Treating dry AMD as a mitochondrial syndrome

Stealth Biotherapeutics, a clinical-stage biopharmaceutical company, is investigating the safety and tolerability of daily subcutaneous injections of elamipretide in patients with intermediate dry AMD. Top-line results of a phase 1 study are expected mid-2017.

"There are currently no FDA-approved treatment options for dry AMD, so we are eager to better understand the effect that elamipretide may have in treating these roughly 13 million patients," said Scott Cousins, MD, trial investigator and professor of Ophthalmology and director of the Duke University Center for Macular Diseases. "Mitochondrial dysfunction that stems from various environmental toxins may be an important causative factor in dry AMD, and in laboratory models, elamipretide appears to prevent mitochondrial dysfunction in the retinal pigment epithelium."

Elamipretide is an investigational drug with the potential to modify disease by preserving energetics and restoring normal energy production in mitochondria, while decreasing oxidative stress created by the over-production of reactive oxygen species. The drug is uniquely able to target the inner mitochondrial membrane through the company’s special formulation designed for both systemic and topical ophthalmic delivery.

Subjects eligible for the 12-week, open-label study must be age 55 or older, have intermediate AMD in at least 1 eye, exhibit drusen but no geographic atrophy or noncentral geographic atrophy. Subjects who have received antiangiogenic therapies are excluded.

The study's primary endpoints are safety and tolerability, while changes from baseline in physical/ophthalmic examinations and feasibility of subcutaneous injections are secondary endpoints.

The company is also evaluating elamipretide for other ophthalmic diseases such as diabetic macular edema, Fuchs Endothelial Corneal Dystrophy and Leber’s Hereditary Optic Neuropathy, as well as for mitochondrial myopathy, heart failure and renal disease.