Athersys, Inc. · OTC:ATHXQ
Athersys — Investor Presentation (September 2021)
48 slides from Athersys's investor presentation, September 2021
Slide 1: Company Overview
Company Overview September 2021 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 late-stage clinical development in the field of innovative cell therapy and regenerative medicine; Late-stage clinical focus is on critical care indications, with three Fast track and two RMAT designations from the FDA; Our
Slide 4: Summary Financial Data (Q2, 2021)
Summary Financial Data (Q2, 2021) Market Cap (as of June 30, 2021) $326M Shares Outstanding 226.3M $ Thousands Six Months ended June 30, 2021 Revenues $- Net loss $(49,067) Net cash used in operating activities $(37,194) Net cash provided by financing activities $43,060 Cash and cash equivalents* $5
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 6
Slide 7: Experienced Executive Leadership
Experienced Executive Leadership Name Title Prior Experience William (B.J.) Lehmann, JD Interim CEO, President & COO John Harrington, PhD EVP and CSO, Board member Ivor Macleod, MBA, CPA CFO Manal Morsy, MD, PhD SVP, Global Regulatory Affairs Maia Hansen, MBA, MS SVP, Operations and Supply Chain Jam
Slide 8: Overview: MultiStem (invimestrocel) Cell Therapy
Overview: MultiStem® (invimestrocel) Cell Therapy 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 Well Characte
Slide 9: 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 11 Cell Culture
Slide 12: Multimodal Biologic Product
Multimodal Biologic Product INFLAMMATION REDUCTION IMMUNOMODULATION (reparative mechanisms) NEUROPROTECTION CYTOPROTECTION ANGIO / VASCULOGENESIS CELLULAR REGENERATION REPLACEMENT 12
Slide 13: Development Focus in Japan
Development Focus in Japan HEALIOS K.K. Partnership & Clinical Program Overview Favorable commercial opportunity in Japan - Well-positioned for accelerated approval under Japanese regulatory framework for cell therapy products, allowing conditional or full approval after exploratory clinical trials
Slide 14: HEALIOS K.K. Partnership & Clinical Program Overview
Development Focus in Japan HEALIOS K.K. Partnership & Clinical Program Overview HEALIOS K.K. Partnership – Solid Foundation for MultiStem in Japan - Initial partnership established January 2016 and expanded in June 2018 - Development & commercialization rights for ischemic stroke, treatment of ARDS
Slide 15: Accelerated Approval System for Cell Therapy Products in Japan
Accelerated Approval System for Commercialization of Cell Therapy Products in Japan Traditional Approach New Framework 15
Slide 16: MultiStem Therapy for Ischemic Stroke
MultiStem Therapy for Ischemic Stroke 16
Slide 17: Potential for Cell Therapy in Ischemic Stroke
Potential for Cell Therapy in Ischemic Stroke Leading cause of serious disability and third leading cause of Ischemic Stroke Example: 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 18: MultiStem Could Meaningfully Extend the Treatment Window for Stroke Patients
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 19: Therapeutic MOAs of IV Administration of MultiStem for Stroke
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 20: MultiStem Stroke Clinical Trial: Results from MASTERS-1
MultiStem Stroke Clinical Trial Results from MASTERS-1 (Phase 2) MultiStem Administration for Stroke Treatment and Enhanced Recovery Study
Slide 21: 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. 21
Slide 22: 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 following ischemic stroke Key Eligibility Criteria – Original Design ̶ Cortical cerebral ischemic strokes with substantial, persistent deficits Cortical Stroke ̶ Randomized
Slide 23: How Excellent Outcome Was Measured in MASTERS-1
How Excellent Outcome Was Measured Using Three Standard Stroke Scales in the MASTERS-1 trial To be assigned an “Excellent Outcome”, patients need to receive excellent outcome scores IN ALL THREE standard stroke scales outlined below: NIHSS (NIH Stroke Scale) – Scoring 0 or 1 is considered an excelle
Slide 24: 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 25: Treatment w/ MultiStem Shows 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) ∆ at Day ∆ at Day Day 90 Day 365 90 365 MultiStem 15.4% 23.1% (n=65) 14.9%
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 between the initial 90-day recovery and one-year, compared to placebo period Excellent Outcome (%) Excellent Outcome
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: 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 – actively enrolling subjects) Intravenous administration of investigation product (MultiStem cell therapy or placebo) within 1
Slide 30: The Healios TREASURE study in Japan
