24-Month-Treatment with-MD1003 (High Doses of Biotin) in Progressive Multiple Sclerosis: Results of the MS-SPI Trial Extension Phase (S49.004)

Abstract

Objectives: To present the results at 24 months of the MS-SPI trial Background: High dose of Biotin, a co-enzyme for acetylCoA carboxylase, a potentially key-enzyme in myelin synthesis was investigated in progressive MS in a randomized placebo-controlled trial (MS-SPI). Results at 12 months demonstrated improvement of a significant proportion of patients (p=0.005), decreased mean change EDSS (p=0.014) and stabilisation of the clinical global impression of change (p<0.0001). Methods: MS-SPI is a randomized, double-blind, placebo-controlled (2:1) trial of oral biotin 300 mg / day in patients with secondary (SPMS) or primary (PPMS) progressive MS. Duration of the placebo-controlled was 48 weeks. The blinded phase was followed by a 12 months pre-planned extension phase where patients under placebo were switched to receive MD1003. Patients and physicians remained blinded to the treatment administered during the first phase of the study. Results: The last patient will complete the study in December 2015. The database will be locked in January 2016. Full statistical analyses will be available by March 2016. Results of clinical endpoints will be presented and discussed in the context of other trials in progressive MS. Conclusions: The 24-month results of the MS-SPI trial will be discussed in the context of development of high doses of pharmaceutical grade biotin as a novel treatment for progressive MS. Study supported by MedDay Pharmaceuticals

Disclosure: Dr. Tourbah has received personal compensation for activities with Biogen Idec, MedDay Pharmaceuticals, Sanofi-Genzyme, Novartis, Merck Serono, and Teva Pharma as a consultant/lecturer. Dr. Lebrun Frenay has nothing to disclose. Dr. Edan has nothing to disclose. Dr. Clanet has nothing to disclose. Dr. Papeix has nothing to disclose. Dr. Vukusic has received personal compensation for activities with Bayer-Schering, Biogen Idec, Genzyme, Novartis, Merck Serono, Roche, Sanofi Aventis, and Teva Neuroscience. Dr. De Seze has nothing to disclose. Dr. Debouverie has received research support from Biogen Idec, Genyzyme, Merk Serono, and Novartis. Dr. Gout has nothing to disclose. Dr. Clavelou has received personal compensation for activities with Genzyme, Teva, Novartis, Almirall, and Biogen Idec. Dr. Defer has received research support from Novartis and Merck Serono Dr. Laplaud has nothing to disclose. Dr. Moreau has received personal compensation for activities with Biogen Idec, Sanofi-Aventis Pharmaceuticals, Genzyme corporation, Teva Neuroscience, Bayer Schering, Merck Serono, Novartis, and Almirall as a consultant. Dr. Labauge has nothing to disclose. Dr. Brochet has received personal compensation for activities with Biogen, Novartis, Teva, Genzyme, Merck Serono Roche, Bayer as a consultant and speaker. Dr. Sedel holds stock and/or stock options in Medday Pharmaceuticals. Dr. Pelletier has nothing to disclose.