Arcturus Therapeutics Announces Allowance of IND & Approval of Clinical Trial Application (CTA) for ARCT-810, a First-in-Class Investigational mRNA Medicine to Treat Ornithine Transcarbamylase Deficiency
Clinical Plan Includes Healthy Volunteers in New Zealand and OTC-Deficient Patients Across Several Sites in United States
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SAN DIEGO, April 13, 2020 (GLOBE NEWSWIRE) -- Arcturus Therapeutics (the “Company”, NASDAQ: ARCT), a leading clinical-stage messenger RNA medicines company focused on the discovery, development and commercialization of therapeutics for rare diseases and vaccines, today announced the acceptance of two clinical trials for its flagship asset ARCT-810, also known as LUNAR-OTC, a first-in-class mRNA therapeutic being developed to treat ornithine transcarbamylase (OTC) deficiency. The Company’s Investigational New Drug (IND) application for a Phase 1b study in patients with OTC deficiency was allowed to proceed by the U.S. Food and Drug Administration (FDA), and an additional Clinical Trial Application (CTA) for a Phase 1 study in healthy volunteers was approved by the New Zealand Medicines and Medical Devices Safety Authority (Medsafe). OTC deficiency is a life-threatening genetic disease that results in high blood ammonia levels and can cause seizures, coma, and death in untreated patients. Present standard of care, which comprises low protein diet and drugs to remove toxic ammonia from the body, does not effectively prevent life-threatening spikes of ammonia in many patients. There are no disease modifying therapies approved for OTC deficiency.
“Allowance to proceed into human trials represents a significant milestone for Arcturus as we become a clinical-stage company with a candidate that may provide new hope to patients suffering from ornithine transcarbamylase deficiency,” said Joseph Payne, President & CEO of Arcturus Therapeutics.
Dr. Steve Hughes, Chief Development Officer of Arcturus, stated, “Arcturus continues to establish itself as a world leader in the field of intravenously-dosed messenger RNA therapeutics. Our team looks forward to ushering ARCT-810 efficiently through the clinic and to providing OTC-deficient patients access to this potentially disease-modifying messenger RNA therapy.”
The primary endpoint for both studies includes evaluation of safety and tolerability. Multiple biomarkers, including ureagenesis assay, plasma OTC activity, plasma ammonia and orotic acid in the urine, are being evaluated as exploratory endpoints. The program plans to enroll up to 30 healthy volunteers in the Auckland Clinical Studies (ACS) site in New Zealand, and up to 12 OTC-deficient patients recruited across several sites in the U.S. The first healthy subjects are expected to be enrolled in New Zealand soon, with the first patients enrolled under the IND in Q3 or Q4, depending on the status of SARS-CoV-2 infections in the U.S.
ARCT-810, is a low-dose, systemically administered, investigational mRNA medicine that utilizes Arcturus' novel messenger RNA construct and proprietary LUNAR® delivery system to deliver OTC messenger RNA to liver cells. In 2019, the FDA granted Orphan Drug Designation to the drug substance of ARCT-810 for the treatment of the rare disease OTC deficiency supported by the promising results of preclinical studies. Expression of OTC enzyme in the liver can potentially restore urea cycle activity to detoxify ammonia, thereby potentially preventing neurological damage and removing the need for liver transplantation.
The GMP manufacturing campaign for ARCT-810 is complete, with drug product amounts sufficient to support early clinical trials. ARCT-810 batches were manufactred utilizing Arcturus’ proprietary processes for both mRNA drug substance and LUNAR® formulated drug product.
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