Azafaros announces completion of 12-week Phase 2 RAINBOW study
Azafaros to present data from PRONTO study in patients with GM1 and GM2 gangliosidoses at the 20th annual WORLDSymposium Four posters with baseline data from the natural history (PRONTO) study in...
Azafaros™ Phase 2 RAINBOW study, evaluating nizubaglustat in GM2 and NPC patients, is now fully enrolled Topline data from the study, expected for mid-2024, will support two pivotal Phase...
LEIDEN, The Netherlands--(BUSINESS WIRE)--Azafaros B.V. today announced that the first patient has been enrolled into its Phase 2 RAINBOW study (NCT05758922). The clinical trial is being conducted in Brazil and the US and will evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics across two doses of its lead asset, AZ-3102, in patients with GM2 gangliosidosis and Niemann-Pick disease type C (NP-C).
Azafaros Appoints Chief Operating Officer and Head of Finance
LEIDEN, Netherlands--(BUSINESS WIRE)--Azafaros B.V. today announced its progress in ongoing interactions with Health Authorities regarding its lead asset, AZ-3102.
Azafaros Strengthens Medical and Scientific Management Team Reflecting Clinical Progress
LEIDEN, The Netherlands--(BUSINESS WIRE)--Azafaros B.V. today announced that it has received Investigational New Drug (IND) clearance from the United States Food and Drug Administration (FDA) to conduct a clinical Phase 2 trial for its lead asset, AZ-3102, for the treatment of GM2 gangliosidosis (GM2) and Niemann-Pick disease type C (NP-C). In addition to the IND clearance, the FDA has granted Fast Track Designation for the investigation of AZ-3102 for GM1 and GM2 gangliosidoses and NP-C. Fast Track is a process designed to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need.1
LEIDEN, The Netherlands--(BUSINESS WIRE)--Azafaros B.V. today announced that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) for AZ-3102, a novel small molecule with a unique dual mode of action, in Niemann-Pick disease type C (NP-C). The designation was based on promising preclinical data of AZ-3120 in a NP-C mouse model, recently presented at the 18th Annual WORLDSymposium™. AZ-3102, Azafaros’ lead program, is currently in clinical development as a potential treatment for the rare lysosomal storage diseases GM1 and GM2 Gangliosidoses and has completed a successful first-in-human clinical study in healthy subjects showing positive safety, tolerability, and pharmacodynamics data. The compound already received ODD from the FDA for GM2 Gangliosidosis including both Sandhoff and Tay-Sachs diseases. Based on its mode of action, AZ-3102 has broad applicability in addressing these inherited metabolic disorders.
LEIDEN, The Netherlands--(BUSINESS WIRE)--Azafaros B.V. announced positive clinical data from its first-in-human Phase 1 study with AZ-3102, the company’s lead program in development as a potential treatment for pediatric neurogenetic lysosomal storage disorders (LSDs). AZ-3102 is an azasugar, orally available, small molecule designed to be a potent and selective inhibitor of two target enzymes involved in glycolipid metabolism by modulating the metabolism of glycosphingolipids. Azafaros also presented the design of PRONTO, a prospective longitudinal global study of neurological disease trajectory in children living with late-infantile or juvenile onset of GM1 or GM2 Gangliosidoses.