14 Nov, 2014 Positive Initial Phase 2 Data with Revusiran (ALN-TTRsc)
We announced positive initial data from our Phase 2 clinical trial with revusiran (ALN-TTRsc), an investigational RNAi therapeutic targeting transthyretin (TTR) for the treatment of TTR cardiac amyloidosis. Initial results from the pilot Phase 2 study demonstrated that revusiran was generally well tolerated in patients with significant disease burden. In addition, data showed an up to 98.2% knockdown of both mutant and wild-type forms of TTR – the disease-causing protein. As would be expected with the short treatment duration of five weeks, there were no significant changes observed in exploratory clinical measurements.
TTR cardiac amyloidosis represents a significant unmet medical need with no approved therapies. These new data further support our belief that revusiran holds promise as a potential new treatment option for patients.