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Pasadena Pharmaceuticals Company Begins Study of New Cystic Fibrosis Treatment

Published on Tuesday, August 11, 2020 | 10:21 am
 

A Pasadena-based pharmaceuticals company has begun human trials of a new drug intended to treat cystic fibrosis, the company announced Tuesday.

Arrowhead Pharmaceuticals has begun a Phase 1/2 study of ARO-ENaC, an inhaled drug meant to combat the respiratory symptoms of cystic fibrosis through inhibiting the expression of certain genes through a process known as RNA interference, the company said in a written statement.

“The Phase 1/2 clinical study, AROENaC1001, is designed to assess safety, tolerability, and pharmacokinetics and potentially provide an early assessment of efficacy in patients with CF,” said Arrowhead Pharmaceuticals Chief Medical Officer Dr. Javier San Martin.

“We believe ENaC, or epithelial sodium channel, is a target with great potential for many CF patients that may not be eligible for existing therapies due to their specific genotypes, commonly called class I patients, and for those that have an inadequate response to therapy,” he said. “Our preclinical work on ARO-ENaC has been highly promising and we are eager to see how these results translate to humans.”

The study involved 24 healthy volunteers and 30 cystic fibrosis patients to evaluate the safety and effectiveness of the new treatment.

The medication is primarily intended to help with dehydration and mucous retention in the lungs experienced by people with cystic fibrosis, “a rare disease caused by genetic mutations that lead to progressive deterioration in lung function,” according to the statement.

More information about drugs under development at Arrowhead Pharmaceuticals is available on the company’s website at arrowheadpharma.com.

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