The first patient has now been screened in the OPTION 2 trial (NCT04375878), which has opened at three clinical sites to start.
Researchers intend to enroll a total of 30 CF patients at 15 sites across the U.S. and Poland, and AzurRx anticipates providing top-line data from the study in early 2021.
“The initiation of the OPTION 2 trial is a major clinical milestone for the company,” James Sapirstein, CEO of AzurRx, said in a press release. “We look forward to the successful completion of the trial and to reporting our topline results of OPTION 2 in Q1 2021.”
Many CF patients experience symptoms involving the digestive system, which are often associated with the production of a thick mucus in the pancreas that blocks the release of the digestive enzymes needed to break down food. This deficiency in exocrine pancreatic enzymes, resulting in a patient’s inability to digest food properly, is referred to as EPI.
To treat EPI, CF patients often need pancreatic enzyme replacement therapy (PERT), a treatment designed to supplement enzyme levels to the digestive system. Enzymes used in PERT are typically derived from pigs or other animals, prompting the development of alternative formulations not derived from animals.
The active ingredient in MS1819 is a synthetic enzyme derived from yeast cells, thereby avoiding the use of animal products.
MS1819 is taken orally via an enteric capsule, which is coated in a polymer designed to withstand breakdown in the stomach, to deliver the MS1819 enzyme to the intestines.
The open-label, crossover OPTION 2 study is designed to evaluate the safety, tolerability, and efficacy of MS1819. Enrolled patients will be divided into two groups, with one receiving standard care PERT pills and the other receiving a 2.2 g or 4.4 g daily dose of MS1819.
After three weeks of taking either PERT pills or MS1819, patients will be switched to the other treatment for an additional three-week period.
To evaluate the efficiency of MS1819, a primary goal of the study, a stool sample will be analyzed for its fat content at the end of each three-week period. This test, which measures the coefficient of fat absorption, can determine the efficiency of fat digestion.
Secondary measurements of MS1819 efficiency include comparisons of stool weight, signs and symptoms of malabsorption in patients, and the coefficient of nitrogen absorption, which is also used to detect enzymatic inefficiency.
The recruitment and clinical testing for the trial is being assisted by the Cystic Fibrosis Institute, a nonprofit that supports patients with CF in the Chicago area.
“We are excited to be a clinical trial site in the OPTION 2 study and to continue participating in AzurRx’s ongoing efforts to find a safe non-porcine enzyme replacement therapy for our patients with cystic fibrosis,” said Steven Boas, MD, president and CEO of the Cystic Fibrosis Institute. “I am confident that we will be able to meet our enrollment targets for the trial in a timely fashion.”
The dosages for OPTION 2 were determined based on previous data from a Phase 2 trial (NCT03746483) called OPTION, for which the primary goals were to evaluate safety and determine a proper dosage for future investigation.
“We have designed the OPTION 2 efficacy study based on the results from the OPTION bridging dose safety study, and are optimistic that the enteric capsules delayed-release delivery of the 2.2 g and 4.4 g doses of MS1819 will enable us to achieve our primary and secondary efficacy endpoints in this study,” said James Pennington, MD, chief medical officer of AzurRx.
Previous results also indicated that MS1819 is safe and well tolerated in patients. With the OPTION 2 trial, AzurRx aims to determine an optimal dosage that can be used for a future Phase 3 study, and will continue monitoring MS1819’s safety.
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