Athersys, Inc. · OTC:ATHXQ
Athersys — Investor Presentation (July 2022)
48 slides from Athersys's investor presentation, Jul 13, 2022
Slide 1: Changing the Future of Medicine
Changing the Future of Medicine Athersys Corporate Presentation NASDAQ: ATHX www.Athersys.com
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: We Are Changing the Future of Medicine
We Are Changing the Future of Medicine • How? Early intervention with MultiStem® therapy following serious ischemic, traumatic and infectious conditions enhances healing by regulating an overactive immune response and re-establishing homeostasis. • Built on exceptional science, we are innovators cha
Slide 4: Advanced Portfolio Targeting Areas of Substantial Unmet Need
Advanced Portfolio Targeting Areas of Substantial Unmet Need KEY: = Enrolling; in progress = Completed enrollment / study Indication Clinical Trial Phase 2 Phase 3 BLA Regulatory Target Market • Fast Track designation MASTERS-1 • RMAT designation • ~800K annual cases in the U.S. • Special Protocol A
Slide 5: Managed by an Exceptional Team with Broad Experience
Managed by an Exceptional Team with Broad Experience in Pharmaceutical and Life Science Industries Name Title Prior Experience Daniel Camardo, MBA CEO, Board member Maia Hansen, MBA, MS COO EVP, Head of Regenerative Medicine & Robert W. Mays, PhD Head of Neuroscience EVP, Head of Global Manal Morsy,
Slide 6: Scientific Advisory Board – Neurology Key Opinion Leaders
Scientific Advisory Board – Neurology Key Opinion Leaders David Chiu, MD, FAHA S. Thomas Carmichael, MD, PhD Professor and Elizabeth Blanton Wareing Chair Professor and Chair in the Eddy Scurlock Stroke Center Frances Stark Chair Houston Methodist Hospital, Department of Neurology Weill Cornell Medi
Slide 7: Strong Foundation in Science Has Created an Immense Opportunity
Strong Foundation in Science Has Created an Immense Opportunity Built on We have an Our platform is We are EXCELLENT SCIENCE INNOVATIVE THERAPY ROBUST AND DIVERSE LEADING THE WAY - with dozens of with strong intellectual with broad applicability – in innovating cell publications - providing property
Slide 8: Unique Attributes of MultiStem Cell Therapy Provide a Competitive Edge
Unique Attributes of MultiStem Cell Therapy (invimestrocel) Provide a Competitive Edge Promotes healing and tissue repair Off-the-shelf and easy administration By regulating the overactive immune response after an MultiStem is easily administered intravenously or injury, our therapy can potentially
Slide 9: Multipotent Adult Progenitor Cells (MAPC) are a Distinct Type of Adult Stem Cell
Multipotent Adult Progenitor Cells (MAPC) are a Distinct Type of Adult Stem Cell • There is a growing body of evidence supporting the promise of stem cell therapy. • MAPC are not embryonic stem cells, they are derived from healthy adult bone marrow. • There are many different types of stem cells, MS
Slide 10: MAPCs are a Preferred Type of Stem Cell
MAPCs are a Preferred Type of Stem Cell - Giving Us a Unique Opportunity MultiStem® Robust scalability with ability to serve large commercial markets Ethical source of stem cells, derived from healthy volunteers > 15 years of preclinical and clinical data demonstrating tolerability and efficacy Broa
Slide 11: Objective: Dramatically Improve the Recovery Path
Objective: Dramatically Improve the Recovery Path 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. 4. MultiStem cell Supports preservation of therapy intervention life and
Slide 12: MASTERS-1: Impact Across Outcome Measures
MASTERS-1: Impact Across Outcome Measures Patients who received treatment ≤ 36 hours after stroke onset Placebo group (n=61) MultiStem treatment group (n=31) p = 0.03* p < 0.01* 25% p = 0.09 10% p = 0.14 29% 32% Day 90 % Patients - Death or life- 16.1% 13% threatening adverse events 6.6% 8.2% (60% d
Slide 13: Supporting Data from our Completed Phase 2 ARDS Study (MUST-ARDS)
Supporting Data from our Completed Phase 2 ARDS Study (MUST-ARDS) Placebo / SOC group (n=10) MultiStem treatment group (n=20) Patients alive and off ventilator by day 7 20% Placebo 40% MultiStem-treated 18.5 45% days 25% 6.5 days Day 28 Day 28 27% Increase in ICU-free days in MultiStem-treated group
Slide 14: Multimodal Mechanism of Action: Interaction with Multiple Cells of the Immune System
Multimodal Mechanism of Action: Interaction with Multiple Cells of the Immune System MultiStem® M1 M2 Regulatory Endothelial cells Neutrophils T cells Macrophages Macrophages T cells Promotes MΦ2: Reduces Neutrophil: Promotes Treg: • M2 Phenotype • Infiltration • Differentiation • Anti-inflammatory
Slide 15: Deep Understanding of Therapeutic MOAs of IV Administration of MultiStem for Stroke
Deep Understanding of Therapeutic MOAs of IV Administration of MultiStem for Stroke MultiStem works through regulation of Ischemic Stroke multiple factors and pathways important to brain recovery following a stroke. Inflammation after stroke leads to greater tissue loss 1 and scarring in the brain.
