ONL Therapeutics Randomizes First Patient in Phase 2 GALAXY Trial for Geographic Atrophy

ONL Therapeutics has announced the randomization of the first patient in its Phase 2 GALAXY clinical trial (NCT06659445) evaluating xelafaslatide (formerly known as ONL1204) for the treatment of geographic atrophy (GA) associated with dry age-related macular degeneration (AMD).

Xelafaslatide: A Novel Fas Inhibitor for Retinal Neuroprotection

Xelafaslatide is a small-molecule Fas inhibitor designed to prevent cell death in retinal cells, including photoreceptors, which are critical for vision. According to ONL Therapeutics, the therapy aims to provide neuroprotective benefits across a range of retinal conditions by targeting the Fas signaling pathway, a key mechanism involved in retinal cell death.

The compound’s nonproprietary name, xelafaslatide, was recently approved by both the World Health Organization and the United States Adopted Names Council. The formulation under evaluation is ONL1204 Ophthalmic Solution, delivered via intravitreal injection.

About the GALAXY Phase 2 Trial

The GALAXY trial is set to enroll approximately 324 patients across clinical sites in the United States, Canada, and the European Union. The study will assess xelafaslatide’s safety and efficacy using three treatment arms, which include two dose levels and two dosing intervals, either every 12 weeks or every 24 weeks.

The trial’s primary endpoint is the rate of GA lesion growth, as measured by fundus autofluorescence (FAF) at 48 weeks. Additional data will be collected up to 72 weeks to evaluate long-term outcomes.

Expert Commentary and Clinical Potential

Dr. David R.P. Almeida, Executive Chairman of Erie Retina Research, highlighted the potential of xelafaslatide to improve GA treatment outcomes while reducing the frequency of injections:

“With its novel therapeutic pathway targeting Fas and dosing every three to six months, xelafaslatide has the potential to make a significant positive impact for patients while also lowering the treatment burden associated with currently approved GA therapies.”

Dr. David N. Zacks, Chief Scientific Officer at ONL Therapeutics, emphasized the significance of this milestone:

“The continued support of the retina specialist community for the GALAXY trial underscores the strong interest in xelafaslatide and its unique and differentiated mechanism of action targeting Fas. We are committed to advancing xelafaslatide as a potential breakthrough neuroprotection therapy for GA to help clinicians address the needs of patients facing this progressive, vision-threatening disease. Enrolling the first patient is a critical step in our search for more effective and durable GA treatments.”