Slide 1: Corporate Presentation 2016: Creating Breakthrough Therapies from Natural Human Immunity
Corporate Presentation 2016 NASDAQ: XBIT Creating Breakthrough Therapies from Natural Human Immunity
Slide 2: Presentation Contains Forward Looking Statements
1 This presentation contains forward - looking statements, including declarations regarding management's beliefs and expectations,that involve substantial risks and uncertainties . In some cases, you can identify forward - looking statements by terminology such as "may," "will," "should," "would," "
Slide 3: XBiotech Facilities (New 48 Acre Campus Location)
2 30,000 ft 2 R&D Laboratories, Manufacturing, Administrative 16 ,000 ft 2 Clinical Operations, Manufacturing Filling Suite, Vivarium 40,000 ft 2 Manufacturing, Quality Control Laboratory, Administrative XBiotech Facilities (New 48 acre Campus Location)
Slide 4: Snapshot December 2016
3 • Incorporated in 2005, IPO April 2015 (NASDAQ XBIT) • 107 Employees • 86,000 ft 2 Operations in Austin, Texas • Operations include R&D, Manufacturing, Sales & Marketing (planned) • Incorporated in United States, Switzerland, Germany, Japan, Canada • Over 100 Clinical Sites in Operation in Various
Slide 5: Corporate Board
4 Corporate Board Dr. Fabrizio Bonanni , Former Executive VP Operations Amgen, Developed World’s Largest Biologics Manufacturing Operation Thorpe McKenzie Founding Partner of Tiger Fund, One of the Most Iconic Investment Funds in the History of Wall Street. John Simard Founder CTL ImmunoTherapies ,
Slide 6: XBiotech Today
5 Pending Marketing Authorization for First - In - Class Therapy for Colorectal Cancer Rich Pipeline. Three Fast - Tracked Development Paths: Oncology , Cardiovascular, Infectious Disease 100% Ownership of Assets , $ 0 Debt World’s Leading Disposable Manufacturing Program (GMP Certified Oct. 2016) X
Slide 7: The Human Antibody Repertoire: A Remarkable Platform for Breakthrough Medicines
6 Healthy Human Volunteers with Natural Immunity to Disease The Human Antibody Repertoire: A Remarkable Platform for Breakthrough Medicines
Slide 8: Program Pre - Clinical Phase I Phase II Phase III Pending Marketing Authorization 2
7 Program 1 Pre - Clinical Phase I Phase II Phase III Pending Marketing Authorization 2 Symptomatic Colorectal Cancer Advanced Colorectal Cancer Type II Diabetes Peripheral Vascular Disease Psoriasis Acne Pyoderma Gangrenosum Hidradentitis Suppurativa S. aureus Bacteremia C. difficile Influenza Herp
Slide 9: Anti-Inflammatory Programs
8 Anti - Inflammatory Programs • Type II Diabetes • Peripheral Vascular Disease • Pyoderma Gangrenosum • Psoriasis • Hidradenitis Supperativa • Acne
Slide 10: Peer-Reviewed Publications on Clinical Findings
9 Peer - Reviewed Publications on Clinical Findings • MABp1, a first - in - class true human antibody targeting interleukin - 1α in refractory cancers: an open - label, phase 1 dose - escalation and expansion study • Xilonix , a novel true human antibody targeting the inflammatory cytokine interleuk
Slide 11: Clinical Publications (Continued)
10 • An open label, phase 2 study of MABp1 monotherapy for the treatment of acne vulgaris and psychiatric comorbidity • A randomized phase II study of Xilonix , a targeted therapy against interleukin 1α, for the prevention of superficial femoral artery restenosis after percutaneous revascularization
Slide 12: Oncology
11 Oncology • Xilonix Monotherapy in Colorectal Cancer: Two Phase III Programs • Development of Novel Endpoint Radiological/Quality of Life Measure : A Direct Measure of Clinical Benefit and a S urrogate Measure of Anti - Cancer Activity • Xilonix Suitable for Combination with Multiple Therapies • P
Slide 13: Xilonix Therapy in Colorectal Cancer
12 Xilonix Therapy in Colorectal Cancer 40% of Advanced Colorectal Cancer Patients that Complete an 8 Week Treatment with Xilonix Achieve Clinical Response … That is Nearly Twice as Many Compared to Placebo. ( Per Protocol Population for a Randomized Double - Blind Placebo Controlled Phase III Study
Slide 14: Achieving the Clinical Response Means
13 Achieving the Clinical Response Means: • Twice as Likely to Have Stable Disease 1 • Five T imes Less Likely to Have Serious Adverse Events 2 1 24.1% of Patients Achieving Primary Endpoint had stable disease at 8 weeks vs 11.7% for those that did not (p=0.0062) 2 5.7% of Patients Achieving Primary
Slide 15: Clinical Response Associated with Wide Ranging Improvement in Health
14 Clinical Response Associated with Wide Ranging Improvement in Health x Increase in Lean Body Mass (1.4kg) x Improved Global Quality of Life x Improved Role Function x Improved Emotional Function x Improved Social Function x Reduced Pain x Reduced Fatigue x Increased Appetite ( P < 0.001 for each
