MIN-102 (leriglitazone) is an orally bioavailable selective PPAR gamma agonist that is one of the metabolites of pioglitazone and has been shown to have improved access to the central nervous system and better safety profile. Several research studies in both animal and cell models have identified that the peroxisome-proliferator activator receptor gamma (PPARγ)/PPARγ coactivator 1 alpha (Pgc1a) pathway is dysregulated when there is frataxin deficiency thus making this pathway a potential therapeutic target for FA.

Minoryx Therapeutics conducted a clinical trial that was a multicenter, double-blind and placebo-controlled study with leriglitazone. 39 Friedreich's ataxia patients were enrolled, with 32 completing the study. Today they announced that topline results show improvement of relevant disease biomarkers in the brain and spinal cord in this proof of concept Phase 2 study. Treatment with leriglitazone resulted in PPARg engagement, within the target range, in all patients as assessed by the relevant biomarker (adiponectin). Results of the trial show modulation of the Frataxin pathway and restoration of the bioenergetics deficits by leriglitazone in Friedreich's ataxia patients. They also reported that leriglitazone was well tolerated with some anticipated adverse events (side-effects) known to drugs with this mechanism of action.

Minoryx indicates that based on these results they are advancing the program and will be meeting with regulatory agencies, FDA and EMA, and planning a confirmatory study in FA.

Thank you to all of the individuals with FA who volunteered, investigators, and study sites for your participation and conduct of this important research trial and to Minoryx for their commitment to developing treatments for FA and their efficiency and transparency in sharing results from the trial. In the coming months, as Minoryx Therapeutics determines the next steps for the clinical development of this program, we will share that information with the community.

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