PTC Therapeutics launches vatiquinone late-stage study in Friedreich ataxia

 

• PTC Therapeutics (PTCT -1.6%) has initiated Phase 3 MOVE-FA study evaluating vatiquinone (PTC743) in children and young adults with Friedreich ataxia, a genetic, neuro-degenerative movement disorder, typically diagnosed in childhood or adolescence.
• Vatiquinone has been granted FDA Orphan Drug and Fast Track Designation for Friedreich ataxia.
• The trial will evaluate vatiquinone versus placebo in ~110 children and young adults, with primary endpoint of the change from baseline in the modified Friedreich ataxia rating scale, with key secondary endpoints assessing ambulation (ability to walk without the need for any kind of assistance) and activities of daily living.
• Vatiquinone is an investigational small molecule that inhibits 15-Lipoxygenase, an enzyme that is a key regulator of the oxidative stress and inflammation response pathways that underpin neurological disease pathology.
• Last month, the company launched study of vatiquinone for mitochondrial epilepsy.
• https://seekingalpha.com/news/3639969-ptc-therapeutics-launches-vatiquinone-late-stage-study-in-friedreich-ataxia