Alnylam Pharmaceuticals saw shares of its stock jump by 32% after sharing positive topline results for a late-stage study of its RNA interference (RNAi) therapeutic, vutrisiran, for the treatment of ATTR amyloidosis with cardiomyopathy (ATTR-CM).

As per the 24 June announcement, the Phase III HELIOS-B study (NCT04153149) met its primary endpoint of demonstrating statistically significant reductions in the composite of all-cause mortality and recurrent cardiovascular events across both monotherapy and overall study populations. The monotherapy group reported a 33% reduction in this composite endpoint (hazard ratio (HR) = 0.672) while the overall population reported a reduction of 28% (HR = 0.718).

The biopharma also shared that vutrisiran demonstrated statistically significant improvements across all secondary endpoints, which included disease progression measures such as change from baseline in the 6-minute walk test (6MWT), Kansas City Cardiomyopathy Questionnaire (KCCQ), and New York Heart Association (NYHA) Class all at 30 months. All-cause mortality measured for up to 42 months decreased by 35% and 36% in the monotherapy and overall populations, respectively.

HELIOS-B is a multi-centre, double-blind, parallel assignment study that randomised 655 adult patients into experimental treatment and placebo groups at a 1:1 ratio. Patients in the experimental arm were administered 25mg subcutaneous vutrisiran at a three-month interval during the blinded period of the protocol that lasted for 36 months. Randomisation was stratified based on the patients’ use of Pfizer’s Vyndaqel/Vyndamax (tafamidis)—an approved ATTR treatment— at baseline, the difference in ATTR disease type, and NYHA Class I or II and age.

Vutrisiran was also observed to have consistent effects on these key patient subgroups such as those treated with vutrisiran who were also on baseline Vyndaqel or Vyndamax. In the press release, CEO Yvonne Greenstreet, MBChB added that Alnylam’s treatment has the potential to become the new standard of care for this disease. Vyndamax or Vyndaqel, is currently the most common treatment option for patients with ATTR-CM. Most recently, the National Health Service (NHS) introduced Pfizer’s medication for the treatment of ATTR-CM in the UK.

According to GlobalData’s consensus forecasts, Vyndamax/Vyndaqel is expected to generate total sales of $5.6bn in 2026 while vutrisiran is anticipated to generate global sales of $3.6bn in 2030. GlobalData is the parent company of Pharmaceutical Technology.

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As study results demonstrate an improvement of cardiovascular outcomes across the evaluated patient groups treated with vutrisiran, Alnylam is “moving with urgency” to file the data with regulators to bring the treatment to the market, said Alnylam CMO Dr. Pushkal Garg in the announcement. Submissions to global regulatory agencies will be initiated in the course of 2024, with a New Drug Application (NDA) filed with a Priority Review Voucher to the U.S. Food and Drug Administration (FSA) expected later in the year.