ASCO 2025 | Results from the VERIFY trial: the encouraging efficacy of rusfertide in patients with PV

The Phase III VERIFY study was a study looking at the agent rusfertide in patients with polycythemia vera who are requiring consistent phlebotomies. It’s an agent that we looked at in the Phase II REVIVE trial and in that trial we saw that rusfertide had the ability to decrease phlebotomies and control hematocrit in patients with polycythemia vera. This is a treatment goal of ours in patients with polycythemia vera because we know consistent control of hematocrit correlates with a decreased thrombotic risk. Patients with polycythemia vera oftentimes are overproducing blood cells and we have to perform recurrent therapeutic phlebotomies whereby we drain them of their blood repeatedly over and over to achieve this hematocrit goal. Unfortunately, this often makes people feel quite terrible with their disease and often ties them to the healthcare system because they’re having to come back in for consistent or recurrent phlebotomies. 

We aim to use our understanding of the iron regulation system, if you will, to try to better control hematocrit, utilizing an agent called rusfertide, which actually is a mimetic of a protein called hepcidin. Hepcidin is like the master regulator of iron homeostasis in the body and actually decides if iron is distributed to the bone marrow for continued red blood cell production. Hepcidin really acts in settings of low iron or iron deficiency, whereby hepcidin is downregulated and really allows iron to freely flow to the bone marrow for more red blood cell production. The problem lies in the fact that polycythemia vera patients are making too many red blood cells, but they’re also iron deficient, and so all the iron they do absorb goes right to the bone marrow for continued red blood cell production, and we have to then tackle that with more phlebotomies. Instead, we decided let’s tackle this with a mimetic of hepcidin called rusfertide. So in these patients, rusfertide actually causes iron to not be delivered into the blood and not be given to the bone marrow, thereby controlling red blood cell production through the restriction of iron through the provision of rusfertide. 

So in this Phase III trial, we took patients that had polycythemia vera who were requiring phlebotomies and randomized them one-to-one to receive placebo versus rusfertide in addition to their current standard of care. And the results were quite interesting. We found we were virtually able to eliminate the need for phlebotomies in these patients, consistently control their hematocrit. Also what we saw was really interesting was it had the ability to improve disease-related symptoms, specifically things like fatigue that can be quite plaguing for patients who live with this disease for a long time. 

So in summary, what we saw from VERIFY was an agent that can be given, get rid of the need for phlebotomy, get people away from the healthcare system, give them a bit more freedom back and potentially improve their symptoms and hopefully this data will support using this as an improved agent in the near future.

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