Novartis : Q3 2020 Results Investor presentation

Novartis Investor Relations

Q3 2020 Results

Investor presentation October 27, 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 future revenues from any such products; or regarding the impact of the COVID-19 pandemic on certain therapeutic areas including dermatology, ophthalmology and the Sandoz retail business, and on drug development operations; or regarding potential future, pending or announced transactions; regarding potential future sales or earnings of the Group or any of its divisions; 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 our collaboration with the African Union to supply medicines for treatment of COVID-19. 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: liquidity or cash flow disruptions affecting our ability to meet our ongoing financial obligations and to support our ongoing business activities; 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; the impact of a partial or complete failure of the return to normal global healthcare systems including prescription dynamics, particularly in ophthalmology, in the fourth quarter of 2020; 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 transactions described, may not be realized or may be more difficult or take longer to realize than expected; 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; 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, investigations or 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.

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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	Chief Legal Officer
Susanne Schaffert
President, Novartis Oncology

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Vas Narasimhan

Chief Executive Officer

Company overview

4 Novartis Q3 Results | October 27, 2020 | Novartis Investor Presentation

Novartis delivered a solid quarter with double digit core operating income growth and strong innovation milestones

Solid operational performance	Delivering on innovation
Continuing operations1, % cc vs. PY
		Approvals	Positive readouts	Positive CHMP opinion
Net sales	Core OpInc
	16%	US for RMS		DME2	Leqvio®
				Hyperlipidemia
					(inclisiran)
		EU for PedPsO		Chronic GvHD
	11%	JP for nr-axSpA
		ABL001	CML
		EU for		Sickle cell
					disease
		HR+/HER2- aBC		FL (IA)
4%
		EU for CRSwNP	LNP023	PNH, C3G (IA)
0%
Q3 2020	YTD 2020	LNP023: PRIME (C3G), EU orphan drug designation (PNH, C3G, IgAN), US orphan drug designation
		(PNH, C3G); LMI070: US orphan drug designation (Huntington's disease)

All abbreviations on slide 143 CRSwNP - Severe chronic rhinosinusitis with nasal polyps 1. Refers to continuing operations as defined on page 42 of the Condensed Interim Financial Report, excludes Alcon, includes the businesses of Innovative Medicines and Sandoz, as well as the continuing corporate functions 2. Ph3 KITE study

5 Novartis Q3 Results | October 27, 2020 | Novartis Investor Presentation

Strong performance from key growth drivers for Q3

Key growth driver sales Q3 2020

Sales		Growth vs. PY	Growth vs. PY
USD Million		USD Million	cc
632		202	45%
291		131	79%
1,012		75	7%
442	62		16%
183	60		50%
335	56		18%
397	52		14%
51	51		nm
49	45		nm
220	43		25%
122	43		51%
83	40		95%

nm - not meaningful

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

50%				Adakveo®
											Mayzent®
41%						Beovu®
					Piqray®
											Xiidra®
33%								Lutathera®
											Kymriah®
27%										Kisqali®
									Ilaris®
											Zolgensma®
											Jakavi®
											Tafinlar+Mekinist®
											Promacta®
											Entresto®
											Cosentyx®
Q3 2017 Q3 2018 Q3 2019 Q3 2020			Other1

1. Includes Tasigna®, Xolair®, Aimovig®, Tabrecta ®, Luxturna® , Kesimpta®, Enerzair ® and Atectura®

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Zolgensma®: Cumulative sales of >USD 1bn since launch solidifies success of gene therapy as new class

Sales evolution

9M sales USD 666m

USD million				291
	186		205	169
	170
160	41
	44	100
150	145	126		122
15		105

Q3 highlights

• US sales in Q3 showed signs of rebounding from pandemic disruption across all patient segments
• 74% of newborns are now being screened for SMA
• Shifted to incident: 83% Q3 2020 vs. 34% Q3 2019
• Access pathways in 9 EU countries established: 25% of population
• Germany: 50% of ex-US sales in Q3

1. Majority of early patients coming from prior treatment with nusinersen o Over 30% of patients >2 years old

12

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

ex-US US

Geographic expansion

• Japan: rapid uptake with immediate full reimbursement
• Latest approval in Brazil (August)
• Pending approvals expected H2 2020/ H1 2021: Switzerland (Fast Track), Canada (Priority Review), Australia, Argentina, South Korea, Taiwan

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Sandoz YTD sales in line with PY, core OpInc continues to grow

Biopharmaceuticals key growth driver offset by US oral solids and COVID-19 impact

Sandoz Q3 and YTD performance overview	Biopharmaceuticals growing double digit
Net sales	Core OpInc	Q3 sales	9M sales
Vs. PY, in cc
19%		20%
	13%
	8%
0%

-3%					-6%	-4%
		Q3 2020		9M 2020			Retail		Biopharmaceuticals

Performance key drivers

• Biopharmaceuticals1 strong growth driven by Europe and RoW; Europe, Sandoz biosimilars market leader, growing share to ~27% (~23% in Jul 20192)
• Retail decline impacted by oral solids decline in US and COVID-19 impact on patient traffic
• Gross margin increase from product mix and productivity; reduced SG&A spending from lower activities due to COVID-19

1. Biopharmaceuticals include biosimilars, biopharmaceutical contract manufacturing and Glatopa® 2. Source: IQVIA PADDS July 2020 vs. July 2019

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

• 15 ongoing / upcoming expected major launches
• 80+ submissions planned to 20221
• 50+ late stage programs2

Major launches

In-market growth drivers

Novel assets

Pharmaceuticals	Oncology
Leqvio® (inclisiran)	Sabatolimab
Ligelizumab	Asciminib
Iptacopan	Canakinumab
Iscalimab	177Lu-PSMA-617
Tropifexor	TNO155
UNR844	LXH254
CEE321	Spartalizumab
Remibrutinib
Ianalumab
Branaplam
LNA043
Pelacarsen
CSJ117
QBW251

New indications

Pharmaceuticals	Oncology
				Alpelisib
				PROS
HS
GCA
LP		TNBC
JIA
LN		HER2+ aBC
				Ovarian cancer
				HNSCC
post-AMI
HFpEF		HR+/HER2- BC
				(adj)
DME
RVO		r/r
DR
FL
				r/r DLBCL
AVXS-101		1st relapse
IT
				cGVHD
Food allergy		aGVHD

SELECT EXAMPLES

1. US, Europe, China, Japan 2. Pivotal projects / registration

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Advancing our late stage pipeline

Selected assets

	Asset	Indication		Current status	Next milestone (expected)
	Leqvio® (inclisiran)	Hyperlipidemia	Registration	US / EU submissions complete; positive CHMP opinion	FDA action date December 2020
				ORION-4 ongoing
Pharmaceuticals	Ofatumumab (OMB157)	RMS	Registration	US approval received August 2020, JP submission July 2020	EU approval H1 2021
(Kesimpta® in US)			FDA recommended pivotal confirmatory study to supplement existing	following further discussions with health
	Entresto®	HFpEF,	Registration	HFpEF filed	FDA action expected by H1 2021
		post-AMI	Ph3	PARADISE-MI enrollment complete	PARADISE-MI study results H1 2021
	AVXS-101 IT	SMA IT	Ph3	Partial clinical hold	Update on pivotal study to be provided
				STRONG data	authorities
	Ligelizumab (QGE031)	CSU	Ph3	PEARL 1 and 2, superiority studies vs. Xolair® ongoing	Enrollment complete with results
					expected in H2 2021, submission 20221
	Alpelisib (BYL719)	PROS	Ph2	Real World Evidence (RWE) ongoing	US submission H1 20211
	Asciminib (ABL001)	CML	Ph3	ASCEMBL Ph3 study met its primary endpoint, received FDA fast track	First submission H1 2021
				designation; evaluating earlier lines of therapy
Oncology	Canakinumab (ACZ885)	NSCLC	Ph3	CANOPY-1 enrollment complete, DMC recommend continuation after IA	Final CANOPY-1 readout H2 2021; final
			CANOPY-2 enrollment complete	CANOPY-2 readout H1 2021
				CANOPY-A enrollment continues
	177Lu-PSMA-617	mCRPC 3/4L	Ph3	VISION trial enrollment completed and waiting for completion of primary	VISION trial readout H1 2021
				endpoint (rPFS or OS) events
				Planning Ph3 studies in pre-taxane mCRPC and mHSPC
	Kisqali®	Adjuvant BC	Ph3	NATALEE study with protocol amendment implemented to increase	MONALEESA-2 OS readout H2 2021
				sample size (additional 1,000 patients)	NATALEE Ph3 aBC final readout 2022

1. COVID-19 related delay

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Emerging pipeline assets continue to progress

Selected assets

Pharmaceuticals

Oncology

Asset	MoA	Indication		Current status	Next milestone (expected)
Iptacopan	Factor B	PNH, IgAN,	Ph2	Positive Ph2 results in PNH and in C3G	Single PNH pivotal trial to start 2020;
(LNP023)	inhibitor	C3G,		US orphan designation received for PNH and C3G	C3 glomerulopathy, IgA nephropathy
		iMN, aHUS		EU orphan designation received for PNH, C3G, IgAN	Ph3 studies to start H1 2021
				EU PRIME designation for C3G
Remibrutinib	Bruton's tyrosine kinase	CSU, Sjögren's	Ph2	Ph2b study in CSU and adaptive Ph2 in Sjögren's ongoing	Phase 2b study in CSU to read out H2 2021
(LOU064)	inhibitor
Iscalimab	Anti-CD40 monoclonal	Kidney Tx, Liver	Ph2	Ph2 studies in kidney transplant, liver transplant and	Anticipated regulatory submission for
(CFZ533)	antibody	Tx, Sjögren's		Sjögren's ongoing	kidney transplant 2023 assuming acceptance
					of the risk prediction score
Pelacarsen	Antisense oligonucleotide	CVRR-Lp(a)	Ph3	Ph3 outcomes study (HORIZON) initiated 2020	Ph3 outcomes readout 2024
(TQJ230)	targeting Lp(a)			FDA Fast Track designation received
Branaplam	mRNA splicing modulator	SMA	Ph2	Ph1/2 ongoing	SMA Ph1/2 first readout 2021
(LMI070)		HD	Ph1	FDA granted Orphan Drug Designation for HD	HD Ph2 to start in 2021
Sabatolimab	Anti-TIM-3 monoclonal	HR-MDS	Ph3	Pivotal Ph2 and confirmatory Ph3 in MDS ongoing	Submission for MDS in 2021 in US
(MBG453)	antibody	unfit AML	Ph2	Ph2 in unfit AML (combo HMA + venetoclax) initiated
				September 2020
LXH254	B/C-RAF inhibitor	m RAS/RAF	Ph1	Clinical studies ongoing, evaluating LXH254 in combination	Ph2 metastatic melanoma trial
		NSCLC and		with LTT462 (ERKi), Mekinist®, Kisqali® and spartalizumab	to start 2020
		melanoma
TNO155	SHP2 inhibitor	Solid tumors	Ph1	Broad combination strategy with multiple Ph1 combo	Continued enrollment in all 3 trials
				studies ongoing including spartalizumab, Kisqali®,
				nazartinib, MRTX8491

