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A new treatment called casdatifan showed promising responses in a phase 1 trial looking at the best dose to use for previously treatment patients with advanced kidney cancer. More than a third of patients responded to treatment, and the time to when the treatment stopped working and the cancer started growing again (progression-free survival) was a year, which is very promising in patients who are on their second, third, fourth or even fifth line of treatment.
Studies looking at the genes of people with kidney cancer have shown mutations in a gene called the von Hippel-Lindau (VHL) gene. This results in high levels of a protein called hypoxia-inducible factor, or HIF-2α in tumour cells. HIF-2α causes changes in the cancer cells resulting in the growth of the tumour. A new treatment, called a HIF-2α inhibitor, is a tablet that blocks the action of HIF-2α and tumour growth. Casdatifan is a type of HIF-2α inhibitor and is similar to belzutifan.
There were 121 patients in the study. Patients were split into four groups and treated with different doses of casdatifan ranging from 50 mg twice daily to 150 mg once daily. The 100 mg daily dose was found to be the best dose. Patients were followed for more than a year.
Further, 60% of patients on the 100 mg dose did not have disease progression in the first 12 months. the disease was controlled in 8 in 10 patients in the study.
Casdatifan had manageable side effects in all patients in the study. Serious treatment-related side effects were reported by about a third of patients. Severe or life-threatening side effects were anaemia and low blood oxygen levels. Less than 1 in 10 patients stopped treatment because of side effects.
Further studies are ongoing to assess casdatifan in combination with immunotherapy or TKIs for first- or second-line treatment of advanced kidney cancer, and casdatifan alone for patients with favourable-risk disease.


