corporate presentation - jefferies · pbc ww ocaliva net sales $20.6m 1q 2017 continue enrollment...
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1
Corporate PresentationJune 2017
2
Safe Harbor & Disclaimer Statement
This presentation contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. 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: Intercept's ability to successfully commercialize Ocaliva® (obeticholic acid) in PBC, and
Intercept's ability to maintain its regulatory approval of Ocaliva in PBC in the United States and the European Union; 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 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 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 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 product candidates; Intercept's ability to successfully commercialize OCA in indications other than
PBC and its other product candidates; the size and growth of the markets for Intercept's 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 that it may receive for its products
from payors; the success of competing drugs that are or become available; 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; regulatory
developments in the United States and other countries; the performance of third-party suppliers and manufacturers; Intercept's need for and ability to
obtain additional financing; Intercept's estimates regarding expenses, future revenues and capital requirements and the accuracy thereof; Intercept's
use of cash, short-term investments and the proceeds from the offering; Intercept's ability to attract and retain key scientific or management
personnel; and other factors discussed under the heading "Risk Factors" contained in Intercept’s Annual Report, Quarterly Reports and other filings
with the Securities and Exchange Commission. All information in this presentation is as of the date hereof, and Intercept undertakes no duty to update
this information unless required by law.
This presentation presents adjusted operating expense, which is a non-GAAP measure, both on a historical and projected basis. Adjusted operating
expense should be considered in addition to, but not as a substitute for, operating expense that Intercept prepares and announces in accordance with
GAAP. Intercept excludes certain items from adjusted operating expense, such as the one-time net expense of $45.0 million for the proposed
settlement of the purported securities class action lawsuit, stock-based compensation and depreciation, that management does not believe affect
Intercept's basic operations and that do not meet the GAAP definition of unusual or nonrecurring items.
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Building a Specialty Focused Business in Progressive Non-Viral Liver Diseases
Novel therapeutic approach in
progressive non-viral liver diseases
OCA is an approved, first in class farnesoid X receptor (FXR) agonist that is active in the liver and
gut, with an extensive safety database (>1,600 clinical trial subjects, >675 patient years of exposure)
Worldwide rights (outside of Japan, China and Korea)
Patent terms projected to expire at various times through 2033
PBC:
Ocaliva® for Primary Biliary
Cholangitis
Indication for the treatment of PBC in adults with an inadequate response to UDCA, or as
monotherapy in adults unable to tolerate UDCA
$20.6M in 1Q 2017 world-wide net sales
Steady growth expected; solid long-term market opportunity to make Ocaliva standard of care for
second line treatment
COBALT Phase 4 confirmatory outcomes trial ongoing
NASH:
Significant Market Opportunity
Breakthrough therapy designation from FDA for NASH w/ liver fibrosis
