VIDEO: Potential of mutant-specific therapeutics in myelofibrosis

The landscape for MF is definitely changing. We're seeing, you know, therapies and combinations upfront. We're even seeing therapies before JAK inhibitors. There's another selinexor study where you get selinexor upfront and then you only add on the JAK inhibitor if you have a suboptimal response after assessment, so that's an interesting design. I'm very excited about the MDM2 inhibitor story. I'm still excited about Imetelstat. I still think that there's a path forward for pelabresib. I think all of this is good and it helps the field move forward. It gives patients options.

What's really gonna be on the horizon, which is still a little early, but we might start getting some hints of it later this year are mutant-specific therapeutics, so we have the mutant CALR antibody by Incyte, which is really moving forward. I think that has a potential to be transformative and it's early phase first-in-human study, so too early to say anything right now, but I remain optimistic about that study and then in the JAK2 mutants arena, we've got Ajax with a type II selective inhibitor and Incyte's JAK2V617F selective inhibitor, so different approaches, different angles, on the same theme of shutting down the JAK-STAT signaling pathway more effectively. In preclinical modeling, all of these look really exciting and potentially transformative. I do think that at some point, we're going to hit on some of these.

I applaud the patients that come to centers like mine and get involved in studies 'cause it really moves the field forward and helps the greater patient population but also helps them. We're fortunate. It's a rare disease, but it's a collaborative group and I think there's a unified vision of trying to really have the science lead us forward in terms of clinical development and that's exciting.