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.
I googled "business phones". There was Vonage Business Services... in the paid ad section at the top. The actual search return for VBS didn't turn up until the fourth page.
Did I say it wasn't going to be approved?
What I do believe is that it will be years before meaningful royalties ( $100M/year) come from Movantik. And the label is going to play a role in it.
I'll add the details from the briefing document.
1) One died in car accident. I am certain this was due to Mova.
--Study 07 Patient E4073006 was a 54-year-old male in the naloxegol 12.5 mg group with diabetes. He was in a serious traffic accident on Day 146 (Day 60 of Study 07), after a “blackout”
attributed to hyperglycemia. The patient refused to be admitted to the hospital and left the hospital against medical advice. On Day 147, the patient was found dead. The autopsy listed the cause of death as ischemic heart disease secondary to coronary artery disease. This event was adjudicated as a CV death.
2) One died during an operation due to complications. This must have been because of Mova.
--Study 04 Patient E4068050 was a 73-year-old male in the naloxegol 12.5 mg group with multiple CV risk factors. He had a SAE of acute MI on Day 16 that led to surgery for aortic valve replacement and a coronary artery bypass graft, which was complicated by pneumonia, sepsis, and renal failure. The SAE of cardiac valve replacement on Day 19 resulted in the patient’s death on Day 49. This event was adjudicated as a CV death.
For example, BofA analyst Steve Byrne models peak adjusted Movantik sales of $380M in 2020. Let's say sales peak at $500M with an overly generous 20% royalty. That's $100M to NKTR in 2020. Less than that in the years leading up, minus the expenses for the NKTR share of the post approval study.
Now you know of something even close.
There were six patient CV deaths in the Movantik studies according to the FDA briefing documents, all of whom had taken Movantik at some point. The FDA documents supersede all of the AZN and NKTR press releases over the years and should be considered the definitive reference.
The Movantik label can matter too. OREX's Contrave got approved the other day with a 47 page label and a black box warning. The stock got hit hard.
But maybe the label won't matter much. The big thing for most people is the milestone money. There are concerns about how fast the market will ramp up even in the best case and what peak sales might be. Meaningful royalty money could take many years.