Delivery Mechanisms for Large Molecule Drugs: Successes and failures of leading technologies and key drivers for market success

Published by: Business Insights

Published: Jan. 1, 2009 - 138 Pages

Table of Contents

Delivery Mechanisms for Large Molecule Drugs

Executive summary

Introduction

Drivers for new platform developments

Resistors of change

Key emerging technologies

Systemic targeting technologies

Ease of use systems

Conclusions

Chapter 1 Introduction

Summary

Introduction

The emergence of large molecule therapeutics

Definitions

Technology platform definitions

Product coverage

Market coverage

Leading technologies coverage

The measures for market success

Chapter 2 Drivers of new platform developments

Summary

Introduction

The growth of the large molecule market

Therapy area growth drivers

Clinical development spend

Cost-effective manufacturing

Existing failure rates

Unmet clinical needs

Boosting patient compliance

Overcoming stability, bioavailability and toxic effects

Improving efficacy

Chapter 3 Risk, costs and technology maturity

Summary

Introduction

Risk of failure with new technologies

Unknown drug candidate pharmacokinetics

Solubility and instability with oral candidates

Bioavailability

Toxicity and unknown long-term effects

The shifting regulatory framework

Case study: Insulin delivery and investor confidence

The impact of cost and revenue on the decision to innovate

Immaturity concerns

Maturity of the delivery technologies

Chapter 4 Key emerging technologies

Summary

The forecast market impact

Nanotechnology to enhance solubility profiles

The evolving nanotechnology industry

The development pipeline

Leading clinical applications

Parenteral delivery systems

Dermal platform systems

Nanostructured materials; oral and depot system use

Novel oral drug delivery systems

Investigative nanoshells, nanofilms and active control

Advances in microelectronics

Existing electronic applications

The development pipeline for microelectronics

Microchip technologies

Inkjet technology for drug delivery

Chapter 5 Systemic targeting techniques

Summary

Introduction

Systemic passive targeting techniques

Stealth technologies: Immune system evasion

PEGylation technologies

PEGylation in clinical pipelines

Preclinical PEGylation investigation

The limitations of PEG

Next generation PEGylation

Systemic active targeting techniques

Antibody techniques

Antibody fragments

Binding specificity

Novel combination technologies to improve targeting

Cost-effective manufacture

The development pipeline

The emergence of IgG4 antibody therapies

Small modular Immunopharmaceuticals as antibody alternatives

Pipeline novel conjugate technologies

Antibody fragments in targeted carrier systems

Investigational protein carrier Prodrug complexes

Clotting factor conjugate targeting

Molecular trojan horse techniques

Chapter 6 Ease of use systems

Summary

Introduction

Pulmonary delivery technologies

Particle engineering technologies for pulmonary delivery

Vaporization techniques and delivery control

Applications of electronics

Needle-free transdermal delivery

Leading technology platforms

Needle-free pressure-based systems

Microinjection platforms for intra-epidermal delivery

EMEA filing for first microinjection system

technology platform

Electrotransport systems

Electroporation in transdermal delivery

TransPharma Medical ltd’s RF-Microchannel technology

Novel approaches to active intra-epidermal delivery

Laser drug delivery systems

Thermal energy platform

Chapter 7 Conclusions

Summary

Introduction

Pharma vs biotech large molecule R&D investment

Leading technologies

Growth in particle engineering technologies

The impact of new routes of administration

Large molecule drug delivery market growth and maturity

Current and future market impact

Therapy area impact

Timeline of impact

Summary of technology success and impact

Appendix

Index

Methodology

Methodology

MedTRACK platform identification

Glossary

List of Figures

Figure 1.1: The role of drug delivery in the product R&D pipeline

Figure 1.2: Biopharmaceutical company dependence on large molecule drugs*

Figure 1.3: Defining the pathway from proprietary technology to clinical use

Figure 2.4: The global pipeline for chemical and biologic drugs, October 2008

Figure 2.5: Number of pipeline biologic drug candidates and products, by therapy area, October 2008

Figure 2.6: Pharma R&D spend 2004-2009e

Figure 2.7: Biotech R&D spend ($bn), 2004-2009e

Figure 2.8: Pharmacokinetic effects; resistors of market growth and opportunity for new technologies

Figure 3.9: Key innovative technologies, clinical drug failures and discontinued products, November 2008

Figure 3.10: Development pipeline for insulin devices, human insulins and analogues, October 2008

Figure 3.11: Discontinued insulin devices, human insulins and analogues, platforms for delivery, per year 2001-2008

Figure 3.12: Key particle engineering technologies; industry size and maturity

Figure 3.13: Key route of administration technologies; industry size and maturity

Figure 4.14: Investment deals and clinical applications in nanotechnology drug delivery platforms, 2002-Q2 2008

Figure 4.15: Product pipeline; large molecule nanotechnology innovations

Figure 4.16: Maturity of electronic active delivery platforms in transmembrane and pulmonary delivery systems

Figure 5.17: The market advantage of targeted drugs

Figure 5.18: Passive targeting strategies for large molecule delivery

Figure 5.19: The benefits of PEGylation to improve pharmacological profiles

Figure 5.20: Active targeting strategies for large molecule delivery

Figure 5.21: The global MAb product pipeline by phase, Q4 2008

Figure 5.22: Antibody fragmentation platforms - Competitive advantage

Figure 5.23: Antibody fragments: separating targeting domains

Figure 6.24: Transdermal and transmembrane active platform technologies, November 2008

