Novartis Q2 2020 Results

Q2 2020 ResultsInvestor Presentation

July 21, 2020

Novartis AG

Investor Relations

Disclaimer

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

This presentation contains forward-looking statements within the meaning of the United States Private Securities Litigation Reform Act of 1995, that can generally be identified by words such as “potential,”

“expected,” “will,” “planned,” “pipeline,” “outlook,” or similar expressions, or by express or implied discussions regarding potential new products, potential new indications for existing products, 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.

Participants

Vas NarasimhanChief Executive Officer

Harry KirschChief Financial Officer

Marie-France TschudinPresident, Novartis Pharmaceuticals

Susanne SchaffertPresident, Novartis Oncology

John TsaiHead of Global Drug Development and CMO

Richard SaynorCEO, Sandoz

Shannon Thyme KlingerChief Legal Officer



Company overview

US approval NSCLC

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

5 simultaneous approvals in

Japan


Continuing operations1, % cc vs. PY

Strong operational performance Delivering on innovation

Strong H1 performance despite the impact of COVID-19H1 results more representative of performance as Q1 forward purchasing largely reversed in Q2

-1%

6%

19%

6%

Q2 2020

H1 2020

Net sales Core OpInc

Robust pandemic response

Human capital Health and safety of associates, patients, third parties2

Supply chain Operations remain stable with customer service levels at a record high

Clinical trialsDisruptions 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

For footnotes see slide 49

Growth drivers continuing strong momentum


Key growth driver sales H1 2020 Key growth drivers and launches,

as % of Innovative Medicines sales

212

73

159

96

190

48

170

86

108

139

60

73

371

7990

34

74

4870

68

26

31 10069

3562

60

360

225

97

169

169

147

118

102

86H1 2018H1 2017 H1 2019 H1 2020

Mayzent®

Tafinlar+Mekinist®

Adakveo®

Jakavi®

Beovu®

Piqray®

Xiidra®

Kisqali®

Lutathera®

Kymriah®

Zolgensma®

Ilaris®

Cosentyx®

Promacta®

Entresto®

Other1

nm – not meaningful 1. Includes Tasigna®, Xolair®, Aimovig® and Luxturna®

24%

30%

37%

47%

SalesUSD Million

Growth vs. PYUSD Million

Growth vs. PYcc

1,149 50%

375 nm

1,874 15%

825 28%

169 nm

153 nm

320 64%

211 106%

102 nm

737 19%

413 33%

628 20%

Q1 20 Q2 20

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

Received conditional approval with broad label1

Day One access program

Germany: agreements with 90% of sick funds

Early access in other countries

Fast uptake in 1st month of launch

Others Requests continue in pre-approved markets

(e.g. Middle East, Russia)

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


AVXS-101 IT

partial clinical

hold

Continued dialogue with FDA

Planning pre-BLA meeting

Submission expected 2021

Geographical

expansions

Approvals expected H2 2020 / early

2021: Switzerland (Fast Track),

Canada (Priority Review), Israel,

Australia, Argentina, South Korea,

Brazil

Manufacturing

expansion

Continued progress with Colorado

and North Carolina sites expected

operational 2021

Q2 highlights Regulatory and other milestones

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

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


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-19 negatively impacted hospital / pharmacy traffic lowering demand

US oral solids and dermatology decline and partnership terminations

% cc vs. PY H1 results more representative of performance as Q1

forward purchasing largely reversed in Q2

Net sales Core OpInc

-9%

1%

26%

1%

Q2 2020

H1 2020

Ofatumumab

Inclisiran

TQJ230

LNP023

Iscalimab

Ligelizumab

LNA043

Tropifexor

UNR844

CEE321

LOU064

VAY736

LMI070

QBW251

MBG453

Asciminib

Canakinumab

Spartalizumab177Lu-PSMA-617

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

In-market

growth drivers

Major launches

New indications

Novel assets

15 ongoing / upcoming major launches

80+ major submissions planned to 2022

50+ late stage programs1Cosentyx® HS

Cosentyx® GCA

Cosentyx® LP

Cosentyx® JIA

Cosentyx® LN

Entresto® post-AMI

Entresto® HFpEF

Beovu® DME

Beovu® RVO

Beovu® DR

Beovu® PDR

Ofatumumab pediatric

AVXS-101 IT

Xolair® food allergy

Alpelisib PROS

Piqray® TNBC

Piqray® HER2+ aBC

Piqray® ovarian cancer

Piqray® HNSCC

Kisqali® HR+/HER2- BC (adj)

Kymriah® FL

Kymriah® DLBCL

Jakavi® cGVHD

Jakavi® aGVHD

1. Ph3 / in registration

SELECT EXAMPLES


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

Advancing our late stage pipelineSelected 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®

w/ spartalizumab

mBRAF V600 +

melanoma

Ph3 readout on track 2020 Submission on track by end of 2020

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

2L (CANOPY-2) studies

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

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

2021

177Lu-PSMA-617 mCRPC VISION Ph3 trial: slower than expected event accumulation

rate; preparations ongoing for starting earlier line studies

Event driven trial; readout expected H1 2021

Entresto® HFpEF, post-AMI HFpEF filed

PARADISE-MI enrollment complete

FDA action expected by H1 2021

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)

ongoing

Ph3 enrollment on track to complete 2020;

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

Emerging pipeline assets continue to progressSelected assets


Asset MoA Indication Current status Next milestone

Ph

arm

aceu

ticals

LNP023 Factor B

inhibitor

PNH, IgAN, C3G,

iMN, aHUS

Ph2 parallel development for 3 rare renal diseases and

paroxysmal nocturnal haemoglobinuria (PNH)

Single PNH pivotal trial expected to start 2020;

C3 glomerulopathy, IgA nephropathy

Ph3 expected to start Q1 2021

Remibrutinib

(LOU064)

Bruton tyrosine kinase

inhibitor

CSU, Sjögren’s Ph2b study in CSU and adaptive Ph2 in Sjögren’s ongoing Phase 2b study in CSU expected to readout

2021

Iscalimab

(CFZ533)

Anti-CD40 monoclonal

antibody

Kidney Tx, Sjögren’s Ph2b studies in kidney transplant, Sjögren’s ongoing Anticipated regulatory submission for

kidney transplant 2023

TQJ230 Antisense

oligonucleotide

targeting LP(a)

CVRR-Lp(a) Ph3 outcomes study (HORIZON) initiated 2020 Ph3 outcomes readout expected 2024

On

co

log

y

MBG453 Anti-TIM-3 monoclonal

antibody

HR-MDS,

unfit AML

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

MDS-2)

Ph2 in unfit AML (combo HMA + venetoclax)

expected to start 2020

LXH254 B/C-RAF inhibitor m RAS/RAF NSCLC

and melanoma

Clinical studies ongoing, evaluating LXH254 in combination

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

Ph2 metastatic melanoma trial

expected to start 2020

TNO155 SHP2 inhibitor Solid tumors Broad combination strategy with multiple Ph1 combo

studies ongoing including spartalizumab, Kisqali®,

nazartinib, MRTX849

Continued enrollment in all 3 trials,

including recently started trial with

MRTX8491

1. Study sponsored by Mirati Therapeutics


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)

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

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)

Ethical

standards

Pricing

& access

Global

health

Corporate

citizenship

Progressing on our journey of building trust with society

Financial review

and 2020 guidance


% USD % cc % USD % cc

Net Sales 11,347 -4 -1 23,630 3 6

Core Operating income 3,669 1 6 7,846 14 19

Operating income 2,352 -12 -4 5,096 4 11

Net Income 1,867 -11 -4 4,040 2 9

Core EPS (USD) 1.36 1 6 2.92 15 19

EPS (USD) 0.82 -10 -3 1.77 3 11

Free Cash Flow 3,631 1 5,652 3

Change vs. PYContinuing operations

USD million

Q2

2020

H1

2020

Change vs. PY

Strong H1 performance despite impact of COVID-19


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 wel l 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.

2

2

2

1

2 2

Net sales

change vs. PY

Core operating

income

change vs. PY Core margin

Core margin

change vs. PY

Net sales

change vs. PY

Core operating

income

change vs. PY Core margin

Core margin

change vs. PY

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

Q2 2020 H1 2020

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


Continuing operations1


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.

Jun 28Apr 30Mar 31Feb 29 May 31

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


100%

125%

50%

75%

Growth Drivers

(excl. Cosentyx®)2

Recent Launches1

Beovu®, Lucentis® & Xiidra®

Cosentyx®

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®

IM Weekly Sales Evolution4 weeks rolling, indexed to Q4 weekly sales avg.

Impact of Beovu

launch pre-ASRS

communication

Beginning of COVID-19 lockdowns

across US and Europe

Q2 Cosentyx® (US data)

Recovered in June to Q4 2019 levels

Increasing market share in dermatology and

rheumatology

Mature Ophthalmology3

Ophthalmology, dermatology and new patient starts are more impacted

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


Key drivers of core operating income (continuing operations)vs. PY (cc)

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-19 related negative impact

on Lucentis® and mature ophtha

+ Innovative Medicines growth

drivers, and launches1

+ Productivity

− Increased Gx erosion2

− Increased investments in pre-

launch activities and launches

− Lapping Xiidra® acquisition

H2 2020

1. Including Zolgensma®, Mayzent®, Aimovig®, Xiidra®, Piqray® 2. Including Afinitor ®, Exjade®, Sandostatin® LAR and Ophtha brands

ILLUSTRATIVE

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

Key drivers vs. PY:

+ Higher operating income

(adjusted for non-cash items)

− Lower divestment proceeds

H1 2020 free cash flow increased to USD 5.7bn


5.7

H1 2019

5.5

H1 2020

+3%

Continuing operations1 free cash flow2

USD billion


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

Currency impact vs. PY%pts, assuming mid-July exchange rates prevail in 2020

Expected currency impact for full year 2020

SimulationActual

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

2019

FX impact on Net sales FX impact on Core operating income

-3-2

-3-5

-6-5

Q4FYFY Q1 FYQ4

-4

Q3Q2 Q1 Q2 Q3 FY

-1 to -2-3

0

-3

0

0 to -1

2019 2020 2020

20

H1 performance solidAccelerating our digital transformation


Pharmaceuticals net sales USD billion, growth in % cc

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®

H1 sales grew +8% as growth drivers continued momentum

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

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

3.3

0.1

7.8 6.9

H1 2019

0.8

4.0

H1 2020

11.2 11.7

+8%

Mature products3Growth drivers1 Recent launches2

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


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®

534 614

324330

Q2 2020Q2 2019

858944

+12%

Ex-US

US

Sales evolutionUSD million, % cc 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

Sales now showing recovery towards pre-COVID dynamics

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

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

Further approvals

Launching nr-axSpA in US / EU (1.7m potential patients)

AS approved in China

PedPsO positive CHMP opinion

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

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

Strong dermatology position

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

Ready to accelerate in rheumatology 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

Potential to expand into multiple indications

For footnotes see slide 49

Strong Entresto® performance driven by underlying demand


221308

272

Q2 2019 Q2 2020

421

580

200

+40%

Ex-US

US

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

Sales evolutionUSD million, % cc

Beovu® US label updatedNew data reinforces need for fluid control in wAMD


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

Beovu® benefit-risk profile remains positive

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, continuing launches

Updated labels in US, JP, CH,

AUS3, adding clarifying safety

language to warnings &

precautions4

Beovu® now approved in 30

countries, launches ongoing

New post-hoc data confirms lower levels of retinal fluid are associated with better BCVA1

Beovu® better in reducing retinal fluid (IRF/SRF)

Adjusted mean BCVA change Wk12-96 (ETDRS letters)

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

27

Potential for broad and early high efficacy in RMS 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

Powerful sustained efficacy

Favorable safety

Precise and targeted B-cell therapy

Flexibility through at home self-administration


✓ Highly customized approach

for early adopters

✓ Flexible and customer-centric

onboarding process

Launch leverages established position and deep customer insights

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

✓ Rapid and broad availability of

Ofatumumab upon approval

Inclisiran launch preparations are progressing –FDA action date Dec 2020


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

Addressing non-clinical barriers to uptakeAdvancing 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

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

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

30

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)

0.4

0.2

0.9

0.4

1.1

Oncology net sales USD billion, % cc

0.2

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.

