CHRONIC LYMPHOCYTIC LEUKEMIA - OPPORTUNITY ANALYSIS AND FORECASTS TO 2018 · reference code gdhc017poa | publication date june 2014 chronic lymphocytic leukemia - opportunity analysis

REFERENCE CODE GDHC017POA | PUBLICAT ION DATE JUNE 2014

CHRONIC LYMPHOCYTIC LEUKEMIA - OPPORTUNITY ANALYSIS AND FORECASTS TO 2018

Chronic Lymphocytic Leukemia - Opportunity Analysis and Forecasts to 2018 2 © GlobalData. This report is a licensed product and is not to be copied, reproduced, shared or resold in any form.


Executive Summary

The table below presents the key metrics for

chronic lymphocytic leukemia (CLL) in the six

major pharmaceutical markets (6MM) (US, France,

Germany, Italy, Spain, and UK), during the forecast

period from 2013–2018.

Chronic Lymphocytic Leukemia: Key Metrics in the Six Major Pharmaceutical Markets, 2013–2018

2013 Epidemiology

Incident Population 37, 795

2013 Market Sales

US $ 821m

5EU $571m

Total $1.4bn

Key Events (2013–2018) Level of Impact

Loss of MabThera (rituximab) marketing exclusivity in the EU ↓

US approval of Gazyva (obinutuzumab) for first-line CLL in 2013 (EU approval expected in 2014)

↑↑

US and EU approval of Imbruvica (ibrutinib) for relapsed/refractory CLL in 2014

↑↑↑

US and EU approval of idelalisib for relapsed/refractory CLL in 2014 ↑↑↑

Loss of Levact/Treanda (bendamustine) marketing exclusivity in 2014 in the EU, and in 2015 in the US

↓↓

US and EU approval of Imbruvica in the first-line setting in 2016 ↑

US and EU approval of IPI-145 for relapsed/refractory CLL in 2016 ↑

US and EU approval of idelalisib in the first-line setting in 2017 ↑

2018 Market Sales

US $2bn

5EU $1.3bn

Total $3.3bn Source: GlobalData 5EU = France, Germany, Italy, Spain, and UK

New Entrants will Drive Strong Growth in the US and EU CLL Market from 2013–2018

GlobalData estimates the CLL market in the US

and 5EU (France, Germany, Italy, Spain, and UK)

in 2013 to be valued at $1.4 billion. The market is

defined as sales from branded drugs, including

Rituxan (rituximab), Arzerra (ofatumumab),

Treanda (bendamustine) and Gazyva

(obinutuzumab). Although there are more incident

cases of CLL in the 5EU, the majority of sales in

2013, $821m (59%), were generated in the US due

to higher drug prices. Sales in the 5EU were

estimated to be $571m (41%) in 2013.

By the end of the forecast period in 2018, the

market is expected to grow to $3.3 billion at a

Compound Annual Growth Rate (CAGR) of 18.8%.

GlobalData expects the launch of premium-priced

new therapies, such as Gazyva, Imbruvica

(ibrutinib), idelalisib, and IPI-145, for CLL patients

with high unmet needs will drive overall market

growth in the 6MM covered in this report during the

forecast period.

Major drivers of the CLL market in the US and 5EU

during the forecast period will include:

Rapid uptake of four new branded drugs,

including Gazyva, Imbruvica, idelalisib, and

IPI-145

Premium pricing for new branded drugs, as

they address high unmet needs (as already

observed with Imbruvica in the US)



Executive Summary

Dose-continuous regimens of the new targeted

therapies, which means that patients will

remain on drugs longer, instead of receiving a

fixed course of therapy

Use of a combination approach to maximize

the efficacy of new drugs, resulting in

increased sales of branded drugs

Increased total treated patient pool due to

fewer patients being enrolled in clinical trials

and more patients being treated with branded

drugs

A rapidly aging population, which will increase

CLL incidence

Barriers to the growth of the CLL market in the US

and 5EU during the forecast period will include:

Pressure for cost-consciousness, which will

limit premium pricing for new entrants in the

5EU

The loss of marketing exclusivity for Treanda in

CLL (2014 in the EU, and 2015 in the US),

which will occur during a time when its use, in

combination with new drugs, is expected to

grow substantially

The figure below illustrates the global sales for CLL

in the US and 5EU during the forecast period from

2013–2018.

