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Q4 and FY 2018 ResultsInvestor Presentation

January 30, 2019

Novartis AG

Investor Relations

Disclaimer

Novartis Q4 and FY 2018 Results | January 30, 2019 | 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 outcome, or financial or other impact on Novartis, of

the proposed spin-off of our Alcon Division, or of the proposed divestiture of certain portions of our Sandoz Division business in the US; or regarding the potential impact of the share

buyback plan; or regarding potential future sales or earnings of the Group or any of its divisions or potential shareholder returns; or regarding potential future credit ratings of the

Group; or by discussions of strategy, plans, expectations or intentions. Such forward-looking statements are based on the current beliefs and expectations of management 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. You should not place undue reliance on these statements. In

particular, our expectations could be affected by, among other things: global trends toward healthcare cost containment, including ongoing government, payer 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 proposed transactions or the development of the products described in this Annual Report; the potential that the strategic benefits,

synergies or opportunities expected from the proposed transactions 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 that 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 litigation with respect to the proposed transactions, 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; our performance on environmental, social and governance measures; general political, economic and trade 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; 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.

Vas NarasimhanCompany overview

Harry KirschFinancial review

Paul HudsonPharma highlights

Susanne SchaffertOncology highlights

Agenda

Vas NarasimhanChief Executive Officer

Company overview

We aim to become a leading medicines company Powered by advanced therapy platforms and data science


Go big on data and digital

Passionate about

productivity and margins...

Embrace operational excellence

Build trust with society

While building a new culture

and lasting impact

Unleash the power of our people

Transform Sandoz

We are focusing

the company

Spin Alcon

Building advanced therapy platforms

Driving growth through

cutting-edge platforms...

Leading pipeline

We delivered strong growth with operating leverage in Q4 and FY 2018


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 53 of the Condensed Financial Report

Q4 (% cc) Full year (% cc)

Sales Core OpInc Sales Core OpInc

Group1 +6% +11% +5% +8%

Innovative Medicines +9% +13% +8% +11%

Sandoz -2% -5% -3% -3%

Alcon +4% 0% +5% +10%




Passionate about





and lasting impact


Transform Sandoz

We are focusing

the company

Spin Alcon




Leading pipeline

We took major steps to focus the company in 2018, while building leading advanced therapy platforms


All trademarks are the property of their respective owners 1. Announced or closed in 2018 2. The planned 100% spin-off of Alcon remains subject to certain conditions precedent, such as no material adverse events, receipt of necessary

authorizations as well as tax rulings and opinions and shareholder approval at the AGM in February 2019; completion expected in H1 2019 3. The announced sale of Sandoz US dermatology and oral solids portfolio to Aurobindo is subject to

the completion of customary closing conditions expected in 2019 4. Sale of our anti-bacterial portfolio to Boston Pharmaceuticals is one example of portfolio prioritization. Others include out-licensing of BJG398 to QED Therapeutics, FGF401

to EverNov, LXS196 to IDEAYA.

Deals1 to build new platformsExits1 to focus the company

Gene

therapy

Cell

therapy

Radioligand

therapy

2

3

4

http://seekvectorlogo.com/aurobindo-pharma-vector-logo-ai/

Alcon delivered margin expansion and consistent growth; on track for spin-off H1 20191


2016 2017 2018

17.6%17.3%

17.9%

1. The planned 100% spin-off of Alcon remains subject to certain conditions precedent, such as no material adverse events, receipt of necessary authorizations as well as tax rulings and opinions and shareholder approval at the AGM in February

2019; completion expected in H1 2019 2. Alcon Division 2016 and 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;

figures above represent Alcon results as a division of Novartis and as such do not include costs associated with establishing Alcon as a separate company, which are expected to be approximately 1% of sales

Strong 2018 results

Sales growth driven by innovation; ATIOLs, Systane®

Complete and Dailies Total1® Multifocal

Margin expansion driven by higher sales and

improved gross margin

Capital markets days held Q4 2018; further investor

engagement planned Q1 2019

Shareholder vote on spin-off February 28 at AGM1

Share ratio of 5 Novartis shares: 1 Alcon share

On track for spin-off Q21; see shareholder brochure:https://www.novartis.com/sites/www.novartis.com/files/2019-novartis-agm-alcon-en.pdf

Alcon core

margin2

% of USD sales

2016 20182017

-1%

4%5%Alcon full

year sales

growth

cc

https://www.novartis.com/sites/www.novartis.com/files/2019-novartis-agm-alcon-en.pdf

Our Sandoz transformation will help enable us to compete in a more challenging environment


Reshaping the portfolio... ...while driving efficiency

Geographic focusLeading in biosimilars: 8 marketed products, more to come Lean cost structure

SKU

rationalization

Manufacturing

footprint

optimization

Regional

consolidation

All trademarks are the property of their respective owners 1. The announced sale of Sandoz US dermatology and oral solids portfolio to Aurobindo is subject to the completion of customary closing conditions expected in 2019

1

http://seekvectorlogo.com/aurobindo-pharma-vector-logo-ai/

1. Investments in organic business

2. Growing annual dividend in CHF

3. Value-creating bolt-ons2

4. Share buybacks

We remain disciplined and shareholder-focused in our capital allocation


100% tax-neutral spin1 of Alcon to shareholders

to enable focus on core medicines business

Proposing 22nd consecutive dividend increase3

~USD 15bn M&A bolt-ons in 2018

100+ collaboration and licensing deals completed

~USD 17bn of net share buybacks over past 5 years,

including purchases under new USD 5bn program4

Novartis

priorities

1. The planned 100% spin-off of Alcon remains subject to certain conditions precedent, such as no material adverse events, receipt of necessary authorizations as well as tax rulings and opinions and shareholder approval at the AGM in

February 2019; completion expected in H1 2019 2. Includes M&A and BD&L 3. Proposed dividend increase for 2018 at AGM 2019 4. Share buyback of up to USD 5bn announced on June 29, 2018




Passionate about





and lasting impact


Transform Sandoz

We are focusing

the company

Spin Alcon




Leading pipeline

Our in-line growth brands provide a solid foundation for continued growth

13 Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation

FY Sales USD million

vs. PYcc

Growth vs. PYUSD million

766

200

167

521

307

282

119

159

70

2,837

1,028

1,174

1,155

977

167

235

1,039

76

36%

102%

35%

31%

24%

nm1

nm1

12%

nm1

1 Not meaningful

4 additional blockbusters in 2018

Lutathera®

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

With 10+ potential blockbuster1 launches planned in the next 2 years


1. Individual assets with expected peak sales >USD 1bn across all indications 2. The brand name Mayzent™ has been provisionally approved by the FDA and EMA for the investigational product siponimod (BAF312) , but the product itself has

not been approved for sale in any country 3. The brand name Zolgensma™ has been provisionally approved by the FDA for the investigational product AVXS-101 (onasemnogene abeparvovec-xxxx), but the product itself has not received

marketing authorization or BLA approval from any regulatory authorities

2020

INC280 NSCLC

OMB157Relapsing MS

PDR001 comboMetastatic Melanoma

QVM149Asthma

SEG101Sickle Cell Disease

Entresto®

HFpEF

Cosentyx®

nrAxSpA

2018

Lutathera®

NET

Kymriah®

DLBCL

Aimovig®

Migraine

2019

RTH258nAMD

Zolgensma™3

SMA Type 1

BYL719Advanced breast cancer

Mayzent™2

SPMS

Launched 2018

3

4

7

Planned launches

2019 expected catalysts to continue the momentum


1. The brand name Zolgensma™ has been provisionally approved by the FDA for the investigational product AVXS-101 (onasemnogene abeparvovec-xxxx), but the product itself has not received marketing authorization or BLA approval from

any regulatory authorities 2. The brand name Mayzent™ has been provisionally approved by the FDA and EMA for the investigational product siponimod (BAF312), but the product itself has not been approved for sale in any country

