The FDA granted Orphan Drug Designation to a novel menin-mixed lineage leukemia (menin-MLL) inhibitor KO-539 for the treatment of acute myeloid leukemia (AML).
KO-539 is a selective small molecule inhibitor of the menin-MLL protein-protein interaction. MLL-rearranged leukemias are characterized by chromosomal translocations of the MLL gene that are primarily found in patients with AML and acute lymphoblastic leukemia (ALL). These translocations form oncogenes encoding MLL fusion proteins, which play a causative role in the onset, development and progression of MLL-rearranged leukemias.
In a company press release, Troy Wilson, PhD President and Chief Executive Officer of Kura Oncology said, “Orphan Drug Designation for AML represents a significant milestone in the development of KO-539. This decision by the FDA follows the clearance of our investigational new drug (IND) application in March 2019 and recognizes the potential for KO-539 to address a high unmet need for patients suffering from AML. We are very encouraged by our preclinical data in genetically defined subsets, such as tumors with MLL fusions and rearrangements and NPM1 mutations, and we are in the final stages of study startup for our Phase 1 clinical trial in relapsed or refractory AML.”
The FDA’s Orphan Drug Designation program provides drug companies incentives to develop treatments for rare diseases. The FDA Office of Orphan Products Development determines if a drug qualifies as an orphan product and provides orphan status to drugs defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases that affect fewer than 200,000 people in the United States.