Retinitis pigmentosa drug proved safe in Phase I/IIa study

Ophthalmologist looks at retinal scan

Endogena Therapeutics has completed the dose-escalation stage of its Phase I/IIa study of EA-2353 in retinitis pigmentosa (RP) with no clinically relevant or dose-limiting adverse events.

Given the positive safety and tolerability profile, the study will now enrol patients into the expansion cohort, using the highest dose evaluated in the study to explore the potential efficacy of the compound.

The Phase I/IIa study is conducted in collaboration with Lead Investigator, Mark Pennesi, Professor of Ophthalmology at the Casey Eye Institute in Oregon, US.

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 nine 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.

New treatment paradigm

RP is a serious and debilitating condition, 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, 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 ageing and genetic disorders.

Other products in Endogena’s pipeline include treatment for dry age-related macular degeneration (AMD), which is approaching IND-enabling studies, and earlier programmes in idiopathic pulmonary fibrosis (IPF) and hematopoietic recovery.

Related Articles

Join FREE today and become a member
of Drug Discovery World

Membership includes:

  • Full access to the website including free and gated premium content in news, articles, business, regulatory, cancer research, intelligence and more.
  • Unlimited App access: current and archived digital issues of DDW magazine with search functionality, special in App only content and links to the latest industry news and information.
  • Weekly e-newsletter, a round-up of the most interesting and pertinent industry news and developments.
  • Whitepapers, eBooks and information from trusted third parties.
Join For Free