Belzutifan, an oral hypoxia-inducible factor 2α inhibitor, for Von Hippel-Lindau disease–associated pancreatic neuroendocrine tumors

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Introduction: Constitutive activation of hypoxia-inducible factor 2α (HIF-2α) drives tumor growth, which include pancreatic lesions (serous cystadenomas and pancreatic neuroendocrine tumors [pNETs]), in Von Hippel-Lindau (VHL) disease. Belzutifan, an oral HIF-2α inhibitor, is approved for patients with VHL disease based on the results of this ongoing open-label phase 2 study (NCT03401788).

Aim(s): To evaluate patients with VHL-associated pNETs treated with belzutifan.

Materials and methods: Treatment-naïve adults with germline VHL alterations, measurable and localized/nonmetastatic renal cell carcinoma (RCC), and ECOG PS of 0 or 1 received belzutifan 120 mg once daily. End points included ORR of pNETs (primary) and all pancreatic lesions (secondary) per RECIST v1.1 by an independent review committee.

Conference:

Presenting Author:

Authors: Else T, Jonasch E, Iliopoulos O, Rathmell W, Narayan V,

Keywords: VHL, pancreatic neuroendocrine tumor, pNET, HIF-2α, belzutifan,