Corporate Presentation World Stem Cell Summit, San Antonio, Texas December 3, 2014 Rev. 1214
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Forward Looking Statement
This presentation contains forward-looking statements. Such forward-looking statements
include but are not limited to that Cesca Therapeutics Inc. will provide unmatched world-
class capability and service to its clients. 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.
Contact:
Cesca Therapeutics Inc.
http://www.cescatherapeutics.com
Investor Contact: Kirin Smith, ProActive Capital Group
+ 1-646-863-6519, or [email protected]
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Cesca TherapeuticsCorporate Snapshot
NASDAQ Symbol KOOL
Market Capitalization $44.7 Million
Shares Outstanding 40.3 Million
Options Outstanding 2.2 Million
Warrants Outstanding 5.1 Million
Stock Price $1.11
Average Trading Volume 71,697
Revenue (TTM) $16 Million
Total Assets $59 Million
Debt None
Corporate Headquarters Rancho Cordova, CA
Clinical Research (GLP Labs) Emeryville, CA
India Operations (CRO & GMP Labs) Gurgaon, India
Stock price and market cap as of November 26, 2014
Financials as of September 30, 2014
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Cesca TherapeuticsAn integrated regenerative medicine company in late stage clinical trial
Completely Integrated Regenerative Medicine Company
− Big R: Biological and Engineering Development Teams
− CRO: Clinical Research Organization
− Commercial: Currently Manufactures & Sells > 150,000 devices per annum
Strong I.P. in Cell Therapy Devices & Treatment Methods
− Devices; patented platform technology (> 40 issued patents)
− Methods; 3 patent pending methods for CLI, AMI and General Ischemia
First Commercially Viable Autologous Cell Therapy
− Late stage (pivotal) clinical trial for Critical Limb Ischemia
− Safe & Effective; in the regulatory “sweet spot”
− Rapid; 60-90 minute bedside protocol
− Low cost delivery (no external GMP manufacturing or cold supply chain)
Opportunity to Create Substantial Stockholder Value
− Multiple therapies - 8 pilot & Phase 1b clinical trials
− M&A/bolt on opportunities to help us further fill out our tools capabilities
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Fortis PartnershipCRO embedded in New Delhi state-of-the-art tertiary care hospital
Cesca is exclusive regenerative medicine
provider to Fortis
• 72 hospitals (6 countries)
• 10,000 inpatient beds
• 15,000 outpatients per day
• Experienced clinical research staff
• 2x as many sites as Kaiser
Physician/patient access
World class clinical facilities and equipment
Lobby partner with government
Embedded CRO Benefits
• Only cell therapy specialty CRO worldwide
• US FDA registered; FDA accepted foreign trials
• Over 600 patients treated
• Better Control over trial management
• Speed to completion
• 1/5 cost of US/ Europe patient related clinical
trials
Rx Clinical Trial Advantage ($M) Pilot P1/P1b Total
Cesca Investment $2 $7 $9
US Equivalent Investment $17 $28 $45
Non-Dilutive Clinical Trial Funding Benefit $36
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Cesca Regulatory AdvantageLower risk = speed to market
• IND/BLA• 3 Phase Trial• Higher Trial Patient Pop
• IDE/PMA• 2 Phase Trial• Lower Trial Patient Pop
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Cesca SurgWerksTM
Indication Specific POC Process Control
SurgWerks POC Advantages:
• Integrated Optimized Devices
• “Smart” cell VXP Cell Processing
System
• Integrated QC System
• Software Driven
• Vision Based
• Autologous (Safe and Fast)
• Data Validated
• Highly Consistent
• Removes Autologous Dose Variability
• 600 Patients Treated Across Eight
Clinical Indications
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Late Stage Development in Ischemic Cardiovascular
Disease
