Dose Escalation Study to Evaluate the Safety/Tolerability and Efficacy of EA-2353 in Subjects With Retinitis Pigmentosa (Endogena Therapeutics)
In the summer of 2022, Endogena launched its first phase 1/2a study of EA-2353 for retinitis pigmentosa (RP). EA-2353 is a novel small-molecule that aims to activate stem cells in the retina to produce and replace lost or damaged photoreceptors. EA-2353 is designed to work independently of the underlying gene mutation. 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 February 6, 2023, the investigation of EA-2353 for the treatment of RP received U.S. FDA Fast Track designation.
On April 5, 2023, Endogena announced that the dose-escalation stage of the phase 1/2a study has been successfully completed. Fourteen participants with genetically confirmed RP received repeated intravitreal injections (eye injections) in one eye. No clinically relevant or dose-limiting adverse events were identified.
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 using the highest dose that demonstrated safety and tolerability in the initial Phase 1/2a trial.
What this means for Usher syndrome: EA-2353 could be a game changer for all RP patients, including those 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.
