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EHA 2026 | Bezuclastinib in advanced systemic mastocytosis: the APEX trial
Daniel DeAngelo, MD, PhD, Dana-Farber Cancer Institute, Boston, MA, discusses the safety and efficacy of bezuclastinib in advanced systemic mastocytosis (SM), highlighting findings from the APEX trial (NCT04996875) and commenting on its potential approval. He also provides insight into the safety profile of this agent, noting that no significant hematologic or non-hematologic adverse events were observed. This interview took place at the 31st Congress of the European Hematology Association (EHA) in Stockholm, Sweden.
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Transcript
So, bezuclastinib is a selective KIT inhibitor, and it’s being studied in both indolent or non-advanced SM in the SUMMIT trial, and in advanced systemic mastocytosis in the APEX trial. And at EHA, we’re going to present the pivotal data from the APEX in advanced systemic mastocytosis. What we saw was a very high overall response rate, both by the primary endpoint using the modified IWG criteria, as well as the pure pathologic response rate, or PPR...
So, bezuclastinib is a selective KIT inhibitor, and it’s being studied in both indolent or non-advanced SM in the SUMMIT trial, and in advanced systemic mastocytosis in the APEX trial. And at EHA, we’re going to present the pivotal data from the APEX in advanced systemic mastocytosis. What we saw was a very high overall response rate, both by the primary endpoint using the modified IWG criteria, as well as the pure pathologic response rate, or PPR. We saw a very impressive safety and durability so far, with some limited follow-up.
Bezuclastinib is being submitted for three indications, non-advanced SM, Summit trial, advanced SM, APEX trial, and recently the GIST trial that was presented at ASCO, the PEAK trial. I think it’s going to get approved based on very favorable efficacy and safety data. We did not see significant hematologic or non-hematologic adverse events. There was no intracranial bleeding, for example. This agent doesn’t cross the blood-brain barrier. There was a very low risk of hepatic adverse events, but other than that, patients were able, which is important, were able to stay on the dose that they were prescribed for a very long period of time. Bezuclastinib has a relatively benign safety profile. As mentioned, there was a slight increase in ALT, AST, or transaminitis, about 3%, had a grade 3 or higher. This was completely manageable with either dose interruption and rarely dose reduction, but we did not see a lot of dose discontinuation. Other than white hair, which is an on-target, off-organ effect, if you will, it inhibits wild-type KIT as well, and mild dysgeusia, or alteration in taste, there are very limited other off-target side effects. It does not cross blood-brain barriers. There was no cognitive decline. We did not see any bleeding issues. And because it doesn’t hit PDGFR alpha or beta, there was very limited edema, both periorbital and peripheral.
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