Hematopoietic Stem Cell Therapy (i.e. BMT)
Cell Therapy Priorities
IDE/PMAIntra-operative therapies
510(k)Intra-laboratory devices
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• Primary Mechanism: Paracrine Effect and EPCs– Drives Revascularization
(Collaterals)
• Implanted cells migrate to ischemic regions
• Implanted cells secrete cytokines for mobilization of local tissue stem cells
• Implanted cells secrete cytokines for mobilization of bone marrow stem cells
– Revascularization drives regeneration
Vascular Cell TherapiesUnderlying Science for Ischemic Diseases (Mechanism of Actions)
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Vascular Cell TherapiesConfirming Our MOA for Ischemic Diseases with Human Data
IM Delivery of BMMNCs : > 21 Day Life but < 28 Days BMMNC (with EPCs) Initiate Tube Formation
Odaka et al (2011) PLoS One. 6(10):e25487
Verified In Human Trials Ph I/II
Sanghi et al 2013, AMIRST IAMIRST cell product – Cook catheter study, 2014
Kang et al CMAJ, 2004
Bukhari et al, 2013, CLIRST IIn vitro bioactivity data, CLIRST III
Critical Limb Ischemia (CLIRST)
Acute Myocardial Infarction (AMIRST)
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Critical Limb IschemiaCompelling clinical vascular results (CLIRST I)
All patients “no option” and near term leg amputation recommended
12 Month F/U Data
• Major Amputation Free Rate post SurgWerks™ Therapy PP = 82.4%
• Reduction in VAS Pain Score from 7.8±0.97 to 0.2±0.58
• Improvement in 6 minute walk test from 14.5m to 157m
• N=17 patients
Major RevascularizationTrial Results
CompletedPhase I/II
IDE PMA Path
Pivotal
Submitted IDE Nov 2014
1st Subject Q1 2015Day 0 Day 365
CLIRST I CLIRST III
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Critical Limb IschemiaIDE U.S. Pivotal Clinical Study Application (CLIRST III)
• Double Blinded Randomized
Controlled Trial
• 12 Month Follow Up
• Subjects = 224
80% Power, delta of 20pp, dropout of 7%
• Up to 60 Clinical Trial Sites
Study Principal Investigator:
• Dr. Richard Powell, M.D.
Chief, Vascular Surgery
Dartmouth
EndpointsTrial Design
Primary: Major Amputation Free Survival
Secondary: - Time to Treatment
Failure- Wound Healing- SPP- QoL- ABI- Pain- AEs/MACE
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• 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 Mo F/U)
Cardiac Tissue RepairTrial Results
Acute Myocardial InfarctionCompelling clinical cardiac results (AMIRST I)
PilotCompleted
Ph II
Submit Q1 2015Begin June Qtr. 2015
(India DCGI)
AMIRST I AMIRST II
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CellWerksTM Bone Marrow TransplantAutomating Clinical Major Mismatch & Haploidentical HSCT Transplant
• High - CD34 recoveries = 77.7%
• Low - hematocrit <12%
• Faster - Neutrophil engraftment = Day 18
• Faster - Platelet engraftment = Day 35
Haploidentical Clinical Results
Major ABO Clinical Results
Faster,Lower Cost,Higher Cell Recovery
510(k) Q2 2015
6,000 = new patients WW
$1,500 = Price of treatment
$9 M = Addressable market
• Enables HLA half match from parent or
children
• Fortis-TotipotentRX Center World Class
Transplant Center
• CellWerks Reduces Expensive Reagent
Usage
• CellWerks Enables Cell Washing without
high stem cell loss
6,000 = new patients in India
$25,000 = Price of treatment
$150 M = Addressable market
Enabling 6k new transplants/year in India
Phase I/II Q2 2015
Improves Access for Pediatric BMT
(40% are mismatched)
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Clinical Trial Pipeline Major MilestonesSubstantial clinical value drivers
CLI Milestones Calendar Quarter
FDA IDE/PMA Pivotal Trial Submission Completed in Nov 2014
FDA Approval of Trial Design Q1 2015
First Patient Enrolled Q1 2015
Pivotal Phase Completed Late 2016
CLI PMA Submission (commercialization dependent on US FDA) Early 2017
