Ionis Pharmaceuticals has recently released the data from their clinical trial of IONIS-ENAC-2.5Rx, a treatment for cystic fibrosis. According to Newswire, the study showed very positive results, with healthy volunteers seeing a significant decrease in the expression of epithelial sodium channel.
About Cystic Fibrosis
Cystic fibrosis is a genetic disorder that is characterized by progressive damage to the respiratory and digestive systems. Those with cystic fibrosis do not have the slippery mucus that is normally found in the lungs. Instead they have thick and sticky mucus which builds up in their system. This buildup causes clogs in the airways, which then traps bacteria and causes breathing problems, infections, lung damage, and respiratory failure. It can also block digestive enzymes, which makes it difficult to absorb nutrients. Cystic fibrosis is a recessive disorder, meaning that the mutated gene must be passed down by both parents. The gene responsible for this condition affects the protein that regulates salt movement. The mutation in the gene varies in severity as well.
Symptoms of cystic fibrosis affect the respiratory and digestive systems. They include persistent coughing and wheezing, shortness of breath, difficulty exercising, frequent lung infections, stuffy noses, trouble with gaining weight, constipation, male infertility, salty-tasting skin, and exercise intolerance.
About IONIS-ENAC-2.5Rx and The Study
IONIS-ENAC-2.5Rx is an antisense medication that is intended to reduce the expression of epithelial sodium channel (ENaC) in the lungs. As this channel is hyperactive in those with cystic fibrosis, researchers hope that this treatment will reduce symptoms.
It was evaluated in a single dose escalating study in which the primary endpoint was to evaluate pharmacokinetis and safety. 32 participants were enrolled and were given either 3, 10, 37.5, or 100 mg of IONIS-ENAC-2.5Rx through a Pari eFlow mesh nebulizer once a week. For the next 13 weeks, participants were followed by researchers. In order to evaluate the final result, they were given a fiberoptic bronchoscopy.
Researchers found that there was a mean decrease in ENaC expression of 55.6%. This result is very positive, and medical professionals and patients are excited to see how further development of this treatment goes.