X4 Pharmaceuticals, Inc. · NASDAQ:XFOR
X4 Pharmaceuticals — Investor Day (June 2024)
31 slides from X4 Pharmaceuticals's investor day presentation, Jun 27, 2024
Slide 1: Mavorixafor in Chronic Neutropenia: Interim Phase 2 Data Investor Event
1 Mavorixafor in Chronic Neutropenia Interim data from ongoing Phase 2 clinical trial Investor Event June 27, 2024 Exhibit 99.2
Slide 2: Forward-Looking Statements
22 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 session and any documents or materials distributed at or in co
Slide 3: Today's Agenda
33 Today’s Agenda Guest Speakers Peter E. Newburger, MD Physician-scientist, Professor and Vice Chair for Research, Department of Pediatrics/Division of Hematology-Oncology, UMass Chan Medical School. Editor-in- chief, Pediatric Blood & Cancer Jean Donadieu, MD, PhD Pediatrician and epidemiologist,
Slide 4: X4's Growing Momentum Addressing Unmet Needs in Rare Immune Disorders
44 X4’s Growing Momentum Addressing Unmet Needs in Rare Immune Disorders 1. WHIM (Warts, Hypogammaglobulinemia, Infections, Myelokathexis) See www.xolremdi.com for Prescribing Information; 2. Current funds include $82 million in cash and equivalents as of March 31, 2024 + $105 million in proceeds fr
Slide 5: Positive Interim Phase 2 Results Support Potential of Mavorixafor in CN
55 Positive Interim Phase 2 Results Support Potential of Mavorixafor in CN Summary of today’s presentation Pivotal Trial in CN initiated Compelling Commercial Opportunity Global, pivotal 4WARD Phase 3 clinical trial now screening patients across multiple international sites Significant rare disease
Slide 6: Overview of Chronic Neutropenia
6 Overview of Chronic Neutropenia Overview of Chronic Neutropenia
Slide 7: Chronic Neutropenia: Well Defined Market with Limited Treatment Options
77 Chronic Neutropenia: Well Defined Market with Limited Treatment Options 1. X4 Market Research, July 2023 – data on file; ICD-10 Code Research (2017-2023). 2. https://www.cancernetwork.com/view/fda-approves-new-indication-neupogen- chronic-neutropenia ~50,0001 U.S. Prevalence: total diagnosed with
Slide 8: Risk of Serious, Recurrent Infections Correlated to Severity of CN
88 • Frequent and/or severe 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 Classification2 Absolute Neutrophil Count (ANC) Severe (Grade 4) <500 c
Slide 9: Mavorixafor's Validated Mechanism of Action
9 Overview of Chronic Neutropenia Mavorixafor’s Validated Mechanism of Action
Slide 10: Validated Mechanism Shown to Increase Circulating Neutrophils
1010 Validated Mechanism Shown to Increase Circulating Neutrophils 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. 4. Stone ND et al, Antimicrob Agents Chemoth
Slide 11: Mavorixafor Sustainably Raised ANC over Weeks in Phase 3 4WHIM Trial
1111 Mavorixafor Sustainably Raised ANC over 52 Weeks in Phase 3 4WHIM Trial * Week 13 p=0.0049, Week 26 p=0.0397, Week 39 p=0.0196. a. Calculated as the mean of absolute cell counts over the 24-hour assessment period; b. At week 52, 3 of 17 placebo patients were given mavorixafor in advance of thei
Slide 12: ANC Increase Resulted in Clinical Infection Benefits in Phase 3 4WHIM Trial
1212 ANC Increase Resulted in Clinical Infection Benefits in Phase 3 4WHIM Trial1,2 Mean ANC increases of >500 cells/μL reduced infection rate, duration, and severity ~60% reduction in annualized infection rate Fewer patients with severe infection Average infection duration was 5 weeks shorter Total
Slide 13: Interim Phase 2 CN Trial Results
13 Overview of Chronic Neutropenia Interim Phase 2 CN Trial Results
Slide 14: Objectives of Interim Analysis of Phase 2 Clinical Trial in Chronic Neutropenia
1414 Objectives of Interim Analysis of Phase 2 Clinical Trial in Chronic Neutropenia • Explore durability of Phase 1b study results showing 100% response (>500 cells/µL) to single dose of mavorixafor +/- G-CSF1 Assess the ability of mavorixafor monotherapy to durably increase ANC in severe CN partic
Slide 15: Assessing Mavorixafor in 6-Month CN Phase 2 Clinical Trial
1515 Assessing Mavorixafor in 6-Month CN Phase 2 Clinical Trial 1. Modifications to G-CSF dosing allowed after Month 2 at doctor’s discretion; additional data expected in late 2024. 2. For one participant, trough = first available sample Mavorixafor dosed orally once-daily with or without background
Slide 16: Interim Analysis of Six-Month Phase 2 Clinical Trial in Chronic Neutropenia
1616 Interim Analysis of Six-Month Phase 2 Clinical Trial in Chronic Neutropenia Today’s presentation is focused on monotherapy and stable-dose G-CSF groups Participant Disposition as of May 14, 2024 Data Cut Types of chronic neutropenia studied: Idiopathic = 14; Congenital = 7; Cyclic = 2 1. Three
