Stealth’s Elamipretide in Clinical Trial for Leber’s Neuropathy

Stealth BioTherapeutics, a biopharma that focuses on drugs for mitochondrial dysfunction, recently announced the start of a Phase 2 clinical trial exploring elamipretide (previously known as Ocuvia) in the form of eye drops for the treatment of Leber’s Hereditary Optic Neuropathy.

Stealth’s lead candidate, elamipretide, works by preserving energy processes and restoring normal energy production in mitochondria, while decreasing oxidative stress.

This rare condition, affecting only 35,000 patients worldwide, is caused by an inherited mitochondrial dysfunction in retinal ganglion cells — neurons present on the inner surface of the eye’s retina. The disrupted energy-producing processes in mitochondria lead to permanent damage of the cells, causing vision loss and blindness in a patient group dominated by men who are affected by the disease in late adolescence or early adulthood.

“Elamipretide offers hope for patients suffering from this rare ophthalmic disease, for which there is no FDA-approved treatment,” said study primary investigator Alfredo Sadun from the UCLA Doheny Eye Institute. “The loss of vision can be sudden and devastating, often occurring in both eyes within a few weeks’ time. Unfortunately, the resulting vision loss is usually permanent, underscoring the desperate need for effective treatment options.”

The study, called ReSIGHT, will be a prospective double-blind study where elamipretide eye drops will be compared to placebo in 12 Leber’s patients with a particular mitochondrial DNA mutation, termed m.11778G>A. Patients will receive the drug twice daily for 12 weeks.

Patients included in the study had to be 18-50 years old when they started experiencing vision loss in the second eye. The study also requires that this vision loss occurred no more than 10 years before the start of the study.

Participants will be their own controls, as they will be randomized to receive elamipretide in either one or both eyes. The study will mainly focus on safety and tolerability aspects, but will also include measurements of change in visual function and response of retinal ganglion cells as secondary outcomes.

“As a community, we are eager for treatment options for Leber’s Hereditary Optic Neuropathy, as the disease has a significant impact on patients’ lives. The initiation of this trial presents an important step forward for the Leber’s Hereditary Optic Neuropathy community,” said leading Leber’s neuropathy advocate Lissa Poincenot.

“ReSIGHT is an important trial to further both our orphan development program and our understanding of elamipretide’s potential to treat diseases affecting the back of the eye,” said Stealth BioTherapeutics CEO Reenie McCarthy. “We look forward to findings from this trial, which are anticipated in the second half of 2017.”