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April 29, 2014
Concert's First Drug: Not So Great
Concert Pharmaceuticals, famous among medicinal chemists as the apparent leaders in the "deuterated drug" strategy, has reported clinical trial results for their lead compound, CTP-499. Unfortunately, it missed its primary endpoint of improving albumin/creatinine ratios in patients. There are some signs that it might be doing some good, and some room to argue that this endpoint is not as good a choice as it seemed to be at the time, but overall, these are not good numbers.
And as this article in Xconomy shows, the initial read on these numbers actually hit last summer, while Concert was readying an IPO. They pulled it, realizing the effect that the results would have on their fund-raising abilities, and took the compound into a second 24-week round of testing, the results of which we're seeing now. Fortunately for Concert, another compound (CTP-354) showed enough promise in the interim to be the peg for their public offering, which went off in February (pretty much just in time, given what's happened in the biotech market since then).
It's a bit alarming to realize how much of a small company's fortunes depend on, well, fortune. Maybe that should be a capital-F Fortune, the blindfolded goddess depicted in allegorical paintings holding a large wheel on which people's standing rose and fell. You'd like to be able to control things, and you try to as much as possible, but a lot is riding on things that are outside of anyone's control whatsoever: what the results are, and what the scientific and financial weather is when they appear.
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