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MPN Workshop of the Carolinas 2025 | The most promising emerging therapies and developments in PV and ET

Lucia Masarova, MD, The University of Texas MD Anderson Cancer Center, Houston, TX, discusses emerging therapies and promising developments in the field of polycythemia vera (PV) and essential thrombocythemia (ET). Dr Masarova highlights the potential of interferons, mutant calreticulin (mutCALR)-targeted therapies, and Type II JAK inhibitors in these diseases. This interview took place at the 2nd Annual MPN Workshop of the Carolinas, held in Charlotte, NC.

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Transcript

Oh, that’s an interesting question. I think there’s a lot of attention sparked in interferons, where we have some data in interferons for ET in, for example, the second-line setting, the study that compared ropeg versus anagrelide that was presented in these last two conferences, that showed significantly improved, actually all outcomes for patients with ET...

Oh, that’s an interesting question. I think there’s a lot of attention sparked in interferons, where we have some data in interferons for ET in, for example, the second-line setting, the study that compared ropeg versus anagrelide that was presented in these last two conferences, that showed significantly improved, actually all outcomes for patients with ET. And there were two studies that finished for PV and ET and there’s currently an MF study happening with ropeg. So those are interesting to see as a potential disease modifiers. 

I think the biggest boom is the calreticulin approach where we have great results very early with a median therapy of only about 26 weeks, but already data in ET patients refractory to frontline setting, that we’re taking anti-calreticulin monoclonal antibody. And then the set of responses, including calreticulin molecular, were actually stunning. So I think that’s the most hot topic. 

There are already more developments in there to armor, to create bispecific, even CAR-T cells to digest and target the calreticulin, which is very hot in the space. There are studies also in early myelofibrosis, but now we’re talking about PV/ET. So for ET, I think the calreticulin, for PV, also the interferons, better space, and also the mutant JAK inhibitors. So now we are opening a new space, a new era of JAK inhibition, where we are kind of building on to the first approved, which are type I ATP kind of like competing drug inhibitors, which all of the currently approved are type I. And now we are looking at mutant-specific or type IIs. So that’s an exciting space, and I cannot wait to see the results.

 

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