1. Study sponsored by Mirati Therapeutics

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LNP023 (iptacopan) in PNH

All patients on LNP023 monotherapy retained Hb levels without breakthrough hemolysis

Significant reduction of residual hemolysis in eculizumab-treated patients

Individual time profiles of hemoglobin (g/L)

Hemoglobin (g/L)

Represents the last dose of eculizumab

• Better hematological response (LDH, Hb) as add-on
• Effect maintained in absence of eculizumab (7/10 patients stopped)
• Favorable safety & tolerability profile

LLN - Lower limit of normal PNH - Paroxysmal nocturnal hemoglobinuria

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LNP023 (iptacopan) in C3G

Positive Ph2 interim analysis supports rapid Ph3 initiation

Significant reduction in proteinuria1

Adjusted geometric mean (80% CI) of ratio to baseline for UPCR (24h urine collection) over time

0.51 ( 0.42, 0.63)	✓ Significant reduction in proteinuria
p = 0.0005 (one sided)
	✓ Stabilization of renal function (eGFR)
	✓ Favorable safety & tolerability profile

Dose escalation to 200mg bid (w4) native kidney, n=12

eGFR - Estimated glomerular filtration rate UPCR - Urine protein to creatinine ratio C3G - C3 glomerulopathy 1. Presented at American Society at Nephrology Kidney Week 2020

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Integrating ESG across all our operations as we strive for more sustained impact on our journey to become an ESG leader

Adopted ambitious targets:	Healthcare industry's 1st	Received upgrades to ESG scores
	Access to innovation1	sustainability-linked bond (SLB),	from third party ratings agencies
	Global health programs2	1st SLB incorporating social targets	including MSCI (see slide 37)

• Full carbon neutrality by 20303

ESG - strengthen, measure, increase transparency

Strengthening areas where we have the largest impact, reinforcing our commitment to measure what matters, communicate in a transparent way

1. 200% increase in patients reached in Low income / lower middle-income countries (LMICs) with Strategic Innovative Therapies (2025 targets vs. 2019 baseline). 2. Global health programs 50% increase in patients reached in LMICs (2025 targets vs. 2019 baseline) 3. Scope 1,2,3 (2030 targets vs. 2016 baseline)

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Harry Kirsch

Chief Financial Officer

Financial review and 2020 guidance

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Strong 9M performance on top and bottom line

1	Q3	Change vs. PY
Continuing operations
USD million	2020	% USD	% cc	2
Net Sales	12,259	1	0
Core Operating income 2	4,069	9	11
Operating income	2,412	2	9
Net Income	1,932	-5	0
Core EPS (USD)2	1.52	8	9
EPS (USD)	0.85	-6	0
Free Cash Flow 2	2,697	-32

9M	Change vs. PY
2020	% USD	% cc	2
35,889	2	4
11,915	12	16
7,508	3	11
5,972	-1	6
4.44	12	16
2.62	0	7

8,349 -12

1. Refers to continuing operations as defined on page 42 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 54 of the Condensed Interim Financial Report

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Core margin expansion of +3.6% points YTD

Innovative Medicines core margin at 36.3%

Continuing operations1,2

Q3 2020		9M 2020

	Core operating
Net sales	income		Core margin
change vs. PY	change vs. PY	Core margin	change vs. PY
(in % cc)	(in % cc)	(%)	(%pts cc)

	Core operating
Net sales	income		Core margin
change vs. PY	change vs. PY	Core margin	change vs. PY
(in % cc)	(in % cc)	(%)	(%pts cc)

Innovative Medicines	1	9	35.8	2.6	5	13	36.3	2.7
Sandoz	-3	8	27.2	2.8	0	19	25.4	4.2
Continuing Operations	0	11	33.2	3.2		4	16	33.2	3.6

1. Refers to continuing operations as defined on page 42 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 54 of the Condensed Interim Financial Report

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COVID-19 continued to negatively impact demand in Q3, particularly in dermatology and ophthalmology

IM weekly sales evolution 2020

4 weeks rolling, indexed to Q4 weekly sales average

150%		COVID-19 lockdowns across US & Europe				Recent Launches1
125%							Growth drivers
						(excl. Cosentyx®)2
							Cosentyx®
100%							Beovu®, Lucentis®,
						Xiidra®
75%							Other Ophthalmology3
50%
Feb 29	Mar 31	Apr 30	May 31	Jun 30	Jul 31	Aug 31	Sep 30

Note: lines represent rolling four-week sales average as compared to average weekly sales of Q4 2019. 1. Recent launches include Adakveo®, Aimovig®, Kisqali®, Kymriah®, Lutathera®, Mayzent®, Piqray®, Tabrecta® and Zolgensma®. 2. Growth drivers include Entresto®, Ilaris®, Jakavi®, Promacta®, Tafinlar®+Mekinist® and Xolair® . 3. Includes Luxturna®

18 Novartis Q3 Results | October 27, 2020 | Novartis Investor Presentation

2020 Novartis full year guidance

Barring unforeseen events; growth vs. PY in cc

Continuing operations | full year guidance1

vs. PY (cc)

Sales expected to grow mid single digit

• IM Division expected to grow mid single digit
• Sandoz expected to grow broadly in line with prior year, decreased from low single digit

Core operating income expected to grow low double digit to mid teens, upgraded from low double digit

1. Our guidance assumes that we see a continuation of the return to normal global healthcare systems including prescription dynamics, particularly ophthalmology, in Q4 2020. In addition, we assume that no Gilenya and no Sandostatin LAR generics enter in 2020 in the US.

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Q4 core OpInc expected to be impacted by higher investments including for Kesimpta® and Leqvio® (inclisiran)

Quarterly growth vs. PY
%pts, constant currency
Sales					Core Operating Income
					34			Low double digit
13							11		to mid teens
			Mid single			Mid to high
			Low to mid
						single digit
			digit
			single digit		6
		0
	-1								FY
Q1	Q2	Q3	Q4	FY	Q1	Q2	Q3	Q4
				Actual	Illustrative

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Expected currency impact for full year 2020 and 2021

Currency impact vs. PY

%pts, assuming late-October exchange rates prevail in 2020 and 2021

FX impact on Net Sales

			1		1			1 to 2
-3	-2	-3				-1
	FY Q1	Q2 Q3	Q4		FY FY
2019		2020		2021

FX impact on Core Operating Income

						0 to 1
			-2	-3	-4
-5		-5
-6
FY	Q1	Q2	Q3	Q4	FY	FY

2019

2020

2021

Actual Simulation

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Marie-France Tschudin

President, Novartis Pharmaceuticals

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Solid 9 months performance with strong momentum across growth drivers

Pharmaceuticals net sales	9M sales grew +6% cc, as growth drivers continued momentum
USD billion, growth in % cc		 Cosentyx® (USD 2,886m, +12% cc)
Recent launches1			 Entresto® (USD 1,781m, +48% cc)
Growth drivers2
Mature products3
+6%			Q3: Solid performance (sales +2% cc) despite COVID-19 impact
17.2	17.9		 Entresto® (USD 632m, +45% cc)
0.4	1.3	+37%	 Cosentyx® (USD 1,012m, +7% cc)
5.2	6.2	 Zolgensma® (USD 291m)
11.6			 Xiidra® (USD 99m, -3% cc)
10.3	-9%	 Lucentis® sales return to pre-COVID-19 level (USD 515m, +0% cc)
			 Mature ophthalmology4 products down -20%5
9M 2019	9M 2020
1. Zolgensma®, Xiidra®, Beovu® , Mayzent®, Aimovig®, Luxturna®, Kesimpta® , Enerzair® and Atectura®. 2. Cosentyx®, Entresto®, Xolair® , Ilaris® 3. All other brands. 4. Includes Luxturna®.	5. Includes generic impact primarily for Travatan®.

23 Novartis Q3 Results | October 27, 2020 | Novartis Investor Presentation

Cosentyx® maintains position in dermatology and outperforms the market in rheumatology

Sales evolution
USD million, % cc
Ex-US
US
+7%
937	1,012
336	372
601	640
Q3 2019	Q3 2020

COVID-19 pandemic still impacting market in Q3 both in US and EU

• Patient visits estimated to be 70%-90% versus pre-COVID-191
• New patient starts slower to recover in dermatology than in rheumatology, particularly in US2

Maintains strong dermatology position, outgrows rheumatology market3,4

• PsO YoY: US TRx +11% vs. market +7%, maintaining strong NBRx share ~16%
• SpA YoY: US TRx +20% vs. market +7%, leading NBRx share ~30%

Drivers of continued strong growth

• Increased biologics penetration in existing indications (all regions)
• LCM: 300mg auto-injector & PFS (CHMP +ve opinion), pediatric PsO (EMA)
• Geographic expansion current indications. e.g. China
• LCM new indications: Up to 6 indications, up to 3.5m addressable patients

Please see slide 142 for references TRx = Total Prescriptions; NBRx = New-to-Brand Prescriptions; nr-axSpA = non-radiographic Axial Spondyloarthritis; PedPsO = Pediatric Psoriasis; CHMP = Committee for Human Medicinal Products.

24 Novartis Q3 Results | October 27, 2020 | Novartis Investor Presentation

Entresto® delivers strong growth based on increasing use as foundational therapy in HFrEF

Sales evolution

USD million, % cc

Ex-US

US

+45%

632

430318

210

220314

Q3 2019

Q3 2020

Strong momentum across geographies

• US increased penetration in HFrEF population (+43% vs. PY); weekly NBRx reached >4,000 in September1
• China fueled ex-US growth (+104% cc vs. PY)
• Europe delivered strong double-digit growth (+36% cc vs. PY)

Confident in future growth prospects

• 4.8m HFrEF patients in G7 could benefit from Entresto®2
• Geographic expansion with Japan launch in CHF (August)
• Potential new indications
• • HFpEF review by US FDA ongoing
  • PARADISE-MItop-line results expected in H1 2021

HFrEF - Heart failure with reduced ejection fraction. HFpEF - Heart failure with preserved ejection fraction. CHF - Congestive Heart Failure. Post-AMI - post Acute Myocardial Infarction 1. US NBRx - IMS New to Brand w/e Oct 2nd, 2020 2. Eligible patients defined as prevalent HFrEF patients within each market's label. G7 = US, CA, JP, DE, FR, IT, UK

25 Novartis Q3 Results | October 27, 2020 | Novartis Investor Presentation

Beovu® now approved in more than 45 countries

Continuing to progress on clinical development and launch roll out

Launch progressing well

• US demand recovered vs. Q2, stabilized ~1,2k vials / week
• Relatively strong launch uptake in DE, JP given context: ~6% market share in wAMD in Germany8
  ~10% market share in wAMD in Japan9
• Correlation between fluid and vision in wAMD demonstrated in 3 brand agnostic post-hoc analyses1,2,3
• In two H2H studies Beovu® outperformed aflibercept in achieving superior fluid resolution (IRF and/or SRF)10
• Label updates received in 45 countries

