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25 th Annual ROTH Conference NYSE MKT: BTX March, 2013.

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25 th Annual ROTH Conference NYSE MKT: BTX March, 2013
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Page 1: 25 th Annual ROTH Conference NYSE MKT: BTX March, 2013.

25th AnnualROTH Conference

NYSE MKT: BTX

March, 2013

Page 2: 25 th Annual ROTH Conference NYSE MKT: BTX March, 2013.

Forward Looking Statements

2

The matters discussed in this presentation include forward looking statements which are subject to various risks, uncertainties, and other factors that could cause actual results to differ materially from the results anticipated. Such risks and uncertainties include but are not limited to the success of BioTime in developing new stem cell products and technologies; results of clinical trials of BioTime products; the ability of BioTime and its licensees to obtain additional FDA and foreign regulatory approval to market BioTime products; competition from products manufactured and sold or being developed by other companies; the price of and demand for BioTime products; and the ability of BioTime to raise the capital needed to finance its current and planned operations. Any statements that are not historical fact (including, but not limited to statements that contain words such as "will," "believes," "plans," "anticipates," "expects," "estimates") should also be considered to be forward-looking statements. Forward-looking statements involve risks and uncertainties, including, without limitation, risks inherent in the development and/or commercialization of potential products, uncertainty in the results of clinical trials or regulatory approvals, need and ability to obtain future capital, and maintenance of intellectual property rights. As actual results may differ materially from the results anticipated in these forward-looking statements they should be evaluated together with the many uncertainties that affect the business of BioTime and its subsidiaries, particularly those mentioned in the cautionary statements found in BioTime's Securities and Exchange Commission filings. BioTime disclaims any intent or obligation to update these forward-looking statements.

Page 3: 25 th Annual ROTH Conference NYSE MKT: BTX March, 2013.

The Health Care Challenge & Opportunity

A global tsunami of health care costs from ineffectiveness of drugs to treat chronic degenerative disease (CDD)

• CDD due to lack of cellular regeneration

• 80% Americans have CDD of aging

• 50% of Americans have two CDDs

• 95% costs in aging is CDD

Page 4: 25 th Annual ROTH Conference NYSE MKT: BTX March, 2013.

The Health Care Challenge & Opportunity

4

• 1974 – Gene cloning technology developed

• 1976 - Moratorium on rDNA research initiated led to established guidelines on rDNA research

• 1989 – First $B product EPO

• Today, products from the use of rDNA technology are ubiquitous

• >140 clinical trials

• Current Global Market $75 B

Recombinant DNA Technology

• 1975 - Hybridoma technology developed

• 1997- First $B Product Rituximab

• Advances in Mab Engineering

• Today, eight of the 20 best-selling biotechnology drugs in therapeutic monoclonal antibodies

• > 200 clinical trials

• Current Global Market $44 B

Monoclonal Antibodies

•1998 – Embryonic Stem Cells allowing for the first time in the history of medicine the manufacture of all human cellular components

•2001 – U.S. Federal funding restriction (reversed in 2009)

•2010 – 1st hES Clinical trial

• Future – 1st $B product

Regenerative Medicine

Progressive evolution of new & more interventional products over past four decades

Page 5: 25 th Annual ROTH Conference NYSE MKT: BTX March, 2013.

The BioTime Opportunity

5

• Consolidating key stem cell assets- ES Cell International- Geron stem cell assets- HyStem hydrogels

• Integration with PureStem technology• Focus on near-term products• Significant clinical milestones for 2013

Page 6: 25 th Annual ROTH Conference NYSE MKT: BTX March, 2013.

PureStem Manufacturing Technology

6

Human embryonic progenitor (hEP)cell lines: > 200 diverse cell types isolated

DiversityPrecise identityPurityScalabilityPatents pending

Skeletal Muscle

Kidney

Smooth Muscle

Blood/BrainBarrier

Page 7: 25 th Annual ROTH Conference NYSE MKT: BTX March, 2013.