2.2

0.5

1.8

4.6

H1 2019

0.9

4.1

H1 2020

6.9 7.2

+6%

Base business3Recent launches1 Growth drivers2


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


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

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

(MONALEESA-2) expected 2021

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

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

Net sales USD m, % cc

111

159

Q2 2019 Q2 2020

+49% 202

320

H1 2019 H1 2020

+64%

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

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


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

FDA granted RMAT1 designation to Kymriah® for r/r follicular

lymphoma; submission expected 2021

Net sales USD m, % cc

58

118

Q2 2019 Q2 2020

+103%

103

211

H1 2019 H1 2020

+106%

1. Regenerative Medicine Advanced Therapy

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


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)

High unmet medical need and strong clinical profile 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

Omni-channel launch amid pandemic conditions

Monotherapy studies: Phase 3, brain

metastases, tumor agnostic

Moving into combinations:

PD-L1 high 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

Development plan tomaximize potential

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

2020 catalysts: Maintaining long-term momentum

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

Potential catalysts Selected examples

Major approvals1 Ofatumumab (OMB157)

Relapsing MS

Tabrecta™(capmatinib)5

NSCLC

Inclisiran (KJX839)

Hyperlipidemia (US)

Enerzair® / Atectura®

Asthma

Cosentyx®

nr-AxSpA

Major

submissions2

Inclisiran (KJX839)

Hyperlipidemia (EU)

Alpelisib (BYL719)

PROS

Entresto®

HFpEF (US)

Spartalizumab (PDR001) combo

Metastatic melanoma

Major readouts3

(Phase 3)

Jakavi®

Chronic GvHD

Beovu®

DME

Canakinumab

1st line NSCLC (IA)

Asciminib (ABL001)

Chronic Myeloid Leukemia

Entresto®

Post-acute MI (IA)

Phase 3 starts TQJ2306

CVRR

MBG453

MDS

LNP023

PNH

Alpelisib (BYL719)

Multiple indications4

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


Appendix

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


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

-15.9

-26.5-7.0

-9.9

-0.1

M&A transactions1 Treasury share

transactions, net

5.7

Dividends

0.7

Dec 31, 2019 OthersFree Cash Flow Jun 30, 2020

-10.6

(USD bn)

H1 2020 H2 2020

Regulatory

decisions and

opinions

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

mutation (EU)✓

3 Entresto® HFpEF (US) H1 2021

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) ✓

Major

expected

submissions

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)

and Tafinlar® & Mekinist®Metastatic melanoma (US/EU)

177Lu-PSMA-617 mCRPC (US) 2021

Major

expected trial

readouts*

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 ✕ Missed

2020 expected pipeline milestones

*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


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


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

Spectr

um

of in

flam

mato

ry e

vents 4% IOI4.4% 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% Vasculitis3.3% 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% Occlusion2.1% 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

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

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

~0Difference in

overall vision lossOverall vision loss similar across Beovu (7.4%)

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

Our pipeline projects at a glance


Biosimilars not included as we only disclose biosimilars that have moved into Phase 3 CRM: Cardiovascular, Renal & Metabolism. IHD: Immunology, Hepatology & Dermatology. NS: NeuroScience.

Phase 1/2 Phase 3 Registration Total

ONCOLOGY 51 22 2 75

PHARMACEUTICALS 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

Novartis submission scheduleNew Medical Entities: Lead and supplementary indications


2020 2021 2022 2023 ≥2024

asciminibABL001

CML 3L

Lead

MBG453HR-MDS

Lead

ligelizumabQGE031

Chronic urticaria

Lead ECF843Dry eye

Lead

remibrutinibLOU064

Chronic spontaneous urticaria

Lead

LNP023PNH

Lead

SAF312COSP

Lead

CPK850RP

Lead

LMI070SMA

Lead

MIJ821Depression

Lead

AVXS-201OAV201

Rett syndrome

Lead

LNA043Osteoarthritis

Lead

tropifexorLJN452

NASH

Lead

tropifexor&cenicrivirocLJC242

NASH

Lead

177Lu-NeoB177Lu-NeoB

Multiple Solid Tumors

Lead

VPM0871st line CRC / 1st line RCC

Lead

CEE321Atopic Dermatitis

Lead

LXE408Visceral leishmaniasis

Lead

ganaplacideKAF156

Malaria uncomplicated

Lead

TQJ230CVRR-Lp(a)

Lead

cipargaminKAE609

Malaria severe

Lead

UNR844Presbyopia

Lead QBW251COPD

Lead

CSJ117Asthma

LeadspartalizumabPDR001

m BRAF V600+ melanoma (+Taf/Mek)

Lead

ianalumabVAY736

AIH

Lead

LE

AD

IN

DIC

AT

ION

S

iscalimabCFZ533

Renal Tx

Lead

LAG525Solid Tumors

Lead

177Lu-PSMA-R2177Lu-PSMA-R2

Prostate cancer

Lead

177Lu-PSMA-617177Lu-PSMA-617

mCRPC 3L

Lead

ianalumabVAY736

pSjS

LCM

remibrutinibLOU064

SjS

LCM

ofatumumabOMB157

Ped MS

LCMiscalimabCFZ533

SjS

LCM

tropifexorLJN452

NASH (combos)

LCM

MBG453Unfit AML

LCM

crizanlizumabSEG101

Sickle cell anaemia with crisis ped

LCM LNP023iMN

LCM

cipargaminKAE609

Malaria uncomplicated

LCM

LNP023C3G

LCM

LNP023IgAN

LCM

canakinumabACZ885

Adjuvant NSCLC

LCM spartalizumabPDR001

Malignant melanoma (combo)

LCMcanakinumabACZ885

NSCLC 2L

LCM

canakinumabACZ885

NSCLC 1L

LCM

NE

W I

ND

ICA

TIO

NS

capmatinibINC280

Solid tumors

LCM

MBG453Maintenance for MRD+ AML

LCM

inclisiranKJX839

CVRR-LDLC

LCM

LNP023aHUS

LCM

iscalimabCFZ533

Liver Tx

Lead

Novartis submission scheduleSupplementary indications for existing brands

2020 2021 2022 2023 ≥2024

AVXS-101onasemno-gene abepar-vovec, OAV101

SMA IT

LCM

alpelisib, BYL719PROS

LCM

Entrestosacubitril/valsartan, LCZ696

Post-AMI

LCM

Xolairomalizumab, IGE025

Auto-injector

LCM

Xolairomalizumab, IGE025

Food allergy

LCMBeovubrolucizumab, RTH258

DME

LCM

Jakaviruxolitinib, INC424

Acute GVHD

LCM

Promactaeltrombopag, ETB115

Food effect free formulation

LCM


Chronic GVHD

LCM

Kymriahtisagenlecleucel-T, CTL019

r/r DLBCL 1st relapse

LCM


r/r Follicular lymphoma

LCM

Tafinlardabrafenib, DRB436

HGG/LGG - Pediatrics

LCM

Cosentyxsecukinumab, AIN457

AS H2H

LCM


Hidradenitis suppurativa

LCM


SpA IVIV

LCM

Kisqaliribociclib, LEE011

HR+/HER2- BC (adj)

LCM

Piqrayalpelisib, BYL719

Ovarian cancer

LCM

Promactaeltrombopag, ETB115

Radiation sickness syndrome

LCM

Beovubrolucizumab, RTH258

RVO

LCM

Tafinlardabrafenib, DRB436

Tyroid cancer

LCM

Beovubrolucizumab, RTH258

Diabetic retinopathy

LCM


TNBC

LCM


HER2+ adv BC

LCM Cosentyxsecukinumab, AIN457

Lupus Nephritis

LCMJakaviruxolitinib, INC424

Myelofibrosis (combination)

LCM


Pediatrics Acute GVHD

LCM


GCA

LCM


1L high risk ALL, pediatrics & young adults

LCM


Pediatrics Chronic GVHD

LCM


HNSCC 2/3L

LCM

Kymriahtisagenlecleucel-T, CL019

r/r DLBCL (+ pembro)

LCM

LCMCosentyxsecukinumab, AIN457

Lichen Planus

LCM Mayzentsiponimod, BAF312

Pediatric MS

LCM


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

Entresto EUa

sacubitril/valsartan, LCZ696

Pediatric HF

LCM

Adakveocrizanlizumab, SEG101

Sickle cell anaemia new formulations

LCM

denosumabGP2411

anti RANKL mAb

BioS

Coartemartemether + lumefantrine, CCA566

Malaria uncomplicated, <5kg patients

LCM

Lutathera177Lu-oxodotreotideb)

GEP-NET 1L G3

LCM Aimovigerenumab, AMG334

Pediatric Migraine

LCM

Novartis pipeline in registration


Oncology

Code Name Mechanism Indication(s)

BYL719 Piqray PI3Kα inhibitorPIK3CA mutant HR+, HER2 (-) postmenopausal adv BC 2nd line

(+fulv)

SEG101 Adakveo® P-selectin Inhibitor Sickle cell disease

Immunology, Hepatology, Dermatology


AIN457 Cosentyx IL17A Inhibitor Ped Psoriasis 2ml Auto-injector

Neuroscience


OMB157 ofatumumab CD20 Antagonist r MS

Respiratory Disease


IGE025 Xolair IgE Inhibitor Nasal polyps

Global Health


LAM320 Lamprene® SMPD1 Inhibitor Tuberculosisa)

