CAMBRIDGE, Mass.--(BUSINESS WIRE)--Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, announced today initial positive results from its ongoing Phase 1 clinical trial with ALN-AS1, an investigational RNAi therapeutic targeting aminolevulinic acid synthase 1 (ALAS1) for the treatment of acute hepatic porphyrias. The Phase 1 study is being performed in asymptomatic “high excreter” (ASHE) patients, who carry the genetic mutation of acute intermittent porphyria (AIP) and have elevated levels of aminolevulinic acid (ALA) and porphobilinogen (PBG), the toxic heme intermediates that mediate porphyria attacks. Study results showed that single subcutaneous doses of ALN-AS1 resulted in an up to 82% lowering of ALA and an up to 93% lowering of PBG, providing human proof-of-concept for this investigational RNAi therapeutic as a potential therapy for AIP and other acute hepatic porphyrias. In addition, through analysis of exosomal mRNA preparations from serum and urine, it was demonstrated that ALN-AS1 treatment resulted in potent, dose-dependent, and durable silencing of ALAS1 mRNA in liver. Further, 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 AIP patients suffering from recurrent attacks in early 2016. In addition, Alnylam presented initial data from the EXPLORE trial, a multinational, prospective observational study of patients with hepatic porphyrias suffering from recurrent attacks.
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