Alnylam shares positive phase 3 results for vutrisiran in ATTR cardiomyopathy

Alnylam Pharmaceuticals has announced promising findings from its phase 3 HELIOS-B trial, which evaluated the investigational RNAi therapeutic vutrisiran for treating transthyretin amyloidosis (ATTR) with cardiomyopathy. The study involved 655 adult participants who were randomly assigned to receive either vutrisiran or a placebo every three months for up to three years. Following this period, all eligible patients were transitioned to an open-label extension phase where they continued receiving vutrisiran.

At the European Society of Cardiology Congress, detailed results were presented, revealing that vutrisiran significantly lowered the risk of all-cause mortality and recurrent cardiovascular events by 28% in the overall study population. This included both patients who took vutrisiran alone and those who combined it with standard treatments like SGLT2 inhibitors and Pfizer's Vyndaqel/Vyndamax (tafamidis). The analysis showed even more pronounced benefits in the subgroup treated solely with vutrisiran. In this group, the therapy reduced the risk of all-cause mortality and recurrent cardiovascular events by 33%, and the risk of death by 35% at the 42-month mark. In the overall patient population, mortality decreased by 31% during the double-blind period and by 36% at month 42.

Vutrisiran also showed positive outcomes across various clinical measures of disease progression. One such measure was the six-minute walk test, and consistent benefits were observed across different patient subgroups. ATTR amyloidosis is a serious, often underdiagnosed, and rapidly progressing disorder. It arises from the misfolding of transthyretin (TTR) proteins, which deposit as amyloid in multiple organs, including the heart and nerves. Patients can present symptoms of polyneuropathy, cardiomyopathy, or both.

Designed to target and reduce TTR levels, vutrisiran leverages the body's natural processes to quickly lower these toxic proteins. It is already approved under the brand name Amvuttra for treating polyneuropathy caused by ATTR in adults. Pushkal Garg, the Chief Medical Officer of Alnylam, highlighted the significance of these findings, stating that the rapid TTR knockdown observed with vutrisiran led to improvements in survival rates, reductions in cardiovascular hospitalizations, and slower disease progression compared to the placebo. The benefits were consistent regardless of background stabilizer use, providing hope for broader applicability.

While the results have not yet undergone regulatory review, Garg emphasized that the data suggest vutrisiran could emerge as a new standard of care for treating ATTR with cardiomyopathy, a condition that remains progressive and ultimately fatal with limited treatment options. Alnylam Pharmaceuticals is optimistic about the future and is gearing up for global regulatory submissions for vutrisiran later this year, aiming to offer a new lifeline for patients battling this challenging disease.

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