- OCA met the primary endpoint of alkaline phosphatase (ALP)
reduction at 24 weeks
- AESOP represents a successful proof of concept for OCA in a
second cholestatic liver disease
Conference call scheduled for 8:30 a.m.
ET today
Intercept Pharmaceuticals, Inc. (Nasdaq:ICPT) (Intercept), a
biopharmaceutical company focused on the development and
commercialization of novel therapeutics to treat progressive
non-viral liver diseases, today announced that the Phase 2 AESOP
trial evaluating obeticholic acid (OCA) for the treatment of
patients with primary sclerosing cholangitis (PSC) met its primary
endpoint. Patients who initiated OCA 5 mg with the option to
titrate to 10 mg achieved a statistically significant reduction in
alkaline phosphatase (ALP) as compared to placebo (p<0.05).
AESOP is a 24-week, double-blind, placebo-controlled,
dose-ranging trial evaluating the efficacy and safety of OCA
compared to placebo in 77 patients with PSC, followed by a two-year
long term safety extension (LTSE) open-label phase which is
currently ongoing. Patients were randomized to one of three
treatment groups: placebo, OCA 1.5 – 3 mg, and OCA 5 – 10 mg (with
dose titration occurring at the 12-week midpoint). Approximately
half the patients were receiving ursodeoxycholic acid (UDCA)
treatment at baseline and continued on a stable dose during the
trial. The primary endpoint of the study was the change in ALP
relative to placebo at week 24 for the OCA 5 – 10 mg group. Results
for the intent-to-treat population are shown below.
(U/L) |
Placebo (N = 25) |
OCA 1.5-3 mg (N = 25) |
OCA 5-10 mg (N = 26) |
Mean Baseline ALP |
563 |
423 |
429 |
Least Squares (LS) Mean Change from Baseline in ALP at Week 12 |
-53 |
-57 |
-135* |
LS Mean Change from Baseline in ALP at Week 24 |
-27 |
-105 |
-110*† |
LS Mean Percent Change from Baseline at Week 24 |
+1% |
-22%* |
-22%* |
* p<0.05† Primary endpoint was ALP change for OCA 5-10 mg
compared to placebo at week 24.
Pruritus is a common symptom of PSC and was the most common
adverse event observed in the AESOP trial, occurring in 46%, 60%
and 67% of patients in the placebo, OCA 1.5 – 3 mg and OCA 5 – 10
mg groups, respectively. Pruritus severity increased with OCA
treatment in a dose-dependent manner. One (4%) patient in the OCA
1.5 – 3 mg group and three (12%) in the 5 – 10 mg group
discontinued treatment due to pruritus compared to none with
placebo.
Other treatment emergent adverse events were similar across all
three arms and the proportion of patients completing the
double-blind period was similar across treatment groups (84%, 76%
and 81% for placebo, OCA 1.5-3 mg and OCA 5-10 mg, respectively).
Of these patients, 59 of 61 (97%) chose to participate in the LTSE
phase.
“Currently there are no therapies proven to be effective in
treating patients with PSC, a chronic liver disease that results in
significant morbidity and mortality,” said Kris V. Kowdley, MD,
Director, Liver Care Network and Organ Care Research, Swedish
Medical Center, Seattle, and one of the lead investigators of the
trial. “The results of this Phase 2 study of OCA are encouraging
because patients achieved improvement in ALP, a marker of chronic
cholestasis. There is a pressing need for innovation in this
disease and, given these results, additional studies are warranted
to better define the role of OCA as a treatment for PSC.”
“These results provide proof of concept for OCA in a second
cholestatic liver disease with a very high unmet need,” said David
Shapiro, M.D., Chief Medical Officer of Intercept. “We look forward
to sharing the complete results from AESOP with the hepatology
community at an upcoming scientific congress. We believe these data
warrant further investigation and look forward to speaking with PSC
thought leaders and regulators to help inform our future
development plans."
Conference Call on July 31st at 8:30 a.m. ET
Intercept will discuss the AESOP results during its second quarter
2017 financial results conference call and webcast on July 31st at
8:30 a.m. ET. The live event will be available on the investor page
of Intercept's website at http://ir.interceptpharma.com or by
calling (855) 232-3919 (toll-free domestic) or (315) 625-6894
(international) five minutes prior to the start time (no passcode
is required). A replay of the call will be available on Intercept's
website approximately two hours after the completion of the call
and will be archived for two weeks.
About AESOPAESOP is a Phase 2 randomized,
double-blind, placebo-controlled, dose-finding evaluation of the
efficacy and safety of 24 weeks of treatment with obeticholic acid
(OCA) compared to placebo in 77 patients with PSC. The primary
endpoint of the AESOP trial is the LS mean change in serum alkaline
phosphatase (ALP) levels, as compared to placebo. Patients with
well-controlled irritable bowel disease (IBD) at baseline were
permitted to enroll in the AESOP trial and patients receiving
ursodeoxycholic acid (UDCA) treatment at baseline (approximately
50% of patients) were permitted to continue on a stable dose.
