Frédéric Sedel – MedDay – 2015

Preliminary data has shown that the drug MD1003 can stop the progression of the disease in patients with primary or secondary progressive multiple sclerosis (MS) and improve their symptoms. Among the 23 patients with progressive MS treated for an average of 9.2 months with the drug MD1003, 21 patients (91.3% of them) improved. The positive effects of the drug MD1003 may be linked to an increase in energy production in demyelinated neurons and stimulation of myelin repair. Analysis of two clinical trials involving 250 patients with progressive MS is currently underway and should confirm the previous results.

Adrenomyeloneuropathy (AMN) and progressive MS have similarities including a secondary energy problem leading to progressive axonal degeneration. One patient with AMN was treated with the drug MD1003 for 5 months and showed clinical improvement comparable to the effects seen in progressive MS. The objectives of the trial are to assess the efficacy and safety of MD1003 in patients with AMN. Sixty patients from 4 different centers (two in France, one in Spain, and one in Germany) will initially be divided into 2 groups: a group of 20 patients will receive a placebo while the second group of 40 patients will receive the drug MD1003. The placebo study will last 12 months and will be followed by a 12-month extension phase where all patients will receive the drug. The primary end point will measure, before and after 12 months of treatment, the average change observed during the 2MWT walking test measuring the distance covered in 2 minutes. Secondary end points will include:

• the TW25FW walk test measuring walking time over a distance of approximately 8 meters,
• time to get up and walk (or timed chair test)
• the Euroqol ED-5D and MOS SF-36 quality of life questionnaires
• a Qualiveen urinary function questionnaire.

Exploratory analyzes will also be carried out in certain centers, such as MRIs, measurement of nerve conduction speed and assessment of muscle strength. The recruitment of all patients will be completed in mid-June 2015.