The surgeons who treat it call it “the most common crippling hand disease you’ve never heard of”: Dupuytren's contracture, an incurable, sometimes painful disorder where cords of fibrotic tissue build up in the hand, causing the fingers to bend toward the palm.
“It’s so prevalent, but it hides in plain sight,” Kurt Harrington, a biotech industry veteran and founder of San Diego-based Ventoux Biosciences, told Fierce Biotech Research in an interview. About 5% of people in the U.S. have the disease, most of them over the age of 50. Harrington himself developed it much younger: Now in his 50s, his first symptoms showed up in his 30s. He’s since had multiple rounds of surgery and radiation to keep the condition at bay.
Those treatments and a drug called Xiaflex, marketed by Endo International, are the only therapies available to patients. Now, Ventoux is positioning itself to add another option. On March 12, the company announced that its lead candidate, a commercially available drug that the biotech has repurposed and renamed VEN-201, successfully reduces skin fibrosis in mice. Ventoux also revealed that it has launched a fundraising round to take the drug—the identity of which the biotech will not yet reveal—from preclinical research to a first-in-human study.
“It sounds corny, but I formed this company to literally try and make a difference,” Harrington said. He’s not the only one in his family who stands to benefit: His father has the disease, too, and just underwent his eighth surgery.
The results announced by Ventoux came from experiments on mouse models of scleroderma, a skin condition mechanistically similar to Dupuytren’s that causes tight, hard nodules to form in the skin. Daily injections of VEN-201 into the animals’ fibrotic tissue cut down on its thickness and the density of the collagen within it, as well as tissue remodeling overall, relative to untreated mice.
VEN-201 is a small molecule that already has FDA approval for other conditions; it has been used in more than 90 countries for over 20 years, according to the company. Ventoux is in the process of patenting it for use in Dupuytren’s and hasn’t yet disclosed the name of the repurposed drug. (If all goes according to plan, it’ll do so later this year, Harrington said.)
There are at least three or four potential targets that it could be affecting, and previous research shows that it has some antifibrotic activity in other disease models, Ventoux Chief Scientific Officer Ken Lipson, Ph.D., explained.
While the mouse model used in the study is far from a mirror image of Dupuytren’s, “it’s encouraging that we have been able to reproduce or confirm that the agent that we’re going to be using does show some antifibrotic activity,” he said.
Ventoux was completely self-funded until last November, when Harrington began fundraising among friends and family. To take their proof-of-concept drug from animals to humans, he’s hoping to raise between $1.2 million and upward of $1.5 million from investors. He believes that the prevalence of the condition not just in the U.S. but in European countries combined with a relatively unsaturated market make developing treatments for Dupuytren's a good bet.
“We routinely say that in the U.S. and Europe alone, there’s over 40 million patients,” he said. “The market is extremely big.”
Harrington’s resume includes stints at AstraZeneca, Biogen and leadership roles at smaller startups with exits. While working at Biogen as a senior marketing manager, he was part of the team that launched the rheumatoid arthritis drug Rituxan in partnership with Roche's Genentech. That experience gave him the insight to draw comparisons between the market for rheumatoid arthritis drugs and for those that could treat Dupuytren’s.
“When you think about rheumatoid arthritis and the number of treatments for RA, there’s three or four different classes of drugs and upwards of 16 medications. Many of them are disease-modifying drugs,” Harrington said. “There are zero disease-modifying drugs for Dupuytren’s and yet it has three and a half times the prevalence of RA.”
While Dupuytren’s isn’t life-threatening, the ramifications can be serious. Surgery, radiation and Xiaflex only stop symptoms for a while, meaning patients will inevitably have to have more treatment as it progresses. They sometimes resort to amputation to keep from going through the cycle over and over again.
“It’s one of the more common reasons for elective amputation,” Harrington said. “They just get so at the end of their rope that there’s nothing else, and their fingers are almost in the way.”
Harrington is clear that VEN-201 will not be a cure for Dupuytren's. However, unlike the other options, he hopes it can be used to keep the disease from reaching a point where more drastic measures are necessary.
“If we can have something available early on to help patients and slow the disease down, it’ll be wonderful,” he said.