JW Pharmaceutical (KRX:001060) 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 (WODC USA 2026) in Boston, Massachusetts, from June 9 to 11.
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JW Pharmaceutical presents DDC-02 during an Oral Presentation Session (Image: JW Pharmaceutical)
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 behavioral function.
DDC-02 is an orally administered small-molecule candidate discovered by JW Pharmaceutical. The compound has been shown to modulate intracellular signaling pathways involved in neurodevelopment and neural circuit function. In preclinical studies, DDC-02 restored impaired cognitive and behavioral 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 behavioral 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 behavior, despite their distinct genetic origins.
Noteworthy was the observation that cognitive and behavioral 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.
Dr. 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,” adding “The observation that cognitive and behavioral 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.”
He added, “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.”
About Pitt-Hopkins Syndrome (PTHS)
PTHS is an ultra-rare neurodevelopmental disorder caused by loss-of-function mutations in the TCF4 (Transcription Factor 4) gene. The condition is characterized by severe intellectual disability, profound speech impairment, autism-like features, breathing abnormalities, and epilepsy. Its prevalence is estimated at approximately one in 30,000-40,000 individuals. There are currently no approved disease-modifying therapies, and treatment is primarily focused on symptom management and supportive care.
About Fragile X Syndrome (FXS)
FXS is an inherited neurodevelopmental disorder caused by mutations in the FMR1 (Fragile X Messenger Ribonucleoprotein 1) gene and is the most common inherited cause of intellectual disability. Clinical manifestations include learning disabilities, cognitive impairment, autism spectrum-related features, anxiety, and hyperactivity. The disorder affects approximately one in 4,000 males and one in 8,000 females. No disease-modifying therapies have been approved to date. The global FXS treatment market was estimated at approximately USD 1.2–2.6 billion in 2024, with continued growth anticipated as multiple investigational therapies advance through clinical development.
About Rett Syndrome (RTT)
Rett syndrome is a rare neurodevelopmental disorder primarily caused by mutations in the MECP2 (Methyl-CpG Binding Protein 2) gene and predominantly affects females. Patients typically experience apparently normal early development followed by progressive loss of language and motor skills, repetitive hand movements, gait abnormalities, and seizures. The disorder affects approximately one in 10,000–15,000 individuals worldwide. In 2023, the U.S. Food and Drug Administration approved trofinetide (Daybue®), the first therapy specifically indicated for Rett syndrome. Annual treatment costs are estimated at approximately USD 375,000 per patient, underscoring the significant unmet medical need and the substantial value attributed to effective therapies in this field.
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