Shares of Arrowhead Pharmaceuticals (Nasdaq: ARWR) were down 26% at $62.84 mid-morning today, after the US biotech notified regulatory agencies, institutional review boards, and investigators that effective immediately it is voluntarily pausing AROENaC1001, a Phase I/II clinical study of ARO-ENaC.
This is the company’s investigational RNA interference (RNAi) therapeutic being developed as a treatment for patients with cystic fibrosis (CF), and the pause decision follows receipt of a preliminary update from an ongoing chronic toxicology study in rats that contained unexpected signals of local lung inflammation.
The company has instructed investigators to pause new screening, enrollment, and any further dosing of investigational ARO-ENaC pending additional data from the ongoing chronic rat toxicology study and an additional ongoing chronic primate toxicology study.
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