Slide 16: Spleen Role in Inflammatory Damage in the Brain following Stroke
Spleen 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 occurs in the brain - but creates permanent immunological impairment. Fluoro-Jade Stain
Slide 17: Expansive Therapeutic Opportunity
Expansive Therapeutic Opportunity : Over 17 Years of Preclinical and Clinical Research Publications Years of preclinical research and data support mechanism of action hypothesis and demonstrates that MultiStem has the potential to treat a variety of clinical indications with unmet need. • Ischemic S
Slide 18: Partnerships, Collaborations and Funding Organizations Over the Years
Partnerships, Collaborations and Funding Organizations Over the Years Corporate Partnerships Collaborations Funding Organizations
Slide 19: Scalable Manufacturing Platform Supports Production to Serve Large Markets
Scalable Manufacturing Platform Supports Production to Serve Large Markets Favorable Biology Scalable Production Platforms Practical Storage & Distribution • MultiStem is developed from a • Cells are expandable in bioreactors, as • Demonstrated product stability – long special class of MAPC® that we
Slide 20: Proprietary Manufacturing Process with Exponential Expansion Capabilities
Proprietary Manufacturing Process with Exponential Expansion Capabilities • One healthy human donor can be expanded to hundreds of thousands of clinical/commercial doses. • Robust intellectual property covering a defined manufacturing process. One Donor → Master and Working Cell Banks → Expanded in
Slide 21: Distinctive, Efficient, and Scalable Technologies: Bioreactor and SIFU
Distinctive, Efficient, and Scalable Technologies Bioreactor SIFU™ “Secure Integrated Freezer Unit” • Development of proprietary cryogenic storage system known as SIFU™ Technology • Enables transformational commercial scale at lower cost • Simplifies product preparation to a single step after order
Slide 22: Current MultiStem Clinical Trials
Current MultiStem Clinical Trials MASTERS-2 [PHASE 3] for Ischemic Stroke Most advanced program is in Phase 3 for treatment of ischemic stroke (with Fast Track and RMAT designations) and is being conducted under a Special Protocol Assessment (SPA) from the FDA. MACOVIA [PHASE 2/3] for Acute Respirat
Slide 23: Large Market Opportunities in Lead Indications
Large Market Opportunities in Lead Indications Targeted Market – Full Market Size Value Creation current - potential development • Reduced hospital stays, mortality, • Moderate to moderate-severe strokes • ~800,000 new cases in U.S. annually rehabilitation Ischemic • ~2.2 million annually in U.S., E
Slide 24: Ischemic Stroke
Ischemic Stroke 24
Slide 25: Stroke Facts
Stroke Facts Ischemic Stroke Hemorrhagic Stroke A stroke occurs when the blood supply is interrupted or reduced to part of the brain. This prevents brain cells Caused by leaking or Caused by a blocked artery from getting oxygen and nutrients, leading to cell death bursting of a blood vessel and tiss
Slide 26: Over 15 Years of Research Supporting our Stroke Program
Over 15 Years of Research Supporting our Stroke Program Time window is based on Our pursuit of developing a therapy for ischemic stroke is learnings from MASTERS-1, based on years of preclinical and clinical research and now 18 - 36 hours many peer-reviewed scientific publications Time window was Ph
Slide 27: 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 28: MASTERS-1: Clinical Improvement Observed Across the Severity Spectrum
MASTERS-1: Clinical Improvement Observed Across the Severity Spectrum Patients who received treatment ≤ 36 hours after stroke onset Distribution of Subjects by Group using mRS scores – mRS "shift" analysis – over time 0 1 2 3 4 5/6 0 1 2 3 4 5/6 3.2% 3.2% 9.7% 22.6% 16.1% 38.7% 9.7% MultiStem 12.9%
Slide 29: MASTERS-2: Current Pivotal Phase 3 Clinical Study in Ischemic Stroke
MASTERS-2: Current Pivotal Phase 3 Clinical Study in Ischemic Stroke • Randomized, double-blind, placebo-controlled clinical trial, actively enrolling up to 300 patients in leading stroke centers in U.S. and Internationally • IV Administration of 1.2 billion MultiStem cells or placebo; Set up for su
Slide 30: TREASURE Study by HEALIOS KK in Japan
TREASURE Study by HEALIOS KK in Japan Topline data announced May 2022 • TREASURE study conducted in Japan • Sakigake designation • 206 subjects: 104 MultiStem-treated; 102 placebo • 48 sites in Japan involved in the trial • Moderate-to-moderate-Severe strokes (NIHSS 8-20 at baseline) • Patients get
Slide 31: Key TREASURE Results
