Anixa Biosciences, Inc. (ANIX)

Overview from the website explaining what they do and why their approach is unique to everyone elses:


www.anixa.com/car-t/overview


Immunotherapy Background and Overview

For many decades, the primary approaches to cancer treatment were surgery, chemotherapy, and radiation therapy. Better targeted therapies such as monoclonal antibodies and other small molecule and biological drugs have been developed over the last 25 years. More recently the exciting field known as immunotherapy has enabled the development of drugs that modulate the immune system to battle cancer.

We know that our bodies, through the function of our immune systems, are able to “cure” cancer. It is now known that some number of cells in our bodies are becoming cancerous all the time. These transformations are driven by many factors including genetics and environment. Most of the time without us being consciously aware, these cells are destroyed and cleared by our immune system. As our immune function declines with age, some cells are able to survive and eventually become tumors, either liquid or solid. We also know that the tumors themselves have developed mechanisms to suppress immune cell function. Such is the case with pediatric tumors as well adult cancers.

Considering this understanding, the field of immunotherapy tries to boost the ability of our immune cells to battle the cancer. There are a number of methods that scientists have devised to induce our immune systems to battle cancer. One of those approaches is known a Chimeric Antigen Receptor T-cell technology, better known by the acronym, CAR-T.
CAR-T therapy is a treatment where a patient’s T-cells, a type of immune cell, are removed from the body. These cells are then modified in a laboratory through the advanced techniques of genetic engineering, and increased in number, and then infused back into the patient. In many ways, this is the ultimate personalized medicine. The specific genetic engineering is what makes the patient’s own T-cells even more powerful as a tumor fighter. This genetic engineering causes a special receptor to be present on the surface of the T-cell. This receptor is known as the CAR—the Chimeric Antigen Receptor.

While CAR-T treatment is complex, it has captured the attention of researchers and investors because it has produced remarkable responses in both children and adults suffering from specific types of liquid cancers, for whom other treatments had failed and for whom there were no other options.
While CAR-T has shown spectacular results with some liquid tumors, to date there has not been any unequivocal positive results with solid tumors in humans. Anixa believes that one of the primary reasons that CAR-T efficacy in solid tumors has been elusive is due to the lack of known antigens on the surface of solid tumor cells. To understand this key attribute, it is useful to review key characteristics of the liquid tumor successes.

The specific tumors for which CAR-T has demonstrated effectiveness in humans, are all diseases of B-Cells (a type of white blood cell). These are leukemias and lymphomas that result when B-cells become cancerous. The FDA approved CAR-T therapies for B-cell tumors that target or attack a highly specific protein on B-cells known as CD-19. It turns out that CD-19 is selective to B-cells and does not exist on other cells in the body. Therefore, when a CAR-T is designed to attack this protein, it will not target other cells or organs in the body. Unfortunately, the CAR-T therapy will kill healthy B-cells as well as the cancerous B-cells. Luckily, with proper care and supplementation, the patient can survive without any B-cells.

Science and our Program

Recently, our collaborators demonstrated that ovary cells which have a protein receptor on their surface called the Follicle Stimulating Hormone Receptor (FSHR) can be targeted with CAR-T technology. Studies also show that FSHR only exists on ovary cells in an adult female. In addition, there exists a hormone called Follicle Stimulating Hormone (FSH) that binds exquisitely to the FSHR. This hormone-receptor combination has evolved over millions of years to be highly selective.

The unique expression of FSHR on only one type of cell in an adult female is analogous to the CD-19 situation with B-cells. Similarly, women can survive without their ovaries. Therefore, we feel that CAR-T technology where the CAR is FSH and the antigen is FSHR, could be similar to the B-cell situation and our proprietary (patents filed) approach could be one of the first, if not the first, effective CAR-T therapy for a solid tumor.

