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Q2 2018 ResultsInvestor Presentation

July 18, 2018

Novartis AG

Investor Relations

Disclaimer

Novartis Q2 2018 Results | July 18, 2018 | Novartis Investor Presentation 2

This presentation contains forward-looking statements within the meaning of the United States Private Securities Litigation Reform Act of 1995, that can generally be identified by words such as “potential,” “expected,”

“will,” “planned,” “pipeline,” “outlook,” or similar expressions, or by express or implied discussions regarding potential new products, potential new indications for existing products, or regarding potential future revenues

from any such products; or regarding the proposed 100% spinoff of the Alcon Division, including express or implied discussions regarding the potential financial or other impact on Novartis, and the potential strategic

benefits, synergies or opportunities expected as a result of the proposed spinoff; or regarding the potential impact on Novartis of the completed acquisition of AveXis Inc., including express or implied discussions

regarding potential future sales or earnings of Novartis, and any potential strategic benefits, synergies or opportunities expected from the acquisition; or regarding the potential financial or other impact of the other

significant acquisitions and reorganizations of recent years; or regarding the potential impact of the share buyback; or regarding potential future sales or earnings of the Novartis Group or any of its divisions or potential

shareholder returns; or by discussions of strategy, plans, expectations or intentions. You should not place undue reliance on these statements. Such forward looking statements are based on our current beliefs and

expectations regarding future events, and are subject to significant known and unknown risks and uncertainties. Should one or more of these risks or uncertainties materialize, or should underlying assumptions prove

incorrect, actual results may vary materially from those set forth in the forward looking statements. There can be no guarantee that any new products will be approved for sale in any market, or that any new indications

will be approved for any existing products in any market, or that any approvals which are obtained will be obtained at any particular time, or that any such products will achieve any particular revenue levels. Neither can

there be any guarantee that the proposed 100% spinoff of the Alcon Division will be approved by our shareholders, or that it will be completed, or completed as currently proposed, or at any particular time. Nor can

there be any guarantee that Novartis will be able to realize any of the potential strategic benefits, synergies or opportunities as a result of the proposed 100% spinoff of the Alcon Division, or that the proposed spinoff

will in fact maximize shareholder value. Neither can there be any guarantee that Novartis will be able to realize any of the potential strategic benefits, synergies or opportunities as a result of the significant acquisitions

and reorganizations of recent years. Nor can there be any guarantee that shareholders will achieve any particular level of shareholder returns. Neither can there be any guarantee that the Group, or any of its divisions,

will be commercially successful in the future, or achieve any particular credit rating or financial results. In particular, our expectations could be affected by, among other things: global trends toward health care cost

containment, including government, payor and general public pricing and reimbursement pressures and requirements for increased pricing transparency; regulatory actions or delays or government regulation generally,

including potential regulatory actions or delays with respect to the development of the products described in this presentation; the potential that the proposed 100% spinoff of the Alcon Division may not be approved by

our shareholders, or that it may not be completed, or completed as currently proposed, or at any particular time; the potential that the strategic benefits, synergies or opportunities expected from the proposed 100%

spinoff of the Alcon Division may not be realized or may take longer to realize than expected, or that the proposed spinoff may not in fact maximize shareholder value; the potential that the strategic benefits, synergies

or opportunities expected from the significant acquisitions and reorganizations of recent years may not be realized or may take longer to realize than expected; the inherent uncertainties involved in predicting

shareholder returns; the uncertainties inherent in the research and development of new healthcare products, including clinical trial results and additional analysis of existing clinical data; our ability to obtain or maintain

proprietary intellectual property protection, including the ultimate extent of the impact on Novartis of the loss of patent protection and exclusivity on key products which commenced in prior years and will continue this

year; safety, quality or manufacturing issues; uncertainties regarding actual or potential legal proceedings, including, among others, actual or potential product liability litigation, litigation and investigations regarding

sales and marketing practices, intellectual property disputes and government investigations generally; uncertainties involved in the development or adoption of potentially transformational technologies and business

models; general political and economic conditions, including uncertainties regarding the effects of ongoing instability in various parts of the world; uncertainties regarding future global exchange rates; uncertainties

regarding future demand for our products; and uncertainties regarding potential significant breaches of data security or data privacy, or disruptions of our information technology systems; and other risks and factors

referred to in Novartis AG’s current Form 20-F on file with the US Securities and Exchange Commission. Novartis is providing the information in this presentation as of this date and does not undertake any obligation to

update any forward-looking statements as a result of new information, future events or otherwise.

Agenda

1 Group review Vas Narasimhan

2 Financial review Harry Kirsch

3 Closing Vas Narasimhan

4 Q&A Team


4

Continuing our transformation into a focused medicines company

Novartis Q2 2018 Results | July 18, 2018 | Novartis Investor Presentation

Focusing as a medicines company:

Completed divestment of OTC JV stake to GSK

Proposed 100% spinoff of Alcon Division1

Prioritizing therapeutic areas (e.g. exit of

infectious disease research)

Building our innovative portfolio and capabilities:

Completed AAA acquisition

Completed AveXis acquisition

Licensed Luxturna® ex-US from Spark

2017 post Alcon

spinoff

24%

29%

47%

3%

5%

13%

19%

20%

38%

2%

Vaccines3OTCAlcon2

SandozOncology BUPharmaceuticals BU

Animal Health

2014 pre portfolio

transformation

See appendix slide 34 for references

Group net sales by division (restated)

Solid growth with operating leverage in Q2


1. Core results, constant currencies and free cash flow are non-IFRS measures. Further details regarding non-IFRS measures can be found starting on page 54 of the Condensed Interim Financial Report

Sales Core operating income

Group1 +5% +7%

Innovative Medicines +8% +12%

Sandoz -2% -5%

Alcon +5% +14%

51

76

16

53

129

211

82

68

Continued momentum of key growth drivers in Innovative Medicines (+8% cc)

6 Novartis Q2 2018 Results | July 18, 2018 | Novartis Investor Presentation

Q2 sales USD million

vs. PYcc

Growth vs. PYUSD million

701

239

292

76

284

239

59

16

40%

113%

38%

nm2

28%

24%

nm2

nm2

1. AAA total includes sales of Lutathera® and radiopharmaceutical diagnostic products obtained in the acquisition of Advanced Accelerator Applications S.A. 2. Not meaningful

1

http://www.novartisoncology.com/products/jakavi.jsp

Cosentyx®: Leading IL-17A inhibitor showed strong growth in Q2, driven by underlying demand


Q2 sales of USD 701m (+40% cc)

Strong US performance driven by demand and

enhanced access

– Q2 TRx growth +81%1 increase vs. PY

US label update to include inhibition of joint

structural damage progression in PsA at week 24

vs. placebo

Sales evolutionUSD m, % cc

Q2 2017 Q2 2018

490

701

Ex-US

US

+40%

See appendix slide 34 for references

Entresto®: Standard of care in heart failure treatment saw sales more than double in Q2


Q2 sales of USD 239m (+113% cc)

Underlying demand remains strong in the US, with

robust NBRx and Q2 TRx up +82% vs. PY1

Solid ex-US sales of USD 110m (+150% cc)

Newsflow supporting Entresto® momentum:

– RWE from CHAMP-HF registry showed improvements in

symptom frequency and QoL with Entresto®2

– PARAGON interim analysis planned in Q3; trial expected

to continue to completion in 2019

– Phase IV studies, e.g. TRANSITION (in-hospital initiation),

to report in H2 2018

Sales evolutionUSD m, % cc

Q2 2017 Q2 2018

110

239

Ex-US

US

+113%

RWE – real world evidence See appendix slide 34 for references

Aimovig® (erenumab): First-in-class migraine prevention drug off to a strong start in the US


FDA approved on May 17 for the preventive treatment of

migraine in adults

High unmet need reflected in strong patient demand and

physician activation

Access secured with key plans (incl. ESI Preferred National

Formulary and Anthem)

CHMP positive opinion on May 31; approval expected Q3

Australia registration July 3; Switzerland approved July 13

See appendix slide 34 for references Aimovig® is in collaboration with Amgen. Companies co-commercialize in the US (Amgen books sales to third parties), Novartis has Aimovig® exclusive rights in rest of world excluding Japan

US patients (cumulative NBRx)1

185 947

2,481

4,622

6,771

8,916

5/25/18 6/1/18 6/8/18 6/15/18 6/22/18 6/29/18

Oncology grew +10% (cc), behind continued strong performance of key growth drivers

10

Promacta®/Revolade®

292

210

+38%

Q2 2018Q2 2017

Tafinlar® + Mekinist®

284

216

Q2 2018

+28%

Q2 2017

239

186

Q2 2018

+24%

Q2 2017

Jakavi®

Thrombopoietin receptor agonist

with the broadest indication

Received FDA Priority Review

for 1L use in SAA

Market leader in BRAF+

targeted therapy

Received FDA approval for

adjuvant melanoma

Market leader in myelofibrosis in

Europe

Polycythemia vera potential at

least as big as myelofibrosis


USD m, % cc

Executing against recent Oncology launches in complex and competitive environments

Novartis Q2 2018 Results | July 18, 2018 | Novartis investor presentation 11

- 1

51 67

101

140

Jan Feb Mar Apr May Jun

Q2 sales of USD 59m

Positioned well to grow in Europe, with

reimbursement in UK, ES, DE

Rolling out refined messages and targeting in the

US

Completed MONALEESA 3 & 7 submissions in Q2

Lutathera®

US new patients

Kisqali®

EU countries launched

Kymriah®

Global regulatory status

Q2 sales of USD 16m

Approved in US for r/r DLBCL

Received positive CHMP opinion for both r/r

pediatric & young adult ALL and r/r DLBCL in Q2

Q2 sales of USD 24m

Fast ramp-up of centers in the US, with >50 centers

already actively prescribing

Broad payer coverage with 60% of lives covered

CMS pass-through achieved July 2018

Included in NCCN guidelines after 3 months

Reimbursement achieved in several EU countries

Positive CHMP

opinion

Received FDA

approval for two

indications

Filing underway

in 2018

CANADA

USA

SOUTH

AMERICA

AFRICA

EUROPEASIA

JAPAN

AUSTRALIA

Sandoz and Alcon Q2 performance continued in line with recent trends


Sandoz net sales down -2%

(cc), impacted by US price

erosion

Ex-US net sales grew +5% (cc)

Global Biopharmaceuticals

sales up +34% (cc)

Alcon continued strong growth

momentum with net sales up

+5% (cc)

Core operating income up

+14% (cc)

Core margin at 18.6%,

increasing 1.5% pts (cc)

Advancing our pipeline of potential blockbuster launches


1. Exact launches and timing depends on filing date, HAs decisions and timelines 2. Aimovig® is in collaboration with Amgen. Companies co-commercialize in the US (Amgen books sales to third parties), Novartis has Aimovig® exclusive rights

in rest of world excluding Japan 3. Source: EvaluatePharma World Preview 2018

Expected launches6

3Aimovig®2

Migraine

Kymriah®

DLBCL

Lutathera®

NET

OMB157

Relapsing MS

QVM149

Asthma

Entresto®

HFpEF

Cosentyx®

nrAxSpA

SEG101

Sickle cell disease

20181 20201

QAW039

Asthma

ACZ885

CV risk reduction

BAF312

SPMS

RTH258

nAMD

20191

AVXS-101

SMA

4

External recognition3

in value creation from

pipeline products

in value creation from

advanced therapies

#1

#1

SPMS submission completed Q2 2018 (awaiting file acceptance), Priority Review Voucher used; anticipated approval early 2019


BAF312 (siponimod): First and only drug shown1 to reduce disability progression in a true SPMS population

Reduced the risk of disability progression,

not driven by effect on relapses2

Cognitive processing speed significantly improved3

0 0.2 0.4 0.6 0.8 1 1.2 1.4

Principal stratum: non relapsing patients4

Favors siponimod Favors placebo

12 months

18 months

3m-CDP

24 months

12 months

18 months

24 months

Risk ratio (95% CI)

0.80 (0.56; 1.08)

0.86 (0.57; 1.24)

0.82 (0.48; 1.32)

