漢汀頓症的治療商機

Abstract

Introduction

Huntington' s disease (HD) is a genetic neurological disorder characterized by uncontrolled body movements and cognitive dysfunction. No drugs are approved to treat the disorder, leaving a high level of unmet need. Commercial opportunity abounds, but the small patient population deters some companies. However, because of novel treatment approaches, interest in HD research is growing and companies may be able to tap into the enormous commercial potential, especially if a drug can treat multiple neurological diseases.

Questions Answered in This Spectrum Report:

• Huntington' s disease has a small patient population across all markets. What are the advantages and disadvantages for drug developers? What effect does orphan-drug status have on a drug' s potential?
• No therapies are approved specifi cally for HD; all current therapies are used off-label and provide only symptomatic relief. Which drugs are being investigated in clinical trials? What are the diffi culties? When will a drug launch for HD?
• Novel treatment approaches including disease-modifying stem-cell and gene therapies are making some headway. What companies are investigating HD treatments and what are the most promising treatment approaches? At what stage are these treatments, and what are the chances of their launching for HD?

Scope:

• Overview of Huntington' s disease: genetics, pathophysiology, epidemiology, symptoms, and diagnosis.
• Current treatment approach: no therapies are approved for HD; several drugs are used off-label to treat symptoms.
• Emerging therapies: disease-modifying agents, including Amarin' s Miraxion (Phase III) and Avicena' s HD-02 (Phase II), and symptomatic therapies, including Prestwick' s Xenazine (preregistered) and NeuroSearch' s ACR-16 (Phase II).
• Novel treatment approaches: stem-cell transplantation and gene therapy.
• Challenges and outlook: the future of the HD market.

Mentioned in This Spectrum Report:

Companies:

• Abbott
• Amarin
• Amgen
• AstraZeneca
• Avicena
• Bristol-Myers Squibb
• Carlsson Research
• Ceregene
• Edison Pharmaceuticals
• Eli Lilly
• Endo Pharmaceuticals
• EnVivo
• Janssen
• Medivation
• MethylGene
• NeuroSearch
• Novartis
• Pfi zer
• Prestwick Pharmaceuticals
• Renovis
• Sanofi -Aventis
• Wyeth

Drugs:

• ACR-16
• Amantadine (Symmetrel)
• Artane (trihexyphenidyl)
• Atomoxetine (Strattera)
• CERE-120
• Citalopram (Celexa)
• CoEnzyme Q10
• Depakene (valproic acid)
• Depakote (valproic acid and divalproex sodium)
• Dimebon
• HD-02
• Memantine (Namenda)
• Minocycline
• Miraxion
• NXY-059
• Phenylbutyrate
• Prozac (fl uoxetine)
• Rilutek (riluzole)
• Risperdal (risperidone)
• SER-AP-ES (reserpine)
• Seroquel (quetiapine)
• Sinemet (levodopa)
• Xenazine (tetrabenazine)
• Zoloft (sertraline)

Table of Contents

• Executive Summary
  • Strategic Considerations
  • Stakeholder Implications
• Introduction
• Overview of Huntington' s Disease
  • The Genetics of Huntington' s Disease
  • Pathophysiology
  • Disease Onset, Symptoms, and Diagnosis
  • Epidemiology and Orphan Disease Status
• Current Treatment Strategies
• Emerging Therapies
  • Disease-Modifying Agents
    • Amarin' s Miraxion
    • Avicena' s HD-02
    • MethylGene and EnVivo' s Phenylbutyrate
    • Neurotrophic Factors: BDNF, GDNF, CNTF
  • Symptomatic Emerging Therapies
    • Prestwick Pharmaceuticals' Xenazine
    • NeuroSearch' s ACR-16
    • Sanofi -Aventis' s Rilutek
    • Medivation' s Dimebon
• Challenges and Opportunities

Tables:

• 1. Inheritance Patterns of Huntington' s Disease
• 2. Effect of CAG Repeats on Huntington' s Disease Development
• 3. Agents in Commercial Development for the Treatment of Huntington' s Disease
• 4. Agents Under Academic Investigation for the Treatment of Huntington' s Disease

Figures:

• 1. Brain Structures Implicated in Huntington' s Disease
• 2. Prevalence of Huntington' s Disease in Select Geographic Regions

Sidebars:

• Novel Approaches to the Treatment of Huntington' s Disease: Stem-Cell Transplantation
• Novel Approaches to the Treatment of Huntington' s Disease: Gene Therapy