May 12, 2025 Press Release for Alnylam

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

May 12, 2025

CAMBRIDGE, Mass.--(BUSINESS WIRE)--May 12, 2025-- 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
Session: Novel Insights into Heart Failure Therapeutics
Monday, May 19, 9:00 – 10:00 CEST, 3:00 – 4:00 A.M. EST
Presenter: Antoine Bondue, Belgium

Design and Rationale of a Phase 3 Study to Evaluate Efficacy and Safety of Nucresiran (ALN-TTRsc04) in Patients with Transthyretin Amyloidosis with Cardiomyopathy
Session: Research Methodology
Monday, May 19, 14:00 – 15:00 CEST, 8:00 – 9:00 A.M. EST
Presenter: Marianna Fontana, United Kingdom

Vutrisiran In Patients With Transthyretin Amyloidosis with Cardiomyopathy In HELIOS-B Who Had Progressed On Tafamidis
Session: Evolving Treatment Paradigms in Heart Failure: SGLT2 Inhibition to TTR Stabilisation and Beyond
Tuesday, May 20, 9:21 – 9:30 CEST, 3:21 – 3:30 A.M. EST
Presenter: Jose Gonzalez-Costello, Spain

About AMVUTTRA^® (vutrisiran)
AMVUTTRA^® (vutrisiran) is an RNAi therapeutic that delivers rapid knockdown of variant and wild-type transthyretin (TTR), addressing the underlying cause of transthyretin (ATTR) amyloidosis. Administered quarterly via subcutaneous injection, vutrisiran is approved and marketed in more than 15 countries for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR-PN) in adults. In Europe, it is administered as a subcutaneous injection once every three months, either by a healthcare professional, or self-administered by patients or their caregivers. Vutrisiran is also in development for the treatment of ATTR amyloidosis with cardiomyopathy (ATTR-CM), which encompasses both wild-type and hereditary forms of the disease.

About ATTR
Transthyretin amyloidosis (ATTR) is an underdiagnosed, rapidly progressive, debilitating and fatal disease caused by misfolded transthyretin (TTR) proteins, which accumulate as amyloid deposits in various parts of the body, including the nerves, heart and gastrointestinal tract. Patients may present with polyneuropathy, cardiomyopathy, or both manifestations of disease. There are two different forms of ATTR – hereditary ATTR (hATTR), which is caused by a TTR gene variant and affects approximately 50,000 people worldwide, and wild-type ATTR (wtATTR), which occurs without a TTR gene variant and impacts an estimated 200,000 – 300,000 people worldwide.^1-4

About RNAi
RNAi (RNA interference) is a natural cellular process of gene silencing that represents one of the most promising and rapidly advancing frontiers in biology and drug development today. Its discovery has been heralded as “a major scientific breakthrough that happens once every decade or so,” and was recognized with the award of the 2006 Nobel Prize for Physiology or Medicine. By harnessing the natural biological process of RNAi occurring in our cells, a new class of medicines known as RNAi therapeutics is now a reality. Small interfering RNA (siRNA), the molecules that mediate RNAi and comprise Alnylam’s RNAi therapeutic platform, function upstream of today’s medicines by potently silencing messenger RNA (mRNA) – the genetic precursors – that encode for disease-causing or disease pathway proteins, thus preventing them from being made. This is a revolutionary approach with the potential to transform the care of patients with genetic and other diseases.

About Alnylam Pharmaceuticals
Alnylam (Nasdaq: ALNY) has led the translation of RNA interference (RNAi) into a whole new class of innovative medicines with the potential to transform the lives of people afflicted with rare and prevalent diseases with unmet need. Based on Nobel Prize-winning science, RNAi therapeutics represent a powerful, clinically validated approach yielding transformative medicines. Since its founding in 2002, Alnylam has led the RNAi Revolution and continues to deliver on a bold vision to turn scientific possibility into reality. Alnylam has a deep pipeline of investigational medicines, including multiple product candidates that are in late-stage development. Alnylam is executing on its “Alnylam P⁵x25” strategy to deliver transformative medicines in both rare and common diseases benefiting patients around the world through sustainable innovation and exceptional financial performance, resulting in a leading biotech profile. Alnylam is headquartered in Cambridge, MA.

¹ Hawkins PN, Ando Y, Dispenzeri A, et al. Ann Med. 2015;47(8):625-638.
² Gertz MA. Am J Manag Care. 2017;23(7):S107-S112.
³ Conceicao I, Gonzalez-Duarte A, Obici L, et al. J Peripher Nerv Syst. 2016;21:5-9.
⁴ Ando Y, Coelho T, Berk JL, et al. Orphanet J Rare Dis. 2013;8:31.

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Christine Regan Lindenboom
(Investors and Media)
+1-617-682-4340

Josh Brodsky
(Investors)
+1-617-551-8276

Source: Alnylam Pharmaceuticals, Inc.