Novartis : Q1 2020 Results Presentation

April 28, 2020 at 04:18 am EDT

Novartis AG

Investor Relations

Q1 2020 Results

Investor Presentation

April 28, 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," 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 f uture revenues from any such products; or regarding potential manufacturing or supply chain disruptions; or regarding our estimates of the impact of past and f uture COVID-19 related forward purchasing on sales and on core operating income in the future; or regarding the impact of the COVID-19 pandemic on clinical trials, and research and development timelines; or regarding potential future or pending transactions; 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; or regarding the Group's liquidity or cash flow positions and its ability to meet its ongoing financial obligations and operational needs; or regarding drug discovery collaboration efforts and support of clinical trials for existing Novartis medicines and a commitment to donate up to 130 million doses of generic hydroxychloroquine to support the global COVID-19 pandemic response. 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: disruptions of our manufacturing or supply chain impacting our ability to meet demand for our products in the f uture; liquidity or cash flow disruptions affecting our ability to meet our ongoing financial obligations and to support our ongoing business activities; uncertainties regarding the impact of past and future COVID-19 related forward purchasing on sales and core operating income in the future; the impact of the COVID-19 pandemic on enrollment in, initiation and completion of our clinical trials in the future, and research and development timelines; global trends toward healthcare cost containment, including ongoing government, payer 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 development of the products described in this presentation; the potential that the strategic benefits, synergies or opportunities expected from the acquisition of the Japanese business of Aspen Global Incorporated, and other transactions described, may not be realized or may be more difficult or take longer to realize than expected; potential adverse reactions to the transaction by customers, suppliers or strategic partners; dependence on key personnel of Aspen Global Incorporated; 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 business conditions, including the effects of and efforts to mitigate pandemic diseases such as COVID-19; 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.

Enbrel®is a registered trademark of Amgen, I nc. Humira®and Skyrizi®are registered trademarks of Abbvie Inc. Siliq®is a registered trademark Valeant Pharmaceuticals International, I nc. Stelara®, Tremfya®and Simponi®are registered trademarks of Janssen Biotech, Inc. Taltz®is a registered trademark of Eli Lilly and Company. Cimzia®is a registered trademark of UCB Group of Companies.

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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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Company overview

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Maintaining strong operational performance while supporting the global response to COVID-19

Strong operational performance	Pipeline delivering	Robust pandemic response
						Positive CHMP opinion;
Continuing operations	1	+34%		Japan approval	Stable manufacturing & supply

% cc vs. PY					100% sites operational
			Positive CHMP opinion in nr-axSpA


							Key regulatory submissions on track
					Ofatumumab	Filing accepted in US, EU
					>9,100 remote monitoring visits
+13%					New ways of working
		Inclisiran	Filing accepted in US, EU
				22%			400+ disease education online sessions in China
					HFpEF submitted in US
9%				External collaborations

			Approval in EU, JP, others3	Therapeutics Accelerator, ACTIV partnership


		Sales		Core OpInc			Clinical investigation
	Capmatinib	Priority Review
	Estimate2:			3 sponsored trials, 32 IIT proposals supported
		COVID-19 related forward purchases	TQJ230	Fast Track designation	COVID-19 response funds and donations


		Excluding COVID-19 related forward purchases
				USD 40m fund, 130m doses hydroxychloroquine

2. We provide these management estimates based on the best data available to Novartis, as we believe this information is helpful to our investors to better understand Q1 underlying business performance 3. Switzerland, Canada, Australia

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COVID-19 response

Relentless commitment to our associates, patients, HCPs and society while helping to ensure business continuity

Associates		Patients		HCPs		Society

Focusing on	Helping ensure	Embracing new	Playing a pivotal
employees'	safety and	ways of working	role in the global
health and safety	uninterrupted supply		response

Business continuity

Clinical trials

Manufacturing and supply chain

Medical and commercial activities

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Business continuity - manufacturing and supply chain

Mitigating actions in place to facilitate supply chain integrity and safeguard our people and patients

Suppliers	Own Operations		Customers

All major suppliers operational,	All internal sites operational,		Strong supply reliability performance,
no impact foreseen	stable supply outlook		all launch brands on track


		Mitigating actions

Transparency across the value chain

Close collaboration with suppliers
End-to-endsupply chain tracking
Assessment of critical materials and alternative sources

Assess and adjust inventory

Assessment of inventory levels/policies
Stock replenishments

Adapt how we operate

Supporting employee health and safety
Scenario planning and optimize capacity
Close collaboration with local HAs

Identify and secure logistics capacity

Change mode of transport,re-route,pre-book, leverage freight capacity jointly

Assess realistic customer demand

Market insights to estimate demand
Respond to shortage/buying behavior

Strong financial condition, cash collections or liquidity

<2%	>6months	>99.5%
Sales supported by APIs single-sourced	Inventory for key brands	Customer Service Level
from China and India		across Innovative Medicines YTD

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Business continuity - clinical trials

Regulatory submissions for 2020 remain on track while COVID-19 impacts on clinical trials are manageable

Key regulatory submissions on track

Inclisiran (KJX839)

Hyperlipidemia (EU)

Entresto®

HFpEF (US)

AVXS-101 IT

SMA

Alpelisib (BYL719)

PROS

177Lu-PSMA-617

mCRPC

Spartalizumab (PDR001) combo

Metastatic melanoma

Clinical trial strategy

Continuous trial-by-trial assessment of safety & data integrity

300+ trials1| 96,000 patients

Planning(25% of clinical trials1) Continue study start-up planning activities

Recruitment (22%)

Paused in affected areas, while pivoting to and fully leveraging recovering areas

Maintenance (37%)

Continue with current mitigations

Close out (16%)

Continue database lock and clinical study report submissions

Slowdowns in new enrollments of ongoing studies and start-up of new studies

Mitigation to minimize impact

Enabled by real-time digital technologies

Direct-to-patient medication delivery Home nursing services

Remote medical monitoring Virtual safety assessments

>9,100 remote monitoring visits2+2,500 users on SENSE platform <24h time to detect, evaluate, respond to site-level actions

1. GDD trials only 2. As of April 24, 2020

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Associates

Broad array of initiatives implemented to support our associates throughout the crisis

Job safety

Employee wellbeing

Ways of working & reward

No job losses related to COVID-19	12 calendar days paid leave


Paused ongoing restructurings	Childcare assistance


Give back to society		Virtual volunteering
	GlobalGiving via SPARK


Wellbeing initiatives		TIGNUM X App
	Virtual coaching sessions


Online learning		Coursera for family / friends
	Khan Academy


Digital tools for Field Force	Adapted sales incentive schemes


Increased protective measures	Recognition payment

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HCPs and patients

Quickly embraced new ways of working with HCPs and patients

Patients	Prioritized patient-oriented	HCPs	Scaled up multi-channel
	digital solutions		engagement

Access to online drug refill

China: partnered with top 3 e-pharmacies

Online disease education live broadcasting

China: ~12m people, 400+ sessions

Access to direct-to-patient services US: Patient platform Phreesia processing ~60m patient intakes annually

Supporting patient organizations

Web meetings

China: 900k HCPs in 31k web meetings1

South Korea: 20x participants increase in webinars

Rep-triggered WeChat and email

China: engaged ~64k HCPs1

Free licenses for remote detailing Veeva Engage licenses

HCP portal

Partnerships with P2P HCP and in-workflow platforms

US: Doximity with >1m HCPs

1. From February 1 to April 17

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Society

Playing an important role in addressing COVID-19 pandemic to make a material difference

COVID-19 response funds and donations

USD 40mCOVID-19 Response Funds

Hydroxychloroquine: commitment

to donate 130m dosesthrough May, reaching over 60 countries, 50m doses shipped to date to 25countries.

External collaborations

Co-chairedCOVID-19

Therapeutics Accelerator under

coordination of Bill & Melinda GatesFoundation

Member ofCOVID-19 direct

partnershiporganized by Innovative Medicines Initiative

Member ofACTIV¹ partnership

planned by NIH²

Member ofR&D Leaders

Consortium

Internal discovery

Launched drug discovery efforts including collaborationwith University of California, Berkeley

Screening selected chemical librariesinternally for potential antiviral activity

Identified partners to contribute our unique biomarker capabilities and

expertise

Clinical investigations

Novartis-sponsored studies

Investigator-Initiated Trials (IIT) proposals supported

Approved Managed Access requests and institution /government requests

1. Accelerating COVID-19 Therapeutic Interventions and Vaccines. 2. NIH: National Institutes of Health

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Society - clinical investigations

Supporting clinical investigations for promising drugs both company-led and investigator-initiated

Novartis-sponsored	32 IIT proposals supported
Phase 3 studies	including:
Canakinumab	Secukinumab
(Cytokine storm)	(Cytokine storm)
Ruxolitinib¹	Imatinib
(Cytokine storm)	(SARS-CoV replication)
Hydroxychloroquine	Valsartan
(Anti-viral and immunomodulator)	(ACE2 expression)
	Omalizumab
	(Antiviral effect)

Access initiatives

IP initiative to support broad hydroxychloroquine access if approved for COVID-19 (e.g. non-exclusive voluntary licenses)

Individual MAP requests approved in ~4 hours

697 individual MAP requests and

23 institution / government requests approved2

As of April 24, 2020 1. In collaboration with Incyte 2. Canakinumab, ruxolitinib, tesidolumab/LFG316

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Monitoring potential impact of the pandemic

Patient / physician	Payor / healthcare	Clinical trial /
dynamics	system dynamics	regulatory dynamics

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Strong operational performance from growth drivers

