10-Q
Q30001515673http://fasb.org/us-gaap/2021-01-31#OtherNoncurrentAssetsMemberfalsehttp://fasb.org/us-gaap/2021-01-31#PrepaidExpenseAndOtherAssetsCurrenthttp://fasb.org/us-gaap/2021-01-31#OtherNoncurrentAssetsMember--12-31http://fasb.org/us-gaap/2021-01-31#PrepaidExpenseAndOtherAssetsCurrent0001515673us-gaap:FairValueInputsLevel1Member2021-09-300001515673rare:LicenseAgreementMemberrare:REGENXBIOIncorporatedMember2020-03-012020-03-310001515673rare:CollaborationAndRoyaltyMemberrare:KyowaKirinCollaborationMember2021-07-012021-09-300001515673us-gaap:RoyaltyMember2021-07-012021-09-300001515673us-gaap:AccumulatedOtherComprehensiveIncomeMember2020-07-012020-09-300001515673rare:DaiichiSankyoCoLtdMember2021-01-012021-09-300001515673rare:CrysvitaMemberus-gaap:ProductMember2020-01-012020-09-300001515673srt:EuropeMember2020-07-012020-09-300001515673us-gaap:ProductMember2021-01-012021-09-300001515673us-gaap:RetainedEarningsMember2020-01-012020-09-300001515673rare:ConvertiblePromissoryNoteMember2021-01-012021-09-300001515673us-gaap:RoyaltyMember2020-01-012020-09-300001515673rare:EuropeanUnionAndSwitzerlandAndTurkeyMemberrare:CollaborationAndRoyaltyMemberrare:KyowaKirinCollaborationMember2021-01-012021-09-300001515673rare:UnitedStatesAndCanadaMemberrare:CollaborationAndRoyaltyMemberrare:KyowaKirinCollaborationMember2021-07-012021-09-300001515673rare:CollaborationAndLicenseMemberrare:CrysvitaMember2020-07-012020-09-300001515673rare:InvestmentsInArcturusAndSolidCommonStockMember2020-12-310001515673us-gaap:AccumulatedOtherComprehensiveIncomeMember2021-09-300001515673rare:LicenseAndTechnologyAccessAgreementMemberrare:DaiichiSankyoCoLtdMemberrare:LicensesAgreementMember2021-01-012021-09-300001515673rare:CollaborationAndLicenseMemberrare:EuropeanUnionAndSwitzerlandAndTurkeyMemberrare:CrysvitaMember2020-01-012020-09-300001515673rare:CollaborationAndLicenseMember2020-01-012020-09-300001515673rare:RoyaltyPurchaseAgreementMemberrare:RPIFinanceTrustMembersrt:MaximumMember2019-12-310001515673us-gaap:LicenseAgreementTermsMemberrare:UnitedStatesAndCanadaMemberrare:KyowaKirinCollaborationMember2021-01-012021-09-300001515673us-gaap:EmployeeStockMember2021-07-012021-09-300001515673us-gaap:FairValueInputsLevel2Memberrare:USGovernmentTreasuryAndAgencySecuritiesMember2021-09-300001515673rare:ArcturusResearchCollaborationAndLicenseAgreementMember2021-07-012021-09-300001515673us-gaap:LicenseAgreementTermsMemberus-gaap:ProductMemberrare:KyowaKirinCollaborationMember2021-01-012021-09-300001515673us-gaap:RetainedEarningsMember2021-01-012021-09-300001515673us-gaap:RoyaltyMemberrare:CrysvitaMember2021-01-012021-09-300001515673srt:NorthAmericaMember2021-01-012021-09-300001515673us-gaap:ProductMemberrare:MepseviiMember2021-01-012021-09-300001515673rare:SolidBiosciencesIncMember2021-01-012021-09-3000015156732019-12-310001515673us-gaap:AccumulatedOtherComprehensiveIncomeMember2021-01-012021-09-300001515673us-gaap:AccumulatedOtherComprehensiveIncomeMember2020-09-300001515673us-gaap:LicenseAgreementTermsMemberrare:ProfitShareRevenueAndRoyaltiesMemberrare:KyowaKirinCollaborationMember2021-09-300001515673rare:DaiichiSankyoCoLtdMember2021-07-012021-09-300001515673us-gaap:SellingGeneralAndAdministrativeExpensesMember2020-07-012020-09-300001515673rare:RoyaltyPurchaseAgreementMemberrare:RPIFinanceTrustMembersrt:MinimumMember2019-12-310001515673us-gaap:AdditionalPaidInCapitalMember2020-01-012020-09-300001515673us-gaap:LicenseAgreementTermsMemberus-gaap:ProductMemberrare:KyowaKirinCollaborationMember2020-07-012020-09-300001515673rare:KyowaKirinCollaborationMember2021-07-012021-09-300001515673us-gaap:FairValueInputsLevel2Memberrare:USGovernmentTreasuryAndAgencySecuritiesMember2020-12-310001515673rare:CollaborationAndLicenseMemberrare:DaiichiSankyoCoLtdMember2020-07-012020-09-300001515673us-gaap:CommonStockMember2020-12-310001515673us-gaap:FairValueInputsLevel2Memberrare:CertificateOfDepositsAndTimeDepositsMember2021-09-300001515673rare:EuropeanUnionAndSwitzerlandAndTurkeyMemberus-gaap:RoyaltyMemberrare:KyowaKirinCollaborationMember2020-07-012020-09-300001515673us-gaap:RoyaltyMember2020-07-012020-09-300001515673us-gaap:PrepaidExpensesAndOtherCurrentAssetsMemberus-gaap:LicenseAgreementTermsMemberrare:KyowaKirinCollaborationMember2020-12-310001515673us-gaap:AccumulatedOtherComprehensiveIncomeMember2020-06-300001515673us-gaap:LicenseAgreementTermsMemberrare:CommercialAndDevelopmentActivityReimbursementsMemberrare:KyowaKirinCollaborationMember2021-09-300001515673rare:UnderwrittenPublicOfferingMember2020-10-310001515673us-gaap:CommonStockMemberrare:SolidBiosciencesIncMember2021-01-012021-09-300001515673rare:ArcturusResearchCollaborationAndLicenseAgreementMember2021-09-300001515673us-gaap:AssetBackedSecuritiesMemberus-gaap:FairValueInputsLevel2Member2020-12-310001515673us-gaap:RetainedEarningsMember2020-12-310001515673rare:LicenseAndTechnologyAccessAgreementMemberrare:DaiichiSankyoCoLtdMember2021-01-012021-09-300001515673us-gaap:RetainedEarningsMember2020-09-300001515673us-gaap:RetainedEarningsMember2020-06-300001515673us-gaap:CorporateBondSecuritiesMember2021-09-300001515673rare:CollaborationAndLicenseMemberrare:EuropeanUnionAndSwitzerlandAndTurkeyMemberrare:CrysvitaMember2021-07-012021-09-300001515673us-gaap:CommonStockMemberrare:SolidBiosciencesIncMember2020-12-310001515673us-gaap:LicenseAgreementTermsMemberrare:CommercialAndDevelopmentActivityReimbursementsMemberrare:KyowaKirinCollaborationMember2020-12-310001515673us-gaap:LicenseAgreementTermsMemberus-gaap:ProductMemberrare:KyowaKirinCollaborationMember2021-07-012021-09-300001515673us-gaap:AdditionalPaidInCapitalMember2021-07-012021-09-300001515673us-gaap:EmployeeStockMember2020-07-012020-09-300001515673us-gaap:LicenseAgreementTermsMembersrt:ScenarioForecastMemberrare:KyowaKirinCollaborationMember2013-08-012022-12-310001515673rare:CollaborationAndLicenseMemberrare:CrysvitaMember2021-07-012021-09-300001515673rare:ArcturusResearchCollaborationAndLicenseAgreementMember2019-12-310001515673rare:UnitedStatesAndCanadaMemberrare:CollaborationAndRoyaltyMemberrare:KyowaKirinCollaborationMember2021-01-012021-09-300001515673us-gaap:CommonStockMember2021-09-300001515673us-gaap:AdditionalPaidInCapitalMember2020-06-3000015156732021-10-280001515673us-gaap:CommonStockMemberrare:SolidBiosciencesIncMember2021-07-012021-09-300001515673us-gaap:RetainedEarningsMember2019-12-310001515673us-gaap:FairValueInputsLevel1Memberus-gaap:MoneyMarketFundsMember2021-09-300001515673us-gaap:ResearchAndDevelopmentExpenseMember2021-07-012021-09-300001515673srt:LatinAmericaMember2021-01-012021-09-300001515673rare:OptionsToPurchaseCommonStockAndRSUsMember2021-01-012021-09-300001515673rare:DaiichiSankyoCoLtdMemberrare:SublicenseAgreementMember2021-01-012021-09-300001515673rare:EuropeanUnionAndSwitzerlandAndTurkeyMemberrare:CollaborationAndRoyaltyMemberrare:KyowaKirinCollaborationMember2021-07-012021-09-300001515673us-gaap:FairValueInputsLevel2Memberus-gaap:CommercialPaperNotIncludedWithCashAndCashEquivalentsMember2020-12-310001515673rare:CollaborationAndLicenseMemberrare:DaiichiSankyoCoLtdMember2021-01-012021-09-300001515673rare:TwoThousandFourteenIncentivePlanMember2021-09-300001515673rare:CommonStockWarrantsMember2020-01-012020-09-300001515673us-gaap:LicenseAgreementTermsMembersrt:ScenarioForecastMemberrare:KyowaKirinCollaborationMember2023-01-012023-01-010001515673rare:OtherMember2021-09-300001515673rare:DaiichiSankyoCoLtdMemberrare:StockPurchaseAgreementMember2020-01-012020-03-3100015156732020-09-300001515673rare:TwoThousandFourteenIncentivePlanMember2021-01-012021-09-300001515673us-gaap:ResearchAndDevelopmentExpenseMember2021-01-012021-09-300001515673rare:CollaborationAndRoyaltyMemberrare:KyowaKirinCollaborationMember2020-01-012020-09-300001515673rare:ArcturusResearchCollaborationAndLicenseAgreementMember2020-01-012020-09-300001515673rare:OptionsToPurchaseCommonStockAndRSUsMember2020-07-012020-09-300001515673us-gaap:CommonStockMember2019-12-310001515673us-gaap:RoyaltyMember2021-01-012021-09-300001515673rare:OpenMarketSaleMember2021-01-012021-09-300001515673rare:ArcturusResearchCollaborationAndLicenseAgreementMember2019-06-300001515673rare:CrysvitaMemberus-gaap:ProductMember2021-07-012021-09-300001515673us-gaap:RoyaltyMemberrare:CrysvitaMember2021-07-012021-09-300001515673us-gaap:ProductMemberrare:DojolviMember2020-07-012020-09-300001515673rare:ArcturusResearchCollaborationAndLicenseAgreementMember2021-08-3100015156732020-01-012020-12-310001515673rare:OptionsToPurchaseCommonStockAndRSUsMember2021-07-012021-09-300001515673rare:CollaborationAndRoyaltyMemberrare:KyowaKirinCollaborationMember2020-07-012020-09-300001515673rare:RoyaltyPurchaseAgreementMemberrare:RPIFinanceTrustMember2021-09-3000015156732021-07-012021-09-300001515673us-gaap:CommercialPaperNotIncludedWithCashAndCashEquivalentsMember2021-09-300001515673rare:USGovernmentTreasuryAndAgencySecuritiesMember2020-12-310001515673us-gaap:AdditionalPaidInCapitalMember2020-09-300001515673rare:StockPurchaseAgreementMemberrare:DaiichiSankyoCoLtdMember2021-09-300