First Sites Selected for Enhanced Adrulipase Formulation Phase 2 Trial
Reformulation packs the fat-digesting enzyme in micro-granules to reach small intestine
First Wave Biopharma has selected the initial sites for the Phase 2 clinical trial of a new adrulipase formulation to treat exocrine pancreatic insufficiency (EPI) in people with cystic fibrosis (CF).
“We believe that our reformulation of adrulipase offers the potential to provide a dramatically improved treatment option for EPI patients,” said James Sapirstein, president and CEO of First Wave BioPharma, in a company press release. “We look forward to proceeding with this important trial.”
Collaborating with the contract research organization Rho, the First Wave secured the cooperation of Steven Boas, MD, and the Cystic Fibrosis Institute and Cystic Fibrosis Center of Chicago; Daniel Layish, MD, and the Central Florida Pulmonary Group in Orlando; and Craig Nakamura, MD, and Children’s Lung Specialists in Las Vegas.
EPI occurs when the pancreas isn’t able to release sufficient enzymes for digestion, particularly to break down fats, resulting in food not being digested properly. It commonly occurs in people with CF, due to mucus accumulating in the pancreas, which prevents the organ from secreting the enzymes.
There are more than 30,000 patients with EPI caused by CF in the U.S., according to the Cystic Fibrosis Foundation, and about 90,000 patients with EPI due to chronic pancreatitis, according to the National Pancreas Foundation. Chronic pancreatitis occurs when the pancreas is damaged by inflammation.
The current standard EPI treatment is pancreatic enzyme replacement therapy (PERT), wherein enzymes derived from pigs are used to aid digestion.
The new formulation packs the adrulipase enzyme (a fat-digesting enzyme, or lipase) in micro-granules to safely get to the small intestine — traversing the acidic environment of the stomach — and help break down fats. Adrulipase is derived from yeast as a non-animal derived therapy for EPI to be administered orally.
According to First Wave, it has the potential to reduce the daily pill burden associated with currently available treatments for EPI associated with CF and chronic pancreatitis.
“Currently available therapies, such as PERT, are able to manage the symptoms of the disease but require patients to take as many as 40 capsules per day. This ‘pill burden’ presents a substantial challenge for EPI patients,” Boas said. “We are hopeful that this enhanced formulation of adrulipase will be able to address the symptoms of EPI while also providing a more convenient dosing option for patients.”
First Wave recently requested authorization from the U.S. Food and Drug Administration to start the Phase 2 trial. The company expects to report top-line data from the trial by mid-2023.
Last month, the company met with the researchers at the North American Cystic Fibrosis Conference in Philadelphia, who concluded that the new adrulipase formulation could overcome the challenges associated with PERT therapy.