A pancreatic enzyme replacement product that is not derived from pigs — Sollpura (liprotamase) — was as good at maintaining the height and weight of cystic fibrosis (CF) patients as a pig-derived product, according to a clinical trial.
The information came from the extension period of Anthera’s Phase 3 clinical trial, called SOLUTION (NCT02279498). It compared Sollpura and Pancreaze (pancrelipase), a pig-derived pancreatic enzyme replacement therapy (PERT). The objective was to prove that Sollpura is as good as the already approved Pancreaze.
Sollpura is being developed for patients with CF or other diseases that have caused low digestive enzyme levels, a condition known as exocrine pancreatic insufficiency.
Pancreatic insufficiency is common in CF patients. It results from the thick mucus associated with the disease blocking the discharge of pancreatic enzymes needed for digestion. PERT helps ensure that the body can absorb nutrients from food.
Unlike commercially available PERT, a tablet designed to disintegrate only after it leaves the stomach, Sollpura is not derived from pigs. It is made up of a powder containing three digestive enzymes: crystalline lipase, crystalline protease, and amorphous amylase.
To study Sollpura’s effectiveness, researchers recruited CF patients older than 7 who had exocrine pancreatic insufficiency and were responding well to current PERT therapy.
The extension, which ran for 13 weeks, started right after the six-week trial.
Both Sollpura and Pancreaze maintained patients’ height and weight, the extension study showed. In fact, there was a slight increase in patients’ height.
In patients younger than 17, both treatments maintained or led to a slight increase in height and weight. Since height increased more than weight, patients experienced a slight drop in body mass index (BMI) during the study.
“We are pleased to see the continued tolerability and maintenance of weight and height in all of the Sollpura-treated cystic fibrosis patients with EPI [exocrine pancreatic insufficiency] during the Extension Period and, in particular, increases in weight and height in patients less than 17 years of age,” William Shanahan, Anthera’s chief medical officer, said in a press release.
“The 20-week data provides support that Sollpura may offer patients an alternative to existing, porcine[pig]-based pancreatic enzyme replacement therapies,” Shanahan added.
Forty percent of the patients treated with Sollpura experienced adverse events. Among Pancreaze-treated patients, the number was slightly higher, 54 percent. Researchers ascribed the difference to a lower rate of infection-triggered lung exacerbations in the Sollpura group — 9.2%, versus 20.6% in those treated with Pancreaze.
Similarly, 9.3 percent of Sollpura-treated patients experienced respiratory disorders, compared with 15.9 percent in the Pancreaze group. Mild, moderate, and severe adverse events were all less common in the Sollpura-treated group.
Anthera acquired Sollpura from Eli Lilly in 2014, and started the SOLUTION trial in late 2015. In December 2016, it became apparent that the trial had missed its primary objective of lower fat absorption than Pancreaze by a narrow margin. Sollpura matched Pancrease in nitrogen absorption, however.
At the time, the company said the fat-absorption failure might have resulted from dose escalation difficulties caused by the trial design. To obtain regulatory approved, Anthera needs to conduct another trial demonstrating that Sollpura is a good as Pancreaze.
The company is about to start a new Phase 3 trial, called RESULT (NCT03051490). It expects to deliver results by late 2017 or early 2018.