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ASCO 2025 | 5-year follow-up results from LITESPARK-004

Eric Jonasch, MD, The University of Texas MD Anderson Cancer Center, Houston, TX, discusses 5-year follow-up results from the LITESPARK-004 study (NCT03401788), which evaluated the HIF-2α inhibitor belzutifan, in von Hippel-Lindau (VHL) disease-associated neoplasms. After 5 years of follow-up, belzutifan continues to demonstrate durable antitumor activity and a manageable safety profile, consistent with prior reports. This interview took place during the 2025 American Society of Clinical Oncology (ASCO) Meeting in Chicago, IL.

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Transcript

Yeah, so the LITESPARK-004 study is a study looking at belzutifan, which is a HIF2-alpha inhibitor in individuals with von Hippel-Lindau disease, which is a rare hereditary disorder that affects the eyes, spine, brain, kidneys, adrenal glands. And what we’ve seen with five-year follow-up is that the responses that we initially saw in the kidneys have continued to persist and improve...

Yeah, so the LITESPARK-004 study is a study looking at belzutifan, which is a HIF2-alpha inhibitor in individuals with von Hippel-Lindau disease, which is a rare hereditary disorder that affects the eyes, spine, brain, kidneys, adrenal glands. And what we’ve seen with five-year follow-up is that the responses that we initially saw in the kidneys have continued to persist and improve. We’ve also seen that the responses in the hemangioblastomas have improved as well, and also in the pancreatic neuroendocrine tumors. So we’re seeing that in all the three organs that we really were focusing on. Maintenance of response, we’re seeing that the majority of patients are still on treatment. We’re not seeing any new safety signals. So overall, this clearly remains an excellent choice for individuals with von Hippel-Lindau disease.

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Disclosures

Consulting or Advisory Role – Aveo; Eisai; Exelixis; GlaxoSmithKline; Ipsen; Merck; NiKang Therapeutics; Novartis; Takeda; Telix Pharmaceuticals
Research Funding – Abbvie (Inst); Arrowhead Pharmaceuticals (Inst); Aveo (Inst); Corvus Pharmaceuticals (Inst); Merck (Inst); NiKang Therapeutics (Inst); ProfoundBio (Inst); Telix Pharmaceuticals (Inst)
Travel, Accommodations, Expenses – Pfizer