X4 Pharmaceuticals, Inc. · NASDAQ:XFOR

X4 Pharmaceuticals — Technical Presentation (November 2022)

11 slides from X4 Pharmaceuticals's technical presentation, November 2022

  1. Slide 1: Administration of Mavorixafor Increases White Blood Cell Counts Across Different Disease States

    Administration of Mavorixafor, an Orally Available CXCR4 Antagonist, Increases Total White Blood Cell, Neutrophil, Lymphocyte, and Monocyte Counts Across Different Disease States David C. Dale,1,* David F. McDermott,2 Diego Cadavid,3 Kelly Walkovich,4 Julia T. Warren,5 Jeffrey Matous,6 Xia Luo,3 Wei

  2. Slide 2: Disclosures

    Disclosures • David C. Dale has served as a consultant for and has received research funding and honoraria from X4 Pharmaceuticals 2

  3. Slide 3: Oral CXCR4 Antagonist Mavorixafor Is Under Investigation in Clinical Trials

    Oral CXCR4 Antagonist Mavorixafor Is Under Investigation in Clinical Trials for Various Disease States • Leukopenia, including neutropenia, lymphocytopenia, and monocytopenia, is a common feature of many diseases and may lead to increased susceptibility to common and opportunistic infections in affe

  4. Slide 4: WHIM Syndrome: Improvements in Hematological Parameters and Infection Rates in Phase 2

    WHIM Syndrome Improvements Were Observed in Hematological Parameters and Infection Rates With Mavorixafor Treatment in a Phase 2 Trial A phase 2 trial (NCT03005327) evaluated the safety and tolerability of escalating doses of mavorixafor (50–400 mg QD) in adults with WHIM syndrome.1 Mavorixafor is c

  5. Slide 5: Chronic Neutropenic Disorders: WBC Increases With a Single Dose

    Chronic Neutropenic Disorders Increases in WBC Counts Were Observed With a Single Dose of Mavorixafor An ongoing phase 1b, open-label, multicenter trial (NCT04154488) evaluating the safety, tolerability, and early efficacy of mavorixafor (400 mg once daily) alone or with concurrent G-CSF use across

  6. Slide 6: Fold Change by Type of Chronic Neutropenic Disorders and G-CSF Use

    Chronic Neutropenic Disorders Fold Change by Type of Chronic Neutropenic Disorders and G-CSF Use Mean maximum fold change in ANC Mean maximum fold change in ANC by by type of CNDs (n=15)a G-CSF use (n=15)a 6 8 day 1 after a single dose of mavorixafor day 1 after a single dose of mavorixafor Mean max

  7. Slide 7: Waldenström's Macroglobulinemia: Mavorixafor With Ibrutinib

    Waldenström’s Macroglobulinemia Sustained Improvements in Hematological Parameters Were Observed With Mavorixafor in Combination With Ibrutinib An ongoing phase 1b trial (NCT04274738) evaluates mavorixafor (200–600 mg QD) in combination with ibrutinib (420 mg QD) in participants with WM with MYD88 a

  8. Slide 8: Advanced ccRCC: Mavorixafor With Axitinib

    Advanced Clear Cell Renal Cell Carcinoma Improvements Were Observed in Hematological Parameters With Mavorixafor in Combination With Axitinib A phase 1/2 trial (NCT02667886) evaluated the safety and tolerability of escalating doses of mavorixafor (200–600 mg QD) in combination with axitinib (5 mg tw

  9. Slide 9: Conclusions

    Conclusions Ongoing studies in WHIM syndrome, CNDs, WM, and completed studies in ccRCC show that1,2: ─ Oral administration of mavorixafor alone or in combination with G-CSF, ibrutinib, or axitinib increased total WBC and all differential leukocyte counts examined over both short-term or long-term tr

  10. Slide 10: Acknowledgements

    Acknowledgements • The authors would like to thank the study participants, study sites, vendors, and everyone involved in the clinical trials and the clinical development of mavorixafor • Ibrutinib used in WM clinical trial was provided by Pharmacyclics LLC, an AbbVie Company • Axitinib used in ccRC

  11. Slide 11: Thank you!

    PHARMACEUTICALS Thank you!

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