Following fast on the heels of Sanofi's ($SNY) approval of the oral multiple sclerosis (MS) drug Aubagio, Biogen Idec ($BIIB) has outlined its full pivotal case for BG-12, with an impressive set of efficacy and safety data that is likely to pave the way to a blockbuster approval before year's end.
Scientists published the results from the two big Phase III studies for BG-12 (dimethyl fumarate), demonstrating that both of the treatment regimens studied in the pivotal program cut the annual MS relapse rate by about half. The twice-daily dose was associated with a drop in new or expanded lesions by 71% to 99%. And there was a 38% reduction in progression to disability.
Just as important, BG-12 delivered fresh evidence of its relatively clean safety profile, a big concern in a field now dominated by treatments associated with some severe, and occasionally fatal, side effects. Flushing and gastrointestinal symptoms, like vomiting, were the most common side effects, but tended to taper off after the first two weeks of therapy. The results confirmed the general safety profile of a comparable fumarate already in use, which is likely going to add to its appeal to regulators.
"The safety track record is well known and appears to be very strong," study leader Robert Fox told The New York Times.
"Because MS is a chronic disease, we look for treatment options that not only control relapses but also slow patients' disease progression for as long as possible," said Ralf Gold, the lead author of the DEFINE study published in The New England Journal of Medicine. "In DEFINE, dimethyl fumarate demonstrated efficacy, as well as positive safety and tolerability profiles, which is a very attractive combination for an MS treatment."
While Biogen Idec CEO George Scangos inherited the late-stage BG-12 program from his predecessor at the company, it's been Exhibit A for investors making the case for the big biotech's turnaround. Biogen is a longtime leader in the MS field, a fact that has not been lost on analysts like Cowen's Eric Schmidt, who has bullishly forecast peak sales at $3 billion. Others believe that an overly rosy projection on revenue is likely to leave some backers unhappy if the drug falls short of that high mark. The market for MS has grown to $12 billion.
Biogen Idec's share price has almost tripled over the past two years.
- here's the press release
- get the story from The New York Times