Data from an extension phase of a Phase 3 clinical trial, given in an oral presentation by MedDay, reported that the biotin MD1003 showed effectiveness over time as a possible treatment of non-active, progressive multiple sclerosis (MS).
The data were presented at the recent 2nd Congress of the European Academy of Neurology (EAN) in Denmark by Professor Ayman Tourbah, the trial’s principal investigator, in the talk “High doses of biotin in progressive multiple sclerosis: extension phase results of the MS-SPI trial.”
MS-SPI, as the clinical trial and its two-year follow-up is known, evaluated MD1003 at a total dose of 300 mg per day (100 mg, 3 times a day) to treat patients with not-active progressive MS. Participants had to demonstrate disease progression of disability in the previous two years with no evidence of inflammatory activity, and were randomly assigned to receive either MD1003 (n=103) or placebo (n=51) for 12 months, according to a news release.
The study continued in an extension phase, during which all patients received the drug but remained uninformed of whether they had been treated with MD1003 in the first phase.
MedDay presented data in April 2015, at the American Academy of Neurology’s (AAN) annual meeting that year, showed the trial met its primary endpoint — a reversal in disease progression as measured Expanded Disability Status Scale (EDSS) or time to walk 25 feet (TW25) — potentially making MD1003 the first drug able to treat progressive MS.
Results from the extension phase, first presented at AAN 2016, determined sustained efficacy for up to two years and further established a good safety profile for the drug.
MD1003 is a highly concentrated form of biotin, a vitamin that activates some enzymes involved in cell growth and myelin production. The drug, which is already commercially available in certain European countries under early access programs, has previously shown efficacy in patients with progressive MS. It acts by increasing a route of cellular energy production, protecting against the breakdown of nerve cell axons. It also activates enzymes that are setting the pace on myelin repair by being involved in the production of myelin constituents.
MS is a demyelinating disease in which the insulating covers of nerve cells (myelin) in the brain and spinal cord are damaged. This damage disrupts the ability of parts of the nervous system to communicate, resulting in a range of signs and symptoms, including physical, mental, and sometimes psychiatric problems.
EAN 2016 ran from May 28 to May 31 in Copenhagen, Denmark.