X4 Pharmaceuticals (XFOR) Investor Presentation - Slideshow

Company Overview - X4 Pharmaceuticals is developing novel therapies targeting diseases resulting from CXCR4 pathway dysfunction to improve immune cell trafficking[6] - Mavorixafor (X4P-001) is the company's lead product candidate, a first-in-class, oral, small molecule allosteric antagonist of chemokine receptor CXCR4[6] - The company has multiple clinical trials underway, including a global registrational Phase 3 trial of mavorixafor in WHIM syndrome[6] Mavorixafor and WHIM Syndrome - Mavorixafor is a targeted treatment for diseases driven by immune-cell trafficking deficits, with WHIM syndrome as a lead indication[11] - In a Phase 2 trial, 71% of patients achieved maximum Time Above Threshold (TAT) for neutrophil counts, and 85% achieved maximum TAT for lymphocyte counts[34] - Clinical yearly infection rates decreased from 4.63 to 2.41 (a 48% reduction) at 300 mg and 2.14 (a 54% reduction) at 400 mg of mavorixafor in the Phase 2 extension trial[37] - The company is conducting a global registrational Phase 3 trial in WHIM syndrome, with a primary endpoint of absolute neutrophil count time above threshold (TATANC) where the threshold is defined as 500 cells/uL[43] Other Indications and Financials - The company is also investigating mavorixafor in Waldenström's Macroglobulinemia (WM) and Severe Congenital Neutropenia (SCN) in Phase 1b trials[6, 11] - In Waldenström's Macroglobulinemia (WM), patients with CXCR4 mutations have a Very Good Partial Response (VGPR) rate of 9.5% compared to 44.4% for wild-type[51] - As of March 31, 2020, the company had $117.0 million in cash, expected to fund operations into 2022[82]