Catalysts Selected examples

Key

approvals15 Zolgensma™1

SMA Type 1 (US/EU/JP)

Mayzent™2

SPMS (US/EU/JP)

Brolucizumab (RTH258)Neovascular AMD (US)

Alpelisib (BYL719)Breast Cancer (US)

Major

submissions20 Ofatumumab (OMB157)

Relapsing MS (US/EU)

Crizanlizumab (SEG101)Sickle Cell Disease (US/EU)

Brolucizumab (RTH258)Neovascular AMD (US/EU/JP)

INC280NSCLC (US/JP)

PDR001 comboMetastatic Melanoma (US/EU)

Major

late-stage

readouts

6 Zolgensma™1

SMA Type 2

Fevipiprant (QAW039)Asthma

Entresto®

HFpEF

Cosentyx®

nrAxSpA

Ofatumumab (OMB157)Relapsing MS


Advanced therapy platforms with the potential to expand the game-board


Small

molecules

Large

molecules

Cell

therapy

Gene

therapy

Radioligand

therapy

Oncology

Cardio-Metabolic

IHD

Neuroscience

Ophthalmology

Respiratory

(Illustrative)

1. Partnership with the Wyss Institute for Biologically Inspired Engineering at Harvard University and the Dana-Farber Cancer Institute 2. Collaboration with Xencor 3. Collaborations with Intellia Therapeutics and Caribou Biosciences

4. Proposed acquisition; transaction subject to the completion of customary closing conditions 5. Collaboration with Cellular Biomedicine Group in China

AAA EndocyteCAR-TCBMG5

Cell for

Cure4

Intellia &

Caribou3

Bispecific

antibodies2Novel

bio-materials1

Transcription

factors

Targeted

protein

degradation

Inhaled

biologics

Novel

bio-materials

AveXis

NIBR

Portfolio

Luxturna®

7 programs in clinic over next yearManufacturing expansion ongoing for AAV-based

gene therapies

Gene therapy platform with rapidly expanding pipeline, deep manufacturing expertise in AAV


Flexible, disposable

manufacturing platform

Operational facility in

Chicago; ongoing build-

out in North Carolina

Capabilities across AAV

vectors; deep CMC

expertise

Selected assets Indication Stage Next milestone

AVXS-101 (AAV9) SMA Filed Regulatory approval(s) 1H19

CGF166 (Ad5) Hearing loss Phase 1

CPK850 (AAV8) Retinitis pigmentosa Phase 1

AVXS-201 RTT (AAV9) Rett Syndrome Preclinical IND 1Q19

AVXS-301 SOD1 (AAV9) InheritedALS-SOD1 Preclinical IND 2Q19

AVXS-401 Undisclosed Preclinical IND 2H19

AVXS-501 Undisclosed Preclinical IND 4Q19 / 1Q20




Passionate about





and lasting impact


Transform Sandoz

We are focusing

the company

Spin Alcon




Leading pipeline

We are committed to driving consistent margin expansion


1. Source: Novartis analysis of average 2016 core margin of Large Pharma peer companies

+ Acceleration of key growth drivers

+ Resource allocation and productivity

programs in commercial units

+ Cross-divisional synergies: Novartis

Technical Operations, Novartis Business

Services, Procurement

− Generics (mainly Afinitor®, Exjade®, Gilenya®)

− Launch investments for potential future

blockbustersIM Division

2017

IM Division

2018

Large Pharma

average1

3132

Mid 30s

Innovative MedicinesCore margin (%)

With an aggressive productivity agenda


Initiative Goal Progress

NTONovartis Technical

Operations ~USD 2bn

savings by 2020

primarily driven by

these functions1;

increased focus on

cash conversion cycle

Announced 16 plant transformations in 2018, including 8 exits

Announced significant restructuring in Switzerland

Developing predictive capabilities to reduce inventory, increase efficiency & automation

NBSNovartis Business

Services

Driving transformation through standardization, simplification & footprint optimization

Announced global restructuring plans

Launched effort to automate high-impact global processes

ProcurementCross-Divisional

Savings targets in all units across major spend categories

Recruited new Head of Procurement from low-margin industry

1. Includes USD 1bn of savings from the NTO network transformation announced in January 2016.

Advancing an enterprise-wide digital transformation


We’re reimagining medicine with data and digitalOur digital lighthouse projects are kick-starting our enterprise-wide transformation but we need the

commitment of all 125,000 of us to make it a success. Let’s #GoDigital!

Click on the hotspots below for more information

Mining clinical trial

data for new insights

DATA42

Bringing clinical trials

to the patient

Patient-centric remote

trials

Combining medicines

with cutting-edge

technology

Digital therapeutics

Transforming Pharma

commercial models

Pharma ACT

Data repository

for Oncology

DROID

End-to-end tender

optimization

AI tender

Innovative digital

solutions for customers

Around/beyond the pill

End-to-end

process automation

NBS 2.0

Building digital

capabilities

HR

More ef cient

manufacturing

NTO 2.0

Launch excellence

along the patient funnel

Meeting patients and

HCPs along the journey

innovateStrengthening our

innovation core

operateWorking smarter to deliver greater

value

engageMaximizing the return

of our commercial operations

Digital community Tech enablers Data & advanced analytics External partners Digital awareness hub

More efficient

manufacturing

Data & advanced analytics

All trademarks are the property of their respective owners




Passionate about





and lasting impact


Transform Sandoz

We are focusing

the company

Spin Alcon




Leading pipeline

Culture transformation is key to our successInitiated 5-year journey in 2018


Reshaped to a more diverse Executive Committee (over 50% new members)

Rolled out new culture vision to create an empowered organization

100% of top leaders going through leadership “academy”; 100% of managers

to receive digital upward feedback on an ongoing basis

Digitally enabled tools

Inspired

Curious

Unbossed

Focused effort to build lasting trust with society


Established Ethics, Risk &

Compliance function, led by

Executive Committee member

Embedding principles-based

decision-making in the

organization

Ethical

Standards

Integrating Access

Principles into overall

business strategy

Improved ranking in Access

to Medicines Index to #2

Pricing and

Access

Renewed commitment to

malaria with USD 100m

investment

Established Global

Partnership for Zero Leprosy

Global Health

Challenges

Approved new environmental

sustainability targets, incl.

carbon neutrality by 2025

Helped lead Pat-INFORMED

initiative, making patents

available online

Corporate

Citizenship

Continued to improve

transparency and evolve

reporting

Increased reporting on

Financial, Environmental and

Social (FES) impact on society

Stakeholder

Engagement

Well-positioned for top and bottom-line growth


In-market growth drivers

Cosentyx®, Entresto®, Kisqali®, Lutathera®, Kymriah®, Promacta®, Tafinlar®+Mekinist®, Jakavi®, Biosimilars

