According to Arcturus Therapeutics, the FDA has cleared an investigational new drug application allowing the company to proceed with a Phase 2 MAD trial of its ARCT-032 inhaled mRNA therapy in people with cystic fibrosis. The nebulized therapy, which is designed to deliver CFTR mRNA, has both orphan drug and rare pediatric disease designations from the FDA and orphan medicinal product designation from the EMA.
The company announced in late 2020 that it would develop ARCT-032, which is based on the company’s LUNAR (lipid-enabled and unlocked nucleomonomer agent modified RNA) technology, for the treatment of CF. The Cystic Fibrosis Foundation has provided funding to support development of the therapy.
Arcturus Chief Medical Officer Juergen Froehlich commented, “The Phase 2 Study May Proceed notification allows us to investigate ARCT-032 as a potential treatment for CF patients and provides the opportunity to further validate our LUNAR technology to deliver mRNA via inhalation. The study is designed to evaluate the safety and effectiveness of ARCT-032 administered for several weeks at multiple dose levels in people with CF who do not qualify for, or benefit from, CFTR modulator therapy.”
Read the Arcturus Therapeutics press release.
