First Wave, Rho Team Up to Test New Adrulipase Treatment for CF
Phase 2 trial of new formulation expected to start before year's end
First Wave BioPharma is teaming up with Rho for a Phase 2 clinical trial to test their new adrulipase formulation, designed as a treatment for exocrine pancreatic insufficiency (EPI) in people with cystic fibrosis (CF).
“We are eager to evaluate the potential of our new formulation in the proof-of-concept Phase 2 clinical trial, which we expect to initiate before year-end,” James Sapirstein, First Wave’s president and CEO, said in a press release.
First Wave is a clinical-stage biopharmaceutical company focused on developing new treatments for gastrointestinal diseases — the symptoms of which are common among CF patients. Rho is a contract research organization (CRO) that offers drug development services.
Adrulipase alfa is a lipase or fat-digestive enzyme derived from yeast. It is designed to break up fat molecules in the digestive tract of patients and allow their absorption as nutrients. The experimental therapy is administered orally.
Testing adrulipase alfa for EPI in CF
First Wave is advancing two Phase 2 clinical programs for adrulipase as a treatment for EPI in patients with CF, and among those with chronic pancreatitis, a disorder in which the pancreas is damaged from inflammation.
EPI is characterized by a blockage of the release of digestive enzymes — from the pancreas into the intestines — due to a thick buildup of mucus, a hallmark of CF. That leads to improper food digestion, particularly of fats. According to First Wave, deficiencies of these enzymes can cause diarrhea and weight loss in patients.
The condition is common among the CF patient population.
First Wave recently developed a new optimized formulation of adrulipase. This formulation ensures that the therapy is packed in acid-resistant micro-granules, protecting it from the stomach’s acidity. That’s followed by a fast release in the small intestine, where adrulipase is expected to mix with food to exert its therapeutic effects.
“We are preparing to initiate the planned Phase 2 trial of our new microgranule delivery formulation for adrulipase and are pleased to have Rho as our CRO partner given the company’s proven expertise and experience managing our prior adrulipase clinical programs,” Sapirstein said, adding, “Research … indicates the new adrulipase formulation should deliver the drug in the intended area of the gastrointestinal tract.”
In addition, preliminary data suggest that the new formulation has the potential to significantly reduce the number of pills needed to attain the desired therapeutic effect.
With the current porcine pancreatic enzyme replacement therapy (PERT) option — a cocktail of pig-derived versions of the missing enzymes — EPI patients can take as many as 40 capsules daily, according to First Wave. The company cites this as a substantial burden for patients.
Through the agreement with Rho, First Wave is now expecting to launch the Phase 2 trial for adrulipase alfa before the close of 2022.
“We are delighted to continue our work with First Wave BioPharma and are eager to use the expertise we’ve gained from managing prior clinical programs involving adrulipase,” said Jack Modell, MD, Rho’s chief medical officer.