Edison Pharmaceuticals Begins EPI-743 Trial For Pearson Syndrome
By Cyndi Root
Edison Pharmaceuticals announced in a press release that the FDA has approved a Phase II study of EPI-743. The study titled, “A Phase 2 Safety and Efficacy Study of EPI-743 (vincerinone) in Children with Pearson Syndrome” will evaluate the investigational drug for a rare mitochondrial disease. The FDA recently granted Fast Track status for EPI-743 in Friedreich’s ataxia. Mathew Klein, MD, FACS, Chief Medical Officer of Edison Pharmaceuticals, said, “Pearson syndrome is a very rare mitochondrial disease with a devastating clinical outcome. We are working with our clinical investigator team worldwide to accelerate enrollment in this trial and to systematically explore whether EPI-743 can offer benefit for this patient population.”
Pearson Syndrome
Pearson syndrome is a rare and fatal mitochondrial disease wherein mitochondrial genes are deleted. Most patients die in the first few years of life and suffer from macrocytic anemia, neutropenia, and thrombocytopenia during their short lives. Those few that survive develop neurological, muscular, and ophthalmologic disorders as in Kearn-Sayre syndrome. The prevalence of Pearson Syndrome is estimated at less than 1:1,000,000. No comprehensive therapy is available, as the disease course is highly variable and affects multi-systems. Therapy consists of treating intermittent crises and making the patient as comfortable as possible.
EPI-743
Edison is developing EPI-743 for a wide range of mitochondrial diseases such as Leigh syndrome and Cobalamin C defect. In February, Edison formed a $4.3 billion partnership with Dainippon Sumitomo Pharma Co., Ltd. of Japan to develop agents like EPI-743. The partnership is supported by the mitochondrial disease community including the Mitochondrial Disease Action Committee (MitoAction) and the United Mitochondrial Disease Foundation (UMDF).
EPI-743 is a small molecule drug in the class of drugs known as para-benzoquinones. EPI-743 controls oxidative stress by encouraging synthesis of glutathione. The Phase II study was approved by the FDA’s Office of Hematology and Oncology Products, as Edison submitted the application to that department. Edison believes that Pearson syndrome has significant hematological implications and, therefore, that FDA department is best positioned to lend expertise.
Due to the rarity of the condition, participants are being recruited for the Phase II trial on an international basis. Trial participants will receive EPI-743 for 12 months. Investigators are looking for evidence of metabolic crises, sepsis, and liver failure.