艾意凱-生物制藥行業(yè)展望2024

領(lǐng)袖視角1領(lǐng)袖視角生物制藥行業(yè)展望2024領(lǐng)袖視角2L.E.K.咨詢7920192020202120222023**173020192020202120222023** 5420192020202120222023**7920192020202120222023**173020192020202120222023** 5420192020202120222023**XBI指數(shù)價格*250200148 100 1001188710087500IPO融資^ 100218 185 185150100500風險投資250203200150155150風險投資25020320015015515010050020192020202120222023**總并購交易價值250200150100100566030505660300*2023年之前的XBI指數(shù)價格截至年底**2023年風險投資價值于2023年12月15日更新（來自Pitchbook其他指標則于2023年11月30日更新（來自CapitalIQ因此可能會低估最終年度融資價值^2023年不包括KenvueIPO，籌資總額約為38億美元注釋：IPO=首次公開募股；VC=風險投資；XBI=SPDR標普生物科技；美國IPO和并購交易包括CapitalIQ中從行業(yè)分類中選擇的已宣布和已完成的交易，這些行業(yè)分類包括制藥、生物技術(shù)和生命科學、生物技術(shù)、制藥、醫(yī)藥產(chǎn)品、藥品研究與開發(fā)；PitchBook數(shù)據(jù)未經(jīng)PitchBook分析師審核資料來源：PitchBook（風險投資CapitalIQ（XBI指數(shù)價格、IPO融資、并購總交易價值）衰退做好準備2。領(lǐng)袖視角生物制藥行業(yè)展望2024領(lǐng)袖視角3L.E.K.咨詢多行業(yè)玩家對并購交易非常感興趣，希望借此抵消未來因失去市場獨占權(quán)和《通貨膨脹削減法案》10,11領(lǐng)袖視角生物制藥行業(yè)展望2024領(lǐng)袖視角4L.E.K.咨詢ModernizingandEnsuringPBMAccountabilityAct）13。.除了美國，生物制藥企業(yè)還需盡早針對歐洲市場動態(tài)進行規(guī)劃14。歐洲衛(wèi)生技術(shù)評估網(wǎng)絡(luò)如InSilicoMedicine的INS018_055在特發(fā)性肺纖維化的二期臨床數(shù)據(jù)15、RelayTherapeutics的領(lǐng)袖視角生物制藥行業(yè)展望2024領(lǐng)袖視角5L.E.K.咨詢變革性篩選驗證增量式臨床試驗設(shè)計全渠道互動人力資源分析具有內(nèi)部數(shù)據(jù)訪問功能的類似ChatGPT的平臺領(lǐng)袖視角生物制藥行業(yè)展望2024領(lǐng)袖視角6L.E.K.咨詢領(lǐng)袖視角生物制藥行業(yè)展望2024領(lǐng)袖視角7L.E.K.咨詢28Biopharma、OrnaTherapeutics等公司正在探索的新型體內(nèi)CAR-T細胞制備方法。30A型血友病（輝瑞和SangamoTherapeutics的基因療法Giroctocogenefitelparvovec預計將于2024年中期發(fā)布三期臨床數(shù)據(jù)）33和B型血友?。ㄝx瑞和Spark的Fidanacogeneelaparvovec，領(lǐng)袖視角生物制藥行業(yè)展望2024領(lǐng)袖視角8L.E.K.咨詢瘤治療領(lǐng)域，Moderna和默克正在個體化mRNA癌癥疫苗V940與Keytruda聯(lián)合治療高危黑色素是否存在first-in-class和first-in-indication的研發(fā)機會。最后，放射性藥物和雙特異性抗體這9L.E.K.咨詢公司類別適應(yīng)癥資產(chǎn)里程碑CLLorSLL,R/R(3L+)PDUFA：3月14日PDUFA：3月14日百時美施貴寶強生CD19CART,auto.BreyanziMultipleMyeloma,R/R,(2L+)CarvyktiBCMACART,auto.AdV;BKV;CMV;EBV;HHV-6;JCV頂線數(shù)據(jù)：第三至四季度AllovirMVSTcell,allo.PosoleucelClassicalHodgkinLymphomaAB-101withAFM13NKcellwithinnatecellengager頂線數(shù)據(jù)：第三至四季度ArtivaOvariancancer,Platinum-resistantADP-A2M4CD8AdaptimmuneTCR-T公司適應(yīng)癥里程碑資產(chǎn)類別MetachromaticleukodystrophyLVV-transducedCD34+enrichedstemcellsLibmeldy(atidarsageneautotemcel)OrchardTherapeuticsPDUFA：3月18日RocketPharmaceuticalsLVV-transducedstemcellsMarnetegrageneautotemcelPDUFA：3月31日LAD-I公司類別適應(yīng)癥資產(chǎn)里程碑CLLorSLL,R/R(3L+)PDUFA：3月14日PDUFA：3月14日百時美施貴寶強生CD19CART,auto.BreyanziMultipleMyeloma,R/R,(2L+)CarvyktiBCMACART,auto.AdV;BKV;CMV;EBV;HHV-6;JCV頂線數(shù)據(jù)：第三至四季度AllovirMVSTcell,allo.PosoleucelClassicalHodgkinLymphomaAB-101withAFM13NKcellwithinnatecellengager頂線數(shù)據(jù)：第三至四季度ArtivaOvariancancer,Platinum-resistantADP-A2M4CD8AdaptimmuneTCR-T公司適應(yīng)癥里程碑資產(chǎn)類別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)輝瑞/SparkTherapeuticsFidanacogeneelaparvovecPDUFA：第二季度HemophiliaBHemophiliaAGiroctocogene?telparvovec輝瑞頂線數(shù)據(jù)：第二至三季度AAV6vectorOligonucleotide(Telomeraseinhibitor)PDUFA：6月16日ImetelstatGeronCorporationIonisPharmaceuticals頂線數(shù)據(jù)：第一至二季度LICA(PKKinhibitor)Donidalorsen領(lǐng)袖視角生物制藥行業(yè)展望2024細胞療法臨床一期臨床二期臨床三期基因療法臨床一期臨床二期臨床三期公司資產(chǎn)類別適應(yīng)癥臨床一期臨床二期里程碑臨床三期公司資產(chǎn)類別適應(yīng)癥臨床一期臨床二期里程碑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頂線數(shù)據(jù)：第一至二季度chylomicronemiaARO-APOC3ArrowheadPharmaceuticals頂線數(shù)據(jù)：第一至二季度chylomicronemiasyndromeInhibitor)領(lǐng)袖視角生物制藥行業(yè)展望2024領(lǐng)袖視角10L.E.K.咨詢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.”/2023/06/29/ai-generated-drug-begins-clinical-trials-in-human-patients.html16RelayTherapeutics,“CorporatePresentation.”/static-files/d5fd1def-064f-4347-83a5-032d7727dc4417BioITWorld,“BigPharma,ModernaonAnAI-EmpoweredEraofDrugDiscovery.”/news/2023/11/08/big-parma-moderna-on-an-ai-empowered-era-of-drug-discovery18BioITWorld.“BigPharma,ModernaonAnAI-EmpoweredEraofDrugDiscovery.”/news/2023/11/08/big-pharma-moderna-on-an-ai-empowered-era-of-drug-discovery19Veeva,“VeevaPulseFieldTrendsReport.”/resources/veeva-pulse-field-trends-report/領(lǐng)袖視角生物制藥行業(yè)展望2024領(lǐng)袖視角20Tealium,“HowtoCreateReal-Time,PersonalizedExperiencesinPharma.”/blog/customer-experience/how-to-create-real-time-personalized-experiences-in-pharma/21Barclays,“3Q23:Afami-celBLASubmissiononTrackfor4Q23;Lete-celProgramUpdateinEarly2024.”22Pressrelease,“AffimedAnnouncesINDClearanceforaPhase2ClinicalTrialInvestigatingAFM13andAB-101CombinationTherapy.”https:///en/news-release/2023/05/23/2674077/0/en/Affimed-Announces-IND-Clearance-for-a-Phase-2-Clinical-Trial-Investigating-AFM13-and-AB-101-Combination-Therapy.html23ClinicalT,“APhase2StudyofCRG-022inPatientsWithRelapsed/RefractoryLargeB-cellLymphoma.”https://classic.clinicaltrials.gov/ct2/show/NCT0597272024Pressrelease,“AlloVirAnnouncesPlanstoCompleteEnrollmentinThreePhase3PosoleucelStudiesin2023.”/news-releases/news-release-details/allovir-announces-plans-complete-enrollment-three-phase-325PharmaceuticalTechnology,“Imugenelicensespotentialfirst-to-marketallogeneicCARTforbloodcancers.”https://www.pharmaceutical-/analyst-comment/imugene-allogeneic-car-t-blood-cancer/26Pressrelease,“AllogeneTherapeuticsReportsFourthQuarterandFullYear2022FinancialResultsandProvidesBusinessUpdate.”