Novartis AG

Investor Relations

Q2 2020 Results

Investor Presentation July 21, 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 our estimates of the impact of past and future COVID-19 related forward purchasing on sales and on our performance; or regarding the impact of the COVID-19 pandemic on clinical trials, and research and development timelines; 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 efforts to provide a not-for-profit portfolio of medicines for symptomatic 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 ophthalmology, in the second half 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 that commenced in prior years and is expected to continue this year; safety, quality, data integrity, or manufacturing issues; uncertainties involved in the development or adoption of potentially transformational technologies and business models; uncertainties regarding actual or potential legal proceedings, 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.

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

2 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

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

3 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

Company overview

4 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

Strong H1 performance despite the impact of COVID-19

H1 results more representative of performance as Q1 forward purchasing largely reversed in Q2

Strong operational performance

Delivering on innovation

Continuing operations1, % cc vs. PY

US approval NSCLC

Robust pandemic response

Human capital

Health and safety of associates, patients, third

Net sales

Q2 2020 H1 2020

6%

Core OpInc

19%

6%

US/EU approval nr-axSpA, China AS, CHMP PedPsO

EU conditional approval SMA IV

EU approval asthma

+ve CHMP opinion HR+/HER2- aBC

Label update in US

+ve CHMP opinion CRSwNP

parties2

Supply chain

Operations remain stable with customer service levels at a record high

Clinical trials

Disruptions managed with SENSE and Site Cockpit digital technologies

Collaborations & drug discovery

Ph3 clinical trials with canakinumab and ruxolitinib3, 35+ investigator-initiated trials

COVID-19 Access portfolio

(15 medicines offered to 79 LICs, LMICs)4

-1%

5 simultaneous approvals in

Japan

For footnotes see slide 49

5 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

Growth drivers continuing strong momentum

Key growth driver sales H1 2020

Sales

Growth vs. PY

Growth vs. PY

USD Million

USD Million

cc

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

1,149

212

159

371

50%

375

170

190

360

nm

1,874

139

86

225

15%

825

96

73

169

28%

169

90

79

169

nm

153

74

73

147

nm

320

70

48

118

64%

211

48

60

108

106%

102

68

34

102

nm

737

69

31

100

19%

413

62

35

97

33%

628

60

26

86

20%

nm - not meaningful

Q1 20

Q2 20

47%

37%

30%

24%

H1 2017

H1 2018

H1 2019

H1 2020

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

Adakveo®

Mayzent®

Beovu®

Piqray®

Xiidra®

Lutathera®

Kymriah®

Kisqali®

Zolgensma®

Ilaris®

Jakavi®

Tafinlar+Mekinist®

Promacta®

Entresto®

Cosentyx®

Other1

6 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

Zolgensma® (USD 205m): Growth driven by geographic expansion

Q2 highlights

USD 205m Strong global momentum despite COVID-19

60% newborns screened by end Q2, Medicaid access 86% of lives covered, permanent J-code in place Some COVID-related disruption normalizing

Regulatory and other milestones

AVXS-101 IT

Continued dialogue with FDA

partial clinical

Planning pre-BLA meeting

Submission expected 2021

hold

Received conditional approval with broad label1 Day One access program

Germany: agreements with 90% of sick funds Early access in other countries

Geographical expansions

Approvals expected H2 2020 / early 2021: Switzerland (Fast Track), Canada (Priority Review), Israel, Australia, Argentina, South Korea, Brazil

Fast uptake in 1st month of launch

Others

Requests continue in pre-approved markets

(e.g. Middle East, Russia)

Manufacturing

Continued progress with Colorado

expansion

and North Carolina sites expected

operational 2021

1. Patients with SMA and a clinical diagnosis of Type 1 or SMA patients with up to three copies of the SMN2 gene. The approval covers babies and young children with SMA up to 21 kg according to the approved dosing guidance

7 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

Sandoz H1 results highlight continuing good performance, despite negative Q2 impact of COVID-19

Net sales

Core OpInc

% cc vs. PY

H1 results more representative of performance as Q1

Q2 2020 H1 2020

1%

26%

1%

forward purchasing largely reversed in Q2

H1 performance drivers

  • 25% sales growth Biopharmaceuticals
  • +3% Retail ex-US
  • Rapid Aspen Japan integration
  • Continuous gross margin and core ROS improvement

Other dynamics

  • COVID-19negatively impacted hospital / pharmacy traffic lowering demand
  • US oral solids and dermatology decline and partnership terminations

-9%

8 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

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

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

Novel assets

Ofatumumab

MBG453

Major launches

Inclisiran

Asciminib

TQJ230

Canakinumab

In-market

LNP023

Spartalizumab

Iscalimab

177Lu-PSMA-617

growth drivers

Ligelizumab

LNA043

Tropifexor

UNR844

CEE321

LOU064

VAY736

LMI070

QBW251

SELECT EXAMPLES

New indications

Cosentyx® HS

Alpelisib PROS

Cosentyx® GCA

Piqray® TNBC

Cosentyx® LP

Piqray® HER2+ aBC

Cosentyx® JIA

Piqray® ovarian cancer

Cosentyx® LN

Piqray® HNSCC

Entresto® post-AMI

Kisqali® HR+/HER2- BC (adj)

Entresto® HFpEF

Kymriah® FL

Beovu® DME

Kymriah® DLBCL

Beovu® RVO

Jakavi® cGVHD

Beovu® DR

Jakavi® aGVHD

Beovu® PDR

Ofatumumab pediatric

AVXS-101 IT

Xolair® food allergy

1. Ph3 / in registration

9 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

Advancing our late stage pipeline

Selected assets

Asset

Indication

Current status

Next milestone

2020

Ofatumumab (OMB157)

RMS

In registration

FDA action date September 2020

Inclisiran (KJX839)

Hyperlipidemia

US / EU submissions complete; review on track

FDA action date December 2020

Alpelisib (BYL719)

PROS

Real World Evidence (RWE) Ph2 ongoing

US submission expected H2 2020

Tafinlar®+Mekinist®

mBRAF V600 +

Ph3 readout on track 2020

Submission on track by end of 2020

w/ spartalizumab

melanoma

Asciminib (ABL001)

CML

Pivotal study in 3L

On track for readout 2020; first submission expected Q1 2021

Canakinumab (ACZ885)

NSCLC

Enrollment complete in 1L (CANOPY-1) and

DMC IA data readout (CANOPY-1) expected Q4 2020;

2L (CANOPY-2) studies

CANOPY-1 and CANOPY-2 readouts & filings expected 2021

177Lu-PSMA-617

mCRPC

VISION Ph3 trial: slower than expected event accumulation

Event driven trial; readout expected H1 2021

rate; preparations ongoing for starting earlier line studies

2021

Entresto®

HFpEF, post-AMI

HFpEF filed

FDA action expected by H1 2021

PARADISE-MI enrollment complete

PARADISE-MI study results expected 2021

AVXS-101 IT

SMA IT

Partial clinical hold: continued dialogue with FDA

Planning pre-BLA meeting; submission expected 2021

Ligelizumab (QGE031)

CSU

PEARL 1 and 2, superiority studies vs. Xolair® (Ph3)

Ph3 enrollment on track to complete 2020;

ongoing

readout and submission expected H2 2021

Kisqali®

Adjuvant BC

NATALEE study on track, enrollment ongoing

MONALEESA-2 OS readout expected 2021

NATALEE Ph3 aBC readout expected 2022

10 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

Emerging pipeline assets continue to progress

Selected assets

Pharmaceuticals

Asset

MoA

Indication

Current status

Next milestone

LNP023

Factor B

PNH, IgAN, C3G,

Ph2 parallel development for 3 rare renal diseases and

Single PNH pivotal trial expected to start 2020;

inhibitor

iMN, aHUS

paroxysmal nocturnal haemoglobinuria (PNH)

C3 glomerulopathy, IgA nephropathy

Ph3 expected to start Q1 2021

Remibrutinib

Bruton tyrosine kinase

CSU, Sjögren's

Ph2b study in CSU and adaptive Ph2 in Sjögren's ongoing

Phase 2b study in CSU expected to readout

(LOU064)

inhibitor

2021

Iscalimab

Anti-CD40 monoclonal

Kidney Tx, Sjögren's

Ph2b studies in kidney transplant, Sjögren's ongoing

Anticipated regulatory submission for

(CFZ533)

antibody

kidney transplant 2023

TQJ230

Antisense

CVRR-Lp(a)

Ph3 outcomes study (HORIZON) initiated 2020

Ph3 outcomes readout expected 2024

oligonucleotide

targeting LP(a)

Oncology

MBG453

Anti-TIM-3 monoclonal

HR-MDS,

Ph3 study in HR-MDS initiated Jun 2020 (STIMULUS-

Ph2 in unfit AML (combo HMA + venetoclax)

antibody

unfit AML

MDS-2)

expected to start 2020

LXH254

B/C-RAF inhibitor

m RAS/RAF NSCLC

Clinical studies ongoing, evaluating LXH254 in combination

Ph2 metastatic melanoma trial

and melanoma

with LTT462 (ERKi), Mekinist®, Kisqali® and spartalizumab

expected to start 2020

TNO155

SHP2 inhibitor

Solid tumors

Broad combination strategy with multiple Ph1 combo

Continued enrollment in all 3 trials,

studies ongoing including spartalizumab, Kisqali®,

including recently started trial with

nazartinib, MRTX849

MRTX8491

1. Study sponsored by Mirati Therapeutics

11 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

Progressing on our journey of building trust with society

Ethical standards

Pricing & access

Global health

Corporate citizenship

Established robust governance

Tone from the top (zero tolerance)

Ethics Risk Compliance (ERC) Officer member of Executive Committee

Link ethics to performance management (including sales force and executives)

Continued use of data analytics to generate compliance insights

>200 country monitoring visits annually, 500 ERC associates globally

Continuously evolving

Trust & Reputation Committee chaired by CEO

Launching Code of Ethics, elevating principles-based policy

Enterprise risk management approach, third party risk assessments, human rights

Evolving peer-to-peer medical education towards digital (incl. new CIA)

Resolved long-standing legacy legal matters

Speaker programs settlement (2002-2011)

Independent charitable co-pay foundations settlement (2010-2014)

Sandoz resolves US generic drug antitrust criminal investigation (2013-2015)

FCPA investigations now closed (2007-2015)

12 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

Financial review and 2020 guidance

13 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

Strong H1 performance despite impact of COVID-19

Continuing operations1

Q2

Change vs. PY

USD million

2020

% USD

% cc2

Net Sales

11,347

-4

-1

Core Operating income 2

3,669

1

6

Operating income

2,352

-12

-4

Net Income

1,867

-11

-4

Core EPS (USD)2

1.36

1

6

EPS (USD)

0.82

-10

-3

Free Cash Flow 2

3,631

1

H1

Change vs. PY

2020

% USD

% cc2

23,630

3

6

7,846

14

19

5,096

4

11

4,040

2

9

2.92

15

19

1.77

3

11

5,652 3

  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. Core results, constant currencies and free cash flow are non-IFRS measures. Further details regarding non-IFRS measures can be found starting on page 54 of the Condensed Financial Report.

14 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

Continuing operations delivering core margin expansion of 3.8%pts cc vs. PY

Continuing operations1

Q2 2020

H1 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

5

35.9

1.3

7

16

36.5

2.8

Sandoz

-9

1

22.0

2.2

1

26

24.5

4.9

Continuing Operations

-1

6

32.3

2.1

6

19

33.2

3.8

  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. Core results, constant currencies and free cash flow are non-IFRS measures. Further details regarding non-IFRS measures can be found starting on page 54 of the Condensed Financial Report.

15 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

Many therapy areas impacted by COVID-19 early in Q2, seeing rebound in June

Ophthalmology, dermatology and new patient starts are more impacted

IM Weekly Sales Evolution

4 weeks rolling, indexed to Q4 weekly sales avg.

Beginning of COVID-19 lockdowns

Impact of Beovu

across US and Europe

125%

launch pre-ASRS

Recent Launches1

communication

Growth Drivers

(excl. Cosentyx®)2

100%

Beovu®, Lucentis® & Xiidra®

Cosentyx®

Q2 Cosentyx® (US data)

75%

Recovered in June to Q4 2019 levels

Increasing market share in dermatology and

rheumatology

50%

Mature Ophthalmology3

Feb 29

Mar 31

Apr 30

May 31

Jun 28

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® and Zolgensma® 2. Growth drivers

include Entresto®, Ilaris®, Jakavi®, Promacta®, Tafinlar®+Mekinist® and Xolair®

3. Includes Luxturna®

16 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

Core OpInc growth in H2 expected to be impacted by increased generic erosion

Key drivers of core operating income (continuing operations)

vs. PY (cc)

ILLUSTRATIVE

H1 2020

  • Continued strong momentum in Innovative Medicines growth drivers and launches1 uptake
  • Productivity and lower spend driven by COVID-19 related lockdowns
  • Gx erosion2
  • COVID-19related negative impact

on Lucentis® and mature ophtha

H2 2020

  • Innovative Medicines growth drivers, and launches1
  • Productivity
  • Increased Gx erosion2
  • Increased investments in pre- launch activities and launches
  • Lapping Xiidra® acquisition

1. Including Zolgensma®, Mayzent®, Aimovig®, Xiidra®, Piqray®

2. Including Afinitor ®, Exjade®, Sandostatin® LAR and Ophtha brands

17 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

2020 FY guidance1 tightened within prior guidance ranges

Continuing operations | barring unforeseen events; growth vs. PY in cc

Sales expected to grow mid single digit

  • IM Division expected to grow mid single digit
  • Sandoz expected to grow low single digit

Core operating income expected to grow 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 H2 2020. In addition, we assume that no Gilenya and no Sandostatin LAR generics enter in 2020 in the US

18 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

H1 2020 free cash flow increased to USD 5.7bn

Continuing operations1 free cash flow2

USD billion

+3%

Key drivers vs. PY:

5.7

5.5

+

Higher operating income

(adjusted for non-cash items)

−

Lower divestment proceeds

H1 2019

H1 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. Free cash flow is a non-IFRS measure. An explanation of non-IFRS measures can be found on page 54 of the Condensed Interim Financial Report

19 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

Expected currency impact for full year 2020

Currency impact vs. PY

%pts, assuming mid-July exchange rates prevail in 2020

FX impact on Net sales

FX impact on Core operating income

0

-2

0 to -1

-1 to -2

-3

-3

-3

-3

-4

-5

-5

-6

FY

Q1

Q2

Q3

Q4

FY

FY

Q1

Q2

Q3

Q4

FY

2019

2020

2019

2020

Actual Simulation

20 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

21 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

H1 performance solid

Accelerating our digital transformation

Pharmaceuticals net sales

USD billion, growth in % cc

H1 sales grew +8% as growth drivers continued momentum

Entresto® sales +50%, Cosentyx® sales +15%

+8%

11.7

11.2

0.8

0.1

3.34.0

7.86.9

H1 2019

H1 2020

  • Zolgensma® sales USD 375m, Xiidra® sales USD 169m

Q2: COVID-19 had negative sales impact

  • Lucentis® down -24% due to market decline
  • Mature ophthalmology4 products down -32%5
  • Cosentyx® up +12% despite COVID-19 impact

Accelerating digital transformation

  • Pivoted to hybrid F2F / virtual promotion and patient support for in-market brands and launches
  • Leading virtual scientific and medical engagement at congresses

Growth drivers1 Recent launches2 Mature products3

1. Cosentyx®, Entresto®, Ilaris®, Xolair® 2. Zolgensma®, Xiidra®, Aimovig®, Luxturna®, Mayzent® and Beovu® 3. All other brands 4. Includes Luxturna® 5. Includes generic impact primarily for Travatan®

22 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

Cosentyx® grew faster than PsO and SpA market in US despite COVID-19 impact

Sales evolution

USD million, % cc

Ex-US

US+12%

Market growth declined due to COVID-19

  • Visits declined 50%1 in EU and 80-90%2 in US at peak

Cosentyx® US growth above market3,4 due to strong safety, efficacy and broad access

