H1 2023 Financial Highlights
Rare Disease
Key ongoing clinical-trial highlights
TRIAL
POPULATION PATIENTS
DESIGN
Elafibranor
ELATIVE
Phase III
2L PBC
161
Placebo
or
elafibranor
NCT04526665
Bylvay
ASSERT
Alagille syndrome
63
83
Phase III
NCT04674761
Placebo
or
Bylvay
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
Change from baseline in
scratching score
U.S. regulatory approval
H1 2023
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.
28View entire presentation