ONL Therapeutics Completes Enrollment in Phase 2 Study of First-in-Class Fas-Inhibitor in Patients with Macula-Off Rhegmatogenous Retinal Detachment

More than 135 patients recruited in four months for Phase 2 study of a single intravitreal (IVT) injection of ONL1204 Ophthalmic Solution as adjunct to standard-of-care surgery

Topline data readout expected in 1H2024

ANN ARBOR, Mich., Aug. 28, 2023 (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 completion of patient enrollment 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 assess 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 37 sites across the United States.

“Completing enrollment of over 135 patients in this U.S.-based Phase 2 study is a critical milestone as we continue our efforts to advance ONL1204 for patients suffering from retinal diseases such as RRD,” said David Esposito, chief executive officer of ONL Therapeutics. “We look forward to sharing topline results from this study in the first half of next year and to completing our Phase 1b clinical studies in the chronic indications of geographic atrophy associated with age-related macular degeneration and progressing open-angle glaucoma, both of which are being conducted at sites in Australia and New Zealand.”

“We are pleased with how quickly our participating clinical sites were able to recruit for our Phase 2 RRD study. The level of enthusiasm and partnership from our sites has been tremendous as we work toward our common goal of bringing a novel neuroprotection therapy to patients suffering from RRD,” said David Zacks, M.D., Ph.D., co-founder and chief scientific officer of ONL Therapeutics. “We are extremely grateful to the patients, families and investigators involved for their dedication and commitment to this study.”
    
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).

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 www.onltherapeutics.com.

CONTACT: Company Contact:
Linda Kemnitz
ONL Therapeutics, Inc.
lkemnitz@onltherapeutics.com