Look at this agreement for improved irinotecan. And MACK retains all US rights to MM-398. You'd be singing hosannas to HR if he negotiated these terms for NKTR-102.
My favorite recent corona comment... "Sadly, they are apples and oranges apart .. I'll leave it with ya for ya to wonder." English translation... "I have absolutely no clue myself, if I did I would explain it".
Under the terms of the agreement, Merrimack receives a $100 million upfront payment from Baxter, which will be recorded by Baxter as a special pre-tax in-process research and development charge in the third quarter of 2014. Merrimack is also eligible to receive $120 million in regulatory milestone payments related to the first pancreatic cancer indication as these milestones are achieved, and $280 million in development and regulatory milestone payments for a second pancreatic cancer indication.
Merrimack is also eligible to receive $220 million in future development and regulatory milestone payments related to two additional indications. Merrimack has the potential to receive $250 million in sales milestone payments, as well as tiered royalties on net sales of MM-398 in the licensed geographies.
MM-398 is nanoliposomal encapsulation of irinotecan. It had a successful Phase 3 extending the PFS in metastatic pancreatic cancer, replacing irinotecan in FOLFIRI.
Contrast the approaches in developing new, improved irinotecan. MACK improves FOLFIRI and is now moving forward in a partnership. NKTR tries single agent in colorectal cancer (going nowhere), then ovarian cancer (going nowhere), now breast cancer. NKTR-102 may prove to be better than MM-398, but there is no denying which company has better focus on drug development.
Difference between disease maintenance and cure is that maintenance produces recurring revenue and cure is one time only. The method of payment to BLUE, if the technology succeeds, is important. Would BLUE be paid a big amount for the single treatment, or would they be paid a small amount each year that the patient remains disease-free? That's why VRTX Kalydeco is so highly valued and GILD Solvadi not so much.
AF has it right.
Until recently, orphan drugs were dealt with informally by the FDA. Because there are few patients for such drugs, it didn't make sense for companies to run full blown studies. It would be a money loser even if the drug proved out. Instead, there was an informal compassionate use process. To get promising/useful orphan drugs to patients, doctors would submit paperwork for an investigator sponsored IND. That would allow patients to get drugs that hadn't gone through the formal approval process, and also provide feedback to the FDA about the efficacy and safety of the drugs.
The problem with that process is that there was no real incentive for pharma to develop new orphan drugs. So the current orphan drug process was implemented. Companies that develop and successfully test such drugs are virtually guaranteed profit and are given market exclusivity. CPRX is exploiting a loophole. They are taking a drug that has been off-patent for years. Although unapproved, 3,4-dap has been used successfully for decades. If there were problems, the FDA would have shut it down. CPRX did no science or discovery, that was done by others long ago. By jumping in and running a Phase 3, they will be able start charging patients a king's ransom and shut out all competition.
About CPRX... their management is a bunch of slimeballs, brothers under the skin with Martin Shkreli. Their main drug, Firdapse, is a generic given away for free for the past twenty years to Lambert-Eaton Myasthenic Syndrome (LEMS) patients by a small private drug company, Jacobus Pharmaceuticals. CPRX is trying to exploit FDA rules about orphan drugs to gain exclusive rights to market it in the US and start charging patients many $10K's/year. I hope you won't be offended if I root against you in that one investment.
Search for "Catalyst Pharma: Orphan Drug Poseur, Profiteer".
I haven't followed CARA closely. My impression of kappa-agonists is that they seem to have narrowly focused markets. In Phase 2's, CR845 has been shown to reduce post-surgical use of other opioids. Now there is a new Phase 2 with hemodialysis patients experiencing uremic pruritus. To use a baseball analogy, you might hit a few singles with this class of drugs, but it's hard to imagine hitting a home run.
