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Clinical Trial Design Improvements Could Spur Drug Development for Autism and Traumatic Brain Injury, While Biomarkers Appear Key in the Discovery of Treatments for Amyotrophic Lateral Sclerosis

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Decision Resources Group finds that neurologists specializing in autism spectrum disorder (ASD) consider its heterogeneity a key obstacle to developing pharmacotherapies that effectively address core behavioral symptoms—the greatest unmet need among patients with ASD. Relying on differences in clinical presentation to guide patient selection for clinical trials is put forward as a possible way to enhance the chance of observing a therapeutic effect, a suggestion that is echoed by neurologists treating traumatic brain injury (TBI) patients, a neurological condition with similarly complex pathophysiological underpinnings. For amyotrophic lateral sclerosis (ALS), a rare disease that in most cases is not caused by genetic mutations known to date, the development of biomarkers is a prerequisite to discovering effective disease-modifying interventions, according to interviewed experts.

Other key findings from the Niche Markets and Rare Diseases reports entitled Autism Spectrum Disorder, Traumatic Brain Injury and Amyotrophic Lateral Sclerosis:

  • ASD affects more than 1.5 million patients in the United States and the five major European markets (France, Germany, Italy,Spain, and the United Kingdom), a number that is expected to grow over our ten-year study period, underscoring a large market opportunity for novel behavioral treatments. However, the ASD pipeline is limited, and Roche’s RG-7314 is the only small molecule therapy currently in trials for social responsiveness—a core behavioral symptom of ASD.
  • Interviewed neurologists are discouraged by repeat failures of investigational TBI agents but look forward to results from the ongoing, large-scale, observational TRACK-TBI and CENTER-TBI studies. Data from these studies, which aim to inform clinical trial design, and validate biomarkers and outcome measures, could greatly facilitate the development of sorely-needed new drugs for TBI.
  • The ALS pipeline is active and comprises of a variety of agents, from small molecules to stem-cell therapies, targeting different pathways thought to underlie the disease. Interviewed experts express varying degrees of interest in pipeline agents but generally anticipate extensive prescribing of Cytokinetics’ Phase III clinical candidate, tirasemtiv, if it becomes available.

Comments from Decision Resources Group Director Nadja Rozovsky, Ph.D.:

  • “Limited understanding of underlying pathophysiological mechanisms is a common theme across ASD, TBI, and ALS and one of the greatest obstacles to the development of effective treatments for each disease. Interviewed neurologists are hopeful that ongoing genetic research into ASD and disorders with similar manifestations, genetic and other basic research into ALS, and large-scale observational trials in TBI would yield some of the needed clues to facilitate the development of novel treatments.”
  • “While commercial interest in ALS is strong, fewer pharmaceutical companies remain active in ASD or TBI, highlighting challenges that have confounded drug development for these indications. However, the large size of both the ASD and TBI patient populations, coupled with a pressing unmet need, underscore a strong commercial potential for each.”

For more information on purchasing this report, please email questions@teamDRG.com.

Source: PR Newswire