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Advanced Cell Technology
Corporate Presentation
March 2010
1
This presentation is intended to present a summary of ACT’s (“ACT”, or “Advanced
Cell Technology Inc”, or “the Company”) salient business characteristics.
The information herein contains “forward-looking statements” as defined under the
federal securities laws. Actual results could vary materially. Factors that could cause
actual results to vary materially are described in our filings with the Securities and
Exchange Commission.
You should pay particular attention to the “risk factors” contained in documents we
file from time to time with the Securities and Exchange Commission. The risks
identified therein, as well as others not identified by the Company, could cause the
Company’s actual results to differ materially from those expressed in any forward-
looking statements.
Cautionary Statement Concerning Forward-Looking
Statements
2
ACT Therapeutics
ACT Proprietary Human
Therapeutic Programs
Treatment Clinical Stage
Blastomere Program
Development of embryonic stem cell lines without
destruction of embryo
Pre-Clinical
Retinal Pigment Epithelium
(RPE) Program
Treatment of Age-related Macular Degeneration
(AMD) and Retinal Degenerative Diseases
Clinical IND
awaiting
FDA approval
Myoblast Program
Treatment of Heart Disease, Heart Attack and
Heart Failure
Phase II
Hemangioblast program
Treatment of Diseases and Disorders of the
Circulatory and Vascular Systems
Pre-Clinical
3
Pending
Multiple next generation technologies complementary to
existing patents and claims
PendingMultiple use patents for heart disease
StatusUse Patents
PendingCombinations of myoblasts and stem cells
PendingCombinations of myoblasts and muscle survival factors
PendingCatheter delivery of cells
AwardedMedia for preservation and transplant of muscle cells
AwardedStem cells capable of forming nerve or muscle cells
AwardedMuscle cells
StatusComposition of Matter Patent
Intellectual Property Protection
ACT owns or licenses over 150 patents and patent applications related to
stem cell and regenerative therapies
4
Institutional Collaborators
Advanced Cell’s Institutional Collaborators include:
Casey Eye Institute Mayo Clinic
Moran Eye Institute UCSF
Harvard Johns Hopkins
Stanford Sloan Kettering
University of Florida University of Iowa
University of Illinois U.C. Berkeley
Colorado State University
5
Blastomere Program: Synthesizing Stem Cells
without Harm to the Embryo
•Company scientists successfully generate stem
cell lines without destruction of embryo
•Cell lines retain potential to form all cells in the
human body
•Potential to treat a range of human diseases
•Technology has been reproduced and peer-
reviewed on several occasions
•National Institute of Health (NIH) proposing
expanded funding regulations to accommodate
ACT’s lines
6
First Proven Alternative hESC Method
•Enables Derivation of New hESC Lines
via Pre-implantation Genetic Diagnosis
(PGD) Method, Preserving Development
Potential of the Embryo
•7 hESC lines awaiting NIH approval for
funding
•Technology is used to develop RPE cells for
our clinical trials for Stargardt’s disease
Hemangioblasts
Differentiated from
Blastomere hESC Lines
7
RPE Program: Why RPE?
• Pigmented epithelium cells are easy to identify
• Small dosage vs. other therapies
• The eye is an immune-privileged site, thus no risk
of rejection at the injection site
• Ease of administration
– Doesn’t require a separate approval by the FDA (universal applicator)
– Procedure is already used by eye surgeons; no new skill set required
for doctors
• RPE cell therapy may positively impact over 200 retinal diseases
8
RPE Program Status
Complete Stage 1 of Phase I Clinical Trial for
Stargardt’s disease2010 – 4th Quarter
Developed the “Proof of Concept” for ACT’s RPE
cells in RCS animal model2006
Pre – IND meeting with FDA2008
Filed IND for Stargardt’s disease2009
FDA approval2010 – 3rd Quarter
9
GMP-compliant Human RPE Cells Derived from Embryonic Stem Cell
Lines Rescue Visual Function In a Rat Model for Photoreceptor
Degeneration
“Our results show that a well-characterized GMP-compliant embryonic stem cell-derived RPE
cells can survive after transplantation to the sub-retinal space of RCS rats, integrate into host
RPE layer without migration into the retina and continue to express at least some of the
molecules characteristic of RPE. The cells are effective in rescuing photoreceptors from
degeneration and result in significant rescue of visual function over sham-operated animals for
the long term.”
