Pharnext: Shareholder Letter -2-

DJ Pharnext: Shareholder Letter

Pharnext 
Pharnext: Shareholder Letter 
 
29-Oct-2020 / 08:30 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, 08:30 a.m. CET, October 29, 2020 - Pharnext SA (FR0011191287 - ALPHA), 
an advanced late-stage clinical biopharmaceutical company pioneering new approaches to 
developing innovative drug combinations based on big genomics data and artificial 
intelligence ("Company") using its PLEOTHERAPY platform, today published a Letter 
to Shareholders from its Chief Executive Officer, Dr. David Horn Solomon. 
 
Dear Fellow Shareholder, 
 
2020 is a pivotal year for Pharnext's development, and we look forward to continued 
growth and progress in the remainder of the year and ahead. Over the course of this 
year, we have made several operational and clinical advances, including important 
leadership appointments, addition of highly qualified independent directors to our 
Board, and most importantly, we received guidance from the FDA on the development path 
forward for our lead asset, PXT3003, for the treatment of Charcot-Marie-Tooth disease 
type 1A (CTM1A), a rare and highly debilitating disease. We believe that we are 
well-positioned to initiate and execute our second pivotal Phase III clinical trial for 
our lead program, PXT3003, in CMT1A. 
 
Our Focus on PXT3003 and Upcoming Pivotal Phase III Clinical Trial in CMT1A 
 
We continue to focus most of our resources on developing PXT3003 for CMT1A with the 
goal of obtaining marketing approval from FDA and EMA. PXT3003 is a novel combination 
of naltrexone, baclofen and sorbitol, taken orally, and has Orphan Drug Designation 
from both FDA and EMA. Currently, there are no approved therapies to treat CMT1A, and 
PXT3003 is the most advanced pharmaceutical product candidate in development. Given 
there are over 100,000 CMT1A patients across the US and EU5 markets with little 
competition, we believe that the global annual commercial revenue opportunity for 
PXT3003 exceeds $1 billion. Our Company has been transformed and is dedicated to 
successfully bringing PXT3003 to the market to treat CMT1A patients, improving their 
quality of life and reducing the burden on their families and caregivers. 
 
Key Events in 2020: 
 
Strengthened senior management with key hires 
 
I joined the Pharnext team as the Chief Executive Officer in April 2020. Having spent 
almost 30 years in the pharmaceutical industry and serving as the Chief Executive 
Officer of Silence Therapeutics and Zealand Pharma previously, I have a tremendous 
appreciation for Pharnext's vision and rich pipeline assets. 
 
I am honored to have been given the opportunity to lead Pharnext through this next 
phase of growth. Our PLEOTHERAPY platform, by leveraging genomic big data and 
artificial intelligence, truly offers a novel and innovative approach to identifying 
potential treatments for a variety of indications. I am grateful to Daniel Cohen who, 
as a visionary founder, explored the opportunities to leverage the PLEOTHERAPY 
platform to continue to build our pipeline. 
 
In August 2020, Dr. Adrian Hepner joined Pharnext as Chief Medical Officer and Head of 
R&D. Dr. Hepner has more than 30 years of industry experience in biomedical research, 
clinical drug development and medical affairs. Prior to joining Pharnext, he held 
executive positions at Eagle Pharmaceuticals, Avanir Pharmaceuticals, BioDelivery 
Sciences International (BDSI) and UCB BioSciences, Inc. Dr. Hepner received an M.D. and 
Ph.D. in Psychiatry and Neurology from the University of Buenos Aires. He also 
completed a post-doctoral fellowship in Neuro-Psychopharmacology at the University of 
Ottawa and additional training at Harvard University. Adrian's drive and passion for 
drug development has led to 8 NDAs resulting in approved medicines. 
 
The senior management team also includes Peter Collum, our Chief Financial and Business 
Officer, who joined Pharnext in July 2019 after a 17-year career in life sciences 
investment banking with both Bank of America and MTS Health Partners, as well as a 
5-year career in the pharmaceutical industry with Roche as an engineer. Peter brings to 
our company a remarkable knowledge of financial markets and the pharmaceutical 
industry. 
 
Together with the other talented members of management, we believe we now have a strong 
and complete team of executives and employees with expertise and invaluable experience 
to position the Company to meet our strategic objectives and ultimately develop 
therapies for patients in need. 
 
Interaction with the U.S. Food and Drug Administration ("FDA") 
 
In June 2020, we had a productive meeting with the FDA to discuss the regulatory path 
forward for PXT3003 and appreciate the agency's continued collaborative approach in our 
clinical advancement of PXT3003. Based on FDA guidance, Pharnext is preparing to 
conduct an additional pivotal Phase III trial for which the FDA has recommended that 
the primary endpoint be the Overall Neuropathy Limitations Scale ("ONLS"). This study 
will have two arms to compare high dose and placebo. I would like to point out that in 
our original Phase III study, the high dose patients showed encouraging responses using 
the same primary endpoint, ONLS. We expect to launch this trial before the end of Q1 
2021. In the meantime, we are running an open label study for patients enrolled in the 
original Phase III trial. 
 
We submitted a Special Protocol Assessment (SPA) to the FDA on September 15 in order to 
further define the new Phase III study protocol. 
 