858

944

Sales now showing recovery towards pre-COVID dynamics

PsO QoQ: TRx +5% vs. market 0%, NBRx -24% vs. market -30%

324

330

SpA QoQ: TRx +9% vs. market +2%, NBRx -16% vs. market -21%

Further approvals

534614

Q2 2019

Q2 2020

  • Launching nr-axSpA in US / EU (1.7m potential patients)
  • AS approved in China
  • PedPsO positive CHMP opinion

TRx = Total Prescriptions; NBRx = New-to-Brand Prescriptions; nr-axSpA = non-radiographic Axial Spondyloarthritis; PedPsO = Pediatric Psoriasis; CHMP = Committee for Human Medicinal Products. 1. IQVIA COVID-19 Tracker, EU5 Countries - Wave 1, 19th June 2020. 2. Spherix Global Insights, "Multi-Specialty Impact of COVID-19". 3. IQVIA National Prescription Audit for Dermatology WE 06/26/2020; market includes Enbrel®, Humira®, Siliq®, Skyrizi™, Stelara®, Taltz®, Tremfya®. 4. IQVIA National Prescription Audit for Rheumatology WE 06/26/2020; SpA market includes Cimzia®, Enbrel®, Humira®, Simponi®, Stelara®, Taltz®

23 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

Cosentyx® poised to maintain strong position in growing dermatology market, set to accelerate in rheumatology

>340k patients reached, >5 years efficacy and safety data, strong 1st-line access

Strong dermatology position

USD 17bn market WW, double-digit growth long-term

15% biologic penetration1,2

8/10 patients achieve clear or almost clear skin7

Dedicated studies in scalp, nails, palmoplantar8

Poised to remain biologic of choice for 2/3 of patients with multiple manifestations6

Ready to accelerate in rheumatology

USD 12bn market WW, double-digit growth long-term

14% axSpA, 23% PsA biologic penetration3,4

Strong joint efficacy in AS9 and PsA10 with growing guideline11 support

PREVENT reinforces substantial benefits across axSpA spectrum

Additional evidence generation in PsA12

Potential to expand into multiple indications

Dermatology: PedPsO, HS, Lichen Planus, totaling >3m patients5

Rheumatology: jPsA/ ERA, GCA, Lupus Nephritis, totaling >500k patients5

Potential label updates: 300mg AI/PFS, flexible PsO dosing, IV for SpA

For footnotes see slide 49

24 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

Strong Entresto® performance driven by underlying demand

Sales evolution

USD million, % cc

Ex-US

US

+40%

580

421272

200

221308

Q2 2019

Q2 2020

Maintains strong growth despite COVID-19 market slowdown

  • Q2 sales of USD 580m, strong demand across geographies
  • US weekly NBRx rebounded to >3,800 in June1

Strong foundation for future growth

  • 25% of 3.4m eligible HFrEF population received Entresto® in G72
  • FDA accepted HFpEF file
  • PARADISE post-AMI on track for readout mid-2021
  • Geographical expansion with Japan approval; launch expected H2 2020

HFrEF - Heart failure with reduced ejection fraction HFpEF - Heart failure with preserved ejection fraction 1. US NBRx - IMS New to Brand w/e 06/19/20; 2. IQVIA NPA - TRx March '20

25 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

Beovu® US label updated

New data reinforces need for fluid control in wAMD

Characterization complete, root-cause analysis ongoing

SRC completed post hoc review of reported post-marketing events and concluded similar events were present in H&H. SRC noted similar overall rates of vision loss between H&H treatment arms.

Launched coalition with 25 experts to evaluate root-causes, risk factors, mitigation and treatment options

Regular updates on brolucizumab.info

Progressing label updates,

New post-hoc data confirms lower levels of

continuing launches

retinal fluid are associated with better BCVA1

Updated labels in US, JP, CH,

Adjusted mean BCVA change Wk12-96 (ETDRS letters)

AUS3, adding clarifying safety

language to warnings &

precautions4

Beovu® now approved in 30

countries, launches ongoing

Beovu® benefit-riskprofileremainspositiveBeovu® better in reducingretinalfluid(IRF/SRF)

DA = Disease Activity; H&H = Hawk & Harrier; SRC = Safety Review Committee; IRF = Intraretinal Fluid; PED = Pigment Epithelial Detachment; SRF = Subretinal fluid 1. Source: Schmidt-Erfurth et al. A comparison of the therapeutic

response between brolucizumab and aflibercept in the HAWK & HARRIER trials using deep learning-based OCT analysis. ARVO Annual Meeting, May 2020 2: IRF, SRF, PED 3. Ongoing in all other countries where Beovu is approved 4. Retinal vasculitis and/or retinal vascular occlusion that may result in severe vision loss

26 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

Ofatumumab has the potential to become a 1st choice for a broad range of RMS patients and physicians

Potential for broad and early high efficacy in RMS

Powerful sustained efficacy

Favorable safety

Precise and targeted B-cell therapy

Flexibility through at home self-administration

Based on strong ASCLEPIOS I & II data

Superior efficacy for relapses, MRI activity Substantial reductions in disability progression1

9/10 patients had no evidence of disease activity in year 22 No significant signals of infections/ malignancies

Launch leverages established position and deep customer insights

✓ Rapid and broad availability of

✓ Highly customized approach

✓ Flexible and customer-centric

Ofatumumab upon approval

for early adopters

onboarding process

RMS = Relapsing Multiple Sclerosis; 1. CDW, confirmed disability worsening and CDP, confirmed disability progression are interchangeable terms, defined by an increase ≥1.5 EDSS points for patients with baseline EDSS of 0, increase of ≥1.0 EDSS points patients with baseline EDSS of 1.0-5.0 and increase of t ≥0.5 EDSS points for patients with baseline EDSS of 5.5. 2. Hauser S.L. et al. Ofatumumab vs Teriflunomide in Relapsing Multiple Sclerosis: Analysis of No Evidence of Disease Activity (NEDA-3) from ASCLEPIOS I and II Trials. Poster presented at EAN, 23-26 May 2020 LB62

27 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

Inclisiran launch preparations are progressing - FDA action date Dec 2020

Advancing launch preparations

FDA action date Dec 2020

Review ongoing

MAA submitted to Swissmedic in May

Clinical trial application accepted in June

NHS partnership on track

Addressing non-clinical barriers to uptake

Access

Allowing rapid patient onboarding at

point of care

Affordability

Ensuring lowest possible co-pay for

majority of patients

Adherence

Enabled by twice yearly HCP

administration

System costs

Aligning to ASCVD objectives of

systems of care

Source : US Truven Data, Safford M, et al. Am J Prev Med. 2015; 48(5): 520-527, National Center for Chronic Disease Prevention and Health Promotion , Division for Heart Disease and Stroke Prevention, American Heart Association CVD

Burden Report (accessed 06/20), Wong ND, et al. J Clin Lipidol. 2016;10(5):1109-1118; 1. Adapted from: Ference BA et al. J Am Coll Cardiol. 2018; 72(10); 1141-1156-

28 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

29 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

Recent launches and growth drivers more than offset generic impact in H1 2020

Oncology net sales

USD billion, % cc

+6%

7.2

6.9

.4

0.5

0.9

0.4

1.8

2.2

4.6

4.1

1.1

H1 sales up +6% cc, despite significant Gx erosion

Strong uptake of recent launches in Q2

  • Kisqali® (USD 159m, +49% cc), Kymriah® (USD 118m, +103% cc)
  • Piqray® (USD 79m), Adakveo® (USD 21m), Tabrecta™
  • Lutathera® (USD 105m, -3% cc) impacted by COVID-19

Growth drivers continued double-digit performance in Q2

  • Promacta® / Revolade® (USD 422m, +23% cc), Tafinlar® + Mekinist® (USD 371m, +12% cc), Jakavi® (USD 310m, +14% cc)

H1 2019

H1 2020

Recent launches1

Growth drivers2

Base business3

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

3. Base business - other brands.

30 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

Kisqali®: Strong performance despite CDK4/6 market slowdown in the US

Net sales

H1 sales up +64% cc, reflecting continued share gains as the only

USD m, % cc

CDK4/6 inhibitor with two positive OS readouts; third OS readout

+64%

(MONALEESA-2) expected 2021

320

  • Kisqali® has a differentiated profile vs. other CDK4/6 inhibitors, with preferential inhibition to CDK4 vs. CDK6, and a high concentration to inhibit the target

+49%202

159

111

  • US overall CDK4/6 class growth has been slowing down due to delays in new patient starts (NBRx down ~10% in Q2 vs. PY)1
  • Rolled out in-home monitoring to support patients and HCPs during the pandemic

NATALEE adjuvant study on track to complete enrollment of 4k

patients in 2020

Q2 2019

Q2 2020

H1 2019

H1 2020

1. IQVIA Raw NBRx data, March 16, 2020 - May, 2020.

31 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

Kymriah®: Sales more than doubled in H1, despite COVID impact on healthcare systems

Net sales

USD m, % cc

+106%

211

+103%

118 103

  • H1 sales up +106% cc, with strong growth in US and EU
  • No interruption of supply during COVID-19
  • Over 25 countries covering at least one indication and more than 240 qualified treatment centers
  • Continued to expand our global manufacturing network: Stein and Les Ulis sites approved for commercial supply

58

  • FDA granted RMAT1 designation to Kymriah® for r/r follicular lymphoma; submission expected 2021

Q2 2019

Q2 2020

H1 2019

H1 2020

1. Regenerative Medicine Advanced Therapy

32 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

Tabrecta™ (capmatinib): US launch off to an encouraging start; gearing up for launch in Japan

High unmet medical need and strong clinical profile

3-4% of NSCLC patients have METex14 mutations, associated with poor prognosis and modest benefit from existing therapies

~53k addressable population worldwide

ORR in 1L of ~70% and 2L of ~40% by BIRC and 7 intracranial responses observed in post hoc analysis (n=13)

Omni-channel launch amid pandemic conditions

Launched in US simultaneously with FDA- approved METex14 CDx test

Strong early market response and positive customer feedback:

  • >20k visitors on patient website and >9k visitors on HCP website within 1 month
  • >1m views for first 3 nights of Tabrecta™ Livestream Week on Twitter
  • >30 leading lung cancer institutions have started patients on treatment

Received Japan approval on June 29

Development plan to maximize potential

Monotherapy studies: Phase 3, brain metastases, tumor agnostic

Moving into combinations:

  • PD-L1high expressers regardless of MET status, in combination with pembrolizumab
  • METex14 skipping regardless of PD-L1 status, in combination with spartalizumab
  • MET amplified Post-EGFR, in combination with osimertinib

Opportunity to serve an additional 40k patients

Approved in US for treatment of metastatic NSCLC with exon 14 skipping mutation ORR = Overall Response Rate, BIRC = Blinded Independent Review Committee, METex14 = MET exon 14 skipping (METex14, PD-L1 = Programmed death-ligand 1, EGFR = epidermal growth factor receptor, CDx = companion diagnostic

33 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

34 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

2020 catalysts: Maintaining long-term momentum

Potential catalysts

Major approvals1

Major submissions2

Major readouts3 (Phase 3)

Phase 3 starts

Selected examples

Ofatumumab (OMB157)

Tabrecta™(capmatinib)5

Inclisiran (KJX839)

Relapsing MS

NSCLC

Hyperlipidemia (US)

Enerzair® / Atectura®

Cosentyx®

Asthma

nr-AxSpA

Inclisiran (KJX839)

Alpelisib (BYL719)

Hyperlipidemia (EU)

PROS

Entresto®

Spartalizumab (PDR001) combo

HFpEF (US)

Metastatic melanoma

Jakavi®

Beovu®

Canakinumab

Chronic GvHD

DME

1st line NSCLC (IA)

Asciminib (ABL001)

Entresto®

Chronic Myeloid Leukemia

Post-acute MI (IA)

TQJ2306

MBG453

CVRR

MDS

LNP023

Alpelisib (BYL719)

PNH

Multiple indications4

1. First approval in any market. 2. First submission in any market 3. Readouts enabling submission, label change or pivotal trial initiation 4. HER2+ aBC, TNBC, ovarian cancer, head and neck cancer 5. Received FDA Priority Review designation 6. Received FDA Fast Track designation

35

Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

Conclusion

  • Strong H1 performance, confirming FY 2020 guidance
  • Growth drivers on track to sustain performance
  • Pipeline delivering, excited about mid to late stage assets

36 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

Appendix

Net debt increased by USD 10.6bn mainly due to The Medicines Company acquisition

(USD bn)

-10.6

-15.9

-7.0

-0.1

-26.5

0.7

-9.9

5.7

Dec 31, 2019

Dividends

M&A transactions1

Free Cash Flow

Treasury share

Others

Jun 30, 2020

transactions, net

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

38 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

2020 expected pipeline milestones

H1 2020

H2 2020

✓ Achieved

✕ Missed

Regulatory decisions and opinions

Major expected submissions

Major expected trial readouts*

Beovu®

nAMD (EU/JP)

✓

Adakveo®

Sickle cell disease (EU)

Cosentyx®

nr-axSpA (EU/US)

✓

Tabrecta™ (capmatinib)

NSCLC (US/JP)

✓

Cosentyx®

AS (CN)

✓

Cosentyx®

Pediatric psoriasis (EU)

✓3

Ofatumumab (OMB157)

Relapsing MS (US)

H2 20202

Cosentyx®

nr-axSpA (JP)

Piqray®

HR+/HER2- aBC with PIK3CA

✓3

Entresto®

HFpEF (US)

H1 2021

mutation (EU)

Enerzair®

Asthma (EU/JP)

✓4

Inclisiran (KJX839)

Hyperlipidemia (US)

Tafinlar® & Mekinist®

Adjuvant melanoma (CN)

✓

Xolair®

Nasal Polyposis (US/EU)

✓3

Xiidra®

DED (EU)

✕

Zolgensma® IV

SMA (EU/JP)

✓

Entresto®

HFpEF (US)

✓

Alpelisib (BYL719)

PROS (US)

Inclisiran (KJX839)

Hyperlipidemia (EU)

✓

AVXS-101 IT

SMA (US)

2021

Cosentyx®

Juvenile PsA / enthesitis-related

arthritis (US/EU)

Spartalizumab (PDR001)

Metastatic melanoma (US/EU)

and Tafinlar® & Mekinist®

177Lu-PSMA-617

mCRPC (US)

2021

Entresto®

Post-acute MI1

✓

Asciminib (ABL001)

CML 3L

Tropifexor (LJN452)

NASH

✓

Beovu®

DME

UNR844

Presbyopia

✓

Jakavi®

chronic GVHD

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. FDA extended review with regulatory action now expected September 2020 3. Positive CHMP opinion received 4. EU approval July 2020, Japan approval June 2020

39

Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

Beovu: outcomes of the safety review committee analysis of HAWK & HARRIER data

Spectrum of inflammatory events

4%

IOI

4.4% IOI rate in H&H trials (reported as 4% in US PI)

4.6% IOI rate assessed by SRC in the post hoc unmasked analysis

3%

Vasculitis

3.3% Retinal vasculitis rate as assessed by SRC in the post-hoc unmasked

analysis of cases of interest (only 1 case reported by investigators in H&H)

2%

Occlusion

2.1% Retinal vascular occlusion rate as assessed by SRC in the post-hoc

unmasked analysis (RAO reported at 0.8% in H&H trials)

<1%

Vision loss due

<1% probability of losing 15 letters or more due to IOI or retinal vasculitis

to these AEs

as assessed by the SRC in their post-hoc unmasked analysis

~0

Difference in

Overall vision loss similar across Beovu (7.4%)

overall vision loss

and Eylea (7.7%) arms at w96, as assessed in H&H and noted by SRC

IOI = Intraoccular Inflammation; PI = Prescribing Information; RAO = Retinal Artery Occlusion; AE = Adverse Event

40 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

Our pipeline projects at a glance

Phase 1/2

Phase 3

Registration

Total

ONCOLOGY

51

22

2

75

PHARMACEUT ICALS

62

21

7

90

Cardiovascular, Renal, Metabolism

12

4

2

18

Immunology, Hepatology, Dermatology

26

6

2

34

Neuroscience

5

4

1

10

Ophthalmology

5

3

0

8

Respiratory

8

3

1

12

Global Health

6

1

1

8

Total

113

43

9

165

Biosimilars not included as we only disclose biosimilars that have moved into Phase 3

CRM: Cardiovascular, Renal & Metabolism. IHD: Immunology, Hepatology & Dermatology. NS: NeuroScience.

