JW Pharmaceutical Confirms Cognitive Function Restoration with Rare Neurodevelopmental Disorder Drug Candidate

Preclinical Research Results Presented at WODC USA

Improvements in Cognitive and Behavioral Functions Observed in Models Including Pitt-Hopkins Syndrome

Aiming to Enter Global Clinical Trials by 2028

JW Pharmaceutical has released its preclinical research results for a candidate treatment for rare neurodevelopmental disorders. The company plans to expand the indications to various rare neurodevelopmental disorders, aiming to enter global multinational clinical trials by 2028.

JW Pharmaceutical announced on the 11th that it presented the preclinical research results and global development strategy for its candidate treatment for rare neurodevelopmental disorders, 'DDC-02,' at the 'World Orphan Drug Congress USA 2026 (WODC USA 2026),' the world's largest conference specializing in rare drugs, held in Boston, USA, from June 9 (local time).

A representative from JW Pharmaceutical is introducing the mechanism of action and key preclinical study results of 'DDC-02' through an oral session at the World Orphan Drug Congress USA 2026 (WODC USA 2026), a conference specializing in orphan drugs held in Boston, USA, from the 9th to the 11th (local time). JW Pharmaceutical

DDC-02, discovered in-house by JW Pharmaceutical, is an oral small molecule new drug candidate that regulates intracellular signaling pathways involved in neurodevelopment and neural circuit function.

According to research results, DDC-02 restored impaired cognitive and behavioral functions to normal animal levels in preclinical animal models of various neurodevelopmental disorders, including Pitt-Hopkins Syndrome (PTHS), Fragile X Syndrome (FXS), and Rett Syndrome (RTT).

JW Pharmaceutical explained, "DDC-02 suggests that it can improve cognitive and behavioral functions by affecting synaptic plasticity and neural circuit function. This indicates the potential for application to multiple rare neurodevelopmental disorders that share the common pathology of cognitive and behavioral dysfunction, regardless of the specific genetic causes of each disorder."

In this study, the restoration of cognitive and behavioral functions to normal levels was also observed in adult animal models with sufficiently progressed symptoms. The company confirmed that this demonstrates functional reorganization and recovery are possible even in mature neural circuits.

JW Pharmaceutical is considering Pitt-Hopkins Syndrome as the first clinical indication for DDC-02 and is pursuing development with the goal of entering global multinational clinical trials by 2028. Afterward, the company plans to expand indications to various rare neurodevelopmental disorders, including Fragile X Syndrome and Rett Syndrome.

A company representative stated, "DDC-02 is a promising candidate that has demonstrated consistent efficacy in neurodevelopmental disorder models caused by different genetic factors. In particular, it is meaningful that we observed restoration of cognitive and behavioral functions to normal levels even in adult models. We plan to develop DDC-02 as a new treatment option for patients with rare neurodevelopmental disorders through future global clinical development and strategic partnerships."

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