BioTimePresentation

Report
25th Annual
ROTH Conference
NYSE MKT: BTX
March, 2013
Forward Looking Statements
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.
2
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
The Health Care Challenge & Opportunity
Progressive evolution of new & more
interventional products over past four decades
Recombinant DNA
Technology
• 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
Monoclonal Antibodies
• 1975 - Hybridoma technology
developed
• 1997- First $B Product
Rituximab
• Advances in Mab Engineering
• Today, eight of the 20 bestselling biotechnology drugs in
therapeutic monoclonal
antibodies
• > 200 clinical trials
Regenerative Medicine
•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
• Current Global Market $44 B
4
The BioTime Opportunity
• 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
5
PureStem Manufacturing Technology
Human embryonic progenitor (hEP)
cell lines: > 200 diverse cell types isolated
 Diversity
 Precise
identity
 Purity
 Scalability
 Patents
pending
6
ReneviaTM – Cell Delivery Device
Injectable, biocompatible, and biodegradable
Cells in Renevia Matrix (Crosslinked Collagen and Hyaluronate)
Low COGs, Stable Product,
Potential Widespread Applications
HyStem Hydrogels – Competitive Advantages
Injectable
Stays as liquid
for ~ 20 minutes
Polymerizes
safely in vivo
Supports survival of
cells and 3-D structure
3-D Lattices
Heparin-mediated
Cells in Sponge
Slow Release
Multiple
Formulations
Durable
Films
Cast Hydrogel
8
ReneviaTM – Development
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, noninferiority, 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
9
ReneviaTM – A Cell Delivery Device

ReneviaTM
Regulatory Strategy



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
Expected ReneviaTM CE Mark in 2015
ReneviaTM Timeline
Complete cGMP
Manufacture
Treatment
Effect Clinical
Trial Initiated
CE Mark
Renevia™
Safety
Clinical
Trial
Initiated
Final
Dossier
Submission
Pivotal
Clinical Trial
Initiated
ISO 13485
Certification
Q4
2012
Q1
Q2
2013
Q3
Q4
Q1
Q2
2014
Q3
Q4
Q1
2015
10
Potential ReneviaTM Applications
•
•
•
•
•
Heart Disease
Alzheimer’s
Diabetes
Stroke
Renal Failure
✔
✔
✔
✔
✔
165 B
69 B
92 B
28 B
33 B
Regen. Med. (2008) 3(3), 351–363
11
Subsidiaries
OncoCyte Corporation
Cancer diagnostics and anti-cancer therapies
Cell Cure Neurosciences Ltd.
Age-related macular degeneration (AMD)
OrthoCyte Corporation
Cartilage repair
ReCyte Therapeutics, Inc.
Age-related vascular disorders
ES Cell International PTE Ltd.
Clinical-grade master stem cell banks
LifeMap Sciences, Inc.
Stem cell data base and research products
BioTime Acquisition Corp..
Expected Contribution of Geron and BioTime Assets
12
OncoCyte Corporation- PanC-DxTM
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 antibodybased blood test.
mAb production for seven antigens in process
Specificity of >50 mAbs characterized
Dynamic 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
13
PanC-DxTM Cancer Diagnostic

