Alnylam Reports Positive Interim Results From Phase I Trial Of ALN-TTRsc

(RTTNews) - Alnylam Pharmaceuticals Inc. (ALNY), a RNAi therapeutics company, announced positive interim results from its Phase I clinical trial of ALN-TTRsc, a subcutaneously delivered RNAi therapeutic targeting the transthyretin, or TTR, gene for the treatment of, TTR-mediated amyloidosis or ATTR.

The company said the results showed that ALN-TTRsc administration led to consistent and statistically significant knockdown of serum TTR protein levels of up to 94%. In addition, knockdown of TTR, the disease causing protein in ATTR, was found to be rapid, dose dependent, and durable. To date, ALN-TTRsc has been found to be generally safe in the study, the company noted.

Akshay Vaishnaw, EVP and Chief Medical Officer of Alnylam, said: "These new data support our belief that ALN-TTRsc has the potential to be an important therapeutic for the treatment of familial amyloidotic cardiomyopathy. We are well positioned for continued execution on this program, which includes the initiation of a pilot Phase II study in FAC patients by the end of this year, and - assuming positive results - start of a pivotal Phase III trial with ALN-TTRsc by the end of 2014."

In 2012, Alnylam entered into an exclusive alliance with Genzyme, a Sanofi company, to develop and commercialize RNAi therapeutics, including ALN-TTR02 and ALN-TTRsc, for the treatment of ATTR in Japan and the broader Asian-Pacific region.