7 May 2018 - - Cambridge, Massachusetts-based RNA interference therapeutics developer Alnylam Pharmaceuticals, Inc. (NASDAQ: ALNY) has reached alignment with the US Food and Drug Administration on a pivotal study design for lumasiran, an investigational RNAi therapeutic for the treatment of primary hyperoxaluria type 1 (PH1), the company said.
The company and the FDA have agreed on a primary endpoint for the pivotal study based on reduction of urinary oxalate at six months, a biomarker directly linked to the pathophysiology of PH1 and known to be well correlated with disease progression, and on a study size of approximately 25 patients with PH1.
The ongoing Phase 1/2 Part B study is designed as a randomized, single-blind, placebo-controlled trial.
Lumasiran is designed to reduce the hepatic levels of the GO enzyme, thereby depleting the substrate necessary for oxalate production, which directly contributes to the pathophysiology of PH1. Lumasiran utilises Alnylam's enhanced stabilisation chemistry -GalNAc-conjugate technology, which enables subcutaneous dosing with increased potency and durability and a wide therapeutic index.
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.