Cambridge, MA-based startup X4 Pharmaceuticals just bagged $27 million to take its two lead drugs — one for cancer, the other for rare disease — to Phase II and III trials.
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.
Backed with cash from Genzyme founder Henri Termeer, the company has taken the asset through Phase I/II testing in renal cell carcinoma and melanoma.
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.
WHIM is an immunodeficiency disease caused by genetic mutations that cause the patient to be susceptible to certain infections. There are no approved therapies for WHIM syndrome. X4 is now in a Phase II/III pivotal study in WHIM patients that could bring the drug to market.
This latest infusion of cash will help take the programs forward. The Series B round was led by Boston’s Cormorant Asset Management, with participation from other new and existing investors.
“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, in a statement. “This financing recognizes the progress we have made as well as the important milestones that lie ahead in 2018.”
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