Global Sales for CLL By Region (US and 5EU), 2013–2018

59%

41%

Global CLL Market 2013$1.4bn

60%

40%

US

5EU

Global CLL Market 2018$3.3bn

Source: GlobalData

Imbruvica and Idelalisib will Undergo Rapid Uptake in the Second-Line, Driven by the Lack of Standard Treatment Options and High Unmet Need in this Patient Population

Historically, CLL patients who did not experience a

long response after initial therapy had limited

therapeutic options and a high degree of clinical

unmet need for second-line treatment that could



Executive Summary

extend survival after primary relapse. Because of

the lack of standard-of-care treatment options,

many relapsed/refractory patients are enrolled in

clinical trials for investigative therapies. However,

with the anticipated launch of Imbruvica and

idelalisib in the US and EU in 2014, GlobalData

expects that the treatment of these patients will

undergo significant change. In phase II and III

trials, Imbruvica and idelalisib have shown strong

efficacy and tolerability, which, in correlation with

the high level of unmet need in this patient

population, will drive very rapid uptake of both

drugs. Furthermore, the completion of trials

studying the combination of these new agents with

BR (bendamustine and Rituxan), as well as the

results of studies of Imbruvica and idelalisib in the

first-line treatment of elderly patients, which are

expected by 2016, will contribute towards

establishing these two drugs as the most

successful branded therapies for CLL. With total

combined sales of $2 billion in 2018, GlobalData

forecasts Imbruvica and idelalisib to capture more

than 60% of the total CLL market by then.

Second-to-Market Idelalisib will become the Market Leader in the Relapsed/Refractory Setting in 2018

Despite the fact that idelalisib will launch in the US

in Q3 2014, several months after the approval of

Imbruvica by the Food and Drug Administration

(FDA) in February 2014, GlobalData foresees that

idelalisib will eventually emerge as the market

leader in the relapsed/refractory setting by 2018. In

contrast to Imbruvica, which initially launched as a

monotherapy, idelalisib will be used in combination

with the standard CLL backbone agent, Rituxan.

The ability to combine idelalisib with Rituxan

represents a safer alternative for

relapsed/refractory patients who are unfit to

receive chemoimmunotherapy regimens.

GlobalData believes this will provide idelalisib with

a significant advantage over Imbruvica, which has

demonstrated an inhibitory effect on anti-CD20-

mediated cytotoxicity, suggesting that it may be

incompatible in combination with anti-CD20

antibodies such as Rituxan. Furthermore, safety

concerns regarding Imbruvica’s interaction with

anticoagulants and antiplatelet drugs will further

limit its uptake among elderly patients. In the US,

GlobalData foresees idelalisib’s patient share in

the relapsed/refractory setting to overtake

Imbruvica’s share by 2017. Imbruvica’s sales will,

however, remain higher during the forecast period

because of its premium pricing over idelalisib. In

Europe, although growing pressure to reduce

healthcare expenditures is likely to constitute a

hurdle to obtaining premium pricing for an agent

being used in combination with a drug that is

already expensive, such as Rituxan, GlobalData

believes that idelalisib will capture both greater

patient and market shares than Imbruvica, as

Imbruvica will not have the same time-to-market

advantage in Europe. In 2018, sales of Imbruvica

and idelalisib in the 6MM are forecast to reach $1b

and $984m, respectively.



Executive Summary

The figure below provides a competitive

assessment of the key marketed and pipeline

drugs in the first-line treatment of CLL.

