Patient Screening to Begin for Phase 2 Trial of Adrulipase Formulation
The trial will replace PERT with new EPI treatment
A Phase 2 clinical trial of a new optimized formulation of adrulipase, a yeast-derived enzyme for treating exocrine pancreatic insufficiency (EPI) in adults with cystic fibrosis (CF), is about to start.
First Wave BioPharma, its developer, anticipates patient screening will start early this month, with top-line results expected by mid-year. The Phase 2 SPAN trial expects to enroll 12 CF patients, 18 or older, who will be recruited at three sites in the U.S.
“We are very pleased to announce the initiation of the Phase 2 clinical trial investigating our new formulation of adrulipase as a potential treatment for EPI associated with cystic fibrosis and chronic pancreatitis,” said James Sapirstein, First Wave’s president and CEO, in a press release.
EPI is common in CF, affecting more than 30.000 people in the U.S., according to the Cystic Fibrosis Foundation.
A feature of CF is the accumulation of thick mucus, which prevents the release of enzymes needed for digestion, particularly for breaking down fats, when it occurs in the pancreas, This leads to EPI, which is also common in chronic pancreatitis when the pancreas is damaged by inflammation.
The current standard EPI treatment is pancreatic enzyme replacement therapy (PERT), or enzyme supplements typically from pigs.
Adrulipase is a lipase (fat-digesting enzyme) to be taken orally. The new formulation encloses the enzyme in micro-granules to protect it from the stomach’s acidic environment and safely reach the small intestine to break down fats.
Presently, as many as 40 capsules a day may be required to get the desired therapeutic effect. The new formulation is expected to reduce that number.
“Preclinical research has demonstrated the new adrulipase formulation is able to deliver the drug in the intended area of the gastrointestinal tract where it can provide the desired therapeutic effect,” Sapiristein said. “This Phase 2 clinical trial is designed to test that capability as a key step in our goal to provide patients with a more effective and convenient therapeutic option for EPI associated with cystic fibrosis and chronic pancreatitis.”
In November, First Wave submitted an investigational new drug application with the U.S. Food and Drug Administration (FDA) to study the new formulation. No additional comments were raised during the 60-day review period.
SPAN is an open-label dose escalation study to assess the new formulation’s safety, tolerability and effectiveness.
Its primary efficacy measure is the coefficient of fat absorption (CFA, a measure of the percentage of absorbed fat in the diet). Secondary measures include stool weight, symptoms of malabsorption, and the coefficient of nitrogen absorption (CNA), a marker of protein absorption.
The study will recruit participants with a CFA of at least 80%. A CFA above 80% is considered the minimum for a therapeutic effect in CF patients with EPI and chronic pancreatitis.
Participants will be switched from their standard PERT to the new adrulipase formulation, starting on a low dose that gradually increases over three weeks if the patient isn’t clinically controlled and CFA will be compared to the study’s start. The therapy’s safety will be evaluated a week after the treatment period ends.