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”.
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.