Here's a case for buying bluebird bio (BLUE).
You might have heard of the hereditary disease beta-thalassemia (BT) major. With this disease, the body produces defective hemoglobin. People born with BT major are condemned to a life of frequent blood transfusions. That causes numerous problems, not the least of which is excessive iron which has to be removed through painful procedures. The only curative treatment is a bone marrow transplant, but that requires a very close match to be successful.
BLUE has developed a virus that "infects" bone marrow cells and causes them to produce normal hemoglobin. The procedure is to extract bone marrow from patient, expose it to the virus for two days, then reinject into the patient. Because the patient's own bone marrow is used, there are no rejection problems. The procedure was tried on two patients about seven years ago. One patient became transfusion free after six months and remains so today. The second produced some good hemoglobin, but not enough to completely eliminate the need for transfusions. Recently two other patients were treated with a second generation virus. -- Both patients became transfusion free after *two weeks*!
BLUE is treating several more BT patients now. More importantly, BLUE is testing the treatment on patients with sickle cell disease, which is caused by different defect in the hemoglobin molecule. Like BT, sickle cell disease causes a multitude of problems and can only be cured by bone marrow transplant. Preliminary results of these trials should be available by the end of the year.
If the results confirm, BLUE could be a multi-billion dollar company. A year from now I expect BLUE to trade at either $100+ or less than $10 per share. There are other applications in the pipeline, but nothing that's going to pay off for a long time if at all. I have about 5% of my money in it, all I'm willing to put into a speculative biotech. You may want to consider it.
Good luck all!
It could be something fixable like Omontys... no wait, Takeda gave Omontys back to AFFY, and the AFFY BoD decided to dissolve the company.
Most of the Q1 GDP decline was due to a dramatic drop in health care spending. A hospital REIT may not be the best investment despite the yield.
So what happened in the first quarter? Evidently, several things. Number one, if you haven’t noticed, the deductibles for most of the ACA programs were quite high, often running as much as $5000 (which, for what it’s worth, is the deductible on my own insurance program – buying a lower deductible is significantly more expensive than simply paying the higher deductible. Go figure.)
The high deductibles were a shock to many people who were used to more-traditional health insurance. They postponed some services and started looking for transparency of pricing for the more expensive services. It is no longer uncommon for a patient to ask for a prescription for an MRI that they can take to another provider across the street who will charge them half of what the hospital provider will. If you’re paying it out of pocket, you begin to pay attention to what you’re paying.
Further, there were a lot of people who didn’t get Obamacare insurance in the first few months and had to wait until March or April for their insurance to kick in. Other people have lost their insurance inexplicably because insurers are losing control of their internal management systems amid all the turmoil. People are postponing what they can until their insurance kicks in or gets reinstated.
I still think the next shoe to drop may be in the third and fourth quarter when hospitals begin to realize that they have significant cash-flow problems. Estimates are that we have about 10% too many hospitals, and the creative destruction of the new healthcare system is going to relieve us of that excess. Only the strong and well-managed will survive. This is of course going to create turmoil in the whole healthcare employment world, etc., etc.
I'm with you corona. FEV1 to get a script, another FEV1 at six months to get the script renewed, then FEV1 annually to keep it renewed. If mm is correct about it being used as an alternative for special occasions, then sales are going to be poor. The big bucks come from daily use at every meal. It's hard to see big pharma stepping up with that black box warning. Would MNKD really try to build its own sales force? It looks a whole lot like AMRN right now.
FEV1 typically means referral to a respiratory therapist. Now required for each and every patient before being prescribed Afrezza. Good luck with that.
FEV1 is typically done by a respiratory therapist, not your GP who prescribes insulin. That means a separate appointment with a referral. It's now required for each and every patient before being prescribed Afrezza. Good luck with that.
How many insurance companies are going to pay for this for an insulin prescription? How many doctors are going to do this? Not many I'm guessing.
BOXED WARNING: RISK OF ACUTE BRONCHOSPASM IN PATIENTS WITH CHRONIC LUNG DISEASE
Acute bronchospasm has been observed in patients with asthma and COPD using AFREZZA. AFREZZA is contraindicated in patients with chronic lung disease such as asthma or COPD. Before initiating AFREZZA, perform a detailed medical history, physical examination and spirometry (FEV1) to identify potential lung disease in all patients.
What AMPE says - "The present enrollment of over 355 patients provides an adequate power of 88%, which the company believes is more than sufficient for statistical evaluation."
What AMPE means - "The data we have aren't close to meeting the 95% CI and aren't going to get any better, so we're going to wing it with what we have."
FRANKFURT/LONDON, June 23 (Reuters) - Bayer has struck an alliance with U.S. biotech firm Dimension Therapeutics to develop a gene therapy for the treatment of haemophilia A, marking renewed interest in an approach to tackle the cause of diseases at a cellular level.
Gene therapy involves inserting corrective genes into malfunctioning cells to get them to work again without further use of drugs. Dimension has been looking into using viruses to carry the genes to the affected cells.
Gene therapy has seen more than 20 years of experiments and a series of disappointments. However, Amsterdam-based UniQure is about to start selling its gene therapy drug Glybera in Europe as a treatment for the ultra-rare disease lipoprotein lipase deficiency (LPLD) with partner Chiesi.
U.S. biotech firm Bluebird Bio last week reported positive results with an experimental gene therapy for another blood disorder, sending its shares soaring.