BMT Milestones Time Period
ABO Mismatch FDA 510(K) Submission Q1 2015
Haplo Pilot Phase Trail Data Published Q3 2015
Haplo India DCGI Submission for Marketing 2016
AMI Milestones Time Period
DCGI Phase II Trial Submission Q1 2015
Phase II First Patient Treated Q3 2015
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Cesca TherapeuticsMarket Cap Snapshot
Cell Therapy Peer Company TCKR Market Capitalization (000’S)
Vericel Corporation “Aastrom
Biosciences Inc.”VCEL $66,000
Athersys ATHX $109,000
BioTime, Inc. BTX $263,000
Nuo Therapeutics (Cytomedix) CMXI $34,000
Neuralstem, Inc. CUR $237,000
Cytori Therapeutics Inc. CYTX $39,000
NeoStem Inc. NBS $137,000
Osiris Therapeutics, Inc. OSIR $452,000
Pluristem Therapeutics Inc. PSTI $177,000
StemCells Inc. STEM $78,000
Average Market Cap $159,000
Cesca Therapeutics KOOL $42,000
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Cesca’s SurgWerksTM & CellWerksTM
Industry Leader in Vascular & Hemotology
Significant commercial experience, proven technical reliability
• Over 20,000 patients treated @ the POC with Cesca Cell Technologies
• Over 2,500 pediatric patients treated with AXP processed cord blood stem cells
• Over 600,000 cord samples processed (laboratory processing & storage)
• Near six-sigma disposable quality levels
Clinically validated, proprietary protocols & method patents
• Proprietary, smart platforms
• Proprietary cell formulations addressing multiple disease indications
• Advantage with IDE regulatory strategy to be first device cell therapy “in a box”
IP Suite (device and algorithm patents)
• 43 Design and device patents
• Three treatment patents (pending) for AMI, CLI and Ischemia diseases (U.S. national phase)
• 8 pilot & phase 1b clinical trials
• 7 clinical algorithms
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Cesca TherapeuticsRegenerative Medicine Investor Checklist
Safe – Autologous; Understood safety
Clinically effective – Yes; Bone Marrow Cells
Large markets – Multiple; Ischemia & BMT Key
Regulatory hurdles – Lower; US IDE Path
Cost effective – Yes; Significantly Lower then Autologous GMP
Reimbursable – Well positioned
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Management HighlightsChief Executive Officer & Director
Robin C. Stracey - Interim CEO having served on Cesca Board of Directors for the past three years. Prior experience
includes President and Chief Executive Officer at both publicly-traded and privately held companies in the life sciences sector,
including Integrated Fluidics, Inc., Cantimer Incorporated, and Applied Imaging Corporation.
President & DirectorKenneth L. Harris - Joined Cesca in February 2014 pursuant to merger. Prior experience includes Chairman and CEO
of TotipotentRX /MK Alliance, Inc, and Corporate Senior Vice President and Global President of Biosciences of Pall Corporation.
Chief Financial Officer
Dan T. Bessey - Joined Cesca in March 2013. Prior experience includes CFO of SureWest Communications and Vice
President of Finance, Controller and Director of Corporate Finance.
Chief Biologist
Mitch Sivilotti – Joined Cesca in February 2014 pursuant to merger. Prior experience includes President, Director and
Chief Biologist of TotipotentRX/MK Alliance, Inc. and Pall Corporation.
VP, Quality & Regulatory Affairs
Raymond DeGrella – Consulted for Cesca as VP of Quality and Management Representative since 2012. In March
2014 assumed current role. Former experience includes Vice President Advanced Supply Chain of Beckman Coulter, and
Abbott Laboratories.
VP, OperationsKen Pappa – Joined Cesca in April 2006 as Director of Finance and has held several managerial roles until October 2012
when he took on his current role. Prior experience includes Manufacturing Controller and Senior Operations Manager for
Hewlett Packard-Agilent Technologies.