Arcturus Therapeutics

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Arcturus Therapeutics Achieves Program Milestones and Refines Agreement with Cystic Fibrosis Foundation Therapeutics

July 23, 2018 at 7:00 AM EDT

SAN DIEGO, July 23, 2018 (GLOBE NEWSWIRE) -- Arcturus Therapeutics Ltd. (NASDAQ:ARCT), a leading RNA medicines company, today announced the achievement of key milestones as part of its agreement with Cystic Fibrosis Foundation (CF Foundation) Therapeutics, Inc. The completion of these success-driven milestones has triggered an undisclosed payment to Arcturus from the CF Foundation to advance LUNAR-CF, an mRNA therapeutic program to treat cystic fibrosis.

Arcturus recently presented preclinical data at the Cystic Fibrosis Foundation Research Conference during June 18-21 in Jackson Hole, Wyoming. The presentation can be found here: Presentation

Highlights of the data presented include:

  • Identification of novel human mRNAs that yielded higher functionally-active CFTR protein levels compared to natural sequence.
  • Achieved proof-of-concept for efficient nebulized delivery of mRNA to bronchial epithelial cells.

Given the success of the ongoing program, Arcturus and the CF Foundation have agreed to expand the scope of activities included in the research agreement and have amended the milestone criteria to further define the plan to advance the program into clinical development. With support from the CF Foundation, Arcturus will accelerate its efforts to develop an mRNA-based therapeutic for the treatment of cystic fibrosis using its wholly-owned LUNAR® lipid-mediated delivery system and proprietary mRNA optimization, design, and production process.

“We are pleased to report that LUNAR-CF has advanced through key milestones with the support of the CF Foundation,” said Joseph Payne, President & CEO of Arcturus Therapeutics. “This enhanced agreement underscores the CF Foundation’s continued commitment to our cystic fibrosis development program. We are confident that we have mapped out the best path forward to efficiently drive our promising lead candidate into the clinic. Our goal is to develop a transformative therapeutic that has the potential to treat a broad population of cystic fibrosis patients, regardless of mutation type, as we drive long-term growth and value for our shareholders.”

About the Cystic Fibrosis Foundation
The CF Foundation’s mission is to cure cystic fibrosis and to provide all people with the disease the opportunity to lead full, productive lives by funding research and drug development, promoting individualized treatment, and ensuring access. Through its venture philanthropy model, the CF Foundation provides early stage funding to biotechnology and pharmaceutical companies to develop breakthrough drugs for adults and children with cystic fibrosis.  Nearly every CF-specific drug available today was made possible with the foundation’s financial support. For more information, refer to

About Arcturus Therapeutics Ltd.

Founded in 2013 and based in San Diego, Arcturus Therapeutics Ltd. (NASDAQ:ARCT) is an RNA medicines company with enabling technologies – UNA Oligomer chemistry and LUNAR® lipid-mediated delivery. Arcturus’ diverse pipeline of RNA therapeutics includes programs pursuing rare diseases, Hepatitis B, non-alcoholic steatohepatitis (NASH), cystic fibrosis, and vaccines. Arcturus’ versatile RNA therapeutics platforms can be applied toward multiple types of RNA medicines including small interfering RNA, messenger RNA, replicon RNA, antisense RNA, microRNA and gene editing therapeutics. Arcturus owns LUNAR lipid-mediated delivery and Unlocked Nucleomonomer Agent (UNA) technology including UNA Oligomers, which are covered by its extensive patent portfolio (140 patents and patent applications, issued in the U.S., Europe, Japan, China and other countries). Arcturus’ proprietary UNA technology can be used to target individual genes in the human genome, as well as viral genes, and other species for therapeutic purposes. Arcturus’ commitment to the development of novel RNA therapeutics has led to partnerships with Janssen Pharmaceuticals, Inc., part of the Janssen Pharmaceutical Companies of Johnson & Johnson, Ultragenyx Pharmaceutical, Inc., Takeda Pharmaceutical Company Limited, Synthetic Genomics Inc., CureVac AG and Cystic Fibrosis Foundation Therapeutics Inc. For more information, visit, the content of which is not incorporated herein by reference. 

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 regarding strategy, future operations, collaborations, future financial position, prospects, plans and objectives of management are forward-looking statements. Examples of such statements include, but are not limited to, statements relating to the collaboration with the Cystic Fibrosis Foundation having the potential to identify, develop or commercialize any potential product; statements relating to Arcturus’ future operations and its ability to successfully initiate and complete clinical trials; the nature; strategy and focus of Arcturus; the achievement of any future milestones; the development and commercial potential and potential benefits of any product candidates of Arcturus; and the continuation or modification of collaborative arrangements. Arcturus may not actually achieve the plans, carry out the intentions or meet the expectations or projections disclosed in any forward-looking statements and you should not place undue reliance on such forward-looking statements. Actual results and performance could differ materially from those projected in any forward-looking statements as a result of many factors, including without limitation, an inability to develop and market product candidates.  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 20-F for the fiscal year ended December 31, 2017, filed with the SEC on May 14, 2018 and in subsequent filings with, or submissions to, the SEC. Except as otherwise required by law, Arcturus disclaims any intention or obligation to update or revise any forward-looking statements, which speak only as of the date they were made, whether as a result of new information, future events or circumstances or otherwise.


Arcturus Therapeutics, Ltd.
(858) 900-2666
Attn: Neda Safarzadeh

Arcturus Investor Contacts
Michael Wood
LifeSci Advisors LLC
(646) 597-6979

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Source: Arcturus Therapeutics, Inc.