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LEADING
REGENERATIVE
MEDICINE
May 2014
presented at:
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. Ropes Gray
Cautionary Statement Concerning Forward-Looking Statements
3
Ophthalmology
Autoimmune/
Inflammation
Drug Delivery
Transplantable
Photoreceptor Progenitors
Transplantable
Ganglion Nerve Progenitors
DSEK Corneal Transplant Tissue
Neuroprotective/Neuroregulatory
Biologics
Multiple Sclerosis
Alzheimer’s Disease
Inflammatory Bowel Disease
Sepsis
Wound Healing
Engineered Platelets & MK
• Factor XIII • Wound Healing
• Anti-cancer
Engineered RPE
Robust Clinical and Preclinical Programs
Lupus
Engineered Photoreceptors
Transplantable
RPE Cells
Autoimmune/
Inflammation
Drug Delivery
Multiple Sclerosis
Alzheimer’s Disease
Inflammatory Bowel Disease
Sepsis
Wound Healing
Engineered Platelets & MK
• Factor XIII • Wound Healing
• Anti-cancer
Engineered RPE
Lupus
Engineered Photoreceptors
4
Ophthalmology
Transplantable
Photoreceptor Progenitors
Transplantable
Ganglion Nerve Progenitors
DSEK Corneal Transplant Tissue
Neuroprotective/Neuroregulatory
Biologics
Robust Clinical and Preclinical Programs
Transplantable
RPE Cells
Pre-clinical Development Phase 1 Phase 2
Transplantable
Photoreceptor Progenitors
Transplantable
Ganglion Nerve Progenitors
DSEK Corneal Transplant Tissue
Neuroprotective/Neuroregulatory
Biologics
Dry Age-Related Macular
Degeneration (US)
Stargardt’s Macular
Dystrophy (US & UK)
Myopic Macular
Degeneration (US)
Dry AMD (US)
Projected 2H-2014/1H-2015
SMD (US & UK)
Projected 2H-2014
Advanced Cell Technology’s
Regenerative Ophthalmology Programs
focus on preserving and repairing
neurosensory retina and cornea function
TM
Engineered RPE
Retina
Structure of the Retina
5
6
Life Support to Photoreceptors
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.
7
Life Support to Photoreceptors
Failure of RPE cells
results in many
degenerative diseases
Stargardt’s disease (SMD)
Myopic Macular Dystrophy (MMD)
Age-related macular degeneration (AMD)
Age-Related Macular Degeneration will Soon Take on Aspects of an Epidemic
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.
Prevalence rates of late
AMD quadruple per
decade
40%
30%
20%
10%
50-5940-49 60-69 70-79 80+
Age
Intermediate AMD
Late AMD
%Prevalence(U.S.)
8
The projected number of people worldwide with age-
related macular degeneration in 2020 is 196 million,
increasing to 288 million in 2040 - Wong et al. Lancet January 2014
Dissociated RPE Cells can be injected into the subretinal
space and will rebuild functional RPE monolayer where
native RPE is compromised or lost.
• Simple Handling
• Optimized for large scale and centralized manufacturing
• Small Dose Size translates into ACT’s ability to manufacture
50,000-100,000 per year in existing facilities.
Therapeutic Thesis
9
Product Cold Chain
is Easily Scaled for
Global Sales
• RPE layer is surgically accessible -
- least invasive means to locally deliver cells
• Adult RPE translocation studies included
dissociated cell formats
• hESC-derived RPE supported by both In Vitro
and In Vivo Studies at ACT and OHSU
Cell Therapy for RPE, Achievable by a Small Company
10
Small dosage size
• less than 200K cells
Relatively Immune-privileged site
• minimal immunosuppression
Ease of administration
• no separate device approval
Unique measuring and observation environment
• measurable anatomical and functional endpoints
January 2013: FDA approved additional 4 subject “better vision”
cohorts in each trial.
