Nizubaglustat Phase 2 RAINBOW study data on NPC and GM2 gangliosidosis presented by Azafaros

Azafaros, based in Leiden, Netherlands, has reported data from its ongoing Phase 2 RAINBOW study, which evaluates the safety and effectiveness of its primary drug candidate, nizubaglustat, in treating patients with Niemann-Pick disease type C (NPC) and GM2 gangliosidosis. The findings were shared at the Society for the Study of Inborn Errors of Metabolism (SSIEM) annual symposium in Porto, Portugal.

The initial results from the RAINBOW study, which is double-blind and placebo-controlled, involved 13 patients and focused on assessing the safety, pharmacodynamics, and pharmacokinetics of nizubaglustat. The data showed that the drug was well-tolerated and had a favorable safety profile. All participants have now advanced to the extension phase of the trial. Preliminary unblinded results from this phase, involving 10 patients, indicated that 8 patients showed either improvement or stabilization in their SARA (Scale for the Assessment and Rating of Ataxia) scores. Furthermore, a decrease in seizure frequency was noted among participants who had daily seizures prior to the study.

The insights gained from the RAINBOW study will inform the dosage for Azafaros' planned Phase 3 pivotal studies, which are expected to commence in 2025.

At the same symposium, Azafaros also unveiled 12-month follow-up data from the PRONTO trial, a natural history study examining late-infantile and juvenile GM1 and GM2 gangliosidoses. PRONTO is the most extensive prospective natural history study of these rare diseases, providing essential information for the continued development of nizubaglustat and contributing to the broader research community focused on gangliosidoses.

Dr. Stefano Portolano, CEO of Azafaros, highlighted the significance of these findings, stating that the RAINBOW trial results reinforce the positive safety profile of nizubaglustat and show promising early efficacy trends. He emphasized the importance of discussing these findings with the scientific community and expressed gratitude to the patients and their families for their participation in the study. Dr. Portolano also mentioned that the data from both RAINBOW and PRONTO would help maintain the momentum of their clinical program and advance the compound to pivotal trials in the upcoming year.

The RAINBOW study is designed to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of nizubaglustat (AZ-3102) in patients with GM2 and NPC diseases. Conducted at three sites in Brazil, the study involved 13 patients aged over 12 years. The first part of the study randomized patients to receive either a high dose, low dose of nizubaglustat, or a placebo. All patients then moved to the second part, receiving either a high or low dose of nizubaglustat for 52 weeks.

The PRONTO study, on the other hand, is a prospective natural history trial aimed at understanding the progression of neurological diseases in late-infantile and juvenile GM1 and GM2 gangliosidoses. Participants, aged between 2 and 20 years, were enrolled from six countries, with at least 21 of them providing 12-month follow-up data. The study utilized clinical scales, caregiver questionnaires, and actigraphy to gather data.

Nizubaglustat is an orally available, brain-penetrant azasugar with a unique dual mode of action, developed as a potential treatment for rare lysosomal storage disorders with neurological involvement, such as GM1 and GM2 gangliosidoses and NPC. It has received multiple designations and support from regulatory bodies, including the FDA and EMA, for its potential to treat these rare diseases.

Azafaros, founded in 2018, focuses on developing disease-modifying therapeutics for rare genetic diseases. The company is led by a team of experienced industry experts and supported by leading investors like Forbion, BioGeneration Ventures, BioMedPartners, Asahi Kasei Pharma Ventures, and Schroders Capital.

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