rodman conference, september 2012

31
Leading Regenerative Medicine Rodman & Renshaw Global Investment Conference September 2012

Upload: advanced-cell-technology-inc

Post on 08-Sep-2014

224 views

Category:

Technology


0 download

DESCRIPTION

Rodman & Renshaw Global Investment Conference September 2012 9/10/2012

TRANSCRIPT

Page 1: Rodman Conference, September 2012

Leading Regenerative Medicine

Rodman & Renshaw Global Investment Conference September 2012

Page 2: Rodman Conference, September 2012

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

2

Page 3: Rodman Conference, September 2012

Multiple Pluripotent Cell Platforms • Single Blastomere-derived Embryonic Stem Cells

• Generating hESC without Destruction of Embryo • Utilizes a single cell biopsy • Our hESC lines are pluripotent – can make any cell in the body.

• Induced Pluripotency Stem Cells (iPS)

• Early Innovator in Pluripotency (before iPS was even a term!) • Recipient of National Institutes of Health Director's Opportunity Award • Seminal paper identifying replicative senescence issue for vector-derived iPS cells • Leading publication on protein induced iPS lines - avoids genetic manipulation with nucleic acid vectors • Controlling Filings (earliest priority date) to use of OCT4 for inducing pluripotency

3

Final Product Definition: hESC-derived products will be manufactured using a cell line made in 2005 from single cell isolated without the destruction of any embryos

Page 4: Rodman Conference, September 2012

RPE Clllllll Pllllll

Page 5: Rodman Conference, September 2012

5

retina

Life Support to Photoreceptors

Page 6: Rodman Conference, September 2012

6

Life Support to Photoreceptors

Detoxifies photoreceptor layer Maintains Bruch’s Membrane

• natural antiangiogenic barrier • prevents abnormal blood vessel growth • immune privilege of retina

Absorbs stray light and protects from UV

Provides critical nutrients, growth factors, ions and water

• photoreceptors see no blood Recycles Vitamin A

• maintains photoreceptor excitability Function of

RPE Layer

Page 7: Rodman Conference, September 2012

7

RPE Cells Therapy

Early Stage AMD (10-15M)

Intermediate AMD (5-8M)

Late Stage AMD (1.75M)

U.S. Patient Population

ACT’s RPE Cell Therapy should address the full range of dry AMD patients. • Halt progression of vision loss in early stage

patients • Restore some visual acuity in later stage

patients Dry AMD represents more than 90 percent of all cases of AMD North America and Europe alone have more than 30 Million dry AMD patients who should be eligible for our RPE cell therapy

On the Rise: Population demographics (“baby boomers”) combined with increased longevity predicts an increase of 50 percent or more in the incidence rate of AMD.

Page 8: Rodman Conference, September 2012

RPE Engraftment and Function – Pre-clinical

8

RPE cells rescued photoreceptors and significantly slowed lose of vision in

animal models of macular degeneration

control treated

Injected human RPE cells repair monolayer structure in eye

Photoreceptor layer

photoreceptor layer is only 0 to 1 cell thick

without treatment

Page 9: Rodman Conference, September 2012

• Established GMP process for differentiation and purification of RPE – Virtually unlimited supply from stem cell source – Characterized to optimize performance

• Ideal Cell Therapy Product

– Centralized Manufacturing – Small Doses – Easily Frozen and Shipped – Simple Handling by Doctor

GMP Manufacturing

9

Product Cold Chain is Easily Scaled for Global Sales

Page 10: Rodman Conference, September 2012

RPE Clinical Program – to date

10

World-leading eye surgeons and retinal clinics participate in clinical trials, DSMB and Scientific Advisory Board

ClinicalTrials.gov US: NCT01345006, NCT01344993 UK: NCTO1469832

• US Clinical Trial Sites • Jules Stein Eye (UCLA) • Wills Eye Institute • Bascom Palmer Eye Institute • Massachusetts Eye and Ear Infirmary

Stargardts: 4 Patients Treated (Cohort 2 begun) Dry AMD: 4 Patients Treated (Cohort 2 begun)

• European Clinical Trial Sites • Moorfields Eye Hospital • Edinburgh Royal Infirmary

Stargardts: 3 Patients Treated (Cohort 1 complete)

Page 11: Rodman Conference, September 2012

Surgical Overview

11

Procedure: • 25 Gauge Pars Plana Vitrectomy • Posterior Vitreous Separation

(PVD Induction) • Subretinal hESC-derived RPE

cells injection • Bleb Confirmation • Air Fluid Exchange

Page 12: Rodman Conference, September 2012

Preliminary Results

12

• Structural evidence confirmed cells had attached and persisted

• No signs of hyperproliferation, abnormal growth, or rejection

• Anatomical evidence of hESC-RPE survival and engraftment.

