Esophageal Disorders

October 23, 2017

Regeneron Pharmaceuticals and Sanofi announced positive results from a phase II study of dupilumab in adults with active moderate-to-severe eosinophilic esophagitis. The primary endpoint of the study was the change from baseline to week 10 in the Straumann Dysphagia Instrument (SDI) score, a patient-reported measure of swallowing difficulty on a zero to nine point scale, with nine indicating more severe symptoms. A total of 47 patients were randomized into two treatment groups in this 12-week treatment study, and both groups had a mean baseline SDI score of 6.4. Patients received either dupilumab 300mg weekly following a 600mg loading dose or placebo. At week 10, patients who received dupilumab 300mg weekly reported a significant improvement in the ability to swallow with a three point reduction in their SDI score (45% improvement) compared to 1.3 points (19% improvement) for those patients who received placebo (p=0.0304). The mean change in the Eosinophilic Esophagitis Endoscopic Reference Score (EoE-EREFS) was significantly reduced by 1.9 points from baseline (48% improvement) in patients who received dupilumab weekly compared to 0.3 points (7% improvement) for those who received placebo at 12 weeks (p=0.0006). The mean baseline score for the dupilumab group was 3.9 and for the placebo group was 4.3. There were no new significant safety concerns in this trial. Higher rates of injection site reactions were observed on dupilumab versus placebo. Dupilumab recently received Orphan Drug Designation from the FDA for the potential treatment of eosinophilic esophagitis.

May 16, 2011

Meritage Pharma released results from a phase IIb trial of their proprietary oral budesonide suspension (OBS) for esophageal eosinophilia. This double-blind, randomized, placebo-controlled trial, PEER, enrolled 82 pediatrics ages two to 18 years who received low, medium or high dose OBS or placebo daily for 12 weeks. Treatment response was defined as subjects with <6 eosinophils (EOS) per high power field (HPF) in all esophageal levels and >50% symptom score reduction at the end of 12 weeks of treatment. Data are from 71 subjects who completed the treatment. At end of treatment, there were statistically significant greater percentages of responders in both the medium and high dose groups compared to placebo (52%, 47% and 5%, respectively). Subjects in the medium and high dose groups also demonstrated statistically significant histologic remission (<1 EOS per HPF) compared to placebo, as well as statistically significant reduction of endoscopic findings and epithelial inflammation. OBS was well tolerated.