X4 Pharmaceuticals(US:XFOR)2025-05-01 14:32Mavorixafor Development and Commercialization - XOLREMDI® (mavorixafor) is approved and launched in the U S in May 2024 for WHIM syndrome, marking the first therapy for this condition[8, 32] - A Marketing Authorization Application (MAA) for WHIM syndrome has been submitted to and accepted by the EMA in January 2025, with potential EU approval and launch expected in the first half of 2026 via a partnership with Norgine[8] - The company anticipates full enrollment in the 4WARD trial for chronic neutropenia in the third or fourth quarter of 2025, with top-line data expected in the second half of 2026[8] Chronic Neutropenia (CN) Market and Unmet Needs - There are an estimated 50,000 chronic neutropenia patients in the U S , with approximately 15,000 representing a high unmet need and a minimum addressable market for mavorixafor[9, 49] - Currently, only injectable Granulocyte Colony-Stimulating Factor (G-CSF) is approved for severe chronic neutropenia, highlighting the need for innovation and alternative treatments[9, 10] - 67% of targeted WHIM hematologists in the U S would also be potential prescribers for chronic neutropenia if mavorixafor is approved for that indication[42] Clinical Trial Results and Analysis - Post-hoc analysis of the WHIM Phase 3 trial showed that 5 out of 13 mavorixafor-treated participants achieved the 4WARD "Responder" threshold, compared to 0 out of 16 in the placebo group[72] - In a Phase 2 study of mavorixafor in chronic neutropenia, mean Absolute Neutrophil Count (ANC) reached normal levels (≥ 1,500 cells/µL) at 3 and 6 months of treatment in the monotherapy group[59] - Physicians substantially reduced G-CSF dosage in 75% of eligible patients in the Phase 2 chronic neutropenia study, while maintaining normal mean ANC levels (>1,500 cells/µL)[68, 67] Financial Position - The company reported funds of $87 7 million as of March 31, 2025, which is expected to support operations into the first half of 2026[8]