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Video

Targeting t(4; 14) Myeloma with a New Oral MMSET Inhibitor | Saad Usmani, MD

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• December 23, 2025

Description

Saad Usmani, MD, shares early results from a first-in-class oral MMSET/NSD2 inhibitor, KTX-1001, designed for high-risk multiple myeloma patients with translocation 4;14. In a dose escalation study with 40 patients, KTX-1001 showed promising activity and manageable side effects, with thrombocytopenia as the main concern. This oral therapy could provide a new targeted option for patients with aggressive myeloma, including those with additional genetic risk factors.

On this video

Transcript

Hello.

My name is Saad Usmani.

I'm the chief of the myeloma service at Memorial Sloan Kettering Cancer Center in New York.

So one of the studies that I have presented here at ASH in Orlando is, the dose escalation phase one study of the first in class oral MMSET/NSD2 inhibitor called ktx-1001 on behalf of the study team,

translocation 4;14 is making up about 10 to 15% of the newly diagnosed multiple myeloma patients.

And historically, these patients used to be considered high risk.

And so what what happens with this translocation is that there is, an over expression of MMSET, which is also called NSD2.

So this particular, oral inhibitor can help down regulate,

epigenetically H3K36me2 and that helps in reducing the oncogenic potential and proliferation of these cancer cells.

So that's the whole concept in terms of science.

This was a dose escalation study with the objective of safety.

We had 40 patients who participated in this study so far.

And it's gone through nine different dose cohorts.

And we are getting to the dose limiting toxicity, which appears to be thrombocytopenia.

And we're starting to see some single agent activity.

One thing I do want to point out is that this is a single agent dose escalation.

So steroids were not part of, the treatment strategy here.

It's important to note that the study was enriched for patients who have translocation 4;14.

So almost half of the patients had translocation 4;14.

19 of the 40 patients had it.

And then in addition to translocation 4;14,

some patients actually also had either gain of 1q or deletion of 1p.

Along with that.

And then four out of the 40 patients also had deletion 17p.

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