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Vertex Pharmaceuticals Incorporated Message Board

qdelfan 84 posts  |  Last Activity: Jul 11, 2016 2:19 AM Member since: Sep 30, 2007
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  • Reply to

    mrna technology

    by qdelfan Jul 6, 2016 10:23 AM
    qdelfan qdelfan Jul 11, 2016 2:19 AM Flag

    Thanks Third, for your assessment of these new platforms, and their potential challenges to overcome to be successful. Of course 15 years ago. there was lots of doubt about the likelihood of small molecule CFTR potentiators or correctors as well. If the Vertex HBE in vitro assays show successful production of CFTR using gene editing or mRNA insertion techniques in treated CF HBE cells, I would hope that nuclease degradation would not be too much different in the human body. I look forward to hearing about these test results in the coming years to see if these technologies can possibly deliver on their promise.

  • Reply to

    mrna technology

    by qdelfan Jul 6, 2016 10:23 AM
    qdelfan qdelfan Jul 6, 2016 5:35 PM Flag

    Third what are your impressions of the prospects of the mRNA platform from Moderna Therapeutics to treat CF versus gene editing technology licensed from CRSPR?

  • qdelfan by qdelfan Jul 6, 2016 10:23 AM Flag

    David Altshuler, VRTX CSO, announced VRTX is partnering with yet another company with a novel cutting edge approach to treating CF. So now VRTX has commercial rights to Pharion's EnAc drug, CRSPR's technology, and Moderna's mRNA platform to test it's ability to improve CFTR function. Judging from VRTX stock price reaction, the analysts apparently like the news.

  • On increased volume DXCM share price performed so well today in spite of market turmoil. I look forward to DXCM approaching new 52 week highs as the FDA Adcom meeting to approve the G5 sensor for insulin dosing without fingersticks approaches next month.

  • qdelfan qdelfan Jun 21, 2016 2:32 AM Flag

    JPMorgan provided its bullish outlook on DexCom, Inc.
    DXCM 6.39%
    on Monday. The bank believes there is an 80 percent chance of positive vote in the upcoming FDA Panel, scheduled on July 21.

    According to JPMorgan, they're confident in their view of positive outcome after conducting number of interviews with company representatives, industry experts, regulatory consultants, and thoroughly reviewing the data. The analysts see a stock price increase of 25 percent to the year-end in case of positive panel support of FDA approval, and a rise of 15-20 percent if the panel considers the current data insufficient and decides to wait for additional trials.

    FDA approval will give Dexcom a non-adjunctive/fingerstick replacement label, which "would mean that G4 and G5 would be approved for patients to make insulin dosing decisions off of the CGM readings. Dexcom already has this label in Europe with G5."

  • qdelfan qdelfan Jun 21, 2016 2:29 AM Flag

    Actually JP Morgan analyst report is the reason the stock moved up on rising volume

  • Listen to the replay or read the transcript. Very positive growth story over the next few years. I would not be surprised to have a takeover offer from a larger diagnostics company e.g. JNJ or Abbott in the next 12 months.

  • FDA meeting to weigh whether Dexcom's G5 CGM can replace fingersticks for treatment decisions

    By Brian Dolan
    June 06, 2016

    Next month the FDA is hosting a public advisory committee meeting to discuss a change to the intended use of Dexcom's G5 Mobile Continuous Glucose Monitoring System (CGM) device that would allow the company to market the G5 as a CGM patients can base treatment decisions on.

  • Well worth listening to. Dexcom is several steps ahead of their competition. Historically, Dexcom's product accuracy and innovative connectivity has distinguished their product. Their next gen products soon to be released will be even more desirable in terms of smaller size of unit and even greater ease of use. Their Google partnership will open up the type 2 DM market with a much greater growth potential in the years to come..

  • Stock would get more positive attention from analysts if a thought leaders in CRC treatment at ASCO commented on and endorsed the significance of the MM-151 data.

  • qdelfan by qdelfan Jun 3, 2016 12:01 AM Flag

    Expect positive phase 3 trial data presentation at EUCF meeting in Switzerland next week for orkambi treatment of 508dd cf patients ages 6-11. Analyst covering VRTX will likely raise revenue and eps estimates in the coming quarters as sales of orkambi ramp higher when the 2500 US patients with 508dd mutation in this age group will likely be prescribed treatment quickly. This ramp up in sales will be due to demand from parents when the word is out their Cf children, (with less severe pre-existing lung damage compared to older CF patients) are more likely to have less severe initial chest tightness symptoms and more immediate benefit from treatment. Slowing progression of CF at a younger age by starting treatment of the underlying cause of CF with Orkambi will likely prevent the more severe lung damage seen in older CF patients. That's the path to a more normal adult life for these children and their parents will be eager to get them on treatment if it is more easily tolerated in the first months of treatment compared to the older and sicker 508dd CF patients currently approved by the FDA for treatment with Orkambi.

