Stealth BioTherapeutics Announces Positive Data on Mitochondria-based Therapy Ocuvia for Diabetic Macular Edema

Stealth BioTherapeutics, a privately held biopharmaceutical company committed to developing mitochondrial therapies to treat both common and rare diseases, recently announced data from its ReVIEW clinical trial of Ocuvia in patients with diabetic macular edema (DME).

The data was recently presented during Stealth’s second Mitochondrial Science and Medicine Series symposium, which took place in New York City and was focused on the role of mitochondria in ophthalmology.

ReVIEW, a clinical trial involving DME patients, assessed the efficacy, tolerability and safety of Ocuvia, a drug agent that aims to treat mitochondrial dysfunction. DME is caused by retinal vessel leakage and accumulation of macular fluid, ultimately leading to washed-out or blurry vision as well as frequent blindness.

The clinical trial examined a total of 15 patients with DME that received Ocuvia for a period of 28 days. Patients were subject to randomization to receive the drug 0.3- and 1%- in ascending doses. Results from the study showed that the drug met its primary efficacy and safety endpoints, with no observed decreases in visual acuity. The results also showed that Ocuvia was able to reduce the thickness in the central subfield by 32% and 27%, respectively, in both 0.3- and 1%- treated patient groups.

“Although ReVIEW was a safety and tolerability study, a reduction of macular edema in these patients demonstrates Ocuvia’s potential,” said Dr. Jeffrey Heier, a leading retinal specialist. “Ocuvia holds promise for patients to complement and improve retinal care.”

“Ocuvia may be the new paradigm for ophthalmology, reversing disease rather than just slowing angiogenesis,” commented Dr. Pravin Dugel, Chairman for the American Academy of Ophthalmology Subspecialty Day, describing Ocuvia’s clinical benefits and the role of mitochondria in eye diseases.

Duirng the symposium, Dr. Scott Cousins, an age-related macular degeneration (AMD) researcher and clinician, presented data on Ocuvia in patients with dry AMD.

Dr. Alfredo Sadun, a renowned neuro-ophthalmologist, also presented his insights regarding rare mitochondrial optic neuropathies common to more than 20 orphan inherited diseases. Stealth is initiating a clinical study in inherited optic neuropathies with its ReSIGHT trial for Leber’s Hereditary Optic Neuropathy, a rare genetic mitochondrial disease causing sudden and permanent loss of vision.

“ReVIEW’s results are encouraging and demonstrate the importance of mitochondria in debilitating diseases of the eye,” said Chief Executive Officer Travis Wilson. “We continue to broaden our ophthalmology program with Ocuvia, recruiting for the ReSIGHT study later this year, and initiating trials in rare anterior segment diseases and corneal dystrophies, both underserved patient populations with few treatment options.”

Mitochondrial disorders are believed to arise either as a result of inherited genetic defects or through damage caused by a variety of common diseases. In either case, the result is the creation of excessive oxidative stress and reduced energy production. Dysfunctional mitochondria are a key element in a variety of serious, debilitating diseases, both rare and common, and, as such, are a promising therapeutic target.