Cardiac amyloidosis future or investigational therapies

Several investigational products targeting ATTR amyloid are being studied in clinical trials. These include small interfering RNA (siRNA) molecules which reduce the production of the amyloid precursor misfolded protein and ATTR stabilization molecules.

Potential future treatment options targeting cardiac TTR amyloidosis include:

• This is a siRNA molecule which has shown promise in patients with polyneuropathy from hereditary TTR amyloidosis. A cardiac subgroup study showed favorable biomarker results as well.^[1]

• AG10 is a selective, oral TTR stabilizer which mimics a protective TTR mutation known as T119M. In a phase 2 clinical trial, AG10 was found to be safe and effective. This trial randomized 49 patients with mutant or wild-type TTR amyloid cardiomyopathy with NYHA class II to III heart failure symptoms to AG10 400 mg, 800 mg, or placebo twice daily for 28 days, and found the medication to be well tolerated and to achieve near-complete stabilization of TTR.^[2] A phase 3 trial (ATTRIBUTE-CM) is ongoing (NCT03458130).

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