Minoryx Therapeutics, a company specializing in the development of innovative treatments for orphan central nervous system (CNS) diseases, today announces that it has completed recruitment in the FRAMES phase 2 clinical trial of its novel PPARγ agonist, leriglitazone (MIN-102), in patients with Friedreich’s Ataxia.

FRAMES is a multicenter, randomized, double-blind, placebo-controlled trial that will assess the efficacy and safety of leriglitazone in patients with Friedreich’s Ataxia. Recruitment of 39 patients in four European countries was completed in just four and a half months, well ahead of schedule. The patients, aged 12-60 years, will receive leriglitazone, administered once a day as an oral suspension, for a total of one year. The primary objective of the trial is to monitor the effect on disease progression. This will be measured through state-of-the art imaging of the spinal cord. Secondary objectives include safety and tolerability, effect on additional clinical measures, such as patient reported outcomes, functional disability scores and exploratory biomarkers.

“We are very pleased with the strong support from the medical community and patients who made it possible to fully recruit FRAMES ahead of schedule, demonstrating the need for novel disease-modifying treatment options for patients suffering from this life-threatening disease,” said Marc Martinell, CEO of Minoryx. “We look forward to completing treatment of all patients in this study and reporting on the data, which is expected by the end of 2020.”

View the full press release here

Posted on 08/10/19