Profile: Alnylam Pharmaceuticals Inc (ALNY.O)
1 Jul 2016
Alnylam Pharmaceuticals, Inc., incorporated on May 8, 2003, is a biopharmaceutical company. The Company is engaged in developing therapeutics based on ribonucleic acid (RNA) interference (RNAi). The Company is focused on the use of its N-acetylgalactosamine (GalNAc)-conjugate strategy for delivery of small interfering RNAs (siRNAs). Its pipeline of investigational RNAi therapeutics focuses in three Strategic Therapeutic Areas (STArs): Genetic Medicines, with a pipeline of RNAi therapeutics for the treatment of rare diseases; Cardio-Metabolic Disease, with a pipeline of RNAi therapeutics toward genetically validated, liver-expressed disease in cardiovascular and metabolic diseases, such as dyslipidemia, hypertension, non-alcoholic steatohepatitis (NASH) and type II diabetes, and Hepatic Infectious Disease, with a pipeline of RNAi therapeutics for the treatment of hepatic infectious diseases.
The Company's Genetic medicines include Patisiran (ALN-TTR02), Revusiran (ALN-TTRsc), Fitusiran (ALN-AT3), ALN-CC5, ALN-AS1, and additional genetic programs, including ALN-AAT, ALN-GO1 and ALN-TMP. Products for Cardio-Metabolic Disease STAr include ALN-PCSsc and other cardio-metabolic disease programs ALN-AC3 and ALN-ANG targeting Angiopoetin-like 3 (ANGPTL3). Products for Hepatic Infectious Disease STAr include ALN-HBV, ALN-HDV and ALN-PDL. The Company also owns Enhanced Stabilization Chemistry (ESC), GalNAc-conjugate delivery.
The Company is developing Patisiran (ALN-TTR02) for the treatment of TTR-Mediated Amyloidosis ((ATTR Amyloidosis). The Company is conducting APOLLO Phase III clinical trial of patisiran. The APOLLO Phase III clinical trial is a randomized, double-blind, placebo-controlled, global study designed to evaluate the efficacy and safety of patisiran in ATTR patients with familial amyloidotic polyneuropathy (FAP). The Company is also conducting a Phase II OLE study to evaluate the safety and tolerability of Patisiran administration.
The Company is developing Revusiran (ALN-TTRsc) using its STC-GalNAc-conjugate-siRNA delivery platform enabling subcutaneous dose administration for the treatment of TTR-Mediated Amyloidosis (ATTR). The Company is conducting ENDEAVOUR Phase III clinical trial of revusiran in TTR-mediated familial amyloidotic cardiomyopathy. The ENDEAVOUR trial is a randomized, double-blind, placebo-controlled study designed to evaluate the efficacy and safety of revusiran in patients with Familial amyloid cardiomyopathy (FAC). The Company is also conducting a Phase II OLE study, which is designed to evaluate the tolerability of revusiran administration in ATTR amyloidosis patients with cardiomyopathy.
ALN-AT3 is an investigational RNAi therapeutic for the treatment of Hemophilia and Rare Bleeding Disorders (RBD). Fitusiran utilizes the Company's ESC-GalNAc-siRNA conjugate delivery platform. The Company is focused on initiating two Phase III program clinical trials in severe Hemophilia A (HA) and Hemophilia B (HB) patients.
ALN-CC5 is an investigational RNAi therapeutic for the treatment of complement-mediated diseases. ALN-CC5 utilizes its ESC-GalNAc conjugate technology. The Company's is evaluating ALN-CC5 in an ongoing Phase I/II study, which is being conducted in three parts: Part A, Part B and Part C. Parts A and B are randomized (3:1, drug:placebo), double-blind, placebo-controlled, single ascending dose (SAD) and multiple ascending dose (MAD) studies, respectively. Part C is an open-label, multi-dose study into be done in approximately 20 patients with paroxysmal nocturnal hemoglobinuria (PNH).
ALN-AS1 is a subcutaneously administered, investigational RNAi therapeutic for the treatment of hepatic porphyrias. ALN-AS1 utilizes the Company's ESC-GalNAc conjugate technology. It is evaluating ALN-AS1 in an ongoing Phase I study being conducted in three parts. Parts A and B are placebo-controlled, randomized (3:1, drug:placebo), single-blind, single-dose (Part A) and multi-dose (Part B), dose-escalation studies.
