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portefeuille (98.92)

biopharmaceuticals FOLD



June 29, 2010 – Comments (54)

you might want to buy some FOLD shares.



54 Comments – Post Your Own

#1) On June 29, 2010 at 3:44 PM, portefeuille (98.92) wrote:

Spain will win, by the way ...

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#2) On June 29, 2010 at 3:52 PM, portefeuille (98.92) wrote:

#1 1:0!

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#3) On June 29, 2010 at 5:46 PM, outoffocus (22.87) wrote:


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#4) On June 29, 2010 at 5:56 PM, portefeuille (98.92) wrote:

oh, I have written this mostly to be able to write a comment saying "I told you so" in a few months ...

I think zzlangerhans will like it for the first time when he makes his next call on FOLD (see here) and I am sure he will write a much better pitch than I could.

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#5) On June 29, 2010 at 5:56 PM, portefeuille (98.92) wrote:

Spain did win, by the way ...

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#6) On June 29, 2010 at 9:23 PM, ChrisGraley (28.48) wrote:

OK porte,

I bought this stock with absolutely zero research, because I was busy at work and a guy that I think is a pretty good investor that really doesn't plug stocks, told me to buy FOLD and not only buy FOLD, but buy it NOW! I tried to get the buy in under the wire, but it looks like it won't be filled until tomorrow. Our CEO came in for a visit today, so I've been very distracted. I want to start to research this stock, but I'm wary to do so, because I never make knee jerk reactions like this, I'm holding off my research till tomorrow and know nothing about protein folding. Can you give me a little bit of hope before I set myself up for pain tomorrow?

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#7) On June 29, 2010 at 9:55 PM, portefeuille (98.92) wrote:

#3,6 okay, okay, you win. I was a little distracted as well when I wrote this post (by the Spain - Portugal match). The "now" part (including the "(hehe)") was of course to some degree to be taken as added humour, I do not have any information that lets me think it will start a "huge rally from here". I almost always buy (and sell) stocks in tranches, especially when I try to "catch falling knifes", so for me the exact time of the buys and sells of those tranches is not all that important.

What should give you a little preliminary comfort is the fact that at the end of the quarter ended March 31, 2010 they had this "balance sheet"


and they currently have a market capitalisation of around $57 million.

I will try to gather the "science part" of the story in a convenient form. That might take a little time ...

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#8) On June 29, 2010 at 10:07 PM, portefeuille (98.92) wrote:

the chart.


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#9) On June 29, 2010 at 10:12 PM, portefeuille (98.92) wrote:

some added comfort maybe.


Leerink Swann Initiates Coverage on Amicus Therapeutics (FOLD) with a Market Perform; Oral Chaperones for Orphan Diseases: Speculative but Intriguing

March 31, 2010 7:32 AM EDT

Leerink Swann initiates coverage on Amicus Therapeutics (Nasdaq: FOLD) with a Market Perform rating. Price target $3.50. 

Leerink analyst says, "We believe the lack of major near-term drivers may limit upside for FOLD shares in 2010. Data from FOLD's lead product candidate..." 

To see all the upgrades/downgrades on shares of FOLD, visit our Analyst Ratings page. 

Amicus Therapeutics, Inc. is a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of orally administered, small molecule drugs, known as pharmacological chaperones, for the treatment of a range of human genetic diseases.




Canaccord Initiates Coverage on Biotechnology and Drugs Stocks

April 6, 2010 8:48 AM EDT

Canaccord initiates coverage on Amicus Therapeutics (Nasdaq: FOLD) & Avanir Pharmaceuticals (Nasdaq: AVNR) with a Buy rating.


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#10) On June 29, 2010 at 10:21 PM, portefeuille (98.92) wrote:

#9 found a better version here.


NEW YORK (AP) -- A Leerink Swann Research analyst started coverage of Amicus Therapeutics Inc. with a "Market Perform" rating Wednesday, citing a lack of upcoming catalysts needed to drive shares.

Leerink analyst Joseph Schwartz also gave the stock a $3.50 price target.

"Data from Amicus' lead product candidate Amigal, currently enrolling Phase III clinical trials for Fabry disease, is not expected until mid-2011," he said, in a note to investors. "Amigal is a relatively high risk program, which addresses an estimated $500 million market opportunity in the U.S."

Fabry disease is a disorder in which the body lacks the ability to store some fats.

He said physicians are impressed with the late-stage study's design, though it could still find success difficult.

