Reversal of disability MD1003 in not-active progressive MS real world experience: the French Grand-Est cohort
ECTRIMS Online Library. Tourbah A. 09/13/19; 278576; P1375
Ayman Tourbah
Ayman Tourbah
Contributions
Abstract

Abstract: P1375

Type: Poster Sessions

Abstract Category: Therapy - Neuroprotection and Repair

A. Tourbah1, J. De Sèze2, M. Debouverie3, E. Berger4, G. Matthey3, N. Collongues5, G. Romain6, C. Castex7, T. Moreau8

1Faculté de Médecine de Reims URCA, Reims, 2Neurology CHU Strasbourg, Strasbourg, 3Neurology CHU Nancy, Nancy, 4Neurology, CHU Besançon, Besançon, 5Hopital Civil Strasbourg, Strasbourg, 6Biostatistic, CHU Dijon, Dijon, 7Santé Publique et Innovation CHU Reims, Reims, 8Neurology CHU Dijon, Dijon, France

Objectives: This study describes improvement in EDSS with MD1003 (high dose pharmaceutical-grade biotin) in PPMS and SPMS from five centers in the French early access program.
Background: Phase IIb trials of MD1003 demonstrated reversal of disability in patients potentially by neurorpair or reversing energy failure.
Methods: Clinical measures of 579 MD1003 patients including EDSS change (1 point if EDSS< 6, 0.5 points EDSS>5.5)
Results: 579 Patients mean age of 57.1±10.1; sex ratio F:M 1.6:1;; 376 SPMS patients and 203 PPMS patients. 21% treated with a concomitant immunotherapy. Patients were followed for a mean of 12.7 ± 6.7 years (3-33 months )
Efficacy (non-confirmed EDSS) as of 4/2019 (to be updated at presentation): 13% of patients improved, 79% were improving or stable and 21% worsening from their last measure to M0. Improvement was similar between PPMS and SPMS (11% vs 15%). 7% improved at M3 (81% stable or improving) with 9% worsening. 81% improving or stable at M12 (14% improving, 9% worsening). . 21% improved (82% stable or improved, and 1% worsening) in Year 2. PPMS trended to greater improvement in year 2 than SPMS (30% vs 17%).
Conclusions: This is the largest real life MS cohort of an early access program. A majority of patients improved or were stable on treatment. . A DMT effect is possible as improvement is seen after 1 year of treatment particularly evident in year 2 for PPMS patients. MD1003 appears to be a well-tolerated efficacious treatment for reversing disease progression in a population with few therapeutic options.
Disclosure:
Ayman Tourbah has received consulting and lecturing fees, travel grants and research support from Medday Pharmaceuticals, Biogen, Sanofi-Genzyme, Novartis, Merck, Teva Pharma, and Roche
Jérôme De Sèze, Marc Debouverie, Eric Berger served as consultant, board and therapeutical trials for Novartis
Thibault Moreau reports receiving consulting fees and speaking fees from Biogen Idec, Sanofi Aventis, Genzyme, Teva Pharma, Bayer Schering, Merck Serono, Roche, Almirall and Novartis, Roche.
Nicolas Collonques, Guillaume Mattey, Gaëlle Romain and Cédric Castex have nothing to disclose
Investigator sponsored study supported by MedDay Pharmaceuticals

Abstract: P1375

Type: Poster Sessions

Abstract Category: Therapy - Neuroprotection and Repair

A. Tourbah1, J. De Sèze2, M. Debouverie3, E. Berger4, G. Matthey3, N. Collongues5, G. Romain6, C. Castex7, T. Moreau8

1Faculté de Médecine de Reims URCA, Reims, 2Neurology CHU Strasbourg, Strasbourg, 3Neurology CHU Nancy, Nancy, 4Neurology, CHU Besançon, Besançon, 5Hopital Civil Strasbourg, Strasbourg, 6Biostatistic, CHU Dijon, Dijon, 7Santé Publique et Innovation CHU Reims, Reims, 8Neurology CHU Dijon, Dijon, France

Objectives: This study describes improvement in EDSS with MD1003 (high dose pharmaceutical-grade biotin) in PPMS and SPMS from five centers in the French early access program.
Background: Phase IIb trials of MD1003 demonstrated reversal of disability in patients potentially by neurorpair or reversing energy failure.
Methods: Clinical measures of 579 MD1003 patients including EDSS change (1 point if EDSS< 6, 0.5 points EDSS>5.5)
Results: 579 Patients mean age of 57.1±10.1; sex ratio F:M 1.6:1;; 376 SPMS patients and 203 PPMS patients. 21% treated with a concomitant immunotherapy. Patients were followed for a mean of 12.7 ± 6.7 years (3-33 months )
Efficacy (non-confirmed EDSS) as of 4/2019 (to be updated at presentation): 13% of patients improved, 79% were improving or stable and 21% worsening from their last measure to M0. Improvement was similar between PPMS and SPMS (11% vs 15%). 7% improved at M3 (81% stable or improving) with 9% worsening. 81% improving or stable at M12 (14% improving, 9% worsening). . 21% improved (82% stable or improved, and 1% worsening) in Year 2. PPMS trended to greater improvement in year 2 than SPMS (30% vs 17%).
Conclusions: This is the largest real life MS cohort of an early access program. A majority of patients improved or were stable on treatment. . A DMT effect is possible as improvement is seen after 1 year of treatment particularly evident in year 2 for PPMS patients. MD1003 appears to be a well-tolerated efficacious treatment for reversing disease progression in a population with few therapeutic options.
Disclosure:
Ayman Tourbah has received consulting and lecturing fees, travel grants and research support from Medday Pharmaceuticals, Biogen, Sanofi-Genzyme, Novartis, Merck, Teva Pharma, and Roche
Jérôme De Sèze, Marc Debouverie, Eric Berger served as consultant, board and therapeutical trials for Novartis
Thibault Moreau reports receiving consulting fees and speaking fees from Biogen Idec, Sanofi Aventis, Genzyme, Teva Pharma, Bayer Schering, Merck Serono, Roche, Almirall and Novartis, Roche.
Nicolas Collonques, Guillaume Mattey, Gaëlle Romain and Cédric Castex have nothing to disclose
Investigator sponsored study supported by MedDay Pharmaceuticals

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