Alnylam Pharmaceuticals Inc 2024年度报告

partners.Wealso advanced exciting effortstodeliverRNAitherapeutics tonewtissues, whichwillallowustobroadentherangeofdiseases we seektotreat. world,submittingregulatory applicationsintheU.S.,EU,Japan, andothermarkets, w ithadditionalfilingstofollowin2025. Aswe grow, we continuetobuilda culturethatinspires,supports, andattractspassionate peoplewhoare eagertotake onthechallenges inherent in developingimportantnew medicines. Alnylam was widely recognized againlast yearasanexceptionalworkplace by numerousorganizations, includingUSAToday,ScienceMagazine,TheBoston Globe,andLinkedln.Wealso earned certificationasa GreatPlacetoWork®i n11countries and ranked amongFortune'sbest workplacesforparents and millennials. INCOMBINEDGLOBALPRODUCTREVENUES Ourambitiontoestablish Alnylamastheleader inATTRamyloidosis is reflected inourcontinued investment inpotential new treatmentoptionsfor patients beyondvutrisiran.Wereportedinterimpositive resu Its from aPhase1studyofournext-generationproductcandidateindevelopment forthetreatmentofATTRamyloidosis,nucresi ran(ALN-TTRsc04), which delivered profoundTTRlowering withbiannual dosing.Weplantobegin pivotalstudiesinbothATTRcardiomyopathy and polyneuropathyinthefirsthalfof2025. INVESTIGATIONALNEWDRUGAPPLICATIONFILINGSEXPECTEDIN2025FORALNYLAM-LEDPROGRAMS Finally,wewere featured o nNewsweek'slistofAmerica'sMostSociallyResponsible Companiesforthethirdyear ina row, and maintained an 'A' Environmental, Social, andGovernance rating withMSCI.Inour annual CorporateResponsibilityReport,weoutlinedourstrongcommitmenttoenabling accesstoourmedicines, engaging inourcommunities,promoting health equity, andmitigatingourenvironmenta l impact.+ RNAitherapeuticsholdthe power to amplify lifebysilencingdisease,andweareleading the RNAi revolutionthatischangingmedicine." PROGRAMSIN CLINICAlMENTBYTHEENDOF2025 Ourmid-stage pipeline features groundbreakingopportunitiesfortransformational medicines poisedtomake a profound impacton human health.Wemadeimportantprogress w ithZilebesiranforpeoplewithhypertension,reportingpositive resultsfromtheKARDIA-2Phase2studyshowing significantadditive blood pressureloweringin combinationwithadditional standard-of-carehypertensivemedicines.Weplantobegin a cardiovascularoutcomestrial this year. Similarly,we reported positiveinit ial resultsfromthemultiple doseportionofthePhase1studyofmivelsiraninearlyonset Alzheimer's disease,init iated aPhase2 studyofmivelsiranincerebral amyloidangiopathy, and began a Phase 1 studyofALN-HTT02,bringinghopetopatientswithHuntington's disease. Four years ago, w iththelaunchofourpsx25strategy,we soughttoestablish Alnylam amongtheforemostbiotechnology companies.Nowwe are focusedonournextchapter.RNAitherapeuticslholdthepowertoamplifylifeby silencing disease. Themomentinfrontofusismorepromising than any before andtheopportunit iesmoreextraordinary. ForAlnylam,webelievethebest is yettocome. YVONNEGREENSTREET,MD,MBACHIEFEXECUTIVE OFFICER WeexpandedourpipelinewithfourproprietaryClinicalTrial Applications and applicationsbyourcollaboration OUR ELINE COMMITMENTTOSCIENCE& INNOVATION MIVELSIRANisour investigationalRNAitherapeutic targeting amyloid precursor proteinfor the treatment of Alzheimer'sdisease(AD)andcerebral amyloid angiopathy(CAA).In2024,weinitiated aPhase2 studyofmivelsiraninCAA,the second most commoncauseof hemorrhagicstroke. AdditionalPhase1resultsandPhase2 study initiationinAD-theleadingtype ofdementia-areexpected in2025. The advancementofourflagshipTTRfranchise is driving a new chapterofgrowthand innovation for Alnylam.Weare investing inourrapidly expandingpipeline and developing programs acrossmultiple therapeutic areas for rare andcommondiseases w ithhighunmetneed.These are some highlights: ZILEBESIRANisour investigationalRNAitherapeuticindevelopment for the treatment ofhypertension inhighunmetneedpopulations.In2024,wereported positivePhase2 results fromKARDIA-2;patients with inadequately controlledhypertension showed significant additive loweringofbloodpressureon topofa standard-of-careoral antihypertensive.In2025,we expecttoshareresults from theKARDIA-3Phase2studyandtoinitiate aPhase3 cardiovascular outcomes trial;this programisbeing developedincollaborationwithRoche. ALN-HTT02isour investigationalRNAitherapeutic targeting huntingtin(HTT)indevelopment for the treatment of Huntington'sdisease(HD).HDisaprogressive,fatalneurodegenerativediseasewith no approveddisease-modifying treatments. Availabletreatment optionsarelimited to symptommanagement, highlighting the urgentneedfornewtherapies.In2024,we initiated aPhaselbstudy ofALN-HTT02;this programisbeingdevelopedincollaboration withRegeneron. Ourworkextends far beyondthetherapieswedevelop. Corporate responsibility isintegratedthroughoutourbusiness,fromthe way we listentopatients anddesign clinical trials,tohowweempowerouremployees, advance ethica l standards,and reduceourenvironmental impact.Weensureourbusiness initiativesemphasize sustainabilityandtheinclusionofenvironmental,