Business Wire

Pharnext Announces Findings Evaluating Charcot-Marie-Tooth Neuropathy Score

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

Kite’s Yescarta™ (Axicabtagene Ciloleucel) Becomes First CAR T Therapy Approved by the FDA for the Treatment of Adult Patients With Relapsed or Refractory Large B-Cell Lymphoma After Two or More Lines of Systemic Therapy18.10.2017 21:43Pressemelding

Kite, a Gilead Company, (Nasdaq: GILD) today announced that the U.S. Food and Drug Administration (FDA) has granted regular approval to Yescarta™ (axicabtagene ciloleucel), the first chimeric antigen receptor T cell (CAR T) therapy for the treatment of adult patients with relapsed or refractory large B-cell lymphoma after two or more lines of systemic therapy, including diffuse large B-cell lymphoma (DLBCL) not otherwise specified, primary mediastinal large B-cell lymphoma (PMBCL), high-grade B-cell lymphoma, and DLBCL arising from follicular lymphoma (transformed follicular lymphoma, or TFL). Yescarta is not indicated for the treatment of patients with primary central nervous system lymphoma. This press release features multimedia. View the full release here: http://www.businesswire.com/news/home/20171018006639/en/ CAR T therapy is a breakthrough in hematologic cancer treatment in which

IFF Launches Re-Imagine…™ Programs to Tap Unmet Consumer Opportunities, Speed Innovation18.10.2017 20:15Pressemelding

Regulatory News: International Flavors & Fragrances Inc. (NYSE:IFF) (Euronext Paris:IFF), a leading innovator of sensory experiences that move the world, launched their Re-Imagine programs to accelerate innovation and increase agility to capture unmet opportunities in the changing food and beverage market. Based on a combination of future trends analysis, consumer insights, and a modernized cross-category development process, the programs guide the Company’s research and development efforts to ensure an innovation pipeline that addresses evolving consumer needs and desires. “The Re-Imagine programs area tangible expression of our commitment to advance taste for our customers – and consumers,” said IFF Chairman and CEO Andreas Fibig. “It is another way in which we show how we are dedicated to being our customers’ partner of choice as we progress on our business strategy, imbu

Sparkol Launches VideoScribe 318.10.2017 13:24Pressemelding

Introducing VideoScribe Version 3 by Sparkol. Already used by over two million people across the world to create engaging explainer videos, Version 3 of the highly intuitive software comes packed full of new features, including: An improved user experience and slick UI - animate easier than ever before Enhanced search - find exactly the image you need in record time Intelligent image recommendations - focus your creativity on your video instead Smoother performance - maximum productivity when using the software VideoScribe customers include the world’s largest brands (HP, BBC, Sky and the University of British Columbia), businesses across every sector, leading education providers, award-winning teachers, charities, campaigners, and coun

Piraeus Bank: Agreement for the Sale of Serbian Operations18.10.2017 13:06Pressemelding

Piraeus Bank announces that it has entered into an agreement to sell its Serbian banking and leasing operations to Direktna Banka A.D., a local Serbian banking group that has been strongly growing its presence in the market, for a total cash consideration between €58mn up to €61mn, depending on the financial performance of the divested assets until completion of the transaction, through a combination of direct sale price and simultaneous reduction of capital in Piraeus Bank Beograd AD. The transaction is conditional upon the usual corporate and regulatory approvals, including those of the National Bank of Serbia and the Hellenic Financial Stability Fund. The transaction represents another step towards the implementation of Piraeus Bank’s Restructuring Plan commitments, as those were agreed with the Directorate General of Competition of the European Commission. The transactio

Introducing InVision Studio, The World’s Most Powerful Screen Design Tool18.10.2017 13:01Pressemelding

InVision, the product design platform powering the world’s best digital experiences, today introduced Studio, a powerful new tool that will change the way screen design is done. InVision Studio was created by working closely with the world’s best design teams and finding inspiration in how they create beloved digital products. The launch of Studio comes at a time when modern organizations are shaping and redefining entire markets through their focus on digital customer experience. InVision already powers the product design process of more than 3 million people at tens of thousands of companies, including eighty percent of the Fortune 100 and brands like Airbnb, Uber, HBO, Amazon, IBM, Nike, and Slack. This new product reflects a major evolution of the digital product design workflow. With Studio, InVision has developed an end-to-end platform for designing digit

SmartStream and Credit Suisse Expand their Agreement from the Processing of Invoices and Reconciliations to Now Include Listed Derivatives Brokerage Fees18.10.2017 12:00Pressemelding

SmartStream Technologies, the financial Transaction Lifecycle Management (TLM®) solutions provider, today announced that its original agreement with Credit Suisse was recently expanded to include the processing of Listed Derivatives Brokerage fees which began in February of this year. This expansion in SmartStream’s partnership with Credit Suisse complements its existing managed service solution for the processing of invoices and reconciliations within Credit Suisse’s OTC Fixed Income Derivatives, US Listed Equity Options and Cash Equities businesses across brokerage, clearing and exchange fees operations, which began in June of 2016. The managed service enables the bank to advance the operational efficiencies created through the use of the utility-based solutions offered by SmartStream, utilizing their proprietary execution fees and expense management software. By centralizing the ent

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

Besøk vårt presserom