Amylyx Begins Process of Discontinuing Relyvrio/Albrioza for Treatment of ALS

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Amylyx Pharmaceuticals announced that it has made the decision to discontinue Relyvrio/Albrioza (AMX0035), their drug for amyotrophic lateral sclerosis (ALS), in the United States and Canada, stemming from the results of the Phase III PHOENIX trial. Despite the drug no longer being available, patients who are currently taking it will still have the ability to receive treatments free of charge. After meeting with regulatory bodies and stakeholders, the company said that it intends to continue ALS research and data collection, with key findings to be shared at the upcoming American Academy of Neurology (AAN) annual meeting in Denver later this month.¹

“While this is a difficult moment for the ALS community, we reached this path forward in partnership with the stakeholders who will be impacted and in line with our steadfast commitment to people living with ALS and other neurodegenerative diseases. The decision to remove Relyvrio/Albrioza from the market and provide therapy free of charge for those who wish to continue was informed by the PHOENIX trial results, engagement with regulatory authorities, and discussions with the ALS community. Thank you to each and every person who shared feedback with us and continues to support our commitment to the ALS community,” said Joshua Cohen and Justin Klee, co-CEOs of Amylyx, in a press release.

Amylyx also plans on continuing research on AMX0035 for the treatment of Wolfram syndrome and progressive supranuclear palsy. As a result of these changes, Amylyx stated that it plans on undergoing corporate restructuring, including a significant workforce reduction, to extend their financial runway into 2026. The company said that this will lead to better support for ongoing clinical trials and research commitments.¹

“Our pipeline is supported by compelling clinical and preclinical science demonstrating the potential of AMX0035 and AMX0114 in neurodegenerative diseases. AMX0035 was designed to slow or mitigate neurodegeneration by targeting endoplasmic reticulum stress and mitochondrial dysfunction, two connected central pathways that lead to cell death and neurodegeneration. We are investigating AMX0035 in diseases where these two pathways are implicated, which includes Wolfram syndrome and progressive supranuclear palsy,” said Camille L. Bedrosian, MD, chief medical officer, Amylyx, in the press release. “We look forward to presenting interim data from our Phase II HELIOS study of AMX0035 in Wolfram syndrome, a rare, genetic, fatal neurodegenerative disease with no FDA-approved treatment options, later this month. In addition, our Phase III ORION study remains ongoing to evaluate AMX0035 for the treatment of progressive supranuclear palsy, a rare neurodegenerative disorder characterized as a tauopathy. We continue to plan for an interim analysis to evaluate the data from ORION that is now expected in mid-2025.”

Amylyx released the results from the PHOENIX trial last month. As previously alluded to, the study failed to meet its primary endpoint of a statistically significant reduction in Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R) total score at week 48 compared with placebo. The study, which included 664 adults living with ALS, found no major differences between Relyvrio and the placebo.^2,3

In 2022, Public Citizen’s Health Research Group asked the FDA not to approve AMX0035 because of inconclusive data from a small clinical trial and an open-label extension study.

“As the FDA should have never approved AMX0035, the company’s announcement that the drug will be withdrawn from the market is welcome news,” said Robert Steinbrook, director, Health Research Group, Public Citizen, in a press release. “Although amyotrophic lateral sclerosis is a devastating disease and effective treatments are urgently needed, the FDA’s approval of AMX0035 allowed the company to make tens of millions of dollars of profits from selling a worthless drug for which there was never evidence of efficacy. Patients were duped, individuals and payors needlessly spent hundreds of millions of dollars, and a company was unjustly enriched. All of these consequences were preventable, and the FDA must take steps to ensure they never happen again. The FDA should never approve a drug without clear evidence of efficacy.”⁴

References

1. Amylyx Pharmaceuticals Announces Formal Intention to Remove RELYVRIO®/ALBRIOZA™ from the Market; Provides Updates on Access to Therapy, Pipeline, Corporate Restructuring, and Strategy. Amylyx. April 4, 2024. Accessed April 5, 2024. https://www.amylyx.com/news/amylyx-pharmaceuticals-announces-formal-intention-to-remove-relyvrior/albriozatm-from-the-market-provides-updates-on-access-to-therapy-pipeline-corporate-restructuring-and-strategy

2. Relyvrio Falls Short of Primary Endpoint in Phase III PHOENIX Trial in Patients with ALS. Pharmaceutical Executive. March 8, 2024. Accessed April 5, 2024. https://www.pharmexec.com/view/relyvrio-falls-short-of-primary-endpoint-in-phase-iii-phoenix-trial-in-patients-with-als

3. Amylyx Pharmaceuticals Announces Topline Results From Global Phase 3 PHOENIX Trial of AMX0035 in ALS. Amylyx. March 8, 2024. Accessed April 5, 2024. https://www.amylyx.com/news/amylyx-pharmaceuticals-announces-topline-results-from-global-phase-3-phoenix-trial-of-amx0035-in-als

4. Manufacturer Withdraws Amyotrophic Lateral Sclerosis Drug AMX0035 (Relyvrio) From Market. Public Citizen. April 4, 2024. Accessed April 5, 2024. https://www.citizen.org/news/fda-withdraws-amyotrophic-lateral-sclerosis-drug-amx0035-relyvrio-from-market/