X4 Pharmaceuticals (XFOR) investor relations material

Executive summary

• FDA approved XOLREMDI (mavorixafor) for WHIM syndrome in April 2024; U.S. launch began May 2024.

• Entered exclusive license and supply agreement with Norgine for Europe, UK, Australia, and New Zealand in January 2025.

• Implemented a strategic restructuring in February 2025, reducing workforce by 30% and closing Vienna facility.

• Completed sale of a Priority Review Voucher for $105 million in Q2 2024.

• Ongoing pivotal Phase 3 trial (4WARD) for mavorixafor in chronic neutropenia.

Financial highlights

• Total revenue for six months ended June 30, 2025: $30.8M, up from $0.6M year-over-year, driven by Norgine license and increased product sales.

• Net loss for six months ended June 30, 2025: $25.5M, compared to net income of $39.1M in prior year (which included $105M gain on PRV sale).

• Cash, cash equivalents, and marketable securities as of June 30, 2025: $63.0M; accumulated deficit: $540.8M.

• Operating expenses decreased due to restructuring: R&D down $3.8M and SG&A down $6.1M year-over-year for the six-month period.

• Product revenue for six months ended June 30, 2025: $2.7M, up from $0.6M year-over-year.

Outlook and guidance

• Cash and equivalents will not fund operations for the next 12 months; additional capital required to meet debt covenants and continue as a going concern.

• Strategic restructuring expected to reduce annual spending by $30–35M and support operations into the first half of 2026, subject to compliance with loan covenants.

• Ongoing efforts to raise capital through equity offerings, debt, and partnerships; $40M equity purchase agreement with Lincoln Park signed in June 2025.