Ionis' inhaled antisense drug shows promise as CF treatment

By The Science Advisory Board staff writers

October 13, 2020 -- Ionis Pharmaceuticals has released positive data from a clinical trial evaluating IONIS-ENAC-2.5Rx, an investigational antisense medicine designed to reduce expression of epithelial sodium channel in cystic fibrosis (CF) patients.

The results show that the medicine significantly decreased expression of epithelial sodium channel in subjects by an average of 55.6% at the 75-mg dose delivered via a Pari eFlow mesh nebulizer. In preclinical studies, epithelial sodium channel messenger RNA (mRNA) reductions of greater than 40% resulted in significant improvement in mouse models of CF lung disease.

The medication is administered directly to the lungs once weekly and has been established as tolerable and safe. The results are promising for aerosol delivery of various medicines in the company's portfolio.

The company plans to initiate a clinical trial to evaluate IONIS-ENAC-2.5Rx in patients with chronic obstructive pulmonary disease (COPD) associated with chronic bronchitis later this year.

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