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ASH 2021 | Phase III trial of rusfertide in patients with polycythemia vera

Phase II studies have demonstrated that rusfertide, a hepcidin mimetic, is very well tolerated and effectively achieves hematocrit control in patients with polycythemia vera (PV). Treatment with rusfertide essentially eliminated phlebotomy requirements, as well as improving symptomatic iron deficiency: an unaddressed clinical challenge in patients with PV. Srdan Verstovsek, MD, PhD, University of Texas MD Anderson Cancer Center, Houston, TX, shares the details of the planned Phase III investigation, following the promising results demonstrated to date. The multicenter, randomized, blinded trial will compare the safety and efficacy of rusfertide versus placebo as an add on to current PV therapy. The 32-week study period will be followed by an open-label treatment phase and a long-term extension. Dr Verstovsek gives his thoughts on the promise of rusfertide as a new therapy in the near future. This interview took place at the 63rd ASH Annual Meeting and Exposition congress in Atlanta, GA.

Transcript (edited for clarity)

Rusfertide, or PTG-300, is hepcidin mimetic. That means, that it regulates the iron metabolism in the body of the person. Now, iron metabolism is a very important part of the polycythemia vera patient’s biology, because the iron is utilized for growth of red blood cells. Polycythemia vera patients have too many red blood cells. And the goal of therapy is to decrease the growth of the red blood cells to maintain the hematocrit, which is a percent of blood that is red blood cells below 45%...

Rusfertide, or PTG-300, is hepcidin mimetic. That means, that it regulates the iron metabolism in the body of the person. Now, iron metabolism is a very important part of the polycythemia vera patient’s biology, because the iron is utilized for growth of red blood cells. Polycythemia vera patients have too many red blood cells. And the goal of therapy is to decrease the growth of the red blood cells to maintain the hematocrit, which is a percent of blood that is red blood cells below 45%. So people do phlebotomies, people use cytoreductive therapy. And here, with this hepcidin mimetic, we have a possibility of affecting, in a positive way, polycythemia vera patients, in a novel way, by a redistribution of iron in the body of polycythemia vera patients. What rusfertide does, it keeps the iron inside the liver, inside the spleen, inside the reticuloendothelial system. There is less iron for cell growth in the bone marrow. So giving rusfertide to PV patients, eliminates the need for phlebotomy. Red blood cells stay low. That’s the primary goal of polycythemia vera therapy.

In the ongoing Phase II studies, there are two presented at ASH this year, it was extremely effective. Almost every single patient, immediately after starting therapy with rusfertide, eliminated the need for phlebotomy. People also feel better. So the signs of activity are clear. It’s very safe. It’s given once a week under the skin. There are not many side effects, whatsoever. Some may be related to the injection under the skin. And therefore, the Phase III study, which I presented, is planned for patients with polycythemia vera, that may be just treated with phlebotomy, or are already receiving cytoreductive therapy, to normalize the counts, but it’s not working. Any patient that has too many phlebotomies given, that has polycythemia vera, will be eligible for a prospective randomized blinded study, between rusfertide and placebo, to see whether it will be approved as a new therapy in the near future.

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