Abstract Details

Title Effect of MD1003 (high dose Pharmaceutical grade Biotin) in the Treatment of Progressive MS: Long-term Safety Data at 48 Months of Follow-up

Topic Multiple Sclerosis

Presentation(s) P3 - Poster Session 3 (5:30 PM-6:30 PM)

Poster/Presentation
Number 15-043

Objective To evaluate the safety of MD1003 in patients (pts) with progressive multiple sclerosis (PMS) during the open-label extension phase of the MS-SPI study.

Background MS-SPI was a 12-month (M) double-blind study of MD1003 (n=103) vs placebo (n=51) in 154 pts with nonactive PMS. In the double-blind phase, MD1003 reversed MS-related disability in 13% of treated pts and had a safety profile that was similar to that of placebo (Tourbah et al. 2016). In the open-label extension phase, all pts received MD1003 after M12. Here, we present safety data at 48 months of follow-up (shown as MD1003>MD1003 [MM] vs placebo>MD1003 [PM]).

Design/Methods A total of 133 pts (MM: 91; PM: 42) entered the extension phase. At M12, M18, M24, M30, M36, M42, and M48, we assessed discontinuations, adverse events (AEs), and serious adverse events (SAEs).

Results In the open-label extension phase (M12 to M48), the main reasons for treatment withdrawal (n=68 pts) were: perceived lack of efficacy (29 pts), consent withdrawal (21 pts), and AEs (7 pts). Discontinuations due to AEs in the extension phase were related to: MS relapse (2 pts), stroke, cancer, loss of weight, suspicion of myopathy, and cytomegalovirus. AEs were experienced by 78% vs 88% of pts in MM and PM groups, respectively. Of pts who entered the extension phase, 35.3% (47/133) experienced ≥1 SAE from M12 to M48: 35.1% of MM pts and 35.7% of PM pts. We did not observe that MS relapse rate was influenced by exposure to MD1003.

Conclusions Results from the MS-SPI study at 4 years of follow-up indicate that MD1003 is well-tolerated. No new safety signals have been detected in the extension phase. These data confirm previous observations made after 3 years of follow-up.

Authors/Disclosures
Jerome De Seze PRESENTER	Jerome De Seze has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Pharma. Jerome De Seze has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Pharma. Jerome De Seze has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Pharma. Jerome De Seze has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Pharma. Jerome De Seze has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Pharma. Jerome De Seze has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Pharma. Jerome De Seze has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Pharma. Jerome De Seze has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Pharma.
Gilles Edan	Gilles Edan has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Celgene. Gilles Edan has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Merck. Gilles Edan has received personal compensation in the range of $0-$499 for serving on a Speakers Bureau for Biogen. Gilles Edan has received personal compensation in the range of $0-$499 for serving on a Speakers Bureau for Merck.
Thibault Moreau	Thibault Moreau has nothing to disclose.
Bruno Brochet, MD, FEAN (University of Bordeaux)	Prof. Brochet has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Merck. Prof. Brochet has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for celgene. Prof. Brochet has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for jansen. Prof. Brochet has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for biogen. Prof. Brochet has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for novartis. Prof. Brochet has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for merck. Prof. Brochet has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for biogen. Prof. Brochet has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for celgene.
Frederic Sedel	No disclosure on file
Ayman P. Tourbah (Department of Neurology University Hospital Raymond Poincaré)	No disclosure on file

��ɫ��

��ɫ��