MD1003 (biotin)

MD1003 is a highly concentrated form of biotin, a vitamin that activates some enzymes involved in cell growth and myelin production.

It is a tablet taken three times a day.

SPI2 trial

This phase 3 trial will test the benefits of 300 mg MD1003 daily for people with both primary and relapse-free secondary progressive MS. The trial will involve 600 people with MS and is due to finish in September 2019.

Researchers will test the safety and effectiveness of MD1003 at improving disability (and in particular mobility) for people with progressive MS compared with placebo.

In September 2016, MedDay published the results from a phase 3 clinical trial (MS-SPI). This trial tested the effectiveness of MD1003 as a treatment for primary progressive or relapse-free secondary progressive MS.

The trial involved 144 people that took either:

• MD1003 (doses ranging from 100 - 600 mg) daily for 24 months
• a placebo for 12 months followed by 300 mg of MD1003 daily for 12 months

The trial primarily focused on how effective MD1003 was at improving disability (especially mobility) in people with progressive MS. Approximately 13% of people taking MD1003 experienced an improvement in disability after 9 months, which was maintained at 12 months. No one taking placebo experienced any improvement. Improvement in disability was measured by the Expanded Disability Status Scale (EDSS) or a timed 25-foot walk.

Optic Neuritis

MedDay has also carried out a phase 3 clinical trial testing the effectiveness of MD1003 as a treatment for optic neuritis. 93 participants with MS who experience optic neuritis (progressive and non-progressive forms) took either:

• 300 mg of MD1003 daily for 12 months
• a placebo for 12 months then 300 mg of MD1003 for 6 months

Researchers found that MD1003 did not significantly improve clearness of vision or the thickness of the retina (the light sensitive nerve fibre layer at the back of the eye) compared with placebo.

A pilot study involving 23 participants with primary or secondary progressive MS revealed that high doses of MD1003 could improve disability in MS.

This was a non-blinded study (doctors and participants were aware they were taking MD1003), where people took 100-300 mg of MD1003 per day for 2 to 36 months. Of the 23 people taking part, 21 showed evidence of some kind of improvement, including improved vision, cognition or walking.

MD1003 was well tolerated in the phase 3 trial.

Researchers found that MD1003 interferes with some clinical tests that use biotin, like the blood test used to assess thyroid function. Five people taking MD1003 were diagnosed as having an overactive thyroid, these were later found to be false positives.

There are no treatments available for progressive MS yet.

MedDay are running a second phase 3 study in the US, with some UK sites. The trial is now open for recruitment, for more information see the UK clinical trials gateway.

If this trial is successful the treatment can then be submitted for licensing. We'll keep you updated on the progress of MD1003.