ONL Therapeutics randomizes first patient in phase 2 GALAXY trial evaluating xelafaslatide

ONL Therapeutics announced the first patient has been randomized in its phase 2 GALAXY (NCT06659445) trial evaluating xelafaslatide (formerly ONL1204) in patients with geographic atrophy (GA) associated with dry age-related macular degeneration (AMD).

According to the company, xelafaslatide is a small-molecule Fas inhibitor designed to protect key retinal cells, including photoreceptors, from cell death that occurs across multiple retinal diseases and conditions. The company noted that xelafaslatide is the newly established nonproprietary name for ONL1204 Ophthalmic Solution following approval from the World Health Organization and the United States Adopted Names Council.

GALAXY will enroll approximately 324 patients across sites in the United States, Canada, and the European Union. Xelafaslatide will be delivered via intravitreal injection across 3 arms, which include 2 dose levels and 2 treatment frequencies (every 12 weeks or every 24 weeks). The primary endpoint is the rate of growth of the GA lesion area in patients treated with xelafaslatide versus sham as assessed by fundus autofluorescence (FAF) measured at 48 weeks. The company also stated that additional timepoints will be measured out to 72 weeks.

David R.P. Almeida, MD, MBA, PhD, FRCSC, executive chairman of Erie Retina Research in Erie, Pennsylvania, and David N. Zacks, MD, PhD, chief scientific officer of ONL Therapeutics, commented on the phase 2 trial in a press release from the company.

“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,” said Almeida.¹

“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,” added Zacks.¹

GALAXY builds upon the company’s phase 1b study (NCT04744662) that demonstrated xelafaslatide to be generally safe and well tolerated. The 1b study enrolled and followed patients for 24 weeks to assess the growth rate of their lesions. They were then randomized to receive either a low dose (50 micrograms), a high dose (200 micrograms), or sham injection.³ The trial enrolled 28 patients in Australia and New Zealand.²

References:

1. ONL Therapeutics announces randomization of first patient in global phase 2 GALAXY trial of Xelafaslatide (ONL1204) in patients with geographic atrophy (GA) associated with Dry AMD. Published October 28, 2025. Accessed October 28, 2025. https://www.globenewswire.com/news-release/2025/10/28/3175222/0/en/ONL-Therapeutics-Announces-Randomization-of-First-Patient-in-Global-Phase-2-GALAXY-Trial-of-Xelafaslatide-ONL1204-in-Patients-with-Geographic-Atrophy-GA-Associated-with-Dry-AMD.html

2. ONL1204 Ophthalmic Solution in Patients with Geographic Atrophy Associated with Age-related Macular Degeneration. NCT04744662. Accessed October 28, 2025. https://clinicaltrials.gov/study/NCT04744662?tab=table

3. Borkar D. Crago SM. ASRS 2024: Fas inhibition with ONL1204 for the treatment of geographic atrophy. Published July 18, 2024. Accessed October 28, 2025. https://www.ophthalmologytimes.com/view/asrs-2024-fas-inhibition-with-onl1204-for-the-treatment-of-geographic-atrophy