Pharnext 
Pharnext: Shareholder Letter 
21-Jun-2021 / 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. 
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PARIS, France, 8:30 a.m. CET, June 21, 2021 - 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(TM) platform, today published a Letter to 
Shareholders from its Chief Executive Officer, Dr. David Horn Solomon. 
 
 
Dear Fellow Shareholders, 
 
In recent days, since we announced our financing on 07 June 2021, our share price has dropped significantly, raising 
questions from you. I thought I should therefore write to explain more details of our financing and our progress on 
PXT3003 in Charcot-Marie-Tooth disease type 1A ('CMT1A'). Financing this key asset and working towards an approval will 
benefit patients and shareholders and is why we work and invest in healthcare and why biotech companies exist. 
 
2021 has already seen our Company accomplish a significant key milestone: enrolling the first patient in March in our 
pivotal Phase III study of PXT3003, the PREMIER trial. As you are familiar, PXT3003 is our lead asset for the treatment 
of CTM1A, a rare and highly debilitating disease. In April, we announced topline results of the interim analysis from 
the ongoing open-label Phase III extension study (PLEO-CMT-FU), which followed the first double-blind Phase III 
(PLEO-CMT), suggesting a good safety profile and sustained efficacy of PXT3003 as measured by the Overall Neuropathy 
Limitation Scale ('ONLS'), after 4.5 years of total trial time. More recently, we secured a financing for a total 
amount of up to EUR81million over the next thirty-six months through a convertible bond program to extend the cash runway 
of the company and fund the PREMIER trial through to the data readout in Q3 2023. 
 
Financing 
This long-term financing, should the Company maintain the ability to draw down all the tranches over the next 36 
months, will thus allow the funding and completion of our pivotal Phase III in CMT1A and more globally extend the cash 
runaway of Pharnext to support its growth and development. We understand the frustration around the dilution associated 
with the convertible bond financing, but given the timing, the current development stage of our company and non-optimal 
market conditions, we feel the financing in this way benefits the company for several reasons: 
  ? Flexibility: The financing has been structured to ensure Pharnext is in control of the timing of drawing down the 
    various tranches based on our cash needs (for more details, please refer to the press release issued on June 7, 
    2021) . In addition, depending on circumstances, and in particular if we can access other sources of financing, we 
    may not need to draw down the full amount. Indeed, we do not exclude to pursue a capital increase, including on a 
    U.S. stock market. 
  ? Dilution: An equity raise would have created a significant one-time dilution for shareholders instead of spreading 
    the dilution over several years with the current financing. Morevover, subcribers of such an equity raise also have 
    the possibility to rapidly trade their shares in the stock market. Finally, part of the first tranche of the 
    current financing has been used to redeem the outstanding convertible bonds issued by the Company on February 3, 
    2021 for EUR2.9 million, avoiding additional dilutive impact. 
  ? Opportunity: This financing does not prevent us from pursuing deals and partnership discussions, or new 
    opportunities that could come up from our PLEOTHERAPY platform which we are currently working on, to expand and 
    diversify our pipeline and thus create value. We can, in these instances, withhold the drawing-down of convertible 
    bonds which are under the Company's call. 
 
Our Focus on PXT3003 and our Pivotal Phase III Clinical Study in CMT1A, the PREMIER Trial 
We continue to focus a substantial proportion of our resources on developing PXT3003 with the ultimate goal of 
obtaining marketing approval from the FDA and EMA to improve everyday life of patients with CMT1A. Currently, there are 
no approved therapies to treat CMT1A, and PXT3003, which has Orphan Drug Designation from both the FDA and EMA, is the 
most advanced pharmaceutical product candidate in development for this disease. Given there are over 100,000 CMT1A 
patients across the US and EU5 markets, there is little competition in the CMT1A therapeutic field and our current 
understanding of the market, we believe that the worldwide annual peak sales opportunity for PXT3003 could exceed USD1 
billion within 5 years post commercial launch. 
 
