Recent VRTX stock price action, Ian Smith's confident response to the MS analyst's query at last week's MS healthcare investor conference about the pricing potential of 809/770 (the clinical trial data justifies a 'high value'), and the higher VRTX price targets announced in the past few days from GS and DB suggest the rollover data will be positive and a catalyst to moving the stock price higher along with the NDA filing and quick approval by the FDA given it's 'breakthrough drug' designation.
Any concern about the 809/770 combo efficacy may not hold after longer time? Ph3 results show that the improvement is less at the last time point, and slightly higher in the time point before.
Oct 9-11 is NACF annual meeting, with numerous Vertex clinical trial results being presented including interim results from 809/770 rollover study which will demonstrate sustained efficacy and safety data from the 1000+ patients in TRAFFIC and TRANSPORT, as well as reproducible response in the former placebo group of patients who now are receiving 809/770. Also the NDA flifing for 809/770 may be filed around the time of this meeting. Given the recently raised VRTX price targets by multiple influential analysts (GS, DB etc.), this should be an exciting few weeks for CF patients and VRTX investors.
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.
Adam F. predicted this will double in 2014. So far it's going in that direction.
Sentiment: Strong Buy
before year end? A quick perusual of the last quarters earnings transcript did not provide any. Except perhaps some preliminary combo data with 661.
With today's announcement of Roche buying ITMN, and Merck's recent acquisition of IDIX, one could speculate that VRTX could be a potential acquisition target for a large pharms looking for a ready to market growth franchise like it's CF drugs.
Pfizer/Fold Rx, Sanofi/Genzyme, Abbvie/Galapagos, and Gilead all have active research programs in CF which are years behind Vertex, and all with a need to bolster their pipeline with ready to market breakthrough drugs. I suspect Wall Street analyst speculation on the next big takeover will be mentioning VRTX as a potential target once again on Monday.
I thought I separated compound 31 and stem cell research in a separate paragraph. Stem cell research is a big plus for VRTX in my opinion.
your post is confusing, you obviously refer to this Vertex paper
Journal of Medicinal Chemistry, 2014;57(8):3382-3400.
which describes a classical medicinal chemistry effort, which has no relation to stem cell therapy
HPS alpha/beta selective inhibitor has been identified and published as a drug target for Huntington disease and VRTX has "compound 31"in works that crosses the blood brain barrier and decreases Htt level in brain. Data in mouse published in Nature chemistry magazine 4/2014. J L said Huntington disease was VRTX next target after CF and VRTX was not interested in spending $$ on VX 509 in Jan 2014.
It amazes me to see a small molecule developer like VRTX has so much future investments and moves in Stem cell therapies when all the initial stem cell companies failed to bring any product to the market.
Epix Pharmaceuticals-As of July 2009, the company was in the process of asset liquidation due to insufficient funds to stay afloat.
Tolerx-In 2008, Tolerx was named one of Fierce Biotech’s Fierce 15. In October 2011, Tolerx was shut down due to an unsuccessful Phase III trial in patients recently diagnosed with Type 1 diabetes.
Talk about a reversal of fortunes.
Three years after being heralded as the fastest drug launch ever and quickly racking up $1 billion in sales, the Incivek hepatitis C treatment will be discontinued this coming October by Vertex Pharmaceuticals..................do your research. These are the same people that brought you Epix and Tolerx. Make their money from selling shares not curing diseases. Please not another tirade from some bleeding heart that says I need to buy shares and BELIEVE in the "project". Business whatever it may be NEEDS to make profits. TY n GL!
Sentiment: Strong Sell