Phase II/III study of MD 1003 (biotin) shows benefits and safety in multiple sclerosis- MedDay

MedDay has announced full study results from the MS-SPI and MS-ON trials, including the MS-SPI extension data on MD 1003 (biotin) in multiple sclerosis (MS), which will be presented at the 2016 American Academy of Neurology Annual Meeting. Phase IIb/III MS-SPI and MS-ON studies tested the efficacy of MD 1003, a pharmaceutical-grade biotin administered at a dose of 300 mg per day in the treatment of MS and particularly of the most difficult to treat “not-active progressive MS,” for which there is no approved drug. Overall these data show the best effect size ever observed to date and confirm the good safety profile of MD 1003.

The MS-SPI study was designed to assess the potential of MD1003 to reverse disease progression in patients with not-active progressive MS. The primary endpoint was met with 12.6% of patients in the MD 1003 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 MD 1003 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 MD 1003 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 MD 1003 reversed progression in some patients switched to MD 1003.

The MS-ON study was designed to investigate whether MD1003 could accelerate recovery following incomplete remission of an acute relapse and/or specifically improve patients with progressive disability. Final results showed that, overall, patients who received MD 1003 improved slightly more than patients who received the placebo (average of 3.1 letters in the MD 1003 arm versus 1.8 letters in the placebo arm). However the difference did not reach statistical significance. Patients in both the MD 1003 and placebo groups continued to improve during the extension phase (mean of 4.25 letters in the initial MD 1003 arm versus 4.0 letters in the placebo arm switched to MD1003). Findings from both MS-SPI and MS-ON trials show that, overall, MD 1003 is well tolerated. The incidence of adverse events was similar across the two groups in both studies.