Novartis : investors presentation

Novartis AG

Investor Relations

Q4 and FY 2019 Results

Investor Presentation

January 29, 2020

Disclaimer

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," "may," "could," "would," "anticipate," "seek," or similar expressions, or by express or implied discussions regarding potential new products, potential new indications for existing products, potential product launches, or regarding potential future revenues from any such products; or regarding the development or adoption of potentially transformational technologies, treatment modalities and business models; or regarding potential future or pending transactions, including the potential outcome, or financial or other impact on Novartis, of the proposed divestiture of certain portions of our Sandoz Division business in the US; or regarding the potential impact of share buybacks; or regarding potential future sales or earnings of the Group or any of its divisions or potential shareholder returns; 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, payor and general public pricing and reimbursement pressures and requirements for increased pricing transparency; uncertainties regarding potential significant breaches of data security or data privacy, or disruptions of our information technology systems; 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 presentation; the potential that the proposed divestiture of certain portions of our Sandoz Division business in the US or the planned acquisition of the Japanese operations and associated assets of Aspen Global Incorporated, may not be completed in the expected time frame, or at all; the potential that the strategic benefits, synergies or opportunities expected from the acquisition of The Medicines Company, the proposed divestiture of certain portions of our Sandoz Division business in the US, or the planned acquisition of the Japanese operations and associated assets of Aspen Global Incorporated, and other transactions described, may not be realized or may be more difficult or take longer to realize than expected; the successful integration of The Medicines Company into the Novartis Group and the timing of such integration; potential adverse reactions to the transaction by customers, suppliers or strategic partners; dependence on key personnel of The Medicines Company; dependence on third parties to fulfill manufacturing and supply obligations; the uncertainties involved in predicting shareholder returns; the uncertainties 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 is expected to continue this year; safety, quality, data integrity, or manufacturing issues; uncertainties involved in the development or adoption of potentially transformational technologies and business models; uncertainties regarding actual or potential legal proceedings, including, among others, product liability litigation, disputes and litigation with business partners or business collaborators, government investigations generally, litigation and investigations regarding sales and marketing practices, and intellectual property disputes; 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; 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.

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Participants

Vas Narasimhan	John Tsai
Chief Executive Officer	Head of Global Drug Development and CMO
Harry Kirsch	Richard Saynor
Chief Financial Officer	CEO, Sandoz
Marie-France Tschudin	Shannon Thyme Klinger
President, Novartis Pharmaceuticals	Group General Counsel
Susanne Schaffert
President, Novartis Oncology

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In 2019, we kept executing on our strategy

Focus Novartis as a leading medicines company powered by advanced therapy platforms and data science

Our focus

Our priorities

Focus our company	Strengthen our core	Deliver transformative	Embrace operational	Go big on data and
and capital		innovation	excellence every day	digital

Accelerate	Unleash the power of	Build trust with society
key geographies	our people

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We have focused Novartis as a medicines company

Medicinal chemistry	Diversified	Portfolio	Focused medicines company powered by
and industrials	healthcare group	transformation	advanced therapy platforms and data science
1920 - 1996	1996 - 2009	2009 - 2017	2018 - 2019

		Acquired	Divested OTC1	Acquired	Acquired	Spun off	Acquired	Acquired

	USD 3.9bn	USD 13bn	USD 8.7bn	USD 2.1bn	USD 28bn2	USD 3.4bn3	USD 9.7bn
1. OTC - Consumer Healthcare	2. Alcon market capitalization on close of 1stday of trading	3. USD 3.4bn upfront + potential milestone payments of up to USD 1.9bn

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Transformative innovation

Leading pipeline with strong replacement power

Scale

Value

Innovation

# of projects1

114PHASE 1 / 2

37PHASE 3

13REGISTRATION

Estimated 2024 sales from

products launched between 2019-242

#1Replacement power

Company A

Company B

Company C

Company D

Company E

Company F

Company G

Company H

Company I

16	Advanced platform
therapies
in clinical development

	Pipeline potentially
	first-in-class /
~90%first-in-indication
~80%	Target areas of high
	unmet need

1. Including Global Health, excluding Sandoz. 2. Innovative medicine product sales excl. Vaccines and LCM products (e.g. new formulations, combinations with off patent molecules); compound-based analysis (Phase 2 and 3) with additional indications allocated to 1stlaunch. Inclisiran included. Source: Novartis peer group analysis based on data from Evaluate Pharma (download from November 27, 2019)

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Transformative innovation

2019 was a breakthrough year for innovation

5NME approvals of potential blockbusters		30+major submissions		30+clinical data readouts
aSPMS		Select examples		Select examples
			Entresto®(JP)			Zolgensma®
SMA			Cosentyx®nr-AxSpA(US/EU)			Cosentyx®
			Ofatumumab (US)			Ofatumumab
Breast cancer			Adakveo®(US/EU)			Entresto®
			Beovu®(US/EU/JP)			Fevipiprant
Wet AMD			INC280 (US)			Kisqali®
			QVM149 / QMF149 (EU/JP)			INC280
Sickle cell disease			Inclisiran (US)1			Inclisiran1
aSPMS - Active secondary progressive multiple sclerosis SMA - Spinal muscular atrophy	AMD - age-related macular degeneration 1. Readout / submission by The Medicines Company

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Transformative innovation

2020 catalysts maintaining long-term momentum

Potential catalysts

Major approvals1

Major submissions2

Major readouts3(Phase 3)

Phase 3 starts

Select examples

Ofatumumab (OMB157)	Capmatinib (INC280)	Cosentyx®
Relapsing MS	NSCLC	nr-axSpA
QVM / QMF 149	Entresto®	Inclisiran (KJX839)
Asthma	HFpEF (US)	Hyperlipidemia (US)
Inclisiran (KJX839)	AVXS-101 IT4	Alpelisib (BYL719)
Hyperlipidemia (EU)	SMA	PROS
177Lu-PSMA-617	Spartalizumab (PDR001) combo	Entresto®
mCRPC	Metastatic melanoma	HFpEF (US)
177Lu-PSMA-617	Beovu®	Entresto®
mCRPC	DME	Post-acute MI (IA5)
Asciminib (ABL001)	Kisqali®	Jakavi®
Chronic Myeloid Leukemia	Breast cancer(MONALEESA-2 OS)	Chronic GvHD
TQJ230	LNP023	MBG453
CVRR	PNH	MDS
Tropifexor (LJN452)	Alpelisib (BYL719)	Beovu®
NASH	Multiple indications6	PDR

1. First approval in any market. 2. First submission in any market 3. Readouts enabling submission, label change or pivotal trial initiation 4. FDA placed a partial clinical hold based on findings in a small preclinical animal study 5. Planned interim analysis expected Q1 2020 (full readout 2021) 6. HER2+ aBC, TNBC, ovarian cancer, head and neck cancer, PROS

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Transformative innovation

Innovation driving growth in China

Number of NDA approvals1

50+

x2

25

2015-2019	2020-2024
Actual	Expected

• Growth driven by regulatory approvals from innovative pipeline, market access and optimizing resources
• 13 NME approvals over the past 5 years, and 22 NRDL listings since 2017
• Average # of NDAs expected to double in the next 5 years
• • 5 average NDA approvals / year2015-2019
  • 10 average NDA approvals expected / year2020-2024
  • 50+ NDA approvals planned over the next 5 years
• Goal to deliver >90% of 2024+ China submissions simultaneously with global submission

1. NDA approvals of new compounds and new indications

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Operational excellence

Delivered strong performance in 2019

Continuing operations1, FY 2019, TSR as of YE 2019

47.4 bn	+9%
NET SALES (USD)	vs. 2018(cc2)
14.1 bn	+17%
CORE OPERATINGINCOME2(USD)	vs. 2018(cc2)
12.9 bn	+15%
FREE CASH FLOW2(USD)	vs. 2018(USD)

33.5%	+1.8% pts
IM CORE MARGIN2(%)	vs. 2018(cc2)
5.28	+17%
CORE EPS2(USD)	vs. 2018(cc2)
22.3%	Top tier
1-YEAR TSR3(%)	RANKING 3

1. Continuing operations as defined on page 45 of the Condensed Financial Report, excludes Alcon, includes the businesses of Innovative Medicines and Sandoz (including the US generic oral solids and dermatology portfolio), as well as the continuing corporate functions. 2. Constant currencies (cc), core results and free cash flow are non-IFRS measures. An explanation of non-IFRS measures can be found on page 58 of the Condensed Financial Report. 3. TSR in USD from Jan 1st2019, using 1 day average price at start and 3 month average price at end; ranking when compared to the global HC peer group as defined in the Novartis 2019 Annual Report

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Operational excellence

Strong operational performance from growth drivers

Selected growth driver sales

	Sales	Growth vs. PY	Growth vs. PY
	USD Million	USD Million	cc
	3,551		714	28%
	1,726		698	71%
	361		361	nm
Lutathera®	441		274	160%
	480		245	111%
	1,416		242	23%
	278		202	nm
	192		192	nm
	1,338		183	20%
	1,114		137	20%
	1,173		134	19%
	671		117	25%
	116		116	nm
nm - not meaningful

Growth drivers and recent launches as % of Innovative Medicines sales

				35%				Adakveo®
									Luxturna®
									Mayzent®
									Beovu®
		27%						Aimovig®
							Piqray®
									Xiidra®
	20%								Kymriah®
								Zolgensma®
									Lutathera®
14%									Kisqali®
								Jakavi®
									Ilaris®
									Xolair®
									Tafinlar®+Mekinist®
									Promacta®
									Entresto®
2016	2017	2018	2019				Cosentyx®

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Operational excellence

Focused on launch excellence for 15 ongoing and upcoming major launches

				Inclisiran (KJX839)
				Hyperlipidemia
Mayzent®				Entresto®
aSPMS				HFpEF
Zolgensma®IV		Ofatumumab (OMB157)		AVXS-101 IT
SMA		Relapsing MS		SMA
Piqray®		Capmatinib (INC280)		Alpelisib (BYL719)
Breast Cancer		NSCLC		PROS
Beovu®		Cosentyx®		177Lu-PSMA-617
Wet AMD		nr-axSpA		mCRPC
Adakveo®		QVM / QMF 149		PDR001 combo
Sickle cell disease		Asthma		Metastatic melanoma
2019	2020	2021

Ongoing Upcoming

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Operational excellence

Zolgensma®strong launch (June) with FY 2019 sales of USD 361m

Broad access, strong patient demand

	Q3 end (~)	YE (~)
Patients treated commercially	100	200
Commercial lives covered	90%	97%
Medicaid lives covered	30%	>50%
Newborns screened1	30%	39%
Approval rate for on-label patients		>99%

Global Managed Access Program initiated

Next steps

		Intrathecal formulation on partial clinical
		hold - regulatory discussions ongoing
		CHMP opinion anticipated Q1 2020
		Approval anticipated in H1 2020
		Decisions anticipated late 2020 or early
Others
2021 in Switzerland, Canada, Australia

1. Expected to reach 70% by end of 2020

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Operational excellence

Committed to driving consistent margin expansion

Innovative Medicines

Core margin (% rounded)

Mid to high

Mid 30s

34 30s

31 32

2017	2018	2019 near term medium
		term

• Sales momentum of key growth drivers and operational excellence on upcoming launches
• Productivity programs in Novartis Technical Operations and Novartis Business Services
• Resource allocation in commercial units

• Generic erosion
• Launch investments for potential future blockbusters, including inclisiran

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Operational excellence

Advancing deep transformations in NTO and NBS

Strategic levers

Organization

Footprint

Procurement

Data & Digital

NTO	NBS
Reduced ~1,800FTEs across global functions and	On track to reduce ~600FTEs by 2022
site operations (net of site exits)	Associates in NGSCs from <40% to >50%
10sites exited	Footprint reduction through sale, lease-back, and
9additional exits announced	Activity-Based Working (20+offices)
102out of 210 warehouses consolidated	Moved to singleservice provider for REFS
28%reduction in suppliers for finished product, API	NewChief Procurement Officer
45%reduction in suppliers for indirect materials	Optimizing terms with top 100suppliers
Delivering advanced analytics solutions on asset,	Continued investments in tech enablers
material andinventory management	Re-designofkey enterprise processes

On track for ~USD 2bn savings by end of 2020; new efforts expected to deliver additional ~USD 1.5bn savings medium term

NTO - Novartis Technical Operations; Baseline EOY 2016 NBS - Novartis Business Services; Baseline EOY 2018 NGSCs - Novartis Global Service Centers API - Active pharmaceutical ingredients REFS - Real estate and facility services

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Operational excellence

Sandoz delivered accretive growth in 2019 by implementing refined strategy

Sales growth (vs. PY, cc)

Refined strategy

+2%GLOBAL

+7%EX-US

+16%BIOPHARMACEUTICALS

Core operating income growth (vs. PY, cc)

+10%

Geographic priorities

Increasing autonomy

Portfolio update

• EU: Solidifying #1 position
• JP: Closing Aspen acquisition and investing
• US: Stabilizing the business
  In the process of concluding oral solids business divestment
  Launching pegfilgrastim
• Creating Sandoz TechOps organization
• Building biosimilar pipeline further - trastuzumab / natalizumab deals
• Appealing US Erelzi®decision
• Gx Advair®discontinued further development

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Go big on data and digital

Four core elements to our digital transformation

Scale 12 digital		Make Novartis digital		Become the #1 partner		Pursue bolder moves
lighthouses			in tech ecosystem

Spanning the entire value chain, from development to commercial operations

In full flight with 2-3yearimplementation horizon

Investing in technology platforms, including CRM,

MDM, API

>1,500 associates mobilized	Scale novel partnership
Rapidly build Data Science	accelerator: the Novartis

and AIcapabilities	Biome
Move to One Digitalglobal	Complementinternal skills
and capabilities
collaboration platform
Dedicated leadership	Closely linked to business
priorities
capability program

Getting ready for disruptive healthcare scenarios through large-scale alliances, e.g.:

• Microsoft: AI Innovation Lab
• AWS1: TechOps optimization
• Tencent: Heart Failure patient solution in China

1. AWS: Amazon Web Services

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Unleash the power of our people

Broad set of initiatives to drive culture change

Inspired		Curious		Unbossed
Connect to our purpose		Go big on learning	Build leadership
and provide an inspiring				self-awareness and
working environment				capabilities

	Spark live to 83,000 associates,		Coursera: ~3,500 courses completed	18 countries visited by CEO in 2019
	230,000 recognitions given in 2019		by 7,000+ users (~85,000 hours)1		Unbossed Leadership Experience
	Minimum 14 weeks paid leave for all		LinkedIn Learning: ~14,000 courses		(ULE) to be completed in 2020 for the
	parents, regardless of gender		available, 12,500+ users1		top 300 leaders in the company

1. As of YE 2019

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Build trust with society

In 2019, we made important progress on our efforts to build lasting trust with society

Ethical Standards	Pricing & Access	Global Health	Corporate Citizenship
Rolled out globally the new Third	BroughtLIC & LMIC prices in	Signedpartnership for SCD	Joined theUN Equal Pay
Party Risk Management system	line with EU5 average	with the Government of Ghana	International Coalition
Drafting the new Code of Ethics,	Outlined new access strategy	JoinedGlobal Chagas Disease	Achieved44% female
representation in management
co-created with our associates	forSub-Saharan Africa	Coalition

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Build trust with society

Introducing ambitious 2020 ESG targets which are deeply embedded in our operating model

Holistic set of ESG targets for 2020...

Pillar	Target
Ethical	Transparency on clinical trials
Standards	Strengthen Third Party Risk Management
	Fully integrate Human Rights into TPRM
Pricing	Increase patient reach
& Access	Enhance access
	Implement pricing principles
Global	Malaria: Advance development of new drugs
Health	Sickle cell disease: Expand coverage
	Chagas: Progress on clinical trial
Corporate	Reduce energy & carbon
Citizenship	Reduce waste
	Reduce water

... deeply embedded in our operating model

Systematically reviewed

Tracked bi-monthly at the Trust & Reputation Committee, a sub-committee of the Executive Committee of Novartis (ECN) chaired by the CEO

Linked to compensation

Cascaded into ECN personal objectives, and directly impacting compensation

Transparently disclosed

To be included in 2020 Annual Report, providing disclosure on our goals and progress

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Build trust with society

Bold long-term aspirations across the ESG spectrum

Select examples

	Reducelaunch time lag			Achieve carbon
			neutrality in own
	to 3 monthsin LMICs
			operations by 2025
	Implement access			Deliver on UN EPIC
	strategy foradvanced			andLGBTI equity
	therapies in LMICs			pledges
	Transform treatment of			Holistically address
	malaria withUSD 100m			sickle cell disease in
	committed in R&D1			Ghana
					www.novartis.com/nisreport2019
1. Commonwealth Heads of Government Meeting April 2018, commitment over the next 5 years	LMICs - low and middle income countries

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In-market growth drivers, major launches and a rich pipeline expected to sustain our long-term growth

• 15ongoing / upcoming major launches
• 80+major submissions planned to 2022
• 50+late stage programs1

Major launches

In-market growth drivers

Novel assets

Inclisiran	MBG453
TQJ230	Asciminib
LNP023	Canakinumab
Iscalimab	Capmatinib
Ligelizumab	Spartalizumab
AD portfolio2	177Lu-PSMA-617
LNA043
Tropifexor
Ofatumumab
UNR844
QVM / QMF149

New indications

Cosentyx®HS	Alpelisib PROS
Cosentyx®GCA	Piqray®TNBC
Cosentyx®LP	Piqray®HER2+ aBC
Cosentyx®JIA	Piqray®ovarian cancer
Cosentyx®LN	Piqray®HNSCC
Entresto®post-AMI	Kisqali®HR+/HER2- BC (adj)
Beovu®DME	Kymriah®FL
Beovu®RVO
Beovu®DR
Beovu®PDR
Ofatumumab pediatric
AVXS-101 IT

S E L E C T E X A M P L ES

1. Ph3 / in registration 2. AD portfolio - atopic dermatitis portfolio incl. ZPL389, CEE321

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Strong Pharmaceuticals growth driven by Cosentyx®, Entresto®and recent launches

Pharmaceuticals net sales

USD billion, growth in % cc

		+7%			+12%
				23.3
						0.7
		21.5
20.0
3.9	5.5	7.1
16.1	16.0	15.5

201720182019

Key growth drivers1Recent launches2Established products3

Strong sales momentum driven by growth drivers

• Mainly Cosentyx®and Entresto®
• Maintaining solid performance of established products

Focus on launches to deliver next phase of growth

• Launched Zolgensma®, Beovu®and Mayzent®in US
• Xiidra®acquired and integrated into Ophtha franchise
• Added inclisiran to CRM pipeline

CRM - Cardiovascular, Renal and Metabolism 1. Cosentyx®, Entresto®, Xolair®, Ilaris®2. Zolgensma®, Xiidra ®, Aimovig®, Luxturna®, Mayzent®and Beovu®3. All other brands

25 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

Cosentyx®Q4 sales driven by strong demand and broad access across indications and regions

Cosentyx®sales evolution				Strong performance, above market
USD million
							Q4 sales up +21% cc
	Ex-US					3.6bn
							PsO TRx outperform market (+27% YoY vs. +12%)1
	US
		2.8bn				937	965	SpA TRx growing >2x faster than market2
						858
				806	791			Maintain momentum
		701	750
									nr-axSpA submitted to FDA in Dec
580
								Pediatric PsO submitted to EMA in Nov
										Expected to maintain broad access in 2020
									Upgrading expected peak sales >USD 5bn
Q1	Q2	Q3	Q4	Q1	Q2	Q3	Q4
			2018				2019

