915 days left


REGN analysis is going smoothly. Should have the final outcome for you next week.

Can you really have a drug pricing hearing without me? That’s fine. I’ll be out in 2 years and we can do it then. Sanctimonious ignorance won’t be eliminated in politics anytime soon.

In a bombshell paper in The Lancet, researchers found that Novartis Access, which donates key drugs to poor countries such as Kenya for $1 per month, did NOT help access to medicine. Systems are complex, they say. They are in the U.S., too.

Papers I’ve Read

MeCP2 isoform e1 mutant mice recapitulate motor and metabolic phenotypes of Rett syndrome. Ciernia et al. Hum Mol Genet 2019.
Rett Syndrome is very promising to drug developers. It’s a somewhat large monogenic illness. Knowing exactly what is ‘going wrong’ in a disease is the first step to designing a treatment. Unfortunately all that is really known about Rett is there is a MeCP2 dosage issue. Nobody knows what MeCP2 does, and the fact that most patients are female creates an interesting gene dosage/X-inactivation question (MeCP2 is on the X chromosome). At four exons, two isoforms of MeCP2 are spliced. MeCP2-exon2 proteins don’t seem to cause disease while MeCP2-exon1 proteins do. This group created the former and spent a lot of time on metabolic profiling, which is interesting but they don’t quite recapitulate the phenotype of the human illness in their mice. Progress will probably be slow in this illness but keep in mind AveXis (now a part of Novartis) has started a phase 1 for their gene therapy.

Clinical Trials I’ve Noticed

Study With Lu AF20513 in Patients with Mild Alzheimer’s Disease (AD) or Mild Cognitive Impairment (MCI) Due to AD. Lundbeck A/S.
Lundbeck has had this weird Abeta vaccine in Phase 1 for 5 years or so. I don’t think it will work, but one never knows. It’s certainly an interesting approach–not one that hasn’t been tried though. I believe Elan had some other active immunization program that blew up (this might be why the trial has taken so long).

10 thoughts on “2/23/2019”

  1. Hello Martin,
    I hope everything goes well for you.
    Just want to thank you for your insight on ONCE and the whole gene therapy for hemophilia scene that is paying off.
    Which field do you think hold great potential breakthroughs right now?


    1. There is lots and lots of youtube material available. Hours and hours of footage. Go to investing episodes by Martin Shkreli. Not much on gene therapy. He has got broad approach.

  2. Thanks for bringing ONCE into my/our attention! Your insight in the YouTube investment series helped understand your work flow and diligence. When every other “investment guide” charges $$$ for very little in value, this was a breath of fresh air. Real legend!

  3. Hi Martin, what do you think of SNY/LXRX’s work with Sotagliflozin? PDUFA date is March 22, 2019 and the Advisory Committee Meeting voted 8-8 that benefits outweigh risks on January 17, 2019. From what I’ve heard the FDA generally follows the Advisory Committee’s recommendation.

  4. Congratulations to your guys at Alexion. Solaris sales were astounding this quarter. Great call on this company. You got well functioning brain and you are quiet intelligent and disciplined. You are the caliber type of people with the great thinkers. Too bad people like are either kept low key and in hiding or are imprisoned. I am sure there has got to be another great biotech / science / math business investor savy individual. But I really think they are hush hush and the people around them don’t let a peep out. That’s kind of the same method used to keep children or victims imprisoned in their own homes. It’s sometimes a generational thing. But I am glad your parents taught you well about independence and self reliance. All the best.

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