Corporate Presentation January 2017

2

Disclaimer

References herein to this presentation (the “Presentation”) shall mean and include this document, any oral presentation accompanying this document provided by Pharnext (the "Company") and any further information that may be made available in connection with the subject matter contained herein. This Presentation has been prepared by the Company and is provided for information purposes only. This document does not purport to contain comprehensive or complete information about the Company and is qualified in its entirety by the business, financial and other information that the Company is required to publish in accordance with the rules, regulations and practices applicable to companies listed on the Alternext Market of Euronext Paris, including, in particular, the risk factors set out in the Company’s document de base registered by the French Financial Markets Authority (Autorité des marchés financiers) on June 2, 2016 under number I.16- 050. Information and other data appearing in such publications, and certain figures and numbers appearing in this document have been rounded. Consequently, the total amounts and percentages appearing in tables and elsewhere may not necessarily equal the sum of the individually rounded figures, amounts or percentages. No representation, warranty or undertaking, express or implied, is made as to the accuracy, completeness or appropriateness of the information and opinions contained in this Presentation, or its use for any purpose, and no reliance should be placed on any information or opinions contained herein. The Company, its subsidiaries and representatives accept no responsibility for and shall not, under any circumstance, be held liable for any loss or damage that may arise from the use of this document or the information or opinions contained in it. In particular, this document contains information on the Company’s markets and competitive position, and more specifically, on the size of its markets. This information has been drawn from various sources or from the Company’s own estimates which may not be accurate and thus no reliance should be placed on such information. The information and opinions contained in this document are provided as of the date of this document only and may be updated, supplemented, revised or amended, and thus such information is subject to change at any time. The Company is not under any obligation to update the information, statements or opinions contained in this Presentation. 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. This Presentation does not constitute or form any part of any offer to sell, or the solicitation of an offer to buy or subscribe for, any shares or securities in the Company, in the United States or in any other jurisdiction. All persons accessing this document are deemed to agree to all the limitations and restrictions set out above.

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Company Overview

• Public biopharmaceutical company (ALPHA) leader in synergic combinatorial medicine

• Late-stage pipeline focused on neurodegenerative diseases (orphan & common) with 2 clinical-stage products respectively in Phase 2 and Phase 3 clinical trials

‒ PXT3003 in pivotal Phase 3 for treatment of an orphan peripheral neuropathy: Charcot-Marie-Tooth Disease Type 1A (CMT1A)

‒ PXT864 with a novel MoA in Phase 2 for treatment of Alzheimer’s Disease and with high potential in other major neurodegenerative indications such as Parkinson’s and ALS

• Universal R&D platform: PLEOTHERAPY® to identify and develop synergic combinations of repositioned drugs at low doses

‒ More efficient, safer, faster and affordable drug development process

‒ Applicable to:

• Any disease

• Any compound in ongoing R&D development for multiple indications

• Founded and led by a deeply experienced management team with the support of exceptional science and clinical advisors

4

Robust Late-Stage Pleotherapy Development Pipeline

PRODUCT INDICATION PRECLINICAL PHASE 1 PHASE 2a PHASE 2b PHASE 3

EXPECTED NEXT EVENT

PXT3003

CMT1A Adults

Top-line results expected in Q2 2018

CMT1A Children

Other peripheral neuropathies

PXT864

Alzheimer’s Disease

Launch of Phase 2b in H2 2017 – Results expected in 2019/2020

Parkinson’s Disease

Amyotrophic Lateral Sclerosisb

Note (a): The CMT program went through Phase 2 directly after the preclinical results. Phase 1 was not required by French health authorities (ANSM) Note (b): Amyotrophic Lateral Sclerosis is also called Charcot Disease *9th Clinical Trials on Alzheimer's Disease (CTAD) Conference on December 8-10, 2016 in San Diego, CA

(no Phase 1 required)a

Results of Phase 2a

presented at CTAD*

PHASE OF DEVELOPMENT

PXT3003

6

Characteristics / Symptoms

Chronic, severe, debilitating inherited neuropathy leading to muscle atrophy in extremities: walking and hand disabilities up to handicap

Population ~100,000 people affected with mild to moderate CMT1A in US and EU

Treatment Options No drug approved or in clinical development, only supportive care

Outcome In most patients, disease is stabilized while a fair proportion is even improved

