X4 Pharmaceuticals, Inc. · NASDAQ:XFOR
X4 Pharmaceuticals — Investor Presentation (May 2024)
26 slides from X4 Pharmaceuticals's investor presentation, May 22, 2024
Slide 1: Enabling a better future for people with rare immune disorders
Enabling a better future for people with rare immune disorders PROGRESS PATIENTS May 2024 Exhibit 99.1
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 session and any documents or materials distributed at or in con
Slide 3: X4 Pharmaceuticals Overview
3 X4 Pharmaceuticals Overview 1. WHIM (Warts, Hypogammaglobulinemia, Infections, Myelokathexis); 2. Current funds include $82 million in cash and equivalents as of March 31, 2024 + $105 million in proceeds from PRV sale (May 2024) + $20 million from debt draw down from loan facility with Hercules Ca
Slide 4: Mavorixafor: Pipeline in a Product via CXCR4 Antagonism
4 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 5: Advancing Mavorixafor in Chronic Neutropenic Disorders and WHIM Syndrome
5 Advancing Mavorixafor in Chronic Neutropenic Disorders and WHIM Syndrome Indication Preclinical Phase 1 Phase 2 Phase 3 FDA Approved XOLREMDITM (mavorixafor) WHIM Syndrome (Warts, Hypogammaglobulinemia, Infections and Myelokathexis) U.S. launch underway EU submission in late 2024 / early 2025 Mavo
Slide 6: WHIM Syndrome: a Combined Primary Immunodeficiency and Chronic Neutropenic Disorder
6 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 7: Until Now, WHIM Syndrome Managed with Treatments Not Addressing Underlying Cause
7 Until Now, WHIM Syndrome Managed with Treatments Not Addressing Underlying Cause References: 1. Dale DC, Firkin F, Bolyard AA, et al, Blood. 2020;136(26):2994-3003; 2. Geier CB, Ellison M, Cruz R, et al, J Clin Immunol. 2022;42(8):1748-1765; 3. Kiss C, Connoley D, Connelly K, et al, Antibiotics, 2
Slide 8: Now FDA approved! XOLREMDI
Now FDA approved! (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 Count 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: Improvements Across Infection Assessments over 52 Weeks
11 4WHIM: Patients Treated with XOLREMDI Experienced Improvements Across Infection Assessments over 52 Weeks versus Placebo1 ~60% reduction in annualized infection rate 1. Badolato R, et al. Blood. Published online April 21, 2024;blood.2023022658. Fewer patients with severe infection On average, inf
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: Commercial Strategy Overview: Targeted Education, Engagement, and Access
13 Commercial Strategy Overview: Targeted Education, Engagement, and Access ➢ Educate on WHIM syndrome ➢ Provide diagnostic support ➢ Engage at key medical conferences ➢ Target key hematologists & immunologists ➢ Communicate targeted MOA and clinical profile ➢ Drive adoption and uptake in appropriat
Slide 14: Targeted Approach to Covering the U.S. WHIM Market
14 Targeted Approach to Covering the U.S. WHIM Market Refined Target List of ~3,500 HCPs (primarily immunologists and hematologists) Range: 250 to 300 Range: 150 to 200 Range: 50 to 100 Range: 25 to 50 Range: 10 to 25 Range: 1 to 10 • Focused engagement with ~20 top thought leaders • Partnering with
Slide 15: XOLREMDI Addressing High Unmet Need with Targeted Innovation
15 XOLREMDI Addressing High Unmet Need with Targeted Innovation Annual Price* Reflects Value • Patients >50 kg = 400 mg daily = $496,400 annually • Patients ≤50 kg = 300 mg daily = $372,300 annually Potential to address high burden of disease and strengthen relevant aspects of patients’ immune funct
Slide 16: Chronic Neutropenia: No Innovation in More Than 30 Years
16 Injectable Granulocyte Colony-Stimulating Factor (G-CSF) • Inconvenient daily injections • Frequent treatment-related/treatment-limiting bone pain and other adverse events Chronic Neutropenia: No Innovation in More Than 30 Years 1Only One ~50,0001 U.S. Prevalence: total diagnosed with Chronic Neu
Slide 17: Chronic neutropenic disorders: infection risk by severity
17 1. 2. 1. https://ctep.cancer.gov/protocoldevelopment/electronic_applications/docs/ctcae_v5_quick_reference_8.5x11.pdf ; 2. Jan Palmblad, Carlo Dufour, Helen A. Papadaki, Haematologica, Vol. 99 No. 7 (2014): July, 2014; 3. Sicre de Fontbrune F, et al. Blood. 2015;126(14):1643-1650; 4.Bodey, GP, et
Slide 18: What Makes a Difference to Chronic Neutropenia Patients and Their Physicians?
