AzurRx BioPharma's OPTION 2 Trial Of MS1819 In Cystic Fibrosis Fails, What Next?

(RTTNews) - AzurRx BioPharma Inc.'s (AZRX) phase IIb study of MS1819 for the treatment of exocrine pancreatic insufficiency in patients with cystic fibrosis did not consistently meet the primary efficacy endpoint.

The trial, dubbed OPTION 2, compared the safety, tolerability and efficacy of MS1819 against the current standard of care, porcine pancreatic enzyme replacement therapy pills. The primary efficacy endpoint was the coefficient of fat absorption (CFA).

Some patients were able to achieve CFA at levels beyond what is required to demonstrate non-inferiority with PERT therapies, but the majority did not, according to the trial results.

What next?

A phase 2 study evaluating the tolerability and efficacy of escalating doses of MS1819, in combination with PERT (pancreatic enzyme replacement therapy) pills, in order to increase CFA levels and relieve abdominal symptoms in CF patients is ongoing.

This trial completed enrollment of 18 patients last month, and top-line data are expected in the second quarter of 2021.

MS1819, whose active ingredient is a synthetic, fat-cleaving enzyme called lipase, derived from yeast cells, is administered as an enteric-coated capsule.

AzurRx believes that the underlying cause of the MS1819's uneven efficacy performance in the OPTION 2 Trial lies with the enteric capsule formulation.

While the enteric coating protects the capsule from breaking down in the stomach acid, the trial data suggests it may dissolve too slowly in the small intestine to release the lipase enzyme in time to aid with proper digestion and nutrient absorption, noted the company. So, a new formulation for MS1819, employing a capsule filled with acid-resistant granules, or microbeads, is going to be developed.

The company is in discussions with contract manufacturers to develop this new formulation, and a phase 2 study to evaluate the new formulation's efficacy is expected to be initiated without any delay.

AZRX closed Wednesday's trading at $1.34, up 3.08%. In extended trading, the stock fell nearly 30% and was at $0.94.