Ionis’ inhaled antisense medicine demonstrates potential as a novel treatment for cystic fibrosis | News

CARLSBAD, Calif., Oct. 13, 2020 /PRNewswire/ —┬áIonis Pharmaceuticals, Inc. (NASDAQ: IONS), the leader in antisense therapeutics, announced today that data from a clinical trial of IONIS-ENAC-2.5Rx demonstrated a significant decrease in the expression of epithelial sodium channel (ENaC) in subjects with cystic fibrosis (CF). The study showed a mean 55.6 percent decrease (p<0.05) in ENaC mRNA expression at the 75 mg dose in the multidose segment of the trial. The study represents the first time an antisense medicine delivered directly to the lung via a nebulizer has shown a significant reduction in ENaC messenger RNA levels. In preclinical studies, ENaC mRNA reductions of 40 percent or more resulted in significant improvement in mouse models of CF lung disease.

IONIS-ENAC-2.5Rx is an investigational antisense medicine designed to reduce the expression of ENaC in the lung. ENaC is believed to be hyperactive in cystic fibrosis, which is caused

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Ionis’ inhaled antisense medicine demonstrates potential as a novel treatment for cystic fibrosis

CARLSBAD, Calif., Oct. 13, 2020 /PRNewswire/ —┬áIonis Pharmaceuticals, Inc. (NASDAQ: IONS), the leader in antisense therapeutics, announced today that data from a clinical trial of IONIS-ENAC-2.5Rx demonstrated a significant decrease in the expression of epithelial sodium channel (ENaC) in subjects with cystic fibrosis (CF). The study showed a mean 55.6 percent decrease (p

IONIS-ENAC-2.5Rx is an investigational antisense medicine designed to reduce the expression of ENaC in the lung. ENaC is believed to be hyperactive in cystic fibrosis, which is caused by mutations in the cystic fibrosis transmembrane regulator gene. Data from the Phase 1 study will be presented via e-poster at the 2020 North American Cystic Fibrosis Conference, which will hold virtual sessions and discussions Oct. 21-23.

Cystic fibrosis is a life-threatening disease affecting approximately 30,000 people in the U.S. and about 70,000 worldwide. Although CF is a multisystem disease, the main cause of

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