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X4 Pharmaceuticals Reports First Quarter 2022 Financial Results and Provides Corporate Update

Top-line data from pivotal 4WHIM Phase 3 clinical trial in WHIM syndrome expected in 4Q22 Clinical and regulatory updates from ongoing chronic neutropenia

articleX4 Pharmaceuticals, Inc.May 12, 20224/company/x4-pharmaceuticals-inc/news/x4-pharmaceuticals-reports-first-quarter-2022-financial-results-and-provides-corporate-update
X4 Pharmaceuticals Reports First Quarter 2022 Financial Results and Provides Corporate Update

About this update from X4 Pharmaceuticals, Inc.

[{"type":"text","content":"Top-line data from pivotal 4WHIM Phase 3 clinical trial in WHIM syndrome expected in 4Q22 Clinical and regulatory updates from ongoing chronic neutropenia Phase 1b study anticipated during 3Q 2022; Waldenström’s macroglobulinemia Phase 1b study now fully enrolled with results expected in second half of 2022 Conference call to be hosted today at 8:30 a.m. EDT BOSTON, May 12, 2022 (GLOBE NEWSWIRE) -- X4 Pharmaceuticals, (Nasdaq: XFOR), a leader in the discovery and development of novel oral CXCR4-targeted small molecule therapeutics to benefit people with rare immune system disorders, today reported financial results for the first quarter ended March 31, 2022. “As we quickly approach top-line results from our 4WHIM pivotal Phase 3 trial for WHIM syndrome by the end of this year, the X4 team continues to identify and expand the breadth of individuals diagnosed with this rare disease. Initiatives aimed at supporting diagnosis and driving disease awareness, such as our PATH4WARD program offering free genetic sequencing to patients, and publishing peer-reviewed research to help define novel pathogenic genetic variants, are adding to the population of those known to be affected by WHIM and chronic neutropenia,” said Paula Ragan, Ph.D., President and Chief Executive Officer of X4 Pharmaceuticals. “Specifically, to support those affected by chronic neutropenia, we are expanding the availability of genetic testing through PATH4WARD and evaluating patients with congenital, idiopathic, and cyclic chronic neutropenia in our ongoing Phase 1b study. We look forward to providing both clinical data and regulatory updates over the coming months on this important initiative.” Recent Highlights Presented posters at the CIS 2022 Annual Meeting that detailed the PATH4WARD genetic testing program to aid in the diagnosis of WHIM syndrome and chronic neutropenia, and the characterization of a novel pathogenic CXCR4 variant (S341Y).Presented preclinical data on mavorixafor’s ability to significantly enhance the tumor-cell killing activity of the leading commercial and clinical stage Bruton Tyrosine Kinase Inhibitors (BTKi) including ibrutinib, zanubrutinib, pirtobrutinib (LOXO-305) and nemtabrutinib (ARQ-531), at the 2022 AACR Annual Meeting.Completed enrollment in the ongoing Phase 1b study of mavorixafor in combination with ibrutinib in patients with W...

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