001515673rare:InvestmentsInArcturusAndSolidCommonStockMember2021-09-300001515673us-gaap:ProductMemberrare:MepseviiMember2020-01-012020-09-300001515673rare:InvestmentsInArcturusAndSolidCommonStockMemberus-gaap:FairValueInputsLevel1Member2020-12-3100015156732020-07-012020-09-300001515673us-gaap:FairValueInputsLevel2Member2021-09-300001515673rare:OpenMarketSaleMember2021-05-012021-05-310001515673rare:ConvertiblePromissoryNoteMember2021-09-300001515673us-gaap:CommonStockMemberrare:SolidBiosciencesIncMember2020-10-012020-12-310001515673us-gaap:CommonStockMemberrare:ArcturusResearchCollaborationAndLicenseAgreementMember2021-07-012021-09-300001515673rare:ArcturusResearchCollaborationAndLicenseAgreementMember2020-05-012020-05-3100015156732021-06-300001515673us-gaap:CommonStockMemberrare:SolidBiosciencesIncMember2021-09-300001515673rare:ArcturusResearchCollaborationAndLicenseAgreementMember2020-12-310001515673us-gaap:AccumulatedOtherComprehensiveIncomeMember2021-06-300001515673us-gaap:AdditionalPaidInCapitalMember2020-07-012020-09-300001515673rare:CollaborationAndLicenseMember2020-07-012020-09-3000015156732021-09-300001515673srt:NorthAmericaMember2020-07-012020-09-300001515673us-gaap:RetainedEarningsMember2021-06-300001515673us-gaap:SellingGeneralAndAdministrativeExpensesMember2020-01-012020-09-300001515673us-gaap:AccumulatedOtherComprehensiveIncomeMember2020-01-012020-09-300001515673us-gaap:RetainedEarningsMember2020-07-012020-09-300001515673us-gaap:RetainedEarningsMember2021-07-012021-09-300001515673us-gaap:FairValueInputsLevel1Memberus-gaap:MoneyMarketFundsMember2020-12-310001515673rare:ArcturusResearchCollaborationAndLicenseAgreementMember2021-01-012021-09-300001515673rare:EuropeanUnionAndSwitzerlandAndTurkeyMemberus-gaap:RoyaltyMemberrare:KyowaKirinCollaborationMember2021-07-012021-09-300001515673rare:GeneTxBiotherapeuticsLLCMemberrare:ProgramAgreementAndUnitholderOptionAgreementMember2021-01-012021-09-300001515673us-gaap:AdditionalPaidInCapitalMember2021-06-300001515673rare:LicenseAgreementMemberrare:REGENXBIOIncorporatedMember2020-01-012020-03-3100015156732020-12-310001515673us-gaap:ProductMember2021-07-012021-09-3000015156732020-01-012020-09-300001515673us-gaap:CommonStockMemberrare:ArcturusResearchCollaborationAndLicenseAgreementMember2020-07-012020-09-300001515673rare:DaiichiSankyoCoLtdMember2021-09-300001515673rare:SolidBiosciencesIncMember2020-10-310001515673srt:LatinAmericaMember2021-07-012021-09-300001515673rare:MahziTherapeuticsIncMemberrare:ConvertiblePromissoryNoteMember2021-09-300001515673us-gaap:ProductMemberrare:DojolviMember2021-07-012021-09-300001515673rare:DaiichiSankyoCoLtdMemberrare:StockPurchaseAgreementMember2021-01-012021-09-300001515673srt:EuropeMember2021-07-012021-09-300001515673us-gaap:FairValueInputsLevel2Memberrare:DebtSecuritiesInGovernmentSponsoredEntitiesMember2021-09-300001515673rare:CollaborationAndLicenseMemberrare:DaiichiSankyoCoLtdMember2021-07-012021-09-300001515673rare:MahziTherapeuticsIncMemberus-gaap:PreferredStockMemberrare:ConvertiblePromissoryNoteMemberus-gaap:SubsequentEventMember2021-10-012021-10-310001515673us-gaap:FairValueInputsLevel1Member2020-12-310001515673us-gaap:FairValueInputsLevel2Memberus-gaap:CorporateBondSecuritiesMember2021-09-300001515673us-gaap:AdditionalPaidInCapitalMember2021-01-012021-09-300001515673us-gaap:FairValueInputsLevel2Memberrare:TimeDepositsMember2020-12-310001515673us-gaap:SellingGeneralAndAdministrativeExpensesMember2021-01-012021-09-300001515673us-gaap:CommonStockMemberrare:ArcturusResearchCollaborationAndLicenseAgreementMember2021-01-012021-09-300001515673us-gaap:CommonStockMember2021-07-012021-09-300001515673rare:CrysvitaMemberus-gaap:ProductMember2020-07-012020-09-300001515673rare:ConvertiblePromissoryNoteMember2020-07-012020-07-310001515673rare:RoyaltyPurchaseAgreementMemberrare:RPIFinanceTrustMember2021-01-012021-09-300001515673us-gaap:FairValueInputsLevel2Memberus-gaap:CommercialPaperNotIncludedWithCashAndCashEquivalentsMember2021-09-300001515673us-gaap:EmployeeStockMember2021-01-012021-09-300001515673rare:UnderwrittenPublicOfferingMember2020-10-012020-10-310001515673rare:LicenseAgreementMembersrt:MaximumMemberrare:REGENXBIOIncorporatedMember2020-03-310001515673rare:SolidBiosciencesIncMemberrare:CollaborationAgreementMember2020-10-012020-10-310001515673us-gaap:ResearchAndDevelopmentExpenseMember2020-07-012020-09-300001515673us-gaap:CommonStockMemberrare:ArcturusResearchCollaborationAndLicenseAgreementMember2020-12-310001515673us-gaap:AssetBackedSecuritiesMember