MedDay provides update on pioneering pivotal Phase III study design in Progressive Multiple Sclerosis
~ Phase III data to be disclosed at AAN Annual Meeting in Clinical Trials Plenary Session on April 24th 2015~
Paris, France, April 7 2015 – MedDay, a biotechnology company focused on the treatment of nervous system disorders, today provided further information about the design of its pivotal clinical trial (MS-SPI) to investigate the efficacy and safety of MD1003 in the treatment of primary and secondary progressive multiple sclerosis, a major area of unmet medical need. Data from the MS-SPI study will be presented at the Clinical Trials Plenary Session at The American Academy of Neurology (AAN) Annual Meeting, Washington DC, on Friday April 24th at 1200 EST.
MS-SPI is a randomized, double-blind, multicenter, placebo-controlled (2:1) trial of MD1003, 300 mg/day, in patients with progressive MS who have demonstrated progression in the 2 years prior to enrolment.
A total of 154 patients with a baseline EDSS (Expanded Disability Status Scale) score of between 4.5 and 7 were enrolled from 16 MS reference centers across France. Treatment duration was one year.
The primary endpoint for the study was defined as the proportion of patients who improved at nine months (M9), with confirmation at 12 months (M12). Improvement was defined as either a decrease in EDSS (by at least 1 point for baseline EDSS ≤5.5 and 0.5 points for EDSS ≥6) or an improvement in TW25 (a timed 25-foot walk) of at least 20%. The comparison for each outcome was the best EDSS and TW25 scores obtained at the screening and randomisation visits.
The main secondary endpoints evaluate the effect of MD1003 in stabilizing or slowing down the rate of progression. These endpoints include the change in EDSS between M0 and M12, the proportion of patients with progression at M9 confirmed at M12 and the change in TW25.
Frédéric Sedel, MD, Chief Executive Officer of MedDay, commented: “This trial was particularly ambitious. This is the first time that a study in progressive MS has evaluated the proportion of patients improved at M9 and confirmed at M12. This challenging clinical endpoint was designed during discussions with European and US regulators. We look forward to presenting the results of the trial at AAN later this month.”
Another important objective of the trial is to evaluate the safety of long-term treatment with MD1003 300 mg/day. Serious and non-serious adverse events recorded during the trial will also be presented.
MS is the most common disabling neurological disease among young adults, with first symptoms typically manifesting between 20 and 40 years of age. In the majority (85%) of cases, patients experience an initial phase of relapsing-remitting neurological dysfunction (RRMS), which typically evolves into a secondary progressive disease at a later point in the clinical course (SPMS). Once MS is in the progressive phase, individuals experience a gradual worsening of neurological disability leading to problems with vision, walking, incontinence, cognitive changes, fatigue, and pain. Primary progressive MS (PPMS) characterized by disease progression from onset is less common, affecting 10–15% of patients.
Despite these different initial clinical phenotypes, the time to reach certain disability milestones and the ages at which the milestones are reached are similar for patients with PPMS and SPMS. Recent guidelines have therefore proposed to group PPMS and SPMS within a single entity called “progressive disease”. The overall prevalence of patients with progressive disease is estimated to be at least 40% of all MS patients.
Full session details and data presentation listings for the 2015 Annual Meeting can be found through the AAN website: www.aan.com/conferences/2015-annual-meeting.
MD1003 is an investigational medicine thought to have both pro-myelinotic effects and to enhance the supply of energy for nerve impulse transmission. MD1003 is an active pharmaceutical ingredient administered at a dose of 300 mg /day. It has patent protection in the EU and US for dose and use in multiple sclerosis. MD1003 has a mode of action which potentially influences two targets related to progressive MS: (1) it activates acetyl-CoA carboxylases (ACC1 and ACC2), the rate-limiting enzymes in the synthesis of long chain fatty acids required for myelin synthesis; and (2) it activates the Krebs cycle in demyelinated axons to increase energy production.
MD1003’s proof of concept has been obtained in a pilot open label study involving 23 subjects with primary and secondary progressive MS. Results were positive with up to 90% of subjects exhibiting clinical improvement over time. Treatment efficacy was also assessed using electrophysiology studies and magnetic resonance spectroscopy. Results were published this year in the Journal of Multiple Sclerosis and Related Disorders. For more information on the study, please see: www.msard-journal.com/article/S2211-0348(15)00006-1/abstract.
Scientific Advisory Board
Prof. Alan Thompson (Chair, UCL, UK); Prof. Jack Antel (McGill, Canada); Dr Robert Fox (Cleveland, USA); Prof. Reinhard Hohlfeld (Munich, Germany); Prof. Jean Pelletier (Marseille, France); Prof. Per Soelberg Sorensen (Denmark); and Prof. Ayman Tourbah (Reims, France, principal investigator in the study).
MedDay is a privately held biotechnology company developing new drugs for nervous system disorders. The company was founded in 2011 by Frédéric Sedel, MD, PhD (Chief Executive Officer); and Guillaume Brion, MD (Chief Operating Officer). In April 2013, InnoBio, a biotechnology fund managed by BPIFrance, and Sofinnova Partners together invested in MedDay. The Company’s most advanced pipeline candidate is MD1003 for the treatment of primary and secondary progressive multiple sclerosis. For more information, please see: medday-pharma.cdmail.biz.
For more information, please contact:
Consilium Strategic Communications
Mary-Jane Elliott, Jonathan Birt, Ivar Milligan, Laura Thornton
Tel: +44 (0)20 3709 5700
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