Key growth driver sales Q1 2020

Sales	Growth vs. PY	Growth vs. PY
USD Million	USD Million	cc

Key growth drivers and launches, as % of Innovative Medicines sales

569		212	62%
170		170	nm
930		139	19%
403	96		33%
90	90		nm
74	74		nm
161	70		82%
366	69		26%
68	68		nm
213	62		44%
318	60		27%
93	48		109%

nm - not meaningful

46%

36%

29%

23%

Q1 2017

Q1 2018

Q1 2019

Q1 2020

1. Includes Tasigna®, Xolair®, Aimovig®and Luxturna®

Adakveo®

Mayzent®

Beovu®

Piqray®

Xiidra®

Kymriah®

Lutathera®

Kisqali®

Zolgensma®

Ilaris®

Jakavi®

Tafinlar+Mekinist®

Promacta®

Entresto®

Cosentyx®

Other1

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Sandoz: Strong underlying momentum in Q1, further benefiting from COVID-19 related forward purchasing

Biopharmaceutical sales

USD million

+31% cc

450

335351

Q1'18 Q1'19 Q1'20

Performance

Sales of USD 2.5bn +11% cc (includingCOVID-19 related forward purchasing), driven by biosimilars which continue to grow strongly
Europe sales USD 1.4bn +19% cc
Strong underlying operational results
COVID-19supports Q1 retail growth
Successful ongoing Sandoz and NTO transformation with core gross margin improvement and functional cost decline

US divestment to Aurobindo

Mutual agreement to terminate
Opportunity to optimize US business

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Zolgensma®: US growth momentum continues; robust IV data presented, CHMP positive opinion

Q1 highlights

USD 170m	Continued broad access & patient
	demand drove Q1 sales
	Intravenous data showed significant,
	clinically meaningful benefit including
	prolonged event-free survival, motor
	milestone achievement and durability
	up to 5 years post-dosing
	FDA completed review of its August
	2019 Form 483 response with no
	further enforcement action

Regulatory milestones

CHMP positive opinion (March)

EC decision confirming approval expected by June 2020

Japan approval (March)

Reimbursement expected by the end of

H1 2020, pending agreement

OthersDecisions anticipated late 2020 or early 2021 in Switzerland, Canada, Australia, Argentina, South Korea, Brazil

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AVXS-101 IT: Compelling clinical profile; regulatory engagement ongoing

STRONG data demonstrates compelling profile

Next steps

Strong efficacy

Robust response

Replaces chronic administration

Safety profile

With a mean 6-point increase in Hammersmith1, twice the clinically meaningful threshold

With nearly all (92%) achieving a clinically meaningful response

With a single, one-time dose

Consistent with IV AVXS-101 program

Additional pre-clinical data requested by FDA to lift IT clinical hold expected to be generated in studies planned / initiated

Plan to engage with FDA Q2 to clarify scope of data required

Plan to approach FDA for pre-BLA meeting based on STRONG data, which confirm the positive benefit/risk of IT formulation

BLA submission timing: dependent on FDA feedback, could range from H2 2020 to 2021

1. Efficacy data reflective of patients between 2-5 years of age who received Dose B

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On track for 2020 catalysts

Maintaining long-term momentum

Potential catalysts

Major approvals1

Major submissions3

Major readouts5(Phase 3)

Phase 3 starts

Selected examples

Ofatumumab (OMB157)	Capmatinib (INC280)7	Inclisiran (KJX839)
Relapsing MS	NSCLC	Hyperlipidemia (US)
QVM / QMF 149	Cosentyx®2
Asthma	nr-AxSpA

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 (IA)
Asciminib (ABL001)	Kisqali®	Jakavi®
Chronic Myeloid Leukemia	Breast cancer(MONALEESA-2 OS)	Chronic GvHD

TQJ2308	LNP023	MBG453
CVRR	PNH	MDS
Tropifexor (LJN452)	Alpelisib (BYL719)	Beovu®
NASH	Multiple indications6	PDR

1. First approval in any market. 2. Positive CHMP received	3. First submission in any market	4. FDA placed a partial clinical hold based on findings in a small preclinical animal study	5. Readouts enabling submission, label change
or pivotal trial initiation 6. HER2+ aBC, TNBC, ovarian cancer, head and neck cancer 7. Received FDA Priority Review designation 8. Received FDA Fast Track designation

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Strong Q1 performance and news flow, setting Pharmaceuticals up for continued growth in 2020

Pharmaceuticals net sales	Strong underlying momentum across portfolio
(incl. COVID-19 related forward purchasing)		Solid demand for growth drivers and established brands
USD billion, growth in % cc

		+14%						Q1 COVID-19 effects net positive, expected to reverse
		6.1				Benefit of forward buying for orals / self-administered

								therapies, expected to reverse in later quarters

5.5					0.4			
						Negative impact on HCP-administered products
1.6			2.0
				Rich newsflow on launch brands

3.9			3.7				Beovu®approved in EU and JP
				Positive CHMP opinion for Cosentyx®nr-axSpA

									Inclisiran file accepted in US and EU
	Q1 2019	Q1 2020		Ofatumumab submitted in EU
	Growth drivers1		Recent launches2			Established products3

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

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Cosentyx®: Solid start in 2020 with 19% YoY growth and further evidence of efficacy in joints

Sales evolution

USD million, % cc

Ex-US

+19%

930

791

354

317

474576

Q1 2019

Q1 2020

Strong underlying demand across indications

PsO TRx +27% YoY vs. market +17%1
SpA TRx +30% YoY vs. market +13%2

Strengthened value proposition

Positive CHMP opinion fornr-axSpA
FDA approval forup-titration to 300mg in AS
EMA submission of 300mg / 2mL PFS and autoinjector
ULTIMATE showed early significant effect on joint synovitis

Significant additional growth potential

nr-axSpAlaunch expected Q2, completing axSpA spectrum
Large remaining biologic penetration potential in all indications3

1. IQVIA National Prescription Audit for Dermatology WE 03/27/2020; market includes Enbrel®, Humira®, Siliq®, Skyrizi™, Stelara®, Taltz®, Tremfya®. 2. IQVIA National Prescription Audit for Rheumatology WE 03/27/2020; SpA market includes Cimzia®, Enbrel®, Humira®, Simponi®, Stelara®, Taltz®. 3. PsO: Prevalent to mod+ severe Treated pool is from DRG; Bx treated : DRG + IQVIA patient equivalents. PsA and Axial SpA: Epidemiology, diagnosed, treated and Bx pool and aligned with DRG, latest country inputs (internal assumption based multiple data sources).

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Entresto®: Strong Q1 fueled by demand acceleration in key markets

Sales evolution

USD million, % cc

Ex-US

		+62%

		569
		276
357
158
199	293


Q1 2019	Q1 2020
1. IQVIA NPA - TRx March '20;	2. DRG, IQVIA; NRDL- National Reimbursement Drug List.
Ejection Fraction

Strong momentum across geographies

All-timehighs in US NBRx >4,500, TRx +46% YoY1
Strong acceleration in China following NRDL listing
Co-promotionagreement with Otsuka in JP, ahead of expected launch in H2 2020

Poised for continued growth

~75% of 3.4m eligible HFrEF patient population remaining in G72
Submitted HFpEF file to FDA
PARADISEpost-AMI on track for readout mid-2021

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

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Beovu®: Strong initial uptake based on efficacy. Working with RS community to understand rare safety signal1

After thorough SRC review, Novartis continues to consider benefit-risk to be positive

Retinal vasculitis2	1.30/ 10,000 injections
Retinal vascular occlusion3	1.95/ 10,000	injections

Retinal vasculitis + retinal	2.60/ 10,000	injections
vascular occlusion2

Rare safety signal1transparently communicated
Updating safety information in Beovu®PI

Continuing root-cause analysis/ clinical trial program and progressing access to Beovu®

Extensive investigation in collaboration with external experts to identify root cause of signal
Continuing most comprehensive Ph3/4 aVEGF clinical trial program to date
DME studies and MERLIN fully recruited, other studies subject to evolution ofCOVID-19
Approved in top 9ex-US markets4in Q1 2020
Long exclusivity in US and EU

SRC = Safety Review Committee; PI = Prescribing Information; IOI = intraocular inflammation; specific diagnoses vary depending on the exact location in the eye and can include iritis and uveitis, among others; DME = Diabetic Macular Edema;

1. Retinal vasculitis and/or Retinal vascular occlusion that may result in severe vision loss. Typically these events occur in the presence of IOI. brolucizumab.info will be updated to reflect these updated rates regularly.Event rates are discrete: There is no double counting between categories. 2. Inflammation of retinal blood vessels that can be a specific diagnosis or part of localized (e.g., IOI) or systemic inflammatory disorder. For some of the cases assessed, it was not clear whether the occlusion was of arterial and/or venous origin. Events typically occur in the presence of IOI. 3. Blockage of any retinal blood vessel - artery or vein - due to any number of causes. Includes physician reports of retinal artery occlusion, retinal artery thrombosis, retinal artery embolism, retinal ischaemia, arterial occlusive disease and retinal vascular occlusion. 4. EU5, UK, CH, JP, Canada

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Ophthalmology franchise severely affected by COVID-19, with significant impact on prescriptions across conditions

Franchise sales evolution

USD million, % cc

	+5%

	1,196
1,161
		68		Beovu®

533	90				Xiidra®


	487		Lucentis®

628

551Other

Q1 2019

Q1 2020

Significant impact of COVID-19 on ophthalmology care

Clinic shutdowns, emergency only appointments, elective surgeries postponed, reduced patient flow
"Injection-only"visits fixed schedule visits1

