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Vertex Pharmaceuticals Incorporated Message Board

qdelfan 51 posts  |  Last Activity: Jun 25, 2015 8:35 PM Member since: Sep 30, 2007
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  • Reply to

    Watch AH trading

    by thirdmeinvestor Jun 25, 2015 5:47 PM
    qdelfan qdelfan Jun 25, 2015 8:35 PM Flag

    Based on stock price AH, may see FDA approval of Orkambi tomorrow.

  • Reply to

    Berkelygenie, dumontlaw and huzykalex...

    by kylem8409 Jun 24, 2015 1:49 AM
    qdelfan qdelfan Jun 24, 2015 11:37 AM Flag

    The market is signaling approval on the PDUFA date (if not earlier) and early approval is not an important issue in the commercial success of Orkambi. Down the road (years away) there may be competition, but not for years. In the meantime VRTX execs, well aware of the risks of a short product lifecycle from the telaprevir experience, are proceeding aggressively with improvements on it's own existing approved and nearly approved CF treatments with combination treatments of 661 and 770, 2nd gen correctors with orkambi or 661/770, and adding EnAc inhibitors from Parion into the mix to show further clinical endpoint improvements to enhance benefit to CF patients and remain competitive in the CF market. The non CF pipeline of drugs as you noted in your May 12 post may be even greater revenue creators for the company. "Expert" opinions that are bothering you regarding the 'small' 3% improvement in FEV-1, are missing the more important point. Orkambi is the first breakthrough drug in the treatment of 508dd CF patients to slow the progression of the disease. 30-40% reduction in exacerbations is meaningful in patients with just a few years left in their life expectancy, and is a significant pharmaco-economic benefit in terms of negotiating pricing. It will likely be improved on further with the combination of drugs being developed by VRTX. Breakthroughs in pharma usually generate competition. The key is VRTX is going to aggressively try to improve on it's own success in the treatment of CF and not sit back and wait to see if any competitors can do it better.

  • 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

    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

    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

    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

    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)

  • qdelfan qdelfan Jun 11, 2015 10:22 AM Flag

    Perhaps the VRTX presentation to take place later this morning at the GS Healthcare conference will ignite a greater VRTX stock price move up after VRTX executive discuss the implications for worldwide approval of Orkambi after interval data release of PROGRESS study earlier today. Hopefully some mention will also be made about the market potential for the rest of the VRTX pipeline to the GS investor conference attendees.

  • 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.

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

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

    WS01.3

    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)


    WS01.4

    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)


    WS01.5

    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

    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.

  • Curr Mol Pharmacol. 2013 Mar;6(1):3-12.

    ENaC inhibitors and airway re-hydration in cystic fibrosis: state of the art.

    Althaus M1.

    Author information

    Abstract

    Cystic fibrosis (CF) is a hereditary disease caused by mutations in the gene encoding the chloride channel "cystic fibrosis transmembrane conductance regulator" (CFTR). The lack of functional CFTR in CF airways leads to impaired ion and fluid homeostasis of the fluid layer which lines the airway surfaces (ASL). The ASL is important for proper ciliary beat and clearance of mucus from the airways. According to the "low volume hypothesis", CF airway epithelia hyperabsorb sodium via the epithelial sodium channel (ENaC). Although the contribution of ENaC to CF pathogenesis is still under debate, there is convincing data demonstrating that re-hydration of the ASL might improve mucociliary clearance in CF patients. ASL re-hydration might, amongst other things, be achieved by a block of airway transepithelial sodium absorption with inhibitors of ENaC. This mini-review article describes the role of ENaC in ASL fluid homeostasis and rehydration, and summarizes the current state of the art in the discovery and establishment of compounds which inhibit ENaC activity and may represent pharmacological tools for the treatment of CF.

    PMID: 23547930 [PubMed - indexed for MEDLINE]

  • Reply to

    parion collaboration

    by gladpick Jun 5, 2015 9:30 AM
    qdelfan qdelfan Jun 5, 2015 4:48 PM Flag

    2 different mechanisms, and P-1037 not involved to my understanding with misfold or trafficking of CFTR. But it's mode of action is supposed to augment the hydration of the airway via a mechanism that is additive to the benefit of restoring a functional CFTR with orkambi or other combinations of correctors and ivacaftor. Additive net benefite hopefully results in improvement in all endpoint parameters used to measure benefit of these drugs ie synergistic effect.

  • Reply to

    parion collaboration

    by gladpick Jun 5, 2015 9:30 AM
    qdelfan qdelfan Jun 5, 2015 11:49 AM Flag

    Here's an analyst's assessment of VRTX collaboration with Parion:

    "Canaccord Genuity analyst Adam Walsh weighed in with an optimistic view on Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX), reiterating a Buy recommendation on the stock with a price target of $150, after the biotechnology company announced collaboration with Parion to develop epithelial sodium channel (ENaC) inhibitors. The stock is currently down 0.48% at $126.33 in mid-day trading

    Walsh wrote, “Investors have long awaited news on the Vertex biz-dev front. They finally got it, and we like the deal. It fits well with the company’s core CF competency (with a call option on additional pulmonary diseases), comes at a reasonable price, and any new drug approvals could be leveraged into the existing CF infrastructure.”

    Furthermore, “We derive a 12-month price target of $150 per share based on a probability-adjusted NPV of Vertex’s two lead products Kalydeco and VX-809/Kalydeco. Our model assumes 95% probability of success for VX-809/Kalydeco. Our NPV excludes net cash per share of $4.62, as well as VX-661, next-generation correctors and other pipeline candidates, which would be upside if successful.”

  • 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."

  • 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.

  • Reply to

    THE FDA COMMITTEE VOTE THAT COULD SINK VERTEX

    by cashbar1 May 28, 2015 8:07 PM
    qdelfan qdelfan Jun 3, 2015 9:25 AM Flag

    What happened to "Thank you for calming me down?"

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