The drug did have enough phase-II trial results to move on. The press releases (not quite scientific reports) use phrases like "did better than" instead of giving hard percentages or odds of success. There sample may also have been too small, because the results for "symptoms" versus "inflammation seen on scope" were a little different. They tested 50mg, 200mg, and 600mg. There was some evidence 600mg might be too much, and there was also some evidence that 200mg did not work as well as 50 or 600.That pattern is most likely the result of a small sample.
There was phase-II evidence that it worked equally well for both people who had never tried a biologic and for people who had failed a biologic. But, the phase-III trial is focusing only on people who have failed a biologic. This might mean they are going after the more desperate patients, or that they are trying to preserve the biologic market - but it could just mean that they can't in good conscience ask anyone to take the chance of landing in the placebo group if they have not yet tried more-proven biologics.
The drug may be important because it is not a biologic. Many people aren't even aware what makes something a "drug" or a "biologic". I read, "A biologic is manufactured in a living system such as a microorganism, or plant or animal cells. Most biologics are very large, complex molecules or mixtures of molecules. Many biologics are produced using recombinant DNA technology." So, this is an anti-inflammatory drug produced by chemistry and not by a living organism and not involving DNA. It is also an attractive anti-inflammatory drug because it targets the signal path in the immune system thought to be most crucial for stopping IBD inflammation, and otherwise leaves other immune responses alone. So, less suppression of the overall immune system.
Of the many signal paths in the immune system, this targets only interleukin-23 (IL-23).
Should folks want to be in this trial? Depends. IF you have UC that is not responding to any mesalamines, immune suppressors, or biologics, then yes. It may be worth a try even if you risk getting worse while sitting untreated in the placebo group. After 12 or 40 weeks, everyone eventually gets the drug in the 3-yr
open label study.
If you are responding to an existing treatment, maybe let the more desperate folks do the trial. You can always switch to the drug later - but why risk getting in the placebo group if you are currently taking something that is working.
They have laid out a multi-year phase-III plan of study. If it continues to prove effective it could go to FDA in 3 to 5 years. It will be interesting to watch this, as well as other therapies, in the pipeline.
Post Edited (DBwithUC) : 12/5/2018 8:19:51 AM (GMT-7)