AzurRx BioPharma has reached its patient enrollment goal in the ongoing Phase 2 trial testing the effectiveness of MS1819-SD, the company’s investigational therapy for the treatment of patients with exocrine pancreatic insufficiency (EPI) associated with cystic fibrosis (CF).
MS1819-SD is a recombinant (artificial) form of an enzyme found in yeast (Yarrowia lipolytica) that breaks down fatty molecules, called lipase. The purpose of the therapy is to use this enzyme to help patients who lack certain pancreatic enzymes digest fatty molecules found in food.
In October 2018, the U.S. Food and Drug Administration (FDA) approved the company’s request to launch a Phase 2 clinical trial to assess the effects of MS1819-SD in EPI patients due to CF.
The multi-center, randomized, open-label, Phase 2 OPTION trial (NCT03746483) was designed to assess the safety, tolerability, and efficacy of MS1819-SD compared with standard treatment with porcine pancreatic enzyme replacement therapy (PERT).
In OPTION there will be two crossover periods, in which patients initially treated with MS1819-SD will switch to PERT, and vice versa.
The trial’s primary outcomes include assessing the safety and efficacy of MS1819-SD after six weeks of treatment by monitoring treatment-related adverse events, and by measuring the patients’ coefficient of fat absorption (CFA). Secondary outcomes will include measuring stool weight and looking for signs and symptoms of food malabsorption, both at week 6. AzurRx launched the trial in December 2018.
“AzurRx is very pleased to report that we have reached our enrollment target of 30–35 patients that was established when the OPTION study was initiated in December 2018. We are appreciative of the interest from the scientific community to work with patients in developing this drug, and the interest from the investment community to fund the company as we have the resources to complete the study on schedule,” Thijs Spoor, chief executive officer of AzurRx, said in a press release.
OPTION is underway at sites across the U.S. and Poland. According to AzurRx, top-line data from the study should be available this summer.