The next five or ten years of biopharma investing is basically all about gene therapy. After AVXS, who knows what is possible. Abeona (ABEO), Axovant (AXON), Audentes (BOLD), BioMarin (BMRN), Oxford, PTC (PTCT), Spark (ONCE), UniQure (QURE), Rocket (RCKT), Sangamo (SGMO), RGNX, Sarepta (SRPT), Solid (SLDB), Ultragenyx (RARE), Voyager (VYGR) are all players. My guess is an equal-weighted or capitalization-weighted index of the above would outperform pretty much any other index.

You have a number of big pharmaceutical companies that have no idea what a vector is: Johnson & Johnson, Merck, Amgen, Eli Lilly. Most of those companies are also scared of orphan drugs. Pretty much the only company “all-in” is Novartis, who I often consider to be the smartest large drug company (not saying much), with Pfizer in second and GSK dabbling a tiny bit. Even the big orphan companies are gene therapy-lite: Alexion doesn’t have anything, Shire has very little and Sanofi has very little. BioMarin, of course, has the next huge AAV for hemophilia (for now).

Amazing to me that Sandoz is withdrawing from Copaxone market. Years ago this was supposed to be the most lucrative hard-to-make generic, and now they can’t make a profit selling it. Generics is a nasty business sometimes!

An HIV vaccine is within reach. What level of protection would be necessary for a commercially viable (ACIP recommended) product? 50%?

Paper’s I’ve Read
Zen and the Art of Making a Bayesian Espresso. Zhang et al. Neuron 98;1066-1068.
With a title like this, I could not help myself. The actual paper by Konovalov and Krajbich is a simple sequence learning neuroscience study. I still haven’t read Pirsig’s classic “Zen and the Art of Motorcycle Maintenance”.

Clinical Trials
Efficacy and Safety Study of BIIB074 in Participants with Trigeminal Neuralgia. BIIB074 vs. Placebo. Biogen.
This is a small molecule acquired by Biogen a few years ago, now in Phase 3. It’s a Nav 1.8 (pretty sure). Vertex is moving their Nav 1.7, where the data look spectacular. So, we’re a few years away from powerful non-opioid pain relief. I think the Vertex drug is far better than this one, but not too sure.

Safety, Tolerability and Pharmacokinetics of Single Ascending Doses of REL-1017 (d-Methadone). Relmada.
Surprised RLMD is still around. Probably on its last breaths.

I’m reading the new Tesla biography. It is engaging but brief,
I’m also reading a book about the Russian composer Prokohiev as I prepare writing my own microtonal symphonies.

There’s a WSJ article entitled “Hanging with the Hedge Fund Bros to get the Story”. I haven’t read it. “Bro”, will never, ever be an insulting moniker to me. Being male is not something to feel guilty about, despite the protestations of some media. When I was labeled “Pharma Bro”, I had no idea that it was a derivative of the pejorative “Tech Bro”. Apparently, there are “Writer Bro”s as well. As most of you know, I’m sick of establishment identity politics. With Vox criticizing Animal Crackers packaging for implicit bias, why does anyone give a voice to the easily offended? Even the Germans, who must have a deeply shaped and apologetic national identity, for you know, trying to take over and exterminate the world, don’t face the same shaming American males seem to have to endure.

I’m teaching matrices to some of my friends here. Their excitement at this new way of looking at numbers is thoroughly satisfying.

dentate gyrus – part of the hippocampus. The DG receives input from the entorhinal cortex, related to new stimuli. The DG is thought to be instrumental in new memories and spatial processing. There is a lot to unpack here–folks have spent their whole lives studying this tiny structure.

11 thoughts on “8/30/18”

  1. Pharma Bro was simply a fun thing to call you. Has nothing to do with white males or anything like that. What self victimizing bull crap this?

    You’ve been listening to much to alright fan boys and Trumpies thinking white men are under some kind of attack. People outside the media don’t even care about this shit. There are some extremist that run around and right articles

    People need to step outside and stop hiding in these internet bubbles where it skews reality of what people say it do. I live in an area where literally no one talks about race, sex, or hitching about white men. You literally have to be looking for these crazy social just warrior types on college campus or internet.

    Republicans and white men now ironically are offended by everything now too it seems like. Everyone of your post is some kind of self victimizing bull crap. Oppression contest are damn hilarious. It’s like how Milo Yiannopolis started crying victim after his whole point of doing those college speaking tours was to call out self victimizing. Accept some God damn responsibility for something, and maybe you wouldn’t be in prison. Harsh right? If you can dish it out, you can take it!

    You really make it hard to defend you anymore.

    1. Martin fucking created a medication that saves dying children from a slow death via paralysis because they have one simple error in their genetic code. He started a company while he was dirt poor and made over a billion because he’s a fucking genius and knows how to save people’s lives. The only thing he could even be accused of is stealing money from the company that HE FUCKING STARTED, because “he tweets too much,” and god knows if thats even true. Martin is a pure genius and no one else even understands why he’s in trouble in the first place. Daraprim is a fucking amazing drug, tell me what price you’re going to market a medication for only 300 patients? Do you have any patents, have you ever filed for a US patent? Do you know anything about the pharmaceutical industry? You don’t. Martin deserves to raise the price on his drugs even higher, he’s one of the few Pharma CEO’s who actually knows science proficiently, and the lives of many dying patients would do better in his hands. He is extremely intelligent and polite, what have you done for the world? Nothing..

      1. lol Daraprim is a horrible drug, the whole point of making it expensive was to research an alternative and in the meantime, hopefully discourage doctors from prescribing it.

  2. Wow Jane, chill.
    Thanks Martin! I wish you could expound more on AXON. Losing my ass on it. I drank the “Vivek is a genius kool-aid”.

      1. Not offended just thought Martin would be too smart to not eat up Trumpian/white nationalist propaganda. After Trump won, all I see is white guys coming out of the woodwork acting like oppressed victims. Is it not enough for their guy to win the presidency? These guys all love to be victims because they can’t control the narrative anymore.

        people. I like the guy, but I don’t worship his ass and think of the shit he does helps him. He always comes off exactly how his critics think he is.

  3. How are you Martin? Hope all is well. Thank you for all your contributions. Best of luck. I hope you will get to enjoy freedom in the near future.

  4. Loving the blog Martin! I’ve noticed you have some decent thoughts about Novartis… what are your thoughts about Sandoz buying the rights to the generic epinephrine from Adamis? Much love can’t wait till you’re a free man!!

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