q3 2018 results - novartis · core ros 18.6% (+1.3% pts) alcon q3 net sales usd million, % cc 788...

Q3 2018 Results

Investor Presentation

October 18, 2018

Novartis AG

Investor Relations

Disclaimer

Novartis Q3 2018 Results | October 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 potential financial or other impact on Novartis, and the potential strategic benefits, synergies or opportunities expected as a result of the

proposed spinoff of our Alcon Division or of the proposed divestiture of certain portions of our Sandoz Division in the US or of the proposed acquisition of Endocyte; 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

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portions of our Sandoz Division in the US or the proposed acquisition of Endocyte will receive necessary approvals, or that they 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 transactions. Nor can there be any guarantee

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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

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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;

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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.

Additional Information and Where to Find It

This communication may be deemed to be solicitation material in respect of the proposed acquisition of Endocyte by Novartis AG. In connection with the proposed acquisition, Endocyte intends to file relevant

materials with the SEC, including a proxy statement in preliminary and definitive form. Stockholders of Endocyte are urged to read these materials (including any amendments or supplements thereto)

and all other relevant documents filed with the SEC when such documents become available, including Endocyte’s definitive proxy statement, because they will contain important information

about the proposed acquisition. Investors and security holders are able to obtain the documents (once available) free of charge at the SEC’s web site, http://www.sec.gov, or from Endocyte by going to its

investor relations web site at http://investor.endocyte.com/investor-relations

Participants in Solicitation

Novartis AG and its directors and executive officers, and Endocyte and its directors and executive officers, may be deemed to be participants in the solicitation of proxies from the holders of Endocyte shares of

common stock in respect of the proposed acquisition. Information about the directors and executive officers of Novartis AG is set forth in the excerpts of Novartis AG’s Annual Report for 2017, which was

furnished to the SEC on Form 6-K on January 24, 2018 and incorporated by reference into Novartis AG’s Annual Report on Form 20-F for the fiscal year ended December 31, 2017. Information about the

directors and executive officers of Endocyte is set forth in the proxy statement for Endocyte’s 2018 Annual Meeting of Stockholders, which was filed with the SEC on March 23, 2018. Information regarding

interests of Novartis AG’s and Endocyte’s respective participants in the solicitation, will be set forth in the proxy statement relating to the proposed acquisition and other materials to be filed with the SEC in

connection with the proposed acquisition.

Agenda

1 Group review Vas Narasimhan

2 Financial review Harry Kirsch

3 Closing Vas Narasimhan

4 Q&A Team


Delivered strong accretive growth in Q3, with key

innovation milestones on advanced therapy platforms

4 Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation

Group performance, % cc

Sales Core OpInc

+6%

+9%

Innovation milestones

AVXS-101

BAF312

Endocyte1

1. The acquisition of Endocyte is subject to customary closing conditions, including receipt of regulatory approvals and Endocyte stockholders approval. Until closing, Endocyte will continue to operate as a separate and independent company

Q3 9M

SalesUSD million

Growth vs. PYUSD million cc

SalesUSD million

Growth vs. PYcc

750 37% 2,031 37%

271 113% 710 117%

295 32% 844 37%

291 33% 842 31%

Lutathera® 56 nm1 86 nm1

248 27% 721 27%

72 184% 175 nm1

20 nm1 48 nm1

56

67

46

194

47

143

68

20

Strong performance driven by Innovative Medicines

5 Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation

1. Not meaningful

Key growth drivers

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

Cosentyx®

established as leading differentiated

IL-17A inhibitor


Sales evolutionUSD million, % cc

Q3 2017 Q3 2018

556

750Ex-US

US

+37%

1. Humira is a registered trademark of AbbVie Inc. 2. TRx Share in Rheum specialty, IMS TRx allocated using SHS APLD (June 2018) factors where only PsO/PsA/AS uses are carved in for TNFs

Q3 sales USD 750m (+37% cc)

Strong growth momentum across all indications

and geographies

Cosentyx® surpassed Humira®1 in TRx in PsA2

in US

Five year data in PsA and AS show sustained

benefits, complementing long-term data in

psoriasis

Cosentyx®

gaining share in a growing and

competitive psoriasis market


10%

13%

12M ending 7/2017 12M ending 7/2018

+31%

Cosentyx® OthersStelara® TNFs Tremfya®

Dermatology US NRx evolution1

Cosentyx® prescriptions (NRx) +64%1 in a

growing US biologics market (+31%)1

Cosentyx® effectively treats the multiple

manifestations of the psoriatic disease

2/3 of patients have - beyond plaque psoriasis -

scalp, nail, palmoplantar and/or joint involvement2

Cosentyx® real-world evidence confirms safety

and efficacy benefits

1. NRx Share in Dermatology from SHS June 2018. NRx Share amongst Dermatology writers 2. Data on File. Corrona LLC. Corrona Report: Real-World Data From the Corrona Psoriasis Registry® June 15, 2018

All trademarks are the property of their respective owners

Entresto®

sales driven by strong uptake in all

launched markets


Q3 US sales USD 151m (+104% cc),

ex-US sales USD 120m (+126% cc)

TRANSITION data show Entresto® can be

initiated early and safely in hospitalized patients1

PIONEER study to be presented as late-breaker

at AHA in November

PARAGON study continues as planned following

interim analysis, results expected mid-2019

Sales evolutionUSD million, % cc

Q3 2017 Q3 2018

128

271

+113%

1. Wachter R. et al., Initiation of sacubitril/valsartan in hospitalized patients with reduced ejection fraction heart failure after hemodynamic stabilization: Primary results of TRANSITION study. Data presented at: ESC 2018, Aug 25-29

Ex-US

US

Lutathera®

off to a strong start in US and gaining

reimbursement in Europe


Strong launch momentum with Q3 sales of

USD 56m (YTD sales of USD 86m)

>1,100 patients in US October YTD1

>85 centers in US actively prescribing

Broad US payer coverage with 70% of lives

covered

2 months after UK reimbursement, 18 centers

actively prescribing52

399

1'123

Q2 2018Q1 2018 Q3 2018

1. Data as of October 16, 2018

US – number of doses

Q3 2018 performance

Sandoz continuing to execute strategy of focusing on

complex generics and biosimilars


Sandoz sales -4% (cc) as US sales

declined -17% (cc) due to continued

industry-wide pricing pressure

Ex-US sales grew +2% (cc)

Global Biopharmaceuticals grew +21% (cc)

