Alnylam rises on positive top-line results for vutrisiran in hATTR amyloidosis

US RNAi therapeutics company Alnylam Pharmaceuticals (Nasdaq: ALNY) saw its shares close up nearly 10% at $139.40 on Thursday, after it announced that the HELIOS-A Phase III study of vutrisiran, an investigational RNAi therapeutic in development for the treatment of transthyretin-mediated (ATTR) amyloidosis, met its primary and both secondary endpoints at nine months in patients with hATTR amyloidosis with polyneuropathy.

The primary endpoint was the change from baseline in the modified Neuropathy Impairment Score (mNIS+7) at nine months as compared to historical placebo data from the APOLLO Phase III study of patisiran. The two secondary endpoints were changes in quality of life assessed by the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy (Norfolk QoL-DN) and gait speed assessed by the timed 10-meter walk test (10-MWT) compared to historical placebo.

Vutrisiran met the primary endpoint (p less than 0.001) and achieved statistically-significant results (p less than 0.001) for each of the Norfolk QoL-DN and 10-MWT secondary endpoints. In addition, vutrisiran treatment showed improvement compared to placebo on the exploratory cardiac biomarker endpoint, NT-proBNP (nominal p less than 0.05). Vutrisiran also demonstrated an encouraging safety and tolerability profile.