CF Foundation gives $9M more to advance mRNA therapy in testing

Patient trial of ARCT-032, aiming to treat all with CF, may open this year

Margarida Maia, PhD avatar

by Margarida Maia, PhD |

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The Cystic Fibrosis (CF) Foundation is giving up to $9 million in additional support to advance clinical testing of ARCT-032, a messenger RNA therapy with the potential to benefit anyone with CF, regardless of their disease-causing mutation.

Arcturus Therapeutics, the inhalation therapy’s developer, is using the funding to support a Phase 1b clinical study in people with CF, planned to open later this year in New Zealand. It also intends to put some of the money into research needed to support a Phase 2 clinical study, planned for 2024.

ARCT-032 contains a piece of messenger RNA (mRNA) that serves as a blueprint for making CFTR, the protein that is missing or faulty in people with CF. The mRNA is packaged in tiny, fat-like particles that unload their cargo into lung cells after ARCT-032 is inhaled.

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“Delivering the therapy to the correct cells is a significant challenge, and we are excited by the progress Arcturus is making in this area,” JP Clancy, MD, senior vice president of clinical research at the CF Foundation, said in a press release from the group.

CF is caused by mutations in CFTR, a gene that provides instructions for making a protein of the same name. CFTR controls the movement of salts and water in and out of cells, and problems with the protein lead to thick and sticky mucus building in the lungs and other organs.

More than 2,000 disease-causing mutations are known. CFTR modulators work to improve the protein’s function, but they are mutation specific and not indicated for CF caused by some rare or nonsense mutations — those that introduce a stop signal early in the protein’s production, leading to unstable CFTR.

By delivering a working copy of mRNA directly into the airways, the “therapy offers a way to treat all people with CF, especially those who can’t tolerate or don’t respond to modulator treatment,” Clancy said. This includes people who carry two copies of rare or nonsense mutations.

ARCT-032 uses Arcturus’ LUNAR delivery vehicles, which consist of fat-like particles designed to help the mRNA more easily enter the airways and cells there. Lung cells then are expected to be able to make a fully functional CFTR protein.

A Phase 1a clinical study (NCT05712538) has finished dosing healthy adults, the company reported. This study’s main goal is to evaluate the safety and tolerability of single ascending doses of inhaled ARCT-032 versus a placebo for up to four weeks. The treatment’s pharmacokinetics — its movement into, through, and out of the body — will also be studied.

This latest funding brings CF Foundation support for ARCT-032 to about $25 million.

“We are thrilled to extend our productive relationship with the Cystic Fibrosis Foundation, and we are grateful for their meaningful financial support,” Pad Chivukula, PhD, chief scientific officer at Arcturus, said in a company press release.

The investment is part of the foundation’s Path to a Cure, a $500 million initiative whose goal is to accelerate the development of treatments that address the root cause of CF.