What happens will happen this Tuesday. IMHO the Biomarin executives would not be the first people I would ask over to dinner this Thursday. Humble and forthright? No.
Wednesday I am going fishing ...hopefully will put a Cod on the Rod ....
The people on the board are to be commended... as we take more time than analysts before conclusions are formed.
Red, Nerd, Thig, Grey, Finkel, KG, Speed, Fox, MD, Simp and many others ... we peeled the onion and called a spade a spade.
Long (and much richer for it) since 1999.
AVII developed an Ebola therapeutic in under a week. How's that for a reputation setter?
I bet you cannot even come close, while locked in your mothers basement for six months.
it's extremely interesting that what we have been stating on this board for years was actually factual...
this board is better than misguided, economically driven analysts who tilt towards the most recent steak dinner provided to them at the Capital Grille...
We knew drisa was garbage and that you cannot give already suffering kids "sugar" with harmful side-effects
Star (Arnold)... go to confession - you have much to account for
“As a clinician with extensive experience treating patients with Duchenne muscular dystrophy, it is apparent based on these data, that the boys treated with eteplirsen in this study are showing more signs of stability than would normally be expected at this stage of the disease. Remaining ambulatory is critical to greater independence over a longer period of time and a 151 meter difference indicates a marked slowing of disease progression,” said Jerry Mendell, M.D. director of the Center for Gene Therapy and Muscular Dystrophy at Nationwide Children’s Hospital, Columbus, Ohio., and lead author of the publication.
“The safety and tolerability demonstrated by eteplirsen is also important, as exon skipping therapies will require lifelong dosing to be effective. For over three years, it continues to be one of the safest drugs I have used in my career.”
What about just backing the safest and most effective drug tested to date? Why back a drug that is proven to be toxic and NOT proven to be effective? Do we REALLY want to be giving kids the "also ran" treatment, while putting them AT RISK in the process?
Did you miss the memo that Drisa is toxic? Would GSK drop a multi-billion dollar drug if it was effective and safe?
Pull you head out of sand, take a breath and face reality.
Is Pat Furlong backing a toxic and ineffective drug is lieu of a non-toxic effective drug.. for kids with a life threatening disease?
Isn't that a bit diabolical and twisted to say the least?
Drisa is toxic.. where is the proof that it as helped anyone?
ETEP has correlation to dystrophin production.
ETEP has correlation to dystrophin production to improved 6MWT vs. natural history.
What does Drisa have other than injection site pain/swelling, hospitalizations etc..?
What does BMRN specifically have to warrant this drug to be injected into very sick kids
with a TERMINAL DISEASE when there is CLEARLY a better alternative?
What is ultra disgusting is that shorts are attacking a company which is fighting to save the lives of children, children DYING a slow death from a horrific disease. The data proves that ETEP works. DMD experts stated last week that ETEP is amazing, a game changer. ETEP will be a life saver. Drisa is toxic, cannot be dosed high enough and flat out does not work.
Everyone with a brain and heart should be pulling for and pushing Sarepta - period.
You're delusional. Look at the facts, look at the data. We now have YEARS of history and RESULTS.
BMRN has zilch.
Get a grip jelly fish.
People make bad decisions all the time.. so do companies. You would think a company would make better decisions, if the CEO and board do their research, take their time etc..
IMHO, the purchase Prosensa by Biomarin was a bad decision. Why? It's clear that their is no definitive evidence that Drisa works - ZERO. Placing a $900M bet on ten 7-year olds in my opinion is fool hardy.
Where is the data? Was GSK wrong in dumping Drisa - I doubt it. The gamble here is if the drug works, with there is no evidence that it does - there is no data to correlate efficacy to dystrophin levels - there are no advocates/parents/patients clamoring for approval - ZERO....and I am sad to say that.
Fortunately, this is not the case for Etep...thankfully since the beginning of human kind we have a legitimate treatment for this disease.. as disease I helped raise money for beginning over forty years ago.
Thank you Sarepta.
Sentiment: Strong Buy
Today's news is bigger than you think. Today's news is a gift for the shorts - A WARNING SHOT. Pay attention shorts - you have little time.
This panel which has been assembled is there for one reason - THE DATA. Sarepta's recent hires are there for one reason - THE DATA. All of the recent grants for employee's... THE DATA.
If the data was not EXCELLENT - this panel would not exist - period. You heard Wilton and Mutoni before congress - Eteplirsen is a game changer.
If you're too stupid to cover now, so be it.
Pigs get fed, hogs get slaughtered.
The data has brought these people to the table..............
Can you say $150.00?
Where is the proof, any legitimate proof that it works?
Would GSK drop it like a hot potatoe if it displayed any evidence of efficacy?
The only thing we really know about Drisa is that it is toxic and some smarmy french guys gambled with $900M on it, in an attempt to seemingly dupe future job candidates at the administration.
IMHO Biomarin is trying to dupe the FDA with krap data..which from what I can tell has no legs to stand on.
when I heard them say "....the drug did not fail the trial, the trial failed the drug" I almost barfed and laughed at the same time.
Drisa is toxic.
If patients and parents of patients had the choice of taking the red pill (drisa) or the blue pill (Etep).....which one would you think they would take?
It's not even a choice.