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25th AnnualROTH Conference
NYSE MKT: BTX
March, 2013
Forward Looking Statements
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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.
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
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• 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
The BioTime Opportunity
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• 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
PureStem Manufacturing Technology
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Human embryonic progenitor (hEP)cell lines: > 200 diverse cell types isolated
DiversityPrecise identityPurityScalabilityPatents pending
Skeletal Muscle
Kidney
Smooth Muscle
Blood/BrainBarrier
Injectable, biocompatible, and biodegradable
ReneviaTM – Cell Delivery Device
Cells in Renevia Matrix (Cross-linked Collagen and Hyaluronate)
Low COGs, Stable Product, Potential Widespread Applications
HyStem Hydrogels – Competitive Advantages
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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
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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
ReneviaTM – A Cell Delivery Device
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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
Potential ReneviaTM Applications
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• 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
Subsidiaries
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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
OncoCyte Corporation- PanC-DxTM
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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
PanC-DxTM Cancer Diagnostic
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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
Subsidiaries
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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
Cell Cure Neurosciences Ltd.
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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
Cell Cure Neurosciences Ltd.
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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
Subsidiaries
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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
OrthoCyte Corporation
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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
OrthoCyte- Cartilage Progenitors
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E15
Subsidiaries
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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
Regenerative Medicine
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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
ReCyte Therapeutics, Inc.
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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
Subsidiaries
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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
LifeMap Sciences, Inc.
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• 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
LifeMap BioReagents
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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
Subsidiaries
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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
BioTime Acquisition Corp.
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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…
BioTime Acquisition Corp.
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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…
BioTime Acquisition Corp.
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Stem Cell Assets Being Contributed from Geron
BioTime Acquisition Corp.
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• 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
Current Product Pipeline
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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
BioTime (NYSE MKT: BTX)
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$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:
BioTime’s Key Milestones
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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
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>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