Corporate PresentationJanuary 2020

DisclaimerReferences herein to this presentation (the “Presentation”) shall mean and include this document, any oral presentation accom panying this document provided by Pharnext SA (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 (as such term is defined herein) or attending any meeting where this Pre sentation 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.This Presentation contains inside information with regard to the Company and/or its securities. Recipients of the Presentation should not deal or encourage any other person to deal in the securities of the Company until the transaction described in thi s Presentation is either announced or abandoned by the Company. Dealing in securities of the Company when in possession of inside information would r esult in liability for breach of insider dealing restrictions under applicable law, including French and U.S. law.

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2

Experienced Leadership Team

Serguei Nabirotchkin, PhD – Chief Biology Officer & Co-

Founder

Xavier Paoli, MSc – Chief Commercial Officer

Daniel Cohen, MD, PhD – Chief Executive Officer & Co-Founder

Viviane Bertrand, PhD – Chief Preclinical Drug Development Officer

Susanne Dorn, MSc – Chief Regulatory Officer

Philippe Rinaudo, PhD – Chief Data Officer

François Chamoun, Master 2 Business Laws (DESS) & DJCE –

Chief Legal Officer

3

Rodolphe Hajj, PhD – Chief Pharmacology Officer

Peter Collum, Chief Financial Officer & Chief Business

Officer

Corporate Overview

PLEOTHERAPYTM Artificial Intelligence (AI) Platform

Maps disease networks and identifies combination drugs to

hit multiple highly relevant disease targets

Our Mission:

• Leverage proprietary

PLEOTHERAPYTM AI

platform to develop

novel combination

therapeutics using both

generic compounds and

NCEs across various Tx

areas

• Continue development

and gain approval for

PXT3003 in CMT1A and

PXT864 in AD

PXT3003 (CMT1A) – Phase 3

PXT864 (AD) – Pre-Phase 2b

PIPELINE COLLABORATIONS

Over 20 additional indications mapped for further pipeline expansion

Tasly (Chinese rights for PXT3003 and joint venture)

Galapagos (taking multiple programs into preclinical testing)

5

Corporate Highlights

5

PLEOTHERAPYTM AI platform scalable across multiple Tx areas

with both generic and NCE combinations

Identifies drug combos for multiple relevant disease targets

Known safety profile of existing drugs

Faster transit through R&D process and POC

Known safety of generics allowing direct access to Phase 2

Phase 3 CMT1A program (~$1Bn WW sales potential)

Top-line results from extension study of PXT3003 in 1Q20

Large Potential

Opportunity

Enhancing

Probability of

Clinical Success

Improved Capital

Efficiency

Advanced Lead

Program: PXT3003

Phase 2a data in AD

Ability to efficiently grow pipeline with PLEOTHERAPY platform

AD Candidate pre-

Ph2b + Pipeline

Potential

PL

AT

FO

RM

PIP

EL

INE

Pipeline and Expected Milestones*

6

PXT3003

PXT864

CMT1A

Adults

CMT1A

Pediatrics

Extension study results – 1Q2020

Second phase 3 trial:

• Planned launch - 2H-2020

• Topline results expected - late 2022 / early 2023

Alzheimer’s

disease

Amyotrophic

lateral sclerosis

Pre-Phase 2b

Product Indication Preclinical Phase 1 Phase 2a Phase 2b Phase 3 Expected Milestone

* Forward-looking statements, see risk factors set out in Pharnext’s document de base registered by the French Financial Markets Authority under number I.16-0050

Launch of Ped Ph3 trial – TBD

Pre-Phase 2a

PXT3003 Overview

7

PXT3003 Highlights

▪ Addresses Charcot Marie Tooth disease Type 1A (CMT1A): unmet need with no approved drugs to treat

chronic and debilitating disease

▪ Data from first Phase 3 study shows strong efficacy signal

▪ PXT3003 not only halted decline, but demonstrated improvement over baseline

▪ Second Phase 3 study to initiate 2H-2020 based on FDA guidance

▪ Revenue potential of approximately $1Bn worldwide

o More than 100,000 potential patients with mild-to-moderate CMT1A (US and EU5)

▪ IP protection through 2030, including composition of matter

▪ US orphan drug exclusivity1

▪ FDA Fast Track Designation

8 1Assuming late 2023 approval and ODE through 2030, generic entry in late 2031.

