Biotech Showcase January 13, Mike Bruch, CFO Dalip Sethi, Ph.D. Sr. Clinical Scientist

Transcription

1 Biotech Showcase January 13, 2016 Mike Bruch, CFO Dalip Sethi, Ph.D. Sr. Clinical Scientist

2 Forward Looking Statement This presentation contains forward-looking statements. These statements involve risks and uncertainties that could cause actual outcomes to differ materially from those contemplated by the forward-looking statements. A more complete description of risks that could cause actual events to differ from the outcomes predicted by our forward-looking statements is set forth under the caption "Risk Factors" in Cesca Therapeutics annual report on Form 10-K and other reports we file with the Securities and Exchange Commission from time to time, and you should consider each of those factors when evaluating the forward-looking statements. 2 NASDAQ: KOOL

3 Company Background Ticker Symbol NASDAQ: KOOL Share Price $0.16 Shares Outstanding 42.8M Market Capitalization $7M Revenue (TTM) Cash Current Liabilities Total Equity Corporate Headquarters Clinical Research (GLP Labs) India Operations (CRO & GMP Labs) $15.2M $6.6M $8.9M $32.3M Rancho Cordova, CA Emeryville, CA Gurgaon, India Stock information and market capitalization as of January 7, 2016 Financials as of September 30, NASDAQ: KOOL

4 Cesca Therapeutics In A Nutshell We Develop and Market Integrated Cellular Therapies and Delivery Systems that Advance the Safe and Effective Practice of Regenerative Medicine. Broadly Adaptable Technology Platform with Multiple Applications Highly Leverageable Large and Growing Market Opportunities $16 Billion+ Addressable Market Focus on Critical Unmet Medical Needs Ischemic Cardiovascular Indications Bone Marrow Transplantation Orthopedic Protocols to Follow 2 Platforms for Growth with Compelling Competitive Advantages Cesca s SurgWerks TM & CellWerks TM System Unique Point-of-Care Approach Highly Resource Efficient Operating Model (Fortis) Relatively Low Regulatory Bar - IDE Pathway Highly Compelling Initial Clinical Data CLI Phase III Pivotal Approved by FDA AMI Phase II Approved by IRB Strong Team with Decades of Development and Commercial Experience Depressed Valuation and Numerous Near Term Value Creation Opportunities 4 NASDAQ: KOOL

5 Board Members and Advisors Craig W. Moore Chairman Former Director at NxStage Former Chairman and CEO at Everest Healthcare Services Corporation Held senior management positions at American Hospital Supply / Baxter Healthcare Former Director at Biologic System Corporation Mahendra Rao, MD, PhD Director Vice President at New York Stem Cell Foundation Former Director at the NIH Center for Regenerative Medicine Former Vice President of Regenerative Medicine at Life Technologies, Inc. Former Chief of Neurosciences at the National Institute of Aging Denis Rhein Director 30+ years of corporate banking and securities experience at Deutsche Bank AG Founder and Director at EMR Vermögensverwaltung GmbH Founder and Director at Illumisound GmbH Robin Stracey Director & CEO 30+ years of life science and healthcare company experience, including DuPont, Dade Behring (Siemens Healthcare) and Thermo Fisher Scientific Former CEO of Applied Imaging (now Danaher Corporation), Cantimer and Integrated Fluidics Sol Hamburg, MD, PhD Advisor Graduate of the New York University School of Medicine. Board certified by the American Board of Internal Medicine in hematology, medical oncology, and internal medicine. Currently serves as a clinical professor of medicine at the UCLA School of Medicine and as the CEO of the Tower Cancer Research Foundation. Rick Powell, MD Advisor Section Chief of Vascular Surgery and Professor of Surgery and Radiology at Geisel School of Medicine Former Assistant Professor of Surgery at Yale Medical School Member of the Dartmouth-Hitchcock and Dartmouth-Hitchcock Health Boards of Trustees and Vice Chair of the Board of Trustees of The Hitchcock Foundation National or International Principle Investigator on multiple Gene Therapy and Stem Cell Therapy clinical trials Joe Mills, MD Advisor Chief of the Division of Vascular Surgery & Endovascular Therapy at the Baylor College of Medicine Professor and chief of Vascular Surgery at the University of Arizona College of Medicine Co-editor of the prestigious "Rutherford's Vascular Surgery" (7th and 8th editions), the go-to textbook for vascular surgery Author of nearly 300 peer-reviewed journal articles and book chapters, and the principal investigator for over 40 clinical trials 5 NASDAQ: KOOL

