Acute Myeloid Leukemia Treatment Gets Orphan Drug Designation

Escend Pharmaceuticals, Inc. receives second orphan drug designation from FDA for ES-3000.

Escend Pharmaceuticals, Inc., a privately held company focused on the development of small molecule therapeutics for oncology orphan diseases, announced that the FDA Office of Orphan Products Development (OOPD) has granted orphan drug designation for its lead product candidate, ES-3000, for the treatment of acute myeloid leukemia (AML). The FDA previously granted ES-3000 orphan designation for the treatment of chronic myeloid leukemia (CML).

“This accomplishment validates the potential significance ES-3000 can have in shifting the AML treatment paradigm,” said Saira Bates, co-founder and CEO of Escend. “The treatment of patients with AML is complicated by relapse and refractory disease which is often attributed to the resistance of leukemic stem cells to standard therapeutics. ES-3000 will be used in conjunction with standard agents to target leukemic stem cells for more durable remissions.”

The FDA orphan drug designation provides seven years of marketing exclusivity and certain incentives, including federal grants, tax credits, and waived FDA fees. Successive drug approvals for ES-3000 in the treatment of CML and AML give Escend the possibility of up to 14 years of drug exclusivity.

About ES-3000 and AML

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

Acute myeloid leukemia (AML) is the most common acute leukemia in adults with the lowest survival rate. In the U.S., about 20,000 people each year are diagnosed with AML. Leukemic stem cells are dependent on an over expression of β-catenin, which is the target for ES-3000. Escend’s initial objective is to develop ES-3000 for use with current standard of care in the treatment of AML to provide more durable remissions and reduce relapses.

About Escend

Escend’s strategy is to select drug candidates with established clinical safety profiles that have not yet achieved U.S. marketing approval and match them to oncology orphan indications, where their effects on specific cellular pathways can be leveraged for the development of novel therapeutics. https://www.escendpharma.com.

(Source: PR Newswire)