Sunyoung Kim | Executive Head
JW Pharmaceutical

Day 1 - World Orphan Drug Congress USA 2026 @ 11:50

A Novel Oral Therapy for Rare Neurodevelopmental Disorders: Preclinical Efficacy in PTHS, FXS, and RTT with Potential Disease Modification and Expansion into ASD

Pitt-Hopkins syndrome (PTHS), Fragile X syndrome (FXS), and Rett syndrome (RTT) are severe neurodevelopmental disorders lacking effective disease-modifying therapies. DDC-02 is a first-in-class, orally available small molecule that modulates the CCND2–CDK5 axis, inducing Axin1 degradation and activating β-catenin–mediated transcription to restore gene networks underlying cognitive and behavioral function.

DDC-02 demonstrated robust cognitive and behavioral improvements in mouse models of PTHS and FXS and zebrafish models of PTHS and RTT, supporting a trans-diagnostic, disease-modifying mechanism. The compound is CNS-penetrant, suitable for oral administration, and advancing toward clinical evaluation, with a Phase 1/2 trial planned in PTHS patients.

By directly targeting core neurodevelopmental pathways, DDC-02 represents a differentiated therapeutic approach with potential applicability across genetically distinct NDDs, including broader autism spectrum disorders.