Pharnext advances analysis of data from the pivotal Phase III study of PXT3003 in Charcot-Marie-Tooth disease type 1A

Photo credit © ChaunuPictures

( — Pharnext SCA, a late-stage clinical biopharmaceutical company developing novel therapies for neurodegenerative diseases with no satisfactory therapeutic solution, provides an update on the ongoing analysis of data from the pivotal Phase III trial (FIRST trial) of PXT3003 , its drug candidate in Charcot Marie-Tooth disease type 1A (CMT1A).

As a reminder, on December 11, 20231, Pharnext announced that on the primary efficacy endpoint of the PREMIER trial, the ONLS (Overall Neuropathy Limitation Scale) which measures functional motor disability, patients with mild to moderate CMT1A showed signs of improvement whether on treatment or placebo, rather than the slow decline typical of the natural progression of CMT1A.
This unexpected improvement in the placebo group makes it difficult to interpret the results based on this criterion. However, other data from the trial suggested that there was no deterioration in the condition of patients under treatment, which is a positive sign in the context of a degenerative disease like CMT1A.

Additionally, the trial reaffirmed the high safety profile of the treatment already established in all previous studies. This safety profile is crucial for the treatment of a chronic disease like CMT1A.

On December 19, 20232, Pharnext announced that analyzes of pre-specified subgroups of the PREMIER trial suggested an efficacy signal, including a better response in treated patients with a BMI less than 25 or in elderly patients. under 45 years old.

Furthermore, the analysis of the data revealed that although the study as a whole had been randomized into two arms (PXT3003 and placebo), the distribution of the two treatments within each clinical investigation center was not. not been done in a balanced manner. It appears that in some centers, patients only received placebo and in others, patients only received the treatment. However, a ‘post-hoc’ analysis showed that by removing the data from centers which had less than two patients per arm, i.e. 50 patients (13%), we obtained a statistically significant difference between the treated group and the group under treatment. placebo on the CMTNS score.


Source link -87