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As far as political beliefs, the problem started when the pandemic was just starting and little was know about how to best mitigate the spread, and how best to treat it. Instead of focusing on those issues, the previous administration and a number of other prominent Congressional members immediately started demonizing the Chinese, and that led to an increase in hate crimes against Asians.
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.
Then how did PFE’s Pavlovid manage to do $22B in 2022 sales??
Have you gotten any feedback from people in the biotech industry on the ideas for gene-therapy programs that you posted? TIA
COVID antiviral, Pomotrelvir
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.
"Gold Rush For “Gene Therapy”
After the FDA lowered the entry barrier for mRNA vaccines (thanks to Covid), biotech companies saw an opportunity in gene therapy. Now, they’re doubling down on it.
• For biotech companies, the logic is simple. Faster FDA approvals mean a shorter time to market for a new drug or therapy. This dramatically reduces the time frame and returns on investment for biotech companies and their investors.
• Historically, clinical trials take years when the therapy generates no revenue. But with mRNA vaccines, some pharmaceutical companies went from testing to Emergency Use Authorization (EUA) and revenue generation in a few months.
• There were 2,900 clinical trials for gene therapies from 1989 to 2018. Last year, it was about 2,500, reports the Center for Biologics Evaluation and Research.
• As such, the biotech industry has recently seen record venture capital and private equity investment, accelerating growth for some companies.
• See five biotech companies on Barchart’s radar this month."
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!
I suspect you are in the minority here then. This board is 90% dedicated to small bios as you very well know so most folks here (myself included) are degenerate gamblers to some degree!
PS: I average 40% cash in my accounts dedicated to bios so I guess that puts me in the "conservative" camp too. Right now half my money is also in treasuries but that is as much a function of being in my mid 50s as anything I suppose
PS: I don't follow ARAV so no comment but good luck w it
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
I wish I had the gumption to go short these companies - the hit rate is so much higher than trying to pick the winners
I would say that it will be hard for ARQT to make serious money in topical derm. I can’t think of a topical derm product that became more than a modest seller.
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.
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?
One of the few PFE calls I listened to last year was dominated by analysts asking about their oral GLP-1 agonists (interestingly they had 2 they were progressing at the time one with more favorable pK amd the other slightly more advamced IIRC)
I think it’s just the sheer size of the commercial opportunity. Analysts are talking 15-25B in annual sales for LLY’s dual agonist and it can’t even be dosed orally
If the FDA ends up rejecting MDGL’s NASH NDA it’s unclear what effect that would have on VKTX, but it’s not crazy to be concerned that VKTX might incur a steep sell-off.
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)
TGTX
Great FDA Approval timing in this season of giving! I guess these guys with the lawsuits have a bit of a problem now.
It is definitely getting frothy out there.
Briumvi’s claim to fame is its dosing schedule: a 1-hour every 24 weeks, after the loading-dose regimen of a 4-hour infusion and a 1-hour infusion two weeks later.
toxic autism causing Vaccines
there may also be one more piece of hidden reality. Any breakthrough technology happening in the Big Pharma world,that stands to disrupt the big money system, is something BP wont be happy to see . as long as it threatens BP profits... they have reason to fight against it, and wall street shenanigans is one way they do it. short attack the company. drag the approval process, the FDA is their gatekeeper. etc. People label this concept as a conspiracy theory. its just common sense. BP will allow a medical gadget to succeed but a cancer cure is not allowed.
I agree its very possible to see a suppressed price in a Range from 4 dollars to 5 or 6 for a while. But also possible the wall street short attackers will try to be relentless and take it down to 3 and 2 dollars.
The science is good. and thats why it threatens Big Pharma money system. Many small innovative biotech companies have suffered the same misery. Its the corrupt big medical money system. It wont change until Trump administration intervenes and forces it to stop. Its a big swamp that needs to be transformed.
I think its idealism investing and I get sucked into it myself. All my biotech investments have done this giant surge and giant collapse. AVXL,GALT,Biotime, remember that one? CTIX,IPIX,now MRKR. I cant think of any that were independent from Big Pharma that made it to the top unscathed.
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.
So PL and rfj now that you have seen the PR what are your thoughts?
We'll agree to disagree but I'm 100% confident this is what went down. You are obviously entitled to your opinion. You are correct though, the company was taken by surprise. AACR went behind their backs and released the data. Did the company know they were going to do that. Absolutely not.
For everyone saying that the company mismanaged this, AgAiN, it wasn't on the company. It was on AACR.
I am disappointed with how the company handles this very promising early result with such an inexpereinced junior faculty presenting of it.
He could say something like "Immunotherapy often takes time to achieve the maximum results than chemotherapy. Our short term (~6 months) results are very promising. Given the lack of toxicity and safety with this immunotherapy regimen, we certainly hope we will have an equavalent or better outcome when the treatment continues."
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.
-13% premarket, FWIW
Somebody really messed up at Healio by breaking the embargo.
Key points:
More than half of patients with pancreatic adenocarcinoma showed clinical responses when treated with an investigational multiantigen-specific T-cell therapy, according to the results of an ongoing phase 1 clinical trial presented at the ACCR’s Immune Cell Therapies for Cancer conference in San Francisco.
The study included three treatment arms in which researchers evaluated the safety and feasibility of multiTAA T cells as well as progression-free and overall survival. Arm A included patients with unresectable or metastatic pancreatic adenocarcinoma whose disease responded to standard first-line chemotherapy. Arm B comprised patients with progressive or refractory disease after first-line chemotherapy. Finally, Arm C included patients with potentially resectable disease.
Treatment Arm A included one patient with a complete response, two with disease progression, five with stable disease or ongoing response (range, 6-9 months) to therapy, and two who are too early to evaluate.
Arm B included patients with progressive disease. The study showed that three patients continued disease progression, while three other patients showed ongoing stable disease (range, 1-6 months).
Arm C included patients with potentially resectable disease; these patients received one preoperative infusion of T cells and are still receiving postoperative infusions and adjuvant therapy.
I don't think this has been much of a drop.
Highly unlikely to be able to file on just the data being presented from a single arm single site, even if there are some responses (especially if those responses are in the chemo arm)
correct overall response rate branches out to mean:
1.) Complete response
2.) partial response
3.) stable disease
All those above translate into the overall response rate for a study.
If you believe that the pancreatic cancer data is a make it or break it event then, sure, they should raise cash before they present data if they don't expect a good reaction from the market. My reasoning for not using the shelf as an indicator of success is that even if data is worse than expected and we take a hit to the SP we will have plenty of time between now and when cash is actually needed to raise some.
The shelf was just standard operating procedure. It should not be used to come to a conclusion about the upcoming data. Either way there or plenty of other hints that are a lot more convincing as to what the data will look like.
Agree with this, but would add that this data will be fairly preliminary, correct? I think they finished recruiting earlier this year right? MRKR website says the study is active but not recruiting, but clinicaltrials.gov says it's still recruiting