X4 Pharmaceuticals, Inc. · NASDAQ:XFOR
X4 Pharmaceuticals — Arsanis — Merger or Acquisition (November 2018)
20 slides from X4 Pharmaceuticals's merger presentation, Nov 27, 2018
Slide 1: Arsanis and X4 Pharmaceuticals Merger
Arsanis and X4 Pharmaceuticals Merger November 27, 2018 Exhibit 99.1
Slide 2: Forward Looking Statements
Forward Looking Statements Certain statements in this communication regarding the proposed merger and other contemplated transactions (including statements relating to satisfaction of the conditions to and consummation of the proposed merger; the expected ownership of the combined company; the alter
Slide 3: Participants and Additional Information
Participants and Additional Information No Offer or Solicitation: This communication shall not constitute an offer to sell or the solicitation of an offer to sell or the solicitation of an offer to buy any securities, nor shall there be any sale of securities in any jurisdiction in which such offer,
Slide 4: Transaction Overview
Transaction Overview X4 Pharmaceuticals to merge with Arsanis in stock for stock transaction Combined company will operate as X4 Pharmaceuticals, Inc. Paula Ragan, X4’s president and chief executive officer, will maintain this role and the remainder of the management team at X4 are expected to conti
Slide 5: X4 Pharmaceuticals: Company Overview
X4 Pharmaceuticals: Company Overview 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, $75 million in capital raised to-date (Cormorant Asset Management largest inves
Slide 6: X4 Pharmaceuticals: Building a Global Rare Disease Franchise
X4 Pharmaceuticals: Building a Global Rare Disease Franchise Rare disease focus provides opportunity for rapid path to global commercialization with disease-modifying lead $1B+ market potential across multiple rare diseases Multiple clinical trials planned while building towards Phase 3 top-line res
Slide 7: 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 8: Pipeline
Product Candidate Indication Stage of Development Next Anticipated Milestone(s) Preclinical Phase 1 Phase 2 Phase 3 X4P-001 WHIM syndrome 1H:2019 – Initiate Phase 3 trial 2021 – Top-line Phase 3 data Severe Congenital Neutropenia (SCN) 1H:2019 – Initiate Phase 1/2 1H:2020 – Data from Phase 1/2 Walde
Slide 9: Strategy Focused on Strong and Steady Value Build
Strategy Focused on Strong and Steady Value Build Towards a Diversified Commercial Rare Disease Business 2021 1H19 1H19 2H19 1H20 2H20 003 PID P1B Data Initiate WHIM P3 Trial Initiate WM P1/2 Trial WM P1/P2 Data 003 PID IND Initiate SCN P1/2 Trial SCN P1/P2 Data WHIM P3 Topline Data 002 GBM P1B Data
Slide 10: 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 11: Overview: X4P-001 for WHIM
Overview: X4P-001 for WHIM WHIM Disease results from “gain-of-function” mutations in the CXC chemokine receptor 4 gene X4P-001 Phase 2 studies complete; expected to enter Phase 3 in 1H19 Proof of concept in WHIM previously demonstrated with Mozobil (twice-daily injectable CXCR4 antagonist)1 We belie
Slide 12: >1,000 WHIM Patients Estimated in US
>1,000 WHIM Patients Estimated in US: X4 Primary Research (2017) MDs from 5 specialties self-identified having genetically confirmed or highly suspected WHIM patients Extrapolated Prevalence from WHIM Genetically Confirmed Patients Per MD Subset analysis - 24 MDs care for 62 genetically confirmed WH
Slide 13: WHIM Disease Goal: Treatment with X4P-001
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) 1. McDermott et al; Blood, 2014 Proof of Concept in WHIM Pre
Slide 14: Phase 2 Study Design: Neutrophil Counts Biomarker
Phase 2 Study Design: Neutrophil Counts Biomarker Phase 2 Goal: Daily Neutrophil and Lymphocyte Counts Exceed Target Thresholds Intra-Patient Dose Escalation Inclusion Criteria Endpoints Open label 50 mg to 400 mg QD n=4-10 patients Neutrophil count: ANC ≤400/μL or Lymphocyte count: ALC ≤650/μL or b
Slide 15: Phase 2: Achieved Threshold Targets In Most Patients
Phase 2: Achieved Threshold Targets In Most Patients Neutrophil and Lymphocytes Mobilized; Pan-Leukopenia Addressed Patients Started with an ANC of 50 – 100 cells/ul Prior to Treatment DRC Recommended 400 mg QD for Phase 3 Trial 300 mg QD (n=7) 400 mg QD (n=3) Neutrophil Threshold = 600 cells/mL 3 p
Slide 16: Dramatic Reduction in Wart Burden Consistent with Disease Modifying MOA
Dramatic Reduction in Wart Burden Consistent with Disease Modifying MOA Hands: Pre-Rx Hands: At 26-wks Rx Right Thumb Pre-Rx Right Thumb At 26-wks Rx Right Middle Finger Pre-Rx Right Middle Finger at 26-wks Rx Patient Remains on Treatment Patient 001-006
Slide 17: Phase 3 Trial of X4P-001 in WHIM – Expected Initiation in 1H 2019
Phase 3 Trial of X4P-001 in WHIM – Expected Initiation in 1H 2019 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
Slide 18: X4P-001: Additional Opportunities
X4P-001: Additional Opportunities Severe Congenital Neutropenias (SCN) Rare blood disorder Prevalence of 2,000-3,000 patients in US and EU Characterized by abnormally low levels of certain white blood cells (neutrophils <500 cell/ul) Genetic drivers: May be inherited as either an autosomal dominant
Slide 19: IO Strategy: Leveraging Biological Expertise Via Partnering
IO Strategy: Leveraging Biological Expertise Via Partnering Three On-Going Trials: Demonstrating PoC Phase 1b – melanoma biomarker with pembrolizumab Phase 1b – clear cell renal cell carcinoma with nivolumab Phase 1b/2a – clear cell renal cell carcinoma with axitinib Clinical collaboration with Pfiz
Slide 20: Key Benefits of the Merger
Key Benefits of the Merger Leverages X4’s deep experience in rare disease products and its advanced pipeline with Arsanis’ European infrastructure and related scientific research expertise Provides near-term capital to support the Phase 3 trial of X4P-001, a potentially disease modifying treatment f