Market Report Source

Overview

At the forefront of novel technologies; momentum is steadily building for the development of stem cell products to address cardiovascular disease. The most active areas are those with high clinical unmet need heart failure, cardiomyopathy and ishemic heart disease.

Scope of this report:

• Analysis of the current unmet needs in cardiovascular disease markets
• Opinion leader commentary on key clinical trials and market landscape
• Review of the clinical and commercial status of stem cell therapy for cardiovascular disease
• Review of the clinical and commercial status of gene therapy for cardiovascular disease

Research and analysis highlights:

Datamonitor’s analysis demonstrates how the most innovative drugs offer the greatest growth potential for their respective markets. In the mature cardiovascular market, commercial success must be derived from innovative approaches with unique clinical benefits.

The array of innovative gene and cell products emerging from young companies exposes investment opportunities for venture capitalists, Big Pharma, as well as partnering opportunities for technology companies that can offer compatible catheters, or cell delivery devices.

There awaits an outstanding commercial opportunity for innovators who address clinical unmet need in this market, as a number of companies will see their key revenue generators melt away as they lose patent protection.

Key reasons to read this report:

• Understand the need for innovation in the cardiovascular market.
• Up to date insight into the commercial and clinical status of cardiovascular gene and cell therapies
• Understand the barriers to commercialization of novel therapies, with expert commentary

Table of Contents

CHAPTER 1 EXECUTIVE SUMMARY
Objective of the analysis
Datamonitor Key insights
Related reports
Acknowledgements

CHAPTER 2 BACKGROUND
Business Implications
Introduction
Scope
Definition of unmet needs
Clinical unmet needs in cardiovascular markets
Commercial Unmet Needs in cardiovascular markets
Commercial opportunities across cardiovascular indications
Response to the unmet need
Summary

CHAPTER 3 NOVEL DRUG TARGETS IN CV MEDICINE
Business Implications
Introduction
Bridging the “innovation gap”
Cardiovascular drug targets
New technologies allow expansion into novel targets
A lack of commercially viable drug targets
Biologics drive market growth
Monoclonal antibodies (MAbs)
Application of MAbs in cardiovascular disease

• ReoPro (abciximab) profile

MAbs in the cardiovascular pipeline

• BI-204 for atherosclerosis
• TB-402 for thrombosis

Therapeutic vaccines
The need for therapeutic vaccines for chronic cardiovascular disease

• Angiotensin vaccines for hypertension
• Protherics’s Angiotensin Therapeutic Vaccine
• Cytos Biotechnology’s CYT006-AngQ
• Outlook for angiotensin vaccines

CHAPTER 4 STEM CELLS FOR USE IN CV MEDICINE
Business Implications
Introduction
The biology of stem cells
Derivation
Embryonic stem cells (ESCs)
Adult stem cells (ASCs)
The use of embryonic versus adult stem cells
Proliferation
Differentiation and pluripotency
Allogeneic versus autologous stem cells
The development timeline for stem cell technology
The exploitation of stem cells
Stem cell therapy
Use in regenerative medicine and tissue engineering
Stem cell transplantation
Stem cells for use in drug discovery
Screening arrays of compounds
Predictive toxicology
Stem cell therapy in cardiovascular medicine
Current status
The need for cardiovascular regeneration

• Myocardial infarction and myocardial ischemia
• Heart failure

Stem cell suitability for cardiovascular regeneration

• Embryonic stem cells (ESCs)
• Adult stem cells (ASCs)

Clinical development of stem cells for cardiovascular disease
Current clinical trial focus
Clinical trial results
Summary and future focus
Future clinical trial focus
Commercial landscape
International markets
Key players and products
Company Profiles

• Aastrom Biosciences
• Advanced Cell Technology
• Angioblast Systems
• Athersys
• BioHeart
• Cardio3BioSciences
• Osiris Therapeutics

Development hurdles for stem cell therapy
Commercial risk associated with small companies
Research capacity
Regulation
Ethics and the perception of stem cells
Reimbursement
Intellectual property

CHAPTER 5 GENE THERAPY FOR USE IN CV MEDICINE
Business Implications
Introduction
Key definitions
Genomics
Gene therapy
The evolution of gene therapy
Discovery timeline
Approaches to gene therapy

