Dr Masarova on the Rationale for Investigating Ropeginterferon Alfa-2b in Essential Thrombocythemia

Lucia Masarova, MD, assistant professor, Department of Leukemia, Division of Cancer Medicine, The University of Texas MD Anderson Cancer Center, discusses the single-arm multicenter phase 2 EXCEED-ET trial (NCT05482971) of ropeginterferon alfa-2b-njft (Besremi) in adult patients with essential thrombocythemia.

At the 2023 ASCO Annual Meeting, Masarova and colleagues overviewed information from the ongoing phase 1/2 EXCEED-ET trial (NCT05482971) evaluating the efficacy, safety, and tolerability of ropeginterferon alfa-2b-njft in adult patients with essential thrombocythemia.

Pegylated interferons have been utilized in the treatment of patients with essential thrombocythemia and polycythemia vera over the past few decades, Masarova notes. These types of agents of interest for the treatment of patients in these populations due to possible disease-altering activity observed with their use, she adds, noting that interferons have the ability to attack the malignant clones of the disease.

Ropeginterferon alfa-2b-njft was previously approved by the FDA in November 2021 for use as a treatment in patients with polycythemia vera. The rationale for EXCEED-ET is to potentially bring the compound to patients with essential thrombocythemia, Masarova concludes.

The study is enrolling patients with essential thrombocythemia who are naïve to cytoreductive treatments, or who are pre-exposed to hydroxyurea or anagrelide. They also need to have a platelet count of at least 450 x 109/L at screening. Patients are receiving ropeginterferon alfa-2b-njft subcutaneously starting at 250 µg at week 0, 350 µg at week 2, and 500 µg at week 4. The agent is then continued at 500 µg every 2 weeks for up to 56 weeks.