JW Pharmaceutical to Present Preclinical Results of Novel Drug Candidate for Rare Pediatric Brain Diseases at World Orphan Drug Congress

JW Pharmaceutical announced on May 14 that it will participate in the '2026 World Orphan Drug Congress USA,' which will be held in Boston, USA, from June 9 to 11, to present the preclinical research results of its novel drug candidate for rare pediatric brain diseases, 'DDC-02 (provisional code name)'.

DDC-02 is a first-in-class oral small-molecule compound being developed as a treatment for rare genetic pediatric brain diseases, including Pitt-Hopkins syndrome.

A view of the JW Pharmaceutical building in Gwacheon, Gyeonggi Province. JW Pharmaceutical

Focusing on the reduced Wnt signaling observed in these diseases, JW Pharmaceutical has been conducting research on small-molecule new drug substances that can normalize the deficient signaling pathway.

The DDC-02 candidate to be presented this time was derived using JW Pharmaceutical's proprietary AI-based drug development platform, 'JWave.' In particular, genomics analysis through this platform enabled a deeper molecular understanding of the diseases and the mechanism of action, enhancing both research efficiency and precision.

Currently, DDC-02 is undergoing various safety evaluations. JW Pharmaceutical plans to use its presentation at the congress to share research achievements and development potential of this candidate with the global market and to seek diverse opportunities for collaboration.

A JW Pharmaceutical representative stated, "This presentation at the congress is a meaningful opportunity to showcase JW's innovative drug candidate for rare pediatric brain diseases on the global stage. Through in-depth exchanges with experts in the field of rare diseases, we aim to expand opportunities for technological alliances and other collaborations, and to accelerate the development of new drugs that can improve patients' quality of life."

Meanwhile, the World Orphan Drug Congress USA is a global event dedicated to the development of treatments for rare diseases. It brings together major pharmaceutical and biotechnology companies, as well as government and regulatory agencies, patient organizations, and investors—key stakeholders in the rare disease drug development ecosystem—to share the latest trends and innovative case studies.

This content was produced with the assistance of AI translation services.