Key Takeaways
- The FDA has lifted the clinical hold on Amylyx’s AMX0114, allowing the Phase 1 LUMINA trial to proceed in the U.S. and Canada.
- The trial will enroll 48 ALS patients and will test multiple doses of AMX0114, targeting calpain-2, a protein linked to neurodegenerative diseases.
- Amylyx aims to gather early cohort data by the end of this year, following recent challenges including the market withdrawal of their previous drug, Relyvrio.
FDA Approves Amylyx’s ALS Candidate for Clinical Trials
Amylyx Pharmaceuticals has received the green light from the FDA to proceed with its Phase 1 clinical trial for AMX0114, an experimental treatment for amyotrophic lateral sclerosis (ALS). This follows a previous clinical hold imposed by the FDA due to concerns regarding dosing levels. Initially, Amylyx proposed a starting dose of 12.5 mg for AMX0114, but the FDA lowered this threshold and sought more data, leading to the hold. Consequently, Amylyx shifted its focus to conducting trials in Canada during this period.
The Phase 1 trial, named LUMINA, will now also include sites in the U.S. The study is scheduled to start in Canada in early 2025, with 48 ALS patients expected to participate. Each participant will be administered up to four doses of either a placebo or AMX0114.
AMX0114 is an antisense oligonucleotide designed to inhibit calpain-2, a protease associated with various neurodegenerative diseases. Dr. Camille Bedrosian, Chief Medical Officer of Amylyx, expressed optimism about AMX0114’s potential in treating ALS, citing positive results from preclinical efficacy studies that underscore the role of calpain-2 in axonal degeneration.
The company is also reporting that AMX0114 has shown favorable safety profiles in early preclinical studies, suggesting it may be well tolerated in the patient population. Furthermore, the anticipated early cohort data from the trial is expected to be available later this year.
This trial is a critical step for Amylyx, especially after the setbacks faced with its previous drug, Relyvrio, which was withdrawn from the market after failing to demonstrate improved physical function in a Phase 3 study. This failure prompted significant organizational changes, including a workforce reduction of 70%, and a strategic pivot towards acquiring a new drug, a GLP-1 receptor antagonist from Eiger Biopharma for $35.1 million. The newly acquired drug, avexitide, is preparing for a commercial launch, with Dan Monahan appointed as the new Chief Commercial Officer to lead this initiative.
As Amylyx advances with AMX0114 and strengthens its pipeline, the ongoing developments in both clinical trials and commercial strategies will be pivotal in reshaping the company’s future in the competitive biopharmaceutical landscape.
The content above is a summary. For more details, see the source article.