Pharnext : Présentation corporate de Pharnext

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New Therapies for

Neurodegenerative Diseases

CORPORATE PRESENTATION

March 2024

Disclaimer	2

References herein to this presentation (the "Presentation") shall mean and include this document, any oral presentation accompanying this document provided by Pharnext S.C.A. (the "Company") and any further information that may be made available in connection with the subject matter contained herein.

By viewing or receiving or reading this Presentation or attending any meeting where this Presentation is made, you agree to be bound by the limitations, qualifications and restrictions set out below:

This Presentation is confidential, is intended for the recipient only and thus may not be forwarded, reproduced, redistributed or passed to any other person or published in whole or in part for any purpose. If this document has been received in error, it must be returned immediately to the Company. By receiving this Presentation, you become bound by the above-referred confidentiality obligation. Failure to comply with such confidentiality obligation may result in civil, administrative or criminal liabilities.

Recipients of the Presentation should not deal or encourage any other person to deal in the securities of the Company.

This Presentation is not directed to, or intended for distribution to or use by, any person or entity that is a citizen or resident or located in any locality, state, country or other jurisdiction where such distribution, transmission, publication, availability or use would be contrary to law or regulation or which would require any registration or licensing within such jurisdiction.

No securities of the Company have been registered under the U.S. Securities Act of 1933, as amended (the "U.S. Securities Act"), or under any state securities laws, and the securities of the Company may not be offered or sold in the United States (or to, or for the account or benefit of U.S. Persons) except pursuant to an exemption from, or a transaction not subject to, the registration requirements of the U.S. Securities Act. The Company has no current intention to register in the United States any portion of its securities or to conduct a public offering of any of its securities in the United States.

No action has been undertaken or will be undertaken to make an offer to the public of securities requiring a publication of a prospectus in the United Kingdom or any member State of the European Economic Area (each a "Relevant State"). As a result, the securities may not and will not be offered in any Member State, except pursuant to Article 1(4) or Article 3(2) of Regulation (EU) 2017/1129 of June 14, 2017 (the "Prospectus Regulation") or under other circumstances not requiring the Company to publish a prospectus pursuant to the Prospectus Regulation and/or the applicable regulations in such Relevant States. For the purposes of this paragraph, "securities offered to the public" in a given Relevant State means a communication to any persons, in any form and by any means, presenting sufficient information on the terms of the offer and the securities to be offered, so as to enable an investor to decide to purchase or subscribe for those securities, as the same may be varied in that Relevant State.

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All statements in the Presentation other than statements of historical fact are or may be deemed to be forward-looking statements. These forward-looking statements are not guarantees of future performance and involve a number of known and unknown risks and uncertainties. These risks and uncertainties, and other factors, could adversely affect the outcome of the forward- looking statements, and actual results could differ materially from those contemplated in the statements. As a result, you are cautioned not to rely on such forward-looking statements. Forward-looking statements speak only as of the date of this document and the Company expressly disclaims any obligation or undertaking to update or re-issue any forward-looking statements contained in this Presentation.

Especially in the context of the current health crisis, these risks and uncertainties, include among other things, the uncertainties inherent in research and development of new products, including future clinical trial results and analysis of clinical data (including post marketing data), decisions by regulatory authorities, such as the Food and Drug Admnistration or the European Medicines Agency, regarding whether and when to approve any drug, device or biological application that may be filed for any such product and candidates, as well as their decisions regarding labelling and other matters that could affect the availability or commercial potential of such product candidates.

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1. Corporate Overview

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At a Glance…

• Euronext Paris-listed, Paris HQ with operations across France and the US
• R&D efforts concentrated on Charcot-Marie-Tooth Disease type 1A (CMT1A)
  A rare inherited debilitating peripheral neuropathy
• First-in-classlate-stage asset in development for CMT1A
• • PXT3003 (baclofen, naltrexone, sorbitol) with Phase 2, Phase 3 and Pivotal Phase 3 data

Pharnext: Strong Fundamentals for Success	5

PXT3003 - Clinical & Regulatory

Encouraging Efficacy and Safety Profile

Based on Phase IIa, 1st Phase IIIb & Extensionc

and Pivotal Phase III (PREMIER Trial)d studies data

Orphan Drug (US + EU) - Fast track (US) Status

New Legal Structure		Experienced
A Limited Partnership with Shares (French S.C.A.*)		Management Team
Impactful Supervisory Board		Strong Industry and Financial Expertise
	Financial, Medical, Regulatory,
Deep expertise in clinical development, neuroscience,		Manufacturing, Quality Commercial
strategy and bus dev

*Société en Commandite par Actions

PXT3003 - Business Case

Blockbuster Potential (US + EU) - Global rights (ex-China)

Favorable Market Situation

High Unmet Medical Need, No Available Treatment, very Early-Stage Competitor Pipelines

