ICYMI: First European Patient Enrolled in PXT3003 Trial for CMT1A

In case you missed it – a news release from biopharmaceutical company Pharnext SA (“Pharnext”) shared that on July 12, 2021, the first European patient enrolled in the Phase 3 PREMIER clinical trial. Altogether, the trial is designed to evaluate PXT3003 for patients with Charcot-Marie-Tooth disease type 1A (CMT1A). Although the trial has been ongoing within the United States, with the first patient being dosed in March 2021, the enrollment of the first European patient highlights the clinical program’s ongoing development. As there are no approved treatment options for patients with CMT1A, PXT3003 also has the potential to fill a huge unmet need.


According to Pharnext, PXT3003 is an orally administered solution made up of sorbitol, naltrexone, and baclofen. The treatment, administered twice daily, helps down-regulate PMP22 overexpression. Through this, Pharnext believes that PXT3003 could improve neuronal signaling, even in malfunctioning peripheral nerves, as well as protect muscle cells, the axon, and the neuromuscular junction. Thus far, PXT3003 has shown benefits for patients with CMT1A in prior Phase 2 and 3 clinical trials. Additionally, the treatment received Orphan Drug designation in both the European Union and the U.S.

Now, the first European patient has enrolled in the Phase 3 PREMIER clinical trial. The patient enrolled at the University Hospital La Timone. Altogether, 350 patients across North America, Europe, and Israel will enroll. During the trial, researchers seek to determine the safety, efficacy, and tolerability of PXT3003 in patients with mild-to-moderate CMT1A. Patients will be evaluated over a 15-month study period. Throughout the trial, researchers will use options such as the Overall Neuropathy Limitations Scale and Charcot-Marie-Tooth Neuropathy Score (among others) to determine improvements. Study results should be available in the latter part of 2023.

Charcot-Marie-Tooth Disease (CMT)

As explained above, the treatment is designed for patients with Charcot-Marie-Tooth disease type 1A. But what is Charcot-Marie-Tooth disease (CMT), or hereditary motor and sensory neuropathy? Although rare, CMT is considered one of the most commonly inherited neurological disorders. A variety of gene mutations are associated with CMT. In fact, the CMT subtype (such as 1A) differs based on the specific gene abnormalities. However, regardless of the form, these mutations cause peripheral muscles to degenerate. Over time, this prevents the muscles and nerves from communicating, causing issues. CMT1A is considered the most common form of Charcot-Marie-Tooth disease. Learn more about CMT1A.

Symptoms associated with CMT include:

  • Decreased reflexes
  • Foot deformities, such as high arches or hammertoes
  • Muscle weakness in the lower extremities and distal muscles
  • Foot drop
  • Frequent tripping
  • Muscle atrophy and weakness in the hands
  • Difficulty walking or balancing
  • Increased calf muscles
  • Feeling of “pins and needles”
Jessica Lynn

Jessica Lynn

Jessica Lynn has an educational background in writing and marketing. She firmly believes in the power of writing in amplifying voices, and looks forward to doing so for the rare disease community.

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