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The Lessons of Intercept and NASH

Over at LifeSciVC, Tom Hughes has a post about Intercept Pharmaceuticals and their wild ride with an FXR ligand for non-alcoholic steatohepatitis (NASH). Anyone who owned ICPT will recall that period vividly, since the positive news from the clinical trial sent the company’s stock from an already-not-cheap $72/share to a where’s-the-oxygen-tank peak of $445 in two days of bug-eyed trading. NASH is potentially a big market, and there’s really nothing out there to treat it, so Intercept’s investors (many of them apparently fresh converts to the cause) decided that the company was set to do very well for itself indeed.
Hughes points out that NASH is not an easy thing to get into, though. It’s underserved for a reason – there are a lot of biological and mechanistic question marks, and the clinical trials are no stroll through the tulip beds, either . There’s no way to be sure that you’re having the desired effects without doing a series of liver biopsies on the patients, and even at that the FDA still has to issue some key regulatory guidance about whether these tissues markers will be sufficient for approval. His thesis is that it took a small company like Intercept to accept all these risks.
He’s probably right, especially when you couple them with an uncertain market size – potentially large, but who knows for sure? It’s a hard sell at a big company, where the feeling can be that if you’re going to go to all that trouble, you might as well aim for a larger market while you’re at it, and one with more clarity. Adding a few hundred million (maybe) to the sales figures of Leviathan Pharma might not get upper management all that excited, especially since so much stuff would have to get pioneered along the way. But adding a few hundred million to the revenues of a small company that has nothing else – now that’s something worth suiting up for. All the talk about how small companies are so much more nimble and innovative (which is not always true, but certainly not all talk, either) might just come down to the old line about necessity being the mother of invention. Or Samuel Johnson’s old line about when a man is to hanged in a fortnight that it concentrates his mind wonderfully – that one, too.
Intercept, for its part, has seen its stock fall back to the lowly $250-300 range, which probably isn’t thrilling for the folks who bought in at $445. Actually, it fell back to that range almost immediately, then worked its way back over $400 again, and now back down to this, so there have been plenty of opportunities for hair loss among its investors. When the FDA issues its guidance in the NASH area, it will presumably go through another blast of frenzied trading, and good luck to whoever’s in it (long or short) when that happens. And I haven’t checked, but I’ll bet that the options on this one would cost you plenty, too. From a safe distance, it should be quite a show.

5 comments on “The Lessons of Intercept and NASH”

  1. MedChem says:

    An interesting post. In my early thirties I had a through medical checkup and profiling. From the blood work it appeared that I had a very high triglycerides and elevated (1.5 to 2 fold) liver enzyme count. An additional tests followed and the doctor quickly ruled out usual suspects like Hepatitis A/B and what was known then as non A/B (which is Hepatitis C) that may inflame and also elevate liver enzymes during the active phase of infection. After some more time when things did not fall in place (Liver enzyme count at this point in time returned near normal) but the triglycerides were still stubbornly high. At this point in time he suggested liver biopsy and recommended a gastroenterologist of great reputation. The biopsy procedure itself was brutal and he used gauze 24 needle to puncture by liver to get some tissue and the fluid out. I was in pain for the next two days but my anxiety were laid to rest when he informed me that I had a “fatty liver” but otherwise OK. He asked to cut down on my binge drinking (the surprise was, I do not drink!) and other fat rich food. Fast forward to the present, am in my late fifties and am still with this condition except that I am maintaining my BMI, diet and also keeping other markers in control (cholesterol, Sugar. So, I am completely surprised that you do not have to drink alcohol to get this condition. I am not sure I will be taking medication for this condition knowing well that the drug will have to work in the liver. So the FDA guidance is well warranted. It is my understanding that this conditions is widely prevalent in countries rich in fatty food (South Asia. And, people may not know that they have this condition.

  2. NMH says:

    @1- if you have not, get a DEXA exam to find out how fat you really are. Other measures can greatly underestimate obesity.
    I was shocked about 3 years ago when with a BMI around 28 I had 33% BF by DEXA. So now Im on a nearly vegan diet and have dropped to almost 20% Presumably my liver is not as fat.

  3. Morten G says:

    Why on earth would fatty foods give you NASH?
    Alcoholic fatty liver = lots of energy produced in liver during detoxification / catabolism of alcohol.
    Non-alcoholic fatty liver = (probably also) lots of energy produced in liver during detoxification / catabolism. Fat isn’t going into your liver, it is going out. Fructose and galactose however can only be metabolised in the liver. If you had low glycogen and not-high blood glucose they would be converted into glucose and stored or exported. But if you have significant amounts of fructose and galactose then you also have large amounts of glucose.

  4. AR says:

    My understanding is that the fat in NAFLD and NASH patients is just along for the ride. Once you have NASH cutting all fat from diet has no effect on disease progression.

  5. Anonymous says:

    Fat intake does not equal fat in the liver. It’s carbs that mostly get converted to fat in the liver. High protein/low carb diets reverse NASH but the question is what they do to your heart and kidneys. What’s good for one organ isn’t always good for another.

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