X4 Pharmaceuticals, Inc.

X4 Pharmaceuticals announced a $60 million private placement financing led by Coastlands Capital, Bain Capital Life Sciences, and New Enterprise Associates to support continued development of mavorixafor.

• The FDA has approved novel therapies for Duchenne Muscular Dystrophy, including ITF Therapeutics' Duvyzat (givinostat), which targets histone deacetylases to potentially slow muscle deterioration. • Breakthrough gene therapy Lenmeldy received approval for Metachromatic Leukodystrophy, offering the first targeted treatment option with significant survival benefits for pediatric patients. • Multiple rare diseases saw first-ever treatments, including Xolremdi for WHIM syndrome, dual approvals for Niemann-Pick Disease Type C, and Ctexli for Cerebrotendinous Xanthomatosis.

X4 Pharmaceuticals' Marketing Authorization Application (MAA) for mavorixafor to treat WHIM syndrome is under EMA review, potentially the first EU-approved drug for the condition.

The FDA approved Madrigal's Rezdiffra, the first treatment for metabolic dysfunction-associated steatohepatitis (MASH), addressing a significant unmet need in liver disease.

X4 Pharmaceuticals announced positive Phase 2 results for mavorixafor in chronic neutropenia (CN), demonstrating increased neutrophil counts.

X4 Pharmaceuticals reported positive Phase 2 clinical data for mavorixafor in chronic neutropenia (CN), showing increased neutrophil counts.

The FDA has approved XOLREMDI (mavorixafor) for patients 12 years and older with WHIM syndrome, marking the first targeted therapy for this rare immunodeficiency.