ONL Therapeutics Receives NEI Grant to Help Advance ONL1204 Program

ONL Therapeutics, Inc., a biopharmaceutical company developing novel therapies for preserving sight in a range of retinal diseases, today announced that the company has been awarded a Commercialization Readiness Pilot (CRP) grant of approximately $1.0 million from the National Eye Institute (NEI) to continue advancement of ONL1204, the company’s lead therapeutic candidate. ONL1204 is a novel, first-in- class small molecule Fas inhibitor designed to protect key retinal cells, including photoreceptors, from cell death that occurs in a range of retinal diseases and conditions. Death of these retinal cells, both direct and via inflammatory signaling, is the root cause of vision loss and the leading cause of blindness.

ONL intends to use the funding from this CRP grant to support the remaining ONL1204 preclinical development activities required for submission of an investigational new drug (IND) application. The company is initially developing ONL1204 for the treatment of retinal detachment, a condition for which the compound has been granted orphan drug designation by the United States Food and Drug Administration (FDA). While initial development efforts are focused on retinal detachment, preclinical in vivo data along with a growing body of literature support potential application in several additional ocular diseases including age related macular degeneration (AMD).

ONL has previously received Phase I and Phase II Small Business Innovation Research (SBIR) research grants from the NEI that have helped support the company’s preclinical development work to date. This includes establishment of proof-of- concept for Fas inhibition in rodent models of retinal detachment, the identification and formulation of ONL1204, and initial pharmacokinetic and toxicology research for the compound. The CRP program which has awarded the latest grant is designed to provide additional financial resources to assist companies in transitioning previously funded SBIR Phase II projects to the commercialization stage.

“We are excited and grateful for this additional NEI grant funding as it allows ONL to build upon the promising preclinical work that we have conducted to date. The area of retinal cell protection represents a critical unmet medical need that we believe can have an important impact on the preservation of vision for patients with a range of ocular diseases and conditions,” said John Freshley, chief executive officer of ONL Therapeutics. “By providing the funds that will help us advance ONL1204 to the point of IND filing, the NEI is playing a key role in research that may one day make a difference in the lives of ocular disease patients. We appreciate their support and look forward to completing the important work that this grant is making possible.”

In addition to the CRP grant, ONL has also recently raised more than $1.0 million through an interim financing involving both existing and new investors, including Novartis and Invest Michigan. Dr. Cynthia Grosskreutz, Global Translational Medicine Head for Ophthalmology at Novartis joins the ONL Board of Directors as an observer. These funds will also be used to complete IND-enabling activities and advance ONL1204 toward human clinical trials. “The CRP grant, renewed commitment from our existing investors, and new commitment from investors such as Novartis validates the clinical and commercial potential of our technology and provides ONL access to unique ocular drug development expertise,” stated Mr. Freshley. “The combined funding will accelerate our development efforts aimed at moving ONL1204 into the clinic.”