AzurRx BioPharma Will Conduct Phase 2b Trial of MS1819 at TDN Clinical Sites

AzurRx BioPharma has been authorized to conduct its Phase 2b OPTION 2 clinical trial evaluating MS1819, its investigative therapy for exocrine pancreatic insufficiency (EPI) associated with cystic fibrosis (CF), at sites that are part of the Therapeutics Development Network (TDN).

TDN is the largest network of CF clinical trials in the world. Supported by the Cystic Fibrosis Foundation, the TDN allows access to 91 certified U.S. care centers that specialize in CF research and facilitate patient recruitment and implementation of clinical trials. TDN’s approval comes on the heels of an authorization by an Institutional Review Board (IRB).

“We are very pleased to receive the continued support of the Therapeutics Development Network for our Phase 2b OPTION 2 trial, which will allow for full study participation in the TDN sites,” James Pennington, MD, chief medical officer of AzurRx, said in a press release.

MS1819 is an artificial form of lipase, an enzyme normally produced in the pancreas to help digest fats. Derived from the yeast Yarrowia lipolytica MS1819 is being investigated as a form of treatment for EPI associated with CF and chronic pancreatitis.

EPI is a condition in which the pancreas is unable to produce a sufficient amount of enzymes that help digest food, while chronic pancreatitis is a condition in which chronic inflammation prevents the pancreas from performing its normal function.

Unlike other forms of pancreatic enzyme replacement therapy (PERT), MS1819 does not contain any animal-based components.

“Patients have expressed a desire for non-porcine [not pig] derived alternatives to the current PERT therapies given ongoing safety and dietary concerns and now more recent concerns around availability given supply chain issues in China due to the COVID-19 pandemic and prior swine flus,” said James Sapirstein, CEO of AzurRx.

“We believe that our yeast-based recombinant enzyme therapy has the potential to become an effective and safer therapy for CF patients, with a more consistent and reliable manufacturing process and supply chain,” Sapirstein said.

The new Phase 2b trial OPTION 2 (NCT04375878) will assess the safety, tolerability, and effectiveness of oral enteric capsules containing 2.2 or 4.4 grams of MS1819 compared to current standard care with porcine PERT pills in CF patients with EPI. (Enteric capsules prevent medicines from being released in the acidic conditions of the stomach before reaching the intestine.)

The trial is expected to enroll about 30 participants and to reach its target enrollment goal by the end of the year. The study is expected to be completed in early 2021.

“We are optimistic that either the 2.2g or 4.4g dose of MS1819, in conjunction with the use of enteric capsules for delayed-release drug delivery, will enable us to successfully achieve our primary and secondary efficacy endpoints [goals]. A study conducted through the TDN provides a valuable platform for conduct of OPTION 2 in the U.S. and we look forward to beginning our enrollment,” Pennington said.

OPTION 2 builds on OPTION (NCT03746483), a recently completed six-week Phase 2 trial that investigated the safety and effectiveness of MS1819 against porcine PERT in CF patients with EPI.

Data from the OPTION trial showed that MS1819 was safe and was able to increase fat absorption in CF patients with EPI.

A separate Phase 2 trial (NCT04302662) assessing the the safety, tolerability, and effectiveness of increasing doses of MS1819 in combination with PERT in CF patients with EPI also is ongoing.