Endogena Therapeutics: Phase 1/2a clinical trial for the treatment of retinitis pigmentosa complete

No clinically relevant or dose-limiting adverse events were identified after repeated intravitreal injections. Given the positive safety and tolerability profile, the study will now enroll patients into the expansion cohort, using the highest dose evaluated in the study to explore the potential efficacy of the compound.

The phase 1/2a study is conducted in collaboration with Endogena’s Lead Investigator, Mark Pennesi, MD, PhD, Professor of Ophthalmology at the Casey Eye Institute in Oregon, USA, to examine the safety, tolerability and preliminary efficacy of EA-2353 administered by intravitreal injection in patients with RP (ClinicalTrials.gov identifier: NCT05392751). A total of 14 patients with RP due to any pathologic genetic mutation are being recruited across up to six sites in the USA. The first patient was dosed in July 2022 and 9 patients have been treated to date during the dose escalation phase.

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 US FDA in May 2021, and Fast Track Designation in February 2023.

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.

Matthias Steger, PhD, MBA, CEO of Endogena, said: “We are edging closer to a treatment for this devastating condition, and I’m very encouraged by the safety and tolerability profile in this dose escalation stage. We have the best people working tirelessly to bring this novel treatment to the patients who need it as fast as possible.”

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 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.