High-dose biotin trial produces disappointing results

This week, MedDay Pharmaceuticals announced that the second pivotal Phase 3 trial (SPI2) of MD1003 (biotin), an investigational product for primary and secondary progressive MS has failed to meet its primary and secondary endpoints.

The trial evaluated the safety and effectiveness of three daily doses of high dose (100mg) biotin compared to placebo in 642 people living with progressive MS without recent relapse. The main measure of the trial was an improvement in disability which could be a lower EDSS score or a reduction in the time to walk 25 feet. The study failed to demonstrate an improvement in either of these measures compared to the placebo group.

MD1003 is a highly concentrated formulation of biotin.  Biotin is one of the B-group vitamins (vitamin B7). It has been suggested that 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.

An earlier clinical trial of 154 participants with progressive MS had provided initial evidence that high doses of biotin may impact disability progression. An improvement in disability after 9 months of treatment which was still evident at 12 months was the main measure of this earlier trial. Over 12% of the MD1003 group met this criteria compared to none in the placebo group.

MedDay have said they will present more detailed results from the study in April at the American Academy of Neurology (AAN) meeting which will take place in Toronto.

For more see: https://www.medday-pharma.com/2020/03/10/medday-reports-top-line-data-from-phase-iii-trial-spi2-for-treatment-of-progressive-forms-of-multiple-sclerosis/