By John Vandermosten, CFA
AzurRx BioPharma, Inc. (AZRX) announced the start of a new a Phase II study. The effort will examine combination use of MS1819-SD and pancreatic enzyme replacement therapy (PERT) for cystic fibrosis (CF) patients with severe exocrine pancreatic insufficiency (EPI). This is a subset of CF patients that are suffer from malnutrition and fail to absorb sufficient fat despite taking the maximum dose of PERT therapy. It is an unmet need and represents about a third of total CF patients.
The new study will be conducted at six sites in Hungary with an enrollment target of 28. 24 of this group are expected to complete all dosing. This population of CF patients exhibiting severe EPI is currently prescribed at or near the 10,000 IU of lipase/kg/day, with dose limitations due to risks from fibrosing colonopathy. The patients will receive combination therapy adding MS1819-SD to their assigned dose of PERT therapy in an effort to increase digestion of fats and achieve nutritional goals. The trial will serve as a dose escalation study, starting with 700 mg/d, then increasing to 1120 mg/d and finally to 2240 mg/d. It is expected to last 100 days and provide a readout in early 2020. Primary endpoints for the Phase II are safety and coefficient of fat absorption (CFA).
View Exhibit I - Phase 2a CP Trial Design
We see this as an efficient strategy to address a patient population with few alternatives. The CF population with severe EPI currently only has an option of the insertion of a feeding tube while taking the maximum dose of PERT therapy along with an extremely high level of caloric intake. If successful, this may provide a viable option which will enable patients to achieve their nutritional goals through non-invasive measures.
View Exhibit II - Updated Pipeline Graphic
AzurRx has completed enrollment in its Phase II OPTIONS study in a head to head trial with PERT. Results from the effort are expected to be provided in a September release. Initial observations have been positive with excellent site conduct and safety, faster than expected enrollment and low withdrawal rates. Now that the OPTIONS trial is wrapping up, management has turned its attention toward a complementary indication. The new study for severe EPI provides additional data and expands the utility of MS1819 and we believe will increase the chances of eventual regulatory approval.
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By John Vandermosten, CFA