Biogen Idec ($BIIB) kept excitement alive for its blockbuster hopeful BG-12 this morning with a full set of data from its first pivotal trial on the oral multiple sclerosis drug. The Weston, MA-based biotech powerhouse released the full data on the DEFINE trial this morning at a meeting in Europe after generating positive buzz on the program with initial data revealed in April.
While Biogen watchers await data expected later this year from the second of the two pivotal trials, dubbed CONFIRM, the company gave no reason to think that those results won't continue to make the program shine. The 1,237-patient DEFINE trial showed that patients who took BG-12 twice a day had a reduced annualized relapse rate of 53% and a reduced risk of disability progression of 38%. And there appeared to be no new safety-related red flags to threaten the success of the drug.
BG-12 is Biogen's best chance at launching a new blockbuster and competing in the market for oral therapies for multiple sclerosis, which is now most often treated with injectable medications. For Biogen, the world's leading provider of MS drugs, the MS pill could also become the firm's first new drug to garner FDA approval since the injectable drug for MS called Tysabri, which initially got the green light for U.S. sales in 2004. The MS pill would follow Novartis's Gilenya, the first FDA-approved oral med for the nerve-damaging disease. And other firms such as Teva Pharmaceutical ($TEVA) and Sanofi ($SNY) also have horses in this race.
"Overall the [BG-12] dataset lived up to investors' high expectations, showing considerable activity and a clean safety profile," Eric Schmidt, an analysis at Cowen, wrote in a note to investors this morning. "Moreover, reaction from physicians to the dataset appears almost uniformly positive."
The latest data from DEFINE also show that MRI scans of patients with relapsing remitting forms of MS who took the drug had significant reductions in brain lesions compared with those in the placebo group.
"The significant clinical and radiological responses in DEFINE are further evidence that BG-12 may become an oral therapy of choice for MS patients," Doug Williams, Biogen's R&D head, said in a statement. "Results from our second Phase 3 trial, CONFIRM, will provide additional insight into BG-12's profile, as well as a comprehensive data set to further discussions with regulatory authorities. We anticipate releasing top-line data from CONFIRM later this year."
- here's the release
- see the Reuters report