Rare Disease Report

Pharnext's Charcot-Marie-Tooth Extension Study Begins. NDA Filing in 2019?

MARCH 16, 2017
James Radke
Pharnext announced it has begun the extension study to its Phase 3 pivotal trial, PLEO-CMT, that is testing the use of PXT3003 in patients with mild-to-moderate Charcot-Marie-Tooth disease Type 1A (CMT1A).
PXT3003 is an oral fixed-low dose synergistic combination of (RS)-baclofen, naltrexone hydrochloride and D-sorbitol, developed by Pharnext.
Data from PLEO-CMT is expected to be available the end of 2018 and data from PLEO-CMT-FU in 2019.  

Data from the 2 trials, assuming they are positive, are expected to be used in the drug's regulatory applications in the U.S. and Europe in 2019.

Pivotal Study

PLEO-CMT is a pivotal, multi-center, randomized, double blind, fifteen-month, placebo-controlled, adaptive design Phase 3 study that was initiated in December 2015. Patient enrollment was completed in December 2016 with 323 patients in 30 sites across Europe, the United States and Canada. The primary endpoint is change in the Overall Neuropathy Limitation Scale (ONLS) score at 12 and 15 months.

Extension Study

PLEO-CMT-FU is a 9-month extension study designed to assess the long-term safety and tolerability of PXT3003. Patients in the PLEO-CMT trial are eligible to continue taking the drug or to switch from placebo to the drug. The primary endpoints are safety and tolerability of PTXT3003.

About Charcot-Marie-Tooth Disease

Charcot-Marie-Tooth (CMT) disease is an inherited disorder affecting the peripheral motor and sensory neurons. Although it is an inherited condition, the main signs and symptoms such as decreased muscle size and weakness may not present until adolescence or early adulthood. Many of the early signs of the disease, however, can be spotted in children.
Symptoms vary with each person but common early symptoms may include clumsiness, slight walking difficulties, weak leg muscles, fatigue.
Other common symptoms of CMT as it progresses include:
  • foot deformity (very high arched foot/feet)
  • difficulty lifting foot at the ankle (foot drop)
  • curled toes (known as hammer toes)
  • loss of lower leg muscle, which leads to skinny calves
  • numbness or burning sensation in the feet or hands
  • “slapping” when walking (feet hit the floor hard when walking)
  • weakness of the hips, legs, or feet
  • leg and hand cramps
  • loss of balance, tripping, and falling
  • difficulty grasping and holding objects and opening jars and bottles
At the first annual Patient-Centered Charcot-Marie-Tooth Summit on October 6th, 2016, we spoke with René Goedkoop, Chief Medical Officer of Pharnext about PXT3003 and the Phase 3 study.

In the clip below, Holly Waters, a CMT patient talks about the disease and its impact on her life.   


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