RE:RE:RE:Shortcuts
I don't want to split hairs, but although phase I goal may be toxicity, ask any oncologist and they will tell you that regardless of the stated goal, they will be watching for melting tumors.
I remember Oxigene, I think it was, who had a phase I trial for their anti-angiogenesis drug in thyroid cancer. Whatever part of the population they were treating, it was one with no alternative treatements. In phase I, a single (1!) patient's tumor disappeared, and the stock went from someting like $2 to $20 over night. Investors didn't wait for long term durability (though they should have); it was only 1 patient; but it caught people's attention.
As an aside, subsequent trials showed that, while it may have a place in the onc's arsenal for thyroid cancer, it was not a game changer, and the stock eventually declined. However, my point is that if you are treating advanced cancers or patients who failed other therapies and you get significant responses in phase I, you will get a very significant market reaction. Yes, the dosage may not be optimal, but surely they will have some sort of dose escalation structure that allows them to test until they have toxicity, possibly up to the point of a tumor regression response.
White probably won't suggest that is what they are looking for, but you can bet that is just as big an interest as toxicity. By the way, speaking of toxicity, you should visit a chemo clinic to see the patients and see what levels of toxicity are deemed "acceptable". I remember one guy who was waiting for his next treatment. He was skin in bones as he sat in the waiting area with the other patients (looked like a bunch of concentration camp survivors who had just been given nice clothes), holding a garbage can between his knees and he wretched non stopfor about 30 minutes, and this was residual effect from his previous treatment...he hadn't yet received this day's treatment...and nobody in the room batted an eyelash. They were used to it in that part of the hospital.