Stealth BioTherapeutics recently announced the initiation of the Phase 2 ReVEAL clinical trial to assess the efficacy, safety, and tolerability of its topical eye formulation (elamipretide) to treat patients with Fuchs corneal endothelial dystrophy (FCED), a disease linked to dysfunctional mitochondria.
FCED is a genetic disease that affects the eye and leads to vision problems. The condition is characterized by endothelial cell death that primarily affects the front surface of the eye (cornea), and can progress to blindness if untreated.
According to the Genetics Home Reference, FECD is a fairly common condition, thought to be caused by the loss of mitochondrial energetics, and affects about 4 percent of the U.S. population over the age of 40. Currently, the only available and effective treatment for restoring lost vision is corneal transplantation.
Stealth’s lead candidate, elamipretide (formerly known as Bendavia and Ocuvia), targets the inner mitochondrial membrane, preserving energetics, restoring normal energy production in mitochondria and, at the same time, reducing oxidative stress.
“Many severe ocular diseases, including Fuchs’, are linked to dysfunctional mitochondria and are characterized by a state of increased oxidative stress and impaired energetic components of the eye. We are aiming to safely restore these energetics and to show a clinical benefit with elamipretide in visual acuity and other functional measures,” Mark Bamberger, Stealth BioTherapeutics’ chief scientific officer, said in a press release. “This new trial furthers our ongoing commitment to uncovering the role mitochondrial dysfunction plays in ocular disorders, and how we might address unmet needs across several indications where impaired energetics are a final common pathway.”
The ReVEAL clinical trial (NCT02653391), currently recruiting participants, is a randomized, placebo-controlled, paired-eye study taking place at two sites — in Boston, Massachusetts, and Cincinnati, Ohio — and plans to enroll 16 patients with mild to moderate corneal edema. Participants will be treated twice daily with the formulation and a control ophthalmic solution for 12 weeks. The primary endpoints are the safety and tolerability, and the secondary endpoints include reduction in central corneal thickness, and improved visual acuity. First data from the trial are expected in the third quarter of 2016.
More information on the trial, including how to participate, is available through its clinical trials.gov site.