Charcot-Marie-Tooth Disease Type 1A

SYMPTOMSMuscle atrophy in extremities causing

severe walking and hand disabilities,

pain, cramps and fatigue

DIAGNOSIS~50% of patients have symptoms

before the age of 20, confirmed by genetic

testing

NATURAL

HISTORY

Genetic disease; symptoms starting in

teenage years, slowly declining

through life, resulting in braces,

surgery and wheelchair

POPULATIONMore than 100,000 people affected

with mild to moderate CMT1A in US

and EU5 (core market)

TREATMENT

OPTIONS

No drug approved; only supportive care

available

No other candidates in late stage clinical

development

9

Chronic, Severe, Debilitating Inherited Neuropathy

BACLOFEN

PXT3003 Novel Design and Mechanism of Action

Opioid/ Alcohol dependence

Spasticity

Constipation

CMT1A disease at-a-glance Network analysis Design of PXT3003

Current Indication

Opioid receptors

GABAreceptors

Muscarinic receptors

Current Dose

50mg

120mg

1.4mg

12mg

OPIOIDReceptors

MUSCARINICReceptors

GABAReceptors

420mg

15g

CMT1A - Axonaldysfunction andmuscle loss

NALTREXONE

SORBITOLNormal

10

Pharnext Dose

All Components of PXT3003 Synergistic to Activity in CMT1A

1 10

1 05

1 00

9 5

9 0

1 1 5

1 25

120

1 3 0M yelin atio n

%Im

pro

ve

m e

nt

m y

elin

len

gth

S

S in g le D u o s

* * *

S

P lac eb o B C L +N L X B C L + S R B N L X + S R B P X T3 0 0 3

4

5

6

7G rip s t re n g th a t e n d o f tr ia l

Gri

ps

tre

ng

th(N

)

(ch

an

ge f

rom

ba

se

lin

e)

***

###

#######

#

In CMT1A neurons on myelination in vitro In CMT1A animals in vivo

***p<0.001 vs CMT1A placebo/Control, ANOVA + Dunnett test#p<0.05, ###p<0.001 vs PXT3003, ANOVA + Dunnett test

S: synergy

BCL = Baclofen (GABA receptor)

NTX = Naltrexone (opioid receptor)

SRB = Sorbitol (muscarinic receptor)

11

Robust Phase 2 Results for PXT3003 in CMT1AExploratory multi-center, randomized, double-blind, placebo-controlled Phase 2 study

DOSE 1n=21

DOSE 3n=19

PLACEBOn=19

DOSE 2n=21

Dose Reduction

1/10001/2001/250

1/5001/1001/125

1/1001/201/25

NAL

BAC

SOR

80Mild to

Moderate Patients

• All doses safe and well

tolerated

• Effect achieved at 12 months with Dose 3, which was used to design the Ph3 study

Source: Attarian et al, Orphanet Journal of

Rare Diseases (2014),9:19912

20%

10%

0%

-10%

%IM

PR

OV

EMEN

Tin

ON

LS

DOSE 3

PLACEBO

BASELINE 12 Mths

DOSE 2

DOSE 1

PHASE 2 DURATION: 12 MONTHS

Efficacy and dose-effect demonstrated with Overall Neuropathy Limitation Scale (ONLS)

PLEO-CMT: First Pivotal Phase 3 Study Design and EndpointsInternational, randomized, double-blind, placebo-controlled

Primary endpoint: ONLS after 12 -15 months

▪ ONLS: a 12-point scale evaluating disability

▪ 90% of the patients scored 2-4 (mild-to-moderate)

▪ A 0.3-point ONLS difference vs. placebo was determined to be

meaningful

- Stabilizing or even improving disease versus placebo or natural

yearly evolution estimated at 0.1/0.2 point.

- 50% Placebo standard deviation (Cohen J., 1988)

▪ FDA and EMA agreed on using ONLS as the primary endpoint

for this study.

Secondary endpoints analysis include:

▪ 10-meter walk test (10-MWT)

▪ Nine-hole peg test (9-HPT)

▪ 2 subsets of CMTNSv2 (CMT Impairment Score)

(Clinical + Electrophysiological items = CMTNSv2)

- Sensory subset*

- Clinical subset = purely clinical items (CMTES)**

ONLS = Overall Neuropathy Limitation Scale

* Sensory subset of CMTNSv2: items 1,4 and 5** CMTES is derived from CMTNSv2, items 1 to 7 excluding nerve conductions

13

PHASE 3 DURATION: 15 MONTHS

SOR

0.7 mg 6 mg 210 mg

PLACEBO

DOSE 3(Phase 2

highest dose)

DOSE 4

(2x Dose 3)

NAL

BAC

SOR

323Mild to

Moderate Patients

(age:16 – 65 y)1.4 mg12 mg420 mg

Phase 3 Study – CMC Event Interrupted High Dose ArmStatistical Significance at Interruption with Original Stat Plan

