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Celgene's (NASDAQ: CELG) $500 Million Cancer Bet

One Drug to Treat Them All

Written by Jason Stutman
Posted March 31, 2014

How many different types of organs are there in the human body?

The number might actually surprise you.

When asked this question, most people are quick to count the usual suspects: stomach, kidneys, heart, liver, pancreas, lungs, spleen, bladder, and so forth. That gives us eight, but we're still way off.

Let's not forget the gallbladder, appendix, trachea, thyroid, interior vena cava, superior vena cava, thymus gland, larynx, lymph nodes, and hypothalamus...

We could keep on going, but that would probably take a while, and it's not my intention to bore you. After all, we just named 18 different organs, and we're still not even a quarter of the way there yet. To cut to the chase, there are a total of 78 different types of organs in the human body, each with its own structure and function.

And of these 78 organ types, how many are susceptible to their own specialized versions of cancer?

Just about every single one.

In fact, there are over 200 different variations of cancer that have specifically evolved to wreak havoc across the widely varying landscapes of the body, and each one has its own operating mechanisms and unique defense systems.

Or at least that's what we initially thought...

And it's for this reason that the true holy grail of biotechnology — an overarching “cure for cancer” — has been so elusive to scientists since Hippocrates first diagnosed the disease nearly 2,500 years ago.

Yet in just the last 10 years, researchers have pinpointed what may actually be the key to finding a common cure: a common defense system used by virtually all forms of cancer.

For the first time in history, we're looking at the possibility of a single drug treating all forms of cancer... and human trials are about to get underway.

Breaking Cancer's Camouflage

A decade ago, researchers at Stanford University School of Medicine found an abnormally high concentration of a specific protein called CD47 when analyzing leukemia cells. Normally found on healthy blood cells, these CD47 proteins function as “do not kill” flags for passing immune cells known as macrophages.

What this meant was that the leukemia cells were hiding from our immune system by masking themselves with a common bodily protein — they were pretending to be healthy living cells. At first, this was simply seen as a clever form of molecular camouflage. But CD47 is turning out to have implications on a far grander scale.

Fast forward to the present, and the same Stanford researchers are gearing up for potentially groundbreaking human trials surrounding this discovery. Amazingly, CD47 was found in high concentration not just on leukemia cells but “on every single primary tumor that [was] tested,” according to Stanford biologist Irving Weissman.

The implication of the research is that not just leukemia but also breast, ovary, colon, brain, bladder, liver, and prostate cancers all wear this same form of camouflage. Figure out how to break it, and there's your common cure.

Excitingly, this is exactly what scientists are aiming to accomplish right now, and so far, the results are more than promising.

By developing an anti-CD47 antibody, researchers have already successfully blocked cancer's “do not kill” flag in human tumors transplanted into mice. The results varied by tumor type, but it was always a positive outcome.

In bladder tumors, the anti-CD47 antibody reduced cancer migration to lymph nodes by 90%. In colon cancer trials, tumors shrank in size by an average of 68%. And in breast cancer, the antibody eliminated cancer entirely.

Of course, as with any drug this early in development, there are two very important questions left to be answered. First is whether or not these results will transfer over to a more complex human system. Targeting a tumor implanted into a rodent's leg is an ideally isolated environment; the human body is not.

Second is the matter of safety and side effects. There is strong warrant for toxicity concern here considering the fact that CD47 is so commonly found in our bodies. While anti-CD47 antigens seem to be more attracted to the high concentrations of the protein on cancer cells, they will still disturb healthy functioning cells containing the protein.

When treating leukemia with the drug, researchers found a temporary decrease in red blood cells, meaning the macrophages were now viewing them as foreign intruders. Fortunately, the mice were able to compensate and replace these cells without any adverse effects.

If toxicity levels are as limited in human trials, anti-CD47 antigens could be the most significant medical discovery since penicillin. At this point, though, we'll have to wait and see.


The Company with the Cure

As an investor, the obvious question to ask here is which company will have the rights to this potential blockbuster. At this point, that answer is unclear because the patents to this specific antigen (known as Hu5F9) still belong to the Board of Trustees of the Leland Stanford Junior University, with no record of outside contracts.

But after some digging, we now know who else is currently pursuing anti-CD47 antigens, and they have a telling connection to a publicly traded company.

On June 27, 2012, Celgene Corporation (NASDAQ: CELG) announced a significant option and license agreement with small and privately owned biopharmaceutical company Inhibrx.

Celgene, of course, didn't say much about what it was after with the potentially $500 million-plus agreement, but President of Research and Early Development Tom Daniel had the following to share at the time:

“Inhibrx has developed an antibody with strong pre-clinical study results on a highly validated target with very promising therapeutic potential.”

Alone, the statement wasn't too revealing, but when put into context now, it's almost certain what this deal was all about. Though it couldn't have been pinpointed at the time, evidence now heavily points to Celgene's interest in CD47 antibodies.

In February of 2013, Inhibrx filed patent WO2013119714 A1, CD47 Antibodies and Methods of Use Thereof. Though officially filed nearly eight months following the agreement with Celgene and not published until August of 2013, the patent holds a priority date of Feb 6, 2012 — just four months before the agreement was inked.

As far as I know, Inhibrx's next most recent antibody patent filing was back in July 2008, making it highly probable that the CD47 antibody is what Celgene was referring to in its public statement last year.

To support this thesis further, Celgene is also listed as a collaborator on a multi-year, $18.7 million funded development project led by the University of Oxford focused on using CD47 antibodies to treat leukemia. It certainly seems the company is highly interested in pursuing this route.

Although Celgene already resides amongst the heavyweights of biotech, its $59 market cap is relatively small compared to players like Gilead (NASDAQ: GILD) and Amgen (NASDAQ: AMGN). If the company moves forward with CD47 human clinical trials, don't be surprised if it begins approaching the $100 billion mark.

The potential for a common cancer cure would be enough to drive the market absolutely mad.

Turning progress to profits,

  JS Sig

Jason Stutman

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