Optimizing our core Gx business:

Divesting US oral solids and dermatology

(~USD 0.6bn sales in H1 2018) to Aurobindo1

Expanding biosimilars in Europe:

Ramping up Rixathon® and Erelzi®

Approval for Hyrimoz® and Zessly®

Positive CHMP opinion for pegfilgrastim

Driving portfolio differentiation

1. Announced on September 6th, 2018 agreement to sell selected portions of Sandoz US portfolio (US dermatology business and US oral solids portfolio) to Aurobindo Pharma USA Inc; expected completion 2019

11

Continued momentum with Q3 sales +5% (cc):

– Surgical +7% (cc) mainly AT-IOLs and

consumables

– Vision Care +3% (cc) with double digit

growth of Dailies Total1® and Systane ®

Q3 core operating income grew +1% (cc),

reflecting impact of growth investments

YTD core operating income grew +14% (cc),

core ROS 18.6% (+1.3% pts)

Alcon Q3 net salesUSD million, % cc

788797

Q3 2017 Q3 2018

+3%

924

966

Q3 2017 Q3 2018

+7%

Surgical Vision Care

Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation

Alcon continues to show solid sales growth as a result

of improved operations and customer relationships

Key data readouts and regulatory milestones in Q3


ADHF – acute decompensated heart failure Copaxone® is a registered trademark of Teva Pharmaceuticals LTD 1. Submission in Japan initiated

Advanced

therapy

platforms

AVXS-101

SMA Type 1

New analysis reiterates rapid onset of action (WMS 2018)

Simultaneous global submissions in US, EU, and Japan1

Kymriah® EU approvals in pALL and DLBCL

Luxturna® CHMP positive opinion

Potential

first-in-class

therapies

BAF312 Filed for SPMS in US and EU

BYL719 SOLAR-1 study met its primary endpoint, ESMO 2018 presentation

Driving

in-line

brands

Gilenya® Superiority vs. Copaxone® (ASSESS study)

Entresto® HFrEF: PIONEER-HF data to be presented at AHA

HFrEF: TRANSITION demonstrated initiation of Entresto® shortly after stabilization of ADHF well tolerated

HFpEF: PARAGON following interim analysis, study continue as planned with top-line results expected mid-2019

Aimovig® EU approval for migraine prevention

Kisqali® MONALEESA 3 and 7 data added to US label

Tafinlar® +

Mekinist®EU approval for adjuvant melanoma

Biosimilar

pegfilgrastim

CHMP positive opinion

ACZ885 Cardiovascular risk reduction: FDA Complete Response Letter October 2018

Speed: Rapid increase in mean CHOP-INTEND compared

to Spinraza® (nusinersen)4

AVXS-101 START demonstrated transformational

efficacy in Type I SMA (>90% unassisted sitting2)


Rapid onset, sustained efficacy regardless of severity

Baseline Mean age 3.4 months; swallow function 7/12 (58%); CHOP-INTEND

28 points

Efficacy 15/15 (100%) alive and without need for permanent ventilation at 24

months4

9/10 (90%) achieved CHOP-INTEND ≥ 40 by 8 months1

11/12 (92%) achieved CHOP-INTEND >50 during the 24-month study2

11/12 (92%) achieved sitting unassisted during the 24-month study

Durability Continue to achieve major motor milestones with gene therapy alone

during long-term follow-up study2,3

Data is from Cohort 2 in clinical trial, final analysis N=12 1. At day 245 (8 mos), 10 out of 12 Cohort 2 patients in pivotal study had an assessment 2. Two-year data presented as an oral presentation at AAN 2018 3. Data on file. LTFU

data presented as an oral presentation at AAN 2018 4. Poster P.182 at WMS 2018 Spinraza® is a registered trademark of Biogen MA Inc.

Simultaneous regulatory license applications show

significant progress for AVXS-101 in SMA Type 1


Initiated submission in mid-September, anticipate completion by year end

Potential approval in 1H 2019

Sakigake designation

Japan

BLA submitted to FDA at the end of Q3

Potential approval in 1H 2019 (6 months from file acceptance)

Breakthrough Therapy designation

US

MAA submitted to EMA in early Q4

Potential approval in mid-2019 (7 months from file acceptance)

PRIME designation

EU

AVXS-101 clinical development program continues to

enroll rapidly


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 2018, 6 patients enrolled

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

Initiated in Q1 2018, patient enrollment complete

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

Initiated Q2 2018, 6 patients enrolled

REACHSingle i.t. dose

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

12 patients enrolled2

i.v. – Intravenous; i.t. – Intrathecal 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


Novartis announces agreement to acquire Endocyte,

expanding nuclear medicines platform1

High unmet need

in prostate cancer

Prostate cancer expected to be USD 11bn market globally in 20242

177Lu-PSMA-617 expected to provide an additional treatment option, in particular for

refractory patients

First-to-market

potential in lead

product

177Lu-PSMA-617 potentially first-in-class PSMA radioligand therapy (RLT) in mCRPC;

enrollment of Ph3 initiated3

First therapeutic targeting PSMA of any kind, with potential applications for soft tissue,

bone disease and prostate cancer

Significantly de-risked profile given strong Ph2 and preclinical data, and regulatory

discussions4

Expand nuclear

medicines platform

Adds potential second RLT to follow the successful launch of Lutathera®

Potential to further develop 177Lu-PSMA-617 to enter earlier lines of therapy

Potential to expand platform via 225Ac-PSMA-617

mCRPC – metastatic castration-resistant prostate cancer PSMA – prostate-specific membrane antigen 1. The acquisition of Endocyte is subject to customary closing conditions, including receipt of regulatory approvals and Endocyte

stockholders approval. Until closing, Endocyte will continue to operate as a separate and independent company 2. EvaluatePharma, October 2018 3. Endocyte announced enrollment of first patient on June 5, 2018 4. See Endocyte press

releases on June 5, 2018 and September 10, 2018


177Lu-PSMA-617 uses a small molecule ligand to target

a radioactive atom to PSMA-expressing cancer cells

Source: Endocyte Investor Presentation October 2018

RLT that utilizes high affinity targeting ligand to direct potent

radiotherapy to prostate cancer cells

177Lu-PSMA-617 pairs PSMA targeting ligand (PSMA-617) to

radioactive atom (177Lutetium)

