Rare Daily Staff

X4 Pharmaceuticals said it completed a $27 million series B financing that will be used to fund a pivotal study of its lead clinical candidate X4P-001, a treatment for the rare primary immunodeficiency disease WHIM syndrome.

WHIM is an acronym for warts, hypogammaglobulinemia, infections, and myelokathexis, the clinical manifestations of WHIM syndrome. It is caused by genetic mutations in the CXCR4 receptor gene, which creates susceptibility to certain types of infections. Current therapy is limited to treatment of acute infections with antibiotics or prevention through the use of intravenous immunoglobulin or G-CSF. There is no approved therapy for the treatment of WHIM syndrome.

The company’s lead experimental therapy X4P-001, an orally-delivered small molecule drug, inhibits the chemokine CXCL12 from binding to the CXCR4 receptor. The CXCR4/CXCL12 pathway has been shown to play an essential role in the normal trafficking of key immune cells to direct immune surveillance throughout the body. In certain rare diseases of the immune system, genetic mutations in the CXCR4 gene result in over-active signaling, resulting in immunodeficiency. Because CXCL12 is overproduced in the tumor microenvironment and promotes mechanisms in tumor growth, it is also pursuing X4P-001 as a potential immunotherapy for patients with kidney cancer.

“We are now in a strong position to advance our lead candidate in WHIM syndrome into a pivotal study in 2018 and to advance our proof of concept studies in oncology,” said Paula Ragan, president and CEO of X4. “This financing recognizes the progress we have made as well as the important milestones that lie ahead in 2018.”

November 22, 2017

Photo: Paula Ragan, president and CEO of X4

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