The FDA has granted Endogena Therapeutics Inc. clearance for its investigational new drug (IND) application for EA-2351, a small-molecule approach developed for the treatment of geographic atrophy (GA).
Refresh me on Endogena.
Based in Zürich, Switzerland, the clinical-stage biotechnology company is focused on developing first-in-class endogenous regenerative medicines to repair and regenerate tissues and organs, with the goal to address unmet medical needs linked to aging and genetic diseases.
Of note, Endogena has US and Canadian-based operations in San Francisco, California and Toronto, Canada, respectively.
Talk about the company’s technology approach.
Endogena combines stem cell biology, artificial intelligence, and a small-molecule design to target adult stem- and progenitor cells (endogenous cells) to create a novel drug discovery approach that can target selective regulation for controlled tissue repair via small molecules.
Now wait… isn’t the company developing a candidate for RP?
You’re right (and it is)! That intravitreally-administered compound is called EA-2353 (note the ‘3’), which is different from EA-2351. Granted orphan drug designation in 2021 (and fast track designation in February 2023), EA-2353 is currently in clinical trials for the treatment of retinitis pigmentosa (RP). See coverage on it here and here.
Ahh… so now tell me about EA-2351.
EA-2351 is a gene-independent, small-molecule approach similar to EA-2353 in that it targets specific cell populations—in this case, retinal pigment epithelium (RPE) cells, which are key to regenerating and restoring photoreceptor function—through different pathways via intravitreal administration.
Once activated, these activated RPE cells can translate to the areas of the eye where RPE cells have died (due to age-related macular degeneration [AMD]), effectively allowing for these cells to develop into healthy RPE cells and promote healing.
The ultimate goal: potentially enabling AMD patients to regain some of their lost vision.
Watch the video below.
How is this treatment different from other AMD therapies?
Essentially, according to Endogena, by facilitating a patient’s body to produce new healthy RPE cells. Other treatments do not use gene editing or inject foreign cells into the body.
Gotcha. So what’s next?
Per the company, a first-in-human study on EA-2351 is planned to kick off in 2024. Stay tuned for more details!