2021-09-300001515673us-gaap:AccumulatedOtherComprehensiveIncomeMember2020-12-310001515673srt:MaximumMemberrare:ArcturusResearchCollaborationAndLicenseAgreementMember2019-06-012019-06-300001515673us-gaap:AdditionalPaidInCapitalMember2019-12-310001515673us-gaap:CostOfSalesMember2020-07-012020-09-300001515673us-gaap:CostOfSalesMember2021-01-012021-09-300001515673us-gaap:ProductMember2020-01-012020-09-300001515673rare:UnderwrittenPublicOfferingMember2020-01-012020-12-310001515673srt:MaximumMemberrare:BayerHealthCareLLCMemberrare:ResearchDevelopmentAndCommercializeAAVGeneTherapyProductsAgreementMember2021-09-300001515673us-gaap:AssetBackedSecuritiesMemberus-gaap:FairValueInputsLevel2Member2021-09-300001515673us-gaap:MoneyMarketFundsMember2020-12-310001515673rare:CrysvitaMemberus-gaap:ProductMember2021-01-012021-09-300001515673us-gaap:LicenseAgreementTermsMemberrare:ProfitShareRevenueAndRoyaltiesMemberrare:KyowaKirinCollaborationMember2020-12-310001515673us-gaap:ProductMember2020-07-012020-09-300001515673us-gaap:CommonStockMember2021-06-300001515673us-gaap:CommonStockMember2021-01-012021-09-300001515673rare:ArcturusResearchCollaborationAndLicenseAgreementMember2021-08-012021-08-310001515673rare:RoyaltyPurchaseAgreementMemberrare:RPIFinanceTrustMember2019-12-012019-12-310001515673srt:EuropeMember2020-01-012020-09-300001515673rare:LicenseAndCollaborationAgreementMemberrare:MereoBioPharma3LimitedMember2021-01-012021-01-310001515673rare:USGovernmentTreasuryAndAgencySecuritiesMember2021-09-300001515673us-gaap:AssetBackedSecuritiesMember2020-12-310001515673rare:DaiichiSankyoCoLtdMember2020-12-310001515673rare:ConvertiblePromissoryNoteMember2020-07-310001515673rare:PclAndHek293PlatformsMemberrare:LicenseAndTechnologyAccessAgreementMemberrare:DaiichiSankyoCoLtdMember2021-09-300001515673srt:EuropeMember2021-01-012021-09-300001515673rare:KyowaKirinCollaborationMember2020-07-012020-09-300001515673us-gaap:CorporateBondSecuritiesMember2020-12-310001515673rare:DaiichiSankyoCoLtdMember2020-01-012020-09-300001515673us-gaap:ProductMemberrare:MepseviiMember2021-07-012021-09-300001515673srt:LatinAmericaMember2020-07-012020-09-300001515673us-gaap:RoyaltyMemberrare:EuropeanUnionAndSwitzerlandAndTurkeyMemberrare:KyowaKirinCollaborationMember2020-01-012020-09-300001515673country:US2021-01-012021-09-300001515673rare:SolidBiosciencesIncMember2020-01-012020-12-310001515673us-gaap:CommonStockMember2020-01-012020-09-300001515673rare:CollaborationAndRoyaltyMemberrare:KyowaKirinCollaborationMember2021-01-012021-09-300001515673rare:CollaborationAndLicenseMemberrare:DaiichiSankyoCoLtdMember2020-01-012020-09-300001515673rare:EuropeanUnionAndSwitzerlandAndTurkeyMemberus-gaap:LicenseAgreementTermsMemberrare:KyowaKirinCollaborationMember2021-01-012021-09-300001515673us-gaap:ProductMemberrare:DojolviMember2020-01-012020-09-3000015156732020-06-300001515673us-gaap:ProductMemberrare:MepseviiMember2020-07-012020-09-300001515673us-gaap:CommonStockMemberrare:ArcturusResearchCollaborationAndLicenseAgreementMember2020-01-012020-12-310001515673rare:CertificateOfDepositsAndTimeDepositsMember2021-09-300001515673rare:LicenseAndTechnologyAccessAgreementMemberrare:DaiichiSankyoCoLtdMember2021-09-300001515673us-gaap:CommonStockMemberrare:ArcturusResearchCollaborationAndLicenseAgreementMember2021-09-300001515673us-gaap:SellingGeneralAndAdministrativeExpensesMember2021-07-012021-09-300001515673rare:TimeDepositsMember2020-12-310001515673us-gaap:RoyaltyMemberrare:CrysvitaMember2020-01-012020-09-300001515673rare:GeneTxBiotherapeuticsLLCMembersrt:MaximumMemberrare:ProgramAgreementAndUnitholderOptionAgreementMember2021-01-012021-09-300001515673rare:EuropeanUnionAndSwitzerlandAndTurkeyMemberrare:CollaborationAndRoyaltyMemberrare:KyowaKirinCollaborationMember2020-01-012020-09-300001515673us-gaap:PrepaidExpensesAndOtherCurrentAssetsMemberus-gaap:LicenseAgreementTermsMemberrare:KyowaKirinCollaborationMember2021-09-300001515673rare:ArcturusResearchCollaborationAndLicenseAgreementMember2020-07-012020-09-300001515673us-gaap:FairValueInputsLevel2Memberrare:OtherMember2021-09-300001515673us-gaap:LicenseAgreementTermsMemberrare:KyowaKirinCollaborationMember2021-01-012021-09-300001515673srt:MaximumMemberrare:ArcturusResearchCollaborationAndLicenseAgreementMember2015-10-012015-10-310001515673rare:LicenseAndCollaborationAgreementMemberrare:MereoBioPharma3LimitedMember2021-01-012021-03-310001515673us-gaap:CommonStockMember2020-07-012020-09-300001515673rare:CollaborationAndLicenseMemberrare:EuropeanUnionAndSwitzerlandAndTurkeyMemberrare:CrysvitaMember2021-01-012021-09-300001515673us-gaap:CommonStockMember2020-06-3000015156732021-01-012021-09-300001515673rare:TwoThousandFourteenEmployeeStockPurchasePlanMember2021-09-300001515673rare:ArcturusResearchCollaborationAndLicenseAgreementMember2020-12-012020-12-310001515673us-gaap:ResearchAndDevelopmentExpenseMember2020-01-012020-09-300001515673rare:CollaborationAndLicenseMember2021-07-012021-09-300001515673rare:CollaborationAndLicenseMemberrare:CrysvitaMember2021-01-012021-09-300001515673us-gaap:LicenseAgreementTermsMemberus-gaap:ProductMemberrare:KyowaKirinCollaborationMember2020-01-012020-09-300001515673us-gaap:AdditionalPaidInCapitalMember2020-12-310001515673rare:OptionsToPurchaseCommonStockAndRSUsMember2020-01-012020-09-300001515673us-gaap:CommonStockMember2020-09-300001515673rare:GeneTxBiotherapeuticsLLCMemberrare:ProgramAgreementAndUnitholderOptionAgreementMember2020-01-012020-03-310001515673srt:LatinAmericaMember2020-01-012020-09-300001515673us-gaap:CostOfSalesMember2020-01-012020-09-300001515673us-gaap:CommercialPaperNotIncludedWithCashAndCashEquivalentsMember2020-12-310001515673us-gaap:EmployeeStockMember2020-01-012020-09-300001515673rare:CollaborationAndLicenseMemberrare:CrysvitaMember2020-01-012020-09-300001515673us-gaap:AccumulatedOtherComprehensiveIncomeMember2019-12-310001515673us-gaap:LicenseAgreementTermsMemberrare:KyowaKirinCollaborationMember2020-01-012020-09-300001515673rare:UnitedStatesAndCanadaMemberrare:CollaborationAndRoyaltyMemberrare:KyowaKirinCollaborationMember2020-07-012020-09-300001515673us-gaap:MoneyMarketFundsMember2021-09-300001515673us-gaap:CommonStockMemberrare:ArcturusResearchCollaborationAndLicenseAgreementMember2020-01-012020-09-300001515673rare:ArcturusResearchCollaborationAndLicenseAgreementMember2020-01-012020-12-310001515673srt:MaximumMemberrare:ConvertiblePromissoryNoteMemberrare:DiaCartaLtdMember2020-07-012020-07-310001515673us-gaap:RoyaltyMemberrare:EuropeanUnionAndSwitzerlandAndTurkeyMemberrare:KyowaKirinCollaborationMember2021-01-012021-09-300001515673rare:InvestmentsInArcturusAndSolidCommonStockMemberus-gaap:FairValueInputsLevel1Member2021-09-300001515673us-gaap:FairValueInputsLevel1Memberrare:USGovernmentTreasuryAndAgencySecuritiesMember2020-12-310001515673us-gaap:AdditionalPaidInCapitalMember2021-09-300001515673srt:MaximumMemberrare:LicenseAndCollaborationAgreementMemberrare:MereoBioPharma3LimitedMember2021-01-310001515673us-gaap:RetainedEarningsMember2021-09-300001515673us-gaap:FairValueInputsLevel2Memberus-gaap:CorporateBondSecuritiesMember2020-12-310001515673us-gaap:CommonStockMemberrare:ArcturusResearchCollaborationAndLicenseAgreementMember2019-12-310001515673rare:EmploymentInducementPlanMember2021-09-300001515673us-gaap:RoyaltyMemberrare:CrysvitaMember2020-07-012020-09-300001515673rare:ArcturusResearchCollaborationAndLicenseAgreementMember2019-06-012019-06-300001515673rare:BayerHealthCareLLCMemberrare:ResearchDevelopmentAndCommercializeAAVGeneTherapyProductsAgreementMember2021-01-012021-09-300001515673us-gaap:AccumulatedOtherComprehensiveIncomeMember2021-07-012021-09-300001515673srt:MaximumMemberrare:SolidBiosciencesIncMemberrare:CollaborationAndLicenseAgreementMember2020-10-012020-10-310001515673rare:UnitedStatesAndCanadaMemberrare:CollaborationAndRoyaltyMemberrare:KyowaKirinCollaborationMember2020-01-012020-09-300001515673us-gaap:PrepaidExpensesAndOtherCurrentAssetsMemberrare:DaiichiSankyoCoLtdMember2021-09-300001515673srt:NorthAmericaMember2020-01-012020-09-300001515673rare:DebtSecuritiesInGovernmentSponsoredEntitiesMember2021-09-300001515673rare:MahziTherapeuticsIncMemberrare:ConvertiblePromissoryNoteMember2020-12-012020-12-310001515673rare:DaiichiSankyoCoLtdMember2020-07-012020-09-300001515673us-gaap:FairValueInputsLevel2Member2020-12-310001515673rare:CollaborationAndLicenseMember2021-01-012021-09-300001515673srt:MaximumMember2021-01-012021-09-300001515673srt:NorthAmericaMember2021-07-012021-09-300001515673us-gaap:ProductMemberrare:DojolviMember2021-01-012021-09-300001515673rare:KyowaKirinCollaborationMember2020-01-012020-09-300001515673rare:KyowaKirinCollaborationMember2021-01-012021-09-300001515673us-gaap:CostOfSalesMember2021-07-012021-09-300001515673rare:MahziTherapeuticsIncMemberrare:ConvertiblePromissoryNoteMember2020-12-310001515673rare:GeneTxBiotherapeuticsLLCMemberrare:ProgramAgreementAndUnitholderOptionAgreementMember2019-08-012019-08-31iso4217:USDxbrli:sharesrare:Segmentxbrli:purexbrli:sharesrare:DiseaseTargetutr:Qiso4217:USD

