First-Ever U.S. Pivotal Phase 3 Clinical Study in Eosinophilic Esophagitis (EoE) Completes: Takeda's Investigational Therapy Meets Co-Primary & Key Secondary Efficacy Endpoints

LEXINGTON, Mass., Oct. 28, 2019 /PRNewswire/ -- Takeda Pharmaceuticals U.S.A., Inc., ("Takeda") (TSE:4502/NYSE:TAK)Aannounced today that Budesonide Oral Suspension (BOS), its investigational therapy for the treatment of eosinophilic esophagitis (EoE), achieved co-primary and key secondary efficacy outcomes with statistical significance compared to placebo in the first of two pivotal Phase 3 studies. Positive results on clinical endpoints were presented from a randomized, double-blind, placebo-controlled trial that investigated the safety and efficacy of BOS over 12 weeks of treatment among adolescent and adult patients aged 11 to 55.1AThis is the first pivotal Phase 3 study investigating an EoE treatment ever to be reported in the United States (U.S.)2 and is the largest EoE clinical trial program conducted to date, globally.3,4 Results onAhistologic, symptomatic and endoscopic endpoints were presented today during a Presidential Plenary Session of the 2019 American College of Gastroenterology Annual Scientific Meeting (Oct 25-30, San Antonio, TX).1ABOS is an investigational, novelAmucoadherent topical corticosteroid formulation being evaluated in the United States for the treatment of EoE in adolescents and adults. "I am very much looking forwardAto presenting positive results onAthe clinical endpoints ofAthis Phase 3 trial of Budesonide Oral Suspension, the first U.S. Phase 3 study in patients with EoE to assess histologic, dysphagia, and endoscopic response to a medical treatment," said Ikuo Hirano, MD, Professor of Medicine, Gastroenterology and Hepatology, Northwestern University Feinberg School of Medicine. "Patients with EoE have long suffered with dysphagia and esophageal inflammationAin the absence of an FDA-approved treatment. The results from this Phase 3 study of a medication specifically designed for esophagealAdelivery in EoE are promisingAin addressing an unmet need in the management of EoE."EoE is a rare,5 chronic immune-mediated disease of the esophagus that occurs when eosinophils accumulate in the esophagus, resulting in inflammation.6 It is estimated that approximately 1 in 2,000 people in the U.S. has EoE.7 Children, adolescents and adults with EoE can experience difficulty eating and/or swallowing, potentially resulting in discomfort and pain.8 EoE that is uncontrolled can lead to esophageal remodeling and fibrosis, resulting in dysmotility and/or food impaction.9AIn this clinical study, patients aged 11 to 55 years with EoE and dysphagia were randomized 2:1 to receive 2.0 mg BOS or placebo twice daily for 12 weeks. Following a placebo lead-in period of up to six weeks, a total of 318 patients received at least one dose of double-blind therapy (BOS, n=213; placebo, n=105). The co-primary efficacy endpoints of this study were histologic response and dysphagia symptom response after an initial 12 weeks of therapy. Histologic response was measured as having a peak eosinophil count less than or equal to six eosinophils per high-powered field, and dysphagia symptom response was measured as a greater than or equal to 30 percent reduction from baseline to final treatment as assessed by the Dysphagia Symptom Questionnaire (DSQ), a patient-reported outcome measurement tool. The key secondary efficacy endpoint included change in DSQ score from baseline to final treatment. Additionally, the change in EoE Endoscopic Reference Score (EREFS) from baseline to final treatment period was evaluated as a secondary endpoint. The clinical trial also evaluated the safety of BOS, including measuring the number and types of adverse events. The majority of treatment-emergent adverse events (TEAEs) were mild to moderate in severity and similar between the BOS- and placebo-treated groups. This is the first of two pivotal Phase 3 studies; the findings presented during ACG reflect aggregate data only because the second Phase 3 double-blind, placebo-controlled maintenance study is ongoing.1AThe BOS clinical development program is the first U.S. pivotal Phase 3 program investigating a treatment for EOE based on U.S. Food and Drug Administration (FDA) guidance. The program assesses safety and efficacy in patients with EoE during both an initial 12 weeks of treatment and for a longer duration in the ongoing extension study.2,10,11,12 In parallel with the BOS Phase 2 clinical studies, the DSQ was developed and validated as part of the BOS program. The DSQ met FDA guidelines for use in the registration trials in EoE, and the DSQ is now an established tool in this field of clinical study.13,14,15,16
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