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2

Highlights

▪ Pharnext, based on the positive pivotal Phase 3, intends to file its NDA formarketingapproval in 2019 in the US and Europe for a potential launch in 2020

▪ CMT1A patients have no approved drug to treat their chronic neuropathy and Pharnext

PXT3003 could be the first therapy aimed at treating this severe debilitating disease

▪ PXT3003 has blockbuster potential

▪ Strong IP portfolio including composition of matter through 2030 (without extensions) for

PXT3003

▪ Having succeeded Phase 3 with its first drug candidate coming from its powerful

Pleotherapy platform, Pharnext is committed to developing additional product candidates

for other major unmet medical needs

3

4

Daniel Cohen, MD, PhDCo-founder, Chief Executive Officer

Serge Fitoussi, MDChief Medical Officer

Rodolphe Hajj, Ph.D.Chief Pharmacology Officer

Serguei Nabirotchkin, PhDChief Biology Officer & Co-Founder

Xavier PaoliChief Commercial Officer

Amit KohliChief Operating Officer

Experienced leadership team

PleotherapyTM Universal R&D PlatformStarting with Big Data, High-Science & Analytics

In Silico Proprietary AI & ExpertSystem

Disease

Network

Knowledge

Integration

Virtual

Screening

Preclinical

> 2,000

approved drugs

50

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

6-12 mo ≈ 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

5

Pipeline and Expected Milestones

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

PXT3003

PXT864

CMT1A

Adults

CMT1A

Pediatrics

NDA & MAA submission

in H2 2019

Launch of Phase 3

in H1 2019

Alzheimer’s

disease

Amyotrophic

lateral sclerosis

Phase 2b initiation

expected in 2019

POC & Phase 2a initiation

expected in 2019

6

Charcot-Marie-Tooth Disease Type 1A

SYMPTOMSMuscle atrophyin extremities which causes

severe walking and hand disabilities, pain,

cramps and fatigue

DIAGNOSIS About 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

POPULATION~100,000 people affected with mild to

moderate CMT1A in US and EU5

TREATMENT

OPTIONS

No drug approved

Only supportive care available

There is no stabilizing treatment

BARRIERS

TO ENTRYRobust IP including composition of matter until at

least 2030; orphan drug status in the US and EU

7

Chronic, Severe, Debilitating Inherited Neuropathy

Discovery of PXT3003

57 Drugsselectedto screen

2,000+ drugs

Virtual screening

Down-regulation of Pmp22 in

Schwann cells 22 positive drugs (39%)

3 prioritized

NALTREXONE

BACLOFEN

SORBITOL

In CMT1A, excess Pmp22

protein is produced, leading

to instability and loss of

myelin

8

PXT3003 Design and Mechanism of Action

Opioid/ Alcohol dependence

Spasticity

Constipation

Current Indication

Opioid receptor

GABAreceptor

Muscarinic receptor

Dose Reduction

50mg

120mg

1.4mg

12mg

OPIOIDReceptor

MUSCARINICReceptor

GABAReceptor

420mg

15g

Preclinical data demonstrate that PXT3003 acts on different cell types of the motor unit in CMT1A

Network analysis Design of PXT3003

CMT1A - Axonaldysfunction andmuscle loss

NALTREXONE

BACLOFEN

SORBITOLNormal

9

Mechanism of action of PXT3003 in CMT1A

Increases axonal diameter and

number of myelinated axons

Downregulates PMP22 Improves Akt/Erk signaling dysbalance

Schwann cell differentiation

*p<0.05 vs TG vehicle placebo

10

Mechanism of action of PXT3003 in CMT1A

11 Very fast fibers in red

Improves muscle function

Restores functional Neuromuscular junctions

Ve h ve h P XT 3 0 0 3

7 0

8 0

9 0

1 0 0

% I

nn

erv

ate

d N

MJ **

C M T 1 A R a tsW T

**

TG high dose

WT placebo TG placebo

All components of PXT3003 contribute to its activity in CMT1A

110

105

100

9 5

9 0

115

125

120

130M 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 la ce b 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 tre 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/vehicle, Dunnett test S: synergy$p<0.05, $$$p<0.001 vs PXT3003, Dunnett test

BCL = Baclofen (GABA receptor)

NTX = Naltrexone (opioid receptor)

SRB = Sorbitol (muscarinic receptor)

12

Robust Exploratory Phase 2 Results for PXT3003 in CMT1A

Abbreviations: ONLS, Overall Neuropathy Limitations Scale.

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

endpoint in a multi-center, randomized, double-blind, placebo-controlled Phase 2 study

PHASE 2 DURATION: 12 MONTHS

LOWDOSE

n=21

HIGHDOSE

n=19

PLACEBO

n=19

INTERMEDIATEDOSE

n=21

Dose Reduction

1/10001/2001/250

1/5001/1001/125

1/1001/201/25 20%

10%

0%

-10%

%IM

PR

OV

EMEN

Tin

Ove

rall

Neu

rop

ath

y Li

mit

atio

nSc

ale

INTERMEDIATE

HIGH

LOW

PLACEBO

BASELINE 12 Mths

NAL

BAC

SOR

80Mild to

Moderate Patients

• All doses safe and well tolerated

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

Source: Attarian et al, OrphanetJournal of Rare Diseases (2014), 9:199

13

Phase 2A Trial of PXT3003 in CMT1A:Promising & Significant Efficacy Results

Imp

rove

men

tHigh dose Significant

improvement

vs placebo (p = 0.007)

vs baseline (p = 0.0006)

Intermediate dose

Positive efficacy results on composite z-score*

Placebo

Low dose

Significant dose effect(p = 0.003)

*Z-score summing all objective measures: clinical measures (CMTES , ONLS , PEG test, walk test, QMT) and electrophysiology variables.

