SEED Therapeutics' ST-01156 Receives FDA Rare Pediatric Disease and Orphan Drug Designations

Key Insights

• SEED Therapeutics' ST-01156, a novel molecular glue targeting RBM39, has been granted Rare Pediatric Disease and Orphan Drug designations by the FDA.
• ST-01156 degrades RBM39, an RNA splicing factor relevant in multiple solid tumor indications, and is being advanced towards an IND filing expected in H1 2025.
• The Rare Pediatric Disease designation provides SEED Therapeutics the potential to receive an FDA priority review voucher upon ST-01156 approval.

SEED Therapeutics has announced that the U.S. Food and Drug Administration (FDA) has granted both Rare Pediatric Disease and Orphan Drug designations to its investigational molecular glue, ST-01156. This agent is designed to degrade RBM39, an RNA splicing factor implicated in various solid tumor indications.

Mechanism of Action and Clinical Development

ST-01156 represents a novel approach to cancer therapy by targeting RBM39, a protein crucial for RNA splicing. Aberrant RNA splicing is a hallmark of many cancers, making RBM39 a compelling therapeutic target. By selectively degrading RBM39, ST-01156 aims to disrupt cancer cell growth and survival.

SEED Therapeutics is currently advancing ST-01156 toward an Investigational New Drug (IND) application, with the filing anticipated in the first half of 2025. This timeline reflects the company's commitment to rapidly advancing this promising therapeutic into clinical trials.

Regulatory Designations and Potential Benefits

The FDA's Rare Pediatric Disease designation is granted for drugs intended to treat rare diseases that primarily affect children. If ST-01156 receives FDA approval, SEED Therapeutics may be eligible to receive a priority review voucher, which can be used to expedite the review of a subsequent marketing application.

The Orphan Drug designation provides incentives for companies developing therapies for rare diseases, including market exclusivity and tax credits. These designations underscore the FDA's recognition of the unmet medical need in pediatric cancers and the potential of ST-01156 to address this need.

Corporate Developments

In addition to the regulatory news, SEED Therapeutics also announced that BeyondSpring (BYSI), its largest shareholder, has entered into definitive agreements to sell a portion of its Series A-1 Preferred Shares of SEED. Following the completion of these transactions, BeyondSpring is expected to retain approximately 14.4% of SEED’s outstanding shares. This corporate restructuring does not impact the development of ST-01156.