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Cogstate Establishes Rare Disease and Pediatric Center of Excellence

April 5, 2018

Dr. Pam Ventola appointed to lead implementation as Senior Science Director, Rare Disease and Pediatric Center of Excellence

New Haven, CT – April 5, 2018: The cognitive science company, Cogstate Ltd (ASX.CGS) today announced the establishment of a Rare Disease and Pediatric Center of Excellence to lead all activities related to the selection and administration of cognitive and behavioral outcome measures in rare disease and pediatric clinical trials. This new center of excellence provides pharmaceutical sponsors with industry-leading clinical and scientific expertise focused on the selection, adaptation, optimization, validation and correct administration of cognitive and behavioral measures that address the unique challenges of clinical studies in rare disease and pediatric patient populations.

The group will be led by Pam Ventola, PhD, who will apply her scientific leadership experience in pediatric neuropsychology and specifically developmental and genetic disorders to drive the implementation of Cogstate’s center of excellence, overseeing project teams and consultants with deep expertise in rare disease and pediatric trial design and execution.

“For rare disease trials, we at Cogstate approach endpoint selection with an inherent understanding of cognitive development, which is critical in rare diseases, as individuals often present with intellectual disabilities and complex presentations across multiple domains of functioning,“ said Dr. Ventola. “Furthermore, we appreciate that a patient-centric approach is essential in rare disease trials. These studies are often quite small and every data point is critical, therefore when selecting endpoints, we need to ensure the measures are appropriate for, and acceptable to, the patient group.”

Brad O’Connor, Cogstate CEO commented, “It is estimated that 350 million people worldwide suffer from rare diseases—half being children—and 95% go untreated. An important part of bringing new medicines to market for these underserved populations is ensuring the endpoints in the trials are appropriate and that the tests themselves are consistently executed across the study. We’re proud to formalize our long history of supporting pediatric and rare disease research with this center of excellence, and we are thrilled that an expert as accomplished as Dr. Ventola has joined us to lead these efforts.”

This center of excellence further enhances Cogstate’s strong rare disease and pediatric clinical development capabilities and allows the company to expand its support for the cognitive and behavioral measures in these trials. To date, Cogstate has supported over 40 pediatric and rare disease trials in areas such as pediatric cancers, neurological and developmental disorders, Lysosomal storage disorders, psychiatric disorders, hematologic disorders, liver disorders, rare epileptic syndromes, metabolic disorders and a range of other rare genetic disorders.

About Pam Ventola, PhD
Pam Ventola is Senior Science Director, Rare Disease and Pediatric Center of Excellence at Cogstate, where she provides pharmaceutical sponsors with strategic oversight and expert guidance throughout all stages of their study planning and execution – from endpoint selection, rater training and strategic monitoring to final statistical analysis. Dr. Ventola joins Cogstate from the Yale University School of Medicine, Child Study Center where she continues to hold a faculty appointment as Assistant Clinical Professor. A licensed clinical psychologist, Dr. Ventola is an expert in pediatric neuropsychology and specifically developmental and genetic disorders. Her research interests involve developmental disabilities, and among her recent research is a study on brain-based mechanisms of treatment response in autism spectrum disorder (ASD). She is also utilizing eye-tracking paradigms as a novel outcome of clinical trials in ASD and is involved in studies on novel pharmacological therapeutics in individuals with ASD. Dr. Ventola received her Doctor of Philosophy in Clinical Psychology from University of Connecticut and completed her clinical training and Postdoctoral Fellowship at the Yale University School of Medicine, Child Study Center. She serves on the editorial review board of multiple academic journals. Specifically, she serves as a reviewer for the Journal of Autism and Developmental Disorders, Journal of the American Academy Child and Adolescent Psychiatry, Clinical Psychology Review, and the Autism International Journal of Research and Practice. Dr. Ventola has authored numerous peer-reviewed manuscripts, book chapters and scientific presentations.

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