NONALCOHOLIC STEATOHEPATITIS (NASH) - OPPORTUNITY ANALYSIS ... · EVENT-DRIVEN UPDATE REFERENCE...
Transcript of NONALCOHOLIC STEATOHEPATITIS (NASH) - OPPORTUNITY ANALYSIS ... · EVENT-DRIVEN UPDATE REFERENCE...
NONALCOHOLIC STEATOHEPATITIS (NASH) - OPPORTUNITY ANALYSIS AND FORECASTS TO 2017 -
EVENT-DRIVEN UPDATE
REFERENCE CODE GDHC034POA | PUBLICAT ION DATE MARCH 2014
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NONALCOHOLIC STEATOHEPATITIS (NASH) – OPPORTUNITY ANALYSIS AND FORECASTS TO 2017
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Executive Summary
NASH: Key Metrics in Six Major Pharmaceutical Markets
2012 Epidemiology
Prevalent Population – 6MM 59.3m
2012 Market Sales
US $208.4m
5EU $24.5m
Total $232.9m
Pipeline Assessment
Number of drugs in Phase IIb 4
Number of first-in-class drugs 4
Most Promising Pipeline Drugs 2017 Sales
GFT505 (Genfit) $581.8m
Obeticholic acid (OCA) (Intercept Pharmaceuticals/Dainippon Sumitomo Pharma [DSP])
$706.3m
Key Events (2012–2017) Level of Impact
GFT505 Launch in the US, 5EU in Q4 2017
↑↑↑
OCA Launch in the US in Q4 2017 ↑↑↑
2017 Market Sales
US $1.28bn
5EU $77.0m
Total $1.36bn Source: GlobalData. 5EU = France, Germany, Italy, Spain, UK; 6MM = US, 5EU.
End of Forecast Surge in Sales for NASH from 2012–2017
GlobalData estimates the 2012 off-label sales for
nonalcoholic steatohepatitis (NASH) to be
approximately $233m across the US and five major
European markets of France, Germany, Italy,
Spain, and the UK. By forecast end in 2017,
GlobalData expects sales to grow to about
$1.36bn, with a compound annual growth rate
(CAGR) of 42.2% across the 6MM. We expect the
US to claim the most sales, contributing $1.28bn of
global sales and a CAGR of 43.8%.
Major drivers of growth in the NASH market are
attributed to two factors:
The growing rate of obesity and diabetes
globally.
The launch of Genfit’s GFT505 and
Intercept/DSP’s obeticholic acid (OCA) in
2017. In OCA’s favor, its Phase II/IIb trial was
stopped early in January 2014, after a planned
interim analysis showed that the primary
endpoint of the study had been met. While for
GFT505, Genfit is aligning itself to register
GFT505 among the first approved therapies for
NASH and the company received Fast Track
designation for the GFT505 NASH program on
February 14, 2014.
We expect the US’ growth will be due in part to
the launch of both of these products, while
growth in the 5EU will be predominantly driven
by GFT505 during the forecast period.
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Executive Summary
Major barriers to the growth of the NASH market
are attributed to:
Slow development of therapies specific for
NASH.
Unclear understanding of the pathophysiology
of NASH.
Inefficient diagnostic tools for NASH that may
lead to delayed diagnosis of NASH.
Sales for NASH by Region, 2012–2017
89%
2%4%2%
2%1%
US
France
Germany
Italy
Spain
UK
2012 Total Sales:$232.9m
94%
1%1%
2%1%
1%
2017 Total Sales:$1,356.1m
Source: GlobalData.
Emerging Market Players Employing Strategies to Improve Diagnostics and Trial Designs
The NASH market is virtually vacant aside from off-
label therapies that do not provide a “one size fits
all” approach. The potential for market leaders to
emerge is wide open to all contenders. We expect
Gilead and Novo Nordisk to gain market entry after
the end of the forecast period in 2017; however,
with their market knowledge, their products
simtuzumab and liraglutide should do well
commercially.
As for newcomers Genfit and Intercept/DSP, fame
will be claimed since their products will likely
launch at the end of the forecast in Q4 2017, thus
giving them an early foothold in the NASH market
space.
