艾意凯-生物制药行业展望2024

领袖视角1领袖视角生物制药行业展望2024领袖视角2L.E.K.咨询7920192020202120222023**173020192020202120222023** 5420192020202120222023**7920192020202120222023**173020192020202120222023** 5420192020202120222023**XBI指数价格*250200148 100 1001188710087500IPO融资^ 100218 185 185150100500风险投资250203200150155150风险投资25020320015015515010050020192020202120222023**总并购交易价值250200150100100566030505660300*2023年之前的XBI指数价格截至年底**2023年风险投资价值于2023年12月15日更新（来自Pitchbook其他指标则于2023年11月30日更新（来自CapitalIQ因此可能会低估最终年度融资价值^2023年不包括KenvueIPO，筹资总额约为38亿美元注释：IPO=首次公开募股；VC=风险投资；XBI=SPDR标普生物科技；美国IPO和并购交易包括CapitalIQ中从行业分类中选择的已宣布和已完成的交易，这些行业分类包括制药、生物技术和生命科学、生物技术、制药、医药产品、药品研究与开发；PitchBook数据未经PitchBook分析师审核资料来源：PitchBook（风险投资CapitalIQ（XBI指数价格、IPO融资、并购总交易价值）衰退做好准备2。领袖视角生物制药行业展望2024领袖视角3L.E.K.咨询多行业玩家对并购交易非常感兴趣，希望借此抵消未来因失去市场独占权和《通货膨胀削减法案》10,11领袖视角生物制药行业展望2024领袖视角4L.E.K.咨询ModernizingandEnsuringPBMAccountabilityAct）13。.除了美国，生物制药企业还需尽早针对欧洲市场动态进行规划14。欧洲卫生技术评估网络如InSilicoMedicine的INS018_055在特发性肺纤维化的二期临床数据15、RelayTherapeutics的领袖视角生物制药行业展望2024领袖视角5L.E.K.咨询变革性筛选验证增量式临床试验设计全渠道互动人力资源分析具有内部数据访问功能的类似ChatGPT的平台领袖视角生物制药行业展望2024领袖视角6L.E.K.咨询领袖视角生物制药行业展望2024领袖视角7L.E.K.咨询28Biopharma、OrnaTherapeutics等公司正在探索的新型体内CAR-T细胞制备方法。30A型血友病（辉瑞和SangamoTherapeutics的基因疗法Giroctocogenefitelparvovec预计将于2024年中期发布三期临床数据）33和B型血友病（辉瑞和Spark的Fidanacogeneelaparvovec，领袖视角生物制药行业展望2024领袖视角8L.E.K.咨询瘤治疗领域，Moderna和默克正在个体化mRNA癌症疫苗V940与Keytruda联合治疗高危黑色素是否存在first-in-class和first-in-indication的研发机会。最后，放射性药物和双特异性抗体这9L.E.K.咨询公司类别适应症资产里程碑CLLorSLL,R/R(3L+)PDUFA：3月14日PDUFA：3月14日百时美施贵宝强生CD19CART,auto.BreyanziMultipleMyeloma,R/R,(2L+)CarvyktiBCMACART,auto.AdV;BKV;CMV;EBV;HHV-6;JCV顶线数据：第三至四季度AllovirMVSTcell,allo.PosoleucelClassicalHodgkinLymphomaAB-101withAFM13NKcellwithinnatecellengager顶线数据：第三至四季度ArtivaOvariancancer,Platinum-resistantADP-A2M4CD8AdaptimmuneTCR-T公司适应症里程碑资产类别MetachromaticleukodystrophyLVV-transducedCD34+enrichedstemcellsLibmeldy(atidarsageneautotemcel)OrchardTherapeuticsPDUFA：3月18日RocketPharmaceuticalsLVV-transducedstemcellsMarnetegrageneautotemcelPDUFA：3月31日LAD-I公司类别适应症资产里程碑CLLorSLL,R/R(3L+)PDUFA：3月14日PDUFA：3月14日百时美施贵宝强生CD19CART,auto.BreyanziMultipleMyeloma,R/R,(2L+)CarvyktiBCMACART,auto.AdV;BKV;CMV;EBV;HHV-6;JCV顶线数据：第三至四季度AllovirMVSTcell,allo.PosoleucelClassicalHodgkinLymphomaAB-101withAFM13NKcellwithinnatecellengager顶线数据：第三至四季度ArtivaOvariancancer,Platinum-resistantADP-A2M4CD8AdaptimmuneTCR-T公司适应症里程碑资产类别MetachromaticleukodystrophyLVV-transducedCD34+enrichedstemcellsLibmeldy(atidarsageneautotemcel)OrchardTherapeuticsPDUFA：3月18日RocketPharmaceuticalsLVV-transducedstemcellsMarnetegrageneautotemcelPDUFA：3月31日LAD-ITransfusion-DependentBetaThalassemiaVertex/CRISPRTherapeuticsCRISPR-CAS9autologousCD34+PDUFA：3月30日Exa