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.
Clever Marty, about closed distribution and new doses/formulations.
Tiopronin is a very simple compound to make in a laboratory and it has been off-patent almost forever. But in order to get a another generic approved, you need to show equivalence to the existing drug. By going to closed distribution, RTRX effectively prohibits a potential competitor from obtaining Thiola to run an equivalence study. A standard Phase 3 for a competing tiopronin formulation would probably then need to have placebo as a control arm. But it would be unethical to deny cystinuria patients access to Thiola for an efficacy study. Going ahead with new doses and formulations of Thiola while discontinuing existing ones would present a moving target for any company trying to make its own generic formulation.
Besides, Marty says insurance companies, not patients, will be paying most of the bill, so what's the problem?
More good searches for those interested.
"Thiola, Retrophin, Martin Shkrell, Reddit, and More"
"A generic drug company (Retrophin) buys up the rights to a cheap treatment for a rare kidney disorder. And promptly jacks the price up 20x. A look at what they're up to"
On the Calendar of Events page, above the little calendar, there is a link labeled Past Events. That page has the MS presentation and other past presentations. The audio from MS is available, but presentation material hasn't been uploaded yet. There is presentation material from the previous events. I don't think much changed.
COO Walsh presented. Not much new. Beta-thal patients continue transfusion free. Marked globin levels continue to increase for the two recent patients, expected to flatten out after one year. The beta-thal patients treated to date produced a small amount of good globin prior to the treatment, that is contributing to the success. High levels of marked globin give hope that treatment may even be curative for "beta-zero" (no good globin) patients. Twenty additional patients in the process of treatment, two of which are sickle cell. At least one has received a transplant. Next interim readout by the end of the year, Walsh said a "handful" of new patients plus updates of existing patients will be provided.
Shkreli must have identified another generic drug that he can buy and raise the price by 20 times. It will be another happiest day of his life.
Shkreli/RTRX is increasing the price of Tipronin from $1.50 to $31 per pill. That's $113K/year now for a typical patient for an old generic drug.
From John Mauldin this week.
And then Friday, as if to compound the hilarity, Irish short-term bond yields went negative. Specifically, roughly three years ago Irish two-year bonds yielded 23.5%. Today they yield -0.004%! In non-related un-news from Bizarro World, the Spanish sold 50-year bonds at 4% this week. Neither of these statistics yielded up by Bloomberg makes any sense at all. I mean, I understand how they can technically happen and why some institutions might even want 50-year Spanish bonds. But what rational person would pay for the privilege of owning an Irish bond? And does anyone really think that 4% covers the risk of holding Spanish debt for 50 years? What is the over-under bet spread on the euro’s even existing in Spain in 50 years? Or 10, for that matter?