Ionis Pharmaceuticals has announced data from a Phase 1 SAD/MAD study of its IONIS-ENAC-2.5Rx nebulized antisense therapy in cystic fibrosis patients demonstrating a mean 55.6% reduction of epithelial sodium channel (ENaC) messenger RNA in cystic fibrosis patients who received a 75 mg dose in the MAD portion of the study. According to the company, preclinical studies in mouse models of CF demonstrated that reductions of ENaC mRNA of at least 40% resulted in significant improvement.
The SAD portion of the study enrolled 32 subjects who received either 3, 10, 37.5, or 100 mg of IONIS-ENAC-2.5Rx via a PARI eFlow nebulizer. In the MAD portion of the study, 24 subjects got once weekly doses of 10, 37.5, or 75 mg plus an extra dose administered in the first week. Another 8 subjects received a 37.5 mg dose three times per week for a total of 10 doses.
Ionis CEO Brett P. Monia said, “We are very encouraged by these data, which demonstrate attractive tolerability and safety for IONIS-ENAC-2.5Rx with substantial target reduction and the convenience of once weekly administration. The data also confirm our expectations for aerosol delivery of antisense medicines for lung diseases based on a wealth of preclinical data. These results point to the exciting potential for aerosol delivery of other Ionis medicines that we plan to advance to the clinic, including treatments for chronic obstructive pulmonary disease, or COPD, and severe asthma.”
Read the Ionis press release.