14

Dec 2015

Study Start

Sept 2017

High

dose stop

Mar 2018

Study End

Oct 2017: Variability

Nov 2017: Futility read

Phase 3 Trial n=323 patients

44 completers + 12 dropouts = 56

41 completers + 13 dropouts = 54

42 completers + 12 dropouts = 54

High Dose (Dose 4) n=113

Low Dose (Dose 3) n=109

Placebo n=101

44 completers + 11 dropouts = 55

38 completers + 9 dropouts = 47

85 completers

80 completers

49 completers5 completers

per original stat plan* at CMC event, ONLS p=0.012

modified SAP*, ONLS p=0.008

per original SAP*, ONLS p=0.04

CMC event

July 2017

Germany stop

( all arms)

52 patients – no data

due to CMC event

Oct 2018

Study

Read-out

*Original statistical plan adapted to account for “missing data” from stopping high dose arm due to CMC event (using ICH guidelines)

Statistical model not modified (ANCOVA) but adaptation of populations considered

• Original plan → primary population = FAS (n=323) / missing data due to drop-out (DO) imputed like placebo for all study arms (conservative)

• Modified SAP → primary population = mFAS (n=235 = completers + DO related to treatment) / missing data due to DO related to drug imputed like Placebo for all

study arms / missing data due to all other DO excluded from analysis (considered at random)

PLEO-CMT: ONLS and 10MWT in SAP# Primary Population

# Statistical Analysis Plan frozen and sent to USFDA before unblinding the data

*, ** Dose 4 vs Placebo, ANCOVA with multiple imputation (Missing data implemented by multiple imputations following the placebo trend) *** Average of 12 and 15 Month, or 12 Month if 15 Month is missing15

Dose 4 (n=55)

Dose 3 (n=93)

Placebo (n=87)

p = 0.008

ONLS

p = 0.016

10mWT

Baseline 6 months 12+15 months*** Baseline 6 months 12+15 months***

PLEO-CMT: Safety and Tolerability

▪ Treatment emergent adverse events (TEAE) similar among three groups; majority mild

▪ TEAEs leading to treatment withdrawal similar in all three groups

▪ Single treatment-related serious TEAE occurred in lower dose group (gastroenteritis)

▪ Safe and well tolerated; showed a similar safety profile as in Phase 2

16

Large Commercial Opportunity in CMT1A

▪ Significant unmet need in CMT1A: no pharmacological treatments currently available; only supportive care

options (PT, surgery, pain management)

▪ No other candidates in late stage clinical development

▪ More than 100,000 potential patients in our core market, mild to moderate CMT1A (US and EU5)

▪ 25,000 potential patients already located via patient associations and market research

▪ 5 pricing / market research studies with consistent feedback on US and EU pricing

17

Other Anticipated PXT3003 Milestones and Anticipated Path Forward

Feb 2, 2019: FDA Fast Track Designation granted

Q1 2020: Expect to receive top-line results of extension study (PLEO-CMT-FU) of long-term

safety and efficacy of PXT3003

1H 2020: Expect to finalize second Phase 3 trial protocol in CMT1A with FDA

2H 2020: Expect to Initiate second Phase 3 trial in CMT1A (potential for data in late 2022/early

2023)

2019 / 2020: Continued assessment of commercialization options for geographical areas within

US, EU, Japan and ROW; China commercialization rights are licensed to Pharnext & Tasly’s joint

venture

18

PXT864 Overview

19

Novel AD Approach:Correcting chemical imbalance in the diseased brain

20

Glu GABA

PXT864 to break viciouscircle

Disease at-a-glance

GABAreceptors

Glutamatereceptors

Acamprosate

Baclofen

Healthy brain Diseased brain

Glu: Glutamate

Glu GABA

- +

GABA

Glu

GABA

Glu

Aβ -Tau

Vicious circle occurring in AD brain

(Govindpani 2017; Talantova 2013)

GABA

Glu

“Therapeutics that correct the E/I imbalance in early AD may prevent neuronal dysfunction, cell loss and cognitive impairments associated with later stages of the disease”