41 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

Novartis submission schedule

New Medical Entities: Lead and supplementary indications

2020

2021

2022

2023

≥2024

spartalizumab

Lead

177Lu-PSMA-617

Lead

LAG525

Lead

177Lu-NeoB

Lead

LNA043

Lead

SAF312

Lead

CSJ117

Lead

PDR001

177Lu-PSMA-617

Solid Tumors

177Lu-NeoB

Osteoarthritis

COSP

Asthma

m BRAF V600+ melanoma (+Taf/Mek)

mCRPC 3L

Multiple Solid Tumors

asciminib

Lead

remibrutinib

Lead

177Lu-PSMA-R2

Lead

tropifexor

Lead

AVXS-201

Lead

TQJ230

Lead

ABL001

LOU064

177Lu-PSMA-R2

LJN452

OAV201

CVRR-Lp(a)

CML 3L

Chronic spontaneous urticaria

Prostate cancer

NASH

Rett syndrome

LEAD INDICATIONS

MBG453

Lead

iscalimab

HR-MDS

CFZ533

Renal Tx

ECF843

ligelizumab

Lead

QGE031

Dry eye

Chronic urticaria

LNP023

PNH

Lead

VPM087

Lead

tropifexor&cenicriviroc

Lead

LMI070

Lead

ganaplacide

Lead

1st line CRC / 1st line RCC

LJC242

SMA

KAF156

NASH

Malaria uncomplicated

Lead

CEE321

Lead

CPK850

Lead

MIJ821

Lead

cipargamin

Lead

Atopic Dermatitis

RP

Depression

KAE609

Malaria severe

Lead

ianalumab

Lead

UNR844

Lead

QBW251

Lead

LXE408

Lead

VAY736

Presbyopia

COPD

Visceral leishmaniasis

AIH

NEW INDICATIONS

canakinumab

LCM

canakinumab

LCM

capmatinib

LCM

spartalizumab

LCM

remibrutinib

LCM

inclisiran

LCM

ACZ885

ACZ885

INC280

PDR001

LOU064

KJX839

NSCLC 2L

Adjuvant NSCLC

Solid tumors

Malignant melanoma (combo)

SjS

CVRR-LDLC

canakinumab

LCM

LNP023

LCM

crizanlizumab

LCM

iscalimab

Lead

tropifexor

LCM

LNP023

LCM

ACZ885

C3G

SEG101

CFZ533

LJN452

iMN

NSCLC 1L

Sickle cell anaemia with crisis ped

Liver Tx

NASH (combos)

LNP023

LCM

MBG453

LCM

iscalimab

LCM

ofatumumab

LCM

cipargamin

LCM

IgAN

Maintenance for MRD+ AML

CFZ533

OMB157

KAE609

SjS

Ped MS

Malaria uncomplicated

LNP023

LCM

MBG453

LCM

ianalumab

LCM

aHUS

Unfit AML

VAY736

pSjS

42 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

Novartis submission schedule

Supplementary indications for existing brands

2020

2021

2022

2023

≥2024

alpelisib, BYL719

LCM

Kymriah

LCM

Kisqali

LCM

Lutathera

LCM

Jakavi

LCM

Kymriah

LCM

Cosentyx

LCM

Aimovig

LCM

PROS

tisagenlecleucel-T, CTL019

ribociclib, LEE011

177Lu-oxodotreotideb)

ruxolitinib, INC424

tisagenlecleucel-T, CL019

secukinumab, AIN457

erenumab, AMG334

r/r DLBCL 1st relapse

HR+/HER2- BC (adj)

GEP-NET 1L G3

Pediatrics Chronic GVHD

r/r DLBCL (+ pembro)

GCA

Pediatric Migraine

Kymriah

LCM

Promacta

LCM

tisagenlecleucel-T, CTL019

eltrombopag, ETB115

r/r Follicular lymphoma

Radiation sickness syndrome

Tafinlar

LCM

Adakveo

LCM

dabrafenib, DRB436

crizanlizumab, SEG101

HGG/LGG - Pediatrics

Sickle cell anaemia new formulations

Promacta

LCM

Cosentyx

LCM

eltrombopag, ETB115

secukinumab, AIN457

Food effect free formulation

SpA IVIV

Jakavi

LCM

Cosentyx

LCM

ruxolitinib, INC424

secukinumab, AIN457

Chronic GVHD

Hidradenitis suppurativa

Jakavi

LCM

Cosentyx

LCM

ruxolitinib, INC424

secukinumab, AIN457

Acute GVHD

AS H2H

Beovu

LCM

Xolair

LCM

brolucizumab, RTH258

omalizumab, IGE025

DME

Food allergy

AVXS-101

LCM

Entresto EUa

LCM

onasemno-geneabepar-vovec, OAV101

sacubitril/valsartan, LCZ696

SMA IT

Pediatric HF

Xolair

LCM

omalizumab, IGE025

Auto-injector

Entresto

LCM

sacubitril/valsartan, LCZ696

Post-AMI

Piqray

LCM

Jakavi

LCM

Kymriah

LCM

Cosentyx

LCM

Mayzent

LCM

alpelisib, BYL719

ruxolitinib, INC424

tisagenlecleucel-T, CTL019

secukinumab, AIN457

siponimod, BAF312

TNBC

Pediatrics Acute GVHD

1L high risk ALL, pediatrics & young adults

Lichen Planus

Pediatric MS

Piqray

LCM

Jakavi

LCM

Piqray

LCM

Cosentyx

LCM

alpelisib, BYL719

ruxolitinib, INC424

alpelisib, BYL719

secukinumab, AIN457

HER2+ adv BC

Myelofibrosis (combination)

HNSCC 2/3L

Lupus Nephritis

PiqrayLCM

alpelisib, BYL719 Ovarian cancer

TafinlarLCM

dabrafenib, DRB436 Tyroid cancer

BeovuLCM

brolucizumab, RTH258 Diabetic retinopathy

BeovuLCM

brolucizumab, RTH258

RVO

CoartemLCM

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

denosumabBioS

GP2411

anti RANKL mAb

a. Approved in US. b.177Lu-dotatate in US.

43 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

Novartis pipeline in registration

Lead indication

3 lead indications

Oncology

Respiratory Disease

Code

Name

Mechanism

Indication(s)

Code

Name

Mechanism

Indication(s)

BYL719

Piqray

PI3Kα inhibitor

PIK3CA mutant HR+, HER2 (-) postmenopausal adv BC 2nd line

IGE025

Xolair

IgE Inhibitor

Nasal polyps

(+fulv)

SEG101

Adakveo®

P-selectin Inhibitor

Sickle cell disease

Immunology, Hepatology, Dermatology

Cardiovascular, Renal, Metabolism

Code

Name

Mechanism

Indication(s)

Code

Name

Mechanism

Indication(s)

AIN457

Cosentyx

IL17A Inhibitor

Ped Psoriasis

2ml Auto-injector

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

LAM320

Lamprene®

SMPD1 Inhibitor

Tuberculosisa)

a) WHO Pre-Qualification

44 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

Novartis pipeline in Phase 3

6 lead indications

Lead indication

Oncology

Code

Name

Mechanism

Indication(s)

177Lu-PSMA-617

177Lu-PSMA-617

Targeted Radioligand Therapy

mCRPC

177Lu-

Lutathera®

Targeted Radioligand Therapy

GEP-NET 1L G3

oxodotreotide 3)

ABL001

asciminib

BCR-ABL Inhibitor

CML 3L

ACZ885

canakinumab

IL-1b Inhibitor

NSCLC 1L

NSCLC 2L

Adjuvant

NSCLC

BYL719

Piqray®

PI3Kα inhibitor

HER2+ adv BC

TNBC

HNSCC 2/3L

Ovarian cancer

CTL019

Kymriah

CD19 CART

r/r Follicular

1L high risk

r/r DLBCL 1st

lymphoma

ALL, pediatrics

relapse

and young

adults

ETB115

Promacta®

Thrombopoietin receptor (TPO-R)

Radiation sickness syndrome

Food effect free formulation

Agonist

INC424

Jakavi

JAK1/JAK2 Inhibitor

Acute GVHD

Chronic GVHD

LEE011

Kisqali®

CDK4 Inhibitor

HR+/HER2- BC (adj)

MBG453

MBG453

TIM3 Antagonist

HR-MDS

PDR001

Spartalizumab

PD1 Inhibitor

m BRAF V600+ melanoma (+Taf/Mek)

Solid tumors

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

SpA IVIV

suppurativa

ACZ885

canakinumab

IL-1b Inhibitor

COVID-19 induced respiratory disease

QGE031

ligelizumab

IgE Inhibitor

Chronic urticaria

Ophthalmology

Code

Name

Mechanism

Indication(s)

RTH258

Beovu®

VEGF Inhibitor

Diabetic retinopathy

RVO

DME

  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
  2. Approved in US
  3. 177Lu-dotatate in US

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 IT 1)

neuron (SMN1) gene

OMB157

ofatumumab

CD20 Antagonist

Ped MS

Respiratory Disease

Code

Name

Mechanism

Indication(s)

IGE025

omalizumab

IgE Inhibitor

Food allergy

Auto-injector

INC424

Jakavi®

JAK1 Inhibitor

COVID-19 related pneumonia

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 HF 2)

(ARNI)

TQJ230

TQJ230

Anti-Apo(a) ASO targeting Lp(a)

CVRR-Lp(a)

Global Health

Code

Name

Mechanism

Indication(s)

COA566

Coartem®

-

Malaria uncomplicated, <5kg patients

Biosimilars

Code

Name

Mechanism

Indication(s)

GP2411

denosumab

anti RANKL mAb

Denosumab BioS

45 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

Novartis pipeline in Phase 2

30 lead indications

Lead indication

Oncology

Neuroscience

Code

Name

Mechanism

Indication(s)

BYL719

alpelisib

PI3Kα inhibitor

PROS

BLZ945

BLZ945

-

Solid tumors

CTL019

Kymriah

CD19 CART

r/r DLBCL (+ pembro)

EGF816

nazartinib+capmatinib Opdivo

EGFR Inhibitor

NSCLC (combo)

INC280

capmatinib

Met Inhibitor

NSCLC EU1)

Solid tumors

NSCLC

Met Inhibitor + spartalizumab

HCC

INC424

Jakavi®

JAK1/JAK2 Inhibitor

Myelofibrosis (combination)

LAG525

LAG525

LAG3 Inhibitor

Solid Tumors

MBG453

MBG453

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)

BAF312

Mayzent®

S1P1 Modulator

Stroke

BLZ945

BLZ945

CSF-1 Inhibitor

ALS

LMI070

branaplam

Survival motor neuron protein

SMA

MIJ821

MIJ821

NR2B Inhibitor

Depression

Respiratory Disease

Code

Name

Mechanism

Indication(s)

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

Code

Name

Mechanism

Indication(s)

ADTP02

ADTP02

-

NASH (Combos)

AIN457

Cosentyx®

IL17A Inhibitor

GCA

Lichen Planus

CFZ533

iscalimab

CD40 Inhibitor

Renal/Liver Tx

SjS

HS

LJC242

tropifexor&cenicriviroc

CCR2 Inhibitor, FXR agonist

NASH (combos)

LJN452

tropifexor

FXR agonist

NASH

NASH (combos)

LNA043

LNA043

ANGPTL3 Agonist

Osteoarthritis

LOU064

remibrutinib

BTK Inhibitor

CSU

SjS

LYS006

LYS006

Anti-inflammatory

Acne

Colitis ulcerative

HS

VAY736

ianalumab

BAFF-R Inhibitor

pSjS

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

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

LNP023

CFB Inhibitor

PNH

IgAN

C3G

iMN

aHUS

LTW980

LTW980

-

Hypertriglyceridemia

Global Health

Code

Name

Mechanism

Indication(s)

AFQ056

AFQ056

mGluR5 Antagonist

Addiction

KAE609

cipargamin

PfATP4 inhibitor

Malaria severe

Malaria uncomplicated

KAF156

ganaplacide

-

Malaria uncomplicated

LXE408

LXE408

Protozoan Inhibitor

Visceral leishmaniasis

46 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation1) Approved in US & JP

Novartis pipeline in Phase 1 (1 of 2)

37 lead indications

Lead indication

Oncology

Code

Name

Mechanism

Indication(s)

177Lu-NeoB

177Lu-NeoB

Radioligand therapy target GRPR

Multiple solid tumors

177Lu-PSMA-R2

177Lu-PSMA-R2

Radioligand therapy target PSMA

Prostate cancer

ADPT01

NIR178, LAG525, spartalizumab, canakinumab, capmatinib

LAG3 Inhibitor,PD1 Inhibitor

TNBC

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/JAK2 Inhibitor

Myelofibrosis (combination)

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

MBG453

MBG453 (combos)

TIM3 Antagonist

Cancers

MCM998

MCM998, LXG250

BCMA CART, CD19 CART

Multiple myeloma

MIK665

MIK665

MCL1 Inhibitor

Haematological malignancy

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)

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

VPM087

IL1B Antagonist

1st line CRC / 1st line RCC

WNT974

WNT974 + spartalizumab

Porcupine Inhibitor

Solid tumors

WVT078

WVT078

-

Multiple myeloma

YTB323

YTB323 ± ibrutinib

CD19 CART

Haematological malignancy

47 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

Novartis pipeline in Phase 1 (2 of 2)

Lead indication

37 lead indications

Immunology, Hepatology, Dermatology

Global Health

Code

Name

Mechanism

Indication(s)

Code

Name

Mechanism

Indication(s)

CEE321

CEE321

Pan JAK Inhibitor

AD

KAF156

ganaplacide

-

Malaria prophylaxis

DFV890

DFV890

-

Anti-inflammatory therapy

FIA586

FIA586

-

NASH

LRX712

LRX712

-

Osteoarthritis

MAS825

MAS825

-

Inflammatory diseases

MHS552

MHS552

-

Autoimmune Indications

MHV370

MHV370

-

SjS

SLE

Neuroscience

Code

Name

Mechanism

Indication(s)

OAV201

AVXS-201

MECP2 gene therapy

Rett syndrome

Respiratory Disease

Code

Name

Mechanism

Indication(s)

CMK389

CMK389

IL-18 Inhibitor

Sarcoidosis

LTP001

LTP001

-

Respiratory Diseases

Cardiovascular, Renal, Metabolism

Code

Name

Mechanism

Indication(s)

HSY244

HSY244

-

Atrial fibrillation

MBL949

MBL949

-

Diabetes

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

48 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

References

Slide 5

nr-axSpA

Non-radiographic axial spondyloarthritis

PedPsO

Pediatric psoriasis

aBC

Advanced breast cancer

CRSwNP

Severe chronic rhinosinusitis with nasal polyps

LIC / LMICs

Low income / lower middle-income countries

  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. COVID-19Good Practice Guidance distributed to suppliers
  3. Readout expected H2 2020. Discontinued hydroxychloroquine clinical trial for COVID-19 due to acute enrollment challenges but continue to supply the drug for ongoing investigator-initiated trials and upon government requests
  4. This is addition to previously announced USD 40m COVID-19 response funds to support public / community health initiatives, healthcare infrastructures and various industry collaborations

Slide 24

RCT

Randomized Controlled Clinical Trial

PsO

Psoriasis

PedPsO

Pediatric Psoriasis

HS

Hidradenitis Suppurativa

PsA

Psoriatic Arthritis

AS

Ankylosing Spondylitis

nr-axSpA

non-radiographic Aaxial Spondyloarthritis

jPsA & ERA

Juvenile arthritis / enthesitisrelated arthritis

GCA

GCA = Giant Cell Arteritis

WW

WW = Worldwide

1

Based on 'WW IQVIA total brand sales' and 'Indication level brand data for G6 PSO (2019)';