"According to physicians, it may be challenging to demonstrate broad efficacy in the pivotal Phase III trial because of the high genetic variability of the disease on which Amicus' technology is predicated," he said.

Looking further ahead, Schwartz cited a positive outlook for use of the company's technology in other areas, including combinations with enzyme replacement therapy.

Shares of Amicus rose 1 cent to $3.39 in morning trading. Over the past year the stock has traded at a high of $13.50 last June and a low of $3.04 earlier this month. Shares fell sharply in October after the company reported negative study results from its potential Gaucher disease drug Plicera and ended its partnership with Shire PLC on treatments for genetic disorders. The company also cut jobs and its chief financial officer resigned.


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#11) On June 29, 2010 at 10:31 PM, portefeuille (98.92) wrote:


Zacks #1 Rank Additions for Thursday

On Thursday May 6, 2010, 6:34 am EDT

Here are 5 stocks added to the Zacks #1 Rank ("strong buy") List today:

Acacia Research Corp (NasdaqGM: ACTG - News)
Acergy SA (NasdaqGS: ACGY - News)
America Service Group Inc (NasdaqGS: ASGR - News)
Amicus Therapeutics, Inc (NasdaqGM: FOLD - News)
Anaren, Inc (NasdaqGS: ANEN - News)


(from here)

I don't really "believe" in Zacks ratings.

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#12) On June 29, 2010 at 10:33 PM, portefeuille (98.92) wrote:

this is a good starting point.

Corporate Presentation May 2010 (pdf)

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#13) On June 29, 2010 at 10:38 PM, Momentum21 (96.57) wrote:

thanks about JAV today? I held on to a few shares this time around as per your suggestion... : )

 This and AZN saved my day a bit... 

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#14) On June 29, 2010 at 10:48 PM, WallstreetKnight (40.64) wrote:

Hmm I've purchased a stock you "compelled" before, but what's the underlying reason for this pitch?

You seem to have a lot of conviction... or maybe that was the football talking... hah 

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#15) On June 29, 2010 at 10:50 PM, topsecret09 (86.79) wrote:

 Like the fact that he ended up here Instead of (OREX) John F. Crowley
Chief Executive Officer and Chairman Mr. Crowley became president and CEO of Amicus in January 2005 and was appointed Chairman of the Board in February 2010. Previously he was founding president and CEO of Orexigen Therapeutics. Preceding Orexigen, Mr. Crowley was senior vice president at Genzyme Therapeutics, a position he assumed after overseeing the sale of Novazyme Pharmaceuticals to Genzyme in September 2001. Mr. Crowley was the founding president and CEO of Novazyme that was developing a novel treatment for Pompe disease. He previously served in several senior management roles with the Bristol-Myers Squibb Company (BMS), including director of the Executive Committee for the U.S. Medicines Group, director of Business Strategy for the U.S. Pharmaceuticals Group, and director of U.S. Area Marketing for the Neuroscience and Infectious Disease Division. Preceding his experience at BMS, Mr. Crowley worked as a business strategy consultant for Marakon Associates. Mr. Crowley began his professional career as a litigation associate in the Health Care Practice Group of the Indianapolis-based law firm of Bingham, Summers, Welsh & Spilman.

Mr. Crowley is involved with several charitable and community organizations, including serving on the Board of Directors of St. Peter's University Health Care System. Mr. Crowley's involvement with biotechnology stems from the 1998 diagnosis of two of his children with Pompe disease - a fatal neuromuscular disorder. Mr. Crowley and his family have been featured on the cover of The Wall Street Journal and on The Today Show, CNBC and The Paula Zahn Show on CNN. In 2006, Geeta Anand, Pulitzer prize winning writer for The Wall Street Journal, authored a book on Mr. Crowley entitled "The Cure: How a Father Raised $100 Million- and Bucked the Medical Establishment- in a Quest to Save His Children." (

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#16) On June 29, 2010 at 10:54 PM, topsecret09 (86.79) wrote:

Pompe disease is an inherited lysosomal storage disorder caused by deficiency of an enzyme called acid α-glucosidase (Gaa). The role of Gaa within the body is to break down glycogen, the form of sugar stored in living cells for use as energy.  Reduced or absent levels of Gaa activity leads to the accumulation of glycogen in the affected tissues, including the heart, skeletal muscles (including those involved with breathing), liver, and nervous system. This accumulation of Gaa is believed to cause progressive muscle weakness and respiratory insufficiency in individuals with Pompe disease. Pompe disease can occur in infants, toddlers, or adults, and the prognosis varies according to the time of onset and severity of symptoms.

