Arrowhead Pharmaceuticals has recently submitted an application to the New Zealand Medicines and Medical Devices Safety Authority for the clearance of a clinical trial. They want to test their cystic fibrosis treatment, ARO-ENaC, for safety, tolerability, and effects. If approved, they will begin with AROENaC1001, a phase 1/2a study.
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 affects one in every 2,500 to 3,500 Caucasian babies, and it is even rarer in other ethnicities.
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.
In those affected by cystic fibrosis, epithelial sodium channel alpha subunit (αENaC) levels are heightened and cause dehydration in the airways and lower levels of mucociliary transport. ARO-ENaC is intended to lower the levels of αENaC. It is an RNA interference (RNAi) therapy and uses Arrowhead’s TRiM platform.
If Arrowhead gains the clearance they applied for, they will begin their phase 1/2a AROENaC1001 trial. In this study they will evaluate the tolerability, safety, and pharmacokinetic effects of ARO-ENaC in healthy patients and those with cystic fibrosis. The primary endpoints are changes in lung clearance index and forced expiratory volume.
If this trial is approved, cystic fibrosis patients may have a new treatment option.
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