Curis Shares New Data from TakeAim Leukemia Study

Curis, Inc., a biotechnology firm dedicated to advancing the development of emavusertib (CA-4948), has recently announced updated findings from their ongoing TakeAim Leukemia study. This study focuses on relapsed or refractory acute myeloid leukemia (AML) and the updated data will be shared at the upcoming ASCO and EHA conferences. The study has now expanded its dataset to include 30 patients, up from the previous five.

The expanded dataset reveals new information about 25 additional patients. Specifically, these patients were divided into two groups based on their genetic mutations: those with the FLT3 mutation (FLT3m) and those with U2AF1/SF3B1 splicing factor mutations (SFm). These patients had undergone fewer than three lines of prior therapy and were administered emavusertib as monotherapy at the recommended phase 2 dose (RP2D) of 300 mg taken twice daily.

In the FLT3m cohort, 12 relapsed/refractory AML patients were part of the study. Among these patients, prior treatments included venetoclax (8 out of 12), hypomethylating agents or HMA (9 out of 12), and FLT3 inhibitors (9 out of 12). Preliminary results show that six of the 11 response-evaluable patients exhibited objective responses, with three patients achieving complete remission (CR), one achieving CR with partial hematologic recovery (CRh), and two reaching a morphologic leukemia-free state (MLFS). Four patients were still undergoing treatment at the data cut-off. Notably, three out of three patients who had not previously been treated with FLT3 inhibitors showed objective responses, and three of eight patients who had progressed following prior FLT3 inhibitor treatment also exhibited objective responses. All responders demonstrated complete normalization of bone marrow blast counts, and one patient proceeded to allogenic stem cell transplantation. Five out of the six responses occurred within a single treatment cycle.

The SFm cohort included 20 relapsed/refractory AML patients. Previous treatments among these patients included venetoclax (18 out of 20) and HMA (17 out of 20). Preliminary data indicated that four out of 18 response-evaluable patients achieved objective responses (CR/CRh/MLFS). Eight patients were ongoing at the data cut-off point, and among these, one demonstrated MLFS and three non-responding patients showed increased neutrophil counts. All responders in this group had received prior HMA treatment, and three had also undergone venetoclax treatment. Two patients were not response-evaluable. Like the FLT3m cohort, all responders in the SFm group also showed complete normalization of bone marrow blast counts, and one patient proceeded to allogenic stem cell transplantation.

Dr. Robert Martell, Chief Scientific Officer of Curis, highlighted the clinical significance of increased neutrophil counts in non-responding patients, as low neutrophil counts often lead to fatal infections in AML patients. Meanwhile, James Dentzer, President and CEO of Curis, expressed optimism about the continued efficacy of emavusertib, both as a monotherapy and in combination with other treatments for AML.

Curis is actively involved in advancing emavusertib, which is also being tested in other studies, including the Phase 1/2 TakeAim Lymphoma study in combination with the BTK inhibitor ibrutinib and as a frontline therapy for AML. Emavusertib has received Orphan Drug Designation from the U.S. FDA for the treatment of AML and MDS.

The company is backed by a collaboration with Aurigene and has licensed its rights to Erivedge® to Genentech for the treatment of advanced basal cell carcinoma. Curis faces various challenges and risks, including the need for substantial additional capital, potential regulatory hurdles, and competition from other drugs. However, the company remains focused on its mission to develop innovative treatments in oncology.