A Phase 3 study of AMX0035
Latest Information Update: 15 Mar 2025
At a glance
- Drugs Sodium phenylbutyrate/ursodoxicoltaurine (Primary)
- Indications Wolfram syndrome
- Focus Registrational; Therapeutic Use
- Sponsors Amylyx Pharmaceuticals
- 04 Mar 2025 According to an Amylyx Pharmaceuticals media release, Week 48 data from the Phase 2 HELIOS trial will inform the design of this Phase 3 trial of AMX0035 in Wolfram syndrome.
- 13 Aug 2024 New trial record
- 08 Aug 2024 According to an Amylyx Pharmaceuticals media release, the company is engaging with stakeholders, including the FDA, and planning for a single Phase 3 clinical trial. Amylyx will share additional details on the clinical trial design once finalized.