Corporate Presentation September 2016

Disclaimer The statements made in this presentation may include forward-looking statements regarding the future 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 of planned regulatory submissions, manufacturing capabilities and strategy for expansion of the ERYCAPS platform. Although we believe that the expectations contained in this presentation are reasonable, these forward-looking statements are only estimations based upon the information available to ERYTECH Pharma S.A. as of the date of this presentation. Except as required by law, we expressly disclaim any responsibility to publicly update or revise our forward-looking statements, whether as a result of new information, future events or otherwise. Thus, the forward-looking statements herein involve known and unknown risks and uncertainties and other important factors such that actual future operations, opportunities or financial performance may differ materially from these forwardlooking statements. Undue reliance should not be placed on forward looking statements, which speak only as of the date hereof. All forward-looking statements contained herein are qualified in their entirety by the foregoing cautionary statement.

2

ERYTECH, a late-stage orphan oncology company Innovative and versatile ERYCAPS technology platform Encapsulation of therapeutic compounds in red blood cells (RBC) Strong IP protection Broadly applicable

Lead product eryaspase (GRASPA®) in late stage clinical development MAA in ALL under review by EMA Phase 2b in AML Phase 2 in pancreatic cancer US Phase 1 in ALL

Targeting markets with high unmet medical needs Acute leukemia (ALL & AML) Other rare cancers Orphan diseases

Growth beyond lead programs Global expansion, focus on US Other indications Other pipeline products Attractive platform opportunities

ALL: Acute Lymphoblastic Leukemia; AML: Acute Myeloid Leukemia; EMA: European Medicines Agency; MAA: Marketing Authorization Application 3

ERYTECH: key corporate facts Key Corporate Facts • • • •

Global Presence

Founded in 2004; HQ in Lyon, France; office in Cambridge, US 75 FTEs, including 30% PhD/MD Manufacturing in Lyon and Philadelphia Commercialization partnerships with Recordati in EU and TEVA in Israel

Boston Philadelphia

Lyon

Offices cGMP units

•

-

•

Shareholder Breakdown

IPO May 2013 on Euronext Paris € 18 million raised

Secondary offerings Oct 14: € 30 million raised; 68% in US - Dec 15: € 25 million raised; 61% in US -

• •

Market Cap(1): € 158 M Cash: € 35.6 million at end of Q2 2016

(1) As at September 8, 2016

4

ERYCAPS, an innovative and versatile technology platform Entrapment of drug substance inside donor-derived red blood cells using hypotonic/hypertonic stress Proprietary ‘osmotic fragility’ process ensures required amount of drug in each RBC batch Molecules from 1 to 500 kDalton (peptides, enzymes, antigens, ...)

Controlled lysis (hypotonic stress)

Protected by 13 patent families

Resealing (hypertonic stress)

Industrialized in commercial scale GMP manufacturing facility 5

ERYCAPS, an industrialized process ready for commercialization

Photo salle de contrôle 1

Prescription

Sourcing

2

Purchase of pack of compatible RBC

3

Preparation

Encapsulation

Identification of the key parameters(1)

Automated process 3 - 8 hours

4

Batch release

5

Shipping

Quality control Release by QP

Patient

Est. 24 hours

•

Centralized EU GMP production facility in Lyon, France. Sized for the first two years of marketing. GMP & ISO certified

•

US production facility in Philadelphia, at the premises of the American Red Cross, validated for clinical trial production

(1) Measurement of osmotic fragility, verification of blood typing group documents, expiry date, volume, hematological parameters.

6

Late stage clinical pipeline focused on tumor starvation therapies Mode of action

Product Candidate/ Program

Drug substance

Indication Discovery

Preclinical

EU US

Tumor starvation

AML

EU then EU/US

NHGlobal lymphoma

Immunotherapy Enzyme replacement

Methionineγ-lyase

TBD

eryadi

Arginine deiminase

TBD

ERYMMUNE

Tumor antigens

TBD

ERY-ERT

Therapeutic enzymes

TBD

Phase 3/ EMA/FDA Pivotal review

Commercial Rights

Israel

Pancreatic EU then EU/US cancer

erymet

Phase 2

Europe

ALL

Eryaspase Asparaginase (GRASPA®(1))

Phase 1

(1) Brand name for eryaspase in Europe and Israel 7

US and RoW

Asparaginase, cornerstone treatment in ALL therapy

Concept

•

Target

•

•

Therapy •

•

Limitations •

Starving cancer cells by affecting their supply of essential nutrients Asparagine, essential nutrient for most tumor cells (1), but not for normal cells Asparaginase, degrades asparagine and deprives tumor cells of asparagine Proven efficacy; cornerstone in Phi-neg pediatric ALL therapy Important side effects (allergies, coagulation disorders, pancreatic and hepatic toxicities) limiting treatment utility, especially in patients with poor performance status Neutralizing antibodies leading to decreased efficacy

(1) Asparagine synthetase (ASNS) deficient cells, unable to synthetize asparagine. 8

Current forms of asparaginase essentially used in children •

Three asparaginase products marketed: native L-asparaginase, Oncaspar and Erwinaze

•

Asparaginase use generally follows a “cascade schema” (1) -

Toxicity limits use of asparaginase in patients with poor performance status: children in relapse, adults and elderly ALL patients Number of patients(2) (EU & US)

•

Start treatment with native asparaginase or PEG asparaginase, both E. coli derived Switch to Erwinaze if administration of E. coli-asparaginase no longer tolerated Omit from chemotherapy protocols if further asparaginase treatment not feasible

12 000 10 000 8 000 6 000 4 000 2 000 0

(1) (2)

New ALL cases per year (incl relapse) Treated with E.coli aspa (native and PEG) Treated with Erwinaze Children (55) 9

eryaspase (GRASPA®), a novel ‘bioreactor’ to degrade asparagine Prolonged activity and reduced toxicity thanks to encapsulation of the asparaginase inside the RBC

Y

The circulating asparagine is actively pumped into the RBC…

Longer activity Less toxicity

Asparagine Asparaginase Antibodies

The RBC membrane prevents interactions between the asparaginase and the antibodies

…where the encapsulated asparaginase hydrolyses it to aspartate and ammonia

10

Prolonged activity and reduced toxicity to enable paradigm shift

eryaspase to meet the needs of patients currently not receiving asparaginasebased treatments ALL

Enable use of asparaginase in adults and elderly Unmet needs: ca 50% of patients currently not treated with asparaginase products

Current asparaginases:

Est. >17,000(1) cases per year (EU & US)

Provide safer asparaginase to relapsed children and adults

Native asparaginase & Oncaspar® (est. $200 million) Erwinase (est. $200 million) Children

eryaspase, attractive alternative to current asparaginases

Adults

Become asparaginase of choice in first line ALL

(1) Newly diagnosed and relapsed ALL patients. Source: company estimates based on American Cancer Society (www.cancer.org) data for US and Surveillance of Rare Cancers in Europe (www.rarecare.eu) data 11 for Europe

EMA review of MAA for GRASPA for treatment in R/R ALL ongoing • • •

•

Pivotal Phase 2/3 study comparing safety and efficacy of GRASPA vs native Lasparaginase in relapsed & refractory ALL patients (1 to 55 years of age; N=80) Study completed in 2014. Full study results presented in plenary session at ASCO 2015; 3 poster presentations at ASH 2015 Key conclusion: GRASPA is an effective alternative option for patients who have received prior asparaginase therapy Co-primary endpoints - Significantly reduced risk of allergic reactions (0% vs 46%; p