What is the target of Aceminib leukemia targeting drug?

Aximini, an innovative leukemia-targeted drug, its key target isABL1 fusion protein, especially the abnormal protein produced by BCR-ABL1 gene mutations that are closely related to chronic myelogenous leukemia (CML). This drug, also known as Asciminib or Scemblix, has attracted widespread attention in the medical community, mainly due to its unique mechanism of action and significant clinical effects.

Aximinib’s mode of action is different from traditional tyrosine kinase inhibitors (TKIs). It specifically targets the ABL myristoyl pocket (STAMP), an allosteric site that effectively inhibits the tyrosine kinase activity of BCR-ABL1. Traditional TKIs usually work by binding to the ATP-binding site of the BCR-ABL1 protein, while aceminib provides a new inhibitory mechanism by acting on another part of the kinase, the ABL myristoyl pocket.

In clinical trials, Aceminib showed significant effects onBCR-ABL1 inhibition. For example, in one study, patients with chronic-phase Philadelphia chromosome-positive chronic myelogenous leukemia (Ph+ CML-CP) who had previously received at least two TKIs had significantly improved major molecular response rates (MMR) and complete cytogenetic response rates (CCyR) after receiving aciminib. Specifically, at week 24, the MMR rate in the aximinib group reached 25%, which was significantly higher than the 13% in the control group; and at week 96, this rate increased to 38%. These data fully demonstrate the superior efficacy of Aceminib in targeting the BCR-ABL1 fusion protein.

In addition, Aceminib has also demonstrated better tolerability and a lower discontinuation rate, allowing patients to maintain treatment for a longer period of time, thereby further improving the therapeutic effect. Overall, aximini provides a new treatment option for patients with chronic myelogenous leukemia with its unique target selection and significant clinical effect.