of operating cash ($13 million), boosted the firm’s share price through a reverse split in which shareholders traded in eight of their shares for one each, and brought in a couple new senior managers on the development side.
The big thing Aastrom is looking forward to are results from a mid-stage trial among patients with critical limb ischemia. This condition, in which people get clogged blood vessels in the legs that choke off circulation, causes an estimated 160,000 amputations a year. Doctors sometimes use tiny stents to prop open blocked arteries in the legs, but they don’t always work, and about three-fourths of patients with the disease die within four or five years, Mayleben says.
Based on animal studies, Aastrom hopes to show that its treatment offers a new approach, by helping foster growth of new blood vessels to improve circulation. The company has been running the most rigorous clinical trial yet to suggest its treatment has promise. This study, called Restore-CLI, enrolled a total of 90 patients who were randomly assigned to the cell therapy or a placebo treatment.
Back in February, Aastrom said that its treatment reached the primary goal of the study in an interim analysis of the first 46 patients who went through six months of follow up. Researchers found that those on the Aastrom cell therapy were able to keep some of the most severe complications of their disease (amputations, large wounds, and gangrene) at bay for a longer period of time. Details of those results are expected to be presented at the Society of Vascular Surgery in Boston on June 11, Mayleben says. Before the end of this month, Aastrom hopes to meet with the FDA and get some feedback on what the next step of development—a Phase III clinical trial program—ought to look like to confirm this finding. By the end of 2010, Aastrom hopes to have the full set of data on all 90 patients, he says.
A lot of key questions will have to be hashed out with the FDA before people get too excited about this being a stem-cell breakthrough. What the study goals ought to be, how many studies are required, how many patients need to be enrolled are some of the big questions that the company will have to work out with the FDA. Aastrom knows that where there’s hype, skepticism will surely follow, so it will have some high hurdles to clear with the scientific community.
All that means Aastrom will have to work hard in the coming months and years to gather proof if it’s ever going to blaze a trail with a stem-cell therapy for severe vascular disease. The company currently has about 18 to 20 months of operating cash in the bank, by which time it should have more positive data rolling in to support a higher stock valuation, Mayleben says.
“We’ve been trying to distinguish ourselves with the science and rigor of Phase II studies. It’s been lacking a bit in the industry, because companies are often under-capitalized,” Mayleben says. “But it’s an exciting time for our company. We have a great opportunity.”
