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Corporate PresentationFebruary 2026 Nasdaq: XFOR
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Forward-Looking Statements 2 •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 connection with the presentation, contains forward-looking statements within the meaning of applicable securities laws, including the Private Securities Litigation Reform Act of 1995, as amended. These statements may be identified by the words “may,” “will,” “could,” “would,” “should,” “expect,” “plan,” “anticipate,” “intend,” “believe,” “estimate,” “predict,” “project,” “potential,” “continue,” “target,” or other similar terms or expressions that concern X4's expectations, strategy, business, plans, or intentions. Forward-looking statements include, without limitation, implied or express statements regarding: X4’s expectations as to the timing for full enrollment in the ongoing 4WARD Phase 3 study in chronic neutropenia; the results of that study and the timing of a potential sNDA submission in the United States; the timing and prospects for regulatory approval in chronic neutropenia; benefits of cost savings measures; the ability to successfully restructure existing indebtedness; the potential number of patients in the United States with WHIM syndrome or chronic neutropenia and the potential market for mavorixafor due to unmet potential patient needs, as well as related market opportunities; and the sufficiency of X4’s cash resources and pro-forma cash resources, including expectations regarding X4’s cash runway.•Any forward-looking statements in this presentation are based on management's current expectations and beliefs. These forward-looking statements are neither promises nor guarantees of future performance, and are subject to a variety of risks and uncertainties, many of which are beyond X4’s control, which could cause actual results to differ materially from those contemplated in these forward-looking statements, including the risks that: X4 may be unable to advance and commercialize mavorixafor to treat chronic neutropenia or to gain ex-U.S. approval for the treatment of WHIM; the expected sufficiency of X4’s existing cash resources and runway may not be accurate; the expected availability, content, and timing of clinical data from X4’s ongoing clinical trials of mavorixafor may be delayed or unavailable, including the ongoing Phase 3 clinical trial in chronic neutropenia; trials, studies and research programs may not have satisfactory outcomes; earlier trials and studies may not be predictive of later trials and studies; the design and rate of enrollment for clinical trials, including the ongoing Phase 3 clinical trial evaluating mavorixafor in certain chronic neutropenic disorders may not enable successful completion of the trial(s); the commercial opportunity for mavorixafor in chronic neutropenic disorders may be smaller than anticipated; X4 may be unable to obtain and maintain regulatory approvals; adverse safety effects may arise from the testing or use of the company’s product and product candidates; and other risks and uncertainties, including those described in the section entitled “Risk Factors” in X4’s most recent Annual Report on Form 10-K filed with the Securities and Exchange Commission (SEC) and in subsequent filings X4 makes with the SEC from time to time. X4 undertakes no obligation to update the information contained in this presentation to reflect new events or circumstances, except as required by law.•Certain information contained in this presentation relates to or is based on studies, publications, surveys and other data obtained from third-party sources and X4’s own internal estimates and research. While X4 believes these third-party sources to be reliable as of the date of this presentation, it has not independently verified such information. Finally, while X4 believes its own internal research is reliable, such research has not been verified or validated by any independent source. X4 is the owner of various trademarks, trade names and service marks. Certain other trademarks, trade names and service marks appearing in this presentation are the property of third parties. Solely for convenience, the trademarks and trade names in this presentation are referred to without the ® and TM symbols, but such references should not be construed as any indicator that their respective owners will not assert, to the fullest extent under applicable law, their rights thereto.
