X4 Pharmaceuticals, Inc. · NASDAQ:XFOR
X4 Pharmaceuticals — Q3 2022 Earnings Presentation
18 slides from X4 Pharmaceuticals's earnings presentation, Nov 3, 2022
Slide 1: 3Q 2022 Earnings Call: The Road to 4WHIM Data and Beyond
3Q 2022 Earnings Call: The Road to 4WHIM Data and Beyond November 3, 2022
Slide 2: Forward-Looking Statements
Forward-Looking Statements This presentation contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. These statements may be identified by the words “may,” “will,” “could,” “would,” “should,” “expect,” “plan,” “anticipate,” “intend,
Slide 3: A Unique Investment Opportunity with Significant Near-Term Milestones
A Unique Investment Opportunity with Significant Near-Term Milestones Sharp focus on chronic Unparalleled expertise in neutropenic (CN) disorders, CXCR4 biology including WHIM syndrome Antagonizing the CXCR4/CXCL12 Bringing innovative treatments to axis proven to increase the ~50,000 estimated patie
Slide 4: X4 Focus on a Broad Range of Chronic Neutropenic Disorders
X4 Focus on a Broad Range of Chronic Neutropenic Disorders ~50,000 Estimated Chronic Neutropenia Patients in the U.S.1 Idiopathic Cyclic Congenital ~40,000 ~5,000 ~3,000 Most commonly diagnosed chronic Typically, a 21-day cycle Rare hematological genetic diseases neutropenia Autosomal-dominant disor
Slide 5: WHIM Syndrome: Immunodeficiency Disorder Associated with Chronic Neutropenia
WHIM Syndrome: Immunodeficiency Disorder Associated with Chronic Neutropenia Combined primary immunodeficiency affecting both children and adults arts. Driven by underlying HPV infection that can increase the risk W of HPV-related cancer Range of Assessments Help Establish a WHIM Diagnosis H ypogamm
Slide 6: Phase 3 Study: Results Expected 4Q 2022
Phase 3 Study: Results Expected 4Q 2022 Primary Endpoint Assessed 3 mos. 6 mos. 9 mos. 12 mos. Mavorixafor (Goal N = 9-14) Rollover to Established 1:1 Open-label Baseline Randomization Extension (Treatment) Placebo (Goal N = 9-14) • Enrollment: over-enrolled with 31 patients from 12 countries; LPLV
Slide 7: Phase 3 Study: Primary Endpoint and Key Assessments
Phase 3 Study: Primary Endpoint and Key Assessments Assessment of correcting Assessment of correcting Assessment of impact on neutropenia lymphopenia quality of life & other outcomes Primary Endpoint = TATANC Top Secondary Endpoint = TAT ALC Safety and Benefit (p value of treatment vs placebo) (p va
Slide 8: Positive WHIM Phase 2 Results Informed Phase 3 Design
Positive WHIM Phase 2 Results Informed Phase 3 Design Increases in TATANC seen in Phase 2 Increases in TATALC seen in Phase 2 TATANC/ALC ranges of 2-24 hours Used to power Phase 3 to >95% in All responded with >4x increase seen in patients on Phase 3 dose primary & top secondary endpoint in peak ANC
Slide 9: Phase 2 Results Informed Phase 3 Design: Clinical Impact
Phase 2 Results Informed Phase 3 Design: Clinical Impact Thumb of Affected WHIM Patient In Phase 2 study Overall positive Patient Global Impression of Change (PGIC)1 Improvement in warts Improvement in infections Pre-Treatment Following 55 weeks of treatment 1. Dale et al, Poster, ASH, 2021. 9
Slide 10: Breakthrough Therapy Designation (BTD): FDA Guidance & Engagement
Breakthrough Therapy Designation (BTD): FDA Guidance & Engagement • Multiple FDA interactions over several years • FDA input: • Informed on study design, endpoints, analyses • Recommended “composite endpoint” for infection burden • Recognition of the rarity, heterogeneity of WHIM syndrome 10
Slide 11: Mavorixafor: the First Potential Treatment for WHIM Syndrome
Mavorixafor: the First Potential Treatment for WHIM Syndrome Diverse Efforts to Support Physician Education & Diagnosis Medical Education (MSLs) Clinical Publications, Conferences, Symposium Sponsorships Existing neutropenia registries (NNN, SCNIR) Patient Advocacy Global WHIM registry in developmen
Slide 12: Timeline and Commercial Strategy
Timeline and Commercial Strategy EU Regulatory and Territories Outside of US / Initial Focus on the U.S. Commercial Launch Key European countries 4Q 2022 2H 2023 1H 2024 2024 2024 Phase 3 Data Expected NDA submission Expected U.S. launch EMA filing ~12 Months Partnership in 4Q 2022 early 2H 2023 in
Slide 13: Commercial Leadership – Introduction to Mark Baldry, Chief Commercial Officer
Commercial Leadership – Introduction to Mark Baldry, Chief Commercial Officer • 30+ years’ experience in global life science commercial strategy and operations • Particularly skilled in: • Building high-performing commercial teams • Developing effective global strategies • Launching new products int
Slide 14: Leveraging Significant Rare Disease Expertise for Potential 2024 U.S. Launch
Leveraging Significant Rare Disease Expertise for Potential 2024 U.S. Launch Physician & Patient Education/Engagement Building the Commercial Team U.S. and EU Payer Engagement Patients Marketing/Branding Manufacturing & Distribution (U.S.) 14
Slide 15: WHIM Efforts Align Well with Potential Expansion into CN Disorders
WHIM Efforts Align Well with Potential Expansion into CN Disorders WHIM syndrome and CN physicians and researchers significantly overlap • Non-malignant hematology specialists • Immunologists Path to Patient foundations provide support to all CN Launch • National Neutropenia Network (NNN) + Educatio
Slide 16: Mavorixafor: Data Support Further Development across Broad CN Disorders
Mavorixafor: Data Support Further Development across Broad CN Disorders Potential to be Only Oral Therapy to Treat Chronic Neutropenia Demonstrated Δ ANC >500 cells/µL WHIM Syndrome Congenital Cyclic Idiopathic ~50,000 diagnosed U.S. patients 16
Slide 17: Significant Near-Term Milestones / Meaningful Growth Potential
Significant Near-Term Milestones / Meaningful Growth Potential Q3 2022 Q4 2022 1H 2023 2H 2023 Phase 1b 4WHIM Phase 3 Initial CN Regulatory CN Results Trial Readout Path Clarity 4Q 2022 Additional CN Data WHIM NDA Submission Expected: Advancing mavorixafor • Durability & ANC data into Phase 1b/Pha
Slide 18: Q&A Session / Conclusion
PHARMACEUTICALS Q&A Session / Conclusion