Athersys, Inc. · OTC:ATHXQ
Athersys — Investor Presentation (March 2020)
42 slides from Athersys's investor presentation, March 2020
Slide 1: Company Overview
Company Overview March 2020 NASDAQ: ATHX www.Athersys.com 1
Slide 2: Forward Looking Statements
Forward Looking Statements This presentation has been prepared solely for informational 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, or the Securitie
Slide 3: Athersys Company Snapshot
Athersys Company Snapshot (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 & economic i
Slide 4: Summary Financial Data (Q4, 2019)
Summary Financial Data (Q4, 2019) Market Cap (as of December 31, 2019) $197M Shares Outstanding 159.8M $ Thousands Year ended December 31, 2019 Revenues $5,633 Net loss $(44,582) Net cash used in operating activities $(35,326) Net cash provided by financing activities $19,887 Cash and cash equivalen
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 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
Slide 7: Experienced Executive Leadership
Experienced Executive Leadership Name Title Prior Experience Gil Van Bokkelen, PhD Chairman & CEO William BJ Lehmann President & COO McKinsey & Company, Inc.; Wilson,, and B.A. from the University of Notre Dame) Ivor Macleod Chief Financial Officer Amgen; Scripps Clinic (Ph.D. in Cancer Biology from
Slide 8: Overview: MultiStem Cell Therapy
Overview: MultiStem® Cell Therapy Technology & Product Summary: No Ethical Concerns Highly Scalable MultiStem is derived from Millions of doses may be the bone marrow of healthy, produced from a single consenting adult donors donor bank Based on Proprietary Promotes Healing Administered Systemically
Slide 9: Practical: Simple to Prepare & Easy to Administer
Practical: Simple to Prepare & Easy to Administer 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 SIFU™ Technology ❖ Proprietary cryogenic storage system ❖ Simplifies product preparation to a single step after order entry (and capable of processing multiple doses simultaneously) ❖ Secur
Slide 11: Scalable Manufacturing: Key Competitive Advantage
Scalable Manufacturing: 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 Culture 11
Slide 12: Multimodal Biologic Product
Multimodal Biologic Product INFLAMMATION REDUCTION IMMUNOMODULATION (reparative mechanisms) NEUROPROTECTION CYTOPROTECTION ANGIO / VASCULOGENESIS CELLULAR REGENERATION REPLACEMENT 12
Slide 13: Potential for Cell Therapy in Ischemic Stroke
Potential for Cell Therapy in Ischemic Stroke Example: Leading cause of serious disability and third leading cause of Ischemic Stroke Damaged area mortality globally Clot or plaque Annually ~17 Million globally - including ~800,000 stroke victims in U.S., ~2.2 Million across U.S. + EU + Japan combin
Slide 14: MultiStem Therapy Could Meaningfully Extend the Treatment Window
MultiStem Therapy Could Meaningfully Extend the Treatment Window for Stroke Patients 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® Therapy 36
Slide 15: Spleen's Role in Inflammatory Damage in the Brain following Stroke
Spleen’s Role in Inflammatory Damage in the Brain following Stroke 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 impairment) F
Slide 16: Deep Understanding of Therapeutic MOA's of IV Administration of MultiStem
Deep Understanding of Therapeutic MOA’s of IV Administration of MultiStem for Stroke 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 in the brain.
