X4 Pharmaceuticals (XFOR) Investor Presentation - Slideshow

Mavorixafor & Clinical Trials - Mavorixafor (X4P-001) is a first-in-class, oral, small molecule allosteric antagonist of chemokine receptor CXCR4 [6] - A global registrational Phase 3 trial of mavorixafor in WHIM syndrome is ongoing [6] - Phase 1b trials are underway in Waldenström's macroglobulinemia and Severe Congenital Neutropenia [6] - Phase 2 open label extension trial for WHIM is ongoing and Phase 3 trial initiated [18] WHIM Syndrome - In WHIM syndrome, infection rates decreased by 54% at 400 mg of mavorixafor [35] - Phase 2 trial data showed a 45-fold increase in TATANC versus lower doses [32] - The estimated U S WHIM population is over 3,500 [23] - The Phase 3 trial in WHIM has a primary endpoint of neutrophil count time above threshold (TAT) of 500 cells/uL [40] Waldenström's Macroglobulinemia (WM) - In Waldenström's Macroglobulinemia, 30-40% of patients have WHIM-like mutations in CXCR4 [47] - WM patients with CXCR4 mutations have ~4-fold likelihood of ibrutinib discontinuation [50] Severe Congenital Neutropenia (SCN) - Severe Congenital Neutropenia is characterized by abnormally low levels of neutrophils (<1,500 cell/ul) [62] Financials - X4 Pharmaceuticals had $1056 million in cash as of June 30, 2020, expected to fund operations into 2022 [85]