Alnylam Presents New HELIOS-B Data on Vutrisiran for Transthyretin Amyloidosis at HFSA 2024
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the foremost company in RNAi therapeutics, has announced new findings from the HELIOS-B Phase 3 study evaluating vutrisiran, an investigational RNAi therapeutic aimed at treating ATTR amyloidosis with cardiomyopathy (ATTR-CM). These findings were unveiled during the Late Breaking Clinical Research Session 1 at the Heart Failure Society of America (HFSA) Annual Scientific Meeting 2024.
ATTR-CM progression is marked by thickening of the cardiac walls, degradation in both systolic and diastolic function, and a rise in cardiac stress and injury biomarkers, specifically NT-pro-BNP and Troponin I. According to Pushkal Garg, M.D., Chief Medical Officer at Alnylam, the latest data illustrate that vutrisiran mitigates disease progression across various aspects of cardiac structure and function, as well as NT-proBNP and Troponin I levels, in a contemporary patient cohort. Dr. Garg emphasized the rapid knockdown of TTR achieved by vutrisiran, leading to early positive effects on cardiac biomarkers and echocardiographic measures, which suggests a disease-modifying potential. He also indicated that vutrisiran could become a first-line therapy for ATTR amyloidosis with cardiomyopathy, with multiple global regulatory submissions expected by the year’s end.
The analysis of echocardiographic data over a 30-month period showed that vutrisiran treatment significantly slowed disease progression in patients with ATTR-CM, as compared to a placebo. The treatment effects of vutrisiran were either comparable or superior in the monotherapy population. Significant improvements in diastolic and systolic functions were recorded at 12 and 18 months, respectively, in the overall population. Key findings included a statistically significant reduction in left ventricular wall thickness, left ventricular mass index, and improvements in diastolic function measures like the E/A and E/e’ ratios, among other parameters.
Moreover, the study also examined cardiac biomarkers, revealing a 32% reduction in NT-proBNP levels and a similar reduction in Troponin I levels in the overall population treated with vutrisiran, compared to placebo. In the monotherapy group, these reductions were even more pronounced. Among patients who were on tafamidis at baseline, vutrisiran led to an 18% reduction in NT-proBNP levels and a 10% reduction in Troponin I levels. These improvements were statistically significant and consistent across all subgroups.
Detailed results from the HELIOS-B study were also presented at the European Society of Cardiology annual congress and published in The New England Journal of Medicine on August 30, 2024. Alnylam plans to discuss these findings further at its upcoming TTR Investor Day on October 9, 2024, in New York City.
The HELIOS-B study (NCT: NCT04153149) was a Phase 3, randomized, double-blind, placebo-controlled multicenter global study designed to assess the efficacy and safety of vutrisiran in reducing all-cause mortality and recurrent cardiovascular events in patients with ATTR amyloidosis with cardiomyopathy. The study included 655 adult patients, randomized to receive either vutrisiran 25mg or placebo subcutaneously every three months over a double-blind period of up to 36 months. Following this period, eligible patients could continue to receive vutrisiran in an open-label extension phase.
Vutrisiran, marketed under the name AMVUTTRA®, is an RNAi therapeutic that targets the underlying cause of transthyretin (ATTR) amyloidosis by reducing mutant and wild-type transthyretin (TTR) protein levels. Administered quarterly via subcutaneous injection, AMVUTTRA is approved in more than 15 countries for treating the polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR-PN) in adults. The drug is also under development for treating ATTR amyloidosis with cardiomyopathy (ATTR-CM), covering both wild-type and hereditary forms of the disease.
Transthyretin amyloidosis (ATTR) is a rapidly progressing, debilitating, and often fatal disease caused by the accumulation of misfolded transthyretin proteins in various body parts, including the nerves and heart. ATTR exists in hereditary and wild-type forms, affecting a significant number of people globally. Vutrisiran offers a promising therapeutic avenue for this challenging condition.
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