Efficacy and Safety of AG10 in Subjects With Transthyretin Amyloid Cardiomyopathy

Inclusion Criteria:

• Have an established diagnosis of ATTR-CM with either wild-type TTR or variant TTRgenotype

• Have a history of heart failure evidenced by at least one prior hospitalization forheart failure or clinical evidence of heart failure without prior heart failurehospitalization manifested by signs or symptoms of volume overload or elevatedintracardiac pressures or heart failure symptoms that required or require ongoingtreatment with a diuretic.

• New York Heart Association (NYHA) Class I-III symptoms due to ATTR cardiomyopathy.

• On stable doses of cardiovascular medical therapy

• Completed ≥150 m on the 6MWT on 2 tests that are within 15% of total distance walkedprior to randomization

• Biomarkers of myocardial wall stress, NT-proBNP level ≥300 pg/mL at screening

• Have left ventricular wall (interventricular septum or left ventricular posteriorwall) thickness ≥12 mm

Exclusion Criteria:

• Had acute myocardial infarction, acute coronary syndrome or coronaryrevascularization, or experienced stroke or transient ischemic attack within 90 daysprior to screening

• Has hemodynamic instability

• Likely to undergo heart transplantation within a year of screening

• Confirmed diagnosis of primary (light chain) amyloidosis

• Biomarkers of myocardial wall stress, NT-proBNP level ≥8500 pg/mL at screening

• Measure of kidney function, eGFR by MDRD formula <15 mL/min/1.73 m2

• Current treatment with marketed drug products and other investigational agents forthe treatment of ATTR-CM

• Current treatment with calcium channel blockers with conduction system effects (e.g.verapamil, diltiazem). The use of dihydropyridine calcium channel blockers isallowed. The use of digitalis will only be allowed if required for management ofatrial fibrillation with rapid ventricular response