Vatiquinone Phase 3 Trial Initiated for Potential Treatment of Friedreich Ataxia 

The registration-directed phase 3 MOVE-FA study (NCT04577352) evaluating vatiquinone (PTC743; PTC Therapeutics, South Plainfield, NJ) in children and young adults with Friedreich ataxia (FA) has begun. Vatiquinone has been evaluated in a number of clinical studies and demonstrated an effect on mortality risk and a number of neurologicand neuromuscular symptoms.

Treatment with vatiquinone had a favorable safety profile and improved disease severity after 24 months of treatment relative to age- and stage-matched healthy individuals assessed by the FA rating scale (FARS) score in phase 2 FA ataxia by the Food and Drug Administration (FDA). The phase 3 MOVE-FA study has a target enrollment of 110 children and young adults with FA and will evaluate 18 months of treatment with vatiquinone vs placebo for improvement baseline on the modified FARS. Ability to walk and carry out activities of daily living with vatiquinone treatment vs placebo will also be assessed. 
 
"The initiation of another pivotal trial this year is a major milestone for PTC," said Stuart W. Peltz, PhD, chief executive officer, PTC Therapeutics, Inc. "This is the third clinical trial initiated this year based on compounds using our Bio-e platform. Vatiquinone is an exciting orally bioavailable small molecule that targets 15-lipoxygenase, the key enzyme regulating signaling pathways that control neuroinflammation and oxidative stress. Both pathways are important players in determining the pathology and disease progression seen in Friedreich ataxia patients. Previous clinical trial results give us confidence in vatiquinone's potential in treating patients living with this devastating disease."