|Traded as||NASDAQ: FOLD|
Amicus Therapeutics is a public American biopharmaceutical company based in Cranbury, New Jersey. The company went public in 2007 under the NASDAQ trading symbol FOLD. This followed a 2006 planned offering and subsequent withdrawal, which would have established the trading symbol as AMTX Prior to their IPO, Amicus was funded by a variety of venture capital firms including Radius Ventures, Canaan Partners and New Enterprise Associates.
The therapeutic focus of Amicus is on rare and orphan diseases, particularly disorders collectively called lysosomal storage disorders. The company's product development is based largely on the Chaperone-Advanced Replacement Therapy (CHART®) platform and has concentrated on development of enzyme replacement therapies (ERTs). In 2014, Amicus was noted as having, arguably, "the broadest portfolio of small molecule pharmacological chaperones" in the pharmaceutical industry.
As of February 2014, the company had no marketed products, its most advanced candidate being migalastat (trade name Galafold), a pharmacological chaperone treatment for Fabry disease which aims to stabilize endogenous mutant alpha-galactosidase. In parallel with advancement of migalastat monotherapy, the company engaged in a collaboration with GlaxoSmithKline and JCR Pharmaceutical to investigate coformulation with recombinant alpha-galactosidase (JR-051). This collaboration lasted for three years, from 2010 to 2013.
Amicus has no manufacturing capability of its own, relying on contract manufacturing alone.
In 2008, the company expanded from its single site in New Jersey to a second research site in San Diego.
In late 2009, the company faced a major financial setback with the termination of a multi-year collaboration agreement with Shire; at the time of the termination, financing for operations was done primarily "through private placements of our redeemable convertible preferred stock, proceeds from our initial public offering and from our prior collaboration agreement with Shire." A corporate restructuring ensued with a concomitant 20% reduction in the workforce.
In 2010, the company received a US$500,000 grant from the The Michael J. Fox Foundation to support studies conducted in collaboration with David Geffen School of Medicine at UCLA. Amicus also received in 2010 a US$210,300 grant from the Alzheimer's Drug Discovery Foundation to support pre-clinical work in collaboration with Icahn School of Medicine at Mount Sinai through the Alzheimer's Disease Research Center.
In November 2013, Amicus acquired competitor Callidus Biopharma, thereby obtaining proprietary materials and intellectual property for the enzyme replacement therapy treatment of Pompe disease.
In September 2015, the company acquired Scioderm for up to $947 million in cash and stock.
The role of chief executive officer have been vested in John F. Crowley since 2005. In 2010, Crowley succeeded Donald Hayden, Jr. as the company's chairman of the board; Hayden remained associated with Amicus through the new role of Lead Independent Director. The executive posts of chief operating officer and chief financial officer are vested in Bradley L. Campbell and William D. Baird III, respectively. As of 2010, the company's chief scientific officer was David Lockhart.
Beyond the C-Suite, as of 2010 Brandon Wustman held the post of Director of Exploratory Biology.