than just the data from Defend-1. The company is planning a second study, called Defend-2, to confirm the result. It will have a similar design, and the company hopes to begin enrolling patients this year. If the first trial produces positive results, it’s fair to assume that will speed up patient recruitment for the second study, Ringler says.
Previous trials have given Tolerx reason to be confident in the current study, Ringler says. Those trials have shown that patients on the drug produce more insulin than those in a control group for at least 18 months. Ringler also says that without breaking the blind that’s supposed to protect the Defend-1 study from biases, it can be inferred from analysis of blood samples that patients receiving the Tolerx drug are producing 100 percent to 200 percent more T regulatory cells than those on the placebo. As with any drug that suppresses a component of the immune system, I had to ask whether Tolerx’s drug appears to make patients more vulnerable to infections. “We have not seen a single case of an opportunistic infection,” Ringler says. One reason might be that the Tolerx drug leaves other major immune system components intact, he says.
It will be interesting to see how the data from Defend-1 affects Tolerx as a business. The company has considered an IPO at least twice in the past, and it may again. “I’m an opportunist,” Ringler says, when asked if he’d try again if the market turns favorable. But there’s no rush to raise capital. Tolerx has about 70 employees, but the Glaxo deal brought its cash burn rate down to the point where it now has “many years” of capital to keep the business operating, Ringler says. He’s not giving a timetable for when Defend-2 results might be available, and when Tolerx might be ready to submit an application for FDA approval of its first drug, but when I speculated it might be around 2013, he said that would be “close.”
All of those milestones are important, but none of it will matter much if the Defend-1 results turn out poorly by year end. Ringler sounds awfully confident the results will be in his favor.
“This is the most important year in the company’s history,” Ringler says. “It’s the one I’m most excited about. We have so many value-driving events to look forward to.”