1. IQVIA US National Prescription Audit for Dermatology WE 12/20/2019; market includes Enbrel®, Humira®, Siliq®, Skyrizi®, Stelara®, Taltz®, Tremfya®2. IQVIA US National Prescription Audit for Rheumatology WE 12/20/2019; SpA market includes Cimzia®, Enbrel®, Humira®, Simponi®, Stelara®, Taltz®All trademarks are the property of their respective owners

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Entresto®solidifying leadership position in 2019 as an essential first-choice treatment in heart failure

Entresto®sales evolution

USD million, cc

	Ex-US			1.7bn
	US
		1.0bn			518
			421	430
				318	357
			271
		239
200

Q1

Q2

Q3

Q4

Q1

Q2

Q3

Q4

20182019

Strong momentum

• Q4 sales +65% driven by increased demand in hospital and ambulatory settings
• US TRx growth (+48% vs. PY)

Poised for further growth acceleration

• New data on reverse cardiac remodeling,in-hospital use and QoL
• Inclusion on China NRDL, supporting expanded use
• US regulatory submission for HFpEF on track for Q1
• Japan launch expected in H2

AHA - American Heart Association; QoL - Quality of Life; NRDL - National Reimbursement Drug List; HFpEF - Heart Failure with preserved Ejection Fraction

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Strong start to Beovu®US launch with excellent customer feedback and broad access

Strong uptake in 3 months since launch1	Broad access and reimbursement
84%	of US retina specialists have	Jan 1	permanent J-Code received,
Beovu®available in their office	providing greater reimbursement
			confidence
90%	of retina specialists who don't	95%	positive benefits verification
currently use Beovu®plan to	outcomes to date2

use it in next 6 months

EU approval expected Q1 2020, JP approval expected Q2 2020

1. Novartis commissioned Retina Specialist Panel Online Survey, Dec 2019. 2. Covered or covered with restrictions

28 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

Ofatumumab (OMB157) is poised to set a new standard for simple, broad and early B-cell therapy adoption

Unsurpassed efficacy and favorable safety supporting broad and early use in RMS

45.9%risk reduction (p<0.001) in 24-week CDW post-hoc analyses with revised definition used in OPERA trials1

[%]	14
rate
12
event	10							TER 10.5%
8
cumulative
6										HR (95% CI): 0.541
4
						OMB 5.6%	(0.381; 0.768)
of	2
K-M estimate
0
	0	3	6	9	12	15	18	21	24	27

• Regulatory file submitted in US and EU
• Easy-to-useautoinjector available upon launch, enabling simple at-homeself-administration and improved patient experience
• Strong scientific presence at major congresses and in markets through 2020
• Engaging with payers on rapid, broad early

Study month

access

CDW - Confirmed Disability Worsening Source: ASCLEPIOS I & II data presentation available here 1. Adapted from the OPERA trials, Hauser et al. 2017. Different to the ASCLEPIOS study, a disability "progression" was defined as an increase from the baseline EDSS score of at least 1.0 point (or 0.5 points if the baseline EDSS score was >5.5) that was sustained for at least 12 (24) weeks, used in OPERA trials

29 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

Inclisiran (KJX839), preparing to launch potentially transformative cholesterol-lowering therapy

Differentiated asset

Efficacy	Potent, durable, consistent LDL-C
	reduction >50%
Safety	Profile similar to placebo (no liver,
	muscle, renal nor platelet signals)
	in entire clinical program
Convenience	Durable efficacy with only 2
	subcutaneous injections per year,
	less patient abandonment
Adherence	Payers confidence reinforced by
	physician administration dosing
	regimen

Preparing for launch

Organizational		MDCO now subsidiary of Novartis, expected to
		be fully integrated end March 2020
Regulatory		US and EU files submitted1
	JP bridging program in progress
	CN local development aligned with CFDA
Commercial		Finalizing hiring and training of US teams
	Value-based pricing and flexible access strategy
	Engaging with payers and health systems to
		enable broad and affordable access, including
		population models (e.g. UK NHS)
		Scaling up supply

1. Regulatory submissions filed by The Medicines Company

30 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

Focus in 2020 will be launches and preparing for next big bets, building on the strong foundation

Maximize growth drivers

Cosentyx®: nr-axSpA launch doubles addressable axSpA population

Entresto®: NRDL-driven expansion in CN, JP approval expected

Deliver on new launches

Beovu®: Global rollout and DME / RVO expansion

Ofatumumab: Broad and early access in CoEs and community

Inclisiran: Broad & affordable access

Mayzent®: Urgency to treat and patient services optimization

Xiidra®:Return to growth

Prepare for next big bets

TQJ230: Lp(a) awareness and testing

Iscalimab: Educate on unmet need, leveraging transplant legacy

Ligelizumab: Build US infrastructure

Tropifexor: Drive disease awareness given asymptomatic nature of NASH

LNP023: Educate physicians on complement-driven renal & hematologic diseases

31 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

32 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

Strong Oncology growth driven by recent launches and growth drivers

Oncology net sales

USD billion, growth in cc

				+9%					+10%
								14.4
				13.4
				1.3
12.3
	0.5
	0.1			3.3		3.9
2.5
9.7		9.6		9.2

201720182019

Key growth drivers1Recent launches2Established products3

Potential future growth

• Strong uptake of recent launches
• Growth drivers deliverdouble-digit performance
• Resource allocation/productivity to fuel strategic investment (i.e. launches, China)

• Generic impact4
• Healthcare cost containment / pricing

1. Including Promacta®/Revolade®, Jakavi®(marketed by Novartis ex-USA), Tafinlar®+ Mekinist®	2. Including Kisqali®, Kymriah®, Lutathera®, Piqray®, Adakveo®3. All other brands, including those with generic competition in the market
4. Afinitor®, Exjade®, Glivec®and Sandostatin®LAR in EU

33 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

Accelerated sales and innovation reinforce the long-term growth of our breast cancer portfolio

2019 sales in USD m

43

6

67

~40% of HR+/HER2- breast cancer patients have a PIK3CA mutation,

associated with poor prognosis

NCCN guidelines updated, PIK3CA testing rate ~25% FY 2019

Foundation Medicine PIK3CA CDx tissue approved Q4 2019, plasma

anticipated Q2 2020

"EPIK" development program for 5 new indications1with potential

Q2

Q3

Q4

155

91111123

Q1

Q2

Q3

Q4

opportunity to serve an additional ~100k patients

Only CDK 4/6 inhibitor that consistently demonstrated superior OS in 2

pivotal Phase 3 studies in 2019

Early signs of OS accelerating US growth momentum in Q4

Strong growth across key geographies ex-US in Q4 2019

Potential registration for high and intermediate adjuvant BC as early as 2022

based pre-planned interim analysis (NATALEE, 3-year treatment duration)

1. TNBC, HER2+ aBC, ovarian cancer, HNSCC; alpelisib PROS

34 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

Adakveo®is off to a solid start in US (approved Nov)

SCD Patients in US

60% are 16yr old +

90% have >1 VOC +

100K

60K

54K

• The only approved therapy for reduction in frequency of VOCs in SCD
• Commercial product available within 2 days of approval
• Payer engagement ongoing:
• • Community centers driving initial uptake
  • Permanent J CODE anticipated July 2020
  • Medicaid coverage criteria approved in Florida, Kentucky, Maryland, Washington, Delaware and Alabama

VOCs - Vaso-occlusive crises SCD - Sickle cell disease

35 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

Maximizing momentum for our growth drivers and new launches, while preparing for next big bets

Maximize growth drivers

Kisqali®: Continued strong growth driven by M3 and M7 OS data

Lutathera®: Leverage potential to grow in earlier lines of treatment

Promacta®/Revolade®: Growth in ITP and

uptake in 1L SAA in the US and Japan

Deliver on new launches

Piqray®: Maximize US launch momentum and expand further to EU markets

Adakveo®: Enable access in larger US accounts and continue education and patient activation

Kymriah®: Drive further capacity increase to meet strong demand in pALL and

DLBCL

Prepare for next big bets

Capmatinib:Establish awareness of

MET's role in NSCLC among HCPs /

patient community

Spartalizumab (PDR001) combo: Build

on Taf/Mek leadership position in metastatic melanoma

177Lu-PSMA: Further evolve commercial infrastructure for best in class launch

Canakinumab: Focus on medical education ontumor-promotinginflammation

36 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

37 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

2019 financial results in line with upgraded guidance

Group full year guidance(as revised in October 2019)		FY 2019 vs. PY
in cc		in cc

"Salesexpected to grow high single digit"	9%
"Core operating incomeexpected to grow mid to high teens"	17%





38 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

Strong sales growth drove double digit increases in core operating income and free cash flow

Continuing operations1	Q4	Change vs. PY
USD million	2019	% USD	% cc2
Net Sales	12,403	8	9
Core Operating income 2	3,462	11	13
Operating income	1,823	34	37
Net Income	1,129	-7	-6
Core EPS (USD)2	1.32	14	15
EPS (USD)	0.50	-6	-4
Free Cash Flow 2	3,488	20

FY	Change vs. PY
2019	% USD	% cc2
47,445	6	9
14,112	12	17
9,086	8	14
7,147	-44	-41
5.28	12	17
3.12	-43	-40
12,937	15

1. Continuing operations excludes Alcon and includes the Sandoz US dermatology and oral solids portfolio business
2. Constant currencies (cc), core results and free cash flow arenon-IFRS measures. An explanation of non-IFRS measures can be found on page 58 of the Condensed Financial Report

39 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

2019 free cash flow increased to USD 12.9bn driven by higher operating income

Continuing operations1free cash flow2

USD billion

		+15%		Key drivers vs. PY:
			12.9	+Higher operating income
11.3	(adjusted for non-cash items)

Offsetting one-time effects:

+

−

Real estate divestment proceeds

Prior year OTC JV dividend and GSK milestone income

20182019

1. Continuing operations excludes Alcon and includes the Sandoz US dermatology and oral solids portfolio business. 2. Free cash flow is a non-IFRS measures. An explanation of non-IFRS measures can be found on page 58 of the Condensed Financial Report

40 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

Novartis proposes 23rdconsecutive dividend increase to the AGM: 2.95 CHF / share1

							2019 dividend yield 3.2%
							2019 dividend growth 3.5%3
3.0				CHF			2.85CHF 2.84USD	2.95CHF	3.04USD
2.5					USD
2.0

1.5

1.0

0.5

0.0

1996 1997 1998 1999 2000 2001 2002 2003 2004 2005 2006 2007 2008 2009 2010 2011 2012 2013 2014 2015 2016 2017 2018 2019

1. Proposal to shareholders at the 2020 Annual General Meeting, taking place on February 28, 2020 2. Converted at historic exchange rates at the dividend payment dates as per Bloomberg; assumes an exchange rate of USD/CHF of 0.9690 as of December 31, 2019 for 2019 3. In CHF

41 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

Core margin expansion of +1.9%pts

Continuing operations

	Q4 2019			FY 2019
					Core operating
		Core margin 2		Net sales	income 2		Core margin 2
	Core margin 2	change vs. PY		change vs. PY	change vs. PY	Core margin2	change vs. PY
	(%)	(%pts cc)2		(in % cc)	(in % cc) 2	(%)	(%pts cc)2
Innovative Medicines	31.5	0.7	11	18	33.5	1.8
Sandoz	20.8	1.5	2	10	21.5	1.5
Continuing Operations1	27.9	0.8		9	17	29.7	1.9

1. Continuing operations excludes Alcon and includes the Sandoz US dermatology and oral solids portfolio business. 2. Core results, constant currencies and free cash flow are non-IFRS measures. An explanation of non-IFRS measures can be found on page 58 of the Condensed Financial Report

42 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

2020 Novartis full year guidance

Barring unforeseen events; growth vs. PY in cc

Focused medicines company | full year guidance

Excl. Sandoz US oral solids & dermatology businesses1

Sales expected to grow mid to high single digit

• IM Division expected to growmid to high single digit
• Sandoz expected to growlow single digit

Core operating income expected to grow high single to low double digit

Key assumption: Guidance above includes the forecast assumption that no Gilenya®or Sandostatin®LAR generics enter in 2020 in US

1. The announced sale of Sandoz US dermatology and oral solids portfolio to Aurobindo, expected to close during Q1 2020. 2019 FY sales and core operating of the Sandoz US oral solids and dermatology businesses were approximately USD 1.1bn and 0.3bn, respectively.

43 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

FY 2020 Guidance on other financial KPIs

Barring unforeseen events (in cc)

Focused medicines company | full year guidance

Excl. Sandoz proposed US portfolio sale to Aurobindo1from both 2018 and 2019

Core net	Expenses expected to increase by around 0.2bn vs. 2019 reflecting
financial result	additional financing costs to acquire The Medicines Company
Core tax rate	FY core tax rate expected to be broadly in line with 2019

1. The announced sale of Sandoz US dermatology and oral solids portfolio to Aurobindo, expected to close during Q1 2020. 2019 FY sales and core operating of the Sandoz US oral solids and dermatology businesses were approximately USD 1.1bn and 0.3bn, respectively.

44 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

45 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

Delivered strong performance in 2019

Continuing operations1, FY 2019, TSR as of YE 2019

47.4 bn	+9%
NET SALES (USD)	vs. 2018(cc2)
14.1 bn	+17%
CORE OPERATINGINCOME2(USD)	vs. 2018(cc2)
12.9 bn	+15%
FREE CASH FLOW2(USD)	vs. 2018(USD)

33.5%	+1.8% pts
IM CORE MARGIN2(%)	vs. 2018(cc2)
5.28	+17%
CORE EPS2(USD)	vs. 2018(cc2)
22.3%	Top tier
1-YEAR TSR3(%)	RANKING 3

1. Continuing operations as defined on page 45 of the Condensed Financial Report, excludes Alcon, includes the businesses of Innovative Medicines and Sandoz (including the US generic oral solids and dermatology portfolio), as well as the continuing corporate functions. 2. Constant currencies (cc), core results and free cash flow are non-IFRS measures. An explanation of non-IFRS measures can be found on page 58 of the Condensed Financial Report. 3. TSR in USD from Jan 1st2019, using 1 day average price at start and 3 month average price at end; ranking when compared to the global HC peer group as defined in the Novartis 2019 Annual Report

46 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

In-market growth drivers, major launches and a rich pipeline expected to sustain our long-term growth

• 15ongoing / upcoming major launches
• 80+major submissions planned to 2022
• 50+late stage programs1

Major launches

In-market growth drivers

Novel assets

Inclisiran	MBG453
TQJ230	Asciminib
LNP023	Canakinumab
Iscalimab	Capmatinib
Ligelizumab	Spartalizumab
AD portfolio2	177Lu-PSMA-617
LNA043
Tropifexor
Ofatumumab
UNR844
QVM / QMF149

New indications

Cosentyx®HS	Alpelisib PROS
Cosentyx®GCA	Piqray®TNBC
Cosentyx®LP	Piqray®HER2+ aBC
Cosentyx®JIA	Piqray®ovarian cancer
Cosentyx®LN	Piqray®HNSCC
Entresto®post-AMI	Kisqali®HR+/HER2- BC (adj)
Beovu®DME	Kymriah®FL
Beovu®RVO
Beovu®DR
Beovu®PDR
Ofatumumab pediatric
AVXS-101 IT

S E L E C T E X A M P L ES

1. Ph3 / in registration 2. AD portfolio - atopic dermatitis portfolio incl. ZPL389, CEE321

47 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

Q&A session

Vas Narasimhan	John Tsai
Chief Executive Officer	Head of Global Drug Development and CMO
Harry Kirsch	Richard Saynor
Chief Financial Officer	CEO, Sandoz
Marie-France Tschudin	Shannon Thyme Klinger
President, Novartis Pharmaceuticals	Group General Counsel
Susanne Schaffert
President, Novartis Oncology

48 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

Appendix

Sales performance driven by Innovative Medicines

Key growth drivers Q4

	Sales	Growth vs. PY	Growth vs. PY
	USD Million	USD Million	cc
	518	200	65%
	186	186	nm
	965	159	21%
	155	95	166%
	90	90	nm
	96	68	nm
	67	67	nm
	380	50	16%
	356	43	15%
	293	37	17%
	303	35	16%
Lutathera®	107	26	31%
	178	23	16%
nm - not meaningful

50 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

Expected currency impact for full year 2020

Currency impact vs. PY

%pts, assuming late-January exchange rates prevail in 2020

FX impact on Net sales

-1					0 to -1
-3	-1 to -2
Q4	FY		Q1	FY
	2019	2020

FX impact on Core operating income

-2				-1 to -2
	-3
	-5
Q4	FY	Q1			FY
	2019		2020

Actual Simulation

51 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

Net debt slightly reduced by USD 0.3bn in 2019 mainly driven by strong FCF

+0.3

-16.2									0.2	-15.9
					2.9
	-6.6
	-3.8
			-5.3	12.9
Dec 31, 2018	Dividends	M&A transactions Treasury share	Free Cash Flow1Net debt Alcon2	Others	Dec 31, 2019

transactions, net

1. Continuing operations excludes Alcon, includes the businesses of Innovative Medicines and Sandoz (including the US generic oral solids and dermatology portfolio), as well as the continuing corporate functions. 2. Includes the net de- recognition of USD 0.6bn cash and cash equivalents and USD 3.5bn of financial debts related to the Alcon spin-off.

52 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

2019 pipeline milestones

	H1 2019				H2 2019	✓Achieved*	✕Missed*
Regulatory	Mayzent®1	SPMS (US)	✓		BYL719 (Piqray®)		HR+ Breast Cancer (US)	✓1
Kymriah®	Ped / Young Adult r/r ALL (JP)	✓		Beovu®		nAMD (US)	✓
decisions and
Kymriah®	r/r DLBCL (JP)	✓		Lucentis®		RoP (EU / JP)	✓
opinions
Promacta®	Severe aplastic anaemia (EU)	✕		Lucentis®		Diabetic Retinopathy (EU)	✓
	Zolgensma®IV	SMA (US)	✓		Mayzent®		SPMS (EU / JP)	✓2
	Zolgensma®IV	SMA (EU / JP)	EU Q1 2020	Xolair®		Pollinosis (JP)	✓
	JP H1 2020
Major	Brolucizumab (RTH258)	nAMD (US / EU / JP)	✓		Cosentyx®		nr-axSpA (US / EU / JP)	✓
Crizanlizumab (SEG101)	Sickle Cell Disease (US / EU)	✓		Entresto®		HF-rEF (JP)	✓
expected
Mayzent®1	SPMS (JP)	✓		Entresto®		HF-pEF (US / EU)	Q1 2020
submissions
				Capmatinib (INC280)	NSCLC (US / JP)	✓
					Ofatumumab (OMB157)	Relapsing MS (US / EU)	✓3
					PDR001 (combination	Metastatic Melanoma (US / EU)	H2 2020
					with Tafinlar®+Mekinist®)
					QVM / QMF 149		Asthma (EU / JP)	✓
Major	AVXS-101 IT	SMA	✓		Cosentyx®		nr-axSpA		✓
Zolgensma®IV	SMA presymptomatic	✓		Entresto®		HF-pEF		(✓)4
expected trial
				Fevipiprant (QAW039)	Asthma		(✓)5
readouts
				Ofatumumab (OMB157)	Relapsing MS	✓
					PDR001 (combination	Metastatic melanoma	H2 2020
					with Tafinlar®+ Mekinist®)
*Represents achieving on-time readout of the data, irrespective of trial outcome 1. Piqray®FDA approval achieved in H1 2019.	2. Positive CHMP opinion Nov 2019, EMA approval Jan 2020	3. EU submission early January 2020 4. Study

narrowly missed primary endpoint, but showed benefit in pre-specified large subgroups including women and patients with lower ejection fraction 5. LUSTER 1&2 readouts completed and did not meet the clinically relevant threshold for reduction in rate of moderate-to-severe exacerbation.

