FDA clears IND application from ONL Therapeutics for ONL1204 ophthalmic solution

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. (Adobe Stock image)

ONL Therapeutics Inc. today announced the 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.

According to a news release, 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.

David Esposito, CEO of ONL Therapeutics, pointed out that the opening of the company’s IND for ONL1204 allows it to take an important step forward in its goal of developing “first-in-class therapeutics to protect the vision of patients with retinal disease. “

“We look forward to initiating our U.S.-based Phase 2 study in acute retinal detachment in the next quarter,” he said in a statement. “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.”

Moreover, David N. Zacks, MD. PhD. chief scientific officer of ONL Therapeutics, noted that the drug candidate is drawing praise in the retina community as an adjunct therapy for patients undergoing standard of care surgery for macula-off retinal detachment.

“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,” he said.

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.

According to the company, its 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 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.