Alnylam to Present Progress in Transforming the Treatment of Cardiovascular Disease with RNAi Therapeutics at European Society of Cardiology Congress 2025
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.
About AMVUTTRA ® (vutrisiran)
AMVUTTRA ® (vutrisiran) is an RNAi therapeutic that delivers rapid knockdown of transthyretin (TTR), addressing the underlying cause of transthyretin (ATTR) amyloidosis. It is marketed in more than 15 countries for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR-PN) in adults and it is also approved for the treatment of the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults in the US, Europe, Brazil, Japan and UAE. Administered quarterly via subcutaneous injection, AMVUTTRA is the first and only RNAi therapeutic approved for the treatment of both the cardiomyopathy manifestations of ATTR amyloidosis and the polyneuropathy manifestations of hereditary transthyretin-mediated amyloidosis (hATTR).
For US Media only: For more information about AMVUTTRA, including the full U.S. Prescribing Information, visit AMVUTTRA.com. For EU Media: For the full EU Summary of Product Characteristics, see https://www.ema.europa.eu/en/documents/product-information/amvuttra-epar-product-information_en.pdf.
About Zilebesiran
Zilebesiran is an investigational, subcutaneously administered RNAi therapeutic in development for the treatment of hypertension to reduce cardiovascular risk in high unmet need populations. Zilebesiran targets angiotensinogen (AGT), the most upstream precursor in the Renin-Angiotensin-Aldosterone System (RAAS), a cascade which has a role in blood pressure (BP) regulation. Zilebesiran inhibits the synthesis of AGT in the liver, potentially leading to durable reductions in AGT protein, and ultimately, in the vasoconstrictor angiotensin (Ang) II. Zilebesiran utilizes Alnylam's Enhanced Stabilization Chemistry Plus (ESC+) GalNAc-conjugate technology, which enables infrequent biannual subcutaneous dosing, increased selectivity and the potential to achieve continuous control of BP to impact cardiovascular risk. The safety and efficacy of zilebesiran have not been established or evaluated by the FDA, EMA or any other health authority. Zilebesiran is being co-developed and co-commercialized by Alnylam and Roche.
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 Cardiovascular Disease and Hypertension
Cardiovascular disease (CVD) is a global health crisis and a leading cause of death worldwide, responsible for approximately 20 million deaths annually. 5,6 Hypertension is the primary cause of and number one modifiable risk factor for CVD. 7 An estimated 1 in 3 adults worldwide have hypertension, and, despite wide availability of antihypertensives, up to 80% of all patients, and up to a third of treated patients, do not reach and maintain blood pressure (BP) targets. 8 Even when blood pressure appears well managed, continuous control of BP may remain suboptimal, leading to variability in BP during the 24-hour period and in the long-term, putting patients at greater risk of cardiovascular events and end organ damage. 9-15 These patients require novel approaches that not only reduce BP, but also lower overall cardiovascular risk.
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. 16 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. 17 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. 16 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 P 5 x25 ' 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.
Alnylam Forward-Looking Statements
This press release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. All statements other than historical statements of fact regarding Alnylam's expectations, beliefs, goals, plans or prospects including, without limitation, Alnylam's expectations regarding the potential of RNAi therapeutics to transform the treatment of cardiovascular disease and genetic and other conditions; Alnylam's ability to advance innovative therapies for patients living with rare and more prevalent conditions underserved by current treatment options; the safety and efficacy of vutrisiran for the treatment of ATTR-CM and the safety and efficacy of zilebesiran for the treatment of hypertension; and Alnylam's ability to achieve its ' Alnylam P 5 x25 ' strategy should be considered forward-looking statements. Actual results and future plans may differ materially from those indicated by these forward-looking statements as a result of various important risks, uncertainties and other factors, including, without limitation, risks and uncertainties relating to Alnylam's ability to successfully execute on its ' Alnylam P 5 x25 ' goals; Alnylam's ability to discover and develop novel drug candidates and delivery approaches and successfully demonstrate the efficacy and safety of its product candidates; the pre-clinical and clinical results for Alnylam's product candidates; actions or advice of regulatory agencies and Alnylam's ability to obtain and maintain regulatory approval for its product candidates, as well as favorable pricing and reimbursement; successfully launching, marketing and selling Alnylam's approved products globally; delays, interruptions or failures in the manufacture and supply of Alnylam's product candidates or its marketed products; obtaining, maintaining and protecting intellectual property; Alnylam's ability to manage its growth and operating expenses through disciplined investment in operations and its ability to achieve a self-sustainable financial profile in the future; Alnylam's ability to maintain strategic business collaborations; Alnylam's dependence on third parties for the development and commercialization of certain products; the outcome of litigation; the potential risk of future government investigations; and unexpected expenditures; as well as those risks more fully discussed in the 'Risk Factors' filed with Alnylam's 2024 Annual Report on Form 10-K filed with the Securities and Exchange Commission (SEC), as may be updated from time to time in Alnylam's subsequent Quarterly Reports on Form 10-Q, and in other filings that Alnylam makes with the SEC. In addition, any forward-looking statements represent Alnylam's views only as of today and should not be relied upon as representing Alnylam's views as of any subsequent date. Alnylam explicitly disclaims any obligation, except to the extent required by law, to update any forward-looking statements.
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 GBD 2021 Causes of Death Collaborators. Lancet. 2024;403:2100-2132.
6 Lindstrom M, DeCleene N, Dorsey H, et al. J Am Coll Cardiol. 2022;80:2372-2425.
7 Yusuf S, Joseph P, Rangarajan S, et al. Lancet. 2020;395:795-808.
8 NCD Risk Factor Collaboration (NCD-RisC). Lancet. 2021;398:957-980.
9 Ebinger JE, Driver M, Ouyang D, et al. eClinicalMedicine. 2022;48:101442.
10 Kario K. Prog Cardiovasc Dis. 2016;59:262-281.
11 Doumas M, Tsioufis C, Fletcher R, et al. J Am Heart Assoc. 2017;6:e006093.
12 Mezue K, Goyal A, Pressman GS, et al. J Clin Hypertens. 2018;20:1247-1252.
13 Rothwell PM, Howard SC, Dolan E, et al. Lancet. 2010;375:895-905.
14 Tatasciore A, Renda G, Zimarino M, et al. Hypertension. 2007;50:325-332.
15 Mokadem ME, Boshra H, El Hady YA, et al. J Hum Hypertens. 2019;34:641-647.
16 Elbashir SM, Harborth J, Lendeckel W, et al. Nature. 2001;411(6836):494-498.
17 Zamore P. Cell. 2006;127(5):1083-1086.
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