Pharnext Announces that the Data Safety Monitoring Board Recommends Continuing the Ongoing Phase 3 Trial of PXT3003 for Charcot-Marie-Tooth Disease Type 1A

PARIS–(BUSINESS WIRE)– Regulatory News:

Pharnext
SA (FR00111911287 – ALPHA), a French biopharmaceutical
company developing an advanced portfolio of products in the field of
neurodegenerative diseases, today announced that the independent Data
Safety Monitoring Board (DSMB) has completed its first pre-specified
safety evaluation of PXT3003 in the ongoing PLEO-CMT Phase 3 clinical
trial. Based on a review of safety data from 100 patients who completed
at least three months of study treatment, the DSMB recommended
continuing the PLEO-CMT study as planned.

PLEO-CMT is an international pivotal Phase 3 study that was initiated in
December 2015 and is planned to enroll 300 patients with mild to
moderate CMT1A from Europe and the U.S. by the end of December 2016.
Patients will be randomized in three arms – placebo and two PXT3003
doses – and will receive study treatment over 15 months. 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.

The DSMB is an independent body of experts drawn from the fields of
clinical medicine, biostatistics and study methodology, chartered to
provide recommendations to Pharnext upon regular pre-specified review of
the accumulated data during the conduct of the clinical trial.

“We believe this clinical trial has the potential to be a crucial
turning point in the effort to finally provide an efficacious treatment
for patients suffering from CMT1A,” said Daniel Cohen, M.D.,
Ph.D., Co-Founder and Chief Executive Officer of Pharnext.
“Today’s therapeutic options are very limited and mostly palliative in
nature. Our PLEODRUG® PXT3003 has already demonstrated safety,
tolerability and improvements in CMT1A patient disability in a Phase 2
trial. Given this positive safety assessment by the DSMB, we are hopeful
to bring this much-needed potential therapy to patients suffering from
this debilitating condition upon completion of this Phase 3 trial.”

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 which will enroll 300
patients aged 16 and older with mild to moderate CMT1A in Europe and the
U.S. Diagnosis of CMT1A will be 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
dosage variations 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

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