Biogen Idec bets it can succeed on a risky Phase III ALS gamble

Bloomberg biotech scribe Meg Tirrell has been plumbing Biogen Idec's ($BIIB) late-stage drug for ALS and finds some good reasons to believe that they are on to something big--perhaps even blockbuster-sized.

First, dexpramipexole had to stand up to George Scangos' review process when he took the helm a couple of years ago. Anxious to trim away any low percentage shots at an approval, Scangos was won over by the data and market profile. The drug--in-licensed from Knopp Biosciences--did well in Phase II, demonstrating a significant slowing in disease progression after a brief 12 weeks of treatment at the highest dose of 300 mg daily. A later stage of the trial tracked a significant mortality benefit comparing the high and the low dose. And if the data hold up in the late-stage trial, that could translate into a sizable market of a billion dollars-plus, considering the woeful absence of any effective therapy for patients.

But Scangos had been skeptical for a good reason. A whole slate of prospective treatments has been shot down in the clinical trial process, underscoring how little is known about Lou Gehrig's disease and what needs to be done to slow or stop it. But it's late-stage study--testing 150 mg twice daily--enrolled patients in just a few months. Now late-stage data is due out later this year. A win here would help further burnish Scangos' reputation for turning around the one-time biotech laggard. 

"This is far from a long shot," CEO Scangos told investors at the annual J.P. Morgan confab in San Francisco in January. "We are genuinely hopeful that we will be able to provide meaningful therapy for the thousands, tens of thousands, of ALS patients who right now have very little to help them."

- here's the story from Bloomberg