0.67 (0.44; 0.93)

0.71 (0.42; 1.09)

0.71 (0.37; 1.21)

6m-CDP

SPMS submission expected Q3 2018; anticipated approval Q4 2019

-1.0

-0.5

0.0

0.5

1.0

1.5

2.0

2.5

P=0.0125

P=0.00045

Ch

an

ge

fro

m b

as

eli

ne

in

SD

MT

ora

l s

co

re

(n=999)

(n=509)

(n=919)

(n=446)

(n=312)

(n=139)

(n=642)

(n=306)

Month6 12 18 24

siponimod

placebo

See appendix slide 34 for references CDP – confirmed disability progression

AVXS-101: US regulatory submission anticipated in H2 2018 as planned

Novartis Q2 2018 Results | July 18, 2018 | Novartis investor presentation 15

Commercial product comparable to the product used in the Phase 1 trial

Phase 1 data in SMA Type 1 will form the primary basis for the BLA submission (H2 2018);

all available clinical data will be submitted (incl. some preliminary Phase 3 STR1VE data)

An integrated overview of the safety findings from the development program1 will be

provided

1. Includes CL-101, LT-001, CL-102. CL-303, CL-304 trials

Newborn screening for SMA is now officially recommended by US HHS for inclusion in the

Recommended Uniform Screen Panel (RUSP)

Pre-BLA

meeting

Black dashed line denotes SMA Type 1 natural history (Finkel et al., 2014)

Rapid onset; sustained efficacy

regardless of severity at treatment1

Most patients achieving CHOP-INTEND >50,

above natural history benchmarks

All patients alive and without need for

permanent ventilation 2 years post-GRT

Patients observed in long-term follow-up

continue to achieve new milestones

Only 4 of 12 patients able to swallow safely at

enrollment, reflecting disease severity; 11 of 12

swallowing safely for oral feeding at month 24

follow up

1. Presented at AAN 2018; text bullets reflect Cohort 2 (proposed therapeutic dose), n=12

0

10

20

30

40

50

60

0 10 20 30Natural

History1


New data1 confirm AVXS-101 could be foundational gene replacement therapy (GRT) for SMA Type 1

CH

OP

-IN

TE

ND

Score

s

Age1 (months)

16

AVXS-101 clinical development plan to cover all pediatric patients and SMA types


2014-2017 Q1 2018 Q2 2018 Q3 2018 Q4 2018 Q1 2019 Completion3

SMA

Type 1

START (CL-101) 1

(n=15; Open Label, Dose

Comparison, Single i.v. dose)

2033

2020

2020

SMA

Type 2

2019

Pre-symptomatic SMA

Type 1,2,3

2020-20234

Pediatric SMA

Type 1,2,3

TBC

STR1VE (CL-303 / NCT03306277) – Single i.v. dose

Initiated in Q4 2017, patient enrollment complete

STR1VE EU (CL-302 / NCT03461289) – Single i.v. dose

Initiated Q2

STRONG (CL-102 / NCT03381729) – Dose Comparison, Single i.t. dose

Started in Q1 2018; dose escalation enrolled; expansion starting in Q3

SPR1NT (CL-304 / NCT03505099) Single i.v. dose

Initiated Q2

REACHSingle i.t. dose

START Long-term follow-up (LT-001 / NCT03421977)

12 patients enrolled2

i.v. – Intravenous; i.t. – Intrathecal See appendix slide 34 for references

AVXS-101 ready for launch in 2019

18

WMS 2018: Anticipate updated pivotal data from STR1VE in SMA Type 1

AAN 2019: Anticipate data from SPR1NT, the Phase 3 pre-symptomatic study

STRONG: Phase 1 study in patients with SMA Type 2 expected at an upcoming medical

meeting, once the data are sufficiently robust as to be meaningful

US: On track for H2 2018 BLA submission (Breakthrough Therapy designation)

EU: Submission anticipated in H2 2019 (PRIME designation)

Japan: Pre-submission discussions planned Q3 2018 (Sakigake designation)

Commercial scale-up underway: Manufacturing facility in the Chicago area fully

operational, product inventory currently in production

170,000-square foot facility in Durham, NC: Expected to be fully operational in 2020


Clinical

readouts

Regulatory

status

Manufacturing

Agenda




4 Q&A Team


Summary of Q2 financial results


1. Core results, constant currencies and free cash flow are non-IFRS measures. Further details regarding non-IFRS measures can be found starting on page 54 of the Condensed Interim Financial Report 2. Not meaningful due to the CY

benefit from a USD 5.7 billion net gain recognized from the sale of our stake in the GSK consumer healthcare joint venture. 2017 Q2 Net Income USD 2.0bn and EPS USD 0.84

Group1

USD million

Q2

2018

Change vs. PY

% USD % cc

Net sales 13,158 7 5

Core operating income 3,541 9 7

Operating income 2,484 9 6

Net income 7,768 nm2 nm

Core EPS (USD) 1.29 6 4

EPS (USD) 3.34 nm2 nm

Free cash flow 3,562 10

Q2 core EPS +4% (cc), including the impact from discontinuation of OTC JV core income


1.29

1.18

Q2 2017

+4%

Q2 2018excluding

OTC JV1

1.22

Q2 2017excluding

OTC JV1

Core EPSUSD, % cc

1. Q2 2017 core EPS adjusted to exclude core income recognized from OTC JV

Key drivers vs. PY:

+ Higher core operating income

+ Lower shares from 2017 share

buyback program

− Discontinuation of OTC JV core

income from April 1, 2018

+

+

Q2 Group core margin increased to 26.9%, driven by Innovative Medicines


Q2 2018

Net saleschange vs. PY

Core OpIncchange vs. PY

Core margin%

Core marginchange vs. PY

% cc % cc % pts cc

Innovative Medicines 8 12 32.2 1.2

Sandoz -2 -5 19.5 -0.6

Alcon 5 14 18.6 1.5

Group 5 7 26.9 0.5

2018 Group full year guidance re-confirmed

23

Barring unforeseen events (in cc)

Full year guidanceGrowth vs. PY in cc

Group sales expected to grow low-to-mid single digit

IM Division to grow mid single digit

Sandoz revised downwards to decline low single digit

Alcon revised upwards to grow mid single digit

Group core operating income expected to grow mid-to-high single digit


Agenda




4 Q&A Team


Conclusion


Delivering a solid operational performance

Advancing our pipeline, including potential blockbuster launches

On track for full year guidance

Continuing our transformation to a focused medicines company

Agenda




4 Q&A Team


Appendix

2018 pipeline milestones


H1 2018 H2 2018

Regulatory

decisions and

opinions

Kymriah® DLBCL (US) ✓ Aimovig®2 Migraine (EU) =6

Tafinlar® + Mekinist® Adjuvant melanoma (US) ✓ Kymriah® Pediatric and young adult r/r ALL (EU) =6

Lutathera® NET (US) ✓ Kymriah® DLBCL (EU) =6

Gx Advair®1 Asthma, COPD (US) ✕ Tafinlar® + Mekinist® Adjuvant melanoma (EU) + ATC (US) (✓7)

Aimovig®2 Migraine (US) ✓ Gilenya® Pediatric MS (US) ✓

Glatopa® 40mg Relapsing MS (US) ✓ GP2017 Adalimumab BS (EU) =

LA-EP2006 Peg-filgrastim BS (EU) =

GP1111 Infliximab BS (EU) ✓

GP20138 Rituximab BS (US) ✕

Submissions ACZ885 CV risk reduction (US/EU) ✓ BAF312 MS (EU) =

BAF312 MS (US) ✓ RTH258 nAMD (US/EU) =

Kisqali® Advanced BC (US/EU)3 ✓ BYL719 HR+ BC (US/EU) =

Cosentyx® AS (JP) ✓ AVXS-101 SMA9 (US) =

CTL019 Pediatric ALL + DLBCL (JP) ✓

Promacta® 1st line SAA (US/EU) ✓

Major trial

readouts

Kisqali® Advanced BC (MONALEESA-3) ✓4 LIK06610 Obesity ✓

LJN452 NASH ✓5 BYL719 HR+ BC =

INC280 ALK- cMET amplified NSCLC =

Entresto® HFpEF (interim analysis) =

✓ Achieved ✕ Missed = On track

1. Complete Response Letter received from FDA; Advair® is a registered trademark of Glaxo Group Ltd. 2. Aimovig® is in collaboration with Amgen. 3. Indication expansion based on MONALEESA-3 & 7 results 4. Data presented at

ASCO 5. IA readout achieved as planned in H1, data to be presented in H2 6. Positive CHMP opinion receive 7. US approval in ATC received; EU approval in adj. melanoma still outstanding

8. Complete Response Letter received from FDA 9. i.v. formulation in type 1 SMA 10. Program discontinued

AveXis offers an attractive new gene therapy platform with potential beyond SMA


Selected assets Indication Status

AVXS-101 (AAV9) SMA Pivotal studies

CGF166 (Ad5) Hearing loss Phase 1b

CPK850 (AAV8) Retinitis pigmentosa Phase 1b

AVXS-201 RTT Rett Syndrome Preclinical

AVXS-301 SOD1 Inherited ALS-SOD1 Preclinical

Homology Medicines collaboration Ophthalmology & hematology Preclinical

Gene therapy A Undisclosed Preclinical

Gene therapy B Undisclosed Preclinical

AveXis projects

H1 2018 Group core margin increased to 26.6% driven by IM Division and Alcon


H1 2018

Net saleschange vs. PY

Core OpIncchange vs. PY

Core margin%

Core marginchange vs. PY

% cc % cc % pts cc

Innovative Medicines 7 8 31.8 0.5

Sandoz -3 -2 19.7 0.2

Alcon 6 21 19.4 2.3

Group 5 6 26.6 0.3

Key drivers vs. PY:

+ Cash flow from operating activities

− Higher intangible investments

H1 2018 free cash flow at USD 5.5bn

31

+12%

H1 2017

5.54.9

H1 2018

Group free cash flow USD bn


+

H1 2018 net debt broadly in line with 2017 Y/E

32

USD bn

-0.3-8.3

-3.5-7.0

-19.2-19.0

0.4

-0.2

Jun 30,

2018

Dec 31,

2017

Dividends

5.5

13.0

Treasury share

transactions,

net

AveXis, Inc.

Acquisition2

Proceeds from

sale of OTC JV

AAA1

Acquisition2

Free Cash

Flow

Others


1. Advanced Accelerator Applications S.A. 2. Net of cash acquired

Currency impact vs. PYIn % points, assuming mid-July exchange rates prevail in 2018

2017

Expected currency impact for H2 and FY 2018

33

FX impact on net sales FX impact on core operating income

Simulation

1

-3-2

27

-1

01

-2-2

26

-1

Q2FY FYQ4Q4Q3 Q3Q1FYQ1FY Q2

2017 2018 2018


Actual

References


Slide 4: Focused medicines company

1. The planned spinoff is subject to general market conditions, tax rulings and opinions, final Board endorsement and shareholder approval at the AGM in February

2019; completion expected in H1 2019 2. Alcon 2014 net sales include the Ophthalmic OTC and Diagnostics products transferred from the Innovative Medicines

Division from January 1, 2018, and exclude the remaining Ophthalmic Pharmaceuticals business transferred to the Innovative Medicines Division from January 1,

2016 3. Excluding sales from the Blood Transfusions Diagnostics Unit divested to Grifols S.A. on January 9, 2014

Slide 7: Cosentyx®

1. Projected based on IQVIA week ending June 29, 2018. Growth rate projected as reported; 67% if excluding free drug access program (FDAP) volume.

Slide 8: Entresto®

1. Based on IQVIA week ending June 29, 2018. 2. Khariton, Y, Fonarow, GC, et al. Association Between Sacubitril/Valsartan Initiation and Health Status

Outcomes in Heart Failure with Reduced Ejection Fraction: Findings from the CHAMP-HF Registry. Data presented at the European Society of Cardiology Heart

Failure (ESC-HF); 2018 May 26-29; Vienna, Austria.

