Transthyretin

There's other stabilizers in this disease.

So there's an entire category of medicines that has now emerged around TTR amyloid cardiomyopathy based on the work that we did there, based on the work that we did there at the time.

So it's exciting to watch this field develop.

I mean, I would envision that there are other cardiovascular diseases that would develop in the same way.

And genetic testing is becoming routinely sitting behind many cardiovascular diseases.

And for example, there's one form of amyloid that is a genetically mediated form of cardiac disease, as an example.

And so therefore, there are genetic tests available to diagnose that component of TTR amyloid.

So we believe that will begin to be the case across a broad range of cardiovascular diseases.

Transthyretin amyloid cardiomyopathy in Japan.

Patient characteristics and the diagnosis of transthyretin amyloid cardiomyopathy in Japan.

The final original research article presents findings from a nationwide Japanese registry of patients with transthyretin amyloid cardiomyopathy.

So as you had mentioned, you know, there are different types of cardiac amyloid.

Um, there's the ATTR, uh, amyloid, which is the one that people are more familiar with and cardiologists see more often.

Uh, and then the one that are less commonly seen by cardiologists but also we think is vitally important is AL amyloid, uh, which is a disorder caused by light chain production.

You know, the light chains are, uh, made in the bone marrow, and when there is a, uh, plasma cell dyscrasia, the bone marrow are making too many, uh, light chains, and these light chains can then misfold and aggregates into amyloids, which then deposit in different tissues and organs.

So it's very, very important that we recognize this and also treat this promptly.

Great.

In contrast to ATTR or wild type amyloid, which many people are familiar is now treatable with tafamidis and other similar class drugs, you're tackling a subgroup for which therapy really is lacking for cardiac involvement.

And to do that, you tested a novel approach which involved the administration of a monoclonal antibody.

This week is the first of many ESC recap shows.

I'll do the single AF trial, CMR guide trial, two ATTR cardiomyopathy studies, PVI sham AF, STAR-E, and I think an important observational study of ICD benefit over the past decades.

First of all, it was great to see so many listeners.

I met someone, my first person from Russia that I've ever met, and that was cool.

And I think it begins, begins to disprove the theory about scar and ICD benefit.

We definitely need more studies to test this theory that a scar identifies people that an ICD will be useful for.

All right, next topic is a single versus dual therapy in ATTR cardiomyopathy.

Now, two studies from ESC informed a curious question of using two agents in these patients with ATTR cardiomyopathy.

The extreme, asymmetric risk-reward driving these targets is just remarkable when you break it down.

In our 55% base case, projecting out to year 5 in 2031, we assume LAMPA-Z captures just 20% of the treated US HAE market and NEX-C launches successfully in 2029 for ATTR.

Assuming they hit a 25% net margin by then, and applying a conservative 25X exit multiple, that trials a price target of $51.39.

But look what happens in the 25% high case.

Together, we're advancing the knowledge that defines excellence in cardiac care while celebrating the difference you make every day.

I'd like to welcome our audience to today's episode where we are going to be discussing trans-thyretin, amyloid cardiomyopathy, or ATTR.

Today's guest is Morgan Lewis.

Morgan, could you introduce yourself, please?

you so much.

We're looking forward to learning from you more about this disease that might not be as rare as we once thought.

And I'm wondering if you could start us off talking about ATTR just in general.

What is it? What does an ATTR diagnosis mean for patients?