Pharnext’s PXT3003 Featured at the American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) 2017 Annual Meeting
Regulatory News:
Pharnext SA (Paris:ALPHA) (FR00111911287 - ALPHA), a biopharmaceutical company pioneering a new approach to the development of innovative drugs based on the combination and repositioning of known drugs, today announced that data related to PXT3003, the Company’s lead PLEODRUG™, in development for the treatment of Charcot-Matrie-tooth type 1A (CMT1A) disease will be presented in a poster viewing session at the AANEM 2017 Annual Meeting, September 13-16, in Phoenix, Arizona, U.S.
Details are as follow:
| Date & Time | Title | |
|
September
|
Poster #90:
“A multicenter, double-blind,
placebo-controlled, pivotal Phase III study
|
The abstract is available online at : http://www.aanem.org/Meetings/Annual-Meeting/Abstracts
About PXT3003
PXT3003, Pharnext’s lead PLEODRUG™ in development for the treatment of Charcot-Marie-Tooth type 1A disease (CMT1A), is a novel, synergistic, low-dose combination of baclofen, naltrexone, and D-sorbitol formulated as an oral solution given twice-daily. PXT3003 has multiple main mechanisms of action: a synergistic inhibition of PMP22 gene overexpression associated with myelination improvement, direct nerve protection and additional positive effects on other cellular types: muscle cells, neuromuscular junctions and immune cells. PXT3003 obtained positive results in a Phase II clinical trial in 80 adult patients with CMT1A. In 2014, the EMA and FDA granted orphan drug designation to PXT3003 for the treatment of CMT1A in adults. An international pivotal Phase III trial (PLEO-CMT) in over 300 adult patients with CMT1A is now underway at 30 sites across Europe, the U.S. and Canada.
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 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 new optimal lower doses. These PLEODRUG™ offer several key advantages: efficacy, safety and intellectual property including several product or composition of matter patents already granted. The Company is supported by a world-class scientific team.
The company Pharnext is listed on Euronext Growth Stock Exchange in
Paris (ISIN code: FR00111911287).
For more information, visit www.pharnext.com
To view this piece of content from cts.businesswire.com, please give your consent at the top of this page.
View source version on businesswire.com: http://www.businesswire.com/news/home/20170907006130/en/
Contact information
Pharnext
Xavier Paoli, +33 (0)1 41 09 22 30
Chief
Commercial Officer
contact@pharnext.com
or
Investor
Relations (Europe)
MC Services AG
Anne Hennecke, +49 211
529252 22
anne.hennecke@mc-services.eu
or
Media
Relations (Europe)
ALIZE RP
Caroline Carmagnol
Margaux
Pronost
+33 (0)1 44 54 36 64
pharnext@alizerp.com
or
Pharnext
René
Goedkoop, +33 (0)1 41 09 22 30
Chief Medical Officer
contact@pharnext.com
or
Investor
Relations (U.S.)
Stern Investor Relations, Inc.
Matthew
Shinseki, +1 212-362-1200
matthew@sternir.com
or
Media
Relations (U.S.)
Russo Partners
Tony Russo
Scott
Santiamo
tony.russo@russopartnersllc.com
scott.santiamo@russopartnersllc.com
+1
212-845-4251
+1 718-344-5843
or
Financial
Communication (France)
New CAP
Emmanuel Huynh, +33 (0)1 44
71 20 40
pharnext@newcap.eu
About Business Wire
For more than 50 years, Business Wire has been the global leader in press release distribution and regulatory disclosure.
Subscribe to releases from Business Wire
Subscribe to all the latest releases from Business Wire by registering your e-mail address below. You can unsubscribe at any time.
