Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced that the Company will present data from its transthyretin amyloidosis (ATTR) and hypertension programs at the upcoming American Heart Association (AHA) Scientific Sessions 2024, November 16–18, 2024.

Data presentations for the Company’s flagship TTR franchise include new findings from an analysis of the HELIOS-B Phase 3 study of vutrisiran, an RNAi therapeutic in development for the treatment of ATTR amyloidosis with cardiomyopathy (ATTR-CM), as well as interim results from a Phase 1 study evaluating ALN-TTRsc04, a next generation RNAi therapeutic in development for the treatment of ATTR amyloidosis. Full results from the HELIOS-B study, the largest and most contemporary ATTR-CM study to date, were published in The New England Journal of Medicine earlier this year. The study achieved all 10 pre-specified primary and secondary endpoints. In addition, the Company will present a new analysis of the KARDIA-1 Phase 2 study of zilebesiran, an investigational RNAi therapeutic in development for the treatment of hypertension.

ATTR Abstracts A Phase 1, Single Ascending Dose Study to Evaluate ALN-TTRsc04, a Next-Generation RNA Interference Therapeutic, in Healthy Participants for Potential Treatment of Transthyretin Amyloidosis Poster 4135443: Session: Cutting Edge Clinical Cardiomyopathies Research (Abstract Oral Session) Sunday, November 17, 2024, 8:30 a.m. (CST), 9:30 a.m. (EST) Presenting Author: Ali Murad

Impact of vutrisiran on outpatient worsening heart failure in patients with transthyretin amyloidosis with cardiomyopathy in the HELIOS-B trial Poster 4171680: Session: Amyloid, Hypertrophic, and Danon Cardiomyopathies: Targeted Therapies and Specific Populations (Featured Science) Monday, November 18, 2024, 10:21 a.m. (CST), 11:21 a.m. (EST) Presenting Author: Marianna Fontana

Hypertension Abstracts Impact of Long-Term Blood Pressure Variability on Adverse Cardiovascular Outcomes in High- and Low-Risk Populations Poster MDP1200: Session: The Threats of Hypertension to Life and Healthy Organ Function (Moderated Digital Poster Session) Sunday, November 17, 2024, 10:40 a.m. (CST), 11:40 a.m. (EST) Presenting Author: Lynne Krohn

Home Blood Pressure Reductions with Zilebesiran in Patients with Mild-To-Moderate Hypertension are Consistent with Ambulatory and Office Blood Pressure Reductions in the KARDIA-1 Study Poster MDP746: Session: Hypertension Gets Exciting! New Drugs! New Technologies! What Should I Choose? (Moderated Digital Poster Session) Sunday, November 17, 2024, 3:45 p.m. (CST), 4:45 p.m. (EST) Presenting Author: Dion Zappe

About AMVUTTRA® (vutrisiran)

AMVUTTRA® (vutrisiran) is an RNAi therapeutic that delivers rapid knockdown of mutant and wild‑type transthyretin (TTR), addressing the underlying cause of transthyretin (ATTR) amyloidosis. Administered quarterly via subcutaneous injection, AMVUTTRA is approved and marketed in more than 15 countries for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR-PN) in adults. 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. For more information about AMVUTTRA, including the full U.S. Prescribing Information, visit AMVUTTRA.com.

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.5 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.6 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.5 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. For more information about our people, science and pipeline, please visit www.alnylam.com and engage with us on X (formerly Twitter) at @Alnylam, or on LinkedIn, Facebook, or Instagram.

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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.

5 Elbashir SM, Harborth J, Lendeckel W, et al. Nature. 2001;411(6836):494-498.

6 Zamore P. Cell. 2006;127(5):1083-1086.

 

Alnylam Pharmaceuticals, Inc.

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

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

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