53 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

2020 expected pipeline milestones

H1 2020	H2 2020	✓Achieved	✕Missed

Regulatory decisions and opinions

Major expected submissions

Major expected trial readouts*

Beovu®	nAMD (EU/JP)		Adakveo®	Sickle cell disease (EU)
Cosentyx®	nr-axSpA (EU/US)		Capmatinib (INC280)	NSCLC (US/JP)
Cosentyx®	AS (CN)		Cosentyx®	Pediatric psoriasis (EU)
Ofatumumab (OMB157)	Relapsing MS (US)		Cosentyx®	nr-axSpA (JP)
Piqray®	HR+/HER2- aBC with PIK3CA		Entresto®	HFpEF (US)
mutation (EU)
QVM149	Asthma (EU/JP)		Inclisiran (KJX839)	Hyperlipidemia (US)
Tafinlar®& Mekinist®	Adjuvant melanoma (CN)		Xolair®	Nasal Polyposis (US/EU)
Xiidra®	DED (EU)
Zolgensma®IV	SMA (JP/EU)
Entresto®	HFpEF (US)		Alpelisib (BYL719)	PROS (US)
Inclisiran (KJX839)	Hyperlipidemia (EU)	✓	AVXS-101 IT	SMA (US)
			Cosentyx®	Juvenile PsA / enthesitis-related arthritis
			(US/EU)
			Spartalizumab (PDR001)	Metastatic melanoma (US/EU)
			and Tafinlar®& Mekinist®
			177Lu-PSMA-617	mCRPC (US)
Entresto®	Post-acute MI1		Asciminib (ABL001)	CML 3L
Tropifexor (LJN452)	NASH		Beovu®	DME
UNR844	Presbyopia		Jakavi®	chronic GVHD
			Kisqali®	aBC (MONALEESA-2 OS)
			177Lu-PSMA-617	mCRPC

*Achieved = on-time readout of data, irrespective of trial outcome. 1. Interim analysis readout

54 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

Capitalizing on the rich pipeline, faster and broader access in China

NDA Approval

NRDL Access

FY 2019 sales:

2019

2020

USD 2.2bn

Achieved

Planned

Cosentyx®

Tasigna®

Cosentyx®

Pataday®

Zykadia®

PsO

Pediatric CML

AS

Allergic Conj.

ALK+NSCLC 1stline

Gilenya®

Tafinlar®+Mekinist®

Mayzent®

Tafinlar®+Mekinist®

MS

BRAF+ mM

RMS

Adj. Melanoma

Afinitor ®

Exelon®Patch

Lucentis®

Vigamox®

Cosentyx®

Tafinlar®+Mekinist®

Votrient®

TSC-SEGA and

AD

DME/RVO/CNV

Bacterial Conj.

PsO

BRAF+ mM

aRCC

GI/LUNG NET

Entresto®

Exjade®

Revolade®

Xolair®

Gilenya®

Tasigna®

Zykadia®

Chronic HF

IOL

ITP

Asthma

MS

Adult CML

ALK+NSCLC 2ndline

Eucreas®

Jakavi®

Ultibro®

Sandostatin®LAR

Tasigna®

Zykadia®

T2D

Myelofibrosis

COPD

Acromegaly and

Pediatric CML

ALK+NSCLC 1stline

GEP NET

55 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

Building depth across our core therapeutic areas…

O N C O L O GY

Select
commercial
assets
		Spartalizumab combo
		Metastatic Melanoma
		177Lu-PSMA-617
		mCRPC
Select		Canakinumab (ACZ885)
	Lung
pipeline
Asciminib (ABL001)
assets		CML
		MBG453
		MDS, AML

P H A R M A C E UTI C A LS

CRM		IHD		Neuroscience			Ophthalmology		Respiratory
					1
					2
LNP023		LNA043		Ofatumumab (OMB157)			UNR844		QVM149
Renal diseases		Primary Osteoarthritis		MS			Presbyopia		Asthma
TQJ230		Iscalimab (CFZ533)		LMI070			ECF843		CSJ117
CVRR		Transplant / Sjögren's		SMA			Dry Eye		Asthma
Inclisiran (KJX839)		Ligelizumab (QGE031)					SAF312		QBW251
Hyperlipidemia		CSU / CIU					Chronic Ocular Pain		COPD

Adriforant (ZPL389)

AD

Tropifexor (LJN452)

NASH

LOU064

CSU

CRM - Cardiovascular, Renal & Metabolism IHD - Immunology, Hepatology & Dermatology 1. Aimovig® is developed in collaboration with Amgen 2. Luxturna®marketed ex-US

56 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

...while strengthening our innovative platforms

G E NE THE RA P Y

CE L L THE RA P Y

RA DI O - L IG AND THE RA P Y

Select

1

commercial

assets

Zolgensma®

CPK850

CD19 CAR-T

CD19 CAR-T

177Lu-PSMA-617

177Lu-PSMA-R2

SMA

Retinis Pigmentosa

DLBCL in 1st relapse

r/r Follicular Lymphoma

mCRPC

Prostate Cancer

AVXS-101 IT

CD19 CAR-T

CD19 CAR-T

177Lu-NeoB

SMA

r/r DLBCL in combo with pembro

Adult r/r ALL

Various cancers

AVXS-201

CD19 CAR-T

CD19 CAR-T

Select

Rett Syndrome

r/r CLL in combo with ibrutinib

Pediatric NHL

AVXS-301

CD19 CAR-T

CD19 CAR-T

pipeline

1st line high risk pediatric and

ALS

r/r DLBCL in combo with ibrutinib

assets

young adult ALL

AVXS-401

CD19 CAR-T

CD19 CAR-T

Friedrieich's Ataxia

BCMA&CD19

CD22&CD19

AVXS-501

CD19 CAR-T

CD19 CAR-T

Undisclosed

CD123

EGFRv3

AVXS-601

Undisclosed

1. Luxturna® marketed ex-US

Includes preclinical and launched programs

57 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

Our pipeline projects at a glance

	Phase 1/2	Phase 3	Registration	Total
O N C O LO G Y	57	20	3	80
P H A R MA C E U T IC A LS	57	17	10	84
Cardiovascular, Renal, Metabolism	9	5	1	15
Immunology, Hepatology, Dermatology	22	6	2	30
Neuroscience	9	0	2	11
Ophthalmology	5	3	1	9
Respiratory	7	3	3	13
Global Health	5	0	1	6
B IO S IMILA R S	0	1	0	1
Total	114	38	13	165

58 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

Novartis submission schedule

New molecular entity: lead and new indications

2020

2021

2022

2023

≥2024

TQJ230

spartalizumab

Lead

asciminib

Lead

ECF843

Lead

LOU064

Lead

177Lu-NeoB

Lead

LNA043

Lead

CPK850

Lead

PDR001

ABL001

Dry eye

Chronic spontaneous urticaria

177Lu-NeoB

Osteoarthritis

RP

CVRR-Lp(a)

m BRAF V600+ melanoma

(+Taf/Mek)

CML 3L

Multiple Solid Tumors

ganaplacide

177Lu-PSMA-617

Lead

MBG453

Lead

iscalimab

Lead

VPM087

Lead

tropifexor

Lead

AVXS-201

Lead

INDICATIONS

177Lu-PSMA-617

HR-MDS

CFZ533

1st line CRC / 1st line RCC

LJN452

OAV201

KAF156

mCRPC

Renal/Liver Tx

NASH

Rett syndrome

Malaria

ligelizumab

Lead

LNP023

Lead

adriforant

Lead

tropifexor&cenicriviroc

Lead

LMI070

Lead

cipargamin

QGE031

PNH

ZPL389

LJC242

SMA

KAE609

Chronic urticaria

Atopic dermatitis

NASH

Malaria

CSJ117

Lead

CEE321

Lead

SAF312

Lead

MIJ821

Lead

LXE408

Severe asthma

Atopic Dermatitis

COSP

Depression

Visceral leishmaniasis

LEAD

denosumab

BioS

ianalumab

Lead

UNR844

Lead

QBW251

Lead

GP2411

VAY736

Presbyopia

COPD

anti RANKL mAb

AIH

canakinumab

LCM

canakinumab

LCM

spartalizumab

LCM

capmatinib

LCM

MBG453

LCM

LOU064

LCM

LNP023

INDICATIONS

ACZ885

ACZ885

PDR001

INC280

LCM

Unfit AML

SjS

iMN

MBG453

NSCLC 2L

Adjuvant NSCLC

Malignant melanoma (combo)

Solid tumors

canakinumab

LCM

LNP023

LCM

crizanlizumab

LCM

iscalimab

LCM

tropifexor

LCM

inclisiran

ACZ885

C3G

SEG101

CFZ533

LJN452

LNP023

NSCLC 1L

Sickle cell anaemia w ith crisis

SjS

NASH (combos)

Hyperlipidemia

crizanlizumab

LCM

LNP023

LCM

MBG453

LCM

ianalumab

LCM

ofatumumab

LCM

cipargamin

SEG101

IgAN

Fit AML

VAY736

OMB157

KAE609

Sickle cell anaemia new formulations

pSjS

Ped MS

Malaria

NEW

AML

Compared to past reports, we have categorized submission schedules into NMEs (lead & new indications) and supplementary indications for existing brands

59 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

Lead

Lead

Lead

Lead

LCM

LCM

LCM

Novartis submission schedule

Supplementary indications for existing brands

2020			2021
alpelisib, BYL719	LCM	Kymriah
PROS		tisagenlecleucel-T, CTL019
		r/r DLBCL 1st relapse
Cosentyx	LCM	Kymriah
secukinumab, AIN457		tisagenlecleucel-T, CTL019
Psoriasis 300mg AI		r/r Follicular lymphoma
Cosentyx	LCM	Tafinlar
secukinumab, AIN457		dabrafenib, DRB436
PsA H2H		Neoplasm Pedia
Cosentyx US	LCM	Promacta
secukinumab, AIN457		eltrombopag,	ETB115
Ped Psoriasis		Food effect free formulation
AVXS-101	LCM	Jakavi
onasemno-geneabepar-vovec, OAV101	ruxolitinib, INC424
SMA IT		Chronic GVHD
Xolair	LCM	Jakavi
omalizumab, IGE025		ruxolitinib, INC424
CSU (for CN)		Acute GVHD
Entresto	LCM	Beovu
valsartan+sacubitril, LCZ696		brolucizumab, RTH258
HFpEF		DME
		Xolair
		omalizumab,	IGE025
		Food allergy
		Xolair
		omalizumab,	IGE025
		Auto-injector

Entresto

valsartan+sacubitril, LCZ696 Post-AMI

Lamprene

clofazimine, LAM320 Tuberculosis

2022

LCMKisqali

ribociclib, LEE011 HR+/HER2- BC (adj)

LCMPromacta

eltrombopag, ETB115 Radiation sickness syndrome

LCMCosentyx

secukinumab, AIN457 SpA IVIV

LCMCosentyx

secukinumab, AIN457 Hidradenitis suppurativa

LCMCosentyx

secukinumab, AIN457 AS H2H

LCMEntresto EUa

valsartan+sacubitril, LCZ696 Pediatric HF

LCM

LCM

LCM

LCM

LCM

2023

LCMPiqray

alpelisib, BYL719

TNBC

LCMPiqray

alpelisib, BYL719 HER2+ adv BC

LCMPiqray

alpelisib, BYL719 Ovarian cancer

LCMKymriah

tisagenlecleucel-T, CTL019 Adult r/r ALL

LCMTafinlar

dabrafenib, DRB436 Tyroid cancer

LCMBeovu

brolucizumab, RTH258 Diabetic retinopathy

Beovu

brolucizumab, RTH258

RVO

LCMPiqray

alpelisib, BYL719 HNSCC 2/3L

LCMJakavi

ruxolitinib, INC424 Pediatrics Acute GVHD

LCM177Lu-PSMA-R2

177Lu-PSMA-R2 Prostate cancer

LCM

LCM

LCM

LCM

				≥2024
				Jakavi
LCM		Kymriah	LCM
			tisagenlecleucel-T, CTL019		ruxolitinib, INC424
			1L high risk ALL, pediatrics & young adults	Pediatrics Chronic GVHD
					Cosentyx
LCM		Kymriah	LCM
			tisagenlecleucel-T, CTL019		secukinumab, AIN457
			r/r DLBCL (+ pembro)		GCA
LCM		Jakavi	LCM	Cosentyx
			ruxolitinib, INC424		secukinumab, AIN457
			Myelofibrosis (combination)		Lichen Planus

LCM		Cosentyx	LCM
		secukinumab, AIN457
		Lupus Nephritis
LCM		Cosentyx	LCM
		secukinumab, AIN457
		Ankylosing spondylitis
LCM		Mayzent	LCM
		siponimod, BAF312
		Ped MS

a) Approved in US

Compared to past reports, we have categorized submission schedules into NMEs (lead & new indications) and supplementary indications for existing brands

60 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

Novartis pipeline in registration

6 lead indications

Lead indication

Oncology

Code	Name	M echanism	Indication(s)
BYL719	Piqray	PI3Kα inhibitor	PIK3CA mutant HR+, HER2 (-) postmenopausal adv BC5 2nd line
(+fulv)
INC280	capmatinib	Met Inhibitor	NSCLC
SEG101	Adakveo®	P-selectin Inhibitor	Sickle cell disease

Immunology, Hepatology, Dermatology

Code	Name	M echanism	Indication(s)
AIN457	Cosentyx	IL17A Inhibitor	Ped Psoriasis	nr-axSpA

Ophthalmology

Code	Name	Mechanism	Indication(s)
RTH258	Beovu	VEGF Inhibitor	nAMD

Neuroscience

Code	Name	M echanism	Indication(s)
OAV101	Zolgensma®	Gene therapy	SMA IV
OMB157	ofatumumab	CD20 Antagonist	r MS

Respiratory Disease

Code	Name	M echanism	Indication(s)
IGE025	Xolair	IgE Inhibitor	Nasal polyps
QMF149	Indacaterol acetate	Long acting β2-adrenergic	Asthma
	+mometasone furoate	agonist + inhaled corticosteroid
QVM149	Indacaterol acetate	Long acting β2-adrenergic	Asthma
	+mometasone fuorate	agonist + long-acting muscarinic
	+glycopyrrnium bromide	antagonist + inhaled
		corticosteroid

Cardiovascular, Renal, Metabolism

Code	Name	Mechanism	Indication(s)
KJX839	inclisiran	siRNA (PCSK9)	Hyperlipidemia

Global Health

Code	Name	Mechanism	Indication(s)
LAM320	Lamprene®	SMPD1 Inhibitor	Tuberculosis

61 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

Novartis pipeline in Phase 3

5 lead indications

Lead indication

Oncology

Code	Name	Mechanism	Indication(s)
177Lu-PSMA-617	177Lu-PSMA-617	Targeted Radioligand Therapy	mCRPC
ABL001	asciminib	BCR-ABL Inhibitor	CML 3L
ACZ885	canakinumab	IL-1b Inhibitor	NSCLC 1L	NSCLC 2L	Adjuvant
NSCLC

BYL719	Piqray®	PI3Kα inhibitor	HER2+ adv BC	TNBC	HNSCC 2/3L	Ovarian cancer
CTL019	Kymriah	CD19 CART	r/r Follicular	1L high risk	r/r DLBCL 1st	Adult r/r ALL
			lymphoma	ALL, pediatrics	relapse
				and young
				adults
ETB115	Promacta®	Thrombopoietin receptor (TPO-R) Radiation sickness syndrome	Food effect free formulation
		Agonist
INC424	Jakavi	JAK1/2 Inhibitor	Acute GVHD	Chronic GVHD
LEE011	Kisqali®	CDK4 Inhibitor	HR+/HER2- BC (adj)
PDR001	Spartalizumab	PD1 Inhibitor	m BRAF V600+ melanoma (+Taf/Mek)
SEG101	crizanlizumab	P-selectin Inhibitor	Sickle cell anemia new formulation

Respiratory Disease

Code	Name	Mechanism	Indication(s)
IGE025	Xolair®	IgE Inhibitor	CSU (for CN)	Auto-injector	Food allergy

Cardiovascular, Renal, Metabolism

Code	Name	Mechanism	Indication(s)
KJX839	inclisiran	siRNA (PCSK9)	Hyperlipidemia
LCZ696	Entresto®	AT-II / NEP,NEP,AGTR1,AGTR2 Inhibitor	Post-AMI	HFpEF	Pediatric HFa
TQJ230	TQJ230	Anti-Apo(a) ASO targeting Lp(a)	CVRR-Lp(a)

Biosimilars

Code	Name	Mechanism	Indication(s)
GP2411	denosumab	anti RANKL mAb	Denosumab BioS

Immunology, Hepatology, Dermatology

Code	Name	Mechanism	Indication(s)
AIN457	Cosentyx	IL17A Inhibitor	Lupus	Psoriasis	Hidradenitis	AS H2H	PsA H2H
			Nephritis	300mg AI	suppurativa
QGE031	ligelizumab	IgE Inhibitor	Chronic spontaneous urticaria

Ophthalmology

Code	Name	Mechanism	Indication(s)
RTH258	Beovu®	VEGF Inhibitor	Diabetic retinopathy	RVO	DME

a) Approved in US

62 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

Novartis pipeline in Phase 2

27 lead indications

Lead indication

Oncology		Neuroscience

Code	Name	M echanism	Indication(s)
ACZ885	canakinumab	IL-1b Inhibitor	Sickle cell anaemia
BYL719	alpelisib	PI3Kα inhibitor	PROS
CTL019	Kymriah	CD19 CART	r/r DLBCL (+ pembro)
EGF816	nazartinib+capmatinib Opdivo EGFR Inhibitor	NSCLC
INC280	capmatinib	Met Inhibitor	Solid tumors
		Met Inhibitor + spartalizumab	HCC	NSCLC
INC424	Jakavi®	JAK1/2 Inhibitor	Myelofibrosis (combination)
LAG525	LAG525	LAG3 Inhibitor	Solid Tumors
MBG453	MBG453	TIM3 Antagonist	HR-MDS	Unfit AML	AML	Fit AML
NIR178	NIR178, spartalizumab	Ad2AR Inhibitor, PD1 Inhibitor	Cancers
PDR001	spartalizumab	PD1 Inhibitor	Nasopharyngeal cancer	Metastatic melanoma (combo)
SEG101	crizanlizumab	P-selectin Inhibitor	Ped sickle cell anaemia with
			crisis

Immunology, Hepatology, Dermatology

Code	Name	Mechanism	Indication(s)
AIN457	Cosentyx®	IL17A Inhibitor	SpA IVIV		GCA	Lichen Planus
CFZ533	iscalimab	CD40 Inhibitor	Renal/Liver Tx		SjS	HS	T1DM
LJC242	tropifexor&cenicriviroc	CCR2 Inhibitor, FXR agonist	NASH
LJN452	tropifexor	FXR agonist	NASH		Nash (combos)
LNA043	LNA043	ANGPTL3 Agonist	Osteoarthritis
LOU064	LOU064	BTK Inhibitor	Chronic spontaneous urticaria	SjS
LYS006	LYS006	Anti-inflammatory	Acne
VAY736	ianalumab	BAFF-R Inhibitor	AIH		pSjS	SLE
ZPL389	adriforant	HRH4 Antagonist	Atopic dermatitis

