New Fusion Protein Effective, Safe in First-Line MDS therapy

By Andrew Moreno - Last Updated: June 26, 2024

In a recent phase II study conducted in adults with untreated higher-risk myelodysplastic syndromes (MDS), a combination of a new fusion protein with azacitidine proved to be an effective and well tolerated first line therapy. These results were presented at the 2024 ASCO® Annual Meeting.

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The open-label, multicenter study had 57 enrolled patients. This cohort was 71.9% male and had a median age of 64 years. 43.9% of the cohort had high-risk disease and 31.6% very high-risk disease, as defined by the Revised International Prognostic Scoring System, and none of the patients was eligible for stem cell transplant or intensive chemotherapy.

The fusion protein under investigation, IMM01, was administered to the patients intravenously at a dosage of 2.0 mg/kg/week and subcutaneous azacitidine at 75 mg/m2 on days one through seven per 28-day cycle. The median duration of follow-up in the cohort after starting the regimen was 12.8 months.

In the 51 patients where efficacy could be assessed, the overall response rate was 64.7% and 29.4% of the cohort had complete response (CR). Of the patients, 15.7% had both marrow CR (mCR) and hematologic improvement (HI), while 5.9% had HI only and 13.7% mCR only. The estimated 12-month progression-free survival was 54.4%.

Next-generation sequencing analysis performed in the study found that NPM1 mutations significantly correlated with treatment response, especially CR.

Regarding grade 3 or higher treatment-related adverse events, leukopenia occurred in 78.9% of the cohort, thrombocytopenia in 66.7%, neutropenia in 66.7%, lymphopenia in 56.1%, anemia in 43.9%, infection in 15.8%, and pneumonia in 10.5%. When low-dose priming was not used, occurrence of grade 3 or higher hemolysis was 1.8%.

Reference

Yang W, Gao S, Yan X, et al. Latest results of a phase 2 study of IMM01 combined with azacitidine (AZA) as the first-line treatment in adults with higher risk myelodysplastic syndromes (MDS). Abstract #6510. Presented at the 2024 American Society of Clinical Oncology Annual Meeting; May 31-June 4, 2024; Chicago, Illinois.

Post Tags:ASCO24
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