SEED Therapeutics Receives China NMPA Clearance for ST-01156 Molecular Glue Degrader in Rare Cancer Trial
Key Insights
SEED Therapeutics (search) received IND clearance from China's NMPA for ST-01156, enabling inclusion of Chinese clinical sites in the company's planned global expansion study following completion of the ongoing first-in-human dose-escalation trial.
ST-01156 is a molecular glue degrader targeting RBM39 (search) that achieved complete tumor regression in preclinical xenograft models of Ewing sarcoma (search), neuroblastoma (search), and KRAS-mutant colon cancer (search).
The drug has received FDA Orphan Drug and Rare Pediatric Disease designations for Ewing sarcoma (search), a rare pediatric malignancy with no new approved therapies in over 30 years.
SEED Therapeutics (search) announced that China's National Medical Products Administration (NMPA) has cleared the Investigational New Drug (IND) application for ST-01156, the company's lead molecular glue degrader targeting historically undruggable proteins. This regulatory milestone enables the inclusion of Chinese clinical sites in SEED's planned global expansion study, which will follow completion of the ongoing first-in-human dose-escalation trial in patients with advanced solid tumors.
Novel Molecular Glue Platform Targets Undruggable Proteins
ST-01156 represents the first clinical candidate from SEED's proprietary RITE3™ platform, which rationally identifies optimal E3 ligases to target disease-causing proteins and enables discovery of drug-like molecular glues for multiple indications. The compound induces selective degradation of RBM39 (search), a key regulator of RNA splicing and transcription that is overexpressed and required for survival in a broad range of cancers.
The drug has received both Orphan Drug and Rare Pediatric Disease designations from the U.S. FDA for Ewing sarcoma (search), a rare pediatric malignancy with no new approved therapies in over 30 years.
Compelling Preclinical Efficacy Data
In preclinical models, ST-01156 demonstrated remarkable therapeutic potential, achieving complete tumor regression in xenografts of Ewing sarcoma (search), neuroblastoma (search), and KRAS-mutant colon cancer (search). The compound also showed strong target engagement across these models, providing compelling rationale for clinical evaluation.
"Our preclinical data show complete tumor regression in multiple xenograft models and strong target engagement, providing a compelling rationale to evaluate ST-01156 across RBM39 (search)-dependent tumors such as Ewing sarcoma (search), hepatocellular carcinoma (search), and KRAS-mutant cancers (search)," said Dr. James Tonra, President and Chief Scientific Officer of SEED.
Patient-derived models have identified additional RBM39 (search)-dependent tumors that will help inform enrollment in the expansion study.
Global Development Strategy Advances
The NMPA clearance represents a significant step in SEED's global development strategy for ST-01156. Dr. Lan Huang, Co-Founder, Chairman, and CEO of SEED, emphasized the strategic importance of this milestone: "The NMPA's clearance of our IND for ST-01156 marks an important step in SEED's global development strategy. With our dose-escalation study now underway, we are laying the groundwork for an expansion study focused on RBM39 (search) mechanism-targeted cancers."
The upcoming expansion study will include clinical sites across the United States, Europe, China, and other regions, broadening access to diverse patient populations and accelerating understanding of ST-01156's therapeutic potential across RBM39 (search)-dependent tumors.
Distinguished Scientific Leadership and Industry Support
SEED Therapeutics (search) was co-founded by four prominent scientific leaders, including Nobel Laureate Prof. Avram Hershko, discoverer of the ubiquitin-proteasome system, and Dr. Lan Huang, who solved the first E3 Ligase (search) structure. The founding team also includes Prof. Ning Zheng from the University of Washington, who pioneered RING-finger E3 Ligase structures and coined the term "molecular glue," and Prof. Michele Pagano from NYU Grossman School of Medicine, a preeminent expert on E3 ligase biology.
The company has secured support from major pharmaceutical companies, with Eli Lilly and Eisai serving as cornerstone investors and collaborators. SEED's broader pipeline includes nine molecular glue programs spanning oncology, neurodegeneration, immunology, and virology, supporting the company's mission to unlock undruggable disease targets through targeted protein degradation with small-molecule precision.