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Vertex Pharmaceuticals Incorporated Message Board

qdelfan 38 posts  |  Last Activity: Aug 15, 2015 12:17 PM Member since: Sep 30, 2007
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  • Reply to

    Historic Day

    by mblock66 Jul 24, 2015 12:00 PM
    qdelfan qdelfan Jul 26, 2015 10:52 AM Flag

    Papa, it's certainly your right to criticize the company anytime you like. I agree that the company needs to achieve timely approvals of the rest it's pipeline to deliver sustained growth and profitability for shareholders. However, it's not 'too late' for such progress and the VRTX pipeline now will have the funding needed without further dilution (unlike GLPG) to develop these drugs. I guess one would like as a shareholder a smooth predictable flow of clinical trials to insure one's investment. As evidenced by the slowing growth at BIIB on Friday, it's never easy or smooth for the most successful companies. Perhaps at next week's Q2 conference call we will get more information about the timing and progress for clinical trials in both CF and non CF assets at VRTX. as well as the projections for growth from Kalydeco and Orkambi.

    If VRTX various projects in oncology, influenza, spinal cord injury, Huntington's, chronic MS, as well as it's next generation CF treatments start clinical trials over the next 12 months, I believe that the long term growth plans expected from management will, eventually be successful. It just takes one or two blockbusters to make it to the top tier of companies in pharma. Any success in it's pipeline of drugs offering breakthroughs in such difficult to treat to treat conditions will likely continue VRTX growth. In the meantime, I enjoy the moments like this where VRTX has delivered a success that means so much to CF patients and their families, as well as to long term shareholders as ourselves, and is just in the first innings (to use the baseball analogy) of the development of this market.

  • Good call. Most all the senior analysts participated themselves for a change rather than sending surrogates. I expect a positive reaction from the market tomorrow, following the answers given by Leiden and his team, which certainly voiced optimism about the expected revenue growth to be generated by Orkambi,
    Leiden clearly articulated Orkambi revenue will be financing the ongoing clinical trials in both CF an non CF drugs in development this year and next. While specific metrics about Orkambi sales during the first few weeks of the launch were not discussed, I did not sense that there was any denials or unexpected delays from health insurers paying for the care of 508dd CF patients in the U.S. who are being prescribed Orkambi since the launch less than one month ago. That's good news for CF patients and VRTX investors

  • qdelfan qdelfan Jun 11, 2015 12:10 AM Flag

    These specific Vertex clinical trial results are being presented tomorrow at the Brussels meeting:


    Lumacaftor in combination with ivacaftor in patients with Cystic Fibrosis who are homozygous for the F508del-CFTR mutation
    15:30 – 15:45

    Stuart Elborn (Belfast, United Kingdom)


    VX-661 in combination with ivacaftor in patients with Cystic Fibrosis and the F508del-CFTR mutation
    15:45 – 16:00

    Joseph Pilewski (Pittsburgh, United States)


    An open-label study of the safety, pharmacokinetics, and pharmacodynamics of ivacaftor in patients aged 2 to 5 years with Cystic Fibrosis and a CFTR gating mutation: The KIWI study
    16:00 – 16:15

    Margaret Rosenfeld (Seattle, United States)

    Also, Simon Bedford, who heads VRTX international sales and marketing is speaking at the conference in a session focusing on the process of making VRTX drugs available to all CF patients who can benefit from them in the EU.

  • Reply to

    Orcambi labeling discussions with fda

    by verityvoila Jun 12, 2015 9:37 AM
    qdelfan qdelfan Jun 12, 2015 10:14 AM Flag

    Smith also said in the same breath that approval could come at any time now, so his comments about orkambi labeling yet to take place are probably pro forma activities that are already in 'rough draft' with final touches completed as approval is granted. I suspect the release of the PROGRESS data yesterday may be the final elements needed to be added to the drug insert to gain approval for Orkambi (a sustained clinical benefit without serious adverse effects)

  • Reply to

    Historic Day

    by mblock66 Jul 24, 2015 12:00 PM
    qdelfan qdelfan Jul 26, 2015 3:23 AM Flag

    How ironic that this thread starts with the topic 'historic day' and shares the hope and happiness of mblock66, redsox2004, and the happiness of Verity over the fact that 508dd CF patients are finally receiving without delay by health insurers, prescriptions of Orkambi in the first weeks of launch following FDA approval giving them hope that their loved ones can live a longer and more healthy life.