Competitive Assessment of Key Marketed and Pipeline Drugs – First-Line CLL

2.50

3.00

3.50

2.50 3.00 3.50

Com

mer

cial

Sco

re

Arzerra

Gazyva

Chl+R FCR Imbruvica

idelalisib

Clinical Score

Source: GlobalData

The figure below provides a competitive

assessment of the key marketed and pipeline

drugs in the treatment of relapsed/refractory CLL.

Competitive Assessment of Key Marketed and Pipeline Drugs – Relapsed/Refractory CLL

2.0

2.5

3.0

3.5

2.0 2.5 3.0 3.5 4.0

Com

mer

cial

Scor

e

Arzerra

BR

IPI-145

Imbruvicaidelalisib

Clinical Score Source: GlobalData

Uptake of the New Agents Will be Slower in the First-Line Setting, Limited by Reduced Uptake Among Younger CLL Patients

GlobalData expects that the uptake of the new

agents, idelalisib and Imbruvica, will be slower in

the first-line setting, where high rates of use of the

gold-standard FCR (fludarabine,

cyclophosphamide, and Rituxan) regimen among

young, fit CLL patients will limit the ability of these

new agents to penetrate the first-line market.

GlobalData’s primary research revealed high levels

of satisfaction with the safety and efficacy profile of

FCR in this patient population, and so far, drug

developers have largely refrained from making any

direct comparisons with FCR in their clinical

development programs. Instead, GlobalData

anticipates that these new agents will be targeted

at the elderly patient population. However, data

from their late-stage trials will not be available until

June 2015 (Imbruvica) and June 2016 (idelalisib).

GlobalData expects that Gazyva, which is already

approved in the first-line setting in the US, will also

have a slower uptake, as its approval in

combination with chlorambucil may limit its initial

use. However, this could change when data from a

Phase III trial investigating its use in combination

with FC (fludarabine and cyclophosphamide) and

bendamustine become available in 2016.

Furthermore, as Roche’s blockbuster Rituxan

faces 5EU and US patent expirations in 2013 and

2018, respectively, GlobalData anticipates that the

company’s formidable franchise management skills



Executive Summary

will ensure the evolution of CLL management from

Rituxan to Gazyva over the forecast period. Thus,

despite the clear demonstration of Gazyva’s

clinical superiority to Rituxan, GlobalData expects

that Gazyva will capture 20% of all first-line patient

share by 2018.

What Do the Physicians Think?

CLL experts interviewed by GlobalData were

enthusiastic about the pipeline agents, particularly

as they are keen to get away from using

chemotherapy-based regimens in the vastly elderly

CLL population. Although chemoimmunotherapy

regimens such as FCR can provide high efficacy

and long-term disease control in young or fit CLL

patients, the short and long-term toxicities

associated with the use of fludarabine are not

viewed favorably by either patients or physicians.

“Intensive chemoimmunotherapy, that’s not what

people are interested in at all. The issue is getting

away from chemotherapy.”

US Key Opinion Leader, March 2014

“You need to be minded [mindful] of the long-term

toxicities of FCR, such as myelodysplasia and

acute leukemia. There have been a percentage of

cases [involving these diseases], so imagine if you

are going to give that [FCR] to patients who have

[already] received that [FCR] twice, for example;

that could be a problem. So, I think in those

patients, probably one will try to use one of the

newer agents.”

European Key Opinion Leader, March 2014

While KOLs had favorable impressions of

Imbruvica and idelalisib, they acknowledged that,

due to the present lack of mature data, they could

not be certain of the long-term benefit provided by

these drugs. For example, these new agents have

not yet demonstrated a significant benefit in

minimal residual disease (MRD) negativity, raising

the possibility that they may not be able to induce

lengthy remissions similar to those seen with

standard FCR therapy. Thus, until mature data

suggest otherwise, or trials to compare the new

agents with FCR are conducted, KOLs expressed

hesitancy to replace FCR as the first-line treatment

of choice in young or fit CLL patients. GlobalData

thus expects that the use of Imbruvica and

idelalisib will initially be largely reserved for elderly,

17p, or relapsed/refractory patients for whom other

efficacious therapeutic options are otherwise

lacking.

