Alnylam Pharmaceuticals has shared detailed results from its phase 3 study of vutrisiran, an investigational RNAi therapeutic in development for the treatment of ATTR amyloidosis with cardiomyopathy (ATTR-CM).
The data were presented at a session at the European Society of Cardiology Congress 2024 and results were also published in the New England Journal of Medicine.
Alnylam had shared the positive top-line results back in June, revealing that the HELIOS-B study met all 10 of its primary and secondary endpoints, across both the overall and monotherapy populations, with statistical significance.
In the overall population, vutrisiran reduced the risk of all-cause mortality and recurrent cardiovascular events by 28%. Mortality in this population was significantly reduced by 31% during the double-blind period and by 36% up to 42 months. In the monotherapy population, vutrisiran significantly reduced the risk of all-cause mortality and recurrent cardiovascular events by 33% and significantly reduced the risk of mortality by 35% up to 42 months.
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.
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.
In June 2022, the drug, branded Amvutta, was approved for the treatment of polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis. Alnylam plans to file a supplemental NDA for the new indication, ATTR-CM, using a Priority Review Voucher.
Current treatment options for ATTR-CM include Pfizer's tafamidis, branded as Vyndaqel and Vyndamax, which works by stabilizing production of the protein transthyretin.
While Alnylam believes the recent data suggests that vutrisiran has the potential to become a new standard of care treatment for ATTR-CM, investors weren't on board, with the company's shares falling 7% with the most recent news.
