Slide 1: Advanced Critical Care With MultiStem Cell Therapy
Advanced Critical Care With MultiStem Cell Therapy Investor Overview
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: Table of Contents
Table of Contents 5 Corporate Overview 23 Acute Respiratory Distress Syndrome 9 Clinical Indications Summary 31 Mechanism of Action 12 Ischemic Stroke 37 Manufacturing 19 Trauma 3
Slide 4: Athersys Corporate Summary
Athersys Corporate Summary • Athersys is a clinical-stage biotech company focused on the next generation of therapies for critical health issues • A leader in the field of allogeneic cell therapy, which expands cells from healthy donors into a cellular therapy that acts to minimize inflammatory dama
Slide 5: MultiStem: Proprietary Cell Therapy Platform
MultiStem: Proprietary Cell Therapy Platform MultiStem® Cell Therapy Platform: Ethical, Versatile & Favorable Tolerability Adult bone marrow-derived stem cells Off-The-Shelf Product Allogenic, no tissue matching, IV administration of up to 1.2 billion cells per dose Scalability, Stability & Consiste
Slide 6: MultiStem Platform Advantages: Innovation From Donor to Patient
MultiStem Platform Advantages: Innovation From Donor to Patient • A leader in larger-scale manufacturing development • Over 10 years of successful GMP manufacturing with 170 batches and 1,100 doses produced • One healthy human donor can be expanded to hundreds of thousands of clinical & commercial d
Slide 7: Lead Indications
Lead Indications Ischemic Stroke ARDS Trauma 3 million non-fatal injuries per year 150,000 deaths, and ~2/3 of trauma Frequency ~800,000 strokes per year in the US 200,000 cases in the U.S. annually patients will experience SIRS (Systemic Inflammatory Response Syndrome) Up to 50% mortality rate Lead
Slide 8: Significant Clinical Work in Targeted Indications
Significant Clinical Work in Targeted Indications Phase 1 Phase 2 Phase 3 Phase 3 Pivotal Trial • Global • Fast Track / RMAT* / SPA* Designations • Interim Analysis Expected 3Q23 MASTERS-1 MASTERS-2 • Enrollment Completion Expected 2Q24 Ischemic Stroke • Data Readout Expected 2Q25 * RMAT: Regenerati
Slide 9: MultiStem Cell Therapy Platform: Opportunities Across Serious Unmet Need Indications
MultiStem® Cell Therapy Platform: Opportunities Across Serious Unmet Need Indications NONCLINICAL NONCLINICAL IND READY PHASE 3 Ischemic Stroke Hemorrhagic Stroke Parkinson's Disease Rheumatoid Arthritis NONCLINICAL Congestive Heart NONCLINICAL E IND READY PHASE Trauma Hypoxic Ischemia Alzheimer’s D
Slide 10: Strengthened & Experienced Leadership
Strengthened & Experienced Leadership Broad Experience in Pharmaceutical & Life Science Dan Camardo, MBA Maia Hansen, MBA Kasey Rosado Chief Executive Officer Chief Operations Officer Interim Chief Financial Officer Manal Morsy, MD, PhD, MBA Robert (Willie) Mays, PhD EVP, Regulatory Affairs EVP, Reg
Slide 11: Ischemic Stroke
Ischemic Stroke Overview Unmet Medical Need MultiStem Clinical Trials 11
Slide 12: Stroke Overview
Stroke Overview Impact Background High Unmet Need Only 30% of patients qualify for current standard of care (thrombolytics / mechanical thrombectomy) both of which have limited treatment windows and patient eligibility Ischemic Stroke Hemorrhagic Stroke Caused by leaking or High Burden on Healthcare
Slide 13: Unmet Medical Need in Stroke: Only Approved Ischemic Stroke Treatments
Unmet Medical Need in Stroke: Only 2 Approved Ischemic Stroke Treatments Thrombolytics Mechanical Thrombectomy MultiStem® Cell Therapy Mechanism of Removal of the clot Clot dissolving medications Modulation of the immune system Action using a catheter device Potentially applicable to 90 - 95% Only 1
