Arcturus Therapeutics Announces Initiation of Dosing ARCT-810 in Patients with Ornithine Transcarbamylase (OTC) Deficiency
The ongoing Phase 1b study will evaluate approximately twelve patients with OTC deficiency. ARCT-810 will be administered at doses between 0.2 mg/kg and 0.4 mg/kg, and all dose levels are expected to be within the therapeutic range, based on preclinical study data. The Phase 1b study will assess safety, tolerability and pharmacokinetics, as well as various exploratory biomarkers of drug activity.
Arcturus previously announced that it had completed the ARCT-810 Phase 1, dose escalation study in healthy subjects, at doses up to 0.4 mg/kg. In that study, ARCT-810 was found to be generally safe and well tolerated; no severe adverse events were observed, and no steroid pretreatment was required. The pharmacokinetic profile of ARCT-810 was favorable, and preliminary data shows that no ARCT-810 lipid was detectable in the plasma beyond 48 hours following drug administration.
“We are so encouraged by the progress of the ARCT-810 study in OTC deficiency and the promise this novel mRNA therapeutic approach holds for saving and improving the lives of patients affected with this devastating disorder,” said
“The initiation of dosing in the ARCT-810 Phase 1b clinical trial in patients with OTC deficiency builds upon the favorable prior Phase 1 study results, as well as encouraging preclinical data which showed that ARCT-810 resulted in robust OTC protein expression, improvements in ureagenesis and plasma ammonia, and increased survival,” said
About Ornithine Transcarbamylase (OTC) deficiency
Ornithine Transcarbamylase (OTC) deficiency is a serious urea cycle disorder with a prevalence of approximately 10,000 worldwide. 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. There are no FDA approved medicines for OTC deficiency.
ARCT-810 utilizes Arcturus' LUNAR® lipid-mediated delivery platform to deliver OTC messenger RNA to liver cells. Expression of OTC enzyme in the liver of patients with OTC deficiency has the potential to restore normal urea cycle activity, preventing neurological damage and the need for liver transplantation. The ARCT-810 program is supported by preclinical data in OTC deficiency murine models demonstrating that dosing of LUNAR-OTC results in robust ornithine transcarbamylase protein expression and activity resulting in improvements in ureagenesis and plasma ammonia and in increased survival.
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 ability or timing to initiate or complete preclinical and clinical development programs, including clinical studies planned for ARCT-810, the efficacy or safety of ARCT-810 or other Company programs, the likelihood of success of the Company’s drug candidates, including ARCT-810, or of the Company’s technology platforms, including STARR™ mRNA Technology or LUNAR® lipid-mediated delivery, the likelihood that any preclinical or clinical data will be predictive of future clinical results or sufficient for regulatory approval, the ability to enroll subjects in clinical trials, the likelihood that the success of ARCT-810, if successful, will be predictive of success of any future therapeutics of the Company and the impact of general business and economic conditions 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
IR and Media Contacts
Kendall Investor Relations