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AZRX: Final Results from Phase II EPI Trial

09/24/2018
By John Vandermosten, CFA

NASDAQ:AZRX

AzurRx BioPharma, Inc. (NASDAQ:AZRX) announced the results from their Phase IIa trial of MS1819-SD, a recombinant lipase, for the treatment of exocrine pancreatic insufficiency (EPI) caused by chronic pancreatitis. Today’s results were in line with previous interim levels and presented a coefficient of fat absorption (CFA) of 21.8%. The results were statistically significant with a p-value of 0.002. This low p-value suggests that individual patient results were tightly clustered around the indicated level.

The CFA of 21.8% compares favorably to previous interim reported results of greater than 20% to 21%. Other endpoints were also positive, including Bristol stool scale, number of daily evacuations and weight of stool, which were consistent with the CFA results. CMO Dr. Pennington was pleased at the high level of statistical significance of the results and announced his expectation that the Phase IIb portion of the trial in cystic fibrosis (CF) will begin later in 2018. Below, we provide the previous interim updates for the trial:

‣ 2Q:18 – Dose response > 20%; 3 patients
‣ September 2018 – Dose response > 21%; 6 patients
‣ April 2018 – Dose response > 21%; 9 patients

This compares to the dose response of just over 16% that was achieved in the Phase 1/2a FLIP110 study. The Phase II in CF slated to begin this fall will examine a different population than the Phase IIa and will enroll CF patients.

Phase 2a for EPI was an open-label, dose escalation trial with the goal of identifying the safety of escalating doses and dose response in patients with chronic pancreatitis. The trial initially targeted enrollment of 12 to 15 patients with EPI; however, results were sufficiently significant to end the trial at 11 patients. It was conduted in conjuntion with Mayoly Spindler Laboratories and launched November 17, 2016.


View Exhibit I - Phase 2a Trial Design

The positive and consistent results from the Phase IIa continue to be supportive of improved digestion and absorption of nutrients for patients that lack the necessary enzymes to digest fats. Shifting away from porcine-based lipase enzymes to MS1819 provides a host of benefits that includes an improved side effect profile, reduced contamination risk, consistency in enzyme content and reduced size and number of capsules that are needed.

MS1819 CP Phase II in Cystic Fibrosis

Dr. James Pennington, who previously ran Anthera’s (ANTH) Phase III Sollpura program, will guide AzurRx through the clinical trial process. His experience with the CF population makes him a particularly valuable asset to guide AzurRx through the next clinical trial steps. Dr. Pennington will also bring select members of his previous team to help advance MS1819 forward towards regulatory approval.

Despite the slow pace of the previous EPI trial, we think that the CF study will run more smoothly for several reasons due to the nature of the CF population and Dr. Pennington’s experience. Dr. Pennington has already supervised a trial in a similar population to the one that will be examined in the next round, and was able to advance 127 patients in 17 months in the SOLUTION study and then another 140 patients in 11 months in the RESULT study at Anthera. The CF population, which will be examined in the study, is a much easier group to manage given the greater degree of focus on health management. This gives us greater confidence that AzurRx will be able to advance this candidate through these studies at a similar rate.

While the details of the clinical protocol for the Phase II in CF for MS1819 are not yet available, it is likely that the trial will be designed as an active comparator to Creon, or other standard of care. AzurRx will provide an update on details following their discussions with the FDA which we believe are currently ongoing.

Summary

The results of the Phase IIa trial have confirmed the clinical activity of MS1819 and provided evidence of its safety. The impressive p-value is suggestive of a very tight clustering of observations around 22% improvement and a stable response to MS1819. We anticipate additional details from the Phase IIa to be available before the end of 2018 and presented at a conference next year. AzurRx is on track to launch the next Phase II trial for CF prior to the end of 2018. Today’s results indicate that the company continues to be on track with its development plan.

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