Looking at the Phase 2 results, they saw good neurological recovery (measured by GOS) after 1/3/6 months in 20%/40%/47% of patients getting drug vrs approx 3%/19%/32% of patients getting placebo (improvements of 6 fold / 2 fold / and 45%).
There were only 67 patients in the trial (30 Dex, 37 placebo), but the strong drug effect is readily apparent. And in the more injured GCS 4-6 cohort, at 6 months there was an approx THREE FOLD improvement in good neurological outcome in the Dex patients vrs placebo.
If the Phase 3 results are equally compelling (of course that's always the big "if"), how could the FDA not approve such a drug, considering the current lack of treatments, and the drug's excellent safety profile? Wouldn't a 6 month 45% GOS improvement favoring Dex over placebo easily be sufficient for approval?
And there are reasons to expect even better results from the Phase 3 -
A) MGCS is lower for the Phase 3 (approx 3.8 vrs 4.21 for the Phase 2), so the Phase 3 patients have had a higher level of brain trauma, and thus are more likely to show drug effect.
B) GOSE is more sensitive than GOS.
C) The motor component, the most sensitive part of the GCS score, must be 2-5 in the Phase 3.
D) More stringent CT criteria in the Phase 3 to confirm frontal lobe trauma.
E) The Phase 3 will able to show drug effect between age groups (a 20 year old vrs a 60 year old for example) - a drug effect that wouldn't previously have been counted.
F) Phase 3 enrollment criteria was very stringent - they accepted only 1 patient in 10. |
