Our Lead Programme

Our Lead Programme

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 clinical development as a potential treatment for the rare lysosomal storage diseases GM1 and GM2 gangliosidoses and Niemann-Pick disease type C. Azafaros has completed a successful first-in-human clinical study in healthy subjects, showing a favourable safety and tolerability profile and positive pharmacokinetics and pharmacodynamics data.

The compound has already received ODD from the FDA for GM2 Gangliosidosis (Sandhoff and Tay-Sachs diseases) and Niemann-Pick disease type C.

Nizubaglustat's potential in other lysosomal storage disorders

Based on its mode of action, nizubaglustat has broad applicability in addressing other LSDs with intracellular accumulation of glycosphingolipids. Promising preclinical data in a Niemann-Pick disease type C study display a reduction of tremor levels and sparing of cerebellar Purkinje cell depletion, two hallmarks in the untreated disease model. Supported by the Orphan Drug Designation we received from the FDA, we plan to advance nizubaglustat as a potential disease-modifying treatment for Niemann-Pick disease type C.