Alnylam Pharmaceuticals Inc 2025年度报告

ALYSSAGENE POSITIVE FORHUNTINGTON’SDISEASE, USA 81% YEAR-OVER-YEARPRODUCT REVENUEGROWTH VS 2024 70+ $3BCOMBINED 2025 NETPRODUCT REVENUE COUNTRIES WITHCOMMERCIAL PRESENCE(DIRECT OR THROUGHDISTRIBUTOR) We ended 2025 with a cash and investment balance ofapproximately $2.9 billion and achieved GAAP profitabilityfor the first time, a pivotal milestone that reflects ourstrong growth and financial discipline. Sustainingprofitability will allow us to fund the investment we planin our burgeoning pipeline of new medicines that mayhave the potential to prevent, halt, or reverse disease.In doing so, we are advancing the vision that has guidedAlnylam since its founding: realizing RNA interferenceas a new class of medicines capable of transforminghuman health. 2025 was a transformationalyear for Alnylam, markedby exceptional progressacross the business andthe remarkable start to thelaunch of AMVUTTRA®(vutrisiran) for patients withATTR cardiomyopathy. In 2025, we made significant progress across our pipeline,which includes a number of medicines in developmentwith multi-billion-dollar revenue potential. We initiated3 Phase 3 studies: the TRITON-PN and TRITON-CM studiesof our next-generation TTR silencer nucresiran, and theZENITH cardiovascular outcomes trial of zilebesiran forhypertension, a study that underscores our growingcommitment to cardiovascular disease. The AMVUTTRA launch has fundamentally changed ourtrajectory—accelerating our growth, enabling greaterinvestment in our next wave of medicines, and opening anew era of unprecedented opportunity for the company. Within neuroscience, we are advancing our programswith urgency and conviction given the enormous unmetmedical need in this field. Mivelsiran is designed to preventamyloid deposition in the brain and is being evaluated ina Phase 2 study in cerebral amyloid angiopathy, with aPhase 2 study in Alzheimer’s disease planned to begin inthe first half of 2026. We are also advancing ALN-HTT02in a Phase 1 study for Huntington’s disease, with initialresults expected this year. AMVUTTRA has the potential to become the new standardof care in the large and growing TTR market. Its increasinguse by both newly diagnosed patients and those whosedisease is progressing on other therapies underscores itscompelling clinical profile and reflects strong engagementfrom physicians, patients, and payers. Outside the U.S.,regulatory approvals in Japan, the EU, and numerousadditional markets will enable us to deliver AMVUTTRA topatients across the globe. The AMVUTTRA launch drove our robust commercialperformance in 2025, with combined net product revenuerising to $2.987 billion, 81% greater than in 2024. TTRFranchise revenue doubled from the prior year, withsales of AMVUTTRA and ONPATTRO® (patisiran) generating$2.487 billion—or more than $800 million above ouroriginal 2025 guidance. Our Rare Disease businesscontinued its steady growth, with sales of GIVLAARI®(givosiran) and OXLUMO® (lumasiran) increasing 18%year-over-year to $500 million. Sustaining profitabilitywill allow us to fund theinvestment we plan in ourburgeoning pipeline of newmedicines that may havethe potential to prevent,halt, or reverse disease.” Leqvio® (inclisiran), a product discovered by Alnylam,has become one of Novartis’ top-selling medicines, whileour partner Sanofi received FDA approval in 2025 forQfitlia® (fitusiran), another Alnylam-discovered medicine,highlighting the power of our RNAi platform and thegenerational technology we are advancing. 2.5K+ Employeesworldwide TTR Franchiserevenue vs 2024 Goals achievedby end of 2025 Phase 3 studiesinitiated in 2025 25+ Programs in clinicaldevelopment by endof 2025 Within hematology, we are focused on advancingALN-6400, which holds exciting potential toaddress a wide range of bleeding disorders, with aPhase 2 study underway now for a lead indication,hereditary hemorrhagic telangiectasia. In metabolic diseases, we see a compelling opportunityto address significant gaps in treatment left by GLP-1medicines. ALN-4324 holds the potential to be a novelinsulin sensitizer for people with type 2 diabetes, whileALN-2232 is our first candidate for obesity and weight Within hematology, weare focused on advancingALN-6400, which holdsexciting potential toaddress a wide range ofbleeding disorders...” Continuing our long heritage of setting and achieving bold5-year goals, we have launched ourAlnylam 2030strategy,which reflects our enormous ambition for the companyand commits us to achieving market leadership in ATTRamyloidosis, driving long-term growth through sustainableinnovation, and delivering exceptional financial resultswith discipline and agility. We are building an extraordinary company with thepotential to help millions of people. In the transformationalyear just concluded, we firmly established Alnylam amongthe top biotechnology companies in the world. Now weintend to continue our ascent, advancing our science,expanding our reach, and delivering even gre