Founded by Genzyme Alumni, Cambridge’s X4 Pharma Scores $27M

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The company plans to use the cash to advance its two lead drug programs into the clinic.

X4 Pharmaceuticals announced it had closed a $27 million Series B financing. The round was led by Cormorant Asset Management with new and existing investors joining in.

The company plans to use the proceeds to advance its two lead drug programs into the clinic. One is for renal cell carcinoma, which will move into Phase II clinical trials. The other is for WHIM syndrome, a primary immunodeficiency disease, which will advance to Phase III trials.

“We are incredibly pleased to have the support of Cormorant Asset Management and additional new and existing investors to support our mission of bringing innovative treatments to patients with cancer and rare diseases,” said Paula Ragan, X4’s president and chief executive officer, in a statement. “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. This financing recognizes the progress we have made as well as the important milestones that lie ahead in 2018.”

X4 Pharmaceuticals was founded in 2015 by Henri Termeer, former chairman, president and chief executive officer of Genzyme Keith Flaherty, director of the Henri and Belinda Termeer Center for Targeted Therapies at the Cancer Center at Massachusetts General Hospital, Renato Skerlj, vice president of Drug Discovery and Preclinical Development at Lysosomal Therapeutics, and Richard Peters, president and chief executive officer of Merrimack Pharmaceuticals. Both Skerlj and Peters had previous roles at Genzyme.

Brittany Meiling, writing for Endpoints News, says, “X4 has moved quickly. The company emerged in 2015 with a $37.5 million launch round and an oncology asset it had picked up from Sanofi‘s toss pile. The company’s oral, small molecule drug candidates inhibit the binding of chemokine CXCL12 to CXCR4, a receptor-ligand pair that plays a role in normal immune surveillance.”

She notes that the company has pushed its asset through Phase I/II testing in renal cell carcinoma and melanoma, but adds, “Interestingly, the company has found an additional application for CXCR4 inhibitors (and faster path to market) by testing the tech in patients with a rare disease called WHIM syndrome.”

There are currently no approved therapies for WHIM syndrome, which is caused by genetic mutations that make the patient susceptible to infections.

“Having been an investor in the earliest stages of X4, we have witnessed the strong progress the company has made and the impact these treatments are having for patients,” said Bihua Chen, Cormorant’s chief executive officer and portfolio manager. “X4 has built a differentiated approach rooted in strong science and clear regulatory pathways. We are pleased to support X4’s continuing work in developing and commercializing innovative therapies for primary immunodeficiencies and cancer.”

The company also announced today that David Dale will present the first clinical data from its trial of X4P-001-RD in patients with WHIM syndrome at the 59th Annual Meeting of the American Society for Hematology (ASH) on Dec. 9. Dale is a professor of medicine at the University of Washington Medicine in Seattle.

“We are very pleased to share preliminary data showing that X4P-001-RD has a meaningful impact on the levels of circulating white blood cells in this severely immunodeficient patient population,” Ragan said in a statement. “These data demonstrate the potential of X4P-001-RD to benefit patients with WHIM who otherwise have no approved treatment options and support our goal of advancing toward a pivotal study.”

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