2

Bx treated : DRG + IQVIA patient equivalents (2019);

3

Evaluate Pharma, SpA Market - Bx & Orals (2019);

4

PsA and axial SpA: Epidemiology, diagnosed, treated and Bx pool and aligned with DRG, latest CPO inputs

(internal assumption based multiple data sources) (2019)

5

Referring to US+EU5 countries

6

Corrona LLC, data on file. Corrona Report: Real-World Data from the Corrona Psoriasis Registry®. June

15, 2018. Study names

7

CLEAR, CLARITY

8

SCALP, TRANSFIGURE, GESTURE

9

MEASURE

10

EXCEED, FUTURE

11

Gossec L, Baraliakos X, Kerschbaumer A, et al. EULAR recommendations for the management of psoriatic

arthritis with pharmacological therapies: 2019 update Annals of the Rheumatic Diseases 2020;79:700-712

12

MAXIMISE, ULTIMATE, SERENA

49 Novartis Q2 Results | July 21, 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

granules (12.5, 31.25 mg in capsules); liquid formulation

adult formulation (50, 100, 200 mg bid))

(1mg/ml and 4mg/ml concentration) and adult

formulation (50, 100, 200 mg bid)

Pediatric patients from 1 month to < 18 years of age with

Pediatric patients with heart failure due to systemic left

Target Patients

heart failure due to systemic left ventricle systolic

ventricle systolic dysfunction who have completed study

dysfunction

CLCZ696B2319

H2-2021; (Analysis of 110 pts from Part 2 formed the basis

for pediatric submission in Apr-2019 and approval by the US

Expected Completion

FDA in Oct-2019 for the treatment of symptomatic HF with

2022

systemic left ventricular systolic dysfunction in children aged

1 year and older)

Publication

TBD

TBD

52 Novartis Q2 Results | July 21, 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 cardiovascular (CV) death or heart failure (HF)

Measures

Composite Score (GCCS)

hospitalization

• Sacubitril/valsartan 50, 100, and 200 mg bid with

• Sacubitril/valsartan 50 mg, 100 mg, 200 mg bid/placebo

Arms/Intervention

placebo of valsartan

of enalapril

• Valsartan 40, 80, and 160 mg bid tablets with placebo

• Enalapril 2.5 mg, 5 mg, 10 mg bid / placebo of

for sacubitril/valsartan

sacubitril/valsartan

Target Patients

Patients with chronic heart failure with preserved ejection

Japanese heart failure patients (NYHA Class II-IV) with

fraction

reduced ejection fraction

Expected Completion

2022

Q1-2019 (actual); H1-2021 (open-label extension)

Publication

TBD

Planned in Q3-2020: Primary manuscript in Circ J

53 Novartis Q2 Results | July 21, 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

Measures

hospitalizations

baseline to Week 24

• Sacubitril/valsartan 50 mg, 100 mg and 200 mg bid and

• Sacubitril/valsartan or placebo 50 mg, 100 mg, and 200

matching placebo

• Enalapril 2.5 mg, 5 mg and 10 mg bid and matching

Arms/Intervention

mg bid

placebo

• Valsartan or placebo 40 mg, 80 mg, and 160 mg bid

• Valsartan 40 mg, 80 mg, 160 mg bid and matching

placebo

Target Patients

Heart failure patients (NYHA Class II-IV) with preserved

Heart failure patients (NYHA Class II-IV) with preserved

ejection fraction

ejection fraction

Expected Completion

2019 (actual)

2019 (actual)

• Sep-2019: Primary manuscript (ARNI in HFpEF.

Solomon S et al; NEJM. DOI: 10.1056/NEJMoa1908655)

• Mar-2020: Published (NTproBNP, putative placebo

• May-2020 - Published: Study design (Wachter et al;

analysis);

ESC-HF)

Publication

• Jun-2020: Submitted (renal outcomes, cognitive

• Aug-2020 - Planned: Primary data presentation at ESC

function);

latebreaker; Publication EHJ Q3-2020.

• Q3/Q4-2020 Planned: Urgent HF visits, regional

• Q3-2020 - Planned: Baseline data publication

differences, win ratio, adjudicated vs reported endpts;

Subgroups (mode of death, MRA, age, gender).

54 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

Entresto® - Angiotensin II Receptor Neprilysin Inhibitor (ARNI)

Study

NCT03909295 (CLCZ696D1301E1 - extension study)

NCT02924727 PARADISE-MI (CLCZ696G2301)

Indication

Heart failure chronic

Post-acute myocardial infarction

Phase

Phase 3

Phase 3

Patients

52

5,670

Primary Outcome

Number of participants with Adverse Events (AEs) and

Time to the first occurrence of a confirmed composite

endpoint (cardiovascular (CV) death, heart failure (HF)

Measures

Serious Adverse Events (SAEs)

hospitalization, or outpatient heart failure)

• Sacubitril/valsartan 50 mg, 100 mg, 200 mg bid / placebo

Arms/Intervention

• Sacubitril/valsartan 50 mg,100 mg,200 mg film coated

of ramipril/valsartan

tablets

• Ramipril 1.25 mg, 2.5 mg, and 5 mg bid / placebo of

sacubitril/valsartan / placebo for valsartan

Japanese heart failure patients (NYHA Class II-IV) with

Post-AMI patients with evidence of LV systolic dysfunction

Target Patients

preserved ejection fraction after CLCZ696D2301

and/or pulmonary congestion, with no known prior history of

(PARAGON-HF)

chronic HF

Expected Completion

Q4-2019(actual)

H1-2021

Publication

TBD

• Q3-2020 - Planned: PARADISE-MI study design;

• Q4-2020 - Planned; PARADISE-MI baseline chars

55 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

KJX839 - siRNA (regulation of LDL-C)

Study

NCT03060577 ORION-3 (CKJX839A12201E1)

NCT03814187 ORION-4 (CKJX839A1KJX839B12301)

Hypercholesterolemia inc. Atherosclerotic Cardiovascular

Hypercholesterolemia inc. Heterozygous Familial

Indication

Disease (ASCVD) and ASCVD risk equivalents

Hypercholesterolaemia (HeFH)

Heterozygous Familial Hypercholesterolaemia (HeFH)

Phase

Phase 2

Phase 3

Patients

~374: 284 in Group 1 and 90 in Group 2

~15,000

A composite of major adverse cardiovascular events,

LDL-C reduction at Day 210 for Group 1 subjects

defined as:

Primary Outcome

Changes in other lipids and lipoproteins and reduction of

• Coronary heart disease (CHD) death;

Measures

LDL-C of more than 50% for patients that are above LDL-C

• Myocardial infarction;

goal ; longer term exposure and safety.

• Fatal or non-fatal ischaemic stroke; or

• Urgent coronary revascularization procedure

Arms/Intervention

Target Patients

Expected Completion

Publication

• Group 1 - inclisiran 300mg sc every 6 months until day

Arm 1: every 6 month treatment KJX839 300mg (given by

720 and then on Day 810, followed by every 6 months for a

subcutaneous injection on the day of randomization, at 3

planned duration of 4 years

months and then every 6-months) for a planned median

• Group 2- Evolocumab 140mg s.c. injection every 2

duration of about 5 years

weeks for 360 days, followed by inclisiran 300mg on Day

Arm 2: matching placebo (given bysubcutaneous injection

360, Day 450 and then every 6 months for a planned

on the day of randomization, at 3 months and then every 6-

duration of 4 years.

months) for a planned median duration of about 5 years.

Patients with HeFH or pre-existing atherosclerotic

Patient population with mean baseline LDL-C ≥ 100mg/dL;

long- 5 year- follow-up time is designed to show best in-

cardiovascular disease (ASCVD) on background statin +/-

class CV outcomes (25% benefit).

ezetimibe therapy

2022

2025

TBD

TBD

56 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

KJX839 - siRNA (regulation of LDL-C)

Study

NCT03851705 ORION-5 (CKJX839A12304)

NCT03399370 ORION-8 (CKJX839A12305B)

Hypercholesterolemia inc. Homozygous Familial

Hypercholesterolemia inc. Heterozygous Familial

Indication

Hypercholesterolaemia (HeFH) and Homozygous Familial

Hypercholesterolemia (HoFH)

Hypercholesterolemia (HoFH)

Phase

Phase 3

Phase 3

Patients

56 randomized 2:1 (inclisiran: placebo)

2967 entered the study

Primary Outcome Measures

Arms/Intervention

Target Patients

Expected Completion

Publication

LDL-C reduction at Day 150

The effect of inclisiran treatment on the proportion of

subjects achieving prespecified low density lipoprotein

Changes in PCSK9, other lipids and lipoproteins and

cholesterol(LDL-C)targets at end of study. The safety and

reduction of LDL-C of more than 20%

tolerability profile of long term use of inclisiran

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

on Day 1 and Day 90

Inclisiran 300mg on day 1 (placebo patients in feeder study)

• Part 2: inclisiran on Day 180 for patients who were

or placebo on Day 1 (inclisiran patients in feeder study )

randomized to the placebo group only, inclisiran on Day

then inclisiran 300mg on Day 90 and every 6 months for a

270 and then every 6 months for a planned duration of 2

planned duation of 3 years

years for all patients

Patients with HeFH or pre-existing atherosclerotic

Patients with HoFH

cardiovascular disease (ASCVD) on background statin +/-

ezetimibe therapy and risk equivalents (patients from

ORION 9, 10 & 11 studies)

Primary: Q3-2020; Final: H2-2021

2023

TBD

TBD

57 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

LNP023 - Factor B inhibition of the complement alternative pathway

Study

NCT03373461 (CLNP023X2203)

NCT04154787 (CLNP023D12201)

Indication

IgA nephropathy (IgAN)

Idiopathic membranous nephropathy (iMN)

Phase

Phase 2

Phase 2

Patients

146

72

Primary Outcome

Change from baseline of log transformed UPCR derived 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

Expected Completion

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

H2-2021

2022

TBD

TBD

58 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

LNP023 - Factor B inhibition of the complement alternative pathway

Study

NCT03832114 (CLNP023X2202)

NCT03955445 (CLNP023B12001B)

Indication

C3 glomerulopathy (C3G)

C3 glomerulopathy (C3G)

Phase

Phase 2

Phase 2 (open-label extension)

Patients

27

27 (from ongoing Phase 2, potential patient from Ph3)

Primary Outcome Measures

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

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

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

Arms/Intervention

Target Patients

Expected Completion

Publication

Increasing doses of LNP023 up to 200mg bid:

• Cohort A: Native kidney patients

• Open-label LNP023 200mg bid

• Cohort B: Kidney transplanted patients

Patients with C3 glomerulopathy

Patients with C3 glomerulopathy

H1-2021

2025

TBD

TBD

59 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

LNP023 - Factor B inhibition of the complement alternative pathway

Study

NCT03439839 (CLNP023X2201)

NCT03896152 (CLNP023X2204)

Indication

Paroxysmal nocturnal hemoglobinuria (PNH)

Paroxysmal nocturnal hemoglobinuria (PNH)

Phase

Phase 2

Phase 2

Patients

15

10

Primary Outcome Measures

Arms/Intervention

Reduction of chronic hemolysis, based on LDH level at

Reduction of PNH associated hemolysis, based on

percentage of patients with 60% reduction in LDH or LDH

Week 13

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

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

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

• Arm 1: 4wks treatment LNP023 25mg bid followed by

period

8wk treatment LNP023 100mg bid and 2yr extension

• Cohort 2: 5 patients receiving LNP023 50mg bid, in

LNP023 100mg bid

addition to SoC, for minimum 2 weeks with 3yr treatment

• Arm 2: 4wks treatment LNP023 50mg bid followed by

extension period. Dose may be increased D15 onwards to

8wk treatment LNP023 200mg bid and 2yr extension

200mg bid if LDH not within limit of normal or reduced by at

LNP023 200mg bid

least 60% compared to Baseline.

Patients with PNH, showing signs of active hemolysis

Target Patientsdespite treatment with SoC (defined as an antibody with anti C5 activity).

Patients with PNH, showing signs of active hemolysis, not treated with any other complement inhibitor less than 3 months prior to study start Day 1

Expected Completion

Primary endpoint: Q2-2020

Primary endpoint: Q2-2020

Extension period: 2023

Extension period: 2022

Publication

In preparation (PoC study)

TBD

60 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

TQJ230 - Antisense oligonucleotide targeting apolipoprotein(a) mRNA

Study

NCT04023552 Lp(a)HORIZON (CTQJ230A12301)

Indication

Cardiovascular risk reduction

Phase

Phase 3

Patients

7,680

Primary Outcome

Time to the first occurrence of MACE (cardiovascular death,

non-fatal MI, non-fatal stroke and urgent coronary re-

Measures

vascularization)

Arms/Intervention

TQJ230 80 mg injected monthly subcutaneously or

matched placebo

Target Patients

Patients with a history of Myocardial infarction or Ischemic

Stroke, or a clinically significant symptomatic Peripheral

Artery Disease, and Lp(a) ≥ 70 mg/dL

Expected Completion

2024

Publication

TBD

61 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

Immunology, Hepatology & Dermatology

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

Study

NCT03663335 CIRRUS I (CCFZ533A2201)

NCT03905525 TWINSS (CCFZ533B2201)

Indication

Kidney transplantation

Sjögren's syndrome

Phase

Phase 2B

Phase 2B

Patients

676

260

Primary Outcome Measures

Arms/Intervention

Composite event (BPAR, Graft Loss or Death) over 12

Change in EULAR Sjögren's syndrome Disease Activity

months post-transplantation and post conversion (for

Index (ESSDAI) score and EULAR Sjögren's syndrome

maintenance cohort)

Patient Reported Index (ESSPRI) score

• Two cohorts: de novo TX and maintenance

• Three dose arms of CFZ533

• Test Arms: CFZ533 + MMF + corticosteroids

• Placebo

• Standard of Care: TAC + MMF + corticosteroids

Target Patients

Kidney transplant recipients

Patients with Sjögren's syndrome

Expected Completion

2022

2023

Publication

Manuscript of PoC trial to be submitted in Q1-2020

Manuscript of PoC trial published in The Lancet-

Rheumatology January 23, 2020

63 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

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

Study

NCT03781414 CONTRAIL I (CCFZ533A2202)

Indication

Liver transplantation

Phase

Phase 2

Patients

128

Primary Outcome

Proportion of patients with composite event (BPAR, Graft

Measures

Loss or Death) over 12 months

• Control/Standard of Care: TAC + MMF + Corticosteroids

Arms/Intervention

•

CFZ533 dose A + MMF + Corticosteroids

•

CFZ533 dose B + MMF + Corticosteroids

Target Patients

Expected Completion

Publication

Liver transplant recipients

2023

TBD

64 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

Cosentyx® - Anti IL-17

Study

NCT03504852 (CAIN457A2324)

NCT03589885 MATURE (CAIN457A2325)

Indication

Psoriasis

Psoriasis

Phase

Phase 3B

Phase 3

Patients

331

122

Primary Outcome

PASI 90 response and IGA mod 2011 0 or 1 response after

Measures

16 weeks of treatment

PASI 75 response and IGA mod 2011 0 or 1 response after 12 weeks of treatment

•

Secukinumab 300 mg every 2 weeks after weekly doses

• Secukinumab 2 mL (300 mg) auto-injector

Arms/Intervention

till Week 4

• Secukinumab 2 x 1 mL (150 mg each) prefilled syringe

•

Secukinumab 300 mg every 4 weeks after weekly doses

•

Placebo 2 mL auto-injector

till Week 4

•

Placebo 2 x 1 mL prefilled syringe

Target Patients

Expected Completion

Publication

Subjects (≥90kg) with moderate to severe plaque psoriasis

Subjects with moderate to severe plaque psoriasis

Q3-2020

Q4-2020

TBD

TBD

65 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

Cosentyx® - Anti IL-17

Study

NCT02471144 (CAIN457A2310)

NCT03668613 (CAIN457A2311)

