Azafaros to Present at BIO International Convention 2025 Following Successful €132M Series B Financing

Azafaros, a clinical-stage biotechnology company developing novel therapies for rare lysosomal storage disorders (LSDs), today announced that it will present at the 2025 BIO International Convention taking place in Boston, USA, from 16-19 June. The company’s presentation is scheduled for Monday, June 16 at 16:45 ET in Room 153B.

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 and Niemann-Pick disease type C (NPC).

This announcement follows Azafaros’ recently completed and oversubscribed €132 million Series B financing round, which will support advancing nizubaglustat into pivotal clinical development. The company is on track to initiate two Phase 3 trials in GM1/GM2 gangliosidoses and NPC in 2025.

Earlier this year, Azafaros reported positive topline results from its ongoing Phase 2 study evaluating nizubaglustat in patients with GM2 gangliosidosis and NPC. The current Ph2 extension study demonstrated a favorable safety profile after more than 12 months of treatment. In addition, preliminary improvements or stabilization in key clinical endpoints were observed in the majority of patients, underscoring promising efficacy trends for nizubaglustat.

“We are excited to share our latest progress at BIO 2025, including the continued clinical development of nizubaglustat which holds strong potential as a meaningful treatment option for patients with devastating neurodegenerative lysosomal disorders,” said Stefano Portolano, CEO at Azafaros. “With the support of our recent financing round and encouraging data from our ongoing trials, we can continue our rapid momentum to bring this product into pivotal clinical studies as we work to develop a potential new solution for this area where there is a significant unmet need.”

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 Rare Paediatric 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 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 leading healthcare investors including Jeito Capital, Forbion Growth, Seroba, Pictet Group and a syndicate of leading Dutch and Swiss existing investors including Forbion Ventures, BioGeneration Ventures (BGV), BioMedPartners, Asahi Kasei Pharma Ventures, and Schroders Capital.

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