Studies in vitro and in vivo (in animals) demonstrate excellent effectiveness of our CAR-T approach. (Clinical Cancer Research, 23(2) January 15, 2017, 441-453).
Even more interesting is the fact that FSHR appears to be expressed on the surface of blood vessels in tumors of other types such as prostate, colon, lung, pancreatic and others. This is an important point: While FSHR does not exist on cells in healthy adults except for ovaries in women and testis in men, it does appear on blood vessels that nourish other tumors. Therefore, should this therapy work for ovarian cancer patients, it may also work for multiple other cancers.

Markets

The markets for solid tumors is much larger than the markets for leukemias and lymphomas, especially B-cell cancers. Therefore, we feel that the scope of opportunities for our CAR- T therapy is very large should we demonstrate efficacy in solid tumors. Our first indication will be ovarian cancer, followed by other tumor types.

Here is a list of upcoming catalysts:

(from Tailwinds)

tailwindsresearch.com/2019/01/anixa-doesnt-disappoint/


2019…The Year of Good News

I don’t want to understate the risks involved in Anixa. The path to them pulling off the double whammy of making both products big successes is fraught with potential disasters. One should always be aware of the risks here.

The good news is that the path to success gets much harder in 2020. That is when sales of Cchek could disappoint, or the CAR-T therapy could fail in treatment. For the meantime, we are like a race horse being led into the starting gate; the final outcome is undetermined and tough to achieve, but I’m pretty sure we’ll be involved when the starting gun goes off.

Which is a way of saying that the catalysts for 2019 are significant, yet highly achievable in my opinion. Let’s break them down chronologically.

・Cchek partner. Sounds like it’s close to being a done deal. Will be announced in Q1.  (done)

・CAR-T IND filed with FDA. This is a Q2 event that in which Dr. Kumar has shown a high degree of confidence.

・Cchek CLIA approval. This requires a lab to do confirmatory testing on prostate studies. If the results are close to those shown in previous studies, which have been awesome and continually improving, the Lab Director should certainly approve the diagnostic. This is likely an end of Q2 event. (done)

・FDA approves the IND for CAR-T. Assuming the IND is filed with no material changes to the preliminary data shown by Anixa, there’s no reason to assume the FDA will not green light this drug ASAP. It’s been shown as efficacious and is looking to cure patients with a deadly disease and no other possible alternatives. This is also an end of Q2 event.

・Cchek is launched. Q3 event that follows up on CLIA approval.

・CAR-T patients are selected, trial begins. This is also a Q3 event that follows on FDA approval of the IND.

Thus, you have 6 major catalysts that are coming between now and the end of Q3. There are also many other smaller catalysts that are likely to occur along the way. All of these catalysts are significant value creation events. And, they all appear, in the grand scheme of biotech, to be relatively low risk events. In Tailwinds’ opinion, the path to getting both products through to their Q3 goals has bumps but no major hurdles. I think success is highly likely.

This unique combination of outsized potential reward combined with less risk, relative to the potential upside, is why I continue to buy shares in ANIX and exactly why I made it my stock of the year for 2019.

ANIX pushed back their IND filing date to the end of the year, with trial commencement now set for 2019/2020.

magnus,

I appreciate the critical feedback. All solid points. Of the 3 stocks I currently own, this is by far the riskiest and the only one whose success is going to be defined by a binary point (whether their CAR-T works or not). Based on your questions it sounds like you weren't exactly sure on a few important details (especially regarding Cchek). Please let me know if/how any of the clarifications I made below changes your opinion on this one.

I agree that the chance for success is small, as it is for all biotech companies in the very early stages trying bring an experimental drug to market. Almost all fail. The reason why this one is different (don't laugh ) imo, is because it has been already proven to work in liquid cancers. JUNO and KITE's CAR-T drugs have been smashing successes totally changing the landscape of cancer therapy. The science behind ANIX's approach only targeting FSHR instead of CD-19 is theoretically the same. Of course now they have to prove it in humans, that's the risk, but the science is really sound here based on the CAR-T template.

