Amylyx Pivots to GLP-1 Space, Acquires Phase III-Ready Asset from Eiger

From left: Amylyx Co-CEOs Josh Cohen and Justin Kl

From left: Amylyx Co-CEOs Josh Cohen and Justin Kl

After pulling its ALS drug Relyvrio from the market, Amylyx Pharmaceuticals is looking to target the GLP-1 space with the purchase of Eiger BioPharmaceuticals’ avexitide, which has been studied for the treatment of hyperinsulinemic hypoglycemia.

Amylyx Pharmaceuticals announced Wednesday it has acquired a glucagon-like peptide-1 receptor antagonist, avexitide, from Eiger BioPharmaceuticals that is ready for Phase III trials and has been granted FDA Breakthrough Therapy Designation for post-bariatric hypoglycemia and congenital hyperinsulinism.

The novel, first-in-class GLP-1 was picked up for $35 million from Eiger, which entered Chapter 11 proceedings in April 2024 with plans to sell all its assets. According to Amylyx, avexitide has already been investigated in five clinical trials for post-bariatric hypoglycemia (PBH), a common complication of bariatric surgery, and has also been studied in congenital hyperinsulinism (HI). Avexitide has also been granted an FDA Orphan Drug Designation in hyperinsulinemic hypoglycemia and a Rare Pediatric Disease Designation in congenital HI.

The company is placing avexitide into its pipeline and is planning to start Phase III studies in PBH in the first quarter 2025. The company is also starting conversations with the HI community to start a Phase II study at the Children’s Hospital of Philadelphia. Josh Cohen, co-CEO of Amylyx, said the company plans to get the Phase III data by 2026 and to go to market by 2027.

So far, Phase II and Phase IIb studies of avexitide in PBH have shown a statistically significant reduction in hypoglycemic events characterized by low blood glucose.

Justin Klee, co-CEO of Amylyx, told BioSpace that the company is searching for assets with a clear mechanism of action for targeting diseases..

“We want to have compelling preclinical and clinical data, and many ways, most importantly, an opportunity to make a real impact and help people with high-end medical needs,” Klee said. “And in many ways, this was the first asset that really checked all the boxes.”

Klee noted that GLP-1 agonists are helpful in conditions characterized by too much glucose and insufficient insulin. Still, some diseases have too much insulin and not enough glucose, which is what avexitide is meant to counter.

Amylyx’s targeting of the GLP-1 space follows the company’s decision to pull its ALS drug Relyvrio off the shelves earlier this year and to axe around 70% of its workforce under a restructuring plan. The ALS drug failed its Phase III PHOENIX trial and could not improve scores on the Revised Amyotrophic Lateral Sclerosis Functional Rating Scale at 48 weeks versus placebo, while falling short of its secondary endpoints.

The company has been focused on its lead asset, AMX0035, for adults with Wolfram syndrome. In April 2024, a Phase II study showed that the candidate improved pancreatic function and glycemic control.