2024年医药研发年度回顾(英文)

WhiľePaper PharmaR&D AnnualReview2024 Lookbackaľkeypipelineľrendsfromľhelasľ12monľhssoyoucanforecasľsuccessin2024 Conľenľs Welcome03 Inľroducľion:ToľalPipelineSize05 Pleasanľouľlookforpharmaasľhingsconľinueľoheaľup DevelopingSysľems:The2024PipelinebyPhase08 Clinicaldevelopmenľadvancessľillfaceasľiffheadwind TopCompanies11 Someenjoyamomenľinľhesun,buľforoľhersľhefuľurelooksmorehazy TherapiesandDiseases19 CancersľillľakingR&Dbysľorm RegionalVariaľions31 Whereľheouľlookseemsľobebrighľesľ MechanismsandTargeľs54 Immuno-oncologymakeshaywhileľhesunshines TypesofPipelineDrugs59 Isľheconľinuedmoveinľobioľechsľarľingľomeeľafrosľyrecepľion? Whaľ’sľheForecasľforPharma?62 Plenľyofraysofsunshineshouldholdoffanysľormcloudsonľhehorizon AbouľľheAuľhor65 Welcome WelcomeľoPharmaprojecľs’2024reviewofľrendsinpharmaceuľicalR&D WelcomeľoPharmaprojecľs’2024reviewofľrendsinpharmaceuľicalR&D.Forover30yearsnow,I’vebeenľakinganannual lookaľľheevoluľionofpharmaR&D,andinľhisarľicleI’llľakeľheľemperaľureofľheindusľryaľľhesľarľof2024.We’llassessindusľryľrendsbyexaminingľhepipelinebycompany,ľherapeuľicarea,disease,ľargeľ,anddrugľype,usingdaľaprimarilyfromPharmaprojecľs,parľofľheCiľelinesuiľe ofproducľs,whichhasbeenľrackingglobaldrugdevelopmenľsince1980.ThisreporľwillbefollowedupbyourannualsupplemenľreviewingľheNewAcľiveSubsľance(NAS)launchesforľheyearjusľpassed.Buľhere,we’llexaminehowľhepharmaR&Dclimaľeischanging,lookaľwhereľhepressureisrisingandfalling,andľryľodeľerminewhereľheouľlookissunnyandwheresľormcloudsaregaľhering.Hopefully,iľwillhelpyouľoľakeľheindusľry’sľemperaľureandforecasľ iľsouľlook. Regularreadersofľhisreporľ(whichhasbeenrunningsince1993,soispresenľedhereiniľs32ndediľion)willknowľhaľ,inrecenľyears,I’veľhreadedadifferenľľhemeľhrougheachediľion,ľohighlighľpoinľs,ľodrawanalogies,andľoaddaliľľlecharacľerinľowhaľcouldoľherwisebearaľherlengľhynarraľiveľhroughaparadeofsľaľisľics,charľs,andľables—dullerľhanaDecemberdayinDoncasľer.Themesselecľedsofarhaveincludedasľronomy,movies,ľhenaľuralworld,music,foodanddrink,scienceficľion,ľravel,and,lasľyear,liľeraľure.Thisyear,asyoumayhaveguessed,Idecidedľohangmy(rain)haľonsomeľhingľhaľaffecľsusalleveryday:ľheweaľher,andľhealliedmosľimporľanľissueofourľimes,climaľechange. Sinceourearlycave-dwellingdays,ľheacľiviľiesofhumanshavebeensomewhaľgoverned byľhevagariesofľheweaľher.Weneededľoknowwhencondiľionswerebesľforhunľing,andwheľherourcropswouldflourishorfail.Theearliesľaľľempľsľopredicľľheweaľherwerelargelyunscienľificandbasedonprophecy,asľrology,orprayersľoinvisibleweaľhergods.Aswiľhsomanyľhings,ľheancienľGreekswereľhefirsľľoľurnsupersľiľioninľoscience,and infacľľheľerm“meľeorology”comesfromľheGreekmeaning“ľhesľudyofľhingshighinľheair.”Theacknowledgedfaľherofľhescience isArisľoľle,whopublishedaľreaľiseonľhesubjecľasearlyas350BC.Ofcourse,accuraľeinsľrumenľsľomeasureweaľher-relaľedphenomenaonlyreallycamealongwiľhľheEnlighľenmenľ,buľweaľherforecasľsweresľillnoľoriouslyunreliable,especiallyincounľrieslikeľheUKwhereIambased,asliľľleas50yearsago.Iľwasonlywiľhľhegrowľhofcompuľingpower,andwiľhiľ,compuľermodelingalgoriľhms,ľhaľforecasľerswereableľoprovidepredicľionswiľhanymeasureofcerľainľy.Thekey,asalways,isdaľa—andloľsofiľ.Myhopeisľhaľľhisreporľwillfulfillľhesamefuncľion: iľwillprovideenoughinformaľionforyouľodeľerminewhichparľsofľheindusľryarelikelyľobebaskinginunbrokensunshine,andwhicharepreparingforaperiodofunseľľledcondiľions. Whileourprimaryfocusisnaľurallyonľheshorľľerm;i.e.,wheľherweneedľopuľonanexľrajumperorľobringourumbrellas,ľhelong-ľermľrendsarewhaľexerciseľhemindsofboľhclimaľescienľisľsandpharmaindusľryfollowers.Inľhepasľfewdecades,ľheľopicofľheweaľherhasgrownfrombeingaparochial consideraľionľosomeľhingľhreaľeningourveryexisľence.Buľ,infacľ,ľheideaľhaľourimpacľonľheplaneľischangingiľirrevocablyhasbeenaroundlongerľhanyoumighľľhink.Iľwasaľľheendofľhe19ľhcenľurywhenscienľisľsfirsľpredicľedľhaľchangesinaľmosphericcarbondioxidelevelscouldsubsľanľiallyalľeraplaneľ’ssurfaceľemperaľureľhroughľhegreenhouseeffecľ,andjusľpriorľoWorldWarIIwhenGuyCallendarconnecľedcarbondioxideincreases inEarľh’sownaľmosphereľoglobalwarming.Parľicularlysinceľheľurnofľhemillennium,ľheevidenceseemsirrefuľableľhaľ,unlessacľionisľakenandľakenfasľ,wearebuildingľowardsaľhreaľľoourconľinuedexisľence.Thepharmaindusľryhasofcourseweaľheredamoreacuľeexisľenľialľhreaľrecenľly—ľheCOVID-19pandemic,whichiľhelpedľodeliverusfrom—andhasemergedľoenjoyamomenľinľhesun.Buľboľhclimaľechangeandľhecoronaviruscrisisareevidenceofľhefacľľhaľweneedľoidenľify—andacľon—emergingľrendsveryquickly.Wehaveľhedaľa;iľ’swhaľwedowiľhiľľhaľcounľs. Ofcourse,changesinclimaľepaľľernspredaľehumansandľheľransformaľionaleffecľofľheindusľrialrevoluľion.Wiľhinľhepasľmillionyears,ľherehavebeenaľleasľeighľcyclesoficeagesandwarmerperiods,alongwiľhshorľercyclessuchasľheLiľľleIceAgeinľheNorľhAľlanľicbeľweenľhe16ľhand19ľhcenľuries,whichfamouslyledľoLondon’sRiverThamesfreezingoverandľheVicľorian“FrosľFairs”heldľhere.Whileclimaľeisboundľohaveinherenľvariabiliľy,ľhisch