“FCR will likely remain the treatment of choice for

younger, fit, ‘go-go’ patients. New agents will be

used for older patients and those [who are] unfit for

FCR. Trials [are] need[ed] to determine the role of

the new agents in unfit patients, first, and if they

work, trials [will be needed] to compare FCR with

new agents as first-line therapy in all kinds of

patients….It will take time.”

European Key Opinion Leader, February 2014



Executive Summary

“So, we know that if you give FCR, and you

achieve complete remission, you are within the

group of patients who are what we call MRD-

negative. Their remission is deeper; it’s greater

quality. These patients are going to do extremely

well with nothing else for periods of five to 10 years

without any further treatments. But, with the

ibrutinib-type agent, you don’t seem to get…or it’s

not clear whether you get, negative minimal

residual disease so frequently. There may be a

percentage of patients [who do get negative MRD],

but [it is] very small, and you need to treat [them]

continuously for a very long period time.”

European Key Opinion Leader, March 2014

“I would really need a trial comparing front-line

FCR and front-line FC-Gazyva, and showing that

there is really a benefit in giving FC-Gazyva. So,

unless we…have such a trial, I am not sure that

the choice of Gazyva, knowing the price and

knowing the possibility to use subcutaneous

rituximab, will soon be so high.”

European Key Opinion Leader, February 2014

While it is difficult to predict exactly what role each

of the new agents will occupy in the future CLL

treatment paradigm, GlobalData anticipates that

the management of CLL will continue to evolve

rapidly, especially as physicians are confident that

they now have the necessary tools to achieve

better responses in a wider variety of CLL patients.

“We have just been presented with a whole bunch

of fantastic new tools that we are only beginning to

learn how to use. We don’t know how to use them

in combination. There is going to be a whole lot of

unexpected findings along the way that are going

to make us change our thinking. We have got a big

project ahead of us. We have got much better

tools. Our patients are probably going to do better,

but I would be very foolish to make any real

predictions.”

US Key Opinion Leader, April 2014


Table of Contents


1 Table of Contents

1 Table of Contents ....................................................................................................................... 8

1.1 List of Tables .................................................................................................................... 12

1.2 List of Figures ................................................................................................................... 14

2 Introduction ............................................................................................................................... 16

2.1 Catalyst ............................................................................................................................. 16

2.2 Related Reports ................................................................................................................ 16

2.3 Upcoming Related Reports ............................................................................................... 17

3 Disease Overview ..................................................................................................................... 18

3.1 Etiology ............................................................................................................................. 18

3.2 Pathophysiology ................................................................................................................ 18

3.3 Staging and Prognostic Markers ....................................................................................... 19

3.3.1 Rai and Binet Staging System ....................................................................................... 19

3.3.2 Chromosomal Abnormalities and Other Prognostic Markers ......................................... 20

3.4 Symptoms ......................................................................................................................... 21

3.5 Quality of Life .................................................................................................................... 22

4 Disease Management ............................................................................................................... 23

4.1 Diagnosis .......................................................................................................................... 23

4.2 Treatment Overview .......................................................................................................... 23

4.2.1 First-Line Treatment ...................................................................................................... 25

4.2.2 Second-Line Treatment and Beyond ............................................................................. 26

4.3 Response Criteria for Evaluating the Treatment Outcome................................................. 26

5 Epidemiology ............................................................................................................................ 28

5.1 Disease Background ......................................................................................................... 28

5.2 Risk Factors and Comorbidities ........................................................................................ 29

5.3 Global Trends ................................................................................................................... 31

5.3.1 Incidence ....................................................................................................................... 31


Table of Contents


5.3.2 Survival from CLL – US and 5EU .................................................................................. 34

5.4 Forecast Methodology ....................................................................................................... 34

