Profile: Alnylam Pharmaceuticals Inc (ALNY.O)
Alnylam Pharmaceuticals, Inc. (Alnylam) is a biopharmaceutical company developing therapeutics based on ribonucleic acid interface (RNAi). Its Alnylam 5x15, is focused on the development and commercialization of novel RNAi therapeutics for the treatment of genetically defined diseases. Its core programs in clinical or pre-clinical development are: ALN-TTR for the treatment of transthyretin-mediated amyloidosis (ATTR); ALN-APC for the treatment of hemophilia; ALN-PCS for the treatment of severe hypercholesterolemia; ALN-HPN for the treatment of refractory anemia, and ALN-TMP for the treatment of hemoglobinopathies, including beta-thalassemia and sickle cell anemia. The Company has three partner-based programs in clinical or pre-clinical development, including ALN-RSV01 for the treatment of respiratory syncytial virus (RSV), infection, ALN-VSP for the treatment of liver cancers and ALN-HTT for the treatment of Huntington’s disease (HD). In March 2014, the Company acquired Merck's wholly owned subsidiary Sirna Therapeutics, Inc.
ALN-TTR TTR-Mediated Amyloidosis (ATTR)
The Company’s advanced core product development program, ALN-TTR, targets the transthyretin (TTR), gene for the treatment of ATTR. ATTR is a hereditary, systemic disease caused by a mutation in the TTR gene, of which over 100 mutations have been identified. TTR protein is produced primarily in the liver and is normally a carrier for thyroid hormone and retinol binding protein. The Phase1 clinical trial for ALN-TTR01 was conducted in Portugal, Sweden, the United Kingdom and France as a randomized, blinded, placebo-controlled, single-dose escalation study in up to 36 patients with ATTR.
ALN-APC is an RNAi therapeutic targeting protein C, a genetically defined target, for the treatment of hemophilia. Hemophilia is a hereditary disorder caused by genetic deficiencies of various blood clotting factors, resulting in recurrent bleeds into joints, muscles and other internal organs. ALN-APC provides a pharmacologic strategy to reproduce the human genetics observed with co-inheritance of prothrombotic factors in hemophilia.
ALN-PCS Severe Hypercholesterolemia
ALN-PCS is a systemically delivered RNAi therapeutic targeting proprotein convertase subtilisin/kexin type 9, or PCSK9, for the treatment of severe hypercholesterolemia. PCSK9 is involved in the regulation of low-density lipoprotein receptor (LDLR), levels on hepatocytes and the metabolism of LDL cholesterol (LDL-c), which is commonly referred to as bad cholesterol. PCSK9 is a protein that is produced by the liver and circulates in the bloodstream.
ALN-HPN Refractory Anemia
ALN-HPN is a systemically delivered RNAi therapeutic targeting the hepcidin pathway, specifically through transferrin receptor subtype 2 (TFR2), a genetically validated gene in iron homeostasis, for the treatment of refractory anemia. Anemia is the clinical manifestation of a decrease in circulating red blood cell mass and is usually detected by low blood hemoglobin concentrations. Symptoms include fatigue and dizziness. Anemia of chronic disease (ACD), occurs in patients with end-stage renal disease, cancer, chronic inflammatory disease and in certain genetic conditions.
ALN-RSV Respiratory Syncytial Virus (RSV) Infection
ALN-RSV is an RNAi therapeutic for the treatment of RSV infection. RSV is a contagious virus that causes infections in both the upper and lower respiratory tract. RSV infects nearly every child by the age of two years and is responsible for a percentage of hospitalizations of infants, children with lung or congenital heart disease, the elderly and adults with immune- compromised systems, including lung transplant recipients. It has formed collaborations with Cubist and Kyowa Hakko Kirin for the development and commercialization of RNAi products for the treatment of RSV.
ALN-VSP Liver Cancer
ALN-VSP is a systemically delivered RNAi therapeutic for the treatment of advanced solid tumors with liver involvement. In August 2011, the Company announced the completion of a Phase I clinical trial for ALN-VSP, which was its first delivered RNAi therapeutic to enter clinical development.
ALN-HTT Huntington’s Disease (HD)
The Company is developing ALN-HTT, a drug-device product incorporating an RNAi therapeutic candidate targeting the huntingtin gene, delivered using an implantable infusion device, for the treatment of HD. HD is an inherited and progressive brain disease that results in uncontrolled movements, loss of intellectual faculties, emotional disturbance and premature death.
The Company competes with Merck & Co., Inc., Sirna Therapeutics, Inc., Novartis, Takeda, Kyowa Hakko Kirin, Marina Biotech, Inc., Calando, Quark, Silence Therapeutics plc, Tekmira, Sylentis, Dicerna Pharmaceuticals, Inc., Rosetta Genomics, Santaris Pharma A/S, miRagen Therapeutics, Inc., Mirna Therapeutics, Inc. and Asuragen, Inc.
Alnylam Pharmaceuticals Inc
3rd Floor, 300 Third Street
CAMBRIDGE MA 02142