Endogena Therapeutics receives FDA Orphan Drug designation

Following the closing of an oversubscribed Series A round capped at $8 million, Zurich based Endogena has obtained the FDA Orphan Drug Designation approval for its lead program targeting retinitis pigmentosa. This is the first small-molecule cell regeneration treatment to be developed for the leading cause of inherited blindness.

Endogena Therapeutics is a preclinical-stage biotech company that discovers and develops first-in-class endogenous regenerative medicines to repair and regenerate tissues and organs. Its compound EA-2353 is the first small-molecule being developed for endogenous photoreceptor regeneration with clinical trials anticipated to start by the end of the year. The treatment works by selective regulation of the endogenous adult stem- and progenitor cells for controlled tissue repair. Four further candidates are in the pileline.

The company has received Orphan Drug Designation from the US Food and Drug Administration (FDA) for EA-2353. Commenting on this achievement Matthias Steger, CEO of Endogena, said: “Receiving orphan drug designation for EA-2353 is an important milestone to emerge from our innovative endogenous regenerative platform and confirms its potential for a medical treatment paradigm shift for degenerative diseases, in this case for retinitis pigmentosa patients who so desperately need it. Subject to IND approval, we anticipate enrolling our first patients into the clinical trial by the end of this year.”

The FDA’s Office of Orphan Drug Products grants orphan status to support development of medicines for underserved patient populations, or rare disorders, that affect fewer than 200,000 people in the U.S. This provides benefits including market exclusivity once approved, exemption of FDA application fees and tax credits for qualified clinical trials.

Oversubscribed Series A round
This milestone comes a few months after closing an oversubscribed first round of Series A financing, capped at $8 million. This first round of Series A financing was led by existing investors, Rejuveron Life Sciences AG and DEFTA Partners, with new investors joining the syndicate. The funding will enable the company to complete first clinical proof-of-concept studies of its novel treatment paradigm for patients with devastating degenerative eye conditions, as well as develop additional programs, leveraging the company’s artificial intelligence-driven drug discovery platform.

(Press release / RAN)