ONL Therapeutics, a University of Michigan spin-out working on sight-preserving therapies for retinal diseases, announced that it has changed course in its drug development program. The company has promoted a new lead drug candidate and has expanded its planned clinical studies to include both dry and wet forms of age-related macular degeneration (AMD).
John Freshley, ONL’s president and CEO, said the new lead drug, ONL1204, is an analog of the old lead, ONL101, which is a small-molecule peptide that inhibits the death of photoreceptor cells. The new compound is a more attractive candidate for development, Freshley added, because it’s 10 times as potent, in tests done in a petri dish and in animal models. The company expects clinical trials to go forward as planned early next year.
“It was unexpected,” Freshley said of the test results. The new drug candidate “has much better properties for formulation and clinical use. ONL101 is still viable, but 1204 is so much better. We caught it at just the right time, before we had invested too much money in 101.”
Another factor in deciding to promote a new lead drug, Freshley explained, was ONL101’s lack of “composition of matter” intellectual property, meaning the company wasn’t able to patent the molecule itself. “We patented the use of the molecule in the eye to treat diseases,” he said, noting ONL101’s orphan drug designation in retinal detachment. “That’s good, but investors prefer the actual molecule patented.”
In the past few months, ONL has generated data in two distinct dry AMD in vivo models demonstrating that its drug candidate protects both photoreceptors and the underlying retinal pigment epithelium (RPE) layer of cells.
“If we confirm that, it’s a potential game-changer,” Freshley said, adding that it would immediately open up a market worth approximately $9 billion, as dry AMD affects millions of patients. “There are no similar drugs currently on the market. There are a couple of others in development, but they target completely different pathways.”
Dry AMD, because its biology is so complex, has “killed many biotech companies,” Freshley said. He said Genentech had some success last year with an anti-inflammatory drug that was able to reduce the death of RPE by approximately 20 percent in clinical trials, which Freshley described as only modest improvement.
“We think if our data confirms what we’ve found so far, we can stop cell death and protect vision for an extended period of time,” he said. “We’ve really accelerated our development plan and changed our investors’ perspective.”
Freshley said what’s clear to him is that there’s a big appetite in the market for a successful vision-protection company. “We have an opportunity to build an important and impactful company around vision protection, and I think that’s what we’ll do,” he said.