Cardiac amyloidosis future or investigational therapies

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Editor-In-Chief: C. Michael Gibson, M.S., M.D. [1]; Associate Editor(s)-in-Chief: Raviteja Guddeti, M.B.B.S. [2]; Aarti Narayan, M.B.B.S [3]

Overview

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.

Future or Investigational Therapies

Potential future treatment options targeting cardiac TTR amyloidosis include:

Patisiran:

  • 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

  • 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).

References

  1. Kristen AV, Ajroud-Driss S, Conceição I, Gorevic P, Kyriakides T, Obici L (February 2019). "Patisiran, an RNAi therapeutic for the treatment of hereditary transthyretin-mediated amyloidosis". Neurodegener Dis Manag. 9 (1): 5–23. doi:10.2217/nmt-2018-0033. PMID 30480471.
  2. Judge, Daniel P. (10/29/2019). "Transthyretin Stabilization by AG10 in Symptomatic Transthyretin Amyloid Cardiomyopathy". Check date values in: |date= (help)


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