Meritage Pharma, Inc. announced that it has initiated a Phase 2 clinical trial to evaluate oral budesonide suspension (OBS) for the treatment of adolescents and young adults with eosinophilic esophagitis (EoE), an allergic inflammatory condition of the esophagus. EoE is a chronic disease characterized by symptoms related to esophageal dysfunction and eosinophil-predominant inflammation. The trial is being conducted at multiple U.S. clinical centers that specialize in the treatment of patients with gastrointestinal diseases.
The double-blind, randomized, placebo-controlled 16-week study will enroll about 100 patients aged 12 to 25 with confirmed EoE and documented problems swallowing food. Patients will be evaluated after treatment of OBS or placebo by performing biopsies of the esophagus to assess eosinophilic infiltration. In addition, a symptom scoring tool will be used to evaluate patients' response to therapy. More information about the study may be found at www.clinicaltrials.gov, identifier NCT01642212.
Meritage's initial 12-week Phase 2 placebo-controlled dose-ranging trial evaluating 71 pediatric patients met the primary endpoint of reduction of esophageal eosinophilia and improvement in clinical symptom score. OBS was well tolerated with no serious adverse events.
EoE is an emerging disease that has been increasingly recognized by the medical community with a dramatic rise in its incidence and diagnosis over the last decade. The disease occurs when eosinophils, a type of white blood cell involved in allergic reactions, infiltrate the surface of the esophagus. This eosinophil infiltration leads to inflammation of the esophagus and is believed to cause the persistent or relapsing symptoms of EoE. A variety of stimuli may trigger this allergic process including certain foods and environmental allergens. People with EoE commonly have other allergic conditions such as asthma or eczema.
Symptoms of EoE vary among age groups. Infants and toddlers experience feeding difficulties and failure to thrive. School-aged children may have nausea, vomiting and stomach pain. Adolescents and adults most often present with symptoms of dysphagia (difficulty swallowing) and food impaction. Other symptoms may include reflux that does not respond to proton pump inhibitor therapy, heartburn, chest pain and poor appetite.
"We look forward to our ongoing collaboration with stakeholders from industry, academia, patient advocacy associations and the FDA to evaluate OBS as a potential therapy for EoE," said Elaine Phillips, Ph.D., CEO of Meritage Pharma, Inc. "Patients with EoE struggle for months or years to the initial diagnosis only to find there are no approved pharmacologic therapies. We are eager to continue our clinical program to evaluate OBS to enable patients to thrive, despite their disease."
OBS is a proprietary viscous oral formulation of budesonide that is designed to coat the esophagus where the drug can act locally. Budesonide is the active pharmaceutical ingredient in several products approved by the U.S. Food and Drug Administration (FDA), including products for the treatment of pediatric asthma, allergic rhinitis and Crohn's disease.
Budesonide is a corticosteroid and has an established safety profile in those diseases. It works by decreasing inflammation in the treated area. The FDA has granted Orphan Drug Status designation to OBS for the treatment of patients with EoE. Orphan drug designation generally provides the developer with market exclusivity for the product for seven years following FDA approval, in addition to other incentives.