Barriers to entry In addition to robust IP until at least 2030, orphan drug status in US and EU5 provides market exclusivity

Expected Peak Sales

Approx. > $ 1 billion (US +EU5)

Overview of Charcot Marie Tooth Disease Type 1A

7

Design of PXT3003

GABA receptor Baclofen

Sorbitol

Naltrexone Opioid receptor

Muscarinic receptor

Disease at a glance

CMT 1A is due to overexpression of PMP22 protein in Schwann cells that are impaired

Cell Body

Myelin Sheath

Axon

Basement membrane

Normal Death

Myelin Impairment

Suffering

Muscle

Schwann cells constitute myelin which isolates and nurtures axons

Myelin Sheath

Schwann cell

Targeting the Cause of the Disease with PXT3003

Network analysis

Muscarinic receptor

The CMT network

Lowers expression of PMP22

gene

Improves myelination

Preserves axons

Improves motor

function

GABA receptor

1

2

3

4

Opioid receptor

8

PXT3003 Significantly Reduces PMP22 Gene Expression

In vitro PMP22 gene expression in Schwannoma cells treated with PXT3003 and its single components for 8 hours (Chumakov et al., 2014)

Re

lati

ve P

MP

22

to

Mp

z m

RN

A

Exp

ress

ion

leve

l (%

of

con

tro

l)

60

70

80

90

100

Control Baclofen100 nM

Naltrexone100 nM

Sorbitol10 µM

PXT3003

PMP22 Expression

9

0 2 0 4 0 6 0 8 0

H o t p la te

In c lin e d p la n e

B a r te s t

G rip s t re n g th

N u m b e r o f to ta l m y e lin a te d a x o n s

M o to r n e rve c o n d u c t io n ve lo c it y

C o m p o u n d m u s c le a c t io n p o te n t ia l

P M P 2 2 e x p re s s io n

% Im p ro v e m e n t

*

*

* *

* *

*

*

Non-clinical endpoints

Clinical endpoints

PXT3003 Shows Outstanding and Consistent Improvement in Peripheral Neuropathies

% re

co

ve

ry

o

f s

urfa

ce

b

ea

rin

g p

aw

s

N o r m a l C o n tr o l

(p la c e b o )

B C L N T X S R B P X T -3 0 0 3

0

2 0

4 0

6 0

8 0

1 0 0

1 2 0

6 8 %

*

* *

n s

C ru s h e d

CMT1A Rat Model Mouse Sciatic Nerve Crush Model

BCL: Baclofen NTX: Naltrexone SRB: Sorbitol

*CMAP : Compound Muscle Action Potential **MNCV : Motor Nerve Conduction Velocity

68%

In vivo efficacy of PXT3003 in the PMP22 rat adult model of CMT1A, treated for 2-5 months (Chumakov et al., 2014)

In vivo efficacy of PXT3003 in the nerve crush mouse model. Mice were treated for 13 days after nerve crush (Chumakov et al., 2014) * P < 0.05 vs. placebo ns: not significant

*

**

10

4-arm study

Low dose

n=21

Intermediate

Dose

n=21

High Dose

n=19

Placebo

n=19

80 mild-to-

moderate adult

patients with

CMT1A

• 12-month treatment

• 6 reference centers in France

• Placebo-controlled, randomized and double-blind

• Oral liquid formulation given twice daily

Assessment Measures

• PXT3003: Naltrexone + Baclofen + Sorbitol

• Efficacy

• Safety and tolerability

• 11 endpoints including Overall Neuropathy Limitation Scale (ONLS)*

• No Phase 1 needed

Phase 2a Trial Design

*ONLS is a scale for doctor investigation assessing disability

Baclofen

Naltrexone

1/1000

1/200

1/250

1/500

1/100

1/125

1/100

1/20

1/25

• Same fixed ratio of 3 different doses ranging from 1/1,000 to 1/20 of approved drugs

Dosing

Phase 2a Trial Overview

Sorbitol

Naltrexone

Baclofen

11

PXT-3003: Positive Phase 2 Results in Adults

• 2 composite clinical scores: ONLS (disability) and CMTNS (impairment)

• 4 individual functional measures: walk (6MWT), ankle flexion, hand grip, finger agility (9HPT)

• 5 electrophysiological measures assessing state of Myelin (MCV, SCV, DML) or Axons (CMAP, SNAP)

Note: ITT analysis. ONLS = Overall Neuropathy Limitations Scale; CMTNS = CMT Neuropathy Scale; 6MWT = 6 meter walk test; 9HPT = 9-Hole Peg Test; MCV = Motor Conduction Velocity; SCV = Sensory Conduction Velocity; DML = Distal Motor Latency; CMAP = Compound Muscle Action Potential; SNAP = Sensory Nerve Action Potential.

