- Thursday, May 4, 2023 @ 8:00 am
Endogena Therapeutics Inc., a clinical-stage biotech company, has completed patient enrollment ahead of schedule in its Phase 1/2a trial of EA-2353 for the treatment of retinitis pigmentosa (RP).
Endogena Therapeutics Inc., a clinical-stage biotech company, has 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 in April, the ongoing trial is now in the expansion cohort stage. The trial (NCT05392751) is examining the safety, tolerability, and preliminary efficacy of intravitreal EA-2353, a first-in-class small molecule that selectively activates endogenous retinal stem and progenitor cells to potentially preserve and restore visual function. The trial has enrolled 14 patients with RP due to any pathologic genetic mutation and is being conducted across five US sites.
The first patient started treatment in July 2022 and topline interim data is anticipated in early 2024.
Matthias Steger, PhD, MBA, CEO of Endogena Therapeutics: “Getting this far in a remarkably short time is testament to the outstanding execution of our clinical team in collaboration with InFocus Clinical Research. There’s a tremendous enthusiasm surrounding our trial and completing enrollment 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.”
About EA-2353
EA-2353 is a first-in-class small molecule that selectively activates endogenous retinal stem and progenitor cells, which differentiate into photoreceptors and can potentially preserve and restore visual function. This gene-independent treatment approach has significant advantages in RP, which has multiple genetic causes. EA-2353 was granted Orphan Drug Designation by the US FDA in May 2021, and Fast Track Designation in February 2023.
About retinitis pigmentosa (RP)
RP is a serious and debilitating condition. It consists of a group of inherited diseases causing slow and progressive retinal degeneration and loss of vision, for which there is currently no treatment for most patients. It is a leading cause of inherited blindness, with an estimated 1.5 million people worldwide presently affected.