Since there is no cure or one specific therapy
currently available for NASH, R&D strategies in
this market space are complicated. Complications
range from unraveling the pathophysiology of
NASH, to designing diagnostic tools for more
accurate diagnosis and staging, to the design of
clinical trials. As our expert leaders have agreed,
NASH needs an approved therapy that is cost-
effective and touts a desirable efficacy and safety
profile.
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Executive Summary
Vast Unmet Needs in the NASH Market
The level of unmet needs for NASH, including both
environmental and clinical, is high and includes
patient awareness, physician education, and
approved therapies specific to NASH. These
issues compound the reasons why the NASH
market space is very sparse. A series of unknowns
exist for the disease, due to its heterogeneous
nature, and its slow progression poses problems
for drug development. Vitamin E, the current gold-
standard therapy, dominates what little drug
therapy NASH market there is. We expect both
Genfit’s small molecule GFT505 and Intercept
Pharmaceuticals/DSP’s small molecule OCA to
claim patient share in all subpopulations of NASH.
These products will likely solve the top unmet need
– the dire need for an approved therapy for NASH.
Opportunities Will Remain for Tools that Provide Accurate Diagnosis
There is a major opportunity for new entrants to
design better biomarkers than the current liver
enzymes: alanine aminotransferase (ALT) and
aspartate aminotransferase (AST) as non-invasive
surrogates for Nonalcoholic Fatty Liver Disease
(NAFLD). Testing may also include cytokeratin-18
(CK18), which may provide clues to hepatocyte cell
death. In addition, markers that can assess liver
stages are needed, since CK18 is also elevated in
other liver conditions.
Currently, NASH patients may be diagnosed by
chance during routine serological examination, or
via invasive liver biopsy. Most NASH patients will
be in the mid/late stage of the disease once
diagnosed. A more accurate diagnostic tool for
determining the extent of fibrosis in NASH patients
will also be paramount. That said, we expect these
opportunities to remain unmet during the five-year
forecast period from 2012–2017.
2017 Launches of Novel Pipeline Products Will Forever Change the Future NASH Treatment Landscape
Twelve molecules of interest across all stages of
development make up the pipeline for NASH.
However, GlobalData believes there are four
therapies with the potential to revolutionize the
treatment paradigm for NASH in the near term.
These four novel, potential first-in-class therapies,
which are antifibrotic and antidiabetic by
therapeutic class, will target subpopulations
(obese, diabetic, and “other” or healthy donors)
within NASH.
The four most promising NASH pipeline products
are:
Genfit’s GFT505: anticipated to launch in Q4
2017 across the 6MM.
Intercept Pharmaceuticals / Dainippon
Sumitomo Pharma’s obeticholic acid (INT-
747/DSP-1747): anticipated to launch in Q4
2017 in the US, with an expected launch in the
EU after the forecast period of 2012–2017.
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Executive Summary
Gilead’s simtuzumab: anticipated to launch
after the forecast period of 2012–2017.
Novo Nordisk’s liraglutide (Victoza): the
marketed type 2 diabetes brand is anticipated
to launch (with NASH as a new indication) after
the forecast period of 2012–2017.
Below figure provides a competitive assessment of
the most promising NASH pipeline products.
Competitive Assessment of Mid-Stage Pipeline Agents in NASH, 2012–2017
Vitamin E
Pioglitazone (Actos)
SimtuzumabGFT505 & OCA
Liraglutide
0.0
0.5
1.0
1.5
2.0
2.5
3.0
3.5
4.0
0 1 2 3 4 5
Com
mer
cial
Sco
re
Clinical Score Source: GlobalData.
What Do the Physicians Think?
In regards to comparing profiles on Galmed’s
Aramchol (an early-stage pipeline product for
NASH), GFT505, and OCA, an EU KOL
interviewed by GlobalData responded with:
“…these three drugs attack the problem through
three different ways – that is good because there
are more chances at least one of them may work.
Also, operating through different pathways,
sometime in the future [they] may get combined [to
allow for combination treatment].”