For more information on Pompe disease, click here.

Program Overview

Amicus is utilizing a new technology in the development of treatments for genetic diseases.  Pharmacological chaperone technology involves the use of small molecules that selectively bind to and stabilize proteins in cells, leading to improved protein folding and trafficking, and increased activity. In our Pompe program, we are investigating the use of AT2220 to bind to destabilized GAA enzyme (acid alpha glucosidase or acid α-glucosidase) and thereby restore its intended biological function of degrading glycogen substrate in lysosomes. The chemical name for AT2220 is 1-deoxynojirimycin hydrochloride.

Clinical Trials

Following successful completion of multiple Phase 1 studies of AT2220 in healthy volunteers, Amicus initiated a Phase 2 clinical trial of AT2220 in adults with Pompe disease in June, 2008. Based on safety data from both preclinical and Phase 1 studies, the approved Phase 2 trial protocol involved initial treatment with a high dose of AT2220. Two patients enrolled in the trial experienced self-reported muscle weakness and subsequently withdrew from the trial. The events were categorized by the site investigator as serious and probably related to treatment with AT2220.

In Feburary 2009, Amicus announced the Company has suspended enrollment for the Phase 2 clinical trial of its investigational drug AT2220 for the treatment of Pompe Disease and that it has received a clinical trial hold from the FDA (Food and Drug Administration).

In September 2009, the FDA agreed to Amicus' proposal for a Phase 1 study and subsequently converted the clinical hold of AT2220 to a partial hold to allow the conduct of this study.   In October 2009 the Company initiated a Phase 1 study of AT2220.  The primary objective of this study is to evaluate the pharmacokinetics of AT2220 in muscle tissue in healthy adult subjects.  Results from this study are expected in the first half of 2010.
For the press release on these clinical trials, click here

Additional information regarding Pompe clinical trial(s) sponsored by Amicus can be found by visiting www. and searching the keyword “Amicus".

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#17) On June 29, 2010 at 10:54 PM, portefeuille (98.92) wrote:

#12 for parts of that presentation a good introduction might be this.

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#18) On June 29, 2010 at 10:58 PM, WallstreetKnight (40.64) wrote:


What catalyst or factor are you playing at - and at what interval (of time) are you looking to hold? 

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#19) On June 29, 2010 at 11:01 PM, portefeuille (98.92) wrote:

#13 well done. I guess you can sell those JAV shares now or just wait until they are taken from you via the takeover. As I wrote in comment #4 here I never had any JAV shares. I should have bought some!

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#20) On June 29, 2010 at 11:02 PM, ChrisGraley (28.48) wrote:

Ok, so they seem to be good at cash management and the chart looked good before I bought it. That's not too bad. Seriously though, I took the meaning of porte saying "buy now" as that they discovered the secret to an everlasting life though.

You don't usually do that.

I missed the hee hee, but you always say hee hee, so I wouldn't have known if this was a good hee hee or a bad hee. hee.

I'll study the stock tomorrow, but don't do anymore buy now posts! It's like my butcher telling me he's suddenly a vegetarian! 

Oh, and the rest of you stooges better start giving porte more recs! He's hard to figure out, but he's right more than I am and he's honest and unassuming. He's not looking for worship, he's just sharing what he knows. If I had half of his sense of numbers, I would be 4 times further in life than I am now. I've lived a life of hard knocks, but porte has a greater education that he puts to good use.

This is not a 3 rec post. it's at least a 30 rec post.

start reccing now.

If FOLD folds, then call me the failure. 


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#21) On June 29, 2010 at 11:03 PM, WallstreetKnight (40.64) wrote:

His pitch for ITMN netted me 30% in the span of 12 hrs haha.

So I am somewhat intrigued. 

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#22) On June 29, 2010 at 11:11 PM, portefeuille (98.92) wrote:

I could easily from the top of my head name 50 stocks that I made very bad calls on (at least so far very bad). I actually gave the placement of the "(hehe)" some thought, I wanted to place it right next to the "buy now!" at first and then thought it looks better in the next line. oh well ...

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#23) On June 29, 2010 at 11:12 PM, portefeuille (98.92) wrote:

from the top of my head

I guess it is 

"off the top of my head"

at least according to google.

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#24) On June 29, 2010 at 11:16 PM, portefeuille (98.92) wrote:

#22 for some reason I consider my HLCS "outperform" calls my worst. no idea why no one has ever complained ...

