Amylyx Reports Phase 3 PHOENIX Trial Results for AMX0035 in ALS Treatment

Amylyx Pharmaceuticals recently reported the outcomes of the PHOENIX study, a Phase 3 clinical trial investigating AMX0035 for the treatment of amyotrophic lateral sclerosis (ALS). The study, involving 664 participants, did not achieve its primary or secondary goals, with no significant difference in the ALS Functional Rating Scale (ALSFRS-R) total score between the treatment and placebo groups after 48 weeks.

Despite the disappointing results, the study data indicated that AMX0035 maintained a favorable safety profile, being generally safe and well-tolerated by participants. Amylyx plans to present the full data set at an upcoming medical conference and publish the findings in a peer-reviewed journal later in the year.

In light of the PHOENIX study's results, Amylyx will engage in discussions with regulatory authorities and the ALS community over the next two months. The company is considering various options for RELYVRIO/ALBRIOZA, which may include a voluntary withdrawal from the market. For now, the treatment remains accessible to ALS patients, although Amylyx has halted its promotion.

The company's co-CEOs, Justin Klee and Joshua Cohen, expressed their surprise and disappointment, emphasizing their commitment to the ALS community and the importance of sharing the study's findings. They also highlighted the potential of AMX0035 in other neurodegenerative diseases, such as Wolfram syndrome and progressive supranuclear palsy, where ongoing studies are expected to yield results in the coming years.

The PHOENIX study's failure to meet its endpoints raises questions about the efficacy of AMX0035 in treating ALS, contrasting with previous positive results from the CENTAUR trial. However, the safety data from PHOENIX reinforces the drug's tolerability, which is a crucial factor in the treatment of chronic conditions like ALS.

Amylyx's ongoing commitment to research and development in neurodegenerative diseases is evident in their continued studies of AMX0035 and AMX0114, an investigational antisense oligonucleotide targeting calpain-2, a gene implicated in ALS pathogenesis. The company's dedication to the ALS community is underscored by its engagement with stakeholders and its pursuit of potential treatments for this currently incurable disease.

In summary, while the PHOENIX study's results were not as hoped, Amylyx remains dedicated to advancing treatments for ALS and other neurodegenerative conditions, with ongoing studies and a commitment to sharing insights from its research efforts.