"Azafaros Receives FDA's IND Clearance and Fast Track Designation for Lead Asset AZ-3102"
Read the press release from January 5, 2023 here:
Read the press release from January 9, 2023 here:
Received Investigational New Drug (IND) clearance from the FDA to conduct a clinical Phase 2 trial for its lead asset, AZ-3102, for the treatment of GM2 and Niemann-Pick disease type C (NP-C).
Azafaros’s RAINBOW Phase 2 trial is a multinational, double-blind, placebo-controlled, parallel-group trial investigating the safety, tolerability, and pharmacokinetics of a once-daily oral administration of AZ-3102 for 12 weeks in GM2 and NP-C patients between 12 and 20 years of age.
Professor Marc C. Patterson is the principal investigator for the U.S. part of the study.
To join our fight, as well as fund vital research for a cure, support the Australian NPC Disease Foundation in its goals of "Research. Cure. Persevere."
Ways to support NP-C in Australia: