ONL Therapeutics: Addressing Retinal Eye Disease

Dr. David Zacks in his Kellogg Eye Center lab on 6/29/09.

As a clinician and an associate professor of ophthalmology in the Kellogg Eye Center, Dr. David Zacks specializes in disorders of the retina. The problem that fascinates and perplexes him most is this: Even after receiving treatment for retinal disease, patients continue to lose vision. So what are the molecular mechanisms that cause the ongoing death of photo- receptors, those cells that capture light and convert it into brain signals?

Zacks and his research team found a possible answer. “When cell death occurs, a protein known as the Fas-receptor becomes activated,” he says. “By blocking activation of Fas-receptors with a 12-amino acid peptide, we discovered that we could preserve 80 percent of the photoreceptor cells that would normally die. And while the compound doesn’t restore vision, it does stop further damage.”

The idea of launching ONL Therapeutics began during early-stage conversations with Tech Transfer specialists and Mentor-in-Residence Thomas Collet. They warned us that it would be a long, arduous road,” Zacks says. “But we view this compound as a potential platform for stopping cell death in many diseases. And we were driven forward by the prospect of helping patients.” Working closely with Collet, Zacks put together a leadership team consisting of Raili Kerppola, former vice president of drug development at Bausch and Lomb, and Dr. Jeff Jamison, an expert in drug development from Kalamazoo. “I know the odds of success are relatively low for any start-up,” Zacks readily admits. “But I believe we’ll all come out winners, regardless—as human beings and as scientists who are pursuing translational research.”

ONL Therapeutics, now under the leadership of CEO John Freshley, is the first and only company focused on preventing the death of photoreceptors—the root cause of vision loss and the leading cause of blindness.

Of the long journey from research to market, Freshley explained, “None of these things come out of the lab ready. We need resources to validate the commercial opportunities, not just scientific opportunities.”

ONL Therapeutics benefitted from critical “gap” funding that generated the preliminary data that was instrumental in helping secure a $1.3 million Small Business Innovation Research (SBIR) award from the National Eye Institute. The company also secured $1 million in investment from a syndicate led by the Lansing, Mich.-based Capital Community Angels, with the Michigan Economic Development Corp-backed Biosciences Research and Commercialization Center at Western Michigan University, ONL management, and other investors.

“We wouldn’t have had the data we needed for the SBIR without University of Michigan’s support,” said Freshley “The smaller infusions of capital – investments that wouldn’t be attractive to anyone else – help you get through the early fires and there are always fires. Now, with more capital from investors and the SBIR, we have really been able to accelerate the program, including the identification of a new and improved drug candidate called ONL1204.”

“The basic science is the easy part,” Freshley added. “The hard part is being able to develop a product out of it.”

Next big thing on Freshley’s radar is to close a Series A for ONL. In the next year, he anticipates testing ONL’s new drug candidate in clinic trials.

“It’s clear that there’s an appetite for a company that protects people’s vision,” he said. “It’s about using the research to help patients see better, for longer, and everyone wins when that happens.”