“Ligand” is a small molecule designed to bind to PSMA, a protein

highly expressed on the cell surface of most prostate cancer cells

Once bound, the 177Lutetium atom releases an energetic beta

particle that kills the cancer cell


Strong Ph2 clinical data

PSA PFS50 patients

Overall SurvivalFirst 30 patients

Patients

PSA response%

Sustained response rates

in Ph2 trial expansion1

PSA PFS and OS correlate to PSA response

and compare favorably to historical benchmarks1

Months

p-value comparing PSA <50%

group to PSA ≥ 50% group;

Updated data cut-off since

Lancet publication1

Su

rviv

al

Pro

bab

ilit

y

<30%13/50 (26%)

≥30%37/50 (74%)

≥50%31/50 (62%)

50

00

-50

-100

Su

rviv

al

Pro

bab

ilit

y

Source: Endocyte Investor Presentation October 2018 1. Hofman, Michael (2018, June). Lutetium-177 PSMA617 theranostics in mCRPC: interim results of a phase 2 trial. ASCO 2018, Genitourinary cancer P5040


Pivotal Ph3 VISION trial design with FDA agreement

to rPFS as alternative primary endpoint to OS1

177Lu-PSMA-617

Choice of NAAD3

or not

Best supportive

care2

Best supportive

care2

Choice of NAAD3

or not

2:1

Stratified

for balance

Source: Endocyte Investor Presentation October 2018 1. Endocyte stated demonstrating benefit in rPFS (radiographic Progression Free Survival) versus control, with no detriment to OS, sufficient for full approval; regardless of the

outcome of rPFS assessment, Endocyte intends to continue to follow patients in VISION trial to assess final OS alternative primary endpoint as per Endocyte press release on September 10, 2018 2. Best supportive care - palliative 3.

NAAD - novel androgen axis drug (abiraterone or enzalutamide)

750 patients, enrollment

initiated

In September, FDA agreed

to rPFS as an alternative

primary endpoint to OS as

sufficient for full approval1

Key secondary endpoints:

ORR, time to symptomatic

skeletal events

Patient inclusion:

mCRPC

Bone and/or soft tissue

disease

PSMA-positive scan (~80%)

≥1 prior taxane

≥1 prior NAAD3


Transaction highlights

Financing Funded through existing cash

Financial

Benefits

Expected to contribute to Group sales in 2021

Attractive IRR

Other

Transaction unanimously approved by the Boards of Directors of both companies

Closing is subject to Endocyte stockholder approval and satisfaction of customary closing

conditions

Endocyte stockholders to receive USD 24 per share in cash

Values Endocyte at approximately USD 2.1bn on a fully diluted equity basisConsideration


Expected next steps

Endocyte to file a preliminary proxy statement with the SEC relating to a shareholders

meeting to be held to obtain shareholders approval of the transaction

Closing by H1 2019, subject to an affirmative vote by Endocyte’s stockholders to

adopt the merger agreement and satisfaction of customary closing conditions

Novartis

transaction

Publications on other ongoing investigator initiated clinical trials of 177Lu-PSMA-617 in

prostate cancer patients expected in 2018

Endocyte stated rPFS endpoint expected end 2019 and final OS endpoint end 20201

Endocyte

development and

regulatory status

1. Endocyte Investor Presentation October 2018 Note: The acquisition of Endocyte is subject to customary closing conditions, including receipt of regulatory approvals and Endocyte stockholders approval. Until closing, Endocyte will

continue to operate as a separate and independent company

Previewing R&D and investor update on November 5


Additional detailed updates

Entresto® pEF and PIONEER-HF

Cosentyx® clinical development program

QAW039 program

BAF312, OMB157, Gilenya®

RTH258 2-year data

Oncology late-stage pipeline update

Deep dive

AVXS-101

October 22, 2018: ESMO 2018 investor call including BYL719 SOLAR-1 data

Note: November 5: R&D and investor update and live audio webcast; main presentation starts at 13.00 h UK time and will finish at 17.30 h

Agenda




4 Q&A Team


% USD % cc % USD % cc

Net Sales 12,779 3 6 38,631 7 5

Core Operating income 3,555 5 9 10,436 8 7

Operating income 1,939 -18 -13 6,870 5 3

Net Income 1,624 -22 -18 11,420 nm nm

Core EPS (USD) 1.32 2 6 3.90 7 5

EPS (USD) 0.70 -21 -17 4.92 nm nm

Free Cash Flow 3,301 8 8,778 10

Change vs. PYGroup

1

USD million

Q3

2018

9M

2018

Change vs. PY

Summary of Q3 and 9M 2018 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 55 of the Condensed Interim Financial Report 2. Not meaningful due to the current

year benefit from a USD 5.7 billion net gain recognized from the sale of our stake in the GSK consumer healthcare joint venture in the second quarter

2

Innovative Medicines driving Q3 margin expansion;

9M margin increase driven by IM Division and Alcon


Q3 2018 9M 2018

Net sales

change vs. PY

Core operating

income

change vs. PY

Core

margin

Core margin

change vs. PY

Core

margin

Core margin

change vs. PY

% cc % cc % %pts cc % %pts cc

Innovative Medicines 9 16 33.7 2.1 32.4 1.1

Sandoz -4 -3 22.4 0.2 20.5 0.2

Alcon 5 1 17.1 -0.7 18.6 1.3

Group 6 9 27.8 0.8 27.0 0.5

Alcon 9M 2018 core margin at 18.6%, expanding vs.

prior year trough

26

23.8%18.6%

9M 2015 9M 2016 9M 2017 9M 2018

18.5% 17.3%

1. Alcon Division 9M 2015, 9M 2016 and 9M 2017 net sales and core margin restated to include the Ophthalmic OTC and Diagnostics products, transferred from the Innovative Medicines Division from January 1, 2018

Expected key drivers to expand core

margin to low-to-mid 20’s by 2023:

+ Improved gross margin from sales

uptake, favorable product mix and

manufacturing efficiencies

+ Cost leverage from existing

infrastructure and reallocating

resources to growth drivers


Alcon core margin1 (% of USD sales)

5.05.1

9M 2015 9M 2016 9M 2017 9M 2018

4.9

5.4

Alcon net sales1 (USD bn)

2018 Group full year guidance: Sales revised

upwards, core operating income confirmed


Barring unforeseen events (in cc)

Full year guidanceGrowth vs. PY in cc

Group Sales revised upwards to expected to grow mid single digit

IM Division revised upwards to grow mid to high-single digit

Sandoz to decline low single digit

Alcon to grow mid single digit

Group Core operating income expected to grow mid to high single digit

Significant Fx headwinds expected in H2 and 2019


Currency impact vs. PYIn %pts, assuming mid-October exchange rates prevail in 2018

