Totally agree about the tightness of the DMD community. They're not banded together looking for a new way to control their child's cholesterol. Their connection is forged at the most elemental level: their children are afflicted with a fatal disease, and they will leave no stone unturned to identify the avenues with the most promise. To suggest that BMRN's market clout or marketing prowess will sway these parents to overlook drisa's deficiencies is absurd. If you were in these parents' shoes, would you? Does anyone know if Adcom will be streamed or otherwise available for consumption? I'll gag if all we have is AF's tweets, but it promises to be great theater.
Nice post indeed. My only quibble is the phrase "smoking gun." I think of it more as a pyrotechnic, or incendiary, that has yet to ignite. Shorts might want to check with that Giants linebacker about what happens when you're standing next to something that instantly moves its energy from "potential" to "kinetic."
Even if the call floor is set at $100? To the SEC, they say "Hey, that's the price level we set. We are not responsible for any market force that takes the price BEYOND that level" -- hence, no insider trading? Thanks.
I'm toying with this scenario, and hope that someone can point out why it can't work.
We've got a short interest through the roof, but we also have a drug (and platform) that is poised to achieve Big Things. Just for purposes of round numbers, say that PharMax wants to buy us for $100 a share. Announcing such a proposed deal would do... what? The easy answer is "push the pps up to just under $100," baking in a discount that the deal "might' not happen. But what happens to the shorts? Unless there is a fixed tender date (i.e., shareholders as of such-and-such date, which would create a "cap" at $100) , their need to cover would have to push the pps up significantly in the short term. PharMax, anticipating such a move, could load up on calls that would cut the price of their buyout by a substantial margin. It's a scenario made possible by our distinctive anomaly -- i.e., our extremely high short interest -- and it "seems" to make sense? So I'll toss it out there for comment, hoping that the discourse is more interesting than this "cult" nonsense.
I get the min 40 by Adcom. What's the number on the day we hear about filing -- especially if the news arrives before the 60 day mark? THAT could be a true feeding-frenzy day.
Agreed. So we gain the news of the NDA filing...which sets up 10 weeks of.... what? .....until adcom. I cringe when I think about the pear trees the shorts will try to plant during that "dead" period. If there were ever a time for us to pull the trigger on a partnership somewhere in our pipeline, that would be the time to do it. Interesting times ahead, to say the least.
Selected excerpts from Boston Globe story, Aug 6, 2014.
“This really illustrates why technologies like ours are a critical asset to our nation’s infrastructure,” Berry said. “You just don’t know what the next threat is around the corner. We’ve demonstrated over the years that we can adapt our technology to go from bug to drug within days.” (Diane Berry, Sarepta’s vice president of global health policy and government affairs)
The Department of Defense, which owns the inventory of Sarepta’s Ebola treatment, has steered billions of dollars to pharmaceutical companies, research labs, and universities since 9/11 and the subsequent anthrax attacks launched through the US mail.
Sarepta, better known for its development of a drug to treat Duchenne muscular dystrophy that company officials expect the FDA to approve next year, began working on an Ebola treatment in 2010 through a program funded by the Department of Defense. The company was awarded up to $291 million to develop treatments for Ebola and Marburg viruses.
To cite one example: thanks to ebola, Tekmira is having a difficult time reminding people that they are an HBV biopharm. I think we played this Marburg piece of news very deftly, allowing the imprimatur of the New England Journal to do all the talking.
Some here have suggested that suitors are probably kicking our tires. If that's true, we just threw a nice infectious-disease chip onto the turnip truck for the suitors' accountants to include in their value computations. Can't hurt, right?
This may be more a testament to the market's indifference to AF, who remains the primary source for the adcom scheduling, yes? And it's interpolating to then conclude that the NDA will be filed.
I'm totally OK with drifting a bit until confirmation arrives, if only because the resultant pop will be that much bigger. And a larger pop might catch more shorts unawares, which is a very good thing.
It's all good.
Loved this note: "Analyst Debjit Chattopadhyay has a total average return of 31.9% and a 67.4% success rate. Chattopadhyay has a 71.5% average return when recommending SRPT, and is ranked #18 out of 3681 analysts."
DC ain't gonna risk his street cred on a spec play. He flat-out knows Sarepta, and he's been waiting for this. (Like most of us have.)
