At first glance, the $1.1 million financing announced by Halo Therapeutics on Tuesday looked like your average case of a biotech startup scraping together enough money to get its early-stage compound into pivotal human trials. But this funding was anything but average. That’s because the money came from 12 nonprofit foundations pursuing a common goal: They all want to find new treatments for Duchenne Muscular Dystrophy (DMD), a rare but debilitating muscle disease that leads to early death in young boys. And they all believe Halo might have a promising drug candidate—an experimental pill whose main ingredient was derived from an herb that’s been used in Chinese medicine since 4730 B.C.
Before we review this unusual story of drug development, let’s take a closer look at this week’s news. Halo’s new round of financing consists of traditional grants, plus notes that offer a fixed return to the nonprofits based on specific milestones that Halo achieves while developing the drug, called HT-100. The company expects to begin mid-stage human trials in the second half of this year. “If Halo adds value and we can repay that money, we want to offer [the groups] the opportunity to use it to develop other therapeutics,” says CEO Marc Blaustein. “We want to put the value back into the patient community.”
Halo itself originated from that patient community. It was founded by Minneapolis-based Nash Avery Foundation and Stockbridge, MA-based Charley’s Fund, two groups dedicated to accelerating research and drug development in DMD. Benjamin Seckler, a physician and president of Charley’s Fund, was scouring the medical literature a few years back looking for remedies that might help his son, Charley, age 11. He came across halofuginone, a drug being developed by an Israeli company called Collgard Biopharmaceuticals.
The drug’s active ingredient, made from the Chinese herb dichroa febrifuga, had been tested by the U.S. military as an anti-malaria compound in the 1940s and later was synthesized by the pharmaceutical industry. Blaustein, who is the company’s sole full-time employee, says that experiments in mouse models of DMD suggested that Collgard’s compound has three key attributes: It fights fibrosis, which is a harmful buildup of collagen in muscles. It reduces the inflammation that causes muscle damage and degradation. And it seems to promote muscle regeneration. “We hope this will enhance the body’s natural repair mechanisms,” Blaustein says. Halo bought the drug from Collgard in March of last year.
Blaustein says it wasn’t difficult to get a dozen patient organizations on board for the company’s funding. “This is a very educated, very committed patient-advocacy community,” he says. “Universally they’re all parents of boys suffering from this terrible disease who are very active in reaching out to the scientific community.” The backers come from all over the U.S. and U.K., and many of the foundations are named for children who have suffered from DMD. They include Michael’s Cause in Bellevue, NY, Ryan’s Quest in Hamilton, NJ, and Zubin’s Wish in Seattle.
Several other companies are working on different approaches to attacking DMD. New York based Retrophin, for example, is developing an engineered version of the protein that’s lacking in patients who have the disease. And Bothell, WA-based AVI Biopharma is working on a drug that attempts to correct the genetic defect underlying DMD. Blaustein says the more companies searching for therapies, the better. “Our hope is we’ll end up with a cocktail approach,” he says. “We need to address different parts of the pathology of the disease so these boys can get as close to normal functioning as possible.”
Collgard completed several safety trials in people before transferring its drug to Halo, Blaustein says. He hopes the company will complete the Phase 2 efficacy trials within two years. He adds that he’s actively looking for “strategic financial or pharmaceutical partners” and has generated interest from some drug companies already.
For now, Halo has plenty of resources to take HT-100 into the next stage of testing, thanks to the pooled resources of the patient advocacy groups. Blaustein says he’s not aware of another case of so many nonprofits coming together to fund a single drug, but he won’t be surprised if more such arrangements emerge. “We all know we need to find new models that work to get innovative therapies out to patients.” he says. “We think this is one model that holds some real promise.”
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