Phase 3 REGENERATE trial - Announced complete enrollment of interim analysis cohort in May
2017; Data expected in 1H 2019
Phase 2 CONTROL trial readout in mid-2017
Broad NASH clinical development program planned
Significant Upside from Further OCA
Indications and Pipeline
Phase 2 AESOP trial in primary sclerosing cholangitis (PSC) readout in mid-2017
OCA in development for additional non-viral, progressive liver diseases
INT-767 Phase 1 recently completed, Phase 2 in NASH w/ liver fibrosis expected to start in 2H 2017
4
Pipeline Focused on Progressive Liver Diseases with High Unmet Need
Own worldwide rights to all programs (except Japan, China & Korea for OCA)
Preclinical Phase 1 Phase 3Phase 2 ApprovedProduct / Population
INT-777 (TGR5 Agonist)
NASH w/ fibrosis
INT-767 (Dual FXR / TGR5 Agonist)
Primary Biliary Cholangitis (PBC)
Nonalcoholic Steatohepatitis (NASH)
Primary Sclerosing Cholangitis (PSC)
Biliary Atresia
OCA (FXR Agonist)
5
Key Recent and Upcoming 2017 Milestones
PBC
WW Ocaliva net sales $20.6M 1Q 2017
Continue enrollment of Phase 4 COBALT trial 2017
NASH
Complete enrollment of interim analysis cohort in Phase 3
REGENERATE trialMay 2017
Report Phase 2 CONTROL results Mid-2017
Initiate Phase 3 OCA cirrhosis trial 2H 2017
Initiate INT-767 Phase 2 trial 2H 2017
PSC Report Phase 2 AESOP results Mid-2017
6
The PBC Opportunity
7
Primary Biliary Cholangitis (PBC) Overview
NATURAL HISTORY:
Increased ALP is observed early in the disease
Hepatocellular damage marked by increases in AST, ALT, GGT
Elevation of bilirubin occurs with advanced disease
PREVALENCE:
Disease of women (10:1)
1 in 1,000 women >40 years old
DIAGNOSIS:
Non-invasive diagnosis (blood test)
Elevated alkaline phosphatase (ALP) & anti-mitochondrial antibody (AMA)
Pruritus (itching) and fatigue are signature symptoms
CholestasisHepato-
cellular
damageFibrosis Cirrhosis
HealthyLiver
CirrhoticLiver
8
Ocaliva Was Approved in May 2016
Granted accelerated approval in
PBC in combination with
ursodeoxycholic (UDCA) in adults
with an inadequate response to
UDCA or as monotherapy in adults
unable to tolerate UDCA
Approval based on a reduction in
alkaline phosphatase (ALP)
Launched June 2016
9
~19,000 PBC Patients In U.S. Initial Launch Focus
130k
55k 50k 47k
30k
15k 4k
47%1
91%
93%2
64%
33%
0.04%
1. Access to healthcare assumed in calculation at 88.6%
2. 8% of patients under treater care are intolerant or discontinued due to failure on UCDA. 85% of patients under treater care are currently treated with UDC
3. UDCA discontinue patients represent 9% of patients who are either UDCA treated or UDCA intolerant or discontinue
ICPT Market Research
9%3
Estimates based on FYE2016
Prevalence Diagnosed Under treater care UDCA Treated /
UDCA discontinue
intolerance or failure
UDCA treated
uncontrolled
(ALP > ULN)
UDCA treated
uncontrolled
(ALP > 1.67)
UDCA discontinue
tolerability / failure
Launch focusing on patients
matching POISE criteria
10
1Q Ocaliva Commercial Update
*Source IMS; $ in millions
Weekly U.S. Ocaliva Prescription Data*
TR
x
0
50
100
150
200
250
300
350
400
450
1/2017 2/20176/2016 4/20173/201711/20169/20168/20167/2016 5/201712/201610/2016 6/2017
$4.7
$13.4
$19.8
$0.1
$20.6
1Q 20174Q 2016
$13.4
3Q 2016
$4.7
2Q 2016
ex-U.S. Sales
U.S. Sales
11
We’ve Made Significant Progress on the U.S. Launch
In the first 9 months of launch we accomplished:
– Good traction with high-decile physicians
– Increased education and awareness overall by hosting >350 PBC disease and branded
speaker programs, reaching > 3000 physicians and office staff
– Achieved >282 million lives covered with average time from Rx to drug to patients ~3 weeks
Now that we are entering the next phase of launch, we plan to focus beyond our high decile
physicians:
– Expanding our efforts to reach deeper into our target list
– Recognizing that while these physicians represent a larger portion of overall market, they
will require greater disease education
11
We expect steady quarter over quarter growth
12
We Are Making Good Progress for Ocaliva Internationally
Revenues
– $0.8M in 1Q 2017 sales
– Ex-U.S. to contribute modestly to Ocaliva sales in 2017
• Germany & France key early access markets
Pricing & Reimbursement
– We have made good progress in Europe & Canada
– Rapid reimbursement decision by NICE within four months of EMA approval
• Formal publication of the guidance in April
EASL 2017
– New EASL treatment guidelines published; Ocaliva recommended as second line therapy
12
13
The NASH Opportunity
14
NASH: A Worldwide Health Issue
1: Wree, A. et al. Nat. Rev. Gastroenterol. Hepatol. 10, 627–636 (2013)
2: Wong et al. Hepatology 2014; 59(6):2188-95
3: Tateishi et al. J of Gastroenterology. June 2014
Changes in the lifestyle and diet of the global population are fuelling a worldwide surge in
obesity and the increasing prevalence of NAFLD/NASH
NASH linked to increased dietary consumption of fructose and PUFAs1
NASH expected to become the leading cause of liver transplant by 20201
Growing NASH-related HCC among liver transplants (8% in 2008 to 14% in 2012)2,
with up to 40% of HCC in non-cirrhotic patients3,4
4: Dyson et al. J. Hepatology 2014; 60(1):110-7
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Broad and Comprehensive NASH Clinical Plan for OCA
Trial Description Status
Ph
ase 3 REGENERATE Efficacy & Safety in NASH w/ Fibrosis
Completed enrollment of interim
analysis cohort in May 2017
Cirrhosis Program Efficacy & Safety in NASH w/ Cirrhosis Initiate in 2H 2017
Ph
as
e 2
Un
derw
ay/
Pla
nn
ed CONTROL OCA & Statin Effects on LDL and Lipid Metabolism Data in mid-2017
Pediatric NASH Efficacy & Safety in Pediatric NASH Patients In planning
NASH Registry In planning
Ph
as
e2
co
mp
lete
d
FLINT Efficacy & Safety in Non-Cirrhotic NASHTetri et al. The Lancet 2015; 385:
956-65
Japanese NASH Efficacy & Safety in Japanese NASH PatientsConducted by Sumitomo Dainippon
Pharma
Diabetes/NAFLD Diabetes + NAFLD Euglycemic ClampMudaliar et al, Gastroenterology 2013;
145:574-82
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Liver Fibrosis: Associated with Long-term Outcomes
1: Angulo et al, Gastroenterology 2015;
2: Retrospective analysis of 619 patients diagnosed with NAFLD over median follow-up of 12.6 years
Liver Fibrosis, But No Other Histologic Features, Associates With Long-term
Outcomes of Patients With Nonalcoholic Fatty Liver Disease
Paul Angulo, MD, David E. Kleiner, MD, PhD, Sanne Dam-Larsen, MD, PhD, Leon
A. Adams, MBBS, PhD, Einar S. Bjornsson, MD, Phunchai Charatcharoenwitthaya,
MD, Peter R. Mills, MD, Jill C. Keach, Heather D. Lafferty, MB, ChB, Alisha Stahler,
Svanhildur Haflidadottir, MD, Flemming Bendtsen, MD, PhD
10.9x
3.8x2.9x 2.6x
1.9x 1.6x
0.3x0
2
4
6
8
10
12
FibrosisStage 4
FibrosisStage 3
FibrosisStage 2
Current Smoker FibrosisStage 1
Diabetes Statin Use
Hazard Ratio of Death or Liver Transplant1,2
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OCA is the FXR Agonist That Has Met Primary and Key Secondary Histologic
Endpoints in a Well-Controlled Phase 2 Trial
17
43%
21%
0%
5%
10%
15%
20%
25%
30%
35%
40%
45%
50%
OCA Placebo
>1 Stage Fibrosis Improvement
w/o Worsening of NASH* (Stage 2 or 3 Fibrosis)2
p=0.0059n=58 n=58
35%
19%
0%
5%
10%
15%
20%
25%
30%
35%
40%
45%
OCA Placebo
Primary Analysis
> 1 Stage Fibrosis Improvement
p=0.004n=102 n=98
1: Data from FLINT trial published online in Tetri et al. The Lancet and Supplementary Appendix on November 7, 2014; All p-values compared to placebo.