Dimension Therapeutics will receive an upfront payment of $20 million and may get development and commercialisation milestone payments of up to $232 million. Bayer will also make royalty payments based on future product sales.
Standard haemophilia A therapies are often administered intravenously multiple times a week and may be required for life.
The German drugmaker's established haemophilia A therapy Kogenate had 1.2 billion euros ($1.6 billion) in sales last year. It has two more drug candidates against the type A of the hereditary bleeding disorder in the third and last phase of testing on humans that is required for regulatory approval.
HR is doing a decent job of increasing the market cap, but the dilution has knocked down the PPS. About 30% diluted in the past five years. After an extended lull, there will be a lot of market moving events in the next year.
I took a position in BLUE on Tuesday. That company could be a ten-bagger or more if the technology works for sickle cell, or worthless if the early results for beta-thalassemia turn out to be a flash in the pan. Should know much more by the end of the year.
Also cashing out some REIT's. This inflation talk can't be good for them even if the Fed tries to tamp down interest rates.
Good luck all!
Except maybe for a milestone payment (which may be all PGNX is angling for), subcu Relistor for chronic pain isn't going to sell very much with an oral product on the market. SLXP/PGNX needs to get the oral product out. I wouldn't be surprised to see Movantik used off-label in palliative care patients if it gets approved.
I mangled a few words when describing the instances of deaths in the Movantik studies. Properly stated, there were six CV deaths among study participants. All had taken Movantik at some point, there were no CV deaths among placebo/usual care only participants. Five of the six were high CV risk patients. There appears to be a trend, that Movantik adds to the risk of death in susceptible patients. Although the Adcom panel didn't find an overt signal, they didn't ring the all-clear bell either. Even if the CRL is reversed for subcu Relistor, it's hard to see how oral Relistor for chronic pain patients gets approved without a controlled, long term safety study. Do you think SLXP would see approval for oral Relistor for palliative care patients only? That might be possible.
dr. vin and ca_fisherman,
We've learned to never disagree with klaus. He's like the crazy uncle who lives in the upstairs back bedroom. He never lets facts get in the way of a good post. The best course of action is to nod your head and move on.
That said, there is general consensus on the NKTR board that oral Relistor for chronic pain won't be approved without additional safety data. Existing data for Relistor essentially falls into three categories.
1. SC Relistor field use for palliative care, aka body of evidence. By definition, this is uncontrolled short term use, and intermittent. There's no way you can evaluate Relistor as a contributing factor in cause of death among these patients.
2. SC Relistor long term safety study, about one year of as needed use and uncontrolled.
3. Oral Relistor short term efficacy study, four weeks daily followed by eight weeks as needed, controlled.
Oral Relistor is intended for use over a long, long period. Perhaps daily for many years, maybe even life. It goes without saying that the PK of the oral version is much different from the subcu. I'm not sure you can project the safety of long term use of the oral version from the existing safety data. NCT01186770 was a one year controlled safety study that morphed into the twelve week efficacy study.
All six subjects who died during the Movantik studies had taken the drug at some time, and five had high CV risk. I believe there is a very real risk that the FDA will require a black box warning against use in high risk CV patients pending further study. What do we know about the daily use of Relistor over a one year period?
PGNX completed a successful Phase III efficacy trial on the oral formulation. SLXP is trying to slide by with uncontrolled safety data for subcu Relistor for non-terminal patients on the basis that it is both difficult and unethical to subject people to a year of placebo injections. Now SLXP is trying to use this uncontrolled data to suffice for approval for both the subcu AND oral formulations. Good luck with that. Maybe I'm the only one who believes this, but I would be shocked if the FDA approved oral Relistor without a controlled one year safety study, perhaps enriched with high risk CV patients.
NKTR is the only company that really benefits from the decision over the next couple of years. That comes from the fat upcoming milestone payments that reduce the cash flow/dilution risk from the high cost R&D. I don't think that Movantik sales are going to ramp quickly. What money AZN collects will be funneled back into the post approval studies. SLXP/PGNX and CBST are at least two years away from an approved oral product. In fact, I think this is a net negative for SLXP/PGNX, especially if subcu Relistor doesn't get approved for chronic pain.
OT: Check out what BLUE reported today. If they can even come close to replicating the results for sickle cell anemia, that will be a multi-billion dollar company.
I didn't buy back, still don't own any. I absolutely agree with you, unless an unlikely CRL for more safety data in high risk patients materializes, Movantik will be the first oral drug to market with at least a two year head start. SLXP/PGNX is hosed. Even if subcu Relistor is approved, who is going to use it if an oral drug with comparable performance is available?
Finally free today and able to see what happened. With regards to your comments, hijacked, we did note that all six CV deaths reported from the naloxegol clinical trials in the FDA briefing material. All six involved patients who took naloxegol at some point. No participants who were in the placebo/usual care only group had a CV death. Five out of the six patients who died had a CV history when they entered the study. I haven't had time to review all the data, did AZN/NKTR document the percentage of study participants who entered at high CV risk?
I don't think we can conclude the results are all sweetness and light. There are a range of possible outcomes here. There could be a CRL for AZN to collect more data on high risk patients prior to approval, but that doesn't seem likely. A post approval study is highly likely. One thing that could gum up the works is a black box warning against use in patients at CV risk until the risk is resolved in a post approval study. That wouldn't affect the milestone payments but would certainly put a dent in future sales.