Doctors are more plugged into the InterWeb than most -- and they will instantly know the PRECISE merit/drawback profile of both drugs. These are physicians who practice pediatric medicine, and every pediatrician I know thinks twice before prescribing an aspirin to kids. In marketing, there are push strategies (think "sales force") and pull strategies (think "building loyalty among customers". We're the poster child for a pull strategy, and our best sales force will be the people staffing the telephone. And all we need to pull the physicians on board is one NEJOM article that waxes poetically about Granny's lemonade.....
Worth revisiting Ed's remarks on the Q1 concall. The high five he gives his Genzyme bud (no, not THAT Bud) may indeed resonate across the RNA therapeutic landscape....
"Before we open the call to questions, I would like to note that we have recently hired Dr. Bruce Wentworth as Vice President of Biology. Bruce joins us from Genzyme where he most recently oversaw rare disease research. In this role he led a team evaluating therapeutic options for various rare diseases such as DMD, congenital muscular dystrophy, FSHD and Pompe disease.
Bruce help develop novel methods to treat muscle diseases using antisense oligonucleotides monoclonal antibodies and small molecules. Bruce will now lead our research team and structuring our research portfolio to maximize our PMO chemistry beyond DMD focusing on moving our other candidates in the pipeline forward including a adult-onset Pompe disease, Myostatin inhibition and other rare CNS and neurogenetic diseases as well as anti-bacterial agents.
Bruce will also oversee a process we are creating to streamline the development process for follow-on in rare exons. Bruce will be critical in developing leading products for important indications utilizing what we believe is a best-in-class chemistry across the RNA therapeutic landscape."
** a large number of parents are going to push for it **
I'll buy into that vision if I can preview comments from ANY parents who love the drug, based on the benefit(s) they have witnessed with their child. I get that PF can bang the drum and muster staunch PPMD supporters, but where are the rank-and-file, boy-in-the-street testimonials? If there's a din or a clamor, someone is doing a good job of keeping a lid on it.
And here's why I think that view will NOT work. No less an authority deems this adcom "the biggest thing EVER in sliced biotech," which alludes to the high-profile stakes of this FDASIA game. If the FDA catches even a whiff of a problem, there is no way the feds will push a problematic drug out the door, only to be assailed in weeks/months/years to come when kids who have take it begin to REALLY suffer. Lots of people are tuning in for this reality show, and the outcome needs to be squeaky clean. (But I can envision the wording on etep's black box warning: "ingesting double your body weight of this drug may or may not produce health effects, because the FDA cannot identify ANY dosage that provokes a safety issue.")
I have no problem with people promoting their alternate view of the universe -- misguided though it may be -- unless they start hurling personal insults in the process. But the lowest of the low are people (like this amoeba) who feign interest in the welfare of the boys while pursuing their nickel-a-post agendas. There is something missing from their cortex, certainly.
There's a reason why John McCain hasn't reared back and socked The Donald. McCain lost full range of motion in his arms when he was being tortured in Hanoi.
I'm way ahead of the time curve here, but I'm operating from Sandy's success scenario.
I hope every one of our boys participates in the Adcom process. Even Trump should endorse these kids as true heroes.
Gee, what a treat: AF pledges to live tweet the proceedings. I'd rather hear from Don Seiffert of the BBJ.
The FDA gets exactly what it wants and need: a high-profile stage upon which it can demonstrate its fealty to Congress and total obeisance to FDASIA.
Play this out. If the back-to-back days expose the shallowness of data mining on one side, and the depth of evidence of efficacy and 100% safety on the other, the feds will have an easy choice: a green light for etep, and a demand for more trials on drisa. And that will be the last we ever hear of that toxin.
I hope Ian is up to the task of post-adcom PR. Our kids should be on TODAY, and I think Jerry Mendell deserves a little limelight, too. Same with some of the people from the adcom dais. Here's their script: "We're used to dealing with lots of gray areas. There was nothing gray about this drug, and we feel really, really good about our decision to make it available to kids."
If I'm the TODAY producer, I'd want to hear from Jerry Lewis, too.
Heard anything lately from PPMD? If PF steps to the microphone, will she start her comments by citing her conflicts of interest? Here's my prediction: after we hear from the feds that our NDA will be filed, we will see Sarepta cites beginning to show up in her blog and on her website. As Hamburg's hubby would say, there's nothing wrong with hedging your bets.
Last prediction: I think Marge will make an appearance at Adcom, and endorse the "new path" the agency has demonstrated at the proceedings. Same with Elizabeth Warren. Hmm. The last time we saw THAT pair together they were participating in an impromptu TV interview in Boston.....