Every day represents an opportunity to alter the course of diseases with high unmet needs and the people they affect–and a responsibility to support and potentially improve the lives of people, families, and communities around the world.
AMX0035 is an oral, fixed-dose combination of sodium phenylbutyrate (PB) and taurursodiol (TURSO; also known as ursodoxicoltaurine outside of the U.S.). AMX0035 was designed to slow or mitigate neurodegeneration by targeting endoplasmic reticulum (ER) stress and mitochondrial dysfunction, two connected central pathways that lead to cell death and neurodegeneration.
Preclinical
IND-Enabling Studies
Phase 1
Phase 2
Phase 3
Commercial
Wolfram Syndrome
Progressive Supranuclear Palsy (PSP)
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Avexitide is an investigational, first-in-class glucagon-like peptide-1 (GLP-1) receptor antagonist with FDA Breakthrough Therapy and Rare Pediatric Disease (HI only) designations and highly statistically significant data and well-tolerated safety profile replicated across multiple clinical trials.
Preclinical
IND-Enabling Studies
Phase 1
Phase 2
Phase 3
Commercial
Post-Bariatric Hypoglycemia (PBH)
Congenital Hyperinsulinism (HI)
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AMX0114 is an investigational antisense oligonucleotide (ASO) designed to target the gene encoding calpain-2 (CAPN2). Calpain-2 is a calcium-dependent protease and a critical effector of axonal degeneration (also known as Wallerian degeneration) in ALS and other neurodegenerative diseases.
Preclinical
IND-Enabling Studies
Phase 1
Phase 2
Phase 3
Commercial
Amyotrophic Lateral Sclerosis (ALS)
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