Pipeline Insight:Orphan Diseases in CNS - Part II

Content

• 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
  • Sizing The Als Market
  • 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
  • Drug Overview
  • 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