Phase III Study of Experimental MS Therapy Meets Primary Endpoint, Results to be Presented at ANN Meeting
MedDay, a French biotechnology company that studies treatments for nervous system disorders, including multiple sclerosis, announced that MD1003, a highly-concentrated biotin, is effective for treatment of progressive multiple sclerosis, according to results from a recent Phase III trial.
MD1003 may have two beneficial effects: 1) increasing myelin, the fatty nerve-insulating substance that is destroyed in MS and 2) increasing energy to nerve cells so that they can communicate more effectively. Researchers previously studied the drug in what is called “a proof of concept” study. A total of 23 people with primary and secondary progressive MS received the drug in that initial trial, and 90% of subjects showed some clinical improvement over time. Results for that study appeared in the Journal of Multiple Sclerosis and Related Disorders.
MedDay researchers plan to present the results of a Phase III study for the first time at the Clinical Trials Plenary Session at The American Academy of Neurology (AAN) Annual Meeting in Washington DC, on Friday April 24th at 12:00 pm EST. Phase III is the final step before a drug can be assessed for approval and use in humans. The success of a Phase III study is based on whether or not the “primary endpoint” is met. In this study of MD1003, the primary endpoint consisted of the number of patients who improved at 9 months and also at 12 months. In terms of what was defined as improvement, the investigators measured either a decrease in EDSS or an improvement in a timed 25-foot walk of at least 20%. EDSS stands for “Expanded Disability Status Scale,” and is a standard way of measuring the severity of MS in a specific patient.
The researchers studied 154 participants with an EDSS score of between 4.5 and 7 at 16 different clinical locations in France for a period of one year.
“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,” stated 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,” noted Frédéric Sedel, MD, Chief Executive Officer of MedDay.
With the completion of this trial and the successful outcome, approval of the medication and clinical use in humans may rapidly follow.