A First-in-Human Phase 1 Study of a Tumor-Directed RNA-Interference Drug against HIF2α in Patients with Advanced Clear Cell Renal Cell Carcinoma

• Published in: Clinical cancer research Vol. 30; no. 11; pp. 2402 - 2411
• Main Authors: Brugarolas, James, Obara, Gregory, Beckermann, Kathryn E., Rini, Brian, Lam, Elaine T., Hamilton, James, Schluep, Thomas, Yi, Min, Wong, So, Mao, Zhongping Lily, Gamelin, Erick, Tannir, Nizar M.
• Format: Journal Article
• Language: English
• Published: United States American Association for Cancer Research 03.06.2024
• Subjects: Adult; Aged; Aged, 80 and over; Antineoplastic Agents - administration & dosage; Antineoplastic Agents - adverse effects; Basic Helix-Loop-Helix Transcription Factors - genetics; Biological Agents & Therapies; Carcinoma, Renal Cell - drug therapy; Carcinoma, Renal Cell - genetics; Carcinoma, Renal Cell - pathology; Clinical Trial Results; Clinical Trials: Targeted Therapy; Female; Genitourinary Cancers; Humans; Kidney Neoplasms - drug therapy; Kidney Neoplasms - genetics; Kidney Neoplasms - pathology; Male; Middle Aged; RNA Interference; RNA, Small Interfering - administration & dosage; RNA, Small Interfering - genetics

ARO-HIF2 is an siRNA drug designed to selectively target hypoxia-inducible factor-2α (HIF2α) interrupting downstream pro-oncogenic signaling in clear cell renal cell carcinoma (ccRCC). The aims of this Phase 1 study (AROHIF21001) were to evaluate safety, tolerability, pharmacokinetics, and establish...