More thoughts on gene therapy taking over the world of medicine. This will take years and execution is still key–having any old vector won’t do the trick. Nevertheless, SRPT, QURE, PTCT, BOLD, ABEO, SGMO, RCKT, FOLD, FCSC, AXON, etc. are all intriguing investments.

Thanks again to all of my friends & family who navigate my social media tirelessly for me (including the occasional troll!). James, Dan, Taylor, Mark, Dad and those who won’t be named–you make all of this so much easier! It is great to receive emails, posts, tweets, etc. by jail-email. Anyway, continuing a LinkedIn conversation–it is not a unique idea that online news companies are NOT a good business model. Apparently we touched a nerve. Just look at TST! It is pretty much the consensus that these types of companies lose money, forever. Building a brand like WSJ or NYT takes a very long time and a ton of money. I’m not even sure it is possible anymore. Even by diving into the sludge with salacious and often erroneous or fraudulent content doesn’t guarantee a product. High-integrity journalism (nowhere to be seen) definitely reduces margins. And remember, this is all during a peak-margin period. In a recession, it’s over. Perhaps I’ll be a buyer of last resort. But again, it is not news that news doesn’t sell.

Clinical Trials I Noticed

Study of Efficacy and Safety of Investigational Treatments in Patients with Moderate to Severe Hidradenitis Suppurativa. Novartis.
This trial studies Novartis’ CD40 mab CFZ533, which I’ve noted is in a few other indications, and a fairly unknown and mysterious candidate LYS006. HS is one of the lesser-known TNF-constellation autoimmune diseases. Once you’ve done RA, psoriasis, PsA, Crohn’s, UC, pediatric forms of the aforementioned and AS, uveitis, Bechets or HS is next on the list for your pan-autoimmune medicine.

KPL-301 for Subjects with Giant Cell Arteritis. mavrilimumab. Kiniksa Pharmaceuticals.
This is a really interesting company that is probably a good long investment. KNSA is the stock ticker, about $1 billion market cap. KPL-301 is AZ’s mavrilimumab, a GM-CSF antibody. I played with one of these types of drugs at KBIO. That might have been a G-CSF antibody? Pretty similar. Either way, an interesting autoimmune angle. The old RA data for this drug was pretty good. I’m not sure why it wasn’t moved forward, but repurposing for even narrower indication is akin to what I did with sparsentan.

A Drug-drug interaction study between PF-06650833 and PF-06651600 follow multiple doses in healthy participants. Pfizer.
DDI combining Pfizer’s IRAK4 and JAK3 inhibitors. Don’t knock out the whole immune system while you’re at it!

A Study to Evaluate the Efficacy and Safety of 2 Dose Regimens of Intravenous TAK-954 for the Prophylaxis and Treatment of Postoperative Gastrointestinal Dysfunction in Participants Undergoing Large- and Small-Bowel Resection. Takeda.
This was TD-8954, a 5-HT4 antagonist (I think antagonist! could be agonist?). Takeda seems set on conquering the GI autoimmune diseases and all affiliated conditions. Smart.

From Molecules to Cognition: Inhibitory Mechanisms in ASD and NF1. Lovastatin. University of Coimbra.
Sad to try lovastatin, a relatively inert first-generation statin on autism patients. Can’t do any harm other than false hope. The first small study looked good, the large study, of course, failed to replicate. So here’s try 3. A desperate community, to be sure.

Delgocitinib Cream for the Treatment of Moderate to Severe Atopic Dermatitis During 8 Weeks in Adults, Adolescents, and Children. LEO Pharma.
Topical JAK. Finally?

Ibrutinib in Steroid Refractory Autoimmune Hemolytic Anemia. Dr. Nikitin.
Could work!

A Study to Compare Bioavailability of Naloxone Nasal Spray, Naloxone HCl IV and IM in Healthy Volunteers. Insys.
Two studies from Insys on what is their putative Narcan competitor. I guess they can get you addicted on the left hand and rescue you with the right.

12 thoughts on “2/27/2019”

  1. I’m going to update my LI profile pic from my chubby law student (who knew how to eat) to regular pic of myself because this is how I look…. to find out if you are RACIST!!!!

  2. Hey Martin, I would love to hear your thoughts on spectrum pharmaceuticals (SPPI) and its Late stage drug Rolontis. P.S. Free da man

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