Clinical Translation of Precision Medicine in a First Clinically & Biologically Defined Subgroup of Patients with Autism Spectrum Disorder

<p xmlns:xsi="http://www.w3.org/2001/XMLSchema-instance" class="first" dir="auto" id="d1771143e105">Limited behavioral DSM-5 diagnostic criteria fails to acknowledge the significant heterogeneity present in neurodevelopmental disorders such as Autism Spectrum Disorder (ASD), contributing to repeated clinical trial failures in the field. To address this challenge, STALICLA has developed a unique precision neuroscience data platform, Databased Endophenotyping Patient Identification (DEPI), that enables the identification of stratified patient subgroups and advances tailored treatment options for ASD. A first clinically and biologically defined subgroup of ASD patients, named ASD-Phen1, was clinically validated in 2 prospectives biosampling studies (Greenwood Genetic Center, Cincinnati Children’s Medical Center) with an observed incidence of 20-25% of the enrolled ASD population (Perez-Cano, et al. 2024). This subgroup was then biologically validated through multi-omics characterization (metabolic, metabolomic, transcriptomic) revealing specific molecular signatures for ASD-Phen1 patients that were found to be reversed by STALICLA's precision medicine STP1 (a combination of a PDE4/3 inhibitor (ibudilast) and an NKCC1 antagonist (bumetanide)). Ensuing STP1 Phase 1b in ASD-Phen1 established good safety-tolerability, strong target engagement signals (EEG), and superiority on recognized-clinical efficacy endpoints (ABC, SRS, NIH toolbox) as well as convincing PK/PD profile (Erickson, et al. 2024). <p xmlns:xsi="http://www.w3.org/2001/XMLSchema-instance" dir="auto" id="d1771143e107"/><p xmlns:xsi="http://www.w3.org/2001/XMLSchema-instance" dir="auto" id="d1771143e108"/><p xmlns:xsi="http://www.w3.org/2001/XMLSchema-instance" dir="auto" id="d1771143e109"/><p xmlns:xsi="http://www.w3.org/2001/XMLSchema-instance" dir="auto" id="d1771143e110"/><p xmlns:xsi="http://www.w3.org/2001/XMLSchema-instance" dir="auto" id="d1771143e111"/>