Alnylam Pharmaceuticals, Inc. to Share Progress Across its Transthyretin Amyloidosis Franchise Including Additional Analyses of the Helios-B Phase 3 Study Results at Heart Failure 2025 Congress
Published on 05/12/2025
at 12:00 pm BST - Modified on 05/12/2025
at 12:50 pm BST
Alnylam Pharmaceuticals, Inc. announced that the Company will present the latest data from its transthyretin amyloidosis (TTR) franchise at the upcoming Heart Failure 2025 Congress, a scientific congress of the European Society of Cardiology, taking place May 17-20 in Belgrade, Serbia. The latest analyses of the HELIOS-B Phase 3 study of vutrisiran in patients with ATTR amyloidosis with cardiomyopathy (ATTR-CM), including further outcomes data on cardiovascular hospitalizations and urgent heart failure visits up to 42-months, will be presented as a late-breaking abstract in the "H hottest Trials and Trial Updates 1" session. Data from the HELIOS-B study supported the recent approvals of AMVUTTRA®?
(vutrisiran) for the treatment of the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis in adults in the U.S. and Brazil. These data also supported the positive opinion from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) recommending approval of vutrisiran for the same indication. AMVUTTRA is an RNAi therapeutic that works upstream to deliver rapid knockdown of transthyretin, addressing the disease at its source, with four subcutaneous doses per year.
Additional updates to be presented include the design and rationale of the TRITON-CM Phase 3 study of nucresiran (ALN-TTRsc04), an investigational next-generation TTR silencer, in patients with ATTR-CM, as well as an additional analysis from the HELIOS- B study of vutrisirAN in patients with ATTR- CM who experienced disease progression while being treated with tafamidis.
Alnylam Pharmaceuticals, Inc. is a biopharmaceutical company. The Company is engaged in discovering, developing, manufacturing and commercializing novel therapeutics based on ribonucleic acid interference (RNAi). Its marketed products include AMVUTTRA (vutrisiran) for the treatment of hereditary transthyretin-mediated amyloidosis (hATTR) with polyneuropathy in adults; ONPATTRO (patisiran) for the treatment of the polyneuropathy of hATTR amyloidosis in adults; GIVLAARI (givosiran) for the treatment of adults with acute hepatic porphyria; OXLUMO (lumasiran) for the treatment of primary hyperoxaluria type 1, and Leqvio (inclisiran), which is being developed and commercialized by its partner, Novartis AG, for the treatment of adults with hypercholesterolemia or mixed dyslipidemia. In addition, the Company develops Cemdisiran to treat complement-mediated diseases, Belcesiran for the treatment of alpha-1 liver disease, Elebsiran, Zilebesiran, ALN-APP, ALN-HSD, Fitusiran, and others.
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Alnylam Pharmaceuticals, Inc. to Share Progress Across its Transthyretin Amyloidosis Franchise Including Additional Analyses of the Helios-B Phase 3 Study Results at Heart Failure 2025 Congress