Reduced prescriptions in the 4 weeks ending April 103

aVEGF scripts	Down 15%TRx, down 12%NBRx2
Dry eye disease	Down 6%TRx, down 46%NBRx2
Other ophthalmology	Down 33%TRx2

1. Retina Society recommendations 2. IQVIA prescription data; w/e 10 April 2020 3. 4 weeks average vs. prior 4 weeks average

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Ofatumumab: Has potential to set a new standard for simple, broad and early B-cell therapy use in RMS

Can provide broad and early high efficacy treatment for RMS

Potential to be first choice for broad range of RMS patients

	Powerful sustained efficacy	MS market volume	mAbs
	Favorable safety	share by class3

		22%
	Precise and targeted B-cell therapy
	44%Orals
	Flexibility through at home self-administration

Based on strong ASCLEPIOS I&II data	34%
Superior efficacy for relapses, MRI activity
BRACE
Substantial reductions in disability progression1

Lower levels of NfL2
No significant signals of infections/ malignancies

PDUFA date June 2020, CHMP expected Q1 2021

1. CDW, confirmed disability worsening and CDP, confirmed disability progression are interchangeable terms, defined by an increase ≥1.5 EDSS points for patients with baseline EDSS of 0, increase of ≥1.0 EDSS points patients with baseline EDSS of 1.0-5.0 and increase of t ≥0.5 EDSS points for patients with baseline EDSS of 5.5 2. NfL levels at month three measured as adjusted geometric mean levels and difference is geometric mean ratio (GMR) 3. MS Market = BRACE + Orals + mAbs ; Volume = Standard Units converted to days of therapy (DOT); DOT normalizes dosing schedules to be comparable for different therapies Source: IQVIA PADDS

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Inclisiran: Preparing to launch first-in-class siRNA cholesterol-lowering treatment with twice yearly dosing

Persistent and underserved patient population in ASCVD

#1	CV disease and greatest cause of death
globally1
50m	Patients with ASCVD or FH in key markets2
60%	Patients treated with statins do not meet goal3
and are at risk of LDL-C accumulation over time

On track for approval as early as December 2020

Regulatory submissions accepted by FDA/ EMA
ORION-9,ORION-10,ORION-11 published in NEJM4
- durable and potentLDL-C reduction up to 52%
- twice yearly dosing
- administration by HCP
Progressing health systems partnering to accelerate access and improve patient outcomes

CV = Cardiovascular; ACVD = Aherosclerotic Cardiovascular Disease; FH = Familial Hypercholesterolemia; LDL-C = Low Density Lipoproein Cholesterol; HCP = Healthcare Professional; 1. McClellan M, et al. Circulation. 2019;139:e44-e54,

2. DRG (2019); 3. Boekholdt et al. Very Low LDL-C levels and CVD Risk JACC VOL 64.No 5 2014:485-94. 4. Raal FJ., et al. NEJM. March 18 2020. DOI: 10.1056/NEJMoa1913805, Ray K., et al. NEJM. March 18, 2020. DOI:10.1056/NEJMoa1912387

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Recent launches and growth drivers off to a strong start in Q1 2020

Oncology net sales

(incl. COVID-19 related forward purchasing) USD billion, % cc

			+12%	3.6


						0.4
3.3
0.2
0.9			1.1


		2.2
						2.1


	Q1 2019				Q1 2020
	Recent launches1		Growth drivers2		Base business3

Q1 key highlights

Strong uptake of recent launches, including Piqray®and Adakveo®, as well as Kisqali®(USD 161m, +82% cc)
Growth drivers continueddouble-digit performance, led by Promacta®/Revolade®(USD 403m, +33% cc), Tafinlar®+ Mekinist®(USD 366m, +26% cc)
Net positive impact fromCOVID-19-related forward purchasing, expected to reverse in later quarters
Delivered strong growth despite significant Gx erosion

1. Recent launches include Kisqali®, Kymriah®, Lutathera®, Piqray®, Adakveo®2.Growth drivers include Promacta®/Revolade®, Jakavi®(marketed by Novartis ex-US), Tafinlar®+ Mekinist®.

3. Base business - other brands.

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Piqray®: Growth momentum reflects strong launch execution and clear unmet need

Net sales	74
67
USD million

	43

Q2'19 Q3'19 Q4'19 Q1'20

Q1 sales driven by expanded coverage and strong Rx momentum; blockbuster potential in current indication alone
Continued uptake in PIK3CA testing, with goal to reach a rate of 40% by YE 2020
Foundation Medicine PIK3CA CDx plasma anticipated Q2 2020
Expanding geographical footprint with approvals in 13 markets; CHMP opinion expected Q2 2020
Progressing with "EPIK" development programs: study protocols for HER2+ advanced BC, TNBC, ovarian cancer, PROS1have been aligned with the FDA

1. RWE study protocol

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Adakveo®: Off to a strong start in the US with net sales of USD 15m in Q1

Strong uptake in Q1

320	Accounts have ordered Adakveo®and
	~75% have repeated orders
60%	Of high-volume accounts1have ordered
Adakveo®, while 40% await P&T review
96%	Brand awareness among surveyed
	hematologists

Payer coverage and further expansion

C / JC code as of April 1, J code as of July 1, improving

	reimbursement confidence
12	State Medicaid programs from the top 23 states for
SCD prevalence have published policies

2	Ex-US approvals (Brazil, India)
EU approval expected H2 2020

SCD - Sickle cell disease 1. High patient volume accounts defined as accounts with >200 patients

30 Novartis Q1 Results | April 28, 2020 | Novartis Investor Presentation

Financial review and 2020 guidance

31 Novartis Q1 Results | April 28, 2020 | Novartis Investor Presentation

Strong underlying performance and COVID-19 related forward purchasing drove Q1 results

Continuing operations1	Q1	Change vs. PY
USD million	2020	% USD	% cc2
Net Sales	12,283	11	13

Core Operating income 2	4,177	28	34
Operating income	2,744	22	30

Net Income	2,173	16	24

Core EPS (USD)2	1.56	29	34
EPS (USD)	0.96	19	27

Free Cash Flow 2	2,021	8

Excluding COVID-19 related forward purchases and lower than expected spend, we estimate3:

Sales growth to be approximately9% (cc)
Core operating income growth to be approximately22% (cc)

The COVID-19 related impacts, +USD 0.4bnon sales and core operating income, are expected to reverse in the remainder of 2020

1. Refers to continuing operations as defined on page 33 of the Condensed Interim Financial Report, excludes Alcon, includes the businesses of Innovative Medicines and Sandoz, 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 43 of the Condensed Interim Financial Report 3. We provide these management estimates based on the best data available to Novartis, as we believe this information is helpful to our investors to better understand Q1 underlying business performance

32 Novartis Q1 Results | April 28, 2020 | Novartis Investor Presentation

Strong underlying performance excluding COVID-19 benefits with core margin expansion of +3%pts

Continuing operations1

			3
2	2	2	2
	2		2
2	2	2	2

The COVID-19 related impacts are expected to reverse in the remainder of 2020

33 Novartis Q1 Results | April 28, 2020 | Novartis Investor Presentation

Operational sales momentum expected to remain throughout the year while investing in new launches

Sales growth (cc), illustrative

13%			Mainly lapping Xiidra®
	9%	acquisition and H2

	2019 launches

Mid to high single digit

Q1 2020	COVID-19 Stocking	Q1 vs. PY excl.	Gx Erosion	Others	FY 2020
vs. PY	normalization	COVID-19 estimate1			vs. PY

Core OpInc growth (cc), illustrative

34%	Sales and investments

22%	in upcoming launches
including Inclisiran
	High single to
	low double digit

Q1 2020	COVID-19 Stocking	Q1 vs. PY excl.	Gx Erosion	Launches &	FY 2020
vs. PY	normalization	COVID-19 estimate1		Growth Drivers	vs. PY

1. We provide these management estimates based on the best data available to Novartis, as we believe this information is helpful to our investors to better understand Q1 underlying business performance

34 Novartis Q1 Results | April 28, 2020 | Novartis Investor Presentation

FY 2020 key assumptions

Barring unforeseen events (in cc)

Continuing operations full year guidance key assumptions

Sales and core		Retaining the Sandoz US Oral Solids and Dermatology businesses
operating income		impacts sales and core operating income growth by approximately -1%pt
	Return to normal prescription and consumption dynamics during Q2 in
		our major markets
		No Gilenya®or Sandostatin®LAR generics enter in 2020 in the US
Core net		Expenses expected to increase by around 0.2bn vs. 2019 reflecting
financial result		additional financing costs to acquire The Medicines Company

We will closely monitor the business dynamics and provide any additional guidance at Q2 earnings

35 Novartis Q1 Results | April 28, 2020 | Novartis Investor Presentation

2020 Novartis full year guidance confirmed

Barring unforeseen events; growth vs. PY in cc

Continuing operations | full year guidance1

Including the expected impact from retaining the Sandoz US oral solids & dermatology businesses2

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

1. Includes the forecast assumption that we see a return to normal prescription and consumption dynamics during Q2 in our major markets. The guidance also includes the forecast assumption that no Gilenya®and no Sandostatin®LAR generics enter in 2020 in the US 2. 1%pt negative impact on both sales and core operating income growth

36 Novartis Q1 Results | April 28, 2020 | Novartis Investor Presentation

Q1 free cash flow increased to USD 2bn

Continuing operations1free cash flow2

USD billion

		+8%		Key drivers vs. PY:
		2.0	+Higher operating income

1.9
	(adjusted for non-cash items)

