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Advanced Cell Technology
Corporate Presentation
October 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
State of the Company
• Phase I/II ESC trial fully-funded (and not
affected by court ruling) and awaiting FDA
approval
• “Embryo-safe” cell lines may qualify for
federal funding, despite recent court ruling
• Actively pursuing alternatives to
accelerate development of programs
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
4
Robust Patent Portfolio
• RPE Program
• Broad protection for production of RPE cells from human ES Cells
• Includes two issued US Patents
• Cover use of hESC-dervied RPE cells for treatment of retinal
degenerative disorders
• Single Blastomere
• Pending patent application for key technology
• Induced Pluripotency (iPS)
• ACT has earliest priority date to use of key regulatory factors required for
generating iPS cells.
• Transdifferentiation
• Broad filings directed to transdifferentiation without viral vectors
• SCNT
• Dominant issued patents
• Parthenogenesis
• Aquired Infigen patents which are controlling in parthenogenesis
5
Realizing the fruits of more than a
decade of important discoveries….
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
6
• The Sherley v Selebius case presents a major challenge
for the regenerative medicine sector, without a doubt.
• However, ACT has anticipated this development for
some time
• ACT has been taking high-level, face-to-face meetings in
DC with all the relevant players, in both houses and on
both sides of the aisle, as well as with NIH and HHS.
• ACT‟s Single Blastomere technique for isolating
hESC‟s is likely not subject to the language Judge
Lamberth used in Preliminary Injunction.
• ACT stands ready to make these cells available to
the research community, if approved.
7
With Crisis, Comes Opportunity
Current Challenge to hESC Federal Funding
Single Blastomere Technology
• Company scientists successfully generate stem cell lines
without destruction of embryo
• Utilizes PGD extraction of single blastomere
• PGD is safe and routine – has been used in thousands of
pregnancies in United States and Europe alone.
• Cell lines retain potential to form all cells in the human body.
• Resulting human ES cell lines have been demonstrated to be
more robust and reproducible than traditional ICM-derived
lines.
• Technology has been reproduced and peer-reviewed on
several occasions, and is currently being used in preclinical
studies and a product awaiting FDA approval for IND study.
8
Blastomere Program:
hESCs Without Embryo Harm
• Enables Derivation of New hESC Lines via Pre-
implantation Genetic Diagnosis (PGD) Method,
Preserving Development Potential of the Embryo
• Offers source of autologous ES cells for donor
during his/her life, and closely matched allogeneic
source for blood relatives.
• 4 hESC lines awaiting NIH approval for funding – embryos
from which these lines were derived were not destroyed.
• Technology is used to develop RPE cells for our clinical
trials for Juvenile Macular Degeneration. (no Federal
Funding)
• Technology is used to develop Hemangioblasts –
potential source for red blood cells and platelets.
Hemangioblasts
Differentiated from
Blastomere hESC Lines
9
Blastomere Program:
Proven Alternative hESC Method with added Benefits
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
10
ACT’s RPE Program IND - Status
• Received Orphan Indication
• Application for IND Being Reviewed
11
• Initial IND for Stargardt’s Disease
• Trial Design Dovetails Into Second IND, for Dry AMD
• Represents $25-30 Billion Worldwide Market, With No
Effective Therapies Currently Available
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
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
13
Catheter Trial – Responder Analysis
14
NYHA Classification at 6 month vs Baseline for all Patients
Improved No Change Worsened
Myoblast 83% 17% 0%
Control 0% 64% 36%
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
15
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
16
Hemangioblast Program: Overview
The HG cell is the precursor to all cell types in the
circulatory and vascular systems
17
Hemangioblast Program: Synthesizing Blood Cells
18
The Advanced Cell Technology Team
World Class Scientific Team Led By
Seasoned Management Team
Dr. Robert Lanza, M.D. – Chief Scientific Officer
Dr. Jonathan Dinsmore, Ph.D. – Myoblast Project Advisor
Matthew Vincent, Ph.D. – Business Development and IP Strategy
19
William M. Caldwell IV – Chairman & CEO
Edmund Mickunas – Vice President of Regulatory
Roger Gay, PhD – Senior Director of Manufacturing
Rita Parker – Director of Operations
Bill Douglass – Director of Corporate Communications & Social Media
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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ACT Presents Advanced Cell Therapies and Blastomere Stem Cell Technology

  • 1. Advanced Cell Technology Corporate Presentation October 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. State of the Company • Phase I/II ESC trial fully-funded (and not affected by court ruling) and awaiting FDA approval • “Embryo-safe” cell lines may qualify for federal funding, despite recent court ruling • Actively pursuing alternatives to accelerate development of programs 3
  • 4. 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 4
  • 5. Robust Patent Portfolio • RPE Program • Broad protection for production of RPE cells from human ES Cells • Includes two issued US Patents • Cover use of hESC-dervied RPE cells for treatment of retinal degenerative disorders • Single Blastomere • Pending patent application for key technology • Induced Pluripotency (iPS) • ACT has earliest priority date to use of key regulatory factors required for generating iPS cells. • Transdifferentiation • Broad filings directed to transdifferentiation without viral vectors • SCNT • Dominant issued patents • Parthenogenesis • Aquired Infigen patents which are controlling in parthenogenesis 5 Realizing the fruits of more than a decade of important discoveries….
