Business Wire

Pharnext Announces Findings Evaluating Charcot-Marie-Tooth Neuropathy Score

2.2.2017 07:00 | Business Wire

Del

Regulatory News:

Pharnext SA (Paris:ALPHA) (FR00111911287 - ALPHA), a French biopharmaceutical company developing an advanced portfolio of products in the field of neurodegenerative diseases, announced today publication in PLoS One of statistical findings evaluating the clinical scale Charcot-Marie-Tooth Neuropathy Score (CMTNS) for application in Charcot-Marie-Tooth Disease Type 1A (CMT1A).

CMTNS is the first clinical scale dedicated to quantify impairment and measure disease progression in CMT patients. It has been used as the primary endpoint in most completed clinical trials for CMT1A to date. However, its ability to measure responses to treatment has not yet been demonstrated.

In the article titled: “A Rasch Analysis of the Charcot-Marie-Tooth Neuropathy Score (CMTNS) in a Cohort of Charcot-Marie-Tooth Type 1A Patients” authors Wenjia Wang et al. report that CMTNS remains particularly useful for measuring disease severity in CMT1A. However, CMTNS appears less sensitive in assessing mild forms of the disease. Therefore, the authors recommend further refinement of the scale to better assess therapeutic efficacy in all forms of CMT1A: mild, moderate and severe.

Daniel Cohen, M.D., Ph.D., Co-Founder and Chief Executive Officer of Pharnext, said “One of the major challenges of CMT1A trial design is selecting a clinically meaningful endpoint. The findings confirm the limitations of the CMTNS. They support Pharnext’s decision, endorsed by the European and American regulatory agencies, not to use CMTNS as the primary efficacy endpoint in our pivotal PLEO-CMT trial for patients suffering from mild to moderate forms of CMT1A, and to use the Overall Neuropathy Limitations Scale (ONLS) instead. The ONLS has been used as the primary endpoint to assess treatment efficacy in other peripheral neuropathies pivotal trials.”

The publication can be found here: http://journals.plos.org/plosone/article?id=10.1371/journal.pone.0169878

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 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

About Pharnext
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

Contact information

Pharnext
Pierre Schwich
Chief Financial Officer
+33 (0)1 41 09 22 30
investors@pharnext.com
or
Investors Relations (Europe)
NewCap
Julie Coulot, +33 (0)1 44 71 20 40
pharnext@newcap.eu
or
Media Relations (Europe)
ALIZE RP
Caroline Carmagnol
Margaux Pronost
+33 (0)1 44 54 36 64
pharnext@alizerp.com
or
Investors Relations (U.S.)
Stern Investor Relations, Inc.
Sarah McCabe, +1 212-362-1200
sarah@sternir.com
or
Media Relations (U.S.)
Russo Partners
Tony Russo, Ph.D.
Victoria Meissner, M.D.
tony.russo@russopartnersllc.com
victoria.meissner@russopartnersllc.com
+1 212-845-4251
+1 646-942-5627

Om Business Wire

Business Wire
Business Wire
24 Martin Lane
EC4R 0DR London

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

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

Keio Plaza Hotel Tokyo Starts "Ikebana" Flower Arrangement Workshops16.8.2017 14:00Pressemelding

Keio Plaza Hotel Tokyo, one of Japan’s most prestigious international hotels located in Shinjuku, Tokyo, starts special 45 minute classes to teach overseas guests about “Ikebana” flower arrangement which will be held on every other Thursday beginning from August in a private room for JPY3,000 per participant. Instruction and commentary will be provided by the renowned flower arrangement artist Hiroki Maeno. This Smart News Release features multimedia. View the full release here: http://www.businesswire.com/news/home/20170816005431/en/ From August, 2017, Keio Plaza Hotel Tokyo starts special 45 minute classes to teach overseas guests about "Ikebana" flower arrangement workshops. (Photo: Business Wire) Flower arrangement artist Hiroki Maeno has created special and unique flower arrangements for display in the main lobby of our hotel with each cha

Maxion Wheels to Feature Market Leading Commercial Vehicle Steel Wheels at COMTRANS 201716.8.2017 13:00Pressemelding

