FOLD - Amicus Therapeutics, Inc. - Page 4

somebody must know something, this thing has steadily increased the last 3 weeks and DOUBLED in the last 3 months

Amicus Therapeutics Provides 2011 Business Outlook and Expected Key Milestones
Significant progress expected to further establish Company as a leader in rare diseases

Press Release Source: Amicus Therapeutics On Tuesday January 11, 2011, 8:00 am EST
CRANBURY, N.J., Jan. 11, 2011 /PRNewswire/ -- Amicus Therapeutics (Nasdaq:FOLD - News) today will provide the Company's business outlook and expected key milestones for 2011 at the 29th Annual J.P. Morgan Healthcare Conference.

At the conference, Amicus is providing an update on its three key areas of focus: Amigal (migalastat hydrochloride) for the treatment of Fabry Disease, the evaluation of pharmacological chaperones co-administered with ERT, and the investigation of pharmacological chaperones for the treatment of diseases of neurodegeneration. The Company intends to identify key milestones expected in 2011 across these three areas, including results from the following studies:


Phase 3 study of Amigal for Fabry Disease in 2H11





Phase 2 study of Amigal co-administered with enzyme replacement therapy (ERT) for Fabry Disease in 2H11





Phase 2 study of AT2220 co-administered with ERT for Pompe Disease in 2H11





Late-stage preclinical proof of concept studies of AT3375 for Parkinson's Disease, including completion of additional IND-enabling activities, in 2H11.




"2011 promises to be a transformational year for Amicus. This is an exciting time in the rare disease field and we are uniquely positioned to develop new therapies for patients and to build value for our shareholders," said John F. Crowley, Chairman and CEO of Amicus. "This year we intend to achieve multiple milestones, led by our anticipated Phase 3 results for Amigal in Fabry Disease, which we expect to achieve in collaboration with our new partner, GSK Rare Diseases. In addition, we intend to move forward with Phase 2 studies evaluating chaperones co-administered with enzyme replacement therapy (ERT) in both Fabry and Pompe diseases. Finally, we expect important progress in our preclinical programs investigating the use of pharmacological chaperones in genetically defined sub-populations of Parkinson's disease and Alzheimer's disease."

Financial Guidance

The Company expects to begin 2011 with a cash balance of approximately $100 million and to spend between $45 and $55 million on 2011 operating expenses (net of cost sharing and milestones related to GSK collaboration). The current cash position, including anticipated payments from GSK in connection with the collaboration, is expected to be sufficient to fund the Company's operations and capital expenditure requirements through the anticipated commercial launch of Amigal in the United States.

In 2011, the Company intends to evaluate additional business development opportunities to further build shareholder value. The Company indicates that it is actively exploring a range of opportunities with multiple potential partners.



Could be an interesting year for this one. They have alot of catalyst coming up.

I have to say....you deserve all the money in the world! your mid-long term picks always make money!

aaaaaaaaaaaaaaaand that's enough for me! took long enough but a nice run. Gotta think it's going to pull back hard, switching sides to a short for now at least back into the mid 4 range

nothing like making money on both sides!!! we'll be down to the mid 4's at LEAST soon, volume will die out and we'll settle back in low 4's, mid 3's maybe again

ticker matches the chart....

Amicus announces positive results from Phase II Pompe disease study
5 days 1 hours 54 minutes ago - Datamonitor via Comtex
Amicus Therapeutics, Inc., a biopharmaceutical company, has announced positive preliminary results from all four dose cohorts in a Phase II study to evaluate the safety and pharmacokinetic effects of the pharmacological chaperone AT2220 co-administered with enzyme replacement therapy, or ERT, for Pompe disease.

Myozyme and Lumizyme (alglucosidase alfa, or recombinant human GAA enzyme, rhGAA) are the first and only approved treatments for Pompe disease. Based on the Study 010 results, Amicus expects to initiate a repeat-dose clinical study in the third quarter of 2013.

For people with Pompe disease, deficient GAA enzyme leads to the accumulation of glycogen in tissues affected by disease (primarily muscle). Preclinical data1 demonstrated that AT2220 in combination with ERT enhances rhGAA enzyme activity, reduces glycogen accumulation, and potentially mitigates ERT-related immunogenicity in a mouse model of Pompe disease. In Study 010, co-administration of AT2220 to Pompe patients increased rhGAA enzyme activity and enhanced rhGAA enzyme uptake into muscle tissue compared to ERT alone.

John Crowley, chairman and CEO stated, "Study 010 has established human proof-of-concept that AT2220-ERT co-administration increases GAA enzyme activity in muscle. We look forward to initiating our repeat-dose clinical study to investigate the effect of AT2220-ERT co-administration on ERT stability and activity, ERT-related immunogenicity, and other clinical measures. We believe that co-administration may deliver significant benefits compared to ERT alone and become an important therapy for people with Pompe disease."

