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Kidney cancers frequently have mutations in a gene called the von Hippel-Lindau (VHL) gene, resulting in high levels of a protein called hypoxia-inducible factor, or HIF2. This results in a number of changes in the cancer cells and their surrounding environment that cause them to grow. ARO-HIF2 uses RNA to block the action of HIF2.
The study enrolled 17 patients. One person had a partial response with approximately 65% tumour shrinkage and 5 had stable disease. Furthermore, 4 people remain on the drug with stable disease, with treatment durations between 12 and 24 weeks. ARO-HIF2 has been generally well tolerated with no cases of anaemia reported so far.
The study is still ongoing and more patients are being recruited to look at the safety and effectiveness of this new medicine.