18 April 2018 - - Cambridge, Massachusetts-based RNA interference therapeutics developer Alnylam Pharmaceuticals, Inc. (NASDAQ: ALNY) has presented new results from the Phase 1 and Phase 1/2 open-label extension studies of givosiran at the European Association for the Study of the Liver 53rd Annual International Liver Congress, the company said.
Treatment with givosiran, an investigational RNAi therapeutic targeting aminolevulinic acid synthase 1 (ALAS1) for the treatment of acute hepatic porphyrias, led to rapid, dose-dependent, and durable lowering of induced ALAS1 mRNA in patients with recurrent attacks.
Lowering of ALAS1 resulted in corresponding reductions in both aminolevulinic acid (ALA), believed to be the primary neurotoxic intermediate responsible for disease manifestations, and porphobilinogen (PBG).
Compared with a once quarterly dose regimen, monthly dosing led to consistent and sustained lowering of ALA and PBG of greater than 80%, relative to baseline, mean reductions in annualized attack rate of 83%, and annualized hemin use of 88%, relative to placebo.
The Phase 1 study of givosiran (Part C) was conducted as a randomized, double-blind, placebo-controlled study in 17 patients with acute intermittent porphyria who experienced recurrent porphyria attacks. Following the treatment phase, all patients were eligible to receive givosiran in an open-label extension study.
Alnylam Pharmaceuticals is a biopharmaceutical company that discovers, develops, and commercializes novel therapeutics based on RNA interference. Its pipeline of investigational RNAi therapeutics is focused on genetic medicines, cardio-metabolic diseases, and hepatic infectious diseases.