Abstract Library

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#3848 LITESPARK-015 cohort B1 – A phase 2 open-label study of Belzutifan (a HIF-2α inhibitor) monotherapy in patients with von Hippel-Lindau (VHL) disease–associated tumors

Introduction: Patients (pts) with von Hippel-Lindau (VHL) disease need novel targeted therapies. Hypoxia-inducible factor 2α (HIF-2α) is a key oncogenic driver in VHL disease. Belzutifan (MK-6482), a HIF-2α inhibitor, has shown antitumor activity in pts with advanced renal cell carcinoma (RCC) and pancreatic neuroendocrine tumors (pNETs); more data are needed in VHL disease–associated localized tumors.

Conference:

Presenting Author:

Authors: Walter T, Gong K, Nakamura E, Iliopoulos O, Jimenez C,

Keywords: von Hippel-Lindau disease, belzutifan, MK-6482,

#3585 Belzutifan, an oral hypoxia-inducible factor 2α inhibitor, for Von Hippel-Lindau disease–associated pancreatic neuroendocrine tumors

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).

Conference:

Presenting Author:

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

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

#3405 A phase 2 open-label study of belzutifan (a HIF-2α inhibitor) monotherapy in patients with advanced/metastatic pheochromocytoma/paraganglioma or pancreatic neuroendocrine tumors

Introduction: Patients with advanced pheochromocytoma/paraganglioma (PPGL) or pancreatic neuroendocrine tumor (panNET) are in need of novel targeted therapies. Hypoxia-inducible factor 2α (HIF-2α) is one of the key oncogenic drivers in neuroendocrine tumors like PPGL and somatostatinomas. Hypoxia signaling pathway alterations or other mechanisms that stabilize HIFs are common in some PPGLs and panNETs. Belzutifan (MK-6482), a HIF-2α inhibitor, has shown antitumor activity in advanced renal cell carcinoma and von Hippel-Lindau (VHL) disease–associated tumors, including panNET.

Conference:

Presenting Author:

Authors: Jimenez C, Hadoux J, Del Rivero J, Das S, Iliopoulos O,

Keywords: pheochromocytoma, paraganglioma, pancreatic neuroendocrine tumor, belzutifan, MK-6482, metastatic,