[EU] key opinion leader, June 2013
GlobalData believes that Novo Nordisk’s liraglutide
(Victoza) is a pipeline drug worth tracking since it
has demonstrated a good preliminary safety and
efficacy profile. While discussing the NASH
pipeline, a KOL from the EU confirmed our
analysis of the product and conveyed excitement
over the product.
“The GLP-1 analogues…liraglutide [Novo Nordisk’s
Victoza] [is the pipeline drug that I am excited
about].”
[EU] key opinion leader, June 2013
While discussing the pipeline products, one KOL
explained why they did not think the Fatty-
Acid/Bile-Acid Conjugates (FABACs) inhibitors
such as Aramchol were going to succeed:
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Executive Summary
“I don’t think they [Fatty-Acid/Bile-Acid Conjugates
inhibitors] are going to be extremely effective.
Several have been tried with modest effects. It is
unlikely this is going to be the best game-changer.
A number of bile acids have been tested in NASH
and they have had modest effect.”
[US] key opinion leader, June 2013
Other KOLs discussed the types of R&D strategies
that should be employed by pharmaceutical
companies, and explained what may work and
what has failed and thus should be avoided:
“…to reduce apoptosis and hepatic inflammation
[is an important strategy]…[as well as a drug that
is] shown to be safe and reduce liver enzymes.
Drugs that reduce insulin resistance or glucose
levels are being tested by pharmaceutical
companies for this. Companies which are looking
to reduce liver fat have not had any impressive
effect that I think are truly going to make it.”
[US] key opinion leader, June 2013
“The greatest thing would be to find a NASH drug
and an antifibrotic drug that would be combined.
Combination therapy in patients who need it...”
[EU] key opinion leader, June 2013
“There is a huge trial going on for an anti-LOXL2
monoclonal antibody [Gilead’s simtuzumab]. It is
an antifibrotic drug, but it’s developed mainly for
NASH…it acts by blocking fibrosis... If it does well
in NASH [referring to the positive results it had
during primary sclerosing cholangitis trials], then it
will change the world!”
[EU] key opinion leader, June 2013
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Table of Contents
1 Table of Contents
1 Table of Contents ......................................................................................................................... 7
1.1 List of Tables ........................................................................................................................ 11
1.2 List of Figures ....................................................................................................................... 13
2 Introduction ................................................................................................................................ 14
2.1 Catalyst ................................................................................................................................ 14
2.2 Related Reports ................................................................................................................... 15
3 Disease Overview ...................................................................................................................... 16
3.1 Etiology and Pathophysiology ............................................................................................... 16
3.1.1 Etiology ........................................................................................................................... 16
3.1.2 Pathophysiology ............................................................................................................. 17
3.2 Symptoms ............................................................................................................................ 20
4 Epidemiology.............................................................................................................................. 21
4.1 Disease Background ............................................................................................................ 21
4.2 Risk Factors and Comorbidities ............................................................................................ 22
4.2.1 Increased age is associated with a worsened disease progression, prognosis, and
mortality .......................................................................................................................... 22
4.2.2 Being a male is an independent risk factor in morbidly obese NASH cases .................... 23
4.2.3 Metabolic conditions, such as type 2 diabetes, considerably increase the risk of NASH . 24
4.3 Global Trends ....................................................................................................................... 25
4.3.1 US .................................................................................................................................. 26
4.3.2 5EU ................................................................................................................................ 27
4.4 Forecast Methodology .......................................................................................................... 29
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Table of Contents
4.4.1 Sources Used ................................................................................................................. 30
4.4.2 Forecast Assumptions and Methods ............................................................................... 32
4.4.3 Sources Not Used........................................................................................................... 35
4.5 Epidemiology Forecast of NASH (2012–2022) ..................................................................... 36
4.5.1 Total Prevalent Cases of NASH ...................................................................................... 36
4.5.2 Age-Specific Prevalent Cases of NASH .......................................................................... 38
4.5.3 Sex-Specific Prevalent Cases of NASH .......................................................................... 39
4.5.4 Age-Standardized Prevalence of NASH .......................................................................... 41
4.6 Discussion ............................................................................................................................ 41
4.6.1 Conclusions on Epidemiological Trends ......................................................................... 41
4.6.2 Limitations of the Analysis .............................................................................................. 42
4.6.3 Strengths of the Analysis ................................................................................................ 43
5 Current Treatment Options ......................................................................................................... 44
5.1 Overview .............................................................................................................................. 44
