Amylyx Pharmaceuticals, Inc.

Amylyx Pharmaceuticals, Inc. (NASDAQ: AMLX) is a clinical-stage biopharmaceutical company dedicated to developing novel therapies for communities with high unmet medical needs, with a focus on endocrine conditions and neurodegenerative diseases. The company's website is located at www.amylyx.com.

The company's lead investigational asset is avexitide, a first-in-class glucagon-like peptide-1 receptor antagonist designed to treat post-bariatric hypoglycemia and congenital hyperinsulinism. Avexitide has received Breakthrough Therapy Designation from the FDA for both indications, along with Rare Pediatric Disease Designation and Orphan Drug Designation. The company is advancing the LUCIDITY Phase 3 clinical trial, an approximately 75-participant, multicenter, randomized, double-blind, placebo-controlled study evaluating avexitide's efficacy and safety in participants with post-bariatric hypoglycemia following Roux-en-Y gastric bypass surgery.

Beyond avexitide, Amylyx is developing AMX0035, an oral fixed-dose combination of sodium phenylbutyrate and taurursodiol, for Wolfram syndrome, a rare neurodegenerative disease. The company presented positive Week 48 data from its Phase 2 HELIOS trial demonstrating sustained improvements in pancreatic beta cell function and visual acuity. AMX0114, an investigational antisense oligonucleotide targeting calpain-2, is being evaluated in the Phase 1 LUMINA clinical trial for amyotrophic lateral sclerosis. Additionally, AMX0318, a novel GLP-1 receptor antagonist selected as a development candidate in January 2026, is being pursued for long-acting administration in post-bariatric hypoglycemia and other rare diseases.

The company's intellectual property portfolio includes 23 issued U.S. patents, 257 granted foreign patents, over 15 pending U.S. patent applications, and over 115 pending foreign patent applications as of December 31, 2025. Amylyx relies on third-party contract manufacturing organizations for production of its investigational compounds in compliance with current Good Manufacturing Practice requirements. The company has assembled a team of pharmaceutical industry technical operations leaders with extensive experience in manufacturing, quality, regulatory compliance, and project management to oversee third-party manufacturers and maintain quality standards.