Dosing complete in SPAN Phase 2 study of adrulipase
New formulation being tested as treatment for exocrine pancreatic insufficiency in CF
First Wave BioPharma has finished dosing in its Phase 2 clinical study, SPAN, in which a new adrulipase formulation is being tested as a potential exocrine pancreatic insufficiency (EPI) treatment in people with cystic fibrosis (CF).
With the last patient dosed, the company remains on track for next month’s reporting of top-line data, which primarily will provide an overview of how safe the treatment is and whether it is effective in its ability to help break down (digest) fats from food.
“We remain positioned to report topline data in July, which is expected to provide an initial indication of the new formulation’s ability to release adrulipase in the small intestine where the drug is designed to mix with food and deliver its therapeutic benefit,” James Sapirstein, First Wave’s president and CEO, said in a press release.
CF causes thick, sticky mucus to build up in the lungs and clog the airways, making it difficult to breathe. As it does in the lungs, thick mucus also builds up in the pancreas, where it blocks the release of enzymes needed for digestion. This can result in EPI.
Many patients need pancreatic enzyme replacement therapy (PERT) to supply the needed enzymes and release them in the small intestine, where they help digest food to help the body function properly and maintain health. Currently available PERTs typically are derived from pigs.
Adrulipase is a fat-digesting enzyme derived from yeast that’s designed to improve the breakdown of fats in the small intestine. Its use is expected to reduce the number of pills patients need to take each day.
“Ultimately our goal is to provide patients with a safe and effective therapy that allows individuals with CF to gain control over EPI, while diminishing the daily pill burden,” Sapirstein said.
The SPAN clinical trial
The open-label study dosed 13 adults with CF at three sites in the U.S. Each patient is given a low dose of oral adrulipase for three weeks. If the patient does not respond well, adrulipase is given at a medium dose, which is increased to a high dose if needed.
“Dosing the final patient is a key milestone in the Phase 2 SPAN clinical trial investigating our enhanced enteric [intestinal] microgranule delivery formulation of adrulipase for the treatment of EPI in CF patients,” Sapirstein said.
The study’s primary goals (outcome measures) are safety and change in coefficient of fat absorption (CFA) after the three-week treatment period. CFA is a measure of how well the body is absorbing fats from food.
Secondary outcomes measures include stool weight, signs and symptoms of poor nutrient absorption (malabsorption), and coefficient of nitrogen absorption, which is an assessment of how well the body is absorbing proteins.
FirstWave also recently announced the publication of two patent applications by both the U.S. Patent and Trademark Office and the International Bureau of the World International Patent Organization. The patents cover adrulipase’s composition of matter and methods of use, and they were published under the Patent Cooperation Treaty, which includes more than 150 countries worldwide.
“The patent publications for our enhanced enteric microgranule delivery formulation of adrulipase and its use for the treatment of malabsorption highlight First Wave’s ability to introduce important drug innovations for the treatment of underserved disease indications,” Sapirstein said in another press release.