Study 010 investigated single ascending oral doses of AT2220 co-administered with Myozyme or Lumizyme in patients with Pompe disease. The doses of AT2220 were 50 mg (Cohort 1), 100 mg (Cohort 2), 250 mg (Cohort 3), and 600 mg (Cohort 4). Each patient received one infusion of ERT alone, and then a single oral dose of AT2220 one hour before the next ERT infusion. Single doses of AT2220 co-administered with ERT were well-tolerated, with no drug-related adverse events reported. In addition, AT2220 was cleared from muscle to near-undetectable levels by Day 7 in all four cohorts.

24-hour plasma PK was measured during and after each infusion. Plasma rhGAA activity increased in 23 out of 23 patients (100%) following co-administration and the increases were dose-related. These data suggest that co-administration increases the amount of stabilized, properly folded, and active rhGAA enzyme available for uptake into tissue.

Muscle biopsies were taken to measure GAA enzyme uptake into muscle tissue, with and without AT2220. In Cohort 1, all 4 patients had muscle biopsies on Day 7. In Cohorts 2-4, muscle biopsies were taken on Day 3 for half the patients, and on Day 7 for the other half of patients.

In Cohort 1, no consistent change in GAA enzyme activity was observed at day 7. In Cohorts 2, 3, and 4 the results show that more enzyme is taken up into muscle tissue following AT2220 co-administration compared to ERT alone. The effect was most pronounced at the highest (600 mg) dose of AT2220.

At Day 3 the GAA enzyme activity in muscle following co-administration compared to ERT alone in patients with evaluable biopsies increased by the following; 25% in Cohort 2 (n=3), 7% in Cohort 3 (n=3), and 133% in Cohort 4 (n=2). At Day 7 the GAA enzyme activity in muscle was lower relative to Day 3, as expected based on the cellular half-life of the enzyme. However, following co-administration compared to ERT alone in patients with evaluable biopsies the following increases were sustained: 20% in Cohort 2 (n=3), 40% in Cohort 3 (n=2), and 20% in Cohort 4 (n=3).

nice if you caught the bounce but mayb losing steam at halfway back to pre gap levels from the recent lows

Amicus Therapeutics Presents Additional 6-Month Results from Phase 3 Fabry Monotherapy Study at LDN World Symposium
2 days 1 hours 13 minutes ago - GlobeNewswire via Comtex


Amicus Therapeutics (Nasdaq:FOLD), today announced additional 6-month (Stage 1) results from the first ongoing Phase 3 global registration study (Study 011) of investigational oral migalastat HCl monotherapy (150 mg, every-other-day) in males and females with Fabry disease who had genetic mutations identified as amenable to migalastat HCl in a cell-based assay. Stage 1 results were highlighted in an oral platform presentation at the Lysosomal Disease Network WORLD Symposium (LDN WORLD) by Dr. Fatih Ezgu, Gazi University.

Study 011 consists of a 6-month, double-blind period (Stage 1) when subjects received migalastat HCl 150 mg or placebo, a 6-month open label-follow up period (Stage 2) when all patients received migalastat HCl, and an ongoing 12-month open-label extension.

Initial top-line Stage 1 results previously reported in December, 2012 for the primary endpoint in Stage 1 did not meet statistical significance. The pre-specified primary and secondary analyses of the primary endpoint numerically favored migalastat HCl over placebo. In the primary responder analysis, 13/32 (41%) in the migalastat HCl group versus 9/32 (28%) in the placebo group demonstrated a 50% or greater reduction in kidney interstitial capillary GL-3 from baseline to month 6 (p=0.3). Taken alone the secondary analysis of the absolute percent change in kidney interstitial capillary GL-3 from baseline to month 6 showed a median reduction of 41% in the migalastat HCl group versus a median reduction of 6% in the placebo group (p=0.093).

Study 011 entry criteria, particularly elevated urine GL-3, were intended to enrich for patients with higher interstitial capillary GL-3, and more measurable disease burden. Although all patients had detectable interstitial capillary GL-3 at baseline, a number of patients had low levels of GL-3 at baseline, making it difficult to detect a significant difference in responders between the 2 treatment groups. Clearance of kidney interstitial capillary GL-3, a marker of treatment effect, is being measured by histology in evaluable kidney biopsies from baseline to month 6 (Stage 1) as well as baseline to month 12 (Stage 2). Stage 2 results remain blinded at this time.

Dr. Ezgu said, "The 6-month data from Study 011 are encouraging, including a post-hoc subgroup analysis presented today, and we continue to evaluate patients in this ongoing study. There is still an unmet medical need for Fabry disease treatments. Migalastat HCl may potentially offer an oral treatment for Fabry patients with amenable mutations based on these results from Study 011 and earlier clinical studies, and potentially forthcoming data from ongoing studies."

less than stellar phase III