−

Higher working capital*

Accounts receivables, supporting sales growth

Accounts payables, due to lower spending

Q1 2019

Q1 2020

*Overall cash conversion cycle measures broadly in line with historical average

Refers to continuing operations as defined on page 33 of the Condensed Interim Financial Report, excludes Alcon, includes the businesses of Innovative Medicines and Sandoz, as well as the continuing corporate functions
Free cash flow is anon-IFRS measure. An explanation of non-IFRS measures can be found on page 43 of the Condensed Interim Financial Report

37 Novartis Q1 Results | April 28, 2020 | Novartis Investor Presentation

Expected currency impact for full year 2020

Currency impact vs. PY

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

FX impact on net sales

FX impact on core operating income

-3	-2	-4	-3
	-5
			-6		-6
				-7


FY	Q1	Q2	FY	FY	Q1	Q2	FY

2019

2020

2019

2020

Actual Simulation

38 Novartis Q1 Results | April 28, 2020 | Novartis Investor Presentation

39 Novartis Q1 Results | April 28, 2020 | Novartis Investor Presentation

Conclusion

Advancing broad range of efforts with our associates, patients, HCPs and society to support the global response toCOVID-19
Continuing to deliver our medicines and advance our innovative pipeline as reflected in our strong operational performance in Q1
Maintaining our full year outlook

40 Novartis Q1 Results | April 28, 2020 | Novartis Investor Presentation

Appendix

Net debt increased by USD 13.9bn mainly due to acquisitions and the annual dividend payment

-13.9


-15.9


			-7.0

										0.3	-29.8
									0.7

					-9.9	2.0

Dec 31, 2019	Dividends	M&A	Free Cash	Treasury share	Others	Mar 31, 2020
					transactions1		Flow	transactions, net

1. Mainly the acquisition of The Medicines Company for USD 9.6bn (excluding cash acquired of USD 0.1bn)

42 Novartis Q1 Results | April 28, 2020 | Novartis Investor Presentation

2020 expected pipeline milestones

	H1 2020			H2 2020	✓Achieved	✕Missed
Regulatory	Beovu®	nAMD (EU/JP)	✓	Adakveo®	Sickle cell disease (EU)
Cosentyx®	nr-axSpA (EU/US)	✓2	Capmatinib (INC280)	NSCLC (US/JP)
decisions and
Cosentyx®	AS (CN)		Cosentyx®	Pediatric psoriasis (EU)
opinions
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 (EU/JP)	✓3
Major	Entresto®	HFpEF (US)	✓	Alpelisib (BYL719)	PROS (US)
Inclisiran (KJX839)	Hyperlipidemia (EU)	✓	AVXS-101 IT4	SMA (US)
expected
				Juvenile PsA / enthesitis-related
submissions				Cosentyx®
			arthritis (US/EU)
				Spartalizumab (PDR001)	Metastatic melanoma (US/EU)
				and Tafinlar®& Mekinist®

				177Lu-PSMA-617	mCRPC (US)
Major	Entresto®	Post-acute MI		Asciminib (ABL001)	CML 3L
expected trial	Tropifexor (LJN452)	NASH		Beovu®	DME
readouts1	UNR844	Presbyopia	✓	Jakavi®	chronic GVHD
			Kisqali®	aBC (MONALEESA-2 OS)

				177Lu-PSMA-617	mCRPC
1. Achieved = on-time readout of data, irrespective of trial outcome	2. Positive CHMP received, filing underway in US	3. Positive CHMP received, JP approval received	4. Now expected to file H2 2020 to H1 2021

43 Novartis Q1 Results | April 28, 2020 | Novartis Investor Presentation

Our pipeline projects at a glance

Phase 1/2	Phase 3	Registration	Total

O N C O LO G Y	52	21	3	76


P H A R MA C E U T IC A LS	58	20	11	89

Cardiovascular, Renal, Metabolism	12	5	1	18

Immunology, Hepatology, Dermatology	22	5	3	30

Neuroscience	5	3	2	10

Ophthalmology	5	3	1	9

Respiratory	8	2	3	13

Global Health	6	2	1	9

B IO S IMILA R S	0	1	0	1
Total	110	42	14	166
CRM: Cardiovascular, Renal & Metabolism. IHD: Immunology, Hepatology & Dermatology.	NS: NeuroScience.

44 Novartis Q1 Results | April 28, 2020 | Novartis Investor Presentation

Novartis submission schedule

New medical entities: lead and new indications

2020

2021

2022

2023

≥2024

TQJ230

spartalizumab

Lead

asciminib

Lead

ECF843

Lead

LAG525

Lead

177Lu-PSMA-R2

Lead

ianalumab

Lead

UNR844

Lead

PDR001

ABL001

Dry eye

Solid Tumors

177Lu-PSMA-R2

VAY736

Presbyopia

CVRR-Lp(a)

m BRAF V600+ melanoma

(+Taf/Mek)

CML 3L

Prostate cancer

AIH

ganaplacide

177Lu-PSMA-617

Lead

MBG453

Lead

LOU064

Lead

177Lu-NeoB

Lead

LNA043

Lead

CPK850

Lead

INDICATIONS

177Lu-PSMA-617

HR-MDS

Chronic spontaneous urticaria

177Lu-NeoB

Osteoarthritis

KAF156

mCRPC 3L

Multiple Solid Tumors

Malaria uncomplicated

ligelizumab

Lead

iscalimab

Lead

VPM087

Lead

tropifexor

Lead

LMI070

Lead

cipargamin

QGE031

CFZ533

1st line CRC / 1st line RCC

LJN452

SMA

KAE609

Chronic urticaria

Renal Tx

NASH

Malaria severe

CSJ117

Lead

adriforant

Lead

tropifexor&cenicriviroc

Lead

MIJ821

Lead

LXE408

Severe asthma

ZPL389

LJC242

Depression

Visceral leishmaniasis

Atopic dermatitis

NASH

LEAD

LNP023

Lead

CEE321

Lead

SAF312

Lead

QBW251

Lead

PNH

Atopic Dermatitis

COSP

COPD

AVXS-201

Lead

OAV201

Rett syndrome

canakinumab

LCM

canakinumab

LCM

capmatinib

LCM

spartalizumab

LCM

LOU064

LCM

LNP023

INDICATIONS

ACZ885

LCM

INC280

LCM

PDR001

LCM

SjS

iMN

LNP023

MBG453

ianalumab

NSCLC 2L

Adjuvant NSCLC

Solid tumors

Malignant melanoma (combo)

canakinumab

LCM

LNP023

LCM

crizanlizumab

LCM

iscalimab

Lead

tropifexor

LCM

inclisiran

ACZ885

C3G

SEG101

CFZ533

LJN452

KJX839

NSCLC 1L

Sickle cell anaemia w ith crisis ped

Liver Tx

NASH (combos)

CVRR-LDLC

LNP023

LCM

MBG453

LCM

iscalimab

LCM

ofatumumab

LCM

cipargamin

IgAN

Maintenance for MRD+ AML

CFZ533

OMB157

KAE609

SjS

Ped MS

Malaria uncomplicated

NEW

aHUS

Unfit AML

VAY736

pSjS

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

45 Novartis Q1 Results | April 28, 2020 | Novartis Investor Presentation

Lead

LCM

Novartis submission schedule

Supplementary indications for existing brands

2020

2021

20222023

≥2024

alpelisib, BYL719	LCM	Kymriah
PROS		tisagenlecleucel-T, CTL019
		r/r DLBCL 1st relapse

LCM

KisqaliLCM

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

PiqrayLCM

alpelisib, BYL719

TNBC

PiqrayLCM

alpelisib, BYL719 HNSCC 2/3L

Kymriah

tisagenlecleucel-T, CL019 r/r DLBCL (+ pembro)

LCMJakavi

ruxolitinib, INC424 Pediatrics Chronic GVHD

LCM	Cosentyx	LCM
	secukinumab, AIN457
	Lupus Nephritis

CosentyxLCM

secukinumab, AIN457 Psoriasis 2ml Auto-injector

Cosentyx	LCM
secukinumab, AIN457
PsA H2H

Cosentyx US	LCM
secukinumab, AIN457
Ped Psoriasis

AVXS-101LCM

onasemno-geneabepar-vovec, OAV101

SMA IT

XolairLCM

omalizumab, IGE025 CSU (for CN)

EntrestoLCM

valsartan+sacubitril, LCZ696 HFpEF

Kymriah

tisagenlecleucel-T, CTL019 r/r Follicular lymphoma

Tafinlar

dabrafenib, DRB436 HGG/LGG - Pediatrics

Promacta

eltrombopag, ETB115

Food effect free formulation

Jakavi

ruxolitinib, INC424

Steroid refractory chronic GVHD

Jakavi

ruxolitinib, INC424

Steroid refractory acute GVHD

Beovu

brolucizumab, RTH258

DME

Xolair

omalizumab, IGE025 Food allergy

Xolair

omalizumab, IGE025 Auto-injector

Entresto

valsartan+sacubitril, LCZ696 Post-AMI

Lamprene US

clofazimine, LAM320 Tuberculosis

LCM

PromactaLCM

eltrombopag, ETB115 Radiation sickness syndrome

AdakveoLCM

crizanlizumab, SEG101

Sickle cell anaemia new formulations

Cosentyx	LCM
secukinumab, AIN457
SpA IV

Cosentyx	LCM
secukinumab, AIN457
Hidradenitis suppurativa

Cosentyx	LCM
secukinumab, AIN457
AS H2H

Entresto EUa	LCM
valsartan+sacubitril, LCZ696
Pediatric HF

PiqrayLCM

alpelisib, BYL719 HER2+ adv BC

PiqrayLCM

alpelisib, BYL719 Ovarian cancer

KymriahLCM

tisagenlecleucel-T, CTL019 Adult r/r ALL

TafinlarLCM

dabrafenib, DRB436 Tyroid cancer

BeovuLCM

brolucizumab, RTH258 Diabetic retinopathy

BeovuLCM

brolucizumab, RTH258

RVO

CoartemLCM

artemether + lumefantrine, CCA566 Malaria uncomplicated, <5kg patients

denosumabBioS

GP2411

anti RANKL mAb

JakaviLCM

ruxolitinib, INC424 Pediatrics Acute GVHD

KymriahLCM

tisagenlecleucel-T, CTL019

1L high risk ALL, pediatrics & young adults

Lutathera

177Lu-oxodotreotideb)GEP-NET 1L

Jakavi

ruxolitinib, INC424 Myelofibrosis (combination)

