ACT is conducting three clinical trials for dry age-related macular degeneration (AMD) and Stargardt's disease (SMD) using retinal pigment epithelium (RPE) cells derived from human embryonic stem cells. The trials have shown no adverse events and persistence of the transplanted cells, with functional vision improvements in most patients. ACT has additional clinical programs planned or underway for myopic macular degeneration and has a pipeline of other ophthalmology and regenerative medicine programs. It has strong intellectual property around RPE cell production and therapy. Upcoming milestones include further patient follow-up data and the potential start of Phase II trials. ACT is led by an experienced management team and board of directors.
2. 2
Cautionary Statement Concerning Forward-Looking Statements
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. Ropes Gray
3. 3
Three clinical trials aimed at very large unmet medical needs
• Dry Age related Macular Degeneration and Stargardt’s Disease affect nearly 30 million
people in the US and EU, expected to grow by 50% over next 10 years
World-class cell biology capabilities
• ACT scientific team continues to develop best-in-class, and only-in-class cell lines which
create several preclinical opportunities across many disease areas
Strong Intellectual Property Estate
• 190 patents filed, 37 issued which provide ACT with sustainable competitive advantage
for decades to come
Active partnering program to monetize non-core assets
• ACT management is dedicated to monetizing non-core pre-clinical assets and
continues to optimize portfolio of opportunities to fuel future clinical programs
Several Key Inflection Points Provide Near-term
and Long-term Growth Opportunities
4. 4
Pre-clinical/
in vitro
POC –
Animal Studies IND Approved Phase I Phase II Phase III Approval
Dry AMD
SMD
MMD
Photo-
receptors
Ganglion
Neurons
Cornea
Platelets
Mesenchymal
Stem Cells
Robust Development Pipeline Provides Multiple Opportunities to
Commercialize and Partner
Potential Gov’t Funding
First Priority Based
On Current
Funding
Advance into
Phase I and
Partner
Based on POC results,
pursue appropriate
funding and collaborations
OphthalmologyPrograms
5. The Retina the light-sensitive
tissue lining the inner surface of
the eye
Retina
Structure of the Retina
5
6. Provides nutrients and growth factors
• photoreceptors see no blood
Recycles Vitamin A
• maintains photoreceptor excitability
Detoxifies photoreceptor layer
Maintains Bruch’s Membrane
• natural antiangiogenic barrier
• immune privilege of retina
Absorbs stray light / protects from UV
RPE Layer has
multiple
critical roles
in the
health and
function
of photoreceptors and
the retina as a whole.
Life Support for Photoreceptors
6
7. Failure of RPE cells
results in many
degenerative diseases
Stargardt’s disease
Myopic Macular Dystrophy
Age-related macular degeneration (AMD)
Life Support for Photoreceptors
7
8. 8
Prevalence of AMD Increases Exponentially with Age
Data from http://www.nei.nih.gov/eyedata/ and
U.S. Census Bureau Publication “65+ in the United States”, P23-209
Exponential rise in prevalence and incidence
rates with age, with prevalence rates of late
AMD quadrupling per decade
133.7 142.2 173.4 202.7 207.4 205.5
37.4 52.9
64.8
81.9 105.3 121
80+
65-79
2000 2010 2020 2030 2040 2050
Developed Countries
“macular degeneration will soon
take on aspects of an epidemic”
- former Director of the National Eye Institute Dr Carl Kupfer
60%
50%
40%
30%
20%
10%
50-5940-49 60-69 70-79 80+
Age
Intermediate AMD
Late AMD
%Prevalence(U.S.)
9. 9
Prevalence of AMD Increases Exponentially with Age
Exponential rise in prevalence and incidence
rates with age, with prevalence rates of late
AMD quadrupling per decade
The currently 30 Million American and
European AMD patients are projected
to become 45 Million patients by 2025
10. RPE Therapy- Rationale
• Massive unmet medical need
• Small dosage size
– less than 200K cells
• Immune-privileged site
– minimal immunosuppression
• Ease of administration
– no separate device approval
• Unique measuring and observation environment
10
11. Surgical Overview
11
Procedure:
• 25 Gauge Pars Plana Vitrectomy
• Posterior Vitreous Separation
• Subretinal hESC-derived RPE cells
injection
• Bleb Confirmation
• Day Surgery/Sedation only
12. Jules Stein
(UCLA)
Mass
Eye & Ear
Infirmary
Wills Eye
Institute
Bascom
Palmer Eye
Institute
Moorfields
Eye
Hospital
Edinburgh
Royal
Infirmary
World renowned leadership to help us navigate the
clinical path and ultimately support market launch
Clinical Trials being led by World Leaders in Ophthalmology
12
13. • No Adverse Events
• Persistence of cells
• Impact on Acuity
Recorded functional visual
improvements in majority of patients.
