Conference call July 25 2019

July 2019

Inhibiting NOX enzymes to treat multiple diseases with high medical need

Euronext: GKTX

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Ph2 PBC highlight the potential of setanaxib as an anti-fibrotic therapy in the liver, lung, skin, kidney and other organs

Genkyotex: Establishing NOX inhibition as a new therapeutic class

Page 3

� We discover and develop oral small molecule NOX inhibitors— Activation of NOX enzymes is key in many multifactorial diseases— World Health Organization (WHO) recognized NOX inhibitors as a new therapeutic class

� Lead asset setanaxib (GKT831): a potent anti fibrotic oral small molecule— JDRF-funded Phase 2 trial in kidney fibrosis (DKD) ongoing— NIH-funded Phase 2 trial in idiopathic pulmonary fibrosis (IPF) to be launched in 2019— Further potential in NASH, PSC, and immuno-oncology

� PBC Phase 2 provides clinical evidence of anti-fibrotic activity in liver fibrosis – a reduction of ~3kPa indicating an average of 1 point liver fibrosis reduction

� Partnership with Serum Institute of India Private Ltd valued at up to €150 million + royalties

� Trading on Euronext as GKTX since March 2017— Cash and cash equivalents of €4.5 million as of June 30 2019, cash runway to April 2020

Discovery platform delivers broad pipeline in diseases with high medical needSetanaxib Phase 2 PBC data support development in multiple fibrotic diseases

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Setanaxib - Liver Fibrosis Primary Biliary Cholangitis (PBC)Top-line results published May 2019

Setanaxib - Kidney Fibrosis Diabetic Kidney Disease (DKD) in T1D(IIT1 funded by JDRF2 - Trial launched in H2 2017)

Setanaxib - Lung Fibrosis Idiopathic Pulmonary Fibrosis (IPF)(IIT funded by US NIH3 – IND approved by FDA)

NOX1 inhibitors Preclinical

Novel NOX inhibitors

Vaxiclase Pertussis vaccine (Licensed to SIIPL)

Preclinical Phase 1 Phase 2 Phase 3Program

Discovery

1Investigator initiated trial2 Juvenile Diabetes Research Foundation3 National Institutes of Health

Setanaxib’s unique efficacy and safety profile can address the unmet medical need

Primary Biliary Cholangitis (PBC):an orphan disease in the large liver fibrosis market

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Source 1 : In Europe, USA and Japan. Boonstra K. et al. Epidemiology of primary sclerosing cholangitis and primary biliary cirrhosis: a systematic review. J Hepatol. 2012 May;56(5):1181-8

� Disease overview• Chronic autoimmune liver disease - progressive destruction of bile ducts• Prevalence of between 2 - 40 cases per hundred thousand-population1

• Women make up about 90% of PBC cases• Diagnosis based on presence of auto-Abs and elevated markers of cholestasis

including alkaline phosphatase (ALP) & gamma glutamyl transpeptidase (GGT)

� Therapy• Current medications mainly target cholestasis and include generic anti-

cholestatic drugs (UDCA and fibrates) and obeticholic acid

� Unmet medical need• Anti-fibrotic agents to delay disease progression and obviate transplant• Effective agents targeting itching and fatigue to restore quality of life• Safe, well tolerated therapy suitable for combination with anti-cholestatic

therapies (UDCA, fibrates, OCA) Primary Biliary Cholangitis

Inflammation and scar tissue destroy ducts

Normal bile ducts

Gallbladder

Hepatic ductCommon bile duct

Liver

Cystic duct

Setanaxib

� Primary efficacy endpoint: change in GGT at week 24

� Key secondary endpoints: liver stiffness assessed by Fibroscan®, changes in ALP & QoL

� Key eligibility criteria

— ALP ≥1.5XULN & GGT ≥1.5XULN (stratification according to baseline GGT (> or < 2.5XULN)

— On UDCA for ≥ 6 months & stable dose for ≥ 3 months – stable UDCA dose continued throughout 24-week treatment period

— Exclusion of history of cirrhosis with complications or current MELD score ≥ 15

— ALT > 3XULN or total bilirubin > 1XULN

— Prohibited medications: fibrates and obeticholic acid (12-week wash out)

Setanaxib was evaluated in a large 24-week Phase 2 trial

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Placebo

Setanaxib 400mg once a day (OD)

Setanaxib 400mg twice a day (BID)

Follow up

Follow up

Follow up

111 randomized (initial target 102)

ALP ≥1.5XULNGGT ≥1.5XULN

Inadequate biochemical response to UDCA

Baseline Week 6 Week 24

Setanaxib

Liver fibrosis progressively disrupt liver structure and function

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Setanaxib

F3 F4

Liver fibrosis is the best predictor of long-term outcomes in multiple liver diseases

