Endogena Therapeutics transitions into clinical stage with its novel regenerative treatment for blindness caused by retinitis pigmentosa


Endogena Therapeutics Inc. announced today that the first patient has been treated in a phase 1/2a clinical study of its lead product, EA-2353, a photoreceptor regeneration treatment for retinitis pigmentosa (RP).

RP is a group of inherited diseases causing slow and progressive retinal degeneration and loss of vision, for which there is currently no treatment for most patients. It is a leading cause of inherited blindness, with an estimated 1.5 million people worldwide presently affected.

EA-2353 takes a novel, small-molecule approach and selectively activates endogenous retinal stem and progenitor cells, which differentiate into photoreceptors and can hence potentially preserve or restore visual function. This gene-independent treatment approach has significant advantages in RP, which has multiple genetic causes. EA-2353 has been granted orphan drug designation by the U.S. Food and Drug Administration in May 2021.

The phase 1/2a dose-escalation study will examine the safety, tolerability and the preliminary efficacy of EA-2353 administered by intravitreal injection. A total of 14 patients with RP due to any pathologic genetic mutation will be recruited across up to six sites in the USA.

Principal InvestigatorMark Pennesi, MD, PhD, Professor of Ophthalmology at the Casey Eye Institute in Oregon, USA, said: “This is very exciting and I am hopeful that this new treatment has the potential to be a major game-changer for patients with RP who currently have no other options.”

Matthias Steger, PhD, MBA, CEO of Endogena, said: “It’s a tribute to the Endogena team’s dedication and firm belief in this new treatment paradigm that we’ve reached the significant milestone of commencing the first studies in patients with RP. For the first time, we’re able to offer real hope to RP patients.”

Endogena’s artificial intelligence-driven drug discovery platform, combined with cutting edge knowledge of molecular pathways that regulate retinal stem cells and retinal pigment epithelial cells, provides a potential new treatment paradigm to tackle degenerative conditions related to aging and genetic disorders. Beyond EA-2353, other products in Endogena’s pipeline include a treatment for dry age-related macular degeneration (AMD), which is approaching IND-enabling studies.

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