    And then we have upset with investors on this board, apparently not satisfied with the fact this company, even after a horrible week for biotech in general, has a market cap of over $30 billion, attributable only to it's CF drugs. These drugs are not considered by Vertex critics as being cost effective or sufficiently beneficial to justify the current market cap.

    Let's consider the facts: successful companies in the orphan drug markets e.g. ALXN, have been priced at even greater valuations than VRTX because they offer the possibility of breakthrough treatments in lethal diseases that have until now have had no real treatment for the underlying cause of the disease. VRTX took the financial risk to spend the last 15+ years to develop (with the assistance of the CFF funding less than 10% of the total cost) Ivacaftor, lumacaftor, VX 661, and the second generation correctors after acquiring Aurora Biosciences. Since becoming CEO, Leiden has focused the cash resources available to VRTX (following the loss of the market for telaprevir) on getting FDA approval of the CF drugs to achieve the needed revenue that will now permit the clinical development of the rest of the VRTX pipeline. He out-licensed VX 787 to JNJ/Jansen to accelerate monetization of that asset for VRTX without draining it's cash resources needed to develop the CF drugs. The proceeds from the CF drugs will now finance the future clinical trials for all the drugs in the VRTX pipeline. Simply put, VRTX's future R&D success will be financed by Leiden's focus on succeeding in CF. IMHO a good strategy

  • Reply to

    shire buying baxalta

    by harvey_smiths Aug 4, 2015 9:19 AM
    qdelfan qdelfan Aug 4, 2015 10:31 AM Flag

    Hostile takeover bid currently proposed by Shire for Baxalta. The offer seems like a relatively small premium from the current price. If a larger pharma wanted VRTX, the offer would need to give a greater value for the potential of the Vertex pipeline, both CF and non-CF assets. Daily articles in the media coming out about potential takeover bids of VRTX. Today's market action suggests that this time it may be really happen. Any guesses as to the amount a potential buyer would pay in a buyout offer? A proposed buyout would be more likely to be a hostile takeover requiring a shareholder vote, given VRTX management's long-term vision to be a large independent biotech company. How high a price would be needed to win a takeover vote put to VRTX shareholders?

  • Reply to

    Why ENaC? And why is it synergistic??

    by verityvoila Jun 5, 2015 9:24 AM
    qdelfan qdelfan Jun 6, 2015 5:19 PM Flag

    Third, beautiful explanation. Thank you. I am sure Fred Van Goor's lab did the in vitro testing on CF patient's human bronchial epithelial cells (HBE assay) to demonstrate the synergy of Parion's ENaC inhibitors with orakambi, to merit the investment by VRTX in this collaboration with Parion. Vertex has consistently shown positive clinical trial results when this HBE assay work has predicted a clinical benefit, so I am quite optimistic that the clinical benefit from ENaC inhibitors will be proven in CF patients, irregardless of their underlying CFTR mutation, and augment the improvement already proven in treating CF patients who benefit from ivacaftor, lumacaftor, and 661. I am hopeful, as well, for an extended label indication for ENaC inhibitors to treat the other non-CF chronic pulmonary diseases that would benefit from increased airway cell surface hydration (COPD, non CF bronchiectasis, pulmonary ciliary dyskinesia) since it has been many years since a new medical breakthrough has been made in the treatment of these chronic debilitating lung diseases.

  • Reply to

    parion collaboration

    by gladpick Jun 5, 2015 9:30 AM
    qdelfan qdelfan Jun 5, 2015 10:10 AM Flag

    The invitro testing of orkambi and P-1037 showed greater potential benefit to patients than the effects of either drug tested alone. This synergstic benefit is the key to improving further the lung function of CF patients who can benefit from orkambi. P-1037 may also provide a new treatment option for the most difficult to treat CF mutations who due not benefit from CFTR potentiators and correctors, as well as the larger population of non CF pulomonary disease patients who suffer from COPD, non-CF bronchiectasis and ciliary beat dyskinesia.