FX impact on

Net sales

FX impact on

Core operating income

Simulation

-1

62

-3 -3 -2 -1

72

-4 -5

Q2 FYFYQ4FY Q1 FYQ3 Q1 Q2

0

Q3

0

Q4 FY FY

0

-3 to -4

2017 2018

Actual

2019 2017 2018 2019

Agenda




4 Q&A Team


Conclusion


Innovation momentum continued

On track for full year guidance

Delivered strong accretive growth

Progression of advanced therapy platform, including agreement

to acquire Endocyte

Agenda




4 Q&A Team


Appendix


Plaque psoriasis

34% n=699

with psoriatic arthritis

38% n=784with scalp involvement

38% n=778

with palmoplantar

involvement

11% n = 223

with nail involvement

16% n = 326

2/3 of patients have – beyond plaque psoriasis –

scalp, nail, palmoplantar and/or joint involvement

1. Data on File. Corrona LLC. Corrona Report: Real-World Data From the Corrona Psoriasis Registry® June 15, 2018.

Only 34% have plaque

psoriasis alone

Gilenya 0.5mgGilenya 0.25mgCopaxone 20mg

Gilenya®

ASSESS trial shows superior efficacy over

Copaxone®

in relapsing-remitting MS


ARR - Annualized relapse rate AE - Adverse events NS – Not statistically significant 1. Post-approval commitment to FDA; randomized, controlled Ph4 study; 1064 patients 2. A formal comparison of fingolimod 0.5mg vs. 0.25mg was

not planned (study was not powered for such a comparison) Copaxone® is a registered trademark of Teva Pharmaceuticals LTD

Gilenya® 0.5mg - significant 40.7% relative reduction in

ARR vs. Copaxone®

Gilenya® 0.25mg - numerical risk reduction (NS) in ARR

vs. Copaxone®

Safety consistent with the known safety profile

Copaxone® had more discontinuations due to AE and

unsatisfactory treatment effects

Gilenya® 0.5mg is the first therapy to show superiority in reducing relapses vs.

Copaxone® in a controlled head-to-head trial1,2

Aggregate ARR up to Month 12

N = 324 N = 366 N = 345

-40.7%p=0.0138

p=NS

Key drivers vs. PY:

+ Cash flows from operating activities

− Higher net intangible investments

9M 2018 free cash flow at USD 8.8bn


8.8

9M 2017 9M 2018

8.0

+10%

Group free cash flow USD billion

YTD 2018 net debt decreased by USD 1.9bn mainly

driven by net proceeds from M&A transactions


USD billion

-19.0-17.1

-7.0 -1.4-3.5

-8.3

Dec 31, 2017 Treasury share transactions, net

Dividends AAA1

Acquisition2

0.3

AveXis, Inc. Acquisition2

13.0

Proceeds from sale of GSK consumer

healthcare joint venture

8.8

Free CashFlow

Others Sep 30, 2018

+1.9

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

Q3 2018 Core EPS +6% cc, including the impact from

discontinuation of OTC JV core income


1.23

Q3 2017 Q3 2017Adjusted1

Q3 2018

1.29

1.32

+6%cc

Core EPSUSD, % cc

1 Q3 2017 core EPS adjusted to exclude core income recorded from OTC JV

Key drivers vs. PY:

+ Higher core operating income

+ Lower shares from share buyback

programs

− Discontinuation of OTC JV core income

from April 1st 2018

2018 pipeline milestones


H1 2018 H2 2018

Regulatory

decisions and

opinions

Kymriah® DLBCL (US) ✓ Aimovig™2 Migraine (EU) ✓

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

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

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

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

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

LA-EP20065 Peg-filgrastim BS (EU) =

GP1111 Infliximab BS (EU) ✓

GP20136 Rituximab BS (US) ✕

ACZ885 CV risk reduction (US)9 ✕

Submissions ACZ885 CV risk reduction (EU) ✓ BAF312 SPMS (EU) ✓

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

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

Cosentyx® AS (JP) ✓ AVXS-101 SMA7 (US) ✓

CTL019 Pediatric ALL + DLBCL (JP) ✓

Promacta® 1st line SAA (US/EU) ✓

Major trial

readouts

Kisqali® Advanced BC (MONALEESA-3) ✓4 LIK0668 Obesity ✓

LJN452 NASH (Interim Analysis) ✓ BYL719 HR+ BC ✓

INC280 ALK- cMET amplified NSCLC =

Entresto® HFpEF (Interim Analysis) ✓

✓ Achieved ✕ Missed = On track

1. Complete Response Letter received from FDA after Q4 2017 results; Advair® is a registered trademark of Glaxo Group Ltd. 2. Aimovig® is developed in collaboration with Amgen 3. Indication expansion based on MONALEESA-3 & 7 results

4. Data presented at ASCO; 5. Positive CHMP opinion received 6. Complete Response Letter (CRL) received from FDA after Q1 2018 results 7. i.v. formulation in type 1 SMA; submitted in EU October 2018 8. Program discontinued

9. CRL received from FDA October 2018

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. Non-alcoholic steatohepatitis

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

20. Acute myeloid leukemia

21. Chronic Obstructive Pulmonary Disease

22. Secondary Progressive Multiple Sclerosis

23. IV formulation Spinal Muscular Atrophy Type 1

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

25. IT formulation Spinal Muscular Atrophy Type 2/3

Planned filings 2018 to 2022

Combination abbreviations:

fulv fulvestrant

tmx tamoxifen

gsn goserelin

NSAI Non-steroidal aromatase inhibitor

Taf Tafinlar® (dabrafenib)

Mek Mekinist® (trametinib)

39Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation

ABL001CML4 3rd line

QGE031CSU/CIU16

2021

ACZ8851st Line NSCLC

ACZ8852nd Line NSCLC

Kymriah®r/r Follicular Lymphoma

Kymriah ®CLL22

Kymriah ®r/r DLBCL in 1st relapse

Entresto®

Heart failure (PEF)13

INC280 NSCLC6

Cosentyx®

nrAxSpA12

OMB157Relapsing multiple sclerosis

2019

SEG101Sickle cell disease

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

and others (same as originator)

