You've lost it corona.
Why would I hope for it? I want with all my heart superior cures for these deadly diseases. But like a broken clock that is correct twice each day, mm has it right with Nektarphilia. When you let emotions drive your investment decisions, don't be surprised when things don't work out.
NAPOLI-1 is more than "currently underway", the results are in.
CAMBRIDGE, Mass., June 25, 2014 (GLOBE NEWSWIRE) -- Merrimack Pharmaceuticals, Inc. (Nasdaq:MACK) today announced detailed results from NAPOLI-1, a large, randomized, three-arm Phase 3 study of MM-398, a nanoliposomal encapsulation of irinotecan, in patients with metastatic pancreatic cancer previously treated with gemcitabine-based therapy. The combination of MM-398 with 5-fluorouracil (5#$%$) and leucovorin extended overall survival and significantly increased progression free survival (PFS) and overall response rate compared to the control arm of 5#$%$ and leucovorin alone.
Top line results of NAPOLI-1 released in May showed that the combination of MM-398 with 5#$%$ and leucovorin achieved an overall survival of 6.1 months versus the 4.2 month survival demonstrated by the control arm of 5#$%$ and leucovorin alone. The primary log-rank analysis of overall survival was statistically significant (p=0.012), with a corresponding hazard ratio of 0.67.
In combination with 5#$%$ and leucovorin, MM-398 also demonstrated a statistically significant advantage in PFS, with a median of 3.1 months compared to 1.5 months in the control arm (HR = 0.56, 95% CI [0.41-0.75], p=0.0001). The combination arm also showed a statistically significant difference in overall response rate compared to the control arm (16% and 1%, respectively, p
Abraxane is albumin-coated paclitaxel. How might you pegylate this? NKTR-105 pegylated docetaxel, NKTR's "new and improved" taxane, disappeared without a trace after its Phase 1 completed.
MM-398 is liposomal encapsulated irinotecan. It succeeded in a Phase 3 against advanced pancreatic cancer and an NDA for it is coming soon.
Pegylation isn't the only way to improve these old cancer drugs. Reformulation seems to work wonders.
--"a working drug"
That's not the issue. What's being determined is whether NKTR-102 is a better drug. It's a superiority study, so NKTR-102 loses all ties, and loses all minor victories too. NKTR-102 needs to crush TPC.
Cancer drugs are known for having very long tails, OS doesn't form the textbook bell curve. It's why you have the occasional five and ten year survivors for the nastiest, like GBM and pancan. You need to correlate survival in a statistically significant manner with predetermined biomarkers in a pivotal study. Like the drugs that treat HER2+ breast cancer.
NKTR has had the data in hand for well over one month, enough time to clear up problems. I think NKTR intentionally kept the results blinded prior to the CC. It was the ethical thing to do. It takes several weeks minimum after the data is unblinded to create an appropriate corporate response, whether the data are good or bad. There wasn't enough time prior to the earnings release for that to happen. HR acknowledged it in early January.
To avoid creating the appearance of misleading the public one way or another, the best thing to do is to keep the data blinded. But now that the CC is over, it's time to unblind. It doesn't take very long to do simple topline analysis to see whether the primary endpoint was met. It takes longer to do the secondary endpoint and subset analysis. The biomarker research adds complexity. It takes even longer to craft an appropriate corporate strategy and how to present it to the public. I believe that process is underway right now.
It looks like no one else is answering your question. BEACON results will be out by the end of March. The company was blinded to the results at the time of the earnings release on Tuesday. But I'll bet you anything that two seconds after the CC completed, HR immediately directed company analysts to unblind the data. I wouldn't be surprised if the topline results are known right now.
Because single agent irinotecan isn't the standard of care, only 80 patients signed up for this trial in five years. I believe NKTR stated that a lot of this trial was done in India, where access to current standard-of-care drugs isn't always a given.
Don't forget the CRC study, 102+Erbitux vs. irinotecan+Erbitux, another dead end. On the other hand, MACK's better irinotecan MM-398 has been successfully combo'd in FOLFIRI to treat metastatic pancan. I'm sure there will be other improved-FOLFIRI studies coming, CRC is a big one. You can have the best drug in the world, but if you can't apply it correctly, it doesn't do anyone any good.
Even if statistical significance was achieved, it is doubtful that 102 would be approved. Single agent irinotecan isn't the standard of care for CRC. Might as well shut it down. Kind of disappointing that 102 could
not achieve stat-sig over irinotecan. Hope that's not an omen for BEACON.
Bayer does have a pegylated Factor VIII product under development. BAY 94-9027 has completed its Phase 2/3. FDA and EMA submission is expected in 2H15.
I'll throw in my two cents.
I do not think there is a problem on how BEACON is being run. To the contrary, BEACON is a model of how a Phase 3 should be designed and executed. If BEACON succeeds, I doubt there will be any issues regarding the study protocol.
Here are things that can be questioned. One issue is the limited Phase 2 data that formed the basis for the Phase 3. Phase 2 was single arm with only 35 patients at the dosage being tested in Phase 3. The results weren't totally compelling. In yesterday's CC, there were analyst questions about NKTR's use of single arm studies in Phase 2. Today's standard is to run controlled Phase 2's, really a mini Phase 3. NKTR's response didn't seem very convincing. A second issue is the use of single agent NKTR-102. Irinotecan is typically used in combination treatments. MACK's successful enhanced irinotecan study against pancreatic cancer substituted MM-398 for irinotecan in FOLFIRI.
Inhaled Amikacin is a wildcard. The 30% royalty is quite juicy. I'm not sure how fast NKTR can ramp up manufacture of the inhalers. After years in the doldrums, antibiotics are now a hot product, see the CBST buyout. We need to keep an eye on potential competition.
Only if "CRUSHED" is a synonym for confirmed.
We got your volume today. Lots of volatility, some want out, others want in.
BAX-855 is the best bet for monetization. Most preapproval candidates these days don't get as much upfront as HR negotiated for Movantik a few years ago. It's among the best deals I've ever seen, I give credit to HR for that. Do you think lightning will strike twice?
I don't delete threads, if it's missing it's because of the cr-ppy Yahoo (jerks) message boards.
If you consider the early 2020's at best for 102 approval (if BEACON fails) as a vital company asset, who am I to disagree.
Doberstein presented BAX-855 royalties in a CC a few months ago. He stated 5% under $1.2B, 13% over. Let's do the math, sales vs. royalties. I believe the ramp will be a few hundred million per year (which is faster than Eloctate), and it won't be a significant driver until the end of the decade. I wouldn't be surprised if HR elects to sell off the stream to Royalty Pharma, like he did other assets, for quick cash.
Beyond DTC ads (I was hoping to see a Super Bowl ad promoting Movantik showing some sweating addict straining on the can), AZN has to get Movantik on insurance formularies as a preferred drug. To get a feel for that, check your health insurance policy over the next few months. Then there is the physician education process. Since Movantik works well within 24 hours of usage, don't be surprised if it is used as needed rather than daily by many people, especially if there is a large copay. I feel politically incorrect saying this, but there is a good chance that diversion could be an important driver of sales.
--Why dont you come up with some positive info for a change
Except maybe for hijacked and occasionally cow, no one identifies risk items. It seems like the only risk most posters see is whether NKTR goes to 500pps instead of 700pps. How do you model the future earnings of NKTR?