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CORPORATE PRESENTATION
MAY 2020
The statements made in this presentation may include forward-looking statements regarding thefuture operations of ERYTECH Pharma S.A., including estimates of target market opportunity,timing of planned clinical trials and results from those trials, regulatory strategy and timing ofplanned regulatory submissions, manufacturing capabilities and strategy for expansion of theERYCAPS platform. Although we believe that the expectations contained in this presentation arereasonable, these forward-looking statements are only estimates based upon the informationavailable to ERYTECH Pharma S.A. as of the date of this presentation. The company'sexpectations regarding the effects of COVID-19 on the Company's trials and development may beincorrect. Except as required by law, we expressly disclaim any responsibility to publicly update orrevise our forward-looking statements, whether as a result of new information, future events orotherwise. Thus, the forward-looking statements herein involve known and unknown risks anduncertainties and other important factors such that actual future operations, opportunities orfinancial performance may differ materially from these forward-looking statements. Undue relianceshould not be placed on forward-looking statements, which speak only as of the date hereof. Allforward-looking statements contained herein are qualified in their entirety by the foregoingcautionary statement.
Forward Looking Statements
33
Leader in Red Blood Cell-based Cancer Therapeutics
Months
Phase 2b in 2L Pancreatic Cancer
OVERALL SURVIVAL (ITT)
Reproducible encapsulation of therapeutic compounds in red
blood cells
Focus on oncology, targeting cancer cells’ altered amino acid
metabolism through encapsulated asparaginase
Lead product candidate eryaspase, first asparaginase to show
efficacy in solid tumors; strongest survival benefit observed in any
2L pancreatic cancer study to date
Phase 3 trial in 2L pancreatic cancer ongoing in EU and US;
Phase 2 in 1L TNBC and Phase 2 IST in 2L ALL ongoing
Industrialized production: own cGMP production facilities in the
United States and Europe
Listed on Nasdaq and Euronext; cash runway into Q1 2021
reduction in
risk of death
(HR, 0.60;
P=0.008)
40%
ALL: Acute Lymphoblastic Leukemia; TNBC: Triple-Negative Breast Cancer
Hammel et al., European Journal of Cancer, 2019
4
Late Stage Clinical Pipeline and Preclinical Programs
4
Mode of actionProduct Candidate
or ProgramIndication Discovery Pre-clinical Phase 1 Phase 2
Phase 3/
Pivotal
Next Anticipated
Milestone
Cancer metabolism
Tumor starvation
eryaspase/GRASPA®
(asparaginase)
Pancreatic Cancer
2L
Interim: YE 2020
Final: 2H 2021
Triple Negative
Breast Cancer 1L Final: 2021
Acute Lymphoblastic
Leukemia 2L
Interim: 1H 2020
Final: 2H 2020
Pancreatic cancer
1LLaunch: 2H 2020
erymethionase
(methionine-γ-lyase)Solid tumors
Immune
modulation
Immune-oncology
Tolerance inductionTBD*
Enzyme
replacement
Therapeutic
enzymesMetabolic diseases
1L First Line; 2L Second Line; IST Investigator Sponsored Trial; NOPHO : Nordic Organization of Pediatric Hematology and Oncology
* To be determined by SQZ Biotech
TRYbeCA-1
IST
TRYbeCA-2
NOPHO IST
5
Red Blood Cells, Ideal Carriers for Amino Acid Lowering Agents
Well known
biocompatibility
Routine allogeneic use
Protective membrane
Reduced toxicity
Prolonged activity
Readily available at blood
banks
Established sourcing
Most abundant cell in
human body
Extensive biodistribution
Permeable membrane
Potential to load with drugs
Plasma metabolites can pass
Long circulating life
Prolonged activity
5
6
ERYCAPS, an Innovative Technology Platform
Reproducibly encapsulating drug substances inside allogeneic red blood
cells using proprietary hypotonic loading technology
Reproducible
proprietary ‘osmotic fragility’ process enables
reproducible loading
Fast
industrialized process can deliver tailored therapy
within 24 hours
Broad range
peptides, RNA, proteins, antibodies, …
Strong IP
265 granted patents in 15 patent families
Controlled
Hypotonic Loading
Hypertonic
Resealing
Drug substance
Pores
6
7
Fully-owned GMP
production capacity in EU
and the US
Established partnerships
with blood banks in EU and
the US
More than a decade
experience in manufacturing
and supply chain management
Validated supply &
manufacturing model – 3000+
batches, 500+ patients
treated,10+ countries
Industrialized and Scalable ProcessP
RE
SC
RIP
TIO
N
Sourcing of
compatible RBC
PREPARATION
2
SOURCING
1
BATCH RELEASE
4
ENCAPSULATION
3
SHIPPING
5
Identification of key
parameters
ERYCAPS automated
Manufacturing
(3-5 hours)
QC Control
Release by QPShipping
Patient
Preparation2
Sourcing1
Batch releaseEncapsulation3 4
Shipping5
Possible in 24h
7
8
Targeting Cancer Cells Amino Acid Metabolism, an Emerging Field
Many cancers exhibit increased demand
for specific amino acids, becoming
dependent on exogenous supply or de
novo synthesis of these amino acids1-3 Depleting select amino
acids in blood (e.g.
asparaginase)
Blocking uptake by
blocking transporters
(e.g., LAT 1-4)
Inhibiting biosynthetic or
catabolic enzymes (e.g.
arginase inhibitor)
Amino acids
1. Lukey et al., Drug Discovery Today, 2017
2. Pavlova & Thompson, Cell Metabolism, 2016
3. Luengo et al., Cell Chemical Biology, 2017 8
9
CURRENT
USE
• Cornerstone treatment in Phi-neg pediatric acute lymphoblastic leukemia (ALL)
• Four forms of L-asparaginase products currently marketed in ALL; worldwide sales
est. $400M in 2018
• E-coli derived: Native asparaginase: e.g., Kidrolase (Kyowa Hakko)
Recombinant asparaginase: Spectrila (medac)
Pegylated asparaginase: Oncaspar (Servier)
• Erwinia derived: Erwinaze (Jazz Pharmaceuticals)
MODE OF
ACTION
Asparaginase breaks down circulating asparagine (and glutamine), and deprives
tumor cells of amino acids essential for their growth
• Important side effects (e.g., allergies, coagulation disorders, pancreatic and hepatic
toxicities) limit therapeutic window to essentially pediatric ALL
• Beyond ALL, some use in AML and lymphoma; no known use in solid tumors
Asparaginase, First Amino Acid Targeting Anticancer Drug1
1. Lukey et al., Drug Discovery Today, 2017
Mode of action
Current use
Limitations
9
10
Active transporters in membrane
of red cell pump circulating
asparagine and glutamine into
the red cell…
Y
Asparagine and glutamine
Asparaginase
Antibody
…where the encapsulated
L-asparaginase hydrolyzes the
asparagine and glutamine
Eryaspase (GRASPA®️), Enabling Broader Use of Asparaginase
A novel circulating ‘bioreactor’ to degrade asparagine (and glutamine)
Prolonged activity
Reduced toxicity
Potential to provide
asparaginase to patients
that are unable to
tolerate non-
encapsulated products
Degradation products
10
11
• ERYTECH started development in fragile ALL patients, confirming the concept of prolonged activity and
reduced toxicity in different clinical trials, including a Phase 2/3 trial in Relapsed/Refractory ALL:
Positive Results in ALL Confirmed Proof of Concept
Key results of Phase 2/3 trial in R/R ALL Eryaspase (N=26) L-ASP (N=28)
% of patients with ≥ 1 hypersensitivity reaction during induction** 0% 46%
Mean duration of asparaginase activity above 100 U/L during induction (days)** 18.9 8.5
Complete Remission rate (CR)* 65% 36%
Overall Survival (OS) HR = 0.72
** Statistically significant (p<0.001) * Statistically significant (p<0.05). Presented at ASH 2014
• The favorable safety profile observed in these trials enabled ERYTECH to pursue the development of
bringing eryaspase to the much larger unmet medical need of solid tumors
11
12
Pancreatic Cancer, Large Unmet Medical Need
• Pancreatic cancer is one of the leading
causes of cancer death in the world, with an
increasing incidence1,2
• In 2018, approximately 185,000 people were
newly diagnosed with pancreatic cancer in
Europe and the United States1
• Overall prognosis for pancreatic cancer is
poor, with a 5-year survival rate of <10%2
• Approximately 50% of pancreatic cancer
patients are eligible for second line treatment3
• Chemotherapy remains main treatment
option; limited progress in development of
new treatment options
203020202010
Pro
jecte
d c
an
ce
r d
ea
ths in
th
e U
S (
10
00
’s)
0
20
40
60
160
Year
Lung and bronchus
Pancreas
Liver
Leukaemia
80
140
Colon and rectum
Breast
Prostate
Bladder
1. WHO Cancer Today, Globocan 2018; 2. Rawla et al. WJO 2019 ; 3. Hua et al. Onco Targets Ther, 2018 12
13
• Pancreatic cancer (PAC) is characterized by extensive reprogramming of cellular metabolism leading to
increased asparagine and glutamine requirements. Deprivation of glutamine and asparagine results in loss of
cancer cell viability1
• In vitro studies demonstrated promising anti-cancer effects for asparaginase against human pancreatic tumor
cell lines2-3
• Attempts at evaluating asparaginase in solid tumor indications in the clinic have been unsuccessful due to
toxicity4-7
• In a Phase 1 trial in metastatic pancreatic cancer, eryaspase demonstrated an acceptable safety profile8-9
• The tolerability of eryaspase and the potential role of metabolic dysregulation in this disease provided
rationale for further clinical evaluation
Altered Metabolic Pathways in PAC, a Role for Asparaginase?
1. Kawanda Int J Clin Oncol 2017; 2. Cui, Cancer Res 2007; 3. Dufour, Pancreas 2012;
4. Lessner Cancer Treat Rep 1980; 5. Hays Molec Clin Oncol 2013; 6. Agrawal Cancer 2003;
7. Borad Cancer Investigation 2015; 8. Bachet Pancreas 2015; 9. ClinicalTrials.gov: NCT01523808 13
14
Phase 2b Trial in 2L Pancreatic Cancer Launched in 2014
Co-primary endpoints
Ra
nd
om
ize
2 to
1 03
option
Patients (N=141)
PFS and OS in ASNS 0/1
subpopulation
Key secondary endpoints
• PFS and OS (all comers)
• Objective response rate
• Disease control rate
• Safety and tolerability
• Quality of life
Eryaspase +
chemotherapy
(gemcitabine or
mFOLFOX)
Chemotherapy alone
(gemcitabine or
mFOLFOX)
• ≥18 years
• Stage III or IV
• One prior systemic
chemotherapy in
advanced setting
• Measurable disease
• ECOG PS 0 or 1
Reference: ClinicalTrials.gov: NCT02195180
ECOG: Eastern Cooperative Oncology Group, PS: performance status, FOLFOX: 5FU, leucovorin and oxaliplatin; GERCOR: Multidisciplinary Group in Oncology
ASNS 0/1 subpopulation: patients with no/low asparagine synthetase expressing tumors
Stratified by chemotherapy
14
Trial in collaboration with the GERCOR in 16 clinical sites in France
15
40%reduction in risk of death (HR 0.60; P=0.008)
I T T P O P U L A T I O N
Eryaspase +
chemotherapy
(n=95)
Chemotherapy
(n=46)
Events, n (%) 82 (86.3) 42 (91.3)
Median OS,
months
(95% CI)
6.0
(4.8–6.6)
4.4
(3.0–5.0)
P value 0.008
0
10
20
30
40
50
60
70
80
90
100
Surv
ival pro
babili
ty (
%)
Months0 2 4 6 8 10 12 14 16 18 20 22 24 26 28 30 32 34 36
No. at risk
E +CT 95 83 67 45 29 24 15 11 8 8 4 4 4 1 1 1 1 0
CT 46 35 22 15 8 6 3 0
For reference: Onyvide Phase 3: HR 0.67
I T T P O P U L A T I O N
Eryaspase Phase 2b Trial
1. Hammel et al., European Journal of Cancer, 2019;
Events n (%) 79 (83%) 40 (87%)
OS HR (95% CI)
P-value
0.60 (0.40, 0.88)
0.008
Median OS (mo) 6.0 4.4
OS rate at 1yr
OS rate at 18m
15.8%
8.4%
6.5%
0%
Clear Overall Survival (OS) Benefit1
15
HR, Hazard Ratio; OS, Overall Survival
16
All patients (E+CT n=95, CT n=46)
ASNS 0/1+ (E+CT n=66, CT n=32)
ASNS 2+/3+ (E+CT n=29, CT n=14)
Male (E+CT n=53, CT n=30)
Female (E+CT n=42, CT n=16)
<6 months (E+CT n=31, CT n=14)
≥6 months (E+CT n=64, CT n=32)
0 (E+CT n=29, CT n=11)
1 (E+CT n=63, CT n=32)
<3 (E+CT n=90, CT n=44)
Normal (E+CT n=19, CT n=6)
Elevated (E+CT n=66, CT n=31)
mFOLFOX6 (E+CT n=11, CT n=5)
Gemcitabine (E+CT n=84, CT n=41)
OR+SD (E+CT n=63, CT n=30)
PD (E+CT n=30, CT n=16)
0.60 (0.40, 0.87)
0.63 (0.39, 1.01)
0.52 (0.26, 1.03)
0.47 (0.29, 0.77)
0.95 (0.51, 1.79)
0.35 (0.16, 0.73)
0.76 (0.48, 1.20)
0.78 (0.37, 1.66)
0.52 (0.33, 0.82)
0.61 (0.41, 0.90)
0.25 (0.07, 0.88)
0.79 (0.50, 1.25)
0.21 (0.05, 0.82)
0.65 (0.43, 0.97)
0.79 (0.50, 1.26)
0.44 (0.20, 0.94)
1 Hazard Ratio and 95% CI
ASNS category
Gender
Time from randomization
ECOG
Number of metastatic sites
CA19.9 level at baseline
Chemotherapy type
Response to prior therapy
I T T P O P U L A T I O N
Eryaspase Phase 2b Trial
Overall Survival Benefit Consistent Across Subgroups1
161. Hammel et al., European Journal of Cancer, 2019;
17
I T T P O P U L A T I O N
Pro
gre
ssio
n-f
ree s
urv
iva
l pro
babili
ty (
%)
0
10
20
30
40
50
60
70
80
90
100
Months0 4 6 8 10 122 14 16
No. at risk
E +CT 95 36 18 12 6 4 3 0
CT 46 9 6 1 1 1 0
Eryaspase + chemotherapy
(n=95)
Chemotherapy (n=46)
Events, n (%)
70 (73.7)
36 (78.3)
Median PFS, months
(95% CI) 2.0
(1.8–3.4)1.6
(1.4–1.8)
P value 0.006
Similar Progression-Free Survival Benefit1
44%reduction in the risk of progression
(HR, 0.56; P=0.005)
I T T P O P U L A T I O N
Eryaspase Phase 2b Trial
Events n (%) 70 (74%) 36 (78%)
OS HR (95% CI)
P-value
0.56 (0.37, 0.84)
0.005
Median PFS
(weeks)
8.6 6.9
1. Hammel et al., European Journal of Cancer, 2019;
18
47,4
11,6
23,9
6,5
0
5
10
15
20
25
30
35
40
45
50
Disease control rate Overall response rate
Pa
tie
nts
, %
Eryaspase +chemotherapy
Chemotherapy
I T T P O P U L A T I O N
Eryaspase Phase 2b Trial
new lesions
new lesions
** CR**
PD
SD
OR
PD
SD
ORPD = Progressive disease
SD = Stable disease
PR = Partial response
CR= Complete response
OR = Overall response (PR +CR)
DCR = Disease control rate (OR+SD)
OROR
SD
SD
Improved Response Rates and Fewer New Lesions1
181. Hammel et al., European Journal of Cancer, 2019;
19
No Additional Toxicity Over Chemotherapy Alone
0 10 20 30 40 50 60 70 80
Asthenia
Nausea
Anemia
Vomiting
Thrombocytopenia
Abdominal pain
Diarrhea
Decreased appetite
Pyrexia
Constipation
Neutropenia
GGT increased
Physical health deterioration
Antibody test positive
Weight decreased
Peripheral edema
Upper abdominal pain
Stomatitis
ALT increased
Hypokalemia
Neuropathy peripheral
Fatigue
Back pain
Cough
Mucosal inflammation
Alopecia
Hyperthermia
Lymphopenia
Hyperglycemia
AST increase
Anxiety
Hypoalbuminemia
Insomnia
All grade adverse events (%)
Chemotherapy
Eryaspase +chemotherapy
> Grade 3/4 >=1 SAE
50%45%
77%
86%
Patients with ≥1 Grade 3 or 4 AE
Patients with≥1 SAE
TREATMENT EMERGENT EVENTS
REGARDLESS OF RELATIONSHIP TO
STUDY DRUG
Eryaspase Phase 2b TrialI T T P O P U L A T I O N
19
20
Reference: ClinicalTrials.gov: NCT03665441
ECOG, Eastern Cooperative Oncology Group; PS, performance status
FOLFIRI, 5FU/leucovorin/irinotecan; the irinotecan can be Onivyde (nanoliposomal irinotecan or NALIRI)
03
option
Patients (N ≈ 500)
• ≥18 years
• Stage III or IV PAC
• One prior systemic
chemotherapy in
advanced setting
