The P-III study results published in Nature Medicine evaluating Daybue vs PBO in 187 girls and young women aged 5-20yrs.
The results showed an improvement over PBO on co-primary efficacy EPs, mean change from baseline to 12wk. in RSBQ total score was −5.1 vs −1.7, and LSM changes from baseline to 12wk. in the RSBQ total score, based on the MMRM analysis was −4.9 vs −1.7, change from baseline for all RSBQ domain subscores were numerically in Daybue’s favour
Mean CGI-I scores @12wk. (3.5 vs 3.8), MMRM analysis showed an improvement with an LSM difference of −0.3, a similar benefit was observed in the subgroup analysis irrespective of age, baseline RSBQ severity & category of documented disease-causing MECP2 mutation severity
Ref: BusinessWire | Image: Acadia
Related News:- Acadia Pharmaceuticals Launches Daybue (trofinetide) for the Treatment of Rett Syndrome in the US
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