If successful, the data results from the upcoming Phase III trial will be used to 
support the New Drug Application ("NDA") for PXT3003 in CMT1A. 
 
Appointment of new directors to the Board 
 
As part of our 2020 Annual General Meeting, the Company proposed a transformation of 
the Board consistent with the Company's vision of developing and commercializing 
PXT3003 in the U.S. and Europe for CMT1A. The newly appointed directors have strong 
biopharmaceutical and business expertise focused on clinical development, neurology, 
strategy, transactions, value creation and governance. The newly appointed directors 
include: 
 
  · Alexandre Berda - Managing Director of CB Lux, Pharnext's largest shareholder. 
 
  · Dr. Jean Combalbert - Founder and CEO of Epics Therapeutics SA, Chairman of the 
  Board of Syndesi Therapeutics SA. 
 
  · Dr. Elisabeth Svanberg - Chief Development Officer at Ixaltis SA, Board-certified 
  general surgeon and Associate Professor of Surgery. 
 
  · Joshua Schafer - Chief Strategy and Business Development Officer of Mallinckrodt 
  Pharmaceuticals and Board member of Shuttle Pharmaceuticals. 
 
  · Dr. David Horn Solomon - Chief Executive Officer of Pharnext SA, Chairman of the 
  Board for Advicenne Pharma (PARIS: ADV) and Rexgenero in London. 
 
  · Dr. Lawrence Steinman - Zimmerman Professor of Neurology and Neurological Sciences, 
  Pediatrics and Genetics at Stanford University. 
 
In addition to the newly appointed directors, the Pharnext Board of Directors also 
includes Michel de Rosen (Chairman), Pierre Bastid, Kenneth Lee and Dr. Philippe 
Pouletty. Full biographies of Board Members can be found on Pharnext's website at 
https://pharnext.com/about [1]. We truly believe we have the right Board in place to 
oversee our value creation opportunity and effectively execute under their expertise. 
 
Entered a Research Collaboration with Charcot-Marie-Tooth Association 
 
In September, we signed a research collaboration with the Charcot-Marie-Tooth 
Association (CMTA), a United States patient advocacy group, to investigate novel 
biomarkers associated with CMT1A. 
 
The primary objective of this collaboration is to identify and validate potential 
treatment responsive CMT1A biomarkers that could be further explored in future clinical 
studies, in particular the upcoming Phase III study of PXT3003. Notably, this 
collaboration will evaluate the potential of TMPRSS5, a recently identified Schwann 
cell-specific biomarker in CMT1A patients, to confirm if it can be used to assess 
treatment response in future clinical trials. We are very excited about this 
collaboration and look forward to conducting research that will potentially bring 
valuable insights for further development of PXT3003. 
 
The Future for Pharnext 
 
2020 has been a very productive and event-driven year for Pharnext. Our plan is to 
build on this momentum by advancing drug candidates from our PLEOTHERAPY pipeline 
through clinical development and, subject to regulatory approval, ultimately bring them 
to the market to fulfil the significant unmet medical need in our target patient 
population. Pharnext estimates that the funding requirements to complete our Phase III 
trial in CMT1A are approximately &euro70 million. Pharnext intends to fund its internal 
clinical development efforts through one or more equity raises, anticipated to be 
targeted in whole or in part at US and European institutional life science investors, 
at times and on terms that will be subject to prevailing market conditions. Pharnext 
expects to also benefit from the participation of existing shareholders in these equity 
raises further evidencing their support of the Company's strategy and future 
development path. 
 
With Best Regards, 
 
David Horn Solomon 
 
Chief Executive Officer 
 
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 and benefits 
from orphan drug status in Europe and the United States. PXT864 has generated 
encouraging Phase II results in Alzheimer's disease and will be advanced through 

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DJ Pharnext: Shareholder Letter -2-

partnerships. Pharnext has developed a new drug discovery paradigm based on big 
genomics data and artificial intelligence: PLEOTHERAPY. Pharnext identifies and 
develops synergic combinations of drugs called PLEODRUG. 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. More information 
can be found at www.pharnext.com [2]. 
 
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 document de base filed with the AMF 
on June 2, 2016 under number I.016-0050 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 
 
[e-mail] 
 
[phone] 
 
Media Relations              Media Relations (U.S.) Media Relations (France) 
(International) 
 
                             Rooney Partners LLC    Ulysse Communication 
Consilium Strategic 
Communications 
 
                             Jeffrey Freedman       Bruno Arabian 
 
Mary-Jane Elliott 
 
                             [e-mail] [phone] 
 
Sukaina Virji 
 
                             [phone]        barabian 
                                                    [3]@ulysse-communication.com 
Alexandra Harrison 
 
                                                    Pierre-Louis Germain 
[e-mail] 
 
                                                    [phone] 
 
                                                    [e-mail] 
 
                                                    [phone] 
Investors Relations (U.S.)   Financial 
                             Communication (Europe) 
 
Stern Investor Relations, 
Inc.                         Actifin 
 
Janhavi Mohite               Stéphane Ruiz 
 
[e-mail]   [e-mail] 
 
[phone]              [phone] 
 
Regulatory filing PDF file 
 
File: Shareholder Letter [4] 
1143800 29-Oct-2020 CET/CEST 
 
 
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