Robust IP Protection and Orphan status

Composition of Matter Patents

aPhase 2:https://clinicaltrials.gov/ct2/show/NCT01401257?term=PXT3003&draw=2&rank=5dPREMIER trial:https://clinicaltrials.gov/ct2/show/NCT04762758?term=PXT3003&draw=2&rank=3

bPLEO-CMTtrial:https://clinicaltrials.gov/ct2/show/NCT02579759?term=PXT3003&draw=2&rank=4

cPLEO-CMT-FUtrial:https://clinicaltrials.gov/ct2/show/NCT03023540?term=PXT3003&draw=2&rank=2

Pharnext New Legal Structure: Stabilized Shareholding and Governance	6

Subsidiary

Pharnext

Developpementa

As General Partner of Pharnext

• • Liable for Pharnext's Debts As General Manager of Pharnext
• Control of Pharnext's Operations
• 
  Supervisory Boardc

Limited

Partnership with

Shares*

Warrants

for 1/3 shares as of January 1st, 2024

Pharnext

Shareholdersb

Own Pharnext shares

• ADVANTAGES

• Ensure Pharnext governance stability
• Protect Pharnext against hostile takeovers
• Facilitate discussion engagement with potential financial, industrial, and pharma partners

*French S.C.A.: Société en Commandite par Actions	ccomposed of Josh Schafer, Jim Kuo and Larry Steinman as of February 28h, 2023
a« commandité » in French
b« commanditaire » in French

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2. PXT3003 in CMT1A

Charcot-Marie-Tooth Disease Type 1A (CMT1A)	8
Chronic, Severe, Debilitating Inherited Peripheral Neuropathy

CMT1A	Most common form of CMT (~50%)
	Chronic, severe, progressively debilitating, inherited neuropathy resulting from
	a duplication of the PMP22 gene, causing demyelination of peripheral nerves
SYMPTOMS	Muscle atrophyin extremities causing severe leg and arm disabilities,
	pain, cramps and fatigue
DIAGNOSIS	Symptoms (e.g. high arched feet), slow motor NCV <38 m/s in upper limbs
	defined CMT type 1 (demyelinating), subtype confirmed by genetic testing
	(gold standard). Average time to diagnosis: 1.5yr
NATURAL	Genetic disease; symptoms starting in teenage years, progressively declining
HISTORY	through life, often requiring braces, surgery and wheelchair (5%)
	Not life threatening but evidence of reduced life expectancy
POPULATION	Rare disease with > 100,000 peopleaffected with mild to moderate CMT1A in
	US and EU5 (core markets)
TREATMENT	No approved drugs; only limited supportive care available
OPTIONS	No other candidates in late-stageclinicaldevelopment (most advanced PhI/II)

Sources: Orphanet; Pharnext market research, Shy M.E. et al. Neurology 2008;70(5):378-83; Bird T.D. Charcot- Marie-Tooth Hereditary Neuropathy Overview, in GeneReviews®, Pagon R.A. et al.

Editors, Seattle (WA), last revision: September 1, 2016; Globaldata, C. DiVincenzo et al. The allelic spectrum of Charcot-Marie-Tooth disease in over 17,000 individuals with neuropathy. Mol Genet Genomic Med. 2014 Nov;2(6):522-9. Vaeth S et al. BMJ Open 2017;7:e018048.

Overall Neuropathy Limitation Scale (ONLS)

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Validated* Scale to Evaluate Functional Motor Disability in CMT1A Patients

* Graham RC & Hughes RAC; J Neurol Neurosurg Psychiatry_2006; 77;973-976

Charcot-Marie-Tooth Disease Type 1A

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Illustrated Natural Long-Term Progression of Disease on ONLS Scale1

ONLS Scale

0

Clinical progression must be considered

over long time periods in CMT1A

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CLINICAL

2

3

4

5

DECLINE

6

9

7

8

10

Teenage*

Young Adult

Senior Adult

Unilateral walking

Leg braces or

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Abnormal gait, minor

support, difficulty using

wheelchair, cannot use

issues with arms

arms for daily tasks

arms for daily tasks

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Y e a r 1

Y e a r 1 0

Y e a r 2 0

Y e a r 3 0

Y e a r 4 0

Progressive slow clinical declining through life*

*Heterogeneity in age of onset, disease progression and disease severity may lead to the under-recognition or late diagnosis of individuals with initially mild or late-onset CMT1A2,3,4,5

Sources: 1Natural progression on ONLS adapted from Shy, et al (2008) + placebo group decline observed in Pharnext Phase II and III studies; 2Rossor A.M. et al. Nat Rev Neurol 2013;9(10):562- 71; 3Li J. Ann Clin Transl Neurol 2017;4(8):601-7;4Li J. et al. Mol Neurobiol 2013;47(2):673-98;5Bienfait H.M. et al. J Neurol 2006;253(12):1572-80.