Intercept Pharmaceuticals, Inc.
ICPT (Intercept), a clinical stage biopharmaceutical company focused
on the development and commercialization of novel therapeutics to treat
chronic liver and intestinal diseases, announced today the publication of the
meta-analysis performed by the Global Primary Biliary Cirrhosis Study Group
(Global PBC Study Group) in the December issue of Gastroenterology. In the
largest meta-analysis of individual PBC patient data conducted to date,
researchers confirmed that levels of ALP and bilirubin predicted clinical
outcomes of patients with PBC.
Of the 4,845 patients included in the analysis, 1,118 reached a clinical
outcome defined as liver transplantation or death. For the first time, the
researchers reported a log-linear association between ALP values and liver
transplant-free survival. At one year after study enrollment, an ALP level of
two times upper limit of normal (ULN) best predicted patient outcome (C
statistic, 0.71) but not significantly better than other lower ALP thresholds
such as 1.67 times ULN. Of patients with ALP levels less than or equal to two
times ULN, 84% survived for over a ten year follow-up period compared with 62%
of those with levels exceeding two times ULN (p < 0.0001). Elevated bilirubin
levels were strongly predictive of a worse prognosis and only 41% of such
patients had not had a liver transplant or died over the subsequent 10 years
compared with 86% of patients with normal bilirubin levels (p < 0.0001).
The Global PBC Study Group analysis also showed that ALP and bilirubin were
correlated with clinical outcomes consistently over time and in all the
patient subgroups evaluated. Specifically, ALP was predictive of
transplant-free survival in PBC patients both on and off standard of care
treatment, those with histologically advanced or early stage disease, patients
45 years or younger or over 45 years at the time of diagnosis, female or male,
and irrespective of the year of diagnosis.
"This study represents the largest international collaboration in PBC and
provided us with a wealth of data supporting the use of this biochemical
endpoint for therapeutic clinical trials," said senior author Bettina Hansen,
Ph.D., Department of Gastroenterology and Hepatology, Erasmus University
Medical Centre in Rotterdam. "But the key takeaway from this analysis for
everyday clinical practice is that the lower a patient's ALP level and having
a normal bilirubin level correlate with an improved prognosis for patients
with PBC."
The Global PBC Study Group consists of 15 leading PBC centers in eight
countries that contributed to a clinical outcomes database of more than 6,000
patients with PBC. Data were analyzed under the direction of Dr. Bettina
Hansen, Dr. Willem J. Lammers, Dr. Henk van Buuren and colleagues at Erasmus
University Medical Centre in Rotterdam, the Netherlands. Intercept was a
sponsor of this independent academic research program but was not involved in
the study design, data collection, analysis or publication.
"We believe the Global PBC Study Group's research supports the clinical
relevance of a primary endpoint based on ALP and bilirubin in clinical trials
of patients with PBC," said David Shapiro, M.D., Chief Medical Officer of
Intercept. "More broadly, it should enhance monitoring of disease progression
and lead to better dialogue between clinicians and patients."
"The work done by the Global PBC Study Group exemplifies the value of such
cooperative initiatives in generating large clinical datasets that may provide
evidence for the utility of proposed surrogate endpoints and a better
understanding of the natural history of diseases such as PBC," said Mark
Pruzanski, M.D., CEO of Intercept. "We hope the Global PBC Study Group's work
continues in the future and would like to thank all of the researchers and
patients involved in making such an invaluable contribution to the medical
community's understanding of this rare disease."
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