Alnylam to Share Progress Across its Transthyretin Amyloidosis Franchise Including Additional Analyses of the HELIOS-B Phase 3 Study Results at Heart Failure 2025 Congress

Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced that the Company will present the latest data from its flagship 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 “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.

Presentation Details

Vutrisiran Reduces All-Cause Mortality, Cardiovascular Mortality, and Cardiovascular Events in Patients with Transthyretin Amyloid Cardiomyopathy: Analysis from the HELIOS-B Trial
Session: Hottest Trials and Trial Updates 1
Saturday, May 17, 11:50 – 11:58 CEST, 5:50 – 5:58 A.M. EST
Presenting Author: Marianna Fontana, United Kingdom

Clinical Presentation and Treatment Landscape of Patients with Transthyretin Amyloidosis With Cardiomyopathy: A Real-world Study in Five European Countries and Japan
Session: Novel Insights into Heart Failure Therapeutics
Sunday, May 18, 13:00 – 13:45 CEST, 7:00 – 7:45 A.M. EST
Presenter: Caroline Morbach, Germany

Utility of Genetic Testing For Diagnosing hATTR Patients: Results from a European and Middle East Genetic Testing Program