Injectable, biocompatible, and biodegradable

ReneviaTM – Cell Delivery Device

Cells in Renevia Matrix (Cross-linked Collagen and Hyaluronate)

Low COGs, Stable Product, Potential Widespread Applications

Page 8: 25 th Annual ROTH Conference NYSE MKT: BTX March, 2013.

HyStem Hydrogels – Competitive Advantages

8

Polymerizes safely in vivo

Stays as liquid for ~ 20 minutes

Supports survival of cells and 3-D structure

Cast Hydrogel Cells in Sponge

Injectable

Multiple Formulations

Durable Films 3-D Lattices Heparin-mediated Slow Release

Page 9: 25 th Annual ROTH Conference NYSE MKT: BTX March, 2013.

9

Indication: As a delivery matrix for autologous adipose derived cells to correct subcutaneous lipoatrophy arising from trauma, oncologic resection, or congenital defect.

3 Phase Clinical Evaluation of ReneviaTM

• Phase 1: Safety - open label, ReneviaTM only, subcutaneous injection, 10 subjects, 4 week follow up.

• Phase 2: Treatment Effect - open label, ReneviaTM + ADSCs, 15 patients, 3-6 months follow up.

• Phase 3: Pivotal - randomized, controlled, evaluator blind, non-inferiority, ReneviaTM + ADSCs vs. Cell-Assisted Lipotransfer, # of patient per group TBD, 6 months follow up.

• Primary endpoints: Aesthetic Improvement Score & photo review

• Secondary endpoint: Change in subcutaneous defect volume

• Single Site: The Stem Center, Palma de Mallorca, Spain

ReneviaTM – Development

Page 10: 25 th Annual ROTH Conference NYSE MKT: BTX March, 2013.

ReneviaTM – A Cell Delivery Device

10

Adipose tissue restoration with autologous ASCs Medical device (CE Mark in the EU then CBER/CDRH) Outsource cGMP manufacture Near-term opportunity, low cost for approval

ReneviaTM

Regulatory Strategy

ReneviaTM Timeline

2012

Q4 Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4

2013 2014

ISO 13485 Certification

Final Dossier

Submission

CE Mark Renevia™

Complete cGMPManufacture

Q1

2015

Safety Clinical

Trial Initiated

Treatment Effect Clinical Trial Initiated

Pivotal Clinical Trial

Initiated

Expected ReneviaTM CE Mark in 2015

Page 11: 25 th Annual ROTH Conference NYSE MKT: BTX March, 2013.

Potential ReneviaTM Applications

11

• Heart Disease ✔ 165 B• Alzheimer’s ✔ 69 B• Diabetes ✔ 92 B• Stroke ✔ 28 B• Renal Failure ✔ 33 B

Potentia

l

U

tility

Potentia

l

A

pplicab

le

U.S

. Mar

ket

Regen. Med. (2008) 3(3), 351–363

Page 12: 25 th Annual ROTH Conference NYSE MKT: BTX March, 2013.

Subsidiaries

12

OncoCyte CorporationCancer diagnostics and anti-cancer therapies

OrthoCyte CorporationCartilage repair

ES Cell International PTE Ltd.Clinical-grade master stem cell banks

LifeMap Sciences, Inc.Stem cell data base and research products

Cell Cure Neurosciences Ltd.Age-related macular degeneration (AMD)

ReCyte Therapeutics, Inc.Age-related vascular disorders

BioTime Acquisition Corp..Expected Contribution of Geron and BioTime Assets

Page 13: 25 th Annual ROTH Conference NYSE MKT: BTX March, 2013.

OncoCyte Corporation- PanC-DxTM

13

Screening in asymptomatic patients is the largest market opportunity

PanC-DxTM Pan diagnostic to detect the presence of various

human cancers, including cancers of the breast, lung, bladder, uterus, stomach, and colon, during routine check-ups using a low-cost antibody-based blood test.

mAb production for seven antigens in processSpecificity of >50 mAbs characterizedDynamic testing for use in ELISA and point of

care formats currently underway.Initially develop and seek regulatory approval of

PanC-DxTM in Europe Expected launch in 2014

Page 14: 25 th Annual ROTH Conference NYSE MKT: BTX March, 2013.

