The drug: BG-12 (dimethyl fumarate)
The disease: Multiple sclerosis
The developer: Biogen Idec
Peak projections: $2 billion-$3 billion (RBC Capital Markets' best-case estimate)

A major goal of biopharma R&D in multiple sclerosis has been to develop oral drugs that can combat the relapsing and remitting form of the nerve-damaging disease. BG-12--Biogen Idec's ($BIIB) contender for an oral alternative to replace the injectable meds that now dominate the market--has captivated the industry watchers with upbeat data this year from the first of two major Phase III trials.

Last week, Biogen revealed full data from the pivotal, 1,237-patient DEFINE trial. There was a 53% reduction in annualized relapse rate in patients who took the MS pill twice a day, and risk of disability progression among those patients fell 38%. No new safety-related signals appeared to jeopardize the prospects of the drug. The MS pill might also come with lower safety risks than Novartis' Gilenya, the first FDA-approved MS pill, which is associated with increased risk of infection.

Later this year, Biogen is expected to reveal data from the second pivotal trial of BG-12, called CONFIRM, and that study will provide further evidence of the drug's safety and efficacy in MS patients. If the data look good, Biogen could be on its way to its first FDA approval of a new drug since 2004, when the MS treatment Tysabri got its initial market green light from the agency. Biogen CEO George Scangos (photo), whose company has been on a roll for most of his some 15 months as chief executive, will have a potential blockbuster on his hands to grow the company's world-leading position in the MS drug market.

A bunch of biopharma groups have been seeking to break into the oral MS drug business, but so far Merck KGaA and Teva Pharmaceutical have stumbled with their experimental pills, and Sanofi left analysts unimpressed with its latest batch of data. So it appears to be Biogen's race to lose right now.

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