Alnylam reports positive late-stage heart drug data

Alnylam Pharmaceuticals has recently announced promising results from its HELIOS-B phase 3 trial of vutrisiran, an RNA interference (RNAi) therapeutic, which is being explored for its efficacy in treating ATTR amyloidosis with cardiomyopathy. The trial, which included 655 participants, successfully reached its primary endpoint, demonstrating a significant decline in both all-cause mortality and recurrent cardiovascular incidents. The trial's findings revealed an overall reduction of 28% in these adverse events, while the group receiving only the monotherapy (as opposed to also receiving Pfizer's tafamidis) experienced a 33% reduction.

In addition to achieving its primary goal, vutrisiran showed substantial improvements in all secondary endpoints. These secondary outcomes involved crucial indicators of disease advancement, such as the 6-minute walk test, the Kansas City Cardiomyopathy Questionnaire, and the New York Heart Association Class at the 30-month mark. Furthermore, a pre-specified secondary analysis indicated a reduction in all-cause mortality by 36% in the overall group and 35% in the monotherapy group.

ATTR amyloidosis is characterized by the accumulation of abnormal transthyretin proteins in tissues, leading to organ dysfunction. This condition can be either hereditary or acquired, and it frequently impacts the heart and nervous system.

In June 2022, the drug vutrisiran, which is marketed under the brand name Amvutta, received approval for the treatment of polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis. With the robust data from the HELIOS-B trial, Alnylam Pharmaceuticals now intends to submit a supplemental New Drug Application (NDA) for this new indication, leveraging a Priority Review Voucher to expedite the process.

Vutrisiran functions as an RNA interference therapeutic, utilizing a natural cellular process to silence specific genes. By targeting and reducing the production of the transthyretin (TTR) protein, vutrisiran aims to prevent the misfolding and accumulation of these proteins as amyloid deposits in various tissues, including the heart, nerves, and gastrointestinal tract.

Should vutrisiran receive approval for the treatment of ATTR amyloidosis, it will enter the market in competition with Pfizer’s tafamidis, which is sold under the brand names Vyndaqel and Vyndamax. Tafamidis was first approved in 2019 and has since been a leading treatment for this condition.