I have done a little investigation into VRTX Multiple Sclerosis regenerative medicine program in collaboration with Cleveland Clinic. Program has been going on since 2009 and it is an exciting medicine to stimulate cells to produce mylin sheath. Biogen is obviously has its finger into the similar research. Lets hope Traffic and Transport show absolute FEV1 of at least 6%. After that we could go full speed on the highway. Thank you for the AIDS project update.
It will be fun if study 102 cohort 4 in heterozygous on high dose VX 809 shows statistically significant improvement in FEV-1. This cohort has roll over study as well. Stuart Arbuckle in one of the conferences has said that FDA has asked them to do this cohort in heterozygous. Biostaticians at FDA must be seeing something. We may not have to wait for a 2 nd corrector. Two previous CEO's have stop selling their shares since 12/2013.
Why Cowen analyst think that either Traffic or Transport is going to fail and he is putting 40% chance to it.? Similar trial design so why he think one is going to fail when other one will not?
so if you read inhaled corticosteroid trials for COPD, after 6 months there is no improvement in relative FEV1. With Ivacaftor FEV1 continue to improve after 48 weeks and Lumacaftor suppose to follow the same pathway. I think even in worst case scenerio where Absolute FEV1 percentage point is 3 or 4 at 6 months, but in 1 year it will become 5 or higher i.e trial will be positive at some point in time. I think once data is available and more mature in the follow up studies, investigators will look at the 45-60 ml gain in the lung function from the base line rather then % points. I think going with absolute FEV1 is better going forward as a primary end point.
If there drug decreases exacerbation of CF x 2 in the first 12 months, it adds FEV1 of 5%, based on previous exacerbation studies.
CORRECT ME IF I AM WRONG.
Absolute FEV-1= Worst lung function show biggest improvement (measures quantity)
Relative FEV-1= Fastest improvement in lowest lung function ( measures duration)
Tobi trial had relative FEV-1.
Doing a second phase II with VX 135 400 mg and Daclatasvir 60 mg caries a merit. First toxicity issue can be settled, secondly, clear there is a trend for drug dose response at higher dose and SVR 4 of 91% can become 100%. ( I have never heard from Alios or from VRTX that VX 135 has a narrow therapeutic index). GILD bought PS7977 in 02/2012 for 11 B. At that point GILD market cap was only 42 B. After that they are now sitting at 130 B. Just imagine the value created by PS 7977. Two to three year lead in drug market is nothing. They need to listen to their scientists and not to please their financial analysts. Financial analyst are invested in GILD and they don't want their GILD investments at risk.
If I understand correctly, VRTX had agreement with BMS to do TWO phase II studies with VX 135 and Daclatasvir. I don't know if it is practical or not but why don't VRTX conduct another cohort of VX 135 dose 400 mg and Daclatasvir 60 mg in 10 patients and monitor them closely and see if hepatotoxicity happens ( answer should be out in 3 months). If no hepatotoxicity, (which could be related to a combination of Ribavirin with VX 135) VX 135 will be good to go. If VRTX pull out of Alios, it will lose not only an opportunity to have some market share of Hep C market in 2-3 years, it is a possibility that Alios will be free to go on its own to make a deal with BMS while VRTX is out.
And if 95% of CD4 cell are alive and functional, they will either hunt down HIV virus or will signal foreign body reaction to form a granulomatous reaction to contained and calcify HIV hides out.
Canadian Public health authority has reported an Avian flu ( H1 N5) related death today. NOT GOOD.
Severe H1 N1 totally unexpected cases in United States this year resulting in several deaths in the last 4 weeks. Tamiflu is ineffective once virus incorporates into the DNA. Patient who were immune suppressed and even took flu shots in Sep 2013, have tested positive for Influenza A.
VX 787 is an urgent need for years to come.
Check out "flu view from CDC". There is an epidemic of H1N1 this year i.e swine flu of 2009. Flu kills 36000 mostly young folks each year. It costs nearly 10 B to economy each year from illness. Flue shot takes 2 weeks to work and some people don't take shot/ or mist due to one or other reasons.
91% if you exclude the 1 patient who didn't finish the therapy. It is close to SVR 4 of 93 % in the GS 7977 and Daclatasvir trial. This is very attractive for BMY in my opinion.
is a better surrogate marker than FEV-1 for predicting pulmonary exacerbation in CF patients especially in younger CF. FEV-1 is insensitive end point in adolescent patient where lung damage is not enough to show obstruction in the exhalation air flow. Published in Thorax 01/2014.
Ivacaftor improves LCI already published in the past. Younger patient may not show a powerful change in FEV-1 but actually are the best candidates for the treatment to preserve lung function. This provides a strong argument for R117H mutation approval in all age group.
Group that has done is this discovery and publication is too small and I am not sure how much is the involvement of University of California at San Francisco? Only VRTX can elaborate. Bottom line it will come to IP. VRTX has to fund and take the risk but it is worth it.
HGSI now GSK has some work on Caspase and I am sure now GILD and many others are going to jump on this one. VRTX 765 has advantaage of being in human trials for over 500 patients, so it is safe and they could easily advance this thing into II B very quickly in HIV positive patients.
It is good to be hard working and smart but if you can get lucky too, that's what one needs in Wall Street. VX 765 is a caspase -1 inhibitor which has been struggling to find an indication and going through rare disorders as published by NIH papers, Psoriasis and Treatment resistance partial epilepsy but no real luck. I was hopping to see if some one could do a pilot study for Cardiac arrest patients with anoxic brain injury to see if this could be supplemental to hypothermia to reverse or prevent anoxic brain injury, but didn't see much. Today, I read Science and Nature and my jaw dropped. Baby this is BIGGGGGGGGG!!!!!!!!!! VX 765 could be a potential candidate to prevent AIDS. Jeff Laiden will be talking about this in JP Morgan conference for sure.