Stalicla’s precision therapeutic enters the clinic

Autism spectrum disorder (ASD) is a group of neurodevelopmental disorders (NDDs) currently diagnosed based on core behavioral features. Globally, ASD is highly prevalent and affects approximately 10 million patients in Europe and the United States and more throughout the world. There are no biological criteria for this diagnosis. ASD is characterized by high levels of clinical and genetic heterogeneity, supporting the existence of ASD biological subtypes requiring differential pharmacological treatment strategies.

By combining real-world patient data and its proprietary systems biology and AI-powered drug discovery platform called DEPI (Databased Endophenotyping Patient Identification), STALICLA is uncovering novel patterns of non-behavioral and biologically defined parameters amongst patients with ASD. STALICLA has already identified ASD Phenotype 1, a first subpopulation of patients with idiopathic ASD that can be identified by distinct multi-omics biomarkers.

“Despite the high prevalence of neurodevelopmental disorders in the U.S. and Europe, most NDDs are diagnosed and treated based on behavioral-based classification systems alone,” said Study Investigator, Craig Erickson, MD, Cincinnati Children’s Hospital Medical Center. “I look forward to investigating the potential of STP1 in the first clinically validated subpopulation of patients with idiopathic ASD.”

The double-blind, placebo-controlled, first-in-human study is being conducted at Cincinnati Children’s Hospital Medical Center in Cincinnati, Ohio. The primary objective of the trial is to evaluate the safety and tolerability of a two-week oral treatment with STP1 in adult patients who match the ASD-Phen1 clinical criteria. Two ascending dose cohorts of approximately 12 patients each will be enrolled. The study will also assess the pharmacokinetic and pharmacodynamic effects of STP1 and includes exploratory efficacy surrogate endpoints.