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Vutrisiran for hATTR amyloidosis with polyneuropathy

Presented by
Dr David Adams, Centre Hospitalier Universitaire BicĂȘtre, France
AAN 2021
Phase 3, HELIOS-A
Vutrisiran offers an additional treatment option for patients with hereditary transthyretin-mediated (hATTR) amyloidosis, also known as ATTRv amyloidosis. This investigational RNA interference (RNAi) therapeutic agent significantly improved neuropathy impairment, quality of life, and gait speed compared with an external placebo group.

hATTR amyloidosis is a rare, underdiagnosed, rapidly progressive, and fatal disease caused by misfolded transthyretin that accumulates as amyloid fibrils in multiple tissues and organs. Vutrisiran is an RNAi therapeutic which targets liver-expressed variant and wild-type transthyretin. Dr David Adams (Centre Hospitalier Universitaire BicĂȘtre, France) presented the 9-month efficacy and safety from the phase 3, open-label HELIOS-A study (NCT03759379) of vutrisiran [1]. The study enrolled 164 patients, who were randomised 3:1 to vutrisiran or to patisiran, a reference comparator RNAi therapeutic approved for hATTR amyloidosis with polyneuropathy. The placebo group from the APOLLO trial (n=77; NCT01960348) served as external control.

Vutrisiran met the primary endpoint, which was change in neuropathy impairment as measured by Modified Neuropathy Impairment Score +7 (mNIS+7) versus placebo after 9 months. Mean change was -2.24 and +16.76 in the treatment and placebo group, respectively (P<0.00001). Both secondary endpoints were also met after 9 months; there were statistically significant improvements versus placebo in quality of life (Norfolk QoL-DN): -3.3 and 12.9, respectively (P<0.00001), as well as in gait speed (10-Meter Walk Test [10-MWT]): -0.001 and -0.133 (P=00003). The effects on neuropathy and QoL were seen across all patient subgroups. Vutrisiran also led to rapid and sustained reduction in serum transthyretin levels. It had an acceptable safety profile and favourable benefit-risk profile.

  1. Adams D, et al. HELIOS-A: 9-month Results from the Phase 3 Study of Vutrisiran in Patients with Hereditary Transthyretin-Mediated Amyloidosis with Polyneuropathy. S13.001, AAN 2021 Virtual Congress, 17-22 April.

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