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EHA 2026 | Results from the Phase III RISE UP study investigating mitapivat in sickle cell disease
In this interview, Biree Andemariam, MD, University of Connecticut, Farmington, CT, presents encouraging results from the Phase III RISE UP trial (NCT05031780), which examined the efficacy of mitapivat in patients with sickle cell disease (SCD). Dr Andemariam highlights that mitapivat treatment led to a statistically significant hemoglobin (Hb) response, and among patients who achieved an Hb response, additional clinical benefits were observed. These included reduced rates of sickle cell pain crises (SCPC), associated hospitalizations, and fatigue. This interview took place at the 31st Congress of the European Hematology Association (EHA) in Stockholm, Sweden.
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Transcript
So I’m very excited about the RISE UP Phase III clinical trial results that were presented today. RISE UP was a global randomized placebo-controlled clinical trial examining the role of a new first-in-class treatment for people living with sickle cell disease. And today I gave the top-line results of a global clinical trial that enrolled over 200 individuals living with sickle cell disease...
So I’m very excited about the RISE UP Phase III clinical trial results that were presented today. RISE UP was a global randomized placebo-controlled clinical trial examining the role of a new first-in-class treatment for people living with sickle cell disease. And today I gave the top-line results of a global clinical trial that enrolled over 200 individuals living with sickle cell disease. So what I was able to share today was the result of two primary endpoints, one being hemoglobin response and one being the rate of sickle cell pain crises or SCPCs that people experience over the course of a year. And what we found is that there was a statistically significant increase in hemoglobin compared to placebo. 40.6% of patients responded compared to only 2.9% of those on placebo. What we found is that this was related to improvements in hemolytic anemia. One thing that’s really important to highlight is the subgroup analysis of hemoglobin responders versus non-responders. And what we saw in that subgroup analysis is that hemoglobin responders actually had a reduction in sickle cell pain crises annually, as well as the sickle cell pain crises that require hospital visits or ER visits. It also reduced their length of hospital stay. It also improved their fatigue, and fatigue is one of the most important symptoms that individuals living with sickle cell disease struggle with. Also, compared to placebo, individuals who were randomized to receive mitapivat were less likely to undergo transfusions during the 52-week study period and had a significant reduction in the number of blood transfusion units that they received. So this can be just so important, not only to how they feel, their degree of anemia, but also a reduction in pain crises as well as transfusion support. Also, what’s really important to note is that the hemoglobin responder group had a 1.6 gram per deciliter increase in their average hemoglobin between weeks 24 and 52, which is amazing.
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Disclosures
Research funding: Afimmune, Agios Pharmaceuticals, Inc., American Society of Hematology, Connecticut Department of Public Health, US Health Resources and Services Administration, Hemanext, Novartis, Novo Nordisk, Pfizer; Advisory board participation/consultancy: Accordant, Afimmune, Agios Pharmaceuticals, Inc., Beam Therapeutics, bluebird bio, Bristol Myers Squibb, Chiesi, Editas, Global Blood Therapeutics, Fulcrum Therapeutics, Hemanext, Novo Nordisk, Octapharma, Pfizer, Protagonist, Roche, Sanofi Genzyme, Vertex Pharmaceuticals.
