Azafaros B.V. has announced that its lead asset, nizubaglustat, has been granted orphan drug designation from regulatory authorities in both the United States and the European Union for the treatment of GM1 gangliosidosis. Furthermore, the company's Clinical Trial Application (CTA) has been approved in multiple European countries for two global Phase 3 studies investigating the drug’s efficacy and safety in GM1/GM2 gangliosidoses and Niemann-Pick Type C (NPC). Azafaros anticipates initiating these trials in Q2 2025.
The orphan drug designations from the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) provide strategically important advantages in drug development, particularly during the regulatory process, according to Azafaros. These designations cover GM1 gangliosidosis, a rare disease for which there is currently no approved treatment available.
Clinical Trial Plans
The planned Phase 3 trials will be conducted across major regions, including the United States, Europe, Latin America, and other select countries. These trials aim to confirm the positive safety profile and preliminary efficacy trends observed in the company's Phase 2 study of nizubaglustat in GM2 and NPC patients, which demonstrated improvements or stabilization of clinical endpoints in the majority of patients.
Nizubaglustat: A Potential First-in-Class Therapy
Nizubaglustat is a small molecule, orally available, and brain-penetrant azasugar with a unique dual mode of action. It is being developed as a potential treatment for rare lysosomal storage disorders with neurological involvement, including GM1 and GM2 gangliosidoses and Niemann-Pick disease type C (NPC).
Stefano Portolano, Chief Executive Officer at Azafaros, stated that nizubaglustat, with its dual mode of action, represents a significant leap forward compared to existing therapies and is positioned to be the first-in-class therapy for GM1 and GM2 gangliosidoses and the best-in-class therapy for NPC. He also acknowledged the limited treatment options available to patients with these diseases and expressed gratitude to them and their families for their contribution to the company's clinical progress.
About GM1 and GM2 Gangliosidoses and NPC
GM1 and GM2 gangliosidoses are lysosomal storage disorders caused by the accumulation of GM1 or GM2 gangliosides, respectively, in the central nervous system (CNS). This accumulation leads to progressive and severe neurological impairment and premature death, primarily affecting infants and children. Currently, there are no disease-modifying treatments available.
Niemann-Pick disease type C (NPC) is a progressive, life-limiting neurological lysosomal storage disorder caused by mutations in the NPC1 or NPC2 gene, resulting in aberrant endosomal-lysosomal trafficking and the accumulation of various lipids, including gangliosides, in the CNS. The onset of the disease can occur throughout the lifespan, from prenatal life through adulthood.
