X4 Pharmaceuticals, Inc. · NASDAQ:XFOR
X4 Pharmaceuticals — Investor Presentation (January 2025)
32 slides from X4 Pharmaceuticals's investor presentation, Jan 13, 2025
Slide 1: Enabling a better future for people with rare immune disorders
Enabling a better future for people with rare immune disorders PROGRESS PATIENTS January 2025 Exhibit 99.2
Slide 2: Forward-Looking Statements
2 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 co
Slide 3: X4's Momentum Addressing Unmet Needs in Rare Immune Disorders
3 X4’s Momentum Addressing Unmet Needs in Rare Immune Disorders 1. WHIM (Warts, Hypogammaglobulinemia, Infections, Myelokathexis); 2. Projected runway excludes any potential U.S. sales of XOLREMDI. Fully integrated company delivering on the promise of mavorixafor XOLREMDI® (mavorixafor) approved by
Slide 4: X4 and Norgine Enter into Exclusive Licensing Agreement for Mavorixafor
4 X4 and Norgine Enter into Exclusive Licensing Agreement to Commercialize Mavorixafor in Europe, Australia, and New Zealand – January 2025 Maximizing the global potential of mavorixafor through strategic partnership • Leverages Norgine’s existing infrastructure and successful track record in commer
Slide 5: Mavorixafor: Pipeline in a Product via CXCR4 Antagonism
5 Mavorixafor: Pipeline in a Product via CXCR4 Antagonism Validated mechanism shown to alleviate neutropenia and lymphopenia Targeted Mechanism • CXCR4 regulates movement of white blood cells throughout the body2 • CXCR4 antagonism has been shown to increase the migration of cells from the bone marr
Slide 6: Advancing Innovation for Patients
6 Advancing Innovation for Patients Indication Pre- clinical Phase 1 Phase 2 Phase 3 FDA Approved WHIM Syndrome (Warts, Hypogammaglobulinemia, Infections, Myelokathexis) Progress on U.S. commercialization EU MAA submission by early 2025 Mavorixafor Chronic Neutropenia (Congenital, Autoimmune, Idiopa
Slide 7: WHIM Syndrome: a Combined Primary Immunodeficiency and CN Disorder
7 Lifelong impact2 Chronic, congenital disorder Commonly presents in childhood, with median age of diagnosis of 5.5 years of age Lower life expectancy vs. the general population3,4 due to sepsis, irreversible organ damage, recurrent pneumonia, and certain cancers WHIM Syndrome: a Combined Primary Im
Slide 8: U.S. Launch in May 2024
U.S. Launch in May 2024 (zōl-RĔM-dee) For use in patients 12 years of age and older with WHIM syndrome (warts, hypogammaglobulinemia, infections and myelokathexis) to increase the number of circulating mature neutrophils and lymphocytes. See full prescribing information at xolremdi.com
Slide 9: 4WHIM: the Largest Phase 3 Clinical Trial to Date in WHIM Syndrome
9 4WHIM: the Largest Phase 3 Clinical Trial to Date in WHIM Syndrome XOLREMDI was studied in a global, randomized, double-blind, placebo-controlled, Phase 3 trial conducted in 31 patients with WHIM syndrome Primary endpoint • Improvement in absolute neutrophil count (ANC) as measured by the mean tim
Slide 10: 4WHIM: XOLREMDI Significantly Increased Time Above Key Immune Cell Thresholds
10 4WHIM: XOLREMDI Significantly Increased Time Patients Stayed Above Key Immune Cell Count Thresholds over 52 Weeks versus Placebo Primary endpoint Significantly increased mean hours per day above the threshold for neutrophils Key secondary endpoint Significantly increased mean hours per day above
Slide 11: 4WHIM: ANC Increase Resulted in Clinical Infection Benefits
11 1. Badolato R, et al. Blood (2024) 144 (1): 35–45. 2. Badolato R. et al. Oral Presentation at Annual Meeting of the Clinical Immunology Society, May 2023. 3.Total infection score calculated by summing the number of infection events weighted by severity and divided by the total exposure time (in y
Slide 12: 4WHIM: Treatment Generally Well Tolerated
12 4WHIM: Treatment Generally Well Tolerated; Majority of Adverse Reactions Mild to Moderate in Severity Adverse Reactions Section of Product Label1 (≥10% and at a frequency higher than placebo in 4WHIM) 1. XOLREMDI package insert. Please see Important Safety Information and full Prescribing Informa
Slide 13: Addressing High Unmet Need with Targeted Innovation
13 Addressing High Unmet Need with Targeted Innovation Potential to address high burden of disease and strengthen patients’ immune function Demonstrated efficacy & safety profile with oral formulation Targets the underlying cause of WHIM syndrome via CXCR4 antagonism First and only FDA-approved ther
Slide 14: XOLREMDI U.S. Launch Update – November 2024
14 XOLREMDI® U.S. Launch Update – November 2024 1. HCPs (healthcare practitioners) reached through in-person and digital engagement; 2. X4 Market Research: HCP Tracking Study of immunologists and hematologists – Pre-launch (Mar 2024), Post- launch (October 2024) • 50+ conferences attended since laun
Slide 15: WHIM Experience Builds Strong Foundation in Chronic Neutropenia