  • Reply to

    GLPG/AbbVie bet on triple combo

    by papaalien2000 May 6, 2016 5:09 AM
    qdelfan qdelfan May 8, 2016 3:33 AM Flag

    EU CF meeting next month should give insight into efficacy of VX 661/770 as Phase 3 studies from VRTX are reported at this meeting. It may soon replace lumacaftor in 508dd cf with better well as become standard treatment of gating mutations with a 508 allele if it demonstrates incremental benefit over kalydeco treatment alone. It will open the door for Vertex to treat the 508d heterozygote'het min'CF population with a second corrector,

  • Reply to

    P1037 ( VX 137) failed

    by rojospan Apr 27, 2016 10:48 PM
    qdelfan qdelfan Apr 27, 2016 11:32 PM Flag

    Rojo, ENaC Monotherapy was not expected to yield positive clinical results in this initial Phase 2 trial, and the important outcome was 137 had a clean safety profile using it as monotherapy. The theory of clinical efficacy is based on improvements in ciliary beat frequency in combining this ENaC inhibitor with VRTX potentiator/corrector combinations in vitro. The clinical trials combining VX 137 with Orkambi or 661/kalydeco in the various subpopulations of CF mutations will proceed since that is where the positive clinical results based on the in vitro tests are most likely to show synergistic/additive clinical benefit. Obviously not every investment in acquired drug candidates pay off, but remember the Virochem acquisition, while failing to get viable hep C treatments, has succeeded in at this Canadian VRTX research lab in developing new drugs to treat IBD which are entering clinical trails this year as announced last week with a French privately held company. The cervical spinal cord injury drug candidate is another acquired drug candidate entering clinical trials this year representing a potential break through drug. Effective safe non-opiate painkillers would be medical advance to minimize the problem of addiction and dependence on opiates for chronic orthopedic pain patients suffering from failed back and neck treatments in addition to patients with pain from OA. With appropriate labelling on these drugs about avoiding overuse of OA joints when taking these drugs to prevent accelerating joint damage from uninhibited activity, in many patents, the risks of NSAIDS and opiates would certainly make VX 150 a preferable option. Lastly CRISPR program is a long shot, but a lot of companies are now pursuing gene editing technology to treat genetic disease and David Altschuler, VRTX CSO, has the expertise to collaborate with VRTX chosen partner to develop this technology as a possible cure for CF and Sickle cell disease. IMHO, its an investment worth pursing

  • ENTEROME Bioscience SA, a pioneer in the development of pharmaceuticals and diagnostics based on the gut microbiome, has signed an exclusive worldwide license agreement with Vertex Pharmaceuticals Inc. (VRTX) to research, develop and commercialize novel small molecule FimH antagonists for use in the treatment of inflammatory bowel diseases (IBD).

    The first clinical trials to start this year. These are the drugs being developed at VRTX Canadian research site in Laval Quebec.

  • Reply to

    Watch for breakout

    by zchnrsym Apr 5, 2016 11:34 AM
    qdelfan qdelfan Apr 5, 2016 12:17 PM Flag

    More important is the reason for the breakout. Investors may be betting on better than expected sales of orkambi in first quarter and perhaps positive guidance from vertex management at the first quarter conference call regarding the rest of the year's sales and clinical trial data release timeline from the R&D pipeline. Also takeover speculation in the sector may include Vertex as the PfizerAllergandeal unravels and these two companies start looking for alternativeacquistion candidates whose shares are currently greatly discounted and have a rich R&D pipeline of potential blockbuster drugs.

  • Reply to

    from the fly

    by gladpick Apr 1, 2016 12:58 PM
    qdelfan qdelfan Apr 2, 2016 12:13 AM Flag

    The remaining minority of 508dd homozygous CF patients not yet taking Orkambi this year will eventually get treated either with Orkambi (as word gets out from long term data that it provides sustained benefit to improving the health and longevity of patients taking it) or perhaps with the improved potentiator/corrector combination from Vertex (661/770) currently in phase 3 trials, after it gets approved and everyone with this mutation switches to the next generation of corrector treatments with even better results.

