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AzurRx BioPharma Announces Promising Results from Trial Testing MS1819 for EPI in CF Patients


The investigational therapy MS1819 showed positive safety results in a Phase 2 clinical trial for the treatment of exocrine pancreatic insufficiency (EPI) in people with cystic fibrosis (CF), AzurRx BioPharma announced.

In people with CF, EPI can occur when mucus builds up and blocks the ducts of the pancreas. This can prevent important enzymes from getting into the digestive tract, making it difficult for those affected to adequately absorb nutrients from their food.

The current standard care for EPI in CF is porcine enzyme replacement therapies (PERT). As its name suggests, this involves taking supplemental versions of the missing pancreatic enzymes that are derived from pigs.

Like PERT, MS1819 is an enzyme-based therapy; but instead of using pig enzymes, it uses enzymes from the yeast Yarrowia lipolytica.

The Phase 2 OPTION clinical trial (NCT03746483) compared PERT and MS1819 in 32 adults with CF-induced EPI. In the first three weeks of the trial, participants were assigned randomly to treatment with either PERT or MS1819; in the next three weeks, participants crossed over and received the other treatment.

Participants were followed for an additional two weeks after the six-week-long treatment period. Stool samples were collected in order to assess nutrient absorption.

The trial was designed to evaluate the safety profile of MS1819. Results showed that the therapy’s safety profile was “excellent,” according to the company, as there were no serious adverse side effects.

The study was not powered for efficacy — that is, for statistical reasons, the sample size is too small to compare the effectiveness of the treatments with mathematical certainty.

Nonetheless, preliminary efficacy data suggested that the coefficient of fat absorption (CFA, the percentage of dietary fat that gets absorbed) for MS1819 was 56% and for PERT 86%, which is a meaningful efficacy result. AzurRx noted that for about half of the participants, MS1819 resulted in a CFA that was comparable to PERT.

A secondary efficacy goal, coefficient of nitrogen absorption (CNA), was comparably high for both treatments — 93% in MS1819 vs. 97% in PERT.

“We are thrilled to have seen such favorable safety and meaningful efficacy data in this Phase 2 study,” Jim Pennington, MD, the chief medical officer of AzurRx, said in a press release.

“Importantly, the data were consistent and confirm results seen in prior clinical studies. We are grateful to all the investigators who worked diligently to bring this study to completion on time, and also want to thank the patients and their caregivers for taking the time to participate in the trial. We are eager to move forward with what we consider a logical and promising next trial to increase the dose for CF patients,” Pennington said.

AzurRx is planning to meet with the U.S. Food and Drug Administration (FDA) before the end of this year to discuss a Phase 2b/3 clinical trial to further test higher doses and/or different delivery methods of MS1819.

“We look forward to meeting with the FDA to discuss these results and our next steps. Clearly, these data are exciting for patients, who currently face debilitating symptoms, and we look to advancing to market a therapy that has the potential to improve the quality of life in these patients,” said Thijs Spoor, CEO of AzurRx.

Michael Konstan, MD, professor at Case Western Reserve University School of Medicine and principal investigator in the OPTION trial, said “With these data showing MS1819 to be safe and to have the potential to support fat absorption, we have considerable reason to be optimistic for the next steps in non-porcine enzyme development.”

Marisa Wexler MS

Marisa, a science writer, holds an MS in Cellular and Molecular Pathology from the University of Pittsburgh, where she studied novel genetic drivers of ovarian cancer. She specializes in cancer biology, immunology, and genetics. Marisa began working with BioNews in 2018, and has written about science and health for SelfHacked and the Genetics Society of America. She also writes/composes musicals and coaches the University of Pittsburgh fencing club.

Fact Checked By

Patricia Silva, PhD

Patrícia holds her PhD in Medical Microbiology and Infectious Diseases from the Leiden University Medical Center in Leiden, The Netherlands. She has studied Applied Biology at Universidade do Minho and was a postdoctoral research fellow at Instituto de Medicina Molecular in Lisbon, Portugal. Her work has been focused on molecular genetic traits of infectious agents such as viruses and parasites.

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