PDR001 + Tafinlar®+Mekinist®

Metastatic BRAF V600+ melanoma

QVM149Asthma

QMF149Asthma

New molecule

New indication

New formulation

Biosimilars

LCI699Cushing’s disease

Lucentis®

ROP10

BYL719a + fulvHR+, HER2 (-) postmenopausal

adv. BC9 2nd line

RTH258nAMD7

2018

LAM320MDR8 tuberculosis

Lucentis®

Diabetic retinopathy

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

2022

BYM338Sarcopenia

VAY736Primary Sjoegren’s syndrome

Kisqali ®HR+, HER2 (-) BC9 (adjuvant)

Cosentyx®

AS H2H19

Rydapt®AML20 (FLT3 wild type)

INC280NSCLC6 (EGFRm)

Kymriah+ pembrolizumab - r/r DLBCL

ACZ885Adjuvant NSCLC

PDR001 comboMetastatic Melanoma

RTH258Retinal vein occlusion

CFZ533Sjorgen’s Syndrome

KAE609Malaria

EMA401Peripheral neuropathic pain

CNP520Alzheimer’s disease

BYM338Hip fracture recovery

KAF156 Malaria

ECF843d

Dry eye

CAD106Alzheimer’s disease

LHW090Resistant hypertension

HDM201Acute myeloid leukemia

CFZ533Solid Organ Transplant

CSJ117Severe Asthma

AVXS-201Rett Syndrome

LJN452Non-alcoholic steatohepatitis

QBW251COPD21

ZPL389Atopic dermatitis

UNR844Presbyopia

LMI070Spinal muscular atrophy

VAY785e

Non-alcoholic steatohepatitis

VAY736Autoimmune Hepatitis

ABL001CML4 1st line

MTV273Multiple myeloma

LOU064Chronic spontaneous urticaria

VPM087CRC 1L/RCC 1L24

LJC242Non-alcoholic steatohepatitis

a) US filing, submitted in EU

awaiting HA acceptance

Entresto®

Post-acute myocardial infarction

RTH258Diabetic macular edema

QAW039Asthma

2020

Jakavi®Acute GVHD14

Cosentyx®

PsA H2H17

Jakavi®Chronic GVHD14

XolairNasal Polyps

AVXS-101SMA Type 2/325

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

25. IT formulation SMA Type 2/3

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

40Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation

FTY720 Pediatric multiple sclerosis

Cosentyx®

nrAxSpA9

Entresto®

Heart failure (PEF)13

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

Kisqali®b + fulvHR+, HER2(-) postmenopausal

adv. or metastatic BC5 1st/2nd line

Entresto®

Post-acute myocardial infarction

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)

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.

Lucentis®

ROP16

QMF149Asthma

QVM149Asthma

OMB157 Relapsing multiple sclerosis

RTH258Diabetic macular edema

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

QAW039Asthma

RTH258nAMD6

LCI699Cushing’s disease

BYL719 + fulvHR+, HER2 (-) postmenopausal

adv. BC5 2nd line

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)

VAY785b

Non-alcoholic steatohepatitis

VAY736Autoimmune Hepatitis

Kymriah ®r/r Follicular Lymphoma

Kymriah ®CLL22

GP2013 (rituximab, US)Follicular lymphoma, DLBCL12 and


GP2017 (adalimumab) Arthritides, plaque psoriasis and others

(same as originator)

PDR001 comboMetastatic Melanoma

Kymriah ®r/r DLBCL in 1st relapse

PDR001 + Tafinlar®+Mekinist®

Metastatic BRAF V600+ melanoma

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


Kymriah ®+ pembrolizumab - r/r DLBCL

HDM201Acute myeloid leukemia

LOU064Chronic spontaneous urticaria

MTV273Multiple myeloma

CFZ533Solid Organ Transplant

ACZ885Sec. prev. CV events1

LTW888db

Retinopathy

New molecule

New indication

New formulation

Biosimilars

AVXS-101SMA Type 2/325

VPM087CRC 1L/RCC 1L24

CSJ117Severe Asthma

RTH258Retinal vein occlusion

Lucentis®

Diabetic retinopathyBAF312 SPMS20

CFZ533Sjorgen’s Syndrome

AVXS-201Rett Syndrome

LJC242Non-alcoholic steatohepatitis

AVXS-101SMA Type 123

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

Key changes vs. Q2 2018 presentation

Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation42

New additions

Trials taken out (operational decision-points achieved)

Study Program Indication Phase Patients

NCT03200717 IO-PAZ (CPZP034A2410) Votrient® Renal cell carcinoma Phase 2 100

NCT02354508 SWITCH (CSOM230C2413) Signifor® Acromegaly Phase 3 123

Study Program Indication Phase Patients

NCT03668613 (CAIN457A2311) Cosentyx® Psoriasis Phase 3 80

NCT03517540 TANDEM (CLJC242A2201J) LJC242 Non-alcoholic steatohepatitis Phase 2 200

NCT03437278 (CQGE031C2202) QGE031 Chronic spontaneous urticaria Phase 2 48

NCT03481634 KESTREL (CRTH258B2301) RTH258 Diabetic eye disease Phase 3 534

NCT03481660 KITE (CRTH258B2302) RTH258 Diabetic eye disease Phase 3 356

NCT03512197 (CPKC412E2301) Rydapt® Acute myeloid leukemia Phase 3 502

NCT03474965 (CSEG101B2201) SEG101 Prevention of VOC in pediatric patients with Sickle Cell Disease Phase 2 100

NCT03484923 (CPDR001J2201) Tafinlar®+Mekinist® Previously treated unresectable or metastatic melanoma Phase 2 135

Cardio-Metabolic

Entresto®

- Angiotensin receptor neprilysin inhibitor (ARNI)

44

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

Indication Heart failure in pediatric patients Heart failure

Phase Phase 2/3 Phase 4

Patients 360 1,002

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 Q4-2018

Publication TBD

• IA presented at ESC-2018;

• 10-wk data submitted EHJ (not yet accepted);

• 26-wk data presentation planned for ESC-HF May-2019

with goal of simultaneous publication if possible (not yet

accepted)


45

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

Indication Heart failure Heart failure, reduced ejection fraction

Phase Phase 3 Phase 3

Patients 520 225

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 2022H1-2019 (primary); 2020 (open-label extension)

Publication TBD TBD

Entresto®



46

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

Indication Heart failure Acute myocardial infarction

Phase Phase 3B/4 Phase 3

Patients 887 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 (actual) 2020

Publication

• Presentation planned AHA Nov-2018;

