Biogen Idec (NASDAQ: BIIB) announced today positive top-line results from DEFINE, the first of two pivotal Phase 3 clinical trials designed to evaluate the investigational oral compound BG-12 (dimethyl fumarate) as a monotherapy in people with relapsing-remitting multiple sclerosis (RRMS). Results showed that 240 mg of BG-12, administered either twice or three times a day, met the primary study endpoint, demonstrating a highly statistically significant reduction (p<0.0001) in the proportion of patients with RRMS who relapsed at two years compared with placebo. Both doses of BG-12 also met all of the secondary study endpoints, providing a statistically significant reduction in annualized relapse rate, in the number of new or newly enlarging T2 hyperintense lesions, in new gadolinium-enhancing (Gd+) lesions, and in the rate of disability progression as measured by the Expanded Disability Severity Scale (EDSS) at two years.
DEFINE was a global, randomized, double-blind, placebo-controlled, dose-comparison study to determine the efficacy and safety of BG-12 in people with RRMS. In addition to meeting the primary and all secondary endpoints, initial data from the trial showed that BG-12 demonstrated a favorable safety and tolerability profile. The overall incidence of adverse events and serious adverse events was similar among the placebo group and both BG-12 treatment groups. The safety profile was consistent with what was seen in the published Phase 2 study of BG-12. Further analyses of the DEFINE study are ongoing, and the company anticipates presenting detailed data at a future medical meeting.
"The significant clinical responses seen in the DEFINE study represent an important step forward in the development of BG-12 for multiple sclerosis (MS)," said Douglas Williams, Ph.D., Biogen Idec's Executive Vice President of Research and Development. "We are very pleased with these data and believe that BG-12 has the potential to offer MS patients a highly effective oral treatment option with a strong safety profile."
Data from scientific studies indicate that BG-12 has the potential to be distinctive by reducing the entry into and the action of inflammatory cells on the Central Nervous System (CNS), as well as potentially protecting CNS cells from oxidative stress and death by activation of the Nrf-2 pathway.
BG-12 received Fast Track designation from the U.S. Food and Drug Administration (FDA) in 2008. In addition to DEFINE, another Phase 3 RRMS clinical trial, CONFIRM, is currently underway. This study is evaluating BG-12 and an active reference comparator, glatiramer acetate, against placebo on clinical relapse, magnetic resonance imaging (MRI) measures of MS, progression of disability, and safety. Results from CONFIRM are expected in the second half of 2011.