2013 Aegis Capital Healthcare Conference, Sept 26th 2013

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1 LEADING REGENERATIVE MEDICINE

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Transcript of 2013 Aegis Capital Healthcare Conference, Sept 26th 2013

Page 1: 2013 Aegis Capital Healthcare Conference, Sept 26th 2013

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LEADING REGENERATIVE

MEDICINE

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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

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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

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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

Oph

thal

mol

ogy

Prog

ram

s

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The Retina the light-sensitive tissue lining the inner surface of the eye

Retina

Structure of the Retina

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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

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Failure of RPE cells results in many degenerative diseases Stargardt’s disease Myopic Macular Dystrophy Age-related macular degeneration (AMD)

Life Support for Photoreceptors

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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%

50-59 40-49 60-69 70-79 80+ Age

Intermediate AMD Late AMD

% P

reva

lenc

e (U

.S.)

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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

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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

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Surgical Overview

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Procedure: • 25 Gauge Pars Plana Vitrectomy • Posterior Vitreous Separation • Subretinal hESC-derived RPE cells

injection • Bleb Confirmation

• Day Surgery/Sedation only

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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

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• 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

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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

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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

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Presenter
Presentation Notes
Y
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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 Treatment Phase II

Patient Treatment MMD Trial

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Sep 2013 July 2014

Phase I

Phase I

Presenter
Presentation Notes
Y
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Expanding Clinical Programs

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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

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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

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Therapeutic Pipeline

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Retinal Neural Progenitor cells & Isolated Protective Factors

Photoreceptor Loss, Modulation of Müller Cells Protection of Retinal Ganglion cells (Glaucoma)

Corneal Endothelium

Corneal Disease

Hemangioblast cells Ischemic retinopathy – diabetic retinopathy, vascular occlusions

Mesenchymal Stromal Cells

Ocular - Glaucoma, Uveitis, Retinitis Pigmentosa Autoimmune Diseases Inflammatory Diseases or disorders

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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”

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ACT Corporate Overview

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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

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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

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