Ophthalmology

Code	Name	Mechanism	Indication(s)
CPK850	CPK850	RLBP1 AAV	RP
ECF843	ECF843	rh-Lubricin	Dry eye
LKA651	LKA651	EPO Inhibitor	DME
SAF312	SAF312	TRPV1 Antagonist	COSP
UNR844	UNR844	disulfide bonds Modulator	Presbyopia

Code	Name	Mechanism	Indication(s)
AFQ056	AFQ056	mGluR5 Antagonist	Addiction
BAF312	Mayzent®	S1P1 Modulator	Ped MS	Stroke
BLZ945	BLZ945	CSF-1 Inhibitor	ALS
LMI070	branaplam	Survival motor neuron protein	SMA
MIJ821	MIJ821	NR2B Inhibitor	Depression
OMB157	ofatumumab	CD20 Antagonist	Ped MS

Respiratory Disease

Code	Name	Mechanism	Indication(s)
ACZ885	canakinumab	IL-1b Inhibitor	Sarcoidosis
CJM112	CJM112	IL-17A Inhibitor	Asthma
CSJ117	CSJ117	TSLP Inhibitor	Severe asthma
LOU064	LOU064	BTK Inhibitor	Asthma
QBW251	QBW251	CFTR Potentiator	COPD
VAY736	ianalumab	BAFF-R Inhibitor	IPF

Cardiovascular, Renal, Metabolism

Code	Name	Mechanism	Indication(s)
CFZ533	iscalimab	CD40 Inhibitor	Lupus Nephritis
LMB763	nidufexor	FXR Agonist	Diabetic Nephropathy
LNP023	LNP023	CFB Inhibitor	PNH	IgAN	C3G	iMN

Global Health

Code	Name	Mechanism	Indication(s)
KAE609	cipargamin	PfATP4 inhibitor	Malaria	Malaria severe
KAF156	ganaplacide	-	Malaria
LXE408	LXE408	Protozoan Inhibitor	Visceral leishmaniasis

63 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

Novartis pipeline in Phase 1 (1 of 2)

33 lead indications

Lead indication

Oncology

Code	Name	Mechanism	Indication(s)
177Lu-NeoB	177Lu-NeoB	Radioligand therapy target GRPR	Multiple solid tumors
177Lu-PSMA-R2	177Lu-PSMA-R2	Radioligand therapy target PSMA	Prostate cancer
ADPT01	NIR178, LAG525, spartalizumab, canakinumab, capmatinib	LAG3 Inhibitor,PD1 Inhibitor	TNBC
BLZ945	BLZ945 + spartalizumab	CSF-1 Inhibitor + PD1 Inhibitor	Solid tumors
CSJ137	CSJ137	Growth Factor Inhibitor	Anaemia
CTL019	Kymriah®	CD19 CART	Lymphoma
DKY709	DKY709 + spartalizumab	-	Cancers
EGF816	nazartinib + LXH254, ribociclib, capmatinib, Opdivo, Mekinist	EGFR Inhibitor	NSCLC
HDM201	HDM201 + MBG453, venetoclax	MDM2 Inhibitor	Haematological malignancy
JEZ567	JEZ567	CD123 CART	AML
JJO686	JJO686	CD22 CART	ALL
LHC165	LHC165 + spartalizumab	TLR7 Agonist	Solid tumors
LSZ102	LSZ102, ribociclib, alpelisib	SERD	BC
LXF821	LXF821	EGFR CART, PD1 Inhibitor	Glioblastoma multiforme
LXH254	LXH254 (combos)	cRAF Inhibitor	Solid tumors	Solid tumors
MAK683	MAK683	EED Inhibitor	Cancers
MAS825	MAS825	-	Inflammatory diseases
MBG453	MBG453 (combos)	TIM3 Antagonist	Cancers
MCM998	MCM998, LXG250	BCMA CART, CD19 CART	Multiple myeloma
MIK665	MIK665	MCL1 Inhibitor	AML	Haematological malignancy	AML (combo)
NIS793	NIS793, spartalizumab	TGFB1 Inhibitor, PD1 Inhibitor	Solid tumors
NIZ985	NIZ985, spartalizumab	IL-15 Agonist	Solid tumors
NJH395	NJH395	-	Solid tumors
NZV930	NZV930, spartalizumab, NIR178	CD73 Antagonist	Solid tumors
PDR001	spartalizumab, CJM112, LCL161	PD1 Inhibitor, TIM3 Antagonist	AML	Solid tumors (combo)	Solid tumors (combo)	Solid tumors (combo)
SQZ622	SQZ622	CD123xCD3 Modulator	AML
TNO155	TNO155	SHP2 Inhibitor	Solid tumors (single agent)	Solid tumors (combo)
VAY736	ianalumab + ibrutinib	BAFF-R Inhibitor,BTK Inhibitor	Haematological malignancy
VOB560	VOB560	-	Cancers
VPM087	VPM087	IL1B Antagonist	1st line CRC / 1st line RCC
WNT974	WNT974 + spartalizumab	Porcupine Inhibitor	Solid tumors
WVT078	WVT078	-	Multiple myeloma
YTB323	YTB323 + ibrutinib	CD19 CART	Haematological malignancy

64 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

Novartis pipeline in Phase 1 (2 of 2)

34 lead indications

Lead indication

Immunology, Hepatology, Dermatology

Code	Name	M echanism	Indication(s)
DFV890	DFV890	-	Multiple Indications
LRX712	LRX712	-	Osteoarthritis
MHS552	MHS552	-	Autoimmune Indications
MHV370	MHV370	-	SLE

Neuroscience

Code	Name	M echanism	Indication(s)
OAV101	AVXS-101	Survival motor neuron protein	SMA IT1
		gene therapy
OAV201	AVXS-201	MECP2 gene therapy	Rett syndrome

Respiratory Disease

Code	Name	M echanism	Indication(s)
CMK389	CMK389	IL-18 Inhibitor	Sarcoidosis

Cardiovascular, Renal, Metabolism

Code	Name	M echanism	Indication(s)
HSY244	HSY244	-	Atrial fibrillation
LTW980	LTW980	-	Hypertriglyceridemia
MBL949	MBL949	-	Diabetes

Global Health

Code	Name	Mechanism	Indication(s)
KAF156	ganaplacide	-	Malaria prophylaxis

1. FDA placed a partial hold on AVXS-101 intrathecal clinical trials for SMA patients based on findings in a small pre-clinical animal study

65 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

Abbreviations

AIH	Autoimmune hepatitis	IT	Intrathecal formulation
ALL	Acute lymphoblastic leukemia	IV	Intravenous formulation
ALS	Amyotrophic lateral sclerosis	JIA	Juvenile idiopathic arthritis
AMI	Acute myocardial infarction	LP	Lichen planus
AML	Acute myeloid leukemia	LN	Lupus nephritis
AS H2H	Ankylosing spondylitis head-to-head study versus adalimumab	mCRPC	Metastatic castration-resistant prostate cancer
BC	Breast cancer	MDR	Multi-drug resistant
C3G	C3 glomerulopathy	MDS	Myelodysplastic syndrome
CDx	Companion diagnostics	MS	Multiple sclerosis
CCF	Congestive cardiac failure	nAMD	Neovascular (wet) age-related macular degeneration
CLL	Chronic lymphocytic leukemia	NASH	Non-alcoholic steatohepatitis
CML	Chronic myeloid leukemia	nr-axSpA	Non-radiographic axial spondyloarthritis
CRC	Colorectal cancer	NRDL	National reimbursement drug list
COPD	Chronic obstructive pulmonary disease	NSCLC	Non-small cell lung cancer
COSP	Chronic ocular surface pain	PDR	Proliferative diabetic retinopathy
CSU	Chronic spontaneous urticaria	PEF	Preserved ejection fraction
CVRR-Lp(a)	Secondary prevention of cardiovascular events in patients with elevated levels of lipoprotein (a)	PNH	Paroxysmal nocturnal haemoglobinuria
CVRR-LDLC	Secondary prevention of cardiovascular events in patients with elevated levels of LDLC	PsA H2H	Psoriatic arthritis head-to-head study versus adalimumab
DME	Diabetic macular edema	RCC	Renal cell carcinoma
DLBCL	Diffuse large B-cell lymphoma refractory	PROS	PIK3CA related overgrowth spectrum
FL	Follicular lymphoma	RA	Rheumatoid arthritis
GCA	Giant cell arteritis	rMS	Relapsing multiple sclerosis
GVHD	Graft-versus-host disease	ROP	Retinopathy of prematurity
HCC	Hepatocellular carcinoma	RP	Retinitis pigmentosa
HFpEF	Chronic heart failure with preserved ejection fraction	RVO	Retinal vein occlusion
HF-rEF	Chronic heart failure with reduced ejection fraction	SAA	Severe aplastic anemia
HNSCC	Head and neck squamous cell carcinoma	SjS	Sjögren's syndrome
HS	Hidradenitis suppurativa	SLE	Systemic lupus erythematosus
IgAN	IgA nephropathy	SMA	Spinal muscular atrophy type 1 (IV formulation)
iMN	Membranous nephropathy	SpA	Spondyloarthritis
IOL	Iron overload	SPMS	Secondary progressive multiple sclerosis
ITP	Immune thrombocytopenia	TNBC	Triple negative breast cancer
IPF	Idiopathic pulmonary fibrosis	T1DM	Type 1 Diabetes melitus

66 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

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. Q3-2019 presentation

New trials added

Study	Program	Indication	Phase	Patients
NCT04156620 INVIGORATE-1 (CAIN457P12301)	Cosentyx®	Axial spondyloarthritis	Phase 3	500
NCT04065841 ELIVATE (CLJN452D12201C)	LJN452	Non-alcoholic steatohepatitis (NASH)	Phase 2	210
NCT03373461 (CLNP023X2203)	LNP023	IgA nephropathy	Phase 2	146
NCT03439839 (CLNP023X2201)	LNP023	Paroxysmal nocturnal hemoglobinuria	Phase 2	15
NCT03832114 (CLNP023X2202)	LNP023	C3 glomerulopathv	Phase 2	27
NCT03896152 (CLNP023X2204)	LNP023	Paroxysmal nocturnal hemoglobinuria	Phase 2	10
NCT03955445 (CLNP023B12001B)	LNP023	C3 glomerulopathy	Phase 2	27
NCT04154787 (CLNP023D12201)	LNP023	Idiopathic membraneous nephropathy	Phase 2	72
NCT04109313 (CLOU064A2201E1)	LOU064	Chronic spontaneous urticaria (CSU)	Phase 2	250
NCT03988608 (CETB115E2202)	Promacta®/	Previously untreated or relapsed/refractory severe aplastic anemia	Phase 2	20
	Revolade®	or recurrent aplastic anemia
NCT04047472 HOBBY (CRTH258A2307)	RTH258	Macular degeneration	Phase 3	494

Trials removed (operational decision-points achieved)

Study	Program	Indication	Phase	Patients
NCT02059291 CLUSTER (CACZ885N2301)	Ilaris®	Hereditary periodic fevers	Phase 3	203
NCT03578367 ASC4MORE (CABL001E2201)	ABL001	Chronic myeloid leukaemia (CML)	Phase 2	80

68 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

Cardiovascular, Renal and Metabolic

Entresto®- Angiotensin receptor neprilysin inhibitor (ARNI)

Study	NCT02678312 PANORAMA HF (CLCZ696B2319)	NCT03785405 (CLCZ696B2319E1 - extension study)
Indication	Heart failure in pediatric patients	Heart failure in pediatric patients
Phase	Phase 2/3	Phase 3
Patients	360	240
Primary Outcome	Part 1: Pharmacodynamics and pharmacokinetics of	Number of participants with Adverse Events (AEs) and
sacubitril/valsartan LCZ696 analytes
Measures	Serious Adverse Events (SAEs)
Part 2: Efficacy and safety compared with enalapril
	• Part 1: Sacubitril/valsartan 0.8 mg/kg or 3.1 mg/kg or
	both; 0.4 mg/kg or 1.6 mg/kg or both (single doses).
	• Part 2: enalapril/placebo 0.2 mg/kg bid (ped. formulation	• Single arm, open label sacubitril/valsartan (pediatric
Arms/Intervention	1mg/ml) and adult formulation (2.5, 5, 10 mg bid);	formulation granules (12.5, 31.25 mg in capsules); liquid
Sacubitril/valsartan (LCZ696)/placebo: Ped. formulation	formulation (1mg/ml and 4mg/ml concentration) and
	granules (12.5, 31.25 mg in capsules); liquid formulation	adult formulation (50, 100, 200 mg bid))
	(1mg/ml and 4mg/ml concentration) and adult
	formulation (50, 100, 200 mg bid)
	Pediatric patients from 1 month to < 18 years of age with	Pediatric patients with heart failure due to systemic left
Target Patients	heart failure due to systemic left ventricle systolic	ventricle systolic dysfunction who have completed study
	dysfunction	CLCZ696B2319
	H2-2021; (Analysis of 110 pts from Part 2 formed the basis
	for pediatric submission in Apr-2019 and approval by the US
Expected Completion	FDA in Oct-2019 for the treatment of symptomatic HF with	2022
	systemic left ventricular systolic dysfunction in children aged
	1 year and older)
Publication	TBD	TBD

70 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

Entresto®- Angiotensin receptor neprilysin inhibitor (ARNI)

Study	NCT02554890 PIONEER-HF (CLCZ696BUS01)	NCT02661217 TRANSITION (CLCZ696B2401)
Indication	Heart failure, reduced ejection fraction	Heart failure, reduced ejection fraction
Phase	Phase 3B/4	Phase 4
Patients	881	1,002
Primary Outcome	Percentage change from baseline in N-terminalpro-brain	Assessing the percentage of patients who achieve the target
dose of 200 mg bid LCZ696 at 10 weeks after
Measures	natriuretic peptide (NT-proBNP)
randomization
	•	Sacubitril/valsartan (LCZ696) 24/26 mg, 49/51 mg or	•	Pre-discharge treatment initiation - LCZ696 (50, 100,
Arms/Intervention		97/103 mg bid or matching placebo		200 mg bid)
• Enalapril (2.5 mg, 5 mg, and 10 mg) bid or matching	•	Post-discharge treatment initiation - LCZ696 (50, 100,
		placebo		200 mg bid)
Target Patients	Patients with HFrEF (LVEF<40%) hospitalized for ADHF	Heart failure patients with reduced ejection-fraction
and stable for more than 24 hours	hospitalized for an acute decompensation event
Expected Completion	Q3-2018(actual)	Q4-2018(actual)
	• Mar-2019 ACC: 4wk OLE data, and core study data on	•	28-May-2019 TRANSITION primary publication -
		published EJHF
		biomarkers, de novo HF, hospitalizations, & prior
		•	Secondary data presentations: de novo HF and
		exposure
			ACEi/ARB naive sub-groups presented at ESC-HF
Publication	•	Apr-2019 Circulation: Research letter on composite
	Congress in May 2019 and submitted to EJHF in Jun-
		endpoint (Circulation. 2019;139:00-00)
			2019 (expected publication Q3-2019)
	• Q3-2019 ESC: Secondary abstracts submitted
	•	Planned in Q4-2019:26-weeks data presentation at
	•	Planned in Q1-2020: RAAS naive and de novo HF
		ESC-2019 in Paris

71 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

Entresto®- Angiotensin receptor neprilysin inhibitor (ARNI)

Study	NCT02884206 PERSPECTIVE (CLCZ696B2320)	NCT02468232 PARALLEL-HF (CLCZ696B1301)
Indication	Heart failure	Heart failure, reduced ejection fraction
Phase	Phase 3	Phase 3
Patients	592	225
Primary Outcome	Change from baseline in the CogState Global Cognitive	Time to the first occurrence of the composite endpoint -
either cardiovascular (CV) death or heart failure (HF)
Measures	Composite Score (GCCS)
hospitalization
	• Sacubitril/valsartan 50, 100, and 200 mg bid with	•	Sacubitril/valsartan 50 mg, 100 mg, 200 mg bid/placebo
Arms/Intervention	placebo of valsartan		of enalapril
• Valsartan 40, 80, and 160 mg bid tablets with placebo	•	Enalapril 2.5 mg, 5 mg, 10 mg bid / placebo of
	for sacubitril/valsartan		sacubitril/valsartan
Target Patients	Patients with chronic heart failure with preserved ejection	Japanese heart failure patients (NYHA Class II-IV) with
fraction	reduced ejection fraction
Expected Completion	2022	Q1-2019(actual);H1-2021(open-label extension)
Publication	TBD	Planned in H1-2020: Core study primary manuscript in Circ
J

72 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

Entresto®- Angiotensin receptor neprilysin inhibitor (ARNI)

Study	NCT01920711 PARAGON-HF (CLCZ696D2301)	NCT03066804 PARALLAX (CLCZ696D2302)
Indication	Heart failure, preserved ejection fraction	Heart failure, preserved ejection fraction
Phase	Phase 3	Phase 3
Patients	4,822	2,577
Primary Outcome	Cumulative number of primary composite events of	Change in NT-proBNP from baseline to week 12
cardiovascular (CV) death and total (first and recurrent) HF	and change in 6 minute walk distance (6MWD) from
Measures
hospitalizations	baseline to Week 24
			• Sacubitril/valsartan 50 mg, 100 mg and 200 mg bid and
	•	Sacubitril/valsartan or placebo 50 mg, 100 mg, and 200	matching placebo
	• Enalapril 2.5 mg, 5 mg and 10 mg bid and matching
Arms/Intervention		mg bid
	placebo
	•	Valsartan or placebo 40 mg, 80 mg, and 160 mg bid
	• Valsartan 40 mg, 80 mg, 160 mg bid and matching
			placebo
Target Patients	Heart failure patients (NYHA Class II-IV) with preserved	Heart failure patients (NYHA Class II-IV) with preserved
ejection fraction	ejection fraction
Expected Completion	Q3-2019(actual)	Q4-2019(actual)
	• Sep-2019: Primary manuscript published (Angiotensin-
		Neprilysin Inhibition in Heart Failure with Preserved
Publication		Ejection Fraction. Solomon S et al; NEJM. DOI:	TBD
	10.1056/NEJMoa1908655)
	• Sep-2019: ESC: Late breaker presentation of primary
		results

73 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

Entresto®- Angiotensin receptor neprilysin inhibitor (ARNI)

Study	NCT03909295 (CLCZ696D1301E1 - extension study)	NCT02924727 PARADISE-MI (CLCZ696G2301)
Indication	Heart failure chronic	Post-acute myocardial infarction
Phase	Phase 3	Phase 3
Patients	63	5,650
Primary Outcome	Number of participants with Adverse Events (AEs) and	Time to the first occurrence of a confirmed composite
endpoint (cardiovascular (CV) death, heart failure (HF)
Measures	Serious Adverse Events (SAEs)
hospitalization, or outpatient heart failure)
		• Sacubitril/valsartan 50 mg, 100 mg, 200 mg bid / placebo
Arms/Intervention	• Sacubitril/valsartan 50 mg,100 mg,200 mg film coated	of ramipril/valsartan
tablets	• Ramipril 1.25 mg, 2.5 mg, and 5 mg bid / placebo of
		sacubitril/valsartan / placebo for valsartan
	Japanese heart failure patients (NYHA Class II-IV) with	Post-AMI patients with evidence of LV systolic dysfunction
Target Patients	preserved ejection fraction after CLCZ696D2301	and/or pulmonary congestion, with no known prior history of
	(PARAGON-HF)	chronic HF
Expected Completion	Q4-2019(actual)	H1-2021
Publication	TBD	TBD

