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Vertex Pharmaceuticals Incorporated Message Board

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  • VRTX was just upgraded by UA (underground stock alerts- google them ) to a BUY with a new price target, LOOK WHAT IT IS!!! that's HUGE. i'm subscribed to UA's free newsletter, their subscriber base is huge, the last time they alerted a buy the stock ended up going up 1350% in a very short period, no way this will happen with our stock but it's still very positive. i've made over $200k with their free alerts so far so it's definitely not something to sneeze at.

  • VRTX was just upgraded by UA (underground stock alerts- google them ) to a BUY with a new price target, LOOK WHAT IT IS!!! that's HUGE. i'm subscribed to UA's free newsletter, their subscriber base is huge, the last time they alerted a buy the stock ended up going up 1350% in a very short period, no way this will happen with our stock but it's still very positive. i've made over $200k with their free alerts so far so it's definitely not something to sneeze at.

  • Of course I have mommy-colored glasses on, but nothing he says gives me pause: We always knew that in vitro the combo wasn't getting a ton of correction (to 30ish percent of CFTR function). And yes, in vitro doesn't always translate to clinic, but for the Vertex meds it has had a good correlation (no misses so far). Yes, it is complex, which is why they'll likely have 3 correctors eventually. The US conference had great presentation on the various defects and complexity which 809 only addresses one. None of that changes what Phase 2 showed as "good" results and which 661 replicated nearly exactly. While I'd love a 10% FEV improvement, I expect between 4 - 5%, but that should be good enough for approval, especially when coupled with weight gain and other improvements. Two final points: The EU doctors in general seem to be more pessimistic or reserved and as for the lack of anecdotal evidence: The study is blinded and participants know not to talk. One who blogged on 661 appeared may have been kicked out of study. Most CFers aren't going to risk it by talking because they need this trial!

  • So some more digging and I'm somewhat baffled. The downgrade is to market perform with an unchanged target of 76. if the combo is going to fail, thats horribe advise! http://www.streetinsider.com/Downgrades/Bernstein+Downgrades+Vertex+%28VRTX%29+to+Market+Perform/8833127.html. This summary also doesnt indicate failure and to me indicates a misunderstanding of 661 which isn't 809s successor for dual combo, but only triple. http://blogs.barrons.com/stockstowatchtoday/2013/11/01/vertex-pharmaceuticals-filling-in-the-gap/
    Then this summarizes some of the reasons http://www.fiercebiotech.com/story/bernstein-analyst-predicts-likely-flop-vertexs-crucial-combo-study/2014-06-16 which also seem flawed. We knew of neg interaction with 770 and 809 which is why 770 is dosed higher. And this implies success when it speaks of concern for reimbursement out of us. It is also nonsense to say it will hold up 661 which is already in trials and has break through therapy, so if the concern with 809 is interaction, 661 addresses that. But it also makes no sense given near mirror results of 809 and 661. A hold recommendation based on uncertainty makes sense but saying the trial is going to fail but with a hold and 70 price target is internally inconsistent so either he's an idiot or the spin of failure is inaccurate.

  • Nothing better during Independence week than that sweet smell of BBQ and Shorts Burning on VRTX!
    Reward the science not the pessimism and the hope for meaningful treatment for real sufferers of CF.

    Sentiment: Strong Buy

  • Analyst Rating Network
    6/25/2014 Leerink Swann Boost Price Target Outperform $125.00

  • From an article posted on the Gladstone Institue web site:

    Once the scientists discovered this key process, as described in Nature, they began to investigate how the body senses the fragments of HIV’s DNA in the first place, before alerting the enzyme caspase-1 to launch an immune response in the CD4 T cells. To identify the so-called DNA sensor, the scientists found a way to genetically manipulate CD4 T cells in spleen and tonsil tissue. In doing so, they discovered that reducing the activity of a protein known as IFI16 inhibited pyroptosis, explained Zhiyuan Yang, PhD, a Gladstone postdoctoral fellow who is one of the paper’s two lead authors.

    “This identified IFI16 as the DNA sensor, which then sends signals to caspase-1 and triggers pyroptosis,” says Kathryn M. Monroe, PhD, the Science paper’s other lead author, who completed the research while a postdoctoral fellow at Gladstone. “We can’t block a process until we understand all of its steps—so this discovery is critical to devising ways to inhibit the body’s own destructive response to HIV. We have high hopes for the upcoming clinical trial.”

