Drug intelligence / Profile preview

xelafaslatide

Development stage
Phase 2
Lead developer
ONL Therapeutics
Modality
Peptides, Small Molecules
Administration
Intravitreal
01

Overview

Xelafaslatide is an investigational, first-in-class synthetic peptide small-molecule Fas (CD95) inhibitor being developed by ONL Therapeutics for neuroprotection in retinal diseases, including geographic atrophy (GA) secondary to dry age-related macular degeneration, rhegmatogenous macula-off retinal detachment, and glaucoma.[1][2][5][11][12] By antagonizing the Fas receptor and blocking Fas–Fas ligand interaction, it is designed to prevent apoptosis of key retinal cells, particularly photoreceptors, with the goal of slowing lesion growth and preserving visual function when delivered by intermittent intravitreal injection.[1][2][4][5][9][11][12] Xelafaslatide (formerly ONL1204 ophthalmic solution) has completed phase 1 and 1b trials in GA, macula-off retinal detachment, and open-angle glaucoma, has orphan drug designation in the United States for macula-off retinal detachment, and is currently being evaluated in the global phase 2 GALAXY trial for GA associated with dry AMD.[2][5][6][8][11][12][13]

Other names
xelafaslatide
02

Targets

FASN (Fatty acid synthase)

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