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Amicus Therapeutics, Inc. (FOLD) Message Board

tredleon 138 posts  |  Last Activity: Sep 19, 2014 10:11 AM Member since: Sep 21, 1999
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  • tredleon tredleon Sep 19, 2014 10:11 AM Flag

    Try again bubba - today's presentation is the only listing under the Upcoming Events calendar. DUH!

  • tredleon tredleon Sep 19, 2014 9:50 AM Flag

    How stupid of the company to post the "Event" and the streaming link on their website when nobody can get access. It is unusual for these scientific presentations to be available to investors - that is why most companies don't schedule them in the Investor section of their website.

  • Reply to

    wife of p3 trial participant

    by mclim73 Sep 17, 2014 11:40 PM
    tredleon tredleon Sep 19, 2014 8:45 AM Flag

    maddog - The STS trial began in Sep 2011 (and finished enrollment in Dec 2013), so the longest a patient could have survived in the trial is roughly 36 months, which is not dramatically outside the "normal" range for an expected median of 22 months? In any case, as I indicated before, I don't think the survivors come into play at all in the interim look - I think the IDMC can only assess the results based on those that have passed. If a larger portion of the patient deaths are in the control group, I'm sure they have the ability to assess enrollment/randomization stats and verify there are no imbalances in how each arm has enrolled. Either way, if TH-302 is benefiting the majority of patients, the assessment of median survival for those that have passed will reveal that. If TH-302 is somehow only benefiting a sub-set of patients, that may only be revealed in the final analysis.

  • Reply to

    wife of p3 trial participant

    by mclim73 Sep 17, 2014 11:40 PM
    tredleon tredleon Sep 19, 2014 7:25 AM Flag

    dtic - no doubt, this has taken some patience. My hope was that some of the earlier trials in GBM, MM, etc. might draw some attention to how "broad" the potential for TH-302 is outside pancreatic and STS, but it has been a slog. If this interim look at STS isn't a big win, we are looking at another year of waiting for the payoff.

  • tredleon tredleon Sep 18, 2014 4:01 PM Flag

    The flaw in your thinking is that treating sick patients will "stop the outbreak" - the outbreak can only be stopped by carefully managing those exposed as quickly as possible, so it does not spread. Treating them after they have potentially exposed other people to the virus won't change the spread equation.

  • Reply to

    wife of p3 trial participant

    by mclim73 Sep 17, 2014 11:40 PM
    tredleon tredleon Sep 18, 2014 2:58 PM Flag

    I get your point, and I'm sure if the IDMC saw that there were no patient deaths in the treatment arm, they would have stopped the trial even before the targeted interim look at 224 "events". Theoretically, if the randomization/enrollment was done evenly from the get go, there should be an imbalance in the # of deaths in the TH-302 arm vs the control arm - e.g. the control arm could have 120 deaths and the treated arm could have 104 deaths - but the median survival computed on those 104 patients should reveal that differential, if they are truly dying slower. The fact that most cancer trials involve marginal improvements (20% or less) in survival dictate that you can't "extrapolate" a median by including survivors at an interim look - whatever imbalance that may be there at the interim may not hold up at the end or there may be just as many "long-tail" survivors in the control arm?

  • Reply to

    wife of p3 trial participant

    by mclim73 Sep 17, 2014 11:40 PM
    tredleon tredleon Sep 18, 2014 1:54 PM Flag

    Only those that have passed are included in the computation of the interim median. That is why it is usually very difficult to generate a positive result at an interim look (besides the higher hazard ratio required), as the interim results can be dominated by the sickest patients that pass the quickest. If there is a "long tail" of patients in the TH-302 group (i.e. patients that live well beyond the median), their impact on the median may not be revealed until the final data is out.

  • tredleon tredleon Sep 17, 2014 6:36 PM Flag

    You are correct, with one caveat - it was a single-arm trial and the 21.5 mo median OS was compared against the 12.8 mo median OS from the control arm (Dox only) of another study. Supposedly, median OS for Dox-only in STS is historically in the 8-12 mo range, so the 12.8 comparison is hopefully the high end of that range? They stated at a recent presentation that they are not aware of any recent changes in "standard of care" that would cause survival in STS to be higher than the historical studies.

  • Reply to

    70 Million $$$

    by bridgeconstructor Sep 17, 2014 10:39 AM
    tredleon tredleon Sep 17, 2014 10:58 AM Flag

    But it will take $70M more to bring it to market, assuming a future pivotal trial is successful. The issue isn't what they have, it is what they don't have - i.e. the capital necessary to carry forward. With stellar results in one of the pending trials, they may be able to partner, but they are in such a weak bargaining position, any deal they would reach at this stage would be one-sided. Better to sell the whole company, but again, they would be selling from a position of weakness, even if they find someone that believes in REO.

  • Between the company's enthusiasm for this drug and its prospects and the market's response to its progress/prospects. It seems every trial provides nuggets of hope for future trials to potentially validate, but the completed trials have issues which leave the results inconclusive - with H&N it was the early termination of patient dosing due to fever, with pancreatic it was wild-type KRAS patients distorting the results - and since the remaining trials are not under the company's control, you can probably guess that there will be issues with those, as well. In the long run, Reo may find a niche in many different cancer treatments, but that is years away from validation, assuming they can find the money (or buyer) to carry on.

