Azafaros has enrolled the first participant in its Phase II RAINBOW trial of AZ-3102 in GM2 gangliosidosis and Niemann-Pick disease type C (NP-C) patients.

Being conducted in the US and Brazil, the 12-week, multicentre, randomised, placebo-controlled, double-blind trial has been designed for evaluating the pharmacokinetics, pharmacodynamics, tolerability, and safety of AZ-3102 across two doses.

A total of 12 participants, aged between 12 and 20 years, including six with NP-C and six with GM2, are planned to be enrolled in the trial.

In the trial, each participant will receive either a low dose, a high dose, or a placebo.

The company noted that the participants who complete the 12-week trial period will have a chance to join a double-blind extension trial, subject to approval from the health authorities of the country.

The trial’s objective is to determine AZ-3102’s clearance from the body and the effect of two different therapy doses in patients for identifying the target dose for the company’s planned Phase III pivotal trials.

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Azafaros CEO Stefano Portolano 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 paediatric population in a Phase III efficacy study, which we plan to initiate in 2024.”

A new, orally available, brain-penetrant azasugar, AZ-3102 has a unique, dual mode of action.

It inhibits two key enzymes that are involved in the metabolism of glycosphingolipids.