Edison Pharmaceuticals is one of a new group of pharmaceutical companies developing drug treatments for inherited mitochondrial diseases such as Leigh syndrome, Friederich’s ataxia, Leber’s hereditary optic neuropathy (LHON), and acquired mitochondrial dysfunction in Parkinson’s disease and ALS.
Inherited Mitochondrial Disease
Inherited mitochondrial diseases are genetic disorders caused by defects in the cells’ ability to make and regulate energy. Due to the role mitochondria plays in the generation and regulation of energy metabolism, mitochondrial diseases affect all organ systems. Most often, these conditions cause impairments in the neurological, cardiovascular, endocrine, hepatic, muscular, and renal systems and can lead to death.
Treating Mitochondrial Disease with Redox Therapy
Edison Pharmaceuticals’ research efforts are focused on the control of cellular energy and metabolism through a class of drugs called “redox” drugs. Mitochondrial diseases are caused by defects in DNA electron transfer. Edison is targeting treatments that regulate the flow of electrons, essential to the generation and regulation of energy. Beyond mitochondrial disease, Edison plans to apply this same knowledge to the development of treatments for other neurological diseases with shared disease mechanisms.
Edison’s Drug Pipeline
EPI-743, also known as Vincerinone, is a drug candidate in clinical development for inherited mitochondrial diseases. EPI-743 is a type of CoEnzyme Q10, which is found naturally in our cells and is essential for proper energy production within the mitochondria. EPI-743 works like CoEnzyme Q10, as an antioxidant, carrying electrons along the energy chain to make energy in the form of ATP. EPI-743 is reported to be more potent and more highly absorbed than naturally occurring CoEnzyme Q10. The FDA granted Orphan Drug status to EPI-743 for the treatment of Leigh syndrome in June 2014.
EPI-589, also known as (R)-troloxamide Quinone, is claimed to have significant effects on modifying Glutathione (GSH) levels.GSH is found in nearly all cells and plays an important role, together with other antioxidants, in maintaining the effective clearing of free radicals.
Friedreich’s ataxia – A Phase 2B Double-Blind, Placebo-Controlled, Clinical Trial of EPI-743 in Friedreich’s Ataxia
Leigh syndrome – A Phase 2B Randomized, Placebo-Controlled, Double-Blind Clinical Trial of EPI-743 in Children with Leigh Syndrome
Rett syndrome – A Placebo Controlled Trial of EPI-743 on Children with Rett Syndrome
Cobalamin C defect – A Placebo Controlled Trial of EPI-743 on Neurologic and Visual Function in Patients with Cobalamin C Defect
NIH Undiagnosed Diseases of Redox and Metabolism – A Placebo Controlled Trial of EPI-743 on Diseases of Mitochondrial or Metabolic Defects
MELAS – Safety and Efficacy of EPI-743 in Patients with MELAS
Parkinson’s disease – Effects of EPI-743 on Visual and Neurological Function in Patients with Parkinson’s Disease
ALS – A Phase 2A Safety and Biomarker Study of EPI-589 in Subjects With Amyotrophic Lateral Sclerosis
Edison Pharmaceuticals believes unraveling the science of mitochondrial disease holds promise for treating a family of devastating diseases. By gaining insight into the basic underpinninings of how our bodies make and regulate energy metabolism, Edison believes that the emerging field of mitochondrial medicines will provide key insights into the biology of aging.