Progressing clinical development in wAMD, DME

KITE pivotal DME study reported positive topline results1

• Non inferiority to aflibercept on BCVA at year 1
• Superior CST improvement versus aflibercept in a secondary endpoint (over week 40-52)
• 3-monthlydosing maintained for > 50% of patients11

KESTREL: Awaiting read-out of 2nd pivotal trial in DME2

KINGFISHER: Readout expected H1 2021 in DME

MERLIN: Readout expected in H1 2021 in wAMD

Please see slide 142 for references

26 Novartis Q3 Results | October 27, 2020 | Novartis Investor Presentation

Kesimpta® early launch indicators are positive

Potential to become a 1st choice high efficacy DMT for patients, physicians and payers

HCP engagement translating into adoption

90%+

(>5,800) of MS prescribing targets reached

90%+

of field force territories have adopted Kesimpta®

2.5%1

NBRx share 8 weeks post launch

Securing rapid and broad access

• Commercial bridging program
• Engagement plans ongoing and on-track
• CVS & Aetna commercial formularies, first Medicare win, Blue Cross Blue Shield regional accounts

Patient initiation seen as simple, easy and fast

• Favorable customer feedback from HCPs & patients

2020 Maximizing demand and patient initiations

Free goods account for vast majority of initial demand, NBRx key lead indicator

Please see slide 142 for references

27 Novartis Q3 Results | October 27, 2020 | Novartis Investor Presentation

Leqvio® receives CHMP positive opinion

25 million patients in EU with ASCVD, the primary cause of death1,2,3,4

Leqvio®

For the treatment of primary hypercholesterolaemia or mixed dyslipidaemia

Worldwide launch preparation progressing well

• EU approval expected Dec/Jan for all 27 EU member states, and others including UK, Norway
• FDA action date December 2020
• Germany launch H1 2021
• UK launch H2 2021, population health agreement with NHS on track
• New filings in Canada, Australia, Singapore, Indonesia
• China, clinical trial application accepted in June
• Launch expected to address non-clinical barriers: access, adherence, costs to the system

Please see slide 142 for references

28 Novartis Q3 Results | October 27, 2020 | Novartis Investor Presentation

Susanne Schaffert

President, Novartis Oncology

29 Novartis Q3 Results | October 27, 2020 | Novartis Investor Presentation

Strong momentum across growth brands and launches YTD, partly offset by generic erosion

Oncology net sales
USD billion, % cc
Recent launches1
Growth drivers2
Base Business4
Gx3
	+4%
10.6	.2	10.9
0.9	1.5
0.2
2.8		+32%
	3.4
2.8	0.9	2.7
		0%
4.2		3.3	-19%
9M 2019		9M 2020

9M net sales up +4% (cc)

• Recent launches and growth drivers up +32% (cc)

Q3: Continuing momentum of recent launches and growth drivers

• Kisqali® (USD 183m, +50% cc)
• Promacta® / Revolade® (USD 442m, +16% cc)
• Jakavi® (USD 335m, +18% cc)
• Tafinlar® + Mekinist® (USD 397m, +14% cc)
• Piqray® (USD 83m, +95% cc)
• Kymriah® (USD 122m, +51% cc)

Q3: Offset by Gx erosion, COVID-19 impact

• Generic impact mainly Afinitor®, Exjade®
• Lutathera® (USD 119m, -1% cc) continued impact from COVID-19

1. Recent launches include Kisqali®, Lutathera®,Kymriah®, Piqray®, Adakveo® , Tabrecta™ 2.Growth drivers include Promacta®/Revolade®, Tafinlar®+ Mekinist®, Jakavi® (marketed by Novartis ex-US) 3. Gx including Afinitor®, Exjade®, Glivec® and Sandostatin® 4. Base business - other brands

30 Novartis Q3 Results | October 27, 2020 | Novartis Investor Presentation

Kisqali®: Strong performance driven by positive overall survival results

Sales evolution

USD million, % cc

Ex-US

US

		+50%	183
123 0.2	.2	83
60	.9
		100
63
Q3 2019		Q3 2020

Strong performance despite market impact from COVID-19

• Sales driven by consistent overall survival (OS) benefit from two pivotal Ph3 trials1
• CDK market overall impacted by pandemic as patient screening suppressed, leading to continued decline in NBRx (down ~20%)2

Differentiation within the CDK4/6 class increasingly recognized

• Kisqali® provides selective and preferential inhibition of CDK4 over CDK6
• Highest rating of any CDK4/6i on ESMO Magnitude of Clinical Benefit Scale

Upcoming catalysts expected to drive growth

• MONALEESA-2OS results in metastatic setting expected H2 2021 (event-driven)
• NATALEE adjuvant study has potential to make Kisqali® the only CDK4/6i with broad applicability in intermediate and high risk populations (70% of adjuvant patients); final readout expected 2022

1. MONALEESA-7 and -3 2. IQVIA NBRx data: Jul/Aug 2020 average vs. average Q3 2019

31 Novartis Q3 Results | October 27, 2020 | Novartis Investor Presentation

Vas Narasimhan

Chief Executive Officer

32 Novartis Q3 Results | October 27, 2020 | Novartis Investor Presentation

Conclusion

Solid quarter with double-digit core OpInc growth

FY 2020 core OpInc expected to grow low double digit to mid teens

Mid / late stage pipeline advancing; Kesimpta® launched, Leqvio® positive CHMP opinion

Progressing on our journey to become an ESG leader

33 Novartis Q3 Results | October 27, 2020 | Novartis Investor Presentation

Appendix

Strong 9M operational performance from growth drivers

Key growth driver sales 9M 2020

Sales	Growth vs. PY	Growth vs. PY
USD Million	USD Million	cc
1,781	573	48%

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

666		491	nm
2,886		300	12%
1,267	231		24%
236	187		nm
503	178		59%
268	166		164%
153	153		nm
1,134	152		17%
333	151		82%
963	142		19%
633	140		30%

48%

38%

31%

25%

9M 2017

9M 2018

9M 2019

9M 2020

Adakveo®

Mayzent®

Beovu®

Piqray®

Xiidra®

Kymriah®

Lutathera®

Kisqali®

Ilaris®

Zolgensma®

Jakavi®

Tafinlar+Mekinist®

Promacta®

Entresto®

Cosentyx®

Other1

nm - not meaningful

1. Includes Tasigna®, Xolair®, Aimovig®, Tabrecta ®, Luxturna® , Kesimpta®, Enerzair ® and Atectura®

35 Novartis Q3 Results | October 27, 2020 | Novartis Investor Presentation

9M 2020 FCF decreased to USD 8.3bn

Continuing operations1 free cash flow2
USD billion
				-12%				Key drivers vs. PY:
							+	Higher operating income
9.4		8.3
								(adjusted for non-cash items)
										−	Legal settlements
										−	Lower divestment proceeds
		9M 2019			9M 2020

1. Refers to continuing operations as defined on page 42 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 54 of the Condensed Interim Financial Report

36 Novartis Q3 Results | October 27, 2020 | Novartis Investor Presentation

Strong governance, enhanced processes and focus on material ESG factors improved rating agencies scores

AgencyScore

2,7 Risk score Controversy level

2,8		ESG score
	2	ESG score
2,8		WAKI
		ESG score
		MSCI Global Compact
3		Ranking
4		Climate
		Water

4

ESG score

Score
Current	Previous
▲ 21	30
▲ 3	5
► B-	B-
▲ 4.7	4.1
▲ 4.9	4.3
▲ A	BBB

• Watchlist Fail

▲3.2	2.9
► A-	A-
▲ A	B
▼74	77

Sector Ranking1

Current Previous

▲ 3 / 392	14	/ 165
► 4 / 395	3	/	233
▲ 1	/ 6	6	/	6
▲ 2-4 / 6	8	/	11
▲ 2	/ 20	3	/	20
▲ 3	/ 155	4-9 / 15
▼ 8	/ 61	4	/	50

Score change drivers

Strong management of material ESG issues

Strong corporate governance, positive on settlement of legacy issues

Outperforming on access, ethics. Recent update on US generics industry controversy

Improvement across each pillar

Recent settlements, ethics policies, employee engagement strategy. R&D, product portfolio address access in underserved markets and robust vs. industry peers

Industry leader in access to medicine management (Access Principles)

Leadership level: Strong water policies, governance, environmental strategy

US generics industry controversy; Novartis remains member of DJSI World and EU index6

Top tier sector leading performance

Novartis access programs / management best-in-class

WAKI - Weighted-average key issue score 1. Peer group as defined by each ESG rating agency	2. 2020 / 2019 scores 3. Published every 2nd year. Result shown shows 2018 / 2016 scores 4. 2019 / 2018 scores 5. Based on Novartis
official peer group 6. Novartis has been a member of the DJSI World and EU index since 2002	7. Updated October 15, 2020 8. Updated September 25, 2020

37 Novartis Q3 Results | October 27, 2020 | Novartis Investor Presentation

2020 expected pipeline milestones

		H1 2020			H2 2020		✓ Achieved	✕ Missed
Regulatory		Beovu®	nAMD (EU/JP)	✓	Adakveo®		Sickle cell disease (EU)	✓2
	Cosentyx®	nr-axSpA (EU/US)	✓	Tabrecta™ (capmatinib)		NSCLC (US/JP)	✓
decisions
	Cosentyx®	AS (CN)	✓	Cosentyx®		Pediatric psoriasis (EU)	✓
and opinions
	Ofatumumab (OMB157)	Relapsing MS (US)	✓	Cosentyx®		nr-axSpA (JP)	✓
				(H2 2020)
		Piqray®	HR+/HER2- aBC with	✓	Entresto®		HFpEF (US)	H1 2021
			PIK3CA mutation (EU)
		Enerzair®	Asthma (EU/JP)	✓	Leqvio® (inclisiran)		Hyperlipidemia (US)
							Hyperlipidemia (EU) - originally 2021	✓2
		Tafinlar® & Mekinist®	Adjuvant melanoma (CN)	✓	Xolair®		Nasal Polyposis (US/EU)	✓3
		Xiidra®	DED (EU)	✕
		Zolgensma® IV	SMA (EU/JP)	✓
Major		Entresto®	HFpEF (US)	✓	Alpelisib (BYL719)		PROS (US)	H1 2021
						(COVID related)
expected		Leqvio® (inclisiran)	Hyperlipidemia (EU)	✓	AVXS-101 IT		SMA (US)	✕4
submissions					Cosentyx®		Juvenile PsA / enthesitis-related	H1 2021
							arthritis (US/EU)
					Spartalizumab (PDR001)		Metastatic melanoma (US/EU)	✕
					and Tafinlar® & Mekinist®
					177Lu-PSMA-617		mCRPC (US)	2021
Major		Entresto®	Post-acute MI1	✓	Asciminib (ABL001)		CML 3L	✓
	Tropifexor (LJN452)	NASH	✓	Beovu®		DME	✓
expected trial
	UNR844	Presbyopia	✓	Jakavi®		chronic GVHD	✓
readouts*
				Kisqali®		aBC (MONALEESA-2 OS)	2021
					177Lu-PSMA-617		mCRPC	H1 2021
*Achieved = on-time readout of data, irrespective of trial outcome	1. Planned study readout 2021; preplanned DMC interim analysis readout completed in March	2. Received positive CHMP opinion 3. European Commission approval
received, US filing underway	4. FDA recommended pivotal confirmatory study to supplement existing STRONG data

38 Novartis Q3 Results | October 27, 2020 | Novartis Investor Presentation

Our Q3 pipeline projects at a glance

Phase 1/2	Phase 3	Registration	Total
ONCOLOGY	52	21	1	74
PHARMACEUT ICALS	64	20	6	90
Cardiovascular, Renal, Metabolism	12	4	2	18
Immunology, Hepatology, Dermatology	27	6	1	34
Neuroscience	6	3	1	10
Ophthalmology	5	3	0	8
Respiratory	8	3	1	12
Global Health	6	1	1	8
BIOSIMILARS	0	1	0	1
Total	116	42	7	165
CRM: Cardiovascular, Renal & Metabolism. IHD: Immunology, Hepatology & Dermatology.	NS: NeuroScience.

