The treatment is a small molecule that activates endogenous retinal stem and progenitor cells

Endogena Therapeutics has revealed that it has completed patient enrolment for its phase 1/2a trial concerning the company’s EA-2353 candidate. The therapy has been developed to treat retinitis pigmentosa (RP).

The dose-escalation stage was successfully completed in April, with the ongoing trial now in the expansion cohort stage. The trial – known as NCT05392751 – is examining the tolerability, safety and preliminary efficacy of intravitreal EA-2353.

The treatment is a small molecule that activates endogenous retinal stem and progenitor cells with a view to preserving and restoring visual functionality. The study has enrolled 14 patients with RP due to any pathologic genetic mutation and is being conducted widely across five US sites.

Meanwhile, the first patient commenced treatment in July 2022 and top-line interim data is expected next year.

Matthias Steger, CEO at Endogena Therapeutics, reflected: “Getting this far in a remarkably short time is a testament to the outstanding execution of our clinical team in collaboration with InFocus Clinical Research.

He added: There’s a tremendous enthusiasm surrounding our trial and completing enrolment ahead of schedule has only been possible thanks to the invaluable contribution of the patients, their families, the investigators and their study staff. We are all driven by the hope of finding a treatment for this rare and devastating disease.”

RP remains a very serious and debilitating condition. It consists of a group of inherited diseases resulting in progressive retinal degeneration and loss of vision. It is a leading cause of inherited blindness, with an estimated 1.5 million people worldwide presently impacted. At present, there is no treatment for most patients.