北海康成-B2023年中期报告

北 海 康 成 製 藥 有 限 公 司CANbridge Pharmaceuticals Inc. （於開曼群島註冊成立的有限公司）股份代號 :1228 25791028464748505153 2023630 0.00001 TFDA 89 Nexus WayCamana BayGrand CaymanKY1-9009Cayman Islands Derek Paul Di Rocco Ogier Global (Cayman) Limited89 Nexus WayCamana BayGrand CaymanKY1-9009Cayman Islands Richard James GregoryJames Arthur Geraghty 183171712-1716 38821618 15516A131 3A10 James Arthur Geraghty 1228 97927 Richard James Gregory Derek Paul Di RoccoJames Arthur GeraghtyRichard James Gregory www.canbridgepharma.com ®(CAN101)IIMPS IIERTII20185 •20215IIERT2023630739 •109586 ®CAN108(IBAT) •20235291(ALGS)•202412023(NRDL)•(biliary atresia, BA)2Mirum Pharmaceuticals, Inc.MirumMirum2023•20231•2023(PFIC) C A N106PNH)(MG)C520185PNH •CAN106PNH1bCAN106C5(LDH) •1b2023PNH2024 CAN008(GBM)CD95-Fc •CAN008GBM2 CAN103(GD)ERT GD 2018 5 •1/2A2023B•CAN103GDERT (AAV)(SMA)2018 5 •Jason WestWest •2023(ASGCT)ASGCTscAAVSMAhSMN1co-hSMN1scAAV-CMVen/CB-hSMN1SMAFDASMA •202263034.78.424.2%202363043.1®® •2022630158.315.39.7%2023630143.0 •2022630249.030.812.4%2023630218.2 •2022630228.924.510.7%2023630204.4218.22022630249.0 2012202363014(IND) 20236301181780%(CHARD) 2012 II(LSDs)AALGSPFIC BA 20209II®(CAN101)20217PNHCAN106IND20222CAN106 I20236PNHCAN106 1bLDH20221®ALGS2023520227BAII2023520227CAN103 I20231II CAN008GBM2018CAN008IINDCAN008II202110GBMCAN008 II20233II 2023630LogicBio TherapeuticsAAV sL6520231ChanSMAAAV(CNS) 198320161,09020201,3515.5%2020203011.0%20303,833 202013203025934.5% 2018121 80%(Novartis)SMAZolgensma® 202259127 202112102023630 ®(CAN101) •®IIERTERT®(CAN101) •20209®(CAN101)MPS II®(CAN101)GC Pharma10IElaprase®®(CAN101)•20215®(CAN101)2023630739109586• CAN108® •IBATALGSFDAPFICBA1,700ALGSPFIC200MirumALGSIIb118MirumPFICIII93PFIC1PFIC2 PFIC3 PFIC4 PFIC6PFICIIIzMirumsNDAPFICMirum20219ALGSFDA202212 •MirumALGSPFIC BA •20235291ALGSALGS202412023 •ALGSPFICBAFDAMirum20235BAIIEMBARK1BAEMBARK2023 1ALGSNDA2023 •Mirum202361.6202275.1 CAN106 •CAN106C5PNHC5CAN106PK/PDCAN106PNHC5 •20192020WuXi Biologics Ireland LimitedPrivus Biologics, LLCPrivusPNHC5CAN106 •20236CAN106PNH1bCAN10624C53C50.5CAN106CAN106PNHPNHI2023PNH2024 •2022202261722141a202221a •CAN106FDAMGCAN10650%MG7 CAN008 (asunercept) •CAN008CD95-FcGBMCAN008CD95LCD95CAN008CD95LCD95T •CAN008GBMGBMI200400CAN0084001257%(4/7)IIApogenixII50%46GBM •20236CAN008GBMIIII11921CAN008 400GBM(PFS)(OS)2024II •CAN008GBMFDA(EMA)EMACAN008CAN008GBMCAN008 II •CAN0083 CAN103 •CAN103βGDERT •GD IIIICAN103 I/IIGD2269.HKCAN103ERTIIIIGD •I/IIA2023B •CAN103ERT •20235Jason WestWestFractyl Health Inc.AAVWestCRISPR Therapeutics AGCRISPRDNACRISPRWest •UMass ChanHorae2023ASGCTSMASMAASGCThSMN1co-hSMN1scAAVSMAscAAV-CMVen/CB-hSMN1SMAFDASMA •UMass ChanscAAVhSMN1co-hSMN1SMA •2022LogicBio®TherapeuticsAlexion, AstraZeneca Rare Disease • GC PharmaMirumCDMO (KOL) 202263034.78.4202363043.1®® 202263012.63.8202363016.4 202263022.24.5202363026.7202363062.0%202263063.8% 20226306.42.120236308.52023630 202263042.64.3202363038.32023630 202263055.67.4202363048.2 2022630158.315.32023630143.0 20226302.520236304.5 2023630283.649.9220.75.37.720221231463.1 2023630 202363034.72022123137.614.420.34.0%4.2%26.87.9 2023630103.6%20221231181.6%2023630 20236306.1%202212315.4% 2023630 202363020236303.0202263027.02022630 2023630CANbridge Biomed Limited202363012.4 2023630 2023630 202261020222021202278 20211210604.0 •45.4%CAN008CMC •24.0% 4.3%Hunterase® (CAN101) •12.6%(PNH)C5CAN106 •3.6%CAN103 •3.5%CAN108 •1.8% •12.0%CAN201CAN202 •16.8% •7.2%CAN008CMCCAN008•1.3%•3.0%•1.0%•4.3% 20236302022630 2023630XV(a)XV78(b)352(c) *2023630424,392,920(1)CTX Pharma Holdings Limited26,042,380(2)15,000,000JQX 2021 Gift Trust(3)733,0509,481,420202211111,000,0001,000,000(4)James Arthur Geraghty700,000James ArthurGeraghty1,250,000(5)Richard James Gregory300,000(6)250,000 2023630XVXV78352 2023630XV233365% *2023630424,392,920(1)CTX Pharma Holdings Limited26,042,380CTX PharmaHoldings Limited(2)201232620,554,860WuXi PharmaTech Healthcare Fund I L.P.19,792,100WuXi PharmaTech Healthcare Fund I L.P.WuXi PharmaTech Healthcare FundI L.P.WuXi PharmaTechHealthcare Fund I L.P.(3)RA Capital Management, L.P.RA Capital Healthcare Fund, L.P.47,185,140RA Capital Nexus Fund, L.P.9,137,400Blackwell Partners LLC-Series A3,913,050RA Capital Healthcare Fund, LPRA CapitalHealthcare Fund GP, LLCRA Capital Nexus Fund, LPRA Capital Nexus Fund GP, LLCRA CapitalHealthcare Fund, L.P.RA Capital Nexus Fund, L.P.RA Capital Management, L.P.RA CapitalManagement, L.P.RA Capital Healthcare Fund, L.P.RA Capital Nexus Fund, L.P.Blackwell Partners LLCPeter KolchinskyRA Capital Management, L.P.Anna Inge Leonore KolchinskyPeter Kolchinsky(4)Qiming Venture Partners IV, L.P.Qiming Managing Directors Fund IV, L.P.Qiming Venture PartnersQiming GP IV, L.P.Qiming Venture Partners IV, L.P.Qiming Corporate GP IV, Ltd.Qiming GP IV, L.P.Qiming GP IV, L.P.Qiming Corporate GP IV, Ltd.Qiming Venture Partners IV, L.P.Qiming Managi