A Phase II Study of Safety and Efficacy of AMX0035 in Adult Patients With Wolfram Syndrome
Latest Information Update: 16 May 2025
At a glance
- Drugs Sodium phenylbutyrate/ursodoxicoltaurine (Primary)
- Indications Wolfram syndrome
- Focus Adverse reactions; Proof of concept; Therapeutic Use
- Acronyms HELIOS
- Sponsors Amylyx Pharmaceuticals
Most Recent Events
- 12 May 2025 Results presented in an Amylyx Pharmaceuticals media release.
- 08 May 2025 According to an Amylyx Pharmaceuticals media release,the company is planning to present Week 48 data from the ongoing Phase 2 HELIOS trial of AMX0035 in Wolfram syndrome to be presented at the Joint Congress of the European Society for Pediatric Endocrinology (ESPE) and the European Society of Endocrinology (ESE) taking place from May 10-13.
- 02 Apr 2025 Planned End Date changed from 30 Jan 2026 to 31 Dec 2026.