ONL Therapeutics

ONL Therapeutics is a clinical-stage biopharmaceutical company dedicated to developing novel therapies for protecting and improving the vision of patients with retinal diseases. The company's primary focus is on advancing a breakthrough technology designed to protect key retinal cells from Fas-mediated cell death, which is the root cause of vision loss and blindness across various retinal conditions. ONL Therapeutics' lead product, ONL1204 Ophthalmic Solution, is a first-in-class small molecule Fas inhibitor designed as a neuroprotectant for key retinal cells, including photoreceptors.

ONL1204 is being developed for multiple retinal conditions. In February 2023, the US Food and Drug Administration (FDA) authorized the company to proceed with an Investigational New Drug (IND) application for ONL1204 Ophthalmic Solution. The company initiated a Phase 2 clinical study of ONL1204 in patients with macula-off rhegmatogenous retinal detachment (RRD) in the US in April 2023. This randomized, controlled study aims to enroll 135 patients across three arms, evaluating the safety and efficacy of a single intravitreal injection of ONL1204 as an adjunct to standard-of-care surgical repair.

In addition to RRD, ONL Therapeutics is conducting Phase 1 billion clinical trials for ONL1204 in patients with geographic atrophy (GA) associated with age-related macular degeneration (AMD) and progressing open-angle glaucoma in Australia and New Zealand. Preliminary results from the Phase 1 billion trial in GA associated with dry AMD showed promising outcomes, with patients treated with ONL1204 demonstrating reductions in the rate of growth of the GA lesion after six months of treatment compared to sham patients. The company is also exploring the potential of ONL1204 in other retinal conditions, including inherited retinal degeneration (IRD), also known as retinitis pigmentosa.

ONL Therapeutics is developing a platform of products for use in a wide range of blinding diseases, including retinal detachment, glaucoma, AMD, and IRD. The company's unique approach to preserving vision through Fas inhibition has the potential to address significant unmet medical needs in the field of ophthalmology.