and consistent results across preclinical and clinical studies in Phase II and Phase III (PLEO-CMT and PLEO-CMT-FU). 
More information can be found at https://pharnext.com/en/pipeline/pxt3003. 
 
 
About the PREMIER Trial 
The PREMIER trial is an international, randomized, double-blind, two-arm placebo-controlled, pivotal Phase III study, 
evaluating the efficacy and safety of PXT3003 versus placebo in mild-to-moderate CMT1A patients, over a 15-month 
period. The dose of PXT3003 tested in the PREMIER trial corresponds to the high dose ('HD') tested in the prior Phase 
III trial ('PLEO-CMT'). As agreed with regulatory agencies, the primary efficacy endpoint will be the Overall 
Neuropathy Limitations Scale ('ONLS') which measures functional motor disability. The secondary endpoints include the 
following outcome measures: 1) 10-Meter Walk Test ('10mWT'), 2) Quantified Muscular Testing (bilateral foot 
dorsiflexion dynamometry), 3) Patient Global Impression of Severity ('PGI-S'), 4) Patient Global Impression of Change 
('PGI-C'), 5) Charcot-Marie-Tooth Neuropathy Score, version 2 ('CMTNS-v2'), and 6) Quantified Muscular Testing (hand 
grip). Safety and tolerability will be monitored throughout the study. Further information on the PREMIER trial can be 
found on the ClinicalTrials.gov website (study identification number: NCT04762758) here. 
 
 
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 lead 
products in clinical development. PXT3003 completed an international Phase III trial with positive topline results for 
the treatment of Charcot-Marie-Tooth disease type 1A ('CMT1A') and benefits from orphan drug status in Europe and the 
United States. An international pivotal Phase III study of PXT3003 in CMT1A, the PREMIER trial, is currently ongoing. 
PXT864 has generated encouraging Phase II results in Alzheimer's disease and will be advanced through partnerships. 
Pharnext has developed a new drug discovery paradigm based on big genomics data and artificial intelligence: 
PLEOTHERAPY(TM). Pharnext identifies and develops synergic combinations of drugs called PLEODRUG(TM). More information 
can be found at www.pharnext.com. 
Pharnext is listed on the Euronext Growth Stock Exchange in Paris (ISIN code: FR0011191287). 
 
 
Disclaimer 
This press release contains certain forward-looking statements concerning Pharnext and its business, including in 
respect of timing of and prospects for clinical trials and regulatory submissions of the Company's product candidates 
as well as a potential financing transaction, the use of proceeds therefrom and cash runway. Such forward-looking 
statements are based on assumptions that Pharnext considers to be reasonable. However, there can be no assurance that 
the estimates contained in such forward-looking statements will be verified, which estimates are subject to numerous 
risks including the risks set forth in Pharnext's URD approved by the AMF on November 9, 2020 under number N° R. 20-029 
as well as in its annual periodic management reports and press releases (copies of which are available on 
www.pharnext.com) and to the development of economic conditions, financial markets and the markets in which Pharnext 
operates. The forward-looking statements contained in this press release are also subject to risks not yet known to 
Pharnext or not currently considered material by Pharnext. The occurrence of all or part of such risks could cause 
actual results, financial conditions, performance or achievements of Pharnext to be materially different from such 
forward-looking statements. Pharnext disclaims any intention or obligation to publicly update or revise any 
forward-looking statements, whether as a result of new information, future events, or otherwise. 
This press release and the information that it contains do not constitute an offer to sell or subscribe for, or a 
solicitation of an offer to purchase or subscribe for, Pharnext shares in any country, including the United States. The 
Company's securities may not be offered or sold in the United States absent registration or an exemption from 
registration; any public offering of securities to be made in the United States will be made by means of a prospectus 
that may be obtained from the issuer that will contain detailed information about the Company and management, as well 
as financial statements. 
 
 
 
 
 
 
Contacts 
 
 
David Horn Solomon 
Chief Executive Officer 
contact@pharnext.com 
 
+33 (0)1 41 09 22 30 
 
 
                                                                    Media Relations (France) 
Media Relations (International) 
                                   Financial Communication (Europe) Ulysse Communication 
Consilium Strategic Communications 
                                   Actifin                          Bruno Arabian 
Mary-Jane Elliott 
                                   Ghislaine Gasparetto             barabian@ulysse-communication.com 
Sukaina Virji 
                                   ggasparetto@actifin.fr           +33 (0)6 87 88 47 26 
Alexandra Harrison 
                                   +33 (0)6 21 10 49 24             +33 (0)1 81 70 96 30 
pharnext@consilium-comms.com 
 ----------------------------------------------------------------------------------------------------------------------- Regulatory filing PDF file File: Shareholder Letter June 2021

1208884 21-Jun-2021 CET/CEST


 
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June 21, 2021 02:31 ET (06:31 GMT)