Halo Therapeutics may be the most virtual biotech company on the threshold of a mid-stage study. CEO Marc Blaustein counts himself as the only full-timer, supported by a cast of about 17 people who help support the development of a single program: HT-100 for Duchenne muscular dystrophy.
So far, Halo has been financed by two DMD patient organizations: Charley's Fund and the Nash Avery Foundation. They've reviewed the animal studies, the clinical results when Israel's Collgard had it in clinical studies for bladder cancer, the scientific literature on the treatment's anti-fibrotic properties and recently gained an endorsement of the science from an influential group of independent neuromuscular experts. Halo now wants to test its treatment in a mid-stage study of about 80 DMD patients. And Blaustein is scouting for around $8 million in industry money to fund the Phase II.
"Our preference is a strategic partner who can share our vision and move at the pace we can move," says Blaustein. "As a virtual company we have a core team of outstanding, experienced developers with DMD expertise." And Halo has done it with "no physical infrastructure" in place.
Duchenne muscular dystrophy is one of those orphan diseases that fits the classic portrait of an unmet medical need. There are no treatments, aside from corticosteroids. It appears early in boys, cripples them as youths and then kills them as young men.
DMD also has a clear trigger: A mutation in the dystrophin gene that blocks production of the protein needed to support muscle tissue development. And several companies--ranging from the Dutch biotech Prosensa, to AVI in Seattle and PTC Therapeutics, have developed experimental therapies intended to at least partially mend the genetic condition to trigger a surge in dystrophin production.
"When boys miss this protein their muscles become much more susceptible to damage,' says Blaustein. Physically, they endure a vicious cycle, with an inflammatory response that damages muscles further as well as a fibrotic effect that disables the regeneration of healthy muscles. "HT-100 tips that balance back," he says, with an anti-inflammatory, anti-fibrosis approach that spurs muscle regeneration.
Importantly, this is not a genetic fix. Halo sees HT-100 as a potential monotherapy as well as a possible addition to a combination therapy with one of the other treatments now in development.
It's certainly not an easy disease to tackle, though. Last fall Genzyme returned most of its rights to PTC's ataluren. Just days ago AVI announced that its mid-stage data demonstrated an increase in the production of dystrophin, but its stock took a beating as investors reacted to the absence of efficacy seen among patients in the study.
Blaustein wants to get the mid-stage trial designed and underway in the second half of this year.