Stakeholder Opinions: Spinal Cord Injury - Attractive niche market with multi-billion dollar potential

Introduction

Advances in understanding the pathophysiology of spinal cord injury (SCI) over the past 2 decades have given rise to several therapeutic strategies that are presently being examined in Phase II/I clinical trials. Furthermore, President Obama's March 2009 decision to overturn restrictions on human embryonic stem cell research represents a new era in the field of spinal cord injuries.

Scope

*Analysis of the patient potential of spinal cord injury across the seven major markets and rest of world.

*Review of key unmet clinical needs in the treatment of spinal cord injury and current pipeline treatments.

*Identification of key opportunities and threats facing developers of treatments for spinal cord injury.

*Insight from four internationally recognized key opinion leaders in the field of spinal cord injuries.

Highlights

Therapeutic interventions for SCI need not necessarily provide a complete restoration of anatomic connections in order to lead to functional improvements and enhance patients' quality of life. Controversy continues to surround the use of methylprednisolone as an acute treatment for SCI, indicating a need for approved neuroprotective treatments.

Alseres Pharmaceuticals's Cethrin (a recombinant fusion protein) has demonstrated a measurable effect on neurological recovery after SCI in a Phase I/IIa clinical trial. However, with its Phase II clinical program currently on hold, future development of Cethrin is uncertain.

President Obama's March 2009 decision to lift restrictions on US federal funding for research involving human embryonic stem cells has provided developers with a greater deal of freedom with regards to research into SCI.

Reasons to Purchase

*Quantify the incidence of spinal cord injury and identify key clinical unmet needs.

*Assess the opportunities and threats facing developers in the spinal cord injury market.

*Utilize pipeline product profiles to identify potential in-licensing opportunities.

