MS1819 Clinical Trials Advancing for CF


According to BioMedWire, biopharmaceutical company AzurRx BioPharma (“AzurRx”) decided, in mid-November, to advance a series of clinical trials evaluating MS1819 for patients with cystic fibrosis. In particular, the clinical trials are analyzing the safety, efficacy, and tolerability of MS1819 for severe cystic fibrosis-related exocrine pancreatic insufficiency (EPI).


The Cystic Fibrosis Foundation (CFF) explains that MS1819 is:

a non-porcine (not pig-derived) enzyme for individuals with CF who have exocrine pancreatic insufficiency. It is a man-made version of a lipase enzyme taken from the yeast Yarrowia lipolytica.

MS1819 is orally administered. The therapy is designed to enhance fat absorption, thus improving patient outcomes. Prior clinical trials on MS1819, such as this one reported on in 2017, highlight the drug’s favorable safety profile. Although the prior trial was performed on patients with EPI relating to chronic pancreatitis as opposed to cystic fibrosis, MS1819 does seem to be fairly well-tolerated.

At this time, AzurRx is running two Phase 2 clinical trials on MS1819. The first is a Phase 2b trial evaluating MS1819 as a monotherapy. Additionally, the company is running a Phase 2 trial evaluating MS1819 in conjunction with the current standard-of-care, called porcine pancreatic enzyme replacement therapy (PERT). Data from both clinical trials should be available at some point in 2021.

Cystic Fibrosis (CF)

An estimated 70,000 people worldwide have cystic fibrosis (CF), a progressive genetic disorder characterized by digestive and respiratory damage. Of those 70,000, about half are believed to be Americans. Cystic fibrosis occurs in around 1 in every 2500-3500 Caucasian births within the United States. Though the disorder does occur in other ethnicities, it is much less prevalent.

Normally, our CFTR gene plays a role in salt movement. However, CFTR gene mutations inhibit this ability. As a result, rather than having healthy and slippery mucus, people with CF have thick, sticky mucus. When this mucus accumulates in airways, it traps bacteria, causes infections and inflammations, and leads to lung damage. Additionally, this mucus builds up within the digestive system. By inhibiting digestive enzymes from being released, many patients are unable to absorb important nutrients. CF symptoms include:

  • Exercise intolerance
  • Shortness of breath/difficulty breathing
  • Infertility in men
  • Salty skin
  • Constipation
  • Chronic coughing or wheezing
  • Frequent lung infections
  • Fatty stools
  • Stuffy nose
  • Difficulty gaining weight

Exocrine Pancreatic Insufficiency (EPI)

According to WebMD, exocrine pancreatic insufficiency (EPI):

causes problems in how you digest food. Your pancreas doesn’t make enough of the enzymes that your body needs to break down and absorb nutrients.

The National Pancreas Foundation further explains that EPI is usually associated with another condition that affects the pancreas, such as chronic pancreatitis (CP) or cystic fibrosis (CF). Patients with CF already have difficulty with nutrient absorption. EPI also prevents the breakdown of food and proper nutrient digestion. Thus, the National Pancreas Foundation states:

It’s especially important that people with EPI eat a nutrient-rich diet—along with taking pancreatic enzyme replacement therapy with every meal and snack—since EPI interferes with nutrient absorption.

Jessica Lynn

Jessica Lynn

Jessica Lynn has an educational background in writing and marketing. She firmly believes in the power of writing in amplifying voices, and looks forward to doing so for the rare disease community.

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