5.2 Product Profiles – Major Off-Label Brands ............................................................................ 46
5.2.1 Vitamin E (numerous generics) ....................................................................................... 46
5.2.2 Pioglitazone (Actos) ........................................................................................................ 50
6 Unmet Needs Assessment and Opportunity Analysis ................................................................. 54
6.1 Overview .............................................................................................................................. 54
6.2 Unmet Needs Analysis ......................................................................................................... 55
6.2.1 Lack of Approved Therapies ........................................................................................... 55
6.2.2 Identification of Biomarkers for Early Diagnosis and Endpoints....................................... 56
6.2.3 Physician Awareness Due to Understanding of Pathophysiology .................................... 56
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Table of Contents
6.3 Opportunity Analysis ............................................................................................................. 57
6.3.1 Therapies with Targeted MOA to NASH ......................................................................... 57
6.3.2 Better Models to Understand Drug Efficacy .................................................................... 57
6.3.3 Future Tool to Measure Fibrosis in NASH Patients ......................................................... 57
7 R&D Strategies .......................................................................................................................... 58
7.1 Overview .............................................................................................................................. 58
7.1.1 Understanding Multiple Factors of NASH ........................................................................ 58
7.1.2 Potential for a Personalized Approach ............................................................................ 58
7.2 Clinical Trial Design .............................................................................................................. 59
7.2.1 Differences in Diagnosis, Monitoring, and Sensitive Imaging Techniques Are Needed ... 60
7.2.2 Patient Recruitment Issues ............................................................................................. 60
7.2.3 Appropriate Endpoints .................................................................................................... 61
7.2.4 Clinical Regulatory Guidelines ........................................................................................ 62
8 Pipeline Assessment .................................................................................................................. 64
8.1 Overview .............................................................................................................................. 64
8.2 Promising Drugs in Clinical Development ............................................................................. 65
8.2.1 GFT505 .......................................................................................................................... 65
8.2.2 Obeticholic Acid (INT-747) .............................................................................................. 69
8.2.3 Simtuzumab (Formerly GS-6624) ................................................................................... 74
8.2.4 Liraglutide (Victoza) ........................................................................................................ 77
8.3 Innovative Early-Stage Approaches ...................................................................................... 81
8.3.1 Targeting Cytokines ........................................................................................................ 83
9 Pipeline Valuation Analysis ........................................................................................................ 85
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Table of Contents
9.1 Clinical Benchmark of Key Pipeline Drugs ............................................................................ 85
9.2 Commercial Benchmark of Key Pipeline Drugs .................................................................... 88
9.3 Competitive Assessment ...................................................................................................... 90
9.4 Top-Line Five-Year Forecast ................................................................................................ 90
9.4.1 US .................................................................................................................................. 94
9.4.2 5EU ................................................................................................................................ 95
10 Appendix .................................................................................................................................... 97
10.1 Bibliography.......................................................................................................................... 97
10.2 Abbreviations...................................................................................................................... 106
10.3 Methodology ....................................................................................................................... 109
10.4 Forecasting Methodology ................................................................................................... 109
10.4.1 Diagnosed NASH Patients ............................................................................................ 109
10.4.2 Percent Drug-Treated Patients ..................................................................................... 110
10.4.3 Drugs Included in Each Therapeutic Class ................................................................... 110
10.4.4 Launch Dates ............................................................................................................... 111
10.4.5 General Pricing Assumptions ........................................................................................ 111
10.4.6 Individual Off-Label Drug Assumptions ......................................................................... 112
10.4.7 Pricing of Pipeline Agents ............................................................................................. 112
10.5 Physicians and Specialists Included in this Study ............................................................... 113
10.6 About the Authors ............................................................................................................... 114
10.6.1 Author ........................................................................................................................... 114
10.6.2 Epidemiologist .............................................................................................................. 115
