Effect of MD1003 (High Doses of Biotin) in Chronic Visual Loss Related to Optic Neuritis in Multiple Sclerosis (MS-ON): Results of a Pivotal Randomized Double Masked Placebo Controlled Study (S49.005)

Abstract

Objectives: To report the results of the MS-ON study evaluating the efficacy of MD1003 over placebo in relapsing remitting or progressive MS patients with visual loss related to optic neuropathy Background: MD1003 (High dose of Biotin) has recently demonstrated efficacy in decreasing progression and improving walking disability in patients with primary and secondary progressive multiple sclerosis (MS) Methods: This is a randomized, double-blind, multicenter, placebo-controlled (2:1) trial of oral biotin 300 mg / day in MS patients with visual loss related to optic neuropathy. All patients had at least one eye with visual acuity ETDRS score ≤ 72 for at least 6 months. We included patients either with visual loss following an optic neuritis relapse (ON relapses, n=62) or patients with chronic progressive optic neuritis (progressive ON, n=31). Treatment duration was 24 weeks. The primary endpoint was the mean change in 100[percnt] contrast visual acuity at “month 6” from baseline of the selected eye (defined as the eye with the worst visual acuity and showing worsening during the past 3 years). Other endpoints included visual evoked potentials, automated visual field perimetry, optic coherence tomography and patient reported outcome measures. Results: The last patient completed the study in August 2015. The database was locked in late September 2015. Full statistical analyses are awaited. Baseline characteristics: 93 MS patients (62 with On relapses and 31 with progressive ON) from 19 sites across France and UK were randomized. Results of primary and secondary endpoints will be presented during the meeting. Conclusions: Results will be discussed in the context of development of high doses of pharmaceutical grade biotin as a novel treatment in MS. The study was 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. Arndt has received personal compensation for activities with Bayer as a member of the scientific advisory board. Dr. Vighetto has nothing to disclose. Dr. de Burghgraeve has nothing to disclose. Dr. Pelletier has nothing to disclose. Dr. Papeix has nothing to disclose. Dr. Lebrun Frenay has nothing to disclose. Dr. Labauge has nothing to disclose. Dr. Clanet has nothing to disclose. Dr. Toosy has received personal compensation for activities with Biogen Idec. Dr. Laplaud has nothing to disclose. Dr. Vermersch has received personal compensation for activities with Biogen Idec, Sanofi, Bayer, Novartis, Merck Serono, GlaxoSmithKline, and Almirall. Dr. Vermersch has received research support from Biogen Idec, Sanofi, Bayer, and Merck Serono. 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. Debouverie has received research support from Biogen Idec, Genyzyme, Merk Serono, and Novartis. Dr. Clavelou has received personal compensation for activities with Genzyme, Teva, Novartis, Almirall, and Biogen Idec. Dr. Heinzlef has received personal compensation for activities with Genzyme as an advisory board member, from Almirail as a speaker, and Merck Serono as a consultant. Dr. De Seze has nothing to disclose. Dr. Defer has received research support from Novartis and Merck Serono Dr. Sedel holds stock and/or stock options in Medday Pharmaceuticals. Dr. Gout has nothing to disclose.