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Pharnext’s PLEODRUG™ PXT3003 to be Featured at the Upcoming Hereditary Neuropathy Foundation (HNF) Annual Patient-Centered Summit


Regulatory News:

Pharnext SA (FR0011191287 - ALPHA) (Paris: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 it will participate at the upcoming Hereditary Neuropathy Foundation (HNF) Annual Patient-Centered Summit for Charcot-Marie-Tooth (CMT) and Hereditary Neuropathy With Liability To Pressure Palsies (HNPP), on November 3, 2017, in Boston (United States). Pharnext will present, in a poster and during an oral session, an update on its ongoing pivotal Phase 3 clinical trial evaluating its lead PLEODRUG™ PXT3003, in development for the treatment of Charcot-Marie-Tooth type 1A disease (CMT1A).

This unique event will bring together patients, caregivers, industry representatives, researchers and clinicians. This year, the summit will focus on pain. The objective is to come to an understanding of pain in the CMT/HNPP Community, including its impact on quality of life.

Details of the presentations are as follows:

  • Oral Session, November 3, 2017, from 4.15pm to 5.15 pm EST
    • Title : Update on Current Clinical Trials for CMT/HNPP
    • Presenters : René Goedkoop, MD, Chief Medical Officer, Pharnext, France and Kenneth Attie, MD, VP-Medical Research, Acceleron, United States
  • Poster session, November 3, 2017, all day
    • Title : “Status of the ongoing phase III study assessing the efficacy and safety of PXT3003 for CMT1A (PLEOCMT).” S. Attarian et al.
    • Presenter: René GoedKoop, MD, Chief Medical Officer, Pharnext, France

For more information about the event please visit

About Hereditary Neuropathy Foundation
Hereditary Neuropathy Foundation (HNF) is a US non-profit 501(c)3 organization whose mission is to increase awareness and accurate diagnosis of Charcot-Marie-Tooth disease (CMT) and related inherited neuropathies, support patients and families with critical information to improve quality of life, and support research. HNF has notably developed the Therapeutic Research in Accelerated Discovery (TRIAD) program, a collaborative effort with academia, government, and industry to support and develop treatments for CMT. For further information, visit

About PXT3003
PXT3003, developed using Pharnext’s R&D platform PLEOTHERAPY™, is a novel oral fixed-low dose combination of (RS)-baclofen, naltrexone hydrochloride and D-sorbitol with Orphan Drug Designation in Europe and the United States.

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.

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

Contact information

René GoedKoop, +33 (0)1 41 09 22 30
Chief Medical Officer
Investor Relations (Europe)
MC Services AG
Anne Hennecke, +49 211 529252 22
Investor Relations (U.S.)
Stern Investor Relations, Inc.
Matthew Shinseki, +1 212 362 1200
Financial Communication (France)
Stéphane Ruiz, +33 (0)1 56 88 11 15
Media Relations (Europe)
Caroline Carmagnol
Margaux Pronost
+33 (0)1 44 54 36 64
Media Relations (U.S.)
Marion Janic, +1 212 223 4017

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