39 Novartis Q3 Results | October 27, 2020 | Novartis Investor Presentation

Novartis pipeline in Phase 1 (1 of 2)

38 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	ADPT01	-	TNBC (combos)	Colorectal Cancer (combos)
ADPT03	ADPT03		Sickle cell Anemia
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 (combo)
HDM201	HDM201 + MBG453, venetoclax	MDM2 Inhibitor	Haematological malignancy
INC424	Jakavi	JAK1 / 2 Inhibitor	Myelofibrosis (combination)
JBH492	JBH492	-	Haematological Malignancy
JEZ567	JEZ567	CD123 CART	AML
KAZ954	KAZ954	-	Solid tumors
LHC165	LHC165 + spartalizumab	TLR7 Agonist	Solid tumors
LXF821	LXF821	EGFR CART	Glioblastoma multiforme
LXH254	LXH254 (combos)	cRAF Inhibitor	Solid tumors	Solid tumors
MAK683	MAK683	EED Inhibitor	Cancers
MCM998	MCM998, LXG250	BCMA CART, CD19 CART	Multiple myeloma
MIK665	MIK665	MCL1 Inhibitor	AML (combo)
NIS793	NIS793, spartalizumab	TGFB1 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	AML	Solid tumors (combo)
PHE885	PHE885	BCMA Cell therapy	Multiple Myeloma
SQZ622	SQZ622	CD123xCD3 Modulator	AML
TNO155	TNO155	SHP2 Inhibitor	Solid tumors (single agent)	Solid tumors (combo)	Solid tumors (combo)
VAY736	ianalumab + ibrutinib	BAFF-R Inhibitor	Haematological malignancy
VOB560	VOB560	-	Cancers
VPM087	gevokizumab	IL1B Antagonist	CRC 1st line
WNT974	WNT974 + spartalizumab	Porcupine Inhibitor	Solid tumors
WVT078	WVT078	-	Multiple myeloma
YTB323	YTB323 ± ibrutinib	CD19 CART	Haematological malignancy

40 Novartis Q3 Results | October 27, 2020 | Novartis Investor Presentation

Novartis pipeline in Phase 1 (2 of 2)

38 lead indications

Lead indication

Immunology, Hepatology, Dermatology

Global Health

Code

Name

Mechanism

Indication(s)

Code

Name

Mechanism

Indication(s)

CEE321

CEE321

Pan JAK Inhibitor

AD

KAF156

ganaplacide

-

Malaria prophylaxis

DFV890

DFV890

-

Anti-inflammatory therapy

FIA586

FIA586

-

NASH

MAS825

MAS825

-

Inflammatory diseases

MHS552

MHS552

-

Autoimmune Indications

MHV370

MHV370

-

Sjögren's

SLE

Neuroscience

Code

Name

Mechanism

Indication(s)

OAV201

AVXS-201

MECP2 gene therapy

Rett syndrome

LMI070

branaplam

mRNA splicing modulator

Huntington

Respiratory Disease

Code

Name

Mechanism

Indication(s)

LTP001

LTP001

-

Respiratory Diseases

Cardiovascular, Renal, Metabolism

Code

Name

Mechanism

Indication(s)

HSY244

HSY244

-

Atrial fibrillation

MBL949

MBL949

-

Obesity related diseases

41 Novartis Q3 Results | October 27, 2020 | Novartis Investor Presentation

Novartis pipeline in Phase 2

Oncology

Code	Name	Mechanism	Indication(s)
BYL719	alpelisib	PI3Kα inhibitor	PROS
BLZ945	BLZ945	CSF-1 Inhibitor	Solid tumors
INC280	capmatinib	Met Inhibitor	NSCLC EU1)	Solid tumors	NSCLC	NSCLC
					(Combo)	(Combo)
INC424	Jakavi®	JAK1 Inhibitor	Myelofibrosis (combination)	Pediatrics Acute	Pediatrics
					GVHD	Chronic GVHD
MBG453	sabatolimab	TIM3 Antagonist	Unfit AML
NIR178	NIR178, spartalizumab	Ad2AR Inhibitor, PD1 Inhibitor	Cancers
PDR001	spartalizumab	PD1 Inhibitor	Metastatic melanoma (combo)
SEG101	crizanlizumab	P-selectin Inhibitor	Ped sickle cell anaemia with
			crisis

Immunology, Hepatology, Dermatology

Code	Name	Mechanism	Indication(s)
ADTP02	ADTP02	-	NASH (Combos)
AIN457	Cosentyx®	IL17A Inhibitor	GCA		Lichen Planus
CFZ533	iscalimab	CD40 Inhibitor	Renal Tx		Sjögren's	HS	Liver Tx
LJC242	tropifexor&cenicriviroc	FXR agonist, CCR2 Inhibitor	NASH (combos)
LJN452	tropifexor	FXR agonist	NASH		NASH (combos)
LNA043	LNA043	ANGPTL3 Agonist	Osteoarthritis
LOU064	remibrutinib	BTK Inhibitor	CSU / CIU		Sjögren's
LRX712	LRX712	-	Osteoarthritis
LYS006	LYS006	Anti-inflammatory	Acne		Colitis ulcerative	HS
VAY736	ianalumab	BAFF-R Inhibitor	Sjögrens		AIH	SLE

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

1. Approved in US & JP

				30 lead indications
								Lead indication
Neuroscience
Code	Name		Mechanism	Indication(s)
BAF312	Mayzent®		S1P1 Modulator	Stroke
BLZ945	BLZ945		CSF-1 Inhibitor	ALS
LMI070	branaplam		mRNA splicing modulator	SMA
MIJ821	MIJ821		NR2B Inhibitor	Depression
Respiratory Disease
Code	Name		Mechanism	Indication(s)
CMK389	CMK389		IL-18 Inhibitor	Pulmonary sarcoidosis
CSJ117	CSJ117		TSLP Inhibitor	Asthma
DFV890	DFV890	-	COVID-19 related pneumonia
LOU064	remibrutinib		BTK Inhibitor	Asthma
MAS825	MAS825	-	COVID-19 related pneumonia
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®		Angiotensin II Receptor	nHCM
			Neprilysin Inhibitor (ARNI)
LMB763	nidufexor		FXR Agonist	Diabetic Nephropathy
LNP023	iptacopan		CFB Inhibitor	PNH	IgAN	C3G			iMN	aHUS
LTW980	LTW980	-	Hypertriglyceridemia
Global Health
Code	Name		Mechanism	Indication(s)
AFQ056	AFQ056		mGluR5 Antagonist	Cocaine use disorder
KAE609	cipargamin		PfATP4 inhibitor	Malaria severe	Malaria uncomplicated
KAF156	ganaplacide	-	Malaria uncomplicated
LXE408	LXE408		Protozoan Inhibitor	Visceral leishmaniasis

42 Novartis Q3 Results | October 27, 2020 | Novartis Investor Presentation

Novartis pipeline in Phase 3

Oncology

Code	Name	Mechanism	Indication(s)
177Lu-PSMA-617	177Lu-PSMA-617	Targeted Radioligand Therapy	mCRPC
177Lu-	Lutathera®	Targeted Radioligand Therapy	GEP-NET 1L G3
oxodotreotide1)
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
			lymablphoma		ALL, pediatrics		relapse
					and young
					adults
ETB115	Promacta®	Thrombopoietin receptor (TPO-R)	Radiation sickness syndrome		Food effect free formulation
		Agonist
INC424	Jakavi®	JAK1 Inhibitor	Acute GVHD		Chronic GVHD
LEE011	Kisqali®	CDK4 Inhibitor	HR+/HER2- BC (adj)
MBG453	sabatolimab	TIM3 Antagonist	HR-MDS
SEG101	crizanlizumab	P-selectin Inhibitor	Sickle cell anemia new formulation

Immunology, Hepatology, Dermatology

Code	Name	Mechanism	Indication(s)
AIN457	Cosentyx®	IL17A Inhibitor	Lupus Nephritis	Hidradenitis	AS H2H	PsA / axSpA
				suppurativa		IVIV
ACZ885	canakinumab	IL-1b Inhibitor	COVID-19 induced respiratory disease
QGE031	ligelizumab	IgE Inhibitor	CSU / CIU

Ophthalmology

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

5 lead indications

Lead indication

Neuroscience

Code	Name	Mechanism	Indication(s)
AMG334	Aimovig®	CGRPR antagonist	Ped Migraine
BAF312	Mayzent®	S1P1 Modulator	Ped MS
OAV101	AVXS-101	Gene Therapy, Survival motor	SMA IT2)
		neuron (SMN1) gene

Respiratory Disease

Code	Name	Mechanism	Indication(s)
IGE025	Xolair®	IgE Inhibitor	Food allergy	Auto-injector
INC424	Jakavi®	JAK1 Inhibitor	COVID-19 related pneumonia3)

Cardiovascular, Renal, Metabolism

Code	Name	Mechanism	Indication(s)
KJX839	inclisiran	siRNA (regulation of LDL-C)	CVRR-LDLC
LCZ696	Entresto®	Angiotensin II Receptor Neprilysin Inhibitor	Post-AMI	Pediatric HF4)
		(ARNI)
TQJ230	pelacarsen	ASO targeting Lp(a)	CVRR-Lp(a)

Global Health

Code	Name	Mechanism	Indication(s)
COA566	Coartem®	-	Malaria uncomplicated, new formulation <5kg patients

Biosimilars

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

1. 177Lu-dotatate in US. 2. FDA placed a partial hold on AVXS-101 intrathecal clinical trials for SMA patients based on findings in a small pre-clinical animal study. 3. Not aimed at label change. 4. Approved in US.