• Publication submitted to NEJM Q4-2018 (not yet

accepted)

TBD

Entresto®



47

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

Entresto®



Ilaris®

- Anti IL-1β

48

Study NCT01327846 CANTOS (CACZ885M2301)

Indication Cardiovascular risk reduction

Phase Phase 3

Patients 10,061

Primary Outcome

MeasuresTime 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 Jun-2017; Open-label extension continues till H1-2020

Publication

• Published cancer results in Lancet, Aug-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

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

• Chronic kidney disease data presented at ACC 11 Mar-2018; published in J Am Coll Cardiol, May 29, 2018 vol 71 no 21

• Published On-treatment IL-6 data in the European Heart Journal and ESC, Munich, Aug 26, 2018


Immunology, Hepatology

& Dermatology

Cosentyx®

- Anti IL-17

50

Study NCT01544595 (CAIN457A2302E1 – extension study)NCT01640951 SCULPTURE (CAIN457A2304E1 –

extension study)

Indication Psoriasis Psoriasis


Patients 1,146 675

Primary Outcome

Measures

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)

The number and percentage of subjects having any adverse

event

Arms/Intervention

• Secukinumab 150 mg


• 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 moderate to severe chronic plaque-type

psoriasis completing preceding psoriasis phase III studies

with secukinumab


psoriasis

Expected Completion 2017 (actual) 2017 (actual)

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

10.1111/bjd.15656

• 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)


Cosentyx®

- Anti IL-17

51

Study NCT02471144 (CAIN457A2310) NCT03066609 (CAIN457A2318)



Patients 169 543

Primary Outcome

Measures

The percentage of Participants achieving a 75%

Improvement from Baseline in PASI Score at week 12

Psoriasis Area and Severity Index (PASI) 75 response and

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

week 12

Arms/Intervention

• Secukinumab low dose

• Secukinumab high dose

• Placebo

• Etanercept (comparator)



• Placebo

Target PatientsPatients from 6 to less than 18 years of age with severe

chronic plaque


psoriasis with or without psoriatic arthritis comorbidity

Expected Completion 2023 Q1-2019

Publication TBD TBD


Cosentyx®

- Anti IL-17

52

Study NCT02826603 CLARITY (CAIN457A2326) NCT03668613 (CAIN457A2311)


Phase Phase 3B Phase 3

Patients 1,102 80

Primary Outcome

Measures



week 12



week 12

Arms/Intervention• Secukinumab 300 mg

• Ustekinumab 45 mg/ 90 mg

• Secukinumab low dose

• Scukinumab high dose

Target Patients Patients with moderate to severe plaque psoriasisPediatric patients of age 6 to <18 years, with moderate to

severe plaque psoriasis


Publication

• Abstract Winter Clin Derm (US) Jan-2018

• Abstract to EADV in 2018

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

(ongoing)

• Encore Abstract AAD 2019

TBD


Cosentyx®

- Anti IL-17

53

Study NCT02748863 ALLURE (CAIN457A2323) NCT02745080 EXCEED (CAIN457F2366)

Indication Psoriasis Psoriatic Arthritis


Patients 214 850

Primary Outcome

Measures


Investigators' Global Assessment (IGA) 0 or 1 responseAmerican College of Rheumatology 20 (ACR20) response

Arms/Intervention

• Secukinumab 300 mg (2 mL PFS device)

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

• Placebo

• Secukinumab 300 mg s.c.

• Adalimumab 40 mg s.c.

Target Patients Adult subjects with moderate to severe plaque psoriasis Patients with active psoriatic arthritis


Publication• Submission Journal TBC Q2-2019

• Abstract at AAD in 2019TBD


Cosentyx®

- Anti IL-17

54

Study NCT03031782 (CAIN457F2304) NCT01863732 (CAIN457F2305E1 – extension study)

Indication Psoriatic Arthritis Ankylosing spondylitis


Patients 80 300

Primary Outcome

MeasuresTime to flare in Part 2

Assessment of spondyloarthritis international society criteria

/ ASAS 20 response

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

• Placebo

• Secukinumab 75 mg in PFS

• Secukinumab 150 mg in PFS

Target PatientsJuvenile Idiopathic Arthritis subtypes of Psoriatic and

Enthesitis-related ArthritisPatients with active ankylosing spondylitis

Expected Completion 2021 Q2-2018 (actual)

Publication TBD

• 3-year results: Manuscript published in Clinical and

Experimental Rheumatology in May-2017

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


Cosentyx®

- Anti IL-17

55

Study NCT01892436 FUTURE 1 extension (CAIN457F2306E1) NCT01649375 MEASURE 2 (CAIN457F2310)

Indication Psoriatic arthritis Ankylosing spondylitis


Patients 460 219

Primary Outcome

Measures

Proportion of subjects that have a positive clinical response

to treatment (individual improvement) in disease activity

according to ACR20 (or ACR50 or ACR 70)

Assessment of SpondyloArthritis International Society /

ASAS 20 response

Arms/Intervention• Secukinumab 75 mg




• Placebo

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

Expected Completion Q1-2018 (actual) Q4-2018

Publication

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

Rheumatol. 2016; 68 (suppl 10)

• 3 years results: Manuscript submitted in Q4-2017

• 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


Cosentyx®

- Anti IL-17

56

Study NCT01752634 FUTURE 2 (CAIN457F2312) NCT02008916 MEASURE 3 (CAIN457F2314)



Patients 399 222

Primary Outcome

Measures

Proportion of subjects achieving American College of

Rheumatology 20 (ACR20) response criteria

Assessment of Spondyloarthritis International Society

criteria / ASAS 20 response

Arms/Intervention

• Secukinumab (AIN457) 150 mg s.c.



• Placebo s.c.

