VERIFY Study: Rusfertide Shows Long-Lasting Control | Andrew Kuykendall, MD

Hi, my name is Andrew Kirgenevich from Moffitt Cancer Center in Tampa, Florida. And here at ASH in 2025, we were able to present the 52-week data, parts 1a and part 1b of the phase 3 VeriFy study, which was looking at Rusfertide, a hepcidin mimetic, compared to placebo in patients with polycythemia vera. So earlier this year, we got to present the part 1a data, which is the primary results of the VeriFy study at ASCO. And there we showed that Rusfertide, compared to placebo, was able to meet its primary endpoint and all key secondary endpoints. It was able to achieve hematocrit control, stable hematocrit control, decreased risk for or decreased need for phlebotomies, as well as symptomatic improvement in patients with polycythemia vera compared to placebo. Here we expanded upon the VeriFy study results that had been previously presented to go out to week 52. So the study was done in an interesting way, where parts 1a was a randomized portion, but in part 1b, patients who were initially randomized to placebo actually rolled over onto Rusfertide. So here we got to see those patients who initially didn't frequently receive a response compared to Rusfertide. We got to see what happened to them. And fortunately, what we saw was actually quite expected and relatively boring, because it was exactly what we thought we would see, which is patients who went onto Rusfertide from placebo had rapid control of their hematocrit, did not need phlebotomies, and really the curves came back together with Rusfertide in many different parameters. And so we looked at various different like impact on iron, as well as different impacts on blood counts and various different things like mean cell volume, which is kind of a marker of how red blood cells officially use iron. And what we saw is really those patients who were on placebo continue to get that benefit as they switched over to Rusfertide. And those patients who stayed on Rusfertide maintained their benefit. Actually we saw increased response rates for those, suggesting maybe patients that didn't respond, the few patients that didn't respond in part 1a, did respond in part 1b. And so it was very exciting data from a large phase three trial, and based upon these results, Rusfertide are actually seeking regulatory approval as we speak.