X4 Pharmaceuticals, Inc. · NASDAQ:XFOR
X4 Pharmaceuticals — Investor Presentation (August 2022)
25 slides from X4 Pharmaceuticals's investor presentation, Aug 5, 2022
Slide 1: Corporate Overview August 2022
Corporate Overview August 2022
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: A Unique Investment Opportunity with Significant Near-Term Milestones
3 A Unique Investment Opportunity with Significant Near-Term Milestones Unparalleled expertise in CXCR4 biology Antagonizing the CXCR4/CXCL12 axis proven to increase the expansion, maturation and mobilization of white blood cells, including neutrophils Mavorixafor – a late-stage clinical CXCR4 antag
Slide 4: Seasoned Executive Leadership Team Continues to Execute on Mission
4 Seasoned Executive Leadership Team Continues to Execute on Mission PAULA RAGAN, Ph.D. President & CEO ADAM MOSTAFA Chief Financial Officer MARY DIBIASE, Ph.D. Chief Operating Officer DIEGO CADAVID, M.D. Chief Medical Officer ART TAVERAS, Ph.D. Chief Scientific Officer
Slide 5: The Dysregulated Immune System: Immunodeficiencies
5 The Dysregulated Immune System: Immunodeficiencies Chronic Immunosuppression • Life-long severe and/or life-threatening infections • Reduced/no response to vaccines • Increased cancer risk • High morbidity (bronchiectasis, hearing loss) and life-threatening sepsis Caused By • Low white blood cell
Slide 6: CXCR4 Plays Key Role in the Expansion, Maturation & Mobilization of Immune Cells
6 Hematopoietic Stem Cell Committed Lymphoid Progenitor Cell T Lymphocytes B Lymphocytes Multipotent Stem Cell Committed Myeloid Progenitor Cell Neutrophils Monocytes BONE MARROW BLOOD Maturation MobilizationExpansion Mature neutrophil pool in bone marrow is 20 times higher than in circulation1 Sour
Slide 7: Mavorixafor: Realizing the Potential of CXCR4 Antagonism in an Oral Capsule
7 Mavorixafor: Realizing the Potential of CXCR4 Antagonism in an Oral Capsule Only oral CXCR4 antagonist in development • Small molecule with high potency and selectivity • Durable half-life supporting once-daily dosing • 2 or 4 capsules, once per day (WHIM syndrome) Antagonizes Both Wild-Type and M
Slide 8: Mavorixafor Clinical Data: Broad Durable Increases in Peripheral White Blood Cell Counts
8 Mavorixafor Clinical Data: First Oral Treatment with Broad Durable Increases in Peripheral White Blood Cell Counts Across All Populations Studied Mavorixafor in WHIM SyndromeMavorixafor in Healthy Volunteers Stone et al, Antimicrobial Agents and Chemotherapy, July 2007 ASH 2021. Poster #2186. WBC:
Slide 9: Pipeline: Significant Upcoming Milestones Expected
9 Pipeline: Significant Upcoming Milestones Expected Candidate Indication Preclinical Phase 1 Phase 2 Phase 3 Expected Milestones Target Patient Populations C H R O N IC N E U T R O P E N IC D IS O R D E R S (I n -h o u s e ) Mavorixafor WHIM Syndrome 1 (Warts, Hypogammaglobulinemia, Infections and
Slide 10: WHIM Syndrome: an Immunodeficiency Affecting Children and Adults
10 WHIM Syndrome: an Immunodeficiency Affecting Children and Adults Leanne’s Story - Living with WHIM Syndrome Pancytopenia: All White Blood Cells Affected & Reduced Severe bacterial infections In multiple organ systems: bronchiectasis (lung), hearing loss (ear), cellulitis (skin) Viral infections &
Slide 11: Root Cause of WHIM Syndrome: Over-Signaling of CXCR4
11 Root Cause of WHIM Syndrome: Over-Signaling of CXCR4 CXCR4 Receptor Mutations cause over-signaling Impacting immune cell expansion, maturation, and mobilization • WHIM Syndrome: Spectrum of clinical presentations mostly with (and sometimes without) CXCR4 mutations • W: warts • H: hypogammaglobuli
Slide 12: EHA 2020: Phase 2 WHIM Trial Demonstrates Activity Across Phase 3 Endpoints
12 EHA 2020: Phase 2 WHIM Trial Demonstrates Activity Across Phase 3 Endpoints Major (>600%) Increase in Neutrophil Counts (TATANC) >80% Reduction in Annualized Infection Rates WHIM Phase 2 Trial Design • Intra-patient dose- escalation: safety, PK • Endpoint: clinically relevant blood measurement: "
Slide 13: WHIM Phase 2 OLE: Continued Improvements Seen Across Patients
13 WHIM Phase 2 OLE: Continued Improvements Seen Across Patients * Improvements in warts categorized as either a reduction of wart size, decrease in number of warts, and/or the lack of new wart development. Reference range. Hemogram (units/reference range) WBC - White blood cells (x109 / 3.5-10.5),