F1 F2

Non-invasive assessment of liver fibrosis with Fibroscan®In PBC, liver stiffness ≥ 9.6 kPa corresponds to advanced liver fibrosis of ≥F3

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Elas

ticity

(kPa

)

• In multiple liver diseases including PBC, NASH and PSC, liver stiffness correlates with the histologic liver fibrosis stage (F0 to F4)1

• In PSC, elevated liver stiffness is associated with adverse disease outcomes, including liver transplant, hepatic complication and death1

• Our pre-defined cutoff value of 9.6 kPa has been extensively validated and used in previous trials1

1Corpechot C et al. Baseline Values and Changes in Liver Stiffness Measured by Transient Elastography Are Associated With Severity of Fibrosis and Outcomes of Patients With Primary Sclerosing Cholangitis. Gastroenterology 2014;146:970–979. Corpechot C et al. Assessment of Biliary Fibrosis by Transient Elastography in Patients With PBC and PSC. Hepatology 2006;43:1118-1124. Park CC et al. Magnetic Resonance Elastography vs Transient Elastography in Detection of Fibrosis and Noninvasive Measurement of Steatosis in Patients with Biopsy-proven Nonalcoholic Fatty Liver Disease. Gastroenterology 2017; 152(3): 598–607.

Liver stiffness is an indicator of liver inflammation, cholestasis and fibrosis

Fibrosis stage

Histologic fibrosis score

Liver stiffness

Setanaxib

Phase 2 trial of setanaxib in PBC: Baseline patient characteristics

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Baseline characteristics in line with the targeted population of active PBC patients

1 Once daily; 2 Twice daily

Baseline patient characteristics Placebo Setanaxib400mg OD

Setanaxib400mg BID ALL

N 37 38 36 111

Age (years) 56 (9) 57 (9) 56 (9) 56 (9)

Females (%) 95 79 94 89

Body weight (kg) 73 (15) 73 (13) 70 (16) 72 (15)

UDCA dose (mg/kg) 13.0 (4.1) 15.9 (5.6) 16.4 (10.4) 15.1 (7.3)

Liver stiffness measurement (kPa) 10.7 (7.0) 12.5 (13.7) 8.3 (3.7) 10.7 (9.5)

GGT (IU/L) 227 (200) 242 (167) 242 (181) 237 (182)

ALP (IU/L) 300 (141) 302 (121) 346 (164) 315 (143)

ALT (IU/L) 43 (16) 45 (22) 56 (35) 48 (26)

AST (IU/L) 43 (17) 44 (21) 50 (31) 46 (24)

Total bilirubin (µmol/L) 10.7 (4.3) 11.1 (4.6) 10.4 (4.6) 10.7 (4.5)

hsCRP (mg/L) 4.8 (4.6) 5.8 (5.2) 5.1 (5.1) 5.3 (4.9)

Values expressed as mean (±SD)

Setanaxib

Setanaxib 400mg BID achieves significant reduction in GGT over the24-week treatment period (p<0.002)

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Perc

ent c

hang

e in

GG

T fr

om B

asel

ine

-7%

-12%

Change in GGT for 400mg BID is highly significant at week 6 (IA) and throughout the 24-week treatment period. Unexpectedly, statistical significance was lost at week 24

Treatment duration (week)

Percent changes in GGT (%)

-6.2-7.5

-5.5

-5.2-8.4

-7

-11.8

1.7

-4.5 -4.9

-17

-22

-18.6 -18.7 -19

-30

-25

-20

-15

-10

-5

0

5

10

15

0 2 6 12 18 24

Mean ± SEM

Placebo (n=37)

Setanaxib 400mg OD (n=38)

Setanaxib 400mg BID (n=36)

p=NS

p<0.002 400mg BID vs placebo over 24 weeksp<0.01

Setanaxib

Setanaxib 400mg BID achieves significant reduction in ALP over the24-week treatment period (p<0.001)

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Perc

ent c

hang

e in

ALP

from

Bas

elin

e

Treatment duration (week)

-3.6 -1.4

-0.6 -0.5

-3.1

-5.5

-8.6

-3.9

-7.8-9.7

-13

-16.3-14.6

-15.8

-12.9

-20

-15

-10

-5

0

50 2 6 12 18 24

Percent changes in ALP (%)

p<0.001

p=0.049

p<0.001 400mg BID vs placebo over 24 weeksMean ± SEM

Placebo (n=37)

Setanaxib 400mg OD (n=38)

Setanaxib 400mg BID (n=36)