  • qdelfan qdelfan Jun 11, 2015 9:43 AM Flag

    Additional announcement from VRTX about presenting in Brussels the interval analysis of PROGRESS rollover study confirms sustained clinical benefits of Orkambi at 48 weeks of treatment of over one thousand dd508 homozygote CF patients over all clinical endpoints and no reported changes in incidence of adverse effects since release of Phase 3 results (TRAFFIC and TRANSPORT). Orkambi's safety and benefit is proven at two years in the largest CF study ever undertaken. FDA approval should be forthcoming at anytime since this may be the data FDA wanted before officially granting approval to market Orkambi.

  • Reply to

    P 1037/ GS 5737

    by rojospan Jun 14, 2015 12:28 AM
    qdelfan qdelfan Jun 14, 2015 11:42 AM Flag

    Vertex has in vitro proof of potential clinical benefit of ENaC blockers in CF HBE assays CF, and this benefit has been both alone and additive to the improvements in vitro when combined with orkambi and kalydeco. Whether the ENAC benefit will be realized in COPD is more difficult to predict since VRTX does not have an experience with an assay that it has been able to rely on for that indication comparable to the HBE assay used by Van Goor's CF lab, but given the potential market for COPD, it is certainly worth pursing more advanced clinical trials if the prior Phase 1 trials showed some potential benefit, Why GILD gave up on the development of ENaC is unclear, but obviously GILD execs did not have enough confidence in the Phase 1 trial results in COPD to pursue further investment. The HBE assay results in CF for VRTX are reason enough for VRTX to make the investment in Parion's ENaC blocker development program. Extending use of ENaC blockers to patients with COPD, non-CF bronchiectasis, and cilia dyskinesia would be 'icing on the cake'.

  • Reply to

    P 1037/ GS 5737

    by rojospan Jun 14, 2015 12:28 AM
    qdelfan qdelfan Jun 14, 2015 11:26 PM Flag

    When Orkambi gets FDA approval, positive cash flow from it's sales will fund multiple clinical trials including Parion drugs, second gen CF correctors and the non CF pipeline. VRTX prior drug candidate failures will be unimportant when so many potential future candidates have a shot at becoming the next successful breakthrough drug, Parion's drugs are administered by inhalation, so potential systemic adverse effects such as abnormal taste are likely to be minimal if the drug is being delivered locally to the airways where it's clinical benefit is needed. New VRTX executives, charged with selection of the drugs deemed worth the cost/risks of development are not the same people who disappointed with some of the failed drug candidates in the past. Jeff Leiden and his new CSO, David Altshuler, are now in charge of drug development choices at VRTX and I believe them to be more astute in understanding the genetic/molecular basis of drug treatment which will allow them to make better choices for the VRTX R&D pipeline.

  • Reply to

    Debut of another Orkambi partner

    by thirdmeinvestor Jun 16, 2015 11:27 PM
    qdelfan qdelfan Jun 17, 2015 9:23 AM Flag

    Difficult to say which assay is 'better' since Van Goor's lab has been working to develop these tests for the past 15 to 20 years, and they have consistently predicted positive clinical trials of the drugs being vetted by VRTX for clinical use as potentiators and correctors in CF. Obviously GLPG has similar assays since these assays are published scientific techniques discussed in scientific meetings by CF researchers for years. The point of GLPG emulating Van Goor's lab work IMHO is that it shows the confidence potential competitors like GLPG have in Van Goor's lab results, since they are employing similar techniques to predict which drug candidates to test in clinical CF trials. The main difference so far is that GLPG HBE assays do not yet have the track record of VRTX's CF lab in terms of late stage clinical trial results to give them a proven record of success. Of course that does not mean they will not have a good predictive value, it just means 'time will tell' after the GLPG clinical trials are completed in the next few years. In the meantime, VRTX execs have stated just last week (Ian Smith at the GS healthcare conference) that they are monitoring GLPG results closely and are aggressively working to improve the results of it's own CF cocktail with it's own pipeline of second gen correctors and with collaborations with companies like Parion using drugs with complementary mechanisms to augment clinical benefit across all CF mutations and with possible applications to non-CF chronic lung disease. VRTX is certainly on the right track given the fact this potentialcompetitor is using thesame techniques and similar types of drugs. Vertex is not standing idly by waiting to see is GLPG CF pipeline is better and certainly seems to be pushing forward with it's own efforts to have competitive combinations of drugs to improve treatment to as many CF patients as possible. All good news for the CF community.

