Amylyx Pulls ALS Treatment Off Market With a Few Thousand Still on Treatment as of Late '23

Amylyx Pharmaceuticals recently announced the process with the FDA and Health Canada to voluntarily discontinue the marketing for Relyvrio for amyotrophic lateral sclerosis (ALS) based on phase 3 PHOENIX trial results.

Approved in late September 2022, Relyvrio (sodium phenylbutyrate/taurursodiol) will no longer be available for new patients as of yesterday, according to an Amylyx news release.

Patients currently on therapy can transition to a free drug program if they choose, upon consultation with their physician.

According to a spokesperson with Amylyx, "the number of people living with ALS taking Relyvrio remained stable in the fourth quarter of 2023 compared to the previous quarter."

The spokesperson said that by the end of the third quarter in 2023, Amylyx reported roughly 3,900 people living with ALS on the therapy in the U.S.

They currently don't have the numbers for Canada patients.

The decision to discontinue Relyvrio and offer therapy free of charge for continuing patients was influenced by the trial results, regulatory engagement and discussions with the ALS community, Joshua Cohen and Justin Klee, Co-CEOs of Amylyx, said in the news release.

The PHOENIX trial, a global, 48-week, randomized, placebo-controlled Phase 3 clinical trial, did not meet its primary endpoint of statistical significance (p=0.667) as measured by the change from baseline in the Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R) total score at Week 48, according to an announcement made in early March.

Secondary endpoints also did not show statistical significance.

"While the Phase 3 PHOENIX trial did not meet its primary endpoint in amyotrophic lateral sclerosis, the trial did reinforce in a larger and broader population of people living with ALS that AMX0035 is generally safe and well-tolerated," an Amylyx spokesperson said

Amylyx plans to present the PHOENIX trial data at an the at the American Academy of Neurology (AAN) Annual Meeting in Denver and online on April 16, 2024.

The company will also publish the results in a medical journal later this year, the release said.

The organization plans to continue evaluating and sharing discoveries from the trial and collect available survival data as recommended by ALS specialists.

As part of their commitment to neurodegenerative disease research, Amylyx shared they are advancing two key programs: investigating Relyvrio AMX0035 in Wolfram syndrome and progressive supranuclear palsy (PSP), and AMX0114, another ALS drug candidate.

AMX0035 targets endoplasmic reticulum stress and mitochondrial dysfunction, pathways associated with neurodegeneration, while AMX0114 aims to inhibit calpain-2, an enzyme linked to neurofilament biology.

Amylyx also plans to present interim data from the Phase 2 HELIOS study of AMX0035 in Wolfram syndrome later in April and continue the Phase 3 ORION study for PSP treatment, with an interim analysis expected in mid-2025, shared in the release.

Plans to initiate a clinical trial for AMX0114 are set to begin in the second half of this year, according to Amylyx.

This story first appeared on Managed Healthcare Executive.