The phase 2 study will evaluate the safety and efficacy of ARCT-032, an inhaled mRNA therapy for cystic fibrosis patients, which previously received Orphan Drug Designation.
RT’s Three Key Takeaways:
- FDA Clearance for Phase 2 Study: The FDA has issued a “study may proceed” notification for Arcturus Therapeutics’ investigational drug ARCT-032, allowing the company to begin a phase 2 trial to evaluate its safety and efficacy in cystic fibrosis patients.
- Focus on Inhaled mRNA Therapy: ARCT-032 is an inhaled mRNA therapeutic designed to deliver a functional cystic fibrosis transmembrane conductance regulator (CFTR) gene to the lungs, aiming to restore CFTR activity in cystic fibrosis patients.
- Orphan Drug and Rare Pediatric Disease Designations: ARCT-032 has been granted Orphan Drug Designation and Rare Pediatric Disease Designation by the FDA, along with Orphan Medicinal Product Designation from the European Medicines Agency.
Arcturus Therapeutics Holdings Inc, a global messenger RNA medicines company focused on the development of infectious disease vaccines and addressing unmet medical needs within liver and respiratory rare diseases, announced that the US Food and Drug Administration (FDA) has issued a “study may proceed” notification for the company’s investigational new drug (IND) application, ARCT-032, to treat cystic fibrosis.
FDA clearance of the ARCT-032 IND application enables the company to initiate a phase 2 multiple ascending dose study to evaluate the safety, tolerability, and efficacy of ARCT-032 in people with cystic fibrosis.
Investigational mRNA Therapeutic
ARCT-032 is an inhaled investigational mRNA therapeutic designed to express normal functional cystic fibrosis transmembrane conductance regulator (CFTR) in the lungs of individuals with cystic fibrosis. ARCT-032 has received Orphan Medicinal Product Designation from the European Medicines Agency and Orphan Drug Designation, along with Rare Pediatric Disease Designation, from the FDA to treat cystic fibrosis.
ARCT-032 utilizes Arcturus’ Lunar lipid-mediated aerosolized platform to deliver CFTR messenger RNA to the lungs. Lung disease is the leading cause of morbidity and mortality in people with cystic fibrosis. Expression of a functional copy of the CFTR mRNA in the lungs of people with cystic fibrosis 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 CFTR expression and function in human bronchial epithelial cells.
“The phase 2 ‘study may proceed’ notification allows us to investigate ARCT-032 as a potential treatment for [cystic fibrosis] patients and provides the opportunity to further validate our LUNAR technology to deliver mRNA via inhalation,” says Juergen Froehlich, MD, chief medical officer of Arcturus Therapeutics, in a release. “The study is designed to evaluate the safety and effectiveness of ARCT-032 administered for several weeks at multiple dose levels in people with [cystic fibrosis] who do not qualify for, or benefit from, CFTR modulator therapy.”
