Andrew Kuykendall, MD, Moffitt Cancer Center, Tampa, Florida, presented updated week 52 results from the phase 3 VERIFY trial evaluating rusfertide, a weekly, self-administered, subcutaneously injected first-in-class hepcidin mimetic,  among patients with polycythemia vera (PV). 

The data confirmed durable hematocrit control, improved symptom scores, and reduced need for phlebotomy among patients who continued rusfertide, while those who crossed over from placebo achieved similar rapid responses. 

Dr Kuykendall concluded, “We got to present a lot of good data, really building upon what we already presented, showing great response rates, great tolerability.”

Transcript:

Hi, I'm Andrew Kuykendall. I'm an associate member in the Department of Malignant Hematology at Moffitt Cancer Center in Tampa, Florida.

At ASH 2025 we got to present the results of the phase 3 VERIFY study, presented actually part 1a and part 1b. This was a study we previously presented at ASCO earlier this year. In that portion we presented the 1a part of the study, which was the primary assessment and the week 32 data. 

Here, we actually expanded upon that and presented the week 52 data. Why is that important? The study was designed where patients were randomized. These are patients with polycythemia vera. They were randomized, either rusfertide versus placebo and they were monitored for 32 weeks. The primary end point was the ability of rusfertide to decrease the eligibility for phlebotomy or basically control hematocrit from weeks 20 to 32. There were also some key secondary end points that were looked at from weeks 0 to 32.

What we saw there in the part 1 portion of the study is that rusfertide met all its primary end points and all key secondary end points. Those end points really reflected the ability to control hematocrit in a stable fashion as well as some key patient reported outcomes, suggesting that it would improve fatigue and Myelofibrosis Total Symptom Score (MFSAF TSS), which is a kind of disease specific symptom assessment form. In the part 1A, we showed kind of compared to placebo, rusfertide met primary and key secondary endpoints, and then part 1b is important because at week 32, everyone who was on placebo rolled over onto rusfertide and everyone who was on rusfertide continued therapy. We got to see kind of the durability of data when you look at the rusfertide-treated patients and you also got to see additional response rate data when you look at those patients on placebo who rolled over.

Fortunately, what we saw was actually quite boring. It was exactly what you'd like to see, which is those patients who stayed on rusfertide were able to consistently maintain control of their hematocrit throughout the next 20 weeks. From weeks 30 to 52 actually saw maybe some increased response rates suggesting maybe patients that didn't achieve a response in the first portion of the study then gained a response in the second portion of the study. 

Those patients who were on placebo who rolled over onto rusfertide, what we saw was very similar to what we saw is patients originally randomized to rusfertide, we saw response rates of around 77%. Those patients were rapidly able to get their hematocrit under control, not need phlebotomies, get nice and stable control of the hematocrits moving forward. We also looked to here a little bit more in detail at some iron parameters suggesting that iron deficiency is largely abrogated with the addition of rusfertide, ferritin levels rising. We know that iron deficiency is something that causes our patients a lot of symptoms.

We got to present a lot of good data, really building upon what we already presented, showing great response rates, great tolerability. We saw patients who were on placebo respond, and also some key metrics in terms of iron. I’m really excited for where this drug's going and based upon the data we presented this week. Rusfertide will be seeking regulatory approval in the coming months.

Source:

Kuykendall A, Bankar A, Pettit K, et al. Rusfertide or placebo plus current standard-of-care therapy for polycythemia vera: Durability of response and safety results through week 52 from the randomized controlled phase 3 VERIFY study. Dec 6-9, 2025; Orlando, FL. Abstract: 81