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#25) On June 29, 2010 at 11:17 PM, portefeuille (98.92) wrote:

#15,16 good information.

feel free to add more.

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#26) On June 29, 2010 at 11:21 PM, topsecret09 (86.79) wrote:

24) On June 29, 2010 at 11:16 PM, portefeuille (99.97) wrote:

#22 for some reason I consider my HLCS "outperform" calls my worst. no idea why no one has ever complained ...   Yea,that one fooled me too...   Insiders bought In the millions of shares at about $2.60,so they were fooled too !!  :)    TS

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#27) On June 29, 2010 at 11:25 PM, portefeuille (98.92) wrote:

#15 a movie on that.

Extraordinary Measures (1.2).

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#28) On June 29, 2010 at 11:26 PM, Momentum21 (96.57) wrote:

Porte - I sold at 2.18 straight away...the way the day was going I wasn't going to quibble about .02! 

I do recall seeing JAV on your "favorites list" which gave me motivation to stay with it until the deal was done. I was betting that at some point in time I was going to get at least $1.40 or so (even if the deal fell through) and the risk was definitely worth the reward given the viability of the company and the prospect of getting approvals in the US later this year. 

So thanks for you obsessive behavior...: )

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#29) On June 29, 2010 at 11:29 PM, WallstreetKnight (40.64) wrote:

Hmm, but you're doing pretty good for the security pitches that get their own blog post...

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#30) On June 29, 2010 at 11:33 PM, portefeuille (98.92) wrote:

news that caused the plunge in October 2009.

Amicus Therapeutics Announces Preliminary Results of Phase 2 Study with Plicera(TM) for Gaucher Disease

Amicus' Plicera Disappoints in Trial

Amicus Therapeutics Announces Third Quarter 2009 Financial Results and Strategic Business Updates



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#31) On June 29, 2010 at 11:41 PM, portefeuille (98.92) wrote:

#28 My "favourites list" is not doing so great lately I'm afraid, I think I will not add too many new stocks until the performance recovers a little.

#29 I get the feeling that once I think they "deserve" their own headline they start going nowhere. So it might actually be a "headline curse" ...

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#32) On June 29, 2010 at 11:50 PM, WallstreetKnight (40.64) wrote:

Are you picking up real-money shares in the AM?

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#33) On June 30, 2010 at 12:07 AM, portefeuille (98.92) wrote:

#32 I have bought some today (see comment #10 here).

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#34) On June 30, 2010 at 1:15 AM, portefeuille (98.92) wrote:

#9,10 Leerink Swann LLC was “Placement Agent" for the latest stock/warrents offering (see here).

The "warrent liability" item in the balance sheet posted in comment #7 above comes from that offering. 


As John mentioned earlier during the first quarter we raised $17.1 million of net proceeds through a registered direct offering of 4.95 million shares of common stock and wants to purchase an additional 1.85 million shares of common stock

We allocated $3.3 million of our net proceeds to the warrants which we then classified as a liability on our balance sheet. Each quarter the warrant liability would be mark-to-market with changes recorded as the non-cash, non-operating line item in our P&L. The change in fair value of our warrant liability during the first quarter of 2010 was $0.02 million.


(from here)

Considering the exercise price of $4.43 per Warrant Share and the current share price of around $2.05 that liability will be "marked down" at the end of the current quarter if the stock does not strongly recover by then.

So the Leerink Swann analyst that was quoted in comment #10 above was not really "neutral" but the fact that they were able to raise that money at that price should be "comforting".


In January, we provided financial guidance and at this time we reiterate our expectations that cash spend in 2010 will be $40 to $50 million and that current cash and marketable securities will be sufficient to fund operations into the second half of 2011.


(also from that earnings call transcript)

They "allocated" part of the fresh money to the European registration study.


A second milestone for us in the quarter we completed as you all know a register direct offering that yielded net proceeds of about $17 million. This was an important event for us because it not only strengthens our financial position it also allows us to dedicate additional resources to the Amigal global Phase III program in particular we can now begin that 012 study the European registration study earlier than we had planned.


(from here again)

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#35) On June 30, 2010 at 1:32 AM, portefeuille (98.92) wrote:

okay, now to the "real thing". The "important" parts of that earnings call (here and here) and that presentation (pdf) are this part


Let me spend a minute discussing why we remain confident in the Amigal Phase III reprogram. In addition to our agreements with regulatory authorities, we remained very confident this program for two major reasons. First we have designed the Phase-III studies with specific entry criteria that enrich the study population in ways that we believe increase the profitability for positive results from the study.