As expected from already

approved drugs used at

low doses, PXT-3003 was confirmed safe

p = 0.05

Dose-Dependent Clinical Efficacy at 12 Months

Low Dose vs. Placebo Intermediate Dose vs. Placebo High Dose vs. Placebo

Broad Efficacy In Relevant Set Of Endpoints

12

Promising and Significant Phase 2a Efficacy Results

• Improvement on ONLS recommended by FDA/EMA as primary endpoint for Phase 3

• ONLS improvement sufficient by FDA/EMA for approval if reproduced in Phase 3

• 18.6% improvement vs. placebo (p=0.04) in ONLS ( 0,4 point ONLS)

• FDA / EMA consider that 0,3 point ONLS improvement / Placebo is enough for approval ( Cohen’s Factor)

Main Efficacy Criteria (ONLS)

PHD M12 vs. Plb M12 <0.05 PDose Effect <0.01 PHD M12 vs. Baseline M12 = 0.06

13

PXT3003 Promotes Improvement, Not Just Stabilization

9 6 4

Declining (21%)

7 3 9

Declining (48%)

PXT3003 n = 19

Placebo n = 19

p = 0.047

Treated / placebo Patients ranked by

size of change, then placed side

by side

1 2 3 4 5 6 7 8 9 10 12 14 16 18

Over one year, disease may change (improving or declining) or stay stable PXT3003 lowers the number of declining patients

PXT3003 produces more improvement or less decline in patients

Improving patients

Declining patients

n = 4

% c

han

ge

p = 0.008*

improving

stable

declining

Response to treatment based on average ONLS / CMTNS at 12 Months

n = 9

n = 4

n = 9

11 13 15 17 19

• PP Z-score high dose vs. baseline at 12 months p<0.01

14

Ongoing Phase 3 Trial in U.S., Canada and Europe Now Fully Enrolled

Placebo

323 mild-to-

moderate

patients

(age >16)

with CMT1A and

PMP22 gene

duplication

Phase 3 Trial Design

Endpoints defined with FDA & EMA

• Primary endpoint: change in ONLS

• Secondary endpoints: clinical, functional, electrophysiological and quality of life

Phase 2

high dose

0.7 mg

6 mg

210 mg

2 x Phase 2

high dose

1.4 mg

12 mg

420 mg

Baclofen Sorbitol

Naltrexone

Baclofen

• Pivotal, randomized (1:1:1), double blind, placebo-controlled, three arms

• International study in 30 centers across the US, EU and Canada

• 15-month double blind + 9-month extension study

• Formulation: oral liquid solution given twice a day

• Key milestones:

‒ Adaptive design analysis at 80 patients in Q4 2017

‒ Futility analysis at 100 patients to be completed in Q4 2017

‒ Top-line results expected in Q2 2018

‒ Potential market approval in 2019 - first FDA exemption from single drug testing in man in a combination therapy

PXT864

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Characteristics / Symptoms

Irreversible, progressive neurodegeneration leading to a state of dementia

Population

1 in 8 people 65+ suffer from Alzheimer’s disease

~100 million cases expected by 2050 unless effective treatments discovered

Seventh leading cause of death in USA

Treatment Options Poorly efficient on symptoms with frequent side effects and tolerability issues

Outcome Any better symptomatic relief or disease-modifying effects would be a success

Barriers to entry IP including composition of matter patents for PXT864

Expected Peak Sales

An efficient treatment could be a $5-$20 billion market opportunity

Significant co-prescription opportunities with potentially competitive treatments

Alzheimer’s Disease Overview

17

New MoA: Targeting to Restore a Disrupted Excitatory-Inhibitory Balance

Disease at a glance

Glutamate and GABA are the major excitatory and inhibitory neurotransmitters of the brain