5.4.1 Sources Used................................................................................................................ 36

5.4.2 Sources Not Used ......................................................................................................... 38

5.4.3 Forecast Assumptions and Methods, Diagnosed Incident Cases .................................. 38

5.4.4 Forecast Assumptions and Methods, 5-Year Diagnosed Prevalent Cases .................... 40

5.4.5 Forecast Assumptions and Methods, Rai Stage at Diagnosis ........................................ 40

5.5 Epidemiological Forecast for CLL (2013–2023)................................................................. 41

5.5.1 Diagnosed Incident Cases of CLL ................................................................................. 41

5.5.2 Age-Specific Diagnosed Incident Cases of CLL ............................................................ 42

5.5.3 Sex-Specific Diagnosed Incident Cases of CLL ............................................................. 44

5.5.4 Age-Standardized Diagnosed Incidence of CLL ............................................................ 45

5.5.5 5-Year Diagnosed Prevalent Cases of CLL ................................................................... 46

5.5.6 Diagnosed Incident Cases of CLL by Rai Stage at Diagnosis ........................................ 48

5.6 Discussion ........................................................................................................................ 48

5.6.1 Epidemiological Forecast Insight ................................................................................... 48

5.6.2 Limitations of the Analysis ............................................................................................. 49

5.6.3 Strengths of the Analysis ............................................................................................... 50

6 Current Treatment Options ....................................................................................................... 51

6.1 Overview ........................................................................................................................... 51

6.2 Product Profiles – Major Brands ........................................................................................ 52

6.2.1 Rituxan/MabThera (rituximab) ....................................................................................... 52

6.2.2 Arzerra (ofatumumab) ................................................................................................... 56

6.2.3 Campath (alemtuzumab) ............................................................................................... 60

6.2.4 Treanda/Levact (bendamustine) .................................................................................... 63

6.2.5 Gazyva/Gazyvaro (obinutuzumab) ................................................................................ 66

6.2.6 Imbruvica (ibrutinib) ....................................................................................................... 69


Table of Contents


7 Unmet Needs Assessment and Oppportunity Analysis ............................................................. 75

7.1 Overview ........................................................................................................................... 75

7.2 Unmet Needs Analysis ...................................................................................................... 76

7.2.1 Unmet Need: Therapies that are tolerable for elderly, unfit patients .............................. 76

7.2.2 Unmet Need: Efficacious Therapies for High-Risk 17p Deletion Patients ...................... 77

7.2.3 Unmet Need: Safe and Efficacious Therapeutic Options for Relapsed/Refractory Patients ........................................................................................................................ 78

7.2.4 Unmet Need: Prognostic Markers to Determine the Best Treatment Strategy ............... 79

7.2.5 Unmet Need: Clinical Trials Designed to Reflect the Average CLL Patient .................... 80

7.3 Opportunity Analysis ......................................................................................................... 80

7.3.1 Opportunity: Development of Better Tolerated Therapies .............................................. 80

7.3.2 Opportunity: Development of Combination Therapy ...................................................... 81

7.3.3 Opportunity: Increasing the Cost-Effectiveness of New Therapies ................................ 81

7.3.4 Opportunity: Development of a Prognostic Index ........................................................... 82

7.3.5 Opportunity: Identification of Patients Who Could Benefit from Early Treatment ............ 83

8 R&D Strategies ......................................................................................................................... 84

8.1 Overview ........................................................................................................................... 84

8.1.1 Moving Towards a Chemotherapy-Free Future ............................................................. 84

8.1.2 Targeting the High-Risk 17p Deletion Population .......................................................... 85

8.1.3 Targeting the B-Cell Receptor Signaling Pathway in CLL .............................................. 85

8.1.4 Combination Therapy .................................................................................................... 87

8.1.5 Oral Formulations .......................................................................................................... 88

8.2 Clinical Trial Design .......................................................................................................... 89

8.2.1 Re-Assessing Treatment Outcome Criteria to Support the Development of New Agents to Treat CLL ................................................................................................................. 89

8.2.2 Evaluating CLL Clinical Trial Endpoints ......................................................................... 90

8.2.3 Selecting Suitable Comparator Arms and Combinations ............................................... 91

8.2.4 Current Clinical Trial Design .......................................................................................... 93


Table of Contents


9 Pipeline Assessment................................................................................................................. 95

9.1 Overview ........................................................................................................................... 95

9.2 Promising Drugs in Clinical Development .......................................................................... 96

9.2.1 Idelalisib (GS-1101, CAL-101) ....................................................................................... 96

9.2.2 IPI-145 ........................................................................................................................ 101

9.2.3 ABT-199 ...................................................................................................................... 104

9.3 Innovative Early-Stage Approaches ................................................................................ 108

9.3.1 CTL019 ....................................................................................................................... 109

9.3.2 Otlertuzumab (TRU-016) ............................................................................................. 110

9.3.3 Afuresertib ................................................................................................................... 111

9.3.4 GS-9973 ...................................................................................................................... 112

9.3.5 NOX-A12 ..................................................................................................................... 113

10 Pipeline Valuation Analysis ..................................................................................................... 114

10.1 Clinical Benchmark of Key Pipeline Drugs ...................................................................... 114

10.2 Commercial Benchmark of Key Pipeline Drugs ............................................................... 117

10.3 Competitive Assessment ................................................................................................. 118

10.4 Top-Line Five-Year Forecast ........................................................................................... 120

10.4.1 US ............................................................................................................................... 123

10.4.2 5EU ............................................................................................................................. 123

11 Appendix................................................................................................................................. 124

11.1 Bibliography .................................................................................................................... 124

11.2 Abbreviations .................................................................................................................. 138

11.3 Methodology ................................................................................................................... 142

11.4 Forecasting Methodology ................................................................................................ 142

11.4.1 Diagnosed CLL Patients .............................................................................................. 142

11.4.2 Percentage of Drug-Treated Patients .......................................................................... 143

11.4.3 Drugs Included in Each Therapeutic Class .................................................................. 143


Table of Contents


11.4.4 Launch and Patent Expiry Dates ................................................................................. 144

11.4.5 General Pricing Assumptions ...................................................................................... 144

11.4.6 Individual Drug Assumptions ....................................................................................... 145

11.4.7 Generic Erosion .......................................................................................................... 147

11.4.8 Pricing of Pipeline Agents............................................................................................ 147

11.5 Physicians and Specialists Included in This Study .......................................................... 148

11.6 About the Authors ........................................................................................................... 149

11.6.1 Authors........................................................................................................................ 149

11.6.2 Epidemiologist ............................................................................................................. 150

11.6.3 Global Head of Healthcare .......................................................................................... 151

11.7 About GlobalData ............................................................................................................ 152

11.8 Disclaimer ....................................................................................................................... 152

1.1 List of Tables

Table 1: Rai and Binet Staging of CLL....................................................................................................... 20

Table 2: iwCLL Symptoms of Progressive CLL .......................................................................................... 22

Table 3: Definitions of the Response to Treatment of CLL ......................................................................... 27

Table 4: CLL Clinical Stages at Diagnosis ................................................................................................. 29

Table 5: Risk Factors and Comorbidities for CLL ....................................................................................... 30

Table 6: Trends in the Age-Adjusted Incidence of CLL in the US, All Ages, 2003–2010 ............................. 32

Table 7: Trends in the Age-Adjusted Incidence of CLL (Cases per 100,000 Population) in France, All Ages,

1980–2012 .................................................................................................................................. 33

Table 8: Trends in the 5-Year Relative Survival (%) of CLL in the US and 5EU, Both Sexes, 1993–2009... 34

Table 9: Sources of Epidemiological Data Used to Forecast the CLL Diagnosed Incident and Prevalent

