VEGF is a key target in renal cell carcinoma (RCC) and several inhibitors of this protein (including sorafenib) have been approved as therapeutic options for this disease. Following in the steps of its relatives, axitinib, a second-generation inhibitor of VEGF, had already shown efficacy in phase II clinical trials in patients with sorafenib-refractory RCC, suggesting that axitinib could be an effective second-line treatment of patients with this malignancy. To further confirm these observations, Brian I. Rini and collaborators have now reported on a randomized phase III trial (AXIS) that compared the efficacy of axitinib and sorafenib as second-line therapy.

The trial included 723 patients with advanced RCC, 361 of whom received axitinib (at a starting dose of 5 mg BID increased to 7 mg and 10 mg in the absence of adverse effects) and 362 of whom received sorafenib (starting dose of 400 mg) for a median duration of treatment of around 5 months in both groups. The median progression-free survival (PFS) was 6.7 months in the axitinib arm and 4.7 months in the sorafenib arm and both regimens showed similar toxicity. As the efficacy of axitinib was compared with that of sorafenib instead of placebo, this difference in PFS proves axitinib's clinical efficacy as second-line therapy. When asked about future plans, Rini is hopeful, “a front-line trial with axitinib has completed accrual and results are pending. This trial will hopefully lead to FDA approval”.