Benzinga | Jul 22, 2020 08:01AM EDT

AzurRx BioPharma Reports Initiation Of Phase 2b Trial Of MS1819 In Cystic Fibrosis, First Patient Screened, 3 Sites Activated; Sees Topline data Q1'21

AzurRx BioPharma, Inc. (NASDAQ:AZRX) ("AzurRx" or the "Company"), a company specializing in the development of non-systemic, recombinant therapies for gastrointestinal diseases, today announced that it has initiated its Phase 2b OPTION 2 clinical trial to investigate MS1819 in cystic fibrosis (CF) patients with exocrine pancreatic insufficiency (EPI) with the activation of three clinical sites and initial screening of the first patient. Top line data from the study are anticipated in Q1 2021.

The Phase 2b multi-center study is designed to investigate the safety, tolerability, and efficacy of MS1819 (2.2 and 4.4 gram doses in enteric capsules) in a head-to-head comparison against the current porcine enzyme replacement therapy (PERT) standard of care. The primary efficacy endpoint is the coefficient of fat absorption (CFA). Planned enrollment is expected to include approximately 30 CF patients in the U.S. and Poland.

"We are excited to be a clinical trial site in the OPTION 2 study and to continue participating in AzurRx's ongoing efforts to find a safe non-porcine enzyme replacement therapy for our patients with cystic fibrosis," commented Steven R. Boas, M.D., FAAP, FACCP, President and CEO, The Cystic Fibrosis Institute. "I believe that the clinical protocol provides optimal safety conditions for participating patients and care providers, given concerns about COVID-19, and I am confident that we will be able to meet our enrollment targets for the trial in a timely fashion."

Dr. James Pennington, Chief Medical Officer of AzurRx, added, "We have designed the OPTION 2 efficacy study based on the results from the OPTION bridging dose safety study, and are optimistic that the enteric capsules delayed-release delivery of the 2.2g and 4.4g doses of MS1819 will enable us to achieve our primary and secondary efficacy endpoints in this study. We thank our clinical collaborators and participating patients for their support of OPTION 2 and look forward to our clinical results."

"The initiation of the OPTION 2 trial is a major clinical milestone for the Company," said James Sapirstein, Chief Executive Officer of AzurRx. "Previous clinical trials have demonstrated that MS1819 is safe and well tolerated. With this trial, we are moving ahead to determine the optimal dose for MS1819 in preparation for a pivotal Phase 3 study. We look forward to the successful completion of the trial and to reporting our topline results of OPTION 2 in Q1 2021."

Phase 2 OPTION 2 Trial Design

The Phase 2b OPTION 2 multi-center trial is designed to investigate the safety, tolerability and efficacy of MS1819 (2.2 and 4.4 gram doses in enteric capsules) in a head-to-head comparison versus the current standard of care, porcine pancreatic enzyme replacement therapy pills. The OPTION 2 trial will be an open-label, crossover study, conducted in 15 sites in the U.S. and Europe. A total of 30 CF patients 18 years or older will be enrolled. MS1819 will be administered in enteric capsules to provide gastric protection and allow optimal delivery of enzyme to the duodenum. Patients will first be randomized into two cohorts: to either the MS1819 arm, where they receive a 2.2 gram daily oral dose of MS1819 for three weeks; or to the PERT arm, where they receive their pre-study dose of PERT pills for three weeks. After three weeks, stools will be collected for analysis of coefficient of fat absorption. Patients will then be crossed over for another three weeks of the alternative treatment. After three weeks of cross-over therapy, stools will again be collected for analysis of CFA. A parallel group of patients will be randomized and studied in the same fashion, using a 4.4 gram daily dose of MS1819. All patients will be followed for an additional two weeks after completing both crossover treatments for post study safety observation. Patients will be assessed using descriptive methods for efficacy, comparing CFA between MS1819 and PERT arms, and for safety.