Cases, and the Rai Stage at Diagnosis ........................................................................................ 35

Table 10: 6MM, Diagnosed Incident Cases of CLL, Both Sexes, Ages ≥40 Years, N, 2013–2023 ................ 41


Table of Contents


Table 11: 6MM, Age-Specific Diagnosed Incident Cases of CLL, Both Sexes, N (Row %), 2013 ................. 43

Table 12: 6MM, Sex-Specific Diagnosed Incident Cases of CLL, Ages ≥40 Years, N (Row %), 2013 ........... 44

Table 13: 6MM, 5-Year Diagnosed Prevalent Cases of CLL, Both Sexes, Ages ≥40 Years, N, 2013–2023 .. 47

Table 14: Leading Treatments for CLL ........................................................................................................ 52

Table 15: Product Profile – Rituxan ............................................................................................................. 54

Table 16: Rituxan SWOT Analysis .............................................................................................................. 56

Table 17: Product Profile – Arzerra ............................................................................................................. 58

Table 18: Arzerra SWOT Analysis ............................................................................................................... 60

Table 19: Product Profile – Campath........................................................................................................... 61

Table 20: Campath SWOT Analysis ............................................................................................................ 62

Table 21: Product Profile – Treanda ............................................................................................................ 64

Table 22: Treanda SWOT Analysis ............................................................................................................. 65

Table 23: Product Profile – Gazyva ............................................................................................................. 67

Table 24: Gazyva SWOT Analysis .............................................................................................................. 69

Table 25: Product Profile – Imbruvica .......................................................................................................... 72

Table 26: Imbruvica SWOT Analysis ........................................................................................................... 74

Table 27: Overall Unmet Needs – Current Level of Attainment .................................................................... 76

Table 28: BCR Signaling Pathway Inhibitors in Clinical Development in CLL ............................................... 87

Table 29: Design of Current Phase III Trials in CLL ..................................................................................... 93

Table 30: CLL – Late-Stage Pipeline, 2014 ................................................................................................. 96

Table 31: Product Profile – Idelalisib (GS-1101, CAL-101) .......................................................................... 99

Table 32: Idelalisib SWOT Analysis........................................................................................................... 101

Table 33: Product Profile – IPI-145 ........................................................................................................... 102

Table 34: IPI-145 SWOT Analysis ............................................................................................................. 104


Table of Contents


Table 35: Product Profile – ABT-199 ......................................................................................................... 106

Table 36: ABT-199 SWOT Analysis .......................................................................................................... 108

Table 37: Early-Stage Pipeline Products in CLL ........................................................................................ 109

Table 38: Clinical Benchmark of Key Pipeline Drugs – First-Line CLL ........................................................ 115

Table 39: Clinical Benchmark of Key Pipeline Drugs – Relapsed/Refractory CLL ...................................... 116

Table 40: Commercial Benchmark of Key Pipeline Drugs .......................................................................... 117

Table 41: Top-Line Sales Forecasts ($) for CLL, 2013–2018 ..................................................................... 121

Table 42: Key Events Impacting Sales for CLL, 2013–2018 ...................................................................... 122

Table 43: CLL Market in the US and 5EU – Drivers and Barriers, 2013–2018............................................ 122

Table 44: Key Launch Dates ..................................................................................................................... 144

Table 45: Key Patent Expiries ................................................................................................................... 144

1.2 List of Figures

Figure 1: Treatment Algorithm for CLL ....................................................................................................... 24

Figure 2: 6MM, Diagnosed Incident Cases of CLL, Both Sexes, Ages ≥40 Years, N, 2013–2023 ................ 42

Figure 3: 6MM, Age-Specific Diagnosed Incident Cases of CLL, Both Sexes, N, 2013 ................................ 43

Figure 4: 6MM, Sex-Specific Diagnosed Incident Cases of CLL, Ages ≥40 Years, N, 2013 ......................... 45

