Preclinical data highlights potential therapeutic action and protection against neurodegeneration

Mataró, Barcelona, Spain and Gosselies, Belgium, November 16, 2020 - Minoryx Therapeutics, a Phase 3 clinical stage biotech company focused on the development of differentiating treatment options in orphan central nervous system (CNS) disorders with high unmet need, today announces the publication in ‘Neurobiology of disease’ of preclinical data on its lead compound, leriglitazone, showing potential therapeutic action and protection against neurodegeneration, in particular against Friedreich’s Ataxia (FRDA).

The preclinical data, published on November 7, 2020, supports the on-going clinical development in treating FRDA. The results show that the company’s lead compound, leriglitazone, improves impairments that are derived from frataxin loss - the genetic deficiency that causes FRDA.

As key highlights, the publication shows than in FRDA models, leriglitazone:

• Increases frataxin levels and mitochondrial markers
• Protects from neurodegeneration by improving mitochondrial function
• Prevents the formation of lipid droplets in frataxin-deficient cardiomyocytes
• Rescues the motor function deficit in transgenic mice
• Emerges as a potential therapeutic agent for Friedreich’s Ataxia

“At Minoryx, we’re committed to making new therapies available for patients suffering from severe, orphan diseases. Leriglitazone is currently in clinical development for the treatment of a range of orphan central nervous system (CNS) disorders and is currently completing a pivotal phase 2/3 clinical trial in Adrenomyeloneuropathy (AMN) and a phase 2 proof of concept trial in Friedreich’s Ataxia,” said Dr. Marc Martinell, co-founder and CEO of Minoryx. “This preclinical data provides further evidence of the potential for therapeutic action and protection against neurodegeneration in Friedreich’s Ataxia.”