Azafaros’ Phase 2 RAINBOW study, evaluating nizubaglustat in GM2 and NPC patients, is now fully enrolled

• Topline data from the study, expected for mid-2024, will support two pivotal Phase 3 trials in GM1 and GM2 Gangliosidoses and Niemann-Pick disease type C
• Number of patients recruited into the RAINBOW trial exceeded the target
• The aim of the RAINBOW study is to determine the pharmacodynamics and pharmacokinetics of two different doses of nizubaglustat in patients

Leiden, The Netherlands, 11 December 2023 – Azafaros B.V. today announces that it has exceeded the target number of patients in the enrolment of its Phase 2 clinical trial, RAINBOW (NCT05758922), treating patients with severe rare metabolic diseases.

The success in enrollment is a major milestone for Azafaros’ lead asset, nizubaglustat, which is being developed to treat patients with GM1 and GM2 gangliosidoses and Niemann-Pick disease type C (NPC).

The 12-weeks study is a randomised, double-blind, placebo-controlled, multi-center trial which evaluates the safety, tolerability, pharmacokinetics, and pharmacodynamics across two doses of nizubaglustat in patients with GM2 gangliosidosis and NPC.

The trial is being conducted at sites across Brazil, with safety, pharmacokinetic and pharmacodynamic data from ongoing patient follow-ups expected in mid-2024.

The aim of the RAINBOW study is to determine the 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.

Professor Roberto Giugliani, MD, Chief of the Medical Genetics Service at the Hospital Clinicas de Porto Alegre in Brazil and Lead Principal Investigator for the RAINBOW study, said:

“We are pleased to be involved in this important study. Patients with GM1 and GM2 gangliosidoses currently have no approved specific therapeutic options, and improved therapies for NPC patients will be welcomed by clinicians, the patients, and their families.”

Dr Daniel Almeida do Valle is a paediatric neurologist at the Hospital Pequeno Príncipe in Curitiba, Brazil, who enrolled a considerable number of patients onto the RAINBOW study.

“It is a great achievement in a clinical trial to exceed the target number of patients, especially with such rare diseases as GM2 and NPC,” he commented.

“RAINBOW is a very important study which will enable Azafaros to find the correct doses which can treat patients in the future,” he added. “It will increase our knowledge for the future development of nizubaglustat, and we are truly grateful for the patients’ participation in the study.”

Dr Stefano Portolano, CEO at Azafaros, said: “Exceeding the recruitment goal is an important step forward in our ambition to provide a new therapeutic solution for these rare lysosomal storage disorders, where patients have no or limited treatment options. Our team has worked tirelessly to reach this point and the focus is now on preparing the next steps for two Phase 3 studies. We would like to thank all patients, their families, patient organisations and clinicians for their participation and support in this important research.”