74 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

LNP023 - Factor B inhibition of the complement alternative pathway

Study	NCT03373461 (CLNP023X2203)	NCT04154787 (CLNP023D12201)
Indication	IgA nephropathy (IgAN)	Idiopathic membranous nephropathy (iMN)
Phase	Phase 2	Phase 2
Patients	146	72

Primary Outcome	Change from baseline of log transformed UPCR derived
Measures	from the 24h urine collections at Baseline and Day 90

Change from baseline of UPCR derived from 24hr urine collections at Baseline and Week 24

Arms/Intervention

Target Patients

Expected Completion

Publication

• Placebo

• LNP023 Dose 1 - 10mg bid	• LNP023 Dose - 200mg bid
• LNP023 Dose 2 - 50mg bid	• LNP023 Dose - 50mg bid
•	LNP023 Dose 3	- 200mg bid	• Rituximab
•	LNP023 Dose 4	- 100mg bid (Part 2 only)
			Patients with biopsy proven iMN who are at high risk of
Patients with biopsy-verified IgA nephropathy	disease progression defined on the basis of antibody anti-
			PLA2R titre and proteinuria
2021		2022
TBD		TBD

75 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

LNP023 - Factor B inhibition of the complement alternative pathway

Study	NCT03832114 (CLNP023X2202)	NCT03955445 (CLNP023B12001B)
Indication	C3 glomerulopathy (C3G)	C3 glomerulopathy (C3G)
Phase	Phase 2	Phase 2 (open-label extension)
Patients	27	27 (from ongoing Phase 2, potential patient from Ph3)

Primary Outcome Measures

Cohort A: Ratio to Baseline of UPCR to Week 12 derived from 24hr urine collection

Cohort B: Change from Baseline in C3 Deposit Score (based on immunofluorescence microscopy) at Week 12

Characterize the effect of LNP023 treatment on a composite renal response endpoint at 9 months (1. a stable or improved eGFR and, 2. a reduction in proteinuria and 3. an increase in C3 compared to the CLNP023X2202 baseline visit)

Arms/Intervention

Target Patients

Expected Completion

Publication

Increasing doses of LNP023 up to 200mg bid:
• Cohort A: Native kidney patients	• Open-label LNP023 200mg bid
• Cohort B: Kidney transplanted patients
Patients with C3 glomerulopathy	Patients with C3 glomerulopathy
2021	2025
TBD	TBD

76 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

LNP023 - Factor B inhibition of the complement alternative pathway

Study	NCT03439839 (CLNP023X2201)	NCT03896152 (CLNP023X2204)
Indication	Paroxysmal nocturnal hemoglobinuria (PNH)	Paroxysmal nocturnal hemoglobinuria (PNH)
Phase	Phase 2	Phase 2
Patients	15	10

Primary Outcome	Reduction of chronic hemolysis, based on LDH level at	Reduction of PNH associated hemolysis, based on
percentage of patients with 60% reduction in LDH or LDH
Measures	Week 13
below upper limit of normal up to 12 weeks of treatment.
	• Cohort 1: 10 patients receiving LNP023 200mg bid, in
	addition to SoC, for 13 weeks with 3yr treatment extension	•	Arm 1: 4wks treatment LNP023 25mg bid followed by
	period	8wk treatment LNP023 100mg bid and 2yr extension
Arms/Intervention	• Cohort 2: 5 patients receiving LNP023 50mg bid, in	LNP023 100mg bid
addition to SoC, for minimum 2 weeks with 3yr treatment	•	Arm 2: 4wks treatment LNP023 50mg bid followed by

extension period. Dose may be increased D15 onwards to 200mg bid if LDH not within limit of normal or reduced by at least 60% compared to Baseline.

8wk treatment LNP023 200mg bid and 2yr extension LNP023 200mg bid

	Patients with PNH, showing signs of active hemolysis	Patients with PNH, showing signs of active hemolysis, not
Target Patients	despite treatment with SoC (defined as an antibody with anti	treated with any other complement inhibitor less than 3
	C5 activity).	months prior to study start Day 1
Expected Completion	Primary endpoint: 2020	Primary endpoint: 2020
Extension period: 2023	Extension period: 2022
Publication	In preparation	TBD

77 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

Immunology, Hepatology & Dermatology

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

Study	NCT03663335 CIRRUS I (CCFZ533A2201)	NCT03905525 TWINSS (CCFZ533B2201)
Indication	Kidney transplantation	Sjögren's syndrome
Phase	Phase 2B	Phase 2B
Patients	676	260

Primary Outcome Measures

Arms/Intervention

Composite event (BPAR, Graft Loss or Death) over 12	Change in EULAR Sjögren's syndrome Disease Activity
months post-transplantation and post conversion (for	Index (ESSDAI) score and EULAR Sjögren's syndrome
maintenance cohort)	Patient Reported Index (ESSPRI) score
• Two cohorts: de novo TX and maintenance	• Three dose arms of CFZ533
• Test Arms: CFZ533 + MMF + corticosteroids
• Placebo
• Standard of Care: TAC + MMF + corticosteroids

Target Patients	Kidney transplant recipients	Patients with Sjögren's syndrome
Expected Completion	2022	2022
Publication	Manuscript of PoC trial to be submitted in Q1-2020	Manuscript of PoC trial to be published in Q1-2020

79 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

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

Study	NCT03781414 CONTRAIL I (CCFZ533A2202)
Indication	Liver transplantation
Phase	Phase 2
Patients	128

Primary Outcome	Proportion of patients with composite event (BPAR, Graft
Measures	Loss or Death) over 12 months
	• Control/Standard of Care: TAC + MMF + Corticosteroids
Arms/Intervention	• CFZ533 dose A + MMF + Corticosteroids
	• CFZ533 dose B + MMF + Corticosteroids

Target Patients

Expected Completion

Publication

Liver transplant recipients

2022

TBD

80 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

Cosentyx®- Anti IL-17

Study	NCT03504852 (CAIN457A2324)	NCT03589885 MATURE (CAIN457A2325)
Indication	Psoriasis	Psoriasis
Phase	Phase 3B	Phase 3
Patients	331	122
Primary Outcome	PASI 90 response and IGA mod 2011 0 or 1 response after	PASI 75 response and IGA mod 2011 0 or 1 response after
Measures	16 weeks of treatment	12 weeks of treatment

Arms/Intervention

Target Patients

Expected Completion

Publication

•	Secukinumab 300 mg every 2 weeks after weekly doses	•	Secukinumab 2 mL (300 mg) auto-injector
	till Week 4	•	Secukinumab 2 x 1 mL (150 mg each) prefilled syringe
•	Secukinumab 300 mg every 4 weeks after weekly doses	•	Placebo 2 mL auto-injector
	till Week 4	•	Placebo 2 x 1 mL prefilled syringe
Subjects (≥90kg) with moderate to severe plaque psoriasis	Subjects with moderate to severe plaque psoriasis
Q3-2020	Q4-2020
TBD	TBD

81 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

Cosentyx®- Anti IL-17

Study	NCT02471144 (CAIN457A2310)	NCT03668613 (CAIN457A2311)
Indication	Psoriasis	Psoriasis
Phase	Phase 3	Phase 3
Patients	162	84
Primary Outcome	Psoriasis Area and Severity Index (PASI) 75 response and	Psoriasis Area and Severity Index (PASI) 75 response and
Investigators' Global Assessment (IGA) 0 or 1 response at	Investigators' Global Assessment (IGA) 0 or 1 response at
Measures
week 12	week 12
	•	Secukinumab low dose
Arms/Intervention	•	Secukinumab high dose	•	Secukinumab low dose
•	Placebo	•	Secukinumab high dose
	•	Etanercept (comparator)
Target Patients	Patients from 6 to less than 18 years of age with severe	Pediatric patients of age 6 to <18 years, with moderate to
chronic plaque psoriasis	severe plaque psoriasis
Expected Completion	2023	2023
Publication	TBD	TBD

82 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

Cosentyx®- Anti IL-17

Study	NCT03066609 (CAIN457A2318)
Indication	Psoriasis
Phase	Phase 3
Patients	543
Primary Outcome	Psoriasis Area and Severity Index (PASI) 75 response and

Measures	Investigators' Global Assessment (IGA) 0 or 1 response at
week 12

Arms/Intervention

Target Patients

Expected Completion

Publication

• Secukinumab 300 mg
• Secukinumab 150 mg
• Placebo

Patients with moderate to severe chronic plaque-type psoriasis with or without psoriatic arthritis comorbidity

Q1-2019(actual)

• Week 16 results: Poster presented at: 2019 American Academy of Dermatology (AAD) Annual Meeting,
• March1-5, 2019, Washington, D.C.
• 52-weekresults: Poster at EADV 2019, Madrid 9-13 October, 2019

83 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

Cosentyx®- Anti IL-17

Study	NCT03031782 (CAIN457F2304)	NCT03769168 (CAIN457F2304E1 - extension study)
Indication	Psoriatic arthritis	Psoriatic arthritis
Phase	Phase 3	Phase 3
Patients	80		64
Primary Outcome	Time to 33 flares	Number of participants with JIA ACR30 response
Measures
Arms/Intervention	•	Secukinumab (pre-filled syringe) 75 mg	•	Secukinumab 75 mg/0.5 ml
•	Placebo	•	Secukinumab 150 mg/1.0 ml
Target Patients	Juvenile idiopathic arthritis subtypes of psoriatic and	Patients with juvenile idiopathic arthritis subtypes of juvenile
enthesitis-related arthritis	psoriatic arthritis and enthesitis related arthritis
Expected Completion	H1-2021	2025
Publication	TBD	TBD

84 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

Cosentyx®- Anti IL-17

Study	NCT01892436 FUTURE 1 extension (CAIN457F2306E1)	NCT01649375 MEASURE 2 (CAIN457F2310)
Indication	Psoriatic arthritis	Ankylosing spondylitis
Phase	Phase 3	Phase 3
Patients	460	219
Primary Outcome	Proportion of subjects that have a positive clinical response	Assessment of SpondyloArthritis International Society /
to treatment (individual improvement) in disease activity
Measures	ASAS 20 response
according to ACR20 (or ACR50 or ACR 70)
	•	Secukinumab 75 mg	•	Secukinumab 75 mg
Arms/Intervention	•	Secukinumab 150 mg
•	Secukinumab 150 mg
	•	Placebo
Target Patients	Patients with active psoriatic arthritis	Patients with active ankylosing spondylitis
Expected Completion	Q1-2018(actual)	Q4-2018(actual)
			• Primary 52 week results: Baeten D & Sieper J, et al. N
	• 3 year results: ACR 2016; Mease PJ et al. Arthritis		Engl J Med 2015;373:2534-48
	•	2 year results: Marzo-Ortega, et al. Arthritis Care Res
		Rheumatol. 2016; 68 (suppl 10)
			2017 Feb 24. doi: - 10.1002/acr.23233
	•	3 years results: Manuscript published in September
	•	3 year results: Marzo-Ortega, et al. RMD 2017
Publication		2018 (Mease PJ, et al. RMD Open 2018;4:e000723.
	•	5 year results: EULAR 2019; Marzo-Ortega H, et al.
		doi:10.1136/rmdopen-2018-000723)
			FRI0379. Annals of the Rheumatic Diseases
	•	5 year results: accepted for publication in ACR open
		2019;78:873.
		Rheumatology in September 2019
		•	5 year results; manuscript target submission Oct 2019

85 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

Cosentyx®- Anti IL-17

Study	NCT01752634 FUTURE 2 (CAIN457F2312)	NCT02008916 MEASURE 3 (CAIN457F2314)
Indication	Psoriatic arthritis	Ankylosing spondylitis
Phase	Phase 3	Phase 3
Patients	399	222
Primary Outcome	Proportion of subjects achieving American College of	Assessment of Spondyloarthritis International Society
Measures	Rheumatology 20 (ACR20) response criteria	criteria / ASAS 20 response
	•	Secukinumab (AIN457) 150 mg s.c.	•	Secukinumab 10 mg/kg / 300 mg
	•	Secukinumab (AIN457) 75 mg s.c.
Arms/Intervention	•	Secukinumab 10 mg/kg / 150 mg
•	Secukinumab (AIN457) 300 mg s.c.
	•	Placebo
	•	Placebo s.c.
Target Patients	Patients with active psoriatic arthritis	Patients with active ankylosing spondylitis
Expected Completion	Q1-2019(actual)	Q1-2018(actual)
			• 16 weeks results: PANLAR congress in Apr-2016
	• Primary results: McInnes IB, et al. Lancet.	• 52 weeks results: Pavelka et al. Arthritis Research &
		2015;386:1137-46		Therapy 2017
Publication	•	2 years results: McInnes et al, Rheumatology	• 2 year results: Presented at ACR in Nov-2017
	2017;56:1993-2003	•	3 year (EOS) results: To be presented (ORAL) at
	•	5 year (EOS) manuscript ongoing; to be submitted in		PANLAR April 2019
		Oct-2019	•	3 year (EOS) manuscript submitted in May-2019;
				awaiting journal decision
86 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

Cosentyx®- Anti IL-17

Study	NCT01989468 FUTURE 3 (CAIN457F2318)	NCT02159053 MEASURE 4 (CAIN457F2320)
Indication	Psoriatic arthritis	Ankylosing spondylitis
Phase	Phase 3	Phase 3
Patients	416	350
Primary Outcome	American College of Rheumatology 20 (ACR20) response in	Assessment of Spondyloarthritis International Society
Measures	subjects treated with secukinumab vs. placebo	criteria / ASAS 20 at week 16
	•	Secukinumab (AIN457) 150 mg s.c.	•	Secukinumab 150 mg s.c. with loading
Arms/Intervention	•	Secukinumab (AIN457) 300 mg s.c.	•	Secukinumab 150 mg s.c. without loading
	•	Placebo	•	Placebo
Target Patients	Patients with active psoriatic arthritis	Patients with active ankylosing spondylitis
Expected Completion	Q2-2018(actual)	Q2-2018(actual)
Publication	52 week results: Nash et al, Arthritis Research & Therapy	•	Week 104 (EOS) manuscript: Kivitz et al, Rheumatol
2018, 20:47		Ther https://doi.org/10.1007/s40744-018-0123-5

87 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

Cosentyx®- Anti IL-17

Study	NCT02294227 FUTURE 4 (CAIN457F2336)	NCT02404350 FUTURE 5 (CAIN457F2342)
Indication	Psoriatic arthritis	Psoriatic arthritis
Phase	Phase 3	Phase 3
Patients	342	990
Primary Outcome	Assessment of American College of Rheumatology 20	American College of Rheumatology 20 (ACR20) response at
Measures	(ACR20)	Week 16
	•	Secukinumab 150 mg with loading	•	Secukinumab 150 mg load
	•	Secukinumab 150 mg no load
Arms/Intervention	•	Secukinumab 150 mg without loading
•	Secukinumab 300 mg load
	•	Placebo
	•	Placebo
Target Patients	Patients with active psoriatic arthritis	Patients with active psoriatic arthritis
Expected Completion	Q1-2018(actual)	Q1-2019(actual)
			• 24 week results published: Mease P, et al. Annals of the
	•	52 week results: abstract presented at PANLAR		Rheumatic Diseases 2018;77:890-897.
Publication		congress (Apr-2018)	• 52 week results presented at EULAR and ACR 2018
•	2 year (EOS) results published: Rheumatology	•	52 week manuscript accepted (Rheumatology, October
		Therapy. 2019 Jun 21. doi: 10.1007/s40744-019-0163-5.		2019, in press)
			•	2 year (EOS) results presented at EULAR 2019

88 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

Cosentyx®- Anti IL-17

Study	NCT01863732 (CAIN457F2305E1 - extension study)	NCT02745080 EXCEED (CAIN457F2366)
Indication	Ankylosing spondylitis	Psoriatic arthritis
Phase	Phase 3	Phase 3
Patients	300	850
Primary Outcome	Assessment of spondyloarthritis international society criteria	American College of Rheumatology 20 (ACR20) response
Measures	/ ASAS 20 response
Arms/Intervention	•	Secukinumab 75 mg in PFS	•	Secukinumab 300 mg s.c.
•	Secukinumab 150 mg in PFS	•	Adalimumab 40 mg s.c.
Target Patients	Patients with active ankylosing spondylitis	Patients with active psoriatic arthritis
Expected Completion	Q2-2018(actual)	Q1-2020
	• 3-year results: Manuscript published in Clinical and
		Experimental Rheumatology in May-2017
	• 4-year results: Presented at ACR in Nov-2017
	• 4 year results manuscript published; Rheumatology,
Publication		Volume 58, Issue 5, May 2019, Pages 859-868,	•	Manuscript target submission Jan-2020
	•	5 year (EOS) results manuscript published; Baraliakos X,

et al. RMD Open 2019;5:e001005. doi: 10.1136/rmdopen-2019-001005

89 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

Cosentyx®- Anti IL-17

Study	NCT02696031 PREVENT (CAIN457H2315)	NCT03259074 SURPASS (CAIN457K2340)
Indication	Non-radiographic axial spondyloarthritis	Ankylosing spondylitis
Phase	Phase 3	Phase 3
Patients	555	837
Primary Outcome	The proportion of participants who achieved an ASAS 40	No radiographic structural progression as measured by
response (Assessment of SpondyloArthritis International	modified Stoke Ankylosing Spondylitis Spine Score
Measures
Society criteria);	(mSASSS)
	•	Secukinumab 150 mg load	•	Secukinumab 150/300 mg
Arms/Intervention	•	Secukinumab 150 mg no load
•	Adalimumab biosimilar 40 mg
	•	Placebo
Target Patients	Patients with non-radiographic axial spondyloarthritis	Patients with active ankylosing spondylitis
Expected Completion	Week 52: Q3-2019(actual); Final: H1-2021	2022
	• Abstract (16 week results) submitted as a late breaker to
Publication		ACR 2019	TBD
	•	Manuscript target submission Jan-2020

90 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

Cosentyx®- Anti IL-17

Study	NCT03713619 SUNSHINE (CAIN457M2301)	NCT03713632 SUNRISE (CAIN457M2302)
Indication	Hidradenitis Suppurativa (HS)	Hidradenitis Suppurativa (HS)
Phase	Phase 3	Phase 3
Patients	471	471
Primary Outcome	Proportion of participants with Hidradenitis Suppurativa	Proportion of patients with Hidradenitis Suppurativa Clinical
Measures	clinical response (HiSCR)	Response (HiSCR)
	•	Secukinumab 300 mg every 2 weeks	•	Secukinumab 300 mg every 2 weeks
Arms/Intervention	•	Secukinumab 300 mg every 4 weeks	•	Secukinumab 300 mg every 4 weeks
•	Placebo (every 2 weeks)	•	Placebo (every 2 weeks)
	•	Placebo (every 4 weeks)	•	Placebo (every 4 weeks)
Target Patients	Subjects with moderate to severe Hidradenitis Suppurativa	Subjects with moderate to severe Hidradenitis Suppurativa
Expected Completion	H2-2021	H2-2021
Publication	Preliminary results in EADV (most likely) in 2021	Preliminary results in EADV (most likely) in 2021