For Cohort 2a – can enroll subjects 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
11
4 Approved Trials – 3 U.S. and 1 U.K.
Initial Trial Design is Ascending Dosage Safety Study
12 Subjects – Four Cohorts of 3 subjects each
Inclusion Criteria
BCVA: 20/800 or worse
Inclusion Criteria
BCVA: 20/400 or worse
Inclusion Criteria
BCVA: 20/100 or worse
Inclusion Criteria
BCVA: 20/400 or worse
Inclusion Criteria
BCVA: 20/400 or worse
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
12/16 patients treated
10/12 patients treated
Enrolling – 12 patients total
Current Status of P1 Trials enables Planning for P2
34 Patients Treated to Date
12
Early Hypothesis Developed for PII,
Vetted with Investigators, Dialoging with MHRA and FDA
Jules Stein
(UCLA)
Mass
Eye & Ear
Infirmary
Wills Eye
Institute
Bascom
Palmer Eye
Institute
Moorfields
Eye
Hospital
Edinburgh
Royal
Infirmary
Clinical Trials being led by World Leaders in Ophthalmology
13
World Renowned Leadership to help us navigate the Clinical
Path and Ultimately Support Market Launch
Delivery of Cells Builds on Common Surgery
14
Procedure:
• 25 Gauge Pars Plana Vitrectomy
• Posterior Vitreous Separation
• Subretinal hESC-derived RPE cells injection
• Bleb Confirmation
• Day Surgery/Sedation only
Vitrectomy is the 3rd Most Frequently
Performed Ophthalmic Surgery; 1.1m
globally, 300k in US, per year
Elapsed Time Since Transplant – as May 2014
15
US
Dry AMD
US
SMD
UK
SMD
Interim Data Analysis
Interim Data Analysis
2 years 3 years1 year
* US AMD & SMD Cohort 2a
subjects range from 1-12 months
post-transplant
Cohort
2a
Overview of Interim Data
16
Manuscript detailing interim data under review at impactful journal
Co-authored by participating surgeons and company
o Clean safety profile
o Evidence of engraftment and persistence of transplanted RPE cells
o Persisting Improvement of Visual Acuity for many patients
A number of patients with clinically significant improvements in BCVA
AMD-207 improved 20/400 to 20/40
• Improvement has persisted through follow-up visits (> 1 year)
SMD-001 improved from HM to 20/500
• Improvement (with further gain) has persisted through follow-up visits (>2 year)
Based on advice from all 5 clinical trial sites and participating surgeons, as
well as input from regulators, we are advancing to phase 2 studies
› Active dialog with MHRA and FDA
› Anticipate P2 commencement in 2H2014
Exemplars of
Patients With
Improved
Visual Acuity
Expanding RPE Program to Address an Additional Leading Cause of Blindness
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.
As the cause of legal blindness, MMD is ranked:
• 7th in the United Sates
• 4th in Hong Kong
• 2nd in mainland China and Japan
Jules Stein Eye (UCLA) and ACT to Initiate Phase I/II Study
Second Generation RPE Cell Therapy Products
By engineering the master stem cell bank used to manufacture RPE
cells, the transplanted RPE cells can express
• Anti-angiogeneic agents
Reduce occurrence of choroidal neovascularization (wet AMD).
• Complement factor D, Factor C5 and/or Factor C3 Inhibitors
Activation of alternative complement pathway implicated in disease
progression for certain patients
• Anti b-amyloid agents
Drusen deposits resemble amyloid deposits.