Slide 9: Aimovig®

1. Source: IQVIA Data as of 7/11/18; Restated IQVIA NBRx and TRx volume during 5/25 – 6/29 corrects for overstatement in the mail channel by ~30% lower than

reported in NPA

Slide 14: BAF312

1. Kappos et al, The Lancet 2018; 391 (10127): 1263-73. 2. Cree BAC, et al. Uncoupling the impact on relapses and disability progression: siponimod in relapsing

and non-relapsing patients with secondary progressive multiple sclerosis in the phase III EXPAND study. AAN, Los Angeles 2018 3. Benedict RHB, et al. Impact of

siponimod on cognition in patients with secondary progressive multiple sclerosis: results from phase 3 EXPAND study. AAN, Los Angeles, 2018. 4. Principal

stratum method: estimate effect in patients who would not have relapsed on-study by Month 12, Month 18 and Month 24, regardless of treatment received

5. Analyses were predefined for months 12 and 24

Slide 17: AVXS-101

1. AVXS-101-CL-101 study published in N Engl J Med 2017; 377:1713-1722 2. As of June 22, 2018 3. Expected primary completion as per clinicaltrials.gov 4.

2020 for 2 copy cohort and up to 2023 for 3 and 4 copy cohort

Clinical Trials Update Includes selected ongoing or recently concluded global trials of Novartis development programs/products which are

in confirmatory development or marketed (typically Phase 2 or later).

For further information on all Novartis clinical trials, please visit: www.novartisclinicaltrials.com

1. Secondary prevention of cardiovascular events

2. Diffuse large B-cell lymphoma

3. Severe aplastic anemia

4. Chronic myeloid leukemia

5. Long-acting release

6. Non-small cell lung cancer

7. Neovascular age-related macular degeneration

8. Multi-drug resistant

9. Breast cancer

10. Retinopathy of prematurity

11. Indolent non-Hodgkin’s lymphoma

12. Non-radiographic axial spondyloarthritis

13. Preserved ejection fraction

14. Graft-versus-host disease

15. Neuroendocrine tumors

16. Chronic spontaneous urticaria / chronic idiopathic urticaria

17. Psoriatic arthritis head-to-head study versus adalimumab

18. Ankylosing spondylitis head-to-head study versus adalimumab

19. Acute myeloid leukemia

20. Chronic obstructive pulmonary disease

21. Chronic lymphocytic leukemia

22. IV formulation type 1 SMA

23. 1st line colorectal cancer / 1st line renal cell carcinoma

Planned filings 2018 to 2022

KAE609Malaria

ABL001CML4 3rd line

EMA401Peripheral neuropathic pain

CNP520Alzheimer’s disease

BYM338Hip fracture recovery

QGE031CSU/CIU16

BYM338Sarcopenia

VAY736Primary Sjoegren’s syndrome

Entresto®

Post-acute myocardial infarction

RTH258Diabetic macular edema

LCI699Cushing’s disease

BAF312 (EU)SPMS21

QAW039Asthma

Entresto®

Heart failure (PEF)13

Lucentis®

ROP10

INC280 NSCLC6

KAF156 Malaria

Kisqali® + fulvHR+, HER2 (-) postmenopausal

adv. or metastatic BC9 1st/2nd line

Kisqali® + tmx + gsn/or NSAI + gsnHR+, HER2 (-) premenopausal adv. or metastatic BC9 1st line

Cosentyx®

nrAxSpA12

BYL719 + fulvHR+, HER2 (-) postmenopausal

adv. BC9 2nd line

OMB157Relapsing multiple sclerosis

RTH258nAMD7

2022202020192018 2021

Jakavi®Acute GVHD14

LJN452Non-alcoholic steatohepatitis

Combination abbreviations:

fulv fulvestrant

tmx tamoxifen

gsn goserelin

NSAI Non-steroidal aromatase inhibitor

Taf Tafinlar® (dabrafenib)

Mek Mekinist® (trametinib)

QBW251COPD20


Kisqali ®HR+, HER2 (-) BC9 (adjuvant)

Cosentyx®

PsA H2H17

Cosentyx®

AS H2H18

LAM320MDR8 tuberculosis

ZPL389Atopic dermatitis

Rydapt®AML19 (FLT3 wild type)

UNR844Presbyopia

SEG101Sickle cell disease

LA-EP2006 (pegfilgrastim, US)Chemotherapy-induced neutropenia

and others (same as originator)

INC280NSCLC6 (EGFRm)

Jakavi®Chronic GVHD14

PDR001 + Tafinlar®+Mekinist®

Metastatic BRAF V600+ melanoma

ECF843Dry eye

LMI070Spinal muscular atrophy

VAY785Non-alcoholic steatohepatitis

CAD106Alzheimer’s disease

XolairNasal Polyps

VAY736Autoimmune Hepatitis

CTL019 (Kymriah ® US)+ pembrolizumab - r/r DLBCL

ACZ885Adjuvant NSCLC

ACZ8851st Line NSCLC

ACZ8852nd Line NSCLC

LHW090Resistant hypertension

ABL001CML4 1st line

CTL019 (Kymriah® US)r/r Follicular Lymphoma

CTL019 (Kymriah ® US)CLL21

CTL019 (Kymriah ® US)r/r DLBCL in 1st relapse

QVM149Asthma

QMF149Asthma

PDR001Metastatic Melanoma

MTV273Multiple myeloma

HDM201Acute myeloid leukemia

LOU064Chronic spontaneous urticaria

CFZ533Solid Organ Transplant

New molecule

New indication

New formulation

Biosimilars

AVXS-101Spinal Muscular Atrophy22

VPM087CRC 1L/RCC 1L23

CSJ117Severe Asthma

Lucentis®

Diabetic retinopathy

RTH258Retinal vein occlusion

36

http://deis.novartis.intra/deis.asp?VAM627A

http://deis.novartis.intra/deis.asp?SEB001X

http://deis.novartis.intra/deis.asp?VAK694A




12. Diffuse large B-cell lymphoma

13. Preserved ejection fraction

14. Graft-versus-host disease

15. Multi-drug resistant

16. Retinopathy of prematurity

17. Severe aplastic anemia

18. Acute myeloid leukemia

19. Acute lymphoblastic leukemia

20. Secondary progressive multiple sclerosis

21. Long-acting release

22. Chronic lymphocytic leukemia

23. IV formulation type 1 SMA

24. 1st line colorectal cancer / 1st line renal cell

carcinoma

Pipeline of key projects in confirmatory development

Early Clinical Trials Registration Trials – Phase III / Pivotal In Registration

1. Chronic myeloid leukemia

2. Non-small cell lung cancer

3. Chronic spontaneous urticaria / chronic idiopathic urticaria

4. Neuroendocrine tumors

5. Breast cancer

6. Neovascular age-related macular degeneration

7. Secondary prevention of cardiovascular events

8. Indolent non-Hodgkin’s lymphoma

9. Non-radiographic axial spondyloarthritis

10. Psoriatic arthritis head-to-head study versus adalimumab

11. Ankylosing spondylitis head-to-head study versus adalimumab


FTY720 Pediatric multiple sclerosis

Cosentyx®

nrAxSpA9

Entresto®

Heart failure (PEF)13

Kisqali® + tmx + gsn/or NSAI + gsnHR+, HER2(-) premenopausal adv. or metastatic BC5 1st line

Kisqali® + fulvHR+, HER2(-) postmenopausal

adv. or metastatic BC5 1st/2nd line

Entresto®

Post-acute myocardial infarction

CTL019 (Kymriah® b US)r/r DLBCL12

Jakavi®Acute GVHD14

Cosentyx®

PsA H2H10

Cosentyx®

AS H2H11

LAM320MDR15 tuberculosis

Kisqali®

HR+, HER2(-) BC5 (adjuvant)

Jakavi®Chronic GVHD14

Combination abbreviations:fulv fulvestrant

ltz letrozole

tmx tamoxifen

gsn goserelin

NSAI Non-steroidal aromatase inhibitor

Taf Tafinlar® (dabrafenib)

Mek Mekinist® (trametinib)

Lucentis®

ROP16

QMF149Asthma

QVM149Asthma

OMB157 Relapsing multiple sclerosis

RTH258Diabetic macular edema

Tafinlar® + Mekinist® b

BRAF V600+ melanoma (adjuvant)

Promacta®/Revolade®

SAA17 1st line

Rydapt®AML18 (FLT3 wild type)

LA-EP2006 (pegfilgrastim, US)Chemotherapy-induced neutropenia and

others (same as originator)

XolairNasal Polyps

SEG101Sickle cell disease

BAF312 (EU) SPMS20

QAW039Asthma

RTH258nAMD6

LCI699Cushing’s disease

BYL719 + fulvHR+, HER2 (-) postmenopausal

adv. BC5 2nd line

CTL019 (Kymriah ® b US)Pediatric/young adult ALL19

ACZ885Adjuvant NSCLC2

ACZ8851st Line NSCLC2

ACZ8852nd Line NSCLC2

CAD106Alzheimer’s disease

KAE609Malaria

BYM338 Hip fracture recovery

EMA401Peripheral neuropathic pain

INC280 NSCLC2

ABL001CML1 3rd line

CNP520Alzheimer’s disease

ECF843Dry eye

ABL001CML1 1st line

KAF156Malaria

LJN452Non-alcoholic steatohepatitis

QGE031CSU/CIU3

QBW251COPD

LMI070Spinal muscular atrophy

LHW090Resistant hypertension

VAY736Primary Sjoegren’s syndrome

BYM338 Sarcopenia

ZPL389Atopic dermatitis

UNR844Presbyopia

INC280NSCLC2 (EGFRm)

VAY785Non-alcoholic steatohepatitis

VAY736Autoimmune Hepatitis

CTL019 (Kymriah ® US)r/r Follicular Lymphoma

CTL019 (Kymriah ® US)CLL22

Aimovig™ aMigraine prophylaxis

GP2013 (rituximab, US)Follicular lymphoma, DLBCL12 and


GP2017 (adalimumab) Arthritides, plaque psoriasis and others

(same as originator)

PDR001Metastatic Melanoma

CTL019 (Kymriah ® US)r/r DLBCL in 1st relapse

PDR001 + Tafinlar®+Mekinist®

Metastatic BRAF V600+ melanoma

LA-EP2006 (pegfilgrastim, EU)Chemotherapy-induced neutropenia and


CTL019 (Kymriah ® US)+ pembrolizumab - r/r DLBCL

HDM201Acute myeloid leukemia

LOU064Chronic spontaneous urticaria

MTV273Multiple myeloma

CFZ533Solid Organ Transplant

ACZ885Sec. prev. CV events1

LTW888 bRetinopathy

New molecule

New indication

New formulation

Biosimilars

AVXS-101Spinal Muscular Atrophy23

VPM087CRC 1L/RCC 1L24

CSJ117Severe Asthma

RTH258Retinal vein occlusion

Lucentis®

Diabetic retinopathy

BAF312 (US) SPMS20

a) In collaboration with Amgen; companies to co-commercialize in the US,

Novartis to have AMG 334 exclusive rights in rest of world excluding Japan

b) Approved in US, submitted in EU

37

http://deis.novartis.intra/deis.asp?FTY720D

http://deis.novartis.intra/deis.asp?DNK333B

http://deis.novartis.intra/deis.asp?LBS834A


http://deis.novartis.intra/deis.asp?RFB002D





http://deis.novartis.intra/deis.asp?AHT956A






http://deis.novartis.intra/deis.asp?CAD106A







http://deis.novartis.intra/deis.asp?ATI355A












http://deis.novartis.intra/deis.asp?AHT956A

Key changes vs. Q1 2018 presentation


New additions

Trials taken out (operational decision-points achieved)

Study Program Indication Phase Patients

NCT03320941 (CLIK066B1201) LIK066 Obesity Phase 2 130



NCT01077518 (COMB157E2301) Arzerra® Refractory iNHL (3rd Line) Phase 3A 346

Study Program Indication Phase Patients

NCT03249714 SAKURA (COMB157G1301) OMB157 Multiple sclerosis Phase 2 60

NCT03280030 (CPKC412A2220) Rydapt® Acute myeloid leukemia Phase 2 66

Cardio-Metabolic

Entresto® - Angiotensin receptor neprilysin inhibitor (ARNI)

