ONL Therapeutics, the Ann Arbor, MI-based biotech startup working on sight-preserving therapies for patients with retinal diseases, is one step closer to clinical trials, thanks to a cash infusion from the National Eye Institute (NEI).
Last week, the NEI awarded ONL a $1 million Commercialization Readiness Pilot grant to continue advancing ONL1204, the company’s lead drug target, toward human testing.
According to CEO John Freshley, ONL1204 is a small-molecule peptide designed to protect critical retinal cells, including photoreceptors, from the damage caused by a range of retinal diseases and conditions. When these retinal cells die, the patient experiences a loss of vision. ONL1204 inhibits retinal cell death, the leading cause of blindness, and preserves vision for an extended period of time, he adds.
The NEI grant is “very focused” on moving ONL1204 forward, Freshley says, and the company is planning to use the grant money to finish the pre-clinical development work necessary to apply for its first human study. ONL’s lead compound has already received orphan designation from the FDA; it is initially being developed for the treatment of retinal detachment, but Freshley says ONL is also exploring its use in the treatment of other conditions, such as glaucoma and age-related macular degeneration. He hopes to start the first phase of human clinical trials in 18 to 24 months.
The NEI grant comes a few months after the company raised more than $1 million in interim financing from both new and existing investors, including Invest Michigan and Novartis. Cynthia Grosskreutz, the director of global translational medicine for ophthalmology at Novartis, has joined ONL’s board of directors as an observer as part of the investment. Freshley says ONL is in the midst of converting the interim financing into a Series A round.
ONL previously scored Phase I and Phase II Small Business Innovation Research (SBIR) awards, and Freshley says this time around, the company applied for the NEI grant at the suggestion of one of the SBIR program’s administrators. The Commercialization Readiness Pilot program, overseen by the federal government, has so far not been renewed.
“It’s a bit of a surprise,” Freshley says. “[The SBIR administrator] has implored us to be successful—to validate the value of the program so Congress will renew it.”