JW Pharmaceutical said on the 11th that preclinical studies of DDC-02, a candidate therapy for rare neurodevelopmental disorders, showed the potential to restore cognitive and behavioral functions.
The company presented these findings at the World Orphan Drug Congress (WODC USA 2026), held in Boston from the 9th to the 11th (local time).
According to the presentation, DDC-02 commonly restored cognitive and behavioral functions in animal models of rare neurodevelopmental disorders arising from different genetic causes, including Pitt-Hopkins syndrome (PTHS), fragile X syndrome (FXS), and Rett syndrome (RTT).
DDC-02 is an oral small-molecule drug candidate discovered in-house by JW Pharmaceutical. It is being developed to modulate intracellular signaling pathways involved in neurodevelopment and neural circuit function.
In preclinical studies, DDC-02 restored impaired cognitive and behavioral functions in various neurodevelopmental disorder animal models to levels seen in normal animals. The company said this confirmed that DDC-02 may improve cognitive and behavioral functions by affecting synaptic plasticity and neural circuit function.
Notably, it emphasized that the data suggest potential application across several rare neurodevelopmental disorders that share the common pathophysiology of cognitive and behavioral dysfunction, rather than being limited to the genetic cause of a specific disease.
A noteworthy point is that a return to normal levels of cognitive and behavioral functions was also observed in adult animal models in which symptoms had progressed substantially.
Because neurodevelopmental disorders arise from abnormalities in neural circuits formed early in development, it is known to be difficult to restore functions once symptoms become fixed. However, this study showed meaningful improvement even in adult models, suggesting that functional reorganization and recovery may be possible in mature neural circuits.
JW Pharmaceutical is reviewing Pitt-Hopkins syndrome as the first clinical indication for DDC-02 and is developing the program with a goal of entering a global multi-country clinical trial in 2028. It then plans to expand target diseases to conditions such as fragile X syndrome and Rett syndrome.
During the conference, the company held business development (BD) meetings with global pharmaceutical and biotech corporations and investment institutions and discussed various forms of collaboration, including co-development and technology transfer.
A JW Pharmaceutical official said, "DDC-02 is a candidate that has shown consistent efficacy in neurodevelopmental disorder models caused by different genetic factors," adding, "It is meaningful that we observed a return to normal levels of cognitive and behavioral functions even in adult models."
The official added, "We will accelerate development to provide a new treatment option to patients with rare neurodevelopmental disorders through global clinical development and strategic partnerships."