X4 Pharmaceuticals, Inc. · NASDAQ:XFOR
X4 Pharmaceuticals — Investor Presentation (April 2019)
20 slides from X4 Pharmaceuticals's investor presentation, Apr 1, 2019
Slide 1: Rare Diseases Innovate - April 2019
April 2019 RARE DISEASES RARE DISEASES INNOVATE 4 Exhibit 99.1
Slide 2: Forward Looking Statements
Forward Looking Statements Certain statements in this presentation, particularly statements relating to: our plans for, or progress, scope, cost, duration or results or timing for the initiation, completion or availability of results of development of mavorixafor (X4P-001) or any of our other produc
Slide 3: Overview: Building a Global Rare Disease Franchise
Overview: Building a Global Rare Disease Franchise Developing treatments designed to have a clear and profound impact for patients suffering with rare diseases, including WHIM syndrome, and patients with rare cancers Founded in 2014, listed on Nasdaq:XFOR in March 2019 Headquarters in Cambridge, MA
Slide 4: Proven Leadership Team with Rare Disease Expertise
Proven Leadership Team with Rare Disease Expertise Key management and advisors involved with R&D and launch of only approved CXCR4 antagonist - Mozobil Paula Ragan, PhD CEO Ken Gorelick, MD CMO Adam Mostafa CFO Mary DiBiase, PhD VP of Technical Operations and Quality Celeste DiJohnson VP of Clinical
Slide 5: Pipeline
Pipeline Two oncology trials have concluded: P1b biomarker in melanoma and P1b in ccRCC. Final publications expected in 4Q19 Intend to enter into a strategic partnership for future development and potential commercialization for mavorixifor for ccRCC and other potential immuno-oncology indications P
Slide 6: CXCR4/CXCL12 and Immune System Responses
CXCR4/CXCL12 and Immune System Responses Infection Response Bacterial Viruses Fungal/Other Homeostasis Neutrophil homing Lymphocyte homing Dendritic cell trafficking Cancer Chemo-resistance/mets CTL trafficking Suppressor cell trafficking Adapted from Blood 2013 121:1501-1509 CXCL12 CHEMOKINE GRADIE
Slide 7: WHIM Syndrome: Significant Unmet Medical Need
WHIM Syndrome: Significant Unmet Medical Need Rare genetic primary immunodeficiency disease that results from “gain-of-function” mutations in the single gene that encodes the CXCR4 receptor Patients typically have chronic, critically low white blood cell counts, including neutrophils and lymphoctyes
Slide 8: Mavorixafor: Phase 3 Ready for WHIM Syndrome
mavorixafor: Phase 3 Ready for WHIM Syndrome Completed open-label, dose escalation Phase 2 trial Clinically meaningful improvement in neutropenia Patients showed improvements in certain other signs and symptoms Favorable safety profile Five patients continuing to receive mavorixafor in Phase 2 open-
Slide 9: WHIM: Genetic Mutations in CXCR4 Create Abnormal Trafficking of White Blood Cells
Blood Stream Bone Marrow WHIM Disease Goal: Treatment with X4P-001 Normal CXCR4 Signaling Hyperactive CXCR4 Signaling Corrected CXCR4 Signaling Normal WHIM: Genetic Mutations in CXCR4 Create Abnormal Trafficking of White Blood Cells (WBCs)
Slide 10: WHIM Phase 2 Study: Assess Neutrophil Counts Biomarker
WHIM Phase 2 Study: Assess Neutrophil Counts Biomarker “Time Above Threshold” Metric Objective: Increase Daily Neutrophil Counts (ANC) Above Threshold As Measured Over 24 hours: Time Above Threshold (“TAT”) Intra-Patient Dose Escalation Open label 50 mg to 400 mg once daily (QD) n = 8 patients Inclu
