X4 Pharmaceuticals, Inc. · NASDAQ:XFOR
X4 Pharmaceuticals — Corporate Update (April 2024)
26 slides from X4 Pharmaceuticals's corporate update, Apr 29, 2024
Slide 1: Investor Call
Investor Call April 29, 2024 PROGRESS PATIENTS
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 session and any document or material distributed at or in connect
Slide 3: Welcome and Overview; Label Highlights; Commercial Launch Strategy
Welcome and Overview Paula Ragan, PhD, President & Chief Executive Officer Label Highlights and Supporting Clinical Data Christophe Arbet-Engels, MD, PhD, Chief Medical Officer Commercial Launch Strategy & Execution Mark Baldry, Chief Commercial Officer Conclusion & Q&A
Slide 4: X4: delivering innovation for people with rare immune disorders
X4: delivering innovation for people with rare immune disorders 2014 2022 2023 2024 X4 Founded to advance X4 announces positive top-line FDA accepts NDA for FDA approves XOLREMDITM CXCR4-targeted results from pivotal, global, mavorixafor in WHIM (mavorixafor), the first and only therapeutics for peo
Slide 5: Now FDA approved!
Now FDA approved! (zōl-RĔM-dee)
Slide 6: WHIM syndrome is a combined primary immunodeficiency and a chronic neutropenic disorder
WHIM syndrome is a combined primary immunodeficiency and a chronic neutropenic disorder1 Heterogeneous presentation2 Most frequently characterized by: Lifelong impact2 Chronic, congenital disorder Neutropenia Hypogammaglobulinemia Commonly presents in childhood, with median age (98%) (65%) of diagno
Slide 7: The underlying cause of WHIM syndrome, CXCR4 pathway dysregulation, impairs movement of leukocytes
The underlying cause of WHIM syndrome, CXCR4 pathway dysregulation, impairs movement of leukocytes critical to a healthy immune response1 In WHIM syndrome, CXCR4 variants can result in decreased mobilization of white blood cells into peripheral circulation Lymphocyte Immunodeficiency in WHIM syndrom
Slide 8: A selective, oral CXCR4 antagonist that targets the underlying cause of WHIM syndrome
A selective, oral CXCR4 antagonist that targets the underlying cause of WHIM syndrome XOLREMDI inhibits binding of the CXCL12 ligand to the CXCR4 receptor, inhibiting its downstream activation. XOLREMDI increases mobilization of neutrophils and lymphocytes into the peripheral blood Reference: XOLREM
Slide 9: Label Highlights & Supporting Clinical Data
Label Highlights & Supporting Clinical Data Christophe Arbet-Engels, MD, PhD Chief Medical Officer
Slide 10: Until now, WHIM syndrome has been managed with treatments that do not address its underlying cause
Until now, WHIM syndrome has been managed with treatments that do not address its underlying cause Symptomatic Treatments G-CSF IVIg Antibiotics & Antivirals • Not specifically indicated for WHIM syndrome • No adequate and well controlled trials evaluating safety and efficacy in patients with WHIM s
Slide 11: XOLREMDI (mavorixafor) U.S. Label highlights
XOLREMDITM (mavorixafor) U.S. Label highlights XOLREMDI is approved for use in patients 12 years of age and older with WHIM syndrome (warts, Indication Statement hypogammaglobulinemia, infections and myelokathexis) to increase the number of circulating mature neutrophils and lymphocytes. Contraindic
Slide 12: 4WHIM was the largest Phase 3 clinical trial to date in WHIM syndrome
4WHIM was 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 Assessed Study Population XOLREMDI (N= 14) ~90% • Adults and adolescents (1
Slide 13: 4WHIM Results: XOLREMDI significantly increased time above key immune cell count thresholds
4WHIM Results: XOLREMDI significantly increased the time patients stayed above key immune cell count thresholds over 52 weeks Primary endpoint Key secondary endpoint Significantly increased mean hours per day Significantly increased mean hours per above the threshold for neutrophils day above the th
