Phase 2 IMAGO trial of SHP625 fails to meet primary endpoints in pediatric patients with ALGS

Shire plc (LSE: SHP, NASDAQ: SHPG) today announced that the 13-week Phase 2 IMAGO trial of its investigational compound SHP625 (LUM001) did not meet the primary or secondary endpoints in the study of 20 pediatric patients with Alagille syndrome (ALGS), a rare, life-threatening genetic disorder that presents with chronic cholestasis (accumulation of bile acids in the liver) and severe pruritus (itching). The primary endpoint was the change from baseline in serum bile acid levels as compared to placebo. The secondary endpoint of pruritus was assessed using the novel ItchRO™ instrument.

Mean serum bile acid levels and pruritus at the end of the study were lower in both SHP625 and placebo treated groups as compared to baseline. However, in a post-hoc analysis, a positive correlation between percent changes from baseline in serum bile acid levels and pruritis was observed in the SHP625 treated group. The number of patients in the placebo treated group was too small to make an accurate assessment of this relationship.

There were no treatment emergent serious adverse events in this study. As expected, the most common adverse events were diarrhea and abdominal pain, which were more frequent with SHP625 than with placebo.

"We have gained important insights from these first results from one of several phase 2 studies in the SHP625 development program," said Philip J. Vickers, Ph.D., Head of Research and Development, Shire. "We remain committed to continuing the ongoing studies of SHP625 in ALGS and other indications."

In addition to IMAGO, two larger placebo-controlled phase 2 studies in ALGS are in progress, one of which has pruritus as the primary endpoint. SHP625 is also being studied in progressive familial intrahepatic cholestasis, primary biliary cirrhosis and primary sclerosing cholangitis.