AzurRx BioPharma, Inc. has recently informed that the company has dosed the first patients in its Phase II OPTION clinical study aimed at investigating MS1819-SD in cystic fibrosis (CF) patients who experience exocrine pancreatic insufficiency (EPI).

Supposedly, the multi-center Phase II trial has been designed to explore the efficacy, tolerability and safety of MS1819-SD, comparing it directly with the existing porcine enzyme replacement therapy (PERT) care standard. Around 30 CF patients are planned to be enrolled in the study, with top-line results being expected in mid-2019.

According to AzurRx BioPharma, the OPTION study primarily involves a six week long non-inferiority and coefficient of fat absorption (CFA) evaluation to compare MS1819-SD with the standard of care PERT in patients with EPI resulting from cystic fibrosis. Its cross-over study design would be able to leverage input from the CF community as well as from the U.S. Food and Drug Administration. The nearly 30 patients to be enrolled in the trial would be aged 18 years or above, the company stated.

Michael W. Konstan, M.D., Case Western Reserve University School of Medicine’s Professor of Population & Quantitative Health Sciences as well as Pediatrics, who is also the Vice Dean for its Translational Research, mentioned that for a long time the CF community has identified the unmet need regarding a non-porcine based pancreatic enzyme option, as an alternative to current treatments of EPI in CF patients.

The company said that in a recently confirmed Phase II trial in the chronic pancreatitis setting, a good tolerability and favorable safety profile was exhibited by MS1819-SD. Besides, the highest studied dose had shown a statistically significant improvement in CFA of 21.8%.

AzurRx’s CEO, Thijs Spoor commented that dosing the first patients in the company’s OPTION trial of MS1819-SD in CF patients is representative of the firm’s remarkable achievement, demonstrating that it is on track to finish the Phase 2 study by mid-2019, the previously informed timeline.