Abstract
Overview
Introduction
The ALS market was worth $148m in 2006 across the seven major markets. With
the only approved treatment for ALS, Sanofi-Aventis' s Rilutek (riluzole),
facing generic incursion in 2012-13, future growth in this market is dependent
on the success of pipeline drugs.
Scope
- Scenario based analysis and forecasts of the commercial potential of the
ALS market to 2016.
- Analysis of patient potential, marketing factors, commercial
attractiveness and clinical attractiveness of key late-stage pipeline drugs.
- Strategic recommendations for maximizing the potential of drugs in this
niche orphan disease market.
- Indication-specific sales forecasts of marketed and key pipeline drugs to
2016.
Report Highlights
A lack of new entrants, and the possibility of polytherapy, results in
Sanofi-Aventis' s Rilutek (riluzole) dominating the market until patent expiry
in 2012-13 in Datamonitor' s base scenario.
Orphan drug status, rapid uptake following launch, limited risk to the
company' s share price if drugs fail, and possible indication expansions make
long-term investment in ALS a commercially viable option.
A scenario forecast based on the clinical success of rhIGF-1 (recombinant
human insulin-like growth factor I) related therapies reveals a potentially
lucrative market. However, with a high attrition rate of ALS Phase III
clinical trials, and mixed efficacy data in Phase II, the results of currently
underway studies will be pivotal.
Reasons to Purchase
- Access the lucrative potential of this underdeveloped neurodegenerative
disease therapeutic market.
- Benchmark key late-stage compounds against the current leading therapy
Sanofi-Aventis' s Rilutek (riluzole).
- Understand the opportunities in this market based on the comments of
interviewed key opinion leaders.
Table of Contents
- ABOUT DATAMONITOR HEALTHCARE
- About the CNS pharmaceutical analysis team
- CHAPTER 1 EXECUTIVE SUMMARY
- Scope of the analysis
- Contributing experts
- Datamonitor insight into the amyotrophic lateral sclerosis market
- Key metrics
- Scenario forecasts for ALS pipeline drugs and total market value
- Base scenario
- rhIGF-I /IPLEX successful clinical development scenario
- Datamonitor pipeline assessment summary
- Related reports
- CHAPTER 2 MARKET OVERVIEW
- Definition of amyotrophic lateral sclerosis
- Segmentation of amyotrophic lateral sclerosis
- Etiology
- Etiology of ALS is largely unknown
- Prognosis of amyotrophic lateral sclerosis sufferers
- Epidemiology
- Diagnosis and treatment rates
- Diagnosis of ALS-ruling out other diseases first
- Treatment rates of ALS-the uptake of Rilutek
- Market size
- Rilutek (riluzole)
- Drug overview
- Clinical trial information
- Pivotal Rilutek efficacy trial
- Phase III survival trial
- Efficacy has broadly been accepted
- Rilutek sales
- Rilutek uptake
- Unmet need in amyotrophic lateral sclerosis
- Clinical trial design
- CHAPTER 3 FUTURE MARKET POTENTIAL
- Scenario forecasts for ALS total market value
- Base scenario
- rhIGF-I /IPLEX successful clinical development scenario
- Forecast Rilutek sales
- Little switching between Rilutek and new therapies
- Rilutek patent protection
- US-Patent US-05527814 should protect Rilutek until 2013
- 5EU-new use patent should protect Sanofi-aventis until late 2012
- Japan-new use patent should protect Sanofi-aventis until late 2012
- CHAPTER 4 PIPELINE OVERVIEW AND DYNAMICS
- Pipeline overview
- Classification of pipeline products
- Advantages of developing an ALS treatment
- NINDS funding of clinical trials
- For mid-to-large cap companies, ALS offers little risk
- Indication expansion post-approval for ALS offers future potential
- Orphan drug designation has additional benefits
- Current comparator therapy-Rilutek
- CHAPTER 5 LATE-STAGE DRUG ANALYSIS & FORECASTS
- Pipeline comparative forecasts
- Two scenarios have been explored
- rhIGF-I-recombinant human insulin-like growth factor I
- Drug overview
- Forecasts to 2016
- Base scenario
- rhIGF-I /IPLEX successful clinical development scenario
- Datamonitor drug assessment summary-IGF-1/IPLEX successful clinical
development scenario
- Marketing factors-rhIGF-I /IPLEX successful clinical development
scenario
- Marketing of rhIGF-I likely if the NINDS-sponsored trial is
successful
- rhIGF-I will quickly be replaced with the combination product IPLEX
- Cost of IGF-1
- Patient potential
- Satisfaction of unmet needs
- Better/alternative drugs to slow the progression of the disease
- Clinical trial data
- Cochrane Database Systematic Review of rhlGF-1 for ALS
- First clinical trial investigating the use of rhIGF-I in ALS
- Smaller negative trial investigating rhIGF-I and ALS
- Ongoing clinical trial program
- IPLEX (mecasermin rinfabate, rhIGF-I/IGFBP-3)
- Drug overview
- Forecasts to 2016
- Base scenario
- IGF-1/IPLEX successful clinical development scenario
- Datamonitor drug assessment summary-IGF-1/IPLEX successful clinical
development scenario
- IPLEX market penetration-two scenarios
- Marketing factors
- Withdrawal from US short-stature market
- Development for ALS likely to receive a higher priority
- IPLEX cost and dose
- IPLEX market penetration-two scenarios
- Patient potential
- Satisfaction of unmet needs
- Clinical trial data
- Radicut (edaravone, MCI-186)
- Drug overview-Phase III
- Lack of clinical data precludes assessment of commercial potential
- Clinical trial data
- Ongoing clinical trials
- E0302 (mecobalamin)
- Drug overview-Phase II/III
- Forecasts to 2016-applicable in both forecast scenarios
- Datamonitor drug assessment summary-IGF-1/IPLEX successful clinical
development scenario
- Marketing factors
- Cost of E0302 in Japan
- Uptake of E0302 in Japan
- E0302/mecobalamin in the ex-Japan markets
- Patient potential
- Satisfaction of unmet needs
- Clinical trial data
- Ongoing clinical trials
- Creatine-based preparations
- ALS-02-creatine monotherapy
- Marketing factors
- Creatine supplements are widely available over-the-counter
- Possible advantage of physician monitoring and reimbursement
- Potential cost and maximum revenue for ALS-02.
