JW Pharmaceutical announced on June 11 that it presented the preclinical research results and global development strategy for its rare neurodevelopmental disorder treatment candidate DDC-02 at the World Orphan Drug Congress USA 2026, held in Boston from June 9 to 11.
DDC-02 is an oral small molecule drug candidate discovered using JW Pharmaceutical's proprietary AI-based drug development platform, JWave. The platform facilitated genomic analysis, leading to a molecular understanding of the disease and the elucidation of its therapeutic mechanisms.
During the presentation, DDC-02 demonstrated the ability to restore cognitive and behavioral functions in animal models of various rare neurodevelopmental disorders, including Pitt-Hopkins syndrome (PTHS), Fragile X syndrome (FXS), and Rett syndrome (RTT), which are caused by different genetic factors.
The research findings indicated that DDC-02 effectively improved cognitive and behavioral functions across multiple preclinical animal models of neurodevelopmental disorders, including PTHS, FXS, and RTT.
Notably, even in adult animal models with advanced symptoms, cognitive and behavioral functions were restored to normal levels. This suggests the potential for functional improvement in neurodevelopmental disorders, which were previously thought to have limited recovery prospects.
JW Pharmaceutical is considering Pitt-Hopkins syndrome as the first clinical indication for DDC-02 and aims to initiate global multi-national clinical trials by 2028. The company plans to expand indications to various rare neurodevelopmental disorders thereafter.
A representative from JW Pharmaceutical stated, "DDC-02 has shown consistent efficacy across models of neurodevelopmental disorders with different genetic causes. We plan to develop it into a new treatment option for patients with rare neurodevelopmental disorders through global clinical development and strategic partnerships."
* This article has been translated by AI.
Copyright ⓒ Aju Press All rights reserved.