Slide 14: Design of Mavorixafor Global Phase 3 Trial in WHIM
14 Design of Mavorixafor Global Phase 3 Trial in WHIM (Patients ≥12 years of age) • Primary Endpoint: Biomarker of time above threshold (>500 cells per microliter) for absolute neutrophil count (TATANC); average of four assessment timepoints • Secondary Endpoints: TATALC, infections, wart burden, in
Slide 15: Understanding the Definition and Diagnosis of WHIM Syndrome
15 1. Pathogenic / likely pathogenic CXCR4 mutations – including variants causing gain-of-function CXCR4 signaling 2. Myelokathexis Understanding the Definition and Diagnosis of WHIM Syndrome For the treatment of WHIM Syndrome in patients ≥12 years Diagnosis of WHIM Syndrome: Clinical and Laboratory
Slide 16: Chronic Neutropenia Beyond WHIM
16 Chronic Neutropenia Beyond WHIM Chronically immunocompromised patients due to sustained or intermittent low neutrophil counts (neutropenia); can be caused by • G-CSF response defects • Elevated bone marrow CXCL12 downregulating G-CSF production • Stem cell loss • Differentiation/maturation arrest
Slide 17: Chronic Neutropenia Management: Currently Approved Treatments Have Significant Limitations
17 Chronic Neutropenia Management: Currently Approved Treatments Have Significant Limitations and High Side-Effect Burden Injections of G-CSF • Once or twice-daily at 5-6 mg/kg to reduce severe neutropenia and risk of infections • “Dose down-titration” and reduced frequency often implemented to aid
Slide 18: > 20,000 People Estimated with Chronic Neutropenia in the U.S.
18 > 20,000 People (aged >12) Estimated with Chronic Neutropenia in the U.S.1: Thousands Have Major Health Impact with >2 Serious Infection Events per Year 1. Eligible patients in the TriNetX USA database who had an ANC <1500 cells/μl in calendar year of interest and two additional ANC <1500 cells/μ
Slide 19: Phase 1b Trial: Studies Activity of Mavorixafor in Broad CN Population
19 Protocol Version 5 • 14 days of treatment with mavorixafor • Blood sampling over 6 hours on days -1, 1 and 14 • Severe (ANC<500/uL) chronic idiopathic neutropenias on concomitant G-CSF • Selected severe congenital neutropenia conditions with or without concomitant G-CSF • Clinic only study Protoc
Slide 20: ASH 2021: Mavorixafor Impact on WBCs in Chronic Neutropenia Patients
20 ASH 2021: Mavorixafor Impact on WBCs in Chronic Neutropenia Patients Meaningful Responses* to Single Dose in Idiopathic Chronic Neutropenia Patients Absolute Neutrophil Count (ANC) * ASH 2021. Poster #2186. >2-fold increase in ANC after single dose of mavorixafor Similar increases in: ✓ Total Whi
Slide 21: Significant Opportunity for Mavorixafor in Chronic Neutropenia
21 Significant Opportunity for Mavorixafor in Chronic Neutropenia ✓More than 5,000 patients in the U.S. with chronic neutropenia at significantly increased rates of hospitalization with severe infections ✓Only available treatment is injectable, with limited tolerability and frequent injections ✓Mavo
Slide 22: Building to Support a Continuum of Patients with Chronic Neutropenic Disorders
22 Building to Support a Continuum of Patients with Chronic Neutropenic Disorders " Classic WHIM WHIM Expansion: VUS, Myelokathexis, and Other Chronic Neutropenia Efficiently leveraging resources and time across programs
Slide 23: Recent and Key Upcoming Expected Milestones
23 Recent and Key Upcoming Expected Milestones Recent Events: • Raised gross proceeds of ~$56 million from PIPE financing in June 2022 • Amended debt facility with Hercules Capital extending interest-only payment period into 2024 • Announced sharpening of corporate focus and resources on development
Slide 24: Strong Balance Sheet Supports Expected Upcoming Milestones
24 Strong Balance Sheet Supports Expected Upcoming Milestones 1 Pro-forma for June 30, 2022 cash and restricted cash balance of $48.7 plus estimated net proceeds from PIPE financing that closed on July 6, 2022. 2 As described in detail in our most recent Form 10-Q, our agreement with Hercules Capita
Slide 25: U.S. Headquarters North Beacon Street, 4th Floor Boston, MA 02134 Research Facilities Helmut-Qualtinger-Gasse 2
25 U.S. Headquarters 61 North Beacon Street, 4th Floor Boston, MA 02134 Research Facilities Helmut-Qualtinger-Gasse 2 A-1030 Vienna, Austria www.x4pharma.com NASDAQ: XFOR
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