918 days left


Lots of interesting things happening!
ICPT might be more commercially viable than the DOA pronouncement given. We’ll see! Will not be simple, for sure. But I wouldn’t figure in zero revenue, either.

When I spoke on the RDEB complex of KRYS ABEO and FCSC, I wanted to make a few things clear. 1) I have NEVER recommended any of these stocks and do not know them well at all. After two hours of research my (very) preliminary conclusion is buying the entire complex will probably achieve a positive return. FCSC is a strange company with a very bad capital structure and a partner I don’t trust (XON). If they dump XON it would be a more valuable company. KRYS only has reported two patients. ABEO is also a company in flux with tons of projects. It is very hard to tell which company will do well, FCSC may not even survive, after all. I don’t have any positions but I’m watching eagerly as this is really rough disease and the global opportunity is in the billions (value, if not revenue).

ACET filed and shocked with a sale of the chemical business for quite the price.

Papers I’ve Read

Trastuzumab Emtansine for Residual Invasive HER2-Positive Breast Cancer. von Minckwitz et al. NEJM 2019.
Roche hits it out of the park with Kadcyla in this trial. It’s still a bit unclear if you’d rather take Perjeta, Kadcyla or neratinib at this point, but it almost all benefits Roche’s attempt to transition away from Herceptin in any event. HR=0.50 for invasive disease or death is a bit shocking in such (relatively) well-controlled patients.

Residual Disease after Neoadjuvant Therapy – Developing Drugs for High-Risk Early Breast Cancer. Prowell, Beaver & Pazdur. NEJM 2019.
This whiny editorial bemoans the lack of a pathway to shortcut large phase 3s in relatively indolent early-stage cancers. One cannot have it both ways. A robust surrogate requires robust validation, in which case, you’ve so substantially improved the disease from hard outcomes, one cannot possibly be upset about requiring such large trials. At that point, you’re treating a different illness, akin to primary prevention vs. secondary prevention in CVD. If you don’t have a robust surrogate endpoint, then why risk the accelerated approval for a disease that is admittedly indolent and within striking distance for a surrogate?
In HRPC, PSA might become a viable endpoint due to the same circumstances. I just view this narrative as solutionless and fruitless. Pazdur’s suggestion of enriching for sick patients also misses the point that trials should reflect intended population and you’d be making serious errors in generalizing to broad populations if you did as suggested.


When I don’t post for some time, it is because I am very busy. What have I been doing? Mostly reading back issues of PLoS One, Nature Communications, Hum Mol Genet and other favorites. If you don’t hear from me, you should actually be HAPPY, as I am occupied and entertained. Daily posts would be a bad sign!

Good luck to Bernie Sanders, my old buddy! YOU CAN STILL WIN!

24 thoughts on “2/20/2019”

  1. Btw some dude made a “whatever happened to Martin Shkreli” video yesterday and so far it has 115k views. Even the commoners miss you.

  2. Hope all is going well Martin, I’m a 24 year old disabled veteran. If you ever want to talk about anything hit me up freely.

  3. Good evening Martin,
    I’m Gregory a 24 year old military veteran and wish you nothing but the best. Things get better with time. Hit me up if you ever want to talk.

  4. There’s also quite a bit of comedic material he has provided. The above comment was a snippet from his comedy channel on YouTube – Pay-to-call (minus the emoji stuff).
    Some of my stuff 💜

  5. Hey Martin, was just wondering if you got my letter, I know you’re busy and can’t respond, but know I take all your analyses and words into account and am still very interested in learning from you.

  6. Martin,

    I have watched you since you began to live stream. Would often have you in the background while I would study to complete my degree. Maybe this message won’t mean to you as much as negative criticism, but I just wanted to briefly let you know that many people like me anticipate your return. Hopefully you decide to continue livestreaming aspects of your life. I found the way you were able manipulate gullible people to be naturally entertaining. If you can, please write more about the expectations you have of your life within the personal section of your blog.


  7. Ever look at Ziopharm ( $ZIOP ) … they booted RJ Kirk from their board,
    Eliminated the toxic $XON $160m Pref/debt,
    now advancing 3 platforms (TCR program at NCI, CD19 CARt, and IL-12 w Regeneron)
    Attractive Solid Tumor science and size of mkt is billions.
    RJ Kirk dumped his 10m shares, stock still heavily shorted but the game has changed

  8. Hey Martin, what do you think about Regeneron’s REGN4461 weight loss drug? It claims to be a LEPR antibody, working by increasing the amount of leptin receptors which would result in more leptin binding to these receptors. Regeneron thinks that when the hypothalamus sees this increased leptin binding, it will respond with a feeling of satiety. YDo you think it will end up like Regeneron’s other weight loss endeavors or do you think this could be a hit drug? You’ve been around the block before in terms of Regeneron and weight loss, does this seem like the drug that you famously shorted or are there key differences which could affect how efficient it is?

  9. What do you think of REGN5061? There isn’t much info on it, but regeneron says that it acts as a GFRα3 antibody and will be used as a pain medicine. From my limited understanding of biochemistry, GFRα3/artemin receptors have not been heavily researched, but they have some involvement in dopamine which is interesting. What is your opinion on this drug? Do you think it will work?

  10. Another player in the EB space is PRNB. Might be worth taking a look at them, but its a small molecule approach not GT. They had an IPO last year.

  11. Hey, Mr. Shkreli just letting you know I personally miss your streams and everyone else does. It doesn’t matter what you did, honestly, I think anyone logical enough would’ve done the same. Just two questions, one, after your prison stint are you still going to do tech? Two, what’s your opinion on Alexandria Ocasio-Cortez? Good luck man. Look after yourself.

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