NEJM publishes Boehringer Ingelheim's BIBF 1120 phase II trial on IPF

Boehringer Ingelheim's investigational tyrosine kinase inhibitor (TKI) BIBF 1120 demonstrated a positive trend in reducing lung function decline in patients with idiopathic pulmonary fibrosis (IPF), according to phase II clinical trial results published today online in the New England Journal of Medicine (NEJM). IPF is a progressive and severely debilitating lung disease with a high mortality rate, for which there are no approved treatments in the United States.

In the study, known as TOMORROW (To Improve Pulmonary Fibrosis with BIBF 1120), patients treated with 150 mg of BIBF 1120 twice daily demonstrated a 68 percent reduction in the rate of forced vital capacity (FVC) decline compared to placebo. FVC is the volume of air that is expelled into a spirometer following maximum inhalation. FVC, which is a test that measures lung function, is a part of the examinations conducted in IPF patients and is scientifically accepted for assessment of IPF treatment effects. Patients treated with 150 mg of BIBF 1120 twice daily also had a lower incidence of acute exacerbations, defined as sudden deterioration of clinical status, compared with placebo. Acute exacerbations are associated with rapid disease progression, severe abrupt decline in FVC and high mortality.

In addition, treatment with 150 mg of BIBF 1120 twice daily resulted in a small decrease in impairment of quality of life, as measured by the St. George's Respiratory Questionnaire (SGRQ). SGRQ scores measure the impact of quality of life, with higher scores – as well as increasing scores – signaling greater impairment. In contrast, increased impairment was reported among patients receiving placebo.

Gastrointestinal symptoms and liver transaminase increases were more frequent in patients receiving 150 mg of BIBF 1120 twice daily than placebo and adverse events leading to discontinuation were mostly diarrhea, nausea and vomiting.

"People who suffer from IPF are in great need of a safe and effective treatment to preserve lung function so they can maintain physical activity and reduce the impact on their independence for as long as possible," said Luca Richeldi, MD, PhD, lead study author and director of the Research Centre for Rare Lung Diseases, University of Modena and Reggio Emilia, Modena, Italy. "The positive trends in slowing the decline in lung function over time, reducing the incidence of acute exacerbations and improving the quality of life with BIBF 1120 are a promising proof of concept."

BIBF 1120 received orphan-drug designation from the U.S. Food and Drug Administration in June 2011. Two pivotal phase III clinical trials are currently underway enrolling a total of 970 patients in 20 countries. The first patients entered the trials in April and May 2011, respectively. For more information about the phase III trials or to learn how to enroll, please visit clinicaltrials.gov (identifiers NCT01335464 and NCT01335477).

"The results of the phase II clinical trial for BIBF 1120 in IPF give us the confidence to continue assessing the compound's potential in phase III clinical trials," said Christopher Corsico, MD, MPH, senior vice president, Medicine and Regulatory, North America, Boehringer Ingelheim Pharmaceuticals, Inc. "Boehringer Ingelheim remains committed to identifying an effective treatment for IPF to help bridge the unmet therapeutic need for the thousands of people suffering from this fatal disease."

TOMORROW Data at ERS 2011 Annual Congress

The NEJM paper will be presented at the European Respiratory Society (ERS) 2011 Annual Congress on Monday, Sept. 26, 2011. Additional results of the TOMORROW trial will be presented during oral sessions at ERS on Sunday, Sept. 25, 2011 and at a BI-sponsored symposium on Monday, Sept. 26, 2011. Key presentations include:

• Efficacy of BIBF 1120 in patients with IPF is dose-dependent: results from the TOMORROW trial (Sunday, Sept. 25, 2011, 8:30-8:45 a.m. CEST)
• Effect of baseline FVC on preservation of lung function with BIBF 1120: results from the TOMORROW trial (Sunday, Sept. 25, 2011, 8:45-9 a.m. CEST)
• Presentation of the NEJM TOMORROW paper; Efficacy of a tyrosine kinase inhibitor in idiopathic pulmonary fibrosis at the ERS/NEJM "Late Breaking Research Session" (Monday, Sept. 26, 2011, 1:18-1:36 p.m. CEST)
• Satellite symposium: Idiopathic Pulmonary Fibrosis: Evolutions in Diagnosis and Treatment  (Monday, Sept. 26, 2011, 5:15-7:15 p.m. CEST)