3 lead indicationsLead indication

a) WHO Pre-Qualification

Cardiovascular, Renal, Metabolism


KJX839 inclisiran siRNA (regulation of LDL-C) Hyperlipidemia

LCZ696 Entresto Angiotensin II Receptor

Neprilysin Inhibitor (ARNI)

HFpEF

Novartis pipeline in Phase 3


Oncology


177Lu-PSMA-617 177Lu-PSMA-617 Targeted Radioligand Therapy mCRPC

177Lu-

oxodotreotide 3) Lutathera® Targeted Radioligand Therapy GEP-NET 1L G3

ABL001 asciminib BCR-ABL Inhibitor CML 3L

ACZ885 canakinumab IL-1b Inhibitor NSCLC 1L NSCLC 2LAdjuvant

NSCLC

BYL719 Piqray® PI3Kα inhibitor HER2+ adv BC TNBC HNSCC 2/3L Ovarian cancer

CTL019 Kymriah CD19 CART r/r Follicular

lymphoma

1L high risk

ALL, pediatrics

and young

adults

r/r DLBCL 1st

relapse

ETB115 Promacta® Thrombopoietin receptor (TPO-R)

Agonist

Radiation sickness syndrome Food effect free formulation

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



AIN457 Cosentyx IL17A Inhibitor Lupus Nephritis Hidradenitis

suppurativa

AS H2H SpA IVIV

ACZ885 canakinumab IL-1b Inhibitor COVID-19 induced respiratory disease

QGE031 ligelizumab IgE Inhibitor Chronic urticaria



KJX839 inclisiran siRNA (regulation of LDL-C) CVRR-LDLC

LCZ696 Entresto® Angiotensin II Receptor Neprilysin Inhibitor

(ARNI)

Post-AMI Pediatric HF 2)

TQJ230 TQJ230 Anti-Apo(a) ASO targeting Lp(a) CVRR-Lp(a)

Biosimilars


GP2411 denosumab anti RANKL mAb Denosumab BioS


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


AMG334 Aimovig® CGRPR antagonist Ped Migraine

BAF312 Mayzent® S1P1 Modulator Ped MS

OAV101 AVXS-101 Gene Therapy, Survival motor

neuron (SMN1) gene

SMA IT 1)

OMB157 ofatumumab CD20 Antagonist Ped MS

Global Health


COA566 Coartem® - Malaria uncomplicated, <5kg patients

Respiratory Disease


IGE025 omalizumab IgE Inhibitor Food allergy Auto-injector

INC424 Jakavi® JAK1 Inhibitor COVID-19 related pneumonia

Ophthalmology


RTH258 Beovu® VEGF Inhibitor Diabetic retinopathy RVO DME

Novartis pipeline in Phase 2


Oncology


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



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


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

Neuroscience


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


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



CFZ533 iscalimab CD40 Inhibitor Lupus Nephritis T1DM

LCZ696 Entresto® Angiotensin II Receptor

Neprilysin Inhibitor (ARNI)

nHCM

LMB763 nidufexor FXR Agonist Diabetic Nephropathy

LNP023 LNP023 CFB Inhibitor PNH IgAN C3G iMN aHUS

LTW980 LTW980 - Hypertriglyceridemia

Global Health


AFQ056 AFQ056 mGluR5 Antagonist Addiction

KAE609 cipargamin PfATP4 inhibitor Malaria severe Malaria uncomplicated

KAF156 ganaplacide - Malaria uncomplicated

LXE408 LXE408 Protozoan Inhibitor Visceral leishmaniasis


1) Approved in US & JP

Novartis pipeline in Phase 1 (1 of 2)


Lead indication

Oncology


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

37 lead indications

Novartis pipeline in Phase 1 (2 of 2)


Lead indication

37 lead indications



CEE321 CEE321 Pan JAK Inhibitor AD

DFV890 DFV890 - Anti-inflammatory therapy

FIA586 FIA586 - NASH

LRX712 LRX712 - Osteoarthritis

MAS825 MAS825 - Inflammatory diseases

MHS552 MHS552 - Autoimmune Indications

MHV370 MHV370 - SjS SLE

Neuroscience


OAV201 AVXS-201 MECP2 gene therapy Rett syndrome

Respiratory Disease


CMK389 CMK389 IL-18 Inhibitor Sarcoidosis

LTP001 LTP001 - Respiratory Diseases



HSY244 HSY244 - Atrial fibrillation

MBL949 MBL949 - Diabetes

Global Health


KAF156 ganaplacide - Malaria prophylaxis

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

References


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

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-19 Good 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

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)

52

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

Measures

Part 1: Pharmacodynamics and pharmacokinetics of

sacubitril/valsartan LCZ696 analytes

Part 2: Efficacy and safety compared with enalapril

Number of participants with Adverse Events (AEs) and

Serious Adverse Events (SAEs)

Arms/Intervention

• 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

1mg/ml) and adult formulation (2.5, 5, 10 mg bid);

Sacubitril/valsartan (LCZ696)/placebo: Ped. formulation

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

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

formulation (50, 100, 200 mg bid)

• Single arm, open label sacubitril/valsartan (pediatric

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

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

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

Target Patients

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

heart failure due to systemic left ventricle systolic

dysfunction

Pediatric patients with heart failure due to systemic left

ventricle systolic dysfunction who have completed study

CLCZ696B2319

Expected Completion

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

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

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

systemic left ventricular systolic dysfunction in children aged

1 year and older)

2022

Publication TBD TBD


53

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

Measures

Change from baseline in the CogState Global Cognitive

Composite Score (GCCS)

Time to the first occurrence of the composite endpoint -

either cardiovascular (CV) death or heart failure (HF)

hospitalization

Arms/Intervention

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

placebo of valsartan

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

for sacubitril/valsartan

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

of enalapril

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

sacubitril/valsartan

Target PatientsPatients with chronic heart failure with preserved ejection

fraction

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

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



54

Study NCT01920711 PARAGON-HF (CLCZ696D2301) NCT03066804 PARALLAX (CLCZ696D2302)

Indication Heart failure, preserved ejection fraction Heart failure, preserved ejection fraction


Patients 4,822 2,572

Primary Outcome

Measures

Cumulative number of primary composite events of

cardiovascular (CV) death and total (first and recurrent) HF

hospitalizations

Change in NT-proBNP from baseline to week 12

and change in 6 minute walk distance (6MWD) from

baseline to Week 24

Arms/Intervention

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

mg bid

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

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

matching placebo

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

placebo

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

placebo

Target PatientsHeart failure patients (NYHA Class II-IV) with preserved

ejection fraction

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

ejection fraction

Expected Completion 2019 (actual) 2019 (actual)

Publication

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

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

• Mar-2020: Published (NTproBNP, putative placebo

analysis);

• Jun-2020: Submitted (renal outcomes, cognitive

function);

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

differences, win ratio, adjudicated vs reported endpts;

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

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

ESC-HF)

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

latebreaker; Publication EHJ Q3-2020.

• Q3-2020 – Planned: Baseline data publication



55

Study NCT03909295 (CLCZ696D1301E1 – extension study) NCT02924727 PARADISE-MI (CLCZ696G2301)

Indication Heart failure chronic Post-acute myocardial infarction


Patients 52 5,670

Primary Outcome

Measures

Number of participants with Adverse Events (AEs) and

Serious Adverse Events (SAEs)

Time to the first occurrence of a confirmed composite

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

hospitalization, or outpatient heart failure)

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

tablets

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

of ramipril/valsartan

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

sacubitril/valsartan / placebo for valsartan

Target Patients

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

preserved ejection fraction after CLCZ696D2301

(PARAGON-HF)

Post-AMI patients with evidence of LV systolic dysfunction

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

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



56

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

Indication

Hypercholesterolemia inc. Atherosclerotic Cardiovascular

Disease (ASCVD) and ASCVD risk equivalents

Heterozygous Familial Hypercholesterolaemia (HeFH)

Hypercholesterolemia inc. Heterozygous Familial

Hypercholesterolaemia (HeFH)


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

Primary Outcome

Measures

LDL-C reduction at Day 210 for Group 1 subjects

Changes in other lipids and lipoproteins and reduction of

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

goal ; longer term exposure and safety.

A composite of major adverse cardiovascular events,

defined as:

• Coronary heart disease (CHD) death;

• Myocardial infarction;

• Fatal or non-fatal ischaemic stroke; or

• Urgent coronary revascularization procedure

Arms/Intervention

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

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

planned duration of 4 years

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

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

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

duration of 4 years.

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

subcutaneous injection on the day of randomization, at 3

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

duration of about 5 years

Arm 2: matching placebo (given bysubcutaneous injection

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

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

Target Patients

Patients with HeFH or pre-existing atherosclerotic

cardiovascular disease (ASCVD) on background statin +/-

ezetimibe therapy

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

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

class CV outcomes (25% benefit).

Expected Completion 2022 2025

Publication TBD TBD

KJX839 – siRNA (regulation of LDL-C)


57

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

IndicationHypercholesterolemia inc. Homozygous Familial

Hypercholesterolemia (HoFH)

Hypercholesterolemia inc. Heterozygous Familial

Hypercholesterolaemia (HeFH) and Homozygous Familial

Hypercholesterolemia (HoFH)


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

Primary Outcome

Measures

LDL-C reduction at Day 150

Changes in PCSK9, other lipids and lipoproteins and

reduction of LDL-C of more than 20%

The effect of inclisiran treatment on the proportion of

subjects achieving prespecified low density lipoprotein

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

tolerability profile of long term use of inclisiran

Arms/Intervention

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

on Day 1 and Day 90

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

randomized to the placebo group only, inclisiran on Day

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

years for all patients

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

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

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

planned duation of 3 years

Target Patients Patients with HoFH

Patients with HeFH or pre-existing atherosclerotic

cardiovascular disease (ASCVD) on background statin +/-

ezetimibe therapy and risk equivalents (patients from

ORION 9, 10 & 11 studies)

Expected Completion Primary: Q3-2020; Final: H2-2021 2023

Publication TBD TBD

KJX839 – siRNA (regulation of LDL-C)


58

Study NCT03373461 (CLNP023X2203) NCT04154787 (CLNP023D12201)

Indication IgA nephropathy (IgAN) Idiopathic membranous nephropathy (iMN)


Patients 146 72

Primary Outcome

Measures

Change from baseline of log transformed UPCR derived from

the 24h urine collections at Baseline and Day 90

Change from baseline of UPCR derived from 24hr urine

collections at Baseline and Week 24

Arms/Intervention

• Placebo

• LNP023 Dose 1 – 10mg bid



• LNP023 Dose 4 – 100mg bid (Part 2 only)

• LNP023 Dose – 200mg bid

• LNP023 Dose – 50mg bid

• Rituximab

Target Patients Patients with biopsy-verified IgA nephropathy

Patients with biopsy proven iMN who are at high risk of

disease progression defined on the basis of antibody anti-

PLA2R titre and proteinuria

Expected Completion H2-2021 2022

Publication TBD TBD

LNP023 – Factor B inhibition of the complement alternative pathway


59

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

Increasing doses of LNP023 up to 200mg bid:

• Cohort A: Native kidney patients

• Cohort B: Kidney transplanted patients

• Open-label LNP023 200mg bid

Target Patients Patients with C3 glomerulopathy Patients with C3 glomerulopathy


Publication TBD TBD



60

Study NCT03439839 (CLNP023X2201) NCT03896152 (CLNP023X2204)

Indication Paroxysmal nocturnal hemoglobinuria (PNH) Paroxysmal nocturnal hemoglobinuria (PNH)


Patients 15 10

Primary Outcome

Measures

Reduction of chronic hemolysis, based on LDH level at

Week 13

Reduction of PNH associated hemolysis, based on

percentage of patients with 60% reduction in LDH or LDH

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

Arms/Intervention

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

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

period

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

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

extension period. Dose may be increased D15 onwards to

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

least 60% compared to Baseline.

