First Wave BioPharma Announces First Patient Dosed in Phase 2 SPAN Clinical Trial of Enhanced Adrulipase Formulation

First Wave BioPharma, Inc. recently announced that the first patient was dosed in the Phase 2 SPAN clinical trial investigating an enhanced enteric microgranule delivery formulation of adrulipase for the treatment of exocrine pancreatic insufficiency (EPI) in patients with cystic fibrosis (CF). Topline results from the study are anticipated by mid-2023.

The Phase 2 multi-center clinical trial (NCT05719311) is designed to investigate the safety, tolerability and efficacy of an enteric microgranule delivery formulation for adrulipase in a titrated dose-escalation study involving an estimated twelve (12) patients. The primary efficacy endpoint is the coefficient of fat absorption (CFA), with secondary endpoints of stool weight, signs and symptoms of malabsorption and coefficient of nitrogen absorption (CNA). Three clinical trial sites in the US will be participating in the trial.

“Dosing of the first patient in the Phase 2 SPAN clinical trial is a significant milestone for First Wave BioPharma and a key inflection point toward our goal of developing adrulipase to one day replace porcine-derived pancreatic enzyme replacement therapy (PERT) as the standard of care for EPI associated with CF and chronic pancreatitis,” said James Sapirstein, President and CEO of First Wave BioPharma. “With dosing initiated, we now look forward to a topline data readout from the Phase 2 SPAN trial by mid-2023, which we hope will confirm the favorable results produced in the in vitro studies of the enteric microgranule delivery formulation for adrulipase.”

“EPI is a debilitating gastrointestinal condition common to patients with CF that can result in numerous, life-altering complications, including malnutrition,” said Steven Boas, MD, Founder of the Cystic Fibrosis Institute and Cystic Fibrosis Center of Chicago. “A safe and effective therapy that allows individuals with cystic fibrosis to gain control over EPI, while diminishing the daily pill burden required with PERT, is an important medical need that would have a profound impact on the lives of patients.”

The Phase 2b SPAN trial is designed to investigate the safety, tolerability and efficacy of a new enteric microgranulation formulation of adrulipase. The SPAN trial is an open-label study that will be conducted at three sites in the U.S. A total of 12 cystic fibrosis patients, 18 years or older are expected to be enrolled. The trial design employs a dose titration strategy. Patients will be screened at baseline to ensure that they have a coefficient of fat absorption (CFA) of at least 80%. Eligible patients will then be switched from their commercial enzyme product to adrulipase. Each patient will be started on a low dose of adrulipase. If the patient is not clinically controlled, the patient will be switched to a medium dose, and if not controlled on this dose, the patient will be advanced to a high dose. The titrations will be carried out over a three-week period, after which a CFA will be obtained. End-of-study CFAs will be compared to the baseline CFAs in a descriptive fashion. A post-treatment safety visit will be conducted one week after completing the treatment period.

Adrulipase is a recombinant lipase enzyme administered as an oral, non-systemic biologic capsule for the treatment of exocrine pancreatic insufficiency (EPI) associated with cystic fibrosis (CF) and chronic pancreatitis (CP). Adrulipase is derived from the Yarrowia lipolytica yeast lipase and is designed to break up fat molecules in the digestive tract of EPI patients so that they can be absorbed as nutrients. EPI is a condition characterized by deficiency of the exocrine pancreatic enzymes, resulting in a patient’s inability to digest food properly, or maldigestion. The deficiency in this enzyme can be responsible for greasy diarrhea, fecal urge and weight loss. There are more than 30,000 patients in the U.S. with EPI caused by cystic fibrosis according to the Cystic Fibrosis Foundation and approximately 90,000 patients in the U.S. with EPI caused by chronic pancreatitis according to the National Pancreas Foundation.

First Wave BioPharma is a clinical-stage biopharmaceutical company specializing in the development of targeted, non-systemic therapies for gastrointestinal (GI) diseases. The company is currently advancing a therapeutic development pipeline with multiple clinical stage programs built around its two proprietary technologies – the biologic adrulipase, a recombinant lipase enzyme designed to enable the digestion of fats and other nutrients, and niclosamide, an oral small molecule with anti-inflammatory properties. First Wave is advancing two Phase 2 clinical programs built around adrulipase for the treatment of exocrine pancreatic insufficiency (FW-EPI) in patients with cystic fibrosis (CF) and chronic pancreatitis (CP). In developing adrulipase, First Wave is seeking to provide CF and CP patients with a safe and effective therapy to control EPI that is non-animal derived and offers the potential to dramatically reduce their daily pill burden. The company is also advancing multiple programs involving niclosamide, including FW-UP for ulcerative proctitis and ulcerative proctosigmoiditis, FW-UC for ulcerative colitis, and FW-CD for Crohn’s disease. First Wave BioPharma is headquartered in Boca Raton, FL. For more information, visit www.firstwavebio.com.