Kura Oncology Announces FDA Clearance of Investigational New Drug Application for Menin-MLL Inhibitor KO-539

Kura Oncology (NASDAQ:KURA) has announced that the US Food and Drug Administration (FDA) has cleared the company’s investigational new drug (IND) application for KO-539, a small molecule inhibitor of the menin-mixed lineage leukemia (menin-MLL) protein-protein interaction.

As quoted in the press release:

“Despite recent advances in the treatment of AML, genetically defined subsets of acute leukemias such as MLL-rearrangements and NPM1 mutations remain areas of high unmet need,” said Troy Wilson, Ph.D., J.D., President and Chief Executive Officer of Kura Oncology. “Our data suggest that KO-539 drives robust and persistent responses in these preclinical models by induction of differentiation in AML blasts, a mechanism that is distinct from and potentially complementary to existing therapies. We are very encouraged by these preliminary results, and we look forward to beginning clinical testing of KO-539 next quarter.”

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), the most common type of cancer in children. These translocations form oncogenes encoding MLL fusion proteins, which play a causative role in the onset, development and progression of MLL-rearranged leukemias.

The target genes of the MLL fusion proteins are also found to be overexpressed in a broader subset of AMLs characterized by oncogenic driver mutations in genes such as NPM1. These mutations also appear to be dependent on the interaction between menin and MLL, suggesting that the menin-MLL complex is a central node in epigenetic dysregulation driven by distinct oncogenic driver mutations known to be important in AML and other hematologic malignancies.

In preclinical studies, KO-539 has demonstrated potent and selective inhibition of the proliferation of MLL-rearranged leukemia cell lines. Kura has also generated preclinical data showing robust and durable efficacy in multiple in vivo models of AML characterized by MLL-rearrangements or oncogenic driver mutations in genes such as NPM1.

Click here to read the press release.