Pharnext Opens First U.S. Study Site For Its PLEO-CMT International Pivotal Phase 3 Trial In Charcot-Marie-Tooth Disease Type 1A

French pharmaceutical company Pharnext announced the opening of the first U.S. trial site for its PLEO-CMT pivotal Phase 3 clinical trial of its lead pleodrug PXT-3003 in Charcot-Marie-Tooth Disease Type 1A (CMT1A).

Pharnext is an advanced clinical stage biopharmaceutical company developing novel therapeutics for severe orphan and common neurological disorders. One of the company’s leading therapeutics is PXT3003 for the treatment of CMT1A. PXT-3003 is an oral fixed-low dose combination of (RS)-baclofen, naltrexone hydrochloride, and D-sorbitol.

CMT1A is the most common type of CMT, affecting at least 125,000 people across Europe and the U.S. Duplication of the PMP 22 gene is responsible for creating the symptoms associated with CMT1A, such as progressive muscle atrophy of legs and arms causing walking, running, balance problems, and abnormal hand functioning.

At least 5% of CMT1A patients end up in wheelchairs, and may also experience mild to moderate sensitive disorders. Signs of CMT1A usually appear during adolescence and progressively evolve throughout the patient’s life.  

Currently, there is no cure or treatments approved for use. Managing the disease is limited to orthotics, leg braces, physical, and occupational therapy or surgery.

But we are embarking on a major milestone in finding a cure by opening the first U.S trial site for the PLEO-CMT pivotal Phase 3 clinical trial of PXT-3003 for CMT1A.

Daniel Cohen, M.D., Ph.D., Chairman, Chief Executive Officer, and Co-Founder of Pharnext, states:

“Opening the first U.S. trial site is an important milestone in our PLEO-CMT Phase 3 trial program after opening the first sites in France, Germany and Belgium since December 2015. Our hope is that this international Phase 3 study will support the promising results we saw in previous studies with PXT3003 and bring long-needed relief to patients suffering from CMT1A.”

The Hereditary Neuropathy Foundation supports Pharnext and this clinical trial, as we get closer to finding a cure.

Allison Moore, Founder and Chief Executive Officer of HNF states:

“There is currently no approved treatment for patients suffering with CMT. We are strong supporters of Pharnext’s initiative to bring help to these patients in the form of an effective therapy. We are happy to be able to provide information about this clinical trial on our website and provide patients with the information they need to find trial sites. We have been supported by Pharnext since 2014 to improve awareness of CMT and other rare neuropathies notably with such resources as the Global Registry for Inherited Neuropathies (GRIN) or the CMT Connect program recently launched in the U.S.”

The clinical trial began enrolling patients in December 2015 in Europe, aimed at determining whether PXT-3003 is effective and well-tolerated in CMT1A patients. The study expects to enroll a total of 300 patients across 28 centers in the United States (California, Connecticut, Florida, Massachusetts, Minnesota, Missouri, New York and Washington) and in Europe (France, Germany, UK, Spain, the Netherlands and Belgium).

These centers will be progressively opened and activated in 2016. The first trial site in the U.S. has just been activated at Saint-Louis University in Missouri.

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