RARE Daily

X4 Cuts Staff, Narrows Focus to Conserve Cash

July 21, 2022

X4 Pharmaceuticals said it would cut its staff by 20 percent and streamline resources to focus on advancing its lead clinical candidate, mavorixafor, in WHIM syndrome and other chronic neutropenic disorders, while discontinuing work on oncology programs and relying on partnerships to advance them.

Photo: Paula Ragan, president and CEO of X4 Pharmaceuticals

The cuts come less than a month after the company announced a $55 million PIPE offering and amendment to a loan facility that extends the interest-only period by up to 12 months to reduce expenses by $20 million dollars.

The company said the measures are expected to result in an estimated $5 million reduction in spending in 2022 and a $20 million reduction in spending in 2023.

X4’s lead clinical candidate is mavorixafor, a first-in-class, small molecule antagonist of chemokine receptor CXCR4 that is being developed as a once-daily oral therapy. Due to mavorixafor’s ability to antagonize CXCR4 and improve the healthy maturation and mobilization of white blood cells, the company believes it has the potential to provide therapeutic benefit across a wide variety of diseases, including a range of chronic neutropenic disorders and certain types of cancer.

The efficacy and safety of mavorixafor is being evaluated in a global phase 3 clinical trial in patients with WHIM syndrome, a rare, inherited, primary immunodeficiency disease caused by genetic mutations in the CXCR4 receptor gene. It is also studying mavorixafor in two phase 1b clinical trials – one as a monotherapy in patients with chronic neutropenia disorders including congenital, idiopathic, and cyclic neutropenia, and one in combination with ibrutinib in patients with Waldenström’s macroglobulinemia, a rare B-cell lymphoma.

“The sharpening of our strategic focus towards developing treatments for those with chronic neutropenic disorders is intended to maximize the impact to all key stakeholders, including patients and shareholders,” said Paula Ragan, president and CEO of X4 Pharmaceuticals. “We believe that commercializing mavorixafor and providing a new therapeutic option to individuals with life-threatening CN disorders has the potential to revolutionize the treatment landscape, which is currently only served by injectable therapies that have been associated with high-burden side effects and increased cancer risk in some patients.”

Author: Rare Daily Staff

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