Shares of thinly-traded nano-cap biotech AzurRx BioPharma Inc AZRX are trading sharply lower following a clinical readout from the company.
AzurRx is working on developing non-systemic, recombinant therapies for gastrointestinal diseases. The company said the results of the Phase 2 OPTION trial that is evaluating its MS1819 in the treatment of exocrine pancreatic insufficiency in cystic fibrosis showed that the primary efficacy endpoint of coefficient of fat absorption, or CFA, was comparable to that in a previous Phase 2 study in patients with chronic pancreatitis.
The dose used in both studies was 2 gram per day, in line with the agreement with the FDA as a bridging dose.
Despite the study not being powered for statistical significance, the data showed meaningful efficacy results, with about 50% of patients showing CFAs high enough to reach non-inferiority with porcine enzyme replacement therapies, or PERT, AzurRx said.
However, the company noted CFA of the MS1819 treatment arm was 56% compared to 86% for the PERT treatment arm.
Suggesting that protease supplementation is not required with MS1819, the coefficient of nitrogen absorption was comparable between the MS1819 arm and the PERT arm.
"With these data showing MS1819 to be safe and to have the potential to support fat absorption, we have considerable reason to be optimistic for the next steps in non-porcine enzyme development," said Dr. Michael Konstan, the principal investigator of the study.
AzurRx said it looks forward to meeting with the FDA before the year end to discuss these results and its next steps. The company plans to discuss with the regulatory agency the Phase 2b/3 trial design exploring the use of higher doses and/or enteric-coated capsules to ensure higher MS1819 activity in the duodenum.
AzurRx shares were down 30.32% to 62 cents at time of publication.
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