French biopharmaceutical company Pharnext has begun the Phase III extension trial (PLEO-CMT-FU) of PXT3003 for the treatment of patients with mild-to-moderate charcot-marie-tooth disease type 1A (CMT1A).
Developed using the firm's research and development platform PLEOTHERAPY, PXT3003 is an oral fixed-low dose synergistic combination of (RS)-baclofen, naltrexone hydrochloride and D-sorbitol.
The multi-centre, double blind, nine-month follow-up extension Phase III trial will evaluate the long-term safety and tolerability of PXT3003.
The PLEO-CMT-FU trial is part of the PXT3003 Phase III clinical programme, which includes another Phase III trial PLEO-CMT.
Pharnext chief medical officer René Goedkoop said: “The initiation of this second international Phase III trial marks an important milestone for the whole PXT3003 clinical development programme as it aims to confirm the long-term safety and tolerability profile of PXT3003."
The multi-centre, randomised, double blind, 15-month, placebo-controlled, adaptive design Phase III PLEO-CMT trial was initiated in December 2015 to assess PXT3003 in 323 patients enrolled at 30 sites across Europe, the US and Canada.
The PLEO-CMT-FU trial will enrol patients who have completed the ongoing PLEO-CMT trial.
The top-line results from the PLEO-CMT trial are expected in the second quarter of next year, while those from the PLEO-CMT-FU trial are expected in the second quarter of 2019.
PXT3003 has secured orphan drug designation for the treatment of CMT1A in adults in Europe and the US in 2014.
Pharnext plans to apply for the marketing authorisation of the drug in these countries in the first quarter of 2019.