X4 to receive a €28.5 million upfront payment and up to €226 million in potential regulatory and commercial milestone payments in addition to tiered, double-digit royalties up to the mid-twenties
Upfront non-dilutive funds strengthen X4's balance sheet as enrollment ramps up in the company's global Phase 3 clinical trial in chronic neutropenia
Agreement underscores Norgine's commitment to bring transformative therapies to patients in need in these key strategic territories
BOSTON and UXBRIDGE, United Kingdom, Jan. 13, 2025 (GLOBE NEWSWIRE) -- X4 Pharmaceuticals (Nasdaq: XFOR), a company driven to improve the lives of people with rare diseases of the immune system, and Norgine, a leading European specialist pharmaceutical company, today announced that they have entered into an exclusive licensing and supply agreement under which Norgine will commercialize mavorixafor in Europe, Australia, and New Zealand following regulatory approvals.
Mavorixafor is a selective CXCR4 receptor antagonist approved in the U.S. and marketed by X4 as XOLREMDI®, an oral, once-daily treatment for patients 12 years of age and older with WHIM syndrome, a rare primary immunodeficiency. X4 expects to announce shortly the submission of a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for mavorixafor in the treatment of WHIM syndrome, for which it has been granted Orphan Drug Designation by both the EMA and the U.S. Food and Drug Administration. X4 is also developing mavorixafor to treat chronic neutropenia (CN) and is currently conducting a global, pivotal Phase 3 clinical trial in certain CN disorders.
"This strategic agreement is a significant milestone for X4 as we seek to maximize the global potential of mavorixafor and bring in funding for our ongoing global, Phase 3 trial in chronic neutropenia," said Paula Ragan, Ph.D., President and Chief Executive Officer of X4 Pharmaceuticals. "We believe Norgine to be the ideal partner due to their impressive infrastructure and successful commercialization track record in specialty pharmaceuticals, as well as a shared focus on putting patients first. We look forward to expanding access to mavorixafor and continuing to address the unmet needs of those with rare immune disorders."
Janneke van der Kamp, Chief Executive Officer of Norgine, commented on the announcement: "We are very pleased to partner with X4 in this underserved, rare disease space and expand access to mavorixafor to patients in Europe, Australia, and New Zealand. If approved by the respective regulatory bodies, mavorixafor would be the first treatment targeting a key underlying cause of WHIM syndrome, a disease characterized by low white blood cell counts and frequent and/or serious infections. Through this agreement, we continue to expand our innovative portfolio of products and our expertise across rare diseases and specialty markets. This important milestone for our company further underscores Norgine's position as a partner of choice across Europe and ANZ."
Under the terms of the license and supply agreement, X4 will receive €28.5 million in upfront consideration and up to €226 million contingent upon the achievement of certain regulatory and commercial milestones, in addition to escalating double-digit royalties of up to the mid-twenties on any future net sales in the licensed territories. X4 and Norgine will collaborate closely on regulatory filings, with X4 continuing to be responsible for the ongoing global, pivotal Phase 3 4WARD clinical trial evaluating mavorixafor in CN. Norgine will be responsible for all market access and commercialization activities and will eventually hold all marketing authorizations in the licensed territories. X4 will manufacture and supply mavorixafor to Norgine.
About X4 Pharmaceuticals X4 is delivering progress for patients by developing and commercializing innovative therapies for those with rare diseases of the immune system and significant unmet needs. Leveraging expertise in CXCR4 and immune system biology, X4 has successfully developed mavorixafor, an orally available CXCR4 antagonist that is currently being marketed in the U.S. as XOLREMDI® in its first indication. The company is also evaluating additional uses of mavorixafor and is conducting a global, pivotal Phase 3 clinical trial in people with certain chronic neutropenic disorders. X4 is headquartered in Boston, Massachusetts and operates a research center of excellence in Vienna, Austria.
