Alnylam to Showcase Latest Data from TTR Franchise and Hypertension Program at American Heart Association Scientific Sessions 2024
Alnylam Pharmaceuticals (Nasdaq: ALNY) will present data from its ATTR amyloidosis and hypertension programs at the AHA Scientific Sessions 2024. The presentations include new analysis from the HELIOS-B Phase 3 study of vutrisiran for ATTR amyloidosis with cardiomyopathy, interim results from a Phase 1 study of ALN-TTRsc04, and new analysis of the KARDIA-1 Phase 2 study of zilebesiran for hypertension. The HELIOS-B study, published in The New England Journal of Medicine, achieved all 10 pre-specified primary and secondary endpoints.
Alnylam Pharmaceuticals (Nasdaq: ALNY) presenterà dati sui suoi programmi per l'amyloidosi ATTR e l'ipertensione durante le Sessioni Scientifiche AHA 2024. Le presentazioni includeranno nuove analisi dallo studio di Fase 3 HELIOS-B su vutrisiran per l'amyloidosi ATTR con cardiomiopatia, risultati intermedi da uno studio di Fase 1 di ALN-TTRsc04, e nuove analisi dello studio di Fase 2 KARDIA-1 su zilebesiran per l'ipertensione. Lo studio HELIOS-B, pubblicato nel New England Journal of Medicine, ha raggiunto tutti e 10 i principali obiettivi primari e secondari predefiniti.
Alnylam Pharmaceuticals (Nasdaq: ALNY) presentará datos de sus programas de amiloidosis ATTR y hipertensión en las Sesiones Científicas AHA 2024. Las presentaciones incluirán un nuevo análisis del estudio de Fase 3 HELIOS-B de vutrisiran para la amiloidosis ATTR con cardiomiopatía, resultados intermedios de un estudio de Fase 1 de ALN-TTRsc04 y un nuevo análisis del estudio de Fase 2 KARDIA-1 de zilebesiran para la hipertensión. El estudio HELIOS-B, publicado en el New England Journal of Medicine, alcanzó los 10 objetivos primarios y secundarios predefinidos.
알니람 제약사(나스닥: ALNY)는 2024 AHA 과학 회의에서 ATTR 아밀로이드증 및 고혈압 프로그램에 대한 데이터를 발표할 예정입니다. 발표 내용에는 심근병증이 동반된 ATTR 아밀로이드증 치료를 위한 vutrisiran의 HELIOS-B 3상 연구에서의 새 분석, ALN-TTRsc04에 대한 1상 연구의 중간 결과, 그리고 고혈압 치료를 위한 zilebesiran의 KARDIA-1 2상 연구에 대한 새로운 분석이 포함됩니다. HELIOS-B 연구는 New England Journal of Medicine에 발표되었으며, 사전에 정의된 10개의 주요 및 부차적 목표를 모두 달성했습니다.
Alnylam Pharmaceuticals (Nasdaq: ALNY) présentera des données de ses programmes sur l'amyloïdose ATTR et l'hypertension lors des Sessions Scientifiques AHA 2024. Les présentations incluront une nouvelle analyse de l'étude de phase 3 HELIOS-B sur le vutrisiran pour l'amyloïdose ATTR avec cardiomyopathie, des résultats intermédiaires d'une étude de phase 1 sur l'ALN-TTRsc04, ainsi qu'une nouvelle analyse de l'étude de phase 2 KARDIA-1 sur le zilebesiran pour l'hypertension. L'étude HELIOS-B, publiée dans le New England Journal of Medicine, a atteint tous les 10 objectifs principaux et secondaires prédéfinis.
Alnylam Pharmaceuticals (Nasdaq: ALNY) wird auf den AHA-Wissenschaftlichen Sitzungen 2024 Daten aus seinen ATTR-Amyloidose und Hypertonie-Programmen präsentieren. Zu den Präsentationen gehören neue Analysen aus der HELIOS-B-Studie der Phase 3 zu vutrisiran für ATTR-Amyloidose mit Kardiomyopathie, Zwischenresultate aus einer Phase-1-Studie zu ALN-TTRsc04 und neue Analysen der Phase-2-Studie KARDIA-1 zu zilebesiran für Hypertonie. Die HELIOS-B-Studie, veröffentlicht im New England Journal of Medicine, erreichte alle 10 vorab festgelegten primären und sekundären Endpunkte.
- HELIOS-B Phase 3 study achieved all 10 pre-specified primary and secondary endpoints
- Research published in prestigious The New England Journal of Medicine
- None.
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
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
| _________________________ |
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. |
View source version on businesswire.com: https://www.businesswire.com/news/home/20241030248743/en/
Alnylam Pharmaceuticals, Inc.
Christine Regan Lindenboom
(Investors and Media)
+1-617-682-4340
Josh Brodsky
(Investors)
+1-617-551-8276
Source: Alnylam Pharmaceuticals, Inc.
FAQ
What data will Alnylam (ALNY) present at AHA Scientific Sessions 2024?
What were the results of Alnylam's (ALNY) HELIOS-B Phase 3 study?
When will Alnylam (ALNY) present at AHA Scientific Sessions 2024?
