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Press Release

Pharnext Announces Support of the First Patient-Centered Charcot-Marie-Tooth Summit Organized by the Hereditary Neuropathy Foundation

Posted on: 04 Oct 16

Regulatory News:

Pharnext (Paris:ALPHA) (ISIN FR00111911287), a French biopharmaceutical company developing an advanced portfolio of products in the field of neurodegenerative diseases, today announced its support of the first annual Patient-Centered Charcot-Marie-Tooth (CMT) Summit organized by the Hereditary Neuropathy Foundation (HNF) on Thursday Oct. 6, 2016 in New York City.

The landmark event will focus on innovative content involving patients, caregivers, industry representatives, researchers and clinicians presenting the latest research data, sharing personal stories, learning about novel products as well as engaging in interactive discussions and Q&A sessions. Pharnext supports this HNF initiative as a part of a partnership initiated with the foundation in 2014 aimed to increase awareness around the disease and support the development of a stronger CMT patient and research community. Pharnext executives will attend the summit, meet with the various stakeholders of the CMT and rare diseases communities, and share the latest translational research findings.

For more information on the event, visit

“This summit is a major milestone in our CMT awareness program and gives us an opportunity to draw attention to the disease and educate the public about life with this rare genetic condition,” said Allison Moore, Founder and Chief Executive Officer of the Hereditary Neuropathy Foundation (HNF). The event is unique in that it directly involves patients in discussions, and connects them with clinicians and scientists. We hope that bringing physicians, researchers, industry, patients, as well as family and friends of people who live with CMT together will lead to more patient-oriented clinical care, make research more centered around patient needs and help find new solutions for people affected by CMT.”

Daniel Cohen, M.D., Ph.D., Co-Founder and Chief Executive Officer of Pharnext, added “Pharnext is honored to be a part of the summit and supports the efforts of this inspiring initiative. PXT3003, our lead candidate for the treatment of patients with CMT1A, is currently in an ongoing Phase 3 clinical trial, making it important for us to meet with patients and their families to keep our research as patient-focused as possible and to understand the quality of life issues they face daily. We are also proud to be part of this effort to start the dialogue across all groups and give a voice to these patients.”

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 PMP 22 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 sensory 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 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 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 “pleodrugs” 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

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Last updated on: 04/10/2016

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