SOHO 2023 | Current and emerging therapies for polycythemia vera

It’s a quite nicely moving field where we actually have already approved agents, there was lately brought in ropeginterferon for patients with high-risk or low-risk polycythemia vera. That has also now entered a clinical trial called ECLIPSE that is going to evaluate the currently established and approved administration, which is at the beginning escalation by 50 micrograms every other week under the skin until we reach the target dose of 500. However, because it’s a slower escalation, we found out that maybe some patients that have a lot of disease to start with, might suffer consequences of uncontrolled disease by the time we reach the control dose. So, the other arm we will be comparing is a faster escalation using something we call the three-step. We’ll go 250 micrograms, 350 micrograms and 500 micrograms every other week, so that we reach the target dose faster. So that is very exciting to learn and see what we can gain from it, and how we can better customize the delivery. That is a very promising agent that actually is now even moving in ET and pre-fibrotic myelofibrosis, so lots of excitement about ropeginterferon. We’ve had two studies actually at this conference to show. And then we have a very important and physiologically smart hepcidin mimetic, which is an iron metabolism regulator drug that seems like a very interesting alternative to phlebotomies, or add-on therapies to other patients that we need to control phlebotomy needs. It was actually investigated in the Phase II REVIVE study, that was added to any therapy patients were on if they required phlebotomy, and rusfertide was shown to be extremely effective in eliminating the needs of phlebotomy. This was achieved in 83% of these patients during the initial 28-week treatment interval. So that is another very interesting agent that is currently enrolling in Phase III clinical trials. And it’s going to show us what would be the best place to get this agent in the treatment field, so that is another very interesting area of different drugs. We have very well established ruxolitinib, or Jakafi, in the second-line setting in these patients. Very exciting data came from Claire Harrison from the MAJIC-PV trial, which was another study that evaluated the effect of ruxolitinib on patients with PV, specifically thrombotic events, progression to myelofibrosis, event-free survivals, and clearly showed superiority. So that was a very interesting finding that we can actually be assured that the agent also does a lot for control of the disease, including vascular complications. There are currently studies that are placing these agents in a frontline setting as well, so we will learn how to better sequence them, as it looks like we will have a competing field.