LCMCosentyx

secukinumab, AIN457

GCA

LCMCosentyx

secukinumab, AIN457 Lichen Planus

LCM	Mayzent	LCM
	siponimod, BAF312
	Ped MS

LCM

Approved in US
177Lu-dotatate in US

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

46 Novartis Q1 Results | April 28, 2020 | Novartis Investor Presentation

Novartis pipeline in registration

6 lead indications

Lead indication

Oncology

Code	Name	Mechanism	Indication(s)
BYL719	Piqray	PI3Kα inhibitor	PIK3CA mutant HR+, HER2 (-) postmenopausal adv BC 2nd line
(+fulv)

INC280	capmatinib	Met Inhibitor	NSCLC
SEG101	Adakveo®	P-selectin Inhibitor	Sickle cell disease

Immunology, Hepatology, Dermatology

Code	Name	Mechanism	Indication(s)
AIN457	Cosentyx	IL17A Inhibitor	Ped Psoriasis	nr-axSpA	Psoriasis 2ml Auto-injector

Ophthalmology

Code	Name	Mechanism	Indication(s)
LIF606	Xiidra EU	LFA-1 antagonist	Dry Eye

Neuroscience

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

Respiratory Disease

Code	Name	Mechanism	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	Tuberculosisa)

a) WHO Pre-Qualification

47 Novartis Q1 Results | April 28, 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
177Lu-	Lutathera®	Targeted Radioligand Therapy	GEP-NET 1L
oxodotreotide 3)
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

Immunology, Hepatology, Dermatology

Code	Name	Mechanism	Indication(s)
AIN457	Cosentyx	IL17A Inhibitor	Lupus	Hidradenitis	AS H2H	SpA IVIV
			Nephritis	suppurativa
QGE031	ligelizumab	IgE Inhibitor	Chronic spontaneous urticaria

Ophthalmology

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

FDA placed a partial hold onAVXS-101 intrathecal clinical trials for SMA patients based on findings in a small pre-clinical animal study
Approved in US
177Lu-dotatate in US

Neuroscience

Code	Name	Mechanism	Indication(s)
BAF312	Mayzent®	S1P1 Modulator	Ped MS
OAV101	AVXS-101	Survival motor neuron protein	SMA IT 1)
		gene therapy
OMB157	ofatumumab	CD20 Antagonist	Ped MS

Respiratory Disease

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

Cardiovascular, Renal, Metabolism

Code	Name	Mechanism	Indication(s)
KJX839	inclisiran	siRNA (PCSK9)	CVRR-LDLC
LCZ696	Entresto®	AT-II / NEP,NEP,AGTR1,AGTR2 Inhibitor	Post-AMI	Pediatric HF 2)	HFpEF
TQJ230	TQJ230	Anti-Apo(a) ASO targeting Lp(a)	CVRR-Lp(a)

Global Health

Code	Name	Mechanism	Indication(s)
COA566	Coartem®	-	Malaria uncomplicated, <5kg patients
LAM320	Lamprene®	SMPD1 Inhibitor	Tuberculosis US

Biosimilars

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

48 Novartis Q1 Results | April 28, 2020 | Novartis Investor Presentation

Novartis pipeline in Phase 2

30 lead indications

Lead indication

Oncology		Neuroscience

Code	Name	M echanism	Indication(s)
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
INC424	Jakavi®	JAK1/2 Inhibitor	Myelofibrosis (combination)
LAG525	LAG525	LAG3 Inhibitor	Solid Tumors
MBG453	MBG453	TIM3 Antagonist	HR-MDS	Unfit AML
NIR178	NIR178, spartalizumab	Ad2AR Inhibitor, PD1 Inhibitor	Cancers
PDR001	spartalizumab	PD1 Inhibitor	Solid tumors (combo)	Metastatic melanoma (combo)
SEG101	crizanlizumab	P-selectin Inhibitor	Ped sickle cell anaemia with
			crisis

Immunology, Hepatology, Dermatology

Code	Name	M echanism	Indication(s)
AIN457	Cosentyx®	IL17A Inhibitor	GCA	Lichen Planus
CFZ533	iscalimab	CD40 Inhibitor	Renal/Liver Tx	SjS	HS
LJC242	tropifexor&cenicriviroc	CCR2 Inhibitor, FXR agonist	NASH (combos)
LJN452	tropifexor	FXR agonist	NASH	NASH (combos)
LNA043	LNA043	ANGPTL3 Agonist	Osteoarthritis
LOU064	LOU064	BTK Inhibitor	CSU	SjS
LYS006	LYS006	Anti-inflammatory	Acne	Colitis ulcerative
VAY736	ianalumab	BAFF-R Inhibitor	pSjS	AIH	SLE
ZPL389	adriforant	HRH4 Antagonist	AD

Code	Name	Mechanism	Indication(s)
BAF312	Mayzent®	S1P1 Modulator	Stroke
BLZ945	BLZ945	CSF-1 Inhibitor	ALS
LMI070	branaplam	Survival motor neuron protein	SMA
MIJ821	MIJ821	NR2B Inhibitor	Depression

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	T1DM
LCZ696	Entresto®	AT-II / NEP,NEP,AGTR1,AGTR2	nHCM
		Inhibitor
LMB763	nidufexor	FXR Agonist	Diabetic Nephropathy
LNP023	LNP023	CFB Inhibitor	PNH	IgAN	C3G	iMN	aHUS
LTW980	LTW980	-	Hypertriglyceridemia

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

Global Health

Code	Name	Mechanism	Indication(s)
AFQ056	AFQ056	mGluR5 Antagonist	Addiction
KAE609	cipargamin	PfATP4 inhibitor	Malaria Severe	Malaria uncomplicated
KAF156	ganaplacide	-	Malaria uncomplicated
LXE408	LXE408	Protozoan Inhibitor	Visceral leishmaniasis

49 Novartis Q1 Results | April 28, 2020 | Novartis Investor Presentation

Novartis pipeline in Phase 1 (1 of 2)

37 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	r/r DLBCL (+ pembro)
DKY709	DKY709 + spartalizumab	-	Cancers
EGF816	nazartinib + LXH254, ribociclib, capmatinib, Opdivo, Mekinist	EGFR Inhibitor	NSCLC
HDM201	HDM201 + MBG453, venetoclax	MDM2 Inhibitor	Haematological malignancy
INC424	Jakavi	JAK1/2 Inhibitor	Myelofibrosis (combination)
JEZ567	JEZ567	CD123 CART	AML
JJO686	JJO686	CD22 CART	ALL
KAZ954	KAZ954	-	Solid tumors
LHC165	LHC165 + spartalizumab	TLR7 Agonist	Solid tumors
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 (combo)	Haematological malignancy
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 (combos)	PD1 Inhibitor, TIM3 Antagonist	AML	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

50 Novartis Q1 Results | April 28, 2020 | Novartis Investor Presentation

Novartis pipeline in Phase 1 (2 of 2)

Lead indication

37 lead indications

Immunology, Hepatology, Dermatology

Global Health

Code

Name

Mechanism

Indication(s)

Code

Name

Mechanism

Indication(s)

DFV890

Multiple Indications

KAF156

ganaplacide

Malaria prophylaxis

CEE321

Pan JAK Inhibitor

LRX712

Osteoarthritis

MHS552

Autoimmune Indications

MHV370

SLE

Neuroscience

Code

Name

Mechanism

Indication(s)

OAV201

AVXS-201

MECP2 gene therapy

Rett syndrome

Respiratory Disease

Code

Name

Mechanism

Indication(s)

CMK389

IL-18 Inhibitor

Sarcoidosis

LTP001

Respiratory Diseases

Cardiovascular, Renal, Metabolism

Code

Name

Mechanism

Indication(s)

HSY244

Atrial fibrillation

MBL949

Diabetes

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

51 Novartis Q1 Results | April 28, 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

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

54Novartis Q1 Results | April 28, 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

55Novartis Q1 Results | April 28, 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	2,572

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	2019(actual)	2019(actual)

	• Sep-2019: Primary manuscript (ARNI in HFpEF.
		Solomon S et al; NEJM. DOI: 10.1056/NEJMoa1908655)	•	Q2-2020 Study design publication (manuscript is
	• Sep-2019: ESC: Late breaker presentation of primary		accepted in ESC Heart Failure)
Publication		results	•	Q3-2020 Baseline data publication
	• Mar-2020: Effects across full range of EF, effects on	• Q3-2020 Primary data presentation at ESC congress
		NTproBNP in HFpEF, SBP in HFpEF, Subgroups (mode	•	Q3/Q4-2020 Primary data publication
		of death, MRA, age, gender).

