Vertily, I am glad to hear your child will benefit next year! Phase 2 661/770 trial results in ddf508 like your child may show incremental benefit over 809/770 in the first quarter next year, so stay tuned for next generation 661/770 treatment moving into Phase 3 trials early next year and hopefully approved in 2016 for the broader lable of all 508d patients (hetero and homozygotes) for acheiving even greater efficacy
Here is the excerpt from Feuerstein's column in TSC:
Biotech Stock Mailbag: ProQR, Vertex Pharma, Tekmira, Agios
BY Adam Feuerstein
09/26/14 - 07:00 AM EDT |
Stocks in this article: VRTXPRQRTKMRAGIOGSKSRPT
The next big pipeline event from Vertex is the phase II study of VX-661 in homozygous F508del cystic fibrosis patients. Data expected early next year. Again, I'm hearing Vertex management sounds increasingly confident about the prospects for '661. If '661 is a winner, odds increase that Vertex can craft a combination therapy to reach the heterozygous F508del patients. There's even some speculation (thanks, ISI's Mark Schoenebaum) that Vertex might go after a treatment for heteros with a Kalydeco/'661 combination, instead of a triple-drug regimen.
Lastly, Vertex is assuring investors that operating expenses will be under control. No more crazy spending.
Thanks Q! We're "lucky" though as our little one is ddf508 so next year! But so happy for even better benefits and for all the heteros! Is it possible to see the mailbag if I google somehow?
Verity, I agree this looks promising (for your child and the rest of the CF community waiting for this next breakthrough) in treatment of 508d heterozygotes! Adam Feuerstein's recent biotech mail bag column on TSC quoted Mark Schoenbaum,,the biotech analyst at ISI, as saying that a 661/770 treatment for 508 heterozygotes is possible based on the data you noted in clinical trials and in vitro results that Vertex has published, and the confidence that Vertex execs have been expressing lately about their 661/770 results. My hope is your child will have an approved CFTR modulator 661/770 first generation treatment in 2016.
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.
Think VRTX had went up quite dramatically going into earnings. A 4% pullback was healthy and necessary. VRTX can now resume its upward trend.
Their drug if it survives phase 2 , phase 3 and the approval process is an anti inflammation drug. There are already anti inflammation drugs in the market. It is my understanding that cystic fibrosis does cause inflammation of lungs and passage way. So an anti inflammation drug can only deal with one of the symptoms of the disease just like antibiotics such as Azithromycin that also deal with the infection symptom of the disease. Here is a quote from the CF Foundation about treating inflammation:
"For example, ibuprofen, which is an anti-inflammatory, was found by CF Foundation-supported researchers to benefit people with cystic fibrosis. It can slow the rate at which lung function usually declines for some people with CF. However, patients need to be closely monitored by their CF physician if taking this drug for CF."
However, VRTX CF drug franchise deals with the genetic disease itself.
More and more players in CF nowadays! Never enough me! The following is from their web site..."Our lead product Resunab™ is a novel oral anti-inflammatory drug that is expected to commence Phase IIa clinical trials for the treatment of cystic fibrosis and scleroderma, pending U.S. Food and Drug Administration (FDA) approval in the second half of 2014."
But you're missing the really important caveat. Revenues are expected to grow tenfold in the next five years. That's why Wall Street is valuing the shares over $100 now. I'm actually glad to see Vertex take a pause here, it's jumped a bit too fast recently IMHO. But if you looks at the 2016 options, which are very expensive, one can't walk away without thinking that analysts believe Vertex is going much, much higher.
dd. Wrong. I was in a clinical trial several years ago, and none of the things you're suggesting every happened. Landmark drugs are going to be expensive. Everybody knows that...except for you.
You obviously don't know the intricacies involved when patients get a very expensive drug in their hands...enough money to buy a small house with...
"...sell to their CF neighbor?" What in planet earth's name are you talking about? Dude, ever know anyone with CF on a list waiting for a lung transplant? Ever walked through a pediatric ward and seen a young child with CF on a ventilator? Price is too high? Are you kidding?
symbol instead if VRTX is a better representation of this stock. I am not sure if I would have believed on VX 135 hepatic toxicity data coming from a Maldives study and dumps the entire Hep C franchise. As I expected JNJ jumped on Alios ship in no time. CC boring and non-productive.
Sometimes, the maneuvers can even be illegal when patients skip their meds and sell to their CF neighbor. The price is too HIGH!
Not much YOY to justify this price IMO
Great! Except revenues down substantially vs. 2013 Q3 and guidance light for remainder of year. Not good enough for a high flyer. With a price way up here, any slightly negative news will tank it. Too much risk for the price.
The .57 cent expected loss has to be compared to the actual .37 cent loss, not the .72 cent GAAP EPS. When you talk Wall Street expectations, they remove certain GAAP items (like stock options expense) that are non-cash to arrive at Non-GAAP EPS. What this report tells us is that Vertex is on its way to profitability at a faster clip than Wall Street's expectation.
However, I didn't take this in to account. :)
BO (AP) -- BOSTON (AP) -- Vertex Pharmaceuticals Inc. (VRTX) on Tuesday reported a loss of $170.1 million in its third quarter.
On a per-share basis, the Boston-based company said it had a loss of 72 cents. Losses, adjusted for stock option expense and non-recurring costs, came to 37 cents per share.
The results surpassed Wall Street expectations. The average estimate of analysts surveyed by Zacks Investment Research was for a loss of 54 cents per shareSTON
Hard to tell with after hours action.. The street figured .57cts .. we lost .72cts .......The big deal is the money influx to come.. I'm long.