Athersys, Inc. · OTC:ATHXQ
Athersys — Investor Presentation (November 2019)
42 slides from Athersys's investor presentation, November 2019
Slide 1: Athersys, Inc. Overview
Athersys, Inc. Overview Company Overview Presented by: Rob Perry VP, Head of Product Supply NASDAQ: ATHX and Operations www.Athersys.com November 2019
Slide 2: Forward Looking Statements
Forward Looking Statements NASDAQ: ATHX 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 Securities Act of 1933, as amended,
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 of care and quality-of-life burden Lead pr
Slide 4: Summary Financial Data (Q3, 2019)
Summary Financial Data (Q3, 2019) NASDAQ: ATHX Market Cap (as of September 30, 2019) $207M Shares Outstanding 155.3M $ Thousands Nine Months ended September 30, 2019 Revenues $5,346 Net loss $(34,659) Net cash used in operating activities $(25,173) Net cash provided by financing activities $14,994 C
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 substantial unmet medical need NASDAQ: ATHX Neurological, Inflammatory & Immune, Cardiovascular, 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, JD President & COO University of Notre Dame) Amgen; Scripps Clinic (Ph.D. in Cancer Biology from Stanford John Harri
Slide 8: MultiStem Cell Therapy
MultiStem Cell Therapy NASDAQ: ATHX Technology & Product Summary: No Ethical Concerns Highly scalable MultiStem is derived from Millions of doses may be the bone marrow of produced from a single healthy adult donors donor bank Based on Proprietary Promotes Healing Given Systemically or Well Characte
Slide 9: Practical: Simple to Prepare & Easy to Administer
Practical: Simple to Prepare & Easy to Administer NASDAQ: ATHX Hospital Pharmacy to Patient in < 1 hour 9
Slide 10: Enabling Efficient Storage, Access, Product Prep & Inventory Management
Enabling Efficient Storage, Access, Product Prep & Inventory Monitoring/Management in the Hospital Environment NASDAQ: ATHX SIFU™ Technology ❖ Proprietary cryogenic storage system ❖ Secure access – inventory is protected at all times ❖ Real time informatics on product inventory, storage, system acce
Slide 11: Scalable Manufacturing: Key Competitive Advantage
Scalable Manufacturing: Key Competitive Advantage NASDAQ: ATHX 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 12: Multimodal Biologic Product
Multimodal Biologic Product NASDAQ: ATHX INFLAMMATION REDUCTION IMMUNOMODULATION (reparative mechanisms) NEUROPROTECTION CYTOPROTECTION ANGIO / VASCULOGENESIS CELLULAR REGENERATION REPLACEMENT 12
Slide 13: Opportunity for Cell Therapy in Ischemic Stroke
Opportunity for Cell Therapy 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 M
Slide 14: 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 15: Spleen's Role in Inflammatory Damage in the Brain following Stroke
Spleen’s Role 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: 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 17: Published Preclinical Literature
Content lists avaiable at ScanceDiact Experimental Neurology SCIENTIFIC REP£3RTS Journal of Neurotrauma open ELSEVIER Journal homepage: www.eleevier.com/locate/yexn Intravenous multipotent adult progenitor cell treatment decreases inflammation leading to functional recovery following Intravenous mul
Slide 18: 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 19: Established & Well Accepted Regulatory Endpoints for Ischemic Stroke
Established & Well Accepted Regulatory Endpoints for Ischemic Stroke NASDAQ: ATHX There are currently 2 well accepted regulatory endpoints for therapies being evaluated for efficacy in treating acute ischemic stroke: Excellent Outcome ̶ Proportion of patients that achieve an excellent score in each
Slide 20: Clinical Sites Participating in MASTERS-1 Trial
Clinical Sites Participating in MASTERS-1 Trial NASDAQ: ATHX Double-blind, randomized, placebo-controlled Phase 2 study conducted at 33 leading international stroke centers across the U.S. and the U.K. 20
Slide 21: MultiStem Clinical Study in Ischemic Stroke: MASTERS-1 (B01-02)