Slide 14: Clinical Trials: MultiStem for Ischemic Stroke
Clinical Trials MultiStem for Ischemic Stroke MASTERS-1 TREASURE (Healios) MASTERS-2 Phase - # Subjects Phase 2 - 126 subjects Phase 2/3 - 206 subjects Phase 3 - 300 subjects Date Conducted 2010 - 2016 2017 - 2022 2018 - Present # Sites - Countries 33 - US, UK 48 - Japan 39 - US, UK, EU, Taiwan, Aus
Slide 15: MASTERS-1: Phase 2 Ischemic Stroke Trial Results
MASTERS-1: Phase 2 Ischemic Stroke Trial Results Treatment with MultiStem Shows Meaningful Benefit Proportion of Subjects Treated within 36 Hours Achieving Excellent Outcome Increases Over Time (Excellent Outcome = Patients Achieving NIHSS 0 or 1 and mRS 0 or 1, and Barthel Index >95) Excellent Outc
Slide 16: TREASURE Study by HEALIOS KK in Japan
TREASURE Study by HEALIOS KK in Japan Topline Data Announced May 2022 and Full Data in October 2022 Favorable results at one year in recovery measures Sakigake designation • Indicates achievement of functional independence 206 patients with moderate-to-moderate-severe • Reflects clinically relev
Slide 17: Ischemic Stroke (MASTERS-2) Ongoing Pivotal Phase 3 Study
Ischemic Stroke (MASTERS-2) Ongoing Pivotal Phase 3 Study Randomized, double-blind, placebo-controlled clinical trial, actively enrolling up to 300 patients in leading stroke centers in U.S. and internationally, under SPA agreement IV administration of 1.2 billion MultiStem® cells or placebo; based
Slide 18: Trauma
Trauma Overview MultiStem Clinical Trials 18
Slide 19: Trauma Overview
Trauma Overview Background Impact Trauma is the leading cause of death for people under the age of 45 and the leading cause of quality-of-life years lost In the US, trauma accounts for over 150,000 deaths and The economic cost of trauma amounts to $671 billion over 3 million non-fatal injuries per y
Slide 20: Trauma: Heterogeneity in Cause, Impact and Severity
Trauma: Heterogeneity in Cause, Impact and Severity Extensive Supporting Preclinical/Clinical Work • Acute pulmonary injury Initial traumatic injury can • Porcine models demonstrate improvement in lung function from treatment with result in SIRS related MultiStem complications such as: • Multipotent
Slide 21: MATRICS Ongoing Phase 2 Study
MATRICS Ongoing Phase 2 Study Randomized, double-blind, placebo-controlled clinical trial, evaluating MultiStem in 156 patients following resuscitation from hemorrhagic trauma Underway at The University of Texas Health Science Center at Houston (UTHealth Houston) and Memorial Hermann-Texas Medical C
Slide 22: Acute Respiratory Distress Syndrome (ARDS)
Acute Respiratory Distress Syndrome (ARDS) Overview MultiStem Clinical Trials Value Drivers 22
Slide 23: ARDS Overview
ARDS Overview Background Impact ARDS occurs when inflammation effects the lungs, often as a result of infection (such as COVID-19) or trauma No currently approved therapies for ARDS ~200,000 cases in the U.S. annually + ~2.2 M globally Extended, intensive hospitalized care in ICU, where most patient
Slide 24: Clinical Trials: MultiStem for ARDS
Clinical Trials MultiStem for ARDS MUST-ARDS ONE-BRIDGE Phase 3 Pending Phase - # Subjects Phase 1/2 - 36 subjects Phase 2 - 35 subjects Phase 3, pending, 80 Designation RMAT, Fast Track Orphan Designation Orphan Designation Date Conducted 2015 – 2019 2019 – 2021 TBD Countries US, UK Japan Japan • F
Slide 25: MUST-ARDS: Results from Exploratory Clinical Study in ARDS
MUST-ARDS: Results from Exploratory Clinical Study in ARDS All Subjects (in efficacy cohort) MultiStem Placebo Patients who rated complete independence Number of Patients 20 10 in self care at 1 year Ventilator-free days (median) 18.5 6.5 40% Placebo Mortality (d28) 25% 40% 80% MultiStem-treated Pat
