ONL Therapeutics has published data from its Phase 1b study of xelafaslatide (formerly ONL1204 Ophthalmic Solution), an investigational small-molecule inhibitor of the Fas pathway, designed to protect retinal cells from degeneration, targeting Geographic atrophy (GA) associated with dry Age-related macular degeneration (AMD).
The multicenter Phase 1b trial included a dose-escalation/open-label component and a randomized, sham-controlled natural history/treatment component. Patients received intravitreal injections of xelafaslatide (50 µg, 100 µg or 200 µg) and were monitored over a 24-week period for safety, as well as lesion growth and visual acuity.
According to the published results in Ophthalmology Science, xelafaslatide was generally safe and well tolerated at all evaluated dose levels. In addition, treated eyes showed signals suggestive of slower GA lesion growth and some improvements in best-corrected visual acuity compared with untreated (sham) eyes.
ONL said the findings support continued development of xelafaslatide, and the company has already initiated a larger global Phase 2 trial (GALAXY trial) to further evaluate efficacy and safety in GA patients.