Figure 6.25: Investment in and maturity of active transdermal delivery

Figure 7.26: Big biotech v big pharma large molecule patent applications, 2003-2007, global

Figure 7.27: Particle engineering technologies in drug R&D pipelines, by phase, October 2008

Figure 7.28: Industry growth and investment, leading innovative drug delivery platforms

Figure 7.29: Growth in technology deals; 1998-2007

Figure 7.30: Impact of new technology platforms developments on therapy area pipelines

Figure 7.31: Therapy area focus of innovative technology product candidates, October 2008

Figure 7.32: New medical device technologies, anticipated market impact

Figure 7.33: Emerging particle engineering technologies, anticipated market impact

Figure 7.34: The impact of new delivery technologies; timeline for success

Figure 7.35: Measures of technology success

List of Tables

Table 1.1: Nektar’s leading innovative technology pipeline

Table 1.2: Needle free delivery; Key routes of administration

Table 1.3: Technology market coverage

Table 2.4: The global pipeline for chemical and biologic drugs, October 2008

Table 3.5: Key innovative technologies, clinical drug failures and discontinued products, November 2008

Table 3.6: Key route of administration technologies; industry size and maturity

Table 4.7: Nanotechnology drug delivery platforms, large molecule vs small molecule applications, November 2008

Table 4.8: Nanoparticles as drug delivery carriers

Table 4.9: Leading clinical parenteral drug delivery

Table 5.10: Clinical PEGylation stealth targeting technologies

Table 5.11: Antibody fragment products, clinical applications

Table 5.12: Armagen’s proprietary CNS product pipeline: Trojan horse conjugate delivery

Table 6.13: Small molecule success of membrane transport technologies, November 2008

Table 6.14: Clinical use electronic pulmonary delivery technologies

Table 6.15: Transdermal and transmembrane active platform technologies, November 2008

Table 6.16: Novel electroporation platforms; transdermal alternatives

Table 7.17: Innovative technology products in R&D pipelines, October 2008

Table 7.18: Industry maturity and investment, leading innovative drug delivery platforms

Table 7.19: Growth in technology deals, 1998-2007

Table 7.20: Therapy area focus of innovative technology product candidates, October 2008

Abstract

• Nanotechnology will have the greatest impact on the drug delivery market. However, the immaturity of the technology is likely to delay marketed presence over the next 7-10 years.
• Antibody fragmentation and PEGylation technologies are the leading targeted large molecule particle engineering formulas with marketed drug product presence. However, antibody fragments have suffered 33 candidate failures compared with 12 PEGylated products.
• Active transdermal technologies have generated the greatest number of technologies and devices amongst large molecule delivery innovations, with 16 clinical and 8 preclinical drug/device combination products and 49 stand-alone devices.
• Electronic delivery is set to have the greatest impact upon the device industry. Electronic device control is more advanced within the field of pulmonary delivery than transdermal delivery, with the average pulmonary-based product in early clinical phase I compared to late-stage preclinical investigation for transdermal. There are, however, 25 transmembrane electroporation technologies currently being developed,
in comparison to 12 for electronic active pulmonary delivery.

• Understand the drivers of new delivery platform developments with this report’s analysis of therapy area growth drivers, clinical development spend and unmet clinical need.
• Identify the risks and opportunities associated with emerging delivery technologies by measuring the risk potential and maturity of innovative platforms.
• Evaluate the latest developments in systemic targeting technologies by using this report’s analysis of innovations and pipeline progress for the latest active and passive targeting techniques.
• Discover which technologies have the greatest potential within large molecule product markets in the future with this report’s comparative analysis of growth metrics for leading platforms and an evaluation of their established clinical drug application.
• Assess recent innovations in pulmonary delivery technologies and needle-free
transdermal delivery with this report’s analysis of clinical and preclinical developments and commercial potential.

• High failure rates for new technologies. The high failure rates of drugs to which pioneering delivery techniques have been applied have made investors cautious.
• Unknown clinical safety and efficacy profiles have made it harder to determine appropriate parameters for success in clinical application.
• Immaturity of technologies. Many of the technology platforms profiled in this report are in the early stages of application to clinical drug candidates. Those that have achieved success in marketed drug candidates already have ‘next generation’ alternatives in technology pipelines.
• Unknown clinical pharmacokinetics. Many of these platforms remain in such an immature stage that they have yet to be applied to drug candidates. In vivo
experimental use in drug candidates can never accurately predict success once a technology has reached maturity. Even for those technologies with established use in R&D pipelines, long-term clinical efficacy remains unknown.
• Regulation of the new technologies. While the clinical performance of new particle engineered drug molecules or active delivery devices remain unknown, regulatory bodies only have existing data-measure demands on which to benchmark their expectations. This framework will be shifted in line with emerging clinical performance datasets.

• Which delivery technologies will have the greatest impact on the large molecule market in the short, mid, and long-term?
• How mature are the different delivery technologies and what is their pipeline presence in terms of application to R&D drugs?
• Will particle engineering technologies drive injectable formulas to dominate the market?
• Which therapy areas will benefit most from growth in the different technologies?
• How can the risk-profiles associated with clinical use be most effectively minimized?
• What are the leading novel platforms?
• Who are the targets for out-licensing and co-development of platforms for clinical use?
• What are the leading technology platforms within different classifications and how have they achieved their growth?

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