Results from Retrospective Natural History Study of Patients with Familial Amyloidotic Cardiomyopathy (FAC)

Results from Retrospective Natural History Study of Patients with Familial Amyloidotic Cardiomyopathy (FAC)

We presented results from a retrospective natural history study evaluating disease progression in transthyretin (TTR)-mediated amyloidosis (ATTR amyloidosis) patients with familial amyloidotic cardiomyopathy (FAC). These results were presented at a meeting with members of the Association of Black Cardiologists (ABC) at the American College of Cardiology (ACC) Annual Scientific Session, held March 14 – 16, 2015. Amongst other findings, study results showed a mean decline of 140 meters in 6-minute walk distance (6-MWD) over an 18-month period in FAC patients with mild-to-moderate heart failure. Also at the ACC meeting, we presented complete results from our Phase 2 clinical trial with revusiran, an investigational RNAi therapeutic for the treatment of FAC. Consistent with preliminary results presented last year, revusiran achieved an up to 98.2% knockdown of serum TTR – the disease-causing protein – and was found to be generally well tolerated.




The findings from the retrospective natural history analysis confirm the significant disease burden in patients with FAC. Further, they give us confidence that our ENDEAVOUR Phase 3 trial of revusiran in FAC patients is appropriately designed to show the potential impact of TTR lowering on functional decline. In addition, these data provide important context for our ongoing Phase 2 open-label extension study of revusiran in patients with TTR cardiac amyloidosis. We are hopeful that an RNAi therapeutic that stops production of the disease-causing protein has the potential to halt progression in patients with TTR cardiac amyloidosis and provide an important treatment option for management of this disease.



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