• In vivo versus ex vivo gene therapy

Gene delivery strategies
The use of vectors

• Vector types

Delivery modalities

• Systemic delivery
• Non-systemic delivery

Disease targets for gene therapy
Research activity by indication
Cancer
Inherited diseases
Other targets
Gene therapy targets in cardiovascular disease
Therapeutic angiogenesis

• Coronary artery disease (CAD) and myocardial ischemia
• Peripheral arterial disease (PAD)

Heart failure

• The beta-adrenergic (beta-AR) receptor signaling pathway
• SERCA2a

Hypertension

• ACE gene linked to high blood pressure in men

Targets in dyslipidemia

• LDL-cholesterol and triglycerides
• HDL-cholesterol
• Other dyslipidemic targets

Gene therapies in the drug development pipeline
Current status
Current status of clinical trials of gene therapy
Gene therapy for coronary artery disease
Gene therapy for peripheral arterial disease
Gene therapy for heart failure

• The beta-adrenergic receptor (beta-AR) signaling pathway
• SERCA2a

Gene therapy for dyslipidemia
Gene therapy for hypertension
Future perspectives in gene therapy

• Summary of preclinical and clinical research
• Restenosis

Commercial landscape
International markets
Key players and products

• Cardium Therapeutics

Development hurdles for gene therapy
Research and informatics

APPENDIX
Bibliography
About Datamonitor

• About Datamonitor Healthcare
• About the Cardiovascular Disease analysis team
• Report methodology
• Disclaimer

List of Tables
Table 1: Summary of clinical unmet needs across the main cardiovascular indications, 2008
Table 2: Family classification of drug targets
Table 3: ReoPro: Overview
Table 4: Key clinical trials using stem cells in acute myocardial infarction, 2008
Table 5: Key clinical trials using stem cells in heart failure, 2008
Table 6: Key unknowns in cardiovascular stem cell therapy
Table 7: Stem cell products currently in development for cardiovascular indications, 2008
Table 8: Stem cell companies developing products for cardiovascular indications (Listed)
Table 9: Stem cell companies developing products for cardiovascular indications (Non-listed)
Table 10: Comparison of different gene vectors used for gene transfer
Table 11: Potential delivery methods for different vectors in cardiovascular disease
Table 12: Genetic targets in cardiovascular disease
Table 13: Effects of overexpressing SR Ca2+ pump in myocytes isolated from failing human hearts
Table 14: Key clinical trials in gene therapy for CAD
Table 15: Key clinical trials in gene therapy for PAD

List of Figures
Figure 1: Chapter 2 – Business Implications
Figure 2: The $ sales plateau in the cardiovascular market, 2006-2016
Figure 3: US patent expiries of the top 10 cardiovascular brands, 2006-2016
Figure 4: Summary of unmet needs in cardiovascular indications, 2008
Figure 5: Chapter 3 – Business Implications
Figure 6: Technological advances expand the conceptual ‘druggable’ space
Figure 7: Key characteristics influencing pharmaceutical commercial attractiveness
Figure 8: The mechanism through which the renin-angiotensin-aldosterone system induces systemic hypertension
Figure 9: Chapter 4 – Business Implications
Figure 10: Definition of terms in the stem cell differentiation process
Figure 11: US and UK milestones in the development of stem cell technology, 1950-2008
Figure 12: Summary of current focus of stem cell technology in cardiovascular disease, 2008
Figure 13: Expert comment: maximizing useful progenitor cells per sample volume is an important consideration
Figure 14: Development hurdles for embryonic stem cells
Figure 15: Expert comment: future clinical trial focus for stem cell therapy in cardiovascular disease
Figure 16: Cardiovascular stem cell therapies in clinical development, 2008
Figure 17: Proposed research, development and marketing protocol for stem cell therapies
Figure 18: Chapter 5 – Business Implications
Figure 19: Timeline of key developments in gene therapy
Figure 20: In vivo versus ex vivo gene transfer
Figure 21: Reserch activity in gene therapy, according to number of clinical trials, by indication
Figure 22: Summary of current focus of gene therapy research in cardiovascular disease, 2008
Figure 23: Reserch activity in gene therapy, according to number of clinical trials, by location
Figure 24: Cardiovascular stem cell therapies in clinical development, 2008