Azafaros RAINBOW study update: Completion of 12 week phase 2

Azafaros has just announced the successful completion of its 12-week Phase 2 clinical study, RAINBOW, evaluating their lead asset, nizubaglustat, in patients with rare diseases.

This study, conducted in Brazil, involved 13 patients aged 12 and above affected by GM2 gangliosidosis and Niemann-Pick disease type C (NPC). It is a randomized, double-blind, placebo-controlled study, aimed to assess the safety, pharmacodynamics, and pharmacokinetics of two different doses of nizubaglustat.

Prof. Dr. Roberto Giugliani, Lead Principal Investigator for the study, highlighted nizubaglustat’s potential as a first-in-class therapy, offering hope for patients and families affected by these rare diseases.

Chris Freitag, Chief Medical Officer at Azafaros stated that top line results of the RAINBOW study are expected in Q2 2024, with further presentations to the scientific community slated for later this year.

The full press release can be read below or read on Azafaros website.

Azafaros announces completion of 12-week Phase 2 RAINBOW study evaluating lead asset nizubaglustat in rare disease patients

Leiden, The Netherlands, March 12, 2024 – Azafaros B.V. today announced the completion of its 12-week Phase 2 clinical study, RAINBOW(Phase 2 RAINBOW study NCT05758922).

The randomized, double-blind, placebo-controlled study, conducted in Brazil, involved 13 patients from the age of 12 years who are affected by GM2 gangliosidosis or Niemann-Pick disease type C (NPC). The aim of the RAINBOW study is to determine the safety, pharmacodynamics, and pharmacokinetics of two different doses of nizubaglustat in patients, in order to identify the target dosage for Azafaros’ planned Phase 3 pivotal studies.

“This is an important milestone because nizubaglustat is a first-in-class therapy that represents a leap forward from other agents.  It has a dual activity that targets with high potency some of the fundamental biology of the diseases and it might offer promise in pivotal studies.”

Prof. Dr. Roberto Giugliani, Chief of the Medical Genetics Clinical Research Group at the Hospital de Clinicas de Porto Alegre and Lead Principal Investigator for the study

The main part of the study is now complete and patients are entering the extension phase, in which all of them will be given the study drug. Top line results of the RAINBOW study are expected to be announced in Q2 2024 and presented to the scientific community later this year.

 “This is a major milestone on our journey to provide patients and their families with a potentially transformative treatment. With the RAINBOW data, we will be able to identify the optimal dose for a pediatric population in our Phase 3 efficacy study, which we plan to initiate as soon as the dose is determined, and the required approvals are in place. We are looking forward to presenting the data from RAINBOW to the scientific community. Azafaros is grateful to the patients and their families who made the decision to participate in this study.”

Chris Freitag, Chief Medical Officer at Azafaros