43 Novartis Q3 Results | October 27, 2020 | Novartis Investor Presentation

Novartis pipeline in registration

2 lead indications

Lead indication

Oncology

Respiratory Disease

Code

Name

Mechanism

Indication(s)

Code

Name

Mechanism

Indication(s)

SEG101

Adakveo®

P-selectin Inhibitor

Sickle cell disease1)

IGE025

Xolair®

IgE Inhibitor

Nasal polyps2)

Immunology, Hepatology, Dermatology

Cardiovascular, Renal, Metabolism

Code

Name

Mechanism

Indication(s)

Code

Name

Mechanism

Indication(s)

AIN457

Cosentyx®

IL17A Inhibitor

300 mg AI

KJX839

inclisiran

siRNA (regulation of LDL-C)

Hyperlipidemia

LCZ696

Entresto®

Angiotensin II Receptor

HFpEF

Neprilysin Inhibitor (ARNI)

Neuroscience

Global Health

Code

Name

Mechanism

Indication(s)

Code

Name

Mechanism

Indication(s)

OMB157

ofatumumab

CD20 Antagonist

r MS 3)

LAM320

Lamprene®

SMPD1 Inhibitor

Tuberculosis4)

1. Approved in US, CHMP pos. opinion received. 2. Approved in EU. 3. Approved in US as Kesimpta®. 4. WHO Pre-Qualification.

44 Novartis Q3 Results | October 27, 2020 | Novartis Investor Presentation

Novartis submission schedule

New Medical Entities: Lead and supplementary indications

LEAD INDICATIONS

NEW INDICATIONS

2021

2022

2023

≥2024

177Lu-PSMA-617

Lead

ligelizumab

Lead

iscalimab

Lead

177Lu-NeoB

Lead

LNA043

Lead

AVXS-201

Lead

pelacarsen

Lead

AAA617A1	QGE031	CFZ533	AAA603A1	Osteoarthritis	OAV201	TQJ230
mCRPC 3L	CSU / CIU	Renal Tx	Multiple Solid Tumors		Rett syndrome	CVRR-Lp(a)

asciminib

Lead

ECF843

Lead

177Lu-PSMA-R2

Lead

tropifexor

Lead

LMI070

Lead

ganaplacide

Lead

ABL001

Dry eye

AAA602A1A1

LJN452

Huntington's disease

KAF156

CML 3L

Prostate cancer

NASH

Malaria uncomplicated

sabatolimab

Lead

iptacopan

Lead

gevokizumab

Lead

tropifexor&cenicriviroc

Lead

MIJ821

Lead

cipargamin

Lead

MBG453

LNP023

VPM087

LJC242

Depression

KAE609

HR-MDS

PNH

1st line CRC / 1st line RCC

NASH

Malaria severe

spartalizumab

Lead

CPK850

Lead

QBW251

Lead

LXE408

Lead

PDR001

RP

COPD

Visceral leishmaniasis

Malignant melanoma (combo)

CEE321

Lead

UNR844

Lead

CSJ117

Lead

mavoglurant

Lead

Atopic Dermatitis

Presbyopia

Asthma

AFQ056

Cocaine use disorder

ianalumab

Lead

SAF312

Lead

VAY736

COSP

Sjögren's syndrome

remibrutinib

Lead

LOU064

CSU / CIU

canakinumab

LCM

canakinumab

LCM

capmatinib

LCM

iscalimab

LCM

tropifexor

LCM

iptacopan

LCM

ACZ885

ACZ885

INC280

CFZ533

LJN452

LNP023

NSCLC 2L

Adjuvant NSCLC

Solid tumors

Liver Tx

NASH (combos)

iMN

Canakinumab1)

LCM

iptacopan

LCM

crizanlizumab

LCM

iscalimab

LCM

LMI070

LCM

cipargamin

LCM

ACZ885

LNP023

SEG101

CFZ533

SMA

KAE609

NSCLC 1L

C3G

Sickle cell anaemia with crisis ped

Sjögren's syndrome

Malaria uncomplicated

iptacopan

LCM

sabatolimab

LCM

ianalumab

LCM

leqvio

LCM

LNP023

MBG453

VAY736

KJX839

IgAN

Maintenance for MRD+ AML

AIH

CVRR-LDLC

iptacopan

LCM

sabatolimab

LCM

remibrutinib

LCM

LNP023

MBG453

LOU064

aHUS

Unfit AML

Sjögren's syndrome

1. Depending on timing of final read-out submission may move to early 2022.

45 Novartis Q3 Results | October 27, 2020 | Novartis Investor Presentation

Novartis submission schedule

Supplementary indications for existing brands

20211)

alpelisib, BYL719

PROS

Kymriah

tisagenlecleucel, CTL019 r/r DLBCL 1st relapse

Kymriah

tisagenlecleucel, CTL019 r/r Follicular lymphoma

Tafinlar

dabrafenib, DRB436 HGG/LGG - Pediatrics

Jakavi

ruxolitinib, INC424 Chronic GVHD

Jakavi

ruxolitinib, INC424 Acute GVHD

Beovu

brolucizumab, RTH258

DME

Xolair

omalizumab, IGE025 Auto-injector

Entresto

sacubitril/valsartan, LCZ696 Post-AMI

2022

2023

≥2024

LCM

Adakveo

LCM

Kisqali

LCM

Jakavi

LCM

Kymriah

LCM

Cosentyx

LCM

Aimovig

LCM

crizanlizumab, SEG101

ribociclib, LEE011

ruxolitinib, INC424

tisagenlecleucel, CTL019

secukinumab, AIN457

erenumab, AMG334

Sickle cell anaemia new formulations

HR+/HER2- BC (adj)

Pediatrics Chronic GVHD

1L high risk ALL, pediatrics & young adults

GCA

Pediatric Migraine

LCM

Promacta

LCM

Lutathera

LCM

Jakavi

LCM

Piqray

LCM

Cosentyx

LCM

Mayzent

LCM

eltrombopag, ETB115	177Lu-oxodotreotide2)	ruxolitinib, INC424	alpelisib, BYL719	secukinumab, AIN457	siponimod, BAF312
Radiation sickness syndrome	GEP-NET 1L G3	Pediatrics Acute GVHD	HNSCC 2/3L	Lichen Planus	Pediatric MS

LCM

Promacta

LCM

Piqray

LCM

Jakavi

LCM

Piqray

LCM

Cosentyx

LCM

eltrombopag, ETB115

alpelisib, BYL719

ruxolitinib, INC424

alpelisib, BYL719

secukinumab, AIN457

Food effect free formulation

TNBC

Myelofibrosis (combination)

HER2+ adv BC

Lupus Nephritis

LCM

Cosentyx

LCM

Piqray

LCM

Kymriah

LCM

Tabrecta

LCM

Coartem

LCM

secukinumab, AIN457

alpelisib, BYL719

tisagenlecleucel, CTL019

capmatinib, INC280

artemether + lumefantrine, CCA566

PsA / axSpA IVIV

Ovarian cancer

r/r DLBCL (+ pembro)

Solid tumors

Malaria uncompl., formula for <5kg

LCM

Cosentyx

LCM

Tafinlar

LCM

secukinumab, AIN457

dabrafenib, DRB436

AS H2H

Thyroid cancer

LCM

Cosentyx

LCM

Beovu

LCM

secukinumab, AIN457

brolucizumab, RTH258

Hidradenitis suppurativa

Diabetic retinopathy

LCM

Xolair

LCM

Beovu

LCM

omalizumab, IGE025

brolucizumab, RTH258

Food allergy

RVO

LCM

Entresto EU3)

LCM

denosumab

BioS

sacubitril/valsartan, LCZ696

GP2411

Pediatric HF

anti RANKL mAb

LCM

1. AVXS-101 IT filing timelines TBC based on HA feedback. 2. 177Lu-dotatate in US. 3. Approved in US

46 Novartis Q3 Results | October 27, 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 II 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 adult
	granules (12.5, 31.25 mg in capsules); liquid formulation	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 heart	Pediatric patients with heart failure due to systemic left
Target Patients	ventricle systolic dysfunction who have completed study
failure due to systemic left ventricle systolic dysfunction
	CLCZ696B2319
	2022; (Analysis of 110 pts from Part 2 formed the basis for
	pediatric submission in Apr-2019 and approval by the US FDA
Read-out Milestone(s)	in Oct-2019 for the treatment of symptomatic HF with systemic	2022
	left ventricular systolic dysfunction in children aged 1 year and
	older)
Publication	TBD	TBD

49 Novartis Q3 Results | Oct 27, 2020 | Novartis Investor Presentation

Entresto® - Angiotensin II 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
Measures	Composite Score (GCCS)	cardiovascular (CV) death or heart failure (HF) hospitalization
	• Sacubitril/valsartan 50, 100, and 200 mg bid with placebo	• Sacubitril/valsartan 50 mg, 100 mg, 200 mg bid/placebo of
Arms/Intervention	of valsartan	enalapril
• Valsartan 40, 80, and 160 mg bid tablets with placebo for	• Enalapril 2.5 mg, 5 mg, 10 mg bid / placebo of
	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
Read-out Milestone(s)	2022	Primary: Q1-2019(actual); Extension (open-label):H1-2021
Publication	TBD	Submitted for Q4-2020: Primary manuscript in Circ J

50 Novartis Q3 Results | Oct 27, 2020 | Novartis Investor Presentation

Entresto® - Angiotensin II 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 baseline
Measures
hospitalizations	to Week 24
	•	Sacubitril/valsartan or placebo 50 mg, 100 mg, and 200 mg	•	Sacubitril/valsartan 50 mg, 100 mg and 200 mg bid and
		matching placebo
Arms/Intervention		bid
	•	Enalapril 2.5 mg, 5 mg and 10 mg bid and matching 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
Read-out Milestone(s)	2019 (actual)	2019 (actual)
	•	Sep-2019: Primary manuscript (ARNI in HFpEF. Solomon S
		et al; NEJM. DOI: 10.1056/NEJMoa1908655)	• Study design (Wachter et al; ESC-HF),May-2020
	•	Mar-2020: Published (NTproBNP, putative placebo analysis)	•	Primary data presented at ESC latebreaker, Aug-2020
Publication	• Jun-2020: Submitted (renal outcomes, cognitive function)	• Baseline data publication in EJHF (expected publication Q4-
	• Q4-2020 Planned: Urgent HF visits, regional differences,		2020), accepted Sep-2020
		win ratio, adjudicated vs reported endpts; Subgroups (mode	• Planned Primary Publication High Tier Journal in H1-2021
		of death, MRA, age, gender)

51 Novartis Q3 Results | Oct 27, 2020 | Novartis Investor Presentation

Entresto® - Angiotensin II Receptor Neprilysin Inhibitor (ARNI)

