ONL Therapeutics Provides Update on Novel Photoreceptor Protection Platform

ONL Therapeutics, Inc. recently provided an update on the company’s ongoing drug development program to block the programmed cell death (apoptosis) of photoreceptors. Death of photoreceptors, which occurs in a range of retinal diseases, is the root cause of vision loss and leading cause of blindness. The company’s recent research and development efforts have led to a number of key milestones, including the promotion of a new lead development candidate and the expansion of its planned clinical program to include both dry and wet forms of age-related macular degeneration (AMD) based on promising preclinical in vivo data.

As a result of ongoing research, ONL has recently identified and patented a novel, first-in-class small molecule peptide, ONL1204, with an attractive profile for inhibiting the Fas pathway, a primary mediator of photoreceptor apoptosis. Researchers have successfully demonstrated the link between Fas pathway activation and a number of retinal diseases and conditions, including retinal detachment and both wet and dry AMD. ONL1204 is an analog of Met12, ONL’s first-generation Fas inhibitor, and has demonstrated significantly enhanced potency both in vitro and in vivo as compared to Met12, while also displaying improved pharmaceutical properties. Based on these significant attributes, ONL has promoted ONL1204 to its lead development candidate and intends to advance the compound into clinical trials in retinal detachment in the first half of 2016.

While initial development efforts are focused on retinal detachment, ONL has also recently generated preliminary data in two distinct dry AMD in vivo models demonstrating that its novel Fas inhibition platform protects both photoreceptors and the underlying retinal pigment epithelium (RPE). This is consistent with a growing consensus in the scientific literature about the role of the Fas pathway in dry AMD. Research is increasingly demonstrating that in dry AMD, Fas not only plays a role in photoreceptor death, but also in the death of the RPE that causes Geographic Atrophy (GA).

Based on the company’s new data in AMD, ONL is expanding its preclinical and clinical development planning to validate its findings and support initiation of clinical studies in both dry and wet AMD. This work will be conducted concurrently with ONL’s clinical development in retinal detachment. Combined, the estimated market for these initial indications that ONL plans to target is greater than $12 billion globally.

“We are excited about the important recent advances we have made in connection with our novel photoreceptor protection technology program. ONL1204 is a potent molecule that displays an excellent profile as a therapeutically relevant Fas inhibitor with applications to address key unmet needs of retinal disease patients,” said John Freshley, ONL’s President and Chief Executive Officer. “Our new data in models of dry AMD is particularly exciting as this disease has such extreme unmet needs and protection of RPE would represent a true therapeutic breakthrough.”

ONL Therapeutics (ONL) is a biopharmaceutical company committed to protecting and improving the vision of patients with retinal disease. By advancing a novel breakthrough technology designed to protect photoreceptors against apoptosis, ONL is pioneering an entirely new approach to preserving sight. The death of photoreceptors, which is the root cause of vision loss and leading cause of blindness worldwide, is implicated in a wide range of retinal diseases.

ONL is advancing a novel, first-in-class, small molecule peptide for the protection of photoreceptors. While initial development efforts are focused on retinal detachment, a condition for which the company has been granted orphan drug designation, preclinical in vivo data along with a growing body of literature support potential application in age related macular degeneration (AMD) and other chronic retinal diseases. For more information, visit www.onltherapeutics.com.