    The Phase 2 trial—which will test an existing anti-inflammatory’s ability to block inflammation and pyroptosis in HIV-infected people—promises to validate a variety of expected advantages to this therapy. For example, by targeting the human body, or host, instead of the virus, the drug is likely to avoid the rapid emergence of drug resistance that often plagues the use of ARVs. The anti-inflammatory may also provide a bridge therapy for the millions without access to ARVs, while also reducing persistent inflammation in HIV-infected people already on ARVs. Many suspect this inflammation drives the early onset of aging-related conditions such as dementia and cardiovascular disease. By reducing inflammation, the drug might also prevent expansion of a reservoir of latent virus that hides in the body where it thwarts a cure for HIV/AIDS

  • They are often times wrong than right.

    The truth is that most analysts work for large companies with large stakes in certain stocks. They must give those stocks good (or bad) recommendations to maintain some profit for their company.

    The true masters (Buffett, Klarmen, etc) just buy the stock that they believe in. They put their money where their mouth is.

    I could be wrong but the VX-770 and VX-809 combination had some strong data to support going into phase 3. In the end you can theorize all you want about what the results are but it is the hard clinical outcome that speaks the loudest, because you really don't know fully what goes on inside us, due to the complex nature of the human body when it interacts with chemicals. The phase 2 data was pretty convincing which showed a 8.6% improvement in FEV1 in the VX-770 +VX-809 arm compared to placebo at week 7. Ivafactor showed a 10.6% improvement at week 24 compared to placebo. So if there was no difference, the difference seen at week 8 had to disappear which can happen but not sure how I can explain it. If it were drug-drug interaction I am not sure you would've seen the differences in week 8. Poor selection of patients could also explain but that would be such a rookie mistake (although it can happen). It is also a double blind study so I am not sure what he heard in the conference, but it is pretty much impossible to predict the results of the study, even if you are the investigator. This is such a large study and I am not sure who he talked to and what he heard but unless it was something illegal, I don't know why people are paying so much attention to this analysts based on what he heard in a conference.

    I could be wrong but this is just so weird. We will see. I am VRTX long.

    Sentiment: Strong Buy

  • Tip Ranks
    Michael Yee RBC Capital
    6/25/14
    Vertex Pharm. (VRTX ) Buy Rating

  • Pancreatic cancer cell line data is superior to any other drug combo I have known. Have you ever seen 106 patients in phase 1 trial? It shows their confidence on this therapy. They have been running trial under the radar since 09/2012 in England and have very positive reviews. I smile when people talk about CF franchise. and reimbursement. Do people have any idea about the history of CF and drugs that have failed CF treatment. Remember CF gene therapy flu virus vector failure?

  • As Rojospan found out, Vertex is running a clinical trial of VX-970 which potentiates tumor destruction by inhibiting tumor DNA damage repair. The targeted patients have advanced squamous non-small-cell lung carcinoma, or small-cell lung carcinoma. Surgery or radiation is out of question for these patients although for localized solid tumors VX-970 would be an ideal drug to potentiate radiotherapy.
    VX-970 is an inhibitor of ATR, which along with ATM and DNA PK is a signaling molecule to trigger DNA damage repair and also to stop cell-cycling. Etoposide, gemcitabine, and/or cisplatin are DNA damaging agents to be given along with VX-970. The scientific basis of this trial is highly impressive, which was published a month ago. The management will probably disclose the cancer program along with VX-765 based AIDS treatment trial, and MS program.

  • Cystic Fibrosis - Quest For Kalydeco

    May 14
    .
    Some GREAT inside info from someone who likes our page for those Cystic Fbrosis double F508del people.............."My daughter carries the two copies of delta 508 just before Christmas of 2012 she was really bad she had an infection that was not responding to antibiotics she came home for Christmas and then in January she had a turn for the worst her body couldnt fight the infection she stayed in hospital for 8 weeks she had numerous pneumothoraxs ended up on a ventilator and it was touch and go for a few days but she pulled through it then when she got a bit better came the conversation that we all dread from the consultants its time to start discussing lung transplants my daughter said "im not ready for this" and started to fight again as all cfs do she managed to get het lung function to 38% then a few months after we came of holiday we saw a new trial that was going to be taken place the trial for kalydeco and lumocaftor she had to blow 40% to get on the trial which she did but it was a double blinded study for 6 months. After the 6 months which ended in march she was given the active drug ....so to cut a long story short we went to clinic today sats was 97% weight up to 55 kilos and lung function was a whopping 59% so please whoever decides who gets these drugs please look at the facts of how lives are changing look at the faces of the families the relief it brings to the cfs knowing that they can breathe better and then tell us that its not being funded due to costs. My daughter has not been in hospital for 12 months and has not had any ivs in 12 months she is enjoying life and thats what lifes all about. It really is like looking at a different person!" .... What a fantastic message. Thank you!!)