 

 

UNITED STATES

SECURITIES AND EXCHANGE COMMISSION

WASHINGTON, DC 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 September 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 No. 001-36276

 

ULTRAGENYX PHARMACEUTICAL INC.

(Exact name of registrant as specified in its charter)

 

 

Delaware

 

 

27-2546083

(State or other jurisdiction of incorporation or organization)

 

 

(I.R.S. Employer Identification No.)

 

60 Leveroni Court
Novato, California

 

94949

(Address of principal executive offices)

 

(Zip Code)

(415) 483-8800

(Registrant’s telephone number, including area code)

Not Applicable

(Former Name, Former Address and Former Fiscal Year, if Changed Since Last Report)

 

Securities registered pursuant to Section 12(b) of the Act:

Title of each class

Trading Symbol

Name of each exchange on which registered

Common Stock, $0.001 par value

RARE

The 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. YesNo

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). YesNo

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 filer

 

 

Accelerated filer

 

Non-accelerated filer

 

 

Smaller 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

As of October 28, 2021, the registrant had 68,162,225 shares of common stock issued and outstanding.

 

 


 

ULTRAGENYX PHARMACEUTICAL INC.

FORM 10-Q FOR THE QUARTER ENDED SEPTEMBER 30, 2021

INDEX

 

 

 

 

 

 

 

Page

 

 

 

 

 

CAUTIONARY NOTE REGARDING FORWARD-LOOKING STATEMENTS

 

1

 

 

 

 

 

Part I –

 

Financial Information

 

 

 

 

 

 

 

 

 

 

 

Item 1.

 

Financial Statements

 

 

 

 

 

 

 

 

 

 

 

 

 

Condensed Consolidated Balance Sheets

 

2

 

 

 

 

 

 

 

 

 

 

 

Condensed Consolidated Statements of Operations

 

3

 

 

 

 

 

 

 

 

 

 

 

Condensed Consolidated Statements of Comprehensive Loss

 

4

 

 

 

 

 

 

 

 

 

 

 

Condensed Consolidated Statements of Stockholders’ Equity

 

5

 

 

 

 

 

 

 

 

 

 

 

Condensed Consolidated Statements of Cash Flows

 

7

 

 

 

 

 

 

 

 

 

 

 

Notes to Condensed Consolidated Financial Statements

 

8

 

 

 

 

 

 

 

 

 

Item 2.

 

Management’s Discussion and Analysis of Financial Condition and Results of Operations

 

22

 

 

 

 

 

 

 

 

 

Item 3.

 

Quantitative and Qualitative Disclosures About Market Risk

 

34

 

 

 

 

 

 

 

 

 

Item 4.

 

Controls and Procedures

 

35

 

 

 

 

 

Part II –

 

Other Information

 

 

 

 

 

 

 

 

 

 

 

Item 1.

 

Legal Proceedings

 

35

 

 

 

 

 

 

 

 

 

Item 1A.

 

Risk Factors

 

35

 

 

 

 

 

 

 

 

 

Item 2.

 

Unregistered Sales of Equity Securities and Use of Proceeds

 

70

 

 

 

 

 

 

 

 

 

Item 3.

 

Defaults Upon Senior Securities

 

70

 

 

 

 

 

 

 

 

 

Item 4.

 

Mine Safety Disclosures

 

70

 

 

 

 

 

 

 

 

 

Item 5.

 

Other Information

 

70

 

 

 

 

 

 

 

 

 

Item 6.

 

Exhibits

 

71

 

 

 

 

 

 

 

 

 

Signatures

 

 

 

72

 

 

 

 


 

CAUTIONARY NOTE REGARDING FORWARD-LOOKING STATEMENTS

This Quarterly Report on Form 10-Q (the Quarterly Report) contains forward-looking statements that involve risks and uncertainties. We make such forward-looking statements pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995 and other federal securities laws. All statements other than statements of historical facts contained in this Quarterly Report are forward-looking statements. In some cases, you can identify forward-looking statements by words such as “anticipate,” “believe,” “contemplate,” “continue,” “could,” “estimate,” “expect,” “forecast,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “seek,” “should,” “target,” “will,” “would,” or the negative of these words, or other comparable terminology. These forward-looking statements include, but are not limited to, statements about:

our commercialization, marketing, and manufacturing capabilities and strategy;
our expectations regarding the timing of clinical study commencements and reporting results from same;
the timing and likelihood of regulatory approvals for our product candidates;
the anticipated indications for our product candidates, if approved;
the potential market opportunities for commercializing our products and product candidates;
our expectations regarding the potential market size and the size of the patient populations for our products and product candidates, if approved for commercial use;
the impact of the COVID-19 pandemic and related health measures on our business, financial condition and liquidity;
estimates of our expenses, revenue, capital requirements, and our needs for additional financing;
our ability to develop, acquire, and advance product candidates into, and successfully complete, clinical studies;
the implementation of our business model and strategic plans for our business, products and product candidates and the integration and performance of any businesses we have acquired or may acquire;
the initiation, timing, progress, and results of ongoing and future preclinical and clinical studies, and our research and development programs;
the scope of protection we are able to establish and maintain for intellectual property rights covering our products and product candidates;
our ability to maintain and establish collaborations or strategic relationships or obtain additional funding;
our ability to maintain and establish relationships with third parties, such as contract research organizations, contract manufacturing organizations, suppliers, and distributors;
our financial performance and the expansion of our organization;
our ability to obtain supply of our products and product candidates;
the scalability and commercial viability of our manufacturing methods and processes;
developments and projections relating to our competitors and our industry; and
other risks and uncertainties, including those listed under Part II, Item 1A. Risk Factors.

Any forward-looking statements in this Quarterly Report reflect our current views with respect to future events or to our 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 discussed under Part II, Item 1A. Risk Factors and elsewhere in this Quarterly Report. 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.

This Quarterly Report also contains estimates, projections, and other information concerning our industry, our business, and the markets for certain diseases, including data regarding the estimated size of those markets, and the incidence and prevalence of certain medical conditions. Information that is based on estimates, forecasts, projections, market research, or similar methodologies is inherently subject to uncertainties and actual events or circumstances may differ materially from events and circumstances reflected in this information. Unless otherwise expressly stated, we obtained this industry, business, market, and other data from reports, research surveys, studies, and similar data prepared by market research firms and other third parties, industry, medical and general publications, government data, and similar sources.

1


 

PART I. FINANCIAL INFORMATION

Item 1. Financial Statements

 

ULTRAGENYX PHARMACEUTICAL INC.

