Azafaros Completes Phase 2 RAINBOW Trial of Nizubaglustat for Rare Diseases

Azafaros, a clinical-stage company, has achieved a significant advancement in its research by completing the 12-week Phase 2 RAINBOW study. The study, which was a randomized, double-blind, and placebo-controlled trial, took place in Brazil and included 13 patients, aged 12 and above, suffering from GM2 gangliosidosis or Niemann-Pick disease type C (NPC). The primary objective was to evaluate the safety, pharmacodynamics, and pharmacokinetics of two dosages of nizubaglustat, a drug developed by Azafaros, to determine the optimal dosage for future Phase 3 studies.

The study has now reached its main conclusion, with participants moving into an extension phase where they will receive the study medication. It is anticipated that the preliminary results will be disclosed in the second quarter of 2024 and shared with the scientific community later in the year.

Prof. Dr. Roberto Giugliani, the Lead Principal Investigator and Chief of the Medical Genetics Clinical Research Group at the Hospital de Clinicas de Porto Alegre, highlighted the importance of the study, noting that nizubaglustat's dual mode of action is a significant advancement over other treatments. This unique characteristic targets the fundamental biological aspects of the diseases with high potency, offering hope for the pivotal studies to come.

Chris Freitag, Azafaros' Chief Medical Officer, expressed that this milestone is a crucial step towards offering a potentially transformative treatment to patients and their families. With the data from RAINBOW, the company plans to identify the best dose for pediatric patients in Phase 3 efficacy studies. Azafaros intends to initiate these studies as soon as the dosage is confirmed and the necessary approvals are secured. The company is eager to present the RAINBOW data to the scientific community and is grateful to the participants and their families for their involvement.

Nizubaglustat is a novel azasugar with a unique dual action mechanism, designed to treat rare lysosomal storage disorders with neurological involvement such as GM1 and GM2 gangliosidoses and NPC. It has received various designations and support from regulatory bodies in the United States and Europe, including Rare Pediatric Disease Designations, Orphan Drug Designations, and Orphan Medicinal Product Designation.

GM1 and GM2 gangliosidoses are severe lysosomal storage disorders that affect infants and children, leading to progressive neurological impairment and often resulting in early death. Niemann-Pick disease type C is a progressive neurological disorder caused by genetic mutations, impacting lipid trafficking and leading to the accumulation of lipids in the CNS.

Azafaros was founded in 2018 with a focus on understanding rare genetic diseases and developing a pipeline of therapeutics. The company is backed by a consortium of Dutch and Swiss investors and is committed to challenging traditional development pathways to expedite the delivery of new drugs to patients in need.