PROTAC molecules targeting PAX3:FOXO1 fusion protein for rhabdomyosarcoma treatment

BACKGROUND

Fusion-positive rhabdomyosarcoma (FP-RMS) is an aggressive childhood cancer characterized by the PAX3::FOXO1 fusion protein, which drives tumor growth and progression. This subtype accounts for about 80% of alveolar rhabdomyosarcoma cases and is associated with poor prognosis. Current treatments, including chemotherapy, radiation, and surgery, lack specificity for the PAX3::FOXO1 oncoprotein, resulting in limited efficacy and significant side effects. The development of targeted therapies that can specifically degrade PAX3::FOXO1 represents a promising strategy to improve treatment efficacy and patient survival in FP-RMS. Previous attempts to inhibit PAX3::FOXO1 have shown promise, but direct degradation of the protein using PROTAC technology offers a novel and potentially more effective approach.

Benefit

• First PROTAC molecule targeting PAX3::FOXO1, offering a novel approach to treat FP-RMS.
• Potentially more specific and effective than current treatments for FP-RMS.
• May reduce side effects compared to traditional chemotherapy by targeting cancer-specific proteins.
• Could improve survival rates for patients with FP-RMS.

Market Application

• Potential use in targeted therapy development by pharmaceutical companies specializing in oncology and rare pediatric cancers.
• Advanced application of PROTAC technology by biotechnology firms focusing on protein degradation platforms for cancer treatment.
• Possible integration into clinical trials conducted by research institutions and academic medical centers studying rhabdomyosarcoma and other pediatric sarcomas.
• Enhanced incorporation in preclinical and clinical development processes by Contract Research Organizations (CROs) specializing in cancer therapeutics.
• Promising application in companion diagnostic development by companies creating tests to identify patients with PAX3::FOXO1 fusion-positive rhabdomyosarcoma.

Publications

US Provisional Application Filed