15 in-market

blockbusters

as of YE 2018

10+ potential

blockbusters expected

to launch by 2020

Savings of ~USD 2bn

expected by 20201

Patent expiries

primarily in near term

Key launches

Zolgensma™, Mayzent™, RTH258, Aimovig®, OMB157, BYL719, SEG101, Biosimilars

Productivity improvements

NTO transformation, NBS transformation, Procurement initiative, Digital initiatives

Generic erosion

Exjade®

+

+

+

–

2019 2020 2021

Gilenya® (timing TBC)2Afinitor®

NTO – Novartis Technical Operations NBS – Novartis Business Services The brand name Zolgensma™ has been provisionally approved by the FDA for the investigational product AVXS-101 (onasemnogene abeparvovec-xxxx), but the product

itself has not received marketing authorization or BLA approval from any regulatory authorities. The brand name Mayzent™ has been provisionally approved by the FDA and EMA for the investigational product siponimod (BAF312), but the product

itself has not been approved for sale in any country. 1. Includes USD 1bn of savings from the NTO network transformation announced in January 2016 2. Gilenya US compound patent expiration August 2019; dosing regimen patent expiration

December 2027

(Illustrative)

Harry KirschChief Financial Officer

Financial review

2018 financial results in line with guidance

FY 2018

vs. PYIn cc

Group full year guidance (January 2018)In cc

“Sales are expected to grow low to mid single digit” 5%

8%“Core operating income expected to grow mid to high

single digit”

27

Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation

% USD % cc % USD % cc

Net Sales 13,269 3 6 51,900 6 5

Core Operating income 3,387 5 11 13,823 8 8

Operating income 1,299 -37 -29 8,169 -5 -5

Net Income 1,194 -40 -32 12,614 64 64

Core EPS (USD) 1.25 3 9 5.15 6 6

EPS (USD) 0.52 -39 -32 5.44 66 66

Free Cash Flow 2,939 20 11,717 12

Change vs. PYGroup

1

USD million

Q4

2018

FY

2018

Change vs. PY

Summary of Q4 and FY 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 53 of the Condensed Financial Report

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

(in % cc) (in % cc) (%) (%pts cc) (%) (%pts cc)

Innovative Medicines 9 13 30.7 1.1 32.0 1.0

Sandoz -2 -5 19.6 -0.6 20.3 -0.1

Alcon 4 0 15.7 -0.7 17.9 0.8

Group 6 11 25.5 1.2 26.6 0.7

Q4 2018 FY 2018

Innovative Medicines driving Q4 margin expansion; FY margin increase driven by IM Division and Alcon


2018 Key drivers vs. PY:

+ Cash flows from operating activities, mainly:

+ Higher core operating income

+ GSK milestone receipt (divested Vaccines business)

− Higher net intangible investments

FY 2018 free cash flow at USD 11.7bn


FY 2016 FY 2017 FY 2018

10.49.5

11.7

+12%

Group free cash flow USD billion

Novartis proposes 22nd consecutive dividend increase to the AGM: 2.85 CHF / share1


1. Proposal to shareholders at the 2019 Annual General Meeting, taking place on February 28, 2019 2. Converted at historic exchange rates at the dividend payment dates as per Bloomberg; assumes an exchange rate of USD / CHF of

0.9862 as of December 31, 2018 for 2018

0.0

0.5

1.0

1.5

2.0

2.5

3.0

20101996 1997 1998 1999 200220012000 2003 2004 2005 2006 20092007 2008 2011 2012 2013 2014 2015 2016 2017 2018

CH

F 2

.80

US

D 2

.94

CH

F 2

.85

US

D 2

.89

CHF USD

2019 Novartis full year guidanceBarring unforeseen events (in cc); Growth vs PY in cc


Current Group structureNo change to current Group Structure

New focused medicines companyExcl. Alcon1 & Sandoz proposed US portfolio sale to

Aurobindo2 from both 2018 and 2019

1. The planned 100% spinoff of Alcon remains subject to certain conditions precedent, such as no material adverse events, receipt of necessary authorizations as well as tax rulings and opinions and shareholder approval at the AGM in February

2019; completion expected in H1 2019 2. The announced sale of Sandoz US dermatology and oral solids portfolio to Aurobindo, expected to close during 2019, is subject to the completion of customary closing conditions. Estimated 2018 FY

Sales and Core OpInc of the Sandoz US Oral solids and Dermatology businesses were approximately USD 1.2bn and 0.3bn, respectively.

Key Assumption: All guidance includes forecast assumption that no Gilenya® generics enter in 2019. However, generic competitors may still launch at risk

Net Sales Expected to grow low to mid single digit

IM Division to grow mid single digit

Sandoz to decline low single digit

Alcon to grow low to mid single digit

Expected to grow mid single digit

IM Division to grow mid single digit

Sandoz to be broadly in line with prior year

Core Operating

Income

Expected to grow mid single digit

Alcon Core OpInc margin expected to expand

Expected to grow mid to high single digit

FY 2019 Guidance on other financial KPIsBarring unforeseen events (in cc)


New focused medicines companyExcl. Alcon1 & Sandoz proposed US portfolio sale to Aurobindo2 from both 2018 and 2019

Core

net financial

expense

FY expected to be broadly in line vs. 2018 current Group structure

Core

tax rateFY core tax rate expected to increase slightly to around 16%

1. The planned 100% spinoff of Alcon remains subject to certain conditions precedent, such as no material adverse events, receipt of necessary authorizations as well as tax rulings and opinions and shareholder approval at the AGM in February

2019; completion expected in H1 2019 2. The announced sale of Sandoz US dermatology and oral solids portfolio to Aurobindo, expected to close during 2019, is subject to the completion of customary closing conditions

Expected currency impact for full year 2019


Currency impact vs. PY%pts, assuming late-January exchange rates prevail in 2019

SimulationActual

FX impact on

Net sales

FX impact on

Core operating income

-3

1

-5-2

-6 -7-3

FYQ4 FY Q4Q1 FY Q1 FY

0

20182018 2019 2019

Pharma highlights

Paul HudsonCEO Novartis Pharmaceuticals

Cosentyx®: Strong growth driven by demand, well positioned across indications


Q4 sales USD 806m (+33% cc) with consistent growth

US (+34% cc) and ex-US (+32% cc)

- Continued demand growth, US YoY TRx +29% Dermatology,

+49% in Rheumatology1

- Gaining share in a growing and competitive psoriasis market

2019: expected to maintain strong access in US

Continue to advance science in psoriatic disease

- ARROW (readout expected end of 2019) expected to confirm

importance of IL-17A vs. IL-23

- Cosentyx® has demonstrated efficacy in the multiple

manifestations of psoriatic disease

PREVENT (nrAxSpA) read-out and submission

expected end 2019

Quarterly sales evolutionUSD million

Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4

0

20

40

60

80

100

120

2.1bn

410490

556615

2017 2018

580

701 750

2.8bnEx-USUS

806

US TRx1 (quarterly in 000s)

1. IMS NPA TRx, Cosentyx® restated in Aug 2017 to include free product, Q4 estimated with WE 12/21/2018 data

Entresto® achieves blockbuster status – reinforcing strong therapy position in heart failure1


Early indicators of accelerated hospital initiation (PIONEER & TRANSITION)

Significantly reduces NT-proBNP in stabilized ADHF patients (PIONEER)

