The P-II (INSPIRE) trial evaluates Zygel (250/500mg, qd) in 30 children & adolescents aged 4 through 15yrs. with 22q11.2 deletion syndrome at 2 clinical sites in Australia and 1 in the US
The results showed an improvement from baseline in multiple efficacy assessments @14wks. including ADAMS, ABC-C, PARS – R & CGI-I, 75% were rated as “improved”, “much improved” or “very much improved” with 62.5% being “much improved” or “very much improved”, was well tolerated, and the safety profile was consistent with prior data from other Zygel clinical trials
1 patient discontinued treatment due to AEs not related to Zygel. The company has chosen to focus its resources on 22q & FXS, both of which have been received ODD from the US FDA
Ref: GlobalNewswire | Image: Zynerba
