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

DTCP Closes USD 350 Million Venture and Growth Fund Including Capital From Institutional and Corporate Investors25.6.2019 09:10:00 CESTPressemelding

DTCP announced today that it has secured additional capital commitments from two corporate sponsors for its second Venture and Growth Fund: SK Telecom, the largest mobile operator in Korea and frontrunner in the fields of mobile network, AI, IoT, media and platform services, and ZEISS, an internationally leading technology enterprise operating in the fields of optics and optoelectronics, headquartered in Germany. The new investors join Deutsche Telekom, HarbourVest Partners, Neuberger Berman private equity fund sand others as Limited Partners (LPs) in the fund which boasts total commitments of USD 350 million and is now closed for further investors. The fund looks to deploy tickets between USD 5 and 50 million into Enterprise SaaS companies that: (i) operate in the sectors Cloud and Network Infrastructure, 5G/IoT, Digital Enterprise, Cybersecurity, Artificial Intelligence, and Marketing Technology; (ii) typically feature annualized recurring revenues of USD 5 million or more and (iii)

ClickFellas Chooses Digital Element to Increase Accuracy of IP Geotargeting Services25.6.2019 08:02:00 CESTPressemelding

Digital Element, the global geolocation data and services provider, today announced that ClickFellas, a new advertising technology company, has chosen its NetAcuity EdgeTM technology to improve ad targeting for its clients. By incorporating Digital Element’s technology, ClickFellas is able to identify the consumer’s location, internet service provider, connection speed, mobile carrier, organisation name and proxy type without the user becoming personally identifiable. This highly accurate and granular IP data is used to facilitate standalone geotargeting and, when combined with additional factors such as gender, purchase intent and history data, allows Clickfellas to create tighter targeting profiles. Additionally, proxy data is used to help combat click fraud. “Digital Element’s technology helps us not only improve the accuracy of geotargeted advertising being served but also, ensures that we limit fraudulent traffic,” commented Franz Kanehl, Co-Founder ClickFellas Ltd. “For us, the b

iD Mobile Signs New MDS Global Contract to Take Advantage of Cloud-Based BSS Solution25.6.2019 08:00:00 CESTPressemelding

MDS Global Ltd, a leading BSS-as-a-Service provider today announced that iD Mobile, one of the UK's largest mobile virtual network operators, has engaged in a new contract with the company, adopting MDS Global's CMP 8, a fully cloud-enabled Monetisation Platform designed to scale on-demand for new subscriber and business growth. Seeking greater flexibility to integrate social media and other self-serve and community applications, and to take advantage of the benefits of using Google's Cloud infrastructure, iD Mobile's move to MDS Global's CMP 8, the BSS-as-a-service platform that underpins the company's VNOnDemand solution, offers on-demand scalability for growth. Fully enabled for any public or private environment, iD Mobile can deploy new features and services within hours, keeping with the demands of today's feature-hungry consumers. The UK-based MVNO, offers customers an online and app-based portal giving them the freedom to tailor their iD Mobile package based on consumption requi

Tech Veteran Tej Kohli Calls for a Democratisation of AI25.6.2019 08:00:00 CESTPressemelding

Tej Kohli, the London-based tech investor and entrepreneur, says that the hype around AI is still premature and that too much hype is creating a “bubble of enthusiasm” that risks causing disillusionment amongst the public when AI products do not start quickly improving their lives as promised. He says that AI will only be able to change the world when there is an accessible user interface which enables more entrepreneurs to build AI products that can enable all people to access the full benefits of the AI economy. This will require concerted international collaboration as well as major capital investment. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20190625005075/en/ Tej Kohli is a prominent investor in the global artificial intelligence economy (Photo: Business Wire) Kohli is ebullient about the prospects of AI to build a better world and has reiterated his belief that an exponential global AI economy could be worth $150 t

Banque du Caire, One of the Largest State-owned Banks in Egypt, Goes Live With Temenos to Redefine Digital Customer Experience25.6.2019 08:00:00 CESTPressemelding

Temenos (SIX: TEMN), the banking software company, today announces that Banque du Caire has gone live with Temenos Infinity, the omni-channel digital banking product to transform the bank’s multiple banking channels into a fast, seamless and compelling customer journey. At this first stage of the Temenos Infinity implementation, the bank has gone live with digital channels servicing corporate and retail customers. The second phase of the implementation will deliver enhanced customer engagement with the Temenos Infinity real time marketing, digital engagement and digital analytics modules. Temenos Infinity will allow Banque du Caire, which currently operates a network of 225 branches, to increase product agility and take new products and services quicker to market. Temenos Infinity was implemented through APIs as an independent front office platform on the bank’s existing third party core banking system. The bank is also undergoing a core banking system overhaul after choosing the cloud

Eneco Holdings, Inc. to Spark Fuel Revolution with Low-Price, High-Concentration, Scalable Hydrogen-generation Technology25.6.2019 07:30:00 CESTPressemelding

Eneco Holdings, Inc. is committed to the research and development of eco-friendly fuel technologies that reduce emissions of greenhouse gases. In 2015, we developed an emulsion fuel that achieves a complete fusion of 50% oil and 50% water. When used to drive buses on public roads, this fuel cuts carbon dioxide emissions roughly in half—the first achievement of its kind in Japan. This highly acclaimed technology is already installed and operating in Indonesia, Bangladesh, and the Philippines, and is set for introduction in Singapore, Australia, China, Laos, and Myanmar. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20190625005393/en/ The Press Conference (Photo: Business Wire) Given the urgency of reducing greenhouse gas emissions worldwide, Eneco Holdings, Inc. has turned its attention to hydrogen gas in a bid to drive the transition to a zero-carbon economy. Although hydrogen fuel has already captured worldwide attention as