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

8wk treatment LNP023 100mg bid and 2yr extension

LNP023 100mg bid

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

8wk treatment LNP023 200mg bid and 2yr extension

LNP023 200mg bid

Target Patients

Patients with PNH, showing signs of active hemolysis

despite 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 CompletionPrimary endpoint: Q2-2020

Extension period: 2023

Primary endpoint: Q2-2020

Extension period: 2022

Publication In preparation (PoC study) TBD



61

Study NCT04023552 Lp(a)HORIZON (CTQJ230A12301)

Indication Cardiovascular risk reduction

Phase Phase 3

Patients 7,680

Primary Outcome

Measures

Time to the first occurrence of MACE (cardiovascular death,

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

vascularization)

Arms/InterventionTQJ230 80 mg injected monthly subcutaneously or

matched placebo

Target PatientsPatients 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

TQJ230 – Antisense oligonucleotide targeting apolipoprotein(a) mRNA


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

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

months post-transplantation and post conversion (for

maintenance cohort)

Change in EULAR Sjögren’s syndrome Disease Activity

Index (ESSDAI) score and EULAR Sjögren’s syndrome

Patient Reported Index (ESSPRI) score

Arms/Intervention

• Two cohorts: de novo TX and maintenance

• Test Arms: CFZ533 + MMF + corticosteroids

• Standard of Care: TAC + MMF + corticosteroids

• Three dose arms of CFZ533

• Placebo

Target Patients Kidney transplant recipients Patients with Sjögren's syndrome


Publication Manuscript of PoC trial to be submitted in Q1-2020Manuscript of PoC trial published in The Lancet-

Rheumatology January 23, 2020


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

Measures

Proportion of patients with composite event (BPAR, Graft

Loss or Death) over 12 months

Arms/Intervention

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

• CFZ533 dose A + MMF + Corticosteroids

• CFZ533 dose B + MMF + Corticosteroids

Target Patients Liver transplant recipients


Publication TBD


Cosentyx® - Anti IL-17

65

Study NCT03504852 (CAIN457A2324) NCT03589885 MATURE (CAIN457A2325)

Indication Psoriasis Psoriasis

Phase Phase 3B Phase 3

Patients 331 122

Primary Outcome

Measures

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

16 weeks of treatment

PASI 75 response and IGA mod 2011 0 or 1 response after

12 weeks of treatment

Arms/Intervention

• Secukinumab 300 mg every 2 weeks after weekly doses

till Week 4

• Secukinumab 300 mg every 4 weeks after weekly doses

till Week 4

• Secukinumab 2 mL (300 mg) auto-injector

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

• Placebo 2 mL auto-injector

• Placebo 2 x 1 mL prefilled syringe

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

Expected Completion Q3-2020 Q4-2020

Publication TBD TBD



66

Study NCT02471144 (CAIN457A2310) NCT03668613 (CAIN457A2311)

Indication Psoriasis Psoriasis


Patients 162 84

Primary Outcome

Measures

Psoriasis Area and Severity Index (PASI) 75 response and

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

week 12



week 12

Arms/Intervention

• Secukinumab low dose

• Secukinumab high dose

• Placebo

• Etanercept (comparator)

• Secukinumab low dose

• Secukinumab high dose

Target PatientsPatients from 6 to less than 18 years of age with severe

chronic plaque psoriasis

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

severe plaque psoriasis


Publication TBD TBD



67

Study NCT03066609 (CAIN457A2318)

Indication Psoriasis

Phase Phase 3

Patients 543

Primary Outcome

Measures



week 12

Arms/Intervention

• Secukinumab 300 mg


• Placebo

Target PatientsPatients with moderate to severe chronic plaque-type

psoriasis with or without psoriatic arthritis comorbidity

Expected Completion Q1-2019 (actual)

Publication

• Week 16 results: Poster presented at: 2019 American

Academy of Dermatology (AAD) Annual Meeting,

• March 1–5, 2019, Washington, D.C.

• 52-week results: Poster at EADV 2019, Madrid 9-13

October, 2019

• Manuscript Publication under assessment



68

Study NCT03031782 (CAIN457F2304) NCT03769168 (CAIN457F2304E1 – extension study)

Indication Psoriatic arthritis Psoriatic arthritis


Patients 80 64

Primary Outcome

MeasuresTime to 33 flares Number of participants with JIA ACR30 response

Arms/Intervention• Secukinumab (pre-filled syringe) 75 mg

• Placebo

• Secukinumab 75 mg/0.5 ml

• Secukinumab 150 mg/1.0 ml

Target PatientsJuvenile idiopathic arthritis subtypes of psoriatic and

enthesitis-related arthritis

Patients with juvenile idiopathic arthritis subtypes of juvenile

psoriatic arthritis and enthesitis related arthritis


Publication TBD TBD



69

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

Indication Psoriatic arthritis Ankylosing spondylitis


Patients 460 219

Primary Outcome

Measures

Proportion of subjects that have a positive clinical response

to treatment (individual improvement) in disease activity

according to ACR20 (or ACR50 or ACR 70)

Assessment of SpondyloArthritis International Society /

ASAS 20 response

Arms/Intervention• Secukinumab 75 mg




• Placebo

Target Patients Patients with active psoriatic arthritis Patients with active ankylosing spondylitis


Publication

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

Rheumatol. 2016; 68 (suppl 10)

• 3 years results: Manuscript published in September

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

doi:10.1136/rmdopen-2018-000723)

• 5 year results: Published in ACR Open Rheumatology.

November 14, 2019

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

Engl J Med 2015;373:2534–48

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

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

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

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

FRI0379. Annals of the Rheumatic Diseases

2019;78:873.

• 5 year results; Published in Lancet Rheumatology, June

2020



70

Study NCT01752634 FUTURE 2 (CAIN457F2312) NCT02008916 MEASURE 3 (CAIN457F2314)

Indication Psoriatic arthritis Ankylosing spondylitis


Patients 399 222

Primary Outcome

Measures

Proportion of subjects achieving American College of

Rheumatology 20 (ACR20) response criteria

Assessment of Spondyloarthritis International Society

criteria / ASAS 20 response

Arms/Intervention

• Secukinumab (AIN457) 150 mg s.c.



• Placebo s.c.

• Secukinumab 10 mg/kg / 300 mg

• Secukinumab 10 mg/kg / 150 mg

• Placebo

Target Patients Patients with active psoriatic arthritis Patients with active ankylosing spondylitis


Publication

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

2015;386:1137–46

• 2 years results: McInnes et al, Rheumatology

2017;56:1993-2003

• 5 years: published Lancet Rheumatology in March 2020

• 16 weeks results: PANLAR congress in Apr-2016

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

Therapy 2017

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

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

PANLAR April 2019

• 3 year (EOS) manuscript published in ACR Open

Rheumatology in January 2020



71

Study NCT02745080 EXCEED (CAIN457F2366)

Indication Psoriatic arthritis

Phase Phase 3

Patients 850

Primary Outcome

MeasuresAmerican College of Rheumatology 20 (ACR20) response

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



72

Study NCT02696031 PREVENT (CAIN457H2315) NCT03259074 SURPASS (CAIN457K2340)

Indication Non-radiographic axial spondyloarthritis Ankylosing spondylitis


Patients 555 837

Primary Outcome

Measures

The proportion of participants who achieved an ASAS 40

response (Assessment of SpondyloArthritis International

Society criteria);

No radiographic structural progression as measured by

modified Stoke Ankylosing Spondylitis Spine Score

(mSASSS)

Arms/Intervention

• Secukinumab 150 mg load

• Secukinumab 150 mg no load

• Placebo

• Secukinumab 150/300 mg

• Adalimumab biosimilar 40 mg

Target Patients Patients with non-radiographic axial spondyloarthritis Patients with active ankylosing spondylitis

Expected Completion Week 52: Q3-2019 (actual); Final: H1-2021 2022

Publication

• Abstract (16 week results) presented at ACR 2019

• Abstract (52 week results) presented at EULAR 2020

• Manuscript submitted in Mar-2020 (awaiting decision)

• Study design manuscript published. Baraliakos et al.

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



73

Study NCT03713619 SUNSHINE (CAIN457M2301) NCT04179175 (CAIN457M2301E1)

Indication Hidradenitis Suppurativa (HS) Hidradenitis Suppurativa (HS)


Patients 471 745

Primary Outcome

Measures

Proportion of participants with Hidradenitis Suppurativa

clinical response (HiSCR)

Proportion of patients with Hidradenitis Suppurativa Clinical

Response (HiSCR)

Arms/Intervention

• Secukinumab 300 mg every 2 weeks


• Placebo (every 2 weeks)




Target Patients Patients with moderate to severe Hidradenitis Suppurativa

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

• Preliminary results in AAD (most likely) in 2022Study design SHSA 2020



74

Study NCT03713632 SUNRISE (CAIN457M2302)

Indication Hidradenitis Suppurativa (HS)

Phase Phase 3

Patients 471

Primary Outcome

Measures

Proportion of patients with Hidradenitis Suppurativa Clinical

Response (HiSCR)

Arms/Intervention





Target Patients Subjects with moderate to severe Hidradenitis Suppurativa

Expected Completion Weak 16 DBL: H2-2021; Final: 2022

Publication• Study design SHSA 2020

• Preliminary results in EADV (most likely) in 2021



75

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

Indication Axial spondyloarthritis Axial spondyloarthritis


Patients 500 380

Primary Outcome

Measures

The proportion of subjects achieving an ASAS40

(Assessment of SpondyloArthritis International Society

criteria) response

The proportion of subjects achieving American College of

Rheumatology 50 (ACR50) response criteria

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

• Placebo intravenous (i.v.) regimen

• Secukinumab intravenous (i.v.) regimen

• Placebo intravenous (i.v.) regimen

Target Patients Patients with active axial spondyloarthritisPatients with active psoriatic arthritis (PsA) despite current

