Arcturus Therapeutics Reassumes Full Worldwide Rights to ARCT-810, a Clinical Development Candidate for Ornithine Transcarbamylase (OTC) Deficiency
The preclinical development program for ARCT-810, including Investigational New Drug Application (IND) enabling studies, remains on track. Arcturus is planning to file an IND for ARCT-810 with the
CureVac remains committed to developing additional assets within the Arcturus collaboration utilizing CureVac mRNA and Arcturus LUNAR® delivery technology. CureVac and Arcturus will announce when the next program has been selected.
“We have had a very productive collaboration with CureVac on ARCT-810 and are pleased that we have secured all of the rights to this clinical development candidate. We are enthusiastic about the value of ARCT-810 and we believe it has the potential to be a transformational treatment for patients suffering from OTC deficiency,” said
Arcturus plans to present further data and updates on the progress of ARCT-810 at the “TIDES: Oligonucleotide & Peptide Therapeutics” conference, to take place
ARCT-810, Arcturus’ first development candidate, represents a novel approach to treat ornithine transcarbamylase deficiency (OTCD). ARCT-810 is based on mRNA and also utilizes Arcturus’ propriety lipid library and employs the Company's LUNAR® delivery platform to safely and effectively deliver OTC mRNA to hepatocytes. ARCT-810 is an mRNA replacement therapy designed to enable OTC-deficient patients to naturally produce healthy functional OTC enzyme in their own liver cells. Arcturus is currently on track to submit an Investigational New Drug Application (IND) to the FDA in the fourth quarter of 2019. ARCT-810 is advancing toward the clinic on the strength of preclinical proof-of-concept data, demonstrating that LUNAR technology can deliver mRNA to liver cells and results in expression of functional OTC protein in animal models. Replacing the deficient OTC protein restores the urea cycle pathway, resulting in reduced plasma ammonia and urinary orotate concentrations.
About Ornithine Transcarbamylase Deficiency (OTCD)
OTC deficiency is caused by mutations in the OTC gene which leads to a non-functional or deficient OTC enzyme. OTCD is the most common urea cycle disorder. Urea cycle disorders are a group of inherited metabolic disorders that make it difficult for afflicted patients to remove toxic waste products, as proteins are digested. OTC deficiency is a life-threatening genetic disease. OTC is a critical enzyme in the urea cycle, which takes place in liver cells, and converts ammonia to urea. This conversion does not occur properly in patients with OTC deficiency and ammonia accumulates in their blood, acting as a neurotoxin and liver toxin. This can cause severe symptoms including vomiting, headaches, coma and death. OTC deficiency is an inherited disease that can cause developmental problems, seizures and death in newborn babies. It is an X-linked disorder, so is more common in boys. Patients with less severe symptoms may present later in life, as adults. There is currently no cure for OTC deficiency, apart from liver transplant. However, this treatment comes with significant risk of complications such as organ rejection, and transplant recipients must take immunosuppressant drugs for the rest of their lives. Current standard of care for OTC patients is a low-protein diet and ammonia scavengers to try and prevent patients from accumulating ammonia. These treatments do not address the underlying cause of disease.
About Arcturus Therapeutics Ltd.
Founded in 2013 and headquartered in San Diego, California,
This press release contains “forward-looking statements” that involve substantial risks and uncertainties for purposes of the safe harbor provided by the Private Securities Litigation Reform Act of 1995. Any statements, other than statements of historical fact, included in this press release regarding strategy, future operations, collaborations, future financial position, prospects, plans and objectives of management, including statements relating to the status of the preclinical development program for ARCT-810, the date that an IND may be filed with the
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