Sixteen patients in the Phase 2 trial for a new Fuchs’ corneal endothelial dystrophy (FCED) treatment have started receiving an eye drop targeting corneal mitochondria.
It is hoped the medication will be the first to halt or reverse the disease, which is commonly developed in middle age or later life.
Mitochondria act as the powerhouses of the body’s cells, and have been linked – when they malfunction – to FCED, which can ultimately cause blindness, Stealth BioTherapeutics chief scientific officer, Mark Bamberger, told OT.
He noted: “The vision system is a very energy-dependent biologic function.”
The medication in the Stealth BioTherapeutics-owned drops, elamipretide, targets these mitochondria to protect and restore its energy-producing function.
Mr Bamberger, explained: “Many severe ocular diseases, including Fuchs’, are linked to dysfunctional mitochondria … We are aiming to safely restore [mitochondrial] energetics.”
The 16 subjects in the US study, being conducted in Boston and Cincinnati, have mild to moderate corneal oedema. Twice daily, each receives one drop of elamipretide in their randomly selected treatment eye and a control drop in the control eye, for 12 weeks.
“We expect that comparing treatment versus control in the same trial participant should reduce the amount of variability that would be seen if we instead compared control participants with treated participants.”
Initial results on the medication’s safety, side effects and efficacy should be available later this year.
“[We] will be initiating clinical trials evaluating the topical eye drop formulation of elamipretide in Leber’s hereditary optic neuropathy, a genetic mitochondrial disease, and dry age-related macular degeneration. Data from these trials is anticipated in … 2017,” Mr Bamberger concluded.