10.6.3 Global Head of Healthcare ............................................................................................ 115
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Table of Contents
10.7 About GlobalData ............................................................................................................... 117
10.8 Disclaimer .......................................................................................................................... 117
1.1 List of Tables
Table 1: Symptoms of NASH ....................................................................................................................... 20
Table 2: Risk Factors and Comorbidities for NASH ...................................................................................... 22
Table 3: Reported Prevalence of NASH in the US and 5EU ......................................................................... 26
Table 4: Reported Prevalence of NAFLD in the US and 5EU ....................................................................... 26
Table 5: 6MM, Sources of Prevalence Data Used in the NASH Epidemiological Forecast ............................ 30
Table 6: 6MM, Prevalent Cases of NASH, Ages ≥20 Years, Men and Women, N, Selected Years, 2012–2022 .............................................................................................................................................. 37
Table 7: 6MM, Prevalent Cases of NASH, By Age*, Men and Women, N (Row %), 2012 ............................. 38
Table 8: 6MM, Prevalent Cases of NASH, Ages ≥20 Years, By Sex, N (Row %), 2012 ................................ 40
Table 9: Off-Label Treatments for NASH ..................................................................................................... 46
Table 10: Product Profile – Vitamin E ............................................................................................................ 47
Table 11: Vitamin E SWOT Analysis in NASH, 2013 ...................................................................................... 49
Table 12: Product Profile – Pioglitazone ........................................................................................................ 51
Table 13: Pioglitazone SWOT Analysis in NASH, 2013 .................................................................................. 53
Table 14: Overall Unmet Needs in NASH – Current Level of Attainment ........................................................ 55
Table 15: Current Clinical Trial Design of Key Pipeline Drugs for NASH, 2013 ............................................... 63
Table 16: NASH – Mid-Late-Stage Pipeline, 2013 .......................................................................................... 65
Table 17: Product Profile – GFT505............................................................................................................... 67
Table 18: GFT505 SWOT Analysis, 2013 ...................................................................................................... 69
Table 19: Product Profile – Obeticholic Acid (INT-747)................................................................................... 71
Table 20: Obeticholic Acid (INT-747) SWOT Analysis, 2013 .......................................................................... 73
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Table of Contents
Table 21: Product Profile – Simtuzumab ........................................................................................................ 75
Table 22: Simtuzumab SWOT Analysis, 2013 ................................................................................................ 77
Table 23: Product Profile – Liraglutide ........................................................................................................... 78
Table 24: Liraglutide Safety Profile from the LEAD Program .......................................................................... 80
Table 25: Liraglutide SWOT Analysis, 2013 ................................................................................................... 81
Table 26: Early-Stage Pipeline Products in NASH, 2013 ................................................................................ 82
Table 27: Clinical Benchmark of Key Pipeline Drugs for NASH, 2013 ............................................................ 87
Table 28: Commercial Benchmark of Key Pipeline Drugs for NASH, 2013 .................................................... 89
Table 29: Top-Line Sales Forecasts ($m) for NASH, 2012–2017 ................................................................... 91
Table 30: Key Events Impacting Sales for NASH, 2017 ................................................................................. 93
Table 31: NASH Market: US and 5EU – Drivers and Barriers, 2012–2017 ..................................................... 93
Table 32: Key Launch Dates........................................................................................................................ 111
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Table of Contents
1.2 List of Figures
Figure 1: Two-Hit and Multiple-Hits Theories in NASH ................................................................................. 18
Figure 2: Distinct-Hit Pathogenesis in NASH ............................................................................................... 19
Figure 3: Stages of Liver Disease ................................................................................................................ 20
Figure 4: 5EU, Prevalence of NAFLD and Obesity, Ages ≥20 Years, Men and Women, % ........................... 28
Figure 5: 6MM, Prevalent Cases of NASH, Ages ≥20 Years, Men and Women, N, Selected Years, 2012–
2022 ............................................................................................................................................. 37
Figure 6: 6MM, Prevalent Cases of NASH, By Age*, Men and Women, N, 2012 .......................................... 39
Figure 7: 6MM, Prevalent Cases of NASH, Ages ≥20 Years, By Sex, N, 2012 ............................................. 40
Figure 8: Liver Biopsy to Confirm NASH ...................................................................................................... 45
Figure 9: Competitive Assessment of Mid-Stage Pipeline Agents in NASH, 2012–2017 ............................... 90
Figure 10: Global Sales for NASH by Region, 2012–2017 ............................................................................. 92
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Introduction
2 Introduction
2.1 Catalyst
Currently, the NASH market is very dynamic, with growing interest in the therapeutic area
showcased by these recent market events:
Intercept Pharmaceuticals announced that the Phase II/IIb FLINT trial of its pipeline candidate
OCA had been stopped early, after a planned interim analysis showed that the primary
endpoint of the study had been met. The January 9, 2014 announcement led to a surge in the
company’s share price.