CONDENSED CONSOLIDATED BALANCE SHEETS

(Unaudited)

(In thousands, except share amounts)

 

 

September 30,

 

 

December 31,

 

 

2021

 

 

2020

 

Assets

 

 

 

 

 

Current assets:

 

 

 

 

 

Cash and cash equivalents

$

197,538

 

 

$

713,526

 

Marketable debt securities

 

539,579

 

 

 

488,007

 

Accounts receivable, net

 

25,383

 

 

 

23,093

 

Inventory

 

15,191

 

 

 

13,048

 

Prepaid expenses and other current assets

 

69,193

 

 

 

57,630

 

Total current assets

 

846,884

 

 

 

1,295,304

 

Property, plant, and equipment, net

 

119,069

 

 

 

73,515

 

Equity investments

 

86,215

 

 

 

155,375

 

Marketable debt securities

 

204,243

 

 

 

10,506

 

Right-of-use assets

 

36,696

 

 

 

40,524

 

Intangible assets, net

 

130,869

 

 

 

131,113

 

Goodwill

 

44,406

 

 

 

44,406

 

Other assets

 

16,419

 

 

 

8,812

 

Total assets

$

1,484,801

 

 

$

1,759,555

 

 

 

 

 

 

 

Liabilities and Stockholders’ Equity

 

 

 

 

 

Current liabilities:

 

 

 

 

 

Accounts payable

$

15,575

 

 

$

12,923

 

Accrued liabilities

 

109,660

 

 

 

108,491

 

Contract liabilities

 

 

 

 

59,219

 

Lease liabilities

 

10,698

 

 

 

8,976

 

Total current liabilities

 

135,933

 

 

 

189,609

 

Lease liabilities

 

33,344

 

 

 

39,251

 

Deferred tax liabilities

 

33,306

 

 

 

33,306

 

Liability related to the sale of future royalties

 

348,073

 

 

 

335,665

 

Other liabilities

 

471

 

 

 

7,349

 

Total liabilities

 

551,127

 

 

 

605,180

 

Stockholders’ equity:

 

 

 

 

 

Preferred stock — 25,000,000 shares authorized; nil outstanding as of September 30, 2021 and
   December 31, 2020

 

 

 

 

 

Common stock — 250,000,000 shares authorized; 67,961,963 and 66,818,520 shares issued
   and outstanding as of September 30, 2021 and December 31, 2020, respectively

 

68

 

 

 

67

 

Additional paid-in capital

 

2,884,695

 

 

 

2,773,195

 

Accumulated other comprehensive income

 

54

 

 

 

689

 

Accumulated deficit

 

(1,951,143

)

 

 

(1,619,576

)

Total stockholders’ equity

 

933,674

 

 

 

1,154,375

 

Total liabilities and stockholders’ equity

$

1,484,801

 

 

$

1,759,555

 

See accompanying notes.

2


 

ULTRAGENYX PHARMACEUTICAL INC.

CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS

(Unaudited)

(In thousands, except share and per share amounts)

 

 

Three Months Ended September 30,

 

 

Nine Months Ended September 30,

 

 

2021

 

 

2020

 

 

2021

 

 

2020

 

Revenues:

 

 

 

 

 

 

 

 

 

 

 

Collaboration and license

$

55,048

 

 

$

66,924

 

 

$

197,998

 

 

$

144,300

 

Product sales

 

21,950

 

 

 

11,215

 

 

 

56,809

 

 

 

25,760

 

Non-cash collaboration royalty revenue

 

4,649

 

 

 

3,331

 

 

 

13,210

 

 

 

9,428

 

Total revenues

 

81,647

 

 

 

81,470

 

 

 

268,017

 

 

 

179,488

 

Operating expenses:

 

 

 

 

 

 

 

 

 

 

 

Cost of sales

 

4,175

 

 

 

2,348

 

 

 

12,499

 

 

 

648

 

Research and development

 

113,417

 

 

 

87,314

 

 

 

374,140

 

 

 

280,984

 

Selling, general and administrative

 

53,883

 

 

 

42,123

 

 

 

160,551

 

 

 

131,891

 

Total operating expenses

 

171,475

 

 

 

131,785

 

 

 

547,190

 

 

 

413,523

 

Loss from operations

 

(89,828

)

 

 

(50,315

)

 

 

(279,173

)

 

 

(234,035

)

Interest income

 

408

 

 

 

1,338

 

 

 

1,488

 

 

 

6,054

 

Change in fair value of equity investments

 

25,702

 

 

 

(11,520

)

 

 

(25,963

)

 

 

91,348

 

Non-cash interest expense on liability related to the sale of future
    royalties

 

(8,683

)

 

 

(8,582

)

 

 

(25,618

)

 

 

(25,093

)

Other income (expense)

 

(415

)

 

 

547

 

 

 

(1,277

)

 

 

308

 

Loss before income taxes

 

(72,816

)

 

 

(68,532

)

 

 

(330,543

)

 

 

(161,418

)

Provision for income taxes

 

(182

)

 

 

(313

)

 

 

(1,024

)

 

 

(1,137

)

Net loss

$

(72,998

)

 

$

(68,845

)

 

$

(331,567

)

 

$

(162,555

)

Net loss per share, basic and diluted

$

(1.08

)

 

$

(1.13

)

 

$

(4.91

)

 

$

(2.73

)

Weighted-average shares used in computing net loss per share,
    basic and diluted

 

67,875,363

 

 

 

60,687,177

 

 

 

67,533,671

 

 

 

59,564,163

 

See accompanying notes.

3


 

ULTRAGENYX PHARMACEUTICAL INC.

CONDENSED CONSOLIDATED STATEMENTS OF COMPREHENSIVE LOSS

(Unaudited)

(In thousands)

 

 

Three Months Ended September 30,

 

 

Nine Months Ended September 30,

 

 

2021

 

 

2020

 

 

2021

 

 

2020

 

Net loss

$

(72,998

)

 

$

(68,845

)

 

$

(331,567

)

 

$

(162,555

)

Other comprehensive income (loss):

 

 

 

 

 

 

 

 

 

 

 

Foreign currency translation adjustments

 

(166

)

 

 

374

 

 

 

(348

)

 

 

414

 

Unrealized gain (loss) on available-for-sale securities

 

(12

)

 

 

(645

)

 

 

(287

)

 

 

746

 

Other comprehensive income (loss):

 

(178

)

 

 

(271

)

 

 

(635

)

 

 

1,160

 

Total comprehensive loss

$

(73,176

)

 

$

(69,116

)

 

$

(332,202

)

 

$

(161,395

)

See accompanying notes.