Slide 17: Published Preclinical Literature
ELSEVIER Intravenous multipotent adult progenitor cell therapy for traumatic brain injury: Preserving the blood brain barrier via an interaction with splenocytes Peter A. Walker*®, ShinilK, Shah*"4, Fernando Jimenez”, Michael H. Gerber**, Hasen Xue, Rochelle Cutrone®, Jason A. Hamilton, Robert W. Ma
Slide 18: MultiStem Stroke Clinical Trial
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 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 of three esta
Slide 20: Clinical Sites Participating in MASTERS-1 Trial
Clinical Sites Participating in MASTERS-1 Trial 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
MultiStem Clinical Study in Ischemic Stroke: MASTERS-1 (B01-02) Trial Design Overview IV Administration of MultiStem or placebo within 24 – 36 hours of ischemic stroke Key Eligibility Criteria – Original Design ̶ Cortical cerebral ischemic strokes with substantial, persistent deficits Cortical Strok
Slide 22: Summary MASTERS-1 Baseline Demographics Information
Summary MASTERS-1 Baseline Demographics Information 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+device
Slide 23: Final Trial Results: Significant Benefit at One Year
Final Trial Results: Treatment w/ MultiStem Shows Significant Benefit at One Year 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% (n=65) 14.9%
Slide 24: MultiStem Improvement Evident Across the Severity Spectrum
MultiStem Improvement Evident Across the Severity Spectrum – Early-Treated MultiStem Subjects v. Placebo 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% 26.2% 42.6% P
Slide 25: mRS Distribution (Shift) at One Year – ITT
mRS Distribution (Shift) at One Year – ITT 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 >95 PLACEBO
Slide 26: Improvement in Excellent Outcome Following Treatment with MultiStem
Improvement in Excellent Outcome Following Treatment with MultiStem Therapy 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 Patients Early-trea
Slide 27: Other Findings from MASTERS-1 Study
Other Findings from MASTERS-1 Study 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 Administrati
Slide 28: Additional Clinical Observations from the MASTERS-1 Stroke Study
Additional Clinical Observations from the MASTERS-1 Stroke Study 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 Events MultiSt
Slide 29: Clinical Biomarker Data: Impact on Key Inflammatory Markers
Clinical Biomarker Data: MultiStem has an Impact on Key Inflammatory Markers 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, controlling
Slide 30: 2016 Partnership with Development Focus in Japan
2016 Partnership with Development Focus in Japan Alliance Overview: Initial partnership with Healios established January 2016 - Therapeutic focus on treating ischemic stroke patients in Japan - Economic impact to ATHX: $15 million up front payment with > $225 million total additional potential payme
Slide 31: Accelerated Approval System for Cell Therapy Products in Japan
Accelerated Approval System for Commercialization of Cell Therapy Products in Japan Traditional Approach New Framework 31
Slide 32: 2018 Alliance Expansion
2018 Alliance Expansion 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 ARDS and certain t
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) Trial Overview – (focused on North America and Europe – initiated and enrolling subjects) Intravenous administration of investigation product (MultiStem cell therapy or placebo) wit
Slide 34: Acute Inflammatory Pulmonary Disease
Acute Inflammatory Pulmonary Disease Acute Respiratory Distress Syndrome (ARDS) afflicts approximately 500,000 patients in Europe, the United States and Japan combined annually – Note: An outbreak of highly infectious pathogens (e.g. SARS, Coronavirus, Influenza) that result in severe acute pulmonar
Slide 35: COVID-19 and other emergent outbreaks resulting in pulmonary distress syndrome
COVID-19 and other emergent outbreaks resulting in pulmonary distress syndrome 1 2 3 4 5 Pulmonary hyperinflammatory No current treatment for Subject becomes symptomatic and response can occur, leading to severe Normal healthy subject is Subject is infected but is ARDS, only ventilator immune respon
Slide 36: MultiStem Reduces Inflammation in Human Donor Lungs
MultiStem Reduces Inflammation in Human Donor Lungs with Ischemic Reperfusion Injury 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 designated
Slide 37: MUST-ARDS Trial – Primary Results
MUST-ARDS Trial –Primary Results Presented at American Thoracic Society (ATS) Annual Meeting - May 20, 2019 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 Mu
Slide 38: Healios ONE-BRIDGE study in Japan evaluating pneumonia-induced ARDS
The Healios ONE-BRIDGE study in Japan is evaluating impact on pneumonia-induced ARDS Post-hoc analysis of Pneumonia-Induced ARDS (from MUST-ARDS) (Severe cases – PaO2/FiO2 Ratios at Day 0, Pre-infusion < 150) MultiStem Placebo Day-28 Mortality 20% 50% Ventilator-free days (mean) 14.8 7.5 Ventilator-
Slide 39: Other Key Findings from MUST-ARDS Trial
Other Key Findings from MUST-ARDS Trial Among MultiStem treated patients (1 dose – administered IV within 4 days of being diagnosed with ARDS and placed on ventilator): Consistent safety and tolerability observed Rapid improvement observed among MultiStem treated subjects – Meaningfully higher proba
Slide 40: Other Neurological Injury & Disease Areas of Interest
Other Neurological Injury & Disease Areas of Interest 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 Injury (TBI)
Slide 41: Opportunity in Trauma
Opportunity in Trauma 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 personnel (battlefie
Slide 42: Summary
Summary Multiple important objectives achieved recently ‒ Healios advancement of TREASURE trial (w/ Sakigake designation) ‒ Fast Track and RMAT designations granted by FDA for our Phase 3 Stroke program… Positive Scientific Advice from by EMA ‒ Launch of (and continued enrollment in) MASTERS-2 trial