This follows the announcement of the latest data from the RESULT Phase 3 clinical trial (NCT03051490), which failed to demonstrate Sollpura’s effectiveness. The treatment was unable to significantly improve fat absorption, which was the primary goal of the trial.
The company is planning to evaluate all trial data. However, it will suspend Sollpura’s ongoing clinical studies, which include the extension study of the RESULT trial, the SIMPLICITY study (NCT02734810), and the EASY trial (NCT02823964).
“We are greatly disappointed by the findings of the RESULT study,” Craig Thompson, president and CEO of Anthera Pharmaceuticals, said in a press release. “We would like to extend our deepest gratitude to the patients and their families, study investigators, and the cystic fibrosis community for the support they have provided in the clinical development of Sollpura.”
Thick mucus overproduction is a characteristic symptom of cystic fibrosis (CF). This can alter the functioning of the pancreas, making it unable to discharge the enzymes the gut needs for a normal digestion — a condition called pancreatic insufficiency. To overcome this, CF patients can receive pancreatic enzyme replacement therapies (PERT) to help ensure that nutrients are effectively absorbed from food.
PERTs, however, are usually derived from pigs, which can represent serious health risks.
The unique formulation of Sollpura is based on non-pig-derived lipase, protease, and amylase enzymes. It was designed to help digestion while reducing the risk of patients developing a pig-related disease or conditions such as gout and kidney impairment.
This time, researchers adjusted dose levels and frequency to meet individual participants’ needs. The trial design was approved by the Cystic Fibrosis Foundation’s Therapeutics Development Network and the European Cystic Fibrosis Society Clinical Trial Network.
A total of 140 CF patients were recruited in several clinical sites across North America, Europe, and Israel. They were randomized to receive Pancreaze in a mean dose of 6,527 units/kg per day, or Sollpura in a starting dose approximately 25 percent higher than their pre-study PERT dose.
During the trial, 59 percent of the participants needed further Sollpura dose adjustment, yielding a mean dose of 8,673 units/kg per day.
Although some of the patients who received Sollpura maintained or improved fat absorption compared to baseline levels, more patients experienced a worsening. As for nitrogen absorption, the study’s secondary outcome measure of treatment effectiveness, the reported values were similar to those observed with standard Pancreaze.