Meritage Pharma Announces Positive Results of Oral Budesonide Suspension for Treatment of Eosinophilic Esophagitis in Adolescent and Adult Patients

September 25, 2014

SAN DIEGO, Sept. 25, 2014 /PRNewswire/ -- Meritage Pharma, Inc. announced today positive Phase 2b results of oral budesonide suspension (OBS) for the treatment of adolescents and adults with eosinophilic esophagitis (EoE), a chronic disease characterized by symptoms related to esophageal dysfunction and eosinophil-predominant inflammation. OBS achieved the co-primary endpoints of reduction in inflammation (p< 0.0001) and symptom of dysphagia (p=0.0096) with statistical significance. In addition, statistical significance was demonstrated for other secondary endpoints including histopathologic score, endoscopic score and physician global impression of disease activity. OBS was well tolerated.

"While previous studies have identified success in diminishing inflammation, this is the first study to show that a medication can significantly improve both symptoms and esophageal inflammation. These exciting results should provide new hope to those affected by eosinophilic esophagitis," said Glenn Furuta, M.D., Director, Gastrointestinal Eosinophilic Diseases Program Children's Hospital Colorado and Professor of Pediatrics, University of Colorado School of Medicine.

The double-blind, randomized, placebo-controlled 16-week study enrolled 119 patients aged 11 to 40 with confirmed EoE and documented problems swallowing food. The trial was conducted at approximately 25 U.S. clinical centers and a 24-week open label extension is ongoing. The effectiveness of OBS versus placebo was measured by a dysphagia symptom score determined from patient daily responses to the electronic Dysphagia Symptom Questionnaire™ (DSQ) tool and by evaluating the change in eosinophilic inflammation in biopsy samples from the esophagus at the beginning and end of treatment. Meritage plans to present the full results at an appropriate medical meeting in the future.

"An important component of this successful trial was the first use of our proprietary clinical symptom tool that captures patient reported measures of dysphagia, or difficulty swallowing, enabling us to clearly discriminate symptom response between the active and placebo treatment groups," said Elaine Phillips, Ph.D., President and Chief Executive Officer of Meritage Pharma. "Combined with our first Phase 2b trial, we have robust data demonstrating the potential for oral budesonide suspension as a therapeutic option for the large and growing EoE patient population that lacks an approved medication."

Meritage's initial 12-week Phase 2 placebo-controlled dose-ranging trial evaluating 71 pediatric patients met the combined primary endpoint of reduction of esophageal eosinophilia and improvement in clinical symptom score as assessed by the treating physician. 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. The diagnosed EoE population is approximately 180,000 in the U.S. and is expected to reach 272,000 in the U.S. and 305,000 in the five largest European countries by 2018. EoE may be triggered by a variety of stimuli including certain foods and environmental allergens. People with EoE commonly have other allergic conditions such as asthma or eczema and may experience symptoms for months or years prior to the initial diagnosis. 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. While eliminating certain foods from diets may help some patients, there are no FDA approved therapies.

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 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. 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.

About Meritage Pharma

Meritage Pharma is committed to the development of prescription products based on safe and effective molecules for the treatment of gastrointestinal and atopic diseases. The company was founded in 2008 by a management team that has an established track record of building successful specialty pharmaceutical companies and in identifying and developing novel products for atopic diseases. More information about Meritage Pharma is available at www.meritagepharma.com. Meritage™ is a trademark of Meritage Pharma, Inc.

Forward-Looking Statements

Meritage Pharma cautions you that statements included in this press release that are not a description of historical facts may be forward-looking statements. The inclusion of forward looking statements should not be regarded as a representation by Meritage Pharma that any of its plans will be achieved. Actual results may differ materially from those set forth in this press release due to the risks and uncertainties inherent in Meritage Pharma's business including, without limitation, statements about: difficulties or delays in developing, obtaining regulatory approval, manufacturing and commercializing its products; unexpected performance or side effects of its products that could delay or prevent development or commercialization; the scope and validity of patent protection for its products; competition from other pharmaceutical companies; and its ability to obtain additional financing to support its operations. All forward looking statements are qualified in their entirety by this cautionary statement and Meritage Pharma undertakes no obligation to revise or update this press release to reflect events or circumstances after the date hereof.

SOURCE Meritage Pharma