Skip to Main Content

NEW ORLEANS — An emerging class of genetically targeted drugs is inducing remissions in about one-third of patients with advanced leukemia, according to updates Saturday from separate clinical trials.

Two biotechs — Syndax Pharmaceuticals and Kura Oncology — are developing their own versions of so-called menin inhibitors that have the potential to become effective treatments for certain types of genetically defined acute myeloid leukemia (AML), a rapidly progressing bone marrow cancer that affects adults.


These drugs target a genetic “rearrangement” called KMT2A and a mutation affecting a protein called NPM1 — both of which make AML harder to treat with currently approved medicines. The drugs work by blocking the interaction of two proteins that prevents bone marrow cells from developing or differentiating into healthy cells.

Unlock this article by subscribing to STAT+ and enjoy your first 30 days free!


Create a display name to comment

This name will appear with your comment

There was an error saving your display name. Please check and try again.