Ipsen的Bylvay胆道闭锁III期BOLD试验未达到主要终点

Ipsen (IPSEY) has announced topline results from its pivotal Phase III BOLD study of Bylvay in children with biliary atresia following Kasai hepatoportoenterostomy. The trial did not meet its primary endpoint of improving native liver survival compared to placebo.
Biliary atresia (BA) is a rare, life-threatening pediatric liver disease and the leading cause of liver transplantation in infants, often before the age of two. With no approved medical therapies, treatment options remain limited to surgery or transplant.
Bylvay (odevixibat), a once-daily oral ileal bile acid transport inhibitor, is already approved in other rare pediatric liver diseases, including Progressive Familial Intrahepatic Cholestasis (PFIC) and Alagille syndrome. In the BOLD trial, the drug was evaluated for its potential to delay or reduce the need for liver transplantation in biliary atresia, a condition that remains the leading cause of pediatric liver transplants worldwide.
The study enrolled infants across multiple global centers, making it the largest and most comprehensive dataset ever assembled in biliary atresia. While the primary endpoint was not achieved, safety findings were consistent with Bylvay's established profile in approved indications. Investigators highlighted that the trial provides valuable insights into disease progression and patient outcomes, offering a foundation for future research.
Dr. Saul J. Karpen, lead investigator, highlighted the importance of the dataset, noting that BOLD represents a landmark effort in understanding biliary atresia. Ipsen's Head of R&D, Christelle Huguet, expressed gratitude to patients, caregivers, and investigators for their commitment, underscoring Ipsen's dedication to advancing understanding in rare pediatric liver disease. Safety data from BOLD remained consistent with the established profiles of Odevixibat in approved indications.
IPSEY has traded between $30.05 and $51.10 over the past year. The stock closed Thursday's trading at $47.11.
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