X4 Pharmaceuticals (XFOR) 2024 Cantor Fitzgerald Global Healthcare Conference summary

Key milestones and achievements

• Celebrated ten-year anniversary with first drug approval, XOLREMDI, for WHIM syndrome in April.

• MAA filing planned for early 2025 to expand global reach; ex-U.S. partnerships under exploration.

• Commercial launch underway, supported by a national sales team and patient advocacy initiatives.

• Patient hub X4Connect launched to assist with insurance and reimbursement processes.

• Sale of PRV for $105 million added to balance sheet, extending financial runway to late next year.

Clinical data and development pipeline

• XOLREMDI demonstrated 60% reduction in infection rate and significant decrease in infection duration in WHIM patients.

• Phase II data in chronic neutropenia (CN) showed durable neutrophil count elevation and strong safety profile.

• Phase III CN trial underway, targeting completion next year, with final phase II data expected in November.

• Drug being studied in autoimmune, congenital, and idiopathic CN subgroups, with expectations of efficacy across all.

• Phase III endpoints include ANC response and infection rate, mirroring successful WHIM trial design.

Market opportunity and strategy

• WHIM syndrome market estimated at 1,000 diagnosed U.S. patients; CN market at 50,000 diagnosed, with 15,000 as initial target.

• Similar 15,000-patient target market estimated in Europe; global partnerships being pursued.

• Commercial and clinical synergies between WHIM and CN, leveraging same physician networks.

• Physician education, disease awareness, and genetic testing programs deployed to improve diagnosis and access.

• Ongoing efforts to reduce reliance on G-CSF, offering a safer, oral alternative for chronic treatment.