Indication

Psoriasis

Psoriasis

Phase

Phase 3

Phase 3

Patients

162

84

Primary Outcome

Psoriasis Area and Severity Index (PASI) 75 response and

Psoriasis Area and Severity Index (PASI) 75 response and

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

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

Measures

week 12

week 12

•

Secukinumab low dose

Arms/Intervention

•

Secukinumab high dose

•

Secukinumab low dose

•

Placebo

•

Secukinumab high dose

•

Etanercept (comparator)

Target Patients

Patients from 6 to less than 18 years of age with severe

Pediatric patients of age 6 to <18 years, with moderate to

chronic plaque psoriasis

severe plaque psoriasis

Expected Completion

2023

2023

Publication

TBD

TBD

66 Novartis Q2 Results | July 21, 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

Expected Completion

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 under assessment

67 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

Cosentyx® - Anti IL-17

Study

NCT03031782 (CAIN457F2304)

NCT03769168 (CAIN457F2304E1 - extension study)

Indication

Psoriatic arthritis

Psoriatic arthritis

Phase

Phase 3

Phase 3

Patients

80

64

Primary Outcome

Time to 33 flares

Number of participants with JIA ACR30 response

Measures

Arms/Intervention

• Secukinumab (pre-filled syringe) 75 mg

• Secukinumab 75 mg/0.5 ml

• Placebo

• Secukinumab 150 mg/1.0 ml

Target Patients

Juvenile idiopathic arthritis subtypes of psoriatic and

Patients with juvenile idiopathic arthritis subtypes of juvenile

enthesitis-related arthritis

psoriatic arthritis and enthesitis related arthritis

Expected Completion

H1-2021

2025

Publication

TBD

TBD

68 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

Cosentyx® - Anti IL-17

Study

NCT01892436 FUTURE 1 extension (CAIN457F2306E1)

NCT01649375 MEASURE 2 (CAIN457F2310)

Indication

Psoriatic arthritis

Ankylosing spondylitis

Phase

Phase 3

Phase 3

Patients

460

219

Primary Outcome

Proportion of subjects that have a positive clinical response

Assessment of SpondyloArthritis International Society /

to treatment (individual improvement) in disease activity

Measures

ASAS 20 response

according to ACR20 (or ACR50 or ACR 70)

•

Secukinumab 75 mg

•

Secukinumab 75 mg

Arms/Intervention

•

Secukinumab 150 mg

•

Secukinumab 150 mg

•

Placebo

Target Patients

Patients with active psoriatic arthritis

Patients with active ankylosing spondylitis

Expected Completion

2018 (actual)

2018 (actual)

• Primary 52 week results: Baeten D & Sieper J, et al. N

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

Engl J Med 2015;373:2534-48

• 2 year results: Marzo-Ortega, et al. Arthritis Care Res

Rheumatol. 2016; 68 (suppl 10)

2017 Feb 24. doi: - 10.1002/acr.23233

• 3 years results: Manuscript published in September

• 3 year results: Marzo-Ortega, et al. RMD 2017

Publication

2018 (Mease PJ, et al. RMD Open 2018;4:e000723.

• 5 year results: EULAR 2019; Marzo-Ortega H, et al.

doi:10.1136/rmdopen-2018-000723)

FRI0379. Annals of the Rheumatic Diseases

• 5 year results: Published in ACR Open Rheumatology.

2019;78:873.

November 14, 2019

• 5 year results; Published in Lancet Rheumatology, June

2020

69 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

Cosentyx® - Anti IL-17

Study

NCT01752634 FUTURE 2 (CAIN457F2312)

NCT02008916 MEASURE 3 (CAIN457F2314)

Indication

Psoriatic arthritis

Ankylosing spondylitis

Phase

Phase 3

Phase 3

Patients

399

222

Primary Outcome

Proportion of subjects achieving American College of

Assessment of Spondyloarthritis International Society

Measures

Rheumatology 20 (ACR20) response criteria

criteria / ASAS 20 response

• Secukinumab (AIN457) 150 mg s.c.

• Secukinumab 10 mg/kg / 300 mg

• Secukinumab (AIN457) 75 mg s.c.

Arms/Intervention

• Secukinumab 10 mg/kg / 150 mg

• Secukinumab (AIN457) 300 mg s.c.

• Placebo

• Placebo s.c.

Target Patients

Patients with active psoriatic arthritis

Patients with active ankylosing spondylitis

Expected Completion

2019 (actual)

2018 (actual)

• 16 weeks results: PANLAR congress in Apr-2016

• Primary results: McInnes IB, et al. Lancet.

• 52 weeks results: Pavelka et al. Arthritis Research &

Therapy 2017

2015;386:1137-46

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

Publication

• 2 years results: McInnes et al, Rheumatology

• 3 year (EOS) results: To be presented (ORAL) at

2017;56:1993-2003

PANLAR April 2019

• 5 years: published Lancet Rheumatology in March 2020

• 3 year (EOS) manuscript published in ACR Open

Rheumatology in January 2020

70 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

Cosentyx® - Anti IL-17

Study

NCT02745080 EXCEED (CAIN457F2366)

Indication

Psoriatic arthritis

Phase

Phase 3

Patients

850

Primary Outcome

American College of Rheumatology 20 (ACR20) response

Measures

Arms/Intervention

• Secukinumab 300 mg s.c.

• Adalimumab 40 mg s.c.

Target Patients

Patients with active psoriatic arthritis

Expected Completion

Q1-2020

Publication

Published in the Lancet in May-2020

71 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

Cosentyx® - Anti IL-17

Study

NCT02696031 PREVENT (CAIN457H2315)

NCT03259074 SURPASS (CAIN457K2340)

Indication

Non-radiographic axial spondyloarthritis

Ankylosing spondylitis

Phase

Phase 3

Phase 3

Patients

555

837

Primary Outcome

The proportion of participants who achieved an ASAS 40

No radiographic structural progression as measured by

response (Assessment of SpondyloArthritis International

modified Stoke Ankylosing Spondylitis Spine Score

Measures

Society criteria);

(mSASSS)

• Secukinumab 150 mg load

• Secukinumab 150/300 mg

Arms/Intervention

• Secukinumab 150 mg no load

• Adalimumab biosimilar 40 mg

• Placebo

Target Patients

Patients with non-radiographic axial spondyloarthritis

Patients with active ankylosing spondylitis

Expected Completion

Week 52: Q3-2019(actual); Final: H1-2021

2022

• Abstract (16 week results) presented at ACR 2019

• Study design manuscript published. Baraliakos et al.

Publication

• Abstract (52 week results) presented at EULAR 2020

Clinical Drug Investigation (2020) 40:269-278.

• Manuscript submitted in Mar-2020 (awaiting decision)

72 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

Cosentyx® - Anti IL-17

Study

NCT03713619 SUNSHINE (CAIN457M2301)

NCT04179175 (CAIN457M2301E1)

Indication

Hidradenitis Suppurativa (HS)

Hidradenitis Suppurativa (HS)

Phase

Phase 3

Phase 3

Patients

471

745

Primary Outcome

Proportion of participants with Hidradenitis Suppurativa

Proportion of patients with Hidradenitis Suppurativa Clinical

Measures

clinical response (HiSCR)

Response (HiSCR)

• Secukinumab 300 mg every 2 weeks

Arms/Intervention

• Secukinumab 300 mg every 4 weeks

• Secukinumab 300 mg every 2 weeks

• Placebo (every 2 weeks)

• Secukinumab 300 mg every 4 weeks

• Placebo (every 4 weeks)

Patients with moderate to severe hidradenitis suppurativa

Target Patients

Patients with moderate to severe Hidradenitis Suppurativa

completing either of the core trials AIN457M2301 (NCT

0313632) or AIN567M2302 (NCT03713619)

Expected Completion

Weak 16 DBL: H2-2021; Final: 2022

2025

Publication

• Study design SHSA 2020

Study design SHSA 2020

• Preliminary results in AAD (most likely) in 2022

73 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

Cosentyx® - Anti IL-17

Study

NCT03713632 SUNRISE (CAIN457M2302)

Indication

Hidradenitis Suppurativa (HS)

Phase

Phase 3

Patients

471

Primary Outcome

Proportion of patients with Hidradenitis Suppurativa Clinical

Measures

Response (HiSCR)

• Secukinumab 300 mg every 2 weeks

Arms/Intervention

• Secukinumab 300 mg every 4 weeks

• Placebo (every 2 weeks)

• Placebo (every 4 weeks)

Target Patients

Expected Completion

Publication

Subjects with moderate to severe Hidradenitis Suppurativa

Weak 16 DBL: H2-2021; Final: 2022

  • Study design SHSA 2020
  • Preliminary results in EADV (most likely) in 2021

74 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

Cosentyx® - Anti IL-17

Study

NCT04156620 INVIGORATE-1 (CAIN457P12301)

NCT04209205 INVIGORATE-2 (CAIN457P12302)

Indication

Axial spondyloarthritis

Axial spondyloarthritis

Phase

Phase 3

Phase 3

Patients

500

380

Primary Outcome Measures

Arms/Intervention

The proportion of subjects achieving an ASAS40

The proportion of subjects achieving American College of

(Assessment of SpondyloArthritis International Society

Rheumatology 50 (ACR50) response criteria

criteria) response

•

Secukinumab intravenous (i.v.) regimen

•

Secukinumab intravenous (i.v.) regimen

•

Placebo intravenous (i.v.) regimen

•

Placebo intravenous (i.v.) regimen

Target Patients

Patients with active axial spondyloarthritis

Patients with active psoriatic arthritis (PsA) despite current

or previous NSAID, DMARD and/or anti-TNF therapy

Expected Completion

2022

2022

Publication

TBD

TBD

75 Novartis Q2 Results | July 21, 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

Primary Outcome Measures

Arms/Intervention

Target Patients

Expected Completion

Proportion of patients in clinical remission on canakinumab

Number of patients with clinical response; Clinical response

who are able to remain in remission following canakinumab

is defined as survival without ever requiring invasive

dose tapering (reduced canakinumab dose or prolonged

mechanical ventilation from day 3 to day 29

canakinumab dosing interval)

•

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 &

Publication

EULAR in Q2-2019

Planned manuscript submission in Q4-2020

•

Planned manuscript in 2019: Remission & flexible dosing

submitted in Q4-2019

76 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

LJN452 - FXR Agonist

Study

NCT02855164 (CLJN452A2202)

NCT04065841 ELIVATE (CLJN452D12201C)

Indication

Non-alcoholic steatohepatitis (NASH)

Non-alcoholic steatohepatitis (NASH)

Phase

Phase 2

Phase 2

Patients

345

210

Adverse event profile of different doses; determine the dose

relationship of LJN452 on markers of hepatic inflammation

Proportion of patients with resolution of NASH and no

Primary Outcome

in NASH (ALT and AST); determine dose-response

worsening of fibrosis OR improvement in fibrosis by at least

Measures

relationship of LJN452 on liver fat content by changes in

one stage without worsening of NASH at Week 48

quantitative MRI; determine effect of LJN452 on liver fibrosis

compared with baseline

by biopsy

• Arm A: combination therapytropifexor + licogliflozin

• Arm B: tropifexor monotherapytropifexor (+ licogliflozin

Arms/Intervention

• Multiple LJN452 doses and placebo

placebo)

• Arm C: licogliflozin monotherapylicogliflozin (+ tropifexor

placebo)

Target Patients

Patients with non-alcoholic steatohepatitis (NASH)

Adult patients with non-alcoholic steatohepatitis (NASH)

and liver fibrosis

Expected Completion

Q2-2020(actual)

2022

• Primary (interim) data abstract submitted to AASLD in

Publication

Q3-2019

Planned in H1-2023

• Manuscript to be submitted in Q4-2020

77 Novartis Q2 Results | July 21, 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

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

• Long-term safety and tolerability

Measures

4

• Arm 1 Low dose of LOU064 orally in the morning (once daily) and

matching placebo in the evening from Day 1 to 85

• Arm 2 Medium dose of LOU064 orally in the morning (once daily) and

matching placebo in the evening from Day 1 to 85

Arms/Intervention• Arm 3 High dose of LOU064 orally in the morning (once daily) and matching placebo in the evening from Day 1 to 85

  • 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
  • Selected dose of LOU064 taken orally twice a day (morning and evening) from day 1 to week 52

Target Patients

Adults with CSU inadequately controlled by H1-antihistamines

Patients with CSU who have participated in preceding

studies with LOU064

Expected Completion

Q2-2021

2022

Publication

TBD

TBD

78 Novartis Q2 Results | July 21, 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

Measures

event profile, vital signs and laboratory parameters

•

Tropifexor

Arms/Intervention

•

Cenicriviroc

•

Tropifexor + cenicriviroc

Target Patients

Expected Completion

Publication

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

Q4-2020

Manuscript to be submitted in H1-2021

79 Novartis Q2 Results | July 21, 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 to

Long-term safety; number of participants with treatment-

Measures

achievement of complete hives response at week 12

emergent adverse events

• Ligelizumab 24mg q4wks for 20 weeks

• Ligelizumab 72mg q4wks for 20 weeks

Arms/Intervention

• Ligelizumab 240mg q4wks for 20 weeks

Ligelizumab 240 mg q4wks open label for 52 weeks

• Ligelizumab 120mg single dose

• Omalizumab 300mg q4wks for 20 weeks

• Placebo q 4wks for 20 weeks

Adult patients with chronic spontaneous urticaria inadequately

Adult patients with chronic spontaneous urticaria inadequately

controlled with H1-antihistamines at approved or increased doses,

Target Patients

controlled with H1-antihistamines at approved or increased

alone or in combination with H2-antihistamines or leukotriene

doses, alone or in combination with H2-antihistamines or

receptor antagonists.

leukotriene receptor antagonists.

Expected Completion

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, WCD,

GUF 2018; NEJM publication (3 Oct 2019);

EADV, PAAM, ACAAI, UCARE

• Secondary results presented in 2019 at: AAD, EAACI, WCD,

Publication

• Exploratory results presented/ planned in 2020: AAAAI,

EADV, PAAM, ACAAI, UCARE.

