AzurRx BioPharma slumps as OPTION 2 trial of MS1819 in CF fails

US clinical-stage biopharma AzurRx BioPharma (Nasdaq: AZRX) saw its shares plunge nearly 24% to $1.02 last week, after the company released top-line results from its Phase IIb OPTION 2 clinical trial investigating MS1819 in cystic fibrosis (CF) patients with exocrine pancreatic insufficiency (EPI) that did not consistently meet the primary efficacy goals.

OPTION 2 was designed as a Phase IIb multicenter study to investigate the safety, tolerability and efficacy of MS1819 (in enteric capsules) in a head-to-head comparison against the current porcine enzyme replacement therapy (PERT) standard of care for the treatment of exocrine pancreatic insufficiency (EPI) in patients with cystic fibrosis. The primary efficacy endpoint was the coefficient of fat absorption (CFA), with secondary endpoints of stool weight, signs and symptoms of malabsorption and coefficient of nitrogen absorption (CNA).

The trial also included an extension arm that used an immediate release MS1819 capsule, allowing the company to compare data from the existing arm that uses enteric (delayed release) capsules with data from the new arm, and ultimately select the optimal delivery method.