15 WHIM Experience Builds Strong Foundation in Chronic Neutropenia (CN) *Claims analysis, Oct 2024; XOLREMDI (mavorixafor) is being exclusively promoted in the U.S. for its approved indication of WHIM syndrome; mavorixafor is currently being studied as an investigational therapy for use in chronic n
Slide 16: Chronic Neutropenia: No Innovation in More Than 30 Years
16 Injectable Granulocyte Colony-Stimulating Factor (G-CSF) • Approved to treat severe chronic neutropenia in 19952 • Used as a chronic daily injection or as rescue during serious infection episodes • Frequent treatment-related / treatment-limiting bone pain other adverse events, and long-term risk
Slide 17: Risk of Serious, Recurrent Infections Correlates with Neutrophil Counts in CN
17 • 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
Slide 18: Unmet Needs in Chronic Neutropenia: Patients and Physicians Eager for Innovation
18 Unmet Needs in Chronic Neutropenia: Patients and Physicians Eager for Innovation 1. https://www.cancernetwork.com/view/fda-approves-new-indication-neupogen-chronic-neutropenia Jolan Walter, MD, PhD ““The administration [of G-CSF] is painful and also can have long-term consequences.” Jean Donadieu
Slide 19: Significant Opportunity to Address Unmet Needs in CN Community
19 Significant Opportunity to Address Unmet Needs in CN Community 1. X4 Market Research, July 2023 – data on file; ICD-10 Code Research (2017-2023). 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 20: Successful Phase 2 Study of Mavorixafor in Chronic Neutropenia
20 Successful Phase 2 Study of Mavorixafor in Chronic Neutropenia 1. The neutrophil life-cycle is 10-14 days (https://doi.org/10.3389/fimmu.2021.766620); Phase 2 ANC measurements over 6 months assess bone marrow status and durability of neutrophil production. 2. ANC assessments (6 blood draws over 8
Slide 21: Mavorixafor Monotherapy Durably and Meaningfully Increased Mean ANC
21 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 6
Slide 22: Mavorixafor Monotherapy Increased Mean ANC in Severe CN
22 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 conta
Slide 23: Physicians Substantially Reduced G-CSF, Maintaining Normal Mean ANC
23 Physicians Substantially Reduced G-CSF, Maintaining Normal Mean ANC G-CSF: • Given the option, physicians chose to substantially reduce injectable G-CSF therapy in 9 of 12 (75%) eligible1 patients • 89% (8 of the 9) had G-CSF adjusted at earliest possible timepoint (following Month 2 visit) • 33%
Slide 24: Neutrophil Functionality Assessed in Phase 2 Sub-Study
24 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 Phagocyt
Slide 25: Neutrophil Functionality Comparable to Healthy Donors Pre- and Post-Mavorixafor
25 Neutrophil Functionality Comparable to Heathy Donors Pre- and Post-Mavorixafor Mean percentage of functional neutrophils remained comparable to healthy donor controls prior to and following 6 months of mavorixafor treatment Notes: Samples assessed for neutrophil functionality were limited by prox
Slide 26: Phase 2 Chronic Neutropenia Study Safety Summary
26 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 (%
Slide 27: Phase 2 Results Support Mavorixafor Potential in CN
27 Phase 2 Results Support Mavorixafor Potential in CN and Raise Confidence in Success of Ongoing Phase 3 4WARD Trial Meaningful increases in circulating functional neutrophils expected to reduce infection risk in CN Phase 3 population Phase 2 Findings Yes, mavorixafor durably and meaningfully incre
Slide 28: 4WARD Phase 3 Trial On Track to Fully Enroll in Mid-2025
28 4WARD Phase 3 Trial On Track to Fully Enroll in Mid-2025 – November 2024 Update 1. Discussions ongoing with U.S. FDA on finalization of ANC response endpoint. For more on the 4WARD trial: NCT06056297 ~40% of planned sites now initiated; participants being dosed across multiple countries Recruitme
Slide 29: Continuing to Deliver Progress for Patients
29 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 Global, pivotal 4WARD Phase 3 CN trial initiated Positive Phase 2 CN data derisk ongoing 4WARD trial Ex-U.S. partner secu
Slide 30: U.S. Headquarters North Beacon Street, 4th Floor Boston, MA 02134 Research Center of Excellence
NASDAQ: XFOR U.S. Headquarters 61 North Beacon Street, 4th Floor Boston, MA 02134 Research Center of Excellence Helmut-Qualtinger-Gasse 2 A-1030 Vienna, Austria www.x4pharma.com
Slide 31: Seasoned Executive Leadership Team
31 Seasoned Executive Leadership Team Experienced in research, development, & commercialization of first-in-class, innovative therapies c ADAM MOSTAFA Chief Financial Officer c PAULA RAGAN, Ph.D. President & CEO c MARY DIBIASE, Ph.D. Chief Operating Officer c MARK BALDRY Chief Commercial Officer cCH
Slide 32: Balance Sheet Supports Expected Upcoming Milestones
32 Balance Sheet Supports Expected Upcoming Milestones 1. Funds as of September 30, 2024; 2. Projected runway excludes any potential U.S. sales of XOLREMDI; 3. Exclusive licensing and supply agreement completed with Norgine to commercialize mavorixafor in Europe, Australia, and New Zealand (announce
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