Pipeline Insight: Orphan Diseases in CNS - Amyotrophic Lateral Sclerosis
Scope
Report Highlights
Reasons to Purchase
Table of Contents
- ABOUT DATAMONITOR HEALTHCARE - page 2
- About the CNS pharmaceutical analysis team - page 2
- CHAPTER 1 EXECUTIVE SUMMARY - page 3
- Scope of the analysis - page 3
- Contributing experts - page 4
- Datamonitor insight into the amyotrophic lateral sclerosis market - page 4
- Key metrics - page 6
- Scenario forecasts for ALS pipeline drugs and total market value - page 6
- Base scenario - page 7
- rhIGF-I /IPLEX successful clinical development scenario - page 8
- Scenario forecasts for ALS pipeline drugs and total market value - page 6
- Datamonitor pipeline assessment summary - page 9
- Related reports - page 10
- CHAPTER 2 MARKET OVERVIEW - page 12
- Definition of amyotrophic lateral sclerosis - page 13
- Segmentation of amyotrophic lateral sclerosis - page 13
- Etiology - page 14
- Etiology of ALS is largely unknown - page 14
- Prognosis of amyotrophic lateral sclerosis sufferers - page 15
- Epidemiology - page 16
- Diagnosis and treatment rates - page 17
- Diagnosis of ALS-ruling out other diseases first - page 17
- Treatment rates of ALS-the uptake of Rilutek - page 18
- Market size - page 19
- Sizing the ALS market - page 19
- Rilutek (riluzole) - page 21
- Drug overview - page 21
- Clinical trial information - page 22
- Pivotal Rilutek efficacy trial - page 22
- Phase III survival trial - page 23
- Efficacy has broadly been accepted - page 24
- Rilutek sales - page 24
- Rilutek uptake - page 25
- Unmet need in amyotrophic lateral sclerosis - page 27
- Clinical trial design - page 30
- CHAPTER 3 FUTURE MARKET POTENTIAL - page 31
- Scenario forecasts for ALS total market value - page 32
- Base scenario - page 33
- rhIGF-I /IPLEX successful clinical development scenario - page 34
- Forecast Rilutek sales - page 34
- Little switching between Rilutek and new therapies - page 35
- Rilutek patent protection - page 36
- US-Patent US-05527814 should protect Rilutek until 2013 - page 36
- 5EU-new use patent should protect Sanofi-aventis until late 2012 - page 37
- Japan-new use patent should protect Sanofi-aventis until late 2012 - page 37
- Scenario forecasts for ALS total market value - page 32
- CHAPTER 4 PIPELINE OVERVIEW AND DYNAMICS - page 38
- Pipeline overview - page 39
- Classification of pipeline products - page 40
- Advantages of developing an ALS treatment - page 42
- NINDS funding of clinical trials - page 42
- For mid-to-large cap companies, ALS offers little risk - page 43
- Indication expansion post-approval for ALS offers future potential - page 43
- Orphan drug designation has additional benefits - page 43
- Current comparator therapy-Rilutek - page 43
- CHAPTER 5 LATE-STAGE DRUG ANALYSIS & FORECASTS - page 44
- Pipeline comparative forecasts - page 45
- Two scenarios have been explored - page 45
- rhIGF-I-recombinant human insulin-like growth factor I - page 46
- Drug overview - page 46
- Forecasts to 2016 - page 47
- Base scenario - page 47
- rhIGF-I /IPLEX successful clinical development scenario - page 48
- Datamonitor drug assessment summary-IGF-1/IPLEX successful clinical development scenario - page 50
- Marketing factors-rhIGF-I /IPLEX successful clinical development scenario - page 51
- Marketing of rhIGF-I likely if the NINDS-sponsored trial is successful - page 51
- rhIGF-I will quickly be replaced with the combination product IPLEX - page 51
- Cost of IGF-1 - page 52
- Patient potential - page 53
- Satisfaction of unmet needs - page 54
- Better/alternative drugs to slow the progression of the disease - page 54
- Clinical trial data - page 54
- Cochrane Database Systematic Review of rhlGF-1 for ALS - page 54
- First clinical trial investigating the use of rhIGF-I in ALS - page 55
- Smaller negative trial investigating rhIGF-I and ALS - page 56
- Ongoing clinical trial program - page 56
- IPLEX (mecasermin rinfabate, rhIGF-I/IGFBP-3) - page 57
- Drug overview - page 57
- Forecasts to 2016 - page 58
- Base scenario - page 58
- IGF-1/IPLEX successful clinical development scenario - page 59
- Datamonitor drug assessment summary-IGF-1/IPLEX successful clinical development scenario - page 61
- IPLEX market penetration-two scenarios - page 61
- Marketing factors - page 62
- Withdrawal from US short-stature market - page 62
- Development for ALS likely to receive a higher priority - page 62
- IPLEX cost and dose - page 63
- IPLEX market penetration-two scenarios - page 64
- Patient potential - page 64
- Satisfaction of unmet needs - page 64
- Clinical trial data - page 65
- Radicut (edaravone, MCI-186) - page 65
- Drug overview-Phase III - page 65
- Lack of clinical data precludes assessment of commercial potential - page 66
- Clinical trial data - page 66
- Ongoing clinical trials - page 67
- Drug overview-Phase III - page 65
- E0302 (mecobalamin) - page 67
- Drug overview-Phase II/III - page 67
- Forecasts to 2016-applicable in both forecast scenarios - page 68
- Datamonitor drug assessment summary-IGF-1/IPLEX successful clinical development scenario - page 69
- Marketing factors - page 69
- Cost of E0302 in Japan - page 69
- Uptake of E0302 in Japan - page 70