CASEY EYE
INSTITUTE
10
Myoblast Program Highlights
Target Market for Myoblast Program
Sufferers of Heart Failure, Chronic Heart
Failure and patients with scarred or
ischemic (dead) heart tissue caused by
or related to heart attack
Program Status
Clearance from FDA to Proceed with
Phase II Clinical Trials in the U.S.
Charlestown, MA GMP Facility
11
Myoblast Program: Clinical Trial Summary
Phase 1
LVAD
Phase 1
CABG
Phase 1b
CABG
Phase 1b
Catheter
Patients 6 patients 12 patients 12 patients
23 Patients
(12 treated,
11 control)
Indication
LVAD Bridge
to Transplant
+CABG +CABG Catheter Injection
Dose
Single Dose -
300 MM Cells
Escalating –
10, 30, 100 and
300 MM Cells
Single Dose -
300 MM Cells
Escalating -
30, 100, 300 and
600 MM Cells
Primary
Endpoint
Safety Safety Safety Safety
Result
Demonstrated
cell survival in
humans
Dose
escalation, Safe
at all doses
Survival of cells
(cardiac
imaging),
improved
symptoms and
function
Safe at all doses,
improved
symptoms and
function
12
Myoblast Program: Phase II Clinical Trial
 80 total patients (1:1 treatment vs. control)
 Double-blind, placebo-controlled (sham procedure)
 Catheter delivery of cells: percutaneous targeted delivery
 Three-month, six-month, and 12-month follow-up
 Endpoints: Improvement in HF symptoms and Ventricular
Volumes
 Primary endpoint: Improvement in heart failure symptoms
measured by “Kansas City Cardiomyopathy Questionnaire”
 Supporting endpoints: Ventricular volumes, Six minute walk test
 Interim data analysis at six-months by Independent Review Board
13
Hemangioblast Program: Partnership
• Joint Venture with leading Korean stem cell
developer CHA Biotech Co.
• The J.V., ‘Stem Cell & Regenerative
Medicine International’, is focused on the
development of human blood cells and
related products
14
Hemangioblast Program: Overview
The HG cell is the precursor to all cell types in the
circulatory and vascular systems
15
Hemangioblast Program: Synthesizing Blood Cells
16
The ACT Therapeutic Timeline
Timetable for Therapeutic Program Milestones Period
Received Orphan indication from Department of Health
and Human Services
Q1 2010
Completion of end of life tumorgenicity & biodistribution
Q1 2010
Receive FDA clearance to commence with Phase I Clinical
Trials
Q3 2010
Treat 1st Patient in history with a hECS-based therapy Q3 2010
1st Quadrant Safety Data on Treated Patients Q4 2010
File 2nd Phase I IND for another Retinal Disease
2nd half
2010
Announce 2nd cellular-based IND in area other than
retinal program
2nd half
2010
studies for RPE
17
The Advanced Cell Technology Team
World Class Scientific Team lead by
Seasoned Management Team:
Dr. Robert Lanza, M.D. – Chief Scientific Officer
Dr. Jonathan Dinsmore, PhD – Myoblast Project Advisor
Edmund Mickunas – Vice President of Regulatory
William Caldwell IV – Chief Executive Officer
Rita Parker – Director of Operations
Roger Gay, PhD – Senior Director of Manufacturing
Matthew Vincent, PhD – IP / Licensing Advisor
18
State of the Company
• Two years of cash available
• Phase I/II trial fully funded
• Anticipating cell lines will qualify for
federal funding
• Actively pursuing development partners
offshore to accelerate development of
programs
19
Thank you for your time
For more information, visit www.advancedcell.com
Advanced Cell Technology is traded on the OTC BB, symbol: ACTC

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BioEurope Presentation, March 2010

  • 1. Advanced Cell Technology Corporate Presentation March 2010 1