Yes, doing trials cost money, but that's another reason why I find ANIX appealing. They've announced that it will cost them under $2M to complete P1. Currently ANIX has around $4M+ cash on hand so obviously that's not going to last forever. However, two key points 1) Their partners (Moffitt, Wistar, etc...) are helping with costs of development, and 2) Eventually the plan is to use revenue from Cchek (the cancer diagnostic) to fund the company. They have stressed that they do not plan to raise cash at this time (of course I always take statements like that with a grain of salt), but that if/when they do, that they won't need much.

Cchek doesn't need any more studies nor FDA approval. Instead of going the FDA route, they went the CLIA route (which most companies do for diagnostic products). ResearchDx is the their partner and the lab that will produce the tests. ResearchDx is a *CLIA-certified lab (see bottom) which means once they run and verify their own independent tests of the product then they can ramp up for commercialization. That verification process was completed two weeks ago and ANIX confirmed the that they are still on schedule for a Q3 launch for prostate cancer only:

www.prnewswire.com/news-releases/anixa-biosciences-announces-completion-of-cchek-analytical-verification-at-clia-laboratory-300848404.html

The Cchek test is just a simple blood sample that ideally will be available at any hospital or clinic. The sample is sent to ResearchDx where they process the results in their lab. The whole thing will cost less than $100 and there is a quick turn around for results. They will probably have to do some promotion, but knowing how the current standard of care (PSA test) is so begrudged by doctors due to its inaccuracy, I think they will gladly (and quickly) welcome this test into their practices.

I think the plan for now is see how early sales for Cchek do before making a decision on whether or not to do a raise.

One last point I want to emphasize. Dr. Jose Conejo-Garcia from Moffitt, one of the leading cancer centers/hospitals in the world, invented this technology. ANIX owns it. They are literally doing this together which is a really big deal, imo:

www.prnewswire.com/news-releases/moffitt-cancer-center-and-anixa-biosciences-announce-completion-of-pre-ind-meeting-with-fda-for-car-t-therapy-300734682.html

Moffitt Cancer Center and Anixa Biosciences Announce Completion of Pre-IND Meeting with FDA for CAR-T Therapy

Dr. Jose Conejo-Garcia, chair of the Department of Immunology at Moffitt Cancer Center, the inventor of the technology, and the Principal Investigator of the team developing the therapy added, "Our CAR-T is the first in human therapy of its kind and the feedback we received from the FDA was quite helpful. We are excited to be so close to bringing our investigational therapy to ovarian cancer patients."



Thanks again for the contrarian feedback. It was helpful!



*Once a lab is CLIA-certified by the FDA they can determine through their own verification process which diagnostic products to commercialize. 

Tailwinds had a sit down conversation with the CEO and CFO last week. Some good details on their business model, why dilution isn't much of a fear (if any), the CLIA approval path and timeline for profitability for Cchek, and why insiders have been buying shares on the open market.

In full disclosure, I dumped my position last week. Nothing has changed in my sentiment towards what they are doing and management etc..., and I plan to be back in shortly. While there may be a brief jump when they launch Cchek in Q3, I think it will be a little while before it is revved up and making a splash on the market (Tailwinds even mentions this in his recent blog, but I honestly was thinking this before I read it). The CART-T IND filing has also been pushed back by Moffitt to 2nd half of the year, which imo, means Q4. Where I may slightly disagree with Tailwinds is that the P1 IND filing will be a big event for investors. It's a huge milestone for the company, but I have been around long enough to see highly anticipated IND filings go off with a resounding thud. I'm not saying their won't be a temporary bounce, but it is a long way to P1 data readouts. 

So in summary, I am still very enthused with this company. I just don't feel the immediate urgency to own shares. It's #1 on my watchlist. 

I'm glad I got out of this one a few weeks ago. I still like the scientific potential here, but I felt like it was going to be a while for them to prove themselves (CAR-T for non-liquid tumor therapy) to the market. Thanks magnus, for the reality check. Since then a motion to dismiss a lawsuit against the CEO for warrant pricing (hello ACTC, lol) was denied by a judge which has sent the pps in a down spin. It will ultimately be resolved and things will continued ahead as planned, imo. But for now I am happy to be out of this one. Cchek data is going to be presented in the next few days.