56Novartis Q1 Results | April 28, 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	52	5,670

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

57Novartis Q1 Results | April 28, 2020 | Novartis Investor Presentation

KJX839 - small interfering RNA (siRNA) inhibitor of proprotein convertase subtilisin/kexin type 9 (PCSK9)

Study	NCT03060577 ORION-3 (CKJX839A12201E1)	NCT03814187 ORION-4 (CKJX839A1KJX839B12301)

	Hypercholesterolemia inc. Atherosclerotic Cardiovascular	Hypercholesterolemia inc. Heterozygous Familial
Indication	Disease (ASCVD) and ASCVD risk equivalents
Hypercholesterolaemia (HeFH)
	Heterozygous Familial Hypercholesterolaemia (HeFH)


Phase	Phase 2	Phase 3

Patients	~374: 284 in Group 1 and 90 in Group 2	~15,000

		A composite of major adverse cardiovascular events,
	LDL-C reduction at Day 210 for Group 1 subjects	defined as:
Primary Outcome	Changes in other lipids and lipoproteins and reduction of	•	Coronary heart disease (CHD) death;
Measures	LDL-C of more than 50% for patients that are above LDL-C	•	Myocardial infarction;
	goal ; longer term exposure and safety.	•	Fatal or non-fatal ischaemic stroke; or
		• Urgent coronary revascularization procedure

Arms/Intervention

Target Patients

Expected Completion

Publication

•	Group 1 - inclisiran 300mg sc every 6 months until day	Arm 1: every 6 month treatment KJX839 300mg (given by
720 and then on Day 810, followed by every 6 months for a	subcutaneous injection on the day of randomization, at 3
planned duration of 4 years	months and then every 6-months) for a planned median
•	Group 2- Evolocumab 140mg s.c. injection every 2	duration of about 5 years
weeks for 360 days, followed by inclisiran 300mg on Day	Arm 2: matching placebo (given bysubcutaneous injection
360, Day 450 and then every 6 months for a planned	on the day of randomization, at 3 months and then every 6-
duration of 4 years.	months) for a planned median duration of about 5 years.
Patients with HeFH or pre-existing atherosclerotic	Patient population with mean baseline LDL-C ≥ 100mg/dL;
cardiovascular disease (ASCVD) on background statin +/-	long- 5 year- follow-up time is designed to show best in-
ezetimibe therapy	class CV outcomes (25% benefit).

Primary endpoint: 2022	Primary endpoint: 2024
TBD	TBD

58Novartis Q1 Results | April 28, 2020 | Novartis Investor Presentation

KJX839 - small interfering RNA (siRNA) inhibitor of proprotein convertase subtilisin/kexin type 9 (PCSK9)

Study	NCT03851705 ORION-5 (CKJX839A12304)	NCT03399370 ORION-8 (CKJX839A12305B)

	Hypercholesterolemia inc. Homozygous Familial	Hypercholesterolemia inc. Heterozygous Familial
Indication	Hypercholesterolaemia (HeFH) and Homozygous Familial
Hypercholesterolemia (HoFH)
	Hypercholesterolemia (HoFH)


Phase	Phase 3	Phase 3

Patients	56 randomized 2:1 inclisiran: placebo	2967 entered the study

Primary Outcome Measures

Arms/Intervention

Target Patients

LDL-C reduction at Day 150	The effect of inclisiran treatment on the proportion of
subjects achieving prespecified low density lipoprotein
Changes in PCSK9, other lipids and lipoproteins and
cholesterol(LDL-C)targets at end of study. The safety and
reduction of LDL-C of more than 20%
tolerability profile of long term use of inclisiran

Part 1: inclisiran 300mg on Day 1 and Day 90 or placebo

on Day 1 and Day 90	Inclisiran 300mg on day 1 (placebo patients in feeder study)
• Part 2: placebo on Day 180, inclisiran on Day 270 and
or placebo on Day 1 (inclisiran patients in feeder study )
then every 6 months for a planned duration of 2 years or
then inclisiran 300mg on Day 90 and every 6 months for a
for placebo patients in part 1 inclisiran on Day 180, Day
planned duation of 3 years
270 and then every 6 months for a planned duration of 2

years

	Patients with HeFH or pre-existing atherosclerotic
Patients with HoFH	cardiovascular disease (ASCVD) on background statin +/-
ezetimibe therapy and risk equivalents. Patients from

	ORION 9, 10 & 11 studies

Expected Completion	Primary endpoint: 2021	Primary endpoint: 2023

Publication	TBD	TBD

59Novartis Q1 Results | April 28, 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

H2-2021		2022

TBD		TBD

60Novartis Q1 Results | April 28, 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

H1-2021	2025

TBD	TBD

61Novartis Q1 Results | April 28, 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 Measures

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
Week 13
below upper limit of normal up to 12 weeks of treatment.


• Cohort 1: 10 patients receiving LNP023 200mg bid, in

Arms/Intervention

addition to SoC, for 13 weeks with 3yr treatment extension period

Cohort 2: 5 patients receiving LNP023 50mg bid, in addition to SoC, for minimum 2 weeks with 3yr treatment 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.

Arm 1: 4wks treatment LNP023 25mg bid followed by 8wk treatment LNP023 100mg bid and 2yr extension LNP023 100mg bid
Arm 2: 4wks treatment LNP023 50mg bid followed by 8wk treatment LNP023 200mg bid and 2yr extension LNP023 200mg bid

Target Patients

Expected Completion

Publication

Patients with PNH, showing signs of active hemolysis	Patients with PNH, showing signs of active hemolysis, not
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

Primary endpoint: Q4-2020	Primary endpoint: Q4-2020
Extension period: 2023	Extension period: 2022

In preparation	TBD

62Novartis Q1 Results | April 28, 2020 | Novartis Investor Presentation

TQJ230 - Antisense oligonucleotide targeting apolipoprotein(a) mRNA

Study	NCT04023552 Lp(a)HORIZON (CTQJ230A12301)

Indication	Cardiovascular risk reduction

Phase	Phase 3

Patients	7,680

Primary Outcome	Time to the first occurrence of MACE (cardiovascular death,
non-fatal MI, non-fatal stroke and urgent coronary re-
Measures
vascularization)


Arms/Intervention	TQJ230 80 mg injected monthly subcutaneously or
matched placebo


Target Patients	Patients with a history of Myocardial infarction or Ischemic
Stroke, or a clinically significant symptomatic Peripheral

	Artery Disease, and Lp(a) ≥ 70 mg/dL

Expected Completion	2024

Publication	TBD

63Novartis Q1 Results | April 28, 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 published in The Lancet-
Rheumatology January 23, 2020

65Novartis Q1 Results | April 28, 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

2023

TBD

66Novartis Q1 Results | April 28, 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

67Novartis Q1 Results | April 28, 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

68Novartis Q1 Results | April 28, 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
Manuscript Publication under assessment

69Novartis Q1 Results | April 28, 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

70Novartis Q1 Results | April 28, 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	2018(actual)	2018(actual)
	• 3 year results: ACR 2016; Mease PJ et al. Arthritis	• Primary 52 week results: Baeten D & Sieper J, et al. N
		Rheumatol. 2016; 68 (suppl 10)
			Engl J Med 2015;373:2534-48
	•	3 years results: Manuscript published in September
	•	2 year results: Marzo-Ortega, et al. Arthritis Care Res
		2018 (Mease PJ, et al. RMD Open 2018;4:e000723.
			2017 Feb 24. doi: - 10.1002/acr.23233
		doi:10.1136/rmdopen-2018-000723)
		•	3 year results: Marzo-Ortega, et al. RMD 2017
Publication	•	5 year results: Philip J. Mease, Arthur Kavanaugh,
•	5 year results: EULAR 2019; Marzo-Ortega H, et al.
		Andreas Reimold, et al. "Secukinumab Provides
			FRI0379. Annals of the Rheumatic Diseases
		Sustained Improvements in the Signs and Symptoms of
			2019;78:873.
		Psoriatic Arthritis: Final 5‐year Results from the Phase 3
		•	5 year results; manuscript accepted and to be published
		FUTURE 1 Study." ACR Open Rheumatology. November
			in Q2-2020
		14, 2019. https://doi.org/10.1002/acr2.11097

71Novartis Q1 Results | April 28, 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	2019(actual)	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 &
		Therapy 2017
		2015;386:1137-46
		•	2 year results: Presented at ACR in Nov-2017
Publication	•	2 years results: McInnes et al, Rheumatology
•	3 year (EOS) results: To be presented (ORAL) at
		2017;56:1993-2003
			PANLAR April 2019
	•	5 years: published Lancet Rheumatology in March 2020
	• 3 year (EOS) manuscript published in ACR Open

				Rheumatology in January 2020

72Novartis Q1 Results | April 28, 2020 | Novartis Investor Presentation

Cosentyx®- Anti IL-17

Study	NCT02745080 EXCEED (CAIN457F2366)

Indication	Psoriatic arthritis

Phase	Phase 3

Patients	850
Primary Outcome
American College of Rheumatology 20 (ACR20) response
Measures


Arms/Intervention	•	Secukinumab 300 mg s.c.
• Adalimumab 40 mg s.c.


