Addex gets approval for clinical trial
By BFN News
Wednesday 24 April, 2013 at 08:05 AM
Addex Therapeutics has been given the go-ahead in the Netherlands to start a phase 1 study of ADX71441, a GABA-B receptor positive allosteric modulator (PAM).
The company plans to initiate clinical testing at the Centre of Human Drug Research in Leiden, in the first half of this year and expects to deliver top-line safety, pharmacokinetic and biomarker data by year end.
Addex previously announced achievement of positive 'proof of concept' for ADX71441 in a validated pre-clinical model of Charcot-Marie-Tooth 1A (CMT1A) neuropathy as well as a number of other disease indications.
The company plans to move quickly to Phase 2a testing of ADX71441 for the treatment of CMT1A, an orphan disease, in 2014. The clinical trial application was submitted to the authorities in March.
CMT1A is a rare (1:5,000) hereditary motor and sensory demyelinating peripheral neuropathy (also known as Hereditary Motor and Sensory Neuropathy, HMSN) which is caused by an intrachromosomal duplication and consecutive toxic over-expression of the PMP22 gene on chromosome 17. CMT1A is one of the most common inherited peripheral nerve-related disorders, which is passed down through families in an autosomal dominant fashion.
The disease can be highly debilitating, including being wheelchair bound, and is often accompanied by severe cases of neurological pain. There is no known cure for this incapacitating disease
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