X4 Pharmaceuticals is laying off 30% of its staff and halting preclinical R&D as the immune system specialist channels its resources into recently approved rare disease drug mavorixafor.
The therapy, which is branded as Xolremdi, received the FDA green light in April 2024 in patients 12 and older with WHIM syndrome (warts, hypogammaglobulinemia, infections and myelokathexis).
Xolremdi was the first drug specifically FDA-approved to treat WHIM, which is a rare, combined primary immunodeficiency and chronic neutropenic disorder caused by an oversignaling of the CXCR4 pathway. The condition causes white blood cells to get stuck in a patient’s bone marrow, preventing them from circulating and protecting the body from infection.
In a Feb. 6 release, X4 said a restructuring was now necessary so the company can “optimize” its U.S. promotion of the drug as well as keep the money flowing to a phase 3 trial of the therapy in chronic neutropenia, a term for an abnormally low concentration of neutrophils in the blood.
It means that 43 employees—equivalent to 30% of the biotech’s employees—are being laid off, along with the discontinuation of the company’s “research efforts” and the closure of a facility in Vienna. All preclinical programs will also be paused.
In their place, the U.S. commercial field team and supporting roles will be scaled up across the company.
Overall, the changes should save about $30 million to $35 million in annual spending, which will pave a cash runway into the first half of 2026.
“This strategic restructuring is being implemented to improve our operational efficiency and capital efficiency as we continue to maximize the global market opportunity for mavorixafor and to benefit the largest number of patients we can worldwide,” X4 CEO Paula Ragan, Ph.D., said in this morning’s release.
“We expect this organizational redesign to sharpen our focus on the execution of our ongoing global pivotal phase 3 clinical trial of mavorixafor in chronic neutropenia while we continue to build WHIM communities through both our U.S. commercial presence and through global partnerships,” Ragan added.
X4 has estimated that there are about 1,000 WHIM patients in the U.S. However, Ragan told Fierce last year that given the ultrarare nature of the disease and the fact that it is likely underdiagnosed, “we will ultimately know what the true prevalence is once the drug is approved over many years.”
Today isn’t the first time that X4 has laid off staff to keep its cash flow under control. Back in 2022, the company reduced its head count by 20% and halted its oncology program in order to focus solely on mavorixafor.