Target Patients	Patients with active psoriatic arthritis

Expected Completion	Q1-2020

Publication	•	Manuscript will be published in Apr-2020

73Novartis Q1 Results | April 28, 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 submitted in Mar-2020

74Novartis Q1 Results | April 28, 2020 | Novartis Investor Presentation

Cosentyx®- Anti IL-17

Study	NCT03713619 SUNSHINE (CAIN457M2301)	NCT04179175 (CAIN457M2301E1)

Indication	Hidradenitis Suppurativa (HS)	Hidradenitis Suppurativa (HS)

Phase	Phase 3	Phase 3

Patients	471	745

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
Arms/Intervention	•	Secukinumab 300 mg every 4 weeks	•	Secukinumab 300 mg every 2 weeks
•	Placebo (every 2 weeks)	•	Secukinumab 300 mg every 4 weeks

	•	Placebo (every 4 weeks)
			Patients with moderate to severe hidradenitis suppurativa
Target Patients	Patients with moderate to severe Hidradenitis Suppurativa	completing either of the core trials AIN457M2301 (NCT
			0313632) or AIN567M2302 (NCT03713619)
Expected Completion	H2-2021	2025

Publication	Preliminary results in EADV (most likely) in 2021	TBD

75Novartis Q1 Results | April 28, 2020 | Novartis Investor Presentation

Cosentyx®- Anti IL-17

Study	NCT03713632 SUNRISE (CAIN457M2302)

Indication	Hidradenitis Suppurativa (HS)

Phase	Phase 3

Patients	471

Primary Outcome	Proportion of patients with Hidradenitis Suppurativa Clinical
Measures	Response (HiSCR)

	• Secukinumab 300 mg every 2 weeks
Arms/Intervention	•	Secukinumab 300 mg every 4 weeks
•	Placebo (every 2 weeks)

	•	Placebo (every 4 weeks)

Target Patients

Expected Completion

Subjects with moderate to severe Hidradenitis Suppurativa

H2-2021

Publication

Preliminary results in EADV (most likely) in 2021

76Novartis Q1 Results | April 28, 2020 | Novartis Investor Presentation

Cosentyx®- Anti IL-17

Study	NCT04156620 INVIGORATE-1 (CAIN457P12301)	NCT04209205 INVIGORATE-2 (CAIN457P12302)

Indication	Axial spondyloarthritis	Axial spondyloarthritis

Phase	Phase 3	Phase 3

Patients	500	380

Primary Outcome	The proportion of subjects achieving an ASAS40	The proportion of subjects achieving American College of
(Assessment of SpondyloArthritis International Society
Measures	Rheumatology 50 (ACR50) response criteria
criteria) response


Arms/Intervention	•	Secukinumab intravenous (i.v.) regimen	•	Secukinumab intravenous (i.v.) regimen
•	Placebo intravenous (i.v.) regimen	•	Placebo intravenous (i.v.) regimen


Target Patients	Patients with active axial spondyloarthritis	Patients with active psoriatic arthritis (PsA) despite current
or previous NSAID, DMARD and/or anti-TNF therapy

Expected Completion	2022	2022

Publication	TBD	TBD

77Novartis Q1 Results | April 28, 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 submitted in Q4-2019

78Novartis Q1 Results | April 28, 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	Planned in H1-2023
	•	Manuscript to be submitted in Q4-2020

79Novartis Q1 Results | April 28, 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

80Novartis Q1 Results | April 28, 2020 | Novartis Investor Presentation

LJC242 - FXR agonist + CCR2/CCR5 inhibitor

Study	NCT03517540 TANDEM (CLJC242A2201J)

Indication	Non-alcoholic steatohepatitis

Phase	Phase 2

Patients	193

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

81Novartis Q1 Results | April 28, 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 respect	Long-term safety; number of participants with treatment-
Measures	to achievement of complete hives response at week 12	emergent adverse events

	•	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 inadequately	Adult patients with chronic spontaneous urticaria inadequately
	controlled with H1-antihistamines at approved or increased
Target Patients	controlled with H1-antihistamines at approved or increased
doses, alone or in combination with H2-antihistamines or
	doses, alone or in combination with H2-antihistamines or
	leukotriene receptor antagonists.
	leukotriene receptor antagonists.

Expected Completion	2017(actual)	2019(actual)

	• Primary results: Presented at EAACI 2018, EADV 2018,	• Primary results: AAD 2019;
	• Secondary results presented in 2019 at: AAD, EAACI,
		and GUF 2018; NEJM publication (3 Oct 2019);
		WCD, EADV, PAAM, ACAAI, UCARE
	• Secondary results presented in 2019 at: AAD, EAACI,
Publication	• Exploratory results presented/ planned in 2020: AAAAI,
	WCD, EADV, PAAM, ACAAI, UCARE.
		EAACI, EADV, ACAAI; Encoring all at GUF
	•	Exploratory results presented/ planned in 2020: AAAAI,
	• 5 Manuscripts 2020: core results extension; time to loss
		EAACI, EADV, ACAAI; Encoring all at GUF
		response, fast response; angioedema; data visualization

82Novartis Q1 Results | April 28, 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

83Novartis Q1 Results | April 28, 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	• 2020: C2302E1 extension study (NCT04210843) design EAACI
• Primary results to be presented in 2022 (e.g. EAACI, PAAM, EADV)

	•	Manuscript to be submitted in 2022

84Novartis Q1 Results | April 28, 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

Publication	•	Manuscript to be submitted in 2020	TBD

85Novartis Q1 Results | April 28, 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

86Novartis Q1 Results | April 28, 2020 | Novartis Investor Presentation

Neuroscience

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

88Novartis Q1 Results | April 28, 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)

89Novartis Q1 Results | April 28, 2020 | Novartis Investor Presentation

Gilenya®- S1P-R modulator

Study	NCT01633112 ASSESS (CFTY720D2312)

Indication	Relapsing remitting multiple sclerosis (RRMS)

Phase	Phase 3B

Patients	1,064

Primary Outcome	Comparison of 2 doses (0.25 mg and 0.5 mg) of fingolimod
to glatiramer acetate (20 mg) in reducing the annualized
Measures
relapse rate up to 12 months


	• Fingolimod 0.5 mg orally
Arms/Intervention	•	Fingolimod 0.25mg orally
	• Copaxone®20 mg s.c.
Target Patients	Patients with relapsing-remitting multiple sclerosis

Expected Completion	2018(actual)

	• Primary data presentation at AAN in 2019
Publication	•	Primary manuscript submitted in February 2020,
		publication expected by Jun-2020

90Novartis Q1 Results | April 28, 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 (Part 2)

Publication	TBD

91Novartis Q1 Results | April 28, 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

92Novartis Q1 Results | April 28, 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

93Novartis Q1 Results | April 28, 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(actual)	2025

Publication	TBD	TBD

94Novartis Q1 Results | April 28, 2020 | Novartis Investor Presentation

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	33	22

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	ICNMD 2020, Manuscript planned H1-2021	MDA 2020, Manuscript planned H2-2020

95Novartis Q1 Results | April 28, 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	30	6

	• [2 copies of SMN2] Percentage of participants achieving
	functional independent sitting for at least 30 seconds at
Primary Outcome	any visit	Proportion of participants sitting without support
Measures	• [3 copies of SMN2] 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	MDA 2020 (interim)	TBD

96Novartis Q1 Results | April 28, 2020 | Novartis Investor Presentation

Zolgensma®- SMN1 gene replacement therapy

Study	NCT03381729 STRONG (CL-102)

Indication	Type 2 spinal muscular atrophy

Phase	Phase 1

Patients	51

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] (actual)

Publication	MDA 2020, Manuscript planned for 2H 2020

97Novartis Q1 Results | April 28, 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

Q3-2020

Manuscript submissionQ4-2020
Abstract submission to congressQ3-2020

99Novartis Q1 Results | April 28, 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

		• 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

Expected Completion	Interim Analysis: 2022; Final: 2023	Interim Analysis: Q4-2020 (PFS); Final: 2022 (OS)

		Johnson B et al. Presented at AACR-NCI-EORTC 2019
Publication	TBD	(safety run-in)
Manuscript submission Q4-2020 (safety run-in)

Abstract submission to congress H1-2021

100Novartis Q1 Results | April 28, 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

Abstract submission to congress H1-2021

101Novartis Q1 Results | April 28, 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	2018(actual)

	• Andre F, et al. Presentation at ESMO 2018
Publication	• Andre et al. Manuscript N Engl J Med 2019;380:1929-
	1940.

102Novartis Q1 Results | April 28, 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	2018(actual)

	• Angelucci E, et al. Presentation at ASH 2018
Publication	•	Angelucci E, et al. Manuscript Ann Intern Med 2020 Mar
		24 [Online ahead of print]

103Novartis Q1 Results | April 28, 2020 | Novartis Investor Presentation

INC280 - MET Inhibitor

Study	NCT02414139 (CINC280A2201)
Indication	EGFR Wild-type, ALK negative advanced Non-small Cell
Lung Cancer (NSCLC)


Phase	Phase 2

Patients	364
Primary Outcome
Overall Response Rate (ORR)
Measures


	•	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
Arms/Intervention	•	Treatment-naïve pts. with MET dysregulation
	•	Pre-treated pts with MET dysregulation - second line

• Treatment-naïve pts with cMET mutations regardless of cMET GCN

Target Patients

Expected Completion

Publication

Adult patients with EGFR wild-type (wt), ALK-negative advanced/ metastatic NSCLC with either MET amplification or MET mutations

2019(actual)

Wolf J, et al. Presented at ASCO 2019
Wolf J, et al. Manuscript submittedQ1-2020

104Novartis Q1 Results | April 28, 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	310	330

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	2019(actual)	Interim Analysis: 2019(actual); Final: Q3-2020

	• Zeiser R, et al. Manuscript N Engl J Med (accepted Q1-
Publication		2020, not yet published)	•	Manuscript submission in H2-2020
• Zeiser R, et al. Abstract accepted for presentation at	•	Abstract submission to congress in H2-2020

		EBMT Q3-2020

105Novartis Q1 Results | April 28, 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

106Novartis Q1 Results | April 28, 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: H2-2022

Publication	TBD

107Novartis Q1 Results | April 28, 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.	Abstract submission to congress in H2-2020
Presentation at ICML 2019

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

108Novartis Q1 Results | April 28, 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

109Novartis Q1 Results | April 28, 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

110Novartis Q1 Results | April 28, 2020 | Novartis Investor Presentation

PDR001 - PD-1 checkpoint inhibitor

Study	NCT02967692 COMBI-i (CPDR001F2301)