Abbreviations: LD, Low Dose; ID, Intermediate Dose; HD, High Dose; PL, Placebo.

14

PLEO-CMT: Pivotal Phase 3 Study Design and Endpoints

International, multi-center, randomized, double-blind, placebo-

controlled pivotal Phase 3 study

Primary endpoint: Disability measured by the change in ONLS

scale in CMT1A patients treated for 12 to 15 months

▪ ONLS is a 12-point scale evaluating disability

▪ 90% of the patients in the study had a score between 2-4

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

meaningful (Cohen J., 1988)

▪ FDA and EMA agreed to use ONLS as the primary endpoint for

this study

Secondary endpoints analysis include:

▪ 10 meter walk test (10MWT)

▪ Nine-hole peg test performed by non-dominant hand

▪ CMT Impairment Score (Clinical and Electrophysiological =

CMTNSv2)

- sensory items*

- 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

PHASE 3 DURATION: 15 MONTHS

SOR

0.7 mg 6 mg 210 mg

PLACEBO

Phase 2HIGH DOSE

2x Phase 2 HIGH DOSE

NAL

BAC

SOR

323Mild to

Moderate Patients

(age:16 – 65 y)1.4 mg12 mg420 mg

15

PLEO-CMT: Course of the Study

▪ Higher dose discontinuation for unexpected formulation / stability issues without safety concern

▪ Higher dose patients continued to openlabel extension study with 2x volume oflower dose

▪ Only blind data were analyzed

LD: Lower Dose

HD: Higher Dose

16

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

Multiple statistical methodologies supported these compelling positive results* Statistical Analysis Plan frozen and sent to USFDA before unblinding the data** Higher dose vs Placebo, ANCOVA with multiple imputation*** Average of 12 and 15 Month, or 12 Month if 15 Month is missing

n = 55 n = 93 n = 87

**p = 0.008 **p = 0.016

17

PLEO-CMT: Ranked individual ONLS change from baseline

▪

higher dose

placebo

IMPROVERS

31% (OR=2,1)

18%

▪ DECLINERS

higher dose 14%

placebo 23% (OR=1,8)

▪ PXT3003 higher dose generatesBOTH

- Less decline

- Improvement

31% on drug improved

18% on placebo improved

Higher

14% on drug worsened

23% on placebo worsened

18

PLEO-CMT: Safety and Tolerability

▪ Treatment emergent adverse events (TEAE) were similar amongst the three groups and the majority were mild

▪ TEAEs leading to treatment withdrawal were similar in all three groups

▪ A single serious TEAE leading to treatment withdrawal occurred in the lower dose group

(benin thyroïd adenoma)

▪ PXT3003 was safe and well tolerated and showed a similar safety profile as seen in Phase 2

19

Path Forward

Q1 2019: Type B meeting request with FDA

1H 2019: Initiation of Phase 3 pediatric study of PXT3003 in CMT1A

2H 2019: Results of extension study (PLEO-CMT-FU) of the long-term safety and

tolerability of PXT3003

2H 2019: Submission of New Drug Application (NDA) to FDA and Marketing

Authorization Application (MAA) to EMA

2020: Commercial launch

20

Large Market Opportunity

Significant unmet need: no pharmacological treatments

currently available for CMT1A; only supportive care options

~100,000 mild-to-moderate CMT1A patients in US and Europe

Initial independent pricing studies show blockbuster potential

58,000 42,000 100,000

TOTAL

+ =

EU5

21

Launch Plan

Commercial Rights

• US, EU, Japan and ROW commercial rights retained by Pharnext

• China rights licensed to Pharnext & Tasly’s joint venture

Commercial Strategy

• With these Phase 3 results, Pharnext is strongly committed to prepare filing in Europe

and the US

• Pharnext will assess the most adequate commercialization options for various

geographical areas

22

Other Facts

• IP: composition of matter granted (and orphan drug exclusivity)

• Regulatory: No need for factorial design in patients

• Pricing

• Compounding

23

Summary

▪ Pharnext, based on the positive pivotal Phase 3, intends to file its NDA formarketingapproval in 2019 in the US and Europe for a potential launch in 2020

▪ CMT1A patients have no drug to treat their chronic neuropathy and PharnextPXT3003

could be the first approved therapy aimed at treating this severe debilitating disease

▪ PXT3003 has blockbuster potential

▪ Having succeeded Phase 3 with its first drug candidate coming from its powerful

Pleotherapy platform, Pharnext is committed to developing additional product candidates

for other major unmet medical needs

24