RAINBOW study design enables rapid advancement into the company’s planned Phase III efficacy trial

Leiden, The Netherlands, May 22- 2023 – Azafaros B.V. today announced that the first US site for the Phase II RAINBOW study, led by Dr Marc Patterson of the Mayo Clinic in Minnesota, has received Institutional Review Board approval. The clinical trial (NCT05758922) is being conducted in Brazil and the US and will evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics across two doses of its lead asset, AZ-3102, in patients with GM2 gangliosidosis and Niemann-Pick disease type C (NP-C).
AZ-3102 is a novel, orally available, brain-penetrant azasugar, engineered to have a unique, dual mode of action by inhibiting two key enzymes involved in the metabolism of glycosphingolipids. Azafaros is developing the compound as a potentially disease-modifying treatment in severe metabolic disorders including GM1 and GM2 gangliosidoses and NP-C.


The Phase 2 RAINBOW study is a randomized, double-blind, placebo-controlled, multicenter, 12-week trial assessing the safety, tolerability, pharmacokinetics, and pharmacodynamics of AZ-3102 in patients with GM2 gangliosidosis and NP-C. The study will enroll 12 patients in total (6 with GM2 and 6 with NP-C), aged 12 to 20 years and able to swallow, each of whom will receive either the low dose, high dose, or a placebo. Patients who complete the 12-week study period will be offered an extension, if approved by the country’s health authorities. The aim of the short study is to determine the clearance of AZ-3102 from the body and the effect of two different doses in patients to identify the target dose for Azafaros’ planned Phase 3 pivotal study.

About AZ-3102
AZ-3102 is an orally available 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 (NP-C). For more information www.azafaros.com
At the start of 2023, AZ-3102 received three Rare Pediatric Disease Designations (RPDD) from the United States Food and Drug Administration (FDA) for the treatment of GM1 and GM2 gangliosidoses, and NP-C. The asset also received Orphan Medicinal Product Designation (OMPD) from the European Medicines Agency (EMA) for the treatment of GM2 gangliosidosis, as well as an Innovation Passport from the UK Medicines and Healthcare Products Regulatory Agency (MHRA) for the treatment of GM1 and GM2 gangliosidoses.

In 2022, the compound received Fast Track Designation for GM1 and GM2 gangliosidoses as well as NP-C and Orphan Drug Designations (ODD) for GM2 gangliosidosis (Sandhoff and Tay-Sachs Diseases) and NP-C from the FDA.

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 available.

About Niemann-Pick Disease Type C (NP-C)
Niemann-Pick disease type C (NP-C) 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 happens throughout the lifespan of an affected individual, from prenatal life through adulthood. The mainstay of therapy is symptom management.

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 patients and their families new treatment options. The company’s lead clinical-stage program is AZ-3102, a small molecule azasugar, orally available and brain penetrant, with the potential to treat GM1 gangliosidosis and GM2 gangliosidosis (Tay-Sachs and Sandhoff diseases) and Niemann Pick disease type C (NP-C). By applying its know-how, 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, and Schroders Capital.

For further information:
Azafaros B.V.
Email: [email protected] www.azafaros.com