Athersys, Inc. · OTC:ATHXQ
Athersys — Investor Presentation (June 2019)
37 slides from Athersys's investor presentation, June 2019
Slide 1: Athersys, Inc. Overview Corporate Overview
Athersys, Inc. Overview Corporate Overview Presented by: Rob Perry NASDAQ: VP, Head of Product ATHX and Operations Supply www.Athersys.com June, 2019
Slide 2: Forward Looking Statements
Forward Looking Statements This presentation has been prepared by us solely for information purposes. This presentation includes, and our responses to various questions may include, “forward-looking statements” within the meaning of Section 27A of The statements the Securities Act and discussions of
Slide 3: Company Snap Shot (NASDAQ: ATHX)
Company Snap Shot (NASDAQ: ATHX) NASDAQ: ATHX Established international leader in the development of innovative cell therapy and regenerative medicines Multiple clinical programs – emphasis on critical care indications with substantial unmet need, high cost and quality-of-life burden Lead program, M
Slide 4: Summary Financial Data (FY, 2018)
Summary Financial Data (FY, 2018) NASDAQ: ATHX Market Cap $229M Shares Outstanding 148.88M $ Thousands Year Ended December 31, 2018 Revenues $24,291 Operating Expenses (49,953) Other income, net 1,379 Net loss $(24,283) Net cash used in operating activities $(13,350) Net cash provided by financing a
Slide 5: Our Focus: Development of best in class regenerative medicine therapies
Our Focus: Development of best in class regenerative medicine therapies for areas of NASDAQ: ATHX substantial unmet medical need Neurological, Cardiovascular, Inflammatory & Immune and Other Indications with an Emphasis in the Critical Care Segment 5
Slide 6: ATHX Regenerative Medicine Pipeline
ATHX Regenerative Medicine Pipeline NASDAQ: ATHX 6
Slide 7: Experienced Executive Leadership
Experienced Executive Leadership NASDAQ: ATHX Name Title Prior Experience Gil Van Bokkelen, PhD Chairman & CEO McKinsey & Company, Inc.; Wilson,, and B.A. from the William BJ Lehmann President & COO University of Notre Dame) Amgen; Scripps Clinic (Ph.D. in Cancer Biology from John Harrington, PhD CS
Slide 8: MultiStem Cell Therapy
NASDAQ: ATHX Product Profile MULTISTEM® CELL THERAPY Based on Proprietary Promotes Healing and Given Systemically or Long Storage Life and MAPC Technology Tissue Repair Locally High Production Yield Off the shelf administration Works through multiple IV, catheter, injection, > 7 years of stability d
Slide 9: Practical: Simple to Prepare & Easy to Administer
Practical: Simple to Prepare & NASDAQ: ATHX Easy to Administer Hospital Pharmacy to Patient in < 1 hour 9
Slide 10: Scalable Manufacturing: Key Competitive Advantage
Scalable Manufacturing: NASDAQ: ATHX Key Competitive Advantage Distinctive and Robust Expansion Profile + Integration with Advanced Bioreactor Technology Enables Unprecedented Commercial Scale Commercial Manufacturing Commercial Scale Bioreactors 3D Small Scale Bioreactors Traditional 2D Cell Cultur
Slide 11: Multimodal Biologic Product
Multimodal Biologic Product NASDAQ: ATHX INFLAMMATION REDUCTION IMMUNOMODULATION (reparative mechanisms) NEUROPROTECTION CYTOPRTOTECTION ANGIO /VASCULOGENESIS CELLULAR REGENERATION REPLACEMENT 11
Slide 12: Challenges & Opportunity in Ischemic Stroke
Challenges & Opportunity in Ischemic Stroke NASDAQ: ATHX Example: Leading cause of disability and third leading Ischemic Stroke Damaged area cause of mortality globally Clot or plaque Annually ~800,000 stroke victims in U.S. >2.2 Million (U.S. + EU + Japan), and ~17 Million globally Note: >3.4 Milli
Slide 13: MultiStem Therapy Could Greatly Extend the Treatment Window for Stroke Patients
MultiStem Therapy Could Greatly Extend the Treatment Window for Stroke Patients NASDAQ: ATHX Time to Stroke Treatment Tissue Plasminogen 3 - 4½ hours Activator (tPA) Relevant to ≤ 10% Stroke patients Mechanical 6 hours Recent extension limited to specific types of patients Thrombectomy MultiStem® Th
