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ESC 2026 | Emerging therapeutic strategies and clinical trials in ATTR-CM
Ronald Witteles, MD, Stanford University, Stanford, CA, discusses the evolving treatment landscape for transthyretin amyloid cardiomyopathy (ATTR-CM). Dr Witteles highlights investigational approaches including TTR-lowering agents, as well as emerging amyloid-depleting agents designed to remove existing deposits, emphasizing that these strategies remain under clinical evaluation. This interview took place during the 2026 European Society of Cardiology (ESC) Congress in Munich, Germany.
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Transcript
So the clinical trial space remains extremely active in this disease. I would put it broadly into the agents that are knocking down TTR production. We talked about them already, the two furthest along, that’s nucresiran and what’s called nex-z, which is the CRISPR. And then agents that are designed as so-called depleters to remove amyloid deposits. And so there’s two current large Phase III trials with two different depleter agents...
So the clinical trial space remains extremely active in this disease. I would put it broadly into the agents that are knocking down TTR production. We talked about them already, the two furthest along, that’s nucresiran and what’s called nex-z, which is the CRISPR. And then agents that are designed as so-called depleters to remove amyloid deposits. And so there’s two current large Phase III trials with two different depleter agents. I would say depleters are extremely attractive to patients and many clinicians because the idea that all that our therapies can do is greatly slow or maybe even, for all practical purposes, halt ongoing TTR deposits, but we’re not doing anything about those that are there, people are disappointed that we can’t do more. I will say my own opinion is that the early phase data of depleters is interesting, but far from overwhelmingly compelling. So I could actually believe any outcome from the Phase III trials. I could believe that they will be very important parts of our future therapeutic landscape, and I could believe the trials will fail. So, okay, well, that’s why we do trials to find out. So I’ll be excited to ultimately to see those, as well as the other two trials I mentioned. None of them are going to be imminently reporting out though, so we have a little bit more time.
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