Amylyx is pursuing regulatory approval for new treatments in rare diseases following the withdrawal of its ALS drug Relyvrio, according to a June 15 article. The company, originally focused on neurodegenerative diseases such as ALS, is now developing therapies for conditions with high unmet needs.
“We only work in areas where there are either no treatments or substantially inadequate treatments,” Amylyx co-CEO Justin Klee said.
Founded in 2013 by Klee and co-CEO Josh Cohen while undergraduates at Brown University, Amylyx initially targeted Alzheimer’s disease before shifting focus to ALS after discussions with researchers. The company received U.S. Food and Drug Administration approval for Relyvrio in September 2022 based on Phase 2 trial data showing slowed progression of ALS. However, a subsequent Phase 3 trial failed to show significant benefit, leading Amylyx to withdraw the drug from U.S. and Canadian markets in April 2024 and reduce its workforce by approximately 70%.
Amylyx’s current pipeline includes avexitide—a GLP-1 receptor antagonist acquired from Eiger BioPharmaceuticals—which is being developed for post-bariatric hypoglycemia (PBH), a complication affecting about 160,000 people in the United States who have undergone bariatric surgery. “People get these episodes at quite frequent times where their blood sugar goes so low that the brain stops functioning properly,” Klee said about PBH. The company expects topline results from a pivotal study of avexitide in the third quarter and plans to submit a New Drug Application if results are positive.
The company is also testing AMX0035—previously marketed as Relyvrio—for Wolfram syndrome, a rare neurodegenerative disorder with both neurological and endocrine symptoms. In May 2025, Amylyx announced positive long-term data from a Phase 2 trial showing stabilization or improvement across several disease outcomes after treatment with AMX0035.
ALS remains central to Amylyx’s mission. Its next candidate for ALS is AMX0114, an investigational antisense oligonucleotide targeting calpain-2 (CAPN2), which plays an essential role in axonal degeneration seen in ALS patients. In December, early-stage clinical results indicated that AMX0114 was generally well-tolerated among participants.
