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X4 Pharmaceuticals Announces Key Enrollment Milestone Achievements in Ongoing Mavorixafor Clinical Trials and Reports Second Quarter Financial Results
Twenty-three patients now enrolled in ongoing pivotal Phase 3 trial in WHIM syndrome, surpassing minimum needed for primary endpoint analyses; enrollment to
About this update from X4 Pharmaceuticals, Inc.
[{"type":"text","content":"Twenty-three patients now enrolled in ongoing pivotal Phase 3 trial in WHIM syndrome, surpassing minimum needed for primary endpoint analyses; enrollment to complete in 3Q21 with top-line data expected in 4Q22 Minimum number of patients now enrolled to determine optimal dosing of mavorixafor in Waldenström’s macroglobulinemia Phase 1b trial; continue to expect high-dose safety and efficacy data, as well as clinical response outcomes, in 4Q21 Initial data from Phase 1b trial in Severe Congenital Neutropenia (SCN) expected in 4Q21, as company explores expanded use of mavorixafor across broader chronic neutropenia populations Conference call today at 8:30 a.m. ET BOSTON, Aug. 03, 2021 (GLOBE NEWSWIRE) -- X4 Pharmaceuticals, Inc. (Nasdaq: XFOR), a leader in the discovery and development of novel therapies targeting diseases of the immune system resulting from dysfunction of the CXCR4 pathway, today reported financial results for the second quarter and six months ended June 30, 2021. The company also announced key enrollment milestone achievements for its lead product candidate, mavorixafor, a novel, oral small molecule currently being evaluated in a Phase 3 clinical trial (4WHIM) for patients with WHIM (warts, hypogammaglobulinemia, infections, and myelokathexis) syndrome and in two Phase 1b clinical trials for patients with Waldenström’s macroglobulinemia and Severe Congenital Neutropenia (SCN) and chronic neutropenia disorders, respectively. “We are very encouraged by the strong interest from both the physicians and patients participating in our mavorixafor clinical programs,” said Paula Ragan, Ph.D., President and Chief Executive Officer of X4 Pharmaceuticals. “With enrollment nearly complete in the 52-week placebo-controlled Phase 3 trial in WHIM syndrome, our initial indication for mavorixafor, and continued supportive data coming from our ongoing open-label Phase 2 trial in WHIM, we are starting to ramp up our pre-commercial planning, as we now look forward to Phase 3 top-line data in the fourth quarter of 2022. In addition, we are making strong progress in our ongoing Phase 1b clinical trial in Waldenström’s and intend to announce preliminary high-dose data along with certain response measures from this trial in the fourth quarter of 2021 that we believe will build on the low- and mid-dose data we presented at EHA this past J...