ONL Therapeutics, Inc., a clinical-stage biopharmaceutical company developing novel therapies for protecting the vision of patients with retinal disease, today announced that the first patient has been dosed in its Phase 2 clinical trial of ONL1204 Ophthalmic Solution in patients with macula-off rhegmatogenous retinal detachment (RRD).
The ongoing Phase 2 clinical trial is a randomized, controlled study designed to enroll 135 patients across three arms, including two treatment groups and a sham group. The goal of the study is to evaluate the safety and efficacy of a single intravitreal (IVT) injection of ONL1204 as an adjunct to standard-of-care surgical repair in subjects with macula-off RRD at approximately 30 sites across the United States.
“We are excited to reach this important milestone in bringing ONL1204 to patients and clinicians in the United States for the first time,” said David Zacks, M.D., Ph.D., co-founder and chief scientific officer of ONL Therapeutics. “With its unique and differentiated mechanism of action targeting the Fas activation pathway, we look forward to advancing our clinical development for ONL1204 as a potential breakthrough neuroprotection therapy to address unmet medical needs across a number of disease areas including RRD.”
In addition, the company will continue its focus on generating interim data and completing its ongoing Phase 1b studies in the chronic indications of geographic atrophy associated with age-related macular degeneration and progressing open-angle glaucoma that are being conducted at sites in Australia and New Zealand.
About ONL1204 Ophthalmic Solution
ONL1204 is a novel, 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 ONL1204 currently includes a Phase 2 study in the U.S. for the treatment of macula-off retinal detachment (NCT05730218), a condition for which the compound has been granted orphan drug designation by the United States Food and Drug Administration (FDA). The company is also conducting a Phase 1b clinical trial in patients with geographic atrophy (GA) associated with age-related macular degeneration (AMD) (NCT04744662) and a Phase 1b clinical trial in patients with progressing open-angle glaucoma (NCT05160805) at sites in Australia and New Zealand. Preclinical work is ongoing to enable clinical trials in other disease indications, including inherited retinal degeneration (IRD; also known as retinitis pigmentosa).
ONL Therapeutics, Inc.