MD1003 is a highly-concentrated pharmaceutical-grade biotin (vitamin H). The dosage is 300 mg/day corresponding to 10,000 times the recommended daily intake of biotin. As such, MD1003 is no longer a food supplement: because of potential toxicity and new therapeutic properties at this dosage, it is an active pharmaceutical ingredient.
A patent protecting the dose and use in multiple sclerosis (MS) has been allowed in the U.S. and Europe.
Biotin is a key co-factor for enzymes involved in energy production and synthesis of myelin. Biotin has potentially two targets related to progressive MS: (1) it activates the Krebs cycle in demyelinated axons to increase energy production; (2) it activates acetyl-CoA carboxylases (ACC1 and ACC2), the rate-limiting enzymes in the synthesis of long chain fatty acids required for myelin synthesis.
The proof of concept has been obtained in a pilot open label study involving 23 subjects with primary and secondary progressive MS. Results were remarkably positive with up to 90% of the subjects who exhibited clinical improvement over time. Treatment efficacy was also assessed using electrophysiology studies and magnetic resonance spectroscopy in some patients.
Two phase 2b/3, placebo-controlled, pivotal studies are ongoing, involving 250 patients and 21 MS reference centers in France and the UK (ClinicalTrials.gov Identifiers: NCT02220933 and NCT02220244).
The company works closely with regulatory agencies to ensure the design of its clinical projects meet the highest standards.
Pharmacokinetic studies in healthy volunteers, animal toxicology studies and pre-clinical proof of concept studies are being performed.
In addition, a phase 2b/3 clinical study has started in the adult form of X-linked adrenoleukodystrophy. This clinical trial is funded by the European Leukodystrophy Association (ELA) and involves four reference centers in France, Germany and Spain.