X4 Pharmaceuticals, Inc. · NASDAQ:XFOR
X4 Pharmaceuticals — Technical Presentation (September 2019)
15 slides from X4 Pharmaceuticals's technical presentation, Sep 30, 2019
Slide 1: Data from Phase 2a Trial of Mavorixafor in Combination with Axitinib in Advanced ccRCC
DATA from Phase 2a Trial of Mavorixafor in Combination with Axitinib in Advanced Clear Cell Renal Cell Carcinoma ESMO 2019 CONGRESS, BARCELONA INVESTOR CONFERENCE CALL SEPTEMBER 30, 2019 Exhibit 99.2
Slide 2: Agenda
AGENDA Welcome Stephanie Carrington, Investor Relations Overview of Mavorixafor and Oncology Data Lynne Kelley, MD, Chief Medical Officer Data from Phase 2a portion of open-label Phase 1/2 trial of mavorixafor (X4P-001) in combination with axitinib (Inlyta®) in patients with advanced clear cell rena
Slide 3: Forward Looking Statements
FORWARD LOOKING STATEMENTS This presentation contains forward-looking statements. All statements other than statements of historical facts contained in this presentation are forward-looking statements, including, but not limited to, statements regarding X4’s plans for the development of mavorixafor
Slide 4: Overview of Mavorixafor (X4P-001)
Overview of Mavorixafor (X4P-001) Oral once a day small molecule CXCR4 antagonist that allosterically inhibits the binding by CXCL12/SDF1-α, the only known CXCR4 ligand1 Single-agent chronic treatment with 400 mg QD of mavorixafor was observed to be well-tolerated with only Grade 1 treatment-related
Slide 5: Mavorixafor: Mechanism of Actions
Mavorixafor: Mechanism of Actions
Slide 6: Mavorixafor Increases Tumor Immune Cell Infiltration with CD8+ T-cells and Activation in Phase 1b Trial
Mavorixafor Increases Tumor Immune Cell Infiltration with CD8+ T-cells and Activation in Phase 1b Trial Multiplex immunohistochemistry staining of tumor samples from a melanoma patient treated with 400 mg QD mavorixafor monotherapy. Biopsy samples obtained pre-dose and after 3 weeks of mavorixafor m
Slide 7: Role of CXCR4 and Renal Cell Carcinoma
Role of CXCR4 and Renal Cell Carcinoma Approximately 70% of sporadic clear cell renal cell carcinoma (RCC) patients have a loss of VHL gene function that drives tumor angiogenesis by increasing VEGF receptor expression1 A number of tyrosine kinase inhibitors (TKIs) that target the VEGF pathway have
Slide 8: Data from Phase 2a portion of open-label Phase 1/2 Clinical trial
Data from Phase 2a portion of open-label Phase 1/2 Clinical trial Poster Discussion Session at ESMO Abstract #2521: Safety and Efficacy of the Oral CXCR4 Inhibitor X4P-001 + Axitinib in Advanced Renal Cell Carcinoma Patients: An Analysis of Subgroup Responses by Prior Treatment Lead study investigat
Slide 9: Study Design
Study Design Phase 1/2, multi-center, open-label trial, n = 65 for safety analyses Treatment: 400 mg mavorixafor (200 mg BID or 400 mg QD) + 5 mg BID axitinib Patient populations: patients with histologically confirmed clear cell RCC who have received at least 1 prior systemic therapy and ECOG ≤ 2 T
Slide 10: Demographic and Baseline Characteristics
Demographic and Baseline Characteristics
Slide 11: Combination treatment with mavorixafor + axitinib generally well-tolerated with manageable safety profile
Combination treatment with mavorixafor + axitinib was observed to be generally well-tolerated in the trial with a manageable safety profile Ten patients (15%) discontinued combination therapy due to treatment-related AEs (mavorixafor or axitinib) of creatinine increase (3 pts); hypertension (2 pts);
Slide 12: Combination treatment demonstrated clinical benefit in heavily pretreated RCC patients
Combination treatment with X4P-001 (mavorixafor) + axitinib demostrated clinical benefit in trial in heavily pretreated RCC patients with advanced disease Objective Response Rates: All clinically evaluable pts (N = 62): 29% Immediate prior TKI therapy (N = 34): 18% Immediate prior IO therapy (N = 18
Slide 13: Subgroup-specific clinical benefit based on immediate prior therapy received
Subgroup-specific clinical benefit of mavorixafor + axitinib combination treatment based on immediate prior therapy received
Slide 14: Conclusion and Future Direction
Conclusion and Future Direction Conclusion: Combination therapy with 400 mg QD Mavorixafor + 5 mg BID axitinib was observed in the trial to be generally well-tolerated with a manageable safety profile Mavorixafor + axitinib demonstrated encouraging mPFS in this heavily pretreated advanced RCC patien
Slide 15: Data from Phase 2a Trial of Mavorixafor in Combination with Axitinib in Advanced ccRCC
DATA from Phase 2a Trial of Mavorixafor in Combination with Axitinib in Advanced Clear Cell Renal Cell Carcinoma ESMO 2019 CONGRESS, BARCELONA INVESTOR CONFERENCE CALL SEPTEMBER 30, 2019
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