Key TREASURE Results Favorable results from treatment in Good Recovery measures (indicating achievement of functional independence) at one year, reflecting slower and less robust expected recovery for this population One Year MultiStem Placebo p-value* Excellent Outcome 15.4% 10.8% n.s. Global Recov
Slide 32: TREASURE: MultiStem Improvement Over Time Relative to Placebo
TREASURE: MultiStem Improvement Over Time Relative to Placebo Proportion of Patients Achieving mRS <=2 and Barthel Index >=95 mRS <=2 Barthel Index >=95 50 50 45 45 40 40 * 35 35 Proportion (%) Proportion (%) 30 30 25 25 20 20 15 15 10 10 5 5 0 0 7 30 90 365 7 30 90 365 Days from Stroke Days from St
Slide 33: Summary of Key Findings
Summary of Key Findings • Less impact in this older population on achievement of “excellent or ideal” health status post-stroke: primary outcome – Excellent Outcome (NIHSS<=1, mRS<=1 and Barthel Index >=95) – not significant at day 90 and one year • Evidence of improvement in functional “independenc
Slide 34: TREASURE Read-through to MASTERS-2
TREASURE Read-through to MASTERS-2 Patient Age – Important Factor Related to Treatment Benefit and represents major expected difference between TREASURE and MASTERS-2 Box = middle quartiles Limited concentration of Oldest in MASTERS-2 • TREASURE Representative patient (<80-years) results most releva
Slide 35: Read-through: Improvement from MultiStem-treatment in Representative Patient Population
Read-through: Improvement from MultiStem-treatment in Representative Patient Population from TREASURE Improvement in measures of functional independence and good recovery 90 Days One Year MultiStem Placebo MultiStem Placebo mRS Shift Odds Ratio=1.7, p=0.13 Odds Ratio=1.8, p=0.06 (MASTERS-2 primary)
Slide 36: Substantial Value Drivers for Treating Stroke with MultiStem
Substantial Value Drivers for Treating Stroke with MultiStem Based on the MASTERS-1 data, MultiStem-treated patients are likely to experience: • Reduced hospital stays, reduced mortality, reduced/lower intensity rehabilitation, and reduced institutionalization. • Lower rates of disability, contribut
Slide 37: Key Takeaways from TREASURE data
Key Takeaways from TREASURE data • TREASURE results confirm key hypotheses and design of MASTERS-2 study, even though Primary Endpoint not significant • Results from TREASURE patients most representative for MASTERS-2 suggest high potential for success (primary outcome mRS shift) for MASTERS-2 study
Slide 38: Acute Respiratory Distress Syndrome (ARDS)
Acute Respiratory Distress Syndrome (ARDS) 38
Slide 39: ARDS: Acute Respiratory Distress Syndrome
ARDS: Acute Respiratory Distress Syndrome About + Statistics Implications Treatment + Life After ARDS • Occurs when our lungs are • No currently approved treatment for ARDS • Most patients placed on mechanical severely injured, often by ventilation to help them breathe, often infection or trauma • 3
Slide 40: 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 41: 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 Mortality (d90) 26% 43% 82% Increase in Ventilator-free Cohort 2 – COVID-19 Induc
Slide 42: 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 43: Substantial Value Drivers for Treating ARDS with MultiStem
Substantial Value Drivers for Treating ARDS with MultiStem • Based on our MUST-ARDS 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 of mechanical ventilators
Slide 44: Serious Trauma
Serious Trauma: Blunt force injury to the body, such as falls, motor vehicle accidents, or any type of penetrating injury 44
Slide 45: Trauma: Heterogeneity in Cause, Impact and Severity
Trauma: Heterogeneity in Cause, Impact and Severity Extensive Relevant Preclinical/Clinical Work Pathophysiology • Acute pulmonary injury • Porcine models demonstrate improvement in lung function from treatment with MultiStem • Multipotent adult progenitor cells decrease cold ischemic injury in ex v
Slide 46: MATRICS: Phase 2 Clinical Study in Trauma
MATRICS: Phase 2 Clinical Study in Trauma • Randomized, double-blind, placebo-controlled Phase 2 clinical trial • First of its kind – working with UTHealth on this single center trauma trial being conducted at a Memorial Hermann Hospital, a leading Tier 1 Trauma Center in Houston • Actively enrollin
Slide 47: Progress & Important Upcoming Milestones
Progress & Important Upcoming Milestones Goal/Objective Progress Upcoming Milestones • >50 publications by Athersys and collaborating scientists • Recent important articles re: MOA (e.g., Treg, MUST-ARDS) • Upcoming Pivotal Readouts Therapeutic Validation • 12 clinical studies and strong evidence of
Slide 48: Thank you!
Thank you! 48