Biotin, is a new treatment for MS?

A second phase 3 study of the investigational drug MD1003 (MedDay Pharmaceuticals), a highly concentrated pharmaceutical-grade biotin being studied for multiple sclerosis (MS), did not meet its primary and secondary endpoints, the company announced.

Biotin is a coenzyme for carboxylases, which are enzymes critical in energy metabolism and production of fatty acids. It targets two mechanisms that might be involved in progressive MS: promoting myelination and increasing energy production. It's hypothesized that biotin may help to slow, stop, or even reverse the progression of disability associated with demyelination.

The study known as SPI2 was designed to confirm the results of the first positive phase 3 study (MS-SPI). In that trial patients with progressive MS taking MD1003 had significant improvement in disability at 9 months, which was confirmed at 12 months, compared with those taking placebo.

The SPI2 trial was a randomized, double-blind, and placebo-controlled study evaluating safety and efficacy of MD1003 (100 mg three times daily) vs placebo in 642 patients with progressive MS without recent relapses (also called not-active progressive MS).

The primary endpoint for the study was reversal of functional disability as measured by the proportion of patients with an improvement in either the Expanded Disability Status Scale (EDSS) or in the time needed to walk 25 feet (TW25) over a 12-month period and confirmed at 15 months.

Secondary endpoints included the relative reduction in the risk of disability progression, global impression of response to treatment evaluated independently by both the patient and the evaluating physician, and mean change in TW25.

Additional exploratory endpoints incorporated in this trial included brain MRI measures, quality of life measures, and measurements of ambulation using an exercise tracker.

There were no treatment-emergent safety signals. The company did not provide any specific efficacy details at this time. They plan to report detailed results of the SPI2 trial in April at the American Academy of Neurology (AAN) 2020 Annual Meeting in Toronto, Canada.