• Measurable disease
• ECOG PS 0 or 1
Primary endpoint
Ra
nd
om
ize
1:1
Chemotherapy
(gemcitabine+nabpaclitaxel
or FOLFIRI)
plus eryaspase
• Overall Survival
Key secondary endpoints
• Progression-free survival
• Objective response rate
• Disease control rate
• Safety and tolerability
• Quality of life
Chemotherapy alone
(gemcitabine+nabpaclitaxel
or FOLFIRI)
Stratification by ECOG PS, chemotherapy regimen
and time since diagnosis of advanced disease
TRYbeCA-1, Phase 3 trial in 2L Pancreatic Cancer Launched in 2018
Pascal Hammel Manuel HidalgoCo-PI, Weil Cornell Medicine, New York, U.S.Co-PI, Hôpital Beaujon, Paris, France
20
Trial expected to run in approximately 100 clinical sites in 11 European countries and the U.S.
21
• More than 75% of total expected projected patients enrolled
• On track for full enrollment in Q4 2020*
• Trial approved and enrolling patients in 11 countries in Europe and the United States
• 65+ clinical sites activated
• Safety data of first 150 and 320 patients reviewed by independent data monitoring committee in
October 2019 and April 2020 respectively:
• No safety issues identified
• Recommendation to continue trial as planned
• Interim analysis (for superiority) expected around year-end 2020*
• Based on comparison of OS when 2/3 of events will have occurred
• Two outcomes possible: continue as planned or stop for superiority (no futility analysis planned)
TRYbeCA-1 on Track for Complete Enrollment in 4Q20
21
*subject to COVID-19 uncertainty
22
Phase 2 proof of concept trial ongoing in Triple-Negative Breast Cancer (TNBC) in Europe
• Approximately 64 patients with newly diagnosed metastatic disease
• Randomized 1-1: chemotherapy (gemcitabine/carboplatin) +/- eryaspase
• Primary endpoint: objective response rate
• Approximately 20 sites activated
• Results expected in 2021
Phase 1 IST in 1L pancreatic cancer in the United States in preparation (N=12-18)
• Evaluation of safety of eryaspase in combination with FOLFIRINOX
• Launch of trial expected in 2H 2020
Phase 2 IST in 2L ALL in Nordic countries of Europe nearing complete enrollment (N=50)
• Interim update expected in 1H 2020, full results by year-end 2020
Broadening Scope to Other Indications
Head of the Medical Oncology
Clinic at Jules Bordet Cancer
Institute Brussels, Belgium
PI: Ahmed Awada
22
23
Own cGMP production in Europe and US, scaled
for clinical and early-commercial manufacturing
• Europe: Lyon (France)
• 12 cleanrooms
• United States: Princeton, NJ
• 16 cleanrooms, 4 equipped
Industrialized and Scalable Manufacturing
23
24
Preclinical Programs
Erymethionase, methionine-gamma-lyase encapsulated in RBC
• Promising preclinical data in gastric cancer and glioblastoma
suggest potential as a new treatment approach against cancers
that rely on methionine metabolism
• Further preclinical data show potential synergistic effect of
methionine restriction with immune checkpoint inhibitors and
asparaginase
ERYZYME, encapsulating enzymes used in certain enzyme
therapies to treat rare metabolic diseases
• E.g. Arginase-1-deficiency: preclinical data showing sustained
lowering of arginine levels with arginine deiminase encapsulated
in RBC in arginase deficient mice
