MedDay, a biotechnology company dedicated to developing therapies for nervous system disorders, recently announced encouraging data on its pivotal Phase III clinical trial (MS-SPI) assessing the safety and efficacy of the company’s investigational therapy MD1003 for the treatment of progressive multiple sclerosis (MS). The data was presented at The 1st Congress of the European Academy of Neurology (EAN), held June 20 to 23, 2015 in Berlin, Germany.
MS is a chronic, progressive neurodegenerative disorder that is the result of an attack on the central nervous system (which includes the brain, spinal cord and optical nerves) by the body’s own immune system, causing inflammation, damage to the myelin layer that covers and protects neurons affecting motor function (coordination, balance, speech and vision) and leading to irreversible neurological disability and paralysis. It is estimated that more than 2.3 million people in the world suffer from the disease.
Progressive MS is estimated to affect at least 40% of all MS patients. It is characterized by a gradual, steady progression of disability, leading to impaired vision and walking, pain, fatigue, incontinence and cognitive changes. Patients usually have a poor response to treatment and there is little or no recovery. Progressive MS can be either primary, where patients develop the form of the disease from the time of diagnosis, or secondary, where patients initially experience a relapsing-remitting phase of neurological dysfunction that later evolves into a secondary progressive disease. Primary progressive MS only affects 10 to 15% of the MS patients, while the majority (85%) develop secondary progressive MS.
MD1003 is an experimental drug that has pro-myelinotic properties and is thought to improve the supply of energy for nerve impulse transmission. It acts by activating acetyl-CoA carboxylases (ACC1 and ACC2), which are the rate-limiting enzymes in the fatty acids synthesis required for myelin formation, and by activating the Krebs cycle in demyelinated axons to increase energy production.
The MS-SPI study is a multicenter, randomized, double-blind, placebo-controlled trial assessing MD1003 (300 mg/day) in patients with progressive MS in 16 different medical centers in France. In total, 154 MS patients who had a baseline Expanded Disability Status Scale (EDSS) score of 4.5 to 7 were evaluated. The study was carried out for 12 months.
MS-SPI trial primary endpoint was the proportion of patients who exhibited disease improvement at 9 months (M9) and 12 months (M12) after MD1003 treatment. Improvement was established as a decrease in EDSS score or an improvement of at least 20% in a timed 25-foot walk test (TW25).
MedDay now reports that MD1003 treatment for 12 months results in an improvement of the Clinical Global Impression of change, a scale that assesses disease improvement or worsening relative to a baseline state. A significantly better Clinical Global Impression score was observed among patients under MD1003 treatment in comparison to patients in the placebo group. The primary endpoint of the trial was met, with the EDSS score decreasing in the MD1003 group while it progressed in the placebo group. Researchers estimated that in comparison to MS patients in the placebo group, patients taking the MD1003 treatment had a 67% decreased risk of disease progression.
“We are pleased to announce further positive findings which confirm the promising MD1003 phase 3 data announced earlier this year at AAN [The American Academy of Neurology Annual Meeting]. MD1003 remains the only drug to date that has demonstrated an ability to decrease the rate of disease progression and improve a significant proportion of patients with progressive MS,” said the Chief Executive Officer of MedDay Dr. Frédéric Sedel in the news release. “A second phase III placebo-controlled trial is underway looking at the effect of MD1003 in MS patients with permanent visual loss following optic neuritis and we look forward to announcing data from this trial later this year and potentially investigating a drug filing thereafter.”