ASH 2022 | Updates from a Phase II study of bomedemstat for the treatment of essential thrombocythemia
Harinder Gill, MBBS, PDipID, MD, MRCP, FRCP, FRCPath, FHKCP, FHKAM, The University of Hong Kong, Hong Kong, China, shares some insights into a Phase II study evaluating the safety and efficacy of bomedemstat for the treatment of essential thrombocythemia (ET; NCT04254978). Dr Gill first highlights the rationale behind targeting LSD1 in ET, and then discusses the efficacy of this agent, and molecular responses observed in patients. This interview took place at the 64th ASH Annual Meeting and Exposition congress held in New Orleans, LA.
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Transcript (edited for clarity)
So, for this particular study, this is one of the novel agents, exciting novel agents that we’ve mentioned. Bomedemstat is an LSD1 inhibitor, and LSD1 is over-expressed in the malignant megakaryocytes. And these malignant megakaryocytes are responsible for all these downstream production of various factors that results in the disease phenotype that we see in essential thrombocythemia. Not only the symptoms, but also the risk of disease progression and the quite prominent megakaryopoiesis...
So, for this particular study, this is one of the novel agents, exciting novel agents that we’ve mentioned. Bomedemstat is an LSD1 inhibitor, and LSD1 is over-expressed in the malignant megakaryocytes. And these malignant megakaryocytes are responsible for all these downstream production of various factors that results in the disease phenotype that we see in essential thrombocythemia. Not only the symptoms, but also the risk of disease progression and the quite prominent megakaryopoiesis.
So this particular agent in this study was a Phase II study with a daily dosing of bomedemstat and the primary endpoint assessment, all our patients achieved a platelet control with a target of 400 or below, so it’s very effective. And the important thing about this study is we also observed significant molecular responses in our patients. And so that’s an impressive thing about this study. It’s basically perfect responses as well as evidence of disease modification. So we’re eagerly awaiting for future Phase III studies to prove the efficacy of this particular agent, and I think it’s a very exciting agent that we are all looking forward to.
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