On January 10, 2014 Genfit presented further information on the clinical program of its Phase
IIb pipeline candidate GFT505. This presentation highlighted that Genfit is aligning itself to
register GFT505 among the first approved therapies for NASH.
On February 14, 2014, Genfit announced that the US FDA had granted Fast Track designation
to the GFT505 NASH program.
GlobalData’s assessment of the NASH market revealed that it is extremely sparse, and off-
label therapies such as vitamin E and pioglitazone (Takeda’s Actos) make up the treatment
paradigm. The lack of efficacious therapies specific for treating NASH will allow several
pipeline products to emerge uncontested. This report analyzes the four most advanced pipeline
products, two of which we estimate will launch at the end of the forecast period in 2017. The
four products are:
Genfit’s peroxisome proliferator-activated receptor alpha, delta (PPARa,d) agonist, GFT505.
Anticipated to launch in Q4 2017 in the US and 5EU.
Intercept Pharmaceuticals/Dainippon Sumitomo Pharma’s farnesoid X receptor (FXR),
obeticholic acid (INT-747/DSP-1747). Anticipated to launch in Q4 2017 in the US. We expect
launch in the EU after the forecast period of 2012–2017.
Gilead’s lysyl oxidase-like 2 (LOXL2) inhibitor, simtuzumab.
Novo Nordisk’s glucagon-like peptide-1 (GLP-1), liraglutide (Victoza).
The NASH market is extremely sparse, and off-label therapies such as vitamin E and pioglitazone (Takeda’s Actos) make up the treatment paradigm. The lack of efficacious therapies specific for treating NASH will allow several pipeline products to emerge uncontested
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Introduction
As mentioned, GFT505 and OCA will be virtually uncontested once they enter the market. We
expect each therapy to gain patient share from the three subpopulations (obese, diabetic, and
other [defined as healthy donors]) within NASH. Regardless of the novelty each new therapy brings
to the paradigm, their presence will be welcomed with open arms within the NASH community.
2.2 Related Reports
GlobalData (2013). Type 2 Diabetes – Global Drug Forecast and Market Analysis to 2022, July
2013, GDHC54PIDR.
GlobalData (2013). Obesity – Global Drug Forecast and Market Analysis to 2022, October
2013, GDHC50PIDR.
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Appendix
10.7 About GlobalData
GlobalData is a leading global provider of business intelligence in the Healthcare industry.
GlobalData provides its clients with up-to-date information and analysis on the latest developments
in drug research, disease analysis, and clinical research and development. Our integrated business
intelligence solutions include a range of interactive online databases, analytical tools, reports and
forecasts. Our analysis is supported by a 24/7 client support and analyst team.
GlobalData has offices in New York, Boston, London, India and Singapore.
10.8 Disclaimer
All Rights Reserved.
No part of this publication may be reproduced, stored in a retrieval system or transmitted in any
form by any means, electronic, mechanical, photocopying, recording or otherwise, without the prior
permission of the publisher, GlobalData.