Aaron Gerds, MD, MS

Dr. Aaron Gerds explained when treatment with rusfertide will become accessible for patients with a myeloproliferative neoplasm like polycythemia vera.

When Rusfertide May Become Accessible for Those with Polycythemia Vera

 to explain which factors will determine if treatment with rusfertide will become an accessible treatment option for patients with a myeloproliferative neoplasm like polycythemia vera. This conversation came following a presentation of data from the phase 3 VERIFY trial.

Overall, investigators shared that the investigational agent was able to 

reduce or eliminate the need for phlebotomies

 (a procedure which removes extra blood cells from the body by using a needle to take blood from the vein), according to Gerds, which would improve the quality of life of patients and help with symptom control.

In the interview with CURE, Gerds, who is an assistant professor of medicine at Cleveland Clinic Taussig Cancer Institute, also highlighted what 

patients should know about the VERIFY trial

What factors will determine whether rusfertide becomes a widely accessible option for patients, and how can individuals advocate for access to emerging treatments like this one?

These are just the initial, top-line results — the week 32 data

. The week 52 data is expected [this year (2025)], so you can mark that on your calendars. I believe the longer-term data will be helpful in answering those questions.

The company that makes the drug is certainly in conversations with regulatory authorities, asking, “What else is needed to have a good chance of approval to submit that new drug application?” That's going to be the next step: gathering all the data, the entire packet, and putting together a new drug application to submit to the regulatory authorities, or the U.S. Food and Drug Administration (FDA) in this case. Then, that packet or application, if you will, gets reviewed by the FDA. There's a number of reviews, hearings, and all kinds of processes that go along with that. At the back end of that, that's when we'll have a decision on regulatory approval.

Regulatory approval is just that next step. When you say, “access and widely available,” the step after that is insurance coverage. Here in the United States, our healthcare system is often run by insurance companies. What will that regulatory approval look like? What will it include? What levels of disease and what parameters? Then there will be guidelines that come out, for example, from the NCCN, which will make guideline recommendations on the use of rusfertide.

These two factors will be taken together, along with pricing and other factors. Then the insurance companies will decide what they will and won't cover, and that may actually impact access for a lot of patients.

“Protagonist and Takeda announce positive topline results from phase 3 VERIFY study of rusfertide in patients with polycythemia vera,” by Protagonist Therapeutics and Takeda. News release.

Transcript has been edited for clarity and conciseness.

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Emerging treatments like rusfertide may reduce phlebotomies and improve life for patients with polycythemia vera, according to Dr. Aaron Gerds.

New Therapies Advance for Rare Blood Cancer Polycythemia Vera

Dr. Aaron Gerds, assistant professor of medicine at Cleveland Clinic Taussig Cancer Institute, emphasized the growing momentum in treatment development for polycythemia vera, a rare blood cancer. While the disease may not receive widespread attention, he noted that researchers are actively pursuing new therapies, including rusfertide and other agents targeting the hepcidin pathway.

Rusfertide, in particular, may reduce or eliminate the need for phlebotomies, potentially improving patients’ quality of life. Gerds highlighted that multiple new therapies are being studied — such as givinostat in a phase 3 trial — and stressed that even in rare diseases, progress is being made.

What would you like patients to take away from today's conversation?

There are a couple of important things to note. One is that we are developing — and continue to develop — new therapies for polycythemia vera. Even though it’s a rare disease and not often highlighted in the broader pantheon of diseases presented at ASCO, it’s still a very important condition, especially for the people who have it.

There is a group of us who are very invested in this disease and are working hard to develop new therapies for it. Rusfertide isn’t the only one in development — there are also other drugs targeting the hepcidin pathway, as well as agents outside that pathway. For example, givinostat is currently in a randomized phase 3 trial as well as a registration-type study, also seeking approval.

Even though it’s rare, we are working hard to develop better treatment options. That’s the most important takeaway.

Specifically, with rusfertide, it’s exciting because it’s a new therapy that reduces or even eliminates the need for phlebotomies. That can really improve patients’ quality of life, which is important. Having another tool in our arsenal to care for these patients is exciting and can offer a lot to our patients.

Meta: Dr. Aaron Gerds breaks down the outcomes of the phase 3 VERIFY trial, investigating rusfertide, for patients with polycythemia vera, a type of blood cancer.

Breaking Down the VERIFY Trial For Patients With Polycythemia Vera

Data from the phase 3 VERIFY trial were shared at the 2025 ASCO Annual Meeting, where investigators noted that rusfertide 

may be a potential treatment option for patients

 with polycythemia vera, a type of blood cancer, according to Dr. Aaron Gerds. This is because the investigational therapeutic agent was able to 

, a procedure which removes extra blood cells from the body by using a needle to take blood from the vein, improving quality of life and symptom control.

