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FDA grants orphan drug designation for Endogena’s lead program targeting retinitis pigmentosa

First small-molecule cell regeneration treatment to be developed for the leading cause of inherited blindness

Endogena Therapeutics Inc., which discovers and develops endogenous regenerative medicines, announced today that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation for its EA-2353 ophthalmic suspension targeting retinitis pigmentosa, a rare condition that causes slow and progressive loss of vision.

Endogena’s development 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.

The concept is driven by cutting-edge knowledge of molecular pathways that regulate retinal stem cells, which generate photoreceptors during development and are dormant in the adult mammalian eye. This approach is gene-independent, which has significant advantages in inherited retina diseases that have multiple genetic causes.

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. We have completed our IND enabling studies and, subject to IND approval, we anticipate enrolling our first patients into the clinical trial by the end of this year.”

Mark Pennesi, MD/PhD, the Kenneth C. Swan Associate Professor of Ophthalmology at the Casey Eye Institute in Portland (OR), USA, who will lead the phase I/IIa studies, said: “I’m eager to begin clinical proof-of-concept studies to establish whether Endogena’s approach could offer a new treatment paradigm in retinitis pigmentosa. This novel method of tissue repair holds great promise for patients with this devastating degenerative eye condition.”

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.