  • Reply to

    Debut of another Orkambi partner

    by thirdmeinvestor Jun 16, 2015 11:27 PM
    qdelfan qdelfan Jun 17, 2015 3:35 AM Flag

    N30's GSNOR inhibitor was tested as monotherapy in CF patients last year, given as an IV infusion, and did not demonstrate much efficacy. Combining N30's drug with Orkambi or Kalydeco in CF patients already getting proven benefit fromVRTX drugs is trying to justify getting N 30's drugs approved as an add-on treatment to boost clinical benefit to VRTX drugs, much the same as Parion's drugs are being tested. Clearly, the n30 drugs did not 'measure up' to meriting investment by VRTX. They likely failed to show the sufficient evidence to predict clinical benefit in the HBE assay in Van Goor's lab, where Parion's drugs did. VRTX execs have been talking for most of last year about meeting with various other companies to discuss collaboration to enhance the clinical benefit to CF patients with it's drugs. The winners and losers in that dialog are becoming more evident based on the science that predicts clinical trial outcomes from the testing of these compounds in the HBE assay in VRTX CF research lab.

  • Reply to

    Berkelygenie, dumontlaw and huzykalex...

    by kylem8409 Jun 24, 2015 1:49 AM
    qdelfan qdelfan Jun 24, 2015 10:10 AM Flag

    Rojo, you seemed very optimistic and hopeful for Vertex on May 12. Reread your post below, which I agree with, and remember the market would not be giving VRTX a $32 billion market cap for no reason. GLPG/ABBV CF drugs, if successful, will take years to get approval and VRTX as you note in your post below has a deep and competitive pipeline for multiple drugs for both improving and expanding indications for treatment of subpopulations of CF patients and the non CF drugs which will be breakthroughs in so many other disease states.

    37 Billion company for sure

    by rojospan • May 12, 2015 7:36 PM Flag

    10% royalities for VX Ivacaftor, Orkambi and VX 661 were sold by CF foundation for 3.7 billion in Nov 2014. Guess what simple math tells me ? 37 B only for CF franchise. VX 661 is positive in 2 phase II trial consistenly and its failure chances are very low after Orkami almost approval.
    VX 970 ( ART inhibitor) is another blockbuster ready for intial read out. VX 787 is progressing nicely and Cethrin should start phase II b in July 2015. I can almost bet you that VX 745 will score big in Alzheimer Dementia and John Alum will sell his virtual company EIP biopharm and Alzheimer patents to VRTX.

    I am expecting huge run from here. VRTX is a long term buy and shorting it is equal to losing your luck. Institutions owns it any way and they will do their math and initial manipulations to get cheap retail shares.
    Congratulations to all my long friends including thirdme ( smarty), Dr. harveysmith, gladpick, verity and last but not least my positive approach friend qdelfan.

    VRTX is next DNA ( Genentech)

  • Reply to

    I have nothing about cahbar.

    by stevedavis811 Jul 9, 2015 3:36 PM
    qdelfan qdelfan Jul 11, 2015 2:18 AM Flag

    The point is trading is not everyone's philosophy of how to invest. Buy and hold investing in a company with a successful long term growth strategy can pay off handsomely for the patient long term investor who does not panic over the gyrations of the market that are inevitable over the years one holds a stock. I have definitely gotten 'older' over the past 15 years, watching my investment in VRTX run from an average price of $17 to it's current value, with a lot of ups and downs in the process of going up almost 8 times my purchase price to date. They did not pay me a dividend while waiting, but my dividend paying stocks have not delivered a total return anywhere close to
    VRTX, a company whose growth as a commercially successful pharmaceutical company is just beginning.