Setanaxib

Post-hoc analyses explored the loss of statistical significance at Week 24 andthe therapeutic benefits achieved with setanaxib

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Setanaxib

• Exploring week-24 GGT data

• Analyses explored potential causes for the loss of statistical significance at week 24

• Correlation between changes in GGT and ALP at week 24

• The correlation between changes in GGT and ALP was assessed to confirm that the observed reductions in GGT and ALP reflected a consistent reduction in cholestatic injury

• Responder analysis

• Analyses were carried out to further explore the therapeutic benefits achieved with Setanaxib

• A key question was whether setanaxib also reduced cholestatic injury in patients with elevated liver stiffness, in addition to reducing liver stiffness

Normal distribution of GGT data was observed for the placeboand 400mg BID groups

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Chan

ge in

GG

T fr

om

Base

line

to W

eek

24 (%

)

• A normal distribution of data is one in which the majority of data points occur within a small range, with few outliers on the higher and lower ends of the data range

• Mean (average) and median values are similar

• Standard deviations are small compared to the mean value

• At week 24, distribution of GGT data in the placebo and 400mg BID groups was normal

Setanaxib

Post-hoc analyses identified non-normal data distribution in the 400mg ODgroup as the reason for the loss of statistical significance at week 24

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Chan

ge in

GG

T fr

om

Base

line

to W

eek

24 (%

)

• At week 24, distribution of GGT data in the 400mg OD group was considered as non-normal

• Non-normal data distribution in the 400mg OD group caused the loss of statistical significance atweek 24 for the 400mg BID group

Setanaxib

Setanaxib 400mg BID achieved statistical significance for primary endpointafter correction of non-normal distribution (p=0.02)

Page 15

-13%

Primary endpoint: change in GGT at Week 24

Primary endpoint: change in GGT Interim analysis (Week 6)

Final analysis(Week 24)

Statistical method Without correction for non-normal distribution

With correction for non-normal distribution*

400mg BID compared to placebo p<0.01 p=0.3 p=0.02

Secondary endpoints (as per protocol) Final analysisChange in GGT over 24-week treatment period p<0.002Change in ALP over 24-week treatment period p<0.001

Secondary endpoints: change in GGT & ALP over the 24-week treatment period

*Log transformation

Setanaxib

Efficacy of setanaxib confirmed by correlated reductions in cholestatic markers

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400mg ODPlacebo 400mg BID -24%

-13%

-9%

Correlation between changes in ALP and GGT at week 24

Percent change in ALP at Week 24 (%)

Perc

ent c

hang

e in

GG

T at

Wee

k 24

(%)

Pearson’s correlation coefficient = 0.61p value <0.0001 Placebo

Setanaxib 400mg ODSetanaxib 400mg BID

Setanaxib

In the full population setanaxib prevents progression of liver stiffness

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Perc

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e in

live

r st

iffne

ss a

t Wee

k 24

(%)

Trend seems to be dose dependent. Baseline values are 10.7 for placebo, 12.5 for 400mg OD and 8.3 kPa for 400mg BID

-0.5

-0.4

-0.3

-0.2

-0.1

0

0.1

0.2

0.3

0.4

0.5

Abso

lute

cha

nge

in li

ver

stiff

ness

at W

eek

24 (k

Pa)

Percent change in liver stiffness (%) Absolute change in liver stiffness (kPa)

-6

-5

-4

-3

-2

-1

0

1

2

3

4

5 +4%

-5%

+1%

+0.4

+0.1

-0.4

N=32 N=33

N=26N=32 N=33

N=26

400mg ODPlacebo 400mg BID

Median values Median values

Setanaxib

Setanaxib achieved clinically meaningful reduction in liver stiffness inpatients with estimated liver fibrosis score of ≥ F3

Page 18

Patients with baseline liver stiffness < 9.6 kPa

Patients with baseline liver stiffness ≥ 9.6 kPa

0

2

4

6

8

10

12

14

16

0

2

4

6

8

10

12

14

16Week 24Baseline

Median values Median values

Live

r stif

fnes

s at

Bas

elin

e an

d w

eek

24 (k

Pa)

Placebo(n=18)

400mg OD(n=21)

400mg BID(n=20)

Placebo(n=17)

400mg OD(n=14)

400mg BID(n=14)

Live

r stif

fnes

s at

Bas

elin

e an

d w

eek

24 (k

Pa)

12.7

14.2 14.113.1

12.2

9.1

Upper limit of normal (7 kPa)

Setanaxib

5.7 6.2 6.2 6.37.0 6.8

In just 24 weeks of treatment setanaxib achieves average reduction of3kPa – an estimated one-point fibrosis score reduction

