Yes, the big takeaway from it for me is what it takes to commercialize or even run pivotal studies with BLUE's gene therapy products.
There were two big items that Leschly identified. The first is the ability to produce the lenti-virus in quantity. The second is the ability to fully process the extracted cells within 48 hours. BLUE can do them onesie-twosie. It's another thing to have a solid quality controlled processes and processing centers to do these tasks. Big pharma is good at these tasks. BLUE is trying to develop the capabilities in-house. This will require a large amount of capital (I predict more secondaries) and represents real risk. It's risk beyond those of clinical trial success that most of us are focused on today. It's a lot different from the gene splicing research that most associate with BLUE. We need to track these other capabilities closely.
I believe the approach is designed work in both hemophilia A cases.
One interesting point is that the half life of ACE910 in preclinicals with primates was 3 weeks. BAX-855 had a half life of about 23 hours in primates. Although very early in clinical studies, this could radically alter and improve hemophilia A treatment if it pans out.
Here's a link to a full paper describing the biochemical engineering that went into developing the molecule. I know it's something you'll really appreciate. Search for "Identification and Multidimensional Optimization of an Asymmetric Bispecific IgG Antibody Mimicking the Function of Factor VIII Cofactor Activity".
We missed this a couple of years ago. Now it's on the Roche roadmap. Although it's way off in the future (2017+), RG6013/ACE910 (and gene therapy too) both have the potential to make existing factor VIII treatments obsolete.
Search for "Hemostatic Effect of a Novel Bispecific Antibody (ACE910) Against Activated Factor IX and Factor X in an Acquired Hemophilia A Model"
Conclusion: The bispecific antibody against FIXa and FX, ACE910, exerted FVIII-mimetic cofactor activity in vitro. Furthermore, a single dose of ACE910 demonstrated hemostatic activity comparable to twice-daily repeated doses of 10 U/kg porcine FVIII in vivo. Moreover, ACE910 exhibited high subcutaneous bioavailability and approximately three-week half-life in a non-human primate. Our bispecific antibody against FIXa and FX is a subcutaneously injectable, long-acting agent that removes the need to consider the induction or presence of FVIII inhibitors and may establish a novel principle for the prophylactic treatment of hemophilia A patients.
BAX counting on BAX-855 to, well, stop the bleeding. BAX-855 submission by end of 2014 and on the market by the end of 2015. BAX also plans on bringing next generation factor VIII products (BAX-826 and gene therapy) into the clinic in the next 12 to 18 months. From the BAX CC.
Ludwig N. Hantson - President, BioScience
Well thanks for question, Matt. First of all we continue to expect solid growth across the hemophilia franchise, both on the U.S. side as well as the international side. As far as the ADVATE U.S. market share is concerned we gave you guidance that we expect a decline of high single digits in between the Biogen launch and the 855 launch.
VG, RNG, EGHT all up over 7% today. Screws being put to certain hedge funds with margin calls and short squeezes. Heavily dumped stocks like ACI and MNKD up over 10% too. I love to see the big boys get whacked, poor little rich guys!
And how many acres of power lines to carry it away?
I believe the fusion reactor was developed to power overseas military bases. It solves a nasty logistical problem of getting ample secure electricity in hostile lands. First civilian applications probably will be to provide power to isolated settlements like Arctic villages.
Headline: "Hedge funds bleed as AbbVie reconsiders Shire bid".
Some of them levered up their positions and are getting margin calls. Don't you feel bad for them...NOT.
That's just it, Lucentis and Fovista are combined in a single syringe, no additional injections are necessary. Patients won't even know the difference when being injected. If the Lucentis+Fovista combo works better than Lucentis alone, that will become the new standard of care, that will be what the additional use of squalamine will have to improve. OPHT is more than one year into the Fovista Phase 3.
The goalposts are likely to move during the study. Even if squalamine meets the proposed FDA endpoints, the standard of care will probably become Lucentis+Fovista. Then eye doctors will want to know how much squalamine improves that combo before they prescribe it. The world doesn't stand still.
OK, went from 5% to 30% cash in ten minutes, talked myself into it. I'll sleep better tonight. Let's hear it for cheap online trading!
Shades of 2008... I just saw the DJI drop about 100 points in two minutes. I'm heavy into REIT's and even preferred stocks for dividends right now, they have been holding up quite well. But I'm getting antsy, probably won'[t lose much by selling now and buying back later.
Can reduced sales of bonders be far behind? The switch to copper has pretty much played out. I wouldn't be surprised to see a nasty guide down for the next quarter when earnings are reported.
It's 4forallMannkind now. All the Manniacs who claimed True Religion, it looks like you were worshipping at the wrong altar. You can still sell and get a good pair of jeans at Gap.
-- "If BEACON data is as good as Ph 2"
The Phase 2 data was single arm. The Phase 3 was started on the basis of a data-mined 16 patient subset. The one thing you can say that was exceptional about Phase 2 was the side effect profile. The main thing Phase 3 has going for it is that statistical OS/HR non-inferiority is probably all that is needed for NKTR to pursue approval. The money NKTR stands to get from sales or partnership, which is what really matters and will drive the stock price, directly depends on the superiority/inferiority trend.
Whether or not you like the company's prospects, it should be good for a 10-20% run into the data release in December. I'd be shocked if BLUE didn't trade into the $40's before then. Biotech stock advances into important data releases have been one of the surest trades in the market the past few years.
Probably going nowhere if it gets approved by EMA. Think of approval as a hunting license. You still need to go out and shoot the deer. To sell anything more than a miniscule amount, it needs to be written into the standard of care. Given the mediocre results and the unlikeliness of FDA approval, vosaroxin will be lucky to end up listed as a possible drug of last resort. The world is littered with approved drugs with little or no sales.