For phase 1 what you really want is a good safety profile, anything on top of that is a bonus.
But of course if you see no activity i.e. no benefit in phase 1, then the company won't progress to phase 2.
So the fact they are seeing some activity i.e. stable disease at low doses, then it is correct to say it is 'encouraging' because the real action begins once they ramp up the dosage in cohort 3 and 4.
For the two patients in the second cohort showing stable disease, it is likely that they are still being monitored and the treatment may either continue to have an effect over time or it will cease to be effective and then the patient will progress and they will stop treatment.
What we want to see are the new centres up and running soon, as that will speed up recruitment. Their aim is to get all 4 cohorts done by the end of this year. They will then likely choose the most likely dose to have the best impact, and then do one last round of patients to get a feel of what type of benefit it will achieve in a large patient population rather than the smaller 3-4 patients they've been recruiting in each cohort. So they might do 10-15 patients using an optimised dose, just to test the waters. That may take us out to mid or late 2023 before the final results are known, though as like today it seems they will give updates as the trial progresses.. so if they do see a partial or complete response.. that in itself may be a price sensitive announcement to make at any time IMO of course.
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Ann: Encouraging data from 2nd dose cohort in CLTX CAR T trial, page-11
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