A Pasadena-based biopharmaceutical company halted testing of a potential cystic fibrosis treatment drug after rats involved in the clinical study showed signs of inflamed lungs.
Investors Business Daily said Arrowhead stocks tumbled 26.5 percent to 62.49 on the stock market Friday after news of the drug trial pause was announced.
Arrowhead Pharmaceuticals has scored a string of success in recent years and won several multimillion-dollar deals with major drugmakers, including last month’s agreement with an Ireland-based firm that could be worth up to $660 million for the development of a new therapy meant to treat uncontrolled gout.
The company said Friday they have informed regulatory agencies, institutional review boards, and investigators that they were pausing AROENaC1001, a Phase 1/2 clinical study of ARO-ENaC, the investigational RNA interference therapeutic being developed to treat cystic fibrosis, effective immediately. The treatment aims to reduce the production of a protein that causes airway dehydration and aids in the transport of mucus, the company said.
The decision to pause was made after receiving a preliminary update from an ongoing chronic toxicology study about the effect on lab rats. The company has instructed investigators to pause new screening, enrollment, and any further dosing of ARO-ENaC until additional data from the chronic rat toxicology study and an additional chronic primate toxicology study are available.
“The safety of patients that participate in clinical trials of our investigational medicines is paramount to us at Arrowhead,” said Dr. Javier San Martin, chief medical officer at Arrowhead. “After we receive the full data from these studies, we will assess whether there is an acceptable path forward for further clinical investigation. While we determine our next steps with ARO-ENaC, we look forward to continued progress with the other 8 clinical drug candidates in our pipeline as well as our rapidly expanding preclinical initiatives, including additional lung-targeted programs.”
Dr. Christopher Anzalone, Arrowhead president and CEO, said this is difficult news for cystic fibrosis patients who are in need of new therapeutic options.
“We place above all else our obligation to ensure the safety of those enrolled in our clinical trials,” Anzalone said. “Even though the preliminary information we received from the chronic toxicology study in rats may not necessarily bear directly upon the safety of continuing the current Phase 1 study, we believe that we need to better understand the findings and how they may translate to humans before we treat additional patients. This may delay our pulmonary program a bit, but it’s just part of drug development.”
Anzalone said the company remains committed to the pulmonary platform and they’re moving ahead aggressively with their other pulmonary programs, of which two have been nominated as clinical candidates.
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