-celBioengineeredAAVvector(AAV-Spark100)辉瑞/SparkTherapeuticsFidanacogeneelaparvovecPDUFA：第二季度HemophiliaBHemophiliaAGiroctocogenefitelparvovec辉瑞顶线数据：第二至三季度AAV6vectorOligonucleotide(Telomeraseinhibitor)PDUFA：6月16日ImetelstatGeronCorporationIonisPharmaceuticals顶线数据：第一至二季度LICA(PKKinhibitor)Donidalorsen领袖视角生物制药行业展望2024细胞疗法临床一期临床二期临床三期基因疗法临床一期临床二期临床三期公司资产类别适应症临床一期临床二期里程碑临床三期公司资产类别适应症临床一期临床二期里程碑LowerLowerriskmyelodysplasticsyndromeADC(Humanizedanti-HER2IgG1Breastcancer,HER2+(3L+);Breastcancer,HER2+(3L+);Others*PDUFA：第三至四季度阿斯利康/第一三共Enhertutopoisomeraseinhibitor+tetrapeptidelinker)HAEHAEFamilialARO-APOC3ArrowheadPharmaceuticals顶线数据：第一至二季度chylomicronemiaARO-APOC3ArrowheadPharmaceuticals顶线数据：第一至二季度chylomicronemiasyndromeInhibitor)领袖视角生物制药行业展望2024领袖视角10L.E.K.咨询1Asof11/30/2023comparedwithdateofpeakXBIprice(2/8/2021).2L,“CashPreservationintheBiopharmaceuticalIndustry:NavigatingUncertainty.”/insights/hea/us/ei/cash-preservation-biopharmaceutical-industry-navigating-uncertainty3Jefferies,“OctoberBiotechFunding.”4BiopharmaDive,“Lessonsfromabiotechdownturn:Fundingchallenges,anIPOdryspellandwhattoexpectin2024.”https://www./news/biotech-startup-financing-ipo-downturn-lessons/697855/5NewYorkTimes,“KKRBuysMinorityStakeinLifeSciencesInvestorCatalioCapital.”/articles/kkr-buys-minority-stake-in-life-sciences-investor-catalio-capital-e4ee84e6BiopharmaDive,“Afterarecordrun,fewerbiotechsaregoingpublic.Here’showthey’reperforming.”/news/biotech-ipo-performance-tracker/587604/7Biospace,“5LifeSciencesIPOsin2023—andtheFutureForecast.”/article/5-life-sciences-ipo-in-2023-and-the-future-forecast-/8Stifel.9NYTimes,“BidenMakesLowerDrugPricesaCenterpieceofHis2024Campaign.”/2023/08/30/us/politics/biden-medicare-drug-prices-2024-campaign.html?unlocked_article_code=1.7Ew.Ap0i.jTXwQDDVtSrO&smid=url-share10PositionsofotherRepublicanpresidentialcandidatesonrepealingdrugpricingprovisionsoftheInflationReductionActarenotyetfullyclear.11AssociatedPress,“Trumpmakeslate-termbidtolowerprescriptiondrugcosts.”/article/donald-trump-medication-prescription-drug-costs-medicare-prescription-drugs-e171198402445755b920842ded293b5912C,“HR5378.”/bill/118th-congress/house-bill/5378/text#toc-H3EB5C78AAFDE4912BE147826E4A93E9F13C,“ModernizingandEnsuringPBMAccountabilityAct.”/imo/media/doc/092823_mepa_legislative_text.pdf14L,“EUnetHTA21—TransitiontoEU-wideHTAs:ImplicationsforPharma.”/insights/hea/eu/ei/eunethta-21-transition-eu-wide-htas-implications-pharma#:~:text=The%20EU%20HTA%20regulation%20formulated,key%20for%20a%20successful%20JCA.15CNBC,“ThefirstfullyA.I.-generateddrugentersclinicaltrialsinhumanpatients.”/2023/06/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