Combination Therapy Shows Encouraging Results in Patients with Clear Cell Renal Cell Carcinoma

Mathew Shanley

Updated results from the Phase 1 part of an ongoing Phase 1/2 study of X4P-001-IO in combination with Inlyta (axitinib) to treat patients with clear cell renal cell carcinoma (ccRCC) were announced this morning by X4 Pharmaceuticals.

ccRCC is a form of kidney cancer that initiates in the lining of the small tubes in the kidney. It is the most common variation of adult kidney cancer, and its advanced stage accounts for 20% of all diagnoses.

The data presented at the 2017 AACR-NCI-EORTC Molecular Targets and Cancer Therapeutics Conference on October 29 in Philadelphia follows data previously presented in September at the European Society of Medical Oncology (ESMO) 2017 Congress in Madrid that exhibited encouraging disease control rates and durable clinical responses in patients with ccRCC receiving X4)-011-IO plus axitinib.

The new results concluded that the combination showed an objective response rate (ORR) of 29% (4/14) including 1 patient achieving a confirmed complete response (CR). Additionally, there was an encouraging disease control rate of 93% (13/14).

“The combination of CXCR4 inhibition and VEGFR inhibition shows promising clinical results in this very difficult to treat population of patients with ccRCC. These results suggest that X4P-001-IO may address some of the limitations and augment the clinical utility of axitinib, which is a clinically meaningful drug in the treatment of patients with advanced metastatic ccRCC,” said Michael Atkins, MD, Deputy Director, Georgetown Lombardi Comprehensive Cancer Center, William M. Scholl Professor of Oncology at Georgetown University School of Medicine, and lead investigator of the study in a press release. “These results, while early, are very promising with a strong disease control rate and a manageable safety profile.”

The study’s intention is to assess different doses of X4P-001 both given alone and in combination with axitinib to determine its safety and tolerability. The dose escalation segment of the study enrolled 16 patients with advanced ccrRCC as of the October 2, 2017 data cutoff date, and the presentation included information and highlights including the confirmed CR in 1 patient.

The most common treatment-related adverse events (AEs) among patients enrolled in the study were hypertension, diarrhea, fatigue, nausea, decreased appetite, headache, and dry eye. Overall, the combination was considered to be safe and well tolerated, and no grade 4 or 5 AEs were reported in any of the patients.

Based on study results, a dose of 400 mg X4P-001-IO once daily with 5 mg Inlyta twice daily was selected for the upcoming Phase 2 portion of the study, which will evaluate the clinical efficacy as measured by ORR, duration of response (DOR), and progression free survival (PFS), in addition to exploring the correlation of biomarkers with efficacy.

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Image courtesy of Nephron; WikiMedia Commons
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