• Clinically increased pigmentation within the bed of the transplant

• Recorded functional visual improvements in both patients

Page 13: Rodman Conference, September 2012

Images of hESC-RPE transplantation site in SMD Patient

13

SD-OCT image collected at month 3 show survival and engraftment of RPE Migration of the transplanted cells to the desired anatomical location

3mo post-op

Page 14: Rodman Conference, September 2012

Media Coverage

14

First Hints That Stem Cells Can Help Patients Get Better

Headline: “Stem Cell Treatment for Eye Diseases Shows Promise”

Headline: “Some Promising Findings on Embryonic Stem Cells”

Page 15: Rodman Conference, September 2012

Intellectual Property – RPE Program • Dominant Patent Position for Treating Retinal Degeneration

• Broad Coverage for Manufacturing RPE Cells from hESC

• Coverage for RPE Cells derived from other pluripotent stem cells (including iPS cells) • Methods of manufacturing, use of RPE cells, and pharmaceutical formulations

• Vigilant Filing on Improvements • Extends patent life cycle, with significance to commercialization • Include composition-of-matter claims (cell preparations, pharmaceutical preparations, etc.)

15

Page 16: Rodman Conference, September 2012

Price Justification

16

Justification

Clinical Unmet Need

Clinical Efficacy

Patient Prevalence

Pharmaco-economic Data

Patient Advocacy Groups

More Important

Less Important

Both private and public payers are most interested in understanding

how new therapies will deliver enhanced clinical value.

RPE Therapy provides pricing

justification across all categories of consideration

Page 17: Rodman Conference, September 2012

RPE Program - Investment Thesis

17

There is an immense need for treatments against dry AMD and other atrophic forms of macular degeneration.

• Small Doses • Immunoprivileged – allogeneic source of cells • Noninvasive monitoring of retina

Market potential: More than 50 million potential patients in major markets.

• Even a one percent (1%) market penetration for RPE cell therapy in dry AMD represent a $5-10 Billion market opportunity.

• Orphan indications are also meaningful: Estimating a 10% market penetration with reoccurring treatments every 3-5 years, Stargardt’s disease can be a $100+ million/year product.

Page 18: Rodman Conference, September 2012

Interest from Industry

• Clear interest in ocular products by big pharma and global biotech

• Steady developing interest in regenerative medicine and stem cell-derived therapeutics Not a matter of if we will partner, but more

likely a question of when to partner

18

Page 19: Rodman Conference, September 2012

Partnering Asset Overview

• Intellectual Property – Aggressively pursuing core patents and follow-on inventions – Creating the “thicket behind the picket”

• Clinical Grade Cells – RPE cells – the right cell for the task – Master Stem Cell Bank – Preliminary safety data

• IND package • Our Experience! • Clinical Trial Sites

– Knowledge leaders

19

Page 20: Rodman Conference, September 2012

Tllllllllll Plllllll l

Olllll Plllllll

Page 21: Rodman Conference, September 2012

Retinal Pigment Epithelial Cells Macular Degeneration - dry AMD, Stargardt’s Disease, MMD 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

light

retina

RPE

layer

Ph

otor

ecep

tors

21

Page 22: Rodman Conference, September 2012

ACT MSC Pllllll

Page 23: Rodman Conference, September 2012

Mesenchymal Stem Cells in Therapy

23

Mesenchymal stem cells (MSCs) regulate immune responses, providing therapeutic potential for treating autoimmune or inflammatory diseases.

• MSCs can be used allogeneic: without matching between donors and recipients.

• Adult-derived MSCs have already been used therapeutically in clinical trials.

• Potential uses in a wide range of autoimmune conditions, such as multiple sclerosis, lupus, and Crohn's disease, among others.