E/I imbalance = GLU excitation / GABA inhibition

Upstream of Aβ -Tau imbalanced cells overproduce each

21

Working memory: Combination

% A

lte

rnatio

n

Nor

mal

Con

trol

(Place

bo) AC

PBC

L

PXT864

45

50

55

60

65

70

75

******

S

Acamprosate BaclofenAcamprosate Baclofen

Working memory: Dose-response of single drugs

% A

ltern

ati

on

Nor

mal

A+

Place

bo

A+

ACP 0

.032

A+

ACP 0

.08

A+

ACP 0

.2

A+

ACP 0

.4

A+

ACP 0

.8

A+

ACP 1

.6

A+

ACP 8

.2

A+

ACP 4

1

A+

BCL

0.41

A+

BCL

0.48

A+

BCL

1.2

A+

BCL

2.1

A+

BCL

3

A+

BCL

10.3

45

50

55

60

65

70

75

***

PXT864 Demonstrates Synergistic Efficacy in AD Animals

(Chumakov et al., 2015)

PLEODIAL: Exploratory Phase 2a Trial Design

• 45 mild naïve AD patients treated by 3 doses:

• Clinically diagnosed but low mean Log Abeta 42/40

• 7 centers in France

• Assessed at 0,3,6,9 months

• 9 clinical endpoints, open label, single blind

• Biomarker: Plasma Aβ42/40 assessed by Quanterix

Memory Orientation Language Attention Visuospatial Executive function speed Daily activity Social interaction

Adas Cog

CDRSB

IADL

TMT A

TMT B

ZAZZO

Apathy Inventory

DSST

ISAAC

Functions assessed by each endpoint

22

p = 3e-13 Highest drug exposure versus historical placebo

p = 0.013 All patients versus historical placebo

PXT864: CDR-SB Analysis Based on Plasma Drug ExposureHigher Dose Could Rapidly Generate Partial Recovery Vs Less Decline With No Safety Concerns

***

***

*

*

Acamprosate Baclofen

Approved dose 2000 mg 80 mg

Ingested dose 3 40 mg 24 mg

Next dose to be tested 400 mg 24 mg

Imp

rove

me

nt

23

Biogen EMERGE : 9 months high dose Aducanumab: ̴ 0,7 pts decline

Both analyses with patients with sufficient exposure (increasing dose limited by safety concerns)

Biogen ENGAGE : 9 months high dose Aducanumab: ̴ 0,5 pts decline

Plasma Aβ42/40 Analysis Based on Plasma Drug ExposureImprovement at 3 and 6 Months, but a Higher Dose Could Rapidly Generate Sustained Effect

Drug exposure = Cmax for both drugs

(n=45)(n=10)(n=10)(n=25)

Imp

rove

me

nt

24

Plasma Aβ42/40 3-Month Improvement Correlates With Clinical Improvement at 9 Months:

Suggests a Delayed Effect “From Molecular To Clinical”

25

Biomarker 3-Month improvement Biomarker 3-Month improvement

3-M

on

thC

DR

-SB

imp

rove

men

t

3-M

on

thC

om

po

site

sco

re (

CS)

* im

pro

vem

ent

9-M

on

thC

DR

-SB

imp

rove

men

t

9-M

on

thC

om

po

site

sco

re (

CS)

im

pro

vem

en

t

* Composite score of all clinical endpoints.

PXT864 in Alzheimer’s Disease Overview

Higher doses of PXT864 have potential to demonstrate a sustained therapeutic effect on

Alzheimer’s Disease, due to the following advantages:

▪ Strong safety profile

▪ Can be co-administered with already approved drugs in AD

▪ Can be synergistic with other NCEs to create a powerful novel new entity

26

PLEOTHERAPYTM AI Platform Overview

27

PLEOTHERAPYTM Universal R&D PlatformStarting with Big Data

In Silico AI & Expert System

Disease

Network

Knowledge

Integration

Virtual

Repositioning

Preclinical

2,000+

approved drugs50

candidate drugs(filtered for PK, toxicity, safety, IP)

6 months ≈ 2 years

1

Pleodrug™

In Vitro

Screening

Clinical

≈ 7 years

Phase 1(not always mandatory)

Phase 2A/B

Phase 3

In Vivo

Test

ApprovalDisease

25 Positive Drugs

4 synergistic combos

28

Human geneticsGWAS ( Genome Wide Association Study )

Virtual Repositioning Step 1 Disease Associated Molecular Network

A Fraction of the Whole Interactome

BIG DATA

29

Genome Wide Association Studies(GWAS) e.g. from AD patients

Protein – Protein Interaction

Known Drug-Targetrelationships

Literature

EXPERTS AI

e.g. AD Associated Molecular Network

Virtual Repositioning Step 2 Identifying Candidate Drugs from Drug Data Base and Disease Associated Molecular Network

BIG DATA

30

Genome Wide Association Studies(GWAS) e.g. from AD patients

Protein – Protein Interaction

Literature

EXPERTS AI

e.g. AD Associated Molecular Network

Known Drug-Targetrelationships

Candidate drugs Experimental

Testing

RALBP1

metabolite

metabolite

metabolite

metabolite

metaboliteGRM 5

mTOR

metabolitetransport

metabolite

metabolite

PITPNC1

PITPNC1

SNCA

PLD 1

metabolite

TH EM 2

metabolite

metabolite

ACH E

metabolitePLD 1/2

PLD 2

NFĸB1

metabolite

metabolite

metabolite

metabolite

SGPP2

PPARγ

PD GF

metaboliteeffect

PLA

?

