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

PARIS–(BUSINESS WIRE)– 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 http://www.hnf-cure.org/cmtsummit/

“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 www.hnf-cure.org

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 www.pharnext.com

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