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Addressing unmet medical needs in hematologyRestructured Company Delivering on the Promise of CXCR4 antagonist Mavorixafor 1. WHIM (Warts, Hypogammaglobulinemia, Infections, Myelokathexis), a rare primary immunodeficiency and chronic neutropenic disorder. 3 •Mavorixafor (XOLREMDI®) approved for the treatment of WHIM syndrome1 in 2024•X4 is now focused on completing the Pivotal Phase 3 4WARD Chronic Neutropenia study •Full enrollment expected in Q3 2026•Top-line data expected in 2H 2027•Potential US FDA approval in 2028 MAXIMIZING THE POTENTIAL OF MAVORIXAFOR IN CHRONIC NEUTROPENIACORPORATE RESTRUCTURING FOLLOWING RECAPITALIZATION•$240M recently raised from blue chip investors including Fidelity, Counterpoint Global and Blackstone•New C-Suite appointments from previous CTI BioPharma senior management team •50% reduction in workforce and on-going cost reductions to reduce monthly burn•Cash runway through 2028
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Former CTI BioPharma Senior Management sold company to SOBI for $1.7B in 2023X4’s Leadership Team – Turnaround, Drug Approval and Commercialization Experience 4 cDavid KirskeChief Financial Officer cAdam Craig, MD PHDExecutive Chair cJohn VolponePresident and COO
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Validated mechanism with the potential to increase neutrophil counts and decrease infection ratesMavorixafor: An oral agent designed to alleviate neutropenia 1. Bainton DF (1980) The Cell Biology of Inflammation, vol 2, pp 1–25. Amsterdam: Elsevier/North-Holland. 2. Furze RC, et al, Immunology. 2008. 3. Mosi, RM, et al, Biochem Pharmacol, 2012. 5 Targeted Mechanism•CXCR4 regulates movement of white blood cells throughout the body2•CXCR4 antagonism results in the migration of white blood cells from the bone marrow3,4 Modified figure from reference 1 Orally active CXCR4 Antagonist•Mavorixafor raises circulating blood levels of neutrophils and lymphocytes4,5,6,7 •Top-line pivotal Phase 3 study in Chronic Neutropenia data expected in 2H 2027•U.S. patent protection expected through 2038 4. Stone ND et al, Antimicrob Agents Chemother. 2007.5. Badolato R, et al. Blood. Published online April 21, 2024;blood.2023022658.6. Warren, JT et al, Oral Presentation American Society of Hematology 2022.7. US Product Label XOLREMDI 2024.
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Chronic Neutropenia – Increasing Risk of Serious Infections and Hospitalization 1. https://ctep.cancer.gov/protocoldevelopment/electronic_applications/docs/ctcae_v5_quick_reference_8.5x11.pdf. 2. Palmblad J, Dufour C, Papadaki HA. Haematologica. 2014 Jul;99(7):1130-1133. 3. Sicre de Fontbrune F, et al. Blood. 2015;126(14):1643-1650. 6 •Frequent and/or serious infections are the primary clinical consequence of chronic neutropenic disorders3•Infections may lead to frequent hospitalizations or result in life-threatening complications, including death4,5 NIH Classification2Absolute Neutrophil Count (ANC)Severe (Grade 4)<500 cells/µLModerate (Grade 3)500 - 1,000 cells/µLMild (Grade 2)1,000 - 1,500 cells/µLNon-clinical (Grade 1)1,500 = Lower Limit of Normal (LLN)1500 500 0 ANC (cells/µL) Increasing Neutrophil Counts >500 cells/µL Clinically Meaningful6,7,8 Highestinfection risk Lowestinfection risk1000 Normal risk 4. Donadieu J, et al. Expert Rev Hematol. 2021;14(10):945-960. 5. Salehi T, et al. Iran J Allergy Asthma Immunol. 2012;11(1):51-56. 6. Platzbecker, U, et al. Blood. 2019 Mar;133(10):1020-1030. 7. Donadieu J, et al. Expert Rev Hematol. 2021 Oct;14(10):945-960.8. Newburger PE, et al. Seminars in Hematology 2013 Jul;50(3):198-206.
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Large number of primary CN patients experiencing severe/life threatening and recurrent infectionsChronic Neutropenia Opportunity – High unmet need in ~15K patients in U.S. 1. Analysis of claims data derived from Veeva Compass 2+ claims with ICD-10 codes: D70.0, D70.4, D70.8, D70.9 between January 2023 – April 2025. 2. Secondary Causes: drug/chemo induced neutropenia, chemotherapy, Transplant patients, ESRD, CKD, MDS, Antipsychotics/anticonvulsants, anemia, dialysis. Duffy Null mutation is a mild form of Neutropenia that was excluded. 3. US HCP Quantitative Market Research Survey, December 2024 (n=95 Hematologists/Oncologists) 7 U.S. PatientsØPrimary Chronic Neutropenia1ØExcluding secondary causes2 ~57,0001 ØSerious recurring Infections2ØInadequately treated ~22,000 ØModerate and Severe Disease3-5ØANC <1000 cells/µL ~15,000 US Target Market for Mavorixafor in CN January 2023 – April 2025 Of the patients you have personally managed in the last 12 months what % of your primary CN patients fall into the following categories?