Pipeline Insight: Orphan Diseases in CNS - Part I: Huntington's Disease

Pipeline Insight: Orphan Diseases in CNS - Part I: Huntington's Disease

Introduction

The current Huntington’s disease market is comprised of off-label prescriptions of psychotropic drugs. In the near term, two pipeline drugs are set to enter the market, demonstrating the viability of this disease as a primary marketing indication. In the longer term, with a strong genetic link and reliable diagnostic test, prophylactic treatment with a disease modifying drug could prove lucrative.

Scope

ABOUT DATAMONITOR HEALTHCARE 2
About the CNS pharmaceutical analysis team 2
CHAPTER 1 EXECUTIVE SUMMARY 3
Scope of the analysis 3
Contributing experts 4
Interviewed Key Opinion Leaders 4
Datamonitor insight into the Huntington's disease market 5
Key metrics 7
Datamonitor pipeline assessment summary 8
Related reports 8
Upcoming reports 9
CHAPTER 2 MARKET OVERVIEW 11
Definition of Huntington's disease 12
Current market overview 12
Current therapies are limited to managing the symptoms associated with HD and co-morbidities 12
No treatment proven to alter the course of the disease 13
Current treatment classes 13
Current market value and trends 14
Antipsychotics and monoamine-depleting agents 15
Antidepressants and anxiolytics 18
Anti-Parkinsonism drugs 19
Etiology of Huntington's disease 20
Risk of inheriting HD 20
Prognosis for HD patients 21
Epidemiology of Huntington's disease 22
Base scenario 22
High prevalence rate patient population scenario 24
Very low prevalence in Japan 26
Increasing future trend in patient population 26
Unmet need in Huntington's disease 27
Slowing the progression of the disease 28
Reduced movement disorders 28
Reduced depression, anxiety and psychosis 28
CHAPTER 3 FUTURE MARKET POTENTIAL 30
Future market scenarios for symptomatic patients 31
Base prevalence future market value forecast 31
High prevalence future market value 32
Price of future HD drugs 32
Current cost of CNS drugs comparable to a new disease modifying HD therapy 32
Theoretical disease modifying drug 35
Pre-symptomatic ``at risk`` population - the lucrative untapped market 37
Scenarios matrix summary 40
CHAPTER 4 PIPELINE OVERVIEW AND DYNAMICS 41
Pipeline drugs market potential 42
Pipeline overview 43
Definition of current comparator therapy 44
Classification of pipeline products 44
Recently discontinued or failed projects 44
CHAPTER 5 LATE-STAGE DRUG ANALYSIS & FORECASTS 46
Xenazine (tetrabenazine) 47
Drug overview 47
Forecasts to 2016 48
Datamonitor drug assessment summary 50
Marketing factors 51
Market penetration 51
Xenazine pricing assumptions 52
Patient potential 52
Satisfaction of unmet needs 53
Clinical trial data 53
Tetrabenazine as antichorea therapy in Huntington's disease, Phase III study 53
Phase III extension study 54
Older studies investigating tetrabenazine in HD 55
ACR16 56
Drug overview 56
Forecasts to 2016 58
Datamonitor drug assessment summary 60
Marketing factors 61
Market penetration 61
NeuroSearch should concentrate marketing efforts on the motor dysfunction benefits of ACR16 62
ACR16 pricing assumptions 63
Patient potential 64
The patient potential of treating depression 64
Positive effects on psychosis and agitation may also be seen in larger-scale trials 64
Even if chorea is not adequately treated with ACR16, the patient potential would be high 65
Significant alleviation of chorea may still be shown in larger-scale clinical trials 65
Satisfaction of unmet needs 66
Clinical trial data 66
Planned Phase III trials 66
Phase II study: ACR16C007 67
Pre-clinical data - What is a dopamine stabilizer? 68
HD-02 - Ultra-pure creatine 68
Drug overview 68
Forecasts to 2016 69
Marketing factors 70
Patient potential 70
Satisfaction of unmet needs 71
Clinical trial data 71
Planned Phase III trial 71
Phase I/II study 71
Independent study on creatine 72
Dimebon 73
Drug overview 73
Forecasts to 2016 74
Clinical trial data 74
Ongoing Phase II trial 74
Completed Phase I study 75
Further development and submission 75
BIBLIOGRAPHY 76
Journals 76
Websites 78
APPENDIX A 82
Methodology 82
Datamonitor forecast methodology for the HD market 82
Epidemiology forecasts 82
Product forecasts 82
Definition of a standard unit 83
Contributing experts 85
Interviewed NeuroSearch company executives 85
APPENDIX B 86
About Datamonitor 86
About Datamonitor Healthcare 86
Datamonitor Healthcare's therapy area capabilities 87
About the Disease analysis team 88
Disclaimer 89
List of Tables
Table 1: HD base scenario prevalence and patient population in the 6MM, 2007 23
Table 2: HD high prevalence scenario patient population in the 6MM, 2007 25
Table 3: HD prevalence and patient population in Japan, 2007 26
Table 4: Price of comparable CNS drugs, 2007 34
Table 5: Matrix of HD market value dependent on patient population and price in the 6MM, 2007 40
Table 6: R&D clinical-stage pipeline in Huntington's disease, 2007 43
Table 7: Xenazine key assumptions and events, 2007 49
Table 8: Key clinical and commercial attractiveness factors for Xenazine 51
Table 9: ACR16 key assumptions and events, 2007 59
Table 10: Key clinical/commercial attractiveness factors for ACR16 61
Table 11: Datamonitor drug assessment parameters 84
List of Figures
Figure 1: Forecast of total 6MM market size and key pipeline drugs, 2004-2016, 7
Figure 2: Drug assessment summary for HD treatments, 2007 8
Figure 3: Total sales revenue of the six major HD markets by region, 2003-07 14
Figure 4: Total sales revenue of the six major HD markets by drug class, 2003-07 15
Figure 5: Proportion of HD-specific sales revenue attributed to atypical versus typical antipsychotics in the US, 2004 and 2006 17
Figure 6: Proportion of HD-specific sales revenue attributed to atypical versus typical antipsychotics in the 5EU, 2004 and 2006 18
Figure 7: Genetic risk of developing HD 21
Figure 8: Unmet needs in HD treatment, 2007 27
Figure 9: Base prevalence future market value for HD ($m), historical sales 2004-06, forecast sales, 2007-2016, 6MM 31
Figure 10: High HD prevalence scenario future market value for HD ($m), historical sales 2004-06, forecast sales, 2007-2016, 6MM 32
Figure 11: Potential total HD market value ($m), 2004-2016 36
Figure 12: Drug assessment summary for Theoretical Drug X for the treatment of HD, 2007 37
Figure 13: Summary of HD market and pipeline drugs potential ($m), historical sales 2004-06, forecast sales, 2007-2016 42
Figure 14: Xenazine HD specific sales revenue forecast ($m), historical sales 2004-06, forecast sales 2007-2016 48
Figure 15: Drug assessment summary for Xenazine in HD, 2007 50
Figure 16: ACR16 HD-specific sales revenue forecast ($m), historical sales 2004-06, forecast sales, 2007-2016 58
Figure 17: Drug assessment summary for ACR16 in HD, 2007 60