Azafaros to Present at J.P. Morgan’s 43rd Annual Healthcare Conference

Azafaros, a company focused on developing treatments for the unmet needs of patients with rare lysosomal storage disorders, today announced that it will present at J.P. Morgan’s 43^rd Annual Healthcare Conference on Thursday, January 16, 2025.

The company’s presentation will begin at 8:30 a.m. PT/11:30 am (Eastern Time).

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 (NPC).

Positive topline data reported earlier this year from the company’s successful Phase 2 study investigating nizubaglustat in GM2 and NPC patients demonstrated that the compound had a positive safety profile. Preliminary improvements or stabilization of clinical endpoints were observed in the majority of patients, highlighting encouraging early efficacy trends for the compound.

Azafaros is preparing to initiate Phase 3 studies with nizubaglustat in GM1/GM2 gangliosidoses and NPC in Q2, 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 disease type C (NPC).

Nizubaglustat has received the following designations and support:

United States Food and Drug Administration (FDA)
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.
Fast Track Designation and IND clearance for GM1/GM2 gangliosidoses and NPC

European Medicines Agency (EMA)
Orphan Medicinal Product Designation (OMPD) for the treatment of for GM1 and GM2 gangliosidosis GM2 Gangliosidosis.

UK Medicines and Healthcare Products Regulatory Agency (MHRA)
Innovation Passport for the treatment of GM1 and GM2 gangliosidoses.

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), resulting 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 disease type C (NPC)
Niemann-Pick disease type C 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 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 a syndicate of leading Dutch and Swiss investors including Forbion, BioGeneration Ventures (BGV), BioMedPartners, Asahi Kasei Pharma Ventures, and Schroders Capital.

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