Latest releases from Business Wire
Vertex Receives CHMP Positive Opinion for ALYFTREK ® , a New Once-Daily CFTR Modulator for the Treatment of Cystic Fibrosis28.4.2025 09:03:00 EEST | Press release
Vertex Pharmaceuticals (Nasdaq: VRTX) today announced that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion for ALYFTREK®(deutivacaftor/tezacaftor/vanzacaftor)for the treatment of people with cystic fibrosis (CF) ages 6 years and older who have at least one non-class I mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. “Our goal has always been to serially innovate to help people with cystic fibrosis live healthier and longer lives. If approved, this new medicine would be indicated for people with CF ages 6 years and older with at least one non-class I mutation, meaning more patients would be eligible for a medicine that gets them closer to normal levels of sweat chloride,” said Carmen Bozic, M.D., Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer, Vertex. “CFTR modulators have already revolutionized the way we treat CF and I am encoura
Prilenia Enters into a Collaboration and License Agreement with Ferrer for the Commercialization and Co-Development of Pridopidine in Europe and Other Select Markets28.4.2025 08:02:00 EEST | Press release
Prilenia Therapeutics B.V., a biopharmaceutical company driven by an unwavering commitment to scientific excellence and accelerating progress for people affected by Huntington’s disease (HD) and amyotrophic lateral sclerosis (ALS), today announced that it has entered into a collaboration and license agreement with Ferrer for the commercialization and further development of pridopidine in Europe and other select markets. Pridopidine is a potent and highly selective, orally administered sigma-1 receptor (S1R) agonist designed to regulate key neuroprotective mechanisms often impaired in neurodegenerative diseases such as HD and ALS. Under the terms of the agreement, Prilenia will receive an upfront payment of approximately €80 million plus up to €45 million in near-term development, regulatory, and commercial milestones. The total deal is valued at up to approximately €500 million in upfront and total milestone payments. In addition, Prilenia will receive tiered double-digit royalties on
Ferrer Enters Into a Collaboration and License Agreement With Prilenia for the Commercialization and Co-Development of Pridopidine28.4.2025 08:00:00 EEST | Press release
Ferrer, an international B Corp pharmaceutical company with an increasing focus on rare neurological diseases, and Prilenia Therapeutics B.V.,a clinical-stage biotech company, have announced the signing of a strategic co-development and license agreement in which Ferrer obtains the rights to develop, manufacture and commercialize Pridopidine in the European Region, the Middle East and North African Region, the Southern African Region, the Central and South American Region, and the Commonwealth of Independent States Region. Pridopidine, a potent and highly selective, orally administered sigma-1 receptor agonist, designed to regulate key neuroprotective mechanisms that are often impaired in neurodegenerative diseases, is a promising candidate for the treatment of Huntington’s Disease (HD), a rare inherited neurodegenerative disease, with a high unmet medical need1. It has been studied in more than 1,700 people and long-term safety data of up to 7 years duration are available from previou
Innorna Announces FDA Rare Pediatric Disease and Orphan Drug Designations Granted to IN013 for Treatment of Wilson Disease27.4.2025 12:36:00 EEST | Press release
Innorna, a clinical-stage biotechnology company revolutionizing mRNA therapeutics with its innovative lipid nanoparticle (LNP) delivery technology, today announced the U.S. Food and Drug Administration (FDA) has granted both Rare Pediatric Disease Designation (RPDD) and Orphan Drug Designation (ODD) to its investigational mRNA therapy, IN013, for treating Wilson Disease (WD). This dual achievement accelerates the clinical development of IN013, advancing Innorna’s mission to deliver transformative therapies for WD patients. About FDA Designations The Rare Pediatric Disease Designation (RPDD) incentivizes therapies for serious or life-threatening diseases affecting fewer than 200,000 U.S. patients, primarily those aged 18 or younger. Sponsors may qualify for a Priority Review Voucher (PRV) upon approval to expedite FDA review of a subsequent drug application. The Orphan Drug Designation (ODD) supports therapies targeting rare diseases (affecting fewer than 200,000 U.S. patients) by provi
Everen Specialty Appoints Carla Greaves Chief Underwriting Officer25.4.2025 20:00:00 EEST | Press release
Everen Specialty, a Bermuda-based (re)insurer for energy markets worldwide, today announced the appointment of Carla Greaves as its new Chief Underwriting Officer (CUO). This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20250425273777/en/ Carla Greaves Ms. Greaves will join the Executive Leadership Team of the Everen Group, based in the Bermuda office, later this year. She succeeds Jane Peterson, Interim CUO, who will continue in a consultancy capacity to facilitate the transition. With more than 30 years of underwriting and leadership experience in the (re)insurance industry, Ms. Greaves brings a wealth of expertise and a proven track record of success in the Casualty market where she is recognized for building high-performing teams, driving profitable growth, and successfully navigating complex market environments. Prior to joining Everen Specialty, Ms. Greaves held increasingly senior leadership positions, most recently servin
In our pressroom you can read all our latest releases, find our press contacts, images, documents and other relevant information about us.
Visit our pressroom