Clinical Trial Highlights

Rare Disease Key ongoing clinical-trial highlights TRIAL POPULATION PATIENTS DESIGN Elafibranor ELATIVE Phase III 2L PBC 161 Placebo or elafibranor NCT04526665 PRIMARY ENDPOINT Response to treatment defined as ALP < 1.67 x ULN and total bilirubin ≤ ULN and ALP decrease ≥ 15 percent STATUS Primary endpoint met Bylvay ASSERT Alagille syndrome Phase III 33 63 Placebo or Change from baseline in scratching score U.S. regulatory approval June 2023 Bylvay NCT04674761 Bylvay BOLD Biliary atresia 205 Placebo or Phase III Bylvay NCT04336722 Proportion of patients who are alive and have not undergone a liver transplant after 104 weeks of study treatment Recruiting¹ IPSEN Innovation for patient care 1. Recruitment status as per ct.gov, June 2023. 2L: second line; PBC: primary biliary cholangitis; ALP: alkaline phosphatase; ULN: upper limit normal; PDUFA: Prescription Drug User Fee Act. 22 22

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