2. Post-hoc analysis
*Defined as no increase in hepatocellular ballooning or lobular inflammation
Phase 2 FLINT1 trial included 200 paired biopsies over 72 weeks– Primary endpoint met: decrease in the NAS of at least two points with no increase in the fibrosis score
– OCA improved all components of NASH, including fibrosis, steatosis, inflammation & ballooning
– OCA was generally well tolerated based on safety and tolerability data
A significantly greater proportion of OCA patients achieved an improvement of at least one fibrosis stage in both the primary analysis and a post-hoc analysis of FLINT in a REGENERATE-matched patient cohort
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REGENERATE: Randomized Global Phase 3 Trial to Evaluate the Impact on NASH with Fibrosis of
Obeticholic Acid Treatment
Placebo
OCA 10 mg
OCA 25 mg
End of Study
72 week Interim
Analysis
Primary endpoints:
• Fibrosis Improvement with no
worsening of NASH
OR
• NASH Resolution2 with no
worsening of fibrosis
Event-Driven
Final Analysis*
~350
sites
~2,000
patients
Interim histology analysis at 72 weeks in 750 patients planned to serve as basis for filing for approval
Announced complete enrollment in May 2017; Data expected in 1H 2019
*EOS endpoint: Occurrence of pre-specified number of clinical events
Entry Criteria1:• Biopsy-confirmed NASH
• Fibrosis stage 2 or stage 3
1Exploratory group of NASH patients with stage 1 liver fibrosis with comorbid risk factors (defined as diabetes, obesity or active liver inflammation (ALT >1.5X ULN)) will also be enrolled, but not included in the
primary endpoint analyses2Hepatocyte ballooning score of 0 & residual or no inflammation (“objective definition”)
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Phase 2 CONTROL Trial : Combination of OCA And Statins for Monitoring of Lipids
Placebo
OCA 10 mg
OCA 25 mg
Baseline lipid
profile
OCA 5 mg
OCA 10 mg
OCA 25 mg
2-year Open Label Extension Study
Week
16
Atorvastatin
10mg
Atorvastatin
20mg
Titrated
Atorvastatin Statin Use per EU/US Guidelines
• Evaluate the impact of varying doses of OCA on LDL and lipid metabolism
• Evaluate the impact of low doses of statin therapy to modulate LDL in
combination with OCA treatment
• Data expected mid-2017
N=80
20
The PSC Opportunity
21
Primary Sclerosing Cholangitis (PSC): Overview
PSC is an autoimmune cholestatic liver disease
PSC is a significant market opportunity
Estimated prevalence in U.S. ~40,0001
Typically more complicated & aggressive than PBC2
~75% of PSC patients have IBD, principally ulcerative colitis3
Biliary obstructions & infections
Cholangiocarcinoma & liver cancer
Orphan indication with high unmet need: no approved treatment
UDCA often used, although not recommended by AASLD4
Regulatory pathway to be determined
1: ICPT Market Research
2: Robbins and Cotran pathological basis of disease
3: Bambha et al. Gastroenterology 2003; 125:1364-1369
4: Chapman et al. Hepatology 2010; 51:660-678
EPIDEMIOLOGY:
DISEASE COURSE:
TREATMENT:
Hirschfield, et al; The Lancet. June 2013
22
Phase 2 AESOP Trial: Assessment of Efficacy and Safety of OCA in PSC
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Placebo
OCA 5 mg
Randomization
OCA 1.5 mgOCA 3 mg
OCA 10 mg
Week 12
Titration
Week 24
Screening
< 30 days
from Day 0
Open label
LTSE
• ~75 patients
• Proof of principle study, second cholestatic disease
• Primary endpoints: change from baseline in ALP; safety
• Data expected mid-2017
23
First Quarter 2017 Financial Results
23
Quarter Ended
3/31/20172017 Guidance
Net Product Revenue $20.6
Gross : Net 10-15% 10-15%
COGs De minimis De minimis
Interest Expense $7.2 ~$30.0
GAAP Operating Expense $105.0
Adjusted Operating Expense1 $90.1 $380 - $420
Cash Position $608.0
1Excludes non-cash items such as stock-based compensation and other non-cash items; see reconciliation table on slide 23All values in millions
24
Reconciliation Table
24
Three Months Ended
March 31
2017 2016