• Anti-Inflammatory agents
IL-1, IL-2, IL-3, and TNF-α antagonists
Recombinant Lipocortin – a potent anti-inflammatory protein
18
Intellectual Property – RPE Program
Dominant Patent Positions in Major Markets
for Platform Technology and Treatments
• Broad Coverage for Manufacturing RPE Cells
• Broad protection of pharmaceutical preparations of RPE cells
Covers RPE cell suspensions for injection
Covers scaffolded RPE layers for implantation
Covers polarized monolayers of cells
• Covers RPE Cells derived from any pluripotent stem cell source
- From hESC to iPS cells to pluripotent stem cells yet to be invented
19
First Mover and First-in-Clinic Efforts
Translate into Broad First-to-File Patents
Every practical
formulation format for
use in human patients
20
Therapeutic Pipeline -
Other Ocular Programs
Retinal Pigment Epithelial Cells
 Macular Degeneration - dry AMD
 Retinitis Pigmentosa
 Photoreceptor protection
Hemangioblast cells
 Ischemic retinopathy
– diabetic retinopathy, vascular occlusions
Retinal Neural Progenitor cells
Isolated Protective Factors
 Photoreceptor Loss, Modulation of Müller Cells
 Protection of Retinal Ganglion cells (Glaucoma)
Corneal Endothelium, Corneal Epithelium,
Descemet’s Membrane
 Corneal Disease
Mesenchymal Stromal Cells
 Glaucoma, Uveitis
 Retinitis Pigmentosa
 Management of Ocular Surfaces
21
Retina
Ophthalmology Pipeline Extends Beyond RPE
22
Pluripotent
Stem Cell
Eye Field
Stem Cell
RPE
Photoreceptor
Ganglion
Proprietary Methods for Deriving Various Cell Types of the Retina
Purity of Resulting
Cell Preparations
Approaches 100%
23
Cell lines
Percentage of PAX6
+ neural stem cells
Percentage of Nestin
+ neural stem cells
ES lines (N=3) 92%-98% 95%-99%
ES lines, blastomere technology
(N=2)
94%-99% 95%-99%
iPS lines, Episomal Vector (N=4) 90%-99.6% 96%-100%
iPS lines, mRNA (N=2) 92%-98% 96%-100%
• Evaluated in multiple hESC and iPS cells lines
• Highly synchronized culture
Highly Reproducible with Various Pluripotent Stem Cell Sources
24
Neurosensory Retina – Photoreceptor Replacement
We have demonstrated that
subretinally injected hESC-
and iPSC-derived photoreceptor progenitors can
migrate to correct anatomical site in retina, differentiate,
and functionally rescue vision.
Carrying out disease model POC studies for demonstrating utility in
treatment of:
• Late stage macular degeneration
• Retinitis Pigmentosa (RP)
• Night Blindness
Unique photoreceptor progenitor
(“PhRP-x”) maintains plasticity to form
both rods and cones
25
When delivered systemically
• Scotopic (shown) and Phototopic ERG
preservation of rod and cone function
• ERG recovery is cell-dependent (CSA
removal reverses ERG recovery)
• Also observed preservation of ONL
thickness relative to control.
Only the PhRP-x progenitors have
paracrine neuroprotective activity
– earlier stage high purity retinal
progenitors do not.
