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Company type | Public |
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Nasdaq: FOLD Russell 2000 Component | |
Industry | Biotechnology |
Founded | February 4, 2002 [1] : 28 |
Headquarters | , United States |
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Number of employees | 508 (31 Dec 2018) [1] : 28 |
Website | www |
Footnotes /references [1] [2] |
Amicus Therapeutics, Inc. is a public American biopharmaceutical company based in Philadelphia, PA. [3] The company went public in 2007 under the NASDAQ trading symbol FOLD. [4] This followed a 2006 planned offering and subsequent withdrawal, which would have established the trading symbol as AMTX [5] [6] Prior to their IPO, Amicus was funded by a variety of venture capital firms including Radius Ventures, Canaan Partners and New Enterprise Associates. [7]
The therapeutic focus of Amicus is on rare and orphan diseases, particularly disorders collectively called lysosomal storage disorders. [8] : 2 The company has focused on pharmacological chaperones and enzyme replacement therapy. [8] : 2, 18 [9] : 95
In 2008, the company expanded from its single site in New Jersey to a second research site in San Diego. [10]
In late 2009, the company faced a major financial setback with the termination of a multi-year collaboration agreement with Shire for its lead compound, migalastat, and two other products. [11] : 24 Amicus cut 20% of its workforce. [11] : 24
The next year, the company signed an exclusive license agreement with GlaxoSmithKline for migalastat; GSK paid $60 million in upfront and equity payments while promising up to $170 million to help develop their products, [12] GSK terminated the arrangement in 2013. [12]
In November 2013, Amicus acquired competitor Callidus Biopharma for its enzyme replacement therapy treatment for Pompe disease. [8] : 3, 12
In September 2015, the company acquired Scioderm for $229 million in cash and stock, with potentially $618 million more in biodollars. Scioderm had a drug in Phase III trials for epidermolysis bullosa; additionally, if the drug was approved the FDA would give its owner a priority review voucher, which itself can be sold to another company. [13] That drug failed its Phase III trial in 2017 and Amicus abandoned it. It had paid out around half of the milestones to Scioderm's shareholders prior to closing down the program. [14]
Also in September 2015, Amicus announced that it would submit a new drug application (NDA) for accelerated approval of migalastat to the FDA by the end of 2015. [15] An FDA committee recommended approval in April 2016, but the FDA rejected the application in November for having insufficient data in November 2016. [16] The drug was approved in the European Union in May 2016. [17]
In 2016 Amicus acquired MiaMed which was working on drugs to treat CDKL5 deficiency; Amicus paid $1.8M in cash and around $4.7M in stock, with $83M in biodollars. [18]
After Scott Gottlieb became FDA commissioner in 2017, the CEO of Amicus began lobbying him directly for the FDA to accept the NDA and in February 2018 the FDA accepted it and promised a response by August 2018. [19]
In September 2018, Amicus announced that it had acquired Celenex, which had ten early stage gene therapies. Amicus paid $100 million upfront and could pay an additional $352 million if developmental milestones are achieved. [20]
GSK plc is a British multinational pharmaceutical and biotechnology company with global headquarters in London. It was established in 2000 by a merger of Glaxo Wellcome and SmithKline Beecham, which was itself a merger of a number of pharmaceutical companies around the Smith, Kline & French firm.
Biogen Inc. is an American multinational biotechnology company based in Cambridge, Massachusetts, United States specializing in the discovery, development, and delivery of therapies for the treatment of neurological diseases to patients worldwide. Biogen operates in Argentina, Brazil, Canada, China, France, Germany, Hungary, India, Italy, Japan, Mexico, Netherlands, Poland, Sweden, and Switzerland.
Fabry disease, also known as Anderson–Fabry disease, is a rare genetic disease that can affect many parts of the body, including the kidneys, heart, brain, and skin. Fabry disease is one of a group of conditions known as lysosomal storage diseases. The genetic mutation that causes Fabry disease interferes with the function of an enzyme that processes biomolecules known as sphingolipids, leading to these substances building up in the walls of blood vessels and other organs. It is inherited in an X-linked manner.
Exelixis, Inc. is a genomics-based drug discovery company located in Alameda, California, and the producer of Cometriq, a treatment approved by the U.S. Food and Drug Administration (FDA) for medullary thyroid cancer with clinical activity in several other types of metastatic cancer.
Vertex Pharmaceuticals Incorporated is an American biopharmaceutical company based in Boston, Massachusetts. It was one of the first biotech firms to use an explicit strategy of rational drug design rather than combinatorial chemistry. It maintains headquarters in South Boston, Massachusetts, and three research facilities, in San Diego, California, and Milton Park, Oxfordshire, England.
Neurocrine Biosciences, Inc. is an American biopharmaceutical company founded in 1992. The company is headquartered in San Diego, California, and led by CEO Kevin Gorman. Neurocrine develops treatments for neurological and endocrine-related diseases and disorders. In 2017, the company's drug valbenazine (Ingrezza) was approved in the US to treat adults with tardive dyskinesia (TD).
Priority review is a program of the United States Food and Drug Administration (FDA) to expedite the review process for drugs that are expected to have a particularly great impact on the treatment of a disease. The priority review voucher program is a program that grants a voucher for priority review to a drug developer as an incentive to develop treatments for disease indications with limited profitability.
Sirtris Pharmaceuticals, Inc. was a biotechnology company based in Cambridge, MA that developed therapies for type 2 diabetes, cancer, and other diseases. Conceived in 2004 by Harvard University biologist David Sinclair and Andrew Perlman, and founded that year by Sinclair and Perlman, along with Christoph Westphal, Richard Aldrich, Richard Pops, and Paul Schimmel, the company was focused on developing Sinclair's research into activators of sirtuins, work that began in the laboratory of Leonard P. Guarente where Sinclair worked as a post-doc before starting his own lab.
Christoph Westphal is an American biomedical businessman.
Intepirdine (INN; developmental codes SB-742457, RVT-101) is a selective 5-HT6 receptor antagonist with potential cognition, memory, and learning-enhancing effects. It was under development by GlaxoSmithKline for the treatment of Alzheimer's disease and demonstrated some preliminary efficacy in phase II clinical trials. GSK chose not to continue development and sold the rights to Axovant Sciences for $5 million in December 2014.
Alexion Pharmaceuticals, a subsidiary of AstraZeneca, is a pharmaceutical company headquartered in Boston, Massachusetts that specializes in orphan drugs to treat rare diseases.
Alnylam Pharmaceuticals, Inc. is an American biopharmaceutical company focused on the discovery, development and commercialization of RNA interference (RNAi) therapeutics for genetically defined diseases. The company was founded in 2002 and is headquartered in Cambridge, Massachusetts. In 2016, Forbes included the company on its "100 Most Innovative Growth Companies" list.
Catalyst Pharmaceuticals, Inc. is a biopharmaceutical company based in Coral Gables, Florida. The company is developing medicines for rare diseases, including the phosphate salt of amifampridine for the treatment of Lambert–Eaton myasthenic syndrome (LEMS). The drug is referred to under the trade name Firdapse, which was approved by the FDA for approved use in children 6 years and older with LEMS in addition to the prior approval for use in adults with LEMS on November 28, 2018. Firdapse commercially launched in January 2019.
Scioderm, acquired by Amicus Therapeutics in 2015, was a rare disease company focused on developing a treatment for Epidermolysis Bullosa (EB), a rare genetic disease characterized by extremely fragile skin and recurrent blister formation. There are currently no approved therapies for EB. Scioderm was developing a topical treatment known as SD-101, or Zorblisa, aimed at triggering wound reduction and closure, and a reduction in body surface area coverage of blisters and lesions.
Henri A. Termeer was a Dutch biotechnology executive and entrepreneur who is considered a pioneer in corporate strategy in the biotechnology industry for his tenure as CEO at Genzyme. Termeer created a business model adopted by many others in the biotech industry by garnering steep prices— mainly from insurers and government payers— for therapies for rare genetic disorders known as orphan diseases that mainly affect children. Genzyme uses biological processes to manufacture drugs that are not easily copied by generic-drug makers. The drugs are also protected by orphan drug acts in various countries which provides extensive protection from competition and ensures coverage by publicly funded insurers. As CEO of Genzyme from 1981 to 2011, he developed corporate strategies for growth including optimizing institutional embeddedness nurturing vast networks of influential groups and clusters: doctors, private equity, patient-groups, insurance, healthcare umbrella organizations, state and local government, and alumni. Termeer was "connected to 311 board members in 17 different organizations across 20 different industries" He has the legacy of being the "longest-serving CEO in the biotechnology industry.
Ionis Pharmaceuticals, Inc. is a biotechnology company based in Carlsbad, California, that specializes in discovering and developing RNA-targeted therapeutics. The company has three commercially approved medicines: Spinraza (Nusinersen), Tegsedi (Inotersen), and Waylivra (Volanesorsen) and has four drugs in pivotal studies: tominersen for Huntington’s disease, tofersen for SOD1-ALS, AKCEA-APO(a)-LRx for cardiovascular disease, and AKCEA-TTR-LRx for all forms of TTR amyloidosis.
Adverum Biotechnologies, Inc., formerly known as Avalanche Biotechnologies, is a publicly traded clinical stage gene therapy company located in Redwood City, California. The company is targeting unmet medical needs for serious ocular and rare diseases, including wet age-related macular degeneration.
Migalastat, sold under the brand name Galafold, is a medication for the treatment of Fabry disease, a rare genetic disorder. It was developed by Amicus Therapeutics. The US Food and Drug Administration (FDA) granted it orphan drug status in 2004, and the European Commission followed in 2006. The European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) granted the drug a marketing approval under the name Galafold in May 2016.
Ultragenyx is an American biopharmaceutical company involved in the research and development of novel products for treatment of rare and ultra-rare genetic diseases for which there are typically no approved treatments and high unmet medical need. The company works with multiple drug modalities including biologics, small molecule, gene therapies, and ASO and mRNAs in the disease categories of bone, endocrine, metabolic, muscle and CNS diseases.
CRISPR Therapeutics AG is a Swiss–American biotechnology company headquartered in Zug, Switzerland. It was one of the first companies formed to utilize the CRISPR gene editing platform to develop medicines for the treatment of various rare and common diseases. The company has approximately 500 employees and has offices in Zug, Switzerland, Boston, Massachusetts, San Francisco, California and London, United Kingdom. Its manufacturing facility in Framingham, Massachusetts won the Facilities of the Year Award (FOYA) award in 2022. The company’s lead program, exagamglogene autotemcel, or exa-cel, was granted regulatory approval by the US Food and Drug Administration (FDA) in December 2023.
There have been five withdrawn IPOs so far this month, including ... Amicus Therapeutics.