14 Oct, 2015 Pre-Clinical Data and Advancement of ALN-TTRsc02 for Transthyretin-Mediated Amyloidosis
We presented pre-clinical data with ALN-TTRsc02, an investigational RNAi therapeutic targeting transthyretin (TTR) for the treatment of TTR-mediated amyloidosis (ATTR amyloidosis). In pre-clinical studies, including those in non-human primates (NHPs), ALN-TTRsc02 achieved potent and highly durable knockdown of serum TTR of up to 99% with multi-month durability achieved after just a single dose, supportive of a potentially once quarterly dose regimen. Results from studies comparing TTR knockdown activity of ALN-TTRsc02 to that of revusiran showed that ALN-TTRsc02 has a markedly superior TTR knockdown profile. Further, in initial rat toxicology studies, ALN-TTRsc02 was found to be generally well tolerated with no significant adverse events at doses as high as 100 mg/kg.
Based on these pre-clinical data, ALN-TTRsc02 is the most potent and durable investigational RNAi therapeutic discovered at Alnylam to date. We believe that ALN-TTRsc02 has the potential to provide a best-in-class therapeutic option for the management of ATTR amyloidosis with a low volume, once quarterly subcutaneous dose regimen. We are completely committed to improving the lives of patients with ATTR amyloidosis and look forward to the advancement of patisiran, revusiran and ALN-TTRsc02, as we continue in our efforts to bring this patient population the best treatment options possible.