 

4


 

ULTRAGENYX PHARMACEUTICAL INC.

CONDENSED CONSOLIDATED STATEMENTS OF STOCKHOLDERS’ EQUITY

(Unaudited)

(In thousands, except share amounts)

 

 

 

Common Stock

 

 

Additional
Paid-In

 

 

Accumulated
Other
Comprehensive

 

 

Accumulated

 

 

Total
Stockholders'

 

 

 

Shares

 

 

Amount

 

 

 Capital

 

 

Income (Loss)

 

 

Deficit

 

 

Equity

 

Balance as of June 30, 2021

 

 

67,764,950

 

 

$

68

 

 

$

2,850,016

 

 

$

232

 

 

$

(1,878,145

)

 

$

972,171

 

Stock-based compensation

 

 

 

 

 

 

 

 

27,005

 

 

 

 

 

 

 

 

 

27,005

 

Issuance of common stock under
   equity plan awards, net of tax

 

 

197,013

 

 

 

 

 

 

7,674

 

 

 

 

 

 

 

 

 

7,674

 

Other comprehensive loss

 

 

 

 

 

 

 

 

 

 

 

(178

)

 

 

 

 

 

(178

)

Net loss

 

 

 

 

 

 

 

 

 

 

 

 

 

 

(72,998

)

 

 

(72,998

)

Balance as of September 30, 2021

 

 

67,961,963

 

 

$

68

 

 

$

2,884,695

 

 

$

54

 

 

$

(1,951,143

)

 

$

933,674

 

 

 

 

Common Stock

 

 

Additional
Paid-In

 

 

Accumulated
Other
Comprehensive

 

 

Accumulated

 

 

Total
Stockholders'

 

 

 

Shares

 

 

Amount

 

 

 Capital

 

 

Income (Loss)

 

 

Deficit

 

 

Equity

 

Balance as of December 31, 2020

 

 

66,818,520

 

 

$

67

 

 

$

2,773,195

 

 

$

689

 

 

$

(1,619,576

)

 

$

1,154,375

 

Stock-based compensation

 

 

 

 

 

 

 

 

78,324

 

 

 

 

 

 

 

 

 

78,324

 

Issuance of common stock under
   equity plan awards, net of tax

 

 

1,143,443

 

 

 

1

 

 

 

33,176

 

 

 

 

 

 

 

 

 

33,177

 

Other comprehensive loss

 

 

 

 

 

 

 

 

 

 

 

(635

)

 

 

 

 

 

(635

)

Net loss

 

 

 

 

 

 

 

 

 

 

 

 

 

 

(331,567

)

 

 

(331,567

)

Balance as of September 30, 2021

 

 

67,961,963

 

 

$

68

 

 

$

2,884,695

 

 

$

54

 

 

$

(1,951,143

)

 

$

933,674

 

 

 

 

Common Stock

 

 

Additional
Paid-In

 

 

Accumulated
Other
Comprehensive

 

 

Accumulated

 

 

Total
Stockholders'

 

 

 

Shares

 

 

Amount

 

 

 Capital

 

 

Income (Loss)

 

 

Deficit

 

 

Equity

 

Balance as of June 30, 2020

 

 

60,413,756

 

 

$

60

 

 

$

2,220,963

 

 

$

1,284

 

 

$

(1,526,720

)

 

$

695,587

 

Stock-based compensation

 

 

 

 

 

 

 

 

20,527

 

 

 

 

 

 

 

 

 

20,527

 

Issuance of common stock under
   equity plan awards, net of tax

 

 

401,934

 

 

 

1

 

 

 

19,192

 

 

 

 

 

 

 

 

 

19,193

 

Other comprehensive loss

 

 

 

 

 

 

 

 

 

 

 

(271

)

 

 

 

 

 

(271

)

Net loss

 

 

 

 

 

 

 

 

 

 

 

 

 

 

(68,845

)

 

 

(68,845

)

Balance as of September 30, 2020

 

 

60,815,690

 

 

$

61

 

 

$

2,260,682

 

 

$

1,013

 

 

$

(1,595,565

)

 

$

666,191

 

See accompanying notes.

5


 

ULTRAGENYX PHARMACEUTICAL INC.

CONDENSED CONSOLIDATED STATEMENTS OF STOCKHOLDERS’ EQUITY

(Unaudited)

(In thousands, except share amounts)

 

 

 

Common Stock

 

 

Additional
Paid-In

 

 

Accumulated
Other
Comprehensive

 

 

Accumulated

 

 

Total
Stockholders'

 

 

 

Shares

 

 

Amount

 

 

 Capital

 

 

Income (Loss)

 

 

Deficit

 

 

Equity

 

Balance as of December 31, 2019

 

 

57,838,220

 

 

$

58

 

 

$

2,086,863

 

 

$

(147

)

 

$

(1,433,010

)

 

$

653,764

 

Issuance of common stock in
   connection with license
   agreement, net of issuance costs

 

 

1,243,913

 

 

 

1

 

 

 

55,267

 

 

 

 

 

 

 

 

 

55,268

 

 Issuance of common stock in
   connection with at-the-market
   offering, net of issuance costs

 

 

283,333

 

 

 

 

 

 

20,391

 

 

 

 

 

 

 

 

 

20,391

 

Stock-based compensation

 

 

 

 

 

 

 

 

63,112

 

 

 

 

 

 

 

 

 

63,112

 

Issuance of common stock upon
    exercise of warrants and under
    equity plan awards, net of tax

 

 

1,450,224

 

 

 

2

 

 

 

35,049

 

 

 

 

 

 

 

 

 

35,051

 

Other comprehensive income