MultiStem Clinical Study in Ischemic Stroke: MASTERS-1 (B01-02) NASDAQ: ATHX Trial Design Overview • IV administration of MultiStem or placebo 24-36 Key Eligibility Criteria – Original Design hours post onset of ischemic stroke Cortical Stroke - Double-blind, placebo-controlled - Dose escalation pha
Slide 22: Selected Baseline Demographics Information
Selected Baseline Demographics Information NASDAQ: ATHX Patient sample MultiStem Placebo n=65 n=61 Age, mean, range 61.8 62.6 41-83 37-80 Sex, male 53.8% 54.1% NIHSS, mean, median 13.4 13.3 13.0 13.0 MRI DWI Lesion size, mL, mean, 51.6 54.8 median 42.3 41.1 Administered iv tPA 44.6% 47.5% iv tPA+dev
Slide 23: 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) D at Day D at Day Day 90 Day 365 90 365 MultiStem 15.4% 23.1%
Slide 24: MultiStem Improvement Evident Across the Severity Spectrum
MultiStem Improvement Evident Across the Severity Spectrum – Early-Treated MultiStem Subjects v. Placebo NASDAQ: ATHX mRS Shift Analysis Day 7 Day 30 0 1 2 3 4 5/6 0 1 2 3 4 5/6 p=0.006 p=0.015 3.2% 3.2% MultiStem 9.7% 29.0% 38.7% 16.1% MultiStem 12.9% 25.8% 29.0% 25.8% 3.2% 3.2% Placebo 4.9% 26.2%
Slide 25: mRS Distribution (Shift) at One Year – ITT
mRS Distribution (Shift) at One Year – ITT NASDAQ: ATHX 0 1 2 3 4 5/6 p=0.09 MultiStem 7.7% 20.0% 21.5% 30.9% 9.2% 10.8% More excellent Fewer deaths, outcomes More independence LTEAs 1.6% Placebo 11.5% 29.5% 31.1% 8.2% 18.0% 0% 10% 20% 30% 40% 50% 60% 70% 80% 90% 100% Excellent Outcome Barthel Index
Slide 26: 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 27: 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 28: 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 29: Clinical Biomarker Data: MultiStem Impact on Key Inflammatory Markers
Clinical Biomarker Data: MultiStem has an Impact on Key Inflammatory Markers NASDAQ: ATHX Circulating CD3+ T-cell Levels, Safety / ITT Population MultiStem Placebo Day 0 18.5% 18.8% Day 2 18.4% 23.3% Change (0.1%) 4.5% P=0.001 Note: Evaluates available data from ITT population; for cytokine analyses
Slide 30: 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 31: 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 31
Slide 32: Recent Alliance Expansion
Recent Alliance Expansion NASDAQ: ATHX Expanded Collaboration – Announced June, 2018 Healios obtains expanded exclusive license to certain programs in Japan and globally – Under terms of the collaboration expansion, Healios obtained development and commercialization rights to include treatment of AR
Slide 33: 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 34: Other Portfolio Programs
NASDAQ: ATHX Other Portfolio Programs
Slide 35: Acute Inflammatory Pulmonary Disease
Acute Inflammatory Pulmonary Disease Focusing on ARDS ❖ Acute Respiratory Distress Syndrome (ARDS) afflicts approximately 500,000 patients in Europe, the United States and Japan combined annually, and >670,000 patients in China ❖ ARDS represents a major area of unmet medical need, with high mortalit
Slide 36: Acute Pulmonary Medicine
Acute Pulmonary Medicine NASDAQ: ATHX MultiStem conveys benefit through multiple mechanisms relevant to acute pulmonary inflammatory damage – Published data illustrates impact on reducing inflammatory damage in pulmonary system – Upregulation of reparative cell types and pathways Athersys and collab
Slide 37: 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 38: ARDS Trial – Initial Results
ARDS Trial – Initial Results (Presented at ATS – May 20, 2019) NASDAQ: ATHX Exploratory Ph. 2 MUST-ARDS Trial: Randomized, double-blind, placebo-controlled trial evaluating patients through 28-day clinical assessment (standard) with one year follow up. All Subjects MultiStem Placebo Number 20 10 Ven
Slide 39: The Healios ONE-BRIDGE study is evaluating pneumonia-induced ARDS
The Healios ONE-BRIDGE study is evaluating pneumonia-induced ARDS NASDAQ: ATHX 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 40: 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 41: 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 42: Summary
Summary NASDAQ: ATHX Multiple important objectives achieved recently ‒ 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 prog