ONL Therapeutics Receives U.S. FDA Clearance of Investigational New Drug Application for ONL1204 Ophthalmic Solution

First-in-class small molecule Fas inhibitor with unique mechanism of action is designed to provide neuroprotection for retinal cells

ANN ARBOR, MI – February 1, 2023

ONL Therapeutics, Inc., a clinical-stage biopharmaceutical company developing novel therapies for protecting the vision of patients with retinal disease, today announced the U.S. Food and Drug Administration (FDA) has authorized the company to proceed with the Investigational New Drug (IND) application for ONL1204 Ophthalmic Solution, a first-in-class small molecule Fas inhibitor designed as a neuroprotectant for key retinal cells. The company intends to initiate a Phase 2 clinical study of ONL1204 Ophthalmic Solution in patients suffering from macula-off rhegmatogenous retinal detachment (RRD) at sites in the U.S. in the second quarter of 2023.  

“The opening of our IND for ONL1204 allows us to take an important step forward in our mission to develop first-in-class therapeutics to protect the vision of patients with retinal diseases,” commented David Esposito, chief executive officer of ONL Therapeutics. “We look forward to initiating our U.S.-based Phase 2 study in acute retinal detachment in the next quarter. At the same time, we remain focused on continuing to generate additional clinical data for ONL1204 in chronic indications from our ongoing Phase 1b studies in patients with geographic atrophy associated with age-related macular degeneration and progressing open-angle glaucoma throughout this year.”  

“There is excitement in the retina community for a first-of-its-kind neuroprotection agent as an adjunct therapy for patients undergoing standard of care surgery for macula-off retinal detachment,” said David N. Zacks, M.D., Ph.D., and chief scientific officer of ONL Therapeutics. “Physicians recognize that there is a significant unmet medical need for a therapy that can help protect a patient’s photoreceptors during the critical period following diagnosis but before surgery, and the role that such a treatment may play in potentially delivering better visual outcomes following reattachment surgery.”

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 macular-off retinal detachment, 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), both being conducted 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). In addition, the company continues to advance a novel gene therapy approach for Fas inhibition.   

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. ONL is developing a platform of products for use in a wide range of blinding diseases, including retinal detachment, glaucoma, AMD and IRD. 

For more information about ONL Therapeutics, please visit

Company Contact

ONL Therapeutics, Inc.
Linda Kemnitz

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