PanC-DxTM Cancer Diagnostic

14

2011

Q4 Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4Q3

2012 2014

Marker Validation

Antibody Production

Final Kit Developed

CE Mark

Regulatory Strategy CE Mark (EU) then CDRH/OIVD (U.S.) Near-term opportunity, low cost for approval

Timeline leading to 2014 launch

Patient Study Completion

Antibody Screening

2013

Q4Q3Q2Q1

Page 15: 25 th Annual ROTH Conference NYSE MKT: BTX March, 2013.

Subsidiaries

15

OncoCyte CorporationCancer diagnostics and anti-cancer therapies

OrthoCyte CorporationCartilage repair

ES Cell International PTE Ltd.Clinical-grade master stem cell banks

LifeMap Sciences, Inc.Stem cell data base and research products

Cell Cure Neurosciences Ltd.Age-related macular degeneration (AMD)

ReCyte Therapeutics, Inc.Age-related vascular disorders

BioTime Acquisition Corp..Expected Contribution of Geron and BioTime Assets

Page 16: 25 th Annual ROTH Conference NYSE MKT: BTX March, 2013.

Cell Cure Neurosciences Ltd.

16

OpRegen® – RPE Cells for the Treatment of AMD

7.3 million have early stage dry AMD in US

Any effective treatment expected to achieve blockbuster sales

OpRegen® – Suspension of retinal pigment epithelial (RPE) cells for dry age-related macular degeneration (AMD)

OpRegen® Plus – Matrix bound RPE cells for dry AMD

Partnered with TEVA

Page 17: 25 th Annual ROTH Conference NYSE MKT: BTX March, 2013.

Cell Cure Neurosciences Ltd.

17

Planned IND in 2013/2014

Phase I/IIa Study of hESC-RPE in Progressive Dry-Form Age-Related Macular Degeneration (12 patients, 1 year follow up time, single doses)Primary endpoint: SafetySecondary endpoint: Efficacy

• Visual Acuity• Maintenance of graft size over serial measurements (graft survival)• Reduced expansion of geographic atrophy in grafted sites vs. non- grafted sites (in the same eye)• Perimetry- extent and depth of central scotomata

• Single site: Hadassah, Israel Duration (enrollment and follow up): 18 months

Page 18: 25 th Annual ROTH Conference NYSE MKT: BTX March, 2013.

Subsidiaries

18

OncoCyte CorporationCancer diagnostics and anti-cancer therapies

OrthoCyte CorporationCartilage repair

ES Cell International PTE Ltd.Clinical-grade master stem cell banks

LifeMap Sciences, Inc.Stem cell data base and research products

Cell Cure Neurosciences Ltd.Age-related macular degeneration (AMD)

ReCyte Therapeutics, Inc.Age-related vascular disorders

BioTime Acquisition Corp..Expected Contribution of Geron and BioTime Assets

Page 19: 25 th Annual ROTH Conference NYSE MKT: BTX March, 2013.

OrthoCyte Corporation

19

Developing regenerative therapeutics for orthopedic applications

Cartilage cannot regenerate on its own Osteoarthritis and spinal disc degeneration have

a significant impact on mobility and health - > 26 million people in U.S. and growing

Drug treatments target the reduction of pain and inflammation as opposed to repairing tissue

Studies in animal models of IVD disease underway

- Non-hypertrophic progenitors- Off the shelf approach

Page 20: 25 th Annual ROTH Conference NYSE MKT: BTX March, 2013.

OrthoCyte- Cartilage Progenitors

20

E15

Page 21: 25 th Annual ROTH Conference NYSE MKT: BTX March, 2013.

