Interim data from an ongoing Phase 2a clinical trial indicate that the investigational therapy MS1819-SD may provide therapeutic benefits for patients with cystic fibrosis (CF) who have exocrine pancreatic insufficiency (EPI), a digestive disorder.
The trial (ACTRN12616000962437) is being conducted in patients with chronic pancreatitis (CP) who have EPI, but the drug is being developed for both CP and CF patients with EPI, according to a news release.
MS1819 is an engineered recombinant enzyme derived from the yeast Yarrowia Lipolytica. Developed by AzurRx BioPharma in collaboration with Laboratoires Mayoly Spindler, MS1819-SD is an oral capsule engineered to help overcome nutrient malabsorption in chronic pancreatitis and CF patients caused by chronic pancreatic inflammation and impaired discharge of pancreatic enzymes needed for digestion.
Previous studies have demonstrated that MS1819 is a potent lipase that can actively metabolize long-chain fats while eliminating the pork viral contamination risk that other existing drugs have.
The ongoing Phase 2a study is being conducted in Australia and New Zealand and is planned to enroll about 12 patients with EPI due to chronic pancreatitis. This open-label trial will evaluate the effectiveness of increasing doses of MS1819-SD based on the analysis of the coefficient of fat absorption (CFA). Adverse effects related to the treatment will also be evaluated, particularly the prevalence of digestive symptoms or immunoallergic effects.
Because this is an open-label study, all participants will receive the study drug MS1819. Each patient will complete four treatment periods in which they will receive one of the tested doses: 280 mg, 560 mg, 1,120 mg, or 2,240 mg per day, for a maximum treatment time of 60 days.
Results from the first six patients treated with MS1819-SD showed that the highest dose tested induced a 21% improvement on CFA values compared to baseline. Researchers also observed that treatment was able to induce a maximal absolute CFA of about 57%.
Secondary endpoints evaluated during the trial, such as the number of daily evacuations and weight of stool, also showed positive trends that were in accordance with the CFA results.
No significant adverse events associated with the treatment were reported, nor were alterations to patients’ nutritional status. Following the positive treatment effects seen so far, fecal nitrogen assessments also showed favorable trends.
“We are encouraged by these Phase 2a data,” Thijs Spoor, CEO of AzurRx BioPharma, said in the news release. “They further confirm the activity of MS1819-SD and also demonstrate its dose response characteristics. Additionally, secondary efficacy endpoints are consistently aligning with the CFA data and the safety profile of MS1819-SD remains favorable.”
These preliminary results show encouraging effectiveness and safety data for MS1819-SD as an improvement on existing porcine-derived therapy to treat EPI.