Maxion Wheels, the world’s largest wheel manufacturer, announced today its participation in the biennial COMTRANS / 17 International Commercial Vehicle Show held in Moscow, Russia from September 4 – 9, 2017, in Hall 15/ Stand 15-432. Customers and visitors to the Maxion Wheels exhibit will have the opportunity to learn more about the state-of-the-art commercial vehicle steel wheels that have supported the success of global original equipment manufacturers for almost 100 years. One of the key products on display will be the new 8.5” x 24” wheel for tubeless tire applications in Russia, the Middle East and Africa. The wheel boasts 15 percent less weight than the prior generation wheel, enhancing tire life and improving fuel efficiency. ABOUT MAXION WHEELS Maxion Wheels, a division of IOCHPE-MAXION S.A., is a leading wheel manufacturer for passenger cars,

Out-of-this-World Science Project to be Featured as Keynote at SC17’s High Performance Computing Conference in Denver16.8.2017 13:00Pressemelding

Professor Philip Diamond, Director General of the international Square Kilometer Array (SKA) project, will be the keynote speaker at SC17, the International Conference for High Performance Computing, Networking, Storage and Analysis in Denver, Nov. 12-17. This Smart News Release features multimedia. View the full release here: http://www.businesswire.com/news/home/20170816005129/en/ SC17 Keynote Speaker Philip Diamond (Photo: Business Wire) SKA is an international collaboration to build the world’s largest radio telescope that will change our understanding of space as we know it. Professor Diamond, accompanied by Dr. Rosie Bolton, SKA Regional Centre Project Scientist, will take SC17 attendees around the globe and out into the deepest reaches of the observable universe as they describe the SKA’s international partnership that will map and study the e

Scientist.com Ranks No. 155 on the 2017 Inc. 5000 List of Fastest-Growing Companies16.8.2017 12:06Pressemelding

Scientist.com, the leading marketplace for outsourced scientific services, today announced that it ranked no. 155 on Inc. magazine’s 2017 List of the 5000 Fastest-Growing privately-owned companies in America, and it ranked no. 11 in the country in the Health category. The San Diego-based company builds private marketplaces that connect many of the world’s largest life science research organizations with a global network of thousands of research suppliers. “Using Scientist.com, research organizations of pharmaceutical companies, biotech start-ups and academic and research institutes can get near instant access to thousands of the latest research technologies and services,” stated Paul A. Stone, JD, Partner at 5AM Ventures, a Scientist.com investor. “The marketplace saves researchers time, reduces costs, promotes access to innovation and ensures regulatory compliance. Scientist.com is im

CMB Successfully Goes-Live on Murex’s MX.3 Platform16.8.2017 08:00Pressemelding

Murex, a global leader in trading, risk management and processing solutions, is pleased to announce the successful launch of the MX.3 platform at China Merchants Bank (CMB). As one of China’s largest commercial banks, CMB financial market department currently averages 8,000 trades per day with a portfolio of 320,000 live trades on MX.3. CMB decided to launch the project to implement an enterprise-wide solution for their capital markets teams based in Shenzhen and Shanghai. This was done in order to improve control over their end-to-end trading cycle from front-to-back through risk, streamline and automate their operations, and enable new business. Murex’s MX.3 platform was selected as it provided the functional, geographical and product coverage that was required by the bank. Covering Front Office, Processing, Finance, and Risk Management, MX.3 also provides VaR analytics across all asset

euNetworks Adds New High Density Fibre Network in Manchester16.8.2017 08:00Pressemelding

euNetworks, a provider of bandwidth services in Europe, today announced it has made further investment in Manchester in the United Kingdom, having built out a new high density fibre network covering over 40 route kilometres in the city. This follows initial expansion into Manchester’s MediaCityUK in May 2016, which supported euNetworks’ Media customer’s bandwidth needs, as well as enabling a seamless network experience from London through to Manchester and Dublin and onwards to Europe. This Smart News Release features multimedia. View the full release here: http://www.businesswire.com/news/home/20170816005067/en/ euNetworks Fibre in Manchester (Photo: Business Wire) This new network in Manchester city was designed for resilience, with fully diverse nodes. euNetworks undertook a combination of new construction and duct acquisition to complete this network

I vårt presserom finner du alle våre siste saker, kontaktpersoner, bilder, dokumenter og annen relevant informasjon om oss.

Besøk vårt presserom