• Secukinumab 10 mg/kg / 300 mg

• Secukinumab 10 mg/kg / 150 mg

• Placebo


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

• 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


Cosentyx®

- Anti IL-17

57

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



Patients 416 350

Primary Outcome

Measures

American College of Rheumatology 20 (ACR20) response in

subjects treated with secukinumab vs. placebo

Assessment of Spondyloarthritis International Society

criteria / ASAS 20 at week 16

Arms/Intervention



• Placebo

• Secukinumab 150 mg s.c. with loading

• Secukinumab 150 mg s.c. without loading

• Placebo


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

Publication52 week results: Nash et al, Arthritis Research & Therapy

2018, 20:4752 week results: manuscript to be submitted in Q1-2018


Cosentyx®

- Anti IL-17

58

Study NCT02294227 FUTURE 4 (CAIN457F2336) NCT02404350 FUTURE 5 (CAIN457F2342)

Indication Psoriatic arthritis Psoriatic arthritis


Patients 342 990

Primary Outcome

Measures

Assessment of American College of Rheumatology 20

(ACR20)

American College of Rheumatology 20 (ACR20) response at

Week 16

Arms/Intervention

• Secukinumab 150 mg with loading

• Secukinumab 150 mg without loading

• Placebo

• Secukinumab 150 mg load

• Secukinumab 150 mg no load


• Placebo

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

Expected Completion Q1-2018 (actual) Q2-2019

Publication

• 52 week results: abstract to be presented at PANLAR

congress (Apr-2018)

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

• 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


Cosentyx®

- Anti IL-17

59

Study NCT02696031 PREVENT (CAIN457H2315) NCT03259074 SURPASS (CAIN457K2340)

Indication Non-radiographic Axial Spondyloarthritis Ankylosing spondylitis


Patients 555 837

Primary Outcome

Measures

The proportion of participants who achieved an ASAS 40

response (Assessment of SpondyloArthritis International

Society criteria);

No radiographic structural progression as measured by

modified Stoke Ankylosing Spondylitis Spine Score

(mSASSS)

Arms/Intervention


• Secukinumab 150 mg no load

• Placebo

• Secukinumab 150/300 mg

• adalimumab biosimilar 40 mg

Target Patients Patients with non-radiographic axial spondyloarthritis Patients with active ankylosing spondylitis

Expected Completion 2019 2022

Publication TBD TBD


Ilaris®

- Anti IL-1β

60

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)


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

61

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 2020

Publication TBD


LJC242 - FXR agonist + CCR2/CCR5 inhibitor

62

Study NCT03517540 TANDEM (CLJC242A2201J)

Indication Non-alcoholic steatohepatitis

Phase Phase 2

Patients 200

Primary Outcome

Measures

• Evaluation of safety and tolerability of combination

therapy (tropifexor + cenicriviroc) by monitoring adverse

event profile, vital signs and laboratory parameters

Arms/Intervention

• Tropifexor

• Cenicriviroc

• Tropifexor + Cenicriviroc

Target PatientsAdult patients with non-alcoholic steatohepatitis (NASH) and

liver fibrosis


Publication TBD


QGE031 - Anti-IgE

Study NCT02477332 (CQGE031C2201) NCT02649218 (CQGE031C2201E1)

Indication Chronic spontaneous urticaria Chronic spontanenous urticaria

Phase Phase 2B Phase 2B

Patients 382 226

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

Arms/Intervention

• Ligelizumab 24mg q4wks



• Ligelizumab 120mg single dose

• Omalizumab 300mg q4wks

• Placebo q 4wks

Ligelizumab 240 mg q4wks open label

Target Patients Patients with chronic spontaneous urticaria Patients with chronic spontaneous urticaria

Expected Completion 2017 (actual) H2-2019

PublicationPrimary results: Presentated at EAACI 2018; manuscript

expected in Q4-2018Primary results manuscript submission in Q4-2019


QGE031 - Anti-IgE

Study NCT03437278 (CQGE031C2202)

Indication Chronic spontaneous urticaria

Phase Phase 2

Patients 48

Primary Outcome

MeasuresChange in the 7 day Urticaria Activity Score (UAS7)

Arms/Intervention

• Ligelizumab 120 mg q4wks

• Ligelizumab 24 mg q4wks

• Placebo / ligelizumab 120 mg q4wks

Target PatientsAdolescents from 12 to <18 years of age, with chronic

spontaneous urticaria


Publication Manuscript to be submitted in 2021


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

Aimovig® – CGRP receptor antagonist

67

Study NCT03096834 LIBERTY (CAMG334A2301) NCT03333109 EMPOWER (CAMG334A2302)

Indication Migraine Migraine


Patients 246 880

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) Dose 1

• AMG334 (erenumab) Dose 2

• Placebo

Target PatientsAdult episodic migraine patients who have failed prophylactic

migraine treatmentsAdult episodic migraine patients

Expected Completion 2017 DBT phase (actual); 2021 OLE phase 2020

Publication Planned in 2019 (more details will follow) TBD


CNP520 - BACE inhibitor

CAD106 - active beta-amyloid immunotherapy

68

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

69

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

70

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 Q4-2018 Q4-2018

Publication TBD TBD


EMA401 - Angiotensin II type 2 receptor antagonist

71

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

72

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)

• Chitnis T, Arnold DL, Banwell B, et al. Trial of Fingolimod

versus Interferon Beta-1a in Pediatric Multiple Sclerosis..

N Engl J Med. 2018; 379: 1017-1027.

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

ECTRIMS 2017

• Primary manuscript: TBD


Gilenya®

- S1P-R modulator

73

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

Expected Completion Q3-2018 (actual)

Publication TBD


LMI070 - SMN2 RNA splice modulator

74

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

Expected Completion H2-2019

Publication TBD


OMB157 - Anti-CD20

75

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

76

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


Publication TBD


Oncology

ABL001 – Specific, allosteric Bcr-Abl kinase inhibitor

78

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

80

Study NCT02437318 SOLAR-1 (CBYL719C2301)

Indication HR + aBC

Phase Phase 3

Patients 572

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


PublicationSubmitted for ESMO 2018

Manuscript to be submitted in Q4-2018


Kymriah®

– CAR-T therapy

81

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

82

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


Publication Congress submission for Q4-2018


INC280 - MET Inhibitor

83

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


Publication• Congress presentation in Q4-2018

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


Jakavi®

- JAK1/2 inhibitor

84

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

85

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

86

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 Abstract submitted to congress for Q4-2018


PDR001 – PD-1 checkpoint inhibitor

87

Study NCT02967692 COMBI-i (CPDR001F2301)

Indication BRAFV600 mutant metastatic melanoma

Phase Phase 3

Patients

538

Part 1 (safety-run in): 9; Part 2 (biomarker cohort): 27; Part 3

(Phase III, randomized, placebo controlled): 532

Primary Outcome

MeasuresProgression-Free Survival (PFS)

Arms/Intervention

• Spartalizumab 400mg i.v. Q4W + Tafinlar 150mg BID +

Mekinist 2 mg

• Placebo + Tafinlar 150 mg BID + 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