40

Study NCT02678312 PANORAMA HF (CLCZ696B2319) NCT02661217 TRANSITION (CLCZ696B2401)

Indication Heart failure in pediatric patients Heart failure

Phase Phase 2/3 Phase 4

Patients 360 1,000

Primary Outcome

Measures

Part 1: Pharmacodynamics and pharmacokinetics of

LCZ696 analytes

Part 2: Efficacy and safety compared with enalapril

Assessing the percentage of patients who achieve the target

dose of 200 mg bid LCZ696 at 10 weeks after

randomization

Arms/Intervention

• Part 1: LCZ696 0.8 mg/kg or 3.1 mg/kg or both

• Part 2: Enalapril is 0.2 mg/kg bid; LCZ696: 3.125 mg

granules and adult formulation (50, 100, 200 mg bid)

• Pre-discharge treatment initiation - LCZ696 (50, 100,

200 mg bid)

• Post-discharge treatment initiation - LCZ696 (50, 100,

200 mg bid)

Target Patients

Pediatric patients from1 month to < 18 years of age with

heart failure due to systemic left ventricle systolic

dysfunction

Heart failure patients with reduced ejection-fraction

hospitalized for an acute decompensation event

Expected Completion 2021 Q3-2018

Publication TBD TBD


41

Study NCT02884206 PERSPECTIVE (CLCZ696B2320) NCT02468232 PARALLEL-HF (CLCZ696B1301)

Indication Heart failure Heart failure, reduced ejection fraction

Phase Phase 3 Phase 3

Patients 520 220

Primary Outcome

Measures

Change from baseline in the CogState Global Cognitive

Composite Score (GCCS)

Time to the first occurrence of the composite endpoint -

either cardiovascular (CV) death or heart failure (HF)

hospitalization

Arms/Intervention

• LCZ696 50, 100, and 200 mg bid with placebo of

valsartan

• Valsartan 40, 80, and 160 mg bid tablets with placebo

for LCZ696

• LCZ696 50 mg, 100 mg, 200 mg bid/placebo of Enalapril

• Enalapril 2.5 mg, 5 mg, 10 mg bid / placebo of LCZ696

Target PatientsPatients with chronic heart failure with preserved ejection

fraction

Japanese heart failure patients (NYHA Class II-IV) with

reduced ejection fraction

Expected Completion 2022 H1-2019

Publication TBD TBD



42

Study NCT02554890 PIONEER-HF (CLCZ696BUS01) NCT02924727 PARADISE-MI (CLCZ696G2301)

Indication Heart failure Acute myocardial infarction


Patients 882 4,650

Primary Outcome

Measures

Percentage change from baseline in N-terminal pro-brain

natriuretic peptide (NT-proBNP)

Time to the first occurrence of a confirmed composite

endpoint (cardiovascular (CV) death, heart failure (HF)

hospitalization, or outpatient heart failure)

Arms/Intervention

• sacubitril/valsartan (LCZ696)

• sacubitril/valsartan (LCZ696) matching placebo

• enalapril

• enalapril matching placebo

• LCZ696 50 mg, 100 mg, 200 mg bid / placebo of

ramipril/valsartan

• Ramipril 1.25 mg, 2.5 mg, and 5 mg bid / placebo of

LCZ696 / placebo for valsartan

Target PatientsPatients with HFrEF (LVEF<40%) hospitalized for ADHF

and stable for more than 24 hours

Post-AMI patients with evidence of LV systolic dysfunction

and/or pulmonary congestion, with no known prior history of

chronic HF

Expected Completion Q3-2018 2020

Publication TBD TBD



43

Study NCT01920711 PARAGON (CLCZ696D2301) NCT03066804 PARALLAX (CLCZ696D2302)

Indication Heart failure, preserved ejection fraction Heart failure, preserved ejection fraction


Patients 4,800 2,200

Primary Outcome

Measures

Cumulative number of primary composite events of

cardiovascular (CV) death and total (first and recurrent) HF

hospitalizations

Change in NT-proBNP from baseline to week 12

Arms/Intervention• LCZ696 50 mg, 100 mg and 200 mg

• Valsartan 40 mg, 80 mg and 160 mg

• LCZ696 50 mg, 100 mg and 200 mg bid

• Enalapril 2.5 mg, 5 mg and 10 mg bid

• Valsartan 40 mg, 80 mg, 160 mg bid

• Placebo to match LCZ696 sacubitril/valsartan

• Placebo to match enalapril

• Placebo to match valsartan

Target PatientsHeart failure patients (NYHA Class II-IV) with preserved

ejection fraction

Heart failure patients (NYHA Class II-IV) with preserved

ejection fraction

Expected Completion H2-2019 2020

Publication TBD TBD



Ilaris® - Anti IL-1β

44

Study NCT01327846 CANTOS (CACZ885M2301)

Indication Cardiovascular risk reduction

Phase Phase 3

Patients 10,061

Primary Outcome

Measures

Time to first occurrence of major adverse cardiovascular event, which is a composite of CV death, non-fatal MI, and

stroke

Arms/Intervention

• Canakinumab 50 mg + standard care therapy



• Placebo + standard care therapy

Target Patients Post-myocardial infarction patients on standard of care with elevated hsCRP

Expected Completion Core portion of study completed June 2017; Open-label extension continues till H1-2020

Publication

• Published cancer results in Lancet, August 2017

• Published CV outcomes in NEJM, Sept 21, 2017 vol 377 no 12

• Published pre-planned secondary analysis on relationship of CRP and CVRR; presented at AHA as late breaker

Nov 2017

• Presented at ACC 2018 and published T2DM secondary endpoint

• Chronic kidney disease data was presented at ACC 11 Mar 2018; results to be published in the up-coming months.

• Op Ed published in Fortune. V Narashimhan, Novartis CEO, Mar 2018

• Chronic kidney disease data was presented at ACC 11 Mar 2018; published in J Am Coll Cardiol, May 29, 2018 vol

71 no 21Novartis Q2 2018 Results | July 18, 2018 | Novartis Investor Presentation

Immunology, Hepatology & Dermatology

Cosentyx® - Anti IL-17

46

Study NCT02404350 FUTURE 5 (CAIN457F2342)NCT01640951 SCULPTURE (CAIN457A2304E1 –

extension study)

Indication Psoriatic arthritis Psoriasis


Patients 990 675

Primary Outcome

Measures

American College of Rheumatology 20 (ACR20) response at

Week 16

The number and percentage of subjects having any adverse

event

Arms/Intervention

• Secukinumab 150 mg load

• Secukinumab 150 mg no load


• Placebo

• Fixed-time interval regimen secukinumab 150 mg

• Retreatment at start of relapse secukinumab 150 mg

• Fixed-time interval regimen secukinumab 300 mg

• Retreatment at start of relapse secukinumab 300 mg

• Open label secukinumab 300 mg

Target Patients Patients with active psoriatic arthritisPatients with moderate to severe chronic plaque-type

psoriasis

Expected Completion Q2-2019 2017 (actual)

Publication

• 24 week results late breaker presented in ACR in Nov-

2017

• 24 week data; manuscript submitted to Annals of

Rheumatic Disease in Nov 2017

• 52 week data; to be presented at ACR 2018

• 3-years results: Br J Dermatol; 5 June 2017. doi:

10.1111/bjd.15706

• 5-years results: Submitted to JEADV; 14 February 2018

doi: 10.1111/jdv.14878

• 5-years presented at EAD Sept 2017 (late-breaker)



47

Study NCT01863732 (CAIN457F2305E1 – extension study) NCT03259074 SURPASS (CAIN457K2340)

Indication Ankylosing spondylitis Ankylosing spondylitis


Patients 300 837

Primary Outcome

Measures

Assessment of spondyloarthritis international society criteria

/ ASAS 20 response

No radiographic structural progression as measured by

modified Stoke Ankylosing Spondylitis Spine Score

(mSASSS)

Arms/Intervention• Secukinumab 75 mg in PFS

• Secukinumab 150 mg in PFS

• Secukinumab 150/300 mg

• adalimumab biosimilar 40 mg

Target Patients Patients with active ankylosing spondylitis Patients with active ankylosing spondylitis

Expected Completion Q2-2018 (actual) 2022

Publication

• 3-year results: Manuscript published in Clinical and

Experimental Rheumatology in May-2017

• 4-year results: Presented at ACR in Nov-2017

TBD



48

Study NCT01649375 MEASURE 2 (CAIN457F2310) NCT02008916 MEASURE 3 (CAIN457F2314)

Indication Ankylosing spondylitis Ankylosing spondylitis


Patients 219 222

Primary Outcome

Measures

Assessment of SpondyloArthritis International Society /

ASAS 20 response

Assessment of Spondyloarthritis International Society

criteria / ASAS 20 response

Arms/Intervention

• Secukinumab 75 mg


• Placebo

• Secukinumab 10 mg/kg / 300 mg

• Secukinumab 10 mg/kg / 150 mg

• Placebo

Target Patients Patients with active ankylosing spondylitis Patients with active ankylosing spondylitis

Expected Completion Q4-2018 Q1-2018 (actual)

Publication

• Primary 52 week results: Baeten D & Sieper J, et al. N

Engl J Med 2015;373:2534–48

• 2 year results: Marzo-Ortega, et al. Arthritis Care Res

2017 Feb 24. doi: - 10.1002/acr.23233

• 3 year results: Marzo-Ortega, et al. RMD 2017

• 16 weeks results: PANLAR congress in Apr-2016

• 52 weeks results: Pavelka et al. Arthritis Research &

Therapy 2017

• 2 year results: Presented at ACR in Nov-2017



49

Study NCT02159053 MEASURE 4 (CAIN457F2320) NCT01989468 FUTURE 3 (CAIN457F2318)

Indication Ankylosing spondylitis Psoriatic arthritis


Patients 350 416

Primary Outcome

Measures

Assessment of Spondyloarthritis International Society

criteria / ASAS 20 at week 16

American College of Rheumatology 20 (ACR20) response in

subjects treated with secukinumab vs. placebo

Arms/Intervention

• Secukinumab 150 mg s.c. with loading

• Secukinumab 150 mg s.c. without loading

• Placebo

• Secukinumab (AIN457) 150 mg s.c.


• Placebo

Target Patients Patients with active ankylosing spondylitis Patients with active psoriatic arthritis

Expected Completion Q2-2018 (actual) Q2-2018 (actual)

Publication 52 week results: manuscript to be submitted in Q1-201852 week results: Nash et al, Arthritis Research & Therapy

2018, 20:47



50

Study NCT01752634 FUTURE 2 (CAIN457F2312) NCT01892436 FUTURE 1 extension (CAIN457F2306E1)

Indication Psoriatic arthritis Psoriatic arthritis


Patients 399 460

Primary Outcome

Measures

Proportion of subjects achieving American College of

Rheumatology 20 (ACR20) response criteria

Proportion of subjects that have a positive clinical response

to treatment (individual improvement) in disease activity

according to ACR20 (or ACR50 or ACR 70)

Arms/Intervention




• Placebo s.c.



Target Patients Patients with active psoriatic arthritis Patients with active psoriatic arthritis

Expected Completion Q2-2019 Q1-2018 (actual)

Publication

• Primary results: McInnes IB, et al. Lancet.

2015;386:1137–46

• 2 years results: McInnes et al, Rheumatology

2017;56:1993-2003

• 3 year results: Abstract to be submitted to EULAR

congress in Jun-2018

• 3 year results: ACR 2016; Mease PJ et al. Arthritis

Rheumatol. 2016; 68 (suppl 10)

• 3 years results: Manuscript submitted in Q4-2017



51

Study NCT03031782 (CAIN457F2304) NCT02745080 EXCEED (CAIN457F2366)

Indication Psoriatic Arthritis Psoriatic Arthritis


Patients 80 850

Primary Outcome

MeasuresTime to flare in Part 2 American College of Rheumatology 20 (ACR20) response

Arms/Intervention• Secukinumab (pre-filled syringe) 75 mg

• Placebo

• Secukinumab 300 mg s.c.

• Adalimumab 40 mg s.c.

