MD1003

MD1003 (also known as biotin or Cerenday) is an oral medication that is in development for progressive MS. It's currently in phase 3 clinical trials for primary and secondary progressive MS and optic neuritis.
Current phase of trial: phase 3

Type of MS: progressive MS and optic neuritis (inflammation of the optic nerve)

How does MD1003 work?

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

How is MD1003 taken?

It is a tablet taken orally.

Latest research

SPI2 trial

This phase 3 trial will test the benefits of MD1003 for people with both primary and relapse-free secondary progressive MS. The trial will involve 300 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.

MS-SPI trial

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:

  •  300 mg of MD1003 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.

Earlier results

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) were split into two groups, receiving either:

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

The aim was to test the effect of MD1003 on the clearness of vision and the thickness of the retina (the light sensitive nerve fibre layer at the back of the eye). The results did show that MD1003 had some improvement compared to placebo but this was not significant and the overall study did not show positive results. The group that saw the most benefit was those with progressive forms of optic neuritis.

Pilot study

A small pilot study involving 23 participants with primary or secondary progressive MS demonstrated that high doses of MD1003 may lead to improvements in disability. This was a non-blinded study (doctors and participants were aware they were taking MD1003), where participants were given 100-300 mg of MD1003 per day for 2 to 36 months. 21 out of the 23 taking part showed evidence of some kind of improvement, including improved vision, cognition or walking.

What are the side effects of MD1003?

No significant side effects were reported during the small pilot study.

How does MD1003 compare with current therapies?

MD1003 has not yet been compared with current therapies in clinical trials.

When is MD1003 likely to become available?

MedDay have announced that they will run a confirmatory phase 3 study in the USA following the initial phase 3 trial. It will therefore be a few years before we know if this is an effective treatment for people with MS.

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