Though detailed data has not yet been released, Biogen Idec has reported “top-line” results from the first phase three trial to evaluate the investigational oral compound BG-12 (dimethyl fumarate) as a monotherapy in people with relapsing-remitting multiple sclerosis (RRMS).
Results of the DEFINE 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” in the proportion of people with RRMS who relapsed at two years compared with placebo, Biogen said. The company noted that both doses of BG-12 also provided a “statistically significant” reduction in annualized relapse rate, in the number of new or newly enlarging T2 hyperintense lesions, in new gadolinium-enhancing lesions, and in the rate of disability progression as measured by the Expanded Disability Severity Scale (EDSS) at two years.
Initial data from the trial showed that BG-12 demonstrated a favorable safety and tolerability profile, according to Biogen. 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 two study of BG-12. Further analyses of the DEFINE study are ongoing, and the company anticipates presenting detailed data at a future medical meeting.
Data from scientific studies suggest 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 three 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.