Alnylam Pharmaceuticals

History

In 2002, Alnylam was founded by scientists Phillip Sharp, Paul Schimmel, David Bartel, Thomas Tuschl, and Phillip Zamore, and by investors Christoph Westphal and John Kennedy Clarke; John Maraganore was the founding CEO. The company was named after Alnilam, a star in Orion’s belt. The spelling was modified to make it unique. In 2003, Alnylam merged with the German pharmaceutical company, Ribopharma AG. The newly formed company also received $24.6 million in funding from private-equity firms. On February 27, 2004, Alnylam Pharmaceuticals filed for an IPO. The company raised $26 million and began trading as ALNY on the Nasdaq stock exchange.

In 2005, the company partnered with Medtronic to develop drug-device combinations to treat neurodegenerative disorders. Alnylam partnered with Biogen Idec to discover and develop potential treatments of progressive multifocal leukoencephalopathy in 2006. In July 2007, Alnylam entered into a nonexclusive alliance with the Swiss drug company, Hoffmann-La Roche, in which Alnylam received $331 million upfront in exchange for access to its technology platform. In September 2007, Alnylam partnered with Isis Pharmaceuticals to found the company Regulus Therapeutics, a company focused on discovery, development, and commercialization of microRNA therapeutics.

In 2009, Alnylam formed alliances with Cubist Pharmaceuticals and Kyowa Hakko Kirin to market a drug developed to treat the respiratory syncytial virus. Alnylam expanded its previous collaboration with Medtronic to include the CHDI Foundation in its Huntington's Disease focused research in 2010. In 2011, Alnylam and GlaxoSmithKline partnered together to develop RNAi technology enhancing vaccine production. Alnylam entered into a 10-year alliance with Monsanto in 2012, in order to develop biotech solutions for the farming industry by using natural molecules and developing them for use in crop protection. In October 2012, the company formed a partnership with Sanofi Genzyme to develop and commercialize a treatment for transthyretin-mediated amyloidosis, a hereditary disease in Asia. In February 2013, Alnylam formed a partnership with The Medicines Company to develop and commercialize a drug to treat a genetic form of high cholesterol.

In July 2013, during a Phase I trial Alnylam demonstrated statistically significant reduction of a protein called transthyretin, or TTR and demonstrated human efficacy with intravenous and subcutaneous modes of administration. In 2014, Sanofi Genzyme acquired a 12 percent stake in Alnylam and increased its rights to several of the company's drugs for $700 million. In a separate transaction Alnylam announced that it had purchased Merck & Co.'s Sirna Therapeutics, for $25 million cash and $150 million in stock.

In 2016, the company purchased land in Norton, Massachusetts to build a manufacturing facility.

Therapeutic concept

Most drug discovery seeks molecules which associate with a "target" associated with a disease and interfere with its function, thus remediating the disease at a molecular level. Examples include small molecule HIV protease inhibitors, which are taken orally, and via the bloodstream, make their way to cells infected with the HIV virus, and block a key enzyme, virally encoded enzyme, HIV protease, by occupying its active site, thereby refusing access of a key viral component, and preventing a key step in the HIV virus life cycle (scission of the HIV polyprotein).

A different approach targets the production of a protein required for the development of the disease. RNAi was discovered to be a natural mechanism for silencing genes through cleavage and degradation of mRNA, first discovered in C. elegans in 1998 by Andrew Fire and Craig Mello. Genes encode the information necessary for cells to synthesize proteins, and proteins made abnormally cause many human diseases. When a mutant gene is silenced, the cell stops making the abnormal protein specified by that gene, potentially improving the course of the disease. In 2001, Alnylam founders began using small interfering RNAs, known as siRNAs, to silence genes by targeting mRNA in mammalian cells.

Products

In 2016, Alnylam Pharmaceuticals had 18 potential treatments in various development stages across three Strategic Therapeutic Areas (STArs), Genetic Medicine, Cardio-Metabolic Disease and Hepatic Infectious Disease. In 2016, the company had two candidates in phase III studies, patisiran, a treatment targeting transthyretin (TTR) for the treatment of TTR-mediated amyloidosis (ATTR), in patients with familial amyloidotic polyneuropathy (FAP) and revusiran, for the treatment of familial amyloidotic cardiomyopathy (FAC).