Problem with these studies has been identified. VX 770 doses used in these lab experiments are way higher than what are used in human trial. Most of the drugs work well in lab but they fail in human trial and I am glad that something fails in lab but working in real life. The investigator in one study has conflict of interest and he has work on a drug similar to VX 770 and he believes the other drug will work better in combo.
I am extremely upbeat on VX 970. I think they are working on West nile and yellow fever virus as well. They has some research on PKC theta for autoimmune disease which selects the blockade of T cell function so that viral immunity is intact i.e JC virus issues with drugs like Tysabri and Rituxan etc.
The articles by Veit et al. (McGill U. group) and Cholon et al. (UNC group) in the July 23 issue of Science Translational Medicine reported results of their independently obtained cell culture work into the effects of Ivacaftor on F508del CFTR that has been transported to, and localized to the plasma membrane by the action of Lumacaftor. A preliminary results of the UNC group had been presented in the European CF conference last year. Analysts asked about those at the conference. Van Goor responded to the questions. So, Vertex is well aware of their work. What is new is that their results in the two papers are complementary in a number of observations and support the conclusion that Ivacaftor rolls back, to a degree when dosed chronically, the very rescue action engineered by Lumacaftor. All in cell culture. However, the two reports contradict each other in a major way on the effect of Ivacaftor on wild type (normal) CFTR. The work of Veit et al. reports that there is no effect of chronically delivered Ivacaftor on the function of wild type CFTR, but the work of the UNC group reports a large effect. This latter work, of course, contradicts the superb clinical efficacy observed with Ivacaftor, which have been dosed for three years continuously.
Nevertheless, their work appear to support the notion that the putative antagonism within the synergy between Iva and Luma is the root cause of rather modest efficacy of the combo in treating homozygous F508del CFers.
Tuesday's conference call may address Vertex plans in more detail regarding it's own internal development of next generation correctors to be used with 661 and Kalydeco which had been previously mentioned as potential treatment for the vast majority of CF patients including 508 heterozygotes as well as homozygotes. The first clinical trials have been mentioned in past conference calls to be started at the end of this year or in early 2015. From a more positive perspective, the number one biotech analyst at ISI group, Mark Shoenebaum, in recent CNBC interviews and in his reports, has been quite positive about the potential for Vertex plans to treat the vast majority of the CF population with this combination of drugs, and sees further upward valuation in the company in the mid term from this triple drug combination, as positive clinical trial data is read out next year Given the competition chasing Vertex and the lesson learned from telaprevir being replaced so quickly, there likely will be questions during the Q&A during Tuesday's conference call from analysts like Geoff Porges or Andrew Fein from HC Wainwight whose reports have been focused on the future commercial competiitve risks facing Vertex in it's CF franchise. A confident response from Vertex executives to these inquiries will help convince analysts that Vertex is aggressively developing more effective next generation treatment to help most all patients with CF and that Vertex will remain the leader in the CF field.
What was his analysis Oct. 2000? Last time Vertex was this high. Maybe Mr. Wainwright will buy all your shares?
Cashing out and retiring. Not that it really will affect the price, they only held less than 1% anyway.
Tell that to the children and adults with the mutation that Kalydeco works for. See if they think its worth it..... They are going from lung functions in the 60's and 70's to over 100. No matter what it costs saving lives is the primary driver here and as long as it is doing that, those pills are priceless to those that benefit from them, hence they will find a way to pay for it to live
VRTX insiders got over $10 million cash from selling VRTX stock. Are they silly? Some bad news may have not been published. VRTX will fall like a knife!!!
Forget about the price (marked to market) chart. Look at the volume chart. Big volume right before the close. Even the incompetents at the Fed know something is strange in biotechs. CYNK anyone?
Papa, the company succeeded in transferring genes to red blood cells is bluebird bio. I agree that gene therapy for CF would be a big challenge and it may take more than 10 years, but transplanting normal CFTR to the lung cell nucleus are being attempted in UK as I said before.
Thank you Rojo for this update on VX 509. You may be right about the decision to do the phase 3 trials for VX 509 in RA in house, but given the competition for existing biologics already FDA approved and being used widely to treat the autoimmune diseases 509 could treat, is the cost of the lengthy trials in the various disease indications likely to result in a significant market for an oral small molecule alternative with the efficacy described above? Is it safer/ more effective than existing biologics, or is it simply going to marketed as more convenient but just equally effective/safe as existing parenteral treatments which are already widely adopted ? Would the latter advantage alone be likely to be worth the cost of development, by Vertex or is this better to license, like VX 787 to a larger pharma (if one was interested) which is better funded to absorb the development costs?
Van Vollenhoven group presented the data in Annual European Congress of Rheumatology and ACR 20 of 61-62% with various doses, Moderator of the conference at the conclusion did talk positively about this drug's phase II B trial with Methotrexate. This event on 06/14/2014 is now published and available in Medscape. I think VX 809 data was so important that VX 509 became non event full. With Hep C program gone, I think VRTX may will move forward with phase III. VX 509 now has a name " Decernotinib" and also a web site IP has been reserved for it and this makes me think that work is in progress.
I also have done some back ground work on VX 970 and it seems like VRTX has been working on ATR kinase inhibitors for a while and they have portfolio of molecules,
Good luck to all longs.