Alnylam Files Clinical Trial Application to Initiate a Phase 1 Study for ALN-AS1, a Subcutaneously Administered, Investigational RNAi Therapeutic Targeting Aminolevulinic Acid Synthase-1 (ALAS-1) for the Treatment of Hepatic Porphyrias

Alnylam Pharmaceuticals, Inc. (Nasdaq:ALNY), a leading RNAi therapeutics company, announced today that it has filed a Clinical Trial Application (CTA) with the Swedish Medical Products Agency (MPA) to initiate a Phase 1 clinical trial with ALN-AS1, a subcutaneously administered investigational RNAi therapeutic targeting aminolevulinic acid synthase 1 (ALAS-1) for the treatment of hepatic porphyrias, including acute intermittent porphyria (AIP). Per the filed CTA, the Phase 1 trial of ALN-AS1 will be performed first in AIP patients who are asymptomatic “high excreters” (ASHE) – patients with a mutation in the porphobilinogen deaminase (PBGD) gene and elevated urinary aminolevulinic acid (ALA) and porphobilinogen (PBG) levels, but no recent symptoms of a porphyria attack – and then in AIP patients who experience recurrent porphyria attacks. Following approval of the CTA, the company expects to initiate the Phase 1 study in mid-2015, with initial data expected to be reported in early 2016. ALN-AS1 now becomes the company’s fifth clinical stage program in its Genetic Medicine Strategic Therapeutic Area (STAr) and the sixth clinical pipeline program overall. [ABSTRACT FROM PUBLISHER]