  • 6. 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 6
  • 7. • The Sherley v Selebius case presents a major challenge for the regenerative medicine sector, without a doubt. • However, ACT has anticipated this development for some time • ACT has been taking high-level, face-to-face meetings in DC with all the relevant players, in both houses and on both sides of the aisle, as well as with NIH and HHS. • ACT‟s Single Blastomere technique for isolating hESC‟s is likely not subject to the language Judge Lamberth used in Preliminary Injunction. • ACT stands ready to make these cells available to the research community, if approved. 7 With Crisis, Comes Opportunity Current Challenge to hESC Federal Funding
  • 8. Single Blastomere Technology • Company scientists successfully generate stem cell lines without destruction of embryo • Utilizes PGD extraction of single blastomere • PGD is safe and routine – has been used in thousands of pregnancies in United States and Europe alone. • Cell lines retain potential to form all cells in the human body. • Resulting human ES cell lines have been demonstrated to be more robust and reproducible than traditional ICM-derived lines. • Technology has been reproduced and peer-reviewed on several occasions, and is currently being used in preclinical studies and a product awaiting FDA approval for IND study. 8 Blastomere Program: hESCs Without Embryo Harm
  • 9. • Enables Derivation of New hESC Lines via Pre- implantation Genetic Diagnosis (PGD) Method, Preserving Development Potential of the Embryo • Offers source of autologous ES cells for donor during his/her life, and closely matched allogeneic source for blood relatives. • 4 hESC lines awaiting NIH approval for funding – embryos from which these lines were derived were not destroyed. • Technology is used to develop RPE cells for our clinical trials for Juvenile Macular Degeneration. (no Federal Funding) • Technology is used to develop Hemangioblasts – potential source for red blood cells and platelets. Hemangioblasts Differentiated from Blastomere hESC Lines 9 Blastomere Program: Proven Alternative hESC Method with added Benefits
  • 10. 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 10
  • 11. ACT’s RPE Program IND - Status • Received Orphan Indication • Application for IND Being Reviewed 11 • Initial IND for Stargardt’s Disease • Trial Design Dovetails Into Second IND, for Dry AMD • Represents $25-30 Billion Worldwide Market, With No Effective Therapies Currently Available
  • 12. 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 12
  • 13. 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 13
  • 14. Catheter Trial – Responder Analysis 14 NYHA Classification at 6 month vs Baseline for all Patients Improved No Change Worsened Myoblast 83% 17% 0% Control 0% 64% 36%
  • 15. 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 15
  • 16. 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 16
  • 17. Hemangioblast Program: Overview The HG cell is the precursor to all cell types in the circulatory and vascular systems 17
  • 19. The Advanced Cell Technology Team World Class Scientific Team Led By Seasoned Management Team Dr. Robert Lanza, M.D. – Chief Scientific Officer Dr. Jonathan Dinsmore, Ph.D. – Myoblast Project Advisor Matthew Vincent, Ph.D. – Business Development and IP Strategy 19 William M. Caldwell IV – Chairman & CEO Edmund Mickunas – Vice President of Regulatory Roger Gay, PhD – Senior Director of Manufacturing Rita Parker – Director of Operations Bill Douglass – Director of Corporate Communications & Social Media
  • 20. Thank you for your time For more information, visit www.advancedcell.com Advanced Cell Technology is traded on the OTC BB, symbol: ACTC