Amylyx is a clinical-stage biotech focused on developing therapies for rare diseases with no approved treatments. Its lead asset is avexitide, a first-in-class GLP-1 receptor antagonist targeting post-bariatric hypoglycemia (PBH), a condition affecting an estimated 160,000 people in the U.S. who develop persistent, dangerous hypoglycemia following bariatric surgery. PBH is caused by an exaggerated GLP-1 response that drives excessive insulin secretion; avexitide blocks the GLP-1 receptor on pancreatic beta cells to stabilize blood glucose. It is administered as a once-daily subcutaneous injection. Avexitide holds FDA Breakthrough Therapy and Orphan Drug designations, and Amylyx is currently running LUCIDITY, a Phase 3 trial with enrollment complete as of early 2026, with topline data expected in Q3 2026 and a commercial launch targeted for 2027. Beyond avexitide, Amylyx is advancing AMX0035, an oral combination therapy targeting ER stress and mitochondrial dysfunction in Wolfram syndrome, a rare fatal neurodegenerative disease affecting roughly 3,000 people in the U.S., with a Phase 3 planned for the second half of 2026. Amylyx is also developing AMX0114, an antisense oligonucleotide targeting axonal degeneration in ALS, currently in Phase 1, and AMX0318, a long-acting GLP-1 receptor antagonist in early development. Amylyx generates no revenue and funds operations through equity raises, with roughly $344M in cash as of Q3 2025.
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