The Cystic Fibrosis Foundation (CFF) will give up to $15 million to Arcturus Therapeutics to advance the development of an experimental RNA therapy, called LUNAR-CF, to treat CF patients with all types of mutations in the CFTR gene.
The funding will establish a multi-year program aimed at boosting the development of LUNAR-CF and other RNA-based treatments for CF, develop methods to deliver RNA therapies to lung cells, and file an investigational new drug (IND) application for LUNAR-CF. If the U.S. Food and Drug Administration (FDA) approves the application, Arcturus will have a green light to test its therapy on humans.
“We are delighted with the expanded financial support from the CF Foundation, and we believe that we are now sufficiently funded to advance the LUNAR-CF program into filing an IND application,” Joseph Payne, president and CEO of Arcturus, said in a press release.
Messenger RNA (mRNA) therapies are a novel approach to treat genetic diseases. mRNAs are the molecules that give instructions about protein production to cells. By delivering a non-mutated mRNA to target cells, these mRNA therapies allow the production of functional proteins.
Arcturus focuses on developing and manufacturing mRNA therapies and delivery systems — the substances that protect the mRNA molecule until it reaches the target cell, avoiding its degradation — to treat diseases like CF, ornithine transcarbamylase deficiency, hepatitis B, and non-alcoholic fatty liver disease.
The LUNAR delivery system consists of lipid (fat)-based nanoparticles that fuse to the target cells delivering the RNA therapy. They can be delivered by several methods, such as being injected into the bloodstream or inhaled.
Previous animal studies performed in non-human primates and mice showed that treatment with the LUNAR system led to a significant increase in protein production, with no safety concerns.
LUNAR-CF is the first treatment developed by Arcturus. It is an inhaled therapy that delivers a functional mRNA copy of the cystic fibrosis transmembrane conductance receptor (CFTR) — the protein whose malfunction causes CF — to lung cells. The therapy is intended to treat all patients regardless of their specific mutations in the CFTR gene.
Preclinical data in animal models showed that the LUNAR technology effectively delivered the CFTR mRNA to epithelial lung cells, which later produced a functional CFTR protein.
“We are pleased with the progress we have made in our agreement with the CF Foundation, including preclinical proof of concept studies, demonstrating that LUNAR is able to deliver mRNA efficiently into lung epithelial cells in animals and is compatible with nebulization,” said Payne.
The company expects to submit the IND application to the FDA in the second half of 2020.