Glu GABA GABA

Glu

Healthy brain Alzheimer’s brain

No Aβ Aβ

Increased Aβ toxicity and level

Disrupted Excitatory- Inhibitory balance

Glutamate neuron

GABA neuron

Design of PXT864

Breaking the vicious circle

GABA receptor Baclofen

Acamprosate Glutamate receptor

Increased Aβ toxicity and level

Disrupted Excitatory- Inhibitory balance

Network analysis implicates glutamate/GABA imbalance

in Alzheimer’s disease

GABA receptor

Glutamate receptor

Network analysis

18

Poly-Efficacy of PXT864

Protects vessels against Aβ toxicity

Preserves synaptic integrity

Protects neurons against Aβ toxicity

Synergistic with standard of care

Decreases inflammation

Pharnext Pleodrug PXT864

Baclofen

Acamprosate

Reduces Aβ levels

Alleviates cognitive deficits in Alzheimer animals

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PXT864 Alleviates Cognitive Deficits in AD Animals

PXT864 synergistically restores memory performance to control levels in Aβ

intoxicated mice

PXT864 improves learning ability in amyloid precursor protein transgenic mice – a mouse

model of Alzheimer’s

In vivo efficacy of PXT864 in mice intoxicated intracerebroventricularily with Aβ, treated for 7 days (Chumakov et al., 2015). PXT864/day: 0.16 mg/kg ACP + 2.4 mg/kg BCL *** P < 0.001 vs. placebo S: synergistic.

In vivo efficacy of PXT864 in APP transgenic mice over producing Aβ, treated after the onset of the disease for 4 weeks (Chumakov et al., 2015). PXT864/day: 0.4 mg/kg ACP + 6 mg/kg BCL * P < 0.05 vs. transgenic mice (Tg) ¤ P < 0.05 global significance of treated group vs. Tg nTg: non-transgenic control mice

20

PXT864 Synergistic with SoC Aricept (DNPZ) in AD Animals

20

20

Drug /day Dose (mg/kg) HED (mg/day)

ACP 0.064 0.32

BCL 0.96 4.8

DNPZ 0.25 1.25

In vivo efficacy of PXT864+Aricept in mice intoxicated intracerebroventricularily with Aβ, treated for 9 days *** P < 0.001 vs. placebo ACP: acamprosate; BCL: baclofen; DNPZ: donepezil (Aricept) S: synergistic.

Y-maze

% A

ltern

ation

Nor

mal

Con

trol

(place

bo)

ACP+B

CL

ACP+D

NPZ

BCL+

DNPZ

Tri-The

rapy

45

50

55

60

65

70

75 *** ***

S

A

P a s s iv e A v o id a n c e :

S te p th ro u g h la te n c y

La

ten

cy

(s

)

Norm

al

Contr

ol

(pla

cebo)

AC

P+B

CL

AC

P+D

NP

Z

BC

L+D

NP

Z

Tr i-T

hera

py

1 0 0

1 5 0

2 0 0

2 5 0*** ***

S

A

S S

21

Month 1 Month 2 Month 3

placebo

challenge de-challenge re-challenge

T PXT864 PXT864 PXT864 +/- Aricept

Month 3 to 9

PLEODIAL I PLEODIAL II

Outcome

• Phase 2a trial completed end 2015

• 45 patients with mild Alzheimer’s Disease

• 5 French memory centers

• Single-blind crossover study

• 12-week duration

• 6-month follow-up for safety

• 3 doses tested

• Formulation: pill given twice daily

Design

• Significant improvement in cognitive function measured by ADAS-cog during active treatment

Challenge De-challenge Re-challenge

Highly Promising Phase 2a Results Presented at 2016 CTAD Meeting

Combine with Aricept

22

Networks suggest similar imbalance in Parkinson’s and ALS

Publication of Preclinical Results in Parkinson’s in Nature Scientific Reports (2015)

PXT864 could be developed in Parkinson’s disease and Amyotrophic Lateral Sclerosis (ALS)

Potential in Other Neurodegenerative Diseases

Pleotherapy Platform

24

Lower than combinations EFFICACY (COMBINATION)

More responders and higher response per responder

Difficult to predict SAFETY Likely higher (already approved drugs at low dose)