  • One rationale for his view the combo will fail is the in vitro results showing kalydeco causes instability..."First, it now appears that Vx770 (Kalydeco) shifts the temperature stability curve for DelF508 CFTR downwards (counteracting positive effect of Vx809). Second, with prolonged exposure to Vx770 in cell culture the stability of CFTR corrected by Vx809 appears to erode," notes Porges. "Third, as previously described in the literature, indeed Vx809 appears to be a so-called class II corrector, binding at the interface of the NBD1 and MSD1/MSD2 domains of the protein. This has limitations; stabilizing the protein at this site has relatively modest activity in isolation, and the addition of a second corrector to either stabilize the NBD1 domain directly, or to act as a pharmacological chaperone, appears to be necessary to restore clinically relevant levels of CFTR function."

    At first I thought he was talking about interaction between 770 and 809 but on further thought the problem is with kalydeco and df508 as these quotes show. Not only though do we have phase 2 studies shaping clinical benefit with 809 and 770, but we have the recent 661 kalydeco trial with 551 and df508 heteros which showed an additional stat sign benefit. 661 works on same nbd1 domain as 809 and kalydeco has same instability causing properties on df508 allay so the additional instability does not outweigh the corrective action. Thirdmeinvestor I'd love your thoughts on the science!

  • Reply to

    oversold

    by helpallaretaken Jun 16, 2014 3:22 PM

    Oversold...yep, but unless good new comes out soon, it will get worse, here's a few reasons why;
    1) Investors are anxious. Everyone knows Vertex is near their announcement date. People will make/lose LOTS of dough no matter the outcome...(shorts made money today because of irresponsible and negative based reporting - spin is everything, right?)

    2) K+ 809 is [foolishly] being compared to Kalydeco in 551 patients. DD5008 patients ARE NOT 551 patients! Each CF patient must be treated individually. (To compare 551 patients to DD508 is blatant discrimination)

    3) Expectations for people from outside the CF world are misunderstood & should be ruled non-factors. Yet thanks to bloggers without any scientific merit, these people receive attention for their misleading reports.

    4) Misunderstood advantages: As a CF-Father, that's me, one who works deeply in the biopharmaceutical industry (a new fact for those who have read my occasional posts), & I speak for thousands...we would walk 2,000 miles or more on broken glass to keep any person with CF out of the hospital, off antibiotics, steroids, mucus thinners, AND SO MUCH MORE. How hard is this to understand? Do people including investors know how much it costs to put a CF patient in the hospital 2-5 times per year? How about a lung transplant? Nah, investors don't care...I get it.

    5) Negative non DD-508 patients and caregivers exist. Some don't grasp that a K+809 approval makes it easier for the FDA to move 661 quickly through the FDA process b/c clinical biomarkers will be set! Yep...it's true!

    Last word, unless there's a tox issue; (even with as little as 0% FEV1 improvement...yes 0%) K+809 MUST be approved - especially if exasperations & hospitalizations have reduced + there's been improvement in weight gain. The cost of keeping people off other meds and out of the hospital is simply much more than can be imagined.

    Sentiment: Strong Buy

  • "It's not their job to "save the stock""....I'm not sure if you're daft or joking but the primary responsibility(i.e. Fiduciary Duty)of management is to "maximize shareholder wealth". The mandate of the scientists is to bring to market commercially viable drugs.
    Investors own the company and management works at their behest. All R&D is done on the investor's dime with the expectation of a return commensurate with risk. It's a model that has worked for centuries and if you'd like to research it, it's called Capitalism. I think your reference is to charity, which is also a very admirable, although less efficient, means of financing research.
    I'm hopeful this is a prime example of the successful way in which nvestors seeking return are able to facilitate true good. But spare me your misguided sentiment that management isn't responsible to shareholders.

  • The value of VX-809 is in its long term data. The study showed that it works. If it continues to work, the clinical benefits will amplify, like ivacaftor, with long term use.

    VX-809 is the bridge to VX-661. That is where the true value of VRTX's CF pipeline is.

    I am VRTX long.

    Sentiment: Strong Buy

  • Reply to

    Adam knows his stuff.. going higher

    by biotraderman Jun 25, 2014 12:17 PM

    The share price could double in 18 months from the sales of Kalydeco and VX-809.

  • Reply to

    Adam knows his stuff.. going higher

    by biotraderman Jun 25, 2014 12:17 PM

    Yep, $90 is gonna be a bargain in 12 months. that's why the biggies are try to push the price down to get people to sell them their shares. they know there is massive upside in the future. VRTX is the next GILD.

  • Sure looks like it is tanking right now....oh wait nvm its up $2.60 today alone, about to break $100. Better cover!!!!!!!

VRTX
92.80-0.23(-0.25%)Aug 28 4:00 PMEDT

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