Strong sales growth driven by execution & new data PIONEER data2 supports early Entresto® useComposite of Death, HF re-hospitalization, LVAD, Listing for Transplant

USD 318m (+76% cc) Q4 sales

Blockbuster in 2018 and doubling sales vs. 2017

ADHF – Acute Decompensated Heart Failure FIR – First Interpretable Results LVAD – Left Ventricular Assist Device 1. Entresto® is approved in HFrEF and ongoing HFpEF trial expected to read out 2019. 2. Published in New England

Journal of Medicine, Nov 2018: DOI: 10.1056/NEJMoa1812851

Global sales, USD million

84 110 128 185 200

239 271

318

Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4

ex-US US

2017 2018

507m

1,028m

Expected newsflow 2019 FIR: PARALLEL-HF (Japan registration trial HFrEF) Q2 2019; PARAGON-HF in HFpEF Q3 2019

46% reduction,

driven by a 44%

reduction in HF

hospitalization

SPMS SMA Type 1 nAMD

EXPAND demonstrates

unprecedented efficacy in SPMS

Targeting MS patients when

progression becomes noticeable,

regardless of relapses

FDA action date expected H1

CHMP opinion expected H2

Launch expected in both

geographies in 2019

2019: Preparing to launch next wave of growth drivers


Mayzent™1 (BAF312)

Rapid improvement in milestone achievement,

previously unseen survival benefit, and durable

effect

ICER draft report affirms value of Zolgensma™ up

to USD 4 to 5m at the appropriate ultra-rare

disease threshold3

Label discussion on-going with FDA; US and Japan

approvals expected H1 2019, and Europe mid-2019

SMA Type 2 STRONG data readout planned at

AAN 2019

Zolgensma™2 (AVXS-101)

Brolucizumab has the potential to

address important unmet needs in

nAMD – dries better

HAWK and HARRIER year-2 data

reaffirm superiority in key secondary

endpoints from year one versus

aflibercept in patients with nAMD

– Less retinal fluid4

– Fewer injections5

– Uncompromised vision6

On track for launch end 2019

RTH258 (brolucizumab)

See slide 58 for references

Oncology highlights

Susanne SchaffertCEO Novartis Oncology

Oncology grew +9% (cc) FY 2018, behind continued strong performance of key growth drivers


Promacta®/Revolade®

USD m, % cc

867

1,174

FY 2018FY 2017

+35%

Tafinlar® + Mekinist®

873

1,155

FY 2017 FY 2018

+31%

Thrombopoietin receptor agonist with

the broadest indication

Strong SAA growth in Japan and 1L

SAA approval in US

Market leader in BRAF+ targeted therapy

Approved for adjuvant treatment of

melanoma across major geographies

including US & EU

50 – 75% of eligible myelofibrosis patients

treated with Jakavi®

Double digit growth for both myelofibrosis

and polycythemia vera indications

Jakavi®

777

977

FY 2017 FY 2018

+24%

Novartis building leadership in radioligand therapy with highly-complex, scaled, on-demand manufacturing capability


Lutathera® strong uptake continues

Strong launch momentum, Q4 sales USD 81m

In 2018 US: >1,400 new patients; >100 centers actively prescribing

Broad US payer coverage with >80% of lives covered

Following UK reimbursement in Q3, 19 centers actively prescribing

52

399

1,123

1,726

Q3 ’18Q1 ’18 Q2 ’18 Q4 ’18

US, number of doses

2018 FY sales:

USD 167m

HospitalIsotope supply Production

177Lu Isotope

Produced in nuclear reactors

Precursor Isotope176Lu

Target Isotope177Lu

Octreotide

DOTATATE

Lutathera®

labeling

DOTATATE

Aseptic vial filling

Lutathera®

Aseptic conditions

On demand production

Patient

delivery

Patient dose

Standard dose of 7.4GBq/cycle

Aseptic conditions

Requires unique manufacturing centers, supply chain

expertise, and relationships with nuclear medicine centers

Radioligand therapy platform with growing pipeline in solid tumors


Product Disease (target) Status

177Lu

PSMA-617

Prostate cancer

(PSMA)Ph3 VISION study

initiated 2Q 2018

177Lu

PSMA-R2 Ph1 / 2 study initiated 2Q 2018

68Ga

PSMA-R2Ph1 / 2 study initiated 2Q 2018

18F

CTT1057Ph1 study completed

177Lu

NeoBOMB1

Breast cancer

Colorectal cancer

Lung cancer

Prostate cancer

GIST (GRPR)

Ph1 study to open 1H 2019

68Ga

NeoBOMB1Ongoing Ph1 ISS in GIST

Ph2 study initiated 2Q 2018

177Lu

FF-10158

Glioblastoma

(Integrin

Alphavbeta 3/5)

Preclinical

68Ga

FF-10158 Preclinical

Preclinical Phase I Phase II Phase III Filing Marketed

Therapeutic

PET Diag.

PET Diagnostic

Therapeutic

PET Diagnostic

Therapeutic

PET Diag.

Therapeutic

Expands Novartis nuclear medicine platform

– 177Lu-PSMA-617 potentially first-in-class PSMA radioligand

therapy following successful launch of Lutathera®

– Opportunity to further develop 177Lu-PSMA-617 to enter

earlier lines of therapy

Ph3 VISION trial enrollment initiated for 177Lu-PSMA-617 in mCRPC

– FDA agreed to rPFS as an alternative primary endpoint to OS

– Expected rPFS read-out and filing in 2020

Endocyte further establishes leadership positionRadioligand therapy being explored across full range of solid

tumors

rPFS – radiographic progression free survival

Pipeline of 10 clinical programs and 4 FIH, with large

pre-clinical effort

CAR-T type Indication Phase 1 Ph 2/Pivotal Phase 3 Submitted Approved

CD19 CAR-T Pediatric & young adult r/r ALL

CD19 CAR-T r/r DLBCL

CD19 CAR-T DLBCL in 1st relapse

CD19 CAR-T r/r FL

CD19 CAR-Tr/r DLBCL in combination with

pembrolizumab

CD19 CAR-T Adult r/r ALL

CD19 CAR-T r/r CLL combination with ibrutinib

CD19 CAR-T Pediatric NHL

CD19 CAR-T1st L high risk pediatric and young

adult ALL

CD19 CAR-T r/r DLBCL combo with ibrutinib

Other targets

(UPenn partner)

BCMA&CD19, CD22&CD19,

CD123, EGFRv3

US, EU

US, EU

Starting 2019

Started 2018

Starting 2019

Starting 2019

Started 2018

Started 2018

Novartis cell therapy platform with broad pipeline and building global manufacturing scale for cell processing


MORRIS

PLAINS

CELL FOR

CURE1 FRAUNHOFER

STEIN

1. Currently operating through manufacturing agreement; proposed acquisition and transaction subject to the completion of customary closing conditions

Starting 2019

Starting 2019

Starting 2019

Preparing to manufacture

Currently manufacturing

CBMG

FBRI

Building global scale for lentiviral vector-based therapies;

established collaborations, working to deliver globally

Sickle cell

disease (SCD)

Preparing to launch the next growth drivers


PIK3CA mutation in ~40% of

HR+/HER2- aBC patients and

associated with poor prognosis;

currently no targeted treatments

Alpelisib in combination with

fulvestrant is the first and only

therapy specifically for

HR+/HER2- aBC patients1 with a

PIK3CA mutation

Potential US launch H2 2019

HR+/HER2-

breast cancer

Alpelisib (BYL719)