Slide 17: 100% of Evaluated Study Participants Achieved Target ∆ANC at Month 6
1717 100% of Evaluated Study Participants Achieved Target ∆ANC at Month 6 Percentage of Participants Achieving Target ANC Increase of >500 cells/µL at Each Timepoint TOTAL PARTICIPANTS (n=14) • All had at least one timepoint to assess target ∆ANC Completed Participants (n=6) • 6 (out of 14) complete
Slide 18: Mavorixafor Monotherapy: Durable Increases in Mean ANC
1818 0 500 1000 1500 2000 2500 Mean Mean ANC +/- SE Month 3 (n=8)a Month 6 (n=3)b Month 1 (n=10) Baseline (n=10) Mavorixafor Monotherapy: Durable Increases in Mean ANC a. Samples from one (1) participant who completed the study were unevaluable at M3. b. Samples from one (1) participant who complete
Slide 19: Mavorixafor Monotherapy: Robust Daily Coverage in ANC
1919 0 500 1000 1500 2000 2500 3000 Baseline (n=10) Month 1 (n=10) Month 3 (n=8) Month 6 (n=3) M ea n AN C ce lls /µ L ANC Trough ±SE Mavorixafor Monotherapy: Robust Daily Coverage in ANC • Mean ANC Peak exceeded 1500 cells/µL Lower Limit of Normal (LLN) through 6 months • Mean ANC Trough increased
Slide 20: Mavorixafor Monotherapy: Severe CN Participants Achieve Target ANC Increase
2020 0 500 1000 1500 Mean M ea n AN C (c el ls /µ L) Mavorixafor Monotherapy: Severe CN Participants Achieve Target ANC Increase a. Samples from one (1) participant who completed the study were unevaluable at M3 Month 3 Month 6Month 1Baseline (n=5) (n=5) (n=3)a (n=2) Mean ANC +/- SE Severe Chronic N
Slide 21: Mavorixafor + Stable-Dose G-CSF: Robust Increases in ANC from Baseline
2121 0 1000 2000 3000 4000 5000 Mean M ea n AN C C ha ng e (c el ls /µ L) Mavorixafor + Stable-Dose G-CSF: Robust Increases in ANC from Baseline Month 3 (n=4) Month 6 (n=3) Month 1 (n=4) Mean Change from Baseline in ANC +/- SE Normalized to Baseline ANC • Due to G-CSF dose/ANC variability • ANCs at
Slide 22: Phase 2 Chronic Neutropenia Study Safety Summary from Interim Analysis
2222 Phase 2 Chronic Neutropenia Study Safety Summary from Interim Analysis Overall safety profile consistent with prior studies No new safety issues when dosed in combination with G-CSF No deaths and no drug-related serious adverse events (SAEs) Most frequent adverse events GI related: nausea and d
Slide 23: Interim Phase 2 Results Support Advancing to Pivotal Phase 3 CN Clinical Trial
2323 Interim Phase 2 Results Support Advancing to Pivotal Phase 3 CN Clinical Trial • Raised participants’ mean ANC above the lower limit of normal at Months 3 and 6 • Lowered potential infection risk by improving grade of neutropenia Mavorixafor monotherapy durably increased ANC in severe CN partic
Slide 24: Phase 3 CN Trial & Market Opportunity
24 Overview of Chronic Neutropenia Phase 3 CN Trial & Mark t Opportunity
Slide 25: 4WARD Pivotal, Global Phase 3 Trial in Most Common CN Indications
2525 4WARD Pivotal, Global Phase 3 Trial in Most Common CN Indications Participants now in screening Mavorixafor (50%) +/- G-CSF Placebo (50%) +/- G-CSF Global, Double-Blind, Placebo-Controlled Trial (same dosing as Phase 3 4WHIM trial) Screening Baseline Visit 1: 1 Randomization ANC assessments at
Slide 26: Data to Date Support 4WARD Phase 3 CN Trial Primary Endpoint
2626 Successful mavorixafor 4WHIM Phase 3 trial: • Mavorixafor significantly increased participants’ mean hours per day above ANC threshold of 500 cells/µL • ANC elevation >500 cells/ µL resulted in: • ~60% reduction in annualized rate of infection • 5 weeks shorter duration of infection • Fewer pat
Slide 27: Significant Opportunity to Address Unmet Needs in the CN Community
2727 Significant Opportunity to Address Unmet Needs in the CN Community X4 Market Research, July 2023 – data on file; ICD-19 Code Research (2017-2023). Current Use of G-CSF in ~15,000 U.S. CN Population with High Unmet Needs Mavorixafor to reduce / replace G-CSF Mavorixafor monotherapy Chronic G-CSF
Slide 28: Conclusions
28 Overview of Chronic Neutropenia Conclusions
Slide 29: Mavorixafor Holds Potential to Address Needs of CN Community
2929 Mavorixafor Holds Potential to Address Needs of CN Community Only approved option has significant and treatment- limiting side effects and risks1 CN patients continue to experience frequent / severe infections despite G-CSF use • Mavorixafor: potential for well tolerated, chronic, oral, once-da
Slide 30: X4 Well Positioned to Deliver on Promise of Mavorixafor
3030 X4 Well Positioned to Deliver on Promise of Mavorixafor Priority Review Voucher monetized U.S. approval & launch for WHIM syndrome April 2024 Laying a strong foundation for XOLREMDI U.S. sales & market growth Positive interim data from Phase 2 study support and de-risk Phase 3 CN trial Global,
Slide 31: Q&A Session
31 Q&A Session