Healthcare Quality, Patient Safety & Policy

Alnylam to Present RNAi Advances at ESC Congress 2025

Company to Webcast Investor Event on August 30, 2025, at 1:00 p.m. EDT (7:00 pm CEST)

Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced that the Company will present new data from its hypertension and transthyretin amyloidosis (ATTR) programs at the upcoming European Society of Cardiology (ESC) Congress 2025, taking place in Madrid, Spain, from August 29 – September 1, 2025. These presentations will highlight the potential of RNAi therapeutics to transform the treatment of cardiovascular disease and reinforce Alnylam’s commitment to advancing innovative therapies for patients living with rare and more prevalent conditions underserved by current treatment options.

Highlights include data from Cohort A of the KARDIA-3 Phase 2 study evaluating the investigational RNAi therapeutic, zilebesiran, in patients with uncontrolled hypertension and high cardiovascular risk. This analysis will be presented as a late-breaking abstract in the Hot Line 4 session on August 30, 2025.

Data from the Company’s flagship TTR franchise will also be presented, including new analyses from the HELIOS-B Phase 3 study of AMVUTTRA® (vutrisiran), which delivers rapid knockdown of transthyretin, in patients with ATTR amyloidosis with cardiomyopathy (ATTR-CM). These presentations include results of 12-month follow-up from the ongoing open-label extension (OLE) period of HELIOS-B, which will provide further insights into the sustained longer-term benefits of treatment with AMVUTTRA, and an analysis examining the effect of AMVUTTRA on days lost to death and/or hospitalization (DLDH).

ESC Congress Presentation Details

Hypertension Abstracts
KARDIA-3: Zilebesiran as add-on therapy in adults with hypertension and established cardiovascular disease or at high cardiovascular risk
Hot Line 4 Session Presentation
Time: Saturday, August 30, 2025, 4:30 – 4:45 pm (CEST), 10:30 – 10:45 am (EDT)
Presenting Author: Neha Pagidipati, U.S.
Room: Madrid, Main Auditorium

Impact of zilebesiran, an investigational RNA interference therapeutic targeting hepatic angiotensinogen, on renin–angiotensin system biomarkers in patients with mild-to-moderate hypertension
Moderated ePoster: Treatment of Hypertension: Randomized Controlled Trials
Time: Saturday, August 30, 2025, 5:15 – 6:00 pm (CEST), 11:15 am – 12:00 pm (EDT)
Presenting Author: Michael A Weber, U.S.
Room: Station 4, Research Gateway

ATTR Abstracts
Vutrisiran reduces days lost to death and/or hospitalization versus placebo in patients with transthyretin amyloidosis with cardiomyopathy in the HELIOS-B trial
Moderated ePoster: Multidisciplinary Care in Heart Failure
Time: Sunday, August 31, 2025, 9:15 – 10:00 am (CEST), 3:15 – 4:00 am (EDT)
Presenting Author: Mazen Hanna, U.S.
Room: Station 3, Research Gateway

HELIOS-B: 12-month results from the open-label extension period of vutrisiran in patients with transthyretin amyloidosis with cardiomyopathy
Abstract Session: Therapeutic Advances in Amyloid Cardiomyopathy
Time: Sunday, August 31, 2025, 11:15 – 11:25 am (CEST), 5:15 – 5:25 am (EDT)
Presenting Author: Pablo Garcia-Pavia, Spain
Room: Science Box 2 (Research Gateway)

Investor Webcast Information
Alnylam management will discuss the KARDIA-3 results in a live event which will be webcast on August 30, 2025, at 1:00 p.m. ET (7:00 pm CEST). The webcast will be available on the Investors section of the Company’s website at www.alnylam.com/events. An archived webcast will be available on the Alnylam website approximately two hours after the event.

AMVUTTRA® (vutrisiran) INDICATION AND IMPORTANT SAFETY INFORMATION

Indications
In the EU, AMVUTTRA® (vutrisiran) is indicated for the treatment of:

  • hereditary transthyretin amyloidosis in adult patients with stage 1 or stage 2 polyneuropathy (hATTR-PN).
  • wild-type or hereditary transthyretin amyloidosis in adult patients with cardiomyopathy (ATTR-CM).

Important Safety Information

Reduced Serum Vitamin A Levels and Recommended Supplementation
Vutrisiran treatment leads to a decrease in serum vitamin A levels. Supplementation of approximately, but not exceeding, 2500 IU to 3000 IU vitamin A per day is advised for patients taking vutrisiran. Patients should be referred to an ophthalmologist if they develop ocular symptoms suggestive of vitamin A deficiency (e.g., night blindness).

Adverse Reactions
Commonly reported adverse reactions with vutrisiran were injection site reactions and increase in blood alkaline phosphatase and alanine transaminase.

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