•Experiencing recurrent serious infections4 •Moderate (ANC 500 - 999 cells / µL) or Severe ( <500 cells / µL) with recurrent serious infections US Quantitative Survey of 95 Hematologists/Oncologists3HCP self-reported estimates 4. Recurrent serious infections defined in quantitative survey3 as: at least 2 infections in the past year, requiring the use of antibiotics (intravenous, oral, or topical) AND/OR requiring a visit to healthcare facility (including but not limited to emergency room visit, urgent care facility, primary care physician’s office, or in-patient hospitalization). 5. Veeva Compass claims analysis from 1/2023-4/2025: ~15,000 patients (7% congenital, 93% idiopathic) with 1+ infection (congenital) or 6+ infections (idiopathic) to approximate moderate to severe disease
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Broad opportunity for mavorixafor use as monotherapy or in combination with G-CSF Significant Opportunity to Address Unmet Needs in CN Community 1. X4 Market Research, July 2023 – data on file; ICD-10 Code Research (2017-2023). 2. G-CSF – Granulocyte Colony Stimulating Factor. 8 Current use of G-CSF within primary CN patient populations1•~32% of patients on continuous G-CSF therapy•~26% of patients receiving sporadic/acute G-CSF•~42% of patients not receiving G-CSF therapy MavorixaforMonotherapy Treating patients who are:•Naïve to G-CSF•Intolerant or unresponsive to G-CSF•Using G-CSF acutely, on demand Dose reductions of G-CSF to:•To lessen pain and discomfort•To reduce the long-term risk of malignancies MavorixaforCombinationTherapy
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1320222225263032323435 Intravenous treatment optionsAlternative mechanisms of action to G-CSFSafer treatment optionsDecrease in acute antimicrobial dose / freq.Decrease in G-CSF dose / frequencyLower frequency of infectionsDecrease in proph. antimicrobial dose / freq.Decreased severity of infectionsMore tolerable treatment optionsStabilization of ANC levelsOral treatment options 010203040 Oral Treatment ranked as top unmet need by CN treating physicians 1. US HCP Quantitative Market Research, Sept 2024 (n=95 Hematologists with ~20 CN pts each). 9 Unmet Needs For CN Patients1 (Times Ranked in Top 3 by Respondents; N = 95 Physicians) CN
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10 Phase 2 Proof of Concept Study of Mavorixafor in Chronic Neutropenia (CN)Phase 2 Study N=23 patients Baseline Assessed Safety and Durability of ANC Levels over 6-Month PeriodOnce-Daily Oral Mavorixafor: Monotherapy +/-G-CSF DAY -1 Month 1Month 3 Month 6Study Visits Assessments at Baseline, Month 1, Month 3, and Month 6•At Each Visit: up to 7 blood samples drawn over 8 hours•Daily Mean ANC: mean of absolute neutrophil counts from blood draws over the 8-hour period•ΔANC: ANC at Peak minus ANC at Trough (T0)2 Timepoint Efficacy Assessments
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Study group representative of typical CN population with history of prior infectionsPhase 2 Clinical Study in Chronic Neutropenia: Participant Disposition 1.Congenital CN participants included those with ELANE variant (n=2), VPS13B variant (Cohen syndrome), G6PC3 variant/ deficiency, SRP54 variant (SDS-like syndrome), WASp variant (Wiskott-Aldrich syndrome)2.Modifications to G-CSF dosing allowed after Month 2 at physician’s discretion 11 Participant Disposition (n=23)Type of CNIdiopathic15Congenital1 6Cyclic2SexMale10Female13Mean Age34 Mavorixafor + G-CSF(mean baseline ANC 4034 cells/µL; range 470-11183 cells/µL)BaselineStable G-CSF Total4Adjusted G-CSF2 Total9 Mavorixafor Monotherapy(mean baseline ANC 750 cells/µL; range 180-2356 cells/µL)BaselineTotal10 Phase 2 Study Enrolled a Total of 23 Participants
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Mavorixafor Monotherapy Increased Mean ANC 1. Data set contains two LOCF (last observation carried forward) values: one value missing at M3 assessment, one value missing at M6. 2. One patient discontinued prior to Month 3 assessment (no change from data set presented on 6/27/2024). 3. One patient discontinued prior to Month 6 assessment (no change from data set presented on 6/27/2024). 12 Mean ANC reached normal levels (ANC ≥ 1,500 cells/µL) at 3 and 6 months of treatment Pre-mavorixaforOn mavorixafor 0 500 1000 1500 2000 2500 Me an Mean ANC +/- SE1 Month 3(n=9)2Month 6(n=8)3Month 1(n=10)Baseline(n=10)
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13 Substantial Reductions in G-CSF on Study G-CSF:•Treating physicians chose to reduce injectable G-CSF therapy in 9 of 12 (75%) eligible patients•Three patients with dose adjustments had G-CSF usage stopped by 6 months •Mean ANC maintain at normal levels (>1,500 cells/µL) through Month 6 •Data demonstrates the potential to reduce G-CSF usage with mavorixafor 52% Reduction 70% Reduction -80-70-60-50-40-30-20-100 Month 3n=8Month 6n=9Baseline1 0%Reduction Key TakeawaysMean G-CSF Reduction Over Time