Slide 14: Deep Understanding of Therapeutic MOA's of IV Administration of MultiStem for Stroke
Deep Understanding of Therapeutic MOA’s of IV Administration of MultiStem for Stroke NASDAQ: ATHX MultiStem works through regulation of multiple Ischemic factors and pathways important to brain recovery Stroke following a stroke. 1 Inflammation after stroke leads to greater tissue loss and scarring
Slide 15: Role of Spleen in Inflammatory Damage in the Brain following Stroke
Role of Spleen in Inflammatory Damage in the Brain following Stroke NASDAQ: ATHX In preclinical models of ischemic stroke, removing the spleen before a surgically induced stroke significantly reduces the inflammatory damage that typically occurs in the brain (but also creates permanent immunological
Slide 16: MultiStem Stroke Clinical Trial
NASDAQ: ATHX MultiStem Stroke Clinical Trial Results from MASTERS-1 (Phase 2) MultiStem Administration for Stroke Treatment and Enhanced Recovery Study
Slide 17: Clinical Sites Participating in Trial
Clinical Sites Participating in Trial NASDAQ: ATHX London (2) seatbens a yp \ Double blind, randomized, placebo controlled Phase 2 study conducted at 33 leading international stroke treatment centers across the U.S. and U.K. aw
Slide 18: Final Trial Results: Treatment w/ MultiStem Shows Significant Benefit at One Year
Final Trial Results: Treatment w/ MultiStem Shows Significant Benefit at One Year NASDAQ: ATHX Proportion of Subjects Achieving Excellent Outcome Increases Over Time (Patients Achieving NIHSS 0 or 1 and mRS 0 or 1, and Barthel Index >95) ∆ at Day ∆ at Day Day 90 Day 365 90 365 MultiStem 15.4% 23.1%
Slide 19: Improvement in Excellent Outcome Following Treatment with MultiStem Therapy
Improvement in Excellent Outcome Following Treatment with MultiStem Therapy NASDAQ: ATHX In contrast to longstanding clinical experience, MultiStem treated patients exhibit meaningful improvement beyond the initial 90 day recovery period Excellent Outcome (%) Excellent Outcome (%) All Treated Patien
Slide 20: Other Findings from MASTERS-1 Study
Other Findings from MASTERS-1 Study NASDAQ: ATHX Safety: Intravenous MultiStem well tolerated by Stroke patients – No infusional or allergic reactions, and no abnormal patterns in safety labs or vital signs – Adverse events consistent with expectations and experience for stroke patients of this type
Slide 21: Additional Clinical Observations from the MASTERS-1 Stroke Study
Additional Clinical Observations from the MASTERS-1 Stroke Study NASDAQ: ATHX Among the most severely disabled stroke patients, a substantial reduction observed in serious and life threatening adverse events Severe Stroke (NIHSS 15+) Subjects % Subjects with Grade 3-5 Adverse Events Through Day 30 E
Slide 22: Clinical Biomarker Data: MultiStem has an Impact on Key Inflammatory Markers
Clinical Biomarker Data: MultiStem has an Impact on Key Inflammatory Markers NASDAQ: ATHX Note: Evaluates available data from ITT population; for cytokine analyses, controlling for differences in baseline values and outliers Impact on key biomarkers (circulating immune cells and key inflammatory cyt
Slide 23: Development Focus in Japan
Development Focus in Japan NASDAQ: ATHX HEALIOS K.K. Partnership Overview Initial partnership with Healios established January 2016 - $15 million up front payment with > $225 million total additional potential payments, subject to certain credits, plus tiered double-digit royalties Initial therapeut
Slide 24: Recently Implemented Accelerated Approval System for Cell Therapy Products in Japan
Recently Implemented Accelerated Approval System for Commercialization of Cell Therapy Products in Japan Traditional Approach New Framework 24
Slide 25: Recent Healios Alliance Expansion
