Azafaros, a company focused on developing treatments for the unmet needs of patients with rare lysosomal storage disorders, announced its participation in J.P. Morgan’s 43rd Annual Healthcare Conference. The presentation is scheduled for January 16, 2025, at 8:30 a.m. PT/11:30 a.m. ET.
The focus of the presentation will be on nizubaglustat, Azafaros' lead product, which is being developed as a potential treatment for rare lysosomal storage disorders with neurological involvement, including GM1/GM2 gangliosidoses and Niemann Pick type C (NPC). Positive topline data from the company’s Phase 2 study in GM2 and NPC patients have shown that nizubaglustat has a positive safety profile, with preliminary improvements or stabilization of clinical endpoints observed in the majority of patients.
Nizubaglustat is a small molecule, orally available and brain penetrant azasugar with a unique dual mode of action. It has received several designations and support from regulatory bodies, including Rare Pediatric Disease Designations, Orphan Drug Designations, Fast Track Designation, and IND clearance from the United States Food and Drug Administration (FDA), as well as Orphan Medicinal Product Designation from the European Medicines Agency (EMA) and an Innovation Passport from the UK Medicines and Healthcare Products Regulatory Agency (MHRA).
GM1 gangliosidosis and GM2 gangliosidosis (Tay-Sachs and Sandhoff diseases) are lysosomal storage disorders that lead to progressive and severe neurological impairment and premature death, primarily affecting infants and children. Currently, there are no disease-modifying treatments available for these conditions. Azafaros is preparing to initiate Phase 3 studies with nizubaglustat in GM1/GM2 gangliosidoses and NPC in Q2, 2025.