- Patient potential
- Satisfaction of unmet needs
- Clinical trial data
- Maximum voluntary isometric contraction
- Double-blind trial of survival and disease progression
- Small trial in advanced ALS patients monitoring respiratory function
- Clinical trial to examine the muscular effects of creatine in ALS
patients
- Pilot study investigating creatine' s effect on muscle strength in
ALS patients
- Ongoing clinical trials
- ALS-08-creatine in combination with minocycline or celecoxib
- Minocycline combination shown to be inferior to celecoxib combination
- Minocycline monotherapy proved to be detrimental to patients
- Celecoxib combination-non-futile compared to historical controls
- Celecoxib monotherapy trial-no clear efficacy as a monotherapy
- Arimoclomol
- Drug overview-Phase II
- Clinical trial data
- Multi-center Phase IIa clinical trial monitoring safety and
tolerability of arimoclomol
- Open-label extension clinical trial of arimoclomol in ALS
- Ongoing clinical trial program
- Other ALS pipeline drugs
- FP-0011
- Drug overview-Phase II
- Clinical trial data
- Cereact
- Drug overview
- Clinical trial data
- Talampanel
- Copaxone (glatiramer acetate)
- KNS-760604 (R-pramipexole)
- Ceftriaxone
- Coenzyme Q10
- BIBLIOGRAPHY
- Journals
- Websites
- Datamonitor reports
- APPENDIX A
- Methodology
- Datamonitor forecast methodology
- Product forecasts
- Definition of a Standard Unit
- Contributing experts
- APPENDIX B
- About Datamonitor
- About Datamonitor Healthcare
- Datamonitor Healthcare' s therapy area capabilities
- About the Disease analysis team
- Disclaimer
- List of Tables
- Table 1: Prevalence estimates of ALS across the 7MM, 2007
- Table 2: Rilutek uptake in the three major market regions, 2006
- Table 3: Cost of Rilutek (riluzole), 2006/07
- Table 4: Key late-stage R&D pipeline in ALS, 2007
- Table 5: Drug classes and modes of action for key ALS pipeline drugs
- Table 6: Estimated cost of Rilutek, Avonex and rhIGF-I , 2006
- Table 7: Datamonitor drug assessment parameters
- List of Figures
- Figure 1: ALS market size and forecast-base scenario, 2006-16
- Figure 2: ALS market size and forecast-IGF-1/IPLEX scenario, 2006-16
- Figure 3: Drug assessment summary for ALS treatments - IGF-1/IPLEX
successful clinical development scenario, 2007
- Figure 4: Total ALS market sales revenue ($m), 2003-06.
- Figure 5: Prescription revenue versus prescription volume of Rilutek
and other drugs in the seven major ALS markets, 2006
- Figure 6: Rilutek revenue by market, 2006
- Figure 7: Key unmet needs in ALS treatment
- Figure 8: ALS market size and forecast-base scenario, 2003-2016
- Figure 9: ALS market size and forecast-IGF-1/IPLEX scenario, 2003-2016
- Figure 10: Percentage change in Rilutek sales in the US Q3 2002 to Q2
2007
- Figure 11: Rilutek sales revenue forecast across the 7MM, 2007-16
- Figure 12: Origin of ALS key late-stage pipeline drugs, 2007
- Figure 13: Forecast sales revenue for key ALS pipeline drugs, 2007-16
- Figure 14: rhIGF-I sales revenue forecast based on the rhIGF-I /IPLEX
successful clinical development scenario, 2007-16
- Figure 15: Datamonitor' s drug assessment summary for rhIGF-I
- Figure 16: IPLEX base scenario ALS sales revenue forecast, 2007-16.
- Figure 17: IPLEX sales revenue forecast, rhIGF-I/IPLEX successful
clinical development scenario, 2007-16
- Figure 18: Datamonitor' s drug assessment summary for IPLEX
- Figure 19: E0302 (mecobalamin) ALS sales forecast, 2007-16
- Figure 20: Datamonitor' s drug assessment summary for E0302
(mecobalamin)
- Figure 21: Datamonitor drug assessment summary of pipeline molecular
targeted therapies in development for hematological malignancies, 2007
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