Authored by Karl Simpson
Nestled in the Bio-Cat cluster in Barcelona, Spain is the orphan drug therapeutics company, Minoryx Therapeutics, which is developing new medicines for the treatment of inborn errors in metabolism. The company is addressing these particular rare diseases via its proprietary platform that it believes will develop a new generation of pharmacological chaperones which are non-competitive with the natural substrate and therefore demonstrate improved pharmacological properties.
Minoryx Therapeutics this month, April 2014, announced that the European Commission has granted orphan drug designation to MIN-101 for the treatment of X-linked Adrenoleukodystrophy (ALD). MIN-101 is based in Pioglitazone, a potent agonist for peroxisome proliferator-activated receptor-gamma (PPAR gamma) marketed for the treatment of type-2 diabetes mellitus, showed efficacy in animal models of ALD.
ALD is a rare inherited neurodegenerative disease which is chronically debilitating and life threatening. This rare disease is caused by mutations in the ABCD1 gene located on the X-chromosome that encodes the peroxisomal membrane protein ALDP which is involved in the transmembrane transport of very long-chain fatty acids.
The clinical spectrum of ALD ranges from isolated adrenocortical insufficiency and slowly progressive myelopathy to devastating cerebral demyelination, leading to paralysis, dementia and death.
Minoryx is pursuing its approach is other rare diseases too, all programmes are pre-clinical.
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