MD1003, a high-dose biotin developed by MedDay, slowed or prevented further disease progression among progressive multiple sclerosis (MS) patients in a Phase 3 clinical trial, researchers announced at the Oct. 25–28 7th Joint ECTRIMS-ACTRIMS Meeting in Paris, France.
The effects of the treatment were seen to be upheld over time and benefitted all patients in the trial similarly, offering hope to progressive MS patients that a new treatment for their condition may be on the horizon.
Presented by a team of researchers in France, the poster titled, “Effect of MD1003 (High-Dose Biotin) for the treatment of progressive MS: 36-month follow-up data,” highlighted robust effects of high-dose biotin over several years.
The MS-SPI Phase 3 trial (NCT02220933) included patients with progressive MS with no inflammatory disease activity. Patients were randomly assigned to treatment with 100 mg of MD1003 three times daily, or a placebo.
In total, 103 patients received MD1003 and 51 were on placebo treatment. Participants in the placebo group (42 patients) were switched to MD1003 after 12 months.
After nine months, 13% of MD1003-treated patients had improved their Expanded Disability Status Scale (EDSS) scores and performed better on the timed 25-foot walk, a measure of mobility and leg function. Meanwhile, none of the patients receiving a placebo had improved. This proportion remained stable over the next two years.
Researchers noted that while the change in EDSS was halted among placebo-treated patients when they switched to MD1003, they remained at a higher level throughout the study, suggesting that earlier treatment is advisable.
At 36 months, 67% of those who received MD1003 throughout the study had experienced an adverse event, compared to 79% of those who first received a placebo.
The team suggested that the effects of MD1003 appear sustainable over time, the drug is able to halt disease progression, and it is well-tolerated.
In a separate poster also presented at the meeting, “Effect of MD1003 (High-Dose Biotin) in spinal progressive multiple sclerosis (MS-SPI): subgroup analyses,” researchers demonstrated that the treatment’s effects were similar for people with primary or secondary progressive MS, or those with high or low EDSS scores in the MS-SPI Phase 3 trial.
The effects were independent of treatment with another disease-modifying drug and of intensive physical therapy.
Finally, the study, “MD1003 in progressive multiple sclerosis: 24-month brain MRI results of the MS-SPI trial,” presented by Douglas Arnold from McGill University Health Centre in Canada, discussed the pseudo-atrophy phenomenon.
Arnold showed that after 12 months, people treated with MD1003 had a lower whole brain and gray matter volume compared to those in the placebo group.
After 24 months, the MD1003 group appeared to have stabilized, while those who had been on placebo for the first 12 months experienced reduced volumes when switched to MD1003, an effect that was maintained in the following 12 months.
No difference in brain volume between the two groups could be detected at 24 months.
Researchers argue that this decrease is linked to a lowering of brain water volume, and may be the result of increased energy production triggered by high biotin doses.
Nonetheless, the results showed that brain energy metabolism is a key factor in progressive MS that can be targeted with high doses of biotin.