The small Cambridge, MA-based company Dart Therapeutics has been racing along the startup track in recent months. In September, it assembled a veteran executive team headed by CEO Gene Williams, a Genzyme veteran. This month, it in-licensed its second drug candidate for Duchenne Muscular Dystrophy, and made a deal that might identify other potential medicines against the disabling and deadly neuromuscular disease.
Dart is becoming a proving ground, not only for experimental drugs, but also for a new way of creating a drug development company. It’s one of the few enterprises founded by non-profit disease foundations to find therapies for specific illnesses. In Dart’s case, the two founding groups include parents of the young boys who will likely die in early adulthood from Duchenne Muscular Dystrophy (DMD) unless researchers can improve on current treatments.
Bringing a sense of urgency to such quests, patients’ foundations that once simply donated to drug firm research are now moving into business roles themselves—seeking a return on their investment in company R&D, setting up venture funds, and even starting biotechnology firms.
“They’re thinking so much differently from patient education and patient support groups,” says Elliot Goldstein, an experienced drug industry executive who joined Dart as chief operating officer in August.
Dart’s non-profit co-founders, Charley’s Fund and the Nash Avery Foundation, also financed the formation of a Dart affiliate, Halo Therapeutics of Newton, MA. In May, the two non-profits assembled a “syndicate” of 12 foundations that pitched in $1.1 million to support early-stage work on Halo’s drug candidate HT-100. The pill contains halofuginone, a potential remedy for the malformed muscle fibers and inflammation associated with Duchenne Muscular Dystrophy. Goldstein says Halo will eventually be absorbed by Dart, which shares much of the same leadership.
Dart announced March 20 that it has acquired rights from Belgium-based Galapagos NV to a compound it renamed as DT-200, a possible therapy that could rebuild muscle size and strength. On Tuesday, Dart announced a collaboration with Charlottesville, VA-based Biovista, a pharmaceutical data-mining company that will search for drugs Dart can develop for DMD.
With two drugs headed for clinical trials, DT-200 and HT-100, and an appetite for more pipeline candidates, Dart is now looking for Series B funding from venture firms and other investors outside the disease foundation realm, Goldstein says. As the expenses of clinical testing rise, Dart will need to find partners with deeper pockets who can bring drugs through late-stage clinical testing and to the market, he says.
“If you can’t get that kind of investor in, you’re going to have a problem,” Goldstein says.
Drug industry observers have wondered whether a mission-driven biotech company owned by patients’ foundations could mesh with
