Business Wire

Pharnext to Announce Top-Line Results from the Pivotal Phase 3 Trial of PXT3003 for the Treatment of Charcot-Marie-Tooth Type 1A Disease by October 2018

Del

Regulatory News:

Pharnext SA (Paris:ALPHA) (FR0011191287 - ALPHA), a biopharmaceutical company pioneering a new approach to the development of innovative drug combinations based on big data genomics and artificial intelligence, today announced an update from its ongoing Phase 3 clinical program (PLEO-CMT and PLEO-CMT-FU studies) evaluating PXT3003 for the treatment of Charcot-Marie-Tooth type 1A disease (CMT1A) in adults. Top-line results from the pivotal PLEO-CMT study are now expected by October 2018.

Prof. Daniel Cohen, M.D., Ph.D., Pharnext’s Co-Founder and Chief Executive Officer said: “We are thrilled to bring this Phase 3 clinical trial to completion and we now expect to disclose top-line results by October of this year. Our PLEODRUG™ PXT3003 has already shown initial signals of efficacy in our Phase 2 trial in CMT1A. We are hopeful we can bring this much-needed therapy to patients suffering from this debilitating condition, as they currently have limited therapeutic options, most of which are palliative in nature.”

Pharnext’s first-in-class PLEODRUG™ PXT3003, developed using Pharnext’s R&D platform, PLEOTHERAPY™, is a novel oral fixed-low dose combination of baclofen, naltrexone and sorbitol, with orphan drug designation both in Europe and the United States. PXT3003 has already demonstrated safety and tolerability, and the highest dose showed consistent evidence of improvement beyond disease stabilization in CMT1A in the Phase 2 trial where efficacy was assessed using both the Charcot-Marie-Tooth Neuropathy Score (CMTNS) and the Overall Neuropathy Limitations Scale (ONLS) as main endpoints. The results from this Phase 2 trial were published in the Orphanet Journal of Rare Diseases.

PXT3003 is currently being evaluated in a pivotal, multi-center, randomized, 15-month, double-blind, placebo-controlled Phase 3 clinical study (“PLEO-CMT”). The trial was initiated in December 2015 and has enrolled patients aged 16 and older with mild-to-moderate CMT1A in 30 sites across Europe, the U.S. and Canada. The primary endpoint is the change in ONLS score at 12 and 15 months of treatment. Additional outcome measures are being assessed including functional and electrophysiological endpoints.

Patients who had completed the Phase 3 PLEO-CMT trial have been enrolled in a multi-center, 9-month, Phase 3 follow-up extension study (“PLEO-CMT-FU”) initiated in March 2016. The study was designed to assess the long-term safety and tolerability of PXT3003 .

Following the successful completion of multiple milestones, including a pre-specified evaluation by the independent Data Safety Monitoring Board in September 2017, a blind variability analysis in November 2017 and a futility analysis in November 2017, Pharnext now expects to announce top-line results from the pivotal PLEO-CMT study by October 2018.

About CMT1A

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 Pharnext

Pharnext is an advanced clinical-stage biopharmaceutical company developing novel therapeutics for orphan and common neurodegenerative diseases that currently lack curative and/or disease-modifying treatments. Pharnext 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 has developed a new drug discovery paradigm based on big genomic data and artificial intelligence: PLEOTHERAPY™. The Company identifies and develops synergic combinations of drugs called PLEODRUG™ offering several key advantages: efficacy, safety and robust intellectual property. The Company was founded by renowned scientists and entrepreneurs including Professor Daniel Cohen, a pioneer in modern genomics and is supported by a world-class scientific team.

Pharnext is listed on Euronext Growth Stock Exchange in Paris (ISIN code: FR0011191287).
For more information, visit http://www.pharnext.com/

Contact information

Pharnext
René Goedkoop, +33 (0)1 41 09 22 30
Chief Medical Officer
contact@pharnext.com
or
Financial Communication (France)
Actifin
Stéphane Ruiz, +33 (0)1 56 88 11 15
sruiz@actifin.fr
or
Investor Relations (U.S.)
Stern Investor Relations, Inc.
Matthew Shinseki, +1 212 362 1200
matthew@sternir.com
or
Investor Relations (Europe)
MC Services AG
Anne Hennecke, +49 211 529252 22
anne.hennecke@mc-services.eu
or
Media Relations (Europe)
Ulysse Communication
Bruno Arabian, +33 (0)1 81 70 96 30
barabian@ulysse-communication.com
or
Media Relations (U.S.)
RooneyPartners
Kate L. Barrette, +1 212 223 0561
kbarrette@rooneyco.com

Om Business Wire

Business Wire
Business Wire
24 Martin Lane
EC4R 0DR London

+44 20 7626 1982http://www.businesswire.co.uk

(c) 2018 Business Wire, Inc., All rights reserved.

