LITESPARK-004: 36 month follow-up of belzutifan in Von Hippel-Lindau syndrome

Ramaprasad Srinivasan, MD, PhD, National Cancer Institute, Bethesda, MD, discusses results with more than 3 years of follow-up from the Phase II LITESPARK-004 study (NCT03401788) of belzutifan, a HIF-2α inhibitor, in patients with Von Hippel-Lindau (VHL) syndrome. 61 patients with tumors what did not require surgical intervention were initially enrolled, with the primary endpoint being objective response rate (ORR) and the secondary endpoints being safety and duration of response (DOR). ORR was reached in patients receiving belzutifan, with adverse events being predominantly grade 1-2. Belzutifan additionally resulted in fewer patients needing surgery, the current standard of care. This interview took place at the European Society for Medical Oncology (ESMO) 2022 Congress in Paris, France.

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