By John Vandermosten, CFA
AzurRx BioPharma, Inc. (AZRX) announced the completion of the Phase IIa MS1819-SD trial on June 29, 2018 and expects to provide a formal report presenting the data in the fall of 2018 at an as yet unidentified scientific conference. As a reminder, the trial was a Phase 2a open-label, dose escalation study conducted in France, Australia and New Zealand with the goal of identifying the safety of escalating doses and dose response. 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. Below we provide a summary of the trial design.
View Exhibit I – Phase 2a Trial Design
During the study, AzurRx provided interim updates, which found the coefficient of fat absorption improving by greater than 21% in evaluable patients. Favorable trends were also observed on other endpoints, including Bristol stool scale, number of daily evacuations and weight of stool, and these were consistent with the CFA results. While no summary data was provided in conjunction with the end of the trial, company CMO Dr. Pennington noted that the results confirm the safety and efficacy of MS1819 and support the start of a Phase IIb.
Coefficient of fat absorption (CFA) updates
‣ 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 IIb slated to begin this fall will examine a different population than the Phase IIa and will enroll cystic fibrosis (CF) patients. Leading the charge will be Dr. James Pennington, who was added to the team in May. He was formerly the CMO for Anthera Pharmaceuticals (ANTH) where he managed two Phase III trials enrolling the same CF population. The duration of these trials was approximately a year to a year and a half enrolling approximately 130 to 140 patients in each. We anticipate that the rate of enrollment for the CF trial will be more rapid based on the characteristics of the CF population and that it will take from 12 to 18 months to complete. Trial design is not fully known at this point, but we expect that the endpoints will examine efficacy against an active comparator that represents standard of care.
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.
View Exhibit II – AzurRx Pipeline
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By John Vandermosten, CFA