DSMB gives Pharnext green light to continue Charcot-Marie-Tooth drug study



Image: Pharnext

French biopharmaceutical company Pharnext has said today that Data Safety Monitoring Board (DSMB) has recommended continuing of the PLEO-CMT study of PXT3003, after reviewing safety data from 100 Charcot-Marie-Tooth patients who completed at least three months of study treatment.


PLEO-CMT is an international pivotal Phase 3 study that was initiated in December 2015 and is planned to enroll 300 patients with mild to moderate CMT1A from Europe and the U.S. by the end of December 2016, Pharnext has said. According to the plan, patients will be randomized in three arms – placebo and two PXT3003 doses – and will receive study treatment over 15 months. PXT3003, developed using Pharnext’s R&D platform PLEOTHERAPY, is a novel oral fixed-low dose combination of (RS)-baclofen, naltrexone hydrochloride and D-sorbitol, the French drugmaker has explained.

The DSMB is an independent body of experts drawn from the fields of clinical medicine, biostatistics and study methodology, chartered to provide recommendations to Pharnext upon regular pre-specified review of the accumulated data during the conduct of the clinical trial.

“We believe this clinical trial has the potential to be a crucial turning point in the effort to finally provide an efficacious treatment for patients suffering from CMT1A,” said Daniel Cohen, M.D., Ph.D., Co-Founder and Chief Executive Officer of Pharnext. “Today’s therapeutic options are very limited and mostly palliative in nature. Our PLEODRUG PXT3003 has already demonstrated safety, tolerability and improvements in CMT1A patient disability in a Phase 2 trial. Given this positive safety assessment by the DSMB, we are hopeful to bring this much-needed potential therapy to patients suffering from this debilitating condition upon completion of this Phase 3 trial.”

CMT disease encompasses a heterogeneous group of inherited, progressive, chronic peripheral neuropathies. CMT type 1A (CMT1A), the most common type of CMT, is an orphan disease affecting at least 125,000 people in Europe and the U.S.

Leave a Reply

Please log in using one of these methods to post your comment:

WordPress.com Logo

You are commenting using your WordPress.com account. Log Out /  Change )

Google+ photo

You are commenting using your Google+ account. Log Out /  Change )

Twitter picture

You are commenting using your Twitter account. Log Out /  Change )

Facebook photo

You are commenting using your Facebook account. Log Out /  Change )


Connecting to %s