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MD1003 (biotin)

Other names: Qizenday

MD1003 is a highly concentrated formulation of biotin under investigation for secondary and primary progressive multiple sclerosis. It is taken as a capsule, three times a day.

In December 2017, the company developing high dose biotin for progressive MS, informed the European Medicines Agency (EMA) that it wished to withdraw its licence application for the treatment of progressive multiple sclerosis.

Summary

MD1003 for secondary progressive MS: Phase III

MD1003 for primary progressive MS: Phase III

MD1003 is a highly concentrated formulation of biotin under investigation for secondary and primary progressive multiple sclerosis. It is taken as a capsule, three times a day.

  • Biotin activates enzymes involved in cellular energy production and myelin synthesis.
  • A small pilot study has provided initial evidence that high doses of biotin might have an impact on disability and progression. A phase III clinical trial showed some evidence of a small improvement in disability.
  • No significant side effects have been reported so far. 

How does MD1003 work?

Biotin, also known as vitamin H or coenzyme R, is one of the B-group vitamins (vitamin B7). It is necessary for cell growth, the production of fatty acids, and the metabolism of fats and amino acids, the building blocks of proteins. At the cellular level, it activates enzymes involved in energy production and synthesis of myelin.

Very high doses of biotin may be effective in MS by promoting myelin repair through activation of an enzyme involved in myelin synthesis and by enhancing energy production in demyelinated nerves.

MD1003 is a highly-concentrated formulation of biotin. The doses being used in clinical trials correspond to 10,000 times the recommended daily intake of biotin. At this dose, it is not considered a food supplement and is being developed as a pharmaceutical preparation. Neurologists are warning that people should not start taking large quantities of biotin supplements which are manufactured to a lower quality than the pharmaceutical grade biotin used for this study.

How is MD1003 taken?

MD1003 is taken by mouth, as a capsule, three times a day.

MD1003 research

What are the results so far?

A small pilot study of 23 people with primary and secondary progressive MS has provided initial evidence of effectiveness and safety of high doses of biotin. This was an open study - in other words both the people with MS and their doctors knew what treatment they were receiving; this can bias the results. Participants were treated with high doses of biotin (100-300 mg/day) for 2 to 36 months (average of 9.2 months). 21 out of 23 participants showed evidence of improved disability, 2 to 8 months after starting treatment.

In another small open study, 43 people with progressive MS took a single daily dose of 300mg high dose biotin for one year. Disability levels (EDSS) were recorded at the start and every three months. None of the participants' EDSS scores improved. One-third (38-43%) worsened, most often with increased leg weakness, worsened balance, and more falling. 

  • MS-SPI - MD1003 compared to placebo in primary and secondary progressive MS

Investigators recruited 154 people with secondary or primary progressive MS who were having increasing difficulty with walking and leg weakness (EDSS 4.5 - 7).

Participants took either MD1003 or placebo for the first 12 months, then all participants took MD1003 for a further 12 months. Participants continued with any medications or other treatments they were already taking. Approximately half of the participants in each group were taking fampridine, and approximately 40% in each group were taking a disease modifying drug.

The main measure of the study was improvement in disability after 9 months of treatment which was still evident at 12 months. Improvement in disability could be either a reduction in EDSS or a reduction in the time to walk 25 feet. Slightly less than 13% of the MD1003 group and none of the placebo group met this criteria.

At month 24, 14 of 91 (15%) participants taking MD1003 throughout the study and 5 of 42 (12%) of participants who switched to MD1003 had reduced MS-related disability.

  • MS-ON - MD1003 compared to placebo in chronic visual loss related to optic neuritis in multiple sclerosis

This phase III trial recruited 93 people with long term visual problems resulting from multiple sclerosis related optic neuritis. Participants took 300mg/day MD1003 or placebo for 24 weeks, followed by all participants receiving MD1003 for another 24 weeks. People taking MD1003 improved slightly more than those in the placebo group, though the difference was not statistically significant.

What further research is planned?

  • SPI2 - MD1003 compared to placebo in primary and secondary progressive MS

SPI2 will compare MD1003 (300mg/day) or placebo taken as a capsule three times a day in approximately 300 people with primary or secondary progressive MS from North America and Europe, including study centres in Edinburgh, London and Manchester. Participants will take either MD1003 or placebo for the first 15 months then all particpants will take MD1003 for a further 12 months. The main measure of the study will be the number of people with an improvement in their disability, defined as either a lower EDSS score or a reduction in the time to walk 25 feet.
Estimated completion date September 2019.
Further details of this study.

Side effects

In the MS-SPI study there were no serious side effects. Mild to moderate side effects included urinary tract infections and headache and were reported for both placebo and MD1003 groups, suggesting that side effects were not caused by biotin.

High levels of biotin in the blood can interfere with laboratory tests and cause incorrect test results which may go undetected and could lead to misdiagnosis and inappropriate treatments. Anyone taking biotin should tell their health team.