To further discuss this data, Gerds sat down for an interview with CURE, in which he 

highlighted what patients should know about the VERIFY trial

What should patients know about the VERIFY trial, presented at this year’s ASCO Annual Meeting?

The ASCO Annual Meeting is always a challenging conference for those of us in hematology. Hematologic neoplasms often represent a smaller portion of the larger ASCO meeting, and sometimes you really have to sift through numerous abstracts to find something truly impressive. However, this year, we had the fortunate event of a hematologic malignancy being featured as a plenary abstract. This is the prestigious session where they select the top few abstracts to present to a huge, enormous crowd, genuinely highlighting the importance of this research.

The fact that it concerns polycythemia vera is particularly significant for a somewhat rare blood cancer that often doesn't receive much attention. It's a disease we've known a great deal about for a long time but have relatively few approved therapies. As of today, there are only two FDA-approved drugs for polycythemia vera: Jakafi (ruxolitinib), approved in the second-line setting, and Besremi (ropeginterferon alfa-2b-njft), approved for all settings. Given this limited therapeutic landscape, a new, effective drug like rusfertide, which presents a completely different mechanism, is absolutely warranted for the field.

The trial presented at this year's ASCO meeting was a classic, definitive phase 3 registration study. It included patients who received the drug and those who did not, allowing for a thorough comparison with all the statistical power needed for regulatory approval. This was the trial in question.

Dr. Aaron Gerds breaks down the outcomes of the phase 3 VERIFY trial, investigating rusfertide, for patients with polycythemia vera, a type of blood cancer.

Breaking Down the VERIFY Trial for Patients With Polycythemia Vera

Expanding on a New Way to Manage Polycythemia Vera Without Iron Deficiency

The VERIFY trial explores rusfertide’s role in managing polycythemia vera by controlling red blood cell production without causing iron deficiency symptoms, explained by Dr. Aarron Gerds from the Cleveland Clinic. Traditional treatment with phlebotomies reduces red cells by inducing iron deficiency, which leads to fatigue and other symptoms.

Rusfertide works by trapping iron, thereby tricking the bone marrow into reducing red cell production while avoiding typical iron deficiency side effects. Dr. Gerds highlights this as a novel approach in polycythemia vera treatment and contrasts it with related myelofibrosis therapy, where hepcidin levels are lowered to treat anemia.

Dr. Aaron Gerds is an assistant professor of medicine at Cleveland Clinic Taussig Cancer Institute specializing in myeloproliferative neoplasms, myelodysplastic syndromes and stem cell transplantation.

What do we currently know about how rusfertide affects patient symptoms? is there any evidence it improves overall quality of life compared with standard phlebotomy?

The way we typically treat polycythemia is with phlebotomies, which lead to iron deficiency — and that’s how they control red blood cell production. But iron deficiency itself can cause a lot of symptoms: fatigue, muscle and joint aches, and even pica, a craving to eat ice or dirt. Many of the symptoms patients experience are tied to iron deficiency, which is part of how phlebotomies work to treat polycythemia vera.

Rusfertide works differently. It’s a synthetic version of hepcidin, the body’s natural regulator of iron. Think of hepcidin like a cork in a bottle: your body stores iron in that bottle, and hepcidin keeps the cork in place so the iron can’t get out. When rusfertide is present, iron stays trapped in those stores, and the bone marrow is tricked into thinking the body is iron deficient, even though it’s not. That way, you can control red blood cell production without causing the typical symptoms of iron deficiency. It’s a clever approach.

We’ve known about hepcidin for a long time — it’s something we learned about in medical school — but this is really the first time we’re harnessing it in this way to treat polycythemia vera.

Here’s an even cooler part: we’re using this same pathway in the opposite direction for myelofibrosis, a closely related disease. In myelofibrosis, we often see anemia, so we’re trying to lower hepcidin levels to help boost red cell production. In polycythemia vera, we’re effectively raising hepcidin to lower red cells, and in myelofibrosis, we’re lowering hepcidin to increase them. In the same field, we’re manipulating the same pathway in two different ways to treat two different problems.

The VERIFY trial explores rusfertide to manage polycythemia vera without causing iron deficiency symptoms, Dr. Aaron Gerds explained.

Aaron Gerds, MD, MS

When Rusfertide May Become Accessible for Those with Polycythemia Vera

New Therapies Advance for Rare Blood Cancer Polycythemia Vera

Breaking Down the VERIFY Trial for Patients With Polycythemia Vera

Expanding on a New Way to Manage Polycythemia Vera Without Iron Deficiency

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