Azafaros to Present at J.P. Morgan’s 44th Annual Healthcare Conference

Azafaros, a company building a portfolio to become a leader in lysosomal storage disorders with the goal of addressing neurological symptoms, today announced that it will present at J.P. Morgan’s 44^th Annual Healthcare Conference on Thursday, January 15.

The company’s presentation will begin at 9:30 am PT/12:30 am ET/6:30 am CET.

The presentation will focus on the company’s lead product, nizubaglustat, a potential treatment for rare lysosomal storage disorders with neurological involvement including GM1/GM2 gangliosidoses and Niemann-Pick type C disease ((NPC).

Two pivotal Phase 3 studies investigating nizubaglustat in GM1/GM2 gangliosidoses and NPC are currently enrolling, with data expected in 2027. The studies build on positive data from a robust clinical program including the Phase 2 RAINBOW study and the PRONTO natural history study. The Phase 3 studies are financed following the company’s successful completion of €132 million series B round completed in 1H 2025.

About nizubaglustat

Nizubaglustat is a small molecule, orally available and brain penetrant azasugar with a unique dual mode of action, developed as a potential treatment for rare lysosomal storage disorders with neurological involvement, including GM1 and GM2 gangliosidoses and Niemann-Pick type C disease (NPC).

Nizubaglustat has received Rare Pediatric Disease Designations (RPDD) for the treatment of GM1 and GM2 gangliosidoses and NPC, Orphan Drug Designations (ODD) for GM1 and GM2 gangliosidosis (Sandhoff and Tay-Sachs Diseases) and NPC, as well as Fast Track Designation and IND clearance for GM1/GM2 gangliosidoses and NPC from the US Food and Drug Administration (FDA). Additionally, nizubaglustat has been awarded Orphan Medicinal Product Designation (OMPD) for the treatment of GM1 and GM2 gangliosidoses by the European Medicines Agency (EMA) and Innovation Passport for the treatment of GM1 and GM2 gangliosidoses from the UK Medicines and Healthcare Products Regulatory Agency (MHRA).

About GM1 and GM2 gangliosidoses

GM1 gangliosidosis and GM2 gangliosidosis (Tay-Sachs and Sandhoff diseases) are lysosomal storage disorders caused by the accumulation of GM1 or GM2 gangliosides respectively, in the central nervous system (CNS). This results in progressive and severe neurological impairment and premature death. These diseases mostly affect infants and children, and no disease-modifying treatments are currently available.

About Niemann-Pick type C disease (NPC)

Niemann-Pick Type C disease is a progressive, life-limiting, neurological, lysosomal storage disorder, caused by mutations in the NPC1 or NPC2 gene and aberrant endosomal-lysosomal trafficking, leading to the accumulation of various lipids, including gangliosides in the CNS. The onset of the disease can happen throughout the lifespan of an affected individual, from prenatal life through adulthood.

About Azafaros

Azafaros is a clinical-stage company founded in 2018 with a deep understanding of rare genetic disease mechanisms using compound discoveries made by scientists at Leiden University and Amsterdam UMC and is led by a team of highly experienced industry experts. Azafaros aims to build a pipeline of disease-modifying therapeutics to offer new treatment options to patients and their families. By applying its knowledge, network and courage, the Azafaros team challenges traditional development pathways to rapidly bring new drugs to the rare disease patients who need them. Azafaros is supported by leading healthcare investors including Forbion, Jeito Capital, Seroba, Pictet Group, BioGeneration Ventures (BGV), BioMedPartners, Asahi Kasei Pharma Ventures, and Schroders Capital.

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