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To me, as a trained virologist who is currently working in virology and spent most of 2020 and 2021 on COVID work, the idea that COVID-19 didn't come from a lab beggars belief. This is independent of political leanings as mine are definitely not those of politicians promoting the lab leak theory.
It's really quite simple statistics -- the chance of a new and highly virulent virus emerging independently *in the same city* that houses a lab studying coronaviruses is almost impossibly low.
I am not accusing the Chinese of engineering the virus or deliberately releasing it. Someone just got sloppy in a lab with a sample from the wild, infected themselves, and brought the infection home with them.
I worked in a lab at University of Chicago that was set up for BSL3 containment, although it was not being used for that purpose at the time I was there. It is *very* easy to see how a lab leak can occur without extreme diligence.
I believe there has already been an instance of someone using AI to refine neurotoxins.
https://www.theverge.com/2022/3/17/22983197/ai-new-possible-chemical-weapons-generative-models-vx
Aside from its applications in developing drugs and poisons: I am all for major advances in science, but I truly believe that we need to step on AI right now. It would be nice if AI would deliver on the science fiction promise of a utopia for all, but what really is going to happen is that a very large number of middle- and upper-middle class knowledge workers are going to be put out of their jobs, and the profits from AI are going to be tucked into a very small number of pocket books. We're going to end up with a crisis that makes the industrial revolution look trivial.
I have to admit that I find it very hard to see the value proposition in gene therapy investment over the next 5 years. It requires a tremendous investment to get a gene therapy approved for a single rare disease, to the point where they can't charge even close to enough to justify development costs.
There is no doubt that there will be tremendous potential value in gene therapy in the future, but it will require a fundamental change in the way the FDA reviews and approves these treatments. Instead of requiring a full submission for each rare disease, the only way gene therapy for rare disease is going to be feasible is if the FDA moves toward a platform approval approach in which the FDA approves the gene delivery modality (AAV, lentiviral) and then has a much more streamlined and less stringent process for approving the transgene payload for individual diseases.
All of this will change if and when gene therapy can be utilized for more common diseases. Then it makes sense to do the full approval process.
In any case I don't see that happening soon. But all of these tiny companies trying to move gene therapies for a few hundred patients through the full approval process doesn't make a lot of sense from an investment standpoint. I'm not saying that these companies are necessarily a bad investment, just that you are going to be waiting a long, long time for value to be unlocked, and a lot of them are going to die of starvation before the landscape shifts.
If I were to invest in a gene therapy company, I would avoid the tiny companies and invest in those that have a large amount of capital and preferably not focused entirely on gene therapy.
Also I've completely forgotten who's who around here. I have met some of you in person but I can't link the user names to people any more!
ARAV
Well as much as I like to keep my portfolio conservative (outside of VKTX and TGTX) I decided to take a flyer on ARAV as a "biotech value."
Seems to me that they are very substantially undervalued with a late-phase unique biologic for use in ovarian, cc-renal, and pancreatic primarily in combination with chemo, TK inhibitors, or nivo. Phase 3 data "mid-2023" and BLA Q4 2023 if successful.
Current market cap is ~128M at $2.15/share -- looking at the data including the multiple fast track designation I would think that it would be worth multiples of this?
I am mostly here so you can talk me out of this
ARQT
Anyone here following this as I start to research it?
They seem to have a broad and deep portfolio of dermatology drugs. Roflumilast cream is their first product, approved in psoriasis and likely to be approved for atopic dermatitis and seborrheic dermatitis based on the Phase 3 trial results. So that provides them with revenue to expand their portfolio.
In addition they have 2 *topical* JAK inhibitors for alopecia areata, chronic hand eczema, and vitiligo and they just acquired a biologic but it is preclinical so who knows?
Or is the topical derm space so full of generics that they are never going to make any money?
They have something close to half a billion in the bank so plenty of resources moving forward. So about half their market cap ($1.02B) in cash.
The FDA proposed those endpoints and confirmed that they were likely to predict clinical benefit to support accelerated approval.
There are very few instances where the FDA has specified appropriate endpoints, the endpoints were met in clinical trials without major safety concerns, and the FDA failed to approve.
While your idea isn't completely outside the bounds of reality, it's a bit of a reach.
VKTX FYI
I have no idea who has a little VKTX but the conference call was interesting last night.
Phase 2B results for the TRbeta agonist still anticipated for 1H2023
Results for the GLP-1/GIP agonist coming before end of Q12023. I was mostly thinking about this as a sideshow but analysts are weirdly excited about it?
Results for the X-ALD program due 2H2023
$155 million in the bank so no dilution in the foreseeable future (meaning at least 2 years)
They are actively seeking partners for the NASH program, which of course is an enormous market.
Editorial comment: I think this may have the best risk:reward in the biotech universe right now because their molecule is similar to that of MDGL's and is thus de-risked to the extent that a novel molecular entity can be. The company has a market cap that is roughly 1/3rd that of AKRO, which has a demonstrably inferior drug and no pipeline to speak of. And I guess based on all the excitement last night about the GLP-1/GIP agonist there is significant value there too despite it being early? I don't know why they were so interested in it.
*Edited, I accidentally said that the results for the dual agonist will be from a 2B trial -- that's the TRbeta agonist (sorry)
It is definitely getting frothy out there.
Roth Capital Markets on Tuesday maintained a Buy rating on Marker Therapeutics Inc (NASDAQ:MRKR) stock, noting that the company's multi-antigen targeted T-cell therapy in solid cancer tumors “just might work.”
Analyst Tony Butler also set a $10 price target on the stock, which currently trades at around $5.75 a share.
Marker Therapeutics on July 20 released some positive results from a Phase I trial of its MultiTAA T-cell therapy in patients with pancreatic cancer conducted at the Baylor College of Medicine. The company discussed the data during a conference call with investors Tuesday.
The company said the T-Cell therapy, in combination with chemotherapy, showed some promise in shrinking or stabilizing tumors.
Under Marker Therapeutics’ therapy, a population of T-cells attacks multiple cancer targets and works to activate a patient's immune system to trigger anti-tumor activity.
“Based on the encouraging data, we believe this could provide an upside to our current price target,” Butler wrote.
Looking ahead, Butler also said there’s a 70% chance the company’s therapy will win government approval followed by commercialization during 2024.
“After the launch we assume relatively rapid uptake with competitive peak penetrations the US (30%), Europe (20%), and China (10%),” he added.
MRKR
Just listened to the Webex they had this morning on the pancreatic data. Surprised by the 30% drop in response to what I consider wildly positive (albeit limited) data in pancreatic cancer. Even if the data were negative, which they are not, pancreatic is just an early-stage side show.
Talk about inefficient markets!
Also Lumpy if you read this--I won't post on the other board because one of the moderators is horribly misinformed about even the basics of biotech and keeps deleting my posts and others when they contradict his misinformation.
I've been working and investing in biotech for the last 20 years and this is literally the worst handling I've ever seen of a study, period.
Just f-up after f-up; setting expectations too high, the ASCO submission debacle, moving the teleconference around, and now they let AACR set the tone with a surprise article consisting of a mediocre interview with junior faculty with no media training whatsoever.
Someone needs to be fired, first and foremost their amateur hour PR agency, which was presumably responsible for their disaster of a website, and then maybe some heads need to roll in management.
These data are excellent, none of this needed to happen.
Yeah the level of jackassery around this trial has been absolutely astounding.
First they submit to the wrong track at ASCO.
Then they mess up the teleconference timing.
Then AACR undercuts them by PRing unexpectedly.
But the data are really good.
Somebody really messed up at Healio by breaking the embargo.
Key points: