Amicus Therapeutics is an American biopharmaceutical company located in Cranbury, New Jersey. In 2007 the company went public after a planned offering in 2006 followed by a withdrawal. The offering would have made AMTX the established symbol for trading. Amicus was funded by numerous capital firms including New Enterprise Associates, Radius Ventures and Canaan Partners.
Amicus Therapeutics focuses on rare and orphan diseases although their main objective is a disorder called lysosomal storage. The development of the company’s products is based on the Chaperone-Advanced Replacement Therapy also referred to as CHART. They have concentrated on developing therapies for enzyme replacement. Amicus’s reputation expanded in 2004 when their broadcast portfolio was noted in the pharmaceutical industry for small molecule pharmacological chaperones.
Amicus Therapeutics had no products marketed in 2014 and their most likely candidate was migalastat which uses the trade name of Galafold. The product is a treatment for Fabry disease with the goal of stabilizing endogenous mutant alpha-galactosidase. The company is also involved with JCR Pharmaceutical and GlaxoSmithKline in the investigation of coformulation with recombinant alpha-galactosidase. This partnership began in 2010 and continued until 2013.
Amicus Therapeutics expanded from their only site in New Jersey in 2008 to an additional research facility located in San Diego, California. By the end of 2009 the termination of a long collaboration with Shire caused financial setbacks. At this point in time private placements were used to finance their operations. This occurred with the use of proceeds secured with their first public offering, preferred stock and the agreement they had with Shire. The began restructuring at a corporate level to reduce their workforce by twenty percent.
Amicus Therapeutics received a United States grant in 2010. The $500,000 was received from the Michael J. Fox Foundation to support collaborative studies at UCLA with the David Geffen School of Medicine. An additional $210,300 grant was received in 2010 from the foundation for Alzheimer Drug Discovery. The grant supported their pre-clinical work and was a collaboration between Amicus and Mount Sinai’s Icahn School of Medicine. Amicus acquired the company Scioderm in September of 2015 for $947 million in stocks and cash.
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