We have high hopes of success in our PREMIER trial for several reasons: 
  ? The Phase II and the first double-blind Phase III (PLEO-CMT) studies have shown encouraging and consistent safety 
    and efficacy data. 
  ? As recently announced in April 2021, the interim analysis topline data from the ongoing open-label Phase III 
    Extension Study (PLEO-CMT-FU) suggests sustained benefits for CMT1A patients after 4.5 years of total trial time. 
    Pharnext will continue reporting long-term data from the ongoing extension study on an annual basis. 
  ? The design of the PREMIER trial has the same primary endpoint (ONLS measuring patients' functional motor 
    disability), same patient population (adult mild-to-moderate CMT1A patients) and same PXT3003 formulation (oral 
    solution) as those used in the previous and ongoing Phase III program (PLEO-CMT and PLEO-CMT-FU trials). 
 
The PREMIER trial is ongoing, as planned, following enrollment of the first patient in the US in March 2021 despite the 
COVID-19 pandemic. Ten sites so far have been activated in North America and are actively screening dozens of CMT1A 
patients. Additional sites are planned to be activated in the US and Canada during the summer 2021, and we are still on 
track to announce the activation of the first EU and Israeli sites in Q3 2021. We are confident that we will complete 
enrollment in the PREMIER trial in Q2 2022 as previously announced which will allow our company to disclose topline 
results of the pivotal Phase III study in Q3 2023. 
 
The Future for Pharnext 
We now have the financing in place to fund and complete our pivotal Phase III clinical study of PXT3003, the PREMIER 
trial, which, if successful, will ultimately form the basis of a marketing authorization dossier, together with the 
data from a pre-clinical factorial combination study in the CMT1A rat model, to be submitted to the FDA and the EMA. 
This submission could occur within H1 of 2024 according to the usual regulatory timelines. All this could potentially 
bring a new therapeutic option for CMT1A patients. Being granted marketing authorization in Europe and the US for 
PXT3003 in CMT1A would represent a tremendous value creation opportunity for both patients and our shareholders in the 
coming years, and this is what we are entirely committed to achieving. 
In parallel, we will pursue the evaluation of growth opportunities for our company to potentially enrich our R&D 
pipeline, either internally by advancing drug candidates from our PLEOTHERAPY(TM) platform through clinical 
development; or through R&D partnership and/or deals with other biopharmaceutical companies or renowned academic 
research institutions. 
 
I will keep you informed on the progress of our business plan on a regular basis, and I sincerely thank you again for 
your continued support of our Company. 
 
With Best Regards, 
 
David Horn Solomon 
Chief Executive Officer 
 
 
 
About Charcot-Marie-Tooth Disease Type 1A ('CMT1A') 
Charcot-Marie-Tooth ('CMT') disease encompasses a heterogeneous group of inherited, severe, debilitating, progressive 
and chronic peripheral neuropathies. CMT1A, the most common type of CMT, is an orphan disease with a prevalence of 1/ 
5000 people affecting about 150,000 people in Europe and the U.S. and about 1,500,000 people worldwide. The genetic 
mutation responsible for CMT1A is a duplication of the PMP22 gene coding for a peripheral myelin protein. The 
duplication of this gene results in overexpression of the PMP22 protein and failure of Schwann cells to produce normal 
myelin (neuronal sheath). The lack of a normal myelin structure and function leads to abnormal peripheral nerve 
conduction and axonal loss. As a result of peripheral nerve degradation, patients suffer from progressive muscle 
atrophy in both the legs and arms causing problems with walking, running and balance as well as abnormal hand 
functioning. They might also suffer from mild to moderate sensory disorders. First symptoms usually appear during 
adolescence and will progressively evolve throughout life. Patients with the most severe form of CMT1A end up in 
wheelchairs, representing at least 5% of cases. 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. More 
information can be found at https://pharnext.com/en/disease/charcot-marie-tooth. 
 
 
 
About PXT3003 
PXT3003 is a novel fixed-dose synergistic combination of baclofen, naltrexone and sorbitol formulated as an oral 
solution given twice a day. The three individual components of PXT3003 were selected to downregulate the overexpression 
of PMP22 protein, leading to improvement of neuronal signaling in dysfunctional peripheral nerves that are an essential 
part of the pathophysiology of this disease. PXT3003 could also have a positive effect on other cellular types of the 
motor unit such as the axon (direct protection), neuromuscular junctions or muscle cells. PXT3003 has shown promising 

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