Arcturus Therapeutics Holdings Inc. (Nasdaq: ARCT), a global leader in messenger RNA medicines, focused on addressing unmet medical needs in infectious diseases and rare liver and respiratory conditions, unveiled Phase 1 findings in healthy volunteers and interim Phase 1b data in cystic fibrosis (CF) patients for ARCT-032, an investigational mRNA therapeutic for inhalation. The presentation took place at the 47th European Cystic Fibrosis Conference in Glasgow, Scotland.
The administration of ARCT-032 demonstrated safety and tolerability in both healthy volunteers and the initial four CF patients, with no reports of serious adverse events. Notably, the Phase 1b trial displayed enhancements in Forced Expiratory Volume in 1 second (FEV1) among the four CF adults following two inhaled doses. On Day 8, the absolute change in percent predicted FEV1 averaged +4.0%, with a relative change averaging +5.8%, indicating encouraging improvements consistent with earlier findings in CF ferret models.
Of the participants in Phase 1b, one had 2 Class I mutations, while the remaining three had F508del mutations and were undergoing treatment with Trikafta®.
Bronchospasm or febrile reactions were not observed in CF participants. However, dose-related mild-to-moderate febrile reactions were noted in some healthy volunteers. Transient declines in FEV1 among healthy volunteers were addressed with albuterol pretreatment, a common bronchodilator.
Dr. Juergen Froehlich, Chief Medical Officer of Arcturus Therapeutics, expressed satisfaction with the positive Phase 1 results and Phase 1b interim data, highlighting the safety profile and encouraging lung function improvements observed in all currently dosed CF participants. The completion of the Phase 1b trial is anticipated shortly, paving the way for further evaluation of ARCT-032’s potential to enhance lung function in CF patients through larger, multiple-dose clinical studies.
Cystic fibrosis is a life-limiting condition characterized by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, leading to inadequate chloride transport in the airways. This results in thickened mucus, airway obstruction, and susceptibility to infections and inflammation. While current CFTR modulator therapies aim to alleviate symptoms by enhancing CFTR channel function, their efficacy varies among patients due to underlying genetic mutations.
