• Phase 2 RAINBOW study data showed that treatment with nizubaglustat in patients with GM2 gangliosidosis (GM2) and Niemann-Pick type C disease (NPC) reduced disease progression and seizure burden
• Topline results from the study announced in 2024 demonstrated that nizubaglustat was safe and well tolerated, with encouraging signs of efficacy
• The published data further supports the two ongoing registrational Phase 3 studies with nizubaglustat in GM1/GM2 and NPC

Azafaros, a private company building a portfolio to become a leader in lysosomal storage disorders (LSDs), focused on addressing neurological symptoms, today announced the publication of clinical data from its Phase 2 (RAINBOW) study in the peer reviewed journal, Molecular Genetics & Metabolism.

The published manuscript reports efficacy, safety, pharmacokinetic and pharmacodynamic data which evaluated nizubaglustat in patients with genetically confirmed GM2 gangliosidosis and NPC. As previously stated in the topline results announcement in July 2024, the RAINBOW study met its primary objective of demonstrating that nizubaglustat was safe and well tolerated. The study also showed encouraging signs of clinical efficacy, reducing disease progression and seizure burden in patients treated with nizubaglustat.

“The publication of the RAINBOW data in a peer-reviewed journal represents an important milestone for Azafaros and for the broader lysosomal disease community,” said Stefano Portolano, Chief Executive Officer at Azafaros. “These data further support the therapeutic potential of nizubaglustat to address the neuronopathic symptoms in diseases where there is a strong unmet medical need and reinforce our confidence as we advance our two Phase 3 studies treating patients with GM1/GM2 gangliosidoses and NPC.”

“The publication of the RAINBOW study results represents an important step in advancing the scientific understanding of nizubaglustat in GM2 gangliosidosis and Niemann-Pick type C disease,” said Professor Roberto Giugliani, Principal Investigator of the RAINBOW study. “The data support the safety profile observed in the study and provide encouraging evidence for continued clinical development in these devastating rare diseases.”

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 RAINBOW

The RAINBOW study was a randomized, double-blind, placebo-controlled Phase II study evaluating the safety, tolerability, and pharmacological profile of oral nizubaglustat in patients with GM2 gangliosidosis or Niemann-Pick type C disease. The study aimed to determine appropriate dosing and assess how the drug is processed and acts in the body. Following completion of the 12-week placebo-controlled phase, patients entered an ongoing extension phase in which all participants received nizubaglustat. Topline results from the study were announced in 2024.

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.

View source version on businesswire.com: https://www.businesswire.com/news/home/20260604044697/en/

The study also showed encouraging signs of clinical efficacy, reducing disease progression and seizure burden in patients treated with nizubaglustat.