X4 Pharma initiates clinical study of X4P-001 in combination with Opdivo

Combination studied as treatment for patients with advanced clear cell renal cell carcinoma.

The primary objective of the newly-initiated study is to evaluate the safety and tolerability of X4P-001 in combination with Opdivo (nivolumab), an approved immunotherapy for the treatment of advanced RCC after failure of prior anti-angiogenic therapy that blocks a signal preventing T-cells from attacking cancer. The study will enroll patients who have not responded to nivolumab. In addition to safety and tolerability, the trial will evaluate early signs of biological activity using biomarkers, and clinical efficacy as measured by objective response rate and progression free survival over a 12-month time frame. Multiple US cancer centers with leading renal cell carcinoma researchers will participate in the study.

“Enabling the immune system to recognize and attack cancer cells is one of the most promising new approaches to improving outcomes for cancer patients,” said Dr David McDermott, Associate Professor of Harvard Medical School and Director of the Biologic Therapy Kidney Cancer Program at the Beth Israel Deaconness Medical Center and X4 study investigator. “While benefical for some patients, single-agent immunotherapy treatments have room for significant improvements in the durability and number of responses. The evaluation of combination immunotherapies is an important next step in cancer research. We are hopeful that X4P-001’s complementary mechanism of CXCR4 antagonism with PD-1 inhibition will demonstrate an innovative approach to modulating the immune system that yields improved patient outcomes.”

“Initation of this study is an important milestone in our strategy to develop X4P-001 in ccRCC in combination with dual classes of existing approved drugs,” said Paula Ragan, President and CEO of X4. “Approved therapies address certain processes in the tumour micro-environment, but we believe more can be done to address the complex biology of challenging cancers, like ccRCC. By modulating cell trafficking in the micro-environment where cancer hijacks normal immune function, X4P-001 may be synergistic with other cancer therapies to result in enhanced responses.”

In addition to this new Phase I/II study of X4P-001 in combination with Opdivo, X4 has another Phase I/II study ongoing in patients with advanced ccRCC to evaluate X4P-001 in combination with Inlyta (axitinib), a kinase inhibitor approved for the treatment of advanced RCC after failure of one prior systemic therapy, and a Phase Ib biomarker study in patients with advanced melanoma to evaluate X4P-001 in combination with Keytruda (pembrolizumab) .