X4 Pharmaceuticals, Inc. · NASDAQ:XFOR
X4 Pharmaceuticals — Investor Presentation (October 2026)
20 slides from X4 Pharmaceuticals's investor presentation, October 2026
Slide 1: Corporate Presentation
Nasdaq: XFOR Corporate Presentation October 2026
Slide 2: Forward-Looking Statements
Forward-Looking Statements • This presentation, including any printed or electronic copy of these slides, the talks given by the presenters, the information communicated during any delivery of the presentation and any question and answer sessions and any documents or materials distributed at or in c
Slide 3: Delivering on the Promise of CXCR4 antagonist Mavorixafor
Delivering on the Promise of CXCR4 antagonist Mavorixafor Focused on improving the lives of people with rare hematology diseases MAXIMIZING THE POTENTIAL OF REBUILT & REPOSITIONED THE COMPANY MAVORIXAFOR IN CHRONIC NEUTROPENIA OVER THE LAST 12 MONTHS • Mavorixafor (XOLREMDI®) approved for the • New
Slide 4: X4's Leadership Team – Turnaround, Drug Approval and Commercialization Experience
X4’s Leadership Team – Turnaround, Drug Approval and Commercialization Experience Former CTI BioPharma Senior Management sold company to SOBI for $1.7B in 2023 c MD PHD Adam Craig, c John Volpone David cKirske Executive Chair President and COO Chief Financial Officer 4
Slide 5: Mavorixafor: An oral agent designed to alleviate neutropenia
Mavorixafor: An oral agent designed to alleviate neutropenia Validated mechanism with the potential to increase neutrophil counts and decrease infection rates Targeted Mechanism • CXCR4 regulates movement of white blood cells throughout the body2 • CXCR4 antagonism results in the migration of white
Slide 6: Chronic Neutropenia (CN) – Increasing Risk of Serious Infections and Hospitalization
Chronic Neutropenia (CN) – Increasing Risk of Serious Infections and Hospitalization NIH Classification2 Absolute Neutrophil Count (ANC) Increasing Neutrophil Counts >500 cells/µL Clinically Meaningful6,7,8 Severe (Grade 4) <500 cells/µL 1000 Lowest infection risk Moderate (Grade 3) 500 - 1,000 cell
Slide 7: Chronic Neutropenia Opportunity – High unmet need in ~15K patients in U.S.
Chronic Neutropenia Opportunity – High unmet need in ~15K patients in U.S. Large number of primary CN patients experiencing severe/life threatening and recurrent infections U.S. Patients Ø Primary Chronic Neutropenia1 January 2023 – April 2025 Ø Excluding secondary ~57,0001 causes2 US Quantitative S
Slide 8: Significant Opportunity to Address Unmet Needs in CN Community
Significant Opportunity to Address Unmet Needs in CN Community Broad opportunity for mavorixafor use as monotherapy or in combination with G-CSF Treating patients who are: Dose reductions of G-CSF • Naïve to G-CSF aimed: Mavorixafor Mavorixafor • Intolerant or unresponsive • To lessen pain and Combi
Slide 9: Oral Treatment ranked as top unmet need by CN treating physicians
Oral Treatment ranked as top unmet need by CN treating physicians CN Unmet Needs For CN Patients1 (Times Ranked in Top 3 by Respondents; N = 95 Physicians) 35 Oral treatment options 34 Stabilization of ANC levels 32 More tolerable treatment options 32 Decreased severity of infections 30 Decrease in
Slide 10: Phase 2 Proof of Concept Study of Mavorixafor in Chronic Neutropenia
