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

Posted: 13.03.24

Major milestone reached in bringing new treatment options to patients and their families.

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.

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.

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, said: “This is an important
milestone because nizubaglustat has a dual mode of action that represents a leap forward from other
agents. This dual activity targets some of the fundamental biology of the diseases with high potency
and it might offer promise in pivotal studies.”

Chris Freitag, Chief Medical Officer at Azafaros, said: “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

About nizubaglustat
Nizubaglustat is a small molecule, orally available and brain penetrant azasugar with a unique dual
mode of action, developed as a potential treatment for rare lysosomal storage disorders with
neurological involvement, including GM1 and GM2 gangliosidoses and Niemann-Pick disease type C

Nizubaglustat has received the following designations and support:
United States Food and Drug Administration (FDA)
Rare Pediatric Disease Designations (RPDD) for the treatment of GM1 and GM2 gangliosidoses and
Orphan Drug Designations (ODD) for GM2 gangliosidosis (Sandhoff and Tay-Sachs Diseases) and NPC.
Fast Track Designation and IND clearance for GM1 and GM2 gangliosidoses.
European Medicines Agency (EMA)
Orphan Medicinal Product Designation (OMPD) for the treatment of GM2 gangliosidosis.
UK Medicines and Healthcare Products Regulatory Agency (MHRA)
Innovation Passport for the treatment of GM1 and GM2 gangliosidoses.

About GM1 and GM2 Gangliosidoses
GM1 gangliosidosis and GM2 gangliosidosis (Tay-Sachs and Sandhoff diseases) are lysosomal storage
disorders caused by the accumulation of GM1 or GM2 gangliosides respectively, in the central nervous
system (CNS), resulting in progressive and severe neurological impairment and early death. These
diseases mostly affect infants and children, and no disease-modifying treatments are currently

About Niemann-Pick Disease Type C (NPC)
Niemann-Pick disease type C (NPC) is a progressive, life-limiting neurological lysosomal storage
disorder caused by mutations in the NPC1 or NPC2 gene and aberrant endosomal-lysosomal
trafficking, leading to the accumulation of various lipids, including gangliosides in the CNS. The onset
of disease can happen throughout the lifespan of an affected individual, from prenatal life through

About Azafaros
Azafaros is a clinical-stage company founded in 2018 with a deep understanding of rare genetic
disease mechanisms, a compound library from Leiden University, and led by a team of highly
experienced industry experts. Azafaros aims to build a pipeline of disease-modifying therapeutics to
offer new treatment options to patients and their families. By applying its knowledge, network and
courage, the Azafaros team challenges traditional development pathways to rapidly bring new drugs
to the rare disease patients who need them. Azafaros is supported by a syndicate of leading Dutch
and Swiss investors including Forbion, BioGeneration Ventures, BioMedPartners, Asahi Kasei Pharma
Ventures, and Schroders Capital.

For further information:
Azafaros B.V.

ICR Consilium
Amber Fennell, Ashley Tapp
+44 (0)20 3709 5700