September 3rd, 2020

Pharnext and the Charcot-Marie-Tooth Association Enter Collaboration

Pharnext and the Charcot-Marie-Tooth Association Enter Collaboration

Collaboration focused on identifying and characterizing key biomarkers in Charcot-Marie-Tooth disease type 1A


Pharnext, an advanced clinical-stage biopharmaceutical company pioneering new approaches to develop innovative drug combinations based on big genomics data and artificial intelligence, today announced a research collaboration with the United States patient advocacy group, the Charcot-Marie-Tooth Association (CMTA), to investigate novel biomarkers associated with the Charcot-Marie-Tooth disease type 1A (CMT1A), the most common subtype of the disease.


The primary objective of this collaboration is to identify and validate potential treatment responsive CMT1A biomarkers that could be further explored in future clinical studies, in particular the upcoming Phase III study of PXT3003, Pharnext's lead drug candidate. Pharnext intends to investigate blood samples collected from mild to moderate CMT1A patients enrolled in the first Phase III study of PXT3003. Notably, this collaboration will evaluate the potential of TMPRSS5, a recently identified Schwann cell-specific biomarker in CMT1A patients, to confirm if it can be used to assess treatment response in future clinical trials. As TMPRSS5 is actually part of a broader neurology panel that tests many additional potential biomarkers, the collaboration between Pharnext and the CMTA could lead to the identification of additional biomarkers in CMT1A that are not described yet.