JW Pharmaceutical has presented preclinical findings and its global development strategy for DDC-02, a therapeutic candidate for rare neurodevelopmental disorders, at the World Orphan Drug Congress USA 2026 in Boston.
The presentation featured data demonstrating the effects of DDC-02 across animal models of several rare neurodevelopmental disorders, including Pitt-Hopkins syndrome (PTHS), fragile X syndrome (FXS), and Rett syndrome (RTT), each of which arises from distinct genetic causes. Across these models, DDC-02 consistently restored deficits in cognitive and behavioural function.
DDC-02 is an orally administered small-molecule candidate discovered by JW Pharmaceutical. The compound has been shown to modulate intracellular signalling pathways involved in neurodevelopment and neural circuit function. In preclinical studies, DDC-02 restored impaired cognitive and behavioural performance to levels comparable to those observed in healthy control animals across models of PTHS, FXS, and RTT. These findings suggest that DDC-02 may improve cognitive and behavioural function by modulating synaptic plasticity and neural circuit activity. Taken together, the data indicate DDC-02’s potential applicability across multiple rare neurodevelopmental disorders sharing common deficits in cognition and behaviour, despite their distinct genetic origins.
Cognitive and behavioural function was restored to normal levels even in animals with well-established disease phenotypes. Neurodevelopmental disorders are generally believed to be difficult to reverse once symptoms have become established, as they arise from abnormalities occurring during early stages of brain development. The ability of DDC-02 to restore function in these models suggests that meaningful recovery may be achievable even after disease manifestations have emerged, highlighting the potential for functional restoration within mature neural circuits.
JW Pharmaceutical is evaluating Pitt-Hopkins syndrome as the lead indication for DDC-02 and is targeting entry into global multi-regional clinical studies in 2028. It plans to subsequently expand development into additional rare neurodevelopmental disorders, including fragile X syndrome and Rett syndrome.
The company also outlined its global development and partnering strategy during WODC USA 2026. Throughout the conference, JW Pharmaceutical held business development meetings with pharmaceutical companies, biotechnology firms, and investment groups to explore a range of collaboration opportunities, including co-development partnerships and licensing transactions.
Sun Young Kim, executive head of R&D strategy at JW Pharmaceutical said: “DDC-02 has demonstrated consistent efficacy across neurodevelopmental disorder models with distinct genetic etiologies.
“The observation that cognitive and behavioural function was restored to normal levels even in adult disease models is encouraging. We believe these findings support the potential of DDC-02 as a novel therapeutic approach for patients with rare neurodevelopmental disorders. We expect to advance DDC-02 through global clinical development and strategic partnerships to bring a meaningful new treatment option to patients and families affected by these conditions.”