For example, subjects enrolled in study 011 must have both a genetic mutation that is known to respond to Amigal as well as an elevated base line level of the sub straight which we will measure as the primary end point of the study.

Second, the Phase II and Phase II extension study data give us confidence related to the likelihood the meeting the primary end point and study 011 and a potential for a positive longer term impact on kidney function which is in course the most common cause in morbidity and mortality in Fabry. For example when one performs the Phase III primary end point analysis on the patients from the Phase II study who meet the Phase III entry criteria, the result is very encouraging.

When it comes to longer term impact on real function, we have recently observed encouraging results as well. There represented the focus of the data update from our ongoing Phase II, extension study at the world meeting John, mentioned this past February. I will certainly review that briefly. As a quick reminder 26 subjects completed either 12 or 24 weeks of treatment with Amigal during the initial Phase II studies and 23 of those subjects enrolled in a separate voluntary long-term extension study designed to evaluate the long-term safety and efficacy of Amigal.

It is important to highlights the 15 subjects have been treating with Amigal for approximately two to three years and that eight subjects have been treated with a drug for more than three years. And the significant period of time on the drug is import for several reasons. Most importantly it gives us added confidence in the safety if the drug. Of note to date we still have not observed any drug related serious adverse events.

In addition it allows us the opportunity to evaluate longer term measures of efficacy which in Fabry is primarily focused on kidney function as I mentioned finally it gives us confidence more qualitatively as well considering the patients and physicians are continuing to choose to take Amigal rather than return to enzyme replacement therapy.

Today 19 subjects continue to receive treatment in this ongoing extension study. So returning to the recent data update we presented this update with the focus on renal function which we are measuring in the study by two measures. Estimated glomerular filtration rate or eGFR and proteinuria. To summarize the preliminary data we presented, indicated that eGFR remains stable out two to three years for all subjects continuing in the extension study and that trends of reduced proteinuria continue to be observed in subject identified as responders to Amigal.

What was particularly encouraging to us was that the eGFR result in the study compare excuse me favorably to the previously published eGFR literature in untreated and ERT treated Fabry patients. So in summary the Phase II and Phase II extension study results to date suggest that Amigal is generally well tolerated and then responder treatment resulted, in increased the enzyme levels, decrease GL3 level and what appears to be positive impact on renal function.

Overall, we are very encouraged by the data and believe they further support the expectations of success for the Phase III studies of Amigal and as we said before we believe Amigal has potential to be a very important treatment option for patients suffering with Fabry disease.


(from here)

and the corresponding slides #5-16. That is the story they are currently selling and that is the point zzlangerhans will surely comment on once he makes his next call on FOLD (I think he will also mention the cash and he might also mention the fact that he has so far been right on every FOLD call he has made (all "underperform" calls at much higher prices quite some time ago.)) ... 

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#36) On June 30, 2010 at 1:35 AM, portefeuille (98.92) wrote:

so now all we have to do is wait for zzlangerhans (if it were to keep falling I might buy a second tranche tomorrow, I would feel better about it if he said it's okay, hehe).

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#37) On June 30, 2010 at 10:48 AM, portefeuille (98.92) wrote:

Just in case you actually want to buy or sell FOLD shares. Use limit orders with limits that make sense (that usually means not too far away from the price of the last few trades that had decent volume. A look at the order book might be helpful as well.). The daily trading volume for FOLD has been pretty low for the last few months, especially if you subtract the very large trades.

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#38) On June 30, 2010 at 10:54 AM, portefeuille (98.92) wrote:


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#39) On June 30, 2010 at 11:58 AM, portefeuille (98.92) wrote:


That "volume spike" at the end of trading on Friday, June 25 was due to the latest Russell 3000 index reconstitution (FOLD was deleted from that index "as of Monday, June 28", see here.).

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#40) On June 30, 2010 at 4:09 PM, portefeuille (98.92) wrote:

we might be safe (see here).

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#41) On July 07, 2010 at 12:13 AM, ibitegirls (< 20) wrote:

wow, great info. thank you!

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#42) On July 12, 2010 at 12:12 PM, Momentum21 (96.57) wrote:

Picked some up at 1.95 today...

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#43) On July 16, 2010 at 10:00 PM, portefeuille (98.92) wrote:

Corporate Presentation July 2010 (pdf)

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#44) On July 18, 2010 at 11:04 PM, ChrisGraley (28.48) wrote:

porte, I noticed that you closed this one at a loss and it's trading less than cash ATM. from zzlangerhans pitch, I saw that they burn through a lot of cash quarterly, but the potential reward if the phase III  is outstanding and would easily double your initial buy-in.

