Well it is a Finance board, so talking about SP is completely appropriate. And I don't think anybody here thinks that getting any improvement to CF patients is a bad thing!
But Vertex flopped w/Incivek and for those around a long time, cast your mind back to Epilepsy, RA, Flu, HCV partnership etc....For the number of scientists at VRTX they do not have a promising pipeline or track record. Also for a company with 1800 people, they are not all needed for CF. So what is the strategy....that's what people are asking. Keep in mind, many of the 'Vertex critics" you mention are CF doctors treating patients and they are not happy with the cost of these drugs and the pressure it puts on families (and insurance companies...let's face it the costs are passed through in some form to everyone else). I've made my money on VRTX but will step back for a while until they have a better story. This is an investor board so I like to read posts that give the good and bad of potential investments.
So, IMO management has two reasons for currently only talking about CF.
First, with the Orkambi price tag they want to emphasize to society as a whole that Vertex is a CF company devoted to addressing the unmet needs of CF patients, both those not currently served by the company and those who are and can benefit from even better drugs. Thus the North Carolina deal.
Second, the CEO is taking a more traditional Pharma approach to talking about his pipeline. Established companies who fund themselves with product revenues limit for competitive reasons their public discussion of what they have in their labs and clinics. No revenue biotechs need to excite investors who are the source of their funding and so talk earlier and more about their research. Mr. Leiden is playing it more close to the vest. Whether this is just smart or a way to manage his image time will tell. He certainly does make a good presentation, but then so did Carly Fiorina at HP before she screwed things up so royally.
What I get more interested in is Why the company hasn't said Anything about the two annual meeting resolutions that did not go management's way. May be this week will provide some answers but for now they just look like petulant, possibly passive aggressive children.
Finally, for those who are so concerned about Vertex being only a CF company I offer this thought: It is only possible to have a Second or Third or so on successful product when you have a sustainably successful First product and Vertex is rightly attending to that absolute precondition.
Papa, it's certainly your right to criticize the company anytime you like. I agree that the company needs to achieve timely approvals of the rest it's pipeline to deliver sustained growth and profitability for shareholders. However, it's not 'too late' for such progress and the VRTX pipeline now will have the funding needed without further dilution (unlike GLPG) to develop these drugs. I guess one would like as a shareholder a smooth predictable flow of clinical trials to insure one's investment. As evidenced by the slowing growth at BIIB on Friday, it's never easy or smooth for the most successful companies. Perhaps at next week's Q2 conference call we will get more information about the timing and progress for clinical trials in both CF and non CF assets at VRTX. as well as the projections for growth from Kalydeco and Orkambi.
If VRTX various projects in oncology, influenza, spinal cord injury, Huntington's, chronic MS, as well as it's next generation CF treatments start clinical trials over the next 12 months, I believe that the long term growth plans expected from management will, eventually be successful. It just takes one or two blockbusters to make it to the top tier of companies in pharma. Any success in it's pipeline of drugs offering breakthroughs in such difficult to treat to treat conditions will likely continue VRTX growth. In the meantime, I enjoy the moments like this where VRTX has delivered a success that means so much to CF patients and their families, as well as to long term shareholders as ourselves, and is just in the first innings (to use the baseball analogy) of the development of this market.
Q, there is no doubt that Kalydeco and Orkambi are major achievements in treatment of CF that are a blessing for many patients. But that doesn't relief management from shaping the future of the company and to create a sustainable stream of revenue. In my opinion, VRTX has not offered much information others than referring to announcements to be made in the coming years. Second generation correctors were supposed to enter clinic in 2014. Where are they? Galapagos has a better version of Kalydeco in the clinic. Where is VRTX back-up for Kalydeco? Where are clinical candidates outside CF? Why go after cancer candidates that improve chemotherapy when the company's focus is on orphan drugs?
I am a long time investor in VRTX, cost average of my shares is below $20, so I do not complain about my capital gain, but I dare to criticize the management of the company I am invested in when I feel somethings missing.
How ironic that this thread starts with the topic 'historic day' and shares the hope and happiness of mblock66, redsox2004, and the happiness of Verity over the fact that 508dd CF patients are finally receiving without delay by health insurers, prescriptions of Orkambi in the first weeks of launch following FDA approval giving them hope that their loved ones can live a longer and more healthy life.
And then we have upset with investors on this board, apparently not satisfied with the fact this company, even after a horrible week for biotech in general, has a market cap of over $30 billion, attributable only to it's CF drugs. These drugs are not considered by Vertex critics as being cost effective or sufficiently beneficial to justify the current market cap.
Let's consider the facts: successful companies in the orphan drug markets e.g. ALXN, have been priced at even greater valuations than VRTX because they offer the possibility of breakthrough treatments in lethal diseases that have until now have had no real treatment for the underlying cause of the disease. VRTX took the financial risk to spend the last 15+ years to develop (with the assistance of the CFF funding less than 10% of the total cost) Ivacaftor, lumacaftor, VX 661, and the second generation correctors after acquiring Aurora Biosciences. Since becoming CEO, Leiden has focused the cash resources available to VRTX (following the loss of the market for telaprevir) on getting FDA approval of the CF drugs to achieve the needed revenue that will now permit the clinical development of the rest of the VRTX pipeline. He out-licensed VX 787 to JNJ/Jansen to accelerate monetization of that asset for VRTX without draining it's cash resources needed to develop the CF drugs. The proceeds from the CF drugs will now finance the future clinical trials for all the drugs in the VRTX pipeline. Simply put, VRTX's future R&D success will be financed by Leiden's focus on succeeding in CF. IMHO a good strategy
As I have said on many occasions, The Board should let go of Leiden and hire someone who is capable of multitasking. All Leiden thinks is CF. The sad part is he had nothing to do with the initial development of the CF franchise. So I do not understand his fixation with CF. VRTX has over 1800 employees, many of whom are scientists or support scientists with their research. So what are they doing? Where is the leadership in VRTX to have these scientists' work go through trials. Is Leiden is saying they have not produced anything worthwhile?
Honestly I don't think Leiden was looking beyond CF seriously enough. I hope orkambi works well but the efficacy is not like kalydeko and there is risk that the uptake won't be as aggressive as planned and/or patients will discontinue if it's not beneficial enough. This comes back to the strategy, or lack thereof. It's CF or bust with no backup. It also has to be said that the executives and board have lined pockets at this company. Verging on disgusting. Patients will find a way to pay and they know it.
this has been my strongest concern for quite some time (years!):
wonder when they will bring more compounds to the clinic. The cancer candidates seem to be locked up in phase I and do not really fit Leiden's strategy to focus on orphan drugs.
i hope you all have a very bright future! That said, from a SP perspective I'll get back in when it's 80-100. Strong headwinds ahead like the growing outrage over the costs of these drugs along with the fact that Vertex is still a one trick pony. Add in the current market downturn (especially Bio) and I don't see the current market cap as being sustainable. I know Vertex well, worked there and know a lot of people still there. Big concerns around their failure to advance anything else but CF while the SP and poison pill made them too expensive for M&A.
Congratulations, MB! Crossing fingers for you. Our 3 month supply is being Fed Exed as I type this message. Insurance was a SNAP! Doctors did NOT wait for a 3 month appointment...our clinic initiated the whole process. Less than 2 weeks from order to shipping...like we hit the "EASY" button.
Sentiment: Strong Buy
Its been a long hard battle but today my wife's first dose of Orkambi arrived. $64k worth of drugs were overnighted and she takes her first pill today after morning treatment. I have been a shareholder for a long time and it was hard to watch Kalyedeco happen and she couldn't take it. Now being double delta 508 our time has arrived for new treatment. Insurance really didn't cause much of a delay. It exciting times from a personal level. Fingers crossed for a very positive reaction to the drug.
FWIW, Smurph 3 posted this morning on Yahoo 'market pulse' blog for VRTX the following data likely taken from IMS prescription data tracking service:
"Orkambi scripts in second week of launch were 145, up from 14 prior week"
Thanks Verity. Good to know that CF doctors are prescribing Orkambi ASAP, and not waiting for routine appointments to start their 508dd patients on this treatment. Hopefully their medical insurance is approving the treatment quickly after it is prescribed, without too much red tape.
I would guess that with the release of orkambi, there would be quite a few doctors visits that weren't in the normal schedule.That's just trying to put myself in the shoes of somebody that needs this drug.
You will likely get a number on July 29 from the company during it's Q2 conference call. Initial Orkambi prescriptions are being given to patients at their normal scheduled follow up visits to their CF clinics, so I anticipate that if the visits occur routinely on quarterly basis, most patients will be getting prescriptions in the first 6 months, and the only limiting factor will be processing paperwork for insurance approval, which may drag out the actual approval process a bit longer, especially for Medicaid patients who have more bureaucracy to deal with. In one year, most US 508dd CF patients will be on Orkambi, and EU regulatory approval for Orkambi by year end will allow EU CF patients to start getting treatment next year as well.