X4 Pharmaceuticals (XFOR) Corporate presentation summary

Strategic focus and restructuring

• Prioritizing development of mavorixafor for chronic neutropenia, with pivotal Phase 3 4WARD study underway and full enrollment expected in Q3 2026.

• $240M raised from major investors, new C-suite from CTI BioPharma, and a 50% workforce reduction to extend cash runway through 2028.

• Cash position of $268.8M post-financing, fully funding operations through anticipated product launch.

Product and clinical development

• Mavorixafor (XOLREMDI) approved for WHIM syndrome in 2024; targeting chronic neutropenia as next major indication.

• Oral CXCR4 antagonist shown to increase neutrophil counts and reduce infection rates in Phase 2 studies.

• Phase 2 data demonstrated normalization of ANC and significant reduction or stoppage of G-CSF usage, with favorable safety profile.

• Pivotal Phase 3 4WARD study is a global, double-blind, placebo-controlled trial with top-line data expected in 2H 2027 and potential FDA approval in 2028.

Market opportunity and unmet need

• Approximately 15,000 U.S. patients with moderate to severe chronic neutropenia experience recurrent, serious infections and are inadequately treated.

• Oral treatment is ranked as the top unmet need by treating physicians, with mavorixafor positioned as monotherapy or in combination with G-CSF.

• Large market opportunity supported by clear medical need for a well-tolerated oral agent.