A clinical trial testing AzurRx BioPharma‘s investigational therapy MS1819, in combination with the current standard care for the treatment of severe exocrine pancreatic insufficiency (EPI) in patients with cystic fibrosis (CF), has dosed its first two participants, AzurRx announced.
“In the midst of a global pandemic and unpredictable obstacles, we are very encouraged to see our Combination therapy trial moving forward at one of the clinical testing sites in Turkey,” James Sapirstein, president and CEO of AzurRx, said in a press release.
The current standard-of-care for EPI is the use of porcine-derived pancreatic enzyme replacement therapy (PERT), where pancreatic enzymes derived from pigs are used to functionally replace the missing pancreatic enzymes in the digestive tract. However, even with PERT, many people with EPI will have worse-than-typical fat digestion, which can result in unpleasant symptoms and impair proper nutrition.
MS1819 contains a synthetic fat-cleaving enzyme (lipase) derived from yeast cells, designed to aid in fat digestion for people with EPI. The medication is administered in oral capsules. Since its active ingredient is derived from yeast (a type of fungus), the investigational therapy avoids potential concerns and sensitivities with the use of animal products.
The open-label, multicenter, Phase 2 combination trial (NCT04302662), sponsored by AzurRx, is assessing the safety and efficacy of MS1819 in combination with each participant’s established daily dose of PERT. MS1819 is being administered in immediate release capsules in increasing doses of 700, 1,200, and 2,240 mg per day, for 15 days in total.
According to AzurRx, preliminary results from the initial five patients in the study indicated that the combination therapy meaningfully increased the amount of dietary fat participants absorbed during digestion — the coefficient of fat absorption (CFA) — at each of the three doses tested.
More specifically, mean CFA at the MS1819 700 mg/day plus standard care group was 88.4%, 87.2% in the 1,200 mg/day dose group, and 86.5% in the 2,240 mg/day group, compared to baseline level (78.4%).
Improvements also were seen in secondary parameters, such as body weight, stool properties (consistency, weight, and frequency of evacuations), and the incidence of steatorrhea (excessive amount of fat in feces).
No adverse safety events were reported.
The study is still enrolling participants in Hungary and Turkey; more information is available here. According to the company, there are five active sites recruiting in Turkey.
“We expect to release topline results [from the trial] in the second quarter of next year,” Sapirstein said. “We are optimistic the results will build on the encouraging data we received earlier this year and hope we will be one step closer to improving the lives of thousands of patients suffering from cystic fibrosis.”
MS1819 also is being evaluated in another clinical trial, OPTION 2 (NCT04375878), which is comparing MS1819 to traditional pig-derived PERT in people with EPI. The trial is recruiting in the U.S. and Poland; more information is available here.
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