Alnylam (ALNY) is a super-innovator in a sector of innovators. I love the company but I dislike the stock for the reason that they are developing an unproven technology. Alnylam is one of the few biotech companies working in the newest and most challenging class of therapeutics; RNAi. Unlike small molecules and protein therapeutics, RNAi based drugs work at the gene level and have the potential to be more potent and more specific. Its principal drug candidate comprises ALN-RSV01, a phase I clinical stage product for the treatment of lung infections caused by respiratory syncytial virus. The company also engages in the research and development of other drug candidates for the treatment of various diseases, including pandemic flu, cystic fibrosis, neuropathic pain, spinal cord injury, Parkinson's disease, Huntingto's disease, ocular diseases, and other diseases. Alnylam Pharmaceuticals has alliances with Merck & Co., Inc.; Novartis AG; and Medtronic, Inc. for the development and commercialization of various RNA interference system products. It also has a cooperative research and development agreement with the U.S. Army Medical Research Institute of Infectious Diseases to discover RNAi therapeutics targeting viral organisms, including hemorrhagic fever viruses. The company has collaboration agreement with University of Texas Southwestern Medical Center at Dallas to evaluate approaches for reducing LDL-cholesterol levels using RNAi therapeutics directed to a disease target, called proprotein convertase subtilisn/kexin type 9. Alnylam also has collaboration agreement with Inex Pharmaceuticals Corp. for the systemic delivery of RNAi therapeutics; and with Biogen Idec, Inc. to develop a treatment for neurological disorders. The company was founded in 2002 and is headquartered in Cambridge, Massachusetts.
I don't see a blockbuster in the RSV product. Alliances with other pharmaceutical companies are nice but those companies could withdraw at any point. The biodefense contracts are there only to provide some cash to support the rest of the business and should not be considered a continued source of funds.The company has approximately $115 million in cash and valued at $530 million. The stock is trading at $16.5, near an all time high. With the uncertainty around its technology and its only clinical candidate in early phase I, it is highly probable that the company will need to raise cash in the future again. At this point, ALNY is very speculative and will be volatile for many years. I have it on a watch list and recommend a "Don't buy" at this point.