Positive Results from Phase 3 CONFIRM Clinical Trial Show Efficacy and Safety of Oral BG-12 in Multiple Sclerosis

Today Biogen Idec BIIB +2.26% announced that detailed positive data from CONFIRM, the second Phase 3 clinical trial of oral BG-12 (dimethyl fumarate) in people with relapsing-remitting multiple sclerosis (RRMS), will be presented in three platform presentations at the 64th Annual Meeting of the American Academy of Neurology (AAN) in New Orleans. In CONFIRM, BG-12 demonstrated efficacy across a variety of clinical and radiological outcome measures, as well as favorable safety and tolerability profiles. These data, along with results from BG-12’s first Phase 3 study, DEFINE, were included in regulatory applications that were submitted to U.S. and EU regulatory agencies early this year.

“Results from CONFIRM complement the profile we have seen for BG-12 throughout its clinical development program, which now includes robust data sets from two global, placebo-controlled Phase 3 pivotal studies with more than 2,600 multiple sclerosis (MS) patients,” said Douglas E. Williams, Ph.D., Biogen Idec’s executive vice president of Research and Development. “If approved by regulators, we believe BG-12 could be an important new oral therapeutic option for MS patients.”

CONFIRM Efficacy Results

CONFIRM was a global, placebo-controlled clinical trial to determine the efficacy and safety of 240 mg of BG-12, administered either twice a day (BID) or three times a day (TID), in people with RRMS. The study included glatiramer acetate (GA; 20 mg subcutaneous daily injection) as a reference comparator. Both active treatments were compared to placebo.

BG-12 met the CONFIRM study’s primary endpoint by significantly reducing annualized relapse rate (ARR) by 44 percent for BID and by 51 percent for TID (p<0.0001 for both) compared to placebo over two years. GA reduced ARR by 29 percent (p=0.0128) compared to placebo over two years.

BG-12 met the study’s secondary relapse endpoint by significantly reducing the proportion of patients who relapsed at two years by 34 percent for BID (p=0.0020) and by 45 percent for TID (p<0.0001) compared to placebo. GA provided a 29 percent reduction (p=0.0097) in the proportion of relapsing patients compared to placebo over the same time period.

BG-12 also met magnetic resonance imaging (MRI) endpoints in a cohort of patients, demonstrating a significant effect on MS brain lesions. Reductions in new brain lesion counts were evident within the first year of treatment and were sustained throughout the study. At two years compared to placebo:

— BG-12 reduced the number of new or newly enlarging T2-hyperintense lesions (secondary endpoint) by 71 percent for BID (p<0.0001) and by 73 percent for TID (p<0.0001), while GA provided a 54 percent reduction (p<0.0001).

— BG-12 reduced the number of new non-enhancing T1-hypointense lesions (secondary endpoint) by 57 percent for BID (p<0.0001) and by 65 percent for TID (p<0.0001), while GA provided a 41 percent reduction (p=0.0021).

— BG-12 reduced the odds of having more gadolinium-enhancing (Gd+) lesions (tertiary endpoint) by 74 percent for BID (p<0.0001) and by 65 percent for TID (p=0.0001), while GA provided a 61 percent reduction (p=0.0003).

“Until we find a cure for MS, there is a need for new treatments that address this debilitating disease,” said Robert J. Fox, M.D., medical director of the Mellen Center for Multiple Sclerosis at Cleveland Clinic and principal investigator of the CONFIRM clinical trial*. “The strong efficacy and safety results we have observed in the CONFIRM study suggest BG-12 may be a positive addition to the current MS treatment paradigm.”

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