AD CY2

D RD 2

ANXA3

D RD 5

metabolite

ASAH 1

metabolite

metabolite

metabolite

PRIM A1

metabolite

metabolite

PLAUST14

Virtual Repositioning Step 1 Example of Alzheimer’s Disease – Associated Sub Network

metabolite

Proteins

encoded by

genes with

statistical

association

with

Alzheimer’s

Disease

Proteins

encoded by

genes without

statistical

association

with

Alzheimer’s

Disease

32

RALBP1

metabolite

metabolite

metabolite

metabolite

metaboliteGRM 5

mTOR

metabolitetransport

metabolite

metabolite

PITPNC1

PITPNC1

SNCA

PLD 1

metabolite

TH EM 2

metabolite

metabolite

ACH E

metabolitePLD 1/2

PLD 2

NFĸB1

metabolite

metabolite

metabolite

metabolite

SGPP2

PD GF

metaboliteeffect

?

ANXA3

D RD 5

metabolite

ASAH 1

metabolite

metabolite

metabolite

PRIM A1

metabolite

metabolite

PLAUST14

metabolite

Genetic association

Drugged Target

Virtual Repositioning Step 2 Crossing Drug Data Base And Alzheimer’s Disease – Associated Sub Network

Candidate drugs ready for experimental screening

33

AI

con

tin

uu

m

Human (computer assisted)Computer (human assisted)

2007

2007

33

2021

2021

2017

2017

Other omics

Phase 2017-2021 (AI-assisted platform):

1. New generation GWAS module: machine learning &

deep learning-based imputation (by end 2019) +

disease risk prediction (projected 2020)

2. Other Omics data (Big Data)

3. Network building powered by AI, learned from

Pharnext’s existing networks and methods

4. Network analysis powered by AI (ex: synergy

prediction)

• And other ML projects …Network construction

module

DiseaseNetworks

Genetic Data

(GWAS)

GWAS analysis module

LiteraturePatents

Other databases (proteins, pathways,

other PPI, etc.)

IngenuityReaxys

databases

Experts*2

* 1

* 3

* new

ML tools

* 4

AI assisted

Virtual Repositioning Powered by AI and Big Data with New Machine

Learning (ML) ToolsReduced from 1 year to 1 quarter now, aiming to reduce to a few weeks

5-year plan for automation 34

BACLOFEN

Discovery of PXT3003

57 Drugs selected to screen

2,000+ drugs

Virtual screening

Down-regulation of Pmp22 in

Schwann cells 22 positive drugs (39%)

3 prioritized, 1 selected

NALTREXONE

SORBITOL

In CMT1A, excess Pmp22

protein is produced, leading

to instability and loss of

myelin

34

35

33 Drugs selected to screen

2,000+ drugs

Virtual screening

Protection of:

- Neurons

- Synapses

- Vessels25 positivedrugs (76%)

91 combinations tested

In AD, excess of β-Amyloid

is produced, leading to

neuronal/glial dysfunction

and cell death

Discovery of PXT864

Acamprosate Baclofen

Aβ-induced toxicity

32 positivecombos

4 combos prioritized, 1 selected

Summary Corporate Highlights

▪ Pharnext represents an opportunity with a combination of an AI-based drug development platform with an advanced

lead program with Phase 3 data and potential for FDA approval by late 2023

▪ Three distinct value drivers – PXT3003, PXT864 and PLEOTHERAPYTM AI Platform

• PXT3003 in Phase 3 for CMT1A

- Phase 3 data showing strong efficacy

- ~$1B WW potential revenue opportunity / IP through 2030

• PXT864 in Phase 2 for AD – strategy to out-license to commercial partner

- Novel mechanism, upstream of Aβ and tau mechanism approaches

- Subset with high drug plasma concentration showed improved CDR-SB above baseline for mild AD

patients

• PLEOTHERAPYTM AI platform – potential to produce pipeline across all therapeutic areas

- Approximately two years from start to initiation of human POC studies

36