Oct 30, 2024 Press Release for Alnylam
Alnylam to Showcase Latest Data from TTR Franchise and Hypertension Program at American Heart Association Scientific Sessions 2024
Oct 30, 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
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
Presenting Author:
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)
Presenting Author:
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)
Presenting Author:
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)
Presenting Author:
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
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
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1 Hawkins PN, Ando Y, Dispenzeri A, et al. Ann Med. 2015;47(8):625-638. |
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2 Gertz MA. Am J Manag Care. 2017;23(7):S107-S112. |
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3 Conceicao I, Gonzalez-Duarte A, Obici L, et al. J Peripher Nerv Syst. 2016;21:5-9. |
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4 Ando Y, Coelho T, Berk JL, et al. Orphanet J Rare Dis. 2013;8:31. |
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5 Elbashir SM, Harborth J, Lendeckel W, et al. Nature. 2001;411(6836):494-498. |
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6 Zamore P. Cell. 2006;127(5):1083-1086. |
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VP, Investor Relations & Corporate Communications investors@alnylam.com 617-551-8276
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