Amylyx Pharmaceuticals has announced that it will be withdrawing its ALS drug, Relyvrio, from the U.S. and Canada after the treatment failed in a key late-stage trial. This decision has led to a 70% reduction in the company’s workforce, with 384 full-time employees at the end of 2023. Relyvrio was approved in 2022 after lobbying by patient groups, as there were limited options to treat the fatal disease. ALS causes progressive paralysis and affects around 60,000 people in the U.S. and Europe. The approval of Relyvrio came after a rare turnaround by the FDA’s advisers, who initially rejected the drug due to lack of persuasive data. The approval was based on mid-stage trial data showing that the treatment slowed disease progression and extended life expectancy.
Despite initial approval and positive mid-stage trial data, a larger late-stage study failed to confirm the reported benefits of Relyvrio, with no significant difference seen in patients treated with the drug versus those who received a placebo. The drug, with a list price of $158,000 per year in the U.S., generated sales of approximately $381 million in 2023. Amylyx Pharmaceuticals expects to incur charges of around $19 million as part of its restructuring, which is expected to be completed by the end of the third quarter. Following the announcement of the drug withdrawal, the company’s shares closed about 83% lower, with over $1 billion wiped out from its market value on March 8. Despite the setback, Amylyx Pharmaceuticals plans to continue trials of its lead experimental drug, AMX0035, in conditions such as Wolfram syndrome and progressive supranuclear palsy, as well as focus on AMX0114 in ALS.
The withdrawal of Relyvrio and the subsequent restructuring of Amylyx Pharmaceuticals reflect the challenges faced by the company in developing effective treatments for ALS. ALS is a devastating disease with limited treatment options, making the failure of Relyvrio even more disappointing. The initial approval of the drug based on mid-stage trial data highlights the complexities of drug development and the need for larger and more comprehensive studies to confirm efficacy. The significant impact on the company’s market value and workforce reduction underscore the financial implications of drug development failures and the need for companies to carefully manage resources and investments in research and development.
The decision to focus on advancing trials of AMX0035 and AMX0114 in other conditions highlights Amylyx Pharmaceuticals’ commitment to developing innovative treatments for neurological disorders. While the failure of Relyvrio is a setback, the company remains dedicated to addressing unmet medical needs in conditions such as Wolfram syndrome and progressive supranuclear palsy. The company’s efforts to pivot towards other promising drug candidates demonstrate its resilience and determination to make a meaningful impact in the field of neurology. As research in ALS and other neurological disorders continues to evolve, Amylyx Pharmaceuticals will play a crucial role in advancing the development of new therapies and treatments to improve patient outcomes and quality of life.
In conclusion, the withdrawal of Relyvrio marks a significant setback for Amylyx Pharmaceuticals, but the company remains focused on advancing its pipeline of experimental drugs in other neurological conditions. The challenges faced in developing effective treatments for ALS highlight the complexity of drug development and the importance of conducting robust clinical trials to confirm efficacy. Despite the financial and workforce implications of the drug withdrawal, Amylyx Pharmaceuticals remains committed to its mission of developing innovative therapies for neurological disorders. As the company continues to pursue new drug candidates and expand its research efforts, it will contribute to the advancement of treatments for ALS and other devastating conditions, ultimately improving the lives of patients and their families.
