It's not completely statistically meaningless hottuna. Agree...

It's not completely statistically meaningless hottuna. Agree that the sample sizes are tiny and that is why the company hasn't gotten carried away. However, whether the numbers are "meaningless" or not depends partly on the prior probabilities. This is not a coin toss. Many of these patients are those who have failed all other treatments, so the expected probability of success under the null hypothesis that the treatment has no effect will be very small. To get a circa 50% of some response rate is encouraging for this cohort. To me it means, hey there could be something in this, let's explore more.

I agree it is impossible to make definitive conclusions from this study, but I think it is important to bear in mind the patient selection and also the purpose of the study being dose escalation for safety and efficacy. It's hard to argue that the results are bad. I wouldn't call them meaningless. At the very least it provides a suggestion for trial continuation. As usual with clinical trials, time will tell. We now await the results from cohort 2, that's pretty much all we can do now...