Arcturus Announces Regulatory Approval to Proceed with Phase 2 Study of ARCT-810 mRNA Therapeutic Candidate for Ornithine Transcarbamylase (OTC) Deficiency
The ARCT-810 Phase 2 study is a randomized, double-blind, placebo-controlled, nested single and multiple ascending dose design for adolescents and adults with OTC deficiency. The study will enroll up to 24 subjects across two dose levels. Safety and tolerability of ARCT-810 are the primary study objectives, and pharmacokinetic and pharmacodynamic measures of urea cycle function will be performed over a 12- week treatment period to access exposure and efficacy of this novel treatment.
“We are pleased to initiate this multiple dose Phase 2 clinical trial of ARCT-810 in OTC deficiency patients. Advancement to a Phase 2 study is supported by the safety results from our ARCT-810 Phase 1 trial in
About Ornithine Transcarbamylase (OTC) deficiency
Ornithine Transcarbamylase (OTC) deficiency is a serious urea cycle disorder with a prevalence of approximately 10,000 people worldwide. A lack of OTC, a critical urea cycle enzyme in liver cells, results in high blood ammonia levels that can cause diminished cognitive ability, seizures, coma, and death. There are no approved medicines addressing the root cause of OTC deficiency.
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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 ability to enroll subjects in clinical trials, the timing of any results (including data readouts), 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, 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
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