EAACI, EADV, ACAAI; Encoring all at GUF

• Exploratory results presented/ planned in 2020: AAAAI,

• 5 Manuscripts 2020: core results extension; angioedema;

EAACI, EADV, ACAAI; Encoring all at GUF

sleep/work impairment/rescue medication; data visualization

80 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

QGE031 - Anti-IgE

Study

NCT03437278 (CQGE031C2202)

NCT04210843 (CQGE031C2302E1)

Indication

Chronic spontaneous urticaria / Chronic idiopathic urticaria

Chronic spontaneous urticaria

Phase

Phase 2

Phase 3

Patients

48

800

Primary Outcome Measures

Arms/Intervention

Change in the 7 day Urticaria Activity Score (UAS7)

The proportion of subjects with well-controlled disease

(UAS7 ≤ 6) at week 12

• Ligelizumab high dose q4wks for 24 weeks

• Ligelizumab Dose 1 and 3

• 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

Expected Completion

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

81 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

QGE031 - Anti-IgE

Study

NCT03580369 Pearl 1 (CQGE031C2302)

NCT03580356 Pearl 2 (CQGE031C2303)

Indication

Chronic spontaneous urticaria

Chronic spontaneous urticaria

Phase

Phase 3

Phase 3

Patients

1,050

1,050

Primary Outcome

Absolute change from baseline in UAS7 (Urticaria Activity

Absolute change from baseline in UAS7 (Urticaria Activity

Measures

Score) at week 12

Score) at week 12

• Ligelizumab dose A q4w for 52 weeks

• Ligelizumab dose A q4w for 52 weeks

• Ligelizumab dose B q4w for 52 weeks

• Ligelizumab dose B q4w for 52 weeks

Arms/Intervention

• Omalizumab 300 mg q4w for 52 weeks

• Omalizumab 300 mg q4w for 52 weeks

• Placebo q4w from randomization to wk20, then

• Placebo q4w from randomization to wk20, then

ligelizumab dose B from wk24 to wk52

ligelizumab dose B from wk24 to wk52

Target Patients

Adolescents and adults with chronic spontaneous urticaria

Adolescents and adults with chronic spontaneous urticaria

inadequately controlled with H1-antihistamines

inadequately controlled with H1-antihistamines

Expected Completion

H2-2021

H2-2021

• Study design presented at UCARE 2018

Publication

• Primary results to be presented in 2022 (e.g. EAACI, PAAM, EADV)

• Manuscript to be submitted in 2022

82 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

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

Study

NCT02962895 (CVAY736A2201)

NCT03217422 AMBER (CVAY736B2201)

Indication

Primary Sjögren's syndrome

Autoimmune hepatitis

Phase

Phase 2B

Phase 2/3

Patients

180

80

Primary Outcome

Safety and efficacy of VAY736 in primary Sjögren's

Alanine aminotransferase (ALT) normalization

Measures

syndrome (pSS)

Arms/Intervention

•

VAY736

• VAY736

•

Placebo

• Placebo control with conversion to active VAY736

Target Patients

Patients with moderate to severe primary Sjögren's

Autoimmune hepatitis patients with incomplete response or

syndrome (pSS)

intolerant to standard treatment of care

Expected Completion

Q2-2020(actual)

2023

Publication

• Manuscript to be submitted in 2020

TBD

83 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

Neuroscience

Aimovig® - CGRP receptor antagonist

Study

NCT03096834 LIBERTY (CAMG334A2301)

NCT03333109 EMPOWER (CAMG334A2302)

Indication

Migraine

Migraine

Phase

Phase 3

Phase 3

Patients

246

900

Primary Outcome

Percentage of patients with a 50% response in the reduction

Change from baseline in monthly migraine days at the last

Measures

of Monthly Migraine Days (MMD)

month (Month 3) of the double-blind treatment period

• Subcutaneous injection of AMG334 (erenumab)

•

AMG334 (erenumab) Dose 1

Arms/Intervention

•

AMG334 (erenumab) Dose 2

• Subcutaneous injection of placebo

•

Placebo

Target Patients

Adult episodic migraine patients who have failed prophylactic

Adult episodic migraine patients

migraine treatments

Expected Completion

2017 DBT phase (actual); H1-2021 OLE phase (final DBL)

Q1-2020(actual)

• Planned for Q1-2020 (Neurology - rejected): PROs and

prespecified subgroup analysis (DBT phase) submitted

Publication

to JNNP in June 2020

Planned for H2-2020

• Planned for Q2-2020: 1Y OLE (submitted to Neurology)

• Planned for Q4 2020: 2Y OLE Abstracts completed for

EAN, AHS and EHF in 2020

85 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

Aimovig® - CGRP receptor antagonist

Study

NCT03867201 DRAGON (CAMG334A2304)

Indication

Migraine

Phase

Phase 3

Patients

550

Primary Outcome

Change from baseline in monthly migraine days during the

Measures

last 4 weeks of the 12-week treatment period

Arms/Intervention

• Subcutaneous injection of AMG334 (erenumab) 70 mg

• Subcutaneous injection of placebo

Target Patients

Adult chronic migraine patients

Expected Completion

2022 DBT phase; 2024 OLE phase

Publication

Planned in Q3-2022 (DBT) and H1-2025 for OLE

86 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

Gilenya® - S1P-R modulator

Study

NCT01633112 ASSESS (CFTY720D2312)

Indication

Relapsing remitting multiple sclerosis (RRMS)

Phase

Phase 3B

Patients

1,064

Primary Outcome

Comparison of 2 doses (0.25 mg and 0.5 mg) of fingolimod

to glatiramer acetate (20 mg) in reducing the annualized

Measures

relapse rate up to 12 months

Arms/Intervention

Target Patients

Expected Completion

Publication

  • Fingolimod 0.5 mg orally
  • Fingolimod 0.25mg orally
  • Copaxone® 20 mg s.c.

Patients with relapsing-remitting multiple sclerosis

2018 (actual)

  • Primary data presentation at AAN in 2019
  • Primary manuscript accepted by JAMA Neurology in June 2020

87 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

LMI070 - SMN2 RNA splice modulator

Study

NCT02268552 (CLMI070X2201)

Indication

Type 1 spinal muscular atrophy

Phase

Phase 1/2

Patients

39

Primary Outcome

Number of participants with adverse events (AEs), serious

Measures

adverse events (SAEs) and deaths

Branaplam oral, once weekly:

• Part 1:

5 ascending doses

Arms/Intervention

•

Part 2:

2 different dose levels

•

Part 3: patients continue on initial dose assigned in Part

1 or Part 2

Target Patients

Expected Completion

Publication

Patients with type 1 spinal muscular atrophy

Q3-2020 (Part 2)

TBD

88 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

Mayzent ® - S1P-R modulator

Study

NCT01665144 -EXPAND (CBAF312A2304)

Indication

Secondary progressive multiple sclerosis

Phase

Phase 3

Patients

1,652

Primary Outcome Measures

The delay in time to confirmed disability progression as

measured by EDSS (Expanded Disability Status Scale)

Arms/Intervention

Target Patients

Expected Completion

Publication

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

Patients with secondary progressive multiple sclerosis

Core in 2016/Extension in 2024

Siponimod versus placebo in secondary progressive multiple sclerosis (EXPAND): a double-blind, randomised, phase 3

study. Kappos L et al. Lancet 2018; 391:1263-73 DOI: https://doi.org/10.1016/S0140-6736(18)30475-6

89 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

OMB157 - Anti-CD20

Study

NCT02792218 Asclepios I (COMB157G2301)

NCT02792231 Asclepios II (COMB157G2302)

Indication

Multiple sclerosis

Multiple sclerosis

Phase

Phase 3

Phase 3

Patients

900

900

Primary Outcome

Annualized Relapse Rate (ARR) - number of confirmed

Annualized Relapse Rate (ARR) - number of confirmed

relapses in a year calculated based on cumulative number

relapses in a year calculated based on cumulative number

Measures

of relapses by patient adjusted for time-in-study by patient

of relapses by patient adjusted for time-in-study by patient

Arms/Intervention

•

Ofatumumab subcutaneous

•

Ofatumumab subcutaneous

•

Teriflunomide oral

•

Teriflunomide oral

Target Patients

Patients with relapsing forms of multiple sclerosis

Patients with relapsing forms of multiple sclerosis

Expected Completion

Q3-2019(actual)

Q3-2019(actual)

Publication

Primary manuscript planned in H1-2020

Primary manuscript planned in H1-2020

90 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

OMB157 - Anti-CD20

Study

NCT03249714 APOLITOS (COMB157G1301)

NCT03650114 ALITHIOS (COMB157G2399)

Indication

Multiple sclerosis

Multiple Sclerosis

Phase

Phase 2

Phase 3

Patients

60

2010

Primary Outcome

Reduced cumulative number of Gd-enhanced T1 lesions

across 4 MRI scans at week 12, 16, 20 and 24 (ofatumumab

Measures

vs placebo)

Evaluate the long-term safety and tolerability of ofatumumab 20 mg subcutaneous (sc) once every 4 (q4) weeks in subjects with RMS from the first dose of ofatumumab

Arms/Intervention

• Ofatumumab 20 mg subcutaneous injections

• Ofatumumab 20 mg every 4 weeks

• Placebo

Target Patients

Patients with relapsing forms of multiple sclerosis

Patients with relapsing MS

Expected Completion

Q1-2020(actual)

2028

Publication

TBD

TBD

91 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

Zolgensma® - SMN1 gene replacement therapy

Study

NCT03461289 STRIVE-EU(CL-302)

NCT03306277 STRIVE (CL-303)

Indication

Type 1 spinal muscular atrophy

Type 1 spinal muscular atrophy

Phase

Phase 3

Phase 3

Patients

33

22

Primary Outcome

• Achievement of independent sitting for at least 30

Proportion of participants sitting without support

seconds

Measures

• Event-free survival

Arms/Intervention

Open-label,single-arm,single-dose, intravenous

Open-label,single-arm,single-dose, intravenous

Target Patients

Patients with spinal muscular atrophy Type 1

Patients with Spinal Muscular Atrophy Type 1

Expected Completion

Q4-2020

Q4-2019(actual)

Publication

WMS 2020, Manuscript planned H1-2021

MDA 2020, Manuscript submission Jul-2020

92 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

Zolgensma® - SMN1 gene replacement therapy

Study

NCT03505099 SPR1NT (CL-304)

NCT03837184 STRIVE Asia Pacific (CL-306)

Indication

Spinal muscular atrophy

Type 1 spinal muscular atrophy

Phase

Phase 3

Phase 3

Patients

30

6

• [2 copies of SMN2] Percentage of participants achieving

functional independent sitting for at least 30 seconds at

Primary Outcome

any visit

Proportion of participants sitting without support

Measures

• [3 copies of SMN2] Percentage of participants achieving

the ability to stand without support for at least 3 seconds

at any visit

Arms/Intervention

Open-label,single-arm,single-dose, intravenous

Open-label,single-arm,single-dose, intravenous

Target Patients

Pre-symptomatic patients with spinal muscular atrophy and

Patients with spinal muscular atrophy Type 1

multiple copies SMN2

Expected Completion

H2-2021

H2-2021

Publication

MDA 2020 (interim), Manuscript planned in H1-2021

TBD

93 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

Zolgensma® - SMN1 gene replacement therapy

Study

NCT03381729 STRONG (CL-102)

Indication

Type 2 spinal muscular atrophy

Phase

Phase 1

Patients

51

Primary Outcome

• Safety and tolerability, incidence of adverse events

• Proportion of patients achieving Standing Milestone

Measures

• Change in Hammersmith Functional Motor Scale

Arms/Intervention

Open-label,single-arm,single-dose, intrathecal

Target Patients

Patients with spinal muscular atrophy with 3 copies of SMN2

Expected Completion

Q4-2019 [Cohort B] (actual); TBD [Cohort C]1

Publication

MDA 2020, Manuscript planned for 2H 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

94 Novartis Q2 Results | July 21, 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

Patients with chronic myelogenous leukemia in chronic

Target Patients

phase, previously treated with 2 or more tyrosine kinase

inhibitors

Expected Completion

Q3-2020

Publication

•

Manuscript submission Q4-2020

• Abstract submission to congress Q3-2020

96 Novartis Q2 Results | July 21, 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

Primary Outcome Measures

Arms/Intervention

Target Patients

Expected Completion

Publication

• Safety run-in part: Incidence of dose limiting toxicities

Disease free survival (primary), overall survival (key

•

Double-blind, randomized, placebo-controlled part:

secondary)

Progression free survival (PFS)

•

Overall survival (OS)

• Canakinumab 200mg q3w sc for 18 cycles

• Canakinumab or matching placebo in combination with

pembrolizumab and platinum-based doublet

• Placebo q3w sc for 18 cycles

chemotherapy

Patients with:

Patients with

• High-risk NSCLC (AJCC/UICC v.8 stage II-IIIA and IIIB

• Histologically confirmed Stage IIIB, IV NSCLC with no

(T>5cm N2)) after complete resection and standard of

prior systemic anticancer therapy

care adjuvant cisplatin-based chemotherapy

• Squamous and non-squamous NSCLC

• All histologies

• No EGFR mutation and ALK rearrangement

Interim Analysis: 2022; Final: 2023

Interim Analysis: Q4-2020; Final: 2022

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

97 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

ACZ885 - IL1β inhibitor

Study

NCT03626545 CANOPY-2 (CACZ885V2301)

Indication

Phase

Patients

Primary Outcome Measures

Arms/Intervention

Target Patients

Expected Completion

Publication

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

Phase 3

240

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

Patients with:

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

H1-2021

Abstract submission to congress H1-2021

98 Novartis Q2 Results | July 21, 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

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

Expected Completion

2018 (actual)

2023

•

Andre F, et al. Presentation at ESMO 2018

Publication

•

Andre et al. Manuscript N Engl J Med 2019;380:1929-

TBD

1940.

99 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

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

Study

NCT00940602 TELESTO (CICL670A2302)

Indication

Iron overload

Phase

Phase 2

Patients

224

Primary Outcome

To compare deferasirox to placebo with regard to event-free

survival in low and int-1 risk MDS patient with transfusional

Measures

iron overload

Arms/Intervention

•

Deferasirox, iron chelator

•

Placebo

Target Patients

Patients with myelodysplastic syndromes (low/int-1 risk) and

transfusional iron overload

Expected Completion

2018 (actual)

• Angelucci E, et al. Presentation at ASH 2018

Publication

• Angelucci E, et al. Manuscript Ann Intern Med

2020;172:513-522.

100 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

INC280 - MET Inhibitor

Study

NCT02414139 (CINC280A2201)

Indication

EGFR Wild-type, ALK negative advanced Non-small Cell Lung

Cancer (NSCLC)

Phase

Phase 2

Patients

364

Primary Outcome

Overall Response Rate (ORR)

Measures

• Pre-treated pts. with MET GCN: ≥ 6; ≥ 4 and < 6; < 4

• Pre-treated pts. with MET mutations regardless of cMET GCN

as second or third line

Arms/Intervention

• Treatment-naïve pts. with MET dysregulation

• Pre-treated pts with MET dysregulation - second line

• Treatment-naïve pts with cMET mutations regardless of cMET

GCN

Target Patients

Expected Completion

Publication

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

2019 (actual)

  • Wolf J, et al. Presented at ASCO 2019
  • Wolf J, et al. Presentation at ASCO 2020 (cohort 1 and 5a)
  • Groen H, et al. Presentation at ASCO 2020 (cohort 6)
  • Wolf J, et al. Manuscript submitted Q1-2020

101 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

Jakavi® - JAK1/2 inhibitor

Study

NCT02913261 REACH2 (CINC424C2301)

NCT03112603 REACH3 (CINC424D2301)

Indication

Steroid-refractory acute graft vs. host disease (SR aGVHD)

Steroid-refractory chronic graft vs. host disease (SR cGVHD)

Phase

Phase 3

Phase 3

Patients

310

330

Primary Outcome

Overall Response Rate (ORR) at 28 Days

Overall Response Rate (ORR) at 183 Days

Measures

Arms/Intervention

•

Ruxolitinib 10mg bid

•

Ruxolitinib 10mg bid

•

Best available therapy (BAT)

•

Best available therapy (BAT)

Target Patients

Patients with SR aGVHD

Patients with SR cGVHD

Expected Completion

2019 (actual)

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

• Zeiser R, et al. Manuscript N Engl J Med 2020;382:1800-

Publication

1810.

