RNA therapy: the next big thing after monoclonal antibodies?

Published by: Datamonitor

Published: Oct. 27, 2008 - 80 Pages

Table of Contents

CHAPTER 1 EXECUTIVE SUMMARY

Key findings

Why RNA therapy?

RNA therapy is not yet validated

Longer-term sales driver

High potential brings investment

Can RNA therapy deliver on its promise?

CHAPTER 2 BACKGROUND TO RNA THERAPY

Key findings

Why the interest in RNA therapy?

RNA therapies hit the central dogma of biology

Gaining access to non-druggable targets

mRNA splicing yields further potential targets

Gene therapy also offers significant potential, but has fallen out of favour

History/ discovery

Antisense silencing

Antisense technology has been available for over 30 years

Phenomenon of sense RNA also causing inhibition

Emergence of RNA interference

RNA interference discovered in 1998

Accelerated entry of RNAi-based products into the clinic

CHAPTER 3 RNA THERAPY & PIPELINE OVERVIEW

Key findings

RNA therapy triggers

Only one RNA therapy has reached the market to date

Mechanisms of gene silencing

RISC-independent silencing

RISC-mediated (RNAi) pathway offers greater potency

Analysis of the RNA therapy pipeline by trigger

Late-stage pipeline largely consists of antisense oligonucleotides

Antisense RNA therapies are at a more advanced stage of development

Focus has shifted towards RISC-mediated silencing (RNAi) and siRNA

Next-generation structures maintain interest in antisense

Focus on antisense and siRNA, but variants are emerging

Pipeline is dominated by siRNA and antisense

Further silencing technologies are emerging

miRNA offers a wealth of additional targets

CHAPTER 4 DETAILED PRODUCT PIPELINE

Key findings

Pipeline by product

Late-stage pipeline

Vitravene (formivirsen): only marketed RNA therapy

Genasense (oblimersen): struggle to demonstrate efficacy

Mipomersen (ISIS 301012): setbacks, but strong profile

Alicaforsen: Phase III failure for Crohn's disease

Bevasiranib: challenging Lucentis in the maintenance of AMD

Early-stage clinical pipeline

Pre-clinical pipeline

Pipeline by company

Discontinued products

Forecast RNA therapy sales

Comparison to the uptake of monoclonal antibodies

CHAPTER 5 DELIVERY AND THERAPY AREA ANALYSIS.

Key findings

Therapy area analysis

Pipeline by therapy area

Novel technology requires high levels of unmet need

Therapeutic focus driven by delivery technology

Therapy area by trigger type

Delivery remains key to success

Administration

CHAPTER 6 APPENDIX

References

Journals

Websites

Datamonitor reports

Abbreviations

List of Tables

Table 1: RNA therapy pipeline, marketed to Phase III

Table 2: RNA therapy pipeline, Phase II and Phase I

Table 3: RNA therapy pipeline, pre-clinical

Table 4: Discontinued RNA therapy products

Table 5: Segmented RNA therapy sales to 2020, $m

List of Figures

Figure 1: RNA therapy offers access to novel, disease-relevant targets

Figure 2: RNA therapy pipeline by development stage

Figure 3: Illustrative RNA therapy market to 2020, $m

Figure 4: Market capitalization of leading RNA therapy companies

Figure 5: Considerations for successful RNA therapy development

Figure 6: The central dogma of biology and potential role of RNA therapy in relation to traditional 'drugging' approaches

Figure 7: Technological advances expand the 'druggable' space

Figure 8: Key milestones in RNA therapy

Figure 9: RNA therapy pipeline by development stage

Figure 10: RNA therapy pipeline by involvement of the RNA-induced silencing complex, RISC

Figure 11: Classification of RISC-independent silencing triggers

Figure 12: Cellular processes involved in gene silencing

Figure 13: Classification of RISC-mediated (RNAi) gene silencing triggers

Figure 14: RNA therapy pipeline by development stage, split by silencing trigger technology

Figure 15: RNA therapy pipeline by silencing trigger technology , split by development stage

Figure 16: RNA therapy pipeline by development stage and company

Figure 17: RNA therapy pipeline by company, split by development stage (largest weighted companies only)

Figure 18: RNA therapy pipeline by company, split by trigger type (largest weighted companies only)

Figure 19: Estimated RNA therapy market to 2020, $m

Figure 20: Comparison of estimated RNA therapy sales versus initial growth of the monoclonal antibody market, $m

Figure 21: RNA therapy pipeline by development stage and therapy area

Figure 22: RNA therapy pipeline by therapy area, split by trigger type

Figure 23: Challenges associated with RNA therapy delivery

Figure 24: Administration routes used for RNA therapy

Abstract

Introduction

RNA therapies have been in development for a number of years, but are yet to achieve any notable commercial success. Improvements have been made following initial attempts and the technology may finally be coming to fruition.

Scope

• Strategic review of the different RNA therapy technology offerings
• Assessment of the commercial potential of key products
• Analysis of the RNA therapy pipeline according to key metrics
• Guidance on the factors that dictate RNA therapy design and the markets best suited to offer returns

Highlights

With the loss of patent protection on maturing blockbusters, innovative new products are required if the pharmaceutical industry is to maintain sales. RNA therapy potentially provides a rich source of such products, allowing targets that are not 'druggable' by other technologies to be exploited.

Despite a number of attempts, only one RNA therapy has reached the market. However, new mechanisms such as RNA interference have given rise to a generation of pipeline candidates characterized by improved safety and efficacy.

While sales on the scale of those enjoyed by monoclonal antibodies seem a way off, evidence suggests RNA therapy is now on course to deliver commercial returns.

Reasons to Purchase

• Assess the 134 RNA therapies in preclinical development and beyond
• Understand how RNA therapies fit alongside small molecule drugs, therapeutic proteins and monoclonal antibodies
• Understand reasons for the lack of approvals to date and how to maximize chances of commercial success

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