The Healios TREASURE study in Japan Announced patient enrollment completed, August 2021 Treatment Evaluation of Acute Stroke for Using in Regenerative Cell Elements Objective: To evaluate the efficacy and safety of intravenous administration of HLCM051 (MultiStem) compared with placebo in subjects w
Slide 31: MultiStem Therapy for ARDS
MultiStem Therapy for ARDS 31
Slide 32: Treating Acute Respiratory Distress Syndrome (ARDS)
Treating Acute Respiratory Distress Syndrome (ARDS) Acute Respiratory Distress Syndrome (ARDS) afflicts approximately 500,000 patients in the United States, Europe and Japan combined annually – Multiple triggers for ARDS including viral pathogens, trauma, aspiration or other causes – Note: An outbre
Slide 33: Acute Respiratory Distress Syndrome (ARDS)
Acute Respiratory Distress Syndrome (ARDS) ARDS represents a major area of unmet medical need, with high mortality and morbidity, and typically requires extended intensive care hospitalization (e.g., ICU) and high intensity intervention (e.g., ventilation) Many ARDS patients will need to be placed i
Slide 34: Life After ARDS – For many, the struggles continue
Life After ARDS – For many, the struggles continue For the patients that survive ARDS, many have trouble getting back to functional independence, self care and the quality of life they once enjoyed Prolonged ventilation can cause pulmonary fibrosis/scarring, resulting in longer term health challenge
Slide 35: 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 36: MUST-ARDS Trial – Primary Results
MUST-ARDS Trial – Primary Results 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. Primary Endpoints: Safety and tolerability within 4 hours of MultiStem therapy administra
Slide 37: MUST-ARDS Trial – Primary Results Presented at ATS
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 M
Slide 38: 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): Rapid improvement observed among MultiStem treated subjects – Meaningfully higher probability of achieving ventilator independence
Slide 39: Why MultiStem is uniquely suited for Emergent Viral Outbreaks
Why MultiStem Cell Therapy is uniquely suited for Emergent Viral Outbreaks w/ Potential to Cause ARDS A range of recent outbreaks involving viral pathogens have shown the potential to induce ARDS (including SARS, MERS, H1N1 and COVID-19) MultiStem is not pathogen specific or dependent – it is believ
Slide 40: Pivotal Study Evaluating MultiStem to Treat Pathogen-induced ARDS
Pivotal Study Evaluating MultiStem Administration to Treat Pathogen-induced ARDS Phase 2/3 Trial Overview – Randomized, Double-Blind, Placebo-Controlled Trial Focused on Pulmonary Critical Care Sites in the United States (Authorized by FDA w/ Fast Track and RMAT designation) Intravenous administrati
Slide 41: MultiStem ARDS Clinical Trial in Japan
MultiStem ARDS Clinical Trial in Japan Topline Data from ONE-BRIDGE study by Partner HEALIOS K.K.
Slide 42: The Healios ONE-BRIDGE study in Japan
The Healios ONE-BRIDGE study in Japan Intravenous administration of investigational product (MultiStem cell therapy or placebo) following onset of acute respiratory distress syndrome (ARDS) Cohort 1 - 30 patient open-label, randomized study designed to evaluate the efficacy and safety of MultiStem (
Slide 43: Topline Results from Healios ONE-BRIDGE Study
Topline Results from Healios ONE-BRIDGE Study Cohort 1 Pneumonia-induced ARDS Patients MultiStem Standard Enrollment, N = 30 therapy (HLCM051 (MultiStem), n = 20; Primary Endpoint Standard therapy, n = 10) Ventilator-free days (VFD; the number of days out of 28 during which a ventilator 20 days 11 d
Slide 44: MultiStem Therapy for Traumatic Injuries
MultiStem Therapy for Traumatic Injuries 44
Slide 45: 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 46: Trauma Phase 2 Trial Objectives (MATRICS)
Trauma Phase 2 Trial Objectives (MATRICS) Multistem Administration for Trauma Related Inflammation and ComplicationS Currently enrolling a Phase 2 trauma trial with UTHealth at a leading Tier 1 Trauma Center in U.S. with funding provided by MTEC (Department of Defense) and the Memorial Hermann Found
Slide 47: Analyst Coverage
Analyst Coverage Firm Analyst Buy/Hold/Sell Bank of America Greg Harrison Buy SMBC Nikko Securities David Hoang Buy Needham and Co. Chad Messer Buy Dawson James Jason Kolbert Buy WBB Securities Stephen Brozak Buy 47
Slide 48: Priorities
Priorities 1. Advance our MultiStem (invimestrocel) cell therapy clinical programs, and work closely with our partner, Healios, to prepare for applications for marketing approval in Japan. • Ischemic stroke ‒ MASTERS-2 – enrollment ongoing ‒ TREASURE study (Healios) – enrollment complete August 2021