Slide 11: Phase 2: Achieved Maximum TAT In Most Patients
Phase 2: Achieved Maximum TAT In Most Patients Neutrophil and Lymphocytes Mobilized; Pan-Leukopenia Addressed Threshold is 500 cells/mL The three pts below threshold had dose increased to 400 mg QD 400 mg QD (n=3) Assessments Result Neutrophil Counts > Threshold 5 of 7 patients (71%): maximum TAT Ly
Slide 12: Dramatic Reduction in Wart Burden and Infection Rates
Dramatic Reduction in Wart Burden Through 55 Weeks Reductions in Infections Rate Compared to Historical Rates 1. Dale et al, ASH, 2019; 2. McDermott, et a. Blood, 2014. Minimal infections in three patients dosed for over 9-months (0.08 infections/pt/month)1 Historical infection rates reported in WHI
Slide 13: Phase 3 Trial in WHIM Syndrome – Expected 2Q19 Initiation
Phase 3 Trial in WHIM Syndrome – Expected 2Q19 Initiation Meets entry criteria X4P-001 (n=9) Placebo (n=9) 52 weeks Establish AUC Baseline 1:1 Random-ization Roll-over to Open-label Study 400 mg QD dosing in patients 12 years of age or higher Primary endpoint: biomarker of neutrophil count time abov
Slide 14: Label Expansion: Phase 1 Trial in Severe Congenital Neutropenia
Primary Immunodeficiency Label Expansion : Phase I Trial in Severe Congenital Neutropenia Rare blood disorder Characterized by abnormally low levels of certain white blood cells (neutrophils <1,500 cell/ul)1 From birth, fevers, severe bacterial infections (at times life-threatening), pneumonias, ora
Slide 15: CXCR4 in Cancer: Waldenström's Macroglobulinemia (WM)
CXCR4 in Cancer: Waldenström’s Macroglobulinemia (WM) Rare Form of Non-Hodgkin’s Lymphoma Estimated prevalence of >13,000 in US and EU1 Annual incidence: 1000-1500 in US2; ~1,800 in EU3 Signs and Symptoms Elevated IgM and other blood-markers Hepatomegaly, splenomegaly, skin purpura ~8-year survival
Slide 16: CXCR4 WHIM R/R Waldenström's: Poor Clinical Outcomes vs. Wild-type
CXCR4WHIM R/R Waldenström’s: Poor Clinical Outcomes vs. Wild-type Phase 1/2 Study Targets CXCR4-Mutant Population Very Good Partial Response (VGPR) Rates: 9.5% vs. 44.4% for wild-type; no Complete Responses (CRs) in either1 Median time to major response of 6 months vs. 2 months for wild-type1 Median
Slide 17: IO Strategy: Goal of Leveraging Biological Expertise Via Partnering
IO Strategy: Goal of Leveraging Biological Expertise Via Partnering Completed Trials Demonstrate Single Agent Activity & Proof of Mechanism Phase 1b In Treatment Naïve Melanoma Phase 1b In Progressing RCC w/Opdivo On-Going Phase 2a ccRCC Trial: Mavorixafor + Axitinib 65-patients; multi-national, ful
Slide 18: Epidemiology Suggests Significant Market Opportunity
Epidemiology Suggests Significant Market Opportunity Partnering with World Class Organizations to Increase Awareness in Primary Immunodeficiencies 1. Represents CXCR4-mutatant patients; 30% to 40% of total WM estimate of 13,000 patients Clinical Epidemiology (Estimated patients in US & EU) 2,000 – 3
Slide 19: Significant Progress Expected 2019 to 2021
Significant Progress Expected 2019 to 2021 Target Date Milestones 2H19 Phase 2a ccRCC PFS data readout Mid 2019 EMA Orphan Drug Designation for WHIM 4Q19-1Q20 WHIM patient identification update 2019 Commence Phase I trial in SCN 2019 Commence Phase 1/2 in Waldenstrom’s 1H20 New pipeline molecules: 0
Slide 20: Rare Diseases Innovate
April 2019 RARE DISEASES RARE DISEASES INNOVATE 4
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