Corbus Pharma (CRBP) Reports Agreement with FDA on Phase 2b Cystic Fibrosis Study Design with Pulmon
Multi-national Phase 2b is designed to provide clear evidence of clinical benefit of lenabasum (formerly known as anabasum) in patients with CF
Study will enroll ~415 participants with CF 12 years of age and older regardless of underlying CFTR mutation, infection, or background medications
First patient expected to be dosed this quarter
Management to host conference call and webcast on Tuesday, January 30th at 10:30 a.m. EST
Corbus Pharmaceuticals Holdings, Inc. (NASDAQ: CRBP) ("Corbus" or the "Company"), a clinical stage drug development company targeting rare, chronic, serious inflammatory and fibrotic diseases, today announced that it has reached agreement with the U.S. Food and Drug Administration ("FDA") regarding the design of its next study of lenabasum (formerly known as anabasum), a novel, oral, pro-resolving drug, in the treatment of cystic fibrosis ("CF"). The Company and the FDA agreed that the event rate of pulmonary exacerbation is an acceptable sole primary efficacy endpoint for the clinical development program to support registration of lenabasum for the treatment of CF. Event rate of pulmonary exacerbation is the average number of pulmonary exacerbations per subject per time period. The FDA also agreed to the inclusion of adolescents 12-17 years of age alongside adults in the Phase 2b study. The Company expects the first patient to be dosed during this quarter.
The Phase 2b multicenter, double-blinded, randomized, placebo-controlled study will enroll approximately 415 subjects with CF who are at least 12 years of age and at increased risk for pulmonary exacerbations. Secondary efficacy outcomes include other measures of pulmonary exacerbations, change in Cystic Fibrosis Questionnaire-Revised Respiratory domain score and change in forced expiratory volume in 1 second (FEV1), % predicted. The study will be conducted in approximately 100 sites across North America, Europe, Israel and Australia. Subjects will be centrally randomized to one of three cohorts to receive lenabasum 20 mg twice per day, lenabasum 5 mg twice per day, or placebo twice per day for 28 weeks, with 4 weeks follow-up off active treatment. This Phase 2b CF study was designed with input from the Therapeutic Development Network of the Cystic Fibrosis Foundation and the European Cystic Fibrosis Society Clinical Trials Network.
"Pulmonary exacerbations are a key driver of morbidity and mortality in cystic fibrosis," said Barbara White, M.D., Chief Medical Officer of Corbus. "We now have agreement from the FDA that the event rate of pulmonary exacerbations is an acceptable primary efficacy outcome in the clinical development program to support registration of lenabasum for the treatment of CF. The FDA's input underscores the importance of new treatments that reduce the number of pulmonary exacerbations. With about 415 patients treated for 6-months, the next Phase 2 study is large enough and long enough to potentially provide statistically significant data regarding benefit of lenabasum in pulmonary exacerbations in CF."
Lenabasum is the only CF treatment currently in development designed to trigger the resolution of inflammation without immunosuppression. Its mechanism of action which reduces inflammation is applicable to all CF patients regardless of underlying CFTR mutation, infection, or background medications. Lenabasum reduced inflammatory cells and mediators in sputum and was associated with a favorable safety profile, reduced proportion of subjects with pulmonary exacerbations, and longer time to first pulmonary exacerbation in an earlier, completed 85-subject double-blinded, placebo-controlled 16-week Phase 2 study.
Yuval Cohen, Ph.D., Chief Executive Officer of Corbus, added, "The outcome of our meeting with the FDA represents a very significant milestone for our CF development program and builds on the foundation provided by the clinical data from our previous Phase 2 study. Lenabasum is the first pro-resolving drug to be tested for efficacy in CF and the first experimental therapeutic to be tested in CF patients with the event rate of pulmonary exacerbation as the sole primary efficacy endpoint, without a requirement from the FDA to consider FEV1% as a co-primary endpoint. We look forward to working closely with the stakeholders within the CF community on successfully executing this first of its kind trial."
Lenabasum was granted Orphan Drug Designation and Fast Track status for the treatment of CF by the FDA in 2015 and Orphan Drug Status from the European Medicines Agency (EMA) in 2016.
Conference Call and Webcast Information
Corbus management will host a conference call for investors, analysts and other interested parties on Tuesday, January 30, 2018 at 10:30 a.m. EST to discuss the Company's upcoming Phase 2 study evaluating lenabasum for the treatment of CF.
The conference call and live webcast will be accompanied by a slide presentation. To participate in the call, please dial (877) 407-3978 (domestic) or (412) 902-0039 (international). The live webcast and accompanying slides will be accessible on the Events page of the Investors section of Corbus website, www.corbuspharma.com, and will be archived for 60 days.