Transcript
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Dr. Hanna:
New long-term data around acoramidis as a treatment for transthyretin amyloid cardiomyopathy, or ATTR-CM, were just released at AHA 2024. What were these data, and how will it impact patient care?
This is ReachMD, and I'm Mazen Hanna.
So what is ATTR cardiomyopathy? This is an infiltrative cardiomyopathy due to the liver-derived protein transthyretin, or TTR, that dissociates from its tetrameric structure in misfolded monomers and amyloid fibrils that accumulate throughout the heart. The resulting thickening and stiffening of the heart leads to heart failure, atrial arrhythmias, and conduction disease, and the challenge in managing this condition is due to its progressive nature, which leads to worsening heart failure, recurrent hospitalizations, decreased functional capacity and quality of life, as well as decreased survival.
So what is acoramidis? Acoramidis is a transthyretin, or TTR, stabilizer that, by binding to TTR's tetrameric form, prevents the initial dissociation misfolding. And acoramidis provides near-complete stabilization with doses according to the ATTRibute-Cardiomyopathy protocol.
So ATTRibute-Cardiomyopathy was a randomized clinical trial for patients with ATTR-CM randomized to acoramidis or placebo, followed for 30 months with a hierarchical primary endpoint of all-cause mortality, CV-related hospitalizations, change in NT-ProBNP, and change in 6-minute walk distance. And the original trial was highly positive with statistically significant reductions in the primary endpoint, in addition to a 50% reduction in CV-related hospitalization.
Now, the open-label extension study that was presented at AHA took patients who had completed 30 months and agreed to the open-label extension and followed them out to 42 months. Those on acoramidis continued on acoramidis, and those on placebo switched to acoramidis. And the key open-label endpoints, primary was long-term safety and tolerability, and secondary was time to first event for all-cause mortality or first CV hospitalization.
And what this open-label extension study showed was a 34% relative risk reduction in all-cause mortality at month 42 that was highly statistically significant. Furthermore, there was a 41% risk reduction in CV-related hospitalization. Again, highly statistically significant. Furthermore, there were no new safety signals identified with acoramidis versus placebo.
So what are the clinical implications of ATTRibute-Cardiomyopathy and its open label extension study, and what does this mean for your patients?
The first is that, by stabilizing TTR with acoramidis, we can improve survival and reduce CV-related hospitalizations quite favorably. And the earlier and longer this treatment is given, the better the outcomes. As such, we emphasize that early intervention is needed for effective treatment, and early treatment depends on early diagnosis.
As I wrap up today, I would like my colleagues to keep amyloid heart disease on the differential diagnosis of patients presenting with symptoms of heart failure, atrial fibrillation, and conduction disease and to make every effort to pursue the diagnosis. Today, we have treatment options that can modify the disease progression and give our patients better lives. Now that acoramidis has been FDA-approved, we have an additional treatment option for this disease.
That's all the time we have today. Thank you for tuning in. I'm Dr. Mazen Hanna, and this has been ReachMD.
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