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ASCO 2025 | The Phase III PyramIDH trial: ivosidenib as a treatment for HMA-naive MDS with IDH1 mutation
Valeria Santini, MD, University of Florence, Florence, Italy, comments on the Phase III PyramIDH (NCT06465953) trial, which will investigate ivosidenib as a treatment for hypomethylating agent (HMA)-naive, IDH1-mutated myelodysplastic syndromes (MDS). The study will compare ivosidenib to azacitidine, aiming to elucidate whether ivosidenib can be used as a comparable frontline therapy. This interview took place during the 2025 American Society of Clinical Oncology (ASCO) Meeting in Chicago, IL.
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Transcript
And also at ASCO, I presented a poster with a study design, a very interesting new approach with ivosidenib, that is a specific oral drug targeting IDH1-mutated myelodysplastic syndromes. And the idea of this study is to treat HMA-naive IDH1-mutated MDS with any IPSS-M score but eligible for azacitidine and compare in a randomized fashion treatment with ivosidenib monotherapy to azacitidine monotherapy...
And also at ASCO, I presented a poster with a study design, a very interesting new approach with ivosidenib, that is a specific oral drug targeting IDH1-mutated myelodysplastic syndromes. And the idea of this study is to treat HMA-naive IDH1-mutated MDS with any IPSS-M score but eligible for azacitidine and compare in a randomized fashion treatment with ivosidenib monotherapy to azacitidine monotherapy. This is a very interesting and new approach that will be dedicated in this specific study to elucidate whether this kind of treatment is really comparable to azacitidine. There has been a study by the French group showing that indeed with ivosidenib you can obtain a CR rate in high-risk MDS of nearly 50%, 48%, with an overall response rate of 78%, showing that this drug that is presently approved only for relapsed, refractory, high-risk MDS can be used as frontline therapy. In fact, the patient population, I told you, would be also with lower intermediate MDS, but eligible for hypomethylating agents, not in Europe, but in the US and in other countries. The study endpoints will be the achievement at full months of CR or PR. And then of course, there will be secondary endpoints like the duration of this response, the time to achieve this response and the AML transformation rate as well as others. I think that with these, we are opening new doors for treatment of lower risk MDS and for higher risk MDS in first line. Of course, a total oral therapy is what we are looking for for our patients. So in this sense, we have targeted drugs like ivosidenib that could be promoted to first-line treatment, both for lower intermediate patients with other cytopenias than only anemia and for higher risk. So I think it’s a very promising moment. The name of this new study is a Phase III study that is comparing, as I said, ivosidenib to azacitidine and it’s called Pyramid. So let’s hope that we can really climb this pyramid and obtain good results for our patients.
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