  • 2. This presentation is intended to present a summary of ACT’s (“ACT”, or “Advanced Cell Technology Inc”, or “the Company”) salient business characteristics. The information herein contains “forward-looking statements” as defined under the federal securities laws. Actual results could vary materially. Factors that could cause actual results to vary materially are described in our filings with the Securities and Exchange Commission. You should pay particular attention to the “risk factors” contained in documents we file from time to time with the Securities and Exchange Commission. The risks identified therein, as well as others not identified by the Company, could cause the Company’s actual results to differ materially from those expressed in any forward- looking statements. Cautionary Statement Concerning Forward-Looking Statements 2
  • 3. ACT Therapeutics ACT Proprietary Human Therapeutic Programs Treatment Clinical Stage Blastomere Program Development of embryonic stem cell lines without destruction of embryo Pre-Clinical Retinal Pigment Epithelium (RPE) Program Treatment of Age-related Macular Degeneration (AMD) and Retinal Degenerative Diseases Clinical IND awaiting FDA approval Myoblast Program Treatment of Heart Disease, Heart Attack and Heart Failure Phase II Hemangioblast program Treatment of Diseases and Disorders of the Circulatory and Vascular Systems Pre-Clinical 3
  • 4. Pending Multiple next generation technologies complementary to existing patents and claims PendingMultiple use patents for heart disease StatusUse Patents PendingCombinations of myoblasts and stem cells PendingCombinations of myoblasts and muscle survival factors PendingCatheter delivery of cells AwardedMedia for preservation and transplant of muscle cells AwardedStem cells capable of forming nerve or muscle cells AwardedMuscle cells StatusComposition of Matter Patent Intellectual Property Protection ACT owns or licenses over 150 patents and patent applications related to stem cell and regenerative therapies 4
  • 5. Institutional Collaborators Advanced Cell’s Institutional Collaborators include: Casey Eye Institute Mayo Clinic Moran Eye Institute UCSF Harvard Johns Hopkins Stanford Sloan Kettering University of Florida University of Iowa University of Illinois U.C. Berkeley Colorado State University 5
  • 6. Blastomere Program: Synthesizing Stem Cells without Harm to the Embryo •Company scientists successfully generate stem cell lines without destruction of embryo •Cell lines retain potential to form all cells in the human body •Potential to treat a range of human diseases •Technology has been reproduced and peer- reviewed on several occasions •National Institute of Health (NIH) proposing expanded funding regulations to accommodate ACT’s lines 6
  • 7. First Proven Alternative hESC Method •Enables Derivation of New hESC Lines via Pre-implantation Genetic Diagnosis (PGD) Method, Preserving Development Potential of the Embryo •7 hESC lines awaiting NIH approval for funding •Technology is used to develop RPE cells for our clinical trials for Stargardt’s disease Hemangioblasts Differentiated from Blastomere hESC Lines 7
  • 8. RPE Program: Why RPE? • Pigmented epithelium cells are easy to identify • Small dosage vs. other therapies • The eye is an immune-privileged site, thus no risk of rejection at the injection site • Ease of administration – Doesn’t require a separate approval by the FDA (universal applicator) – Procedure is already used by eye surgeons; no new skill set required for doctors • RPE cell therapy may positively impact over 200 retinal diseases 8
  • 9. RPE Program Status Complete Stage 1 of Phase I Clinical Trial for Stargardt’s disease2010 – 4th Quarter Developed the “Proof of Concept” for ACT’s RPE cells in RCS animal model2006 Pre – IND meeting with FDA2008 Filed IND for Stargardt’s disease2009 FDA approval2010 – 3rd Quarter 9
