The US Food and Drug Administration (FDA) has designated the investigation of Endogena Therapeutics’ EA-2353 for the treatment of retinitis pigmentosa (RP) as a fast track development program.
Fast track is a process designed to enable patients to benefit earlier from new drugs for serious conditions.
Endogena Therapeutics’ 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 advantages in RP, which has multiple genetic causes. EA-2353 was granted orphan drug designation by the US FDA in May 2021.
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.
Endogena Therapeutics 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, U.S., to examine the safety, tolerability and preliminary efficacy of EA-2353 administered by intravitreal injection in patients with RP.
Fourteen patients with RP due to any pathologic genetic mutation are being recruited across up to six sites in the U.S., and the first patient was dosed in July 2022.
Matthias Steger, CEO of Endogena Therapeutics, said: “This acknowledgement by the FDA of the potential of EA-2353 for RP gives hope for patients living with this devastating degenerative disease. 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.”
Beyond EA-2353, other products in Endogena Therapeutics’ pipeline include a treatment for dry age-related macular degeneration (AMD), which is approaching IND-enabling studies, and earlier programs in idiopathic pulmonary fibrosis (IPF) and hematopoietic recovery.