ONL Therapeutics reported that data from its phase 1b study of xelafaslatide, a first-in-class small molecule Fas inhibitor, have been published in the journal Ophthalmology Science. Xelafaslatide is designed to protect retinal cells, including photoreceptors, from cell death in a range of retinal conditions, including geographic atrophy (GA) associated with dry age-related macular degeneration.

The phase 1b study included a dose-escalation/open-label component with 6 participants and a randomized, double-masked, sham-controlled natural history/treatment component with 22 participants. Dosing ranged from 50 μg to 200 μg, with intravitreal injections administered either once or twice, 12 weeks apart. Participants were followed for 24 weeks after dosing.

Results showed that xelafaslatide was generally safe and well tolerated. Efficacy assessments indicated signals of slowed GA lesion growth in treated eyes compared to untreated eyes. Best-corrected visual acuity was also monitored as a secondary endpoint.

ONL Therapeutics has initiated GALAXY, a global phase 2 trial evaluating efficacy and safety in GA. The compound has also been studied in retinal detachment and glaucoma, with orphan drug designation granted for macula-off retinal detachment. RP