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


Publication TBD TBD


Ilaris® - Anti IL-1β

76

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

Proportion of patients in clinical remission on canakinumab

who are able to remain in remission following canakinumab

dose tapering (reduced canakinumab dose or prolonged

canakinumab dosing interval)

Number of patients with clinical response; Clinical response

is defined as survival without ever requiring invasive

mechanical ventilation from day 3 to day 29

Arms/Intervention• Canakinumab dose reduction

• Canakinumab dose interval prolongation

• Canakinumab

• Placebo

Target PatientsPatients with Systemic Juvenile Idiopathic Arthritis (SJIA)

(Pediatric)Patients With COVID-19-induced pneumonia

Expected Completion 2018 (actual) Q4-2020

Publication

• Remission & flexible dosing – presented at ISSAID & EULAR in Q2-2019

• Planned manuscript in 2019: Remission & flexible dosing submitted in Q4-2019

Planned manuscript submission in Q4-2020


LJN452 - FXR Agonist

77

Study NCT02855164 (CLJN452A2202) NCT04065841 ELIVATE (CLJN452D12201C)

Indication Non-alcoholic steatohepatitis (NASH) Non-alcoholic steatohepatitis (NASH)


Patients 345 210

Primary Outcome

Measures

Adverse event profile of different doses; determine the dose

relationship of LJN452 on markers of hepatic inflammation

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

relationship of LJN452 on liver fat content by changes in

quantitative MRI; determine effect of LJN452 on liver fibrosis

by biopsy

Proportion of patients with resolution of NASH and no

worsening of fibrosis OR improvement in fibrosis by at least

one stage without worsening of NASH at Week 48

compared with baseline

Arms/Intervention • Multiple LJN452 doses and placebo

• Arm A: combination therapytropifexor + licogliflozin

• Arm B: tropifexor monotherapytropifexor (+ licogliflozin

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

Publication

• Primary (interim) data abstract submitted to AASLD in

Q3-2019

• Manuscript to be submitted in Q4-2020

Planned in H1-2023


LOU064 – Bruton's tyrosine kinase (BTK) inhibitor

78

Study NCT03926611 (CLOU064A2201) NCT04109313 (CLOU064A2201E1)

Indication Chronic spontaneous urticaria (CSU) Chronic spontaneous urticaria (CSU)


Patients 308 250

Primary Outcome

Measures

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

4• Long-term safety and tolerability

Arms/Intervention

• 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


• Arm 3 High dose of LOU064 orally in the morning (once daily) and


• 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-antihistaminesPatients with CSU who have participated in preceding

studies with LOU064

Expected Completion Q2-2021 2022

Publication TBD TBD


LJC242 - FXR agonist + CCR2/CCR5 inhibitor

79

Study NCT03517540 TANDEM (CLJC242A2201J)

Indication Non-alcoholic steatohepatitis

Phase Phase 2

Patients 193

Primary Outcome

Measures

• Evaluation of safety and tolerability of combination

therapy (tropifexor + cenicriviroc) by monitoring adverse

event profile, vital signs and laboratory parameters

Arms/Intervention

• Tropifexor

• Cenicriviroc

• Tropifexor + cenicriviroc

Target PatientsAdult patients with non-alcoholic steatohepatitis (NASH) and

liver fibrosis


Publication Manuscript to be submitted in H1-2021


QGE031 - Anti-IgEStudy 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

Measures

Establish dose-response relationship of QGE031 with respect to

achievement of complete hives response at week 12

Long-term safety; number of participants with treatment-

emergent adverse events

Arms/Intervention

• Ligelizumab 24mg q4wks for 20 weeks



• Ligelizumab 120mg single dose

• Omalizumab 300mg q4wks for 20 weeks

• Placebo q 4wks for 20 weeks

Ligelizumab 240 mg q4wks open label for 52 weeks

Target Patients

Adult patients with chronic spontaneous urticaria inadequately

controlled with H1-antihistamines at approved or increased doses,

alone or in combination with H2-antihistamines or leukotriene

receptor antagonists.

Adult patients with chronic spontaneous urticaria inadequately

controlled with H1-antihistamines at approved or increased

doses, alone or in combination with H2-antihistamines or

leukotriene receptor antagonists.


Publication

• Primary results: Presented at EAACI 2018, EADV 2018, and

GUF 2018; NEJM publication (3 Oct 2019);

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

EADV, PAAM, ACAAI, UCARE.

• Exploratory results presented/ planned in 2020: AAAAI,

EAACI, EADV, ACAAI; Encoring all at GUF

• Primary results: AAD 2019;

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

EADV, PAAM, ACAAI, UCARE

• Exploratory results presented/ planned in 2020: AAAAI,

EAACI, EADV, ACAAI; Encoring all at GUF

• 5 Manuscripts 2020: core results extension; angioedema;

sleep/work impairment/rescue medication; data visualization


QGE031 - Anti-IgE

Study NCT03437278 (CQGE031C2202) NCT04210843 (CQGE031C2302E1)

Indication Chronic spontaneous urticaria / Chronic idiopathic urticaria Chronic spontaneous urticaria


Patients 48 800

Primary Outcome

MeasuresChange in the 7 day Urticaria Activity Score (UAS7)

The proportion of subjects with well-controlled disease

(UAS7 ≤ 6) at week 12

Arms/Intervention

• Ligelizumab high dose q4wks for 24 weeks

• Ligelizumab low dose q4wks for 24 weeks

• Placebo / ligelizumab high dose q4wks for 8 / 16 weeks

• Ligelizumab Dose 1 and 3

• Ligelizumab Dose 2 and 3

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

spontaneous urticaria

Patients who completed studies CQGE031C2302,

CQGE031C2303, CQGE031C2202 or CQGE031C1301


Publication

• Study design was presented at PAAM (Peds Allergy &

Asthma Meeting) and at UCARE meeting 2019

• Baseline characteristics 2020/21

• Primary results to be presented in late 2021/2022 (e.g.

EAACI, PAAM, EADV)

• Manuscript to be submitted in 2022

Study design presented at 2020 EAACI


QGE031 - Anti-IgE

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

Indication Chronic spontaneous urticaria Chronic spontaneous urticaria



Primary Outcome

Measures

Absolute change from baseline in UAS7 (Urticaria Activity

Score) at week 12

Absolute change from baseline in UAS7 (Urticaria Activity

Score) at week 12

Arms/Intervention

• Ligelizumab dose A q4w for 52 weeks

• Ligelizumab dose B q4w for 52 weeks

• Omalizumab 300 mg q4w for 52 weeks

• Placebo q4w from randomization to wk20, then

ligelizumab dose B from wk24 to wk52

• Ligelizumab dose A q4w for 52 weeks

• Ligelizumab dose B q4w for 52 weeks

• Omalizumab 300 mg q4w for 52 weeks

• Placebo q4w from randomization to wk20, then

ligelizumab dose B from wk24 to wk52

Target PatientsAdolescents and adults with chronic spontaneous urticaria

inadequately controlled with H1-antihistamines

Adolescents and adults with chronic spontaneous urticaria

inadequately controlled with H1-antihistamines

Expected Completion H2-2021 H2-2021

Publication

• Study design presented at UCARE 2018

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

• Manuscript to be submitted in 2022


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

Measures

Safety and efficacy of VAY736 in primary Sjögren's

syndrome (pSS)Alanine aminotransferase (ALT) normalization

Arms/Intervention• VAY736

• Placebo

• VAY736

• Placebo control with conversion to active VAY736

Target PatientsPatients with moderate to severe primary Sjögren's

syndrome (pSS)

Autoimmune hepatitis patients with incomplete response or

intolerant to standard treatment of care

Expected Completion Q2-2020 (actual) 2023

Publication • Manuscript to be submitted in 2020 TBD


Neuroscience

Aimovig® – CGRP receptor antagonist

85

Study NCT03096834 LIBERTY (CAMG334A2301) NCT03333109 EMPOWER (CAMG334A2302)

Indication Migraine Migraine


Patients 246 900

Primary Outcome

Measures

Percentage of patients with a 50% response in the reduction

of Monthly Migraine Days (MMD)

Change from baseline in monthly migraine days at the last

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

Arms/Intervention• Subcutaneous injection of AMG334 (erenumab)

• Subcutaneous injection of placebo

• AMG334 (erenumab) Dose 1

• AMG334 (erenumab) Dose 2

• Placebo

Target PatientsAdult episodic migraine patients who have failed prophylactic

migraine treatmentsAdult episodic migraine patients

Expected Completion 2017 DBT phase (actual); H1-2021 OLE phase (final DBL) Q1-2020 (actual)

Publication

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

prespecified subgroup analysis (DBT phase) submitted

to JNNP in June 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

Planned for H2-2020


Aimovig® – CGRP receptor antagonist

86

Study NCT03867201 DRAGON (CAMG334A2304)

Indication Migraine

Phase Phase 3

Patients 550

Primary Outcome

Measures

Change from baseline in monthly migraine days during the

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


Gilenya® - S1P-R modulator

87

Study NCT01633112 ASSESS (CFTY720D2312)

Indication Relapsing remitting multiple sclerosis (RRMS)

Phase Phase 3B

Patients 1,064

Primary Outcome

Measures

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

to glatiramer acetate (20 mg) in reducing the annualized

relapse rate up to 12 months

Arms/Intervention

• Fingolimod 0.5 mg orally

• Fingolimod 0.25mg orally

• Copaxone® 20 mg s.c.