The only biotech I have money in today is BLUE. There is a incredible wow-factor in what they achieved in the several patients with beta-thalassemia major they have treated. There is going to be an incremental readout with a few new BT-major patients before the year is out, and at least one with sickle cell patients next year. The only cure for both diseases is bone marrow transplant, but there are few matches for patients and rejection issues, including death, are a never ending problem. BLUE's approach is to extract patient's bone marrow, "infect" it with a proprietary virus that makes the cells produce normal globin, then reinsert the marrow. It appears to have all the benefits of a successful transplant without the rejection concerns. By this time next year, I expect BLUE to trade well over $100 or in the single digits, depending on the updated results. In other words, it's highly speculative, but it has a huge potential payoff, especially if sickle cell works.
CARA has peripheral kappa-agonist CR845 in the clinic, both oral and IV versions. CARA also has CR665 in preclinicals, in which they claim 500 fold separation of doses compared to the 15 fold separation for the unnamed drug(s) in the NKTR PR.
I wonder how sustainable the dividend is. A lot depends on the performance of the CT Legacy portfolio. They did yet another capital raise. It's hard to believe there are still huge opportunities in the US, are they going to buy in Europe now?
Movantik approved with no pre-approval CV study
As expected, FDA approved AZN/NKTR’s Movantik (naloxegol) for opioid induced
constipation (OIC). Based on the outcome of the June advisory committee held for
the OIC class we did not expect a pre-approval or post-approval cardiovascular
outcomes trial (CVOT) to be required. AZN/NKTR is required to conduct a postapproval
healthcare database surveillance study which will commence after launch
(NKTR will pay one-third of the $5-$15mn estimated cost over 7-10 years). We
removed our modest risk-adjustment for Movantik, which raised our PO to $11 (from
$10). We remain cautious on the OIC market opportunity due to relatively low
physician interest owing to an assumed acceptable efficacy from OTC laxatives. We
maintain our Underperform rating on NKTR shares.
Launch expected in 1H 2015
AZN will launch Movantik in 1H 2015 after DEA acts on proposed de-scheduling of
the drug (from schedule II to an uncontrolled drug). We model peak US sales of
$425mn in 2020 ($85mn in royalties to NKTR). In addition to regulatory milestones,
NKTR is also eligible for up to $375mn in sales based milestones. The Movantik
label appears relatively benign with no mention of cardiovascular safety concerns.
Drug-drug interactions and serious side effects (potential opioid withdrawal and
stomach tears) are listed in the label.
From Marty's press release:
Mr. Shkreli continued, “We have made significant progress under Marc’s financial direction and he will be leaving the company in a strong financial position for future growth. We look forward to Marc’s ongoing contributions during this transition period and wish him well in his future endeavors.”
From the latest 10-Q:
These conditions raise substantial doubt about the Company’s ability to continue as a going concern. These unaudited condensed consolidated financial statements do not include any adjustments relating to the recovery of assets or the classification of liabilities that might be necessary should the Company be unable to continue as a going concern.
It gets better and better. RTRX CFO Marc Panoff resigned to "pursue other career opportunities", i.e. avoid going to jail. Independent director Jeffrey Paley also resigned. The resignation of Mr. Paley triggered the NASDAQ delisting process as RTRX no longer has the three required independent directors on its audit committee.
Article this morning said there was hedge fund selling in a lot of the momo stocks yesterday as they try to get into the Alibaba IPO. BLUE is definitely a momo stock.
Looked up the Feuerstein-Ratain rule. It pertains to Phase 3 studies of cancer drugs (like NKTR-102 MBC). If the market cap of the stock is less than $300M four months before the release of the Phase 3 data, then the Phase 3 study will fail. Since 2000, the rule has 100% accuracy, it successfully predicted 36 study failures. Market cap above $300M, there is about 60% chance of success. NKTR market cap is well above $300M.
BLUE is an interesting speculation with explosive potential. BLUE is only 3% of my portfolio, if it drops more I may add a couple hundred shares. If BT and SCD fail, BLUE is going to the single digits, but it won't wipe me out. The science from this distance looks as good as any early clinical stage biotech I've seen.
FWIW, the data on patients-to-date was peer reviewed and presented at the 19th Annual Congress of the European Hematology Association (EHA) . The PI Dr. Marina Cavazzano is well respected in the field.