Arcturus Therapeutics Announces Clinical Trial Application for ARCT-032 Received Approval to Proceed into First-in-Human Studies to Treat Cystic Fibrosis
“Arcturus continues to progress ARCT-032, a potential mRNA medicine for people with cystic fibrosis regardless of their underlying mutation type, through the approval of a CTA to proceed into First-in-Human studies,” said
About Cystic Fibrosis
Cystic fibrosis is a life-shortening disease with a worldwide distribution. Mutations in the Cystic Fibrosis Transmembrane (CFTR) gene result in a reduction or absence of CFTR quantity and/or function in the airways, causing insufficient chloride transport to maintain airway surface homeostasis. CF mucus is more difficult to clear, thus clogging the airways and leading to infection, inflammation, respiratory failure, or other life-threatening complications. Currently approved CFTR modulator therapies are designed to increase function of the CFTR channel to help reduce symptoms yet are ineffective in many people with CF as a result of their underlying mutations.
ARCT-032 will utilize Arcturus' LUNAR® lipid-mediated aerosolized platform to deliver CFTR messenger RNA to the lungs. Expression of a functional copy of the CFTR mRNA in the lungs of people with CF has the potential to restore CFTR activity and mitigate the downstream effects that cause progressive lung disease. The ARCT-032 program is supported by preclinical data in rodents, ferrets and primates, as well as demonstrating restoration of expression and function in human bronchial epithelial cells.
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, are forward-looking statements, including those regarding strategy, future operations, the expectations for or likelihood of success of any collaborations, the likelihood of success (including safety and efficacy) of ARCT-032, the planned initiation, design or completion of clinical trials (including the planned ARCT-032 Phase 1 study), the likelihood that preclinical data will be predictive of future clinical results, the likelihood that ARCT-032 will provide broad, or any, clinical benefit (including the ability of ARCT-032 to restore healthy CFTR protein), the ability to enroll, and timing for enrollment of, subjects in clinical trials, the timing and nature of any study results, the likelihood that a patent will issue from any patent application, and the impact of general business and economic conditions. 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. These statements are only current predictions or expectations, and are subject to known and unknown risks, uncertainties, and other factors that may cause our or our industry’s actual results, levels of activity, performance or achievements to be materially different from those anticipated by the forward-looking statements, including those discussed under the heading "Risk Factors" in Arcturus’ most recent Annual Report on Form 10-K, and in subsequent filings with, or submissions to, the
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