NEED

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 tumors can be treated with surgery and radiotherapy, but few, if any efficacious therapies are available for patients with metastatic tumors; 25-30 % die within 5 years of diagnosis.

GIST is an exception among soft-tissue sarcomas having targeted therapy options available. A genetic mutation in either KIT or platelet-derived growth factor alpha (PDGF-alpha) receptor allows their abnormal activation leading to continuous growth and division of tumor cells. About 40 % of patients are treated with KIT and PDGFR-alpha targeting therapies such as imatinib. Eventually most GISTs become resistant to therapy, and novel approaches to treat GISTs are needed.

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