RE: Example of a Phase 1a (dose escalation) data summary5 Jan 2023 16:51
One thing that is instructive, as illustrated by that trial, is in the summary but there is more detail on slide 13 here: http://ir.crisprtx.com/static-files/bfba41c6-5c28-4681-9002-bdfcef21c3f7
The Dose Levels are Cell dose (CAR+ T cells)
N is te number of patients in each cohort
DL1, 30x10(sup6), N=3, Overall response: Inactive. A big zero.
DL2, 100x10(sup6), N=3, Overall response: 1 (of the 3), (33%)
DL3, 300x10(sup6), N=6,Overall response : 3 (50%)
DL3.5, 450x10(sup6), N=6, Overall response: 4 (67%)
DL4, 600x10(sup6), N=8, Overall response: 6 (75%)
The same trend for Complete response.
The increases were 3.3x, 3x, 0.5x and 0.33x, i.e. big increases until an effect was found and then decreasing percentage (but constant level) increases.
Contrast that with the AVA6000 dose levels:
Initial: 80mg/m²
DE1: 120mg/m² (0.5x increase)
DE2: 160mg/m² (0.33x increase)
DE3: 200mg/m² (0.25x increase)
These are decreasing percentage but constant level increases, which indicate that it was working even from the initial dose level. As also supported by promoting AVA3993 to preclinical and all the other indications of success we have witnessed since.