MitoAction and UMDF Excited About Start of Edison Pharma EPI-743 Phase 2B Leigh Syndrome Clinical Trial

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Patient Advocacy Groups Say Clinical Trial Offers Hope to Patients

Edison Pharmaceuticals’ announcement today of the start of an EPI-743 Phase 2B Leigh Syndrome clinical trial has brought hope and optimism to MitoAction and the United Mitochondrial Disease Foundation (UMDF) about the future for patients and families affected by mitochondrial disease.

The clinical trial, entitled “A Phase 2B Randomized, Placebo-Controlled, Double-Blind Trial of EPI-743 in Children with Leigh Syndrome,” has already enrolled its first patient. According to Edison Pharmaceuticals, the placebo-controlled study will last six months and have a six-month extension phase during which all children will receive EPI-743. Edison is seeking children who are between one and 12 years of age who possess genetic confirmation of Leigh syndrome and meet certain disease severity criteria.    

“The start of this phase of the trial has been eagerly anticipated by patients and families with mitochondrial disease around the world,” said Cristy Balcells, RN., MSN, executive director of MitoAction. “As the first FDA clinical trial for mitochondrial disease, the EPI-743 Leigh syndrome clinical trial serves as a beacon of hope for our community." More information about the study can be found at

Four clinical trial sites have been selected in the United States: Lucile Packard Children’s Hospital at Stanford University Medical Center, Palo Alto, CA; Akron Children’s Hospital, Akron, OH; Seattle Children’s Hospital, Seattle, WA; and Texas Children’s Hospital - Baylor University, Houston, TX.

Charles A. Mohan, Jr., CEO and Executive Director of the UMDF, shares Balcells hope for the patient community. “Dr. Guy Miller and his team at Edison are offering our patients and families a potential lifeline,” Mohan said. “While the current trial is seeking patients with Leigh syndrome, we can only hope that EPI-743 starts us all down the road towards additional treatments and potential cures for mitochondrial disease.”

Both Mohan and Balcells strongly encourage patients around the world to join the North American Mitochondrial Disease Consortium (NAMDC) patient registry. As protocols become available for additional trials for EPI-743, or other new treatments, those who are on the registry are likely to be the first to be able to enter the trials. Patient databases and registries follow strict patient privacy guidelines and are offered at no cost to the patient. Likewise, for patients who qualify and enroll in an EPI-743 trial, there is no cost to the patient. Both UMDF and MitoAction have information about the NAMDC on their respective websites. “It is our collective hope that by galvanizing the mitochondrial disease community, we can assist in enrollment of at least 30 children with Leigh syndrome by January 2013,” Balcells said. “UMDF and MitoAction are working to educate the entire patient community, because we remain optimistic that additional trials and options for more patients will become available in late 2013,” Mohan said.

About MitoAction:
Based in Massachusetts, MitoAction's mission is to improve quality of life for all who are affected by mitochondrial disorders through support, education and advocacy initiatives. MitoAction's vision is to create a community of support that reaches every child, adult and caregiver affected by a mitochondrial disease. More information about MitoAction can be obtained at

About the UMDF:
Based in Pennsylvania, the UMDF works to promote research and education for the diagnosis, treatment and cure of mitochondrial diseases and to provide support for affected individuals and families. More information about UMDF can be obtained at

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Cliff Gorski
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