Register for free to join our community of investors and share your ideas. You will also get access to streaming quotes, interactive charts, trades, portfolio, live options flow and more tools.
Register for free to join our community of investors and share your ideas. You will also get access to streaming quotes, interactive charts, trades, portfolio, live options flow and more tools.
CLDN:
Share price obviously depends on your view of the likely outcome. Perhaps the only reasonable point I can make is that some caution is warranted if the outcome is "positive." The joint frailty model is not the same as an all cause mortality endpoint, so I think risk exists even with a positive outcome. In Cupid-1, the joint frailty model gave statistical significance for the high dose group, but after 3 years of follow-up, mortality did not. The FDA may require additional data.
Regarding AAV persistence and the next step: I doubt they'd return to animal models. The company does have a higher dose trial ongoing at 2.5e13 viral genomes, which suggests that they have their own internal curiosities about dosing and benefit. Only they can answer whether that curiosity is based on a nagging concern or confidence in seeing an increased benefit. My bet would be on the former.
As for Cupid-1's high dose arm being a false positive, I can't make a meaningful quantitative argument. It's more a gut feeling based on the preclinical data, and the fact that I've sat through many presentations describing trials with large effects in tiny CV cohorts. Acquired heart failure is a relatively heterogenous population, and many of the classifications are still based on symptomatic findings. For that reason, I would be cautious of depending on the outcome of a 23 patient study.
BMY / QURE:
QURE / BMY:
I suspect they're going to go after congenital cardiomyopathies rather than acquired.
In that regard, I doubt Uniqure's S100 program is going to be an area of focus for BMY.
SGYP:
I own it and got into it a while ago thanks to JQ. Admittedly I don't have too large an exposure here.
My expectations for this are as a modest buyout candidate. Safety appears to be decent, and I think we'll get a clear view of efficacy soon enough.
RTRX:
Re: RTRX
RTRX:
I think there's also a "discretionary bonus" to Shkreli of $575000 that he then turned around and used to pay off his own obligation.
This sounds like nothing but theft from the company, which would make him a criminal.
RTRX:
Am I understanding this 8K correctly? Did Shkreli basically use the company to pay off his own obligations?
Re: BLPH
LOL... you'd be right
Many patients in these groups become refractory to NO and NO-releasing agents because the endothelium and underlying smooth no longer respond at the molecular level.
And I'm quite sure that the reason for that lack of response is not because the NO isn't being delivered in a "pulsatile" fashion.
Re: TKAI
Apologies for the late reply, but I had not looked into this one before.
With Galeterone I get suspicious when a company is dosing one of their drugs at 2.5 g per day. That suggests to me that it is rather promiscuous.
But both of Tokai's drugs are primarily CYP17 targeted, which overlaps them with abiraterone. I think abiraterone is inferior to enzalutamide (which is primarily AR targeted), so that aspect of their drugs doesn't excite me. With galeterone they claim CYP17 and AR effects, but that seems dubious to me that their drug magically targets two different enzymes (this is the one dosed at 2.5 g/day). Also, the claims for truncated AR are correlative only... the patients that test positive for this variant also have the full length variant. Therefore you can't separate where galeterone is acting.
I actually think ARN-509 is interesting, but it's not as exciting to play with Lilly involved.
MDVN:
For those interested, US sales of Xtandi for the quarter came in at 230 million. In my opinion, this is a really solid drug with increasing revenues. There is a strong development roadmap for enzalutamide, so the only glaring weakness in MDVN is the lack of a pipeline.
The relevant 8K language:
RE: FLKS IPO
Interesting wrinkle in this company is that MacKinnon is a seriously excellent, top notch scientist. In fact, it has surprised me that he's not been more involved in start-ups because his work really looks at the type of data that pharma companies rely on.
But alternatively, this might reflect the bubble that many academic scientists live in. By that, I mean their strong dedication and acumen in one field (basic research) makes one surprised at their seeming naivete when entering another (drug development).
CAR-T / Safety:
I would be mindful of CNS adverse events.
Cardio3:
Very exciting. Can't wait to tune in. But I suspect I'll have very little nice things to say. I like the fact that they're already pivoting away from their cardiovascular work. Never too soon to bring in the shiny new object for investors to look at.
Also, when it comes to tag lines, I think they're definitely in the top 10:
"We care. We cure."
Re: Hydrophobic "coating"
Saw that earlier today and also thought of that application. However, I wonder if going from normal vessel surface to that highly hydrophobic surface would cause turbulent blood flow and promote coagulation?
The demos are mesmerizing though.
Just out of curiosity, do you know if anyone keeps a rolling graph of one of the healthcare index / ETFs versus funds raised in the sector? I know that the latest bolus has been tracked, but I'm curious if historically it has panned out as a way of predicting peaks in the sector.
OT: Deflategate
From my calculations, assuming mol of air is constant and volume is essentially constant, a ball inflated to 12 PSI at 72°F, or 22°C, would deflate to 11.1 PSI at 32°F, or 0°C.
Deflategate is a 2 PSI difference? If so, the balls would have had to be inflated at significantly higher than room temperature for the Ideal Gas Law to explain it (provided I've made no calculation mistake).
Re: MDVN
Re: CNAT
Re: CNAT
Re: MDVN
Re: Pan / Isoform specific inhibition
Re: CNAT
Re: CNAT
I have to admit I don't really understand the rationale for CNAT's drug. It's usually a lack of proper apoptotic signaling that causes problems physiologically, and this drug seems to want to exacerbate that.
In fact, there are lymphoproliferative disorders that are defined by caspase mutations that lower their activity. So I'm not sure where the company is going with this.
Re: Fluff PR
I was actually looking at Cardio3 this weekend to see if there was a way to time / trade the stock on the downside.
Their PR is fantastically fluffy. They put out a press release every time their ongoing phase 3 trial in Europe opened a center in one of the EU countries. Suffice to say that's a lot of PRs announcing trial site openings.
Almost better, they put out a PR noting that their work was mentioned in a review published in Nature Reviews Cardiology. At least one of the authors of that review worked at Cardio3 for some type, 2 of the authors have, at least in the past, had financial interests associated with the company, and another of the authors is now on the board of directors.
Fluff... and maybe even unethical fluff.
---
For those interested, Cardio3 has a futility analysis for one of their phase 3 trials scheduled for March, 2015. I'm fairly certain this trial is a giant bust, but uncertain if there is enough leeway in the futility analysis to allow the company to simply proceed with a failing trial.
FMI / PPHM:
PPHM / FMI
Re: Immunogenicity
FGEN:
Re: FGEN:
It could also be a bystander effect because BMY continues to bet on fibrosis:
http://www.fiercebiotech.com/story/bristol-myers-collaborate-calibr-fibrosis-drugs/2015-01-05
TRIL:
Re: TRVN
Re: TRIL
CAPR:
I'm not interested in any of the companies that are injecting magical cells into the bloodstream that magically hone to the infarcted / fibrotic area of the heart to magically clear the infarcted / fibrotic cells and magically regenerate new cardiomyocytes in their place. That seems to be their lead program, so not super exciting for me.
Also, I went to grad school with their CEO. I can't say that I was ever (*ahem*) inspired.
CLDN:
TRIL:
SCTPF / TRIL:
I still think there are questions about potential immunogenicity of the engineered protein. Plenty of caveats here.