  • 10. GMP-compliant Human RPE Cells Derived from Embryonic Stem Cell Lines Rescue Visual Function In a Rat Model for Photoreceptor Degeneration “Our results show that a well-characterized GMP-compliant embryonic stem cell-derived RPE cells can survive after transplantation to the sub-retinal space of RCS rats, integrate into host RPE layer without migration into the retina and continue to express at least some of the molecules characteristic of RPE. The cells are effective in rescuing photoreceptors from degeneration and result in significant rescue of visual function over sham-operated animals for the long term.” CASEY EYE INSTITUTE 10
  • 11. Myoblast Program Highlights Target Market for Myoblast Program Sufferers of Heart Failure, Chronic Heart Failure and patients with scarred or ischemic (dead) heart tissue caused by or related to heart attack Program Status Clearance from FDA to Proceed with Phase II Clinical Trials in the U.S. Charlestown, MA GMP Facility 11
  • 12. Myoblast Program: Clinical Trial Summary Phase 1 LVAD Phase 1 CABG Phase 1b CABG Phase 1b Catheter Patients 6 patients 12 patients 12 patients 23 Patients (12 treated, 11 control) Indication LVAD Bridge to Transplant +CABG +CABG Catheter Injection Dose Single Dose - 300 MM Cells Escalating – 10, 30, 100 and 300 MM Cells Single Dose - 300 MM Cells Escalating - 30, 100, 300 and 600 MM Cells Primary Endpoint Safety Safety Safety Safety Result Demonstrated cell survival in humans Dose escalation, Safe at all doses Survival of cells (cardiac imaging), improved symptoms and function Safe at all doses, improved symptoms and function 12
  • 13. Myoblast Program: Phase II Clinical Trial  80 total patients (1:1 treatment vs. control)  Double-blind, placebo-controlled (sham procedure)  Catheter delivery of cells: percutaneous targeted delivery  Three-month, six-month, and 12-month follow-up  Endpoints: Improvement in HF symptoms and Ventricular Volumes  Primary endpoint: Improvement in heart failure symptoms measured by “Kansas City Cardiomyopathy Questionnaire”  Supporting endpoints: Ventricular volumes, Six minute walk test  Interim data analysis at six-months by Independent Review Board 13
  • 14. Hemangioblast Program: Partnership • Joint Venture with leading Korean stem cell developer CHA Biotech Co. • The J.V., ‘Stem Cell & Regenerative Medicine International’, is focused on the development of human blood cells and related products 14
  • 15. Hemangioblast Program: Overview The HG cell is the precursor to all cell types in the circulatory and vascular systems 15
  • 17. The ACT Therapeutic Timeline Timetable for Therapeutic Program Milestones Period Received Orphan indication from Department of Health and Human Services Q1 2010 Completion of end of life tumorgenicity & biodistribution Q1 2010 Receive FDA clearance to commence with Phase I Clinical Trials Q3 2010 Treat 1st Patient in history with a hECS-based therapy Q3 2010 1st Quadrant Safety Data on Treated Patients Q4 2010 File 2nd Phase I IND for another Retinal Disease 2nd half 2010 Announce 2nd cellular-based IND in area other than retinal program 2nd half 2010 studies for RPE 17
  • 18. The Advanced Cell Technology Team World Class Scientific Team lead by Seasoned Management Team: Dr. Robert Lanza, M.D. – Chief Scientific Officer Dr. Jonathan Dinsmore, PhD – Myoblast Project Advisor Edmund Mickunas – Vice President of Regulatory William Caldwell IV – Chief Executive Officer Rita Parker – Director of Operations Roger Gay, PhD – Senior Director of Manufacturing Matthew Vincent, PhD – IP / Licensing Advisor 18
  • 19. State of the Company • Two years of cash available • Phase I/II trial fully funded • Anticipating cell lines will qualify for federal funding • Actively pursuing development partners offshore to accelerate development of programs 19
  • 20. Thank you for your time For more information, visit www.advancedcell.com Advanced Cell Technology is traded on the OTC BB, symbol: ACTC