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Mavorixafor

Phase 3

Neutropenia | Small molecule | Hematology |X4 Pharmaceuticals, Inc.|Last Updated: Jun 5, 2026

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
RandomizedDouble-BlindPLACEBO_CONTROLLEDDMCBiomarker
Total Trials2
Total Enrollment208
FDA Designations
No designations recorded
Clinical Trials (2)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT06056297A Study of Mavorixafor in Participants With Congenital and Acquired Primary Autoimmune and Idiopathic Chronic Neutropenic Disorders Who Are Experiencing Recurrent and/or Serious InfectionsPHASE3 RECRUITING 176 — — Jun 6, 2024Nov 1, 2027Jun 5, 2026114 United States, Argentina +23
NCT04154488A Study of Mavorixafor in Participants With Congenital Neutropenia and Chronic Idiopathic Neutropenia DisordersPHASE1 COMPLETED 32 — — Oct 16, 2021Aug 27, 2024Aug 6, 20257 United States
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Study Endpoints
Primary Endpoints
Co-primary Endpoint: Annualized Infection Rate Based on Infections Adjudicated by Blinded Infection Adjudication Committee (BIAC) During the Treatment Period
Up to 52 Weeks
Co-primary Endpoint: Number of Participants Meeting the Definition of a Positive Absolute Neutrophil Count (ANC) Response
Up to 52 weeks

Positive ANC response: Increase of ANC \>500 cells/microliter (µL) from baseline.

Number of Participants With Treatment-Emergent Adverse Events (TEAEs) After a Single Dose of Mavorixafor
Baseline through Day 1 and 7 days follow-up
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) After Multiple Doses of Mavorixafor
Baseline through Month 6 and 30 days follow-up
Change From Baseline in Absolute Neutrophil Count (ANC) to 8 hours Post-dose On Day 1
Baseline, 8 hours Post-dose On Day 1
Change From Baseline in ANC to Month 6
Baseline, Month 6
Secondary Endpoints
Infection Severity Based on Common Terminology Criteria for Adverse Events (CTCAE) Adjudicated by a BIAC During the Treatment Period
Up to 52 Weeks
Infection Duration Based on Duration of Infections Adjudicated by a BIAC During the Treatment Period in Those Participants who Developed Infections
Up to 52 Weeks
Antibiotic Use Due to Infection, Characterized by the Frequency of Antibiotic Use During the Treatment Period
Up to 52 Weeks
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Study Design & Arms
AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
MavorixaforEXPERIMENTALParticipants will receive mavorixafor orally once daily starting from Day 1 through Week 52.
PlaceboPLACEBO_COMPARATORParticipants will receive placebo to match mavorixafor orally once daily starting from Day 1 through Week 52.
Interventions
NameTypeDescription
MavorixaforDRUGMavorixafor will be administered per schedule specified in the arm description.
PlaceboDRUGPlacebo will be administered per schedule specified in the arm description.
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Eligibility Criteria
Age Range12 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites114

Key Inclusion Criteria: * Diagnosis of congenital or acquired primary autoimmune and idiopathic chronic neutropenic disorder ≥6 months prior to the screening visit that is not attributable to medications, active or recent infections or malignancy. * Congenital Neutropenia, including but not limited...

Countries:United StatesArgentinaAustraliaBelgiumCanadaColombiaCzechiaFranceGeorgiaGermanyGreeceHungaryIndiaIsraelItalyMalaysiaPortugalRomaniaSerbiaSpainSwitzerlandThailandTurkey (Türkiye)UkraineUnited Kingdom
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Recent Changes (Last 90 Days)
LOWJun 5, 2026NCT06056297lastUpdatePostDate: changed
LOWJun 5, 2026NCT06056297lastUpdatePostDate: changed
LOWJun 5, 2026NCT06056297lastUpdatePostDate: changed
LOWJun 5, 2026NCT06056297lastUpdatePostDate: changed
LOWMay 26, 2026NCT06056297primaryCompletionDate: changed
LOWMay 24, 2026NCT06056297studyFirstPostDate: changed