Edison Pharmaceuticals today announced the initiation of a phase 2B study entitled, "Safety and Efficacy Study of EPI-743 on Visual Function in Patients with Friedreich's Ataxia."
The trial is a placebo-controlled study lasting six months, followed by an extension phase in which all subjects will receive EPI-743. Subjects must be between 18 and 45 years of age, possess genetic confirmation of Friedreich's ataxia, and meet certain disease severity criteria. The primary endpoint of the trial is visual function, with secondary endpoints including neurological and neuromuscular function and disease-relevant biomarkers. More information on study specifics is available on ClinicalTrials.gov.
This trial is being conducted with the assistance of investigators from the Friedreich's Ataxia Research Alliance (FARA) Clinical Research Network and FARA's Patient Registry. "FARA is excited about the commencement of the EPI-743 Friedreich's ataxia clinical trial, and is working closely with Edison Pharmaceuticals and Clinical Research Network investigators to expedite enrollment," stated FARA President Ronald Bartek . "This trial represents the importance of public-private partnership in drug development and the culmination of FARA-sponsored translational research, initiated with both FARA and National Institutes of Health (NIH) support."
Three clinical trial sites have been selected in the United States: University of South Florida – Tampa, Florida; Children's Hospital of Philadelphia– Pennsylvania; and University of California– Los Angeles, California. Enrollment has been initiated at University of South Florida.
"Given the encouraging clinical findings reported to date for EPI-743 in mitochondrial disease, we are eager to determine whether these results are distensible to Friedreich's ataxia," stated FARA Clinical Research Network investigator Theresa A. Zesiewicz, MD, FAAN, Professor of Neurology, Director of the University of South Florida Ataxia Research Center and one of the trial's principal investigators.
Edison Pharmaceuticals, Inc.