15 years / high attrition rate DEVELOPMENT RISK /TIME

Low attrition rate because of higher safety & efficacy/10 years

Composition of matter on a new molecule

IP Composition of matter on new combinations of off-patent drugs

High / challenged TARGET PRICE Lower / affordable

PHARNEXT APPROACH

MONOTHERAPY Monomolecule

PLEOTHERAPY Synergistic combination of repositioned drugs at low dose

CLASSICAL APPROACH

Novel Multi-Target Approach to Address Diseases with No Treatments

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We Build Map of the Disease Network and Identify the Best Lower Dose Combinations to Treat the Disease

Drug A

Monotherapy

One drug at high dose for one therapeutic target

Pleotherapy

Synergistic low-dose combination of drugs for any indication

Drug A

Drug B

Drug C

26

50 candidate drugs

>2,000 available drugs

25 positive drugs

Discovering the complex molecular network of a disease => inventory of

all possible therapeutic targets

4 synergistic combos

In Human

In Silico Screening

In Vitro

Faster, Safer, More Efficient and Affordable Drug Development Process

1 PLEODRUG

Screening and repositioning approved drugs at low-dose in order to identify

synergistic combinations

Drug A

Drug B

Drug C Protein C

Protein B

Protein A

27

Our R&D Process is 5 Years Faster

DEVELOPMENT PHASE

In human Preclinical

Development Drug

Identification Target

Identification

RESEARCH PHASE

Classical Model: A long and expensive process lasting 15 years with high attrition rates

Pharnext Model: A faster development of effective and safe drugs at reduced cost lasting 10 years

RESEARCH PHASE DEVELOPMENT PHASE

In human In

vivo In

vitro

In silico screening

Network Mapping

3 years 7 years

6 years 9 years

(no Phase 1 required)

28

High-Value Opportunities in a Broad Spectrum of Diseases

Note 1: ALS = Amyotrophic Lateral Sclerosis Note 2: Includes glioma, hepatoma, melanoma, lung cancer, prostate cancer Note 3: SLE = Systemic Lupus Erythematosus

PXT3003

PXT864

Preclinical pipeline

HIGH BLOOD FATS

TOXIC NEUROPATHIES

NEUREGULIN SIGNALING

ALZHEIMER’S DISEASE

POLYCYSTIC KIDNEY

AXONAL GROWTH

MEMORY

HYPERTENSION STROKE

CORONARY DISEASE

SCHIZOPHRENIA

ALS(1)

MYOPATHIES

SLE(3)

ULCERATIVE COLITIS

PARKINSON’S DISEASE

CANCER(2)

DOWN’S

SYNDROME

RHEUMATOID

ARTHRITIS

DIABETIC

NEUROPATHIES

CROHN’S DISEASE

TYPE 2 DIABETES

CMT MULTIPLE SCLEROSIS

PSORIASIS ASTHMA

26 disease networks already mapped, many of which await therapeutic targeting

Corporate Summary

30

Deeply Experienced Management Team

Prof. Daniel Cohen, MD, PhD CEO & Founder

Catherine Chalandon VP Operations & Strategy

Pierre Schwich Chief Financial Officer

René Goedkoop, MD Chief Medical Officer

Serguei Nabirotchkin, PhD Chief Biology Officer & Co-Founder

Xavier Paoli Director of Commercialization Strategy

31

Pharnext Business Model

• Orphan indications (e.g. CMT1A, ALS)

‒ Stand-alone development and commercialization

‒ Opportunistically enter into partnership

• Common indications (e.g. Alzheimer's, Parkinsons)

‒ Seek partnership after proof of concept (Phase 2a/2b)

• R&D partnership from PLEOTHERAPY® platform

‒ Use ongoing R&D compounds (NCEs) from partners to develop new combinations with off patent drugs

• In the same indication

• Or in new indications

32

Financial Summary

Operating Expenses (6 months) 7 667

Revenues (6 months) 1 993

Cash 30 561

Equity 13 601

Liabilities 20 341

Data (pro-forma post IPO) in K€ as of June 30, 2016

Listed on Euronext Alternext Stock Exchange in Paris (ISIN code: FR00111911287)

10,784,615 Shares Outstanding and 11,817,940 Fully Diluted (as of 10/31/16)

33

PXT3003 in CMT1A

H2: Adaptive and futility analysis

Q2: Top-line results of Phase 3 trial

Q1:Submission of NDA/MAA (FDA + EMA)