Figure 5: 6MM, Age-Standardized Diagnosed Incidence of CLL (Cases per 100,000 Population), Ages ≥40

Years, by Sex, 2013 .................................................................................................................... 46

Figure 6: 6MM, 5-Year Diagnosed Prevalent Cases of CLL, Both Sexes, Ages ≥40 Years, N, 2013–2023 .. 47

Figure 7: 6MM, Diagnosed Incident Cases of CLL by Rai Stage at Diagnosis, Ages ≥40 Years, N, 2013 .... 48

Figure 8: Gazyva– Phase II and III Trials .................................................................................................... 68

Figure 9: Imbruvica – Phase II and III Trials................................................................................................ 72

Figure 10: The BCR Signaling Pathway in CLL ............................................................................................ 86


Table of Contents


Figure 11: The Evolving CLL Treatment Landscape ..................................................................................... 88

Figure 12: Idelalisib – Phase II and III Trials ................................................................................................. 97

Figure 13: IPI-145 – Phase Ib and III Trials ................................................................................................ 103

Figure 14: ABT-199 – Phase II and III Trials ............................................................................................... 106

Figure 15: Competitive Assessment of Key Marketed and Pipeline Drugs – First-Line CLL ......................... 119

Figure 16: Competitive Assessment of Key Marketed and Pipeline Drugs – Relapsed/Refractory CLL ........ 120

Figure 17: Global Sales for CLL by Region (US and 5EU), 2013–2018 ....................................................... 121



Introduction

2 Introduction

2.1 Catalyst

In the past decade, chronic lymphocytic leukemia (CLL) treatment has primarily relied upon

chemoimmunotherapy regimens. However, the global CLL market will welcome four new drugs

during the forecast period between 2013 to 2018 – Gazyva (obinutuzumab), Imbruvica (ibrutinib),

idelalisib, and IPI-145 – that will dramatically alter the CLL treatment landscape and challenge the

dominance of chemoimmunotherapy regimens. In particular, GlobalData expects patients with high

unmet needs, such as the elderly and relapsed/refractory patients, will have significantly improved

therapeutic options leading to rapid uptake of these drugs. In addition, the premium price these

drugs will demand coupled with a rapidly aging population will altogether drive robust growth of the

CLL market.

2.2 Related Reports

GlobalData (2014). Non-Small Cell Lung Cancer (NSCLC) – Global Drug Forecast and Market

Analysis to 2022 – Event-Driven Update, April 2014, GDHC002EPIDR.

GlobalData (2014). Pancreatic Cancer – Opportunity Analysis and Forecasts to 2017, March

2014, GDHC016POA.

GlobalData (2013). Bladder Cancer – Opportunity Analysis and Forecasts to 2017, December

2013, GDHC014POA.

GlobalData (2013). Acute Myeloid Leukemia (AML) – Opportunity Analysis and Forecasts to

2017, August 2013, GDHC003POA.

GlobalData (2013). Chronic Myeloid Leukemia (CML) – Global Drug Forecast and Market

Analysis to 2022, April 2013, GDHC103PIDR.



Introduction

2.3 Upcoming Related Reports

GlobalData (2014). Non-Hodgkin’s Lymphoma (NHL) – Opportunity Analysis and Forecasts to

2018.

GlobalData (2014). HER2+ Breast Cancer – Global Drug Forecast and Market Analysis to

2023.

GlobalData (2014). Renal Cell Carcinoma (RCC) – Global Drug Forecast and Market Analysis

to 2023.

GlobalData (2014). HER2- Breast Cancer – Global Drug Forecast and Market Analysis to

2023.


Appendix


11.7 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, San Francisco, Boston, London, India, Korea, Japan,

Singapore, and Australia.

11.8 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.

Documents

CHRONIC LYMPHOCYTIC LEUKEMIA - OPPORTUNITY ANALYSIS AND FORECASTS TO 2018 · reference code gdhc017poa | publication date june 2014 chronic lymphocytic leukemia - opportunity analysis