Significantly improve multiple health and functional indicators, subcutaneous injection of RNAi therapy announced the latest phase 3 trial results

On September 7, 2021, Alnylam Pharmaceuticals announced the latest results obtained in a phase 3 clinical trial of its RNAi drug vutrisiran in the treatment of patients with transthyretin-mediated (ATTR) amyloidosis

Hereditary transthyretin-mediated (hATTR) amyloidosis is a hereditary and progressive debilitating and fatal disease caused by mutations in the TTR gene

Vutrisiran is a subcutaneously administered RNAi therapeutic drug that is being developed to treat ATTR amyloidosis, including hATTR and wild-type ATTR (wtATTR) amyloidosis

▲Introduction of Onpattro and vutrisiran (picture source: Alnylam official website)

The data obtained this time further supports the primary and secondary endpoint results previously reported in the HELIOS-A trial, which was carried out in 164 patients with hATTR amyloidosis with polyneuropathy

The results of data analysis showed that at the 9th month, the trial reached the primary endpoint and all secondary endpoints

In terms of safety, compared with placebo, vutrisiran showed encouraging safety and tolerability characteristics

Reference materials:

[1] Alnylam Presents Additional 9-Month Data from HELIOS-A Phase 3 Study of Vutrisiran at the European ATTR (EU-ATTR) Amyloidosis Meeting.