  • Reply to


    by gladpick Jul 15, 2015 9:50 AM
    qdelfan qdelfan Jul 15, 2015 10:06 AM Flag

    Barron's on online article yesterday about Vertex was released about the time the stock made most of it's upward move yesterday. Perhaps today's early move up is follow through on yesterday's move up in price thanks to that article's remarks about orkambi earnings for H2 2015 more likely to beat analyst's revised estimates. VRTX is now being judged by the growth in it's revenues and earnings as it turns profitable on a sustained basis. Take over speculation may also be a factor but the timing of the Barron's article seems to be the current catalyst for the move up in price.

  • Authorization: 6 months
    Cystic Fibrosis (CF) is an incurable disease inherited through an autosomal recessive pattern. CF causes thick, viscous mucus to form and build up in the lungs, pancreas and other organs leading to severe respiratory and digestive problems as well as other effects. The genetic defect in CF occurs as a result of mutations in the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) gene. More than 1,800 mutations of the CFTR gene have been identified. The most common mutation involves a deletion that codes for phenylalanine at position 508 in the CFTR protein and is known as an F508del. Approximately 45 percent of all CF patients are homozygous for the F508del. Lumacaftor is a CFTR corrector while ivacaftor is a CFTR potentiator. Orkambi is a combination drug containing lumacaftor and ivacaftor that is indicated for the treatment of cystic fibrosis in patients 12 years of age and older who are homozygous for the F508del mutation in the CFTR gene.
     Patient must be ≥12 years old AND
     Patient must have a confirmed diagnosis of Cystic Fibrosis AND
     Patient must be determined to be homozygous for the F508del mutation in the CFTR gene as confirmed by an FDA-approved CF mutation test AND
     Patient has a baseline forced expiratory volume in one second (FEV1) between 40 to 90 percent of the predicted normal value AND
     If the patient is between the ages of 12-18, they must have undergone a baseline ophthalmic examination to monitor for lens opacities/cataracts
     Patient has stable or improved FEV1
     Clinical notes document improvement in patient symptoms
     Patient has LFTs/bilirubin monitored every 3 months for the first year of treatment and annually thereafter.
     Serum ALT or AST

  • qdelfan qdelfan Aug 15, 2015 12:17 PM Flag

    So the 35-40% of the U.S. 508dd CF population on Medicaid for their health insurance will likely be on Orkambi before the end of this year.

  • qdelfan qdelfan Aug 15, 2015 11:47 AM Flag

    The above criteria seem appropriate and reasonable. Patients simply need to qualify for treatment based on FDA's package insert for indications and appropriate monitoring while on treatment. Every six month renewal of authorization will be a necessary burden for health care providers to document proper follow-up while on treatment. Most important is the criteria that patients should be stable or show improvement in FEV-1. That will be fairly achievable based on the long term rollover study results showing patients are maintain their benefit of the drug beyond the first year of treatment. Vertex will be negotiating with each states Medicaid Pharmacy committee to determine pricing but since this process has already been established with Kalydeco, the approval at each state level should be fairly straightforward, to allow patients access to this life extending drug.

  • Reply to

    parion collaboration

    by gladpick Jun 5, 2015 9:30 AM
    qdelfan qdelfan Jun 5, 2015 11:11 AM Flag

    Verity, this is taken from the Parion web site explaining the basis for how their P-1037 drug works:

    "The inhibition of ENaC with Parion compounds blocks the major pathway for mucus dehydration in the lungs, maintaining the viscosity (or stickiness) of mucus such that it can be cleared by the cilia. Parion has developed a portfolio of novel and proprietary ENaC blockers provide long-lasting mucus hydration that is expected to be effective for the treatment of a wide-range of respiratory diseases."

125.02-1.51(-1.19%)9:36 AMEDT