Wall Street finally crediting the potential of the VRTX pipeline. Congrats to all the patient and committed long term investors who saw this potential for so many years and will now see that potential become reality.
Either you are ignorant or a short trying to cover. Insurance does cover it, as it must since their is no alternative treatment...and just breathe a little better? You have no idea how horrible cf is or you would never post that and if you do...and had a conscience you would never post that.
with your alias buddy rrttuuvv (account formed 10/11/14). Stop the scare tactics.
Everyone now has access to the presentation yesterday that demonstrates the 24 week (TRAFFIC/TRANSPORT) improvement in lung function carries forward in PROGRESS out to 48 weeks. Wait, what? 48 weeks, thats almost a year. Good luck to being short.
At Vertex’s earnings conference earlier this week, analysts again pushed Vertex on why it believed it appropriate to include a heterozygote arm for the upcoming Phase 3 661 combo. I had looked at the Phase 2 results and compared to Phase 2 & 3 for the 809 combo in the past, but there was so much noise, given the monotherapy and multi-dosing arms, I wasn’t able to get a good handle on whether 661 provided better clinical results. But Vertex noted it had selected dosing for VX661 at 100 mg and the normal 150 for Kalydeco. With that in mind, I pulled the data again and the 661 combo does translate to better results–double or nearly double when looking at FEV. Here’s the relevant info:
VX661: FEV Mean Relative Change versus placebo: 9.0
VX809: FEV Mean Relative Change versus placebo 6.7, 4.3, 4.4, 5.3 (2 studies @ 2 different dosings).
VX661: FEV Mean Absolute Change versus placebo 4.8
VX809: FEV Mean Absolute versus placebo: 2.6, 2.6, 2.6, 3.0
Also pretty stark is the 5% FEV Improvement:
661: 66.7% had 5% or greater compared to 21.7% for of placebo.
809: 37/46/46/41% had 5% or greater compared to 22/23% placebo
While Phase 2 for 661 was only a 4 week study and 809/770 was 24 weeks, the improvement appeared to plateau at 4 weeks, so the data is pretty comparable.
We know that the 661/Kalydeco combo can work on one allele from the 551/df508 proof of concept theory. Given the results from Phase 2 of 661, I believe that there will be enough improvement to have a broad label including heteros with at least one df508 allele, especially given that there is no current therapy for those individuals.
Q, the strong endorsement by the Committee for the label expansion bodes well for the approval of more important treatment for 508del homozygotes next year. The very likely approval by the FDA to include R117H CFers of 6 to 18 year old would indicate that the FDA is very positive on the Vertex CF program.
And also indicates that panel has a very good understanding of CF, its progressive nature, the need to prevent the damage, and that FEV doesn't tell the full story. This recommendation and approval is such a blessing to the young CFers with R117H who otherwise would have waited until they got older and sicker before getting their "cure." Very excited to see what the 661 combo can do for those who benefit from Kalydeco solo... for some it could be near-fully functioning CFTR. What we dream of.
I expect there is more mutual/hedge fund buying of VRTX coming in the next week as we close out the third quarter and approach the NACF meeting on Oct 9 with new positive clinical data expected as well as the 809/770 NDA filing.
Presentation is between 4 pm-6 pm ET. Friday 10/10/2014 will be an interesting trading day. I am hoping to see continued improvement in absolute FEV1 to greater than 5% in the follow up studies.
The more key presentation is earlier in the day at 11:30 when vertx presents data from the continuation study. See vertex press release at its home page. I predict that fev will remain stable at the results gained...not much higher if at all, but I think there will be a startling decrease in hospitalizations and iv antibiotics. I've done a 180 on the combo thinking first we needed sweat chloride to have big drops,...to we need great improvement in fev to that this drug is going to be a maintainence drug that gives some improvement in fev but mainly helps CFers remain stable and out of the hospital and without further lung damage until we get the tripple combo and that is great for CFers.
If I see VRTX plummeting down anytime to 20s, I am gonna load up on it BIG time. There is ton of potential ahead. It would be a dumb mistake to short such an amazing company.
Don't lose your shorts, shorty.
Sentiment: Strong Buy
You are a FOOL. Do stocks go up every day? What pullback...? The longs here are up pretty well. Your endless drivel is comical.
FDA does not need to to see persistently rising FEV1. The Kalydeco trials for G551D has shown a rapid increase by 2-4 weeks on drug followed by sustainment.
rollover study using 809/770 in over 1000 patients will provide additional new safety and efficacy data supporting quick approval by regulators and pricing commensurate with reduction in hospitalizations and incidence of pulmonary infections and improvements in BMI and QOL questionaire scores.
The NDA and MAA both filed for expedited review, with the breakthrough drug designation in the US means anticipated approvals are on schedule by mid 2015 with sales in the second half of 2015 to help over 20,000 more CF patients worldwide.
The Advisory Committee recommends 13 to 2 in favor of the label expansion for Kalydeco to include 6 years or older R117H mutation holders.
10/29/14 09:47 am ET ... S&P CAPITAL IQ REIT-
ERATES BUY OPINION ON SHARES OF VERTEX
PHARMACEUTICALS (VRTX 114.07****): We
raise our 12-mo. target $20 to $140 on 25X our
2016 EPS est. of $5.62, slightly above peers. Q3
adj. loss per share of $0.37 vs. loss of $0.32 is
$0.10 narrower than our est. Kalydeco sales
were $127M in Q3 and we see 2014 Kalydeco
sales of $460M. We believe VRTX is on-track to
progress its Cystic Fibrosis (CF) franchise to a
greater patient population. The FDA action date
for Kalydeco to treat the R1117H mutation is
Dec. 30, 2014 and VRTX plans to submit a NDA
in Q4 for Kalydeco and Lumacaftor to treat
those with 2 copies of the F508 mutation, the
most common form of CF. /