AzurRx's Stock Falls After MS1819 For Pancreatic Insufficiency Flunks Mid-Stage Study

AzurRx BioPharma Inc AZRX reported topline results from its Phase 2b OPTION 2 trial evaluating MS1819 in cystic fibrosis patients with exocrine pancreatic insufficiency.
MS1819 demonstrated to be safe and well-tolerated. However, the study did not consistently meet the primary efficacy endpoint.
Some patients achieved a coefficient of fat absorption (CFA) at levels beyond what is required to demonstrate non-inferiority with porcine enzyme replacement therapy (PERT) therapies, but the majority did not.
The company believes that the leading cause of the drug's uneven efficacy performance lies with the enteric capsule formulation.
While the enteric coating protects the capsule from breaking down in the stomach acid, it also appears to dissolve too slowly in the small intestine to release the lipase enzyme for proper digestion and nutrient absorption.
The company is planning to pursue a new formulation for MS1819, this one a capsule filled with acid-resistant granules, or microbeads, similar to what is used in CREON, ZENPEP, and other PERT therapies.
MS1819 is a recombinant lipase enzyme for treating exocrine pancreatic insufficiency associated with cystic fibrosis and chronic pancreatitis.
Price Action: AZRX shares dropped 23.9% at $0.988 in the premarket session on the last check Thursday.