The body’s natural process for healing wounds has led AsclepiX Therapeutics to a novel approach to treating diseases of the eye. The startup is now gearing up to test its technology in humans, and it has raised $35 million in Series A financing to support its research.
Cuts and scrapes in the skin spark the formation of new blood vessels and connective tissue that fills the wound. But this process, called angiogenesis, does not continue indefinitely. Within days the body returns to a state of equilibrium, says AsclepiX interim CEO Kevin Slawin.
Forming new blood vessels is important for wound healing, but this process can also play a role in certain diseases. In the eye, angiogenesis contributes to several vision-robbing disorders. The scientific founders of Baltimore-based AsclepiX discovered a peptide that turns off angiogenesis in wounds, and the biotech now has a pipeline of experimental drugs intended to use that approach to treat eye disorders and more.
Lead compound AXT107 is being developed as a treatment for three eye disorders: diabetic macular edema, the “wet” form of age-related macular degeneration, and retinal vein occlusion. The standard of care for these diseases includes biological drugs that bind to and block vascular endothelial growth factor (VEGF), a protein that contributes to blood vessel growth.
Approved anti-VEGF products include Roche’s ranibizumab (Lucentis) and aflibercept (Eylea) from partners Regeneron Pharmaceuticals (NASDAQ: REGN) and Bayer. Both are blockbuster drugs, but the treatments are delivered via an injection into the eye as frequently as once a month—a daunting prospect for some patients.
The AsclepiX peptides work in a different way, binding to a different target. VEGF signaling relies on co-receptors called integrins. Binding to them completely blocks VEGF signaling, Slawin says. The drug also offers a second mechanism of action, activating Tie2, another validated pathway for addressing vascular diseases of the eye. In preclinical testing, Slawin says the AsclepiX drug was as strong or stronger than VEGF inhibitors. He adds that because these peptides naturally evolved with humans, they are less likely to cause side effects, such as inflammation.
The AsclepiX drug could also offer a dosing advantage. In the normal physiological pH of the eye, the injectable drug becomes a gel that provides a sustained release of its therapeutic benefit, says Theresa Heah, chief medical officer and executive vice president of operations at AsclepiX.
“It’s not a drug formulation, it’s the drug itself,” she says. “The peptide forms a gel.”
Heah, who earlier in her career worked on the Bayer team that launched aflibercept, says this gel’s release over a span of months means it’s possible the AsclepiX drug could offer once-a-year dosing.
Asclepix isn’t alone in the hunt for anti-VEGF drugs that can be dosed less frequently. Palo Alto, CA-based Kodiak Sciences (NASDAQ: KOD) is developing antibody drug KSI-301 as a potential treatment for wet AMD. The drug is currently in Phase 2/3 testing.
The AsclepiX peptide drugs are based on technology licensed from Johns Hopkins University where cofounders Aleksander Popel and Jordan Green are professors in the school of medicine. The pair developed computational techniques to screen the human proteome for peptides.
Heah says a clinical trial testing AXT107 could start by the end of this year. Angiogenesis also plays a role in cancer, and she notes that AsclepiX has three earlier-stage candidates being developed for treating solid tumors. The company’s approach also has potential applications addressing inflammation.
The latest round of investment was led by Perceptive Xontogeny Venture Fund (PXV Fund) and included participation from the Perceptive Life Sciences Fund, both of which are managed by Perceptive Advisors. Earlier investors Rapha Capital Management and Barer & Son Capital also invested. AsclepiX is the fifth announced investment from PXV Fund since it launched last December, says Chris Garabedian, the fund’s manager.
“While we were intrigued by the mechanism, what won us over was the 10 studies in the animal models,” he says. “It proved to us the efficacy and the safety, and the likelihood that it would perform in patients in the clinic.”