- E0302/mecobalamin in the ex-Japan markets - page 70
- Patient potential - page 70
- Satisfaction of unmet needs - page 70
- Clinical trial data - page 70
- Ongoing clinical trials - page 72
- Creatine-based preparations - page 72
- ALS-02-creatine monotherapy - page 72
- Drug overview - page 72
- Marketing factors - page 73
- Creatine supplements are widely available over-the-counter - page 73
- Possible advantage of physician monitoring and reimbursement - page 74
- Potential cost and maximum revenue for ALS-02. - page 74
- Patient potential - page 75
- Satisfaction of unmet needs - page 75
- Clinical trial data - page 76
- Maximum voluntary isometric contraction - page 76
- Double-blind trial of survival and disease progression - page 76
- Small trial in advanced ALS patients monitoring respiratory function - page 77
- Clinical trial to examine the muscular effects of creatine in ALS patients - page 77
- Pilot study investigating creatine's effect on muscle strength in ALS patients - page 78
- Ongoing clinical trials - page 79
- ALS-08-creatine in combination with minocycline or celecoxib - page 79
- Minocycline combination shown to be inferior to celecoxib combination - page 79
- Minocycline monotherapy proved to be detrimental to patients - page 79
- Celecoxib combination-non-futile compared to historical controls - page 80
- Celecoxib monotherapy trial-no clear efficacy as a monotherapy - page 80
- ALS-02-creatine monotherapy - page 72
- Arimoclomol - page 81
- Drug overview-Phase II - page 81
- Clinical trial data - page 82
- Multi-center Phase IIa clinical trial monitoring safety and tolerability of arimoclomol - page 82
- Open-label extension clinical trial of arimoclomol in ALS - page 82
- Ongoing clinical trial program - page 83
- Other ALS pipeline drugs - page 84
- FP-0011 - page 84
- Drug overview-Phase II - page 84
- Clinical trial data - page 84
- Cereact - page 85
- Drug overview - page 85
- Clinical trial data - page 85
- Talampanel - page 86
- Copaxone (glatiramer acetate) - page 86
- KNS-760604 (R-pramipexole) - page 87
- Ceftriaxone - page 88
- Coenzyme Q10 - page 88
- FP-0011 - page 84
- Pipeline comparative forecasts - page 45
- BIBLIOGRAPHY - page 89
- Journals - page 89
- Websites - page 94
- Datamonitor reports - page 99
- APPENDIX A - page 100
- Methodology - page 100
- Datamonitor forecast methodology - page 100
- Product forecasts - page 100
- Definition of a Standard Unit - page 100
- Datamonitor forecast methodology - page 100
- Contributing experts - page 102
- Methodology - page 100
- APPENDIX B - page 103
- About Datamonitor - page 103
- About Datamonitor Healthcare - page 103
- Datamonitor Healthcare's therapy area capabilities - page 104
- About the Disease analysis team - page 105
- Disclaimer - page 106
- About Datamonitor - page 103
- List of Tables
- Table 1: Prevalence estimates of ALS across the 7MM, 2007 - page 17
- Table 2: Rilutek uptake in the three major market regions, 2006 - page 26
- Table 3: Cost of Rilutek (riluzole), 2006/07 - page 27
- Table 4: Key late-stage R&D pipeline in ALS, 2007 - page 39
- Table 5: Drug classes and modes of action for key ALS pipeline drugs - page 40
- Table 6: Estimated cost of Rilutek, Avonex and rhIGF-I , 2006 - page 53
- Table 7: Datamonitor drug assessment parameters - page 101
- List of Figures
- Figure 1: ALS market size and forecast-base scenario, 2006-16 - page 7
- Figure 2: ALS market size and forecast-IGF-1/IPLEX scenario, 2006-16 - page 8
- Figure 3: Drug assessment summary for ALS treatments - IGF-1/IPLEX successful clinical development scenario, 2007 - page 9
- Figure 4: Total ALS market sales revenue ($m), 2003-06. - page 20
- Figure 5: Prescription revenue versus prescription volume of Rilutek and other drugs in the seven major ALS markets, 2006 - page 21
- Figure 6: Rilutek revenue by market, 2006 - page 25
- Figure 7: Key unmet needs in ALS treatment - page 28
- Figure 8: ALS market size and forecast-base scenario, 2003-2016 - page 33
- Figure 9: ALS market size and forecast-IGF-1/IPLEX scenario, 2003-2016 - page 34
- Figure 10: Percentage change in Rilutek sales in the US Q3 2002 to Q2 2007 - page 35
- Figure 11: Rilutek sales revenue forecast across the 7MM, 2007-16 - page 36
- Figure 12: Origin of ALS key late-stage pipeline drugs, 2007 - page 42
- Figure 13: Forecast sales revenue for key ALS pipeline drugs, 2007-16 - page 46
- Figure 14: rhIGF-I sales revenue forecast based on the rhIGF-I /IPLEX successful clinical development scenario, 2007-16 - page 49
- Figure 15: Datamonitor's drug assessment summary for rhIGF-I - page 50
- Figure 16: IPLEX base scenario ALS sales revenue forecast, 2007-16. - page 59
- Figure 17: IPLEX sales revenue forecast, rhIGF-I/IPLEX successful clinical development scenario, 2007-16 - page 60
- Figure 18: Datamonitor's drug assessment summary for IPLEX - page 61
- Figure 19: E0302 (mecobalamin) ALS sales forecast, 2007-16 - page 68
- Figure 20: Datamonitor's drug assessment summary for E0302 (mecobalamin) - page 69
- Figure 21: Datamonitor drug assessment summary of pipeline molecular targeted therapies in development for hematological malignancies, 2007 - page 102
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