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 $2 billion in U.S. sales treating CF patients, Zacks Investment Research analyst Jason Napodano said.
Still, Corbus has a long way to go.
"It's too soon to know how significant Resunab will be ...," said a CF Foundation spokeswoman.
CF is caused by a missing or defective gene that makes the body produce a thick, sticky mucus resulting in inflammation and bacterial infections.
This then causes "abnormal inflammation," which leads to fibrosis, and, eventually, death.
"VRTX pipeline beyond CF remains a conundrum."
I totally agree and have said it before several times. Leiden seems to be one of those scientists that can only concentrate on one thing at a time. Unless he gets off of his #$%$, or the BOD requires him, and start taking all of these potential compounds through trials I am afraid we will have a fiasco on our hands similar to Telaprevir. All we need is another company coming up with a better drug for cf and we are dead the way Lleiden is leading VRTX.
At least one positive news from these idiots at NKTR:
"The trial compares NKTR-181 to placebo in opioid-naive patients with chronic low back pain with approximately 200 patients being randomized to each arm."
Of course we all remember the idiotic trial design for 181 which failed. At least the new design makes sense -- compares181 with PLACEBO which should have done originally.
OMG, I can not believe these incompetents finally have come up with a drug design that makes sense!
"discussion with the FDA by the END OF THE YEAR?"
Well the answer is very obvious. The longer this painful episode drags on, the longer amount of time the incompetent HR and the idiots around him have to get more free options and sell them pocketing the money while laughing about how they misled us.
Remember, HR saying "the results of 102 were SO GOOD WE DECIDED TO KEEP IT FOR OURSELVES" rather than partnering it?
I have been investing for over 40 years and invested in many biotech companies as well as observed the operations of many others for possible investments . In all honesty, I can tell you HR is the worst CEO I have encountered.
Sometimes, incompetent CEO's at least have a knack in finding competent people to work for them to compensate for their own incompetency. Unfortunately, the incompetent HR does not have that knack and surrounds himself with incompetent people like himself. That is a double whammy for us long term investors!!
The problem is that he also has recruited some incompetent people for the Board of Directors who keep him in his role instead of firing him.
"This is not something that Nektar derived on it's own the FDA played a big hand it the how"
How do you know that? HR has not said a better efficacy was required by the FDA. If in fact that was the case, HR should have told us about it when he announced the trial design that it was the FDA's requirement.
If in fact the FDA did require better efficacy then let us face it 102 is a failure for the most part.
Regarding the FDA approving 102 for the 2 indications that showed good efficacy, I am not very optimistic because historically the FDA has not allowed cherry picking of data as some optimists here suggest. Of course I hope I am wrong about that.
If the FDA did not require a better efficacy and HR and the idiots around him decided they would go beyond the minimum requirement of proving improved side effects then the HR stooges on the board should fire this incompetent CEO. Enough missteps, enough blunders, enough failures. It is abut time to have a competent person hired to get this sinking ship back afloat!
NKTR Pharmaceuticals uses a process PEGylation conjugate technology to improve existing drugs. It only does that with drugs that are not patented for the very reason that they know patent owned drug owners will sue the hell out of them once they spend tons of money to prove their product is better than the patented drug and they could lose their #$%$ in suck a litigation that they are bound to lose. these patent violators only hope that if their product is an improved version the patent owners will buy them out. I would not want to own such a company because they first of all may not be successful and even if they are the patent owner will not be willing to pay a lot to buy them out.
That is why NKTR only works on drugs whose patents have expired.
Not true. I am here and enjoying PPS near all time high. Good luck to your short. You will need it.
I Think NKTR will add a point or two from here if the BOD immediately fire HR. The problem with PPS has been, is, and will be as long as this incompetent person is allowed to head NKTR.
Frankly after 102 did not meet its end points, to be honest, I was so upset with HR that I proceeded with a post that indicated he should now be fired. I decided not to publish it because of all the unrelated #$%$ that Kennedy and his aliases pollute this board with by not giving him more reasons to publish his unintelligent posts.
In a post, however, I did raise a question that indicated my displeasure with the failure.
Through my posts for the past 7 years I have repeatedly complained about the incompetency of HR only to be lambasted by Multi while he was alive (may God bless his soul - he always did have good intentions though) as well as a few other long term holders.
All along HR and his teams had tooted 102 for its low side effects and for its efficacy to be at least as good as Irinotecan. I do not understand why they moved the goal post higher than that only to end up in failure. One always aims at the minimum and if the results end up better, that is gravy. Even though questions have been raised as to why the higher efficacy requirements were included in the study, HR has not responded as far as I know..
It seems to me if the FDA required higher efficacy for 102 then 102 is dead. If FDA did not require it and the higher efficacy inclusion was HR and his team's idea then they should be fired.
HR and his incompetent scientific and medical officers, in failing several times including not partnering 102, failing 181 for faulty design, and now with 102 fiasco have caused us a lot of pain.
Frankly, the only reason I am sticking with this stock is because of the tremendous potential
in its technology. As far as this management is concerned I believe it is time to give HR his pink slip and hire a competent person to run the show.
" Do not sell here. If you sell you are throwing away 50,000 dollars (on 100K investment). I do not know any other big-cap biotech which can appreciate so much in a few months."
Heed Third's advice. He save me a lot of money. Last year, when I got very impatient with VRTX management I sold all of my VRTX shares when it was at $50+ ( mind you with a good profit since I have been a VRTX investor since its inception). I announced what I had done and why. Thank God Third came to my rescue and posted a comment about my decision -- something similar to his present post -- that got me out of my emotional decision to sell and the fact that I would lose a golden opportunity with my decision. On the basis of his post I bought all my shares back, albeit at a bit higher price, and now the stock is over $110.
Thank you Third for your wise advice.
"I expect it by April/May, there has been no adcom meeting which indicates early approval"
And yet it is trading about $120. One wonders if it is because investors believe it may not be approved. With the results meeting its end points and meeting its statistical significance and no scheduled adcom, it is crazy that VRTX is not trading at at least $150.
"Looks like your wish is being granted"
Not really, as I said it in my post I was hoping it would go down $6 so I could really load up. Nevertheless, I added more shares at low 11. That was a gift. Hope it would go down more so I could add.