Stealth BioTherapeutics Expands Ocuvia’s Clinical Development into Rare Mitochondrial Optic Neuropathies

Ocuvia is one of the first investigational drugs with potential to treat inherited optic neuropathies common in over 20 genetic mitochondrial diseases

Boston, MA – Stealth BioTherapeutics (Stealth), a clinical-stage biopharmaceutical company developing drug candidates for mitochondrial dysfunction, today announced expansion of its ophthalmology program with FDA granting a Type B meeting for inherited optic neuropathies. Optic neuropathies include a diverse group of genetic diseases characterized by vision loss due to mitochondrial dysfunction. Affecting more than 1 in 10,000 individuals, optic neuropathies are common to more than 20 inherited mitochondrial diseases. Stealth’s ophthalmology program is led by Ocuvia, an investigational drug targeting mitochondrial dysfunction to treat common and rare eye diseases. Ocuvia is currently being studied in patients with diabetic macular edema and dry age-related macular degeneration.

“With no FDA-approved therapies for patients with inherited optic neuropathies, there is a clear unmet clinical need,” said Alfredo Sadun, M.D., Ph.D., Director of Neuro-Ophthalmology at the Doheny Eye Institute of the University of California at Los Angeles. “By ameliorating mitochondrial dysfunction, Ocuvia is a promising therapy for these rare genetic optic disorders that progress to profound visual loss.”

Stealth plans to initiate a clinical study in inherited optic neuropathies with its ReSIGHT trial for Leber’s Hereditary Optic Neuropathy (LHON). LHON is a rare genetic mitochondrial disease and the most common inherited optic neuropathy, causing sudden and permanent loss of vision predominately in young men. Lissa Poincenot, a leading LHON patient advocate, added, “We are tremendously excited for Ocuvia’s potential to treat such devastating optic neuropathies and improve the lives of our patients.”

By expanding Ocuvia’s clinical development, Stealth is furthering its commitment to patients with orphan mitochondrial diseases. There are presently no FDA-approved therapies for these patients, and treatment options are often limited to vitamins and supplements. Along with its ophthalmology program in inherited optic neuropathies, Stealth is also recruiting patients in its MMPOWER study with Bendavia. The MMPOWER trial is a multicenter study for the treatment of Mitochondrial Myopathy (MM) in patients with genetic mitochondrial diseases.

“We are focused on our rare disease programs with Ocuvia and Bendavia in hopes of providing patients with the first FDA-approved therapy for inherited mitochondrial diseases,” said Chief Executive Officer Travis Wilson. “We look forward to initiating the ReSIGHT trial later this year and studying Ocuvia’s benefits in this underserved patient population.”

Mitochondria, The Cell’s Powerhouse

Mitochondria are the cell’s powerhouse, responsible for more than 90% of the energy our bodies need to sustain life and support growth. The energetics from mitochondria maintain healthy physiology and prevent disease. In many common and rare diseases, dysfunctional mitochondria are a key component of disease progression.

About Bendavia™ and Ocuvia™

Stealth’s lead candidates, Bendavia and Ocuvia, are investigational drugs with the potential to modify disease through mitoprotection — the ability to preserve energetics and restore normal energy production in mitochondria, while decreasing oxidative stress. These clinical candidates are being developed for both common and rare diseases including mitochondrial diseases where there are no FDA-approved treatments. The underlying science of Bendavia and Ocuvia is supported by more than 100 independent, peer-reviewed publications and abstracts. These mitochondrial-targeted candidates represent a novel therapeutic approach with the potential to address a wide variety of diseases having unmet treatment needs.

Stealth BioTherapeutics: Leading Mitochondrial Medicine

Stealth BioTherapeutics is a privately held biopharmaceutical company committed to bringing mitochondria therapies to patients to treat both common and rare diseases. As a key common element in a variety of serious, debilitating diseases, mitochondria — the cell’s energy source — offer a promising, and yet untapped, target to modify diseases with significant unmet treatment needs. Stealth is expanding its clinical development program to additional therapeutic areas, including cardio-renal diseases, ophthalmic disorders and orphan mitochondrial diseases. By defining the broad potential of its mitochondrial platform and therapies, Stealth is leading mitochondrial medicine.

More information regarding Stealth and its pipeline is available at

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