US-based, clinical stage, biotechnology company X4 Pharmaceuticals has dosed its first patient in a Phase l/ll trial of X4P-001, the company’s lead CXCR4 inhibitor, to treat patients with advanced clear cell renal cell carcinoma (ccRCC).

ccRCC is the most common form of kidney cancer and advanced ccRCC reportedly accounts for around 20% of the patient population.

The Phase l portion of the trial will test the safety and tolerability of increasing doses of X4P-001 in combination with Inlyta (axitinib), a kinase inhibitor approved for the treatment of advanced RCC after failure of one prior systemic therapy.

The trial is designed to establish a maximum tolerated dose (MTD), or a recommended dose if the MTD is not achieved, for the drug combination.

Preliminary results from the Phase l portion of the study are expected by next year, followed by the initiation of the Phase ll portion of the study.

Various cancer centres in the US will participate in the study, for which Pfizer will provide axitinib.

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Dana Farber / Harvard Cancer Centre Kidney Cancer Programme lead and study investigator Dr David McDermott said: "Many ccRCC patients do not achieve durable responses when treated with the currently approved targeted therapies or immunotherapies.

"Based on the pre-clinical evidence in models of kidney cancer and a safety profile generated in prior clinical studies, X4P-001 has the potential to provide a meaningful new treatment option for ccRCC patients in combination with axitinib."

" … We see this as the beginning of multiple opportunities that we will pursue with our portfolio of CXCR4 inhibitors."

X4 Pharmaceuticals noted that X4P-001 is an oral, small molecule inhibitor of CXCR4, or C-X-C receptor type 4, the receptor for the chemokine CXCL12, which is also known as stromal derived factor-1, or SDF-1.

X4P-001 was previously tested in more than 70 subjects in four prior clinical trials in healthy volunteers and HIV-infected patients and was shown to be safe and well tolerated.

X4 Pharmaceuticals president and CEO Paula Ragan said: "Since our recent Series A financing, our team at X4 has rapidly executed on our clinical plan to initiate the study of X4P-001 as a potential treatment for patients with advanced ccRCC, a cancer with serious unmet needs.

"Given the growing evidence that CXCR4 inhibition plays an important role modulating the tumour microenvironment, we see this as the beginning of multiple opportunities that we will pursue with our portfolio of CXCR4 inhibitors."

Image: ccRCC is the most common form of kidney cancer. Photo: courtesy of Ed Uthman.