Page 19

Percent change in liver stiffness Absolute change in liver stiffness

400mg OD (n=14)Placebo (n=17) 400mg BID (n=14)

Abso

lute

cha

nge

in li

ver

stiff

ness

at w

eek

24 (k

Pa)

-5

-4

-3

-2

-1

0

1

2

+0.4

-1.9

-2.7

Mean ± SEM

Perc

ent c

hang

e in

live

r st

iffne

ss a

t wee

k 24

(%)

-35

-30

-25

-20

-15

-10

-5

0

5

10

15

+4.2

-5.3

-20.9

Mean ± SEM

p=0.039

Setanaxib achieves clinically significant reduction in liver stiffness in PBC patients with elevated liver stiffness

~3 kPa

Setanaxib

Patients with the greatest medical need show marked reductions in ALP

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Perc

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hang

e in

ALP

at W

eek

24 (%

)

<9.6 kPa(n=59)

400mg ODPlacebo 400mg BID

All patients(n=104)

≥9.6 kPa(n=45)

Percent reduction in ALP at week 24

Setanaxib

Mean values-30

-25

-20

-15

-10

-5

0

-9%

-13%

-3%-6%

-14%

-3%

-10%

-2%

-24%

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Perc

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hang

e in

GG

T at

Wee

k 24

(%)

400mg ODPlacebo 400mg BID

Percent reduction in GGT at week 24<9.6 kPa

(n=59)All patients

(n=104)≥9.6 kPa

(n=45)

Patients with the greatest medical need show marked reductions in GGT Setanaxib

-40

-30

-20

-10

0

10

20

30

-19%

-13%

-32%

-10%

+9%

-5%

-15%

-8%-5%

Even patients with modest liver stiffness show significant reductions in ALP

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Perc

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hang

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ALP

at W

eek

24 (%

)

<7.3 kPa(n=40)

400mg ODPlacebo 400mg BID

All patients(n=104)

≥7.3 kPa(n=64)

Percent reduction in ALP at week 24

Setanaxib

-25

-20

-15

-10

-5

0

-19%

-12%-10%

-3%

-10%

-13%

-2%-3%

-6%

Setanaxib 400mg BID significantly improves Quality of Life domainsincluding fatigue

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Setanaxib 400mg BID improved quality of life across multiple domains important to PBC patients

1 Once daily; 2 Twice daily

PBC-40 questionnaire

PBC-40 QoL domains PlaceboSetanaxib

400mg OD1Setanaxib

400mg BID2p value (400mg BID vs placebo at week 24)

General symptoms 1.1 1.1 -3.7 0.156Itch (Pruritus) -6.8 -11.4 -9.5 0.443Emotional 8.7 4.9 -16.9 0.031Fatigue 2.4 0.3 -9.9 0.027Social 9.3 8.1 -7.7 0.003Cognitive 5.2 16 -1.9 0.332Mean percent changes from Baseline to Week 24 in Quality of Life domains included in the PBC-40 questionnaire.p values for comparison of changes in the 400mg BID dose against placebo are shown.

• Reduced quality of life is one of the main unmet medical need in PBC, in particular fatigue

• Approved therapies do not improve quality of life

Setanaxib

GKT831 was safe & well tolerated at all doses over the 24-week treatmentperiod

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GKT831

Excellent safety profile supports combination therapy with generically available anti-cholestatic agents including UDCA and fibrates

PlaceboGKT831

400mg ODGKT831

400mg BIDSAEs 1 0 1AEs 121 119 100AEs leading to patient discontinuation 0 2 2AEs leading to drug interruption 1 1 2Gastrointestinal 22 25 25Infections 24 12 11Skin and subcutaneous tissue 12 15 14Nervous system 12 17 9General disorders 14 6 12Musculoskeletal and connective tissue 10 12 6Investigations 3 7 7Injury, poisoning, procedural complications 4 4 5Respiratory, thoracic, and mediastinal 4 5 4Psychiatric disorders 7 1 0Incidence of Treatment-Emergent Adverse Events by System Organ Class (top 10 system organ classes ranked according to AE incidence)

Data supports use of setanaxib in a broad patient population, includingpatients with advanced fibrotic liver disease

Page 25

• Setanaxib shows marked efficacy in difficult to treat patients with advanced disease

• Setanaxib achieved clinically meaningful reductions in liver stiffness in patients with elevated liver stiffness at baseline (≥9.6 kPa)

• Even patients with modest elevation in liver stiffness benefit from to setanaxib, for both liver stiffness and markers of cholestatic injury (GGT and ALP)

• Setanaxib is the first compound to improve quality of life, with a marked effect on fatigue

• Setanaxib was safe and well tolerated at all doses

• The Company is planning the phase 3 program

Setanaxib