SOHO 2025 | Safety and efficacy of ziftomenib in NPM1m/KMT2Ar R/R AML: insights from the KOMET-001 study

Yeah, that data has been now presented several times at key meetings. We hope to have the publication out very soon as well. KOMET-001 was a Phase I, Phase II study that looked at a dose escalation, dose expansion, and a Phase II evaluation of ziftomenib monotherapy for patients with NPM1 or KMT2A alterations. The Phase II focused mainly on NPM1-mutated AML. We presented that data last year or this past year, basically demonstrating that it met its primary endpoint of CR and CRh. And the compound, ziftomenib, now has a PDUFA date in fall of this year, and we shall see if the regulatory agency ultimately provides it with an approval for that indication, NPM1 mutated relapsed/refractory AML. The drug is well tolerated. There is no signal for QT prolongation. It seems to have activity in patients who are traditionally more resistant, such as those who have progressed on venetoclax or have relapsed following transplant. That’s very encouraging. There is a signal for differentiation syndrome, which is also seen with other menin inhibitors and is thought to be a class effect, which is important because we need to monitor for that particular entity very closely. But overall, I hope that it will be a promising addition in terms of targeted therapies for acute myeloid leukemia. There are also combination studies that are currently ongoing that hopefully will have a role for patients in the future.

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