Arcturus Therapeutics Holdings Inc. announced that Arcturus and the Cystic Fibrosis Foundation (CF Foundation) have extended their ongoing agreement. The CF Foundation agreed to increase its financial commitment to approximately $25 million to advance ARCT-032, a novel messenger RNA (mRNA) therapeutic candidate formulated with Arcturus' LUNAR delivery technology. This agreement extends a prior research program, initiated in 2017, which has resulted in the advancement of ARCT-032 into ongoing clinical studies.

ARCT-032 is made up of mRNA that codes for CFTR, encapsulated by lipid nanoparticles utilizing LUNAR delivery technology that has been optimized for inhaled lung delivery. ARCT-032 is agnostic to the underlying mutations associated with the disease and has the potential to provide clinical benefits across a wide range of those living with CF, including those not served by currently approved CFTR modulators. The program has completed dosing in a Phase 1 study in healthy volunteers.

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.

Cystic fibrosis is a life-shortening disease with a worldwide distribution. Mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene result in a reduction or absence of CFTR protein 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 some people with CF because of their underlying mutations.