ERYTECH to Present Pre-Clinical Data at the 13th International Congress of Inborn Errors of Metabolism

Regulatory News:

ERYTECH Pharma (Paris:ERYP) (ADR:EYRYY) (Euronext Paris - ERYP), a clinical-stage biopharmaceutical company developing innovative therapies by encapsulating therapeutic drug substances inside red blood cells, today announced that two abstracts on its preclinical erymethionase and eryminase programs were accepted for poster presentation at the 13^th International Congress of Inborn Errors of Metabolism (ICIEM), being held September 5 – 8, 2017 in Rio De Janeiro, Brazil.

The pre-clinical data will be presented during the poster session of the ICIEM by the lead author of the abstracts, Dr. Emmanuelle Dufour, R&D Project Manager at ERYTECH. The data from the pre-clinical studies demonstrate that encapsulating certain enzymes into red blood cells has the ability to lower toxic metabolites:

• Eryminase, which is arginine deiminase encapsulated in red blood cells, was shown to reduce arginine levels in a disease model of arginase-1 deficiency, supporting a potential treatment approach for hyperargininemia. The study was conducted through a previously announced research collaboration with Queen’s University in Canada.
• Erymethionase, methionine-?-lyase encapsulated in red blood cells, was shown to lower homocysteine levels, supporting a potential treatment approach for homocystinuria. The study was conducted through a previously announced research collaboration with the Fox Chase Cancer Center (FCCC).

Both hyperargininemia and homocystinuria are rare, debilitating genetic diseases, for which there are limited treatment options.

ERYTECH will also be participating in and sponsoring the 2^nd International Homocystinurias Patient-Expert Meeting on Monday, September 4, 2017, a pre-meeting to the ICIEM.

Poster session details to follow:

Poster Sessions: Eryminase, Arginine Deiminase-Encapsulated in Red Blood Cells Effectively Lower Blood Arginine Levels in a Mouse Model of Inducible Hyperargininemia

Poster Sessions: Erymethionase, Methioninase Entrapped in Red Blood Cells: an innovative treatment approach for classical homocystinuria

The poster presentations will be accessible as of September 6, 2017, on ERYTECH’s website at www.erytech.com.

About Homocystinuria

Classical homocystinuria is a rare, inherited genetic disease caused by a deficiency in the enzyme cystathionine beta-synthase (CBS), which is critical for methionine metabolism. Patients are unable to fully metabolize the amino acid methionine, an essential amino acid found in food, which leads to the accumulation of homocysteine and methionine in the blood and urine. High levels of these amino acids are directly linked to morbidity and mortality, often at a young age. Symptoms include severe intellectual disability, eye lens dislocation, thromboembolism, osteoporosis, and seizures.

ERYTECH has entered into a research collaboration with the Fox Chase Cancer Center (FCCC) to demonstrate the potential of ERYTECH’s erymethionase to lower homocysteine and methionine in the homocystinuria mouse model (CBS-deficient mice) developed by Prof. Warren Kruger’s lab at FCCC. Erymethionase is a methionine gamma-lyase (MGL, methioninase) enzyme encapsulated in red blood cells using ERYTECH’s proprietary ERYCAPS platform technology to provide effective, long-acting therapeutic activity with reduced toxicity.

About Hyperargininemia

Hyperargininemia, or arginase-1 deficiency is a rare, inherited disorder of the urea cycle caused by a mutation in the arginase-1 gene, resulting in the accumulation of toxic levels of the amino acid arginine in the blood. Symptoms generally appear in early infancy and include intellectual disability, non-ambulatory muscle stiffness and seizures. Hyperargininemia is a debilitating, progressive disease with very limited treatment options currently available.

ERYTECH, working in collaboration with Queen’s University, hopes to demonstrate the potential of ERYTECH’s eryminase to lower arginine in the inducible arginase-1 deficiency mouse model developed by the laboratory of Prof. Colin Funk of Queen’s University. Eryminase, a product candidate being developed by ERYTECH, consists of an arginine deiminase enzyme encapsulated in red blood cells using ERYTECH’s proprietary ERYCAPS platform technology. ERYTECH believes the encapsulation of therapeutic enzymes in red blood cells can provide effective, long-acting therapeutic activity with reduced toxicity.

