HELIOS-A Phase 3 Results for Vutrisiran Presented at AAN

HELIOS-A Phase 3 Results for Vutrisiran Presented at AAN

Positive 9-month results from the HELIOS-A Phase 3 study of vutrisiran, an investigational RNAi therapeutic in development for the treatment of transthyretin-mediated (ATTR) amyloidosis, were presented in an oral session at the 2021 American Academy of Neurology (AAN) Virtual Annual Meeting held April 19, 2021 in a virtual event.

Read the press release

Adams, et al. – “HELIOS-A: 9-month Results from the Phase 3 Study of Vutrisiran in Patients with Hereditary Transthyretin-Mediated Amyloidosis with Polyneuropathy”

The presentation of HELIOS-A results in a conference call held April 19, 2021 includes additional HELIOS-A 9-month results from the Phase 3 study. Click here to access the conference call slides.

For additional presentations from Alnylam and collaborators presented at AAN, scroll to the bottom of this post.

At 9 months, vutrisiran—administered quarterly via subcutaneous injection—met the primary and all secondary endpoints, with statistically significant improvements in neuropathy, quality of life, and gait speed, and demonstrated an encouraging safety and tolerability profile, relative to the external placebo group.

Additional presentations from Alnylam and collaborators:

Polydefkis, et al. – “Global Open-label Extension: 24-month Data in Patients with hATTR Amyloidosis”

Peltier, et al. – “Impact of Patisiran on Activities of Daily Living and Functional Status in hATTR Amyloidosis”

Polydefkis, et al. – “Neurofilament Light Chain (NfL) as a Potential Biomarker of Treatment Response in Hereditary Transthyretin-Mediated Amyloidosis: Data from the Patisiran Global OLE Study”

Arum, et al. – “Open-label Study of Patisiran in Patients with hATTR Amyloidosis Post-Orthotopic Liver Transplant”

Merkel, et al. – “Evaluation of Patisiran With Concomitant or Prior Use of Transthyretin Stabilizers”

Monro, et al. – “Reduction in Pain During and Between Attacks in Patients with Acute Hepatic Porphyria Treated with Givosiran: A Post-Hoc Analysis of the Phase 3 ENVISION Study”

Erwin, et al. – “Disease Burden and Healthcare Utilization Among Patients with Acute Intermittent Porphyria Experiencing Chronic Neuropathy: Analyses from a National Healthcare Database”


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