Target PatientsJuvenile Idiopathic Arthritis subtypes of Psoriatic and

Enthesitis-related ArthritisPatients with active psoriatic arthritis

Expected Completion 2021 2020

Publication TBD TBD



52

Study NCT02294227 FUTURE 4 (CAIN457F2336) NCT02471144 (CAIN457A2310)

Indication Psoriatic arthritis Psoriasis


Patients 342 169

Primary Outcome

Measures

Assessment of American College of Rheumatology 20

(ACR20)

The percentage of Participants achieving a 75%

Improvement from Baseline in PASI Score at week 12

Arms/Intervention

• Secukinumab 150 mg with loading

• Secukinumab 150 mg without loading

• Placebo

• Secukinumab low dose

• Secukinumab high dose

• Placebo

• Etanercept (comparator)

Target Patients Patients with active psoriatic arthritisPatients from 6 to less than 18 years of age with severe

chronic plaque


Publication

• 52 week results: abstract to be presented at PANLAR

congress (Apr-2018)

• 2 year results: manuscript to be submitted in Q3-2018

TBD



53

Study NCT02748863 ALLURE (CAIN457A2323) NCT01544595 (CAIN457A2302E1 – extension study)

Indication Psoriasis Psoriasis


Patients 214 1,146

Primary Outcome

Measures

Psoriasis Area and Severity Index (PASI) 75 response and

Investigators' Global Assessment (IGA) 0 or 1 response

Cumulative rate of subjects with loss of psoriasis area and

Cumulative rate of subjects with loss of Psoriasis Area and

Severity Index (PASI) 75 response up to week 68 (time = 0

being defined as week 52)

Arms/Intervention

• Secukinumab 300mg (2 mL PFS device)

• Secukinumab 300mg (2 x 1 mL PFS device)

• Placebo



• Placebo

Target Patients Adult subjects with moderate to severe plaque psoriasis

Patients with moderate to severe chronic plaque-type

psoriasis completing preceding psoriasis phase III studies

with secukinumab

Expected Completion Q3-2018 2017 (actual)

Publication• Submission Journal TBC Q2-2019

• Abstract at AAD in 2019

• 2-years results: Br J Dermatol. 2017 May 12. doi:

10.1111/bjd.15656

• 5-years results: Submission to BJD Q3-2018



54

Study NCT02696031 PREVENT (CAIN457H2315) NCT02826603 CLARITY (CAIN457A2326)

Indication Non-radiographic Axial Spondyloarthritis Psoriasis

Phase Phase 3 Phase 3B

Patients 555 1,117

Primary Outcome

Measures

The proportion of participants who achieved an ASAS 40

response (Assessment of SpondyloArthritis International

Society criteria);


Investigators' Global Assessment (IGA) 0 or 1 response at

week 12

Arms/Intervention


• Secukinumab 150 mg no load

• Placebo


• Ustekinumab 45 mg/ 90 mg

Target Patients Patients with non-radiographic axial spondyloarthritis Patients with moderate to severe plaque psoriasis

Expected Completion 2019 Q3-2018

Publication TBD

• Abstract Winter Clin Derm (US) Jan-2018

• Abstract to EADV in 2018

• Submission Journal (16wk 1ry EP IA) Q3-2018

• Encore Abstract AAD 2019



55

Study NCT03066609 (CAIN457A2318)

Indication Psoriasis

Phase Phase 3

Patients 543

Primary Outcome

Measures


Investigators' Global Assessment (IGA) 0 or 1 response at

week 12

Arms/Intervention



• Placebo

Target PatientsPatients with moderate to severe chronic plaque-type

psoriasis with or without psoriatic arthritis comorbidity

Expected Completion Q1-2019

Publication TBD


Ilaris® - Anti IL-1β

56

Study NCT02059291 CLUSTER (CACZ885N2301) NCT02296424 (CACZ885G2306)

Indication Hereditary periodic fevers SJIA - Systemic Juvenile Idiopathic Arthritis

Phase Phase 3 Phase 3B/4

Patients 203 182

Primary Outcome

Measures

To demonstrate significant reduction of disease activity

with canakinumab vs. placebo

Proportion of patients in clinical remission on canakinumab

who are able to remain at an initial reduced canakinumab dose

or prolonged canakinumab dose interval

Arms/Intervention• Canakinumab

• Placebo

• Canakinumab dose reduction

• Canakinumab dose interval prolongation

Target PatientsPatients with, 3 separate disease cohorts TRAPS, HIDS,

and colchicine resistant FMF (Hereditary periodic fevers )

Patients with Systemic Juvenile Idiopathic Arthritis (SJIA)

(Pediatric)

Expected Completion 2017 (actual) 2017 (actual)

Publication

• Safety & efficacy (w16+40) in NEJM in May 2018

(May 17, 2018: N Engl J Med 2018; 378:1908-1919)

• Additional manuscripts in 2018

Manuscript to be submitted in Q4-2018


LJN452 - FXR Agonist

57

Study NCT02855164 (CLJN452A2202)

Indication Non-alcoholic steatohepatitis

Phase Phase 2

Patients 345

Primary Outcome

Measures

Adverse event profile of different doses; determine the dose

relationship of LJN452 on markers of hepatic inflammation

in NASH (ALT and AST); determine dose-response

relationship of LJN452 on liver fat content by changes in

quantitative MRI; determine effect of LJN452 on liver fibrosis

by biopsy

Arms/Intervention Multiple LJN452 doses and placebo

Target Patients Patients with non-alcoholic steatohepatitis (NASH)

Expected Completion H2-2019

Publication TBD


QGE031 - Anti-IgE

Study NCT02477332 (CQGE031C2201) NCT02649218 (CQGE031C2201E1)

Indication Chronic spontaneous urticaria Chronic spontanenous urticaria

Phase Phase 2B Phase 2B

Patients 382 227

Primary Outcome

Measures

Establish dose-response relationship of QGE031 with

respect to achievement of complete hives response at week

12

Long-term safety; number of participants with treatment-

emergent adverse events (AEs)

Arms/Intervention Multiple doses of QGE031/Placebo/Omalizumab QGE031 240 mg

Target Patients Patients with Chronic Spontaneous Urticaria (CSU) Patients with Chronic Spontaneous Urticaria (CSU)

Expected Completion 2017 (actual) H2-2019

PublicationPrimary results: Presentation planned for EAACI 2018;

manuscript expected in Q3-2018TBD


VAY736 – Fully human IgG1/κ anti-BAFF-R mAb

Study NCT02962895 (CVAY736A2201) NCT03217422 AMBER (CVAY736B2201)

Indication Primary Sjoegren's syndrome Autoimmune hepatitis

Phase Phase 2B Phase 2

Patients 180 80

Primary Outcome

Measures

Safety and efficacy of VAY736 in primary Sjoegren's

syndrome (pSS)Alanine aminotransferase (ALT) normalzation

Arms/Intervention• VAY736

• Placebo

• VAY736

• Placebo control with conversion to active VAY736

Target PatientsPatients With Moderate to Severe Primary Sjoegren's

Syndrome (pSS)

Autoimmune hepatitis patients with incomplete response or

intolerant to standard treatment of care


Publication TBD TBD


Neuroscience

AimovigTM – CGRP receptor antagonist

61

Study NCT03096834 LIBERTY (CAMG334A2301) NCT03333109 EMPOWER (CAMG334A2302)

Indication Migraine Migraine


Patients 220 880

Primary Outcome

Measures

Percentage of patients with a 50% response in the reduction

of Monthly Migraine Days (MMD)

Change from baseline in monthly migraine days at the last

month (Month 3) of the double-blind treatment period

Arms/Intervention• Subcutaneous injection of AMG334 (erenumab)

• Subcutaneous injection of placebo

• AMG334 (erenumab)

• Placebo

Target PatientsAdult episodic migraine patients who have failed prophylactic

migraine treatmentsAdult episodic migraine patients

Expected Completion 2017 (actual) 2020

Publication TBD TBD


CNP520 - BACE inhibitorCAD106 - active beta-amyloid immunotherapy

62

Study NCT02565511 GENERATION S1 (CAPI015A2201J) NCT03131453 GENERATION S2 (CCNP520A2202J)

Indication Alzheimer’s disease Alzheimer’s disease

Phase Phase 2B/3 Phase 2B/3


Primary Outcome

Measures

Time to diagnosis of MCI due to Alzheimer's disease or

dementia due to Alzheimer's disease

Change in the Alzheimer's Prevention Initiative Composite

Cognitive (APCC) Test Score

Time to diagnosis of MCI due to Alzheimer's disease or

dementia due to Alzheimer's disease

Change in the Alzheimer's Prevention Initiative Composite

Cognitive (APCC) Test Score

Arms/Intervention

• CAD106 450 µg + Alum 450 µg i.m.

• Placebo to CAD106 + Alum 450 µg i.m.

• CNP520 50 mg oral

• Placebo to CNP520 oral



• Placebo to CNP520 oral

Target PatientsCognitively unimpaired participants aged 60 to 75 years,

with two APOE4 allele (Homozygotes )

Cognitively unimpaired participants aged 60 to 75 years,

with at least one APOE4 allele (Homozygotes or

Heterozygotes) and, if Heterozygotes, with evidence of

elevated brain amyloid


Publication TBD TBD


BAF312 - S1P-R modulator

63

Study NCT01665144 -EXPAND (CBAF312A2304)

Indication Secondary progressive multiple sclerosis

Phase Phase 3

Patients 1,620

Primary Outcome MeasuresThe delay in time to confirmed disability progression as

measured by EDSS (Expanded Disability Status Scale)

Arms/Intervention

• BAF312 (5-day titration: 0.25mg to 1.25mg; Maintenance

dose: 2mg (day 6))

• Placebo

Target Patients Patients with secondary progressive multiple sclerosis

Expected Completion Core in 2016/Extension in 2023

Publication• Presentations at ECTRIMS and AAN 2017

• Lancet March 2018


BYM338 - Activin receptor II B

64

Study NCT02333331 InvestiGAIT (CBYM338E2202) NCT02152761 (CBYM338D2201)

Indication Sarcopenia Hip fracture recovery

Phase Phase 2B Phase 2B

Patients 280 245

Primary Outcome

Measures

Evaluate improvement in physical performance (Change

from baseline at week 24 in Short Physical Performance

Battery)

Change from baseline in total lean body mass measured by

DXA at week 24

Arms/Intervention

• Bimagrumab low dose

• Bimagrumab moderate dose

• Bimagrumab high dose

• Placebo

• Bimagrumab low dose

• Bimagrumab moderate dose

• Bimagrumab high dose

• Placebo

Target Patients Older adults with sarcopenia Patients after surgical treatment of hip fracture

Expected Completion H1-2019 Q4-2018

Publication TBD TBD


EMA401 - Angiotensin II type 2 receptor antagonist

65

Study NCT03094195 EMPHENE (CEMA401A2201) NCT03297294 EMPADINE (CEMA401A2202)

Indication Peripheral neuropathic pain Peripheral neuropathic pain


Patients 360 400

Primary Outcome

Measures

Dose-response in change in weekly mean of the 24-hour

average pain score from Baseline to week 12

Change in weekly mean 24-hour average pain score

from Baseline to Week 12

Arms/Intervention• 3 doses EMA401

• Placebo

• 1 doses EMA401

• Placebo

Target Patients Post-herpetic neuralgia patients Painful diabetic neuropathy

Expected Completion H2-2019 H1-2019

Publication TBD TBD


Gilenya® - S1P-R modulator

66

Study NCT01892722 PARADIGMS (CFTY720D2311) NCT01201356 (CFTY720D2399)

Indication Pediatric multiple sclerosis Relapsing multiple sclerosis (RMS)

Phase Phase 3B Phase 3B/4

Patients 215 4,125

Primary Outcome

Measures

Frequency of relapses in patients treated for up to 24

months (using ARR)Long-term safety and tolerability

Arms/Intervention• Interferon beta-1a i.m.

