CAMBRIDGE, Mass. — Amylyx Pharmaceuticals Inc. said it has nominated AMX0318, a novel long-acting glucagon-like peptide-1 receptor antagonist, as a development candidate for post-bariatric hypoglycemia and other rare diseases, following its identification through a research collaboration with peptide drug discovery company Gubra A/S.
The company said AMX0318 met key selection criteria, including robust chemical stability, strong in vitro potency, evidence of in vivo efficacy and tolerability, high solubility, and a favorable pharmacokinetic profile consistent with long-acting peptide administration.
“We are very pleased to nominate AMX0318 as a development candidate and were highly impressed by Gubra’s proprietary and innovative process, which identified a peptide that surpassed our research target profile for a long-acting GLP-1 receptor antagonist,” said Camille L. Bedrosian, MD, Chief Medical Officer at Amylyx. “AMX0318 has thus far shown robust preclinical and chemical properties, including a pharmacokinetic profile that may support long-acting administration.”
Bedrosian said the program expands Amylyx’s efforts to address diseases linked to GLP-1 receptor signaling. “We are excited about the opportunity to develop additional therapeutic possibilities for people who may benefit from inhibiting GLP-1 receptor activity, including people with PBH and other rare diseases,” she said. “The GLP-1 receptor is a well-characterized biological target and one of the key regulators of glucose-insulin homeostasis.”
Amylyx said AMX0318 has completed extensive preclinical evaluation, including studies of stability, solubility, potency, pharmacokinetics, pharmacodynamics, and in vivo tolerability. The company expects to initiate investigational new drug-enabling studies in 2026, with an IND filing targeted for 2027, pending successful completion of those studies.
Bedrosian said the decision builds on the company’s experience with avexitide, its investigational first-in-class GLP-1 receptor antagonist. “We have strong conviction that inhibiting GLP-1 receptor activity represents an important therapeutic approach given the statistically significant data that avexitide has generated to date,” she said. Amylyx continues to expect completion of recruitment in its pivotal Phase 3 LUCIDITY trial of avexitide in the first quarter of 2026, with topline data anticipated in the third quarter of 2026.
Louise S. Dalbøge, Chief Science Officer at Gubra, said the nomination reflects the strength of the collaboration. “We are pleased to see AMX0318 advance as a development candidate, a milestone that reflects both the strong collaboration between the Gubra and Amylyx teams and the capabilities of Gubra’s proprietary peptide drug discovery platform,” she said. “Our AI-driven streaMLine platform enables multi-parameter optimization of peptide candidates, and we are excited to see this applied successfully to AMX0318 in close collaboration with Amylyx.”
Under the terms of the collaboration, Gubra is eligible to receive more than $50 million in development and commercialization milestones, as well as mid-single-digit royalties on worldwide net sales. The nomination and handover of the development candidate will trigger milestone payments totaling $4 million to Gubra.
