R&D Trends: Systemic Lupus Erythematosus - Benlysta’s FDA approval provides hope for disease treatment, future trial design, and other pipeline candidates

Date: May 1, 2011
Pages: 62
Price:
US$ 3,800.00
Publisher: Datamonitor
Report type: Strategic Report
Delivery: E-mail Delivery (PDF)
ID: R8D99DD2C57EN
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R&D Trends: Systemic Lupus Erythematosus - Benlysta’s FDA approval provides hope for disease treatment, future trial design, and other pipeline candidates
Introduction

There remains considerable debate over the optimum efficacy measure and clinical trial design in SLE. While the SLE Responder Index has proved successful in demonstrating the efficacy of Benlysta (belimumab; Human Genome Sciences/GlaxoSmithKline) such that many other trials are now following suit, it is unclear if this will spur other successes.

Features and benefits
  • Benchmark novel and existing therapies using the ideal target product profiles identified by Datamonitor and access leading rheumatologists’ opinion
  • Support R&D decision making by evaluating lupus clinical trial designs that have set a precedent, as well as analysis of discontinued projects
Highlights

Datamonitor identified 32 drug candidates in development for systemic lupus erythematosus (SLE), with four products in Phase III. Recent negative outcomes of major clinical trials threaten to move industry away from drug development in SLE, but Benlysta’s recent US approval may ignite further commercial interest in this disease.

Two new B-lymphocyte stimulator (BLyS) candidates have emerged within the late-stage SLE pipeline. Eli Lilly’s LY2127399 and Anthera Pharmaceuticals’ A-623 are both being investigated based on the novel SLE Responder Index. While this approach was successful for Benlysta, it remains to be seen if this can be emulated by other candidates.

The FDA published its formal guidance for industry for developing a medical product for SLE in June 2010, pointing to the use of disease activity indices in clinical trials. However, many specialists believe that the answer is to move away from these kinds of instruments and to check objective measures, such as biomarkers.

Your key questions answered
  • What lessons can be learned from past clinical trials in SLE and how can these be applied to novel pipeline candidates?
  • How does the recent approval of Benlysta impact the future of drug development for SLE?
  • What are the most promising trends seen in early stages of the SLE pipeline?
Executive Summary
  Strategic scoping and focus
  Datamonitor key findings
  Related reports

OVERVIEW

  Catalyst
  Summary

CLINICAL PIPELINE OVERVIEW

  Lupus pipeline
    Notable movements in the SLE pipeline
    Overall increase in the number of late-stage candidates
    B-cell directed therapies are leading the way
    Lack of industry-sponsored clinical trials conducted in Japan
  Compounds recently discontinued
    Orencia (abatacept; Bristol-Myers Squibb)
    Apremilast (CC-10004; Celgene)
    NNC-0152-0000-0001 (Novo Nordisk)
    Gusperimus trihydrochloride (NKT-01; Nippon Kayaku)
MEDI-546

TARGET PRODUCT PROFILE

  Off-label treatments are the gold-standard therapies for SLE
  Comparator one: Rituxan/MabThera (rituximab; Biogen Idec/Roche)
  Comparator two: CellCept (mycophenolate mofetil; Roche/Vifor Pharma)
  Target product profile versus current level of attainment

CLINICAL TRIAL DESIGN IN SYSTEMIC LUPUS ERYTHEMATOSUS

  Regulatory guidance on clinical trial design
    FDA finally publishes full formal industry guidance
    Summary of formal FDA guidance for industry in SLE
    Summary of FDA guidance for industry in lupus nephritis
    EMA guidelines
  Similarities can be seen in recent trial designs
  Standard of care
  Endpoints
    Variations in disease activity create a challenge
    Systemic Lupus Erythematosus Disease Activity Index (SLEDAI)
    The British Isles Lupus Assessment Group (BILAG)
    European Consensus Lupus Activity Measure (ECLAM)
    Systemic Lupus Erythematosus Activity Index (SLAM)
    Measurement of flares
    Extent of organ damage is critical to assessing global systemic lupus erythematosus disease activity
    Rheumatologists are only moderately satisfied with current treatment efficacy measurements
    Movement towards composite endpoints
    Quality of life measures used as secondary endpoints
    Steroid-sparing effects
  Future of lupus trial design
    More stringent subsetting of patients

INNOVATIVE EARLY-STAGE APPROACHES

  Biologics make up the majority of the early-stage pipeline
  Interferon inhibitors moving through the pipeline
  Cluster of differentiation drug candidates are seen in all phases of development
    CD20 product still in development for lupus
  Bortozemib: proteasome inhibitor shows early potential in lupus nephritis

THE FUTURE OF DISEASE TREATMENT IN SYSTEMIC LUPUS ERYTHEMATOSUS

  Targeted therapies to reduce dependence on steroids
  Identification of biomarkers will aid in development and treatment

BIBLIOGRAPHY

  Journal papers
  Websites
  Datamonitor reports
  Other

APPENDIX A: SURVEY INFORMATION

  Physician research methodology
  The survey questionnaire

APPENDIX B

  Contributing experts
  Report methodology

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R&D Trends: Systemic Lupus Erythematosus - Benlysta’s FDA approval provides hope for disease treatment, future trial design, and other pipeline candidates
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