Gastric adenocarcinoma
Days post-tumor implantation
Me
an t
um
or
volu
me
(m
m3+
SE
M)
0 10 20 30 40 50 60 70 80
0
200
400
600
800
1000
1200
1400
1600
1800
Vehicle
Erymethionase 80 U/kg
***
****
**
Erymethionase + PN
Tumor regression in gastric cancer model
25
Strategic Collaboration with SQZ Biotechnologies
25
• ERYTECH granted SQZ Biotechnologies, a Cambridge (MA) based cell therapy
company, an exclusive worldwide license to develop antigen-specific immune
modulating therapies employing red blood cell-based technology.
• SQZ to develop RBC-derived therapeutics to induce antigen-specific immune
modulation by combining SQZ’s Cell Squeeze® cell engineering platform with
ERYTECH’s IP and knowhow related to RBC-based therapeutics
• ERYTECH is eligible to receive:
• Upfront and potential milestone payments up to $57 million for the first product
successfully developed by SQZ under agreement
• Royalties on sales, and potential commercial milestone payments up to $50
million for each additional approved product or approved indication
26
FY 2019 Financial Results
26
In thousands of euros FY 2019 FY 2018
Revenues — —
Other income 5,283 4,447
Total operating income 5,283 4,447
Research and development (52,193) (33,467)
General and administrative (17,164) (14,600)
Total operating expenses (69,357) (48,067)
Total operating loss (64,074) (43,621)
Financial income 2,947 5,427
Financial expenses (1,533) (29)
Financial income (loss) 1,414 5,399
Loss before tax (62,660) (38,222)
Income tax 1 (2)
Net loss (62,659) (38,224)
• Net loss of €62.7 million, up €24.5 million
compared to 2018
• Cash position of €73.2 million ($82.2 million)
as of December 31, 2019, compared with
€134.4 million on December 31, 2018 and
€81.9 million at September 30, 2019.
• The €61.2 million decrease in cash position
during the twelve months of 2019 was
mostly comprised of a €43.3 million net
cash utilization in operating activities, and
€19.8 million used for investing activities,
• Cash runway confirmed into Q1 2021
Q1 2020 Financial Highlights
• As of March 31, 2020, cash and cash equivalents totaling €58.6 million (approximately $64.6 million),
• Compared with €73.2 million on December 31, 2019.
• The €14.6 million decrease in cash position in the first quarter 2020 was the result of:
– €16.7 million net cash utilization in operating activities, inline with operating plan;
– €1.1 million used for investing activities;
– €2.4 million generated in financing activities
– €0.7 million favorable currency exchange impact of the U.S. dollar against the euro
• While closely monitoring the budget impact of the COVID-19 pandemic on its operations, the Company
confirms its earlier guidance on sufficient cash position to fund operations into Q1 2021.
28
Key Milestones Anticipated over Next 12 Months
Interim update on Phase 2 IST in 2L acute lymphoblastic leukemia (ALL)
Initiation of Phase 1 IST with eryaspase in 1L pancreatic cancer
Interim (superiority) analysis in TRYbeCA-1
Full results of Phase 2 IST in 2L ALL
Interim results of Phase 1 IST in 1L pancreatic cancer
28
ERYTECH Pharma SA60 Avenue Rockefeller
69008 LyonFrance
ERYTECH Pharma Inc1 Main Street
Cambridge, MA 02142USA
www.erytech.com