Endogena Therapeutics Receives FDA Fast Track Designation for Treatment of Retinitis Pigmentosa

Endogena Therapeutics announced that the US FDA has designated the investigation of its drug EA-2353 for the treatment of retinitis pigmentosa (RP) as a Fast Track development program. This process speeds up the availability of new drugs for serious conditions to patients.

EA-2353 is a novel, small-molecule that selectively activates retinal stem and progenitor cells, potentially preserving or restoring visual function. Its gene-independent approach has advantages in treating RP, which has multiple genetic causes. EA-2353 received orphan drug designation from the US FDA in May 2021.

Retinitis pigmentosa (RP) is a debilitating and serious condition that causes slow and progressive retinal degeneration and loss of vision. There is currently no treatment available for most patients, making it a leading cause of inherited blindness with an estimated 1.5 million people affected globally.

Endogena Therapeutics is conducting a phase 1/2a dose-escalation study with lead investigator Mark Pennesi, MD, PhD, Professor of Ophthalmology at the Casey Eye Institute in Oregon, USA. The study aims to evaluate the safety, tolerability, and preliminary efficacy of EA-2353 when administered through intravitreal injection in RP patients. A total of 14 patients with RP caused by any genetic mutation are being recruited across up to six sites in the US, with the first patient being dosed in July 2022.

The Fast Track designation from the US FDA will allow Endogena Therapeutics to have more frequent communication and more rapid regulatory review of the future new drug application for EA-2353.

Matthias Steger, PhD, MBA, CEO of Endogena, 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.”

Endogena's drug discovery platform, driven by artificial intelligence and informed by cutting edge knowledge of molecular pathways that regulate retinal stem cells and retinal pigment epithelial cells, offers a new treatment approach for degenerative conditions related to aging and genetics. In addition to EA-2353, Endogena's pipeline includes a treatment for dry age-related macular degeneration (AMD), which is nearing IND-enabling studies, and earlier programs for idiopathic pulmonary fibrosis (IPF) and hematopoietic recovery.