2017 2018 2019

PXT864 in Alzheimer’s Disease

H2: Initiation of Phase 2b > 200 patients

H2: Initiation of PXT864 new formulation study

H2: Final results of Phase 2b trial

Upcoming Newsflow

Corporate Presentation January 2017

Appendix Slides

36

PXT864 Protects and Improves Symptoms in Parkinson Disease

Protective effect: Treating BEFORE lesion

L-DOPA antagonism: Treating AFTER lesion

Symptomatic effect: Treating AFTER lesion

After 14 days of treatment, before (protective) or after (symptomatic) PD symptom manifestation, PXT864 improved motor function, protected dopaminergic neuronal cells and did not negatively interact with L-DOPA , paving the way to testing PXT864 in patients already treated with L-DOPA * P < 0.05; *** P < 0.001 vs. placebo (1): Clinical dose of L-DOPA PXT864 dose 1/day: 0.08 mg/kg acamprosate + 1.2 mg/kg baclofen PXT864 dose 2/day: 0.2 mg/kg acamprosate + 3 mg/kg baclofen

37

PXT864 Efficacy in ALS and Positive Interaction with SoC

PXT864 improved synergistically the number of neuromuscular junctions affected in the disease. *** P < 0.001 vs. placebo S: Synergistic

PXT864 interacted positively with Riluzol to increase the number of neuromuscular junctions. *** P < 0.001 vs. placebo # P < 0.05 vs. Riluzole (Rilu)

S

Motor unit number

38

Unparalleled Scientific Advisory Board

Ilya Chumakov, PhD, D-Sc Chairman, Co-founder

Pioneer in genetics, genomics and molecular pharmacology

Prof. David Cornblath, MD Expert in peripheral nerve disorders

Chief editor of Journal of the Peripheral Nervous System and of Journal of the Peripheral Nerve

Prof. Wally Gilbert, PhD Nobel Prize in

Chemistry 1980

Prof. Eric R. Kandel, MD Nobel Prize in

Chemistry 2000

Prof. Jean-Marie Lehn, PhD Nobel Prize in

Chemistry 1987 Innovator in supramolecular chemistry

Prof. Richard Lewis, MD

Expert in inflammatory and hereditary neurological diseases, discoverer of the Lewis-Summer syndrome

Prof. Anthony Schapira, MD Expert in Parkinson’s disease

Prof. Michael Sereda, MD, PhD Expert in hereditary neuropathies, creator of the PMP22 transgenic rat model

39

Shareholdings

13%

31%

18%

16%

4%

18%

Shareholding Structure as of September 2016 Outstanding Shares : 10 784 615

Managers & advisers Truffle Capital

Pierre Bastid Other historical shareholders

Corporate investors Other

18%

29%

16%

17%

4% 16%

Shareholding Structure fully diluted (11 817 940)

Managers & advisers Truffle Capital

Pierre Bastid Other historical shareholders

Corporate investors Other

40

Very Strong Patentability and Enforceability

• 340+ patent applications and ~100 granted patents across 13 diseases worldwide

‒ Composition of matter granted in EU, pending in US

‒ Method of use granted in both EU and US

‒ Formulation claims pending in both EU and US

‒ Composition of matter pending in EU and granted in US

‒ Method of use granted in both EU and US

• Compounding not authorized in US / major EU countries when alternative product approved

PXT3003

PXT864

Program Patent PCT Number Scope Filing Date Expiry Date Status

PXT-3003

CMT2 WO2009068668 Composition of matter for binary compositions covering PXT-3003 Method of use for binary compositions covering PXT-3003

Nov 2008 EU Nov 2028 US Jul 2031

Granted EU Granted EU/US

CMT4 WO2010139627 Composition of matter and method of use for PXT-3003 May 2010 May 2030 Granted EU, Pending US

CMT7 WO2014195394 Formulation for PXT-3003 June 2014 June 2034 Pending EU/US

PXT-864 AD2 WO2009133141 Method of use for baclofen Apr 2009 Apr 2029 Pending EU/US

AD7 WO2012117076 1. Composition of matter and method of use for PXT-864 2. Composition of matter for PXT-864 combined with donepezil

Mar 2012 Mar 2032 Granted EU(1)/US

PXT-864 PD1 WO2013127918 Method of use for PXT-864 in Parkinson’s Feb 2013 Mar 2032 Pending EU/ Granted US

(1) Except composition of matter for PXT-864 which is pending in EU

41

Pharnext’s Published Articles

1. Chumakov et al., Polytherapy with a combination of three repurposed drugs (PXT3003) down-regulates Pmp22 over-expression and improves myelination, axonal and functional parameters in models of CMT1A neuropathy. Orphanet Journal of Rare Diseases 2014, 9:201.

