Edison Pharmaceuticals has commenced a Phase IIb clinical trial of EPI-743, an orally bioavailable small molecule being developed for the treatment of Friedreich's ataxia and other inherited mitochondrial diseases.
The double-blind trial, entitled Safety and Efficacy Study of EPI-743 on Visual Function in Patients with Friedreich's Ataxia, has enrolled its first subject.
This trial is being conducted in collaboration with the Friedreich's Ataxia Research Alliance (FARA) Clinical Research Network and FARA's Patient Registry.
FARA President Ronald Bartek said the organisation is working closely with Edison Pharmaceuticals and Clinical Research Network investigators to expedite enrolment.
"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," Bartek added.
The six-month placebo-controlled study is followed by an extension phase in which all subjects between 18 and 45 years of age will receive EPI-743.
Subjects must possess genetic confirmation of Friedreich's ataxia and should meet certain disease severity criteria.
Visual function is the primary endpoint, while secondary endpoints include neurological and neuromuscular function and disease-relevant biomarkers.
FARA Clinical Research Network investigator, the University of South Florida Ataxia Research Center director, professor of neurology and one of the trial's principal investigators Dr Theresa Zesiewicz said; "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."