The purpose of this study was to evaluate the safety and efficacy of revusiran
(ALN-TTRSC) in patients with transthyretin (TTR) mediated Familial Amyloidotic
Cardiomyopathy. Dosing has been discontinued; patients are being followed-up for safety.

ENDEAVOUR: Phase 3 Multicenter Study of Revusiran (ALN-TTRSC) in Patients With Transthyretin (TTR) Mediated Familial Amyloidotic Cardiomyopathy (FAC)

Transthyretin Amyloidosis (ATTR)

Transthyretin Amyloidosis (ATTR) With Cardiomyopathy

Revusiran

Clinical Trial Site

A Phase 3 Multicenter, Multinational, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of ALN TTRSC in Patients With Transthyretin (TTR) Mediated Familial Amyloidotic Cardiomyopathy (FAC)

The difference between revusiran and placebo group in change from baseline to 18 months
in the total distance walked in 6 minutes

The difference between revusiran (ALN-TTRSC) and placebo group in the percent reduction
in serum TTR levels over 18 months

Number of cardiovascular-related deaths and cardiovascular-related hospitalizations in
the placebo group compared to the revusiran (ALN-TTRSC) treatment group

The difference between revusiran (ALN-TTRSC) and placebo group in the change from
baseline to 18 months in the NYHA class

The difference between revusiran (ALN-TTRSC) and placebo group in the change from
Baseline to 18 months in the Kansas City Cardiomyopathy Questionnaire

Number of cardiovascular-related deaths in the placebo group compared to the revusiran
(ALN-TTRSC) treatment group

Number of cardiovascular-related hospitalizations in the placebo group compared to the
revusiran (ALN-TTRSC) treatment group

Total number of deaths in the placebo group compared to the revusiran (ALN-TTRSC)
treatment group

administered by subcutaneous (SC) injection