Target Patients Patients with relapsing-remitting multiple sclerosis

Expected Completion 2018 (actual)

Publication

• Primary data presentation at AAN in 2019

• Primary manuscript accepted by JAMA Neurology in

June 2020


LMI070 - SMN2 RNA splice modulator

88

Study NCT02268552 (CLMI070X2201)

Indication Type 1 spinal muscular atrophy

Phase Phase 1/2

Patients 39

Primary Outcome

Measures

Number of participants with adverse events (AEs), serious

adverse events (SAEs) and deaths

Arms/Intervention

Branaplam oral, once weekly:

• Part 1: 5 ascending doses

• Part 2: 2 different dose levels

• Part 3: patients continue on initial dose assigned in Part

1 or Part 2

Target PatientsPatients with type 1 spinal muscular atrophy

Expected Completion Q3-2020 (Part 2)

Publication TBD


Mayzent ® - S1P-R modulator

89

Study NCT01665144 -EXPAND (CBAF312A2304)

Indication Secondary progressive multiple sclerosis

Phase Phase 3

Patients 1,652

Primary Outcome MeasuresThe delay in time to confirmed disability progression as

measured by EDSS (Expanded Disability Status Scale)

Arms/Intervention

• BAF312 (5-day titration: 0.25mg to 1.25mg; Maintenance

dose: 2mg (day 6))

• Placebo

Target Patients Patients with secondary progressive multiple sclerosis

Expected Completion Core in 2016/Extension in 2024

Publication

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


https://doi.org/10.1016/S0140-6736(18)30475-6

OMB157 - Anti-CD20

90

Study NCT02792218 Asclepios I (COMB157G2301) NCT02792231 Asclepios II (COMB157G2302)

Indication Multiple sclerosis Multiple sclerosis


Patients 900 900

Primary Outcome

Measures

Annualized Relapse Rate (ARR) - number of confirmed

relapses in a year calculated based on cumulative number

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

Annualized Relapse Rate (ARR) - number of confirmed

relapses in a year calculated based on cumulative number

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

Arms/Intervention• Ofatumumab subcutaneous

• Teriflunomide oral

• Ofatumumab subcutaneous

• Teriflunomide oral

Target Patients Patients with relapsing forms of multiple sclerosis Patients with relapsing forms of multiple sclerosis

Expected Completion Q3-2019 (actual) Q3-2019 (actual)

Publication Primary manuscript planned in H1-2020 Primary manuscript planned in H1-2020


OMB157 - Anti-CD20

91

Study NCT03249714 APOLITOS (COMB157G1301) NCT03650114 ALITHIOS (COMB157G2399)

Indication Multiple sclerosis Multiple Sclerosis


Patients 60 2010

Primary Outcome

Measures

Reduced cumulative number of Gd-enhanced T1 lesions

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

vs placebo)

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

• Placebo• Ofatumumab 20 mg every 4 weeks

Target Patients Patients with relapsing forms of multiple sclerosis Patients with relapsing MS

Expected Completion Q1-2020 (actual)2028

Publication TBD TBD


Zolgensma® - SMN1 gene replacement therapy

92

Study NCT03461289 STRIVE-EU (CL-302) NCT03306277 STRIVE (CL-303)

Indication Type 1 spinal muscular atrophy Type 1 spinal muscular atrophy


Patients 33 22

Primary Outcome

MeasuresProportion of participants sitting without support

• Achievement of independent sitting for at least 30

seconds

• 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



93

Study NCT03505099 SPR1NT (CL-304) NCT03837184 STRIVE Asia Pacific (CL-306)

Indication Spinal muscular atrophy Type 1 spinal muscular atrophy


Patients 30 6

Primary Outcome

Measures

• [2 copies of SMN2] Percentage of participants achieving

functional independent sitting for at least 30 seconds at

any visit

• [3 copies of SMN2] Percentage of participants achieving

the ability to stand without support for at least 3 seconds

at any visit

Proportion of participants sitting without support

Arms/Intervention Open-label, single-arm, single-dose, intravenous Open-label, single-arm, single-dose, intravenous

Target PatientsPre-symptomatic patients with spinal muscular atrophy and

multiple copies SMN2Patients with spinal muscular atrophy Type 1

Expected Completion H2-2021 H2-2021

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



94

Study NCT03381729 STRONG (CL-102)

Indication Type 2 spinal muscular atrophy

Phase Phase 1

Patients 51

Primary Outcome

Measures

• Safety and tolerability, incidence of adverse events

• Proportion of patients achieving Standing Milestone

• 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

Oncology

ABL001 – Specific, allosteric Bcr-Abl kinase inhibitor


Study NCT03106779 ASCEMBL (CABL001A2301)

Indication Chronic myeloid leukaemia (CML)

Phase Phase 3

Patients 233

Primary Outcome

MeasuresMajor Molecular Response (MMR) rate at 24 weeks

Arms/Intervention• ABL001 40 mg bid

• Bosutinib 500 mg

Target Patients

Patients with chronic myelogenous leukemia in chronic

phase, previously treated with 2 or more tyrosine kinase

inhibitors


Publication• Manuscript submission Q4-2020

• Abstract submission to congress Q3-2020

ACZ885 – IL-1β inhibitor


Study NCT03447769 CANOPY-A (CACZ885T2301) NCT03631199 CANOPY-1 (CACZ885U2301)

Indication Adjuvant NSCLC 1st Line Non-small cell lung cancer (NSCLC)


Patients 1,500 627

Primary Outcome

Measures

Disease free survival (primary), overall survival (key

secondary)

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

• Double-blind, randomized, placebo-controlled part:

Progression free survival (PFS)

• Overall survival (OS)

Arms/Intervention• Canakinumab 200mg q3w sc for 18 cycles

• Placebo q3w sc for 18 cycles

• Canakinumab or matching placebo in combination with

pembrolizumab and platinum-based doublet

chemotherapy

Target Patients

Patients with:

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

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

care adjuvant cisplatin-based chemotherapy

• All histologies

Patients with

• Histologically confirmed Stage IIIB, IV NSCLC with no

prior systemic anticancer therapy

• Squamous and non-squamous NSCLC

• No EGFR mutation and ALK rearrangement

Expected Completion Interim Analysis: 2022; Final: 2023 Interim Analysis: Q4-2020; Final: 2022

Publication TBD

Johnson B et al. Presented at AACR-NCI-EORTC 2019

(safety run-in)

Manuscript submission Q4-2020 (safety run-in)

Abstract submission to congress H1-2021

ACZ885 – IL1β inhibitor


Study NCT03626545 CANOPY-2 (CACZ885V2301)

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

Phase Phase 3

Patients 240

Primary Outcome

Measures

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

• Double-blind, randomized, placebo-controlled part:

Overall Survival

Arms/Intervention

• canakinumab in combination with docetaxel

• canakinumab matching-placebo in combination with

docetaxel

Target Patients

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

Expected Completion H1-2021

Publication Abstract submission to congress H1-2021

BYL719 - Alpha-specific PI3K inhibitor

99

Study NCT02437318 SOLAR-1 (CBYL719C2301) NCT04251533 EPIK-B3 (CBYL719H12301)

Indication HR+/HER2- advanced breast cancer with PIK3CA mutation Triple negative breast cancer


Patients 572 566

Primary Outcome

Measures

Progression-free survival (PFS) for patients with PIK3CA

mutant status

Progression-free Survival (PFS) for patients with PIK3CA

mutant status

Arms/Intervention• Fulvestrant 500 mg + alpelisib 300 mg

• Fulvestrant 500 mg + placebo

• Alpelisib 300 mg + nab-paclitaxel 100 mg/m²

• Placebo + nab-paclitaxel 100 mg/m²

Target Patients

Men and postmenopausal women with hormone receptor

positive, HER2-negative advanced breast cancer which

progressed on or after aromatase inhibitor treatment

Patients with advanced triple negative breast cancer with

either Phosphoinositide-3-kinase Catalytic Subunit Alpha

(PIK3CA) mutation or Phosphatase and Tensin Homolog

Protein (PTEN) loss without PIK3CA mutation

Expected Completion 2018 (actual) 2023

Publication

• Andre F, et al. Presentation at ESMO 2018

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

1940.

TBD


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

100

Study NCT00940602 TELESTO (CICL670A2302)

Indication Iron overload

Phase Phase 2

Patients 224

Primary Outcome

Measures

To compare deferasirox to placebo with regard to event-free

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

iron overload

Arms/Intervention• Deferasirox, iron chelator

• Placebo

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

transfusional iron overload


Publication

• Angelucci E, et al. Presentation at ASH 2018

• Angelucci E, et al. Manuscript Ann Intern Med

2020;172:513-522.


INC280 - MET Inhibitor

101

Study NCT02414139 (CINC280A2201)

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

Cancer (NSCLC)

Phase Phase 2

Patients 364

Primary Outcome

MeasuresOverall Response Rate (ORR)

Arms/Intervention

• 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

• 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 PatientsAdult patients with EGFR wild-type (wt), ALK-negative advanced/

metastatic NSCLC with either MET amplification or MET mutations


Publication

• 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


Jakavi® - JAK1/2 inhibitor

102

Study NCT02913261 REACH2 (CINC424C2301) NCT03112603 REACH3 (CINC424D2301)

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


Patients 310 330

Primary Outcome

MeasuresOverall Response Rate (ORR) at 28 Days Overall Response Rate (ORR) at 183 Days

Arms/Intervention• Ruxolitinib 10mg bid

• Best available therapy (BAT)

• Ruxolitinib 10mg bid

• Best available therapy (BAT)

Target Patients Patients with SR aGVHD Patients with SR cGVHD

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

Publication

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

1810.

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

EBMT Q3-2020

• Manuscript submission in H2-2020

• Abstract submission to congress in H2-2020



103

Study NCT03491215 REACH4 (CINC424F12201) NCT03774082 REACH5 (CINC424G12201)

Indication Acute graft versus host disease Chronic graft versus host disease


Patients 45 42

Primary Outcome

Measures

• Measurement of PK parameters

• Overall Response Rate (ORR) • Overall Response Rate (ORR)

Arms/Intervention • Ruxolitinib• Ruxolitinib 5mg tablets / pediatric formulation

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

after allogeneic hematopoietic stem cell transplantation

Pediatric subjects with moderate and severe chronic Graft

vs. Host disease after allogeneic stem cell transplantation


Publication TBD TBD



104

Study NCT04097821 ADORE (CINC424H12201)

Indication Myelofibrosis

Phase Phase 1/2

Patients 130

Primary Outcome

Measures

• Incidence of dose limiting toxicities within the first 2

cycles

• Response rate at the end of cycle 6

Arms/Intervention

• Ruxolitinib

• Ruxolitinib+Siremadlin

• Ruxolitinib+Crizanlizumab

• Ruxolitinib+MBG453

Target Patients Patients with Myelofibrosis (MF)


Publication TBD


Kisqali® - CDK 4/6 inhibitor

105

Study NCT03701334 NATALEE (CLEE011O12301C)

IndicationAdjuvant treatment of hormone receptor (HR)-positive,

HER2-negative, early breast cancer (EBC)

Phase Phase 3

Patients ~4,000

Primary Outcome

Measures

Invasive Disease-Free Survival for using STEEP criteria

(Standardized Definitions for Efficacy End Points in adjuvant

breast cancer trials)

Arms/Intervention• Ribociclib + endocrine therapy

• Endocrine therapy

Target Patients

Pre and postmenopausal women and men with HR-positive,

HER2-negative EBC, after adequate surgical resection, who

are eligible for adjuvant endocrine therapy

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

Publication TBD


Kymriah® – CAR-T therapy

106

Study NCT03568461 ELARA (CCTL019E2202) NCT03876769 CASSIOPEIA (CCTL019G2201J)

Indication Relapsed / refractory follicular lymphoma (FL) 1st line high risk acute lymphoblastic leukemia (ALL)


Patients 113 160

Primary Outcome

MeasuresComplete Response Rate (CRR) Disease Free Survival (DFS)

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

Publication

• ELARA interim analysis – ASH 2020, tbc

• ELARA primary analysis – Planned for ASCO/EHA 2021

• ELARA vs RECORD full analysis – Planned for

ASCO/EHA 2021

• ELARA vs Flatiron – Planned for ASCO/EHA 2021

• ELARA: Primary analysis MS – simultaneous publication

with congress

• ELARA vs RECORD: Full analysis – simultaneous

publication with congress

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

submission H1-2022


Kymriah® – CAR-T therapy

107

Study NCT03570892 BELINDA (CCTL019H2301)

