The Ipsen Investment Case
Rare Disease
Key ongoing clinical-trial highlights
Trial
Palovarotene
Population
Patients
MOVE
Phase III
FOP
(chronic)
107
NCT03312634
IPN60130
Design
Palovarotene - 5mg QD
and upon flare-up,
20mg QD for 28 days,
followed by 10mg for 56
days
Placebo or
FALKON
Phase II
FOP
(chronic)
~90
two dosing regimens of
IPN60130
NCT05039515
Elafibranor
ELATIVE
Phase III
NCT04526665
Placebo
PBC
150
or
elafibranor
Endpoints
Primary: annualized change in
new HO volume
Secondary: subjects with new HO,
number of body regions with HO,
subjects with flare-ups, rate of
flare-ups, safety
Primary: annualized change in
new HO volume and safety
Secondary: change in HO volume
in new HO lesions, number of new
HO lesions, rate and number of
flare-up days, number of body
regions with HO, pain intensity
Response to treatment defined as
ALP < 1.67 x ULN and total
bilirubin ≤ ULN and ALP decrease
≥ 15 percent
Status
H1 2022
regulatory
resubmission (US)
H1 2022
'clock-stop' expiry
(EU)
First patient
commenced dosing
Q1 2022
Data anticipated
2023
IPSEN
Innovation for patient care
QD: once a day; HO: heterotopic ossification; ULN: upper limit normal; ALP: alkaline phosphatase.
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