X4 Pharmaceuticals(US:XFOR)2021-11-19 19:01Mavorixafor's Potential & Target Diseases - Mavorixafor, a Phase 3 candidate, is being advanced to potentially treat over 30,000 patients in the US and EU markets across multiple indications[7] - The company is targeting 1,000-3,700 U S WHIM syndrome patients, 2,000-3,000 U S Waldenström's Macroglobulinemia patients, and 5,000-10,000 U S Chronic Neutropenia patients[10] - The global G-CSF market for all uses is over $4 billion, with the U S market for severe chronic neutropenia estimated at $120-460 million[57] Clinical Trial Updates & Data - Top-line data from the Phase 3 trial in WHIM syndrome is expected in 4Q 2022, with a potential NDA filing in 1Q 2023[7, 98] - Initial data from the Phase 1b trial in Chronic Neutropenia and additional data in Waldenström's Macroglobulinemia are expected in 4Q 2021[7, 98] - Phase 2 trial in WHIM syndrome demonstrated a 6-fold increase in TATANC and over 80% reduction in annualized infection rates[37] Pipeline & Expansion - A new molecule (X4P-002) is expected to enter the clinic in 2H 2022 for oncology indications, targeting other leukemias and lymphomas which is >25,000[7, 10] - The company anticipates additional data at all doses and initial response data (OR, MR) for Waldenström's Macroglobulinemia in 4Q 2021[92] - Newly identified D84H CXCR4 mutation suggests additional ~1,250-2,500 WHIM patients[49] Financial Status - The company had $78 million in cash and equivalents as of September 30, 2021, expected to fund operations into Q4 2022, excluding $19.5 million in net proceeds from equity sale in November 2021[100, 101, 105]