Subsidiaries

21

OncoCyte CorporationCancer diagnostics and anti-cancer therapies

OrthoCyte CorporationCartilage repair

ES Cell International PTE Ltd.Clinical-grade master stem cell banks

LifeMap Sciences, Inc.Stem cell data base and research products

Cell Cure Neurosciences Ltd.Age-related macular degeneration (AMD)

ReCyte Therapeutics, Inc.Age-related vascular disorders

BioTime Acquisition Corp..Expected Contribution of Geron and BioTime Assets

Page 22: 25 th Annual ROTH Conference NYSE MKT: BTX March, 2013.

Regenerative Medicine

22

Human embryonic stem (hES) for limited allogeneic applications

Twin strategies for cell transplantation

• Sites behind the blood brain barrier

• Sites behind synovial membranes

• Cells targeting tumors for destruction

Human induced pluripotent stem (iPS) cells for auto-logous transplantation

• Vascular disease

Page 23: 25 th Annual ROTH Conference NYSE MKT: BTX March, 2013.

ReCyte Therapeutics, Inc.

23

RCX-VP01 for Age-Related Vascular Insufficiency

American Heart Association Report on Heart Disease and Stroke Statistics:2011 Update

Cardiovascular Disease (CVD)Prevalence: • 82,600,000 American adults (>1 in 3) with 1 or more types of CVD• 2200 deaths from CVD per day; average life expectancy shortened 7 years

Stroke (Cerebrovascular Disease)Incidence:• 800,000 new (77%) or recurrent (23%) strokes per year• 1 of every 18 deaths in U.S. in 2007

Healthcare Impact and Cost:• $286 billion total direct and indirect costs of CVD and stroke in U.S. in 2007 • nearly 7 million inpatient cardiovascular operations and procedures in 2007

Page 24: 25 th Annual ROTH Conference NYSE MKT: BTX March, 2013.

Subsidiaries

24

OncoCyte CorporationCancer diagnostics and anti-cancer therapies

OrthoCyte CorporationCartilage repair

ES Cell International PTE Ltd.Clinical-grade master stem cell banks

LifeMap Sciences, Inc.Stem cell data base and research products

Cell Cure Neurosciences Ltd.Age-related macular degeneration (AMD)

ReCyte Therapeutics, Inc.Age-related vascular disorders

BioTime Acquisition Corp..Expected Contribution of Geron and BioTime Assets

Page 25: 25 th Annual ROTH Conference NYSE MKT: BTX March, 2013.

LifeMap Sciences, Inc.

25

• Used in more than 1,000 institutions world-wide including academia, research hospitals, patent offices, and the leading biotech and pharma

• More than 12 Million page visits/year by hundreds of thousands of unique users

• 30% usage growth in the past 12 months

• Consistently leads in top positions for gene search results in Google

The leading human gene database

Page 26: 25 th Annual ROTH Conference NYSE MKT: BTX March, 2013.

LifeMap BioReagents

26

Cells

Powerful Marketing Platform

>12 Million page hits/yr

Genes Diseases

Near-term RevenueSeed for Future

Royalties

BioTime Products

All online% of Sales

Third Party Products

> 1,000 USCN

Antibody kits

>1,000 Prospec-

Tany Technogene

Proteins

Page 27: 25 th Annual ROTH Conference NYSE MKT: BTX March, 2013.

Subsidiaries

27

OncoCyte CorporationCancer diagnostics and anti-cancer therapies

OrthoCyte CorporationCartilage repair

ES Cell International PTE Ltd.Clinical-grade master stem cell banks

LifeMap Sciences, Inc.Stem cell data base and research products

Cell Cure Neurosciences Ltd.Age-related macular degeneration (AMD)

ReCyte Therapeutics, Inc.Age-related vascular disorders

BioTime Acquisition Corp..Contribution of Geron and BioTime Assets

Page 28: 25 th Annual ROTH Conference NYSE MKT: BTX March, 2013.

BioTime Acquisition Corp.

