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A Wicked challenge needs novel solutions

CHALLENGE

Advanced soft-tissue sarcomas are rare and often lethal 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, and treatment options are limited. McKinsey lists soft tissue sarcomas and GIST as commercially attractive cancers presenting significant remaining unmet need.

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 SAR003 specifically targets PDE3A protein which is frequently expressed in GIST and other soft-tissue tumors. 

Solution

We give new life and purpose to anagrelide with targeted sarcoma therapy

SAR003 is a new precision cancer treatment for soft tissue sarcomas, based on a scientific discovery of phosphodiesterase 3 (PDE3) as a novel therapy target. PDE3 is sparsely expressed in normal tissues but highly expressed in certain soft tissue sarcomas making it a suitable target for therapeutic use. PDE3 can be used as a biomarker to select right patients for the targeted therapy.