Bravo!! The CF community is lucky to have people like you, Verity, to fight for this important medication that will benefit so many lives. Your efforts will make a difference.
Verity, I understand your frustration. With Obama giving a shout out to the CF community during his State of the Union Speech in January, and having Bill Elder sitting next to the first lady in the gallery, you would think with the FDA,already designating 809/770 with 'breakthrough drug' status, it would expedite the approval process faster. Is the CF community and it's advocacy groups reaching out to their White House or Congressional connections to motivate the FDA to move the approval process along more efficiently, given the Phase 3 safety and efficacy data submitted by Vertex close to 6 months ago and allow the CF population this very important treatment?.
RBC Analyst remains confident of FDA approval before PDUFA date of July 5, following the May 12 meeting
Market pullback overdue. High-flying biotech/high tech company valuations having expected corrections. 809/770 approval and pricing will return VRTX to new 52 week highs.
IF VX 661/770 shows a 6% improvement in absolute FEV-1 treating twenty CF 508 homozygotes for twelve weeks, the market is going to tell you what insurance companies are likely to pay for such an improvement, (along with the likely associated reduction in hospitalizations and pulmonary infections, improved body weight and nutritional status, lengthened life expectancy and improvement in quality of life in the majority of CF patients), with the movement up in VRTX share price after the data is released, We'll soon find out the answer in the next few days. Good luck to all longs and to the CF community!
It would certainly be contested in federal court if the patent office ruled it a new entity. The court can over rule an initial patent office decision but often it requires a jury trial. Probably more cost effective for VRTX to buyout CNCE if D-Ivacaftor is a significant improvement over the original drug. That will take a few years to prove, and will be watched closely by VRTX management who know Roger Tung well from his years at VRTX.
If you look up the home page for EIP Pharma you'll get some background on the Connection of EIP, whose CEO is John Alam, MD, (oldtimers on this board may recall; he was a formrer CMO at Vertex prior to the development of telaprevir). Vertex has licensed VX 745 to EIP since 2012 and evidently is making some progress in applying it to treat inflammation in the CNS to see if it has any clinical benefit in neurologic diseases like Alzheimers. Vertex program to treat neurologic disease or injury has much greater depth and progress than most investors appreciate for spinal cord injury, Huntington's, chronic MS, pain, and Alzheimers . Thanks Rojo for this update.
Stock price moving to new highs today anticipating CF drug approvals with PDUFA for expanded pediatric label for Kalydeco due tomorrow, and earlier than anticipated 809/770 approval soon to follow........
FYI: a few of my other current medical science company investments: (Do your own due diligence before investing!)
MACK (Merrimack Pharmaceuticals) An oncology company with a it's first drug to be approved at the end of March or early April 2015..Large insider ownership with many shares recently purchased on the open market like the rest of us. Has a partnership with Baxter to market it's most advanced drug outside the U.S. Has a deep pipeline of other drugs that will change the paradigm for treating solid tumors on the molecular level, analogous to individualizing treatment of the various mutations of CF
DXCM (Dexcom) The leader in CGM (continuous glucose monitoring) in the treatment of Type 1 DM. Partnerships with insulin pump makers JNJ (Animus division) and Insulet, and working to eliminate the need for finger stick bloodsugar testing; Has apps to I-phone and Android smart phones allowing parents of pediatric patients, and caregivers monitoring of blood sugars to avoid highs and lows, preventing dangerous complications and hospitalizations. Collaborating it's technology to develop artificial pancreas closed loop insulin delivery system not requiring finger stick blood sugar testing. Just became profitable in the past quarter.
QDEL (Quidel Corp) I'm a fan. (hence my moniker). A leader in the point of care and molecular rapid diagnostics market. Has developed a whole new group of rapid POC (point of care diagnostics) that is starting to gain traction in sales for detection of multiple infections, Huge insider ownership and superb management. It's pipeline has a number of innovative new diagnostic platforms to test for multiple pathogens both quantitatively as well as qualitatively to monitor response to treatment of diseases like HIV.
Roger Tung, CEO of CNCE, was one of the founding scientists at VRTX, and left VRTX after helping to develop telaprevir, to found Concert. His platform of chemically modifying approved drugs with deuterium to see if they have a longer half-life and potentially greater efficacy is yet to be clinically proven, but he is collaborating with a several different pharmaceutical companies to see if this chemical modification has any benefit. VRTX would certainly either license the technology to enhance the effectiveness Ivacaftor if it added benefit, or perhaps buy CNCE outright if the price was right, .and it's pipeline of other possible deuterated modified drugs had further market potential. CNCE reminds me of ENZON which developed a pegylation chemical modification technology applied existing injectable drugs to lengthen their biologic half life (e.g. interferon in treatment of hepatitis C, Erythropoietin in the treatment of anemia) to reduce the frequency of injections. IMO, with a small molecule med like Ivacaftor, dosed orally only twice a day, the extended half-life potential is not as important a benefit as in injectable drugs. Superior clinical benefit would be the key to it's adoption, and that will take a number of years to prove.
Thanks for the kind words. I am also invrsted since Aurora, and am glad to have held most of my shares given the bright future this company has. There has been, as you know, plenty of ups and downs, but the net results over the last 15 years have been stellar. I would, like Glad, keep a core position in Vertex. I think it's future as an investment over the next few years will be similar to Amgen and Celgene and Gilead when they first starting becoming commercially successful pharmaceutical companies. Lots of potential growth ahead in it's pipeline. The long term investors on this chat board may have a different perspective, but I for see the next 3 to 5 years as likely to yield even greater returns for the patient VRTX investor.
Shorting VRTX this month risks exposure to big movement of stock price higher in reaction to positive Phase 2 VX 661/770 data release coming before end of this month and possible early 809/770 approval 4 months ahead of PDUFA date of 7/5/2015 (see 3/6/2015 verityvoila posts for reasons to expect early approval this month). With regard to insider selling, insiders get additional stock options at regular intervals representing their stock based compensation. Some insiders need the money and sell regularly. Others sell occasionally, It has nothing to do with the projected success or failure of this company whose stock price reflects the market's appraisal of future growth. At the current $30+ billion maket cap, current street expectations are for approval of 809/770, (with estimated peak sales of 5-6 billion per year over the next few years) with a July 5, 2015 approval. However, if a four month earlier than expected approval occurs this month, then VRTX eps are going to ramp up faster than current estimates (by $0.26 per share per month according to WF analyst Brian Abraham's estimates) upping near term valuation of VRTX share price considerably. IMHO, not good timing to be short this month
Vertex CF franchise + rest of non-CF pipeline value = over 40 billion? Shorts are covering like crazy today on the possibility.
Cowen investor conference presentation today by Ian Smith just completed. Judging by stock movement since presentation this morning, market likes what was said. Well worth the time to listen.
Mentioned in VRTX annual report released earlier this week. The market likes it.
CNBC interviewed John Schroer, portfolio manager of a large biotech mutual fund ('King' biotech fund at Allianz capital) and his top three biotech picks were CELG, BIIB and VRTX. He went on to explain the recent weakness in VRTX was 'disappointment ' in some investors who expected 661/770 Phase 2B interim results to be released with fourth qtr earnings this past week and got 'spooked' that they were not going to be released until the end of this quarter. Schroer reiterated his confidence that the Vertex CF pipeline would result in at least a ten fold increase in revenue for VRTX over the next few years and that he could see the VRTX share price at over $180 over the next year based on the timely approval of 809/770 and the expansion of the Kalydeco monotherapy label. Of course the success of 661/770 would move that along even quicker. The uncertainly of those results is causing the current sell-off in conjunction with an overall weak stock market in January 2015. IMHO, John Schroer and Third are correct, and all will be well for the patient VRTX investor.
Today's announced invitation of Vertex Regeneron, and Merck execs to the White House this Friday certainly is a sign that the President's personalized medicine initiative mentioned in his SOTU speech is moving forward with these companies likely to be the first recipients of any tax dollars earmarked for R&D into the White House 'pet' research preferences. (Much the same thing occurred in the 1960's when then President Richard Nixon declared his famous war on cancer and earmarked federal funds for cancer research,)
A more important opportunity on Friday is for these execs to remind the president that drugs like lumcaftor/ivacaftor, which already have 'breakthrough drug status' and have completed all required clinical trials, still require another 6 months or more of review (before the PDUFA date of July 5, 2015 in lumacaftor/ivacaftor's case). Why does a 'breakthrough drug' monitored in it's development at every step for the last 5 years by the FDA need six more months to get approval, after all the data has been submitted? The inefficiencies of the federal regulatory process need to be reviewed by the White House as part of his 'accountable care'. CF patients lives are depending on this.