Phase III evaluation of an EOE drug completed in the United States

Compile newborn Takeda pharmaceutical announced on October 28 that the evaluation of tak-721 (budesonide oral suspension, BOS) in the treatment of eosinophilic esophagitis (EOE) reached a common primary and secondary efficacy endpoint in the first study of two key phase III studies, with a statistically significant improvement compared with placebo BOS is a new type of local corticosteroid with mucosal adhesion It is currently being developed in the United States for the treatment of EOE in adolescents and adults This is a randomized, double-blind, placebo-controlled study conducted in 11-55-year-old adolescents and adults with EOE to evaluate the efficacy and safety of BOS treatment for 12 weeks In the study, patients were randomly assigned in a 2:1 ratio to receive 2.0mg of BOS or placebo twice daily for 12 weeks After 6 weeks of placebo introduction, 318 patients received at least one dose of double-blind treatment (Bos, n = 213; placebo, n = 105) The common end points of the study were histological response (peak eosinophil count ≤ 6 eosinophils in high power field) and dysphagia symptom response (DSQ score of dysphagia symptom questionnaire decreased ≥ 30% from baseline to the end of treatment) after the first 12 weeks of treatment The secondary end point was the change of DSQ score from baseline to the end of treatment, and the secondary end point was the change of EEE endoscopic reference score erefs from baseline to the end of treatment It is worth mentioning that this study is the first phase III study successfully completed in the United States to evaluate an EOE drug, and also the largest EOE clinical trial project in the world so far The results showed that after 12 weeks of treatment, the proportion of histological responders and dysphagia responders in Bos group was significantly higher than that in placebo group (the proportion of histological responders: 53.1% vs 1.0%, P < 0.001; the proportion of dysphagia responders: 52.6% vs 39.1%, P = 0.024) In terms of key secondary endpoints, the improvement in the mean DSQ score from baseline to week 12 was also significantly greater in the BOS group than in the placebo group (- 13.0 vs-9.1, P = 0.015) Similarly, compared with placebo group (n = 93), the improvement of the average erefs score in Bos treatment group (n = 202) was significantly greater (- 4.0 points vs - 2.2 points, P < 0.001) In terms of safety, in general, 61.0% of patients reported adverse events (teae: BOS treatment group 61.0%, placebo group 61.0%), and 2.5% of patients experienced dose interruption caused by teae (Bos treatment group 1.4%, placebo group 4.8%) Teaes in ≥ 2% of patients in Bos group or placebo group included nasopharyngitis, sinusitis, esophageal candidiasis, oral candidiasis and upper respiratory tract infection The incidence of oral or esophageal candidiasis teae in the whole study population and each treatment group was less than 5% There were no life-threatening teaes or deaths reported in the study Budesonide molecular formula (source: Wikipedia)