28

Geron will contribute:• IP and tangible assets related to hES programs• Phase 1 trials in acute spinal cord injury & T’ase vaccine• IP and sublicense to autologous cellular immunotherapy program• Interests in non-therapeutic applications

• including products developed and sold by GE HeathcareGeron receives:• Shares in BAC (approx 21.4% interest)• Royalties on sale of products using Geron patents• Shares will be distributed by Geron to their shareholders (following closing)

Terms subject to closing

What Geron Contributes and Receives…

Page 29: 25 th Annual ROTH Conference NYSE MKT: BTX March, 2013.

BioTime Acquisition Corp.

29

BioTime will contribute to BAC:• $5 million in cash (committed through a private investor)• 8.9 MM in BioTime Common Stock to be held by BAC• 8 million BioTime warrants

• At closing, to be distributed by BAC to Geron stockholders• Minority equity interest in OrthoCyte and Cell Cure• Rights to use certain BioTime cell lines

BioTime receives:• Shares in BAC (Approx. 71.6% interest in BAC)• 3.15 MM Warrants in BAC

Terms subject to closing

What BioTime Contributes and Receives…

Page 30: 25 th Annual ROTH Conference NYSE MKT: BTX March, 2013.

BioTime Acquisition Corp.

30

Stem Cell Assets Being Contributed from Geron

Page 31: 25 th Annual ROTH Conference NYSE MKT: BTX March, 2013.

BioTime Acquisition Corp.

31

• Feeder-free culture of hPS cells• hES-derived dendritic cells for treating cancer• TGFbeta inhibitors & Neural• RNA isolation from hPS cells or derivatives• Feeder free hPS cells in drug screening• Medium for hPS cell culture• hPS-derived hepatocytes• Dopaminergic neurons from hPS cells• hPS cell-specific genetic toxicity• Selective antigen expression on hPS cells

>400 issued & pending patent applications worldwide including:

Stem Cell Assets Being Contributed from Geron

Page 32: 25 th Annual ROTH Conference NYSE MKT: BTX March, 2013.

Current Product Pipeline

32

Phase I Phase II Phase III Market PartnerPreclinical

Hextend

Research Products/Database

Teva

Renevia

PanC-DxTM

OpRegen

OTX-CP07

RCX-VP01

HyStem® Hydrogel Products

2014-2015

2014

2013

Page 33: 25 th Annual ROTH Conference NYSE MKT: BTX March, 2013.

BioTime (NYSE MKT: BTX)

33

$4.4MM cash of 12/31/12 Raised additional $16.3 million to date in 2013 Approx. $10MM in investments held by our

subsidiaries

Major Shareholders:

Greenbelt/Greenway/

Al Kingsley (Chairman)

Revenue of $3.9MM in 2012 Avg. qtrly burn rate of $4.9MM per quarter No debt Market Cap approx. $240MM as of 3/15/13

17.5%

Broadwood Capital

(Neal Bradsher, Director)

16.1%

Individual Investor

9.2%

Insiders

& LT Investors

46.7%

BioTime shareholder base with significant long term investors

A Technological Leader with Revenue, Low Burn Rate and Near-term Product Opportunities

Key Statistics:

Page 34: 25 th Annual ROTH Conference NYSE MKT: BTX March, 2013.

BioTime’s Key Milestones

34

Next 12 months…

• Initiation of HyStem ® -Rx clinical trial• IND filing of OpRegen for AMD• PanC-Dx patient study• Corporate partnering and collaborations • Increased revenue from LifeMap BioReagents

Page 35: 25 th Annual ROTH Conference NYSE MKT: BTX March, 2013.

35

>200 purified human cell types manufactured for the first time

Leading patent position Emphasis on near-term products:

- ReneviaTM

- PanC-DxTM

- LifeMap BioReagents Longer–term first-in-class stem cell-

based therapeutics for currently incurable degenerative diseases

Subsidiaries focused on specific disease areas

Positioned to Lead the Commercialization of Pluripotency in Regenerative Medicine

Alameda, California Headquarters

Summary

Page 36: 25 th Annual ROTH Conference NYSE MKT: BTX March, 2013.

Thank You

Michael West, Ph.D.CEOPete [email protected]


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