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EBMT 2025 | Agents of interest in systemic mastocytosis and trials to keep an eye out for
In this video, Deepti Radia, MBBS, BSc, MRCPI, FRCPath, MSc Med Ed, Guy’s and St Thomas’ NHS Foundation Trust, London, UK, outlines the trials to keep an eye out for in the field of systemic mastocytosis (SM). Dr Radia anticipates the results of the SUMMIT (NCT05186753) and APEX (NCT04996875) trials investigating bezuclastinib in indolent SM and advanced SM, respectively. She also shares her excitement over the combination therapies in the pipeline, including bezuclastinib plus azacitidine, and mentions the novel agent elenestinib. This interview took place at the 51st Annual Meeting of the EBMT in Florence, Italy.
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Transcript
So we know that there have been a few trials. Avapritinib has now been licensed based on the PATHFINDER trial. Bezuclastinib is currently being used, and both the Summit for the indolent patients and the APEX trial for the advanced patients are closed. So watch with bated breath to see what the outcome of those are going to be. They look promising, again with deep responses...
So we know that there have been a few trials. Avapritinib has now been licensed based on the PATHFINDER trial. Bezuclastinib is currently being used, and both the Summit for the indolent patients and the APEX trial for the advanced patients are closed. So watch with bated breath to see what the outcome of those are going to be. They look promising, again with deep responses.
Actually more interesting will be the combination trials. So bezuclastinib and azacitidine at the high-risk arm because we are looking at these two components. So, that would be quite interesting to see, because that seems like the natural way to go forward. And those are the current ones in the pipeline.
For the ISM patients, there is a BTK inhibitor with Telios which is being used to look for symptomatic improvement. And moving forward, there is elenestinib, which is the second cousin of avapritinib, if I can call it that. It doesn’t cross the blood-brain barrier for patients with indolent and smoldering disease going to open up. So more trials opening up in the near future.
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Disclosures
Blueprint Medicines/Cogent Biosciences.
