ANN ARBOR, Mich., Dec. 09, 2025 (GLOBE NEWSWIRE) -- ONL Therapeutics, Inc., a clinical-stage biopharmaceutical company developing novel therapies for protecting the vision of patients with retinal disease, today announced the publication of data from its Phase 1b study of xelafaslatide (formerly ONL1204 Ophthalmic Solution) in Ophthalmology Science, the peer-reviewed journal of the American Academy of Ophthalmology.
Xelafaslatide is an investigational, first-in-class small molecule Fas inhibitor designed to protect key retinal cells, including photoreceptors, from cell death that occurs across a range of retinal diseases and conditions, including geographic atrophy (GA) associated with dry age-related macular degeneration (AMD).
Published data from the Phase 1b study demonstrated xelafaslatide to be generally safe and well tolerated. Additional data assessments showed efficacy signals observed after six months, with xelafaslatide-treated eyes slowing GA lesion growth compared to untreated eyes. ONL continues to progress clinical development for its lead compound, having recently initiated GALAXY, a global Phase 2 clinical trial designed to evaluate the efficacy and safety of xelafaslatide in patients with GA.
“Geographic atrophy remains one of the most devastating challenges in ophthalmology,” said David N. Zacks, M.D., Ph.D., chief scientific officer of ONL Therapeutics. “With its unique mechanism of action targeting the Fas pathway and dosing every three or six months, xelafaslatide has the potential to protect vision while easing the treatment burden for patients. Our ultimate goal is to advance this neuroprotection therapy and redefine what is possible for patients living with this progressive disease.”
The Phase 1b multicenter study included a dose-escalation/open-label (DE/OL) component (N=6) and a randomized, double-masked, sham-controlled natural history/treatment (NHS/T) component (N=22). DE/OL participants received a single intravitreal injection of either 50 μg, 100 μg, or 200 μg of xelafaslatide and were followed for 24 weeks. Participants in the NHS/T component were randomized (1:1:1) to either 50 μg or 200 μg of xelafaslatide or sham injection, after a 24-week NHS phase. Two injections were administered 12 weeks apart, and patients were followed for an additional 12 weeks.
The primary endpoint of the study was safety and additional endpoints included measurement of GA lesion area and best-corrected visual acuity. Results from the study support continued evaluation of xelafaslatide as a potential treatment for patients with GA.
About Xelafaslatide (ONL1204 Ophthalmic Solution)
Xelafaslatide is an investigational first-in-class small molecule Fas inhibitor designed to protect key retinal cells, including photoreceptors, from cell death that occurs across a range of retinal diseases and conditions. Death of these retinal cells, through both direct and inflammatory signaling pathways, is the root cause of vision loss and the leading cause of blindness. The company’s later stage clinical development program for xelafaslatide includes a Phase 2 study for the treatment of GA associated with AMD (NCT06659445) and a completed Phase 2 study in the U.S. for the treatment of macula-off retinal detachment (RD) (NCT05730218), a condition for which the compound has been granted orphan drug designation by the United States Food and Drug Administration (FDA). The company has also completed a Phase 1b clinical trial in patients with GA associated with AMD (NCT04744662), a Phase 1b clinical trial in patients with progressing open-angle glaucoma (NCT05160805) and a Phase 1 clinical trial in macula-off RD patients at sites in Australia and New Zealand (NCT03780972).
About Geographic Atrophy (GA) Associated with Dry Age-related Macular Degeneration (AMD)
AMD has become a major cause of visual disability and legal blindness globally. Although generally affecting only the central retina (macula), this region of photoreceptors provides the visual acuity necessary for reading, driving, and the performance of fine vision-related tasks. Associated with aging, cigarette smoking, obesity, diets low in certain nutrients, a lifestyle related to cardiac risk, and a growing list of genetic factors, AMD is becoming an increasingly prevalent public health concern, especially as the global population ages. GA, also called atrophic AMD, is an advanced form of AMD.
About ONL Therapeutics
ONL Therapeutics (ONL) is a clinical-stage biopharmaceutical company committed to developing first-in-class therapeutics to protect and improve the vision of patients with retinal disease. By advancing a breakthrough technology designed to protect key retinal cells from Fas-mediated cell death, ONL is pioneering a new approach to preserving vision.
For more information about ONL Therapeutics, please visit www.onltherapeutics.com.
