Amylyx Pharmaceuticals (AMLX) Announces the Selection of AMX0318 as Development Candidate for PBH and Other Rare Diseases

Amylyx Pharmaceuticals, Inc. (NASDAQ:AMLX) is one of the best performing pharma stocks in 2025. Amylyx Pharmaceuticals, Inc. (NASDAQ:AMLX) announced on January 8 the selection of AMX0318 as a development candidate for post-bariatric hypoglycemia (PBH) and other rare diseases. AMX0318 is a long-acting glucagon-like peptide-1 (GLP-1) receptor antagonist and was identified through a research collaboration with Gubra A/S. Gubra A/S is involved in peptide-based drug discovery and preclinical contract research services.

Camille L. Bedrosian, MD, Chief Medical Officer at Amylyx Pharmaceuticals, Inc. (NASDAQ:AMLX), stated that AMX0318 has exhibited robust preclinical and chemical properties, including “a pharmacokinetic profile that may support long-acting administration.”

Management further demonstrated strong conviction that inhibiting GLP-1 receptor activity marks a significant therapeutic approach, provided the statistically significant data that avexitide, its investigational, first-in-class GLP-1 receptor antagonist, has generated to date. Amylyx Pharmaceuticals, Inc. (NASDAQ:AMLX) expects the completion of recruitment in its pivotal Phase 3 LUCIDITY trial of avexitide by Q1 2026, with topline data expected in Q3 2026.

AMX0318 has completed an elaborate preclinical evaluation, and the company anticipates the program to advance into investigational new drug (IND)-enabling studies later this year. An IND is targeted for 2027, given the pending successful completion of IND-enabling studies.

Amylyx Pharmaceuticals, Inc. (NASDAQ:AMLX) provides therapies for neurodegenerative diseases and amyotrophic lateral sclerosis (ALS). The company’s commercial product is Relyvrio, which is also known as Albrioza in Canada.

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Disclosure: None. This article is originally published at Insider Monkey.