Alnylam reports Phase I data for subcutaneous RNAi therapeutic

Alnylam Pharmaceuticals Inc. (NASDAQ:ALNY) reported top-line data from a single-blind, U.K. Phase I trial in 80 healthy volunteers showing that single ascending doses of 5-300 mg subcutaneous ALN-TTRsc02 led to "robust" knockdown of transthyretin (TTR) that was maintained over 320 days. Mean maximum TTR knockdown with the TTR-mediated amyloidosis candidate was up to 97%. Alnylam said data on sustained TTR knockdown in the trial combined with modeling data support a once-quarterly 25 mg dose of the product. No serious adverse events were reported.

The trial’s primary endpoint is safety. Secondary endpoints include pharmacokinetics and serum TTR and vitamin A levels...