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Verastem Takes a Tumble

Remember Verastem? Back before the current biotech boom got underway (remember the current biotech boom?), they went public shockingly early. It was not a particularly instructive IPO, since the company was targeting cancer stem cells, and those (as discussed here recently) are the subject of both furious research and furious controversy. They went into the clinic with a possible mesothelioma therapy, the FAK inhibitor VS-6063 (defactinib), although you had to wonder how much that one had to do with the company’s putative stem-cell mission.

Well, that part of the story has ended. The company has been enrolling patients in a Phase II trial, using their drug as a maintenance treatment in patients who had already undergone chemotherapy, but didn’t even make it all the way through that before the whole thing was stopped for futility. The company has other compounds in this space still in development, but the implications of this trial’s failure for these efforts are still to be sorted out. The company’s stock is getting hammered today, though, and things don’t look good.

11 comments on “Verastem Takes a Tumble”

  1. innovorich says:

    This is pretend drug discovery.

    The fact that 99% of biotech investors do not understand biotech has both partly caused the current bubble but will also unfortunately take down lots of good companies and good promising science/potential treatments with it, when it bursts.

  2. steve says:

    The rationale behind FAK as a cancer stem cell target is based on Robert Weinberg’s hypothesis that cancer stem cells are derived from epithelial-mesenchymal transitions (EMT) which require FAK. FAK is also involved in cell motility so it is probably important for metastasis. However, these are in vitro observations and to my knowledge (which could be wrong, I don’t know the company’s in vivo data) there hasn’t been a demonstration in vivo that inhibition of FAK alone leads to a therapeutic window where it would inhibit cancers to a greater degree than normal cells. It may be that the compound will do much better if it’s used in combination trials than as a single agent.

  3. steve says:

    Actually, just saw this article that suggests FAK inhibitors could play nicely with immunotherapy approaches (quick buy the stock!). http://dx.doi.org/10.1016/j.cell.2015.09.001
    (P.S. – For what it’s worth, I have no stock or connection to the company…)

  4. J Tyson says:

    The market cap as of a few hours ago was less than half of the $132M cash on hand. I don’t see how this market reaction is at all rational, especially given the quality of Verastem’s people. The founders (Weinberg and Lander) and the lead director (Henri Termeer) are second to none.

  5. Anon says:

    ^ Then a good time for private equity to buy it up and rip out the cash.

  6. ScientistSailor says:

    How come no comments on this? File it under ‘how not to do competitive intelligence’

    http://www.courthousenews.com/2015/09/11/1-billion-claim-over-a-cancer-drug.htm

  7. Ron Burgundy says:

    Hopefully Westphal isn’t marketing snake oil again, ala Sirtris.

    1. innovorich says:

      And yet there is ginger flavored snake oil from Flex Pharma!

  8. Bobby Johnson says:

    you are a fraud, how dare you not cover the recent news that Big Phrama has had the cure to cancer for 40 years and is hiding it to make more money as a cancer curing drug would make about $5.80 yearly. You’ve changed…

  9. El Gorrion says:

    Steve – I remember that Margaret Frame had some in vivo data on the FAK, admittedly GFP tagged FAK, which supported the role of FAK in metastasis. I guess that’s the issue with this target, it’s anti-metastatic (mainly) with relatively little effect on proliferation .

  10. Magrinho says:

    “But is it a selective FAK inhibitor?”
    “It sure is. See, it says it right there on the poster. ‘Potent and selective’.”

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