A novel precision treatment for hard-to-treat cancers
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. Localized tumors can be treated with surgery and radiotherapy, but few, if any efficacious therapies are available for patients with metastatic tumors. In advanced disease the prognosis is poor, the 5-year survival for metastatic soft tissue sarcomas being 10 – 50 %, depending on tumour type.
GIST is an exception among soft-tissue sarcomas having targeted therapy options available. About 40 % of patients are treated with KIT and PDGFRA therapies such as imatinib. However, eventually most GISTs become resistant to therapy, and novel approaches to treat GISTs are needed.
We give new life and purpose to anagrelide with targeted sarcoma therapy
Our SAR003 is a new precision cancer treatment for soft tissue sarcomas, based on a scientific discovery of phosphodiesterase 3A (PDE3A) as a novel therapy target. PDE3A is sparsely expressed in normal tissues but highly expressed in certain soft tissue sarcomas making it a highly promising target for therapeutic use. The mode-of-action makes all GIST patients treatable with SAR003 including the youngest Wild-Type GIST patients.
Pipeline extensions
We are developing our lead compound SAR003 for the treatment of all GIST patients with metastatic disease. The mode-of-action is also suitable for the treatment of other soft tissue sarcoma subtypes and hard to treat tumors expressing PDE3A.