X4 Pharmaceuticals, Inc. · NASDAQ:XFOR
X4 Pharmaceuticals — Corporate Update (November 2022)
19 slides from X4 Pharmaceuticals's corporate update, Nov 29, 2022
Slide 1: Positive 4WHIM Phase 3 Top-Line Results Conference Call & Webcast
Positive 4WHIM Phase 3 Top-Line Results Conference Call & Webcast November 29, 2022 EXHIBIT 99.2
Slide 2: Forward-Looking Statements
2 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,” “inten
Slide 3: On Today's Call
3 On Today’s Call PAULA RAGAN, Ph.D. President & CEO ART TAVERAS, Ph.D. Chief Scientific Officer MARY DIBIASE, Ph.D. Chief Operating Officer ADAM MOSTAFA Chief Financial Officer MURRAY STEWART, DM, FRCP Interim Chief Medical Officer MARK BALDRY Chief Commercial Officer
Slide 4: Mavorixafor – the First Potential Treatment for WHIM Syndrome
4 Mavorixafor – the First Potential Treatment for WHIM Syndrome 15.04 hours (P<0.0001) Mean time of absolute neutrophil count above threshold1 (TAT- ANC) over a 24-hour period, over 52 weeks, treatment vs. placebo (2.75 hours) 1. Threshold = 500 cells/mL; ANC levels below 500 are considered severe n
Slide 5: WHIM Syndrome: Congenital Immunodeficiency Associated with Chronic Neutropenia
5 WHIM Syndrome: Congenital Immunodeficiency Associated with Chronic Neutropenia Primary Clinical Assessments • Neutropenia & lymphopenia (low ANC and ALC) • Repeat infections with long-term effects • In some: wart lesions; cervical test for HPV • In some: Low immunoglobulin (Ig) levels Additional A
Slide 6: CXCR4: Key Role in Maturation & Mobilization of Neutrophils and Lymphocytes
6 CXCR4: Key Role in Maturation & Mobilization of Neutrophils and Lymphocytes Mavorixafor is a CXCR4 Antagonist White Blood Cells (WBCs) are retained in the bone marrow by the CXCL12/CXCR4 axis creating a “reserve” • Bone marrow produces WBCs, including neutrophils and lymphocytes • Creates reserves
Slide 7: 4WHIM Phase 3 Study Design
7 4WHIM Phase 3 Study Design Top-Line Assessments (Today) • Mean TATANC - mean of the 13, 26, 39, and 52-week assessments • Mean TATALC - mean of the 13, 26, 39, and 52-week assessments • Safety and tolerability across 52 weeks >90% of patients have continued in open label extension (OLE) Additional
Slide 8: Demographics & Screening Metrics
8 Demographics & Screening Metrics Mavorixafor (N=14) Placebo (N=17) Adolescents (12 to <18 years) n (%) 7 (50) 8 (47) Adults (≥18 years) n (%) 7 (50) 9 (53) Female Gender n (%) 9 (64) 9 (53) Previous Immunoglobulin Usage n (%) 6 (43) 8 (47) Screening ANC (cells/mL) n mean (SD) median (min, max) 14
Slide 9: Phase 3 Primary Endpoint (TATANC) Met
9 Phase 3 Primary Endpoint (TATANC) Met L S m e a n T A T A N C (9 5 % C I) (h o u rs ) • Mavorixafor significantly improved the time above the threshold of ANC vs. placebo in intent-to-treat (ITT) population • Mean TATANC was 15.04 hours for mavorixafor vs. 2.75 hours for placebo • 5.5-fold improve
Slide 10: TATANC Increased and Maintained Over 52 Weeks vs. Placebo and Baseline
10 TATANC was increased and maintained over 52 weeks vs. placebo and vs. baseline TATANC vs. Time on Treatment: Mavorixafor Durably Increased TATANC Over 52 Weeks (ITT population) 1. At week 52, 3 of 17 placebo patients were given mavorixafor in advance of their TAT measurements as they entered the
Slide 11: Top Key Secondary Endpoint (TATALC) Met
11 Top Key Secondary Endpoint (TATALC) Met • Mavorixafor significantly improved the time above the threshold of ALC vs. placebo in ITT population • Mean TATALC of 15.80 hours for mavorixafor vs. 4.55 hours for placebo • 3.5-fold improvement in TATALC compared with placebo P<0.0001 L S M e a n T A T
Slide 12: TATALC vs. Time on Treatment: Durable Increase Over 52 Weeks
12 TATALC vs. Time on Treatment: Mavorixafor Durably Increased TATALC Over 52 Weeks (ITT population) 1. At week 52, 3 of 17 placebo patients were given mavorixafor in advance of their TAT measurements as they entered the open-label portion of the study. All data are included in ITT analysis. L S M e
Slide 13: Top-Line Safety Data Summary for Randomization Period
13 Top-Line Safety Data Summary for Randomization Period Mavorixafor (N=14) Placebo (N=17) n (%) n (%) Summary Any TEAE 14 (100) 17 (100) Treatment-related TEAE 7 (50) 3 (18) Any Serious AE 5 (36) 2 (12) Treatment-related Serious AE 0 0 Discontinuations due to AE 0 0 Treatment-limiting toxicity 0 0
Slide 14: Summary: 4WHIM Results
14 Summary: 4WHIM Results Met primary endpoint (P<0.0001) of TATANC – clinically meaningful correction of severe chronic neutropenia Durability of TATANC response shown over 52 weeks of treatment Met first secondary endpoint (P<0.0001) of TATALC - clinically meaningful correction of lymphopenia Dura
Slide 15: Next Steps/Expected Milestones to Advance Mavorixafor for WHIM
15 Achieved primary endpoint and key secondary endpoint in 4WHIM Pre-NDA meeting with U.S. FDA Present additional 4WHIM trial results U.S. New Drug Application (NDA) submission for mavorixafor in WHIM syndrome Publish complete 4WHIM trial results Continue with launch preparedness Possible approval1,
Slide 16: Thank You!
16 THANK YOU! Study participants and their families Clinicians, healthcare providers & study sites X4 employees
Slide 17: Successful WHIM Phase 3 Supports Plan to Initiate CN Phase 3
17 Successful WHIM Phase 3 Supports Plan to Initiate CN Phase 3 ✓Primary Endpoint (TATANC) = reduction of severe neutropenia ✓ Placebo-controlled design ✓Favorable tolerability profile over 52 weeks of dosing ✓Durable response WHIM Phase 3 Phase 1/2 CN Trial CN Disorders Phase 3
Slide 18: Significant Near-Term Milestones Expected / Meaningful Growth Potential
18 Significant Near-Term Milestones Expected / Meaningful Growth Potential Phase 1b CN Trial Data 4WHIM Phase 3 Topline Data Initial CN Regulatory Path Clarity Additional CN Data Expected: • Durability & ANC data • Explore G-CSF taper • Long term safety data WHIM NDA Submission Advancing Towards CN
Slide 19: Q&A
Q&A
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