Pharnext: New Data Published in PLOS ONE Demonstrates Early Therapeutic Effects of Pharnext's PLEODRUG PXT3003 in a Transgenic Rat Model of Charcot-Marie-Tooth disease Type 1A

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Pharnext 
Pharnext: New Data Published in PLOS ONE Demonstrates Early Therapeutic Effects 
of Pharnext's PLEODRUG PXT3003 in a Transgenic Rat Model of 
Charcot-Marie-Tooth disease Type 1A 
 
17-Jan-2019 / 08:00 CET/CEST 
Dissemination of a French Regulatory News, transmitted by EQS Group. 
The issuer is solely responsible for the content of this announcement. 
 
*PARIS, France,**8:00 am**, 17 January 2019 (CET)**-**Pharnext SA 
[1](FR0011191287 - ALPHA)*a biopharmaceutical company pioneering a new approach 
to the development of innovative drug combinations based on big genomic data and 
artificial intelligence, today announced the publication of new preclinical 
results for its lead PLEODRUG PXT3003 in PLOS ONE showing that early 
treatment with PXT3003 in a transgenic rat model delays the onset of 
Charcot-Marie-Tooth disease Type 1A (CMT1A). 
The publication, titled "Early short-term PXT3003 combinational therapy delays 
disease onset in a transgenic rat model of Charcot-Marie-Tooth disease 1A 
(CMT1A)," by Prukop _et al._, reported that early treatment with PXT3003 
prevented a set of clinical and molecular manifestations of CMT1A after 
short-term dosing in juvenile _PMP22_transgenic rats, a well established animal 
model of CMT1A. Results from the study indicated that an early postnatal, 
short-term treatment with PXT3003 in CMT1A rats: 
 
  · Delayed disease onset into adulthood; 
 
  · Corrected motor deficits; 
 
  · Ameliorated the disturbed axon caliber distribution with a shift towards 
  large motor axons; 
 
  · Reduced _PMP22_mRNA overexpression; 
 
  · Improved the dysbalanced molecular pathways (AKT/ERK) involved in Schwann 
  cell differentiation. 
 
The paper can be accessed at 
https://journals.plos.org/plosone/article?id=10.1371/journal.pone.0209752 
*Michael Sereda, M.D., Professor of Neurology at the Max Planck Institute of 
Experimental Medicine (MPI-EM) and the University Medical Center (UMG), 
Göttingen, Germany*,said: "We were surprised that only two weeks of early, 
post-natal treatment with PXT3003 dramatically improved the motor phenotype in 
CMT1A rats reaching wildtype levels. Similar to previous preclinical studies, 
this underlines the importance of treating the molecular defects caused by PMP22 
overexpression in CMT1A during this critical time window in myelin development." 
*Daniel Cohen, M.D., Ph.D., Co-Founder and Chief Executive Officer of Pharnext*, 
said: "These results provide key insights into the disease progression of CMT1A 
and the potential of PXT3003 to slow the onset of the disease, particularly in 
children and adolescents with CMT1A. Such findings validate our preclinical data 
on PXT3003 in CMT1A to-date, as well as our clinical data, based on the positive 
top-line Phase 3 data from October 2018. From the findings in this publication, 
we believe that PXT3003 has the potential to change the treatment paradigm for 
children with CMT1A - for whom there are currently no pharmacological treatments 
available - and we look forward to initiating a Phase 3 trial of PXT3003 in 
pediatric patients by the end of 2019." 
Pharnext intends to lauch a pediatric Phase 3 clinical trial in children to 
investigate the safety and efficacy of PXT3003. The investigation plan was agreed 
with the European Medicines Agency. 
 
*About PXT3003 * 
Pharnext's first-in-class PLEODRUG PXT3003, developed using Pharnext's R&D 
platform, PLEOTHERAPY, is a novel oral fixed-dose combination of baclofen, 
naltrexone and sorbitol, with Orphan Drug Designation in EU and the USA. PXT3003, 
Pharnext's lead PLEODRUG, has shown positive results both in preclinical and 
Phase 2 studies for the treatment of CMT1A. These results were published in the 
Orphanet Journal of Rare Diseases (OJRD) in December 20142,3. In preclinical 
studies, PXT3003 inhibited the overexpression of the PMP22 gene, improved 
myelination of peripheral nerves and motor / sensory impairments. In a Phase 2 
clinical trial in 80 adult patients with CMT1A, PXT3003 improved multiple 
efficacy endpoints beyond stabilization, particularly the ONLS scale. In 
addition, PXT3003 was safe and well-tolerated. In December 2015, Pharnext 
initiated the PLEO-CMT study, a pivotal 15-month, double-blind Phase 3 study that 
assessed the efficacy and safety of PXT3003 in 323 CMT1A patients aged 16 to 65 
years. In this study, PXT3003 met the FDA and EMA pre-specified primary endpoint 
of ONLS with a statistically significant difference compared to placebo 
(p=0.008). PLEO-CMT was followed by a 9-month, open-label, follow-up extension 
study, PLEO-CMT-FU, initiated in March 2016 and currently ongoing. PLEO-CMT-FU, 
designed to assess the long-term safety and tolerability of PXT3003, enrolled 
patients who completed the PLEO-CMT study. Pharnext expects to initiate a Phase 3 
trial of PXT3003 in pediatric CMT1A patients by the end of 2019, based on a 
Pediatric Investigation Plan (PIP) agreed upon with the EMA. 
 
*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 USA 
and EU. 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) and nerve dysfunction. As a result, 
patients suffer from progressive muscle atrophy of the limbs causing problems 
with walking, running balance and hand function. At least 5% need wheelchairs. 
They may have a loss of sensation and pain. Patients with CMT1A have reduced 
quality of life. The first symptoms usually appear during childhood and 
progressively evolve throughout patients' lives. 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 Pharnext* 
Pharnext is an advanced clinical-stage biopharmaceutical company developing novel 
therapeutics for orphan and common neurodegenerative diseases that currently lack 
curative and/or disease-modifying treatments. Pharnext has two products in 
clinical development. PXT3003 completed an international pivotal Phase 3 trial 
with positive topline results 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 encouraging Phase 2 results in Alzheimer's disease. Pharnext 
has developed a new drug discovery paradigm based on big genomic data and 
artificial intelligence: PLEOTHERAPY. The Company identifies and develops 
synergic combinations of drugs called PLEODRUG offering several key 
advantages: efficacy, safety and robust intellectual property. The Company was 
founded by renowned scientists and entrepreneurs including Professor Daniel 
Cohen, a pioneer in modern genomics, and 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 www.pharnext.com [1] 
 
*CONTACTS:* 
*Pharnext* 
Rodolphe Hajj 
Chief Pharmacology 
Officer 
[e-mail] 
[phone] 
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tion.com 
[phone] 
 
Regulatory filing PDF file 
 
Document title: Pharnext: New Data Published in PLOS ONE Demonstrates Early 
Therapeutic Effects of Pharnext's PLEODRUG PXT3003 in a Transgenic Rat Model 
of Charcot-Marie-Tooth disease Type 1A 
Document: http://n.eqs.com/c/fncls.ssp?u=OFFVQPJFOD [3] 
766907 17-Jan-2019 CET/CEST 
 
 
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January 17, 2019 02:00 ET (07:00 GMT)