Minoryx Therapeutics receives Orphan Drug Designation from the European Commission for leriglitazone in the treatment of Friedreich’s Ataxia

Mataró, Barcelona, Spain and Charleroi, Belgium, November 12, 2019 – Minoryx Therapeutics, a company that specializes in the development of innovative treatments for orphan Central Nervous System (CNS) diseases, today announces that its lead drug candidate, leriglitazone (MIN-102), has been granted Orphan Drug Designation by the European Commission in the treatment of Friedreich’s Ataxia.

Leriglitazone (MIN-102) is a novel, brain penetrant, orally bioavailable and selective PPARγ agonist that engages the target receptor within the central nervous system. The disease-modifying potential and unique mode-of-action of leriglitazone have been demonstrated in multiple preclinical CNS disease models showing that it has an anti-oxidant, anti-inflammatory and neuroprotective effect. Leriglitazone improves mitochondrial function and biogenesis, promotes remyelination, ameliorates lipid metabolism and delays the progression of neurological disability. Leriglitazone is currently in late-stage clinical development in adrenomyeloneuropathy (AMN) and Friedreich’s Ataxia (FRDA).

“We are very pleased that, following the recent FDA Orphan Drug Designation for leriglitazone in Friedreich’s Ataxia, the European Commission has also granted Orphan Drug Status, further demonstrating the potential of this novel drug candidate and our commitment to changing the lives of patients suffering from severe orphan diseases,” said Marc Martinell, CEO of Minoryx. “Leriglitazone is our lead drug candidate currently in late-stage clinical development in a number of life-threatening orphan CNS diseases. Patient enrollment has been completed in both the AMN and the FRDA studies and we are on track to present our results by the end of 2020.”

Minoryx Therapeutics receives Orphan Drug Designation from the European Commission for leriglitazone in the treatment of Friedreich’s Ataxia