French Pleodrug for Rare Neurological Disease opens up phase III trial in US

27/04/2016 - 2 minutes

Pharnext is opening their first US study sites for a phase III trial in a rare neuropathy – a milestone in the development of Pharnext’s leading candidate.

pharnext_pxt3003_charcot_marie_tooth_cmt1aBased in Paris (France), Pharnext is developing new therapies for neurological conditions, including rare diseases such as Charcot-Marie-Tooth Disease.

Pharnext uses network pharmacology to discover synergistic combinations of drugs (Pleodrugs) that can have a therapeutic effect in a given disease. As the components drugs are already approved, development is faster – meaning bypassing phase I trials is possible.

Pharnext’s most advanced pleodrug is PXT3003. It has already gone through phase II trials, obtaining good results and working as a proof of concept for the Pleotherapy platform.

PXT3003 is now in phase III trials (PLEO-CMT), which expects to enroll 300 patients with Charcot-Marie-Tooth Disease Type 1A (CMT1A). Enrolment will progress during 2016, with the first US site now being opened in Saint-Louis University (Missouri).

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