Phase 2 Proof of Concept Study of Mavorixafor in Chronic Neutropenia Phase 2 Study N=23 patients Assessed Safety and Durability of ANC Levels over 6-Month Period Once-Daily Oral Mavorixafor: Monotherapy +/-G-CSF DAY -1 Study Visits Baseline Month 1 Month 3 Month 6 Timepoint Efficacy Assessments Asse
Slide 11: Phase 2 Clinical Study in Chronic Neutropenia: Participant Disposition
Phase 2 Clinical Study in Chronic Neutropenia: Participant Disposition Study group representative of typical CN population with history of prior infections Phase 2 Study Enrolled a Total of 23 Participants Participant Disposition (n=23) Mavorixafor Monotherapy (mean baseline ANC 750 cells/µL; range
Slide 12: Mavorixafor Monotherapy Increased Mean ANC
Mavorixafor Monotherapy Increased Mean ANC Mean ANC reached normal levels (ANC ≥ 1,500 cells/µL) at 3 and 6 months of treatment Mean ANC +/- SE1 2500 2000 1500 Pre- mavorixafor On 1000 mavorixafor 500 0 Baseline Month 1 Mean Month 3 Month 6 (n=10) (n=10) (n=9)2 (n=8)3 1. Data set contains two LOCF (
Slide 13: Substantial Reductions in G-CSF on Study
Substantial Reductions in G-CSF on Study Mean G-CSF Reduction Over Time Key Takeaways Month 3 Month 6 Baseline n=8 n=9 G-CSF: 0 • Treating physicians chose to reduce injectable G-CSF -10 0% therapy in 9 of 12 (75%) eligible patients -20 Reduction 52% • Three patients with dose adjustments had G-CSF
Slide 14: Mavorixafor maintains normal ANC counts with G-CSF stoppages and dose reductions
Mavorixafor maintains normal ANC counts with G-CSF stoppages and dose reductions Mean ANC in patients taking Mavorixafor + G-CSF Mean ANC +SE 6000 5000 4573 4322 4034 ANC (cells/uL) 4000 3121 3000 2000 1000 0 Baseline Month 1 Mean Month 3 Month 6 (n=13) (n=12) (n=12) (n=12) no neutropenia mild neutr
Slide 15: Phase 2 Chronic Neutropenia Study Safety Summary
Phase 2 Chronic Neutropenia Study Safety Summary Mavorixafor is well tolerated as both monotherapy and in combination with G-CSF • Overall safety profile consistent with prior studies • No new safety issues observed when dosed in combination with G-CSF • Most frequent treatment-related TEAEs1 were m
Slide 16: Pivotal Phase 3 4WARD Study in Chronic Neutropenia
Pivotal Phase 3 4WARD Study in Chronic Neutropenia Full enrollment expected Q4 2026 with top-line data expected H1 2028 Global, Double-Blind, Placebo-Controlled Trial Mavorixafor (50%) Baseline Visit +/- G-CSF and Screening 1:1 Randomization N=126 Placebo (50%) +/- G-CSF ANC assessments at baseline
Slide 17: Financial Position
Financial Position Cash Runway Expected into 2029 June 30, 2026 Fully Diluted Cash Common Stock Outstanding $208 M 145.6 M Fully diluted common stock outstanding includes pre-funded warrants 17
Slide 18: X4 on Track to Deliver Pivotal Phase 3 Data in H1 2028
X4 on Track to Deliver Pivotal Phase 3 Data in H1 2028 Potential FDA approval late 2028/early 2029 • C-Suite team experienced in hematology drug approvals and product launches • Blue chip investor base established after PIPE/CMPO • Cash runway expected into 2029 • Clear proof of concept for mavorixa
Slide 19: Appendix
PHARMACEUTICALS, Appendix
Slide 20: Mavorixafor: Pipeline and Partners
Mavorixafor: Pipeline and Partners Expected Indication Pre-clinical Phase 1 Phase 2 Phase 3 Approved Partners Milestones Full enrollment Chronic Neutropenia Q4 2026 (Congenital, Autoimmune, Global Pivotal Phase 3 Study Ongoing Top-line data Idiopathic) H1 2028 Mavorixafor U.S. FDA Approved Launched
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