Alnylam Reports Positive Results from Phase 3 Study

RNAi therapeutics company Alnylam has announced positive topline results from its Phase 3 study of vutrisiran, an investigational RNAi therapeutic in development for the treatment of ATTR amyloidosis with cardiomyopathy (ATTR-CM), a rare heart condition that is both progressive and fatal.

Transthyretin amyloidosis (ATTR) is an underdiagnosed, rapidly progressive, debilitating and fatal disease caused by misfolded transthyretin (TTR) proteins, which accumulate as amyloid deposits in various parts of the body, including the nerves, heart and gastrointestinal tract. There are two different forms of ATTR – hereditary ATTR which is caused by a TTR gene variant and affects approximately 50,000 people worldwide, and wild-type ATTR, which occurs without a TTR gene variant and impacts an estimated 200,000 – 300,000 people worldwide.

“I’m thrilled by these overwhelmingly positive data from the HELIOS-B study, which suggest that vutrisiran has the potential to address the needs of patients with ATTR amyloidosis with cardiomyopathy, a steadily progressive, debilitating, and ultimately fatal disease,” said Pushkal Garg, Chief Medical Officer of Alnylam. “The results showed that vutrisiran improved cardiovascular outcomes, including survival, function and quality of life in all patient groups with ATTR cardiomyopathy. We are moving with urgency to file these compelling data with regulators to bring this medicine to patients around the world.”

RNAi (RNA interference) is a natural cellular process of gene silencing that represents one of the most promising and rapidly advancing frontiers in biology and drug development today. RNAi therapeutics harness this natural biological process. Small interfering RNA (siRNA), the molecules that mediate RNAi and comprise Alnylam’s RNAi therapeutic platform, function upstream of today’s medicines by potently silencing messenger RNA (mRNA) – the genetic precursors that encode for disease-causing or disease pathway proteins – thus preventing them from being made. (Source: Alnylam Website, 24 June, 2024)