Advanced soft-tissue sarcomas are rare and often lethal cancers.

Soft-tissue sarcomas represent a rare and heterogenous group of mesenchymal tumors, accounting for 1% of all adult cancers and more than 7% of all pediatric cancers. Approximately 0.4% of people will be diagnosed with a soft-tissue sarcoma during their lifetime. Localized soft-tissue sarcomas are treated with surgery, with or without post-operative radiotherapy, with a high success rate, but few (if any) efficacious therapies are available for patients with metastatic tumors; 25-30% of these people die from cancer within 5 years of diagnosis.

GIST is an exception among soft-tissue sarcomas, as there are precision medicines available to treat it. A genetic mutation activates either KIT or platelet-derived growth factor alpha receptors abnormally, which enables the continuous growth and division of tumor cells. About 40% of patients, including those who have metastatic disease or tumors with a high risk of recurrence, are treated with KIT and platelet-derived growth factor receptor alpha-targeting small molecule compounds such as Glivec®. These therapeutics are effective, but eventually most GISTs become resistant to therapy, and novel approaches to treat GISTs are needed.

There is a clear unmet clinical need to identify novel therapeutic options for soft-tissue sarcoma patients. Our first precision medicine product to treat these tumors, SAR001, specifically targets the PDE3A protein, which is frequently expressed in GIST and other soft-tissue tumors.