Published: December 11, 2023

French biopharmaceutical company Pharnext SA announced Dec. 10 that the initial results from its Phase III clinical trial (PREMIER trial) for CMT1A did not meet expectations.

The company said in a news release that analyzing the results was challenging because of the slow progression of CMT1A and the subjective nature of some clinical assessments. Those assessments can be influenced by placebo and training effects, making it difficult to determine the impact of treatment. In particular, the company said, the Overall Neuropathy Limitation Scale (ONLS), which measures functional motor disability, did not confirm findings from its previous clinical trial.

Patients with mild-to-moderate CMT1A experienced improvement on both treatment and placebo, complicating the interpretation of the results based on this endpoint. The company reported that the trial reaffirmed the treatment’s safety profile, established in previous studies.

In its statement, the company said that it will continue to analyze the data in collaboration with potential partners for licensing or acquisition of PXT-3003.

The CMTA would particularly like to thank the members of the CMT community for taking part in the trials, and for their dedication and commitment to achieving our goal of a world without CMT. While the outcome is disappointing, the trial did establish a new ecosystem in CMT, and lays a path for other treatments to follow. The CMTA will continue to work across our community of patients, physicians, and drug developers to enable outcomes and measures that could be tested in future studies. The support from the community is absolutely essential to helping us all understand how to move forward to find treatments and, ultimately a cure, for CMT.

UPDATE: December 19, 2023

Last week, Pharnext shared the disappointing news that initial results from the Phase III trial for PXT-3003 showed no statistical significance between the placebo and the drug for their primary outcome measure (ONLS). Today, following analysis of secondary outcome measure (CMT-NSv2) and additional data, Pharnext announced their intention to seek FDA and EMA approval for PXT-3003. Data analysis on a study of this type and size is complex and will continue as the company prepare their data package for review by approval authorities in the US and Europe.