Vaso-occlusive crises (VOCs) remain

the most important unmet need in

SCD – associated with decrease in

QoL and increase in risk of organ

damage and death

Crizanlizumab significantly reduced

frequency of VOCs regardless of SCD

genotype and/or hydroxyurea use

Received FDA Breakthrough Therapy

designation in 2018; planned US and

EU filings H1 2019

Crizanlizumab (SEG101)

aBC – advanced breast cancer 1. Post-menopausal women who progressed on or after an aromatase inhibitor with or without a CDK4/6 inhibitor (study included one man)

Alpelisib

PI3K-α inhibitor

Vas NarasimhanChief Executive Officer

Closing

We will continue to drive our priorities in 2019


Deliver

financialsSales growth

and margin

expansion

Deliver pipeline targets,

incl. Zolgensma™,

Mayzent™, RTH258

and BYL719 approvals

Maintain high proportion

of first-in-class /

transformative assets

Extend leadership in

cell, gene and

radioligand therapies

Breakthrough

Innovation

Execute Alcon spin and

progress Sandoz

transformation

Drive productivity

through NTO and NBS

transformations

Deliver performance of

in-market growth drivers

Prepare for 10+ potential

blockbuster launches,

incl. 4 in 2019

Operational

Excellence

Scale top 5 digital

initiatives across the

company

Upskill digital

capabilities in all

units and functions

Build Novartis

position within digital

ecosystem

Data & Digital

Leadership

Continue to embed

principles-based

decision-making (P3)

Implement Novartis

Access Principles, with

every new innovative

medicine launch

having an access plan

Progress efforts in

global health

Building Trust

& Reputation

Continue 5-year

journey to transform

culture, with a focus on

strengthening

leadership capabilities

Further increase

diversity and inclusion

Culture

Transformation

Appendix

Our in-line growth brands provide a solid foundation for continued growth


Q4 Full Year

SalesUSD million

Growth vs. PYUSD million cc

SalesUSD million

Growth vs. PYcc

806 33% 2,837 36%

318 76% 1,028 102%

Lutathera® 81 nm1 167 nm1

330 32% 1,174 35%

313 31% 1,155 31%

256 17% 977 24%

60 71% 235 nm1

28 nm1 76 nm1

268 14% 1,039 12%

191

75

25

133

81

67

28

22

21

1. Not meaningful

Key growth drivers 4 additional blockbusters in 2018

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

Cosentyx®: Gaining share in a growing psoriasis market


1. NRx Share in Dermatology from SHS November 2018. NRx Share amongst Dermatology writers All trademarks are the property of their respective owners

Cosentyx®

Tremfya®

Stelara®

TNFs

Others

Dermatology US NRx evolution1

Cosentyx® prescriptions (NRx) +65%1 in a growing US

biologics market (+25%)1 with 10+ treatment options in 20191

12M ending 11/2018

11%

12M ending 11/2017

14%

+25%

+65%

2018 net debt decreased by USD 2.8bn mainly driven by strong FCF, partly offset by the dividend payment


USD billion

-19.0-16.2

-7.0 -1.3

-13.9

Dec 31, 2017 Dividends M&A transactions1Treasury share

transactions, net

13.0

Proceeds from sale

of GSK consumer

healthcare joint

venture

11.7

Free Cash

Flow

0.3

Others Dec 31, 2018

+2.8

1. Including mainly USD 8.3bn for AveXis, Inc., USD 3.5bn for Advanced Accelerator Applications, S.A. and USD 1.8bn for Endocyte, Inc., all net of cash acquired

FY 2018 Core EPS +9% cc, excluding the impact from discontinuation of OTC JV core income


5.08

FY 2017 FY 2018 FY 2017

Adjusted1

FY 2018

Adjusted1

4.86

5.15

4.65

+6%+9%

Core EPSUSD, % cc

1. FY 2017 and 2018 core EPS adjusted to exclude core income recorded from OTC JV

Core EPS as reported

Key drivers vs. PY:

+ Higher core operating income

+ Lower shares from share buyback

programs

− Discontinuation of OTC JV core income

from April 1st, 2018

Advancing pipeline with 30 potential blockbusters1

Potential blockbusters by planned submission year


1. New molecular entity / new indication with expected peak sales >USD 1bn 2. The brand name Zolgensma™ has been provisionally approved by the FDA for the investigational product AVXS-101 (onasemnogene abeparvovec-xxxx), but

the product itself has not received marketing authorization or BLA approval from any regulatory authorities 3. The brand name Mayzent™ has been provisionally approved by the FDA and EMA for the investigational product siponimod

(BAF312), but the product itself has not been approved for sale in any country 4. Including combos

202120192018 202232020

INC280 NSCLC

OMB157Relapsing MS


RTH258nAMD

SEG101Sickle Cell Disease

QVM149Asthma

Entresto®

HFpEF

Cosentyx®

nrAxSpA

Zolgensma™2

SMA Type 1

Mayzent™3

SPMS

BYL719Advanced breast cancer

CNP520Alzheimer’s Disease

ECF843Dry Eye

HDM201Acute Myeloid Leukemia

CFZ533Multiple

CSJ117Severe Asthma

ZPL389Atopic Dermatitis

UNR844Presbyopia

VAY736Multiple

VPM087CRC / RCC

LJN4524

NASH

LOU064CSU

LNP023Nephropathy

MOR106Atopic Dermatitis

177Lu-PSMA-617mCRPC

QAW039Asthma

QGE031CSU / CIU

ACZ885Lung cancer

ABL001CML

Zolgensma™2

SMA Type 2/3

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





2019 expected pipeline milestones


H1 2019 H2 2019

Regulatory

decisions and

opinions

Mayzent™1 SPMS (US) Alpelisib (BYL719) HR+ Breast Cancer (US)

Kymriah® Ped / Young Adult r/r ALL (JP) Brolucizumab (RTH258) nAMD (US)

Kymriah® r/r DLBCL (JP) Lucentis® RoP (EU / JP)

Promacta® Severe aplastic anaemia (EU) Lucentis® Diabetic Retinopathy (EU)

Zolgensma™ SMA Type 1 (US / EU / JP) Mayzent™1 SPMS (EU / JP)

Xolair® Pollinosis (JP)

Submissions Brolucizumab (Q1 2019) nAMD (US / EU / JP) Cosentyx® NRAxSpA (US / EU / JP)

Crizanlizumab (SEG101) Sickle Cell Disease (US / EU) Entresto® HF-rEF (JP)

Mayzent™1 SPMS (JP) Entresto® HF-pEF (US / EU)

INC280 NSCLC (US / JP)

Ofatumumab (OMB157) Relapsing MS (US / EU)

PDR001 (combination with

Tafinlar® + Mekinist®) Metastatic Melanoma (US / EU)

QVM149 Asthma (EU / JP)

Major trial

readouts

Zolgensma™2 SMA Type 2 Cosentyx® nrAxSpA

Entresto® HF-pEF

Fevipiprant (QAW039) Asthma

Ofatumumab (OMB157) Relapsing MS

PDR001 (combination with

Tafinlar® + Mekinist®) Metastatic Melanoma

✓ Achieved ✕ Missed = On track

1. The brand name Mayzent™ has been provisionally approved by the FDA and EMA for the investigational product siponimod (BAF312), but the product itself has not been approved for sale in any country 2. The brand name Zolgensma™

has been provisionally approved by the FDA for the investigational product AVXS-101 (onasemnogene abeparvovec-xxxx), but the product itself has not received marketing authorization or BLA approval from any regulatory authorities