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Mavorixafor maintains normal ANC counts with G-CSF stoppages and doses reductions 14 Data demonstrates the potential to reduce or stop G-CSF usage with mavorixafor no neutropeniamild neutropeniamoderate neutropeniasevere neutropenia Month 3(n=12)Month 6(n=12)Month 1(n=12)Baseline(n=13) 4034457343223121 0100020003000400050006000 Me an ANC (cells/uL) Mean ANC in patients taking Mavorixafor + G-CSFMean ANC +SE
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151.Footnote Placeholder Mean ANC for Moderate and Severe CN in G-CSF Cohort no neutropeniamild neutropeniamoderate neutropeniasevere neutropenia Month 3(n=2)Month 6(n=2)Month 1(n=2)Baseline(n=2) Mean ANC +SE 5791774 36635445 010002000300040005000600070008000 Me an ANC (cells/uL) •2/13 G-CSF cohort subjects were moderate/severe (1 moderate/1 severe)•001-007 was Cyclic CN on Stable G-CSF. Baseline ANC=687 cells/uL•011-009 was Congenital CN whose G-CSF was reduced. Baseline ANC=470 cells/uL 15
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Mavorixafor is well tolerated as both monotherapy and in combination with G-CSFPhase 2 Chronic Neutropenia Study Safety Summary 1. TEAE: treatment-emergent adverse event. 16 Combination (n=13), n (%)Monotherapy (n=10)n (%)Overall (n=23)n (%)Any Related AE10 (76.9)7 (70.0)17 (73.9)Nausea4 (30.8)5 (50.0)9 (39.1)Diarrhea 4 (30.8)3 (30.0)7 (30.4) Treatment-related TEAEs Occurring in >20% of Participants •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 mild/moderate and GI related (nausea and diarrhea)•No drug-related serious adverse events All mild to moderate
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Full enrollment expected Q3 2026 with top-line data expected 2H 2027Pivotal Phase 3 4WARD Study in Chronic Neutropenia 17 Mavorixafor (50%)+/- G-CSF Placebo (50%)+/- G-CSF Global, Double-Blind, Placebo-Controlled Trial ScreeningBaseline Visitand1:1 RandomizationN=176 ANC assessments at baseline and months 1, 2, 3, 6, 9, and 12 months Key Inclusion Criteria for congenital, autoimmune, or idiopathic chronic neutropenia •Absolute Neutrophil Count (ANC): <1,000 cells/µL (moderate and severe disease)•Infection History: 2 or more infections requiring intervention within last 12 monthsPrimary Endpoints: ANC response (>500 cells/µL) and annualized infection rate (AIR)•Both endpoints powered to >96% for ITT populationSecondary Endpoints: Severity and duration of infection, antibiotic use, fatigue, QoL, and safety
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•Increase in the number of U.S. sites•Enhanced engagement with U.S. sites•Consolidating CROs to improve efficiencies•Investment in database mining to identify patients•Global MSL activities solely focused on site interaction and enrollment support Corporate resources dedicated to enrollment enhancing activitiesEmphasis on 4WARD Enrollment 18
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Fully Funded to launchStrong Financial Position 19 (a) Fully diluted common stock outstanding includes pre-funded warrants Cash Fully Diluted Common Stock OutstandingBalance @ 9.30.25$122.2 M88.1MFinancing 10.27.25$146.6 M53.5MTotal post financing$268.8M141.6M
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Potential FDA approval in 2028X4 on Track to Deliver Pivotal Phase 3 Data in 2H 2027 20 •Large market opportunity for mavorixafor in CN in the US•~15,000 patients with moderate and severe disease•Clear medical need for well tolerated oral agent - monotherapy or in combination with G-CSF •New C-Suite team experienced in hematology drug approvals and product launches•Blue chip investor base established after recent PIPE/CMPO•Cash runway through anticipated CN indication launch in 2028 •Clear proof of concept for mavorixafor in CN from Phase 2 study•4WARD study revamped with additional resources•Topline data expected in 2H 2027 with sNDA submission to FDA anticipated late 2027
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Appendix
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22 Mavorixafor: Pipeline and PartnersIndicationPre-clinicalPhase 1Phase 2Phase 3ApprovedPartnersExpected Milestones Mavorixafor Chronic Neutropenia (Congenital, Autoimmune, Idiopathic) Full enrollment 3Q 2026Top-line data 2H 2027 WHIM Syndrome (Warts, Hypogammaglobulinemia, Infections, Myelokathexis) Launched as XOLREMDI® in U.S. 2024EU approval/ launch decision 1H 2026 U.S. FDA Approved Global Pivotal Phase 3 Study Ongoing MAA Under Review in EU Seeking Approvals in MENA Region