Endogena Therapeutics: Dose Escalation Study to Evaluate the Safety/Tolerability and Efficacy of EA-2353 in Subjects With Retinitis

A graphic highlighting USH Science News with the title: "Endogena Therapeutics Completes Dose Escalation in the Phase 1/2A Clinical Trial of EA-2353 for the Treatment of Retinitis Pigmentosa" April 5, 2023

In the summer of 2022, Endogena launched its first dosing of its phase 1/2a study of EA-2353 in retinitis pigmentosa (RP). Endogena is attempting to activate stem cells in the retina with EA-2353, which takes a novel, small-molecule approach, with hopes of producing and replacing lost and damaged photoreceptors, the reason for loss of vision in Usher syndrome and retinitis pigmentosa. EA-2353 is designed to work independently of the underlying gene mutation.

Fourteen participants are enrolled in this trial, all of which will receive intravitreal injections (eye injections) in one eye. All participants show symptoms of RP and have a genetic diagnosis.

On February 6, 2023, the investigation of EA-2353 for the treatment of RP received U.S. FDA Fast Track designation.

This study is being conducted in collaboration with Endogena’s Lead Investigator, Mark Pennesi, MD, PhD, Professor of Ophthalmology at the Casey Eye Institute in Oregon. 

On April 5, 2023, Endogena announced that the dose-escalation stage of the phase 1/2a study has been successfully completed. No clinically relevant or dose-limiting adverse events were identified after repeated intravitreal injections. After the approach has been shown to be safe and tolerable with various doses, the study enrolls patients into the expansion cohort, using the highest dose evaluated to examine how effective this drug therapy could be for patients with RP. 

On May 4, 2023, Endogena announced it had completed patient enrollment ahead of schedule in its Phase 1/2a trial of EA-2353 for the treatment of retinitis pigmentosa (RP). With the dose-escalation stage completed, the ongoing trial is now in the expansion cohort stage.

What this means for Usher syndrome: EA-2353 could be a game changer for all RP patients, not just ones with Usher syndrome. Activated stem cells may enable regeneration of photoreceptors and potentially reinstate light-detection capabilities. In addition, the Fast Track designation gives hope to the community that this treatment could be viable sooner rather than later.