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Overview 1
Catalyst 1
Summary 1
About Datamonitor Healthcare 2
About the Central Nervous System pharmaceutical analysis team 2
Executive Summary 3
Strategic scoping and focus 3
Datamonitor insight into the spinal cord injury market 3
Upcoming reports 5
Table of Contents 6
Chapter 1 Spinal Cord Injury: Patient Potential 7
Key findings 7
Definition of spinal cord injury 8
Classification of spinal cord injury 8
Complete versus incomplete injury 9
Tetraplegia and paraplegia 9
ASIA Impairment Scale 9
Etiology of spinal cord injury 10
Road traffic accidents are the leading cause of SCI in developed countries 11
Pathophysiology of spinal cord injury 12
Primary injury 12
Secondary injury 13
Immediate phase 13
Acute phase 13
Intermediate phase 13
Chronic phase 13
Diagnosis of spinal cord injury 14
Epidemiology of spinal cord injury 15
Seven major markets 15
New cases of spinal cord injury are expected to reach over 19,000 across the seven major markets in 2010 16
Rest of the world 18
Almost 4,000 new cases of spinal cord injury are projected each year in Russia between 2010 and 2020 20
Epidemiological trends in spinal cord injury 21
Gender: spinal cord injury predominantly affects men 21
Age: most spinal cord injuries occur in young adults although the mean age at injury is increasing 22
Mortality: life expectancies of SCI patients have increased due to advances in healthcare 24
Economic consequences of spinal cord injury 25
Chapter 2 Current Treatment Practices and Unmet Needs 27
Key findings 27
Current management of spinal cord injury 28
Surgery 28
Methylprednisolone is a pharmacological treatment option for acute spinal cord injury 29
Rehabilitation 30
Patient care path 31
The acute hospital setting is the key intervention point for neuroprotective treatments 32
For manufacturers of neurorestorative treatments, rehabilitation and community settings are key targets 32
Further opportunities exist in the management of complications of SCI 33
Unmet need in spinal cord injury 33
Neurorestorative treatments 34
Neuroprotective treatments 36
Symptomatic treatments 37
Greater efficacy of treatments for neuropathic pain 37
Treatments for autonomic dysreflexia 41
Improved treatments for spasticity 41
Chapter 3 Market Potential 43
Key findings 43
Datamonitor's estimate of market potential 44
Spinal cord injury: opportunities and threats 44
Opportunities 45
Eligibility for orphan drug/disease status 45
Increasing R&D investment 47
Treatments can command very high price points 48
Specialized area maximizes sales and marketing force effectiveness 49
Technology applications for other conditions 50
Lifting of restrictions on human embryonic stem cell research in the US 51
Prevalence of SCI is higher than previously believed 52
Threats 53
Safety concerns with stem cell therapies 53
Potential reversal of US stem cell regulations 56
Chapter 4 Pipeline Analysis 58
Key findings 58
Clinical trial design in spinal cord injury 59
ICCP guidelines for the conduct of clinical trials in spinal cord injury 59
Spontaneous recovery and statistical power 60
Outcome measures: the ASIA impairment scale forms the standard measure 62
Inclusion and exclusion criteria 63
Clinical trial design 63
Clinical pipeline overview 64
Novartis represents the only Big Pharma company in the sparse clinical pipeline for SCI 64
The pipeline is dominated by neurorestorative treatments for acute SCI 67
Cethrin (BA-210; Alseres Pharmaceuticals) 68
Drug profile: intraoperatively administered Rho GTPase protein inhibitor 68
Development overview 69
Cethrin's development program is currently suspended 69
Phase I/IIa trial results: Cethrin has a measurable effect on neurological recovery after SCI 70
Suspended Phase II trial 72
Product positioning 73
As the leading neuroregenerative pipeline candidate, Cethrin could be first to market 73
SWOT analysis 74
XP19986 (arbaclofen placarbil; Xenoport) 75
Drug profile: GABAb receptor agonist in Phase II development for spasticity due to SCI 75
Development overview 76
Phase II trial results: 20mg and 30mg doses show significant improvements in muscle tone 76
Product positioning 78
XP19986 will be competing with generic baclofen 78
Spasticity in SCI is expected to be the secondary indication for XP19986 79
SWOT analysis 79
ProCord (Proneuron Biotechnologies) 81
Drug profile: autologous blood-derived macrophage therapy for the treatment of acute SCI 81
Development overview 81
Phase II development of ProCord is currently on hold 81
Phase I study results demonstrate good tolerability in patients with acute SCI 83
Product positioning 85
ATI355 (reticulon 4 monoclonal antibody; Novartis AG) 85
Drug profile: first human monoclonal antibody to neutralize Nogo-A 85
Development overview 86
Phase I multicenter trial is due to be completed in Q4 2010 86
Product positioning 88
As a neuroregenerative treatment for acute SCI, ATI355 will be in competition with Cethrin and ProCord 88
Autologous Combination Stem Cell Therapy (TCA Cellular Therapy) 89
SCI represents the least advanced indication for Autologous Combination Stem Cell Therapy 89
Astrostem (RNL Bio) 89
Human clinical study of Astrostem approved by Korean FDA in April 2009 89
Neu2000 (Neurotech Pharmaceuticals) 90
Improved locomotor outcomes associated with Neu2000 in rat model of moderate SCI 90
Cordaneurin (Spinal Cord Therapeutics) 91
Cordaneurin prevents the formation of growth inhibiting scars 91
Preclinical pipeline overview 91
Spinal Cord Therapeutics and SanBio represent key developers in the preclinical SCI pipeline 93
Spinal Cord Therapeutics's two preclinical candidates are targeting chronically injured SCI patients 93
SanBio's two SCI preclinical candidates are allogeneic donor cell treatments 94
Initiation of world's first human clinical trial of embryonic stem cell-based therapy is expected in Q3 2010 95
Neureva is presently seeking a co-development partner for NVA0011 (gacyclidine) 96
Discontinued pipeline candidates for spinal cord injury 96
The future of treatment in spinal cord injury 97
Key opinion leaders view partially restorative therapies to be the most likely advance in the mid-term 97
Combination treatments offer the greatest potential according to key opinion leaders 98
Geographical availability of efficacious SCI treatments will be limited by expertise of physicians and cost 99
Bibliography 101
Books and journal papers 101
Websites 112
Datamonitor reports 119
Appendix 120
Contributing experts 120
Conferences attended 120
Report methodology 120
About Datamonitor 121
About Datamonitor Healthcare 121
About the Central Nervous System pharmaceutical analysis team 122
Disclaimer 124
List of Tables
Table 1: ASIA impairment scale 10
Table 2: Studies of the incidence of spinal cord injuries in the seven major markets 16
Table 3: Incidence of spinal cord injury in the seven major markets, 2010-2020 (actual numbers) 18
Table 4: Studies of the incidence of spinal cord injuries in the rest of the world 19
Table 5: Incidence of spinal cord injury in the rest of world markets, 2010-2020 (actual numbers) 21
Table 6: Average yearly healthcare and living expenses and estimated lifetime costs directly attributable to spinal cord injury 26
Table 7: Studies of the prevalence of neuropathic pain in the spinal cord injury population 38
Table 8: Results of published studies investigating the efficacy of oral pharmacological treatments in neuropathic spinal cord injury pain 40
Table 9: Estimation of the US spinal cord injury market potential 44
Table 10: Spinal cord injury market opportunities and threats 45
Table 11: Products in clinical development for spinal cord injury, 2010 67
Table 12: Cethrin - drug profile, 2010 69
Table 13: Key facts: Phase II trial of Cethrin in patients with acute spinal cord injury 73
Table 14: XP19986 - drug profile, 2010 76
Table 15: ProCord - drug profile, 2010 81
Table 16: Key facts: Phase II trial of ProCord in patients with complete spinal cord injury 82
Table 17: ATI355 - drug profile, 2010 86
Table 18: Key facts: Phase I trial of ATI355 in patients with acute spinal cord injury 87
Table 19: Products in preclinical development for spinal cord injury, 2010 92
Table 20: Discontinued R&D projects in spinal cord injury, 2002-2010 96
List of Figures
Figure 1: Key causes of traumatic spinal cord injury in the US, 2005-09 11
Figure 2: Spinal cord injury patient care path 31
Figure 3: Key unmet clinical needs in spinal cord injury, 2010 34
Figure 4: Comparison of incentives conferred by orphan product policies across the US, EU and Japan, 2010 47
Figure 5: Guidelines for the conduct of clinical trials for spinal cord injury as developed by the ICCP panel 60
Figure 6: Composition of clinical pipeline for spinal cord injury by stage of development 65
Figure 7: Cethrin: Phase I/IIa trial results 71
Figure 8: Cethrin: SWOT analysis 75
Figure 9: XP19986: Phase II trial results 77
Figure 10: XP19986: SWOT analysis 80
Figure 11: Autologous activated macrophage therapy: Phase I trial results 84

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