Exciting Advances in ALS

Amyotrophic lateral sclerosis (ALS) is a relentlessly progressive and fatal neurodegenerative disorder, affecting approximately 3000 Canadians. Despite a relatively stagnant treatment landscape for the past few decades, exciting advances are underway.

Radicava (edaravone), which received Health Canada approval in 2018, was only the second ALS therapy approved in Canada in nearly 20 years. Last week, Health Canada granted approval with conditions to Albrioza (sodium phenylbutyrate and ursodoxicoltaurine), an oral fixed-dose combination therapy that may reduce neuronal cell death as a standalone therapy or when added to existing treatments. The approval was based on the Phase II CENTAUR trial that enrolled 137 patients with ALS and demonstrated a statistically significant and clinically meaningful benefit in functional outcomes with Albrioza versus placebo. The Phase III PHOENIX trial of Albrioza is ongoing with topline results expected in 2024.

Additionally, the HEALEY ALS Platform Trial is testing multiple investigational products in parallel to reduce research costs and trial time, while increasing patient participation. Patients are randomized 3:1 into a treatment arm (zilucoplan, verdiperstat, CNM-Au8, pridopidine or trehalose) or placebo. The estimated study completion date is December 2023.

The recent progress in the ALS treatment landscape alongside the robust and growing ALS drug pipeline are promising advances for patients with ALS.

-Karamjeet