R&D Trends: Autism Spectrum Disorders - Unknown etiology and low industry interest hinders pipeline progression

Introduction

Since 2006, two drugs have gained FDA approval for the symptomatic treatment of irritability in autism spectrum disorders (ASDs). However, the pipeline is also characterized by a high attrition rate, with eight pipeline candidates having been discontinued since 2009. CureMark’s Phase III candidate, CM-AT, currently represents the most advanced compound in the small ASD pipeline.

Features and benefits

• 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 the future treatment in autism spectrum disorders including the development of personalized treatments and the use of biomarkers.

Highlights

The autism spectrum disorders (ASDs) pipeline is 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 and the pipeline is characterized by a high attrition rate.

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.

Your key questions answered

• 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 the conduct of clinical trials in autism spectrum disorders?
• How is the treatment of autism spectrum disorders likely to evolve over the next 10 to 20 years?