/news-releases/news-release-details/allogene-therapeutics-reports-fourth-quarter-and-full-year-202227WilliamBlair,“CELLectHorizons:TheHammerFindsaBiggerNail:CellTherapyforAutoimmuneDisorders.”28FoodandDrugAdministration,“FDAInvestigatingSeriousRiskofT-cellMalignancyFollowingBCMA-DirectedorCD19-DirectedAutologousChimericAntigenReceptor(CAR)TcellImmunotherapies.”/vaccines-blood-biologics/safety-availability-biologics/fda-investigating-serious-risk-t-cell-malignancy-following-bcma-directed-or-cd19-directed-autologous29MananShahetal.,“PromisesandchallengesofadecentralizedCART-cellmanufacturingmodel.”/articles/10.3389/frtra.2023.1238535/full30EvaluatePharma31Reuters,“Insight:Whathappenswhena$2milliongenetherapyisnotenough.”/business/healthcare-pharmaceuticals/what-happens-when-2-million-gene-therapy-is-not-enough-2023-08-12/32Pressrelease,“CRISPRTherapeuticsAnnouncesCompletionofFDAAdvisoryCommitteeMeetingforExagamglogeneAutotemcel(exa-cel)forSevereSickleCellDisease.”/about-us/press-releases-and-presentations/crispr-therapeutics-announces-completion-of-fda-advisory-committee-meeting-for-exagamglogene-autotemcel-exa-cel-for-severe-sickle-cell-disease33Pressrelease,“SangamoTherapeuticsReportsRecentBusinessHighlightsandSecondQuarter2023FinancialResults.”https://investor./news-releases/news-release-details/sangamo-therapeutics-reports-recent-business-highlights-and-634Pressrelease,“FDAAcceptsPfizer’sApplicationforHemophiliaBGeneTherapyFidanacogeneElaparvovec.”https://www.businesswire.com/news/home/20230627431780/en35Pressrelease,“IntelliaTherapeuticsAnnouncesSecondQuarter2023FinancialResultsandHighlightsRecentCompanyProgress.”https:///news-releases/news-release-details/intellia-therapeutics-announces-second-quarter-2023-financial36Pressrelease,“IntelliaTherapeuticsAnnouncesFDAClearanceofInvestigationalNewDrug(IND)ApplicationtoInitiateaPivotalPhase3TrialofNTLA-2001fortheTreatmentofTransthyretin(ATTR)AmyloidosiswithCardiomyopathy.”/news-releases/news-release-details/intellia-therapeutics-announces-fda-clearance-investigational-037Pressrelease,“MerckandModernaInitiatePhase3StudyEvaluatingV940(mRNA-4157)inCombinationwithKEYTRUDA?(pembrolizumab)forAdjuvantTreatmentofPatientswithResectedHigh-Risk(StageIIB-IV)Melanoma.”/news/merck-and-moderna-initiate-phase-3-study-evaluating-v940-mrna-4157-in-combination-with-keytruda-pembrolizumab-for-adjuvant-treatment-of-patients-with-resected-high-riskstage-iib-iv-melanom/38Reuters,“Mercksigns$5.5billiondealwithDaiichiforcancertherapydevelopment.”/business/healthcare-pharmaceuticals/daiichi-sankyo-merck-team-up-22-bln-collaboration-sankyos-antibody-drug-2023-10-19/39FiercePharma,“AZ,Daiichi’sEnhertupoststrialwininnumerouscancertypes.Isatumor-agnosticapprovalnext?”https://www./pharma/az-daiichis-enhertu-fights-various-cancers-tumor-agnostic-approval-next40Pressre