REVIVE: Rusfertide meets the primary endpoint in PV

Compared with placebo, rusfertide was associated with improved response rates in patients with polycythaemia vera (PV). The drug met all the efficacy endpoints and was well-tolerated.

“The peptide hormone hepcidin regulates iron homeostasis and controls the availability of iron for the formation of red blood cells,” explained Dr Marina Kremyanskaya (Mount Sinai, New York, USA). She presented the results of the phase 2 REVIVE trial (NCT04057040), which assessed the safety and efficacy of the hepcidin mimetic rusfertide in participants with PV who had a high phlebotomy burden while they were treated with standard of care therapy [1]. After completion of the phase 1 part of the trial, participants were randomised to placebo or to maintain their last dose of rusfertide. The primary endpoint was the combination of maintaining a haematocrit <45% and not reaching phlebotomy eligibility. In total, 53 participants entered the 12-week randomised withdrawal period and were included in the primary efficacy analysis.

“There was a meaningful reduction in phlebotomy frequency following rusfertide administration,” said Dr Kremyanskaya. 69.2% of the participants in the rusfertide arm and 18.5% in the placebo arm met the primary endpoint, representing a significant benefit for the rusfertide arm (P=0.0003). This result was comparable for participants who were previously treated with phlebotomy alone and those who received phlebotomy plus cytoreductive therapy.

The drug was generally well-tolerated. Grade 1 or 2 injection site reactions were the most common treatment-emergent adverse events. Symptoms associated with PV, like fatigue (31.4%), pruritis (25.7%), and headache (22.9%) were observed. 2 participants discontinued rusfertide, due to mild thrombocytosis and recurrent grade 1 injection site erythema, respectively.

Rusfertide and its effect on the absence of phlebotomy eligibility are currently being validated in the large phase 3 VERIFY trial (NCT05210790).

1. Kremyanskaya M, et al. Targeted therapy of uncontrolled erythrocytosis in polycythemia vera with the hepcidin mimetic rusfertide: blinded randomized withdrawal results of the REVIVE study. Late-breaking oral session, EHA 2023 Annual Congress, 8─11 June, Frankfurt, Germany.

Copyright ©2023 Medicom Medical Publishers



Posted on 2 August 20232 August 2023