Indication

Phase

Patients

Primary Outcome Measures

Arms/Intervention

Target Patients

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

Expected Completion	Q3-2020

Publication	•	Abstract submission to congress in Q3-2020
•	Manuscript submission Q3-2020

111Novartis Q1 Results | April 28, 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

112Novartis Q1 Results | April 28, 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

113Novartis Q1 Results | April 28, 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)

Q2-2020	H2-2022

Publication	Abstract submission to congress in Q4-2020	TBD

114Novartis Q1 Results | April 28, 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	2018(actual)	H2-2021 (pediatric patients ≥6 year old)
2022 (pediatric patients 6 months - 6 year old)

Publication	Abstract submission to congress in Q3-2020 (7.5 mg group)	Abstract submission to congress in Q3-2020

115Novartis Q1 Results | April 28, 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

116Novartis Q1 Results | April 28, 2020 | Novartis Investor Presentation

Tafinlar®- BRAF inhibitor

Study	NCT01677741 (CDRB436A2102)

Indication	BRAFV600 mutant cancers

Phase	Phase 1/2

Patients	86

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	H1-2021

	• Kieran MW et al. Manuscript Clin Cancer Res
	2019;25(24):7294-7302 (PK analysis)
Publication	• Hargrave DR et al. Manuscript Clin Cancer Res
	2019;25(24):7303-7311 (safety/efficacy in low-grade
	gliomas)

117Novartis Q1 Results | April 28, 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)

Target Patients	Children and adolescent patients with BRAF V600 mutation
positive relapsed or refractory high grade glioma (HGG) or
	BRAF V600 mutation positive low grade glioma (LGG)
Expected Completion	2022
Publication
TBD

118Novartis Q1 Results | April 28, 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 accepted for presentation at ASCO Q2-2020

119Novartis Q1 Results | April 28, 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): Q4-2018(actual)
	Final (ORR): Q3-2020

	• 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): Abstract submission to congress Q3-2020

120Novartis Q1 Results | April 28, 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	831

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	Q4-2020

Publication	TBD

121Novartis Q1 Results | April 28, 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	2018(actual)	2023
	•	EURETINA: Sep-2018
	•	AAO: Oct-2018
	•	Primary manuscript published online by The Lancet in
		Sep-2019
Publication		(https://www.thelancet.com/pdfs/journals/lancet/PIIS0140	Submission of publication of 2 year data (Interim Analysis 2)
	-6736(19)31344-3.pdf)	in 2020

	• Submission of manuscript on Pop PK/PD analysis in
		2020
	• Submission of manuscript on time-course of clinical
		response to treatment in 2020

123Novartis Q1 Results | April 28, 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	2018(actual)	2018(actual)

	• Year 1 Manuscript: Dugel P, et al. Ophthalmology 2019 Apr 12; HAWK and HARRIER: Phase 3, Multicenter,
		Randomized, Double-Masked Trials of Brolucizumab for Neovascular Age-Related Macular Degeneration.
Publication	•	Secondary publications planned for 2020 are: Fluid resolution, PCV and CNV subtypes, CST variability, the IPDA, safety
	and VFQ outcomes submitting in Q1-Q3 of 2020

• Abstracts submissions on superior anatomic outcomes/Fluid/PostHoc results are planned for key retinal congresses (WOC; ARVO; ASRS, EURETINA AAO and APVRS) throughout 2020

124Novartis Q1 Results | April 28, 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	2018(actual)	H2-2021

Publication	Planned publication of the attributes of brolucizumab and	Week 52 safety and efficacy data to be submitted as an
durability in Q1-2020	abstract in H1-2021 (KITE and KESTREL)

125Novartis Q1 Results | April 28, 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	Chinese patients with visual impairment due to diabetic
edema (DME)	macular edema

Expected Completion	H2-2021	2022

Publication	Week 52 safety and efficacy data to be submitted as an	Publication planned for 2023
abstract H1 2021 (KITE and KESTREL)

126Novartis Q1 Results | April 28, 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	Publication submission planned for 2022	Publication submission planned for 2022

127Novartis Q1 Results | April 28, 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

128Novartis Q1 Results | April 28, 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 at month 3

Arms/Intervention	•	1.5% solution UNR844-Cl
•	Placebo


Target Patients	Patients with presbyopia

Expected Completion	Q1-2020

Publication	TBD (Original plan to publish at ASCRS in 2020, but ASCRS
got cancelled due to COVID-19)

129Novartis Q1 Results | April 28, 2020 | Novartis Investor Presentation

Respiratory

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

131Novartis Q1 Results | April 28, 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	2019(actual)

Publication	Planned in H2-2020

132Novartis Q1 Results | April 28, 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	2019(actual)	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

133Novartis Q1 Results | April 28, 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	2019(actual)	2019(actual)

	• Japanese J Allergo (B1304/1305 combined); Planned in	• Japanese J Allergo (B1304/1305 combined); Planned in
Publication	H2-2020	H2-2020
	• Abstract for ATS in Q2-2020	• Abstract for ATS in Q2-2020

134Novartis Q1 Results | April 28, 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	2019(actual)	2019(actual)

Publication	•	O. Kornmann et al. Respiratory Medicine 161 (2020)	•	Resp Med; Planned in Q2-2020
• Abstract: D'Andrea et al, presented at ERS Sep-2019	•	Abstract ATS Q2-2020

135Novartis Q1 Results | April 28, 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

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
Manuscript submitted to JACI in Practice,Q1-2020

136Novartis Q1 Results | April 28, 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	Patients with moderate to severe active rheumatoid arthritis

Expected Completion	2018(actual)

	• Wiland, P. et al., presented at EULAR 2019
Publication	•	Wiland, P. et al., BioDrugs, Q2 2020

138Novartis Q1 Results | April 28, 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

Publication

Postmenopausal women with osteoporosis

2022

Study data publications expected for 2024 and beyond. The overall study design will be published at WCO and ECTS congresses 2020.

139Novartis Q1 Results | April 28, 2020 | Novartis Investor Presentation

Global Health

KAF156 - Plasmodium Falciparum Inhibitor - PfCARL mediated

Study	NCT03167242 (CKAF156A2202)

Indication	Malaria

Phase	Phase 2

Patients	~500

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	H2-2021

Publication	TBD

141Novartis Q1 Results | April 28, 2020 | Novartis Investor Presentation

KAE609 - Plasmodium Falciparum Inhibitor - spiroindolone against PfATP4

Study	NCT03334747 (CKAE609A2202)

Indication	Malaria

Phase	Phase 2

Patients	186

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	Q1-2020(actual)

Publication	TBD

142Novartis Q1 Results | April 28, 2020 | Novartis Investor Presentation

Abbreviations

AIH	Autoimmune hepatitis	mCRPC	Metastatic castration-resistant prostate cancer
aHUS	atypical Hemolytic Uremic Syndrome	MDR	Multi-drug resistant
ALL	Acute lymphoblastic leukemia	MDS	Myelodysplastic syndrome
ALS	Amyotrophic lateral sclerosis	MS	Multiple sclerosis
AMI	Acute myocardial infarction	nAMD	Neovascular (wet) age-related macular degeneration
AML	Acute myeloid leukemia	NASH	Non-alcoholic steatohepatitis
AS H2H	Ankylosing spondylitis head-to-head study versus adalimumab	nHCM	Non-obstructive hypertrophic cardiomyopathy
BC	Breast cancer	nr-axSpA	Non-radiographic axial spondyloarthritis
C3G	C3 glomerulopathy	NSCLC	Non-small cell lung cancer
CCF	Congestive cardiac failure	PDR	Proliferative diabetic retinopathy
CLL	Chronic lymphocytic leukemia	PEF	Preserved ejection fraction
CML	Chronic myeloid leukemia	PNH	Paroxysmal nocturnal haemoglobinuria
CRC	Colorectal cancer	PsA H2H	Psoriatic arthritis head-to-head study versus adalimumab
COPD	Chronic obstructive pulmonary disease	RCC	Renal cell carcinoma
COSP	Chronic ocular surface pain	PROS	PIK3CA related overgrowth spectrum
CSU	Chronic spontaneous urticaria	RA	Rheumatoid arthritis
CVRR-Lp(a)	Secondary prevention of cardiovascular events in patients with elevated levels of lipoprotein (a)	rMS	Relapsing multiple sclerosis
CVRR-LDLC	Secondary prevention of cardiovascular events in patients with elevated levels of LDLC	ROP	Retinopathy of prematurity
DME	Diabetic macular edema	RP	Retinitis pigmentosa
DLBCL	Diffuse large B-cell lymphoma refractory	RVO	Retinal vein occlusion
GCA	Giant cell arteritis	SAA	Severe aplastic anemia
GVHD	Graft-versus-host disease	SjS	Sjögren's syndrome
HCC	Hepatocellular carcinoma	SLE	Systemic lupus erythematosus
HFpEF	Chronic heart failure with preserved ejection fraction	SMA Type 1	Spinal muscular atrophy type 1
HF-rEF	Chronic heart failure with reduced ejection fraction	SMA Type 2/3	Spinal muscular atrophy type 2/3
HNSCC	Head and neck squamous cell carcinoma	SpA	Spondyloarthritis
HS	Hidradenitis suppurativa	SPMS	Secondary progressive multiple sclerosis
IgAN	IgA nephropathy	TNBC	Triple negative breast cancer
iMN	Membranous nephropathy	T1DM	Type 1 Diabetes melitus
IPF	Idiopathic pulmonary fibrosis

143 Novartis Q1 Results | April 28, 2020 | Novartis Investor Presentation

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