Alnylam Reports Initial Positive Results From ALN-AS1 Phase 1 Trial

(RTTNews) - Alnylam Pharmaceuticals Inc. (ALNY) reported initial positive results from its ongoing Phase 1 clinical trial with ALN-AS1, an investigational RNAi therapeutic targeting aminolevulinic acid synthase 1 for the treatment of acute hepatic porphyrias. The company said the study results showed that single subcutaneous doses of ALN-AS1 resulted in an up to 82% lowering of aminolevulinic acid and an up to 93% lowering of porphobilinogen.

ALN-AS1 treatment resulted in potent, dose-dependent, and durable silencing of aminolevulinic acid synthase 1 mRNA in liver. The company said ALN-AS1 was found to be generally well tolerated with no clinically significant drug-related adverse events to date.

The company has now transitioned to Part B of the Phase 1 study, in which subjects are receiving monthly subcutaneous dosing, and the company expects to initiate Part C in acute intermittent porphyria patients suffering from recurrent attacks in early 2016.