"This in vitro observation suggests that the addition of P-1037 to the investigational combination of lumacaftor and ivacaftor could enable enhanced function of the cell’s cilia to clear mucus from the cell surface, potentially resulting in improved lung function."
If it works it would be a great improvement in the life of CF patients.
Been trying to think through Vertex's announcement about its collaboration with Parion. Why would Vertex want to work with ENaC? Is seems like investing in the development of hyper-sal to go along with Kalydeco and Orcambi? Is itsn't like it could be co-formulated? Is it to control the testing and assure it is a dual-treatment application to FDA to prevent it from being an option where insurance cover one or the other?
But then I thought more about it and am perplexed as to why ENaC is synergistic to Orcambi? Given what ENaC supposedly does, I don't get how it is synergistic. I think it's great--it will help the lungs even more--but why??
There should be synergy btw ENaC inhibition and chloride transport enhanced by Kalydeco or by Orkambi. Lung function should improve more as a result.
This is great news for the stock and the company right?
The FDA approval could come any day now, with a deadline of July 5th. Every day that goes by without an approval makes people more and more antsy.
You can't even get your own (wrong) figures straight. Please stop trying to deceive people. You clearly don't know what you're talking about.
Your posts are so off base and factually incorrect that I can only assume you're being purposefully deceitful.
The actual facts, summarized:
Mean absolute change in % predicted FEV1 was approx. 3% and mean relative change in % predicted FEV1 was approx. 5% (on the basis of pooled data);
Statistically significant increase in Body Mass Index (BMI)
Delayed time to first pulmonary exacerbation;
30-39% reduction in pulmonary exacerbations (depending on the dose);
39-61% reduction in exacerbations requiring hospitalisation;
45-56% reduction in exacerbations requiring intravenous antibiotics;
Some people on the trial had baseline FEV1 below 40%. While the eligibility criteria was 40%, some dipped below this threshold in the period between screening and first drug. These patients seem to have responded just as well as those with higher baseline FEV1;
Approx. 90% of people on the trial decided to rollover into the open label extension including those who had been taking placebo during the trial period. This says something about how the drug made patients feel in themselves.
Worth noting too: Vertex bounces around within a ~5 dollar frame which I would consider to be reasonable stability; this is not a yo-yo stock. Furthermore, the FDA will approve Orkambi, VRTX will rally for several days as a result and well above the 130 the above poster paid per share. For anyone squeamish, dump your stocks then.
Why would you comment on a drug you clearly know nothing about for a disorder you clearly know even less about?
If every nickel matters to you, you shouldn't be investing in a biotech stock...or rather gambling as that sounds like what you are doing given your e attention to the jockeys chatter before the race. Vertex bounces around a lot....you'll get your money back if you just wait for its next bounce up and then you can break even if you think it's too mch of a risk. The fda approval will be here shortly and then only more positive stuff on the pipeline.
Would you pay $300,000 for a 1.7% questionable improvement in breathing, multiple side effects that half the patients dropped out, and the possible efficacy quickly wore off.
Now traders are saying sell VRTX stock as the share price has broken below a key technical level. I own 1000 shares at $130 and am already underwater $5200. If you were in my shoes, where every nickel matters to me, would you sell and try to buy in lower? Would you just walk away all together? Or would you just ride it out (hard to catch a falling knife) and exit for the FDA decision in 30 days?
One day the stock goes up $5 then another $1 the day after only to give almost all of it back?! Just because of a biotech ETF SELLOFF?! EFFED up!😩
Because it's spring I guess. Seems the spring biotech meltdown is coming later this year. I thought we were safe getting through May be it seems not.