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Press Release

Positive Preclinical Data with MD1003 (high dose pharmaceutical grade biotin) in X-Linked Adrenoleukodystrophy to be presented at European Committee for Treatment and Research in Multiple Sclerosis 2016

MedDay
Posted on: 14 Sep 16

Paris, France, 14 September 2016 - MedDay, a biotechnology company focused on the treatment of nervous system disorders, today announces 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, UK.

 

The poster presentation entitled “MD1003 halts axonal degeneration and locomotor disability in a model of X-linked adrenoleukodystrophy” will take place during “Poster Session 1” on Thursday 15th September 2016 between 15.45 – 17.00 BST.

 

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).

 

Commenting on the results, Frederic Sedel, CEO of MedDay, said: “We are pleased with the encouraging early results from this preclinical study of MD1003 in X-linked Adrenoleukodystrophy. These data demonstrate the potential for MD1003 to provide treatment for patients with neurological diseases beyond progressive multiple sclerosis.”

Editor's Details

Mike Wood
PharmiWeb.com
www.pharmiweb.com
editor@pharmiweb.com

Last updated on: 14/09/2016

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