Study	NCT02924727 PARADISE-MI (CLCZ696G2301)
Indication	Post-acute myocardial infarction
Phase	Phase 3
Patients	5,670
Primary Outcome	Time to the first occurrence of a confirmed composite
endpoint (cardiovascular (CV) death, heart failure (HF)
Measures
hospitalization, or outpatient heart failure)
	• Sacubitril/valsartan 50 mg, 100 mg, 200 mg bid / placebo
Arms/Intervention	of ramipril/valsartan
• Ramipril 1.25 mg, 2.5 mg, and 5 mg bid / placebo of
	sacubitril/valsartan / placebo for valsartan

Target Patients

Read-out Milestone(s)

Publication

Post-AMI patients with evidence of LV systolic dysfunction and/or pulmonary congestion, with no known prior history of chronic HF

H1-2021

• Q4-2020- Planned: PARADISE-MI study design / baseline characteristics
• Planned primary data presentation and publication in H2- 2021

52 Novartis Q3 Results | Oct 27, 2020 | Novartis Investor Presentation

KJX839 - siRNA (regulation of LDL-C)

Study	NCT03060577 ORION-3 (CKJX839A12201E1)	NCT03705234 ORION-4 (CKJX839B12301)
	Hypercholesterolemia inc. Atherosclerotic Cardiovascular	Hypercholesterolemia inc. Heterozygous Familial
Indication	Disease (ASCVD) and ASCVD risk equivalents Heterozygous
Hypercholesterolaemia (HeFH)
	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, defined
	LDL-C reduction at Day 210 for Group 1 subjects	as:
Primary Outcome	Changes in other lipids and lipoproteins and reduction of LDL-	• Coronary heart disease (CHD) death;
Measures	C of more than 50% for patients that are above LDL-C goal ;	• Myocardial infarction;
	longer term exposure and safety.	• Fatal or non-fatal ischaemic stroke; or
		• Urgent coronary revascularization procedure

Arms/Intervention

Target Patients

• Group 1 - inclisiran 300mg sc every 6 months until Day 720	Arm 1: every 6 month treatment KJX839 300mg (given by
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 weeks	duration of about 5 years
for 360 days, followed by inclisiran 300mg on Day 360, Day	Arm 2: matching placebo (given bysubcutaneous injection on
450 and then every 6 months for a planned duration of 4	the day of randomization, at 3 months and then every 6-
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 +/-
ezetimibe therapy

Read-out Milestone(s)	2022	2025
Publication	TBD	TBD

53 Novartis Q3 Results | Oct 27, 2020 | Novartis Investor Presentation

KJX839 - siRNA (regulation of LDL-C)

Study	NCT03851705 ORION-5 (CKJX839A12302)	NCT03814187 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)	2,991 entered the study

Primary Outcome Measures

Arms/Intervention

•	LDL-C reduction at Day 150	• Proportion of subjects achieving prespecified low density
lipoprotein cholesterol (LDL-C) targets at end of study
•	Changes in PCSK9, other lipids and lipoproteins
• Safety and tolerability profile of long term use of inclisiran
• Part 1: inclisiran 300mg on Day 1 and Day 90 or placebo	Inclisiran 300mg on day 1 (placebo patients entered into study
	on Day 1 and Day 90	from ORION 9, 10 & 11) or placebo on Day 1 (inclisiran
• Part 2: inclisiran on Day 180 for patients who were	patients entered into study from ORION 9, 10 & 11) then
	randomized to the placebo group only, inclisiran on Day	inclisiran 300mg on Day 90 and every 6 months for a planned
	270 and then every 6 months for a planned duration of 2	duation of 3 years
	years for all patients

	Patients with HoFH with background statin +/- ezetimibe
Target Patients	therapy

Patients with HeFH or pre-existing atherosclerotic cardiovascular disease (ASCVD) on background statin +/- ezetimibe therapy and risk equivalents (patients from ORION 9, 10 & 11 studies)

Read-out Milestone(s)	Primary: Q3-2020(actual); Final: H2-2021	2023
Publication	TBD	TBD

54 Novartis Q3 Results | Oct 27, 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	~110	72

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

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

	•	Placebo
	•	LNP023 Dose 1 - 10mg bid	• LNP023 Dose - 200mg bid
Arms/Intervention	•	LNP023 Dose 2	- 50mg bid	•	LNP023 Dose - 50mg bid
	•	LNP023 Dose 3	- 200mg bid	•	Rituximab
	• LNP023 Dose 4	- 100mg bid (Part 2 only)

Target Patients

Read-out Milestone(s)

Publication

	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
H1-2021	2022
TBD	TBD

55 Novartis Q3 Results | Oct 27, 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	95 (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)

	Increasing doses of LNP023 up to 200mg bid:
Arms/Intervention	• Cohort A: Native kidney patients	• Open-label LNP023 200mg bid
	• Cohort B: Kidney transplanted patients
Target Patients	Patients with C3 glomerulopathy	Patients with C3 glomerulopathy
Read-out Milestone(s)	H1-2021	2024
Publication	Interim analysis data from Cohort-A presented at American	TBD
Society of Nephrology (ASN 2020)

56 Novartis Q3 Results | Oct 27, 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	16	13
Primary Outcome	Reduction of chronic hemolysis, based on LDH level at Week	Reduction of PNH associated hemolysis, based on
percentage of patients with 60% reduction in LDH or LDH
Measures	13
below upper limit of normal up to 12 weeks of treatment.
	• Cohort 1: 10 patients receiving LNP023 200mg bid, in

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

Arms/Intervention • 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

Read-out Milestone(s)

Publication

Patients with PNH, showing signs of active hemolysis despite	Patients with PNH, showing signs of active hemolysis, not
treatment with SoC (defined as an antibody with anti C5	treated with any other complement inhibitor less than 3
activity).	months prior to study start Day 1
Primary: Q2-2020(actual)	Primary: Q2-2020(actual)
Extension: 2023	Extension: 2022
Antonio M. Risitano, MD, PhD1 et al. Presented at EBMT	TBD
2020 congress

57 Novartis Q3 Results | Oct 27, 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
	Patients with a history of Myocardial infarction or Ischemic
Target Patients	Stroke, or a clinically significant symptomatic Peripheral Artery
	Disease, and Lp(a) ≥ 70 mg/dL
Read-out Milestone(s)	2024
Publication	TBD

58 Novartis Q3 Results | Oct 27, 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	681	260

Primary Outcome Measures

Arms/Intervention

Cohorts 1 and 2-mean iBox risk prediction score at 12 months.	Change in EULAR Sjögren's syndrome Disease Activity Index
Integrative score that will provide a prediction of graft survival	(ESSDAI) score and EULAR Sjögren's syndrome Patient
at year 5	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
Read-out Milestone(s)	2022	Primary (week 24): H1-2022; Final: 2023
Publication	Manuscript to be submitted in Q4-2020	Manuscript of PoC trial published in The Lancet-
Rheumatology January 23, 2020

60 Novartis Q3 Results | Oct 27, 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 Loss
Measures	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

Read-out Milestone(s)

Publication

Liver transplant recipients

Primary (month 12): 2022; Final: 2023

TBD

61 Novartis Q3 Results | Oct 27, 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 Measures

Arms/Intervention

PASI 90 response and IGA mod 2011 0 or 1 response after 16	PASI 75 response and IGA mod 2011 0 or 1 response after 12
weeks of treatment	weeks of treatment
• Secukinumab 300 mg every 2 weeks after weekly doses till	• Secukinumab 2 mL (300 mg) auto-injector
Week 4	• Secukinumab 2 x 1 mL (150 mg each) prefilled syringe
• Secukinumab 300 mg every 4 weeks after weekly doses till	• Placebo 2 mL auto-injector
Week 4	• Placebo 2 x 1 mL prefilled syringe

Target Patients	Subjects (≥90kg) with moderate to severe plaque psoriasis	Subjects with moderate to severe plaque psoriasis
Read-out Milestone(s)	Q3-2020(actual)	Primary (week 16): Q1-2020(actual); Final: Q4-2020
Publication	Publication (primary efficacy) planned in Q4-2020	Publication (16 week primary results) planning in Q4-2020

62 Novartis Q3 Results | Oct 27, 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
Read-out Milestone(s)	Long term safety 2023	2023
Publication	Publication planned in 2021	Publication planned in 2021

63 Novartis Q3 Results | Oct 27, 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
Investigators' Global Assessment (IGA) 0 or 1 response at
Measures
week 12
	•	Secukinumab 300 mg
Arms/Intervention	•	Secukinumab 150 mg
	•	Placebo
Target Patients	Patients with moderate to severe chronic plaque-type
psoriasis with or without psoriatic arthritis comorbidity
Read-out Milestone(s)	Q1-2019(actual)
	•	Week 16 results: Poster presented at: 2019 American
		Academy of Dermatology (AAD) Annual Meeting,
Publication	•	March 1-5, 2019, Washington, D.C.
•	52-week results: Poster at EADV 2019, Madrid 9-13
		October, 2019
	•	Manuscript publication in Q4-2020

64 Novartis Q3 Results | Oct 27, 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 enthesitis-	Patients with juvenile idiopathic arthritis subtypes of juvenile
related arthritis	psoriatic arthritis and enthesitis related arthritis
Read-out Milestone(s)	H1-2021	2025
Publication	Planned publication in 2021	TBD

65 Novartis Q3 Results | Oct 27, 2020 | Novartis Investor Presentation

Cosentyx® - Anti IL-17

Study	NCT01649375 MEASURE 2 (CAIN457F2310)	NCT01752634 FUTURE 2 (CAIN457F2312)
Indication	Ankylosing spondylitis	Psoriatic arthritis
Phase	Phase 3	Phase 3
Patients	219	399
Primary Outcome	Assessment of SpondyloArthritis International Society / ASAS	Proportion of subjects achieving American College of
Measures	20 response	Rheumatology 20 (ACR20) response criteria
	•	Secukinumab 75 mg	•	Secukinumab (AIN457) 150 mg s.c.
	•	Secukinumab (AIN457) 75 mg s.c.
Arms/Intervention	•	Secukinumab 150 mg
•	Secukinumab (AIN457) 300 mg s.c.
	•	Placebo
	•	Placebo s.c.
Target Patients	Patients with active ankylosing spondylitis	Patients with active psoriatic arthritis
Read-out Milestone(s)	2018 (actual)	2019 (actual)
	•	Primary 52 week results: Baeten D & Sieper J, et al. N Engl
		J Med 2015;373:2534-48
	• 2 year results: Marzo-Ortega, et al. Arthritis Care Res 2017	• Primary results: McInnes IB, et al. Lancet. 2015;386:1137-
		Feb 24. doi: - 10.1002/acr.23233		46
Publication	•	3 year results: Marzo-Ortega, et al. RMD 2017	• 2 years results: McInnes et al, Rheumatology 2017;56:1993-
	•	5 year results: EULAR 2019; Marzo-Ortega H, et al.		2003
		FRI0379. Annals of the Rheumatic Diseases 2019;78:873.	•	5 years: published Lancet Rheumatology in March 2020
	•	5 year results; Published in Lancet Rheumatology, June
		2020

