Azafaros has announced that data from the ongoing double-blind, placebo-controlled Phase 2 RAINBOW study investigating its lead asset, nizubaglustat in patients with Niemann-Pick disease type C (NPC) or GM2 gangliosidosis, were presented at the Society for the Study of Inborn Errors of Metabolism (SSIEM) annual symposium in Porto, Portugal.

The results of part one of the study, designed to determine the safety, pharmacodynamics, and pharmacokinetics of the Company’s lead asset nizubaglustat, demonstrated the compound had a positive safety profile and was well-tolerated in the 13 participants in the study.

All patients are now enrolled in the extension phase of the trial, where initial unblinded data on 10 patients showed 8 had improved or stabilized SARA (Scale for the Assessment and Rating of Ataxia) scores. Additionally, a reduction in seizure frequency was observed for the participants who had experienced daily seizures before recruitment into the study.

Data from the RAINBOW study will be used to inform the target dose for Azafaros’ planned Phase 3 pivotal studies, due to be initiated 2025.

At the conference, Azafaros also presented 12-month follow-up data from the company’s PRONTO trial, a natural history study of late-infantile/juvenile GM1 and GM2 gangliosidoses.

PRONTO is the largest prospective natural history study of these two rare diseases, providing valuable insights for the further development of nizubaglustat in GM1 and GM2 and for the gangliosidoses research community in general.

Stefano Portolano, MD, CEO of Azafaros, said: “The RAINBOW trial results support the positive safety profile of nizubaglustat and provide us with encouraging early efficacy trends for the compound. We greatly appreciate the opportunity to discuss these data with the scientific community at the SSIEM annual symposium, and believe our compound offers a potential new solution to patients with rare lysosomal storage disorders with neurological involvement where there is a strong unmet medical need. We look forward to using the insights provided by results from both RAINBOW and PRONTO to keep the strong momentum of our clinical program and move the compound into pivotal trials next year.”

Azafaros is thankful to the patients and their families for their participation in the RAINBOW study.

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About the RAINBOW study

The RAINBOW trial is a Phase 2 clinical study designed to evaluate safety, tolerability, pharmacokinetics and pharmacodynamics of AZ-3102 (nizubaglustat) in patients with GM2 and Niemann-Pick type C (NPC) diseases. The study was conducted across three sites in Brazil, involving 13 patients older than 12 years of age. In the first part of the study patients were randomized to receive either a high dose or low dose of nizubaglustat or placebo. All patients continued into the second part of the study where they are randomized to receive either high or low dose nizubaglustat for 52 weeks.

About the PRONTO study

The PRONTO study is a prospective natural history study designed to assess the progression of neurological disease in late-infantile and juvenile GM1 and GM2 gangliosidoses. The study includes participants with a late-infantile/juvenile form, genetic diagnosis, and who are between 2 and 20 years old and the main objective was to provide insight and understanding of disease progression using clinical scales, caregiver questionnaires, and actigraphy. A total of 30 participants were recruited from 6 countries with at least 12-month follow-up data on 21 participants.

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 GM2 gangliosidosis (Sandhoff and Tay-Sachs Diseases) and NPC.

IND Clearance and Fast Track Designation for GM1/GM2 gangliosidoses and NPC

European Medicines Agency (EMA)

Orphan Medicinal Product Designation (OMPD) for the treatment of 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 early 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 (NPC) 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 by scientists with a deep understanding of rare genetic disease mechanisms, using discoveries made by scientists at Leiden University and Amsterdam UMC. Azafaros is led by a team of highly experienced industry experts and aims to build a pipeline of disease-modifying therapeutics to offer new treatment options to patients and their families. The Azafaros team is dedicated to rapidly bring new drugs to the rare disease patients who need them. The company is supported by a syndicate of leading investors including Forbion, BioGeneration Ventures (BGV), BioMedPartners, Asahi Kasei Pharma Ventures, and Schroders Capital.

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