An "off-the-shelf" cellular drug ready for treatment of a wide range of inflammatory and autoimmune diseases.

Page 24: Rodman Conference, September 2012

Adult Mesenchymal Stem Cells

24

Impacts on Cell Banking

• Limitation on the number of doses that can be generated from adult donors • A few hundred to a thousand doses per cell bank per donor. • Requires constantly creating and validating MSC banks from new donors

Impacts on Potency

• Passaging reduces immunomodulatory potency of MSC’s.

Replicative capacity is a big limitation for adult sources (bone marrow, fat, etc) of allogeneic MSC therapies.

Page 25: Rodman Conference, September 2012

CONTRAST: hESC/iPS – derived MSC

25

Proprietary scalable manufacturing of “young” MSCs

ACT Proprietary Process • hESC-derived MSCs can be expanded to large numbers of cells

• Have qualities similar to fetal MSC’s • Avoid replicative capacity problem of “old” adult MSC’s

Advantages for Manufacturing • Use Single Master Cell Bank

• Simplifies FDA/regulatory process • No need for finding donors

• Less labor-intensive

Page 26: Rodman Conference, September 2012

Preliminary Data

26

Animal Models testing hESC-derived MSC’s

• Substantially decrease and reverse disease conditions in autoimmune models.

• Far more potent than adult (BM) derived MSCs. • Have longer duration of action compared to adult (BM) derived MSCs.

Potential implications of increased potency and duration… • Broader utility of hESC- and iPS-derived MSCs in range of diseases. • Reduced number of cells per dose – improved safety profile. • Longer duration between injections.

CONFIDENTIAL

Page 27: Rodman Conference, September 2012

Potential therapeutic applications for MSCs

27

• >100 autoimmune diseases • Multiple Sclerosis • Osteoarthritis • Aplastic Anemia • Crohn’s Disease/IBS • Chronic Pain • Limb Ischemia • Heart Failure/MI • Stroke • Graft-versus-host Disease

• Spinal Cord Injury • Parkinson’s Disease • Liver Cirrhosis • Emphysema/Pulmonary Diseases • Wound healing

(ulcers/decubitus/burns) • HSC engraftment/irradiated cancer

patients • Eye diseases (uveitis, retinal

degeneration, glaucoma)

Page 28: Rodman Conference, September 2012

MSC – Investment Thesis

28

The New Autoimmune/Anti-Inflammatory Medication

Autoimmune: Markets for effective treatments for autoimmune diseases are vast.

• Forecast to exceed $80 billion by 2020 • CAGR > 10+ percent annually

Beyond Autoimmune: potent MSC therapies likely to have utility in inflammatory disorders and chronic pain.

Page 29: Rodman Conference, September 2012

Financial Update – Strong Balance Sheet

29

• Company ended 2012 Q2 with $10 million cash on hand • $9 million more available under existing equity line • Virtually debt-free • Received shareholder approval for reverse split • Filed application for NASDAQ uplisting and have received initial comments

Other 2012 Milestones (so far) • IRB approvals from Wills Eye Institute, Bascom Palmer Eye Hospital and Massachusetts Eye & Ear • Initiated Europe’s first human ESC-derived transplant at Moorfields Eye Hospital • Published first report of hESC-derived cells transplanted into humans in top medical journal, The Lancet. • Completed Dose Cohort 1 of patients in both U.S. trials; DSMB approval to move to next higher dose • Dosed first SMD and dry AMD patients in 100,000 cell cohorts – no AE’s observed.

Page 30: Rodman Conference, September 2012

ACT Management Team Highly Experienced and Tightly Integrated Management Team

Gary Rabin – Chairman & CEO Dr. Robert Lanza, M.D. – Chief Scientific Officer Edmund Mickunas – Vice President of Regulatory Affairs Kathy Singh - Controller Rita Parker – Director of Operations Dr. Irina Klimanskaya, Ph.D. – Director of Stem Cell Biology Dr. Shi-Jiang (John) Lu, Ph.D. – Senior Director of Research Dr. Roger Gay, Ph.D. - Senior Director of Manufacturing Dr. Matthew Vincent, Ph.D. – Director of Business Development Bill Douglass – Director of Corporate Communications & Social Media

30

Page 31: Rodman Conference, September 2012

Thank you For more information, visit www.advancedcell.com