X4 Pharmaceuticals, Inc. · NASDAQ:XFOR
X4 Pharmaceuticals — Q3 2024 Earnings Presentation
28 slides from X4 Pharmaceuticals's earnings presentation, Nov 13, 2024
Slide 1: 3Q 2024 Business Update & Phase 2 Chronic Neutropenia Study Results
3Q 2024 Business Update & Phase 2 Chronic Neutropenia Study Results November 13, 2024 Exhibit 99.2
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 conn
Slide 3: Today's Agenda
Today’s Agenda 3 Speakers Paula Ragan, PhD President & CEO 01 Welcome 02 XOLREMDI Launch & Phase 3 Chronic Neutropenia Clinical Trial Update 03 Phase 2 Chronic Neutropenia Study Results • Mavorixafor monotherapy • Mavorixafor + adjusted-dose G-CSF • Neutrophil functionality subgroup analysis • Safet
Slide 4: XOLREMDI Launch and Phase 3 Chronic Neutropenia Clinical Trial Update
Overview of Chronic Neutropenia XOLREMDI® Launch and Phase 3 Chronic Neutropenia Clinical Trial Update
Slide 5: U.S. Commercial Launch of XOLREMDI in WHIM Syndrome
U.S. Commercial Launch of XOLREMDI® in WHIM Syndrome1 • 50+ conferences attended since launch (national / regional / local ) • Physician peer-to-peer speaker program launched • Patient campaign initiated • Favorable reimbursement decisions and access: • Published policies represent >150 million cove
Slide 6: WHIM Experience Builds Strong Foundation in Chronic Neutropenia
WHIM Experience Builds Strong Foundation in Chronic Neutropenia (CN) • 67% of targeted WHIM hematologists would also be potential prescribers for CN, if approved in U.S. • X4 engaged with U.S. immunodeficiency and neutropenia patient advocacy groups that serve the WHIM and CN communities *Claims ana
Slide 7: 4WARD Phase 3 Trial On Track to Fully Enroll in Mid-2025
4WARD Phase 3 Trial On Track to Fully Enroll in Mid-2025 For more on 4WARD trial: NCT06056297 ~40% of planned sites now initiated; participants being dosed across multiple countries Recruitment, screening, and dosing ongoing • Expect majority of sites to be initiated in early 2025 12-Month, Global,
Slide 8: Phase 2 Chronic Neutropenia Study Results
Overview of Chronic Neutropenia Phase 2 Chronic Neutropenia Study Results
Slide 9: Risk of Serious, Recurrent Infections Correlates with Neutrophil Counts in CN
• 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 Classification2 Absolute Neutrophil Count (ANC) Severe (Grade 4) <500 cel
Slide 10: Mavorixafor Shown to Increase Circulating Neutrophils, Decrease Infections in WHIM Syndrome
Mavorixafor Shown to Increase Circulating Neutrophils, Decrease Infections in WHIM Syndrome 1. See full prescribing label at www.xolremdi.com; 2. Bainton DF (1980) The Cell Biology of Inflammation, vol 2, pp 1–25. Amsterdam: Elsevier/North-Holland. 10 Mavorixafor: Orally Active CXCR4 Antagonist Mavo
Slide 11: Unmet Needs in Chronic Neutropenia: Patients and Physicians Eager for Innovation
Unmet Needs in Chronic Neutropenia: Patients and Physicians Eager for Innovation 1. https://www.cancernetwork.com/view/fda-approves-new-indication-neupogen-chronic-neutropenia Only Currently Approved Therapy: Injectable Granulocyte Colony-Stimulating Factor (G-CSF) • Approved to treat severe chronic
Slide 12: Significant Opportunity to Address Unmet Needs in CN Community
Significant Opportunity to Address Unmet Needs in CN Community 1. X4 Market Research, July 2023 – data on file; ICD-10 Code Research (2017-2023). 12 Broad Opportunity for Mavorixafor: Monotherapy or in Combination with G-CSF High unmet needs in ~15,000 patients in the U.S.1 • Patients diagnosed with
Slide 13: Phase 2 Clinical Trial in Chronic Neutropenia: Goals and Design
Phase 2 Clinical Trial in Chronic Neutropenia: Goals and Design 1. The neutrophil life-cycle is 10-14 days (https://doi.org/10.3389/fimmu.2021.766620); Phase 2 study’s measurements of ANC over 6 months (at 30-day intervals) assess bone marrow status and durability of neutrophil production. 2. Modifi
Slide 14: Phase 2 Clinical Study in Chronic Neutropenia: Participant Disposition
Phase 2 Clinical Study in Chronic Neutropenia: Participant Disposition 2. Modifications to G-CSF dosing allowed after Month 2 visit 14 Study group representative of typical CN population Phase 2 Study Enrolled a Total of 23 Participants Participant Disposition (n=23) Type of CN Idiopathic 15 Congeni
Slide 15: Questions Addressed Today from Phase 2 CN Study Results
