Erytech pens academic pact to advance rare disease drug

Erytech Pharma has formed a collaboration with Canada’s Queen’s University to advance its drug against the rare metabolic disorder arginase-1 deficiency. The agreement enables Erytech to test its eryminase program in the inducible arginase-1 deficiency mouse model developed at Queen’s.

Lyon, France-based Erytech is best known for encapsulating the asparaginase enzyme in red blood cells to treat acute lymphoblastic leukemia. But more recently the biotech has started applying its encapsulation technology to the treatment of metabolic diseases. The agreement with Queen’s is intended to further this early-stage work.

Erytech hopes to emerge from the collaboration with in vivo proof-of-concept data on eryminase in animal models. The French biotech originally tested eryminase, an encapsulation of arginine deiminase, in solid tumors. But as the enzyme catalyzes a reaction involving arginine, the amino acid that builds up to toxic levels in patients with arginase-1 deficiency, Erytech thinks it may have a future as a rare disease drug.

To test the theory, Erytech has turned to Colin Funk, Ph.D. The Queen’s professor’s laboratory has developed an inducible knockout strain of mice that have arginase-1 deficiency. If eryminase can help these mice clear arginine from their blood, it may also be able to treat a disease that causes intellectual disability, seizures and other symptoms in humans.

“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,” Funk said in a statement.

The collaboration with Funk is the second time in recent months Erytech has looked outside of its walls to drive its expansion into metabolic disease. In March, Erytech formed a collaboration with Fox Chase Cancer Center. As in the Queen’s pact, the FCCC collaboration is designed to deliver in vivo proof-of-concept data on an Erytech drug in a rare, genetic metabolic disorder. 

Erytech’s alliances give it a way to expand its therapeutic scope while remaining internally focused on cancer.