    This equates to most all of the 508dd CF homozygotes being treated with Vertex drugs by 2017. The EU countries, Canada and Australia always take much longer to negotiate a discounted price on these breakthrough drugs, but as we have seen with Kalydeco, in the next year or two, most everyone with CF who qualifies will have access to these life saving drugs. Peak sales at this time are purely speculative, but will surely grow steadily to the 4-6 billion range as the patients gain access to treatment by the end of next year.

    Add in the possibility of the residual function mutations gaining approval for 66l/kalydeco treatment (also in phase 3 clinical trials) and eventually the "het min" CF population (508d heterozygotes) getting access to 'triple therapy' (two correctors with 770) plus the benefits from VX 371 (which can be used in all CF mutations), to be tested in clinical trials started this year, and the CF revenue potential goes even higher. And then consider the potential of the non- CF pipeline currently in multiple clinical trials in oncology, spinal cord injury, pain, and influenza. This stock is undervalued. Some of these buy side analysts are very short sighted.

  • Reply to

    Pro VRTX article on Morningstar

    by whtthefk Apr 1, 2016 10:12 AM
    qdelfan qdelfan Apr 1, 2016 11:46 AM Flag

    I agree the Morningstar article is way better than usual stuff from buy side analysts. It does not mention near term catalysts coming from Vertex specifically advanced stage clinical trial results due over the next 18 months in next generation CF treatments and non CF pipeline assets. Positive results in these clinical trials will raise analyst price targets and protect Vertex from competitors developing CF treatments who are years behind Vertex in their R&D efforts. Vertex non-CF pipeline assets include multiple drugs being tested in oncology, spinal cord injury, pain, and influenza, and they diversify Vertex potential revenue into multiple disease markets with novel breakthrough treatments, many with orphan drug pricing potential.. With near term growth in the approved and soon to be approved CF treatments, IMHO Vertex is greatly undervalued and may be a takeover target from larger pharma looking for both near term revenue growth from CF and the potential from a large diverse pipeline of breaktrhough drugs in multiple diseases. .

  • Investors anticipating 1st quarter revenues will easily beat analyst estimates.

  • Reply to

    Leiden should be fired

    by gladpick Mar 29, 2016 2:04 PM
    qdelfan qdelfan Mar 29, 2016 2:27 PM Flag

    Go to clinical website and type 'VX' in searchbar. and count the number of clinical trials either recruiting patients or about to start. Management probably not crowing about the possiblities until the results of trials are in but many late stage cf trials with vx661 and 371 and influenza vx 787 and pain (a trial underway to treat chronic pain from OA) with interim or final results due this year and early next year. Spinal injury and numerous oncology trials underway as well. They are clearly quietly building a large number of potential blockbuster breakthrough treatments and not sitting idly by. The stock market is in correction this year and analysts are lowering price targets on all pharma ( look at GILD PE ratio). As the analysts see progress in the clinical trials, the same analysts will raise PT as they give potential value to drugs in development. This means once again being patient as a long term investor.

  • Reply to

    God, what is going on?

    by whtthefk Mar 23, 2016 12:20 PM
    qdelfan qdelfan Mar 27, 2016 3:56 PM Flag

    NHS is paying for Kalydeco in the CF gating mutations, as is the rest of the EU, and Canada and Australia, after each country's equivalent of NICE successfully negotiated a discounted price. The same will eventually happen with Orkambi and it's successors currently in development (e.g. 661/770 and future combinations of correctors and potentiators) that represent breakthrough treatment in the underlying cause of CF in the various mutations. The MP's can respond to their voting constituents by asking NHS officials accept the CF Trust's proposal to allow access of 508dd CF patients in the UK to being prescribed Orkambi while NHS officials continue negotiations with Vertex on a compromise price to save lives of the young CF population in the UK and get more data on the long term benefits of Orkambi as it is used on patients in the UK from the CF registry. The UK is not a third world country like Bangladesh and can make the lives of CF patients a priority. The CF patient population deserves access to these new breakthrough drugs as they become available. The mandate of the NHS is to not choose less effective treatments because they are less expensive to treat illness. The mandate is to provide current medical care within budgetary constraints, which can be accomplished by negotiating pricing to provide that treatment. Health care administrators in the developed world have the responsibility ask for additional government funding if needed to provide better care for the population. The voting populace has the right demand the best care available.

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