Phase 3 study of AZ-3102 in patients with GM1/GM2 gangliosidoses
Latest Information Update: 19 May 2025
At a glance
- Drugs Nizubaglustat (Primary)
- Indications GM1 gangliosidosis; GM2 gangliosidoses
- Focus Registrational; Therapeutic Use
- Sponsors Azafaros
Most Recent Events
- 13 May 2025 According to an Azafaros media release, company expects to initiate this study later this year.
- 08 Jan 2025 According to an Azafaros media release, the company's Clinical Trial Application (CTA) for two global Phase 3 studies investigating the drug's efficacy and safety in GM1/GM2 gangliosidoses and Niemann-Pick Type C (NPC) was approved by multiple European countries. Company expects to initiate this two trials in Q2, 2025.
- 19 Nov 2024 According to an Azafaros media release, the company will host a satellite symposium on February 4, 2025 at 12:15, which will outline the company's Phase 3 global development plans for nizubaglustat. These upcoming global Phase 3 studies for GM1 and GM2 gangliosidoses and NPC are set to be conducted across major regions including the United States, Europe, Latin America, and other select countries.