6 Large and Growing Market Opportunities CARDIOVASCULAR THERAPEUTIC INDICATION PRE-CLINICAL/PILOT PHASE FEASIBILITY PIVOTAL REG TYPE MARKET SIZE SurgWerks Point of Care Platform Critical Limb Ischemia (Intra-arterial) 13 Human Subjects Discontinued for IM Delivery IDE/PMA Critical Limb Ischemia (Intra-muscular) 17 Human Subjects IDE/PMA $2.0B Acute Myocardial Infarction (STEMI) 1 Human Pilot Subject India Phase II under App. Prep U.S. Pivotal Proceeding IDE/PMA $0.7B Non-Healing Ulcers 10 Human Pilot Subjects 501(k) $0.8B Ischemic Stroke IDE/MPA $5.2B ORTHOPEDICS SurgWerks Point of Care Platform Spinal Fusion 15 Human Pilot Subjects IDE/PMA $0.8B Osteoarthritis 50 Human Pilot Subjects IDE/PMA $5.0B Non-Union Fractures 49 Human Subjects IDE/PMA $1.1B Avascular Necrosis 10 Human Pilot Subjects IDE $0.8B HAEM/ONC CellWerks Laboratory Platform Bone Marrow Transplant 6 Human Pilot Subjects Haplo & ABO Pilot Underway 501(k) $0.1B Cesca has several potentially blockbuster therapies in development, collectively targeting over $16B in addressable market opportunity. $16B 6 NASDAQ: KOOL

7 Focus on Critical Unmet Medical Needs Clinical Targets Regulatory Pathway Ischemic Cardiovascular Disease Critical Limb Ischemia Myocardial Infarction Hematopoietic Stem Cell Therapy (BMT) ABO Mismatch Haplo-identical SurgWerks & VXP CellWerks IDE/PMA Intra-Operative Therapies ~ $3B Market Opportunity 510(k) Laboratory-Based Processing 7 NASDAQ: KOOL

8 Focus on Critical Unmet Medical Needs Critical Limb Ischemia Most Serious Form of Peripheral Artery Disease Estimated Incidence of approx. 1 Million in the U.S., Fueled by the Epidemic of Diabetes and the Aging Population Results in ~70,000 Major Amputations and 134,000 Minor Amputations per annum in the U.S. Dismal Patient Outcomes Associated with Amputation Cesca Target Population Age 40-85; Rutherford Category 5; No Option (i.e. Exhausted or are at High Risk for Surgical or Endovascular Options) Acute Myocardial Infarction Leading Cause of Death in the United States and most Industrialized Nations Despite Improvements in Pharmacological Treatments, 1 Year Mortality is still ~13% Results in ~450,000 Deaths per annum in the U.S. Cesca Target Population Age 18-75; Experienced a ST-Elevated Myocardial Infarction with a Low Left Ventricle Ejection Fraction of <40% at 72 Hours post Percutaneous Coronary Intervention 8 NASDAQ: KOOL

9 SurgWerks TM Point of Care Platform SurgWerks TM CLI Kit Capital Equipment VXP System Single Use Consumable Kit Compact Hematology Analyzer Compact Cell Analyzer Custom Designed Benchtop Centrifuge Laptop & XpressTRAK Software VXP System Intra-Operative Cart 9 NASDAQ: KOOL Bone Marrow Aspiration Needles Processing and Collection Bag-Set Syringes, Transfer Tubes, Connectors (for Fluid Dispensing) Therapeutic Infusion Needles SafetyGlide Needles etc. Together SurgWerks TM & VXP System offer an integrated offering of optimized components that enables the aspiration, processing, QC and re-injection of autologous stem cells in a single minute procedure within the Operating Room.

