Phase I trial demonstrates the safety of annamycin in patients with AML

A phase I open-label, single-arm US trial demonstrated the safety of annamycin in patients with relapsed/refractory (R/R) acute myeloid leukemia (AML).¹

Annamycin is a next-generation, liposome-formulated anthracycline that, in 2019, received FDA Fast Track designation for the treatment of patients with R/R AML. Annamycin has been designed to avoid the cardiotoxicity and multidrug resistance that often occur with approved anthracyclines.

In this phase I trial, annamycin was given to patients at or below the FDA-established maximum dose for anthracycline, and the cardiotoxicity was determined by echocardiograms and cardiac health biomarkers, such as blood troponin levels. The study met its primary safety endpoint, with no evidence of cardiotoxicity. No unexpected serious adverse events or dose-limiting toxicities were observed in any of the six patients evaluated in this trial.

In addition, a preliminary efficacy assessment showed that one out of six patients achieved CR with incomplete recovery of platelets or neutrophils, and another patient had a substantial remission of leukemia cutis, from diffuse to 3 small lesions.

Further studies may provide evidence for annamycin efficacy in patients with R/R AML while avoiding cardiotoxicity associated with the currently approved anthracyclines.