Stealth Expands Experimental Therapy to Treat Rare Mitochondrial Optic Neuropathies

Stealth Expands Experimental Therapy to Treat Rare Mitochondrial Optic Neuropathies

Stealth BioTherapeutics (Stealth), a privately held biopharmaceutical company committed to bringing mitochondrial therapies to patients to treat both common and rare diseases, recently announced expansion of its ophthalmology program with FDA granting a Type B meeting for genetic optic neuropathies.

Optic neuropathies are common to more than 20 congenital mitochondrial diseases, and affect 1 in every 10,000 individuals. The condition causes loss of vision because of mitochondrial dysfunction.

Ocuvia is Stealth’s leading ophthalmology program drug candidate. The compound targets mitochondrial dysfunction and is meant to work as a treatment to both rare and common eye conditions. The drug is being evaluated in patients suffering from diabetic macular edema and dry age-related macular degeneration.

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“With no FDA-approved therapies for patients with inherited optic neuropathies, there is a clear unmet clinical need,” said  in a recent news release 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.”

The company is planning to begin a clinical trial on patients with inherited optic neuropathies with its ReSIGHT study for Leber’s Hereditary Optic Neuropathy (LHON), a rare inherited mitochondrial disease and the most common inherited optic neuropathy. LHON causes abrupt and permanent vision loss and usually affects 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.”

With the expansion of Ocuvia’s development, the company is continuing its commitment to developing strategies for patients suffering from mitochondrial diseases. Currently, there are no FDA approved drugs to treat patients with these conditions, and often treatments are limited to supplements and vitamins.

Stealth is currently recruiting patients for its MMPOWER clinical trial with Bendavia. The trial is a multicentre study that is evaluating Bendavia for the treatment of patients with Mitochondrial Myopathy (MM) 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.”

 

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