Slide 16: Clinical Responders Nearly Twice as Likely to Live Longer
15 Patients Achieving Clinical Response After 8 Weeks Were Nearly Twice as Likely to Live Longer 1 After 8 weeks on Study 40% of Xilonix Patients Met Primary Endpoint vs 23% on Placebo (p< 0.0033) n =139 P=<0.0001 Patients Achieving Primary Endpoint (Overall Survival 11.7 months) All Other Patients
Slide 17: Summary of Patient Benefit During Treatment with Xilonix
16 Summary of Patient Benefit During an 8 Week Treatment Regimen with Xilonix • Nearly Twice as Likely to Have Increased Survival • Twice as Likely to Have Stable Disease • Improvement in All Life Quality Measures, Including Physical, Functional and Emotional Health • Five Times Less Likely to have
Slide 18: EU Marketing Authorization
17 EU Marketing Authorization • Europe Represents by Far the Largest Unmet Need for Colorectal Cancer Therapy • Discussion with European Medicines Agency for Marketing Approval is Ongoing and Decision Not Expected Until 2017 • XBiotech Plans to Sell and Market Xilonix in Europe Subject to Regulatory
Slide 19: Ongoing Global Phase III Study Under US FDA Fast-Track Program
18 Ongoing Global Phase III Study Under US FDA Fast - Track Program • Double - Blind Placebo Controlled Randomized Study Involving 600+ Patients • Enrollment Completed On Schedule in November 2016 and Results of First Interim Analysis Expected in 1 st Quarter 2017 • First Interim Analysis Enables In
Slide 20: Anti-Infective Programs
19 Anti - Infective Programs • Staphylococcus aureus • Clostridium difficile • Influenza Virus • Ebola • Herpes Varicella Zoster Virus (Chickenpox)
Slide 21: CDC: Staph Classified as Serious Threat
20 CDC: Staph classified as “Serious Threat” • Staph Kills an Estimated 20,000 Patients in the US Every Year 1 • Antibiotic Resistance is Not Being Solved with Small Molecules • No Effective Vaccines and Growing Consensus that Such an Approach is Difficult, if Not Impossible • Repeated Failures Due
Slide 22: FDA Fast Track 514G3 in Phase II Study
21 FDA Fast Track 514G3 in Phase II Study • A Single Dose of Antibody to Mediate Therapy • Neutralize Immune Evasion Mechanism and Facilitate Immune Clearance • Targets All Forms of S. aureus — Including MRSA, MSSA • Double - Blind, Placebo Controlled Randomized Study in 52 Patients • Enrollment Com
Slide 23: Staphylococcus aureus Fc Binding Protein (Protein A)
Staphylococcus aureus Fc Binding Protein (Protein A) Protein A Binds Antibodies by Fc Serum Antibody Fc Portion of Antibody Cartoon Depiction of S. aureus with Antibody Bound by Fc Region
Slide 24: Serum Antibodies Bind ProA Forming Protective Coat on S. aureus
Serum Antibodies Bind ProA Forming Protective Coat on S. aureus • Evade Detection • Neutralize Antibodies • Prevent Immune Control Protect Coat Helps S. aureus
Slide 25: True Human Antibody (514G3) Binds ProA
True Human Antibody (514G3) Binds ProA 514G3 • Fc portion of 514G3 does NOT Bind ProA • 514G3 Binds with Correct Geometry to Block ProA and Mediate Clearance
Slide 26: Clostridium difficile Disease Burden
• 500,000 People in USA Contract Disease Annually • 1/9 Persons Over 65 Years of Age Die Within 30 days of Contracting Disease • 29,000 Deaths Annually in the US Clostridium difficile • Currently No Means of Preventing Disease http:// www.cdc.gov /media/releases/2015/p0225 - clostridium - difficile.
Slide 27: Best Way to Treat is to Prevent
26 Best Way to Treat is to Prevent Pathological Specimen Showing Pseudomembranous Colitis Image: https :// en.wikipedia.org /wiki/ Clostridium_difficile_infection
Slide 28: Developing a Novel Therapy to Prevent Deadly C. difficile Infection
27 Developing a Novel Therapy to Prevent Deadly C . difficile infection • FIRST Oral Delivered Monoclonal Antibody Therapy • Targeting Clinical Launch for Year End - 2017 • Prophylaxis Therapy — Only Approach Designed to Eradicate Bacteria Rather than Target only C. difficile toxins • Safety and Tol
Slide 29: Strategic Manufacturing: Industry Leading Disposable Platform
28 Strategic Manufacturing — Industry Leading Disposable Platform Artist’s Conception Unprecedented Reduction in Cost and Flexibility
Slide 30: Near Term Corporate Goals
29 Near Term Corporate Goals □ Seek Registration for Xilonix in Europe and other markets for CRC □ Establish Sales and Marketing Capability in Key Markets of Europe □ Continue Clinical Development in Colorectal and Other Oncology Indications, Including Combination Therapies □ Develop Pivotal Study f
Slide 31: Thank You
30 Thank You