High-Dose Biotin Drug Improves Progressive Multiple Sclerosis Symptoms

WASHINGTON — An investigational multiple sclerosis drug that contains a high dose of biotin improved standard measures of disability among patients with progressive MS, findings from a phase-3 study indicate.

The drug, called MD1003 delivers 300 mg/day of biotin, a huge increase the usually recommended dose of 30-100 mcg/day.

“We are encouraged that the primary endpoint was met despite the very high bar for treatment response. This result suggests that MD1003 could be an important and efficacious treatment for primary and secondary progressive multiple sclerosis,” study researcher Ayman Tourbah, MD, of Hopital Maison Blanche, in Reims, France said at the American Academy of Neurology 2015 meeting.

Overall, 154 patients with a mean progressive MS duration of 16.6 years, a mean baseline Expanded Disability Status Scale (EDSS) score of 6.1, and evidence of EDSS progression in the preceding two years were randomly assigned to treatment with oral biotin 300 mg/day or placebo for 48 weeks.

In the MD1003 group, 12.6% of patients met the primary endpoint of an Expanded Disability Status Score of at least 1 point for baseline EDSS ≤5.5 and 0.5 points for EDSS ≥6, or an improvement in 25-foot walk time of at least 20%, according to the researchers. None of the patients in the placebo group met the primary endpoint.

Compared with disease progression in the placebo group, patients in the MD1003 group had a mean decrease in EDSS of -0.03 from baseline to month 12 (P=0.015). Overall, 4% of patients in the MD1003 group showed progression on the EDSS, as did 13% of patients in the placebo group (P=0.07).

The drug was generally well tolerated with no significant differences in adverse events between the MD1003 group and the placebo group.

The mechanism of action for the drug is activation of the acetyl-CoA carboxylases and activation of the Krebs cycle — two processes that effect key enzymes in myelin synthesis.

Two separate trials to evaluate MD1003 for visual endpoints in MS-related optic neuritis and the adult form of X-linked adrenoleukodystrophy are also underway.