Shidlovski/iStock via Getty Images
- Alnylam (NASDAQ:ALNY) is trading flat in the pre-market after announcing 18-month data for its Phase 3 study of vutrisiran in transthyretin-mediated (ATTR) amyloidosis. The company said that the trial met all secondary endpoints in patients with hATTR amyloidosis with polyneuropathy.
- Alnylam (ALNY) previously disclosed that the HELIOS-A study met its primary and secondary endpoints at nine months.
- According to 18-month data, vutrisiran demonstrated a statistically significant improvement in clinical endpoints such as neuropathy impairment, quality of life (QoL), gait speed, nutritional status, and overall disability vs. placebo. There was non-inferiority in serum TTR reduction compared to an in-study patisiran (ONPATTRO) arm.
- At 18 months, there were a total of three study discontinuations, including two deaths, which were previously disclosed along with two serious adverse events (SAEs) related to the experimental therapy.
- “These HELIOS-A results show that the improvement in neuropathy impairment and quality of life observed with vutrisiran at 9 months is maintained through Month 18, with the treatment effect increasing over time and an encouraging safety profile,” remarked Rena N. Denoncourt, Vice President, TTR Franchise Lead.
- With an action date of April 14, vutrisiran is currently under the FDA review for polyneuropathy of hereditary ATTR amyloidosis.