RARE Daily

Alnylam Holds Off on Phase 3 Study of Stargardt Disease Therapy as It Weighs Impact of Legislation

October 28, 2022

Alnylam said that it is considering options for the best path toward advancing an RNAi therapeutic for the treatment of Stargardt Disease. At this time, it will not initiate a phase 3 study of its approved product Amvuttra as a treatment for the rare eye disease Stargardt Disease.

The company had previously announced it would begin a phase 3 study in late 2022, but on a call to discuss third quarter financial results said it would delay the start of the study as it continues to evaluate the impact of the Inflation Reduction Act.

The company also said it does not plan to conduct the optional interim analysis for the HELIOS-B phase 3 study in patients with ATTR amyloidosis with cardiomyopathy. The study remains on track for topline results in early 2024.

Amvuttra is an RNAi therapeutic approved in the United States for the treatment of the polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults. It is a double‑stranded small interfering RNA that targets mutant and wild‑type transthyretin messenger RNA. Using Alnylam’s Enhanced Stabilization Chemistry (ESC)-GalNAc-conjugate delivery platform, Amvuttra is designed for increased potency and high metabolic stability to allow for subcutaneous injection once every three months.

Results from the pivotal HELIOS-A phase 3 study demonstrate Amvuttra rapidly reduces serum TTR levels, has the potential to reverse neuropathy impairment relative to baseline, and improves other key measures of disease burden relative to external placebo in patients with the polyneuropathy of hATTR amyloidosis.

Author: Rare Daily Staff

Stay Connected

Sign up for updates straight to your inbox.