•

Manuscript submission in H2-2020

• Zeiser R, et al. Abstract accepted for presentation at

• Abstract submission to congress in H2-2020

EBMT Q3-2020

102 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

Jakavi® - JAK1/2 inhibitor

Study

NCT03491215 REACH4 (CINC424F12201)

NCT03774082 REACH5 (CINC424G12201)

Indication

Acute graft versus host disease

Chronic graft versus host disease

Phase

Phase 2

Phase 2

Patients

45

42

Primary Outcome

• Measurement of PK parameters

• Overall Response Rate (ORR)

Measures

• Overall Response Rate (ORR)

Arms/Intervention

Target Patients

Expected Completion

Publication

• Ruxolitinib

• Ruxolitinib 5mg tablets / pediatric formulation

Pediatric patients with grade II-IV acute graft vs. host disease

Pediatric subjects with moderate and severe chronic Graft

after allogeneic hematopoietic stem cell transplantation

vs. Host disease after allogeneic stem cell transplantation

2023

2026

TBD

TBD

103 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

Jakavi® - JAK1/2 inhibitor

Study

NCT04097821 ADORE (CINC424H12201)

Indication

Myelofibrosis

Phase

Phase 1/2

Patients

130

Primary Outcome

• Incidence of dose limiting toxicities within the first 2

cycles

Measures

• Response rate at the end of cycle 6

•

Ruxolitinib

Arms/Intervention

•

Ruxolitinib+Siremadlin

•

Ruxolitinib+Crizanlizumab

•

Ruxolitinib+MBG453

Target Patients

Expected Completion

Publication

Patients with Myelofibrosis (MF)

2024

TBD

104 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

Kisqali® - CDK 4/6 inhibitor

Study

NCT03701334 NATALEE (CLEE011O12301C)

Indication

Adjuvant treatment of hormone receptor (HR)-positive,

HER2-negative, early breast cancer (EBC)

Phase

Phase 3

Patients

~4,000

Primary Outcome

Invasive Disease-Free Survival for using STEEP criteria

(Standardized Definitions for Efficacy End Points in adjuvant

Measures

breast cancer trials)

Arms/Intervention

• Ribociclib + endocrine therapy

• Endocrine therapy

Pre and postmenopausal women and men with HR-positive,

Target Patients

HER2-negative EBC, after adequate surgical resection, who

are eligible for adjuvant endocrine therapy

Expected Completion

Interim Analysis: H1-2022; Final: H2-2022

Publication

TBD

105 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

Kymriah® - CAR-T therapy

Study

NCT03568461 ELARA (CCTL019E2202)

NCT03876769 CASSIOPEIA (CCTL019G2201J)

Indication

Relapsed / refractory follicular lymphoma (FL)

1st line high risk acute lymphoblastic leukemia (ALL)

Phase

Phase 2

Phase 2

Patients

113

160

Primary Outcome

Complete Response Rate (CRR)

Disease Free Survival (DFS)

Measures

Arms/Intervention

Single-arm study of tisagenlecleucel

Single-arm study of tisagenlecleucel

Target Patients

Adult patients with relapsed or refractory FL

Pediatric and young adult patients with 1st line high risk ALL

Expected Completion

Interim Analysis: Q3-2020

2025

• ELARA interim analysis - ASH 2020, tbc

• ELARA primary analysis - Planned for ASCO/EHA 2021

• ELARA vs RECORD full analysis - Planned for

ASCO/EHA 2021

• High-risk patients (ELIANA/CASSIOPEIA) - Planned

Publication

• ELARA vs Flatiron - Planned for ASCO/EHA 2021

submission H1-2022

• ELARA: Primary analysis MS - simultaneous publication

with congress

• ELARA vs RECORD: Full analysis - simultaneous

publication with congress

106 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

Kymriah® - CAR-T therapy

Study

NCT03570892 BELINDA (CCTL019H2301)

Indication

2nd line Diffuse large B-cell lymphoma (DLBCL)

Phase

Phase 3

Patients

318

Primary Outcome

Event-free Survival (EFS)

Measures

Arms/Intervention

Tisagenlecleucel versus standard of care

Adult patients with aggressive B-cellNon-Hodgkin

Target Patients

Lymphoma after failure of rituximab and anthracycline-

containing frontline immunochemotherapy

Expected Completion

H2-2021

• Westin et al. presentation at SOHO 2019, Bishop et al at

SITC 2019, Bishop et al abstract planned to DGHO

Publication

2020; BELINDA TiP

• Primary analysis Planned for ASH 2021

• Primary manuscript - TBD

107 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

MBG453 - TIM-3 antagonist

Study

NCT03946670 STIMULUS MDS-1 (CMBG453B12201)

NCT04266301 STIMULUS-MDS2 (CMBG453B12301)

Indication

Myelodysplastic syndrome

Myelodysplastic syndrome

Phase

Phase 2

Phase 3

Patients

120

500

Primary Outcome

Complete Remission (CR) rate and Progression Free

Overall survival

Measures

Survival (PFS)

Arms/Intervention

• Experimental: MBG453 + hypomethylating agents

• MBG453 800 mg + azacitidine 75 mg/m2

• Placebo comparator: Placebo + hypomethylating agents

• MBG453 800 mg + azacitidine 75 mg/m2 + placebo

Adult subjects with intermediate, high or very high risk

Patients with intermediate, high or very high risk

Target Patients

Myelodysplastic Syndrome (MDS) as Per IPSS-R, or

Myelodysplastic Syndrome (MDS) as per IPSS-R criteria

Chronic Myelomonocytic Leukemia-2(CMML-2)

Expected Completion

H2-2021

2023

Publication

Abstract submission to congress in H2-2021

TBD

108 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

PDR001 - PD-1 checkpoint inhibitor

Study

NCT02967692 COMBI-i (CPDR001F2301)

Indication

Phase

Patients

Primary Outcome Measures

Arms/Intervention

Target Patients

Expected Completion

Publication

BRAFV600 mutant metastatic melanoma

Phase 3

538

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

(Phase III, randomized, placebo controlled): 532

Progression-Free Survival (PFS)

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

Previously untreated patients with unresectable or metastatic BRAF V600 mutant melanoma

Q3-2020

  • Abstract submission to congress in Q3-2020
  • Manuscript submission Q3/Q4-2020

109 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

PDR001 - PD-1 checkpoint inhibitor

Study

NCT03484923 (CPDR001J2201)

Indication

Previously treated unresectable or metastatic melanoma

Phase

Phase 2

Patients

195

Primary Outcome

Objective Response Rate (ORR)

Measures

• PDR001 400mg i.v. Q4W + LAG525 (to be tested in

unselected patients and LAG-3 positive patients)

Arms/Intervention

• PDR001 400mg i.v. Q4W + capmatinib

• PDR001 400mg i.v. Q4W + canakinumab

• PDR001 400mg i.v. Q4W + ribociclib

Target Patients

Expected Completion

Publication

Adult patients with previously treated unresectable or metastatic melanoma

H2-2021

TBD

110 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

Promacta®/Revolade® - Thrombopoetin receptor agonist

Study

NCT03025698 (CETB115E2201)

NCT03988608 (CETB115E2202)

Indication

Previously untreated or relapsed/refractory severe aplastic

Previously untreated or relapsed/refractory severe aplastic

anemia or recurrent aplastic anemia

anemia or recurrent aplastic anemia

Phase

Phase 2

Phase 2

Patients

60

20

Primary Outcome Measures

Arms/Intervention

PK of eltrombopag at steady state in pediatric patients with

Hematologic response rate

SAA

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

eltrombopag

• Eltrombopag 25 mg film-coated tablets

• Arm A: relapsed/refractory SAA or AA:

hATG/cyclosporine + eltrombopag or cyclosporine +

eltrombopag

Target Patients

Expected Completion

Publication

Pediatric patients from age 1 <18 years with

Chinese patients with refractory or relapsed severe aplastic

relapsed/refractory SAA or recurrent AA after IST or

anemia

previously untreated SAA

2025

2023

TBD

TBD

111 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

Rydapt®- Multi-targeted kinase inhibitor

Study

NCT03280030 (CPKC412A2220)

NCT03591510 (CPKC412A2218)

Indication

Acute myeloid leukemia

Acute myeloid leukemia

Phase

Phase 2

Phase 2

Patients

66

50

Primary Outcome

Incidence of safety events and event free survival

Occurrence of dose limiting toxicities

Measures

Event Free Survival ( EFS)

Arms/Intervention

•

Midostaurin 50 mg

• Chemotherapy followed by Midostaurin

•

Placebo

Target Patients

Expected Completion

Newly diagnosed patients with FLT3-mutated acute myeloid

Newly diagnosed pediatric patients with FLT3 mutated acute

leukemia (AML) from pan-Asia countries

myeloid leukemia (AML)

Interim analysis: Q2-2020(actual); Final: H2-2021

2022

Publication

Abstract submission to congress in Q4-2020

TBD

112 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

SEG101 - p-Selectin inhibitor

Study

NCT03264989 SOLACE-Adults (CSEG101A2202)

NCT03474965 SOLACE-Kids (CSEG101B2201)

Indication

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

Prevention of VOC in pediatric patients with SCD

Sickle Cell Disease (SCD)

Phase

Phase 2

Phase 2

Patients

55

100

Primary Outcome

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

PK/PD and safety of SEG101 at 5 mg/kg

Measures

SEG101 (crizanlizumab) at a dose of 5.0 mg/kg (or 7.5

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

Arms/Intervention

mg/kg for exploratory group) by IV infusion, ±

± Hydroxyurea/Hydroxycarbamide

Hydroxyurea/Hydroxycarbamide

Target Patients

Adult SCD patients with VOC

Pediatric SCD patients with VOC

Expected Completion

2018 (actual)

H2-2021 (pediatric patients ≥6 year old)

2022 (pediatric patients 6 months - 6 year old)

Publication

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

Abstract submission to congress in Q3-2020

113 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

SEG101 - p-Selectin inhibitor

Study

NCT03814746 STAND (CSEG101A2301)

Indication

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

Sickle Cell Disease (SCD)

Phase

Phase 3

Patients

240

Primary Outcome

Rate of VOC events leading to healthcare visit

Measures

•

Crizanlizumab 5.0 mg/kg

Arms/Intervention

•

Crizanlizumab 7.5 mg/kg

•

Placebo

Target Patients

Adolescent and adult SCD patients (12 years and older)

Expected Completion

2022

Publication

TBD

114 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

Tafinlar® - BRAF inhibitor

Study

NCT01677741 (CDRB436A2102)

Indication

BRAFV600 mutant cancers

Phase

Phase 1/2

Patients

85

Primary Outcome

Safety, tolerability and pharmacokinetics

Measures

Arms/Intervention

Single-arm study of oral dabrafenib (dose based on age

and weight)

Target Patients

Pediatric subjects aged 1 year to <18 years with advanced

BRAF V600-mutation positive solid tumors

Expected Completion

H1-2021

• Kieran MW et al. Manuscript Clin Cancer Res

2019;25(24):7294-7302 (PK analysis)

Publication

• Hargrave DR et al. Manuscript Clin Cancer Res

2019;25(24):7303-7311 (safety/efficacy in low-grade

gliomas)

115 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

Tafinlar®+Mekinist® - BRAF inhibitor and MEK inhibitor

Study

NCT02684058 (CDRB436G2201)

Indication

BRAFV600 mutant gliomas

Phase

Phase 2

Patients

142

Primary Outcome

Objective response rate

Measures

Arms/Intervention

Dabrafenib + trametinib (dose based on age and weight)

Target Patients

Children and adolescent patients with BRAF V600 mutation

positive relapsed or refractory high grade glioma (HGG) or

BRAF V600 mutation positive low grade glioma (LGG)

Expected Completion

2022

Publication

TBD

116 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

Tafinlar®+Mekinist® - BRAFV600 inhibitor and MEK inhibitor

Study

NCT02124772 (CTMT212X2101)

Indication

BRAFV600 mutant solid tumors

Phase

Phase 1/2A

Patients

139

Primary Outcome

Safety, tolerability and pharmacokinetics and clinical activity

Measures

Arms/Intervention

Trametinib (dose based on age and weight)

Dabrafenib + trametinib (dose based on age and weight)

Target Patients

Pediatric Subjects Aged 1 Month to <18 Years with

Advanced V600-Mutation Positive Solid Tumors

Expected Completion

H1-2021

Publication

• Geoerger B, et al. Presentation at ASCO 2020

• Manuscript submission Q4-2020

117 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

Zykadia® - ALK inhibitor

Study

NCT02299505 ASCEND-8 (CLDK378A2112)

Indication

ALK activated NSCLC

Phase

Phase 2

Patients

306

Primary Outcome

Part 1: Pharmacokinetics when taken with food

Measures

Part 2: Overall Response Rate (ORR) when taken with food

• Oral LDK378 450 mg once daily taken with food

Arms/Intervention

• Oral LDK378 600 mg once daily taken with food

• Oral LDK378 750 mg once daily fasted

Target Patients

Adult patients with ALK-rearranged(ALK-positive) advanced non-small cell

lung cancer

Part 1 (PK): 2016 (actual)

Expected Completion

Part 2 (ORR): Q4-2018(actual)

Final (ORR): Q3-2020

• Part 1 (PK): Cho BC, et al. J Thorac Oncol. 2017 Sep; 12(9) 1357-1367

Publication

• Part 2 (ORR): Cho B et al. J Thorac Oncol. 2019 Jul; 14(7) 1255-1265

• Final (ORR): Abstract submission to congress Q3-2020

118 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

177Lu-PSMA-617 - Radioligand therapy targeting prostate specific membrane antigen (PSMA)

Study

NCT03511664 VISION (PSMA-617-01)

Indication

PSMA-positive Metastatic Castration-resistant Prostate

Cancer (mCRPC)

Phase

Phase 3

Patients

831

Primary Outcome

•

Radiographic Progression Free Survival

Measures

•

Overall Survival

Arms/Intervention

•

177Lu-PSMA-617 plus BS/BSC

•

BS/BSC alone

Adult patients with PSMA-positive Metastatic Castration-

Target Patients

resistant Prostate Cancer (mCRPC)

Expected Completion

Q1-2021

Publication

TBD

119

Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

Lutathera® - Radioligand therapy targeting somatostatin receptor type 2

Study

NCT03972488 NETTER-2 (CAAA601A22301)

Indication

Gastroenteropancreatic neuroendocrine tumors (GEP-NET)

Phase

Phase 3

Patients

222

Primary Outcome

• Progression Free Survival

Measures

Arms/Intervention

• Lutathera plus long-acting octreotide

• high dose long-acting octreotide

Adult patients with Grade 2 and Grade 3 Advanced GEP-

Target Patients

NET

Expected Completion

2023

Publication

TBD

120

Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

Ophthalmology

Lucentis® - Anti-VEGF

Study

NCT02375971 RAINBOW (CRFB002H2301)

NCT02640664 RAINBOW Extension (CRFB002H2301E1)

Indication

Retinopathy of Prematurity (ROP)

Retinopathy of Prematurity (ROP)

Phase

Phase 3

Phase 3

Patients

224

180

Absence of active Retinopathy of Prematurity (ROP) and

Primary Outcome

unfavorable structural outcome at Week 24, defined as, 1)

To evaluate the visual function of patients by assessing the

survival, 2) no intervention with a second modality for ROP,

visual acuity in the better-seeing eye at the patient's fifth

Measures

3) absence of active ROP and 4) absence of unfavorable

birthday.

structural outcome

• Ranibizumab 0.2 mg (up to 3 injections max)

• Ranibizumab 0.2 mg (up to Week 40, if warranted)

Arms/Intervention

• Ranibizumab 0.1 mg (up to 3 injections max)

• Ranibizumab 0.1 mg (up to Week 40, if warranted)

•

Laser therapy

Target Patients

Male and female preterm infants with bilateral retinopathy of

Male and female preterm infants with bilateral retinopathy of

prematurity (ROP) who require treatment.

prematurity (ROP) who completed RAINBOW.

Expected Completion

2018 (actual)

2023

•

EURETINA: Sep-2018

•

AAO: Oct-2018

• Primary manuscript published online by The Lancet in

Sep-2019

Publication

(https://www.thelancet.com/pdfs/journals/lancet/PIIS0140

Submission of publication of 2 year data (Interim Analysis 2)

-6736(19)31344-3.pdf)

in 2020

• Submission of manuscript on Pop PK/PD analysis in

2020

• Submission of manuscript on time-course of clinical

response to treatment in 2020

122 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

RTH258 - Anti-VEGF

Study

NCT02434328 HARRIER (CRTH258A2302)

NCT02307682 HAWK (CRTH258A2301)

Indication

Neovascular age-related macular degeneration (nAMD)

Neovascular age-related macular degeneration (nAMD)

Phase

Phase 3

Phase 3

Patients

743

1,082

Primary Outcome

Change in Best Corrected Visual Acuity (BCVA) from

Change in Best Corrected Visual Acuity (BCVA) from

Measures

baseline at week 48

baseline at week 48

• Brolucizumab (RTH258) 6 mg/50 µL

• Brolucizumab (RTH258) 3 mg/50 µL

Arms/Intervention

• Brolucizumab (RTH258) 6 mg/50 µL

• Aflibercept 2 mg/50 µL

• Aflibercept 2 mg/50 µL

Target Patients

Subjects with exudative age-related macular degeneration

Subjects with exudative age-related macular degeneration

Expected Completion

2018 (actual)

2018 (actual)

• Year 1 Manuscript: Dugel P, et al. Ophthalmology 2019 Apr 12; HAWK and HARRIER: Phase 3, Multicenter, Randomized, Double-Masked Trials of Brolucizumab for Neovascular Age-Related Macular Degeneration.

