BiotechTracker - BioMS’s Multiple Sclerosis Drug Moves Forward
May 23, 2003 BioMS’s (Toronto: MS) MBP8298 (synthetic myelin basic protein) has generated compelling phase II data that set the stage for more comprehensive investigation of its effects in the management of chronic, progressive multiple sclerosis (CPMS). BioMS is in the process of preparing regulatory submissions for “a pivotal confirmatory clinical trial” slated for initiation this year.  Although clinically and statistically significant therapeutic effects were observed for patients with specific MS susceptibility genes, only clinically significant effects were observed in the overall population. The study design of future trials will need to be one that both sustains the effects observed in susceptibility gene carriers, and makes it possible to observe statistically significant effects at the level of the entire sample such that on-label usage for the general CPMS population becomes possible.  An underpowered, 32-patient, 4-year, two-component, phase II trial in patients with primary or secondary progressive MS was performed. A two-year, randomized, double-blind, placebo-controlled component preceded a two-year, open label component. During the double-blind component, patients received intravenous MBP8298 500 mg every 6 months. A 20-patient sample of DR2 or DR4 MS susceptibility gene carriers were evenly distributed between the MBP8298 and placebo arms. Clinical progression was measured, in standard fashion, by changes in score on the Expanded Disability Status Scale (EDSS).  MBP8298’s two-year comparative therapeutic effects were greatest for susceptibility gene carriers, as disease progression was observed for 0/10 (0%) MBP8298 recipients and 6/10 (60%) placebo recipients (p=0.0108). Even after the open-label component (results at 42 months), only 3 (30%) susceptibility gene carriers had progressed. Thus, the median time to disease progression for the MBP8298 recipients translated to at least four years, compared to two years for placebo recipients (p=0.004). MBP8298 recipients with susceptibility genes were found to have “significant and sustained” reductions in anti-MBP antibodies that both correlated with absence of clinical progression and partly confirmed the drug’s mechanism of action.  In the total study population, 5/16 MBP8298 recipients progressed, versus 9/16 placebo recipients (p=0.29, an insignificant difference). MBP8298’s overall safety profile was reassuring. Given the observation that no statistically significant differences were observed versus the placebo group in terms of results from eight different MRI tests, it becomes imperative that the drug show unequivocal clinical benefit in future study. Clinical benefit trumps imaging benefit, but observations of both would more readily qualify the drug as potentially superior to contemporary agents.  MBP8298 is a synthetic myelin basic protein (MBP) fragment that mimics the portion of the native protein under immunological attack in MS. Its mechanism of action includes provocation of a favorable immune response.
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