" class="no-js "lang="en-US"> Azafaros Announces Study Evaluating GM2 & NP-C Patients
Sunday, May 12, 2024

Azafaros Announces Enrollment of First Patient in Phase 2 RAINBOW Study Evaluating AZ-3102 in GM2 and NP-C Patients

Azafaros has announced that the first patient has been enrolled in its Phase 2 RAINBOW study evaluating AZ-3102 in GM2 and NP-C Patients. The clinical trial 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. Enrollment of the first patient in the study is an important milestone for Azafaros in its mission to bring new treatment options to these patients and their families.

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, 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 a double-blind 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 studies.

Dr. Daniel Almeida do Valle, a Paediatric Neurologist at the Hospital Infantil Pequeno Príncipe in Curitiba, Brazil, where the first patient has been enrolled, said: “A childhood diagnosis of GM2 of NP-C is devastating for patients and their families. There is a great need to provide more treatment options to improve the lives of these patients. The safety and tolerability of AZ-3102 has already been seen in healthy volunteers and I am excited to be working with Azafaros to take AZ-3102 further with the Phase 2 RAINBOW trial. This will help us to further our clinical development of AZ-3102, which has great potential to become a treatment option for patients.”

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: “Children who suffer from GM2 and NP-C are currently dependent on limited and, in some cases, mostly supportive treatment options. AZ-3102 has the potential to truly impact the course of these devastating disorders. Based on the favorable safety and tolerability profile that Azafaros demonstrated in its Phase 1 healthy volunteer study, the Phase 2 RAINBOW trial will now examine these parameters in patients to identify the optimal dose for further clinical development.”

Stefano Portolano, Chief Executive Officer of Azafaros, said: “Today’s announcement demonstrates our commitment to address the significant unmet medical needs of GM2 and NP-C patients. The design of the RAINBOW study allows us to move quickly and advance AZ-3102 as a potentially transformative treatment for these patients by defining the optimal dose for a pediatric population in a Phase 3 efficacy study, which we plan to initiate in 2024.”


  1. Eloxx Pharmaceuticals Announces Final Data Assessment from Phase 2 Combination Clinical Trial of ELX-02 in Class 1 Cystic Fibrosis Patients Read more
  2. Verge Genomics Announces Positive Safety and Tolerability Data from the Phase 1 Clinical Trial of VRG50635, a Potential Best-in-Class Therapeutic for All Forms of ALS Read more
  3. DEM BioPharma Appoints Wendy Young, Ph.D., to Scientific Advisory Board Read more
  4. Confo Therapeutics Enters into Research Collaboration for GPCR-Targeting Antibody Discovery with AbCellera Read more
  5. Vyriad Announces Expansion of T-Cell Lymphoma Trial at Mayo Clinic Read more