Boehringer Ingelheim Pharmaceuticals, Inc. announced that clinical trial enrollment has completed for two phase III studies evaluating the safety and efficacy of nintedanib (BIBF 1120), an investigational compound, in patients with idiopathic pulmonary fibrosis (IPF), being studied at a twice-daily oral dose. There are no approved treatments in the U.S. for IPF, a progressive and severely debilitating lung disease with a high mortality rate: approximately 60 percent of patients with IPF die from the disease within two to five years of diagnosis. IPF is characterized by inflammation and scarring of lung tissue, called fibrosis, and over time, the lungs lose their ability to take in and transfer oxygen into the bloodstream, and vital organs do not get enough oxygen. As a result, individuals with IPF experience shortness of breath and often have difficulty participating in everyday physical activities. Research indicates that IPF may affect approximately 100,000 Americans.
In June 2011, the U.S. Food and Drug Administration (FDA) granted nintedanib orphan drug status, which identifies compounds for rare diseases.
"With no FDA-approved treatments, today's standard of IPF care is limited to oxygen therapy and lung transplant for some patients," said Dr. Kevin R. Flaherty, a study investigator and associate professor in the Division of Pulmonary and Critical Care Medicine, Department of Internal Medicine, University of Michigan Health System. Dr. Flaherty is a paid member of Boehringer Ingelheim's nintedanib clinical trial steering committee. "IPF patients desperately need safe and effective treatments to not only reduce the decline in lung function and eventually decrease mortality, but also to stabilize health-related quality of life and delay or reduce sudden worsening of symptoms, or acute exacerbations. These are hallmarks of IPF and are often times unpredictable and can cause death."
The two global phase III studies are identical in design, constructed as double-blind, randomized, placebo-controlled trials with a 52-week duration and matching twice-daily 150 mg dosing, inclusion criteria and endpoints. The primary endpoint is the annual rate of decline in forced vital capacity (FVC), or the volume of air that is expelled into a spirometer following maximum inhalation. Reductions in FVC are reflected in impaired ventilation capacity of the lungs. Measuring FVC is a part of the examinations conducted in IPF patients and is scientifically accepted for assessment of IPF treatment effects. Secondary endpoints include health-related quality of life, exacerbations, respiratory mortality, overall survival and on-treatment survival. The trials have enrolled a total of 970 patients in 20 countries. The first patients entered the trials in April and May 2011, respectively.
"There is a clear need for approved treatments for IPF patients and we look forward to completing the trials and analyzing results from the phase III studies," said Tunde Otulana, MD, vice president, Clinical Development and Medical Affairs, Respiratory at Boehringer Ingelheim Pharmaceuticals, Inc. "Boehringer Ingelheim is committed to ongoing research which we hope will help identify a safe and effective therapeutic option."
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