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Opportunity to License or Acquire ES-3000, An Orphan Drug for CML and AML

Escend Therapeutics ( is based in San Diego, CA, and develops small molecule therapeutics for oncology orphan drug indications. ES-3000 is an orally bioavailable small molecule (FW ~600 Da) with a unique mechanism of action and established clinical safety. ES-3000 ablates leukemic stem cells as it reduces β-catenin by inhibition of CaMKIIγ activity. Loss of b-catenin impairs the renewal of both CML and AML stem cells.

Sage represents Escend in its efforts to develop a strategic alliance for the further development of its ES-3000 oncology drug. We are pleased to attach an Executive Summary and non-confidential Information Memorandum outlining the partnering opportunity with Escend and ES-3000.

Escend’s lead program is focused on chronic myeloid leukemia (CML) in blast crisis or with suboptimal responses to the standard of care, which is about a third of 87,000 CML patients living with the disease in the US. There will be an estimated 120,000 patients by 2022, when market approval is expected. Given a 30% market penetration and an average cost of $100,000/yr/patient for drugs in this indication, ES-3000 could have upon approval potential annual revenue of $1.2 billion.

Six US provisional patent applications have been filed and freedom-to-operate analysis conducted for ES-3000. Escend’s IP focuses on methods of use and compositions of matter for ES-3000 or analogs for the treatment of leukemic stem cells, cancer stem cells and precancerous stem cells; and methods of use for ES-3000 and other β-catenin inhibitors as immune checkpoint inhibitor conditioning agents.

Orphan drug designation has been received from the FDA for ES-3000 in the treatment of CML. ES-3000 may also have efficacy in treatment of AML.

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