AzurRx BioPharma announced it has started a Phase 2 clinical trial testing the safety, tolerability, and efficacy of MS1819 Spray Dried (MS1819-SD) therapy in cystic fibrosis (CF) patients with exocrine pancreatic insufficiency (EPI).
The trial was approved in October by the U.S. Food and Drug Administration.
Many CF patients develop EPI as a result of mucus accumulation and subsequent blockage of pancreatic ducts. Because of the limited release of digestive enzymes, these patients have problems digesting and absorbing fats, carbohydrates, and proteins. The current standard-of-care treatment is a porcine pancreatic enzyme replacement therapy (PERT), which is a mixture of pancreatic enzymes derived from pigs.
MS1819-SD is a yeast-derived (Yarrowia lipolytica) investigational treatment, designed to restore the limited fat digestion of patients with EPI.
The new trial, called OPTION (NCT03746483), will compare MS1819-SD against the current porcine PERT in CF patients with EPI. The six-week trial will assess fat digestion by measuring patients’ coefficient fat absorption (CFA), a standard test that examines fat absorption.
OPTION is a so-called cross-over study, which means that participants receive both the test treatment and the reference treatment in two separate periods (with a treatment washout between the two periods).
Enrollment is aimed at approximately 30 CF patients, ages 18 and older. Study completion and initial results are expected in 2019.
“We are very appreciative of the input provided by the FDA in designing our cross-over study and the positive feedback from the Cystic Fibrosis Foundation’s Therapeutics Development Network, which enables us to access its 89 accredited U.S. care centers,” James Pennington, MD, chief medical officer of AzurRx, said in a press release.
The Cystic Fibrosis Institute is one of the sites involved in the OPTION trial.
“We are delighted to serve as the first site initiated in the OPTION study,” said Steven R. Boas, MD, president and CEO of the institute. “I believe that MS1819-SD has the potential to improve the lives of patients by reducing their pill burden, which can be as high as 40 digestive enzyme pills per day. Based on my 27 years of experience in treating CF patients, chronic digestive issues are often cited as one of their top concerns, which is also supported by the literature. MS1819-SD may offer a promising solution for this important unmet need.”
A previous Phase 2a study testing MS1819-SD in patients with chronic pancreatitis, another cause of EPI, indicated that the treatment was safe and well-tolerated. In addition, although the study was not powered to test efficacy, MS1819-SD treatment resulted in a significant (21.8%) increase of CFA over initial values.
“The initiation of the OPTION study of MS1819-SD in patients with CF is a significant milestone for AzurRx and follows our successful Phase 2 EPI trial in patients with chronic pancreatitis, which demonstrated both safety and statistically significant efficacy,” said Thijs Spoor, CEO of AzurRx. “We look forward to this trial and to demonstrating it can make a meaningful difference for patients.”