The trial, dubbed OPTION 2 (NCT04375878), aims to enroll 30 CF patients ages 18 and older. Top-line data from the trial is expected in the first half of 2021.
“Enrolling the first patient in our Phase 2b OPTION 2 trial is an important milestone,” James Pennington, MD, chief medical officer of AzurRx, said in a press release. “We anticipate data from this trial will inform the optimal dose for our pivotal Phase 3 study, and look forward to top line results in the first quarter of next year.”
As MS1819’s active ingredient is a synthetic fat-cleaving enzyme (lipase) derived from yeast cells, the therapy avoids concerns over the use of animal products.
The OPTION 2 trial aims to evaluate the safety, tolerability, and efficacy of MS1819 compared to current standard of care, porcine (from pigs) enzyme replacement therapy.
Trial participants will receive either standard PERT or MS1819 at doses of 2.2 grams or 4.4 grams. MS1819 is available in capsules coated with a stomach acid-resistant polymer designed to deliver the medication directly to the intestines (enteric capsules).
After three weeks of treatment with one of the two MS1819 doses or standard PERT, patient stool samples will be collected to analyze their fat content.
From the amount of fat in a stool sample, researchers can infer a patient’s efficiency of fat digestion. This measurement is the trial’s primary endpoint. Secondary trial endpoints include measurements of stool weight, signs and symptoms of malabsorption, and the coefficient of nitrogen absorption — another measure of enzymatic efficiency.
Patients 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 for fat absorption to be compared with the prior samples.
Patients will continue to be monitored for two weeks after completing the treatment-switching period.
Recruitment is currently open at four U.S. locations with plans to expand to 15 locations in the U.S. and Europe.
“We are very pleased to announce that patient dosing in the OPTION 2 trial has begun in the U.S.,” said James Sapirstein, CEO of AzurRx.
“This represents an important milestone in the clinical development of MS1819 as an EPI monotherapy for patients with cystic fibrosis. Despite the challenges posed by the COVID-19 pandemic, the AzurRx team has been able to successfully activate new clinical trial sites and work with investigators to safely admit patients into the trial,” Sapirstein added.
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