US 12,285,424 B2
Methods for treating neutropenia
E. Lynne Kelley, Boston, MA (US); and Sarah Cohen, Boston, MA (US)
Assigned to X4 Pharmaceuticals, Inc., Boston, MA (US)
Filed by X4 Pharmaceuticals, Inc., Boston, MA (US)
Filed on Sep. 9, 2022, as Appl. No. 17/941,509.
Application 17/941,509 is a continuation of application No. PCT/US2021/021713, filed on Mar. 10, 2021.
Claims priority of provisional application 62/987,707, filed on Mar. 10, 2020.
Prior Publication US 2023/0014231 A1, Jan. 19, 2023
Int. Cl. A61K 31/4709 (2006.01); A61K 9/00 (2006.01); A61P 7/00 (2006.01)
CPC A61K 31/4709 (2013.01) [A61K 9/0019 (2013.01); A61P 7/00 (2018.01)] 8 Claims
 
1. A method for treating chronic neutropenia, cyclic neutropenia, or congenital neutropenia in a patient in need thereof, wherein the patient does not have a gain-of-function mutation in the CXCR4 gene, comprising administering to the patient an amount of mavorixafor:

OG Complex Work Unit Chemistry
or a pharmaceutically acceptable salt or composition thereof effective to increase absolute neutrophil count (ANC) to a level greater than or equal to 500 cells/μL and absolute lymphocyte count (ALC) to a level greater than 1000 cells/μL in the patient;
wherein the patient has an absolute neutrophil count (ANC) less than 500 cells/μL at a baseline prior to administering mavorixafor or a pharmaceutically acceptable salt or composition thereof;
the amount of mavorixafor or a pharmaceutically acceptable salt or composition thereof administered is about 200 mg/day, about 300 mg/day, or about 400 mg/day;
the patient has not been diagnosed with WHIM syndrome or with myelokathexis; and
wherein the method results in an increase of ANC to at least 2.0 x the baseline.