Multinational phase 2 trial of belzutifan for advanced pheochromocytoma or paraganglioma

The multinational phase 2 LITESPARK-015 trial evaluated the HIF-2α inhibitor belzutifan in patients with advanced pheochromocytoma or paraganglioma. At a median follow-up of 30.2 months (range 23.3-37.6) the percentage of patients with confirmed objective response was 26% (95% CI 17-38) and the percentage with disease control was 85% (74-92). The median duration of response was 20.4 months (95% CI 8.3-NR) and the median duration of progression-free survival was 22.3 months (13.8-NR). Overall survival rate was 76% at 24 months. Treatment-related adverse events occurred in 99% of patients; anemia of grade 3 was noted in 22%, with treatment-related serious adverse events in 11%. The authors conclude that belzutifan showed antitumor activity with durable responses in patients with advanced pheochromocytoma or paraganglioma.