Dosing Begins in Trial of Cystic Fibrosis Treatment

Arrowhead Pharmaceuticals has began dosing patients in its AROENaC1001 trial, which evaluates ARO-ENaC as a treatment for cystic fibrosis. 54 patients will be treated with this therapy in an effort to understand its effects on both healthy individuals and cystic fibrosis patients.

About Cystic Fibrosis (CF)

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.

Diagnosis often comes at birth, as screening for cystic fibrosis is standard across the United States. In order to complete a screening, doctors must perform blood sampling, genetic testing, and sweat tests. After a diagnosis is obtained, doctors select the correct treatment. The goal is to control infections, remove mucus from the lungs, prevent intestinal blockage, and provide proper nutrition. Methods include antibiotics, anti-inflammatory medications, medicine to induce the coughing up of mucus, bronchodilators, pancreatic enzymes, exercise therapy, chest physical therapy, vest therapy, lung transplants, and surgery to remove bowel blockages, remove nasal polyps, and place a feeding tube.

About the Trial

The first patient was just dosed in Phase 1/2 of the AROENaC1001 trial. It will evaluate ARO-ENaC’s safety, tolerability, efficacy, and pharmacokinetics. The medication itself is an RNAi therapy that is meant to lower the activity of the epithelial sodium channel alpha subunit in the lungs’ airways. It is an inhaled small molecule.

This molecule will be tested in 24 healthy individuals and 30 patients with CF. While researchers will look at ARO-ENaC’s safety and efficacy, they will also study how it impacts patients’ lung clearance index and forced expiratory volume.

Medical professionals and patients hope that this trial is successful and able to move on to further phases, as ARO-ENaC has the potential to help patients who do not benefit from current treatments due to differences in genotypes.

Read more here.

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