Edison Pharma Begins EPI-743 Phase 2 Trial For Pearson Syndrome

Edison Pharmaceuticals announced that it has started the Phase II study of EPI-743 (vincerinone) in pediatric patients with Pearson Syndrome. The U.S. Food and Drug Administration (FDA) Office of Hematology and Oncology Products has also granted approval for the drug’s Investigative New Drug (IND) application.

The trial entitled "A Phase 2 Safety and Efficacy Study of EPI-743 (Vincerinone) in Children with Pearson Syndrome" is a subject-controlled 12 month study in which all involved patients will receive EPI-743. The study’s primary endpoint is the incidence of episodes of sepsis, metabolic crisis and hepatic failure. Secondary endpoints of the study include avoidance of transfusion and other disease-relevant endocrine and neurological outcome evaluations. The trial is a single arm design given the rarity of Pearson Syndrome and is being conducted at multiple sites around the world.

Pearson Syndrome is estimated to affect less than one in one million people. The disease is typically diagnosed in infancy and possibly in neonates. Pearson Syndrome manifests in transfusion-dependent anemia, neutropenia, and pancreatic dysfunction in infants. It is known to be an ultra-rare, fatal mitochondrial disease resulting from mitochondrial deletion. There is currently no treatment for Pearson Syndrome and most afflicted patients die in infancy or early childhood secondary to metabolic disorders or infections. Those who survive past age three develop a neurological syndrome resembling Kearn-Sayre syndrome, characterized by ophthalmologic and neuromuscular impairments.

Mathew Klein, 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.”

EPI-743 is an orally bioavailable small molecule under development for the treatment of pediatric and adult patients with inherited mitochondrial diseases. The drug belongs to the para-benzoquinine class of drugs and uses a redox-based mechanism to augment endogenous glutathione biosynthesis essential to oxidative stress control.