Endogena Therapeutics Inc. announced it has completed patient enrollment ahead of schedule for its phase 1/2a trial assessing EA-2353 for the treatment of retinitis pigmentosa (RP).
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.
Anything else notable about it?
Yes! The FDA granted EA-2353 Orphan Drug designation in 2021 and, most recently, Fast Track designation in February 2023.
Now talk about this study.
The open-label, multiple- ascending, phase 1/2a dose study (NCT05392751) is assessing the safety, tolerability, and preliminary efficacy of EA-2353.
A total of 14 patients (ages 18+) across five sites in the U.S. have been recruited. Inclusion criteria included a diagnosis of RP and a best-corrected visual acuity (BCVA) in the worse eye of 20/50.
Participants will be placed in up to four cohorts:
- Cohort 1 = low dose
- Cohort 2 = mid dose
- Cohort 3 = high dose
- Cohort 4 = maximum tolerated dose
Each of the first three cohorts will treat three patients with four weekly unilateral intravitreal injections of EA-2353 into the study eye.
The first patient was dosed in July 2022.
Wait ... didn’t the company make an announcement recently?
Yes, in April 2023 (see here)... but that was for the conclusion of its dose-escalation stage of the study, which determined and established the recommended dosage for EA-2353.
Data reported from this stage included a favorable safety and tolerability profile, with no clinically-relevant or dose-limiting adverse events following repeat injections.
When can we expect results?
The company anticipates reporting interim data in early 2024.
According to Clinical Trials, the study is expected to be completed in June 2025.