rebel and magnam, ditto that. Cohen is famous for turning on a dime. Long 6 mill one day, short 6 mill the next. I think we've been watching Cohen's traders at work since he bought in. And I suspect nearly all of the movement today was compliments of Steve Cohen. As for J&J being down 30% Rebel, I think it's immaterial to them. As magnam is suggesting, I doubt seriously they give one whit about the market cap of ACHN, or the stock price, ever The $225 mill was the negotiated pay-to-play money at whatever premium in order to get at the crown jewels. JNJ trials are going to either make ACHN a penny stock or a $5 billion+ company.
This is a double if the injection number holds and vision also holds. It's a triple if the injection number holds and vision also improves. And it's a 5X if vision improves as much as Lucentis on a monthly regimen. Just my humble opinion.
Igonber, I haven't studied OCAT much, but on a quick read it seems even more speculative than AAVL and ONCE, both which I own. I'll keep it on my radar screen though - thanks for bringing it up. I'm very excited about gene therapy - I think it will usher in a whole new era of modern medicine. And I think ONCE and AAVL are the two best plays for the eyes.
...and that means the air is out of the ballon? Even funnier!
LOL! Did you just say it may hit $300, but that's ONLY a 50% markup? Dude you're funny!
Above I wrote: "under the belief that the apparent benefit outweighs the often debilitating but non life-threatening side effects loosely called "statin intolerance". As a footnote, I think at some point in the future American medicine will start focusing on non life-shortening or life-lengthening instead of just life-threatening. And PFS and OS (Progression-free and Overall survival) will have a new measure attached, QLS (my made up acronym for Quality of Life Score). I'm not on statins, but the more I read about them (liver and kidney damage, digestive disorders, neurological damage, memory loss, depression, anxiety), the more I want to try to lower my LDL and total cholesterol counts some other way. There is a strong correlation between the rise in anti-depressant sales with statin sales since 1990 as well. The story about PCSK9's and ETC-1002 is far from written yet.
I think the market is pretty smart, George, and, notwithstanding price manipulation by large hedge funds et al that could push the price down, there's a really good reason why BLUE went from $40 to $190.Unlike most effective drugs on the market - the vast majority being inhibitors of one or more proteins or enzymes that in many cases silence or alter a particular gene expression - gene therapy goes to the source: the defective gene. BLUE now looks like they might be 8 for 8 on the cure side, and the SCD case is really just an extension of what has already been proven in beta-thal. So unless there is some off-target proto-oncogene or oncogene activation, the data, however few, is black-and-white 100% compelling. That's opposed to many phase 1 trials that are a gradation of response: X% complete response, Y% partial response, and so on. Then you have the individual metabolic variables that come into play that, so far, we have not seen with lentiglobin. We are witnessing a new frontier in medicine that is every bit as revolutionary as Herceptin was in the 80's. Same thing with CAR T cell therapy, although that is not quite as black and white as this has been so far.
It is perhaps the worst genetic disease an infant can be born with, as for 2-3 years parents must literally watch their child being tortured to death. The baby's skin is so fragile it blisters and falls off to the touch. They must remain bandaged and immobile, and scream with pain constantly. Only 2500 cases per year globally, so it won't get any attention from a public company standpoint.
Maybe this can be Nick's pro-bono work once BLUE is worth north of $20 billion market cap.
Can you imagine Nick at his dinner table tonight? "Karin (his wife), you're not gonna believe this, but I think our company just cured sickle cell." And then his five daughters will chime in, "what's a sickle cell?" And Nick will say," it's a terrible, painful disease that affects 500,000 people around the world who are born with it, where your red blood cells are deformed and can't carry enough oxygen throughout your body. Now we'll be able to cure them so they can lead normal lives!"
George, you're the guy standing next to the stone cave right after The Ressurection, saying, "Oh come on guys, really? Have you seen anyone ELSE do it?" ;-)
Weighing net present value of lifetime treatment versus greatly improved quality of life issues, perhaps this will price in the $200-300k range. It will be hard to price this much lower than beta-thal and still justify the beta-thal price. At 300k delivered to 10% of global SCD population, that's 15 billion in revenues.
All great questions dcx- same ones I have! I'm surprised they didn't mention northstar patient status. I hope no news is good news!
I think research scientists who are CEOs sometimes get lost in the science. Identifying opportunity, marshaling resources, hiring the right people, and shepherding a drug to approval is the domain of a business leader who understands the science, but not necessarily at the molecular level. Nick is that person. And even better, he really CARES about this science and its potential.
The average per patient lifetime cost of treating SCD, calculated in 2008, is $480,000. Adjusted for annual increases, by 2020 that should look more like $650-$700,000. Assuming costs continue to rise, the price of this treatment has clear economic benefit at $500k.
The reason I posted a topic called "doc's arrogance", is that Cardio doc's want to keep statin patients on their statins at all costs, including myalgia and other side effects, under the belief that the apparent benefit outweighs the often debilitating but non life-threatening side effects loosely called "statin intolerance". But the irony and the conundrum of this position is that statin intolerant patients have poor compliance and/or end up on sub-optimal doses, thereby negating the supposed benefits of statins in the first place. So that's why they were pounding their pro-statin drums at the PCSK9 adcomm meeting, but you see the fallacy of their argument: "ETC-1002 had better first prove that it can lower cardio AEs as well as statins for patients who can't or won't take statins!" (That's my paraphrasing there to show the circular illogic to that argument.) this is why I think the FDA will seriously consider a post-approval study on the ability to lower cardio AEs.
This time for sickle cell disease! At 4 1/2 months after treatment, 32% anti-sickling hemoglobin; at 6 months, 45%. BLUE has achieved a functional cure. Amazing is all I can say.