On December 11, 2019 Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) reported that it has submitted an Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA) for a Phase 1b adaptive dose-finding clinical study of ARO-HIF2, the company’s investigational RNA interference (RNAi) therapeutic being developed as a treatment for patients with clear cell renal cell carcinoma (ccRCC) (Press release, Arrowhead Pharmaceuticals, DEC 11, 2019, View Source [SID1234552261]).

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Javier San Martin, M.D., chief medical officer at Arrowhead, said: "ARO-HIF2 is Arrowhead’s first TRiM enabled investigational RNAi therapeutic to target a cell type outside of the liver. Clear cell renal cell carcinoma, or ccRCC, is one of the most common forms of kidney cancer. Most patients with ccRCC have a mutation in the Von Hippel-Lindau gene, rendering them unable to degrade HIF-2α, which leads to accumulation during tumor hypoxia and promotes tumor growth. We believe this makes HIF-2α an attractive target for an RNAi-based intervention."

Pending regulatory review, the company intends to initiate AROHIF21001 (NCT04169711), a Phase 1b adaptive design dose-finding clinical study in patients with advanced ccRCC to evaluate the safety of ARO-HIF2 and to determine the recommended Phase 2 dose. Additional secondary objectives include the assessment of pharmacokinetics and efficacy, based on Response Evaluation Criteria in Solid Tumors (RECIST). An exploratory objective for AROHIF21001 will be gene target knockdown using tumor biopsy.