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

Phase Phase 3

Patients 318

Primary Outcome

MeasuresEvent-free Survival (EFS)

Arms/Intervention Tisagenlecleucel versus standard of care

Target Patients

Adult patients with aggressive B-cell Non-Hodgkin

Lymphoma after failure of rituximab and anthracycline-

containing frontline immunochemotherapy


Publication

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

SITC 2019, Bishop et al abstract planned to DGHO

2020; BELINDA TiP

• Primary analysis Planned for ASH 2021

• Primary manuscript – TBD


MBG453 – TIM-3 antagonist

108

Study NCT03946670 STIMULUS MDS-1 (CMBG453B12201) NCT04266301 STIMULUS-MDS2 (CMBG453B12301)

Indication Myelodysplastic syndrome Myelodysplastic syndrome


Patients 120 500

Primary Outcome

Measures

Complete Remission (CR) rate and Progression Free

Survival (PFS)Overall survival

Arms/Intervention• Experimental: MBG453 + hypomethylating agents

• Placebo comparator: Placebo + hypomethylating agents

• MBG453 800 mg + azacitidine 75 mg/m2

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

Target PatientsAdult subjects with intermediate, high or very high risk

Myelodysplastic Syndrome (MDS) as per IPSS-R criteria

Patients with intermediate, high or very high risk

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

Chronic Myelomonocytic Leukemia-2 (CMML-2)


Publication Abstract submission to congress in H2-2021 TBD


PDR001 – PD-1 checkpoint inhibitor

109

Study NCT02967692 COMBI-i (CPDR001F2301)

Indication BRAFV600 mutant metastatic melanoma

Phase Phase 3

Patients

538

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

(Phase III, randomized, placebo controlled): 532

Primary Outcome

MeasuresProgression-Free Survival (PFS)

Arms/Intervention

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

Mekinist 2 mg

• Placebo + Tafinlar 150 mg bid + Mekinist 2 mg

Target Patients

Previously untreated patients with unresectable or

metastatic BRAF V600 mutant melanoma


Publication• Abstract submission to congress in Q3-2020

• Manuscript submission Q3/Q4-2020


PDR001 - PD-1 checkpoint inhibitor

Study NCT03484923 (CPDR001J2201)

Indication Previously treated unresectable or metastatic melanoma

Phase Phase 2

Patients 195

Primary Outcome

MeasuresObjective Response Rate (ORR)

Arms/Intervention

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

unselected patients and LAG-3 positive patients)

• PDR001 400mg i.v. Q4W + capmatinib

• PDR001 400mg i.v. Q4W + canakinumab

• PDR001 400mg i.v. Q4W + ribociclib

Target PatientsAdult patients with previously treated unresectable or

metastatic melanoma


Publication TBD


Promacta®/Revolade® – Thrombopoetin receptor agonist

Study NCT03025698 (CETB115E2201) NCT03988608 (CETB115E2202)

IndicationPreviously untreated or relapsed/refractory severe aplastic

anemia or recurrent aplastic anemia

Previously untreated or relapsed/refractory severe aplastic

anemia or recurrent aplastic anemia


Patients 60 20

Primary Outcome

Measures

PK of eltrombopag at steady state in pediatric patients with

SAAHematologic response rate

Arms/Intervention

• Eltrombopag 12.5, 25, 50, 75 mg FCT & 25 mg pFOS

• Arm B: previously untreated SAA-hATG/cyclosporine +

eltrombopag

• Arm A: relapsed/refractory SAA or AA:

hATG/cyclosporine + eltrombopag or cyclosporine +

eltrombopag

• Eltrombopag 25 mg film-coated tablets

Target Patients

Pediatric patients from age 1 <18 years with

relapsed/refractory SAA or recurrent AA after IST or

previously untreated SAA

Chinese patients with refractory or relapsed severe aplastic

anemia


Publication TBD TBD


Rydapt®- Multi-targeted kinase inhibitor

112

Study NCT03280030 (CPKC412A2220) NCT03591510 (CPKC412A2218)

Indication Acute myeloid leukemia Acute myeloid leukemia


Patients 66 50

Primary Outcome

MeasuresIncidence of safety events and event free survival

Occurrence of dose limiting toxicities

Event Free Survival ( EFS)

Arms/Intervention• Midostaurin 50 mg

• Placebo• Chemotherapy followed by Midostaurin

Target PatientsNewly diagnosed patients with FLT3-mutated acute myeloid

leukemia (AML) from pan-Asia countries

Newly diagnosed pediatric patients with FLT3 mutated acute

myeloid leukemia (AML)

Expected Completion Interim analysis: Q2-2020 (actual); Final: H2-2021 2022

Publication Abstract submission to congress in Q4-2020 TBD


SEG101 – p-Selectin inhibitor

Study NCT03264989 SOLACE-Adults (CSEG101A2202) NCT03474965 SOLACE-Kids (CSEG101B2201)

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

Sickle Cell Disease (SCD) Prevention of VOC in pediatric patients with SCD


Patients 55 100

Primary Outcome

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

Arms/Intervention

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

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

Hydroxyurea/Hydroxycarbamide

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

± Hydroxyurea/Hydroxycarbamide

Target Patients Adult SCD patients with VOC Pediatric SCD patients with VOC

Expected Completion 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


SEG101 – p-Selectin inhibitor

Study NCT03814746 STAND (CSEG101A2301)

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

Sickle Cell Disease (SCD)

Phase Phase 3

Patients 240

Primary Outcome

MeasuresRate of VOC events leading to healthcare visit

Arms/Intervention

• Crizanlizumab 5.0 mg/kg

• Crizanlizumab 7.5 mg/kg

• Placebo

Target Patients Adolescent and adult SCD patients (12 years and older)


Publication TBD


Study NCT01677741 (CDRB436A2102)

Indication BRAFV600 mutant cancers

Phase Phase 1/2

Patients 85

Primary Outcome

MeasuresSafety, tolerability and pharmacokinetics

Arms/InterventionSingle-arm study of oral dabrafenib (dose based on age

and weight)

Target PatientsPediatric subjects aged 1 year to <18 years with advanced

BRAF V600-mutation positive solid tumors


Publication

• Kieran MW et al. Manuscript Clin Cancer Res

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

• Hargrave DR et al. Manuscript Clin Cancer Res

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

gliomas)

Tafinlar® - BRAF inhibitor


Tafinlar®+Mekinist® - BRAF inhibitor and MEK inhibitor

Study NCT02684058 (CDRB436G2201)

Indication BRAFV600 mutant gliomas

Phase Phase 2

Patients 142

Primary Outcome

MeasuresObjective response rate

Arms/Intervention Dabrafenib + trametinib (dose based on age and weight)

Target Patients

Children and adolescent patients with BRAF V600 mutation

positive relapsed or refractory high grade glioma (HGG) or

BRAF V600 mutation positive low grade glioma (LGG)


Publication TBD


Tafinlar®+Mekinist® - BRAFV600 inhibitor and MEK inhibitor

Study NCT02124772 (CTMT212X2101)

Indication BRAFV600 mutant solid tumors

Phase Phase 1/2A

Patients 139

Primary Outcome

MeasuresSafety, tolerability and pharmacokinetics and clinical activity

Arms/InterventionTrametinib (dose based on age and weight)

Dabrafenib + trametinib (dose based on age and weight)

Target PatientsPediatric Subjects Aged 1 Month to <18 Years with

Advanced V600-Mutation Positive Solid Tumors


Publication• Geoerger B, et al. Presentation at ASCO 2020

• Manuscript submission Q4-2020


Zykadia® - ALK inhibitor


Study NCT02299505 ASCEND-8 (CLDK378A2112)

Indication ALK activated NSCLC

Phase Phase 2

Patients 306

Primary Outcome

Measures

Part 1: Pharmacokinetics when taken with food

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

Arms/Intervention

• Oral LDK378 450 mg once daily taken with food

• Oral LDK378 600 mg once daily taken with food

• Oral LDK378 750 mg once daily fasted

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

lung cancer

Expected Completion

Part 1 (PK): 2016 (actual)

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

Final (ORR): Q3-2020

Publication

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

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

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

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


Study NCT03511664 VISION (PSMA-617-01)

IndicationPSMA-positive Metastatic Castration-resistant Prostate

Cancer (mCRPC)

Phase Phase 3

Patients 831

Primary Outcome

Measures

• Radiographic Progression Free Survival

• Overall Survival

Arms/Intervention• 177Lu-PSMA-617 plus BS/BSC

• BS/BSC alone

Target Patients

Adult patients with PSMA-positive Metastatic Castration-

resistant Prostate Cancer (mCRPC)


Publication TBD

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

Measures• Progression Free Survival

Arms/Intervention• Lutathera plus long-acting octreotide

• high dose long-acting octreotide

Target Patients

Adult patients with Grade 2 and Grade 3 Advanced GEP-

NET


Publication TBD

Ophthalmology

Lucentis® - Anti-VEGF

122

Study NCT02375971 RAINBOW (CRFB002H2301) NCT02640664 RAINBOW Extension (CRFB002H2301E1)

Indication Retinopathy of Prematurity (ROP) Retinopathy of Prematurity (ROP)


Patients 224 180

Primary Outcome

Measures

Absence of active Retinopathy of Prematurity (ROP) and

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

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

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

structural outcome

To evaluate the visual function of patients by assessing the

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

birthday.

Arms/Intervention

• Ranibizumab 0.2 mg (up to 3 injections max)

• Ranibizumab 0.1 mg (up to 3 injections max)

• Laser therapy

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

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

Target PatientsMale and female preterm infants with bilateral retinopathy of

prematurity (ROP) who require treatment.

Male and female preterm infants with bilateral retinopathy of

prematurity (ROP) who completed RAINBOW.

Expected Completion 2018 (actual) 2023

Publication

• EURETINA: Sep-2018

• AAO: Oct-2018

• Primary manuscript published online by The Lancet in

Sep-2019

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

-6736(19)31344-3.pdf)

• Submission of manuscript on Pop PK/PD analysis in

2020

• Submission of manuscript on time-course of clinical

response to treatment in 2020

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

in 2020


RTH258 - Anti-VEGF

Study NCT02434328 HARRIER (CRTH258A2302) NCT02307682 HAWK (CRTH258A2301)

Indication Neovascular age-related macular degeneration (nAMD) Neovascular age-related macular degeneration (nAMD)


Patients 743 1,082

Primary Outcome

Measures

Change in Best Corrected Visual Acuity (BCVA) from

baseline at week 48

Change in Best Corrected Visual Acuity (BCVA) from

baseline at week 48

Arms/Intervention• Brolucizumab (RTH258) 6 mg/50 µL

• Aflibercept 2 mg/50 µL

• Brolucizumab (RTH258) 3 mg/50 µL



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


Publication

• 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.

