Additional Information
Market: NASDAQ
Sector: Pharmaceuticals
EPIC: FOLD
Latest Price: 6.61  (-4.20% Descending)
52-week High: 7.47
52-week Low: 1.82
Market Cap: 520.21M
1 year chart
1 day chart
Amicus Therapeutics
www.amicustherapeutics.com
Amicus Therapeutics, Inc. (Amicus) is a biopharmaceutical company focused on the discovery, development and commercialization of orally administered, small molecule drugs known as pharmacological chaperones for treating a range of diseases, including lysosomal storage diseases and diseases of neurodegeneration.

Amicus Therapeutics shares tank after late stage study fails main goal

20th Dec 2012, 1:27 pm by Deborah Bacal
Amicus Therapeutics (NASDAQ:FOLD) shares plummeted Thursday, a day after the company and GlaxoSmithKline plc (GSK) said a late-stage study of a treatment for Fabry disease didn’t meet the primary goal. Amicus Therapeutics (NASDAQ:FOLD) shares plummeted Thursday, a day after the company and GlaxoSmithKline plc (GSK) said a late-stage study of a treatment for Fabry disease didn’t meet the primary goal.

Amicus Therapeutics (NASDAQ:FOLD) shares plummeted Thursday, a day after the company and GlaxoSmithKline plc (GSK) said a late-stage study of a treatment for Fabry disease didn’t meet the primary goal.

Shares in Amicus retreated more than 45 per cent on the Nasdaq this afternoon, to $3.13. 

The partners announced the 6-month primary treatment period results from the first phase 3 global study of the oral migalastat HCl monotherapy in males and females with Fabry disease. 

The trial randomized a total of 67 patients to receive oral migalastat HCl 150 mg or placebo on an every-other-day (QOD) dosing schedule during a 6-month, double-blind primary treatment period.

Globotriaosylceramide (GL-3) is the lipid substrate that accumulates in tissues of patients with Fabry disease, most notably in the kidney. GL-3 clearance from the kidney interstitial capillaries has been used as a marker of treatment effect in Fabry disease. 

The accumulation of GL-3 is believed to cause the various manifestations of Fabry disease, including pain, kidney failure, and increased risk of heart attack and stroke.

The companies said that 41 per cent in the migalastat HCl treatment group showed a 50 per cent or greater reduction in kidney interstitial capillary GL-3 after 6 months of study treatment versus 28 per cent in the placebo group. This difference did not achieve statistical significance, according to the pre-specified primary endpoint analysis. 

"Consistent with our phase 2 experience, the 6-month results from Study 011 demonstrate notable trends in kidney interstitial capillary GL-3 reduction in favor of migalastat HCl monotherapy compared to placebo," said chairman and CEO of Amicus, John F. Crowley. 

"We look forward to announcing additional 6-month results at the WORLD Symposium in February, including a presentation of important secondary and tertiary endpoints in this study. We also anticipate 12-month results from this study in the first half of 2013."

The company said that once it has the 12-month data, it intends to meet with the FDA to discuss a U.S. approval pathway. "We continue to believe that migalastat HCl may become an important treatment option as an oral monotherapy drug for both men and women with Fabry disease who have amenable mutations," said Crowley. 

So far, no drug-related serious adverse events have been observed.

Amicus is in collaboration with GlaxoSmithKline (GSK) in developing the investigational pharmacological chaperone migalastat HCl for the treatment of Fabry disease. Amicus has commercial rights to all Fabry products in the US, and GSK has commercial rights to all of these products in the rest of world. 

No investment advice


Proactive Investors North America Inc, trades as "Proactiveinvestors USA & Canada".


You understand and agree that no content published on the Site constitutes a recommendation that any particular security, portfolio of securities, transaction, or investment strategy is suitable or advisable for any specific person. You further understand that none of the information providers or their affiliates will advise you personally concerning the nature, potential, advisability, value or suitability of any particular security, portfolio of securities, transaction, investment strategy, or other matter.


You understand that the Site may contain opinions from time to time with regard to securities mentioned in other products, including company related products, and that those opinions may be different from those obtained by using another product related to the Company. You understand and agree that contributors may write about securities in which they or their firms have a position, and that they may trade such securities for their own account. In cases where the position is held at the time of publication and such position is known to the Company, appropriate disclosure is made. However, you understand and agree that at the time of any transaction that you make, one or more contributors may have a position in the securities written about. You understand that price and other data is supplied by sources believed to be reliable, that the calculations herein are made using such data, and that neither such data nor such calculations are guaranteed by these sources, the Company, the information providers or any other person or entity, and may not be complete or accurate.


From time to time, reference may be made in our marketing materials to prior articles and opinions we have published. These references may be selective, may reference only a portion of an article or recommendation, and are likely not to be current. As markets change continuously, previously published information and data may not be current and should not be relied upon.


You understand that we may be providing advertising and/or marketing services to companies mentioned on the site. A full list of companies that are paying for services from us, or our affiliated companies in the UK and Australia can be viewed here