Erytech Pharma established a research collaboration with Queen’s University in Canada to support continued preclinical development of its eryminase candidate for treating the rare metabolic disorder arginase-1 deficiency. Eryminase comprises the enzyme arginine deiminase encapsulated in red blood cells using Erytech’s proprietary Erycaps platform technology.

The collaboration with Queen’s University aims to generate in vivo proof-of-concept that eryminase can lower arginine levels in an inducible arginase-1 deficiency mouse, which has been developed at the University.

Alexander Scheer, Ph.D., CSO at Erytech, commented, “This is our second collaboration in the field of rare metabolic diseases that underscores the scope of our platform and its applicability to highly specialized and rare conditions beyond oncology. We are very pleased to enter this collaboration with Queen’s University and look forward to working closely on this important program with Dr. Funk who specializes in research related to urea cycle disorders.”

Queen’s University’s professor Colin Funk, Ph.D., added, arginase-1 deficiency is a severe, rare disorder affecting a biochemical pathway that disposes of toxic ammonia. Normally, our bodies are very efficient at removing any ammonia that accumulates after eating a protein-rich meal. However, in patients with arginase-1 deficiency, the ammonia is partially detoxified leading to a large accumulation of the amino acid arginine in the patient’s blood and brain. Erytech’s product candidate eryminase aims to reduce the level of arginine in blood and thus has significant potential to reduce negative consequences of this disorder. 

French biopharma Erytech is focused on developing Erycaps-formulated treatments for rare forms of cancer and orphan diseases. The firm’s primary therapeutic focus is blood cancers, including acute lymphoblastic leukemia (ALL) and acute myeloid leukemia (AML). Lead candidate eryaspase (Graspa®) is designed to deprive leukemic cells of asparagine and has successfully completed a European pivotal Phase II/III study in children and adults with relapsed and refractory ALL. A Phase I trial is ongoing in the U.S. to evaluate eryaspase combined with chemotherapy in newly diagnosed adult ALL patients. A Phase IIb European study assessing eryaspase plus chemotherapy in elderly patients with newly diagnosed AML is also ongoing. An investigator-initiated study evaluating eryaspase in ALL patients was started in collaboration with the Nordic Society of Pediatric Hematology and Oncology (NOPHO) in April.

Eryaspase has been granted orphan drug designation for the treatment of ALL, AML, and pancreatic cancer. A Phase IIb trial evaluating eryaspase as second-line therapy for pancreatic cancer has also been completed, with positive results reported.  

In November last year, Erytech withdrew its marketing authorization application (MAA) in Europe for eryaspase in the treatment of AML. The firm said it would not be able to provide the additional data requested by the European Medicine’s Agency’s (EMA) Committee for Human Medicinal Products (CHMP) Day 180 List of Outstanding Issues in the allotted time frame. At the time, Erytech  envisaged resubmitting the MAA around mid-2017.

Erytech is also investigating the potential to use its platform for developing immuno-oncology and for enzyme therapies. In March, the firm established a collaboration with Fox Chase Cancer Center (FCCC) to support preclinical development of its erymethionase candidate for treating homocystinuria. 


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