66 Novartis Q3 Results | Oct 27, 2020 | Novartis Investor Presentation

Cosentyx® - Anti IL-17

Study	NCT02008916 MEASURE 3 (CAIN457F2314)	NCT02745080 EXCEED (CAIN457F2366)
Indication	Ankylosing spondylitis	Psoriatic arthritis
Phase	Phase 3	Phase 3
Patients	222	850
Primary Outcome	Assessment of Spondyloarthritis International Society criteria /	American College of Rheumatology 20 (ACR20) response
Measures	ASAS 20 response
	•	Secukinumab 10 mg/kg / 300 mg	•	Secukinumab 300 mg s.c.
Arms/Intervention	•	Secukinumab 10 mg/kg / 150 mg
•	Adalimumab 40 mg s.c.
	•	Placebo
Target Patients	Patients with active ankylosing spondylitis	Patients with active psoriatic arthritis
Read-out Milestone(s)	2018 (actual)	Q1-2020(actual)
	•	16 weeks results: PANLAR congress in Apr-2016
	•	52 weeks results: Pavelka et al. Arthritis Research &
		Therapy 2017
Publication	•	2 year results: Presented at ACR in Nov-2017	•	Published in the Lancet in May-2020
•	3 year (EOS) results: To be presented (ORAL) at PANLAR	•	Abstracts planned in Q4-2020
		April 2019
	•	3 year (EOS) manuscript published in ACR Open
		Rheumatology in January 2020

67 Novartis Q3 Results | Oct 27, 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
Measures	modified Stoke Ankylosing Spondylitis Spine Score (mSASSS)
Society criteria);
	•	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
Read-out Milestone(s)	Week 52: Q3-2019(actual); Final: H1-2021	2022
	•	Abstract (16 week results) presented at ACR 2019
Publication	•	Abstract (52 week results) presented at EULAR 2020	• Study design manuscript published. Baraliakos et al. Clinical
• Manuscript submitted in Mar-2020 (awaiting decision)		Drug Investigation (2020) 40:269-278.
	•	Abstract and Journal/Congress are planned in Q4-2020

68 Novartis Q3 Results | Oct 27, 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 clinical	Proportion of patients with Hidradenitis Suppurativa Clinical
Measures	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)
Read-out Milestone(s)	Primary (week 16): H2-2021; Final: 2022	2025
Publication	Study design SHSA 2020	Study design SHSA 2020

69 Novartis Q3 Results | Oct 27, 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

Read-out Milestone(s)

Publication

Subjects with moderate to severe Hidradenitis Suppurativa

Primary (week 16): H2-2021; Final: 2022

Study design SHSA 2020

70 Novartis Q3 Results | Oct 27, 2020 | Novartis Investor Presentation

Cosentyx® - Anti IL-17

Study	NCT04156620 INVIGORATE-1 (CAIN457P12301)	NCT04209205 INVIGORATE-2 (CAIN457P12302)
Indication	Axial spondyloarthritis	Psoriatic Arthritis (PsA)
Phase	Phase 3	Phase 3
Patients	500	380

Primary Outcome Measures

Arms/Intervention

Target Patients

Read-out Milestone(s)

Publication

The proportion of subjects achieving an ASAS40 (Assessment	The proportion of subjects achieving American College of
of SpondyloArthritis International Society criteria) response	Rheumatology 50 (ACR50) response criteria
•	Secukinumab intravenous (i.v.) regimen	•	Secukinumab intravenous (i.v.) regimen
•	Placebo intravenous (i.v.) regimen	•	Placebo intravenous (i.v.) regimen
Patients with active axial spondyloarthritis	Patients with active psoriatic arthritis (PsA) despite current or
previous NSAID, DMARD and/or anti-TNF therapy
Primary (week 16): 2022; Final: 2023	2022
TBD	TBD

71 Novartis Q3 Results | Oct 27, 2020 | Novartis Investor Presentation

Cosentyx® - Anti IL-17

Study	NCT04181762 SELUNE (CAIN457Q12301)	NCT04300296 PRELUDE (CAIN457S12201)
Indication	Lupus Nephritis	Lichen Planus
Phase	Phase 3	Phase 2
Patients	460	108

Primary Outcome Measures

Arms/Intervention

Proportion of patients achieving Investigator's Global

Proportion of subjects achieving protocol-defined CRRAssessment (IGA 0/1) score at 16 weeks +30% delta vs placebo

•	Secukinumab 300 mg s.c.	•	Secukinumab 300 mg s.c.
•	Placebo s.c.	•	Placebo s.c.

Target Patients	Patients with active lupus nephritis (ISN/RPS Class III or IV,	Adult patients with biopsy-proven lichen planus not adequately
with or without co-existing class V features)	controlled by topical therapies
Read-out Milestone(s)	2026	2022
Publication	TBD	TBD

72 Novartis Q3 Results | Oct 27, 2020 | Novartis Investor Presentation

Ilaris® - Anti IL-1β

Study	NCT02296424 (CACZ885G2306)	NCT04362813 CAN-COVID (CACZ885D2310)
Indication	SJIA - Systemic Juvenile Idiopathic Arthritis	COVID-19 induced respiratory disease
Phase	Phase 3B/4	Phase 3
Patients	182	450

	Proportion of patients in clinical remission on canakinumab
Primary Outcome	who are able to remain in remission following canakinumab
Measures	dose tapering (reduced canakinumab dose or prolonged
	canakinumab dosing interval)

Number of patients with clinical response; Clinical response is defined as survival without ever requiring invasive mechanical ventilation from day 3 to day 29

Arms/Intervention

Target Patients

Read-out Milestone(s)

Publication

•	Canakinumab dose reduction	•	Canakinumab
•	Canakinumab dose interval prolongation	•	Placebo
Patients with Systemic Juvenile Idiopathic Arthritis (SJIA)	Patients With COVID-19-induced pneumonia
(Pediatric)
2018 (actual)	Q4-2020
• Remission & flexible dosing - presented at ISSAID &

EULAR in Q2-2019	Planned manuscript submission in Q4-2020

73 Novartis Q3 Results | Oct 27, 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 therapy
(tropifexor + cenicriviroc) by monitoring adverse event profile,
Measures
vital signs and laboratory parameters
	•	Arm A: tropifexor (LJN452) dose 1
Arms/Intervention	•	Arm B: cenicriviroc (CVC)
•	Arm C: LJN452 dose 1 + CVC
	•	Arm D: LJN452 dose 2 + CVC

Target Patients

Read-out Milestone(s)

Publication

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

Q4-2020

Abstract planned in H1-2021

74 Novartis Q3 Results | Oct 27, 2020 | Novartis Investor Presentation

LJN452 - FXR Agonist

Study	NCT02855164 FLIGHT FXR (CLJN452A2202)	NCT04065841 ELIVATE (CLJN452D12201C)
Indication	Non-alcoholic steatohepatitis (NASH)	Non-alcoholic steatohepatitis (NASH)
Phase	Phase 2	Phase 2
Patients	351	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 compared
	quantitative MRI; determine effect of LJN452 on liver fibrosis	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	Adult patients with non-alcoholic steatohepatitis (NASH)	Adult patients with biopsy based non-alcoholic steatohepatitis
		(NASH) and liver fibrosis
Read-out Milestone(s)	Q2-2020(actual)	2022
	•	Final data accepted for oral presentation at AASLD in Nov-
Publication		2020​	Planned in H1-2023
	•	Manuscript planned in Q1-2021​

75 Novartis Q3 Results | Oct 27, 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

Arms/Intervention

Change from baseline in weekly Urticaria Activity Score (UAS7) at Week 4

• Long-term safety and tolerability

• 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	• Selected dose of LOU064 taken orally twice
•	Arm 3	High dose of LOU064 orally in the morning (once daily) and matching
a day (morning and evening) from day 1 to
	placebo in the evening from Day 1 to 85
	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
Read-out Milestone(s)	Q2-2021	2022
Publication	Planned in H2-2021	TBD

76 Novartis Q3 Results | Oct 27, 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
Target Patients	controlled with H1-antihistamines at approved or increased	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.
Read-out Milestone(s)	2017 (actual)	2019 (actual)
	•	Primary results: Presented at EAACI 2018, EADV 2018, and	•	Primary results: AAD 2019;
	•	Secondary results presented in 2019 at: AAD, EAACI,
		GUF 2018; Maurer M, et al. N Engl J Med 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,
	•	4 Manuscripts 2020/21: core results extension; angioedema;
		EAACI, EADV, ACAAI; Encoring all at GUF
			sleep/work impairment; data visualization

77 Novartis Q3 Results | Oct 27, 2020 | Novartis Investor Presentation

QGE031 - Anti-IgE

Study	NCT03437278 (CQGE031C2202)	NCT04210843 (CQGE031C2302E1)
Indication	Chronic spontaneous urticaria / Chronic idiopathic urticaria	Chronic spontaneous urticaria / Chronic idiopathic urticaria
Phase	Phase 2	Phase 3
Patients	48	800
Primary Outcome	Change in the 7 day Urticaria Activity Score (UAS7)	The proportion of subjects with well-controlled disease
Measures	(UAS7 ≤ 6) at week 12
	•	Ligelizumab high dose q4wks for 24 weeks	•	Ligelizumab Dose 1 and 3
Arms/Intervention	•	Ligelizumab low dose q4wks for 24 weeks
•	Ligelizumab Dose 2 and 3
	•	Placebo / ligelizumab high dose q4wks for 8 / 16 weeks
Target Patients	Adolescents from 12 to <18 years of age, with chronic	Patients who completed studies CQGE031C2302,
spontaneous urticaria	CQGE031C2303, CQGE031C2202 or CQGE031C1301
Read-out Milestone(s)	H2-2021	2026
	•	Study design was presented at PAAM (Peds Allergy &
		Asthma Meeting) and at UCARE meeting 2019
Publication	•	Baseline characteristics 2020/21	Study design presented at 2020 EAACI
• Primary results to be presented in late 2021/2022 (e.g.
		EAACI, PAAM, EADV)
	•	Manuscript to be submitted in 2022

78 Novartis Q3 Results | Oct 27, 2020 | Novartis Investor Presentation

QGE031 - Anti-IgE

Study	NCT03580369 Pearl 1 (CQGE031C2302)	NCT03580356 Pearl 2 (CQGE031C2303)
Indication	Chronic spontaneous urticarial / Chronic idiopathic urticaria	Chronic spontaneous urticarial / Chronic idiopathic 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 ligelizumab	• Placebo q4w from randomization to wk20, then ligelizumab
	dose B from wk24 to wk52	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
Read-out Milestone(s)	H2-2021	H2-2021
	• Study design presented at UCARE 2018
Publication	• Primary results to be presented in 2022 (e.g. EAACI, PAAM, EADV)
	• Manuscript to be submitted in 2022

79 Novartis Q3 Results | Oct 27, 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 syndrome	Alanine aminotransferase (ALT) normalization
Measures	(pSS)
Arms/Intervention	•	VAY736	•	VAY736
•	Placebo	•	Placebo control with conversion to active VAY736
Target Patients	Patients with moderate to severe primary Sjögren's syndrome	Autoimmune hepatitis patients with incomplete response or
(pSS)	intolerant to standard treatment of care
Read-out Milestone(s)	Q2-2020(actual)	2026
Publication	•	Manuscript to be submitted in Q3-2020	TBD

80 Novartis Q3 Results | Oct 27, 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
Read-out Milestone(s)	Double-blind: 2017 (actual);	Q1-2020(actual)
Extension (open-label):H1-2021
	•	PROs and prespecified subgroup analysis (Double-blind
		phase) submitted to JNNP accepted Aug-2020	• Primary analysis manuscript targeted end 2020
Publication	•	Submitted May 28, 2020 1 year Open-label extension to	•	Abstracts accepted for MTIS in 2020
	Neurology	• Secondary analysis to be submitted to multiple congresses
	•	Planned for Q4-2020: 2Y Open-label extension Abstracts		in 2021
		completed for EAN, AHS, EHF and MTIS in 2020

82 Novartis Q3 Results | Oct 27, 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 last
Measures	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
	Double-blind:2022;

Read-out Milestone(s)	Extension (open-label): 2024
Publication	Planned in H2-2022 for double-blind phase and H1-2025 for
open-label extension phase

83 Novartis Q3 Results | Oct 27, 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 relapse
Measures
rate up to 12 months
	• Fingolimod 0.5 mg orally
Arms/Intervention	•	Fingolimod 0.25mg orally
	•	Copaxone® 20 mg s.c.

