Daniel DeAngelo, MD, PhD, of Dana-Farber Cancer Institute, presented findings at #ASH24 on two abstracts regarding a novel systemic mastocytosis (SM) drug with high response rates & a tool that identifies SM subtypes with 90% accuracy.
And at the American Society of Hematology, we have two exciting abstracts on systemic mastocytosis. Systemic mastocytosis comes in two different flavors. Uh, advanced systemic mastocytosis can be an aggressive, life-threatening, uh, subtype. Uh, it's rare, driven by the kit DH16V in about 90 to 95% of patients. And therefore targeting kit with tyrosine kinase inhibitors has been an avenue that we have explored. There's currently two FDA approved agents, and at Ash, I'm going to be presenting data on a part one dose finding study of bezolastinib. Blatab is a new in class tyrosine kinase inhibitor that targets the kit DH16V and spares many of the other kinases and therefore has minimal brain penetration and it has minimal off target toxicities. So in this uh part one or dose finding study, we were able to determine that the 100 mg twice daily dose uh was the optimal dose. Overall, in the uh 32 patients that were enrolled, 27 of them were invaluable, and we had an overall response rate of 52%. If we focus on just pathologic remission, the uh and response, the uh the response rate was 88% uh overall in these findings. So this was an exciting part one of the study showing really deep reductions in markers of mass cell burden and sustained response. So hopefully this will also uh uh uh parlay into a part two or a registration trial that should uh complete by the beginning of 2025 and report uh at this time next year.
