Stealth BioTherapeutics Initiates Phase 2 Study of Elamipretide in Leber’s Hereditary Optic Neuropathy

Trial augments ongoing clinical development program for rare genetic mitochondrial diseases

BOSTON – June 01, 2016 – Stealth BioTherapeutics (Stealth), a clinical-stage biopharmaceutical company developing investigational drugs for treating mitochondrial dysfunction, today announced the initiation of ReSIGHT, a phase 2 clinical study evaluating the topical eye drop delivery of elamipretide (formerly known as Ocuvia) for the treatment of patients with Leber’s Hereditary Optic Neuropathy (LHON).

Affecting approximately 35,000 people worldwide, LHON is an inherited mitochondrial disease that causes central vision loss stemming from the damage to retinal ganglion cells due to dysfunction of energy-producing complexes in the mitochondria. The disease, which can lead to legal blindness, affects more men than women and often strikes patients in their late teens and early 20s.

“Elamipretide offers hope for patients suffering from this rare ophthalmic disease, for which there is no FDA-approved treatment. 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,” said Alfredo Sadun, M.D., Ph.D., UCLA Doheny Eye Institute, the primary investigator for the study.

The phase 2 prospective, double-blinded, vehicle-controlled ReSIGHT study will evaluate the topical eye drop delivery of elamipretide in 12 LHON subjects who have a specific genetic mitochondrial DNA mutation (m.11778G>A), were 18-50 years of age at the time of vision loss in the second eye, and experienced this vision loss within the last 10 years. Participants will be randomized to receive one drop of elamipretide twice daily for 12 weeks in either one or both eyes. The study’s primary endpoints are safety and tolerability. Secondary endpoints include change in visual function and electrical response of retinal ganglion cells.

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

“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,” Stealth BioTherapeutics Chief Executive Officer Reenie McCarthy said. “We look forward to findings from this trial, which are anticipated in the second half of 2017.”

For additional information on this study or elamipretide, please refer to Stealth’s website and

About Elamipretide

Stealth’s lead candidate, elamipretide (formerly known as Bendavia and Ocuvia), is an investigational drug with the potential to modify disease by preserving energetics and restoring normal energy production in mitochondria, while decreasing oxidative stress. The energetics from mitochondria maintain healthy physiology and prevent disease. This mitochondria-targeted candidate is being developed for both rare and common diseases.

Stealth BioTherapeutics: Leading Mitochondrial Medicine

Stealth BioTherapeutics is a privately held biopharmaceutical company committed to bringing mitochondrial therapies to patients to treat both rare and common diseases. Stealth’s clinical development program is focused along several core therapeutic areas, including orphan mitochondrial diseases, cardio-renal diseases and ophthalmic disorders. More information regarding Stealth and its pipeline is available at

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