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A MyAmyloidosisTeam Member asked a question 💭
Forest Lake, MN
March 1, 2025
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Answer Summary

Members shared detailed information about ongoing depleter drug trials targeting cardiac amyloid deposits, clarifying that these treatments... Read more

Members shared detailed information about ongoing depleter drug trials targeting cardiac amyloid deposits, clarifying that these treatments work by making amyloid soluble and excretable rather than reversing deposits, with trials including Alexion ALXN2220, Novo Nordisk's Amitug, Attralus AT-02, Prothena's Birtamimab, and CAEL-101. Several members described their personal experiences with trial participation, noting strict eligibility requirements such as NT-proBNP levels above 2000 and no implanted devices, while one member shared being in an open-label trial for 18 months and feeling the treatment has helped them remain stable. A recurring theme was cautious optimism about these emerging therapies, with members acknowledging both the promise of new treatments and the reality that some trials have faced setbacks or shown mixed results.

A MyAmyloidosisTeam Member

I’m at the Mayo as I write and part of the trial that went to open label three month ago. In Dec of 2023 I was at Mayo stage 4 still here thinking the drug has benefited me and hopefully others.

April 30, 2025
A MyAmyloidosisTeam Member

Don't forget about Prothena's Birtamimab trial (formerly known as NEOD001). It is hoped that this drug will remove existing amyloid deposits from the body.

March 2, 2025
A MyAmyloidosisTeam Member

Don, yes, depleter drug trials are ongoing. There are 4 different depleter drug trials. The drugs do not "reverse" amyloid deposits. Three are monoclonal antibody type of infusion drugs that make the insoluble amyloid fibrils soluble (they target only the amyloids in the heart) and therefore, excretable. One is Alexion ALXN2220, another is a Novo Nordisk drug, Amitug (NNC6019-0001). It differs from all of the others.
It's designed to break down TTR amyloid deposits and prevent new amyloid from forming. The 3rd depleter drug is one I don't recall.

The 4th one, Attralus AT-02, is a humanized IgG1 monoclonal antibody that is genetically fused with the company's pan-amyloid binding peptide, that enables it to bind to multiple types of amyloid deposits. The AT-02 antibody stimulates the immune system to remove amyloid deposits that are bound by the AT-02. It basically uses macrophages and other immune system "warriors" to surround and then absorb and breakdown amyloid deposits everywhere and anywhere they are in the body.

March 1, 2025
A MyAmyloidosisTeam Member

Yes. Jury’s still out on CAEL-101. The current Phase 3 trial on Mayo IIIa patients is supposed to be completed in the next couple months. From what I’ve seen and heard, skepticism about its efficacy prevails. But, 🤞

March 3, 2025
MyAmyloidosisTeam

Yes, drug trials for reversing heart damage from amyloidosis are actively ongoing. A promising new drug called ALXN2220 has received FDA fast-track status for treating transthyretin amyloidosis with cardiomyopathy (ATTR-CM). Early trials have shown encouraging results, with significant reductions in amyloid buildup in the Show Full Answer

Yes, drug trials for reversing heart damage from amyloidosis are actively ongoing. A promising new drug called ALXN2220 has received FDA fast-track status for treating transthyretin amyloidosis with cardiomyopathy (ATTR-CM). Early trials have shown encouraging results, with significant reductions in amyloid buildup in the heart over a 12-month period. The drug is currently in phase 3 trials to evaluate its potential to extend life expectancy and reduce heart-related complications. What makes this treatment particularly exciting is that while existing treatments like tafamidis can slow disease progression, ALXN2220 may actually help remove proteins that have already been deposited in the heart, potentially reversing damage.

March 1, 2025

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