X4 Pharma Uses $37.5 Million to Push Cancer Therapies into Human Trials

X4 Pharma Uses $37.5M to Push Cancer Therapies into Human Trials
December 14, 2015
By Alex Keown, BioSpace.com Breaking News Staff

CAMBRIDGE, Mass. – Massachusetts based X4 Pharmaceuticals is beginning human trials for its oncology program using CXCR4 inhibitors, the Boston Business Journal reported this morning.

After spending years in stealth mode, the company, helmed by former Genzyme executives, is launching two clinical two clinical studies initiating in 2016 in refractory clear cell renal cell carcinoma and refractory epithelial ovarian cancer with its lead drug candidate, X4P-001.

The company’s pipeline is based on drug compounds that originate from a portfolio of oral CXCR4 inhibitors exclusively licensed from Sanofi , X4 said in a statement. Inhibition of CXCR4, a receptor over-expressed in many cancers, is designed to block non-cancerous immuno-suppressive and pro-angiogenic cells from populating the tumor, thereby disrupting the cancer microenvironment and restoring normal immune surveillance functions. The novel mechanism of CXCR4 inhibition increases the ability of T-Cells to track and destroy cancer. X4 is leveraging its CXCR4 research against past experience working with Genzyme’s plerixafor, which is also a CXCR4 blocker.

In an interview with the Journal, Paula Ragan, X4’s chief executive officer, said the CXCR4 protein “acts as a beacon to attract cells to surround a tumor, effectively hiding the tumor from the body’s T cells that would otherwise destroy them.” Developing a therapy to block the protein will prevent the tumors from hiding and allow it to be treated.

Ragan said the Phase Ia trial for X4P-001 will test safety and dosage in a small trial of about 20 people, the Journal reported. If all goes well the company would start a Phase 2a trial by the end of 2016 in around 50 or 60 patients, the Journal said.

In September, Ragan said a CXCR4 antagonist could potentially be paired with promising oncology drugs like Merck & Co. ’s Keytruda, or Bristol-Myers Squibb ’s Opdivo. Keytruda has been shown to be effective in treating patients with three types of cancer, melanoma, lung cancer and mesothelioma. Opdivo is a treatment of patients with metastatic squamous non-small cell lung cancer (NSCLC) with progression on or after platinum-based chemotherapy.

A second drug program, X4P-002, which is currently in preclinical development, is being optimized for the treatment of brain cancers, and is expected to enter the clinic in 2017. Initially the therapy will focus on glioblastoma multiforme, a rare form of lethal brain cancer.

X4, which launched in 2012, nabbed with $37.5 million in backing earlier this year. One of its key investors and advisers is another former Genzyme executive, Henri Termeer. Genzyme’s Termeer has served as an investor and adviser to multiple pharmaceutical companies in the Boston area, including Artax Biopharma and Lysosomal Therapeutics, the Boston Business Journal reported. Other investors in X4 include Cormorant Asset Management.

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