Total operating expense
(GAAP)$105.0 $127.8
Adjustments:
Stock based compensation 14.1 10.2
Depreciation 0.8 0.7
Litigation settlement - 45.0
Adjusted operating expense $90.1 $71.9
All values in millions
25
Appendix
26
Appendix: NASH
27
Updated REGENERATE Phase 3 Trial Design
27
Interim Analysis End of Study
NPrimary
Endpoints1
Definition of
NASH ResolutionInclusion
Treatment
Arms
Treatment
DurationN Primary Endpoint
Treatment
Duration
Current Study
Design
~750
Fibrosis
improvement
OR NASH
resolution
Hepatocyte
ballooning score
of 0 & residual
or no
inflammation2
Biopsy
proven
NASH3 with
fibrosis stage
2 or 34
OCA 10mg
OCA 25mg
Placebo
72 weeks ~2,000
Occurrence of
pre-specified
number of
clinical events
comprising a
composite
outcomes
endpoint
Event
driven
1Primary endpoints defined as fibrosis improvement with no worsening of NASH OR NASH resolution with no worsening of fibrosis2“Objective definition” of NASH resolution3Central pathologist assessment of definite NASH and NAFLD Activity Score (NAS) > 4 4NASH patients with stage 1 liver fibrosis with comorbid risk factors (defined as diabetes, obesity or active liver inflammation (ALT >1.5X ULN)) also being enrolled as an exploratory cohort
28
NAFLD: Progression to NASH w/ Fibrosis
1: McPherson et al, Journal of Hep, 2015;
2: 108 patients had serial biopsies (mean 6.4 years to repeat biopsy): 27 patients with baseline NAFLD and 81 patients with baseline NASH
44%37%
22%
0%
10%
20%
30%
40%
50%
DevelopedNASH
Developed NASH + Fibrosis
DevelopedNASH w/ Stage 3
Fibrosis
NAFLD: Progression to NASH2
10% 9%
35%
0%
10%
20%
30%
40%
50%
NASH: Progression to Cirrhosis2
F1 F2 F3
Over an average follow-up period of ~6 years1,2:
Evidence of NAFLD progression from steatosis to fibrosing-
steatohepatitis using paired biopsies: implications for prognosis
and clinical management
Stuart McPherson, Tim Hardy, Elsbeth Henderson, Alastair D. Burt, Christopher P. Day, Quentin M. Anstee
29
35%
17%15%
2%
19%
5%
18%
5%
0%
5%
10%
15%
20%
25%
30%
35%
40%
OCA
Pbo
Fibrosis Improvement and Progression
N=102 N=98
P=0.004
1: Data from FLINT trial published online in Tetri et al. The Lancet and Supplementary Appendix on November 7, 2014.
2: All p-values compared to placebo. P-values calculated with the Cochran-Mantel-Haenszel test, stratified by clinic and diabetes status.
Improvement in Fibrosis
> 1-stage
Improvement
Intercept Subgroup Analyses
N=92 N=83
P=0.0018
Fibrosis
Resolution
Fibrosis Progression
N=67 N=68
Not. sig.
Progressed to
Bridging
N=101 N=97
Not. sig.
Progressed to
Cirrhosis
30
22%19%
13%
8%
0%
5%
10%
15%
20%
25%
All Completers All Completers with Definite or Borderline NASHat Baseline
% of Patients w/ NASH Resolution OCA
Pbo
NASH Resolution: Baseline NASH-Only Analysis
P=0.0832,
Not. sig. P=0.0278
N=94 N=87N=102 N=98
~20% of FLINT patients did not have definite NASH at baseline
Post-hoc analysis excluding patients without baseline NASH demonstrates significant response vs.
placebo on NASH resolution
1: Data from FLINT study published online in Tetri et al. The Lancet and Supplementary Appendix on November 7, 2014. Of FLINT patients assessed for secondary endpoints, 10% (19/200) did not have definite/borderline-NASH at baseline
2: All p-values compared to placebo. P-values calculated with the Cochran-Mantel-Haenszel test, stratified by clinic and diabetes status.
Intercept Subgroup Analysis
31
45%
61%
53%
46%
35%
22%
12%
21%
38%35%
31%
19%
13% 13%
0%
10%
20%
30%
40%
50%
60%
70%
Primary Endpoint Steatosis LobularInflammation
Ballooning Fibrosis NASH resolution PortalInflammation
% of Patients w/ Improvement OCA Pbo
FLINT: Primary and Secondary Histological Endpoints
P=0.0002 P=0.001 P=0.006 P=0.03 P=0.004 not. sig.
1: Data from Tetri et al. The Lancet. Published online November 7, 2014.
2: All p-values compared to placebo. P-values calculated with the Cochran-Mantel-Haenszel test, stratified by clinic and diabetes status.
not. sig.