• Fingolimod 0.5 mg/ 0.25 mgSingle-arm study of fingolimod 0.5 mg/day

Target PatientsPediatric patients with multiple sclerosis with five-year

fingolimod extension phasePatients with relapsing multiple sclerosis

Expected Completion Q3-2017 (core phase) / 2023 (extension phase) Q4-2018

Publication

• Primary data presentation: Chitnis T, et al. Presented at

ECTRIMS 2017 (Late Breaker)

• Primary manuscript: TBD

• Primary data presentation: Cohen J, et al presented at

ECTRIMS 2017

• Primary manuscript: TBD


Gilenya® - S1P-R modulator

67

Study NCT01633112 ASSESS (CFTY720D2312)

Indication Relapsing remitting multiple sclerosis (RRMS)

Phase Phase 3B

Patients 1,064

Primary Outcome

Measures

Comparison of 2 doses (0.25 mg and 0.5 mg) of fingolimod

to glatiramer acetate (20 mg) in reducing the annualized

relapse rate up to 12 months

Arms/Intervention

• Fingolimod 0.5 mg orally

• Fingolimod 0.25mg orally

• Copaxone® 20 mg s.c.

Target Patients Patients with relapsing-remitting multiple sclerosis


Publication TBD


LMI070 - SMN2 RNA splice modulator

68

Study NCT02268552 (CLMI070X2201)

Indication Type 1 spinal muscular atrophy

Phase Phase 1/2

Patients 44

Primary Outcome

Measures

Number of participants with adverse events (AEs), serious

adverse events (SAEs) and deaths

Arms/Intervention • Branaplam oral, once weekly, 3 ascending doses

Target PatientsPatients with type 1 spinal muscular atrophy


Publication TBD


OMB157 - Anti-CD20

69

Study NCT02792218 Asclepios I (COMB157G2301) NCT02792231 Asclepios II (COMB157G2302)

Indication Multiple sclerosis Multiple sclerosis


Patients 900 900

Primary Outcome

Measures

Annualized Relapse Rate (ARR) - number of confirmed

relapses in a year calculated based on cumulative number

of relapses by patient adjusted for time-in-study by patient

Annualized Relapse Rate (ARR) - number of confirmed

relapses in a year calculated based on cumulative number

of relapses by patient adjusted for time-in-study by patient

Arms/Intervention• Ofatumumab subcutaneous

• Teriflunomide oral

• Ofatumumab subcutaneous

• Teriflunomide oral

Target Patients Patients with relapsing forms of multiple sclerosis Patients with relapsing forms of multiple sclerosis


Publication TBD TBD


OMB157 - Anti-CD20

70

Study NCT03249714 SAKURA (COMB157G1301)

Indication Multiple sclerosis

Phase Phase 2

Patients 60

Primary Outcome

Measures

Reduced cumulative number of Gd-enhanced T1 lesions

across 4 MRI scans at week 12, 16, 20 and 24 (ofatumumab

vs placebo)

Arms/Intervention• Ofatumumab 20 mg subcutaneous injections

• Placebo

Target Patients Patients with relapsing forms of multiple sclerosis

Expected Completion 2020

Publication TBD


Oncology

ABL001 – Specific, allosteric Bcr-Abl kinase inhibitor

72

Study NCT03106779 (CABL001A2301)

Indication Chronic myeloid leukaemia (CML)

Phase Phase 3

Patients 222

Primary Outcome

MeasuresMajor Molecular Response (MMR) rate at 24 weeks

Arms/Intervention• ABL001 40 mg

• Bosutinib 500 mg

Target Patients

Patients with chronic myelogenous leukemia in chronic

phase (CML-CP), previously treated with 2 or more tyrosine

kinase inhibitors


Publication TBD


ACZ885 – IL1β inhibitor


Study NCT03447769 (CACZ885T2301)

Indication Adjuvant NSCLC

Phase Phase 3

Patients 1,500

Primary Outcome

Measures

Disease free survival (primary), overall survival (key

secondary)

Arms/Intervention• Canakinumab 200mg q3w sc for 18 cycles

• Placebo q3w sc for 18 cycles

Target Patients

Patients with:

• High–risk NSCLC (AJCC/UICC v.8 stage II-IIIA and IIIB

(T>5cm N2)) after complete resection

• All histologies

• With/without EGFR mutation


Publication • TBD

BYL719 - Alpha-specific PI3K inhibitor

74

Study NCT02437318 SOLAR-1 (CBYL719C2301)

Indication HR + mBC

Phase Phase 3

Patients 560

Primary Outcome

Measures

Progression-free survival (PFS) for patients with PIK3CA

mutant status

Arms/Intervention• Fulvestrant 500 mg + alpelisib 300 mg

• Fulvestrant 500 mg + placebo

Target Patients

Men and postmenopausal women with hormone receptor

positive, HER2-negative advanced breast cancer which

progressed on or after aromatase inhibitor treatment


Publication TBD


Kymriah® – CAR-T therapy

75

Study NCT02445248 JULIET (CCTL019C2201) NCT02435849 ELIANA (CCTL019B2202)

Indication Relapsed / refractory DLBCL Pediatric and young adult Relapsed/ refractory ALL


Patients 128 95

Primary Outcome

MeasuresOverall response rate; efficacy and safety of CTL019

Overall remission rate (ORR) - overall remission rate during

the 6 months after CTL019 administration, which includes

CR and CR with incomplete blood count recovery (CRi) as

determined by IRC assessment

Arms/Intervention Single-arm study of single dose of CTL019 Single-arm study of single dose of CTL019

Target PatientsAdult patients with relapsed or refractory diffuse large B-cell

lymphoma (DLBCL)

Pediatric and young adult patients with relapsed and

refractory B-cell acute lymphoblastic leukemia


Publication

• Schuster et al. at ICML 2017; Schuster et al. at EHA

2017; Schuster et al. at ASH 2017

• Borchmann et al. at EHA 2018

• Manuscript submitted in April 2018

• Grupp et al. at ASH 2016

• Buchner et al at EHA 2017

• Maude et al. N Engl J Med. 2018;378:439-48. DOI:

10.1056/NEJMoa1709866


Exjade® - Iron chelation of bis-hydroxy-phenyl triazole type

76

Study NCT00940602 TELESTO (CICL670A2302)

Indication Iron overload

Phase Phase 2

Patients 224

Primary Outcome

Measures

To compare deferasirox to placebo with regard to event-free

survival in low and int-1 risk MDS patient with transfusional

iron overload

Arms/Intervention• Deferasirox, iron chelator

• Placebo

Target PatientsPatients with myelodysplastic syndromes (low/int-1 risk) and

transfusional iron overload

Expected Completion Q2-2018 (actual)

Publication Congress in Q4-2018; Journal TBD


INC280 - MET Inhibitor

77

Study NCT02414139 (CINC280A2201) NCT02335944 (CINC280X2105C)

IndicationEGFR Wild-type, ALK negative advanced Non-small Cell Lung

Cancer (NSCLC)

Non-small Cell Lung Cancer (NSCLC) Patients With EGFR

Mutation

Phase Phase 2 Phase 1/2

Patients 348 177

Primary Outcome

MeasuresOverall Response Rate (ORR)

Phase II Groups 1, 2 and 3: Overall Response Rate (ORR)

Phase II Group 4: Frequency of treatment-emergent adverse

events

Arms/Intervention

• Pre-treated pts. with MET GCN ≥ 6

• Pre-treated pts. with MET GCN ≥ 4 and < 6

• Pre-treated pts. with MET GCN < 4

• Pre-treated pts. with MET mutations regardless of cMET

GCN

• Treatment-naïve pts. with MET dysregulation

• Pre-treated pts with MET dysregulation – second line

• Group 1: EGFRmut NSCLC developing resistance to

EGFR TKI

• Group 2: EGFR TKI-naïve, EGFRmut NSCLC with denovo

T790M mutation

• Group 3: Treatment-naïve, EGFRmut NSCLC

• Group 4: 1-3L EGFRmut NSCLC (with food)

Target Patients

Adult patients with EGFR wild-type (wt), ALK-negative

advanced non-small cell lung cancer (NSCLC) with either

MET amplification or MET mutations and are either

pretreated with 1 or 2 prior lines of systemic therapy or are

treatment-naïve for the advanced stage of disease

Adult Patients With EGFR Mutated Non-small Cell Lung

Cancer

Expected Completion Q4-2018 Q4-2018

Publication• Congress presentation in Q4-2018

• Manuscript submission Q4-2018Congress presentation in Q2-2019


Jakavi® - JAK1/2 inhibitor

78

Study NCT02913261 REACH2 (CINC424C2301) NCT03112603 REACH3 (CINC424D2301)

Indication Steroid-refractory acute graft vs. Host Disease (SR aGVHD) Steroid-refractory chronic graft vs. Host disease (SR cGVHD)


Patients 308 324

Primary Outcome

MeasuresOverall Response Rate (ORR) at 28 Days Overall Response Rate (ORR) at 183 Days

Arms/Intervention• Ruxolitinib 10mg BID

• Best available therapy (BAT)

• Ruxolitinib 10mg BID

• Best available therapy (BAT)

Target Patients Patients with Steroid-refractory Acute GVHD (SR aGVHD) Patients with steroid-refractory chronic GVHD (SR cGVHD)


Publication TBD TBD


Kisqali® - CDK 4/6 inhibitor

79

Study NCT02422615 MONALEESA-3 (CLEE011F2301) NCT02278120 MONALEESA-7 (CLEE011E2301)

Indication Advanced breast cancer – 1st / 2nd line (with fulvestrant) Advanced breast cancer - 1st line (pre-menopausal)


Patients 727 672

Primary Outcome

Measures

Progression Free Survival (PFS) - time from the date of

randomization to the date of the first documented

progression or death due to any cause

Progression Free Survival (PFS) - time from the date of

randomization to the date of the first documented

progression or death due to any cause and assessed

according to RECIST 1.1

Arms/Intervention• Riblociclib 600mg + fulvestrant 500mg

• Placebo of Riblociclib + fulvestrant 500mg

• LEE011 600 mg + NSAI/tamoxifen + goserelin 3.6 mg

• Placebo of LEE011 + NSAI/tamoxifen + goserelin 3.6 mg

Target Patients

Postmenopausal women with hormone receptor positive,

HER2-negative, advanced breast cancer who have received

no or only one line of prior endocrine treatment

Premenopausal women with hormone receptor positive,

HER2-negative, advanced breast cancer

Expected Completion Q1-2018 (actual) 2017 (actual)

Publication• Slamon D, et al. Oral presented at ASCO 2018

• Manuscript published in JCO June 2018

• Tripathy D, et al. Oral presented at SABCS 2017

• Manuscript published in Lancet Oncology May 2018

(online)


LCI699 - Cortisol synthesis inhibitor

80

Study NCT02697734 LINC-4 (CLCI699C2302) NCT02180217 LINC-3 (CLCI699C2301)

Indication Cushing's disease Cushing's disease


Patients 69 132

Primary Outcome

Measures

Demonstrate the superiority of osilodrostat compared to

placebo in achieving a complete response mean urine free

cortisol ≤ upper limit of normal (mUFC ≤ ULN) at Week 12

Compare the complete response rate at the end of the 8-

week randomized withdrawal period

Arms/Intervention• LCI699 / Osilodrostat

• Placebo

Randomized withdrawal design

• LCI699 / Osilodrostat

• Placebo

Target Patients Patients with Cushing's disease Patients with Cushing's disease

Expected Completion 2020 Q2-2018 (actual)

Publication TBD Congress submission Q4-2018


PDR001 – PD-1 inhibitor

81

Study NCT02967692 COMBI-i (CPDR001F2301)

Indication BRAFV600 mutant metastatic melanoma

Phase Phase 3

Patients 538

Primary Outcome

MeasuresProgression-Free Survival (PFS)

Arms/Intervention

• PDR001 400mg i.v. Q4W + Tafinlar 150mg + Mekinist 2

mg

• Placebo + Tafinlar 150 mg + Mekinist 2 mg

Target Patients

Previously untreated patients with unresectable or

metastatic BRAF V600 mutant melanoma


PublicationCongress presentation and manuscript submission planned

H2-2019


Rydapt®- Multi-targeted kinase inhibitor

82

Study NCT00651261 RATIFY (CPKC412A2301) NCT03280030 (CPKC412A2220)

