The FDA has granted Endogena Therapeutics Fast Track Designation status for EA-2353, its investigational candidate, for the treatment of retinitis pigmentosa (RP).
Remind me what Fast Track designation means.
This process is a request made by a company to the FDA for facilitating the development and expediting the review of drugs to treat serious medical conditions and fill an unmet need; essentially, it’s a way to make new drugs available to patients faster.
Unlike a Breakthrough Therapy designation, this can be requested with non-clinical data and/or preliminary clinical evidence.
Talk about EA-2353.
Granted Orphan Drug designation in 2021, 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.
Any clinical trials?
Yes! A phase 1/2a dose-escalation study (NCT05392751) is currently underway to assess the safety, tolerability, and preliminary efficacy of EA-2353 administered via intravitreal injection for RP.
The study includes 14 patients—recruited across six sites in the U.S.—diagnosed with RP. The first patient was dosed in July 2022.
Expected completion date is June 2025.
Significance?
The FDA granting this designation for EA-2353 marks a milestone for the company in its quest to provide a potential new treatment paradigm for degenerative conditions associated with aging and genetic disorders including retinitis pigmentosa and age-related macular degeneration.
Further, Endogena will now have more frequent contact with the FDA in regards to EA-2353 as well as a more rapid regulatory review process when a new drug application might be filed in the future.