MedDay Presents Phase 3 Extension Data on Potential Drug to Treat Progressive MS

Patricia Silva, PhD avatar

by Patricia Silva, PhD |

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MedDayā€™s MD1003 and progressive MS

Data from an extension phase of aĀ Phase 3 clinical trial, given in an oral presentation by MedDay, reportedĀ that the biotinĀ Ā MD1003 showedĀ effectiveness over time as a possibleĀ treatment of non-active, progressive multiple sclerosis (MS).

The dataĀ were presented atĀ the recentĀ 2nd Congress of the European Academy of Neurology (EAN) in DenmarkĀ byĀ ProfessorĀ Ayman Tourbah, the trial’s principal investigator, in the talkĀ “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 treatĀ patients with not-active progressive MS. ParticipantsĀ 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 aĀ news release.

The study continued in anĀ extension phase, during which all patients received theĀ drug but remained uninformed of whether they had been treated with MD1003 in the first phase.

MedDay presented dataĀ in April 2015, atĀ the American Academy of Neurology’s (AAN) annual meeting that year, showed the trialĀ met its primary endpoint ā€” a reversal in disease progression as measuredĀ Expanded Disability Status Scale (EDSS) or time to walk 25 feet (TW25) ā€” potentially making MD1003 the first drug able to treatĀ progressive MS.

Results from the extension phase,Ā firstĀ 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 andĀ 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 aĀ demyelinating diseaseĀ in which the insulating coversĀ ofĀ nerve cells (myelin) in theĀ brainĀ andĀ spinal cordĀ are damaged.Ā This damage disrupts the ability of parts of the nervous system to communicate, resulting in a range ofĀ signsĀ andĀ symptoms, including physical,Ā mental, and sometimes psychiatric problems.

EAN 2016 ran fromĀ May 28 to May 31 inĀ Copenhagen, Denmark.