ONL Therapeutics, a clinical-stage biopharmaceutical company based in Ann Arbor, Michigan, has launched a Phase II study for geographic atrophy (GA), a late-stage form of age-related macular degeneration (AMD). The company focuses on developing therapies to protect and improve vision for patients with retinal diseases, such as AMD and retinal detachment. The drug being tested, ONL1204, is designed to block the activation of the Fas receptor, a protein on the surface of cells that acts as a type of death switch. “This pathway is significant because Fas is a very upstream regulator of cell death and inflammation, and its activation drives disease and cell death in the retina,” says David Zacks, M.D., Ph.D., Edna H. Perkiss Research Professor of Ophthalmology and Visual Sciences; Professor of Ophthalmology and Visual Sciences; and Co-Founder and Chief Scientific Officer of ONL Therapeutics. “This dual action on both cell death and inflammation may address important contributors to worsening disease and vision loss, making our approach distinctive.” Although two approved agents for GA are currently available in the United States, there remains a significant unmet need for novel therapies. ONL’s approach seeks to fill gaps that current treatments do not address. “Conducting a Phase II trial is essential because it allows us to gain more experience in a larger number of patients and to determine the exact dosing required,” says Dr. Zacks. “This will then inform further clinical trials for ultimate approval by the regulatory agencies.” Dr. Zacks emphasizes that he is immensely grateful to the U-M Kellogg Eye Center and Michigan Medicine for enabling this research, supporting the foundational bench research, and assisting in advancing the therapy through clinical trials.