Scientists have identified a promising new drug for renal cell carcinoma (RCC); X4P-001 is an oral drug that blocks the cytokine receptor CXCR4. In an ongoing phase 1/2 trial, X4P-001 has been given to patients with advanced RCC in combination with the tyrosine kinase inhibitor axitinib.
Preliminary results from the phase 1 part of the study have shown that X4P-001 plus axitinib is clinically active in patients with advanced clear cell RCC. Of the 22 clinically evaluable patients so far, the objective response rate was 31.8% and the disease control rate was 86.4%.
The combination is also well tolerated with a manageable safety profile. The most common (≥ 10%) treatment-related adverse events of any grade were diarrhoea, fatigue, high blood pressure, decreased appetite, difficulty speaking, nausea, increased blood creatinine, dry eye, headache, and vomiting. The only adverse event related to treatment and which occurred in more than 5% of patients was high blood pressure (9.8%), which was the only serious adverse event to occur in more than one patient.
The phase 2 part of the study continues to enrol patients, and updated study results will be reported.