10 Technology Advantage - VXP The VXP System delivers automated, rapid, smart-vision based cell enrichment and ensures consistency and reliability. 10 NASDAQ: KOOL

11 Unique Point Of Care Approach SurgWerks TM & VXP System Indication Specific Integrated Offering for the Operating Room (Aspiration; Processing; QC Diagnostics & Delivery) Safe and Fast Autologous Bone Marrow Derived Stem Cells Single Minute Patient Procedure Consistent High Cell Counts and Real Time QC Feedback High Cell Dose Accuracy and Potency Targeted and Optimized Cell Delivery 600 Patients Treated So Far Across Eight Clinical Indications Cesca s SurgWerks TM & VXP System has the potential to revolutionize how autologous cell therapies are conducted. 11 NASDAQ: KOOL

12 Cesca s Partnership With Fortis CESCA is the Exclusive Provider of Regenerative Medicine Services to Fortis Over 70 Hospitals in 5 Countries >10,000 Inpatient Beds >15,000 Outpatients per day Highly Experienced Clinical Research Staff World Class Facilities and Equipment US FDA Registered, ISO Certified and India Licensed Fortis Escorts Heart Institute Ranked #1 in India Cardiovascular Medicine in 2013 Fortis Memorial Research Institute Ranked #2 on List of 30 Most Advanced Hospitals in the World Top Masters In Healthcare Cesca is the Exclusive Provider of Regenerative Medicine Services to Fortis The Fortis relationship provides Cesca with a highly resource-efficient operating model. Rx Clinical Trial Advantage (Saving $M) Pilot P1/P1b Total Cesca Investment $2 $7 $9 US Equivalent Investment $17 $28 $45 Non-Dilutive Clinical Trial Funding Benefit $36 12 NASDAQ: KOOL

13 Regulatory Advantage of IDE/PMA Path Regulatory Risk IND/NDA or BLA Allogeneic Cells Manipulated Cells Homologous or Non-Homologous 3 Phase Trial Larger Trial Patient Population Higher FDA Marketing Approval Threshold HIGH IDE/PMA Autologous Cells Minimally Manipulated Cells Homologous or Non-Homologous 2 Phase Trial Smaller Trial Patient Population Lower FDA Market Approval Threshold LOW IND: Investigational New Drug BLA: Biologics License Application NDA: New Drug Application IDE: Investigational Device Exemption PMA: Pre Market Approval 13 NASDAQ: KOOL

14 Clinical Results Cardiovascular (CLI) Feasibility Trial Results Major Revascularization No Adverse Events Related to Treatment Major Amputation-Free Rate Post- SurgWerks TM Therapy of 82.4% in No- Option Patient Cohort Verified Revascularization, with Improvements in: Collateral Vessel Numbers & Size Ankle Brachial Index (ABI) Transcutaneous Oxygen Pressure (TcPO2) Reduction in pain Claudication Pain Reduced by 97% Rest Pain Reduced by 100% Significant Improvement in 6 min Walk Test The results of Cesca s feasibility trial involving 17 no option CLI patients were highly compelling. Day 0 Day 365 Trial Design N = 17; all no-option patients with near-term leg amputation recommendations 12 month follow-up data Distal Thigh (P=0.01) Proximal Leg (P=0.03) 14 NASDAQ: KOOL

15 Design for CLI Pivotal Trial Approved by FDA Pivotal Trial Design Endpoints Randomized Double Blinded Placebo Controlled 12 Month Follow Up Subjects = 224 (adaptive design up to 312) 204 Subjects approved in the US 1 20 Subjects approved OUS 1 Up to 60 Clinical Trial Sites 1 Blinded Independent Review Committee 1 3:1 Randomization 1 Primary: Major Amputation Free Survival Secondary: Time to Treatment Failure Wound Healing Skin Perfusion Pressure (SPP) Quality of Life (QoL) Ankle Brachial Index (ABI) Pain Study Principal Investigator: Dr. Richard Powell, M.D. Chief, Vascular Surgery Dartmouth 15 NASDAQ: KOOL 1 U.S. FDA approval allowance for the pivotal IDE trial

16 Clinical Results Cardiovascular (AMI) Pilot Trial Results Cardiac Tissue Repair LVEF improvement = from 36% to 60% Stroke volume improvement = from 39.7cc to 80cc Scar remains 11% of total heart mass Normal life resumed N=1 patient (24 Month Follow Up) 16 NASDAQ: KOOL Although only one patient was treated, the results of Cesca s pilot study were very promising. The feasibility trial is now IRB approved and is expected to begin following India DCGI approval.

