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LEIDEN, Netherlands --(BUSINESS WIRE)-- 15.06.2026 --
Azafaros, a private company building a portfolio to become a leader in lysosomal storage disorders (LSDs), focused on addressing neurological symptoms, today announced that it will present at the 2026 BIO International Convention taking place in San Diego, USA, from June 22-25. The company's presentation is scheduled for Monday, June 22 at 4:30 PM in Theater 4.
The presentation will highlight Azafaros' progress in developing nizubaglustat, its lead investigational compound for the treatment of rare lysosomal storage disorders with neurological involvement, including GM1 and GM2 gangliosidoses (GM1/GM2) and Niemann-Pick type C disease (NPC).
Nizubaglustat is currently in two Phase 3 registrational studies (NAVIGATE) treating patients with GM1/GM2 and NPC and expects to report topline data from the studies in 2028.
"BIO provides an important opportunity to update our progress as we continue advancing nizubaglustat through late-stage clinical development," said Stefano Portolano, CEO at Azafaros. "With substantial unmet need remaining in GM1 and GM2 gangliosidoses and NPC, we remain focused on executing our Phase 3 program and preparing for future regulatory interactions aimed at bringing this potential therapy to patients."
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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For further information:
Azafaros B.V.
Email: info@azafaros.com
www.azafaros.com
