Curis doses first patient in CA-4948 phase 1 clinical trial in AML and MDS

CA-4948 phase 1 clinical trial : US biotech company Curis said that it has dosed the first patient with CA-4948 in a phase 1 clinical trial that is evaluating the small molecule IRAK4 kinase inhibitor for the treatment of acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS).

The phase 1 clinical trial for CA-4948 will involve patients having acute myeloid leukemia or high-risk myelodysplastic syndromes with spliceosome mutations like SF3B1 and U2AF1, that drive expression of the long isoform of IRAK4 (IRAK4-L).

According to Curis, IRAK4 plays a key role in the toll-like receptor (TLR) and interleukin-1 receptor (IL-1R) signaling pathways, which are often dysregulated in patients with acute myeloid leukemia and myelodysplastic syndromes.

The primary goal of the CA-4948 phase 1 clinical trial is to establish the maximum tolerated dose and recommended phase 2 dose of the IRAK4 kinase inhibitor, based on safety and tolerability, dose-limiting toxicities (DLTs), and also pharmacokinetic and pharmacodynamic findings.

At least three patients will be taken in at each dose level, beginning with 200 mg twice daily, which was ruled to be safe, capable of achieving relevant levels of drug exposure, and had shown signs of biologic activity and clinical efficacy in a separate, ongoing phase 1 study.

Each treatment cycle will be of 28 days and repeated in the absence of toxicity.

Curis expects to release initial data from the CA-4948 phase 1 clinical trial in Q4 2020.

James Dentzer – President and CEO of Curis said: “We quickly worked with our clinical investigators and the U.S. Food and Drug Administration (FDA) to design a study of CA-4948, our first-in-class IRAK4 inhibitor, in this population. We are pleased to announce today, just six months later, that we have initiated this new study and successfully dosed our first patient.”