32
Appendix: PBC
33
Interconnect® – Our Personalized Patient Support Program
Interconnect financial assistance
programs includes:
– A $0 copay program for patients with
commercial insurance.
– Access to Ocaliva at no cost for
eligible uninsured or underinsured
patients.
Interconnect can also help refer
patients with Medicare or Medicaid to
an independent non-profit
organization that provides financial
assistance to PBC patients
MD
Prescribes
OCALIVA for PBC
Interconnect
Support Services
Patient
Specialty
Pharmacy
Network
•Conduct Benefit
Investigation
•Assist with appeals
•Administer financial
assistance programs
•Patient education
•Compliance and
persistency
• Confirm coverage
• Distribute product
• Report data
Care Coordinator -
Single Point of Contact
34
Global PBC Opportunity
130k116k
55k 50k 47k30k
15k
112k112k
55k48k 46k
29k
15k
290k276k
134k119k
112k
72k
37k
US EU5 Canada ANZ ROE
Global Prevalence / OCA Candidate Assumptions
US ROW
Prevalence (rate / ~2016 pop.)1,2 0.04% 0.035%
Access to healthcare3,4 89.6% 100%
PBC Diagnostic rate (2016) 5 47% 49%
Under treater care5 91% 88%
Treated w/ UDCA6 85.0%
UDCA discontinue – Intolerant/failure5 8.0%
Inadequate responder ALP ≥ULN5,6 71.0%
Adequate responder ALP <ULN5,6 29.0%
1: Kim et al. J. Gastroenterology. 2000; 119(6):1631-6
2: CDA Epidemiology Research
3: US Census Bureau 2015
*: Does not include intolerant patients
4: OCED Health Data 2014
5: Intercept Market Research
6: Lammers el al Gastroenterology. 2014 Dec;147(6):1338-1349 Estimates based on FYE2016
Prevalence Access Diagnosed Under treater care UDCA Treated /
UDCA discontinue
intolerance or failure
UDCA treated
uncontrolled*
(ALP > ULN)
UDCA treated
uncontrolled*
(ALP > 1.67)
35
Appendix: FXR
36
In Animal Models, OCA Activation of FXR has Shown Anti-Fibrotic
Effects in the Liver
36
OCA activation of FXR has anti-fibrotic effects in multiple animal models of chronic liver, intestinal and
renal diseases and has demonstrated the ability to reverse fibrosis in a rat model of toxic cirrhosis1-6
Effects of OCA on Fibrosis in TAA Rats6
Representative macroscopic and microscopic (Sirius red stain with subsequent image analysis) illustration of a vehicle- vs. OCA-treated rat
Vehicle OCA
1. Albanis E, Alvarez CE, Pruzanski M, et al. Hepatology. 2005. Oct;42(4):605A-606A. 2. Vignozzi L, Morelli A, Filippi S, et al. J Sex Med. 2011 Jan;8(1):57-77. 3. Morelli A, Comeglio P, Filippi S, et al. Journal of Steroid Biochemistry and
Molecular Biology 2012;132:80-92. 4. Wang XX, Jiang T, Shen Y, et al. American Journal of Physiology-Renal Physiology 2009;297:F1587-F96. 5. Wang XX, Jiang T, Shen Y, et al. Diabetes. 2010;59:2916-27. 6. Verbeke L, Mannaerts I,
Schierwagen R, et al. Sci Rep. 2016 Sep 16;6:33453.
37
Is There a Rationale for the Therapeutic Potential of FGF19 in
Non-viral Liver Diseases?
37
Fibroblast growth factor 19 (FGF19) is an ileal
protein hormone that plays a role in governing
bile acid homeostasis and metabolic processes
The primary source of endocrine FGF19 is the
ileum, where FGF19 expression is controlled
through FXR activation
– Bile acids released into the intestine after a meal
bind to and activate FXR and thereby induce
expression of FGF19
The therapeutic potential of FGF19 has been
explored in PBC
– This compound did not advance to Phase 31
To date, no investigational compounds with an
FGF19-driven mechanism of action have
demonstrated efficacy in patients with NASH
1. NGM press release. March 2015.