High-dose biotin disappoints


Disappointing news 🙁


MedDay has just announced the second Phase III trial, SPI2, of high-dose biotin (MD1003) is negative. I am sure many people with progressive MS will be disappointed. However, this trial supports why we need to blinded, randomised, controlled trials; i.e. to test hypotheses. Let’s hope some lessons will be learnt from the trial data that will inform future trial design in more advanced MS.

I would like to thank all the people with progressive MS who participated in this study and all the diligent staff for making it happen. It may be a disappointing result, but without negative trial results, we don’t learn.

CoI: I am a member of SPI2 trial steering committee and our centre participated in the trial.

About the author

Prof G

Professor of Neurology, Barts & The London. MS & Preventive Neurology thinker, blogger, runner, vegetable gardener, husband, father, cook and wine & food lover.


  • “It may be a disappointing result, but without negative trial results, we don’t learn.”

    Haven’t we seen this all before: MS-SMART, Cupid (Cannabinoid Use in Progressive Inflammatory brain Disease), INSPIRE…….!

    The Bexarotene trial (potential remyelination therapy) should be reporting this month. I wonder would odds William Hill would give on a positive result?

    The MS Society is seeking to raise £100 million so that “By 2025, we want to be in the final stages of testing treatments for everyone with MS. Treatments that slow or stop disability progression.” Another fund raiser promising the world! I won’t be opening my piggy bank.

    Any thoughts Prof G on why so many of these trials (particularly relating to neuro-protection or re-myelination) fail? People with MS need therapies for protecting nerves and encouraging repair. Learning that a failed trial “will inform future trial design in more advanced MS” gives me no comfort whatsoever.

    • If the MS Society continues to pick duds, that money will be wasted.
      Although the headline result for the CUPID trial was negative, for a variety of reasons, in fact in a sub-group there was a neuroprotective effect. For me the MS Society should be looking to re-investigate cannabis as a neuroprotectant, learning the lessons of the CUPID trial.
      I strongly believe that if done properly there will be a positive effect.

      • I and ProfG tried in a recent drug selection process…the “shit has firmly stuck” and you can’t shift peoples minds that it didn’t work, but you have daat from n=100 and you contniue to ignore something that has clinical evidence, pre-clinical evidence as long as your arm.

    • Let’s wait for the data to be published and we can have a more detailed discussion on the trial design and lessons to be learnt.

  • This is disappointing. After 6 to 10 years of time and millions of dollars of investment. My question is phase 2 trials are not under powered a d yet showed positive effect. Is this pharma not understanding the science or a ill conceived from the beginning? I think it’s time to pharma turned to tech nology to do the initial ground work. Such as AI, Machine learning.

  • They’ve gone a long way with just 42m!

    “MedDay raised €42 million of funding to support the global development of the company.”

    The lesson here is possibly for funders as well.

    • They got much more than €42M; the French government was subsidising the development programme by covering the drug pre-license. Some estimates suggest they got > €200M this way. This is how France gives its Pharma companies a leg-up.

  • Sorry to hear this trial didn’t work. Now we know one way less to try.

    ‘I haven’t failed – I’ve just found 10,000 that won’t work’

    Thomas Edison

  • I was contacted to participate in this trial. Since I was already taking high dose biotin from a supplement supply company for about 6 months with no noticeable improvement I declined. The researchers tried to convince me that the biotin I was purchasing may not be pure. Sufficed to say I am not surprised with the results.

  • Are you implying that this outcome means the end of the road for treatment with Biotin?

    Wouldn’t it rather make sense to re-evaluate the trial endpoints, settle for less ambitious ones, and give it another try?

    The puzzling thing about Biotin ist that it had already received temporary authorization in France and thousands of patients in self-therapy were exchanging experiences in large Facebook communities over more than five years. So there actually has been an overwhelming set of data points amassed for this approach that – while clearly non-blinded – did consistently indicate the same clinical improvements for those 15-20% who responded to treatment.

    These improvements – such as more Energy and improved bladder function- typically did not imply a lower EDSS Score however would have warranted IMHO at least consideration as a therapeutic option for patients suffering from a progressive decline.

    Or is this simply now a question of business cases? Medday wanted to sell the high dose Biotin from their new NL facility and needed to charge astronomical prices for their pills to cover costs and generate profits. So they went for the moonshot of improved EDSS in order to justify a 20k p.a. treatment and failed. Now the VC funding is gone and they simply stop?

    Wouldn’t this then be a perfect case for some patient organization or public group to step in and carry this forward?

    • I suspect end of the Road and End of the Company…Remeber trials kill companies I mean drugs….you seldom get a second bite at a cherry…I can testify to this..That is why I bang on about trial design so much.

    • As you say there seems to have been government support (Stateaid) for this company…surely this is not normal.

      Anyway I heard a talk from the MEDDAY group and because the finding was discovered by accident there wsa never a clear rational for the the studies. They were thinking or neuroprotection studies and these are the trials they did. However, the effect was relatively immediate and so in my mind the agent was a symptom control agent or a remyelination agent as a neuroprotective agent was never going to have a rapid effect. Therefore, perhaps they shot them selves in the foot. The money men and women want a block buster a symptom control agent is a hard sell to get the big bucks and so commercially you get drawn into doing big buck trials…end resut failed approach and no more company and a massive loss to the French Taxpayer.

  • “I am a member of SPI2 trial steering committee”

    On the positive, at least your bank balance doesn’t disappoint! MS research is a business where failure is rewarded.

    • Is this really the time for such comments, “Bob”?
      In case you hadn’t noticed, there’s a bit of an emergency going on.

      • MD2,

        The emergency will pass. It’s a good time to announce bad news when the focus of the media is on something else. But work should continue. I can see 2020 being a nothing year for MS research – less conferences, less research published…. and the lack of progress can be blamed on Corona virus!

  • Presumably some participants in the trial were also on dmts – eg sub cutaneous Cladribine, ocrelizumab(?) – and some weren’t. Is there any analysis of data on responders and non responders amongst those on dmts/not on dmts? Just wondered whether possible to look for ‘add-on’ therapy benefit? (I realise MDs were always sceptical about the biology..!)

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