Neurosensory Retina – Neuroprotection
A differentiated functional phenotype
PhRP-x are photoreceptor progenitors which:
• form both rods and cones
• functionally integrate in ONL
• secrete a neuroprotective agent(s)
• have phagocytic activity for OS
Edward Myles – Interim President, CFO and EVP of Corp
Development
Dr. Matthew Vincent, Ph.D. – Dir., Business Development
Dr. Robert Lanza, MD – Chief Scientific Officer
Dr. Irina Klimanskaya, Ph.D. – Dir., Stem Cell Biology
Dr. Shi-Jiang (John) Lu, Ph.D. – Senior Director of Research
Eddy Anglade, M.D. – EVP, Clinical Development
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 in clinical development
programs for ophthalmology drug products from
early through late-and post-marketing stages
GMP manufacturing to ensure the highest quality
products are delivered to our patients
An Experienced and Dedicated Management Team
26
Michael Heffernan, Chairman CEO – Collegium Pharmaceuticals
Robert S. Langer, Sc.D. Institute Professor, MIT
Zohar Loshitzer
CEO – Presbia, Inc., & Principal in
Orchard Capital
Greg Perry EVP & CFO – Eleven Biotherapeutics
Alan C. Shapiro
Finance Professor and Chairman of the
Department of Finance and Business
Economics (retired) – University of
Southern California
A World-Class Board of Directors
27

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15.15 matthew vincent

  • 2. 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. Ropes Gray Cautionary Statement Concerning Forward-Looking Statements
  • 3. 3 Ophthalmology Autoimmune/ Inflammation Drug Delivery Transplantable Photoreceptor Progenitors Transplantable Ganglion Nerve Progenitors DSEK Corneal Transplant Tissue Neuroprotective/Neuroregulatory Biologics Multiple Sclerosis Alzheimer’s Disease Inflammatory Bowel Disease Sepsis Wound Healing Engineered Platelets & MK • Factor XIII • Wound Healing • Anti-cancer Engineered RPE Robust Clinical and Preclinical Programs Lupus Engineered Photoreceptors Transplantable RPE Cells
  • 4. Autoimmune/ Inflammation Drug Delivery Multiple Sclerosis Alzheimer’s Disease Inflammatory Bowel Disease Sepsis Wound Healing Engineered Platelets & MK • Factor XIII • Wound Healing • Anti-cancer Engineered RPE Lupus Engineered Photoreceptors 4 Ophthalmology Transplantable Photoreceptor Progenitors Transplantable Ganglion Nerve Progenitors DSEK Corneal Transplant Tissue Neuroprotective/Neuroregulatory Biologics Robust Clinical and Preclinical Programs Transplantable RPE Cells Pre-clinical Development Phase 1 Phase 2 Transplantable Photoreceptor Progenitors Transplantable Ganglion Nerve Progenitors DSEK Corneal Transplant Tissue Neuroprotective/Neuroregulatory Biologics Dry Age-Related Macular Degeneration (US) Stargardt’s Macular Dystrophy (US & UK) Myopic Macular Degeneration (US) Dry AMD (US) Projected 2H-2014/1H-2015 SMD (US & UK) Projected 2H-2014 Advanced Cell Technology’s Regenerative Ophthalmology Programs focus on preserving and repairing neurosensory retina and cornea function TM Engineered RPE
  • 6. 6 Life Support to Photoreceptors 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.
  • 7. 7 Life Support to Photoreceptors Failure of RPE cells results in many degenerative diseases Stargardt’s disease (SMD) Myopic Macular Dystrophy (MMD) Age-related macular degeneration (AMD)
  • 8. Age-Related Macular Degeneration will Soon Take on Aspects of an Epidemic 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. Prevalence rates of late AMD quadruple per decade 40% 30% 20% 10% 50-5940-49 60-69 70-79 80+ Age Intermediate AMD Late AMD %Prevalence(U.S.) 8 The projected number of people worldwide with age- related macular degeneration in 2020 is 196 million, increasing to 288 million in 2040 - Wong et al. Lancet January 2014
  • 9. Dissociated RPE Cells can be injected into the subretinal space and will rebuild functional RPE monolayer where native RPE is compromised or lost. • Simple Handling • Optimized for large scale and centralized manufacturing • Small Dose Size translates into ACT’s ability to manufacture 50,000-100,000 per year in existing facilities. Therapeutic Thesis 9 Product Cold Chain is Easily Scaled for Global Sales • RPE layer is surgically accessible - - least invasive means to locally deliver cells • Adult RPE translocation studies included dissociated cell formats • hESC-derived RPE supported by both In Vitro and In Vivo Studies at ACT and OHSU
