FDA Puts Emavusertib Clinical Trial for R/R AML and MDS on Partial Hold

The FDA has placed a partial hold on Curis’ phase 1/2a clinical trial of emavusertib in adult patients with relapsed/refractory (R/R) acute myelogenous leukemia (AML) or high-risk myelodysplastic syndrome (MDS).

The agency decision is requesting additional data from the company, including efficacy and safety data, but also data related to rhabdomyolysis and information on planned emavusertib dosage in a phase 2 study, given that a participant with R/R/ AML who had rhabdomyolysis died in the trial as a result of an identified dose-limiting toxicity of emavusertib.

Emavusertib is an investigational drug that is a small-molecule interleukin-1 receptor-associated kinase 4 (IRAK4) inhibitor that was granted Orphan Drug Designation in 2021.

Patients already enrolled in the trial are allowed to continue taking the drug at 300 mg or lower twice a day, but no new patients can enroll in the study at the moment.