• Increased letters on ETDRS
Charts
• Color perception
• Contrast
• Low light vision
Phase I Trials Exceeding Expectations – no adverse events and persistence of cells
13
14. IND
Approved
50% Patient
Enrollment
100% Patient
Enrollment
U.S. – Dry AMD
U.S. – SMD
U.K. – SMD
U.S. – MMD
12/16 patients treated
10/16 patients treated
8/12 patients treated
Enrolling – 12 patients total
2 years since the first patients were treated
Measurable Improvements in Visual
Acuity for Majority of Treated Patients
Active Clinical Programs in AMD and SMD Indicate Encouraging Results
14
15. January 2013: FDA approved additional 4 patient “better vision”
cohorts in each trial.
For Cohort 2a – can enroll patients with vision as
good as 20/100.
Cohort 1
50K Cells
Cohort 2
100K Cells
Cohort 3
150K Cells
Cohort 4
200K Cells
Cohort 2a
100K Cells
First Treatments informed a more aggressive strategy to treat “better vision” cohort, could
lead to broader label and/or earlier approval
15
16. Several Important clinical milestones 2H 2013, 1H 2014
150K 200K
Cohort 2a
100K
Jan 2014 Jan 2015
Dry AMD
& SMD Trials
Patient follow-up
PII design Patient TreatmentPhase II
Patient TreatmentMMD Trial
16
Sep 2013 July 2014
Phase I
Phase I
17. Expanding Clinical Programs
17
Myopia creates a higher risk of permanent vision loss due
to Myopic Macular Degeneration (MMD)
• Severe near-sightedness causes elongation of the eyeball --
which can cause fissures in RPE layer.
January 2013 - FDA Approved
MMD Phase I/II study
Jules Stein Eye Institute (UCLA) and ACT
18. Intellectual Property – RPE Program
Dominant Patent Position for Treating Retinal Degeneration
Broad Coverage for Manufacturing RPE Cells
Broad protection of pharmaceutical preparations
• RPE cell suspensions
• scaffolded RPE layers.
RPE Cells derived from other pluripotent stem cells
Vigilant filing on improvements
18
20. 20
BY PRODUCING THE
HIGHEST DEGREE OF QUALITY
VETERINARY THERAPEUTICS
WE INTEND TO BE
A LEADER IN
VETERINARY
REGENERATIVE MEDICINE
September 19, 2013 –
“ACT Files Investigational New Animal
Drug (INAD) Application with FDA to Treat
10 Different Disease Indications Using
Pluripotent Stem Cells”
22. Gary Rabin – Chairman and CEO
Edward Myles – CFO and EVP of Corp Development
Dr. Matthew Vincent, Ph.D. – Director of Business
Development
Dr. Robert Lanza, MD – Chief Scientific Officer
Dr. Irina Klimanskaya, Ph.D. – Director of Stem Cell
Biology
Dr. Shi-Jiang (John) Lu, Ph.D. – Senior Director of
Research
Edmund Mickunas – Vice President of Regulatory
Affairs
Dr. Roger Gay, Ph.D. - Senior Director of Manufacturing
Proven business leaders who can
develop and implement corporate
strategy and monetize assets to
maximize shareholder value
World-renowned scientific thought
leaders pushing the cutting edge of
science to develop important
therapies
Deep experience navigating the
complex regulatory landscape from
development to market
GMP manufacturing to ensure the highest quality
products are delivered to our patients
An Experienced and Dedicated Management Team
22
23. Michael Heffernan CEO – Collegium Pharmaceuticals
Robert S. Langer, Sc.D. Institute Professor, MIT
Zohar Loshitzer
CEO – Presbia, Inc., & Principal in
Ochard Capital
Greg Perry EVP & CFO - Immunogen
Alan C. Shapiro
Finance Professor and Chairman of the
Department of Finance and Business
Economics (retired) – University of
Southern California
Gary Rabin
Chairman and CEO – Advanced Cell
Technology
A World-Class Board of Directors
23