2. Attarian et al., An exploratory randomised double-blind and placebo-controlled phase 2 study of a combination of baclofen, naltrexone and sorbitol (PXT3003) in patients with Charcot-Marie-Tooth disease type 1A. Orphanet Journal of Rare Diseases 2014, 9:199.

3. Chumakov et al., Combining two repurposed drugs as a promising approach for Alzheimer’s disease therapy. Scientific Reports (nature) 2015, 4:7608.

4. Foucquier et al., Analysis of drug combinations: current methodological landscape. Pharmacology Research & Perspectives 2015, 3:e00149.

5. Mandel et al., A meta-analysis of randomized double-blind clinical trials in CMT1A to assess the change from baseline in CMTNS and ONLS scales after one year of treatment. Orphanet Journal of Rare Diseases 2015, 10:74.

6. Hajj et al., Combination of acamprosate and baclofen as a promising therapeutic approach for Parkinson’s disease. Scientific Reports (nature) 2015, 5:16084.

7. Toulorge et al., Molecular changes in the postmortem parkinsonian brain. Journal of Neurochemistry 2016, 139:27-58.

8. Wang et al., A Rasch Analysis of the Charcot-Marie-Tooth Neuropathy Score (CMTNS) in a Cohort of Charcot-Marie-Tooth Type 1A Patients. PLOS ONE 2017, (In press).

42

Pharnext’s Oral and Poster Communications

1. Scart-Grès et al., First evidence of PXT-864 effect in the treatment of mild AD: results on 30 patients from the PLEODIAL I study. Oral Presentation at CTAD Philadelphia 2014, November 23.

2. Haddad et al., First evidence of PXT00864 neurological protective effect in scopolamine-induced memory impairment model in humans. Poster at CTAD Philadelphia 2014, November 23.

3. Schmitt et al., P300 ERP component used as biomarker to test the effect of a new fixed combination in scopolamine-induced memory impairment model. Poster at CTAD Philadelphia 2014, November 23.

4. Schmitt et al., Application of spatio-temporal PCA for analyzing Event-Related Potential (ERP). Poster at CTAD Philadelphia 2014, November 23.

5. Bennys et al., Benefits of ERP used as biomarker in early stage clinical development of AD therapeutics: PXT-864 case report. Poster at CTAD Philadelphia 2014, November 23.

6. Boucard et al., Identification and replication of biomarkers of Alzheimer’s disease in blood samples from different cohorts. Oral Presentation at Biomarkers for Brain Disorders: Challenges and Opportunities Cambridge 2015, February 1.

7. Scart-Grès et al., Design of the next phase III trial aiming to confirm efficacy and safety of PXT3003 in the treatment of Charcot-Marie-Tooth Disease Type 1A. Poster at PNS meeting Québec 2015, June 27.

8. Prukop et al., An experimental trial of an early onset short-term treatment with a combinational drug (PXT3003) in the Charcot-Marie-Tooth 1A rat model. Poster at CMTR meeting Venice 2016, September 8.

9. Paoli et al., An online survey of neurologists about Charcot-Marie-Tooth Disease Type 1A. Poster at CMTR meeting Venice 2016, September 8.

10. Laffaire et al., Baseline Analysis of PXT3003 Phase 2 Data Reveals Two Blood Early Candidate Biomarkers for Therapeutic Response in CMT1A. Poster at CMTR meeting Venice 2016, September 8.

11. Touchon et al., 36 Weeks of Treatment with PXT-864 in Mild Alzheimer’s disease: Results from the PLEODIAL Extension Study. Oral Presentation at CTAD San Diego 2016, December 11.

12. Bennys et al., Neurophysiological effect of PXT-864 in mild Alzheimer’s disease patients. Poster at CTAD San Diego 2016, December 11.