91 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

Cosentyx®- Anti IL-17

Study	NCT04156620 INVIGORATE-1 (CAIN457P12301)
Indication	Axial spondyloarthritis
Phase	Phase 3
Patients	500
Primary Outcome	The proportion of subjects achieving an ASAS40
(Assessment of SpondyloArthritis International Society
Measures
criteria) response
Arms/Intervention	• Secukinumab intravenous (i.v.) regimen
• Placebo intravenous (i.v.) regimen
Target Patients	Patients with active axial spondyloarthritis
Expected Completion	2022
Publication	TBD

92 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

Ilaris®- Anti IL-1β

Study	NCT02296424 (CACZ885G2306)
Indication	SJIA - Systemic Juvenile Idiopathic Arthritis
Phase	Phase 3B/4
Patients	182
	Proportion of patients in clinical remission on
Primary Outcome	canakinumab who are able to remain in remission
following canakinumab dose tapering (reduced
Measures
canakinumab dose or prolonged canakinumab dosing
	interval)
Arms/Intervention	•	Canakinumab dose reduction
•	Canakinumab dose interval prolongation
Target Patients	Patients with Systemic Juvenile Idiopathic Arthritis (SJIA)
(Pediatric)
Expected Completion	2018(actual)

• Remission & flexible dosing - presented at ISSAID &

Publication	EULAR in Q2-2019
• Planned manuscript in 2019: Remission & flexible
	dosing to be submitted in Q4-2019

93 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

LJN452 - FXR Agonist

Study	NCT02855164 (CLJN452A2202)	NCT04065841 ELIVATE (CLJN452D12201C)
Indication	Non-alcoholic steatohepatitis (NASH)	Non-alcoholic steatohepatitis (NASH)
Phase	Phase 2	Phase 2
Patients	345	210
	Adverse event profile of different doses; determine the dose
	relationship of LJN452 on markers of hepatic inflammation	Proportion of patients with resolution of NASH and no
Primary Outcome	in NASH (ALT and AST); determine dose-response	worsening of fibrosis OR improvement in fibrosis by at least
Measures	relationship of LJN452 on liver fat content by changes in	one stage without worsening of NASH at Week 48
	quantitative MRI; determine effect of LJN452 on liver fibrosis	compared with baseline
	by biopsy
			• Arm A: combination therapytropifexor + licogliflozin
			• Arm B: tropifexor monotherapytropifexor (+ licogliflozin
Arms/Intervention	•	Multiple LJN452 doses and placebo	placebo)
			• Arm C: licogliflozin monotherapylicogliflozin (+ tropifexor
			placebo)
Target Patients	Patients with non-alcoholic steatohepatitis (NASH)	Adult patients with non-alcoholic steatohepatitis (NASH)
and liver fibrosis
Expected Completion	Q2-2020	2022
	• Primary (interim) data abstract submitted to AASLD in
Publication		Q3-2019	TBD
	•	Manuscript to be submitted in H2-2020

94 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

LOU064 - Bruton's tyrosine kinase (BTK) inhibitor

Study	NCT03926611 (CLOU064A2201)	NCT04109313 (CLOU064A2201E1)
Indication	Chronic spontaneous urticaria (CSU)	Chronic spontaneous urticaria (CSU)
Phase	Phase 2	Phase 2
Patients	308	250
Primary Outcome Measures	Change from baseline in weekly Urticaria Activity Score (UAS7) at Week	•	Long-term safety and tolerability
4
	•	Arm 1 Low dose of LOU064 orally in the morning (once daily) and
		matching placebo in the evening from Day 1 to 85
	•	Arm 2 Medium dose of LOU064 orally in the morning (once daily) and
		matching placebo in the evening from Day 1 to 85
Arms/Intervention	•	Arm 3 High dose of LOU064 orally in the morning (once daily) and	•	Selected dose of LOU064 taken orally twice a day
	matching placebo in the evening from Day 1 to 85		(morning and evening) from day 1 to week 52
	• Arm 4 Low dose of LOU064 orally, twice daily from Day 1 to 85
	• Arm 5 Medium dose of LOU064 orally, twice daily from Day 1 to 85
	• Arm 6 High dose of LOU064 orally, twice daily from Day 1 to 85
	•	Placebo arm Matching placebo, orally, twice daily from Day 1 to 85
Target Patients	Adults with CSU inadequately controlled by H1-antihistamines	Patients with CSU who have participated in preceding
studies with LOU064
Expected Completion	Q3-2020	2022
Publication	TBD	TBD

95 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

LJC242 - FXR agonist + CCR2/CCR5 inhibitor

Study	NCT03517540 TANDEM (CLJC242A2201J)
Indication	Non-alcoholic steatohepatitis
Phase	Phase 2
Patients	200
Primary Outcome	• Evaluation of safety and tolerability of combination

Measures	therapy (tropifexor + cenicriviroc) by monitoring adverse
event profile, vital signs and laboratory parameters

Arms/Intervention

Target Patients

Expected Completion

Publication

• Tropifexor
• Cenicriviroc
• Tropifexor + cenicriviroc

Adult patients with non-alcoholic steatohepatitis (NASH) and liver fibrosis

Q4-2020

Manuscript to be submitted in H1-2021

96 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

QGE031 - Anti-IgE

Study	NCT02477332 (CQGE031C2201)	NCT02649218 (CQGE031C2201E1)
Indication	Chronic spontaneous urticaria / Chronic idiopathic urticaria	Chronic spontaneous urticaria / Chronic idiopathic urticaria
Phase	Phase 2B	Phase 2B
Patients	382	226
Primary Outcome	Establish dose-response relationship of QGE031 with	Long-term safety; number of participants with treatment-
respect to achievement of complete hives response at week
Measures	emergent adverse events
12
	•	Ligelizumab 24mg q4wks for 20 weeks
	•	Ligelizumab 72mg q4wks for 20 weeks
Arms/Intervention	•	Ligelizumab 240mg q4wks for 20 weeks	Ligelizumab 240 mg q4wks open label for 52 weeks
• Ligelizumab 120mg single dose
	•	Omalizumab 300mg q4wks for 20 weeks
	•	Placebo q 4wks for 20 weeks
	Adult patients with chronic spontaneous urticaria	Adult patients with chronic spontaneous urticaria
Target Patients	inadequately controlled with H1-antihistamines at approved	inadequately controlled with H1-antihistamines at approved
or increased doses, alone or in combination with H2-	or increased doses, alone or in combination with H2-
	antihistamines or leukotriene receptor antagonists.	antihistamines or leukotriene receptor antagonists.
Expected Completion	2017(actual)	Q3-2019(actual)
	• Primary results: Presented at EAACI 2018, EADV 2018,	• Primary results: AAD 2019;
Publication		and GUF 2018; NEJM publication (Oct. 3rd);	• Secondary results presented in 2019 at: AAD, EAACI,
• Secondary results presented in 2019 at: AAD, EAACI,	WCD, EADV, PAAM, ACAAI, UCARE; manuscript
		WCD, EADV, PAAM, ACAAI, UCARE.	planned in H1/2020

97 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

QGE031 - Anti-IgE

Study	NCT03437278 (CQGE031C2202)
Indication	Chronic spontaneous urticarial / Chronic idiopathic urticaria
Phase	Phase 2
Patients	48
Primary Outcome	Change in the 7 day Urticaria Activity Score (UAS7)
Measures
	• Ligelizumab high dose q4wks for 24 weeks
Arms/Intervention	•	Ligelizumab low dose q4wks for 24 weeks
	•	Placebo / ligelizumab high dose q4wks for 8 / 16 weeks

Target Patients

Expected Completion

Adolescents from 12 to <18 years of age, with chronic spontaneous urticaria

H2-2021

	• Study design was presented at PAAM (Peds Allergy &
		Asthma Meeting) and at UCARE meeting 2019
Publication	•	Primary results to be presented in 2022 (e.g. EAACI,
		PAAM, EADV)
	•	Manuscript to be submitted in 2022

98 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

QGE031 - Anti-IgE

Study	NCT03580369 Pearl 1 (CQGE031C2302)	NCT03580356 Pearl 2 (CQGE031C2303)
Indication	Chronic spontaneous urticaria	Chronic spontaneous urticaria
Phase	Phase 3	Phase 3
Patients	1,050	1,050

Primary Outcome	Absolute change from baseline in UAS7 (Urticaria Activity	Absolute change from baseline in UAS7 (Urticaria Activity
Measures	Score) at week 12	Score) at week 12
	•	Ligelizumab dose A q4w for 52 weeks	•	Ligelizumab dose A q4w for 52 weeks
	•	Ligelizumab dose B q4w for 52 weeks	•	Ligelizumab dose B q4w for 52 weeks
Arms/Intervention	•	Omalizumab 300 mg q4w for 52 weeks	•	Omalizumab 300 mg q4w for 52 weeks
	•	Placebo q4w from randomization to wk20, then	•	Placebo q4w from randomization to wk20, then
		ligelizumab dose B from wk24 to wk52		ligelizumab dose B from wk24 to wk52
Target Patients	Adolescents and adults with chronic spontaneous urticaria	Adolescents and adults with chronic spontaneous urticaria
inadequately controlled with H1-antihistamines	inadequately controlled with H1-antihistamines
Expected Completion	H2-2021	H2-2021
	• Study design presented at UCARE 2018
Publication	•	Primary results to be presented in 2022 (e.g. EAACI, PAAM, EADV)
	•	Manuscript to be submitted in 2022

99 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

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

Study	NCT02962895 (CVAY736A2201)	NCT03217422 AMBER (CVAY736B2201)
Indication	Primary Sjögren's syndrome	Autoimmune hepatitis
Phase	Phase 2B	Phase 2/3
Patients	180	80

Primary Outcome	Safety and efficacy of VAY736 in primary Sjögren's	Alanine aminotransferase (ALT) normalization
Measures	syndrome (pSS)
Arms/Intervention	•	VAY736	• VAY736
•	Placebo	• Placebo control with conversion to active VAY736
Target Patients	Patients with moderate to severe primary Sjögren's	Autoimmune hepatitis patients with incomplete response or
syndrome (pSS)	intolerant to standard treatment of care
Expected Completion	Q2-2020	2023
	• Late Breaking Abstract to be submitted to American
Publication		College of Rheumatology 30-Sep-2019	TBD
	•	Manuscript to be submitted in 2020

100 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

ZPL389 - H4 receptor antagonist

Study	NCT03517566 ZEST (CZPL389A2203)	NCT03948334 ZESTExt (CZPL389A2203E1 - extension
study)
Indication	Atopic dermatitis	Atopic dermatitis
Phase	Phase 2	Phase 2
Patients	360	360
Primary Outcome	IGA (Investigator's global assessment) response at week 16	Frequency of Adverse Events (AEs) and Serious Adverse
Measures	Events (SAEs)
	•	ZPL389 dose 1	•	ZPL389 Dose 1 + Topical Corticosteroids (TCS) and /or
	•	ZPL389 dose 2		Topical Calcineurin Inhibitors (TCI)
Arms/Intervention	•	ZPL389 dose 3	•	ZPL389 Dose 2 + Topical Corticosteroids (TCS) and /or
	•	ZPL389 dose 4		Topical Calcineurin Inhibitors (TCI)
	•	Placebo
Target Patients	Patients with moderate to severe atopic dermatitis	Adult patients with atopic dermatitis
Expected Completion	H1-2021	2023
Publication	TBD	TBD

101 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

Neuroscience

Zolgensma®- SMN1 gene replacement therapy

Study	NCT03461289 STRIVE-EU(CL-302)	NCT03306277 STRIVE (CL-303)
Indication	Type 1 spinal muscular atrophy	Type 1 spinal muscular atrophy
Phase	Phase 3	Phase 3
Patients	30	20

Primary Outcome		• Achievement of independent sitting for at least 30
Proportion of participants sitting without support	seconds
Measures
	• Event-free survival
Arms/Intervention	Open-label,single-arm,single-dose, intravenous	Open-label,single-arm,single-dose, intravenous
Target Patients	Patients with spinal muscular atrophy Type 1	Patients with Spinal Muscular Atrophy Type 1
Expected Completion	H2-2020	Q4-2019(actual)
Publication	TBD	TBD

103 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

Zolgensma®- SMN1 gene replacement therapy

Study	NCT03505099 SPR1NT (CL-304)	NCT03837184 STRIVE Asia Pacific (CL-306)
Indication	Spinal muscular atrophy	Type 1 spinal muscular atrophy
Phase	Phase 3	Phase 3
Patients	27	6
	• Percentage of participants achieving functional
Primary Outcome	independent sitting for at least 30 seconds at any visit	Proportion of participants sitting without support
Measures	• Percentage of participants achieving the ability to stand
	without support for at least 3 seconds at any visit
Arms/Intervention	Open-label,single-arm,single-dose, intravenous	Open-label,single-arm,single-dose, intravenous
Target Patients	Pre-symptomatic patients with spinal muscular atrophy and	Patients with spinal muscular atrophy Type 1
multiple copies SMN2
Expected Completion	H2-2021	H2-2021
Publication	TBD	TBD

104 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

Zolgensma®- SMN1 gene replacement therapy

Study	NCT03381729 STRONG (CL-102)
Indication	Type 2 spinal muscular atrophy
Phase	Phase 1
Patients	27

Primary Outcome	•	Safety and tolerability, incidence of adverse events
•	Proportion of patients achieving Standing Milestone
Measures
• Change in Hammersmith Functional Motor Scale
Arms/Intervention	Open-label,single-arm,single-dose, intrathecal
Target Patients	Patients with spinal muscular atrophy with 3 copies of SMN2
Expected Completion	Q4-2019 [Cohort B]
Publication	TBD

105 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

Aimovig®- CGRP receptor antagonist

Study	NCT03096834 LIBERTY (CAMG334A2301)	NCT03333109 EMPOWER (CAMG334A2302)
Indication	Migraine	Migraine
Phase	Phase 3	Phase 3
Patients	246	900
Primary Outcome	Percentage of patients with a 50% response in the reduction	Change from baseline in monthly migraine days at the last
Measures	of Monthly Migraine Days (MMD)	month (Month 3) of the double-blind treatment period
	•	Subcutaneous injection of AMG334 (erenumab)	•	AMG334 (erenumab) Dose 1
Arms/Intervention	•	AMG334 (erenumab) Dose 2
•	Subcutaneous injection of placebo
	•	Placebo
Target Patients	Adult episodic migraine patients who have failed prophylactic	Adult episodic migraine patients
migraine treatments
Expected Completion	2017 DBT phase (actual); H1-2021 OLE phase (final DBL)	Q2-2020
	• Planned for Q1-2020 (Neurology): PROs and
		prespecified subgroup analysis (DBT phase)
Publication	• Planned for Q2-2020: 1Y OLE	Planned for H2-2020
	• Planned for Q4 2020: 2Y OLE - TBC. Potentially
		abstract only

106 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

Aimovig®- CGRP receptor antagonist

Study	NCT03867201 DRAGON (CAMG334A2304)
Indication	Migraine
Phase	Phase 3
Patients	550
Primary Outcome	Change from baseline in monthly migraine days during the
Measures	last 4 weeks of the 12-week treatment period
Arms/Intervention	• Subcutaneous injection of AMG334 (erenumab) 70 mg
• Subcutaneous injection of placebo
Target Patients	Adult chronic migraine patients
Expected Completion	2022 DBT phase; 2024 OLE phase
Publication	Planned in Q4-2023 (DBT)

107 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

Gilenya®- S1P-R modulator

Study	NCT01633112 ASSESS (CFTY720D2312)	NCT01201356 LONGTERMS (CFTY720D2399)
Indication	Relapsing remitting multiple sclerosis (RRMS)	Relapsing multiple sclerosis (RMS)
Phase	Phase 3B	Phase 3
Patients	1,064	4,125
Primary Outcome	Comparison of 2 doses (0.25 mg and 0.5 mg) of fingolimod
to glatiramer acetate (20 mg) in reducing the annualized	Long-term safety and tolerability
Measures
relapse rate up to 12 months
	• Fingolimod 0.5 mg orally
Arms/Intervention	•	Fingolimod 0.25mg orally	Single-arm study of fingolimod 0.5 mg/day
	• Copaxone®20 mg s.c.
Target Patients	Patients with relapsing-remitting multiple sclerosis	Patients with relapsing multiple sclerosis
Expected Completion	2018(actual)	2018(actual)
			• Cohen J et al. Extended treatment with fingolimod for
	• Primary data presentation at AAN in 2019	relapsing multiple sclerosis: the 14-year LONGTERMS
Publication	study results (Therapeutic Advances in Neurological
•	Primary manuscript - submission planned in Q4-2019
	Disorders 2019.12:eCollection 2019, doi:
			10.1177/1756286419878324)

108 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

LMI070 - SMN2 RNA splice modulator

Study	NCT02268552 (CLMI070X2201)
Indication	Type 1 spinal muscular atrophy
Phase	Phase 1/2
Patients	39
Primary Outcome	Number of participants with adverse events (AEs), serious
Measures	adverse events (SAEs) and deaths
	Branaplam oral, once weekly:
	• Part 1: 5 ascending doses
Arms/Intervention	• Part 2: 2 different dose levels
	• Part 3: patients continue on initial dose assigned in Part
	1 or Part 2
Target Patients	Patients with type 1 spinal muscular atrophy
Expected Completion	Q3-2020
Publication	TBD

109 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

Mayzent ®- S1P-R modulator

Study	NCT01665144 -EXPAND (CBAF312A2304)
Indication	Secondary progressive multiple sclerosis
Phase	Phase 3
Patients	1,652

Primary Outcome Measures	The delay in time to confirmed disability progression as
measured by EDSS (Expanded Disability Status Scale)

Arms/Intervention

Target Patients

Expected Completion

Publication

• BAF312(5-day titration: 0.25mg to 1.25mg; Maintenance
  dose: 2mg (day 6))
• Placebo

Patients with secondary progressive multiple sclerosis

Core in 2016/Extension in 2023

The Lancet Neurology, Volume 39, No.10127, p1237-1330, March 2018

110 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

OMB157 - Anti-CD20

Study	NCT02792218 Asclepios I (COMB157G2301)	NCT02792231 Asclepios II (COMB157G2302)
Indication	Multiple sclerosis	Multiple sclerosis
Phase	Phase 3	Phase 3
Patients	900	900

Primary Outcome	Annualized Relapse Rate (ARR) - number of confirmed	Annualized Relapse Rate (ARR) - number of confirmed
relapses in a year calculated based on cumulative number	relapses in a year calculated based on cumulative number
Measures
of relapses by patient adjusted for time-in-study by patient	of relapses by patient adjusted for time-in-study by patient
Arms/Intervention	•	Ofatumumab subcutaneous	•	Ofatumumab subcutaneous
•	Teriflunomide oral	•	Teriflunomide oral
Target Patients	Patients with relapsing forms of multiple sclerosis	Patients with relapsing forms of multiple sclerosis
Expected Completion	Q3-2019(actual)	Q3-2019(actual)
Publication	Primary manuscript planned in H1-2020	Primary manuscript planned in H1-2020

111 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

OMB157 - Anti-CD20

Study	NCT03249714 APOLITOS (COMB157G1301)	NCT03650114 ALITHIOS (COMB157G2399)
Indication	Multiple sclerosis	Multiple Sclerosis
Phase	Phase 2	Phase 3
Patients	60	2010

Primary Outcome	Reduced cumulative number of Gd-enhanced T1 lesions	Evaluate the long-term safety and tolerability of ofatumumab
across 4 MRI scans at week 12, 16, 20 and 24 (ofatumumab	20 mg subcutaneous (sc) once every 4 (q4) weeks in
Measures
vs placebo)	subjects with RMS from the first dose of ofatumumab
Arms/Intervention	•	Ofatumumab 20 mg subcutaneous injections	• Ofatumumab 20 mg every 4 weeks
•	Placebo
Target Patients	Patients with relapsing forms of multiple sclerosis	Patients with relapsing MS
Expected Completion	Q1-2020	2025
Publication	TBD	TBD

112 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

Oncology

ABL001 - Specific, allosteric Bcr-Abl kinase inhibitor

Study

NCT03106779 ASCEMBL (CABL001A2301)

Indication

Phase

Patients

Primary Outcome Measures

Arms/Intervention

Target Patients

Expected Completion

Publication

Chronic myeloid leukaemia (CML)

Phase 3

233

Major Molecular Response (MMR) rate at 24 weeks

• ABL001 40 mg bid
• Bosutinib 500 mg

Patients with chronic myelogenous leukemia in chronic phase, previously treated with 2 or more tyrosine kinase inhibitors

H2-2020

Manuscript submission in H2-2020 (journal TBD)

114 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

ACZ885 - IL-1β inhibitor

Study	NCT03447769 CANOPY-A (CACZ885T2301)	NCT03631199 CANOPY-1 (CACZ885U2301)
Indication	Adjuvant NSCLC	1stLine Non-small cell lung cancer (NSCLC)
Phase	Phase 3	Phase 3
Patients	1,500	627

Primary Outcome Measures

Arms/Intervention

Target Patients

Expected Completion

Publication

		• Safety run-in part: Incidence of dose limiting toxicities
Disease free survival (primary), overall survival (key	•	Double-blind, randomized, placebo-controlled part:
secondary)		Progression free survival (PFS)
		•	Overall survival (OS)
•	Canakinumab 200mg q3w sc for 18 cycles	•	Canakinumab or matching placebo in combination with
	pembrolizumab and platinum-based doublet
•	Placebo q3w sc for 18 cycles
	chemotherapy
Patients with:	Patients with
•	High-risk NSCLC (AJCC/UICC v.8 stage II-IIIA and IIIB	•	Histologically confirmed Stage IIIB, IV NSCLC with no
	(T>5cm N2)) after complete resection and standard of		prior systemic anticancer therapy
	care adjuvant cisplatin-based chemotherapy	•	Squamous and non-squamous NSCLC
•	All histologies	• No EGFR mutation and ALK rearrangement
2022	H1-2021
TBD	Johnson B et al. Abstract accepted for presentation at
AACR-NCI-EORTC Oct 2019 (safety run-in)

115 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

ACZ885 - IL1β inhibitor

Study	NCT03626545 CANOPY-2 (CACZ885V2301)

Indication

Phase

Patients

Primary Outcome Measures

Arms/Intervention

Target Patients

Expected Completion

Publication

2nd/ 3rdLine Non-small cell lung cancer (NSCLC)

Phase 3

240

• Safetyrun-in part: Incidence of dose limiting toxicities
• Double-blind,randomized, placebo-controlled part: Overall Survival
• canakinumab in combination with docetaxel
• canakinumabmatching-placebo in combination with docetaxel

Patients with:

• Stage IIIB or IV NSCLCwithout EGFR, ALK,ROS-1 or B- RAF mutation
• Previously treated with platinum therapy and PD(L)1- inhibitor

H1-2021

TBD

116 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

BYL719 - Alpha-specific PI3K inhibitor

Study	NCT02437318 SOLAR-1 (CBYL719C2301)
Indication	HR+/HER2- advanced breast cancer with PIK3CA mutation
Phase	Phase 3
Patients	572
Primary Outcome	Progression-free survival (PFS) for patients with PIK3CA
Measures	mutant status
Arms/Intervention	• Fulvestrant 500 mg + alpelisib 300 mg
• Fulvestrant 500 mg + placebo
	Men and postmenopausal women with hormone receptor
Target Patients	positive, HER2-negative advanced breast cancer which
	progressed on or after aromatase inhibitor treatment
Expected Completion	Q3-2018(actual)
	• Andre F, et al. Presentation at ESMO 2018
Publication	• Andre et al. Manuscript N Engl J Med 2019;380:1929-
	1940.

117 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

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

Study	NCT00940602 TELESTO (CICL670A2302)
Indication	Iron overload
Phase	Phase 2
Patients	224
Primary Outcome	To compare deferasirox to placebo with regard to event-free
survival in low and int-1 risk MDS patient with transfusional
Measures
iron overload
Arms/Intervention	•	Deferasirox, iron chelator
•	Placebo
Target Patients	Patients with myelodysplastic syndromes (low/int-1 risk) and
transfusional iron overload
Expected Completion	Q3-2018(actual)
Publication	•	Angelucci E, et al. Presentation at ASH 2018
•	Angelucci E, et al. Manuscript submitted Q3-2019

118 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

INC280 - MET Inhibitor

Study	NCT02414139 (CINC280A2201)	NCT03647488 (CINC280D2201)
Indication	EGFR Wild-type, ALK negative advanced Non-small Cell	Non-small cell lung cancer
Lung Cancer (NSCLC)
Phase	Phase 2	Phase 2
Patients	364	105
Primary Outcome			Run in part: Assess safety and tolerability of capmatinib and
Overall Response Rate (ORR)	spartalizumab combination.
Measures
		Randomized part: Overall Survival (OS)
	•	Pre-treated pts. with MET GCN: ≥ 6; ≥ 4 and < 6; < 4
	• Pre-treated pts. with MET mutations regardless of
		cMET GCN as second or third line	•	Capmatinib plus spartalizumab
Arms/Intervention	•	Treatment-naïve pts. with MET dysregulation
•	Docetaxel
	• Pre-treated pts with MET dysregulation - second line
	• Treatment-naïve pts with cMET mutations regardless of
		cMET GCN
			Pre-treated adult patients with EGFR wild-type ALK
	Adult patients with EGFR wild-type (wt), ALK-negative	rearrangement negative advanced/metastatic non-small cell
Target Patients	advanced/ metastatic NSCLC with either MET	lung cancer, that has demonstrated progression following one
	amplification or MET mutations	prior platinum doublet and one prior PD-(L)1 checkpoint
			inhibitor
Expected Completion	Q2-2019(actual)	2022
Publication	•	Wolf J, et al. Presented at ASCO 2019	TBD
•	Wolf J, et al. Manuscript submitted Q3-2019

119 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

Jakavi®- JAK1/2 inhibitor

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)
Phase	Phase 3	Phase 3
Patients	308	324
Primary Outcome	Overall Response Rate (ORR) at 28 Days	Overall Response Rate (ORR) at 183 Days
Measures
Arms/Intervention	•	Ruxolitinib 10mg bid	•	Ruxolitinib 10mg bid
•	Best available therapy (BAT)	•	Best available therapy (BAT)
Target Patients	Patients with SR aGVHD	Patients with SR cGVHD
Expected Completion	Q3-2019(actual)	Interim Analysis: Q3-2019(actual); Final:Q3-2020
Publication	•	Manuscript submission in Q4-2019	•	Manuscript submission in H2-2020
•	Zeiser R, et al. Abstract submitted Q4-2019	•	Abstract submission to congress in H2-2020

120 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

Jakavi®- JAK1/2 inhibitor

Study	NCT03491215 REACH4 (CINC424F12201)	NCT04097821 ADORE (CINC424H12201)
Indication	Acute graft versus host disease	Myelofibrosis
Phase	Phase 2	Phase 1/2
Patients	45	130

Primary Outcome Measures

Arms/Intervention

Target Patients

Expected Completion

Publication

• Measurement of PK parameters	• Incidence of dose limiting toxicities within the first 2
	cycles
• Overall Response Rate (ORR)
• Response rate at the end of cycle 6
	•	Ruxolitinib
• Ruxolitinib	•	Ruxolitinib+Siremadlin
•	Ruxolitinib+Crizanlizumab
	•	Ruxolitinib+MBG453
Pediatric patients with grade II-IV acute graft vs. host disease	Patients with Myelofibrosis (MF)
after allogeneic hematopoietic stem cell transplantation
2023	2024
TBD	TBD

121 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

Kisqali®- CDK 4/6 inhibitor

Study	NCT03701334 NATALEE (CLEE011O12301C)
Indication	Adjuvant treatment of hormone receptor (HR)-positive,
HER2-negative, early breast cancer (EBC)
Phase	Phase 3
Patients	~4,000
Primary Outcome	Invasive Disease-Free Survival for using STEEP criteria
(Standardized Definitions for Efficacy End Points in adjuvant
Measures
breast cancer trials)
Arms/Intervention	•	Ribociclib + endocrine therapy
•	Endocrine therapy
	Pre and postmenopausal women and men with HR-positive,
Target Patients	HER2-negative EBC, after adequate surgical resection, who
	are eligible for adjuvant endocrine therapy
Expected Completion	Interim Analysis: H1-2021;Final: 2026
Publication	TBD

122 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

Kymriah®- CAR-T therapy

Study	NCT02445248 JULIET (CCTL019C2201)	NCT03568461 ELARA (CCTL019E2202)
Indication	Relapsed / refractory DLBCL	Relapsed / refractory follicular lymphoma (FL)
Phase	Phase 2	Phase 2
Patients	128	113

Primary Outcome Measures

Arms/Intervention

Target Patients

Expected Completion

Publication

Overall response rate; efficacy and safety of CTL019	Complete Response Rate (CRR)
Single-arm study of single dose of CTL019	Single-arm study of tisagenlecleucel
Adult patients with relapsed or refractory diffuse large B-cell	Adult patients with relapsed or refractory FL
lymphoma (DLBCL)
2017(actual)	Interim Analysis: Q3-2020

• Schuster et al. Presentations at ICML 2017; at EHA 2017; at ASH 2017; at ASH 2018; Borchmann et al.

Presentation at EHA 2018; Bachanova et al.	TBD
Presentation at ICML 2019

• Schuster et al. N Engl J Med.2019;380(1):45-56. doi: 10.1056/NEJMoa1804980. Epub 2018 Dec 1.

123 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

Kymriah®- CAR-T therapy

Study	NCT03876769 CASSIOPEIA (CCTL019G2201J)	NCT03570892 BELINDA (CCTL019H2301)
Indication	1stline high risk acute lymphoblastic leukemia (ALL)	2ndline Diffuse large B-cell lymphoma (DLBCL)
Phase	Phase 2	Phase 3
Patients	160	318

Primary Outcome Measures

Arms/Intervention

Target Patients

Expected Completion

5 year Disease Free Survival (DFS)	Event-free Survival (EFS)
Single-arm study of tisagenlecleucel; retreatment allowed	Tisagenlecleucel versus standard of care
	Adult patients with aggressive B-cellNon-Hodgkin
Pediatric and young adult patients with 1stline high risk ALL	Lymphoma after failure of rituximab and anthracycline-
	containing frontline immunochemotherapy
2025	H2-2021

Publication	TBD	TBD

124 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

MBG453 - TIM-3 antagonist

Study	NCT03946670 STIMULUS MDS-1 (CMBG453B12201)
Indication	Myelodysplastic syndrome
Phase	Phase 2
Patients	120
Primary Outcome	Complete Remission (CR) rate and Progression Free
Measures	Survival (PFS)
Arms/Intervention	• Experimental: MBG453 + hypomethylating agents
• Placebo comparator: Placebo + hypomethylating agents
Target Patients	Adult subjects with intermediate, high or very high risk
Myelodysplastic Syndrome (MDS) as per IPSS-R criteria
Expected Completion	H2-2021
Publication	TBD

125 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

PDR001 - PD-1 checkpoint inhibitor

Study	NCT02967692 COMBI-i (CPDR001F2301)

Indication

Phase

Patients

Primary Outcome Measures

Arms/Intervention

Target Patients

Expected Completion

Publication

BRAFV600 mutant metastatic melanoma

Phase 3

538

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

(Phase III, randomized, placebo controlled): 532

Progression-Free Survival (PFS)

• Spartalizumab 400mg i.v. Q4W + Tafinlar 150mg bid + Mekinist 2 mg
• Placebo + Tafinlar 150 mg bid + Mekinist 2 mg

Previously untreated patients with unresectable or metastatic BRAF V600 mutant melanoma

Q3-2020

Abstract submission to congress in H2-2020

126 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

Rydapt®- Multi-targeted kinase inhibitor

Study	NCT03280030 (CPKC412A2220)	NCT03591510 (CPKC412A2218)
Indication	Acute myeloid leukemia	Acute myeloid leukemia
Phase	Phase 2	Phase 2
Patients	66	50
Primary Outcome	Incidence of safety events and event free survival	Occurrence of dose limiting toxicities
Measures	Event Free Survival ( EFS)

Arms/Intervention

Target Patients

Expected Completion

•	Midostaurin 50 mg	• Chemotherapy followed by Midostaurin
•	Placebo
Newly diagnosed patients with FLT3-mutated acute myeloid	Newly diagnosed pediatric patients with FLT3 mutated acute
leukemia (AML) from pan-Asia countries	myeloid leukemia (AML)
H1-2020	H2-2022

Publication	Abstract submission to congress in H2-2020	TBD

127 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

PDR001 - PD-1 checkpoint inhibitor

Study	NCT03484923 (CPDR001J2201)
Indication	Previously treated unresectable or metastatic melanoma
Phase	Phase 2
Patients	230
Primary Outcome
Objective Response Rate (ORR)
Measures
	•	PDR001 400mg i.v. Q4W + LAG525 600 mg i.v. Q4W
	•	PDR001 400mg i.v. Q4W + capmatinib 400 mg bid orally
Arms/Intervention	•	PDR001 400mg i.v. Q4W + canakinumab 300 mg (s.c)
	Q4W

• PDR001 400mg i.v. Q4W + ribociclib 600 mg p.o QD on Days 1 to 21 of a 28-day cycle

Target Patients

Expected Completion

Publication

Adult patients with previously treated unresectable or metastatic melanoma

H2-2021

TBD

128 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

Promacta®/Revolade®- Thrombopoetin receptor agonist

Study	NCT03025698 (CETB115E2201)	NCT03988608 (CETB115E2202)
Indication	Previously untreated or relapsed/refractory severe aplastic	Previously untreated or relapsed/refractory severe aplastic
anemia or recurrent aplastic anemia	anemia or recurrent aplastic anemia
Phase	Phase 2	Phase 2
Patients	60	20
Primary Outcome	PK of eltrombopag at steady state in pediatric patients with	Hematologic response rate
Measures	SAA

Arms/Intervention

Target Patients

Expected Completion

• Eltrombopag 12.5, 25, 50, 75 mg FCT & 25 mg pFOS
• Arm B: previously untreatedSAA-hATG/cyclosporine +

eltrombopag	• Eltrombopag 25 mg film-coated tablets
• Arm A: relapsed/refractory SAA or AA:
hATG/cyclosporine + eltrombopag or cyclosporine +
eltrombopag
Pediatric patients from age 1 <18 years with	Chinese patients with refractory or relapsed severe aplastic
relapsed/refractory SAA or recurrent AA after IST or
anemia
previously untreated SAA
2025	2023

Publication

TBD

TBD

129 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

SEG101 - p-Selectin inhibitor

Study	NCT03264989 SOLACE-Adults (CSEG101A2202)	NCT03474965 SOLACE-Kids (CSEG101B2201)
Indication	Prevention of Vaso-Occlusive Crises (VOC) in patients with	Prevention of VOC in pediatric patients with SCD
Sickle Cell Disease (SCD)
Phase	Phase 2	Phase 2
Patients	55	100
Primary Outcome	PK/PD and safety of SEG101 (crizanlizumab) at 5 mg/kg	PK/PD and safety of SEG101 at 5 mg/kg
Measures
	SEG101 (crizanlizumab) at a dose of 5.0 mg/kg (or 7.5	SEG101 (crizanlizumab) at a dose of 5 mg/kg by IV infusion
Arms/Intervention	mg/kg for exploratory group) by IV infusion, ±
± Hydroxyurea/Hydroxycarbamide
	Hydroxyurea/Hydroxycarbamide
Target Patients	Adult SCD patients with VOC	Pediatric SCD patients with VOC
Expected Completion	Q4-2018(actual)	H2-2021 (pediatric patients ≥6 year old)
2022 (pediatric patients 6 months - 6 year old)
Publication	Abstract submission to congress in H1-2020	TBD

130 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

SEG101 - p-Selectin inhibitor

Study	NCT03814746 STAND (CSEG101A2301)
Indication	Prevention of Vaso-Occlusive Crises (VOC) in patients with
Sickle Cell Disease (SCD)
Phase	Phase 3
Patients	240

Primary Outcome	Rate of VOC events leading to healthcare visit
Measures

Arms/Intervention

Target Patients

Expected Completion

Publication

• Crizanlizumab 5.0 mg/kg
• Crizanlizumab 7.5 mg/kg
• Placebo

Adolescent and adult SCD patients (12 years and older)

H1-2022

TBD

131 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

Tafinlar®- BRAF inhibitor

Study	NCT01677741 (CDRB436A2102)
Indication	BRAFV600 mutant cancers
Phase	Phase 1/2
Patients	85
Primary Outcome	Safety, tolerability and pharmacokinetics
Measures

Arms/Intervention	Single-arm study of oral dabrafenib (dose based on age
and weight)
Target Patients	Pediatric subjects aged 1 year to <18 years with advanced
BRAF V600-mutation positive solid tumors
Expected Completion	Q3-2020
	• Kieran MW et al. Manuscript Clin Cancer Res; (accepted
	Q3-2019, not yet published) (PK analysis)
Publication	• Hargrave D et al. Manuscript Clin Cancer Res; (accepted
	Q3-2019, not yet published) (safety/efficacy in low-grade
	gliomas)

132 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

Tafinlar®+Mekinist®- BRAF inhibitor and MEK inhibitor

Study	NCT02684058 (CDRB436G2201)
Indication	BRAFV600 mutant gliomas
Phase	Phase 2
Patients	142
Primary Outcome
Objective response rate
Measures
Arms/Intervention	Dabrafenib + trametinib (dose based on age and weight)
	Children and adolescent patients with BRAF V600 mutation
Target Patients	positive relapsed or refractory high grade glioma (HGG) or
	BRAF V600 mutation positive low grade glioma (LGG)
Expected Completion	H2-2021
Publication	TBD

133 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

Tafinlar®+Mekinist®- BRAFV600 inhibitor and MEK inhibitor

Study	NCT02124772 (CTMT212X2101)
Indication	BRAFV600 mutant solid tumors
Phase	Phase 1/2A
Patients	142
Primary Outcome
Safety, tolerability and pharmacokinetics and clinical activity
Measures
Arms/Intervention	Trametinib (dose based on age and weight)
Dabrafenib + trametinib (dose based on age and weight)
Target Patients	Pediatric Subjects Aged 1 Month to <18 Years with
Advanced V600-Mutation Positive Solid Tumors
Expected Completion	H1-2021
Publication	Abstract submission to congress in H1-2020

134 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

Zykadia®- ALK inhibitor

Study	NCT02299505 ASCEND-8 (CLDK378A2112)
Indication	ALK activated NSCLC
Phase	Phase 2
Patients	306
Primary Outcome	Part 1: Pharmacokinetics when taken with food
Measures	Part 2: Overall Response Rate (ORR) when taken with food
	• Oral LDK378 450 mg once daily taken with food
Arms/Intervention	• Oral LDK378 600 mg once daily taken with food
	• Oral LDK378 750 mg once daily fasted
Target Patients	Adult patients with ALK-rearranged(ALK-positive) advanced non-small cell
lung cancer
	Part 1 (PK): 2016 (actual)
Expected Completion	Part 2 (ORR): Q2-2018(actual)
	Final (ORR): Q4-2019
	• Part 1 (PK): Cho BC, et al. J Thorac Oncol. 2017 Sep; 12(9) 1357-1367
Publication	• Part 2 (ORR): Cho B et al. J Thorac Oncol. 2019 Jul; 14(7) 1255-1265
	• Final (ORR): TBD

135 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

177Lu-PSMA-617 - Lu-labelled prostate specific membrane antigen (PSMA)

Study	NCT03511664 VISION (PSMA-617-01)
Indication	PSMA-positive Metastatic Castration-resistant Prostate
Cancer (mCRPC)
Phase	Phase 3
Patients	814

Primary Outcome	•	Radiographic Progression Free Survival
Measures	•	Overall Survival
Arms/Intervention	•	177Lu-PSMA-617 plus BS/BSC
•	BS/BSC alone
	Adult patients with PSMA-positive Metastatic Castration-
Target Patients	resistant Prostate Cancer (mCRPC)
Expected Completion	H2-2020
Publication	TBD

136 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

Ophthalmology

Lucentis®- Anti-VEGF

Study	NCT02375971 RAINBOW (CRFB002H2301)	NCT02640664 RAINBOW Extension (CRFB002H2301E1)
Indication	Retinopathy of Prematurity (ROP)	Retinopathy of Prematurity (ROP)
Phase	Phase 3	Phase 3
Patients	224	180
	Absence of active Retinopathy of Prematurity (ROP) and
Primary Outcome	unfavorable structural outcome at Week 24, defined as, 1)	To evaluate the visual function of patients by assessing the
survival, 2) no intervention with a second modality for ROP,	visual acuity in the better-seeing eye at the patient's fifth
Measures
3) absence of active ROP and 4) absence of unfavorable	birthday.
	structural outcome
	•	Ranibizumab 0.2 mg (up to 3 injections max)	•	Ranibizumab 0.2 mg (up to Week 40, if warranted)
Arms/Intervention	•	Ranibizumab 0.1 mg (up to 3 injections max)
•	Ranibizumab 0.1 mg (up to Week 40, if warranted)
	•	Laser therapy
Target Patients	Male and female preterm infants with bilateral retinopathy of	Male and female preterm infants with bilateral retinopathy of
prematurity (ROP) who require treatment.	prematurity (ROP) who completed RAINBOW .
Expected Completion	Q1-2018(actual)	2023
	•	EURETINA: Sep-2018
	•	AAO: Oct-2018
Publication	•	Primary manuscript published online by The Lancet in	TBD
	Sep-2019
		(https://www.thelancet.com/pdfs/journals/lancet/PIIS0140
		-6736(19)31344-3.pdf)
138 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

RTH258 - Anti-VEGF

Study	NCT02434328 HARRIER (CRTH258A2302)	NCT02307682 HAWK (CRTH258A2301)
Indication	Neovascular age-related macular degeneration (nAMD)	Neovascular age-related macular degeneration (nAMD)
Phase	Phase 3	Phase 3
Patients	743	1,082
Primary Outcome	Change in Best Corrected Visual Acuity (BCVA) from	Change in Best Corrected Visual Acuity (BCVA) from
Measures	baseline at week 48	baseline at week 48
	•	Brolucizumab (RTH258) 6 mg/50 µL	•	Brolucizumab (RTH258) 3 mg/50 µL
Arms/Intervention	•	Brolucizumab (RTH258) 6 mg/50 µL
•	Aflibercept 2 mg/50 µL
	•	Aflibercept 2 mg/50 µL
Target Patients	Subjects with exudative age-related macular degeneration	Subjects with exudative age-related macular degeneration
Expected Completion	Q1-2018(actual)	Q2-2018(actual)
	• Oral presentations including both primary endpoint and key 2nd superior anatomic outcomes at AAO meetings in Nov-
		2017 (1styear results) and Nov-2018 (2ndyear results)
	• Year 1 Manuscript: Dugel P, et al. Ophthalmology 2019 Apr 12; HAWK and HARRIER: Phase 3, Multicenter,
Publication		Randomized, Double-Masked Trials of Brolucizumab for Neovascular Age-Related Macular Degeneration.

• Abstracts submissions on superior anatomic outcomes/Fluid/PostHoc results are planned for key retinal congresses (Angiogenesis/Mac Soc inFeb-2019; WRC in Mar-2019; ARVO in April-2019; ASRC July-2019; EURETINA Sept-2019; AAO Oct-2019 and APVRS Dec-2019

139 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

RTH258 - Anti-VEGF

Study	NCT03386474 (CRTH258A2301E1)	NCT03481634 KESTREL (CRTH258B2301)
Indication	Neovascular age-related macular degeneration (nAMD)	Diabetic eye disease
Phase	Phase 3	Phase 3
Patients	150	534
Primary Outcome	Number of treatment-emergent adverse events	Change from baseline in best-corrected visual acuity
Measures	(BCVA)
	•	Brolucizumab (RTH258) 6 mg/50 µL	•	Brolucizumab (RTH258) 3 mg/50 µL
Arms/Intervention	•	Brolucizumab (RTH258) 6 mg/50 µL
•	Aflibercept 2 mg/50 µL
	•	Aflibercept 2mg/50 uL
Target Patients	Patients with neovascular age-related macular degeneration	Patients with visual impairment due to diabetic macular
who have completed the CRTH258A2301 study	edema (DME)
Expected Completion	Q3-2018(actual)	H2-2021
Publication	TBD	TBD

140 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

RTH258 - Anti-VEGF

Study	NCT03481660 KITE (CRTH258B2302)	NCT04058067 KINGLET (CRTH258B2304)
Indication	Diabetic eye disease	Diabetic macular edema
Phase	Phase 3	Phase 3
Patients	356	268
Primary Outcome	Change from baseline in best-corrected visual acuity	Change in best-corrected visual acuity (BCVA)
Measures	(BCVA)
Arms/Intervention	•	Brolucizumab (RTH258) 6 mg/50 µL	•	Brolucizumab (RTH258) 6 mg/50 µL
•	Aflibercept 2 mg/50 µL	•	Aflibercept 2 mg/50 µL
Target Patients	Patients with visual impairment due to diabetic macular	Patients with visual impairment due to diabetic macular
edema (DME)	edema
Expected Completion	H2-2021	2022
Publication	TBD	TBD

141 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

RTH258 - Anti-VEGF

Study	NCT03917472 KINGFISHER (CRTH258B2305)	NCT03802630 RAPTOR (CRTH258C2301)
Indication	Diabetic macular edema	Retinal vein occlusion
Phase	Phase 3	Phase 3
Patients	500	500
Primary Outcome	Change in best-corrected visual acuity (BCVA) from	Change from baseline in best-corrected visual acuity
Measures	baseline up to week 52	(BCVA) at week 24
Arms/Intervention	•	Brolucizumab (RTH258) 6 mg/50 µL	•	Brolucizumab (RTH258) 6 mg/50 µL
•	Aflibercept 2 mg/50 µL	•	Aflibercept 2 mg/50 µL
Target Patients	Patients with visual impairment due to diabetic macular	Adult patients with visual impairment due to macular edema
edema	secondary to branch retinal vein occlusion
Expected Completion	H2-2021	2022
Publication	TBD	TBD

142 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

RTH258 - Anti-VEGF

Study	NCT03810313 RAVEN (CRTH258C2302)	NCT04047472 HOBBY (CRTH258A2307)
Indication	Retinal vein occlusion	Macular degeneration
Phase	Phase 3	Phase 3
Patients	750	494

Primary Outcome Measures

Arms/Intervention

Change from baseline in best-corrected visual acuity	Change from baseline in best-corrected visual acuity
(BCVA) at week 24	(BCVA) at week 48
•	Brolucizumab (RTH258) 6 mg/50 µL	•	Brolucizumab (RTH258) 6 mg/50 µL
•	Aflibercept 2 mg/50 µL	•	Aflibercept 2 mg/50 µL

Target Patients	Adult patients with visual impairment due to macular edema	Chinese patients with neovascular age-related macular
secondary to central retinal vein occlusion	degeneration
Expected Completion	2023	2023
Publication	TBD	TBD

143 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

UNR844 - Disulfide bonds modulator

Study	NCT03809611 (CUNR844A2203)
Indication	Presbyopia
Phase	Phase 2
Patients	124
Primary Outcome	Change in binocular distance-corrected near visual acuity
Measures	(DNCVA) from baseline
Arms/Intervention	•	1.5% solution UNR844-Cl
•	Placebo
Target Patients	Patients with presbyopia
Expected Completion	Q1-2020
Publication	Planned in ASRCS in 2020

144 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

Respiratory

QAW039 - DP2 receptor antagonist

Study	NCT02555683 LUSTER-1 (CQAW039A2307)	NCT02563067 LUSTER-2 (CQAW039A2314)
Indication	Asthma	Asthma
Phase	Phase 3	Phase 3
Patients	846	846
Primary Outcome	Reduction in the rate of moderate-to-severe asthma	Reduction in the rate of moderate-to-severe asthma
Measures	exacerbations	exacerbations
	•	QAW039 Dose 1	•	QAW039 Dose 1
Arms/Intervention	•	QAW039 Dose 2	•	QAW039 Dose 2
	•	Placebo	•	Placebo
Target Patients	Patients with uncontrolled severe asthma	Patients with uncontrolled severe asthma
Expected Completion	Q4-2019(actual)	Q3-2019(actual)
Publication	Planned in 2020	Planned in 2020

146 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

QAW039 - DP2 receptor antagonist

Study	NCT03215758 ZEAL-1 (CQAW039A2316)	NCT03226392 ZEAL-2 (CQAW039A2317)
Indication	Asthma	Asthma
Phase	Phase 3	Phase 3
Patients	650	650
Primary Outcome
Pre-dose forced expiratory volume in 1 second (FEV1)	Pre-dose forced expiratory volume in 1 second (FEV1)
Measures
Arms/Intervention	•	QAW039	•	QAW039
•	Placebo	•	Placebo
Target Patients	Patients with uncontrolled asthma	Patients with uncontrolled asthma
Expected Completion	Q3-2019(actual)	Q3-2019(actual)
Publication	Planned in 2020	Planned in 2020

147 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

QAW039 - DP2 receptor antagonist

Study	NCT03052517 SPIRIT (CQAW039A2315)	NCT03650400 (CQAW039B2201)
Indication	Asthma	Asthma
Phase	Phase 3	Phase 2
Patients	1,900 - 2,300	24

Primary Outcome Measures

Arms/Intervention

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

SAE and AE leading to discontinuation from study (52 wks	Pharmacokinetics, safety and tolerability
and 160 wks)
•	QAW039 Dose 1	• Fevipiprant Cohort A; Fevipiprant Cohort B; Chewable
•	QAW039 Dose 2
tablet
•	Placebo

Target Patients	Patients with moderate to severe asthma	Children aged 6 to < 12 years with asthma
Expected Completion	For Submission: Q4-2019(actual);Final: 2022	Q3-2020
Publication	TBD	TBD

148 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

QBW251 - CFTR potentiator

Study	NCT04072887 (CQBW251B2201)
Indication	Chronic obstructive pulmonary disease (COPD)
Phase	Phase 2
Patients	900
Primary Outcome	Trough FEV1 (Forced Expiratory Volume in 1 second)
Measures	change from baseline after 12 weeks of treatment
	•	QBW251 450 mg
	•	QBW251 300 mg
Arms/Intervention	•	QBW251 150 mg
•	QBW251 75 mg
	•	QBW251 25 mg
	•	Placebo

Target Patients

Expected Completion

Publication

COPD patients on background triple inhaled therapy (LABA / LAMA / ICS)

H2-2021

TBD

149 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

QMF149 - Long-acting beta2 agonist and inhaled corticosteroid

Study	NCT02892019 (CQMF149G2202)
Indication	Asthma
Phase	Phase 2
Patients	80
Primary Outcome	Trough FEV1
Measures
Arms/Intervention	• Indacaterol acetate 75 μg od (via Concept1 inhaler)
• Indacaterol acetate 150 μg od (via Concept1 inhaler)
Target Patients	Children ≥ 6 to < 12 years of age with asthma
Expected Completion	Q3-2019(actual)
Publication	Planned in H2-2020

150 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

QVM149 - Long-acting beta2 agonist, Long-acting muscarinic antagonist and inhaled corticosteroid

Study	NCT02554786 PALLADIUM (CQVM149B2301)	NCT02571777 IRIDIUM (CQVM149B2302)
Indication	Asthma	Asthma
Phase	Phase 3	Phase 3
Patients	2,216	3,092
Primary Outcome
Trough FEV1	Trough FEV1
Measures
	•	QMF149 150/160 µg od	•	QVM149 150/50/160 µg od
	•	QMF149 150/320 µg od	• QVM149 150/50/80 µg od
Arms/Intervention	• MF 400 µg od	• QMF149 150/160 µg od
	• MF 400 µg bid	• QMF149 150/320 µg od
	•	Salmeterol 50 µg /fluticasone 500 µg bid	•	Salmeterol 50 µg /fluticasone 500 µg bid
	Adult and adolescent (≥12 years) patients with asthma	Adult (≥18 years) patients with asthma inadequately
Target Patients	inadequately controlled on medium/high-dose ICS or low-
controlled on medium/high-dose of LABA/ICS (GINA step ≥4)
	dose LABA/ICS (GINA step ≥ 3)
Expected Completion	Q3-2019(actual)	Q3-2019(actual)
Publication	•	Planned in H1-2020	•	Planned in H1-2020
•	Abstract: van Zyl-Smit et al, presented at BTS Dec-2019	•	Abstract ATS Q2-2020

151 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

QVM149 - Long-acting beta2 agonist, Long-acting muscarinic antagonist and inhaled corticosteroid

Study	NCT03100500 (CQVM149B1305)	NCT03100825 (CQVM149B1304)
Indication	Asthma	Asthma
Phase	Phase 3	Phase 3
Patients	51	94
Primary Outcome	Long-term safety/tolerability: Incidence and severity of	Long-term safety/tolerability: Incidence and severity of
treatment emergent adverse events during the 52 weeks	treatment emergent adverse events during the 52 weeks
Measures
study	study
Arms/Intervention	• Single arm: QMF149 150/320 μg od	• Single Arm: QVM149 150/50/160 μg od
Target Patients	Japanese patients with asthma inadequately controlled	Japanese patients with asthma inadequately controlled
Expected Completion	Q1-2019(actual)	Q2-2019(actual)
Publication	• Planned in H1-2020	• Planned in H1-2020
• Abstract for ATS in Q2-2020	• Abstract for ATS in Q2-2020

152 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

QVM149 - Long-acting beta2 agonist, Long-acting muscarinic antagonist and inhaled corticosteroid

Study	NCT02892344 QUARTZ (CQVM149B2303)	NCT03158311 ARGON (CQVM149B2306)
Indication	Asthma	Asthma
Phase	Phase 3	Phase 3
Patients	802	1,251
Primary Outcome	Trough FEV1	Non-inferiority of Asthma Quality of Life Questionnaire
Measures	(AQLQ)
	•	QMF149 150/80 µg od	• QVM149 150/50/80 μg od
Arms/Intervention	•	QVM149 150/50/160 μg od
• MF 200 µg od
	•	Salmeterol/fluticasone 50/500 μg bid + tiotropium 5 μg od
	Adult and adolescent (≥12 years) patients with mild asthma
Target Patients	inadequately controlled on low-dose ICS or low-dose	Patients with uncontrolled asthma
	LABA/ICS (Gina step 2-3)
Expected Completion	Q1-2019(actual)	Q3-2019(actual)
Publication	•	O. Kornmann et al. Respiratory Medicine 161 (2020)	•	Planned in H1-2020
• Abstract: D'Andrea et al, presented at ERS Sep-2019	•	Abstract ATS Q2-2020

153 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

Xolair ®- anti-IgE antibody

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.
	In addition to standard of care:
Arms/Intervention	•	Omalizumab per approved allergic asthma dosing table for IgE/body weight combinations
	•	Placebo

Target Patients

Expected Completion

Publication

Patients with severe Japanese cedar pollinosis, whose symptoms were inadequately controlled with current recommended therapies

Q1-2019(actual)

• Late breaking abstract was published at AAAAI (American Association of Allergy, Asthma and Immunology) annual meeting, Feb 2019
• Poster published at EAACI (the European Academy of Allergy and Clinical Immunology), Jun 2019
• Oral presentations were made at JRS (Japanese Rhinologic Society), Oct 2019, and Asian Pacific Society of Respirology congress, Nov 2019
• Planned oral/poster presentation at Japan society of Immunology & Allergology in Otolaryngology, Feb 2020
• Planned manuscript to be submitted to JACI in Practice, Q1 2020

154 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

Sandoz Biopharmaceuticals

Hyrimoz®- Biosimilar adalimumab

Study	NCT02744755 ADMYRA (GP17-302)
Indication	Immunology
Phase	Phase 3
Patients	353
Primary Outcome	Change in DAS28-CRP score from baseline to week 12 in
patients treated with GP2017 and patients treated with
Measures
Humira®
Arms/Intervention	•	GP2017
•	US licensed Humira®adalimumab

Target Patients

Expected Completion

Publication

Patients with moderate to severe active rheumatoid arthritis

2018(actual)

• Wiland, P. et al., presented at EULAR 2019
• Wiland, P. et al., BioDrugs, Q4 2019

156 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

GP2411 - Biosimilar denosumab

Study	NCT03974100 (CGP24112301)
Indication	Osteoporosis
Phase	Phase 3
Patients	522
Primary Outcome	Percent change from baseline (%CfB) in lumbar spine Bone
Measures	Mineral Density
	•	GP2411 60 mg /mL subcutaneous injection every 6
Arms/Intervention		months
•	Prolia®60 mg /mL subcutaneous injection every 6

months

Target Patients

Expected Completion

Postmenopausal women with osteoporosis

2022

Publication	Study data publications expected for 2024 and beyond

157 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

Global Health

KAF156 - Plasmodium Falciparum Inhibitor - PfCARL mediated

Study	NCT03167242 (CKAF156A2202)
Indication	Malaria
Phase	Phase 2
Patients	512
Primary Outcome	PCR-corrected adequate clinical and parasitological
Measures	response (ACPR)
Arms/Intervention	• KAF156 and LUM-SDF (different combinations)
• Coartem
Target Patients	Adults and children with uncomplicated Plasmodium
Falciparum Malaria
Expected Completion	H1-2021
Publication	TBD

159 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

KAE609 - Plasmodium Falciparum Inhibitor - spiroindolone against PfATP4

Study	NCT03334747 (CKAE609A2202)
Indication	Malaria
Phase	Phase 2
Patients	210
Primary Outcome	CTCAE grades increase from baseline in alanine
aminotransferase (ALT) or aspartate aminotransferase
Measures
(AST)
Arms/Intervention	•	KAE609
•	Coartem
Target Patients	Adults with uncomplicated Plasmodium Falciparum malaria
Expected Completion	Q4-2020
Publication	TBD

160 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation