MedDay Announces Its Pivotal Phase III Study Of MD1003 In Patients With Progressive Multiple Sclerosis Meets Primary Endpoint

~ Detailed Phase III data will be presented at AAN Annual Meeting in Clinical Trials Plenary Session on April 24th 2015 ~

PARIS--(BUSINESS WIRE)--MedDay, a biotechnology company focused on the treatment of nervous system disorders, today announces that the primary endpoint was met in its pivotal clinical trial MS-SPI. MS-SPI investigated the efficacy and safety of MD1003, a highly-concentrated pharmaceutical-grade biotin administered at a dose of 300 mg/day, in the treatment of progressive multiple sclerosis.

“The trial design and dosing were discussed with US and European regulators and we are pleased the results demonstrate evidence of improvement at one year in patients with progressive worsening MS”

The primary endpoint for the study was defined as the proportion of patients who improved at 9 months, with confirmation at 12 months. Detailed data will be presented for the first time at the Clinical Trials Plenary Session at The American Academy of Neurology (AAN) Annual Meeting, Washington DC, on Friday April 24^th at 1200 EST.

“We are encouraged that the primary endpoint was met despite the very high bar for treatment response. This result, which will be disclosed at AAN on April 24th along with supportive analyses and safety data, suggests that MD1003 could be an important and efficacious treatment for primary and secondary progressive multiple sclerosis,” said Prof. Ayman Tourbah, Principal Investigator of the study, CHU de Reims, Neurology, France.

“The trial design and dosing were discussed with US and European regulators and we are pleased the results demonstrate evidence of improvement at one year in patients with progressive worsening MS,” said Frédéric Sedel, MD, Chief Executive Officer of MedDay.

About MS-SPI

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 two 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.

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.

About MD1003

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 has patent protection in 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 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 (Chairperson, 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).

About MedDay

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: www.medday-pharma.com.