Pharnext Completes Patient Enrollment in International Pivotal Phase 3 Trial of PXT3003 for Charcot-Marie-Tooth Disease Type 1A
20.12.2016 07:00 | 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).
“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.”
Charcot-Marie-Tooth (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. The genetic mutation responsible for CMT1A is a duplication of the PMP22 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. CMT1A 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 PLEO-CMT Trial
PLEO-CMT is a pivotal, multi-center, randomized, double blind, placebo-controlled, three-arm 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. Diagnosis of CMT1A has been 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 doses of PXT3003 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 PXT3003. 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 PXT3003 dose.
For more information about the PLEO-CMT clinical trial, please visit
the following website:
U.S. NIH ClinicalTrials.gov website at: https://clinicaltrials.gov/ct2/show/study/NCT02579759
Pharnext is an advanced clinical stage biopharmaceutical company founded by renowned scientists and entrepreneurs including Professor Daniel Cohen, a pioneer in modern genomics. Pharnext focuses on neurodegenerative diseases and has two lead products in clinical development: PXT3003 is currently in an international Phase 3 trial for the treatment of Charcot-Marie-Tooth disease type 1A and benefits from orphan drug status in Europe and the United States. PXT864 has generated positive Phase 2 results in Alzheimer’s disease. Pharnext is the pioneer of a new drug discovery paradigm: PLEOTHERAPY©. The company identifies and develops synergic combinations of repositioned drugs at low dose. These PLEODRUG© offer several key advantages: efficacy, safety, and intellectual property including several composition of matter patents already granted. The Company is supported by a world-class scientific team.
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
Chief Financial Officer
+33 (0)1 41 09 22 30
+33 (0)1 44 71 20 40
Media Relations (Europe)
+33 (0)1 44 54 36 64
Media Relations (US)
Tony Russo, Ph.D.
Matt Middleman, M.D.
Om Business Wire
Business Wire, a Berkshire Hathaway company, is the global leader in multiplatform press release distribution.
Følg saker fra Business Wire
Registrer deg med din epostadresse under for å få de nyeste sakene fra Business Wire på epost fortløpende. Du kan melde deg av når som helst.
Siste saker fra Business Wire
European Commission Grants New Indication for Soliris® (Eculizumab) for the Treatment of Patients with Refractory Generalized Myasthenia Gravis (gMG)21.8.2017 13:25 | Pressemelding
Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) announced today that the European Commission (EC) approved the extension of the indication for Soliris® (eculizumab) to include the treatment of refractory generalized myasthenia gravis (gMG) in adults who are anti-acetylcholine receptor (AChR) antibody-positive. Soliris is the first and only complement-based therapy approved in the European Union (EU) for this ultra-rare subset of patients.1-4 Patients with refractory gMG can have difficulties walking, talking, swallowing and breathing normally despite therapies currently used for MG. Exacerbations and crises of their disease may require hospitalization and intensive care and may be life-threatening.5-7 Soliris will be launched for this new indication initially in Germany, and Alexion is evaluating launches in additional EU countries. “Patients with refractory gMG have exhausted multiple the
IntegenX Applauds the Passage of the Rapid DNA Act of 201721.8.2017 12:00 | Pressemelding
IntegenX Inc., the global market leader for Rapid DNA human identification, applauds the signing into law of S.139 and HR.510, The Rapid DNA Act of 2017. The legislation will allow law enforcement agencies, under standards and guidelines established by the FBI, to perform real-time DNA testing at time of arrest, within booking stations. Similar to how fingerprint analysis has evolved from a paper and ink practice to a point-of-action technology, DNA testing has now become possible in 90 minutes within a booking station, while a suspect is still in custody. With processing times reduced from weeks to less than two hours, the potential to identify or exonerate a suspect quickly will make a meaningful impact on law enforcement. IntegenX thanks U.S. Senate sponsor Senator Orrin Hatch (R-UT) and lead co-sponsor Senator Dianne Feinstein (D-CA) as well as House sponsor Congressman
