Profile: Alnylam Pharmaceuticals Inc (ALNY.O)
17 Aug 2017
Alnylam Pharmaceuticals, Inc., incorporated on May 8, 2003, is a biopharmaceutical company. The Company is engaged in the discovery, development and commercialization of ribonucleic acid (RNA) interference (RNAi) therapeutics. The Company is focused on the use of its N-acetylgalactosamine (GalNAc)-conjugate platform for delivery of small interfering RNAs (siRNAs). Its pipeline of investigational RNAi therapeutics focuses on three Strategic Therapeutic Areas (STArs): Genetic Medicines, with multiple product candidates for the treatment of rare diseases; Cardio-Metabolic Diseases, with product candidates directed toward genetically validated, liver-expressed disease targets for unmet needs in cardiovascular and metabolic diseases, and Hepatic Infectious Diseases, with product candidates designed to address the global health challenges of hepatic infectious diseases, beginning with hepatitis B and hepatitis D viral infections. Its lead product, Patisiran, is formulated utilizing lipid nanoparticles (LNPs).
The Company's Genetic Medicines include Patisiran, Fitusiran, Givosiran, ALN-CC5, ALN-GO1 and ALN-TTRsc02. Its product for Cardio-Metabolic Diseases is Inclisiran (ALN-PCSsc). Its product for Hepatic Infectious Diseases is ALN-HBV. The Company's investigational RNAi therapeutic in development, Patisiran, targets the transthyretin (TTR), gene for the treatment of patients with polyneuropathy due to hereditary TTR-mediated amyloidosis (hATTR amyloidosis). The Company is conducting APOLLO Phase III clinical trial for Patisiran. The Company is also conducting Phase II Open label extension (OLE) study for Patisiran. Fitusiran is a subcutaneously administered, investigational RNAi therapeutic targeting antithrombin (AT), for the treatment of hemophilia A (HA) and hemophilia B (HB) and rare bleeding disorders (RBD). The Company is evaluating Fitusiran in an ongoing Phase I study in HA and HB patients, with and without inhibitors, as well as in a Phase II OLE study. Fitusiran has received orphan drug designation for HA and HB in the United States and the European Union.
Inclisiran is a subcutaneously administered, investigational RNAi therapeutic targeting proprotein convertase subtilisin/kexin type 9 (PCSK9), for the treatment of hypercholesterolemia. The Company is conducting certain pre-clinical studies, as well as the Phase I clinical study for Inclisiran. The Company is also conducting ORION-1, an ongoing Phase II clinical trial of Inclisiran. Givosiran is a subcutaneously administered, investigational RNAi therapeutic targeting aminolevulinate synthase-1 (ALAS-1), for the treatment of acute hepatic porphyrias (AHPs). The Company is evaluating Givosiran in an ongoing Phase I study. Givosiran has received orphan drug designation in the United States and the European Union for the treatment of AHPs. ALN-CC5 is a subcutaneously administered, investigational RNAi therapeutic targeting the C5 component of the complement pathway for the treatment of complement-mediated diseases. The Company has completed a Phase I/II clinical trial for ALN-CC5.
ALN-GO1 is a subcutaneously administered, investigational RNAi therapeutic, for the treatment of primary hyperoxaluria type 1 (PH1). The Company is conducting dosing in a randomized, single-blind, placebo-controlled Phase I/II clinical trial for ALN-GO1. ALN-GO1 has been granted orphan drug designation in the United States and the European Union for the treatment of PH1. ALN-TTRsc02 is a subcutaneously administered, investigational RNAi therapeutic targeting TTR for the treatment of ATTR amyloidosis. The Company is conducting a Phase I randomized, placebo-controlled and single ascending-dose study in healthy volunteers for ALN-TTRsc02. ALN-HBV is a subcutaneously administered, investigational RNAi therapeutic, targeting the hepatitis B virus, genome for the treatment of chronic hepatitis B. The Company is conducting a Phase I/II randomized, single-blind and placebo-controlled study for ALN-HBV.
The Company competes with Takeda Pharmaceutical Company Limited, Marina Biotech, Inc., Arrowhead Research Corporation, Quark Pharmaceuticals, Inc., Silence Therapeutics plc, Arbutus Biopharma Corporation, Sylentis, S.A.U., Benitec Biopharma Ltd., Dicerna Pharmaceuticals, Inc., WAVE Life Sciences Ltd., Arcturus Therapeutics, Inc., Regulus Therapeutics, Inc., Rosetta Genomics Ltd., F. Hoffmann-La Roche Ltd, miRagen Therapeutics, Inc., Mirna Therapeutics, Inc., Asuragen, Inc., Celgene Corporation, Antisense Therapeutics, Ltd., Sarepta Therapeutics, Inc., Pfizer Inc., Ionis Pharmaceuticals, Inc., GlaxoSmithKline plc, Shire plc, Bayer Healthcare Pharmaceuticals, CSL Behring, Biogen Inc., Novo Nordisk, Spark Therapeutics Inc., uniQure N.V., Dimension Therapeutics, Inc., BioMarin Pharmaceutical Inc., Aegerion Pharmaceuticals, Inc., Regeneron Pharmaceuticals, Inc., Sanofi, Amgen Inc., Recordati S.p.A., Digna Biotech and Alexion Pharmaceuticals, Inc.
Alnylam Pharmaceuticals Inc
300 3rd St
CAMBRIDGE MA 02142-1103
Company Web Links
- BRIEF-Alnylam Pharmaceuticals reports Q2 adj loss per share $1.07
- BRIEF-Alnylam Pharmaceuticals and Sanofi Genzyme report positive results from ongoing phase 2 open-label extension study
- BRIEF-Alnylam and Sanofi Genzyme initiate ATLAS Phase 3 program with investigational RNAi therapeutic fitusiran
- BRIEF-Alnylam Pharmaceuticals reports results for Givosiran
- BRIEF-Alnylam CEO, John Maraganore, named BIO chair for 2017-2018 term