Date: 2016-09-14
Type of information: Presentation of results at a congress
phase: preclinical
Announcement: presentation of results at the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS)
Company: Medday (France)
Product: MD1003 (high doses of pharmaceutical-grade biotin)
Action
mechanism: vitamin. MD1003 is an active pharmaceutical ingredient administered at a dose of 300 mg /day has patent protection in EU and US for dose and use in multiple sclerosis. MD1003 has a mode of action which potentially influences two targets related to progressive MS: (1) it activates acetyl-CoA carboxylases (ACC1 and ACC2), the rate-limiting enzymes in the synthesis of fatty acids required for myelin synthesis, and (2) it activates the Krebs cycle in demyelinated axons to increase energy production.
MD1003’s proof of concept has been obtained in a pilot open label study involving 23 subjects with primary and secondary progressive MS. Results were positive with up to 90% of subjects exhibiting clinical improvement over time. Treatment efficacy was also assessed using electrophysiology studies and magnetic resonance spectroscopy.
Disease: adrenoleukodystrophy
Therapeutic area: Rare diseases - Genetic diseases
Country:
Trial details:
Latest
news: * On September 14, 2016, MedDay announced that new preclinical data in adrenoleukodystrophy will be presented during a poster session at the annual meeting of the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS) in London. The study, which was conducted by The Neurometabolic Diseases Lab led by Professor Aurora Pujol, investigated whether MD1003 could improve the clinical signs of the disease (axonal degeneration and locomotor deficits), and aimed to identify by which molecular and biochemical mechanisms it operates. The results show both preclinical safety and efficacy in two mouse models of X-ALD and reveal that MD1003 halted the late-onset axonopathy, which are the main contributors to disability in progressive neurodegenerative diseases such as X-ALD. Specifically, MD1003 normalized ATP and mtDNA levels in Abcd1- mice spinal cords. This induction of mtDNA is correlated to an increase of mitochondrial biogenesis factors and to inhibition of the alternative NFkB pathway (NFkB2) and downstream cytokine production. Most importantly, the treatment of MD1003 halted the locomotor disability as assessed by treadmill and bar-crossed tests in the mouse model of X-ALD (Abcd1/Abcd2 null mice).
The poster presentation is entitled “MD1003 halts axonal degeneration and locomotor disability in a model of X-linked adrenoleukodystrophy”.
