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Phase 3 Failure Scuttles Cystic Fibrosis Drug

Sollpura didn’t meet noninferiority endpoint versus Pancreaze

Disappointing results in the phase 3 RESULT trial have led Anthera Pharmaceuticals, Inc., to discontinue development of its drug candidate Sollpura for the treatment of exocrine pancreatic insufficiency due to cystic fibrosis.

Sollpura failed to meet the trial’s primary endpoint: noninferiority in improvement of the coefficient of fat absorption (CFA) compared with Pancreaze (Janssen Pharmaceuticals), a pancreatic enzyme replacement therapy (PERT).

The design of the RESULT study was based on the outcome of the previous phase 3 SOLUTION study and included a higher starting dose and more aggressive dose optimizations based on clinical signs and symptoms of malabsorption. In RESULT, all patients randomized to Sollpura received a starting dose about 25% higher than their pre-study porcine PERT dose, and 59% of subjects received further dose adjustments, yielding a mean Sollpura dose (8,673 units/kg per day [range, 2,925–14,941]) that was both substantially higher than the mean dose of the comparator, Pancreaze (6,527 units/kg per day [range, 2,358–10,253]) and higher than the Sollpura dose in the SOLUTION study (mean dose, 7,286 units/kg per day [range 4,478–10,000]).

Although a proportion of patients randomized to Sollpura maintained or improved their CFA from baseline, a higher proportion of patients experienced a worsening. The mean treatment difference in CFA change from baseline was 14.3%, with upper and lower 95% confidence intervals of –18.22 and –10.39. In contrast, the treatment difference in coefficient of nitrogen absorption change from baseline (–1.53%) was well within the 15% noninferiority margin. the presence or absence of concomitant gastric acid suppressants had no meaningful effect on CFA (mean changes from baseline in CFA of –15.06% and –16.58%, respectively).

The RESULT study enrolled 140 patients in North America, Eastern and Western Europe, and Israel.

Anthera plans to evaluate the full data and will suspend further clinical development of Sollpura, including the 20-week extension period of RESULT, the SIMPLICITY study in patients ages 28 days to until their 7th birthday, which was designed to assess Sollpura powder for oral solution, and the EASY study, which was designed to follow the long-term safety of patients on Sollpura. In addition, Anthera plans to evaluate all strategic alternatives.

Source: Anthera; March 12, 2018.

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