X4 Pharmaceuticals (XFOR) Post-EHA Corporate Update - Slideshow

Mavorixafor Clinical Trial & Results - Mavorixafor, a first-in-class CXCR4 antagonist, is being developed as a once-daily oral capsule targeting the mechanism of WHIM syndrome [19] - Phase 2 study data informed the design of the ongoing Phase 3 registration trial [42] - The Phase 3 trial uses a 400 mg orally once daily dose of Mavorixafor [29, 42] - The primary endpoint for the Phase 3 trial is TATANC (time above threshold for absolute neutrophil count) [29, 42] - Top-line Phase 3 data is expected in 2022 [45] Phase 2 Study Outcomes - At doses of 300 or 400 mg/day, the mean TATANC was 12.6 (±9.8) hours (N=7) compared to 2.8 (±3.5) hours or less for patients (N=4) treated at doses of 150 mg or lower [30] - The mean TATALc was 16.9 (±5.8) hours [30] - The yearly infection rate decreased from 4.63 events to 2.27 events on mavorixafor 300 mg and 400 mg once daily [35] - An average 75% reduction in the number of cutaneous warts was observed [40] WHIM Syndrome & Mavorixafor's Potential - There is a large unmet need for effective WHIM treatment [15, 45] - Mavorixafor is a promising targeted therapy that could lead to improved and durable clinical efficacy in patients with WHIM syndrome [45]