A group of researchers presented two papers reporting results of clinical trials with biotin (MD1003) that show promising results.
Presented at the annual AAN conference, April 21, 2016 (Vancouver, BC):
S49.004 - 24-Month-Treatment with-MD1003 (High Doses of Biotin) in Progressive Multiple Sclerosis: Results of the MS-SPI Trial Extension Phase
Ayman Tourbah,1Christine Lebrun-Frenay,2Gilles Edan,3Michel Clanet,4Caroline Papeix,5Sandra Vukusic,6Jerome De Seze,7Marc Debouverie,8Olivier Gout,5Pierre Clavelou,9Gilles Defer,10David Laplaud,11Thibault Moreau,12Pierre Labauge,13Bruno Brochet,14Frederic Sedel,5Jean Pelletier15
1Reims, France, 2Nice, France, 3France, 4Toulouse, France, 5Paris, France, 6Lyon, France, 7Strasbourg, France, 8Nancy, France, 9Clermont Ferrand, France, 10Caen, France, 11Nantes, France, 12Dijon, France, 13Nimes, Cedex 4, France, 14Bordeaux, France, 15Marseille, Cedex 5, France
Disclosures
A. Tourbah: I have received consulting and lecturing fees, travel grants from Biogen Idec, MedDay Pharmaceuticals, Sanofi-Genzyme, Novartis, Merck Serono, and Teva Pharma.. C. Lebrun-Frenay: None. M. Clanet: ; Consulting fees and scientific advisory boards from Teva, Merck Serono, Sanofi Genzyme, Biogenidec, none, none, none, none, none, Biogenidec grant to my department (salary resarch technician). C. Papeix: teva, Novartis, servier, roche. S. Vukusic: Bayer-Schering, Biogen Idec, Genzyme, Novartis, Merck Serono, Roche, Sanofi Aventis and Teva Pharma, Bayer-Schering, Biogen Idec, Genzyme, Novartis, Merck Serono, Roche, Sanofi Aventis and Teva Pharma. J. De Seze: None. M. Debouverie: Biogen-Idec, Genzyme, Merck-Serono, Novartis The organizations mentioned in this statement did not participate in any aspects of the design, execution, analysis, or write-up of this study. O. Gout: O.Gout received consulting and lecture fees from Allergan, Almirall, Bayer-Schering, Biogen Idec, Genzyme, Novartis, Merck Serono, Sanofi, and Teva Pharma. P. Clavelou: Travel : Genzyme, Teva, Novartis Scientific Advisory Board : Almirall, Genzyme, Novartis Speaking : Novartis, Teva, Biogen. G. Defer: Dr Defer received personal compensation for scientific advisory board from Sanofi-Genzyme and Teva pharmaceutical Industries Ltd and has received funding for travel and/or speaker honoraria from Merck, My institution received grant from Novartis and Merck Serono for helping epidemiological and clinical research in MS. D. Laplaud: Yes, honoraria from Biogen, Novartis, Roche, Genzyme and Merck-Serono, grants from Novartis and Genzyme for financial support in basic science. T. Moreau: I report receiving consulting fees and speaking fees from Biogen Idec, Sanofi Aventis, Genzyme, Teva Pharma, Bayer Schering, Merck Serono, Novartis and Almirall., I received personal compensation as Editor in Chief for the journal entitled La Lettre du Neurologue. P. Labauge: None. B. Brochet: consulting, serving on a scientific advisory board, speaking: Biogen, Novartis, Teva, Genzyme, Merck Serono Roche, Bayer, Unconditional grants from Biogen, MedDay, Novartis, Teva, Genzyme, Merck Serono Roche, Bayer. F. Sedel: CEO of MedDay Pharùaceuticals, CEO and chairman of the board of MedDay Pharmaceuticals, shareholder, co-founbder and CEO of Medday pharmaceuticals, Yes, shareholder, co-founder and CEO of Medday pharmaceuticals. J. Pelletier: consulting, serving on a scientific advisory board, speaking: Biogen, Novartis, Teva, Genzyme, Merck Serono Roche, Bayer, Unconditional grants from Biogen, Novartis, Merck Serono.
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
__________________________________
S49.005 - 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
Ayman Tourbah,1Carl Arndt,1Alain Vighetto,2Véronique Deburghgraeve,3Jean Pelletier,4Caroline Papeix,5Christine Lebrun-Frenay,6Pierre Labauge,7Michel Clanet,8Ahmed Toosy,9David Laplaud,10Patrick Vermersch,11Thibault Moreau,12Marc Debouverie,13Pierre Clavelou,14Olivier Heinzlef,15Jerome De Seze,16Gilles Defer,17Frederic Sedel,5Olivier Gout5
1Reims, France, 2Lyon, France, 3Rennes, France, 4Marseille, Cedex 5, France, 5Paris, France, 6Nice, France, 7Montpellier, Cedex 4, France, 8Toulouse, France, 9London, United Kingdom, 10Nantes, France, 11Lille, France, 12Dijon, France, 13Nancy, France, 14Clermont Ferrand, France, 15Poissy, France, 16Strasbourg, France, 17Caen, France
Disclosures
[see Disclosures for the abstract above]
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% 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 [optic neuritis or 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