AzurRx BioPharma is requesting a new patient arm be added to its ongoing Phase 2b trial investigating MS1819 capsules as a treatment for exocrine pancreatic insufficiency (EPI) in people with cystic fibrosis (CF).
The request, filed with the U.S. Food and Drug Administration (FDA), would add a patient group to test MS1819 immediate release capsules, so investigators can compare them with the enteric (delayed release) capsules currently used in the OPTION 2 trial. The goal is to select the best way of delivering this medication.
If the FDA approves the request, patient enrollment for the new arm is expected to begin in December.
“We are pleased to announce that our OPTION 2 trial is well ahead of schedule and almost fully enrolled without any safety issues being observed,” James Pennington, MD, chief medical officer of AzurRx, said in a press release.
“As this trial progresses, we believe there may be significant clinical value in adding a study arm that uses our immediate release capsules,” Pennington added. “Our intention is to identify the optimal delivery method and potentially provide additional scientific support and optionality for MS1819.”
In people with CF, thick mucus forms in organs that include the pancreas, blocking the release of enzymes needed to properly digest food. The EPI that results is usually treated with pancreatic enzyme replacement therapy (PERT), in which digestive enzymes — generally taken from pigs or other animals — are used to replace the enzymes that are lacking.
The active ingredient in MS1819 is a synthetic fat-cleaving enzyme (lipase) derived from yeast cells. As such, the investigational therapy avoids potential concerns and sensitivities with the use of animal products.
An open-label study, OPTION 2 (NCT04375878) is evaluating the safety, tolerability, and efficacy of MS1819 compared with porcine (from pigs) enzyme replacement therapy.
“The key objectives of any Phase 2 program are to test for safety and efficacy,” Pennington said, “but another important goal is to determine appropriate doses and drug delivery method prior to entering the Phase 3 portion of the clinical program.”
The trial, which recently opened enrollment, aims to recruit 30 adults with CF. Enrollment is ongoing at multiple locations in the U.S. and Poland; additional information on trial sites is available here.
Trial participants will be given either standard PERT or MS1819 at doses of 2.2 grams or 4.4 grams. After three weeks of the respective treatment, stool samples will be collected and analyzed to begin determinations of the respective efficacy of the treatments.
Those taking MS1819 will then switch to standard PERT, and vice-versa, for an additional three weeks, after which stool samples will again be collected and analyzed.
“The protocol amendment represents an opportunity to gain valuable additional clinical data with no significant increase in overall trial costs or delays to our timeline projections,” said James Sapirstein, CEO of AzurRx. “We remain on target to release top line data in the first quarter of 2021, as has been our guidance to date.”
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