The FDA grants ES-3000 Orphan Drug Designation for AML

On 28^th December 2016, the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation for ES-3000 for the treatment of Acute Myeloid Leukemia (AML).

The main issues associated with therapy for AML are insensitivity to induction chemotherapy and relapse after remission which often arises as a result of persistent Leukemic Stem Cells (LSCs). LSCs are a small population of stem cells that have properties of differentiation, self-renewal and homeostatic control and they contribute to the maintenance and propagation of cancer cells (Pollyea et al., 2014).

AML depends on LSCs for continued growth and propagation and its presence leads to disease resistance, relapse and often death in AML patients (Pollyea et al., 2014). The fact that LSCs contribute to the subsequent relapse and disease resistance in AML patients has made these group of cells, important targets for AML therapy.

Wnt/ β-catenin plays a critical role in differentiation, cell growth, proliferation and developmental processes. In cancers, Wnt/ β-catenin pathway is aberrantly activated thus a therapeutic target. Additionally, it has been reported that activation of Wnt/ β-catenin signalling pathway can lead to the development of LSCs from either Hematopoietic Stem Cells (HSCs) or Granulocyte Macrophage Progenitors (GMPs) in AML (Ashihara et al., 2015), which thus makes this pathway a potential therapeutic target for this disease.

ES-3000, targets the Wnt/ β-catenin signalling pathway this hindering the development of LSCs. ES-3000 along with standard therapy would be administered with an aim to reduce the LSCs reservoir in AML which may help to provide durable remissions, reduce subsequent relapse and disease resistance in AML patients.   

ES-3000 is currently in the pre-clinical phase for AML treatment and is manufactured by Escend Pharmaceuticals