Pharnext Initiates PLEO-CMT, an International Pivotal Phase 3 Trial, in Charcot-Marie-Tooth disease type 1A

The PLEO-CMT study is designed to assess the efficacy and safety of PXT-3003 in patients suffering from Charcot-Marie-Tooth disease type 1A (CMT1A)

Primary endpoint is improvement of patients' disability measured by the change in Overall Neuropathy Limitation Scale (ONLS)

PARIS, Dec. 15, 2015 /PRNewswire/ -- Pharnext SAS [] today announced the initiation of the PLEO-CMT study, an International pivotal Phase 3 trial of its investigational Pleodrug, PXT-3003, for the treatment of CMT1A. The purpose of this study is to determine whether PXT-3003 is effective and well tolerated in patients with CMT1A. PXT-3003 is a novel oral fixed-low dose combination of (RS)-baclofen, naltrexone hydrochloride and D-sorbitol which has been developed via Pharnext Pleotherapy R&D platform, with the aim to lower toxic PMP22 gene over-expression in CMT1A.

PLEO-CMT is a pivotal, multi-center, randomized, double blind, placebo-controlled, three-arm Phase 3 study which will enroll patients aged 16 and older with mild to moderate CMT1A. Diagnosis of CMT1A will be confirmed genetically through detection of PMP22 gene duplication. Over 15 months, Pharnext will compare in parallel groups the efficacy and safety of two orally administered dosage variations of PXT-3003 to placebo. Efficacy will be assessed through one primary endpoint: change in the ONLS score at 12 and 15 months of treatment to measure improvement of patients' disability with PXT-3003. Additional secondary outcome measures will be assessed including functional and electrophysiological endpoints. A nine month follow-up study is planned thereafter, where all patients who will have completed the first 15 months, will receive the active PXT-3003 dose.

The study expects to enroll 300 patients across 27 centers in Europe (France, Germany, UK, Spain, the Netherlands and Belgium) and in the United States (California, Connecticut, Florida, Massachusetts, Minnesota, Missouri, New York and Washington). These centers will be progressively opened and activated in 2016. The first patient has been screened at the Timone University Hospital in Marseille, France.

The exploratory Phase 2 trial of PXT-3003 demonstrated safety, tolerability and improvements beyond stabilization of CMT1A patient disability as published in the Orphanet Journal of Rare Diseases ( []).


Daniel Cohen, M.D., Ph.D., chairman, chief executive officer and co-founder of Pharnext, said, "This clinical trial is crucial for patients suffering from CMT1A due to the limited therapeutic options available today. Our pleodrug, PXT-3003, has shown much promise in previous multiple studies and represents a great hope as a potential therapy for patients with CMT1A, where only supportive care is currently available."

Richard Lewis, M.D., Ph.D., director of the electromyography and co-director of the neuromuscular clinic, Cedars-Sinai Specialty Clinic, Los Angeles, USA, said, "The preclinical and Phase 2 clinical data Pharnext has published to date make PXT-3003 a worthwhile therapeutic candidate with minimal safety risk for patients with CMT1A. Of course, patients are hopeful for a treatment capable of altering the progressive course of the disease. This International PLEO-CMT Phase 3 trial could bring us closer to helping these patients find such a treatment."

About CMT 1A

Charcot-Marie-Tooth (CMT) disease encompasses a heterogeneous group of inherited, progressive, chronic peripheral neuropathies. CMT type 1A (CMT 1A), the most common type of CMT, is an orphan disease affecting at least 125,000 people in Europe and the U.S. The genetic mutation responsible for CMT 1A is a duplication of the PMP 22 gene coding for a peripheral myelin protein. Overexpression of this gene causes degradation of the neuronal sheath (myelin) responsible for nerve dysfunction, followed by loss of nerve conduction. As a result of peripheral nerve degradation, patients suffer from progressive muscle atrophy of legs and arms causing walking, running, balance problems and abnormal hand functioning. CMT 1A patients end up in wheelchairs in at least 5% of cases. They might also suffer from mild to moderate sensitive disorders. First symptoms usually appear during adolescence and will progressively evolve through patients' life.

To date, no curative or symptomatic medications have been approved and treatment consists of supportive care such as orthotics, leg braces, physical and occupational therapy or surgery.

About Pharnext

Pharnext is an advanced clinical stage biopharmaceutical company developing novel therapeutics that simultaneously target multiple key disease pathways for severe orphan and common neurological disorders. The proprietary research and development platform of Pharnext, based on network pharmacology, is applicable to a broad spectrum of diseases and allows the rapid development of "pleodrugs", synergistic combinations of repositioned drugs with established safety profiles. The company's two lead pleodrugs are PXT-3003 for the treatment of orphan disease Charcot Marie Tooth type 1A (Phase 3 clinical trial initiated) and PXT-864 for Alzheimer's disease (Phase 2 clinical trial ongoing) and other neurologic indications (including Parkinson's disease and amyotrophic lateral sclerosis).

For further information, visit []

Pharnext ContactDaniel Cohen, M.D., Ph.D. Chairman and Chief Executive Officer []

Media Contacts Tony Russo, Ph.D. Matt Middleman, M.D. Russo Partners T: +1-212-845-4251 T: +1-212-845-4272 [] []


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