Questions Addressed Today from Phase 2 CN Study Results Phase 2 CN Study Population Key Questions Mavorixafor Monotherapy • Does mavorixafor monotherapy durably sustain ANC at clinically meaningful levels? Mavorixafor + Adjusted-Dose G-CSF • Are physicians and patients willing and able to adjust G-
Slide 16: Mavorixafor Monotherapy Durably and Meaningfully Increased Mean ANC
Mavorixafor Monotherapy Durably and Meaningfully 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 June
Slide 17: Mavorixafor Monotherapy Durably and Meaningfully Increased Mean ANC in Severe CN
Mavorixafor Monotherapy Durably and Meaningfully Increased Mean ANC in Severe CN 1. Platzbecker, U, et al. Blood. 2019 Mar;133(10):1020-1030. 2. Donadieu J, et al. Expert Rev Hematol. 2021 Oct;14(10):945-960. 3. Newburger PE, et al. Seminars in Hematology 2013 Jul;50(3):198-206. 4. Data set contains
Slide 18: Physicians Chose to Reduce G-CSF in 75% of Eligible Participants
Physicians Chose to Reduce G-CSF in 75% of Eligible Participants 1. One participant discontinued prior to M1 assessment; 2. Results from 3 participants maintained on stable dose G-CSF similar to data set presented on June 27, 2024 (data on file). Mavorixafor + G-CSF Combination Group Clinicians give
Slide 19: Physicians Substantially Reduced G-CSF, Maintaining Normal Mean ANC
Physicians Substantially Reduced G-CSF, Maintaining Normal Mean ANC Key Takeaways: G-CSF: • Given the option, physicians chose to substantially reduce injectable G-CSF therapy in 9 of 12 eligible patients • 89% (8 of the 9) had G-CSF adjusted at earliest possible timepoint (following Month 2 visit)
Slide 20: Neutrophil Functionality Assessed in Participants Enrolled in Phase 2 Sub-Study
Neutrophil Functionality Assessed in Participants Enrolled in Phase 2 Sub-Study 1. Ashley N. Connelly, et. al., Optimization of methods for the accurate characterization of whole blood neutrophils, Scientific Reports, 12:3667 (2022); 2. Ankur Gupta- Wright, et. al., Functional Analysis of Phagocyte
Slide 21: Neutrophils Functional in Healthy Donors and Pre-Treatment Phase 2 Participants
At baseline (pre-treatment): Mean percentage of functional neutrophils in study population were comparable to healthy volunteers Neutrophils Functional in Healthy Donors and Pre-Treatment Phase 2 Participants Notes: ROS results for all subjects demonstrate similar profiles to phagocytosis; Idiopathi
Slide 22: Neutrophil Functionality Maintained After Months of Mavorixafor Therapy
Neutrophil Functionality Maintained After 6 Months of Mavorixafor Therapy Meaningful increases in circulating functional neutrophils expected to reduce infection risk Mean percentage of functional neutrophils remained comparable to healthy donor controls after 6 months of treatment Mean percentage o
Slide 23: Phase 2 Chronic Neutropenia Study Safety Summary
Phase 2 Chronic Neutropenia Study Safety Summary 1. TEAE: treatment-emergent adverse event; 2. No further discontinuations once education on GI symptoms and support were implemented. Chronic mavorixafor generally well tolerated as monotherapy and in combination with G-CSF Combination (n=13), n (%) M
Slide 24: Phase 2 Results Support Mavorixafor Potential in CN and Raise Confidence in Phase 3
Phase 2 Results Support Mavorixafor Potential in CN and Raise Confidence in Success of Pivotal, Phase 3 Trial Mavorixafor generally well tolerated +/- G-CSF Meaningful increases in circulating functional neutrophils expected to reduce infection risk in CN Phase 3 population Phase 2 Findings Yes, mav
Slide 25: Physicians and Patients Eager for an Innovation like Mavorixafor
Physicians and Patients Eager for an Innovation like Mavorixafor Jolan Walter, MD, PhD ““Tapering off G- CSF could be a great alternative for many of our patients.” Jean Donadieu, MD, PhD “If you limit for this group of patients the number of injections of G- CSF … you will win.” “ “I believe that m
Slide 26: Conclusions and Look Ahead to 2025
Overview of Chronic Neutropenia Conclusions and Look Ahead to 2025
Slide 27: Continuing to Deliver Progress for Patients
Continuing to Deliver Progress for Patients >1,000 U.S. patients >15,000 U.S. patients Potential Market Opportunities +WHIM Chronic Neutropenia U.S. Launch of XOLREMDI ongoing Positive Phase 2 CN data support and derisk Phase 3 CN trial Global, pivotal 4WARD Phase 3 CN trial ongoing EU MAA WHIM subm
Slide 28: Q&A Session
Q&A Session