Was there a reason for the close now? 

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#45) On July 20, 2010 at 1:57 PM, portefeuille (98.92) wrote:

#44 Sorry, I had not seen that comment. That was just my "caps" game version of "adding to a position at a lower price". I ended the old "outperform" call and started a new one after some time (usually around 30 minutes later). I still have my FOLD shares and will keep them for at least a few months I think, but you could of course sell a few of your shares today, they are currently at around $2.43.

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#46) On July 21, 2010 at 12:15 AM, ChrisGraley (28.48) wrote:

Thanks porte, I did notice that you bought it again later.


I'll probably hold for a while. Not a lot of risk here until the phase 3 info comes out and the reward is really good. 

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#47) On July 21, 2010 at 12:21 PM, portefeuille (98.92) wrote:

anyone that felt forced by this post to buy FOLD shares and who still does not like FOLD or has no opinion on it can sell those shares now. see this post.

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#48) On July 21, 2010 at 12:34 PM, BenitaApplebum (< 20) wrote:

I must admit that I too was compelled to buy FOLD, with both you and zzlangerhans recommending it.  I was on vacation in Tahoe earlier this month and must have gotten heat stoke since I bought twice as much FOLD as I would have otherwise (I was in such vacation bliss that when you said jump I really did say how high).  When I realized that might not have been wise I got out of half of my investment (at a 15% return in about a week), and let the other half ride (at a 30% return in a few weeks).  Could have been an entirely different story but since it was not, me and my retirement thank you wholeheartedly....    

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#49) On July 21, 2010 at 12:44 PM, portefeuille (98.92) wrote:

#48 great. The only problem is that stories like that one make me wonder how many currently hold stocks that I have recommended only because I have recommended them. I should try to get everyone out of "my stocks" at "post recommendation highs", hehe ...

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#50) On July 21, 2010 at 1:12 PM, BenitaApplebum (< 20) wrote:

I might have exaggerated a bit about the "how high" comment for effect.  I did not buy right when you suggested, but did a bit of homework and agreed with you and decided to try and catch the falling knife.  I am well aware that I might have been slashed along the way, but was rilling to take the risk.  I bet you would be surprised (and maybe a bit horrified and maybe even flattered) to see how many people hold stocks simply because you once recommended them.  With great power comes great responsibility.  But really, thanks for the insights on your comments.  They are really educational and appreciated. 

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#51) On July 23, 2010 at 4:16 AM, 1337172979 wrote:

Yeah, Spain won the World Cup, I remember president Zapatero saying to us that our economy was in the UEFA's Champions League a couple of years ago and can't help laughing my ass off recalling his stupid comments. By watching him on tv hugging soccer players as if they were the Holy Trinity when they came back home you understand how f-ckd the country is and the government's need of bread and circuses.

 A Spaniard fool.

 PS: FOLD? looks like a good opportunity, trading below cash, P/B<1, in historical lows...but chaperone "hypothesis" is still to be validated in clinical trials after Plicera's failure in phase two. Amigal ph II provied good results on safety and tolerability, but not many on efficacy besides activity increase of alpha-Gal. In fact, in Plicera's case, although all patients showed increased GCase levels in their white blood cells, only one had clinically meaningful disease improvement and that's why they decided to stop it and not go on to ph III. As always, having a full time job doesn't let me much free time and I may be missing some information. I don't quite understand why they decided to start ph III trials with Amigal...I'll be happy to hear your thoughts on this.

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#52) On July 23, 2010 at 4:48 AM, portefeuille (98.92) wrote:

I don't quite understand why they decided to start ph III trials with Amigal

They gave an explanation in their latest presentations (see comment #35 above).


For example when one performs the Phase III primary end point analysis on the patients from the Phase II study who meet the Phase III entry criteria, the result is very encouraging.


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#53) On September 09, 2010 at 7:50 AM, 1337172979 wrote:

Right! he describes why they decided to go on to ph III, but what he actually says is that there were no problems in kidney function and and that the enzyme levels increased, which was what happened to Plicera. We can conclude thus far that the drug is safe and well tolerated. Improving renal funciton seems to take very long...

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#54) On October 29, 2010 at 10:35 AM, portefeuille (98.92) wrote:

GSK and Amicus Therapeutics Enter Exclusive Worldwide Agreement to Develop and Commercialize Amigal for Fabry Disease

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