PPG Completes COLORFUL COMMUNITIES Project at Primary School in Kecskemét, Hungary21.8.2017 08:03 | Pressemelding
PPG (NYSE:PPG) today announced the completion of a COLORFUL COMMUNITIES™ project in Kecskemét, Hungary, that helped revitalize the learning environment of the Corvin Mátyás primary school. The Colorful Communities program provides PPG volunteers and products along with financial contributions to bring color and vitality to communities where the company operates around the world, such as in Kecskemét, where the PPG automotive service team operates with 33 employees. The project brought together more than 140 PPG and community volunteers who spent three days at the school revitalizing four classrooms, four changing rooms and the playground. PPG provided more than $20,000 to assist with the project, including more than 460 liters of Héra ® Prémium waterborne interior paint and Trinát ® Aqua wate
Glennmont Partners Completes Refinancing of Sleaford Biomass Plant for £150m21.8.2017 08:00 | Pressemelding
Glennmont Partners has successfully completed the refinancing of the Sleaford Renewable Energy Plant on behalf of its dedicated clean energy fund, Glennmont Clean Energy Fund Europe I, as part of a programme of realisations of value from the Fund. Glennmont Partners is one of Europe’s largest fund managers focusing exclusively on investment in clean energy infrastructure. Glennmont raises long-term capital to invest in alternative power generation projects including wind farms, biomass power stations, solar parks and small-scale hydro power plants. The carefully selected, risk managed investments deliver sustained performance and predictable returns over periods of 10 years or more. Sleaford REP is a 40MWe straw-fired biomass plant located in Lincolnshire, UK. The plant has been in operation since 2014 and is being operated by Burmeister & Wain Scandinavian Contractor (BWSC). The
Boehringer Ingelheim: Advancing anticoagulation care with new data at ESC Congress 201721.8.2017 07:08 | Pressemelding
Boehringer Ingelheim today announced that it will present important new data on the treatment of patients with atrial fibrillation (AF) at the ESC Congress in Barcelona, Spain, 26-30 August 2017. In total, four abstracts have been accepted, investigating different aspects of AF patient treatment. The results of the RE-DUAL PCI™ trial have been selected for one of the Late Breaking Science sessions of the congress. The RE-DUAL PCI™ trial compared dual therapy with triple therapy after percutaneous coronary intervention (PCI) with stent placement in approximately 2,500 adults with atrial fibrillation. Patients were either treated with dabigatran etexilate (Pradaxa®) and a single antiplatelet agent or with the vitamin K antagonist (VKA) warfarin and two antiplatelets. The dabigatran doses investigated in this trial are the same doses which have already demonstrated effective stroke preven
Study Investigates Performance of Masimo PVi® As Part of Goal-Directed Fluid Therapy During Laparoscopic Bariatric Surgery21.8.2017 06:00 | Pressemelding
Masimo (NASDAQ: MASI) announced today the findings of a recently published study in which researchers at Firat University in Turkey evaluated the performance of Masimo PVi®, a noninvasive and continuous measurement of the dynamic changes in perfusion index (Pi) that occur during respiratory cycles, as the basis of a goal-directed fluid therapy (GDFT) protocol during laparoscopic bariatric surgery on mechanically-ventilated patients.1 In the study, Dr. Demirel and colleagues sought to evaluate whether using GDFT guided by PVi on morbidly obese patients undergoing laparoscopic Roux-en-Y gastric bypass (RYGB) surgery might result in less intravenous fluid use without compromising outcomes. They enrolled 60 patients and divided them randomly into control and GDFT groups. The control group’s fluid levels were managed by standard fluid therapy, using mean arterial pressure (MAP) and
I vårt presserom finner du alle våre siste saker, kontaktpersoner, bilder, dokumenter og annen relevant informasjon om oss.Besøk vårt presserom