Regulatory Strategy

CE Mark (EU) then CDRH/OIVD (U.S.)
Near-term opportunity, low cost for approval
Timeline leading to 2014 launch
Antibody
Screening
Marker
Validation
Final Kit
Developed
Antibody
Production
Q3
2011
Q4
Q1
Q2
Q3
2012
Q4
Q1
CE Mark
Patient Study
Completion
Q2
Q3
2013
Q4
Q1
Q2
Q3
Q4
2014
14
Subsidiaries
OncoCyte Corporation
Cancer diagnostics and anti-cancer therapies
Cell Cure Neurosciences Ltd.
Age-related macular degeneration (AMD)
OrthoCyte Corporation
Cartilage repair
ReCyte Therapeutics, Inc.
Age-related vascular disorders
ES Cell International PTE Ltd.
Clinical-grade master stem cell banks
LifeMap Sciences, Inc.
Stem cell data base and research products
BioTime Acquisition Corp..
Expected Contribution of Geron and BioTime Assets
15
Cell Cure Neurosciences Ltd.
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
16
Cell Cure Neurosciences Ltd.
Planned IND in 2013/2014
Phase I/IIa Study of hESC-RPE in Progressive Dry-Form AgeRelated Macular Degeneration (12 patients, 1 year follow up time,
single doses)
Primary endpoint: Safety
Secondary endpoint: Efficacy
• Visual Acuity
• Maintenance of graft size over serial measurements (graft survival)
• Reduced expansion of geographic atrophy in grafted sites vs. nongrafted sites (in the same eye)
• Perimetry- extent and depth of central scotomata
• Single site: Hadassah, Israel
Duration (enrollment and follow up): 18 months
17
Subsidiaries
OncoCyte Corporation
Cancer diagnostics and anti-cancer therapies
Cell Cure Neurosciences Ltd.
Age-related macular degeneration (AMD)
OrthoCyte Corporation
Cartilage repair
ReCyte Therapeutics, Inc.
Age-related vascular disorders
ES Cell International PTE Ltd.
Clinical-grade master stem cell banks
LifeMap Sciences, Inc.
Stem cell data base and research products
BioTime Acquisition Corp..
Expected Contribution of Geron and BioTime Assets
18
OrthoCyte Corporation
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
19
OrthoCyte- Cartilage Progenitors
E15
20
Subsidiaries
OncoCyte Corporation
Cancer diagnostics and anti-cancer therapies
Cell Cure Neurosciences Ltd.
Age-related macular degeneration (AMD)
OrthoCyte Corporation
Cartilage repair
ReCyte Therapeutics, Inc.
Age-related vascular disorders
ES Cell International PTE Ltd.
Clinical-grade master stem cell banks
LifeMap Sciences, Inc.
Stem cell data base and research products
BioTime Acquisition Corp..
Expected Contribution of Geron and BioTime Assets
21
Regenerative Medicine
Twin strategies for cell transplantation
Human embryonic stem
(hES) for limited
allogeneic applications
• Sites behind the blood
brain barrier
• Sites behind synovial
membranes
• Cells targeting tumors
for destruction
Human induced pluripotent
stem (iPS) cells for autologous transplantation
• Vascular disease
22
ReCyte Therapeutics, Inc.
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
23
Subsidiaries
OncoCyte Corporation
Cancer diagnostics and anti-cancer therapies
Cell Cure Neurosciences Ltd.
Age-related macular degeneration (AMD)
OrthoCyte Corporation
Cartilage repair
ReCyte Therapeutics, Inc.
Age-related vascular disorders
ES Cell International PTE Ltd.
Clinical-grade master stem cell banks
LifeMap Sciences, Inc.
Stem cell data base and research products
BioTime Acquisition Corp..
Expected Contribution of Geron and BioTime Assets
24
LifeMap Sciences, Inc.
The leading
human gene database
• 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
25
LifeMap BioReagents
>12 Million page hits/yr
BioTime Products
Third Party Products
Cells
> 1,000 USCN
Antibody kits
Genes
>1,000 ProspecTany Technogene
Proteins
Diseases
Powerful Marketing Platform
Near-term
Revenue
Seed for Future
Royalties
All online
% of Sales
26
Subsidiaries
OncoCyte Corporation
Cancer diagnostics and anti-cancer therapies
Cell Cure Neurosciences Ltd.
Age-related macular degeneration (AMD)
OrthoCyte Corporation
Cartilage repair
ReCyte Therapeutics, Inc.
Age-related vascular disorders
ES Cell International PTE Ltd.
Clinical-grade master stem cell banks
LifeMap Sciences, Inc.
Stem cell data base and research products
BioTime Acquisition Corp..
Contribution of Geron and BioTime Assets
27
BioTime Acquisition Corp.
What Geron Contributes and Receives…
Terms subject to closing
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 Heathcare
Geron 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)
28
BioTime Acquisition Corp.
What BioTime Contributes and Receives…
Terms subject to closing
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
29
BioTime Acquisition Corp.
Stem Cell Assets Being Contributed from Geron
30
BioTime Acquisition Corp.
Stem Cell Assets Being Contributed from Geron
>400 issued & pending patent
applications worldwide including:
• 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
31
Current Product Pipeline
Preclinical
Phase I
Phase II
Phase III
Market
Partner
Hextend
Research Products/Database
HyStem® Hydrogel Products
2014-2015
Renevia
2014
PanC-DxTM
OpRegen
Teva
2013
OTX-CP07
RCX-VP01
32
BioTime (NYSE MKT: BTX)
A Technological Leader with Revenue, Low Burn Rate
and Near-term Product Opportunities
Key Statistics:



$4.4MM cash of 12/31/12
Raised additional $16.3 million to date in 2013
Approx. $10MM in investments held by our
subsidiaries




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
Major Shareholders:
17.5%
16.1%
9.2%
46.7%
Greenbelt/Greenway/
Broadwood Capital
Individual Investor
Insiders
Al Kingsley (Chairman)
(Neal Bradsher, Director)
& LT Investors
BioTime shareholder base with significant long term investors
33
BioTime’s Key Milestones
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
34
Summary
Positioned to Lead the Commercialization of
Pluripotency in Regenerative Medicine



>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 cellbased therapeutics for currently
incurable degenerative diseases
Subsidiaries focused on specific
disease areas
Alameda, California Headquarters
35
Thank You
Michael West, Ph.D.
CEO
Pete Garcia
CFO
510-521-3390
[email protected]

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