Pivekimab sunirine

And this is an FDA approval for, uh, pivecamab sunirin, I'll just call it PS, um, which is a CD one twenty-three antibody drug conjugate.

So CD one twenty-three, uh, is...

Another name for this is IL-3 receptor alpha.

So this is a subunit of the interleukin-3 receptor.

Uh, and IL-3, uh, receptors are pretty low in expression on, on normal hematopoietic stem cells, but are overexpressed in AML cells, maybe some ALL cells, and then blastic plasmacelloid dendritic cell neoplasms, or BPDCN, which is a mouthful.

And so this is the approval for, for PS pivecamab sunirin, which is for, uh, blastic plasmacelloid dendritic cell neoplasm, a disease I've only seen one of these patients.

They quickly got, uh, referred to a higher level center, which is, is appropriate.

So it, it's a very, very uncommon disease.

This is less than one percent of all acute leukemias.

You should think of it as an acute leukemia, even though it doesn't have acute leukemia in the name.

It's blastic.

That's the leukemia part of it.

The acute leukemia is the blastic part, right? Uh, in fact, before two thousand and eight, just to illustrate how [laughs] little we know about this disease, it was called blastic in case cell lymphoma.

Um, but, you know, you will find information about this in like AML guidelines, for example.

Um, it's a challenging diagnosis because it is so rare, uh, and that can likely lead to some of the poor outcomes with this because diagnosis can take a while to be established.

Usually requires, you know, if you're at a, a, a, a primary or secondary center being referred to a tertiary center to get a high level medical pathologist who has seen a few of these, uh, in their career.

It can present with, uh, like asymptomatic skin lesions, cytopenias with or without circulating blasts, and then CNS disease is not that, uh, it's not uncommon, maybe in about ten percent of folks.

And, uh, a median overall survival is often quoted of eight to twelve months, which is not very poor, and that's with treatment.

Uh, and these patients need, uh, an allo stem cell transplant.

Uh, because it is so rare, we don't really have randomized controlled trials.

We have, you know, pretty subpar data, uh, as it comes to the quality of data.

It's a lot of observational data, some retrospective data of this is what we did, and we pieced together thirty patients and, and this is kinda what happened.