Slide 14: 4WHIM Results: improvements across infection assessments over 52 weeks
4WHIM Results: patients treated with XOLREMDI experienced improvements across infection assessments over 52 weeks versus placebo1 Total infection score 40% lower for patients on XOLREMDI versus those on placebo ~60% reduction in On average, infection duration Fewer patients with annualized infection
Slide 15: Treatment was generally well tolerated; majority of adverse reactions mild to moderate
Treatment was generally well tolerated; majority of adverse reactions were mild to moderate in severity Adverse Reactions Section of Product Label1 (≥10% and at a frequency higher than placebo in 4WHIM) XOLREMDI Placebo Published Phase 3 trial data results2 Adverse Reaction showed: (n=14) (n=17) Thr
Slide 16: Commercial Launch Strategy & Execution
Commercial Launch Strategy & Execution Mark Baldry, Chief Commercial Officer
Slide 17: Commercial Strategy Overview: targeted education, engagement, and access
Commercial Strategy Overview: targeted education, engagement, and access Establish XOLREMDI as Support Patient Diagnosis Gain Broad Access Standard of Care in WHIM syndrome ➢ Educate on WHIM syndrome ➢ Target key hematologists & ➢ Mitigate access barriers ➢ Provide diagnostic support immunologists ➢
Slide 18: The right field team to launch an innovative rare disease medicine
The right field team to launch an innovative rare disease medicine Field team recruited from well known rare and ultra- rare organizations Team Expertise • Rare disease space / hematology & immunology Collectively more than • Supporting diagnosis of rare diseases 250 years of demonstrated • Understa
Slide 19: Targeted approach to covering the U.S. WHIM market
Targeted approach to covering the U.S. WHIM market Refined Target List of ~3,500 HCPs Focused KOL Engagement (primarily immunologists and hematologists) ~20 top thought leaders Range: 250 to 300 Range: 150 to 200 Partnering with Range: 50 to 100 Patient Advocacy Networks Range: 25 to 50 Jeffrey Mode
Slide 20: HCPs are seeking a targeted therapy for WHIM Syndrome
HCPs are seeking a targeted therapy for WHIM Syndrome, a serious condition with high burden and unmet need HCP Survey Shown an unbranded profile of XOLREMDI, interest in prescribing was high across HCPs of HCPs believe WHIM syndrome is a serious in all key specialties, receiving an average 92% immun
Slide 21: Patient Support
Patient Support X4ConnectTM helps eligible patients navigate insurance coverage, prior authorization requirements, and financial assistance programs Specialty Pharmacy to help support access Processes and fills XOLREMDI prescriptions with clinical pharmacists X4Connect Care Coordinators • Investigat
Slide 22: XOLREMDI addressing high unmet need with targeted innovation
XOLREMDI addressing high unmet need with targeted innovation Annual Price* Reflects Value Targeted Breakthrough therapy for ultra-rare patient population • Patients >50 kg = 400 mg daily = $496,400 annually • Patients ≤50 kg = 300 mg daily = $372,300 annually First and only FDA-approved therapy indi
Slide 23: Delivering on the promise of XOLREMDI (mavorixafor)
Delivering on the promise of XOLREMDI (mavorixafor) Establishing XOLREMDI as Supporting Patient Diagnosis Gaining Broad Access Standard of Care in WHIM syndrome 23
Slide 24: Conclusion
[design: new divider slide please] Conclusion Paula Ragan, PhD President & Chief Executive Officer
Slide 25: X4: continuing to deliver progress for patients
X4: continuing to deliver progress for patients U.S. Approval & Laying a strong Additional Initiation of pivotal Potential Launch of foundation for Phase 2 CN trial Phase 3 CN trial pipeline XOLREMDI for XOLREMDI data expected anticipated in expansion WHIM syndrome sales & market in 1H 2024 1H 2024
Slide 26: Courtney, living with WHIM syndrome
Courtney, living with WHIM syndrome