Dr Kremyanskaya on the Safety of Rusfertide in Phlebotomy-Dependent PV

Marina Kremyanskaya, MD, PhD, assistant professor, medicine, hematology, and medical oncology, Icahn School of Medicine, Mount Sinai, medical director, Inpatient Oncology Unit, The Mount Sinai Hospital, discusses the safety profile and future implications of rusfertide (PTG-300) in the treatment of patients with phlebotomy-dependent polycythemia vera (PV), as seen in the phase 2 REVIVE trial (NCT04057040).

The REVIVE trial evaluated rusfertide vs placebo in patients with PV requiring phlebotomy. At the 2023 EHA Congress, REVIVE investigators presented data from this open-label, dose-escalation study, which showed that treatment with rusfertide led to significantly higher efficacy compared with placebo in this patient population.

Rusfertide was very well tolerated in the study, and most patients were able to remain on rusfertide treatment for the duration of the study, Kremyanskaya begins. Notably, some patients remained on the study for over 2.5 years by the time of data read out, Kremyanskaya adds. The REVIVE investigators reported that the major adverse effects (AEs) observed with rusfertide were grades 1 and 2, with the most common AE being injection site reactions, she notes.

In the phase 2 trial, rusfertide was given as a subcutaneous injection once weekly, and patients were able to self-administer the treatment, Kremyanskaya expands. As reported, most patients experienced low-grade injection site reactions that were typically limited and improved over time, Kremyanskaya emphasizes. Moreover, most patients did not need to discontinue the study because of injection site reactions, and no grade 4 or 5 AEs were reported. Overall, this drug is well tolerated in patients with PV, she says.

Now, the worldwide, randomized, placebo-controlled, phase 3 VERIFY trial (NCT05210790) is enrolling patients, Kremyanskaya continues. This study will provide investigators with more information about the efficacy of rusfertide in both controlling hematocrit levels and improving patient symptoms, she says. Overall, the VERIFY investigators are looking forward to completing enrollment and analyzing the data from this phase 3 study, she concludes.