Table 1.

Efficacy data from pivotal Phase III clinical trials of dimethyl fumarate

Trial	Subjects	Design	Key clinical outcomes	Key MRI outcomes
DEFINE	RRMS1 (n=1,234)	Placebo-controlled Double-blind Randomized 1:1:1 (240 mg bid, 240 mg tid, placebo) Multicenter 2 years	*Proportion of patients relapsing over 2 years (27% bid, 26% tid, 46% placebo; P<0.001 for both comparisons) *ARR (0.17 bid, 0.19 tid, placebo 0.36; P<0.001 for both comparisons) 2-year confirmed disability progression (16% bid, 18% tid, 27% placebo; P=0.005 bid versus placebo; P=0.01 for tid versus placebo)	Reduction in new or enlarging T2 lesions (85% bid, 74% tid; P<0.001 for both comparisons) Reduction in odds of an increased number of Gd+ lesions at 2 years (90% bid, 73% tid; P<0.001 for both comparisons)
CONFIRM	RRMS1 (n=1,417)	Placebo-controlled Double-blind Open-label for those randomized to GA (reference comparator) Randomized 1:1:1:1 (240 mg bid, 240 mg tid, placebo, GA) Multicenter 2 year	*ARR at 2 years (0.22 bid, 0.20 tid, 0.29 GA, 0.40 placebo; relative reductions 44% bid and 51% tid, P<0.001; GA: 29% P=0.01) Reductions in disability progression (relative reduction: 21% bid P=0.25; 24% tid P=0.20; 7% P=0.7 GA)	Reduction in number of new or enlarging T2 lesions (71% bid, 72% tid, 54% GA; P<0.001 for all comparisons) Reduction in odds of new Gd+ lesions at 2 years (74% bid, 65% tid, 61% GA; P<0.001 for all comparisons)

Note:

^*Primary outcome.

Abbreviations: ARR, annualized relapse rate; bid, twice daily; CONFIRM, Comparator and an Oral Fumarate in RRMS; DEFINE, Determination of the Efficacy and Safety of Oral Fumarate in RRMS; GA, glatiramer acetate 20 mg subcutaneous once daily; Gd+, gadolinium-enhancing; MRI, magnetic resonance imaging; RRMS, relapsing-remitting multiple sclerosis;102 tid, three times daily.