JW Pharmaceutical said on the 14th that nonclinical study results for DDC-02, a new drug candidate for rare pediatric brain disorders, confirmed the potential to restore impaired signal transduction function.
The company plans to unveil these research results at the World Orphan Drug Congress USA 2026, to be held in Boston, the United States, from June 9–11 (local time).
DDC-02 is a first-in-class, orally administered small-molecule compound targeting rare, hereditary pediatric brain disorders such as Pitt-Hopkins syndrome. JW Pharmaceutical, focusing on the commonly observed impairment of Wnt signaling in these diseases, has been researching a new drug candidate that restores this function to normal levels.
The candidate was discovered using JW Pharmaceutical's artificial intelligence (AI)-based integrated drug discovery platform JWave. The company said it enhanced research efficiency and precision by identifying the diseases' molecular mechanisms and the drug's mechanism of action through genomics-based research.
DDC-02 is currently undergoing various safety assessments. JW Pharmaceutical plans to use this conference presentation to highlight the candidate's development potential in the global rare disease market and expand collaboration opportunities such as technology transfer and joint development.
A JW Pharmaceutical official said, "This presentation introduces JW's innovative new drug candidate in the field of rare pediatric brain disorders on the global stage," adding, "Through exchanges with rare disease experts, we will broaden opportunities for technology partnerships and joint research, and accelerate the development of new drugs that can help improve patients' quality of life."