Alnylam Pharmaceuticals shared glowing data from its HELIOS-B phase 3 study of vutrisiran, an RNAi therapeutic being investigated in ATTR amyloidosis with cardiomyopathy.
The study involved 655 participants and hit its primary goal, showing a notable reduction in all-cause mortality and recurrent cardiovascular events. Specifically, the overall group saw a 28% reduction, while the monotherapy group (those not on the standard-of-care treatment, Pfizer's tafamidis) saw a 33% reduction.
Besides the primary goal, vutrisiran also made significant improvements in all secondary endpoints. These included key markers of disease progression like the 6-minute walk test, Kansas City Cardiomyopathy Questionnaire, and New York Heart Association Class at month 30. It also reduced all-cause mortality by 36% in the overall group and 35% in the monotherapy group in a pre-specified secondary analysis.
ATTR amyloidosis occurs when abnormal transthyretin proteins accumulate in tissues, leading to organ dysfunction. It can be hereditary or acquired, often affecting the heart and nerves.
In June 2022, the drug, branded Amvutta, was approved for the treatment of polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis. Now, armed with positive results trial, Alnylam plans to file a supplemental NDA for the new indication, using a Priority Review Voucher.
Vutrisiran is an RNA interference therapeutic. RNAi, a natural process within cells that silences specific genes, is leveraged by drugs like vutrisiran to target and reduce the production of transthyretin (TTR) protein, which misfolds and accumulates as amyloid deposits in various tissues, including the heart, nerves and gastrointestinal tract.
If approved for ATTR amyloidosis, the drug will face off with Pfizer’s tafamidis. Branded as Vyndaqel and Vyndamax, the drug was first approved in 2019.