18 Expanded treatment options, ideally: • Reduced infection rates • Oral formulation • Good safety profile • Alternate therapy to injectable G-CSF and/or • Reduced G-CSF-dose & related toxicities What Makes a Difference to Chronic Neutropenia Patients and Their Physicians? “There is a major diagnost
Slide 19: Assessing Mavorixafor in Six-Month CN Phase 2 Clinical Trial
19 Mavorixafor: Same Oral Dosing as WHIM Phase 3 Baseline Pre-Dose Phase 2 Trial: Safety, Durability of ANC Levels over 6-Month Period Hematologic Assessments including Mean ANC Once Daily Mavorixafor: Monotherapy or with G-CSF1 DAY -1 MONTH 6 1. Modifications in G-CSF allowed after month 2 at docto
Slide 20: Mavorixafor: Potential to Deliver First Innovation in CN in 30+ Years
20 1. All subjects increased absolute neutrophil count (ANC) over first 3 months of dosing (when G-CSF maintained at fixed, baseline dose) Mavorixafor: Potential to Deliver First Innovation in CN in 30+ Years CN Phase 1b Results • ANC increased by >500 cells/µL in 100% (n=25) after single dose Early
Slide 21: CN Clinical Data to Date Support Advancing Mavorixafor into Phase 3 Trial
21 CN Clinical Data to Date Support Advancing Mavorixafor into Phase 3 Trial ✓ First supporting evidence that mavorixafor treatment durably increases ANC ✓ Levels of observed ANC increase ( ANC ≥ 500) correlate with infection risk reduction ✓ Safety profile to date supports chronic treatment with m
Slide 22: CN Pivotal, Global Phase 3 Trial Expected to Initiate in 2Q 2024
22 CN Pivotal, Global Phase 3 Trial Expected to Initiate in 2Q 2024 Key Inclusion Criteria: • Diagnosis: congenital, autoimmune, or idiopathic neutropenia • Absolute Neutrophil Count (ANC): <1500 cells/µL • Infection history: 2 infections requiring intervention within last 12 months Design: double-b
Slide 23: Continuing to Deliver Progress for Patients
23 Continuing to Deliver Progress for Patients Priority Review Voucher monetized U.S. Approval & Launch for WHIM syndrome April 2024 Laying a strong foundation for XOLREMDI U.S. sales & market growth Additional Phase 2 CN trial data expected in late June 2024 Initiation of pivotal Phase 3 CN trial a
Slide 24: 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 25: Seasoned Executive Leadership Team
25 Seasoned Executive Leadership Team Experienced in research, development, & commercialization of first-in-class, innovative therapies c ART TAVERAS, Ph.D. Chief Scientific Officer c ADAM MOSTAFA Chief Financial Officer c PAULA RAGAN, Ph.D. President & CEO c MARY DIBIASE, Ph.D. Chief Operating Offi
Slide 26: Strong Balance Sheet Supports Expected Upcoming Milestones
26 Strong Balance Sheet Supports Expected Upcoming Milestones 1. Current funds include $82 million in cash and equivalents as of March 31, 2024 + $105 million in proceeds from PRV sale (May 2024) + $20 million from debt draw down from loan facility with Hercules Capital, Inc. (May 2024); 2. Projecte
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