Slide 26: ONE-BRIDGE: Healios study in Japan for the treatment of ARDS
ONE-BRIDGE: Healios study in Japan for the treatment of ARDS MultiStem-treated patients experienced: Cohort 1 - Pneumonia-induced ARDS Patients MultiStem SOC Number of Patients 20 10 Ventilator-free days (median) 20 11 82% Increase in Mortality (d90) 26% 43% Ventilator-free days Cohort 2 – COVID-19
Slide 27: Analysis of Pooled MUST-ARDS & ONE-BRIDGE Data
Analysis of Pooled MUST-ARDS & ONE-BRIDGE Data – Results Signal MultiStem Treatment Potential • 60 ARDS (non-COVID) patients – 40 receiving MultiStem treatment – 20 placebo or standard of care • Mostly pneumonia-induced ARDS (85% MultiStem-treated subjects; 90% placebo / SOC subjects) • MultiStem do
Slide 28: Substantial Value Drivers for Treating ARDS with MultiStem
Substantial Value Drivers for Treating ARDS with MultiStem Based on MUST-ARDS clinical data, MultiStem-treated patients have reduced number of days in the ICU, which is the most expensive area of the hospital for healthcare costs MultiStem-treated patients may be weaned off mechanical ventilators so
Slide 29: BARDA ARDS Phase 2 Trial Proposal
BARDA ARDS Phase 2 Trial Proposal BARDA Proposal Process: • May 26, 2023 – Submitted pre-submission inquiries • June 30, 2023 – Submitted final (revised) slide deck and other submission materials • July 10, 2023 – Notified as finalist by BARDA • July 24-28, 2023 – Just Breathe – An ARDS Therapeutics
Slide 30: Mechanism of Action
Mechanism of Action Overview of data from pre-clinical and clinical understanding of mechanism of action 30
Slide 31: MultiStem Overview
MultiStem® Overview Our data show that early intervention with MultiStem therapy after an acute injury enhances healing by regulating an overactive immune response and re-establishing homeostasis. Fundamentally shifts recovery trajectory & clinical outcomes, MultiStem expresses supports preservation
Slide 32: Key Events and Therapeutic Mechanism of Action of MultiStem following Ischemic Stroke
Key Events and Therapeutic Mechanism of Action of MultiStem following Ischemic Stroke Ischemic Stroke Ischemic stroke occurs when a blood clot blocks an 1 artery leading to the brain, resulting in a corresponding loss of neurologic function. Inflammation after stroke can lead to greater tissue 2 los
Slide 33: Multimodal Mechanism of Action: A Living and Dynamic Product
Multimodal Mechanism of Action: A Living and Dynamic Product Capable of Interaction with Multiple Cell Types 33
Slide 34: Consistent Biomarker Impact Observed in Preclinical and Clinical Studies
Consistent Biomarker Impact Observed in Preclinical and Clinical Studies Reflect Mechanism of Action Reduction in acute inflammatory biomarkers from MultiStem treatment observed in ARDS and Ischemic Stroke patients, consistent with previously published preclinical data
Slide 35: Manufacturing
Manufacturing Patented Platform Technology Global Leader Manufacturing Process 35
Slide 36: Expansion Capabilities of MultiStem
Expansion Capabilities of MultiStem • MultiStem is cell therapy based on MAPC® technology that can double rapidly in culture and have robust expansion capabilities, beyond other bone marrow- derived cell therapies, as seen in the chart to the left comparing MultiStem to MSCs • Hundreds of thousands
Slide 37: Scalable Manufacturing Process
Scalable Manufacturing Process 15 Years of Production Experience and Advancements in Cell Therapy: • Proven expertise in efficient, high yielding and innovative processes • Establishment of an essentially closed manufacturing process - unique characteristic in the Cell & Gene Therapy industry • Adva
Slide 38: Thank you!
Thank you! 38