RARE Daily

FDA Grants Fast Track Designation to Endogena’s EA-2353 for the Treatment of Retinitis Pigmentosa

February 6, 2023

Rare Daily Staff

The U.S. Food and Drug Administration granted Fast Track designation to Endogena Therapeutics’ EA-2353 for the treatment of retinitis pigmentosa.

Retinitis pigmentosa (RP) is a serious and debilitating condition. It consists of 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 potentially preserve or restore visual function. This gene-independent treatment approach has significant advantages in RP, which has multiple genetic causes. EA-2353 was granted orphan drug designation by the FDA in May 2021.

Endogena is currently conducting a phase 1/2a dose-escalation study in collaboration with lead investigator, Mark Pennesi, professor of Ophthalmology at the Casey Eye Institute in Oregon, to examine the safety, tolerability, and preliminary efficacy of EA-2353 administered by intravitreal injection in patients with RP. A total of 14 patients with RP due to any pathologic genetic mutation are being recruited across up to six sites in the United States, and the first patient was dosed in July 2022.

Fast Track is a process designed to enable patients to benefit earlier from important new drugs for serious conditions. This Fast Track designation will enable Endogena to have more frequent communications with the FDA on the development of EA-2353 and allow more rapid regulatory review of the future new drug application.

“This acknowledgement by the FDA of the potential of EA-2353 for RP gives hope for patients living with this devastating degenerative disease,” said Matthias Steger, CEO of Endogena. “It is a significant milestone for our company, our investors, and gives recognition to our dedicated team at Endogena, who have been working for the past six years to reach this point.”

Endogena says its artificial intelligence-driven drug discovery platform, combined with cutting edge knowledge of molecular pathways that regulate retinal stem cells and retinal pigment epithelial cells, could provide 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, which is approaching IND-enabling studies, and earlier programs in idiopathic pulmonary fibrosis and hematopoietic recovery.

Photo: Matthias Steger, CEO of Endogena

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