Lead Sponsor

Alexion Pharmaceuticals, Inc.

Age

18 - 90 years

Sex

ALL

Healthy Volunteers

No

•Diagnosis of ATTR-CM with either wild-type or variant TTR genotype
•End-diastolic interventricular septal wall thickness ≥ 12 mm on echocardiography measured at Screening
•NT-proBNP \> 2000 pg/mL at Screening
•Treatment with a loop diuretic for at least 30 days prior to Screening
•History of heart failure NYHA Class II-IV at Screening
•Life expectancy of ≥ 6 months as per the Investigator's judgment
•Males and females of childbearing ability must use contraception

•Known leptomeningeal amyloidosis
•Known light chain (AL) or secondary amyloidosis (AA), or any other form of systemic amyloidosis
•Acute coronary syndrome, unstable angina, stroke, transient ischemic attack, coronary revascularization, cardiac device implantation, cardiac valve repair, or major surgery within 3 months of Screening
•Uncontrolled clinically significant cardiac arrhythmia, per Investigator's assessment
•LVEF \< 30% on echocardiography
•Renal failure requiring dialysis or an eGFR \< 20 mL/min/1.73 m2 at Screening
•Polyneuropathy with PND score IV

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