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) ✓

GP2017 Adalimumab BS (EU) ✓

LA-EP20065 Peg-filgrastim BS (EU) ✓

GP1111 Infliximab BS (EU) ✓

GP20136 Rituximab BS (US) ✕

Submissions ACZ885 CV risk reduction (US/EU) ✓ Mayzent™ SPMS (EU) ✓

Mayzent™ SPMS (US) ✓ RTH258 nAMD (US/EU) Q1 2019

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

Cosentyx® AS (JP) ✓ Zolgensma™ SMA7 (US / EU / JP) ✓

Kymriah® Pediatric ALL + DLBCL (JP) ✓

Promacta® 1st line SAA (US/EU) ✓4

Major trial

readouts

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

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

INC280 ALK- cMET amplified NSCLC ✓

Entresto® HFpEF (Interim Analysis) ✓

✓ Achieved ✕ Missed

The brand name Mayzent™ has been provisionally approved by the FDA and EMA for the investigational product siponimod (BAF312) , but the product itself has not been approved for sale in any country. The brand name Zolgensma™ has

been provisionally approved by the FDA for the investigational product AVXS-101 (onasemnogene abeparvovec-xxxx), but the product itself has not received marketing authorization or BLA approval from any regulatory authorities

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.

MONALEESA 3/7 trials approved in US in July, Europe in December; 4. Approved in US in November; 5. Positive CHMP opinion received 6. Complete Response Letter received from FDA after Q1 2018 results; 7. IV formulation in type 1 SMA;

8. Program discontinued

Added 2018

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

26. Metastatic castration-resistant prostate cancer

Planned filings 2018 to 2023


ABL001CML4 3rd line

QGE031CSU/CIU16

Entresto®

Post-acute myocardial infarction

RTH258Diabetic macular edema

QAW039Asthma

Entresto®

Heart failure (PEF)13

INC280NSCLC6

Cosentyx®

nrAxSpA12

OMB157Relapsing multiple sclerosis

RTH258nAMD7

2023202120202019 2022

Jakavi®Acute GVHD14

Combination abbreviations:

fulv fulvestrant

tmx tamoxifen

gsn goserelin

NSAI Non-steroidal aromatase inhibitor

Taf Tafinlar® (dabrafenib)

Mek Mekinist® (trametinib)

Cosentyx®

PsA H2H17

Cosentyx®

AS H2H19

LAM320MDR8 tuberculosis

ZPL389Atopic dermatitis

Rydapt®AML20 (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

XolairNasal Polyps

ACZ885Adjuvant NSCLC

ACZ8851st Line NSCLC

ACZ8852nd Line NSCLC

Kymriah®

r/r Follicular Lymphoma

Kymriah®

CLL22

Kymriah®

r/r DLBCL in 1st relapse

QVM149Asthma

QMF149Asthma


RTH258Retinal vein occlusion

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

AVXS-201Rett Syndrome

a) US filing, submitted in EU

awaiting HA acceptance

AVXS-101SMA Type 2/325

KAE609Malaria

EMA401Peripheral neuropathic pain

CNP520Alzheimer’s disease

VAY736Primary Sjoegren’s syndrome

KAF156Malaria

LJN452Non-alcoholic steatohepatitis

QBW251COPD21

Kisqali®HR+, HER2 (-) BC9 (adjuvant)

LMI070Spinal muscular atrophy

VAY785Non-alcoholic steatohepatitis

CAD106Alzheimer’s disease

VAY736Autoimmune Hepatitis

Kymriah+ pembrolizumab - r/r DLBCL

ABL001CML4 1st line

HDM201Acute myeloid leukemia

LOU064Chronic spontaneous urticaria

CFZ533Solid Organ Transplant

VPM087CRC 1L/RCC 1L24

CSJ117Severe Asthma

CFZ533Sjorgen’s Syndrome

LJC242Non-alcoholic steatohepatitis

LNP023IgA nephropathy

LNP023Membranous nephropathy


LCI699 USCushing’s disease

Cosentyx®

Hidradenitis suppurativa

177Lu-PSMA-617mCRPC26

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





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



LJN452Non-alcoholic steatohepatitis

QBW251COPD

LMI070Spinal muscular atrophy

UNR844Presbyopia

VAY736Autoimmune Hepatitis

LOU064Chronic spontaneous urticaria

LNP023IgA nephropathy


177Lu-PSMA-617mCRPC26

ABL001CML1 1st line

VAY736Primary Sjoegren’s syndrome

ZPL389Atopic dermatitis

INC280NSCLC2 (EGFRm)

VAY785b

Non-alcoholic steatohepatitis


Kymriah®

+ pembrolizumab - r/r DLBCL

VPM087CRC 1L/RCC 1L24

CFZ533Sjorgen’s Syndrome

LNP023Membranous nephropathy2

LCI699 EUCushing’s disease

Pipeline of key projects in confirmatory development


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

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

c) US Submitted. EU submission Jan 2019

.

Lucentis®

ROP16

Promacta®/Revolade®

SAA17 1st line

SEG101Sickle cell disease

QAW039Asthma

RTH258nAMD6

LCI699 USCushing’s disease

ACZ885Adjuvant NSCLC2

ACZ8851st Line NSCLC2

INC280NSCLC2

ABL001CML1 3rd line

QGE031CSU/CIU3

PDR001 + Tafinlar®+Mekinist®

Metastatic BRAF V600+ melanoma

Lucentis®

Diabetic retinopathy

BAF312SPMS20

LAM320MDR15 tuberculosis

QMF149Asthma

QVM149Asthma

OMB157Relapsing multiple sclerosis

RTH258Diabetic macular edema

Rydapt®AML18 (FLT3 wild type)

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

and others (same as originator)

XolairNasal Polyps

RTH258Retinal vein occlusion

Kymriah®

CLL22

Kymriah®

r/r DLBCL in 1st relapse

CAD106Alzheimer’s disease

KAE609Malaria

EMA401Peripheral neuropathic pain

CNP520Alzheimer’s disease

ECF843Dry eye

KAF156Malaria

HDM201Acute myeloid leukemia

CFZ533Solid Organ Transplant

AVXS-101SMA Type 2/325

CSJ117Severe Asthma

AVXS-201Rett Syndrome

AVXS-101SMA Type 123

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

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 Spinal Muscular Atrophy Type 2/3

26. Metastatic castration-resistant prostate cancer

BYL719a/c + fulvHR+, HER2 (-) postmenopausal

adv. BC9 2nd line

Cosentyx®

nrAxSpA9

Entresto®

Heart failure (PEF)13

Entresto®

Post-acute myocardial infarction

Jakavi®Acute GVHD14

Cosentyx®

PsA H2H10

Cosentyx®

AS H2H11

Kisqali®

HR+, HER2(-) BC5 (adjuvant)

