We are applying our deep understanding of underlying metabolic pathways
At Azafaros, we are applying our deep understanding of underlying metabolic pathways to develop new classes of oral small molecule compounds designed to counteract the pathological effects of accumulated metabolites in several rare metabolic disorders.
Our lead programme is nizubaglustat. The novel small molecule, with a unique dual mode of action, has the potential to offer disease-modifying modalities that are effective regardless of the genotypes of the patients. In addition, nizubaglustat is an oral molecule, offering the potential of convenient administration and life-long treatment at home, significantly preserving the quality of life for patients, especially children. They also avoid the risks associated with gene or enzyme replacement therapies.
Nizubaglustat is currently in late-stage clinical development as a potential treatment for rare lysosomal storage diseases GM1 and GM2 gangliosidoses and Niemann-Pick type C disease. Azafaros has been tested in both Phase 1 and Phase 2 studies and the company initiated two Phase three studies in these indications in Q3, 2025.
The compound has already received multiple regulatory approvals, including Orphan Drug Designation (ODD) from the US Food and Drug Administration (FDA) for GM1 Gangliosidosis, GM2 Gangliosidosis (Sandhoff and Tay-Sachs diseases) and Niemann-Pick type C disease.
Nizubaglustat's potential in other lysosomal storage disorders
Based on its mode of action, nizubaglustat has broad applicability in addressing other potential indications.