The Phase II trial assessed nizubaglustat to treat patients with either GM2 gangliosidosis or Niemann-Pick disease type C. Credit: Kateryna Kon / Shutterstock.

Azafaros has reported positive topline findings from the Phase II RAINBOW clinical trial of nizubaglustat to treat patients with either GM2 gangliosidosis or Niemann-Pick disease type C (NPC).

Carried out at three sites in Brazil, the trial enrolled 13 subjects aged 12 years and above.

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The trial assessed the safety, pharmacokinetics and pharmacodynamics of two varying doses of nizubaglustat.

Patients who have completed the 12-week, placebo-controlled, double-blind portion of the trial have entered the extension portion, which will see all trial subjects receive the drug.

Based on the latest data, the company will select the target dose of nizubaglustat for the prospective Phase III trials in the future.

An orally available and brain-penetrant azasugar possessing a dual mode of action, nizubaglustat is developed to treat rare lysosomal storage disorders that comprise neurological connections, including GM1 and GM2 gangliosidoses and NPC.

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The drug has obtained various designations from the US Food and Drug Administration (FDA), including rare pediatric disease and orphan drug designations to treat GM1 and GM2 gangliosidoses and NPC and GM2 gangliosidosis (Sandhoff and Tay-Sachs Diseases) and NPC, respectively.

It has also obtained IND clearance and fast-track designation for GM1/GM2 gangliosidoses and NPC.

An orphan medicinal product designation was granted by the European Medicines Agency (EMA) to treat GM2 gangliosidosis.

Azafaros CEO Stefano Portolano said: “The results of the Phase II RAINBOW study mark a pivotal moment in the development of nizubaglustat. The dual mode of action sets nizubaglustat apart as a leap forward from other agents.

“We are excited by the results of the study and more than confident that nizubaglustat has the potential to become a convenient, life-changing therapy for patients affected by GM1 and GM2 gangliosidoses and NPC. Azafaros is thankful to the patients and their families for their important contribution to the RAINBOW study. Azafaros is now poised to embark on a global Phase III trial next year.”