Positive Phase III study of vutrisiran in patients with hATTR amyloidosis

22 January 2022

US RNAi therapeutics company Alnylam Pharmaceuticals’ (Nasdaq: ALNY) on Friday revealed new data supporting the case for a new drug it is been developing for transthyretin amyloidosis.

The HELIOS-A Phase III study of Alnylam’s vutrisiran, an investigational RNAi therapeutic in development for the treatment of transthyretin-mediated (ATTR) amyloidosis, met all secondary endpoints measured at 18 months in patients with hATTR amyloidosis with polyneuropathy, including statistically-significant improvements in neuropathy impairment, quality of life (QoL), gait speed, nutritional status and overall disability, relative to placebo, and non-inferiority of serum TTR reduction relative to the within-study patisiran arm. .

However, investors were not impressed, as the firm’s share closed down nearly 8% at $134.80 on Friday. The results were presented in an oral session at the Société Francophone du Nerf Périphérique (SFNP) Annual Meeting.

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