    On a side note, all the remaining active trials are investigator-sponsored and considering that every question raised by the analysts was met with "we're not sure, because it is not our trial", what exactly do these guys do all day?

  • Reply to

    CC says interim couple of weeks.

    by mclim73 Sep 9, 2014 12:36 PM
    tredleon tredleon Sep 10, 2014 6:49 AM Flag

    What's "more better" than a moronic short who not only doesn't understand how the IDMC review works (the company never has access to the data), but uses infantile phrases like "more better"?

  • tredleon tredleon Sep 9, 2014 6:00 PM Flag

    No, they won't reveal it if it is not achieved, because the company won't know - unless it is achieved, the company is blinded from the data. The CEO keeps downplaying the odds of getting the 45%, while trumpeting the Phase II data that achieved a 68% increase vs historical (21.5 vs 12.8), so it is not out of the realm of possibility. No doubt, Phase II benefits typically narrow in the stricter constructs of a controlled Phase III trial, but you would think that if the control group survival is 12 months, then 17.5 months seems plausible?

  • Reply to

    Is THLD a BUY?

    by afalcone7 Sep 8, 2014 9:11 PM
    tredleon tredleon Sep 8, 2014 9:23 PM Flag

    I agree with the conclusion of the article, that THLD is a low-risk, high reward stock, but the article is incorrect in stating that they will be releasing preliminary data from the STS trial in the near future. The pending review by the IDMC will either stop the trial due to overwhelmingly positive data, in which case the data would be released, but if the IDMC decision is to simply let the trial carry on to its designated final data point, then no data will be made available - the company will remain blinded to the data. The analysts that recently began coverage were not counting on an early termination in establishing their double-digit price target, but the market can react negatively if enough momentum players have bought the stock hoping for an early termination?

  • Reply to

    Next catalyst

    by jakebanner1789 Sep 5, 2014 12:48 PM
    tredleon tredleon Sep 6, 2014 7:07 AM Flag

    Nice recap - assuming the STS trial carries on, then hopefully there will be some updates toward year-end on the glio and other trials combining TH-302 with the anti-angiogenics.

  • Reply to

    We are all suckers on this board

    by spiritofbudrolfs Sep 5, 2014 10:42 AM
    tredleon tredleon Sep 5, 2014 10:53 AM Flag

    What's more meaningul, the 3M+ shares traded yesterday above $23 or the 0.5M shares traded today below $23? Answer - neither - it is all just noise until AA is realized next year.

  • Reply to

    Thesis: Approval for Prosensa and Sarepta

    by waugusti Sep 5, 2014 7:08 AM
    tredleon tredleon Sep 5, 2014 8:41 AM Flag

    Conclusion: You're an Idiot - the idea that Etep (or any effective DMD) drug would be restricted to a certain age group is just plain stupid.

  • Reply to

    FDA Guidance Just Released

    by greyzone513 Sep 4, 2014 1:03 PM
    tredleon tredleon Sep 4, 2014 8:28 PM Flag

    My take on the FDA Guidance issue is that the PPMD made it crystal clear in their proposed guidelines that the risk profile of DMD is such that there is no risk that is not worth taking, in light of the 100% certainty of progression (with no potential reversal - once muscle is destroyed, there is no restoring it) toward a gruesome life and certain early death if this disease is not stopped in its tracks at an early age. It seems the FDA is finally getting the message in their expression to be more "flexible". Combine that with the fact that FDASIA does not require certain documented clinical benefit, but only the reasonable likelihood that the treatment provide benefit, and we are starting to tilt toward AA being a slam dunk. A successful 4th biopsy (presumably with newly approved measurement protocols) would certainly put AA over the top, but even if that does not happen, it is hard to imagine any "expert" looking at the lack of progression/improvment in Max, Billy, etc. and concluding that there isn't a reasonable likelihood that Etep is providing them clinical benefit. The idea that 100% of the treated boys have to show the same level of benefit (per the 6MWT) for AA to be likely is simply ridiculous - the experts will understand that issue and drive it home, if need be.

  • Reply to

    What happened -- anyone?

    by smithts777 Sep 4, 2014 11:31 AM
    tredleon tredleon Sep 4, 2014 3:11 PM Flag

    Big investor decided to bail before the inevitable dilution. As we head into Oct and the fiscal year-end of most mutual funds, they will be dumping losers in order to "window dress" their portfolios for the year-end reports - they don't want to issue a report to investors showing they are holding a position in a stock that has lost half its value, or more.

  • Was posted on Jenn's FB showing Max walking off to catch the bus for his first day of 6th grade - looks pretty normal, even burdened by a full backpack. This is not a kid who is struggling to stay ambulant, even though before starting the trial 3 years ago his mom was pulling his brother's scooter out for him to use. Sure, Etep doesn't work!

  • Reply to

    EXEL

    by beachbumsoon325 Sep 2, 2014 4:52 PM
    tredleon tredleon Sep 2, 2014 5:52 PM Flag

    They already have a marketed product - that rule doesn't apply.

FOLD
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