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Aegerion Pharmaceuticals, Inc. (AEGR) Message Board

zwerp2000 75 posts  |  Last Activity: 33 minutes ago Member since: Apr 19, 1999
  • zwerp2000 by zwerp2000 Jun 26, 2014 9:13 AM Flag

    June, 25, 2014 – Today is a landmark day for PPMD and the entire Duchenne community. We are so pleased that at our organization’s 20 year anniversary – after 2 decades of hard work and resolute persistence within the policy, legislative and regulatory arena – we are today able to provide to the US Food and Drug Administration the first-ever patient-initiated guidance to help accelerate development and review of potential therapies for Duchenne muscular dystrophy.

    This guidance is a major milestone for the community and truly incorporates the patient voice – the community’s voice – in well-documented and quantifiable ways in pursuit of our common goal – to end Duchenne.

    Why Develop a Guidance?

    In February 2013, the European Medicines Agency (EMA) developed a guideline document focused on the “clinical investigation of medicinal products for the treatment of Duchenne and Becker muscular dystrophy.”

    We felt that the FDA should have similar guidance and that our well-versed and passionate patient community could come together to provide as much input as possible to inform the document. On December 12, 2013, we convened a national PPMD-FDA Duchenne Policy Forum – with 20 FDA staff members in attendance – where the community made its needs and preferences in drug development known to the Agency. An agreement was reached that we – the Duchenne Community – led by PPMD, would develop the first-ever patient initiated draft guidance on Duchenne.

    After an intensive five month long process, overseen by a steering committee, developed by working groups, and reviewed by a community advisory board, today we officially submitted to the FDA the Duchenne muscular dystrophy community’s draft of the Guidance for Industry: Duchenne Muscular Dystrophy: Developing Drugs for Treatment over the Spectrum of Disease, written to help accelerate the development and review of potential therapies for Duchenne.

    Community Collaboration

    We had over 80 dedicated expert stakeholders working toge

  • Sequential Brands price target raised to $19 from $11 at Canaccord
    Canaccord raised its price target on Sequential Brands following its acquisition of Galaxy Brands, citing expectations for increased licensing revenue and the execution of management's growth strategy through M&A. The firm keeps its Buy rating on the shares.

  • zwerp2000 zwerp2000 Jun 16, 2014 11:31 AM Flag

    easy there fella. i've seen the videos and read the tweets from some of these parents which gives me confidence the data will be strong. however, there may be some patients who plateaued or could have slightly declined. there is such the possibility. however, my point is that even if there is decline by some, people need to realize this is a progressive disease and thus far no drug has been able to stop this progression aside from eteplirsen. we need to look at the natural history and compare those kids who "may" decline to how much of a decline kids usually have without the drug. either way, the results will be remarkable.

  • zwerp2000 zwerp2000 Jun 16, 2014 10:57 AM Flag

    that's why i said "some" of the parents. however, given the size of the patient trial, hearing from about 3 or 4 is pretty substantial and significant.

  • zwerp2000 zwerp2000 Jun 16, 2014 10:50 AM Flag

    well, as we go forward, there is a decent chance the data could drop off a bit which shouldn't be a total shock. dmd is a progressively debilitating disease where there has been no drug yet, aside from eteplirsen, that has shown to slow the progression of this disease. i don't know if eteplirsen is a 100% cure but if it is slowing the diseases down considerably based on historical data then in a perfect world, the stock should react positively. hell, companies that manage to slow the progression of cancer by a few months are hailed as the holy grail yet when we have unprecedented results from eteplirsen, traders scoff at the news and perceive it as negative. bizarre.

  • zwerp2000 zwerp2000 Jun 16, 2014 10:46 AM Flag

    I agree. For me, i follow the tweets of some of the parents and it appears their children are doing phenomenal . I don't know how the others are doing but from what i've read, i am hoping from good data

  • zwerp2000 zwerp2000 Jun 16, 2014 10:37 AM Flag

    What we will likely need with the 144 wk results, if possible, is the individual patient scores and data. If there are some patients who actually improved and some who remained the same or declined slightly (which goes against natural history), we will have some clarity on the true numbers. I think it;s a huge win for SRPT, although the stock would likely get hit, if the children remained just stable or decline slightly. No drug has been able to stabilize the disease so just doing that or slowing the progression is a big win. Companies with cancer drugs that can just slow the disease slightly get richly rewarded. Eteplirsen appears to do so much more than that. Again, you have to wonder what would happen if we gave this SAFE drug earlier in their lives and at higher doses.