Jakavi®Chronic GVHD14

ACZ8852nd Line NSCLC2

Kymriah®

r/r Follicular Lymphoma

Cosentyx®

Hidradenitis suppurativa

LJC242Non-alcoholic steatohepatitis


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







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

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



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



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





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








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




References


Slide 38 – 2019: Preparing to launch next wave of growth drivers

1. The brand name Mayzent™ has been provisionally approved by the FDA and EMA for the investigational product siponimod (BAF312), but the product itself has not been

approved for sale in any country 2. The brand name Zolgensma™ has been provisionally approved by the FDA for the investigational product AVXS-101 (onasemnogene

abeparvovec-xxxx), but the product itself has not received marketing authorization or BLA approval from any regulatory authorities 3. Institute for Clinical Economic Review (ICER)

Draft Evidence Report, page 79 https://icer-review.org/wp-content/uploads/2018/07/ICER_SMA_Draft_Evidence_Report_122018-1.pdf 4. Week 48 – demonstrated superiority in

three secondary endpoints considered key markers of nAMD in clinical practice: central subfield retinal thickness, retinal fluid (intraretinal fluid and/or subretinal fluid) and disease

activity; advantages maintained at Week 96 5. Week 48 – majority of patients (56% and 51%) were maintained on q12w injection interval in HAWK and HARRIER respectively with

remaining patients on q8w regimen (key secondary endpoints); greater than 75% of these patients continued on q12w dosing up to Week 96 6. Met primary efficacy endpoint of

noninferiority to aflibercept in mean change in BCVA with comparable safety to aflibercept; vision gains comparable to aflibercept up to Week 96

https://icer-review.org/wp-content/uploads/2018/07/ICER_SMA_Draft_Evidence_Report_122018-1.pdf

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. Q3 2018 presentation


New additions

Trials taken out (operational decision-points achieved)

Study Program Indication Phase Patients

NCT02152761 (CBYM338D2201) BYM338 Hip fracture recovery Phase 2B 245

NCT02333331 InvestiGAIT (CBYM338E2202) BYM338 Sarcopenia Phase 2B 280

NCT01327846 CANTOS (CACZ885M2301) ACZ885 Cardiovascular risk reduction Phase 3 10,061

Study Program Indication Phase Patients

NCT03578367 (CABL001E2201) ABL001 Chronic myeloid leukaemia (CML) Phase 2 120

NCT03663335 (CFZ533A2201) CFZ533 Kidney transplantation Phase 2 325

NCT03701334 NATALEE (CLEE011012301C) Kisqali® Early breast cancer Phase 3 4,000

NCT03568461 ELARA (CCTL019E2202) Kymriah® Relapsed / refractory follicular lymphoma (FL) Phase 2 113

NCT03580369 PEARL 1 (CQGE031C2302) QGE031 Chronic spontaneous urticaria Phase 3 1,050

NCT03580356 PEARL 2 (CQGE031C2303) QGE031 Chronic spontaneous urticaria Phase 3 1,050

NCT03517566 ZEST (CZPL389A2203) ZPL389 Atopic dermatitis Phase 2 360

Cardio-Metabolic

Entresto® - Angiotensin receptor neprilysin inhibitor (ARNI)

62

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

Indication Cardiac failure congestive Cardiac failure congestive

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 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 from 1 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 (actual)

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)


63

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

Indication Cardiac failure congestive Cardiac failure congestive

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 2022Q4-2019 (primary); 2020 (open-label extension)

Publication TBD TBD



64

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

Indication Cardiac failure congestive 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) H2-2020

Publication

• Presentation planned AHA Nov-2018;

• Publication submitted to NEJM Q4-2018 (not yet

accepted)

TBD



65

Study NCT01920711 PARAGON (CLCZ696D2301) NCT03066804 PARALLAX (CLCZ696D2302)

Indication Cardiac failure chronic Cardiac failure chronic


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 Q3-2019 H1-2020

Publication TBD TBD



Immunology, Hepatology & Dermatology

CFZ533 – Blocking, non-depleting, Fc-silent, anti-CD40 monoclonal antibody

Study NCT03663335 (CFZ533A2201)

Indication Renal transplant

Phase Phase 2b

Patients 325

Primary Outcome

Measures

Composite event (BPAR, Graft Loss or Death)

over 12 months post-transplantation and post

conversion (for maintenance cohort)

Arms/Intervention

Target PatientsDe novo and maintenance kidney transplant

recipients

Expected

Completion2021

Publication TBD


Cosentyx® - Anti IL-17

68

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)



69

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



70

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

• Secukinumab high dose

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

severe plaque psoriasis

Expected Completion Q3-2018 (actual) 2023

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



71

Study NCT02748863 ALLURE (CAIN457A2323) NCT02745080 EXCEED (CAIN457F2366)

Indication Psoriasis Psoriatic arthropathy


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



72

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

Indication Psoriatic arthropathy 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



73

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



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 (actual)

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



74

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 Q1-2019 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



75

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



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

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



76

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

Indication Psoriatic arthropathy Psoriatic arthropathy


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



77

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 2021 2022

Publication TBD TBD


Ilaris® - Anti IL-1β

78

Study NCT02059291 CLUSTER (CACZ885N2301) NCT02296424 (CACZ885G2306)

Indication Autoimmune disorder 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)

• Disease specific publications on TRAPS, FMF and

HIDS in 2019

• Additional manuscripts in 2019

Manuscript to be submitted in Q2-2019


LJN452 - FXR Agonist

79

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

80

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 Urticaria 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) Q2-2019

PublicationPrimary results: Presented at EAACI 2018, EADV 2018, and

GUF 2018; manuscript expected in Q1-2019

Primary results: Late breaker abstract (showing 1 year

treatment results) submitted for AAD in Q1-2019; manuscript

submission expected in Q4-2019


QGE031 - Anti-IgE

Study NCT03437278 (CQGE031C2202)

Indication Urticaria

Phase Phase 2

Patients 48

Primary Outcome

MeasuresChange in the 7 day Urticaria Activity Score (UAS7)

Arms/Intervention

• Ligelizumab high dose q4wks

• Ligelizumab low dose q4wks

• Placebo / ligelizumab high dose q4wks

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

spontaneous urticaria

Expected Completion H2-2020

Publication Manuscript to be submitted in 2021


QGE031 - Anti-IgE

Study NCT03580369 Pearl 1 (CQGE031C2302) NCT03580356 Pearl 2 (CQGE031C2303)

Indication Urticaria Urticaria



Primary Outcome

Measures

Absolute change from baseline in UAS7 (Urticaria Activity

Score) at week 12

Absolute change from baseline in UAS7 (Urticaria Activity

Score) at week 12

Arms/Intervention

• Ligelizumab dose A q4w

• Ligelizumab dose B q4w

• Omalizumab 300 mg q4w

• Placebo q4w from randomization to wk20, then

ligelizumab dose B from wk24 to wk48

• Ligelizumab dose A q4w

• Ligelizumab dose B q4w

• Omalizumab 300 mg q4w

• Placebo q4w from randomization to wk20, then

ligelizumab dose B from wk24 to wk48

Target PatientsAdolescents and adults with chronic spontaneous urticaria

and inadequately controlled with H1-antihistamines

Adolescents and adults with chronic spontaneous urticaria

and inadequately controlled with H1-antihistamines


Publication TBD TBD


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

Study NCT02962895 (CVAY736A2201) NCT03217422 AMBER (CVAY736B2201)

Indication 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) normalization

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

Expected Completion H2-2020 2023

Publication TBD TBD


ZPL389 - H4 receptor antagonists

Study NCT03517566 ZEST (CZPL389A2203)

Indication Atopic dermatitis

Phase Phase 2

Patients 360

Primary Outcome

MeasuresIGA (Investigator's global assessment) response at week 16

Arms/Intervention

• ZPL389 dose 1

• ZPL389 dose 2

• ZPL389 dose 3

• ZPL389 dose 4

• Placebo

Target Patients Patients with moderate to severe atopic dermatitis


Publication TBD


Neuroscience

Aimovig® – CGRP receptor antagonist

87

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 H1-2020

Publication Planned in 2019 (more details will follow) TBD


CNP520 - BACE inhibitorCAD106 - active beta-amyloid immunotherapy

88

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

89

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


EMA401 - Angiotensin II type 2 receptor antagonist

90

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 H2-2020 H1-2020

Publication TBD TBD


Gilenya® - S1P-R modulator

91

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 (actual)

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

92

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

93

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 Q3-2020

Publication TBD


OMB157 - Anti-CD20

94

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

Expected Completion Q3-2019 Q3-2019

Publication TBD TBD


OMB157 - Anti-CD20

95

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

97

Study NCT03106779 (CABL001A2301) NCT03578367 (CABL001E2201)

Indication Chronic myeloid leukaemia (CML) Chronic myeloid leukaemia (CML)


Patients 222 120

Primary Outcome

MeasuresMajor Molecular Response (MMR) rate at 24 weeks Deep molecular response (MR 4.5) at 48 weeks

Arms/Intervention• ABL001 40 mg

• Bosutinib 500 mg

• ABL001 40 mg QD + 400 mg imatinib

• ABL001 60 mg QD + 400 mg imatinib

• Imatinib 400mg QD (continuation treatment)

• Nilotinib 300mg BID (switch to nilotinib treatment)

Target Patients

Patients with chronic myelogenous leukemia in chronic

phase, previously treated with 2 or more tyrosine kinase

inhibitors

CML-CP patients not reaching DMR (MR 4.5) while on 1L

imatinib treatment


Publication TBD 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

99

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


PublicationAndre F, et al. Presentation at ESMO 2018

Manuscript submitted Q4-2018


Kymriah® – CAR-T therapy

100

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

• Schuster et al. N Engl J Med. 2018; epub ahead of print.

DOI: 10.1056/NEJMoa1804980

• 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


Kymriah® – CAR-T therapy

101

Study NCT03568461 ELARA (CCTL019E2202)

Indication Relapsed / refractory follicular lymphoma (FL)

Phase Phase 2

Patients 113

Primary Outcome

MeasuresComplete response rate (CRR)

Arms/Intervention Single-arm study of tisagenlecleucel

Target Patients Adult patients with relapsed or refractory FL


Publication TBD


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

102

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


PublicationAngelucci E, et al. Presentation at ASH 2018

Manuscript submitted Q4 2018


INC280 - MET Inhibitor

103

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 de novo

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 Q2-2019 Q4-2018 (actual)

Publication• Wolf et al. Presentation at ESMO 2018

• Manuscript submission Q2-2019Abstract submission Q2 2019


Jakavi® - JAK1/2 inhibitor

104

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)


PublicationManuscript Submission Q4-19

Congress presentation Q4 2019 TBD


Kisqali® - CDK 4/6 inhibitor

105

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 presentation at ASCO 2018

• Slamon D, et al. J Clin Oncol 36:2465-2472;

DOI:https://doi.org/10.1200/JCO.2018.

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

• Tripathy D, et al. Lancet Oncol 2018; 19: 904–15;

DOI:https://doi.org/10.1016/S1470-2045(18)30292-4


Kisqali® - CDK 4/6 inhibitor

106

Study NCT03701334 NATALEE (CLEE011O12301C)

IndicationAdjuvant treatment of hormone receptor (HR)-positive,

HER2-negative, early breast cancer (EBC).

Phase Phase 3

Patients ~4,000

Primary Outcome

Measures

Invasive Disease-Free Survival for using STEEP criteria

(Standardized Definitions for Efficacy End Points in adjuvant

breast cancer trials)

Arms/Intervention• Ribociclib + endocrine therapy

• Endocrine therapy

Target Patients

Pre and postmenopausal women and men with HR-positive,

HER2-negative EBC, after adequate surgical resection, who

are eligible for adjuvant endocrine therapy


Publication TBD


LCI699 - Cortisol synthesis inhibitor

107

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 H2-2020 Q2-2018 (actual)

Publication TBD Pivonello et al. presented at ICE 2018


PDR001 – PD-1 checkpoint inhibitor

108

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

109

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) H1-2020

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

110

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 (actual)H2-2020 (pediatric patients ≥6 year old)

2021 (pediatric patients 6 months – 6 year old)

Publication Abstract submission Q1-2019 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 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

• Manuscript Q1-2019


Tafinlar®+Mekinist® - BRAF inhibitor and MEK inhibitor

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


PDR001 - PD-1 checkpoint inhibitor

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 + capmatinib 400 mg BID

orally

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

Q4W

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): Cho B et al. Poster Presentation at ESMO

2018; Manuscript submission Q1 2019

Ophthalmology

Lucentis® - Anti-VEGF

121

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.

Expected Completion Q1-2018 (actual) 2023

Publication

• EURETINA: Sep-2018

• AAO: Oct-2018

• Primary manuscript: planned submission by end of 2018

to NEJM

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


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


Publication

• Oral presentations including both primary endpoint and key 2nd superior anatomic outcomes at AAO meetings in Nov-

2017 (1st year results) and Nov-2018 (2nd year results)

• Manuscript on 1st results was submitted to Journal of Ophthalmology Dec-2018; 2nd year result manuscript will be

submitted to J. of Ophtha in Q2-2019

• Abstracts submissions on superior anatomic outcomes/Fluid/PostHoc results are planned for key retinal congresses


RTH258 - Anti-VEGF

Study NCT03481634 KESTREL (CRTH258B2301) NCT03481660 KITE (CRTH258B2302)

Indication Diabetic eye disease Diabetic eye disease


Patients 534 356

Primary Outcome

Measures

Change from baseline in best-corrected visual acuity

(BCVA)

Change from baseline in best-corrected visual acuity

(BCVA)

Arms/Intervention

• RTH258 3 mg/50 µL

• RTH258 6 mg/50 µL

• Aflibercept 2mg/50 uL

• RTH258 6 mg/50 µL


Target PatientsPatients with visual impairment due to diabetic macular

edema (DME)

Patients with visual impairment due to diabetic macular

edema (DME)


Publication TBD TBD


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/50 µL


Target PatientsPatients with neovascular age-related macular degeneration

who have completed the CRTH258A2301 study


Publication TBD


Respiratory

QAW039 – DP2 receptor antagonist

126

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 H1-2020 Q3-2019

Publication TBD TBD



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



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 Q4-2019 (for submission); 2022 (final)

Publication TBD


QMF149 - Long-acting beta2 agonist and inhaled corticosteroid

129

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



131

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


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

134

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


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

136

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 published in RMD Open (20-Nov-

2018)

• 48 week: Manuscript in Q4-2018 (submitted to Journal

Arthritis Research &Therapy on 08-Nov-2018)


Rixathon® - Biosimilar rituximab

137

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)


Hyrimoz® - Biosimilar adalimumab

138

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

140

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

141

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

142

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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