Additional Genetic Medicine Programs
ALN-AAT is a subcutaneously administered, investigational RNAi therapeutic targeting alpha-1 antitrypsin (AAT), for the treatment of AAT deficiency-associated liver disease, also known as alpha-1 liver disease. AAT deficiency is an inherited disorder that results in disease of the lungs and liver. The Company is evaluating ALN-AAT in a randomized, single-blind, placebo-controlled Phase I/II clinical trial being conducted in three parts. Parts A and B are single-dose (Part A) and multi-dose (Part B), dose-escalation studies.
ALN-PCSsc is a subcutaneously administered investigational RNAi therapeutic targeting proprotein convertase subtilisin/kexin type 9 (PCSK9) for the treatment of hypercholesterolemia. The Company has completed Phase I clinical trial with ALN-PCSsc in the United Kingdom as a randomized, single-blind, placebo-controlled, single ascending- and multi-dose study. The Company, through a license and collaboration agreement with The Medicines Company, has initiated a randomized, double-blind, placebo-controlled Phase II clinical study of ALN-PCSsc, known as ORION-1.
Additional Cardio-Metabolic Disease Programs
The Company's ALN-AC3, targeting Apolipoprotein C3, is being developed for the treatment of hypertriglyceridemia. Its ALN-ANG, targeting Angiopoetin-like 3, is being developed for the treatment of hypertriglyceridemia and mixed hyperlipidemia. In addition, the Company is developing ALN-AGT targeting angiotensinogen (AGT) for the treatment of hypertensive disorders of pregnancy, including preeclampsia.
The ALN-HBV Development Candidate program utilizes ESC-GalNAc-siRNA conjugate delivery platform and targets the Hepatitis B Virus (HBV) genome. The Company is focused on filing an IND for this program.
Additional Hepatic Infectious Disease Programs
The Company is also developing early-stage Hepatic Infectious Disease programs, including ALN-HDV and ALN-PDL. ALN-HDV is an investigational RNAi therapeutic targeting the hepatitis delta virus (HDV) for the treatment of HDV infection. ALN-PDL is an investigational RNAi therapeutic targeting hepatocyte-expressed programmed death ligand 1 (PD-L1), an immune checkpoint inhibitor, for the treatment of chronic liver infections.
The Company competes with Takeda Pharmaceutical Company Limited, Kyowa Hakko Kirin Co., Ltd., Marina Biotech, Inc., Arrowhead, Calando Pharmaceuticals, Inc., Quark Pharmaceuticals, Inc., Silence Therapeutics plc, Arbutus Biopharma Corporation, Sylentis, S.A.U., Dicerna Pharmaceuticals, Inc., Wave Life Sciences, Arcturus Therapeutics, Benitec Ltd, Regulus Therapeutics Inc., Rosetta Genomics, Roche, miRagen Therapeutics, Inc., Mirna Therapeutics, Inc., Asuragen, Inc., Pfizer, GlaxoSmithKline, Ionis Pharmaceuticals, Inc., Bayer Healthcare Pharmaceuticals, CSL Behring, Novo Nordisk, Biogen Idec Inc., Celgene, Swedish Orphan Biovitrium, Shire, Apellis Pharmaceuticals, Recordati S.p.A, UniQure, Digna Biotech, Aegerion Pharmaceuticals, Inc., Regeneron Pharmaceuticals, Inc., Sanofi, Amgen Inc., Esperion Therapeutics, Boston University, Antisense Therapeutics, Ltd., Sarepta Therapeutics, Inc. and Alexion Pharmaceuticals.
Alnylam Pharmaceuticals Inc
300 3rd St
CAMBRIDGE MA 02142-1103
Company Web Links
- BRIEF-Alnylam Pharmaceuticals names David-Alexandre Gros Principal Financial Officer
- BRIEF-Bioxcel Corp says begins collaboration with Alnylam to discover novel RNAi therapeutic products
- U.S. RESEARCH ROUNDUP-International Paper, United Technologies, Republic Services
- BRIEF-Alnylam Pharmaceuticals reports Q1 GAAP loss per share $1.21 including items