  • 10. Cell Therapy for RPE, Achievable by a Small Company 10 Small dosage size • less than 200K cells Relatively Immune-privileged site • minimal immunosuppression Ease of administration • no separate device approval Unique measuring and observation environment • measurable anatomical and functional endpoints
  • 11. January 2013: FDA approved additional 4 subject “better vision” cohorts in each trial. For Cohort 2a – can enroll subjects 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 11 4 Approved Trials – 3 U.S. and 1 U.K. Initial Trial Design is Ascending Dosage Safety Study 12 Subjects – Four Cohorts of 3 subjects each Inclusion Criteria BCVA: 20/800 or worse Inclusion Criteria BCVA: 20/400 or worse Inclusion Criteria BCVA: 20/100 or worse Inclusion Criteria BCVA: 20/400 or worse Inclusion Criteria BCVA: 20/400 or worse
  • 12. 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 12/16 patients treated 10/12 patients treated Enrolling – 12 patients total Current Status of P1 Trials enables Planning for P2 34 Patients Treated to Date 12 Early Hypothesis Developed for PII, Vetted with Investigators, Dialoging with MHRA and FDA
  • 13. Jules Stein (UCLA) Mass Eye & Ear Infirmary Wills Eye Institute Bascom Palmer Eye Institute Moorfields Eye Hospital Edinburgh Royal Infirmary Clinical Trials being led by World Leaders in Ophthalmology 13 World Renowned Leadership to help us navigate the Clinical Path and Ultimately Support Market Launch
  • 14. Delivery of Cells Builds on Common Surgery 14 Procedure: • 25 Gauge Pars Plana Vitrectomy • Posterior Vitreous Separation • Subretinal hESC-derived RPE cells injection • Bleb Confirmation • Day Surgery/Sedation only Vitrectomy is the 3rd Most Frequently Performed Ophthalmic Surgery; 1.1m globally, 300k in US, per year
  • 15. Elapsed Time Since Transplant – as May 2014 15 US Dry AMD US SMD UK SMD Interim Data Analysis Interim Data Analysis 2 years 3 years1 year * US AMD & SMD Cohort 2a subjects range from 1-12 months post-transplant Cohort 2a
  • 16. Overview of Interim Data 16 Manuscript detailing interim data under review at impactful journal Co-authored by participating surgeons and company o Clean safety profile o Evidence of engraftment and persistence of transplanted RPE cells o Persisting Improvement of Visual Acuity for many patients A number of patients with clinically significant improvements in BCVA AMD-207 improved 20/400 to 20/40 • Improvement has persisted through follow-up visits (> 1 year) SMD-001 improved from HM to 20/500 • Improvement (with further gain) has persisted through follow-up visits (>2 year) Based on advice from all 5 clinical trial sites and participating surgeons, as well as input from regulators, we are advancing to phase 2 studies › Active dialog with MHRA and FDA › Anticipate P2 commencement in 2H2014 Exemplars of Patients With Improved Visual Acuity
  • 17. Expanding RPE Program to Address an Additional Leading Cause of Blindness 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. As the cause of legal blindness, MMD is ranked: • 7th in the United Sates • 4th in Hong Kong • 2nd in mainland China and Japan Jules Stein Eye (UCLA) and ACT to Initiate Phase I/II Study
  • 18. Second Generation RPE Cell Therapy Products By engineering the master stem cell bank used to manufacture RPE cells, the transplanted RPE cells can express • Anti-angiogeneic agents Reduce occurrence of choroidal neovascularization (wet AMD). • Complement factor D, Factor C5 and/or Factor C3 Inhibitors Activation of alternative complement pathway implicated in disease progression for certain patients • Anti b-amyloid agents Drusen deposits resemble amyloid deposits. • Anti-Inflammatory agents IL-1, IL-2, IL-3, and TNF-α antagonists Recombinant Lipocortin – a potent anti-inflammatory protein 18
