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
Investor Conference Call at
“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.
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
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 manufactured utilizing Arcturus’ proprietary processes for both mRNA drug substance and LUNAR® formulated drug product.
Investor Conference Call:
Today's call will provide additional detail pertaining to the ARCT-810 clinical plan, along with additional information regarding the Company’s COVID-19 vaccine program.
ARCT-810, Arcturus’ first development candidate, represents a novel approach to treat ornithine transcarbamylase deficiency. ARCT-810 is based on Arcturus’ mRNA design construct and proprietary manufacturing process. ARCT-810 also utilizes Arcturus’ extensive and propriety lipid library and employs the Company's LUNAR® delivery platform to deliver OTC mRNA to hepatocytes. ARCT-810 is an investigational mRNA medicine designed to enable OTC-deficient patients to naturally produce healthy functional OTC enzyme in their own liver cells. Replacing the deficient OTC protein has the potential to restore activity of the urea cycle pathway, resulting in reduced plasma ammonia and urinary orotate concentrations.
About Ornithine Transcarbamylase Deficiency
Ornithine transcarbamylase (OTC) deficiency is the most common urea cycle disorder. Urea cycle disorders are a group of inherited metabolic disorders that make it difficult for affected patients to remove toxic waste products as proteins are digested. OTC deficiency is caused by mutations in the OTC gene which leads to a non-functional or deficient OTC enzyme. OTC is a critical enzyme in the urea cycle, which takes place in liver cells, and together with the other enzymes in the urea cycle 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. A lack of the OTC enzyme in liver cells results in high blood ammonia levels and can cause seizures, coma, and death in untreated patients. 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 males. 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.
Founded in 2013 and based in
Forward Looking Statements
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, including those regarding strategy, future operations, collaborations, the likelihood of success efficacy or safety of ARCT-810, the ability to initiate or complete preclinical and clinical development programs, including as a result of the COVID-19 pandemic, the supply and delivery of any product or substance, the likelihood that preclinical data will be predictive of clinical data, and the ability to enroll subjects therein are forward-looking statements. Arcturus may not actually achieve the plans, carry out the intentions or meet the expectations or projections disclosed in any forward-looking statements such as the foregoing and you should not place undue reliance on such forward-looking statements. Such statements are based on management’s current expectations and involve risks and uncertainties, including those discussed under the heading "Risk Factors" in Arcturus’ Annual Report on Form 10-K for the fiscal year ended
Source: Arcturus Therapeutics Holdings Inc.