lead party for a CNS or liver program elects to not enter into the co-co collaboration agreement, then we and Regeneron will enter into a license agreement with respect to such program and leadership of the program will transfer to the other party and the former non-lead party will be the “Licensee” for the purposes of the license agreement.
With respect to the programs directed to C5 complement-mediated diseases, we retain control of cemdisiran monotherapy development, and Regeneron is leading combination product development. Under the C5 co-co collaboration agreement, we and Regeneron equally share costs and potential future profits on any monotherapy program. Under the C5 license agreement, for cemdisiran to be used as part of a combination product, Regeneron is solely responsible for all development and commercialization costs and we will receive low double-digit royalties and commercial milestones of up to $325.0 million on any potential combination product sales. The C5 co-co collaboration agreement, the C5 license agreement, and the Master Agreement have been combined for accounting purposes and treated as a single agreement.
In connection with the Regeneron Master Agreement, Regeneron made an upfront payment of $400.0 million. We are also eligible to receive up to an additional $200.0 million in milestone payments upon achievement of certain criteria during early clinical development for eye and CNS programs. We and Regeneron plan to advance programs directed to up to 30 targets in
13

ALNYLAM PHARMACEUTICALS, INC.
NOTES TO CONDENSED CONSOLIDATED FINANCIAL STATEMENTS
(Unaudited)
the first five years under the Regeneron Collaboration during the Initial Research Term. For each program, Regeneron will provide us with $2.5 million in funding at program initiation and an additional $2.5 million at lead candidate identification, with the potential for approximately $30.0 million in annual discovery funding to us as the Regeneron Collaboration reaches steady state.
Regeneron has the right to terminate the Regeneron Master Agreement for convenience upon ninety days’ notice. The termination of the Regeneron Master Agreement does not affect the term of any license agreement or co-co collaboration agreement then in effect. In addition, either party may terminate the Regeneron Master Agreement for a material breach by, or insolvency of, the other party. Unless earlier terminated pursuant to its terms, the Regeneron Master Agreement will remain in effect with respect to each program until (a) such program becomes a terminated program or (b) the parties enter into a license agreement or co-co collaboration agreement with respect to such program. The Regeneron Master Agreement includes various representations, warranties, covenants, dispute escalation and resolution mechanisms, indemnities and other provisions customary for transactions of this nature.
For any license agreement subsequently entered into, the licensee will generally be responsible for its own costs and expenses incurred in connection with the development and commercialization of the collaboration products. The licensee will pay to the licensor certain development and/or commercialization milestone payments totaling up to $150.0 million for each collaboration product. In addition, following the first commercial sale of the applicable collaboration product under a license agreement, the licensee is required to make certain tiered royalty payments, ranging from low double-digits up to 20%, to the licensor based on the aggregate annual net sales of the collaboration product, subject to customary reductions.
For any co-co collaboration agreement subsequently entered into, we and Regeneron will share equally all costs of, and profits from, development and commercialization activities. Reimbursement of our share of costs will be recognized as a reduction to research and development expense in the condensed consolidated statements of operations and comprehensive loss. In the event that a party exercises its opt-out right, the lead party will be responsible for all costs and expenses incurred in connection with the development and commercialization of the collaboration products under the applicable co-co collaboration agreement, subject to continued sharing of costs through defined points. If a party exercises its opt-out right, following the first commercial sale of the applicable collaboration product under a co-co collaboration agreement, the lead party is required to make certain tiered royalty payments, ranging from low double-digits up to 20%, to the other party based on the aggregate annual net sales of the collaboration product and the timing of the exercise of the opt-out right, subject to customary reductions and a reduction for opt-out transition costs.
Due to the uncertainty of pharmaceutical development and the high historical failure rates generally associated with drug development, we may not receive any milestone or royalty payments from Regeneron under the Regeneron Master Agreement, the C5 license agreement, or any future license agreement, or under any co-co collaboration agreement in the event we exercise our opt-out right.
Our obligations under the Regeneron Collaboration include: (i) a research license and research services, collectively referred to as the Research Services Obligation; (ii) a worldwide license to cemdisiran for combination therapies, and manufacturing and supply, and development service obligations, collectively referred to as the C5 License Obligation; and (iii) development, manufacturing and commercialization activities for cemdisiran monotherapies, referred to as the C5 Co-Co Obligation.
The research license is not distinct from the research services primarily as a result of Regeneron being unable to benefit on its own or with other resources reasonably available, as the license is providing access to specialized expertise, particularly as it relates to RNAi technology that is not available in the marketplace. Similarly, the worldwide license to cemdisiran for combination therapies is not distinct from the manufacturing and supply, and development service obligations, as Regeneron cannot benefit on its own from the value of the license without receipt of supply.
Separately, the cemdisiran monotherapy co-co collaboration agreement is under the scope of ASC 808 as we and Regeneron are both active participants in the development and manufacturing activities and are exposed to significant risks and rewards that are dependent on commercial success of the activities of the arrangement. The development and manufacturing activities are a combined unit of account under the scope of ASC 808 and are not deliverables under ASC Topic 606, Revenue from Contracts with Customers, or ASC 606.
The total transaction price is comprised of the $400.0 million upfront payment and additional variable consideration related to research, development, manufacturing and supply activities related to the Research Services Obligation and the C5 License Obligation. We utilized the expected value method to determine the amount of reimbursement for these activities. We determined that any variable consideration related to sales-based royalties and milestones related to the worldwide license to cemdisiran for combination therapies is deemed to be constrained and therefore has been excluded from the transaction price. In addition, we are eligible to receive future milestones upon the achievement of certain criteria during early clinical development for the eye and CNS programs. We are also eligible to receive royalties on future commercial sales for certain eye, CNS or liver
14

ALNYLAM PHARMACEUTICALS, INC.
NOTES TO CONDENSED CONSOLIDATED FINANCIAL STATEMENTS
(Unaudited)
targets, if any; however, these amounts are excluded from variable consideration under the Regeneron Collaboration as we are only eligible to receive such amounts if, after a drug candidate is identified, the form of license agreement is subsequently executed resulting in a license that is granted to Regeneron. Any such subsequently granted license would represent a separate transaction under ASC 606.
We allocated the initial transaction price to each unit of account based on the applicable accounting guidance as follows, in thousands: