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?
MY data would have been more accurate if instead of "VRTX has over 1000 employees" I would have said "VRTX has over 1800 employees."
What is tragic is that a lot of idiots on this boards are acting like, well, idiots or buffoons by criticizing HR for stupid reasons rather than well based reasons. So long term holders like me just do not criticize HR often because we do not want to join these idiots buffoonery. This is unfortunate because we are not criticizing the incompetent HR for his incompetent actions -- stupid drug trial designs, lost opportunities to partner with reputable drug firms (the results of 102 were so good we decided to keep it for ourselves), etc., etc.
This is really a shame. I wish these idiot childish posters who always talk about HR and his secretaries and his son will go away so that we can constructively criticize HR and ask his cronies on the Board to fire him and hire a competent person.
It is true that the efficacy of Orkambi was not very impressive but i think the lack of high efficacy as well as approval was baked in the PPS already. I think investors after digesting the data etc. recently do not now see any viable catalyst in the near term and therefore are taking profit. I submit it is Leiden's fault for not moving other compounds through trials and just mostly being satisfied with the CF franchise. VRTX has over 1000 employees. How many of those are scientists and those who assist scientist? What are they all doing? These are the reasons I believe many are taking profit rather than sticking with VRTX.
Apparently, my June 16 post speculation about the value of Orkambi having been baked in then PPS has ended up being the case.
At this stage I believe we need a new CEO. Leiden can not be given credit for the CF drugs. The CF drugs were developed by the Aurora Sciences company that VRTX acquired a long time ago and before Leiden even came to VRTX as a board member.
I think Leiden is a hard working and well intentioned person but I do not think he is capable of working on several major projects at the same time. He is very good in taking up ONE project and complete it and that is the CF project. Unfortunately, that is his personality type -- well intentioned but inappropriate to lead a company like VRTX. He is the type of person who is great as a PROJECT MANAGER, and I guess he has been a good project manager for CF but that is it.
Let's hope. On the other hand one could say the upside is already baked into the present PPS.
As i have said many times before,.Leiden's obsession with CF with not much publicity about other non Cf drugs gives investors cold feet. The other issue is that the CF Foundation with its windfall by cashing the VRTX royalties is granting money to other drug companies such as Genzyme and some university scientists to look for better drugs for CF. That as well as other major companies nipping at VRTX CF franchise is making investors uneasy. VRTX investors including myself are asking what is VRTX doing with all of its scientists. It has a bloated payroll. I am hoping Leiden will start realizing that he needs to pay lots of attention to other drugs and move them through the pipeline as fast as possible.
"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.
In addition to verity and Qdelfan input, let me throw in my 2 cents worth.
For the sake of argument, let's just say the FDA is not going to approve Orkambi. In that case they have to reiterate the flimsy reason they gave in their presentation to the ADCOM panel which was the improvements were due to Kalydeco and not due to Lumacaftor. Keep in mind it was the FDA that initially did not approve Kalydeco for the 8,500 additional patients who had 2 copies of F508del mutation genes.
So if they are saying Kalydeco is effective for this new group of patients as well, then logic dictates that they must approve kalydeco alone for these 8,500 additional patients which means VRTX would be able to collect an additional $300,000 for each one of these8,500 patients and does not even need to pay the cost of manufacturing Lumacaftor.
The important thing is the number of scripts filled every week and any uptrends in the numbers. So can any of you smart investigators who post here can find out a source that gives the numbers on a weekly basis? That would be much more helpful than what some of these unknown so called salespeople are saying.
Of course I realize such data may not exist.
It is very important for SRPT management to line up kid who could testify in the FDA's ADCOM meeting that they need this drug to be approved.
I will listening to AF who was posting the proceedings of VRTX ADCOM meeting today. The most compelling impressions made on the panel members were those by patients and their families who testified about how VRTX drug combination helped them. Below ist AF advice to SRPT management:
"SRPT and BMRN take notice: Patient testimony is important!!"
regarding the effect of patients and their family testimony, AF stated the effect that patients testimony had on the ADCOM committee members:
"Morrato admits to being swayed by patient testimony, that stuff matters,"
I have always maintained that lots of these so called FDA scientists who evaluate drug data do not know what they are doing. Let's face it, they are not the best or they will be working for a drug company making many times as much money as they are on government payroll.
BTW, please don't tell me they are a dedicated bunch who are forgoing money so they can help humanity!
Tiny Corbus looks to take on Vertex with experimental lung drug
— 5:40 PM ET 05/04/2015
By Natalie Grover
May 4 (Reuters) - Corbus Pharmaceuticals Holdings Inc's ( CRBP
) experimental drug for cystic fibrosis, a rare lung-scarring disease, could be a real money spinner and pose a challenge to rival Vertex Pharmaceuticals Inc's ( VRTX
) successful treatment, Kalydeco.
Corbus, a tiny biopharmaceutical company, received $5 million from the Cystic Fibrosis Foundation last month to develop the drug, Resunab, and expects to begin a mid-stage study this quarter.
The company hopes Resunab will treat every patient suffering from the disease, compared with Kalydeco, which has approval to treat a small section of patients. The disease currently affects 70,000 people worldwide.
Kalydeco treats certain underlying genetic mutations behind cystic fibrosis (CF), while Resunab aims to manage the inflammation the disease causes.
"We're not focusing on a particular subset of patients, like Vertex's drug," Chief Executive Yuval Cohen said.
Kalydeco, which was granted fast-track status by the U.S. Food and Drug Administration, received approval in 2012, six years after the company started an early stage trial.
Kalydeco, which had sales of about $464 million in 2014, is being tested in combination with other drugs to expand its use.
It is expected to generate peak sales of $1.1 billion in 2020, according to Thomson Reuters Cortellis.
Resunab could bring in more than $2 billion in U.S. sales treating CF patients, Zacks Investment Research analyst Jason Napodano said.
Still, Corbus has a long way to go.
"It's too soon to know how significant Resunab will be ...," said a CF Foundation spokeswoman.
CF is caused by a missing or defective gene that makes the body produce a thick, sticky mucus resulting in inflammation and bacterial infections.
This then causes "abnormal inflammation," which leads to fibrosis, and, eventually, death.