The company has completed patient enrollment in the P-III (PREMIER) trial to evaluate PXT3003 (BID) vs PBO in 387 patients with CMT1A for 15mos. at 52 centers across the US, Canada, EU & Israel. The results are expected in Q4’23
PXT3003 dose corresponds to the high dose used in the prior P-III (PLEO-CMT) & ongoing P-III (PLEOCMT-FU) OLE trial. The 1EPs will be the ONLS that measures functional motor disability & 2EPs include 10mWT, QMT, PGI-S, PGI-C & CMTNS-v2. The safety & tolerability will also be assessed
PXT3003 is a fixed-dose synergistic combination of baclofen, naltrexone & sorbitol formulated as an oral solution. In P-II (PLEO-CMT) & P-III (PLEO-CMT-FU) trials, PXT3003 showed an encouraging & consistent results
Ref: Pharnext | Image: Pharnext
