R&D Trends: Autism Spectrum Disorders – Pipeline Growth Reflects Increasing Interest in Autism
Datamonitor, April 2012, Pages: 62
This report reviews the key trends in the autism spectrum disorders pipeline. The analysis is intended to support R&D decision-making by evaluating the dynamics of the pipeline and hypothesizing future treatments of autism spectrum disorders.
The autism spectrum disorders (ASDs) market is a challenging one to penetrate; since 2006 only two products have been successful in entering this marketplace: Risperdal (risperidone; Johnson & Johnson) and Abilify (aripiprazole; Bristol-Myers Squibb). However, as additional agents near the market, increasing interest in ASDs has led to a sizable growth in the clinical pipeline.
Scope
- Overview of drugs in late- and early-stage clinical development for autism spectrum disorders.
- Identification of the target product profile for future autism spectrum disorders treatments.
- Summary of clinical trial design in autism spectrum disorders including exploration of key endpoints and assessment methodology.
- Identification of key challenges associated with the conduct of clinical trials in autism spectrum disorders.
- Exploration of future treatment in autism spectrum disorders including the development of personalized treatments and the use of biomarkers.
Highlights
- The autism spectrum disorders (ASDs) pipeline remains sparse relative to other conditions that are treated with psychotropic medications. The unknown etiology of ASDs poses a fundamental challenge to the therapeutic discovery process. However, the pipeline growth is encouraging and reflects an increased interest in ASDs.
- The two approved therapies for the management of autism spectrum disorders both target the symptoms of irritability. The comparator therapy identified by Datamonitor is Risperdal (risperidone; Johnson & Johnson) as it was the first to be approved in this indication, demonstrates a strong efficacy profile, and is regarded as a first-line therapy.
- Key challenges associated with the conduct of clinical trials in autism spectrum disorders are: the unknown etiology of the disorders and heterogeneous symptomology. Communication and language impairments commonly associated with autism spectrum disorders further compound these challenges
Reasons to Purchase
- How many drugs are in clinical development for the treatment of autism spectrum disorders? What deters companies from investing in this area?
- What symptoms and mechanisms of action are most frequently targeted? What key companies are involved in the pipeline?
- What is the target product profile and most appropriate comparator therapy?
- What are the key challenges in conducting clinical trials in autism spectrum disorders?
- How is the treatment of autism spectrum disorders likely to evolve over the next 10 to 20 years?
OVERVIEW
- Catalyst
- Summary
EXECUTIVE SUMMARY
- Update details: April 2012
- Strategic scoping and focus
- Datamonitor key findings
- Related reports
CLINICAL PIPELINE OVERVIEW
- Overview of the autism spectrum disorders pipeline
- - The sparse autism pipeline demonstrated encouraging growth and progression over April 2011 to April 2012
- - Emerging features of the autism spectrum disorders clinical pipeline
- Classification of pipeline products- - Each of the eight pipeline candidates targets a different mechanism of action
- Companies involved in the autism spectrum disorders clinical pipeline
- - Pipeline activity is dominated by small, specialist pharmaceutical companies
- Late-stage development compounds recently discontinued
- - Pipeline failures prevalent due to unknown etiology of autism spectrum disorders
TARGET PRODUCT PROFILE
- Clinical targets
- - Clinical trial data indicate a lack of efficacy of investigated therapies
- - Although superior to Risperdal, side effects are also prominent with Abilify treatment
- - Ease of administration is a key consideration in the development of treatments for the pediatric population
- Comparator therapy: Risperdal (risperidone; Johnson & Johnson)
- - Risperdal was the first drug to receive FDA approval for irritability associated with autistic disorder
- - Clinical trial data for Risperdal
- Target product profile versus current level of attainment
- - Target product for the treatment of irritability should demonstrate superior efficacy and side effect-profile to Risperdal
CLINICAL TRIAL DESIGN IN AUTISM SPECTRUM DISORDERS
- Use of clinical trial endpoints varies according to the target behavioral symptom
- - Core symptoms
- - Repetitive behavior
- - Irritability
- - ADHD symptoms
- - Social impairment
- - Typical clinical trial design
- Key challenges in the conduct of clinical trials in autism spectrum disorders
- - Unknown etiology of autism spectrum disorders hinders selection of appropriate targets
- - Heterogeneity of symptomatology complicates patient subgroup identification
- - Conduct of trials in children and young adolescents is complicated by communication difficulties
- Future developments in clinical trial design
- - Clinical trials are likely to include adult autism spectrum disorder patients in the future
- - Increased patient selection will help to increase response rates in clinical trials
INNOVATIVE EARLY-STAGE APPROACHES
- Preclinical development pipeline contracts by 33% between April 2011 and April 2012
- - Scientific rationale determines targets, but clinical efficacy needs to be demonstrated
THE FUTURE OF TREATMENT IN AUTISM SPECTRUM DISORDERS
- Continued research into the etiology of ASDs is essential for development of treatments
- - Elucidating the genetic and environmental interactions will aid treatment discovery
- - Multicenter industry-academic collaboration seeks to develop novel therapies
- Biomarkers will aid the development of personalized treatment options
- - Promise for identification, earlier intervention, and treatment response
- - Subpopulation stratification will increase treatment response
BIBLIOGRAPHY
- Journal papers and publications
- Websites
- Datamonitor reports
APPENDIX
- Contributing experts
- Conferences attended
- Report methodology
TABLES
Table: Products in clinical development for autism spectrum disorders, April 2012
Table: Compounds under investigation for the treatment of ASDs by the NIH or universities, April 2012
Table: Products in clinical development by the National Institutes of Health for autism spectrum disorders, April 2012
Table: Autism spectrum disorder late-stage pipeline candidates discontinued, 2006–12
Table: Risperdal (risperidone; Johnson & Johnson) – drug profile for autism spectrum disorders, 2011
Table: Overview of pivotal trials conducted for Risperdal in autism spectrum disorders
Table: Overview of side effects associated with Risperdal in two pivotal Phase III clinical trials
Table: Risperdal versus the minimum acceptable product profile and target product profile, 2011
Table: Typical Phase III clinical trial design in autism spectrum disorders
Table: Candidates under preclinical investigation for the treatment of autism spectrum disorders, April 2012
FIGURES
Figure: Number of candidates in clinical development for autism spectrum disorders versus other mental health conditions treated with psychotropic medication, April 2012
Figure: Pipeline agents for the treatment of autism spectrum disorders, by development stage, April 2012
Figure: Pipeline agents for the treatment of autism spectrum disorders, split by target indication, April 2012
Figure: Pipeline agents for the treatment of autism spectrum disorders, split by formulation, April 2012
Figure: Pipeline agents for the treatment of autism spectrum disorders, split by target mechanism, April 2012
Figure: Collaborating partners with National Institutes of Health for the investigation of compounds for the treatment of autism spectrum disorders, April 2012
Figure: Datamonitor’s timeline for the evolution of the autism spectrum disorders market, 2006–20
- Celgene Corporation
- Commerzbank AG
- Forest Laboratories Inc.
- GlaxoSmithKline Plc
- Hutchison 3G UK Limited
- Informa plc
- Janssen Pharmaceuticals Inc.
- LVMH Moet Hennessy Louis Vuitton SA
- Wolters Kluwer nv
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