Target Patients

Read-out Milestone(s)

Publication

Patients with relapsing-remitting multiple sclerosis

2018 (actual)

JAMA Neurology, Aug 2020 24

84 Novartis Q3 Results | Oct 27, 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

Arms/Intervention

Target Patients

Read-out Milestone(s)

Publication

Branaplam oral, once weekly:

• Part 1: 5 ascending doses
• Part 2: 2 different dose levels
• Part 3: patients continue on initial dose assigned in Part 1 or Part 2

Patients with type 1 spinal muscular atrophy

Study Part 2: Q3-2020(actual)

Study Part 3: 2023

TBD

85 Novartis Q3 Results | Oct 27, 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	The delay in time to confirmed disability progression as
Measures	measured by EDSS (Expanded Disability Status Scale)
	• BAF312 (5-day titration: 0.25mg to 1.25mg; Maintenance
Arms/Intervention	dose: 2mg (day 6))
	• Placebo
Target Patients	Patients with secondary progressive multiple sclerosis
Read-out Milestone(s)	Primary: 2016 (actual); Extension: 2024
Publication	Lancet 2018; 391:1263-73

86 Novartis Q3 Results | Oct 27, 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	927	955
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 of	relapses in a year calculated based on cumulative number of
Measures
relapses by patient adjusted for time-in-study by patient	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
Read-out Milestone(s)	Q3-2019(actual)	Q3-2019(actual)
Publication	Primary manuscript N Engl J Med 2020; 383:546-557, Aug-	Primary manuscript N Engl J Med 2020; 383:546-557, Aug-
2020	2020

87 Novartis Q3 Results | Oct 27, 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 Measures

Arms/Intervention

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 subjects
vs placebo)	with RMS from the first dose of ofatumumab
•	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
Read-out Milestone(s)	Q1-2020(actual)	2028
Publication	Publication planned for H1-2021	TBD

88 Novartis Q3 Results | Oct 27, 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	Proportion of participants sitting without support	•	Achievement of independent sitting for at least 30 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
Read-out Milestone(s)	Q4-2020	Q4-2019(actual)
Publication	(World Muscle Society) WMS 2020	(Muscular Dystrophy Association) MDA 2020

89 Novartis Q3 Results | Oct 27, 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	2
	• [2 copies of SMN2] Percentage of participants achieving
	functional independent sitting for at least 30 seconds at any
Primary Outcome	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
Read-out Milestone(s)	H2-2021	H2-2021
Publication	(Muscular Dystrophy Association) MDA 2020	TBD

90 Novartis Q3 Results | Oct 27, 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
Read-out Milestone(s)	Cohort B: Q4-2019(actual); Cohort C1: TBC
Publication	(Muscular Dystrophy Association) MDA 2020

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

91 Novartis Q3 Results | Oct 27, 2020 | Novartis Investor Presentation

Oncology

ABL001 - Specific, allosteric Bcr-Abl kinase inhibitor

Study	NCT03106779 ASCEMBL (CABL001A2301)
Indication	Chronic myeloid leukaemia (CML)
Phase	Phase 3
Patients	233
Primary Outcome
Major Molecular Response (MMR) rate at 24 weeks
Measures
Arms/Intervention	•	ABL001 40 mg bid
•	Bosutinib 500 mg
Target Patients	Patients with chronic myelogenous leukemia in chronic phase,
previously treated with 2 or more tyrosine kinase inhibitors
Read-out Milestone(s)	Q3-2020(actual)
	•	Manuscript submission Q4-2020
Publication	• Abstract submission to ASH (American Society of
		Hematology) Q4-2020

93 Novartis Q3 Results | Oct 27, 2020 | Novartis Investor Presentation

ACZ885 - IL-1β inhibitor

Study	NCT03447769 CANOPY-A (CACZ885T2301)	NCT03631199 CANOPY-1 (CACZ885U2301)
Indication	Adjuvant NSCLC	1st Line Non-small cell lung cancer (NSCLC)
Phase	Phase 3	Phase 3
Patients	1,500	627
		• Safety run-in part: Incidence of dose limiting toxicities
Primary Outcome	Disease free survival (primary), overall survival (key	•	Double-blind, randomized, placebo-controlled part:
Measures	secondary)		Progression free survival (PFS)
		•	Overall survival (OS)

Arms/Intervention

Target Patients

Read-out Milestone(s)

Publication

• Canakinumab 200mg q3w sc for 18 cycles	• Canakinumab or matching placebo in combination with
• Placebo q3w sc for 18 cycles		pembrolizumab and platinum-based doublet 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 prior
(T>5cm N2)) after complete resection and standard of care		systemic anticancer therapy
adjuvant cisplatin-based chemotherapy	•	Squamous and non-squamous NSCLC
• All histologies	•	No EGFR mutation and ALK rearrangement
Interim: 2022; Final: 2023	Interim: Q3-2020 (DMC) (actual); Final: 2021
	•	Johnson B et al. Presented at AACR-NCI-EORTC 2019
TBD		(safety run-in)
• Manuscript submission Q4-2020 (safety run-in)
	•	Abstract submission to congress H1-2021

94 Novartis Q3 Results | Oct 27, 2020 | Novartis Investor Presentation

ACZ885 - IL-1β inhibitor

Study	NCT03626545 CANOPY-2 (CACZ885V2301)
Indication	2nd / 3rd Line Non-small cell lung cancer (NSCLC)
Phase	Phase 3
Patients	240
Primary Outcome	• Safety run-in part: Incidence of dose limiting toxicities
• Double-blind, randomized, placebo-controlled part: Overall
Measures
	Survival
	• Canakinumab in combination with docetaxel
Arms/Intervention	•	Canakinumab matching-placebo in combination with
		docetaxel
	Patients with:
Target Patients	•	Stage IIIB or IV NSCLC without EGFR, ALK, ROS-1 or B-
	RAF mutation
	•	Previously treated with platinum therapy and PD(L)1-inhibitor
Read-out Milestone(s)	H1-2021
Publication	Abstract submission to congress H1-2021

95 Novartis Q3 Results | Oct 27, 2020 | Novartis Investor Presentation

BYL719 - Alpha-specific PI3K inhibitor

Study	NCT02437318 SOLAR-1 (CBYL719C2301)	NCT04251533 EPIK-B3 (CBYL719H12301)
Indication	HR+/HER2- advanced breast cancer with PIK3CA mutation	Triple negative breast cancer
Phase	Phase 3	Phase 3
Patients	572	566

Primary Outcome Measures

Arms/Intervention

Target Patients

Read-out Milestone(s)

Publication

Progression-free survival (PFS) for patients with PIK3CA	Progression-free Survival (PFS) for patients with PIK3CA
mutant status	mutant status
•	Fulvestrant 500 mg + alpelisib 300 mg	• Alpelisib 300 mg + nab-paclitaxel 100 mg/m²
•	Fulvestrant 500 mg + placebo	• Placebo + nab-paclitaxel 100 mg/m²
Men and postmenopausal women with hormone receptor	Patients with advanced triple negative breast cancer with
either Phosphoinositide-3-kinase Catalytic Subunit Alpha
positive, HER2-negative advanced breast cancer which
(PIK3CA) mutation or Phosphatase and Tensin Homolog
progressed on or after aromatase inhibitor treatment
Protein (PTEN) loss without PIK3CA mutation
2018 (actual)	2023

• Andre F, et al. Presentation at ESMO (European Society for Medical Oncology) 2018

•	Andre et al. Manuscript N Engl J Med 2019;380:1929-1940.	TBD
•	Andre F et al. Presentation at ESMO (European Society for
	Medical Oncology) 2020

96 Novartis Q3 Results | Oct 27, 2020 | Novartis Investor Presentation

BYL719 - Alpha-specific PI3K inhibitor

Study	NCT04208178 EPIK-B2 (CBYL719G12301)
Indication	HER-2 positive breast cancer
Phase	Phase 3
Patients	548
Primary Outcome	Progression-free survival (PFS)
Measures
Arms/Intervention	•	Alpelisib + trastuzumab + pertuzumab
•	Trastuzumab + pertuzumab
Target Patients	Patients with HER2-positive advanced breast cancer with a
PIK3CA mutation
Read-out Milestone(s)	2025
Publication	TBD

97 Novartis Q3 Results | Oct 27, 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
Read-out Milestone(s)	2018 (actual)
	•	Angelucci E, et al. Presentation at ASH (American Society
Publication		of Hematology) 2018
•	Angelucci E, et al. Manuscript Ann Intern Med
		2020;172:513-522.

98 Novartis Q3 Results | Oct 27, 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
Arms/Intervention		second or third line
•	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

Read-out Milestone(s)

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. Presentation at ASCO 2020 (cohort 1 and 5a)
• Groen H, et al. Presentation at ASCO 2020 (cohort 6)
• Wolf J, et al. Manuscript N Engl J Med 2020; 383:944-957

99 Novartis Q3 Results | Oct 27, 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
Read-out Milestone(s)	2019 (actual)	Final: Q3-2020(actual)
	•	Zeiser R, et al. Manuscript N Engl J Med 2020;382:1800-
Publication		1810.	•	Manuscript submission in H2-2020
• Zeiser R, et al. Oral presentation at EBMT 2020 Presidential	•	Abstract submitted to Congress in Q4-2020
		Plenary

100 Novartis Q3 Results | Oct 27, 2020 | Novartis Investor Presentation