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news.Pharnext has formed a collaboration with OrphanDev, a dedicated platform offering support in regulatory, methodology and logistics for rare diseases.
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OrphanDev’s main mission is to accelerate the development of orphan drugs. This collaboration is related to the International pivotal Phase 3 trial of Pharnext’s investigational Pleodrug, PXT-3003, for the treatment of CMT 1A.
PLEO-CMT aims at recruiting 300 patients in 27 centers both in Europe and the US. For the European part of the trial (18 centers spread over Germany, Belgium, Spain, France, the UK and the Netherlands), OrphanDev will play a critical role in the dissemination and availability of practical and logistical information related to the trial.
Pharnext and OrphanDev had already collaborated on 3 studies related to CMT 1A: PXT-3003 Phase2 study, a meta-analysis and a prospective study (biomarkers).
About PXT-3003
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.
About PLEO-CMT
PLEO-CMT is an International pivotal, multi-center, randomized, double blind, placebo-controlled, three-arm Phase 3 study which will enroll patients
aged 16 to 65 with mild to moderate CMT1A. Over 15 months, Pharnext will compare in parallel groups the efficacy and safety of two orally administered dosage variations of PXT-3003 versus 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 and agreed, will receive a dose of the active product (patients who received placebo will be treated randomly with PXT-3003 dose 1 or 2).
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.