Friday, September 02, 2016MedDay Announces Publication of MD1003 Phase IIb/III Study in the Multiple Sclerosis Journal
Paris, France, 2 September 2016
- MedDay, a biotechnology company focused on the treatment of nervous system disorders, announces the online publication of results from the previously reported Phase IIb/III, MS-SPI study of MD1003 in patients with progressive multiple sclerosis (MS) in the Multiple Sclerosis Journal. The objective of the study was to confirm the efficacy and safety of MD1003 in progressive MS in a double-blind, placebo controlled study.
According to the key findings published in the journal,a total of 13 (12.6%) MD1003-treated patients achieved the primary endpoint versus none of the placebo-treated patients (p = 0.005). MD1003 treatment also reduced Expanded Disability Status Scale (EDSS) progression and improved clinical impression of change compared with placebo. Efficacy was maintained over follow-up, and the safety profile of MD1003 was similar to that of placebo. In conclusion, MD1003 achieved sustained reversal of MS-related disability in a subset of patients with progressive MS and was well tolerated.
The publication titled “MD1003 (high-dose biotin) for the treatment of progressive multiple sclerosis: A randomised, double-blind, placebo-controlled study” can be accessed online via: http://msj.sagepub.com/content/early/20 ... 8.abstract
.MS-SPI study results
The MS-SPI study was designed to assess the potential of MD1003 to reverse disease progression in patients with not-active progressive MS. Patients included in the study had to demonstrate disease progression of disability in the previous two years with no evidence of inflammatory activity. Patients were randomized to receive either MD1003 (n=103) or placebo (n=51) for 12 months, followed by a 12-month extension phase during which all patients received MD1003 but remained blinded as to whether they had received the active drug during the first phase. The primary endpoint was particularly challenging, and defined as the proportion of patients with either improvement of EDSS or TW25 (timed 25-foot walk) after nine months (M9) confirmed at 12 months (M12), which equates to a confirmed reversion of progression.
The primary endpoint was met (p=0.0051) with 12.6% of patients in the MD1003 arm showing a confirmed reversion of progression at M9 (confirmed at M12), compared to none (0%) in the placebo arm. During the 12-month extension phase, patients initially on MD1003 exhibited sustained improvement compared to baseline, with 13.2% of patients showing improvement at M18 (confirmed at M24) and 15.4% of patients showing improvement at M24. When patients in the placebo group were switched to MD1003 for the extension phase, the proportion of responders reached 7.1% at M18 (confirmed at M24) and 11.9% at M24, demonstrating that treatment with MD1003 reversed progression in some patients switched to MD1003.
The primary judgement criterion was supported by the mean change of EDSS from baseline in the overall population. The mean EDSS decreased between M0 and M12 in the MD1003 group (-0.03) compared to progression in the placebo group (+0.13, p=0.014). During the extension phase, the values remained relatively constant compared to baseline in patients maintained on MD1003 (from -0.03 to 0.04 at M24), indicating sustained efficacy, whereas patients initially on placebo stopped progressing on EDSS once switched to MD1003 (from +0.13 at M12 to +0.15 at M24, compared to baseline).
The efficacy demonstrated on EDSS was also supported by the mean clinical global impression of change scale (CGI) which was worse at M12 in the placebo group compared to the MD1003 group (p<0.0001). It remained constant in the extension phase for patients initially on MD1003 and improved after patients were switched from placebo to MD1003. As a result no difference was observed between the two groups at the end of the extension phase (p=0.92).MD1003
is a patented, highly dosed pharmaceutical grade biotin that has already shown efficacy in patients with progressive multiple sclerosis. MD1003 has a unique mode of action which potentially acts on two targets related to progressive MS: (1) it activates the Krebs cycle, the main route for energy production that protects against axonal degeneration and (2) it potentially activates acetyl-CoA carboxylases (ACC1 and ACC2), the rate-limiting enzymes in the synthesis of fatty acids required for myelin repair. The drug is already commercialized in some European countries under early-access programs.