JW Pharmaceutical (001060.KS) has released preclinical research results for 'DDC-02', a drug candidate for rare neurodevelopmental disorders. The company confirmed that cognitive and behavioral functions recovered to normal levels even in adult animal models with sufficiently advanced symptoms, suggesting the potential to expand its indications to various rare neurodevelopmental disorders.
JW Pharmaceutical announced Wednesday that it presented preclinical research achievements and a global development strategy for DDC-02, a drug candidate for rare neurodevelopmental disorders, at the 'World Orphan Drug Congress USA 2026 (WODC USA 2026)', the world's largest conference specializing in orphan drugs, held in Boston from Monday through Wednesday.
DDC-02 is an oral small-molecule drug candidate discovered in-house by JW Pharmaceutical. It is being developed as a mechanism that regulates intracellular signaling pathways involved in neurodevelopment and neural circuit function.
The presentation introduced results showing that DDC-02 commonly restored cognitive and behavioral functions in animal models of rare neurodevelopmental disorders arising from different genetic causes, including Pitt-Hopkins syndrome, Fragile X syndrome, and Rett syndrome.
The research found that DDC-02 restored impaired cognitive and behavioral functions to the level of normal animals across various neurodevelopmental disorder animal models. This result demonstrates the potential for application to multiple rare neurodevelopmental disorders that share the common pathophysiology of cognitive and behavioral function impairment, rather than being limited to specific genetic abnormalities.
Notably, meaningful recovery effects were confirmed even in adult animal models in which symptoms had already sufficiently progressed. Neurodevelopmental disorders have been thought to offer limited functional recovery once symptoms become entrenched, due to neural circuit abnormalities formed in early development. However, DDC-02 showed results suggesting the possibility of functional reconstruction and recovery even in mature neural circuits.
JW Pharmaceutical is reviewing Pitt-Hopkins syndrome, an ultra-rare disease, as the first clinical indication for DDC-02, and is pursuing development with the goal of entering global multinational clinical trials in 2028. It plans to subsequently expand indications to include Fragile X syndrome and Rett syndrome.
During the conference, JW Pharmaceutical also explored the possibility of global partnering, discussing various cooperation measures including joint development and technology transfer through business development meetings with global pharmaceutical and bio companies and investment institutions.
"DDC-02 is a candidate that has confirmed consistent efficacy in neurodevelopmental disorder models arising from different genetic causes," a JW Pharmaceutical official said. "We plan to develop it into a new treatment option for patients with rare neurodevelopmental disorders through future global clinical development and strategic partnerships."
