Abstract Library

#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,

#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,

#3250 High TMB and MSI-high as independent predictors of survival in NENs

Introduction: To date, there are no validated molecular biomarkers for personalized therapy in NENs. Tumor genome sequencing may help unravel NEN molecular landscape and identify novel targets for an individualized approach.

Conference: 18th Annual ENETS Concerence (2021)

Presenting Author:

Authors: La Salvia A, Barroso G, Espinosa Olarte P, Anton Pascual B, Modrego Sanchez A,

Keywords: molecular profiling, neuroendocrine neoplasm, TMB, microsatellite instability (MSI)/mismatch repair deficiency (MMRd) status, prognosis,

#2974 MEK/RAF and PARP as Novel Targets in Neuroendocrine Neoplasms – First Results from a Molecular in vitro Tumor Board

Introduction: Neuroendocrine neoplasms (NEN) are a heterogeneous group comprising well differentiated neuroendocrine tumors (NET) and poorly differentiated neuroendocrine carcinomas (NEC). Although, major progress has been made in the development of targeted therapies in various cancers, establishment of a promising personalized therapy in NEN is still pending. So far, a lack of suitable preclinical models represent a major challenge concerning the validation of a mutational based targeted therapy (MBTT)

Conference: 17th Annual ENETSConcerence (2020)

Presenting Author:

Authors: Viol F, Sipos B, Amin T, Fahl M, Grabowski P,

Keywords: Neuroendocrine neoplasms, Personalized therapy, Preclinical in vitro models, Mutational based targeted therapy,

#2964 Personalized Therapy in a Case of Esophageal NEC Based on Tumor Genome Sequencing

Introduction: High-grade esophageal neuroendocrine carcinomas (NEC) constitute a rare subgroup of neuroendocrine neoplasms, with a particularly aggressive behaviour and unfavourable prognosis. To date, there are no validated biomarkers for personalized therapy and cytotoxic chemotherapy remains the standard of care. However, tumor genome sequencing may help improve NEC molecular landscape knowledge in order to identify novel targets for an individualized approach.

Conference: 17th Annual ENETSConcerence (2020)

Presenting Author: La Salvia A

Authors: La Salvia A, Espinosa Olarte P, Modrego Sanchez A, Riesco Martinez M, Garcia Carbonero R,

Keywords: Esophageal neuroendocrine carcinoma, tumor genome sequencing, immunotherapy, tumor burden, personalized therapy,