88

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) 2020

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

TBD


Rydapt®- Multi-targeted kinase inhibitor

89

Study NCT03512197 (CPKC412E2301)

Indication Acute myeloid leukemia

Phase Phase 3

Patients 502

Primary Outcome

MeasuresEvent free survival

Arms/Intervention• Midostaurin 50 mg

• Placebo

Target PatientsNewly diagnosed patients with FLT3 mutation negative

acute myeloid leukemia (AML)


Publication TBD


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 12.5, 25, 50, 75 mg FCT & 25 mg pFOS

- 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) NCT03474965 (CSEG101B2201)

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

Sickle Cell Disease (SCD) Prevention of VOC in pediatric patients with SCD


Patients 55 100

Primary Outcome

MeasuresPK/PD and safety of SEG101 (crizanlizumab) at 5 mg/kg PK/PD and safety of SEG101 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

SEG101 (crizanlizumab) at a dose of 5 mg/kg by IV infusion

± Hydroxyurea/Hydroxycarbamide

Target Patients Adult SCD patients with VOC Pediatric SCD patients with VOC

Expected Completion Q4-2018 2020

Publication Planned abstract submission in 2019 TBD


Tafinlar®+Mekinist

®- BRAFV600 inhibitor and MEK inhibitor

Study NCT01682083 COMBI-AD (CDRB436F2301) NCT02124772 (CTMT212X2101)

Indication BRAFV600 mutant adjuvant melanoma BRAFV600 mutant solid tumors

Phase Phase 3A Phase 1

Patients 874 142

Primary Outcome

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

Arms/Intervention• Dabrafenib 150 mg BID + 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/InterventionSingle-arm study of oral dabrafenib (dose based on age

and weight)

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

BRAF V600-mutation positive solid tumors


Publication TBD

Tafinlar®

- BRAF inhibitor


Tafinlar®+Mekinist

®- BRAF inhibitor and MEK 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 BID + 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 Q4-2018


Tafinlar®+Mekinist


Study NCT02684058 (CDRB436G2201)

Indication BRAFV600 mutant gliomas

Phase Phase 2

Patients 142

Primary Outcome

MeasuresObjective response rate

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


Tafinlar®+Mekinist


Study NCT03484923 (CPDR001J2201)

Indication Previously treated unresectable or metastatic melanoma

Phase Phase 2

Patients 135

Primary Outcome

MeasuresObjective Response Rate (ORR)

Arms/Intervention

• PDR001 400mg i.v. Q4W + LAG525 600 mg i.v. Q4W

• PDR001 400mg i.v. Q4W + canakinumab 300 mg (s.c)

Q4W

• PDR001 400mg i.v. Q4W + capmatinib 400 mg BID

orally

Target PatientsAdult patients with previously treated unresectable or

metastatic melanoma


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

100

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.


Publication

• EURETINA: Sep-2018

• AAO: Oct-2018

• Primary manuscript: planned submission by end of 2018

TBD


RTH258 - Anti-VEGF

Study NCT02434328 HARRIER (CRTH258A2302) NCT02307682 HAWK (CRTH258A2301)

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


Patients 743 1,082

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) NCT03481634 KESTREL (CRTH258B2301)

Indication Neovascular age-related macular degeneration (nAMD) Diabetic eye disease


Patients 150 534

Primary Outcome

MeasuresNumber of treatment-emergent adverse events

Change from baseline in best-corrected visual acuity

(BCVA)

Arms/Intervention• RTH258 6 mg

• Aflibercept 2 mg

• Brolucizumab 3 mg

• Brolucizumab 6 mg


Target PatientsPatients with neovascular age-related macular degeneration

who have completed the CRTH258A2301 study

Patients with visual impairment due to diabetic macular

edema (DME)

Expected Completion Q4-2018 2021

Publication TBD TBD


RTH258 - Anti-VEGF

Study NCT03481660 KITE (CRTH258B2302)

Indication Diabetic eye disease

Phase Phase 3

Patients 356

Primary Outcome

Measures

Change from baseline in best-corrected visual acuity

(BCVA)

Arms/Intervention• Brolucizumab 6 mg


Target PatientsPatients with visual impairment due to diabetic macular

edema (DME)


Publication TBD


Respiratory

QAW039 – DP2 (CRTh2) antagonist

105

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

Expected Completion 2019 (for submission); 2022 (final)

Publication TBD


QMF149 - Long-acting beta2 agonist and inhaled corticosteroid

108

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 Patients Children ≥ 6 to < 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 od

• Salmeterol 50 µg /fluticasone 500 µg bid

Target Patients

Adult and adolescent (>12 years) 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



110

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


Publication TBD TBD



Study NCT02892344 QUARTZ (CQVM149B2303) NCT03158311 ARGON (CQVM149B2306)



Patients 802 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 (>12 years) patients with in poorly

(i.e., inadequately) controlled asthmaPatients with uncontrolled asthma

Expected Completion Q1-2019 H2-2019

Publication TBD TBD



Study NCT03137784 SILVER (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

113

Study NCT03369704 (CIGE025F1301)

Indication Seasonal allergic rhinitis: Severe Japanese Cedar Pollinosis

Phase Phase 3

Patients 337

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

Expected Completion Q1-2019

Publication

Nov 2018: Late breaking abstract for the American

Association of Allergy, Asthma and Immunology (AAAAI)

annual meeting in Feb 2019


Sandoz

Biopharmaceuticals

Erelzi®

- Biosimilar etanercept

115

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 RMD Open (final submission for

publication on 17-Sep-2018)

• 48 week: Manuscript in Q4-2018 (journal Arthritis Research

&Therapy)


Rixathon®

- Biosimilar rituximab

116

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

• Tony, H-P et al, Arthritis Care & Research, 2018

(accepted for publication)

• 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

117

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) Q3-2018 (actual)

Publication

• 51 week data and switch data, Blauvelt et al. BJD, 2018,

https://doi.org/10.1111/bjd.16890

• Blauvelt et al. presented at ACR 2017, 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, oral presentation at ACR 2018

• Abstract and poster at EULAR 2019

• Manuscript with study results journal TBD


Global Health*

*Previously classified as ‘Established Medicines and Anti-infectives’

Tobramycin – An aminoglycoside antibiotic

119

Study NCT02712983 iBEST-1 (CTBM100G2202)

Indication Bronchiectasis

Phase Phase 2

Patients 105

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

120

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

121

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: Prostate specific antigen

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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