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Corporate Presentation January 2015 Rev. 1214
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Page 1: Corporate Presentation January 2015 - Cesca …cescatherapeutics.com/wp-content/uploads/2014/04/Biotech... · 2015-02-14 · This presentation contains forward-looking statements.

Corporate Presentation

January 2015

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 $41 Million

Shares Outstanding 40.3 Million

Options Outstanding 2.2 Million

Warrants Outstanding 5.1 Million

Stock Price $1.02

Average Trading Volume 100,808

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 January 8, 2015

Financials, including options and warrants outstanding as of September 30, 2014

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• Multiple Therapies – 8 Pilot & Phase

1b Clinical Trials

• M&A/Bold on Opportunities to Help

us Further Fill Out our Tools

Capabilities

• Late Stage (Pivotal) Clinical Trials for

CLI

• Safe & Effective; in the Regulatory

“Sweet Spot”

• Rapid; 90 Minute Bedside Protocol

• Low Cost Delivery (No External GMP

Manufacturing or Cold Supply Chain)

• Existing Manufacturing & Selling

Experience

• Devices; Patented Platform

Technology (>40 Issued

Patents)

• Methods; 3 Patents Pending

Methods for CLI, AMI & General

Ischemia

• Biologics Development

• Device Engineering

Development

• Clinical Development

⁻ Clinical Research

Organization (CRO)

Cesca TherapeuticsAn integrated regenerative medicine company in late stage clinical trial

Research &

Development

Strong Intellectual

Property

Commercialization

Stockholder

Value

Concept to Commercialization

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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

HIGH LOW

Regulatory Risk

IND/NDA or BLA IDE/PMA3 Phase Trial 2 Phase Trial

Higher Trial Patient Population Lower Trial Patent Population

Higher FDA Marketing Approval Threshold

Lower FDA Market Approval Threshold

Allogeneic Cells Autologous Cells

Manipulated Cells Minimally Manipulated Cells

Homologous or Non-Homologous Homologous or Non-Homologous

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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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Clinical Trial PortfolioBlockbuster Therapy Candidates Addressing $16B Market

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Ischemic Cardiovascular Disease

• Critical Limb Ischemia

• Myocardial Infarction

Hematopoietic Stem Cell Therapy (BMT)

• ABO Mismatch

• Haplo-identical

Cell Therapy Priorities

SurgWerks™

CellWerks™

IDE/PMAIntra-operative therapies

510(k)Intra-laboratory devices

Clinical Targets Regulatory Pathway

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Primary Mechanism of Actions• Paracrine Effect and EPCs

⁻ 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 - Revascularization

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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”, 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

Day 0 Day 365

CompletedPhase I/II

IDE PMA Path

Pivotal

Submitted IDE Nov 20141st Subject Q2 2015

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

• Blinded Independent Review Committee

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

- Skin Perfusion Pressure (SPP)

- Quality of Life (QoL)

- Ankle Brachial Index (ABI)

- Pain

- Adverse Events (AEs)

- Major Adverse Cardiac Event (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

Phase II

Submit Q2 2015Begin June Qtr. 2015

(India DCGI)

AMIRST I AMIRST II

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Faster,Lower Cost,Higher Cell Recovery

CellWerksTM Bone Marrow TransplantAutomating Clinical Major Mismatch & Haplo-identical HSCT Transplant

• Higher - CD34 recoveries = 77.7%• Lower - hematocrit <12%• Faster - Neutrophil engraftment = Day 18• Faster - Platelet engraftment = Day 35

Haplo-identical Clinical Results

Major ABO Clinical Results

510(k) Q2 2015

6,000 = new patients WW

$1,500 = Price of treatment

$9 M = Addressable market

• HLA half match from parent or children• Fortis-TotipotentRX Center World Class

Transplant Center• Reduces Expensive Reagent Usage• Cell Washing without high stem cell loss

6,000 = new patients in India

$25,000 = Price of treatment

$150 M = Addressable market

Enabling 6,000 new transplants/year in India

Phase I/II Q3 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 Q2 2015

First Patient Enrolled Q2 2015

Pivotal Phase Completed Late 2016

CLI PMA Submission (commercialization dependent on US FDA) Early 2017

BMT Milestones Calendar Quarter

ABO Mismatch FDA 510(K) Submission Q1 2015

Haplo Pilot Phase Trial Data Published Q3 2015

Haplo India DCGI Submission for Marketing 2016

AMI Milestones Calendar Quarter

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 $72,000

Athersys ATHX $147,000

BioTime, Inc. BTX $353,000

Nuo Therapeutics (Cytomedix) NUOT $44,000

Neuralstem, Inc. CUR $306,000

Cytori Therapeutics Inc. CYTX $48,000

NeoStem Inc. NBS $131,000

Osiris Therapeutics, Inc. OSIR $573,000

Pluristem Therapeutics Inc. PSTI $198,000

StemCells Inc. STEM $78,000

Average Market Cap $195,000

Cesca Therapeutics KOOL $41,000

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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.

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Thank you


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