Arcturus Therapeutics had a melodramatic 2018 — the San Diego biotech’s CEO Joseph Payne was asked to leave for putting his interests before the RNA company, but he fought back with the support of shareholders, ousted the board that spearheaded his ouster and was reinstated. But under Payne, the drug developer has lost the support of collaborator CureVac in the development of a drug for the most common urea cycle disorder — ornithine transcarbamylase deficiency (OTCD), a life-threatening genetic disease.
CureVac and Arcturus joined forces early last year to co-develop up to four mRNA therapeutics for enzyme replacement and antibody generation to tackle rare diseases. Typically, mRNA molecules teach human cells to produce therapeutic proteins or to mount an immune response. Last month, Arcturus said it had elected to develop an mRNA replacement therapy, ARCT-810, which is designed to enable OTC-deficient patients to naturally produce healthy functional OTC enzyme in their liver cells.
Urea cycle disorders are a group of inherited metabolic conditions that thwart the removal of bodily toxic waste products as proteins are digested. OTC is a critical enzyme that converts ammonia to urea; with an OTC deficiency, ammonia accumulates in the blood, acting as a neurotoxin and liver toxin, and causing vomiting, headaches, coma and death.
On Monday, Arcturus $ARCT said CureVac had elected not to support the preclinical development of ARCT-810, but remains committed to their broad collaboration. An application to test the drug in humans is on track and slated for submission in the fourth quarter of 2019, said Arcturus, which also has pacts with Takeda and J&J $JNJ.
“Arcturus has the resources and expertise to advance this program into the clinic,” Payne said in a statement, highlighting that the CureVac partnership remains intact.
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