Indication Acute myeloid leukemia Acute myeloid leukemia


Patients 717 66

Primary Outcome

MeasuresOverall survival Incidence of safety events and event free survival

Arms/Intervention

• Induction and consolidation chemotherapy plus

midostaurin

• Induction and consolidation chemotherapy plus placebo

• Midostaurin 50 mg

• Placebo

Target PatientsNewly diagnosed patients < 60 years of age with FLT3

mutated acute myeloid leukemia (AML)

Newly diagnosed patients with FLT3-mutated acute myeloid

leukemia (AML)

Expected Completion 2016 (actual) H2-2019

Publication

• Stone RM, Manley PW, Larson RA, and Capdeville R.

published February 27, 2018 in Blood Advances

2018;(2:444-453

• H. Gu Drug Metab Dispos. 2018;46(2):109-121

• Planned: Karin Hartman, Haneke Kluin-Nelemans

Journal of Allergy and Clinical Immunology (TBD)

• Planned: Combine into single paper (maintenance and

CIR): Leukemia

• Abstract submitted on May 10, 2018 to 9th JSH

International Symposium


Promacta®/Revolade® – Thrombopoetin receptor agonist

Study NCT03025698 (CETB115E2201)

IndicationPreviously untreated or relapsed/refractory severe aplastic anemia or

recurrent aplastic anemia

Phase Phase 2

Patients 60

Primary Outcome

MeasuresPK of eltrombopag at steady state in pediatric patients with SAA

Arms/Intervention

- Eltrombopag

- Arm B: previously untreated SAA-hATG/cyclosporine +

eltrombopag

- Arm A: relapsed/refractory SAA or AA: hATG/cyclosporine +

eltrombopag or cyclosporine + eltrombopag

Target PatientsPediatric patients from age 1 <18 years with relapsed/refractory SAA

or recurrent AA after IST or previously untreated SAA


Publication TBD


SEG101 – p-Selectin inhibitor

Study NCT03264989 (CSEG101A2202)

IndicationPrevention of Vaso-Occlusive Crises (VOC) in patients with

Sickle Cell Disease (SCD)

Phase Phase 2

Patients 55

Primary Outcome

MeasuresAssess PK/PD of SEG101 (crizanlizumab) at 5 mg/kg

Arms/Intervention

SEG101 (crizanlizumab) at a dose of 5.0 mg/kg (or 7.5

mg/kg for exploratory group) by IV infusion, ±

Hydroxyurea/Hydroxycarbamide

Target Patients Adult sickle cell disease patients with Vaso-Occlusive crises


Publication TBD


Signifor® - Somatostatin Analogue

Study NCT02354508 SWITCH (CSOM230C2413)

Indication Acromegaly

Phase Phase 3

Patients 123

Primary Outcome

Measures

Proportion of patients who achieve biochemical control

defined as GH <1μg/L and IGF-1 <ULN at week 36.

Arms/Intervention• Pasireotide LAR (40 mg)

• Pasireotide LAR (up-titrated 60 mg)

Target Patients Patients with inadequately controlled acromegaly


Publication Congress submission in Q4-2018


Tafinlar®+Mekinist® - BRAF inhibitor and MEK inhibitor

Study NCT01682083 (CDRB436F2301) NCT02124772 (CTMT212X2101)

Indication BRAFV600 mutant adjuvant melanoma BRAFV600 mutant solid tumors

Phase Phase 3A Phase 1

Patients 870 142

Primary Outcome

MeasuresRelapse-free survival (RFS) Safety, tolerability and pharmacokinetics and clinical activity

Arms/Intervention• Dabrafenib 150 mg + trametinib 2 mg

• Placebo

Trametinib (dose based on age and weight)

Dabrafenib + trametinib (dose based on age and weight)

Target PatientsSubjects with BRAFV600 mutation-positive melanoma with

lymph node(s) involvement, after complete resection

Pediatric Subjects Aged 1 Month to <18 Years with

Advanced V600-Mutation Positive Solid Tumors


PublicationLong G.V., et al. N Engl J Med 2017; 377:1813-1823; DOI:

10.1056/NEJMoa1708539TBD


Study NCT01677741 (CDRB436A2102)

Indication BRAFV600 mutant cancers

Phase Phase 1

Patients 86

Primary Outcome

MeasuresSafety, tolerability and pharmacokinetics

Arms/Intervention Single-arm study of oral dabrafenib

Target PatientsPediatric subjects aged 1 year to <18 years with advanced

BRAF V600-mutation positive solid tumors


Publication TBD

Tafinlar® - BRAF inhibitor



Study NCT02039947 COMBI-MB (CDRB436B2204)

Indication BRAFV600 mutant metastatic melanoma

Phase Phase 2B

Patients 126

Primary Outcome

MeasuresIntracranial response (IR) rate

Arms/Intervention Dabrafenib 150 mg + trametinib 2 mg

Target PatientsPatients with BRAF mutation-positive melanoma that has

metastasized to the brain


Publication• MA Davies G.V., et al. Lancet Oncology. 2017.

DOI:10.1016/S1470-2045(17)30429-


Tasigna® - Bcr-Abl, c-Kit and PDGF-R tyrosine kinase inhibitor

Study NCT01698905 ENESTop (CAMN107A2408) NCT01844765 DIALOG (CAMN107A2203)

Indication Second line CML/CML-TFR Newly diag. CML and CML res/intol to imatinib/dasatinib


Patients 163 59

Primary Outcome

Measures

No documented confirmed loss of MR4, no documented loss

of MMR and no re-starting of nilotinib therapy

• Resistant/intolerant Ph+ CML in chronic phase: Rate of

Major Molecular Responder (MMR) at 6 cycles

• Newly diagnosed and untreated Ph+ CML in first chronic

phase: Rate of MMR by 12 cycles

Arms/Intervention • Single-arm study of nilotinib

• Newly diagnosed and untreated Ph+ CML in first chronic

phase

• Resistant/intolerant Ph+ CML in chronic phase

• Resistant/intolerant Ph+ CML in accelerated phase

Target Patients

Adult CML-CP patients who received a minimum of 3 years

of TKI therapy, started off with imatinib treatment for > 4

weeks, then switched to nilotinib for at least 2 years prior to

study entry and achieved MR4.5 on nilotinib, but did not

have documented MR4.5 at the time of switch from imatinib

to nilotinib

Pediatric patients with newly diagnosed Ph+ chronic

myelogenous leukemia (CML) in chronic phase (CP) or with

Ph+ CML in CP or accelerated phase (AP) resistant or

intolerant to either imatinib or dasatinib


Publication Mahon FX, et al. Ann Intern Med. 2018,168(7):461-470• Presentation at SIOP October 13, 2017

• Primary manuscript planned Q2-2018



Study NCT02684058 (CDRB436G2201)

Indication BRAFV600 mutant gliomas

Phase Phase 2

Patients 142

Primary Outcome

MeasuresOverall Response Rate (ORR)

Arms/Intervention Dabrafenib + trametinib (dose based on age and weight)

Target Patients

Children and adolescent patients with BRAF V600 mutation

positive relapsed or refractory high grade glioma (HGG) or

BRAF V600 mutation positive low grade glioma (LGG)


Publication TBD


Votrient® – TKI post-IO

Study NCT03200717 IO-PAZ (CPZP034A2410)

Indication Renal cell carcinoma

Phase Phase 2

Patients 100

Primary Outcome

Measures

Progression free survival (PFS) using local assessment,

according to RECIST v1.1

Arms/Intervention Single-arm: pazopanib 800 mg

Target Patients

Patients with advanced and/or metastatic renal cell

carcinoma after previous therapy with checkpoint inhibitor

treatment


Publication • TBD


Zykadia® - ALK inhibitor


Study NCT02299505 ASCEND-8 (CLDK378A2112)

Indication ALK activated NSCLC

Phase Phase 2

Patients 306

Primary Outcome

Measures

Part 1: Pharmacokinetics when taken with food

Part 2: Overall response rate (ORR) when taken with food

Arms/Intervention

• Oral LDK378 450 mg once daily taken with food

• Oral LDK378 600 mg once daily taken with food

• Oral LDK378 750 mg once daily fasted

Target PatientsAdult patients with ALK-rearranged (ALK-positive) advanced

non-small cell lung cancer

Expected CompletionPart 1 (PK): 2016 (actual)

Part 2 (ORR): Q2-2018 (actual)

Publication

• Part 1 (PK): Cho BC, et al. Journal of Thoracic Oncology;

2017 Jul; 12(9) 1357-1367

• Part 2 (ORR): Congress presentation Q4-2018;

Manuscript submission Q3-2018

Ophthalmology

Lucentis® - Anti-VEGF

94

Study NCT02375971 RAINBOW (CRFB002H2301) NCT02640664 RAINBOW Extension (CRFB002H2301E1)

Indication Retinopathy of Prematurity (ROP) Retinopathy of Prematurity (ROP)


Patients 224 180

Primary Outcome

Measures

To achieve absence of active Retinopathy of Prematurity

(ROP) and unfavorable structural outcome, patients must

fulfill all the following criteria, 1) survival, 2) no intervention

with a second modality for ROP, 3) absence of active ROP

and 4) absence of unfavorable structural outcome

To evaluate the visual function of patients by assessing the

visual acuity in the better-seeing eye at the patient’s fifth

birthday.

Arms/Intervention

• Ranibizumab 0.2 mg


• Laser therapy



Target PatientsMale and female preterm infants with bilateral retinopathy of

prematurity (ROP) who require treatment.

Male and female preterm infants with bilateral retinopathy of

prematurity (ROP) who require treatment.


PublicationEURETINA: Sep. 2018, AAO: Oct 2018,

Primary manuscript: planned submission end of 2018TBD


RTH258 - Anti-VEGF

Study NCT02434328 HARRIER (CRTH258A2302) NCT02307682 HAWK (CRTH258A2301)

Indication Neovascular age-related macular degeneration (nAMD) Neovascular age-related macular degeneration (nAMD)


Patients 860 990

Primary Outcome

Measures

Change in Best Corrected Visual Acuity (BCVA) from

baseline at week 48

Change in Best Corrected Visual Acuity (BCVA) from

baseline at week 48

Arms/Intervention• RTH258 (6 mg/50 µL)

• Aflibercept (2 mg/50 µL)

• RTH258 (3 mg/50 µL)

• RTH258 (6 mg/50 µL)

• Aflibercept

Target Patients Subjects with exudative age-related macular degeneration Subjects with exudative age-related macular degeneration

Expected Completion Q1-2018 (actual) Q2-2018 (actual)

PublicationAbstract/presentation at AAO meeting in Nov-2017 and

Nov-2018

Abstract/presentation at AAO meeting in Nov-2017 and

Nov-2018


RTH258 - Anti-VEGF

Study NCT03386474 (CRTH258A2301E1)

Indication Neovascular age-related macular degeneration (nAMD)

Phase Phase 3

Patients 150

Primary Outcome

MeasuresNumber of treatment-emergent adverse events

Arms/Intervention• RTH258 (6 mg)

• Aflibercept (2 mg)

Target PatientsPatients with neovascular age-related macular degeneration

who have completed the CRTH258A2301 study


Publication TBD


Respiratory

QAW039 – DP2 (CRTh2) antagonist

98

Study NCT02555683 LUSTER-1 (CQAW039A2307) NCT02563067 LUSTER-2 (CQAW039A2314)

Indication Asthma Asthma


Patients 846 846

Primary Outcome

Measures

Reduction in the rate of moderate-to-severe asthma

exacerbations

Reduction in the rate of moderate-to-severe asthma

exacerbations

Arms/Intervention

• QAW039 Dose 1

• QAW039 Dose 2

• Placebo

• QAW039 Dose 1

• QAW039 Dose 2

• Placebo

Target Patients Patients with uncontrolled severe asthma Patients with uncontrolled severe asthma

Expected Completion 2020 H2-2019

Publication TBD TBD


QAW039 - DP2 (CRTh2) antagonist

Study NCT03215758 ZEAL-1 (CQAW039A2316) NCT03226392 ZEAL-2 (CQAW039A2317)



