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

Time: 9:30 am
day: Conference Day 1 AM


  • A first clinically and biologically defined subgroup of ASD patients, named ASD-Phen1, was clinically validated in 2 prospectives biosampling studies with an observed incidence of 20-25% of the enrolled ASD population
  • This subgroup was then biologically validated through multi-omics characterization revealing specific molecular signatures for ASD-Phen1 patients that were found to be reversed by STALICLA's precision medicine STP1
  • 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