17 Anticipated Study Design for AMI Phase II Trial Design Randomized Active-Control 6 Month Safety Follow Up Subjects = 40 Multi-Center Study 1:1 Randomized Study Principal Investigator: Dr. Ashok Seth, M.D., F.R.C.P. Endpoints Primary: Major Adverse Cardiac Events (Safety) Secondary: Left Ventricular Ejection Fraction (LVEF) Left Ventricular Volumes, End- Diastolic Volume (LVEDV) Infarct Size Myocardial Mass Myocardium Viability Quality of Life 17 NASDAQ: KOOL

18 CellWerks TM Bone Marrow Transplant Program ABO Mismatch Program CellWerks TM Medical Device Model Precision Purification of Bone Marrow Aspirate for Transplantation Global Opportunity; Focus on Pediatrics Straightforward 510(k) Regulatory Pathway Haplo-Identical Program Faster, Lower Cost, Higher Cell Recovery Fortis Patient Services Model India Only Focus on Patients with Genetic or Hemato-Oncologic Disorders Addresses Lack of Suitable Matches for Bone Marrow Transplantation Cesca s automated CellWerks TM system takes the lab-based preparation of cells for bone marrow transplantation to a new level. 18 NASDAQ: KOOL

19 Major Clinical Milestones CLI Milestones Q4 15 H1 16 H FDA IDE/PMA Pivotal Trial Amended (resubmission) Approved First Patient Enrolled Interim Analysis Pivotal Phase Completed CLI PMA Submission (commercialization dependent on US FDA) BMT Milestones MXP Intra-Operative Device Approval India Approved & Completed Commercial and Academic Collaboration Announcements ABO Mismatch FDA 510(K) Submission Haplo Pilot Phase Trial Data Published Haplo India DCGI Submission for Marketing AMI Milestones IRB Approval of Trial Design Approved & Completed DCGI Phase II Trial Application Phase II First Patient Treated 19 NASDAQ: KOOL

20 Value Appreciation Potential Peer Group Snapshot Cell Therapy Peer Company (in millions except share price amounts) TCKR Market Cap Share Price Revenue Market Capitalization and Share Price as of January 7, 2016 Revenues for quarter ended September 30, NASDAQ: KOOL Vericel Corporation (Aastrom) VCEL $55 $2.28 $11.0 Athersys Inc. ATHX $104 $1.03 $0.4 Nuo Therapeutics Inc. (Cytomedix) NUOT $6 $0.05 $2.0 Cytori Therapeutics Inc. CYTX $26 $0.16 $2.0 Caladrius BioSciences (NeoStem Inc.) CLBS $24 $0.70 $6.0 Osiris Therapeutics, Inc. OSIR $344 $10.40 $25.0 Pluristem Therapeutics Inc. PSTI $80 $1.01 $0.1 MiMedx Group Inc. MDXG $941 $8.74 $49.0 Capricor Therapeutics, Inc. CAPR $39 $2.40 $1.0 Average Market Cap $180 Cesca Therapeutics Inc. KOOL $7 $0.16 $3.0

21 Summary Numerous Near-Term Value Creation Opportunities Broadly Capable Technology Platform Excellent Initial Clinical Data Large and Growing Market Opportunities ($16B+) Compelling Competitive Advantages Focus on Significant Unmet Medical Needs 21 NASDAQ: KOOL

22 Thank you Contact: Cesca Therapeutics Inc. Investor Contact: Kirin Smith, ProActive Capital Group , or ir@cescatherapeutics.com

23 23 NASDAQ: KOOL