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

Hilton Launches New Brand, Signia Hilton, Delivering Sophisticated Travel While Reimagining Meetings and Events22.2.2019 17:00:00Pressemelding

Hilton (NYSE: HLT) today announced the launch of Signia Hilton, its dynamic, new meetings-and-events-focused brand. The portfolio of hotels is setting out to transform the industry for meeting professionals and sophisticated business travelers by infusing state-of-the-art technology and design into every aspect of the guest experience. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20190222005071/en/ The brand further reinforces Hilton’s commitment to innovation that meets the evolving needs of today’s travelers and will bring premium experiences to top urban and resort destinations around the world. “In our 100th year of hospitality, we are more focused than ever on providing exceptional experiences to all of our guests – and that includes evolving those experiences to meet their changing needs,” said Christopher J. Nassetta, president and CEO, Hilton. “We are proud to launch Signia Hilton, which exemplifies our innovative sp

Axonics® Granted Expanded CE Mark Label; First and Only Sacral Neuromodulation System Approved for Use with Full-Body MRI Scans22.2.2019 16:30:00Pressemelding

Axonics Modulation Technologies, Inc. (NASDAQ: AXNX), a medical technology company focused on the development and commercialization of novel implantable Sacral Neuromodulation (“SNM”) devices for the treatment of urinary and bowel dysfunction, announced today that it has received CE mark approval for 1.5T and 3T full-body magnetic resonance imaging (“MRI”) conditional labeling for the Axonics r-SNM® System. The Axonics r-SNM System is the only implantable SNM system that has received full-body MRI conditional labeling for sale in Europe1. Raymond W. Cohen, Chief Executive Officer of Axonics, said, “Without this labeling, any patient requiring an MRI scan on any body part below the head must have their neurostimulator surgically explanted prior to the MRI scan, resulting in an additional surgery for the patient and additional costs to patients and the healthcare system. This authorization of full-body MRI scans in Europe is another important milestone for Axonics, differentiating our te

Fantastec Joins Forces with Arsenal FC Launching Official Blockchain Collectibles App22.2.2019 14:30:00Pressemelding

Fantastec announced today its first football licensing agreement with Premier League club Arsenal FC for a new blockchain authenticated collectibles app called Fantastec SWAP. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20190222005277/en/ Arsenal players appear on Fantastec SWAP in official licensing deal (Graphic: Business Wire) London-based Fantastec is a leading sports fan technology innovator, and its blockchain based ‘SWAP’ app will have broad appeal to global sports fans, gamers and sports card collectors alike. Fantastec SWAP unlocks unique and authentic club content through the app, like player autographs and exclusive footage. With its innovative blockchain technology, fans around the world can now discover, collect and swap officially licensed club collectibles with other fans with complete trust. “Fantastec SWAP is a game-changer for international football fans as well the sports collectibles industry,” commented

Volkswagen Protects Virtual Key Sharing App with Trustonic Application Protection22.2.2019 13:42:00Pressemelding

Volkswagen is working with mobile cyber security leader Trustonic to enable customers to use smartphones to access their vehicles, and to securely share their digital car keys to grant access to others via a smartphone app. Volkswagen is using the Trustonic Application Protection (TAP) platform to secure the mobile app and ensure that sensitive information and key transfer requests are securely displayed to, and approved by, a real authenticated user on a trusted device and not by hackers or malware simulating a user or device. “The smartphone is becoming the vehicle key of the future and our We Connect service is the interface for this today in the new Volkswagen Passat,” comments Alf Pollex, Head of Infotainment and Connected Car at Volkswagen AG. “The user installs the We Connect app on their smartphone which is then authorized via the infotainment system with a Transaction Number. The Mobile Key will be compatible with Android-based Samsung devices. No mobile network connection is

Mundipharma EDO GmbH: US FDA grants Orphan Drug Designation for etoposide toniribate in relapsed/refractory biliary tract cancer22.2.2019 13:30:00Pressemelding

Mundipharma EDO GmbH, part of the Mundipharma network of independent associated companies, and Imbrium Therapeutics L.P., an operating subsidiary of Purdue Pharma L.P., today announced that the US FDA has granted Orphan Drug Designation (ODD) to etoposide toniribate for the treatment of relapsed/refractory biliary tract cancer, also known as cholangiocarcinoma.3 This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20190222005126/en/ Biliary tract cancer is a rare tumour with approximately 8,000 patients diagnosed in the US every year and 10,571 in Europe.4,5 The FDA grants ODD status to medicines intended for the treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the US. Radical surgery is the only curative treatment for biliary tract cancer but, in most cases, the cancer is inoperable. Patients who fail first-line chemotherapy have limited treatment options and the standard of

Lenovo Data Center Group Delivers Broad Edge Computing Portfolio, Expands Investments in IoT22.2.2019 13:13:00Pressemelding

Next week at MWC Barcelona, Lenovo Data Center Group (DCG) will showcase continued investments in its solutions supporting IoT and edge computing as part of its IoT growth plan over the next few years. Building on the momentum of its fifth consecutive quarter of profit growth, Lenovo DCG is building a portfolio that takes infrastructure to where the data is, whether that be in the traditional data center, in the cloud or increasingly, at the edge. Today, around 10 percent of enterprise-generated data is created and processed outside a traditional centralized data center or cloud. By 2022, Gartner predicts this figure will reach 75 percent. This migration is driving increased concerns around data privacy, security and regulations coupled with challenges of latency, bandwidth and downtime. Lenovo is addressing these challenges by creating a broad portfolio of edge computing offerings that address the different ways that customers want to deploy edge computing solutions for IoT use cases.