Publication

•

Secondary publications planned for 2020 are: Fluid resolution, PCV and CNV subtypes, CST variability, the IPDA, safety

and VFQ outcomes submitting in Q1-Q3 of 2020

•

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

(WOC; ARVO; ASRS, EURETINA AAO and APVRS) throughout 2020

123 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

RTH258 - Anti-VEGF

Study

NCT03386474 (CRTH258A2301E1)

NCT03481634 KESTREL (CRTH258B2301)

Indication

Neovascular age-related macular degeneration (nAMD)

Diabetic eye disease

Phase

Phase 3

Phase 3

Patients

150

534

Primary Outcome

Number of treatment-emergent adverse events

Change from baseline in best-corrected visual acuity

Measures

(BCVA)

• Brolucizumab (RTH258) 6 mg/50 µL

• Brolucizumab (RTH258) 3 mg/50 µL

Arms/Intervention

• Brolucizumab (RTH258) 6 mg/50 µL

• Aflibercept 2 mg/50 µL

• Aflibercept 2mg/50 uL

Target Patients

Patients with neovascular age-related macular degeneration

Patients with visual impairment due to diabetic macular

who have completed the CRTH258A2301 study

edema (DME)

Expected Completion

2018 (actual)

H2-2021

Publication

Planned publication of the attributes of brolucizumab and

durability in Q1-2020

Week 52 safety and efficacy data to be submitted as an abstract in H1-2021 (KITE and KESTREL)

124 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

RTH258 - Anti-VEGF

Study

NCT03481660 KITE (CRTH258B2302)

NCT04058067 KINGLET (CRTH258B2304)

Indication

Diabetic eye disease

Diabetic macular edema

Phase

Phase 3

Phase 3

Patients

356

268

Primary Outcome

Change from baseline in best-corrected visual acuity

Change in best-corrected visual acuity (BCVA)

Measures

(BCVA)

Arms/Intervention

• Brolucizumab (RTH258) 6 mg/50 µL

• Brolucizumab (RTH258) 6 mg/50 µL

• Aflibercept 2 mg/50 µL

• Aflibercept 2 mg/50 µL

Target Patients

Patients with visual impairment due to diabetic macular

Chinese patients with visual impairment due to diabetic

edema (DME)

macular edema

Expected Completion

H2-2021

2023

Publication

Week 52 safety and efficacy data to be submitted as an

Publication planned for 2023

abstract H1 2021 (KITE and KESTREL)

125 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

RTH258 - Anti-VEGF

Study

NCT03917472 KINGFISHER (CRTH258B2305)

NCT03802630 RAPTOR (CRTH258C2301)

Indication

Diabetic macular edema

Retinal vein occlusion

Phase

Phase 3

Phase 3

Patients

500

500

Primary Outcome

Change in best-corrected visual acuity (BCVA) from

Change from baseline in best-corrected visual acuity

Measures

baseline up to week 52

(BCVA) at week 24

Arms/Intervention

• Brolucizumab (RTH258) 6 mg/50 µL

• Brolucizumab (RTH258) 6 mg/50 µL

• Aflibercept 2 mg/50 µL

• Aflibercept 2 mg/50 µL

Target Patients

Patients with visual impairment due to diabetic macular

Adult patients with visual impairment due to macular edema

edema

secondary to branch retinal vein occlusion

Expected Completion

H2-2021

2023

Publication

Publication submission planned for 2022

Publication submission planned for 2023

126 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

RTH258 - Anti-VEGF

Study

NCT03810313 RAVEN (CRTH258C2302)

NCT04047472 HOBBY (CRTH258A2307)

Indication

Retinal vein occlusion

Macular degeneration

Phase

Phase 3

Phase 3

Patients

750

494

Primary Outcome

Change from baseline in best-corrected visual acuity

Change from baseline in best-corrected visual acuity

Measures

(BCVA) at week 24

(BCVA) at week 48

Arms/Intervention

• Brolucizumab (RTH258) 6 mg/50 µL

• Brolucizumab (RTH258) 6 mg/50 µL

• Aflibercept 2 mg/50 µL

• Aflibercept 2 mg/50 µL

Target Patients

Adult patients with visual impairment due to macular edema

Chinese patients with neovascular age-related macular

secondary to central retinal vein occlusion

degeneration

Expected Completion

2023

2024

Publication

TBD

TBD

127 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

UNR844 - Disulfide bonds modulator

Study

NCT03809611 (CUNR844A2203)

Indication

Presbyopia

Phase

Phase 2

Patients

124

Primary Outcome

Change in binocular distance-corrected near visual acuity

Measures

(DNCVA) from baseline at month 3

Arms/Intervention

•

1.5% solution UNR844-Cl

•

Placebo

Target Patients

Patients with presbyopia

Expected Completion

Q1-2020(actual)

Publication

Expected at AAOptom

128 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

Respiratory

INC424 - JAK Inhibitor

Study

NCT04362137 RUXCOVID (CINC424J12301)

Indication

COVID-19 (cytokine storm)

Phase

Phase 3

Patients

402

Proportion of patients who die, develop respiratory failure

Primary Outcome Measures

(requires mechanical ventilation), or require intensive care

unit care

Arms/Intervention

• Ruxolitinib 5 mg tablet given bid

• Placebo

Target Patients

Patients with COVID-19 respiratory disease

Expected Completion (LPLV)

Q4-2020

Publication

Manuscript submission targeted for Q1-2 2021

130 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

QBW251 - CFTR potentiator

Study

NCT04072887 (CQBW251B2201)

Indication

Chronic obstructive pulmonary disease (COPD)

Phase

Phase 2

Patients

956

Primary Outcome

Trough FEV1 (Forced Expiratory Volume in 1 second)

Measures

change from baseline after 12 weeks of treatment

•

QBW251 450 mg

•

QBW251 300 mg

Arms/Intervention

•

QBW251 150 mg

•

QBW251 75 mg

•

QBW251 25 mg

•

Placebo

Target Patients

COPD patients on background triple inhaled therapy (LABA /

LAMA / ICS)

Expected Completion

H2-2021

Publication

TBD

131 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

QMF149 - Long-acting beta2 agonist and inhaled corticosteroid

Study

NCT02892019 (CQMF149G2202)

Indication

Asthma

Phase

Phase 2

Patients

80

Primary Outcome

Trough FEV1

Measures

Arms/Intervention

• Indacaterol acetate 75 μg od (via Concept1 inhaler)

• Indacaterol acetate 150 μg od (via Concept1 inhaler)

Target Patients

Children ≥ 6 to < 12 years of age with asthma

Expected Completion

2019 (actual)

Publication

TBD

132 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

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

Study

NCT02554786 PALLADIUM (CQVM149B2301)

NCT02571777 IRIDIUM (CQVM149B2302)

Indication

Asthma

Asthma

Phase

Phase 3

Phase 3

Patients

2,216

3,092

Primary Outcome

Trough FEV1

Trough FEV1

Measures

• QMF149 150/160 µg od

•

QVM149 150/50/160 µg od

• QMF149 150/320 µg od

•

QVM149 150/50/80 µg od

Arms/Intervention

• MF 400 µg od

• QMF149 150/160 µg od

• MF 400 µg bid

• QMF149 150/320 µg od

• Salmeterol 50 µg /fluticasone 500 µg bid

• Salmeterol 50 µg /fluticasone 500 µg bid

Adult and adolescent (≥12 years) patients with asthma

Adult (≥18 years) patients with asthma inadequately

Target Patients

inadequately controlled on medium/high-dose ICS or low-

controlled on medium/high-dose of LABA/ICS (GINA step ≥4)

dose LABA/ICS (GINA step ≥ 3)

Expected Completion

2019 (actual)

2019 (actual)

Publication

• Abstract: van Zyl-Smit et al, presented at BTS Dec-2019

• Kerstjens H. et al. Lancet Resp Med 2020 (in press)

• Van Zyl-Smit R. et al. Lancet Resp Med 2020 (in press)

133 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

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

Study

NCT03100500 (CQVM149B1305)

NCT03100825 (CQVM149B1304)

Indication

Asthma

Asthma

Phase

Phase 3

Phase 3

Patients

51

94

Primary Outcome

Long-term safety/tolerability: Incidence and severity of

Long-term safety/tolerability: Incidence and severity of

treatment emergent adverse events during the 52 weeks

treatment emergent adverse events during the 52 weeks

Measures

study

study

Arms/Intervention

• Single arm: QMF149 150/320 μg od

• Single Arm: QVM149 150/50/160 μg od

Target Patients

Japanese patients with asthma inadequately controlled

Japanese patients with asthma inadequately controlled

Expected Completion

2019 (actual)

2019 (actual)

• Japanese J Allergo (B1304/1305 combined); Planned in

• Japanese J Allergo (B1304/1305 combined); Planned in

Publication

Q4 2020

Q4 2020

• Sagara H, et al. Abstract presented at ATS 2020

• Nakamura Y, et al. Abstract presented at ATS 2020

134 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

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

Study

NCT02892344 QUARTZ (CQVM149B2303)

NCT03158311 ARGON (CQVM149B2306)

Indication

Asthma

Asthma

Phase

Phase 3

Phase 3

Patients

802

1,251

Primary Outcome

Trough FEV1

Non-inferiority of Asthma Quality of Life Questionnaire

Measures

(AQLQ)

• QMF149 150/80 µg od

•

QVM149 150/50/80 μg od

Arms/Intervention

•

QVM149 150/50/160 μg od

• MF 200 µg od

• Salmeterol/fluticasone 50/500 μg bid + tiotropium 5 μg od

Adult and adolescent (≥12 years) patients with mild asthma

Target Patients

inadequately controlled on low-dose ICS or low-dose

Patients with uncontrolled asthma

LABA/ICS (Gina step 2-3)

Expected Completion

2019 (actual)

2019 (actual)

Publication

• O. Kornmann et al. Respiratory Medicine 161 (2020)

• Gessner C, et al. Respiratory Medicine (2020), doi:

• Abstract: D'Andrea et al, presented at ERS Sep-2019

https://doi.org/10.1016/j.rmed.2020.106021

135 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

Xolair ® - anti-IgE antibody

Study

NCT03369704 (CIGE025F1301)

Indication

Seasonal allergic rhinitis: Severe Japanese Cedar Pollinosis

Phase

Phase 3

Patients

337

Primary Outcome Measures

Mean nasal symptom score, consists of severity of sneezing, rhinorrhea and nasal congestion.

In addition to standard of care:

Arms/Intervention

• Omalizumab per approved allergic asthma dosing table for IgE/body weight combinations

• Placebo

Target Patients

Expected Completion

Publication

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

2019 (actual)

  • Late breaking abstract was published at AAAAI (American Association of Allergy, Asthma and Immunology) annual meeting, Feb 2019
  • Poster published at EAACI (the European Academy of Allergy and Clinical Immunology), Jun 2019
  • Oral presentations were made at JRS (Japanese Rhinologic Society), Oct 2019, and Asian Pacific Society of Respirology congress, Nov 2019
  • Article published in "Allergy in Otolaryngology" (in Japanese), Jan 2020, and in "JACI: In Practice", May 2020

136 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

Sandoz Biopharmaceuticals

Hyrimoz® - Biosimilar adalimumab

Study

NCT02744755 ADMYRA (GP17-302)

Indication

Immunology

Phase

Phase 3

Patients

353

Primary Outcome

Change in DAS28-CRP score from baseline to week 12 in

patients treated with GP2017 and patients treated with

Measures

Humira®

Arms/Intervention

• GP2017

• US licensed Humira® adalimumab

Target Patients

Patients with moderate to severe active rheumatoid arthritis

Expected Completion

2018 (actual)

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

Publication

• Wiland, P. et al., BioDrugs, Q2 2020

138 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

GP2411 - Biosimilar denosumab

Study

NCT03974100 (CGP24112301)

Indication

Osteoporosis

Phase

Phase 3

Patients

522

Primary Outcome

Percent change from baseline (%CfB) in lumbar spine Bone

Measures

Mineral Density

•

GP2411 60 mg /mL subcutaneous injection every 6

Arms/Intervention

months

•

Prolia® 60 mg /mL subcutaneous injection every 6

months

Target Patients

Expected Completion

Publication

Postmenopausal women with osteoporosis

2022

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

139 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

Global Health

KAF156 - Plasmodium Falciparum Inhibitor - PfCARL mediated

Study

NCT03167242 (CKAF156A2202)

Indication

Malaria

Phase

Phase 2

Patients

~500

Primary Outcome

PCR-corrected adequate clinical and parasitological

Measures

response (ACPR)

Arms/Intervention

• KAF156 and LUM-SDF (different combinations)

• Coartem

Target Patients

Adults and children with uncomplicated Plasmodium

Falciparum Malaria

Expected Completion

H2-2021

Publication

TBD

141 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

KAE609 - Plasmodium Falciparum Inhibitor - spiroindolone against PfATP4

Study

NCT03334747 (CKAE609A2202)

Indication

Malaria

Phase

Phase 2

Patients

186

Primary Outcome

CTCAE grades increase from baseline in alanine

aminotransferase (ALT) or aspartate aminotransferase

Measures

(AST)

Arms/Intervention

•

KAE609

•

Coartem

Target Patients

Adults with uncomplicated Plasmodium Falciparum malaria

Expected Completion

Q1-2020(actual)

Publication

TBD

142 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

Indication abbreviations

AD

Atopic Dermatitis

IPF

Idiopathic pulmonary fibrosis

AIH

Autoimmune hepatitis

mCRPC

Metastatic castration-resistant prostate cancer

aHUS

atypical Hemolytic Uremic Syndrome

MDR

Multi-drug resistant

ALL

Acute lymphoblastic leukemia

MDS

Myelodysplastic syndrome

ALS

Amyotrophic lateral sclerosis

MS

Multiple sclerosis

AMI

Acute myocardial infarction

nAMD

Neovascular (wet) age-related macular degeneration

AML

Acute myeloid leukemia

NASH

Non-alcoholic steatohepatitis

AS H2H

Ankylosing spondylitis head-to-head study versus adalimumab

nHCM

Non-obstructive hypertrophic cardiomyopathy

BC

Breast cancer

nr-axSpA

Non-radiographic axial spondyloarthritis

C3G

C3 glomerulopathy

NSCLC

Non-small cell lung cancer

CCF

Congestive cardiac failure

PDR

Proliferative diabetic retinopathy

CLL

Chronic lymphocytic leukemia

PEF

Preserved ejection fraction

CML

Chronic myeloid leukemia

PNH

Paroxysmal nocturnal haemoglobinuria

CRC

Colorectal cancer

PsA H2H

Psoriatic arthritis head-to-head study versus adalimumab

COPD

Chronic obstructive pulmonary disease

RCC

Renal cell carcinoma

COSP

Chronic ocular surface pain

PROS

PIK3CA related overgrowth spectrum

CSU

Chronic spontaneous urticaria

RA

Rheumatoid arthritis

CVRR-Lp(a)

Secondary prevention of cardiovascular events in patients with elevated levels of lipoprotein (a)

rMS

Relapsing multiple sclerosis

CVRR-LDLC

Secondary prevention of cardiovascular events in patients with elevated levels of LDLC

ROP

Retinopathy of prematurity

DME

Diabetic macular edema

RP

Retinitis pigmentosa

DLBCL

Diffuse large B-cell lymphoma refractory

RVO

Retinal vein occlusion

GCA

Giant cell arteritis

SAA

Severe aplastic anemia

GVHD

Graft-versus-host disease

SjS

Sjögren's syndrome

HCC

Hepatocellular carcinoma

SLE

Systemic lupus erythematosus

HFpEF

Chronic heart failure with preserved ejection fraction

SMA Type 1

Spinal muscular atrophy type 1 (IV formulation)

HF-rEF

Chronic heart failure with reduced ejection fraction

SMA Type 2/3

Spinal muscular atrophy type 2/3 (IT formulation)

HNSCC

Head and neck squamous cell carcinoma

SpA

Spondyloarthritis

HS

Hidradenitis suppurativa

SPMS

Secondary progressive multiple sclerosis

IgAN

IgA nephropathy

TNBC

Triple negative breast cancer

iMN

Membranous nephropathy

T1DM

Type 1 Diabetes melitus

143 Novartis Q2 Results | July 21, 2020 | Novartis Investor Presentation

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