Single-center phase 2 trial of cabozantinib for unresectable and progressive metastatic pheochromocytoma or paraganglioma

Metastatic pheochromocytomas and paragangliomas (MPPGs) are orphan diseases often associated with germline pathogenic variants of the SDHB gene. These tumors exhibit a phenotype that is characterized by abnormal angiogenesis. The phase 2 Natalie Trial, at MD Anderson Cancer Center (Houston, TX) evaluated the antiangiogenic multi-TKI cabozantinib in patients with MPPGs. Among 16 for response evaluable patients objective response (primary endpoint) was seen in 4 (ORR 25.0%; 95% CI 7.3-52.4). There were seven grade 3 adverse events in six patients, but no grade 4 or 5 events. The authors conclude that cabozantinib had promising activity in patients with MPPGs.