Stealth BioTherapeutics, a biopharmaceutical firm advancing drug candidates with the potential to treat mitochondrial dysfunction, recently provided an update on both Bendavia and Ocuvia, and also announced the company’s future clinical plans for its orphan mitochondrial disease program at the 2015 United Mitochondrial Disease Foundation (UMDF) National Symposium in Washington, D.C. The UMDF is a top patient advocacy organization that assists those with inherited mitochondrial diseases and whose focus is to promote patient-oriented research and education as well as support its members and families. Inherited mitochondrial diseases include a diverse group of rare genetic disorders that result from mitochondrial dysfunction; such impairments severely affect patients’ well-being and quality of life.
Stealth updated UMDF participants on the status of the company’s MMPOWER study for Mitochondrial Myopathy (MM) and recently announced intentions to initiate a larger MM patient trial. The MMPOWER study is assessing Bendavia as an MM therapy in those with genetic mitochondrial illnesses.
“The current treatment options for mitochondrial diseases are limited to vitamins and supplements. Without investigational drugs such as Bendavia, there would be no prospect for effective therapies to help the children and adults we represent,” noted Charles Mohan, who is the UMDF’s Executive Director.
Stealth also updated conference participants on the company’s ReSIGHT trial for Leber’s Hereditary Optic Neuropathy (LHON) which is a rare genetic mitochondrial disease and the most frequent inherited optic neuropathy that causes loss of vision in young men. Lissa Poincenot, a patient advocate, noted: “We are tremendously excited for Ocuvia’s potential to treat devastating optic neuropathies and improve the lives of our patients.”
Bendavia and Ocuvia target inner mitochondrial membrane dysfunctions and treat both common and rare types of diseases such as orphan mitochondrial diseases, cardio-renal diseases and ophthalmic disorders. Bendavia and Ocuvia act by changing the disease progression through restoring cellular function and energetics. These investigational drugs are well-tolerated and appear thus far to be effective.
Travis Wilson, the company’s CEO, said: “There are more than 270 orphan mitochondrial diseases and no FDA-approved treatments. The impact of these diseases on patient quality of life is very real. By focusing on mitochondrial myopathy and inherited optic neuropathies with Bendavia and Ocuvia, we are addressing important orphan disorders with a mitochondrial genetic basis. We are committed to our rare mitochondrial disease program in hopes of providing patients with the first FDA-approved therapy.”