  • 19. Intellectual Property – RPE Program Dominant Patent Positions in Major Markets for Platform Technology and Treatments • Broad Coverage for Manufacturing RPE Cells • Broad protection of pharmaceutical preparations of RPE cells Covers RPE cell suspensions for injection Covers scaffolded RPE layers for implantation Covers polarized monolayers of cells • Covers RPE Cells derived from any pluripotent stem cell source - From hESC to iPS cells to pluripotent stem cells yet to be invented 19 First Mover and First-in-Clinic Efforts Translate into Broad First-to-File Patents Every practical formulation format for use in human patients
  • 21. Retinal Pigment Epithelial Cells  Macular Degeneration - dry AMD  Retinitis Pigmentosa  Photoreceptor protection Hemangioblast cells  Ischemic retinopathy – diabetic retinopathy, vascular occlusions Retinal Neural Progenitor cells Isolated Protective Factors  Photoreceptor Loss, Modulation of Müller Cells  Protection of Retinal Ganglion cells (Glaucoma) Corneal Endothelium, Corneal Epithelium, Descemet’s Membrane  Corneal Disease Mesenchymal Stromal Cells  Glaucoma, Uveitis  Retinitis Pigmentosa  Management of Ocular Surfaces 21 Retina Ophthalmology Pipeline Extends Beyond RPE
  • 22. 22 Pluripotent Stem Cell Eye Field Stem Cell RPE Photoreceptor Ganglion Proprietary Methods for Deriving Various Cell Types of the Retina Purity of Resulting Cell Preparations Approaches 100%
  • 23. 23 Cell lines Percentage of PAX6 + neural stem cells Percentage of Nestin + neural stem cells ES lines (N=3) 92%-98% 95%-99% ES lines, blastomere technology (N=2) 94%-99% 95%-99% iPS lines, Episomal Vector (N=4) 90%-99.6% 96%-100% iPS lines, mRNA (N=2) 92%-98% 96%-100% • Evaluated in multiple hESC and iPS cells lines • Highly synchronized culture Highly Reproducible with Various Pluripotent Stem Cell Sources
  • 24. 24 Neurosensory Retina – Photoreceptor Replacement We have demonstrated that subretinally injected hESC- and iPSC-derived photoreceptor progenitors can migrate to correct anatomical site in retina, differentiate, and functionally rescue vision. Carrying out disease model POC studies for demonstrating utility in treatment of: • Late stage macular degeneration • Retinitis Pigmentosa (RP) • Night Blindness Unique photoreceptor progenitor (“PhRP-x”) maintains plasticity to form both rods and cones
  • 25. 25 When delivered systemically • Scotopic (shown) and Phototopic ERG preservation of rod and cone function • ERG recovery is cell-dependent (CSA removal reverses ERG recovery) • Also observed preservation of ONL thickness relative to control. Only the PhRP-x progenitors have paracrine neuroprotective activity – earlier stage high purity retinal progenitors do not. Neurosensory Retina – Neuroprotection A differentiated functional phenotype PhRP-x are photoreceptor progenitors which: • form both rods and cones • functionally integrate in ONL • secrete a neuroprotective agent(s) • have phagocytic activity for OS
  • 26. Edward Myles – Interim President, CFO and EVP of Corp Development Dr. Matthew Vincent, Ph.D. – Dir., Business Development Dr. Robert Lanza, MD – Chief Scientific Officer Dr. Irina Klimanskaya, Ph.D. – Dir., Stem Cell Biology Dr. Shi-Jiang (John) Lu, Ph.D. – Senior Director of Research Eddy Anglade, M.D. – EVP, Clinical Development 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 in clinical development programs for ophthalmology drug products from early through late-and post-marketing stages GMP manufacturing to ensure the highest quality products are delivered to our patients An Experienced and Dedicated Management Team 26
  • 27. Michael Heffernan, Chairman CEO – Collegium Pharmaceuticals Robert S. Langer, Sc.D. Institute Professor, MIT Zohar Loshitzer CEO – Presbia, Inc., & Principal in Orchard Capital Greg Perry EVP & CFO – Eleven Biotherapeutics Alan C. Shapiro Finance Professor and Chairman of the Department of Finance and Business Economics (retired) – University of Southern California A World-Class Board of Directors 27