Vutrisiran reduces mortality, CV events in ATTR amyloidosis with cardiomyopathy
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Key takeaways:
- Alnylam announced positive top-line results of its phase 3 trial of vutrisiran for the treatment of ATTR amyloidosis with cardiomyopathy.
- The full results are submitted for presentation at the 2024 ESC Congress.
Alnylam Pharmaceuticals announced the positive top-line results of the phase 3 HELIOS-B trial of its investigational RNA interfering therapy vutrisiran for the treatment of transthyretin amyloidosis with cardiomyopathy.
The HELIOS-B trial was a randomized, double-blind, placebo-controlled multicenter global study designed to assess the efficacy and safety of vutrisiran for the reduction of all-cause mortality and CV events in patients with ATTR amyloidosis with cardiomyopathy, according to a press release from the company.
Researchers randomly assigned 655 adult patients with ATTR amyloidosis with cardiomyopathy to either subcutaneous vutrisiran 25 mg or placebo every 3 months for up to 36 months. A total of 395 participants were not receiving tafamidis (Vyndamax, Pfizer) at baseline. After a double-blind period, eligible patients were able to receive vutrisiran in an open-label extension period.
The HELIOS-B trial met its primary endpoint with a significant reduction in all-cause mortality and recurrent CV events in the double-blind period in the overall population (HR = 0.718; P = .0118) and among participants not receiving tafamidis at baseline (HR = 0.672; P = .0162). The company also reported improvement across all secondary outcomes — 6-minute walk test, Kansas City Cardiomyopathy Questionnaire and NYHA class — in both patient populations (P for all < .025).
Moreover, the trial demonstrated encouraging safety and tolerability of vutrisiran, with similar rates of adverse events leading to study drug discontinuation between the vutrisiran and placebo groups, the company stated in the release.
“I’m thrilled by these overwhelmingly positive data from the HELIOS-B study, which suggest that vutrisiran has the potential to address the needs of patients with ATTR amyloidosis with cardiomyopathy, a steadily progressive, debilitating, and ultimately fatal disease,” Pushkal Garg, MD, chief medical officer of Alnylam, said in the release. “The results showed that vutrisiran improved cardiovascular outcomes, including survival, function and quality of life in all patient groups with ATTR cardiomyopathy. We are moving with urgency to file these compelling data with regulators to bring this medicine to patients around the world.”
The results from the HELIOS-B trial have been submitted as a late-breaking abstract for presentation at European Society of Cardiology Congress, according to the release.
The company reports plans to proceed filing a supplemental New Drug Application with the FDA.
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