NAFLD Whitepaper
NAFLD Whitepaper
NAFLD Whitepaper
Introduction
Currently there are no approved pharmacological years there has been increased interest from our
treatments for non-alcoholic fatty liver disease Trialtrove clients for clinical trials intelligence on the
(NAFLD), including the subtype non-alcoholic treatment of (NAFLD), including the subtype (NASH).
steatohepatitis (NASH). The prevalence of the In the following report, I will leverage Trialtrove
disease is on the rise, as are common comorbidities data to assess the current industry sponsored trials
such as diabetes, insulin resistance and obesity landscape, including patient demographics, trial
that contribute to this disease. NAFLD is a leading locations and which pharmaceutical companies are
cause of liver disease and can potentially result in best positioned to enter the NAFLD market.
hepatocellular carcinoma (HCC). Over the past few
Figure 1. Industry Sponsored Non-Alcoholic Fatty Liver Disease Trials by Start Year
33
31
15
14
10
Figure 2. Top 5 Disease Complications Evaluated in Industry Non-Alcoholic Fatty Liver Disease Trials
90
80
70
60
# of Trials
50
40
30
20
10
0
Hepatic Fibrosis Type 2 Diabetes Dyslipidemia Obesity HCV
Note: Trials involving multiple disease complications may be counted more than once
Figure 3. Top Locations for Industry Non-Alcoholic Fatty Liver Disease Trials
China
Italy
Belgium
Canada
Spain
Japan
Australia
France
Germany
United Kingdom
United States
0 10 20 30 40 50 60 70 80 90
# of Trials
Note: Trials involving multiple locations may be counted more than once
Figure 4. Top 5 Industry Sponsors of Non-Alcoholic Fatty Liver Disease Trials by Phase
16
14
12
10
# of Trials
0
Allergen AstraZeneca Genextra Gilead Sciences Pfizer
16
14
12
10
# of Trials
0
es
ca
ra
an
er
is
bb
ire
isk
io
ct
im
Co
al
ch
in
m
rt
IB
re
rm Pfiz
xt
nc
ne
Sh
ui
rg
he
rd
tic
Kl
-M ova
ar
&
Ro
Du
ne
Sq
GR
le
ie
No
ith
Ze
el
eu
Ph
ck
l
Sc
Ge
N
A
ng
tra
rs
m
ac
er
vo
e
ye
ad
rI
oS
ab
M
As
No
ge
le
ax
an
ha
Gi
ol
rin
Gl
iT
sP
ist
eh
ish
Br
tu
Bo
ub
na
its
Co
NASH Non-NASH
Gilead holds the widest array of top drugs with In November 2016, Allergan completed the
four, all unapproved and three of which are in acquisition of Tobira Therapeutics, which included
Phase II development while one in Phase III. The the drugs cenicriviroc, a Phase II CCR2/5 antagonist
company has two Lysyl oxidase-like 2 inhibitors and evogliptin a DPP-IV inhibitor. Allergan/Tobira
in development, simtuzumab in Phase II, and has six trials total in NAFLD, two of which have
selonsertib in Phase III. Between these two completed. There are two ongoing trials, one
compounds, the company has sponsored just closed for enrollment and one Phase I trial, open
over 50% of their trials in the NAFLD space with for patient recruitment evaluating the combination
their Lysyl oxidase-like 2 inhibitor, showing their with evogliptin for the treatment of NASH. The
commitment to this mechanism of action (MOA). company has two planned trials. One Phase II trial
Currently, selonsertib is in two ongoing trials that is in combination with evogliptin in adults with NASH
are still recruiting patients, while all five of the trials and liver fibrosis, planned to start in the second
involving the Phase II compound simtuzumab have half of 2017. The other is a planned Phase III trial,
completed. Based on this, the company might STELLARIS, projected to start in April 2017 involving
concentrate R&D spending on the Phase III Lysyl adult NASH subjects with stage 2 to 3 liver fibrosis.5
oxidase-like 2 inhibitor and hold off on initiating any
further trials with simtuzumab. While a top sponsor in the space, AstraZeneca’s
activity is spread across a number of different drugs.
Of the top sponsors, Genextra has initiated the Among their five trials currently recruiting patients,
largest number of trials with a single compound. two involve unapproved compounds in Phase II
All eight trials sponsored by this company involve development, their MicroRNA inhibitor RG-125, and
obeticholic acid, a farnesoid X receptor agonist an undisclosed compound. The remaining five trials
currently approved for primary biliary cirrhosis. have completed or terminated. One completed trial
This compound is involved in a further two studies, involved RG-125, while the majority of the remaining
which are taking place in Japan and sponsored involved approved diabetes compounds such as
by Sumitomo Dainippon Pharma, for a total of 10 exenatide and dapagliflozin.
trials, the largest number of NAFLD activity for a
2 Dainippon Sumitomo Pharma, Intercept Pharmaceuticals (2011) Dainippon Sumitomo Pharma and Intercept Pharmaceuticals Announce Agreement
to Develop and Commercialize Obeticholic Acid (INT-747) for Chronic Liver Disease [Press release]. Note: Intercept is a subsidiary company held by
Genextra.
3 Conatus Pharmaceuticals, Pfizer (2010) Conatus Pharmaceuticals acquired the product as part of its acquisition of the Idun subsidiary from Pfizer
[Press release].
4 Novartis (2016) Conatus Pharmaceuticals has entered into an exclusive option, collaboration and license agreement with Novartis for the global
development and commercialization of emricasan [Press release].
5 Allergan (2016) Allergan Successfully Completes Tobira Therapeutics Acquisition [Press release].
Disease Number
Drug Name MOA Sponsor*
Status of Trials
Note: Trials involving multiple drugs may be counted more than once
*() denotes number of trials sponsored where multiple sponsors have initiated trials involving a particular compound
**One trial is a Planned Phase III
***Approved for the treatment of primary biliary cholangitis (PBC)
****Approved for the treatment of type 2 diabetes and obesity
United States Informa’s Pharma intelligence is home of the world’s leading pharma
52 Vanderbilt Avenue and healthcare R&D and business intelligence brands – Datamonitor
11th Floor
New York Healthcare, Sitetrove, Trialtrove, Pharmaprojects, Medtrack,
NY 10017 Biomedtracker, Scrip, Pink Sheet, In Vivo. Pharma intelligence’s brands
USA are trusted to provide over 3000 of the world’s leading pharmaceutical,
+1 646 957 8919 contract research organizations (CRO’s), medical technology,
+1 888 436 3012
biotechnology and healthcare service providers, including the top 10
United Kingdom global pharma and top 10 CRO’s, with an advantage when making
Christchurch Court critical R&D and commercial decisions.
10-15 Newgate Street
London
EC1A 7HD Accurate and timely intelligence about the drug development
United Kingdom pipeline is vital to understanding the opportunities and risks in today’s
+44 20 7017 5000 biopharmaceutical marketplace – whether you are targeting an unmet
medical need, investigating promising new therapies or researching
Japan
Kotakudo Ginza drug development historical trends and treatment patterns. If you are
Building, 7th Floor providing contract research or other services in the pharma industry,
5-14-5 Ginza you need to stand out. A solid understanding of your potential clients’
Chuo-ku pipelines and competition will help you leave a lasting impression.
Tokyo
104-0061
+81 351 487 670
China
23rd Floor
China Online Centre
333 Lockhart Road
Wanchai
Hong Kong
+85 239 667 222
Australia
Level 7
120 Sussex Street
Sydney
NSW 2000
+61 2 8705 6900