Patients 650 650

Primary Outcome

MeasuresPre-dose forced expiratory volume in 1 second (FEV1) Pre-dose forced expiratory volume in 1 second (FEV1)

Arms/Intervention• QAW039

• Placebo

• QAW039

• Placebo

Target Patients Patients with uncontrolled asthma Patients with uncontrolled asthma


Publication TBD TBD


QAW039 - DP2 (CRTh2) antagonist

Study NCT03052517 SPIRIT (CQAW039A2315)

Indication Asthma

Phase Phase 3

Patients 1,900 – 2,300

Primary Outcome

Measures

Long term safety: treatment emergent adverse event (AE),

SAE and AE leading to discontinuation from study (52 wks

and 160 wks)

Arms/Intervention

• QAW039 Dose 1

• QAW039 Dose 2

• Placebo

Target Patients Patients with moderate to severe asthma


Publication TBD


QMF149 - Long-acting beta2 agonist and inhaled corticosteroid

101

Study NCT02892019 (CQMF149G2202)

Indication Asthma

Phase Phase 2

Patients 80

Primary Outcome

MeasuresTrough FEV1

Arms/Intervention• Indacaterol acetate 75 μg (via Concept1 inhaler)

• Indacaterol acetate 150 μg (via Concept1 inhaler)

Target PatientsChildren greater or equal to 6 and less than 12 years of age

with asthma


Publication TBD


QVM149 - LA beta2 agonist, LA muscarinic antagonist, ICS

Study NCT02554786 PALLADIUM (CQVM149B2301) NCT02571777 IRIDIUM(CQVM149B2302)




Primary Outcome

MeasuresTrough FEV1 Trough FEV1

Arms/Intervention

• QMF149 150/160 µg od

• QMF149 150/320 µg od

• MF 400 µg od

• MF 400 µg bid

• Salmeterol 50 µg /fluticasone 500 µg bid

• QVM149 150/50/160 µg od

• QVM149 150/50/80 µg od

• QMF149 150/160 µg od

• QMF149 150/320 µg bid

• Salmeterol 50 µg /fluticasone 500 µg bid

Target Patients

Adult and adolescent patients with uncontrolled asthma

despite med-/high-dose ICS or low-dose ICS/LABA(GINA

step 3)

Adult patients with uncontrolled asthma despite med/high-

dose ICS/LABA (GINA ≥4)


Publication TBD TBD



103

Study NCT03100500 (CQVM149B1305) NCT03100825 (CQVM149B1304)



Patients 51 94

Primary Outcome

Measures

Number of patients who reported treatment emergent

adverse events during the 52 weeks of the study

Number of patients who reported treatment emergent

adverse events during the 52 weeks of the study

Arms/Intervention • Single arm: QMF149 150/320 μg od• Single Arm: QVM149 150/50/160 μg od(Concept1

inhaler)

Target Patients Japanese patients with asthma Japanese patients with Asthma

Expected Completion Q1-2019 H1-2019

Publication TBD TBD



Study NCT02892344 (CQVM149B2303) NCT03158311 ARGON (CQVM149B2306)



Patients 750 1,251

Primary Outcome

MeasuresTrough FEV1

Change from baseline in Asthma Quality of Life

Questionnaire (AQLQ) total score

Arms/Intervention• QMF149 150/80 µg

• MF 200 µg

• QVM149 150/50/80 μg

• QVM149 150/50/160 μg

• Salmeterol/fluticasone 50/500 μ + tiotropium 5 μg

Target PatientsAdult and adolescent patients with in poorly (i.e.,

inadequately) controlled asthmaPatients with uncontrolled asthma

Expected Completion Q1-2019 H2-2019

Publication TBD TBD



Study NCT03137784 (CQVM149B2204)

Indication Asthma

Phase Phase 2

Patients 148

Primary Outcome

Measures

Evaluate the bronchodilator effects of NVA237 (25 ug and 50

ug) compared to placebo in terms of trough FEV1

Arms/Intervention• NVA237 (glycopyrronium bromide) 25/50 μg

• Placebo

Target Patients Asthma patients


Publication TBD


Xolair® – anti-IgE antibody

106

Study NCT03369704 (CIGE025F1301)

Indication Seasonal allergic rhinitis: Severe Japanese Cedar Pollinosis

Phase Phase 3

Patients 346

Primary Outcome

Measures

Mean nasal symptom score, consists of severity of

sneezing, rhinorrhea and nasal congestion.

Arms/Intervention

In addition to standard of care:

• Omalizumab per approved allergic asthma dosing table

for IgE/body weight combinations

• Placebo

Target Patients

Patients with severe Japanese cedar pollinosis, whose

symptoms were inadequately controlled with current

recommended therapies


Publication TBD


Sandoz Biopharmaceuticals

Erelzi® - Biosimilar etanercept

108

Study NCT02638259 (GP15 301)

Indication Immunology

Phase Phase 3

Patients 376

Primary Outcome

Measures

Change in DAS28-CRP score from baseline to week 24 in

patients treated with GP2015 and patients treated with Enbrel

Arms/Intervention• GP2015 50 mg

• EU-authorized Enbrel® 50mg

Target Patients Patients with moderate to severe, active rheumatoid arthritis


Publication

• Kavanaugh et al. Arthritis Rheumatol 2017; 69 (suppl 10)

• 48 week: Abstract to EULAR 2018

• 24 week: Manuscript to Ann Rheum Dis

• 48 week: Manuscript in Q3-2018 (journal TBD)


Rixathon® - Biosimilar rituximab

109

Study NCT02514772 (GP13 302) NCT01419665 (GP13 301)

Indication Immunology Oncology


Patients 107 629

Primary Outcome

Measures

Incidence of adverse events and serious adverse events,

anaphylactic reactions, hypersensitivity; immunogenicity Overall response rate in patients with FL

Arms/Intervention• GP2013

• Rituxan® or MabThera®

• GP2013

• MabThera®

Target PatientsPatients with active Rheumatoid Arthritis, previously treated

with Rituxan or MabThera (ASSIST-RT)

Patients with previously untreated, advanced stage follicular

lymphoma (ASSIST-FL)

Expected Completion 2016 (actual) Q2-2018 (actual)

Publication• ACR Q4-2017 Poster

• Manuscript planned Q2-2018

• Amersdorffer J, et al and Jurczak W., et al presented at

ESMO 2017, Published in Lancet Hematology (doi:

10.1016/ S2352-3026(17)30106-0)


GP2017 - Biosimilar adalimumab

110

Study NCT02016105 ADACCESS (GP17-301) NCT02744755 ADMYRA (GP17-302)

Indication Immunology Immunology


Patients 465 353

Primary Outcome

Measures

PASI 75 response rate at week 16 in patients treated with

GP2017 and patients treated with Humira®

Change in DAS28-CRP score from baseline to week 12 in

patients treated with GP2017 and patients treated with

Humira®

Arms/Intervention• GP2017

• Humira® Adalimumab

• GP2017

• US licensed Humira® Adalimumab

Target PatientsPatients with moderate to severe chronic plaque-type

psoriasisPatients with moderate to severe active rheumatoid arthritis

Expected Completion 2016 (actual) Q4-2017 (actual)

Publication

• Abstract and poster at ACR 2018

• Manuscript with study results British Journal of

Dermatology (BJD) – accepted in May 2018

• Blauvelt et. al. presented at AAD 2017, Blauvelt et.al.

presented at EADV 2017, Blauvelt et al., presented at

ACG 2017, Blauvelt et.al. presented at UEGw 2017

• Abstract and poster at ACR 2018, EULAR 2019

• Manuscript with study results journal TBD


Established Medicines and Anti-infectives

Tobramycin – An aminoglycoside antibiotic

112

Study NCT02712983 iBEST-1 (CTBM100G2202)

Indication Bronchiectasis

Phase Phase 2

Patients 180

Primary Outcome

MeasuresP. aeruginosa density in sputum

Arms/Intervention

Three dose regimens, each of them having 3 treatment

arms:

• Tobramycin inhalation powder

• Tobramycin inhalation powder and placebo

• Placebo

Target PatientsPatients with non-cystic fibrosis bronchiectasis and

pulmonary P. aeruginosa infection


Publication TBD


KAF156 – Plasmodium Falciparum Inhibitor – PfCARL mediated

113

Study NCT03167242 (CKAF156A2202)

Indication Malaria

Phase Phase 2

Patients 512

Primary Outcome

Measures

PCR-corrected adequate clinical and parasitological

response (ACPR)

Arms/Intervention• KAF156 and LUM-SDF (different combinations)

• Coartem

Target PatientsAdults and children with uncomplicated Plasmodium

Falciparum Malaria


Publication TBD


KAE609 – Plasmodium Falciparum Inhibitor – spiroindolone against PfATP4

114

Study NCT03334747 (CKAE609A2202)

Indication Malaria

Phase Phase 2

Patients 150

Primary Outcome

Measures

CTCAE grades increase from baseline in alanine

aminotransferase (ALT) or aspartate aminotransferase

(AST)

Arms/Intervention• KAE609

• Coartem

Target Patients Adults with uncomplicated Plasmodium Falciparum malaria


Publication TBD


Key definitions and trademarks


This presentation contains several important words or phrases that we define as below:AE: Adverse Event

ALL: Acute lymphatic leukemia

AMD: Age-Related Macular Degeneration

AML: Acute myeloid leukemia

Approval: In Pharmaceuticals and Alcon in US and EU; each indication and regulator combination counts as

approval; excludes label updates, CHMP opinions alone and minor approvals

aRCC: advanced renal cell cancer

AS: Ankylosing Spondylitis

bid: twice a day

BC: Breast cancer

BCMA: B-cell maturation antigen

BCVA: best corrected visual acuity

BS: Biosimilars

BTD: Breakthrough therapy designation

CGRP: Calcitonin gene-related peptide

CLL: Chronic lymphocytic leukemia

CM: Chronic migraine

CML: Chronic myeloid leukemia

COPD: Chronic Obstructive Pulmonary Disease

CR: complete remission

CRC: Colorectal Cancer

CSU / CIU: Chronic spontaneous urticaria / Chronic idiopathic urticaria

CVRR: Cardiovascular risk reduction

DLBCL: Diffuse large B-cell lymphoma

DMC: Data monitoring committee

EF: ejection fraction

EM: Episodic migraine

FL: Follicular lymphoma

FPFV: First patient first visit

GBM: Glioblastoma multiforme

HF: Heart failure

HF-pEF: Heart failure with preserved ejection fraction

HFrEF: Heart failure with reduced ejection fraction

HR+/HER2- mBC:Hormone Receptor positive / Human Epidermal growth factor receptor 2 negative metastatic

breast cancer

LoE: Loss of exclusivity

M/M: Multiple myeloma

MF: Myelofibrosis

MI: Myocardial infarction

MS: Multiple sclerosis

NASH: Non-Alcoholic Steatohepatitis

NET: Neuroendocrine tumor

NSCLC: Non-small cell lung cancer

NTD: New Therapeutic Drug

od: once a day

ORR: Overall response rate

OS: Overall survival

PA: Prior authorization

PASI 90: 90% reduction in Psoriasis Area Severity Index from baseline

PFS: Progression free survival

PsA: Psoriatic arthritis

PsO: Psoriasis

PV: Polycythemia vera

PY: Prior year

QoL: Quality of Life

RCC: Renal cell cancer

r/r ALL: relapsed/refractory acute lymphoblastic leukemia

RRMS: relapsing-remitting multiple sclerosis

SCPC: Sickle cell pain crisis

SpA: Spondyloarthropathy

SPMS: Secondary progressive multiple sclerosis

TFR: Treatment-free Remission

TNBC: Triple negative breast cancer

Trademarks

Eylea® is a registered trademark of Regeneron Pharmaceuticals, Inc.

Enbrel®, Epogen® and Neulasta® are a registered trademark of Amgen Inc.

Humira® is a registered trademark of AbbVie Ltd.

Remicade® and Stelara® are registered trademarks of Janssen Biotech, Inc.

Rituxan® is a registered trademark of Biogen Inc

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