Escend Pharmaceuticals, Inc. Receives Orphan Drug Designation from the US Food and Drug Administration (FDA) for ES-3000, its Lead Compound, in the Treatment of Chronic Myeloid Leukemia (CML)

Menlo Park, CA, June 2, 2016/PRNewswire/ -- Escend Pharmaceuticals, Inc., a privately held pharmaceutical company focused on the development and commercialization of small molecule drug candidates for oncology orphan diseases, announced today that its lead compound, ES-3000, has received orphan drug designation from the U.S. Food and Drug Administration (FDA) for the treatment of chronic myeloid leukemia (CML).

"We are extremely pleased with the grant of orphan drug designation for our lead compound in CML. This is an important step in gaining commercial interests for a first-in-class agent that can potentially shift the current treatment paradigm for CML patients,” said Saira Bates, Co-founder & CEO of Escend. “Due to the resistance of leukemic stem cells to tyrosine kinase inhibitors (TKIs), minimal residual disease (MRD) in CML patients is an unmet need which will be addressed with ES-3000."

The FDA orphan drug designation provides 7 years of marketing exclusivity and certain incentives, including federal grants, tax credits, and waived FDA fees.

About ES-3000 

ES-3000 is an orally bioavailable small molecule which ablates leukemic stem cells by reducing β-catenin expression through a novel mechanism of action. Wnt/β-catenin pathway is critical for the survival of cancer stem cells.  ES-3000 is also in development for the treatment of acute myeloid leukemia (AML) and triple negative breast cancer (TNBC). 

About CML

In 2016, approximately 8,220 new cases of CML are expected to be diagnosed in the United States. Although CML is responsive to TKIs, such as imatinib, MRD remains a significant problem limiting long term control of disease, mostly due to the persistence of leukemic stem cells.  In addition, up to 33% of the TKI treated patients will not attain optimal responses, calling for the development of additional therapies addressing minimal residual disease. Currently, there are no FDA approved anti-leukemic stem cell therapies.

About Escend Pharmaceuticals, Inc. 

Escend Pharmaceuticals’ mission is to develop therapeutics for oncology orphan indications by ablating cancer stem cells. Our business strategy is to select small molecule drug candidates with established clinical safety that have not yet achieved US marketing approval and match them to appropriate oncology indications, where their effects on specific cellular pathways can be exploited for the development of novel therapeutics. For further information, please visit