  • 1. This board is a mess. Some of the better posters do not really post here anymore. I can't blame them. There are some that continue to post useful info. I hope they continue to do so

    2. Everyone is waiting for the 144 wk data. What will it reveal? Well, if there is some distance loss/decline by some of the patients on the 6 mwt i expect the shorts to pounce and the stock could tumble. Of course, for me, i want to see that if there is a drop by some or even all, how much and if the patients continue to show stability. Remember, no drug has ever shown to stabilize this disease. Additionally, one could argue that if this drug is given to these patients at an earlier age, before any damage is done, then perhaps they would never really deteriorate or if they did, it would be so slow that it would add years and years to their lives. Also, what if the dosage administered was higher? Maybe that would help even more as the drug appears safe. It's amazing that a company could be worth billions and billions just to come up with a cancer drug that adds a few months to a persons life but SRPT continues to remain severely undervalued

    3. Speaking of valuation, i don;t know why wall st seemingly hates this stock so much. Look at BLUE today on data based on 2 patients. This data is extremely early, not even close to getting approval like SRPT is and yet both have similar market caps. Based on SRPT's data and the fact that they have now been invited to file an NDA, i don;'t understand why the stock does not have a market cap in the multi-billions. any other stock would have such a large valuation. Hopefully this will change soon but boy does wall st seem to hate this company. Of course there are some very big institutions buying in so i am sure much of the depressed stock price has to do with typical wall st BS.

  • Reply to

    Todays 8K filing

    by golderw3 Jun 12, 2014 6:10 PM
    zwerp2000 zwerp2000 Jun 12, 2014 8:10 PM Flag

    you seem to have a good handle on the science. I presume you are expecting strong results this weekend?

  • Reply to

    Data must be excellent

    by golderw3 Jun 12, 2014 2:11 PM
    zwerp2000 zwerp2000 Jun 12, 2014 2:45 PM Flag

    Me too

  • Reply to

    the turd just went red as expected

    by devilsyankeesfan Jun 11, 2014 10:34 AM
    zwerp2000 zwerp2000 Jun 11, 2014 11:03 AM Flag

    shut up already

  • TGTX) ahead of the pharma's presentation at EHA over the weekend.

    Analyst Joseph Pantginis is looking for stronger visibility from the TG-1101/Imbruvica Phase II in B-cell malignancies. The analyst comments, "We expect TG to shine this weekend with what we have called the "match made in heaven" study. We believe these combination to point toward major response rates in the absence of chemotherapy. Depending on the indication (MCL vs. CLL/SLL) Imbruvica monotherapy has delivered overall response rates in the 67-83% range. We believe the underlying hurdle for the combination regimen would need to therefore be in the 90% range or greater. We believe this combo regimen could yield a true "mopping up" strategy in reducing residual disease in patients. We would project that much higher response rates would also have a positive impact on extending survival. There has been some "pre-excitement" regarding this study with some rumors flying around that Pharmacyclics would end up just acquiring TG. To date, though TG has strongly emphasized their goal in playing a major role in marketing its drugs."

    "The Phase II '1101/Imbruvica study is enrolling up to 60 patients with r/r B-cell malignancies (at least one prior treatment regimen). The primary endpoint of the study was to assess the safety of the combination. The secondary endpoint of the study is overall response rate (ORR) up to one year," Pantginis said.

    For an analyst ratings summary and ratings history on TG Therapeutics click here. For more ratings news on TG Therapeutics click here.

    TG Therapeutics closed at $6.61 yesterday.

  • Roth Capital affirms its Focus Pick rating and $15 target price on TG Therapeutics, Inc. (Nasdaq: TGTX) ahead of the pharma's presentation at EHA over the weekend.

    Analyst Joseph Pantginis is looking for stronger visibility from the TG-1101/Imbruvica Phase II in B-cell malignancies. The analyst comments, "We expect TG to shine this weekend with what we have called the "match made in heaven" study. We believe these combination to point toward major response rates in the absence of chemotherapy. Depending on the indication (MCL vs. CLL/SLL) Imbruvica monotherapy has delivered overall response rates in the 67-83% range. We believe the underlying hurdle for the combination regimen would need to therefore be in the 90% range or greater. We believe this combo regimen could yield a true "mopping up" strategy in reducing residual disease in patients. We would project that much higher response rates would also have a positive impact on extending survival. There has been some "pre-excitement" regarding this study with some rumors flying around that Pharmacyclics would end up just acquiring TG. To date, though TG has strongly emphasized their goal in playing a major role in marketing its drugs."

    "The Phase II '1101/Imbruvica study is enrolling up to 60 patients with r/r B-cell malignancies (at least one prior treatment regimen). The primary endpoint of the study was to assess the safety of the combination. The secondary endpoint of the study is overall response rate (ORR) up to one year," Pantginis said.

    For an analyst ratings summary and ratings history on TG Therapeutics click here. For more ratings news on TG Therapeutics click here.

    TG Therapeutics closed at $6.61 yesterday.

  • Reply to

    If J Napadano is right then"....

    by mdexpert69 Jun 4, 2014 8:33 PM
    zwerp2000 zwerp2000 Jun 9, 2014 9:51 AM Flag

    Ooops. Complete miss

  • Reply to

    Have You Ever Seen Growth Like This?

    by rbullock47 Jun 8, 2014 1:52 PM
    zwerp2000 zwerp2000 Jun 8, 2014 2:36 PM Flag

    Yes, when I get an erection

  • Reply to

    Big Article on Ibrutinib on Yahoo

    by korthh Jun 4, 2014 4:55 PM
    zwerp2000 zwerp2000 Jun 5, 2014 7:56 AM Flag

    I never said they were working together. I said TGTX was using PCYC's drug in combination with their drug

  • no surprise

AEGR
30.00+0.91(+3.13%)Jul 25 3:59 PMEDT

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