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.
SCL30A8 is a drug target for treatment of DM II, just like PCSK9 for new hypercholestrolemia drugs from REGN and AMGN, The selection of a new CSO make me believe that VRTX is about to enter a new era of gene therapy and genetic targeting of diseases especially Diabetes. CFO has mention gene therapy in his last two conference calls. VRTX is a screaming buy in my opinion.
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.
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. /
The Yahoo VRTX message board contributors to pay attention to include qdelfan, thirdmeinvestor, rojospan, and gladpick. They know what they're talking about. :-)
been there and would never predict a stock that might be on a tear- go to sleep and don't even look at VRTX for the next year
Sentiment: Strong Buy
Third, in one week the interim data will be announced at the annual NACF meeting in Atlanta, from the rollover study from TRAFFIC/TRANSPORT using 809/770 in the 1,000+ 508d homozygotes CF patients continuing on study drugs. I hope there will also be an announcement about expedited NDA filing for 809/770 as well, to result in prompt FDA review and approval of this breakthrough drug. The data will likely support pricing that exceeds analyst's expectations, and price targets will be raised further once the implications of this data are understood next week.