Pharnext klaar met werving voor internationaal onderzoek naar PXT3003 tegen
PARIJS–(BUSINESS WIRE)– Pharnext SA (Paris:ALPHA) (FR00111911287 – ALPHA), een Frans biofarmaceutisch bedrijf dat een geavanceerd portfolio op vlak van neurodegeneratieve ziekten ontwikkelt, heeft de werving van patiënten voor zijn belangrijke fase 3-onderzoek PLEO-CMT voltooid. Dit onderzoek richt zich op Pharnexts PLEODRUG PXT3003 voor de behandeling van de ziekte van Charcot-Marie-Tooth type 1A (CMT1A).
PLEO-CMT is een cruciaal, in meerdere centra uitgevoerd, dubbelblind en gerandomiseerd onderzoek met een placebo-groep ter controle. Het project begon in december 2015 en heeft inmiddels 323 patiënten met milde tot matige CMT1A geworven op dertig locaties in Europa, de Verenigde Staten en Canada. Patiënten zijn willekeurig onderverdeeld in een groep die een placebo krijgt en twee groepen die vijftien maanden lang een één of twee doses krijgen. Het primaire eindpunt van het onderzoek is een verandering in de zogeheten Overall Neuropathy Limitation Scale (ONLS), zodat kan worden vastgesteld of er een verbetering plaatsvindt na een behandeling met PXT3003 van twaalf tot vijftien maanden. De resultaten van PLEO-CMT verschijnen naar verwachting in de tweede helft van 2018.
Pharnext Completes Patient Enrollment in International Pivotal Phase 3 Trial of PXT3003 for Charcot-Marie-Tooth Disease Type 1A |
|||||
|
PARIS–(BUSINESS WIRE)– Pharnext SA (Paris:ALPHA) (FR00111911287 – ALPHA), a French biopharmaceutical company developing an advanced portfolio of products in the field of neurodegenerative diseases, today announced that it has completed patient enrollment for its international pivotal Phase 3 PLEO-CMT trial of PXT3003, Pharnext’s lead PLEODRUG ©, for the treatment of Charcot-Marie-Tooth Disease Type 1A (CMT1A). PLEO-CMT is a pivotal, multi-center, randomized, double blind, placebo-controlled, adaptive design Phase 3 study that was initiated in December 2015 and has enrolled 323 patients with mild to moderate CMT1A in 30 sites across Europe, the U.S. and Canada. Patients have been randomized to receive either placebo or one of two doses of PXT3003 during 15 months. The primary endpoint of this clinical trial is the change in the Overall Neuropathy Limitation Scale (ONLS) in order to determine improvement of patients’ disability after 12 and 15 months of treatment with PXT3003. Results of the PLEO-CMT trial are expected in the second half of 2018. Patients will be invited to continue treatment with PXT3003 in a 9-month extension study. 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 previous exploratory Phase 2 trial of PXT3003 demonstrated safety, tolerability and improvements beyond stabilization of CMT1A patient disability as published in the Orphanet Journal of Rare Diseases ( http://www.ojrd.com/content/9/1/199). Quotes: “Completing enrollment of our pivotal Phase 3 trial of PXT3003 is a significant milestone and highlights the strength of our clinical operations and management teams, as well as the support of our numerous clinical trial sites, our dedicated clinical investigators and the patient community,” said René Goedkoop, M.D., Chief Medical Officer of Pharnext. Daniel Cohen, M.D., Ph.D., Co-Founder and Chief Executive Officer of Pharnext added: “This clinical trial is highly significant for patients suffering from CMT1A where only supportive care is available today. Our PLEODRUG © represents a great hope for patients suffering from this debilitating disease.” Michael W. Sereda, M.D., Professor of Neurology at the Max Planck Institute, Göttingen said: “PXT3003 has shown much promise in previous preclinical and clinical studies, and the data Pharnext has published to date positions PXT3003 as a prominent and safe therapeutic candidate for patients with CMT1A. This Phase 3 trial could bring us closer to helping our patients find an efficacious treatment capable of altering the progressive course of the disease.” Allison Moore, Founder and Chief Executive Officer of the Hereditary Neuropathy Foundation (HNF) added: “We have been pleased to have the opportunity to contribute to this groundbreaking research by providing U.S. clinical site identification and patient recruitment support for this pivotal Phase 3 trial of PXT3003. Our HNF resources have been instrumental such as the Global Registry for Inherited Neuropathies (GRIN), the online CMT Inspire Community or the recently launched CMT Connect program. We are enthusiastic that our joint effort with Pharnext could result in providing people suffering from CMT1A with much-needed new therapies and look forward to our joint work for years to come.” About CMT1A 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 PLEO-CMT Trial For more information about the PLEO-CMT clinical trial, please visit the following website: About Pharnext The company Pharnext is listed on Euronext Alternext Stock Exchange in Paris (ISIN code: FR00111911287). For more information, visit www.pharnext.com PLEODRUG © and PLEOTHERAPY © are registered trademarks by Pharnext View source version on businesswire.com: http://www.businesswire.com/news/home/20161219005865/en/ Contacts Pharnext |
|||||