• Five presentations will showcase nizubaglustat’s therapeutic potential in Lysosomal Storage Disorders.
• Nizubaglustat is currently being tested in two global Phase 3 studies for Niemann-Pick type C disease and GM1/GM2 gangliosidoses which are currently recruiting.
• Azafaros will host a satellite symposium on nizubaglustat on Day 2 of the symposium discussing the Phase 3 studies.
• WORLDSymposium™ 2026 takes place from February 2 to 6 in San Diego, California.

Azafaros, a company building a portfolio to become a leader in lysosomal storage disorders with the goal of addressing neurological symptoms, today announced that its lead asset, nizubaglustat, will feature in five scientific presentations accepted at the WORLDSymposium™ 2026, the leading global conference on lysosomal diseases, taking place in San Diego, California, USA between February 2-6.

The company’s conference activities include:

Oral Presentations (including Poster numbers 134 and 29-C)

• Long-term data from a Phase II study with oral nizubaglustat for late-infantile/juvenile GM2 and Niemann-Pick Type C diseases (RAINBOW) on February 5, presenting preliminary clinical efficacy and safety data. The data underscore nizubaglustat’s potential as a therapeutic option, reinforcing Azafaros’ commitment to addressing critical unmet needs in lysosomal storage disorders.
• Nizubaglustat reinstates pro-neuronal transcriptional programs in human CLN3 retinal organoids on February 6 (Rapid Fire session) highlighting nizubaglustat as a promising candidate for further preclinical and clinical development in CLN3 Batten disease.

Satellite Symposium on Nizubaglustat Phase 3 Study NAVIGATE

• Azafaros will host a satellite symposium entitled “The value of conducting an 18-month placebo-controlled study in Rare Diseases: GM1, GM2 and NPC; the NAVIGATE experience” on February 3 at 17:45 PT, creating an opportunity to engage key opinion leaders, healthcare professionals and patient advocacy organizations.

Poster Presentations:

• February 4:
  • Participation in clinical trials for neurological lysosomal disease: Patient and family perspectives – Poster number 129, highlighting patient and caregiver experiences.

• February 5:
  • A prospective natural history study for GM1 and GM2: 24-month data from the PRONTO study – Poster number 132, providing insights into disease progression in GM1 and GM2 gangliosidoses.
  • A Phase III study to evaluate the efficacy of nizubaglustat on GM1, GM2, and Niemann-Pick Type C diseases (NAVIGATE) – Poster number 230, outlining the design and objectives of this pivotal trial.

“We are proud to again be able to present our research at the prestigious WORLDSymposium™ 2026 conference, and in particular to showcase the promise of our lead product nizubaglustat,” said Stefano Portolano, Chief Executive Officer at Azafaros. “We strongly believe nizubaglustat has great potential to become a new therapeutic treatment for lysosomal storage disorders, in particular Niemann-Pick Type C disease and GM1 and GM2 Gangliosidoses, where there is a strong unmet medical need.

“Several sites are currently recruiting across key countries in the EU, India, and the US as part of our Phase 3 program, and we look forward to advancing both studies in collaboration with the global medical community and patient associations to bring new treatment solutions 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 disease type C (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), 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 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 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 healthcare investors including Forbion, Jeito Capital, Seroba, Pictet Group, BioGeneration Ventures (BGV), BioMedPartners, Asahi Kasei Pharma Ventures, and Schroders Capital.

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Five Azafaros scientific presentations accepted at WORLDSymposium™ 2026