Endogena Therapeutics Inc. announced that it has successfully completed the dose-escalation stage of its phase 1/2a study on intravitreal injections of EA-2353 for the treatment of retinitis pigmentosa (RP).
What exactly is a dose-escalation stage?
The dose-escalation stage of clinical trials establishes the recommended dosage of a new drug or treatment by increasing the dosage a little at a time in different groups of patients until the highest dosage that does not cause harmful side effects is identified.
Now talk about EA-2353.
EA-2353 is a gene-independent, small-molecule approach that selectively activates endogenous retinal stem and progenitor cells. These differentiated cells break into photoreceptors which then migrate to “fill in the spaces” where apoptotic retinal cells were located allowing for wound repair and the potential to restore or preserve visual function.
Of note, this treatment does not require gene editing or the injection of foreign cells into the body.
Didn’t I just hear about this?
Yes! EA-2353 recently earned fast track designation from the FDA in February 2023; it already received orphan drug designation (ODD) in May 2021.
Tell me about the trial now.
The phase 1/2a study (NCT05392751) is assessing the safety, tolerability, and preliminary efficacy of EA-2353.
A total of 14 patients (ages 18+) across six sites in the U.S. are currently being recruited. Inclusion criteria includes a diagnosis of RP and a best-corrected visual acuity (BCVA) in the worse eye of 20/50.
The first patient was dosed in July 2022 and, as of April 5, 2023, 9 patients have been treated so far.
Anything to report from the dose-escalation stage?
Endogena reported a positive safety and tolerability profile for EA-2353. Following repeated intravitreal injections, no clinically-relevant or dose-limiting adverse events (AEs) were identified.
Given this data, patient enrollment will begin for the expansion cohort of the trial. The highest dosage will be administered in order to determine EA-2353’s potential efficacy.
When can we expect results?
According to the study’s Clinical Trials data, estimated completion is June 2025. So stay tuned!