Alnylam Shares HELIOS-B Phase 3 Vutrisiran Results for ATTR Amyloidosis at ESC Congress

Alnylam Pharmaceuticals, Inc., a leader in RNAi therapeutics, has unveiled detailed results from the HELIOS-B Phase 3 study of vutrisiran, an investigational RNAi therapeutic aimed at treating ATTR amyloidosis with cardiomyopathy (ATTR-CM). The findings were presented at the European Society of Cardiology (ESC) Congress 2024, occurring from August 30 to September 2 in London, UK, and were simultaneously published in The New England Journal of Medicine.

The HELIOS-B study evaluated the efficacy and safety of vutrisiran and achieved all ten of its primary and secondary endpoints with statistical significance. The patient cohort largely consisted of individuals diagnosed with wild-type ATTR-CM, classified as New York Heart Association (NYHA) Class I or II, and were concurrently receiving standard treatments like tafamidis and SGLT2 inhibitors.

Vutrisiran treatment resulted in significant clinical benefits. In the overall population, there was a 28% reduction in the risk of all-cause mortality and recurrent cardiovascular events. Mortality alone was reduced by 31% during the 33-36 month double-blind period and by 36% up to 42 months. In the monotherapy group, a 33% reduction in the composite primary endpoint and a 35% decrease in all-cause mortality up to month 42 were observed.

Beyond mortality and cardiovascular events, vutrisiran showed positive effects on several clinical measures of disease progression, such as the 6-Minute Walk Test, Kansas City Cardiovascular Questionnaire, and cardiac biomarker NT-proBNP. These benefits were consistent across various patient subgroups, including those already receiving tafamidis. Notably, vutrisiran appeared more effective in patients with earlier stages of the disease, indicated by younger age and lower baseline NT-proBNP levels.

Safety and tolerability profiles for vutrisiran remained favorable and consistent with previous studies. Adverse events were comparable between the vutrisiran and placebo groups, with no significant increase in any specific adverse event in the vutrisiran arm compared to placebo.

Dr. Marianna Fontana, a cardiology professor at University College London and an investigator in the HELIOS-B study, emphasized the significance of the findings, noting the potential for vutrisiran to dramatically improve treatment outcomes for ATTR-CM patients, particularly those in the early stages of the disease. Early intervention with vutrisiran could preserve functional capacity and enhance quality of life for patients.

Pushkal Garg, M.D., Chief Medical Officer of Alnylam, expressed pride in presenting the HELIOS-B data, highlighting the demonstrated improvements in survival and reduced cardiovascular hospitalizations associated with vutrisiran. While the results await regulatory review, there is optimism about vutrisiran becoming a new standard of care for ATTR-CM, an otherwise progressive and fatal condition with limited treatment options. Alnylam is preparing to file for regulatory approval globally, including a supplemental New Drug Application with the U.S. FDA later this year.

The HELIOS-B study was a Phase 3, randomized, double-blind, placebo-controlled trial involving 655 adult patients with either hereditary or wild-type ATTR amyloidosis with cardiomyopathy. Participants received either vutrisiran 25mg or a placebo subcutaneously every three months for up to 36 months, followed by an open-label extension period.

In summary, the HELIOS-B Phase 3 study positions vutrisiran as a promising therapeutic option for ATTR-CM, potentially transforming the treatment landscape for this severe and life-threatening condition.

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