About ERYTECH: www.erytech.com

Founded in Lyon, France in 2004, ERYTECH is a clinical-stage biopharmaceutical company developing innovative therapies for rare forms of cancer and orphan diseases. Leveraging its proprietary ERYCAPS platform, which uses a novel technology to encapsulate therapeutic drug substances inside red blood cells, ERYTECH has developed a pipeline of product candidates targeting markets with high unmet medical needs. ERYTECH’s initial focus is on the treatment of blood cancers, including acute lymphoblastic leukemia (ALL) and acute myeloid leukemia (AML), by depriving tumors of nutrients necessary for their survival. ERYTECH, which plans to pursue regulatory approvals for its lead product candidate, eryaspase, also known under the trade name GRASPA^®, reported positive efficacy and safety results from its completed Phase 2/3 pivotal clinical trial in Europe in children and adults with relapsed or refractory ALL. ERYTECH also has an ongoing Phase 1 clinical trial of eryaspase in the United States in adults with newly diagnosed ALL, and a Phase 2b clinical trial in Europe in elderly patients with newly diagnosed AML, each in combination with chemotherapy. ERYTECH believes that eryaspase also has potential as a treatment approach in solid tumors and has completed a Phase 1 study and a Phase 2b clinical trial in France, evaluating eryaspase in patients with second line metastatic pancreatic cancer.

Eryaspase consists of an enzyme, L-asparaginase, encapsulated inside donor-derived red blood cells. L-asparaginase depletes asparagine, a naturally occurring amino acid essential for the survival and proliferation of cancer cells, from circulating blood plasma. ERYTECH produces eryaspase at its own GMP-approved and operational manufacturing site in Lyon (France), and at a site for clinical production in Philadelphia (USA). ERYTECH has entered into licensing and distribution partnership agreements for eryaspase for ALL and AML in Europe with Orphan Europe (Recordati Group), and for ALL in Israel with TEVA, which will market the product under the GRASPA^® brand name. The European Medicines Agency (EMA) and the U.S. Food and Drug Administration (FDA) have granted orphan drug designations for eryaspase for the treatment of ALL, AML and pancreatic cancer.

In addition to eryaspase, ERYTECH is developing two other product candidates that focus on using encapsulated enzymes to target cancer metabolism and induce tumor starvation. ERYTECH is also exploring the use of its ERYCAPS platform to develop cancer immunotherapies and enzyme replacement therapies.

ERYTECH is listed on Euronext regulated market in Paris (ISIN code: FR0011471135, ticker: ERYP) and is part of the CAC Healthcare, CAC Pharma & Bio, CAC Mid & Small, CAC All Tradable, EnterNext PEA-PME 150 and Next Biotech indexes. ERYTECH is also listed in the U.S. under an ADR level 1 program (OTC, ticker EYRYY).

Forward-looking information

This press release contains forward-looking statements, forecasts and estimates with respect to the clinical development plans, business and regulatory strategy, and anticipated future performance of ERYTECH and of the market in which it operates. Certain of these statements, forecasts and estimates can be recognized by the use of words such as, without limitation, "believes”, "anticipates”, "expects”, "intends”, "plans”, "seeks”, "estimates”, "may”, "will” and "continue” and similar expressions. They include all matters that are not historical facts. Such statements, forecasts and estimates are based on various assumptions and assessments of known and unknown risks, uncertainties and other factors, which were deemed reasonable when made but may or may not prove to be correct. Actual events are difficult to predict and may depend upon factors that are beyond ERYTECH's control. There can be no guarantees with respect to pipeline product candidates that the candidates will receive the necessary regulatory approvals or that they will prove to be commercially successful. Therefore, actual results may turn out to be materially different from the anticipated future results, performance or achievements expressed or implied by such statements, forecasts and estimates. Documents filed by ERYTECH PHARMA with the French Autorité des Marchés Financiers (www.amf-france.org), also available on ERYTECH’s website (www.erytech.com) describe such risks and uncertainties. Given these uncertainties, no representations are made as to the accuracy or fairness of such forward-looking statements, forecasts and estimates. Furthermore, forward-looking statements, forecasts and estimates only speak as of the date of this press release. Readers are cautioned not to place undue reliance on any of these forward-looking statements. ERYTECH disclaims any obligation to update any such forward-looking statement, forecast or estimates to reflect any change in ERYTECH’s expectations with regard thereto, or any change in events, conditions or circumstances on which any such statement, forecast or estimate is based, except to the extent required by law.

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