About Primary Sclerosing Cholangitis Primary
sclerosing cholangitis (PSC) is a rare, life-threatening, chronic
cholestatic liver disease characterized by progressive destruction
of bile ducts that leads to the development of cirrhosis and
end-stage liver disease or cancer in a majority of patients.1-4
There are no approved therapies for PSC5, and estimated survival
time from PSC diagnosis to death or liver transplant is 14.5
years.6 Approximately 65% of PSC patients are male4, and 60%-80% of
patients have concomitant inflammatory bowel disease (IBD), most
often ulcerative colitis.4,5,7 Although it is a rare disease, PSC
is the seventh leading indication for liver transplant in adults in
the United States.8,9
About InterceptIntercept is a biopharmaceutical
company focused on the development and commercialization of novel
therapeutics to treat progressive non-viral liver diseases,
including primary biliary cholangitis (PBC), nonalcoholic
steatohepatitis (NASH), primary sclerosing cholangitis (PSC) and
biliary atresia. Founded in 2002 in New York, Intercept now has
operations in the United States, Europe and Canada. For more
information about Intercept, please visit
www.interceptpharma.com.
Safe Harbor Statements This press release
contains "forward-looking statements" within the meaning of the
Private Securities Litigation Reform Act of 1995, including, but
not limited to, statements regarding the epidemiology and
prevalence of PSC, the potential utility of the endpoints used in
the AESOP trial, the potential of OCA to treat patients with PSC,
and our strategic directives under the caption "About Intercept."
These "forward-looking statements" are based on management's
current expectations of future events and are subject to a number
of important risks and uncertainties that could cause actual
results to differ materially and adversely from those set forth in
or implied by such forward-looking statements. These risks and
uncertainties include, but are not limited to: the potential
benefit and commercial potential of Ocaliva in PBC, and Intercept's
ability to maintain its regulatory approval in jurisdictions in
which Ocaliva is approved for use in PBC; the initiation, cost,
timing, progress and results of Intercept's development activities,
preclinical studies and clinical trials; the timing of and
Intercept's ability to obtain and maintain regulatory approval of
OCA in PBC in countries outside the ones in which it is approved
and in indications other than PBC and any other product candidates
it may develop such as INT-767; conditions that may be imposed by
regulatory authorities on Intercept's marketing approvals for its
products and product candidates such as the need for clinical
outcomes data (and not just results based on achievement of a
surrogate endpoint), and any related restrictions, limitations,
and/or warnings in the label of any approved products and product
candidates; Intercept's plans to research, develop and
commercialize its product candidates; Intercept's ability to obtain
and maintain intellectual property protection for its products and
product candidates; Intercept's ability to successfully
commercialize its products and product candidates; the size and
growth of the markets for Intercept's products and product
candidates and its ability to serve those markets; the rate and
degree of market acceptance of any of Intercept's products, which
may be affected by the reimbursement received from payors; the
success of competing drugs that are or become available; regulatory
developments in the United States and other countries; the
performance of third-party suppliers and manufacturers; the
election by Intercept's collaborators to pursue research,
development and commercialization activities; Intercept's ability
to attract collaborators with development, regulatory and
commercialization expertise; Intercept's need for and ability to
obtain additional financing; Intercept's estimates regarding
expenses, revenues and capital requirements and the accuracy
thereof; Intercept's use of cash and short-term investments;
Intercept's ability to attract and retain key scientific or
management personnel; and other factors discussed under the heading
"Risk Factors" contained in our annual report on Form 10-K for the
year ended December 31, 2016 filed on March 1, 2017 as well as any
updates to these risk factors filed from time to time in our other
filings with the Securities and Exchange Commission. All
information in this press release is as of the date of the release,
and Intercept undertakes no duty to update this information unless
required by law.
References
- Molodecky NA, Kareemi H, Parab R, et al. Incidence of primary
sclerosing cholangitis: A systematic review and meta-analysis.
Hepatology. 2011;53:1590-1599.
- Chapman R, Fevery J, Kalloo A, et al. Diagnosis and management
of primary sclerosing cholangitis. Hepatology.
2010;51(2):660-678.
- Karlsen TH, Boberg KM. Update on primary sclerosing
cholangitis. J Hepatol. 2013;59:571-582.
- Bowlus CL. In: Forman LM, ed. Primary Sclerosing Cholangitis:
Current Understanding, Management, and Future Developments.
Switzerland: Springer International Publishing; 2017:1-11.
- Horsley-Silva JL, Rodriguez EA, Franco DL, Lindor KD. An update
on cancer risk and surveillance in primary sclerosing cholangitis.
Liver Int. 2016; doi:10.1111/liv.13354.
- Weismüller TJ, Trivedi PJ, Bergquist A, et al. Patient age,
sex, and inflammatory bowel disease phenotype associate with course
of primary sclerosing cholangitis. Gastroenterology.
2017;152(8):1975-1984.
- Hirschfield GM, Karlsen TH, Lindor KD, Adams DH. Primary
sclerosing cholangitis. Lancet. 2013;382:1587-1599.
- Adam R, et al. Evolution of indications and results of
liver transplantation in Europe. A report from the European Liver
Transplant Registry (ELTR). J Hepatol. 2012
Sep;57(3):675-88.
- Singal AK, et al. Evolving frequency and outcomes of
simultaneous liver kidney transplants based on liver
disease etiology. Transplantation. 2014 Jul
27;98(2):216-21.
Contact
For more information about Intercept Pharmaceuticals, please contact:
Mark Vignola
+1-646-747-1000
investors@interceptpharma.com
Christopher Frates
+1-646-757-2371
media@interceptpharma.com
Intercept Pharmaceuticals (NASDAQ:ICPT)
Historical Stock Chart
From Mar 2024 to Apr 2024
Intercept Pharmaceuticals (NASDAQ:ICPT)
Historical Stock Chart
From Apr 2023 to Apr 2024