• 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


RTH258 - Anti-VEGF

Study NCT03386474 (CRTH258A2301E1) NCT03481634 KESTREL (CRTH258B2301)

Indication Neovascular age-related macular degeneration (nAMD) Diabetic eye disease


Patients 150 534

Primary Outcome

MeasuresNumber of treatment-emergent adverse events

Change from baseline in best-corrected visual acuity

(BCVA)





• Aflibercept 2mg/50 uL

Target PatientsPatients with neovascular age-related macular degeneration

who have completed the CRTH258A2301 study

Patients with visual impairment due to diabetic macular

edema (DME)

Expected Completion 2018 (actual) H2-2021

PublicationPlanned 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)


RTH258 - Anti-VEGF

Study NCT03481660 KITE (CRTH258B2302) NCT04058067 KINGLET (CRTH258B2304)

Indication Diabetic eye disease Diabetic macular edema


Patients 356 268

Primary Outcome

Measures


(BCVA)Change in best-corrected visual acuity (BCVA)





Target PatientsPatients with visual impairment due to diabetic macular

edema (DME)

Chinese patients with visual impairment due to diabetic

macular edema


PublicationWeek 52 safety and efficacy data to be submitted as an

abstract H1 2021 (KITE and KESTREL)Publication planned for 2023


RTH258 - Anti-VEGF

Study NCT03917472 KINGFISHER (CRTH258B2305) NCT03802630 RAPTOR (CRTH258C2301)

Indication Diabetic macular edema Retinal vein occlusion


Patients 500 500

Primary Outcome

Measures

Change in best-corrected visual acuity (BCVA) from

baseline up to week 52


(BCVA) at week 24





Target PatientsPatients with visual impairment due to diabetic macular

edema

Adult patients with visual impairment due to macular edema

secondary to branch retinal vein occlusion


Publication Publication submission planned for 2022 Publication submission planned for 2023


RTH258 - Anti-VEGF

Study NCT03810313 RAVEN (CRTH258C2302) NCT04047472 HOBBY (CRTH258A2307)

Indication Retinal vein occlusion Macular degeneration


Patients 750 494

Primary Outcome

Measures


(BCVA) at week 24


(BCVA) at week 48





Target PatientsAdult patients with visual impairment due to macular edema

secondary to central retinal vein occlusion

Chinese patients with neovascular age-related macular

degeneration


Publication TBD TBD


UNR844 - Disulfide bonds modulator

Study NCT03809611 (CUNR844A2203)

Indication Presbyopia

Phase Phase 2

Patients 124

Primary Outcome

Measures

Change in binocular distance-corrected near visual acuity

(DNCVA) from baseline at month 3

Arms/Intervention• 1.5% solution UNR844-Cl

• Placebo

Target Patients Patients with presbyopia


Publication Expected at AAOptom


Respiratory

INC424 - JAK Inhibitor

130

Study NCT04362137 RUXCOVID (CINC424J12301)

Indication COVID-19 (cytokine storm)

Phase Phase 3

Patients 402

Primary Outcome Measures

Proportion of patients who die, develop respiratory failure

(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


QBW251 - CFTR potentiator

131

Study NCT04072887 (CQBW251B2201)

Indication Chronic obstructive pulmonary disease (COPD)

Phase Phase 2

Patients 956

Primary Outcome

Measures

Trough FEV1 (Forced Expiratory Volume in 1 second)

change from baseline after 12 weeks of treatment

Arms/Intervention

• QBW251 450 mg

• QBW251 300 mg

• QBW251 150 mg

• QBW251 75 mg

• QBW251 25 mg

• Placebo

Target PatientsCOPD patients on background triple inhaled therapy (LABA /

LAMA / ICS)


Publication TBD


QMF149 - Long-acting beta2 agonist and inhaled corticosteroid

132

Study NCT02892019 (CQMF149G2202)

Indication Asthma

Phase Phase 2

Patients 80

Primary Outcome

MeasuresTrough FEV1

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


Publication TBD


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

Study NCT02554786 PALLADIUM (CQVM149B2301) NCT02571777 IRIDIUM (CQVM149B2302)

Indication Asthma Asthma



Primary Outcome

MeasuresTrough FEV1 Trough FEV1

Arms/Intervention

• QMF149 150/160 µg od

• QMF149 150/320 µg od

• MF 400 µg od

• MF 400 µg bid

• Salmeterol 50 µg /fluticasone 500 µg bid

• QVM149 150/50/160 µg od

• QVM149 150/50/80 µg od

• QMF149 150/160 µg od

• QMF149 150/320 µg od

• Salmeterol 50 µg /fluticasone 500 µg bid

Target Patients

Adult and adolescent (≥12 years) patients with asthma

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

dose LABA/ICS (GINA step ≥ 3)

Adult (≥18 years) patients with asthma inadequately

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


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

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



134

Study NCT03100500 (CQVM149B1305) NCT03100825 (CQVM149B1304)



Patients 51 94

Primary Outcome

Measures

Long-term safety/tolerability: Incidence and severity of

treatment emergent adverse events during the 52 weeks

study

Long-term safety/tolerability: Incidence and severity of

treatment emergent adverse events during the 52 weeks

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


Publication

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

Q4 2020

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

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

Q4 2020

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



Study NCT02892344 QUARTZ (CQVM149B2303) NCT03158311 ARGON (CQVM149B2306)



Patients 802 1,251

Primary Outcome

MeasuresTrough FEV1

Non-inferiority of Asthma Quality of Life Questionnaire

(AQLQ)

Arms/Intervention• QMF149 150/80 µg od

• MF 200 µg od

• QVM149 150/50/80 μg od

• QVM149 150/50/160 μg od

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

Target Patients

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

inadequately controlled on low-dose ICS or low-dose

LABA/ICS (Gina step 2-3)

Patients with uncontrolled asthma


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

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

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

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


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

Xolair ® – anti-IgE antibody

136

Study NCT03369704 (CIGE025F1301)

Indication Seasonal allergic rhinitis: Severe Japanese Cedar Pollinosis

Phase Phase 3

Patients 337

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

Arms/Intervention

In addition to standard of care:

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

• Placebo

Target PatientsPatients with severe Japanese cedar pollinosis, whose symptoms were inadequately controlled with current recommended

therapies


Publication

• 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


Sandoz Biopharmaceuticals

Hyrimoz® - Biosimilar adalimumab

138

Study NCT02744755 ADMYRA (GP17-302)

Indication Immunology

Phase Phase 3

Patients 353

Primary Outcome

Measures

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

patients treated with GP2017 and patients treated with

Humira®

Arms/Intervention• GP2017

• US licensed Humira® adalimumab

Target Patients Patients with moderate to severe active rheumatoid arthritis


Publication

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

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


GP2411 - Biosimilar denosumab

139

Study NCT03974100 (CGP24112301)

Indication Osteoporosis

Phase Phase 3

Patients 522

Primary Outcome

Measures

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

Mineral Density

Arms/Intervention

• GP2411 60 mg /mL subcutaneous injection every 6

months

• Prolia® 60 mg /mL subcutaneous injection every 6

months

Target Patients Postmenopausal women with osteoporosis


Publication

Study data publications expected for 2024 and beyond. The

overall study design will be published at WCO and ECTS

congresses 2020.


Global Health

KAF156 – Plasmodium Falciparum Inhibitor – PfCARL mediated

141

Study NCT03167242 (CKAF156A2202)

Indication Malaria

Phase Phase 2

Patients ~500

Primary Outcome

Measures

PCR-corrected adequate clinical and parasitological

response (ACPR)

Arms/Intervention• KAF156 and LUM-SDF (different combinations)

• Coartem

Target PatientsAdults and children with uncomplicated Plasmodium

Falciparum Malaria


Publication TBD


KAE609 – Plasmodium Falciparum Inhibitor – spiroindolone against PfATP4

142

Study NCT03334747 (CKAE609A2202)

Indication Malaria

Phase Phase 2

Patients 186

Primary Outcome

Measures

CTCAE grades increase from baseline in alanine

aminotransferase (ALT) or aspartate aminotransferase

(AST)

Arms/Intervention• KAE609

• Coartem

Target Patients Adults with uncomplicated Plasmodium Falciparum malaria


Publication TBD


Indication abbreviations


IPF Idiopathic pulmonary fibrosis

mCRPC Metastatic castration-resistant prostate cancer

MDR Multi-drug resistant

MDS Myelodysplastic syndrome

MS Multiple sclerosis

nAMD Neovascular (wet) age-related macular degeneration

NASH Non-alcoholic steatohepatitis

nHCM Non-obstructive hypertrophic cardiomyopathy

nr-axSpA Non-radiographic axial spondyloarthritis

NSCLC Non-small cell lung cancer

PDR Proliferative diabetic retinopathy

PEF Preserved ejection fraction

PNH Paroxysmal nocturnal haemoglobinuria

PsA H2H Psoriatic arthritis head-to-head study versus adalimumab

RCC Renal cell carcinoma

PROS PIK3CA related overgrowth spectrum

RA Rheumatoid arthritis

rMS Relapsing multiple sclerosis

ROP Retinopathy of prematurity

RP Retinitis pigmentosa

RVO Retinal vein occlusion

SAA Severe aplastic anemia

SjS Sjögren’s syndrome

SLE Systemic lupus erythematosus

SMA Type 1 Spinal muscular atrophy type 1 (IV formulation)

SMA Type 2/3 Spinal muscular atrophy type 2/3 (IT formulation)

SpA Spondyloarthritis

SPMS Secondary progressive multiple sclerosis

TNBC Triple negative breast cancer

T1DM Type 1 Diabetes melitus

AD Atopic Dermatitis

AIH Autoimmune hepatitis

aHUS atypical Hemolytic Uremic Syndrome

ALL Acute lymphoblastic leukemia

ALS Amyotrophic lateral sclerosis

AMI Acute myocardial infarction

AML Acute myeloid leukemia

AS H2H Ankylosing spondylitis head-to-head study versus adalimumab

BC Breast cancer

C3G C3 glomerulopathy

CCF Congestive cardiac failure

CLL Chronic lymphocytic leukemia

CML Chronic myeloid leukemia

CRC Colorectal cancer

COPD Chronic obstructive pulmonary disease

COSP Chronic ocular surface pain

CSU Chronic spontaneous urticaria

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

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

DME Diabetic macular edema

DLBCL Diffuse large B-cell lymphoma refractory

GCA Giant cell arteritis

GVHD Graft-versus-host disease

HCC Hepatocellular carcinoma

HFpEF Chronic heart failure with preserved ejection fraction

HF-rEF Chronic heart failure with reduced ejection fraction

HNSCC Head and neck squamous cell carcinoma

HS Hidradenitis suppurativa

IgAN IgA nephropathy

iMN Membranous nephropathy

Novartis Q2 2020 Results

Documents

Transcript of Novartis Q2 2020 Results