Recent Healios Alliance Expansion NASDAQ: ATHX Expanded Collaboration – Announced June, 2018 Healios obtains expanded exclusive license to certain programs in Japan and globally + time limited ROFN for exclusive option to certain programs in China – Under terms of the collaboration expansion, Healio
Slide 26: MASTERS-2: Pivotal Phase 3 Study in Ischemic Stroke
MASTERS-2: Pivotal Phase 3 Study in Ischemic Stroke (Authorized by FDA under SPA, w/ Fast Track and RMAT designations) NASDAQ: ATHX Trial Overview – (focused on North America and Europe – initiated and enrolling subjects) Intravenous administration of investigation product (MultiStem cell therapy or
Slide 27: Other Portfolio Programs
NASDAQ: ATHX Other Portfolio Programs
Slide 28: Acute Pulmonary Medicine
Acute Pulmonary Medicine NASDAQ: ATHX Acute Respiratory Distress Syndrome (ARDS) afflicts approximately 400,000 to 500,000 patients in Europe, the United States and Japan annually, and >670,000 patients in China – ARDS represents a major area of unmet medical need, with a high rate of mortality and
Slide 29: MultiStem Reduces Inflammation in Human Lungs with Ischemic Reperfusion Injury
MultiStem Reduces Inflammation in Human Lungs with Ischemic Reperfusion Injury NASDAQ: ATHX Assessment of human lungs isolated from organ donors that exhibit significant inflammation prior to use in transplantation, perfused with MultiStem or vehicle (saline). Note: Organ donor lungs originally desi
Slide 30: ARDS Trial – Initial Results
ARDS Trial – Initial Results (Presented at ATS – May, 20th 2019) NASDAQ: ATHX Trial Design – Phase 1 / 2a Exploratory Trial ‒ Patients evaluated through 28 days for the primary clinical assessment (standard follow up period – patients will be further assessed through a one-year follow-up period) ‒ I
Slide 31: The Healios ONE-BRIDGE study is evaluating pneumonia-induced ARDS
The Healios ONE-BRIDGE study is NASDAQ: ATHX evaluating pneumonia-induced ARDS Post-hoc analysis of Pneumonia-Induced ARDS (Severe cases – PaO2/FiO2 Ratios at Day 0, Pre-infusion < 150) MultiStem Placebo Day-28 Mortality 20% 50% Ventilator-free days 14.8 7.5 ICU-free days 12.0 5.0 Data for severe ca
Slide 32: Other Neurological Injury & Disease Areas of Interest
Other Neurological Injury & Disease Areas of Interest NASDAQ: ATHX IV Administration of MultiStem to Promote & Accelerate Healing & Repair Work conducted in preclinical models – with multiple publications in leading scientific journals Ischemic Stroke Supported by StrokeMAP Acute Traumatic Brain
Slide 33: Opportunity in Trauma
Opportunity in Trauma NASDAQ: ATHX Trauma is the leading cause of death and serious disability among individuals age <45 in the U.S. – Leading cause of life years lost among individuals up to age 75… and third leading cause of death overall – Significant impact on youth, elderly and military personn
Slide 34: Phase 2 Clinical Trial in Acute Myocardial Infarction
Phase 2 Clinical Trial in Acute Myocardial Infarction NASDAQ: ATHX Treating most common type of myocardial infarction (NSTEMI) – Trial builds on successful Phase 1 study data – NSTEMI incidence has been relatively stable, but expected to increase further with aging demographics and other factors (i.
Slide 35: Summary
Summary NASDAQ: ATHX Multiple important objectives achieved ‒ Sakigake priority review designation for ischemic stroke program in Japan (obtained by Healios with our support) ‒ Healios initiation of TREASURE trial ‒ Fast Track and RMAT designations granted by FDA for our Phase 3 Stroke program ‒ Pos
Slide 36: Analyst Coverage for ATHX
Analyst Coverage for ATHX NASDAQ: ATHX Firm Analyst 12 Month Target Price William Blair [Katherine Xu, PhD] $12.00 per share Maxim Group Jason McCarthy $8.00 per share Needham & Company, LLC Chad Messer $6.00 per share WBB Securities Stephen Brozak $9.00 per share 36
Slide 37: Athersys, Inc. Overview Corporate Overview
Athersys, Inc. Overview Corporate Overview Presented by: Rob Perry NASDAQ: VP, Head of Product ATHX and Operations Supply www.Athersys.com June, 2019